Treatment of Duchenne Muscular Dystrophy with Cardiosphere-Derived Cells

Repeated intravenous CDC infusions in patients with late-stage DMD had a measurable but relatively limited beneficial effect, with slowing of disease progression but not improvement of upper-extremity function.

Duchenne muscular dystrophy (DMD) is a sex-linked genetic disorder that produces severe progressive muscular weakness in boys, who typically are wheelchair bound by the second decade of life and die of cardiac or pulmonary impairment by the fourth decade. In DMD, mutations prevent expression of dystrophin, which is essential to protect the integrity of skeletal and cardiac muscles. Treatment with glucocorticoids slows progression of DMD (Lancet 1974; 2:1409), probably by inhibiting inflammation, and is the standard of care. Cardiosphere-derived cells (CDCs) are cardiac progenitor/stromal cells that were originally developed to treat congestive heart failure secondary to myocardial infarction (Nat Biomed Eng 2018; 2:353). They are anti-infla…

Reviewing Author

Daniel B. Drachman, MD

Treatment of Duchenne Muscular Dystrophy with Cardiosphere-Derived Cells

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Reviewing Author