The goal of my research is to identify molecular and genetic differences between human acute myeloid leukemia (AML) stem cells and their normal counterparts and to develop therapeutic strategies directed against these targets. We have used bioinformatic analysis to identify genes and pathways preferentially expressed or activated in LSC. From this analysis we identified a number of cell surface protein markers that are more highly expressed on AML LSC compared to their normal counterparts. We then determined that one of these protein markers, CD47, contributes to leukemia development by blocking the ingestion and removal of leukemia cells by cells of the immune system. Most significant, we determined that blocking monoclonal antibodies directed against CD47 targeted LSC and depleted leukemia in mouse pre-clinical models. Our major focus is the development of therapeutic antibodies directed against CD47 and additional protein markers present in much larger amounts on the external surface of the LSC compared to the normal blood-forming stem cells. We hope to develop a clinical-grade therapeutic antibody for the treatment of AML that will be investigated in clinical trials at the Stanford Cancer Center.
Ludwig Center at Stanford
Lokey Stem Cell Research Building
265 Campus Dr., 3rd Floor
Stanford, California, U.S. 94305-5323
T 650 234 0675