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New medication may be effective in combating rare cholesterol disease

A new report in the journal The Lancet, reports that a new medication known as lomitapide showed promise in its phase 3 clinical trial in combating the the rare cholesterol disorder homozygous familial hypercholesterolemia (HoFH) by significantly lowering "bad" cholesterol, or LDL, which can be detected with cholesterol tests.

Patients who have HoFH have a high levels of LDL because of a genetic mutation in the LDL gene, which inhibits the liver from removing the the bad cholesterol from the blood. HoFH usually results in the development of cardiovascular disorders at a young age and death at about 30 years old.

The study, which was conducted by the Perelman School of Medicine at the University of Pennsylvania looked at the effect of lomitapide on 29 patients, 23 of whom also engaged in a year-long process that gauged the safety and tolerability of the drug. Each patient received lomitapide along with other lipid-lowering therapies and at the end of the efficacy phase, the average LDL levels were reduced by nearly 50 percent.

"The magnitude of this reduction in LDL-C and the fact that some patients reached or approached the LDL-C therapeutic goals is truly remarkable for this high risk population that historically doesn't respond to lipid-lowering drugs," said research author Marina Cuchel, M.D., Ph.D. "A reduction in LDL-C of this magnitude is certainly expected to favorably alter the clinical course of this devastating disease."

High cholesterol risks
The National Institutes of Health (NIH) reports that high blood cholesterol can lead to heart disease, which kills more men and women in the U.S. than any other ailment. Cholesterol is a wax-like substance that accumulates in the walls of the arteries and hardens them over time. This restricts the blood flow to the heart and can eventually lead to a heart attack. The source also notes that many people do not even know they have high cholesterol because there are usually not any symptoms.

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