Biomarker-guided and systemic treatment strategies for meningioma

Meningiomas represent the most common intracranial tumors, overall. While a majority of tumors can be considered indolent, a subset of patients will experience repeated recurrences despite surgical and radiotherapeutic interventions. For these patients, there are only a limited number of promising systemic therapy options available. Effective drug development for meningiomas has been limited by variability in trial design, the clinical trial endpoints being used across studies, heterogeneous tumor biology and patient selection in clinical trials, and inconsistent radiological response assessment criteria. However, over the past years, tremendous progress has been made in understanding the biological and molecular underpinnings of meningiomas. Guidance on clinical trial endpoints and response assessment criteria has also improved clinical trial design and it is widely recognized that patient participation in a clinical trial remains the best way to evaluate novel therapy options and move drug development forward. In this review, we discuss factors of optimal clinical trial design for meningioma clinical trials and review and discuss the evidence supporting the use of various contemporary biomarker-guided and systemic therapy options for patients with meningioma.