Hematti Peiman
Department of Medicine, University of Wisconsin-Madison School of Medicine and Public Health, University of Wisconsin Carbone Cancer Center, Madison, Wisconsin.
Transfusion. 2016 Apr;56(4):32S-5S. doi: 10.1111/trf.13569.
Based on their many different mechanisms of action, presumed immune-privileged status, and relative ease of production, mesenchymal stromal cells (MSCs) are under intensive clinical investigation for treating a wide range of degenerative, inflammatory, and immunologic disorders. Identification of relevant and robust potency assays is not only a regulatory requirement, but it is also the basis for producing and delivering a product that is consistent, safe, and ultimately an effective therapy. Although development of an appropriate potency assay is one of the most challenging issues in cell-based therapies, it is of paramount importance in the process of developing and testing cellular products. Regardless of the many different tissue sources and methods used in culture expansion of MSCs, they possess many of the same morphologic, cell surface markers, and differentiation characteristics. However, MSC products with similar phenotypic characteristics could still have major differences in their biologic and functional attributes. Understanding the different mechanisms of action and establishment of relevant potency assays is of pivotal importance in allowing investigators and regulatory agencies to compare MSCs used in different clinical trials.
基于其多种不同的作用机制、假定的免疫特权状态以及相对容易的生产方式，间充质基质细胞（MSCs）正在接受密集的临床研究，用于治疗多种退行性、炎症性和免疫性疾病。确定相关且可靠的效能测定方法不仅是一项监管要求，也是生产和交付一致、安全且最终有效的治疗产品的基础。尽管开发合适的效能测定方法是基于细胞的治疗中最具挑战性的问题之一，但在细胞产品的开发和测试过程中至关重要。无论在MSCs的培养扩增中使用了多少不同的组织来源和方法，它们都具有许多相同的形态学、细胞表面标志物和分化特征。然而，具有相似表型特征的MSC产品在其生物学和功能属性上仍可能存在重大差异。了解不同的作用机制并建立相关的效能测定方法对于研究人员和监管机构比较不同临床试验中使用的MSCs至关重要。
