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KMID : 0359720090270020163
Journal of the Korean Neurological Association
2009 Volume.27 No. 2 p.163 ~ p.169
Efficacy and Safety of Autologous Bone Marrow-derived Mesenchymal Stem Cell Treatment in Patients With Amyotrophic Lateral Sclerosis
Kim Hyun-Young

Kim Seung-Hyun
CHoi Mi-Ran
Paek Jin-Young
Park Hee-Kyung
Yoon Hyun-Soo
Kim Kyung-Suk
Abstract
Background: This study was performed to investigate the efficacy and safety of intrathecal autologous bone marrowderived mesenchymal stem cells (MSCs) treatment for patients with ALS.

Methods: After a lead-in period for 3 months, 22 patients were treated with MSCs twice at an interval of 1 month. After initial MSCs injection, all patients were followed up for 3 months and their disease course, clinical characteristics were assessed. Disease status of patients were analyzed with ALS functional rating scale-revised (ALSFRS-R) for primary outcome measure, and additional clinical findings after treatment were all collected for secondary outcome measure and safety. Age and disease-duration matched patients with ALS were selected as a control group.

Results: During the follow-up period, MSCs treatment yielded a significant lesser change of ALSFRS-R score, compared to control group (1.54 vs 3.56, p<0.01). Moreover, the slop of decline of ALSFRS-R was significantly lower during the follow-up period, compared to the lead-in period in MSCs treatment group (2.68 vs 1.54, p=0.04), whereas the slopes during the two periods were not different in the control group (3.15 vs 3.56, p=0.37). MSCs treatment was well tolerated except for occurrences of transient headache, low back pain, and myalgia.

Conclusions: Our results show that intrathecal MSCs injection can slow disease progression and might be used as a disease modifying modality as an alternative treatment choice in patients with ALS.
KEYWORD
Autologous bone marrow-derived mesenchymal stem cell, Amyotrophic lateral sclerosis, Efficacy, Safety
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