NEWS NFTHE NEWSLETTER OF THE CHILDREN’S TUMOR FOUNDATION WINTER 2019 NF Innovation Week INSIDE: The Children’s Tumor Foundation (CTF) had a monumental presence in San Francisco this September as neurofibromatosis (NF) patients, clinicians, researchers, families, 3 NF Conference Recap caregivers, volunteers, and data experts came together for a series of educational 8 NF Forum Summary and inspiring events. The NF Conference and NF Forum were held in conjunction at 9 Hackathon the Hyatt Regency Hotel, San Francisco from September 20 – 24, 2019. Nearly 500 10 Discovery Fund Giving clinicians, researchers, pharma representatives, patient advocates, and other healthcare 12 Galas to End NF professionals attended this year’s NF Conference. Adding to this number were over 150 Forum participants, including our Volunteer Leaders, which brought the number of individuals whose sole focus is to end NF to well over 650 people, and all under one roof! The week prior to these annual events, the second NF Hackathon was held to mine the data collected from years of CTF research. Continued on Page 3 Michael Fisher, MD, Andrea Gross, MD, and Brigette Widemann, MD at the NF Conference. FROM the President Annette Bakker, PhD proud to represent such a wonderful group Aidan Fraser received the Strength and of patients and families, and a group of ex- Honor Award on that fun-filled evening. I perts so enthusiastic about serving them. have had the pleasure of getting to know Aidan this year through his work with CTF Autumn is a season of fundraising for the and his advocacy at the FDA. Foundation, and this year many delight- ful events brought our donors together The Children’s Tumor Foundation is dedi- to fund research. At the National Gala in cated to finding treatments for all types of New York City, we celebrated the launch of neurofibromatosis: NF1, NF2, and schwan- the Discovery Fund, a $8 million initiative nomatosis. This year marked the launch of supporting the Foundation’s longstanding the NF2 Accelerator Initiative, and we are s we reflect on the past year commitment to driving the most promis- incredibly excited about the progress be- here at the Children’s Tumor ing NF research. At the Gala, it was an ing made toward treatments for NF2. Foundation, I am deeply honor to express our gratitude to the grateful to all of you, who Discovery Fund Founders: Shelley and NF has no boundaries and CTF’s global Ahave made our success possible, thereby Frank Haughton, Richard Horvitz and activities are moving forward to help improving the lives of all those living with Erica Hartman-Horvitz, and Jim Bob and the 2.5 million people worldwide who neurofibromatosis (NF). Because of you, Laurée Moffett. are living with NF. You can read more we are closer and closer to FDA approved about CTF Europe in this newsletter, and I treatments for NF. NF Hero Brianna Worden was named our especially want to thank WuXi AppTec for incoming 2020 National Ambassador, their help in spreading NF awareness in Nowhere was the importance of your sup- an honor passed to her from our current China this past year. port more palpable than at this year’s NF Ambassador, McKinnon Galloway. Brianna Conference, NF Forum, and NF Hackathon spoke passionately about her journey with in San Francisco, California. Researchers, NF, describing a story full of exceptional Gratefully Yours, clinicians, and data experts from around strength, resilience, and courage. the world gathered to share the latest findings in the field of NF. In the same San At the Detroit beNeFit, which is held each Francisco hotel, patients, volunteers, and year by our esteemed Board Member Dan caregivers came together to learn and Gilbert and his lovely wife Jennifer, another Annette Bakker, PhD encourage one another. I am tremendously remarkable NF Hero was honored. President, Children’s Tumor Foundation Executive Members Board Members Advisory Board Honorary Directors Board of Directors Richard Horvitz Daniel Altman Members Suzanne Earle Chair Bruce R. Korf, MD, PhD Amy Boulas Michie Stovall O’Day Chair, Medical Tracy Galloway Robert Brainin Alan Robbins, MD Advisory Committee Vice Chair Daniel Gilbert Carolyn E. Setlow Randall Stanicky John Golfinos, MD Scott Plotkin, MD, PhD Nate Walker Chair, Clinical Care Treasurer Carol Harrison Kalagher Ed Stern, Advisory Board Gabriel Groisman Simone Manso Pro Bono Counsel Secretary Steven L. McKenzie Peggy Wallace, PhD Laura Perfetti Chair, Research Allan Rubenstein, MD Linda Halliday Martin Michael Peterson Advisory Board Director of Medical Chair Emeritus Liz Rodbell Affairs Emeritus Kenneth Rudd Volunteer Leadership Richard Soll Council Chair Renie Moss Stuart Match Suna Rachel B. Tiven Join nfregistry.org Donate ctf.org/donate Connect Research NEWS NF CONFERENCE RECAP he Conference’s three-and-a- national networks. Keynote Speaker Stephen half-day agenda was packed with Groft, PharmD, of the National Center for Advancing Tsessions covering a vast array of im- Natural History of Neurofibromatosis Translational Science portant topics. With a nod to our opening Brigitte Widemann, MD, of the National keynote speaker and borrowing from the Cancer Institute (NCI) provided an overview follow-up of adult NF1 patients using whole- title of his talk, one could say, the theme of of the Natural History Studies for Drug body MRI, which demonstrates dynamic this year’s Conference was, “From Chal- Development in Rare Disease at the National changes in internal neurofibroma size. lenge to Challenge - and a whole lot in Institutes of Health (NIH). She explained between.” that elucidating the natural history and Jonathan Payne, PsyD, of the Murdoch biology of plexiform neurofibromas will help Children’s Research Institute, Australia, The half-day pre-conference Clinical Care identify strategies for cancer prevention. described preliminary findings of autism Symposium was organized by the CTF spectrum disorder in children with Clinical Care Advisory Board (CCAB), and Invited speaker Belinda Barton, PhD, from NF1 from the multi-site PANDA study, focused on themes of NF diagnostic criteria the Children’s Hospital at Westmead, which established a framework to better revision, newly published guidelines for Australia presented on the natural history understand and treat autism. NF1 management, and the integration of of the NF1 cognitive phenotype in early these guidelines into clinical practice. The childhood. The aim of this study was to Kristin Lee, MD, of the University of Wis- symposium was chaired by CCAB Chair identify early predictors of later cognitive consin-Milwaukee gave a platform talk Scott Plotkin, MD, PhD, of Massachusetts and reading difficulties. describing her team’s work on examining General Hospital. attention problems and anxiety in children Pam Wolters, PhD, of the NCI gave a with NF1, and suggested that psychosocial The opening keynote address of the Confer- platform talk on physical functioning functioning for children with NF1 needs to ence was given by Stephen Groft, PharmD, outcomes in children with NF1 and be regularly monitored over time. senior advisor at the National Center for plexiform neurofibromas on the SPRINT Advancing Translational Science (NCATS) trial, which is a Phase II trial of the MEK In his platform talk, Cody Peer, MS, and one of the leading experts in the rare inhibitor selumetinib. One objective of PhD, of the National Cancer Institute, disease space. His keynote focused on the the study was to evaluate the effects of presented data from a 10-year NIH natural challenges facing the rare disease commu- selumetinib on physical functioning. history study to characterize plexiform nity, and called for researchers to recognize neurofibroma growth, with a goal of using the value of patient registries, natural his- Ina Ly, MD, of Massachusetts General Hospi- this model to predict the growth of these tory studies, research consortia, and inter- tal, presented a platform talk on long-term tumors. NF CONFERENCE Following their presentation, Staci Martin, PhD, of the National PSYCHOSOCIAL ISSUES Cancer Institute, spoke about exploring treatment engagement as OF THE NF PATIENT a key feature of treating chronic pain symptoms and demonstrated that, when given an Acceptance and Commitment Therapy (ACT) Ana-Maria Vranceanu, PhD, and Ethan Lester, PhD, of intervention, 60% of patients participate in at-home practices on a Massachusetts General Hospital/Harvard Medical School, weekly basis. Allison Del Castillo of the Children’s National Health co-chaired this session. Dr. Vranceanu noted the importance System presented data highlighting the relationship between of increasing funding for psychosocial clinical trials, as well as internalizing and externalizing psychiatric symptoms and social ensuring that we retain talented psychologists in the field of functioning within children with NF1. NF. Dr. Lester presented his and Dr. Vranceanu’s research on resiliency across adults, adolescents, and adults with NF2 who are These three talks comprehensively showed that psychosocial deaf. Dr. Lester presented data from three randomized controlled factors are common across all neurofibromatoses and all ages, and Phase II clinical trials, which showed substantial improvement that psychosocial interventions show high promise in improving in resiliency factors and quality of life after group-based quality of life, resiliency, and pain in patients with NF1, NF2, or psychosocial interventions were delivered via secure live video. schwannomatosis. NF NEWS | WINTER 2019 • 3 Research