Umbilical Cord Blood Clinical Trials for Cerebral Palsy

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Cerebral Palsy (CP) is the most common cause of physical disability in children, and currently there is no cure1. The global prevalence of CP is between 0.2 and 0.3 percent with a marked difference between developed and developing countries. South African studies indicate a high prevalence rate for CP, between 1% and 8%.2

Umbilical cord blood (UCB) cell therapy for the treatment of children with Cerebral Palsy is currently being assessed in a number of clinical trials:

Summary of Cerebral Palsy Cord Blood Clinical Trials currently recruiting (Parents Guide to Cord Blood Foundation: https://parentsguidecordblood.org/en/trials/cerebral-palsy)3

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

 

References:

  1. McDonald CA, Fahey MC, Jenkin G, Miller SL. “Umbilical cord blood cells for treatment of cerebral palsy; timing and treatment options”, Paediatric Research, 2018 Jan; 83(1-2):333-344
  2. National Association for persons with Cerebral Palsy South Africa, “Cerebral Palsy in South Africa”: http://www.casualday.co.za/cerebral-palsy-in-south-africa/
  3. Parents’ Guide to cord blood foundation, “Recruiting Trials: Cord Blood”: https://parentsguidecordblood.org/en/trials/cerebral-palsy
  4. Sun J, Song, A, Case L, Mikati M, Gustafson K, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen L, Wease S, Blackwell B, Worley G, Troy J and Kurtzberg J. “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, published online 2017 Oct 28: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5702515/
  5. Sun J, Mikati M, Troy J, Gustafson K, Simmons R, Goldstein R, McLaughlin C, Water-Pick B, Case L, Worley G and Kurtzberg J, “Autologous Cord Blood Infusion for the Treatment of Brain Injury in Children with Cerebral Palsy”, Blood Journal, Volume 126, pages 925: http://www.bloodjournal.org/content/126/23/925/tab-article-info
  6. Duke Health, “Umbilical Cord Blood Improves Motor Skills in Some Children With Cerebral Palsy”, published October 30, 2017. https://corporate.dukehealth.org/news-listing/umbilical-cord-blood-improves-motor-skills-some-children-cerebral-palsy
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