Scientists believe they are just years away from a breakthrough cure for genetic heart conditions that put 260,000 people in the UK at risk of sudden death every year.
Researchers have been awarded £30 million to develop a cure for inherited heart muscle diseases that can kill young people “in the prime of life”.
One of the Cure Heart project leaders, Professor Hugh Watkins, said the research was “a once-in-a-generation opportunity to relieve families of the constant worry of sudden death, heart failure and the potential need for a heart transplant”.
Professor Sir Nilesh Samani, Medical Director at the British Heart Foundation (BHF), said: “This is a defining moment for cardiovascular medicine… [that] may also usher in a new era of precision cardiology.
Professor Hugh Watkins says his team believed they would be ready for clinical trials within five years
(PA)
Using BHF funding, researchers aim to develop first-of-its-kind cures for inherited heart muscle diseases by rewriting DNA to edit or silence faulty genes.
The team, made up of scientists from the UK, US and Singapore, has so far shown that its approaches are successful in animals with cardiomyopathies and in human cells.
The goal is for the therapies to be injected into the arm and stop the progression or potentially cure those living with genetic cardiomyopathies.
The technology can also be used to prevent the disease from developing in family members who carry a defective gene.
“Life Changing”
Max Jarmey, right, as a child with his father Chris
(BHF)
One heart patient said the treatment would be “life-changing”.
Max Jarmey, 27, was diagnosed with arrhythmogenic cardiomyopathy in his teenage years, just a few years after the sudden death of his father Chris.
He was forced to give up the sport after the diagnosis.
Mr Jarmey said: “I’m quite mentally healthy but the first six months after my diagnosis were incredibly difficult. It was terrible to be told I had a condition like ARVC at the age I was told and then to be forced to leave something I love.’
He now lives with an implantable cardioverter defibrillator (ICD), a device that returns his heart to a normal rhythm, protecting him from cardiac arrest.
He said: “When I think about my future, the decision to have children and their future, Cure Heart can make that decision easier. My children may never suffer like I did with this condition. It’s completely life-changing.”
“Once in a Generation”
Every week in the UK, 12 people under the age of 35 die from undiagnosed heart disease, often caused by an inherited disease of the heart muscle called genetic cardiomyopathy.
Around half of all heart transplants are required due to cardiomyopathy and it is estimated that around 260,000 people in the UK are affected by these conditions.
Professor Hugh Watkins, from Oxford University’s Radcliffe Department of Medicine and Cure Heart lead researcher, said: “This is our only chance to free families from the constant worry of sudden death, heart failure and the potential need for a heart transplant.
“After 30 years of research, we have discovered many of the genes and specific genetic defects responsible for various cardiomyopathies and how they work. We believe we will have gene therapy ready to begin testing in clinical trials within the next five years.
Dr. Christine Seidman of Harvard University and one of the leaders of Cure Heart said: “Achieving our mission will be a truly global effort.
“We have pioneered new, ultra-precise gene editing and are experts in the techniques to ensure we deliver our genetic tools straight to the heart safely. Thanks to our world-leading team from three different continents, our initial dream should become a reality.”
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