A preliminary study suggests that a single administration of an experimental gene-editing drug may reduce cholesterol levels. The study, involving 15 volunteers, indicated that a drug utilizing the CRISPR gene-editing technique could safely decrease both cholesterol and triglyceride levels by approximately 50%.
Dr. Luke Laffin, a preventative cardiologist at the Cleveland Clinic and co-investigator, highlighted the potential for this approach to offer a long-term solution to high cholesterol.
The findings were presented at the American Heart Association's annual meeting and published in The New England Journal of Medicine on Saturday. If substantiated by further research, this method could offer an alternative to daily cholesterol-lowering medications in the management of heart disease.
However, Dr. Laffin and other experts emphasized the necessity for extensive additional research to validate the findings and confirm the treatment's safety and durability over time. Dr. Eric Topol, a cardiologist at Scripps Research, noted that while the concept of a single, inexpensive treatment is appealing, gene-editing therapies are currently expensive, and their long-term safety profiles remain to be fully established.
Dr. Kiran Musunuru, scientific director at the Center for Inherited Cardiovascular Medicine at the University of Pennsylvania Perelman School of Medicine, also stated that more studies are required to demonstrate the treatment's protective effects against cardiovascular disease. He added that the safety threshold for gene-editing in otherwise healthy individuals would be higher than for those with pre-existing serious conditions.
The drug is administered intravenously, targeting the liver to disable the ANGPTL3 gene, which is involved in the production of cholesterol and triglycerides. Dr. Steven Nissen, another preventive cardiologist at the Cleveland Clinic involved in the research, explained that the process involves a 'knockout' of the gene, rendering it non-functional.
Samarth Kulkarni, CEO of CRISPR Therapeutics, the drug's developer and study sponsor, indicated the potential global impact of this treatment. These findings are consistent with similar research being conducted by Verve Therapeutics in Boston. Fyodor Urnov, a gene-editing researcher at the University of California, Berkeley, acknowledged the encouraging nature of accumulating clinical data for CRISPR-based heart disease treatments.
The potential cost of this treatment has not been disclosed, but existing gene-editing and gene therapies have been associated with high costs. Millions of individuals globally take daily medications to manage cholesterol and reduce the risk of heart attack or stroke. Non-adherence to medication regimens is a significant factor in the continued prevalence of heart disease, which causes nearly 700,000 deaths annually in the U.S.
Researchers are planning larger, extended studies to assess the long-term safety and efficacy of a single gene-editing drug in protecting against heart attacks and strokes.