Cerebral palsy is a group of disorders affecting a person’s ability to move and maintain balance, and it is the most common physical disability in childhood. Umbilical cord blood, the blood left in the umbilical cord and placenta after birth, is a rich source of stem cells. For two decades, researchers have explored using these cells as a therapeutic option for individuals with cerebral palsy.
The Function of Cord Blood Stem Cells in the Brain
A common misconception is that infused stem cells travel to the brain to replace damaged ones. Instead, the primary mechanism is indirect. The various cells within cord blood have a modulatory effect on the brain’s environment, releasing signaling molecules like cytokines and growth factors that act to reduce inflammation.
This anti-inflammatory action is a proposed benefit, as ongoing inflammation can contribute to further injury in a brain affected by cerebral palsy. The cells from cord blood function like paramedics at an injury site. They do not rebuild damaged structures, but calm the environment, protect existing neurons, and support the brain’s innate repair capabilities.
This process helps create a more favorable environment for neural plasticity, the brain’s ability to reorganize itself by forming new neural connections. Growth factors released by the cord blood cells can encourage the survival of existing neurons and may support oligodendrocyte progenitor cells. These cells are important for producing myelin, the protective sheath around nerve fibers often damaged in cerebral palsy.
Current State of Clinical Research
Clinical trials over the past 20 years, involving over 800 participants, have consistently demonstrated that cord blood infusion is a safe procedure. Several of these trials have also reported measurable improvements in motor function for some children. While the therapy is still considered investigational and not a standard of care, the body of evidence for its potential is growing.
A significant portion of this research has been conducted at Duke University Medical Center. Early studies focused on autologous infusions, where children received their own cord blood banked at birth. One phase 2 study involving 63 children with cerebral palsy found that appropriately dosed infusions could lessen symptoms, establishing a minimum effective dose of 25 million cells per kilogram of body weight.
Subsequent research has expanded to allogeneic treatments, using cord blood from related donors like siblings. An Australian clinical trial investigated the safety and efficacy of using sibling cord blood, confirming the procedure’s safety. The study observed that some children showed greater developmental improvements than anticipated, particularly in gross motor skills, though these changes were less pronounced at the one-year follow-up.
More recent work at Duke has continued to refine the approach, including a study testing higher doses of donor cord blood. This study reinforced that cord blood was beneficial, while also testing mesenchymal stromal cells (MSCs), which did not show a benefit. Research is also occurring in Europe, with programs providing access to autologous cord blood therapy for children with neurological conditions.
The Treatment Process
The source of the cord blood falls into two main categories. The first is autologous treatment, which uses the child’s own umbilical cord blood. This option is only available if parents privately banked their child’s cord blood at birth, which ensures a perfect genetic match and eliminates the risk of rejection.
The second category is allogeneic treatment, which uses cord blood from a donor, most commonly a healthy, immunologically matched sibling. It may also be possible to find a match from an unrelated donor through a public cord blood bank. The success of allogeneic infusions depends on a close tissue type match to minimize potential immune reactions.
The administration of the cord blood is a relatively straightforward medical procedure given as an intravenous (IV) infusion, much like a standard blood transfusion. The process is brief, often completed in under an hour. During the infusion, the child is closely monitored by a medical team for any signs of adverse reactions.
Eligibility for these treatments, conducted within clinical trials or special access programs, is specific. Criteria often include the child’s age, typically between one and six years old, during a period of significant brain development. The type and severity of cerebral palsy are also considered, with studies focusing on children who sustained brain damage before or at birth.
Efficacy and Limitations of Therapy
Cord blood infusion is not a cure for cerebral palsy. The improvements documented in clinical trials are generally modest, although they can be meaningful for children and their families. The therapy does not reverse the underlying brain injury but aims to improve function by supporting the brain’s own repair mechanisms.
A limitation is that not every child who receives the treatment shows improvement, and the factors that determine who will respond best are still under investigation. Research suggests that a higher cell dose is more effective, and younger children may see greater gains in gross motor function. The long-term effects are also still being studied, as some findings indicate initial improvements may become less pronounced over time.
The most significant logistical barrier for many families is the requirement for banked cord blood. For an autologous infusion, the child’s own cord blood must have been privately stored at birth. If a child’s own cord blood is not available, finding a matched sibling donor is the next best option, but this is not possible for many families.
Ongoing research continues to address these limitations by working to identify patient characteristics that predict a positive response. Further studies are needed to determine the optimal cell dosage, timing for administration, and whether repeat infusions could provide additional benefits. The goal is to refine the treatment into a more predictable and accessible therapeutic option.