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

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.