Human Stem Cells Show Potential to Treat Neonatal Brain Injury

Research led by RMIT University has made significant strides in the treatment of neonatal brain injury, particularly for babies born preterm. Every year, approximately 15 million infants worldwide are delivered before 37 weeks of gestation, often resulting in serious health complications. Despite the absence of effective treatments for brain injuries linked to premature birth, the PREMSTEM project has emerged as a promising initiative exploring the use of human mesenchymal stem cells (h-MSCs) for therapeutic purposes.

Exploring Stem Cell Efficacy

The PREMSTEM project, a collaboration involving international experts, aims to determine the optimal timing, method of administration, and dosage of h-MSCs to maximize their benefits for children suffering from early-life brain injuries. According to Associate Professor Bobbi Fleiss from RMIT’s School of Health and Biomedical Sciences, the project seeks to answer three critical questions regarding stem cell treatment.

Using stem cells derived from donated umbilical cord tissue, the research team conducted experiments across various models of brain injury. They assessed the impact of different timing and routes of administration, including delivery via the bloodstream or nasal passages. To manage the extensive data generated, the team collaborated with bioinformaticians to develop a scoring system aimed at identifying the most effective combinations of timing, dosage, and delivery methods.

Promising results indicated that administering stem cells nasally shortly after brain injury occurred yielded the most beneficial outcomes. Fleiss noted, “Our comprehensive pre-clinical studies demonstrate that this type of stem cell can positively affect brain damage in various models of perinatal brain injury.” The research also revealed that h-MSCs had a positive influence on other cells, aiding the brain’s self-repair mechanisms post-trauma.

Innovative Diagnostic Tools

In addition to exploring stem cell therapy, the PREMSTEM project has developed innovative diagnostic tools for assessing brain injuries in infants. Researchers from Physics for Medicine Paris and industry partner Iconeus have created ultrafast ultrasound imaging techniques, allowing clinicians to examine the brain at the bedside.

This new technology can identify abnormalities in blood vessels associated with brain injuries, offering a non-invasive and comfortable diagnostic option for young patients. Fleiss emphasized the significance of these advancements, stating, “This exciting technology can help clinicians better diagnose brain injury in children, allowing families to remain close while their condition is assessed.”

To enhance the project’s scientific endeavors, RMIT Europe played a vital role in stakeholder engagement. Through workshops and interviews with parents, patient advocates, and medical professionals, the team worked to address community concerns regarding the safety and efficacy of stem cell research. This collaborative effort resulted in a communications roadmap designed to guide future clinical research involving families.

Over the course of six years, the PREMSTEM team has demonstrated that h-MSCs can positively impact brain injury and inflammation in laboratory settings. These findings pave the way for future clinical trials focused on delivering family-centered treatments for infants affected by preterm birth.

The research findings are detailed in the Journal of Neuroinflammation. This study represents a significant step forward in understanding how stem cells can be utilized to improve health outcomes for vulnerable populations, particularly those born prematurely.

For more information, refer to the article by Cindy Bokobza et al., titled “A systematic scoring system to optimise the testing of neurotherapeutics in models of perinatal brain injury, with an applied case study of human umbilical-cord MSC,” published in the Journal of Neuroinflammation (2025). DOI: 10.1186/s12974-025-03593-2.