Several new approaches to tackling high cholesterol show great promise for people on the path to better heart health. Some researchers are exploring cutting edge technology, while others are perfecting common therapies long known to support cardiovascular health.

Here are five recent advances worth noting in high cholesterol treatment.

The American Heart Association has long recommended eating fish as part of a heart-healthy diet. Fish is a source of lean protein and omega-3 fatty acids. Past studies have suggested that three types of omega-3 fatty acids reduce the risk of heart disease and heart attack:

  • α-linoleic acid (ALA), found in nuts, seeds, and plant-based oils (like olive and canola oils)
  • eicosapentaenoic acid (EPA), mostly found in fatty fish like salmon, tuna, halibut, and cod
  • docosahexaenoic acid (DHA), also found in fish

New research shows that EPA may have a particularly significant benefit for people with high triglycerides who are at high risk for cardiovascular disease.

Triglycerides a type of fat found in the blood. High levels of triglycerides paired with high levels of low-density lipoprotein (LDL, the “bad” cholesterol”) or low levels of high-density lipoprotein (HDL, the “good” cholesterol) increases the risk for heart attack and stroke.

A 2019 study followed more than 8,000 people who were on statin therapy but still had high triglyceride levels. Participants were tracked for a median duration of 4.9 years.

Those who took a purified form of EPA (icosapent ethyl, brand name Vascepa) twice daily along with statins had a significantly lower risk of heart attack, stroke, and cardiac-related fatality.

New medications show promise in helping the liver function more efficiently to clear the blood of cholesterol to reduce the chances of a future cardiac event in people who are at high risk.

The liver removes excess cholesterol from the bloodstream. The protein proprotein convertase subtilisin/kexin type 9 (PCSK9) is made in the liver and destroys receptors in the liver that get rid of cholesterol. This prevents the liver from flushing the body of bad cholesterol as efficiently as it otherwise could.

People with high levels of PCSK9 tend to develop high cholesterol and heart disease earlier in life.

A class of drugs called PCSK9 inhibitors destroy these proteins to help the liver do its job. There are two types of PCSK9 inhibitors approved to treat high cholesterol and reduce the risk of heart disease:

  • alirocumab (Praluent)
  • evolocumab (Repatha)

These injectable medications are given every two to four weeks. The first injection is usually administered at a doctor’s office.

Recent large, double-blind, placebo-controlled studies of these drugs have found that they reduce the risk of stroke, heart attack, and cardiovascular death in people who have high cholesterol and are at high risk for a cardiac event.

The microbiome is the collection of an estimated 100 trillion bacteria, fungi, and viruses naturally found in the human body. While these microorgansims live mainly in the gastrointestinal tract, they impact a person’s overall health.

Probiotics are microorganisms in the foods we eat that are very similar to the healthy bacteria that grow in the gut.

Past research has found that taking a probiotic supplement may lower LDL cholesterol and improve a person’s LDL to HDL ratio. It may have other benefits for heart health, including lowering blood pressure, inflammatory mediators, blood glucose levels, and body mass index.

A 2018 analysis of 32 clinical trials looking specifically at the effect of probiotics on total cholesterol concluded that they’re effective compared to placebo. Results were more significant for people who had higher levels of baseline total cholesterol and were on long-term probiotic therapy.

More research needs to be done to understand how probiotics benefit the cardiac system, which probiotic strains are most effective, and whether a person’s genetics impact the effectiveness of these supplements.

Familial hypercholesterolaemia (FH) is high cholesterol caused by an inherited genetic mutation. There are two types of FH:

  • Heterozygous FH. This is when a person inherits one FH gene from one parent and one normal gene from the other parent. It affects about 1 in 200 to 244 people.
  • Homozygous FH. This is when a person inherits two FH genes, one from each parent. It affects about 1 in 160,000 to 300,000 people.

Both of these conditions are characterized by very high levels of LDL cholesterol and increased risk for premature cardiovascular disease. However the symptoms of homozygous FH tend to appear earlier, and the condition progresses more rapidly.

A 2014 overview concluded that untreated homozygous FH leads to a buildup of plaque deposits in the arteries known as atherosclerosis by the age of 20 and a life expectancy of age 30. The goal of doctors is early management of high cholesterol and detection of complications in people with this condition.

A new drug evinacumab (Evkeeza) offers new hope for people with homozygous FH. A small clinical trial published in 2020 found a monthly infusion of evinacumab reduced LDL cholesterol levels by 49 percent after 24 weeks.

In February 2021, the Food & Drug Administration (FDA) approved evinacumab for people aged 12 and over with homozygous FH.

This medication is given once per month as an intravenous infusion. Research hasn’t yet established its safety and efficacy for treating high cholesterol due to other causes.

Pregnancy testing is recommended for people who may become pregnant before starting this medication.

PCSK9 inhibitors are powerful tools in the fight against high cholesterol. It’s perhaps not surprising that researchers are looking for new ways to prevent PCSK9 proteins from impacting the cholesterol-lowering actions of the liver.

One such innovation is small-interfering (siRNA) therapy, inclisiran (Leqvio). This biologic drug regulates the expression of targeted genes to lower a person’s PCSK9 levels.

Three recent studies have shown a high rate of efficacy for this medication in people at high risk for cardiovascular disease or who have familial hypercholesterolemia, according to a paper published in 2020.

Participants who received inclisiran therapy once every 6 months saw a 50 percent decline in their LDL cholesterol.

Inclisiran was approved by the December 2020 for use in the European Union. The same month, the FDA declined to approve the drug in the United States after identifying problems with a European manufacturing site. The manufacturers are working to resolve the issue and receive FDA approval.

Nanotechnology is the study and use of very small things. Nanomedicine applies nanotechnology, or very small materials, to diagnose, monitor, control, prevent, and treat diseases.

These technologies may help address unmet medical needs. They can help target a drug to specific sites in the body so it’s effective at a smaller dose with fewer undesirable side effects.

For example, nanomedicine is commonly used in CT or MRI scans to make specific parts of the body stand out in imaging. It’s also commonly used for targeted cancer treatments.

Researchers are also looking into the applications of nanotechnology for diagnosing and treating high cholesterol. Some nanodrugs, for example, could inhibit the expression of genes responsible for cholesterol regulation.

A 2017 study reviewed these approaches. Some of these approaches, such as PCSK9 inhibitors, have already been proven safe and effective and are currently approved by the FDA to treat high cholesterol. Others are still in development.

One exciting development is the use of nanoparticles that mimic HDL or “good” cholesterol. Researchers are looking into using HDL-mimicking nanoparticles to better target drug therapies like statins and to improve diagnostic imaging.

A 2020 paper noted future developments might include delivery of not just statins but novel RNAs, proteins, and DNAs.

In 2020, researchers at Stanford Medical School used nanotechnology to significantly clear arterial plaque from mice with few side effects. In the study, the scientists created nanotubes that were taken up by white blood cells and carried a molecule into the arteries. The molecule turned off the signal in plaque cells that tells the immune system not to clear it out.

Researchers called it the “don’t eat me” signal, the same mechanism that can allow cancer cells to escape being destroyed by the immune system. Once this signal was turned off, the immune system was able to remove the unhealthy cells.

The study showed a 40 percent reduction in female and male mice with less advanced plaque and 20 percent reduction in male mice with more advanced plaque. The Stanford researchers’ approach allowed healthy cells to stay intact while the diseased cells were removed from the body.

These options offer a glimpse into the new possibilities that may exist for future cholesterol treatment.

Researchers are developing new therapies that work alongside established cholesterol treatments like statins. These show great promise for people with hard-to-manage cholesterol levels, such as those with familial hypercholesterolaemia.

Some therapies, such as fish oil and probiotics, are accessible to many people looking to support heart health.