Safety and Efficacy of Allogeneic Adipose Tissue Mesenchymal Stromal Cells in Amyotrophic Lateral Sclerosis Patients, Single-Center, Prospective, Open-Label, Single-Arm Clinical Trial, Long-Term Follow-up

(Pages: 772-778)
Seyed Massood Nabavi, M.D, 1,#,*Shahedeh Karimi Karimi, M.D, 1,#Leila Arab, M.D., Ph.D, 1Leila Sanjari, M.D, 2Soura Mardpour, Ph.D, 1Vajiheh Azimian, Ph.D, 1,3Neda Jarughi, M.Sc, 1Azadeh Ghaheri, Ph.D, 4Seyedeh-Esmat Hosseini, Ph.D, 1,5Nasser Aghdami, M.D., Ph.D, 1Massoud Vosough, M.D., Ph.D, 1,*
Department of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, Tehran, Iran
International Medicine Department, Mostafa Khomeini Medical Center, Shahed University, Tehran, Iran
Core Research Facilities (CRF), Isfahan University of Medical Sciences, Isfahan, Iran
Reproductive Epidemiology Research Center, Royan Institute for Reproductive Biomedicine, ACECR, Tehran, Iran
Nursing Care Research Center, School of Nursing and Midwifery, Iran University of Medical Science, Tehran, Iran
Department of Regenerative Medicine, Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, Tehran, Iran
International Medicine Department, Mostafa Khomeini Medical Center, Shahed University, Tehran, Iran
Core Research Facilities (CRF), Isfahan University of Medical Sciences, Isfahan, Iran
Reproductive Epidemiology Research Center, Royan Institute for Reproductive Biomedicine, ACECR, Tehran, Iran
Nursing Care Research Center, School of Nursing and Midwifery, Iran University of Medical Science, Tehran, Iran
*Corresponding Address: P.O.Box: 1665664511 Department of Regenerative Medicine Cell Science Research Center Royan Institute for Stem Cell Biology and Technology Tehran Iran Emails:seyedmassoodnabavi@gmail.com,masvos@yahoo.com

# These authors contributed equally to this work.

The Cell Journal (Yakhteh) is an open access journal which means the articles are freely available online for any individual author to download and use the providing address. 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.
Nabavi Seyed Massood, Karimi Shahedeh Karimi, Arab Leila, Sanjari Leila, Mardpour Soura, Azimian Vajiheh, Jarughi Neda, Ghaheri Azadeh, Hosseini Seyedeh-Esmat, Aghdami Nasser, Vosough Massoud. Safety and Efficacy of Allogeneic Adipose Tissue Mesenchymal Stromal Cells in Amyotrophic Lateral Sclerosis Patients, Single-Center, Prospective, Open-Label, Single-Arm Clinical Trial, Long-Term Follow-up . Cell J. 2021; 23(7): 772-778.

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder with very limited treatment options. Stem cells have been raised as a new treatment modality for these patients. We have designed a single-center, prospective, open-label, and single arm clinical trial to assess the safety, feasibility, and rather efficacy of administrating allogeneic adipose-derived mesenchymal stromal cells (Ad-MSCs) in ALS patients. We enrolled 17 patients with confirmed ALS diagnosis with ALS Functional Rating Scale-Revised (ALSFRS-R) (<24) and predicted forced vital capacity (FVC) (<40)%. Allogeneic Ad-MSCs were transplanted intravenously for all patients. Follow-ups were done at 24 hours, 2, 4, 6, and 12 months after cell infusion by checking adverse events, laboratory tests, and clinically by ALSFRS-R and FVC. Patients were also followed five years later and ALSFRS-R score was recorded in the survived individuals. There was no report of severe adverse events related to cell infusion. Two patients experienced dyspnea and chest pain 36 and 65 days after cell infusion due to pulmonary emboli. The progressive decrease in ALSFRS-R and FVC levels was recorded and three patients died in the first year. During five years follow up, despite a notable decrease in functional scores, 5 patients survived. Intravenous (IV) infusion of allogeneic Ad-MSCs in ALS patients is safe and feasible. The survival rate of the patients is more than IV autologous MSCs (Registration number: IRCT20080728001031N26).