International Society for Cell and Gene Therapy Clinical Translation Committee recommendations on mesenchymal stromal cells in graft-versus-host disease: Easy manufacturing is faced with standardizing and commercialization challenges Journal Article
| Authors: | Alsultan, A.; Farge, D.; Kili, S.; Forte, M.; Weiss, D. J.; Grignon, F.; Boelens, J. J. |
| Article Title: | International Society for Cell and Gene Therapy Clinical Translation Committee recommendations on mesenchymal stromal cells in graft-versus-host disease: Easy manufacturing is faced with standardizing and commercialization challenges |
| Abstract: | Mesenchymal stromal cells (MSCs) have been used in multiple clinical trials for steroid-refractory moderate-severe (grade II–IV) acute graft-versus-host disease (aGVHD) across the world over the last two decades. Despite very promising results in a variety of trials, it failed to get widespread approval by regulatory agencies such as the U.S. Food and Drug Administration and the European Medicines Agency. What lessons can we learn from this for future studies on MSCs and other cell therapy products? Broad heterogeneity among published trials using MSCs in aGVHD was likely the core problem. We propose a standardized approach in regards to donor-related factors, MSCs-related characteristics, as well as clinical trial design, to limit heterogeneity in trials for aGVHD and to fulfill the requirements of regulatory agencies. This approach may be expanded beyond MSCs to other Cell and Gene therapy products and trials in other diseases. © 2024 International Society for Cell & Gene Therapy |
| Keywords: | clinical trials as topic; cytology; practice guideline; acute graft versus host disease; immunogenicity; gene therapy; graft versus host reaction; medical society; remission; cell therapy; mesenchymal stem cell; biological therapy; gvhd; graft vs host disease; therapy; genetic heterogeneity; mesenchymal stem cells; hematopoietic cell transplantation; clinical trial (topic); procedures; genetic therapy; mesenchymal stroma cell; humans; human; article; cell- and tissue-based therapy; mscs; mesenchymal stem cell transplantation; manufacturing; isct; passage number |
| Journal Title: | Cytotherapy |
| Volume: | 26 |
| Issue: | 10 |
| ISSN: | 1465-3249 |
| Publisher: | Elsevier Science Ltd. |
| Date Published: | 2024-10-01 |
| Start Page: | 1132 |
| End Page: | 1140 |
| Language: | English |
| DOI: | 10.1016/j.jcyt.2024.05.007 |
| PUBMED: | 38804990 |
| PROVIDER: | scopus |
| PMCID: | PMC12046531 |
| DOI/URL: | |
| Notes: | The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in the PDF. Corresponding MSK author is Jaap Jan Boelens -- Source: Scopus |
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