Researchers Find a Way to Mimic Clinical Trials Using Genetics

A technique called Mendelian randomization could be the revolutionary tool drug companies have been waiting for

MIT Technology Review
MIT Technology Review

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Illustration: Mengxin Li

By Gary Taubes

In January 1977, five landmark health surveys, led by the famous Framingham Heart Study, reported a “striking” revelation about HDL cholesterol — a.k.a. the “good cholesterol.” The higher the HDL in a person’s bloodstream, researchers had found, the lower the risk of heart attack. This held true for every age group and both sexes. In fact, HDL was the only reliable predictor of heart disease risk in people over 50, which is the age group people who have heart attacks are likely to fall into.

In observation after observation ever since, the relationship between HDL cholesterol and heart health has been so outrageously robust it’s hard to imagine that HDL doesn’t play a fundamental role in preventing the disease process. This has led drug companies to spend billions of dollars developing and testing HDL-raising drugs, with the expectation that heart attacks will be prevented, lives saved, and investments recouped many times over.

And yet those drugs have universally and spectacularly failed. “Dashing hopes,” as a 2016 New York Times headline proclaimed, “a…

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MIT Technology Review
MIT Technology Review

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