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There is a type of leukemia which occurs mainly in infants, but also in children who have previously received chemotherapy for an unrelated cancer. This type of leukemia is extremely difficult to treat and often comes back despite chemotherapy. We have evidence that this leukemia relies on a protein called MBNL1. By disrupting MBNL1, leukemia cells cannot produce specific proteins they need to grow. As the Julia's Legacy of Hope St. Baldrick's Scholar, Dr. Lee, as proof of concept, has shown that MBNL1 can be blocked by a chemical inhibitor. By using computer models, testing large libraries of drug-like chemicals, and applying special chemistry techniques to improve how effective a drug is, Dr. Lee is looking for a way to block MBNL1 with a drug that can be used in humans. This will lead to a new therapy for this class of leukemias.

A portion of this grant is named for Julia's Legacy of Hope, a Hero Fund that honors her positive and courageous spirit and carries out Julia's last wish: "no child should have to go through what I have experienced". Diagnosed at age 16 with Ewing sarcoma, Julia fought cancer and survived only to be stricken in college with acute myeloid leukemia, a secondary cancer as a result of treatment. Through this Hero Fund, her family hopes to raise awareness and funds for childhood cancer research especially for Adolescent and Young Adult (AYA) patients.