The Canadian Association for Research Excellence in Leukodystrophy (CARELeuko) is Canada's first national research network dedicated to transforming the future for leukodystrophy patients and their families. By bringing together researchers, clinicians, patient advocates, and industry partners, we aim to foster collaboration and accelerate breakthroughs in diagnosis, treatment, and care for leukodystrophy patients and families across Canada and worldwide.

What are Leukodystrophies?

Leukodystrophies (LDs) are a group of rare, genetic disorders affecting the brain's white matter. Predominantly affecting children, they lead to progressive loss of physical and cognitive abilities, and tragically, early death. With an incidence of 1 in 4,733, leukodystrophies remain under-researched and often underfunded, but through collaboration, we are determined to change this.

Why We Need Your Support

Despite advances, critical gaps in LD research remain, leaving many families without answers or hope for treatment.

• 20-30% of patients lack a molecular diagnosis
• The pathophysiology of many LDs is still poorly understood
• There are no effective disease-modifying therapies for most LDs
• Natural history data and important markers for effective treatments are lacking
• Patient and family-reported outcomes on disease impact is limited
• Evidence-based guidelines for the care of affected patients and families do not exist

CARELeuko is committed to addressing these challenges with three core objectives:

1. Identifying and validating new LD-causing genes
2. Advancing our understanding and developing therapeutics through cutting-edge research
3. Improving patient care by studying disease progression, identifying suitable endpoints for clinical trials, and examining disease burden to improve real-time interventions

Our ultimate vision is to establish a Pan-Canadian Leukodystrophy Network, uniting efforts across the country to ensure every patient and family affected by LDs has access to leading-edge diagnostics, treatments, and care.

How You Can Help

By supporting CARELeuko, you are directly contributing to groundbreaking research aimed at:

• Designing and developing individualized clinical care plans and N=1 therapies using personalized drug screening and gene therapies with patient-derived induced pluripotent stem cells (iPSCs)
• Bringing potential therapeutics to PI-initiated clinical trials with industry partners
• Empowering families with knowledge and better care guidelines

Together, we can make a lasting difference for patients and families with leukodystrophy in Canada and around the world. Join us in this fight to bring hope, treatments, and cures where none currently exist.