Timeframe: 2021 – 2022 Goal: Model development Principal Investigator: Mark Yarchoan, MD Study overview: 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 aimed to establish and validate the first orthotopic, syngeneic, preclinical mouse model of …
Timeframe: 2021 – 2022 Goal: Model development Principal Investigator: Benedetta Artegiani, PhD Study Overview: 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 …
Timeframe: 2010 – 2011 Goal: Develop useful disease models to support subsequent research efforts Principal Investigator: Yuzhou Wang, PhD Study overview: Availability of model system to replicate the disease is an essential tool in development of therapeutics. These model systems can be used by researchers for mechanistic investigations, rapid drug screening, and many other purposes. Model …
Timeframe: 2010 Goal: Identify biomarkers for FLC Principal Investigator: Michael Torbenson, MD (Currently at Mayo Clinic) Study overview: 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 collection of …
Timeframe: 2016 – 2019 Goal: Develop a pre-clinical mouse model for fibrolamellar carcinoma Principal Investigator: Dr. Julien Sage, PhD Study overview: 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 many clinical trials. One solution …
Timeframe: 2016 – 2019 Goal: Use zebrafish as a model system for fibrolamellar carcinoma to study the immune system Principal Investigator: Sofia de Oliveira, PhD Study overview: 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 useful, animal models allow …
Timeframe: 2021 Goal: Generate a mouse model of FLC Principal Investigator: Sean Ronnekleiv-Kelly, MD Study overview: The purpose of this study was to generate a robust pre-clinical murine model of FLC that can provide a basis for understanding factors contributing to FLC formation, and for therapeutic development. The study proposed to develop a mouse model …
Timeframe: 2019 – 2021 Goal: Development of a novel human-derived liver progenitor cell line model of fibrolamellar carcinoma Principal Investigator: Khashayar Vakili, MD Study overview: Prior to this effort, the study team’s lab had previously engineered a kidney cell line (HEK-DP) which contains the DNAJB1-PRKACA fusion gene found in FLC tumors. This cell line demonstrated …