Safety Concerns for Experimental LDL-Reduction Drug

An investigational microsomal triglyceride transfer protein inhibitor lowered LDL levels — but had adverse liver effects — in patients with homozygous familial hypercholesterolemia.

Patients with homozygous familial hypercholesterolemia, which is caused by loss-of-function mutations in the LDL receptor gene, respond poorly to conventional LDL-lowering therapy. As an alternative, these patients can undergo LDL apheresis, which must be repeated frequently and is not widely available. Scientists postulate that inhibiting microsomal triglyceride transfer protein (MTP), which plays a role in assembling a precursor to LDL, could reduce LDL production and plasma LDL levels.

In an open-label trial, the investigational MTP inhibitor BMS-201038 was given to six patients with homozygous familial hypercholesterolemia (age 18–40) after they had stopped all lipid-lowering therapy and apheresis. The drug was administered in four, grad…

Reviewing Author

Harlan M. Krumholz, MD, SM

Safety Concerns for Experimental LDL-Reduction Drug

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Reviewing Author