Muscular Dystrophy

Muscular Dystrophy is the generic term for a group of rare hereditary diseases, which are associated with muscular weakness or atrophy. The classification of the different types depends on the affected defective gene. The most common is type Duchenne, followed by the type Becker-Kiener. Both types especially show affection of the muscles in the hip and shoulder girdle area. Due to the inheritance pattern only boys can develop this disease. Globally, about 10 of 100,000 people are affected by muscular dystrophy.

Symptoms and Progression of the Muscular Dystrophy

A faulty muscle protein causes a progressive replacement of muscular tissue by fat or connective tissue and thereby causes a loss of muscle strength. The Duchenne muscular dystrophy manifests itself already in early infancy by frequent falls, whereas the type Becker-Kiener is usually discovered in school age.

The resulting incorrect loading of fit muscles leads to pain and, among other consequences, to spinal curvature. People affected by the severe “malignant” type Duchenne are often confined to a wheelchair at the age of 18 and have a very reduced life expectancy. In contrast, the type Becker-Kiener is often called the “benign” form due to a slower progression. Nevertheless, the life is shortened here too.

Many types of muscular dystrophy also affect the heart and lung muscles and therefore lead to cardiac arrhythmia and respiratory problems. The much less frequently found type of oculopharyngeal dystrophy is characterized by hoarseness and swallowing problems because laryngeal muscles are affected.

Treatment of Muscular Dystrophy

According to the latest research, there is no accepted therapy for the cure of muscular dystrophy. Treatment is thus limited merely to the relief of symptoms. This is, amongst others, done through physiotherapy, which is intended to strengthen and coordinate the still functional muscles. In some cases also surgical therapy to improve the motion capability is recommended. Joint or spinal surgery contribute to the extension of the walking ability. Heart problems are either treated with drugs or by implanting a pacemaker.

The administration of ribonucleic acids, which stabilize the muscles and slow down degradation, also has a slowing effect, but it is no cure. The intake of creatine, which has an performance-enhancing effect on the healthy muscle, is controversial due to its damaging effect on the kidneys.

Stem Cell Therapy of Muscular Dystrophy

In various types of muscle weakness and atrophy, Mesenchymal Stem Cells (MSC) or Stromal Vascular Fraction (SVF) can be used therapeutically. Stem cells are precursor cell of all cells of the body. We now know that the mode of action of mesenchymal stem cells is particularly in the modulation of the immune system and stimulation of the regeneration of tissues and blood vessels by cytokines. Since muscular dystrophy has a degenerative component, the use of mesenchymal stem cells suggests itself.

Mesenchymal stem cells and SVF are isolated from a small amount of the patient’s own fat, which is harvested by liposuction. The stem cells are administered to the affected muscle areas or injected systemically. Due to the regenerative potential of stem cells the progression of the disease may be delayed or stopped. Moreover, the stem cells stimulate stationary stem cells of the muscle tissue to regenerate by means of cytokines. Thereby they could possibly counteract the destruction of other muscle cells and initiate the formation of new muscle fibers. Studies give cause for hope to increase mobility and flexibility of patients through therapy with mesenchymal stem cells.