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Nature 441, 451-456 (25 May 2006) | doi:10.1038/nature04873; Published online 24 May 2006

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Review Article Validating cancer drug targets

John D. Benson1, Ying-Nan P. Chen1, Susan A. Cornell-Kennon1, Marion Dorsch1, Sunkyu Kim1, Magdalena Leszczyniecka1, William R. Sellers1 & Christoph Lengauer1

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A cancer drug target is only truly validated by demonstrating that a given therapeutic agent is clinically effective and acts through the target against which it was designed. Nevertheless, it is desirable to declare an early-stage drug target as 'validated' before investing in a full-scale drug discovery programme dedicated to it. Although the outcome of validation studies can guide cancer research programmes, strictly defined universal validation criteria have not been established.