Gene therapy trial for advanced heart failure promising
Nov 12, 2008 - 1:29:06 PM
Washington, Nov 11 - The first clinical trial of gene therapy for advanced heart failure has shown promising results.
It is a technique for correcting defective genes responsible for the disease by inserting genes into a patient's cells and tissues.
Seven of nine patients given the drug showed improvements over six months in several areas. Two patients did not show improvements.
This approach under CUPID's phase-I trials was shown to have an acceptable safety profile, validated by an independent safety committee and by study investigators.
CUPID stands for calcium up-regulation by percutaneous administration of gene therapy in cardiac disease.
Enrolled patients underwent cardiac catheterisation, to introduce an engineered gene to stimulate enzyme production to help the heart pump more efficiently.
'We are encouraged that this therapy has the potential to help patients with advanced heart failure,' said Donna Mancini, the study's principal investigator and professor of medicine at Columbia University.
More than five million people in the US have heart failure. Patients with severe form of the disease have trouble breathing because the heart muscle is not strong enough to pump fluid out of their lungs.
Approximately 70 percent die of the disease within 10 years, and the five-year survival rate is less than 50 percent.
New York-Presbyterian Hospital / Columbia University Medical Centre was the first to offer the therapy in the New York City area.
The hospital is now recruiting 46 patients for the Phase II CUPID trial to further assess safety and effectiveness in patients with advanced heart failure, said a Columbia press release.
The phase-II randomized, double-blind, placebo-controlled clinical trial will compare the therapy at two- or three-dose levels with placebo.
Data from Phase I trial were presented at the American Heart Association - Scientific Sessions in New Orleans.
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