Currently, only one treatment exists for children affected by cystinosis – a medication called cysteamine. Unfortunately, cysteamine comes with a challenging treatment schedule. Children are often required to take dozens of pills a day at 6-hourly intervals – this includes being woken at night. In addition, they are required to use eye drops every 1–2 hours. “It is vital that cystinosis patients take their medicine, as it is effective in delaying kidney failure and extends life expectancy,” says Dr Hollywood, “however, this drug is not very pleasant to take. It has a bad taste and a horrible odour, and causes unwanted side-effects such as nausea and stomach problems.”
Dr Hollywood leads one of nine research teams chosen to receive a Project Grant from Cure Kids in 2021. For 50 years, Cure Kids has been committed to enabling research that transforms children’s health in New Zealand – and recognised the work of Dr Hollywood and her team as having the potential to change the lives of children with cystinosis. “In this research, we will be testing a new drug called CF10,” says Dr Hollywood, “to see if it is better than cysteamine at reducing cystine and preserving kidney function.
You can read more about CF10 here https://www.curekids.org.nz/our-research/projects/project/a-better-treatment-option-for-kids-with-cystinosis