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Therapy at a Crossroads

Roger J. Packer, MD; Gilbert Vezina, MD

Arch Neurol. 2008;65(11):1419-1424.

Medulloblastoma is the most common malignant childhood brain tumor and, although relatively uncommon in older patients, poses a therapeutic challenge in adults. With current means of therapy, children with nondisseminated medulloblastoma have a high likelihood of long-term survival; 80% or more will be alive 5 years after diagnosis and treatment, with many free of the disease. Even in children with disseminated disease, intensified therapy has been associated with improved survival rates, although some of this improvement may be more apparent than real. The quality of life in long-term survivors is a major issue, and most children who survive have substantial neurologic and cognitive sequelae. The outcome in infants and younger children with medulloblastoma is suboptimal, although there is some evidence to suggest that intensification of therapy has improved the likelihood of disease control. A better understanding of the biological characteristics of medulloblastoma including the cell or cells of origin and the aberrant cellular signaling pathways involved has the promise of dramatically changing tumor stratification and treatment in the near future. However, these biological advances have yet to be integrated into the treatment of medulloblastoma in children or adults.

Author Affiliations: Center for Neuroscience and Behavioral Medicine, Divisions of Neurology and Pediatrics (Dr Packer) and Radiology/Neuroradiology (Dr Vezina), Children's National Medical Center, and Departments of Neurology and Pediatrics (Dr Packer) and Radiology (Dr Vezina), The George Washington University, and Department of Neurology, Georgetown University (Dr Packer), Washington, DC; and Department of Neurosurgery, University of Virginia, Charlottesville (Dr Packer).

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