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Biology-based clinical trials for children with cancer.

Institution: 
Children's Hospital of Philadelphia
Researcher(s): 
Rochelle Bagatell
Grant Type: 
Phase I/II Infrastructure Grants
Year Awarded: 
2009
Type of Childhood Cancer: 
General Pediatric Cancer
Project Description: 
Disease-specific research teams at the Children's Hospital of Philadelphia (CHOP) have been successful in bench to bedside work, having identified key molecules involved in the genesis of neuroblastoma, and subsequently having studied agents that target these molecules in preclinical and clinical settings. From a programmatic point of view, it is logical to build upon this success and apply the bench to bedside paradigm beyond the disease-specific setting. The principle investigators of this application seek to develop a pathway oriented, cross-disease developmental therapeutics (DT) program that will facilitate new drug development in pediatric oncology. With assistance from an ALSF infrastructure award, the CHOP DT team will conduct early phase clinical trials of new therapeutics with an emphasis on biology-based, investigator-initiated trials of molecularly targeted agents in combination with chemotherapy.

Development and implementation of biology-based early phase trials requires collaboration between skilled clinical and basic science researchers. It also requires commitment on the part of investigators, research nurses, data managers, social workers, patients, and families. Many of the components necessary to carry out this work are in place, but they have not yet been integrated to yield a DT program that can develop and execute biology-based early phase clinical trials across tumor types. An Alex's Lemonade Stand Program Infrastructure Grant would substantially benefit the current DT team by supporting a research nurse. This research nurse will facilitate creation of the collaborative research environment required to conduct investigator initiated early phase clinical trials of novel therapies.