An experimental gene-editing drug has shown potential in dramatically lowering cholesterol levels in a small preliminary study, with effects possibly being permanent after a single infusion. Scientists reported these findings on Monday, which could lead to significant advancements in heart disease prevention.
In a study involving 35 patients, the infusion of the drug reduced LDL cholesterol levels by up to 62 percent in those with genetically high levels or heart disease. The reduction has been sustained for 18 months in a subgroup. If confirmed in larger studies, there may be hopes for a one-time treatment for cardiovascular disease, which claims 800,000 American lives annually.
“A curative therapy would change the game,” stated Dr. John H. P. Alexander from Duke University.
Published in The New England Journal of Medicine, this interim analysis is part of a trial that aims to include up to 85 participants and will soon expand to 200 patients. The publication of such a preliminary result is rare for the journal, but the editor in chief, Dr. Eric Rubin, highlighted its significance in applying gene therapy to combat the leading cause of death in the United States.
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