Cystic kidney disease – finding new drug treatments
|Project Leader||Professor C A Johnson, BA (Hons) MA (Oxon) PhD|
|Location||Section of Ophthalmology and Neurosciences, Leeds Institute of Biomedical and Clinical Sciences, St James' University Hospital|
|Grant awarded||21 November 2017|
|Provisional start date||1 February 2018|
|Provisional end date||30 January 2021|
We do not provide medical advice. If you would like more information about a condition or would like to talk to someone about your health, contact NHS Choices or speak to your GP. Please see our useful links page for some links to health information, organisations we are working with and other useful organisations. We hope you will find these useful. We are not responsible for the content of any of these sites.
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.