The CHU Sainte-Justine and the CQDM are proud to announce the creation of the Centre d'expertise sur les maladies rares médiées par le complément (here after the Centre d'expertise), a translational research initiative aiming to revolutionize comprehension, diagnosis and treatment of rare diseases. This project, announced on April 30, boasts a first investment of $789,439 provided by Alexion Canada, the Kidney Foundation of Canada (KFOC), the CHU Sainte-Justine Foundation, and a $141,486 grant from the CQDM supported by the Government of Québec.
From left to right : Jesse Paterson (CQDM), Anne-Marie Alarco, Sandrine Thouzeau-Benghezal, Arnaud Bonnefoy, Dr. Jacques Michaud, Dr. Alexandra Cambier, Dr. Anne-Laure Lapeyraque (all from the CHU Sainte-Justine), Karen Heim (Alexion), Elizabeth Myles (KFOC). Photo credit: CHU Sainte-Justine (Véronique Lavoie)
Complement-mediated diseases are rare diseases that present a number of health challenges in Canada, diagnosis is often a long and complicated process, and treatment options are limited. Tackling these challenges is essential to improving the quality of life of patients with these diagnoses.
Pulling on CHU Sainte-Justine's internationally recognized expertise in the fields of precision medicine and complement-mediated rare diseases and Alexion Canada's leadership in the field of rare diseases, the Centre d'expertise envisions in the coming years:
- Developing and producing more effective diagnostic tests and continuing care for complement-mediated diseases
- Creating an integrated biobank and database
- Implementing tools to standardize disease management (protocols and brochures), as well as initiatives to raise awareness and educate to better equip and support patients and their families
- Creating a simple, centralized process to request technically advanced, cutting-edge tests thanks to the unique working relationship between research and the clinic
- Building a research program dedicated to furthering our understanding and knowledge of complement-mediated diseases, aiming to develop precision diagnostic and therapeutic protocols
- Using activities to raise awareness and training on complement-mediated diseases, both for care teams and patients
- Participating in international partnerships, promoting collaboration and knowledge-sharing, notably through conventions and scientific conferences
The Centre d'expertise is the first of its kind in Canada and could ultimately provide unparalleled access to the tools and discoveries made regarding complement-mediated rare diseases for many care teams and patients. Diagnostic tests could be run in Québec, rather than outside of the country where a large majority are currently done, therefore significantly reducing costs for the health care system and standardizing approaches to care for these diseases. The development of precise and non-invasive diagnostic and continuing care tools could significantly improve the quality of life for patients and their families.
This project is perfectly aligned with the objectives of the 2023-2027 Plan d'action québécois sur les maladies rares, by the ministère de la Santé et des Services sociaux which aims to improve the diagnostic process and care practises of people with these conditions.
"This approach promises to bolster rare disease research and treatment, paving the way for new discoveries and innovation," says Dr. Alexandra Cambier the project's leader, clinician-scientist and manager of the translational research laboratory at the Centre de recherche Azrieli du CHU Sainte-Justine. Dr. Cambier is joined by her partners Dr. Anne-Laure Lapeyraque, rare diseases and precision medicine expert, and Arnaud Bonnefoy, hemostasis and complement disease specialist and scientific development manager in the hemostasis clinical laboratory. Thanks to their combined expertise and their active participation in panels with international experts, the Centre d'expertise disposes of a multidisciplinary team of health care professionals, working simultaneously on research and in the clinic. This infrastructure will support patient care and patients diagnosed with rare diseases, all while contributing to education and awareness of these conditions. Furthermore, it will allow for better understanding, control and reference of laboratory analyses within the health care system, thanks to straightforward interfacing with CHU Sainte-Justine's public laboratories (Optilab).
What are complement-mediated diseases?
Complement-mediated diseases are conditions in which the immune system, specifically the complement proteins, mistakenly attack the body's healthy cells. Normally, these proteins help the body destroy bacteria and viruses, but when they become overactive or dysfunctional they can cause certain diseases. These diseases then can cause inflammation or organ damage, requiring treatments to regulate complement activity and protect healthy cells.
Quotes
"We are proud to be a founding partner and to play a unifying and structuring role in this collaborative effort aimed at addressing the challenges faced by people with rare diseases in Canada. By leveraging expertise in both clinical care and research, this strategic partnership will allow us to investigate new indications within the complement disease family and develop personalized therapeutic modalities."
- Dr Jacques Michaud, Research Director, Centre de recherche Azrieli du CHU Sainte-Justine
"Our government is proud to be supporting research and innovation to make Québec a leader in personalized medicine. This new centre of expertise will speed up access to precise diagnoses made in Québec and significantly improve the quality of life of patients suffering from rare complement-mediated diseases."
- Christopher Skeete, Minister for the Economy, Minister Responsible for the Fight Against Racism, and Minister Responsible for the Laval Region
"By supporting this major project, our government recognizes the specific needs of people with rare diseases and their families, and the necessity to address them in the best possible way, through access to diagnostics, healthcare, and cutting-edge treatments. Québec is establishing itself as a leader in rare disease care, notably thanks to the Politique québécoise pour les maladies rares and the Québec Life Sciences Strategy, which are unique to Québec. We are committed to making a lasting difference by supporting research in this field, in order to offer innovative treatments to patients and to significantly improve their quality of life."
- Christian Dubé, Health Minister
"For many complement-mediated diseases, unique and complex laboratory tests are sent outside of Canada, resulting in even longer wait times for patients to receive an accurate diagnosis and appropriate treatment. The Centre d'expertise aims to bring together under one roof expertise and innovative complement-related tests to accelerate diagnosis and improve access to care. It also seeks to strengthen our collective commitment to building a more resilient and equitable healthcare system, thereby allowing Canadians to access the innovations they need."
- Karen Heim, VP & General Manager, Alexion Canada.
"This project marks a major advancement in the diagnosis and monitoring of complement-mediated diseases. By developing local expertise, we will reduce our dependence on foreign laboratories, which will not only accelerate access to diagnostics for patients but also generate direct economic benefits for Québec. This Centre d'expertise will eventually help strengthen our autonomy, stimulate innovation, and position Québec as a strong player in this cutting-edge field, thereby creating new opportunities for the health and life sciences ecosystem."
- Jesse Paterson, Senior Business Development Director, CQDM.
"The CHU Sainte-Justine Foundation takes great pride in supporting the launch of this groundbreaking center, which harnesses the unique expertise and infrastructure of the CHU Sainte-Justine while rallying partners around a mission we wholeheartedly endorse. Growing beyond what we once thought possible is essential for the future of all children with rare diseases, and the Centre d'expertise sur les maladies rares médiées par le complément marks a significant step in achieving this vision."
- Delphine Brodeur, President and Chief Executive Officer, CHU Sainte-Justine Foundation
"We are proud to partner in this innovative and collaborative effort to improve care and research in Complement-mediated diseases in Canada. These diseases can cause serious damage to the kidneys, making it crucial to diagnosis and treat these diseases quickly and accurately. We are excited to support the Centre d'expertise and its projected goals to improve diagnosis, treatment, and care for patients with rare Complement-mediated diseases that affect kidney function."
- Elizabeth Myles, National Executive Director, The Kidney Foundation of Canada
For more information
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Montréal Québec
Canada H3T 1C5
www.chu-sainte-justine.org
