Novel agents for the medical treatment of endometriosis

Current medical treatments for endometriosis-associated pain, including oral contraceptives, progestins and GnRH agonists, are partially effective and have significant side-effects. The purpose of this review is to present new hormonal and nonhormonal treatment for endometriosis.

At present, the ideal drug that can prevent, inhibit or stop development of endometriosis, reduce associated pain or infertility and allow conception does not exist. New drugs in development for endometriosis modulate GnRH, estrogen and/or progesterone receptors, or target endometriosis-associated inflammation, angiogenesis, adhesion and/or tissue invasion. Most have been tested in rodents, and have been evaluated in more relevant animal models like nonhuman primates (baboons), but only a few, that is GnRH antagonists, have been tested in human randomized controlled trials. Important safety and efficacy issues remain a concern, as steroid receptors, inflammation, adhesion, angiogenesis and tissue invasion are key factors in physiological events like ovulation, menstruation and embryo implantation.

New drugs for the medical treatment of endometriosis targeting both hormonal (GnRH, estrogen and progesterone receptors) and nonhormonal pathways (inflammation, angiogenesis, adhesions, tissue invasion) are promising, but their efficacy and safety need to be established in randomized human trials before they can be used in clinical practice.