Vascular endothelial growth factor and the potential therapeutic use of pegaptanib (Macugen®) in diabetic retinopathy

C. Starita, M. Patel, B. Katz, A. Adamis

Research output: Chapter in Book/Report/Conference proceedingChapter

68 Scopus citations


Both clinical and preclinical findings have implicated vascular endothelial growth factor (VEGF) in the pathophysiology of diabetic macular edema (DME). VEGF is both a potent enhancer of vascular permeability and a key inducer of angiogenesis. VEGF levels are elevated in the eyes of patients with DME, and in animal models of diabetes this elevation coincides with the breakdown of the blood-retinal barrier. Moreover, injection of VEGF (the VEGF165 isoform in particular) into healthy eyes of animals can induce diabetes-associated ocular pathologies.Pegaptanib, a novel RNA aptamer currently used in the treatment of agerelated macular degeneration, binds and inactivates VEGF165 and has been shown in animal models to reverse the blood-retinal barrier breakdown associated with diabetes. These findings formed the basis of a phase II trial involving 172 patients with DME, in which intravitreous pegaptanib (0.3 mg, 1 mg, 3 mg) or sham injections were administered every 6 weeks for 12 weeks, with the option of continuing for 18 more weeks or undergoing laser treatment. Compared to sham, patients receiving 0.3 mg displayed superior visual acuity (p = 0.04) as well as a reduction in retinal thickness of 68 micrometers compared to a slight increase under sham treatment (p = 0.021). These data support the use of pegaptanib in the treatment of DME.

Original languageEnglish (US)
Title of host publicationDiabetic Retinopathy
EditorsGabriele Lang
Number of pages27
StatePublished - Dec 1 2007

Publication series

NameDevelopments in Ophthalmology
ISSN (Print)0250-3751
ISSN (Electronic)1662-2790

ASJC Scopus subject areas

  • Ophthalmology


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