Disruption of PKA RIα phase separation by the oncogenic fusion protein in FLC

Timeframe: 2021 – 2023 Goals: Determine the molecular mechanisms of FLC and identify new pharmacological agents that could lead to effective therapeutics Principal Investigator: Jin Zhang, PhD Study overview: In recent studies, the investigation team discovered that the presence of the FLC oncogenic fusion protein disrupts a membraneless organelle. They showed that loss of this …

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Liquid Biopsy for Active Surveillance of Fibrolamellar Hepatocellular Carcinoma

Timeframe: 2021 – 2023 Goal: Develop a blood-based screening method to detect relapse in FLC patients Principal Investigator: Gary S. Stein, PhD Study overview: This study is focused on developing a blood-based screening method to detect relapse in FLC patients that have been diagnosed and treated by surgery. This screening protocol is intended to be …

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Molecular therapies for fibrolamellar carcinoma (FLC) – extension

Timeframe: 2021 – 2023 Goal: Investigate the potential of heat shock protein 70 (Hsp70) and kinase inhibitors as potential therapeutic options in pre-clinical models Principal Investigator: John Scott, PhD Study overview: In the previously funded work, the investigators discovered that DNAJ-PKAc forms a complex with heat shock protein 70 (Hsp70) and mitogenic kinases (kinase enzymes …

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CRISPR-engineering of human liver organoids to study fibrolamellar carcinoma

Timeframe: 2021 – 2022 Goal: Model development Principal Investigator: Benedetta Artegiani, PhD Study Overview: Research into FLC is complicated because of the limited experimental tools that can be used to study this rare cancer. While tests in laboratory animals such as mice are useful tools, they do not reflect the human disease due to inherent …

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A gene editing approach to develop new and effective models for FLC

Timeframe: 2021 Goal: Generate a mouse model of FLC Principal Investigator: Sean Ronnekleiv-Kelly, MD Study overview: The purpose of this study was to generate a robust pre-clinical murine model of FLC that can provide a basis for understanding factors contributing to FLC formation, and for therapeutic development. The study proposed to develop a mouse model …

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