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间充质干细胞疗法对多发性硬化症患者有帮助吗?一项试点研究报告。

Does mesenchymal stem cell therapy help multiple sclerosis patients? Report of a pilot study.

作者信息

Mohyeddin Bonab Mandana, Yazdanbakhsh Sepideh, Lotfi Jamshid, Alimoghaddom Kamran, Talebian Fatemeh, Hooshmand Farnaz, Ghavamzadeh Ardeshir, Nikbin Behrouz

机构信息

Hematology-Oncology & BMT Research Center, Tehran, Iran.

出版信息

Iran J Immunol. 2007 Mar;4(1):50-7.

Abstract

BACKGROUND

Mesenchymal stem cells (MSCs) with their potential to differentiate into mesodermal and non-mesodermal lineages have several immunomodulatory characteristics. These properties make them promising tools in cell and gene therapy.

OBJECTIVE

To evaluate the potential therapeutic applications of autologous MSC in improving clinical manifestations of MS patients.

METHODS

Ten patients were included in this pilot study. All had progressive disease that had not responded to disease modifying agents including Mitoxantrone. Their Expanded Disability Status Scale (EDSS) score ranged from 3.5 to 6. Patients were injected intrathecally with culture expanded MSCs. They were followed with monthly neurological assessment and a MRI scan at the end of the first year.

RESULTS

During 13 to 26 months of follow up (mean: 19 months), the EDSS of one patient improved from 5 to 2.5 score. Four patients showed no change in EDSS. Five patients' EDSS increased from 0.5 to 2.5. In the functional system assessment, six patients showed some degree of improvement in their sensory, pyramidal, and cerebellar functions. One showed no difference in clinical assessment and three deteriorated. The result of MRI assessment after 12 months was as following: seven patients with no difference, two showed an extra plaque, and one patient showed decrease in the number of plaques.

CONCLUSION

This preliminary report emphasizes on the feasibility of autologous MSC for treatment of MS patients. However, in order to draw a definitive conclusion a larger sample size is required.

摘要

背景

间充质干细胞(MSCs)具有分化为中胚层和非中胚层谱系的潜力,具备多种免疫调节特性。这些特性使其成为细胞和基因治疗中有前景的工具。

目的

评估自体间充质干细胞在改善多发性硬化症(MS)患者临床表现方面的潜在治疗应用。

方法

本初步研究纳入了10名患者。所有患者均患有进展性疾病,对包括米托蒽醌在内的疾病修饰药物均无反应。他们的扩展残疾状态量表(EDSS)评分在3.5至6之间。患者接受了经鞘内注射培养扩增的间充质干细胞治疗。对他们进行每月一次的神经学评估,并在第一年末进行一次磁共振成像(MRI)扫描。

结果

在13至26个月的随访期间(平均:19个月),1名患者的EDSS评分从5改善至2.5。4名患者的EDSS无变化。5名患者的EDSS从0.5增加至2.5。在功能系统评估中,6名患者的感觉、锥体束和小脑功能有一定程度的改善。1名患者的临床评估无差异,3名患者病情恶化。12个月后的MRI评估结果如下:7名患者无差异,2名患者出现额外斑块,1名患者斑块数量减少。

结论

本初步报告强调了自体间充质干细胞治疗MS患者的可行性。然而,为得出明确结论,需要更大的样本量。

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