Consensus recommendations to accelerate clinical trials for neurofibromatosis type 2.
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.
Evans
D Gareth
DG
St. Mary's Hospital, Manchester, United Kingdom.
Kalamarides
Michel
M
Hopital Beaujon, APHP, Clichy, France.
Hunter-Schaedle
Kim
K
Children's Tumor Foundation.
Blakeley
Jaishri
J
Johns Hopkins University, Baltimore, Maryland.
Allen
Jeffrey
J
New York University.
Babovic-Vuskanovic
Dusica
D
Mayo Clinic, Rochester, Minnesota.
Belzberg
Allan
A
Johns Hopkins University, Baltimore, Maryland.
Bollag
Gideon
G
Plexxikon, Inc., Berkeley, California.
Chen
Ruihong
R
Nexgenix Pharmaceuticals.
DiTomaso
Emmanuelle
E
Harvard Medical School/Massachusetts General Hospital, Boston, Massachusetts.
Golfinos
John
J
New York University.
Harris
Gordon
G
Harvard Medical School/Massachusetts General Hospital, Boston, Massachusetts.
Jacob
Abraham
A
Ohio State University, Columbus, Ohio.
Kalpana
Ganjam
G
Albert Einstein College of Medicine, Bronx, New York.
Karajannis
Matthias
M
New York University.
Korf
Bruce
B
University of Alabama, Birmingham, Alabama.
Kurzrock
Razelle
R
M. D. Anderson Cancer Center, Houston, Texas.
Law
Meng
M
Mount Sinai School of Medicine.
McClatchey
Andrea
A
Harvard Medical School/Massachusetts General Hospital, Boston, Massachusetts.
Packer
Roger
R
Children's National Medical Center, Washington, District of Columbia.
Roehm
Pamela
P
New York University.
Rubenstein
Allan
A
Nexgenix Pharmaceuticals.
Slattery
William
W
House Ear Institute, Los Angeles, California.
Tonsgard
James H
JH
University of Chicago Pritzker School of Medicine, Chicago, Illinois.
Welling
D Bradley
DB
Ohio State University, Columbus, Ohio.
Widemann
Brigitte
B
National Cancer Institute, Bethesda, Maryland.
Yohay
Kaleb
K
Cornell University Medical Center, New York, New York.
Giovannini
Marco
M
House Ear Institute, Los Angeles, California.
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