Timeframe: 2024 – 2025 Study overview: Since the discovery in 2014 of the fusion gene (DNAJB1-PRKACA) that drives fibrolamellar carcinoma (FLC), a drug that would selectively inhibit or destroy the chimeric protein (DNAJ-PKAc) encoded by that gene has been considered a “holy grail” for FLC therapy. Such a drug must effectively target DNAJ-PKAc, while only …
Timeframe: 2024 – 2026 Goal: Determine if targeting CDK7 could be a useful treatment approach for FLC. Principal Investigator: Sean Ronnekleiv-Kelly, MD Study overview: Unregulated proliferation of cells is a hallmark of cancer. In other words, cancer cells continue to divide and increase in number when normal cells would stop dividing. A family of 20 …
Timeframe: 2022 – 2023 Goal: Knock-out gene targets in a mouse model of FLC to identify potentially effective drug combinations for patients with advanced FLC Principal Investigator: Sean Ronnekleiv-Kelly, MD Study overview: This study will apply an innovative gene-editing based technology (genome-wide CRISPR knockout screen) on a mouse cellular model to screen all possible cancer …
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: 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 …
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”, …
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 …
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 …
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 …