Scientists say a cutting-edge gene-editing treatment could help people cut harmful high cholesterol levels in half — just after one treatment.
A small trial of 15 people with high cholesterol, despite taking conventional, lifelong medications to lower it, showed significant reductions in two major risk factors for heart disease after receiving a single gene-editing treatment at the highest dose.
The experimental drug uses CRISPR, a gene editing tool that allows scientists to make changes to DNA. In this case, the drug targeted a gene in the liver called ANGPTL3 to lower LDL, or the “bad” cholesterol and triglycerides, which are both linked to heart disease.
“We’ve never had anything that could lower both LDL and triglycerides by around 50 percent,” said Cleveland Clinic cardiologist Dr. Steven Nissen, lead researcher of the first-in-human study of the therapy.
The results of the study were presented Saturday at the American Heart Association’s annual meeting and published in The New England Journal of Medicine.
Cholesterol and triglyceride levels in participants began to drop within two weeks after receiving the treatment, and the results were sustained for at least 60 days after, according to the research.
While this trial was small, future successful trials could be life-changing for many, according to the scientists behind the study.
“Rather than a once-daily pill or monthly injection, this therapy would potentially offer a one-time infusion that is safe and durable for patients with high cholesterol,” Cleveland Clinic’s Dr. Luke Laffin, the study’s co-leader, said.
High LDL, the “bad” cholesterol, can cause plaque to build up in the artery walls, raising the risk of heart attack or stroke. Meanwhile, high triglycerides, another blood fat, can also increase those risks.
The 15 trial participants were from Australia, New Zealand and the U.K. and were all in their 50s and 60s. Thirteen of the participants were men, and all had uncontrolled high cholesterol, triglycerides or both.
“We’re going to try to demonstrate the safety and efficacy of these one-and-done therapies because we think these options are important for patients,” Nissen said.
Three participants had temporary reactions to the therapy, including nausea and elevated liver enzymes; however, their reactions quickly resolved, the scientists said.
With reporting by Reuters.
