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Therapeutic Advances in the Treatment of Glioblastoma: Rationale and Potential Role of Targeted Agents

^a Preston Robert Tisch Brain Tumor Center, Duke University Medical Center, Durham, North Carolina, USA; ^b Center for NeuroOncology, Dana Farber/Brigham and Women’s Cancer Center, Boston, Massachusetts, USA

Key Words. Angiogenesis inhibitors • Glioblastoma multiforme • Targeted therapy • Tyrosine kinase inhibitors

David A. Reardon, M.D., Preston Robert Tisch Brain Tumor Center, Duke University Medical Center, DUMC 3624, Durham, North Carolina 27710, USA. Telephone: 919-684-3457; Fax: 919-684-6674; e-mail: reard003{at}mc.duke.edu and Patrick Y. Wen, M.D., Center for NeuroOncology, Dana Farber/Brigham and Women’s Cancer Center, 44 Binney Street, Boston, Massachusetts 02115, USA. Telephone: 617-632-2166; Fax: 617-632-4773; e-mail: patrick_wen{at}dfci.harvard.edu

Despite advances in standard therapy, including surgical resection followed by radiation and chemotherapy, the prognosis for patients with glioblastoma multiforme (GBM) remains poor. Unfortunately, most patients die within 2 years of diagnosis of their disease. Molecular abnormalities vary among individual patients and also within each tumor. Indeed, one of the distinguishing features of GBM is its marked genetic heterogeneity. Nonetheless, recent developments in the field of tumor biology have elucidated signaling pathways and genes involved in the development of GBM, and several novel agents that target these signaling pathways are being developed. As new details on the genetic characteristics of this disease become available, innovative treatment regimens, including a variety of traditional treatment modalities such as surgery, radiation, and cytotoxic chemotherapy, will be combined with newer targeted therapies. This review introduces these new targeted therapies in the context of current treatment options for patients with GBM. It is hoped that this combined approach will overcome the current limitations in the treatment of patients with GBM and result in a better prognosis for these patients.

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