Establishing priorities and advancing the field of NF1 research

Introduction to NTAP symposia
NTAP convenes leading researchers and innovators from across multiple fields and scientific backgrounds in regular symposia to establish priorities for the future NF1-related research. These symposia provide a deliberate process for assessing the state of the science, reflecting on areas of priority to advance meaningful therapeutics for pNF and cNF and creating a framework for a focused research. These invitation-only events bring together a depth and breadth of expertise that creates unparalleled opportunities for collaboration and innovative thinking about the science of NF1.

In 2016 NTAP assembled a team of experts from various disciplines and backgrounds in Landsdown, Virginia to deeply investigate questions pertinent to cutaneous neurofibroma (cNF.) The teams met monthly to review published and unpublished materials, and created summaries about the material known and unknown that may influence therapeutic development for cNF.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.

From these efforts, the first cNF-related request for applications (RFA) was announced in 2017. Five manuscripts setting the international cNF research agenda were also published. Ten projects were funded in 2018 generating some of the most critical data needed for cNF therapeutic advancement.

Fast Facts
NTAP 2016 Cutaneous Neurofibroma Symposium
Landsdown, VA

The first ever cNF scientific initiative
9 projects awarded totaling $3 million in grants
17 thought leaders reviewed data and identified most urgent needs for cNF research and clinical therapeutic development
5 manuscripts produced
10 new scientific projects created to advance cNF understanding
Launched projects that generated the first genetically engineered mouse models for cNF, identified the cell of origin for cNF, created semi-immortalized cell lines and co-culture systems for cNF.

Scientific partners
Presenters at the symposium are vested scientific partners, collaborating with NTAP to advance efforts in targeting cutaneous neurofibroma.

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)
Cutaneous Neurofibroma Initiative Projects (initiated 2018)