Neural Stem Cell Institute, Rensselaer, NY 12144, USA.
Memorial Sloan Kettering Cancer Center, Center for Stem Cell Biology, New York, NY 10065, USA.
Stem Cell Reports. 2017 Feb 14;8(2):191-193. doi: 10.1016/j.stemcr.2017.01.024.
As stem cell products are increasingly entering early stage clinical trials, we are learning from experience about how cell products may be best assessed for safety and efficacy. In two papers published in this issue of Stem Cell Reports, a human neural stem cell product, HuCNS-SC, failed to demonstrate efficacy in central nervous system repair in two different animal models (Anderson et al., 2017; Marsh et al., 2017), although closely related research-grade cell products showed evidence of efficacy. This indicates the need for increased cell characterization to determine comparability of lots proposed for pre-clinical and clinical use. Without such improvements, pre-clinical data supporting a clinical study might not adequately reflect the performance of subsequent batches of cells intended for use in patients.
随着干细胞产品越来越多地进入早期临床试验，我们从经验中了解到如何最好地评估细胞产品的安全性和有效性。在本期《Stem Cell Reports》上发表的两篇论文中，一种人类神经干细胞产品 HuCNS-SC 在两种不同的动物模型中未能显示出对中枢神经系统修复的疗效（Anderson 等人，2017 年；Marsh 等人，2017 年），尽管密切相关的研究级细胞产品显示出了疗效的证据。这表明需要增加细胞特征分析，以确定拟用于临床前和临床用途的批次的可比性。如果没有这些改进，支持临床研究的临床前数据可能无法充分反映后续用于患者的细胞批次的性能。
