Liquid Biopsy for Active Surveillance of Fibrolamellar Hepatocellular Carcinoma

Timeframe: 2021 – 2022 Goal: Develop a blood-based screening method to detect relapse in FLC patients Principal Investigator: Gary S. Stein, PhD, Department of Biochemistry, University of Vermont Cancer Center 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 …

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Fibrolamellar carcinoma model development and analysis

Timeframe: 2019 – 2022 Goal: Model development Principal Investigator: Nabeel Bardeesy, PhD, Associate Professor, Massachusetts General Hospital, Harvard University Multiple FLC models have recently been developed, which have advanced our understanding of this disease. However, additional model development remains a priority for the FLC community. This effort aims to develop multiple different FLC models as …

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Creating a fibrolamellar cancer dependency map

Timeframe: 2020 – 2023 Goal: Create a comprehensive list of potential drug targets for FLC Principal Investigator: Jesse Boehm, PhD, Institute Scientist, Director of the Broad Cancer Model Development Center This project is part of the Broad Institute’s Rare Cancer Dependency Map Initiative. The project has three main goals to identify potential FLC therapeutics: Developing …

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Development and validation of an orthotopic, syngeneic, mouse model for fibrolamellar hepatocellular carcinoma

Timeframe: 2021 – 2022 Goal: Model development Principal Investigator: Mark Yarchoan, MD, Associate Professor, Oncology/Division of GI Malignancies, Sidney Kimmel Comprehensive Cancer Center 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 aims to establish and …

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CRISPR-engineering of human liver organoids to study fibrolamellar carcinoma

Goal: Model development Principal Investigator: Benedetta Artegiani, PhD, Artegiani Group, Princess Maxima Center 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 differences …

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Development of a human-derived liver progenitor cell line with DNAJB1-PRKACA fusion gene and characterization of mitochondrial dynamics in fibrolamellar carcinoma

Timeframe: 2022 – 2023 Goal: Development of a novel human-derived liver progenitor cell line model of fibrolamellar carcinoma Principal Investigator: Khashayar Vakili, MD, Surgical Director of Liver, Kidney, Intestine, and Multivisceral Transplant Programs, Assistant Professor of Surgery, Harvard Medical School Currently, very few cell-line models of FLC exist, so the development of additional cellular models …

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Blood markers for fibrolamellar

Timeframe: 2010 Goal: Identify biomarkers Principal Investigator: Michael Torbenson, MD, Department of Pathology, Johns Hopkins University (Currently at Mayo Clinic) Early identification of tumors is essential for aggressive treatment. The majority of fibrolamellar carcinoma (FLC) patients have metastatic disease at the time of diagnosis. Therefore, identification of biomarkers for FLC is essential. Using a broad …

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Developing pre-clinical models for fibrolamellar FL-HCC: therapeutic target identification and testing

Timeframe: 2016 – 2018 Goal: Develop a pre-clinical mouse model for fibrolamellar carcinoma Principal Investigator: Dr. Julien Sage, Ph.D., Associate Professor, Department of Pediatrics and Genetics There are few effective therapies for FLC patients and development of improved therapeutics are hampered by the rarity of the disease and the challenge of including pediatric patients in …

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Role of the innate immune system in fibrolamellar hepatocellular carcinoma (FL-HCC) using zebrafish as a model system

Timeframe: 2016 – 2019 Goal: Use zebrafish as a model system for fibrolamellar carcinoma to study the immune system Principal Investigator: Sofia de Oliveira, Ph.D., EMBO Postdoctoral Fellow, Huttenlocher Immunology Lab Few FLC animal models currently exist limiting our ability to study FLC in the context of a complete organism. While cell-based models are extremely …

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