It's one of the clinical trials that we have been watching closely. In November 2016, we were able to give you a behind-the-scenes look at some of the big steps Duke University has seen in cerebral palsy, but Duke University has now officially released its findings from a phase II clinical trial on the safety and efficacy of treating cerebral palsy in children through an infusion of their own umbilical cord blood. The results show great promise as the study moves into a phase III clinical trial.
Duke University findings
Dr. Kurtzberg is the lead on Duke University's cerebral palsy trials
Dr. Joanne Kurtzberg and a team of researchers at Duke University Medical Center found through a double-blind, placebo-controlled phase II clinical trial that "umbilical cord blood improves whole brain connectivity and motor function in young children with cerebral palsy."
The study followed 63 children between the ages of one year and six years who had cerebral palsy because of brain damage incurred before or at birth. The researchers looked at the change in motor function at one year and two years post-infusion.
“We are encouraged by the results of this study, which shows that appropriately dosed infusions of cord blood cells can help lessen symptoms in children with cerebral palsy,” said senior author Joanne Kurtzberg, director of Duke’s Pediatric Blood and Marrow Transplant Program.
The participants were given a range of doses depending on how much cord blood they had stored, from 10 million cells to 50 million cells per approx. two pounds of body weight. The results show that children who received greater than 25 million cells per kilogram (2.2 lb.) of body weight exceeded the gains made by children with smaller doses or who received a placebo.
As it was a cross-over study, children who were part of the control group and who received the placebo the first year became part of the experiment group and were infused with cord blood the following year. This ensured all participants had the chance to undergo the treatment.
Researchers note that children with cerebral palsy will often make gains as they get older but say that children given high dosages in their study exceeded increases that could normally be expected because of age progression. (While called a "high dose" by researchers, it is equivalent to is the number of cells normally provided to stem cell transplant recipients fighting leukemia at Duke.)
In our "behind-the-scenes" coverage, Dr. Kutrzberg said improvements after the first year for high-dose infusions were 30% higher than would have been predicted for age and level of function.
The study used MRI to study progress in whole brain connectivity
Researchers at Duke University used the Peabody Developmental Motor Scales-2 to test for progress but put more weight on the findings from the GMFM-66 method, a 66-item measure designed to assess gross motor function in children with CP. In addition, MRI scans were used to analyze whole brain connectivity.
Cerebral palsy (CP) is the most prevalent motor disorder of childhood, affecting one in 323. It is caused by damage to or abnormal development of the brain, taking its toll on movement and posture but can also cause intellectual disability, seizures, and problems hearing, seeing, and speaking. Through intense and long-lasting physical therapy treatments, more than half of people with cerebral palsy are able to gain the ability to walk independently.
Other signs of hope
Chloe Levine is just one story of a young girl who saw a dramatic turnaround in her cerebral palsy following an infusion of her own cord blood stem cells.
The results of Duke University's study follow a similar study it is conducting on the treatment of autism using umbilical cord blood. The results from its clinical trial on autism also showed promising results, with CNN highlighting one young girl's "supercharged" progress.