Systematic review of outcomes of immunotherapy in medulloblastoma

Medulloblastoma, the most common malignant brain tumor in children, has intrinsic characteristics that pose significant challenges to effective immunotherapy. A systematic review of studies published before 30 June 2025 investigated outcomes of immunotherapy modalities for medulloblastoma. Fifty-six studies met the inclusion criteria, encompassing at least 183 medulloblastoma patients. Nearly half were phase 1 trials (43%), and 18% were non-trial designs. Among 29 studies reporting clinical outcomes, adoptive cellular therapies in combination regimens (n=7; 24%) and immune checkpoint inhibitors (n=6; 21%) were most frequently evaluated. Overall, clinical benefit was limited. Median overall survival ranged from 1.29 to 47 months, and median progession-free survival from 0.79 to 11 months. Progressive disease was reported in 40 patients, partial responses in 13 patients, and complete responses in three patients. The authors conclude that despite increasing trial activity, immunotherapy has shown modest efficacy in patients with medulloblastoma.