Stem cell treatment in Amyotrophic Lateral Sclerosis
Stem cell treatment in Amyotrophic Lateral Sclerosis
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DOI：
10.1016/j.jns.2007.05.016
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发表时间：
2008-02-15
影响因子：
4.4
通讯作者：
Fagioli, Franca
中科院分区：
文献类型：
--
作者：
Mazzini, Letizia;Mareschi, Katia;Fagioli, Franca
肌萎缩侧索硬化症是一种以运动神经元为靶点的进行性致死性神经退行性疾病。其起源尚不清楚，但最近已证实反应性星形胶质细胞增生和小胶质细胞活化在发病机制中的主要作用。在某些情况下，周围神经元与健康的相邻细胞完全阻止运动神经元死亡。因此，干细胞移植可能是一个有前途的治疗策略。在这项研究中，从9例确诊的ALS患者的骨髓中分离MSC。在体外扩增过程中，生长动力学，免疫表型，端粒长度和核型进行了评价。供体和患者之间没有观察到显著差异。患者接受自体骨髓间充质干细胞的椎管内注射在胸部水平和监测4年。没有明显的急性或晚期副作用。未观察到脊髓体积改变或其他异常细胞增殖体征。4例患者用力肺活量和ALS-FRS评分的线性下降明显减慢。我们的研究结果似乎表明，骨髓间充质干细胞是一个很好的机会，为干细胞为基础的治疗ALS和骨髓间充质干细胞的椎管内注射是安全的，从长远来看。进行了一项新的1期研究，以在更多患者中验证这些数据。(C)2007 Elsevier B. V.保留所有权利。
Amyotrophic Lateral Sclerosis is a progressive fatal neurodegenerative disease that targets motor neurons. Its origin is unknown but a main role of reactive astrogliosis and microglia activation in the pathogenesis has been recently demonstrated. Surrounding neurons with healthy adjoining cells completely stops motor neuron death in some cases. Hence stem cell transplantation might represent a promising therapeutic strategy. In this study MSCs were isolated from bone marrow of 9 patients with definite ALS. Growth kinetics, immunophenotype, telomere length and karyotype were evaluated during in vitro expansion. No significant differences between donors or patients were observed. The patients received intraspinal injections of autologous MSCs at the thoracic level and monitored for 4 years. No significant acute or late side effects were evidenced. No modification of the spinal cord volume or other signs of abnormal cell proliferation were observed. Four patients show a significant slowing down of the linear decline of the forced vital capacity and of the ALS-FRS score. Our results seem to demonstrate that MSCs represent a good chance for stem cell cell-based therapy in ALS and that intraspinal injection of MSCs is safe also in the long term. A new phase 1 study is carried out to verify these data in a larger number of patients. (C) 2007 Elsevier B.V. All rights reserved.