New gene therapy found safe for treating coronary heart disease

DALLAS, August 3 -- The first human test of a gene therapy that is injected directly into the oxygen-starved heart muscle has shown the technique to be safe, opening the door to promising new treatments for heart disease, according to a report in today's Circulation: Journal of the American Heart Association.

"The implications are huge. To say anything less than that is an understatement. This addresses a tremendous problem of the large number of very sick patients for whom we can do nothing to help their coronary artery disease," says the study's lead author Todd K. Rosengart, M.D., associate professor of cardiothoracic surgery at Weill Medical College of Cornell University, New York City.

The study found no evidence that either the gene or the method used to deliver it (an inactive virus) caused any adverse side effects. "There is no evidence of inflammation, damage to the heart muscle, or viral effects," says Rosengart.

The small, preliminary study of 21 heart patients was not designed to prove the gene therapy's effectiveness. That will require further research, including clinical trials with large numbers of patients.

A build-up of fatty deposits in heart arteries reduces the flow of blood and can lead to a heart attack. Although the Cornell study was not designed to look at gene therapy's role in treating heart disease, most participants said they experienced less chest pain after the injection. A variety of tests also found that the injection led to a growth of new blood vessels, thereby improving blood flow to oxygen-starved areas of their hearts.

"We don't have enough patients to say anything definite about the effectiveness of this therapy in treating heart disease, but if you look at the trends in the data, they are all in the right direction, which is very encouraging," says Ronald G. Crystal, M.D., professor of medicine and chief of pulmonary and critical care medicine

Contact: Carole Bullock
American Heart Association

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