Cytotherapy. 2025 Sep 26:S1465-3249(25)00846-1. doi: 10.1016/j.jcyt.2025.09.008. Online ahead of print.
ABSTRACT
BACKGROUND AIMS: Induced pluripotent stem cells (iPSCs) are a promising cell-based therapy given their ability to differentiate into various cell types for therapeutic use. Although clinical trials using these cells have recently emerged, such trials are small, heterogenous in design, and examine a broad range of diseases. A systematic scoping review of published clinical studies and registered trials is needed to understand important aspects of study design and identify barriers to greater clinical adoption.
METHODS: A systematic search of published clinical studies was conducted in MEDLINE, Embase and the Cochrane Central Register of Controlled Trials (1946 to January 29, 2025) and ongoing registered clinical trials in Clinical Trials.Gov and the World Health Organization International Clinical Trials Registry Platform (January 29, 2025). Descriptive statistics were used for data analysis. Meta-analysis was not performed given the small number and size of included studies.
RESULTS: We identified 10 published clinical studies and 22 ongoing registered trials utilizing iPSCs to treat a wide range of diseases, including cardiac conditions, ocular disorders, cancer, graft-versus-host disease, and as a source of platelets for transfusion. Published studies were mostly small (only 2 studies reported on more than 4 patients) and uncontrolled, with a total of 115 patients treated. While iPSC characterization was generally aligned with criteria established by the International Society for Stem Cell Research, considerable variability was observed in terms of study design, medical conditions examined, and cell source used for iPSC generation. Moreover, a broad range of iPSC-derived cell products were administered. Variability among studies and ongoing trials may complicate and delay our understanding of the safety and efficacy of iPSC-based therapies.
CONCLUSIONS: iPSCs are poised to have a significant impact on clinical care for patients with conditions such as cardiac disease, cancer, and ocular disorders. We anticipate a period of several more years before the safety and efficacy of iPSC-based therapies can be definitively determined. Standardized study protocols and adherence to iPSC-derived product characterization criteria could facilitate more accelerated approval of iPSC-based therapies.
PMID:41160002 | DOI:10.1016/j.jcyt.2025.09.008
