With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for medication, according to a new pilot study published Saturday in the New England Journal of Medicine.The study was extremely small — only 15 patients with severe disease — and was meant to test the safety of a new medication delivered by CRISPR-Cas9, a biological sort of scissor which cuts a targeted gene to modify or turn it on or off.Preliminary results, however, showed nearly a 50% reduction in low-density lipoprotein, or LDL, the “bad” cholesterol which plays a major role in heart disease — the No.1 killer of adults in the United States and worldwide.AdvertisementAdvertisementAdvertisementAdvertisementThe study, which will be presented Saturday at the American Heart Association Scientific Sessions in New Orleans, also found an average 55% reduction in triglycerides, a different type of fat in the blood that is also linked to an increased risk of cardiovascular disease.“We hope this is a permanent solution, where younger people with severe disease can undergo a ‘one and done’ gene therapy and have reduced LDL and triglycerides for the rest of their lives,” said senior study author Dr. Steven Nissen, chief academic officer of the Sydell and Arnold Miller Family Heart, Vascular & Thoracic Institute at Cleveland Clinic in Ohio.“That’s a dream come true,” Nissen said. “If you’d asked me 15 years ago if we could have done something like this, I would have thought you were crazy,”Today, cardiologists want people with existing heart disease or those born with a predisposition for hard-to-control cholesterol to lower their LDL well below 100, which is the average in the US, said Dr. Pradeep Natarajan, director of preventive cardiology at Massachusetts General Hospital and associate professor of medicine at Harvard Medical School in Boston.AdvertisementAdvertisementAdvertisementAdvertisement“There is evidence to suggest that the optimal cholesterol level is 40 to 50, which is very hard to do through diet and lifestyle,” said Natarajan, who was not involved in the study.“Now, today’s medications can already lower LDL cholesterol to the levels found in the study — in fact, some medicines are a little more potent,” he said. “So we’re going to have to wait and see if this treatment works as well as this first study suggests.”While early, the findings are exciting due to the difficulty of remembering to take a daily medication — and sometimes three or four — to control high cholesterol levels, said preventive cardiologist Dr. Ann Marie Navar, an associate professor of cardiology at UT Southwestern Medical Center in Dallas, Texas.“In spite of having a lot of available therapies, the majority of people do not have their LDL under control,” said Navar, who was not involved in the study.AdvertisementAdvertisementAdvertisementAdvertisement“If you’re 20 and you have really high cholesterol, it may make a lot more sense to have a one-time treatment that doesn’t require you to have to take a pill every single day or shot every two weeks for the next 60 years,” she said. “The potential for this is just enormous.”A lucky mutation to reduce high cholesterolThe idea for a gene-editing treatment came from an unusual source — a genetic mutation. In that scenario, the ANGPTL3, or angiop …
