Great strides are being made in neurofibromatosis research, and the Neurofibromatosis Program at Cincinnati Children’s is helping lead the way. Our leadership in national research consortia allows us to offer a number of novel therapies for patients with neurofibromatosis, malignant peripheral nerve sheath tumors, optic pathway gliomas and plexiform neurofibromas.

Cincinnati Children’s is part of the Children’s Oncology Group, Neurofibromatosis Clinical Research Consortium, Sarcoma Alliance for Research Through Collaboration and Pediatric Brain Tumor Consortium. Our physicians hold leadership positions in many of these consortia, helping to shape national clinical trials that will lead to the next generation’s standard of care. We also offer novel treatments discovered in the laboratory at Cincinnati Children’s.

Promising Laboratory Research

Nancy Ratner, PhD, leads the new Cincinnati Center for Neurofibromatosis Research.  Ratner is an internationally noted researcher in neurofibromatosis-related tumors. She and other scientists at Cincinnati Children’s conduct laboratory research that leads to the development of Phase 1 clinical trials for neurofibromatosis research.

Ratner and her team have already made important discoveries that may lead to better treatments. For example, she has discovered that the timing of the NF1 gene mutation in mice plays a key role in determining whether tumors will form. Researchers in the Neurofibromatosis Program are exploiting this new understanding of tumor formation to find novel methods for treating NF1-related tumors.

Ratner’s lab has also been able to suppress tumor formation in mice using drugs such as rapamycin and everolimus. This discovery and others motivated Brian D. Weiss, MD, and his colleagues to propose rapamycin as a treatment for plexiform neurofibromas. Currently,  Weiss leads the national NF Consortium trial of rapamycin for children and adults with NF1 and life-threatening plexiform neurofibromas, and many of the research and laboratory aspects of the trial are led by researchers at Cincinnati Children’s.