One of the major limitations in finding treatments for brain diseases such as Parkinson’s and Alzheimer’s has been the difficulty acquiring appropriate tissue samples from patients.
“People will give you a bit of their blood or their skin, but they won’t give you a piece of their brain,” says Dr. Edward Fon, Scientific Director of the Early Drug Discovery Unit (EDDU) at The Neuro (Montreal Neurological Institute-Hospital).
But a promising technology developed in the past decade is changing that. The method involves converting adult stem cells into what’s called an “inducible pluripotent stem cell.” These new cells can then be reprogrammed into desired cell types.
“Now, just from a blood sample, we can reprogram those blood cells into stem cells, and then make those stem cells grow into neurons, other types of brain cells, or even three-dimensional brain organoids, that have electrical activity and express the same kinds of genes that cells within a real brain do,” says Dr. Fon.
With these stem cells, researchers have the means to study underlying biological mechanisms and to test new therapies, with the hope of finding more effective treatments for diseases such as Parkinson’s, ALS, and more.
Dr. Fon was one of a team of collaborators, led by Dr. Jack Puymirat (Université Laval), that received a 2014 Brain Canada Platform Support Grant (PSG) to develop the Human inducible pluripotent stem cell (hiPSC) platform. The $1.5 million grant helped make this transformative technology accessible to researchers across Canada.
Brain Canada launched its PSG program after recognizing the essential need for collaborative platforms. By bringing together cutting-edge equipment, technology, and services and making them widely available, these platforms accelerate research outcomes beyond what a single researcher might achieve with just the resources in their own lab.
"Brain Canada’s initial investment and support of the platform was absolutely instrumental in getting things off the ground. Making these stem cells takes a huge amount of resources. But this platform gives us significant economies of scale," says Dr. Edward Fon.
Since receiving the 2014 grant, the hiPSC platform has evolved from a local project at the Université Laval into the Early Drug Discovery Unit (EDDU), an open drug discovery platform at the Montreal Neurological Institute (MNI). Dr. Fon’s colleague, Dr. Thomas Durcan, Associate Director of the EDDU, runs the
day-to-day operations of the platform, which now serves researchers across Canada and has stimulated national and international collaborations with more than 40 team members, 100 users trained, 50 academic collaborators, and 8 industry partners.
Thanks to the MNI’s open science model, everything developed at the EDDU is made fully available to academia and industry, dramatically accelerating medical research and the development of new therapies for devastating neurodegenerative disorders.
Not only is Dr. Fon involved with the platform on an administrative level, he uses it regularly in his work researching Parkinson’s disease.
“At some point, you need to do the work in human models of disease,” he says. “So as a user, the platform has been tremendously beneficial to my research.”