www.nf-paris2018.com 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 1 ENDORSEMENTS / PATRONAGE Endorsements & Patronage List
The conference is endorsed by the following organisations:
ERN EURACAN ERN GENTURIS FIMARAD RESAP
Réseau Sarcome de l’Assistance publique
The conference is under the High Patronage of the following Institutions:
Other Supports: CONTENTS Table of Contents
Welcome Message...... 4
Organization...... 5
Schedule at a Glance...... 7
Conference Co-Chairs...... 9
Keynote Speakers...... 11
Agenda...... 14
Ancillary Meetings...... 26
Participants...... 30
Conference Venue Floor Plan...... 37
Social Program: Networking Session, Concert & Dinner Cruise...... 38
Nearby Restaurants...... 39
List of Chairs and Scientific Committee...... 41
Sponsors...... 42
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2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 3 Dear NF Conference Attendees:
On behalf of the Children’s Tumor Foundation, the European Neurofibromatoses Group and the Association Neurofibromatoses et Recklinghausen, we want to welcome you to the 2018 Joint Global NF Conference. It is very fitting that this unique meeting is being held during CTF’s 40th anniversary year in the beautiful, historic city of Paris, the City of Light, a global center of art, architecture, gastronomy and fashion. And for the next several days, the global epicenter of neurofibromatosis research, clinical care, patient education and advocacy.
With a nod to the famous American composer, George Gershwin, it was Pierre Wolkenstein’s dream some years ago to have “An American in Paris,” in a symbolic message of global unity. Annette Bakker, president of the Children’s Tumor Foundation, shared that dream and agreed for CTF to be that American in Paris, and to bring its annual international conference to this glorious European capital. So for the first time, CTF, whose meeting has grown over the years to over 400 participants, agreed to join forces with the European NF Group, and the NF2 and schwannomatosis specialists with their bi-annual State of the Art meeting, to host the largest-ever gathering of the top NF researchers and clinicians in the world. This meeting will also play host to an international group of patients and patient advocates bringing the participant level to over 700!
Much information will be shared over the coming days—from the promise of MEK inhibitors and the selumetinib clinical trials to the outcomes of CTF-sponsored pivotal workshops that have been held this year—a review and revision of the diagnostic criteria of the neurofibromatoses, and an animal model workshop that considered and classified NF-specific animal models, including the newly-developed and promising pig models. We have expanded the platform for experts in psychosocial issues to engage attendees in a broader discussion of the quality of life of NF patients, and, for the first time, a live surgery workshop is being offered that will have three different operations to view.
We want to remind our participants that one of the goals of this meeting is to foster strong and lasting relationships. While you are here, enjoy the company of old friends, make some new friends, celebrate each other’s successes and discoveries, and build on this knowledge. This is an opportunity to share science amongst friends—science without borders. NF has No Frontiers. Nor do we, the global NF community.
The next several days will be intellectually challenging, but we encourage you to engage in the discourse, challenge the information that will be put forth, and strive to make the science better together to reach the ultimate goal—to end NF. Bienvenue à Paris—Welcome to Paris!
Sincerely,
ANNETTE BAKKER President, Children’s Tumor Foundation
PIERRE WOLKENSTEIN President of the Joint Global NF Conference
MAURIZIO CLEMENTI President, European Neurofibromatoses Group
JEAN MICHEL DUBOIS President, Association Neurofibromatoses et Recklinghausen
4 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 ORGANIZATION Conference & Supporting Institutions
ORGANIZATION
CONFERENCE ORGANIZATIONAL CHART
President of the Joint Global Meeting for Neurofibromatoses: Local Organizing Committee: Pierre Wolkenstein (Créteil, France) Pierre Wolkenstein (Créteil, France) Salah Ferkal (Créteil, France) European Chairs: Françoise Tailhardat (Créteil, France) Michel Kalamarides (Paris, France) Benoît Funalot (Créteil, France) Eric Pasmant (Paris, France) Marie-Laure Armand (Créteil, France) Michel Kalamarides (Paris, France) USA Chairs: Eric Pasmant (Paris, France) Lu Q. Le (Dallas, Texas, USA) Piotr Topilko (Paris, France) Ludwine Messiaen (Birmingham, Albama, USA) Laurent Lantieri (Paris, France) Philippe Decq (Paris, France) Children’s Tumor Foundation: François Doz (Paris, France) Annette Bakker (President Chief Scientific Officer) (NYC, New York, USA) Jacques Grill (Villejuif, France) Patrice Pancza (VP, External Relations) (NYC, New York, USA) International Relationships: Marco Giovannini (Counselor) (Los Angeles, USA) Vincent Riccardi (International Ambassador) (La Crescenta, USA)
THE SUPPORTING INSTITUTIONS CHILDREN’S TUMOR FOUNDATION
Officers - Board of Directors Richard Horvitz - Chair Annette Bakker, PhD - President and Chief Scientific Officer Tracy Galloway - Vice Chair Randall Stanicky - Treasurer Gabriel Groisman - Secretary Bruce R. Korf, MD, PhD - Chair, Medical Advisory Committee Linda Halliday Martin - Chair Emeritus
Members - Board of Directors Honorary Directors Daniel T. Altman John McCarthy Suzanne Earle Rob Brainin Steven McKenzie Michie O’Day William G. Brooks Laura Perfetti Alan H. Robbins, MD Dan Gilbert Michael Peterson Carolyn E. Setlow John Golfinos, MD Kenneth C. Rudd Nate Walker Matthew Hay Rachel B. Tiven Edward M. Stern, Pro Bono Counsel Chad Leathers David Viskochil, MD, PhD Allan E. Rubenstein, MD, Director of Medical Affairs, Emeritus Stuart Match Suna Margaret (Peggy) Wallace, PhD
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 5 ORGANIZATION Supporting Institutions
EUROPEAN NEUROFIBROMATOSES GROUP
Board of Directors Maurizio Clementi (Padua, Italy) - President Gareth Evans (Manchester, UK) Pierre Wolkenstein (Paris, France) - Secretary General Conxi Lázaro (Barcelona, Spain) Thijs van der Vaart (Rotterdam, Netherlands) - Treasurer Juha Peltonen (Turku, Finland) Hildegard Kehrer-Sawatzki (Ulm, Germany) Meena Upadhyaya (Cardiff, UK) Annette Bakker (New York, USA) Ali Varan (Ankara,Turkey)
ASSOCIATION NEUROFIBROMATOSES ET RECKLINGHAUSEN
Board of Directors Jean Michel Dubois - President Julien Thurion - Treasurer Mireille Bourlier - Secretary Valérie Baudet Sophie Devey Peggy LeBoeuf Rosemarie Picot Sandrine Sicard Renée Vaillant Jean-Pierre Villette
NF FRANCE: NATIONAL REFERAL CENTER FOR NEUROFIBROMATOSIS
Pierre Wolkenstein Michel Kalamarides Patrick Combemale
6 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 SCHEDULE Schedule At-A-Glance TIME EVENT LOCATION 08:30 12:30 OPTIONAL SATELLITE SYMPOSIUM: Developing Therapeutics for Cutaneous Neurofibromas PETIT AMPHI
13:30 15:30 EDUCATIONAL SYMPOSIUM (PART 1) – CLINICAL: Diagnostic Challenges GRAND AMPHI
13:30 15:30 ANIMAL MODELS (PART 1) – BASIC: Pathology of Genetically Engineered NF Animal Models: Consensus Report ROOM 201
15:45 16:10 Break ROOM 8
16:10 18:15 EDUCATIONAL SYMPOSIUM (PART 2): Management Challenges GRAND AMPHI FRIDAY
2 NOVEMBER 16:00 18:30 ANIMAL MODELS (PART 2): Pathology of Genetically Engineered NF Animal Models: Consensus Report ROOM 201
13:30 18:00 POUR LES PATIENTS ET LEURS FAMILLES: ETAT ACTUEL DES SOINS CLINIQUES ET DE LA RECHERCHE PETIT AMPHI
13:30 18:00 FOR PATIENTS AND FAMILIES: CURRENT STATE OF CLINICAL CARE AND RESEARCH (English Version) ROOM 69
08:30 08:40 Welcome Remarks GRAND AMPHI
Platform Talk: SPRINT: Phase II Study of the MEK 1/2 Inhibitor Selumetinib (AZD6244, ARRY-142886) in Children with 08:40 09:00 GRAND AMPHI Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
09:00 10:00 KEYNOTE LECTURE: Epigenetics: One Genome, Multiple Phenotypes GRAND AMPHI
09:00 10:00 European Association Meeting PETIT AMPHI
10:00 10:30 Break ROOM 8
10:30 12:00 European Association Meeting PETIT AMPHI
10:30 12:00 PLENARY SESSION: EPIGENETICS GRAND AMPHI
12:00 13:00 Lunch Break
13:00 14:00 KEYNOTE LECTURE: Schwann Cell Biology GRAND AMPHI
SATURDAY 14:00 15:30 PLENARY SESSION: SCHWANN CELLS AND NEUROFIBROMAS GRAND AMPHI 3 NOVEMBER 14:00 16:00 PARALLEL SESSION: SURGERY IN NF1 ROOM 201
14:00 15:30 PARALLEL SESSION: PSYCHOLOGY WORKSHOP ROOM 69
15:30 16:00 Break ROOM 8
16:00 17:50 PLENARY SESSION: PSYCHOSOCIAL IMPACT OF NEUROFIBROMATOSIS GRAND AMPHI
17:50 18:00 POSTER ADVERTISEMENTS GRAND AMPHI
17:50 19:00 Ancillary Meeting: NF Clinics PETIT AMPHI
18:00 19:30 BASIC SCIENCE POSTER SESSION ROOM 101
09:00 10:00 KEYNOTE LECTURE: Malignant Nerve Sheath Tumor: Improving the Prognosis GRAND AMPHI
10:00 10:50 PLENARY SESSION: MUTATIONS IN OTHER CANCERS (PART 1) GRAND AMPHI
10:50 11:20 Break ROOM 8
11:20 12:00 PLENARY SESSION: MUTATIONS IN OTHER CANCERS (PART 2) GRAND AMPHI
12:00 13:00 Lunch Break / Ancillary Meeting PETIT AMPHI
13:00 14:30 NF2 AND SCHWANNOMATOSIS STATE OF THE ART (PART 1) CLINICAL PETIT AMPHI
13:00 14:30 PARALLEL SESSION: GLIOMA – IN/OUT OF NF GRAND AMPHI
13:00 14:35 PARALLEL SESSION: FUNCTIONAL GENETICS AND INTERPRETATION/CLASSIFICATION OF VARIANTS ROOM 201 SUNDAY
4 NOVEMBER 14:30 15:00 Break ROOM 8
15:00 17:00 NF2 AND SCHWANNOMATOSIS STATE OF THE ART (PART 2) PETIT AMPHI
15:00 17:15 PARALLEL SESSION: LEARNING DISABILITIES GRAND AMPHI
15:00 17:15 PARALLEL SESSION: GENOTYPE/PHENOTYPE OF NF1 ROOM 201
17:15 17:25 POSTER ADVERTISEMENTS GRAND AMPHI
17:25 18:55 CLINICAL POSTER SESSION ROOM 8
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 7 SCHEDULE Schedule At-A-Glance TIME EVENT LOCATION KEYNOTE LECTURE: Translational Mouse: From Genetic Models to the Mouse Hospital,and Co-Clinical Trials for 08:30 09:30 GRAND AMPHI Understanding and Treatment of Cancer
09:30 11:00 PLENARY SESSION: MICROENVIRONMENT GRAND AMPHI
09:30 10:50 PARALLEL SESSION: NF2/SCHWANNOMATOSIS PETIT AMPHI
10:50 11:20 Break ROOM 8
11:20 12:50 PLENARY SESSION: PAIN AND PRURITIS IN NEUROFIBROMATOSIS GRAND AMPHI
12:50 13:30 Lunch Break / Late Breaking Abstract Platform Presentation (Clinical)
13:30 14:30 FONDATION ARC POUR LA RECHERCHE SUR LE CANCER KEYNOTE LECTURE: Targeted Therapy in Melanoma GRAND AMPHI MONDAY
5 NOVEMBER 14:30 16:30 PLATFORM PRESENTATIONS – BASIC PETIT AMPHI
14:30 16:30 PLATFORM PRESENTATIONS – CLINICAL GRAND AMPHI
16:30 16:45 Break ROOM 8
16:45 18:15 PLENARY SESSION: MEDICAL AND SURGICAL CARE: A MULTIDISCIPLINARY APPROACH FOR NF GRAND AMPHI
16:45 18:40 PARALLEL SESSION: BIOLOGY OF NF2 AND SCHWANNOMATOSIS PETIT AMPHI
08:30 09:30 ORAL POSTER PRESENTATIONS GRAND AMPHI
09:30 10:00 KEYNOTE LECTURE: Plan France Médecine Génomique 2025 GRAND AMPHI
10:00 11:10 PLENARY SESSION: NF: PAST, PRESENT AND FUTURE GRAND AMPHI
11:10 11:40 Break GALERIE
11:40 12:25 PLENARY SESSION: NF: PRESENT, PAST AND FUTURE GRAND AMPHI TUESDAY
6 NOVEMBER 12:25 13:45 PLENARY SESSION: REVISION OF DIAGNOSTIC CRITERIA GRAND AMPHI
13:45 14:00 Congress Adjourned
15:00 18:00 REiNS Session (By Invitation) ROOM 101
We would like to acknowledge the generous grant support from the National Institute of Neurological Disorders and Stroke of the National Institutes of Health of the 2018 Joint Global Neurofibromatosis Conference.*
*Research reported in this publication was supported by NINDS of the NIH. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
8 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 BIOS 2018 NF Conference Co-Chairs
Marco Giovannini, MD, PhD, UCLA, USA
Marco Giovannini, M.D., Ph.D. is Professor-in-Residence of Head and Neck Surgery at the David Geffen School of Medicine of the University of California Los Angeles (UCLA) and Scientific Director of the UCLA NF2 Multidisciplinary Clinic. A native of Italy, Dr. Giovannini earned his M.D.;Ph.D at the University of Bologna. He conducted research in France at the Institut National Recherche Sante Medicale (INSERM) for many years, as the head of the Unit “Functional Genomic of Solid Tumors”, in Paris. A molecular geneticist by training, Marco Giovannini is a physician-scientist who conducts laboratory and clinical research in the field of neurofibromatoses. His NIH, Department of Defense and private foundation-supported research laboratory develops models of disease to test new therapeutic options and studies mechanism of action of treatments in NF patients.
Michel Kalamarides, MD, PhD, Hopital Pitié-Salpetrière, AP-HP
Michel Kalamarides is a Professor of Neurosurgery at Pitié-Salpetrière Hôpital (Faculté de Medécine, Sorbonne- Université), Paris France.
He is specialized in vestibular schwannoma and meningioma surgery. Within a large INSERM unit- U 1127 at Brain and Spine Institute (Pitié-Salpetrière), he is leading a research group on meningioma, particularly using mouse models to dissect the mechanisms of meningioma initiation and progression.
He is leading the French NF2 reference clinical center and the French NF2 network. With Pr Olivier Sterkers, he organized the first NF2 state of the Art meeting in 2006 in Paris.
Lu Le, MD, PhD, University of Texas Southwestern, USA
Dr. Le is a Dermatologist with a scientific focus on neural-crest derived tissue development, regeneration and tumorigenesis, in particular the biology of neurofibromatosis. He graduated from the Medical Scientist Training Program at UCLA and completed residency training in Dermatology and a postdoctoral fellowship in Cancer Biology from UT Southwestern Medical Center. He is currently an associate professor and holds the Thomas L. Shield, M.D. Professorship in Dermatology at UT Southwestern. As a principal investigator in cancer biology, Dr. Le’s laboratory investigates how tumor cell of origin and tumor microenvironment interplay to regulate NF-associated tumorigenesis. A major contribution of Dr. Le’s laboratory is the generation and exploitation of novel neurofibromatosis models to decipher mechanisms that initiate neurofibroma development and drive their malignant transformation.
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 9 BIOS 2018 NF Conference Co-Chairs
Ludwine Messiaen, PhD, University of Alabama at Birmingham, USA
Ludwine Messiaen, PhD, FACMG received a BSc in Social Work at the Institute for Psychosocial Education in Kortrijk, Belgium, a MS and PhD in Molecular Biology and Cancer Genetics at the University of Ghent, Belgium and joined Faculty of the Center of Medical Genetics at the Ghent University Hospital Belgium where she directed the molecular diagnostic laboratory from 1991 to 2003. She then moved to the US to serve as the director of the Medical Genomics Laboratory at the University of Alabama at Birmingham (UAB), specializing in clinical testing for all rasopathies, neurofibromatoses and schwannomatoses. She is a Professor in Genetics in the UAB Department of Genetics. She is a Fellow of the American College of Medical Genetics and Board Certified in Clinical Molecular Genetics by the American Board of Medical Genetics.
Dr. Messiaen is known for the early development, translation and application of comprehensive RNA-based NF1 testing in the clinical setting and is committed to academic molecular diagnostics. Her group described the presence of 2 NF1 hits in the melanocytes of Café-au-lait macules, but not in the keratinocytes or fibroblasts. This finding along with the work on cultured Schwann cells was translated into a clinical test helpful to diagnose mosaic/segmental NF.
She is interested to fully explore the spectrum of NF1 mutations, including mutations not readily identified by DNA-based analyses of the coding regions (such as deep-intronic splice mutations).
She explores genotype-phenotype correlations using several complementary approaches. She developed and curates a patient database with de- identified genotypes and phenotypes of over 8,500 unrelated mutation-positive individuals.
Her laboratory engages in the identification of novel genes contributing to the neurofibromatoses. This includes involvement in the identification of Legius syndrome. After the SPRED1 gene discovery, she contributed substantially to the quick delineation of the full breath of phenotypes associated with this novel rare disorder. Furthermore, her research on a well-characterized patient cohort led to the identification of the LZTR1 gene as a novel major schwannomatosis-predisposing gene.
Eric Pasmant, PhD, Department of Molecular Genetics, Cochin Hospital, Paris Descartes University, Paris, France
Eric Pasmant, PharmD, PhD, is assistant professor of Paris-Descartes University specialized in molecular genetics. He completed his medical biology residency and molecular genetics fellowship at the Cochin Hospital in Paris. His research has focused on the molecular genetics of variable expressivity of neurofibromatosis type 1 (NF1), and genetics of NF1- associated peripheral nerve sheath tumors and their therapeutic targeting. In addition to his research activity as senior scientist, Eric Pasmant is the technical manager of the Next Generation Sequencing (NGS) platform of the laboratory of molecular genetics at Cochin hospital in Paris.
Pierre Wolkenstein, MD, PhD, Hôpital Henri-Mondor, France
Since the 1990s Dr. Wolkenstein has been coordinating the management and clinical research of neurofibromatoses and, especially neurofibromatosis type 1 in France. He has conducted many clinical studies leading to high standard publications in the field. At the present time, he is head of the French National Referral Center for Neurofibromatoses (Ministry of Health Rare Diseases program) and head of the Department Cancers Immunity Transplantation Infections in Henri-Mondor Hospital (the academic hospital of University Paris Est Créteil (Grand Paris Region)). The cohort of patients in the center is around 2000. His team performed the first face transplant in a patient with neurofibromatosis 1, identified the first modifying gene, conducted therapeutic trials and developed basic research on NF1 animal models. He and his team are collaborating with pharmaceutical companies for developing topical treatment of cutaneous neurofibromas (Pierre Fabre Dermatologie). He has strong links with the French Neurofibromatoses Lay Group and at the present time is the President of its scientific committee. He will also organize in 2018 in Paris the Global Joint Meeting on neurofibromatoses (joint European and Children Tumor Foundation Meeting).
10 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 BIOS 2018 NF Conference Keynote Speakers
Paolo G. Casali, MD, Director, Medical Oncology Unit 2, Fondazione IRCCS Instituto Nazionale Tumori Assoc. Professor of Medical Oncology, University of Milan, Italy Member Executive Board of ESMO
Paolo Giovanni Casali, Associate Professor of Medical Oncology at the University of Milan, is Director of the Medical Oncology Unit 2 (focusing on adult mesenchymal tumours and rare cancer networking) at the Istituto Nazionale Tumori, Milan, Italy, where he also serves as Secretary of the Ethics Committee. He is a member of the Executive Board of the European Society for Medical Oncology (ESMO), as Chair of the Public Policy Steering Committee and the EU Policy Committee.
His clinical and research activities focus on sarcomas, mainly adult soft tissue sarcomas and gastrointestinal stromal tumors (GIST). He is Secretary of the Italian Sarcoma Group and a member of the Soft Tissue & Bone Sarcoma Group of the European Organisation for Research and Treatment of Cancer (EORTC). He is a founder and coEditor-in-chief of Clinical Sarcoma Research, an open-access journal on sarcoma, and a member of the ESMO Sarcoma Faculty.
In the area of rare cancers, he coordinates the Joint Action on Rare Cancers (JARC), carried out within the framework of the Third Health Programme of the European Union (EU) with 34 partners and 18 EU Member States, and chairs the Sarcoma domain of EURACAN, the European Reference Network on rare adult solid cancers. He coordinates the Steering committee of Rare Cancers Europe, an ESMO-launched multistakeholder initiative to address the many issues posed by rare cancers.
He is (co)author of more than two hundreds papers in peer-reviewed medical journals.
Kristjan Jessen, PhD, Professor of Developmental Neurobiology, University College London (UCL), United Kingdom
KR Jessen obtained MSc and PhD degrees in Neuroscience while working in the Department of Anatomy and Developmental Biology, UCL, London. Following a Senior Research Fellowship of the Mental Health Foundation in the laboratory of Martin Raff, Department of Zoology, UCL, he was awarded a Wellcome University Award Lectureship in 1983 in the Department of Anatomy and Developmental Biology, UCL. He has been a Professor of Developmental Neurobiology in the same Department (now Research Department of Cell and Developmental Biology) since 1993. His research is on (i) early Schwann cell development and the biology of the Schwann cell precursor (SCP), (ii) the molecular control of myelination and (iii) the response of Schwann cells to injury and genetic disease, including the processes of de-myelination, axonal regeneration and nerve repair. He became a Fellow of the Academy of Medical Sciences in 2002.
Yves Levy, MD, PhD, Chairman, CEO Institut National de la Santé et de la Recherche Médicale (INSERM), Paris, France
Prof. Yves Levy, MD, PhD, has been CEO and Chairman of Inserm since June 2014 and has also led since that time Aviesan, the French Alliance for Health and Life Sciences. Prof. Levy has developed a research activity in several Inserm units since 1985. He led an Inserm team (U955 Unit) “T cell development in Physiology and in HIV disease” from 2007 to 2014, focused on HIV physiopathogenesis from upstream to translational researches in the field of immune interventions and vaccines. He served as the Director orf the Inserm Unit 899 in Dallas (Texas, USA) from 2010 to 2012. Prof. Levy has created the Labex “Vaccine Research Institute” (VRI). VRI is aimed to face the challenges to develop effective vaccines against HIV. This research program is implemented by a large network of 17 teams and key international opinion leaders in this field from different institutions in France and the US through a unique collaborative network and with a central strategic plan. Since January 2016, Yves Levy is the coordinator of the European HIV Vaccine Alliance (EHVA) Consortium. Prof. Levy holds the positions of chair of the immune-based therapies Committee of the ANRS (French Agency of Research against AIDS and Hepatitis), scientific director of the HIV Vaccine Program of the ANRS, members of the WHO Vaccine Advisory Committee, the Council of the Global HIV Vaccine Enterprise and other scientific and professional societies. Prof. Levy has been appointed member of the UN “Global Health Crises” Task Force by Mr. Ban Ki-Moon (July 2016). Prof. Levy has been appointed Dr. Honoris Causa of the Imperial College of London (October 2016).
Prof. Levy has published around 190 articles and chapter books in international peer-reviewed scientific journals (NEJM, Lancet, Cell, Molecular Cell, J. Exp. Med, PNAS, J. Clin Investigation, Blood).
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 11 BIOS 2018 NF Conference Keynote Speakers
Pier Paolo Pandolfi, MD, PhD, Director, Cancer Center and Cancer Research Institute - Beth Israel Deaconess Medical Center, Boston, USA Chief, Division of Genetics, Department of Medicine, BIDMC
Pier Paolo Pandolfi received his M.D. in 1989 and his Ph.D. in 1995 from the University of Perugia, Italy, after having studied Philosophy at the University of Rome, Italy. He received post-graduate training at the National Institute for Medical Research and the University of London in the UK. He became an Assistant Member of the Molecular Biology Program and the Department of Human Genetics at Memorial-Sloan-Kettering Cancer Center in 1994. Dr. Pandolfi grew through the ranks to become a Member in the Cancer Biology and Genetics Program at the Sloan Kettering Institute; Professor of Molecular Biology and Human Genetics at the Weill Graduate School of Medical Sciences at Cornell University; Professor, Molecular Biology in Pathology and Laboratory Medicine, Weill Medical College at Cornell University; and Head of the Molecular and Developmental Biology Laboratories at MSKCC. Dr. Pandolfi was also the incumbent of the Albert C. Foster Endowed Chair for Cancer Research at Memorial Sloan-Kettering Cancer Center.
Dr. Pandolfi presently holds the Reisman Endowed Chair of Medicine, and is Professor of Medicine and Pathology at Harvard Medical School (HMS). He joined the HMS faculty at Beth Israel Deaconess Medical Center (BIDMC) in 2007 to serve as Scientific Director of the Cancer Center, the Director of the Cancer Genetics Program, and the Chief of the Division of Genetics in the Department of Medicine; he is also a Member of the Department of Pathology at BIDMC. He was recently appointed to serve as the Cancer Center Director and the Director of the Cancer Research Institute at BIDMC and HMS.
Dr. Pandolfi has been the recipient of numerous Awards for his research including the LLSA Scholar Award (1997), the Irma T. Hirschl Trust Award (1999), the Alexandra J. Kefalides Prize for Leukemia Research (1999), the Louise and Allston Boyer Young Investigator Award in Biomedical Research (1999), the Hamdan Award for Medical Research Excellence (2000), the Lombroso Prize for Cancer Research of the Weizmann Institute of Science (2001), the Leukemia and Lymphoma Society’s Stohlman Scholar Award (2001), the William and Linda Steere Foundation Award (2004), the prize for Scientific Excellence in Medicine from the American-Italian Cancer Foundation (2005), the Fondazione Cortese International Award (2008), the Prostate Cancer Foundation Creativity Award (2009) and the Ischia International Award (2009).
He also has been awarded the NIH MERIT Award for superior competence and outstanding productivity in research in 2005. In 2006, Dr. Pandolfi was elected as a member of the American Society for Clinical Investigation (ASCI) and the American Association of Physicians (AAP), and in 2007 as Member of the European Molecular Biology Organization (EMBO).
In 2011, Dr. Pandolfi received the Pezcoller Foundation–AACR International Award for Cancer Research. He was also awarded the Scanno International Award for Medicine in 2012, the Pomilio Ethic International Award in Biomedicine, the European Foundation Guido Venosta Award for Cancer Research in 2013, and the America International Award in 2014.
In 2015, Dr. Pandolfi was knighted by the President of the Italian Republic and received the Medal of Honor as “Officer of the Order of the Star of Italy” (Ufficiale dell'Ordine della Stella d'Italia). Dr. Pandolfi has also received the prestigious Outstanding Investigator Award from the NIH/NCI in 2015.
The research carried out in Dr. Pandolfi’s laboratory has been seminal to elucidating the molecular mechanisms and the genetics underlying the pathogenesis of leukemias, lymphomas and solid tumors as well as in modeling these cancers in the mouse. Dr. Pandolfi and colleagues have characterized the function of the fusion oncoproteins and the genes involved in the chromosomal translocations of acute promyelocytic leukemia (APL), as well as of major tumor suppressors such as PTEN and p53, and novel cancer genes such as POKEMON and INPP4B. The elucidation of the molecular basis underlying APL pathogenesis has led to the development of novel and effective therapeutic strategies. As a result of these efforts, APL is now considered a curable disease. Additional novel therapeutic concepts have emerged from this work and are currently being tested in clinical trials. More recently, Dr. Pandolfi and colleagues have presented a new theory describing how mRNAs, both coding and non-coding, exert their biological functions with profound implications for human genetics, cell biology and cancer biology.
12 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 BIOS 2018 NF Conference Keynote Speakers
Danny Reinberg, PhD, Howard Hughes Institute, Terry and Mel Karmazin Professor of Biochemistry and Molecular Pharmacology, Department of Biochemistry and Pharmacology, NYU Langone School of Medicine at Smilow Research Center, Department of Biochemistry & Molecular Pharmacology, New York
The crux of my research goal over the years has been to understand exactly how RNA polymerase II (RNAPII) acquires selectivity for subsets of genes in a regulated manner. My strategy was to gain a sound grasp of RNAPII-mediated transcription as the template complexity was progressively increased. Efforts by my group through the years initiated with defining the basic prerequisites for RNAPII transcription from a minimal, naked DNA template, and then proceeded to the functional analysis of even more complex arrays of activities that together reconstitute chromatinized templates that exhibit different extents of compaction and others that refurbish the templates into a state amenable to transcription. This latest endeavor entails studies of the post-translational modifications of histones and their potential epigenetic character, and this is the most invigorating stage of my research career.
The epigenetic information contained within the disparate chromatin states is distinct from that of the DNA sequence. Such information is determinant to differential gene expression during development of multi-cellular organisms and to the integrity of adult tissue regeneration throughout cell divisions. Yet, we still do not know how nucleosomes segregate during the process of DNA replication. This fundamental issue is key to understanding the accurate propagation of histone modifications that define distinct chromatin domains and thus retain appropriate gene expression profiles in daughter cells. With this proposal, we intend to tackle this longstanding issue. As well, we will extend our studies of a histone posttranslational modification associated with transcriptional repression and fully explore how this modification is established, maintained and inherited. In keeping with this theme, we will also extend our studies of FACT to explore the role of this histone chaperone in transcription regulation and to identify and characterize FACT-like activities in naturally occurring FACT-deficient cells. It is my goal to contribute to our understanding of these important and interrelated issues using state of the art technologies and “tried and true” biochemical approaches.
Caroline Robert, MD, PhD, Head of the Dermatology Unit, Institut Gustave-Roussy, Villejuif, France Board Member, European Associationi of Onco-Dermatology Melanoma Board Secretary for European Organization for the Research and Treatment of Cancer French Society of Dermatology and Venereology AACR, ASCO
Caroline Robert, M.D., Ph.D. is the Head of the Dermatology Service at Gustave Roussy Cancer Campus and co-director of the Melanoma Research Unit at INSERM 981 Paris-Sud University. She trained at the Paris V University, France and completed a research fellowship at Harvard, Brigham & Women’s hospital in Cancer Immunology and Immunotherapy. She chairs the Melanoma group of the European Organization for the Research and Treatment of Cancer (EORTC) and is board member for the European Association of Onco-Dermatology (EADO), the European Society of Medical Oncology (ESMO), the European Association of Dermato-Venereology (EADV) the French society of Dermatology and Venereology (SFD), the American Society of Oncology (ASCO) and the American Association of Clinical Research (AACR). Her main focuses of interest are clinical and translational Research on Melanoma: immunotherapy and targeted therapy, as well as the study of the cutaneous adverse events of anticancer agents. She is national and international coordinator of many clinical trials of targeted therapy and immunotherapy for melanoma patients, from phase I to III. She has authored more than 240 articles in peer-reviewed scientific journals, including a number of publications on new treatments for metastatic melanoma
Her recent work has focused on identification of new biomarkers for immunotherapy and targeted therapies of patients with melanoma.
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 13 AGENDA Friday · 2 November 2018
08:30 12:30 OPTIONAL SATELLITE SYMPOSIUM PETIT AMPHI
Developing Therapeutics for Cutaneous Neurofibromas Sponsored by the Neurofibromatosis Therapeutics Acceleration Program (NTAP)
Welcome and Overview 08:30 2016-2018: The Changing cNF Landscape (Jaishri Blakeley, MD)
Learning from Cutaneous Neurofibroma Models • Piotr Topilko, PhD – Mouse models of disease 08:40 09:40 • Lu Le, MD, PhD – Cells of origin • Jill Weimer, PhD – Porcine models of disease • Alice Soragni, PhD – 3D organoid models development Cellular and Genetic Targets in Cutaneous Neurofibromas • David Gutmann, MD, PhD – iPS identified genetic targets 09:40 10:15 • Nicolas Ortonne, MD, PhD – Immunologic targets • Robert Allaway, PhD – Human network analysis
Assessing Cutaneous Neurofibromas • Anat Stemmer-Rachamimov, MD, Pierre Wolkenstein, MD, PhD – Classification of cNF 10:15 10:50 • Ashley Cannon, PhD, MGC – Natural history of cNF • Dominique Pichard, MD – Global assessment and severity scales
10:50 11:05 BREAK ROOM 8
Panel discussion with moderated Q/A (Pierre Wolkenstein) • Basic science 11:05 12:05 • Animal models • Clinical trials • EMA • Patient perspective
12:05 12:15 Final comments and adjourn
EDUCATIONAL SYMPOSIUM (PART 1) – CLINICAL Co-chairs: 13:30 15:30 • Dusica Babovic-Vuksanovic, MD, Mayo Clinic, US GRAND AMPHI • Mimi Berman, MD, University of Sydney, Australia • Karin Cunha, PhD, Federal University of Brazil Diagnostic Challenges A Review of the Complex and Unique Testing Approaches Available for the Neurofibromatoses 13:30 13:55 Alicia Gomes, MS, CGC, University of Alabama at Birmingham, US Clinical Overlap with RASopathies 13:55 14:20 Karen Gripp, MD, Nemours/Alfred DuPont Hospital for Children, US
Do Not Forget Constitutional Mismatch Repair Deficiency (CMMRD) 14:20 14:45 Katharina Wimmer, PhD, Genetics Clinic, University of Innsbruck, Austria
Prenatal Diagnostics: From CVS and Amniocentesis to PGD 14:45 15:20 Myra Wick, MD, PhD, Mayo Clinic, US Kristi Jones, PhD, University of Sydney, Children’s Hospital at Westmead, Australia
Less Well Known Dermatological Manifestations in NF1 15:20 15:45 Sirkku Peltonen, MD, PhD, University of Turku, Finland
15:45 16:10 BREAK ROOM 8
14 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 AGENDA Friday · 2 November 2018
ANIMAL MODELS New Animal Models of Neurofibromatosis: Review of Animal Model Histopathology Workshop Palm Springs 2018 13:30 18:30 Co-chairs: ROOM 201 • Marco Giovannini, MD, PhD, UCLA, US • Nancy Ratner, PhD, Cincinnati Children’s Hospital, US • Anat-Stemmer Rachamimov, MD, Harvard University, US
13:30 16:30 Session 1: NF1 – Plexiform Neurofibromas and MPNST Anat Stemmer-Rachamimov: Human pathology – the transition from plexiform 13:30 13:40 neurofibroma to MPNST – ANNUBP. 13:40 13:55 Nancy Ratner: NF1 models – overview of available models and what they have taught us.
Models that develop MPNST through transition pre-existing neurofibroma (stepwise)
13:55 14:05 David Largaespada 14:05 14:15 Nancy Ratner Models that may develop MPNST without transition from atypical or obvious pre-existing benign lesion (ex nuovo) 14:15 14:25 Yuan Zhu 14:25 14:35 Rebecca Dodd 14:35 14:45 Natalie Wu 14:45 15:00 MINI KEYNOTE: Gareth Evans (NF1 mutations in breast cancer)
Nf1-driven models that develop non-MPNST malignancies
15:00 15:10 Bob Kesterson 15:10 15:20 Luis Parada
Anat Stemmer-Rachamimov: Pathology of MPNST formation and other NF1-related 15:20 15:30 malignancies in mouse models.
15:30 16:00 BREAK ROOM 8
16:00 16:35 Session 2: NF1 – Cutaneous Lesions – Neurofibromas and Café au Lait Macules
Adrienne Watson: The pig model – overview of the pig as a disease model and pig 16:00 16:15 models for NF1
16:15 16:25 Kasia Radomska Mouse model of cutaneous neurofibromas
Anat Stemmer-Rachamimov: Summary of pathology of cutaneous lesions in animal 16:25 16:35 models and show the 3 models.
16:30 17:40 Session 3: NF2 and Schwannomatosis Schwannomas
16:30 16:45 MINI KEYNOTE: Brad Welling (Hearing loss mechanisms in NF2)
Marco Giovannini: NF2 and schwannomatosis models of schwannomas – overview of 16:45 17:00 available models and what they have taught us
PeriostinCre;Nf2:
17:00 17:10 Charles Yates
P0delta2-3
17:10 17:20 Marco GIovannini
mGFAPCre;Nf2 and mGFAPCre;Nf2;Smarcb1
17:20 17:30 Jeremie Vitte
Anat Stemmer-Rachamimov: Pathology of benign Schwann cell lesions and 17:30 17:40 schwannoma precursor lesions in DRG and spinal cord and in cranial nerves (w/ special focus on VII and VIII cn.)
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 15 AGENDA Friday · 2 November 2018
17:40 18:10 Session 4: NF2 – Meningiomas
17:40 18:05 Michel Kalamarides: Meningioma models in the mouse – overview Anat Stemmer-Rachamimov: Precursor lesions and benign lesions, Grade II and Grade 18:05 18:10 III meningiomas – pathology
Session 5: Pathology Recommendation for Animal Models of NF1 - NF2/SCH Questions/Discussion 1. Sections – include normal adjacent tissue 18:10 18:30 2. Immuno-panels – standardized workup for new tumors 3. Core laboratories which will share protocols 4. Fixation in formalin for mouse tissue, EDTA for bone 5. Plastic sections and EM in some cases 6. Possible on line atlas of slides
EDUCATIONAL SYMPOSIUM (PART 2) Co-chairs: 16:10 18:15 • Dusica Babovic-Vuksanovic, MD GRAND AMPHI • Mimi Berman, MD • Karin Cunha, PhD
Management Challenges A Holistic Model to Discuss the Impact of Muscular Phenotype on Lifestyle of 16:10 16:35 Individuals with NF1 Juliana Souza, MD, Federal University of Minas Gerais, Brazil
Lessons Learnt from Breast Cancer Screening in High Risk Women—BRCA Screening 16:35 17:00 in Women with NF1, When and How Eva Trevisson, University of Padova, Italy
Oral Manifestations in NF1 17:00 17:25 Karin Cunha, Fluminense Federal University, Brazil
Beyond Esthetic Surgery: Electrodessication for Cutaneous Neurofibromas 17:25 17:50 Hubert Weinberg, Mt. Sinai Hospital, US
Research
Swine Models for Neurofibromatosis 17:50 18:15 David Largaespada, PhD, University of Minnesota, US POUR LES PATIENTS ET LEURS FAMILLES: 13:30 18:00 Etat Actuel des Soins Cliniques et de la Recherche PETIT AMPHI Session francophone / This session will be in French only
FOR PATIENTS AND FAMILIES: 13:30 18:00 Current State of Clinical Care and Research ROOM 69 English version
16 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 AGENDA Saturday · 3 November 2018
08:30 08:40 Welcome Remarks GRAND AMPHI
PLATFORM TALK: SPRINT: Phase II Study of the MEK 1/2 Inhibitor Selumetinib (AZD6244, ARRY-142886) in Children with Neurofibromatosis Type 1 (NF1) and GRAND AMPHI 08:40 09:00 Inoperable Plexiform Neurofibromas (PN) Andrea Gross, MD, National Institutes of Health, National Cancer Institute, US
KEYNOTE LECTURE: Epigenetics: One Genome, Multiple Phenotypes 09:00 10:00 Danny Reinberg, PhD, Howard Hughes Institute, Terry and Mel Karmazin Prof. of GRAND AMPHI Biochemistry and Molecular Pharmacology, NYU Langone School of Medicine, US
09:00 10:00 European Association Meeting PETIT AMPHI
10:00 10:30 BREAK ROOM 8
10:30 12:00 European Association Meeting PETIT AMPHI PLENARY SESSION: EPIGENETICS Co-chairs: 10:30 12:00 • Eric Pasmant, PhD, Universite Paris-Descartes, Hopital Cochin, France GRAND AMPHI • Thomas De Raedt, PhD, Children’s Hospital of Philadelphia, US • Karlyne Reilly, PhD, National Cancer Institute, US
Invited Speakers:
Exploiting Epigenetic Vulnerabilities to Improve Immunotherapy 10:30 10:55 Thomas De Raedt, PhD, Children’s Hospital of Philadelphia, US
Learning from Rare Tumors: Tumor Immunogenicity Beyond Somatic Mutation Burden 10:55 11:20 Josh Waterfall, PhD, Curie Institute, France
PLATFORM TALK: The Genomic Landscape of Schwannomatosis Schwannomas 11:20 11:40 Sheila Mansouri, Canada
PLATFORM TALK: Cutaneous and Plexiform Neurofibromas Maintain Distinct 11:40 12:00 Epigenetic Profiles with Predicted Impact on Tumor Biology Matthew Steensma, US
12:00 13:00 Lunch Break
KEYNOTE LECTURE: Schwann Cell Biology 13:00 14:00 Kristjan Jessen, PhD, Professor of Developmental Neurobiology, University College, GRAND AMPHI London (UK) PLENARY SESSION: SCHWANN CELLS AND NEUROFIBROMAS Co-chairs: 14:00 15:30 • Lu Le, MD, PhD, University of Texas Southwestern Medical Center, US GRAND AMPHI • Juha Peltonen, MD, PhD, University of Turku, Finland • Eduard Serra, PhD, ICTP, Spain
Invited Speakers:
Developmental Origin and Spatiotemporal NF1 Loss of Heterozygosity Leads to 14:00 14:25 Different Types of Cutaneous Neurofibroma Lu Le, MD, PhD, University of Texas Southwestern Medical Center
Pathogenic Mechanisms of Cutaneous Neurofibromas Revealed by Mice Carrying 14:25 14:50 NF1 Loss in Prss56-Expressing Boundary Cap Cells Piotr Topilko, PhD, INSERM, France
PLATFORM TALK: Tonic ATP-Mediated Growth Suppression in Peripheral Nerve Glia 14:50 15:10 Requires Arrestin-PP2 and is Evaded in NF1 Robert Coover, US
PLATFORM TALK: Trametinib in Pediatric Patients with Neurofibromatosis Type 15:10 15:30 1-Associated Plexiform Neurofibroma: A Phase I/IIa Study Christopher L. Moertel, US
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 17 AGENDA Saturday · 3 November 2018 PARALLEL SESSION: SURGERY IN NF1 Co-chairs and speakers: • Laurent Lantieri, MD, PhD, l’Hôpital Européen Georges-Pompidou, France ROOM 201 14:00 16:00 • Xiaojie Hu, MD, PhD, Shanghai Jiaotong University School of Medicine, China • Reinhard Friedrich, MD, University Medical Center, Hamburg, Germany • Joseph Gruss, MD, Seattle Children's Hospital, US
Topic 1: NF plexiform: Coping with the limits of surgical resection: Short term: bleeding, nervous morbidity. Long term: inefficiency. Topic 2: Peripheral nerve surgery: decompression, resection, targeted indications Topic 3: Collaborations with other specialties and interdisciplinary approach Topic 4: Indications (age, locations) limitations of pediatric surgical management, long- term outcomes Topic 5: Resection planning: the means “modern” Planning: 3d modeling, augmented reality etc Topic 6: The limits of conventional plastic surgery: Indications and results VCA
PARALLEL SESSION: PSYCHOLOGY WORKSHOP Participation in this session is by invitation only PROGRAM to be announced ROOM 69 14:00 15:30 Co-chairs: • Staci Martin, PhD, National Institutes of Health, US • Marie-Laure Armand, PhD, Hopital Henri-Mondor, France
15:30 16:00 BREAK ROOM 8
PLENARY SESSION: PSYCHOSOCIAL IMPACT OF NEUROFIBROMATOSIS 16:00 17:50 Co-chairs: GRAND AMPHI • Marie-Laure Armand, PhD, Hopital Henri-Mondor, FR • Staci Martin, PhD, National Institutes of Health, US • Hilda Crawford, PhD, Human Genetics Society of Australia
Invited Speakers:
Supporting the Psychosocial Needs of Individuals with Neurofibromatosis: 16:00 16:25 International Perspectives Belinda Barton, PhD, University of Sydney, Australia
Acceptance and Commitment Therapy (ACT) for Individuals with NF1, Plexiform 16:25 16:50 Neurofibroma Tumors (PNs), and Chronic Pain: Results from a Randomized Controlled Trial Staci Martin, PhD, National Institutes of Health, US
PLATFORM TALK: Improving Quality of Life (QoL) in Internationally Diverse Patients with NF via Secure Live Video: Results of 3 Randomized Controlled Trials (RCTs) in 16:50 17:10 Adults, Adolescents and Patients Who Are Deaf Ana-Maria Vranceanu, US
PLATFORM TALK: Prospective Patient-Reported Outcomes (PROs) Document Clinical Benefit in Children with Neurofibromatosis Type 1 (NF1) and Inoperable 17:10 17:30 Plexiform Neurofibromas (PNs) on SPRINT: A Phase II Trial of the MEK 1/2 Inhibitor Selumetinib (AZD6244,ARRY-142886) Pamela L Wolters, US
NF Clinical Trials Consortium Update 17:30 17:50 Michael Fisher, MD, Children’s Hospital, Philadelphia, US
17:50 19:00 NF CLINICS SATELLITE PETIT AMPHI
17:50 18:00 POSTER ADVERTISEMENTS GRAND AMPHI
POSTER AREA – 18:00 19:30 BASIC SCIENCE POSTER SESSION ROOM 101
18 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 AGENDA Sunday · 4 November 2018
KEYNOTE LECTURE: 09:00 10:00 Malignant Nerve Sheath Tumor: Improving the Prognosis GRAND AMPHI Paolo G. Casali, MD, Dir. Medical Oncology, Unit 2, Fondazione IRCCS Instituto Nazionale Tumori, Assoc. Prof. Medical Oncology University of Milan, Italy
PLENARY SESSION: MUTATIONS IN OTHER CANCERS (PART 1) 10:00 10:50 Co-chairs: GRAND AMPHI • Meena Upadhyaya, PhD, University of Cardiff, Wales, UK • Ophelia Maertens, PhD, Brigham and Women’s Hospital/Harvard University, US
Invited Speakers:
The Expanding Role of NF1 in Sporadic Cancer and Resistance to Targeted Therapies 10:00 10:25 Ophelia Maertens, PhD, Brigham and Women’s Hospital/Harvard University, US
The Landscape of Molecular Alterations and Mechanisms of Progression 10:25 10:50 in NF1 Gliomas Antonio Iavarone, MD, Columbia University, US
10:50 11:20 BREAK ROOM 8
PLENARY SESSION: 11:20 12:00 MUTATIONS IN OTHER CANCERS (PART 2) GRAND AMPHI Chair: • Meena Upadhyaya, PhD
PLATFORM TALK: PEDF Dysregulation Promotes Proliferation and Invasion in 11:20 11:40 Malignant Melanoma with NF1 Driver Mutation Girish Patel, UK
PLATFORM TALK: Molecular and Functional Consequences of Somatic NF1 11:40 12:00 Mutations in Non-Small Cell Lung Cancers Eric Pasmant, France
12:00 13:00 Lunch Break / Ancillary Meeting: NF1-PN: Are we MEKing Progress? PETIT AMPHI
NF2 AND SCHWANNOMATOSIS STATE OF THE ART (Part 1) Clinical PETIT AMPHI 13:00 14:30 Co-chairs: • Michel Kalamarides, MD, PhD, Hopital Pitie, Salpetriere, France • Marco Giovannini, MD, PhD, UCLA, US
Living with NF2 – A Patient’s Perspective 13:00 13:05 McKinnon Galloway, US
NF2/SWN Diagnostic Criteria Revision 13:05 13:30 Gareth Evans, MD, University of Manchester, UK
Genetics of NF2/Schwannomatosis 13:30 14:00 Gareth Evans, MD, University of Manchester, UK
Current and Emerging Drug Treatment Options 14:00 14:30 Scott Plotkin, MD, PhD, Massachusetts General Hospital/Harvard University, US PARALLEL SESSION: GLIOMA – IN/OUT OF NF Co-chairs: 13:00 14:30 • Michael Fisher, MD, Children’s Hospital of Philadelphia, US GRAND AMPHI • David Walker, MD, Children’s Brain Tumour Center, UK • Antonio Iavarone, MD, Columbia University, US
New Insights into the Molecular and Cellular Pathogenesis of Optic Glioma 13:00 13:25 David Gutmann, MD, PhD, Washington University, US
PLATFORM TALK: Synodos for NF1 Glioma: In-Depth Genomic Characterization of Pediatric NF1-Associated Gliomas Reveals Recurrent Cooperating Mutations and an 13:25 13:45 Aggressive Tumor Subset David T. W. Jones, Germany
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 19 AGENDA Sunday · 4 November 2018
PLATFORM TALK: Larger Tumor Volume is Associated with Visual Acuity Loss and 13:45 14:05 Axonal Degeneration in Children with Optic Pathway Gliomas Secondary to NF1 Robert Avery, US
NF1 Optic Pathway Glioma: Treat or Observe? A Proposal for a Randomized 14:05 14:30 Selection of Treatment Versus Observation Amedeo Azizi, MD, PhD, University of Vienna, Austria PARALLEL SESSION: FUNCTIONAL GENETICS AND INTERPRETATION/CLASSIFICATION OF VARIANTS 13:00 14:35 Co-chairs: ROOM 201 • Akihiko Yoshimura, PhD, Keio University, Japan • Eric Legius, MD, PhD, University of Leuven, Belgium • Frans Verheijen, PhD, Erasmus MC, Netherlands
Splice Effect Assessment: The Important First Step of NF1 Variant Interpretation 13:00 13:25 Katharina Wimmer, PhD, University of Innsbruck, Austria
Functional Assessment of NF1 Missense Variants Identified in Individuals Suspected 13:25 13:50 of Neurofibromatosis Type 1 Rick van Minkelen, PhD, Erasmus University Medical Center, Netherlands
Effects of Mutations in SPRED1 on Legius Syndrome and Relationship to NF1 13:50 14:15 Akihiro Yoshimura, PhD, Keijo University, Japan
PLATFORM TALK: Neurofibromin a New SUMO Target Involved in Neurofibromatosis 14:15 14:35 Type 1 Disease Mohammed Bergoug, France
14:30 15:00 BREAK ROOM 8
NF2 AND SCHWANNOMATOSIS STATE OF THE ART 15:00 17:00 PETIT AMPHI (Part 2)
Treatment of Vestibular Schwannomas 15:00 15:20 Michael Link, MD, Mayo Clinic, US
Treatment of Meningiomas 15:20 15:40 John Golfinos, MD, New York University, Langone Medical Center, US
Hearing Rehabilitation 15:40 16:00 Olivier Sterkers, MD, PhD, Hôpital Pitié Salpêtrière, France
Treatment of Spinal Tumors 16:00 16:20 Andrew King, MD, University of Manchester, UK
Peripheral Nerve Schwannomas 16:20 16:40 Allan Belzberg, MD, Johns Hopkins University, US
Management of NF2 in China 16:40 17:00 H. Wu, MD, PhD, 9th People’s Hospital Shanghai Jiao Tong University School of Medicine, China PARALLEL SESSION: LEARNING DISABILITIES Co-chairs: 15:00 17:15 • Thijs van der Vaart, MD, PhD, Erasmus University Medical Center, Netherlands GRAND AMPHI • Jonathan Payne, PsyD, Murdoch Children’s Hospital, Australia • Nicole Ullrich, MD, PhD, Boston Children’s Hospital, Harvard University, US
Invited Speakers:
Multi Parametric Imaging and Behavior in an Early-Phase Mechanism Trial of 15:00 15:25 Simvastatin for NF1-Autism Shruti Garg, PhD, University of Manchester, UK
(cont. of above talk) 15:25 15:50 Stavros Stivaros, PhD, University of Manchester, UK
Social Behavior Deficits in a Spred1 Knockout Mouse Model of Legius Syndrome 15:50 16:15 Hilde Brems, PhD, University of Leuven, Belgium
20 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 AGENDA Sunday · 4 November 2018
PLATFORM TALK: Genotype and Behavioral Phenotypes in Children and Adolescents 16:15 16:35 with Neurofibromatosis Type 1 Andre Rietman, The Netherlands
PLATFORM TALK: Risk of Psychosocial, Cognitive and Health Impairments in Adult 16:35 16:55 Survivors of Childhood Glioma with Neurofibromatosis Type 1 Peter de Blank, United States
PLATFORM TALK: Understanding the NF1 Experience and the Priorities of the NF1- 16:55 17:15 Affected Individuals and Families for Cognitive and Social-Emotional Research Tess Kennedy, United States
PARALLEL SESSION: GENOTYPE/PHENOTYPE OF NF1 Co-chairs: 15:00 17:15 • Ludwine Messiaen, PhD, University of Alabama at Birmingham, US ROOM 201 • Hildegard Kehrer-Sawatzki, PhD, University of Ulm, Germany • Douglas Stewart, MD, National Cancer Institute, US
Invited Speakers:
Genotype-Phenotype Correlations in NF1: The Current State of Knowledge 15:00 15:25 Ludwine Messiaen, PhD, University of Alabama at Birmingham, US
Identification of the Factors Underlying the Cognitive Variance Associated with NF1 15:25 15:50 Ype Elgersma, PhD, Erasmus University Medical Center, Netherlands
Looking Beyond the Lamppost: Exome Sequencing in 92,296 Individuals in a Single US Healthcare System to Investigate Prevalence, Penetrance, and Phenotype in NF1 15:50 16:15 and Legius Syndrome Jung Kim, PhD, National Cancer Institute, US
PLATFORM TALK: Comprehensive Screening of NF1 Missense Mutations 16:15 16:35 James Walker, United States
PLATFORM TALK: NF1 Deficiency is Linked to Estrogen Signaling in Breast Cancer 16:35 16:55 Matthew Steensma, United States
PLATFORM TALK: Genetic Basis of Variable Clinical Expression in NF1 16:55 17:15 Audrey Sabbagh, France
17:15 17:25 POSTER ADVERTISEMENTS GRAND AMPHI
POSTER AREA – 17:25 18:55 CLINICAL POSTER SESSION ROOM 8 Monday · 5 November 2018
KEYNOTE LECTURE: Translational Mouse: From Genetic Models to the Mouse Hospital, 08:30 09:30 and Co-Clinical Trials for Understanding and Treatment of Cancer GRAND AMPHI Pier Paolo Pandolfi, MD, PhD, Director, Cancer Center and Cancer Research Institute – Beth Israel Deaconess Medical Center, Chief, Division of Genetics, Dept. Of Medicine, BIDMC, US
PLENARY SESSION: MICROENVIRONMENT Co-chairs: 09:30 11:00 • Piotr Topilko, PhD, INSERM, France GRAND AMPHI • D. Wade Clapp, MD, Indiana University, US • Alison Lloyd, PhD, University College, London, UK
The Cellular and Extracellular Microenvironment of Cutaneous Neurofibromas 09:30 09:55 Juha Peltonen, MD, PhD, University of Turku, Finland
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 21 AGENDA Monday · 5 November 2018
Cxcr3 Expressing Leukocytes are Necessary for Neurofibroma Formation in Mice 09:55 10:20 Nancy Ratner, PhD, Cincinnati Children’s Hospital, US
PLATFORM TALK: Dermal Fibroblast-Derived Exosomes Enhance Angiogenesis in a 10:20 10:40 Tissue-Engineered Skin Model of Neurofibromatosis Type 1 Vincent Roy, Canada
PLATFORM TALK: Targeting the Hyaluronan-Rich Peripheral Nerve Sheath Tumor 10:40 11:00 Microenvironment to Improve Drug Efficacy and Delivery Bryant John Keller, US
PARALLEL SESSION: NF2/SCHWANNOMATOSIS Co-chairs: PETIT AMPHI 09:30 10:50 • Michel Kalamarides, MD, PhD • Marco Giovannini, MD, PhD
PLATFORM TALK: A Phase 0 Pharmacodynamic and Pharmacokinetic Study of 09:30 09:40 Everolimus in Vestibular Schwannoma (VS) and Meningioma Patients Matthias A. Karajannis, US
PLATFORM TALK: A Single Arm Phase 2 Study of the Dual mTORC1/mTORC2 Inhibitor Vistusertib Provided on an Intermittent Schedule for Neurofibromatosis 2 09:40 09:50 Patients with Progressive or Symptomatic Meningiomas Scott Plotkin, US
PLATFORM TALK: Management of NF2-Associated Vestibular Schwannomas in 09:50 10:00 Children and Young Adults: Influence of Surgery on Tumor Volume and Growth Rate Isabel Gugel, Germany
Clinical Cases Panel Discussion • Thomas Roland, MD, New York University, US • Michael Link, MD, Mayo Clinic, US 10:00 10:50 • Brad Welling, MD, PhD, Massachusetts Eye and Ear Institute, Harvard University, US • Scott Plotkin, MD, PhD, Massachusetts General Hospital, US • John Golfinos, MD, New York University, US • Gareth Evans, MD, University of Manchester, UK
10:50 11:20 BREAK ROOM 8
PLENARY SESSION: PAIN AND PRURITIS IN NEUROFIBROMATOSIS 11:20 12:50 Co-chairs: GRAND AMPHI • Allan Belzberg, MD, Johns Hopkins University, US • Rosalie Ferner, MD, Guy’s and St. Thomas’ NHS Foundation Trust and King’s College, UK • Scott Plotkin, MD, PhD, Massachusetts General Hospital, US
Invited Speakers:
Pathophysiology of Itch 11:20 11:45 Laurent Misery, MD, Universite de Bretagne Occidentale, France
Therapeutic Management of Neuropathic Pain 11:45 12:05 Nadine Attal, MD, PhD, Universite Versailles Saint-Quentin, France
Biology of Neuropathic Pain and Potential Links to NF 12:05 12:30 Michael Caterina, MD, PhD, Johns Hopkins Medical Center, US
PLATFORM TALK: Molecular Mechanisms Underlying Schwannomatosis Pain 12:30 12:50 Larry Sherman, US
Lunch Break LATE BREAKING ABSTRACT 12:50 13:30 PLATFORM TALK: Risk of Contralateral Breast Cancer and Survival in Neurofibromatosis Type 1: A Five Country Cohort Study Gareth Evans, UK
22 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 AGENDA Monday · 5 November 2018
FONDATION ARC POUR LA RECHERCHE SUR LE CANCER KEYNOTE LECTURE: Targeted Therapy in Melanoma 13:30 14:30 Caroline Robert, MD, PhD, Head of Dermatology Unit, Institute Gustave-Roussy, GRAND AMPHI Villejuif, Board Member, European Association of Onco-Dermatology; Melanoma Board Secretary for European Organization for the Research and Treatment of Cancer; French Society of Dermatology and Venereology, AACR, ASCO, France
PLATFORM PRESENTATIONS – BASIC Co-chairs: PETIT AMPHI 14:30 16:30 • Yuan Zhu, PhD, Children’s National Medical Center, US • Conxi Lazaro, PhD, Institut Catala d’Oncologia, Spain
PLATFORM TALK: Transposon Mutagenesis and CRISPR/Cas9 Screening Reveal Pathways Driving Malignant Peripheral Nerve Sheath Tumor Development and 14:30 14:50 Maintenance German Luis Velez Reyes, US
PLATFORM TALK: Early Development and Autism Risk in Neurofibromatosis Type 1 14:50 15:10 Anna Kolesnik, UK
PLATFORM TALK: Antisense Directed Gene Therapy to Treat Intragenic 15:10 15:30 Neurofibromatosis Type I (NF1) Mutations Michael Daniel, US
PLATFORM TALK: Feasibility of Gene Replacement Therapy in NF1-related MPNST 15:30 15:50 Verena Staedtke, US
PLATFORM TALK: Preclinical Assessment of MEK1/2 Inhibitors for Neurofibromatosis Type 2-Associated Schwannomas Reveals Differences in Efficacy 15:50 16:10 and Drug Resistance Development Cristina Fernandez-Valle, US
PLATFORM TALK: Novel Drug Discovery for NF2-Deficient Meningiomas: Brigatinib 16:10 16:30 Causes Tumor Shrinkage in NF2-Deficient Meningiomas Long-Sheng Chang, US PLATFORM PRESENTATIONS – CLINICAL Co-chairs: GRAND AMPHI 14:30 16:30 • David Viskochil, MD, PhD, University of Utah, US • Patrick Combemale, MD, Cancer Research Center of Lyon, France
PLATFORM TALK: NF105: A Neurofibromatosis Clinical Trials Consortium (NFCTC) Phase II Study of Cabozantinib (XL184) for Neurofibromatosis Type 1 (NF1) 14:30 14:50 Associated Plexiform Neurofibromas Michael Fisher, US
PLATFORM TALK: Clinical Presentation and 5 Year Survival of 83 Individuals With NF1 Associated Malignant Peripheral Nerve Sheath Tumour in a National 14:50 15:10 Neurofibromatosis Centre Rosalie Ferner, UK
PLATFORM TALK: Risk of Subsequent Neoplasms (SNs) after a Primary Tumor in 15:10 15:30 Patients with NF1 Michael Fisher, US
PLATFORM TALK: Dorsal Root Ganglia Volume Differentiates Schwannomatosis and 15:30 15:50 Neurofibromatosis Type 2 Tim Godel, Germany
PLATFORM TALK: Use of an NF2 Genetic Severity Score to Incorporate Genotype into 15:50 16:10 Routine Patient Care Dorothy Halliday, UK
PLATFORM TALK: Smile Reanimation Using the Lengthening Temporalis Myoplasty 16:10 16:30 in Patients with Facial Paralysis Andre Panossian, US
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 23 AGENDA Monday · 5 November 2018
16:30 16:45 BREAK ROOM 8
PLENARY SESSION: MEDICAL AND SURGICAL CARE: A MULTIDISCIPLINARY APPROACH FOR NF 16:45 18:15 Co-chairs: GRAND AMPHI • Michel Kalamarides, MD, PhD, Hôpital Pitié Salpêtrière, France • Bruce Korf, MD, PhD, University of Alabama at Birmingham, US • Jaishri Blakeley, MD, Johns Hopkins University, US
NF1:
MPNST within plexiform in difficult location. Plan for surgery relative to oncology treatment (RT + chemo). Pre and post-op planning and outcome data. 16:45 17:00 Presenter: Rosalie Ferner Discussants: Allan Belzberg; AeRang Kim
Diffuse infiltrating of skin and soft tissue 17:00 17:15 Presenter: Bruce Korf (will ask Drs. Korf, Lantieri and Berman to prepare the case) Discussants: Laurent Lantieri, France; Mimi Berman, Australia
Deep plexiform lesion and the considerations for drug versus surgical approaches 17:15 17:30 Presenter: Pierre Wolkenstein (case prepared by Drs. Wolkenstein and Decq) Discussants: Michael Fisher; Philippe Decq NF2:
Decompression of VS in canal and staging relative to avastin or everolimus 17:30 17:45 Presenter: Michel Kalamarides (case to be prepared by Dr. Kalamarides and Karajannis) Discussants: Tom Roland; Mike Link; Matthias Karajannis
Meningiomatosis: rapidly progressive, FLAIR hyperintensity – resect. Slowly progressive, ICP, papilledema, subtle signs. What interventions and when. How to know. 17:45 18:00 Presenter: Scott Plotkin (case to be prepared by Drs. Plotkin, Kalamarides, Avery) Discussants: John Golfinos; Robert Avery; Michele Kalamarides SWN:
Surgery for painful schwannoma: how to identify the symptomatic lesion in a field of schwannomas (and possibly prior surgery) 18:00 18:15 Presenter: Leah Niegenpo (case to be prepared by Drs. Blakeley, Belzberg and Niegenpo) Discussants: Allan Belzberg; Jaishri Blakeley
PARALLEL SESSION: BIOLOGY OF NF2 AND SCHWANNOMATOSIS 16:45 18:40 Co-chairs: PETIT AMPHI • Helen Morrison, PhD, Leibniz Institute on Aging, Germany • Andrea McClatchey, PhD, Harvard University, US • Jeremie Vitte, PhD, UCLA, US
Invited Speakers:
Late Inactivation of Smarcb1 and Additional Nf2 Inactivation are Necessary for 16:45 17:10 Schwannoma Development in Schwannomatosis Patients Jeremie Vitte, PhD, UCLA, US
Correlation of the Tumor-Supressive Function and the Confirmation of NF2 Tina Izard, PhD, Professor, Cell Adhesion Laboratory, Department of Integrative 17:10 17:35 Structural and Computational Biology, Department of Immunobiology, The Scripps Research Institute, US
Peripheral Nerve Microenvironment/Schwann Cells 17:35 18:00 Alison Lloyd, PhD, University College, London, UK
PLATFORM TALK: LZTR1 is a MAM Protein Regulating Mitochondrial Calcium and 18:00 18:20 Respiration in Schwann Cells Asha Kiran Akula, Germany
PLATFORM TALK: Merlin/ERM Proteins Regulate Growth Factor-Induced Macropinocytosis and Receptor Recycling by Organizing the Plasma Membrane: 18:20 18:40 Cytoskeleton Interface Christine Chiasson-Mackenzie, US
24 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 AGENDA Tuesday · 6 November 2018
08:30 09:30 ORAL POSTER PRESENTATIONS GRAND AMPHI
KEYNOTE LECTURE: Plan France Médecine Génomique 2025 09:30 10:00 Yves Levy, MD, PhD, Chairman, CEO Institute National de la Sante et de la Recherche GRAND AMPHI Medicale, INSERM, France
PLENARY SESSION: NF: PAST, PRESENT AND FUTURE 10:00 11:10 Co-chairs: GRAND AMPHI • Annette Bakker, PhD, Children’s Tumor Foundation, US • Maurizio Clementi, MD, University of Padova, IT
10:00 10:05 NF from the Beginning to Now Invited Speakers:
10:05 10:20 Vincent Riccardi, MD, MBA, Neurofibromatosis Institute, US
10:20 10:35 Susan Huson, MD, Clinical Genetics Consultant, UK
10:35 10:40 Arvid Heiberg, MD, Oslo University Hospital, NOR
10:40 11:00 Discussion
11:00 11:40 BREAK GALERIE
PLENARY SESSION: 11:40 12:25 GRAND AMPHI NF: PRESENT, PAST AND FUTURE
NF Research and Treatment – On the Horizon and Beyond
Invited Speakers:
11:40 12:02 Luis Parada, PhD, Memorial Sloan Kettering Cancer Center, US
12:02 12:25 Gareth Evans, MD, St. Mary’s Hospital, University of Manchester, UK PLENARY SESSION: REVISION OF DIAGNOSTIC CRITERIA Co-chairs: • Eric Legius, MD, PhD, University of Leuven, Belgium GRAND AMPHI 12:25 13:45 • Scott Plotkin, MD, PhD, Massachusetts General Hospital/Harvard University, US • Gareth Evans, MD, St. Mary’s Hospital/University of Manchester, UK • Susan Huson, MD, Consultant Geneticist, UK
13:45 14:00 Congress Adjourned
REiNS Session (By Invitation) ROOM 101 15:00 18:00 Response Evaluation in Neurofibromatosis and Schwannomatosis
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 25 ANCILLARY MEETINGS
26 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018
Shortage of Qualified NF Providers: Addressing the Challenge
NF Clinics Satellite Session Saturday, November 3, 2018 5:45 pm - 7:00 pm Petit Amphitheater, Maison de la Chimie
5:45 – 6:00 Welcome and Introductions Annette Bakker, PhD President, Children’s Tumor Foundation
Heather Radtke, MS, CGC Children’s Tumor Foundation
6:00 – 6:15 Nurse-Led Telephone Clinics for Non-Complex NF Patients Sally Trump, RN Guy’s Hospital, London
6:15 – 6:30 Using Telehealth to Increase Access to NF Care Justin Jordan, MD, MPH Massachusetts General Hospital
6:30 – 6:45 Mentorship within the NF Clinic Network Nicole Ullrich, MD, PhD Boston Children’s Hospital
6:45 – 7:00 Training the Next Generation of NF Providers David Stevenson, MD Stanford University, Lucile Packard Children’s Hospital
RSVP to Heather Radtke – [email protected]
Refreshments and light snacks will be provided.
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 27 NF1-PN:NF1-PN: AreAre wewewe MEKMEKinging progress? TH MEKSUNDAYMEK 4 TH NOVEMBER,inging 2018 |progress? 12.00progress? - 13.00 SUNDAY 4 NOVEMBER, 2018 | 12.00 - 13.00 SUNDAY 4TH NOVEMBER, 2018 | 12.00 - 13.00 SUNDAYRoom:Room:SUNDAY Petit Petit 4 4THAmphitheatre, Amphitheatre,TH NOVEMBER, NOVEMBER, MaisonMaison 2018 2018 dede | lala|12.00 12.00Chimie, - -13.00 2813.0028 RueRue SaintSaint Dominique,Dominique, 75007, 75007, Paris Paris Room: Petit Amphitheatre, Maison de la Chimie, 28 Rue Saint Dominique, 75007, Paris Room:Room: Petit Petit Amphitheatre, Amphitheatre, Maison Maison de de la la Chimie, Chimie, 28 28 Rue Rue Saint Saint Dominique, Dominique, 75007, 75007, Paris Paris
12:0012:00 IntroductionIntroduction RogerRoger J J Packer Packer (chair) (chair) 12:00 Introduction Roger J Packer (chair) IntroductionIntroduction RogerRoger J JPacker Packer (chair) (chair) 12:0012:00 Gareth Evans & 12:0512:05 NF1-PN:NF1-PN: thethe currentcurrent treatmenttreatment landscape Gareth Evans & 12:05 NF1-PN: the current treatment landscape DavidGarethDavid Mowatt MowattEvans & David Mowatt 12:0512:05 NF1-PN:NF1-PN: the the current current treatment treatment landscape landscape GarethGareth Evans Evans & & 12:2012:20 FutureFuture treatment treatment optionsoptions for NF1-PN DavidBrigitteDavidBrigitte Mowatt Mowatt Widemann Widemann 12:20 Future treatment options for NF1-PN Brigitte Widemann 12:2012:20 FutureFuture treatment treatment options options for for NF1-PN NF1-PN BrigitteBrigitte Widemann Widemann 12:4012:40 QuestionQuestion && answeranswer sessionsession ChairChair 12:40 Question & answer session Chair 12:4012:40 QuestionQuestion & &answer answer session session ChairChair 13:0013:00 CloseClose ChairChair 13:00 Close Chair 13:0013:00 CloseClose ChairChair
This is a non-promotional meeting sponsored and organised by AstraZeneca. This is a non-promotional meeting sponsored and organised by AstraZeneca. This is a non-promotional meeting sponsored and organised by AstraZeneca. Veeva ID: Z4-12854 | Date of preparation: September 2018 | Date of expiry: September 2019 VeevaThisThis is ID: isa non-promotionalZ4-12854a non-promotional | Date of meetingpreparation: meeting sponsored sponsored September and 2018and organised organised | Date of byexpiry: by AstraZeneca. AstraZeneca. September 2019 Veeva ID: Z4-12854 | Date of preparation: September 2018 | Date of expiry: September 2019 VeevaVeeva ID: ID: Z4-12854 Z4-12854 | Date | Date of preparation:of preparation: September September 2018 2018 | Date | Date of expiry:of expiry: September September 2019 2019 Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS)
NF1-PN:NF1-PN: Are wewe Please join us for our REiNS session November 6th 3pm-6pm MEKMEKinging progress? https://ccrod.cancer.gov/confluence/display/REINS/Home TH SUNDAY 4 TH NOVEMBER,ing 2018 | 12.00progress? - 13.00 REiNS Publications: MEK Working groups and leaders: SUNDAY 4TH NOVEMBER, 2018 | 12.00 - 13.00 SUNDAY 4 NOVEMBER, 2018 | 12.00 - 13.00 Supplement 1 (Neurology 2013, Volume 81, Issue 21) TH • Tumor Imaging/Whole Body MRI Room:Room:SUNDAY Petit Petit 4Amphitheatre, Amphitheatre, NOVEMBER, MaisonMaison 2018 dede lala| 12.00Chimie, - 2813.0028 RueRue SaintSaint Dominique,Dominique, 75007, 75007, Paris Paris 1. Plotkin SR, Blakeley JO, Dombi E, et al. Achieving consensus for clinical Room: Petit Amphitheatre, Maison de la Chimie, 28 Rue Saint Dominique, 75007, Paris (Eva Dombi, Shivani Ahlawat) trials: the REiNS International Collaboration. Room: Petit Amphitheatre, Maison de la Chimie, 28 Rue Saint Dominique, 75007, Paris 2. Wolters PL, Martin S, Merker VL, et al. Patient-reported outcomes in • Functional outcomes neurofibromatosis and schwannomatosis clinical trials. (Scott Plotkin) 3. Fisher MJ, Avery RA, Allen JC, et al. Functional outcome measures for NF1- associated optic pathway glioma clinical trials. • Patient reported outcomes 12:0012:00 IntroductionIntroduction RogerRoger J J Packer Packer (chair) (chair) 4. Plotkin SR, Ardern-Holmes SL, Barker FG, et al. Hearing and facial function 12:00 Introduction Roger J Packer (chair) (Pam Wolters) outcomes for neurofibromatosis 2 clinical trials. 12:00 Introduction Roger J Packer (chair) GarethGareth Evans Evans & & • Visual outcomes 5. Dombi E, Ardern-Holmes SL, Babovic-Vuksanovic D, et al. 12:0512:05 NF1-PN:NF1-PN: thethe currentcurrent treatmenttreatment landscape Gareth Evans & Recommendations for imaging tumor response in neurofibromatosis 12:05 NF1-PN: the current treatment landscape DavidDavid Mowatt Mowatt (Rob Avery, Michael Fisher) clinical trials. David Mowatt 12:05 NF1-PN: the current treatment landscape Gareth Evans & David Mowatt 6. Widemann BC, Blakeley JO, Dombi E, et al. Conclusions and future 12:2012:20 FutureFuture treatment treatment optionsoptions for NF1-PN BrigitteBrigitte Widemann Widemann • Disease Biomarkers directions for the REiNS International Collaboration. 12:20 Future treatment options for NF1-PN Brigitte Widemann (Chetan Bettegowda, Oliver 12:20 Future treatment options for NF1-PN Brigitte Widemann Hanemann) Supplement 2 (Neurology 2016, Volume 82, Issue 7) 12:4012:40 QuestionQuestion && answeranswer sessionsession ChairChair 1. Widemann BC, Plotkin SR. Consensus for NF clinical trials: 12:40 Question & answer session Chair • Neurocognitive outcomes Recommendations of the REiNS collaboration (Supplement II). 12:40 Question & answer session Chair (Jennifer Janusz) 2. Wolters PL, Martin S, Merker VL, et al. Patient-reported outcomes of pain 13:0013:00 CloseClose ChairChair and physical functioning in neurofibromatosis clinical trials. 13:00 Close Chair • Cutaneous neurofibromas 3. Plotkin SR, Davis SD, Robertson KA, et al. Sleep and pulmonary outcomes 13:00 Close Chair (Ashley Cannon, Dominique for clinical trials of airway plexiform neurofibromas in NF1. Pichard) 4. Walsh KS, Janusz J, Wolters PL, et al. Neurocognitive outcomes in neurofibromatosis clinical trials: Recommendations for the domain of • Patient Representation attention. 5. Ahlawat S, Fayad LM, Khan MS, et al. Current whole-body MRI This is a non-promotional meeting sponsored and organised by AstraZeneca. (Scott Plotkin) This is a non-promotional meeting sponsored and organised by AstraZeneca. applications in the neurofibromatoses: NF1, NF2, and schwannomatosis. This is a non-promotional meeting sponsored and organised by AstraZeneca. Veeva ID: Z4-12854 | Date of preparation: September 2018 | Date of expiry: September 2019 For more information about the REiNS International 6. Hanemann CO, Blakeley JO, Nunes FP, et al. Current status and VeevaThis ID: is Z4-12854a non-promotional | Date of preparation: meeting sponsored September 2018and organised | Date of expiry: by AstraZeneca. September 2019 Veeva ID: Z4-12854 | Date of preparation: September 2018 | Date of expiry: September 2019 Collaboration and working groups, please contact recommendations for biomarkers and biobanking in neurofibromatosis. Veeva ID: Z4-12854 | Date of preparation: September 2018 | Date of expiry: September 2019 Raquel Thalheimer, [email protected]
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 29 PARTICIPANTS List published on 23 October 2018 Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL ABRAMOWICZ Anna Institute of Mother and Child [email protected] ACHARYA Jamuna AFRIDI Shazia AKSHINTALA Srivandana New York University AKULA Asha Kiran Leibniz Institute on Aging-Fritz Lipmann Institute [email protected] ALBERT Diane California State University, Sacramento [email protected] ALGHAMDI Mohammed King Abdulaziz university ALLAWAY Robert Sage Bionetworks [email protected] ALLEN Jeffrey [email protected] ALMEIDA Lilian Federal Fluminense University [email protected] ALVESHERE Andrea Western Illinois University [email protected] AMORIM Marta Centro Hospitalar Lisboa Central ANSTETT Kara NYU Langone Health [email protected] ARIMA Yoshimi Keio University School of Medicine ARMAND Marie-Laure APHP [email protected] ARMSTRONG Amy [email protected] ARNOLD Shelley The Children's Hospital at Westmead ATEN Emmelien ATTAL Nadine AUDIC Frédérique HU Henri Mondor AVERY Robert [email protected] AYTER Sükriye TOBB ETU University faculty of Medicine [email protected] AZIZI Amedeo Medical University of Vienna [email protected] BABOVIC-VUKSANOVIC Dusica Mayo Clinic BAKKER Annette BALDWIN Andrea BARNETT TAPIA Carolina BARTON Belinda The Children's Hospital at Westmead [email protected] BASALOM Shuaa [email protected] BAUER Mislen Nicklaus Children's Hospital [email protected] BELZBERG Allan Johns Hopkins Hospital BENEDETTI Helene CNRS BEN-SHACHAR Shay BERGOUG Mohammed Centre de biophysique moléculaire d'Orléans CBM- CNRS BERGSAKER David Kristian Frambu Resource Centre dor Rare Disorders [email protected] BERMAN Yemima [email protected] BHOURI Rakia Hopital Henri Mondor BILSKA Katarzyna Fundacja Instytutu Matki I Dziecka BIOTTEAU Maelle [email protected] BLAKELEY Jaishri Johns Hopkins University BLANCO Ignacio Fundació Institut D’investigació En Ciències De La Salut Germans Trias I Pujol (IGTP) BLANTON Ann SUNY Cortland [email protected] BLOK L.E.R. Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center BOKSTEIN Felix Tel Aviv Sourasky Medical Center [email protected] BONNE Nicolas CHU Lille [email protected] BONOMETTI Nadia General Hospital of Bolzano BORIC KRAKAR Marta UHC Sestre milosrdnice BORNHORST Miriam [email protected] BORRIE Sarah KU Leuven BOTERO-MENESES Juan Sebastián Universidad del Rosario BOULEY Stephanie Geisel School of Medicine at Dartmouth [email protected] BOYER John AstraZeneca BRACKMANN Derald House Clinic [email protected] BRAULT Jennifer Vanderbilt [email protected] BREKELMANS Carlijn BREMS Hilde University Hospital Leuven/ Catholic University Leuven BRIAND Nicola University Hospital Of Martinique BRIL Vera University Health Network BROSSEAU Jean-Philippe University of Texas Southwestern Medical Center [email protected] BROWDER Vidya Children's Tumor Foundation [email protected] BROWN Rebecca Memorial Sloan Kettering Cancer Center [email protected] BROWNE Katherine Oxford University Hospitals NHS Foundation Trust [email protected] BRYANT Nichole Greenville Health System [email protected] BURKE Joy University of Rochester [email protected] BURKITT WRIGHT Emma Manchester University NHS Foundation Trust CAMINADA Philippe Kinderspital Zürich [email protected] CAMPEN Cynthia Stanford [email protected] CANNON Ashley University of Alabama at Birmingham CARRIO Meritxell Fundació Institut D’investigació En Ciències De La Salut Germans Trias I Pujol (IGTP) CASALI Paolo G. Istituto Nazionale Tumori [email protected] CASSIMAN Catherine CASSINA Matteo University of Padova CASTELLANOS Elisabeth Germans Trias I Pujol Research Institute (IGTP) CATERINA Michael Johns Hopkins School of Medicine CAUBO Irene VUmc [email protected] CESARETTI Claudia [email protected] CHAIX Yves HU Henri Mondor CHANEY Katherine CHANG Long-Sheng Nationwide Children's Hosp and The Ohio State U CHEN Zhiguo UT Southwestern Medical Center [email protected] CHEN Hongsai [email protected] CHERFI EPOUSE SEDDIK Neila [email protected] CHIARA Federica University [email protected] CHIASSON-MACKENZIE Christine Massachusetts General Hospital/ Harvard Medical School CHISTOPRUDOVA Anna [email protected]
30 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 PARTICIPANTS Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL CHOCHOLOUS Monika Medical University Of Vienna [email protected] CHOI Byung Tae [email protected] CHOI Kwangmin Cincinnati Childrens Hospital CHOSIDOW Olivier HU Henri Mondor CHRIS Hammond King's College London CIAVARELLI Patricia ipmagna [email protected] CISZEWSKI Lukasz Royal Holloway, University of London [email protected] CLAPP David Indiana University, School of Medicine [email protected] CLEMENTI Maurizio genetics - University od Padova [email protected] COHEN Rony Schneider Children's Medical Center of Israel [email protected] COMBEMALE Patrick [email protected] COOVER Robert Cincinnati Childrens Hospital CORBETT Rob Canterbury District Health Board CORNFORD Melanie AstraZeneca CORNU Philippe COSSON TCHALIKIAN Aurelie COTA Bruno Hospital das Clínicas - Federal University of Minas Gerais [email protected] COTE Lucie CHU St-Justine [email protected] CRAIGEN William Baylor College of Medicine [email protected] CRAWFORD Hilda University of Sydney CROOK Ashley Royal North Shore Hospital [email protected] CUNHA Karin Universidade Federal Fluminense [email protected] CUNNINGHAM Tracy AstraZeneca DABSCHECK Gabriel [email protected] D'AGOSTINO Maria-Daniela DAHLHEIMER Tambra University of MN Masonic Children's Hospital [email protected] DANG Vi-An DANIEL Heather Indiana University [email protected] DANIEL Michael University of Alabama at Birmingham [email protected] DANNA Jeremy Aix-Marseille Université [email protected] DARRIGO Luiz DE BLANK Peter Cincinnati Children's Hospital Medical Center DE RAEDT Thomas CHOP/UPenn [email protected] DE WIT Marie-Claire Erasmus MC [email protected] DECQ Philippe HU Henri Mondor DEKKER Saskia Stichting Neurofibromatose [email protected] DENAYER Ellen DERDAK Joanne NIH DI GIANANTONIO Elena Medical Genetics - AO Padova [email protected] DICKINSON Carolyn University of Rochester DODD Rebecca University of Iowa DOMBI Eva National Cancer Institute [email protected] DONNELLY Deirdre Belfast Hospital & Social Care Trust [email protected] DÖRGELOH Beate Barbara Hannover Medical School DOSER Karoline Danish Cancer Society Research Center DOUCET Emilie Institut de Génomique Fonctionnelle DOZ Francois Institut Curie DRELICH Lauranne Universite de Lille DUBOIS Jean-Michel ANR DUCROT Stéphanie Laboratoire Parole et Langage [email protected] DUFF Chris Wythenshawe Hospital DUONG Tu Anh HU Henri Mondor DUTZMANN Christina Hannover Medical School EARL Dawn Seattle Children's Medical Center [email protected] ECONOMIDES Christina Squire Patton Boggs [email protected] EJERSKOV Cecilie EKLUND Erik ELGERSMA Ype Erasmus MC EMMANOUIL Beatrice [email protected] ENGELSON Celia NYU Langone Health EOLI Marica IRCCS C. Besta [email protected] ESHEL Rina Tel-Aviv Sourasky Medical Center ESPOSITO Silvia [email protected] EUBANKS-RUDD Jacquelyn Johns Hopkins University [email protected] EVANS Gareth University of Manchester EZZDINE Khaled HU Henri Mondor FARRELLY Ellyn LPCH/Stanford [email protected] FENCKOVA Michaela Radboud University Medical Center FERKAL Salah HU Henri Mondor FERLAZZO Mélanie CRCL inserm 1052 FERNANDEZ RODIGUEZ Juana Fundació Institut D’investigació En Ciències De La Salut Germans Trias I Pujol (IGTP) FERNANDEZ-VALLE Cristina UCF [email protected] FERNER Rosalie Guy's and St. Thomas' NHS Foundation Trust FIDLER Meredith Vertex Pharmaceuticals, Inc. [email protected] FISHER Carla AstraZeneca FISHER Michael Children's Hospital of Philadelphia FONSECA Sara FONTE Carla [email protected] FOREMAN Nicholas Children's Hospital Colorado [email protected] FRAMBU Livø Nyhus Frambu resource sentre for rare disorders [email protected] FRANCO Maria Oregon State University FRAYLING Ian [email protected] FRIEDRICH Reinhard E. Eppendorf University Hospital, Univ. of Hamburg [email protected] FUKUTA Hirotaka The Jikei University School of Medicine [email protected] FUMINI Valentina FUNALOT Benoît Henri Mondor GALLO Samanta Università degli Studi di Padova GARCIA JIMENEZ Belen Fundació Institut D’investigació En Ciències De La Salut Germans Trias I Pujol (IGTP)
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 31 PARTICIPANTS Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL GARDNER Kathy University of Pittsburgh [email protected] GARG Shruti University of Manchester GAZDOV Nikola GEL MORENO Bernat Fundació Institut D’investigació En Ciències De La Salut Germans Trias I Pujol (IGTP) GELLER Thomas Johns Hopkins All Children's Hospital [email protected] GEORGE Joshi Salford Royal Foundation Trust [email protected] GHOUALI Meriem Amina Centre Pierre et Marie curie GILLESPIE Andrea NIH [email protected] GIOVANNINI Marco Univeristy of California Los Angeles GODEC Abigail Washington University in St. Louis [email protected] GODEL Tim GODIN Fabienne GOETSCH Allison Ann and Robert H. Lurie Children's Hospital of Chicago [email protected] GOFFINET Laetitia CHRU de Nancy GOLDENBERG Marti GOLFINOS John NYU Langone Health GOMES Alicia UAB Medical Genomics Laboratory [email protected] GONZALEZ MUÑOZ Teresa Spanish National Cancer Research Centre (CNIO) [email protected] GOSLINE Sara Sage Bionetworks [email protected] GOTTESMAN Sally [email protected] GREENWOOD Robert University of North Carolina school of medicine [email protected] GRILL Jacques HU Henri Mondor GRIPP Karen A. I. duPont Hospital for Children/Nemours [email protected] GRISEL Coraline CHU Henri Mondor GROSS Andrea National Institutes of Health [email protected] GRUSS Joseph seattle childrens hospital [email protected] GUGEL Isabel GUILLAMO Jean Sebastien CHU Nimes GUTMANN David Washington University St. Louis [email protected] GUYOTA Jacques HU Henri Mondor HAAGERUP Annette Hospitalsenheden Vest | Aarhus University HAEBICH Kristina Murdoch Children's Research Institute [email protected] HAHN Cindy Texas Neurofibromatosis Foundation [email protected] HALLIDAY Dorothy Oxford University Hospital NHS FoundationTrust [email protected] HALLIDAY Jane Oxford University Hospitals NHS Foundation Trust [email protected] HAMOY-JIMENEZ Geohana UHN-Toronto General Hospital [email protected] HANEMANN Oliver Plymouth University Peninsula Schools of Medicine and Dentistry HARGRAVE Darren UCL Great Ormond Street Institute of Child Health HAWLEY Eric Indiana University School of Medicine [email protected] HEBRARD Berenice Hopital Henri Mondor HEIBERG Arvid Oslo Universitetssykehus, Rikshospitalet [email protected] HEISEY Kara [email protected] HELBING Dario Leibniz Institute on Aging HELLER Jonathan Teton Therapeutics HEMENWAY Molly Univ of Colorado, Children's Hospital Colorado [email protected] HENLEY Susie Guy's and St Thomas' NHS Foundation Trust HENSEN Erik LUMC- department of otorinolaryngology HERNANDEZ CHICO Concepcion Hospital Universitario Ramon y Cajal HIRBE Angela Washington University in St. Louis HOLLANTS Silke UZ Leuven HORVITZ Richard Children's Tumor Foundation [email protected] HU Xiaojie [email protected] HUSON Susan HUTNER Aline East Boston Neighborhood Health Center HUTSELL Stephanie IAVARONE Antonio Columbia University [email protected] IBRAHIM Nageatte Merck & Company, Inc [email protected] IDOWU Kazeem AstraZeneca IKUTA Kunihiro Nagoya University INGERICK Samantha Lucile Packard Children's Hospital at Stanford ISAKSON Sara University of Minnesota [email protected] IZARD Tina The Scripps Research Inst (TSRI) JACKSON Sadhana NIH [email protected] JACKSON Rhonda Johns Hopkins University JANUSZ Jennifer Children's Hospital Colorado [email protected] JEFFERS Lisa Belfast Health and Social Care Trust JESSEN Kristjan University College London [email protected] JIM Carly JOE Dorothy Guy's and St Thomas' NHS Foundation Trust [email protected] JONES Kristi Sydney Children's Hospitals Network - Westmead JONES David Hopp Children's Cancer Center (KiTZ) JORDAN Justin [email protected] JUNG Marie Juliane Leibniz Institute of Aging-Fritz Lipmann Insitute [email protected] KALAMARIDES Michel KALLIONPÄÄ Roope University of Turku [email protected] KAMALY-ASL Ian Royal Manchester Children's Hospital KAMEI Koji kamei clinic KANEPPELE Angelika KANG Joon Won Chungnam National University Hospital [email protected] KARAJANNIS Matthias Memorial Sloan Kettering Cancer Center [email protected] KARWACKI Marek Stowarzyszenie pacjentów z chorobą [email protected] Recklinghausena Alba Julia KAUFMANN Dieter [email protected] KAY Thomas St Vincent's Institute of Medical Research [email protected] KEHRER-SAWATZKI Hildegard [email protected] KELLER Bryant University of Minnesota -- Twin Cities [email protected] KELLETT Mark Manchester Centre for Clinical Neurosciences [email protected] KENBORG Line Danish Cancer Society Research Center [email protected]
32 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 PARTICIPANTS Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL KENNEDY Tess Children's National Health System [email protected] KENT Daniel AstraZeneca KESTERSON Bob University of Alabama at Birmingham [email protected] KHAN Sophia [email protected] KHIRE Uday AlloMek Therapeutics KI Dong Hyuk Dana-Farber Cancer Institute/ Harvard Medical School [email protected] KIM Aerang Children's National Medical Center [email protected] KIM Jung [email protected] KING Melissa Childrens Health System of Texas - Dallas KING Lorien St Joseph regional medical center KING Andrew Salford Royal Hospital [email protected] KIRK George AstraZeneca KISSIL Joseph KLEIN-TASMAN Bonita University of Wisconsin-Milwaukee [email protected] KLESSE Laura UT Southwestern KNIGHT Pamela Children's Tumor Foundation [email protected] KOETSIER Kimberley S. [email protected] KOGA Monji [email protected] KOHLER Catherine Merck [email protected] KOHLMEYER Jordan [email protected] KOKKINOU Eleftheria KOLESNIK Anna KONSITZKE Charles University of Wisconsin - Madison KORF Bruce KORYCINSKA Dorota Stowarzyszenie pacjentów z chorobą Recklinghausena Alba Julia KORYCINSKI Iwo University of Warsaw KORZENIEWSKA Justyna Childrens Memorial Health Institute [email protected] KRAKAR Goran Outpatient pediatric clinic dr Sabol KULKARNI Daks AstraZeneca KUROE Yuriko Gifu college of Nursing [email protected] KUSIKOVA Katarína Národný ústav detských chorôb LA ROSA Salvatore Children's Tumor Foundation LALANCETTE Eve [email protected] LALLEMAND Dominique Faculté de Pharmacie de Paris - Université Paris Descartes LAMBERT Laura UAB TGEMS LAMBROU Akis Moeses Nicosia General Hospital LAMONTAGNE Remy [email protected] LANDVALL Peter [email protected] LANGMEAD Shannon Johns Hopkins University LANTIERI Laurent HU Henri Mondor LAPOINTE Sarah LARABA Liyam Plymouth University [email protected] LARGAESPADA David University of Minnesota [email protected] LARIDON Annick Kempenhaeghe LARSSON Alex [email protected] LASCELLES Karine LAZARO Conxi Catalan Institute of Oncology, Barcelona, Spain [email protected] LE Lu University of Texas Southwestern Medical Center [email protected] LEE Beomhee [email protected] LEGIUS Eric University Hospital of Leuven LEMBERG Kathryn Johns Hopkins School of Medicine LEVY Yves INSERM [email protected] LEWIS Andrea Baylor College of Medicine LI Veetai [email protected] LIGUORO Dominique LIND Lene Holgersen Danish Association for NF [email protected] LINK Michael Mayo Clinic LION-FRANÇOIS Laurence [email protected] LIPKOVSKAYA Vera [email protected] LISTERNICK Robert Ann & Robert H. Lurie Children's Hospital of Chicago LLOYD Alison LOBON IGLESIAS Maria Jesus Faculté de Pharmacie de Paris - Université Paris Descartes LOOK Thomas Dana-Farber Cancer Institute [email protected] LU Jessica [email protected] LUCCHETTA Marta [email protected] LY Ina [email protected] MACCLATCHY Andrea Harvard Medical School [email protected] MACDONALD Maria MAERTENS Ophelia MAIA Rayana [email protected] MAJOR Philippe CHU Ste-Justine [email protected] MANSO Simone [email protected] MANSOURI Sheila University Health Network [email protected] MARCOTTE Erin University of Minnesota [email protected] MARIA ISIS Atallah Gonzalez Centre Hospitalier Universitaire Vaudois [email protected] MARIN Philippe Institut de Génomique Fonctionnelle, CNRS, INSERM, [email protected] Université de Montpellier MARTIN Yolanda Hospital Universitario Ramon y Cajal MARTIN Vanessa Childhood Tumour Trust [email protected] MARTIN Staci National Cancer Institute MASCO Acquah Francis Randhill Academy [email protected] MAUTNER Victor-Felix University Medical Centre Hamburg-Eppendorf MAZIERO Stéphanie Inserm UMR1214 ToNIC [email protected] MAZUELAS Helena Gemans Trias i Pujol Research Institute MCKINNON Galloway CTF Ambassador [email protected] MERKER Vanessa Massachusetts General Hosptal [email protected] MESSIAEN Ludwine
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 33 PARTICIPANTS Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL MEUDT Jennifer University of Wisconsin - Madison MICHAEL George Université Lumière - Lyon 2 MIHAI Adela AstraZeneca [email protected] MILLER David Boston Children's Hospital MILLS Kristen Rensselaer Polytechnic Institute [email protected] MISERY Laurent University Hospital of Brest [email protected] MO Juan UT Southwestern Medical Center [email protected] MOERTEL Christopher University of Minnesota MOHAMMED Taqee MAK College of Pharmacy and MAK Labs [email protected] MOHAN Midhun MOOTS Paul Vanderbilt University Medical Center [email protected] MORALES Susana MORENO Jean-Christophe HU Henri Mondor MORRIS Katrina Royal North Shore Hospital MORRISON Helen Leibniz Institute on Aging MOWATT David MUELLER Amy [email protected] MUND Julie Indiana University School of Medicine [email protected] MURPHY Christopher NATACCI Federica Fondazione IRCCS Cà Granda Ospedale [email protected] Maggiore Policlinico NEMMI Federico Inserm UMR1214 [email protected] NGHIEMPHU Phioanh Leia UCLA [email protected] NGUYEN Remy Tuan Quang NIEVO Marco Children's Tumor Foundation [email protected] NIKITIN Pavel Burdenko National Medical Research Center [email protected] of Neurosurgery NIKRAD Julia University of Minnesota NISHIDA Yoshihiro Nagoya University Hospital [email protected] NOVARESIO Zuzana National Institute of Child Diseases OOSTENBRINK Rianne ErasmusMC [email protected] ORLIC Ivan Ban Can ORTONNE Nicolas HU Henri Mondor OSTROW Kimberly Johns Hopkins University PACKER Roger Children's National Health System [email protected] PANCZA Patrice Children's Tumor Foundation [email protected] PANDOLFI Pier Paolo Beth Israel Deaconess Medical Center/ [email protected] Harvard Medical School PANOSSIAN Andre [email protected] PARADA Luis Memorial Sloan Kettering Cancer Center PARFAIT Béatrice Faculté de Pharmacie de Paris - Université Paris Descartes PARK Su Jung Indiana university School of Medicine PARKINSON David Plymouth University PARRA Alba [email protected] PARROZZANI Raffaele University of Padova [email protected] PASMANT Eric Cochin Hospital and Paris Descartes University [email protected] PATEL Tina Indigo Medical Limited [email protected] PATEL Girish Cardiff University [email protected] PAVONE Rossana PAYNE Jonathan Murdoch Children's Research Institute [email protected] PECRIAUX Adrien HU Henri Mondor PELTONEN Sirkku University of Turku and Turku University Hospital PELTONEN Juha University of Turku [email protected] PEMOV Alexander PERREAULT Sebastien PETERS Hartmut Charité- Universitaetsmedizin Berlin [email protected] PETERSEN Andrea Randall Children's Hospital [email protected] PICHARD Dominique NIAMS, National Institutes of Health PICULELL Katherine Boston Children's Hospital [email protected] PINEAU Marc [email protected] PINNER Jason Sydney Children's Hospital PINSON Stéphane Hospices Civils de Lyon - GHE PIOTROWSKI Arkadiusz Medical University of Gdansk PIOTROWSKI Anna MSKCC [email protected] PIRES Ana Elisabete PIWOWARSKI Alison University of Maryland Medical Center [email protected] PLOTKIN Scott [email protected] POND Dinel Children's Minnesota [email protected] POPPLEWELL Linda Royal Holloway, University of London [email protected] POST Leonard Vivace Therapeutics POYHONEN Minna Helsinki University Hospital [email protected] PRATILAS Christine Johns Hopkins University PRIDE Natalie The Children's Hospital at Westmead [email protected] and University of Sydney PRIMIANO Michelle New York Presbyterian - Columbia University Medical Center PRZYPYSZNY Michele PRZYPYSZNY Children's Tumor Foundation [email protected] QUELLE Dawn University of Iowa [email protected] RABKIN Samuel Massachusetts General Hospital/Harvard Medical School RADOMSKA Katarzyna [email protected] RADTKE Heather Children's Tumor Foundation / Children's Hospital of [email protected] Wisconsin / UW Cancer Center at ProHealth Care RAHAMANI Mohammed Al MAK College of Pharmacy and MAK Labs [email protected] RANDOLPH Linda Children's Hospital Los Angeles RASOLA Andrea University of Padova RATNER Nancy Cincinnati Childrens Hospital [email protected] RAY Lauren Littlest Tumor Foundation REDMAN Carolyn Oxford University Hospitals NHS Foundation Trust [email protected] REICHERT Jamie University of Wisconsin - Madison
34 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 PARTICIPANTS Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL REILLY Karlyne NCI, CCR [email protected] REINBERG Danny Howard Hughes Medical Institute [email protected] at NYU School of Medicine RENNIE Andrea The Children's Hospital of Philadelphia RENSTRUP Jens SpringWorks Therapeutics [email protected] REZENDE Nilton Federal University of Minas Gerais [email protected] RHODES Steven Indiana University School of Medicine, Riley Hospital for Children RICCARDI Vincent The Neurofibromatosis Institute [email protected] RIELEY Margaret Northern Light Eastern Maine Medical Center [email protected] RIETMAN Andre Erasmus Medical Centre ROBERT Caroline Gustave Roussy [email protected] ROBINSON Louise Royal Manchester Children's Hospital RODRIGUES Luiz Federal University of Minas Gerais - Brazil ROLAND, JR. J. Thomas NYU Langone Health [email protected] ROS PENA Andreas Fundació Institut D’investigació En Ciències De La Salut Germans Trias I Pujol (IGTP) RÖSCHEL Anna [email protected] ROSENBAUM Thorsten Sana Kliniken Duisburg [email protected] ROSENMAYR Verena Medical University of Vienna ROSSER Tena [email protected] ROY Vincent [email protected] RUBENSTEIN Allan [email protected] RUKIN GOLD Deborah Rainbow Babies and Children's Hospital RUSTAD Cecilie F. Oslo University Hospital SABBAGH Audrey Université Paris Descartes SAKKA Andreas Indigo Medical [email protected] SALAMON Johannes Martin University Hospital Hamburg-Eppendorf [email protected] SALETTI Veronica [email protected] SALVADOR Hector [email protected] SAMPSON Jan California State University Sacramento SARNOFF Herb Infixion Bioscience, Inc. [email protected] SATO Aimee Seattle Children's Hospital [email protected] SAYA Hideyuki Keio University School of Medicine SCHATZ Ulrich Medical University Innsbruck [email protected] SCHEFFZEK Klaus SCHENCK Annette Radboud University Medical Center [email protected] SCHMIDT Christa St. Olav hospital Trondheim SCHOOK Lawrence University of Illinois SCHORRY Elizabeth Cincinnati Children's Hospital Medical Center [email protected] SEHAILIA Salaheddine Tebessa [email protected] SEHESTED Astrid Copenhagen University Hospital, Rigshospitalet SELLARS Elizabeth UAMS [email protected] SERRA Eduard The Institute for Health Science Research Germans [email protected] Trias i Pujol (IGTP) SHAH Savan [email protected] SHANMUGANAYAGAM Dhanansayan University of Wisconsin - Madison [email protected] SHAO Diane Boston Children's Hospital [email protected] SHARAFI Parisa TOBB University of Economics and Technology, [email protected] Faculty of Medicine SHARMA Sheel [email protected] SHARP Gregory Arkansas Children's Hospital [email protected] SHEA Stephanie Childrens Hospital Colorado SHEERIN Una-Marie SHERMAN Larry Oregon Health & Science University SHIBUYA Peter Children's National Medical Center [email protected] SIGURDSSON Björn SIQVELAND Elizabeth Children's Minnesota SLIWINSKI Agnes HU Henri Mondor SLOAN Lindsey Johns Hopkins [email protected] SLOBOGEAN Bronwyn Johns Hopkins University [email protected] SMITH Miriam The University of Manchester SMITH Lisa Indiana University/Riley Hospital for Children SMITH Katherine Children's Hospital of Philadelphia SO Karen AstraZeneca SOKOLOWSKI Mark University of Minnesota [email protected] SOMMER Katherine University of Minnesota Physicians SONAWANE Poonam SORAGNI Alice UCLA SOUZA Marcio Federal University of Minas Gerais [email protected] SOUZA Juliana SPRUIJT Liesbeth Radboud University Centre Nijmegen, the Netherlands STACK Kathleen Oishei Children's Hospital of Buffalo STAEDTKE Verena Johns Hopkins Univeristy STAPLETON Stacie Johns Hopkins All Childrens Hospital STEENSMA Matt Spectrum Health Medical Group [email protected] STEMMER-RACHAMIMOV Anat MGH [email protected] STEPANOVA Dina Pirogov Russian National Research Medical University [email protected] STERKERS Olivier Hopital Beaujon [email protected] STERN Edward Children's Tumor Foundation [email protected] STEVENSON David Stanford University STEWART Douglas National Cancer Institute [email protected] STIVAROS Stravos Royal Manchester Children’s Hospital [email protected] SU Wang University of British Columbia SUN Daochun Memorial Sloan Kettering Cancer Center SUPPIAH Suganth University of Toronto [email protected] SVERAK Pavlina University of Minnesota [email protected] TAAL Walter Erasmus MC [email protected] TAILHARDAT Françoise HU Henri Mondor TAMULA Mary Anne Leidos Biomedical Research, Inc.
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 35 PARTICIPANTS Attendees List – 2018 Joint Global Neurofibromatosis Conference
LAST FIRST INSTITUTION EMAIL TAZIR Yasmina AstraZeneca TEMIZ Nuri University of Minnesota [email protected] TERANISHI Yu The University of Tokyo [email protected] THALHEIMER Raquel [email protected] THERRIEN Janet Kelly Government Solutions THOMPSON Heather California State University, Sacramento TINSCHERT Sigrid Iflb Berlin TOLEDANO ALHADEF Hagit [email protected] TOMKINS Helen [email protected] TOPILKO Piotr INSERM [email protected] TREVISSON Eva University of Padova [email protected] TRINGALI Stephane HU Henri Mondor TRUMP Sally [email protected] ULLRICH Nicole Boston Children's Hospital/Harvard Medical School UPADHYAYA Meena Cardiff University [email protected] VAASSEN Pia Sana Kliniken [email protected] VALLEE Beatrice CNRS VAN ABEELEN A.M. Kempenhaeghe VAN DEN ENDE Jenneke university hospital Antwerp [email protected] VAN DER VAART Thijs Erasmus MC [email protected] VAN DIJK Sarah Erasmus MC VAN IERLAND Yvette ErasmusMC VAN ITTERSUM Emilie Stichting Neurofibromatose [email protected] VAN MATER David [email protected] VAN MINKELEN Rick Erasmus Medical Center VARAN Ali [email protected] VASILJEVSKI Emily Kids Research, The Children's Hospital at Westmead [email protected] VASSALLO Grace Nationally NHS Commissioned NF1 Service [email protected] VELEZ REYES German University of Minnesota VERHEIJEN Frans Erasmus Medical Center, Rotterdam VERMA Sharad Johns Hopkins University [email protected] VIDAUD Michel Faculté de Pharmacie de Paris - Université Paris Descartes VIDAUD Dominique HU Henri Mondor VISKOCHIL David University of Utah [email protected] VITTE Jeremie UCLA VOGELS Annick University of Leuven [email protected] VON MALTZAHN Julia Leibniz Institute on Aging VUKELJ Simon Children's Tumor Foundation [email protected] WAHLLAENDER Ute WALKER Jim Mass General Hosp [email protected] WALKER David University of Nottingham [email protected] WALLACE Stephanie Seattle Children's Hospital WALLIS Deeann [email protected] WALSH Karin [email protected] WANG Zhihong Wayne State University WANG Xia Moffitt Cancer Center WANG Zhaoyan [email protected] WANG Min [email protected] WASIK Martin WASSEF Michel Faculté de Pharmacie de Paris - Université Paris Descartes WATERFALL Joshua Institut Curie [email protected] WATSON Adrienne Recombinetics Inc. [email protected] WEIMER Jill [email protected] WEINBERG Hubert [email protected] WEISS Brian Cincinnati Children's Hospital Medical Center [email protected] WELLING Duane Bradley Massachusetts Eye and Ear [email protected] WHITCOMB Trish NCI [email protected] WHITE Leah WICK Myra Mayo Clinic [email protected] WICKISER Jonathan University of Texas Southwestern WIDEMANN Hilla WIDEMANN Brigitte NCI [email protected] WIEMER Erik Erasmus University Medical Center / [email protected] Erasmus MC Cancer Institute WILLIAMS Victoria Guy's Hospital [email protected] WILLIAMS Kyle [email protected] WIMMER Katharina [email protected] WIRTANEN Tracy WOJTKIEWICZ Agnieszka [email protected] WOLKENSTEIN Pierre Hôpital Henri-Mondor [email protected] WOLTERS Pamela National Cancer Institute [email protected] WONG Claire Royal North Shore Hospital [email protected] WRIGHT Anna University of Oklahoma Health Sciences Center [email protected] WU Jianqiang WU Hao WU Natalie Cincinnati Children's Hospital Medical Center YAGOUBI Merouane Chu mustapha pacha YATES Charles Indiana University School of Medicine YOHAY Kaleb NYU Langone Health [email protected] YOSHIDA Yuichi [email protected] YOSHIMURA Akihiko Keio University [email protected] YOSHINAGA Akiko Kyoto University Hospital [email protected] YOSHINAGA Daisuke [email protected] YOUNG Melissa Kaleida Health [email protected] ZELCER Shayna [email protected] ZELLER Jacques ZHANG Xiyuan National Cancer Institute [email protected] ZHANG Xiaochun Washington University [email protected] ZHU Yuan Children's National Medical Center at Washington DC
36 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 Conference Room
Poster Area (Basic)
Preview Room Poster Area (Basic)
Poster Area (Clinic)
Entrance Welcome Cloakroom Area
28 rue Saint-Dominique 75007 Paris Tél : 01 40 62 27 00 Fax : 01 45 55 98 62 Mail : [email protected] www.maisondelachimie.com
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 37 SOCIAL PROGRAM Networking Session, Concert & Dinner Cruise
Networking Session Friday, 2 November, late afternoon, at the Congress Center Included in the registration fees
Concert Saturday, 3 November, 20:30, at Casino de Paris
Congress Dinner on the River Seine Monday, 5 November A wonderful way to enjoy the capital in a friendly, relaxed atmosphere while admiring its monuments and bridges at night time: come and join us for a dinner cruise on the Seine River! While savoring refined French cuisine on board and smoothly cruising along the river, you will have magnificent views over of Paris and its beautiful historical landmarks. The Dinner Cruise will be an excellent opportunity for delegates to renew old acquaintances or establish new friendships. Limited number of participants. Price per person: 120€ (incl. VAT)
For more information, contact [email protected]
38 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 NEARBY RESTAURANTS Notable Dining Near the Maison de la Chimie
Chez Françoise Aérogare des Invalides 75007 Paris E-mail : [email protected] Tel. : 01 47 05 49 03 http://chezfrancoise.com/
Brasserie Le Bourbon 1, place du Palais Bourbon 75007 Paris Tel. : 01 45 51 58 27 https://www.brasserie-bourbon.fr/
Loiseau Rive Gauche 5 rue de Bourgogne 75007 Paris Tel. : +33145517942 Fax : +33147537956 http://www.bernard-loiseau.com/en
Restaurant l’Assemblée 13, rue de Bourgogne 75007 PARIS Tel. : 01 45 51 62 07 http://www.hoteldevarenne.com/restaurant-l-assemblee~pid971
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 39 2019 NF CONFERENCE SEPT. 21 - 24 2019 NF FORUM SEPT. 20-22
HYATT REGENCY SAN FRANCISCO
40 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 SPECIAL THANKS TO THE 2018 JOINT GLOBAL CONFERENCE CO-CHAIRS
Marco Giovannini, MD, PhD Ludwine Messiaen, PhD University of California Los Angeles, US University of Alabama at Birmingham, US
Michel Kalamarides, MD, PhD Eric Pasmant, PhD Hôpital de la Pitié-Salpêtrière, AP, HP, France Hôpital Cochin, Université Paris Descartes, France
Lu Le, MD, PhD Pierre Wolkenstein, MD, PhD University of Texas, Southwestern, US Hôpital Henri-Mondor, France
OUR THANKS ALSO TO THE FOLLOWING INDIVIDUALS FOR THEIR EFFORTS:
Scientific Advisory Committee Dusica Babovich-Vuksavonich Lu Le Nilton Rezende Annette Bakker Robert Listernick Vincent Riccardi Yemima Berman Staci Martin Tena Rosser Thomas DeRaedt Ludwine Messiaen Larry Sherman Gareth Evans Christopher Moertel David Stevenson Cristina Fernandez-Valle Helen Morrison Piotor Topilko Rosalie Ferner Laura Papi Nicole Ullrich David Gutmann Eric Pasmant Thijs Van der Vaart Susan Huson Jonathan Payne Ana-Maria Vranceanu Hhildegard Kehrer-Sawatski Sirkku Peltonen Karin Walsh Pamela Knight Scott Plotkin Kaleb Yohay Salvatore LaRosa Heather Radtke Akihiko Yoshimura
Session Chairs Marie-Laure Armand Xiaojie Hu Juha Peltonen Dusica Babovic -Vuksavonic Antonio Iavarone Scott Plotkin Annette Bakker Michel Kalamarides Nancy Ratner Alan Belzberg Hildegard Kehrer-Sawatzki Karlyne Reilly Yemina Berman Bruce Korf Eduard Serra Jaishri Blakeley Laurent Lantieri Anat Stemmer-Rachamimov D. Wade Clapp Conxi Lazaro Douglas Stewart Maurizio Clementi Lu Le Piotr Topilko Patrick Combemale Eric Legius Nicole Ullrich Hilda Crawford Alison Lloyd Meena Upadhyaya Karin Cunha Ophelia Maertens Thijs van der Vaart Thomas DeRaedt Staci Martin Franz Verheijen Rosalie Ferner Andrea McClatchey David Viskochil Michael Fisher Ludwine Messiaen Jeremie Vitte Reinhard Friedrich Helen Morrison David Walker Marco Giovannini Eric Pasmant Akihiko Yoshimura Joseph Gruss Jonathan Payne Yuan Zhu
2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 | 41 SPONSORS Sponsor List
The Joint Global NF Conference organizers would like to thank our sponsors
PLATINUM SPONSOR
BRONZE SPONSORS
42 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018 NF1-PN: Are we MEKing progress?
SUNDAY 4TH NOVEMBER, 2018 | 12.00 - 13.00 Room: Petit Amphitheatre, Maison de la Chimie, 28 Rue Saint Dominique, 75007, Paris
12:00 Introduction Roger J Packer (chair)
Gareth Evans & 12:05 NF1-PN: the current treatment landscape David Mowatt
12:20 Future treatment options for NF1-PN Brigitte Widemann
12:40 Question & answer session Chair
13:00 Close Chair
This is a non-promotional meeting sponsored and organised by AstraZeneca.
Veeva ID: Z4-12854 | Date of preparation: September 2018 | Date of expiry: September 2019
UEMS Accreditation:
The 2018 Joint Global Neurofibromatosis Conference, Paris, France, 02/11/2018-06/11/2018 has been accredited by the European Accreditation Council for Continuing Medical Education (EACCME®) with 27 European CME credits (ECMEC®s). Each medical specialist should claim only those hours of credit that he/she actually spent in the educational activity.
Through an agreement between the Union Européenne des Médecins Spécialistes and the American Medical Association, physicians may convert EACCME® credits to an equivalent number of AMA PRA Category 1 CreditsTM. Information on the process to convert EACCME® credit to AMA credit can be found at www.ama-assn.org/education/earn-credit-participation-international-activities.
Live educational activities, occurring outside of Canada, recognised by the UEMS-EACCME® for ECMEC®s are deemed to be Accredited Group Learning Activities (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada. ORGANIZING SECRETARIAT NEUROFIBROMATOSIS 2018 MCI France - 25, rue Anatole France 92300 Levallois-Perret - France
Phone: +33 1 53 85 82 63 Fax: +33 1 53 85 82 83 Email: [email protected] [email protected]
www.nf-paris2018.com 44 | 2018 Joint Global Neurofibromatosis Conference · Paris, France · November 2-6, 2018