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New cholesterol drugs based on healthy genetic quirks

By | January 2, 2013, 10:54 PM PST

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

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Janet Fang

About Janet Fang

Janet Fang is a contributing editor for SmartPlanet.

Janet Fang

Janet Fang
Contributing Editor, Healthcare

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.

Janet Fang

Janet Fang

Janet does not have financial holdings that would influence how or what she covers.

She writes for SmartPlanet and is not an employee of CBS.

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Not amazingly short... moronically short.
"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 Regenerons Bill Sasiela. In the future, he expects human genetics to continue driving drug development."

In the future expect greedy drug and ag companies to kill us all with incredibly dangerous and irresponsible moves like this.

Anything that involves genetics should require 300+ years of tightly controlled and quarantined trials (at a minimum). It's the only way to be sure.
Posted by i8thecat4
3rd Jan
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