Noted This Week - 2016 Archive
Archive of cancer-related news briefs, by week, for 2016
December 30, 2016
- The FDA placed a clinical hold or partial clinical hold on early-stage trials of vadastuximab talirine (Seattle Genetics), an investigational antibody–drug conjugate targeting CD33, in patients with acute myeloid leukemia who had an allogeneic stem cell transplant, to evaluate the risk of hepatotoxicity. The holds were initiated after six patients were identified with hepatotoxicity and four patients died. Overall, more than 300 patients have been treated with vadastuximab talirine in multiple clinical trials, the company reported.
- Unlike adults, children with cancer treated with radiotherapy or chemotherapy are at high risk of developing cardiac and neurologic problems later in life, a disparity that has never been fully understood. In a new study in Cancer Cell, researchers found that mitochondria of many adult tissues are refractory to pro-apoptotic signaling, leading to cellular resistance to radiation and chemotherapy, whereas mitochondria from tissues in young mice and children are “primed for apoptosis, predisposing them to undergo cell death in response to genotoxic damage.” Using mouse models, the researchers show that apoptotic pathways can be selectively blocked, helping to prevent damage from cancer treatment in normal tissues.
- According to a phase I study reported in The New England Journal of Medicine, selumetinib (AstraZeneca), a selective inhibitor of MEK1/2, reduced the size of plexiform neurofibromas by at least 20% in 17 of 24 children with neurofibromatosis type 1. These partial responses were durable, lasting for a median of 23 cycles (about 2 years), and none of the trial participants experienced disease progression. Because surgical removal of all tumors is rarely feasible—and because tumors that are removed tend to regrow—researchers are seeking more effective treatments for the painful and disfiguring condition.
- The Wall Street Journal reported that President-elect Donald Trump’s nominee to lead the Department of Health and Human Services (DHHS), Rep. Tom Price, traded shares in pharmaceutical and biomedical companies while sponsoring or supporting legislation that could affect their price. The paper said that the Georgia Republican bought and sold stock valued at more than $300,000 in about 40 companies over the past 4 years. The FDA is part of the DHHS.
- The FDA released a guidance document for industry on clinical pharmacology data to support the demonstration of biosimilarity to a reference product. By law, companies seeking FDA approval of a biologic product that is highly similar to an existing one must show that there are no clinically meaningful differences between the products in terms of safety, purity, and potency. The guidance document discusses “concepts related to clinical pharmacology testing,” as well as “approaches for developing the appropriate clinical pharmacology database and the utility of modeling and simulation for designing clinical trials” to meet that requirement.
December 22, 2016
- The FDA granted accelerated approval to the PARP inhibitor rucaparib (Rubraca; Clovis Oncology) for women with advanced ovarian cancer who have been treated with at least two chemotherapies. Eligible patients also need to have deleterious germline or somatic BRCA mutations in their tumors as determined by FoundationFocus CDxBRCA, a companion diagnostic simultaneously approved by the agency. The FDA based its decision on data from two single-arm clinical trials, in which 54% of 106 patients given rucaparib experienced complete or partial tumor shrinkage, with the median duration of response being 9.2 months.
- In other PARP inhibitor news, niraparib (Tesaro) was granted Priority Review by the FDA for platinum-sensitive patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. In a phase III study of 553 patients with and without germline BRCA mutations, niraparib significantly prolonged progression-free survival by 15.5 months and 5.5 months, respectively, compared with placebo. A formal decision from the agency is expected by June 30, 2017.
- The FDA also issued a final guidance document on electronic informed consent in clinical research. The guidance answers 16 key questions, such as how to present details of informed consent to potential trial participants; how and where the process can take place; how to aid subjects in understanding the information; and what steps can be taken to ensure the privacy, security, and confidentiality of the electronic documents.
- According to former Speaker of the House Newt Gingrich, a Republican from Georgia, the Trump administration will be “very science- and technology-oriented.” In an interview with STAT News, Gingrich said he expects the administration to be “committed to FDA reform that will bring more capital into [developing] pharmaceuticals and biologics.” The president-elect and his team are seriously considering keeping Francis Collins, MD, PhD, as director of the NIH, Gingrich added—a move he himself would strongly endorse, because “I know of no one who has the breadth of knowledge of biological research that Collins has.”
- Cambridge, MA–based Merrimack Pharmaceuticals halted its phase II study of MM-302 in HER2-positive metastatic breast cancer, in which patients were randomly assigned to receive the HER2-targeted antibody–liposomal doxorubicin conjugate plus trastuzumab (Herceptin; Roche) or chemotherapy of physician’s choice plus trastuzumab. The median progression-free survival was shorter than expected in both treatment and control arms; an independent Data and Safety Monitoring Board review also concluded that MM-302 would be unlikely to demonstrate benefit over the comparator treatments.
- According to results from a phase III trial, vascular-targeted photodynamic therapy (VTP) is safe and effective for men with low-risk, localized prostate cancer. The study randomly assigned 413 patients to receive VTP—in which padeliporfin, a light-sensitive drug derived from deep-sea bacteria, is activated with a laser to destroy tumor cells—or active surveillance, a standard option for men with low-risk disease. At a median follow-up of 24 months, 49% of the VTP group were in remission, compared with 14% in the control arm; VTP was well tolerated overall, with urine retention being the most common side effect.
December 16, 2016
- President Obama signed the 21st Century Cures Act into law. The final legislation provides $1.8 billion over 7 years for cancer research, with $300 million available for the current fiscal year. Other significant provisions that will affect cancer research and drug development include a new program to support young investigators at the NIH, and an expansion of hiring authority at the FDA so the agency can better recruit and retain staff with the required scientific expertise.
- NIH Director Francis Collins, MD, PhD, said he would continue to lead the agency if asked. Collins told STAT News that he believes in public service and, particularly in light of the 21st Century Cures Act’s successful passage, would consider it a privilege to be invited to remain at the NIH’s helm. Last week, President-elect Donald Trump was urged to do just that by four Republican members of Congress.
- The FDA improved its Drug Safety Labeling Changes Program, making it easier and faster to obtain the latest safety information on approved drugs. Previously, health care professionals had to wait for a monthly release of all product-labeling updates from the FDA. Now, within days of the agency approving new safety information for a given drug, the labeling changes will be available in a publicly accessible database.
- A scant 1.8% of the $26.6 billion collected by state governments from tobacco settlement funds and tobacco taxes in FY 2017 will be spent on smoking cessation programs, according to a joint report from several health organizations. The group reported that although it would take less than 13% of total state tobacco revenues to fund such programs at levels recommended by the Centers for Disease Control and Prevention (CDC), only North Dakota and Alaska are currently doing so. Twenty-nine states and the District of Columbia are spending less than 20% of what the CDC recommends; New Jersey and Connecticut have allocated no state funds for tobacco prevention programs.
- Bristol-Myers Squibb (BMS) announced a major overhaul of its research and development (R&D) and manufacturing sites across the United States. Sites in Wallingford, CT; Hopewell, NJ; and Seattle, WA, will close within the next several years, but the company said in a brief statement that it expects “many of the roles from [the three facilities] will transition to other U.S. locations.” BMS also confirmed plans to “co-locate lab-based discovery and translational medicine activities” at a new R&D site that will be constructed on its campus in Lawrenceville, NJ.
- Natera, a genetic testing company based in San Carlos, CA, added its liquid biopsy expertise to I-SPY 2, a multicenter clinical trial evaluating the safety and efficacy of investigational neoadjuvant therapies for women with newly diagnosed, locally advanced breast cancer. I-SPY 2 has enrolled more than 1,000 patients, with investigators using traditional imaging methods to monitor tumor burden, treatment response, and residual disease. Now, they’ll incorporate serial analyses of circulating tumor DNA, using personalized blood tests created by Natera based on the molecular profile of each patient’s tumor.
December 9, 2016
- Like the House last week, the Senate readily passed the 21st Century Cures Act—by a vote of 94 to 5. The bill is now headed to the White House to be signed into law by President Obama. The Senate also voted to rename the portion of the bill that will provide $1.8 billion in funding for cancer research the “Beau Biden Cancer Moonshot,” in memory of Vice President Joe Biden’s son who died of brain cancer last year at age 46.
- Four Republican members of Congress wrote a letter urging President-elect Donald Trump to keep Francis Collins, MD, PhD, as director of the NIH. Along with the current administration’s other presidential appointees, Collins has said he will tender his resignation, effective January 20, 2017, but maintain his research lab at the NIH. However, Fred Upton (MI), Lamar Alexander (TN), Roy Blunt (MO), and Tom Cole (OK) argued that Collins “is the right person, at the right time, to continue to lead the world’s premier biomedical research agency.”
- Repealing the Affordable Care Act (ACA) will be the first order of business for the new Republican-controlled Congress when it convenes on January 3, Senate Majority Leader Mitch McConnell (KY) told Reuters. Although McConnell did not say when the repeal would go into effect, Senator John Barrasso (WY) suggested that it may be effective within 2 to 3 years. Senator John Thune (SD) added that the ACA will be replaced “step by step,” and that he hoped Democrats would cooperate in giving states, not the federal government, more control over healthcare issues.
- The FDA approved bevacizumab (Avastin; Genentech) combined with chemotherapy—carboplatin plus paclitaxel or gemcitabine—for women with recurrent platinum-sensitive ovarian cancer. The agency’s nod was based on positive data from two phase III studies, including one showing a 5-month improvement in overall survival among those who received the angiogenesis inhibitor. Bevacizumab is now approved for nine distinct uses across six cancer types, including HER2-negative breast cancer, non–small cell lung cancer (NSCLC), and glioblastoma.
- AstraZeneca and Novartis announced positive phase III results for osimertinib (Tagrisso) and ceritinib (Zykadia), respectively, in advanced NSCLC. Among patients with the EGFR T790M resistance mutation who were randomly assigned to receive osimertinib or standard platinum chemotherapy as second-line treatment, the EGFR inhibitor improved progression-free survival (PFS) by nearly 6 months. Meanwhile, among previously untreated patients with ALK-positive NSCLC who were randomly assigned to receive ceritinib or standard first-line chemotherapy, the ALK inhibitor more than doubled PFS—from 8.1 months to 16.6 months.
- According to Elsevier, China is rapidly becoming a powerhouse in cancer research, with more than 17% of the global share of cancer-related publications. This output matches that of the United States in 2005 and is driven by increased research and development spending along with “a shift from socialist economic planning to a more market-driven system over the past decade,” the science publisher stated in a new report. Elsevier is tracking the current state of cancer research worldwide on behalf of the National Cancer Moonshot; this report is the first in a series that will provide insights aimed at accelerating progress in the field.
December 2, 2016
- The House of Representatives overwhelmingly passed the 21st Century Cures Act by a vote of 392 to 26. The White House lauded the bill’s passage, emphasizing that it will help ensure continued funding for important health measures, including $4.8 billion for the National Cancer Moonshot, the Precision Medicine Initiative, and the BRAIN initiative. Next week, this legislation moves on to the Senate, where it has met with more criticism—notably from Senator Elizabeth Warren (D–MA), who opined that it doesn’t provide sufficient funding for the NIH.
- House Representative Tom Price (R–GA), an orthopedic surgeon, has been nominated to head the Department of Health and Human Services. If confirmed by the Senate, Price, who staunchly opposes the Affordable Care Act and has expressed opposition to the National Cancer Moonshot, will oversee the NIH, FDA, and Centers for Disease Control and Prevention (CDC). He has voted against allowing the FDA to regulate tobacco as a drug and against an earlier version of the 21st Century Cures Act; he’s also pushed to repeal the CDC’s Public Health and Prevention Fund.
- The American Association for Cancer Research and the European Organisation for Research and Treatment of Cancer issued a joint call for robust, sustained cancer research funding. Both organizations noted the need to invest in more international partnerships to validate new cancer targets and technologies. They also emphasized that even in a time of tight health budgets, failure to capitalize on the current era of scientific discovery, which has already yielded many promising advances in cancer treatment and prevention, “is not an option.”
- The American Society of Clinical Oncology (ASCO) announced that it will partner with AstraZeneca to maximize the utility and usability of CancerLinQ Discovery, ASCO’s new platform for analyzing real-world cancer care data. CancerLinQ Discovery aims to make customized, deidentified sets of such data—derived from the rapidly growing CancerLinQ database—available to researchers upon request. The hope is that insights gleaned from the data will generate research hypotheses, identify new uses for existing drugs, and yield better ways to manage treatment side effects.
- Pfizer announced positive results from a key study of its investigational monoclonal antibody PF-05280014, a potential biosimilar to trastuzumab (Herceptin; Roche). A total of 690 patients with metastatic HER2-positive breast cancer were randomized to receive PF-05280014 or trastuzumab, plus paclitaxel, as first-line therapy. The difference in objective response rates between the study arms was not statistically significant, the company reported.
- The FDA granted Priority Review to two drug applications: avelumab (EMD Serono) for Merkel cell carcinoma (MCC), and pembrolizumab (Keytruda; Merck) for microsatellite instability–high tumors. If approved, the investigational PD-L1 inhibitor avelumab would be the first therapy indicated for metastatic MCC, a rare and aggressive skin cancer, in the United States. The PD-1 inhibitor pembrolizumab is approved for several cancers, and Merck is seeking to expand its use to DNA repair–defective tumors, which have a high potential for neoantigen production and may be especially responsive to immunotherapy.
November 18, 2016
- CRISPR/Cas9 gene editing was tested in humans for the first time. Researchers at Sichuan University in Chengdu extracted T cells from a clinical trial participant with aggressive lung cancer, used CRISPR/Cas9 to delete the gene for PD-1, then injected the modified, expanded T cells back into the patient. A similar study in the United States is slated to begin in early 2017; its scientific advisor Carl June, MD, PhD, of the University of Pennsylvania in Philadelphia, told Nature that he expects “‘Sputnik 2.0,’ a biomedical duel on progress” between China and the U.S., “which is important since competition usually improves the end product.”
- On his website, President-elect Donald Trump stated that his administration will “reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products.” The details of this proposed reform remain vague, but it’s viewed by some in industry as a boon, according to FierceBiotech, because deregulating the FDA could speed drug approvals. Meanwhile, Republican Senate Majority Leader Mitch McConnell told the biopharma news outlet that the 21st Century Cures Act is a top legislative priority for Congress, and he “want[s] to see us finish that important new measure this year.”
- New guidelines were issued for HER2 testing in gastroesophageal adenocarcinoma (GEA). After conducting a systematic literature review, an expert panel comprising members of the American Society of Clinical Oncology, the College of American Pathologists, and the American Society for Clinical Pathology produced 11 evidence-based recommendations aimed at establishing standards for when and how to accurately assess HER2 status in patients with GEA. They hope these guidelines help clinicians identify patients most likely to benefit from trastuzumab (Herceptin; Roche), a HER2-targeted therapy.
- The FDA granted Priority Review to the multikinase inhibitor midostaurin (PKC412; Novartis) for patients with newly diagnosed FLT3-mutant acute myeloid leukemia (AML). This mutation occurs in about one third of patients, who have a higher relapse rate and faster disease progression; in a phase III trial, participants randomly assigned to receive midostaurin experienced significantly improved overall survival compared with those given placebo. The agency is also reviewing a FLT3 companion diagnostic developed by Novartis in collaboration with Invivoscribe Technologies, and will issue formal decisions on both within 6 months.
- The Leukemia and Lymphoma Society (LLS) committed $40.3 million to advance blood cancer science at leading academic and medical centers worldwide, including Dana-Farber Cancer Institute in Boston, MA; Fondazione Centro San Raffaele in Milan, Italy; and South Australian Health & Medical Research Institute in Adelaide. LLS’s newest investment adds 75 research grants to its diverse portfolio of projects, which include starving AML cells as a novel therapeutic approach; studying an inherited genetic abnormality in RUNX1 that increases the risk of AML; and finding targeted treatments for monoclonal gammopathy of undetermined significance, a frequent precursor to multiple myeloma.
- Pfizer entered into a Cooperative Research and Development Agreement with the NCI to evaluate three immunotherapies in multiple cancers. The pharmaceutical giant will collaborate with the NCI’s Center for Cancer Research in conducting preclinical and clinical studies for PF-04518600 and utomilumab, which target OX40 and 4-1BB, respectively; as well as the PD-L1 inhibitor avelumab.
- Roche launched its cancer immunotherapy Centers of Research Excellence (imCORE) network, a partnership with 21 leading academic centers around the world. The Swiss company will invest nearly $100 million to support collaborations between its scientists and investigators from imCORE’s participating institutions. The overarching goal is to advance the science of cancer immunotherapy through shared technology, data, and expertise.
November 11, 2016
- Scientists in the United States greeted Donald Trump’s election victory with some anxiety. Nature News’s roundup of social media reactions highlighted worries about an “anti-science U.S. president” restricting funding opportunities and increasing scrutiny on research questions. In a Science story, lobbyists wondered how key political appointments—including heads of the NIH and the Department of Energy—would be filled, given the Trump campaign’s seemingly few connections to the scientific community.
- The tobacco industry’s efforts to quash various state cigarette tax hike initiatives met with mixed results. Voters in California approved Proposition 56, which will increase the state cigarette tax by $2.00 per pack. However, Colorado and North Dakota rejected similar measures, which would have resulted in per-pack tax increases of $1.75 and $1.76, respectively.
- Medical marijuana won big on Tuesday night. Voters in Florida, North Dakota, and Arkansas approved initiatives legalizing its use for patients with diseases such as cancer, HIV/AIDS, amyotrophic lateral sclerosis, and post-traumatic stress disorder. In Montana—where medical marijuana has been legal since 2004—voters opted in favor of repealing restrictions, introduced in 2011, that limited dispensaries to just three users and required state review of physicians who prescribed medical marijuana to more than 25 patients per year.
- IBM Watson Health and the Broad Institute in Cambridge, MA, announced a $50 million initiative to discover the basis of cancer drug resistance. Over 5 years, researchers will generate tumor genome sequence data from thousands of initially treatment-responsive patients who develop resistance. Drawing on Watson’s computational and machine learning methods, they hope to identify genomic patterns that shed light on resistance mechanisms and turn that knowledge into new therapies.
- The FDA approved nivolumab (Opdivo; Bristol-Myers Squibb) for patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) previously treated with platinum chemotherapy. The agency’s nod was based on a phase III trial of 361 patients, who were randomly assigned to receive the PD-1 inhibitor or investigator’s choice of chemotherapy. A 2.4-month improvement in median overall survival was seen in the nivolumab arm.
- Bristol-Myers Squibb also reported that a phase III trial of nivolumab in gastric cancer met its primary endpoint of improved overall survival. Patients in this trial, all with advanced or recurrent disease refractory to standard therapy, were randomly assigned to receive immune checkpoint blockade or placebo. Study results will be presented at an upcoming medical meeting.
- According to Reuters, AstraZeneca will no longer seek early approval from the FDA for durvalumab in HNSCC. The competitive landscape has changed, the company said, with the approval of two PD-1 inhibitors—nivolumab and Merck’s pembrolizumab (Keytruda)—for this disease. AstraZeneca will continue to develop durvalumab for non–small cell lung cancer, where the PD-L1 inhibitor has shown considerable potential; pivotal clinical data are expected in the first half of 2017.
November 4, 2016
- The Fred Hutchinson Cancer Research Center in Seattle, WA, named a new immunotherapy clinic after the Bezos family, which has donated more than $30 million for the institute’s research since 2009. The 9,222-square-foot Bezos Family Immunotherapy Clinic will officially open on December 12 and more than double the center’s capacity to conduct immunotherapy-based clinical trials, from five in 2016 to at least 12 in 2017, according to the Seattle Times. The bulk of these trials will focus on evaluating CAR T-cell therapies—not only in hematologic malignancies where they have shown the most promise so far, but also in solid tumors.
- Novartis’s investigational CDK4/6 inhibitor ribociclib (LEE011) was granted Priority Review by the FDA as first-line treatment, combined with letrozole, for postmenopausal women with HR-positive, HER2-negative advanced breast cancer. This combination is simultaneously being reviewed by the European Medicines Agency for the same patient population; in the U.S., the FDA is expected to issue a formal decision within 6 months. At the recent European Society for Medical Oncology Congress in Copenhagen, Denmark, investigators reported phase III results showing that ribociclib plus letrozole reduced the risk of disease progression or death by 44% compared with letrozole alone.
- Celldex Therapeutics acquired Kolltan Pharmaceuticals for $62.5 million. Celldex, headquartered in Hampton, NJ, has been attempting to diversify its pipeline since its experimental brain cancer vaccine rindopepimut failed a phase III trial earlier this year. With New Haven, CT–based Kolltan’s portfolio, which includes monoclonal antibodies against KIT and HER3, as well as a broad discovery effort aimed at modulating the TAM family of receptor tyrosine kinases, Celldex now has seven cancer drug candidates in its pipeline.
- Two institutions at The University of Texas—San Antonio’s Health Science Center and Houston’s MD Anderson Cancer Center—joined forces to create a comprehensive cancer care program in San Antonio. Dubbed the UT Health San Antonio MD Anderson Cancer Center, it will provide adult cancer patients in 38 South Texas counties greater access to innovative clinical trials and translational research, starting in mid-2017.
- Celgene and IBM teamed up to develop IBM Watson for Patient Safety, a cloud-based monitoring platform aimed at improving pharmacovigilance during drug development. Combining Watson’s cognitive computing ability with Celgene’s experience in drug safety and risk management, the new tool will help pharmaceutical companies manage and interpret reports of drug-associated side effects, which IBM noted are increasing in volume and complexity as data sources grow and regulations evolve. The platform’s ability to identify potential drug-safety signals should also increase over time, thanks to Watson’s continuous-learning capabilities.
- According to an analysis by STAT, pharmaceutical companies are funneling more money than ever into Republican coffers this election year—more than $435,000 in the most competitive House races, compared with less than $70,000 for Democrats. Industry officials told the media outlet that they are gearing up for a Clinton administration, if it happens, to revitalize Washington, DC’s push for lower drug prices. As such, by investing in a divided government, drugmakers hope they will be better positioned to foil legislation that damages their interests.
October 28, 2016
- Researchers at Roswell Park Cancer Institute in Buffalo, NY, got the FDA’s go-ahead to evaluate CIMAvax-EGF, a lung cancer vaccine developed in Cuba, in a U.S. clinical trial. Roswell Park also received authorization from the U.S. Department of the Treasury to establish a joint business venture with the Center of Molecular Immunology in Havana, aimed at discovering and developing new biotech products. The phase I/II trial is expected to open in November, and will test whether the combination of CIMAvax-EGF with the checkpoint inhibitor nivolumab (Opdivo; Bristol-Myers Squibb) is better than either alone in treating the recurrence of non–small cell lung cancer (NSCLC).
- The FDA approved pembrolizumab (Keytruda; Merck) as first-line treatment for patients with metastatic NSCLC whose tumors express PD-L1. The decision was based on data from two randomized clinical trials: In one, pembrolizumab improved the median progression-free survival of previously untreated patients by 4.3 months, compared with those given platinum chemotherapy. In the other, the median overall survival of previously treated patients who received two different doses of pembrolizumab improved by 1.9 months and 4.2 months, respectively, compared with those given docetaxel. Pembrolizumab is the first immune checkpoint inhibitor to be greenlighted for initial treatment of this disease.
- The FDA also greenlighted Ventana’s PD-L1 (SP142) assay as a complementary diagnostic for patients with previously treated, metastatic NSCLC who could be candidates for the PD-L1 inhibitor atezolizumab (Tecentriq; Genentech). Atezolizumab was approved last week for this disease. Also indicated for identifying patients with metastatic urothelial carcinoma who may benefit from atezolizumab, SP142 uses both immune-cell and tumor-cell staining to evaluate PD-L1 expression.
- The U.S. Multi-Society Task Force on Colorectal Cancer released a consensus statement on using the fecal immunochemical test (FIT) for colorectal cancer screening. FIT is “an excellent alternative” to colonoscopy for preventing this disease, the authors said, adding that “surprisingly little published evidence supports the predominance of colonoscopy,” even though it accounts for 60% of all colorectal cancer screenings nationwide. Considered noninvasive, low-risk, and cost-effective, FIT is being compared to colonoscopy in head-to-head trials.
- The FDA placed clinical holds on two drug studies. Berkeley, CA–based Aduro Biotech paused patient enrollment for ongoing trials evaluating CRS-207, an immunotherapy based on live, attenuated Listeria monocytogenes, after two patients tested positive for the bacteria. Plymouth Meeting, PA-based Inovio Pharmaceuticals’ phase III study assessing VGX-3100’s ability to eliminate precancerous cervical lesions through immune stimulation was halted before it ever started; the FDA requested additional data to verify the shelf life of the device used to deliver this experimental immunotherapy.
- According to a study in JAMA Internal Medicine, 28.6% of all cancer deaths in the United States are due to cigarette smoking. The authors found that the proportion of smoking-related cancer deaths varied considerably across states, being particularly high in the South—for instance, in Arkansas, Louisiana, Tennessee, West Virginia, and Kentucky, smoking accounted for nearly 40% of cancer deaths among men. Low spending on tobacco control, lax public smoking policies, inexpensive cigarettes, and a general lack of education about the health effects of smoking are the chief factors to blame, the authors reported.
October 21, 2016
- Vice President Joe Biden delivered a report on the Cancer Moonshot, summarizing the work of its Task Force and laying out Biden’s strategic plan for transforming cancer research and care. The report also includes areas of “scientific opportunity” as determined by a Blue Ribbon Panel and announced last month. The report also outlines several new collaborative efforts between the public and private sector. For example, the NCI, Amazon Web Services, and Microsoft are building a sustainable model for maintaining cancer genomic data in the cloud. In addition, using data stored within its own cancer registry database and collection of tissue samples, the Department of Defense is establishing a new study “to transform our understanding of the biological basis of cancer.”
- Genentech announced that the FDA approved atezolizumab (Tecentriq) for the treatment of metastatic non–small cell lung cancer (NSCLC) that has progressed during or after treatment with platinum-containing chemotherapy. Atezolizumab is the first PD-L1 inhibitor given the green light by the agency for NSCLC; in May, it received accelerated approval for the treatment of locally advanced or metastatic urothelial carcinoma. In a phase III trial, median overall survival for patients with NSCLC who received atezolizumab was 13.8 months, compared with 9.6 months for those who received docetaxel.
- In addition, the FDA granted accelerated approval to olaratumab (Lartruvo; Eli Lilly), in combination with doxorubicin, to treat certain patients with soft-tissue sarcoma—specifically those who cannot be cured with radiation or surgery. The drug works by blocking PDGF receptors, which, when stimulated, can cause tumor growth. Olaratumab is the first new therapy approved for the disease since doxorubicin’s approval four decades ago.
- Stand Up To Cancer announced that it will fund a new Dream Team to conduct research on colorectal cancer, providing up to $7 million over 3 years. Letters of intent are due by December 1, with full proposals from those invited to apply due on February 20, 2017. Information about the grant is available at http://www.aacr.org/su2c-colorectal.
- IBM and Quest Diagnostics launched IBM Watson for Genomics, a new service that will combine cognitive computing and genomic tumor sequencing to extend precision medicine to community oncologists throughout the country. Memorial Sloan Kettering Cancer Center in New York, NY, will supplement Watson’s corpus of data with its OncoKB, which the organization describes as “a knowledge base to help inform precision treatment options for cancer patients.” The Broad Institute of MIT and Harvard in Cambridge, MA, will provide additional sequencing as part of the collaboration.
- Merck announced that a phase III trial of pembrolizumab (Keytruda) in patients with advanced urothelial cancer will be stopped early because the PD-1 inhibitor proved superior to a choice of chemotherapies (paclitaxel, docetaxel, or vinflunine) in extending overall survival. Results of the trial, dubbed KEYNOTE-045, will be presented at an upcoming medical conference, the company said.
- The European Medicines Agency approved a drug combination to treat metastatic adenocarcinoma of the pancreas—pegylated liposomal irinotecan hydrochloride trihydrate (Onivyde; Shire) plus 5-fluorouracil and leucovorin—in adults whose disease has progressed following gemcitabine-based chemotherapy. The approval was based upon results of the phase III NAPOLI-1 study, in which patients who received the three-drug combination lived nearly 2 months longer than patients who received only 5-fluorouracil and leucovorin.
October 14, 2016
- Based on phase III trials of two PD-1 inhibitors as first-line treatment for non–small cell lung cancer (NSCLC), oncologists may soon opt for pembrolizumab (Keytruda; Merck) over nivolumab (Opdivo; Bristol-Myers Squibb). Median progression-free survival (PFS) with pembrolizumab was 10.3 months versus 6 months for chemotherapy. Meanwhile, in a separate trial, PFS for patients receiving nivolumab was just 4.2 months, compared with 5.9 months for chemotherapy. The findings were presented at the European Society for Medical Oncology’s (ESMO) 2016 Congress in Copenhagen, Denmark.
- According to other data from phase III trials presented at the ESMO Congress, OncoGenex’s custirsen failed to significantly increase survival in patients with castration-resistant prostate cancer or NSCLC whose disease progressed following initial treatment. In the prostate cancer trial, median overall survival (OS) with custirsen was 14.2 months compared with 13.4 months for a placebo; in the NSCLC trial, median OS was 9 months and 7.9 months, respectively. Custirsen blocks the production of clusterin, which is involved in carcinogenesis and tumor growth—and contributes to drug resistance.
- In another ESMO presentation, researchers reported that AstraZeneca’s MEK inhibitor selumetinib also failed to offer statistically significant improvements in survival in NSCLC. In a phase III trial that included 510 patients with KRAS-mutant NSCLC, median PFS for those taking selumetinib was 3.9 months compared with 2.8 months for those taking a placebo; median OS for the two groups was 8.7 months compared with 7.9 months, respectively.
- Also at the ESMO meeting: Final results of the pivotal phase III NAPOLI-1 study validated the use of irinotecan liposome injection (Onivyde; Merrimack) in combination with fluorouracil and leucovorin for patients with metastatic pancreatic cancer that progressed following treatment with gemcitabine-based therapy. OS for patients receiving the three-drug combination was 6.2 months, compared with 4.2 months for patients who received only fluorouracil and leucovorin. Initial data from the trial led to FDA approval of the irinotecan liposome injection combination in 2015.
- Merck announced that the FDA approved a two-dose regimen for Gardasil 9, its vaccine against nine types of human papillomavirus (HPV), increasing the chances that people ages 9 to 26 will receive maximum protection from the vaccine. The agency had previously approved a three-dose schedule, but many patients did not complete the full series of vaccinations. Gardasil 9 helps prevent cervical, vulvar, vaginal, and anal cancers caused by HPV types 6, 11, 16, 18, 31, 33, 45, 52, and 58.
- The American Society of Clinical Oncology issued global guidelines for cervical cancer screening, follow-up, and treatment of high-risk lesions in an effort to prevent cervical cancer. Experts on the panel that developed the guidelines established four tiers of recommendations based on the resources available in particular countries or regions: basic, limited, enhanced, and maximal resources. The goal is to ensure minimum screening standards while taking into account wide variations in health care systems and financial circumstances.
October 7, 2016
- Celgene acquired Swiss biotech EngMab for $600 million, expanding its pipeline of immunotherapies targeting BCMA, an antigen almost exclusively expressed on plasma cells and multiple myeloma cells. EngMab is developing a bispecific antibody that binds to BCMA on multiple myeloma cells and to CD3 on T cells, triggering T cell–mediated cytotoxicity. Celgene thinks this approach will complement that of bluebird bio, headquartered in Cambridge, MA—last year, the two companies restructured their collaboration to focus on developing bb2121, bluebird’s lead CAR T-cell therapy against BCMA.
- The FDA granted Breakthrough Therapy designation to alectinib (Alecensa; Roche) for patients with advanced ALK-positive non–small cell lung cancer (NSCLC) who have not been previously treated with an ALK inhibitor. The decision was based on a phase III trial comparing alectinib with crizotinib (Xalkori; Pfizer) as first-line therapy for this NSCLC subtype: Among 207 patients, the median progression-free survival was 10.2 months in the crizotinib arm, and not reached in the alectinib arm. Alectinib is already greenlighted under the FDA’s accelerated approval program for patients with advanced ALK-positive NSCLC who have progressed on or cannot tolerate crizotinib.
- The US Oncology Network opted for Myriad Genetics as its preferred provider laboratory for hereditary cancer testing. US Oncology is among the nation’s largest networks of integrated, community-based practices dedicated to advancing evidence-based cancer care, with more than 1,000 affiliated physicians in 350 centers across 19 states. Both organizations will collaborate to carry out hereditary cancer research through the Genetic Risk Evaluation and Testing program within network-affiliated practices, and to create a database linking patient outcomes with genetic test results.
- The Pancreatic Cancer Action Network launched Precision Promise, the first large-scale precision medicine trial for this disease, aimed at doubling survival by 2020. The multi-institution trial will enroll patients into sub-studies of therapies that best match their molecular profiles, and will allow patients to move into a different sub-study if an initial treatment doesn’t prove effective. Precision Promise will initially tackle pancreatic cancer from three approaches—DNA damage repair, stromal disruption, and immunotherapy—and add more sub-studies as more biomarkers and therapies are discovered.
- The NIH increased its 5-year Precision Medicine Initiative (PMI) award to The Scripps Research Institute (TSRI) in La Jolla, CA, from $120 million to $207 million. The money will facilitate TSRI’s dual tasks of enrolling 350,000 individuals directly into the PMI and creating a Participant Technologies Center to develop the mobile applications necessary to enroll, collect data from, communicate with, and retain PMI participants.
- A new immunotherapy-focused company, Achilles Therapeutics, launched with $17.5 million in funding backed by Cancer Research UK and the UK’s National Institute for Health Research. Achilles will bring together research experts from University College London and the Francis Crick Institute, who will focus on designing therapies that target tumor neoantigens. The company has exclusive rights to develop and commercialize neoantigen therapies that emerge from Cancer Research UK’s TRACERx study, which is analyzing intratumoral heterogeneity in 850 people with non–small cell lung cancer and tracking tumor evolution from diagnosis through treatment and relapse.
September 30, 2016
- Santa Monica, CA–based Kite Pharma announced positive results from an interim analysis of its phase II study evaluating KTE-C19, a CAR T-cell therapy targeting CD19 on B cells, against aggressive chemorefractory non-Hodgkin lymphoma (NHL). The 3-month complete response (CR) rate among 51 patients with diffuse large B-cell lymphoma, the most common form of NHL, was 33%; among 11 patients with two less common forms, transformed follicular lymphoma and primary mediastinal B-cell lymphoma, the CR rate was 64%. Based on these interim data, the company intends to file for regulatory approval of KTE-C19.
- A phase III head-to-head comparison of carfilzomib (Kyprolis; Amgen) and bortezomib (Velcade; Takeda) failed to demonstrate the former’s superiority in improving progression-free survival (PFS) among 955 patients with newly diagnosed multiple myeloma. Median PFS was nearly identical among those given melphalan and prednisone in combination with either carfilzomib or bortezomib, both of which are proteasome inhibitors. Amgen noted its disappointment with these results, but said it remains committed to evaluating carfilzomib in combination with other agents for this disease.
- The FDA granted Orphan Drug designation to San Diego, CA–based Fate Therapeutics’ ProTmune for preventing graft-versus-host disease and cytomegalovirus infection in patients who have undergone an allogeneic hematopoietic cell transplant. ProTmune involves programming donor peripheral blood with two small-molecule stem-cell modulators, FT1050 and FT4145, to boost the biological and therapeutic properties of the graft’s immune cells.
- House leaders pledged to pass the 21st Century Cures Act before the end of the year. If implemented, the legislation would increase NIH funding by $10 billion over 5 years. The Senate is considering its own version of the bill.
- Bristol-Myers Squibb is joining forces with Nektar Therapeutics of San Francisco, CA, to assess the efficacy of nivolumab (Opdivo) plus NKTR-214 against melanoma and kidney, colorectal, bladder, and non–small cell lung cancers. NKTR-214 is designed to expand the population of tumor-infiltrating lymphocytes and natural killer cells in the tumor microenvironment. The combination of checkpoint inhibition with T-cell growth could have a synergistic effect, leading to a more dramatic response in some patients.
- According to an animal study in Nature Nanotechnology, iron oxide nanoparticles can activate macrophages in the immune system against cancer, inhibiting the growth of early mammary tumors and lung cancer that has metastasized to the liver. Commercially available as the injectable supplement ferumoxytol (Feraheme; AMAG Pharmaceuticals), these nanoparticles have been used as an anemia treatment, as contrast agents for MRI, and as drug carriers. The findings “suggest that ferumoxytol could be applied ‘off label’ to protect the liver from metastatic seeds and potentiate macrophage-modulating cancer immunotherapies,” the researchers wrote.
September 23, 2016
- Facebook cofounder Mark Zuckerberg and his wife, Priscilla Chan, a pediatrician, announced that their foundation will contribute $3 billion over the next decade to fund basic science research that could "cure, prevent, or manage all diseases by the end of the century." Zuckerberg said he initially thought that goal was "really aggressive," but that he now believes their dream is feasible. Neurobiologist Cornelia Bargmann, PhD, of The Rockefeller University in New York, NY, will lead the effort. Late last year, the couple said they planned to donate the value of 99% of their Facebook shares during their lifetimes.
- Led by the American Association for Cancer Research (AACR), more than 300 organizations joined forces to advocate for robust, sustained, and predictable growth in NIH funding at the fourth annual Rally for Medical Research in Washington, DC, on September 21. The following day, scientists, health professionals, and patient advocates fanned out across Capitol Hill to meet with members of the House of Representatives and the U.S. Senate and their staff to raise awareness about the need for increased investment in the NIH and the critical importance of medical research.
- The AACR released its Cancer Progress Report for 2016. This sixth report highlights advances made against cancer—and the many people who are surviving their disease—thanks to biomedical research. It includes the latest information on cancer development, prevention, and diagnosis, as well as progress in the development of molecularly targeted therapeutics and immunotherapies.
- Brain cancer has replaced leukemia as the most common cause of cancer death in children and adolescents, according to data released by the National Center for Health Statistics, which is part of the Centers for Disease Control and Prevention. In 1999, three out of 10 cancer deaths in children and teens were due to leukemia, whereas one in four were due to brain cancer. By 2014, those percentages reversed, with brain cancer accounting for nearly 30% of cancer deaths among people ages 1 to 19.
- Sellas Life Sciences Group announced that the FDA granted Fast Track Designation to galinpepimut-S for the treatment of malignant pleural mesothelioma (MPM). Patients with MPM in a phase II trial who received the immunotherapeutic had a median overall survival of 24.8 months compared with 16.6 months for patients in the control arm. In addition, the galinpepimut-S induced CD8+ and CD4+ T-cell activation and demonstrated a favorable safety and tolerability profile.
- Memphis, TN's St. Jude Children's Research Hospital and the UK’s Wellcome Trust Sanger Institute agreed to exchange cancer mutation data in an effort to better understand pediatric cancers. By creating a more robust repository of genomic information, researchers hope to make more accurate diagnoses, learn how different DNA mutations contribute to the development of disease, and develop targeted therapies for children.
- The trade organization PhRMA issued sharp criticism of Colombia’s decision to mandate price cuts on imatinib (Gleevec; Novartis), saying that it is without merit. "The medicine has been sold in Colombia at a price negotiated and agreed to by the Colombian government under its existing pricing system, and there is no apparent shortage or evidence of other access issues," says Brian Toohey, senior vice president, international, at PhRMA. "The enforcement of a declaration of public interest as a mechanism to impose superfluous price controls sets a harmful global precedent."
September 16, 2016
- More than $111 million has been pledged to Stand Up To Cancer (SU2C) after its fifth star-studded telecast was aired live on September 9 from Los Angeles, CA, by more than 60 broadcast and cable networks and streaming services across the United States and Canada. During the telecast, plans were announced for the 20th SU2C Dream Team, which will focus on colorectal cancer, as well as two new Interception Dream Teams aimed at finding ways to stop lung cancer and pancreatic cancer, respectively, from ever developing.
- Scientists began moving into the Francis Crick Institute in London, UK, a new facility described by Cancer Research UK as “the biggest biomedical research institute under one roof in Europe,” with a total floor space of nearly 1 million square feet. “The Crick,” which is expected to be fully operational by the beginning of 2017, is so large that it has its own navigation app to indicate the quickest way to various labs and offices. It will house more than 1,000 scientists and 250 staffers in labs designed to get people in different fields to collaboratively study conditions such as heart disease, neurodegenerative disorders, and cancer.
- The FDA granted Fast Track designation to niraparib for the treatment of ovarian cancer, a distinction that should expedite its development and review. An investigational PARP inhibitor from Waltham, MA-based Tesaro, niraparib significantly improved progression-free survival in a phase III trial in BRCA-mutant, BRCA-wild-type, and homologous recombination–deficient patients with recurrent disease. The drug is also being evaluated for BRCA-positive breast cancer, and in combination with pembrolizumab (Keytruda; Merck) and bevacizumab (Avastin; Genentech).
- Cambridge, MA–based Aveo Pharmaceuticals halted its phase II study evaluating ficlatuzumab plus erlotinib (Tarceva; Roche) in patients with EGFR-mutant non-small cell lung cancer. Ficlatuzumab is a monoclonal antibody that binds to the HGF ligand and inhibits HGF/c-Met activity. Slow enrollment followed by higher than expected discontinuation rates “significantly compromised the feasibility of the trial,” the company said.
- An FDA advisory committee concluded unanimously that apaziquone, an investigational drug from Irvine, CA-based Spectrum Pharmaceuticals, was no better than a placebo in delaying bladder cancer recurrence over a 2-year period. An analog of mitomycin C, this prodrug is activated by its hypoxic environment to alkylate DNA and promote apoptosis. Although not obliged to do so, the FDA usually follows the advice of its committees in determining whether to approve a drug; a formal decision is expected by December 11.
- The FDA issued warning letters to 55 tobacco retailers for selling newly regulated tobacco products to minors. This action was prompted by compliance checks at major national retail chains, tobacco specialty stores, and online retailers, approximately 1 month after the agency began enforcing new regulations that make it illegal for electronic cigarettes and cigars, among other tobacco products, to be sold to anyone under 18 years of age.
- The FDA modified nivolumab (Opdivo; Bristol-Myers Squibb) dosing from 3 mg/kg to a flat dose of 240 mg intravenously every 2 weeks. Based on pharmacokinetics analyses, the agency determined that the overall drug exposure at this dose is largely unchanged and not likely to affect safety and efficacy. The modification applies to the treatment of renal cell carcinoma, metastatic melanoma, and non–small cell lung cancer; the recommended dose for classic Hodgkin lymphoma is still 3 mg/kg.
September 9, 2016
- Convened by Vice President Joe Biden to help guide the nation’s “Cancer Moonshot,” the Blue Ribbon Panel issued 10 recommendations to speed cancer research—to make a decade’s worth of progress in 5 years,” Biden says. Among the recommendations: increase patient involvement in cancer research; intensify research on major drivers of pediatric cancers; gain greater understanding of tumor evolution, as well as how drug resistance develops; expand the use of proven prevention and early detection strategies; develop new tools and technologies to deliver more effective therapies to patients; and improve data sharing, access, and analysis. The complete report from the panel—a group of more than 150 scientists, clinicians, patient advocates, and industry representatives—is available at http://bit.ly/CancerMoonshotPanel.
- Recognizing that cancer clinical trials “have become more and more challenging to conduct,” a multidisciplinary working group proposed practical solutions to overcoming administrative and regulatory hurdles, such as document management, site visits, adverse-event reporting, and contract negotiation with clinical research organizations. The collaborative effort was led by the American Society of Clinical Oncology and the American Association of Cancer Institutes.
- Merck announced that the FDA has granted Breakthrough Therapy Designation and Priority Review to pembrolizumab (Keytruda), the company’s anti–PD-1 therapy, for first-line treatment of patients with advanced non-small cell lung cancer whose tumors express high levels of PD-L1. The company said that the FDA should decide whether to approve the drug for this indication by December 24.
- Newton, MA-based Karyopharm Therapeutics reported positive top-line results from its phase IIb study evaluating the activity the activity of selinexor (KPT-330) in multiple myeloma. The single-arm clinical trial is assessing selinexor in combination with low-dose dexamethasone in patients who have already tried multiple therapies. Selinexor binds with and inhibits the nuclear export protein XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus, which is believed to kill cancer cells while sparing normal ones.
- Concerned that women and their physicians may be misled by companies' claims, the FDA issued an alert stating that “there are currently no screening tests for ovarian cancer that are sensitive enough to reliably screen for ovarian cancer without a high number of inaccurate results.” The agency noted that use of these tests in the general population might delay treatment for some women while possibly prompting unnecessary tests and surgery for others. For women at high risk of developing the disease, including those with BRCA mutations, the FDA recommended that physicians refer them to a genetic counselor or gynecologic oncologist.
- The UK's National Institute for Health and Care Excellence (NICE) announced that the National Health Service will not cover Roche’s combination of cobimetinib (Cotellic) and vemurafenib (Zelboraf) for the treatment of patients with advanced BRAF V600 mutation-positive melanoma that has spread and cannot be surgically removed. NICE decided that the potential benefits of the drug combination weren’t great enough to justify the cost. Cobimetinib inhibits MEK1/2; vemurafenib inhibits BRAF.
- In a lengthy article marking the 15th anniversary of the World Trade Center attacks, Newsweek reported that 5,441 of the 75,000 people enrolled in the World Trade Center Health Program have been diagnosed with at least one cancer tied directly to the destruction of the twin towers, with the total number of cancers hitting 6,378 as of June. In addition, the New York, NY, Fire Department has tracked the health of 15,700 firefighters and emergency responders and has found that their cancer rates are 19% to 30% higher than before September 11, 2001.
September 2, 2016
- Swiss drugmaker Novartis said it will dissolve its Cell and Gene Therapies Unit, folding it back in to the company’s oncology unit. The change will lead to about 120 job cuts, mostly in the United States. The change will not affect the development of the company's chimeric antigen receptor T-cell therapy, CTL109, for aggressive pediatric blood cancers, company executives said.
- San Diego, CA–based Scripps Health and The University of Texas MD Anderson Cancer Center in Houston have reached a partnership agreement to create Scripps MD Anderson Cancer Center, which will provide cancer care to adults throughout southern California. The new entity will be overseen by physician and administrative leaders from both organizations, and its patients will have access to MD Anderson's treatment protocols, extensive clinical trials, and translational research. Through joint tumor boards, MD Anderson also will provide opinions related to diagnosis and treatment.
- Roche announced that atezolizumab (Tecentriq) showed statistically significant improvement in overall survival compared with docetaxel chemotherapy in people with locally advanced or metastatic non-small cell lung cancer whose disease progressed on or after treatment with platinum-based chemotherapy. The company did not release any data from the phase III trial of the PD-1 inhibitor, but noted that patients experienced improved survival regardless of their PD-L1 status.
- The human mAb ofatumumab (Arzerra) was approved by the FDA for use in combination with fludarabine and cyclophosphamide for the treatment of relapsed chronic lymphocytic leukemia (CLL). In a phase III trial, progression-free survival in patients who received ofatumumab, fludarabine, and cyclophosphamide was 28.9 months compared with 18.8 months in those who received fludarabine and cyclophosphamide. Ofatumumab targets the CD20 molecule on the surface of CLL cells.
- Amgen acquired global development and commercial rights from Boehringer Ingelheim for BI 836909, a bispecific T-cell engager (BiTE) being evaluated in a phase I trial for the treatment of multiple myeloma; financial terms of the deal were not disclosed. BI 836909 targets B-cell maturation antigen, which is uniformly expressed on multiple myeloma cells. BiTE antibody constructs help place T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins that will cause the cancer cell to die.
- According to a study in Nature Communications, two regions of the Tasmanian devil genome are quickly evolving in response to the rapid spread of devil facial tumor disease, a transmissible and usually fatal cancer first detected 20 years ago. By comparing DNA from the marsupials before and after the 1996 disease outbreak, researchers found that five of seven genes in the two regions are related to cancer or immune function in other mammals, suggesting that the animals are developing resistance to the disease—and that they could avoid extinction.
August 26, 2016
- Pharmaceutical giant Pfizer announced that it will acquire Medivation for about $14 billion. Medivation's drug portfolio includes enzalutamide (Xtandi), which blocks multiple steps in the androgen receptor signaling pathway and is approved for the treatment of advanced metastatic prostate cancer. Two other Medivation drugs—talazoparib and pidilizumab—are in development for the treatment of BRCA-mutated breast cancer and diffuse large B-cell lymphoma, respectively.
- When the FDA recommended against the use of power morcellators in 2014 due to their potential to spread undetected uterine cancer in women undergoing minimally invasive hysterectomy, some experts expressed concern that abdominal procedures would cause more surgical complications. However, despite an increase in abdominal hysterectomy, “no increase in the rate of major perioperative complications was found,” according to a study published in the Journal of the American Medical Association.
- The FDA granted priority review to Clovis Oncology's rucaparib for the treatment of advanced ovarian cancer in patients with BRCA-mutated tumors (as detected by an FDA-approved test) who have been treated with two or more platinum-based chemotherapies. The agency is expected to issue an approval decision by February 23, 2017.
- The FDA also granted Orphan Drug Designation to TG Therapeutics' TG-1202, an oral next-generation PI3Kδ inhibitor for the treatment of patients with chronic lymphocytic leukemia. Researchers are evaluating the drug in a phase III trial as a treatment for patients with newly diagnosed or relapsed disease.
- Russian President Vladimir Putin fired the country's science and education minister, Dmitry Livanov. Olga Vasilyeva, a historian of the Russian Orthodox Church and outspoken admirer of the one-time Soviet dictator Joseph Stalin, has been named Livanov’s successor. The move has prompted additional concern among scientists, who learned earlier this month that the government will lay off about 10,000 researchers, most of whom hail from the country's basic research organization, the Russian Academy of Sciences.
- For the overall population of the United States, heart disease has been the leading cause of death for decades, followed by cancer. However, the mortality burden of cancer now has surpassed that of heart disease in 22 states, according to a report from the National Center for Health Statistics, part of the Centers for Disease Control and Prevention, which presents data through 2014. In 2000, cancer was the leading cause of death in just two states: Alaska and Minnesota.
- According to a study published in Annals of Oncology, women with breast cancer who expected to suffer more and worse side effects from adjuvant hormone therapies before treatment began experienced nearly twice as many side effects as women who thought that the treatment wouldn't cause problems. If women experience too many side effects or poor quality of life, they may stop taking the drugs, potentially affecting the success of the treatment and their survival, the researchers noted. If expectations can predict the risk of experiencing side effects, interventions such as counseling could lower the risk and improve adherence to treatment.
August 19, 2016
- The Association for Molecular Pathology issued a report that addresses the challenges in defining the clinical utility of molecular diagnostics for cancer and inherited diseases. According to the report, “many stakeholders have adopted narrow definitions for the clinical utility of molecular testing as applied to targeted pharmacotherapy in oncology, regardless of the population tested or the purpose of the testing.” In contrast, the report recommends definitions of clinical utility that recognize the full contribution and value of molecular diagnostic testing to improve patient care, an approach that emphasizes that the utility of a test result depends on the context in which it is used to classify a patient's disease and/or guide treatment.
- According to a recent survey designed to measure public attitudes about the Precision Medicine Initiative Cohort Program, a majority of respondents expressed a willingness to participate in the national research effort that aims to enroll 1 million people. After reading a description of the program, 79% of the 2,601 respondents expressed support for the program, and 54% said they would definitely or probably participate if asked. The program, which will launch later this year, aims to improve the prevention and treatment of disease based on questionnaires and data from individuals' medical records about their lifestyle, environment, and genetics.
- Cerulean Pharma announced that it will cut its workforce by nearly 50% after its nanoparticle–drug conjugate CRLX101 failed to improve outcomes in 115 patients with advanced renal cell carcinoma in a phase II trial. The Waltham, MA, company tested the agent in combination with bevacizumab (Avastin) and noted no statistically significant difference in median progression-free survival (PFS) or objective response rate (ORR) compared with standard therapies. Median PFS was 3.7 months for patients who received CRLX101 and 3.9 months for those who received a standard therapy; ORRs were 5% and 14%, respectively.
- iCell Gene Therapeutics of Stony Brook, NY, announced that the FDA granted Orphan Drug Designation to CD4CAR, a chimeric antigen receptor (CAR) T-cell therapy directed against CD4, for the treatment of peripheral T-cell lymphoma (PTCL). Although there are ongoing clinical development programs with CAR T-cells for CD19+ cell hematologic malignancies, CD4+ PTCLs have not been targeted by a CAR therapy in a human trial. The Orphan Drug Designation provides incentives to develop drugs for the treatment, diagnosis, or prevention of rare diseases and disorders that affect fewer than 200,000 people in the United States.
- Although cardiovascular disease (CVD) remains the leading cause of death worldwide, cancer is now the top killer of men in 12 European countries: Belgium, Denmark, France, Israel, Italy, Luxembourg, the Netherlands, Norway, Portugal, Slovenia, Spain, and the UK. Cancer is also the leading cause of death among women in Denmark and Israel. Writing in the European Heart Journal, study researchers attribute the shift to significant improvements in the prevention and treatment of CVD.
- The UK publication The Guardian reported that drug companies are cutting prices on cancer therapies that the UK's National Institute for Health and Care Excellence (NICE) has not found cost effective so that the National Health Service will continue to cover them. The UK's Cancer Drugs Fund had paid for drugs not approved by NICE until it closed in March. Thus far, NICE's cost-effectiveness approach has led to price cuts for crizotinib (Xalkori; Pfizer), bosutinib (Bosulif; Pfizer), pemetrexed (Alimta; Eli Lilly), and cabazitaxel (Jevtana; Sanofi).
August 12, 2016
- The FDA granted accelerated approval to pembrolizumab (Keytruda; Merck) for the treatment of patients with head and neck squamous cell carcinoma whose disease has progressed on or after platinum-containing chemotherapy. The approval was based on a study of 174 patients, 28 (16%) of whom responded to the PD-1 inhibitor. The median duration of response had not been reached at the time of data analysis, but 82% of the responders were disease-free for at least 6 months.
- Bristol-Myers Squibb’s PD-1 inhibitor nivolumab (Opdivo) failed to improve progression-free survival as a first-line monotherapy in patients with advanced non–small cell lung cancer. In June, the company had reported positive data for nivolumab when used as a first-line treatment in combination with its anti-CTLA4 drug ipilimumab (Yervoy). One possible reason for the disappointing results: The trial enrolled a broad population of patients instead of those whose tumors expressed high levels of PD-L1.
- Beijing, China’s Hangzhou CognitiveCare and IBM announced that 21 hospitals across China plan to adopt IBM’s Watson for Oncology, a cognitive computing platform for oncologists to help them deliver personalized, evidence-based treatments. Watson for Oncology analyzes massive volumes of medical literature, which it scores and ranks, quickly summarizes patient records, and presents drug labels and treatment guidelines. Cancer is the leading cause of death in China.
- The American Society of Clinical Oncology and the Society of Gynecologic Oncology issued a guideline for the treatment of certain women with newly diagnosed ovarian cancer. Specifically, the guideline states that neoadjuvant chemotherapy and interval cytoreduction may be the optimal treatment for some of those with stage IIIC or IV epithelial ovarian cancer. The current standard of care is primary cytoreductive surgery followed by chemotherapy.
- According to an announcement in the Federal Register, the U.S. government will continue to refuse to allow the use of medical marijuana, saying that its therapeutic value has not been scientifically proven, The Washington Post reported. Even so, 25 states and the District of Columbia have passed laws permitting some use of marijuana for medical reasons. The Drug Enforcement Administration did say that it will increase the number of facilities federally licensed to grow marijuana for research; currently, only one such facility exists.
- When it comes to health care spending, the United States ranks first in the world, doling out $2.9 trillion a year. However, a study published in the Journal of the National Comprehensive Cancer Network concludes that higher health care spending doesn't necessarily lead to improved outcomes in patients with cancer or to lower mortality rates. According to the study, the only cancer type in which high spending led to lower incidence and mortality was breast cancer. The study's authors say that they don't advocate caps on health care spending, but they do advocate spending more thoughtfully.
August 5, 2016
- CVS Health Corp. will cut its coverage of 35 drugs in 2017, including Novartis's imatinib (Gleevec) and its newer version, nilotinib (Tasigna); and Medivation's enzalutamide (Xtandi). In addition, it will stop covering Amgen's filgrastim (Neupogen), used to treat neutropenia in cancer patients, and replace it with Novartis's biosimilar, filgrastim-sndz (Zarxio). The company said it's “taking a stand against egregious drug price increases that unnecessarily add costs for clients.”
- Two investigational agents received the FDA’s Breakthrough Therapy designation: Novartis's CDK4/6 inhibitor ribociclib plus letrozole for HR-positive, HER2-negative metastatic breast cancer, and MEI Pharma's HDAC inhibitor pracinostat plus azacitidine for acute myeloid leukemia (AML). In a phase III study, the ribociclib–letrozole combination improved progression-free survival in previously untreated postmenopausal women with metastatic disease, compared with letrozole alone. Meanwhile, pracinostat plus azacitidine improved the median overall survival and complete response rate of elderly, newly diagnosed patients with AML in a phase II study, compared with phase III results evaluating azacitidine as monotherapy in a similar patient population.
- Patients with cutaneous T-cell lymphoma (CTCL) responded significantly better to brentuximab vedotin (Adcetris; Seattle Genetics/Takeda) than standard chemotherapy. In a phase III study, the objective response rate was 56.3% among patients with CTCL who received the CD30-targeting antibody–drug conjugate, versus 12.5% in the control arm. Seattle Genetics plans to submit an application for FDA approval for the treatment of CTCL in early 2017; the drug is already approved for certain classic Hodgkin lymphomas.
- Due to disappointing results, Cambridge, MA–based Momenta Pharmaceuticals stopped accruing patients for a phase II study evaluating necuparanib plus paclitaxel and gemcitabine in patients with advanced pancreatic cancer. Momenta's independent data safety monitoring board concluded that the three-drug combination lacked sufficient efficacy to continue patient enrollment. Necuparanib is a novel agent engineered from the blood thinner heparin to have a broad range of antitumor effects.
- According to a phase II study, it's safe to “wait and watch” for some patients with slow-growing metastatic renal cell carcinoma. Investigators reported in The Lancet Oncology that 29 patients with two or fewer sites of metastatic disease and one or no poor prognosis factors remained on active surveillance for a median time of 22.2 months before starting systemic therapy, compared to 8.4 months for 19 patients who had a greater number of metastases and risk factors. There were “no observed adverse effects on quality of life, anxiety, and depression” in the 29 patients, the investigators said, so active surveillance may be “the optimum approach to avoid the certain toxicity of systemic therapy without clearly compromising the benefit of therapy when initiated.”
- The FDA accepted Foundation Medicine’s next-generation sequencing assay, FoundationOne, into its Parallel Review program with the Centers for Medicare and Medicaid Services (CMS). Launched in 2011, the program enables developers to have their tests or devices concurrently evaluated for premarket clearance or approval (FDA) and national coverage determination (CMS), to expedite the path to commercialization. Foundation Medicine anticipates that both agencies could complete their parallel review by the second half of 2017; if successful, FoundationOne could be the first FDA-approved comprehensive genomic profiling assay available to support precision oncology, the company said.
July 29, 2016
- In a recommendation statement published in the Journal of the American Medical Association, the U.S. Preventive Services Task Force announced that it doesn’t have enough evidence to recommend visual screening for melanoma in patients with no known special risk factors for the disease. The authors of a related editorial agree that the evidence doesn't meet the task force's standards, but they argue that “the evidentiary standard needs to be further refined to be appropriate to the modest magnitude of potential harms of a properly performed skin cancer screening.”
- Asuragen's molecular diagnostic QuantideX qPCR BCR–ABL IS kit received clearance from the FDA for monitoring response to therapies such as imatinib (Gleevec; Novartis) for the treatment of patients with chronic myeloid leukemia (CML). The Austin, TX, company said that its product is the first FDA-cleared diagnostic kit for use in CML management. The assay, which detects BCR–ABL1 RNA in blood, is not intended for disease diagnosis.
- In related research, Australian researchers reported in Leukemia that an increase in ABCB1 mRNA in patients with CML may identify those who are likely resistant to first-line treatment with imatinib and subsequent treatment with other tyrosine kinase inhibitors, such as nilotinib (Tasigna; Novartis). Monitoring ABCB1 mRNA levels could signal an impending relapse, the researchers wrote, and identify candidates for an alternative therapy.
- Based on interim results of a phase III trial, Celgene announced that it will not seek FDA approval of lenalidomide (Revlimid) as maintenance therapy for patients with diffuse large B-cell lymphoma. Although patients who received the immunomodulatory agent had statistically significant improvement in progression-free survival compared with a placebo, the drug did not extend overall survival.
- The FDA granted Breakthrough Therapy Designation to the CD38-directed immunotherapeutic daratumumab (Darzalex; Janssen) in combination with lenalidomide and the steroid dexamethasone—or with the proteasome inhibitor bortezomib (Velcade; Millennium) and dexamethasone—for the treatment of patients with multiple myeloma who have received at least one prior therapy. The designation helps expedite the development and review of medicines to treat serious or life-threatening conditions.
- In a paper published in the South African Journal of Science, researchers report finding the earliest known case of cancer, an osteosarcoma, in a toe bone of a human ancestor estimated to have lived 1.7 million years ago; the diagnosis was possible thanks to advances in 3-dimensional imaging. Recovered from a cave in an area of South Africa called the Cradle of Humankind, the specimen, according to the paper’s abstract, “indicates that whilst the upsurge in malignancy incidence is correlated with modern lifestyles, there is no reason to suspect that primary bone tumors would have been any less frequent in ancient specimens.”
July 22, 2016
- New U.K. Prime Minister Theresa May moved George Freeman from his post as minister for life sciences to chair of her policy board in the wake of the Brexit vote. That change leaves the government's life sciences sector without a leader. Fierce Biotech reported that little will change on the life sciences front, at least for now, and that “the Accelerated Access Review, which is assessing ways to cut the time it takes for drugs to pass through development and into the healthcare system, will continue.”
- Princeton, NJ–based Advaxis announced that the FDA granted Fast Track status to the immunotherapy drug axalimogene filolisbac for the treatment of high-risk, locally advanced cervical cancer. The drug attacks human papillomavirus–associated cancers by altering a live strain of Listeria monocytogenes bacteria to generate cancer-fighting T cells. In addition to cervical cancer, the drug is in development for the treatment of head and neck cancer and anal cancer.
- A phase III trial assessing osimertinib (Tagrisso) as a second-line treatment of non–small cell lung cancer prolonged progression-free survival compared with standard chemotherapy in patients with EGFR T790 mutation-positive disease that progressed following EGFR tyrosine kinase inhibitor therapy, AstraZeneca announced. In addition, patients achieved a higher objective response rate and duration of response compared with chemotherapy. Full data will be reported at a future medical conference.
- Amgen and Allergan said that their biosimilar (ABP 980) of Roche’s trastuzumab (Herceptin) was as effective as trastuzumab in a late-state study, with no clinically meaningful differences. Trastuzumab is a biologic drug, which is more complex than standard drugs and cannot be precisely copied. As with generic drugs, biosimilars can be sold following the expiration of patents on the original product.
- According to a study published in the Journal of the American Medical Association, in vitro fertilization (IVF) is not associated with an increased risk of breast cancer compared with other fertility treatments after a median follow-up of 21 years. Because IVF procedures temporarily cause decreased estradiol and progesterone levels, as well as highly elevated hormone levels, it had been thought that IVF might influence breast cancer risk. However, in this study of 25,108 women, there was no significant difference in breast cancer incidence, which was 3% for the IVF group and 2.9% for the non-IVF group.
- Authors of a Focus article in Science Translational Medicine suggested that behavioral economics might be a powerful tool to nudge patients to enroll in clinical trials. For example, telling patients how many others have enrolled in the trial could leverage a “safety-in-numbers” mentality. Another idea: automatically enroll patients in low-risk trials, such as a smoking-cessation study, which would then require them to opt out.
July 15, 2016
- CytRx announced disappointing results from a phase III trial of aldoxorubicin, a relative of the chemotherapy drug doxorubicin, in patients with soft-tissue sarcoma. Aldoxorubicin delayed progression-free survival by a median of 4.17 months compared with 4.04 months for patients treated with a therapy of the investigator’s choice, which could have been doxorubicin. However, CytRx noted that because the FDA interrupted enrollment with a partial clinical hold in 2014, the analysis doesn’t provide sufficient follow-up for patients enrolled after the hold was lifted; further analysis and updated results will be presented at an upcoming medical meeting.
- Last week, the FDA ordered Juno Therapeutics to halt a clinical trial of its CAR-T treatment in adults with B-cell acute lymphoblastic leukemia after two patients developed fatal brain swelling; a third patient died of the same complication in May. This week, the Seattle, WA-company announced that the FDA removed the clinical hold on the phase II trial of JCAR015 after removing the chemotherapeutic fludarabine from the protocol. Used to prepare patients for the CAR-T treatment, fludarabine was blamed for causing a toxic reaction with the reengineered blood cells that caused the swelling.
- Boston, MA–based Ziopharm Oncology disclosed that three patients with recurrent brain tumors died in a phase I study of its gene therapy candidate Ad-RTS-hIL-12, which is injected directly into tumors, plus oral veledimex, which activates Ad-RTS-hIL-12. The company said that two of the deaths were not related to the study drug; the third patient died of a brain hemorrhage 15 days after starting the gene therapy. Ziopharm is determining how to proceed.
- Merck’s anti–PD-1 drug pembrolizumab (Keytruda) may rein in metastatic prostate cancer that no longer responds to androgen deprivation therapy and the androgen receptor antagonist enzalutamide (Xtandi; Medivation/Astellas), researchers reported in Oncotarget. Three of the trial’s first 10 participants experienced rapid declines in PSA, and imaging showed that tumors shrank in two of the three. Pembrolizumab is approved for use in certain patients with melanoma or lung cancer.
- According to MarketWatch, AstraZeneca resolved a patent dispute involving its brand-name hormone therapy Faslodex (fulvestrant) with Sandoz, which is seeking FDA approval for a generic fulvestrant product. U.S. patents are set to expire in early 2021. The agreement prevents Sandoz from launching its own version of the drug until March 2019.
- A new test called EndoPredict outperformed Oncotype DX in predicting the chances that a woman’s ER-positive, HER2-negative breast cancer will spread within 10 years of diagnosis, according to research published in the Journal of the National Cancer Institute. The study found that 5.8% of patients classified as low risk by EndoPredict went on to develop metastatic disease versus 10.1% of those considered low risk by Oncotype DX. Patients with an extremely good prognosis can often skip chemotherapy, so a more exact test could spare many women from that treatment.
- Gene variants associated with red hair, pale skin, and freckles are linked to a higher number of genetic mutations in skin cancers, according to a study published in Nature Communications. The burden of mutations associated with these variants is comparable to an extra 21 years of sun exposure in people without this variant. Even a single copy of a red hair–associated MC1R gene variant increases the number of mutations in melanoma.
July 8, 2016
- The NIH awarded $55 million to the Precision Medicine Initiative (PMI) Cohort Program to help build the necessary partnerships and infrastructure for this landmark longitudinal research effort. The 5-year award will be used for a data and research support center, a participant technologies center, and a network of healthcare provider organizations. The PMI Cohort Program will begin enrolling participants later this year and aims to accrue 1 million Americans by 2020, whose health data, including environmental exposures and lifestyle habits, will be tracked for at least 10 years.
- The NCI launched the Breast Cancer Genetic Study in African-Ancestry Populations initiative to explore genetic and biological factors that contribute to breast cancer risk among black women. Through this $12 million initiative, investigators from multiple institutions will share biospecimens, data, and resources, building a study population of 20,000 black women with breast cancer whose genomes will be compared with those of 20,000 healthy black women, as well as with white women who have the disease. By doing so, the researchers hope to better understand persistent racial disparities in breast cancer risk.
- The FDA published two draft guidances aimed at streamlining regulatory oversight of next-generation sequencing (NGS) tests. One document provides recommendations for developing NGS-based tests for hereditary diseases and demonstrating analytic validity; the other suggests ways for developers to show that their tests are clinically valid, which would speed marketing clearance or approval.
- A phase II study of Juno Therapeutics’ lead CAR T-cell therapy, JCAR015, was placed on clinical hold by the FDA after two patients died last week from cerebral edema. Another patient death occurred earlier in the trial, also from the same adverse event. All study participants were being treated for relapsed or refractory acute lymphocytic leukemia.
- Cambridge, MA–based Merrimack Pharmaceuticals’ investigational HER3-targeting antibody seribantumab received Fast Track designation from the FDA for heregulin-positive advanced or metastatic non-small cell lung cancer (NSCLC). The drug, combined with docetaxel or pemetrexed, is undergoing phase II evaluation. Heregulin-positive disease occurs in more than half of patients with NSCLC, and has been associated with progression and poor prognosis in many other cancers.
- Five Prime Therapeutics of South San Francisco, CA, announced that the FDA granted Orphan Drug Designation to FPA144 for the treatment of gastric cancer. Under study in a phase I trial, FPA144 is an investigational antibody that targets FGFR2b, which can be overexpressed in gastric tumors and is associated with a poor prognosis.
- Bristol-Myers Squibb acquired Cormorant Pharmaceuticals of Stockholm, Sweden. The purchase gives Bristol-Myers Squibb full rights to Cormorant’s pipeline of investigational antibodies that target IL8, which is expressed by multiple solid tumor types and encourages metastasis by suppressing the immune system. The lead candidate, HuMax-IL8, is currently in phase I/II development.
- Merck and Pfizer initiated a phase III study to evaluate the investigational PD-L1 inhibitor avelumab in combination with platinum chemotherapy, or as maintenance treatment following chemotherapy, for previously untreated advanced or metastatic ovarian cancer. Avelumab is already being evaluated in a phase III trial for platinum-resistant/refractory ovarian cancer. Besides checkpoint blockade, this immunotherapy is also thought to mediate antibody-dependent cellular cytotoxicity.
July 1, 2016
- Wednesday was a “national day of action” for Vice President Biden’s Cancer Moonshot initiative, during which he hosted a summit at Howard University in Washington, DC—one of more than 270 events in all 50 states, Puerto Rico, and Guam—and announced new endeavors to support the moonshot. One is a public-private partnership between the NCI and 20 to 30 pharmaceutical companies that will allow researchers to obtain and test investigational and approved drugs on an established list, instead of negotiating with each company separately. Another is the creation of an Oncology Center of Excellence at the FDA to better coordinate the regulatory review of cancer products.
- The U.S. Department of Veterans Affairs (VA) and IBM announced their partnership to help expand access to precision medicine for 10,000 American veterans with cancer. IBM’s Watson for Genomics technology will rapidly scan deidentified DNA sequences from these patients and produce a list of potential therapies, ranked on the quality of evidence, that VA physicians can use. Watson continuously learns from previous interactions and should facilitate personalized cancer care for veterans.
- Foundation Medicine announced plans to release approximately 18,000 genomic profiles of adult cancers from its FoundationCORE database to the NCI’s Genomic Data Commons (GDC) portal, launched in early June. This is the largest dataset of its kind given to the NCI, more than doubling the size of the GDC and making the portal an even more comprehensive resource for precision medicine research. FoundationCORE currently holds data from more than 80,000 clinical cases and will continue evolving over time as new tumor profiles are added through use of the company’s clinical assays.
- Infinity Pharmaceuticals said it would cut 100 jobs after AbbVie ended a collaboration to develop and commercialize duvelisib, a PI3Kδ and PI3Kγ inhibitor. Last month, the Cambridge, MA–based company laid off 21% of its staff (46 researchers) in the wake of discouraging results from a phase II study of duvelisib in refractory indolent non-Hodgkin lymphoma. These additional cuts will leave Infinity with 65 employees, who will continue to pursue a phase III study of the drug in relapsed/refractory chronic lymphocytic leukemia.
- San Ramon, CA–based Galena Biopharma halted a phase III study of NeuVax after an independent analysis of interim data concluded that the investigational HER2-targeting breast cancer vaccine was no better at preventing tumor recurrence than a placebo.
- The price of stock in Waltham, MA–based Tesaro soared after a phase III study showed that niraparib significantly improved progression-free survival in BRCA-mutant, BRCA-wild-type, and homologous recombination–deficient patients with recurrent ovarian cancer. Niraparib is an investigational PARP inhibitor, and the first in this class of therapies to show benefit in a phase III trial.
- The tyrosine kinase inhibitor ibrutinib (Imbruvica; AbbVie) received its fourth Breakthrough Therapy designation from the FDA, as well as Orphan Drug status, for chronic graft-versus-host disease (GVHD) in patients who didn’t respond to a systemic therapy—usually glucocorticoids—for the condition. No approved treatments are available for GVHD, a common complication of stem-cell and bone marrow transplant.
June 24, 2016
- The NIH’s Recombinant DNA Advisory Committee unanimously approved the use of CRISPR-Cas9 to tweak T cells in patients, improving the ability of T cells to attack tumor cells. This application would represent the first human use of CRISPR-Cas9, and the committee’s green light is the first step in allowing a proposed clinical trial to proceed. The study would enroll 15 patients with multiple myeloma, melanoma, or sarcoma, and researchers would then edit out two genes—for PD-1 and the endogenous T-cell receptor—from these patients’ T cells. Approvals are still pending from the FDA and the three medical centers where this study would be conducted: The University of Pennsylvania in Philadelphia, The University of Texas MD Anderson Cancer Center in Houston, and the University of California, San Francisco.
- The NIH rejected a petition from several consumer groups to override the patent on the prostate cancer drug enzalutamide (Xtandi; Astellas), which costs two to four times more in the United States than in other high-income countries. Because federal funds were used to aid the development of enzalutamide, the NIH could authorize the drug’s generic production to “alleviate health and safety needs which are not being reasonably satisfied,” the groups charged. In its response letter, the agency noted that “no information was identified from public sources to suggest that enzalutamide is currently or will be in short supply.”
- In further agency news, the NIH awarded $2.4 million to Kailos Genetics and Huntsman Cancer Institute (HCI) in Salt Lake City, UT, to develop a clinical-grade test for circulating tumor DNA. Research and development for the blood test will occur over a 5-year period at HCI and Kailos’s CLIA-certified next-generation sequencing laboratory to ensure accuracy and reproducibility. The test will be used to monitor patients with breast cancer for disease recurrence and to provide information about resistance mutations.
- Four NCI-designated cancer centers announced the establishment of a research consortium with Celgene to speed the development of next-generation cancer drugs and diagnostics. The participating institutions are Icahn School of Medicine and Columbia University, both in New York, NY; Johns Hopkins University in Baltimore, MD; and the University of Pennsylvania in Philadelphia. Over the next 10 years, each institution will present multiple high-impact research programs to Celgene, and the company has the option to develop and commercialize new therapeutics arising from the consortium’s efforts.
- According to a Swedish study, after imatinib (Gleevec; Novartis) was introduced in 2001, followed by multiple other tyrosine kinase inhibitors (TKI), the life expectancy of patients with chronic myeloid leukemia (CML) has improved dramatically. Researchers estimate that a 55-year-old man diagnosed in 1980 would have a 20.8-year decrease in average life expectancy, but for his peer diagnosed in 2010, the decrease would be only 2.6 years. The life expectancy of patients with CML is now close to that of the general population, the researchers conclude, but due to “an increased incidence of other cancers and cardiovascular morbidity associated with the use of TKIs,” it may never catch up entirely.
- The FDA granted Breakthrough Therapy designation to ruxolitinib (Jakafi; Incyte) for the treatment of acute graft-versus-host disease (GVHD). Acute GVHD is a significant cause of morbidity and mortality in stem-cell transplant recipients, and no approved treatments are available for this condition. Ruxolitinib, a first-in-class JAK1/JAK2 inhibitor, is currently indicated for use in polycythemia vera, a bone marrow disorder that causes overproduction of red blood cells.
- According to a new study, only about 30% of patients with metastatic renal cell carcinoma receiving targeted therapy also elect to have their kidney surgically removed, even though current guidelines call for both treatments if the patient is a good candidate for surgery. The investigators found that surgery was associated with improved survival—a median of 17.1 months, compared with 7.7 months for patients who didn’t undergo the procedure—but that patients who were older, not privately insured, or not treated at an academic medical center often skipped it.
June 17, 2016
- The U.S. Preventive Services Task Force (USPSTF) released a final recommendation statement on screening for colorectal cancer, concluding that the net benefit of starting screening at age 50 and continuing until age 75 is substantial. The net benefit is small for adults between 76 and 85 years of age, and the decision to screen should be an individual one that takes into account overall health and prior screening history. The USPSTF found no head-to-head studies demonstrating that any of the numerous screening strategies available—stool-based, direct visualization, and serology tests—are more effective than others.
- In other colorectal cancer news, targeted exome sequencing of 468 colorectal cancer samples revealed a prognostic role for the APC gene in this disease. Tumors with wild-type APC had a worse prognosis than those with a single truncating APC mutation, but the outcome was poorest for tumors with two truncating APC mutations alongside KRAS and TP53 alterations. According to the study authors, this finding “suggests that sequencing of APC may have clinical utility in the routine staging and potential therapeutic assignment for [colorectal cancer].”
- A bill to prevent pharmaceutical companies from misusing safety rules to block competition from generic drugmakers was introduced by four U.S. senators: Charles Grassley (R-IA), Patrick Leahy (D-VT), Mike Lee (R-UT), and Amy Klobuchar (D-MN). They charged that brand-name companies have taken advantage of the FDA’s Risk Evaluation and Mitigation Strategies program, which ensures safe dispensation of risky drugs, to deny generic manufacturers the drug samples necessary to develop cheaper versions. The bill would allow generic drugmakers facing this and other delay tactics to petition the court for injunctive relief, a speedier process than filing an antitrust lawsuit.
- The FDA granted Fast Track designation to a novel E-selectin antagonist, GMI-1271 (GlycoMimetics), for acute myeloid leukemia (AML). GMI-1271 blocks binding between AML cells and E-selectin, an adhesion molecule in bone marrow cells, which would otherwise enable the tumor cells to escape chemotherapy’s effects by remaining sheltered in the bone marrow microenvironment. At the recently concluded 21st Congress of the European Hematology Association in Copenhagen, Denmark, GlycoMimetics reported encouraging data from its phase I study of GMI-1271 in 19 adult patients with relapsed or refractory AML: The overall response rate was 47%, including eight complete remissions.
- The World Health Organization’s International Agency for Research on Cancer (IARC) reversed its 1991 classification of coffee as “possibly carcinogenic to humans.” In reevaluating epidemiologic evidence from more than 1,000 studies, the IARC found no conclusive evidence for coffee’s carcinogenicity. However, drinking very hot beverages was classified as “probably carcinogenic,” based on limited epidemiological evidence from countries like China and Turkey, and from South America—where tea or maté is traditionally drunk at about 70ºC—that showed an increased risk of esophageal cancer with higher beverage temperature.
- Energy Secretary Ernest Moniz announced his department’s contribution to the National Cancer Moonshot, in the form of supercomputer technology. The Department of Energy’s 17 national laboratories house “some of the fastest and most powerful supercomputers in the world…[that] can make over 20 million billion calculations per second,” Moniz wrote in a post for Medium. Therefore, these machines “have the potential to greatly accelerate the development of cancer therapies by finding patterns in massive datasets too large for human analysis.”
June 10, 2016
- Under a bill approved by a U.S. Senate subcommittee, the NIH would receive a 6.2% budget increase for the 2017 fiscal year (FY)—which amounts to an additional $2 billion—over FY2016, bringing proposed funding for the agency to $34.1 billion. That sum includes an additional $216 million for the NCI, an increase of about 4.1%, and $300 million for the Precision Medicine Initiative, a $100 million–boost over FY2016. Should the bill pass, NCI’s total funding would reach $5.43 billion.
- At the annual meeting of the American Society of Clinical Oncology in Chicago, IL, Vice President Joe Biden announced a project that will allow cancer researchers to more easily share genomic and clinical data. The new Genomic Data Commons, which will receive $70 million from the NCI, will be part of both the National Cancer Moonshot and the Precision Medicine Initiative, which aims to match patients to the best treatments based on genetic abnormalities in their tumors. The information, which will be freely available, will centralize and standardize data collected as part of other large-scale programs, such as The Cancer Genome Atlas, to make it as useful as possible to researchers.
- According to a randomized controlled trial published in JAMA Internal Medicine, graphic pictures on cigarette packaging that show smoking’s potential harms are more effective at prompting people to stop smoking than text-only warnings. Researchers randomly assigned 2,149 smokers to use packs of cigarettes with either pictorial or text-only warnings. After 4 weeks, 40% of those who saw the graphic warnings had quit for at least a day, and 5.7% had quit for at least the week leading up to the study’s end, compared with 34% and 3.8% respectively in the text-only group. The pictorial warnings proved more effective across all subgroups, including sex, race, ethnicity, and socioeconomic status.
- Health Canada approved the use of nivolumab (Opdivo; Bristol-Myers Squibb) for the treatment of adults with advanced or metastatic non–small cell lung cancer (NSCLC) that has progressed during or following treatment with chemotherapy. The drug is also approved in Canada for the treatment of metastatic melanoma and advanced or metastatic renal cell carcinoma. A PD-1 immune checkpoint inhibitor, nivolumab blocks the interaction between a tumor’s PD-1 ligands and T-cells’ PD-1 receptors, prompting the immune system to attack the tumor.
- A law allowing physician-assisted suicide for terminally ill adults with less than 6 months to live went into effect in California. The law requires that patients make three formal requests to their physician—one written request and two verbal requests spaced at least 15 days apart—to receive lethal drugs. The law also stipulates the patients give informed consent and administer the lethal drugs themselves.
- Genentech and its marketing partner OSI Pharmaceuticals agreed to pay $67 million to settle claims that they misled doctors into prescribing the EGFR inhibitor erlotinib (Tarceva) for all patients with NSCLC instead of just for those who had an EGFR mutation or had never smoked, meaning that some patients may have died sooner than they would have if they had received a different drug. The companies said that their communications with physicians complied with the law and that they decided to settle case to avoid a costly litigation.
- However, Genentech did receive some good news related to the drug: The European Commission approved the use of bevacizumab (Avastin; Roche) in combination with erlotinib for first-line treatment of patients with advanced or metastatic NSCLC with EGFR-activating mutations. The decision was based on a phase II study showing a 46% reduction in the risk of disease progression or death for people treated with the combination compared with erlotinib alone, as well as other studies demonstrating the combination’s safety and effectiveness.
June 3, 2016
- The University of Maryland Marlene and Stewart Greenebaum Cancer Center in Baltimore was designated a Comprehensive Cancer Center by the NCI, making it one of only 46 such centers in the United States. The distinction recognizes the Greenebaum Cancer Center’s “high caliber of scientific leadership and robust programs in basic, clinical, and population science research.” As a result of the new designation, the cancer center’s grant will increase by 50%, to $1.5 million annually, and it will be eligible for additional funding from the NCI and other public and private entities.
- The FDA issued a new form for physicians to request expanded access, often called compassionate use, of an investigational drug or biologic for patients who suffer from serious or life-threatening diseases and for whom no comparable or satisfactory alternative therapy or clinical trial is available. “Access to investigational treatments requires the active cooperation of the FDA, industry, and health-care professional in order to be successful,” said FDA Commissioner Robert Califf, MD. “We know that navigating this process can be challenging and time-consuming, and we are committed to reducing the procedural burdens on physicians and patients whenever possible.”
- The FDA approved the cobas EGFR Mutation Test v2 (Roche Molecular Systems), a companion diagnostic test for the detection of exon 19 deletions or exon 21 (L858R) substitution mutations in EGFR to identify patients with metastatic non–small cell lung cancer eligible for treatment with erlotinib (Tarceva; Genentech). The test is already approved for this indication using formalin-fixed paraffin-embedded tissue specimens. The new use is for detection of these mutations in circulating tumor DNA isolated from plasma, making it the first so-called liquid biopsy approved by the FDA.
- Advanced Accelerator Applications’ Netspot, the first kit for the preparation of gallium Ga 68 dotatate injection, a radioactive diagnostic agent for PET imaging, also received a green light from the FDA. This radioactive probe can help locate tumors in adult and pediatric patients with somatostatin receptor–positive neuroendocrine tumors (NET). NETs have receptors for somatostatin, a hormone that regulates the endocrine system. Ga 68 dotatate, a positron-emitting analogue of somatostatin, works by binding to these receptors.
- The combination of Bristol-Myers Squibb’s nivolumab (Opdivo) and ipilimumab (Yervoy) was deemed safe and more effective at treating small cell lung cancer (SCLC) than nivolumab alone. In a phase I/II international trial involving 216 patients who developed progressive disease following standard chemotherapy, 10% of patients treated with single-agent nivolumab responded to therapy, versus approximately 20% of patients treated with one of two combinations of nivolumab and ipilimumab. On the basis of the findings, which will be presented at the American Society of Clinical Oncology Annual Meeting this weekend, two phase III studies have been launched to better assess the therapeutic impact of the immunotherapies on SCLC.
- According to a survey from the Leukemia and Lymphoma Society, 82% of Americans want the next president of the United States to continue to invest in the Moonshot to End Cancer initiative announced earlier this year and headed by Vice President Joe Biden. The survey also found that the 1,025 respondents were almost evenly split on whether the $1 billion government investment was sufficient, with 44% saying that that is the right amount and 42% saying that more funding is needed. In addition, 60% want the money to be used to find cures, while 29% want to focus on better detection methods and 12% want to invest in developing more effective drugs with fewer side effects.
May 27, 2016
- A study in The Lancet suggested that the 2008–2010 economic crisis may have caused an additional 260,000 cancer deaths in 34 countries, including the United States. The researchers extrapolated this number from an observed rise in cancer deaths, between 1990–2010, for every 1% increase in unemployment and every 1% decrease in public healthcare spending. They found that although cancer mortality rose with unemployment, universal health coverage was a protective factor—this was especially the case, the study’s lead author noted, for treatable cancers such as breast, prostate, and colorectal cancer.
- The FDA’s Accelerated Approval program for new cancer drugs was called into question by a study in Mayo Clinic Proceedings. The analysis found that of 25 drugs approved between 2009 and 2014, 56% (14 drugs) lacked supporting evidence aligning the surrogate measures used—including objective response rate and progression-free survival—with actual survival benefit. The FDA is reviewing this issue; however, “it has been widely accepted that the benefit of a drug can be demonstrated by a variety of endpoints, not just overall survival,” a spokesperson told MedPage Today.
- The NIH awarded $142 million over 5 years to the Rochester, MN–based Mayo Clinic to create a biobank for the Precision Medicine Initiative cohort program. The funds will be used to collect, store, and distribute more than 35 million biospecimens for comprehensive genomic analyses. To protect the resource from natural disasters, a core facility at the Mayo’s Florida branch will house about 25% of the collection.
- The NIH is seeking grant applications to investigate exosomes in cancer, given that these microvesicles are secreted in great quantities by tumors and could shed important light on cancer cell biology. Projects focused on the difficult task of designing robust, reproducible methods to isolate pure exosome populations are of particular interest. Other potentially fundable proposals include assessing exosomes as predictive biomarkers and developing ways to better characterize exosome cargo.
- In further agency news, the NCI launched a new online tool to educate the public about distinguishing common moles from melanoma. Called “Moles to Melanoma: Recognizing the ABCDE Features,” the tool provides a series of photos to show users how individual pigmented lesions may change over time. It also explains how to assess a mole or lesion using ABCDE, which stands for asymmetry, border, color, diameter, and evolving appearance.
- Guardant Health launched Project LUNAR to evaluate its liquid biopsy, Guardant360, in early-stage cancer detection. The umbrella trial will involve researchers from multiple institutions and focus on enrolling participants at high risk of breast, ovarian, lung, pancreatic, and colorectal cancers. Meanwhile, Pathway Genomics initiated a trial evaluating its liquid biopsy, CancerIntercept, in detecting thyroid cancer, the company’s third study for this technology; two trials are ongoing in colorectal cancer.
May 20, 2016
- The FDA granted accelerated approval to atezolizumab (Tecentriq; Genentech), a PD-L1–blocking antibody, for the treatment of patients with locally advanced or metastatic urothelial carcinoma whose disease has progressed during or following platinum-based chemotherapy. The approval was based on a multicenter, single-arm trial in 310 such patients whose tumors were analyzed for PD-L1 expression using the Ventana PD-L1 (SP142) Assay, which was also given the green light by the FDA. The overall response rate was 14.8%; however, 26% of patients considered PD-L1 positive (greater than 5% of tumor cells expressing the marker) responded compared with 9.5% of those with tumors considered PD-L1 negative.
- The FDA also gave a thumbs-up to two already-approved drugs for new indications. The agency OK’d lenvatinib (Lenvima; Eisai), a multiple receptor tyrosine kinase inhibitor, in combination with everolimus (Afinitor; Novartis) for the treatment of patients with advanced renal cell carcinoma. In addition, it granted accelerated approval to nivolumab (Opdivo; Bristol-Myers Squibb) for the treatment of classic Hodgkin lymphoma that has progressed following autologous hematopoietic stem cell transplantation and post-transplantation use of brentuximab vedotin (Adcetris; Seattle Genetics), making it the first PD-1 inhibitor approved for a blood cancer.
- Merck KGaA announced promising interim data from a phase I single-arm trial of the PD-L1 inhibitor avelumab for the treatment of Merkel cell carcinoma. The trial included 61 patients previously treated with standard chemotherapy, six of whom experienced a complete response and 12 of whom had significant tumor shrinkage. Currently, there are no approved second-line therapies for the disease.
- Aduro Biotech announced that a clinical trial testing its immunotherapy combination of GVAX Pancreas and CRS-207 failed to increase overall survival in patients who had received at least two prior treatments for pancreatic cancer. In fact, data from the Phase IIb trial showed that patients who received either CRS-207 alone or chemotherapy lived longer (5.4 months and 4.6 months, respectively) than those who received the combination therapy (3.8 months). The company said that “full study findings will be presented at a future scientific congress.”
- Roche filed a lawsuit against the Drug Controller General of India, arguing that the process to approve biosimilars of bevacizumab (Avastin) is flawed and could compromise patient safety. Roche has also sued the Hyderabad, India–based company Hetero Drugs, which aims to market a copy of bevacizumab. Opponents contend that Roche is simply trying to protect its bottom line by blocking competition.
- In other Roche news, the company failed to gain the endorsement of the UK’s National Institute for Health and Care Excellence (NICE) for its breast cancer drug pertuzumab (Perjeta). NICE, which advises the British National Health Service (NHS) on which drugs demonstrate high value for the price, said that more evidence of an overall survival benefit would be necessary to earn its support.
- Leaders of 15 major UK charities wrote to Prime Minister David Cameron encouraging him to review changes at the NHS that could result in 20,000 patients losing access to potentially lifesaving cancer drugs, according to a report by The Telegraph. In 2010, the Cancer Drugs Fund was established to pay for treatments rejected by NICE. However, after costs exceeded the fund’s budget, soon-to-be-released plans will call for funding only those drugs that earn the support of NICE.
- Legislation that took effect in the UK today calls for all tobacco products to be sold in plain, standardized packaging within the next 12 months, meaning that packaging for all cigarettes and hand-rolled tobacco will look the same. Brand names will appear in a standard type face, color, and font; company logos cannot be shown. Graphic images that underscore health warnings will need to cover 65% of the packaging.
May 13, 2016
- Technology entrepreneur Larry Ellison announced that he will give $200 million to the University of Southern California in Los Angeles to create the eponymous Lawrence J. Ellison Institute of Transformative Medicine, which will focus on cancer research. The gift will help fund the construction of a state-of-the-art building with interdisciplinary laboratories in which cancer researchers will draw upon the knowledge of experts from fields such as mathematics, physics, and engineering. Ellison cofounded Oracle and now serves as the company’s chairman.
- In the wake of an independent task force report issued last year, the National Health Service (NHS) unveiled an ambitious action plan to provide world-class cancer care in England, which will include speeding the diagnosis of cancer, obtaining more modern equipment to treat disease, and assisting patients who are coping with the aftereffects of treatment, the UK’s Daily Mail reported. One step in the plan calls for diagnosing patients suspected of having cancer within 28 days of referring them for testing. Groups such as Macmillan Cancer Support say they welcome improvements in cancer care but question how the changes will be funded.
- In other UK health news, the National Institute for Health Care Excellence rejected the use of nivolumab (Opdivo) for the treatment of patients with locally advanced or metastatic nonsquamous, non–small cell lung cancer whose disease has spread following chemotherapy. The UK-based PharmaTimes noted that the PD-1 inhibitor was rejected even though its maker, Bristol-Myers Squibb, presented a variety of funding schemes to make the immunotherapeutic more affordable.
- Two Bristol-Myers Squibb immunotherapies fared better in Europe: The European Commission granted approval to use nivolumab in combination with the CTLA-4 inhibitor ipilimumab (Yervoy) for the treatment of adults with advanced BRAF–wild-type melanoma. The approval was based on data showing an objective response rate of 61% in patients given the combination compared with 11% in those who received ipilimumab alone.
- Meanwhile, in the United States, the FDA granted Orphan Drug Designation to selumetinib (AstraZeneca), an investigational MEK1/2 inhibitor, for the treatment of certain patients with differentiated thyroid cancer. The FDA also deemed PVS-RIPO a Breakthrough Therapy. A genetically engineered poliovirus, PVS-RIPO is under study at Duke University Medical Center in Durham, NC, for the treatment of recurrent glioblastoma. Injected directly into tumors, the oncolytic virus not only infects and kills cancer cells, but also provokes an immune system response, researchers say.
- In an editorial in Science Translational Medicine, former FDA Commissioner Margaret Hamburg, MD, and Sanofi research head Elias Zerhouni, MD, called for international harmonization of drug regulations. They write that “the mosaic of regulations that govern drug development and oversight nation by nation are creating unnecessary barriers to the efficient delivery of safe, innovative, and effective treatments to patients in need.” For example, regulatory agencies in different countries might require additional clinical trials for drug approval, driving up costs and delaying patient access to lifesaving drugs, they say.
- Wisconsin Senator Ron Johnson introduced a bill that would make it easier for terminally ill patients to access experimental drugs outside of a clinical trial. Although dying patients can petition the FDA for access to potentially lifesaving medicines, critics claim that the agency’s compassionate use program is “arbitrary and cumbersome” and want the FDA cut out of the process, STAT reported.
May 6, 2016
- The FDA finalized a rule extending its authority to regulate everything that meets the definition of a “tobacco product.” The agency already regulates cigarettes, cigarette tobacco, roll-your-own tobacco, and smokeless tobacco; it will now oversee electronic nicotine delivery systems, such as electronic cigarettes and vape pens, and hookah tobacco, among others. This final rule goes into effect on August 8.
- The FDA also granted Priority Review to Eli Lilly’s olaratumab and Amgen’s blinatumomab (Blincyto). Olaratumab, an investigational PDGFRα antagonist, is being evaluated in combination with doxorubicin for the treatment of advanced soft-tissue sarcoma that’s not amenable to radiotherapy or surgery. Blinatumomab was approved in 2014 for adult patients with relapsed or refractory Philadelphia chromosome–negative acute lymphoblastic leukemia (ALL); it’s now under review for use in children and adolescents.
- In further agency news, Kite Pharma’s lead product, KTE-C19, secured Orphan Drug designation from the FDA for five hematologic malignancies: primary mediastinal B-cell lymphoma, mantle cell lymphoma, follicular lymphoma, ALL, and chronic lymphocytic leukemia. A CAR T-cell therapy targeting CD19, KTE-C19 already has this designation for diffuse large B-cell lymphoma (DLBCL). Kite is currently evaluating their product in four pivotal studies for patients with various B-cell malignancies, including pediatric and adult ALL, and DLBCL.
- Foundation Medicine launched its blood-based liquid biopsy assay, FoundationACT, for commercial use. FoundationACT targets a 61-gene subset of FoundationOne—which profiles over 300 genes—and is geared toward patients who can’t have a tissue biopsy or don’t have enough tissue for the broader assay. The company also announced an expanded relationship with AstraZeneca to develop companion diagnostic assays, with the hope of better identifying patients most likely to benefit from drugs in AstraZeneca’s pipeline.
- At least three more pharmaceutical suitors have Medivation in their sights, including Pfizer, AstraZeneca, and Novartis. Last week, French drug maker Sanofi offered to buy the San Francisco–based company for roughly $9.3 billion, which Medivation rejected as undervaluing its pipeline. Besides making the prostate cancer drug enzalutamide (Xtandi), Medivation is developing pidilizumab for blood cancers and talazoparib for breast cancer.
- The Colombian health ministry plans to issue a compulsory license to override Novartis’s patent on imatinib (Gleevec), which would permit a pharmaceutical company to make a generic version. This would widen access to the drug and, the government claims, save Colombia about $12 million a year. Novartis has countered, saying that “there is no shortage of Gleevec or evidence of other access issues,” nor is this “a solution to the financial challenge faced by Colombia’s health care sector.&$148;
- Georgia Governor Nathan Deal signed a bill to expand coverage of drugs for patients with stage IV cancer. Dubbed the Honorable Jimmy Carter Cancer Treatment Access Act, it prevents insurance companies from requiring that patients fail to respond to standard therapies before they’re able to access more advanced options. The new law was inspired by the former president’s successful melanoma treatment with the immune checkpoint inhibitor pembrolizumab (Keytruda; Merck).
April 29, 2016
- North Chicago, IL–based AbbVie acquired Stemcentrx in a deal that could total nearly $10 billion. AbbVie aims to cash in on the five drugs that the South San Francisco, CA-based company currently has in clinical trials, including its antibody–drug conjugate Rova-T, which is under study for the treatment of small-cell lung cancer. Under the terms of the agreement, AbbVie will pay Stemcentrx $2 billion in cash and $3.8 billion in stock, as well another $4 billion if the cancer drugs it has in development meet certain regulatory and clinical goals, The Chicago Tribune reported.
- In what could be another blockbuster business deal, the French drug maker Sanofi offered to buy the San Francisco company Medivation for roughly $9.3 billion. Medivation, which is considering the offer, makes the prostate cancer drug enzalutamide (Xtandi). Xtandi is an androgen receptor inhibitor that acts on multiple steps of the androgen receptor signaling pathway in tumor cells.
- The American Society of Hematology issued a list of six priorities to be included in the National Cancer Moonshot, along with recommended action items for each. The priorities include improving early cancer detection and prevention; improving access to clinical trials of novel targeted therapies; focusing on cancer immunotherapy; enhancing data sharing and education; attacking pediatric cancers; and accelerating the approval of effective therapies.
- In a just-issued report, the UK’s Royal College of Physicians (RCP) encouraged cigarette smokers to switch to electronic cigarettes (e-cigarettes), saying that the nicotine-delivery devices are more likely to benefit—not harm—them. The RCP concluded that e-cigarette use in the UK is almost entirely limited to current and former smokers of traditional cigarettes, who turn to the devices as a method of smoking cessation. The report found little evidence that e-cigarettes prompt nonsmokers to pick up the habit.
- Bristol-Myers Squibb announced that the FDA granted Breakthrough Therapy designation to nivolumab (Opdivo) for the treatment of certain head and neck cancers. A PD-1 inhibitor, nivolumab is already approved for the treatment of inoperable or metastatic melanoma, metastatic non-small cell lung cancer that progressed on or after platinum-based chemotherapy, and advanced renal cell carcinoma following antiangiogenic therapy.
- Also on the FDA front, the Princeton, NJ, company Advaxis announced that it was granted Fast Track designation for the its investigational immunotherapeutic ADXS-HER2 for patients with newly diagnosed, nonmetastatic, surgically resectable osteosarcoma. The company’s technology alters a live strain of Listeria monocytogenes bacteria to generate cancer-fighting T cells directed against a cancer antigen. HER2 can be overexpressed in a variety of tumors, including breast, gastric, and ovarian, as well as in pediatric osteosarcoma.
- Fitbit will soon get a workout, partnering with Dana-Farber Cancer Institute in Boston, MA, in an upcoming cancer study. The trial, which aims to enroll 3,200 overweight and obese women with early-stage breast cancer, will assess the impact of weight loss on disease recurrence. Fitbit is donating fitness trackers, wireless devices that sync to an online dashboard, and personalized exercise software to help participants stay motivated—and to aid their coaches and study investigators, who will be charting activity and weight loss.
April 22, 2016
- At the American Association for Cancer Research Annual Meeting 2016 in New Orleans, LA, Vice President Joe Biden received a standing ovation for his speech about the National Cancer Moonshot and some of the steps that need to be taken to speed cancer research, from expediting grant decisions, to funding riskier research projects, to encouraging greater sharing of data. “I believe together we can redesign a new system — or adjust to a new system that better supports your efforts and save lives sooner than otherwise would have been. Because I really do believe we’re on the cusp of breakthroughs that will save lives, benefit all of humanity,” Biden told attendees. “We have to work together. We have to give you the ability to take chances. We have to help you do what you want to do and —why you got involved: Put patients first.”
- Many in the audience for Biden’s talk turned to Twitter to share their thoughts and some of their favorite lines from the VP’s speech. One that seemed to generate universal approval among cancer researchers: “Measure progress by improving patient outcomes — not just publications.”
- Even before Biden took the stage, the National Cancer Moonshot became a topic of conversation during at least one panel discussion at the AACR meeting, thanks to an informal and decidedly unscientific poll. Based on a show of hands, slightly more than half agreed that “moonshot” is the best word to describe the initiative. Some naysayers noted that the original moonshot, conceived to send humans to the moon, had a detailed plan of action from start to finish, which seems impossible to create when it comes to curing cancer and sets up unrealistic expectations.
- Several meeting speakers and attendees called upon the research community for greater access to research findings and patient data stripped of identifying details. Even so, “data has been no more democratic than it is now,” Nancy Davidson, MD, director of the University of Pittsburgh Cancer Institute and AACR’s new president, told early career scientists. “Take advantage of it to propel your own science.”
- Based on “unprecedented registration” rates in the first few months, NCI-MATCH trial researchers have decided to increase the number of patients they hope to accrue from 3,000 to 5,000, said Peter O’Dwyer, MD, director of the developmental therapeutics program at the Abramson Cancer Center in Philadelphia, PA. NCI-MATCH participants have their tumors analyzed for genetic abnormalities that could be targeted by a drug being studied in the trial and, if so, they are assigned treatment based on the abnormality. Researchers seek to determine whether treating cancers according to their molecular abnormalities will show evidence of effectiveness.
- Last year, NYU Langone Medical Center (New York, NY) and the Technion-Israel Institute of Technology (Haifa, Israel) established a new partnership to advance global collaboration in cancer research and therapeutics. This week, the institutions announced the receipt of $9 million in philanthropic support from NYU Langone Trustees Linda and Isaac Perlmutter. The first joint research projects to be funded with the gift will focus on drug-carrying “nanoghosts” that battle melanoma and new treatments for malignant mesothelioma.
- The clinical-stage biopharmaceutical company argenx has agreed to a deal with AbbVie that could be worth up to $685 million to develop and commercialize ARGX-115, a preclinical human antibody targeting GARP. This protein is believed to contribute to the immunosuppressive effects of T cells. Under the terms of the agreement, argenx will receive an up-front payment of $40 million and stands to earn up to $645 million more for meeting various milestones in the development of ARGX-115.
April 15, 2016
- Through his foundation, Napster founder and former Facebook president Sean Parker announced a $250 million grant to launch the Parker Institute for Cancer Immunotherapy, which includes more than 40 laboratories and 300 researchers from six of the country’s leading cancer centers—Memorial Sloan Kettering Cancer Center (New York, NY); Stanford Medicine (CA); the University of California, Los Angeles; the University of California, San Francisco (UCSF); the University of Pennsylvania in Philadelphia; and The University of Texas MD Anderson Cancer Center in Houston. The collaboration, which will include more than 30 industry partners, aims to “broadly disseminate discoveries, and, most importantly, more rapidly deliver treatments to patients.” The Parker Institute will be led by UCSF immunologist Jeffrey Bluestone, PhD, who was named earlier this month to a Blue Ribbon Panel that will help guide Vice President Joe Biden’s National Cancer Moonshot Initiative.
- The American Society of Clinical Oncology issued a statement urging aggressive efforts to increase vaccination against human papillomavirus, which causes nearly all cases of cervical cancer, can cause oral cancers, and is linked to anal, vulvar, vaginal, and penile cancers. The statement calls for greater education among healthcare professionals, policymakers, and patients about how the vaccine can prevent cancer, as well as improved insurance coverage and access for routine vaccination.
- Stand Up To Cancer announced a new research program called Catalyst that will use funding and materials from the pharmaceutical, biotechnology, diagnostic, and medical devices industries to accelerate research on cancer prevention, detection, and treatment. For example, companies could support collaborative research studies on compounds that they are developing. Merck, Bristol-Myers Squibb, and Genentech are charter supporters.
- According to new data published by the Centers for Disease Control and Prevention, even though cigarette smoking declined, overall tobacco use by middle and high school students did not change between 2011 and 2015, as the use of electronic cigarettes (e-cigarettes) climbed. For the second straight year, e-cigarettes were the most commonly used tobacco product among youth: 16% of high school and 5.3% of middle school students were current users of the devices, meaning they had used them at least once in the previous 30 days. Cigarette use was second, with 9.3% of high school and 2.3% of middle school students being current users.
- Bristol-Myers Squibb announced that the FDA granted priority review to nivolumab (Opdivo) for the treatment of classic Hodgkin lymphoma after prior therapies, such as autologous stem cell transplant and brentuximab vedotin (Adcetris; Seattle Genetics), fail. In 2014, the FDA designated nivolumab a Breakthrough Therapy for this indication, a status intended to expedite the development and review of drugs for serious or life-threatening conditions.
- The FDA granted Orphan Drug Designation to Adaptimmune Therapeutics’ T-cell therapy targeting NY-ESO for the treatment of soft-tissue sarcomas, the company said. It plans to initiate pivotal studies in synovial sarcoma at the end of 2016. The designation helps advance therapies for rare cancers with limited therapeutic options, and it provides tax credits, a longer period of marketing exclusivity, and other benefits.
April 8, 2016
- At a U.S. Senate Appropriations Subcommittee hearing on the NIH budget, senators expressed opposition to President Obama's budget request for the agency for fiscal year 2017. The president's proposed budget suggests cutting the NIH's discretionary budget by $1 billion and then “restoring” that money with mandatory funding, but a stream of money to cover mandatory funding has not been identified. NIH Director Francis Collins, MD, PhD, said that the impact of the president’s proposed budget would be “devastating,” and that he wanted annual budget increases of 5% plus inflation.
- The University of Texas MD Anderson Cancer Center in Houston celebrated the dedication of the Sheikh Zayed Bin Sultan Al Nahyan Building for Personalized Cancer Care. Construction of the building was made possible through a transformative grant of $150 million in 2011 by the United Arab Emirates' Khalifa Foundation. The 12-floor, 628,652-square-foot building, now in the second phase of construction, is designed to integrate delivery of basic and clinical research to support personalized cancer care. It will also house a new center for pancreatic cancer research.
- Ahead of a meeting of an external review panel, FDA staff questioned the efficacy and safety of Clovis Oncology's rociletinib for the treatment of patients with advanced T790M-positive EGFR-mutant non–small cell lung cancer who have not responded to other treatments. The experts will discuss whether the drug’s benefits outweigh the potential harms, such as an increased risk of heart problems. The FDA is not required to follow the committee’s recommendations regarding drug approvals but usually does so.
- Adaptimmune Therapeutics announced that the FDA granted Orphan Drug Designation for its T-cell therapy targeting NY-ESO for the treatment of soft-tissue sarcomas. Company officials said that pivotal studies will be initiated in synovial sarcoma around the end of 2016.
- For nonhematologic cancers, such as breast, colorectal, lung, and prostate cancers, the global market for therapies will nearly double from $72.9 billion in 2014 to $140.8 billion in 2021, at a compound annual growth rate of 9.9%, according to a report from business intelligence provider GBI Research. The report states that this robust growth will occur in spite of the expiration of patents on some commercially successful products such as bevacizumab (Avastin; Genentech), cetuximab (Erbitux; Lilly), and trastuzumab (Herceptin; Roche). The company says that there are nearly 3,000 products under development for nonhematologic malignancies.
- The FDA OK’d the marketing of PneumoLiner, the first tissue-containment system for use with certain laparoscopic power morcellators to isolate uterine tissue that is not suspected to contain cancer. Although the device is an effective tissue-containment system, the FDA is requiring the manufacturer to warn patients and health care providers that PneumoLiner has not been proven to reduce the risk of spreading unsuspected cancer during hysterectomy or myomectomy for the removal of fibroids. The agency estimates that 1 in 350 women undergoing such procedures has an unsuspected uterine sarcoma.
April 1, 2016
- Vice President Joe Biden and former New York, NY, Mayor Michael Bloomberg published a column on Bloomberg View claiming that curing cancer is within reach, especially if researchers from multiple sectors work together. They pointed to the Bloomberg-Kimmel Institute for Cancer Immunotherapy, to be created by Johns Hopkins University in Baltimore, MD, with $125 million in private grants, which could serve as a model. "The original mission to the moon was a government-led, -directed, and -funded initiative," they wrote. "The cancer moonshot will be a true partnership between government, the private sector, academia, and the philanthropic community."
- At the upcoming annual meeting of the American Association for Cancer Research, Biden will deliver remarks to the broader cancer research community, including laboratory, translational, and clinical researchers; population scientists; other health-care professionals; and patient advocates, the organization announced. His speech will take place during the plenary session on April 20 in New Orleans, LA, and will be broadcast live online.
- A report issued by the California Life Sciences Association and Boston Consulting Group found that FDA review times for new drugs have steadily declined since 2009, when a review took 21 months on average. That average was cut to 9 months by 2014. Over the last 14-plus years, the fastest reviews have been for treatments for cancer, as well as infectious and rare diseases.
- The FDA approved defibrotide sodium (Defitelio; Jazz Pharmaceuticals) for hepatic veno-occlusive disease, a rare and life-threatening liver condition that can develop in patients who receive a hematopoietic stem cell transplant (HSCT) to treat certain blood or bone marrow cancers. Although less that 2% of patients undergoing HSCT develop veno-occlusive disease, up to 80% of those who do will not survive. The condition causes some veins in the liver to become blocked, leading to swelling and a decrease in blood flow that may lead to liver damage; severe cases can lead to kidney and lung failure.
- South San Francisco–based Oxigene announced that the FDA granted Fast Track Designation to CA4P for the treatment of platinum-resistant ovarian cancer. In a randomized controlled trial, CA4P improved response rates and progression-free survival (PFS) in patients with recurrent ovarian cancer when it was used in combination with bevacizumab (Avastin) compared with bevacizumab alone. The designation means that Oxigene can have more frequent meetings with the FDA to support the drug’s development.
- Actinium Pharmaceuticals said that Iomab-B received Orphan Drug Designation from the FDA for the treatment of refractory and relapsed acute myeloid leukemia in patients over age 55. The New York, NY, company’s radioimmunotherapeutic conditions patients to receive an HSCT. Orphan status provides marketing exclusivity, potential tax cuts, and other benefits to companies developing drugs for the treatment and prevention of rare diseases and disorders.
- After an Independent Data Monitoring Committee found significant improvements in PFS in patients with relapsed or refractory multiple myeloma, Janssen announced that the phase III CASTOR trial assessing daratumumab (Darzalex) will be stopped early; patients in the control arm will be offered the option of receiving daratumumab following disease progression. The CD38-directed monoclonal antibody was approved in November for patients with multiple myeloma who have received at least three prior therapies.
- Under a state law known as I-Stop that went into effect this week, physicians in New York must electronically order medications instead of writing prescriptions by hand or face possible fines, loss of their medical license, or even jail time. The law is intended to help prevent the abuse of painkillers and improve patient safety by increasing accuracy. Minnesota has a similar law but doesn’t set penalties for failure to comply.
March 25, 2016
- Microsoft cofounder Paul Allen will launch an eponymous foundation with $100 million to advance biologic research and increase knowledge and understanding of biology. During a ceremony at the National Academy of Sciences in Washington, DC, the Paul G. Allen Frontiers Group announced several grants, including a $20 million grant to fund the Allen Discovery Center at Tufts University that will help researchers understand the “biological code that determines anatomical structure and function during embryogenesis, regeneration, and tumor suppression.” Tufts will contribute an additional $10 million to the project.
- Pathologists often disagree about whether a woman has breast cancer if a biopsy contains abnormal cells that fall into a diagnostic "gray zone," which includes atypia and ductal carcinoma in situ (DCIS), according to a study published in the Annals of Internal Medicine. Overall, three independent pathologists who reviewed one slide apiece from 240 women agreed on about 98% of diagnoses of invasive breast cancer and benign conditions without atypia. However, they agreed on fewer diagnoses of atypia and DCIS—92%—suggesting that women diagnosed with these conditions get a second opinion.
- Of 2,596 women who received free screening for a panel of 22 genes linked to hereditary cancers from South San Francisco, CA-based Counsyl, 108 tested positive. However, despite having some family history of cancer, 37 women wouldn’t qualify for screening based on criteria issued by the National Comprehensive Cancer Network. The company says that the results of their 3-month program suggest the need to broaden screening guidelines.
- The Wall Street Journal reported that Johnson & Johnson is settling some 100 wrongful-death and product-liability suits alleging that use of its laparoscopic power morcellator spread undetected cancers, mainly hidden uterine sarcomas, which can be shredded during surgery to remove fibroids. The device was pulled from the market in July 2014.
- Canadian Prime Minister Justin Trudeau requested $180.4 million over 3 years for Genome Canada, a nonprofit seeking to advance genomic science by launching collaborative programs and ensuring that researchers can access the latest genomic technology. Earlier this month, Genome Canada announced that it will fund a research network to speed the translation of genomic research into patient care, GenomeWeb reported.
- Although the UK’s National Institute for Health and Care Excellence (NICE) decided late last year that nab-paclitaxel (Abraxane; Celgene) should not be covered when used in combination with gemcitabine for the treatment of newly diagnosed pancreatic cancer, Wales approved the use of nab-paclitaxel as a first-line therapy for these patients. Scotland initially rejected the drug in 2014, saying that it wasn’t cost-effective, but the Scottish Medicines Consortium has since reversed that decision. Both countries typically back NICE rulings.
March 18, 2016
- Developed by researchers at The University of Texas MD Anderson Cancer Center in Houston, a new staging system for breast cancer considers five factors—preclinical stage, estrogen receptor status, HER2 status, grade, and posttreatment pathologic stage. Breast cancers are traditionally staged by tumor size, metastasis, or lymph node involvement at the time of diagnosis, with no assessment of tumor biology. Researchers say that the new tool will refine prognostic stratification of all breast tumors and help clinicians decide which patients would benefit from additional therapy.
- Gilead Sciences stopped six clinical trials of idelalisib (Zydelig) in combination with other cancer drugs after a higher-than-anticipated number of serious adverse events was reported. Idelalisib is approved to treat chronic lymphocytic leukemia (CLL) in combination with rituximab (Rituxan; Genentech), relapsed follicular B-cell non-Hodgkin lymphoma (NHL), and relapsed small lymphocytic lymphoma (SLL). The FDA will review the trials in question, which involve CLL, indolent NHL, and SLL.
- The FDA granted Priority Review to the immunotherapeutic atezolizumab (Roche) based on data from a phase II trial involving patients with locally advanced or metastatic urothelial carcinoma whose disease progressed during or after treatment with platinum-based chemotherapy. Among all participants, median overall survival was 7.9 months, but for patients with higher levels of PD-L1, it was 11.4 months.
- Spectrum Pharmaceuticals announced that the FDA approved Evomela (melphalan) for two uses: as a high-dose conditioning treatment for patients with multiple myeloma undergoing autologous stem cell transplantation, and as a palliative treatment for patients with the disease who cannot take an oral therapy. The drug was approved based on its bioequivalence to the standard formulation of melphalan, which was initially approved in 1964.
- The American Society of Clinical Oncology published its third annual assessment of national trends in cancer care. The report was also presented at a congressional briefing in Washington, DC, during which Texas Representative Michael Burgess described the state of cancer care by quoting Charles Dickens's A Tale of Two Cities: "It was the best of times, it was the worst of times." Although great progress has been made on the scientific front and mortality rates have declined by 1.5% annually over the past decade, the growing cost of cancer care is not sustainable, the report notes.
- Sixty-four percent of 2,211 doctors recently polled thought that regular colon cancer screenings should begin before age 50, the current recommendation of the United States Preventive Services Task Force. The poll was conducted in response to two recent studies—one found that nearly 15% of patients with colorectal cancer are diagnosed before age 50, and another concluded that younger patients are more likely to have advanced disease.
- Vice President Joe Biden named Greg Simon executive director of his cancer "moonshot" initiative, which he chairs. Simon, who has been battling CLL, is the CEO of Poliwogg, a financial services company that invests in life sciences ventures. Earlier in his career, Simon started FasterCures, a nonprofit organization aiming to quickly translate basic science research into new drugs.
March 11, 2016
- The genetic testing company Ambry Genetics launched AmbryShare, a disease-specific public database that includes just-released anonymized, aggregated data from 10,000 patient samples. The company sequenced the exomes of deidentified patients with hereditary breast and ovarian cancers and reportedly found 10-fold more genes implicated in these conditions than previously known. The goal of AmbryShare is to boost understanding of the underpinnings of disease and find new drug targets.
- A Senate committee approved a series of bills that aim to speed the regulatory process and increase medical research. The "breakthrough" bill would soften requirements for—and speed FDA approval of——medical devices by permitting smaller or shorter clinical trials, a move criticized by experts in medical-device safety. The bills are likely to be wrapped into legislation that would fund the FDA and the NIH.
- The FDA approved crizotinib (Xalkori; Pfizer) to treat patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are ROS1-positive. The drug was first approved in 2011 to treat patients with NSCLC whose tumors are ALK-positive.
- Issued by the NCI and other cancer-related organizations, a new report shows that death rates continue to decline for all cancers combined, as well as for most cancer sites, for men and women of all major racial and ethnic populations. From 2003 to 2012, the overall cancer death rate decreased 1.5% per year. However, death rates due to liver cancer have increased the most compared with all other cancer sites: From 2008 to 2012, they increased by an average of 2.8% per year among men and 3.4% per year among women.
- According to a report released by the Department of Health and Human Services, prescription drug spending jumped by 12.6% in 2014; by comparison, it rose about 2% annually between 2008 and 2012. Higher-priced specialty medications, such as those for cancer, are largely responsible for the increase, the report notes.
- Celldex Therapeutics announced that a phase III trial of rindopepimut (Rintega) for the treatment of patients newly diagnosed with EGFRvIII-positive glioblastoma will be discontinued. Based on a preplanned interim analysis, the independent Data Safety and Monitoring Board determined that both the rindopepimut arm and the control arm are performing on par with each other and any differences in overall survival will not be statistically significant.
- The online medical news outlet STAT reported that a government committee in Colombia has recommended issuing a compulsory license for imatinib (Gleevec), which would allow another company to make the drug without permission from Novartis, which holds a patent on it. Taking this step, the committee said, would widen access to the drug and rein-in health care spending. The annual cost of imatinib is about $15,000, but Colombia’s per-capita gross national income is about $8,000.
March 4, 2016
- In a report from the National Academies of Sciences, Engineering, and Medicine, a committee of experts said that less than half of women diagnosed with ovarian cancer receive care that meets standard guidelines issued by multiple organizations. In addition, they said ovarian cancer should not be categorized as a single disease, but a constellation of different cancers involving the ovary. Recent evidence suggests that many ovarian cancers arise in other tissues or cells not intrinsic to the ovary, but researchers do not fully understand how each subtype develops and progresses.
- A wide variety of health-related organizations have committed to improving electronic health records for patients and health care providers, the U.S. Department of Health and Human Services announced. Health technology providers, the nation’s five largest private health systems, and hospital and medical groups agreed to help consumers easily and securely access their electronic health information and assure that the information will be used to effectively and safely benefit their health; help providers share individuals’ health information to receive care from other providers; and implement federally recognized standards, policies, and best practices, including those related to privacy and security.
- The FDA approved everolimus (Afinitor; Novartis) to treat certain neuroendocrine tumors in adults. The approval was based on significant improvement in progression-free survival (PFS); median PFS was 11 months in patients randomized to receive everolimus, versus 3.9 months in those who received a placebo. An mTOR inhibitor, everolimus was previously approved for the treatment of advanced renal cell carcinoma.
- The FDA also approved the use of fulvestrant (Faslodex; AstraZeneca) with palbociclib (Ibrance; Pfizer) to treat a breast cancer subtype—HR-positive, HER2-negative advanced or metastatic disease that has progressed after endocrine therapy. The approval was based on data from a phase III trial showing that the combination improved PFS by 4.9 months over fulvestrant and a placebo.
- Exelixis and Ipsen announced an exclusive licensing agreement for the commercialization and further development of cabozantinib (Cometriq). As part of the deal, Exelixis will receive an up-front payment of $200 million and additional money for reaching regulatory and commercial milestones; Ipsen will have rights to current and future cabozantinib indications. Already approved for the treatment of medullary thyroid cancer, cabozantinib inhibits the activity of tyrosine kinases, including VEGF receptors, MET, AXL, and RET.
- AstraZeneca announced that tremelimumab did not demonstrate an overall survival benefit in the second- or third-line treatment of inoperable malignant mesothelioma in an international phase IIb randomized, double-blind, placebo-controlled trial that included 571 patients. An investigational, selective human antibody directed against CTLA-4, tremelimumab is also being studied alone or in combination with durvalumab, a mAb directed against PD-L1, in other cancers, including non–small cell lung, bladder, head and neck, gastric, pancreatic, liver, and blood cancers.
- There was some good news for mesothelioma, however: SELLAS Life Sciences announced that the FDA granted Orphan Drug Designation to its WT1 cancer vaccine for patients with the disease. The company previously reported that the vaccine improved overall survival and doubled PFS in a phase II trial in patients with mesothelioma. The vaccine stimulates cytotoxic T cells to target and kill WT1-expressing cells, which are found in a number of cancers.
February 26, 2016
- The U.S. Senate confirmed Robert M. Califf, MD, as the FDA's commissioner of Food and Drugs. He replaces Margaret Hamburg, MD, who stepped down at the end of March 2015. For the last year, Califf served as the FDA's deputy commissioner for Medical Products and Tobacco.
- President Barack Obama announced that dozens of government agencies and private-sector organizations have committed to accelerate the Precision Medicine Initiative, which he announced last year. For example, Vanderbilt University and Verily, formerly Google Life Sciences, will launch the first phase of the Precision Medicine Initiative Cohort, laying the foundation for a study of 1 million people who will share their medical information in an effort to transform understanding of health and disease. In addition, the White House released a fact sheet spelling out the key principles of the initiative and outlining the roles of the partners in the venture.
- A new report outlines substantial progress in reducing the mortality gap between blacks and whites for some cancers but that the gap widened or remained constant for others. Published in CA: A Cancer Journal for Clinicians, the report notes that breast cancer death rates between 2008 and 2012 were 42% higher in black women compared with white women. Between 2003 and 2012, colorectal cancer death rates remained about 50% higher in black men compared with white men.
- According to a study published in Pediatrics, the prevalence of human papillomavirus (HPV) was significantly reduced following the introduction of a vaccine targeting four common types of the virus: HPV-6, -11, -16, and -18. Within 6 years of the introduction of the vaccine, the prevalence of these HPV types decreased by 64% among females ages 14 to 19 and 34% among those ages 20 to 24. HPV causes most cervical cancers.
- The FDA approved palbociclib (Ibrance capsules; Pfizer) in combination with fulvestrant for the treatment of women with hormone receptor-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy. In February 2015, the drug, in combination with letrozole, was approved as an initial endocrine-based therapy in postmenopausal women. The new approval is based on a 4.9-month improvement in median progression-free survival in a randomized, double-blind study comparing palbociclib plus fulvestrant to placebo plus fulvestrant.
- The FDA also approved obinutuzumab (Gazyva; Genentech) in combination with bendamustine, followed by obinutuzumab alone, for the treatment of patients with follicular lymphoma who relapsed after, or are refractory to, a rituximab-containing regimen. The drug was previously approved for use with chlorambucil for the treatment of patients with untreated chronic lymphocytic leukemia. The approval was based on a randomized open-label study in which treatment with bendamustine yielded a median PFS of 13.8 months, whereas the median PFS of the combination was not reached.
- The National Institute for Health and Care Excellence in the United Kingdom endorsed the use of nivolumab (Opdivo; Bristol-Myers Squibb) as a monotherapy for adults with inoperable or metastatic melanoma. The decision was based on data showing that nivolumab significantly extended survival in some patients and that it is likely to meet the agency's requirement to show cost effectiveness based on the length and quality of patients' survival.
February 19, 2016
- Researchers at The University of Texas MD Anderson Cancer Center in Houston proposed a new staging system for patients with oropharyngeal cancer (OPC) that more accurately predicts outcomes—and helps identify the most appropriate treatments—than the current TMN Staging System. Outlined in the Journal of Clinical Oncology, the proposed system accounts for biological and clinical differences between human papillomavirus (HPV)-positive OPC and HPV-negative OPC, which is commonly caused by alcohol and tobacco use. Patients with HPV-positive disease typically have better survival rates.
- The FDA designated AstraZeneca’s immunotherapy durvalumab as a Breakthrough Therapy for the treatment of urothelial bladder cancer. Researchers are studying the drug as a monotherapy and in combination with other agents to treat several types of malignancies, including head and neck, gastric, pancreatic, and non-small cell lung cancers.
- The FDA also granted Breakthrough Therapy status to Novartis’s midostaurin (PKC412), designed to treat patients with newly diagnosed acute myeloid leukemia with a FLT3 mutation. The designation was based on results of a phase III trial in which patients who received the drug and standard induction and consolidation chemotherapy had a median overall survival of 74.7 months compared with 25.6 months in patients who received only standard induction and consolidation chemotherapy.
- Philadelphia's University of Pennsylvania unveiled its new $27 million Novartis–Penn Center for Advanced Cellular Therapeutics, which will focus on chimeric antigen receptor T-cell therapy and other personalized cancer treatments. The center has the capacity to double the number of patients in the area who can be treated in clinical trials.
- The Cancer Fund of America, run by James Reynolds Sr., will go out of business following a court battle with the Federal Trade Commission (FTC) and the attorneys general in all 50 states, CNN reported. Years of reporting by the network found that the charity directors collected millions of dollars that never benefited people with cancer, as well as fake donations and faulty recordkeeping. The FTC also sued three related charities, run by Reynolds’s relatives and a friend, for fraud: Children’s Cancer Fund of America, The Breast Cancer Society, and Cancer Support Services.
- According to The Wall Street Journal, the Laura and John Arnold Foundation will contribute “$7.2 million to four organizations to fund research and pilot projects aimed at reining in drug costs and lowering financial barriers for patients.” The largest chunk of funding, about $4.7 million, will support a project at Memorial Sloan Kettering Cancer Center’s Center for Health Policy and Outcomes, New York, NY, that will test pricing that's tied to a drug’s value. Other organizations that will receive funds are the Center for Evidence-based Policy at Oregon Health and Science University in Portland, Brigham and Women's Hospital in Boston, MA, and the National Academies of Sciences Institute of Medicine in Washington, DC.
- Lung cancer cases in the province surrounding Beijing, China, surged by 306% between 1973 and 2012, accounting for nearly one quarter of all cancer deaths in the country, according to figures released by the Tumor Hospital of Hebei Province and reported by The New York Times. The hospital's report didn't delve into the possible causes behind the increase, but the newspaper noted that air pollution in Hebei is “among the worst, if not the worst, in China.” In addition, smoking rates among men in China are much higher than in other countries.
February 12, 2016
- On February 9, President Obama unveiled his budget proposal for fiscal year 2017, somewhat controversially proposing to cut the NIH's base budget by $1 billion, but to then reinstate the cut (as well as provide an $825 million overall increase) by having Congress authorize a new $1.825 million mandatory funding stream for the NIH. Members of Congress are already expressing opposition to this approach, and even if the new mandatory funding is approved, the NIH Institutes' and Centers' budgets will be held flat in FY 2017 (compared to FY 2016). Most of the overall increase ($680 million) will go to the NCI for its cancer "moonshot" initiative; NIH director Francis Collins, MD, PhD, noted in a briefing that "there are exceptional opportunities in cancer research, and cancer is ripe for a big push."
- A new study led by researchers at Dana-Farber Cancer Institute in Boston, MA, provides encouraging evidence that younger women diagnosed with breast cancer are largely following the National Comprehensive Cancer Network's recommendation that they undergo genetic testing for BRCA 1/2 mutations. In analyzing data from nearly 900 women diagnosed with breast cancer at age 40 or younger, the researchers found a notable increase in BRCA testing, from 77% to 95%, between 2006 and 2013. Among these patients, 86% of those found to carry BRCA mutations—and fully half (51%) of those who weren’t—chose to have a double mastectomy; the researchers think this indicates a need for better communication to non–BRCA carriers of their relatively low risk of developing cancer in the other breast.
- Adaptimmune Therapeutics' affinity enhanced T-cell therapy targeting the tumor antigen NY-ESO-1 has been designated a Breakthrough Therapy by the FDA. This designation was based on the results of a phase I/II trial in 12 patients with unresectable, metastatic, or recurrent synovial sarcoma whose tumors expressed NY-ESO-1: 90% of patients who received the target dose, and 75% who received any dose of engineered T cells, responded well and were on long-term follow-up at the time these results were reported in November 2015. Adaptimmune will initiate pivotal studies in synovial sarcoma later this year, besides exploring its immunotherapy in myxoid round cell liposarcoma; studies are also under way in myeloma, melanoma, ovarian cancer, and non–small cell lung cancer.
- The American Society of Clinical Oncology has issued new evidence-based recommendations on the appropriate use of breast tumor biomarker tests to guide treatment for women with early-stage invasive breast cancer and known hormone receptor status. According to the new guidelines, the estrogen receptor (ER), progesterone receptor (PR), HER2 receptor, Oncotype DX, EndoPredict, PAM50, Breast Cancer Index, urokinase plasminogen activator, and plasminogen activator inhibitor type 1 biomarker tests may be used "to guide decisions on adjuvant systemic therapy for certain patients with breast cancer." However, no biomarker test other than those for the ER, PR, and HER2 receptors should be used "to guide choices of specific drugs or treatment regimens."
- Researchers at the University of North Carolina in Chapel Hill have found a way to deliver a cocktail of chemotherapy drugs directly to pancreatic tumors, sparing the rest of the body from harmful side effects. Their method—an implantable device that uses electric fields to drive the drugs into tumors—resulted in at least three times the amount of the highly toxic FOLFIRINOX cocktail (leucovorin, fluorouracil, irinotecan, and oxaliplatin) reaching pancreatic tumors in mice, compared to intravenous delivery of the same drugs. The researchers are aiming to get their device into clinical trials within the next several years.
- Researchers at the University of Liverpool, UK, have developed a device called Odoreader to better diagnose urological cancers. They describe Odoreader, which uses a gas chromatography sensor system, as "an electronic nose" that can detect cancer in urine samples—the device's 30-meter column enables compounds in the urine to travel through at different rates, breaking the urine sample into a "readable format" that can be translated into a diagnostic algorithm. In a pilot study of 155 men presenting to urology clinics, Odoreader diagnosed 58 with bladder cancer, 24 with prostate cancer, and 73 with hematuria—blood in the urine—but no cancer.
- According to scientists at the University of California, Los Angeles, there's a clinically relevant explanation for why patients with head and neck cancers who test positive for human papillomavirus (HPV) respond more favorably to radiation therapy. The scientists showed that radiation transforms HPV-negative tumor cells into cancer stem cells, which are then able to resist the very therapy being used. In reviewing 162 cases of head and neck squamous carcinomas over a two-year period, the researchers correlated patient outcomes with HPV status, and confirmed that "radiation-induced conversion" happened at a higher frequency in HPV-negative patients.
- A phase II study of two different multiple myeloma drug combinations—bortezomib (Velcade; Takeda) plus melphalan and prednisone, or VMP; and lenalidomide (Revlimid; Celgene) plus dexamethasone, or Rd—shows that for patients with multiple myeloma, VMP and Rd are equally effective whether administered in sequential or alternating cycles. In the study, 233 patients were randomized to receive nine cycles of VMP followed by another nine of Rd (18 total cycles), or VMP and Rd alternating each cycle for the same total treatment duration; the progression-free survival was 74%and 80%, respectively. The investigators are now trying to determine which of these drugs would be best given as continuous therapy; meanwhile, others not affiliated with the study have pointed out that the standard of care for this disease will likely evolve now that immunotherapy—namely daratumumab (Darzalex; Johnson & Johnson) and elotuzumab (Empliciti; Bristol-Myers Squibb)—has made its debut.
February 5, 2016
- In conjunction with the National Cancer Moonshot Task Force’s first meeting on February 1, the Obama administration announced plans to kick-start this initiative with $1 billion, of which $195 million already appropriated to the NIH in fiscal year 2016 will be spent immediately on new cancer activities. The President is expected to request another $755 million in his final budget proposal for fiscal year 2017, most of which will go to the NIH, with $75 million allocated for data work at the FDA. A new national public opinion survey shows that a significant majority of Democrats (67%) and more than a third of Republicans (38%) and independents (39%) favor a tax increase to support this initiative.
- According to data from the phase III MAPS trial in France, bevacizumab (Avastin; Genentech) should be considered a suitable addition to standard cisplatin/pemetrexed (Alimta; Eli Lilly) for patients with newly diagnosed pleural mesothelioma. In this study, 448 patients with unresectable disease and no prior chemotherapy were randomly assigned to receive cisplatin and pemetrexed, with or without bevacizumab. The overall and progression-free survival of those in the bevacizumab arm improved by 2.7 months and 1.9 months, respectively, with manageable toxicities.
- The European Medicines Agency has approved osimertinib (Tagrisso; AstraZeneca) for patients with advanced non–small cell lung cancer (NSCLC) whose tumors bear a specific EGFR alteration, T790M. Osimertinib, which was green-lighted by the FDA in November 2015, is the first targeted therapy for this subset of NSCLC; its European approval was based on two phase II studies showing that the drug shrank tumors in 66% of 474 patients. AstraZeneca hopes to make osimertinib a key part of the company’s growth plans, and is continuing to collect data on its safety and efficacy in a phase III study.
- On Monday, Sun Pharma launched a generic version of imatinib mesylate (Gleevec; Novartis) in the United States. Having filed first with the FDA, Sun’s generic tablets are approved for 180 days of marketing exclusivity. During these six months, the company hopes to poach a third of sales by reducing the price of their version by approximately 30%—in the U.S., Gleevec costs up to $90,000 annually.
- New research from Georgia State University suggests that radiation can make tumor cells more susceptible to immunotherapy. In colorectal cancer cells, the researchers found that low-dose ionizing radiation enhanced the expression of multiple death receptors (Fas, DR4, and DR5) and co-stimulatory molecules (4-1BBL and OX-40L), partly as a result of changes in DNA methylation and histone acetylation. The team hypothesizes that these expression changes could allow the immune system to recognize and go after tumor cells that may have previously escaped elimination, thus maximizing responses to immunotherapy.
- The Centers for Disease Control and Prevention’s updated childhood immunization schedule, released on Monday, officially recommends Gardasil 9 over two other approved vaccines for human papillomavirus (HPV). Gardasil 9 protects against nine HPV strains, compared to four for Gardasil and two for Cervarix. The new schedule also recommends that children with a history of sexual abuse receive the HPV vaccine a few years earlier, between ages 9–10 instead of 11–12.
- A new study shows that the time between a positive fecal blood test—part of the screening process for colorectal cancer—and a follow-up colonoscopy varies significantly nationwide. Investigators from the Group Health Research Institute in Seattle, WA, evaluated 62,384 patients, all with positive tests, and found that the median time to a colonoscopy ranged from 41–174 days; older patients (ages 70–89) were at the highest risk for not receiving the procedure, and follow-up rates were also low for patients who had never before been tested for colorectal cancer. Next, the investigators will determine whether these delays in getting colonoscopies affected patient outcomes—information that’s critical for making the colorectal cancer screening process as effective as possible, they say.
- Researchers at The University of Texas MD Anderson Cancer Center in Houston, Duke Cancer Institute in Durham, NC, and Dana-Farber Cancer Institute in Boston, MA, have been jointly awarded $13.4 million from the Patient-Centered Outcomes Research Institute. They’ll launch COMET, a national prospective, randomized study aimed at investigating a key question in breast cancer management: Do women with ductal carcinoma in situ, the disease’s earliest form, require invasive surgery, or is active surveillance (mammograms every six months for five years) sufficient? The researchers will also compare quality-of-life measures, including anxiety and depression, in both groups of patients.
January 29, 2016
- President Barack Obama announced the creation of the White House Cancer Moonshot Task Force, to be chaired by Vice President Joe Biden, who is leading a new national effort to speed progress in the search for cancer cures. The Task Force, which will include the heads of the executive branch departments, agencies, and offices, will work to produce a detailed set of recommendations to improve our understanding of cancer; support greater access to new research, data, and computational capabilities; increase access to treatment; encourage development of new treatments; identify and address unnecessary regulatory barriers; ensure optimal investment of federal resources; and uncover opportunities to develop public–private partnerships.
- Three U.S. senators have announced that they will place a hold on the confirmation of Robert Califf, MD, as head the FDA. One senator, presidential candidate Bernie Sanders of Vermont, has expressed concerns about Califf’s ties to industry; the two others—Edward Markey of Massachusetts and Lisa Murkowski of Alaska, respectively—want the FDA to change the way it approves opioid painkillers and to require labeling of genetically modified salmon. Sixty senators are needed to override a hold.
- The United Kingdom’s (UK) 100,000 Genomes Project announced the enrollment of the first patients with cancer. The project has already recruited patients with certain rare diseases and aims to map the DNA of patients’ tumors alongside their healthy tissue to improve understanding of how a tumor’s genetic makeup affects the development of a patient’s disease and their response to therapy. Launched in 2012, the project has analyzed more than 6,000 genomes thus far.
- Immunocore said that the FDA has designated IMCgp100 as an orphan drug for the treatment of uveal melanoma, making the British biotech eligible for developmental incentives, speedier review, and a period of exclusive marketing. The drug helps the immune system target and kill cancerous cells.
- AstraZeneca announced that the FDA named olaparib (Lynparza) a Breakthrough Therapy for use as a monotherapy for the treatment of prostate cancer with BRCA1, BRCA2, or ATM mutations in patients who have already received taxane-based chemotherapy and at least one hormone therapy, such as abiraterone (Zytiga; Janssen) or enzalutamide (Xtandi; Medivation/Astellas). A PARP inhibitor, olaparib has already been approved for BRCA-mutated ovarian cancer.
- The FDA expanded indications for the PD-1 inhibitor nivolumab (Opdivo; Bristol-Myers Squibb) to include its use with ipilimumab (Yervoy; Bristol-Myers Squibb) for the treatment of BRAF–wild-type and BRAF-mutant unresectable or metastatic melanoma. The FDA also expanded the indication for single-agent nivolumab to cover patients who haven’t received treatment for BRAF–wild-type melanoma.
- Yet more news this week about nivolumab: Bristol-Myers Squibb said that a phase III study evaluating the drug versus investigator’s choice of therapy in patients with recurrent or metastatic platinum-refractory squamous cell carcinoma of the head and neck was stopped early. An independent data monitoring committee concluded that patients taking nivolumab lived significantly longer than those in the control, or investigator’s choice, arm.
- The UK’s National Institute for Health and Care Excellence approved coverage of four cancer therapies: olaparib, for ovarian cancer; enzalutamide and radium-223 dichloride (Xofigo; Bayer) for certain prostate cancers; and panobinostat (Farydak; Novartis) for multiple myeloma.
- The Ovarian Cancer National Alliance and the Ovarian Cancer Research Fund announced that they will merge, creating the largest global ovarian cancer research, legislative advocacy, and patient support organization. Together, the two organizations have raised nearly $70 million over the past two decades to fight the disease. The new entity will be called the Ovarian Cancer Research Fund Alliance.
January 22, 2016
- While attending a World Economic Forum meeting in Davos, Switzerland, that included cancer experts from around the globe, Vice President Joe Biden vowed that the United States would speed the development of promising new drugs and drug combinations. His pledge came just a week after President Obama announced that Biden would lead the effort to cure cancer “once and for all.”
- The FDA approved ofatumumab (Arzerra; Novartis) for the extended treatment of patients with chronic lymphocytic leukemia (CLL) who are in complete or partial response after at least two lines of therapy for recurrent or progressive disease. Ofatumumab was previously approved for untreated patients with CLL for whom fludarabine-based therapy is not appropriate and for patients with CLL that no longer responds to fludarabine and alemtuzumab (Campath; Genzyme).
- The FDA also approved an additional indication for carfilzomib (Kyprolis; Amgen). The drug can now be marketed in combination with dexamethasone, or with lenalidomide plus dexamethasone, for the treatment of patients with multiple myeloma who have received one to three lines of therapy. In addition, the drug is now fully approved as a monotherapy for patients with multiple myeloma who have already received at least one therapy.
- Also in drug news, the FDA granted Priority Review status to lenvatinib mesylate (Lenvima; Eisai) to treat advanced or metastatic renal cell carcinoma. That decision was based on a phase II trial in which lenvatinib—both alone and in combination with everolimus (Afinitor; Novartis)—prolonged progression-free survival and increased objective response rates compared with everolimus alone.
- The New York Genome Center announced that two charitable organizations—the Simons Foundation and the Carson Family Charitable Trust—have pledged up to $100 million to match gifts from other contributors over the next 3 years. The funds will help support the center’s genomic discoveries and advance medical research.
- Many biotech and pharmaceutical companies announced business deals related to immune-oncology while their executives gathered in San Francisco, CA, for the JPMorgan Healthcare Conference. For example, Cambridge, MA-based Surface Oncology announced that it will collaborate with Novartis to develop immunotherapeutics targeting the tumor microenvironment, while its nearby neighbor, Tensha Therapeutics, whose lead product is a BET inhibitor called TEN-010, announced that it will be acquired by Roche. Seattle, WA’s Juno Therapeutics said it will buy AbVitro, of Boston, MA, to obtain a next-generation sequencing platform to help it create engineered T cells against an array of cancer targets.
- The International Committee of Medical Journal Editors (ICMJE) proposed new rules that will require authors to share clinical trial data as a prerequisite for manuscripts to be considered for publication. The goal is to improve the benefit to society from the efforts of patients who volunteer to participate in clinical trials. The proposal is outlined in the Annals of Internal Medicine and 13 other ICMJE journals.
- The American Cancer Society launched a new website to make it easier to find and share cancer statistics: http://cancerstatisticscenter.cancer.org. It provides detailed information on a range of topics, including estimates of new cancer cases and deaths by sex, state, and cancer type for 2016; current cancer incidence, mortality, and survival rates and trends; and risk factors and screening rates by state.
January 8, 2016
- Steady reductions in smoking, combined with advances in cancer prevention, early detection, and treatment, have resulted in a 23% drop in the cancer death rate since its peak in 1991, averting more than 1.7 million cancer deaths through 2012. Between 2002 and 2012, cancer mortality rates have declined by 1.8% a year in men and 1.4% a year in women—changes driven largely by decreases in death rates for lung, breast, prostate, and colorectal cancers. The findings are included in the American Cancer Society’s latest annual report on cancer incidence, mortality, and survival, published in CA: A Cancer Journal for Clinicians.
- In an agreement worth up to $1.6 billion, Bannockburn, IL–based Baxalta will have exclusive option rights for six checkpoint therapies under development by Symphogen, a private biopharmaceutical company based in Copenhagen, Denmark. Symphogen will receive an up-front payment of $175 million and handle R&D of the immuno-oncology therapies through phase I clinical trials. Baxalta is currently negotiating its sale to Dublin, Ireland–based Shire PLC.
- Roche announced updated results of its pivotal phase II single-arm study of the immunotherapy atezolizumab (MPDL3280A) in people with advanced bladder cancer at the 2016 Genitourinary Cancers Symposium of the American Society of Clinical Oncology. Median overall survival was 11.4 months in people with higher levels of PD-L1 expression and 7.9 months in the overall study population. Low levels of PD-L1 expression did not preclude a response, and complete responses were seen regardless of PD-L1 expression.
- The FDA approved an expanded indication for pembrolizumab (Keytruda; Merck) to include first-line treatment of patients with inoperable or metastatic melanoma, making the anti–PD-1 therapy the first to gain approval for untreated advanced melanoma regardless of BRAF status.
- Woodcliff Lake, NJ–based Eagle Pharmaceuticals announced that the FDA approved its alcohol-free formulation of injectable docetaxel for the treatment of breast, non–small cell lung, prostate, and head and neck cancers, as well as gastric adenocarcinoma. The need for this new formulation came to light after the FDA warned in 2014 that docetaxel may cause symptoms of alcohol intoxication.
- Boehringer Ingelheim announced that the FDA granted Breakthrough Therapy Designation to its tyrosine kinase inhibitor BI 1482694 for the treatment of advanced, previously treated EGFR mutation–positive non–small cell lung cancer, based on results of a phase I/II clinical trial. Objective responses were observed in 62% of patients with the T790M mutation, with a disease control rate of 91%. The designation helps expedite the development and review of promising drugs for serious or life-threatening conditions.
- Researchers at The University of Texas MD Anderson Cancer Center reported that consuming high amounts of sugar may increase the risk of breast cancer and metastasis to the lungs. The findings, published in Cancer Research, demonstrated that sucrose intake in mice comparable to that in the typical Western diet increased tumor growth and metastasis due to increased expression of 12-LOX and a related fatty acid called 12-HETE.
- Three-dimensional structures of faulty proteins and maps of cancer’s communication networks have been added to the canSAR database, which was launched in 2011 by researchers in the Cancer Research UK Cancer Therapeutics Unit at The Institute of Cancer Research in London, United Kingdom. The database was created to help researchers use “Big Data” approaches to build a detailed picture of how the majority of known human molecules behave and to design new cancer treatments more effectively. The canSAR database is available online at https://cansar.icr.ac.uk.
- In the wake of CEO Martin Shkreli’s arrest for securities fraud while he worked for another company, KaloBios filed for Chapter 11 reorganization in U.S. Bankruptcy Court in Wilmington, DE. The South San Francisco, CA, company, which has focused on therapies using engineered antibodies to fight cancer, aims to continue development of its experimental leukemia drug, lenzilumab (KB003).
- Celgene will allow a generic version of its multiple myeloma drug lenalidomide (Revlimid) to be manufactured and sold by India’s Natco Pharma and that company’s U.S. partner, Arrow Limited International, a unit of Allergan, beginning January 31, 2026, Reuters reported. That decision was part of a settlement over patent rights for lenalidomide in the United States, which are not set to expire until April 2027.
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