PMID- 34085782 OWN - NLM STAT- MEDLINE DCOM- 20220406 LR - 20220531 IS - 2157-6580 (Electronic) IS - 2157-6564 (Print) IS - 2157-6564 (Linking) VI - 10 IP - 9 DP - 2021 Sep TI - Sibling umbilical cord blood infusion is safe in young children with cerebral palsy. PG - 1258-1265 LID - 10.1002/sctm.20-0470 [doi] AB - 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 x 10(7) cells/kg based on the pre-cryopreservation count (median infused cell dose, 3.3 x 10(7) ; range, 1.8-5.2 x 10(7) ). 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. CI - (c) 2021 The Authors. STEM CELLS TRANSLATIONAL MEDICINE published by Wiley Periodicals LLC on behalf of AlphaMed Press. FAU - Sun, Jessica M AU - Sun JM AUID- ORCID: 0000-0001-8085-1013 AD - Marcus Center for Cellular Cures, Duke University Medical Center, Durham, North Carolina, USA. FAU - Case, Laura E AU - Case LE AD - Department of Physical and Occupational Therapy, Duke University, Durham, North Carolina, USA. FAU - Mikati, Mohamad A AU - Mikati MA AD - Division of Pediatric Neurology, Duke University, Durham, North Carolina, USA. FAU - M Jasien, Joan AU - M Jasien J AD - Division of Pediatric Neurology, Duke University, Durham, North Carolina, USA. FAU - McLaughlin, Colleen AU - McLaughlin C AD - Marcus Center for Cellular Cures, Duke University Medical Center, Durham, North Carolina, USA. FAU - Waters-Pick, Barbara AU - Waters-Pick B AD - Stem Cell Transplant Laboratory, Duke University, Durham, North Carolina, USA. FAU - Worley, Gordon AU - Worley G AD - Division of Pediatric Neurology, Duke University, Durham, North Carolina, USA. FAU - Troy, Jesse AU - Troy J AUID- ORCID: 0000-0001-5410-8146 AD - Marcus Center for Cellular Cures, Duke University Medical Center, Durham, North Carolina, USA. AD - Department of Biostatistics and Bioinformatics, Duke University, Durham, North Carolina, USA. FAU - Kurtzberg, Joanne AU - Kurtzberg J AD - Marcus Center for Cellular Cures, Duke University Medical Center, Durham, North Carolina, USA. LA - eng PT - Clinical Trial, Phase I PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20210604 PL - England TA - Stem Cells Transl Med JT - Stem cells translational medicine JID - 101578022 RN - 0 (HLA Antigens) SB - IM MH - *Cerebral Palsy/therapy MH - Child MH - Child, Preschool MH - Fetal Blood MH - *Graft vs Host Disease MH - HLA Antigens MH - Humans MH - Infant MH - Siblings PMC - PMC8380440 OTO - NOTNLM OT - cellular therapy OT - clinical trials OT - cord blood OT - human cord blood OT - nervous system OT - umbilical cord blood COIS- The authors report grant funding from the Julian Robertson Foundation (principal investigator [PI]: J.K.), the Marcus Foundation (PI: J.K.), and the Dana Foundation (PI: J.M.S.). J.M.S. declared intellectual property rights/patent ownership with Sinocell Technologies, Inc. (licensed data), advisory role with Medical Device Business Services, Biosense Webster (spouse), honoraria from Medtronic (spouse), and research funding from the Marcus Foundation. L.E.C. declared leadership position/employment at Duke University; advisory role with Genzyme Corporation of Sanofi; honoraria from Genzyme Corporation of Sanofi, Sarepta, Muscular Dystrophy Association; and research funding from the Robertson Foundation, the Marcus Foundation, Genzyme Corporation of Sanofi, Amicus, Biogen, Ultragenyx, NS Pharma, Reveragen, Pfizer, CINRG (Cooperative International Neuromuscular Research Group), TRiNDS (Therapeutic Research in Neuromuscular Disorders Solutions), AveXis, and AskBio. J.T. reported intellectual property rights/patent ownership from 62/470431, 16/493754, JP Appln No. 2019-549 537, KR Appln No. 10-2019-7029 841, U.S. Appln No. 16/493754; advisory role AegisCN, LLC, Gamida Cell, Synthetic Biologics, The EMMES Corporation, and Cohortias International; honoraria from Gamida Cell, Synthetic Biologics, and The EMMES Corporation; and research funding from Seattle Genetics and Bristol Myers Squibb. J.K. declared intellectual property rights with pending licensing agreement with CryoCell and serves as the Director of the Carolinas Cord Blood Bank, a U.S. Food and Drug Administration-licensed public cord blood bank located at Duke University and Medical Director of CryoCell, a hybrid cord blood bank. The other authors declared no potential conflicts of interest. EDAT- 2021/06/05 06:00 MHDA- 2022/04/07 06:00 PMCR- 2021/06/04 CRDT- 2021/06/04 09:10 PHST- 2021/03/03 00:00 [revised] PHST- 2020/10/22 00:00 [received] PHST- 2021/03/22 00:00 [accepted] PHST- 2021/06/05 06:00 [pubmed] PHST- 2022/04/07 06:00 [medline] PHST- 2021/06/04 09:10 [entrez] PHST- 2021/06/04 00:00 [pmc-release] AID - SCT312940 [pii] AID - 10.1002/sctm.20-0470 [doi] PST - ppublish SO - Stem Cells Transl Med. 2021 Sep;10(9):1258-1265. doi: 10.1002/sctm.20-0470. Epub 2021 Jun 4.