Researchers receive £3.9 million to take new cystinosis drug into clinical trials

Published on 22, October, 2025

Researchers have been awarded nearly £4 million to take a drug, discovered by an “inspirational” University of Sunderland scientist dedicated to improving the treatment of patients with a rare, life-threatening genetic disease into clinical trials.

Professor Roz Anderson, who lost her life to cancer in the summer of 2018, had worked tirelessly to secure a grant from the Medical Research Council (MRC) which would take her research work – designing drugs to treat and improve the quality of life for cystinosis patients – into preclinical trials.

There is currently no cure for cystinosis, which occurs when the body is unable to correctly process cystine (an amino acid). Normally, a person can process cystine, but for people with the condition, cystine builds up and attacks every part of the body, particularly the kidneys, muscles and eyes. Without treatment, patients can die from kidney and multiorgan failure before the age of 10.

The current and only treatment for the disease, cysteamine, produces unpleasant side effects such as nausea, vomiting, bad breath, a poor body odour, ulcers and gut damage. A large dose of the drug is required four times a day, for life.

Professor Anderson and her team designed a novel prodrug (CF10) that takes the effective treatment directly to the cells that need it, limiting breakdown and avoiding the unpleasant side effects.

In 2018, the MRC Developmental Pathway Funding Scheme awarded £1.6 million to the University of Sunderland and collaborators to complete the pre-clinical development stage started by Professor Anderson.

Now, the team has received a further £3.9 million from the MRC to take the drug into clinical trials.

Professor Herbie Newell, who has a background in bringing drugs from discovery to market whilst at Newcastle University, is leading the project.

He said: “Professor Roz Anderson discovered CF10 with generous funding from Cystinosis Foundation UK, a patient and carer charity – and the latest funding we have received from the MRC will allow us to give CF10 to patients for the first time.

“Roz was an inspirational and outstanding medicinal chemist. The trial this funding has made possible will both offer hope for cystinosis patients and create a lasting legacy for Roz. CF10 has the potential to transform the treatment and lives of patients living with cystinosis.”

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Professor Newell and the University of Sunderland team will be working with High Force Research (Durham), who will make the CF10, Newcastle Specials based at the Royal Victoria Infirmary, part of Newcastle upon Tyne Hospitals NHS Foundation Trust, who will produce the CF10 capsules, University Hospitals Birmingham NHS Foundation Trust (UHB) and the University of Birmingham, who will treat the patients and manage the first phase of the clinical trials and Birmingham Women’s and Children’s NHS Foundation Trust and CEMAS (Wokingham), who will analyse patient samples.  

Patient volunteers will be given increasing doses of CF10 to identify an active dose.

Queen Elizabeth Hospital Birmingham (QEHB), part of UHB, will be the only site in the UK running this trial, with all patients seen at the hospital’s Centre for Rare Diseases - one of the largest centres of its kind in the country.

The trial will be led by Dr Graham Lipkin, a kidney specialist at UHB and an expert in treating patients with cystinosis.

Dr Lipkin said: “I’m incredibly proud to be leading this trial here at QEHB, which could make a real difference for patients with this rare condition. Cystinosis is a dreadful inherited disorder that usually presents in early infancy, initially damaging the kidneys and eyes and often requiring dialysis or a kidney transplant. Over time, it can also cause diabetes, hormone deficiencies, and progressive muscle weakness, resulting in loss of strength, swallowing difficulties and chest problems.

“This partnership between UHB, the University of Birmingham, and the University of Sunderland brings together world-class expertise with the potential to set a new standard of care, delivering a treatment that could be truly life-changing for patients.”

Will Newman, Chair of Cystinosis Foundation UK, said: "For those living with cystinosis, the daily reality is brutal – relentless medication schedules, sleep deprivation, and harsh side effects from cysteamine. CF10 could change everything.

“This new treatment promises fewer doses and virtually no side effects. It’s not just a scientific milestone – it’s hope for a brighter future. We are hugely grateful to Professor Roz Anderson, Professor Herbie Newell and the fantastic team at Sunderland for advancing CF10 into clinical trials, and to our community whose fundraising efforts have been nothing short of heroic, helping a tiny charity punch far above its weight in drug development. If CF10 succeeds, it will be life changing, not just for those in the UK, but for the cystinosis community worldwide."

Dr Adrian Moore, Head of the School of Pharmacy and Pharmaceutical Sciences at the University of Sunderland, said: “Roz embodied the University of Sunderland’s vision that research can change lives and inspire generations to come.

“It has been both a pleasure and an honour for colleagues and external partners to take forward and bring Roz’s work to fruition for the benefit of the cystinosis community. This most recent award from the MRC represents the next step in this exciting and much needed journey.”

Pro Vice-Chancellor of the Faculty of Health Sciences and Wellbeing at the University, Professor Laura Stroud, added: “The award is testament to the determination of a number of dedicated scientists and clinicians, and is a fitting legacy for the late Professor Roz Anderson, on whose inspirational work this builds.

“Most importantly, the trial brings hope to families and children with this rare disease – we are proud to support this ground-breaking work.”

Professor Roz Anderson

A Professor of Pharmaceutical Chemistry, Roz Anderson joined the University in 1987. In her 31-year career as a researcher at the University of Sunderland she worked on the treatment of Alzheimer's disease, diagnosis of bacterial infections, psoriasis and cancer, but her particular passion was the battle against the rare genetic disorder Cystinosis. Typically, Professor Anderson’s work was not confined purely to the lab, and she made a point of meeting and discussing her work with the children affected by this illness and their families, which she maintained was an essential aspect of her work.

Professor Anderson was awarded a WIN Award in the STEM Category at the Network North East Woman Entrepreneur of the Year Awards in 2016. Her WIN Award reflected in part to her strong belief in teamwork and making a difference in society by inspiring future generations. Despite her intense research work, Professor Anderson insisted on working alongside undergraduate and PhD students and post-doctoral scientists, to ensure that young people are advocates for the advancement of medicine and society.

Cystinosis

Cystinosis occurs when the mechanism which stores and removes excess cystine, an amino acid, breaks down. Cystine then accumulates within body cells preventing cells from functioning correctly.

This initially leads to kidney problems and progresses to other parts of the body, including muscles and eyes. Impaired growth is yet another symptom of the condition. In the past, it was rare for Cystinosis patients to survive into adulthood.

However, with better understanding and earlier diagnosis, and the introduction of cysteamine, it is possible for sufferers to lead a longer, fuller life.

Cystinosis is a rare inherited disease, occurring in about one in 200,000 births within developed countries, where both parents carry a faulty gene but don't have the disease, because they also each have a normal gene to compensate. If they both pass the recessive Cystinosis gene to their child, the disease will develop.

Professor David (Herbie) Newell CBE

Professor Newell was the founding Scientific Director of the Northern Institute for Cancer Research at Newcastle University and is Past-Chairman of the British Association for Cancer Research and of the Laboratory Research Division of the European Organisation for Research and Treatment of Cancer. He was Director of Translational Research at Cancer Research UK from 2006 to 2009, and interim Executive Director of Clinical and Translational Research in 2007/8.

He is also an Emeritus Professor of Cancer Therapeutics at Newcastle University. He was awarded a CBE in the 2019 New Year’s Honour List.

Professor Newell was previously involved in the development of the registered cytotoxic anticancer agents carboplatin (Paraplatin®) and ralitrexed (Tomudex®), and until his retirement in 2016 was working on the discovery and development of molecularly-targeted anticancer drugs and associated predictive and pharmacological biomarkers, such as rucaparib (Rubraca®) and erdafitinib (Balversa®).

In addition, he has served on numerous international translational cancer research review panels, most recently in France, Germany, the Netherlands and Malaysia. In 2011, Professor Newell was elected to the UK Academy of Medical Sciences.

Professor Newell was appointed by the University of Sunderland in 2018 to continue the work of Professor Roz Anderson.

Cystinosis Foundation UK

Cystinosis Foundation UK was founded in 1998 by the late Jonathan Terry MBE, who had cystinosis. 

Charity status was achieved in March 1999. Since that time significant funds have been raised for research and family support activity relating to cystinosis, helping many with advice and support, and raising awareness of cystinosis among medical and research communities.

Prior to 2016, the charity provided £1 million to Professor Roz Anderson which resulted in the discovery of CF10. The charity has worked closely with the University of Sunderland in the preclinical development of CF10 and will be central to reviewing proposals for and recruiting patients to the clinical trial of CF10.

Dr Graham Lipkin

Dr Lipkin is a Consultant Kidney Specialist at University Hospitals Birmingham NHS Foundation Trust, with a lifelong professional interest in caring for patients with rare kidney diseases. He played a key role in establishing the Birmingham Centre for Rare Diseases at Queen Elizabeth Hospital Birmingham, where he helped develop a holistic model of care for hundreds of rare conditions, including cystinosis.

Dr Lipkin was also involved in NHS England’s approval of three nationally designated centres - one of which is University Hospitals Birmingham - specialising in cystinosis, improving outcomes for patients and their carers living with this complex, multi-system condition.

Dr Lipkin is a Past President of the UK Kidney Association, which awarded him a Lifetime Achievement Award in 2025, and is currently the Chair of Kidney Care, the UK’s leading kidney patient support charity.

Birmingham Clinical Trials Unit, University of Birmingham

The University of Birmingham’s Birmingham Clinical Trials Unit (BCTU) was one of the first clinical trials units to be awarded UK Clinical Research Collaboration accreditation. After 25 years of groundbreaking work, the BCTU is a renowned leader in its field, providing clinicians with scientific, technical and computing expertise to support every stage of their innovative research. BCTU is a large, leading clinical trials unit with a specific focus on late-phase trials. BCTU’s work is changing clinical practice and plays a major part in the medical treatment and health of people worldwide.

Newcastle Specials

The Newcastle Specials pharmacy production unit, part of Newcastle Hospitals, is a dedicated facility for the production and quality control of medicines. Find out more here.