Safety of sibling cord blood cell infusion for children with cerebral palsy

Kylie Crompton, Iona Novak, Michael Fahey, Nadia Badawi, Katherine J. Lee, Francoise Mechinaud-Heloury, Priya Edwards, Paul Colditz, Trisha Soosay Raj, Janet Hough, Xiaofang Wang, Simon Paget, Kuang-Chih Hsiao, Peter Anderson, Dinah Reddihough

Research output: Contribution to journalArticleResearchpeer-review

4 Citations (Scopus)

Abstract

Cerebral palsy (CP) is a nonprogressive neurological disorder and the most common physical disability of childhood. There is no cure for CP, but stem cells have the potential to improve brain injury and hence function. This phase 1 clinical trial investigated the safety of the intravenous infusion of full-matched sibling cord blood cells for children with CP aged 1 to 16 years. Preliminary efficacy outcomes were also investigated. Twelve participants received 12/12 HLA-matched sibling cord blood cell infusions. One treatable serious adverse reaction to cryoprotectant was observed, and no adverse reactions occurred beyond 24 h after infusion. Gross motor function measure (GMFM-66) scores did not improve compared with baseline beyond what could be expected from developmental levels, and participants had varied changes in the Quality of Upper Extremity Skills Test (QUEST) and Vineland Adaptive Behavior Scales (VABS-II) scores. In conclusion, matched sibling cord blood cell infusion for children with CP is relatively safe when conducted in an appropriate facility. Australian and New Zealand Clinical Trials Registry (ACTRN12616000403437) and Clinicaltrials.gov (NCT03087110).

Original languageEnglish
Pages (from-to)931-939
Number of pages9
JournalCytotherapy
Volume24
Issue number9
DOIs
Publication statusPublished - Sept 2022

Keywords

  • cerebral palsy
  • clinical trial
  • cord blood
  • safety
  • stem cell

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