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Duchenne-Related Stem Cell Heart Therapy Trial Underway

Heart in rib cage illustration


22 February 2016. An early clinical trial testing a stem cell therapy for heart disease among individuals with Duchenne muscular dystrophy treated its first enrolled patient. The trial is conducted by Capricor Inc. in Los Angeles, developer of the treatments code-named CAP-1002, for cardiomyopathy or heart failure related to Duchenne muscular dystrophy.

Duchenne muscular dystrophy is a rare genetic disorder resulting in progressive muscle degeneration and weakness, primarily in the shoulders, arms, hips, and thighs. The disease affects mainly boys starting at age 3 to 5, and caused by a defective gene that fails to produce the protein dystrophin for strengthening muscle fiber and protecting muscles from injury. While life expectancy can vary, people with Duchenne muscular dystrophy do not often survive past their 20s or 30s.

Cardiomyopathy or degeneration of heart muscles leading to heart failure is among the leading causes of death among individuals with Duchenne muscular dystrophy. People with the disorder develop irregular heart beats, shortness of breath, fatigue, and swelling in legs and feet. The condition usually worsens, leading to heart failure in adolescence or early adult ages.

CAP-1002 is Capricor’s lead product designed to regenerate heart tissue from cardiac stem cells. The treatments take a piece of a donor’s or the individual’s own heart tissue, from which are extracted cells known as cardiospheres producing stem cells that are introduced back into the coronary arteries of the patient. Capricor says cardiosphere-derived stem cells release growth factors and cytokines, cells that stimulate specific cell responses, signaling the heart to repair itself.

The clinical trial is an early- and intermediate-stage test of CAP-1002 as a treatment for Duchenne muscular dystrophy. The study is enrolling 24 patients randomly assigned to receive either infusions of CAP-1002 in three coronary arteries serving left ventricle regions of the heart, or receive the usual standard of care. Researchers are looking primarily at safety and tolerability of the treatments, but also evidence of structural change in the heart, as well as changes in mobility and quality of life.

The company was granted orphan drug designation by the FDA for CAP-1002 for the treatment of cardiomyopathy related to Duchenne muscular dystropy. Capricor is also testing CAP-1002 in early and intermediate-stage trials among adults with heart failure or who experienced a heart attack.

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