A One-Time Experimental Treatment Might Control Cholesterol for Life

The only side effect of note is that the drug temporarily causes higher levels of a liver enzyme, says Kathiresan, reflecting the fact that the gene therapy targets liver cells and initially may disrupt levels of liver compounds.

The current trial included people either with a genetic predisposition to high cholesterol or who had early heart events, but Lilly plans to conduct a larger trial of about 200 people—including more people in the latter category who might be more representative of those who have heart disease. The company will also continue to follow the 35 people in this first trial for 15 years to monitor the safety of the gene therapy they received.

For safety reasons, people in the current study also continued taking cholesterol-lowering medications, including statins, during the trial. But future research will address whether at some point, a one-time gene therapy like VERVE-102 could replace statins altogether. “It’s too early to say right now, and a lot will depend on where people start in terms of their cholesterol and where their goal is,” says Kathiresan. “But ultimately, we would love to be in a position where this is the preferred therapy of choice.”