Otolaryngologists See Immunotherapy as Hope for Patients with Head and Neck Cancer

As for NF2 gene therapy, a breakthrough may come via large-animal models, such as the NF1 minipig model, Dr. Welling noted. The animal model “has shown a phenotype remarkably similar to humans with NF1,” including café au lait macules, neurofibromas, and optic pathway gliomas (Commun Biol. 2018;1:158 [doi:10.1038/s42003-018-0163-y]), he said. Such research “may be the key to achieving the advances necessary for gene therapy to get at the root of NF2.”