Escaping the Silo Mentality: Creating a Joint Vision for Gene Therapy in NF2

September 2015

Neurofibromatosis 2 (NF2) is a tumor suppressor syndrome caused by genetic alterations in the NF2 gene. People with NF2 develop multiple nervous system tumors that can result in deafness, facial paralysis, weakness, trouble walking, seizures, and pain. Standard treatment includes surgery but complete resection of multiple tumors is usually not possible. Currently, there are no approved medical treatments for NF2-related tumors, and there are no drugs that treat the underling genetic abnormality. Gene therapy is an attractive option for treating NF2 since replacement of the missing NF2 gene should, in theory, restore normal cellular function. There are multiple roadblocks to developing gene therapy for NF2, including a lack of scientific interaction between the NF2 and gene therapy research communities. Despite significant improvements in adenovirus-associated virus vectors for genetic disease, there is no sustained research into using these vectors to treat NF2. Thus, the timing is right for a critical reevaluation of the potential use of gene therapy for tumor suppressor syndromes. This seminar brings together leading researchers from the NF2 and gene therapy communities at Harvard and at collaborating institutions to discuss the feasibility of gene therapy for NF2 patients.