Cystic kidney disease – finding new drug treatments | Children's Charity

Cystic kidney disease – finding new drug treatments

Project LeaderProfessor C A Johnson, BA (Hons) MA (Oxon) PhD
Project team
  • Dr J Bond, BSc PhD
  • Dr R Foster, BSc PhD
  • Professor J A Sayer, PhD FRCP
LocationSection of Ophthalmology and Neurosciences, Leeds Institute of Biomedical and Clinical Sciences, St James' University Hospital
Other locations
  • School of Chemistry, University of Leeds, Institute of Genetic Medicine, University of Newcastle
Duration3 years
Grant awarded21 November 2017
Provisional start date1 February 2018
Provisional end date30 January 2021
Grant amount£199,786.00
Grant codeGN2628

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Cystic kidney disease is an inherited disease which occurs when there is a fault in a gene controlling the development of cilia. Cilia are tiny hair-like structures that protrude from the surface of most animal cells like antennae. Like antennae, they receive signals from other cells and their surroundings, and help the cell behave appropriately. When cilia do not form properly, this leads to a range of developmental disorders called ciliopathies. Ciliopathies almost always have kidney cysts as a feature, and are a major cause of chronic kidney disease in both children and adults. At any one time, around 5,000 patients in the UK are receiving treatment, either dialysis or a kidney transplant, as a result of an inherited kidney disease. However, there are few effective treatments that can prevent or slow the progression of cystic kidney disease.

The research project
The lack of drugs to prevent or treat cystic kidney disease means that existing medicines, approved to treat other conditions, are being evaluated as potential treatments. These researchers have identified a number of approved medicines and other small molecules that improve cilia formation and function. In this project, they will test if these compounds are effective in cell-based models of ciliopathies and kidney cysts, including kidney cells taken from patients. For the most effective compounds, they will then run safety and efficacy tests in more complex animal models to pave the way for clinical trials. The advantage of developing established medicines to treat new conditions is that their pharmacological and safety profiles are already well understood, and therefore the research could quickly move forward into a first clinical trial within 5 years. This study may identify several potential much-needed new treatments for cystic kidney disease that could then be entered into in a more extensive drug development programme.

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