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Neurofibromatosis Institute Associated Departments Conditions & Treatments Meet Our Team Contact Information     Email This Page Print This Page           Neurofibromatosis Institute Jennifer and Daniel Gilbert Neurofibromatosis Institute The Neurofibromatosis Institute at Children’s National Medical Center, one of the largest in the world, is leading the medical field in the diagnosis, evaluation, and treatment of children and adults with the full range of conditions that relate to this condition. Neurofibromatosis is a genetic disorder in which tumors grow on the nerves and produce skin and bone abnormalities. There are two types of neurofibromatosis: Type 1 - occurs in one of every 4,000 births and affects an estimated 100,000 people in the US Type 2 – occurs in one in every 50,000 births The effects of either type of the disease can vary greatly. Some people are not affected at all while others suffer severe disabilities. Children’s treats more than 1000 patients each year through this program with any of the following conditions: Neurofibromatosis, Type 1 and 2 Plexiform neurofibromas Optic Nerve Gliomas Visual Pathway Gliomas Gliomas in Neurofibromatosis Type 1 Acoustic neuromas Meningiomas Intraspinal ependymomas Children’s Neurofibromatosis Institute has been designated a Neurofibromatosis Center of Excellence by the National Neurofibromatosis Foundation. Children’s program is directed by Roger Packer, MD, executive director of Neuroscience and Behavior Medicine at Children’s, and a leading expert in the care of children with brain tumors. The multidisciplinary program joins experienced staff from across a number of Children’s Divisions, including: Genetics Neurology Neuropsychology Neuroradiology Neurosurgery Hearing and Speech Ophthalmology Otolaryngology The program offers a multi-specialty clinic that offers one central location for patients and families to meet with specialists in genetics, neurology, and ophthalmology. From that clinic, patients and families are referred as needed to specialists in oncology, neurosurgery, neurology, neuropsychology etc The program also offers unparalleled access to innovative biologic therapies for patients with Neurofibromatosis Type 1. In addition, the program staff has extensive experience in assessing neurocognitive and learning difficulties in children with neurofibromatosis. It coordinates clinical trials evaluating these learning disabilities in an attempt to treat them with innovative biologic-based agents. The Children’s Neurofibromatosis Institute provides extensive support for families and children, including the coordination of the largest neurofibromatosis camp for children ages 7-16 on the East Coast. Research and Clinical Trials Children’s Neurofibromatosis Program has been chosen as one of eight US programs to participate in the Neurofibromatosis Clinical Trials Consortium, which is also chaired by Roger Packer, MD. The Consortium is funded by the US Department of Defense, which is supporting a trial to evaluate a new biologic agent, pirfenidone, in children with progressive plexiform neurofibromas. Children’s Neurofibromatosis Institute is also coordinating and/or participating in several additional pediatric clinical trials that: Evaluate and treat children with visual pathway and other gliomas and progressive plexiform neurofibromas Identify and validate new molecular targets for children with progressive gliomas and plexiform neurofibromas Develop molecular-based therapy Evaluate the neurocognitive challenges of neurofibromatosis In addition, the program conducts research on biologically-based therapy of neurofibromatosis through the National Cancer Institute, the Pediatric Brain Tumor Consortium, the Children’s Oncology Group and the newly-formed CTF mouse model consortium. The program also participates in developing new therapeutic treatments for the neurocognitive aspects of neurofibromatosis through the efforts of Maria Acosta, MD, and assesses the agent, lovastatin, for children with neurofibromatosis and learning disabilities. Other efforts in evaluating the learning and other cognitive disabilities in children with neurofibromatosis include two longitudinal studies, in collaboration with Katherine North, MD, from the University of Sydney, that are investigating the type of neurocognitive deficits these children have and how these deficits change over time. Back to Top