New cholesterol drugs based on healthy genetic quirks

Summary:To develop new cholesterol drugs, several companies are mimicking a genetic aberration found in people who have naturally lower cholesterol levels.

To make a cholesterol drug, take a good look at people with low levels of cholesterol.

Some people are protected by a genetic aberration that naturally lowers their cholesterol levels, reducing their incidence of heart attack. Now, several companies are pursuing drugs that mimic this genetic quirk. Technology Review reports.

Low-density lipoprotein (LDL) cholesterol, or “bad cholesterol,” is directly correlated to heart disease. Drugs known as statins, such as Crestor and Lipitor, lower LDL cholesterol by inhibiting an enzyme that plays a role in its production, and these are some of the most widely prescribed drugs on the market in the U.S.

But clinical trials suggest that genetically inspired drugs could lower LDL-cholesterol levels even further, and could especially benefit patients who can’t tolerate statins.

In November, Amgen and Pfizer reported promising signs from mid-stage clinical trials of two drugs that target a gene called PCSK9.

The protein product of PCSK9 regulates LDL cholesterol levels in the blood by decreasing the amount of a receptor that removes LDL cholesterol from the blood. Faulty copies of PCSK9 confer lower-than-average amounts of LDL cholesterol.

A third company, Alnylam, is running early-stage trials of a PCSK9 gene-silencing treatment.

A fourth company, Regeneron, has begun late-stage testing of its PCSK9 inhibitor. Their antibody-based therapeutic binds to the PCSK9 protein and blocks it from interacting with the LDL receptor.

The PCSK9 gene was first identified in 2003. In this case, the time frame of moving from a basic discovery to late-stage clinical trials -- less than 10 years -- is “amazingly short,” says Regeneron’s Bill Sasiela. In the future, he expects human genetics to continue driving drug development.

[Via Technology Review]

Image: PCSK9 via Wikimedia

This post was originally published on

Topics: Innovation


Janet Fang has written for Nature, Discover and the Point Reyes Light. She is currently a lab technician at Lamont-Doherty Earth Observatory. She holds degrees from the University of California, Berkeley and Columbia University. She is based in New York. Follow her on Twitter.

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