In a small, preliminary study, an experimental gene-editing treatment dramatically reduced cholesterol levels — possibly permanently — after just one infusion, scientists reported Monday.
If the results are confirmed in larger studies, researchers hope the discovery could lead to a “one-and-done” way to prevent heart disease on a large scale. Most gene therapies target rare diseases, but cardiovascular disease kills nearly 800,000 Americans a year.
“We have these debates and new guidelines saying we should treat people earlier,” said Duke University cardiologist John HP Alexander, who was not involved in the study. “A curative therapy would be a game changer.”
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The study, published in New England Journal of Medicineis an interim analysis of 35 patients in a trial expected to include up to 85 participants. Everyone has genetically high levels of LDL cholesterol — the “bad” one — or already has heart disease.
Among the 35 patients, a single infusion of the highest dose of the treatment reduced LDL levels by up to 62%. The decline continued in a subgroup treated 18 months ago.
The next step will be a larger study, with 200 patients.
It is unusual for New England Journal of Medicine publish such preliminary results. But “it seems to work really well,” said Eric Rubin, the magazine’s editor-in-chief. He further noted that the trial is an ambitious attempt to apply the latest in gene therapy to the leading cause of death in the United States.
Still, “we need a lot more safety data,” said J. Michael Gaziano, director of preventive cardiology at the Department of Veterans Affairs health system in Boston, who was not involved in the study. The Food and Drug Administration (FDA) requires that all patients in gene therapy studies be followed for 15 years.
High LDL levels are largely treatable with a variety of medications, including traditional daily statin pills. More recent advances include injections that block the protein produced by the PCSK9 gene, generating an effect similar to gene editing.
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But many people can’t or don’t want to take their medicine. Between a third and half of patients stop taking cholesterol medications within a year of starting treatment — even those who have already suffered a heart attack.
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