Funded by Lloyd Family Clinical Scholar Fund
Myeloid malignancies (myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]) are very aggressive blood cancers that have limited treatment options. We have learned that these cancers have “stem cell” populations, which allow the disease to propagate, and are the source of relapse when it occurs. One cannot hope to cure these diseases without eradicating the stem cells, but that has historically been very difficult because so little was known about this cell population. We have recently made some breakthrough discoveries related to these stem cells; they have weaknesses that distinguish them from other cell populations in the body, and those weaknesses, which relate to unique ways in which these cells choose to metabolize energy, can be exploited or targeted with particular therapies. Our project seeks to test the theory that specifically targeting metabolic weaknesses in stem cell populations can lead to deep and durable responses for patients with myeloid malignancies. These discoveries will help us to learn how these diseases can ultimately be cured, and will set the stage for the necessary clinical trials that will be designed to do just that.