NEW DELHI: In a major medical breakthrough, scientists have shown that a one-time gene therapy can sharply lower “bad” cholesterol for at least a year, potentially opening the door to a future where some heart patients may no longer need lifelong cholesterol medication.The experimental therapy, called VERVE-102, reduced harmful LDL cholesterol by up to 62% in patients with inherited high cholesterol and early heart disease, according to a study published in New England Journal of Medicine.If confirmed in larger studies, researchers hope the findings may eventually lead to a “one-and-done” approach to preventing heart disease in large numbers of people. While most gene therapies currently target rare diseases, cardiovascular disease remains one of the world’s leading killers and accounts for nearly 28% of deaths in India.Dr Ambuj Roy, professor of cardiology, AIIMS said the study is an important proof of concept for cholesterol gene editing. “This study shows that in-vivo base editing of PCSK9 can produce substantial and sustained LDL cholesterol reduction after a single administration. If long-term safety is confirmed, gene editing for cholesterol could become a major cardiology breakthrough as lowering cholesterol remains the most important heart disease prevention strategies,” he said.Researchers said the treatment works by permanently switching off a liver gene called PCSK9, which controls cholesterol levels in blood. It uses advanced “base editing” technology, a newer and more precise form of gene editing.The early-stage trial involved 35 patients with familial hypercholesterolemia, an inherited condition that causes dangerously high cholesterol levels from a young age or premature coronary artery disease.Scientists found that the cholesterol reduction increased with higher doses. At highest dose tested, LDL cholesterol dropped by an average of 78 mg/dL, or nearly 62%.Researchers said the effect appeared long-lasting, with cholesterol levels remaining low for at least one year in several participants.High LDL cholesterol is one of the biggest causes of heart attacks, strokes and blocked arteries worldwide. Many patients with inherited cholesterol disorders continue to remain at risk despite taking multiple medicines.The study found no major safety concerns during the trial. In a statement, Eli Lilly and Company said it plans to begin Phase-2 clinical studies of VERVE-102 later this year.
