Our Research

In the last decade, researchers have identified key genetic events that characterize MDS. These cellular and molecular findings can help with disease prognosis, however, how the disease arises and progresses remains unknown and therapies lag behind research. Modern standard of care therapies either aim to boost the defective blood cell production in the bone marrow, or to treat the underlying MDS cell of origin using drugs that inhibit DNA methylation, suppressing rapid cell growth associated with MDS. Unfortunately, most MDS patients still succumb from the defect in blood production associated with the bone marrow failure as a consequence of MDS. In addition, in more than 30% percent of MDS patients, the disease evolves to an aggressive acute myeloid leukemia (AML). These patients often are left with a poor prognosis of MDS and transformed AML that is resistant to chemotherapy and in majority older patients, unable to go through allogeneic stem cell transplant. Targeted therapies from novel and exciting discoveries that are intrinsic to the MDS cell have also very limited potency when tried in MDS patients due to how the disease adapts to evade therapies mainly by generating MDS cells with new mutations.