First trial of new cholesterol drug

3 October 2013        

Lab test

A new approach to cholesterol lowering that targets a regulator of harmful LDL cholesterol could offer new treatment options for patients with high cholesterol, researchers have concluded.

The drug, called ALN-PCS, blocks production of the cholesterol regulator PCSK9, a protein that destroys receptors that normally clear harmful cholesterol from the blood.

In a trial involving 32 healthy volunteers with mildly to moderately raised LDL, or ‘bad’ cholesterol, people were randomly assigned to receive either an injection of ALN-PCS or saline as a placebo.

A single dose of ALN-PCS was shown to cut levels of ‘bad’ cholesterol by up to 57 per cent.

This could offer new hope for those who are resistant to statins.


Our Medical Director, Professor Peter Weissberg, said: “People with extremely high cholesterol are at increased risk of a heart attack and this approach could offer new hope for those who are resistant to statins.

“These initial results add to growing evidence that blocking the action of a certain protein can dramatically lower ‘bad’ LDL cholesterol. More research is now needed to confirm this approach is both safe and effective at preventing heart attacks in the long term before it becomes widely available.”

The study was published in The Lancet.