Creating a comprehensive research strategy for cutaneous neurofibromas

Jaishri Blakeley, Pierre Wolkenstein, Brigitte C. Widemann, James Lee, Lu Q. Le, Rhonda Jackson, Marigo Stathis, Sharad Verma

Research output: Contribution to journalArticle

Abstract

OBJECTIVE: Outside of procedural-based methods, there are currently no established medical treatments for cutaneous neurofibroma (cNF), which afflict up to 99% of patients with NF1. Further, adult patients often report cNF are the greatest burden of living with NF1. The Neurofibromatosis Therapeutic Acceleration Program (NTAP) launched a think tank to address core questions to facilitate development of effective therapeutics for cNF in people with NF1. METHODS: Experts (with and without explicit experience with NF1 or cNF) from multiple scientific and medical disciplines, representing the ranks of academia, industry, and government agencies, were invited to become a member of a team addressing a specific subset of questions pertinent to cNF. Teams met monthly to review published and unpublished materials, and created summaries about the material known and unknown that may influence therapeutic development for cNF. Teams prioritized questions and organized supporting data, which was presented to the entire body of experts by each team at a research summit. RESULTS: Four themes were identified as being relevant to creating a comprehensive research strategy for cNF: (1) establishing definitions of cNF, (2) determining the biology of cNF with respect to tumor initiation, progression, and maintenance, (3) outlining the factors that guide therapies development, and (4) defining core considerations for clinical trials design and optimization for cNF. CONCLUSION: Considerations and key questions for each of the thematic areas were identified and provided basis for a request for applications launched by NTAP focused on cNF and are described in the accompanying articles of this supplement.

Original languageEnglish (US)
JournalNeurology
Volume91
Issue number2
DOIs
StatePublished - Jul 10 2018

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Neurofibroma
Skin
Research
Neurofibromatoses
Therapeutics
Government Agencies
Industry
Maintenance
Clinical Trials

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Creating a comprehensive research strategy for cutaneous neurofibromas. / Blakeley, Jaishri; Wolkenstein, Pierre; Widemann, Brigitte C.; Lee, James; Le, Lu Q.; Jackson, Rhonda; Stathis, Marigo; Verma, Sharad.

In: Neurology, Vol. 91, No. 2, 10.07.2018.

Research output: Contribution to journalArticle

Blakeley, J, Wolkenstein, P, Widemann, BC, Lee, J, Le, LQ, Jackson, R, Stathis, M & Verma, S 2018, 'Creating a comprehensive research strategy for cutaneous neurofibromas', Neurology, vol. 91, no. 2. https://doi.org/10.1212/WNL.0000000000005789
Blakeley, Jaishri ; Wolkenstein, Pierre ; Widemann, Brigitte C. ; Lee, James ; Le, Lu Q. ; Jackson, Rhonda ; Stathis, Marigo ; Verma, Sharad. / Creating a comprehensive research strategy for cutaneous neurofibromas. In: Neurology. 2018 ; Vol. 91, No. 2.
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