UCT expert steers successful study of US cholesterol drug trials

Dr Dirk Blom of the UCT/Groote Schuur's Hospital Division of Lipidology is first author of a New England Journal of Medicine (NEJM) paper showing that novel US drug Evolocumab reduces "bad cholesterol" by a further 57% when added to standard cholesterol-lowering therapy.

High levels of low-density lipoprotein cholesterol, "bad boy" LDLC, is a notorious risk factor for coronary artery disease. The latter is prevalent among South Africans of all population groups.

The drug, designed by pharmaceutical company AMGEN, was also well tolerated, and no safety concerns were identified.

"This is big news in the lipid world," said Blom of the study.

The 52-week study involved 88 sites across South Africa, Europe, Canada, the US, Australia, and Canada.

Other South African researchers that collaborated in this study were based at Tygerberg Hospital, the Paarl Research Centre, and Gauteng's Unitas Hospital.

The results were presented at the recent congress of the American College of Cardiology in Washington.

"It's a big accomplishment in the life of an academic many of us still aspire to," head of the Department of Medicine Professor Bongani Mayosi said of the paper. "Publication in the NEJM is an honour usually reserved for those who're involved in research that may change clinical practice."

Potentially welcome addition to current treatments

Though Evolocumab has yet to be submitted to the US Food and Drug Administration for approval, it's a potentially welcome addition to current cholesterol treatments.

"The medication is very effective for lowering bad cholesterol and will be a useful add-on, but it won't replace the standard drugs that we're using currently," Blom noted.

Existing drugs, though effective, are sometimes not strong enough for all patients, particularly those whose high cholesterol is genetically determined.

"Not everyone can tolerate high doses," Blom explained. "We need this drug for patients who don't respond adequately to or who have side effects such as muscle pain from other drugs, or statins.

"The big bang will come once the trials on cardiovascular outcomes are reported in a few years, if we can show that you don't only lower cholesterol with these drugs but also decrease heart disease."

Also new is that the drug is injected under the skin '“ in the same way diabetics administer insulin '“ except that it only needs to be given once every two or four weeks and not several times a day like insulin.

Patients will be taught to inject themselves at home using an auto-injector, which resembles a pen.

Story by Helen Swingler. Image by Michael Hammond.