The unmet need for therapies capable of repairing the central nervous system (CNS) damage occurring in many diseases including multiple sclerosis (MS) has sparked the interest of the neurological community for stem cell-based therapies. An exhaustive amount of preclinical data has shown that the intravenous administration of mesenchymal stem cells (MSC), a subset of progenitor cells isolated from many mesodermal tissues, effectively ameliorates experimental autoimmune encephalomyelitis (EAE), a model of MS, through the release of anti-inflammatory and neuroprotective molecules. Based on these results, several small pilot clinical trials in subjects with advanced MS have demonstrated that MSC administration is safe and provided an early signal of clinical effectiveness. The current aim of clinicians and scientists interested in the development of MSC-based strategies for the treatment of MS is to have the ultimate demonstration in large clinical trials that MSC can inhibit CNS inflammation and foster tissue repair as realized clinically, with functional recovery, or visualized by magnetic resonance imaging (MRI).

译文

对能够修复包括多发性硬化症 (MS) 在内的许多疾病中发生的中枢神经系统 (CNS) 损伤的疗法的未满足需求,引发了神经学界对基于干细胞的疗法的兴趣。大量的临床前数据表明,静脉内施用间充质干细胞 (MSC) (从许多中胚层组织中分离出的祖细胞的子集) 通过释放有效地改善了MS模型的实验性自身免疫性脑脊髓炎 (EAE) 炎症和神经保护分子。基于这些结果,在患有晚期MS的受试者中进行的一些小型试点临床试验表明,MSC给药是安全的,并提供了临床有效性的早期信号。对基于MSC的MS治疗策略的开发感兴趣的临床医生和科学家目前的目标是在大型临床试验中最终证明MSC可以抑制CNS炎症并促进组织修复,如临床实现的那样,具有功能恢复,或通过磁共振成像 (MRI) 可视化。

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