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Cleveland study finds single-dose treatment could cut cholesterol in half

A diagram shows atherosclerosis in the veins.
Centers for Disease Control and Prevention
Artherosclerosis, a build-up of plaque in the arteries caused by cholesterol and fat, is one of the conditions that could be alleviated by the new treatment.

An experimental drug has the potential to cut cholesterol levels in half in a single dose, according to a new study by the Cleveland Clinic.

The November 8 study published in the New England Journal of Medicine found that 15 patients who received a one-time injection modifying DNA in their liver saw the cholesterol and fat levels in their bloodstream reduced by half. The patients experienced only minor side effects including nausea, back pain and temporarily elevated kidney enzymes, according to the study.

The study offers promise for future treatment in patients, preventing heart attacks and strokes, study author Steven Nissen, chief academic officer of the Heart, Vascular & Thoracic Institute at the Cleveland Clinic, said.

“I spent several decades working every year in the coronary care unit," he said. "And the number of tragic situations that I encountered are countless. The pain and suffering caused by these disorders is just not even calculable.”

The national scope of the problem means the new therapy could have tremendous impact, senior author Dr. Luke Laffin, a Cleveland Clinic cardiologist, said.

"Cardiovascular disease remains the number one killer in the United States," he said. "We have had a variety of new therapies and improvements over the past 40 years, but particularly recently, there's been a plateau in terms of what we can do."

Nearly 39% of Northeast Ohio residents reported having high cholesterol levels in 2023. Tuscarawas County has one of the state's highest incidences of high cholesterol, with more than 43% of residents affected.

The fact that the new therapy requires only one dose simplifies treatment for patients and helps them avoid health risks, Nissen said.

“We are really concerned about the adherence problem," Nissen said. "You give them a statin [typically a once-daily pill] and they don't feel bad, and so they get tired of it and they stop taking it. And the next thing you know, they're in our coronary care unit.”

A lack of adherence is a problem for many patients, Laffin said.

“About half of them discontinue taking [daily medications] after a year," he said.

The next phase of the study will test hundreds more patients beginning next year, Nissen said. He said it may take several years before the drug comes to market.

Stephen Langel is a health reporter with Ideastream Public Media's engaged journalism team.