Scientists from Barts Cancer Institute at Queen Mary University of London and the Francis Crick Institute, have uncovered why patients with a rare type of blood cancer suffer from ineffective red blood cell production, and how vitamin B5 could be combined with existing drugs to improve outcomes.
Myelodysplastic syndromes (MDS) are a type of blood cancer characterised by a stem cell disorder where the production of healthy red blood cells goes wrong. There are no curative treatments for these patients, but some medications do help to slow the progression of the disease.
People with this disease often go on to develop acute myeloid leukaemia (AML) and around half of people become resistant to existing treatments for MDS within 18 months to two years of treatment. These patients are heavily reliant on red blood cell transfusions which can be painful and dangerous due to iron overload in the blood. This means it is important to uncover new ways to treat this disease and prevent the progression to leukaemia.
In their study, published in Science Translational Medicine today, the scientists analysed blood samples from 42 people with MDS. They found that the enzyme COASY is critical in regulating red blood cell production in the bone marrow. Partial loss of the enzyme in MDS patients severely disrupts red blood cell production leading to anaemia.
They then tested whether they could boost red blood cell production using treatments including vitamin B5 supplementation. Treatments with vitamin B5 or another metabolite, succinyl-CoA, increased the maturation of red blood cells.
Kevin Rouault-Pierre, Group Leader at Barts Cancer Institute and supervisor of the study, said: “Current treatments for MDS are often associated with reduced quality of life as well as the increased risk of progression to leukaemia. Understanding the biology behind this stem cell disorder is key to unlocking new treatments of the future.”
“Our next steps will be to further investigate how to boost red blood cell production and work towards testing new treatments in clinical trials.”
Syed Mian, postdoctoral research fellow in the Crick’s Haematopoietic Stem Cell Laboratory, who co-authored with Celine Philippe postdoctoral training fellow at Barts Cancer Institute, says: “Given our elderly population is increasing and age is the dominant risk factor for the development of MDS, we will start to see more and more people with this type of blood cancer.
“Anaemia-related symptoms, such as chronic fatigue, are commonly reported in MDS and the current red blood cell transfusions, although essential, come with potential complications, and also require substantial human and financial resources. Therefore, it’s essential that we find alternative ways to regulate long term red blood cell production in these patients. Our results may also potentially help with treatments of other diseases where patients commonly present with anaemia.”
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Notes to Editors
Reference: Mian, SA. et al. (2023). Vitamin B5 and Succinyl-CoA improve ineffective erythropoiesis in SF3B1 mutated myelodysplasia. Science Translational Medicine.
The Francis Crick Institute is a biomedical discovery institute dedicated to understanding the fundamental biology underlying health and disease. Its work is helping to understand why disease develops and to translate discoveries into new ways to prevent, diagnose and treat illnesses such as cancer, heart disease, stroke, infections, and neurodegenerative diseases.
An independent organisation, its founding partners are the Medical Research Council (MRC), Cancer Research UK, Wellcome, UCL (University College London), Imperial College London and King’s College London.
The Crick was formed in 2015, and in 2016 it moved into a brand new state-of-the-art building in central London which brings together 1500 scientists and support staff working collaboratively across disciplines, making it the biggest biomedical research facility under a single roof in Europe. http://crick.ac.uk/
About The Barts Cancer Institute
The BCI is part of Queen Mary University of London’s Faculty of Medicine and Dentistry and one of the top five cancer research centres in the UK. Our overriding objective is to ensure that our research – which aims to identify ways to prevent cancer and develop better diagnostic techniques and treatments – is relevant to and will impact on cancer patients.
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