How scientists and people with lived experience are working together in the fight against brain diseases

New Frontiers in Research Fund | Published:

Researcher Carol Schuurmans in her lab
Photo: Sunnybrook Health Sciences Centre

One in 10 Canadians are affected by neurological disorders, and the number is expected to increase as the population ages. In a race against time, one research team is developing a “pipeline” approach to treating brain disease, by going from lived experience to the lab, to the clinic, and back.

Carol Schuurmans, a senior scientist and Dixon Family Chair in Ophthalmology Research at the Sunnybrook Research Institute, and a professor of biochemistry at the University of Toronto, is the nominated principal investigator on this game-changing research project.

The project is one of six large-scale, multiyear projects recently selected to receive up to $24 million in Transformation stream funding through the Government of Canada’s New Frontiers in Research Fund (NFRF). The Fund supports transformational projects that bring together researchers from a wide range of institutions and expertise.

People with lived experience—they’re experts in the challenges they face, and the opportunities, as well.

The 24-member interdisciplinary research team includes scientists from across Canada, as well as from Portugal, Germany and Spain. Together, the team aims to develop novel gene therapies to turn common glial cells into healthy neurons, determine the best way to deliver these gene therapies into the brain, then test the effectiveness of the approach for Alzheimer’s disease, stroke and epilepsy.

The researchers are trying something new to them when it comes to engagement: the scientists will be actively advised by people living with brain disease, and their caregivers.

“People with lived experience—they’re experts in the challenges they face, and the opportunities, as well,” says Carrie McAiney, an associate professor at the University of Waterloo, and Schlegel Research Chair in Dementia. McAiney will co-lead this part of the project.

“It’s by working together that we can better understand the experiences of people living with neurological conditions, and the acceptability of potential treatments,” says McAiney.

Portrait of Ravi Menon at the Centre for Functional and Metabolic Mapping

Carol Schuurmans and PhD student Lakshmy Vasan, a member of Schuurmans’ lab team
Photo: Sunnybrook Health Sciences Centre

As Schuurmans explains, the team’s approach to brain repair—converting unhealthy brain cells into new neurons in the brain—is inspired in part by fish, which have a remarkable ability to regenerate. The zebrafish, for example, can regenerate its brain, spinal cord and heart. In humans, this sort of regeneration is limited to the skin and to a few organs, such as the kidney and liver.

“We are reactivating a latent repair pathway that has been lost over evolutionary time,” Schuurmans says.

Previous research in this area has attempted to generate neurons in a variety of different ways. This project distinguishes itself by taking advantage of the latest breakthroughs in science and technology. For example, principal investigator Antonio del Sol, a computational biologist and professor at the University of Luxembourg and CIC bioGUNE (the Center for Cooperative Research in Biosciences) in Spain, will use AI and machine learning to identify which genes should be used in which combinations to convert and reprogram glial cells into precise types of neurons.

The NFRF-funded research team also includes Anna Blakney, an RNA scientist at The University of British Columbia, and cofounder of VaxEquity, a biotech company that is working in partnership with AstraZeneca and creating self-amplifying mRNA for therapeutics and vaccines. Blakney, applying her expertise from working on a self-amplifying RNA vaccine for COVID-19, will be tasked with building RNA technology as a gene delivery tool. A cadre of other researchers on the team will develop packaging and delivery tools, and assess therapeutic efficacy in disease models.

People with lived experience will be involved in three advisory groups on the project: people living with dementia, people living with stroke, and people living with epilepsy. Participants will be recruited from across Canada and participate in virtual group meetings.

The idea is to hear the main concerns of people living with these conditions, understand their risk tolerance when it comes to potential benefits and potential side effects of treatments, and get feedback on how to share the project team’s impacts or findings with the larger public.

[T]he opportunity to make this better for someone else is why they’re around the table.

“One of the things that’s going to make this work is the receptivity of the researchers. It’s novel for them, but they’re willing to take the leap with us,” McAiney says. “We’ve had a few meetings with some researchers on this team, and the people with lived experience really make them think.”

Schuurmans shared an example: People living with dementia told her a therapy that targets bowel and bladder control would make the disorder much easier to manage—something that hadn’t been top of mind for her until she heard it from them.

Over the next six years, hearing directly from people with lived experience will continue to both inspire and push the researchers.

“It’s moving my lab in a new and exciting direction that takes advantage of all our knowledge and technology,” Schuurmans says.

“You hear the excitement they feel about the potential impacts,” McAiney says. “They’re okay with not necessarily benefiting from this, but the opportunity to make this better for someone else is why they’re around the table.”

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