Neurofibromatosis-related tumors: emerging biology and therapies. Issue 1 (February 2015)

• Record Type: Journal Article
• Authors: Karajannis, Matthias A.; Ferner, Rosalie E.
• Is Part Of: Current opinion in pediatrics. Volume 27:Issue 1(2015:Feb.)
• Publication Date: 2015-02
• Subjects: epidermal growth factor receptor -- malignant peripheral nerve sheath tumor -- mammalian target of rapamycin -- meningioma -- mitogen-activated protein kinase -- neurofibromatosis type 1 -- neurofibromatosis type 2 -- optic pathway glioma -- plexiform neurofibroma -- schwannoma; Pediatrics -- Periodicals; Pediatrics -- Periodicals -- Bibliography -- Periodicals; 618.92
• DOI: 10.1097/MOP.0000000000000169
• Languages: English
• ISSNs: 1040-8703

Abstract

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.