Patients in the high-dose SERCA2a group demonstrated improvement and/or stabilization in symptoms, overall heart function, biomarker activity, and ventricular mechanics and function. They also saw a dramatic reduction in cardiovascular hospitalizations, averaging 0.4 days versus 4.5 days in the placebo group.
"Even though heart failure mortality has decreased over the last decade with the help of standard pharmacological and device therapies, patients with advanced heart failure continue to die at high rates. The CUPID trial offers a new therapeutic option for these patients," said Dr. Hajjar.
Led by Dr. Hajjar, the Mount Sinai team discovered the landmark potential of the cardiac-specific target in 1999 and has been pursuing its potential as a treatment delivered via gene therapy in state-of-the-art custom built laboratories at Mount Sinai School of Medicine in New York.
According to the U.S. Centers for Disease Control and Prevention, about 5.8 million Americans suffer from heart failure, and 670,000 new cases are diagnosed each year. One in five people who have heart failure die within one year of diagnosis. In 2010, heart failure will cost the United States $39.2 billion, including the cost of health care services, medications, and lost productivity. Heart failure is most often treated with aggressive medical and device therapy, but has no cure. The most common symptoms of heart failure are shortness of breath, feeling tired, and swelling in the ankles, feet, legs, and sometimes the abdomen.
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