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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
Published on: June 23, 2015
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Therapeutic advances in ADPKD: the future awaits
Ivana Capuano1, Pasquale Buonanno2, Eleonora Riccio3
1Chair of Nephrology "Federico II", Department of Public Health, University of Naples, Via Sergio Pansini, 5, 80131, Naples, Italy. ivanacapuano@libero.it.
Journal of Nephrology
|May 19, 2021
Summary
Autosomal dominant polycystic kidney disease (ADPKD) treatments are advancing beyond current therapies like tolvaptan. New drugs targeting molecular pathways and novel strategies offer hope for changing the disease
Area of Science:
- Nephrology
- Genetics
- Pharmacology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder and a leading cause of end-stage renal disease.
- Mutations in PKD1 and PKD2 genes, encoding polycystins, disrupt cellular functions.
- Current research focuses on understanding ADPKD pathophysiology for improved therapeutic strategies.
Purpose of the Study:
- To review existing and emerging treatments for ADPKD.
- To summarize drugs in clinical practice and preclinical development.
- To highlight novel therapeutic targets and strategies for ADPKD management.
Main Methods:
- Literature review of clinical practice and ongoing research.
- Analysis of randomized controlled trials (RCTs) and preclinical studies.
- Synthesis of information on drugs targeting various molecular pathways.
Main Results:
- Tolvaptan and octreotide-LAR represent new therapeutic options.
- Ongoing RCTs explore drugs targeting cAMP, EGF receptor, AMPK, and sphingolipids.
- Preclinical studies investigate intracellular calcium, cell cycle, inflammation, and cell therapy.
Conclusions:
- The therapeutic landscape for ADPKD is rapidly evolving.
- Multiple promising drug candidates and strategies are under investigation.
- Future management may involve tailored therapies to alter ADPKD's natural history.
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