Expression of BARHL1 in medulloblastoma is associated with prolonged survival in mice and humans

Oncogene. 2011 Nov 24;30(47):4721-30. doi: 10.1038/onc.2011.173. Epub 2011 May 23.

Abstract

Medulloblastoma is the most common malignant brain tumor in childhood, and development of targeted therapies is highly desired. Although the molecular mechanisms of malignant transformation are not fully understood, it is known that medulloblastomas may arise from cerebellar granule neuron precursors. The homeodomain transcription factor Barhl1 is known to regulate migration and survival of granule cell precursors, but its functional role in medulloblastoma is unknown. We show here that the expression of BARHL1 is significantly upregulated during human cerebellar development and in human medulloblastoma samples as compared with the normal adult cerebellum. We also detected high levels of Barhl1 expression in medulloblastomas of Math1-cre:SmoM2 mice, a mouse model for Sonic hedgehog-associated medulloblastomas that we developed previously. To investigate Barhl1 function in vivo during tumor development, we generated Barhl1(-/-)Math1-cre:SmoM2 mice. Interestingly, tumors that developed in these mice displayed increased mitotic activity and decreased neuronal differentiation. Moreover, survival of these mice was significantly decreased. Similarly, low expression of BARHL1 in human medulloblastoma cases was associated with a less favorable prognosis for patients. These results suggest that the expression of Barhl1 decelerates tumor growth both in human and in murine medulloblastomas and should be further investigated with respect to potential implications for individualized therapeutic strategies.

MeSH terms

  • Adolescent
  • Adult
  • Animals
  • Cerebellar Neoplasms / mortality*
  • Cerebellar Neoplasms / pathology
  • Child
  • Child, Preschool
  • Female
  • Hedgehog Proteins / physiology
  • Homeodomain Proteins / analysis
  • Homeodomain Proteins / physiology*
  • Humans
  • Infant
  • Male
  • Medulloblastoma / mortality*
  • Medulloblastoma / pathology
  • Mice
  • Nerve Tissue Proteins / analysis
  • Nerve Tissue Proteins / physiology*
  • Repressor Proteins / analysis
  • Repressor Proteins / physiology*

Substances

  • BARHL1 protein, human
  • Barhl1 protein, mouse
  • Hedgehog Proteins
  • Homeodomain Proteins
  • Nerve Tissue Proteins
  • Repressor Proteins
  • SHH protein, human