Timeframe: 2021 – 2024 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 …
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 …
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 …
Timeframe: 2021 – 2022 Goal: Model development Principal Investigator: Mark Yarchoan, MD Study overview: Efforts to identify novel therapies for FLC have been confounded by a lack of preclinical models that accurately reflect the genetics and biology of the disease. This study aimed to establish and validate the first orthotopic, syngeneic, preclinical mouse model of …
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 …
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 …
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 …