A Phase I Trial for Recurrent Pediatric Brain Cancers Using a Repurposed Generic Drug

Completed Research, Dr. Riggins, Drug, Johns Hopkins Medicine, Mid-Atlantic, Oncology, Pediatric, Rare Disease

Principal Investigator: Dr. Gregory Riggins

Disease: Medulloblastoma, Glioblastoma

Research Description: Medulloblastoma is the most common malignant brain tumor in children, and brain cancers are the second leading cause of pediatric cancer-related deaths. Dr. Riggins’s recent studies have shown the antiparasitic drug mebendazole to be effective in animal models of aggressive brain tumors, including advanced gliomas and medulloblastomas. Because mebendazole has a history of safe use in patients, it has a relatively short pathway to new clinical trials. Dr. Riggins and his team have improved the formulation and anticancer performance of oral mebendazole, and this Phase I trial will examine dosage levels in recurrent pediatric brain cancers resistant to therapy. They hope to demonstrate not only safety, but to eventually provide the evidence and rationale for future Phase II studies.

Funding Partners: Anonymous

CWR funding role: Participating funder

Completed: 2019