Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease.
Henry, Roland G
Lim, Daniel A
Huhn, Stephen L
Barkovich, A James
Stem cell reports
Elsevier (Cell Press)
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Gupta, N., Henry, R. G., Kang, S., Strober, J., Lim, D. A., Ryan, T., Perry, R., et al. (2019). Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease.. Stem cell reports, 13 (2), 254-261. https://doi.org/10.1016/j.stemcr.2019.07.002
Four boys with Pelizaeus-Merzbacher Disease (PMD), an X-linked leukodystrophy, underwent transplantation with human allogeneic central nervous system stem cells (HuCNSSC). Subsequently, all subjects were followed for an additional 4 years in this separate follow-up study to evaluate safety, neurologic function, MRI data and immunologic response. The neurosurgical procedure, immunosuppression and HuCNS-SC transplantation were well tolerated and all four subjects were alive at the conclusion of the study period. At year two, all subjects exhibited diffusion MRI changes at the implantation sites as well as in more distant brain regions. There were persistent, increased signal changes in the three patients who were studied up to year five. Two of four subjects developed donor-specific HLA alloantibodies, demonstrating that neural stem cells can elicit an immune response when injected into the CNS, and suggesting the importance of monitoring immunologic parameters and identifying markers of engraftment in future studies.
Brain, Humans, Pelizaeus-Merzbacher Disease, Isoantibodies, HLA Antigens, Magnetic Resonance Imaging, Treatment Outcome, Stem Cell Transplantation, Transplantation, Homologous, Severity of Illness Index, Follow-Up Studies, Child, Preschool, Infant, Male, Neural Stem Cells
RGH receives research funding from Sanofi-Genzyme, Roche/Genentech, Medday. Consultancy from Novartis, Medday, Roche, Sanofi-Genzyme, Abbvie. Educational programs from Sanofi-Genzyme, TEVA. This clinical study was supported by StemCells Inc.
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External DOI: https://doi.org/10.1016/j.stemcr.2019.07.002
This record's URL: https://www.repository.cam.ac.uk/handle/1810/293724
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