Clinical trial shows eprotirome drug reduces blood cholesterol

A thyroid-hormone-like substance that works specifically on the liver reduces blood cholesterol with no serious side effects. This according to a clinical trial conducted by researchers from the Swedish medical university Karolinska Institutet, amongst other centres, published today in the top-ranking scientific periodical The New England Journal of Medicine.

High cholesterol levels in the blood are primarily treated with a group of drugs called statins, but they are not always sufficiently effective and higher doses commonly cause adverse reactions. A team of researchers, including scientists from Karolinska Institutet, have now shown in a clinical trial that a new drug substance called eprotirome can reduce blood cholesterol effectively in patients who have already received statins. Patients who were given supplementary medication with eprotirome demonstrated levels of harmful blood fats that were up to 30 per lower than those of patients who received a placebo supplementary treatment.

The trial lasted three months and included a total of 189 patients. It remains to be studied whether the drug candidate will be equally safe and effective for a larger group of patients over a longer period of time.

"This drug could help patients who react adversely to statins or be used as a supplementary treatment for those who don't respond well to them," says Professor Bo Angelin, who led the study.

Eprotirome mimics the natural ability of thyroid hormone to stimulate the metabolism of cholesterol, and exerts its effects exclusively on the liver. The development of similar non-selective drugs has previously been stopped on account of the serious adverse effects they have had on other organ systems (e.g. cardiac dilatation and osteoporosis) or on the physiological regulation of thyroid hormones.

Eprotirome has been developed by pharmaceutical company KaroBio in Huddinge, which is financing and participating in the research.

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