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Acute myeloid leukemia (AML) as an aggressive pediatric cancer associated with poor outcomes and few changes in therapies over the past 30 years. The presence of small numbers of persisting leukemia cells after chemotherapy has become an important predictor of leukemia relapse, however, current assays used to detect residual leukemia have limited sensitivity and many patients with “no detectable leukemia” still go on to relapse. This underscores the need to identify and develop novel assays that more accurately determine optimal therapies and that improve upon current leukemia detection approaches for AML. Dr. Meshinchi and colleagues have performed functional genomic profiling (RNA sequencing) in 2000 children, adolescents, and young adults diagnosed with AML over the past 25 years that includes diagnosis, remission, and relapse timepoints. This preliminary data suggests that deep and functional cancer profiling across an unprecedented number of patient samples and timepoints informs relapse risk, enables a precision medicine approach that considers specific alterations within a patient’s specific cancer, and links diagnosis and relapse profiles with the goal of better understanding how/why relapses occur and how best to prevent them. Therefore, Dr. Meshinchi and colleagues plan to leverage this unprecedented dataset to develop an integrated genomic platform that will significantly improve prognostic determination and treatment decisions for children, adolescents, and young adults diagnosed with AML. Successful validation of our assays will therefore fill a critical unmet need in the field of AML, and the resulting product will be an optimized test ready for clinical use.

This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Childhood acute myeloid leukemia (AML) is the second most common leukemia in children, but causes the majority of leukemia related deaths. Current therapies do not adequately treat the disease and more than half of all children diagnosed with AML eventually relapse and die of their disease. Dr. Meshinchi recently performed a comprehensive genetic analysis of pediatric AML and identified mutations in CSF3R gene, the first pediatric-specific mutation in AML. This JJ's Angels St. Baldrick's Research Grant aims to study the function of this mutation to learn how it might be used as a therapeutic target.

This grant is named for the JJ's Angels Hero Fund created in memory of Julianna LaMonica to honor her joyful spirit and strength even in the midst of her battle with cancer. Her story continues to inspire so many to support the cause.