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

doi:10.1016/j.jcyt.2022.01.003

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

Cytotherapy. 2022 Sep;24(9):931-939. doi: 10.1016/j.jcyt.2022.01.003. Epub 2022 Feb 19.

Authors

Affiliations

¹ Murdoch Children's Research Institute, Parkville VIC, Australia; The Royal Children's Hospital, Parkville VIC, Australia; The University of Melbourne, Parkville VIC, Australia. Electronic address: kylie.crompton@mcri.edu.au.
² Cerebral Palsy Alliance Research Institute, University of Sydney, Camperdown NSW, Australia.
³ Monash Children's Hospital, Clayton VIC, Australia; Monash University, Clayton VIC, Australia.
⁴ Cerebral Palsy Alliance Research Institute, University of Sydney, Camperdown NSW, Australia; The Children's Hospital at Westmead, Westmead NSW, Australia.
⁵ Murdoch Children's Research Institute, Parkville VIC, Australia; The University of Melbourne, Parkville VIC, Australia.
⁶ The Royal Children's Hospital, Parkville VIC, Australia.
⁷ Queensland Children's Hospital, South Brisbane QLD, Australia; The University of Queensland, Herston QLD, Australia.
⁸ The University of Queensland, Herston QLD, Australia; Royal Brisbane and Women's Hospitals, Herston QLD, Australia.
⁹ The Royal Children's Hospital, Parkville VIC, Australia; Queensland Children's Hospital, South Brisbane QLD, Australia.
¹⁰ Monash Children's Hospital, Clayton VIC, Australia.
¹¹ Murdoch Children's Research Institute, Parkville VIC, Australia.
¹² The Children's Hospital at Westmead, Westmead NSW, Australia.
¹³ Murdoch Children's Research Institute, Parkville VIC, Australia; Starship Children's Hospital, Auckland, New Zealand; University of Auckland, Auckland, New Zealand.
¹⁴ Murdoch Children's Research Institute, Parkville VIC, Australia; Monash University, Clayton VIC, Australia.
¹⁵ Murdoch Children's Research Institute, Parkville VIC, Australia; The Royal Children's Hospital, Parkville VIC, Australia; The University of Melbourne, Parkville VIC, Australia.

PMID: 35193825
DOI: 10.1016/j.jcyt.2022.01.003

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: cerebral palsy; clinical trial; cord blood; safety; stem cell.

Publication types

Clinical Trial, Phase I
Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Australia
Blood Cells
Cerebral Palsy* / therapy
Child
Child, Preschool
Cord Blood Stem Cell Transplantation* / adverse effects
Fetal Blood
Humans
Infant
Siblings

Associated data

ClinicalTrials.gov/NCT03087110
ANZCTR/ACTRN12616000403437