New therapy for familial hypercholesterolemia reported

Reported by Susan Aldridge, PhD, medical journalist

Research has revealed a promising new approach to cholesterol lowering in familial hypercholesterolemia.
People with familial hypercholesterolemia (FH) run a very high risk of premature heart disease. They develop very high cholesterol levels because of mutations in one of the genes that would normally help keep it under control. Often, patients with FH do not respond well to statin therapy, so new approaches are needed.

A team at the University of Pennsylvania now reveals that targeted inhibition of the microsomal triglyceride transfer protein (MTP) can work to lower cholesterol in people with FH. It is known that mutations in MTP lead to very low levels of low density lipoprotein (LDL or 'bad') cholesterol. Therefore, blocking MTP may have the same effect.

A trial was carried out with an experimental drug called BMS-201038 which has the power to block MTP. Patients with FH were given varying doses of the drug for four weeks and experienced an impressive 51 per cent reduction in LDL cholesterol and a 58 per cent reduction in total cholesterol. Of course, longer trials are now needed, but these initial results look very promising.

Source
New England Journal of Medicine 11th January 2007

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