Biochemical and functional characterization of DNAJ-PKAc modulators

Timeframe: 2025 – 2027 Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: John Scott, PhD Study overview: Before new drugs can move forward into clinical development, scientists must confirm that they work as intended. This effort focuses on enabling this critical step for fibrolamellar carcinoma — validating and understanding how potential drugs interact with …

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Molecular therapies for fibrolamellar carcinoma (FLC) – continuation

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 …

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The Metabolic Implications and Targetability of Mitochondrial Calcium Signaling in FLC

Timeframe: 2025 – 2027 Goal: Investigate alternate means of therapeutically targeting DNAJ-PKAc Principal Investigator: Yasemin Sancak, PhD Study overview: This effort, led by Yasemin Sancak of the University of Washington, aims to study mitochondrial abnormalities in FLC. It has long been known that FLC cancer cells are filled with abnormal mitochondria, but the causes and implications of those mitochondrial changes are …

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Therapeutic modulation of tumor-infiltrating T cell function in fibrolamellar carcinoma

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 …

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How fibrolamellar carcinoma differs from hepatocellular carcinoma in children – Creation of a multi-institutional database including central pathologic review

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 …

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Molecular therapies for fibrolamellar carcinoma (FLC) – extension

Timeframe: 2021 – 2023 Goal: Investigate the potential of heat shock protein 70 (Hsp70) and kinase inhibitors as potential therapeutic options in pre-clinical models Principal Investigator: John Scott, PhD Study overview: In the previously funded work, the investigators discovered that DNAJ-PKAc forms a complex with heat shock protein 70 (Hsp70) and mitogenic kinases (kinase enzymes …

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Molecular therapies for fibrolamellar carcinoma (FLC)

Timeframe: 2019 – 2021 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: A main goal of this project was to determine how the abnormal form of protein kinase A (PKA) present in fibrolamellar carcinoma (FLC) cells …

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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 …

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T cell immunotherapy in fibrolamellar cancer

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 …

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