{"title":"msc的特点:药理学和临床使用的关键质量属性","authors":"Sowmya Viswanathan, Jacques Galipeau","doi":"10.1016/j.stem.2025.05.008","DOIUrl":null,"url":null,"abstract":"Marketing approval for allogenic mesenchymal stromal cells (MSCs) by international regulatory jurisdictions including the US have been granted. Notwithstanding, the long-heralded clinical and commercial breakthrough for MSC products has never fully manifested. The withdrawal of an allogenic MSC product in Europe, based on inefficacious phase 3 results along with setbacks in industry-sponsored, advanced clinical trials of MSCs for COVID-19-related acute respiratory distress syndrome (ARDS) have dampened enthusiasm for MSC products. In this perspective, we highlight the hallmarks of MSC identity and potency, and how these can inform surrogate, sensitive critical quality attributes that correlate with clinical effectiveness in a variety of indications. We further highlight host-dependent pharmacological attributes of MSCs, which together with their critical quality attributes drive the observed clinical responses and thus impact the translational utility of MSCs. We provide a rational pathway to additional MSC regulatory approval and deployment for disorders with unmet medical needs.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"39 1","pages":""},"PeriodicalIF":19.8000,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Hallmarks of MSCs: Key quality attributes for pharmacology and clinical use\",\"authors\":\"Sowmya Viswanathan, Jacques Galipeau\",\"doi\":\"10.1016/j.stem.2025.05.008\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Marketing approval for allogenic mesenchymal stromal cells (MSCs) by international regulatory jurisdictions including the US have been granted. Notwithstanding, the long-heralded clinical and commercial breakthrough for MSC products has never fully manifested. The withdrawal of an allogenic MSC product in Europe, based on inefficacious phase 3 results along with setbacks in industry-sponsored, advanced clinical trials of MSCs for COVID-19-related acute respiratory distress syndrome (ARDS) have dampened enthusiasm for MSC products. In this perspective, we highlight the hallmarks of MSC identity and potency, and how these can inform surrogate, sensitive critical quality attributes that correlate with clinical effectiveness in a variety of indications. We further highlight host-dependent pharmacological attributes of MSCs, which together with their critical quality attributes drive the observed clinical responses and thus impact the translational utility of MSCs. We provide a rational pathway to additional MSC regulatory approval and deployment for disorders with unmet medical needs.\",\"PeriodicalId\":9665,\"journal\":{\"name\":\"Cell stem cell\",\"volume\":\"39 1\",\"pages\":\"\"},\"PeriodicalIF\":19.8000,\"publicationDate\":\"2025-06-05\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cell stem cell\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1016/j.stem.2025.05.008\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CELL & TISSUE ENGINEERING\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cell stem cell","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.stem.2025.05.008","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CELL & TISSUE ENGINEERING","Score":null,"Total":0}
Hallmarks of MSCs: Key quality attributes for pharmacology and clinical use
Marketing approval for allogenic mesenchymal stromal cells (MSCs) by international regulatory jurisdictions including the US have been granted. Notwithstanding, the long-heralded clinical and commercial breakthrough for MSC products has never fully manifested. The withdrawal of an allogenic MSC product in Europe, based on inefficacious phase 3 results along with setbacks in industry-sponsored, advanced clinical trials of MSCs for COVID-19-related acute respiratory distress syndrome (ARDS) have dampened enthusiasm for MSC products. In this perspective, we highlight the hallmarks of MSC identity and potency, and how these can inform surrogate, sensitive critical quality attributes that correlate with clinical effectiveness in a variety of indications. We further highlight host-dependent pharmacological attributes of MSCs, which together with their critical quality attributes drive the observed clinical responses and thus impact the translational utility of MSCs. We provide a rational pathway to additional MSC regulatory approval and deployment for disorders with unmet medical needs.
期刊介绍:
Cell Stem Cell is a comprehensive journal covering the entire spectrum of stem cell biology. It encompasses various topics, including embryonic stem cells, pluripotency, germline stem cells, tissue-specific stem cells, differentiation, epigenetics, genomics, cancer stem cells, stem cell niches, disease models, nuclear transfer technology, bioengineering, drug discovery, in vivo imaging, therapeutic applications, regenerative medicine, clinical insights, research policies, ethical considerations, and technical innovations. The journal welcomes studies from any model system providing insights into stem cell biology, with a focus on human stem cells. It publishes research reports of significant importance, along with review and analysis articles covering diverse aspects of stem cell research.