Patients with high cholesterol often take medication for years to manage it, but a new gene-editing technique could change that.
TEK IMAGE/Science Photo Library/Getty Images
hide signature
switch signature
TEK IMAGE/Science Photo Library/Getty Images
A single dose of an experimental gene-editing drug appears safe and effective in lowering cholesterol levels, possibly for life, according to a small early study published Saturday.
The study, which involved 15 volunteers, found that a single infusion of a drug using the CRISPR gene-editing technique could safely cut cholesterol levels, as well as levels of harmful triglycerides, by about half.
“Instead of being on medication for life, we have the potential to give people a cure,” the doctor said. Luke Laffinpreventative cardiologist at the Cleveland Clinic who helped conduct the study. “It's very interesting.”
The study results were presented on Saturday at American Heart Association Annual Meeting and published in New England Journal of Medicine.
If future research confirms this approach, it could become a powerful new weapon to combat heart diseasethe country's leading killer, freeing people from having to take statins and other cholesterol-lowering drugs every day.
However, Laffin and others cautioned that much more research is needed to confirm the results and ensure that the treatment is safe and long-lasting.
“The idea of an inexpensive, universal [treatment]so you don't necessarily need to take any of these drugs, right now it's an idea—a fantasy—because gene editing is expensive and the long-term safety is unclear,” the doctor said. Eric Topola cardiologist at Scripps Research in California who was not involved in the study.
Other scientists agree with this.
“This is a step in the right direction,” says the doctor. Kiran MusunuruScientific Director of the Center for Hereditary Cardiovascular Medicine at the Perelman School of Medicine at the University of Pennsylvania. He also did not take part in the study.
“This can be a very important tool,” he says. “But to actually prove that it protects against cardiovascular disease, you need to do more research.”
Musunuru and others note that the safety bar will be higher when using gene editing in patients who are otherwise healthy, compared with those who already suffer from serious diseases.
Doctors inject the drug into patients' bloodstreams so it can travel to the liver and turn off the ANGPTL3 gene, which is involved in the production of cholesterol and triglycerides.
“This is a gene knockout. He cuts it. And after that the gene no longer functions,” the doctor said. Stephen Nissenanother preventive cardiologist at the Cleveland Clinic who participated in the study.
Samarth KulkarniCEO of the company CRISPR therapywhich is developing the drug and sponsoring the study, says the approach “has the potential to impact millions of people around the world.”
The findings are consistent with similar approach is being developed by another company Verve Therapeutics in Boston.
“The fact that we now have more clinical evidence for the existence of 'there-there' is of course extremely encouraging,” says Fedor Urnovwho studies gene editing at the University of California, Berkeley. “Getting a CRISPR cure for heart attack would be an extraordinary victory.”
Neither company said how much the treatment might cost, but other gene editing and gene therapy methods have been very expensive, costing millions of dollars per patient.
Millions of people take medications every day to lower their cholesterol and reduce their risk of heart attack or stroke. But heart disease still kills nearly 700,000 people every year in the United States. One important reason: many people stop taking their medications.
“The problem of adherence—people stopping taking their medications—is huge,” Nissen says.
Researchers are planning larger, longer studies to see if a one-time gene-editing drug can safely protect people from heart attacks and strokes for life.
