Nature Medicine | Spoonful of Medicine

First drugs found to inhibit elusive cancer target

By Melinda Wenner Moyer

Ever since scientists discovered the cancer-promoting gene MYC in the late 1970s, researchers have dreamt of developing drugs that inhibit its function. Yet efforts to target MYCactivity have proven unsuccessful, in part because the protein product encoded by the oncogene lacks an obvious target-binding site. Now, however, scientists from a handful of research groups have found a way to inhibit MYCindirectly—by preventing an upstream protein from instigating the expression of MYCand its downstream targets. Buoyed by the promising therapeutic effects that such experimental drugs have had in mice with several types of cancer, companies are racing to test the molecules in clinical trials, despite lingering questions about how, exactly, they work.

“We, like everyone else, are very excited,” says Brian Huntly, a hematologist at the University of Cambridge in the UK. “We’re very keen to get this into humans as soon as possible.”

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