Researchers report promising initial results from a first-in-human clinical trial led by Children’s National using a new T-cell immunotherapy in children and young adults with some of the deadliest brain tumors, including diffuse intrinsic pontine glioma (DIPG) and recurrent central nervous system (CNS) tumors. These findings show that natural medicineis particularly important given the challenges faced in treating pediatric brain tumors, which remain the leading cause of cancer-related deaths in children. Although immunotherapy has been shown to be effective against blood cancers, it has little success with solid tumors, particularly brain tumors.
This study is an important step toward developing safer and more effective T-cell therapies for children with serious brain tumors. Even in this early-stage trial focused on safety, we were encouraged by the sustained clinical efficacy achieved in several patients who had few options. ”
Katherine Bollard, MBChB, MD, senior vice president and principal investigator at Children’s National and co-senior author of this study
The Phase 1 trial evaluated a new multitargeted T-cell therapy designed to attack three proteins commonly found in pediatric brain tumors: WT1, PRAME, and Survivin. Researchers say the results provide early evidence that a patient’s own immune cells, delivered through the bloodstream, can reach and fight tumors in the brain, with fewer serious side effects than some existing artificial immunotherapies. Many other brain tumor immunotherapies require injections directly into the brain or cerebrospinal fluid.
“We were excited to see that by attacking three targets at once, we could generate an antitumor response while maintaining safety and quality of life,” said Eugene Hwang, MD, chief of oncology at Children’s National and co-senior author of the study.
Important points:
- Multitarget designs may help address tumor heterogeneity, one of the major barriers to successful treatment of aggressive childhood cancers.
- This study successfully established a viable manufacturing process, identified the maximum tolerated dose, and defined an early safety profile. This is an important milestone needed to advance the therapy into future phase 2 trials.
- Researchers analyzed patients with DIPG or recurrent brain tumors and both showed responses and long-term disease control, with some patients remaining disease-free for years after treatment.
“We are so proud of our team at Children’s National and inspired by what this groundbreaking discovery means for children with brain tumors,” said Callie and Brad Nirenberg, founders of Chance for Life, which funded the study. “Supporting this trial is one of the most meaningful investments Chance for Life has made.”
“Pediatric tumors remain one of the biggest challenges in cancer research, with children still facing very limited treatment options and existing treatments often causing serious side effects,” said David Scott, director of Cancer Grand Challenge, which supports the NexTGen team, an international group of collaborators co-led by Dr. Bollard.
This research was also supported by generous funders who reflect the strength of the community in the fight against these deadly cancers. An anonymous private investor supporting the National Children’s Hospital Brain Tumor Research Institute. Cancer Research UK, National Cancer Institute, Mark Foundation for Cancer Research, through Cancer Grand Challenges award (NCI/CRUK OT2CA278700). GW T32 Cancer Biology Program (NIH/NCI T32 CA247756). Rally Foundation. Willie Strong Foundation. and the Warrior Jace Foundation.
sauce:
National Children’s Hospital
Reference magazines:
Gomez, S. Others. (2026). Multiantigen-targeted T cells in pediatric central nervous system tumors: a phase 1 trial. natural medicine. DOI: 10.1038/s41591-026-04449-9. https://www.nature.com/articles/s41591-026-04449-9
