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Treatment with Sibling Umbilical Cord Trialed in Young Children with Cerebral Palsy

Review of “Sibling umbilical cord blood infusion is safe in young children with cerebral palsy” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Recent research led by Jessica M. Sun (Duke University Medical Center, Durham, North Carolina, USA) reported on the results of a phase II randomized, placebo-controlled trial of autologous cord blood infusion as a means to treat young children with cerebral palsy [1, 2], a common motor disorder of childhood associated with lifelong problems. This recent trial suggested that children receiving higher doses of cells exhibited a greater degree of improvement in gross motor function [3] (See the STEM CELLS Translational Medicine study here!).

In the hope of expanding cord blood therapy to additional patients, the Sun team recently evaluated the potential of related allogeneic cord blood cells by conducting a phase I, open-label study of a high-dose, single intravenous infusion of human leukocyte antigen-matched or partially matched sibling cord blood cells in fifteen children (ages 1 to 6 years old) with moderate to severe spastic cerebral palsy to evaluate safety and efficacy [4].

Here are all the details of this trial, as described by Sun et al.

  • Over a two-year period following treatment, the authors noted 49 adverse events
    • However, no adverse effect was linked to cord blood infusions, and the study did not observe acute infusion reactions or antibody formation against platelets, red blood cells, or donor-specific human leukocyte antigen
  • At six months, patients demonstrated significant gains in both gross and fine motor function, although donor cells were no longer detected
  • Together, that data suggests that allogeneic partially or fully matched sibling cord blood cells are well tolerated and may induce improvements in gross motor function

The authors next hope to next confirm the observed gains in motor function in children with cerebral palsy receiving this treatment approach in larger randomized, blinded, placebo-controlled trials and further explore the potential efficacy of using cord blood from unrelated donors.

For more on the therapeutic potential of cord blood and new treatments for cerebral palsy, stay tuned to the Stem Cells Portal!

References

  1. Bax M, Goldstein M, Rosenbaum P, et al., Proposed definition and classification of cerebral palsy, April 2005. Developmental Medicine & Child Neurology 2005;47:571-576.
  2. Christensen D, Van Naarden Braun K, Doernberg NS, et al., Prevalence of cerebral palsy, co-occurring autism spectrum disorders, and motor functioning – Autism and Developmental Disabilities Monitoring Network, USA, 2008. Developmental Medicine & Child Neurology 2014;56:59-65.
  3. Sun JM, Song AW, Case LE, 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. Sun JM, Case LE, Mikati MA, et al., Sibling umbilical cord blood infusion is safe in young children with cerebral palsy. STEM CELLS Translational Medicine 2021;10:1258-1265.