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Gene therapy helps patients with severe heart disease grow new blood vessels

Kathleen Blanchard's picture
Gene therapy regrows blood vessels in heart patients.

Researchers have found a way to help patients with severe heart disease grow new blood vessels. In a clinical trial, patients were injected with adenovirus encoding angiogenic growth factor, or AdVEGF121. Ten years later, the patients are doing as well, if not better than patients treated with traditional medical therapy.

The finding is reported by doctors at Baylor College of Medicine, Weill Cornell Medical College (where the clinical trial and review took place) and Stony Brook University Medical Center and published in the journal Human Gene Therapy.

For the study, 31 patients who were not candidates for coronary artery bypass surgery (CABG); who had severe blockages of the blood vessels that supply the heart were given the gene therapy that is injected directly into the heart.

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 said in a press release, "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."

Patients in the study had severe blood vessel disease, meaning there were no options for redirecting blood flow to the heart that is the goal of coronary artery bypass surgery.

Now that ten years have passed, the researchers say they have evidence that the gene therapy works; opening the door for larger clinical trials.

Researchers tested 2 groups of patients. Both groups received gene therapy, but one group also received conventional coronary artery bypass grafting (group B). The researchers say injections of AdVEGF121 helped repair damaged blood vessels.

Ten-year survival rate for group A – the gene therapy only patients – was 40 percent, compared to 31% for group B.

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Follow-up was done by way of interviews and questionnaires; 18 patients died from diseases that ranged from cancer to heart related issues.

"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," Rosengart said.

The authors report there were no adverse effects from gene therapy that was a question at the start of the study.

New clinical trials are being planned by researchers at Weill Cornell Medical College, Weill Cornell Medical College-Qatar and Baylor College of Medicine.

The finding is promising. Severe heart disease from diseased coronary arteries causes symptoms of chest pain with minimal exertion, shortness of breath, inability to perform routine activities of daily living and decreased quality of life. Patients often require oxygen and multiple drug therapies.

Gene therapy shows promise for helping patients with severe heart disease who cannot undergo surgery regrow new blood vessels. If you’re interested in participating in clinical trials, go to clinicaltrials.gov. The gene therapy trials for heart disease are just in the planning stages as of this writing.


Todd K. Rosengart, Muath M. Bishawi, Michael S. Halbreiner, Mathew Fakhoury, Eileen Finnin, Charleen Hollmann, Annie Laurie Shroyer, and Ronald G. Crystal.
Human Gene Therapy

Image credit: Wikimedia commons
Gene therapy phase II trial shows promise for severe heart failure