Registration Number: 121

Conference Abstract Book: page XXXIII

Embryonic Stem Cells in Duchenne Muscular Dystrophy

Olexander I. Smikodub

Cell Therapy Clinic of National Medical University of Ukraine

 and Embryonic Tissues Center EmCell

Solomenskaya Str. 17, POB 80, Kiev 03110, UKRAINE

Under observation were 78 DMD patients aged 3-18.  Diagnosis was confirmed by the genetic analysis.  Treatment was initiated at different stages, with variable stages of muscular atrophy and ambulatory ability (bedridden, wheelchair-bound, ambulant). Patients were administered mesenchymal, ectodermal, and endodermal SC obtained from germ layers of 4-8 weeks old cadaverous embryos’ systems and organs; amounts administered - 0,5-3 ml, cell count - 0,1-100x10⁵/ml.  Our conclusions are based on 9 year’s observation experience of DMD patients. In the course of the first two months after the treatment, strength of different muscle groups increased by 100-700%, and was maintained at a high level for 8-15 months.  Besides, reported was also ROM increase, within the limits of the patient’s stage: stage II patients reported improved gait quality, ability to step on the heel and walk the stairs, raise from the floor with more confidence, and endure bigger loads during the day.  Within first two weeks of post-transplant period, noted was the increase of specific blood enzyme activity that by the end of 4-5^th week was markedly decreased or even normalized. Usually, this effect was maintained for 2-5 months. Observed were also decreased pseudohypertrophy and strain of forearms and calves, contractures of knee and ankle joints, myocardiopathy manifestations, and respiratory insufficiency, especially in stage IVa-IVb patients.  DMD patients require continuous treatment (at least, once every 6-8 months) aimed at aversion of muscular atrophy progression.  Termination of the treatment results in further progression of the disease and limitation of physical activity.  Transplantation of embryonic stem cells terminates DMD progression, notwithstanding the stage of the process, and results in marked improvements.  Reported was restoration of cell immunity indices and decrease of cytotoxic activity of mesenchymal cells.