Cholesterol lowering drug performs well in clinical trial

Statins could be replaced or used as a complementary treatment for patients with high cholesterol, following the positive results of a clinical trial into a new class of drugs. The new drugs that target a newly discovered harmful cholesterol regulator within the body could ultimately replace the traditional treatment method. 

It was found that the levels of LDL cholesterol (bad cholesterol) was reduced by around 57 per cent in healthy volunteer participants in the study who received a single dose of the small interfering ribonucleic acid (siRNA) drug candidate ALN-PCS. On average the reduction was around 40 per cent more than the participants that were treated with a placebo, according to the results that were published in the journal 'The Lancet'.

Kevin Fitzgerald from Alnylam pharmaceuticals, one of the developers of siRNA, said: “If successfully developed, this class of drugs could be an alternative for the one in five people who are resistant to statins, or be combined with statins to produce even greater effects for the many others for whom the current first line treatment does not lower cholesterol enough.”

The new drug works by blocking production of the proprotein convertase subtilisin/kexin type nine (PCSK9) cholesterol regulator, which works to destroy low-density lipoprotein (LDL) receptors. When these receptors are present and are functioning, they remove LDL cholesterol from the blood.

Previous studies have identified that an increase in the concentration or activity of PCSK9 can cause genetic mutations that can lead to a rise in LDL cholesterol. This in turn can cause plaque to build-up within blood vessels and result in health complications. However, mutations that reduce the amount of PCSK9 in the body can work to lower cholesterol.

The drug that was developed to build upon this research was tested upon 32 participants. Each of the volunteers were healthy and aged between 18 and 65, all with slightly raised levels of LDL cholesterol. Each participant received six doses of ALN-PCS intravenously or a placebo. 

It was found that the ALN-PCS was well tolerated and resulted in no significant changes to liver inflammation or function.