Novel Platforms for Developing Therapies for Transformed Gliomas in Patients with NF1

Neurofibromatosis type 1 (NF1) is a disorder affecting more than 100,000 people each year in the United States and is therefore the most common genetic tumor predisposition syndrome. NF1 can result in low grade gliomas (LGG) and less commonly high grade glioma (HGG). While some HGGs seem to arise spontaneously, there is evidence that some HGGs in patients with NF1 arise from LGGs. However, the genomic changes that take place during this transition are not well known. Researchers on this project aim to identify such genomic changes and to develop pre-clinical models. Pre-clinical models are used to study disease progression and drug interactions before therapeutics are administered to real patients. Development of strong models, especially those containing the genomic alterations associated with NF1 will allow for discovery of new therapies in the treatment of LGGs and HGGs. This team will use specimens prepared and provided by the Children’s Brain Tumor Network to pursue this research. This study will allow for the development of pre-clinical models that can be used to identify novel therapies for transformed NF1 gliomas.