Ex Vivo Drug Sensitivity Assay Correlates with Clinical Response and Identifies Panobinostat and Bortezomib as a Potential Novel Drug Combination for Pediatric AML

Pediatric acute myeloid leukemia (AML) is a rare disease with roughly 600 cases diagnosed in the United States each year with minimal improvement in clinical outcomes over the last few decades. Diagnostic pediatric AML samples were assayed using an ex vivo drug sensitivity test to determine the correlation between ex vivo drug response and clinical outcome, and to compare conventional chemotherapy to other drug combinations ex vivo.