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LB951 Establishing a roadmap for therapeutics development for cutaneous neurofibromas

2017; Elsevier BV; Volume: 137; Issue: 10 Linguagem: Inglês

10.1016/j.jid.2017.07.025

1523-1747

Sharad K. Verma, P. Wolkenstein, Lu Q. Le, J. Lee, B. C. Widemann, Isaac Brownell, Kurt Jarnagin, Robert M. Lavker, Eric Legius, Ronald Anderson, Scott R. Plotkin, Hubert Weinberg, Denise Casey, H KO, S. LaRosa, Pamela Knight, Michael K. Parides, Naba Bora, Jennifer Morris, Vincent M. Riccardi, Bruce R. Korf, Jaishri O. Blakeley,

Cancer Treatment and Pharmacology

Cutaneous neurofibromas (cNF) are multicellular tumors involving the skin that are one of the hallmark findings of neurofibromatosis type 1 (NF1), and patients with NF1 often identify these tumors as their greatest burden. As there is no known way to prevent these tumors from developing and current treatments are limited to regional procedure based approaches that have uncertain efficacy, the Neurofibromatosis Therapy Acceleration Program (NTAP) has undertaken the targeting of cNF as part of its strategic mission. Recently, NTAP hosted a cNF summit comprised of leading experts from a range of disciplines representing the ranks of academia, industry, and public agencies in order to create a community of invested experts in this space and identify the key areas of research priority for developing therapies for cNF. This group drafted core questions, reviewed published and unpublished materials, and created summaries about the material that may influence therapeutic development for cNF. Key focus areas for understanding disease pathogenesis, therapies development, and clinical trial optimization were identified and represent a ‘road map’ for accelerating the development of therapies for cNF.