Leuprolide acetate: a drug of diverse clinical applications

Expert Opin Investig Drugs. 2007 Nov;16(11):1851-63. doi: 10.1517/13543784.16.11.1851.


Leuprolide acetate is a synthetic nonapeptide that is a potent gonadotropin-releasing hormone receptor (GnRHR) agonist used for diverse clinical applications, including the treatment of prostate cancer, endometriosis, uterine fibroids, central precocious puberty and in vitro fertilization techniques. As its basic mechanism of action, leuprolide acetate suppresses gonadotrope secretion of luteinizing hormone and follicle-stimulating hormone that subsequently suppresses gonadal sex steroid production. In addition, leuprolide acetate is presently being tested for the treatment of Alzheimer's disease, polycystic ovary syndrome, functional bowel disease, short stature, premenstrual syndrome and even as an alternative for contraception. Mounting evidence suggests that GnRH agonist suppression of serum gonadotropins may also be important in many of the clinical applications described above. Moreover, the presence of GnRHR in a multitude of non-reproductive tissues including the recent discovery of GnRHR expression in the hippocampi and cortex of the human brain indicates that GnRH analogs such as leuprolide acetate may also act directly via tissue GnRHRs to modulate (brain) function. Thus, the molecular mechanisms underlying the therapeutic effect of GnRH analogs in the treatment of these diseases may be more complex than originally thought. These observations also suggest that the potential uses of GnRH analogs in the modulation of GnRH signaling and treatment of disease has yet to be fully realized.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Endometriosis / drug therapy
  • Female
  • Fertility Agents, Female / therapeutic use
  • Gonadotropin-Releasing Hormone / agonists
  • Gonadotropin-Releasing Hormone / analogs & derivatives
  • Gonadotropin-Releasing Hormone / metabolism
  • Humans
  • Leiomyoma / drug therapy
  • Leuprolide / adverse effects
  • Leuprolide / pharmacology
  • Leuprolide / therapeutic use*
  • Male
  • Prostatic Neoplasms / drug therapy
  • Puberty, Precocious / drug therapy
  • Receptors, LHRH / agonists*


  • Fertility Agents, Female
  • Receptors, LHRH
  • Gonadotropin-Releasing Hormone
  • Leuprolide