An exceptional safety profile has been shown in a large number of cell therapy clinical trials that use mesenchymal stromal cells (MSCs). However, reliable potency assays are still lacking to predict MSC immunosuppressive efficacy in the clinical setting. Nevertheless, MSCs are approved in Japan and Europe for the treatment of graft-versus-host and Crohn’s fistular diseases, but not in the United States for any clinical indication. We discuss potential mechanisms of action for the therapeutic effects of MSC transplantation, experimental models that dissect tissue modulating function of MSCs, and approaches for identifying MSC effects in vivo by integrating biomarkers of disease and MSC activity.

大量使用间充质基质细胞 (MSC) 的细胞治疗临床试验显示出卓越的安全性。然而，仍然缺乏可靠的效力测定来预测临床环境中的 MSC 免疫抑制功效。尽管如此，MSCs 在日本和欧洲被批准用于治疗移植物抗宿主病和克罗恩氏瘘疾病，但在美国没有任何临床适应症。我们讨论了 MSC 移植治疗效果的潜在作用机制、剖析 MSC 组织调节功能的实验模型，以及通过整合疾病和 MSC 活性的生物标志物来识别体内MSC 作用的方法。