The HDL mimetic CER-001 remodels plasma lipoproteins and reduces kidney lipid deposits in inherited lecithin:cholesterol acyltransferase deficiency.
J Intern Med
; 291(3): 364-370, 2022 03.
Article
em En
| MEDLINE
| ID: mdl-34761839
ABSTRACT
BACKGROUND:
Kidney failure is the major cause of morbidity and mortality in familial lecithincholesterol 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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Deficiência da Lecitina Colesterol Aciltransferase
Limite:
Humans
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article