Neurotherapeutics

, Volume 14, Issue 2, pp 298–306 | Cite as

Cancer of the Peripheral Nerve in Neurofibromatosis Type 1

  • Verena Staedtke
  • Ren-Yuan Bai
  • Jaishri O’Neill Blakeley
Review

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.

Keywords

Malignant peripheral nerve sheath tumor Malignant transformation Sarcoma Neurofibromatosis Treatment Chemoprevention 

Supplementary material

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Copyright information

© The American Society for Experimental NeuroTherapeutics, Inc. 2017

Authors and Affiliations

  • Verena Staedtke
    • 1
    • 2
  • Ren-Yuan Bai
    • 3
  • Jaishri O’Neill Blakeley
    • 1
    • 2
    • 3
  1. 1.Department of NeurologyJohns Hopkins Medical InstitutionsBaltimoreUSA
  2. 2.Department of OncologyJohns Hopkins Medical InstitutionsBaltimoreUSA
  3. 3.Department of NeurosurgeryJohns Hopkins Medical InstitutionsBaltimoreUSA

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