Consensus Recommendations to Accelerate Clinical Trials for Neurofibromatosis Type 2

• Published in: Clinical cancer research 2009-08, Vol.15 (16), p.5032-5039
• Main Authors: EVANS, D. Gareth, KALAMARIDES, Michel, GOLFINOS, John, HARRIS, Gordon, JACOB, Abraham, KALPANA, Ganjam, KARAJANNIS, Matthias, KORF, Bruce, KURZROCK, Razelle, LAW, Meng, MCCLATCHEY, Andrea, PACKER, Roger, HUNTER-SCHAEDLE, Kim, ROEHM, Pamela, RUBENSTEIN, Allan, SLATTERY, William, TONSGARD, James H, WELLING, D. Bradley, WIDEMANN, Brigitte, YOHAY, Kaleb, GIOVANNINI, Marco, BLAKELEY, Jaishri, ALLEN, Jeffrey, BABOVIC-VUSKANOVIC, Dusica, BELZBERG, Allan, BOLLAG, Gideon, RUIHONG CHEN, DITOMASO, Emmanuelle
• Format: Article
• Language: English
• Subjects: Animals; Antineoplastic agents; Auditory Brain Stem Implants; Biological and medical sciences; clinical trials; Clinical Trials as Topic - methods; Cochlear Implants; Consensus; Drug Evaluation, Preclinical - methods; Drug Evaluation, Preclinical - trends; Health Planning Guidelines; Humans; Medical sciences; Meningeal Neoplasms - therapy; Meningioma - therapy; Neurofibromatosis 2 - diagnosis; Neurofibromatosis 2 - therapy; neurofibromatosis type 2; Neurology; Pharmacology. Drug treatments; Time Factors; Tumors of the nervous system. Phacomatoses
• ISSN: 1078-0432; 1557-3265
• DOI: 10.1158/1078-0432.CCR-08-3011

Purpose: 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. Experimental Design: 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. (Clin Cancer Res 2009;15(16):5032–9)