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In 2003, the US Food and Drug Administration (FDA) approved the first targeted therapy for non-small cell lung cancer (NSCLC). AstraZeneca’s Iressa (gefitinib) had shrunk some patients’ tumors in small clinical trials, and despite some concern about the robustness of the results, regulators OK’d the therapy as an option for patients when chemotherapy and other generalized cancer-attacking treatments had failed. But after only a few months on the market, it was clear that something was amiss: for up to 90 percent of patients, the drug simply didn’t work.
While AstraZeneca struggled to explain this disappointing outcome, within a year three academic groups independently came to the same conclusion: gefitinib, it appeared, was only effective in patients harboring certain mutations in EGFR, a gene coding for ...