Targeting heat shock protein 90 for the treatment of malignant pheochromocytoma

PLoS One. 2013;8(2):e56083. doi: 10.1371/journal.pone.0056083. Epub 2013 Feb 14.

Abstract

Metastatic pheochromocytoma represents one of the major clinical challenges in the field of neuroendocrine oncology. Recent molecular characterization of pheochromocytoma suggests new treatment options with targeted therapies. In this study we investigated the 90 kDa heat shock protein (Hsp90) as a potential therapeutic target for advanced pheochromocytoma. Both the first generation, natural product Hsp90 inhibitor 17-allylamino-17-demethoxygeldanamycin (17-AAG, tanespimycin), and the second-generation synthetic Hsp90 inhibitor STA-9090 (ganetespib) demonstrated potent inhibition of proliferation and migration of pheochromocytoma cell lines and induced degradation of key Hsp90 clients. Furthermore, ganetespib induced dose-dependent cytotoxicity in primary pheochromocytoma cells. Using metastatic models of pheochromocytoma, we demonstrate the efficacy of 17-AAG and ganetespib in reducing metastatic burden and increasing survival. Levels of Hsp70 in plasma from the xenograft studies served as a proximal biomarker of drug treatment. Our study suggests that targeting Hsp90 may benefit patients with advanced pheochromocytoma.

Publication types

  • Research Support, N.I.H., Intramural

MeSH terms

  • Adrenal Gland Neoplasms / drug therapy*
  • Adrenal Gland Neoplasms / metabolism
  • Adrenal Gland Neoplasms / pathology
  • Adrenal Glands / drug effects
  • Adrenal Glands / metabolism
  • Adrenal Glands / pathology
  • Animals
  • Apoptosis / drug effects
  • Benzoquinones / pharmacology
  • Benzoquinones / therapeutic use*
  • Cell Line, Tumor
  • Cell Movement / drug effects
  • Cell Proliferation / drug effects
  • Cells, Cultured
  • Female
  • HSP90 Heat-Shock Proteins / antagonists & inhibitors*
  • HSP90 Heat-Shock Proteins / metabolism
  • Humans
  • Lactams, Macrocyclic / pharmacology
  • Lactams, Macrocyclic / therapeutic use*
  • Mice
  • Mice, Nude
  • Molecular Targeted Therapy*
  • Neoplasm Metastasis / drug therapy
  • Neoplasm Metastasis / pathology
  • Pheochromocytoma / drug therapy*
  • Pheochromocytoma / metabolism
  • Pheochromocytoma / pathology
  • Rats
  • Triazoles / pharmacology
  • Triazoles / therapeutic use*

Substances

  • Benzoquinones
  • HSP90 Heat-Shock Proteins
  • Lactams, Macrocyclic
  • STA 9090
  • Triazoles
  • tanespimycin