The Latest Cholesterol Treatment Breakthroughs

Learn what’s new in research for managing this condition, including medications currently under study.

by Lara DeSanto Health Writer

High cholesterol increases the risk of heart disease, the leading cause of death in the United States, and stroke (the fifth most-common cause), according to the Centers for Disease Control and Prevention (CDC). So it’s no wonder that medical researchers have dedicated a great deal of energy to finding new and improved treatment options. We spoke to the experts to get their take on the most exciting breakthroughs in cholesterol treatment research, from historic drug developments to medications that may be just around the corner.

Statins: The Go-To Drug Treatment for Cholesterol

The first treatment for high cholesterol is typically a prescription for a healthy diet, weight loss, and exercise, says Randy Zusman, M.D., cardiologist and director of the division of hypertension at the Massachusetts General Hospital Heart Center in Boston, MA. But medications can play an important role in treatment as well—especially when lifestyle changes simply aren’t cutting it. The medication doctors typically suggest first? Statins, says Dr. Zusman.

When they were introduced in the 1980s, statins changed the cholesterol-treatment game forever. In fact, at high-intensity doses, statins can reduce LDL (aka “bad”) cholesterol by 50% or more, according to the American College of Cardiology. They prevent cholesterol from forming in the liver and can lower triglycerides as well as raise HDL cholesterol (the “good” kind of cholesterol).

“Statins have proven highly effective at reducing heart attack and stroke,” Dr. Zusman says. Since the ‘80s, a multitude of low-cost options have hit the market. In some cases, people with high cholesterol may benefit from taking another medication in combination with their statin for even greater benefit.

To this day, statins are still the first-line medication for the treatment of high cholesterol, but new med options are on the horizon—good news for those who are unable to tolerate statins due to side effects like muscle aches, says Dr. Zusman. Let’s take a look at what else your doctor might suggest.

Inclisiran: The Newest Approach

Another class of drugs already available to treat high cholesterol are injectable medications called PCSK9 inhibitors. The two drugs for this are alirocumab and evolocumab. A third promising drug currently under study that targets PCSK9 is Inclisiran, says Dr. Zusman. It works a bit differently from the PCSK9 inhibitors.

Inclisiran was only recently approved for use in Europe, explains James Underberg, M.D, lipid specialist at the Center for the Prevention of Cardiovascular Diseases at NYU Langone Health in New York City, and it’s expected to be approved in the United States soon.

“Like some of the coronavirus vaccines, Inclisiran is an RNA-based product that effectively disrupts the pathways that lead to cholesterol production and ultimately elevate cholesterol levels,” Dr. Zusman explains. “We already know from data that it’s effective at reducing cholesterol levels, but we’re awaiting data about whether it protects against heart attack and stroke.”

Another big plus of this drug? It may only need to be taken once or twice a year to provide significant benefits, Dr. Underberg says.

Bempedoic Acid: Statin-like Results Without the Side Effects?

Bempedoic acid is a new drug for cholesterol treatment that was approved in 2020, per the U.S. Food and Drug Association (FDA). It works in a similar fashion to statins, explains Dr. Underberg. However, there are a few key differences—mainly that it’s not as effective as statins at lowering cholesterol. The trade-off comes when you look at side effects.

“One of the common side effects of statins—which you see in about 5% to 10% of people who take them—are muscle aches,” Dr. Underberg explains. “One reason we think that happens is statins act both in the liver and the muscle, where bempedoic acid only acts in the liver, so it may benefit people who can't take statins from that muscular perspective.” Bempedoic acid is taken orally, which some people may prefer over the injectable PCSK9 inhibitors, Dr. Underberg adds.

Unlike PCSK9 inhibitors, Dr. Underberg says that bempedoic acid hasn’t yet been shown to reduce heart attack and stroke risk in a clinical trial—but another trial is in progress. And it’s kind of a big deal.

“This is the first trial with heart disease ever done since statins came out where the drug was not studied on top of a statin,” Dr. Underberg explains. “This drug is being studied in patients who can’t take statins, so we’ll know the effect of it, independent from statins. This is intriguing because all the other drugs—like ezetimibe and PCSK9 inhibitors—are indicated to be used on top of statins.”

Pure EPA Fish Oil

It sounds basic, but it’s true: Plain old fish oil is the next exciting breakthrough that may benefit more people with heart disease. Recent studies are showing that a certain type of fish oil could help lower fats in the blood—namely high triglycerides, which when combined with high “bad” cholesterol or low “good” cholesterol, raise the risk of heart disease.

“Until recently, there were many drugs that lowered triglycerides, but many of them were not cardioprotective,” explains Dr. Zusman. Enter, highly purified eicosapentaenoic (EPA) acid, a fish oil that’s shown to lower triglycerides in patients with a history of diabetes and cardiovascular disease. A 2019 study in the New England Journal of Medicine found that taking 4 grams of pure EPA fish oil on top of a statin could significantly lower the risk of such cardiovascular events. Worth noting: The fish oil we’re talking about—pure EPA fish oil—isn’t sold over the counter. (The kind of oil you find in a store isn’t regulated by the FDA.)

“The over-the-counter fish oil products that our patients use extensively are not pure products but have other things that actually interfere with the beneficial effects of the purified products,” explains Dr. Zusman.

Currently, pure EPA fish oil is only available as a prescription medication called Vascepa (icosapent), which was FDA-approved in 2019 to help reduce the risk of cardiovascular events like heart attack.

New Targets for Cholesterol Drugs

Some research is focusing on finding new targets within the body that could aid in lowering cholesterol. For example, another burgeoning development has to do with lipoprotein(a), a sub-type of LDL cholesterol.

“Elevated levels of lipoprotein(a) are an underrecognized risk factor for people who have risk of heart disease,” explains Dr. Underberg.

So far, there aren’t any drugs approved to specifically target lipoprotein(a)—but that may soon change, as two new drugs are on the horizon that lower lipoprotein(a) using messenger RNA. It will take several years before we have data from the studies on these drugs—but it’s promising news.

Cholesterol Treatment Future Is Bright

The experts agree: It’s a hopeful era for cholesterol treatment research, and there are more options than ever for managing high cholesterol and reducing the risk of heart disease—from medications to lifestyle changes.

“It’s an exciting time to be taking care of people with cholesterol disorders because there’s a lot we can do with medications,” says Dr. Underberg. “But remember: The biggest impact we can make still is getting people to stop smoking, exercise, eat heart-healthy diets, and reduce stress.”

Lara DeSanto
Meet Our Writer
Lara DeSanto

Lara is a former digital editor for HealthCentral, covering Sexual Health, Digestive Health, Head and Neck Cancer, and Gynecologic Cancers. She continues to contribute to HealthCentral while she works towards her masters in marriage and family therapy and art therapy. In a past life, she worked as the patient education editor at the American College of OB-GYNs and as a news writer/editor at WTOP.com.