The Neurobiology and Brain Tumor Program (NBTP) is a comprehensive program that includes world leading neuroscientists, cancer biologists and physicians among its highly interactive membership. The members of the NBTP are drawn from 10 academic departments from across the institution. The Program is organized into three interest groups that share the common goals of advancing understanding of normal and neoplastic development in the central nervous system to improve the outcome of children with brain tumors:
- The fundamental neuroscience group studies the development and functioning of normal and neoplastic neural tissues as well as cell death. Research efforts within the fundamental neuroscience group have been facilitated greatly during the last five years through the obtaining of a Program Project Grant from the NCI to study normal and neoplastic growth in the brain.
- The translational research group provides a forum for direct collaboration between the fundamental neuroscience and brain tumor therapy groups with a focus on advancing understanding of brain tumor biology into the clinic. Translational research efforts within the Program have expanded dramatically. This has been achieved through the formation of multiple inter- and intra-programmatic laboratory and clinical collaborations. As testament to this success 45% (n= 16/35) of all Phase I/II clinical trials opened within the NBTP over the last five years, were based directly on pre-clinical data translated from laboratories across the St. Jude Cancer Center, including those of other Programs.
- The brain tumor therapy group conducts innovative early and late phase clinical trials, as well as studying brain tumor treatment late effects. Members of the brain tumor therapy group provide state-of-the-art pathology, neuro-oncology, neuroimaging, and radiation oncology expertise to the Program that greatly facilitates the conduct of comprehensive cutting edge clinical research. Program members also play leading and collaborative roles in the national consortia including the Children's Oncology Group and the Pediatric Brain Tumor Consortium.
This large coordinated effort by the NBTP to improve the lives of children with brain tumors relies heavily upon the outstanding shared resources provided by the Cancer Center and close collaborative links with other Cancer Center Programs.
Brain tumors are the most common solid malignancies of childhood and are a leading cause of cancer-related death in children. Further, since brain tumors arise within delicate and critical tissues, the treatment of these diseases frequently and significantly impairs the function of survivors. Therefore, the principal aim of the NBTP is to reduce the mortality and morbidity of children with brain tumors through the development of innovative new treatment approaches that are guided by basic and translational research. To achieve this we are pursuing the following major scientific goals:
- Elucidate the molecular and cellular basis of mammalian neuro-development and neuro-degeneration.
- Develop accurate disease risk stratification tools for childhood brain tumors that will allow conventional therapies to be used with greater efficiency.
- Develop effective and less toxic new treatments of childhood brain tumors through increased understanding of the cellular and molecular origins of these diseases.
- Develop interventions that minimize the long-term neuropsychological sequelae of treatment among survivors of pediatric brain tumors.
The NBTP has matured into a fully interactive Program in which advances in understanding of the biology of normal and malignant growth in the nervous system are translated rapidly into therapeutic trials in the clinic. These translational efforts include Phase I, II and III trials of the most common and lethal forms of childhood brain tumors. Initial research efforts that focused on primitive neuroectodermal tumors (PNET) and medulloblastoma have expanded to include basic laboratory and clinical studies of ependymoma and glioma. Detailed molecular studies of tumors obtained prospectively from patients treated within a number of ongoing clinical trials are also underway. These data should provide key information regarding the mode of action of specific molecular targeted therapies as well new and accurate disease-risk staging systems for brain tumors.
Amar Gajjar, MD, and Suzanne J. Baker, PhD, are co-leaders of the program.