BioCentury
ARTICLE | Distillery Therapeutics

Therapeutics: Lipocalin (NGAL; LCN2)

February 18, 2016 8:00 AM UTC

Patient sample and mouse studies suggest 4-phenylbutyric acid could help treat proteinuria associated with chronic kidney disease. Renal tubule levels of NGAL were higher in patients with proteinuric nephropathy and mouse models of the indication than in healthy subjects and normal mice. In urinary samples from a patient with proteinuria, 4-phenylbutyric acid decreased levels of NGAL in the urine compared with healthy controls. In a mouse model of proteinuria, knockout of NGAL decreased renal lesions and increased survival compared with normal NGAL expression. Also in the model, 4-phenylbutyric acid decreased renal levels of NGAL, endoplasmic reticulum stress, renal lesions, and markers of renal dysfunction including interstitial fibrosis and plasma creatine levels in the kidney compared with vehicle. Next steps could include testing the compound in additional models of kidney disease...