Structure of the PCSK9 proteinWIKIMEDIA, EMWA new therapy has shown promise for patients who take cholesterol-lowering statins but suffer the side effects of muscle pain and weakness. The results of a Phase 1 clinical trial published online today (October 3) in The Lancet demonstrated that delivery of an siRNA formulated in a lipid nanoparticle safely and effectively lowered LDL cholesterol.
Researchers at Alynylam Pharmaceuticals in Cambridge, Massachusetts, developed an siRNA that targets proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that typically binds and degrades LDL receptors, thereby increasing the levels of circulating LDL cholesterol. The siRNA, dubbed ALN-PCS, works by preventing the production of PCSK9 protein. Study participants who received the highest dose of ALN-PCS showed a 70 percent decrease in circulating PCSK9 protein, and an average of a 40 percent reduction in LDL cholesterol. Additionally, these participants experienced only mild adverse effects, which were comparable to those experienced by participants who received a placebo.
An accompanying commentary on the clinical trial, written by John Burnett and Amanda Hooper who are both scientists from the University of Western Australia, identified shortcomings, such as the ...
