Cholesterol-lowering medicines are among the most widely used drugs for preventing heart attacks and strokes.
They are especially common among older adults and people who have diabetes, high cholesterol or other cardiovascular risks. New research suggests that some of these medicines may also affect other parts of the body in ways that scientists are still trying to understand.
Cholesterol itself is not automatically harmful. The body needs it to build cells and make certain hormones, but too much of a type commonly called LDL cholesterol can contribute to fatty deposits inside arteries. Over time, these deposits can narrow blood vessels or break apart, increasing the risk of heart attack and stroke.
Statins are the best-known medicines used to lower LDL cholesterol. Drugs such as atorvastatin, sold under the brand name Lipitor, and rosuvastatin, known as Crestor, reduce the liver’s production of cholesterol. They have been studied for decades and can substantially reduce cardiovascular risk in people who are likely to benefit from them.
Another group of medicines is known as PCSK9 inhibitors. These newer treatments help the liver remove more LDL cholesterol from the blood. They can lower cholesterol dramatically and may be used when statins are not enough or when certain patients cannot tolerate standard treatment.
Researchers led by Kitty Pham, a Ph.D. student at the University of South Australia, investigated whether cholesterol-lowering treatments could have effects beyond their intended cardiovascular benefits.
Instead of following people taking the medicines for many years, the team used genetic information to examine biological pathways that mimic some of the drugs’ actions.
This type of research can help scientists investigate possible long-term effects that may be difficult or expensive to study in traditional clinical trials.
Genetic differences are present from birth and can provide clues about how changing a particular biological target might influence several parts of the body. However, genetic evidence does not perfectly reproduce what happens when someone takes a medicine.
The researchers found evidence suggesting that statin-related pathways could be associated with greater body weight and body fat. They also found possible links with lower testosterone levels. Testosterone is present in both men and women and plays roles in sexual health, muscle, bone and other body functions.
The study also produced a potentially positive finding involving the brain. Genetic evidence related to statin action was associated with a larger hippocampus, an area deep inside the brain that is important for memory, learning and emotions. Changes in this brain region have been linked to conditions including dementia and depression.
That finding does not prove that taking a statin will enlarge a person’s hippocampus or prevent dementia. Brain health is influenced by many factors, and genetic studies mainly identify possible biological relationships that need to be tested in other types of research.
Clinical studies would be needed before doctors could consider statins a treatment for protecting the hippocampus.
The researchers also examined pathways targeted by PCSK9 inhibitors. Their analysis raised questions about possible effects involving the lungs. The finding is an early safety signal rather than proof that these medicines damage lung health, and more research is needed to determine whether the association appears in people receiving the drugs.
These results need to be balanced against what is already known about cholesterol treatment. Statins have strong evidence showing that they reduce heart attacks and strokes in appropriate patients. PCSK9 inhibitors have also been shown to lower LDL cholesterol and cardiovascular risk in people who need more intensive treatment.
No patient should stop a cholesterol medicine because of these findings without discussing it with a health professional. Suddenly abandoning treatment may remove protection against serious cardiovascular disease. A doctor can consider possible side effects alongside a person’s cholesterol level, medical history and overall risk.
For people taking statins, changes such as unexplained weight gain can be discussed during routine medical care. Patients using newer cholesterol treatments should also report unusual or persistent symptoms to their health care team.
Reporting symptoms does not mean the medicine is necessarily responsible, because many other conditions can produce similar changes.
The research was published in the British Journal of Clinical Pharmacology. It adds to a growing effort to understand medicines not only by asking whether they achieve their main purpose, but also by examining wider effects throughout the body. This can reveal possible benefits as well as risks that deserve closer investigation.
One strength of the study is its use of large-scale genetic information to explore effects that might take years to become apparent.
At the same time, this is also an important limitation because inherited genetic differences are not identical to taking a drug at a particular dose for a particular length of time. The results should therefore be treated as clues rather than confirmed side effects.
Future studies can test these signals using medical records, long-term patient studies and clinical trials. Researchers will need to determine whether the suggested changes in weight, hormones, brain structure or lung health actually occur because of the medicines and whether they are large enough to matter clinically.
Such work could eventually help doctors choose cholesterol treatments more precisely for individual patients.
For now, the established heart-protective benefits of cholesterol-lowering medicines remain important.
The new findings do not overturn those benefits, but they show why researchers continue to study even familiar medications after they have been used for years. Understanding both intended and unexpected effects can help make effective treatments even safer.
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