Potential of fibrates in the treatment of fatty acid oxidation disorders: revival of classical drugs?
Exposure to fibrates leads to normalization of fatty acid oxidation (FAO) in fibroblasts from patients with myopathic forms of CPT2 deficiency or VLCAD deficiency. Correction of FAO is related to a drug-induced increase of residual enzyme activity, and this could provide a new treatment strategy for these disorders.
Djouadi
F
F
INSERM U393, Hôpital Necker, Tour Lavoisier, 149 rue de Sèvres, 75015, Paris, France.
Aubey
F
F
Schlemmer
D
D
Gobin
S
S
Laforet
P
P
Wanders
R J A
RJ
Strauss
A W
AW
Bonnefont
J P
JP
Bastin
J
J
D044942:Acyl-CoA Dehydrogenase, Long-Chain / Q000096:biosynthesis / Q000172:deficiency / Q000235:genetics; D001629:Bezafibrate / Q000494:pharmacology; D002334:Carnitine O-Palmitoyltransferase / Q000096:biosynthesis / Q000172:deficiency / Q000235:genetics; D002478:Cells, Cultured; D002995:Clofibric Acid / Q000494:pharmacology* / Q000627:therapeutic use; D004790:Enzyme Induction / Q000187:drug effects; D005227:Fatty Acids / Q000378:metabolism; D005347:Fibroblasts / Q000187:drug effects* / Q000201:enzymology; D006801:Humans; D000960:Hypolipidemic Agents / Q000494:pharmacology* / Q000627:therapeutic use; D008052:Lipid Metabolism, Inborn Errors / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D008928:Mitochondria / Q000187:drug effects / Q000201:enzymology; D010084:Oxidation-Reduction / Q000187:drug effects; D047495:PPAR gamma / Q000819:agonists* / Q000378:metabolism; D012333:RNA, Messenger / Q000096:biosynthesis
D005227:Fatty Acids; D000960:Hypolipidemic Agents; D047495:PPAR gamma; D012333:RNA, Messenger; D002995:Clofibric Acid; D044942:Acyl-CoA Dehydrogenase, Long-Chain; D002334:Carnitine O-Palmitoyltransferase; D001629:Bezafibrate