Scientists are looking at two new drugs that target high cholesterol at the genetic level. Lupe Rodríguez/Stocksy

Researchers are reporting that two new treatments can effectively target cholesterol at the genetic level.

Both treatments are aimed at people with a genetic predisposition to high cholesterol for whom diet and exercise aren’t effective.

Experts note that both treatments need more research and likely won’t be ready for approval for years.

Two promising new clinical trials aimed at the genetics of cholesterol were unveiled this week at the American Heart Association’s annual conferenceTrusted Source.

Both drugs, designed to drive down unhealthy levels of cholesterol were shown to be effective and safe, their creators said.

The medications target people born with a genetic predisposition to high cholesterol, increasing the user’s odds of heart attacks and stroke.

Current tools to manage high cholesterol include exercise, diet, and statin medications. These two new drugs would be the first to go after the genetic cause of high cholesterol.

However, neither will hit the market anytime soon, as both will require years of more research before being approved for consumers.

Nonetheless, experts say the results are encouraging.

“The new studies… are really a groundbreaking change in the world of cardiovascular medicine,” said Dr. Spencer Kroll, a physician with the Kroll Medical Group and the Cholesterol Treatment Center in New Jersey and a director of the Northeast Lipid Association.

“Even though they’re in early-stage clinical trials, these studies represent that cholesterol management and cardiovascular disease treatment is a multimodal and individualized process,” Kroll, who was not involved in the research, told Medical News Today. “We are now entering the realm of cholesterol therapy that moves away from the one-size-fits-all statin treatment to therapies that are personalized to the individual patient.”

New drug targets cholesterol at the genetic level

One of the treatmentsTrusted Source comes from Boston-based Verve Therapeutic and uses a gene-editing method called base editing.

It’s given through an IV and targets the PCSK9 gene, which is associated with higher levels of LDL, also known as “bad cholesterol.”

Researchers explain that the drug makes a tiny change to PCSK9, limiting its ability to raise cholesterol. Theoretically, a one-timetreatment should last a lifetime, although test subjects have so far only been followed for six months.

The initial study only had 10 participants and was meant to look at the drug’s safety. Most of the subjects didn’t receive doses big enough to make a measurable difference in cholesterol levels but got through the trial without major side effects or health issues.

The Verve Therapeutic researchers report they did give higher doses to three subjects, whose LDL levels were reduced by more than half.

All the study participants had a genetic condition called heterozygous familial hypercholesterolemiaTrusted Source, characterized by high cholesterol levels from birth. Many with the condition have heart attacks by the time they’re in their 30s or 40s.

“Although this first-in-human trial was very small, it did show a large drop in LDL levels in a few patients,” said Dr. Cheng-Han Chen, an interventional cardiologist and medical director of the Structural Heart Program at MemorialCare Saddleback Medical Center in California who was not involved in the research.

“Most importantly, the study was successful in showing that the “base editing” technique can work in the liver in humans,” Chen told Medical News Today. “These are very initial clinical results that show proof-of-concept in humans, but that require much more research before they can be used on a wider scale.”

The research on this drug has not been published yet in a peer-reviewed journal