Timeframe: 2019 – 2023 Goal: Investigate the potential of heat shock protein 70 (Hsp70) and mitogen-activated protein kinases (MAPKs) as therapeutic options for FLC Principal Investigator: John Scott, PhD Study overview: Precision medicine approaches have identified the underlying genetic defect in FLC as a deletion in chromosome 19. Consequently, FLC patients produce a unique protein …
Timeframe: 2019 – 2023 Goal: Investigate the potential of retinoic acid therapy Principal Investigators: Andrew Yen, PhD and Praveen Sethupathy, PhD Study overview: FLC is driven by the DNAJ-PKAc fusion protein. A potential therapeutic strategy would be to induce loss of this key driver protein. One approach to substantially alter gene expression in cancer cells …
Timeframe: 2019 – 2023 Goal: Assess potential to induce immune response with a FLC therapeutic vaccine Principal Investigator: Mark Yarchoan, MD Study overview: FLC is a rare and often lethal form of liver cancer for which there is no standard treatment option beyond surgery. Immune checkpoint inhibitors are a revolutionary new form of cancer therapy. …
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 (encoding a chimeric protein with a domain of heat shock protein 40, HSP40, fused to a majority of the enzymatically active …
Timeframe: 2019 – 2020 Goal: Assess whether supressing checkpoints or signaling by a specific chemokine (CXCL12) can enhance immune response 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 …
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 …