Fibrolamellar Cancer Foundation

Development of a human-derived liver progenitor cell line with DNAJB1-PRKACA fusion gene and characterization of mitochondrial dynamics in fibrolamellar carcinoma

Timeframe: 2022 – 2023 Goal: Development of a novel human-derived liver progenitor cell line model of fibrolamellar carcinoma Principal Investigator: Khashayar Vakili, MD Study overview: Currently, very few cell-line models of FLC exist, so the development of additional cellular models is critical for the identification and testing of new therapies. Therefore, one of the aims …

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 …

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 …

Development of a human-derived liver progenitor cell line with DNAJB1-PRKACA fusion gene

Timeframe: 2019 – 2021 Goal: Development of a novel human-derived liver progenitor cell line model of fibrolamellar carcinoma Principal Investigator: Khashayar Vakili, MD Study overview: Prior to this effort, the study team’s lab had previously engineered a kidney cell line (HEK-DP) which contains the DNAJB1-PRKACA fusion gene found in FLC tumors. This cell line demonstrated …