Medical Breakthrough: First Ever Drug For Untreatable Cholesterol

Medical Breakthrough: First Ever Drug For Untreatable Cholesterol

According to a recent study conducted in Australia, a new drug called muvalaplin could potentially be the first drug to treat “bad cholesterol” that was previously untreatable. The study, which was published in the Journal of the American Medical Association on August 28th, indicated that muvalaplin was able to reduce lipoprotein(a) or Lp(a), which is a genetic carrier for “bad cholesterol,” by as much as 65% after two weeks of daily treatment.

It’s estimated that around 1 in 5 people globally have high levels of Lp(a), and a serum level above 30 mg/dl can put individuals at risk of heart disease and heart attacks, while levels above 50 mg/dl increase the risk of stroke.

“When it comes to treating high Lp(a), a known risk factor for cardiovascular disease, our clinicians currently have no effective tools in their kit,” Dr. Stephen Nicholls, a cardiologist, the study’s lead researcher, and a professor at Monash University, said in a press release.

According to Dr. Nicholls, Muvalaplin could be a game-changer.

Photo by Nicolas Solerieu on Unsplash

This drug is the first of its kind, designed to specifically target the elusive form of cholesterol known as Lp(a). Dr. Nicholls is excited that the drug can be delivered in the form of an oral tablet, making it more accessible to patients.

However, the study authors cautioned that since the clinical trial only investigated phase 1, which tests for drug safety, it remains uncertain whether lowering Lp(a) with Muvalaplin will reduce cardiovascular risk. Only 89 healthy individuals took Muvalaplin in phase 1 of the clinical trial, and phase 2 is currently ongoing and scheduled to be completed in 2024.

What Is Lipoprotein(a)?

Lipoprotein(a) is a type of LDL particle that is known for transporting “bad cholesterol.” Some researchers question the accuracy of labeling HDL as “good cholesterol” and LDL as “bad cholesterol,” since both types of cholesterol are the same and are transported by different carriers that serve distinct functions. These researchers contend that the harm lies in the carrier and not in the cholesterol that it carries.

While lifestyle and dietary interventions can influence normal LDL levels, the levels of Lp(a) in the body are primarily influenced by genes. Consequently, few treatments can lower Lp(a).

Like all LDL particles, the role of Lp(a) is to transport cholesterol and fat from the liver to the body’s tissues. Some of this cholesterol may adhere to the walls of blood vessels, causing the formation of plaques. Lp(a) is produced in the liver, where it is attached to an additional protein chain called apolipoprotein(a). This extra protein chain makes Lp(a) stickier and may increase its tendency to stick together or accumulate in blood vessel walls.

Muvalaplin and Lipoprotein(a)

Muvalaplin works in the body by blocking the first step that occurs in the liver and causes the proteins to bind together to form a chain.

“This approach mimics naturally occurring variants,” the authors wrote. People with these variants cannot get proteins to interact with each other, resulting in naturally low Lp(a) levels.

It’s important to take care of our health, and sometimes that means trying new treatments. In a study with 89 healthy individuals, researchers looked at the effects of muvalaplin. Some participants were given a fixed dose while others received an increasing dose, and some were given a placebo. Within a day of taking muvalaplin, researchers saw a decrease in Lp(a), especially in those who received higher doses. Overall, the treatment led to a maximum reduction of 60% in Lp(a) levels after accounting for the placebo effect. In fact, almost all participants who received the highest dose had Lp(a) levels below 50 mg/dl after treatment.

This is important information because research shows that reducing Lp(a) levels by 50 mg/dl over five years could lower the risk of a recurring cardiovascular event by 20% over the next five years. It’s always good to be informed about our health and possible treatments, and it’s great to see positive results like these.


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