Brain Tumor Center

Research & Innovation

In the Brain Tumor Center, the care we provide is driven by the latest research. Our team members are leaders in pediatric brain tumor research — and our findings are helping improve our understanding of brain tumors and transform their treatment.

Explore some of our latest research

‘Challenge accepted’: Sophia takes on a brain tumor

In 2023, Sophia Mordini landed the role of a lifetime. A competitive dancer, the 12-year-old would play Clara in her company’s production of “The Nutcracker.” But the performance was bittersweet:…

Forecasting the future for childhood cancer survivors

Children are much more likely to survive cancer today than 50 years ago. Unfortunately, as adults, many of them develop cardiovascular disease, secondary cancers, or both, believed to result from the…

Pediatric high-grade gliomas: Research reveals effective targeting with avapritinib

Pediatric high-grade gliomas, particularly H3K27M diffuse midline gliomas (DMG), are aggressive malignant brain tumors with a poor prognosis. Previous research suggests that platelet-derived growth…

Keep reading about our research and innovation

Areas of active research

Atypical teratoid rhabdoid tumors
CAR T-cell therapy
Diffuse intrinsic pontine gliomas
Embryonal tumors with multilayered rosettes
Ependymomas
Hemispheric high-grade gliomas and gliomatosis cerebri
Pediatric low-grade gliomas

A Phase 1/1B Study of Autologous B7-H3 Chimeric Antigen Receptor T Cells (B7-H3 CAR T) in Children and Young Adults with Recurrent or Progressive CNS Neoplasms Expressing B7-H3 Target

Principal investigator: Susan Chi, MD
Sponsor/IND holder: Robbie Majzner, MD

CAR-T therapy is a type of cellular therapy (immunotherapy), using one’s own immune cells engineered and manufactured to specifically target tumor cells. This is a single institution study and our first CAR-T cell trial for children with recurrent CNS embryonal tumors and ependymoma. The target for this first study is B7-H3, a common antigen target found in several brain cancers. This study is to evaluate the feasibility of individual CAR-T cell manufacturing, and the safety of administering these cells via both intravenously and intraventricularly. We are also evaluating the preliminary effectiveness of this treatment approach in these brain cancers. Future CAR-T trials targeting GD2 are in development, anticipated opening in early 2027.

Learn about the trial.

NE2URON Educational Tumor Board

Co-founded by Kee Kiat Yeo, MD, member of our Brain Tumor Center, the New England NEURO-oncology Network (NE2URON) tumor board aims to enhance pediatric neuro-oncology education for New England-area fellows. The board involves quarterly hour-long webinars that feature case-based discussions and expert reviews on a range of topics, including the diagnosis and management of pediatric neuro-oncologic tumors. Follow us on LinkedIn to catch details of upcoming webinars.

Clinical trials

The Brain Tumor Center is a member of several major clinical trial consortia. These include the Children’s Oncology Group (COG), the Pediatric Neuro-Oncology Consortium (PNOC), the CONNECT Consortium, and the Pediatric Brain Tumor Consortium (PBTC). This means that we can provide our patients with access to a wide range of clinical trials.

Selected publications

Cellular hierarchies of embryonal tumors with multilayered rosettes are shaped by oncogenic microRNAs and receptor-ligand interactions: https://pubmed.ncbi.nlm.nih.gov/40419763/
Dissecting the immune landscape in pediatric high-grade glioma reveals cell state changes under therapeutic pressure: https://pubmed.ncbi.nlm.nih.gov/40315846/
Neuroimmune-competent human brain organoid model of diffuse midline glioma: https://pubmed.ncbi.nlm.nih.gov/39561098/
Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration: https://pubmed.ncbi.nlm.nih.gov/39854822/
SMARCB1 missense mutants disrupt SWI/SNF complex stability and remodeling activity: https://pubmed.ncbi.nlm.nih.gov/40196006/
Treatment Approach for Metastatic Intracranial Germinoma: A Multi-Institutional Experience: https://pubmed.ncbi.nlm.nih.gov/40038880/
A diverse landscape of FGFR alterations andco-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas: https://pubmed.ncbi.nlm.nih.gov/40744913/
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib: https://pubmed.ncbi.nlm.nih.gov/40086436/
H3K27-altered diffuse midline gliomas with MAPK pathway alterations: Prognostic and therapeutic implications: https://pubmed.ncbi.nlm.nih.gov/38037182/