Related Experiment Video

Updated: Aug 1, 2025

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
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Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring

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Autosomal Dominant Polycystic Kidney Disease Therapies on the Horizon

Julie Xia Zhou1, Vicente E Torres1

  • 1Department of Nephrology, Mayo Clinic, Rochester, MN.

Advances in Kidney Disease and Health
|April 23, 2023
PubMed

Insights

Autosomal dominant polycystic kidney disease (ADPKD) treatments focus on vasopressin antagonists like tolvaptan. This review explores improving tolvaptan tolerability and alternative therapies for ADPKD cyst growth.

Area of Science:

  • Nephrology
  • Pharmacology
  • Genetics

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) causes kidney failure through cyst formation.
  • Vasopressin signaling is a key driver of cystogenesis in ADPKD.
  • Tolvaptan is an FDA-approved drug for rapid ADPKD progression, but has tolerability issues.

Purpose of the Study:

  • To review strategies for improving tolvaptan tolerability in ADPKD patients.
  • To discuss alternative vasopressin 2 receptor antagonists, such as lixivaptan.
  • To explore novel pharmacotherapies and dietary interventions for ADPKD.

Main Methods:

  • Review of preclinical and clinical studies on ADPKD treatments.
  • Analysis of vasopressin receptor antagonism and downstream signaling pathways.
  • Evaluation of emerging therapeutic strategies and dietary interventions.

Main Results:

  • Tolvaptan ameliorates cyst growth but causes aquaretic events.
  • Lixivaptan and somatostatin analogs are being investigated as alternatives.
  • New pharmacotherapies targeting different pathways show promise in preclinical studies.

Conclusions:

  • Improving tolvaptan tolerability is crucial for widespread ADPKD treatment.
  • Alternative drug targets and dietary interventions offer new hope for managing ADPKD.
  • Continued research into ADPKD pathophysiology is essential for developing effective therapies.
Keywords:
ADPKD therapiesCell proliferationCyst fluid secretionCyst growth

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