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Embryonic Stem Cells in Treatment of Duchenne Muscular Dystrophy
Embryonic Stem Cells in Treatment of Duchenne Muscular Dystrophy
A.I Smikodub, Cell Therapy Clinic of National Medical University and Embryonic Tissues Center EmCell, Kiev, Ukraine
Observed are 78 DMD patients aged 3-18, 3 of whom are girls. In all cases diagnosis was confirmed at genetic level. Treatment was started at different stages of the disease, with variable stages of muscle atrophy and ambulatory ability (bedridden, wheelchair-bound, ambulant). Patients were administered mesenchymal, ectodermal, and endodermal stem cells obtained from growth zones of 4-8 weeks old cadaverous embryos’ systems and organs; amounts administered varied from 0,5 to 3 ml, cell count - from 0,1 to 100x105/ml. Our conclusions are based on 9 year’s observation experience in DMD treatment. In the course of the first two months after the treatment, strength of different muscle groups increased by 100-700% and was maintained at high level for 8-12-15 months. At this background, increased was also range of motions, within the limits of the patient’s stage. For example, in stage II patients, reported was 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 4-5th 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, one course in 6-8 months, especially during period of maturation and active growth of the child) 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.
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