Retinal angiogenesis suppression through small molecule activation of p53

J Clin Invest. 2013 Oct;123(10):4170-81. doi: 10.1172/JCI67315. Epub 2013 Sep 9.

Abstract

Neovascular age-related macular degeneration is a leading cause of irreversible vision loss in the Western world. Cytokine-targeted therapies (such as anti-vascular endothelial growth factor) are effective in treating pathologic ocular angiogenesis, but have not led to a durable effect and often require indefinite treatment. Here, we show that Nutlin-3, a small molecule antagonist of the E3 ubiquitin protein ligase MDM2, inhibited angiogenesis in several model systems. We found that a functional p53 pathway was essential for Nutlin-3-mediated retinal antiangiogenesis and disruption of the p53 transcriptional network abolished the antiangiogenic activity of Nutlin-3. Nutlin-3 did not inhibit established, mature blood vessels in the adult mouse retina, suggesting that only proliferating retinal vessels are sensitive to Nutlin-3. Furthermore, Nutlin-3 inhibited angiogenesis in nonretinal models such as the hind limb ischemia model. Our work demonstrates that Nutlin-3 functions through an antiproliferative pathway with conceivable advantages over existing cytokine-targeted antiangiogenesis therapies.

Publication types

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

MeSH terms

  • Angiogenesis Inhibitors / pharmacology*
  • Animals
  • Apoptosis
  • Cell Proliferation
  • Cell Survival
  • Cells, Cultured
  • Hindlimb / blood supply
  • Human Umbilical Vein Endothelial Cells / drug effects
  • Human Umbilical Vein Endothelial Cells / physiology
  • Humans
  • Imidazoles / pharmacology*
  • Ischemia / drug therapy
  • Macular Degeneration / drug therapy
  • Mice
  • Mice, 129 Strain
  • Mice, Inbred C57BL
  • Myocytes, Smooth Muscle / drug effects
  • Myocytes, Smooth Muscle / physiology
  • Neovascularization, Physiologic / drug effects*
  • Piperazines / pharmacology*
  • Rats
  • Retinal Vessels / drug effects
  • Retinal Vessels / physiology*
  • Transcriptional Activation / drug effects
  • Tumor Suppressor Protein p53 / genetics
  • Tumor Suppressor Protein p53 / metabolism*

Substances

  • Angiogenesis Inhibitors
  • Imidazoles
  • Piperazines
  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • nutlin 3