Glioblastoma

Glioblastoma (GBM) is a disease that strikes without warning, often manifested by a single seizure or a few days of progressive headaches, yet evaluation with magnetic resonance imaging reveals a large mass that was likely present for months. Its stealth-like behavior belies the hallmark feature of this deadly cancer; it infiltrates normal brain diffusely, early in the disease course and over long distances, making it impossible to detect early or to cure surgically. The median survival time is 15 months with approximately 25% of patients alive at 2 years. The mainstay of treatment is external beam radiation in combination with the oral alkylating agent, temozolomide. The glioblastoma genome has been well characterized, yet no molecular targeted therapies have yet proven effective in the clinic. Improvements in outcome for this devastating disease will require a better understanding of the pathophysiology and the development of robust preclinical models for testing new therapies.