A recent study found that metformin enhanced the recovery of neural precursor cells (NPCs) with sex-dependent effects on neuron growth and cognition in rodents. The study also found that continuing into a Phase 3 clinical trial on humans is safe, and that metformin was associated with better performance than placebo in a subset of humans on working and declarative memory tests.
The results of the University of Toronto and the Hospital for Sick Children (SickKids) study mean that the diabetes medication may be able to repair brain injuries one day.
“No one’s actually shown before that you can take a drug where there’s a known mechanism on endogenous stem cells and demonstrate that it’s even possible to induce brain growth and positive recovery,” said SickKids’ and U of T’s Donald Mabbot, co-author of the study. Mabbot said metformin could be game-changing regarding treatment of childhood brain injuries.
“We’re really moving from a model that says ‘let’s help children manage and compensate for their injury,’ to ‘let’s actually treat the injury itself in an active way by harnessing the brain’s own capacity for repair,’” he said.
Metformin was only sufficient for rescuing neuron growth and behavior in female rodents, but not male rodents.
The study’s human participants were survivors of pediatric brain tumors who had been given cranial radiation.
“This study is so novel compared to most studies because it looked at both animal models and human participants. And we found these really consistent and interesting effects in terms of memory and brain recovery,” said study co-author Cindi Morshead of U of T. “This pre-clinical work in animal models will inform the design of the new study, currently in place, to help the brain recover from injury.”
“I am so excited by this paper since it describes a potential endogenous stem cell-based therapy for brain disorders that are currently untreatable,” said SickKids’ and U of T’s Freda Miller. “And, just as importantly, the metformin story provides a classic example of why we need to support basic research, and why working in collaborative teams is essential.”
The study provides evidence that the stimulation of resident stem cells is a realistic approach for repairing tissues in situations where regeneration doesn’t occur easily.
Several medications containing metformin have been recalled due to contamination with a probable carcinogen, and lawsuits are already being filed on behalf of metformin consumers regarding the contamination.