In summary, this study lays an important foundation for the effective and feasible chemoprevention of malignancies in patients with NF1, which has the potential to delay or perhaps even prevent the malignant transformation of MPNST and other NF1-related malignancies, decrease the need for surgical intervention and reduce the use of antineoplastic therapies in this patient population. The gene discussed is NF1; the disease is malignant peripheral nerve sheath tumor.