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Updated: May 12, 2026

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
Published on: June 23, 2015
New treatments for autosomal dominant polycystic kidney disease
Ming-Yang Chang1, Albert C M Ong
1Kidney Research Center, Department of Nephrology, Chang Gung Memorial Hospital, Chang Gung University College of Medicine, Taoyuan, Taiwan.
Abstract:
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease and results from mutations in PKD1 or PKD2. Cyst initiation and expansion arise from a combination of abnormal cell proliferation, fluid secretion and extracellular matrix defects and results in kidney enlargement and interstitial fibrosis. Since its first description over 200 years ago, ADPKD has been considered an untreatable condition and its management is limited to blood pressure reduction and symptomatic treatment of disease complications. Results of the recently reported TEMPO 3/4 trial thus represent a paradigm shift in demonstrating for the first time that cystic disease and loss of renal function can be slowed in humans. In this paper, we review the major therapeutic strategies currently being explored in ADPKD including a range of novel approaches in preclinical models. It is anticipated that the clinical management of ADPKD will undergo a revolution in the next decade with the translation of new treatments into routine clinical use.
Insights
Autosomal dominant polycystic kidney disease (ADPKD) management is shifting. New research shows treatments can slow cystic disease and kidney function loss in patients, offering hope for future therapies.
Area of Science:
- Nephrology
- Genetics
- Pharmacology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disorder.
- Caused by mutations in PKD1 or PKD2 genes, ADPKD leads to kidney enlargement and fibrosis.
- Historically considered untreatable, management focused on symptom control.
Purpose of the Study:
- To review current and emerging therapeutic strategies for ADPKD.
- To highlight the significance of recent clinical trial results.
- To discuss the future of ADPKD treatment.
Main Methods:
- Review of published literature on ADPKD treatments.
- Analysis of findings from the TEMPO 3/4 trial.
- Exploration of novel preclinical therapeutic approaches.
Main Results:
- The TEMPO 3/4 trial demonstrated that cystic disease progression and renal function decline can be slowed in ADPKD patients.
- This marks a significant advancement from previous management limitations.
- Various novel therapeutic strategies are under investigation in preclinical models.
Conclusions:
- Recent clinical trials represent a paradigm shift in ADPKD management.
- The future of ADPKD treatment is expected to be revolutionized by new therapies.
- Translation of novel treatments into clinical practice is anticipated within the next decade.
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