TY - JOUR
T1 - Acceptability of neural stem cell therapy for cerebral palsy
T2 - survey of the Australian cerebral palsy community
AU - Smith, Madeleine J.
AU - Finch-Edmondson, Megan
AU - Miller, Suzanne L.
AU - Webb, Annabel
AU - Fahey, Michael C.
AU - Jenkin, Graham
AU - Paton, Madison Claire Badawy
AU - McDonald, Courtney A.
N1 - Funding Information:
We thank the following Australian CP Registers for supporting the recruitment of participants for this study: Australian Capital Territory and New South Wales Cerebral Palsy Registers, Victorian Cerebral Palsy Register and Queensland Cerebral Palsy Register. Specifically, we thank all data custodians, with special mention to Sue Reid, Gina O’Grady, Sarah McIntyre, Isabelle Balde, Megan Auld, Jacinta Quartermaine. We thank Alishia Ballintine and the Cerebral Palsy Alliance Stem Cell Reference Group for their invaluable input during the design of this survey. We thank Claire Galea for initial statistical input into study design. We also thank Simone Gibson for input into survey development.
Funding Information:
This project was not directly supported by any funding. MS was supported by an Australian Government Research Training Program Scholarship. SM was supported by a National Health and Medical Research Council Senior Research Fellowship (APP1136216). This work was supported by Victorian Government’s Operational Infrastructure Support Program.
Publisher Copyright:
© 2023, The Author(s).
PY - 2023/12
Y1 - 2023/12
N2 - Background: Neural stem cells (NSCs) have the potential to engraft and replace damaged brain tissue, repairing the damaged neonatal brain that causes cerebral palsy (CP). There are procedures that could increase engraftment of NSCs and may be critical for efficacy, but hold notable risks. Before clinical trials progress, it is important to engage with the CP community to understand their opinions. The aim of this study was to determine the acceptability of NSC therapy for CP in the CP community. Methods: Australian residents with CP and parents/carers of those with CP completed a questionnaire to determine their willingness to use NSCs from three sources (fetal, embryonic and induced pluripotent stem cells) and their willingness to undergo accompanying procedures (neurosurgery, immunosuppression) that carry potential risks. To further explore their views, participants also answered free text questions about their ethical concerns regarding the source of NSCs and their perceptions of meaningful outcomes following NSC treatment. Results: In total, 232 responses were analyzed. Participants were willing to use NSCs from all three cell sources and were willing to undergo NSC therapy despite the need for neurosurgery and immunosuppression. Participants identified a range of outcome domains considered important following NSC treatment including gross motor function, quality of life, independence and cognitive function. Conclusions: Hypothetical NSC therapy was acceptable to the Australian CP community. This study has identified important findings from the CP community which can be used to inform future NSC research, including the design of clinical trials which may help to increase recruitment, compliance and participant satisfaction.
AB - Background: Neural stem cells (NSCs) have the potential to engraft and replace damaged brain tissue, repairing the damaged neonatal brain that causes cerebral palsy (CP). There are procedures that could increase engraftment of NSCs and may be critical for efficacy, but hold notable risks. Before clinical trials progress, it is important to engage with the CP community to understand their opinions. The aim of this study was to determine the acceptability of NSC therapy for CP in the CP community. Methods: Australian residents with CP and parents/carers of those with CP completed a questionnaire to determine their willingness to use NSCs from three sources (fetal, embryonic and induced pluripotent stem cells) and their willingness to undergo accompanying procedures (neurosurgery, immunosuppression) that carry potential risks. To further explore their views, participants also answered free text questions about their ethical concerns regarding the source of NSCs and their perceptions of meaningful outcomes following NSC treatment. Results: In total, 232 responses were analyzed. Participants were willing to use NSCs from all three cell sources and were willing to undergo NSC therapy despite the need for neurosurgery and immunosuppression. Participants identified a range of outcome domains considered important following NSC treatment including gross motor function, quality of life, independence and cognitive function. Conclusions: Hypothetical NSC therapy was acceptable to the Australian CP community. This study has identified important findings from the CP community which can be used to inform future NSC research, including the design of clinical trials which may help to increase recruitment, compliance and participant satisfaction.
KW - Brain injury
KW - Cell therapy
KW - Cerebral palsy
KW - Neurosurgery
KW - Pediatric neurology
KW - Stakeholder engagement
UR - http://www.scopus.com/inward/record.url?scp=85147423558&partnerID=8YFLogxK
U2 - 10.1186/s13287-023-03246-2
DO - 10.1186/s13287-023-03246-2
M3 - Article
C2 - 36737828
AN - SCOPUS:85147423558
SN - 1757-6512
VL - 14
JO - Stem Cell Research & Therapy
JF - Stem Cell Research & Therapy
IS - 1
M1 - 18
ER -