Results of Phase I/IIa clinical trial: Amyotrophic lateral sclerosis (ALS)

The results of our clinical trial trial AMSC-ALS-001 “Safety and efficacy of autologous bone marrow mesenchymal cells (BM-MSC) in treatment of amyotrophic lateral sclerosis. Prospective, non-randomized, open-label study.” (EudraCT No. 2011-000362-35) have been published in Cell Transplantation journal (

Autologous BM-MSC were isolated and expanded under GMP conditions. Patients received 15±4.5 x 106 of BM-MSC via lumbar puncture into the cerebrospinal fluid. Patients were monitored for 6 months before treatment, and then for an 18 month follow-up period. Potential adverse reactions were assessed, and the clinical outcome was evaluated by the ALS functional rating scale (ALSFRS), forced vital capacity (FVC), and weakness scales (WS), to assess muscle strength on the lower and upper extremities. In total, 26 patients were enrolled in the study and were assessed for safety; 23 patients were suitable for efficacy evaluation. After intrathecal BM-MSC application, about 30% of the patients experienced a mild-to-moderate headache, resembling the headaches after a standard lumbar puncture. No suspected serious adverse reactions (SUSAR) were observed. We found a reduction in ALSFRS decline at 3 months after application (p<0.02) that, in some cases, persisted for 6 months (p<0.05). In about 80% of the patients, FVC values remained stable or above 70% for a time period of 9 months. Values of WS were stable in 75% of patients at 3 months after application. Our results demonstrate that the intrathecal application of BM-MSC in ALS patients is a safe procedure, and that it can slow down progression of the disease.

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