Repurposing Drugs for Recurrent Pediatric Brain Tumors

Principal Investigator: Dr. Sarah Leary
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Disease: Pediatric Brain Cancer

Research Description: Pediatric brain tumors are the most common solid tumor malignancy in children, and continue to be a leading cause of pediatric death from disease.  Standard treatment for pediatric brain tumor patients continues to be the chemotherapy and radiation therapy developed decades ago, which results in significant toxicity. Hundreds of new-generation “targeted” cancer therapies have been developed, with success in adults. Over a dozen of these targeted therapies have undergone safety testing in the pediatric population, but none have been successfully incorporated into pediatric brain tumor treatment. This study is a pilot clinical trial in pediatric patients with recurrent brain tumors. Patients whose cancers express a potential target will be treated with a drug developed to inhibit that specific target. All medications prescribed will be FDA-approved drugs for which a pediatric dose has been established, but without indication in pediatric brain tumors.

Funding Partners: Anonymous

CWR funding role: Primary funder

Completed: 2018