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Feasibility of combined treatment for type III spinal muscular atrophy: a pilot study

Authors Sych N, Klunnyk M, Matiyashchuk I, Demchuk M, Ivankova O, Sinelnyk A, Sorochynska K, Skalozub M

Received 29 March 2017

Accepted for publication 15 August 2017

Published 26 September 2017 Volume 2017:5 Pages 167—173


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Prof. Dr. Hongyun Huang

Nataliia Sych, 1 Mariya Klunnyk,1 Iryna Matiyashchuk,1 Mariya Demchuk,1 Olena Ivankova,1 Andriy Sinelnyk,1 Khrystyna Sorochynska,2 Marina Skalozub3

1Clinical Department, 2Stem Cells Bank, 3Laboratory and Biotechnology Department, Cell Therapy Center EmCell, Kyiv, Ukraine

Objective: To find solutions and improve methods of therapy for patients suffering from type III spinal muscular atrophy (SMA-III) by way of a combination of conventional treatment (medicines, physiotherapy) and fetal stem cells (FSCs) transplantation using suspensions extracted from stem cells of fetal liver and brain of human embryos.
Materials and methods: Our study included 7 children suffering from SMA-III, including 5 boys and 2 girls, aged from 3 to 12 years who were allocated into the main group (MG). SMA children in the MG were administered fetal stem cell preparations, mainly suspensions which contained fetal cells of human liver and fetal brain along with standard treatment (treatment by use of medicines if required and physiotherapy). The control group (CG) included 6 children – 3 boys and 3 girls aged from 3.5 to 13 years.
Results: The authors proved effectiveness and safety of treatment using FSCs for patients with SMA-III. Improvement of forced vital capacity of lungs and forced expiratory volume per 1 second was characteristic for the patients of the MG, starting from the period of observation at 6 months after FSCs transplantation. In patients from the CG, such values revealed characteristic improvement at 12 months after FSCs treatment. Significant reductions of alanina aminotransferase, aspartate aminotransferase, creatine phosphokinase, and lactate dehydrogenase ranges have been recorded in the patients of the MG at 6 months after treatment with FSCs. Such values show a significant reduction at 12 months after treatment.
Conclusion: FSCs use in complex treatment of patients with SMA results in stable disease compensation, improvement of laboratory results, and improved ranges of forced vital capacity and forced expiratory volume in 1 second.

Keywords: fetal stem cells, forced expiratory volume per 1 second, forced vital capacity of the lungs, type III spinal muscular atrophy

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