Table 1 Major clinical manifestations of Alström syndrome and corresponding pre-clinical effects of PBI-4050
Clinical features of ALMS | Corresponding pre-clinical effects of PBI-4050 |
|---|---|
Loss of organ function due to fibrosis involving | |
Heart | ↓ heart fibrosis in suprarenal aortic banding in rats |
Lung | ↓ lung fibrosis in bleomycin-induced lung fibrosis in mice |
Liver | ↓ liver fibrosis in CCl4-induced liver fibrosis in rats |
Kidneys (renal failure) | ↓ kidney fibrosis in various animal models of kidney fibrosis |
Type 2 diabetes mellitus | |
Early hyperinsulinemia | Reduces insulin resistance in db/db diabetic mice and db/db eNOS−/− diabetic mice |
Severe insulin resistance | Normalizes glycaemia in diabetic mice |
Late pancreatic failure | Maintains (early treatment) or restores (late treatment) insulin content in pancreatic islets |