This Rare Disease Drug Can Reduce Heart Disease Risk

An exciting new development came out of presentations at the European Society of Cardiology conference in Spain this past weekend.

We often hear about how previously approved FDA drugs for more common diseases may have off-label uses for one of the 7,000 rare diseases that have limited treatment options.
But the Novartis drug ILARIS (hencforth referred to as canakinumab), used to treat rare diseases like CAPS, systemic JIA, and familial Mediterranean fever, has recently shown that it can treat a rather common problem: heart attacks.

Canakinumab works by lowering inflammation through reducing c-reactive protein (CRP), which is a completely novel approach to controlling heart attack risk compared to typical treatments which are aimed at lowering cholesterol. Statistically, 25% of those who have a heart attack will have another within five years, and in 50% of those cases, inflammation is the cause. Some of this inflammation goes undetected, and it can damage arteries making blood cots more likely to form.

The study participants were all on heart medicine in addition to varying doses of canakinumab, or a placebo. Those who were on the medium dose of canakinumab had a 15% lower chance of heart-related issues over the next four years versus those who were on placebos. And while that might not seem like a big improvement in, it makes all the difference when heart disease accounts for 25% of deaths in the United States.

Not only does this present a new potential use for canakinumab, but it will help researchers target inflammation when treating heart diseases, which previously was not a big focus. Another pretty cool result from this study showed that participants taking canakinumab had a lower instance of cancer deaths, especially those involving the lungs. This result will have to be observed further in a focused study, as the drug has the potential to slow tumor growth.

Canakinumab does have side effects, including risk of fatal infections, so it’s not exactly a miracle drug. But it is a promising drug that may have more uses than previously thought. Additionally, canakinumab is super expensive for the treatment of rare disease, so that’s another aspect to think about. Either way, this is good news for the heart disease community and we can’t wait to see where research goes from here.

To know more, check out the study published in the New England Journal of Medicine and further explained by CBS News.

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