Cerebral Palsy Stem Cell Research — 18 Clinical Studies in 1,087 Children and an Umbilical Cord MSC Case (2026)

Summary — Two papers published in 2026 show how far cerebral palsy stem cell research has come. A systematic review in Translational Pediatrics in June gathered 18 clinical studies involving 1,087 children with cerebral palsy and found that several trials reported better gross motor function with cord blood and umbilical cord-derived cells, while side effects were mostly mild and short-lived. In August, a Vinmec research team in Hanoi, Vietnam, described a child who developed cerebral palsy after cardiac arrest and whose GMFM-88 score rose from 54 to 174 after two doses of umbilical cord-derived mesenchymal stem cells.

What is cerebral palsy, and how is it managed today?

Cerebral palsy (脳性まひ in Japanese) results from injury to the developing brain before or shortly after birth and affects movement and posture for life. The injury itself does not worsen, but stiff muscles (spasticity), problems with balance and walking, and difficulties with speech and swallowing can appear together. It is regarded as the most common cause of physical disability in children.

Care today centers on rehabilitation such as physiotherapy, occupational therapy and speech therapy. When spasticity is severe, medicines, botulinum toxin injections or orthopedic surgery may be added. These approaches help a child make the most of existing abilities, but they cannot easily restore brain tissue that has already been injured. That is why regenerative medicine research using cells that calm inflammation and support nerve recovery continues in many countries. An overview of the field is on our cerebral palsy stem cell research page.

What 18 clinical studies show about cerebral palsy stem cell research

This systematic review, published online on June 26, 2026, was written by researchers from the Faculty of Medicine at Jazan University in Saudi Arabia and colleagues in Sudan. The team searched PubMed, Web of Science, the Cochrane Library and ScienceDirect for papers from 2010 to January 2025 and registered its plan in advance with the international registry PROSPERO. Eighteen studies were analyzed: 10 randomized controlled trials, 3 open-label trials and 5 non-randomized studies.

A total of 1,087 children took part, aged from 6 months to 17 years. By cell type, 73.5% (799 children) received umbilical cord-derived products such as cord blood, cord tissue or Wharton's jelly, and 20.3% (221) received bone marrow-derived products such as bone marrow mesenchymal stem cells or bone marrow mononuclear cells. Another 4.3% (47) received approaches based on granulocyte colony-stimulating factor (G-CSF). Intravenous infusion was the most common route (10 studies), followed by intrathecal injection into the spinal fluid through the lower back (6 studies).

The findings are encouraging. The Gross Motor Function Measure (GMFM) was the most widely used outcome. Umbilical cord-derived cells, especially cord blood alone or combined with erythropoietin (EPO), produced moderate improvements in motor ability, often exceeding the threshold for a clinically meaningful change. Several randomized trials reported statistically significant GMFM gains compared with control groups. Cord blood plus EPO showed the largest gains in both motor and cognitive outcomes, and some follow-up studies found that improvements were maintained or continued gradually over 1–2 years after treatment. Bone marrow-derived products also showed improvement, though the size varied between studies.

A case from Vietnam — motor function after two doses of umbilical cord MSCs

The case report, published in Frontiers in Medicine on August 28, 2026, was written by researchers at the Vinmec Research Institute of Stem Cell and Gene Technology (VinUniversity) and Vinmec international hospitals in Hanoi. The patient was a boy with type 2 autoimmune hepatitis who was diagnosed with cerebral palsy after sepsis and cardiac arrest at 12 months of age. At the first assessment he could roll, crawl and sit unsteadily but could not stand or walk on his own, and he spoke no words. His Gross Motor Function Classification System (GMFCS) level was IV and his GMFM-88 score was 54.

The team gave him two doses of mesenchymal stem cells taken from the umbilical cord of a healthy newborn and grown under serum-free conditions, each at 1 million cells per kilogram of body weight. The first dose was given intravenously at 23 months, and the second intrathecally 21 months later; rehabilitation and hepatitis medicine continued throughout. Twenty-one months after the first dose, he could sit without support and pull to stand holding furniture. His GMFM-88 rose to 125, his GMFCS improved to level III, and his upper limb score (QUEST) rose from 55.55 to 87.03. After the second dose, GMFM-88 reached 174 and QUEST 92.59; he took steps holding furniture, spoke in longer sentences and told simple stories. Muscle stiffness and twisting movements also lessened.

Liver function improved as well. Liver enzymes returned to the normal range about six months after the infusion and stayed stable, and the immunosuppressant azathioprine was stopped 10 months after the first dose. No infusion-related adverse events were recorded. The authors note that this is a single observation, that other factors such as rehabilitation may have contributed, and that larger controlled studies are the next step.

How were safety and research stage reported?

According to the review, almost all studies reported safety data, and cell administration was generally well tolerated. Common reactions were short-lived fever, headache, digestive symptoms, mild respiratory infections and back pain after intrathecal injection. Intravenous infusion mainly caused mild infusion reactions, while intrathecal injection had somewhat more procedure-related reactions such as headache or back pain. Worsening of seizures occurred in isolated cases, and one death was judged unrelated to treatment. No treatment-related tumors or transplant reactions were reported during follow-up. The research teams monitored the children before and after each dose to watch for these reactions.

In terms of research stage, stem cell therapy for cerebral palsy is still in clinical trials. Because cell types, doses and dosing schedules differed between studies, the review authors did not pool the results numerically and recommended multicenter trials that follow more children for longer using standardized methods. The trend that larger cell doses, and combined approaches such as cord blood with EPO, gave better results is an important clue for designing the next studies.

What research is under way in Japan?

In Japan, cell-based treatments are carried out after their plans are reviewed under the Act on the Safety of Regenerative Medicine. For cerebral palsy, Nagoya University is running a clinical trial giving stem cells from baby teeth to children in the chronic phase, which we covered in our article on baby tooth stem cells for cerebral palsy. Research aimed at preventing brain injury right after birth is described in our article on neonatal hypoxic-ischemic encephalopathy and cord blood.

Frequently asked questions

Which cells have been used most in cerebral palsy stem cell research?

Of the 1,087 children in the 2026 review, 73.5% received umbilical cord-derived cells such as cord blood, cord tissue or Wharton's jelly. Bone marrow mesenchymal stem cells and bone marrow mononuclear cells came next, and the largest motor and cognitive gains were reported with cord blood combined with EPO.

How old were the children in these studies?

Participants in the included studies ranged from 6 months to 17 years of age. A randomized trial at Duke University in the United States enrolled children aged 2–5, and a randomized double-blind trial in China enrolled children aged 2–12. Criteria differ by study, so age alone does not decide eligibility.

Does rehabilitation continue after the cells are given?

Yes. Most studies in the review combined cell therapy with physiotherapy, occupational therapy and speech therapy, and better results were reported more often when rehabilitation continued. In the Vietnamese case, the child also kept up rehabilitation throughout the study.

Summary

These two 2026 papers show where cerebral palsy stem cell research stands. Across 18 studies with 1,087 children, several randomized trials reported improved gross motor function, mainly with umbilical cord-derived cells, and side effects were mostly mild and short-lived. In one child's case, sitting, standing and speech improved markedly after two doses and steady rehabilitation. Research is now moving toward multicenter trials that follow more children for longer. Which approach suits a child, and what risks are involved, are judged by the treating medical team.

This content summarizes published scientific papers. Names of universities, research institutions and hospitals are given to identify research sources and do not introduce or recommend any medical institution. Suitability for treatment is judged by the treating medical team.

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