Every year, around 15 million babies worldwide are born before 37 weeks of gestation. Being born preterm can disrupt development, and earlier births increase the risk of life-changing health outcomes. There are currently no treatment options for brain injury that occurs because of preterm birth, but progress has been made thanks to PREMSTEM.
Over six years and with support from the European Union and the National Health and Medical Research Council in Australia, our team of international experts has investigated whether human mesenchymal stem cells (h-MSCs) could be the key to helping children affected by early-life brain injury.
New understanding of how stem cells work
One of our primary goals in PREMSTEM was to uncover the answers to three key questions: when we should give the stem cells, how to give them – via the nose or bloodstream – and how many were needed to have a positive effect on models of brain injury.
Using h-MSCs from donated umbilical cord tissue, one of our early tasks was to test their effect in different models of brain injury, varying the timing and route of administration as well as the number of cells.
To manage this large amount of data, PREMSTEM researchers at Inserm and the University of Gothenburg worked with bioinformaticians to create a scoring system to identify the most effective combination of timing, dose and administration route. They saw that the most promising results came when delivering the stem cells via the nose and a short while after brain injury had occurred. This work has been published in the Journal of Neuroinflammation.
“Our comprehensive pre-clinical studies undertaken by multiple teams demonstrate that this type of stem cell can have a positive effect on brain damage in different models of perinatal brain injury. Excitingly we also saw them having a positive impact on other types of cells, helping the brain to repair itself after a traumatic event like preterm birth,” said Bobbi Fleiss from RMIT University.
Technologies to diagnose brain injury at the bedside
Another of our achievements has been to pioneer new brain imaging tools using ultrafast ultrasound that can be used to look into the brain at the bedside. Researchers at Physics for Medicine Paris and industry partner, Iconeus, have developed diagnostic tools that look for blood vessel abnormalities in cases of brain injury.
This exciting technology can help clinicians to better diagnose brain injury in children in a non-intrusive and comfortable way, keeping babies with their families even as we investigate their brains.
Stakeholder opinions embedded in the research
To complement and enrich our scientific endeavours, stakeholder engagement also played a central role in PREMSTEM. Through a series of co-creation activities led by RMIT Europe in collaboration with the Cerebral Palsy Alliance and the Global Foundation for the Care of Newborn Infants, PREMSTEM delivered a series of workshops and interviews with parents, patient advocates, neonatologists, clinicians, nurses and researchers. These online activities were facilitated by Enrique Conches at Punk Design.
The goal was to identify gaps in the information provided to the community about the safety and utility of stem cells and address societal concerns related to stem cell research. These activities resulted in a communications roadmap co-designed by the participants to guide future clinical research with families.
Over six years, PREMSTEM researchers have shown that h-MSCs can positively impact brain injury and inflammation in lab settings, paving the way for future, family-focused clinical trials in humans. Congratulations to the whole consortium!
PREMSTEM consortium: Inserm (coordinator), RMIT University, RMIT Europe, UMC Utrecht, Maastricht University, Radboudumc, University Hospital Essen, Gothenburg University, CNR Institute of Neuroscience, University of Geneva, Iconeus, Cerebral Palsy Alliance, Global Foundation for the Care of Newborn Infants, Inserm Transfert, Chiesi Farmaceutici.
