Grant: Project Grant

Systemic sclerosis (SSc) is an autoimmune disease characterized by widespread scarring and immune system dysfunction. In its more aggressive forms, 30 to 50% of patients can face a significant risk of death shortly after diagnosis. This disease can affect several parts of the body including the skin, lung, heart, joints, nerves, blood vessels and gut. For many patients, their disease does not respond to conventional treatments, resulting in long-term organ damage, decreased quality of life and significant impacts on daily function. As such, there is an important unmet need for advanced therapies in the management of SSc. Recent clinical trials have shown promising results for the use of CAR (chimeric antigen receptor)-based therapies for autoimmune diseases by targeting an immune cell marker called CD19. CAR-based therapies involve modifying a patient's own immune cells to target and fight disease. By targeting and destroying the immune cells implicated in SSc, we can reduce the inflammation and disease activity in these patients. Additionally, there is the potential for this treatment to reset the patient's immune system, ultimately eliminating the need for long-term immunosuppressive treatments. Our multidisciplinary team brings together experts from different fields to conduct a clinical trial of a CAR-based therapy for SSc. The primary goal is to determine the safety and feasibility of this treatment in SSc to help design a larger randomized trial comparing CAR-T therapy to standard of care options. Our team has extensive experience with clinical trials, CAR-based therapies and the management of SSc which uniquely positions us to lead this type of study. In addition, we are focused on advancing a Canadian-made CAR-T product, not only bringing us to the forefront of this innovation but reflecting our dedication to ultimately offering Canadians suffering with autoimmune disease with limited treatment options, access to these advanced cellular therapies.