Efficacy of fetal stem cells in Duchenne muscular dystrophy therapy

Abstract

Efficacy of fetal stem cells in Duchenne muscular dystrophy therapy Nataliia Sych,1 Mariya Klunnik,1 Olena Ivankova,1 Irina Matyaschuk,1 Mariya Demchuk,1 Alla Novytska,1 Inna Arkhipenko,2 Iuliia Shalita,2 Dario Siniscalco31Clinical Department, 2Biotechnology Department, Cell Therapy Center EmCell, Kiev, Ukraine; 3Biomedical Centre for Autism Research and Therapy, Bari, ItalyAbstract: The absence of effective treatment methods for Duchenne muscular dystrophy (DMD) calls for new therapeutic approaches. One of the promising treatment methods for DMD is stem cell therapy. This study demonstrates the impact of fetal stem cells (FSCs) on functional capacity and life quality of DMD patients and the ability of FSCs to prevent DMD-related complications in order to inhibit the disease progression. FSC therapy substantially improves functional capacity, life quality, left ventricular ejection fraction, and forced vital capacity of the lungs of DMD patients; this was confirmed by comparison of neurological, laboratory (aspartate aminotransferase, alanine aminotransferase, creatine phosphokinase, and lactate dehydrogenase), and morphofunctional findings (left ventricular ejection fraction and forced vital capacity) in DMD patients before the treatment, and 6 and 12 months after the FSC treatment.Keywords: life quality, heart failure, respiratory failure, cell therapy