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Prevention and Treatment of Neurofibromatosis Type 1-Associated Malignant Peripheral Nerve Sheath Tumors

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Technical Report,01 Apr 2014,31 Mar 2018

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Columbia University New York United States

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The most common cause of death in Neurofibromatosis Type 1 NF1 patients is malignant peripheral nerve sheath tumor MPNST. MPNSTs are aggressive Schwann cell-derived neoplasms that typically arise from precursor lesions such as plexiform neurofibromas. Although gross total resection of MPNSTs is potentially curative, this occurs in only a small minority of cases. Radiotherapy and chemotherapy have almost no effect on patient mortality. NF1 patients have an approximate 10 lifetime risk of developing an MPNST. Thus, development of safe and effective MPNST preventative therapies could have an important impact on NF1 patient morbidity and mortality. In this grant, we are testing the hypothesis that chronic administration of agents that promote apoptosis andor inhibit pro-survival autophagy will inhibit MPNST formation and progression in transgenic mouse models of MPNST. Specifically, we are examining the mechanisms of action and in vivo utility of two classes of drugs, BH3 mimetics and lysosomotropic agents, on MPNSTs. The drugs that we are testing are approved for human use and could be rapidly advanced into human MPNST clinical trials if our pre- clinical testing yields positive results.

Subject Categories:

  • Anatomy and Physiology
  • Medicine and Medical Research

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