• Chiara Pavanello, Marta Turri, Arianna Strazzella, Patrizia Tulissi, Stefano Pizzolitto, Giovanna De Maglio, Riccardo Nappi, Laura Calabresi, and Giuliano Boscutti.
• Dipartimento di Scienze Farmacologiche e Biomolecolari, Centro E. Grossi Paoletti, Università degli Studi di Milano, Milan, Italy.
• J. Intern. Med. 2022 Mar 1; 291 (3): 364-370.

BackgroundKidney 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.MethodsCER-001 was tested in an FLD patient with dramatic kidney disease for 12 weeks.ResultsInfusions 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.ConclusionThis 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.© 2021 The Authors. Journal of Internal Medicine published by John Wiley & Sons Ltd on behalf of Association for Publication of The Journal of Internal Medicine.

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