Development of targeted therapies for neurofibromatosis type 1 (NF1) related tumors

Neurofibromatosis Type 1 (NF1), previously referred to as von Recklinghausen disease, is a relatively common (1:2,500 to 1:3,000) autosomal dominant, progressive tumor predisposition syndrome characterized by manifestations in many organ systems including neurocutaneous findings and the propensity to develop tumors of the peripheral and central nervous system (Friedman 2002; Korf 2002; Ferner 2007). The natural history of NF1 is poorly understood, and for most NF1 related tumor manifestations the only standard treatment option is surgery (Korf 2001; Ferner et al. 2007). Increasing knowledge of molecular and biologic pathways implied in the development of NF1 related tumors has resulted in the development of treatment trials with targeted agents (Packer et al. 2002).