Research Questions

Prioritizing novel therapeutic agents identified in pre-clinical research for testing in the clinical setting

Predictive disease models of pediatric hematologic cancers can inform efficient testing of possible novel therapeutic agents in the clinical setting. However, to ensure that these models are useful in prioritizing novel therapeutic targets, pre-clinical assessments must be conducted. Conducting such analysis efficiently and consistently will require investigators to share pre-clinical data generated from models (e.g., genetically engineered mouse models, patient-derived xenografts, etc.) that depict hematologic cancers likely to occur in the pediatric patient population as well as pharmacokinetic data.

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Idea No. 974