Potential cure for inherited heart disease a step closer after British Heart Foundation funding
A heart transplant patient has welcomed new research into a treatment which could save his children from the inherited disease which killed his mother and grandfather.
Daniel Gregg, 42, has a heart condition called dilated cardiomyopathy (DCM) - a disease that has devastated his family.
His grandfather died from the disease at the age of 32 and his mother Ann had a cardiac arrest at 34. She died in 2012, more than 10 years after having a heart transplant.
Now there is new hope that his two children, Luke, 18, and Maya, 11, who could have inherited the heart condition, will not suffer the same fate.
Scientists have made a significant step towards developing of an injectable cure for inherited heart muscle conditions which can strike young people down in the prime.
Mr Gregg, from Walton-on-the-Naze in Essex, said: "My main concern now is obviously regarding my children.
"If you look, it's been passed down from father to child, father to child through quite a few generations within my family.
"The odds of one of my children inheriting this is quite likely.
"For me this is fantastic news, if there's a cure. If they can give them an injection that's going to cure this, then for me that's probably the best new you can get."
Speaking about his own diagnosis, Mr Gregg said he was in his mid-forties, enjoyed running and thought everything was fine.
“I had a little time off running after completing a half-marathon," he said. "But when I went to go out running again, I couldn’t do more than a couple of hundred metres without being out of breath.”
Over the next three years, Mr Gregg was regularly in hospital as his condition rapidly worsened.
He was put on the heart transplant list and in May last year he received a new heart.
“Everything is going very well," he said. "I can get out and about again and take the kids and dog for a walk. And I hope to be able to go back to work.
“I have two children and the possibility that they might develop DCM is a concern for me.
"I only need to look at my children to understand the importance of this research. If they were to develop the condition, this could be the breakthrough that saves them.”
Professor Hugh Watkins on the development of a cure for inherited heart conditions
The breakthrough was made by a team of scientists, called CureHeart, who developed an injectable cure for inherited heart muscle conditions that could be available within a few years.
Their work using gene therapy technology was so successful it has now won a £30m award from the British Heart Foundation’s (BHF) Big Beat Challenge.
Professor Hugh Watkins, from the Radcliffe department of medicine at the University of Oxford and lead investigator of CureHeart, said: “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.
"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."
Inherited heart muscle diseases can cause the heart to stop suddenly or cause progressive heart failure in young people.
Every week in the UK, 12 people under the age of 35 die of an undiagnosed heart condition, very often caused by one of these inherited heart muscle diseases, also known as genetic cardiomyopathies.
Dr Christine Seidman, of Harvard University and co-lead of CureHeart, said: “Acting on our mission will be a truly global effort.
"We’ve brought in pioneers in new, ultra-precise gene editing, and experts with the techniques to ensure we get our genetic tools straight into the heart safely.
"It’s because of our world-leading team from three different continents that our initial dream should become reality.”
Prof Sir Nilesh Samani, medical director at the British Heart Foundation, said: “This is a defining moment for cardiovascular medicine.
"Not only could CureHeart be the creators of the first cure for inherited heart muscle diseases by tackling killer genes that run through family trees, it could also usher in a new era of precision cardiology.
"Once successful, the same gene editing innovations could be used to treat a whole range of common heart conditions where genetic faults play a major role.
"This would have a transformational impact and offer hope to the thousands of families worldwide affected by these devastating diseases.”
Want a quick and expert briefing on the biggest news stories? Listen to our latest podcasts to find out What You Need To Know