The Department of Otolaryngology interim chair, Bradford Woodworth, M.D., has received a two-year $275k R21 grant from the National Center for Complementary and Integrative Health to investigate a new therapeutic approach for cystic fibrosis (CF). The new approach developed by Dr. Woodworth and Dr. Cho targets the Ca2+-activated Cl− channel (TMEM16A) using ginsenosides found in Korean red ginseng, a popular natural health treatment in Asia.

Cystic fibrosis is a recessive, genetic disease that affects multiple organs at a time. The disease disrupts the balance of water and electrolyte transport throughout the body and can lead to declining lung function and premature death. Treatment of CF often involves extended hospitalization and can greatly affect patient quality of life.

Although highly effective CFTR modulator treatment often improves CF lung and sinus disease, it does not always work for every patient, and lung function is not often restored to normal levels. Therefore, the new approach developed by Dr. Woodworth and Dr. Cho targets the Ca2 -activated Cl− channel (TMEM16A) using ginsenosides found in Korean red ginseng, a popular natural health treatment in Asia.

Dr. Woodworth and Dr. Do-Yeon Cho previously identified the drug as a TMEM16a channel potentiator. The team aims to test ginsenosides for their ability to potentiate TMEM16A and evaluate their efficacy in improving mucociliary transport and mucus pathology. Woodworth and his team are hopeful that identifying a novel TMEM16A can eventually lead to identifying an independent, universal therapy for patients with CF and improvements in respiratory physiology and clinical outcome of these individuals.

This study could also provide insights into new treatment opportunities for other disorders, such as asthma, chronic sinusitis, and recurrent pancreatitis, where acquired CFTR dysfunction may play a role.