干细胞
医学
干细胞疗法
临床试验
肌萎缩侧索硬化
间充质干细胞
细胞疗法
移植
重症监护医学
神经科学
生物信息学
病理
内科学
心理学
疾病
生物
遗传学
作者
Letizia Mazzini,Angelo L. Vescovi,Roberto Cantello,Maurizio Gelati,Alessandro Vercelli
标识
DOI:10.1517/14712598.2016.1116516
摘要
Despite knowledge on the molecular basis of amyotrophic lateral sclerosis (ALS) having quickly progressed over the last few years, such discoveries have not yet translated into new therapeutics. With the advancement of stem cell technologies there is hope for stem cell therapeutics as novel treatments for ALS.We discuss in detail the therapeutic potential of different types of stem cells in preclinical and clinical works. Moreover, we address many open questions in clinical translation.SC therapy is a potentially promising new treatment for ALS and the need to better understand how to develop cell-based experimental treatments, and how to implement them in clinical trials, becomes more pressing. Mesenchymal stem cells and neural fetal stem cells have emerged as safe and potentially effective cell types, but there is a need to carry out appropriately designed experimental studies to verify their long-term safety and possibly efficacy. Moreover, the cost-benefit analysis of the results must take into account the quality of life of the patients as a major end point. It is our opinion that a multicenter international clinical program aime d at fine-tuning and coordinating transplantation procedures and protocols is mandatory.
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