The research team was
led by Dr Joanne Kurtzberg, director of Duke's Pediatric Blood and Marrow
Transplant Program and the Robertson Clinical and Translational Therapy
Program. Dr Kurtzberg has been a pioneer in testing the therapeutic potential
of umbilical cord blood. Previously conducted research has indicated that it is
safe for children with cerebral palsy to receive an infusion of their own umbilical
cord blood. In the current study, the research team found the approximate
dosage of umbilical cord blood cells to lessen the symptoms of cerebral palsy.
Tools to evaluate the
children's progress were MRI to measure brain connectivity and the Gross Motor
Function Measure (GMFM-66), to analyze the child's ability to crawl, roll,
kneel, and complete other movements based on age and development.
‘An infusion of cells from umbilical cord blood improved motor function and brain connectivity in children with cerebral palsy.’
Based on the amount and
quality of the cord blood each child had in storage, the research team tested
doses from 10 million cells per kilogram of body weight up to 50 million cells
One intravenous dose of
at least 25 million stem cells per kilogram of the child's body weight improves
motor function a year later.
The improvements in motor skills and brain
connectivity were greater than those typically observed for children of similar
age and condition. The GMFM-66 accounts the expected growth based on age and
the severity of the cerebral palsy. Most of the study participants improved
when retested on the GMFM-66 a year after receiving an infusion, even those who
received a placebo.
Children who received
doses of cord blood of at least 25 million cells per kilogram of body weight
progressed in motor skills beyond their expected increases when they were
tested a year after 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. We
still have a lot to learn about this therapy so that it can be optimized and
accessible to more children with cerebral palsy," said Dr. Kurtzberg.
"Now that we have
identified a dosing threshold, we are planning additional studies testing the
benefits of multiple doses of cells, as well as the use of donor cells for
patients whose own cord blood was not banked."
Children with cerebral palsy
gain motor function as they grow and develop and receive occupational and
physical therapies. The improvements are different for each child and could be
subtle, but sometimes even a seemingly small difference is significant.
"For example, a child's ability to turn their hand from facing down to
facing up can change their ability to hold or grasp something, which can make a
big difference in their everyday life," said Dr. Jessica Sun, a pediatric
hematologist-oncologist at Duke and lead author of the paper.
The limitation of the
study is that participants from well-resourced families were also receiving
frequent physical and occupational therapy and those advantages could have
influenced the results.
"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," said Dr Sun.
The findings of the
trial are published in Stem Cells
Cerebral palsy is a
motor disorder affecting young children that cause lifelong disabilities.
Cerebral palsy is a result of in-utero or perinatal brain
injuries such as hemorrhage, stroke or hypoxic insult. Cerebral palsy affects
two to three per 1,000 live births. Children with cerebral palsy have varying
degrees of functional impairments in motor skills and self-mobility. Currently
available treatment options optimize function and quality of life. However,
there are no curative therapies.
- Jessica M. Sun, Allen W. Song, Laura E. Case, Mohamad A. Mikati, Kathryn E. Gustafson, Ryan Simmons, Ricki Goldstein, Jodi Petry, Colleen McLaughlin, Barbara Waters-Pick, Lyon W. Chen, Stephen Wease, Beth Blackwell, Gordon Worley, Jesse Troy, Joanne Kurtzberg. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. STEM CELLS Translational Medicine, 2017; DOI: 10.1002/sctm.17-0102