Compactin inhibits cholesterol synthesis in lymphocytes and intestinal mucosa from patients with familial hypercholesterolaemia.
Compactin (2000 nmol/l), a potent competitive inhibitor of the rate-determinign enzyme for cholesterol synthesis (3-hydroxy-3-methyl-glutaryl coenzyme-A reductase), inhibited by about 90% cholesterol synthesis from 14C-acetate in isolated lymphocytes from healthy control and from patients heterozygous for familial hypercholesterolaemia (F.H.). In isolated intestinal mucosa, a physiologically important tissue for cholesterol synthesis, compactin (2000 nmol/l) suppressed cholesterol synthesis by approximately 50% in controls and F.H. patients. Compactin or its derivatives may prove to be useful in the treatment of hypercholesterolaemia.
D000085:Acetates / Q000378:metabolism; D000924:Anticholesteremic Agents / Q000494:pharmacology*; D002250:Carbon Radioisotopes; D002478:Cells, Cultured; D002784:Cholesterol / Q000096:biosynthesis*; D003864:Depression, Chemical; D004791:Enzyme Inhibitors / Q000494:pharmacology; D006579:Heterozygote; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D006937:Hypercholesterolemia / Q000097:blood / Q000235:genetics*; D066298:In Vitro Techniques; D007413:Intestinal Mucosa / Q000378:metabolism*; D007583:Jejunum / Q000378:metabolism*; D008214:Lymphocytes / Q000378:metabolism*; D008798:Mevalonic Acid / Q000378:metabolism; D009281:Naphthalenes / Q000494:pharmacology*
D000085:Acetates; D000924:Anticholesteremic Agents; D002250:Carbon Radioisotopes; D004791:Enzyme Inhibitors; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D009281:Naphthalenes; D002784:Cholesterol; D008798:Mevalonic Acid