| 근위축성측삭경화증 환자에서 자가 골수유래
중간엽줄기세포치료의 유효성 및 안전성 |
| 김현영, 백진영a 박희경 최미란b 윤현수c 김경숙a 김승현 |
| 한양대학교 의과대학 신경과학교실, 코아스템a, 한양대학교 과학기술대학 분자생물과학부b,
한양대학교 의과대학 해부세포생물학교실 |
| Efficacy and Safety of Autologous Bone Marrow-derived Mesenchymal Stem Cell Treatment in Patients With Amyotrophic Lateral Sclerosis |
| Hyun Young Kim |
| Department of Neurology, College of Medicine, Hanyang University Hospital, Seoul, Korea; Bioengineering Institute,
CoreStem Inc., Seoul, Koreaa; Division of Molecular and Life Sciences, Hanyang University, Ansan, Koreab; Department
of Anatomy and Cell Biology, College of Medicine, Hanyang University, Seoul, Koreac |
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| 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. Key Words: Autologous bone marrow-derived mesenchymal stem cell, Amyotrophic lateral sclerosis, Efficacy, Safety |
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