Related Experiment Video
Updated: May 26, 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
Mechanism-based therapeutics for autosomal dominant polycystic kidney disease: recent progress and future prospects
Ming-Yang Chang1, Albert C M Ong
1Kidney Research Center, Chang Gung Memorial Hospital, Chang Gung University College of Medicine, Taoyuan, Taiwan.
Insights
Autosomal dominant polycystic kidney disease (ADPKD) treatments are advancing, with multiple drug candidates targeting key disease mechanisms like cAMP levels and cell proliferation. Clinical trials are ongoing, offering hope for effective disease-modifying therapies for this common inherited kidney disorder.
Area of Science:
- Nephrology
- Genetics
- Pharmacology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD) is the most prevalent inherited kidney disorder.
- ADPKD affects up to 10% of patients requiring renal replacement therapy.
- Currently, no definitive treatments exist for ADPKD, highlighting the urgent need for disease-modifying drugs.
Purpose of the Study:
- To evaluate recent clinical trial results for ADPKD treatments.
- To review ongoing clinical trials for ADPKD therapies.
- To identify promising drug candidates in the ADPKD pipeline.
Main Methods:
- Review of published clinical trial data for ADPKD.
- Analysis of current and planned clinical trials.
- Assessment of preclinical studies and drug development pipelines.
Main Results:
- Several compounds targeting cAMP levels, cell proliferation, and fluid secretion have shown promise in preclinical models.
- Some compounds have progressed to clinical trials, with more planned.
- A significant number of potential therapeutic candidates are emerging from preclinical research.
Conclusions:
- Multiple therapeutic strategies are being explored for ADPKD, including reducing cAMP, inhibiting cell proliferation, and decreasing fluid secretion.
- While numerous drug candidates exist, clinical translation requires addressing several key challenges.
- Ongoing research and clinical trials are crucial for developing effective disease-modifying treatments for ADPKD.
Abstract:
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease, accounting for up to 10% of patients on renal replacement therapy. There are presently no proven treatments for ADPKD and an effective disease-modifying drug would have significant implications for patients and their families. Since the identification of PKD1 and PKD2, there has been an explosion in knowledge identifying new disease mechanisms and testing new drugs. Currently, the three major treatment strategies are to: (1) reduce cAMP levels; (2) inhibit cell proliferation, and (3) reduce fluid secretion. Several compounds shown to be effective in preclinical models have already undergone clinical trials and more are planned. In addition, a whole raft of other compounds have been developed from preclinical studies. The purpose of this paper is to evaluate the results of recent published trials, review current trials and highlight the most promising compounds in the pipeline. There appears to be no shortage of potential candidates, but several key issues need to be addressed to facilitate clinical translation.
Related Concept Videos
01:29Pharmacogenomics: Identification of New Drug Targets
01:25Chronic Kidney Disease I: Introduction
01:28Chronic Kidney Disease III: Interprofessional Care
01:28Diabetic Nephropathy
01:28Renal Drug Excretion: Tubular Secretion
01:24Site-Targeted Drug Delivery Systems: Polymeric Carriers