Safety and efficacy of bone marrow derived-mesenchymal stem cells transplantation in patients with amyotrophic lateral sclerosis.
| Autor: | Tavakol-Afshari J; Immunology Research Center, Mashhad University of Medical Sciences, Mashhad, Iran., Boroumand AR; Neuroscience Research Center, Mashhad University of Medical Sciences, Mashhad, Iran., Farkhad NK; Immunology Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.; Student Research Committee, Mashhad University of Medical Sciences, Mashhad, Iran., Adhami Moghadam A; Department of Internal Medicine and Critical Care, Islamic Azad University, Mashhad, Iran.; Specialty of Internal Medicine and Critical Care, Head of Army Hospital ICU and Intensive, Iran., Sahab-Negah S; Neuroscience Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.; Shefa Neuroscience Research Center, Khatam Alanbia Hospital, Tehran, Iran., Gorji A; Neuroscience Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.; Shefa Neuroscience Research Center, Khatam Alanbia Hospital, Tehran, Iran.; Department of Neurosurgery, Westfälische Wilhelms-Universität Münster, Münster, Germany.; Department of Neurology, Westfälische Wilhelms-Universität Münster, Münster, Germany.; Epilepsy Research Center, Westfälische Wilhelms-Universität Münster, Münster, Germany. |
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| Jazyk: | angličtina |
| Zdroj: | Regenerative therapy [Regen Ther] 2021 Aug 16; Vol. 18, pp. 268-274. Date of Electronic Publication: 2021 Aug 16 (Print Publication: 2021). |
| DOI: | 10.1016/j.reth.2021.07.006 |
| Abstrakt: | Stem cell-based treatments have emerged as potentially effective approaches to delay the progression of amyotrophic lateral sclerosis (ALS). This study was designed as a single-center, prospective, and open-label study without a placebo control group to assess the safety and efficacy of concurrent intrathecal (IT) and intravenous (IV) administration of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs) in patients with ALS. Autologous BM-MSCs were isolated and expanded under standard conditions. Fifteen patients were neurologically examined before BM-MSCs transplantation (1 × 10 6 cells/kg BW) to evaluate the rate of pre-treatment disease progression. To assess the safety and efficacy, patients were examined at 1, 3, and 6 months following the treatment with BM-MSCs. Adverse reactions were assessed, and the clinical outcome was determined by the evaluation of the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC). No serious adverse reaction was observed after combined IT and IV administration of BM-MSCs. The mean ALSFRS-R and FVC values remained stable during the first 3 months of the treatment. However, a significant reduction in ALSFRS-R and FVC levels was observed in these patients 6 months after BM-MSCs administration. Our study revealed that the concurrent IT and IV application of BM-MSCs in patients with ALS is a safe procedure. Furthermore, our data indicate a temporary delay in the progression of ALS after a single combined IT and IV administration of BM-MSCs. Further studies are required to explore if the repeated applications of BM-MSCs could prolong survival and delay the progression of ALS. Competing Interests: N.K.F, A.G, and S.SN declare that they have no competing interests. J.T.A, A.R.B, and A.A have received research grants from Parnia Stem Cell Institute. (© 2021 The Japanese Society for Regenerative Medicine. Production and hosting by Elsevier B.V.) |
| Databáze: | MEDLINE |
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