Neuro-Oncology Perspectives in Neurofibromatosis Type 1: New Horizons in the Era of MEK Inhibition

December 3, 2020

Access the presentation on Zoom:

Meeting ID: 935 8290 1578
Passcode: 441826


This educational initiative is targeted to neuro-oncologists, pediatric neuro-oncologists, neurosurgeons, neuroradiologists, medical oncologists, neurologists, neuro-oncology researchers, and other members of the neuro-oncology team who care for patients with neurofibromatosis type 1 (NF1). Designed and developed to provide an exhaustive overview of novel and emerging data, as well as establish a foundational context of NF1 disease state complexity, this activity will begin by identifying keystone diagnostic signs, symptoms, and clinical hallmarks in pediatric patients, and describe the genetic etiologies and multi-system pathophysiologies that have historically made NF1-associated plexiform neurofibromas so difficult to treat. Attendees will then gain insights from expert faculty regarding the pivotal shortcomings of traditional treatment approaches and the new horizons that have accompanied the burgeoning emergence of novel pharmacologic therapies, most notably MEK inhibitors. Finally, top-level clinical experts will guide attendees through a case-based section that explores the vital role of the neuro-oncology clinician and researcher in the evolving NF1 management paradigm, and provides activity participants an opportunity to design evidence-based treatment regimens for symptomatic, inoperable NF1-related tumors.

Presented by Creative Educational Concepts, Inc (CEC Oncology) and supported by an independent educational grant from AstraZeneca.

Target Audience

This educational initiative is targeted to neuro-oncologists, pediatric neuro-oncologists, neurosurgeons, neuroradiologists, medical oncologists, neurologists, neuro-oncology researchers, and other members of the interprofessional, multi-disciplinary neuro-oncology team.

Learning Objectives

At the conclusion of this live internet activity, participants will gain the skills and/or knowledge to:
  • Identify the keystone diagnostic symptomatology and clinical hallmarks of neurofibromatosis type 1 (NF1), especially in the pediatric patient population. 
  • Describe the genetic origins and the complex, multi-system pathophysiology of NF1, with an emphasis on the clinical gravity and historical intractability of plexiform neurofibromas.
  • Review traditional approaches for treating plexiform neurofibromas in patients with NF1, including surgery, radiation, and surveillance, and highlight the crucial clinical chasms that remain.
  • Examine emerging pharmacologic therapies for managing symptomatic, inoperable plexiform neurofibromas, including an appraisal of completed and ongoing clinical trials and recent FDA approvals, with a focus on MEK inhibition.
  • Evaluate the role of the neuro-oncology clinician and researcher in developing and delivering novel, cutting-edge NF1 therapeutic approaches and galvanizing ongoing renewal of the treatment calculus.
  • Using a case-based format, design evidence-based management strategies for clinically significant, non-surgical, NF1-associated plexiform neurofibromas and low-grade gliomas.


Additional Information

Course summary
Available credit: 
  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Participation
Course opens: 
Course expires: 
Event starts: 
12/03/2020 - 7:00pm EST
Event ends: 
12/03/2020 - 8:30pm EST

7:00 PM-7:10 PM EST:

Welcome and Introductions/Pre-test
Jaishri Blakeley, MD

7:10 PM-7:25 PM EST:

Embracing Complexity: The Etiology, Pathophysiology, and Clinical Manifestations of Neurofibromatosis Type 1
Bruce R. Korf, MD, PhD (Activity Chair)

7:25 PM–7:50 PM EST:

Navigating Novelty in Plexiform Neurofibroma Management: An Exploration of Emerging Data and Adaptive Strategies
Andrea M. Gross, MD

7:50 PM-8:20 PM EST:

Bridging Chasms in NF1: The Pivotal Placement of the Neuro-Oncology Team in the New Paradigm
Jaishri Blakeley, MD

8:20 PM-8:30 PM EST:

Conversation with the Experts: Audience Q&A/Post-test
Faculty Panel Moderated by Dr. Korf

Live Virtual Event
as part of the 2020 Society for Neuro-Oncology (SNO) Annual Meeting
7:00 PM EST
United States

Bruce R. Korf, MD, PhD
Activity Chair
Associate Dean for Genomic Medicine
School of Medicine
Wayne H. And Sara Crews Finley Endowed Chair in Medical Genetics
University of Alabama at Birmingham (UAB)
Chief Genomics Officer, UAB Medicine
Birmingham, AL

Dr. Korf is the Associate Dean for Genomic Medicine, School of Medicine; Chief Genomics Officer, UAB Medicine; Wayne H. and Sara Crews Finley Endowed Chair in Medical Genetics, Professor of Genetics, Co-Director of the UAB-HudsonAlpha Center for Genomic Medicine, Associate Director for Rare Diseases, Hugh Kaul Personalized Medicine Institute and editor-in-chief of the American Journal of Human Genetics.  He is a medical geneticist, pediatrician, and child neurologist, certified by the American Board of Medical Genetics and Genomics (clinical genetics, clinical cytogenetics, and clinical molecular genetics), American Board of Pediatrics, and American Board of Psychiatry and Neurology (child neurology). Dr. Korf is past president of the Association of Professors of Human and Medical Genetics, past president of the American College of Medical Genetics and Genomics, and current president of the ACMG Foundation for Genetic and Genomic Medicine.  He has served on the Board of Scientific Counselors of the National Cancer Institute and the National Human Genome Research Institute at the NIH. His major research interests are molecular diagnosis of genetic disorders and the natural history, genetics, and treatment of neurofibromatosis. He serves as principal investigator of the Department of Defense funded Neurofibromatosis Clinical Trials Consortium, the Alabama Genomic Health Initiative, and the Southern All of Us Network.  He is co-author of Human Genetics and Genomics (medical student textbook, now in fourth edition), Medical Genetics at a Glance (medical student textbook, now in third edition), Emery and Rimoin’s Principles and Practice of Medical Genetics (now in 6th edition), and Current Protocols in Human Genetics.

Jaishri Blakeley, MD
Director, The Johns Hopkins Comprehensive Neurofibromatosis Center
Professor of Neurology, Neurosurgery, and Oncology
The Johns Hopkins University
Baltimore, MD

Dr. Jaishri Blakeley is the Marjorie Bloomberg Tiven Professor of Neurofibromatosis in Neurology, Oncology and Neurosurgery, director of the Johns Hopkins Comprehensive Neurofibromatosis Center, and the Neurofibromatosis Therapeutic Acceleration Program (NTAP). NTAP was founded in 2012 with the goal of dramatically shifting the landscape of clinical care for people with Neurofibromatosis Type 1 associated plexiform and cutaneous neurofibromas via the core tenants of: focus on therapeutics, foster collaboration, facilitate open and timely sharing of results, and streamline the research process. Dr. Blakeley’s research expertise is in the development of clinical trials for nervous system tumors and specifically, clinical-translational studies including tumor pharmacokinetic and pharmacodynamic investigations, imaging biomarkers and incorporation of patient focused, functional endpoints. Her research, mentorship and programmatic efforts are all in the service of improving outcomes for the patients for whom she is honored to provide care for as an active clinician in neuro-oncology.

Andrea M. Gross, MD
Assistant Research Physician, Pediatric Oncology Branch
National Institutes of Health, Center for Cancer Research
Bethesda, MD


Dr. Andrea Gross is a board-certified pediatrician and pediatric oncologist who earned her medical degree at the University of Connecticut and completed pediatric residency and a chief resident year at Cincinnati Children’s Hospital Medical Center. She completed a pediatric hematology/oncology fellowship at Children’s National Medical Center and is currently an Assistant Research Physician working in the Pediatric Oncology Branch at the National Cancer Institute in the lab of Dr. Brigitte Widemann. Dr. Gross has been the lead associate investigator on the phase 2 trial of selumetinib for patients with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas since 2015, which led to the first FDA approved medication for NF1 in 2020. Her research focuses on clinical trials for tumor predisposition syndromes.  Her areas of interest include developing and utilizing functional outcome measures for tumor predisposition syndromes, working with rare disease patient advocates to increase patient engagement in clinical trial design and dealing with the challenge of medication adherence in the NF1 population.

Planner and Faculty Disclosures

Any person who may contribute to the content of this continuing education activity must disclose relevant relationships (and any known relationships of their spouse/partner) with commercial interests whose products or services are discussed in educational presentations. A commercial interest is defined as any entity producing, marketing, re-selling, or distributing health care goods or services consumed by, or used on, patients. Relevant relationships include receiving from a commercial interest research grants, consultant fees, travel, other benefits, or having a self-managed equity interest in a company.

Disclosure of a relationship is not intended to suggest or condone any bias in any presentation but is made to provide participants with information that might be of potential importance to their evaluation of a presentation.


Bruce R. Korf, MD, PhD–has disclosed that he is a consultant for SpringWorks Therapeutics. He also receives grant/research support from AstraZeneca.

Bryan Taylor, PharmD–has no relevant financial relationships to disclose in relation to the content of this activity.


Jaishri Blakeley, MD–has disclosed that she is a consultant for Astellas, AstraZeneca, Children's Tumor Foundation, and SpringWorks Therapeutics. She also receives grant/research support from GlaxoSmithKline, Lilly, NCI, and Sanofi.

Andrea M. Gross, MD–has no relevant financial relationships to disclose in relation to the content of this activity.

Bruce R. Korf, MD, PhD–has disclosed that he is a consultant for SpringWorks Therapeutics. He also receives grant/research support from AstraZeneca.

Content Reviewer:

Miriam Bornhorst, MD–

In support of improving patient care, Creative Educational Concepts is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.


Medicine (ACCME)
CEC designates this live educational activity for a maximum of 1.5 AMA PRA Category 1 Credits™.  Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Upon completion of a post-test and evaluation, statements of credit for physicians will be issued within 30 business days.

Available Credit

  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Participation
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