Abstract
Purpose of review:
Over the past decade, substantial insight into the biological function of the tumor suppressors neurofibromin (NF1) and Merlin (NF2) has been gained. The purpose of this review is to highlight some of the major advances in our understanding of the biology of neurofibromatosis type 1 (NF1) and neurofibromatosis type 2 (NF2) as they relate to the development of novel therapies for these disorders.
Recent findings:
The development of increasingly sophisticated preclinical models over the recent years has provided the platform from which to rationally develop molecular targeted therapies for both NF1 and NF2-related tumors, such as within the Department of Defense-sponsored Neurofibromatosis Clinical Trials Consortium.
Summary:
Clinical trials with molecular-targeted therapies have become a reality for neurofibromatosis patients, and hold substantial promise for improving the morbidity and mortality of individuals affected with these disorders.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Antineoplastic Agents / therapeutic use
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Child
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Child, Preschool
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Clinical Trials as Topic
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Gene Expression Regulation, Neoplastic
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Genetic Predisposition to Disease
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Humans
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Infant
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Magnetic Resonance Imaging
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Molecular Targeted Therapy / trends
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Nerve Sheath Neoplasms / genetics
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Nerve Sheath Neoplasms / pathology*
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Nerve Sheath Neoplasms / therapy
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Neurofibromatosis 1 / genetics
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Neurofibromatosis 1 / pathology*
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Neurofibromatosis 1 / therapy
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Neurofibromatosis 2 / genetics
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Neurofibromatosis 2 / pathology*
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Neurofibromatosis 2 / therapy
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Neurofibromin 1 / genetics
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Neurofibromin 1 / metabolism*
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Neurofibromin 2 / genetics
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Peripheral Nervous System Neoplasms / genetics
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Peripheral Nervous System Neoplasms / pathology*
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Peripheral Nervous System Neoplasms / therapy
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Skin Neoplasms / genetics
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Skin Neoplasms / pathology*
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Skin Neoplasms / therapy
Substances
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Antineoplastic Agents
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Neurofibromin 1
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Neurofibromin 2