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Page 53
Volume 10 Journal of Neurology & Neurophysiology
ISSN: 2155-9562
Neurology Congress 2019
Vascular Dementia Congress 2019
July 22-24, 2019
JOINT EVENT
conferenceseries.com
July 22-24, 2019 London, UK
&
12th International Conference on Vascular Dementia
32nd European Neurology Congress
Efficacy of stem cell therapy in ambulatory and non-ambulatory children with Duchenne muscular
dystrophy: Phase I–II
Alper Ibrahim Dai1, Osman Baspinar1, Ahmet Yeşilyurt2, Eda Sun3, Çigdem Inci Aydemir4, Olga Nehir Öztel4, Davut Unsal Capkan5, Ferda Pinarli2,
Abdullah Agar6 and Erdal Karaöz3, 4, 7
1Gaziantep University, Turkey
2Diskapi Yildirim Beyazit Research and Education Hospital, Turkey
3Istinye University, Center for Stem Cell Research and Application, Turkey
4Liv Hospital – Center for Regenerative Medicine and Stem Cell Research and Manufacturing, Turkey
5Deva Hospital, Turkey
6University of Travnik, Bosnia and Herzegovina
7Istinye University, Turkey
Purpose: Duchenne muscular dystrophy (DMD) is an X-linked recessive paediatric 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 Duchene muscular dystrophy.
Patients & Methods: Four ambulatory and five non-ambulatory 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.
Biography
Alper İbrahim Dai, graduated from Istanbul University, School of Medicine. He completed his paediatric residency at Jackson Memorial Hospital, University of
Miami / Florida and Children's hospital of West Virginia at Morgantown. As a fellowship he had 3 years of paediatric neurology at SUNY, Children's Hospital at
Buffalo / New York. He had 1 year of EEG / Epilepsy fellowship at Vanderbilt University, Nashville / Tennessee. He has been working at Division of Pediatrics
Neurology at Gaziantep University Hospital, Gaziantep / Turkey for 15 years. His major interests are, phase III clinical trial in anti-epileptic medications and phase
I and II clinical trials in stem cell therapy in children with neurological problems..
Alper İbrahim Dai et al., J Neurol Neurophysiol 2019, Volume 10