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First-in-Human Phase I Study of Fisogatinib (BLU-554) Validates Aberrant FGF19 Signaling as a Driver Event in Hepatocellular Carcinoma

Richard D. Kim, Debashis Sarker, Tim Meyer, Thomas Yau, Teresa Macarulla, Joong-Won Park, Su Pin Choo, Antoine Hollebecque, Max W. Sung, Ho-Yeong Lim, Vincenzo Mazzaferro, Joerg Trojan, Andrew X. Zhu, Jung-Hwan Yoon, Sunil Sharma, Zhong-Zhe Lin, Stephen L. Chan, Sandrine Faivre, Lynn G. Feun, Chia-Jui Yen, Jean-Francois Dufour, Daniel H. Palmer, Josep M. Llovet, Melissa Manoogian, Meera Tugnait, Nicolas Stransky, Margit Hagel, Nancy E. Kohl, Christoph Lengauer, Cori Ann Sherwin, Oleg Schmidt-Kittler, Klaus P. Hoeflich, Hongliang Shi, Beni B. Wolf and Yoon-Koo Kang
Richard D. Kim
1H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
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Debashis Sarker
2King's College London, London, United Kingdom.
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Tim Meyer
3University College London, London, United Kingdom.
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Thomas Yau
4Queen Mary Hospital, Hong Kong, China.
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Teresa Macarulla
5Vall d'Hebron University Hospital and Vall d'Hebrón Institute of Oncology (VHIO), Barcelona, Spain.
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Joong-Won Park
6National Cancer Center Korea, Goyang, South Korea.
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Su Pin Choo
7National Cancer Centre Singapore, Singapore.
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Antoine Hollebecque
8Institute Gustav Roussy, Villejuif, France.
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Max W. Sung
9Mount Sinai Liver Cancer Program, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
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Ho-Yeong Lim
10Samsung Medical Center, Sungkyunkwan University, Seoul, Korea.
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Vincenzo Mazzaferro
11University of Milan, Department of Oncology and Instituto Nazionale Tumori, IRCCS Foundation, Department of Surgery, HPB Surgery and Liver Transplantation, Milan, Italy.
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Joerg Trojan
12Universitätsklinikum Frankfurt, Frankfurt, Germany.
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Andrew X. Zhu
13Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
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Jung-Hwan Yoon
14Seoul National University Hospital, Seoul, South Korea.
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Sunil Sharma
15Huntsman Cancer Institute, Salt Lake City, Utah.
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Zhong-Zhe Lin
16National Taiwan University Hospital, Taipei, Taiwan.
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Stephen L. Chan
17State Key Laboratory of Translational Oncology, The Chinese University of Hong Kong, Hong Kong, China.
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Sandrine Faivre
18Hôpitaux Universitaires Paris Nord Val de Seine, Paris, France.
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Lynn G. Feun
19University of Miami, Miami, Florida.
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Chia-Jui Yen
20National Cheng Kung University Hospital, College of Medicine, National Cheng Kung University, Tainan, Taiwan.
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Jean-Francois Dufour
21University Clinic for Visceral Surgery and Medicine, Inselspital Bern, Bern, Switzerland.
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Daniel H. Palmer
22Liverpool Experimental Cancer Medicine Centre, Liverpool, United Kingdom.
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Josep M. Llovet
9Mount Sinai Liver Cancer Program, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
23Translational Research in Hepatic Oncology Group, Liver Unit, IDIBAPS, Hospital Clinic, University of Barcelona, Barcelona, Catalonia, Spain.
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Melissa Manoogian
24Roche Tissue Diagnostics, Tucson, Arizona.
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Meera Tugnait
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Nicolas Stransky
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Margit Hagel
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Nancy E. Kohl
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Christoph Lengauer
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Cori Ann Sherwin
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Oleg Schmidt-Kittler
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Klaus P. Hoeflich
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Hongliang Shi
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Beni B. Wolf
25Blueprint Medicines Corporation, Cambridge, Massachusetts.
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Yoon-Koo Kang
26Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea.
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  • For correspondence: ykkang@amc.seoul.kr
DOI: 10.1158/2159-8290.CD-19-0555 Published December 2019
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Cited By

Article Information

Volume 9, Issue 12, pp. 1696-1707

DOI 
https://doi.org/10.1158/2159-8290.CD-19-0555
PubMed 
31575541

Published By 
American Association for Cancer Research
Print ISSN 
2159-8274
Online ISSN 
2159-8290
History 
  • Received June 5, 2019
  • Revision received August 26, 2019
  • Accepted September 26, 2019
  • Published first October 1, 2019.

Article Versions

  • Previous version (October 1, 2019 - 05:46).
  • Previous version (November 15, 2019 - 06:06).
  • You are viewing the most recent version of this article.
Copyright & Usage 
©2019 American Association for Cancer Research.

Author Information

  1. Richard D. Kim1,
  2. Debashis Sarker2,
  3. Tim Meyer3,
  4. Thomas Yau4,
  5. Teresa Macarulla5,
  6. Joong-Won Park6,
  7. Su Pin Choo7,
  8. Antoine Hollebecque8,
  9. Max W. Sung9,
  10. Ho-Yeong Lim10,
  11. Vincenzo Mazzaferro11,
  12. Joerg Trojan12,
  13. Andrew X. Zhu13,
  14. Jung-Hwan Yoon14,
  15. Sunil Sharma15,
  16. Zhong-Zhe Lin16,
  17. Stephen L. Chan17,
  18. Sandrine Faivre18,
  19. Lynn G. Feun19,
  20. Chia-Jui Yen20,
  21. Jean-Francois Dufour21,
  22. Daniel H. Palmer22,
  23. Josep M. Llovet9,23,
  24. Melissa Manoogian24,
  25. Meera Tugnait25,
  26. Nicolas Stransky25,
  27. Margit Hagel25,
  28. Nancy E. Kohl25,
  29. Christoph Lengauer25,
  30. Cori Ann Sherwin25,
  31. Oleg Schmidt-Kittler25,
  32. Klaus P. Hoeflich25,
  33. Hongliang Shi25,
  34. Beni B. Wolf25 and
  35. Yoon-Koo Kang26,*
  1. 1H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
  2. 2King's College London, London, United Kingdom.
  3. 3University College London, London, United Kingdom.
  4. 4Queen Mary Hospital, Hong Kong, China.
  5. 5Vall d'Hebron University Hospital and Vall d'Hebrón Institute of Oncology (VHIO), Barcelona, Spain.
  6. 6National Cancer Center Korea, Goyang, South Korea.
  7. 7National Cancer Centre Singapore, Singapore.
  8. 8Institute Gustav Roussy, Villejuif, France.
  9. 9Mount Sinai Liver Cancer Program, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
  10. 10Samsung Medical Center, Sungkyunkwan University, Seoul, Korea.
  11. 11University of Milan, Department of Oncology and Instituto Nazionale Tumori, IRCCS Foundation, Department of Surgery, HPB Surgery and Liver Transplantation, Milan, Italy.
  12. 12Universitätsklinikum Frankfurt, Frankfurt, Germany.
  13. 13Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
  14. 14Seoul National University Hospital, Seoul, South Korea.
  15. 15Huntsman Cancer Institute, Salt Lake City, Utah.
  16. 16National Taiwan University Hospital, Taipei, Taiwan.
  17. 17State Key Laboratory of Translational Oncology, The Chinese University of Hong Kong, Hong Kong, China.
  18. 18Hôpitaux Universitaires Paris Nord Val de Seine, Paris, France.
  19. 19University of Miami, Miami, Florida.
  20. 20National Cheng Kung University Hospital, College of Medicine, National Cheng Kung University, Tainan, Taiwan.
  21. 21University Clinic for Visceral Surgery and Medicine, Inselspital Bern, Bern, Switzerland.
  22. 22Liverpool Experimental Cancer Medicine Centre, Liverpool, United Kingdom.
  23. 23Translational Research in Hepatic Oncology Group, Liver Unit, IDIBAPS, Hospital Clinic, University of Barcelona, Barcelona, Catalonia, Spain.
  24. 24Roche Tissue Diagnostics, Tucson, Arizona.
  25. 25Blueprint Medicines Corporation, Cambridge, Massachusetts.
  26. 26Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea.
  1. ↵*Corresponding Author:
    Yoon-Koo Kang, Asan Medical Center, 88, Olympic-ro 43-gil, Songpa-gu, Seoul 05505, South Korea. Phone: 82-2-3010-3230; Fax: 82-2-3010-8772; E-mail: ykkang{at}amc.seoul.kr
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Cancer Discovery: 9 (12)
December 2019
Volume 9, Issue 12
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First-in-Human Phase I Study of Fisogatinib (BLU-554) Validates Aberrant FGF19 Signaling as a Driver Event in Hepatocellular Carcinoma
Richard D. Kim, Debashis Sarker, Tim Meyer, Thomas Yau, Teresa Macarulla, Joong-Won Park, Su Pin Choo, Antoine Hollebecque, Max W. Sung, Ho-Yeong Lim, Vincenzo Mazzaferro, Joerg Trojan, Andrew X. Zhu, Jung-Hwan Yoon, Sunil Sharma, Zhong-Zhe Lin, Stephen L. Chan, Sandrine Faivre, Lynn G. Feun, Chia-Jui Yen, Jean-Francois Dufour, Daniel H. Palmer, Josep M. Llovet, Melissa Manoogian, Meera Tugnait, Nicolas Stransky, Margit Hagel, Nancy E. Kohl, Christoph Lengauer, Cori Ann Sherwin, Oleg Schmidt-Kittler, Klaus P. Hoeflich, Hongliang Shi, Beni B. Wolf and Yoon-Koo Kang
Cancer Discov December 1 2019 (9) (12) 1696-1707; DOI: 10.1158/2159-8290.CD-19-0555

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First-in-Human Phase I Study of Fisogatinib (BLU-554) Validates Aberrant FGF19 Signaling as a Driver Event in Hepatocellular Carcinoma
Richard D. Kim, Debashis Sarker, Tim Meyer, Thomas Yau, Teresa Macarulla, Joong-Won Park, Su Pin Choo, Antoine Hollebecque, Max W. Sung, Ho-Yeong Lim, Vincenzo Mazzaferro, Joerg Trojan, Andrew X. Zhu, Jung-Hwan Yoon, Sunil Sharma, Zhong-Zhe Lin, Stephen L. Chan, Sandrine Faivre, Lynn G. Feun, Chia-Jui Yen, Jean-Francois Dufour, Daniel H. Palmer, Josep M. Llovet, Melissa Manoogian, Meera Tugnait, Nicolas Stransky, Margit Hagel, Nancy E. Kohl, Christoph Lengauer, Cori Ann Sherwin, Oleg Schmidt-Kittler, Klaus P. Hoeflich, Hongliang Shi, Beni B. Wolf and Yoon-Koo Kang
Cancer Discov December 1 2019 (9) (12) 1696-1707; DOI: 10.1158/2159-8290.CD-19-0555
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