CARELeuko Symposium 2024

The inaugural CARELeuko Symposium was held on September 25, 2024, at the RI-MUHC as a hybrid event. The symposium convened clinicians, scientists, students, patient advocacy organizations, and patient representatives from the RI-MUHC, Quebec, Canada, and USA to discuss the current state of leukodystrophy research and present new ideas and opportunities for collaboration across the RI-MUHC and more broadly.

 

Thirteen scientific talks were presented, covering diverse topics from iPSC technology to murine models, non-human primate research, RNA and gene therapies, natural history and disease burden studies, and clinical trials for rare diseases.

​​

Introduction to Leukodystrophies, the CARELeuko Network and Knowledge Gaps in the Field - Dr. Geneviève Bernard

Some of the Lessons Learned and Gaps in the Study of Leukoencephalopathies in Quebec in the Last Two Decades - Dr. Bernard Brais​

iPSC Technology as a Tool to Study Leukodystrophy Pathophysiology - Alexandra Chapleau

Leukodystrophy Murine Models: Past, Present and Future - Dr. Sabrina Alam

Non-human Primates in ASD Research & Perspective for Leukodystrophies - Dr. Yang Zhou

Therapy Development for Neurodegenerative Diseases - Dr. Thomas M. Durcan

RNA Therapeutics and their Implications for Leukodystrophies - Samuel Gauthier

Gene Therapy and its Implications for Leukodystrophies - Dr. Carl Ernst

Adult-onset Leukodystrophies and Transition of Care to Adult Neurology - Dr. Roberta La Piana

Natural History Studies for Clinical Trial Readiness - Dr. Felipe Villa

Leukodystrophy Disease Burden Research: Current Methodological Approaches and Practical Implications - Adam Le and Laura Lentini

Clinical Trials for Rare Diseases - Dr. John Mitchell

RARE.Qc: The network to advance research on rare diseases in Quebec - Dr. Donald C. Vinh

​

These presentations fostered engaging and important discourse around the current landscape of leukodystrophy research and the potential application of expertise and advanced technologies available at the RI-MUHC to the study of leukodystrophies. The scientific talks were complemented by the poignant perspectives of two patient representatives, who shared their unique experiences and challenges while navigating life having cared/caring for an affected family member.

 

The event culminated in a roundtable discussion to identify critical gaps in leukodystrophy research and clinical care across Quebec and Canada and how our RI-MUHC based network will address those. These discussions informed the development of the inaugural CARELeuko manuscript, which outlines the network’s goals and strategies to address these gaps and advance research and clinical care for leukodystrophies in Canada.