Harmful cholesterol levels cut in half with one-time gene editing drug in early trial

People who have tried medication to lower their dangerously high levels of cholesterol and other blood fats – without success – may benefit from an experimental treatment.

Altering a single gene may help people lower dangerously high levels of cholesterol and other fats in the blood, according to new research presented Saturday at the annual meeting of the American Heart Association in New Orleans.

The Phase 1 clinical trial of 15 people was intended to show whether the experimental gene-editing therapy was safe to use in humans.

It was, the researchers said. It was also effective: One infusion of the medicine drove down low-density lipoprotein (LDL) cholesterol and triglycerides by about half — an effect that could decrease patients’ heart disease risk for the rest of their lives.

“Frankly, if you’d asked me 15 years ago if we would be able to do this, I would have thought you were crazy,” said Dr. Steven Nissen, chief academic officer at the Cleveland Clinic’s Heart, Vascular & Thoracic Institute and one of the study’s investigators. “The results were pretty spectacular.”

The experimental drug employs CRISPR, a gene-editing tool that makes cuts and changes to the body’s genetic code. In this case, it manipulates a single gene in the liver that normally boosts cholesterol levels. Unlike cholesterol-lowering drugs like statins that need to be taken daily, this approach is meant to work permanently after one dose. (CRISPR Therapeutics makes the drug and helped fund the study.)