Analysing MDS stem cells to develop new treatments

Lead researcher - Professor Jacqueline Boultwood, University of Oxford
Single-cell analysis of haematopoietic stem cells in SRSF2 mutant MDS: identification of new therapeutic targets/treatments
Amount awarded: £299,274
Award start date: 01 Sep 2019
Award duration: 2 years, 9 months (33 months)

Myelodysplastic syndrome (MDS) is a common blood cancer, but we have few effective treatments. One way to improve how we treat MDS is to create targeted treatments that attack the specific genetic errors that occur in each person’s MDS.

Professor Boultwood and her team are studying MDS stem cells, which are a small population of cells that can give rise to a steady stream of new MDS cells. They are focusing on stem cells that come from the bone marrow of people with MDS who have a SRSF2 gene change – this type of MDS has a very poor outcome.

By looking at single stem cells from individual people with MDS in unprecedented detail, Professor Boultwood and her team will detect malfunctioning genes and biological pathways in MDS that could be targeted with drugs. Once they’ve identified these errors, they will test drugs that could kill off the MDS cells but leave healthy blood cells alone, with the potential to eradicate the disease. These drug targets could also be applicable to other blood cancers.

If successful, they will test the newly identified drugs in clinical trials. This could lead to better treatments for people with MDS and other blood cancers.