A new understanding of cystic fibrosis

Cystic Fibrosis (CF) is widely referenced as the most common life-shortening disease affecting young Australians today, however with the advances in medical research and treatment, CF patients are now living longer than ever before. With these great improvements in life expectancy, many clinicians are now questioning the unknown long-term effects of living with CF.

Thanks to support from the Cystic Fibrosis Research Trust Auxiliary, Tom Saunders, the 2018/19 Respiratory Medicine Fellow, is being supported to delve deeper into potential long term effects of CF and its treatments. Now in his second year of research of a four year grant, Tom spreads his time between clinical and research work.

“I was motivated to specialise in respiratory medicine because you get to help a huge variety of different kids. Everyone at some point in their life experiences difficulty breathing, and it was during my placement at the RCH that I developed a passion working with children and their families. It was through consultation with Professor Sarath Ranganathan, Director of Respiratory Medicine and Professor David Burgner, Group Leader of the Inflammatory Origins Research Group at MCRI, that we identified the cardiovascular effects of CF as an area desperately needing more research,” said Tom.

What CF patients are hoping to prevent is atherosclerosis. This condition occurs when there’s a build-up of fatty plaque inside the arteries. Over time, this plaque build-up blocks the arteries, stopping the flow of oxygen-rich blood to the organs including the heart and brain, potentially causing a heart attack or stroke.

Cardiovascular disease is the biggest killer of Australians. It is only relatively recently that experts have started showing that inflammation is the key culprit leading to disease, rather than just high blood pressure or smoking causing damage to arteries. And for patients with CF, this is a big concern.

“Patients with cystic fibrosis have lots of inflammation, and are usually underweight. As part of their treatment plans, we strongly recommend they have a high fat diet to help protect the lungs and put needed vitamins into their bodies. While this diet helps to support their childhood treatment, the long term effects on blood vessels and the heart have never been studied before,” Tom said.

To better understand the potential impacts of CF on patient’s arteries, Tom has developed a comprehensive process to measure cardiovascular health and potential long-term risk. Starting with a full family history of heart disease, Tom and his team will build a profile that includes the patient’s height, weight and body mass index. He will be conducting ultrasounds of the larger arteries checking for any existing plaque, looking at the blood vessels at the back of the patient’s eye, and finally taking a blood test to examine the activity of immune cells.

“Our aim is to enrol 50 per cent of Victoria’s children with CF; around 150 young people aged 5 to 18 years. It’s an incredibly ambitious study that no one has done before, especially not at this scale, and we’re hoping it will provide some essential data on a patient’s risk of stroke or heart attack in middle age to improve their quality of life,” said Tom.

“Our hope is that if we can identify early changes that may put them at risk of a heart attack or stroke later in life, we can intervene in childhood and hopefully prevent it ever happening.”

For Tom, this study is forming the basis of his PhD. Currently at the stage of ethics approval, Tom anticipates the research will take around six years to complete; two of which will be dedicated to data collection. As a Respiratory Medicine Fellow, Tom is managing this research alongside his clinical work on the hospital wards and in outpatient clinics.

“Without the support of the CF Research Trust Auxiliary, I wouldn’t be able to have protected time to commit to my research, and this important research just wouldn’t happen. Thank you so much for making this possible!”