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Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral
Jessica M Sun1, Laura E Case2, Colleen McLaughlin1
1Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC, USA.
Insights
Allogeneic umbilical cord blood (AlloCB) shows promise as a cerebral palsy (CP) cell therapy, with AlloCB treatment linked to greater motor function improvements after one year. Umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) were also safe but did not show significant motor function changes.
Area of Science:
- Regenerative Medicine
- Pediatric Neurology
- Cell Therapy
Background:
- Cerebral palsy (CP) is a group of movement disorders due to brain damage.
- Current treatments for CP focus on managing symptoms and improving function.
- Novel cell-based therapies offer potential for neuroprotection and functional recovery in CP.
Purpose of the Study:
- To evaluate the safety and efficacy of allogeneic umbilical cord blood (AlloCB) and human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with CP.
- To compare motor function outcomes between AlloCB, hCT-MSC, and a natural history control group.
Main Methods:
- Ninety-one children with CP were randomized into three groups: AlloCB, hCT-MSC, or natural history control.
- AlloCB group received a single infusion; hCT-MSC group received multiple infusions over 6 months.
- Motor function was assessed using the Gross Motor Function Measure-66 (GMFM-66) and Peabody Developmental Motor Scale.
Main Results:
- Infusions of AlloCB and hCT-MSC were well-tolerated with minimal adverse events.
- No significant motor function improvements were observed at 6 months post-treatment compared to controls.
- At 12 months, the AlloCB group showed significantly greater improvements in GMFM-66 scores compared to the natural history group.
Conclusions:
- High-dose AlloCB infusion is a safe and potentially effective cell therapy for children with CP.
- Further investigation in randomized, blinded, placebo-controlled trials is warranted to confirm AlloCB efficacy.
- While safe, hCT-MSC did not demonstrate significant motor function benefits in this study population.
Aim:
To evaluate safety and motor function after treatment with allogeneic umbilical cord blood (AlloCB) or umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with cerebral palsy (CP).
Method:
Ninety-one children (52 males, 39 females; median age 3 years 7 months [range 2-5 years]) with CP due to hypoxic-ischemic encephalopathy, stroke, or periventricular leukomalacia were randomized to three arms: (1) the AlloCB group received 10 × 107 AlloCB total nucleated cells (TNC) per kilogram at baseline (n = 31); (2) the hCT-MSC group received 2 × 106 hCT-MSC at baseline, 3 months, and 6 months (n = 28); (3) the natural history control group received 10 × 107 AlloCB TNC per kilogram at 12 months (n = 31). Motor function was assessed with the Gross Motor Function Measure-66 (GMFM-66) and Peabody Developmental Motor Scale, Second Edition.
Results:
Infusions (n = 143) were well tolerated, with eight infusion reactions (three in the AlloCB group, five in hCT-MSC) and no other safety concerns. At 12 months, the mean differences (95% confidence intervals [CI]) between actual and expected changes in GMFM-66 score were AlloCB 5.8 points (3.4-8.2), hCT-MSC 4.3 (2.2-6.4), and natural history 3.1 (1.4-5.0). In exploratory, post hoc analysis, the mean GMFM-66 score (95% CI) of the hCT-MSC group was 1.4 points higher than natural history (-1.1 to 4.0; p = 0.27), and the AlloCB group was 3.3 points higher than natural history (0.59-5.93; p = 0.02) after adjustment for baseline Gross Motor Function Classification System level, GMFM-66 score, and etiology.
Interpretation:
High-dose AlloCB is a potential cell therapy for CP and should be further tested in a randomized, blinded, placebo-controlled trial.
What This Paper Adds:
Unrelated donor allogeneic umbilical cord blood (AlloCB) and human umbilical cord tissue-derived mesenchymal stromal cell infusion is safe in young children with cerebral palsy. Significant changes in motor function were not observed 6 months after treatment. One year later, treatment with AlloCB was associated with greater increases in Gross Motor Function Measure-66 scores.
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