Cystinosis Foundation UK is proud to announce a momentous step forward in the fight against cystinosis: researchers at the University of Sunderland have received £3.9 million from the Medical Research Council (MRC) to take a promising new treatment, CF10, into clinical trials.
This milestone is the result of years of tireless work by the late Professor Roz Anderson, an inspirational medicinal chemist whose vision and dedication laid the foundation for CF10—a novel prodrug designed to improve the lives of those living with cystinosis. Roz’s pioneering research, supported early on by Cystinosis Foundation UK’s community fundraising efforts, aimed to reduce the harsh side effects of the current treatment, cysteamine, and offer a more tolerable, effective alternative.
Cystinosis is a rare, life-threatening genetic condition that causes cystine to build up in every cell of the body, damaging organs including the kidneys, muscles, and eyes. Without treatment, children would face kidney failure and multi-organ complications before the age of 10. The only available therapy, cysteamine, (commonly known as Cystagon) requires large doses every six hours for life and causes severe side effects such as nausea, vomiting, ulcers, and strong body odour.
CF10 offers a new approach—delivering treatment directly to the cells that need it, with fewer doses and the anticipation of vastly reduced side effects. The drug has the potential to dramatically improve quality of life for patients and families.
From Discovery to Trial: A Community-Driven Journey
Cystinosis Foundation UK’s early support helped secure £1.6 million from the MRC in 2018 to complete CF10’s preclinical development. Now, with an additional £3.9 million awarded, the drug will enter clinical trials led by Professor Herbie Newell and a team at the University of Sunderland.
The trial will be conducted in partnership with:
- High Force Research (Durham) – CF10 manufacturing
- Newcastle Specials (Royal Victoria Infirmary) – capsule production
- University Hospitals Birmingham NHS Foundation Trust – clinical trial management
- Birmingham Women’s and Children’s NHS Trust and CEMAS (Wokingham) – sample analysis
Dr. Graham Lipkin, a kidney specialist and expert in cystinosis care, will oversee the trial, which will identify the optimal dose of CF10 for patients.
Reflecting on this Achievement
Our Chair, Will Newman, says: “For those living with cystinosis, the daily reality is brutal—relentless medication schedules, sleep deprivation, and harsh side effects. CF10 could change everything. This isn’t just a scientific milestone—it’s hope for a brighter future. We’re deeply grateful to Roz Anderson, Herbie Newell, and the team at Sunderland, and to our community whose fundraising efforts have been nothing short of heroic. If CF10 succeeds, it will be life-changing—not just in the UK, but for cystinosis families around the world. We’d like to thank every single person who has supported Cystinosis Foundation UK in any way over the years – this is all down to your efforts.”
Professor Herbie Newell added: “Roz discovered CF10 with support from the Cystinosis Foundation UK. This trial is a tribute to her legacy and to the families who inspired her work. CF10 has the potential to transform treatment and offer real hope.”
Dr. Adrian Moore, Head of Pharmacy at the University of Sunderland, reflected: “Roz embodied the belief that research can change lives. It’s been an honour to help bring her vision to life for the cystinosis community.”
Professor Laura Stroud, Pro Vice-Chancellor at the University, said: “This award is a testament to the determination of scientists, clinicians, and the Foundation. Most importantly, it brings hope to families affected by this rare disease.”