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Last Updated: 11/05/20

NCI Pediatric in Vivo Testing Program

NCI is soliciting applications for research teams to participate in the NCI Pediatric in Vivo Testing Program in response to RFA-CA-20-034 and applications to serve as the Coordinating Center for the Program in response to RFA-CA-20-041.

Genomic Characterization of Pediatric Preclinical Models

As described in NOT-CA-20-108, NCI is seeking information on pediatric preclinical models [e.g., cell lines and patient-derived xenografts (PDXs)] for which genomic characterization will enhance their research value. NCI plans to characterize approximately 500 pediatric preclinical models. Genomic characterization will include whole exome sequencing, RNA-seq, and DNA methylation array analysis. The data generated will be incorporated with existing genomic characterization data into a Pediatric Preclinical Data Commons that NCI is establishing as part of the Childhood Cancer Data Initiative to serve as a resource to the pediatric research community.

As the intent of performing genomic characterization is to make the results available to the research community, an Institutional Certification(s) using the current NIH template that demonstrates that it is permissible to share individual-level genomic data will be required before models can be submitted for genomic characterization. A Provisional Certification can be supplied as part of the response to the RFI. Guidance on obtaining an Institutional Certification is available at the NIH website.

Research teams wishing to nominate models for genomic characterization are asked to provide the information listed below by October 30, 2020 to Dr. Malcolm Smith ( Include NOT-CA-20-108 in the subject line of the email.

  • A listing of the models and their characteristics. Use the Excel file PDX_GenomicChar_2020_09_30.xlsx, and rename it to add the name of the researcher proposing the models.
  • A Provisional Certification indicating that it will be permissible to share individual-level genomic data for the samples proposed.
  • Indicate in the Excel file the ability to provide 30-50 mg of frozen tissue for PDX models or 1-10 x 106 cells for cell line models.

For inquiries, contact Dr. Malcolm Smith at

Pediatric Preclinical Testing Public-Private Partnership (PPTP3)

NCI plans to continue its support for pediatric preclinical testing in 2021 and beyond through the Pediatric Preclinical Testing Public-Private Partnership (PPTP3). Plans for the PPTP3 were presented at the NCI Board of Scientific Advisors meeting (May 12, 2020). Slides from the presentation are available for download.

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 Panel Principal Investigator Institution
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 listing of the PPTP 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