I had only two years to live, but gene editing has given me hope
ONE of the first heart-failure patients to benefit from a pioneering gene-editing drug said the treatment had given him hope when he was faced with a bleak prognosis.
Married father-of-three
Chidi Egbochue, 57, had suffered gradually worsening health until he was diagnosed with the condition in February and told he could have as little as two years to live.
The City lawyer, who lives in Uckfield, East Sussex, with wife Samantha, 46, and their daughters Amarachi, six, and Nkechi, eight, and son Chinedu, 11, had worse news to come – his illness had been caused by a genetic disease called hereditary ATTR amyloidosis, meaning that it was likely he had passed it on to at least one of his children.
However, having been put on a trial for the medication NTLA-2001, which corrects the faulty DNA that causes the problem with a single dose, Chidi now feels ‘optimistic’.
The experimental drug is based on Nobel Prize-winning genetic-engineering technology known as CRISPR, which allows sections of faulty DNA inside cells to be manipulated.
Early-stage trial results, reported exclusively last week in The Mail on Sunday, showed that the treatment is highly effective in halting the illness in its tracks.
‘The strong likelihood is that there will be no further damage to my heart,’ Chidi said. ‘And there is a possibility, over time, that the existing damage will be reversed. I hope, in the future, if one of my children develop the condition, it will be simple to cure.’
Hereditary ATTR amyloidosis causes the liver to produce defective proteins that form clumps in the cardiac muscle, leading to heart failure. Unlike a heart attack, which happens when the blood supply to the heart is suddenly blocked, heart failure is a chronic condition in which the heart can no longer pump effectively because the muscle has become weakened. Symptoms include crushing fatigue and extreme breathlessness.
There are roughly one million sufferers in the UK, with one in five adults expected to develop the condition. It is primarily caused by blocked arteries reducing blood flow to the heart, but up to one in 20 cases
‘It may allow patients to lead normal lives’
are believed to be caused by ATTR amyloidosis.
So far 12 patients, including Chidi, have been treated in the trial, which is being carried out by NHS cardiologists at The Royal Free Hospital in North London.
Although some patients are born with the genetic fault that causes ATTR amyloidosis, other people develop the mutation spontaneously as adults, meaning it can happen to anyone.
Professor Julian Gillmore, who runs the University College London Centre For Amyloidosis, said: ‘If this trial continues to be successful, the treatment may permit patients who are diagnosed early in the course of the disease to lead completely normal lives without the need for ongoing therapy.’