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Based on progress to date, Dr. Tarlock was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Acute myeloid leukemia (AML) is a cancer of white blood cells and almost half of children diagnosed with AML will not be cured, even with very intensive chemotherapy and in some cases bone marrow transplant. Many of the mutations in the leukemia that contribute to development of the cancer have been identified, but cannot be used for therapeutic benefit, especially in children. Dr. Tarlock and colleagues have performed genomic testing on the cells of many children diagnosed with AML and found that approximately 40% of children abnormally express the protein mesothelin on their leukemia cells. Dr. Tarlock and colleagues will develop a phase I clinical trial to test a new therapy strategy that uses principles of the immune system to deliver chemotherapy only to mesothelin-positive leukemia cells. They will develop a clinical assay for mesothelin detection in AML to identify children who will benefit from mesothelin-targeted therapy, and investigate methods to optimize disease response to mesothelin-targeting immune therapies.

This grant is generously supported by Rhys’ Pieces of the Cure, a Hero Fund created to honor Rhys Goldman and his journey with cancer. He was diagnosed with pre-B acute lymphoblastic leukemia just 2 weeks before his 6th birthday and endured treatment for three years. Rhys missed a lot of school and life during those years but since marking the end of treatment in July 2018, he has been enjoying swimming, singing in a boys’ choir, chess tournaments, playing with his dogs and going to school. Rhys’ Pieces for the Cure was created to ensure more research is funded for the treatment of pediatric cancer that is specifically focused on less toxic cures for kids.