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Ewing sarcoma (EwS) is a bone and soft tissue tumor that most commonly affects children and young adults (AYA). Patients with relapsed/refractory (R/R) EwS continue to face dismal outcomes. While chemotherapy backbones are effective in patients with R/R EwS, outcomes are insufficient, and there is a need to identify combination strategies with novel agents to improve outcomes. Dr. Ganapathi and colleagues have found that EwS is critically dependent on the menin protein, which cooperates with the fusion protein EWS::FLI1 to drive growth and metastasis. Lab and model studies show that blocking menin with Revumenib (FDA approved for pediatric leukemias) powerfully suppresses tumor invasion and metastasis. Dr. Ganapathi and lab will conduct an early phase clinical trial of Revumenib combined with chemotherapy. This combination will ultimately improve outcomes for patients with R/R EwS.

This grant is generously supported by JC Strong, a St. Baldrick’s Hero Fund, established in honor of Jackson Kalbehnn to celebrate his survivorship. Jackson was 5 years old when he was diagnosed with Ewing sarcoma. After 14 cycles of aggressive chemotherapy, 69 nights in the hospital, and a resection on his left leg, his treatment was complete. Today, Jackson shows no evidence of disease and enjoys an active life filled with all things sports related. Through JC Strong, his family hopes to make meaningful difference for every child and family facing Ewing sarcoma.