SUPPORTING INDIVIDUALS, FAMILIES AND RESEARCHERS
IN THE CYSTINOSIS COMMUNITY

£1.6m Grant Awarded for Cystinosis Prodrug Project

A £1.6m grant will help continue the legacy of an “inspirational” Sunderland scientist dedicated to improving the treatment of patients with cystinosis.

Professor Roz Anderson

Professor Roz Anderson

Professor Anderson, who sadly lost her battle with cancer last year, and her team spent 15 years carrying out research to design novel prodrugs to take the effective treatment for cystinosis, cysteamine, directly to the cells that need it, improving its absorption, limiting metabolism and reducing the unpleasant side effects.

Before she died, Roz worked tirelessly to secure a £1.6m grant from the Medical Research Council (MRC) which will take her research work to preclinical trials.

The MRC Developmental Pathway Funding Scheme is now funding a group of leading healthcare experts and collaborators over the next two years to complete the pre-clinical development stage and prepare for clinical trials.

Dr. Adrian Moore, Head of School, Pharmacy and Pharmaceutical Sciences, at the University, said: “The MRC grant is taking Roz and her team’s drug from the University laboratory synthesis stage to what may become the final manufacturing preparation. Roz worked so hard towards the end of her life to ensure this investment was secure and was highly delighted when the money was awarded. We understand it’s one of the highest graded grants that has been awarded through the MRC in the last two years. It’s also the legacy of 15 years of dedicated work by Roz that is offering so much hope to Cystinosis patients and their families.”

Professor Herbie Newell CBE

Professor David (Herbie) Newell CBE

The project will be led by Professor Herbie Newell CBE, who is a world-leading cancer researcher and has a background in bringing drugs from discovery to market. He said: “It is a great privilege to take over as the Principal Investigator on the MRC-funded project to develop a new treatment for patients with the incurable genetic disease cystinosis. Professor Roz Anderson was an outstanding medicinal chemist who I knew for a number of years and admired as a colleague. Her ground-breaking work offers real hope for cystinosis patients, and I look forward to working with colleagues at the University of Sunderland and in pharma to bring this new medicine to patients.”

We are incredibly pleased to be able to share this exciting news with you. Without your fundraising and donations, which enabled Roz and her team to carry out their research over the last 15 years, the prodrug project would not have reached this fantastic milestone. We will keep you updated as to its progress when we can.

Images from https://www.sunderland.ac.uk/more/news/story/fight-continues-against-rare-condition-930.

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