Cell-based therapies

Finally, lentiviral vectors have been shown to transduce human embryonic stem cells. Therefore, this type of gene-therapy vector might also be used in stem cell-based therapies. 

Although pharmacology still relies heavily on small chemical substances, biologicals like recombinant proteins, cell-based therapies, and gene-therapy approaches have great potential for the treatment of broad spectrum of diseases. Therefore, the focus of modem pharmacology should shift towards these innovative therapies. 

Additional future innovations likely to impact upon pharmaceutical biotechnology include the development of alternative product production systems, alternative methods of delivery and the development of engineered cell-based therapies, particularly stem cell therapy. As mentioned previously, protein-based biotechnology products produced to date are produced in either microbial... 

Ozturk, S. and Hu, W.S. (eds). 2006. Cell Culture Technology for Pharmaceutical and Cell-based Therapies. Taylor and Francis. 

Zuk PA, Zhu M, Mizuno H, Huang J, Futrell JW, Katz AJ, Benhaim P, Lorenz HP and Hedrick MH (2001). Multilineage cells from human adipose tissue implications for cell based therapies. Tissue Eng 7 211-228. 

Losordo DW, Dimmeler S. Therapeutic angiogenesis and vasculogenesis for ischemic disease part II cell-based therapies. Circulation 2004 109 2692-2697. 

Doucet, C, Ernou, I., Zhang, Y. Z., et al. (2005), Platelet lysates promote mesenchymal stem cell expansion A safety substitute for animal serum in cell-based therapy applications. J. Cell. Physiol, 205(2), 228-236. 

Volume II, Embryonic and Adult Stem Cells, provides an analysis of various types of stem cells and their therapeutic potential. It concentrates particularly on embryonic and neural stem cells. This volume covers the broader potential of adult stem cells its biology, significance and potential for therapy. This volume also describes the potential of stem cells for autologous transplantation, the stem cell theory of carcinogenesis, particularly the existence of brain tumor stem cells, and the therapeutic potential of gene therapy for cell-based therapy. 

Stem Cell Engineering for Cell-Based Therapy... 

Stem cells are undifferentiated cells that have the ability to self-renew and differentiate into other cell types. Stem cells represent a promising model for cell-based therapy [8]. ESCs are self-renewing pluripotent cells derived from the inner cell mass (ICM) of the blastocyst. 

Reproduced with permission from the Institute of Physics Publishing Ltd. Taupin, P. Stem cell engineering for cell-based therapy. Journal of Neural Engineering (2007) 4(3) R59-63. Copyright 2007, the Institute of Physics Publishing Ltd. http //jne.iop.org. 

Despite these failures, the need still exists for alternative liver therapies. Several new techniques including cell transplantation, tissue-engineered constructs, and extra- and paracorporeal devices seek to relieve some of the demands placed on a compromised liver. Liver assist devices allow the liver to regenerate its function by removing some of the demands. Bridge-to-transplant devices seek to maintain patients until suitable donors are available. Some therapies seek to remove toxins from the blood, and they have a place in the treatment scheme, but due to the complex and multifunctional nature of the organ, some type of cell-based therapy is considered a more complete solution. 

In direct cell implantation, human cells are required but supplies are limited. Production of hepatocytes from pediatric patients has been reported and techniques for preservation are improving. yHfQ characterization of human hepatocytes will aid the development of improved cell-based therapies. The primary safety concern with the use of cell lines, especially implanted cells, is the transmission of toxic factors to the host. 

Melo LG, Pachori AS, Kong D, Gnecchi M. Gene and cell-based therapies for heart disease, FASEBJ2004 18 648-663,... 

Considerations in Design of Preclinical Safety Evaluation Programs to Support Human Cell-Based Therapies... 

Regulation of Cell-Based Therapies in the United States and Europe 753... 

TABLE 33.1 Selected milestones in the field of cell-based therapies... 

REGULATION OF CELL-BASED THERAPIES IN THE UNITED STATES AND EUROPE... 

PRINCIPLES OF PRECLINICAL SAFETY EVALUATION FOR STEM CELL BASED THERAPIES... 

Similar to protein-based biopharmaceuticals, the standard battery of genotox-icity studies is not considered to be relevant for cell-based therapies unless there is a specific cause for concern regarding the nature of any expressed products that would indicate a potential interaction directly with DNA or other chromosomal material [50,52], The conduct of genotoxicity studies for assessing the genotoxic potential of process related contaminants is also not considered to be appropriate [50],... 

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