Oxford-led team awarded £30m to progress heart disease treatment
CureHeart, a research body comprising scientists from UK, US and Singapore have been awarded a £30million grant from the British Heart Foundation.
The group, who are developing an injectable cure for inherited heart muscle conditions, collected the prize having won the BHF Big Beat Challenge.
The Big Beat Challenge was launched to galvanise the international research community to identify a real-world challenge, significant unmet need or opportunity for game-changing innovation in cardiovascular medicine
The international CureHeart team is led by Professor Hugh Watkins of the University of Oxford.
Prof. EWatkins described the £30m award as a “once in a generation opportunity” to provide hope for families struck by these killer diseases.
The winning team, CureHeart, will seek to develop the first cures for inherited heart muscle diseases by pioneering revolutionary and ultra-precise gene therapy technologies that could edit or silence the faulty genes that cause these deadly conditions.
The winners were selected by an International Advisory Panel chaired by Professor Sir Patrick Vallance, Chief Scientific Advisor to the UK Government.
Inherited heart muscle diseases can cause the heart to stop suddenly or cause progressive heart failure in young people. Every week, 12 people under the age of 35 die of an undiagnosed heart condition1 in the UK. very often caused by one of these inherited heart muscle diseases, also known as genetic cardiomyopathies.
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Around half of all heart transplants are needed because of cardiomyopathy and current treatments do not prevent the condition from progressing.
It’s estimated that one in 250 people worldwide - around 260,000 people in the UK - are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children. In many cases, multiple members of the same family will develop heart failure, need a heart transplant, or are lost to sudden cardiac death at a young age.
BHF 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.
“The £30 million from the BHF’s Big Beat Challenge will give us the platform to turbo-charge our progress in finding a cure so the next generation of children diagnosed with genetic cardiomyopathies can live long, happy and productive lives.”
The team will take the revolutionary gene-editing technology of CRISPR to the next level by deploying ultra-precise techniques, called base and prime editing, in the heart for the first time. These ground-breaking approaches use ingenious molecules that act like tiny pencils to rewrite the single mutations that are buried within the DNA of heart cells in people with genetic cardiomyopathies.
The treatment has already been successful in animals with cardiomyopathies and in human cells. The scientific tram behind the research believe the therapies could be delivered through an injection in the arm that would stop progression and potentially cure those already living with genetic cardiomyopathies. It could also be used to prevent the disease developing in family members who carry a faulty gene but have not yet developed the condition.
Sir Patrick Vallance, Chair of the BHF’s International Advisory Panel and Government Chief Scientific Adviser, said: “CureHeart was selected in recognition of the boldness of its ambition, the scale of its potential benefit for patients with genetic heart muscle diseases and their families, and the excellence of the international team of participating researchers.”
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