The main goal of the Pediatric Preclinical Testing Consortium (PPTC) is to develop models that will translate to human clinical practice and effectively prioritize therapies for the treatment of pediatric cancers. An effective Coordinating Center (CC) will be critical to the Consortium's goals by providing broad organizational, scientific, and analytic expertise to support NCI scientists and the PPTC Research Programs. The CC will also be responsible for developing, implementing, and evaluating a comprehensive PPTC research program, providing leadership in data collection and ongoing program evaluation to ensure that the research program is making optimal progress toward the PPTC mission. Providing the organizational and administrative leadership necessary to enable the Consortium to function efficiently will also be a key function of the CC. RTI's goal as the proposed CC for the PPTC over the next 5 years is to optimize Consortium progress in generating reliable data to accelerate the development of more effective treatments for children with cancer by achieving the following specific aims: 1. Guide interactions between Consortium members and the pediatric oncology community at large. Provide the logistical support necessary to run an efficient and productive Consortium through effective teleconferences, webinars, Consortium meetings, and websites, both public and restricted. Employ these tools to facilitate interactions between PPTC members, pediatric clinicians, pharmaceutical companies, and pediatric oncology advocacy organizations to ensure that promising new therapies are utilized efficiently. 2. Generate reliable data that can be used for prioritization of the therapeuic agents in pediatric cancer. Provide efficient and robust procedures for data collection, statistica analysis of data collected from the Research Programs, and bioinformatic analysis of testing results that can relate treatment effects to molecular characteristics of the preclinical models utilized. 3. Provide the Consortium with a comprehensive, yet flexible set of scientific, statisticl and analytic resources needed to develop an innovative research program for creating a functional Consortium for pediatric preclinical testing. Develop tools to track the status of each potential drug candidate through the preclinical process. Document variables in a Consortium-side data dictionary as one element to build the data management system in REDCap.
Recent studies suggest that although more children today are surviving cancer than ever before, young patients who were successfully treated in the 1970s and 80s may live a decade less, on average, than the general population. Also, depending on the type of cancer, the estimated loss of life expectancy ranges from 4 years to more than 17 years. Causes of the premature deaths include recurrences of initial cancer, new cancers caused by drug and radiation therapy, and other delayed complications from cancer treatments. The proposed Consortium will leverage available pediatric preclinical data to prioritize the drugs that can be studied in pediatric clinical trials for development of more effective treatments for children with cancer that result in increased survival and higher quality of life.
