Emerging drugs for endometriosis.
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.


CONCLUSIONS
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.
Guo
Sun-Wei
SW
Shanghai Jiao Tong University School of Medicine, Renji Hospital, and Institute of Obstetric and Gynecologic Research, 145 Shandong Zhong Road, Shanghai 200001, China. hoxa10@gmail.com
D000818:Animals; D055808:Drug Discovery; D015507:Drugs, Investigational / Q000627:therapeutic use*; D004715:Endometriosis / Q000150:complications / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007247:Infertility, Female / Q000209:etiology / Q000517:prevention & control; D010146:Pain / Q000209:etiology / Q000517:prevention & control; D012008:Recurrence; D016896:Treatment Outcome
D015507:Drugs, Investigational