Cholesterol treatment: A single infusion of an experimental gene-editing drug safely cut LDL cholesterol and harmful triglycerides by about 50% in a first-in-human study of 15 volunteers, raising hopes for a one-and-done treatment for heart disease.
The results were presented at the American Heart Association’s annual meeting and published in The New England Journal of Medicine.
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The therapy uses CRISPR to disable ANGPTL3, a liver gene that helps regulate blood lipids. “It’s a knockout of the gene. It cuts it, and after that the gene no longer functions,” said Cleveland Clinic preventive cardiologist Dr Steven Nissen, a study investigator. By turning off ANGPTL3, researchers aimed to produce a durable drop in atherogenic lipids without daily pills.
“Rather than a lifetime worth of medicine, we have the potential to give people a cure,” said Dr Luke Laffin of the Cleveland Clinic, who helped conduct the trial. “It’s very exciting.” The study reported no serious safety signals in this small cohort, though participants will continue to be monitored.
Outside experts urged caution. Dr Eric Topol of Scripps Research called the concept “tremendously promising” but noted that gene editing remains expensive and its long-term safety in otherwise healthy people is unproven. University of Pennsylvania cardiologist Dr Kiran Musunuru said the approach “could be a very important tool,” but larger, longer trials are needed to demonstrate protection against heart attacks and strokes.
CRISPR Therapeutics, which developed and sponsored the study, said a scalable, single-dose therapy “could potentially impact millions” who struggle to maintain daily regimens of statins and other lipid-lowering drugs. Verve Therapeutics is testing a similar strategy using base editing, and early clinical data from both programs suggest the target is viable. Pricing has not been disclosed; other gene-based therapies have run into the millions of dollars per patient.
(With inputs from The Fox News)
