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The use of statins could be replaced “within a decade” by the gene editing drug Verve-101
Drug alters DNA inside cholesterol-regulating cells to stop production
So far, nine have been successfully treated by Verve-101 and testing will be expanded
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Scientists say the statins that millions of people take daily to protect against heart attacks and strokes could be replaced “within a decade” by a gene-editing jab.
The extraordinary new drug manipulates the DNA inside cholesterol-regulating cells to stop production of the substance after a single dose.
It was presented yesterday at the American Heart Association’s annual Scientific Sessions in Philadelphia.
Trial results showed that the single injection reduced “bad” LDL cholesterol by more than 50 percent.
Professor Karol Watson, a cardiologist based in Los Angeles, predicted “a brave new world” in heart treatment: “This trial showed that with (gene) editing you can reduce LDL in a lasting way.”
Daily use of statins could become a thing of the past thanks to a revolutionary new gene-editing drug
The experts behind the treatment, called Verve-101, believe its effect is long-lasting and possibly permanent.
Admitting that the research is at an early stage, they said their findings open up the possibility that high cholesterol can be effectively “cured” or prevented from becoming a problem in the first place.
“This is a potential game-changer in the treatment of heart disease,” said Dr. Sekar Kathiresan, one of the cardiologists behind Verve-101.
He added: “We have shown that a single therapy could be the future.”
So far, nine patients with familial hypercholesterolemia, a genetic condition that causes very high levels of LDL that trigger heart attacks, have been successfully treated.
The trials will soon be expanded to broader groups, including those with existing heart disease. “We hope the treatment will be available by the end of the decade,” Dr. Kathiresan added.
The drug is based on CRISPR technology that can remove and rewrite sections of DNA within cells.
Unlike other CRISPR-based treatments, which can cost more than a million pounds per dose, Verve-101 can be mass-produced, Dr Kathiresan added.
So far, nine patients have been successfully treated and the trials will soon be extended to larger groups.
He said: “We hope it will not cost more than other one-off heart procedures such as stents or heart bypass operations.”
Statins are one of the most prescribed medicines in the UK: around eight million adults take them and more than 71 million statins are prescribed each year.
The drugs can be remarkably effective, reducing the risk of heart attack by up to 50 per cent and the risk of stroke by a third, costing as little as £15 per patient per year.
Professor Watson said the biggest concern about the new treatment was safety: “If you’re editing the human genome, it’s a big deal, so we have to make sure it’s super safe.”
Dr. Andrew Bellinger, chief scientific and medical officer of Verve Therapeutics, said large-scale trials involving thousands of heart disease patients were scheduled to begin in 2025.