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Clinical trial phase 2 of gene therapy to rescue failing heart after successful first trial
Posted on Monday, November 10, 2008 (EST)
UC San Diego is enrolling patients in a Phase 2 clinical trial of a new drug for treating advanced heart failure. The new gene-based therapy stimulates production of an enzyme that enables the heart to pump more effectively.
 
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UC San Diego is enrolling patients in a Phase 2 clinical trial of a new drug for treating advanced heart failure. The “Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease,” or CUPID, is a new gene-based therapy stimulates production of an enzyme that enables the heart to pump more effectively.
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November 10, 2008, (Sawf News) - UC San Diego is enrolling patients in a Phase 2 clinical trial of a new drug for treating advanced heart failure. The “Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease,” or CUPID, is a new gene-based therapy stimulates production of an enzyme that enables the heart to pump more effectively.

The first phase of the CUPID trial, reported on November 9th at the American Heart Association Scientific Sessions 2008, shows that the drug had an acceptable safety profile in the first group of patients. This study will be the second phase of the trial.

“The CUPID trial is designed to rescue a failing heart by replacing an enzyme known to play a critical role in healthy cardiac function,” said Barry Greenberg, M.D., director of the Advanced Heart Failure Program at UC San Diego Medical Center and president of the Heart Failure Society of America. “The objective of the study is not only to improve the symptoms of heart failure, but importantly, to reverse the severity of the disease in patients.”

CUPID is expected to enroll 46 patients with advanced heart failure at 13 U.S. medical centers. The clinical trial drug MYDICAR is delivered in a single dose directly to the heart muscle during a short outpatient procedure performed in a cardiac catheterization laboratory. CUPID is funded by Celladon Corporation, makers of MYDICAR.

Despite important therapeutic advances in drug and device therapies, the prognosis of heart failure patients remains poor. The most common symptoms of heart failure are shortness of breath, feeling tired, and swelling in the ankles, feet, legs, and sometimes the abdomen. There is no cure for heart failure.

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