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

Systemic autoimmune diseases (sAIDs) affect 1 in 10 people and represent a leading cause of death in women under the age of 65 years. Despite the emergence of targeted-biologic therapies, mortality rates for sAIDs have not improved. Emerging cellular therapies represent a promising therapeutic strategy for patients living with sAIDs given their potential to induce sustained remission and limit long-term exposure to glucocorticoids. Encouraging outcomes from a case series of patients with systemic lupus erythematosus, idiopathic inflammatory myopathy, and systemic sclerosis (SSc) have ignited clinical development in this therapeutic area. However, numerous questions remain regarding cellular therapy clinical trial design, ranging from the optimal time to intervene with cellular therapies during a sAID to the appropriate duration of the washout period, conditioning regimen, and management of serious treatment-related adverse events in these patients. Building the infrastructure and multidisciplinary teams necessary to conduct cellular therapy trials for sAIDs also represents a major challenge. To address these critical issues, the National Cancer Institute's Center for Cancer Research and the National Institute of Arthritis and Musculoskeletal and Skin Diseases convened a workshop in which stakeholders from diverse areas addressed strategies to bridge the gap between preclinical and clinical research, foster collaboration between rheumatologists and hematologists-oncologists, and develop optimal treatment protocols grounded in solid science. This manuscript summarizes the main concepts put forward and makes practical recommendations for advancing the development of cellular therapy for sAIDs.