Spoerl Silvia, Peter Robert, Krackhardt Angela M
III. Medical Department, Hematology and Oncology, Klinikum rechts der Isar, Technische Universität München, Ismaningerstr. 22, 81675, München, Germany.
Adv Exp Med Biol. 2016;951:67-76. doi: 10.1007/978-3-319-45457-3_6.
Autologous and allogeneic stem cell transplantation (SCT) represents a therapeutic option widely used for hematopoietic malignancies. One important milestone in the development of this treatment strategy was the development of effective cryopreservation technologies resulting in a high quality with respect to cell viability as well as lack of contamination of the graft.Stem cell preparations have been initially performed within standard laboratories as it is routinely still the case in many countries. With the emergence of cleanrooms, manufacturing of stem cell preparations within these facilities has become a new standard mandatory in Europe. However, due to high costs and laborious procedures, novel developments recently emerged using closed bag systems as reliable alternatives to conventional cleanrooms. Several hurdles needed to be overcome including the addition of the cryoprotectant dimethylsulfoxide (DMSO) as a relevant manipulation. As a result of the development, closed bag systems proved to be comparable in terms of product quality and patient outcome to cleanroom products. They also comply with the strict regulations of good manufacturing practice.With closed systems being available, costs and efforts of a cleanroom facility may be substantially reduced in the future. The process can be easily extended for other cell preparations requiring minor modifications as donor lymphocyte preparations. Moreover, novel developments may provide solutions for the production of advanced-therapy medicinal products in closed systems.
自体和异体干细胞移植（SCT）是治疗造血系统恶性肿瘤广泛应用的一种治疗选择。这种治疗策略发展过程中的一个重要里程碑是有效冷冻保存技术的发展，该技术能保证细胞活力的高质量以及移植物无污染。干细胞制备最初是在标准实验室中进行的，目前在许多国家仍是常规做法。随着洁净室的出现，在这些设施内进行干细胞制备已成为欧洲新的强制标准。然而，由于成本高昂且程序繁琐，最近出现了一些新进展，使用封闭袋系统作为传统洁净室的可靠替代方案。需要克服几个障碍，包括添加冷冻保护剂二甲基亚砜（DMSO）这一相关操作。经过发展，封闭袋系统在产品质量和患者预后方面被证明与洁净室产品相当。它们也符合严格的药品生产质量管理规范。有了封闭系统，未来洁净室设施的成本和工作量可能会大幅降低。该过程可以很容易地扩展到其他需要进行微小修改的细胞制备，如供体淋巴细胞制备。此外，新的进展可能为在封闭系统中生产高级治疗药品提供解决方案。
