Brits at risk of heart attacks to test radical drug that lowers your ‘bad’ cholesterol
- Verve-101 removes a hereditary flaw that causes life-threatening cholesterol
- Statins lower levels by 30 to 50 percent, but only work if taken regularly
- British volunteers will join this year, along with Americans for the 40-person trial
- All 40 patients inherited high cholesterol and had a heart attack
Britons at high risk of heart attacks will join a trial of a gene-editing drug that promises to permanently lower cholesterol.
The groundbreaking drug – codenamed Verve-101 – removes a small genetic defect that causes life-threatening levels of the fatty substance in the blood.
A patient in New Zealand recently became the first in the world to receive an IV. British volunteers will participate later this year, along with others from America, as part of a 40-person trial to test whether the treatment reduces ‘bad’ LDL cholesterol in people — and by how much.
Previous studies in monkeys sharing the same cholesterol gene, PCSK9, have shown remarkable success. LDL fell by nearly 70 percent at the same levels still seen two years later, suggesting the drop could be permanent.
The gene-editing drug – codenamed Verve-101 – removes a small hereditary flaw that causes life-threatening levels of the fatty substance in the blood
Cardiologist and geneticist Dr Sekar Kathiresan, co-founder of Verve Therapeutics, the team behind Verve-101, said, “If it works and is safe, this is the answer to heart attacks – this is the cure.”
Each of the 40 patients in the study will have naturally inherited high cholesterol from a parent – a condition known as familial hypercholesterolemia (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 greater the chance of preventing heart attacks.
While statins are effective at lowering cholesterol — usually lowering levels by 30 to 50 percent — 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 have had a heart attack are taking cholesterol-lowering drugs, leading to “additional heart attacks, strokes and even deaths.”
Verve-101 uses a precision gene editing technique called CRISPR basic editing to erase a single “wrong” letter from the DNA code before replacing it with a “harmless” letter. The effect is that cells in the liver, where cholesterol is made, produce much less of the fatty LDL that can block arteries.
Like Pfizer-BioNTech and Moderna’s Covid vaccines, Verve-101 uses messenger RNA (mRNA) to instruct cells.
In the case of the vaccines, the mRNA tells them to mass-produce the Covid virus’s spike protein so that the immune system can recognize the real thing when it occurs. With Verve-101, the mRNA tells liver cells to make two proteins — one to locate the PCSK9 gene and another to edit it.
dr. Kathiresan said a US study showed that only half of people who have had a heart attack are taking cholesterol-lowering drugs, leading to “additional heart attacks, strokes and even deaths”
Like the vaccines, the mRNA is wrapped in a small fat globule called a ‘lipid anode’ that helps it enter cells. dr. Kathiresan said he was “optimistic” that UK regulators would let the process begin later this year. If all goes well, Verve will publish the first results in 2023 and hopes the drug will be available in 2028.
Cholesterol is made by the liver and carried throughout the body in the blood. The body needs cholesterol to build healthy cells, but high levels can increase the risk of heart disease.
In FH, there is an error in one of the genes involved in removing cholesterol from the blood, which can lead to a buildup.