msc的特点:药理学和临床使用的关键质量属性

IF 19.8 1区 医学 Q1 CELL & TISSUE ENGINEERING
Sowmya Viswanathan, Jacques Galipeau
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引用次数: 0

摘要

同种异体间充质间质细胞(MSCs)已被包括美国在内的国际监管机构批准上市。尽管如此,MSC产品长期以来的临床和商业突破从未完全体现出来。基于无效的3期临床结果,一种同种异体间充质干细胞产品在欧洲被撤回,再加上业界赞助的MSC治疗covid -19相关急性呼吸窘迫综合征(ARDS)的高级临床试验受挫,这些都抑制了对MSC产品的热情。从这个角度来看,我们强调了MSC身份和效力的特征,以及这些特征如何告知与各种适应症的临床有效性相关的替代、敏感的关键质量属性。我们进一步强调了MSCs的宿主依赖的药理学属性,这些属性与它们的关键质量属性一起驱动观察到的临床反应,从而影响MSCs的转化效用。我们为未满足医疗需求的疾病提供了额外的MSC监管批准和部署的合理途径。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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.
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来源期刊
Cell stem cell
Cell stem cell 生物-细胞生物学
CiteScore
37.10
自引率
2.50%
发文量
151
审稿时长
42 days
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.
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