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干细胞:临床试验结果是终点的开始还是开始的结束?

Stem cells: clinical trials results the end of the beginning or the beginning of the end?

作者信息

Behfar Atta, Crespo-Diaz Ruben, Nelson Timothy J, Terzic Andre, Gersh Bernard J

机构信息

Division of Cardiovascular Diseases, Department of Medicine, Mayo Clinic, Rochester, MN, USA.

出版信息

Cardiovasc Hematol Disord Drug Targets. 2010 Sep 1;10(3):186-201. doi: 10.2174/1871529x11006030186.

DOI:10.2174/1871529x11006030186
PMID:20678060
Abstract

With increasing focus on the advance towards curative solutions, it is hard not to be excited by the potential of stem cell-based therapy. Application of the stem cell paradigm to cardiovascular medicine has fostered the evolution of novel approaches aimed at reversing injury caused by ischemic and non-ischemic cardiomyopathy. The feasibility and safety of stem cell use has been established in over 3,000 patients with either recent myocardial infarction or chronic organ failure. Nonetheless, the efficacy of stem cell therapy continues to remain in question. Initial clinical trials have focused on evaluation of multiple adult stem cell phenotypes in their unaltered, naíve state as a "first generation" resource for repair. Though significant strides in perfecting delivery of these biologics to the diseased heart have been achieved, the benefits with regard to myocardial functional recovery have been modest at best. One approach towards optimizing outcome may lie upon preemptive guidance of stem cells down the pathway of myocyte regeneration. As seen with pharmacotherapeutics in the last century, successful translation of "second generation" biotherapeutics in the 21(st) century will require close integration of a community of practice and science to ensure broad application of this emerging technology in the treatment of heart disease.

摘要

随着对治愈性解决方案进展的关注度不断提高,很难不为基于干细胞的疗法的潜力感到兴奋。将干细胞模式应用于心血管医学促进了旨在逆转缺血性和非缺血性心肌病所致损伤的新方法的发展。干细胞使用的可行性和安全性已在3000多名近期心肌梗死或慢性器官衰竭患者中得到证实。尽管如此,干细胞疗法的疗效仍存在疑问。最初的临床试验集中于评估多种未改变的、天然状态的成体干细胞表型,将其作为修复的“第一代”资源。尽管在将这些生物制剂完美递送至患病心脏方面已取得显著进展,但在心肌功能恢复方面的益处充其量也很有限。一种优化结果的方法可能在于对干细胞进行先发引导,使其沿着心肌细胞再生的途径发展。正如上世纪药物治疗的情况一样,21世纪“第二代”生物治疗的成功转化将需要实践和科学界的紧密结合,以确保这项新兴技术在心脏病治疗中的广泛应用。

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引用本文的文献

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Endpoints in stem cell trials in ischemic heart failure.缺血性心力衰竭干细胞试验的终点指标。
Stem Cell Res Ther. 2015 Aug 29;6(1):159. doi: 10.1186/s13287-015-0143-9.
2
Systems-based technologies in profiling the stem cell molecular framework for cardioregenerative medicine.基于系统的技术在心脏再生医学中分析干细胞分子框架。
Stem Cell Rev Rep. 2015 Jun;11(3):501-10. doi: 10.1007/s12015-014-9557-5.
3
What is beyond a qRT-PCR study on mesenchymal stem cell differentiation properties: how to choose the most reliable housekeeping genes.
超越 qRT-PCR 研究的间充质干细胞分化特性:如何选择最可靠的管家基因。
J Cell Mol Med. 2013 Jan;17(1):168-80. doi: 10.1111/j.1582-4934.2012.01660.x. Epub 2013 Jan 11.
4
Regenerative medicine: on the vanguard of health care.再生医学:处于医疗保健的前沿。
Mayo Clin Proc. 2011 Jul;86(7):600-2. doi: 10.4065/mcp.2011.0325.