Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Pulmonary disease

Cystic fibrosis (CF)

Diacylglycerol kinase-i (DGKI); epithelial sodium channel (ENaC)

In vitro and cell culture studies suggest DGKI inhibition could help treat CF. An RNAi screen on airway epithelial cells identified DGKI and ciliary neurotrophic factor receptor as activators of ENaC, which is upregulated in CF. In human airway cells and ex vivo mouse tracheas, pharmacological inhibition of DGKI decreased ENaC levels and activity compared with no inhibition. In human airway epithelial cells from patients with CF, inhibition of DGKI decreased both ENaC activity to levels seen in healthy cells and fluid absorption compared with no inhibition. Next steps could include testing DGKI inhibition in animal models of CF.
Parion Sciences Inc. has the ENaC inhibitor P-552 in
Phase II testing to treat CF and xerostomia.

SciBX 6(39); doi:10.1038/scibx.2013.1103
Published online Oct. 10, 2013

Patent application filed; available for exclusive licensing

Almaça, J. et al. Cell; published online Sept. 12, 2013;
doi:10.1016/j.cell.2013.08.045
Contact: Margarida D. Amaral, University of Lisboa, Lisboa, Portugal
e-mail:

mdamaral@fc.ul.pt