NR5A1 and leukemia: Four of these agents were confirmed to preferentially target KMT2A-r leukemia cells, including the adenosine analog 2-chloroadenosine triphosphate (2-Cl-ATP), SID7969543, an inhibitor of NR5A1, β-carboline-3-carboxylate (β-CCB), an endogenous proconvulsant and anxiogenic benzodiazepine receptor ligand, and oxethazaine, a Na+ channel blocker (Figure 1C).