Glioblastoma: Emerging treatments and novel trial designs

Simple Summary Nowadays, very few systemic agents have shown clinical activity in patients with glioblastoma, making the research of novel therapeutic approaches a critical issue. Fortunately, the availability of novel compounds is increasing thanks to better biological knowledge of the disease. In this review we want to investigate more promising ongoing clinical trials in both primary and recurrent GBM. Furthermore, a great interest of the present work is focused on novel trial design strategies. Management of glioblastoma is a clinical challenge since very few systemic treatments have shown clinical efficacy in recurrent disease. Thanks to an increased knowledge of the biological and molecular mechanisms related to disease progression and growth, promising novel treatment strategies are emerging. The expanding availability of innovative compounds requires the design of a new generation of clinical trials, testing experimental compounds in a short time and tailoring the sample cohort based on molecular and clinical behaviors. In this review, we focused our attention on the assessment of promising novel treatment approaches, discussing novel trial design and possible future fields of development in this setting.