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[Autosomal dominant polycystic kidney disease: is the treatment for tomorrow?]

Emilie Cornec-Le Gall1, Yannick Le Meur2

  • 1Service de néphrologie, hémodialyse et transplantation rénale, hôpital la Cavale-Blanche, 15, boulevard Tanguy-Prigent, 29609 Brest cedex, France; Université de Bretagne occidentale, université Européenne de Bretagne, 3, rue des Archives, 29238 Brest, France; Institut national de la sante et de la recherche medicale (INSERM), U1078, 46, rue Félix-le-Dantec, 29218 Brest cedex 2, France.

Nephrologie & Therapeutique
|August 4, 2014
PubMed

Insights

Autosomal dominant polycystic kidney disease (ADPKD), a common inherited disorder, is progressing towards targeted therapies. Current research focuses on molecules like tolvaptan and somatostatin analogues to slow disease progression.

Area of Science:

  • Nephrology
  • Genetics
  • Pharmacology

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disorder.
  • It accounts for a significant percentage of end-stage renal disease (ESRD) cases.
  • ADPKD has historically lacked effective targeted treatments.

Purpose of the Study:

  • To review signaling pathways implicated in ADPKD.
  • To summarize current and ongoing clinical trials for ADPKD therapies.
  • To discuss promising therapeutic molecules evaluated in preclinical models.

Main Methods:

  • Literature review of signaling pathways in ADPKD.
  • Analysis of published and ongoing clinical trial data.
  • Evaluation of preclinical studies on therapeutic agents in murine models.

Main Results:

  • mTOR inhibitors showed disappointing results in clinical trials.
  • Tolvaptan and somatostatin analogues demonstrated a modest effect on slowing total kidney volume (TKV) progression.
  • Both tolvaptan and somatostatin analogues function by reducing intracellular cyclic AMP (cAMP).

Conclusions:

  • Targeted therapies for ADPKD are emerging.
  • Tolvaptan and somatostatin analogues represent promising therapeutic avenues.
  • Further research and clinical trials are crucial for advancing ADPKD treatment.
Keywords:
Analogues de la somatostatineAutosomal dominant polycystic kidney diseaseChronic kidney diseaseDrug therapyEverolimusGenetic diseasesGénétiqueInhibiteur de la voie des mTORInhibiteurs des récepteurs V2 de la vasopressineInsuffisance rénale chroniquePolykystose rénale autosomique dominanteReceptorsSirolimusSomatostatin analogsThérapeutiqueVasopressin/antagonists and inhibitors

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