Brain Tumor Initiative

GFF’s Brain Tumor Initiative (BTI) focuses on identifying and developing therapeutic strategies to effectively treat neurofibromatosis type 1 (NF1)-associated transformed gliomas. Gliomas are the most common primary central nervous system (CNS) tumors in  NF1 patients, usually involving optic pathways in children. BTI is focused the non-optic pathway gliomas. While most of NF1 gliomas are pilocytic astrocytomas, NF1 patients may also develop higher grade tumors, WHO-Grade II-IV,  that  harbor  additional  gene mutations. In each case, it is often unknown whether the NF1  high-grade  glioma is  sporadic or transformed from a previously existing low-grade glioma.

BTI is conducting clinical studies to provide critical characterization of NF1 high grade glioma and identify the most promising treatments for patients available today. BTI also invests in preclinical research tools to help drug discovery and development. The data and insight generated from these clinical studies and preclinical models will inform the development of new, more effective targeted therapies in the future.

Development Pipeline for Funded Projects

Creating NF1/CDKN2A/ATRX Models for NF1 Associated High Grade Glioma

hiPSC Modeling of NF1 High-Grade Glioma

Natural History of NF1-Associated Non-Optic Pathway Glioma

Clinical Trial Phase 1

Retrospective Study of the Clinical Features and Outcomes of Children and Adults with NF1-Associated High Grade Glioma and Adults with NF1-Associated Low Grade Glioma

Clinical Trial Phase 1

Targeting NF1 Mutant Glioma Oncoproteins with Peptide Centric CAR T Cells

BTI Dream Team

Dr. Thomas De Raedt

Children's Hospital of Philadelphia

David Gutmann, MD, PhD

Washington University in St. Louis

Dr. Chelsea Kotch

Children's Hospital of Philadelphia

Dr. John Maris

Children's Hospital of Philadelphia

Dr. Anna Piotrowski

Children's Hospital of Philadelphia

Dr. Thomas De Raedt

Children's Hospital of Philadelphia

David Gutmann, MD, PhD

Washington University in St. Louis

Dr. Chelsea Kotch

Children's Hospital of Philadelphia

Dr. John Maris

Children's Hospital of Philadelphia

Dr. Anna Piotrowski

Children's Hospital of Philadelphia