Preclinical evaluation of radiation and perifosine in a genetically and histologically accurate model of brainstem glioma

Cancer Res. 2010 Mar 15;70(6):2548-57. doi: 10.1158/0008-5472.CAN-09-2503. Epub 2010 Mar 2.

Abstract

Brainstem gliomas (BSG) are a rare group of central nervous system tumors that arise mostly in children and usually portend a particularly poor prognosis. We report the development of a genetically engineered mouse model of BSG using the RCAS/tv-a system and its implementation in preclinical trials. Using immunohistochemistry, we found that platelet-derived growth factor (PDGF) receptor alpha is overexpressed in 67% of pediatric BSGs. Based on this observation, we induced low-grade BSGs by overexpressing PDGF-B in the posterior fossa of neonatal nestin tv-a mice. To generate high-grade BSGs, we overexpressed PDGF-B in combination with Ink4a-ARF loss, given that this locus is commonly lost in high-grade pediatric BSGs. We show that the likely cells of origin for these mouse BSGs exist on the floor of the fourth ventricle and cerebral aqueduct. Irradiation of these high-grade BSGs shows that although single doses of 2, 6, and 10 Gy significantly increased the percent of terminal deoxynucleotidyl transferase-mediated dUTP nick end labeling (TUNEL)-positive nuclei, only 6 and 10 Gy significantly induce cell cycle arrest. Perifosine, an inhibitor of AKT signaling, significantly induced TUNEL-positive nuclei in this high-grade BSG model, but in combination with 10 Gy, it did not significantly increase the percent of TUNEL-positive nuclei relative to 10 Gy alone at 6, 24, and 72 hours. Survival analysis showed that a single dose of 10 Gy significantly prolonged survival by 27% (P = 0.0002) but perifosine did not (P = 0.92). Perifosine + 10 Gy did not result in a significantly increased survival relative to 10 Gy alone (P = 0.23). This PDGF-induced BSG model can serve as a preclinical tool for the testing of novel agents.

Publication types

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

MeSH terms

  • Animals
  • Brain Stem Neoplasms / drug therapy*
  • Brain Stem Neoplasms / genetics
  • Brain Stem Neoplasms / pathology
  • Brain Stem Neoplasms / radiotherapy*
  • Combined Modality Therapy
  • Disease Models, Animal*
  • Genetic Engineering
  • Glioma / drug therapy*
  • Glioma / genetics
  • Glioma / pathology
  • Glioma / radiotherapy*
  • Inbreeding
  • Mice
  • Mice, Inbred BALB C
  • Phosphorylcholine / analogs & derivatives*
  • Phosphorylcholine / pharmacology
  • Receptor, Platelet-Derived Growth Factor alpha / biosynthesis

Substances

  • Phosphorylcholine
  • perifosine
  • Receptor, Platelet-Derived Growth Factor alpha