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Brain Science Advances 2015, 1(2): 114-124 https://doi.org/10.18679/CN11-6030_R.2015.015
Policy and Regulation | Open Access | Issue | Published: 01 December 2015

Regulations in the United States for cell transplantation clinical trials in neurological diseases

Show Author's Information He Zhu1,§,  Yuanqing Tan1,2,§,  Qi Gu1,3,  Weifang Han1,2,  Zhongwen Li1,  Jason S. Meyer4( ),  Baoyang Hu1( )
State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China
Graduate School of Chinese Academy of Sciences, Beijing 100049, China
ARC Centre of Excellence for Electromaterials Science (ACES), Intelligent Polymer Research Institute, AIIM Facility, Innovation Campus, University of Wollongong, NSW 2522, Australia
Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, Indiana, USA

§ These authors contributed equally to this work.

Keywords:
Parkinson’s disease, transplantation, differentiation, fetal neural stem cells, dopamine neuron
Cite this article:
Zhu H, Tan Y, Gu Q, et al. Regulations in the United States for cell transplantation clinical trials in neurological diseases. Brain Science Advances, 2015, 1(2): 114-124. https://doi.org/10.18679/CN11-6030_R.2015.015
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Abstract Full text About this article
Objective:

This study aimed to use a systematic approach to evaluate the current utilization, safety, and effectiveness of cell therapies for neurological diseases in human. And review the present regulations, considering United States (US) as a representative country, for cell transplantation in neurological disease and discuss the challenges facing the field of neurology in the coming decades.

Methods:

A detailed search was performed in systematic literature reviews of cellular-based therapies in neurological diseases, using PubMed, web of science, and clinical trials. Regulations of cell therapy products used for clinical trials were searched from the Food and Drug Administration (FDA) and the National Institutes of Health (NIH).

Results:

Seven most common types of cell therapies for neurological diseases have been reported to be relatively safe with varying degrees of neurological recovery. And a series of regulations in US for cellular therapy was summarized including preclinical evaluations, sourcing material, stem cell manufacturing and characterization, cell therapy product, and clinical trials.

Conclusions:

Stem cell-based therapy holds great promise for a cure of such diseases and will value a growing population of patients. However, regulatory permitting activity of the US in the sphere of stem cells, technologies of regenerative medicine and substitutive cell therapy are selective, theoretical and does not fit the existing norm and rules. Compiled well-defined regulations to guide the application of stem cell products for clinical trials should be formulated.


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Abstract
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About this article

Regulations in the United States for cell transplantation clinical trials in neurological diseases

Show Author's information He Zhu1,§, Yuanqing Tan1,2,§, Qi Gu1,3, Weifang Han1,2, Zhongwen Li1, Jason S. Meyer4( ), Baoyang Hu1( )
State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China
Graduate School of Chinese Academy of Sciences, Beijing 100049, China
ARC Centre of Excellence for Electromaterials Science (ACES), Intelligent Polymer Research Institute, AIIM Facility, Innovation Campus, University of Wollongong, NSW 2522, Australia
Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, Indiana, USA

§ These authors contributed equally to this work.

Abstract

Objective:

This study aimed to use a systematic approach to evaluate the current utilization, safety, and effectiveness of cell therapies for neurological diseases in human. And review the present regulations, considering United States (US) as a representative country, for cell transplantation in neurological disease and discuss the challenges facing the field of neurology in the coming decades.

Methods:

A detailed search was performed in systematic literature reviews of cellular-based therapies in neurological diseases, using PubMed, web of science, and clinical trials. Regulations of cell therapy products used for clinical trials were searched from the Food and Drug Administration (FDA) and the National Institutes of Health (NIH).

Results:

Seven most common types of cell therapies for neurological diseases have been reported to be relatively safe with varying degrees of neurological recovery. And a series of regulations in US for cellular therapy was summarized including preclinical evaluations, sourcing material, stem cell manufacturing and characterization, cell therapy product, and clinical trials.

Conclusions:

Stem cell-based therapy holds great promise for a cure of such diseases and will value a growing population of patients. However, regulatory permitting activity of the US in the sphere of stem cells, technologies of regenerative medicine and substitutive cell therapy are selective, theoretical and does not fit the existing norm and rules. Compiled well-defined regulations to guide the application of stem cell products for clinical trials should be formulated.

Keywords: Parkinson’s disease, transplantation, differentiation, fetal neural stem cells, dopamine neuron

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Publication history
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Publication history

Received: 09 October 2015
Revised: 27 November 2015
Accepted: 30 November 2015
Published: 01 December 2015
Issue date: December 2015

Copyright

© The authors 2015.

Acknowledgements

This work was supported by the National Basic Research Program (973 Program) of China (No. 2012CBA01307), and the National Natural Science Fund funded by the National Natural Science Foundation of China (No. 31171430).

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