Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-0449 Academic Article uri icon


MeSH Major

  • Antineoplastic Agents
  • Benzimidazoles
  • Cerebellar Neoplasms
  • Hedgehog Proteins
  • Medulloblastoma


  • 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.

publication date

  • September 17, 2009



  • Academic Article



  • eng

PubMed Central ID

  • PMC5317279

Digital Object Identifier (DOI)

  • 10.1056/NEJMoa0902903

PubMed ID

  • 19726761

Additional Document Info

start page

  • 1173

end page

  • 8


  • 361


  • 12