CARELeuko, a Canadian network for research excellence in leukodystrophy
06-01-2025
The Canadian Association for Research Excellence in Leukodystrophy (CARELeuko) is Canada's first national research network dedicated to transforming the lives of leukodystrophy patients and their families. By uniting researchers, clinicians, patient advocates, and industry partners, CARELeuko fosters collaboration to accelerate breakthroughs in the diagnosis, treatment, and care of leukodystrophies. With a shared vision, the network aims to create a brighter future for families affected by these rare, devastating disorders across Canada and beyond.
Leukodystrophies (LDs) are a heterogenous group of hereditary disorders affecting the cerebral white matter. With an incidence of 1 in 4,733, leukodystrophies are under-researched and underfunded, leaving many families without answers or hope. Despite recent advances, critical gaps remain in understanding and addressing LDs. Approximately 20-30% of pediatric and 51-70% of adult patients lack a molecular diagnosis, the underlying mechanisms of many LDs are poorly understood, and there are no effective disease-modifying therapies for most forms. Additionally, gaps in natural history data, patient-reported outcomes, and evidence-based care guidelines limit progress toward better treatment and care.
CARELeuko is determined to address these challenges through three core objectives: identifying and validating new LD-causing genes, advancing therapeutics through cutting-edge research, and improving patient care by studying disease progression, identifying clinical trial endpoints, and understanding disease burden. By establishing a Pan-Canadian Leukodystrophy Network, CARELeuko envisions a future where every patient and family has access to state-of-the-art diagnostics, effective treatments, and comprehensive care, ensuring no family faces this journey alone.
The initiative is led by Dr. Genevieve Bernard, in co-direction with Dr. Roberta La Piana and Dr. Thomas Durcan.
If you'd like more information or if you are interested in joining CARELeuko, please contact adam.le(at)muhc.mcgill.ca
