Pharmacological Approaches in Neurofibromatosis Type 1-Associated Nervous System Tumors

SIMPLE SUMMARY: Neurofibromatosis type 1 (NF1) is a common cancer predisposition genetic disease that is associated with significant morbidity and mortality. In this literature review, we discuss the major pathways in the nervous system that are affected by NF1, tumors that are associated with NF1, drugs that target these pathways, and genetic models of NF1. We also summarize the latest updates from clinical trials that are evaluating pharmacological agents to treat these tumors and discuss the efforts that are being made to cure the disease in the future ABSTRACT: Neurofibromatosis type 1 is an autosomal dominant genetic disease and a common tumor predisposition syndrome that affects 1 in 3000 to 4000 patients in the USA. Although studies have been conducted to better understand and manage this disease, the underlying pathogenesis of neurofibromatosis type 1 has not been completely elucidated, and this disease is still associated with significant morbidity and mortality. Treatment options are limited to surgery with chemotherapy for tumors in cases of malignant transformation. In this review, we summarize the advances in the development of targeted pharmacological interventions for neurofibromatosis type 1 and related conditions.