title: Better understanding of ADPKD results in potential new treatment options: ready for the cure?
abstract: Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common hereditary disorders. It accounts for 6% of the incidence of end-stage renal disease in Europe. Over the last decade, knowledge of the pathology underlying this disease has increased rapidly. Attributing important roles to tubular cell ciliary functioning, cell proliferation and fluid secretion, subsequent alterations in levels of intracellular calcium, adenosine 3',5'-cyclic monophosphate (cAMP) and activation of a variety of cellular kinases, including mammalian target of rapamycin (mTOR), has laid out the foundations for development of potentially effective treatments. In this editorial, the possible therapeutic roles for vasopressin antagonists, rapamycin, somatostatin and roscovitine are discussed. Clinical trials have been started to investigate the efficacy and safety of these agents for treating ADPKD in humans.
authors.affiliation: Division of Nephrology, Department of Internal Medicine, University Medical Center Groningen, Groningen, The Netherlands. esther.meijer@int.umcg.nl
authors.forename: Esther
authors.initials: E
authors.lastname: Meijer
authors.forename: Paul E
authors.initials: PE
authors.lastname: de Jong
authors.forename: Dorien J
authors.initials: DJ
authors.lastname: Peters
authors.forename: Ronald T
authors.initials: RT
authors.lastname: Gansevoort
mesh_terms: D000818:Animals; D065092:Antidiuretic Hormone Receptor Antagonists; D006801:Humans; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000503:physiopathology; D011687:Purines / Q000627:therapeutic use; D000077546:Roscovitine; D020123:Sirolimus / Q000627:therapeutic use; D013004:Somatostatin / Q000627:therapeutic use
chemical_list: D065092:Antidiuretic Hormone Receptor Antagonists; D011687:Purines; D000077546:Roscovitine; D013004:Somatostatin; D020123:Sirolimus