Fibrolamellar Cancer Foundation

Characterization of mitochondria alterations and their functional consequences in fibrolamellar carcinoma (FLC) and FLC-like BAP1 hepatocellular carcinoma

Timeframe: 2022 – 2024 Goal: Identify functional role of mitochondrial alterations in FLC and FLC-Like BAP1 HCC Principal Investigator: Jessica Zucman-Rossi, MD, PhD Study overview: Recently, important research studies have reported specific genomic abnormalities of tumor subtypes. One of these, representing the fusion between DNAJB1 and PRKACA genes, has been described to be typical of …

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

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 …

Development and Characterization of Patient-derived Models of Fibrolamellar Carcinoma

Timeframe: 2023 – 2025 Goal: Evaluate selective inhibitors of polo-like kinase 1 (PLK1) for effectiveness in human models of FLC Principal Investigator: Taran Gujral, PhD Study overview: Abormal cell signaling by the DNAJ-PKAc fusion protein kinase drives the uncontrolled growth of fibrolamellar carcinoma. Thus far, however, drugs that inhibit the fusion kinase also block the …

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 …

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 …

Expansion conditions for fibrolamellar carcinoma (FLC) organoids

Timeframe: 2019 – 2020 Goal: Identify optimal culture conditions for FLC organoidsPrincipal Investigator: Jian Liu, PhD Study overview: Scientific investigators often use cultures, dishes containing tumor cells, to test candidate treatments for a disease. The type of cultures that most accurately reflect the properties of cells as if they were inside the body are “organoids”, …

Validating the RNA signature of fibrolamellar hepatocellular carcinoma

Timeframe: 2015 – 2017 Goal: Validating the RNA signature, expression and function in FLCPrincipal Investigator: Praveen Sethupathy, PhD Study overview: In past work at UNC, a survey of numerous drugs under consideration for FLC use proved non-effective or minimally effective in limiting the growth of the first FLC tumor model. This indicated that completely new …

Bioinformatic and molecular analysis of FLC

Timeframe: 2010 – 2011 Goals: Research coordination and molecular analysis for FLC Principal Investigators: W. David Hankins, MD Study overview: In addition to coordinating ICARE’s fibrolamellar exploration efforts (described separately) during FCF’s one-year partnership with ICARE, Dr. Hankins’ lab received a “Freedom of Pursuit” unrestricted grant, intended to fund bioinformatics and molecular (DNA, RNA and …

International Cancer Alliance for Research and Education (ICARE) research effort coordination

Timeframe: 2010 – 2011 Goals: Identify and launch initial fibrolamellar carcinoma research efforts Principal Investigators: W. David Hankins, MD Study overview: The International Cancer Alliance for Research and Education (ICARE) focuses its programs on building and funding local and international medical academic research teams (I-TEAMS) that identify, initiate and fund cutting-edge research projects to support …