The award, presented by the US-based Dent Disease Foundation and carrying a prize of $50,000, will support the development of a platform to study Dent's disease type 1 (DD1). This is a rare kidney condition caused by mutations in the CLCN5 gene, affecting the kidney's proximal tubule and potentially leading to renal failure.
The project aims to overcome the limitations of current models by employing kidney organoids, which are three-dimensional models that better replicate human kidney physiology and the complex cellular interactions within the organ.
The initial phase will involve testing 20 commercially available drugs on mouse models with DD1, with selection aided by artificial intelligence. Subsequently, human organoids derived from patients will be generated to validate the most promising candidates, allowing for the study of the disease in different clinical contexts.
“"The objective is to establish a platform that allows us to study the disease and more accurately evaluate potential treatments. In this way, we hope to reduce the time needed to translate preclinical results into future clinical trials."
This collaborative study, also involving the Pediatric Nephrology Service at the Hospital Universitari Vall d’Hebron, led by Dr. Gema Ariceta, and the University of Edinburgh, has the potential to open new avenues for studying other kidney diseases and improve therapeutic options for patients with limited choices.
“"The platform to be developed in this project can open new avenues for studying other kidney diseases and contribute to identifying treatments for patients who currently have few therapeutic options."
The Dent Disease Foundation is a US-based non-profit organization dedicated to raising awareness, connecting patients, and advancing research on Dent's disease types 1 and 2.
