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Challenges of developing new drugs for childhood cancers

Frank M Balis, Elizabeth Fox

New anticancer drugs are developed separately in children because of potential differences in drug disposition, tissue/organ sensitivity to the drug’s toxic effects and pathogenesis and tissue of origin of childhood cancers compared with cancers in adults. The focus of new drug development for childhood cancers has shifted to molecularly targeted drugs that selectively inhibit cell-signaling pathways responsible for the malignant phenotype. This new era of drug development will require a process for selecting new agents to study in children, based on target expression and new clinical trial end points and designs that determine the dose based on a therapeutic effect rather than toxicity. For new orally administered agents, pediatric liquid formulations are essential to accurately dose and study new agents in young children.