| Trial registry | Patient Ages | Therapy Type | Address | Principal Investigator |
| NCT01072370 | 1 to 12 | Autologous | Georgia Regents University, USA | James E Carroll |
| NCT02866331 | 2 to 10 | Autologous | Hanyang University Hospital, South Korea | Lee Young-Ho |
| NCT03130816 | 10 months to 20 years | Allogeneic | CHA Bundang Medical Center, South Korea | Kim MinYoung |
| NCT03327467 | Up to 18 years | Allogeneic and/or Autologous | Duke University
|
Joanne Kurtzberg |
| NCT03352310 | Up to 48hrs | Autologous | The Chinese University of Hong Kong, China | Simon Lam |
| NCT03473301 | 24 to 60 months | Allogeneic | Duke University
|
Joanne Kurtzberg |
Cerebral Palsy (CP) is a condition affecting young children that causes lifelong disabilities and currently treatments are supportive, focusing on managing sequelae with physical therapies, medications and surgery. However, there are no therapies to address the underlying brain injury.4,5
Umbilical cord blood stem cells have been shown to improve motor function and brain connectivity in experimental systems via paracrine signalling.4
In a randomized, double-blind, placebo-controlled, phase two clinical trial, 63 children (between ages 1-6) with varied types and severities of spastic cerebral palsy, received a single intravenous dose of between 10-50 million stem cells per kilogram of their body weight to ascertain whether autologous cord blood (ACB) could improve function (Clinicaltrials.gov, NCT01147653).4,6
The improvements were greater than those typically observed for children of similar age and condition, and exceeded the gains made by children who received a lower dose of cells or placebo.6
In the clinical trial, Kurtzberg and colleagues tested doses from 10 million cells per kilogram of body weight up to 50 million cells per kilogram, based on the amount and quality of the cord blood each child had in storage. Among the tools used to evaluate the children’s progress were MRI to measure brain connectivity and the Gross Motor Function Measure (GMFM-66), a standardised analysis of each child’s ability to crawl, roll, kneel and complete other movements based on age and development.5
Children with cerebral palsy are expected to gain motor function as they grow and develop and therefore the GMFM-66 does attempt to account for this expected growth based on age and severity of the cerebral palsy and most children did improve when retested a year after receiving the infusion. However the improvements for the children who received does of at least 25 million cells per kilogram of body weight progressed beyond their expected increases when they were tested a year after infusion.5
Therefore results of the study suggest that appropriately dosed ACB infusion improves brain connectivity and gross motor function in young children with CP.4
Joanne Kurtzberg is now conducting a sibling allogeneic clinical trial at Duke University- there are 90 children between ages 2-5 years.
The researchers are hopeful that cord blood and cell therapy may have a role in treating children with cerebral palsy and brain injury and from the results of these studies, are encouraged to continue this promising research.6
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