The new drug is an siRNA (small interfering RNA) formulated in a lipid nanoparticle (this is a drug designed to knock-out specific genes in the human body). Trials have shown that the drug can be safely and effectively administered and that it can lower
LDL (low-density lipoprotein) cholesterol. LDL particles are often informally called bad cholesterol because they can transport their content of many fat molecules into artery walls, attract macrophages and drive atherosclerosis.
The new drug has shown promise for patients who take cholesterol-lowering statins but suffer the
side effects of muscle pain and weakness. The trial was carried out by Alynylam Pharmaceuticals in Cambridge, Massachusetts.
The new drug works by targeting a protein in the body called
PCSK9. This protein increases the levels of circulating LDL cholesterol in the body.
Commenting on the research, Jean-Claude Tardif of the Montreal Heart Institute in Canada, is quoted by
Nature as saying: "It’s among the first demonstrations of RNA interference working in humans in a safe and tolerable way. It’s very exciting."
The results of the Phase 1 clinical trial have been
published in journal The Lancet. The paper is titled "Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial."
