Heart failure patients may no longer have to wait for and undergo heart transplants in the future if a new trial conducted by researchers from the Imperial College London in the U.K proves that an experimental gene therapy works for individuals with advanced heart failure, a condition that negatively affects a person's quality of life and is associated with premature death.
The new treatment, which involves introducing genes into the heart muscles using a laboratory-engineered virus to increase the levels of a protein that plays a crucial role in the contraction of the heart muscles known as SERCA2a, could be helpful to thousands of people who suffer from heart failure.
In Britain, where the clinical trial of the pioneering treatment will be conducted, nearly 500,000 individuals suffer from the condition with hundreds requiring a new heart. Because there are only few donors, however, only around 200 heart transplants take place in the country each year and two out of 10 patients die waiting for an organ. The get around for those with advanced heart failure is to be fitted with Left Ventricular Assist Device (LVAD), which could normalize their blood flow and keep them alive while they wait for a suitable donor heart.
The study, which is funded by the British Heart Foundation (BHF) and U.S biotech firm Celladon Corporation, aims to assess how much of the gene get to the heart muscle and how well it works particularly on the participants who have antibodies to the virus used to deliver the gene.
"We have adapted the wild virus by removing the viral genes and replacing them with the treatment SERCA gene, so the virus acts like a biological courier to deliver our treatment gene," said trial investigator Alex Lyon, from Imperial College London's National Heart & Lung Institute. "If we demonstrate that the antibody does not block the delivery of gene therapy, many more patients could benefit."
The research team has recruited 24 patients from the Harefield Hospital in London and Papworth Hospital in Cambridgeshire with advanced heart failure who are fitted with a mechanical heart pump to assess how the therapy works. Sixteen of the patients will receive the gene therapy while eight will be given placebo. After six months, the researchers will conduct a biopsy to analyze the amount of gene and protein that go to the heart muscle.
The result of the trial is expected to be available by next year.
