A single agent induces apoptosis of AML blasts and prolongs survival in AML xenograft models.

To improve outcomes for pediatric AML, identifying novel agents with tolerable side effect profiles is critical. Recently, an agent has been shown to reduce signaling of myeloid lineage cells. This FDA-approved drug is well tolerated and has known pediatric dosing that achieves plasma concentrations of 40-80µM. We conducted pre-clinical testing of this agent with AML cell lines and pediatric patient samples. Cell lines demonstrated time and dose-dependent induction of apoptosis.