J Korean Neurol Assoc.  2009 May;27(2):163-169.

Efficacy and Safety of Autologous Bone Marrow-derived Mesenchymal Stem Cell Treatment in Patients With Amyotrophic Lateral Sclerosis

• Kim HY
• Paek JY
• Park HK
• Choi MR
• Yoon HS
• Kim KS
• Kim SH

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

MeSH Terms

Amyotrophic Lateral Sclerosis
Disease Progression
Follow-Up Studies
Headache
Humans
Low Back Pain
Mesenchymal Stromal Cells
Outcome Assessment (Health Care)