As a member of the Pediatric Brain Tumor Clinical Trials Consortium, the CNMC Neuro-Oncology Program proposes to develop, facilitate, and participate in innovative hypothesis-driven, technically challenging, clinical research designed to improve the survival and quality of life of children with primary central nervous system tumors. It is anticipated that these investigations will include, but will not be limited to: the use of novel chemotherapeutic agents; means to overcome the blood-brain barrier, immunotherapeutic approaches; modifications of radiation therapy; and new neurobiologic approaches, such as gene therapy, maturation agents, and anti-angiogenesis agents. The CNMC Neuro-Oncology Program plans on participating in research investigations which will improve the means to diagnose and characterize childhood brain tumors and to develop and participate in carefully monitored innovative diagnostic and therapeutic studies which will lead to future Phase III studies for children with such tumors. Over the past five years, the CNMC Neuro-Oncology Program has evaluated and managed 442 children (new to the institution) with primary central nervous system tumors and has entered over 189 children on Phase I, Phase II, and Phase III clinical investigations. Investigations have been done over this period of time, in concert with private industry and working groups, evaluating novel approaches such as gene therapy, immunotherapy, and approaches to overcome the blood-brain barrier; as well as evaluations of new chemotherapeutic agents, intensification of chemotherapy, and means to increase the efficacy of radiotherapy. The CNMC Neuro-Oncology Program has a well-developed multidisciplinary clinical core which includes a weekly neuro-oncology clinic, a quarterly groupwide neuro- oncology planning meeting, and a regional referral system. The program has a well-designed data management system, and an established system for specimen accrual. State-of-the-art neuroradiologic, neuropathologic, neurosurgical and radiation-oncologic facilities are available. In addition, innovative neurobiologic investigations in childhood brain tumors have been successfully completed and are underway at CNMC. The well-developed program structure, expertise of the CNMC Neuro-Oncology Program, proven commitment to performance of clinical trials, and available facilities should ensure the ability of the CNMC Neuro-Oncology Program to effectively participate in the proposed Pediatric Brain Tumor Clinical Consortium.
