First-in-Class Immunotherapy for Relapsed and Refractory Pediatric Brain Tumors

Project Title:  Phase 1 study of APX-005M CD40 agonist antibody in children with CNS tumor

Researcher: Ira Dunkel, MD

Institutions: Pediatric Brain Tumor Consortium

Study Type: Phase I


This phase I clinical trial is a collaboration to evaluate dosage and side effects of APX005M, an investigational immune activating compound that targets CD40, in pediatric patients with recurrent or refractory brain tumors. CD40 is an immune co-stimulatory receptor essential to the activation of both innate and adaptive immune responses against cancer. There is an unmet need in pediatric oncology for effective treatments for Central Nervous System (CNS) tumors. Immunotherapy is currently considered a promising area of investigation in clinical oncology and it is expected that novel immune-activating agents such as APX005M will provide additional benefit to complement the currently used immune checkpoint inhibitors.


To learn more about this trial, visit


Charity Partners:  Ty Louis Campbell Foundation, A Kids’ Brain Tumor Cure

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