Breakthrough Trial Reveals Positive Impact of Umbilical Cord Blood on Motor Skills in Some Children With Cerebral Palsy

In a groundbreaking randomized clinical trial published in Stem Cells Translational Medicine, the infusion of a child's own umbilical cord blood is shown to enhance brain connectivity and motor function in children with spastic cerebral palsy. The placebo-controlled phase two trial involved 63 children exhibiting varied types and severities of spastic cerebral palsy, a condition often stemming from brain damage before or during birth.

Participants who received a single intravenous dose of at least 25 million stem cells per kilogram of their body weight demonstrated significant improvements in motor function one year later. Notably, these improvements surpassed typical gains observed in children of similar age and condition. In contrast, those who received a lower cell dose or a placebo experienced less progress.

Senior author Dr. Joanne Kurtzberg, the director of Duke’s Pediatric Blood and Marrow Transplant Program, expressed optimism about the study's results, highlighting the potential of appropriately dosed cord blood cell infusions to alleviate symptoms in children with cerebral palsy. Despite these promising outcomes, Dr. Kurtzberg acknowledged the need for further research to optimize and broaden accessibility to this therapy for more children with cerebral palsy.

Dr. Kurtzberg, renowned for her pioneering work on the therapeutic potential of umbilical cord blood, emphasized the safety of receiving an infusion of a child's own cord blood, paving the way for additional studies. Future research will explore the benefits of multiple cell doses and the utilization of donor cells for patients without banked cord blood.

The trial tested doses ranging from 10 million to 50 million cells per kilogram, with evaluations conducted through MRI to measure brain connectivity and the Gross Motor Function Measure (GMFM-66). The GMFM-66 assesses a child's ability to perform various movements based on age and development.

While children with cerebral palsy are expected to gain motor function through growth and traditional therapies, the study revealed that those who received at least 25 million cells per kilogram demonstrated progress beyond the expected increases a year after infusion. Dr. Jessica Sun, lead author of the paper, emphasized that even subtle improvements, such as a change in hand orientation, can significantly impact a child's daily life.

Despite some limitations in participant requirements and potential influences from well-resourced families, the study provides hope for the role of cord blood and cell therapy in treating cerebral palsy and brain injuries. The research, supported by the Robertson Foundation and The Marcus Foundation, encourages ongoing exploration of these promising therapeutic avenues.

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Source: https://corporate.dukehealth.org/news/umbilical-cord-blood-improves-motor-skills-some-children-cerebral-palsy?h=nl