Sunday, August 7, 2022

Scientists are on the verge of a breakthrough cure for genetic heart disease

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Scientists believe they are just a few years away from a breakthrough cure for genetic heart disease, which causes 260,000 people in the UK to die suddenly every year.

Researchers have been awarded £30million to develop a cure for inherited heart muscle disease that can kill young people ‘in the prime of life’.

One of the leaders of the Cure Heart project, Professor Hugh Watkins, said the research represents a “unique opportunity to free families from the constant worry of sudden death, heart failure and the potential need for a heart transplant”.

Professor Sir Nilesh Samani, Medical Director of the British Heart Foundation (BHF), said: “This is a defining moment for cardiovascular medicine… [that] could also herald a new era in precision cardiology.”

With the BHF grant, the researchers want to develop, for the first time, cures for hereditary heart muscle diseases by rewriting DNA to edit or shut down faulty genes.

The team, made up of scientists from the UK, US and Singapore, has so far proven their approaches to be successful in animals with cardiomyopathies and in human cells.

The goal is for the therapies to be injected into the arm and halt progression or potentially cure those living with genetic cardiomyopathies.

The technology could also be used to prevent the disease from developing in family members who carry a faulty gene.

“Life Changing”

One patient with heart disease said the cure would be “life-changing.”

Max Jarmey, 27, was diagnosed with arrhythmogenic cardiomyopathy as a teenager, just years after his father Chris died suddenly.

After his diagnosis, he had to give up the sport.

Mr Jarmey said: “I’m quite resilient mentally, but the first six months after my diagnosis have been incredibly difficult. It was horrifying when I was told that at the age I was told I was suffering from a condition like ARVC and then was forced to give up something I loved.”

He now lives with an implantable cardioverter defibrillator (ICD), a device that shocks his heart back to a normal rhythm, protecting him from cardiac arrest.

He said: “Thinking about my future, the decision to have children and their future, Cure Heart might make that decision easier. My children may never have to suffer like I did with this disease. It’s completely life changing.”

“Once in a Generation”

In the UK, 12 people under the age of 35 die every week from undiagnosed heart disease, often caused by an inherited heart muscle disease called genetic cardiomyopathy.

About half of all heart transplants are needed because of cardiomyopathy and it is estimated that around 260,000 people in the UK are affected by these diseases.

Professor Hugh Watkins, from the Radcliffe Department of Medicine at the University of Oxford and Principal Investigator of Cure Heart, said: “This is our unique opportunity to free families from the constant worry of sudden death, heart failure and the possible need for a heart transplant.

“After 30 years of research, we have discovered many of the genes and specific genetic errors responsible for various cardiomyopathies and how they work. We believe we will have a gene therapy that is ready to be tested in clinical trials within the next five years.”

dr Christine Seidman of Harvard University and co-lead of Cure Heart said: “Delivering on our mission will be a truly global effort.

“We have brought in pioneers in new, ultra-precise gene editing and experts in the techniques to ensure we safely deliver our genetic tools directly to the heart. Thanks to our world-leading team from three different continents, our original dream should become a reality.”

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