Kurtzberg and Team Move Forward With Cerebral Palsy Cord Blood Study
In March, 2010, the Robertson Foundation awarded Duke University $10.2 million to develop a state-of-the-art Translational Cell Therapy Center (CT2), a shared resource facility advancing cell-based therapy research—partly in support of the translational work of Drs. Joanne Kurtzberg, Gordon Worley, Ricky Goldstein, and Mohamad Mikati. As a result of this grant, Dr. Kurtzberg created a team of researchers, including Drs. Gordon Worley, Ricky Goldstein, Alan Song, Jessica Sun, Kate Gustafson, and Mohamad Mikati who launched a study to evaluate whether, in children with cerebral palsy (CP), a single intravenous infusion of a child's own umbilical cord stem cells will cure or at least lessen the severity of the disease.
For decades, Kurtzberg, Director of Duke’s Pediatric Bone Marrow and Transplant program and Director of the Carolinas Cord Blood Bank, and her team have been investigating the therapeutic use of umbilical cord blood stem cells, specifically to treat (and sometimes reverse and even cure) some genetic disorders in children. Now, in a major effort to illustrate improvement in functional status of children with CP (as determined by measures of cognitive, language, motor, or functioning capabilities), Dr. Kurtzberg, Dr. Jessica Sun, the study’s sub-investigator, and the research team are able to move forward with the first placebo-controlled, randomized, crossover clinical trial of its kind.
“We are very excited to have initiated this very important study,” says Dr. Kurtzberg, “which will help us learn whether infusions of a child's own cord blood can lessen the symptoms of cerebral palsy. While there are many anecdotes suggesting that cord blood helps children with CP, it is essential to prove in a randomized trial, whether this is true. If this study shows that cord blood is beneficial, it will have a huge impact on the practices of cord blood collection and banking at birth.”
The team hopes that as a result of their work, more parents, when their child is born, will bank their child’s cord blood and that public cord blood banks will hold a child’s blood long enough to treat the child (if needed), before it is released into the inventory for public use. Kurtzberg also points out, that ultimately the goal of this trial is as much about finding a treatment for CP as it is about discovering what cord blood can do (e.g., reduce inflammation in the brain, produce new hormones to repair damaged brain cells, evolve into new brain cells to replace the damaged ones).
So far, Dr. Kurtzberg has been pleased with the study’s progress, and is especially excited about its public reception. “All children in this study will receive an infusion of their own cord blood,” she explains. “Some will receive the infusion at the start of the study and others will receive their cord blood one year later. Knowing this, parents have been very willing to allow their children to participate in the study to learn whether cord blood is beneficial.”
The team recruited its first participant in July, and since then has enrolled 31 more (as of 02/29/2012), steadily closing in on the recruiting goal of 120 subjects. The first infusion was performed in September, 2010, and the team is on track to complete its final infusion in July, 2012. The study is slated for completion July, 2013.
Recruiting efforts remain underway of children, ages 12 months to 6 years, diagnosed with spastic cerebral palsy, and whose cord blood cells were banked at birth. To learn more, contact June Allison, RN, at 919-668-1100, firstname.lastname@example.org. ClinicalTrials.gov Identifier NCT01147653.