Sibling umbilical cord blood infusion is safe in young children with cerebral palsy.
Jessica M SunLaura E CaseMohamad A MikatiJoan M JasienColleen McLaughlinBarbara Waters-PickGordon WorleyJesse D TroyJoanne KurtzbergPublished in: Stem cells translational medicine (2021)
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.
Keyphrases
- children with cerebral palsy
- umbilical cord
- induced apoptosis
- mesenchymal stem cells
- low dose
- peripheral blood
- stem cell transplantation
- cell cycle arrest
- bone marrow
- cell therapy
- open label
- young adults
- single cell
- red blood cell
- endothelial cells
- endoplasmic reticulum stress
- stem cells
- dendritic cells
- cell death
- signaling pathway
- randomized controlled trial
- high intensity
- cell proliferation
- oxidative stress
- drug induced
- pi k akt
- radiation therapy
- risk assessment
- human health
- hematopoietic stem cell
- acute respiratory distress syndrome
- cerebral palsy