Using in vivo small molecule screens to develop novel therapeutics of MDS5q

Dr Elspeth Payne, Cancer Institute, University of Central London and John Goldman Fellow 2015

Dr Beth Payne

Myelodysplastic syndrome (MDS) is a blood cancer that usually begins with symptoms of anaemia (low numbers of red blood cells). This can progress over time to a more severe illness and eventually leukaemia. Most patients with MDS die from leukaemia of infections as a result of the disease. The only curative treatment for MDS is a stem cell transplant. However, bone marrow transplants require a suitable donor and are often associated with severe side effects, making this treatment unsuitable for many patients. 

As most patients are already in their 60’s or older, there is significant toxicity from the transplant procedure and a lower likelihood that their siblings will be fit enough to donate, if matched. Furthermore, 5 years later, only around a third of patients who receive a transplant will be alive and free from disease. Thus, new less toxic therapies are desperately needed to treat MDS and delay or prevent progression to leukaemia.

This funding award has come at a particularly critical time for me as I make the transition to fully independent investigator, while balancing my MDS research and clinical practice as Consultant Haematologist at UCLH – and looking after my young family. Obtaining funding is a particular challenge for young researchers as they are viewed as more 'risky' than established researchers. Investing in the next generation of leukaemia researchers is absolutely critical to permit continuing high quality research into curing leukaemia and related disorders.

Obtaining funding is a particular challenge for young researchers as they are viewed as more 'risky' than established researchers. Investing in the next generation of leukaemia researchers is absolutely critical to permit continuing high quality research into curing leukaemia and related disorders. Leuka's funding will allow me to develop new models of MDS that can be used to test new drugs. We already have some promising results and hope that our findings can reall have an impact on patients' lives.

Dr Beth Payne