Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndrome and other Bone Marrow Failure Disorders

Myelodysplastic syndromes (MDS) are a heterogenous group of rare diseases that affect the production of normal blood cells within the bone marrow, and are characterised by increased amounts of immature blood cells (blasts), leading to issues such as anaemia, increased susceptibility to infection and reduced blood clotting. It is estimated that approximately 30% of patients that have MDS will also go on to develop acute myeloid leukaemia (AML).This research program will seek to find drugs already used clinically, which could potentially be repurposed for the treatment of MDS. Using established MDS reporter cell lines, we will screen a library containing 3,000 FDAapproved compounds, and take the top hits from these screens and validate these findings in cultured patient bone marrow. A selection of drugs which pass validation will then be tested in a pre-clinical model of MDS to determine therapeutic efficacy.