The U.S. Food and Drug Administration has approved a national clinical trial to study the safety and effectiveness of a drug treatment on patients receiving radiation and chemotherapy for head and neck cancer.
The trial will be led by John Lee, MD, principal investigator and an ear, nose, and throat specialist at the Sanford Cancer Center in Sioux Falls, SD.
Dr. Lee, who was honored in 2010 by the American Cancer Society for his research, has been studying the link between the human papillomavirus (HPV) and the development of head and neck cancers. Dr. Lee's early research led to the discovery that mice treated with the generic drug dichloroacetate (DCA) responded to cancer therapy 30% better. His research team has also tested the treatment on head and neck tumors in mice and found that factors that enhanced the immune system seemed to improve survival rates.
DCA, which has been used in human patients with metabolic disorders, inhibits an enzyme called pyruvate dehydrogenase kinase (PDK) that is involved in the metabolic pathways at the cellular level. PDK is overexpressed in many types of cancer, including the head and neck cancer that is part of this new clinical trial. The trial will evaluate how altering cell metabolism will affect the outcome for participants.
The trial will be a blinded, placebo-controlled research study. All participants will receive the standard treatment for head and neck cancer and will, in addition, receive either the placebo or the study drug. Participants will be monitored closely during treatment by their cancer doctors and Sanford research staff.
A total of 50 patients will be enrolled in the clinical trial. After an initial screening, some patients will receive DCA as an oral medication or through a gastrointestinal tube twice a day for eight weeks. Others will receive placebos during the same period. During that time, participants will be given treatments of chemotherapy drug cisplatin and undergo radiation therapy. The participants' progress will be followed for five years.
The new trial will study the safety of delivering DCA during treatment and also assess the local response rate, overall survival, and relative toxicities for study participants. The researchers also hope to evaluate participants' HPV status and immune response levels, and correlate those findings as part of the trial. Participants will also be evaluated for differences in health-related quality of life.
