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Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis

Title
Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
Author
오기욱
Keywords
STROMAL CELLS; ALS; TRANSPLANTATION; MICROGLIA; CRITERIA; SAFETY; TRIAL
Issue Date
2018-09
Publisher
WILEY
Citation
ANNALS OF NEUROLOGY, v. 84, no. 3, page. 361-373
Abstract
ObjectiveTo assess the safety and efficacy of 2 repeated intrathecal injections of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs) in amyotrophic lateral sclerosis (ALS).MethodsIn a phase 2 randomized controlled trial (NCT01363401), 64 participants with ALS were randomly assigned treatments (1:1) of riluzole alone (control group, n = 31) or combined with 2 BM-MSC injections (MSC group, n = 33). Safety was assessed based on the occurrence of adverse events. The primary efficacy outcome was changes in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score from baseline to 4 and 6 months postinjection. Post hoc analysis includes investigation of cerebrospinal fluid biomarkers and long-term survival analysis.ResultsSafety rating showed no groupwise difference with absence of serious treatment-related adverse events. Mean changes in ALSFRS-R scores from baseline to 4 and 6 months postinjection were reduced in the MSC group compared with the control group (4 months: 2.98, 95% confidence interval [CI] = 1.48-4.47, p < 0.001; 6 months: 3.38, 95% CI = 1.23-5.54, p = 0.003). The MSC group showed decreased proinflammatory and increased anti-inflammatory cytokines. In good responders, transforming growth factor 1 significantly showed inverse correlation with monocyte chemoattractant protein-1. There was no significant difference in long-term survival between groups.InterpretationRepeated intrathecal injections of BM-MSCs demonstrated a possible clinical benefit lasting at least 6 months, with safety, in ALS patients. A plausible action mechanism is that BM-MSCs mediate switching from pro- to anti-inflammatory conditions. A future randomized, double-blind, large-scale phase 3 clinical trial with additional BM-MSC treatments is required to evaluate long-term efficacy and safety. Ann Neurol 2018;84:361-373
URI
https://onlinelibrary.wiley.com/doi/full/10.1002/ana.25302https://repository.hanyang.ac.kr/handle/20.500.11754/119951
ISSN
0364-5134; 1531-8249
DOI
10.1002/ana.25302
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COLLEGE OF MEDICINE[S](의과대학) > MEDICINE(의학과) > Articles
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