Diagnosed with severe coronary artery disease, a group of patients too ill for or not responding to other treatment options decided to take part in a clinical trial testing angiogenic gene therapy to help rebuild their damaged blood vessels. More than 10 years later, in a follow-up review of these patients, doctors at Baylor College of Medicine, Weill Cornell Medical College (where the clinical trial and review took place) and Stony Brook University Medical Center report the outcomes are promising and open the door for larger trials to begin.
The study, which appears online in the journal Human Gene Therapy, followed 31 Weill Cornell patients who were diagnosed with severe coronary artery disease and were given a direct injection into their heart muscle of gene therapy called adenovirus encoding angiogenic growth factor, or AdVEGF121. Study results show the five- and 10-year survival rate of those patients were just as good and, in some cases better, than what is seen in other groups with similar heart issues treated with traditional medical therapy.
"The results of this 10-year gene therapy study are important," said co-senior author Dr. Ronald G. Crystal, chairman and professor of Genetic Medicine at Weill Cornell Medical College. "After long-term follow-up, the patients who received angiogenic gene therapy appear to have improved outcomes. The study results give us greater insight into the safety and effectiveness of gene therapy to rebuild blood vessels in patients living with coronary artery disease."
"At the time when the trial began, there were no comparisons available to tell us what to expect, which is why we are so pleased with the results," said co-senior author Dr. Todd Rosengart, professor and chair of the Michael E. DeBakey Department of Surgery at Baylor College of Medicine, formerly of Stony Brook University Medical Center. "We only had an idea of what the outcome might be based on promising studies in the lab, so there was concern, but those who received this treatment really had no other treatment options."
The common treatment for severe coronary artery disease is coronary artery bypass surgery, which works by redirecting blood flow around the diseased or blocked area of the heart. However, for those involved in this trial, the blood vessels that normally would be used to redirect the flow of blood were not healthy enough to do so.