title: Cancer of the Peripheral Nerve in Neurofibromatosis Type 1.
abstract: The RASopathy neurofibromatosis 1 is an autosomal dominant hereditary cancer syndrome that represents a major risk for the development of malignancies, particularly malignant peripheral nerve sheath tumors (MPNSTs). MPNSTs are unique sarcomas that originate from the peripheral nerve and represent the only primary cancer of the peripheral nervous system. To date, surgery is the only treatment modality proven to have survival benefit for MPNSTs and even when maximal surgery is feasible, these tumors are rarely curable, despite the use of chemotherapy and radiation. In this review, we discuss the current state-of-the-art treatments for MPNSTs, latest therapeutic developments, and critical aspects of the underlying molecular and pathophysiology that appear promising for therapeutic developments in the future. In particular, we discuss the specific elements of cancer in the peripheral nerve and how that may impel development of unique therapies for this form of sarcoma.
authors.affiliation: Department of Neurology, Johns Hopkins Medical Institutions, Baltimore, MD, USA.|Department of Oncology, Johns Hopkins Medical Institutions, Baltimore, MD, USA.
authors.forename: Verena
authors.initials: V
authors.lastname: Staedtke
authors.affiliation: Department of Neurosurgery, Johns Hopkins Medical Institutions, Baltimore, MD, USA.
authors.forename: Ren-Yuan
authors.initials: RY
authors.lastname: Bai
authors.affiliation: Department of Neurology, Johns Hopkins Medical Institutions, Baltimore, MD, USA. jblakel3@jhmi.edu.|Department of Oncology, Johns Hopkins Medical Institutions, Baltimore, MD, USA. jblakel3@jhmi.edu.|Department of Neurosurgery, Johns Hopkins Medical Institutions, Baltimore, MD, USA. jblakel3@jhmi.edu.
authors.forename: Jaishri O'Neill
authors.initials: JO
authors.lastname: Blakeley
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D006801:Humans; D009442:Neurilemmoma / Q000150:complications / Q000235:genetics* / Q000628:therapy*; D009456:Neurofibromatosis 1 / Q000150:complications*; D010524:Peripheral Nervous System Neoplasms / Q000150:complications / Q000235:genetics* / Q000628:therapy*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents
keywords: Chemoprevention; Malignant peripheral nerve sheath tumor; Malignant transformation; Neurofibromatosis; Sarcoma; Treatment