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Experimental cholesterol-lowering drugs shown to be safe and effective in studies


New experimental drugs designed to reduce dangerous levels of cholesterol were found to be safe and effective in two groundbreaking research presented Sunday at an annual meeting of the American Heart Association.

Both drugs target people born with a genetic predisposition to high cholesterol. Although medications like statins, along with diet and exercise, can help these people manage their cholesterol levels, they cannot change the underlying genetic cause.

The two new approaches work in different ways, but with a single mission: attacking the genes responsible for raising cholesterol to change the trajectory of a person’s risk of heart attack and stroke.

Neither treatment had ever been tested in humans before. And both will require years of additional research before being considered for approval by the Food and Drug Administration. Still, experts are impressed by the results.

“There’s no way to call this anything other than revolutionary,” said Dr. Hugh Cassiere, director of intensive care services at South Shore University Hospital, Northwell Cardiovascular Institute in New York. Cassiere did not participate in either study.

A small change in a gene

One treatment, offered by Boston-based Verve Therapeutics, uses a gene-editing approach called base editing. This is an IV infusion of a drug targeting the PCSK9 gene, which plays a key role in the production of LDL, often called “bad” cholesterol.

When the drug focuses on PCSK9, it makes a slight change to the gene. The effect is like that of a permanent gum, removing its ability to raise cholesterol, said Dr. Sekar Kathiresan, co-founder and CEO of Verve.

In theory, the punctual treatment should last a lifetime. So far, patients have only been followed for six months.

Verve’s preliminary study, presented on Sunday, aimed to test the drug’s safety. Ten patients participated. Most received doses that did not cause a measurable difference in their LDL levels but were found to be safe.

However, three patients received higher doses and their LDL cholesterol levels were reduced by more than half. Additional studies will be needed to ensure the treatment remains safe, free of unexpected side effects, and effective.

Verve’s research was limited to people with a genetic condition called heterozygous familial hypercholesterolemia, in which cholesterol levels are extremely high from birth. Many affected people suffer a heart attack at a young age, between 30 and 40 years old.

Kathiresan, a cardiologist who previously worked at Massachusetts General Hospital and was a professor of medicine at Harvard Medical School, has long focused his research on understanding why some people have heart attacks at young ages and why others do not have. He has a strong family history of high cholesterol. In 2012, his brother died of a heart attack at the age of 40.

That’s when Kathiresan decided “to try to develop a therapy that could prevent tragedies like the one that happened in my family.”

It is unclear whether the approach will have a measurable impact on the risk of heart attacks and strokes – this remains to be seen in future studies.

Experts were still optimistic about the technology.

“While larger, longer-term studies are needed to assess both efficacy, durability and safety, this should mark the dawn of an era of gene-targeted therapeutics for cardiovascular disease,” he said. said Dr. Sahil Parikh, director of endovascular services at Columbia Irving University. Medical center in New York. Parikh was not involved in Verve’s research.

Shoot the messenger

The results of a second new therapy were also presented on Sunday.

The results, although early, offer a promising glimpse of what could be the first treatment for a particularly dangerous type of cholesterol called lipoprotein(a).

People with high Lp(a) levels are at extremely high risk of fat and cholesterol buildup in their arteries.

This is because Lp(a) glomerates onto LDL cholesterol, making these LDL particles even stickier and more likely to cause plaque.

It’s like adding super glue to duct tape. And it’s purely genetic, meaning people are born with this high risk. Diet and exercise have no impact on Lp(a) levels.

“It’s essentially incurable,” said study author Dr. Steven Nissen, academic director of the Heart, Vascular and Thoracic Institute at the Cleveland Clinic. “The only way to target such a genetic risk factor is to find a way to interfere with the gene product.”

Nissen and his colleagues used a new approach to correct the way this gene acts.

They used a drug called lepodisiran, which targets mRNA. If this sounds familiar, it should: Most Covid vaccines use mRNA to trigger the body to make an antibody against SARS-CoV-2.

In this case, the mRNA in question instructs the body to produce Lp(a). The drug prevents this from happening, essentially shooting the messenger.

Nissen’s study aimed to test the safety of lepodisiran. It was small, comprising only 48 adults in the United States and Singapore. All had very high levels of Lp(a). Overall, the drug was found to be safe and without major side effects, Nissen said.

But it also significantly reduced their Lp(a) levels. A single injection of lepodisiran reduced Lp(a) by more than 94% for almost a year, the study found.

The results of the study, sponsored by drugmaker Eli Lilly, were published Sunday in the Journal of the American Medical Association.

“This really offers a lot of hope for patients with high lipoprotein(a) levels,” Nissen said. “We are working as fast as possible because patients are dying every day from this disorder. We haven’t been able to treat it and we need to change that.”

Up to 64 million Americans have elevated Lp(a) levels, most commonly people of African and South Asian descent.

Further research is essential. An important question going forward is whether reducing Lp(a) actually reduces cardiac risks.

“We had to wait for this generation of treatments that could directly and specifically target Lp(a) and do it safely to see if that would also lead to fewer heart attacks and strokes,” said Dr. Donald Lloyd-Jones, professor and director of the department of preventive medicine at the Northwestern Feinberg School of Medicine in Chicago. Lloyd-Jones, also a former president of the American Heart Association, was not involved in the lepodisiran study.

Nissen predicts the treatment could one day be used as “an annual vaccine-like treatment for this previously incurable disorder.”

Although many heart problems can be prevented through lifestyle changes such as exercise and a healthy diet, Lloyd-Jones said, the medical community needs therapies to help people whose genes put them at risk. a greater risk of heart attack and stroke.

“We will always need drugs for people who are at very high risk,” he said.

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