‘Gene-silencing’ drug helps lower cholesterol

One dose of the experimental drug cut levels of the “bad” form of cholesterol in healthy volunteers by up to 57 per cent.

Scientists believe the drug, known as ALN-PCS, could provide an alternative treatment for patients with high cholesterol who do not respond well to conventional statins.

The drug employs a concept known as RNA interference (RNAi). Small pieces of the genetic molecule RNA are used to shut off a particular gene by interfering with its coded instructions. In this case, the gene in question produces a protein called PCSK9 that reduces the body’s ability to clear away harmful cholesterol naturally.

“Bad” cholesterol, low-density lipoprotein (LDL), plays a major role in the build up of scale-like deposits on the walls of arteries that impede blood flow and increase the risk of heart attacks and strokes.

Researchers conducting a pilot trial recruited 32 healthy volunteers aged 18 to 65 who all had mildly to moderately raised levels of LDL.

They were randomly assigned either to receive injections of the new drug, or a non-active placebo in the form of a salt water solution.

Infusion of ALN-PCS led to a rapid dose-dependent drop in blood-levels of PCSK9. In volunteers given the highest dose, LDL cholesterol levels also fell by an average of 40 per cent and as much as 57 per cent compared with the placebo group.

The findings are published in medical journal The Lancet.