Genetically predicted Ornithine Metabolites mediate the association between ODC1 Inhibition and Neuroblastoma.

DIFLUOROMETHYLORNITHINE (DFMO), as a drug that targets and inhibits ODC1 gene, has been proven to be effective for high-risk neuroblastoma (HR-NB) and has started clinical trials. The effects of DFMO on NB were still remain unclear. This study employed a drug-targeted Mendelian randomization (MR) approach to investigate the association between genetically predicted metabolites-modulating effects of commonly targeted ornithine decarboxylase-lowering agents and the risk of NB, with the aim of providing a theoretical foundation for the use of ornithine decarboxylase-lowering drugs in NB treatment.