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Imatinib and the long tail of targeted drug development

New molecular insights occasionally lead to the rapid development of therapeutic agents that improve the outcomes of patients with cancer; however, these breakthroughs can be followed by extensive, empirically driven and often unsuccessful efforts at extending the drug to other indications or combinations. Herein, we describe the clinical development of imatinib, a paradigm of rapid molecularly driven drug development, and advocate for a balanced portrayal of the potential of molecularly targeted therapies for cancer.

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Fig. 1: Accumulating evidence and research organization (AERO)8 diagram of published clinical trials of imatinib.


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The work of J.K. is funded by the Canadian Institutes of Health Research (CIHR; EOG111391). Without in any way implying endorsement of the contents of this article, the authors thank members of STREAM and H. Atkins for helpful consultations.

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Correspondence to Jonathan Kimmelman.

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The authors declare no competing interests.

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Carlisle, B.G., Zheng, T. & Kimmelman, J. Imatinib and the long tail of targeted drug development. Nat Rev Clin Oncol 17, 1–3 (2020).

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