Gene Therapy for Patients with Head and Neck Cancer

This chapter reports on the biological activity and biosafety of gene therapy for patients with head and neck cancer. The results of the studies show that clinical response rates have been between 5% and 20%, which is comparable to historical rates using single-agent chemotherapy in recurrent and refractory head and neck squamous cell carcinoma (HNSCC) patients. One of the advantages of this therapy is cytostatic or stabilization of disease in a larger proportion of patients. Gene therapy may produce other clinical effects, such as stabilization of disease and improved quality of life. In most trials, the maximum tolerated dose was not achieved as no significant toxicity was observed at the highest dose tested. The reported toxicity has been limited to pain, bleeding, and fever/chills without any treatment-related deaths. These initial clinical results that were translated from laboratory work using gene therapy can act as the basis for further laboratory research and then clinical trials. A combination of cytostatic gene therapy and cytotoxic standard therapy is also suggested.