Target Identification and Modeling of NF1-associated Low-grade Glioma
Email Principal Investigator
Michael J. Fisher
CBTN Specimen
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
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
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
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
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
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
Olaf Witt, MD
Hopp Children's Cancer Center
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
University of Washington
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
St. Louis Children's Hospital
Institutions
Primary
Operations Center
Children’s Hospital of Philadelphia
Joined onOperations 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
Primary
UCSF Benioff Children's Hospital
Joined onThe 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
Primary
Ann & Robert H. Lurie Children’s Hospital of Chicago
Joined onAnn & 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
Hopp Children's Cancer Center
University of Washington
Satellite
Onboarding
St. Louis Children's Hospital
Joined onFounded 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.
