Advances in the drug clinical trials of neurofibromatosis type I-associated plexiform neurofibromas

Neurofibromatosis type Ⅰ(NF1)is an autosomal dominant genetic disease triggered by mutations of nf1gene, nf1 gene and its encoded protein product neurofibromatoprotein play important roles in tumor supressive activity. Plexiform neurofibroma was the main manifestation among some patients For plexiform neurofibroma, surgical treatment did not have satisfactory effect. meanwhile, traditional radiotherapy and chemotherapy are ineffective. All of those above serve as challenges for clinical treatment and have been received much more attention from study of multimoics and targeting therapy In this Review, the clinical features of NF1-associated plexiform neurofibromasand, the progress regarding investigation of drug targets and clinical trials for the drug of plexiform neurofibroma will be presented. Key words: Neurofibromatosis 1; Neurofibroma, Plexiform; Clinical trial; Review