Fibrolamellar Cancer Foundation

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

Timeframe: 2010 – 2011 Goals: Identify and launch initial fibrolamellar carcinoma research efforts Principal Investigators: W. David Hankins, MD 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 …

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 …

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 …

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 …

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 …

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 …

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 …

Iodine transporter research

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 …

Genomic initiative on Fibrolamellar Hepatocellular Carcinoma

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 …