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Mesenchymal Stem Cells for the Treatment of Idiopathic Pulmonary Fibrosis

机译:间充质干细胞治疗特发性肺纤维化

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Idiopathic pulmonary fibrosis (IPF) is an inexorably progressive lung disease of unknown origin. Prognosis is poor, with limited treatment options available, and the median survival remains just 3–5 years. Despite the use of pirfenidone and nintedanib for the treatment of IPF, curative therapies remain elusive and mortality remains high. Regenerative medicine and the use of cell-based therapies has recently emerged as a potential option for various diseases. Promising results of preclinical studies using mesenchymal stem cells (MSCs) suggest that they may represent a potential therapeutic option for the treatment of chronic lung diseases including IPF. Encouraging results of Phase 1 studies of MSCs various have reduced safety concerns. Nonetheless, there is still a pressing need for exploratory biomarkers and interval end-points in the context of MSCs investigation. This review intends to summarize the current state of knowledge for stem cells in the experimental and clinical setting of IPF, present important safety and efficacy issues, highlight future challenges and address the need for large, multicenter clinical trials coupled with realistic end-points, including biomarkers, to assess treatment efficacy.
机译:特发性肺纤维化(IPF)是一种来源不明的无情进展性肺部疾病。预后较差,可用的治疗方法有限,中位生存期仅为3-5年。尽管使用吡非尼酮和任他尼单抗治疗IPF,但根治性疗法仍然难以捉摸,死亡率仍然很高。再生医学和基于细胞的疗法的使用最近已成为各种疾病的潜在选择。使用间充质干细胞(MSC)的临床前研究的有希望的结果表明,它们可能代表了治疗包括IPF在内的慢性肺部疾病的潜在治疗选择。各种MSC的第1阶段研究的令人鼓舞的结果减少了安全隐患。然而,在MSC研究的背景下,仍然迫切需要探索性生物标志物和区间终点。这篇综述旨在总结IPF实验和临床环境中干细胞的当前知识状态,提出重要的安全性和有效性问题,强调未来的挑战,并解决对大型多中心临床试验以及现实终点的需求,包括生物标志物,以评估治疗效果。

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