Stem Cell Treatment for Muscular Dystrophy (MD)

What Makes our Stem Cell Treatment for Muscular Dystrophy Effective?

Since 2005, we have been developing comprehensive protocols regarding stem cell treatment for muscular dystrophy (MD) to overcome the limitations of conventional therapies. In our protocols, stem cells are combined with specialized therapies for muscular dystrophy that not only focus on helping the patient to cope with their symptoms, but also treat the direct cause of the symptoms by promoting the healing of the affected muscles. We believe that our comprehensive stem cell treatment for muscular dystrophy gives our patients the best chances of improvements, allowing for a better quality of life. Different types of muscular dystrophy can be considered for treatment, such as Duchenne, Becker, Limb Girdle, Fascio-Scapulo Humeral and more.

What is Muscular Dystrophy and how does it affect the patient?

Muscular dystrophy (MD) is a group of inherited progressive muscle disorders which are characterized by muscle weakness, wasting and degeneration.

Most forms of MD can also affect other body organs such as heart, gastrointestinal tract and brain. MDs are caused by mutations in genes that code for specific muscle proteins.

Duchenne Muscular Dystrophy (DMD) is one of the most common and lethal MD which affects 1 in every 3500 males and is caused by mutations in the dystrophin gene. Dystrophin is a protein found in muscles of the entire body, whose role is to ensure muscle fibers stability. A lack of functional dystrophin leads to muscular dystrophy. There are many kinds of muscular dystrophies including Duchenne’s MD, Becker’s MD, Limb Girdle MD and Facioscapulohumeral MD and more.

What happens if Muscular Dystrophy is not treated?

Individuals diagnosed with Muscular Dystrophy characteristically display progressive muscle weakness. Patients with Duchenne muscular dystrophy (DMD) for instance notice the first symptoms in early childhood.

Initial symptoms include leg weakness, increasing convex curvature of the spine, and a waddle-like gait. Continuous loss of muscle fibers results in muscles that get weaker overtime, usually forcing DMD individuals to use a wheelchair by the age of 11 or 12.

In the latter stages, most patients succumb to cardiac or respiratory failure in their twenties, although rare cases of survival into the thirties have been reported. A similar, yet milder type of muscular dystrophy known as Becker muscular dystrophy (BMD) along with other types of muscular dystrophy generally follow a less severe course than DMD.

Are there any conventional treatments available for Muscular Dystrophy patients and what is their efficacy?

So far there is no conventional strategy that shows any clinical benefit for any type of Muscular Dystrophy. Conventional treatment includes supportive care (surgery, corticosteroid administration, medication, physiotherapy etc.) that partially reduces signs and symptoms. Such therapies, however, do not reverse the phenotype nor can they directly target the cause for the pathology. Few treatment options are now available for MD, including pharmacological, gene and cell therapy.

• Pharmacological: Various compounds such as allopurinol, vitamin E and selenium as well as mazindol, a growth hormone inhibitor were deemed ineffective or not beneficial against the progression of DMD.
• Gene therapy provides a promising option in treating MD but still requires significant advances in the knowledge of genes, muscle promoters, viral vectors, immune system surveillance and methods for systemic delivery of vectors.
• Stem cell therapy is based on the delivery of precursor cells (autologous or allogeneic) that contribute to the regeneration of muscle fibers and tissue repair. It is considered to be a safe and effective option when compared to gene and pharmacological therapy. However it is important to note that gains in muscle mass and improvements can only be temporary as stem cells therapy can not treat the defective genes that are the cause of Muscular Dystrophy.