Marsili Luca, Sharma Jennifer, Outeiro Tiago Fleming, Colosimo Carlo
Gardner Family Center for Parkinson's Disease and Movement Disorders, Department of Neurology, University of Cincinnati, Cincinnati, OH 45219, USA.
Department of Neurology, Queens University, Kingston K7L 3N6, ON, Canada.
Biomedicines. 2023 Feb 9;11(2):505. doi: 10.3390/biomedicines11020505.
Stem cell-based therapies (SCT) to treat neurodegenerative disorders have promise but clinical trials have only recently begun, and results are not expected for several years. While most SCTs largely lead to a symptomatic therapeutic effect by replacing lost cell types, there may also be disease-modifying therapeutic effects. In fact, SCT may complement a multi-drug, subtype-specific therapeutic approach, consistent with the idea of precision medicine, which matches molecular therapies to biological subtypes of disease. In this narrative review, we examine published and ongoing trials in SCT in Parkinson's Disease, atypical parkinsonian disorders, Huntington's disease, amyotrophic lateral sclerosis, and spinocerebellar ataxia in humans. We discuss the benefits and pitfalls of using this treatment approach within the spectrum of disease-modification efforts in neurodegenerative diseases. SCT may hold greater promise in the treatment of neurodegenerative disorders, but much research is required to determine the feasibility, safety, and efficacy of these complementary aims of therapeutic efforts.
基于干细胞的疗法（SCT）用于治疗神经退行性疾病具有前景，但临床试验直到最近才开始，而且几年内都不会有结果。虽然大多数SCT主要通过替代丢失的细胞类型来产生对症治疗效果，但也可能存在疾病修饰治疗效果。事实上，SCT可能补充多药、亚型特异性治疗方法，这与精准医学的理念一致，即分子疗法与疾病的生物学亚型相匹配。在这篇叙述性综述中，我们研究了已发表的和正在进行的针对人类帕金森病、非典型帕金森病、亨廷顿病、肌萎缩侧索硬化症和脊髓小脑共济失调的SCT试验。我们讨论了在神经退行性疾病的疾病修饰治疗范围内使用这种治疗方法的益处和陷阱。SCT在治疗神经退行性疾病方面可能有更大的前景，但需要大量研究来确定这些治疗努力的互补目标的可行性、安全性和有效性。
