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Updated: Jun 21, 2026

Implementing Patch Clamp and Live Fluorescence Microscopy to Monitor Functional Properties of Freshly Isolated PKD Epithelium
08:46

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Published on: September 1, 2015

Mammalian target of rapamycin and autosomal dominant polycystic kidney disease

R P Wüthrich1, A L Serra

  • 1Division of Nephrology, University Hospital, Zürich, Switzerland. Rudolf.wuethrich@usz.ch

Transplantation Proceedings
|August 5, 2009
PubMed

Insights

Sirolimus, an mTOR inhibitor, shows promise in slowing Autosomal dominant polycystic kidney disease (ADPKD) progression. This study investigates sirolimus

Area of Science:

  • Nephrology
  • Pharmacology
  • Genetics

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) causes kidney cysts, leading to renal function loss.
  • Mammalian target of rapamycin (mTOR) inhibitors reduced cyst growth in animal models.
  • Sirolimus treatment regressed cystic kidney volumes in ADPKD renal transplant patients.

Purpose of the Study:

  • To investigate if low-dose sirolimus (2 mg/d) retards cyst growth in ADPKD patients.
  • To determine if sirolimus slows renal functional deterioration in ADPKD.
  • To assess the potential of mTOR inhibition to delay renal replacement therapy.

Main Methods:

  • An 18-month, prospective, controlled, open-label clinical trial.
  • Two parallel groups of patients with ADPKD.
  • Sirolimus administered at a low daily dose.

Main Results:

  • Preliminary results were anticipated in 2010.
  • The study aimed to demonstrate a reduction in cyst growth and renal function decline.

Conclusions:

  • Sirolimus may slow ADPKD progression.
  • mTOR inhibition could delay the need for dialysis or transplantation in ADPKD patients.

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