Cord Blood Cell Therapy in Preterm Babies: Safety Signals and Early Outcomes Offer Encouraging Next Steps
Late last year, Australian researchers published world-first results from the CORD-SaFe study, showing that autologous cord blood cell therapy was both safe and feasible for extremely premature babies. The pioneering phase 1 clinical trial, conducted through a partnership between Monash Children’s Hospital, the School of Clinical Sciences at Monash University, and Hudson Institute of Medical Research, demonstrated that a baby’s own umbilical cord blood cells could be collected and reinfused shortly after birth, with no serious side effects reported.
Now, in a newly published follow-up in JAMA Network Open, the same team has shared early developmental outcomes from infants who received the therapy. These findings offer valuable insights into how these babies are progressing in their first year of life, and help lay the groundwork for future clinical trials.
“This isn’t just about whether the therapy is possible—we now have early reassurance about how these babies are developing,” said Associate Professor Atul Malhotra, lead investigator and neonatal specialist. “It’s a step forward in our mission to reduce the burden of brain injury after extremely preterm birth.”
What This Study Adds
The original CORD-SaFe study, published in Lancet eBioMedicine in December 2024, was the first in the world to show that cord blood cells could be safely collected and reinfused into extremely premature babies, those born before 28 weeks’ gestation. These infants face a high risk of brain injury and long-term disability, yet treatment options remain extremely limited. Proving that this kind of personalised cell therapy is both feasible and well tolerated marked a major milestone in neonatal medicine.
The new analysis builds on those findings, reporting how the babies were developing in the first few months of life following discharge from neonatal care. Researchers used both brain scans and early neurological assessments to compare 23 infants who received the therapy with 93 similar infants who did not.
One of the most striking observations was that none of the babies who received the cord blood therapy were assessed as being at high risk of cerebral palsy, based on clinical assessments at follow-up. In comparison, about 7 in 100 babies in the non-treatment group were considered at high risk. These risk assessments were based on developmental signs observed after birth, not pre-existing diagnoses, making the absence of high-risk findings in the treatment group a potentially meaningful signal.
While the study wasn’t designed to test if the therapy works but rather provide a read out of safety, the team notes that this early pattern is encouraging and deserves further investigation.
A Closer Look at the Brain
To better understand how the babies’ brains were developing, the team used MRI scans, a type of detailed imaging, at what’s known as term-equivalent age, or around the time the baby would have reached full-term if carried to 40 weeks. These scans were evaluated using a system called the Kidokoro score, which measures the extent of brain injury in different areas. This structured approach is increasingly used in neonatal research but has rarely been applied in clinical trials of cord blood therapies for preterm infants.
“We’re learning more than just whether this treatment is safe. We’re getting a window into the developing brain,” said Dr Lindsay Zhou, neonatologist and co-lead author. “That’s incredibly powerful, especially in this high-risk population.”
In addition to brain imaging, babies were assessed around 52 to 54 weeks postmenstrual age, roughly one year after conception, using tools designed to spot early signs of developmental delay. These included the Hammersmith Infant Neurological Examination and the General Movements Assessment, which help identify subtle motor changes before a child reaches milestones like walking or sitting independently.
While the study was too small to detect whether the therapy prevents cerebral palsy or other conditions, the consistent finding of no high-risk cases among treated infants offers a positive early signal and highlights the need for larger, longer-term studies.
Looking Ahead: The CORD-CELL Trial
With both safety and early developmental results now published, the next step is already underway: a larger international trial known as CORD-CELL. Also led from Australia, this study will include enough babies to properly test whether the therapy improves long-term outcomes.
Because cerebral palsy is relatively rare, even in very premature infants, a much larger number of participants is needed to understand whether cell therapy makes a significant difference. CORD-CELL is the essential next phase in determining whether this promising treatment can reduce disability and improve quality of life.
“This is a careful and ethical next step,” said Associate Professor Malhotra. “The CORD-CELL trial will help us answer the big question: can this therapy truly change outcomes for these babies?”
If successful, this research could help bring personalised, regenerative therapies into mainstream neonatal care, supporting brain development in the tiniest and most vulnerable patients and offering new hope for families facing the challenges of extreme preterm birth.
About Monash University
Monash University is Australia’s largest university with more than 80,000 students. In the 60 years since its foundation, it has developed a reputation for world-leading high-impact research, quality teaching, and inspiring innovation.
With four campuses in Australia and a presence in Malaysia, China, India, Indonesia and Italy, it is one of the most internationalised Australian universities.
As a leading international medical research university with the largest medical faculty in Australia and integration with leading Australian teaching hospitals, we consistently rank in the top 50 universities worldwide for clinical, pre-clinical and health sciences.
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