Discovery of Molecular Therapeutics for Glaucoma: Challenges, Successes, and Promising Directions

J Med Chem. 2016 Feb 11;59(3):788-809. doi: 10.1021/acs.jmedchem.5b00828. Epub 2015 Sep 25.

Abstract

Glaucoma, a heterogeneous ocular disorder affecting ∼60 million people worldwide, is characterized by painless neurodegeneration of retinal ganglion cells (RGCs), resulting in irreversible vision loss. Available therapies, which decrease the common causal risk factor of elevated intraocular pressure, delay, but cannot prevent, RGC death and blindness. Notably, it is changes in the anterior segment of the eye, particularly in the drainage of aqueous humor fluid, which are believed to bring about changes in pressure. Thus, it is primarily this region whose properties are manipulated in current and emerging therapies for glaucoma. Here, we focus on the challenges associated with developing treatments, review the available experimental methods to evaluate the therapeutic potential of new drugs, describe the development and evaluation of emerging Rho-kinase inhibitors and adenosine receptor ligands that offer the potential to improve aqueous humor outflow and protect RGCs simultaneously, and present new targets and approaches on the horizon.

Publication types

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

MeSH terms

  • Adrenergic Agonists / therapeutic use*
  • Adrenergic Antagonists / therapeutic use*
  • Adrenergic beta-Antagonists / therapeutic use*
  • Animals
  • Drug Discovery*
  • Glaucoma / drug therapy*
  • Humans
  • Ligands
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use*
  • Sympathomimetics / therapeutic use
  • rho-Associated Kinases / antagonists & inhibitors
  • rho-Associated Kinases / metabolism

Substances

  • Adrenergic Agonists
  • Adrenergic Antagonists
  • Adrenergic beta-Antagonists
  • Ligands
  • Protein Kinase Inhibitors
  • Sympathomimetics
  • rho-Associated Kinases