Abstract
Simple SummaryNowadays, 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.
Highlights
Glioblastoma (GBM) is the most common primary brain tumor, with an estimated incidence of 3.22/100,000 persons in the United States and a five-year overall survival of only 6.8% [1,2]
GBM can be diagnosed as a diffuse astrocytic glioma without isocitrate dehydrogenase (IDH) and H3R gene mutations, with microvascular proliferation, necrosis, and/or peculiar molecular features such as TERT mutation, epidermal growth factor receptor (EGFR) amplification, and/or gain of chromosome 7 combined with the loss of chromosome 10 [3,4,5,6]
The addition of TTFields lead to a significant increase in progression-free survival (PFS) (6.7 vs. 4.0 months, HR, 0.63; 95% CI, 0.52–0.76; p < 0.001) and overall survival (OS) (20.0 vs. 16.0 months HR, 0.63; 95% CI, 0.53–0.76; p < 0.001) over standard treatment, without significant difference in adverse events
Summary
Glioblastoma (GBM) is the most common primary brain tumor, with an estimated incidence of 3.22/100,000 persons in the United States and a five-year overall survival of only 6.8% [1,2]. Recurrent GBM can be managed by different approaches [11,12,13], including locoregional treatment and systemic treatments [2,14,15,16,17,18,19]. Several novel discoveries about the molecular, genomic, and biological background of the disease have been determined. None of these improvements translated into a significant progress in terms of therapeutic options. Several drugs and approaches showing promising results in early studies failed to confirm a clinical improvement on large randomized trials. We reviewed more promising experimental systemic treatments for patients in early phase of development, as well as in patients with newly diagnosed and recurrent GBM
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