Single Infusion May Offer Lifelong Cholesterol Control
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Scientists have successfully tested a new gene therapy that dramatically lowers “bad” cholesterol with a single infusion, potentially offering a lifelong solution for those struggling to manage their levels. The experimental treatment works by permanently altering DNA within liver cells.
The therapy targets a specific gene responsible for regulating fats in the bloodstream. By permanently disabling this gene, the body is better able to break down and clear fats. This approach is inspired by individuals born with a natural defect in this same gene, who experience lifelong low cholesterol and a reduced risk of heart disease without apparent harm.
Initial Trial Shows promising Results
A first-in-human study involved 15 patients with persistently high cholesterol despite already being on strong medication. Participants received varying doses of the gene therapy via intravenous infusion.
Results showed a significant reduction in cholesterol levels at the highest dose: “bad” cholesterol (LDL) fell by nearly 49% and triglycerides by 55% just two months after treatment. The protein that hinders fat breakdown decreased by over 70%.Early indications suggest these effects are lasting, though longer-term monitoring is crucial to confirm the permanence of the change.
The technology utilizes CRISPR-Cas9, frequently enough described as “molecular scissors,” to precisely cut and modify DNA. The treatment delivers instructions encased in fat globules, which are absorbed by liver cells and then used to switch off the target gene.
Safety is a Priority, Long-Term Monitoring Planned
The primary focus of this initial study was safety. Side effects were minimal. Three participants experienced mild reactions like back pain and nausea during the infusion, which resolved with a temporary pause. One participant had a temporary increase in liver enzymes that returned to normal within two weeks. Importantly, a death occurred 179 days after treatment at the lowest dose, but was determined to be unrelated to the therapy.
Researchers emphasize the small size of the study and the limited follow-up period. Given the permanent nature of the DNA modification, participants will be monitored for up to 15 years to identify any potential long-term effects. Larger studies are needed to definitively establish the treatment’s safety and confirm its lasting efficacy.
Source: The New England Journal of Medicine
