NF1 and central nervous system cancer: To date, effective drugs targeting downstream molecules involved in this pathway are limited to BRAF-inhibitors in BRAF-mutated gliomas.38 However, preclinical and clinical studies have shown efficacy of MEK-inhibitors in NF1-altered GBMs but not in NF1 wildtype tumors.39–44 Accordingly, NF1 alteration may serve as a predictive factor for therapies targeting the RAS-RAF-MAPK pathway, such as MEK-inhibitors.