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Targeting WDR5 in MLL rearranged Leukemia
MLL1 is one of the most commonly rearranged genes in human AML and ALL in adults and children, and identifies a patient population with particularly poor prognosis. We recently developed OICR-414, a drug-like small molecule that inhibits the essential WDR5-MLL1 interaction in cells. We will assess OICR-414's effectiveness at disrupting MLL1 function and its effects on leukemic cells in order to explore its potential as a therapeutic agent in leukemia.
Small molecule targeting of STAT5 protein's SH2 domain
Our group has developed a new molecule, AC-3-19, that shows significant promise in the lab and in animal models of leukemia. It works by binding to STAT5 protein in cancer cells and switching it off. Significantly, healthy cells do not suffer side effects, meaning the molecule has the potential to become a gentler, more targeted chemotherapy drug.