Platelet-derived growth factor receptors: a therapeutic target in solid tumors

Semin Oncol. 2001 Oct;28(5 Suppl 17):27-33.


Platelet-derived growth factor (PDGF) was one of the first polypeptide growth factors identified that signals through a cell surface tyrosine kinase receptor (PDGF-R) to stimulate various cellular functions including growth, proliferation, and differentiation. Since then, several related genes have been identified constituting a family of ligands (primarily PDGF A and B) and their cognate receptors (PDGF-R alpha and beta). To date, PDGF expression has been shown in a number of different solid tumors, from glioblastomas to prostate carcinomas. In these various tumor types, the biologic role of PDGF signaling can vary from autocrine stimulation of cancer cell growth to more subtle paracrine interactions involving adjacent stroma and even angiogenesis. The tyrosine kinase inhibitor imatinib mesylate (formerly STI571, [Gleevec]; Novartis Pharmaceuticals Corp, East Hanover, NJ) blocks activity of the Bcr-Abl oncoprotein, and was recently approved for several indications in the treatment of chronic myeloid leukemia. Imatinib mesylate is also a potent inhibitor of the PDGF-R kinase and is currently being evaluated for the treatment of PDGF-responsive tumors such as prostate cancer. More clinical trials that investigate both established clinical endpoints of response and benefit, as well as surrogate endpoints that may describe the biologic significance of PDGF-R inhibition in vivo are needed to expand the applications that target the PDGF axis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Agents / therapeutic use
  • Benzamides
  • Clinical Trials as Topic
  • Enzyme Inhibitors / pharmacology*
  • Enzyme Inhibitors / therapeutic use
  • Humans
  • Imatinib Mesylate
  • Isoxazoles / pharmacology*
  • Isoxazoles / therapeutic use
  • Leflunomide
  • Ligands
  • Male
  • Piperazines / pharmacology*
  • Piperazines / therapeutic use
  • Platelet-Derived Growth Factor / metabolism
  • Prostatic Neoplasms / drug therapy*
  • Prostatic Neoplasms / metabolism
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Pyrimidines / pharmacology*
  • Pyrimidines / therapeutic use
  • Receptors, Platelet-Derived Growth Factor / antagonists & inhibitors*
  • Signal Transduction


  • Antineoplastic Agents
  • Benzamides
  • Enzyme Inhibitors
  • Isoxazoles
  • Ligands
  • Piperazines
  • Platelet-Derived Growth Factor
  • Pyrimidines
  • Imatinib Mesylate
  • Protein-Tyrosine Kinases
  • Receptors, Platelet-Derived Growth Factor
  • Leflunomide