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Journal of Neurorestoratology 2017, 5(1): 93-101 https://doi.org/10.2147/JN.S124367
Original Research | Open Access | Issue | Published: 18 April 2017

Combined transplantation of neural precursor cells and olfactory ensheathing cells for the treatment of X-linked adrenoleukodystrophy in children

Show Author's Information Hui Yang1,*,  Yu Zhang1,*,  Zhaoyan Wang1,  Wei Lu1,  Fang Liu1,  Xin Yu2,  Xiaoyan Zheng1,  Yinxiang Yang1,  Zuo Luan1( ),  Suqing Qu1( )
Department of Pediatrics,
Department of Neurological Surgery, Navy General Hospital, Beijing, People’s Republic of China

* These authors contributed equally to this work.

Keywords:
olfactory ensheathing cells, neural precursor cells, X-linked adrenoleukodystrophy, combined transplantation
Cite this article:
Yang H, Zhang Y, Wang Z, et al. Combined transplantation of neural precursor cells and olfactory ensheathing cells for the treatment of X-linked adrenoleukodystrophy in children. Journal of Neurorestoratology, 2017, 5(1): 93-101. https://doi.org/10.2147/JN.S124367
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Abstract Full text About this article

Hematopoietic stem cell transplantation is only suitable for early-stage adrenoleukodystrophy (ALD). In this study, we observed the therapeutic efficacy of combined transplantation of neural precursor cells (NPCs) and olfactory ensheathing cells (OECs) on late-stage X-linked ALD in nine children who were admitted in our hospital between June 2009 and January 2014. Related patient information included onset time 3 months to 1 year, magnetic resonance imaging (MRI) score 11.02±0.90, and neurologic function score 2-3. All patients received combined transplantation of NPCs and OECs by injection around the lateral angle of the frontotemporal-occipital lesion under MRI guidance. It was found that the visual function, sleep, and communication obstacles were improved significantly without evidence of disease progression in six (66.7%) of the nine patients within 1 month after transplantation. In two of the six patients, the lesions became significantly smaller than before, although their MRI scores remained unchanged significantly. In addition, cell therapy did not induce any irreversible adverse event during the study period, indicating that combined transplantation of NPCs and OECs was safe and reliable, and could improve the clinical manifestations of ALD in children within a short time. Although this cell therapy was not able to halt the progression of the disease 1-3 months after transplantation, it could still be used as an early treatment and provide patients with more opportunities for hematopoietic stem cell transplantation, which is the only effective long-term treatment for X-linked ALD at present. The preliminary results from this study suggest that a comprehensive prevention strategy of serial and combined transplantation may improve some functions for ALD patients in the short-term, however, long-term effects need further study.


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About this article

Combined transplantation of neural precursor cells and olfactory ensheathing cells for the treatment of X-linked adrenoleukodystrophy in children

Show Author's information Hui Yang1,*, Yu Zhang1,*, Zhaoyan Wang1, Wei Lu1, Fang Liu1, Xin Yu2, Xiaoyan Zheng1, Yinxiang Yang1, Zuo Luan1( ), Suqing Qu1( )
Department of Pediatrics,
Department of Neurological Surgery, Navy General Hospital, Beijing, People’s Republic of China

* These authors contributed equally to this work.

Abstract

Hematopoietic stem cell transplantation is only suitable for early-stage adrenoleukodystrophy (ALD). In this study, we observed the therapeutic efficacy of combined transplantation of neural precursor cells (NPCs) and olfactory ensheathing cells (OECs) on late-stage X-linked ALD in nine children who were admitted in our hospital between June 2009 and January 2014. Related patient information included onset time 3 months to 1 year, magnetic resonance imaging (MRI) score 11.02±0.90, and neurologic function score 2-3. All patients received combined transplantation of NPCs and OECs by injection around the lateral angle of the frontotemporal-occipital lesion under MRI guidance. It was found that the visual function, sleep, and communication obstacles were improved significantly without evidence of disease progression in six (66.7%) of the nine patients within 1 month after transplantation. In two of the six patients, the lesions became significantly smaller than before, although their MRI scores remained unchanged significantly. In addition, cell therapy did not induce any irreversible adverse event during the study period, indicating that combined transplantation of NPCs and OECs was safe and reliable, and could improve the clinical manifestations of ALD in children within a short time. Although this cell therapy was not able to halt the progression of the disease 1-3 months after transplantation, it could still be used as an early treatment and provide patients with more opportunities for hematopoietic stem cell transplantation, which is the only effective long-term treatment for X-linked ALD at present. The preliminary results from this study suggest that a comprehensive prevention strategy of serial and combined transplantation may improve some functions for ALD patients in the short-term, however, long-term effects need further study.

Keywords: olfactory ensheathing cells, neural precursor cells, X-linked adrenoleukodystrophy, combined transplantation

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Published: 18 April 2017
Issue date: December 2017

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