A One-Time Experimental Treatment Might Control Cholesterol for Life

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Heart disease is the leading cause of death globally. While treatments like cholesterol medication and healthy lifestyle advice can help manage risks, it’s often difficult for people to make and maintain these changes.

 

Scientists are working towards a future where people don’t need to depend on current treatments for heart disease. A recent study published May 25th in the New England Journal of Medicine by Verve Therapeutics (owned by Eli Lilly) shows promising results from a single-dose gene therapy designed to lower cholesterol – a major cause of heart disease. In the study of 35 participants, this therapy reduced “bad” cholesterol (LDL) by 9% to 62%, depending on the amount given.

 

This new therapy focuses on a gene called PCSK9. Researchers discovered about 20 years ago that people with a specific change in this gene naturally had very low levels of “bad” cholesterol (LDL), and therefore a lower risk of heart problems. The PCSK9 gene controls cholesterol levels in the liver, and in these individuals, the gene wasn’t working correctly, leading to lower cholesterol without causing other health issues. This led scientists to create drugs that block the PCSK9 gene, and four such drugs are now available to lower cholesterol. However, these drugs all require injections, and some need to be given by a doctor. Because of this inconvenience, many people – around 30 to 50 percent – stop taking the medication after about a year, which is a similar rate to what’s seen with daily cholesterol pills called statins.

 

VERVE-102 is a gene therapy designed to solve the problem of patients not taking their cholesterol medication consistently. It’s a single treatment that edits genes in the liver to make them continually produce a modified version of PCSK9, which lowers cholesterol. While the genetic change isn’t exactly the same as the one found in people naturally born with low cholesterol, the effect is the same: it deactivates the gene and reduces LDL cholesterol levels.

 

Dr. Sek Kathiresan, head of obesity research at Verve Therapeutics, explains that this research offers early proof we might be able to provide cholesterol protection to people who don’t naturally have it. While more research is needed, these initial findings suggest a potential future where a single treatment could permanently lower high cholesterol.

 

If future, larger studies confirm its safety and effectiveness, this gene therapy could offer a one-time treatment for high cholesterol, eliminating the need for ongoing injections or pills. The therapy works by editing cells in the liver, which naturally renew themselves every few months. Once edited, these cells reproduce, passing on the beneficial genetic change and potentially providing a lifelong reduction in harmful cholesterol. Early results from patient monitoring, lasting about 18 months so far, show consistently low LDL cholesterol levels. Animal studies suggest these low levels are maintained for at least three years, giving researchers confidence that the effects of this treatment will last.

 

The main side effect observed is a temporary increase in a liver enzyme, explains Kathiresan. This is expected, as the gene therapy focuses on liver cells and may initially affect the normal balance of liver compounds.

 

This initial study involved individuals who were either genetically likely to have high cholesterol or had experienced early heart problems. However, Lilly intends to launch a bigger study with around 200 participants, focusing more on those with existing heart disease to get a better understanding of how the treatment works for them. Additionally, Lilly will track the 35 people from this first study for 15 years to ensure the gene therapy remains safe long-term.

 

Participants in this study continued to take their usual cholesterol medications, like statins, for safety throughout the trial. However, researchers plan to investigate if a single gene therapy treatment, such as VERVE-102, could eventually replace those medications. Kathiresan explains it’s too soon to know for sure, as results will depend on each person’s initial cholesterol levels and target goals. Ultimately, the goal is for this therapy to become the most effective treatment option.

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2026-05-28 17:06