Safety and Feasibility of Autologous Bone Marrow Cellular Therapy in Relapsing-Progressive Multiple Sclerosis
Safety and Feasibility of Autologous Bone Marrow Cellular Therapy in Relapsing-Progressive Multiple Sclerosis
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DOI：
10.1038/clpt.2010.44
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发表时间：
2010-06-01
影响因子：
6.7
通讯作者：
Scolding, N. J.
中科院分区：
文献类型：
--
作者：
Rice, C. M.;Mallam, E. A.;Scolding, N. J.
在这项I期研究中，我们评估了6例临床明确的复发进行性多发性硬化症（MS）患者静脉注射自体骨髓（BM）细胞治疗的安全性和可行性，不进行免疫抑制预处理。疗效评估是次要目标，采用临床残疾评定量表、多模态诱发电位（MMEP）记录和磁共振成像（MRI）扫描。收集细胞，过滤并静脉输注，患者耐受良好，未发生任何严重不良事件（ae）。在治疗后的12个月内，临床残疾评分显示无变化（扩展残疾状态评分，EDSS）或改善（MS影响量表-29，MSIS-29）， MMEPs显示神经生理改善。在治疗后3个月的时间里，核磁共振扫描没有显示任何显著的变化。在这一进展性疾病患者的小样本中，缺乏严重的不良反应和有益效果的提示，证明了进行更大规模的II/III期研究，以更全面地评估MS患者动员自体骨髓基质的疗效。
In this phase I study, we assessed the safety and feasibility of intravenous, autologous bone marrow (BM) cell therapy, without immunosuppressive preconditioning, in six patients with clinically definite, relapsing-progressive multiple sclerosis (MS). Assessment of efficacy was a secondary objective and employed clinical disability rating scales, multimodal evoked potential (MMEP) recordings, and magnetic resonance imaging (MRI) scans. Cells were harvested, filtered and infused intravenously in a day-case procedure that was well tolerated by patients and was not associated with any serious adverse events (AEs). Over a period of 12 months after the therapy, clinical disability scores showed either no change (Extended Disability Status Score, EDSS) or improvement (MS impact scale-29, MSIS-29), and MMEPs showed neurophysiological improvement. MRI scans did not show any significant changes over a post-therapy period of 3 months. The lack of serious adverse effects and the suggestion of a beneficial effect in this small sample of patients with progressive disease justify conducting a larger phase II/III study to make a fuller assessment of the efficacy of mobilization of autologous BM in patients with MS.