Research Project

Project Title:

Pharmacoprotection in Cystic Nephropathies

Project Type:

Translational research

Disease group(s):

Tubulopathies, Renal malformations, Pediatric CKD & dialysis

Project Summary:

Cystic nephropathies, which affect 20 million people worldwide, are slowly progressive and in most cases lead to terminal renal failure with the need for renal replacement therapy. Although the molecular mechanisms are well understood, the therapeutic options are very limited and no drug has been approved for the treatment of cystic nephropathy. The aim of the project is to find new therapeutic approaches for cystic kidney disease. It is extremely important to conduct experiments to identify pharmacological substances that effectively suppress cyst formation in the kidneys and relieve the associated symptoms of disease such as uremia, polyuria, polydipsia, cyst pain, enuresis and to halt the previously unavoidable development of progressive renal failure.
In a high-content compound screening on a zebrafish cystic kidney model we recently identified several small molecules with cyst inhibitory. Compounds with high efficacy, low to no toxicity and oral availability will be used for future studies in mammalian models. The effects of these candidate compounds will be further investigated by means of interventional studies in two rodent models of cystic kidney disease (PKD/Mhm and PCK rat).

Lead principal investigator(s):

Franz Schaefer, Heidelberg

Project Period:

08/2020   -   08/2025

Sponsors:

Non-profit foundation, Regional funding agency

Project web page:

http://www.neocyst.de

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