Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosisopen access
- Authors
- Oh, Ki-Wook; Noh, Min-Young; Kwon, Min-Soo; Kim, Hyun Young; Oh, Seong-il; Park, Jinseok; Kim, Hee-Jin; Ki, Chang-Seok; Kim, Seung Hyun
- Issue Date
- Sep-2018
- Publisher
- WILEY
- Citation
- ANNALS OF NEUROLOGY, v.84, no.3, pp.361 - 373
- Indexed
- SCIE
SCOPUS
- Journal Title
- ANNALS OF NEUROLOGY
- Volume
- 84
- Number
- 3
- Start Page
- 361
- End Page
- 373
- URI
- https://scholarworks.bwise.kr/hanyang/handle/2021.sw.hanyang/149393
- DOI
- 10.1002/ana.25302
- ISSN
- 0364-5134
- 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
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