Fibrolamellar Cancer Foundation

A Pilot Study of a DNAJB1-PRKACA fusion kinase vaccine combined with nivolumab and ipilimumab for patients with fibrolamellar carcinoma (FLC)

Timeframe: 2019 – 2023 Goal: Assess potential to induce immune response with a FLC therapeutic vaccine Principal Investigator: Mark Yarchoan, MD Study overview: Many patients with FLC currently receive an immune checkpoint inhibitor off-label. These drugs “take the brakes off” the immune system, enhancing its ability to fight cancer. However, clinical experience suggests that they …

Retinoic acid-induced loss of DNAJB1-PRKACA fusion protein expression

Timeframe: 2019 – 2023 Goal: Investigate the potential of retinoic acid therapy Principal Investigators: Andrew Yen, PhD and Praveen Sethupathy, PhD Study overview: FLC is driven by the DNAJ-PKAc fusion protein. A potential therapeutic strategy would be to induce loss of this key driver protein. One approach to substantially alter gene expression in cancer cells …

Molecular therapies for fibrolamellar carcinoma (FLC)

Timeframe: 2019 – 2021 Goal: Investigate the potential of heat shock protein 70 (Hsp70) and mitogen-activated protein kinases (MAPKs) as therapeutic options for FLC Principal Investigator: John Scott, PhD Study overview: A main goal of this project was to determine how the abnormal form of protein kinase A (PKA) present in fibrolamellar carcinoma (FLC) cells …

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 …

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 …