Optimizing biologically targeted clinical trials for neurofibromatosis

David H. Gutmann, Jaishri O. Blakeley, Bruce R. Korf, Roger J. Packer

Research output: Contribution to journalReview articlepeer-review

Abstract

Introduction: The neurofibromatoses (neurofibromatosis type 1, NF1 and neurofibromatosis type 2, NF2) comprise the most common inherited conditions in which affected children and adults develop tumors of the central and peripheral nervous system. In this review, the authors discuss how the establishment of the Neurofibromatosis Clinical Trials Consortium (NFCTC) has positively impacted on the design and execution of treatment studies for individuals with NF1 and NF2. Areas covered: Using an extensive PUBMED search in collaboration with select NFCTC members expert in distinct NF topics, the authors discuss the clinical features of NF1 and NF2, the molecular biology of the NF1 and NF2 genes, the development and application of clinically relevant Nf1 and Nf2 genetically engineered mouse models and the formation of the NFCTC to enable efficient clinical trial design and execution. Expert opinion: The NFCTC has resulted in a more seamless integration of mouse preclinical and human clinical trials efforts. Leveraging emerging enabling resources, current research is focused on identifying subtypes of tumors in NF1 and NF2 to deliver the most active compounds to the patients most likely to respond to the targeted therapy.

Original languageEnglish (US)
Pages (from-to)443-462
Number of pages20
JournalExpert Opinion on Investigational Drugs
Volume22
Issue number4
DOIs
StatePublished - Apr 2013

Keywords

  • Clinical trials
  • Mouse models
  • NF1
  • NF2
  • Preclinical

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

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