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- Title
The HDL mimetic CER‐001 remodels plasma lipoproteins and reduces kidney lipid deposits in inherited lecithin:cholesterol acyltransferase deficiency.
- Authors
Pavanello, Chiara; Turri, Marta; Strazzella, Arianna; Tulissi, Patrizia; Pizzolitto, Stefano; De Maglio, Giovanna; Nappi, Riccardo; Calabresi, Laura; Boscutti, Giuliano
- Abstract
Background: Kidney failure is the major cause of morbidity and mortality in familial lecithin:cholesterol acyltransferase deficiency (FLD), a rare inherited lipid disorder with no cure. Lipoprotein X (LpX), an abnormal lipoprotein, is primarily accountable for nephrotoxicity. Methods: CER‐001 was tested in an FLD patient with dramatic kidney disease for 12 weeks. Results: Infusions of CER‐001 normalized the lipoprotein profile, with a disappearance of the abnormal LpX in favour of normal‐sized LDL. The worsening of kidney function was slowed by the treatment, and kidney biopsy showed a slight reduction of lipid deposits and a stabilization of the disease. In vitro experiments demonstrate that CER‐001 progressively reverts lipid accumulation in podocytes by a dual effect: remodelling plasma lipoproteins and removing LpX‐induced lipid deposit. Conclusion: This study demonstrates that CER‐001 may represent a therapeutic option in FLD patients. It also has the potential to be beneficial in other renal diseases characterized by kidney lipid deposits.
- Subjects
BLOOD lipoproteins; ACYLTRANSFERASES; LIPIDS; KIDNEY failure; FAMILIAL hypercholesterolemia; RENAL biopsy; KIDNEY diseases
- Publication
Journal of Internal Medicine, 2022, Vol 291, Issue 3, p364
- ISSN
0954-6820
- Publication type
Article
- DOI
10.1111/joim.13404