Funded by the 2013 V Foundation Wine Celebration
Myelodysplastic syndrome (MDS) is a group of clonal blood cell disorders characterized by abnormal-looking cells (cytologic dysplasia) and low cell count (refractory cytopenias) as a result of ineffective blood production (hematopoiesis). About 30-40 percent of MDS cases progress to acute myeloid leukemia (AML), a type of blood cancer, with poor prognosis and short survival time. We have discovered that a novel embryonic stem cell factor also a leukemic stem cell factor SALL4 is involved in the development of MDS and AML. We propose to study the SALL4 pathway in MDS and AML using murine models and test small molecule drugs that can block SALL4 function in MDS/AML. This study will provide new and critical insights and novel pathways in mediating MDS and its progression to AML which will enable us to develop innovative drugs in treating these patients in the near future.
