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results In three mouse models of PKD, treatment with a GCS inhibitor resulted in decreased kidney and plasma levels of glucosylceramide (GL-1) and reduced formation of kidney cysts compared with… Click to show full abstract
results In three mouse models of PKD, treatment with a GCS inhibitor resulted in decreased kidney and plasma levels of glucosylceramide (GL-1) and reduced formation of kidney cysts compared with untreated controls. Analysis of kidney tissue from treated mice showed that inhibiting GCS blocked signaling between protein kinase B (PKB; Akt) and mammalian target of rapamycin (mTOR; FRAP; RAFT1). In three completed Phase 1 studies in healthy subjects, the GCS inhibitor venglustat was generally safe and well tolerated. After repeated-dosing up to 15mg (14 days QD) with venglustat, timeand dose-dependent reductions in plasma GL-1 concentration were observed. No effect on serum creatinine, blood pressure or urinary output was observed in subjects treated with venglustat.
Journal Title: Kidney International Reports
Year Published: 2019
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