Investigational drugs for the treatment of endometriosis, an update on recent developments

Abstract

Endometriosis is a hormone-dependent benign chronic disease that requires a chronic medical therapy. Although currently available drugs are efficacious in treating endometriosis-related pain, some women experience partial or no improvement. Moreover, the recurrence of symptoms is expected after discontinuation of the therapies. Currently, new drugs are under intense clinical investigation for the treatment of endometriosis. Areas covered: This review aims to offer the reader a complete and updated overview on new investigational drugs and early molecular targets for the treatment of endometriosis. The authors describe the pre-clinical and clinical development of these agents. Expert opinion: Among the drugs under investigation, late clinical trials on gonadotropin-releasing hormone antagonists (GnRH-ant) showed the most promising results for the treatment of endometriosis. Aromatase inhibitors (AIs) are efficacious in treating endometriosis related pain symptoms but they cause significant adverse effects that limit their long-term use. New targets have been identified to produce drugs for the treatment of endometriosis, but the majority of these new compounds have only been investigated in laboratory studies or early clinical trials. Thus, further clinical research is required in order to elucidate their efficacy and safety in human.

Case–control study to develop and validate a questionnaire for the secondary prevention of endometriosis

“…This statement is in agreement with the pathogenetic theory of retrograde menstruation. Indeed, the pelvic contamination, as well as the implant risk, is higher due to more regular short periods and heavy bleeding lasting for more than seven days [3,7,45]. In our study, we did not register any connection between regularity of menstrual cycle and endometriosis.…”

“…Several previous studies [2,7] have shown that the characteristics of the menstrual cycle are strictly related to endometriosis: regular, short cycles and heavy bleeding lasting for more than seven days are associated with the development of the disease. This statement is in agreement with the pathogenetic theory of retrograde menstruation.…”

“…Currently, hormonal and biological therapies for the treatment of endometriosis are under intense clinical investigation [7]. However, the majority of these compounds have only been evaluated in pre-clinical studies or early clinical trials.…”

“…Thus, further extensive clinical research is necessary. Of note, late clinical trials on gonadotropin-releasing hormone antagonists (GnRH-ant) showed the most promising results for the treatment of endometriosis [7].…”

Thalidomide Reduces Cell Proliferation in Endometriosis Experimentally Induced in Rats

“…Thus, there still is the need to develop new medications for this purpose. 8 Thalidomide has a potent antiangiogenic effect based on the negative regulation of vascular endothelial growth factor (VEGF). In addition, thalidomide functions as an immunomod-ulator and as an immunosuppressor and anti-inflammatory agent since it acts on the excessive synthesis of Tumor necrosis factor-alpha (TNF-α) and of other cytokines such as interleukin 6, which participate in the genesis of inflammatory pain.…”