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Acute myeloid leukemia (AML) is an aggressive and molecularly heterogeneous cancer. Despite recent progress in developing new agents, front-line therapeutic protocols have not changed markedly over the past two decades and are associated with substantial morbidity and mortality. Accordingly, there is a need to develop novel mechanism-based treatments. Pediatric AML patients with NUP98 (nuclear pore complex protein 98) gene rearrangements (NUP98-r) have particularly dismal outcomes, with overall survival rates of 25-35%. Dr. Tian and colleagues are studying important proteins that contribute to a cell's ability to become cancerous in one of the most common types of NUP98-r AML: NUP98::KDM5A. Dr. Tian is also optimizing a compound's ability to directly inhibit KDM5A. Identification of these targets will allow for development of new drugs in the treatment of NUP98-r pediatric AML.

This grant is funded by Allied World, a global provider of insurance and reinsurance solutions.