This article, published in Nature Reviews Gastroenterology & Hepatology, summarizes the key advances made in understanding the disease since the 2014 discovery that nearly all FLC tumors contain the DNAJB1-PRKACA chimeric fusion transcript. The paper outlines the advances made in understanding the molecular pathways driven by the fusion protein and the potential to identify actionable therapeutic targets using that information; the development and availability of model systems, including cell lines and mouse models to effectively study the cancer; the potential for immunotherapies to play a major role in treatment protocols; and recent results of clinical trials for the disease.
Most importantly, it outlines a roadmap to drive ongoing initiatives and prioritize critical actions for developing effective treatments for the disease. This roadmap reviews key unanswered questions and describes tangible next steps in four key areas:
- Defining the FLC patient base
- Developing targeted molecular therapies
- Developing immunotherapies
- Devising effective clinical regimens.
This roadmap is an outcome of the multi-institutional collaborations fostered by the Fibrolamellar Cancer Foundation’s biennial Fibrolamellar Research Summit where FLC researchers share current advances in laboratory and clinical studies, and assess opportunities to make additional progress. The full article can be read or downloaded below: