Researchers have identified a new way to treat young children with Kawasaki disease (KD) to prevent life-threatening coronary artery damage and heart attacks. 

The WEHI-led study showed that treatment with drugs called mTOR inhibitors can prevent coronary artery damage and the formation of aneurysms, a much-feared complication of KD. 

Importantly, mTOR inhibitors are already known to be safe and effective in other clinical uses, meaning this therapy could potentially be trialled rapidly in the clinic for children with KD. 

Cause unknown 

KD mainly affects children under five years of age, with up to 300 Australian children diagnosed with the disease each year. 

The specific cause is unknown, but it is generally accepted that infections with common respiratory viruses, including coronavirus, can trigger the disease. 

A key clinical feature of KD is inflammation of blood vessels, particularly the coronary arteries that supply blood to the heart. When severe, this inflammation can cause ‘remodelling’ of the coronary arteries leading to narrowing and aneurysms in damaged arteries. 

Risk of heart attack 

Professor Ian Wicks, Joint Head of Clinical Translation at WEHI, said even children with KD who survive aneurysms may sustain long-term damage to their coronary arteries, requiring treatment including surgery. 

“Preventing coronary artery damage is the top priority in KD treatment,” said Professor Wicks. 

“Current treatments effectively reduce the risk of developing potentially deadly aneurysms in most children, but we need more options for children whose KD is resistant to these treatments.” 

Prevention and treatment 

Study co-lead author Dr Angus Stock said the new findings suggested mTOR inhibitors were capable of both preventing and treating these adverse changes in KD patients who developed coronary artery aneurysms.

"mTOR inhibitors have a proven safety and efficacy profile in related clinical uses..."

“This is particularly exciting as mTOR inhibitors have a proven safety and efficacy profile in related clinical uses, such as preventing arteries from re-narrowing after coronary stenting in adults,” he said. 

The pre-clinical findings set the scene for clinical trials to assess a currently-used medicine to prevent coronary artery damage and heart disease in treatment-resistant KD.