Regulations in the United States for cell transplantation clinical trials in neurological diseases
He Zhu1, Yuanqing Tan1,2, Qi Gu1,3, Weifang Han1,2, Zhongwen Li1, Jason S. Meyer4, Baoyang Hu1
1 State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China;
2 Graduate School of Chinese Academy of Sciences, Beijing 100049, China;
3 ARC Centre of Excellence for Electromaterials Science(ACES), Intelligent Polymer Research Institute, AIIM Facility, Innovation Campus, University of Wollongong, NSW 2522, Australia;
4 Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, Indiana, USA
Regulations in the United States for cell transplantation clinical trials in neurological diseases
He Zhu1, Yuanqing Tan1,2, Qi Gu1,3, Weifang Han1,2, Zhongwen Li1, Jason S. Meyer4, Baoyang Hu1
1 State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China;
2 Graduate School of Chinese Academy of Sciences, Beijing 100049, China;
3 ARC Centre of Excellence for Electromaterials Science(ACES), Intelligent Polymer Research Institute, AIIM Facility, Innovation Campus, University of Wollongong, NSW 2522, Australia;
4 Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, Indiana, USA
摘要 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 cellularbased 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.
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 cellularbased 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.
通讯作者:
Baoyang Hu, E-mail:byhu@ioz.ac.cn;Jason S. Meyer, E-mail:meyerjas@iupui.edu
E-mail: byhu@ioz.ac.cn;meyerjas@iupui.edu
引用本文:
He Zhu, Yuanqing Tan, Qi Gu, Weifang Han, Zhongwen Li, Jason S. Meyer, Baoyang Hu. Regulations in the United States for cell transplantation clinical trials in neurological diseases[J]. 临床转化神经科学, 2015, 1(2): 114-124.
He Zhu, Yuanqing Tan, Qi Gu, Weifang Han, Zhongwen Li, Jason S. Meyer, Baoyang Hu. Regulations in the United States for cell transplantation clinical trials in neurological diseases. Translational Neuroscience and Clinics, 2015, 1(2): 114-124.
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