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...
| Main Authors: | , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Royan Institute (ACECR), Tehran
2018-08-01
|
| Series: | Cell Journal |
| Subjects: | |
| Online Access: | https://celljournal.org/journal/article/abstract/5370 |
| _version_ | 1818855323121745920 |
|---|---|
| author | Seyed Massood Nabavi Leila Arab Neda Jarooghi Tina Bolurieh Fatemeh Abbasi Soura Mardpour Vajihe Azimyian Fatemeh Moeininia Saman Maroufizadeh Leila Sanjari Seyedeh Esmat Hosseini Nasser Aghdami |
| author_facet | Seyed Massood Nabavi Leila Arab Neda Jarooghi Tina Bolurieh Fatemeh Abbasi Soura Mardpour Vajihe Azimyian Fatemeh Moeininia Saman Maroufizadeh Leila Sanjari Seyedeh Esmat Hosseini Nasser Aghdami |
| author_sort | Seyed Massood Nabavi |
| collection | DOAJ |
| 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). |
| first_indexed | 2024-12-19T08:06:46Z |
| format | Article |
| id | doaj.art-50ec4948c1c74604a9410f122679006c |
| institution | Directory Open Access Journal |
| issn | 2228-5806 2228-5814 |
| language | English |
| last_indexed | 2024-12-19T08:06:46Z |
| publishDate | 2018-08-01 |
| publisher | Royan Institute (ACECR), Tehran |
| record_format | Article |
| series | Cell Journal |
| spelling | doaj.art-50ec4948c1c74604a9410f122679006c2022-12-21T20:29:44ZengRoyan Institute (ACECR), TehranCell Journal2228-58062228-58142018-08-0120459259810.22074/cellj.2019.5370Safety, 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 TrialSeyed Massood Nabavi0Leila Arab1Neda Jarooghi2Tina Bolurieh3Fatemeh Abbasi4Soura Mardpour5Vajihe Azimyian6Fatemeh Moeininia7Saman Maroufizadeh8Leila Sanjari9Seyedeh Esmat Hosseini10Nasser Aghdami11Department of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Neuroscience, School of Advanced Technologies in Medicine, Tehran University of Medical Science, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranDepartment of Epidemiology and Reproductive Health, Reproductive Epidemiology Research Center, Royan Institute for Reproductive Medicine, ACECR, Tehran, IranIntensive Care Unit, Mostafa Khomeini Hospital, Tehran, IranStudent Research Committee, School of Nursing and Midwifery, Shahid Beheshti University of Medical Sciences, Tehran, IranDepartment of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, IranObjective 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).https://celljournal.org/journal/article/abstract/5370Amyotrophic Lateral SclerosisBone MarrowIntrathecalIntravenousMesenchymal Stromal Cell |
| spellingShingle | Seyed Massood Nabavi Leila Arab Neda Jarooghi Tina Bolurieh Fatemeh Abbasi Soura Mardpour Vajihe Azimyian Fatemeh Moeininia Saman Maroufizadeh Leila Sanjari Seyedeh Esmat Hosseini Nasser Aghdami 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 Cell Journal Amyotrophic Lateral Sclerosis Bone Marrow Intrathecal Intravenous Mesenchymal Stromal Cell |
| 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 | Amyotrophic Lateral Sclerosis Bone Marrow Intrathecal Intravenous Mesenchymal Stromal Cell |
| url | https://celljournal.org/journal/article/abstract/5370 |
| work_keys_str_mv | AT seyedmassoodnabavi safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT leilaarab safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT nedajarooghi safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT tinabolurieh safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT fatemehabbasi safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT souramardpour safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT vajiheazimyian safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT fatemehmoeininia safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT samanmaroufizadeh safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT leilasanjari safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT seyedehesmathosseini safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial AT nasseraghdami safetyfeasibilityofintravenousandintrathecalinjectionofautologousbonemarrowderivedmesenchymalstromalcellsinpatientswithamyotrophiclateralsclerosisanopenlabelphaseiclinicaltrial |