BCC016: Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Medulloblastoma is the most common pediatric malignant brain tumor. While advances in therapy have resulted in improved outcomes for standard risk medulloblastoma, outcomes in high risk medulloblastoma, however, continue to be inferior. In recent years, four distinct molecular subgroups of medulloblastoma have been identified, yet there is a paucity of clinical trials that incorporate molecular risk stratification to outline treatment strategies. Eflornithine or DFMO is an irreversible inhibitor of ornithine decarboxylase, the rate limiting step for polyamine synthesis. The MYC and Hedgehog pathways, both integral in medulloblastoma, have been associated with enhancement of polyamine biosynthesis. We hereby propose a phase II open label, non- randomized, multi-institutional study of DFMO as continuation therapy for molecular high risk/very high risk and relapsed/refractory medulloblastoma.