Ontarians living with debilitating degenerative brain disease or injury may one day have access to powerful cell- and gene-based treatments, thanks to two Toronto-based research teams and support from a unique regenerative medicine commercialization initiative.
In 2021, the Ontario Institute for Regenerative Medicine (OIRM) and C3i Center Inc. (C3i) launched the Kick-Start Innovation Investment Program to transform promising experimental cell and gene therapies into treatments for serious illnesses. From dozens of submissions, two technologies have been selected to receive $100,000 in targeted funding to complete pivotal experiments that will increase the likelihood of attracting early-stage investment and building valuable intellectual property (IP). Teams will engage directly with venture capital (Versant Ventures), investment banking (Bloom Burton & Co.) and private sector expertise to develop a tactical business plan, access additional funding opportunities and strategic support to bring the technology to market.
“The Kick-Start selection committee was looking for new, cutting-edge ideas with the potential to improve how we treat serious diseases and injuries, with the parallel goal of being well-positioned to ultimately drive company creation in Ontario,” says Tim Smith, Chair, OIRM Board of Directors and CEO of Octane Medical Group in Kingston, Ontario. “We were impressed with the breadth and depth of the submissions we received, and we are very excited about the promise of these two technologies.”
Maryam Faiz, assistant professor in the Department of Surgery at the University of Toronto, is one recipient of Kick-Start support. She’s collaborating with a team of researchers – including Shannon Dunn, scientist at the Keenan Research Centre for Biomedical Research at St. Michael’s Hospital, Scott Yuzwa, assistant professor, Department of Laboratory Medicine and Pathobiology at the University of Toronto, and Faiz’s graduate student, Justine Bajohr, MSc. – who are investigating a novel approach to reprogramming support cells in the brain to become cells that repair and regenerate lost myelin in the brain. Myelin is an insulating layer around nerves in the brain and spinal cord that helps send electrical signals between nerve cells; when it is damaged in conditions like multiple sclerosis or through acute brain injury, significant cognitive and physical impairments can result.
“Cell regeneration strategies in the central nervous system (CNS) have focused on transplanting stem cells or mobilizing cells resident in the brain, but these have largely failed,” Faiz says. “A major leap in our technology is the way we are using a type of gene therapy to turn astrocytes already in the brain into oligodendrocytes. We’re excited about Kick-Start support – it’s a perfect fit for the stem cell ecosystem in Ontario, with an eye to commercial technology and building companies. I’m at an earlier stage in my career, but I really haven’t seen anything like it.”
Repairing CNS damage caused by stroke or neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) is the focus of the second team to receive Kick-Start support. Led by Sunnybrook Research Institute Senior Scientist Carol Schuurmans (pictured at left, below), who holds the Dixon Family Chair in Ophthalmology Research, this multi-disciplinary team of investigators* has already demonstrated in pre-clinical studies a ‘designer gene’ therapy/cellular conversion approach to replacing neurons in the brain lost to neurodegenerative diseases and stroke, significantly improving CNS function. Cindi Morshead, chair of the Division of Anatomy, Department of Surgery at the University of Toronto and an expert in stroke modelling (centre photo below), and JoAnne McLaurin, senior scientist at Sunnybrook Health Sciences Centre and a Tier 1 Canada Research Chair in Alzheimer’s Disease Drug Development (pictured at right, below), round out the team.
“What we’re envisioning is a gene treatment that will be applicable to a large number of neurodegenerative diseases by targeting different regions of the brain as needed,” Schuurmans says. “We have demonstrated that we can take endogenous glial cells in the brain and convert them to new neurons using transcription factors that increase the capacity to promote neuronal conversion, with high conversion rates. We have a provisional patent for this work, and so Kick-Start will be used to build additional value in our intellectual property portfolio, advancing technology readiness by exploring long-term efficacy and further de-risking the technology.”
Both technologies boast significant potential upside for the hundreds of thousands of Canadians who suffer from CNS injuries or disease, as well as the Canadian economy, according to C3i CEO Louisa Petropoulos.
“Kick-Start was designed as a bridge to help innovators stick handle experimental technology through the complicated process of commercialization, and increase their chances of success,” she says. “The goal is to get Ontario-made solutions into the market and the clinic, and keep them in Ontario so the health and economic benefits stay here. I don’t think we could have two stronger candidates to achieve this vision.”
Medicine by Design, which receives funding from the Canada First Research Excellence Fund, has made foundational investments in both teams’ research, notably through a large team project award focused on neural reprogramming. Schuurmans’s team is also being supported by Medicine by Design’s Pivotal Experiment Fund, which invests in research that can be spun-off to an early-stage product concept or venture.