A TRPA1 inhibitor suppresses neurogenic inflammation and airway contraction for asthma treatment

J Exp Med. 2021 Apr 5;218(4):e20201637. doi: 10.1084/jem.20201637.

Abstract

Despite the development of effective therapies, a substantial proportion of asthmatics continue to have uncontrolled symptoms, airflow limitation, and exacerbations. Transient receptor potential cation channel member A1 (TRPA1) agonists are elevated in human asthmatic airways, and in rodents, TRPA1 is involved in the induction of airway inflammation and hyperreactivity. Here, the discovery and early clinical development of GDC-0334, a highly potent, selective, and orally bioavailable TRPA1 antagonist, is described. GDC-0334 inhibited TRPA1 function on airway smooth muscle and sensory neurons, decreasing edema, dermal blood flow (DBF), cough, and allergic airway inflammation in several preclinical species. In a healthy volunteer Phase 1 study, treatment with GDC-0334 reduced TRPA1 agonist-induced DBF, pain, and itch, demonstrating GDC-0334 target engagement in humans. These data provide therapeutic rationale for evaluating TRPA1 inhibition as a clinical therapy for asthma.

Publication types

  • Clinical Trial, Phase I
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Animals
  • Asthma / drug therapy*
  • Cohort Studies
  • Disease Models, Animal
  • Dogs
  • Double-Blind Method
  • Female
  • Guinea Pigs
  • Healthy Volunteers
  • Humans
  • Isothiocyanates / administration & dosage
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Middle Aged
  • Neurogenic Inflammation / drug therapy*
  • Pain / chemically induced
  • Pain / drug therapy*
  • Pruritus / chemically induced
  • Pruritus / drug therapy*
  • Pyridines / pharmacology*
  • Pyridines / therapeutic use*
  • Pyrimidines / pharmacology*
  • Pyrimidines / therapeutic use*
  • Rats
  • Rats, Sprague-Dawley
  • TRPA1 Cation Channel / antagonists & inhibitors*
  • TRPA1 Cation Channel / deficiency
  • Treatment Outcome
  • Young Adult

Substances

  • GDC-0334
  • Isothiocyanates
  • Pyridines
  • Pyrimidines
  • TRPA1 Cation Channel
  • TRPA1 protein, human
  • Trpa1 protein, mouse
  • 2,3,4-tri-O-acetylarabinopyranosyl isothiocyanate