University of Alabama at Birmingham, Division of Gastroenterology and Hepatology, 1825 University Boulevard, SHEL613, Birmingham, AL 35213, USA.
Expert Opin Biol Ther. 2011 Sep;11(9):1249-56. doi: 10.1517/14712598.2011.602967. Epub 2011 Jul 25.
Human mesenchymal stem cells (Prochymal brand of remestemcel-L) have been developed for experimental use in Crohn's disease and other conditions. Mesenchymal stems cells (MSCs) have been shown to inhibit inflammatory responses of innate and adaptive immune cells as well as have reparative effects on inflamed tissues. Promising preliminary therapeutic results of MSCs on gastrointestinal graft-versus-host disease have lead to Phase III trials for active Crohn's disease.
This review examines the discovery and characterization of mesenchymal stem cells, their immune effects, their use in animal models of disease, the production and administration of remestemcel-L and the published results of trials of remestemcel-L and alternative MSCs in Crohn's disease.
The Prochymal brand of remestemcel-L represents the successful pharmaceutical development of mesenchymal stem cells for potential therapy in human disease. While preliminary results show promise of this therapy in terms of efficacy and safety, robust trials confirming efficacy results, explanation of the mechanism of response and estimates of effect compared to other biologics and immunosuppressants will be needed before this is an approved and widely accepted therapy.
人类间充质干细胞（Prochymal 品牌的 remestemcel-L）已被开发用于克罗恩病和其他疾病的实验用途。间充质干细胞（MSCs）已被证明可以抑制先天和适应性免疫细胞的炎症反应，并且对炎症组织具有修复作用。MSCs 对胃肠道移植物抗宿主病的初步有希望的治疗结果导致了针对活动性克罗恩病的 III 期试验。
本综述考察了间充质干细胞的发现和特征、它们的免疫作用、它们在疾病动物模型中的应用、remestemcel-L 的生产和管理以及 remestemcel-L 和替代 MSCs 在克罗恩病中的试验结果。
Prochymal 品牌的 remestemcel-L 代表了间充质干细胞在人类疾病潜在治疗中的成功药物开发。虽然初步结果显示这种疗法在疗效和安全性方面有希望，但需要进行强有力的试验来确认疗效结果，解释反应机制，并与其他生物制剂和免疫抑制剂进行效果估计，然后才能获得批准并被广泛接受。
