New Rx for Lower Cholesterol

New type of treatment shown to be effective in lowering cholesterol according to recent trial

/ Author:  / Reviewed by: Robert Carlson, M.D Beth Bolt, RPh

(RxWiki News) High cholesterol is a problem for many people and, over time, it can result in life-threatening heart problems. The first line of defense — statins — are not always helpful for every patient, but a recent study may be paving the way for new types of treatment.

Cholesterol is normally removed from the blood by cholesterol receptors. A natural biological substance normally destroys some of those receptors. The clinical trial tested a drug that may prevent the destroying substance from working properly so that the body can more efficiently take cholesterol out of the blood.

The participants received different doses of the drug. In the group that received a higher dose, participants experienced a significant reduction in bad cholesterol.

The researchers suggested that the new type of treatment could be effective either in addition to statins or as a replacement.

"If you have high cholesterol, talk to your doctor about treatment options."

Kevin Fitzgerald, PhD, of Alynlam Pharmaceuticals, led the study to see if a new type of drug could help lower bad cholesterol.

High cholesterol occurs when there are elevated levels of fat in the blood. Over time, the cholesterol can build up on an artery wall, which can cause blood clots, a heart attack, or stroke.

High cholesterol is usually treated with statins, a type of drug that prevents the liver from producing cholesterol by interfering with an enzyme. However, statins do not work for everyone with high cholesterol.

The study tested the drug ALN-PCS, which is a small interfering ribonucleic acid, or siRNA. The siRNA interferes with the production of PCSK9, a substance in the body that regulates cholesterol. PCSK9 destroys cholesterol receptors that normally take cholesterol out of the blood.

Thirty-two people participated in the trial from between 18 to 65 years old. Each of the participants had slightly raised cholesterol levels. Of the participants, 24 received one dose of ALN-PCS and eight received a placebo — or fake medicine.

The 24 participants who received the medication were assigned to receive one of six doses. The drug was delivered intravenously.

Participants were seen for follow-up visits in the 180 days after they received the dose of the drug.

The researchers looked for any dangerous or otherwise uncomfortable side effects of the drug. They also looked at the effects of the drug on PCSK9 and bad cholesterol levels.

They found that there were no serious adverse side effects of the drug in the group that had been dosed with ALN-PCS. Some mild side effects were reported, like a rash, but they occurred with similar frequency in the placebo group.

Additionally, after the administration of the drug, levels of PCSK9 — the substance that prevents cholesterol receptors from clearing cholesterol from the bloodstream — were significantly reduced.

The drug led to lower levels of bad cholesterol, especially when given at the higher doses. The group given a .4 mg/kg dose of ALN-PCS had a 40 percent reduction in bad cholesterol from baseline in comparison to the placebo.

The researchers concluded that the treatment seemed to be effective in reducing cholesterol levels. Because the trial size was relatively small, they called for further research to be done on the effectiveness of ALN-PCS and the best dosage amounts.

The study was published in The Lancet on October 3.

The research was funded by Alnylam Pharmaceuticals. Some of the researchers declared that they are employed by Anylam Pharmaceuticals.

Review Date: 
October 1, 2013
Last Updated:
October 3, 2013