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Fetal Stem Cells in Dushenne\'s Muscular Dystrophy

Alexander I. Smikodub, Cell Therapy Clinic of the National Medical University and EmCell Clinic

78 patients with Duchenne muscular dystrophy (DMD) aged 3 to 18 years, including three girls, were observed at EmCell clinic. In all cases, the diagnosis was confirmed by genetic analysis.

The stem cell treatment began at different stages of the disease, and the patients differed in the degree of muscle atrophy and ability to move (bedridden, in wheelchairs and walking). Patients were administered mesenchymal, ectodermal and endodermal stem cells derived from germ layers of systems and organs of 5-8-week old embryos. Administered dose ranged from 0.5 to 3 ml, the stem cell content equaled 0.1-100*105/ml.

Our conclusions are based on a nine-year observation of the DMD patients that were treated with stem cells. Within the first two months after the stem cell transplantation, strength of various muscle groups in these patients increased by 100-700% and remained high during 8 to 15 months. In addition, patients’ motor skills improved, depending on the stage of the disease. For example, patients at the second stage of the disease had their walking ability improved. Their ability to rely on the heel and walk up/down the stairs increased, and foot lifting became more confident. Moreover, they had increased ability to tolerate the daily stress.

During the first two weeks after the transplantation, the activity of certain blood enzymes increased, and it markedly decreased or even returned to normal over the following 4-5 weeks. Usually, this effect persisted for 2 to 5 months. There was also a reduction in pseudo hypertrophy and tention of forearm and calf, contracture of knee and ankle joints, the manifestations of myocardiopathy and respiratory failure, especially in patients at the stage IVa-IVb.

Duchenne muscular dystrophy patients need continuous stem cell treatment (at least once every 6-8 months) to stop progression of muscle atrophy. Treatment cessation leads to the further disease progressing and physical activity limitation. Regardless of the stage of the disease, stem cell transplantation stops DMD development and leads to significant improvement in the patient's condition. Among other things, the cellular immunity is restored and cytotoxic activity of mesenchymal cells is reduced.