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Sarah Leary headshotPrincipal Investigator: Dr. Sarah Leary

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.

CWR funding role: Primary funder

Start date: December 21, 2012

Most recent report: Seven patients have been enrolled in the clinical study, and enrollment remains open to new patients. Tissue samples have been collected, and two patients are receiving treatment suggested based on analysis of their samples.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Convert this pilot trial into a nationwide clinical trial for the entire Children's Oncology Group

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