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Myelodysplastic syndromes (MDS) are blood cancers that frequently lead to severe anaemia with very limited treatment options. These cancers are difficult to treat and typically incurable. MDS is one of the most common blood cancers in older people, affecting around 7000 patients in the UK.
Cancer Research UK has awarded £1,000,000 to Dr Onima Chowdhury at the MRC Weatherall Institute of Molecular Medicine and Oxford University Hospitals, together with Dr Kevin Rouault-Pierre at Barts Cancer Institute, Queen Mary University of London, to conduct a clinical trial to test whether treating MDS patients with Vitamin B5 can improve their outcomes. The study has been developed in collaboration with MDS UK, the national patient charity and has strong support from patients with MDS.
The most common genetic mutation detected in MDS patients is in the SF3B1 gene. This mutation affects an enzyme called COASY, which is essential for red blood cell production. Pioneering laboratory research has demonstrated that Vitamin B5 can boost red blood cell production, making it a very attractive oral therapeutic for treating MDS patients with SF3B1 mutations.
This CRUK-funded clinical trial aims to significantly improve care for people with MDS by increasing haemoglobin levels, improving quality of life, and reducing the need for red blood cell transfusions.
The study hopes to show that targeting metabolic weaknesses in SF3B1-mutant MDS can improve red blood cell production and change how disease-driving clones behave.
Speaking about the award, Dr. Chowdhury said:
This is true bench-to-bedside precision medicine. I hope this research can improve the treatment landscape for MDS, repurposing a tablet that should be easy for patients to tolerate. We have designed this trial with a strong experimental medicine component. In parallel with clinical endpoints, we will perform longitudinal cellular and molecular profiling to define biological response using cutting-edge techniques.
Our study has the potential to open new avenues for therapeutic development, positioning metabolic interventions as a promising strategy in the treatment of MDS and anaemia.
Dr Chowdhury has been a Consultant Haematologist in the Oxford University Hospitals since 2018 and a senior clinical fellow in the Mead Lab since 2020.
Learn more about this and other Clinical Research Committee awards here: https://www.cancerresearchuk.org/for-researchers/apply-for-and-manage-your-funding/recently-funded-research/clinical-research-committee-awards