A new gene-silencing drug that can lower cholesterol levels is proving promising in initial clinical test results. The treatment was shown to reduce the levels of low-density lipoprotein (LDL) cholesterol in patients' blood by up to 51 percent in the month following one single treatment. The drug, named Inclisiran, utilizes a technique called RNA interference therapy which targets, and switches off, a specific gene known to be responsible for elevated LDL levels.

A phase 2 clinical trial recruited 497 patients with high cholesterol, 73 percent of which were already taking statins, the current standard treatment for those at risk of cardiovascular disease.

Those patients who received a single dose found cholesterol levels reduced by on average 42 percent, six months following the dose. A group given two doses across three months found levels reduced up to 52 percent after six months. All the non-placebo groups in the study saw their cholesterol levels staying low for at least eight months following the treatment with no additional side effects.

"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," says lead author of the study, Professor Kausik Ray. "These reductions are over and above what can be already be achieved with statins alone or statins plus ezetemibe, another class of cholesterol-lowering drug."

A recently approved monoclonal antibody treatment called Evolocumab has also been finding notable success in reducing LDL cholesterol levels. Evolocumab has also had extraordinary success in broad trials, with LDL cholesterol reductions of up to 60 percent in patients undergoing that treatment.

The novel strength that Inclisiran is showing over Evolocumab is the minimal dosing regime. Ultimately, the authors of the Inclisiran study see the treatment as being as innocuous as visiting your GP once or twice a year for an injection, in much the same way as one would receive a flu vaccination.

"We believe that these clinical visits might only be twice a year at most, so ultimately, they are more convenient and more effective for patients and their health," explains Professor Ray.

The next stage of the trials will increase the volume of patients and asses the long-term safety of the treatment. The study notes that they are in the early stages of this clinical work, adding that much more research is needed before this drug will be publicly available. For those with cholesterol problems though, it seems there are a number of exciting new developments on the horizon.

The team's study is published in the New England Journal of Medicine.

Source: Imperial College London