Krause Mirja, Phan Thanh G, Ma Henry, Sobey Christopher G, Lim Rebecca
The Ritchie Centre, Hudson Institute of Medical Research, Melbourne, VIC, Australia.
Department of Obstetrics and Gynaecology, Monash University, Melbourne, VIC, Australia.
Front Neurol. 2019 Jun 25;10:656. doi: 10.3389/fneur.2019.00656. eCollection 2019.
Stroke is the second leading cause of death and physical disability, with a global lifetime incidence rate of 1 in 6. Currently, the only FDA approved treatment for ischemic stroke is the administration of tissue plasminogen activator (tPA). Stem cell clinical trials for stroke have been underway for close to two decades, with data suggesting that cell therapies are safe, feasible, and potentially efficacious. However, clinical trials for stroke account for <1% of all stem cell trials. Nevertheless, the resources devoted to clinical research to identify new treatments for stroke is still significant (53-64 million US$, Phase 1-4). Notably, a quarter of cell therapy clinical trials for stroke have been withdrawn (15.2%) or terminated (6.8%) to date. This review discusses the bottlenecks in delivering a successful cell therapy for stroke, and the cost-to-benefit ratio necessary to justify these expensive trials. Further, this review will critically assess the currently available data from completed stroke trials, the importance of standardization in outcome reporting, and the role of industry-led research in the development of cell therapies for stroke.
中风是导致死亡和身体残疾的第二大原因，全球终生发病率为六分之一。目前，美国食品药品监督管理局（FDA）唯一批准的缺血性中风治疗方法是给予组织纤溶酶原激活剂（tPA）。针对中风的干细胞临床试验已经进行了近二十年，数据表明细胞疗法是安全、可行且可能有效的。然而，中风的临床试验占所有干细胞试验的比例不到1%。尽管如此，用于确定中风新治疗方法的临床研究资源仍然相当可观（5300万至6400万美元，1-4期）。值得注意的是，迄今为止，四分之一的中风细胞疗法临床试验已被撤回（15.2%）或终止（6.8%）。本综述讨论了为中风提供成功细胞疗法的瓶颈，以及证明这些昂贵试验合理性所需的成本效益比。此外，本综述将批判性地评估已完成的中风试验目前可得的数据、结果报告标准化的重要性以及行业主导研究在中风细胞疗法开发中的作用。
