Department of Rheumatology, University of Basel, Petersgraben 4, CH-4031, Basel, Switzerland.
Nat Rev Rheumatol. 2014 Feb;10(2):117-24. doi: 10.1038/nrrheum.2013.166. Epub 2013 Nov 12.
Mesenchymal stem cells (MSCs) represent a heterogeneous progenitor cell population derived from various sources, including bone marrow, placental and adipose tissues. These cell populations are being extensively investigated for their regenerative, immunomodulatory and tissue-protective properties, and the therapeutic potential of MSCs is officially being tested in patients suffering from ischaemic, inflammatory, autoimmune and degenerative disorders. Unofficially, hundreds of centres worldwide already offer MSCs as a 'miracle' panacea treatment for almost every known human disease. Data from in vitro and animal models suggest that MSCs administered either locally or systemically are able to home to stressed tissue and indeed deliver a protective effect via predominately paracrine factors. Furthermore, dozens of published uncontrolled clinical trials have demonstrated strikingly positive therapeutic effects of MSCs with little acute toxicity; however, no prospective controlled trials have yet confirmed these findings, with the exception of one randomized controlled trial in renal transplantation. Thus, large prospective controlled trials are urgently needed to better understand MSC-based therapies and define their potential utility in the treatment of rheumatic diseases. Herein, I provide my opinions regarding the progress of MSC therapies to date and highlight issues that need to be addressed in the future.
间充质干细胞(MSCs)是一种来源于多种来源的异质性祖细胞群体,包括骨髓、胎盘和脂肪组织。这些细胞群体因其再生、免疫调节和组织保护特性而受到广泛研究,间充质干细胞的治疗潜力正在接受患有缺血、炎症、自身免疫和退行性疾病的患者的正式测试。非官方地说,全球已有数百个中心将 MSCs 作为一种“神奇”的万能疗法,用于治疗几乎所有已知的人类疾病。来自体外和动物模型的数据表明,局部或系统给予的 MSCs 能够归巢到应激组织,并通过主要旁分泌因子发挥保护作用。此外,数十项已发表的非对照临床试验表明,间充质干细胞具有显著的治疗效果,几乎没有急性毒性;然而,除了一项肾移植的随机对照试验外,目前还没有前瞻性对照试验证实这些发现。因此,迫切需要进行大型前瞻性对照试验,以更好地了解基于 MSC 的治疗方法,并确定其在治疗风湿性疾病中的潜在用途。在此,我就 MSCs 治疗的进展提出了自己的观点,并强调了未来需要解决的问题。