In the Beat AML study describing drug sensitivity assays performed ex vivo on primary leukemia cells, RUNX1-mutated AML showed higher sensitivity to PIK3C and mTOR inhibitors and to the multi-kinase vascular endothelial growth factor receptor (VEGFR) inhibitor, suggesting the possibility of targeted therapy for mutated RUNX1; however, this remains to be validated in vivo [26]. This evidence concerns the gene RUNX1 and leukemia.