2017 Neurofibromatosis Highlight - Decreasing the Clinical Impact of Neurofibromatosis – New Clinical Trials Funded Through the Neurofibromatosis Research Program , Neurofibromatosis Research Program, Congressionally Directed Medical Research Programs

Decreasing the Clinical Impact of Neurofibromatosis – New Clinical Trials Funded Through the Neurofibromatosis Research Program

Posted October 24, 2017

Bruce Korf, M.D., Ph.D., University of Alabama at Birmingham
Ana-Maria Vranceanu, Ph.D., Massachusetts General Hospital
Konstantina Stankovic, M.D., Ph.D., Massachusetts Eye and Ear Infirmary

Ana-Maria Vranceanu, Ph.D., Massachusetts General Hospital

Konstantina Stankovic, M.D., Ph.D., Massachusetts Eye and Ear Infirmary

Neurofibromatosis (NF) refers to a group of three genetically distinct disorders that have a spectrum of clinical manifestations. The disorders, NF1, NF2, and Schwannomatosis, can all lead to tumor growth. Other manifestations include learning deficits and cognitive disorders, skin conditions, visual impairments, nervous system disorders and neurological symptoms, vascular disease, musculoskeletal disorders, malignancies, hearing impairments, and chronic pain. Treatments for NF currently include surgery, radiation, and chemotherapy, but several clinical trials are underway to identify more treatment options.

The Congressionally Directed Medical Research Programs’ Neurofibromatosis Research Program (NFRP) is funding several efforts focused on finding better treatments. The NF Clinical Trials Consortium (NFCTC) is being funded for a third grant cycle with a fiscal year 2016 (FY16) award. The Principal Investigator and director of the Operations Center is Dr. Bruce Korf; Dr. Michael Fisher has succeeded Dr. Roger Packer as chair of the Steering Committee. In previous grant cycles (FY06 and FY11 awards), the NFCTC launched seven clinical trials and collaborated with the Sarcoma Alliance for Research through Collaboration on two other trials. One additional trial is set to be launched for the FY11 NFCTC award. The NFCTC has performed more clinical trials than required by the Department of Defense (DOD) by partnering with several companies from the pharmaceutical industry, including Genentech, Exelixis, Pfizer, Medtronic, and Novartis, as well as receiving additional funding from other organizations and private philanthropy, such as the Children’s Tumor Foundation. With the FY16 award, the NFCTC anticipates opening four more trials throughout the cycle of the award, including at least two trials in the first year, and intends to continue leveraging DOD funding to support additional trials.

The NFRP also is funding two separate clinical trials with FY16 awards. Dr. Ana-Maria Vranceanu at Massachusetts General Hospital is performing a trial comparing two stress and symptom management group programs delivered via secure videoconferencing to adults with NF1, NF2, and Schwannomatosis. The goal of both programs is sustained improvement in quality of life. The stress management program determined to be superior through this trial will be disseminated to patients with help from the DOD and the Children’s Tumor Foundation. Dr. Konstantina Stankovic at the Massachusetts Eye and Ear Infirmary and Harvard Medical School is being funded to run a Phase II clinical trial of aspirin for treatment of vestibular schwannomas. Dr. Stankovic’s trial will test whether aspirin can prevent growth of vestibular schwannomas, as well as determine whether biomarkers from patients’ blood would be able to predict a response to aspirin treatment. Findings from these studies will benefit patients and meet the vision and mission of the NFRP – to decrease the clinical impact of NF and enhance the quality of life for persons with the disorders.