This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Cardiovascular disease

Hypertension

Potassium channel K2p3.1 (KCNK3); phospholipase A2 (PLA2)

Studies in patient samples and cell culture suggest activating KCNK3 could help treat pulmonary arterial hypertension (PAH). Predicted loss-of-function mutations in KCNK3 were identified in 3 of 230 patients with idiopathic PAH and 3 of 93 patients with familial PAH. In cell culture, expression of mutant alleles of KCNK3 confirmed the disruption of protein activity. In these cells, a PLA2 inhibitor known to activate wild-type KCNK3 increased the activity of KCNK3 mutants compared with vehicle. Next steps include testing additional KCNK3-activating compounds in vitro and in animal models.

SciBX 6(33); doi:10.1038/scibx.2013.891
Published online Aug. 29, 2013

Patent and licensing status unavailable

Ma, L. et al. N. Engl. J. Med.; published online July 25, 2013;
doi:10.1056/NEJMoa1211097
Contact: Wendy K. Chung, Columbia University Medical Center,
New York, N.Y.
e-mail:

wkc15@columbia.edu