Genetic heart disease cure scientists on brink in 'watershed moment' for medicine

Genetic heart disease cure scientists on brink in ‘watershed moment’ for medicine

Scientists believe they are just years away from a breakthrough cure for the genetic heart disease that puts 260,000 people in the UK at risk of sudden death each year.

Researchers have been given £30million to develop a cure for inherited heart muscle diseases that can kill young people ‘in their prime’.

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

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

Professor Hugh Watkins says his team thought they would be ready for clinical trials within five years

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Professor Hugh Watkins says his team thought they would be ready for clinical trials within five years

(PENNSYLVANIA)

With funding from the BHF, researchers aim to develop the first-ever 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, have so far proven their approaches to be effective 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 people with genetic cardiomyopathies.

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

‘Lifestyle change’

Max Jarmey, right, as a child with his father Chris

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Max Jarmey, right, as a child with his father Chris

(BHF)

A heart patient said the remedy would “change his life”.

Max Jarmey, 27, was diagnosed with arrhythmogenic cardiomyopathy as a teenager, just years after the sudden death of his father Chris.

He was forced to give up the sport after his diagnosis.

Mr Jarmey said: ‘I’m quite robust mentally, but the first six months following my diagnosis were incredibly difficult. It was awful to be told I had a disease like ARVC at the age I was told, and then to have to leave something I loved.

He now lives with an implantable cardioverter defibrillator (ICD), a device that brings his heart back 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 could make that decision easier. My children may never have to suffer like me with this condition. It is completely life changing.

“Once in a Generation”

Every week in the UK, 12 people under the age of 35 die of undiagnosed heart disease, often caused by an inherited condition of the heart muscle 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 conditions.

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

“After 30 years of research, we have discovered many specific genes and genetic defects responsible for different cardiomyopathies, and how they work. We believe we will have gene therapy 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: “Acting on our mission will be a truly global effort.

“We brought in pioneers of new ultra-precise gene editing and experts with the necessary techniques to ensure that our genetic tools enter directly into the heart safely. It is thanks to our leading team on three different continents that our initial dream should come true.

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