Phase III randomized double-blind placebo-controlled trial of metformin for cognitive recovery and white matter growth in paediatric medulloblastoma patients

Because of advances in treatment, survival rates for pediatric brain tumours have improved dramatically in the last 25 years: currently there are at least 30,000 survivors in North America. Unfortunately, the treatment used to cure paediatric brain tumours can result in permanent physical, psychological, and neurological problems. Currently, there are no effective medical therapies to help brain recovery and reduce disability in paediatric cancer. We hope to change this. The drug metformin can promote rescue of cognition and brain growth in animal models by activating stem cells in the brain: we have found that it is safe and feasible to use in children treated for a brain tumor and may improve cognition. We seek to ask if this is indeed true by using a multi-site clinical trial to test the efficacy of metformin treatment for cognitive recovery in children and adolescents treated for medulloblastoma which requires aggressive therapy leaving survivors with significant cognitive impairment. If we find that metformin promotes cognitive improvement and brain growth in pediatric survivors of medulloblastoma, then this may offer a viable therapeutic approach that may significantly improve the quality of life of these vulnerable patients - and provide a model for treatment of late effects in other pediatric cancers.