NF1 and Cutaneous Neurofibroma

Cutaneous neurofibromas (cNF) are an unmet need within the complex syndrome of NF1 and patients with NF1 often identify these tumors as their greatest burden. As part of our commitment to helping patients with NF1, the Neurofibromatosis Therapy Acceleration Program (NTAP) has taken on the targeting of Cutaneous Neurofibromas as part of its overall mission. To launch our efforts in this area, NTAP hosted a summit on cutaneous neurofibromas towards the end of 2016 to create a community of invested thinkers with the resources and time necessary to explore key questions pertaining to developing effective therapeutics for cNF in people with NF1. The intimate and focused 2 day summit was an interactive meeting with the major goal of identifying the key questions for understanding and treating cNF, such that post-meeting NTAP could launch requests for applications (RFAs) aimed at addressing those questions in the next 3 years. Experts, with and without explicit experience with NF1 or cNF, from disciplines across biology, chemistry, dermatology, surgery, skin cancer, regenerative medicine/tissue repair, wound healing, genetics, and immunology, representing the ranks of academia, industry, and government agencies met to discuss at an in-depth level various scientific topics associated with disease pathogenesis and treatment within the following major themes:

• Disease Initiation, Progression, Development
• Therapies Development
• Clinical Trials Design, Optimization, Development

From these efforts, requests for applications (RFAs) will be released throughout 2017 to spur the highest priority research for cutaneous neurofibroma.

The persons listed below (alphabetically) were presenters at the summit, and are vested scientific partners with NTAP in advancing our efforts in targeting cutaneous neurofibroma. A core value of NTAP is collaboration, and this group will work with us to seek partners to help us in our shared cause – to accelerate the development of medicines for patients with cutaneous neurofibroma.

NTAP cutaneous neurofibroma summit scientific partners:

Richard ‘Rox’ Anderson, MD (Harvard, Massachusetts General Hospital)
Jaishri Blakeley, MD (NTAP, Johns Hopkins)
Isaac Brownell, MD, PhD (National Cancer Institute)
Denise Casey, MD (Food and Drug Administration)
Kurt Jarnagin, PhD (Anacor/Pfizer)
Hon-Sum Ko, MD (Food and Drug Administration)
Bruce Korf, MD, PhD (U. Alabama)
Robert Lavker, PhD (Northwestern)
Lu Le MD, PhD (U. Texas, Southwestern)
James Lee, MD (Roivant Pharmaceuticals)
Eric Legius, MD, PhD (U. Leuven)
Scott Plotkin, MD, PhD (Harvard, Massachusetts General Hospital)
Vincent ‘Vic’ Riccardi, MD, MBA (The NF Institute)
Sharad K. Verma, PhD (NTAP, Johns Hopkins)
Hubert Weinberg, MD (Private Practice, and Mt. Sinai)
Brigitte Widemann, MD (National Cancer Institute)
Pierre Wolkenstein, MD, PhD (Paris Est University)