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Goal: Determine the molecular mechanisms of FLC and identify new pharmacological agents that could lead to effective therapeutics Principal Investigator: Jin Zhang, PhD Grant length: Two years 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 …
Goal: Develop a blood-based screening method to detect relapse in FLC patients Principal Investigator: Gary S. Stein, PhD Grant length: One year 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 …
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 Grant length: Two years 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 …
Goal: Model development Principal Investigator: Mark Yarchoan, MD Grant length: One year 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 …
Goal: Develop updated estimate for US national incidence of FLC Investigators: John Gordan, MD, PhD (UCSF); Travis Zack, MD (UCSF); and Kurt Losert (FCF) Grant length: One year 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 …
Goal: Model development Principal Investigator: Benedetta Artegiani, PhD Grant length: Two years 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 …
Goal: Generate a mouse model of FLC Principal Investigator: Sean Ronnekleiv-Kelly, MD Grant length: One year 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 …