INNOVATIVE THERAPY FOR MUSCULAR DYSTROPHY

Muscular DystrophyMuscular dystrophy (MD) is a disease characterized by progressive skeletal muscle weakness, defects in muscle proteins (dystrophin concentration is greatly reduced), and the death of muscle cells and tissue. Diagnosis is based on muscle biopsy, laboratory evaluation (increased levels of creatine phosphokinase) and EMG findings. There are nine major types of MD and most types of MD are multi-system disorders with manifestations in body systems including the heart, gastrointestinal system, nervous system, endocrine glands, eyes, and brain. MD has a strong genetic link. Treatment options are limited. There has been a keen interest in using cells to regenerate muscle tissue and there has been a success in using human cells for MD in mice. There is hope that adipose-derived cells may be effective in regenerating muscle damaged by MD.

Cells And Muscular Dystrophy

There are a number of different types of cells that scientists think may be used in different ways to develop treatments for muscular dystrophy. The main cell-based approaches currently being investigated are:

Producing healthy muscle fibers: Scientists hope that if cells without the genetic defect that causes DMD can be delivered to patients’ muscles, they may generate working muscle fibers to replace the patient’s damaged ones.

Reducing inflammation: In muscular dystrophy, damaged muscles become very inflamed. This inflammation speeds up muscle degeneration. Scientists believe certain types of cells may release chemicals that reduce inflammation, slowing the progress of the disease.

The Center for Regenerative Cell Medicine has developed a specific treatment protocol that attempts to utilize the potential regenerative properties of fat cells. The treatment is systemically deployed and is expected to hone to damaged areas preferentially. This is all done as an outpatient and the entire cellular surgical procedure takes approximately three hours.