Suppression of drusen formation by compstatin, a peptide inhibitor of complement C3 activation, on cynomolgus monkey with early-onset macular degeneration

Adv Exp Med Biol. 2010:703:127-35. doi: 10.1007/978-1-4419-5635-4_9.


For the past 10 years, number of evidence has shown that activation of complement cascade has been associated with age-related macular degeneration (AMD). The genome wide association study in American population with dominantly dry-type AMD has revealed strong association with single nucleotide polymorphism (SNP) of complement genes. Protein composition of drusen, a deposit observed in sub-retinal space between Bruch's membrane and retinal pigment epithelial (RPE), contains active complement molecules in human and monkey. These evidences have leaded us to consider the possibility of suppressing complement cascade in the retina to delay or reverse the onset of AMD. To test is hypothesis we used the C3 inhibitor Compstatin on primate model with early-onset macular degeneration which develop drusen in less than 2 years after birth. Our preliminary result showed drusen disappearance after 6 months of intravitreal injection.

Publication types

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

MeSH terms

  • Age of Onset
  • Animals
  • Complement C3 / antagonists & inhibitors*
  • Complement System Proteins / genetics
  • Disease Models, Animal
  • Humans
  • Intravitreal Injections
  • Macaca fascicularis
  • Macular Degeneration / etiology
  • Macular Degeneration / pathology
  • Macular Degeneration / prevention & control*
  • Peptides, Cyclic / administration & dosage
  • Peptides, Cyclic / pharmacology*
  • Retinal Drusen / etiology
  • Retinal Drusen / pathology
  • Retinal Drusen / prevention & control*


  • Complement C3
  • Peptides, Cyclic
  • compstatin
  • Complement System Proteins