Timeframe: 2016 – 2019 Goal: Understand growth mechanisms and identify potential therapeutic targets Principal Investigators: John Gordan, MD, PhD (UCSF) and Nabeel Bardeesy, PhD (Mass General Hospital) Study overview: The discovery of a genetic change in the protein kinase A (PKA) gene in nearly all cases of fibrolamellar liver cancer (FLC) creates hope that targeted …
Timeframe: 2017 – 2018 Goal: Characterize inhibition of the DnaJ-PKAc chimeric protein in fibrolamellar carcinoma Principal Investigator: Hibba tul Rehman, MD Study overview: The DNAJB1-PRKACA fusion gene produces the DNAJ-PKAc fusion kinase protein, which is present in nearly all FLC tumors and promotes liver tumor formation in mice. Kinases, including fusion kinases, have been successful …
Timeframe: 2016 – 2018 Goal: Investigate DNAJB1-PRKACA fusion kinase function in new fibrolamellar carcinoma models Principal Investigator: Dr. Yi Guo, PhD Study overview: The DNAJB1-PRKACA fusion kinase is found in nearly 100% of FLC cases. While this novel fusion had previously been shown to promote tumors in mice, defining the mechanistic function of DNAJB1-PRKACA and …
Timeframe: 2017 – 2019 Goal: Evaluate the role of Hedgehog and YAP signaling in fibrolamellar carcinoma Principal Investigators: Cynthia Guy, MD and Anna Mae Diehl, MD Fibrolamellar carcinoma has a unique histological appearance consisting of large tumor cells surrounded by thick fibrous bands, the stroma. Molecular crosstalk between tumor and stroma is important in tumor …
Timeframe: 2017 – 2020 Goal: Investigate the role of microRNAs and long non-coding RNAs in fibrolamellar carcinoma and evaluate RNA-based therapeutics Principal Investigator: Praveen Sethupathy, PhD Study overview: This study aimed to leverage genome-scale approaches to discover the molecular factors most critical to FLC tumor formation, metastasis, and drug resistance. MicroRNAs and long non-coding RNAs are …
Timeframe: 2016 – 2019 Goal: Study the interactions between the immune system and FLC in a mouse model Principal Investigator: Kevin Barry, PhD Study overview: Cancer immunotherapies harness the power of the immune system to kill tumors. Checkpoint blockade immunotherapies are an exciting class of cancer immunotherapies that remove the brakes from the immune system …
Timeframe: 2016 – 2019 Goal: Define the dominant immune checkpoint pathway in FLC Principal investigator: Amy K. Kim, MD Study overview: Tumor cells produce immune checkpoint molecules that suppress host immune response and allow evasion from immune responses. The discovery of drugs that block these immune checkpoints have revolutionized current cancer treatment. Anti-PD1 (programmed cell …
Timeframe: 2016 – 2019 Goal: Characterize T-cells in the FLC tumor microenvironment Principal Investigator: Kevin M. Sullivan, MD Study Overview: Immunotherapy is a form of cancer treatment that harnesses the patient’s own immune system to fight the disease. The immune system can precisely target cancer cells, while also minimizing damage to the remainder of the …
Timeframe: 2020 Goal: Assess whether suppressing checkpoints or signaling by a specific chemokine (CXCL12) can enhance immune response in FLC Principal Investigator: Venu Pillarisetty, MD Study overview: Immunotherapy, harnessing the patient’s immune system to precisely target cancer cells, has emerged as a promising approach to treat many cancers. It has been discovered that fibrolamellar carcinoma …