Garbuzova-Davis Svitlana, Ehrhart Jared, Sanberg Paul R
a Center of Excellence for Aging & Brain Repair , University of South Florida, Morsani College of Medicine , Tampa , FL , USA.
b Department of Neurosurgery and Brain Repair , University of South Florida, Morsani College of Medicine , Tampa , FL , USA.
Expert Opin Biol Ther. 2017 Jul;17(7):837-851. doi: 10.1080/14712598.2017.1323862. Epub 2017 May 8.
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive motor neuron degeneration in the brain and spinal cord. Treatment options are limited due to the complexity of underlying disease factors. Cell therapy, using human umbilical cord blood (hUCB) cells may be a promising new treatment for ALS, mainly by providing a protective microenvironment for motor neuron survival. Areas covered: Composition, in vitro and in vivo differentiation of hUCB cells, and the advantages of cord blood as a source of transplant cells are discussed. A brief history of hUCB in treatment of an ALS animal model and the feasibility of these cells in therapy for ALS patients is provided. Current ALS clinical trials are also deliberated. Expert opinion: Among multiple advantages, hUCB cells' production of various anti-inflammatory/growth/trophic factors makes them an attractive cell source for ALS therapy. Biodistribution and optimal hUCB cell dose for transplantation have been determined in preclinical studies. Repeated intravenous cell doses during disease progression may be the best approach for cell-based ALS treatment. Accumulated evidence shows the efficacy of naïve or genetically modified MNC hUCB cells in the treatment of ALS and provide a superior basis for the development of clinical trials in the near future.
肌萎缩侧索硬化症（ALS）是一种致命的神经退行性疾病，其特征是大脑和脊髓中的运动神经元进行性退化。由于潜在疾病因素的复杂性，治疗选择有限。使用人脐带血（hUCB）细胞的细胞疗法可能是一种有前景的ALS新疗法，主要是通过为运动神经元存活提供保护性微环境。涵盖领域：讨论了hUCB细胞的组成、体外和体内分化，以及脐带血作为移植细胞来源的优势。介绍了hUCB治疗ALS动物模型的简要历史以及这些细胞用于治疗ALS患者的可行性。还讨论了当前的ALS临床试验。专家观点：在众多优势中，hUCB细胞产生各种抗炎/生长/营养因子使其成为ALS治疗有吸引力的细胞来源。临床前研究已确定了hUCB细胞移植的生物分布和最佳剂量。在疾病进展期间重复静脉注射细胞剂量可能是基于细胞的ALS治疗的最佳方法。积累的证据表明未处理或基因修饰的hUCB单个核细胞在治疗ALS方面的疗效，并为在不久的将来开展临床试验提供了优越的基础。
