Gene therapy for heart failure results promising

Trials of gene therapy for the treatment of advanced heart failure have produced promising results, with patients making marked improvements in several areas.

Patients enrolled in the CUPID trial (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease) underwent a procedure in which an engineered gene that stimulates production of an enzyme necessary for the heart to pump efficiently was introduced into their heart. Results of the Phase I section of the trial showed that seven of the nine patients treated showed improvements in several areas, including left ventricular function/remodeling. No improvements were seen in the remaining two patients, both of which had pre-existing antibodies to the viral vector delivery system.

"We are encouraged by these initial findings, which indicate that this therapy has the potential to help patients with advanced heart failure," said study leader Dr. Donna Mancini in a news release.

Given the promising results of the Phase I trial, the researchers hope to enroll 46 patients with advanced heart failure for the Phase II trial.

News release: First trial of gene therapy for advanced heart failure shows promising results. NewYork-Presbyterian Hospital/Columbia University Medical Center. November 10th 2008.