Developing Childhood Cancer Therapies

The National Cancer Institute (NCI) www.cancer.gov has awarded grants to teams from the Albert Einstein College of Medicine Children’s Cancer Research Institute, Baylor College of Medicine, Children’s Hospital of Philadelphia, and the Children’s Cancer Institute in Australia, to enable participation in the Pediatric Preclinical Testing Consortium (PPTC) www.ncippt.org.

The PPTC is designed to address an important barrier to developing new drugs to treat childhood cancers and to produce reliable data from studies of laboratory animals used as preclinical models to help to prioritize which agents to pursue in human trials

“Effective prioritization of potential drug candidates is critical,” said Malcolm Smith, MD, PhD, and Associate Branch Chief of Pediatrics in NCI’s Cancer Therapy Evaluation Program. “There is a large universe of anticancer agents being developed for adult cancers, but because of the relatively small number of children with specific cancers, only a limited number of these agents can be studied in pediatric clinical trials. One of the most important findings from the PPTP was that many of the agents showing efficacy against adult cancers had limited activity in pediatric preclinical models.”

According to Dr. Smith, “The PPTP and other research teams have shown that preclinical testing, when combined with knowledge about drug exposures that can be tolerated by mice and humans, provides powerful insight into the likely clinical utility of experimental agents,”

RTI International www.rti.org as the Coordinating Center for the PPTC will provide the necessary administrative coordination, data management, and statistical support so that the consortium can operate smoothly.

RTI will also work with the team members on data collection, statistical analysis of data, and bioinformatics analysis of testing results. The plan is to relate treatment effects to molecular characteristics of the preclinical models and increase translatability of preclinical data to human clinical trials.