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Updated: Feb 16, 2026
03:53
An Orthotopic Sciatic Nerve Xenograft for Neurofibromatosis Type 1 Neurofibromas
Published on: October 10, 2025
540
Neurofibromatosis Clinical Trial Consortium
Roger J Packer1,2,3, Michael J Fisher4, Gary Cutter5
11 From the Center for Neuroscience and Behavioral Medicine, Children's National Health System, Washington, DC, USA.
Journal of Child Neurology
|December 17, 2017
Summary
The Neurofibromatosis Clinical Trial Consortium (NFCTC) advances novel therapies for neurofibromatosis types 1 and 2. Established by the Department of Defense, it expedites clinical trials for rare genetic disorders.
Area of Science:
- Genetics
- Oncology
- Clinical Trials
Background:
- Neurofibromatosis types 1 and 2 (NF1 and NF2) are genetic disorders causing severe complications in children and adults.
- Advances in understanding the genetic basis of NF1 and NF2 have spurred the development of targeted therapies.
- The Neurofibromatosis Clinical Trial Consortium (NFCTC) was founded in 2006 to accelerate clinical trial development.
Purpose of the Study:
- To summarize the achievements and expansion of the NFCTC over its first decade.
- To highlight the successful completion of clinical studies for various NF1 and NF2-related conditions.
- To outline the NFCTC's strategy for translating preclinical research into clinical applications.
Main Methods:
- The NFCTC has conducted clinical trials for pediatric and adult patients with NF1 and NF2.
- Studies focused on plexiform neurofibromas, neurocognitive deficits, low-grade gliomas, and malignant peripheral nerve sheath tumors in NF1.
- A clinical trial for acoustic schwannomas in NF2 is nearing completion.
Main Results:
- The NFCTC has successfully completed multiple clinical studies for NF1 and NF2 patients.
- The consortium has expanded to 19 sites across the United States and Australia.
- Established collaborations facilitate the rapid translation of research findings into clinical practice.
Conclusions:
- The NFCTC has demonstrated significant success in advancing therapeutic development for neurofibromatosis.
- The consortium's growth and collaborative approach position it to accelerate future treatment innovations.
- Continued efforts are focused on translating preclinical discoveries into effective clinical treatments for NF1 and NF2.
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