John Bushweller's lab develops targeted therapy for one subtype of acute myeloid leukemia (published in Science)

Dr. Bushweller's lab has developed a potent, selective, and non-toxic small molecule inhibitor of the CBFB-SMMHC transcription factor fusion protein which is a driver of disease for one subtype of acute myeloid leukemia (AML). This compound shows efficacy in a mouse model of this type of leukemia as well as against patient samples of this type of leukemia. The work is an exciting demonstration of the utility of targeting transcription factor drivers of cancer. As this class of proteins has long been viewed as "undruggable", the success of this agent is a harbinger of further efforts in this area. The work was published in Science: A small-molecule inhibitor of the aberrant transcription factor CBFβ-SMMHC delays leukemia in mice