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).
Keywords
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Article info
Publication history
Published online: February 19, 2022
Accepted:
January 21,
2022
Received:
November 2,
2021
Identification
Copyright
© 2022 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.
