Single-Shot Gene Therapy Shows Promise in Permanently Lowering Cholesterol, Offering Potential Alternative to Statins
London, December 1, 2023 – A โขgroundbreaking new gene therapy, VERVE-102, is demonstrating significant potential โคto permanently reduce cholesterol โคlevels 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 substantiallyโฃ reduce the need for ongoingโ cholesterol management for many patients.
Currently, โstatins – costing โapproximately ยฃ20 a year per patientโฃ – are typically recommended only forโค individuals with a family history of high cholesterol.VERVE-102 takes a different approach,targeting the PCSK9 โขprotein by “switching off” the gene responsible for its production in liver cells.
Theโ injected โtreatment, administered slowly over several โคhours, doesn’t โentirely halt LDL (bad) cholesterol production, as โคthe PCSK9 protein is just โone factor inโ the liver’s regulation of LDL. โฃHowever, recent trials have shown promising results: the lowest dose of the drug reduced LDL cholesterol by โ21โ percent,โ while the highest โdose achieved a 53 percent reduction – both within one month ofโ treatment.
Initial findings โindicate a favorable safetyโ profile, with few reported serious adverse effects.
Dr. Petraco explainedโ the underlyingโ 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 โคcompound within the PCSK9 gene, causing a malfunction in its recipeโ to stop it producing LDL.”
Despite the encouraging results, researchers emphasize the need for further investigation.โ “There is an element of the โฃunknown with gene therapy and long-term follow-up studies โคare essential,” Dr. Petraco cautioned. “But it’sโฃ not an overstatement to say โขVERVE-102 is a gamechanger if itโ is shown to be โขsafeโข and effective inโ larger trials.โ But we need toโข be cautious before widely adopting any new drug.”
A โฃlarger, worldwide โฃtrial is planned,โฃ focusing initially on individualsโ with familial hypercholesterolemia – a genetic condition causing extremely high โcholesterol levels, often present from childhood.Researchers anticipate โคtheโ treatment may eventually benefit aโข broader population, including those resistant to current drugs orโค experiencing intolerableโ side effects.
While the potential benefitsโฃ are significant, cost remains a concern. professor David middleton of Lancaster โคUniversity noted, “Statins are cheap to produce and are โขeffective for most people.” The cost of VERVE-102 has โnot yet been โdisclosed, โฃbut gene-editingโ drugs โare generally expensive,โข though a single dose is theoretically โall that would be required.
Professor Bowman added, “Thisโ new drug is trying to genetically โswitch off the โmechanism that makes ourโ cholesterol rise for good โฃ- and that โsounds amazing โคbecause people do not likeโข taking tablets or having regular โinjections. If it turns out to be effective and safe, โขit really โcould 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)