Defining minimal criteria for peer-reviewed reporting of mesenchymal stromal cell clinical trials for autoimmune diseases.

BACKGROUND: Mesenchymal stromal cells (MSCs) have been employed in clinical trials for their immunomodulatory capabilities, especially in the treatment of autoimmune disorders such as type 1 diabetes. In January (25-26th) 2024, the International Society of Cell and Gene Therapy (ISCT) held a two-day workshop entitled "Cell Therapies for Autoimmune Diseases: MSCs from Biology to Clinical Application" in Paris, France. METHODS: Key opinion leaders in MSC biology, clinical and regulatory backgrounds came together with European, Asian and South American delegates to discuss the need for standardisation in the design, conduct and reporting of MSC clinical trials for autoimmune diseases. RESULTS: The authors of this paper emphasise the need to comply with standard guidelines for protocols and reporting of trials, working towards greater transparency and reproducibility of research. Standardisation in reporting of clinical trials using MSCs for autoimmune disorders is essential so that the reader can interpret data correctly and ensure that meta-analyses are generated from comparable datasets, providing meaningful knowledge and guidelines for those working in the field. CONCLUSION: In this article the key considerations for the effective design and execution of a clinical trial using MSCs for autoimmune disorders, including product characterisation and key manufacturing parameters are discussed. Recommendations are made with respect to minimal criteria for data reporting, so as to move forward the development of MSC therapy and see a successful transition of these drug products to market, thereby addressing a significant unmet clinical need in autoimmunity.

Duke Scholars

Author Joanne Kurtzberg Pediatrics, Transplant and Cellular Therapy