A once-in-a-generation medicine could prove to be the solution for genetic heart conditions, say researchers
A team of researchers from around the world are developing the first-ever cures for inherited heart muscle diseases by rewriting DNA in order to fix faulty genes, the cause of genetic heart conditions. They have already been awarded £30m by the British Heart Foundation (BHF) to progress their research.
The researchers hope that within just a few years this ‘cure’ can be delivered to patients as a simple jab, which could have a huge impact in preventing illness in family members who carry the same faulty genes.
“This is our once-in-generation opportunity to relieve families of the constant worry of sudden death, heart failure and potential need for a heart transplant,” says Professor Hugh Watkins, University of Oxford, and lead investigator of the project, known as CureHeart.
Inherited heart muscle conditions are driven by different abnormalities in the heart – but can cause sudden death or progressive heart failure.
In the UK alone, every week 12 people under the age of 35 die of an undiagnosed heart condition, very often caused by an inherited heart muscle disease, also known as genetic cardiomyopathy.
Those with genetic cardiomyopathies have a 50 per cent risk of passing faulty genes on to their children and often several members of the same family develop heart failure, need a heart transplant, or die at a young age.
Professor Watkins highlighted how common cardiomyopathies are and how action needs to be taken as soon as possible.
“There will be one or two in every school. Every GP surgery will have several patients with these conditions, but there’s quite a range of severity,” he says.
While not all patients would require this new therapy, Professor Watkins stated that “a very large number” would benefit from it.
“After 30 years of research, we have discovered many of the genes and specific genetic faults responsible for different cardiomyopathies, and how they work. We believe that we will have a gene therapy ready to start testing in clinical trials in the next five years,” he says.