Transplantation of Mesenchymal Stromal Cells in Patients With Amyotrophic Lateral Sclerosis: Results of Phase I/IIa Clinical Trial

0477177 - ÚEM 2018 RIV US eng J - Článek v odborném periodiku
Syková, Eva - Rychmach, P. - Drahorádová, I. - Konrádová, Šimona - Růžičková, Kateřina - Voříšek, Ivan - Forostyak, Serhiy - Homola, A. - Bojar, M.
Transplantation of Mesenchymal Stromal Cells in Patients With Amyotrophic Lateral Sclerosis: Results of Phase I/IIa Clinical Trial.
Cell Transplantation. Roč. 26, č. 4 (2017), s. 647-658. ISSN 0963-6897. E-ISSN 1555-3892
Grant CEP: GA ČR(CZ) GA15-06958S; GA ČR(CZ) GBP304/12/G069; GA MŠMT(CZ) LM2015064
Institucionální podpora: RVO:68378041
Klíčová slova: clinical trial * cell-based therapy * stem cells
Obor OECD: Neurosciences (including psychophysiology
Impakt faktor: 2.885, rok: 2017

Amyotrophic lateral sclerosis (ALS) is a progressive untreatable neurodegenerative disorder, leading to the death of the cortical and spinal motoneurons (MNs). Bone marrow-derived mesenchymal stem/stromal cells (BM-MSCs) may represent a new approach to slowing down the progression of ALS by providing neurotrophic support to host MNs and by having an anti-inflammatory effect. We have designed a prospective, non-randomized, open-label clinical trial to assess the safety and efficacy of autologous multipotent BM-MSCs in ALS treatment. Patients received 15 mil. of BM-MSCs 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 (WSs) 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, no suspected serious adverse reactions 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-MSCs in ALS patients is a safe procedure and that it can slow down progression of the disease.
Trvalý link: http://hdl.handle.net/11104/0273554