title: [Autosomal dominant polycystic kidney disease: is the treatment for tomorrow?].
abstract: Autosomal dominant polycystic kidney disease (ADPKD) is the most frequent Mendelian inherited disorder. It covers 6.1% of incident ESRD patients in France in 2011. Long left untreated, this disease will soon benefit from targeted therapies currently under evaluation. Several molecules have already reached the stage of clinical trials: the evaluation of mTOR inhibitors yielded deceiving results and, more recently, 2 different molecules demonstrated a slight impact on the progression of total kidney volume (TKV): tolvaptan, vasopressin receptor-V2 inhibitor and somatostatin analogues; both of these molecules acting throughout the decrease of intracellular AMPc. The purpose of this review is to briefly describe the signaling pathways involved, then to present both the published and ongoing clinical trials and the promising molecules evaluated in murine models.
authors.affiliation: Service de néphrologie, hémodialyse et transplantation rénale, hôpital la Cavale-Blanche, 15, boulevard Tanguy-Prigent, 29609 Brest cedex, France; Université de Bretagne occidentale, université Européenne de Bretagne, 3, rue des Archives, 29238 Brest, France; Institut national de la sante et de la recherche medicale (INSERM), U1078, 46, rue Félix-le-Dantec, 29218 Brest cedex 2, France. Electronic address: emilie.cornec-legall@chu-brest.fr.
authors.forename: Emilie
authors.initials: E
authors.lastname: Cornec-Le Gall
authors.affiliation: Service de néphrologie, hémodialyse et transplantation rénale, hôpital la Cavale-Blanche, 15, boulevard Tanguy-Prigent, 29609 Brest cedex, France; Université de Bretagne occidentale, université Européenne de Bretagne, 3, rue des Archives, 29238 Brest, France.
authors.forename: Yannick
authors.initials: Y
authors.lastname: Le Meur
mesh_terms: D000818:Animals; D065092:Antidiuretic Hormone Receptor Antagonists / Q000627:therapeutic use; D001552:Benzazepines / Q000627:therapeutic use; D019468:Disease Management; D004195:Disease Models, Animal; D015507:Drugs, Investigational / Q000627:therapeutic use; D004847:Epithelial Cells / Q000473:pathology; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007676:Kidney Failure, Chronic / Q000209:etiology; D051379:Mice; D008954:Models, Biological; D015337:Multicenter Studies as Topic; D016891:Polycystic Kidney, Autosomal Dominant / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D016032:Randomized Controlled Trials as Topic; D017483:Receptors, Vasopressin / Q000502:physiology; D012084:Renin-Angiotensin System / Q000187:drug effects; D015398:Signal Transduction / Q000187:drug effects; D013004:Somatostatin / Q000031:analogs & derivatives; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors; D000077602:Tolvaptan
chemical_list: D065092:Antidiuretic Hormone Receptor Antagonists; D001552:Benzazepines; D015507:Drugs, Investigational; D007166:Immunosuppressive Agents; D017483:Receptors, Vasopressin; D000077602:Tolvaptan; D013004:Somatostatin; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases
keywords: Analogues de la somatostatine; Autosomal dominant polycystic kidney disease; Chronic kidney disease; Drug therapy; Everolimus; Genetic diseases; Génétique; Inhibiteur de la voie des mTOR; Inhibiteurs des récepteurs V2 de la vasopressine; Insuffisance rénale chronique; Polykystose rénale autosomique dominante; Receptors; Sirolimus; Somatostatin analogs; Thérapeutique; Vasopressin/antagonists and inhibitors