Open-Label Study of Autologous Bone-Marrow-Derived Mesenchymal Stem Cells in Multiple Sclerosis (P3.279)

OBJECTIVE: Primary: feasibility and safety of intravenously administered bone marrow-derived autologous mesenchymal stem cells (MSCs) at a dose of up to 2,000,000 cells/kg over 12 months by recording the adverse effects (AEs). Secondary: clinical and radiological efficacy. DESIGN/METHODS: We isolated, expanded and administered MSCs to five multiple sclerosis patients (MS). Four patients received only one infusion and one patient received two. Mean dose: 1.6×106 cells per kg of bodyweight (range 1.1-2.0). Analysis variables: 1) AEs during 12 months after infusion. 2) Clinical activity: relapses and disability progression (EDSS) in the 12 months before treatment. 3) Radiological activity: gadolinium enhancing lesion (GEL) on brain MRI during 3 months before bone marrow harvest. This activity was compared with activity 12 months after the infusion. The procedure was approved by AEMPS (Spanish Drug and Health Products Agency) for compassionate use. Patients signed an informed consent. RESULTS: Baseline characteristics: Five patients were analized: three relapsing-remitting MS (RRMS), two secondary progressive MS (SPMS). Mean duration of disease was greater than 10 years in all patients. Two patients with RRMS had never been treated with immunomodulatory treatment. 2 patients with RRMS had relapses 12 months before MSCs infusion, both of whom also had GEL in brain MRI before treatment. EDSS remained stable in previous year. Primary outcomes: no patient developed AEs after treatment and no AEs have been recorded to date. There was no problem for the MSCs obtention. Secondary outcome: in all patients the disability was stable. The patients without clinical neither radiological activity did not show any relapses neither GEL lesions after the treatment. However, the patients with disease activity pre-treatment continued with activity after the treatment. CONCLUSIONS: Our results support the safety and feasibility of MSCs therapy. Although the disability was stable, the treatment wasn’t effective for the control of the inflammatory activity. Disclosure: Dr. Ramo has received research support from Biogen Idec. Dr. Hervas has nothing to disclose. Dr. Andreu has nothing to disclose. Dr. Prosper has nothing to disclose. Dr. Inoges has nothing to disclose. Dr. Kulisevsky has received personal compensation for activities with Merck Serono as a scientific advisory board participant. Dr. Belvis has nothing to disclose.