A groundbreaking discovery suggests that cyclodextrin, a sugar-like substance already approved by the FDA, may offer a new way to treat heart disease by dissolving the cholesterol buildup that clogs arteries. The finding emerged from an unlikely partnership between a mother concerned about her twin daughters' rare genetic condition and international cardiovascular researchers, and it could eventually help millions of people with atherosclerosis .

What Exactly Is Cyclodextrin and How Does It Work?

Cyclodextrin is a type of sugar molecule that researchers discovered can tackle heart disease through two distinct mechanisms. First, it dissolves cholesterol crystals that accumulate in artery walls, allowing the body to excrete them naturally. Second, it reprograms the immune system's response to these cholesterol deposits, reducing the inflammation that drives atherosclerosis .

To understand why this matters, consider how atherosclerosis develops. Your body needs some cholesterol to function, but excess cholesterol builds up as plaque inside artery walls. This plaque consists of cholesterol, calcium, and other substances that make arteries stiff and narrow, restricting blood flow. Eventually, a blood clot can completely block an artery, triggering a heart attack or stroke .

The key innovation is how cyclodextrin changes the behavior of macrophages, which are immune cells responsible for removing harmful substances from the body. Normally, when macrophages encounter cholesterol crystals, they trigger a strong inflammatory response that worsens plaque formation. Cyclodextrin reprograms these cells so they can absorb excess cholesterol while simultaneously reducing inflammation in the artery walls .

How Did Researchers Test This Discovery?

The research began when Chris Hempel, an American mother whose twin daughters have a rare genetic disorder preventing their cells from eliminating cholesterol, read about the role of cholesterol crystals in heart disease. She contacted Eicke Latz, a researcher at the University of Bonn and assistant professor at NTNU's Centre of Molecular Inflammation Research, with a simple but powerful idea: could the experimental drug treating her daughters also help people with conventional heart disease ?

Latz and his international team tested this hypothesis in multiple ways. First, they administered cyclodextrin to mice fed a cholesterol-rich diet that were prone to developing atherosclerosis. The results were striking: cyclodextrin not only prevented new plaque from forming but also reduced existing plaque already present in the animals' arteries .

To confirm the findings would translate to humans, researchers examined biopsies of plaque taken from human carotid arteries (the major blood vessels in the neck). When they treated these tissue samples with cyclodextrin, the cholesterol was removed from the plaques, and the cells within the plaque shifted into a reduced inflammatory state .

Why This Matters for People Who Can't Take Standard Cholesterol Drugs

Although several medications already exist to lower cholesterol, some patients experience side effects that make these drugs intolerable. Cyclodextrin offers a potential alternative pathway for cardiovascular disease treatment, particularly for individuals who struggle with conventional therapies .

The substance has another significant advantage: it's already been approved by the US Food and Drug Administration for use in humans, meaning the regulatory pathway for testing it as a heart disease treatment may be faster than developing an entirely new drug. However, because cyclodextrin has existed for some time, it cannot be patented, which makes pharmaceutical companies less interested in funding its development despite the promising research .

Steps to Understanding Your Cholesterol and Artery Health

• Know Your Numbers: Ask your doctor about your cholesterol levels, including LDL (low-density lipoprotein, the "bad" cholesterol) and HDL (high-density lipoprotein, the "good" cholesterol), as well as your blood pressure and any signs of atherosclerosis.
• Discuss Medication Tolerance: If you experience side effects from standard cholesterol medications like statins, inform your healthcare provider so they can explore alternative approaches or dosages that work better for your body.
• Monitor Cardiovascular Risk Factors: Track lifestyle factors including diet, exercise, smoking status, and stress levels, as these influence cholesterol crystal formation and arterial inflammation.
• Stay Informed About Emerging Treatments: Ask your doctor about new research and clinical trials, especially if conventional treatments haven't worked well for you or if you have a family history of heart disease.

What's Next for This Research?

The findings were so promising that the research team is now seeking funding and an industrial partner to conduct clinical trials in humans. Latz estimates that approximately 1 million euros would be needed to move forward with human testing .

"We saw that cyclodextrin prevented plaque formation. It even reduced the existing plaque the mice had in their arteries," explained Terje Espevik, head of CEMIR at NTNU.

Terje Espevik, Head of Centre of Molecular Inflammation Research at NTNU

The international research team, which included scientists from the University of Bonn, NTNU, the University of Oslo, and institutions in Australia, the USA, Denmark, and Sweden, published their findings in Science Translational Medicine in April 2016 . Remarkably, Chris Hempel, the mother whose insight sparked the investigation, is listed as a co-author on the published research.

While the path from promising laboratory results to approved medication typically takes years, the fact that cyclodextrin is already FDA-approved for other uses could accelerate the approval process if clinical trials confirm its effectiveness in humans. For the millions of people living with atherosclerosis or at high risk for heart disease, this discovery represents a potential new tool in the fight against cardiovascular disease.