A breakthrough gene-editing drug has sparked hopes for a cure for an aggressive form of heart failure that affects up to 50,000 British patients, landmark UK study results show.
This is the first time such a drug – which alters DNA in the body without having to remove tissue – has had results in patients. Only a single dose was required.
The breakthrough, announced yesterday at the American Heart Association conference in Chicago, opens the doors to similar treatments for a variety of conditions, including blindness, high cholesterol and even HIV.
The experimental drug, called NTLA-2001, is based on the Nobel Prize-winning CRISPR gene technology.
So far, 12 patients have been treated in the study, which was carried out by NHS cardiologists from the Royal Free Hospital in London and other clinics.
This is the first time such a drug – which alters DNA in the body without having to remove tissue – has had results in patients. (Picture from a picture agency)
‘This is the first-ever human attempt to edit genes in vivo or in the body, and our study demonstrates that this is possible and 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’t pump effectively because the muscle is weakened.
Symptoms include debilitating fatigue and breathlessness, and patients are often hospitalized due to its severity.
Although some patients are thought to be born with the genetic flaw that causes the disease, other people develop the mutation spontaneously as adults, meaning it can happen to anyone.
So far, 12 patients have been treated in the study, which was carried out by NHS cardiologists at the Royal Free Hospital in London (pictured) and other clinics
Doctors currently have a drug that can slow its progression and give patients a few more years of life, as well as 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.
Only an intravenous infusion of NTLA-2001 produced the same result. No significant side effects were observed.
Prof Gillmore, who heads the Center for Amyloidosis at University College London, said: ‘These results suggest it may halt the progression of the disease or even bring about improvement.
“If this study continues to be successful, the treatment may allow patients diagnosed early in the disease course to lead fully normal lives without the need for ongoing therapy.”
https://www.soundhealthandlastingwealth.com/uncategorized/gene-drug-breakthrough-offers-hope-of-a-cure-for-aggressive-form-of-heart-failure/ Breakthrough in gene drugs gives hope for a cure for the aggressive form of heart failure