We’re testing a new AI assistant to help patients, caregivers, and researchers find information faster. Click the FCF Assistant button in the lower right corner to try it.
Goal: Create a comprehensive list of potential drug targets for FLC Principal Investigator: Jesse Boehm, PhD Grant length: Three years Study overview: This project is part of the Broad Institute’s Rare Cancer Dependency Map Initiative. The project has three main goals to identify potential FLC therapeutics: If fully successful, this effort will nominate high priority …
Grant length: Two years Study overview: Since the discovery in 2014 of the fusion gene (DNAJB1-PRKACA) that drives fibrolamellar carcinoma (FLC), a drug that would selectively inhibit or destroy the chimeric protein (DNAJ-PKAc) encoded by that gene has been considered a “holy grail” for FLC therapy. Such a drug must effectively target DNAJ-PKAc, while only …
Goal: Determine if targeting CDK7 could be a useful treatment approach for FLC. Principal Investigator: Sean Ronnekleiv-Kelly, MD Grant length: Two years, plus extension Study overview: Unregulated proliferation of cells is a hallmark of cancer. In other words, cancer cells continue to divide and increase in number when normal cells would stop dividing. A family …
Goal: Knock-out gene targets in a mouse model of FLC to identify potentially effective drug combinations for patients with advanced FLC Principal Investigator: Sean Ronnekleiv-Kelly, MD Grant length: One year Study overview: This study will apply an innovative gene-editing based technology (genome-wide CRISPR knockout screen) on a mouse cellular model to screen all possible cancer …
Goal: Investigate the impact on FLC growth and survival of inhibiting two specific oncogenes identified in previous works Principal Investigator: Praveen Sethupathy, PhD Grant length: Three years Study overview: Based on previous epigenomic, metabolomic, and microRNA profiling, as well as initial drug studies, the study team has developed two new exciting hypotheses about therapeutic vulnerabilities …
Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: John Scott, PhD Grant length: Two years Study overview: Before new drugs can move forward into clinical development, scientists must confirm that they work as intended. This effort focuses on enabling this critical step for fibrolamellar carcinoma — validating and understanding how potential drugs interact with …
Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: Rafael Couñago, PhD and David Drewry, PhD Grant length: Two years Study overview: Small-molecule inhibitors of protein kinases have transformed cancer research by improving survival rates and enabling more targeted therapies. This progress stems from the fact that many cancers exhibit dysregulated protein kinases. When these …
Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: John Gordan, MD, PhD Grant length: Two years Study overview: Testing drugs in cells and living organisms is a critical step in developing new treatments. For a drug to work, it must move through complex cell structures and, in animals and eventually humans, overcome challenges like …
Goal: Investigate alternate means of therapeutically targeting DNAJ-PKAc Principal Investigator: John Scott, PhD Grant length: Two years Study overview: Previous work has established that the fusion protein interacts with large number of binding partners as compared to the native protein by increased association with the AKAP proteins (the latter acts as a scaffold). One of …