The following is general information introducing research findings. Results reported in studies do not mean the same outcome for any individual, and most of these studies are small or early-stage.
Summary — The neurodevelopmental field covers conditions related to how the brain develops, such as autism spectrum disorder, developmental disorders, cerebral palsy and intellectual disability. The most systematic stem cell clinical research in this field has been carried out by a team at Duke University in the United States: in cerebral palsy, a randomised study reported greater improvement in gross motor function in children who received at least a certain dose of autologous cord blood; in autism spectrum disorder, phase I confirmed safety and phase II reported exploratory signals. Below we summarise the field and its research results and guide you to the condition pages.
Conditions in this field
Neurodevelopmental conditions appear from infancy and affect several areas of development, including movement, language, cognition and social skills. This field includes four topics:
- Autism spectrum disorder — characterised by difficulties with social communication and restricted, repetitive behaviours
- Developmental disorders — a broad category of conditions in which development is delayed or differs from peers
- Cerebral palsy — disorders of movement and posture caused by damage to the developing brain
- Intellectual disability — limitations in intellectual and adaptive functioning beginning in the developmental period
These conditions differ in cause and in the changes they produce in the brain, and children with the same diagnosis vary greatly. When reading research, it is therefore important to check first which diagnoses and ages were studied.
Cells studied and approaches
The most studied cells in this field are the child's own cord blood banked at birth, donor cord blood, and umbilical cord tissue-derived mesenchymal stromal cells grown from donated cord tissue, mostly given intravenously once or a few times. Researchers hypothesised that these cells might modulate immune and inflammatory responses, secrete neuroprotective factors and support the re-formation of brain connections. This differs from the idea that infused cells become brain cells, and the mechanism is still being studied.
Key research results by condition
- Cerebral palsy — in Duke's randomised, placebo-controlled study of autologous cord blood (2017, 63 children aged 1–6), children who received at least a certain dose per body weight showed greater improvement in gross motor function scores (GMFM-66), and increases in imaging measures of brain connectivity were reported. A 2016 systematic review observed a small improvement in motor function in stem cell groups while rating the certainty of evidence as low.
- Autism spectrum disorder — the same team's phase I study (2017, 25 children) confirmed the safety and feasibility of autologous cord blood. A randomised, placebo-controlled phase II study of 180 children aged 2–7 (2020) found no difference in socialisation scores overall, with exploratory differences in some communication and EEG measures reported in children without intellectual disability. A phase I study of cord tissue-derived mesenchymal stromal cells (2020, 12 children) was also carried out.
- Developmental disorders and intellectual disability — large clinical trials designed for the whole category or for intellectual disability itself are still rare; relevant information comes mainly from research on the two diagnoses above.
Background for understanding research in this field
Children develop naturally over time, and rehabilitation and education have large effects, so observations of 'improvement' without a control group cannot establish the effect of cell administration. This is why the cerebral palsy study used a placebo-controlled crossover design and the autism research moved from phase I to a randomised phase II. When reading results, check whether there was a control group, whether assessors were blinded to group allocation, and what the primary outcome was and whether it was met.
On safety, intravenous administration was generally well tolerated in clinical trials, but the U.S. Food and Drug Administration (FDA) and the International Society for Stem Cell Research (ISSCR) urge caution about interventions offered outside research without quality control or ethical review.
Condition pages
The topics below give details for autism spectrum disorder, developmental disorders, cerebral palsy and intellectual disability: overview, standard care, research approaches, reported results and how outcomes are measured.
Frequently asked questions
Q. Is there an approved stem cell treatment for autism spectrum disorder or cerebral palsy?
There is currently no stem cell product approved for autism spectrum disorder or cerebral palsy in Korea, Japan or the United States, among others. Most research so far is at the phase 1 or 2 clinical trial stage.
Q. Which studies in this field carry the most evidence?
Among studies with randomised, placebo-controlled designs, the autologous cord blood study in cerebral palsy (2017) and the cord blood phase II study in autism spectrum disorder (2020) are the leading examples. Both were carried out by a team at Duke University in the United States, and their results are covered in detail on this page and the condition pages.
Q. Can children without banked cord blood take part in research?
Autologous cord blood studies included only children with banked cord blood, but later research has also studied donor cord blood and cord tissue-derived mesenchymal stem cells. Eligibility for ongoing trials can be checked on public registries such as ClinicalTrials.gov.
Q. Does stem cell research replace existing support?
No. Studies tested whether there was added benefit while children continued behavioural and developmental interventions and rehabilitation.
The above summarises results reported in published papers and public-agency materials. University and institute names are given to identify research sources, not to introduce or recommend any medical institution. Reported results do not mean the same effect for everyone, and the suitability of any treatment is decided by the medical team.
References
- Dawson G, Sun JM, Davlantis KS, et al. Autologous cord blood infusions are safe and feasible in young children with autism spectrum disorder: results of a single-center phase I open-label trial. Stem Cells Transl Med. 2017;6(5):1332-1339. View on PubMed
- Dawson G, Sun JM, Baker J, et al. A phase II randomized clinical trial of the safety and efficacy of intravenous umbilical cord blood infusion for treatment of children with autism spectrum disorder. J Pediatr. 2020;222:164-173. View on PubMed
- Sun JM, Dawson G, Franz L, et al. Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. Stem Cells Transl Med. 2020;9(10):1137-1146. View on PubMed
- Sun JM, Song AW, Case LE, et al. Effect of autologous cord blood infusion on motor function and brain connectivity in young children with cerebral palsy: a randomized, placebo-controlled trial. Stem Cells Transl Med. 2017;6(12):2071-2078. View on PubMed
- Novak I, Walker K, Hunt RW, et al. Concise review: stem cell interventions for people with cerebral palsy: systematic review with meta-analysis. Stem Cells Transl Med. 2016;5(8):1014-1025. View on PubMed
- International Society for Stem Cell Research (ISSCR). Guidelines for Stem Cell Research and Clinical Translation (2021). https://www.isscr.org/guidelines
- U.S. Food and Drug Administration. FDA Warns About Stem Cell Therapies (Consumer Update). https://www.fda.gov/consumers/consumer-updates/fda-warns-about-stem-cell-therapies
