Neurological diseases and disorders

Neurological diseases and disorders

Neurological diseases and disorders

Neurological Diseases and Disorders

Decorative shape

Neurological diseases and disorders affect the brain, the spinal cord, and nerves that form an intricate and complex network. These conditions are often difficult to treat, not only because of the risks associated with treatments and surgeries, but in some cases because the underlying causes are poorly understood despite advances in medical research. Approximately 10% of Canadians are affected by neurological disorders, making them among the leading causes of disability in the country. By 2031, the number of Canadians with neurological conditions is expected to increase, with the number of people with Alzheimer’s disease and other dementias, Parkinson’s disease, and traumatic brain injury projected to double.

Stem Cell Network researchers are working to tackle these challenges by studying neural biology and neural stem cells to better understand and treat neurological conditions such as multiple sclerosis (MS), Parkinson’s disease, epilepsy, Alzheimer’s disease, Rett syndrome, stroke, and spinal cord injury. Researchers are developing regenerative therapies that target the brain’s own repair mechanisms, creating laboratory models to better understand disease processes, and advancing treatments from the lab toward clinical trials. From 2016-2028, SCN has invested over $4.4 million in innovative neural research and clinical trials.

Dr. Anastassia Voronova, University of Alberta, Alberta

Cycle 5, Round 1: Early Career Researcher Awardee Unlocking the Brain’s Potential to Repair Itself in Progressive Multiple Sclerosis

Multiple sclerosis (MS) is a chronic neurological disease that affects nearly 100,000 Canadians, disrupting communication between the brain and body. While current treatments can help manage the relapsing-remitting form of MS, they offer little relief for patients with progressive MS—a stage marked by continuous neurological decline and limited treatment options.

A key to treating progressive MS lies in remyelination—the regeneration of the protective myelin coating that insulates nerve fibers and enables normal brain and spinal cord function. In people with MS, this repair process breaks down, leaving nerves vulnerable to damage and dysfunction.

Dr. Anastassia Voronova is exploring a promising new approach to jumpstart the body’s own repair mechanisms. Her research focuses on fractalkine (CX3CL1), a naturally occurring brain molecule that activates a receptor (CX3CR1) found on oligodendrocyte precursor cells—the very cells that can generate new myelin-producing cells. Her team is testing whether drug candidates that mimic fractalkine can safely and effectively stimulate remyelination in the brain.

If successful, this research could pave the way for the first regenerative treatments for progressive MS—offering hope to patients who currently have no therapeutic options.

“Our aim is to harness the brain’s own potential to repair damaged myelin. By targeting a natural regenerative pathway, we hope to develop treatments that restore function and quality of life for people living with progressive MS.”

Dr. Anthony Flamier, Centre hospitalier universitaire Sainte-Justine, Quebec

Cycle 5, Round 1: Early Career Researcher Awardee (jointly funded with the Azrieli Foundation) Restoring Brain Function in Rett Syndrome Through Regenerative Medicine

Rett syndrome (RTT) is a rare and devastating neurological disorder caused by mutations in the MECP2 gene. It primarily affects young girls, leading to profound cognitive and physical disabilities. Despite decades of research, there are still no effective treatments to address the root causes of this condition.

Dr. Anthony Flamier is pursuing a bold new direction by targeting an underexplored feature of RTT: dysfunction in primary cilia—tiny cellular structures essential for brain development and neuronal communication. His team is using patient-derived stem cells to create neurons and brain organoids that model RTT in the lab. By studying how primary cilia are altered in these models—and validating the findings with post-mortem brain tissue—they hope to uncover critical insights into disease mechanisms.

At the same time, the project includes a drug screening platform that will test FDA-approved compounds for their ability to restore cilia function and normalize neuronal activity. This dual approach could fast-track the discovery of therapies that improve brain function in RTT patients.

If successful, this research could open a path to the first regenerative treatments for Rett syndrome—delivering hope to families and advancing Canada’s leadership in neuroscience and precision medicine.

“We’re using cutting-edge tools in regenerative medicine to better understand and treat Rett syndrome. Our goal is to restore function where it’s been lost—and give patients and families a reason to hope.”

Dr. Molly Shoichet, University of Toronto, Ontario

Cycle 5, Round 1: Accelerating Clinical Translation Award Targeted Enzyme Therapy to Promote Brain Repair After Stroke

Stroke is a leading cause of disability, and beyond rehabilitation, there are very few treatment options to support brain repair. After a stroke, the brain forms scar tissue that protects it from further damage—but this same scar also blocks the brain’s ability to heal.

Dr. Molly Shoichet’s team has developed a new enzyme therapy that breaks down this scar tissue in a controlled and localized way, opening the door for brain regeneration when combined with rehabilitation. The team has also created a method to deliver the enzyme directly to the brain for a sustained period.

This project will advance manufacturing of the therapy under GMP (Good Manufacturing Practices) standards and test it in stroke models. At the same time, the team will begin regulatory planning with Health Canada, aiming to move toward a first-in-human clinical trial.

By combining expertise in bioengineering, neuroscience, and neurosurgery, and working with international collaborators, this project could lead to a transformative treatment for stroke recovery. In Canada alone, stroke costs the healthcare system $10.9 billion each year—making even modest improvements highly impactful.

“We’re developing a therapy that clears the way for the brain to heal itself after stroke—unlocking new potential for recovery.”