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Safety, Feasibility of Intravenous and Intrathecal Injection of Autologous Bone Marrow Derived Mesenchymal Stromal Cells in Patients with Amyotrophic Lateral Sclerosis: An Open Label Phase I Clinical Trial
OBJECTIVE: Amyotrophic lateral sclerosis (ALS) is the most severe disorder within the spectrum of motor neuron diseases (MND) that has no effective treatment and a progressively fatal outcome. We have conducted two clinical trials to assess the safety and feasibility of intravenous (IV) and intrathe...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Royan Institute
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6099146/ https://www.ncbi.nlm.nih.gov/pubmed/30124008 http://dx.doi.org/10.22074/cellj.2019.5370 |
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author | Nabavi, Seyed Massood Arab, Leila Jarooghi, Neda Bolurieh, Tina Abbasi, Fatemeh Mardpour, Soura Azimyian, Vajihe Moeininia, Fatemeh Maroufizadeh, Saman Sanjari, Leila Hosseini, Seyedeh Esmat Aghdami, Nasser |
author_facet | Nabavi, Seyed Massood Arab, Leila Jarooghi, Neda Bolurieh, Tina Abbasi, Fatemeh Mardpour, Soura Azimyian, Vajihe Moeininia, Fatemeh Maroufizadeh, Saman Sanjari, Leila Hosseini, Seyedeh Esmat Aghdami, Nasser |
author_sort | Nabavi, Seyed Massood |
collection | PubMed |
description | OBJECTIVE: Amyotrophic lateral sclerosis (ALS) is the most severe disorder within the spectrum of motor neuron diseases (MND) that has no effective treatment and a progressively fatal outcome. We have conducted two clinical trials to assess the safety and feasibility of intravenous (IV) and intrathecal (IT) injections of bone marrow derived mesenchymal stromal cells (BM-MSCs) in patients with ALS. MATERIALS AND METHODS: This is an interventional/experimental study. We enrolled 14 patients that met the following inclusion criteria: definitive diagnosis of sporadic ALS, ALS Functional Rating Scale (ALS-FRS) ≥24, and ≥40% predicted forced vital capacity (FVC). All patients underwent bone marrow (BM) aspiration to obtain an adequate sample for cell isolation and culture. Patients in group 1 (n=6) received an IV and patients in group 2 (n=8) received an IT injection of the cell suspension. All patients in both groups were followed at 24 hours and 2, 4, 6, and 12 months after the injection with ALS-FRS, FVC, laboratory tests, check list of side effects and brain/spinal cord magnetic resonance imaging (MRI). In each group, one patient was lost to follow up one month after cell injection and one patient from IV group died due to severe respiratory insufficiency and infection. RESULTS: During the follow up there were no reports of adverse events in terms of clinical and laboratory assessments. In MRI, there was not any new abnormal finding. The ALS-FRS score and FVC percentage significantly reduced in all patients from both groups. CONCLUSION: This study has shown that IV and IT transplantation of BM-derived stromal cells is safe and feasible (Registration numbers: NCT01759797 and NCT01771640). |
format | Online Article Text |
id | pubmed-6099146 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | Royan Institute |
record_format | MEDLINE/PubMed |
spelling | pubmed-60991462019-01-01 Safety, Feasibility of Intravenous and Intrathecal Injection of Autologous Bone Marrow Derived Mesenchymal Stromal Cells in Patients with Amyotrophic Lateral Sclerosis: An Open Label Phase I Clinical Trial Nabavi, Seyed Massood Arab, Leila Jarooghi, Neda Bolurieh, Tina Abbasi, Fatemeh Mardpour, Soura Azimyian, Vajihe Moeininia, Fatemeh Maroufizadeh, Saman Sanjari, Leila Hosseini, Seyedeh Esmat Aghdami, Nasser Cell J Original Article OBJECTIVE: Amyotrophic lateral sclerosis (ALS) is the most severe disorder within the spectrum of motor neuron diseases (MND) that has no effective treatment and a progressively fatal outcome. We have conducted two clinical trials to assess the safety and feasibility of intravenous (IV) and intrathecal (IT) injections of bone marrow derived mesenchymal stromal cells (BM-MSCs) in patients with ALS. MATERIALS AND METHODS: This is an interventional/experimental study. We enrolled 14 patients that met the following inclusion criteria: definitive diagnosis of sporadic ALS, ALS Functional Rating Scale (ALS-FRS) ≥24, and ≥40% predicted forced vital capacity (FVC). All patients underwent bone marrow (BM) aspiration to obtain an adequate sample for cell isolation and culture. Patients in group 1 (n=6) received an IV and patients in group 2 (n=8) received an IT injection of the cell suspension. All patients in both groups were followed at 24 hours and 2, 4, 6, and 12 months after the injection with ALS-FRS, FVC, laboratory tests, check list of side effects and brain/spinal cord magnetic resonance imaging (MRI). In each group, one patient was lost to follow up one month after cell injection and one patient from IV group died due to severe respiratory insufficiency and infection. RESULTS: During the follow up there were no reports of adverse events in terms of clinical and laboratory assessments. In MRI, there was not any new abnormal finding. The ALS-FRS score and FVC percentage significantly reduced in all patients from both groups. CONCLUSION: This study has shown that IV and IT transplantation of BM-derived stromal cells is safe and feasible (Registration numbers: NCT01759797 and NCT01771640). Royan Institute 2019 2018-08-07 /pmc/articles/PMC6099146/ /pubmed/30124008 http://dx.doi.org/10.22074/cellj.2019.5370 Text en Any use, distribution, reproduction or abstract of this publication in any medium, with the exception of commercial purposes, is permitted provided the original work is properly cited http://creativecommons.org/licenses/by/2.5/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Original Article Nabavi, Seyed Massood Arab, Leila Jarooghi, Neda Bolurieh, Tina Abbasi, Fatemeh Mardpour, Soura Azimyian, Vajihe Moeininia, Fatemeh Maroufizadeh, Saman Sanjari, Leila Hosseini, Seyedeh Esmat Aghdami, Nasser Safety, Feasibility of Intravenous and Intrathecal Injection of Autologous Bone Marrow Derived Mesenchymal Stromal Cells in Patients with Amyotrophic Lateral Sclerosis: An Open Label Phase I Clinical Trial |
title | Safety, Feasibility of Intravenous and Intrathecal Injection of
Autologous Bone Marrow Derived Mesenchymal Stromal Cells
in Patients with Amyotrophic Lateral Sclerosis: An Open
Label Phase I Clinical Trial |
title_full | Safety, Feasibility of Intravenous and Intrathecal Injection of
Autologous Bone Marrow Derived Mesenchymal Stromal Cells
in Patients with Amyotrophic Lateral Sclerosis: An Open
Label Phase I Clinical Trial |
title_fullStr | Safety, Feasibility of Intravenous and Intrathecal Injection of
Autologous Bone Marrow Derived Mesenchymal Stromal Cells
in Patients with Amyotrophic Lateral Sclerosis: An Open
Label Phase I Clinical Trial |
title_full_unstemmed | Safety, Feasibility of Intravenous and Intrathecal Injection of
Autologous Bone Marrow Derived Mesenchymal Stromal Cells
in Patients with Amyotrophic Lateral Sclerosis: An Open
Label Phase I Clinical Trial |
title_short | Safety, Feasibility of Intravenous and Intrathecal Injection of
Autologous Bone Marrow Derived Mesenchymal Stromal Cells
in Patients with Amyotrophic Lateral Sclerosis: An Open
Label Phase I Clinical Trial |
title_sort | safety, feasibility of intravenous and intrathecal injection of
autologous bone marrow derived mesenchymal stromal cells
in patients with amyotrophic lateral sclerosis: an open
label phase i clinical trial |
topic | Original Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6099146/ https://www.ncbi.nlm.nih.gov/pubmed/30124008 http://dx.doi.org/10.22074/cellj.2019.5370 |
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