New drug effectively prevents first heart attacks and strokes

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A new study from researchers at Mass General Brigham has brought encouraging news for people at high risk of heart disease.

The large clinical trial found that adding a drug called evolocumab to standard treatment can significantly reduce the risk of major heart-related problems, such as heart attacks and strokes, even in people who have never had one before.

The results were shared at the American Heart Association Scientific Sessions and published in *The New England Journal of Medicine*, one of the most respected medical journals in the world.

Heart disease remains the number one cause of death globally. It often starts quietly, with cholesterol buildup in the arteries, which can block blood flow to the heart or brain over time. Doctors have long used cholesterol-lowering medications, such as statins, to reduce this risk.

However, some people still face a high chance of having a heart attack or stroke despite treatment.

This new study suggests that evolocumab, a type of medication known as a PCSK9 inhibitor, could offer an extra layer of protection for those most at risk.

Evolocumab works by targeting a protein in the liver called PCSK9. This protein normally limits how much “bad” cholesterol—known as LDL cholesterol—the liver can remove from the blood.

By blocking PCSK9, evolocumab helps the liver clear out more LDL cholesterol, keeping blood vessels healthier and reducing the chance of dangerous blockages.

While earlier research showed that PCSK9 inhibitors can prevent future heart problems in people who already had a heart attack or stroke, this new trial was the first to test whether the drug could prevent these events in people who hadn’t yet experienced one.

The trial, called VESALIUS-CV, involved more than 12,000 participants from different countries. All of them had either atherosclerosis (hardening of the arteries) or diabetes and high LDL cholesterol levels above 90 mg/dL. Importantly, none of the participants had a history of heart attack or stroke before joining the study.

Half of the participants received evolocumab injections every two weeks, while the other half received a placebo, in addition to their usual care. Researchers followed them for about 4.6 years to see how their health changed.

The results were clear: patients who took evolocumab had a 25% lower risk of suffering a serious cardiovascular event—such as a heart attack, stroke, or death from coronary heart disease—compared to those who received a placebo. Even more striking, the rate of heart attacks was reduced by 36% among those taking the drug.

Deaths were also slightly lower in the evolocumab group, at 7.9%, compared to 9.7% in the placebo group. These findings show that lowering LDL cholesterol to very low levels can have a powerful protective effect on heart health.

The study’s lead author, Dr. Erin Bohula, said the results offer hope for preventing the first heart attack or stroke in high-risk patients. She emphasized that prevention is key to saving lives and improving quality of life.

Dr. Marc Sabatine, another researcher involved in the study, added that patients taking evolocumab achieved average LDL cholesterol levels of around 40 mg/dL—levels that he believes should be the goal for people at high cardiovascular risk.

It’s important to note that most patients in the trial were already taking strong cholesterol-lowering drugs such as statins, while some were using milder medications or none at all. Regardless of their background treatment, the benefits of evolocumab were consistent across all groups.

However, the researchers also cautioned that because most participants were white, more studies are needed to see if the results apply to other populations.

When reviewing these findings, the results are impressive. The study demonstrates that aggressively lowering LDL cholesterol—especially using modern medications like evolocumab—can have major benefits even before a first heart event occurs.

This approach could transform how doctors prevent heart disease, shifting focus from treatment after a heart attack to stopping it before it happens. Still, researchers point out that while evolocumab shows strong promise, long-term studies and broader population data will be essential to confirm safety and effectiveness for everyone.

In the meantime, the study adds to growing evidence that cholesterol management is one of the most powerful tools for protecting the heart. For people living with diabetes, atherosclerosis, or high cholesterol, this new drug could represent a life-changing step toward a healthier future.

If you care about stroke, please read studies about how to eat to prevent stroke, and diets high in flavonoids could help reduce stroke risk.

For more health information, please see recent studies about how Mediterranean diet could protect your brain health, and wild blueberries can benefit your heart and brain.

The study is published in New England Journal of Medicine.

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