MBI Researchers to Test Novel Treatments for Medulloblastoma

University of Florida researchers have received approval from the Food and Drug Administration to test two new experimental therapies in first-ever human clinical trials for a subtype of medulloblastoma, the most common pediatric malignant brain tumor.

The two Phase I trials to treat Group 4 medulloblastoma, expected to begin this fall and next spring, will separately be led by Duane Mitchell, M.D., Ph.D., and Elias Sayour, M.D., Ph.D., investigators with UF’s McKnight Brain Institute and the UF Health Cancer Center.

Mitchell’s trial will test combining two distinct techniques: precision adoptive T-cell therapy and immune checkpoint blockade with PD-1 inhibitors. Adoptive T-cell therapy, developed at UF, has been shown to be safe and feasible for children with medulloblastoma in previous trials, Mitchell said, while PD-1 inhibitors are FDA-approved to treat some other types of cancer. The new trial will involve using mRNA expressed from a patient’s own Group 4 medulloblastoma tumor to stimulate T-cell responses directed at the cancer.

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