Vitamin B5 could improve outcomes in patients with a rare type of blood cancer: Study

UK: Vitamin B5, in combination with existing drugs, could improve outcomes in patients with Myelodysplastic syndromes (MDS), a rare type of blood cancer characterized by ineffective red blood cell production. 

In their study, published in Science Translational Medicine today, the scientists from Barts Cancer Institute at the Queen Mary University of London and the Francis Crick Institute analyzed 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.

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 help slow the disease's progression.

People with this disease often develop acute myeloid leukaemia (AML), and around half of the people become resistant to existing treatments for MDS within 18 months to two years of treatment. These patients rely heavily 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 of leukaemia.

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 future treatments.”

“Our next steps will be to investigate further 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 treat other diseases where patients commonly present with anaemia.”

Reference:

Syed A. Mian, Céline Philippe, Eleni Maniati, Tiffany Bergot, Marion Piganeau, Travis Nemkov, Doriana Di Bella, Valle Morales, Andrew J. Finch,Angelo D’Alessandro, Katiuscia Bianchi, Jun Wang, Paolo Gallipoli, Shahram Kordasti, Anne Sophie Kubasch, Michael Cross, Uwe Platzbecker, Daniel H. Wiseman , Dominique Bonnet SCIENCE TRANSLATIONAL MEDICINE 1 Mar 2023 DOI: 10.1126/scitranslmed.abn5135