Gene drug breakthrough for heart patients
A PIONEERING gene-editing drug has raised hopes of a cure for an aggressive form of heart failure that affects up to 50,000 British patients, landmark UK trial results have shown.
This is the first time such a drug – which edits DNA in the body without any tissue having to be removed – has yielded results in patients. Just a single dose was required.
The breakthrough, announced yesterday at the American Heart Association conference in Chicago, throws open the doors to tackling a host of conditions in a similar way, including blindness, high cholesterol and even HIV.
The experimental drug, called NTLA-2001, is based on Nobel Prize-winning genetic engineering technology known as CRISPR.
So far 12 patients have been treated in the trial, carried out by NHS cardiologists at The Royal Free Hospital, London, and other clinics. ‘This is the first-ever human trial of gene editing in vivo, or in the body, and our study proves it’s possible and also safe,’ said lead researcher Professor Julian Gillmore.
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 debilitating fatigue and breathlessness, and patients are often hospitalised due to its severity. Although some patients are thought to be born with the genetic fault that causes the disease, other people develop the mutation spontaneously as adults, meaning it can happen to anyone.
At present, doctors have a medication that can slow its progression, giving patients a few more years of life, and other treatments to ease some of the worst symptoms. However it must be given once every three weeks in hospital for the rest of the patient’s life.
Just one intravenous infusion of NTLA-2001 produced the same result. No significant side effects were seen.
Prof Gillmore, who runs the University College London Centre for Amyloidosis, said: ‘These results indicate it may stop disease progression or even bring about improvement.
‘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.’