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应对新兴干细胞疗法临床转化过程中的机遇与挑战。

Managing the potential and pitfalls during clinical translation of emerging stem cell therapies.

作者信息

Main Heather, Munsie Megan, O'Connor Michael D

机构信息

The University of Sydney, Sydney, Australia.

Stem Cells Australia, Department of Anatomy and Neuroscience, The University of Melbourne, Sydney, Australia.

出版信息

Clin Transl Med. 2014 May 9;3:10. doi: 10.1186/2001-1326-3-10. eCollection 2014.

Abstract

We are moving into a new era of stem cell research where many possibilities for treatment of degenerative, chronic and/or fatal diseases and injuries are becoming primed for clinical trial. These reports have led millions of people worldwide to hope that regenerative medicine is about to revolutionise biomedicine: either through transplantation of cells grown in the laboratory, or by finding ways to stimulate a patient's intrinsic stem cells to repair diseased and damaged organs. While major contributions of stem cells to drug discovery, safety and efficacy testing, as well as modelling 'diseases in a dish' are also expected, it is the in vivo use of stem cells that has captured the general public's attention. However, public misconceptions of stem cell potential and applications can leave patients vulnerable to the influences of profit driven entities selling unproven treatments without solid scientific basis or appropriate clinical testing or follow up. This review provides a brief history of stem cell clinical translation together with an overview of the properties, potential, and current clinical application of various stem cell types. In doing so it presents a clearer picture of the inherent risks and opportunities associated with stem cell research translation, and thus offers a framework to help realise invested expectations more quickly, safely and effectively.

摘要

我们正在步入干细胞研究的新时代,许多治疗退行性、慢性和/或致命疾病及损伤的可能性正准备进入临床试验阶段。这些报道让全球数百万人燃起希望,认为再生医学即将给生物医学带来变革:要么通过移植在实验室培育的细胞,要么通过找到刺激患者自身干细胞修复患病和受损器官的方法。虽然人们也期待干细胞在药物研发、安全性和有效性测试以及“培养皿中的疾病”建模方面做出重大贡献,但干细胞在体内的应用才是吸引公众关注的焦点。然而,公众对干细胞潜力和应用的误解可能会使患者容易受到逐利实体的影响,这些实体兜售未经证实的治疗方法,既没有坚实的科学依据,也没有经过适当的临床试验或后续跟进。本综述简要介绍了干细胞临床转化的历史,并概述了各种干细胞类型的特性、潜力和当前临床应用。这样做能更清晰地呈现与干细胞研究转化相关的内在风险和机遇,从而提供一个框架,以帮助更快、更安全、更有效地实现所投入的期望。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea6f/4049443/6535765ddedc/2001-1326-3-10-1.jpg

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