The NIH is uniquely suited to create a rare CNS cancer initiative. This effort will utilize the following specific resources of the NIH. This has required developing a clinical trial infrastructure, including data management system, to oversee the disease specific therapeutic and natural history studies. Collaborations with experts in imaging technologies to create imaging biomarkers and routine imaging for monitoring response to therapies. This initiative will combine the expertise from both pediatric and adult investigators, benefitting from the varied approaches from these investigators. A tissue repository has been developed to store both tumor and normal tissue for molecular analyses including genomics, metabolomics, and proteomics with the intent of advancing the understanding of tumor biology and pathogenesis leading to better prognostic information, sub-classification and ultimately markers predictive of response to specific treatments. Additionally, specific testing capabilities will be established based on project need. For example, an immunotherapy trial is anticipated and immunologic monitoring capabilities will be needed as a critical component of this study. NIH-based outreach efforts will be utilized to interact with patient advocacy groups to enhance patient participation in these efforts and provide a venue for educational programs to improve patient and caregiver understanding of their uncommon disease.
