In Brief
Cerebral palsy (CP) is a group of lifelong neurological conditions resulting from injury to the developing brain, affecting movement, posture, and overall quality of life.¹ While conventional treatments focus on managing symptoms, emerging research into cellular therapies, such as umbilical cord blood (UCB) and cord tissue mesenchymal stromal cells (MSCs), offers new hope. These therapies leverage the body's own repair mechanisms, induce remyelination, and provide anti-inflammatory and immunomodulatory actions to address the underlying brain injury.¹ Two recent meta-analyses provide preliminary evidence that these treatments are not only safe but also effective in improving gross motor function for individuals with CP, signaling a potential future paradigm shift in care.¹,²
About Cerebral Palsy
Cerebral palsy is the most common motor disability in childhood, typically resulting from an injury to the brain before, during, or shortly after birth.1 The condition affects muscle control, coordination, and balance, and can be associated with other health issues, including intellectual disability, seizures, and vision or hearing problems. While symptoms and severity vary widely, the focus of treatment has traditionally been on rehabilitation and symptom management.1
Recent scientific advancements are exploring the potential of cellular therapies to promote neural repair.1,2 By modulating the inflammatory environment of the injured brain, therapies using MSCs and UCB aim to create conditions that support the body's natural healing processes.1 Cord blood monocytes also have been shown to promote remyelination of damaged neurons in the brain. These approaches hold the potential to improve motor outcomes and enhance the quality of life for those affected by CP.1
Overall Clinical Significance Statement
Together, the following two large-scale analyses provide promising evidence that cellular therapies using cord-tissue-derived MSCs or umbilical cord blood may be able to help improve motor function in young children with CP.
More importantly, this research moves the field closer to confirming whether these therapies work and considering how they can be optimized. The UCB study provides clear, actionable data on the importance of higher cell doses and identifies the patients most likely to benefit—younger children with milder CP. This creates a strong evidence-based foundation for designing
targeted, pivotal Phase 3 trials that are needed to achieve regulatory approval and commercialization of these therapies for children with CP.
Study 1: Mesenchymal Stromal Cells (MSCs) for Cerebral Palsy
A recent comprehensive scoping review and meta-analysis published in Cells evaluated the existing clinical evidence for MSCs as a treatment for cerebral palsy. The study synthesized data from 30 published reports and 10 registered trials, encompassing 1,292 individuals with CP who received MSCs
KEY FINDINGS:
Treatment Profile:
A significant majority of participants (75%) received MSCs derived from umbilical cord tissue. Most individuals (72%) were administered more than one dose, typically through intrathecal (40%) or intravenous (38%) routes. Notably, nearly a third of these treatments (31%) were provided under compassionate or Expanded Access programs, highlighting the demand for new therapeutic options.
Safety:
The analysis confirmed that MSC treatment is safe, with no serious adverse events reported across the included studies. Side effects were generally mild and transient, such as fever or infusion-related reactions.
Efficacy:
The meta-analysis of controlled trials revealed that MSCs led to small, but statistically significant improvements in gross motor function. Given the variability of dosing, routes of administration, types of MSCs, and heterogeneity of patients treated, the authors strongly recommended confirmation of their findings in standardized phase 3 clinical trials.
Study 2: Umbilical Cord Blood (UCB) for Cerebral Palsy
In a landmark individual participant data meta-analysis (IPDMA) published in Pediatrics, researchers analyzed data from 11 studies to provide a robust assessment of UCB therapy for CP.² The analysis included records from 447 unique participants, comparing those who received UCB with a control group.
KEY FINDINGS:
Treatment Profile:
The majority of participants (84%) received allogeneic UCB, primarily from unrelated donors. Of participants receiving allogeneic UCB, 26% had no mismatches across the assessed human leukocyte antigen (HLA) markers. Treatment was typically administered intravenously.
Safety:
The study confirmed the safety of UCB infusions, finding that the rate of serious adverse events was similar between the UCB-treated group (16.0%) and the control group (12.9%).
Efficacy:
UCB treatment resulted in small, but clinically meaningful and statistically significant improvements in GMFM scores at later time points:
- 6 months: 1.36-point improvement
- 12 months: 1.42-point improvement
- No significant effects were seen at 1 or 3 months, suggesting that the benefits of UCB
therapy emerge over time.
Dose-Response Relationship:
A key finding was the link between cell dose and efficacy. Higher doses of total nucleated cells (TNCs) were associated with greater improvements in motor function, particularly at
doses exceeding 50 × 10 TNCs/kg.
Identifying Best Responders:
The analysis identified specific subgroups that benefited most from UCB therapy. The greatest improvements were seen in:
- Younger children (approximately <5 years old)
- Those with milder forms of CP (GMFCS levels I-III)
These findings should guide directions for future research, suggesting that trials should focus on these responder populations and explore dose repsonse to maximize therapeutic benefit.
References: 1. Paton, M.C.B.; Griffin, A.R.; Blatch-Williams, R.; et al. Clinical Evidence of Mesenchymal Stromal Cells for Cerebral Palsy: Scoping Review with Meta-Analysis of Efficacy in Gross Motor Outcomes. Cells 2025, 14, 700. 2. Finch-Edmondson, M.; Paton, M.C.B.; Webb, A.; et al. Cord Blood Treatment for Children With Cerebral Palsy: Individual Participant Data Meta-Analysis. Pediatrics. 2025, 155(5):e2024068999. 3. Cerebral Palsy Alliance Research Foundation. (n.d.). Facts about cerebral palsy.https://cparf.org/what-is-cerebral-palsy/facts-about-cerebral-palsy Accessed 2025-02-03