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U.S. National Institutes of Health
Last Updated: 05/28/15

Childhood Cancer Resources

Pediatric Clinical Trials Cooperative Groups and Consortia

  • Children's Oncology Group
    The Children's Oncology Group (C.O.G.) is supported by the National Cancer Institute to conduct clinical trials devoted exclusively to children and adolescents with cancer. C.O.G. develops and coordinates cancer clinical trials conducted at its more than 200 member institutions, which include cancer centers of all major universities and teaching hospitals throughout the U.S. and Canada, as well as sites in Europe and Australia. C.O.G. members include over 5000 cancer researchers dedicated to saving the lives of children with cancer. C.O.G. was formed by the merger of the four previous children's cancer cooperative groups (CCG, POG, IRSG, NWTSG) in order to accelerate the search for a cure for the cancers of children. Through the C.O.G. network of member institutions, children with cancer, regardless of where they live, can access state-of-the art therapies and the collective expertise of world-renowned pediatric specialists.
  • Children's Oncology Group (C.O.G.) Phase 1/Pilot Consortium
    The primary objective of the C.O.G. Phase 1/Pilot Consortium is to expeditiously develop and implement pediatric phase 1 and pilot studies, thus facilitating the integration of advances in cancer biology and therapy in the treatment of childhood cancer. The Consortium includes approximately 20 institutions. Pharmacokinetic and biological correlative studies are key components of the consortium's phase 1 trials and are increasingly important for new agents with specific molecular targets. The Consortium is funded to conduct pilot studies of promising multi-agent regimens (which may or may not involve a dose escalation). These studies are an important step in the integration of new agents into the therapy of specific childhood cancers and require careful monitoring for toxicity and safety such as can be provided by the Consortium's member institutions. After their initial evaluation for safety in children by the Consortium, the agents and regimens can then be studied within the larger group of C.O.G. institutions to determine their role for in the treatment of specific childhood cancers.
  • Pediatric Brain Tumor Consortium
    The primary objective of the PBTC is to rapidly conduct phase 1 and 2 clinical evaluations of new therapeutic drugs, intrathecal agents, delivery technologies, biological therapies, and radiation treatment strategies in children age 0 - 21 years of age with primary CNS tumors. The PBTC includes nine leading academic institutions with extensive experience in the design and conduct of childhood cancer clinical trials for children with brain tumors. Another objective of the PBTC is to develop and coordinate innovative neuroimaging techniques on a consortium-wide basis. Results from PBTC studies will be available to large international collaborative groups for confirmatory phase 2 and multiagent phase 3 clinical trials. Specifically, direct interactions have been established with the the Children's Oncology Group. This collaborative interaction assures a rapid transition from phase 1 and 2 trials conducted by the PBTC.
  • New Approaches to Neuroblastoma Therapy Consortium
    NANT is a consortium of University and Children's Hospitals funded by the NCI to test promising new therapies for neuroblastoma. NANT members constitute a group of closely collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk neuroblastoma. The group conducts limited-institution clinical trials to test new drugs and new combinations of drugs against high-risk neuroblastoma with the goal that promising therapies can be tested nationally.
  • Childhood Cancer Survivor Study
    The Childhood Cancer Survivor Study (CCSS) was created to gain new knowledge about the long-term effects of cancer and its therapy. This knowledge can help design treatment protocols and intervention strategies that will increase survival and minimize harmful health effects. The CCSS also serves to educate survivors about the potential impacts of cancer diagnosis and treatment on their health, and to provide follow-up care, for example, by creating and implementing programs for the prevention and early detection of late effects. The CCSS is a component of the Long-Term Follow-Up Study, a collaborative, multi-institutional study, funded by the National Cancer Institute, of individuals who survived five or more years after treatment for cancer, leukemia, tumor, or similar illness diagnosed during childhood or adolescence. The CCSS is a retrospectively ascertained cohort of 20,346 childhood cancer survivors diagnosed between 1970 and 1986; it also includes approximately 3,500 siblings of survivors, who serve as control subjects for the study. The CCSS cohort has been assembled through the efforts of 27 participating centers in the United States and Canada and is coordinated by investigators at the University of Minnesota. Initiated in 1993, the study was recently funded by the National Cancer Institute for continuation through 2004.

Pediatric Preclinical Testing Program (PPTP)

  • The NCI-supported Pediatric Preclinical Testing Program (PPTP) is a comprehensive program to systematically evaluate new agents against childhood solid tumor and leukemia models. The PPTP is supported through an NCI research contract to St. Jude Children's Research Hospital (SJCRH) with Dr. Peter Houghton as the Principal Investigator. Testing occurs both at SJCRH and also at subcontract sites that have expertise in specific childhood cancers, including: Children's Hospital of Philadelphia (John Maris), Albert Einstein Medical Center (Richard Gorlick), Duke University (Henry Friedman), Texas Tech Health Sciences Center (Patrick Reynolds), and Children's Cancer Institute Australia (Richard Lock). The primary goal of the PPTP is to identify new agents that have the potential for significant activity when clinically evaluated against selected childhood cancers. The program is based on a substantial body of data showing that appropriate childhood cancer preclinical in vivo models can recapitulate the antitumor activity of known effective agents and can prospectively identify novel agents subsequently shown to have clinical activity against specific cancers of children and adolescents. By facilitating development of a more reliable pediatric new agent prioritization process, the PPTP will contribute to the goal of identifying more effective treatments for children with cancer.
  • Detailed information about the PPTP and its testing procedures is available at the PPTP web site, as is a general overview of the PPTP and its testing program.
  • The PPTP preclinical models have been molecularly characterized for their gene expression profiles and their genomic copy number abnormalities/LOH, as described in a report by Neale, et al. The gene expression and genomic characterization data can be downloaded from the PPTP web site. They can also be accessed through the Pediatric Branch Oncogenomics Section Data Center (see "Pediatric Tumor Affymetrix Database").
  • PPTP Meeting Presentations Describing Testing Results
  • PPTP Publications
  • PPTP Collaborator MTA document
  • Instructions for submitting an agent for evaluation by the Pediatric Preclinical Testing Program
  • Questions concerning the PPTP can be addressed to the PPTP Project Officer, Dr. Malcolm Smith (

Information regarding the inclusion and protection of children enrolled on Federally supported clinical trials

FDA Pediatric Resources

Reports from CTEP-Sponsored Childhood Cancer Meetings