News & Insights

CEMAS Supports Rare Disease Treatment

CEMAS is proud to be supporting the upcoming clinical trial of CF10, a novel treatment for cystinosis, a rare and life-threatening genetic disorder.  The trial is led by the University of Sunderland and has been made possible by a £3.9 million award from the Medical Research Council (MRC) enabling CF10 to progress into its first clinical phase.

About Cystinosis:

Cystinosis is caused by a build-up of cystine, an amino acid, in the body, which damages the kidneys, muscles, eyes and other organs. Without treatment, children suffering from this disease can face kidney and multi-organ failure, often before the age of 10. Current therapies are lifelong and can produce significant negative side effects, making treatment adherence challenging.

The Research: 

CF10, developed by the late Professor Roz Anderson and her team, is designed to deliver treatment directly to affected cells, reducing side effects and improving the patient’s quality of life.  The pre-clinical development stage began in 2018 and the latest MRC funding allows Professor Herbie Newell to advance CF10 into the next phase of development.

CEMAS’ Involvement: 

CEMAS has supported the development of CF10 through the establishment of analytical methods to aid future studies and continues to contribute as the project moves into its first clinical trials. Working under the leadership of the University of Sunderland and alongside University Hospitals Birmingham NHS Foundation Trust, the University of Birmingham, High Force Research and Newcastle Specials, CEMAS will provide analytical expertise to help ensure the trial generates robust, reliable data as it progresses through its clinical stages.

The upcoming trial marks a major milestone for CF10; and a vital step forward for patients living with cystinosis. It also reflects the collaborative effort required to bring complex, high-impact research from the laboratory toward clinical application.

 

For more information about CEMAS’ role in clinical trials, contact us here or e-mail enquiries@cemas.co.uk

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