The study, which was led by Imperial College London’s School of Public Health, tested the effectiveness and safety of bempedoic acid – an oral medication. Bempedoic acid works by blocking a key enzyme in the body, called ATP-citrate, used to make cholesterol. It works in a similar way to statins – the current most common medication for high cholesterol. But statins can have undesirable side effects like headaches, muscle pain and stomach problems, making them difficult for some people to tolerate.
The study involved more than 2,200 patients from around the world with high cholesterol, who were already taking cholesterol-lowering drugs.
The participants were either given bempedoic acid, or took a placebo for one year.
After three months of treatment, researchers found bempedoic acid reduced patients’ LDL (bad) cholesterol levels by an average of 18.1 per cent compared to the placebo group.
The drug was also shown to be well-tolerated by patients, with some increased incidence of gout due to slight increases in levels of uric acid in the blood.
However, there was no increased incidence of serious health conditions between the two groups.
In a second study, researchers looked at data from more than half a million people and used genetic markers to model the likely effects of the treatment over a longer period, comparing these to the effects of statins.
They found the effects of inhibiting the enzyme over a longer time scale reduced the risk of cardiovascular disease with no obvious adverse effects of blocking the pathway.
The benefit was identical to that expected though blocking the enzyme targeted by statins.
According to the researchers, bempedoic acid could be taken by patients in addition to statins and other drugs, or taken alone by people who are unable to tolerate statins.
“One of the key advantages of bempedoic acid is supposed to be that it shouldn’t cause the muscle side effects reported by some statins users, as it is taken up by the liver and needs to be converted into its active form via an enzyme only found in the liver,” said said Professor Kausik Ray, from Imperial College London’s School of Public Health.
“Once converted to the active form the drug cannot leave the liver, so it can’t enter muscles and hence could be of considerable advantage for some.
“It could be an option for patients who are unable to tolerate statins at higher doses, or at all.
“Our genetic studies suggest that the benefit on prevention of heart disease and strokes in ongoing trials should be identical to that achieved through statins.
“Overall, these latest studies show that not only is the treatment generally well-tolerated being comparable with placebo, and potentially safe over longer periods, but that when added to high intensity statin treatment it can help to further reduce LDL cholesterol levels.”
The research has been published in the New England Journal of Medicine.