Experimental Cholesterol-Lowering Drug Shows Promise

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One in six Americans have high cholesterol, but one in five can’t take the popular statin medications to keep their levels down.

Statins are among the most prescribed drugs in the U.S., and while they are effective in keeping cholesterol in check, their side effects, which include muscle damage, higher blood sugar levels, and possible memory deficits, mean some people should avoid them. For them, scientists are working on a new class of medications that may lower cholesterol with fewer adverse effects.

New research from Alnylam Pharmaceuticals, published in the journal Lancet, shows that a single dose of the compound ALN-PCS can cut levels of harmful LDL cholesterol in healthy participants by 36% to 56%. That’s about 40% more than what participants using a placebo experienced.

(MORE: Statin Drugs Linked to Lower Risk of Cognitive Decline)

The study involved 32 volunteers with mild or moderately high LDL cholesterol, who were randomly assigned to either six doses of intravenous ALN-PCS or a placebo. ALN-PCS blocks the production of a protein called PCSK9, which regulates cholesterol levels in the blood by destroying the receptors that are supposed to bind to and clear circulating LDL. According to the study authors, this is the first cholesterol-lowering drug candidate of its kind to show cholesterol-lowering benefits comparable to those of statins.

Because intravenous drugs are more difficult to take than a pill, the drug’s developers are hoping to find an oral formulation for their compound. They will also need to  test their agent in larger studies, at various doses to see if it can become a viable alternative to statins for people who need one.