Clinical trial landscape analysis of stem cell therapy for autoimmune rheumatic diseases

Autoimmune rheumatic diseases (ARDs), including rheumatoid arthritis and osteoarthritis, are debilitating conditions with limited curative options. Stem cell therapy offers promising regenerative and immunomodulatory benefits.

We analyzed global clinical trial data from the Informa Trialtrove database up to 31 March 2025, identifying 449 relevant trials on stem cell therapy for ARDs. Trials were assessed by phase, disease type, stem cell source, and geographic distribution.

Most trials employed mesenchymal stem cells (75.8%), with osteoarthritis (46.7%) and rheumatoid arthritis (12.6%) being the most targeted conditions. China led trial activity (26.7%), driven by supportive policies and funding. While 58.8% of trials were completed, 13.1% were terminated, often due to recruitment or funding limitations. Innovations such as CAR-MSCs, iPSC-derived therapies, and exosome-based strategies are expanding the therapeutic frontier.

Stem cell therapy is rapidly advancing in ARDs, supported by growing clinical evidence and technological innovations. However, standardized, multicenter phase III trials are essential to confirm long-term efficacy and safety. This evolving landscape holds potential to transform AIRD management.