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Updated: Jun 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
[Modern treatment of autosomal dominant polycystic kidney disease]
Wojciech Wołyniec1, Magdalena Maria Jankowska, Bolesław Rutkowski
1Akademia Medyczna w Gdańsku, Klinika i Katedra Nefrologii, Transplantologii i Chorób Wewnetrznych. wwolyniec@wp.pl
Insights
Autosomal dominant polycystic kidney disease (ADPKD) is a leading cause of kidney failure. Current treatments focus on managing symptoms, but new drugs targeting ADPKD
Area of Science:
- Nephrology and Genetics
- Molecular Biology
Context:
- Autosomal dominant polycystic kidney disease (ADPKD) is a common genetic disorder leading to end-stage renal failure (ESRF).
- Current therapeutic strategies for ADPKD primarily involve nephroprotection and management of associated complications.
- Approximately 50% of ADPKD patients require renal replacement therapy by their sixth decade.
Purpose:
- To review recent advancements in understanding ADPKD pathophysiology.
- To explore novel pharmacological agents being investigated for slowing ADPKD progression.
- To highlight the current landscape of ADPKD treatment and future therapeutic directions.
Summary:
- Recent research has elucidated the roles of polycystins, intracellular calcium, and cAMP in ADPKD pathogenesis.
- Investigational drugs include somatostatin receptor agonists, vasopressin V2 receptor antagonists, rapamycin, and tyrosine kinase inhibitors.
- While promising preclinical data exists, the efficacy of these novel agents in humans requires validation through ongoing clinical trials.
Impact:
- Advances in understanding ADPKD mechanisms are paving the way for targeted therapies.
- New drug candidates offer potential to slow disease progression and delay the need for renal replacement therapy.
- Clinical trial outcomes are eagerly anticipated by nephrologists and patients for improved ADPKD management.
Abstract:
Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common causes of end stage renal failure (ESRF). Nevertheless, until now the standard therapy of ADPKD consists merely of nephroprotection and treatment of complications. In the sixth decade of life in about 50% of patients renal replacement therapy is initiated. Surgery procedures such as nephrectomy and renal contraction therapy are performed in some patients. In 21st century numerous studies concerning patophysiology of the disease (role of polycystins, intracellular calcium, cAMP) were published and gave an impulse for searching new drugs that could slower progression of ADPKD. Somatostatin receptor agonists, vasopressin V2 receptor antagonists, rapamycin, and tyrosine kinase inhibitors are among medications that are thought to be effective. Although it is unclear if any of these drugs will be really effective there are strong theoretical backgrounds and promising results of experimental studies on animals. Nephrologists and their patients are waiting for the results of clinical trials that are performed now.
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