Evaluation of world-first study involving stem cells for premature babies with chronic lung disease

Atul Malhotra
Monash researchers with one of the infants involved in the study

Monash University researchers have assessed the long-term outcomes of their world-first study that used placental stem cells in premature babies with severe bronchopulmonary dysplasia, a form of chronic lung disease.

The first-in-human study led by Dr Atul Malhotra (Department of Paediatrics and Monash Children’s Hospital), in collaboration with Professor Euan Wallace (Department of Obstetrics and Gynaecology), and Associate Professor Rebecca Lim (Hudson Institute of Medical Research), tested a potential new therapy to help premature babies with chronic lung disease fight off the debilitating condition.

Each year in Australia, approximately 3,500 neonates are born before 32 weeks gestation. While outcomes for these babies have improved over the past few decades, a significant percentage of them go on to develop chronic lung disease – making their lungs stiff, and breathing difficult.

Using placental stem cells from healthy donor placentas, researchers injected the stem cells into neonates with chronic lung disease to assess the safety of using the therapy, and to help repair damage and possibly prevent the disease.

“We believe these placental stem cells help ‘kick start’ the baby's own immune system to ‘empower’ them to prevent or fight this debilitating condition,” said Dr Malhotra. “Given this was the first time any baby had ever received this therapy, assessing the long-term safety over the first 2 years of life was vital for this research to progress to the next step.”

The study followed the first five infants to receive this therapy until 2 years corrected age and assessed events, growth, respiratory, cardiac, and neurodevelopmental outcomes at 6, 12, 18, and 24 months of age. There were no adverse events noticed that could be attributed to the stem cell administration.

“The primary outcome of the trial was safety. We now know it is safe, next we need to show it is effective and then this therapy could be a potential game changer if we show it works," said Dr Malhotra. A dose-escalation study is currently underway to ascertain the optimum dose likely to be effective in the prevention of chronic lung disease.

With clinicians and scientists trying for decades to find solutions for chronic lung disease related to prematurity, the research signifies the first steps towards finding a solution to this major health problem. A decrease in chronic lung disease rates or its severity will result in these premature babies breathing much more comfortably as they grow older.

“Chronic lung disease has considerable impacts on the baby, the family and the community. If we can lessen the burden of this significant complication, everyone stands to gain from it.”

The outcomes of the study have been published in Stem Cells Journals.