Patients with high cholesterol often take medications for years to control it, but a new gene-editing treatment has the potential to make a difference.
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A single injection of an experimental gene-editing drug appears to be safe and effective for lowering cholesterol, possibly for life, according to a small early study published Saturday.
The study, which included 15 volunteers, found that a single injection of a drug that uses CRISPR gene-editing technology can safely reduce cholesterol, as well as harmful triglyceride levels, by about half.
“Instead of getting a lifelong medication, we have the ability to give people a cure,” Dr. Hans said. Luke Lavignea preventive cardiologist at the Cleveland Clinic who helped conduct the study. “It’s very exciting.”
The results of the study were presented on Saturday American Heart Association Annual Meeting And published in New England Journal of Medicine.
If this approach is confirmed by future research, it could provide a powerful new weapon for combat My heart diseasethe country’s leading killer, frees people from the need to take statins and other cholesterol-lowering drugs every day.
However, Lavin and others cautioned that more research is needed to confirm the results and ensure that the treatment will be safe and long-lasting.
“Inexpensive idea, do it once [treatment]”So you don’t have to take any of these drugs. Right now that’s an idea — a fantasy — because gene editing is expensive, and long-term safety is unclear.” Eric Topola cardiologist at Scripps Research in California who was not involved in the study.
Other scholars agree.
“It’s a step in the right direction,” says the doctor. Kiran Musunuruscientific director of the Center for Cardiovascular Genetic Medicine at the University of Pennsylvania Perelman School of Medicine. He was not involved in the research either.
“It can be a very important tool,” he says. “But to prove its effectiveness in preventing cardiovascular disease, you need to do more study.”
Musunoro and others point out that safety standards will be higher when using gene editing on healthy patients than on those who are already seriously ill.
Doctors introduce the drug into patients’ bloodstream so that it can travel to the liver and disable a gene called ANGPTL3, which is involved in the production of cholesterol and triglycerides.
“It’s a knockout of the gene. It cuts it out. After that, the gene no longer works,” the doctor said. Steven Nissenanother preventive cardiologist at the Cleveland Clinic participating in the research.
Samarth KulkarniCEO of CRISPR therapeuticsThe drug development company that sponsored the study says this approach “could impact millions of people around the world.”
The results are consistent with a similar approach being developed by another company, Biotherapeutics In Boston.
“The fact that we now have more clinical data that suggests ‘there is out there’ is of course very encouraging,” he says. Fyodor Urnovwho studies gene editing at the University of California, Berkeley. “Having a CRISPR drug to treat heart attacks would be an extraordinary win.”
Neither company said how much the treatment would cost, but other gene-editing treatments and gene therapies have been expensive, costing millions per patient.
Millions of people take medications every day to lower their cholesterol and their risk of heart attack or stroke. But heart disease still kills nearly 700,000 people each year in the United States. One of the main reasons: Many people stop taking their medications.
“This adherence problem — that is, people stopping taking their medications — is huge,” Nissen says.
Researchers are planning larger, longer studies to see if a single-use gene-editing drug can safely protect people from heart attacks and strokes for life.