Duchenne Muscular Dystrophy Related Heart Damage May Be Healed Using Cardiac Stem Cell Therapy

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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shutterstock_112836925A team of researchers recently presented their latest results showing how injecting cardiac stem cells in mouse models of Duchenne muscular dystrophy can reverse damage to the heart. These findings carry a new therapeutic promise to patients with muscular waisting disorders. The study was presented in the American Heart Association Scientific Sessions, Chicago that occurred in November 15-16, 2014.

Duchenne muscular dystrophy (DMD) is characterized by absence or defected production of dystrophin, a key protein connecting muscle fibers to the surrounding extracellular matrix leading to degeneration of skeletal, smooth and cardiac muscle. Consequently, cardiac involvement with progressive heart failure and arrhythmias is associated with increased mortality in DMD patients.

Researchers at the Cedars-Sinai Heart Institute injected cardiac stem cells in DMD-mice that exhibited signs of improved heart function and exercise capacity after treatment.

Eduardo Marbán, MD, PhD, director of the Cedars-Sinai Heart Institute and study leading author noted, “Most research into treatments for Duchenne muscular dystrophy patients has focused on the skeletal muscle aspects of the disease, but more often than not, the cause of death has been the heart failure that affects Duchenne patients. Currently, there is no treatment to address the loss of functional heart muscle in these patients.”

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The Cedars-Sinai Heart Institute has a long-term commitment in establishing stem cells to regenerate heart muscle in patients who had a heart attack. The team led by Dr. Marbán in 2009 performed the world’s first procedure in producing heart stem cells from a patients own heart cells. The stem cells were later injected back into the patients’ heart to repair the damage in the heart as a consequence of the heart attack. The study was published in The Lancet journal. Now, the team is applying their intensive knowledge on heart stem cells and translating it to other diseases where there is damaging to the heart, such as Duchenne muscular dystrophy.