An overview of early drug development for endometriosis

Umberto Leone Roberti Maggiore; Simone Ferrero

doi:10.1517/13543784.2016.1126579

An overview of early drug development for endometriosis

Umberto Leone Roberti Maggiore, Simone Ferrero

Expert opinion on investigational drugs · 2015 · vol. 25(2) , pp. 227–247 · doi:10.1517/13543784.2016.1126579 · PMID:26619754 · W2189352963

Review OA: closed CC0 ⤵ 8 in-corpus citations

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This review surveys emerging laboratory and clinical therapies for endometriosis, focusing on gonadotropin-releasing hormone antagonists as a promising but not fully elucidated therapeutic option.

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Abstract

INTRODUCTION: Endometriosis is an estrogen-dependent chronic disease of women of fertile age requiring chronic therapy. Although available drugs have good efficacy and safety profiles, some patients experience partial or no improvement of pain with conventional treatment and recurrence of symptoms after discontinuation of the therapies. For these reasons, many new compounds are currently under investigation for the treatment of endometriosis. AREAS COVERED: This review offers the reader a complete and updated overview on emerging therapies for the treatment of endometriosis. The authors describe, in detail, the laboratory and clinical studies on these therapies and highlight the potential advantages and limitations associated with the administration of these new agents. EXPERT OPINION: Gonadotropin-releasing hormone antagonists are the most intriguing emerging agents for the treatment of patients with endometriosis. It should be noted that while there are a number of drugs under investigation, a large majority of these new compounds have only been investigated in laboratory studies with more extensive research required to better elucidate their efficacy and safety profiles.

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Condition tags

endometriosis

MeSH descriptors

Drug Design Drug Design Endometriosis Gonadotropin-Releasing Hormone Animals Animals Drugs, Investigational Drugs, Investigational Drugs, Investigational Drugs, Investigational Drugs, Investigational Endometriosis Endometriosis Endometriosis Female Female Gonadotropin-Releasing Hormone Gonadotropin-Releasing Hormone Humans Humans

Citation neighborhood (2-hop)

Papers in the corpus that this work cites (lower rings, blue) and that cite this one (upper rings, green). Dot size scales with the paper's in-corpus citation count — bigger dot = more influential within the endo/adeno field. Click a dot to open that paper. Outer rings show 2-hop neighbours — papers reached through the immediate citers/citees. [ collapse to 1-hop ]

References (100)

Cited by (8)

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[1] Altered retinoid uptake and action contributes to cell survival in endometriosis via openalex

[2] A matrix metalloproteinase inhibitor, ONO-4817, retards the development of mammary tumor and the progression of uterine adenomyosis in mice via openalex

[3] Angiostatic agents prevent the development of endometriosis-like lesions in the chicken chorioallantoic membrane via openalex

[4] A novel angiogenesis inhibitor bevacizumab induces apoptosis in the rat endometriosis model via openalex

[5] A novel noninvasive model of endometriosis for monitoring the efficacy of antiangiogenic therapy via openalex

[6] Antiangiogenesis therapy for endometriosis via openalex

[7] Antiangiogenic agents are effective inhibitors of endometriosis via openalex

[8] Anti-angiogenic effects of green tea catechin on an experimental endometriosis mouse model via openalex

[9] Anti-angiogenic treatment strategies for the therapy of endometriosis via openalex

[10] Antiprogestin and/or gonadotropin-releasing hormone agonist for endometriosis treatment and bone maintenance: a 1-year primate study via openalex

[11] A pilot study on the off-label use of valproic acid to treat adenomyosis via openalex

[12] Application of the nuclear factor-kappaB inhibitor BAY 11-7085 for the treatment of endometriosis: an in vitro study via openalex

[13] Application of the nuclear factor-κB inhibitor pyrrolidine dithiocarbamate for the treatment of endometriosis: an in vitro study via openalex

[14] A promise in the treatment of endometriosis: an observational cohort study on ovarian endometrioma reduction by N-acetylcysteine via openalex

[15] Atorvastatin causes regression of endometriotic implants in a rat model via openalex

[16] Bentamapimod (JNK Inhibitor AS602801) Induces Regression of Endometriotic Lesions in Animal Models via openalex

[17] Beta-hydroxyisovalerylshikonin induces apoptosis and G0/G1 cell-cycle arrest of endometriotic stromal cells: a preliminary in vitro study via openalex

[18] Bufalin induces apoptosis and the G0/G1 cell cycle arrest of endometriotic stromal cells: a promising agent for the treatment of endometriosis via openalex

[19] Clinical efficacy of the antiprogesterone RU486 in the treatment of endometriosis and uterine fibroids via openalex

[20] Combating endometriosis by blocking proteasome and nuclear factor-kappaB pathways via openalex

[21] Comparison of the effects of raloxifene and anastrozole on experimental endometriosis via openalex

[22] Costunolide induces apoptosis in human endometriotic cells through inhibition of the prosurvival Akt and nuclear factor kappa B signaling pathway via openalex

[23] Curcumin arrests endometriosis by downregulation of matrix metalloproteinase-9 activity via openalex

[24] Curcumin as anti-endometriotic agent: implication of MMP-3 and intrinsic apoptotic pathway via openalex

[25] Curcumin attenuates TNF-α-induced expression of intercellular adhesion molecule-1, vascular cell adhesion molecule-1 and proinflammatory cytokines in human endometriotic stromal cells via openalex

[26] Current pharmacotherapy for endometriosis via openalex

[27] Deep dyspareunia and sex life after laparoscopic excision of endometriosis via openalex

[28] Detection of apoptosis in human endometriotic tissues via openalex

[29] Dopamine agonist administration causes a reduction in endometrial implants through modulation of angiogenesis in experimentally induced endometriosis via openalex

[30] Effect of the immunomodulator leflunomide on the induction of endometriosis in an experimental rat model via openalex

[31] Effect of tumor necrosis factor-alpha on adhesion of human endometrial stromal cells to peritoneal mesothelial cells: an in vitro system via openalex

[32] Effect of vascular endothelial growth factor inhibition on endometrial implant development in a murine model of endometriosis via openalex

[33] Effects of hyperprolactinemia treatment with the dopamine agonist quinagolide on endometriotic lesions in patients with endometriosis-associated hyperprolactinemia via openalex

[34] Effects of oxidants and antioxidants on proliferation of endometrial stromal cells via openalex

[35] Effects of Pazopanib, Sunitinib, and Sorafenib, Anti-VEGF Agents, on the Growth of Experimental Endometriosis in Rats via openalex

[36] Efficacy of anti-tumor necrosis factor therapy in the treatment of spontaneous endometriosis in baboons via openalex

[37] Efficacy of anti-tumor necrosis factor therapy on endometriosis in an experimental rat model via openalex

[38] Efficacy of imiquimod, an immunomodulatory agent, on experimental endometriosis via openalex

[39] Elagolix, an Oral GnRH Antagonist for Endometriosis-Associated Pain: A Randomized Controlled Study via openalex

[40] Elagolix, an oral GnRH antagonist, versus subcutaneous depot medroxyprogesterone acetate for the treatment of endometriosis: effects on bone mineral density via openalex

[41] Elagolix treatment for endometriosis-associated pain: results from a phase 2, randomized, double-blind, placebo-controlled study via openalex

[42] Endocrine responses to long-term administration of the antiprogesterone RU486 in patients with pelvic endometriosis via openalex

[43] Endometriosis expresses a molecular pattern consistent with decreased retinoid uptake, metabolism and action via openalex

[44] Endostatin inhibits the growth of endometriotic lesions but does not affect fertility via openalex

[45] Epigallocatechin-3-gallate inhibits estrogen-induced activation of endometrial cells in vitro and causes regression of endometriotic lesions in vivo via openalex

[46] Estrogen-induced stromal cell-derived factor-1 (SDF-1/Cxcl12) expression is repressed by progesterone and by Selective Estrogen Receptor Modulators via estrogen receptor alpha in rat uterine cells and tissues via openalex

[47] FR 167653, a p38 mitogen-activated protein kinase inhibitor, suppresses the development of endometriosis in a murine model via openalex

[48] Gonadotrophin-releasing hormone analogue or dienogest plus estradiol valerate to prevent pain recurrence after laparoscopic surgery for endometriosis: a multi-center randomized trial via openalex

[49] Gonadotropin-releasing hormone analogues reduce the proliferation of endometrial stromal cells but not endometriotic cells via openalex

[50] High-dose atorvastatin causes regression of endometriotic implants: a rat model via openalex

[51] High omega-3:omega-6 fatty acid ratios in culture medium reduce endometrial-cell survival in combined endometrial gland and stromal cell cultures from women with and without endometriosis via openalex

[52] High prevalence of endometriosis in infertile women with normal ovulation and normospermic partners via openalex

[53] Human chorionic gonadotrophin attenuates NF-kappaB activation and cytokine expression of endometriotic stromal cells via openalex

[54] Immunological aspects of endometriosis: an update via openalex

[55] Inhibition of macrophage migration inhibitory factor reduces endometriotic implant size in mice with experimentally induced disease via openalex

[56] Inhibition of Macrophage Migration Inhibitory Factor Reduces Endometriotic Implant Size in Mice with Experimentally Induced Disease via openalex

[57] Inhibition of proliferation of endometrial stromal cells by trichostatin A, RU486, CDB-2914, N-acetylcysteine, and ICI 182780 via openalex

[58] Inhibitory effect of curcumin on angiogenesis in ectopic endometrium of rats with experimental endometriosis via openalex

[59] Involvement of nuclear factor-kappaB in macrophage migration inhibitory factor gene transcription up-regulation induced by interleukin- 1 beta in ectopic endometrial cells via openalex

[60] Involvement of the nuclear factor-κB pathway in the pathogenesis of endometriosis via openalex

[61] Laparoscopic surgery for pelvic pain associated with endometriosis via openalex

[62] Leflunomide--an immunomodulator--induces regression of endometrial explants in a rat model of endometriosis via openalex

[63] Macrophage migration inhibitory factor antagonist blocks the development of endometriosis in vivo via openalex

[64] Mechanistic and therapeutic implications of angiogenesis in endometriosis via openalex

[65] Metformin regresses endometriotic implants in rats by improving implant levels of superoxide dismutase, vascular endothelial growth factor, tissue inhibitor of metalloproteinase-2, and matrix metalloproteinase-9 via openalex

[66] Metformin suppresses interleukin (IL)-1beta-induced IL-8 production, aromatase activation, and proliferation of endometriotic stromal cells via openalex

[67] Mifepristone: where do we come from and where are we going? Clinical development over a quarter of a century via openalex

[68] Modern combined oral contraceptives for pain associated with endometriosis via openalex

[69] More than antioxidant: N-acetyl-L-cysteine in a murine model of endometriosis via openalex

[70] Naproxen sodium in dysmenorrhea secondary to endometriosis via openalex

[71] Nonsteroidal antiinflammatory drugs differentially suppress endometriosis in a murine model via openalex

[72] Novel therapies targeting endometriosis via openalex

[73] Nuclear peroxisome proliferator-activated receptors alpha and gamma have opposing effects on monocyte chemotaxis in endometriosis via openalex

[74] On-label and off-label drug use in the treatment of endometriosis via openalex

[75] Oral eicosapentaenoic acid supplementation as possible therapy for endometriosis via openalex

[76] Pain, quality of life and complications following the radical resection of rectovaginal endometriosis via openalex

[77] Peritoneal fluid-mediated enhancement of eutopic and ectopic endometrial cell proliferation is dependent on tumor necrosis factor-alpha in women with endometriosis via openalex

[78] Possible pathophysiological roles of mitogen-activated protein kinases (MAPKs) in endometriosis via openalex

[79] Postoperative medical therapy after surgical treatment of endometriosis: from adjuvant therapy to tertiary prevention via openalex

[80] Prevalence of newly diagnosed endometriosis in women attending the general practitioner via openalex

[81] Prodrug of green tea epigallocatechin-3-gallate (Pro-EGCG) as a potent anti-angiogenesis agent for endometriosis in mice via openalex

[82] Rapamycin induces regression of endometriotic lesions by inhibiting neovascularization and cell proliferation via openalex

[83] Recombinant human TNFRSF1A (r-hTBP1) inhibits the development of endometriosis in baboons: a prospective, randomized, placebo- and drug-controlled study via openalex

[84] Regression of endometrial autografts in a rat model of endometriosis treated with etanercept via openalex

[85] Remission of endometriosis by hyperbaric oxygen treatment in rats via openalex

[86] Retinoic acid suppresses growth of lesions, inhibits peritoneal cytokine secretion, and promotes macrophage differentiation in an immunocompetent mouse model of endometriosis via openalex

[87] Return of chronic pelvic pain from endometriosis after raloxifene treatment: a randomized controlled trial via openalex

[88] SB203580, a p38 mitogen-activated protein kinase inhibitor, suppresses the development of endometriosis by down-regulating proinflammatory cytokines and proteolytic factors in a mouse model via openalex

[89] Selective progesterone receptor modulators and progesterone antagonists: mechanisms of action and clinical applications via openalex

[90] Selective progesterone receptor modulators (SPRMs): a novel therapeutic concept in endometriosis via openalex

[91] Simvastatin inhibits the proliferation and the contractility of human endometriotic stromal cells: a promising agent for the treatment of endometriosis via openalex

[92] Simvastatin protects against the development of endometriosis in a nude mouse model via openalex

[93] Sorafenib inhibits growth, migration, and angiogenic potential of ectopic endometrial mesenchymal stem cells derived from patients with endometriosis via openalex

[94] Suppression of gonadotropins and estradiol in premenopausal women by oral administration of the nonpeptide gonadotropin-releasing hormone antagonist elagolix via openalex

[95] Suppression of IL-1beta-induced COX-2 expression by trichostatin A (TSA) in human endometrial stromal cells via openalex

[96] Systematic review of the effects of aromatase inhibitors on pain associated with endometriosis via openalex

[97] Thalidomide inhibits tumor necrosis factor-alpha-induced interleukin-8 expression in endometriotic stromal cells, possibly through suppression of nuclear factor-kappaB activation via openalex

[98] The antibody-mediated targeted delivery of interleukin-10 inhibits endometriosis in a syngeneic mouse model via openalex

[99] The effects of ergot and non-ergot-derived dopamine agonists in an experimental mouse model of endometriosis via openalex

[100] The effects of metformin and letrozole on endometriosis and comparison of the two treatment agents in a rat model via openalex