The molecular classification of medulloblastoma: driving the next generation clinical trials.

Publication Type:

Journal Article


Current opinion in pediatrics, Volume 24, Issue 1, p.33-9 (2012)


2012, Center-Authored Paper, Clinical Research Division, January 2012


Most children diagnosed with cancer today are expected to be cured. Medulloblastoma, the most common pediatric malignant brain tumor, is an example of a disease that has benefitted from advances in diagnostic imaging, surgical techniques, radiation therapy and combination chemotherapy over the past decades. It was an incurable disease 50 years ago, but approximately 70% of children with medulloblastoma are now cured of their disease. However, the pace of increasing the cure rate has slowed over the past 2 decades, and we have likely reached the maximal benefit that can be achieved with cytotoxic therapy and clinical risk stratification. Long-term toxicity of therapy also remains significant. To increase cure rates and decrease long-term toxicity, there is great interest in incorporating biologic 'targeted' therapy into treatment of medulloblastoma, but this will require a paradigm shift in how we classify and study disease.