Neurosurgical leadership in neuro-oncology clinical trials: A nationwide study

Despite therapeutic advances, central nervous system (CNS) tumors such as glioblastoma and brain metastases remain highly lethal. Neurosurgeons have historically driven innovation in neuro-oncology but may be underrepresented as principal investigators (PIs) in clinical research. We analyzed all 525 U.S.-based neuro-oncology trials listed on ClinicalTrials.gov as of October 2024 to evaluate trends in neurosurgeon-led research, stratified by phase, funding source, and institutional characteristics. Oncologists led 34.8% of trials, whereas neurosurgeons led 21.5%. When normalized to the number of board-certified specialists, neurosurgeons demonstrated greater per-capita leadership than oncologists. Neurosurgeon-led studies were primarily early-phase (36.7% phase I; 5.1% phase III/IV), underscoring their role in translational and first-in-human investigations. At the state level, neurosurgical leadership correlated strongly with the number of neurosurgical residency programs (R² = 0.4835, p < 0.001) and aggregated departmental NIH funding (R² = 0.6146, p < 0.001), with states possessing greater training infrastructure and research resources demonstrating higher rates of neurosurgeon-led clinical trials. Nevertheless, 55.3% of neurosurgeon PIs lacked direct NIH support, highlighting persistent funding barriers. Notably, women were nearly twice as likely as men (1.8×) to serve as PIs, suggesting emerging gender diversity in neurosurgical research leadership. Neurosurgeons maintain a significant presence in neuro-oncology clinical trials, particularly in early translational work. However, their limited representation in NIH-sponsored and late-phase studies reflects enduring structural and funding challenges. Strengthening research mentorship, expanding grant access, and fostering interdisciplinary collaboration may enhance neurosurgical leadership and accelerate therapeutic innovation in neuro-oncology.