title: Pharmacological management of polycystic kidney disease.
abstract: BACKGROUND
  Autosomal-dominant polycystic kidney disease (ADPKD) represents a therapeutic challenge as effective treatment to retard the growth of cysts in the kidneys and the liver has not been available despite decades of intense basic and clinical research.
  
  
  METHODS
  Several clinical trials have been performed in recent years to study the effect of diverse drugs on the growth of renal and hepatic cysts, and on functional deterioration of the glomerular filtration rate. The drug classes that have been tested in randomized clinical trials include the mammalian target of rapamycin (mTOR) inhibitors, sirolimus and everolimus, the somatostatin analogues (octreotide, lanreotide, pasireotide), and most recently, the vasopressin V2 receptor antagonist, tolvaptan. The results with the mTOR inhibitors were disappointing, but more encouraging with the somatostatin analogues and with tolvaptan. Additional drugs are being tested, which include among others, the SRC-ABL tyrosine kinase inhibitor, bosutinib, and the traditional Chinese herbal medication, triptolide. Additional therapeutic strategies to retard cyst growth aim at blood pressure control via inhibition of the renin-angiotensin system and the sympathetic nervous system.
  
  
  CONCLUSIONS
  Given the accumulated knowledge, it is currently uncertain whether drugs will become available in the near future to significantly change the course of the relentlessly progressing polycystic kidney disease.
authors.affiliation: University Hospital, Division of Nephrology , Rämistrasse 100, 8091 Zürich , Switzerland +41 44 255 33 84 ; +41 44 255 45 93 ; rudolf.wuethrich@usz.ch.
authors.forename: Rudolf P
authors.initials: RP
authors.lastname: Wüthrich
authors.forename: Changlin
authors.initials: C
authors.lastname: Mei
mesh_terms: D000818:Animals; D001552:Benzazepines / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D004353:Drug Evaluation, Preclinical; D005919:Glomerular Filtration Rate / Q000187:drug effects; D006801:Humans; D006973:Hypertension / Q000188:drug therapy; D007668:Kidney / Q000187:drug effects* / Q000473:pathology / Q000503:physiopathology; D015282:Octreotide / Q000627:therapeutic use; D009929:Organ Size; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000503:physiopathology; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D000077602:Tolvaptan; D016896:Treatment Outcome
chemical_list: D001552:Benzazepines; D047428:Protein Kinase Inhibitors; D000077602:Tolvaptan; D015282:Octreotide
keywords: bosutinib; everolimus; lanreotide; mammalian target of rapamycin; octreotide; polycystic kidney disease; sirolimus; somatostatin; tolvaptan; triptolide