Timeframe: 2023 – 2025 Goal: Investigate alternate means of therapeutically targeting DNAJ-PKAc Principal Investigator: John Scott, PhD Study overview: Previous work has established that the fusion protein interacts with large number of binding partners as compared to the native protein by increased association with the AKAP proteins (the latter acts as a scaffold). One of …
Timeframe: 2023 – 2028 Goals: Assess the potential of an inhibitor of glutamine metabolism (DRP-104; sirpiglenastat) in combination with an immune checkpoint inhibitor (durvalumab) as a treatment option for unresectable disease Principal Investigators: Marina Baretti, MD and Mark Yarchoan, MD Study overview: This phase Ib/2 clinical study, led by Dr. Marina Baretti and Dr. Mark …
Timeframe: 2023 – 2025 Goals: Model development and understanding the requirements for FLC tumor formation Principal Investigators: Benedetta Artegiani, PhD; and Delilah Hendriks, PhD Study overview: In comparison with more common cancers, research models of FLC have been extremely limited. Drs. Artegiani and Hendriks have addressed this problem by creating “synthetic” models of FLC, generating …
Timeframe: 2023 – 2025 Goal: Evaluate selective inhibitors of polo-like kinase 1 (PLK1) for effectiveness in human models of FLC Principal Investigator: Taran Gujral, PhD Study overview: Abormal cell signaling by the DNAJ-PKAc fusion protein kinase drives the uncontrolled growth of fibrolamellar carcinoma. Thus far, however, drugs that inhibit the fusion kinase also block the …
Timeframe: 2023 – 2024 Goal: Testing the drug zotatifin (eFT226) against human FLC tumors grown in mice Principal Investigator: John Gordan, MD, PhD Study overview: This “proof of concept” study focuses on testing the drug zotatifin (eFT226) for activity against human FLC tumors grown in mice. This drug is being developed by eFFECTOR Therapeutics (Solana …
Timeframe: 2023 – 2025 Goals: Identifying factors in the tumor environment that impair immune responses to FLC and defining potentially effective immunotherapy strategies Principal Investigators: Venu Pillarisetty, MD (University of Washington); and Kevin Barry, PhD (Fred Hutchinson Cancer Center) Study overview: Immunotherapy – harnessing patients’ immune systems to attack tumor cells – has become established …
Timeframe: 2023 – 2024 Goal: Immunocompetent mouse model development Principal Investigator: Julien Sage, PhD Study overview: Dr. Julien Sage has a very longstanding interest in FLC and is recognized as a world leader in the type of genetic modeling in mice proposed in this study. This study proposes to extend Dr. Sage’s team’s previous work …
Timeframe: 2023 – 2025 Goal: Understand the true incidence, accuracy of diagnosis, and optimal treatments for FLC Principal Investigator: Roshni Dasgupta, MD of the Cincinnati Children’s Hospital Medical Center Co-investigators: Study overview: In previous work, the Pediatric Surgical Oncology Research Collaborative (PSORC) collected and analyzed detailed records of 262 patients under age 20 diagnosed with …