CRISPR gene editing works to lower high cholesterol in new study

A single infusion of an experimental gene-editing drug appears safe and effective for lowering cholesterol, possibly for life, according to a small early study released Saturday.

The study, which involved 15 volunteers, found that one infusion of a drug that uses the CRISPR gene-editing technique could safely reduce cholesterol and levels of harmful triglycerides by about half.

“Rather than just medicating for a lifetime, we have the potential to give people a cure,” says Dr. Luke Laffina preventive cardiologist at the Cleveland Clinic who helped conduct the study. “It’s very exciting.”

The results of the research were presented at the fair on Saturday Annual Meeting of the American Heart Association and published in The New England Journal of Medicine.

If confirmed by future research, this approach could provide a powerful new weapon to combat heart diseasethe country’s biggest killer, freeing people from the need to take statins and other cholesterol-lowering drugs every day.

However, Laffin and others cautioned that much more research is needed to confirm the findings and ensure the treatment would be safe and long-lasting.

“The idea of ​​a cheap, one-time solution [treatment]so you don’t have to use any of those drugs, right now that’s an idea – a fantasy – because genetic engineering is expensive and its long-term safety is unclear,” says Dr. Erik Topola cardiologist at Scripps Research in California who was not involved in the study.

Other scientists agree.

“It’s a step in the right direction,” says Dr. Kiran Musunuruscientific director of the Center for Inherited Cardiovascular Medicine at the Perelman School of Medicine of the University of Pennsylvania. He was also not involved in the investigation.

“It could be a very important tool,” he says. “But to actually prove that it is protective against cardiovascular disease, you need to do more research.”

And Musunuru and others note that the bar for safety would be higher if genetic engineering were applied to otherwise healthy patients, compared to those already suffering from serious illness.

Doctors inject the drug into patients’ bloodstreams so that it can travel to the liver and turn off a gene called ANGPTL3, which is involved in the production of cholesterol and triglycerides.

“It’s a knockout of the gene. It cuts it off. And then the gene stops functioning,” said Dr. Steven Nissenanother preventive cardiologist from the Cleveland Clinic involved in the study.

Samarth Kulkarnigeneral manager at CRISPR therapieswhich is developing the drug and sponsoring the study, says the approach “could potentially impact millions of people around the world.”

The findings are consistent with a similar approach being developed by another company, Verve Therapeutics in Boston.

“The fact that we now have more clinical data that there is a ‘there there’ is of course hugely encouraging,” says Fyodor Urnovwho studies gene editing at the University of California, Berkeley. “Having a CRISPR drug for heart attack would be an extraordinary victory.”

Neither company has said how much the treatment might cost, but other gene editing and gene therapies have been very expensive, costing millions 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 in the US every year. One important reason: many people stop taking their medications.

“This problem of adherence — that people stop 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 against heart attacks and strokes for a lifetime.