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Case reports
Patient R., male, DOB 1991, Duchenne Muscular Dystrophy
Transplantation of Embryonic Stem Cells in Treatment of Duchenne Muscular Dystrophy (DMD).
DMD is an X-linked recessive disorder which is caused by a mutation of the gene responsible for production of dystrophin. Dystrophin is a part of a large complex of sacrolemmal proteins and glycoproteins. The lack of dystrophin causes degeneration of cells, especially muscle cells as well as other irritable and contractible cells. Deficiency of dystrophin weakens the sarcolemma causing membrane ruptures and a cascade of events leading to muscle fiber necrosis. This chain of events happens repeatedly in the course of life of a DMD patient.
We suggest that embryonic stem cells enable the following mechanisms:
The pools of genetically intact cells able to produce dystrophin are generated within the body. Dystrophin get into compromised cells of recipient through the mechanisms of cell interactions. In the process of determination, differentiation and morphogenesis transplanted embryonic stem cells produce generations of specialized cells – muscular, nervous, endothelial and others – which are required by damaged tissues and organs.
At present, 6 patients with DMD are observed at Cell Therapy Clinic: 3 Ukrainian boys, 2 boys from the US, and one – from Arab Emirates. Treatment was started at different stages of the disease, which is seen from their age (from 6 to 12 years), degree of atrophy of different muscle groups and also their ability to move (restricted to bed, in the wheel-chair, or on their own).
Our 3-year experience of treating patients with DMD allows for the following conclusions:
Transplantation of embryonic stem cells interrupts or substantially retards the progression of the disease at any stage of its development. After transplantation of embryonic stem cells patients show increase of muscular strength, improvement or even reappearance of lost reflexes, improvement of functions of internal organs and improvement of mental and physical activity. Treatment of patients with DMD requires several courses of therapy (at least one per 4-6-8 months, especially during the increased growth), in order to prevent muscular atrophy. Discontinuance of Embryonic Stem Cell Therapy entails further progress of disease. Embryonic Stem Cell Therapy at any stage of the disease leads to considerable positive results.
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