Incorporation of biomarkers in phase II studies of recurrent glioblastoma.

The survival trends for glioblastoma (GBM) patients have remained largely static, reflecting a lack of improvement in the therapeutic options for patients. Less than 5 % of newly diagnosed GBM survives more than 5 years. Tumor relapse is nearly universal and the majority of patients do not respond to further systemic therapy. The results from phase II studies conducted with recurrent GBM patients have not translated to successful confirmatory studies and thus we have reached a significant roadblock in the development of new treatments for patients with recurrent GBM. The development of new, active, and potentially targeted drugs for the treatment of recurrent GBM represents a major unmet need. The incorporation of diagnostic/companion biomarker combinations into the phase II studies and appropriate stratification of the patients is lagging significantly behind other larger cancer groups such as breast, non-small cell lung cancer, and melanoma. We herein carried out a systematic review of the phase II clinical studies conducted in patients with recurrent GBM (2010-2013 inclusive) to assess the degree of biomarker incorporation within the clinical trial design.