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Sibling umbilical cord blood infusion is safe in young children with cerebral palsy
Jessica M Sun1, Laura E Case2, Mohamad A Mikati3
1Marcus Center for Cellular Cures, Duke University Medical Center, Durham, North Carolina, USA.
Insights
Allogeneic umbilical cord blood (CB) infusions show promise for improving motor function in children with cerebral palsy (CP). This study found the treatment to be safe and potentially beneficial for young CP patients.
Area of Science:
- Regenerative Medicine
- Pediatric Neurology
- Hematology
Background:
- Umbilical cord blood (CB) infusions show potential for improving motor function in children with cerebral palsy (CP).
- Limited access to autologous CB necessitates exploring allogeneic CB for broader therapeutic application.
- Previous studies suggest CB therapy benefits young CP patients.
Purpose of the Study:
- To evaluate the safety and potential efficacy of allogeneic human leukocyte antigen (HLA)-matched or partially matched sibling CB infusions in young children with moderate to severe spastic CP.
- To assess adverse events and immunological responses following CB infusion.
- To measure changes in gross motor function post-infusion.
Main Methods:
- A Phase I, open-label study involving 15 children (aged 1-6 years) with moderate to severe spastic CP.
- Treatment involved a single intravenous infusion of allogeneic sibling CB at a dose of ≥2.5 × 10^7 cells/kg.
- Participants were monitored for adverse events and assessed for motor function changes over a 2-year period.
Main Results:
- No adverse events were directly related to the CB infusions, including infusion reactions or antibody formation.
- Donor cells were undetectable in peripheral blood at 6 months post-infusion.
- Participants showed a mean increase of 4.7 points on the Gross Motor Function Measure-66 and 1 point on the Peabody Gross Motor Quotient at 6 months.
Conclusions:
- Allogeneic CB infusion, when appropriately dosed and HLA-matched or partially matched, is well-tolerated in young children with CP.
- The therapy demonstrates potential benefits for improving gross motor function in this population.
- This approach broadens access to potentially life-changing CB therapy for children with cerebral palsy.
Abstract:
Preclinical and early phase clinical studies suggest that an appropriately dosed umbilical cord blood (CB) infusion has the potential to help improve motor function in young children with cerebral palsy (CP). As many children with CP do not have their own CB available, use of allogeneic cells would extend access to this potentially beneficial therapy to more children. In this phase I, open-label study, 15 children, aged 1 to 6 years, with moderate to severe spastic CP were treated with a single intravenous infusion of allogeneic human leukocyte antigen (HLA) matched or partially matched sibling CB with a cell dose of ≥2.5 × 107 cells/kg based on the pre-cryopreservation count (median infused cell dose, 3.3 × 107 ; range, 1.8-5.2 × 107 ). There were a total of 49 adverse events (AEs) over a 2-year time period, but there were no AEs related to the CB infusions. Specifically, there were no acute infusion reactions and no antibody formation against platelets, red blood cells, or donor-specific HLA antigens. Donor cells were not detected in peripheral blood 6 months later. Six months after infusion, participants were assessed for response and experienced a mean ± SD increase of 4.7 ± 2.5 points on the Gross Motor Function Measure-66 and 1 ± 2.9 points on the Peabody Gross Motor Quotient. Appropriately dosed, allogeneic partially or fully HLA-matched sibling CB infusion is well tolerated and potentially beneficial in young children with CP.
