Cutting-edge clinical trials in stem cell and regenerative medicine

This is part of a continuing series showcasing some of Ontario’s many clinical trial strengths and assets.

In a trailblazing clinical trial taking place in Canada’s largest city, Dr. Michael Fehlings is testing a therapy that involves injecting human neural stem cells into patients with spinal cord injuries. His aim: to help people with paralysis recover mobility in their limbs.

The study being run by Dr. Fehlings, Head of the Spinal Program at Toronto Western Hospital, University Health Network and Co-Chair of the University of Toronto Spine Program, is one of a number of stem cell clinical trials taking place in Ontario that target a range of diseases from arthritis and septic shock to multiple sclerosis.

Ontario is a centre of excellence in stem cell and regenerative medicine. The province has a history of breakthroughs in the field—starting with the first discovery of stem cells—as well as strong clinical programs, an open, collaborative environment and increasing opportunities for commercialization.

“We have great science, fantastic tertiary-care hospitals, world-beating clinical teams and infrastructure that can support excellent clinical trials,” says Dr. Janet Rossant, President and Scientific Director of the Ontario Institute for Regenerative Medicine (OIRM) and an eminent developmental and stem cell biologist. “This puts Ontario and Canada at the cutting edge of stem cell discoveries that can be used to treat devastating diseases.”

Right now in Ontario, cell therapy studies include both investigator-initiated and industry-sponsored trials involving pharmaceutical, biotech and medical device companies. Dr. Rossant says there are trials using drugs to stimulate the stem cells already within the body to work better, for instance repairing damaged neural tissue in order to improve brain function. Others using “pluripotent” stem cells, which give rise to all of the cells in the body, are “opening exciting new possibilities,” she says. These could be transplanted into patients to treat diseases such as macular degeneration and diabetes.

Dr. Fehlings, the Gerald and Tootsie Halbert Chair in Neural Repair and Regeneration and a Senior Scientist at the McEwen Centre for Regenerative Medicine, says that international companies as well as foundations and NGOs view Ontario as attractive for performing clinical trials in the field of stem cells and regenerative medicine. He is currently leading three such multi-centre trials dealing with acute spinal cord injuries. Two involve using drugs to encourage cell regeneration and a third focuses on stem cells to encourage the repair of the central nervous system.

Specifically, the first drug trial is the Riluzole in Spinal Cord Study (RISCIS), a Phase III trial supported by a Swiss not-for-profit foundation called AOSpine. The study is looking at administering riluzole, an anticonvulsant with neuroprotective properties, in patients immediately following traumatic spinal cord injury in order to preserve viable neurological tissue and prevent secondary injury, which often occurs. The second trial is a Phase IIb/III study sponsored by Vertex, a Boston biotechnology company, which is assessing the use of VX-210 to encourage cell regeneration. Finally, the third stem cell trial is a Phase II study supported by StemCells, Inc. of California. It involves micro-injecting purified human neural stem cells above and below the injury site to repair the damaged spinal cord.

The therapies are intended to improve functional recovery, such as restoring hand movements. This “could have a profound impact on patient independence,” says Dr. Fehlings, a University of Toronto professor of neurosurgery.

Beyond Ontario’s “significant cluster of expertise” to run trials, he notes, its publicly funded health-care system facilitates patient enrolment in clinical research. Canada is a particularly good place for clinical trials, including early-stage trials, with a supportive regulatory environment, producing data that is well-accepted in the United States.

Clinical trial strengths in Ontario range from its large, diverse population to its high-quality hospitals, comments Dr. Michael May, President and CEO of the Centre for Commercialization of Regenerative Medicine, a partner organization of OIRM.

Developing and refining human stem cells for preclinical testing can be done in a lab, but it’s quite another thing to grow them uniformly in the numbers needed for human trials and eventually for approved medical procedures. Dr. May says the ability to commercially manufacture high-quality stem cells is one of Ontario’s critical strengths. Having technologies that are clinically tested, developed and manufactured in the province is building an “ecosystem” of companies and has brought important industry partners such as GE Healthcare here, he says. “We’ve created an environment where the private sector is welcome. We speak their language.”

The new Centre for Advanced Therapeutic Cell Technologies at the MaRS Discovery District, announced by Prime Minister Justin Trudeau in early 2016 with $20-million in federal funding, matched by GE Healthcare with another $20-million, will accelerate the development and adoption of cell manufacturing technologies and position Ontario as a global cell therapy hub, Dr. May says. There is also a plan to create a $150-million regenerative medicine venture capital fund in the province, with financing from public and private sources, focused on encouraging emerging companies to undertake clinical trials here.

“This is among the most prolific clinical trials and commercialization centres in this space in the world,” Dr. May adds. “There’s no other place where you’re going to want to do your clinical trials.”

For more information, visit and

More CTO Stories