Muscular dystrophy (MD) refers to a number of inherited diseases characterized by progressive weakness and wasting of the muscles. The most common MD type is Duchenne's muscular dystrophy. Among others are Becker, limb girdle, congenital, facioscapulohumeral, myotonic, oculopharyngeal, distal, and Emery-Dreifuss MD types.
Duchenne muscular dystrophy (DMD) is a hereditary X-linked disease developing as a result of mutation in the dystrophin gene that leads to deficiency of this protein. Dystrofin belongs to the large class of sarcolemmal proteins and glycoproteins. Dystrophin deficiency results in degeneration of cells, especially myocytes, and cells participating in muscle stimulation processes related to contractive activity. Dystrophin deficiency also leads to relaxation of the sarcolemma hitch, which, in turn, results in membrane rupture and launches the sequence of processes resulting in muscle fibre necrosis.
The types of MD differ by the location of the muscles affected, the rate of the disease progress, the age the first symptoms appear, etc. For example, the genes for Duchenne's that are X-linked express only in boys but are carried and passed on by women. Boys with Duchenne's MD are usually in a wheelchair by the age of 12 and rarely live past their teens.
There is no cure for muscular dystrophy but medications and therapy can slow the course of the disease. MD treatment with fetal stem cells proves to be one of the options leading not only to deceleration in muscles wasting but also to some regression of the main disease symptoms.
In the course of MD treatment with stem cells, pools of genetically healthy cells are created in the body of the patient; these cell pools are capable of producing dystrophin which is able to penetrate affected cells. In addition, in the processes of determination, differentiation, and morphogenesis, transplanted stem cells produce pools of specialized cells the patient’s body needs, such as muscular, nervous, endothelial, etc.
At present, Cell Therapy Center EmCell is following up more than 216 patients with Duchenne MD and other forms of muscular dystrophy. MD treatment is carried out at different stages of disease, at different age, at different grades of muscle atrophy, and at different grades of patient’s capacity for independent motion (bedridden, in a wheelchair, able to walk independently).
Our long-term experience of treating patients with muscular dystrophy allows for making the following conclusions:
- Transplantation of fetal stem cells completely halts or considerably slows down disease progression at any stage.
- After transplantation of fetal stem cells, patients experience increase in muscular force, deep reflexes improve or appear, function of internal organs improves, and so does mental and physical activity.
- In order to avoid muscular atrophy, MD treatment should be continuous. Treatment courses must be carried out no less than once in 6–8 months (the frequency is established individually), especially in the intensive growth period.
Duration of the course of MD treatment at Cell Therapy Center EmCell, as a rule, totals 2 days.
Incredible but true: some of our patients in whom DMD diagnosis has been genetically confirmed in childhood at present have reached 29 years of age.
Medical case reports
History of patient R. Diagnosis: DMD
Treatment of DMD in patient R. Medical case report No.1
Treatment of DMD in patient R. Medical case report No.2
Treatment of DMD in patient R. Medical case report No.3
Treatment of DMD in patient R. Medical case report No.4
Assessment of muscle tone in patient R.
Letter from R.'s parents
The letter from the patient M., suffering from DMD, after the third course of stem cell treatment