An experimental gene-editing drug has shown promising results in a small trial, significantly reducing LDL cholesterol levels long-term with just one infusion. This preliminary study suggests a potentially curative effect against heart disease, according to experts.
The study, conducted on 35 participants, revealed a substantial decrease in LDL cholesterol levels — up to 62% — after the highest dose of treatment. This change has persisted for over 18 months in a subgroup of the initial trial participants. If confirmed in larger trials, this one-time treatment approach might offer widespread prevention of heart disease, a leading cause of death in the U.S., responsible for nearly 800,000 deaths annually.
The findings were published in The New England Journal of Medicine, despite being an interim analysis, due to the promising outcomes and the innovative application of gene therapy aimed at combatting heart disease. Dr. Eric Rubin, editor in chief of the journal, noted the ambitious scope of the trial, targeting a prevalent health risk rather than rare diseases typically addressed by gene therapies.
The study will expand to include 85 participants, followed by a larger trial involving 200 patients, to further investigate the long-term effects and efficacy of this treatment.
Dr. John H. P. Alexander, a cardiologist from Duke University, emphasized the potential impact of a curative approach, which could alter current treatment debates and guidelines for heart disease.