From 190 to 80. That’s how quickly Alice Thomas’ cholesterol level dropped in the span of a few months thanks to an experimental gene-editing treatment she received as part of a preliminary study published in The New England Journal of Medicine.

The study, as reported by The New Times, was an interim analysis of 35 patients in a trial, all of whom have genetically high levels of the bad kind of cholesterol— LDL—which is a major contributor to heart disease.

For 64-year-old Thomas, of Lexington, N.C., Social Security is her sole income. Having already had two strokes, she would love to take advanced cholesterol-lowering drugs but can’t get them. And while daily statin pills, the usual go-tos, are cheap, she was unable to tolerate them.

“A curative therapy would change the game,”said Dr. John H. P. Alexander, a cardiologist at Duke University who was not involved with the study. “We have these debates and new guidelines that we should be treating people earlier.”

A potential solution for the leading cause of death in U.S
The study was led by Dr. Sekar Kathiresan, the chief executive at Verve Therapeutics, now a subsidiary of Eli Lilly. Motivated by personal history, Dr. Kathiresan, a cardiologist, has shared that a couple of family members have had heart attacks. And after returning from a run, his brother died of cardiac arrest at age 42.

“We’re going for a medicine that someday could be part of primary care.” Dr. Daniel Skovronsky, chief scientist at Eli Lilly said.

There are some concerns though. Per safety regulations, the Food and Drug Administration requires that all patients in gene therapy studies be followed for 15 years. According to Dr. J. Michael Gaziano, the director of preventive cardiology at the Boston V.A. health care system, there needs to be more safety data around this gene-editing treatment. Dr. Gaziano was not involved in the new study. The New England Journal of Medicine does not usually publish such preliminary results but editor-in-chief Dr. Eric Rubin said “it looks like it works pretty well.”

How the Gene Editing Process Works
First, a gene editing “machine,” or a tiny molecular factory wrapped in a cloak of fat is infused into the trial patients. The fat-coated particles flow through the blood directly to the liver, where they are taken up by cells that remove the fatty wrapper.

Then the editing machine moves along the liver cell’s DNA until it finds its target, a gene called PCSK9. Stopping there, it erases one DNA letter in the gene, replacing it with another.

With this simple change the PCSK9 gene is disabled, preventing cells from making it. Without the PCSK9 gene, the liver pulls more LDL cholesterol out of the bloodstream, keeping the levels lower.

A single infusion of the highest dose of the treatment reduced LDL cholesterol levels by as much as 62 percent in the 35 patients. The change has been sustained in a subgroup whose members were treated 18 months ago, which will be followed by a larger study of 200 patients.

One-and-Done way to prevent heart disease
Cardiovascular disease kills nearly 800,000 Americans a year, according to the CDC.

Although high LDL levels are eminently treatable with an array of medicines, including more recent advances like injected drugs that block the protein made by the PCSK9 gene, too many people cannot or will not take the drugs.

The New York Times reported that between one third and a half of patients stop taking cholesterol-lowering medications within a year of starting them, even people who have had heart attacks.

45-year-old Kristy Faulkner lives in Guilford, Conn., and is among those who need treatment but are reluctant to take a powerful drug. Heart disease runs in her family, and she had a heart attack when she was 42. Even though she understands the importance of the medication, she feels ashamed. “There’s some sort of internal denial, like I can’t be on these meds every day of my life,” she said.

Yale University cardiologist Dr. Erica Spatz is Ms. Faulkner’s cardiologist. She says she hopes Faulkner’s insurance will pay for a PCSK9 inhibitor that only has to be administered every six months. There is “no margin for error,” Dr. Spatz said.

But if a gene-editing treatment were administered just once?

“This kind of therapeutic breakthrough could be a game changer for people like her,” Dr. Spatz said.