Becker's Muscular Dystrophy - Treatment

Treatment

There is no known cure for Becker muscular dystrophy. Treatment is aimed at control of symptoms to maximize the quality of life.

Activity is encouraged. Inactivity (such as bed rest) or sitting down for too long on plane or car rides can worsen the muscle disease. Physical therapy may be helpful to maintain muscle strength. Orthopedic appliances such as braces and wheelchairs may improve mobility and self-care.

Immunosuppressant steroids like Prednisone have been known to help slow the progression of Becker Muscular Dystrophy. The drug contributes to an increased production of the protein Utrophin which closely resembles Dystrophin, the protein that is defective in BMD. IVIG has proved better in the long term, but its price is a limiting factor.

A Utrophin up-regulation drug by UK Company SummitPLC for Duchenne muscular dystrophy (DMD) is expected to enter clinical trials 2012. This drug could also be used for BMD.

A hepatitis C drug, "Debio-025", that has proven safe for use in Europe shows much promise for halting the muscle necrosis seen in the disease. The investigational drug Debio-025 is a known inhibitor of the protein cyclophilin D, which regulates the swelling of mitochondria in response to cellular injury. Researchers decided to test the drug in mice engineered to carry MD after earlier laboratory tests showed deleting a gene that encodes cycolphilin D reduced swelling and reversed or prevented the disease’s muscle-damaging characteristics. The mice were engineered as models of Duchenne muscular dystrophy and forms caused by a deficiency of two structural proteins, delta-sarcoglycan and laminin alpha2. Treatment with Debio-025 was reported to reduce mitochondrial swelling and necrotic manifestations in mice with muscular dystrophy. Debio-025 has passed Phase II clinical trials in Europe.

During the onset of muscular dystrophy, the loss of certain proteins critical to muscle function – such as dystrophin – can lead to contraction-related micro-tears in muscle fibers and an influx of calcium around muscle tissue. When this happens, cyclophilin D is instructed to make the membranes of mitochondria more permeable. This causes mitochondria to be flooded by calcium and reorganize, swell and eventually rupture. This triggers cell death in muscle fibers and leads to the progressive muscle weakness, wasting and often early death associated with muscular dystrophy. There is another way of slowing down the symptoms or altogether stopping them. Stem Cell Treatment has come a long way in providing treatments for various medical conditions including BMD. In 2009 stem cells from a Placental source were being placed in the human body by incisions and injections, most of the time with amazing results which may or may not be long term.These treatments all took place outside of North America. In 2011, the new method of using your own fatty cells and extracting and treating the stem cells found in your fatty cells and introducing them back into the body with intravenous and as well there clinics in the US specializing in this type of stem cell.