Fibrolamellar Cancer Foundation

How fibrolamellar carcinoma differs from hepatocellular carcinoma in children – Creation of a multi-institutional database including central pathologic review

Timeframe: 2023 – 2025 Goal: Understand the true incidence, accuracy of diagnosis, and optimal treatments for FLC Principal Investigator: Roshni Dasgupta, MD of the Cincinnati Children’s Hospital Medical Center Co-investigators: Study overview: In previous work, the Pediatric Surgical Oncology Research Collaborative (PSORC) collected and analyzed detailed records of 262 patients under age 20 diagnosed with …

Selective translational inhibition as a treatment strategy for fibrolamellar liver cancer

Timeframe: 2023 – 2024 Goal: Testing the drug zotatifin (eFT226) against human FLC tumors grown in mice Principal Investigator: John Gordan, MD, PhD Study overview: This “proof of concept” study focuses on testing the drug zotatifin (eFT226) for activity against human FLC tumors grown in mice. This drug is being developed by eFFECTOR Therapeutics (Solana …

Targeting DNAJB1-PRKACA driven signaling dependencies in FLC

Timeframe: 2019 – 2022 Goals: Investigate the potential of AURKA inhibitors for FLC treatment Principal Investigators: John Gordan, MD, PhD (UCSF) and Nabeel Bardeesy, PhD (MGH) Study overview: Even though the DNAJB1-PRKACA gene fusion is sufficient to trigger fibrolamellar liver cancer (FLC), no treatments directed at this target are clinically available. Most FLC patients receive …

Defining incidence of fibrolamellar carcinoma through computational analysis of multiple data types

Timeframe: 2021 – 2022 Goal: Develop updated estimate for US national incidence of FLC Investigators: John Gordan, MD, PhD (UCSF); Travis Zack, MD (UCSF); and Kurt Losert (FCF) Study overview: Rare diseases like fibrolamellar are often poorly understood because of the difficulty in assembling accurate patient cohorts and the lack of ICD billing codes specific …

Flipping the switch on PKA: synthetic lethal approaches to block PKA-driven tumor growth in fibrolamellar liver cancer

Timeframe: 2016 – 2019 Goal: Understand growth mechanisms and identify potential therapeutic targets Principal Investigators: John Gordan, MD, PhD (UCSF) and Nabeel Bardeesy, PhD (Mass General Hospital) Study overview: The discovery of a genetic change in the protein kinase A (PKA) gene in nearly all cases of fibrolamellar liver cancer (FLC) creates hope that targeted …