Developmental biology of fibrolamellar hepatocellular carcinoma

Timeframe: 2010 – 2011 Goal: Understand the developmental biology of fibrolamellar carcinomaPrincipal Investigator: Lola Reid, PhD 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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Analysis of the immunology, imaging and cell biology of fibrolamellar

Timeframe: 2010 – 2011 Goal: Build understanding of the immunology and cell biology of FLC using mouse models of cancer Principal Investigator: Sandy Simon, PhD Study background: This early study was designed to determine if researchers could take advantage of the immune system to identify potential ways to detect and eventually treat fibrolamellar. The immune …

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High priority FLC investigations

Timeframe: 2014 – 2015 Goal: Support series of high priority FLC investigations Principal Investigator: Sandy Simon, PhD 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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Creation of Fibrolamellar Tissue Repository at The Rockefeller University

Timeframe: 2013 – 2014 Goal: Support costs related to establishment of new FLC biospecimen storage facility Principal Investigator: Sandy Simon, PhD Study background: Access to tumor tissue is critical to help cancer researchers understand what drives a disease and how the cancer responds to treatments. Because fibrolamellar is so rare, there has long been a …

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Molecular analysis of FLC tumors

Timeframe: 2013 – 2016 Goal: Conduct genomic-based analysis of FLC patients Principal Investigator: Ron DeMatteo, MD 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

Timeframe: 2019 – 2022 Goals: Investigate the potential of AURKA inhibitors for FLC treatment Principal Investigators: John Gordan, MD, PhD (UCSF) and Nabeel Bardeesy, PhD (MGH) 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

Timeframe: 2017 – 2019 Goal: Understand growth mechanisms and identify potential therapeutic targets Principal Investigator: Raymond Yeung, MD, Professor of Surgery 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

Timeframe: 2020 – 2021 Goals: 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 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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