Research ArticleCancer

AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer

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Science Translational Medicine  18 Nov 2015:
Vol. 7, Issue 314, pp. 314ra185
DOI: 10.1126/scitranslmed.aac5272

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Blocking transcription in tumors, STAT

STAT3 is a transcription factor that plays an oncogenic role in many cancers, which has proven very difficult to target with chemical inhibitors. Now, Hong et al. have demonstrated that antisense technology is a feasible alternative to small-molecule inhibitors for targeting STAT3. The authors used high-affinity next-generation antisense oligonucleotides, which have higher potency than previous generations and can be systemically administered without a lipid vehicle. One of these new antisense oligonucleotides, AZD9150, demonstrated activity in a variety of preclinical cancer models, as well as in cancer patients who have failed one or more previous treatments, paving the way for additional clinical testing of this therapy.

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