Clinical use of a growth hormone receptor antagonist in the treatment of acromegaly

Trends Endocrinol Metab. 2001 Nov;12(9):408-13. doi: 10.1016/s1043-2760(01)00461-1.


The elucidation of the mechanisms by which growth hormone (GH) interacts with its receptor has facilitated the design of compounds that function as GH-receptor antagonists. One such compound, B2036, has been conjugated to polyethylene glycol to produce a drug, pegvisomant, that has a powerful ability to lower circulating concentrations of insulin-like growth factor I (IGF-I), the principal mediator of GH action, in patients with acromegaly and to improve the symptoms and signs associated with GH excess. This article describes the mechanism of action of GH-receptor antagonists, reviews the preclinical and clinical data on the use of pegvisomant and discusses some of the challenges that lie ahead in judging the efficacy of a treatment that, unlike established therapies for acromegaly, does not aim to modify the underlying cause of acromegaly, namely excess GH secretion, but aims to lower serum IGF-I levels to normal.

Publication types

  • Review

MeSH terms

  • Acromegaly / blood
  • Acromegaly / drug therapy*
  • Animals
  • Biomarkers
  • Human Growth Hormone / adverse effects
  • Human Growth Hormone / analogs & derivatives
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Insulin-Like Growth Factor I / analysis
  • Receptors, Somatotropin / antagonists & inhibitors*


  • Biomarkers
  • Receptors, Somatotropin
  • Human Growth Hormone
  • Insulin-Like Growth Factor I
  • pegvisomant