Jaishri O Blakeley1, Pierre Wolkenstein2, Brigitte C Widemann2, James Lee2, Lu Q Le2, Rhonda Jackson2, Marigo Stathis2, Sharad K Verma2. 1. From the Department of Neurology (J.O.B., R.J., M.S., S.K.V.), The Johns Hopkins School of Medicine, The Neurofibromatosis Therapeutic Acceleration Program, Baltimore, MD; Department of Dermatology (P.W.), Paris Est Créteil University, France; Pediatric Oncology Branch (B.C.W.), National Cancer Institute, NIH, Bethesda, MD; Dermavant Sciences (J.L.), Durham, NC; and Department of Dermatology (L.Q.L.), UT Southwestern Medical Center, Dallas, TX. jblakel3@jhmi.edu. 2. From the Department of Neurology (J.O.B., R.J., M.S., S.K.V.), The Johns Hopkins School of Medicine, The Neurofibromatosis Therapeutic Acceleration Program, Baltimore, MD; Department of Dermatology (P.W.), Paris Est Créteil University, France; Pediatric Oncology Branch (B.C.W.), National Cancer Institute, NIH, Bethesda, MD; Dermavant Sciences (J.L.), Durham, NC; and Department of Dermatology (L.Q.L.), UT Southwestern Medical Center, Dallas, TX.
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 Teams met monthly to review published and unpublished materials, and created summaries about the material known and unknown that may influence therapeutic development for cNF.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.
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.
Authors: Lisa Brauer Oliveira; Mauro Geller; Karin Soares Cunha; Alessandra Santos; Allan Bernacchi; Allan E Rubenstein; Sanyu Takirambudde; Spyros Mezitis; Carolina de Almeida Ito Brum; Luiz Guilherme Darrigo; Marcia Gonçalves Ribeiro Journal: Heliyon Date: 2021-03-17
Authors: Robert Galvin; Adrienne L Watson; David A Largaespada; Nancy Ratner; Sara Osum; Christopher L Moertel Journal: Curr Oncol Rep Date: 2021-03-15 Impact factor: 5.075