Promising new treatment for incurable premature lung disease

Hudson Institute researchers have discovered a potential safe and effective treatment for bronchopulmonary dysplasia (BPD), which could save preterm babies from the severe lifelong effects of this incurable premature lung disease.

The researchers, led by Associate Professor Marcel Nold and Dr Claudia Nold, together with senior scientist Dr Ina Rudloff at The Ritchie Centre showed that an anti-inflammatory drug, interleukin 1 receptor antagonist (IL-1Ra), could be given as a preventative measure in the hours after birth to prevent the development of BPD.

The findings of the study have just been published in the Journal of Cellular and Molecular Medicine.

“Having this treatment available will make a world of difference to premature babies and their families. This is a major breakthrough that will result in better understanding and prevention of BPD. This is a huge step forward in ensuring better outcomes for babies,” Life’s Little Treasures Foundation, Co-Founder/CEO, Shusannah Morris said.

A common preterm disease without a cure

Newborn babies’ immature lungs are often unable to cope with the essential and life-saving respiratory support they are placed on in hospital.

Up to 60 per cent of preterm babies develop BPD, an inflammatory lung disease that causes severe injury to the lung tissue and prevents normal lung growth, soon after birth. The more premature a baby is at birth, the higher the risk of BPD. There is currently no safe and effective treatment.

BPD affects the alveoli, the tiny sacs in the lungs that enable the entry of oxygen into the bloodstream, and the clearance of carbon dioxide from the body. Babies with BPD often suffer lifelong, severe, complications, including impaired neurodevelopment, and are highly susceptible to airway infections that may lead to death. As a result, some of these babies and their families will require ongoing medical care into childhood, some even into adulthood.

Timing is key

Previously, the Nolds’ laboratory showed that the anti-inflammatory drug IL-1Ra, currently used in diseases such as rheumatoid arthritis, was effective in preventing BPD. Here, they investigated optimal dose and timing for starting IL-1Ra treatment. IL-1Ra was shown to be most effective at protecting newborns against BPD when given at moderate doses and when started early, ideally within 12 hours of delivery.

“We found that IL-1Ra works best to prevent the development of BPD when it is given immediately after birth, before BPD can kick in and establish itself, and at a lower, rather than higher, dosage,”Dr Nold said.

The team also investigated the potential of protein C, which is currently used mainly as a medication to prevent blood clotting, to prevent BPD from developing.

“The two drugs act in different ways, meaning there might be benefit in using them together to complement one another,” Dr Nold said.

The drugs target distinct pro-inflammatory responses that cause BPD, which prevents the disease from forming, the study showed.

Well-tolerated, established drugs

“Both drugs are well tolerated and are shown to be safe in children, so it appears likely that they could be safe to use in preterm babies as well. This will have to be shown by clinical trials, which we hope can begin soon,” Dr Rudloff said.

“Our findings may pave the way for a preventive treatment for BPD that could be given to premature babies at highest risk of developing the disease.”

The safety profiles of IL-1Ra and protein C are markedly more favourable than those of corticosteroids, which are currently the only drugs available to treat (but not prevent) BPD.

“We are very hopeful that alone or in combination, IL-1Ra and protein C could one day be introduced as a safe therapy for at-risk premature infants,” Dr Nold said.

“With no safe or effective treatment for these babies, this work provides families with new hope and a healthier lifelong outlook.”

How does IL-1Ra work?

IL-1Ra is a natural protein and is the inhibitor of IL-1, one of the most important inflammatory cytokines. The body uses IL-1Ra to curb excessive inflammation. In some diseases that involve chronic inflammation such as rheumatoid arthritis, the patient often does not produce enough IL-1Ra, and in these cases the IL-1Ra can be used to supplement the body’s natural stores and act as an anti-inflammatory drug.

Next steps

Before they can commence a first-in-human trial of the drug in premature babies, the team must undertake an important preclinical study.

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