Acceptability of neural stem cell therapy for cerebral palsy: survey of the Australian cerebral palsy community

Abstract

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.

A bibliometric analysis of cerebral palsy from 2003 to 2022

“…However, the lack of effective models has hindered more in-depth research ( 55 ). Although stem cell therapy is not currently widely used in children with CP, the parents of these children remain optimistic about the potential improvement of stem cell therapy, given the limited treatment options available for CP ( 56 ). Accordingly, stem cell treatments are worthy of exploration and may lead to breakthroughs in the future.…”

Genetically engineered neural stem cells (NSCs) therapy for neurological diseases; state‐of‐the‐art

“…The NSCs can be procured from brain tissue, reprogrammed from somatic cells, or differentiated from pluripotent stem cells, such as embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) 13,14 . They are also found in highly‐specific microenvironments, comprising cell and extracellular components, including ependymal cells, vasculature, extracellular matrix proteins, soluble factors, astrocytes, microglia, and pericytes 15 . While clinical trials in patients are still ongoing, preclinical research on the use of NSCs to treat neurological disorders has shown encouraging results 16–18 .…”

“…extracellular matrix proteins, soluble factors, astrocytes, microglia, and pericytes. 15 While clinical trials in patients are still ongoing, preclinical research on the use of NSCs to treat neurological disorders has shown encouraging results. [16][17][18] The studies conducted in vitro or using animal models have demonstrated that NSCs can be stimulated and activated to differentiate into neurons, thus replacing lost neurons, and also enhance the local microenvironment, promote the growth of blood vessels, adjust inflammatory responses, and restore the homeostasis of the brain.…”

Providing holistic care to children with cerebral palsy treated with transnasal neural stem cell transplantation

“…The remarkable migratory ability of neural stem cells (NSCs) to integrate into damaged areas of the brain provides strong preclinical evidence for the treatment of children with perinatal brain injury (6). It brings hope for neuroregeneration in patients with CP (7,8). In recent years, many scholars have proven the safety and early efficacy of NSCs clinical trials and have focused on further clinical translation (9,10).…”