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

K Crompton; I Novak; M Fahey; N Badawi; KJ Lee; F Mechinaud-Heloury; P Edwards; P Colditz; T Soosay Raj; J Hough; X Wang; S Paget; KC Hsiao; P Anderson; D Reddihough

Journal article

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

K Crompton, I Novak, M Fahey, N Badawi, KJ Lee, F Mechinaud-Heloury, P Edwards, P Colditz, T Soosay Raj, J Hough, X Wang, S Paget, KC Hsiao, P Anderson, D Reddihough

Cytotherapy | ELSEVIER SCI LTD | Published : 2022

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 ..

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University of Melbourne Researchers

Kylie Crompton Author

Kate Lee Author

Xiaofang Wang Author

Dinah Reddihough Author

Grants

Funding Acknowledgements

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. The authors would like to acknowledge the late Prof. Mark Kirk- land, OAM, of Deakin University and Cell Care Australia Pty Ltd, Victoria, Australia, for his contribution to study design and leadership. We also acknowledge Prof. Euan Wallace for his involvement in study design; members of the independent Data Safety Monitoring Com- mittee, Ross Pinkerton, Richard Mitchell, Adam Scheinberg, Catherine Marraffa and Rory Wolfe; and Jacqueline Fleming, Ngaire Elwood, Helen Savoia, Laine Hosking, Megan Kentish, Megan Thorley, Laura Gascoigne-Pees, Dayna Jackson, Jane Valentine and Gavin Hutana for their valuable assistance.This work was generously supported by Cell Care Australia Pty Ltd and the Cerebral Palsy Alliance Research Foundation.