Inhibition of WNT signaling attenuates self-renewal of SHH-subgroup medulloblastoma

Oncogene. 2017 Nov 9;36(45):6306-6314. doi: 10.1038/onc.2017.232. Epub 2017 Jul 17.

Abstract

The SMOOTHENED inhibitor vismodegib is FDA approved for advanced basal cell carcinoma (BCC), and shows promise in clinical trials for SONIC HEDGEHOG (SHH)-subgroup medulloblastoma (MB) patients. Clinical experience with BCC patients shows that continuous exposure to vismodegib is necessary to prevent tumor recurrence, suggesting the existence of a vismodegib-resistant reservoir of tumor-propagating cells. We isolated such tumor-propagating cells from a mouse model of SHH-subgroup MB and grew them as sphere cultures. These cultures were enriched for the MB progenitor marker SOX2 and formed tumors in vivo. Moreover, while their ability to self-renew was resistant to SHH inhibitors, as has been previously suggested, this self-renewal was instead WNT-dependent. We show here that loss of Trp53 activates canonical WNT signaling in these SOX2-enriched cultures. Importantly, a small molecule WNT inhibitor was able to reduce the propagation and growth of SHH-subgroup MB in vivo, in an on-target manner, leading to increased survival. Our results imply that the tumor-propagating cells driving the growth of bulk SHH-dependent MB are themselves WNT dependent. Further, our data suggest combination therapy with WNT and SHH inhibitors as a therapeutic strategy in patients with SHH-subgroup MB, in order to decrease the tumor recurrence commonly observed in patients treated with vismodegib.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anilides / pharmacology
  • Animals
  • Cell Line, Tumor
  • Cerebellar Neoplasms / drug therapy
  • Cerebellar Neoplasms / genetics
  • Cerebellar Neoplasms / metabolism*
  • Cerebellar Neoplasms / pathology
  • Disease Models, Animal
  • HEK293 Cells
  • Hedgehog Proteins / metabolism*
  • Humans
  • Male
  • Medulloblastoma / drug therapy
  • Medulloblastoma / genetics
  • Medulloblastoma / metabolism*
  • Medulloblastoma / pathology
  • Mice
  • Mice, Transgenic
  • Pyridines / pharmacology
  • Random Allocation
  • SOXB1 Transcription Factors / genetics
  • SOXB1 Transcription Factors / metabolism
  • Small Molecule Libraries / pharmacology
  • TRPC Cation Channels / deficiency
  • TRPC Cation Channels / genetics
  • TRPC Cation Channels / metabolism
  • Transfection
  • Tumor Suppressor Protein p53 / deficiency
  • Tumor Suppressor Protein p53 / genetics
  • Tumor Suppressor Protein p53 / metabolism
  • Veratrum Alkaloids / pharmacology
  • Wnt Proteins / antagonists & inhibitors*
  • Wnt Proteins / metabolism
  • Wnt Signaling Pathway*

Substances

  • Anilides
  • Hedgehog Proteins
  • HhAntag691
  • Pyridines
  • SOX2 protein, human
  • SOXB1 Transcription Factors
  • Shh protein, mouse
  • Small Molecule Libraries
  • Sox2 protein, mouse
  • TP53 protein, human
  • TRPC Cation Channels
  • Trpc5 protein, mouse
  • Tumor Suppressor Protein p53
  • Veratrum Alkaloids
  • Wnt Proteins
  • cyclopamine