An alternative treatment for people suffering from high cholesterol has moved a step closer after a successful initial trial.
Investigator Kevin Fitzgerald, from Alnylam Pharmaceuticals, has developed a small interfering ribonucleic acid (siRNA) drug called ALN-PCS.
In the first trial on healthy volunteers, ALN-PCS was found to reduce levels of harmful low-density lipoprotein (LDL) cholesterol by an average of 40% compared to another group who took a placebo drug. In some cases, there was a 57% reduction in the amount of LDL.
It is hoped that ALN-PCS will provide either an additional, or an alternative, treatment option for the millions of people who currently take statins.
While statins work well in many cases, doctors have found that sometimes the drugs do not lower cholesterol sufficiently. It is also estimated that statins do not work on about 20% of patients, which would make ALN-PCS a very useful alternative.
In contrast to statins, ALN-PCS acts by blocking production of the cholesterol regulator PCSK9, which can be dangerous as it destroys LDL receptors that usually clear bad cholesterol from the blood. This in turn means there is greater risk of blood vessels narrowing, increasing the risk of people suffering a stroke.
A total of 32 volunteers, aged from 18 to 65, were involved in the study, with half given differing doses of ALN-PCS and the rest a saline tablet in order to provide a comparison.
The volunteers taking ALN-PCS did not report significantly more adverse events and researchers found there were no changes to their liver function or inflammation.
In the case of volunteers given the greatest dose of ALN-PCS (0.400mg/kg), plasma PCSK9 levels fell by an average of 70% while LDL cholesterol was down by an average 40%, compared to the control group.
Mr Fitzgerald said that the trial represented the first time that a siRNA drug had performed as well as the first line treatment of statins, which on average lead to a 36% to 53% fall in LDL cholesterol.
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