Noted This Week - 2017 Archive
Archive of cancer-related news briefs, by week, for 2017
February 11–24, 2017
- The U.S. Senate confirmed Georgia Representative Tom Price, MD, as secretary of the Department of Health and Human Services (HHS). The NIH and the FDA fall under the aegis of the HHS.
- Genome-editing tools, such as CRISPR/Cas9, have created new research opportunities and potential treatments for both heritable and nonheritable health conditions, but many people consider the genome “ethically inviolable.” However, according to a report released by the National Academy of Sciences and the National Academy of Medicine, heritable germline editing clinical trials could one day be permitted if certain stringent criteria are met, such as the absence of reasonable alternatives, the development of comprehensive plans for long-term multigenerational follow-up, and credible data on the risks and potential health benefits.
- The government of Botswana announced a $100 million initiative to create a pediatric hematology/oncology treatment network and build clinical infrastructure in southern and east Africa, thanks to $50 million from the Bristol-Myers Squibb Foundation and an additional $50 million to be raised by Houston’s Baylor College of Medicine International Pediatric AIDS Initiative at Texas Children’s Hospital. In the United States, about 80% of children with cancer survive, but in sub-Saharan Africa, the survival rate is estimated to be about 10% due to inadequate health care infrastructure and a lack of physicians and health care providers. The initiative, dubbed Global HOPE, aims to address these challenges and improve the ability to diagnose pediatric blood disorders and cancer in Botswana, as well as in Malawi and Uganda.
- The Cleveland Clinic announced that the new Taussig Cancer Center will open on March 6. Costing an estimated $276 million, the seven-story, 377,000-square-foot facility will house all outpatient cancer treatment services, organized by cancer type. For example, all clinical and treatment areas for breast cancer will be located on the same floor to offer greater convenience for patients.
- The FDA approved lenalidomide (Revlimid; Celgene) as maintenance therapy for patients with multiple myeloma following autologous stem cell transplant. The decision was based upon two randomized, controlled trials that included more than 1,000 patients in total and that demonstrated a 15-month and 18-month progression-free survival advantage for those who received lenalidomide compared with those who received a placebo.
- Novartis announced that the FDA granted Priority Review for the expanded use of ceritinib (Zykadia) as a first-line treatment for patients with metastatic non–small cell lung cancer whose tumors are ALK-positive as detected by an FDA-approved test.
- Cell Medica’s investigational immunotherapeutic CMD-003 received Fast Track designation from the FDA for the treatment of Epstein-Barr Virus (EBV)–associated malignancies. EBV is tied to a variety of cancers, including about 15% to 20% of lymphomas. The therapy consists of autologous EBV-specific T cells that target four EBV-associated antigens; the cells are activated, expanded, and infused back into the patient.
February 10, 2017
- The NCI launched the NCTN/NCORP Data Archive, a centralized repository of deidentified patient-level data from phase III studies carried out by trial groups affiliated with the National Clinical Trials Network (NCTN) and NCI’s Community Oncology Research Program (NCORP). “Greater sharing of research data underlies much of the thinking behind the Beau Biden Cancer Moonshot and the initiatives emerging from it,” the agency noted, and the new database “is building on and contributing to this momentum.”
- The FDA gave French biopharmaceutical company Cellectis the go-ahead to begin phase I evaluations of its universal CAR T-cell therapy, UCART123, in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm. These CD123-targeting cells have been gene-edited, using TALEN, so they can be given to multiple patients without the need for donor–recipient matching. This is the first “off-the-shelf” CAR T-cell product to gain FDA approval for clinical trials, which will be led by Weill Cornell Medicine in New York, NY, and The University of Texas MD Anderson Cancer Center in Houston.
- The nonprofit Chan Zuckerberg (CZ) Biohub selected its first cohort of 47 investigators from three institutions: University of California, Berkeley; University of California, San Francisco; and Stanford University in Palo Alto. Each will receive a 5-year appointment and up to $1.5 million for life science research in their areas of expertise, ranging from biology to physics. These funds will be unrestricted, to spur the pursuit of creative, high-risk projects aimed at helping the CZ Biohub achieve its founders’ goal of “curing, preventing, or managing every disease in our children’s lifetime.”
- A phase II trial of the investigational immune checkpoint inhibitor lirilumab (Innate Pharma) failed to demonstrate clinical efficacy in AML. Among 150 elderly patients randomly assigned to receive lirilumab or placebo, leukemia-free survival and other efficacy endpoints were not statistically different between the study arms. France-based Innate is partnering with Bristol-Myers Squibb (BMS) to develop lirilumab, which targets the KIR2DL receptor family; the drug will continue to be evaluated in combination with BMS’s PD-1 inhibitor nivolumab (Opdivo) against several other cancers.
- GW Pharmaceuticals reported positive results from its phase II trial evaluating two cannabis-derived drugs against recurrent glioblastoma multiforme. The London, UK-based company assessed the combination of tetrahydrocannabinol and cannabidiol (THC:CBD) in 21 patients being treated with temozolomide for their disease, who were randomly assigned to receive THC:CBD or placebo as add-on therapy. The 1-year survival rate was 83% among those given the cannabinoid combination, versus 53% in the control arm.
- According to Fierce Biotech, Eli Lilly plans to cut 200 research and development jobs globally. This move has been framed as a “voluntary reallocation program” in which the company will seek out staffers willing to leave of their own volition. A spokesperson stated that Eli Lilly also plans to increase hiring in strategic areas, including immunology, across its United States research sites; Fierce Biotech noted that given the current political climate, “there is value in being seen to be on board with the ‘America First’ approach, particularly as it relates to jobs.”
February 3, 2017
- Six scientific and medical organizations, including the American Association for Cancer Research, called on the Trump administration to consider the negative impact of denying people from seven countries entry to the United States. “We remain deeply concerned that restricting travel will prohibit participation in scientific meetings, where cutting-edge science and treatment methods are often first introduced,” they said. The order will also limit international collaborations, they noted, and “any loss of researchers and physicians will render the U.S. less competitive over time.”
- President Trump’s proposal to slash regulations around drug development had a mixed reception. According to FierceBiotech, one lawmaker expressed concern that Trump “would consider putting the profits of pharmaceutical corporations ahead of safety.” PhRMA and BIO, however, appeared more upbeat about removing “outdated regulations that drive up costs and slow innovation,” estimating that limiting regulation could create 350,000 jobs over the next decade.
- The House Committee on Energy and Commerce asked President Trump to clarify the potential impact of a federal hiring freeze on the FDA. Noting that the Cures Act, signed into law last December, includes provisions to assist the FDA in recruiting and retaining new scientific and technical staff, the committee queried whether this hiring authority would now be frozen, and, if yes, why. “Hamstringing the ability of our most critical public health agency to hire the personnel needed will hamper the FDA’s ability to fulfill its mission,” they concluded.
- Guardant Health announced a multiyear partnership with The University of Texas MD Anderson Cancer Center in Houston, aimed at making comprehensive liquid biopsy a standard part of cancer care. The company will help MD Anderson build multiple liquid biopsy centers to speed the development of novel noninvasive assays using Guardant’s Digital Sequencing technology.
- Roughly 9.9% of patients with colorectal cancer harbor germline cancer susceptibility gene mutations, researchers reported in the Journal of Clinical Oncology. This prevalence is higher than expected: Until now, physicians have focused on patients with hereditary Lynch syndrome (LS), about 3% in all, who are known to be at high risk for colorectal cancer. Germline mutations were found in non-LS genes, notably BRCA1/2; the researchers concluded that genetic factors underlying this disease “extend beyond well-recognized familial syndromes and are markedly more common than previously appreciated.”
- According to a Nature Medicine study, although Ewing sarcomas share a DNA methylation signature, they also exhibit considerable epigenetic heterogeneity. Examining 140 samples of this bone cancer, the researchers observed consistent hypomethylation around EWS–FLI1, the fusion oncogene that marks this disease. Additionally, they reported epigenetic variation among tumors that fell along a continuum between mesenchymal and stem cell signatures; this heterogeneity was particularly pronounced in patients with metastatic disease.
January 27, 2017
- British researchers generated universal chimeric antigen receptor (CAR) T cells to treat two infants with refractory relapsed CD19+ B-cell acute lymphoblastic leukemia, inducing remission prior to allogenic stem cell transplantation. Both responded within 28 days, according a report published in Science Translational Medicine. Currently, CAR T-cell therapies are developed one at time and personalized for each patient, but this new research suggests that an off-the-shelf, ready-made product could be a viable alternative approach.
- A new analysis reveals that the risk of dying from cervical cancer is higher than previously thought—and that significant racial differences in death rates exist. Unlike prior estimates that included women who had had a hysterectomy and were therefore no longer at risk, this analysis, published in Cancer, included only women with a cervix. The researchers found that black women are dying from cervical cancer at a rate 77% higher than previously thought, whereas white women are dying at a rate 47% higher.
- The FDA greenlighted Culver City, CA–based NantKwest’s application to launch a first-in-human clinical trial evaluating genetically modified natural killer (NK) cells against cancer. Researchers at NantKwest have engineered this immune cell type to express a variant of the CD16 receptor, as well as IL2; preclinically, adding these high-affinity (haNK) cells to therapeutic antibodies boosted antitumor immune responses. In the planned phase I trial, the safety of haNK cells will be evaluated in up to 16 patients with metastatic solid tumors.
- According to a study published in Pediatrics, electronic cigarettes, also called e-cigarettes, are attracting adolescents who might not otherwise have started using tobacco products. Some experts have thought that e-cigarettes might be responsible for a decline in cigarette smoking among youth, but this first national analysis found that combined e-cigarette and cigarette use among middle and high school students in 2014 was higher than total cigarette use in 2009.
- An international, 22-member panel of experts—European LeukemiaNet—released updated recommendations for the diagnosis and treatment of acute myeloid leukemia in adults. The new guidelines, published in Blood, were prompted by recent insights into the molecular and genomic causes of the disease, new genetic tests and tests for detecting minimal residual disease, and the development novel therapies.
- Bristol-Myers Squibb reported positive data on its PD-1 inhibitor nivolumab (Opdivo) in two gastrointestinal cancers. In a phase I/II study of 214 patients with advanced hepatocellular carcinoma, the overall response rate (ORR) to nivolumab was 20%, with a 9-month overall survival (OS) of 74%. In a phase III trial of 493 patients with advanced gastric or gastroesophageal junction cancer, the ORR was 11.2% among those who received nivolumab, versus 0% in those given placebo; nivolumab also more than doubled the 12-month OS, from 10.9% to 26.6%.
- The FDA granted Orphan Drug designation to two investigational agents from New York, NY–based TG Therapeutics for treating diffuse large B-cell lymphoma. The combination of TG-1101, a monoclonal antibody targeting CD20, and TGR-1202, a PI3Kδ inhibitor, is being evaluated in a phase II trial for patients with this disease; orphan status qualifies TG Therapeutics for various development incentives, including tax credits toward the costs of clinical trials.
January 20, 2017
- An NIH spokesman announced that Francis Collins, MD, will continue to serve as the director of the NIH—at least temporarily. It remains unclear whether President Donald Trump will formally reappoint Collins or eventually name a successor.
- The U.S. Department of Health and Human Services’ Office of Human Research Protections published a revised, final version of the so-called Common Rule, originally promulgated in 1991, to modernize and strengthen regulations that protect people who take part in scientific studies. Among other changes, the final rule establishes new requirements regarding information that must be given to prospective participants as part of the informed consent process, and it allows for the use of “broad consent,” meaning that subjects agree to the use of their information in unspecified future research. The rule goes into effect on January 19, 2018.
- The U.S. Supreme Court announced that it will wade into a dispute between Sandoz and Amgen regarding when approved biosimilar versions of drugs can be brought to market. A lower court made Sandoz wait 180 days to sell Zarxio (filgrastim-sndz), a biosimilar version of Amgen’s Neupogen (filgrastim), after earning approval from the FDA; Sandoz has argued that giving Amgen 180 days’ notice prior to marketing the drug was sufficient and that the company shouldn’t have to wait another 6 months following FDA approval to sell its product.
- The FDA issued a guidance document for industry on the nonproprietary naming of biologic products. The nonbinding recommendations call for the use of a “distinguishing suffix” composed of four lowercase letters “devoid of meaning” at the end of names for “originator biological products, related biological products, and biosimilar products containing related drug substances when other means to track a specific dispensed product are not readily accessible or available.” The distinguishing suffixes should, according to the agency, “help minimize inadvertent substitution of any such products that have not been determined to be interchangeable.”
- The FDA also published drafts of several other guidance documents for industry. These include documents titled “Multiple Endpoints in Clinical Trials,” “Considerations in Demonstrating Interchangeability with a Reference Product,” and “Medical Product Communications That Are Consistent with the FDA-Required Labeling—Questions and Answers.” Comments on the draft documents can be submitted electronically at www.regulations.gov.
- Following the presidential election in November, the FDA announced that it would not release a final guidance document on the regulation of laboratory-developed tests (LDT). This week, however, the agency issued a “discussion paper” on LDTs to synthesize comments it received following the publication of a draft document in October 2014 that was designed to balance concerns about overlapping and burdensome regulations with fears that a lack of oversight could compromise patient safety. According to the FDA, the document is meant to promote public discussion about LDT management; it contains no enforceable provisions.
- Reuters reported that 22 of the world’s largest drug companies will contribute seed funding of $50 million over the next 3 years to a World Bank project to fight cancer and other noncommunicable diseases in poor countries. Dubbed Access Accelerated, the initiative was announced in Davos, Switzerland, during the World Economic Forum.
- The Princess Margaret Cancer Foundation and the Princess Margaret Cancer Centre at University Health Network in Toronto, Canada, successfully concluded their “Billion Dollar Challenge” ahead of schedule. In less than 5 years, the organizations secured $532 million in philanthropic support and $520 million in research grants. The money will support personalized cancer medicine, including the expansion of research in epigenetics, bioinformatics, and immunotherapy.
January 13, 2017
- Takeda Oncology acquired ARIAD Pharmaceuticals for $5.2 billion. The Japanese company has been looking to replenish its oncology portfolio, with its patent on bortezomib (Velcade), a blockbuster drug for multiple myeloma, expiring this year. This deal nets Takeda two key drugs: ponatinib (Iclusig), approved for chronic myeloid leukemia and Philadelphia chromosome–positive acute lymphoblastic leukemia; and brigatinib, an investigational ALK inhibitor under FDA review for non–small cell lung cancer (NSCLC).
- The NCI Formulary, a public–private partnership between the agency and the pharmaceutical industry, launched with 15 targeted drugs from six companies. The formulary will enable researchers at NCI-designated cancer centers to more quickly access approved and investigational agents for preclinical studies and clinical trials—an otherwise lengthy negotiation process that can take up to 18 months. The NCI expects to double the number of participating companies and available drugs by the end of 2017.
- Smoking costs the global economy more than $1 trillion annually in healthcare expenditures and lost productivity, according to a report from the World Health Organization and the NCI. By 2030, the number of tobacco-related deaths is projected to increase by a third—from 6 million to 8 million annually—with more than 80% of these occurring in low- and middle-income countries.
- The FDA granted Priority Review to Merck’s PD-1 inhibitor pembrolizumab (Keytruda) combined with pemetrexed plus carboplatin as first-line treatment for advanced or metastatic NSCLC, with a decision expected by May 10. If approved, this could be the first regimen combining chemotherapy with an immunotherapeutic agent for lung cancer, the company said.
- The FDA also granted Priority Review to Roche’s atezolizumab (Tecentriq) as first-line treatment for patients with advanced or metastatic urothelial carcinoma who are ineligible for cisplatin chemotherapy. The PD-L1 inhibitor is currently approved for treating patients with bladder cancer or NSCLC whose disease has progressed during or following platinum-based chemotherapy. A decision is expected by April 30.
- According to a study in Cancer, mammography rates increased among women 70 years of age or older under the Affordable Care Act (ACA). The ACA eliminated out-of-pocket expenses for this preventive service, resulting in more women across all income and education levels opting to be tested, the researchers reported. However, there were no appreciable changes in the rates of colonoscopy, also free under the ACA; the researchers noted that “other procedural factors may remain as deterrents”—for instance, the test’s invasiveness.
January 6, 2017
- The American Association for Cancer Research’s (AACR) Project Genomics Evidence Neoplasia Information Exchange (GENIE) made public nearly 19,000 deidentified genomic records collected from patients with cancer who were treated at one of eight renowned medical centers in the United States, Europe, or Canada. The data cover 59 types of cancer, including genomic information on almost 3,000 patients with lung cancer, more than 2,000 patients with breast cancer, and more than 2,000 with colorectal cancer. Data from Project GENIE, which was launched in November 2015, can be accessed through the AACR at www.aacr.org or downloaded from Sage Bionetworks.
- In an interview with the Washington Post, Vice President Joe Biden said he will create a nonprofit organization so he can continue to focus on cancer issues after leaving office. He indicated his desire to “get Congress and advocacy groups to make sure [cancer] treatments are accessible for everyone … and that we have a more rational way of paying for them while promoting innovation.” He added that the new nonprofit, tentatively called the Biden Cancer Initiative, will be based in Wilmington, DE, or Washington, DC.
- The cost of cancer drugs is the top challenge to cancer care, according to 83% of respondents to the Association of Community Centers’ seventh annual Trends in Cancer Programs survey; last year, 45% of respondents cited drug costs as the most significant challenge. A lack of reimbursement for supportive care services, such as survivorship care and financial counseling; the need for greater understanding among patients of what commercial insurance policies will cover; the burden of paperwork; and the need for greater, sustained funding for cancer research rounded out the top five issues.
- According to a study published in JAMA Dermatology, the national incidence of melanoma in the United States continues to increase, but states in the Northeast are bucking that trend. Between 2003 and 2013, melanoma incidence dropped in five of the region’s nine states—Vermont, Rhode Island, New Hampshire, Connecticut, and Massachusetts. The researchers attributed the decline to strong prevention efforts by groups such as the Melanoma Foundation of New England.
- Halozyme Therapeutics in San Diego, CA, reported positive data from a phase II study of its investigational drug PEGPH20 in advanced pancreatic cancer. PEGPH20, a PEGylated form of recombinant human hyaluronidase, is designed to temporarily degrade hyaluronan—a dense component of the tumor microenvironment that constricts blood vessels around cancer cells and impedes drug delivery. In the study, PEGPH20 plus nab-paclitaxel (Abraxane; Celgene) and gemcitabine nearly doubled median progression-free survival—from 4.5 months to 8.6 months—compared with nab-paclitaxel and gemcitabine alone.
- The UK’s drug-cost watchdog, the National Institute for Health and Care Excellence (NICE), OK’d the use of pertuzumab (Perjeta; Roche), in combination with trastuzumab and chemotherapy, for the treatment of HER2-positive, locally advanced breast cancer—as long as Roche provides the drug at a discount, the amount of which was not disclosed. Several months ago, NICE rejected coverage of pertuzumab, saying that the cost outweighed the drug’s long-term benefits. NICE said that Roche agreed to a smaller price discount on trastuzumab emtansine (Kadcyla), but the drug was still not deemed cost effective.
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