Pharmacological management of polycystic kidney disease

Abstract

Introduction: 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.

Areas covered: 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.

Expert opinion: 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.

Keywords::

• bosutinib
• everolimus
• lanreotide
• mammalian target of rapamycin
• octreotide
• polycystic kidney disease
• sirolimus
• somatostatin
• tolvaptan
• triptolide

Declaration of interest

The authors are supported by the Sino Swiss Science & Technology Cooperation (SSSTC) Program, the Hartmann-Müller Foundation, the Swiss National Science Foundation (320030-144093 to RP Wüthrich), and the National Natural Science Foundation of China General Projects (30971368, 30871179 to C Mei). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Notes

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