The key to improving motor function in individuals with cerebral palsy may lie in that person's own umbilical cord.
A study from Duke University Medical Center published last month found that children with spastic cerebral palsy showed improvements in brain connectivity and motor function after receiving infusions of their own umbilical cord blood.
Cerebral palsy is a disorder of motor function, associated with poor control of extremities and with symptoms including involuntary movement, poor balance and difficulty speaking and walking. It is caused by brain damage or abnormal brain development before, during or after birth, and is usually detected in older infants and toddlers.
“Patients can live decades with appropriate support, but they’re unfortunately unable to care for themselves,” said Joanne Kurtzberg, Jerome S. Harris professor of pediatrics and senior author of the study. “Right now, treatment is mostly in the category of physical and occupational therapy.”
Although more invasive procedures—like Botox injections—exist, there is no cure for cerebral palsy to date. These findings suggest, however, that stem cell-rich umbilical cord blood has immense therapeutic potential, signaling the body to repair some of the brain damage that caused the disease in the first place, the researchers hypothesize.
The study enlisted 63 patients between the ages of one and six, each of whom had umbilical cord blood already conserved in a blood bank and whose cerebral palsy ranged from mild to moderately severe.
Patients with the most severe form of cerebral palsy—Gross Motor Classification System Level V—had to be excluded because of the safety risk of travel, Kurtzberg said. The researchers also excluded patients whose cerebral palsy was genetic, since receiving infusions of their own cells would presumably have no effect.
After confirming the patients’ eligibility and setting benchmarks for progress with an MRI scan and several rounds of evaluation by neurologists, physical therapists and other medical specialists, half of the patients received umbilical cord blood infusions and half received a placebo.
Kurtzberg noted that the study did not control for the dose of cord blood administered to each child. Rather, each child was administered whatever dose was available for them. The minimum dose was 10 million cells per kilogram of the child’s body weight at the time of infusion.
One year later, the patients returned for reassessment. The researchers found that children who had received a dose of at least 20 million cells per kilogram of body weight showed the most improvement in brain connectivity and motor function. Thereafter, the placebo and experimental groups switched treatments—the children who had received the placebo treatment were given cord blood infusions, and the children who had received cord blood were given the placebo.
After another year, the scientists reassessed the patients, all of whom had by then received a dose of cord blood at some point in the two years prior. These reassessments further confirmed their conclusion for the most effective dose—children whose doses exceeded 20 million cells per kilogram showed significantly greater improvement than those whose doses were less.
Jessica Sun, assistant professor of pediatrics and lead author of the paper, said in a Duke Health release that the eligibility requirements of the study narrowed its scope, unwittingly targeting wealthier families who could likely afford frequent physical and occupational therapy for their child. This, along with the restrictions in the severity of the disease and age of the patients, limits the applicability of the study’s results, Kurtzberg said.
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Nevertheless, Kurtzberg noted that the findings are exciting. She described future plans to study the effectiveness of donated umbilical cord blood in cerebral palsy patients, noting that many do not have their own supply to use for treatment. She cited unpublished data showing that siblings’ cord blood was also safe and effective to use as treatment for cerebral palsy, and further emphasized her goal of expanding this potential therapy to all children with the disease.
“We are hopeful that cord blood and cell therapy may have a role in treating children with cerebral palsy and brain injury and are encouraged to continue this promising research,” Sun said in the release.