Striking New Stem Cell Data in MS and ALS

Laurie Barclay, MD


November 02, 2010

Safety and Immunological Effects of Mesenchymal Stem Cell Transplantation in Patients with Multiple Sclerosis and Amyotrophic Lateral Sclerosis

Karussis D, Karageorgiou C, Vaknin-Dembinsky A, et al.
Arch Neurol. 2010;67:1187-1194


The goal of this phase 1/2 open-safety clinical trial, which included 15 patients with multiple sclerosis (MS) and 19 patients with amyotrophic lateral sclerosis (ALS), was to assess the feasibility, safety, and immunologic activity of autologous mesenchymal stem cells (MSCs) given intrathecally and intravenously. Patients with MS had a mean Expanded Disability Status Scale (EDSS) score of 6.7 ± 1.0, and those with ALS had a mean ALS Functional Rating Scale (ALSFRS) score of 20.8 ± 8.0.

MSCs were obtained from bone marrow aspiration, were cultured, and were injected intrathecally by means of lumbar puncture in 34 patients and intravenously in 14 patients (mean dose of 63.2 x 10 6 ± 2.5 x 10 6). To locate and follow the migration of MSCs within the brain using magnetic resonance imaging (MRI), 9 patients received MSCs magnetically labeled with the superparamagnetic iron oxide ferumoxides (Feridex IV®).

Although no major adverse effects were reported during the 25-month follow-up, 21 patients had injection-related adverse effects consisting of transient fever, and 15 had headache, mainly related to lumbar puncture. During the first 6 months of observation, the ALS group had a stable mean ALSFRS score, whereas the MS group had an improvement in mean EDSS score from 6.7 ± 1.0 to 5.9 ±1.6.

MRI detected MSCs in the occipital horns of the ventricles, suggesting migration of ferumoxides-labeled MSCs in the meninges, subarachnoid space, and spinal cord. In 20 patients who had follow-up MRI 1 year after transplantation, no unexpected pathology or significant new disease activity was observed.

At 24 hours after MSC transplantation, immunologic testing showed an increase in the proportion of CD4+/CD25+ regulatory T cells, a decrease in lymphocytic proliferative responses, and expression of CD40+, CD83+, CD86+, and HLA-DR on myeloid dendritic cells.


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