Delivering hope: anti-inflammatory drug trial for preterm babies

Blessing was born very early and in a hurry at 25 weeks, but she’s a battler. 

Her parents immediately enrolled her in a drug trial, so, in the future, other preterm babies can beat the odds, too.

The trial is a pilot study of a drug, called ‘anakinra’, already used for inflammatory diseases in older children and adults, which neonatal doctors hope could help very early babies, too.

Blessing was mother Virginia Vavau’s first preterm birth, with three older siblings all born at full term. 

“I wanted to take part to help other preterm babies – and Blessing,” Mrs Vavau says. 

Baby Blessing needed to stay in an incubator in the neonatal unit at Te Whatu Ora Starship Children’s Hospital at Te Toka Tumai Auckland, where neonatologist Dr Gergely Toldi, was one of the team caring for her.

Dr Toldi is also a researcher at Waipapa Taumata Rau, University of Auckland’s Liggins Institute. He is leading the Aotearoa New Zealand arm of the anakinra pilot trial in collaboration with colleagues at Monash University’s School of Clinical Sciences and Hudson Institute of Medical Research in Melbourne, Australia.

Dr Toldi hopes anakinra will help preterm babies for whom inflammation can be very dangerous. 

“Inflammation can lead to lung disease resulting in babies needing breathing support and additional oxygen for long periods of time, even beyond discharge from the neonatal unit,” he says.

“It can also cause bowel problems, which may necessitate surgery, as well as brain injury, leading to long-term developmental problems, and potentially resulting in disability.”

In the trial, anakinra is used daily for the first three weeks of the preterm babies’ lives. 

“This novel therapeutic approach may be a game-changer in our clinical management of preterm babies,” says Dr Toldi.

“Reducing the burden of complications arising from prematurity is a number one healthcare and socioeconomic priority, and this treatment will hopefully do just that.”

Professors Marcel and Claudia Nold, a husband-and-wife team, lead the team of researchers across Monash University, Hudson Institute and Monash Children’s Hospital. See Hudson Institute media release.

The Anakinra pilot study builds on a programme of research on early life inflammation they have conducted since 2009. The pair published an important part of their work this year in the journal, Science Translational Medicine, which identified a particular type of inflammation which drives lung and heart disease in preterm babies.

“This new knowledge allows us to work on ways to control inflammation in preterm babies and prevent lifelong health problems,” Dr Marcel Nold says.

Results from New Zealand whānau will be pooled with those from Australia and analysed in Australia.

After the pilot study, a much larger multi-centre trial is planned, which will test the theory that anakinra effectively ameliorates or even prevents diseases caused by inflammation in preterm babies.