Establishment of an Expert Center for Rare Complement Mediated Diseases

Project of $749 223 over 2 years and 2 months

• Supported by CQDM through:
  Ministère de l’Économie, de l’Innovation et de l’Énergie du Québec (MEIE)
• And by co-funding partners:
  – Alexion Pharma Canada Corp.
  – Fondation CHU Sainte-Justine
  – Fondation canadienne du rein
  – CQDM

Challenge: Pathologies associated with the complement system are often rare diseases that significantly diminish the quality of life for affected patients. In the case of thrombotic microangiopathies, the mortality rate can be as high as 20%. These conditions are frequently challenging to diagnose. Currently, there are few biomarkers available to identify abnormalities in the complement cascade, and samples from Quebec patients often need to be analyzed abroad. This results in delays in diagnosis and the initiation of targeted treatments, as well as substantial additional costs. To address these challenges, a provincial structure that combines research and development, clinical expertise, and complement biology is essential. Such a structure will enhance the management and treatment of these rare complement-mediated diseases by improving diagnostic tools and therapeutic approaches.

Solution: The multidisciplinary team, led by Dr. Alexandra Cambier, in collaboration with Alexion Pharma Canada Corp. will create the first Canadian Expert Center for Rare Complement Mediated Diseases at the Sainte-Justine UHC. The team will develop a panel of robust biomarkers for real-time assessment of complement activation to improve diagnosis and monitoring of these diseases. The team will also centralize requests for clinical advice and create and run a website on these rare diseases, accessible to physicists and families. Finally, this collaborative project will integrate clinical and laboratory test development, as well as associated research and innovation activities.

Expected Achievements/Impact: This collaborative initiative, uniting clinicians and scientists with expertise in complement and complement-mediated diseases, aims to establish Canada’s first Expert Center for rare complement-mediated diseases. The creation of this center will provide patients in Quebec and across Canada with privileged access to advanced diagnostics and specialized medical follow-up, significantly reducing the costs and delays associated with analyses previously conducted abroad. Additionally, Sainte-Justine UHC will further solidify its pivotal role in the field of rare diseases, positioning Quebec as a leader in research and development for complement-mediated diseases and advancing knowledge in precision medicine.