title: An overview of experimental and early investigational therapies for the treatment of polycystic kidney disease.
abstract: BACKGROUND
  At present, treatment of autosomal dominant polycystic kidney disease (ADPKD) is essentially supportive as there is still no specific therapy. However, recent advances with ADPKD pathophysiology have stimulated research for new therapeutic strategies.
  
  
  METHODS
  The aim of this systematic review is to analyze the experimental and early investigational therapies currently under evaluation in this field. Data from completed clinical trials were retrieved from the currently available scientific literature and from the ClinicalTrials.gov website.
  
  
  CONCLUSIONS
  Among the drugs currently being explored, mammalian target of rapamycin inhibitors reduce kidney volume enlargement but their role remains uncertain. The most promising drug is the V2 receptor antagonist tolvaptan, which reduces the increased rate of total kidney volume and slows down glomerular filtration rate decline. The main candidates for the treatment of cysts growth, both in the kidney and in the liver whenever present, are the somatostatin analogues, such as lanreotide and octreotide and more recently pasireotide. As for other therapies, some favorable results have been achieved but data are still not sufficient to establish if these approaches may be beneficial in slowing ADPKD progression in the future.
authors.affiliation: University of Messina, AOU G. Martino PAD C, Department of Internal Medicine and Pharmacology , Via Consolare Valeria, 98100 Messina , Italy +39 090 2212331 ; +39 090 2212331 ; santisi@hotmail.com.
authors.forename: Domenico
authors.initials: D
authors.lastname: Santoro
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Pellicanò
authors.forename: Luca
authors.initials: L
authors.lastname: Visconti
authors.forename: Gianluca
authors.initials: G
authors.lastname: Trifirò
authors.forename: Michele
authors.initials: M
authors.lastname: Buemi
authors.forename: Valeria
authors.initials: V
authors.lastname: Cernaro
mesh_terms: D000818:Animals; D065092:Antidiuretic Hormone Receptor Antagonists / Q000494:pharmacology / Q000627:therapeutic use; D001552:Benzazepines / Q000494:pharmacology / Q000627:therapeutic use; D018450:Disease Progression; D015195:Drug Design; D015507:Drugs, Investigational / Q000494:pharmacology / Q000627:therapeutic use*; D005919:Glomerular Filtration Rate; D006801:Humans; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000503:physiopathology; D000077602:Tolvaptan
chemical_list: D065092:Antidiuretic Hormone Receptor Antagonists; D001552:Benzazepines; D015507:Drugs, Investigational; D000077602:Tolvaptan
keywords: mTOR; monoclonal; polycystic kidney disease; somatostatin; tolvaptan