First genetic treatment for inherited heart disease receives £30mn funding


Research into the first genetic cure for inherited heart muscle diseases which can be fatal to young people has received £30mn in funding from a British charity.

A team of scientists called CureHeart secured the amount from the British Heart Foundation — in one of its largest non-commercial grants — to finance the development of a new treatment for genetic cardiomyopathies which affect around 260,000 people in the UK.

The inherited conditions attack the heart in two ways, either by causing the organ to become progressively damaged over time, or to suddenly stop and cause cardiac death. Those with the gene stand a 50 percent chance of passing it on to their children.

Current treatments for an inherited cardiomyopathy, which can cause death at a young age, do not prevent the condition from progressing and it provides the need for around half of all heart transplants.

The £30mn grant has been entirely funded by the charity’s supporters, the British Heart Foundation said.

The team of scientists from the UK, US and Singapore was selected by a panel chaired by Sir Patrick Vallance, chief scientific adviser to the UK government. He said the team has been chosen “in recognition of the boldness of its ambition [and] the scale of its potential benefit for patients”.

The project aims to create an injectable cure using “ultra-specific” gene therapy technology that could “silence”, or edit, genes that cause inherited cardiac conditions.

The research to develop the cure will be carried out in labs in all three home countries of the researchers, with plans to launch early clinical trials within the next five years in the UK and US.

This is a “once in a lifetime opportunity” to find a cure for genetic cardiomyopathies, said Professor Hugh Watkins, lead investigator of CureHeart who also works at the University of Oxford.

“The team has already proven that these approaches are successful in animals with cardiomyopathies and human cells,” he said. They “could also be used to prevent the disease developing in family members who may carry a faulty gene but have not yet developed the condition”.

Creating the “world’s first genetic cure for a heart disease” would make a “giant leap” in an important area of ​​heart patient care, said Charmaine Griffiths, chief executive of the British Heart Foundation.

“However, we need the continued backing of our supporters to turn science like this into a reality for the millions of people around the world living with heart disease,” she added.



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