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Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: Rafael Couñago, PhD and David Drewry, PhD Grant length: Two years Study overview: Small-molecule inhibitors of protein kinases have transformed cancer research by improving survival rates and enabling more targeted therapies. This progress stems from the fact that many cancers exhibit dysregulated protein kinases. When these …
Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: John Gordan, MD, PhD Grant length: Two years Study overview: Testing drugs in cells and living organisms is a critical step in developing new treatments. For a drug to work, it must move through complex cell structures and, in animals and eventually humans, overcome challenges like …
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: Provide a biological rationale for biomarker-guided testing of antibody drug conjugates in FLC Principal Investigator: Mark Yarchoan, MD Grant length: Six months Study overview: Cancer cells often overexpress many proteins on their surface. Because of these differences, cancer cells can have unique surface “signatures” that can be used to specifically target them with therapies. These molecules can also …
Goal: Use AI-driven drug discovery tools to identify potential binders to the DNAJ-PKAc fusion protein Principal investiogator: Hannes Nissen-Meyer Grant length: One year Study overview: Khumbu proposes to support FLC drug discovery efforts by applying its AI- and quantum mechanics-driven computational platform to identify and optimize small-molecule inhibitors of the DNAJ-PKAc fusion protein, the known …
Goal: To identify and optimize targeted protein degraders that selectively eliminate the cancer-driving DNAJ-PKAc fusion protein in fibrolamellar carcinoma while sparing normal proteins Principal Investigator: Fleur Ferguson, PhD Grant length: Six months Study overview: Dr. Fleur Ferguson (UC San Diego), in collaboration with Dr. Nabeel El-Bardeesy (Massachusetts General Hospital), is exploring whether targeted protein degradation …
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 …