Diamandis Theo, Borlongan Cesar V
From the Department of Neurosugery and Brain Repair, Center of Excellence for Aging and Brain Repair, Morsani College of Medicine, University of South Florida, Tampa.
Stroke. 2015 Feb;46(2):588-91. doi: 10.1161/STROKEAHA.114.007105. Epub 2014 Dec 11.
Many clinical trials have failed despite positive laboratory findings. Stroke clinical trials are no exception, with tissue plasminogen activator (tPA) still the only effective drug for stroke with limited therapeutic window. In order to enhance the successful outcome of novel therapies in the clinic, initiatives for translational research guidelines have been pursued. In particular, the advancement of stem cell therapy for stroke from the laboratory to the clinic has now been guided by a set of recommendations called Stem cell Therapeutics as an Emerging Paradigm for Stroke or STEPS. We review here the major criteria for preclinical studies of stem cells arising from the three STEPS meetings in an effort to further emphasize the need for careful and rigorous assessment of the safety, efficacy, and mechanism of action associated with stem cell therapy for stroke. Learning from our previous mistakes and identifying gaps in knowledge will likely prevent stem cell therapy from becoming yet another statistic of failed clinical trial in stroke.
尽管实验室研究结果呈阳性,但许多临床试验仍以失败告终。中风临床试验也不例外,组织纤溶酶原激活剂(tPA)仍然是治疗中风的唯一有效药物,但其治疗窗口有限。为了提高新疗法在临床上的成功率,人们一直在推行转化研究指南倡议。特别是,从实验室到临床的中风干细胞治疗进展现在受到了一套名为“干细胞治疗作为中风的新兴范例”(简称STEPS)的建议的指导。我们在此回顾了来自三次STEPS会议的干细胞临床前研究的主要标准,以进一步强调对中风干细胞治疗的安全性、有效性和作用机制进行仔细而严格评估的必要性。从我们以前的错误中吸取教训并找出知识空白,可能会防止干细胞治疗成为中风临床试验失败的又一个统计数据。