Neurosurgical Challenges in Neurofibromatosis Type 1: New Horizons in the Era of MEK Inhibition
This educational initiative is targeted to pediatric neurosurgeons, neurosurgeons, neuro-oncologists, neuroradiologists, medical oncologists, pediatric hem/onc nurses, and other members of the interprofessional, multidisciplinary 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 describing 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, with an emphasis on the challenges faced by neurosurgeons 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 neurosurgeon within the evolving NF1 management paradigm, and provides activity participants an opportunity to design evidence-based treatment regimens that explore how safe and effective medical therapies can augment and optimize surgical strategies for symptomatic, inoperable NF1-related tumors.
Presented by Creative Educational Concepts, Inc (CEC Oncology) and supported by an independent educational grant from AstraZeneca.
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 attending the 2020 Annual Meeting of the AANS/CNS Section on Pediatric Neurological Surgery.
- Describe the genetic genesis and the complex, multi-system pathophysiology of NF1, with an emphasis on the clinical gravity and historical intractability of plexiform neurofibromas, especially in the pediatric patient population.
- Review traditional modalities for treating plexiform neurofibromas in patients with NF1, focusing on surgical strategies, 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 neurosurgeon within the interprofessional neuro-oncology team, and identify key ways that emerging medical therapies can augment and optimize surgical approaches in NF1.
- Using a case-based format, design evidence-based management strategies for inoperable, clinically-significant NF1-associated plexiform neurofibromas.
12:00 PM-12:05 PM EST:
Welcome and Introductions/Pre-test
Allan Belzberg, MD (Activity Chair)
12:05 PM-12:15 PM EST:
Capturing Complexity: The Etiology, Pathophysiology, and Diverse Clinical Hallmarks of NF1 in Pediatric Patients
Miriam Bornhorst, MD
12:15 PM–12:35 PM EST:
Employing Novelty in Plexiform Neurofibroma Management: Emerging Data and Non-Surgical Treatment Alternatives
Andrea M. Gross, MD
12:35 PM-12:55 PM EST:
Bridging Clinical Chasms in NF1: The Pivotal Placement of the Neurosurgeon in the New Paradigm
Allan Belzberg, MD
12:55 PM-1:00 PM EST:
Conversation with the Experts: Audience Q&A/Post-test
Faculty Panel Moderated by Dr. Belzberg
Allan Belzberg, MD
Director, Peripheral Nerve Surgery
Co-Director, Neurosurgery Pain Research Institute
Professor of Neurosurgery
Professor of Plastic and Reconstructive Surgery
George J. Heuer Professor of Neurosurgery
Co-Director, Kennedy Krieger Pediatric Nerve Injury Program
The Johns Hopkins University School of Medicine
Dr. Belzberg is professor of Neurosurgery and Plastic & Reconstructive Surgery at the Johns Hopkins School of Medicine. He holds the George J Heuer Chair in Neurosurgery and has a clinical interest in spine and peripheral nerve surgery. Dr. Belzberg is an expert on the surgical management of peripheral nerve disorders. As a member of the multi-disciplinary comprehensive Neurofibromatosis Clinic at Johns Hopkins he treats patients suffering from a variety of benign and malignant tumors. His expertise in both spinal and peripheral nerve surgery allow him to remove the most challenging nerve tumors and reconstruct both the spine and peripheral nerves as required. Patients come from around the globe to benefit from these skills and he has built up a sizable tumor bank with specimens and detailed clinical notes.
Dr. Belzberg is interested in neuropathic pain and is the co-director of the Neurosurgery Pain Research Institute at Johns Hopkins. He has a publication record concerning the pathophysiology of painful nerves including those related to NF and nerve injury. His laboratory described a novel animal model of neuropathic pain that allows the investigator to study both stimulus evoked mechanical hyperalgesia and pain phenotype change of axons exposed to degenerating axons (neuroma). Dr. Belzberg is involved in basic science research concerning gene regulation in Schwannomatosis.
Dr. Belzberg has previously served as the President of the American Society for Peripheral Nerve. He has served as a scientific advisor to a broad spectrum of health care entities including Neurofibromatosis Inc., Spinal Kinetics and Medtronic. He has lectured in more than 30 countries and his contributions to clinical medicine have changed the practice.
Miriam Bornhorst, MD
Gilbert Neurofibromatosis Institute
Children’s National Hospital
Miriam Bornhorst is the Clinical Director of the Gilbert Neurofibromatosis Institute and Medical Director of the Cancer Genetics Clinic at Children’s National Hospital (CNH) in Washington, DC. She also has a joint appointment as an Assistant Professor in Pediatrics at George Washington University Hospital. After receiving her medical degree from Creighton University, Dr. Bornhorst completed pediatric residency and pediatric hematology/oncology fellowship at the University of Michigan followed by neuro-oncology fellowship training at CNH. Her main clinical interests are cancer predisposition, neurofibromatosis type 1, and neuro-oncology. Her research focuses on the early detection and treatment of patients at risk for developing cancer, including biomarker discovery, and metabolism in NF1.
Andrea M. Gross, MD
Assistant Research Physician
Pediatric Oncology Branch
Center for Cancer Research
National Institutes of Health
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.
Bryan Taylor, PharmD–has no relevant financial relationships to disclose in relation to the content of this activity.
Allan Belzberg, MD–has no relevant financial relationships to disclose in relation to the content of this activity.
Miriam Bornhorst, MD–has no relevant financial relationships to disclose in relation to the content of this activity.
Andrea M. Gross, MD–has no relevant financial relationships to disclose in relation to the content of this activity.
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.
CEC designates this live educational activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
This activity is designated for 1.0 contact hours.
Upon completion of a post-test and evaluation, statements of credit for physicians and nurses will be issued within 30 business days.
- 1.00 AMA PRA Category 1 Credit™
- 1.00 ANCC
- 1.00 Participation