Timeframe: 2020 – 2022 Goal: Model development Principal Investigator: Nabeel Bardeesy, PhD Study overview: While multiple FLC models have recently been developed, additional model development remains a priority for the FLC community. This effort aimed to develop multiple different FLC models as resources for the FLC research fibrolamellar cancer research community, collaborating with the Fibrolamellar …
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: 2021 – 2022 Goal: Develop updated estimate for US national incidence of FLC Investigators: John Gordan, MD, PhD (UCSF); Travis Zack, MD (UCSF); and Kurt Losert (FCF) Study overview: Rare diseases like fibrolamellar are often poorly understood because of the difficulty in assembling accurate patient cohorts and the lack of ICD billing codes specific …
Timeframe: 2010 – 2014 Goal: Assess potential of everolimus (RAD001) as a treatment option for FLC Principal Investigator: Ghassan Abou-Alfa, MD Study background and overview: At the time of this trial, the molecular drive of fibrolamellar, the DNAJB1-PRKACA fusion gene, had not been identified. However, several factors suggested that estrogen and estrogen-dependent pathways may play …
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: 2014 – 2015 Goal: Investigate the potential of radioactive iodine as a therapeutic in FLC Principle Investigator: Nancy Carrasco, MD (Currently at Vanderbilt University) Study background: FLC cells express high levels of the iodine transporter NIS, raising the possibility that radioactive iodine may be a potential therapeutic for FLC. Radioactive iodine has a long …
Timeframe: 2013 – 2014 Goal: Identify genetic mutations in FLC Principal Investigator: Sandy Simon, PhD Study background: The goal of this project was to identify genetic mutations present in FLC. Identification of mutations common in FLC tumors is critical to enhancing the understanding of the underlying biology of this tumor and allow for development of …
Timeframe: 2016 – 2017 Goal: Develop new therapeutics for FLC Principal Investigator: Sandy Simon, PhD Investigator Collaboration: Barbara Lyons, PhD (New Mexico State University) Study overview: The goal of this work was to identify potential therapeutic strategies to target FLC. The team identified three independent approaches to analyze based on their laboratory’s work showing that …