In a recent preliminary study, an experimental gene-editing drug demonstrated significant potential in dramatically reducing cholesterol levels with just one infusion. Scientists reported these findings on Monday, suggesting the treatment may have long-term effects.
If further confirmed, this research could pave the way for a preventative treatment against heart disease, a condition responsible for nearly 800,000 deaths annually in the United States. Unlike existing gene therapies that focus on rare diseases, this approach targets a widespread health issue.
Dr. John H. P. Alexander, a cardiologist at Duke University, emphasized the potential for a curative therapy to significantly impact how early treatments are administered to patients. He stated, “A curative therapy would change the game.”
The study, published in The New England Journal of Medicine, provided an interim analysis of 35 patients involved in a trial with plans to expand to 85 participants. All subjects had genetically high levels of LDL cholesterol, known as the bad cholesterol, or existing heart disease.
Results showed that a single high-dose infusion could lower LDL cholesterol levels by up to 62 percent. Moreover, this reduction persisted in a subgroup treated 18 months earlier. The study will eventually progress to include 200 patients.
Dr. Eric Rubin, editor in chief of The New England Journal of Medicine, highlighted the unusual decision to publish preliminary findings. He noted the ambition behind applying advanced gene therapy to combat a leading cause of death in the U.S.
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