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间充质基质细胞治疗克罗恩病的进展。

Advances in mesenchymal stromal cell therapy in the management of Crohn's disease.

机构信息

a Department of Gastroenterology , Guy's and St Thomas' Hospital , London , UK.

b School of Immunology and Microbial Sciences , King's College London , London , UK.

出版信息

Expert Rev Gastroenterol Hepatol. 2018 Feb;12(2):141-153. doi: 10.1080/17474124.2018.1393332. Epub 2017 Nov 2.

Abstract

The aim of therapy in Crohn's disease (CD) is induction and maintenance of remission, promotion of mucosal healing and restoration of quality of life. Even the best treatment regimes, including combinations of biologics and immunomodulators lack durable efficacy and have well documented side effects. Accordingly, there is an unmet need for novel therapies. Mesenchymal stromal cells (MSCs) are a subset of non-hematopoietic stem cells that home to sites of inflammation where they exert potent immunomodulatory effects and contribute to tissue repair. Their utility is being explored in several inflammatory and immune mediated disorders including CD, where they have demonstrated favourable safety, feasibility and efficacy profiles. Areas covered: This review highlights current knowledge on MSC therapy and critically evaluates their safety, efficacy and potential mechanisms of action in CD. Expert commentary: Building on positive early phase clinical trials and a recent phase 3 trial in perianal CD, there is considerable optimism for the possibility of MSCs changing the treatment landscape in complicated CD. Although important questions remain unanswered, including the safety and durability of MSC therapy, optimal adjunctive therapies and their sourcing and manufacturing, it is anticipated that MSCs are likely to enter mainstream treatment algorithms in the near future.

摘要

目的 治疗克罗恩病(CD)的目的是诱导和维持缓解,促进黏膜愈合,恢复生活质量。即使是最好的治疗方案,包括生物制剂和免疫调节剂的联合治疗,都缺乏持久的疗效,并有明确的副作用记录。因此,需要新的治疗方法。间充质基质细胞(MSCs)是一组非造血干细胞,它们归巢到炎症部位,在那里发挥强大的免疫调节作用,并有助于组织修复。它们在几种炎症和免疫介导的疾病中得到了探索,包括 CD,在这些疾病中,它们表现出良好的安全性、可行性和疗效。 涵盖领域:本综述强调了 MSC 治疗的现有知识,并批判性地评估了它们在 CD 中的安全性、疗效和潜在作用机制。 专家评论:基于早期积极的临床试验和最近一项肛周 CD 的 3 期试验,人们对间充质干细胞改变复杂 CD 治疗前景抱有很大的期望。尽管仍有一些重要问题尚未得到解答,包括 MSC 治疗的安全性和持久性、最佳辅助治疗及其来源和制造,但预计间充质干细胞将在不久的将来进入主流治疗方案。

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