Ten million euro grant for gene therapy in rare disease cystinosis  Radboudumc closely involved in Dream Fund Award to the Dutch Kidney Foundation and Kidnie

Every year, hundreds of children in the Netherlands are diagnosed with kidney disease. Globally, 2 million children suffer from kidney disease. Currently, there is no cure. They undergo lifelong treatments and have a much shorter life expectancy than healthy children. Thanks to a contribution of €10 million from the Dream Fund of the National Postcode Lottery, Kidnie, a foundation initiated by the Dutch Kidney Foundation, can now collaborate with, among others, Radboud university medical center to develop a gene therapy for children with cystinosis. 

Cystinosis is a rare hereditary condition in which cystine accumulates, damaging body cells. The kidneys and eyes are the first to be affected. The average life expectancy for a child with cystinosis is a maximum of 50 years. Radboudumc is the national expertise center for cystinosis. With this grant from the National Postcode Lottery’s Dream Fund, a gene therapy is being researched and developed under the leadership of pediatric nephrologist Michiel Schreuder, Professor of Pediatric Nephrology at the Radboudumc Amalia Children's Hospital.

Progress in research

Pediatric nephrologists and biomedical researchers have combined forces to make a cure for kidney diseases in children possible through the Kidnie initiative, founded in collaboration with the Dutch Kidney Foundation. They are focused on rapid progress in gene therapy. Where science was not advanced enough until recently, the techniques are now at a turning point: for the first time, it is possible to target kidney diseases at the source. According to Schreuder, this is essential: 'As long as we don’t break the current system, children will continue to suffer. This project is a blueprint for more effective and patient-centered medical science and is being closely followed worldwide by colleagues.' 

What is gene therapy?

Gene therapy addresses diseases at the source by correcting genetic errors rather than just treating the symptoms. Diseases that were previously incurable can now be permanently cured thanks to new techniques and improved delivery systems (methods and technologies used to introduce genetic material into a patient’s cells). In addition, treatments can be tailored precisely to an individual’s unique DNA. In short, gene therapy opens the door to a better, disease-free future.

From 0% to 25% chance of a cure

'Until now, we have had to tell parents that their child will never get better,' says Schreuder, co-founder of Kidnie. 'Thanks to the Dream Fund, what seemed impossible just ten years ago is now becoming a reality. With gene therapy, we expect to make a cure possible for 25% of children with kidney disease in the future. This means a chance for more than 4,000 children and their families to live a future free of constant hospital visits and uncertainty. For children, this can make the difference between surviving and truly living.'

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