In medicine, it’s a long path from scientific concept to promising treatment.

As a developmental neurobiologist at The Hospital for Sick Children Research Institute and Professor at the University of Toronto, the issue that Dr. Freda Miller and collaborators have been trying to solve for years is how to “turn on” stem cells that are resident in our brains, coax them into becoming neurons, and stimulate them to promote brain repair.

Dr. Miller’s work is based on the novel concept of using a common medication called metformin, which had been previously tested on liver cells, to “turn on” stem cells that are resident in the brain. In 2012, Dr. Miller’s team published a breakthrough: they applied metformin to brain stem cells in a dish and the cells made neurons.

Next, in preclinical testing, it was found that “giving adult mice metformin daily was sufficient to enhance the number of new neurons that were made in a part of the brain that’s important for learning and memory,” she says.

Excitingly, Dr. Miller’s scientific concept has now moved into human clinical trials with her colleagues Dr. Don Mabbott and Dr. Ann Yeh – one involving children with acquired brain injury due to treatment for brain tumours and one involving children with multiple sclerosis. “Now this discovery has a life of its own. If results continue to be positive, our discovery will eventually be used in adults with multiple sclerosis and other kinds of brain injury. This has been a success story, and I am grateful to Brain Canada for not only funding us from the beginning, but also advancing the science and partnerships at each step,” she says.