After co-funding a highly successful international clinical trial that opened for enrollment in 2017, A Kids’ Brain Tumor Cure Foundation, Solving Kids’ Cancer, and the Ty Louis Campbell (TLC) Foundation teamed up again to announce their joint financial support of a Phase 1 clinical trial designed to test the safety and efficacy of a novel immunotherapy developed by Apexigen for children with brain tumors.
The study tested the safety and efficacy of a promising new agent, APX-005M (CD40 agonist antibody), in children with brain tumors. This first-in-class immunotherapy demonstrated the potential for meaningful activity in children with recurrent or refractory central nervous system tumors and represented a new therapeutic option for children newly diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG), a pediatric brain cancer that was universally fatal at diagnosis. The trial was conducted through the Pediatric Brain Tumor Consortium (PBTC), a national network of multidisciplinary research institutions that enabled broader access to this investigational therapy across twelve leading pediatric treatment centers in the United States.
This marked the second immunotherapy trial jointly funded by these three like-minded nonprofits, reflecting their shared confidence in collaborative, multi-institutional research and their continued support of Principal Investigator Dr. Ira Dunkel of Memorial Sloan Kettering Cancer Center. At the time, the clinical trial was open for enrollment, with additional details available through clinicaltrials.gov.
“Collaboration is the driving force behind making progress in research, faster,” said Dr. Ira Dunkel. “Much like nonprofit organizations that have partnered for years to expand their impact, researchers established the Pediatric Brain Tumor Consortium to accelerate progress through multi-center, multidisciplinary studies grounded in rigorous statistical science.” Funding for the PBTC was provided by the US Government from 1999 though 2026.
Dr. Dunkel later reported that trial accrual was progressing well and on schedule, with non-DIPG enrollment concluded in mid-2019—a notable achievement in pediatric brain tumor research. Dose escalation had occurred across three levels, and initiation of the DIPG arm was anticipated based on dosing data from non-DIPG participants.
Collectively, the three nonprofit organizations contributed $120,000 to support the availability of this clinical trial for children with brain tumors. The study provided access to an investigational treatment option with substantially less toxicity than conventional chemotherapy, offering renewed hope for families facing limited therapeutic options.