Setting the scene for better cystinosis treatment

Dr Jennifer Hollywood has discovered that the cancer and organ-rejection drug everolimus has the potential to treat cystine-ravaged cells, especially when combined with the drug cysteamine. It’s hoped that this preclinical work, funded by Cure Kids, will lead to improved outcomes for children battling cystinosis.

Dr Jennifer Hollywood

University of Auckland

Cystinosis is an abnormal build-up of the amino acid cysteine, resulting in tissue damage throughout organs of the body. It is caused by a gene mutation. Children with cystinosis are at risk of bowed bones and rapid kidney dysfunction. Current treatments only delay kidney damage and come with many side effects.

Repurposing an organ-rejection drug

Everolimus/cysteamine combination therapy could lead to improved outcomes for children facing kidney failure caused by cystinosis.

Dr Jennifer Hollywood has discovered that treatment with the cancer and organ-rejection drug everolimus has the potential to treat cystine-ravaged cells, especially when combined with cysteamine, the currently used drug.

To properly investigate everolimus/cysteamine combination therapy, Dr Hollywood has developed the first preclinical animal model of cystinosis in rats which faithfully replicates the disease in humans. It’s hoped that this preclinical work will lead to improved outcomes for children battling cystinosis.

Cure Kids funded this important research

We are assisting Dr Hollywood’s work by providing funding that pays for her time, laboratory and consumables.