Receptor Tyrosine Kinases as Targets for Treatment of Peripheral Nerve Sheath Tumors in NF 1 Patients

摘要

Plexiform neurofibromas (PNF) are a major manifestation of NF1 and develop in 30-50% of the patients. In contrast to cutaneous neurofibroma, which are the hallmark of NF1 but of mainly cosmetic significance, PNF cause various clinical deficits and have a high risk of malignant transformation into malignant peripheral nerve sheath tumours (MPNST) which is the main mortality factor for these patients. No effective medical therapy is available for either PNF or MPNST. This study is aimed to establish in vitro and in vivo models for the NF1-associated benign and malignant tumours and use them to test efficacy of tyrosine kinase receptor inhibitors.