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

Email Principal Investigator
Completed
Specimen
LGG
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Michael J. Fisher

Children’s Hospital of Philadelphia
Philadelphia, PA, USA

CBTN Specimen

15

CBTN Participants

Backer

Children's Tumor Foundation

Completed

About this

Project

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

Scientists

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

Team

William A. Weiss

David T. Jones

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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

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Children’s Hospital of Philadelphia

Philadelphia, PA, USA
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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

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Children’s Hospital of Philadelphia

Philadelphia, PA, USA
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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

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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

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

Chicago, Illinois, USA
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Olaf Witt, MD

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Hopp Children's Cancer Center

Heidelberg, Germany
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Eric Holland, MD, PhD

Dr. Eric Holland is a world-renowned physician-scientist who combines compassionate patient care with exacting laboratory research to discover more effective treatments for brain tumors. As a neurosurgeon, Dr. Holland specializes in caring for patients with glioblastomas, aggressive cancers that are

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University of Washington

1959 NE Pacific St, Seattle, WA 98195
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David H. Gutmann, MD, PhD

Dr. Gutmann is a Washington University neurologist who directs the St. Louis Children's Hospital Neurofibromatosis Program. He is an internationally-recognized expert in neurofibromatosis, and was a member of the research group that originally identified the entire NF1 gene. Dr. Gutmann is the Donal

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St. Louis Children's Hospital

St. Louis, Missouri, USA

Institutions

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Primary

Operations Center

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Children’s Hospital of Philadelphia

Joined on

Operations Center for the Children’s Brain Tumor Tissue Consortium, the Children’s Hospital of Philadelphia (CHOP) is currently ranked 1st nationally for their Pediatric Cancer Program by U.S. News & World Report. CHOP’s Biobank is home to the CBTTC’s pediatric brain and CNS tumor biorepository; the

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Primary

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UCSF Benioff Children's Hospital

Joined on

The Pediatric Brain Tumor Center at UCSF Benioff Children’s Hospital, with campuses in San Francisco and Oakland, harnesses the power of world-class experts from all disciplines related to child brain health, including neurology, neurosurgery, neurocritical care, neurogenetics, neuro-oncology, rehab

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Primary

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

Joined on

Ann & Robert H. Lurie Children’s Hospital of Chicago treats 220 new patients each year with pediatric cancers. The hospital is ranked 17th in the nation for their pediatric oncology program and 11th for their neurology and neurosurgery program by U.S. News & World Report.Established in 1986, the Lur

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Hopp Children's Cancer Center

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University of Washington

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Satellite

Onboarding

St. Louis Children's Hospital

Joined on

Founded in 1879, St. Louis Children’s Hospital (SLCH) is at the forefront of pediatric medicine, providing care for children around the world. As the pediatric teaching hospital for Washington University School of Medicine (WUSM), SLCH offers nationally recognized programs for training and research.