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Validating the RNA signature of fibrolamellar hepatocellular carcinoma

Goal: Validating the RNA signature, expression and function in FLCPrincipal Investigator: Praveen Sethupathy, PhD Grant length: Two years 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 …

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Bioinformatic and molecular analysis of FLC

Goals: Perform molecular analysis for FLC Principal Investigators: W. David Hankins, MD Grant length: One year 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 Protein) analyses …

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

Goals: Identify and launch initial fibrolamellar carcinoma research efforts Principal Investigators: W. David Hankins, MD Grant length: Two years 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 …

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Developmental biology of fibrolamellar hepatocellular carcinoma

Goal: Understand the developmental biology of fibrolamellar carcinomaPrincipal Investigator: Lola Reid, PhD Grant length: One year Study overview: Based on extensive work that Dr. Lola Reid’s Laboratory had already performed on normal liver cells and other forms of liver cancer, the goal of the effort was to attempt to define the development biology stage and …

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High priority FLC investigations

Goal: Support series of high priority FLC investigations Principal Investigator: Sandy Simon, PhD Grant length: One year Study background: In previous work (including the initiatives funded in 2010 and 2011), the study team identified an increase of a patient’s antibodies, in particular the antibody known as IgG (immunoglobin G), in fibrolamellar, sarcomas, and a number …

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Molecular analysis of FLC tumors

Goal: Conduct genomic-based analysis of FLC patients Principal Investigator: Ron DeMatteo, MD Grant length: Two years Study background and overview: At the time of this effort, little was known about the exact mutations driving fibrolamellar carcinoma. Published research on the genomic alterations in FLC was scarce and limited to small studies, with the largest comprised …

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Targeting DNAJB1-PRKACA driven signaling dependencies in FLC

Goals: Investigate the potential of AURKA inhibitors for FLC treatment Principal Investigators: John Gordan, MD, PhD (UCSF) and Nabeel Bardeesy, PhD (MGH) Grant length: Two years Study overview: Even though the DNAJB1-PRKACA gene fusion is sufficient to trigger fibrolamellar liver cancer (FLC), no treatments directed at this target are clinically available. Most FLC patients receive …

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Therapeutic innovations in fibrolamellar cancer

Goal: Understand growth mechanisms and identify potential therapeutic targets Principal Investigator: Raymond Yeung, MD, Professor of Surgery Grant length: Two years Study overview: Fibrolamellar cancer (FLC) is one of the most lethal form of liver cancer in adolescents and young adults. Currently, there is no effective therapy for these patients besides surgery. Armed with a …

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DNAJB1-PKAc-β-catenin-ALCD-dependent activation of cancer genes plays an essential role in fibrolamellar hepatocellular carcinoma

Goal: Document mechanisms driving FLC and test if treatment with inhibitors of β-catenin may be a potential therapeutic approach for FLC Principal Investigator:  Nikolai Timchenko, PhD Grant length: One year Study overview: In the course of studies of pediatric liver cancer, the team at CCHMC has identified chromosomal regions (Aggressive Liver Cancer Domains, ALCDs) in …

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