Road map for fibrolamellar carcinoma: progress and goals of a diversified approach

Abstract

Fibrolamellar carcinoma is a rare liver cancer, which primarily afflicts adolescents and young adults worldwide and is frequently lethal. Given the rarity of this disease, patient recruitment for clinical trials remains a challenge. In November 2017, the Second Fibrolamellar Cancer Foundation Scientific Summit (Stamford, CT, USA) provided an opportunity for investigators to discuss recent advances in the characterization of the disease and its surrounding liver and immune context. The Fibrolamellar Cancer Foundation has thus set out a road map to identify and test therapeutic targets in the most efficient possible manner.

Keywords:

• fibrolamellar carcinoma
• liver cancer
• adolescent
• young adult
• fusion oncogene
• cyclic AMP-dependent protein kinase catalytic subunit
• heat shock protein 40

Acknowledgments

We would like to thank the patients, their families, the Fibrolamellar Cancer Foundation, and their donors for their support. We are particularly grateful to Marna and Charles Davis for their dedication and founding of the Foundation, and John Hopper for his contribution and initiative for supporting the meeting and this report. We thank Praveen Senthupathy, Lola Reid, Kaloyan Tsanov, Pilar Mendoza, and Francisco Barriga for valuable critical feedback. ERK would like to thank Scott Lowe (MSKCC) for his mentorship. SDO is supported by the Cancer Research Institute/Fibrolamellar Cancer Foundation. ERK is supported by an F31 NRSA predoctoral fellowship from the NCI/National Institutes of Health under award number F31CA192835.

Disclosure

GKAA reports institutional research support from Acta Biologica, Agios, Array, Astra Zeneca, Bayer, Beigene, BMS, Casi, Celgene, Exelixis, Genentech, Halozyme, Incyte, Lilly, Mabvax, Novartis, OncoQuest, Polaris Puma, QED, and Roche, and consulting support from 3D Medcare, Agios, Alignmed, Amgen, Antengene, Aptus, Aslan, Astellas, Astra Zeneca, Bayer, Beigene, Bioline, BMS, Boston Scientific, Bridgebio, Carsgen, Celgene, Casi, Cipla, CytomX, Daiichi, Debio, Delcath, Eisai, Exelixis, Genoscience, Gilead, Halozyme, Hengrui, Incyte, Inovio, Ipsen, Jazz, Jansen, Kyowa Kirin, LAM, Lilly, Loxo, Merck, Mina, Newlink Genetics, Novella, Onxeo, PCI Biotech, Pfizer, Pharmacyte, Pharmacyclics, Pieris, QED, Redhill, Sanofi, Servier, Silenseed, Sillajen, Sobi, Targovax, Tekmira,Twoxar, Vicus, Yakult, and Yiviva. The authors report no other conflicts of interest in this work.