Cholesterol management may be achievable by altering just one switch in an individual’s genetic code—potentially for a lifetime.
A pilot study featured in the New England Journal of Medicine demonstrated a novel gene therapy that decreased patients’ low-density lipoprotein (LDL) cholesterol, commonly known as “bad” cholesterol, by nearly 50%, while also reducing triglycerides by an average of 55%.
If forthcoming trials yield similar results, this one-time therapy could serve as an alternative to the combination of medications that millions currently rely on to manage their cholesterol.
LDL cholesterol and triglycerides are lipids produced by the liver; however, excessive accumulation in the bloodstream can lead to fat deposits that may result in cardiovascular diseases, which account for about one-third of deaths in the United States.
“Both LDL cholesterol and triglycerides are linked to severe cardiovascular risks, such as heart attacks, strokes, and mortality,” remarked Steven Nissen, a professor of medicine at the Cleveland Clinic Lerner School of Medicine. BBC Science Focus.
Nissen was part of a research team focusing on lowering cholesterol levels by targeting the ANGPTL3 gene, associated with LDL cholesterol and triglycerides.
About 1 in 250 individuals possess a mutation that deactivates this gene, leading to lower lipid levels in their blood. Nissen noted, “Importantly, the occurrence of cardiovascular diseases in these individuals is also minimal.”
Thanks to CRISPR gene-editing technology, identifying individuals who might benefit from this mutation is no longer just a matter of chance.
Utilizing CRISPR, Nissen and his team developed a treatment to deactivate the ANGPTL3 gene in the liver, which was then infused into 15 patients during an initial safety study.
The treatment significantly reduced participants’ LDL and triglyceride levels within two weeks, and these reductions remained stable after 60 days. Nissen stated, “These changes are anticipated to be permanent.”
Healthcare professionals recommend maintaining LDL cholesterol levels below 100mg/dL to promote heart health. While lifestyle changes can assist, many individuals, particularly those with genetic tendencies to high cholesterol, find it challenging to reach this target.
While existing medications are effective, no drugs simultaneously lower both LDL cholesterol and triglycerides, often requiring patients to take multiple medications daily for life to manage their cholesterol.
“The next phase of the trial is set to commence in the coming months, involving more patients with elevated LDL cholesterol or triglycerides,” Nissen stated.
If the trials continue to succeed, this therapy could serve as a lasting solution against some of the most significant health threats globally.
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Source: www.sciencefocus.com
