Gene drug discovery offers hope for cure for aggressive form of heart failure

Gene drug discovery offers hope for cure for aggressive form of heart failure affecting up to 50,000 UK patients

  • Pioneering drug changes DNA in the body without removing any tissue
  • Only one dose was needed for the twelve patients treated in the UK trial
  • May be possible to use same technology for blindness, high cholesterol and HIV

A pioneering gene-editing drug has raised hopes for a cure for an aggressive form of heart failure that affects up to 50,000 UK patients, results from a landmark UK trial have shown.

This is the first time that such a drug – which edits DNA in the body without any tissue having to be removed – has shown results in patients. Only one dose was needed.

The breakthrough, announced yesterday at the American Heart Association conference in Chicago, paves the way for tackling a host of conditions in the same way, including blindness, high cholesterol and even HIV.

The experimental drug, called NTLA-2001, is based on the 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 in London and other clinics.

This is the first time that such a drug – which edits DNA in the body without any tissue having to be removed – has shown results in patients. (File image)

“This is the first-ever human trial of gene editing in vivo, or in the body, and our study proves that it is possible and also safe,” said lead researcher Professor Julian Gillmore.

Unlike a heart attack, which occurs 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 weak.

Symptoms include debilitating fatigue and shortness of breath, and patients are often hospitalized due to its severity.

Although some patients are thought to be born with the genetic defect that causes the condition, other people develop the mutation spontaneously in adulthood, which means it can happen to anyone.

So far, 12 patients have been treated in the trial, carried out by NHS cardiologists at the Royal Free Hospital in London (pictured) and other clinics

So far, 12 patients have been treated in the trial, carried out by NHS cardiologists at the Royal Free Hospital in London (pictured) and other clinics

Right now, doctors have a drug that can slow its progression, giving patients a few extra years of life, and other treatments to relieve some of the worst symptoms. However, it must be administered once every three weeks in the hospital for the rest of the patient’s life.

A single intravenous infusion of NTLA-2001 produced the same result. No significant side effects were observed.

Professor Gillmore, who heads the University College London Center for Amyloidosis, said: “These results indicate that it can halt disease progression or even lead to improvement.”

“If this trial continues to be successful, the treatment could allow patients diagnosed early in the disease to lead completely normal lives without the need for ongoing treatment.”

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