Trials in the UK to treat heart failure using gene therapy are just weeks away. As many as a million people here have the condition, where the heart is unable to effectively supply blood to other parts of the body and as a result is potentially life-threatening. It is therefore no wonder that this latest stage in the research into a novel treatment for heart failure is eagerly anticipated. There are two clinical studies imminent, which will be conducted in a small selection of patients with this debilitating health problem, where a harmless virus will be used to insert a copy of a gene into their cardiac cells that is understood to code for a protein that will strengthen their heartbeat. This work aims to reverse changes within the heart itself, giving those with the disease a fighting chance.

The trials

In one trial, to be conducted at London’s Royal Brompton Hospital and Glasgow’s Golden Jubilee National Hospital, selected patients from across the globe will have the virus introduced by way of a cardiac catheter running through a leg vein. The other trial will involve 24 UK patients, who have an “artificial heart” that aids their own heart to transport blood, and be run by Harefield and Papworth hospitals. In both studies a genetically modified adeno-associated virus will carry the SERCA2a gene; this virus has been used in previous gene therapy work and is not known to pose any risk to health. If all goes to plan, the introduced genes will increase the size and strength of cardiac muscle cells aiding the heart to beat in a rhythmic manner and will hopefully continue to do so for months and possibly years. Although numerous gene therapy trials around the world have failed in the past and doctors do not want patients with heart failure to get their hopes up, researchers are hopeful that in a matter of years, this form of therapy could potentially be available.

The potential benefits

It has taken twenty years of research to get to this point, but it offers hope for anyone with heart failure. By regaining a heart that is able to beat efficiently, this will improve their well being, helping to reduce severe fatigue and prevent complications such as rapid unintentional weight loss, fluid in the lungs and kidney impairment. It would also help to allow those affected by the disease to live longer, as it is a progressive illness and currently around a third of sufferers die less than a year after being diagnosed.

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