Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-0449

Charles M. Rudin, Christine L. Hann, John Laterra, Robert L. Yauch, Christopher A. Callahan, Ling Fu, Thomas Holcomb, Jeremy Stinson, Stephen E. Gould, Barbara Coleman, Patricia M. LoRusso, Daniel D. Von Hoff, Frederic J. De Sauvage, Jennifer A. Low

Research output: Contribution to journalArticlepeer-review

803 Scopus citations


Medulloblastoma is the most common malignant brain tumor in children. Aberrant activation of the hedgehog signaling pathway is strongly implicated in the development of some cases of medulloblastoma. A 26-year-old man with metastatic medulloblastoma that was refractory to multiple therapies was treated with a novel hedgehog pathway inhibitor, GDC-0449; treatment resulted in rapid (although transient) regression of the tumor and reduction of symptoms. Molecular analyses of tumor specimens obtained before treatment suggested that there was activation of the hedgehog pathway, with loss of heterozygosity and somatic mutation of the gene encoding patched homologue 1 (PTCH1), a key negative regulator of hedgehog signaling.

Original languageEnglish (US)
Pages (from-to)1173-1178
Number of pages6
JournalNew England Journal of Medicine
Issue number12
StatePublished - Sep 17 2009

ASJC Scopus subject areas

  • General Medicine


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