1. Safety of sibling cord blood cell infusion for children with cerebral palsy.
- Author
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Crompton K, Novak I, Fahey M, Badawi N, Lee KJ, Mechinaud-Heloury F, Edwards P, Colditz P, Soosay Raj T, Hough J, Wang X, Paget S, Hsiao KC, Anderson P, and Reddihough D
- Subjects
- Adolescent, Australia, Blood Cells, Child, Child, Preschool, Fetal Blood, Humans, Infant, Siblings, Cerebral Palsy therapy, Cord Blood Stem Cell Transplantation adverse effects
- 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)., Competing Interests: Declaration of Competing Interest Cell Care Australia is a private cord blood bank with a representative on the Trial Steering Committee. There is, therefore, a potential conflict of interest which has been declared to HREC and Steering Committee and is well recognized. No one affiliated with Cell Care Australia was involved in data analysis or interpretation., (Copyright © 2022 International Society for Cell & Gene Therapy. All rights reserved.)
- Published
- 2022
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