Single Dose Cuts Cholesterol 62% for a Year

Imagine lowering dangerous cholesterol levels with a single medical treatment instead of taking pills every day for life. That future just moved closer to reality.

Researchers at University College London tested a new gene therapy called VERVE-102 on 35 adults with dangerously high cholesterol. The treatment, delivered through an IV drip, cut "bad" LDL cholesterol by up to 62% at the highest dose. Even better, those levels stayed low for up to a year.

The therapy works by permanently switching off a single gene in the liver that normally prevents the body from clearing cholesterol from the blood. It mimics what happens naturally in some lucky people born with this gene already turned off. These individuals enjoy low cholesterol their whole lives and rarely suffer heart attacks.

More than 7 million people in the UK and 40 million Americans currently take statins to manage cholesterol. The pills work well, but only if people keep taking them. Research shows about half of patients stop within a year, either because daily medication feels burdensome or because of side effects like muscle aches.

This is where VERVE-102 shines. A single treatment could potentially replace decades of daily pills.

Professor Riyaz Patel, who helped lead the trial, called it "an extremely exciting milestone." The results appeared in The New England Journal of Medicine with no serious safety concerns reported.

The Ripple Effect

High cholesterol contributes to hundreds of thousands of deaths from heart attacks and strokes each year in the UK and US alone. A treatment that keeps cholesterol low without daily medication could transform cardiovascular care for millions.

The therapy doesn't rewrite a person's entire DNA. It makes one targeted change to liver cells only, and it cannot be passed to children. Essentially, it gives people a genetic advantage some are simply born with.

Professor Bryan Williams from the British Heart Foundation praised the impressive results but emphasized that larger, longer studies are needed. The FDA requires 14 years of safety follow-up for gene editing treatments, so researchers will monitor participants carefully.

Some experts urge patience. Professor Kausik Ray from Imperial College London noted that while the technology is theoretically a once-in-a-lifetime treatment, scientists currently have only months of data. Safety issues can take years to emerge.

Still, for people with inherited high cholesterol or early heart disease who face a lifetime of medication, this research offers genuine hope that a better option may be on the horizon.