Therapeutic Development in Neurofibromatosis

Abstract

Although neurofibromatosis (NF) was initially recognized in the nine- teenth century, only in the past two decades we have witnessed a paradigm shift in therapeutics. This progress is driven by the increasing understanding of the natural history of the NF-associated tumors and understanding of the molecular landscape of these disorders. Multiple clinical trials have been launched evalu- ating non-surgical treatment modalities and more studies are in the pipeline. Recently, the NF community has adopted standardized endpoints recommended by the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration established in 2011. Such collaborations among academic, regulatory and supporting communities are crucial for providing the infrastructure needed for advancing the therapeutic development in the field of NF Keywords: neurofibromatosis type I, neurofibromatosis type II, chemotherapy, radiotherapy, therapeutics, clinical trials, targeted therapy 1.