Gene mutation reduces risk of coronary disease  

Niemann-Pick C1-like 1 (NPC1L1) protein is expressed in the small intestine and liver and facilitates the transport of intraluminal dietary cholesterol into enterocytes. This transporter is the target of the drug ezetimibe and inhibition of the transporter is known to significantly reduce plasma levels of LDL cholesterol. In this genetic study, the authors first sequenced […]

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Familial hypercholesterolaemia: new genes found

Familial hypercholesterolaemia is characterised by substantially raised plasma concentrations of low-density lipoprotein cholesterol (LDL-C) and is associated with a risk of coronary heart disease that is five to eight times higher than average. One charity has estimated a saving of £378.7 million from cardiovascular events avoided if all relatives of index cases were identified and […]

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PCSK9 antibody decreases LDL cholesterol in statin-intolerant patients

Approximately 10-20% of patients are unable to tolerate statins or the higher doses needed to achieve LDL cholesterol goals. Perprotein convertase subtilisin/kexin type 9 (PCSK9) mediates the binding and trafficking of LDL receptors, and in phase 1 studies a human monoclonal antibody to to PCSK9 (AMG145)lowered LDL levels. The Goal Achievement after Utilizing an anti-PCSK9 […]

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PCSK9 shows benefit in cholesterol lowering

Despite the potency of high dose statins, many patients fail to reach targets for LDL cholesterol reduction.  Whilst the addition of a second agent such as niacin or ezetemibe results in an additional 10-20% reduction in cholesterol, there remains an unmet need for more potent therapies.  Serum proprotein convertase subtilisin/kexin 9 (PCSK9) binds to low-density […]

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Novel cholesterol lowering agent shows promise

Serum PCSK9 plays an active role in controlling the expression of LDL receptors by targeting them for lysosomal destruction.  REGN727/SAR236553 (REGN727) is a novel human monoclonal antibody which inhibits PCSK9 binding to the LDL receptor; a previous phase 1 proof of concept trial suggested the potential for significant reductions in LDL in familial hypercholesterolaemia. […]

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LDL cholesterol lowering in CKD looks SHARP

While the cardiovascular benefits of LDL cholesterol lowering in patients without kidney disease are well established, the effects on people with moderate to severe kidney disease has remained unproven. The SHARP (Study of Heart and Renal Protection) study was a randomised double-blind trial that included 9270 patients with chronic kidney disease (3023 of whom were […]

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