On Demand - A Targeted Approach to Taming NF1-Associated Tumors: Navigating the Child Neurologist’s Role in an Evolving Treatment Calculus

This educational activity is targeted to pediatric neurologists, neuro-oncologists, neurosurgeons, and other members of the multidisciplinary NF1 care team. Designed and developed to provide an interactive overview of novel and emerging data, as well as establish a foundational context of NF1 disease state complexity and intractability, this activity will begin by reviewing revised diagnostic criteria and hallmark aspects of NF1 natural history and clinical presentation in pediatric patients, and describing the genetic etiologies and multi-system pathophysiology that have historically made NF1-associated tumors so difficult to treat. Supported by a dynamic case-based approach intended to encourage audience engagement, attendees will gain insights from key opinion leaders regarding the pivotal shortcomings of traditional NF1 management modalities and the new horizons that have accompanied the emergence of targeted medical therapies, most notably MEK inhibitors. This discussion will be supported by a detailed appraisal of clinical trial data, treatment recommendations, and approved indications for targeted therapies in NF1. Finally, top-level child neurology experts will guide attendees through a case-driven, real-world exploration of the expanding role of the pediatric neurologist as the NF1 paradigm advances into the era of MEK inhibition.

Supported through an independent educational grant from AstraZeneca.

Target Audience

This educational activity is targeted to pediatric neurologists, neuro-oncologists, neurosurgeons, and other members of the multidisciplinary NF1 care team.

Learning Objectives

  1. Review recently updated NF1 diagnostic criteria and recognize established NF1 clinical manifestations to promote earlier detection and treatment initiation, especially in pediatric patients.
  2. Describe the genetic genesis and multifaceted pathophysiology of NF1, with a focus on the neurological morbidity and historical intractability of symptomatic, inoperable plexiform neurofibromas.
  3. Using a patient case-driven approach: evaluate traditional NF1-associated plexiform neurofibroma treatment modalities, including surgery, radiation, and surveillance, and emphasize how targeted medical therapies are poised to bridge crucial chasms in care.
  4. Appraise completed and ongoing clinical trial data for MEK inhibitors and other novel targeted therapies being studied in NF1-associated tumors, with an emphasis on current treatment recommendations and approved indications.
  5. Discuss the pivotal placement of the pediatric neurologist on the multidisciplinary NF1 treatment team, and design real-world, evidence-based treatment plans using targeted therapies for NF1-associated tumors:
    • Plexiform neurofibromas (PNs)
    • Low-grade gliomas (LGGs)
    • Optic pathway gliomas (OPGs)
    • Malignant peripheral nerve sheath tumors (MPNSTs)

Additional Information

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Course summary
Available credit: 
  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Participation
Course opens: 
Course expires: 


5 min       Welcome and Introductions
20 min     NF1 Etiology, Multisystem Pathophysiology, & Diverse Clinical Manifestations
25 min     Revolutionizing Plexiform Neurofibroma Outcomes with Targeted Medical Therapies
30 min     Practical MEK Inhibitor Strategies for Pediatric Neurologists in the Changing Paradigm
10 min     Conversations with the Experts


Verena Staedtke, MD, PhD (Activity Chair)
Director of Pediatric Neurofibromatosis
The Johns Hopkins Comprehensive Neurofibromatosis Center
Associate Professor of Neurology
Johns Hopkins University
Baltimore, MD

Dr. Staedtke received her degrees from the Charite, Medical School of the Humboldt and Free University in Berlin (Germany).  She completed her pediatric neurology residency and UCNS neuro-oncology fellowship at Johns Hopkins and became a member of the Neurology Faculty in 2015.  Now an Associate Professor, Dr. Staedtke serves as the Director of Pediatric Neurofibromatosis (NF) at the Johns Hopkins Comprehensive Neurofibromatosis Center, where she leads the pediatric NF program and cares for children and adolescents with NF.  In addition to her clinical responsibilities, Dr. Staedtke leads a research laboratory focusing on the discovery of novel antineoplastic therapeutics for nervous system tumors and gene therapy development for NF. Dr. Staedtke received the Francis Collins Scholar Research Award, the 2018 Sontag Foundation Distinguished Scientist Award,  NCI K08 and U01 award in addition to other federal and private funding support.  

Michael J. Fisher, MD
The Children's Hospital of Philadelphia
University of Pennsylvania Perelman School of Medicine
Philadelphia, PA

Dr. Michael Fisher is Professor of Pediatrics (Perelman School of Medicine at the University of Pennsylvania), Chief of the Neuro-Oncology Section (Division of Oncology), Director of the Neurofibromatosis (NF) Program, and the Hubert J.P. and Anne Faulkner Schoemaker Endowed Chair in Pediatric Neuro-Oncology at The Children's Hospital of Philadelphia.  His research focuses on identifying new treatments and novel biomarkers (particularly using new imaging modalities) and exploring functional outcomes for children with tumors associated with NF1.  Dr. Fisher is Chair of the Steering Committee, and previously was Chair of the Plexiform Neurofibroma Committee, of the Department of Defense NF Clinical Trials Consortium. He is a member of the Steering Committee and former Chair of the Visual Outcomes Committee for REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis), an international effort to develop standardized outcome measures for clinical trials.

Nicole Ullrich, MD, PhD
Associate Professor of Neurology
Harvard Medical School
Boston, MA


Dr. Ullrich completed her medical and graduate degrees at Yale University followed by residency in Pediatrics and Neurology at Boston Children’s Hospital, where she then did her fellowship in NeuroOncology before joining the faculty in the Department of Neurology.  Her research focuses on neurologic and oncologic complications of NF1 and the long-term neurologic complications of childhood systemic cancer and brain tumors.  Dr. Ullrich has been involved in the design and execution of several clinical trials for complications of NF1 through the Neurofibromatosis Clinical Trials Consortium, where she is currently the site principal investigator for the Harvard site that includes Boston Children’s Hospital, Dana-Farber Cancer Institute and Massachusetts General Hospital.  Within the consortium, she is former chair of the Low Grade Glioma committee and currently co-chair of the Neurocognitive Committee.  She serves as co-Chair of the Clinical Research Award committee and is a member of the Clinical Care Advisory Board for the Children’s Tumor Foundation.  Lastly, she serves on the board of NF Northeast. Dr. Ullrich recently participated in the international working groups to revise and update the diagnostic criteria for the neurofibromatoses and in the updated AAP health supervision guidelines for children with NF1.

In accordance with the Food and Drug Administration, the speakers have disclosed that there is the potential for discussions concerning off-label uses of a commercial product/device during this educational activity.

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 an entity producing, marketing, re-selling, or distributing health care goods or services consumed by, or used on patients. Relevant relationships include receiving from acommercial 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.

Verena Staedtke, MD, PhD–has no relevant financial relationships to disclose in relation to the content of this activity.
Bryan C. Taylor, PharmD—has no relevant financial relationships to disclose in relation to the content of this activity.

Verena Staedtke, MD, PhD–has no relevant financial relationships to disclose in relation to the content of this activity.
Michael J. Fisher, MD– has disclosed that he is a consultant for AstraZeneca and SpringWorks. He receives Grant/Research support from AstraZeneca and Array BioPharma Inc.
Nicole Ullrich, MD, PhD– has disclosed that she is a consultant for Peer Review Institute for Medical Education. 

Miriam Bornhorst, MD—has disclosed she is a consultant for AstraZeneca. 

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, evaluation, and credit request, statements of credit for physicians will be issued within 30 days.

Available Credit

  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Participation
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