Single-Shot Gene Therapy Shows Promise in Permanently Lowering Cholesterol, Offering Potential Alternative to Statins
London, December 1, 2023 – A groundbreaking new gene therapy, VERVE-102, is demonstrating significant potential to permanently reduce cholesterol levels with a single injection, offering a potential alternative to lifelong medication like statins.Early trial results, hailed as “fabulous” by experts, suggest the treatment could eliminate or substantially reduce the need for ongoing cholesterol management for many patients.
Currently, statins – costing approximately £20 a year per patient – are typically recommended only for individuals with a family history of high cholesterol.VERVE-102 takes a different approach,targeting the PCSK9 protein by “switching off” the gene responsible for its production in liver cells.
The injected treatment, administered slowly over several hours, doesn’t entirely halt LDL (bad) cholesterol production, as the PCSK9 protein is just one factor in the liver’s regulation of LDL. However, recent trials have shown promising results: the lowest dose of the drug reduced LDL cholesterol by 21 percent, while the highest dose achieved a 53 percent reduction – both within one month of treatment.
Initial findings indicate a favorable safety profile, with few reported serious adverse effects.
Dr. Petraco explained the underlying science, stating, “Our genes are like a recipe book, but rather of cakes, they continuously produce proteins with specific functions.” He further clarified that “VERVE-102 swaps one single compound within the PCSK9 gene, causing a malfunction in its recipe to stop it producing LDL.”
Despite the encouraging results, researchers emphasize the need for further investigation. “There is an element of the unknown with gene therapy and long-term follow-up studies are essential,” Dr. Petraco cautioned. “But it’s not an overstatement to say VERVE-102 is a gamechanger if it is shown to be safe and effective in larger trials. But we need to be cautious before widely adopting any new drug.”
A larger, worldwide trial is planned, focusing initially on individuals with familial hypercholesterolemia – a genetic condition causing extremely high cholesterol levels, often present from childhood.Researchers anticipate the treatment may eventually benefit a broader population, including those resistant to current drugs or experiencing intolerable side effects.
While the potential benefits are significant, cost remains a concern. professor David middleton of Lancaster University noted, “Statins are cheap to produce and are effective for most people.” The cost of VERVE-102 has not yet been disclosed, but gene-editing drugs are generally expensive, though a single dose is theoretically all that would be required.
Professor Bowman added, “This new drug is trying to genetically switch off the mechanism that makes our cholesterol rise for good - and that sounds amazing because people do not like taking tablets or having regular injections. If it turns out to be effective and safe, it really could be a fabulous thing for the future.”
(Image: VERVE-102 could lower cholesterol levels with just a single shot, as opposed to keeping patients on medication like statins)
