Targeting DDX3 with a small molecule inhibitor for lung cancer therapy

نویسندگان

  • Guus M Bol
  • Farhad Vesuna
  • Min Xie
  • Jing Zeng
  • Khaled Aziz
  • Nishant Gandhi
  • Anne Levine
  • Ashley Irving
  • Dorian Korz
  • Saritha Tantravedi
  • Marise R Heerma van Voss
  • Kathleen Gabrielson
  • Evan A Bordt
  • Brian M Polster
  • Leslie Cope
  • Petra van der Groep
  • Atul Kondaskar
  • Michelle A Rudek
  • Ramachandra S Hosmane
  • Elsken van der Wall
  • Paul J van Diest
  • Phuoc T Tran
  • Venu Raman
چکیده

Lung cancer is the most common malignancy worldwide and is a focus for developing targeted therapies due to its refractory nature to current treatment. We identified a RNA helicase, DDX3, which is overexpressed in many cancer types including lung cancer and is associated with lower survival in lung cancer patients. We designed a first-in-class small molecule inhibitor, RK-33, which binds to DDX3 and abrogates its activity. Inhibition of DDX3 by RK-33 caused G1 cell cycle arrest, induced apoptosis, and promoted radiation sensitization in DDX3-overexpressing cells. Importantly, RK-33 in combination with radiation induced tumor regression in multiple mouse models of lung cancer. Mechanistically, loss of DDX3 function either by shRNA or by RK-33 impaired Wnt signaling through disruption of the DDX3-β-catenin axis and inhibited non-homologous end joining-the major DNA repair pathway in mammalian somatic cells. Overall, inhibition of DDX3 by RK-33 promotes tumor regression, thus providing a compelling argument to develop DDX3 inhibitors for lung cancer therapy.

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عنوان ژورنال:

دوره 7  شماره 

صفحات  -

تاریخ انتشار 2015