More than 200 people with heart failure are to receive a pioneering form of gene therapy to try to get their hearts beating properly again. "This is the first ever gene therapy trial to target heart failure," says lead investigator Alexander Lyon of Imperial College London.

Heart failure results after damage to the heart muscle causes it to deteriorate, which in turn progressively weakens cells that govern heartbeat. The result is serious fatigue due to the heart's inability to pump blood efficiently. Each year in the UK alone it affects 120,000 people who have never had the condition before, killing a third of them within 12 months.

Doctors will inject participants with harmless viruses that ferry a gene called SERCA2a into their heart muscle. The gene codes for a protein that recycles calcium within heart muscle cells, vital for driving each heartbeat and priming the next one.

In damaged cells, this recycling is impaired. By loading new copies of the gene the aim is to compensate for this decline. "The gene therapy will reset the calcium control," says Lyon.

A preliminary trial of the same therapy three years ago in 39 people demonstrated that it is safe and delivers benefits. Those who got the highest dose of the virus, for example, spent only a tenth as long in hospital as those given a placebo (Circulation, doi.org/bzvxst). The impending follow-up trial will recruit 200 people split equally between the US and Europe.

In a separate trial of the same therapy, doctors will treat 24 people who already have temporary mechanical implants to aid heartbeat while they await heart transplants.

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Gene therapy to repair failing hearts starts trial

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