Repurposing immunosuppressants for antileukemia therapy

Drug repurposing, the strategy to identify new therapeutic use for clinically approved drugs has attracted much attention in recent years. This strategy offers various advantages over traditional approaches to develop new drugs, including shorter development timelines, low cost, and reduced risk of failure. In this issue of EMBO Molecular Medicine, Liu et al show that inosine monophosphate dehydrogenase (IMPDH) inhibitors, the well-known immunosuppressants have a potent therapeutic effect on the aggressive blood cancer, acute myeloid leukemia with MLL rearrangements. Intriguingly, the antileukemia effect of IMPDH inhibitors is mediated, at least in part through the overactivation of TLR signaling and Vcam1 upregulation. The robust antileukemia effect of IMPDH inhibitors, both in vitro and in vivo, together with their mechanistic findings provides a rational basis for repurposing IMPDH inhibitors for antileukemia therapy.