Jerome and Denny study reveals safety signal from genes that mimic drugs

(From Medical Xpress, March 2, 2018)

Study reveals safety signal from genes that mimic drugs

March 2, 2018 by Paul Govern, Vanderbilt University

Prospective mothers taking a new class of cholesterol-lowering drugs might incur higher risk of spina bifida in their future children, according to a study published in the journal Drug Safety by researchers at Vanderbilt University Medical Center.

The first two drugs in the new class, alirocumab and evolocumab, were approved by the Food and Drug Administration in 2015. They're taken by patients who don't respond well to first-line therapy.

The study findings, reported by Rebecca Jerome, MLIS, MPH, Joshua Denny, MD, MS, and colleagues, hinge on a new kind of epidemiological evidence. Nature sometimes beats pharmacology to the punch: name any drug with a targeted molecular mechanism, and there's a chance that there are people walking around with genetic variants that more or less mimic the action of the drug, people who indeed exhibit the benefits and side effects of the drug.

Analyzing the electronic health records of these patients, researchers can learn whether a drug-mimicking variant appears to provide protection against any other medical conditions. That is, they can check for drug-repurposing signals, which could subsequently be tested in clinical trials. With costs for developing new drugs stretching into billions of dollars, drug repurposing is a growing enterprise.

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