Hush now: New gene-silencing drug cuts cholesterol by half in clinical trial

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Nearly 32 percent of adults in the United States have high cholesterol and just less than a third of those have their condition under control, according to the Centers for Disease Control and Prevention. Although Americans have made progress over the last 15 years, the condition is still well above healthy levels.

But a new treatment may help tackle the condition at the source by silencing a gene that contributes to high cholesterol. In clinical trials, a drug known as inclisiran, developed by researchers from Imperial College London, reduced cholesterol by up to 51 percent.

Through RNA interference, inclisiran works by binding to messenger RNA and causing their message to degrade. “By breaking the link the message between the gene and translating the message into a protein, you silence the gene,” Kosh Ray, Imperial College professor and lead author of the study published in the New England Journal of Medicine, told Digital Trends.

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In the study, Ray and his team administered inclisiran or a placebo to almost 500 patients with high levels of low-density lipoprotein (aka bad) cholesterol, which has been shown to increase the risk of heart attacks and strokes. Many of the patients received both inclisiran and statins. After just one month, the subjects who received inclisiran showed a decline in cholesterol levels by up to 51 percent.

Ray explained why the combined statin and inclisiran doses worked so effectively: “The point about statins and why this is better is compliance,” he said. “If patients on statins do not comply with daily regimens they have huge variation in LDL cholesterol so less average reduction over time so less benefit . When we gave our drug patients were locked down to sustained reductions in LDL over time so compliance is not an issue. Even monoclonal antibodies to PCSK9 do not go to the source so if you forget your injection every two weeks the LDL goes up.”