From Genome to Drugs: Where Do We Stand?

Abstract

Adecade has passed since scientists produced a rough draft of the human genome. Since then, modern genomics has produced just one new drug: a treatment for lupus called belimumab (Benlysta), which the U.S. Food and Drug Administration (FDA) approved in March. Whether that ’ s consistent with early, bullish predictions about what genomics might add to the drug arsenal is open to debate. Press coverage from the human genome ’ s early days was certainly optimistic. USA Today , for instance, quoted Francis Collins, M.D., Ph.D., now the director of the National Institutes of Health, who said that decoding the human genome was an “exhilarating moment in history . . . and a major step towards treating a host of diseases for which genomics offers the best hope of prevention and cure.” Fast forward to June of last year, when the New York Times essentially declared genomics a failure for what it was accomplishing for patients. Genomics had been a boon for basic science, wrote staff reporter Nicholas Wade, but it had “produced little for medicine,” largely because, he claimed, most of the disease genes identifi ed by sequencing were too rare to make practical drug targets.