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Transplantation of Mesenchymal Stromal Cells in Patients With Amyotrophic Lateral Sclerosis: Results of Phase I/IIa Clinical Trial
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SYSNO ASEP 0477177 Druh ASEP J - Článek v odborném periodiku Zařazení RIV J - Článek v odborném periodiku Poddruh J Článek ve WOS Název Transplantation of Mesenchymal Stromal Cells in Patients With Amyotrophic Lateral Sclerosis: Results of Phase I/IIa Clinical Trial Tvůrce(i) Syková, Eva (UEM-P) RID
Rychmach, P. (CZ)
Drahorádová, I. (CZ)
Konrádová, Šimona (UEM-P)
Růžičková, Kateřina (UEM-P)
Voříšek, Ivan (UEM-P)
Forostyak, Serhiy (UEM-P) RID, ORCID
Homola, A. (CZ)
Bojar, M. (CZ)Zdroj.dok. Cell Transplantation. - : Sage - ISSN 0963-6897
Roč. 26, č. 4 (2017), s. 647-658Poč.str. 12 s. Jazyk dok. eng - angličtina Země vyd. US - Spojené státy americké Klíč. slova clinical trial ; cell-based therapy ; stem cells Vědní obor RIV FH - Neurologie, neurochirurgie, neurovědy Obor OECD Neurosciences (including psychophysiology CEP GA15-06958S GA ČR - Grantová agentura ČR GBP304/12/G069 GA ČR - Grantová agentura ČR LM2015064 GA MŠMT - Ministerstvo školství, mládeže a tělovýchovy Institucionální podpora UEM-P - RVO:68378041 UT WOS 000400124700010 EID SCOPUS 85017589027 DOI 10.3727/096368916X693716 Anotace 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. Pracoviště Ústav experimentální medicíny Kontakt Lenka Koželská, lenka.kozelska@iem.cas.cz, Tel.: 241 062 218, 296 442 218 Rok sběru 2018
Počet záznamů: 1