Target Identification and Modeling of NF1-associated Low-grade Glioma

Email Principal Investigator
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Michael J. Fisher

Children’s Hospital of Philadelphia
Philadelphia, PA, USA

CBTN Specimen


CBTN Participants


Children's Tumor Foundation


About this


Neurofibromatosis type 1 (NF1) is a genetic disorder that affects multiple systems in the body. Low grade gliomas (LGG) develop in ~20% of children with NF1, occurring most commonly within the optic pathway (optic pathway glioma; OPG). The identification of prognostic factors to identify tumors that are likely to require intervention is crucial in minimizing the fatality of these tumors. Prognostic factors are measurements or markers that can be used to estimate the chance of recovery from disease or the chance of that disease recurring. The current standard of care therapies for symptomatic NF1-LGG involve the use of drugs such as carboplatin, vincristine, and vinblastine. These drugs can also damage dividing cells in zones critical for normal brain development in children. Because of this fact, there is a pressing need to identify molecular risk factors and treatments targeted to NF1-LGG. Progress in this area has been hampered by the rarity of samples usable for this work. Fortunately, the Children’s Brain Tumor Network will provide researchers with the high-quality tumor specimens needed to complete this line of research. In order to accelerate progress this project has a team of researchers with broad expertise applicable to the study of NF1-LGG.

Ask The


Ask the scientists

What are the goals of this project?

Researchers seek to identify risk factors, biomarkers and targets associated with NF1-LGGs as well as identifying possible therapeutic targets.

What is the impact of this project?

This research will accelerate the understanding of NF1-LGGs and possibly lead to advancements in diagnostics, prognostics, and treatment of this disease.

Why is the CBTN request important to this project?

Access to rare, high quality specimens provided by the Children’s Brain Tumor Network allows researchers to carry out this research.

Specimen Data

The Children's Brain Tumor Network contributed to this project by providing germline and tumor DNA and tumor RNA.

Meet The


David H. Gutmann

Stefan Pfister

Eric Holland

William A. Weiss

Olaf Witt

David T. Jones


Executive Board

Michael J. Fisher, MD

Michael J. Fisher, MD, is Chief of the Section of Neuro-Oncology, Director of the Neuro-Oncology Fellowship Program and Director of the Neurofibromatosis Program at Children's Hospital of Philadelphia. He has a special interest in brain tumors and neurofibromatosis.Areas of Expertise: Neurofibromato


Children’s Hospital of Philadelphia

Philadelphia, PA, USA

Scientific Committee

Executive Board

Scientific co-Chair

Principal Investigator

Adam Resnick, PhD

Adam Resnick is the Director of Data Driven Discovery in Biomedicine (D3b) at Children’s Hospital of Philadelphia (CHOP) responsible for leading a multidisciplinary team to build and support a scalable, patient-focused healthcare and educational discovery ecosystem on behalf of all children. He is a


Children’s Hospital of Philadelphia

Philadelphia, PA, USA

Scientific Committee

Executive Board

Principal Investigator

Joanna Phillips, MD, PhD

I am an Associate Professor in the UCSF Departments of Neurological Surgery and Pathology, Director of the UCSF Brain Tumor Research Center Tissue Biorepository and Histology Core, Co-Director of the UCSF Neuropathology BTRC Biomarkers Laboratory, Co-Director of the UCSF Brain Tumor SPORE Bioreposit

UCSF Benioff Children's Hospital.jpg

UCSF Benioff Children's Hospital

San Francisco and Oakland, CA, USA
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Scientific Committee

Executive Board

Executive co-Chair

Principal Investigator

Angela Waanders, MD, MPH

Specialties:Hematology, Oncology, Neuro-Oncology & Stem Cell TransplantationEducation: Tulane University School of Medicine, 2003Postgraduate Training: Fellowship in pediatric hematology/oncology, Children's Hospital of Philadelphia, 2006-2009; Residency is pediatrics, Children's Hospital of Philade


Ann & Robert H. Lurie Children’s Hospital of Chicago

Chicago, Illinois, USA