Cellular Therapy for Systemic Autoimmune Diseases: New Opportunities for Future Research and Interdisciplinary...

Area of Science:

• Immunology
• Rheumatology
• Cellular Therapy

Background:

• Systemic autoimmune diseases (sAIDs) affect 10% of the population and are a major cause of death in women under 65.
• Current targeted-biologic therapies have not improved sAID mortality rates.
• Cellular therapies offer a promising strategy for sAIDs, potentially inducing remission and reducing glucocorticoid dependence.

Purpose of the Study:

• To address critical issues in cellular therapy clinical trial design for sAIDs.
• To foster collaboration between rheumatologists and hematologists-oncologists.
• To develop evidence-based treatment protocols for cellular therapies in sAIDs.

Main Methods:

• Convened a workshop with diverse stakeholders, including experts from the National Cancer Institute and the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
• Discussed strategies to bridge preclinical and clinical research gaps.
• Identified challenges in infrastructure and multidisciplinary team building for sAID cellular therapy trials.

Main Results:

• Identified key questions in clinical trial design, including optimal intervention timing, washout periods, conditioning regimens, and adverse event management.
• Highlighted the need for robust infrastructure and multidisciplinary teams for sAID cellular therapy trials.
• Summarized concepts and provided practical recommendations for advancing cellular therapy development in sAIDs.

Conclusions:

• Cellular therapies hold significant potential for treating sAIDs.
• Standardized clinical trial protocols and collaborative efforts are essential for successful development.
• Further research and strategic planning are required to optimize cellular therapy delivery for sAIDs patients.