- May 03, 2018
In a phase 2 trial involving 63 children, Joanne Kurtzberg and her team gave patients an infusion of their own cord blood that had been banked at birth. The doses ranged from 10 million stem cells per kilogram of body weight to 50 million per kilogram.
A year later, they found that the medium-sized dose, 25 million stem cells per kilogram of body weight, was the most effective one. Children who received this dose showed the biggest improvements in motor function, and fared better than children who had been given a lower dose or placebo.
The gains were measured using the Gross Motor Function Measure (GMFM-66), a standardized analysis of a child’s ability to complete movements — such as crawling and rolling — based on age and development. The study appears in the journal Stem Cells Translational Medicine.
“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 Kurtzberg, Director of Duke’s Pediatric Blood and Marrow Transplant Program and the Robertson Clinical and Translational Therapy Program. “We still have a lot to learn about this therapy so that it can be optimized and accessible to more children with cerebral palsy,” she added.
Cord blood is FDA-approved for the treatment of blood-related disorders, such as sickle cell disease and some blood cancers, and studies are underway in other disorders. Sutter Health is investigating the use of a child’s own cord blood for the treatment of autism, for example. In addition to stem cells, it contains natural killer (NK) cells, which can be engineered to seek and destroy leukemia and lymphoma, a team from the M.D. Anderson Cancer Center found.
There is no cure for cerebral palsy, but symptoms may be treated with drugs or physical therapy, according to the National Institute of Neurological Disorders and Stroke.
The Duke findings on cord blood are promising; however, there are some caveats. Participation in the study was limited to children who had cord blood in storage. Additionally, the researchers said, some of the improvement could be linked to the frequent physical and occupational therapy many of the patients received.
“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,” Kurtzberg said. The team is recruiting patients for a phase 1 trial, in which children aged one to six will be treated with cord blood taken from their siblings.
REFERENCE: Fierce Biotech; 02 NOV 2017; Amirah Al Idrus