Frontiers in Pharmacology (Feb 2019)
Drug Repurposing: The Anthelmintics Niclosamide and Nitazoxanide Are Potent TMEM16A Antagonists That Fully Bronchodilate Airways
- Kent Miner,
- Katja Labitzke,
- Benxian Liu,
- Paul Wang,
- Kathryn Henckels,
- Kevin Gaida,
- Robin Elliott,
- Jian Jeffrey Chen,
- Longbin Liu,
- Anh Leith,
- Esther Trueblood,
- Esther Trueblood,
- Esther Trueblood,
- Kelly Hensley,
- Kelly Hensley,
- Kelly Hensley,
- Xing-Zhong Xia,
- Oliver Homann,
- Brian Bennett,
- Mike Fiorino,
- John Whoriskey,
- Gang Yu,
- Sabine Escobar,
- Min Wong,
- Teresa L. Born,
- Alison Budelsky,
- Mike Comeau,
- Dirk Smith,
- Jonathan Phillips,
- James A. Johnston,
- Joseph G. McGivern,
- Kerstin Weikl,
- David Powers,
- Karl Kunzelmann,
- Deanna Mohn,
- Andreas Hochheimer,
- John K. Sullivan
Affiliations
- Kent Miner
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Katja Labitzke
- Department of Therapeutic Discovery, Amgen Inc., Regensburg, Germany
- Benxian Liu
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Paul Wang
- Department of Therapeutic Discovery, Amgen Inc., Thousand Oaks, CA, United States
- Kathryn Henckels
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Kevin Gaida
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Robin Elliott
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Jian Jeffrey Chen
- Department of Medicinal Chemistry, Amgen Inc., Thousand Oaks, CA, United States
- Longbin Liu
- Department of Medicinal Chemistry, Amgen Inc., Thousand Oaks, CA, United States
- Anh Leith
- Department of Inflammation Research, Amgen Inc., Seattle, WA, United States
- Esther Trueblood
- Department of Comparative Biology and Safety Sciences, Amgen Inc., Seattle, WA, United States
- Esther Trueblood
- Department of Comparative Biology and Safety Sciences, Amgen Inc., Thousand Oaks, CA, United States
- Esther Trueblood
- Department of Comparative Biology and Safety Sciences, Amgen Inc., South San Francisco, CA, United States
- Kelly Hensley
- Department of Comparative Biology and Safety Sciences, Amgen Inc., Seattle, WA, United States
- Kelly Hensley
- Department of Comparative Biology and Safety Sciences, Amgen Inc., Thousand Oaks, CA, United States
- Kelly Hensley
- Department of Comparative Biology and Safety Sciences, Amgen Inc., South San Francisco, CA, United States
- Xing-Zhong Xia
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Oliver Homann
- Genome Analysis Unit, Amgen Inc., South San Francisco, CA, United States
- Brian Bennett
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Mike Fiorino
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- John Whoriskey
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Gang Yu
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Sabine Escobar
- Department of Inflammation Research, Amgen Inc., Seattle, WA, United States
- Min Wong
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Teresa L. Born
- Department of Inflammation Research, Amgen Inc., Seattle, WA, United States
- Alison Budelsky
- Department of Inflammation Research, Amgen Inc., Seattle, WA, United States
- Mike Comeau
- Department of Inflammation Research, Amgen Inc., Seattle, WA, United States
- Dirk Smith
- Department of Inflammation Research, Amgen Inc., Seattle, WA, United States
- Jonathan Phillips
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- James A. Johnston
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Joseph G. McGivern
- Department of Therapeutic Discovery, Amgen Inc., Thousand Oaks, CA, United States
- Kerstin Weikl
- Department of Therapeutic Discovery, Amgen Inc., Regensburg, Germany
- David Powers
- Department of Therapeutic Discovery, Amgen Inc., Thousand Oaks, CA, United States
- Karl Kunzelmann
- 0Institut für Physiologie, Universität Regensburg, Regensburg, Germany
- Deanna Mohn
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- Andreas Hochheimer
- Department of Therapeutic Discovery, Amgen Inc., Regensburg, Germany
- John K. Sullivan
- Department of Inflammation Research, Amgen Inc., Thousand Oaks, CA, United States
- DOI
- https://doi.org/10.3389/fphar.2019.00051
- Journal volume & issue
-
Vol. 10
Abstract
There is an unmet need in severe asthma where approximately 40% of patients exhibit poor β-agonist responsiveness, suffer daily symptoms and show frequent exacerbations. Antagonists of the Ca2+-activated Cl− channel, TMEM16A, offers a new mechanism to bronchodilate airways and block the multiple contractiles operating in severe disease. To identify TMEM16A antagonists we screened a library of ∼580,000 compounds. The anthelmintics niclosamide, nitazoxanide, and related compounds were identified as potent TMEM16A antagonists that blocked airway smooth muscle depolarization and contraction. To evaluate whether TMEM16A antagonists resist use- and inflammatory-desensitization pathways limiting β-agonist action, we tested their efficacy under harsh conditions using maximally contracted airways or airways pretreated with a cytokine cocktail. Stunningly, TMEM16A antagonists fully bronchodilated airways, while the β-agonist isoproterenol showed only partial effects. Thus, antagonists of TMEM16A and repositioning of niclosamide and nitazoxanide represent an important additional treatment for patients with severe asthma and COPD that is poorly controlled with existing therapies. It is of note that drug repurposing has also attracted wide interest in niclosamide and nitazoxanide as a new treatment for cancer and infectious disease. For the first time we identify TMEM16A as a molecular target for these drugs and thus provide fresh insights into their mechanism for the treatment of these disorders in addition to respiratory disease.
Keywords
- TMEM16A antagonist
- niclosamide
- nitazoxanide
- bronchodilator
- desensitization
- airway smooth muscle (ASM)