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Journal of Neurorestoratology  2017, Vol. 5 Issue (1): 167-173    doi: 10.2147/JN.S138227
Original Research     
Feasibility of combined treatment for type III spinal muscular atrophy: a pilot study
Nataliia Sych1, Mariya Klunnyk1, Iryna Matiyashchuk1, Mariya Demchuk1, Olena Ivankova1, Andriy Sinelnyk1, Khrystyna Sorochynska2, Marina Skalozub3
1 Clinical Department;
2 Stem Cells Bank;
3 Laboratory and Biotechnology Department, Cell Therapy Center EmCell, Kyiv, Ukraine
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Abstract  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.

Key wordsfetal stem cells      forced expiratory volume per 1 second      forced vital capacity of the lungs      type III spinal muscular atrophy     
Received: 29 March 2017      Published: 26 December 2017
Corresponding Authors: Nataliia Sych, Clinical Department, Cell Therapy Center EmCell, 37 A Syretska str, 04073 Kyiv, Ukraine Tel +380 68 889 8989 Email infocenter@emcell.com   
Cite this article:

Nataliia Sych, Mariya Klunnyk, Iryna Matiyashchuk, Mariya Demchuk, Olena Ivankova, Andriy Sinelnyk, Khrystyna Sorochynska, Marina Skalozub. Feasibility of combined treatment for type III spinal muscular atrophy: a pilot study. Journal of Neurorestoratology, 2017, 5: 167-173.

URL:

http://jnr.tsinghuajournals.com/10.2147/JN.S138227     OR     http://jnr.tsinghuajournals.com/Y2017/V5/I1/167

1. Kazakov VM.Clinical molecular and genetic classification of muscular dystrophies (scientific review with comments).Neurol J. 2001;3(C):47-52.
2. Yevtushenko SK, Shaymurzin MR, Yevtushenko OS, et al.Early clinical and instrumental diagnostics and therapy for fast and slowly progressive muscular dystrophies and amyotrophias.Int Neurol J. 2007;4(14):8-12.
3. Monani UR, Lorson CL, Parsons DW, et al.A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.Hum Mol Genet. 1999;8(7):1177-1183.
4. Faravelli I, Nizzardo M, Comi GP, Corti S.Spinal muscular atrophy - recent therapeutic advances for an old challenge.Nat Rev Neurol. 2015;11(6):351-359.
5. Hoy SM.Nusinersen: first global statement.Drugs. 2017;77(4):473-479.
6. Gabanella F, Carissimi C, Usiello A, Pellizzoni L.The activity of the spinal muscular atrophy protein is regulated during development and cellular differentiation.Hum Mol Genet. 2005;14(23):3629-3642.
7. Sych N, Klunnik M, ivankova O, et al. Efficacy of fetal stem cells in Duchenne muscular dystrophy therapy.J Neurorestoratol. 2014;2:37-46.
8. Sych NS, Ivankova OV, Klunnyk MO, et al.Fetal stem cells are effective in the treatment of Grade I and II respiratory failure in amyotrophic lateral sclerosis and muscular dystrophy.Transl Neurosci Clin. 2015;1(1):10-16.
9. Bazarova MP.A guide to practical training in clinical laboratory. Kyiv: Vyshcha Shkola. 1981;312.
10. Miller MR, Hankinson J, Brusasco V, et al.Standardisation of spirometry.Eur Respir J. 2005;26(2):319-338.
11. Walczak P, Wojtkiewicz J, Nowakowski A, et al.Real-time MRI for precise and predictable intra-arterial stem cell delivery to the central nervous system.J Cereb Blood Flow Metab. 2017;37(7):2346-2356.
12. Illarioshkin SN, Ivanova-Smolenskaya IA, Markova ED.DNA-Diagnostics and Genetic Consulting in Neurology (Illarioshkin S.N.), 2002; 78-95.
13. Taylor PA, McElmurry RT, Lees CJ, Harrison DE, Blazar BR. Allogenic fetal liver cells have a distinct competitive engraftment advantage over adult bone marrow cells when infused into fetal as compared with adult severe combined immunodeficient recipients.Blood. 2002;99(5):1870-1872.
14. Kazakov VM.Clinical molecular genetic classification of muscular dystrophies (scientific summary with comments).J Neurol. 2001;3:7-12.
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