Erythroid and Megakaryoblastic Leukemia; from Models to Targeted Therapy

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07/04/23

Erythroid and Megakaryoblastic leukemias are rare forms of acute myeloid leukemia, defined by increased blasts with either erythroid or megakaryocytic features. This presentation will discuss molecular and pathologic insights gained from cell culture and murine models of erythroid and megakaryoblastic leukemias. Our analysis has identified FOLR1 as a specific therapeutic target that is uniquely expressed in CBF2AT3/GLIS2 (CBF/GLIS) acute megakaryoblastic leukemia. FOLR1 specific CAR T-cells and Stro-002, a FOLR1 antibody drug conjugate effectively target AML cells with FOLR1 expression resulting in increased survival of CBF/GLIS AMKL xenograft mice. Stro-002 has also shown significant efficacy in limited human studies under compassionate use basis. After viewing this lecture, participants should be able to:

1. Understand basic differences between pediatric and adult AML.
2. Understand controversies regarding diagnosis and evaluation of erythroleukemia
3. Understand the diagnostic features of CBF/GLIS acute megakaryocytic leukemia

Keith Loeb, MD, PhD Associate Professor Translational Science and Therapeutics Division, Fred Hutch Cancer Center Department of Laboratory Medicine and Pathology, University of Washington

01/18/23