Previous treatment strategies for medulloblastoma have shown moderate improvement in response. However, the long-term overall survival remains disappointing, and many survivors have profound long-term complications. Therefore, there is a tremendous need to develop novel, improved, and safer therapeutic strategies for medulloblastoma. The critical tumor-stroma interactions mediated by the PlGF/NRP1 pathway make it an attractive target. Sholler and colleagues report a phase I, open-label, multicenter, dose-escalation study of the anti-PlGF antibody, TB-403, in pediatric subjects. TB-403 treatment was well tolerated and induced stable disease in high-risk, heavily pretreated relapsed medulloblastoma allowing for excellent quality of life. These findings indicate that treatment with TB-403 may represent a potentially transformative therapy for children with medulloblastoma and should be tested in larger studies.

Costimulatory molecules expressed on the surface of T cells—such as 4-1BB, OX40, inducible T-cell costimulator (ICOS), and glucocorticoid-induced tumor necrosis factor receptor (GITR)—are potential targets to enhance T-cell antitumor activity. To...

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