The gene therapy clinical trial and review took place at Weill Cornell Medical College, and was published in the journal Human Gene Therapy. 31 patients took part in the trial, and their wellbeing depended on the results, as they were not candidates for coronary artery bypass surgery (CABG). These patients had severe blockages of the blood vessels that supply the heart, and so their heart was directly injected with the gene therapy.
The researchers reported their findings alongside doctors at Baylor College of Medicine and Stony Brook University Medical Centre. According to a press release from 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 Centre, ‘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 goal of coronary artery bypass surgery is to improve heart health by redirecting blood flow to the heart, and so this wasn’t an option for the patients in this study, who had severe blood vessel disease. However, ten years on, the researchers say they have evidence that the gene therapy works by helping to repair damaged blood vessels, and the results open the door for larger clinical trials.
Of the two groups involved in the study, the first only received the gene therapy whilst group B also received conventional coronary artery bypass grafting. The survival rate for the latter group was 31%, whereas this rose to a 40% ten-year survival rate for group A. Rosengart commented, ‘While there were health issues that needed additional treatments, such as cardiac revascularization and implantable cardioverter-defibrillators among both groups, overall this group of individuals had an outcome greater than what we believe they would have if they had not received the gene therapy.’
The authors added that there were no negative side effects from the therapy, which was a concern at the beginning of the study. With that in mind, and with the promising results of the 10-year trial, researchers at Weill Cornell Medical College, Weill Cornell Medical College-Qatar and Baylor College of Medicine are now planning new clinical trials.