Global clinical trials on stem cell therapy for autoimmune diseases: trends and future directions

Front Immunol. 2025 Jul 24:16:1616231. doi: 10.3389/fimmu.2025.1616231. eCollection 2025.

Abstract

Background: Autoimmune diseases, such as Crohn's disease (CD) and systemic lupus erythematosus (SLE), lead to progressive multi-organ damage due to immune dysregulation and chronic inflammation. Current therapies lack efficacy and safety, often failing to sustain remission. Stem cell therapy has emerged as a promising approach for immune modulation and tissue repair. This study analyzes clinical trial trends and challenges of stem cell therapy in autoimmune diseases.

Methods: Clinical trial data (2006-2025) were extracted from Trialtrove. Strict inclusion criteria were applied, restricting the analysis to interventional trials while excluding observational studies, non-autoimmune disease trials, and records with incomplete information. Descriptive statistics were used to analyze trial phases, disease types, geographic distribution, funding sources, therapeutic mechanisms, and stem cell sources, followed by a comparative evaluation of therapeutic efficacy, combination strategies, and safety profiles across autoimmune indications.

Results: Of the 1,511 global trials, 244 were included after screening and cross-referencing. Most trials (83.6%) were in Phase I-II. CD (n=85), SLE (n=36), and scleroderma (n=32) were the most studied. The U.S. and China led in trial numbers. Academic institutions funded 49.2% of trials. Key therapeutic strategies included immune modulation, tissue repair via growth factors, and anti-infection/anti-proliferative effects. Disease-specific variations were noted in cell sources and administration routes.

Conclusion: Stem cell therapy holds substantial promise for autoimmune disease treatment. Future efforts should prioritize technological innovation, international collaboration, and precision medicine to address current challenges and advance clinical translation.

Keywords: Crohn’s disease; autoimmune diseases; clinical trials; mesenchymal stem cells; stem cell therapy.

Publication types

  • Systematic Review

MeSH terms

  • Autoimmune Diseases* / immunology
  • Autoimmune Diseases* / therapy
  • Clinical Trials as Topic
  • Humans
  • Stem Cell Transplantation* / methods
  • Stem Cell Transplantation* / trends
  • Treatment Outcome