Skip to Content
U.S. National Institutes of Health
Last Updated: 08/31/15

Pediatric Preclinical Testing Consortium (PPTC)

The NCI-supported Pediatric Preclinical Testing Consortium (PPTC) is a program to systematically evaluate novel agents against genomically characterized childhood cancer solid tumor and leukemia in vivo models. The primary goal of the NCI PPTC is to develop high quality preclinical data to help pediatric oncology researchers identify new agents that will show significant activity when clinically evaluated against selected childhood cancers. By supporting a more reliable agent prioritization process, the PPTC contributes to the goal of accelerating the discovery of more effective treatments for children with cancer.

The NCI PPTC testing strategy is based upon research showing that preclinical testing using genomically characterized models, when combined with knowledge about the relative drug exposures tolerated in mice and in humans, provides powerful insight into the likely clinical utility of investigational agents.

The NCI PPTC is supported through cooperative agreement grants to its Coordinating Center and to its Research Programs that perform the testing. PPTC members are listed below:

Role/Tumor PanelPrincipal InvestigatorInstitution
Coordinating CenterDiana Severynse-Stevens, PhDResearch Triangle Institute (Durham, NC)
Sarcoma and Renal TumorsPeter Houghton, PhDGreehey Children's Cancer Research Institute (San Antonio, TX)
NeuroblastomaJohn Maris, MDChildren's Hospital of Philadelphia (Philadelphia, PA)
LeukemiaRichard Lock, PhDChildren's Cancer Institute (Sydney, Australia)
Brain TumorsXiao-Nan Li, MD, PhDTexas Children's Hospital (Houston, TX)
OsteosarcomaRichard Gorlick, MDAlbert Einstein College of Medicine (New York, NY)

The NCI PPTC builds upon ten years of experience with the Pediatric Preclinical Testing Program (PPTP), which collaborated with more than 50 pharmaceutical companies to test novel agents against the program's pediatric preclinical models. A complete listing of the PPTP presentations and publications is available for review. The PPTP found that many agents that are effective for adult cancers have limited activity against pediatric preclinical models. Activity signals were observed for some agents, including selumetinib for BRAF-mutated glioma, the Bcl-2 inhibitor ABT-263 for acute lymphoblastic leukemia (ALL), the MDM2 inhibitor RG7112 for ALL, selected antibody-drug conjugates for models expressing relevant surface antigens, and the PARP inhibitor talazoparib plus low-dose temozolomide for Ewing sarcoma.

Information about the NCI PPTC is available at its website ( or can be obtained by contacting the PPTC Program Director (Dr. Malcolm Smith at or by contacting the PPTC directly at

^ Back to Top
< Previous | Next >