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Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder.
Stem Cells Transl Med. 2020 Jun 12 [Online ahead of print]SC

Abstract

Ongoing neuroinflammation may contribute to symptoms of autism spectrum disorder (ASD) in at least a portion of affected individuals. Mesenchymal stromal cells (MSCs) have demonstrated the capacity to modulate neuroinflammation, but safety and feasibility of MSC administration in children with ASD have not been well established. In this open-label, phase I study, 12 children with ASD between 4 and 9 years of age were treated with intravenous (IV) infusions of human cord tissue mesenchymal stromal cells (hCT-MSCs), a third-party MSC product manufactured from unrelated donor umbilical cord tissue. Children received one, two, or three doses of 2 × 106 cells per kilogram at 2-month intervals. Clinical and laboratory evaluations were performed in person at baseline and 6 months and remotely at 12 months after the final infusion. Aside from agitation during the IV placement and infusion in some participants, hCT-MSCs were well tolerated. Five participants developed new class I anti-human leukocyte antigen (HLA) antibodies, associated with a specific lot of hCT-MSCs or with a partial HLA match between donor and recipient. These antibodies were clinically silent and not associated with any clinical manifestations to date. Six of 12 participants demonstrated improvement in at least two ASD-specific measures. Manufacturing and administration of hCT-MSCs appear to be safe and feasible in young children with ASD. Efficacy will be evaluated in a subsequent phase II randomized, placebo-controlled clinical trial.

Authors+Show Affiliations

The Marcus Center for Cellular Cures, Duke University, Durham, North Carolina, USA.Duke Center for Autism and Brain Development, Duke University, Durham, North Carolina, USA.Duke Center for Autism and Brain Development, Duke University, Durham, North Carolina, USA.Duke Center for Autism and Brain Development, Duke University, Durham, North Carolina, USA.The Marcus Center for Cellular Cures, Duke University, Durham, North Carolina, USA.The Marcus Center for Cellular Cures, Duke University, Durham, North Carolina, USA.Stem Cell Transplant Laboratory, Duke University, Durham, North Carolina, USA.The Marcus Center for Cellular Cures, Duke University, Durham, North Carolina, USA.The Marcus Center for Cellular Cures, Duke University, Durham, North Carolina, USA.The Marcus Center for Cellular Cures, Duke University, Durham, North Carolina, USA.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32531111

Citation

Sun, Jessica M., et al. "Infusion of Human Umbilical Cord Tissue Mesenchymal Stromal Cells in Children With Autism Spectrum Disorder." Stem Cells Translational Medicine, 2020.
Sun JM, Dawson G, Franz L, et al. Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. Stem Cells Transl Med. 2020.
Sun, J. M., Dawson, G., Franz, L., Howard, J., McLaughlin, C., Kistler, B., Waters-Pick, B., Meadows, N., Troy, J., & Kurtzberg, J. (2020). Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. Stem Cells Translational Medicine. https://doi.org/10.1002/sctm.19-0434
Sun JM, et al. Infusion of Human Umbilical Cord Tissue Mesenchymal Stromal Cells in Children With Autism Spectrum Disorder. Stem Cells Transl Med. 2020 Jun 12; PubMed PMID: 32531111.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. AU - Sun,Jessica M, AU - Dawson,Geraldine, AU - Franz,Lauren, AU - Howard,Jill, AU - McLaughlin,Colleen, AU - Kistler,Bethany, AU - Waters-Pick,Barbara, AU - Meadows,Norin, AU - Troy,Jesse, AU - Kurtzberg,Joanne, Y1 - 2020/06/12/ PY - 2019/12/14/received PY - 2020/04/01/revised PY - 2020/04/08/accepted PY - 2020/6/13/entrez PY - 2020/6/13/pubmed PY - 2020/6/13/medline KW - autism KW - cellular therapy KW - clinical trials KW - cord tissue KW - mesenchymal stromal cells JF - Stem cells translational medicine JO - Stem Cells Transl Med N2 - Ongoing neuroinflammation may contribute to symptoms of autism spectrum disorder (ASD) in at least a portion of affected individuals. Mesenchymal stromal cells (MSCs) have demonstrated the capacity to modulate neuroinflammation, but safety and feasibility of MSC administration in children with ASD have not been well established. In this open-label, phase I study, 12 children with ASD between 4 and 9 years of age were treated with intravenous (IV) infusions of human cord tissue mesenchymal stromal cells (hCT-MSCs), a third-party MSC product manufactured from unrelated donor umbilical cord tissue. Children received one, two, or three doses of 2 × 106 cells per kilogram at 2-month intervals. Clinical and laboratory evaluations were performed in person at baseline and 6 months and remotely at 12 months after the final infusion. Aside from agitation during the IV placement and infusion in some participants, hCT-MSCs were well tolerated. Five participants developed new class I anti-human leukocyte antigen (HLA) antibodies, associated with a specific lot of hCT-MSCs or with a partial HLA match between donor and recipient. These antibodies were clinically silent and not associated with any clinical manifestations to date. Six of 12 participants demonstrated improvement in at least two ASD-specific measures. Manufacturing and administration of hCT-MSCs appear to be safe and feasible in young children with ASD. Efficacy will be evaluated in a subsequent phase II randomized, placebo-controlled clinical trial. SN - 2157-6580 UR - https://www.unboundmedicine.com/medline/citation/32531111/Infusion_of_human_umbilical_cord_tissue_mesenchymal_stromal_cells_in_children_with_autism_spectrum_disorder L2 - https://doi.org/10.1002/sctm.19-0434 DB - PRIME DP - Unbound Medicine ER -
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