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.

How will your idea make a difference

Through this initiative, the National Cancer Institute, could foster the development of a data eco-system that allows for such pre-clinical data to be shared in a comprehensive and harmonized fashion. Such an eco-system must allow for easy data deposition and include bioinformatic and interpretation tools that would efficiently predict and/or prioritize novel therapeutic targets to be tested in the clinical setting. Establishing such data-sharing infrastructure could also be instrumental in defining targets that might benefit from being targeted with combination therapies.

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Idea No. 974