Cholesterol could be cut in half with new ‘gene-silencing’ drug, study finds

LONDON — Researchers from Imperial College London have conducted a clinical trial which found that a new class of “gene-silencing” drugs can cut cholesterol levels for patients at risk of heart disease in half.

The drug — called inclisiran — uses RNA interference therapy, a technique known to “switch off” genes, to target those responsible for high levels of cholesterol.

“These initial results are hugely exciting for patients and clinicians,” says Kausik Ray, lead author of the study and professor of School and Public Health at Imperial, in a school release. “We appear to have found a versatile, easy-to-take, safe, treatment that provides sustained lowering of cholesterol levels and is therefore likely to reduce the risk of cardiovascular disease, heart attacks, and stroke.”

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Researchers say the drug “inclisiran” may be a breakthrough treatment in people with high-cholesterol, potentially cutting levels in half.

The study involved 497 patients with high cholesterol at risk for cardiovascular disease and gave them the drug via injection at differing doses and placebos. The results showed that just after one month of receiving a single treatment of the actual drug, levels of LDL cholesterol, or “bad cholesterol,” in participants reduced by 51 percent — better results than with statins, the current standard treatment for high cholesterol. (Just less than three-quarters of the participants were taking statins during the study.)

After six months, those who received one dose saw levels reduced by 42 percent, while the control group saw their cholesterol increase by two percent. Patients on two doses at six months saw levels drop by up to 53 percent.

When tested after eight months, researchers found all patients on the drug had lowered cholesterol levels, and there were no unforeseen recorded side effects.

High amounts of LDL cholesterol, can lead to plaque blocking the arteries, which can result in heart disease and stroke.

Researchers say inclisiran would be given just twice a year to patients, even if they’re already taking statins. The team touts the fact that the procedure would be helpful to patients who often forget to take their statin doses or in those who take the maximum amount allowed, yet still suffer from elevated cholesterol levels.

The study was published in the New England Journal of Medicine and presented this week at the American College of Cardiology’s 66th Annual Scientific Session in Washington.