Novel Approaches & New Therapeutic Platforms
Tannishtha Reya (MCC)
Edward Ball (MCC)
Rafael Bejar (MCC)
Acute Myelogenous Leukemia (AML) is a cancer marked by rapid and uncontrolled growth of immature cells of the myeloid lineage. Although it is the most common acute leukemia in adults, current treatments, which include chemotherapy and bone marrow transplantation, are largely ineffective, leading to relapse and death in most patients. AML also occurs in children, and pediatric AML has a much poorer outcome than other childhood leukemias.In light of these issues, AML represents a disease with a significant unmet medical need. Given that there have been no new therapies for AML in the last 30-40 years, identifying new approaches to target AML is critically important. At a cellular level, AML is heterogeneous and has been shown to be driven by cancer stem cells. Thus strategies aimed at inhibiting cancer stem cell growth and renewal may target the fundamental propagative abilities of the tumor and allow development of a more targeted therapy.Importantly, AML can often arise from early stage myeloid disorders such as myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPN), thus also providing an opportunity to block its progression at earlier stages. Our goal is to develop a new therapeutic agent that can block growth and progression of these myeloid disorders. To this end we have developed antibodies that target a protein required for AML growth. We will test whether these antibodies can block progression of MDS/MPN to AML and improve outcomes. If successful, these exciting studies could to lead to a new treatment strategy for myeloid leukemia.