Drug Lowers Cholesterol in a New Way

A new drug works in an entirely different way than statins — the popular class of drugs doctors commonly prescribe to lower cholesterol and reduce the risk of heart disease. Now, researchers report there is a new option for people who don’t respond to statins or who have bad reactions.

Researchers from the University of Pennsylvania School of Medicine in Philadelphia demonstrated the effectiveness of the new therapy in a recent study. They were able to reduce low-density lipoprotein — “bad” cholesterol — by 51 percent in patients with a genetic disorder that causes extremely high cholesterol levels.

“For the first time, it’s an approach that actually successfully lowers cholesterol in these patients who have very severely elevated cholesterol who really don’t respond much at all to other medications,” said Daniel Rader, M.D., Director of Preventive Cardiology at the University of Pennsylvania.

The new therapy, called BMS-201038 or AEGR-733, blocks a protein that packs low-density lipoprotein (LDL) molecules with cholesterol before they leave the liver. Without the protein, the LDL molecules can’t leave the liver and the patient’s cholesterol levels go down.

Dr. Rader compares the process to what would happen if someone fired all the dockworkers before they could load the ships with cargo — or cholesterol. “The cargo doesn’t get loaded, the LDL boat never leaves the liver, and basically, these people don’t put as much LDL into their blood,” he told Ivanhoe.

This is especially important for patients with homozygous familial hypercholesterolemia, explained Dr. Rader. These patients have such high LDL levels that they sometimes need bypass surgery in their 20s, or even in their teens.

More testing is needed before the medication will be available to patients. Some side effects include loose stool and fatty liver.

SOURCE: Ivanhoe interview with said Daniel Rader, M.D., Director of Preventive Cardiology at the University of Pennsylvania; The New England Journal of Medicine, 2007;356:148-156

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