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Goal: Investigate alternate means of therapeutically targeting DNAJ-PKAc Principal Investigator: John Scott, PhD Grant length: Two years 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 …
Goal: Identify mechanisms driving FLC and identify potential treatment targets Principal Investigator: Nabeel Bardeesy, PhD Grant length: Two years Study overview: Typically, cancers requires activation of a “driver” of uncontrolled growth, such as the DNAJB1-PRKACA (DP) fusion in FLC, coupled with the loss of a “tumor suppressor” function. Tumor suppressors act as “brakes” on cancers, by causing cells that have gone out of …
Goal: Determine if targeting SLC16A14 could be a useful treatment approach for FLC. Principal Investigator: Sean Ronnekleiv-Kelly, MD Grant length: Two years Study overview: Recently, Dr. Ronnekleiv-Kelly used sophisticated genetic engineering to create a highly promising mouse model of FLC and other cancers driven by the DNAJB1-PRKACA (DP) gene fusion. Dr. Ronnekleiv-Kelly’s first goal in …
Goal: Investigate alternate means of therapeutically targeting DNAJ-PKAc Principal Investigator: Yasemin Sancak, PhD Grant length: Two years 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 …
Goal: Investigate the applicability of using an FDA-approved device to treat fibrolamellar patients Principal Investigator: Hugo Jimenez, PhD Grant length: One year, plus extension Study overview: In this study, Dr. Jimenez and his team will conduct initial feasibility testing with a device that delivers low energy amplitude modulated 27.12 MHz radiofrequency electromagnetic fields (AM RF …
Goal: Investigate the potential of retinoic acid therapy Principal Investigators: Andrew Yen, PhD and Praveen Sethupathy, PhD Grant length: One year 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 …
Goal: Identify functional role of mitochondrial alterations in FLC and FLC-Like BAP1 HCC Principal Investigator: Jessica Zucman-Rossi, MD, PhD Grant length: One year Study overview: Recently, important research studies have reported specific genomic abnormalities of tumor subtypes. One of these, representing the fusion between DNAJB1 and PRKACA genes, has been described to be typical of …
Goal: Determine the molecular mechanisms of FLC and identify new pharmacological agents that could lead to effective therapeutics Principal Investigator: Jin Zhang, PhD Grant length: Two years Study overview: In recent studies, the investigation team discovered that the presence of the FLC oncogenic fusion protein disrupts a membraneless organelle. They showed that loss of this …
Goal: Evaluate selective inhibitors of polo-like kinase 1 (PLK1) for effectiveness in human models of FLC Principal Investigator: Taran Gujral, PhD Grant length: Two years 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 …