Novel agents for the treatment of mucositis

Each year, approximately 400,000 people in the United States develop cancer treatment-induced oral or esophageal mucositis. Ulceration and inflammation in the alimentary tract can be so severe that they compromise treatment and survival due to life-threatening complications. These complications may necessitate treatment interruption, dose reduction, or cessation of treatment altogether. Pain and difficulty eating or swallowing commonly occur and contribute to long-term morbidity. Until recently, supportive care alone was the mainstay of treatment for mucositis. Palliative interventions were based on the concept that injury occurred simply at the epithelial level. Although a wide variety of palliative treatments were offered to patients, none proved effective at prevention, and few agents significantly reduced the duration or severity of mucositis. Within the past decade, however, research on the pathogenesis of mucositis has revealed a complex sequence of biological events, each of which has the potential to serve as a therapeutic target. Initial results with selected agents are encouraging, and active research is under way to identify additional new drug targets and candidate drugs, which are reviewed here.