Comprehensive Neurofibromatosis Center

Director: Jeffrey Allen, MD
Co-Directors: J. Thomas Roland, MD, John Golfinos, MD and Matthias Karajannis, MD, MS
Nurse Coordinator: Carole Mitchell, RN, MS
Contact information: 212 263-9695; carole.mitchell@nyumc.org

The NYU Langone Comprehensive Neurofibromatosis (NF) Center is located at the Stephen D. Hassenfeld Children's Center for Cancer and Blood Disorders and was founded in April, 2008 with support from the Children’s Tumor Foundation (CTF) and NYU Langone Medical Center.  Our primary goal is to offer individualized, comprehensive medical and surgical evaluation and care for patients and families with NF1, NF2 and Schwannomatosis.  A dedicated multi-disciplinary team of medical, behavioral and surgical specialists works together to identify and remediate the diverse and often age-related acute and chronic manifestations of these genetic disorders. Our secondary goal is to discover better and safer treatments to patients suffering from the progressive manifestations of NF, especially the tumors of the nervous system, through research and clinical trials. We are the first program in the New York metropolitan area to receive the “Affiliate Clinic” designation from CTF.  The NYU Langone Comprehensive NF Center is part of a national and international network of clinics working to establish a patient registry and clinical trials consortium. We are also proud to announce that as of June, 2011, our NF Center has been invited to become a member of the national NF clinical trials organization, the “NF Consortium” and the only participating Center in New York State.  In the past, the closest member institutions were located in Philadelphia and Washington, DC which was inconvenient for many of the New York area NF patients. This Consortium is funded by a grant from the Department of Defense and consists of 14 academic institutions located throughout the United States. The primary focus of this Consortium is to conduct clinical trials designed to establish additional therapies to treat relatively unexplored or medically refractory complications of NF1 and NF2. Such complications have included growing plexiform neurofibromas, primary brain tumors, and complex behavioral disorders in NF1 patients. We hope to initiate NF2 related trials under their auspices as well. 

Administration of the NF Center

Our team of medical specialists is knowledgeable and experienced in the care of patients with both NF1 and NF2 and committed to offer care to patients of all ages. The NF Center is directed by Jeffrey Allen, MD, a pediatric neurologist/neuro-oncologist who cares for both children and adults with NF1 and NF2. The Co-director of the NF1 Program is Allan Rubenstein, MD, a neurologist with a long-standing interest in NF1. The Co-Directors of the NF2 Programs are J. Thomas Roland, Jr., MD, Chairman of the Department of Otolaryngology, John Golfinos, MD, Chairman of the Department of Neurosurgery and Matthias Karajannis, MD, MS, a pediatric oncologist who leads our clinical trials program for NF patients. A dedicated Nurse Coordinator, Carole Mitchell, RN, MS, facilitates the evaluation of new patients and coordinates the diagnostic and treatment plans.

Location of the NF Center

The NF Center is conveniently located within the Stephen D. Hassenfeld Children's Center for Cancer and Blood Disorders at 160 East 32nd Street in Midtown Manhattan. The Hassenfeld Center is designed to provide comprehensive outpatient care for patients and families with a variety of chronic illnesses, including cancer, blood disorders, autoimmune diseases and certain genetic disorders, such as NF1 and NF2. The supporting staff includes specialists in child lifenutritionneuropsychology, social service, library services and clinical trials management.

Evaluation and Treatment

After making initial contact with the Nurse Coordinator, Carole Mitchell, a phone questionnaire is conducted to assist the staff in organizing the initial patient evaluation and requesting records of prior medical tests and treatments. Depending on age, diagnosis and/or patient preference, the patient will be evaluated by Dr. Allen, Dr. Rubenstein or Dr. Karajannis with the assistance of our Nurse Coordinator, Carole Mitchell. All patients will be interviewed by a social worker to identify special needs and available resources. A comprehensive evaluation and treatment plan will be formulated, which may include additional tests, scans and/or referrals to additional specialists, as indicated.

Referrals are frequently made to our clinical geneticist, John Pappas, MD and our dermatologist, Julie Schaffer, MD. Further evaluations, when indicated, will occur by our team of behavioral and learning specialists, led by David Salsberg, PhD. John Pappas, MD, MS, Director of the NYU Langone Clinical Genetics Services oversees genetic evaluation and testing, assisted by Ellen Moran, MS, the genetic counseling coordinator for our NF Center. Genetic testing is available for neurofibromatosis type 1, Legius syndrome, neurofibromatosis type 2, schwannomatosis as well as other syndromes presenting with skin lesions resembling NF.

Depending on the patient's needs for surgical evaluation and treatment, we make referrals to our surgical specialists in the fields of otolaryngology (ENT), neurosurgery, plastic surgery or orthopedics. All NF2 patients will be referred to Dr. Roland and Dr. Golfinos for discussion about options for surgical treatment and hearing preservation/rehabilitation, including hearing aids and implantable hearing devices such as cochlear (CI) or brainstem auditory implants (ABI). Services for voice, swallowing, balance disorders, tinnitus, and other issues related to cranial nerve dysfunction are also readily available with our physicians and other providers.

Multi-disciplinary meetings serve to address complex clinical issues. Referrals can be made to our Peripheral Nerve Tumor Team, consisting of plastic, orthopedic and neurosurgical specialists, for removal of low-grade and malignant tumors of the peripheral nervous system, such as dermal neurofibromas, plexiform neurofibromas, schwannomas and malignant peripheral nerve sheath tumors. Cancer care will be provided by our team of pediatric and medical oncologists, radiation oncologists and surgeons affiliated with the NYU Langone Cancer Institute.

Patient and family support groups and individual counseling are provided by our nursing, behavioral and social work teams.

Clinical and Translational Research Activities at the NF Center

We are conducting our ownclinical trials with “molecular targeted” drugs in NF2 patients with progressive vestibular schwannomas, including those at risk for hearing loss. We are conducting both phase 0 and 2 trials with an anti-tumor drug, lapatinib, which inhibits two growth-promoting molecules expressed by vestibular schwannomas. We have completed enrollment on our initial phase 2 lapatinib trial involving a total of 21 NF2 patients, and preliminary data indicates that more than 25% of evaluable patients treated have experienced significant tumor shrinkage, with many additional patients remaining on treatment without further tumor growth since starting on study. The phase 0 trial is conducted by the NF2 Experimental Therapeutics Consortium, which is led by NYU Langone and Johns Hopkins University. Patients scheduled for removal of either a single sporadic or NF2-related vestibular schwannoma take labatinib for 10 consecutive days prior to surgery. After removal of their tumor, studies are performed to measure both concentration and activity of the study medication within the tumor. Using this approach, we can screen new medications for potential therapeutic activity. For all NF patients undergoing tumor surgery for any reason, tumor banking is offered to provide specimens for laboratory research.