Emerging drugs for endometriosis

Abstract

Background: Endometriosis is a benign, common gynecological disorder affecting mostly women of reproductive age. It is associated with dysmenorrhea, pelvic pain, and subfertility. The current non-surgical medical treatment is not satisfactory and there is a pressing need for novel therapeutics with better efficacy, tolerability and safety profiles. Objective: To investigate the development of new therapies for endometriosis. Methods: The PubMed database has been extensively searched for all compounds tested preclinically and clinically. Results/conclusion: Besides the three main classes of drugs, i.e., GnRH agonists, progestins, and androgenic agents, there are several classes of compounds that have been tested preclinically and clinically. Surprisingly, a considerable proportion of officially registered Phase II/III clinical trials are listed as completed yet no information on their outcomes is available. Three completed and published Phase II trials found the tested compound disappointing. Another two Phase II clinical trials have been suspended. This apparent gap between the generally promising preclinical findings and the clinical trial outcomes reflects our current woefully inadequate understanding of the mechanisms underlying endometriosis-associated pain and subferitility and recurrence of endometriosis; questions the adequacy and value of animal models of endometriosis that are still in use; highlights the difficulty in developing new therapeutics for endometriosis; and calls for more research in the mechanisms underlying endometriosis-associated pain and subferitility and recurrence of endometriosis.

Keywords::

• clinical trials
• drug
• endometriosis
• pain
• treatment

Acknowledgments

This research was supported in part by grant 074119517 from the Shanghai Science and Technology Commission and grant 30872759 from the National Science Foundation of China. The author thanks Dr Thomas D'Hooghe for his helpful comment on the TNF-α blockers subsection.

Notes