[Autosomal dominant polycystic kidney disease (ADPKD): rapamycin as a new treatment option]

Area of Science:

• Nephrology
• Genetics
• Pharmacology

Background:

• Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by massive kidney enlargement due to cyst formation.
• Cyst growth begins in utero, leading to renal parenchyma destruction and eventual kidney failure, necessitating kidney replacement therapy.
• Currently, no effective therapies exist to halt the progression of ADPKD.

Purpose of the Study:

• To provide an overview of Autosomal dominant polycystic kidney disease (ADPKD).
• To discuss the role of the mammalian target of rapamycin (mTOR) pathway in ADPKD.
• To highlight the potential of mTOR inhibitors, specifically sirolimus, as a therapeutic strategy for ADPKD.

Main Methods:

• Review of existing literature on ADPKD.
• Analysis of animal studies investigating the effects of mTOR inhibition on cystogenesis.
• Examination of current clinical trial data for sirolimus in ADPKD patients.

Main Results:

• Animal models demonstrate that inhibiting mTOR slows down ADPKD progression.
• Sirolimus, an mTOR inhibitor, is currently under investigation in clinical trials for ADPKD treatment.
• Pharmacological inhibition of mTOR represents a potential disease-modifying strategy.

Conclusions:

• ADPKD is a progressive disease with significant morbidity and no current cure.
• Targeting the mTOR pathway with inhibitors like sirolimus shows potential for slowing ADPKD progression.
• Further clinical research is warranted to establish the efficacy and safety of mTOR inhibitors in managing ADPKD.