title: Consensus recommendations to accelerate clinical trials for neurofibromatosis type 2.
abstract: OBJECTIVE
  Neurofibromatosis type 2 (NF2) is a rare autosomal dominant disorder associated primarily with bilateral schwannomas seen on the superior vestibular branches of the eighth cranial nerves. Significant morbidity can result from surgical treatment of these tumors. Meningiomas, ependymomas, and other benign central nervous system tumors are also common in NF2. The lack of effective treatments for NF2 marks an unmet medical need.
  
  
  METHODS
  Here, we provide recommendations from a workshop, cochaired by Drs. D. Gareth Evans and Marco Giovannini, of 36 international researchers, physicians, representatives of the biotechnology industry, and patient advocates on how to accelerate progress toward NF2 clinical trials.
  
  
  RESULTS
  Workshop participants reached a consensus that, based on current knowledge, the time is right to plan and implement NF2 clinical trials. Obstacles impeding NF2 clinical trials and how to address them were discussed, as well as the candidate therapeutic pipeline for NF2.
  
  
  CONCLUSIONS
  Both phase 0 and phase II NF2 trials are near-term options for NF2 clinical trials. The number of NF2 patients in the population remains limited, and successful recruitment will require ongoing collaboration efforts between NF2 clinics.
authors: St. Mary's Hospital, Manchester, United Kingdom.
authors: D Gareth
authors: DG
authors: Evans
authors: Hopital Beaujon, APHP, Clichy, France.
authors: Michel
authors: M
authors: Kalamarides
authors: Children's Tumor Foundation.
authors: Kim
authors: K
authors: Hunter-Schaedle
authors: Johns Hopkins University, Baltimore, Maryland.
authors: Jaishri
authors: J
authors: Blakeley
authors: New York University.
authors: Jeffrey
authors: J
authors: Allen
authors: Mayo Clinic, Rochester, Minnesota.
authors: Dusica
authors: D
authors: Babovic-Vuskanovic
authors: Johns Hopkins University, Baltimore, Maryland.
authors: Allan
authors: A
authors: Belzberg
authors: Plexxikon, Inc., Berkeley, California.
authors: Gideon
authors: G
authors: Bollag
authors: Nexgenix Pharmaceuticals.
authors: Ruihong
authors: R
authors: Chen
authors: Harvard Medical School/Massachusetts General Hospital, Boston, Massachusetts.
authors: Emmanuelle
authors: E
authors: DiTomaso
authors: New York University.
authors: John
authors: J
authors: Golfinos
authors: Harvard Medical School/Massachusetts General Hospital, Boston, Massachusetts.
authors: Gordon
authors: G
authors: Harris
authors: Ohio State University, Columbus, Ohio.
authors: Abraham
authors: A
authors: Jacob
authors: Albert Einstein College of Medicine, Bronx, New York.
authors: Ganjam
authors: G
authors: Kalpana
authors: New York University.
authors: Matthias
authors: M
authors: Karajannis
authors: University of Alabama, Birmingham, Alabama.
authors: Bruce
authors: B
authors: Korf
authors: M. D. Anderson Cancer Center, Houston, Texas.
authors: Razelle
authors: R
authors: Kurzrock
authors: Mount Sinai School of Medicine.
authors: Meng
authors: M
authors: Law
authors: Harvard Medical School/Massachusetts General Hospital, Boston, Massachusetts.
authors: Andrea
authors: A
authors: McClatchey
authors: Children's National Medical Center, Washington, District of Columbia.
authors: Roger
authors: R
authors: Packer
authors: New York University.
authors: Pamela
authors: P
authors: Roehm
authors: Nexgenix Pharmaceuticals.
authors: Allan
authors: A
authors: Rubenstein
authors: House Ear Institute, Los Angeles, California.
authors: William
authors: W
authors: Slattery
authors: University of Chicago Pritzker School of Medicine, Chicago, Illinois.
authors: James H
authors: JH
authors: Tonsgard
authors: Ohio State University, Columbus, Ohio.
authors: D Bradley
authors: DB
authors: Welling
authors: National Cancer Institute, Bethesda, Maryland.
authors: Brigitte
authors: B
authors: Widemann
authors: Cornell University Medical Center, New York, New York.
authors: Kaleb
authors: K
authors: Yohay
authors: House Ear Institute, Los Angeles, California.
authors: Marco
authors: M
authors: Giovannini
mesh_terms: D000818:Animals; D038262:Auditory Brain Stem Implants; D002986:Clinical Trials as Topic / Q000379:methods*; D003054:Cochlear Implants; D032921:Consensus; D004353:Drug Evaluation, Preclinical / Q000379:methods / Q000639:trends; D006287:Health Planning Guidelines; D006801:Humans; D008577:Meningeal Neoplasms / Q000628:therapy; D008579:Meningioma / Q000628:therapy; D016518:Neurofibromatosis 2 / Q000175:diagnosis / Q000628:therapy*; D013997:Time Factors