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Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy – Phase I–II

Authors Dai A, Baspinar O, Yeşilyurt A, Sun E, İnci Aydemir C, Öztel ON, Capkan DU, Pinarli F, Agar A, Karaöz E

Received 21 April 2018

Accepted for publication 20 July 2018

Published 26 October 2018 Volume 2018:8 Pages 63—77

DOI https://doi.org/10.2147/DNND.S170087

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Colin Mak

Peer reviewer comments 2

Editor who approved publication: Prof. Dr. Thomas Müller


Alper Dai,1 Osman Baspinar,2 Ahmet Yeşilyurt,3 Eda Sun,4 Çiğdem İnci Aydemir,5 Olga Nehir Öztel,5 Davut Unsal Capkan,6 Ferda Pinarli,3 Abdullah Agar,7 Erdal Karaöz4,5,8

1Gaziantep University Medical Faculty, Pediatric Neurology Department, Gaziantep, Turkey; 2Gaziantep University Medical Faculty, Pediatric Cardiology Department, Gaziantep, Turkey; 3Diskapi Yildirim Beyazit Research and Education Hospital, University of Health Sciences, Center for Genetic Diagnosis, Ankara, Turkey; 4İstinye University, Center for Stem Cell Research and Application, İstanbul, Turkey; 5Liv Hospital – Center for Regenerative Medicine and Stem Cell Research and Manufacturing, İstanbul, Turkey; 6Deva Hospital, Department of Radiology, Gaziantep, Turkey; 7University of Travnik, Travnik, Bosnia and Herzegovina; 8İstinye University, Medical Faculty, Histology and Embryology Department, İstanbul, Turkey

Purpose: Duchenne muscular dystrophy (DMD) is an X-linked recessive pediatric disorder that ultimately leads to progressive muscle degeneration. It has been known that cell-based therapies were used to promote muscle regeneration. The main purpose of this study was to investigate the effects of allogeneic Wharton jelly-derived mesenchymal stem cells therapy in Duchenne muscular dystrophy.
Patients and methods:
Four ambulatory and five nonambulatory male patients were assessed as having acceptance criteria. Gene expression and immunohistochemical analysis were performed for dystrophin gene expression. The fluorescent in situ hybridization method was used for detection of chimerism and donor–recipient compatibility. Complement dependent lymphocytotoxic crossmatch test and detection of panel reactive antigen were performed. All patients were treated with 2 × 106 cells/kg dose of allogeneic Wharton jelly-derived mesenchymal stem cells via intra-arterial and intramuscular administration. Stability was maintained in patient follow-up tests, which are respiratory capacity tests, cardiac measurements, and muscle strength tests.
Results:
The vastus intermedius muscle was observed in one patient with MRI. Chimerism was detected by fluorescent in situ hybridization and mean gene expression was increased to 3.3-fold. An increase in muscle strength measurements and pulmonary function tests was detected. Additionally, we observed two of nine patients with positive panel reactive antigen result.
Conclusion: All our procedures are well tolerated, and we have not seen any application-related complications so far. Our main purpose of this study was to investigate the effects of allogeneic mesenchymal stem cell therapy and determine its suitability and safety as a form of treatment in this untreatable disorder.

Keywords: Duchenne muscular dystrophy, mesenchymal stem cells, panel reactive antigen, treatment

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