NYT: “One-and-Done Heart Disease Prevention?”

A single infusion of an experimental gene-editing drug seemed to reduce LDL long-term in a small trial. The results may point to something “curative,” one expert said.

If confirmed in larger studies, researchers hope the findings may lead to a one-and-done way to prevent heart disease in large numbers of people. Most gene therapies target rare diseases, but cardiovascular disease kills nearly 800,000 Americans a year.

“We have these debates and new guidelines that we should be treating people earlier,” said Dr. John H. P. Alexander, a cardiologist at Duke University who was not involved with the study. “A curative therapy would change the game.”

The study, published in The New England Journal of Medicine, was an interim analysis of 35 patients in a trial that will involve as many as 85 participants. All have genetically high levels of LDL cholesterol — the bad kind — or heart disease.

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Gene therapies for rare diseases carry multimillion-dollar price tags. But Dr. Daniel Skovronsky, chief scientist at Eli Lilly, said that would not be the case if this treatment were eventually approved.

“That’s not what we’re going for here,” he said. “We’re going for a medicine that someday could be part of primary care.”

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High LDL levels are eminently treatable with an array of medicines, including the old standbys, daily statin pills. More recent advances include injected drugs that block the protein made by the PCSK9 gene, creating the same effect as gene editing.

But too many people cannot or will not take the drugs. Between one third and a half of patients stop taking cholesterol-lowering medications within a year of starting them, even people who have had heart attacks.