Molecular pathways: The hedgehog signaling pathway in cancer

Ross McMillan, William Matsui

Research output: Contribution to journalArticlepeer-review

108 Scopus citations


The Hedgehog (Hh) signaling pathway regulates embryonic development and may be aberrantly activated in a wide variety of human cancers. Efforts to target pathogenic Hh signaling have steadily progressed from the laboratory to the clinic, and the recent approval of the Hh pathway inhibitor vismodegib for patients with advanced basal cell carcinoma represents an important milestone. On the other hand, Hh pathway antagonists have failed to show significant clinical activity in other solid tumors. The reasons for these negative results are not precisely understood, but it is possible that the impact of Hh pathway inhibition has not been adequately measured by the clinical endpoints used thus far or that aberrancies in Hh signal transduction limits the activity of currently available pathway antagonists. Further basic and correlative studies to better understand Hh signaling in human tumors and validate putative antitumor mechanisms in the clinical setting may ultimately improve the success of Hh pathway inhibition to other tumor types.

Original languageEnglish (US)
Pages (from-to)4883-4888
Number of pages6
JournalClinical Cancer Research
Issue number18
StatePublished - Sep 15 2012

ASJC Scopus subject areas

  • Cancer Research
  • Oncology


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