A new drug for one of the leading causes of heart disease

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Calcific aortic valve disease is not only the most common valve disease in the elderly, it’s also the third leading cause of heart disease overall.

For those affected, calcium starts to accumulate in their heart valves and vessels over time, until they harden like bone.

As a result, blood flow out of the heart’s pumping chamber to the body gets obstructed, leading to heart failure.

Yet no medical therapy currently exists. All patients can do is wait for the calcification (or hardening) to become bad enough that they need surgery to replace their valve.

In a new study, researchers discovered a potential drug candidate for heart valve disease that works in both human cells and animals and is ready to move toward a clinical trial.

The research was conducted by a team of scientists from Gladstone Institutes.

Heart valve disease is often diagnosed at an early stage and calcification of the heart valves worsens over the patient’s lifetime as they age.

If doctors could intervene early in life with an effective drug, they could potentially prevent the disease from occurring.

By simply slowing the progression and shifting the age of people who require interventions by 5 or 10 years, they could avoid tens of thousands of surgical valve replacements every year.

This also applies to the millions of Americans—about 1% to 2% of the population—with a congenital anomaly called a bicuspid aortic valve, in which the aortic valve only has two leaflets instead of the normal three.

While some people may not even know they have this common heart anomaly, many will be diagnosed as early as their forties.

In the study, the team searched for drug-like molecules that could correct the overall network that goes awry in heart valve disease and leads to calcification.

Once they had identified a promising drug candidate, they did a “pre-clinical trial” in a mouse model of the disease. They wanted to determine whether the drug-like molecule would actually work in a whole, living organ.

The scientists confirmed that the therapeutic candidate could successfully prevent and treat aortic valve disease.

They hope their approach can offer a new direction that could increase the likelihood of candidate therapies being effective in patients.

One author of the study is Gladstone President and Director of the Roddenberry Stem Cell Center Deepak Srivastava, MD.

The study is published in Science.

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