Cellular and in vivo activity of a novel PI3K inhibitor, PX-866, against human glioblastoma

Neuro Oncol. 2010 Jun;12(6):559-69. doi: 10.1093/neuonc/nop058. Epub 2010 Feb 15.

Abstract

The phosphatidylinositol-3-kinase (PI3K)/Akt oncogenic pathway is critical in glioblastomas. Loss of PTEN, a negative regulator of the PI3K pathway or activated PI3K/Akt pathway that drive increased proliferation, survival, neovascularization, glycolysis, and invasion is found in 70%-80% of malignant gliomas. Thus, PI3K is an attractive therapeutic target for malignant glioma. We report that a new irreversible PI3K inhibitor, PX-866, shows potent inhibitory effects on the PI3K/Akt signaling pathway in glioblastoma. PX-866 did not induce any apoptosis in glioma cells; however, an increase in autophagy was observed. PX-866 inhibited the invasive and angiogenic capabilities of cultured glioblastoma cells. In vivo, PX-866 inhibited subcutaneous tumor growth and increased the median survival time of animals with intracranial tumors. We also assessed the potential of proton magnetic resonance spectroscopy (MRS) as a noninvasive method to monitor response to PX-866. Our findings show that PX-866 treatment causes a drop in the MRS-detectable choline-to-NAA, ratio and identify this partial normalization of the tumor metabolic profile as a biomarker of molecular drug action. Our studies affirm that the PI3K pathway is a highly specific molecular target for therapies for glioblastoma and other cancers with aberrant PI3K/PTEN expression.

Publication types

  • Comparative Study
  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Glioblastoma / drug therapy*
  • Glioblastoma / enzymology*
  • Glioblastoma / pathology
  • Gonanes / pharmacology
  • Gonanes / therapeutic use*
  • Humans
  • Mice
  • Mice, Nude
  • Phosphatidylinositol 3-Kinase / metabolism
  • Phosphoinositide-3 Kinase Inhibitors*
  • Xenograft Model Antitumor Assays / methods

Substances

  • Gonanes
  • PX-866
  • Phosphoinositide-3 Kinase Inhibitors
  • Phosphatidylinositol 3-Kinase