The gene-editing drug - codenamed Verve-101 - removes a tiny inherited defect that causes life-threatening amounts of the fatty substance in the blood

Britons at risk of heart attacks test radical drug that lowers their ‘bad’ cholesterol

Britons at risk of heart attacks test radical drug that lowers their ‘bad’ cholesterol

  • Verve-101 removes an inherited defect that causes life-threatening cholesterol
  • Statins reduce levels by 30-50%, but only work if taken regularly
  • British volunteers will join this year, along with Americans for the trial of 40 people
  • All 40 patients will have inherited high cholesterol and had a heart attack

Britons at high risk of heart attack are to take part in a trial of a gene-editing drug that promises to permanently lower cholesterol.

The breakthrough drug – codenamed Verve-101 – removes a tiny inherited defect that causes life-threatening amounts of the fatty substance in the blood.

A New Zealand patient recently became the first in the world to receive an infusion. British volunteers will join later this year, along with other Americans, in a 40-person trial to test whether the treatment reduces ‘bad’ LDL cholesterol in humans – and by how much.

Previous studies in monkeys, which share the same cholesterol gene, PCSK9, have shown remarkable success. LDL dropped almost 70%, with the same levels still seen two years later, suggesting the drop may be permanent.

The gene-editing drug – codenamed Verve-101 – removes a tiny inherited defect that causes life-threatening amounts of the fatty substance in the blood

Cardiologist and geneticist Dr Sekar Kathiresan, who co-founded Verve Therapeutics, the team behind Verve-101, said: “If it works and is safe, it’s the answer to heart attacks – it’s the cure.”

Each of the 40 patients in the trial will have inherited naturally high cholesterol from a parent – a condition known as familial hypercholesterolaemia (FH) which affects one in 250 Britons. Each will also have had a heart attack before.

Studies show that the longer LDL is kept low, the better the chances of preventing heart attacks.

While statins are effective at lowering cholesterol – typically lowering levels by 30-50% – they must be prescribed by a doctor and they only work if people take them regularly.

But Dr Kathiresan said a US study showed that only half of people who had a heart attack took cholesterol-lowering drugs, leading to “additional heart attacks, strokes and even death”.

Verve-101 uses a precision gene editing technique called CRISPR base editing to erase a single “faulty” letter from the DNA code before replacing it with a “harmless” one. The effect is that cells in the liver, where cholesterol is made, produce far less fatty LDL which can block arteries.

Like the Covid vaccines from Pfizer-BioNTech and Moderna, Verve-101 uses messenger RNA (mRNA) to instruct cells.

In the case of vaccines, the mRNA tells them to mass produce the spike protein of the Covid virus, so the immune system can recognize the real thing when it comes along. With Verve-101, the mRNA tells liver cells to make two proteins – one to locate the PCSK9 gene and another to modify it.

Dr Kathiresan said an American study showed that only half of people who have had a heart attack take cholesterol-lowering drugs, resulting in

Dr Kathiresan said an American study showed that only half of people who had a heart attack took cholesterol-lowering drugs, leading to “additional heart attacks, strokes and even death”.

As with vaccines, mRNA is wrapped in a tiny bubble of fat called a “lipid nanoparticle” that helps it enter cells. Dr Kathiresan said he was “optimistic” that UK regulators would allow the trial to start later this year. If all goes well, Verve will publish the first results in 2023 and hopes to have the drug available by 2028.

Cholesterol is made by the liver and carried around the body in the blood. The body needs cholesterol to build healthy cells, but high levels can increase the risk of heart disease.

With FH, there is a defect with one of the genes involved in removing cholesterol from the blood, which can lead to a buildup.

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