Thanks to the financial support from Project to Cure, our team has made meaningful progress over the past year in understanding how the immune system can be harnessed to fight medulloblastoma, the most common malignant brain tumor in children. A central focus of the team's work has been studying natural killer (NK) cells, a type of immune cell that can recognize and destroy cancer cells without needing prior exposure to them. Through laboratory studies, the team has demonstrated that NK cells can effectively target medulloblastoma cells, and they have identified specific immune pathways that tumors exploit to evade destruction. The program has also developed patient-derived tumor organoids, miniature three-dimensional models grown from actual patient tumors, which allow researchers to study how immune cells interact with a child's specific cancer in a controlled setting. These organoids are being used alongside advanced imaging and genetic sequencing technologies to map the immune landscape of different brain tumor types, including medulloblastoma and diffuse intrinsic pontine glioma (DIPG), one of the most devastating childhood brain cancers. The team recently received its first round of spatial sequencing data from seven DIPG patient samples, which will help identify new targets for immune-based therapies.

The program's progress has been recognized nationally and internationally, with team members selected to present findings at major scientific conferences including the International Society for Pediatric Neurosurgery, the Society for Immunotherapy of Cancer, the American Society of Pediatric Neurosurgeons, and the American Association of Immunologists. Multiple grant submissions are currently under review, and a comprehensive review article summarizing current research on harnessing NK cell function in medulloblastoma is under  peer-review at the journal of Pediatrics, and several additional manuscripts are in development covering spatial genomics of DIPG, use of circulating tumor cells as a liquid biopsy to predict treatment response, and the validation of tumor organoid models. This body of work is positioning Cohen Children's at the forefront of pediatric brain cancer immunotherapy research, with the ultimate goal of translating these laboratory discoveries into safer, more effective treatments for children with brain tumors who have limited options today.