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Cord Blood Trial for Cerebral Palsy in Children Highlights Dosing Effect

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Review of “Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Evidence from animal studies suggests that transplanted umbilical cord blood cells can improve the motor function deficits associated with cerebral palsy via paracrine activation of endogenous repair mechanisms, rather than cellular integration or engraftment [1]. These findings encouraged researchers from the laboratory of Jessica Sun (Duke University Medical Center, Durham, NC, USA) to investigate the therapeutic potential of intravenous infusions of autologous cord blood (ACB) in human patients. 

Their previous study demonstrated safety [2], and their new STEM CELLS Translational Medicine study now reports on a randomized, placebo-controlled trial of intravenous ACB infusion in children with cerebral palsy [3].

So how did this new trial pan out?

  • 63 patients (children aged 1 to 6) tolerated single intravenous infusions of thawed ACB and placebo products
    • Motor function and magnetic resonance imaging brain connectivity studies employed at pre-infusion and following 1 and 2 years to assess outcomes 
  • While the study observed no differences in mean change of motor function scores, the authors did observe a dosing effect at one year
    • Patients receiving higher doses (≥2 × 107 cells per kg) demonstrated significantly greater increases in motor function scores and normalized brain connectivity, above those predicted by age and severity
    • Patients in placebo, low dose and, high dose groups all exhibited gains in motor function above that expected, although scores for the high dose patients exceeded placebo and low dose 
  • The authors highlighted several study limitations, including the small sample size and heterogeneity of patients
    • However, findings correlated well with two previous studies [4, 5]
    • Given the lack of available units of ACB, the authors plan to study the safety and efficacy of allogeneic cell sources

Encouraging results, but as always, only further studies will confirm the potential for autologous cord blood in the treatment of disorders such as cerebral palsy.

To keep up to date with any new studies and trials, stay tuned to the Stem Cells Portal.

References

  1. Drobyshevsky, A., et al., Human Umbilical Cord Blood Cells Ameliorate Motor Deficits in Rabbits in a Cerebral Palsy Model. Dev Neurosci 2015;37:349-62.
  2. Sun, J., et al., Differences in quality between privately and publicly banked umbilical cord blood units: a pilot study of autologous cord blood infusion in children with acquired neurologic disorders. Transfusion 2010;50:1980-7.
  3. Sun, J.M., et al., 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;6:2071-2078.
  4. Kang, M., et al., Involvement of Immune Responses in the Efficacy of Cord Blood Cell Therapy for Cerebral Palsy. Stem Cells Dev 2015;24:2259-68.
  5. Min, K., et al., Umbilical Cord Blood Therapy Potentiated with Erythropoietin for Children with Cerebral Palsy: A Double-blind, Randomized, Placebo-Controlled Trial. STEM CELLS 2013;31:581-591.