Wuchter Patrick, Bieback Karen, Schrezenmeier Hubert, Bornhäuser Martin, Müller Lutz P, Bönig Halvard, Wagner Wolfgang, Meisel Roland, Pavel Petra, Tonn Torsten, Lang Peter, Müller Ingo, Renner Matthias, Malcherek Georg, Saffrich Rainer, Buss Eike C, Horn Patrick, Rojewski Markus, Schmitt Anita, Ho Anthony D, Sanzenbacher Ralf, Schmitt Michael
Department of Medicine V, Heidelberg University, Heidelberg, Germany.
Institute of Transfusion Medicine and Immunology Mannheim, Medical Faculty Mannheim, Heidelberg University, German Red Cross Blood Donor Service Baden-Württemberg-Hessen, Mannheim, Germany.
Cytotherapy. 2015 Feb;17(2):128-39. doi: 10.1016/j.jcyt.2014.04.002. Epub 2014 May 20.
Human mesenchymal stem or stromal cells (MSCs) represent a potential resource not only for regenerative medicine but also for immunomodulatory cell therapies. The application of different MSC culture protocols has significantly hampered the comparability of experimental and clinical data from different laboratories and has posed a major obstacle for multicenter clinical trials. Manufacturing of cell products for clinical application in the European Community must be conducted in compliance with Good Manufacturing Practice and requires a manufacturing license. In Germany, the Paul-Ehrlich-Institut as the Federal Authority for Vaccines and Biomedicines is critically involved in the approval process.
This report summarizes a consensus meeting between researchers, clinicians and regulatory experts on standard quality requirements for MSC production.
The strategy for quality control testing depends on the product's cell composition, the manufacturing process and the indication and target patient population. Important quality criteria in this sense are, among others, the immunophenotype of the cells, composition of the culture medium and the risk for malignant transformation, as well as aging and the immunosuppressive potential of the manufactured MSCs.
This position paper intends to provide relevant information to interested parties regarding these criteria to foster the development of scientifically valid and harmonized quality standards and to support approval of MSC-based investigational medicinal products.
人间充质干细胞或基质细胞（MSCs）不仅是再生医学的潜在资源，也是免疫调节细胞疗法的潜在资源。不同的MSC培养方案的应用严重阻碍了不同实验室实验数据和临床数据的可比性，并对多中心临床试验构成了重大障碍。在欧盟，用于临床应用的细胞产品的生产必须符合良好生产规范，并需要生产许可证。在德国，作为联邦疫苗和生物药品管理机构的保罗·埃利希研究所深度参与审批过程。
本报告总结了研究人员、临床医生和监管专家之间就MSC生产的标准质量要求召开的一次共识会议。
质量控制检测策略取决于产品的细胞组成、生产工艺以及适应症和目标患者群体。从这个意义上讲，重要的质量标准包括细胞的免疫表型、培养基的组成、恶性转化风险，以及所生产的MSCs的老化和免疫抑制潜力等。
本立场文件旨在向相关方提供有关这些标准的相关信息，以促进科学有效的统一质量标准的制定，并支持基于MSC的 investigational medicinal products的批准。
