Abstract
Scientific advances in transplantation biology, immunology, and molecular genetics have suggested exciting possibilities for novel cell-based treatments for a variety of malignant and non-malignant diseases. Many of these cellular therapies are already moving from the research bench to the clinic. The development, clinical evaluation, and dissemination of these therapies is largely dependent on the availability of reliable, clinically feasible methods for the procurement, ex vivo manipulation, storage, and administration of human cells and tissues. In academic centers, laboratories engaged in the preparation of cellular therapy products have typically evolved from bone marrow transplant service laboratories, research laboratories, or hospital-based blood transfusion service laboratories. Since 1989, the US Food and Drug Administration (FDA) has published a series of guidance documents and proposed regulations relevant to cellular therapy products. These new expectations have created substantial challenges for manufacturers, especially for academic-based laboratories that support early phase clinical trials not intended to result in a commercial product.
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References
Rosenberg SA. Immunotherapy of cancer by the systemic administration of lymphoid cells plus interleukin-2. J Biol Resp Mod 1984;3:501–11.
US Food and Drug Administration. Points to consider in the collection, processing, and testing of ex vivo-activated mononuclear leukocytes for administration to humans. Rockville, MD: Center for Biologics Evaluation and Research, 1989.
US Food and Drug Administration. Points to consider in human somatic cell therapy and gene therapy. Rockville, MD: Center for Biologics Evaluation and Research, 1991.
US Food and Drug Administration. Guidance for human somatic cell therapy and gene therapy. Rockville, MD: Center for Biologics Evaluation and Research, 1998.
US Food and Drug Administration. Application of current statutory authorities to human somatic cell therapy products and gene therapy products. Federal Register 1993;58:53248–51.
Kessler DA, Siegel JP, Noguchi PD, Zoon KA, Feiden KL, Woodcock J. Regulation of somatic-cell therapy and gene therapy by the Food and Drug Administration. N Engl J Med 1993;329:1169–73.
US Food and Drug Administration. Current good manufacturing practice in manufacturing, processing, packing, or holding of drugs. 21 CFR Part 210.
US Food and Drug Administration. Current good manufacturing practice for finished pharmaceuticals. 21 CFR Part 211.
US Food and Drug Administration. Reinventing the regulation of human tissue: National performance review. Rockville, MD: Center for Biologics Evaluation and Research, 1997.
US Food and Drug Administration. Proposed approach to the regulation of cellular and tissue-based products. Rockville, MD: Center for Biologics Evaluation and Research, 1997.
US Food and Drug Administration. Establishment registration and listing for manufacturers of human cellular and tissue-based products; Proposed rule. Federal Register 1998;63:26744–55.
US Food and Drug Administration. Suitability determination for donors of human cellular and tissue-based products; Proposed rule. Federal Register 1999;64:52696–23.
US Food and Drug Administration. Current good tissue practice for manufacturers of human cellular and tissue-based products; Inspection and enforcement; Proposed rule. Federal Register 2001;66:1508–59.
US Food and Drug Administration. Establishment registration and listing for manufacturers of human cellular and tissue-based products; Final rule. Federal Register 2001;66:5447–69.
US Food and Drug Administration. Current good manufacturing practice for blood and blood components. 21 CFR, Part 606.
US Food and Drug Administration. Establishment registration and product listing for manufacturers of human blood and blood products. 21 CFR, Part 607.
National Organ Transplant Act of 1984. 42 CFR, Part 121.
US Public Health Service Act, 42 U. S. C., section 361. 21 CFR, Part 610. 12.
US Food and Drug Administration. Validation of procedures for processing of human tissue intended for transplantation. Rockville, MD: Center for Bio-logics Evaluation and Research, 2002 (available at http://www.fda.gov/cber/gdlns/tissval.pdf).
Morbidity and Mortality Weekly Report 2002;51:207–10.
Advisory Panel on Gene and Cell Therapies to the US Pharmacopeia
Biotechnology and Gene Therapy Subcommittee. Cell and gene therapy based products, draft chapter to the US Pharmacopeia. Cytotherapy 2000;2: 123–71.
Standards for hematopoietic progenitor cell and cellular product services, 3rd Edition. American Association of Blood Banks. Bethesda, MD, 2002 (available at http://www.aabb.org).
Standards for tissue banking. American Association of Tissue Banks (available at http://www.aatb.org).
Standards for hematopoietic progenitor cell collection, processing, and transplantation, 2nd Edition. Foundation for the Accreditation of Cellular Therapy, 2002 (available at http://www.unmc.edu/community/fahct/default/htm).
Standards for blood and marrow progenitor cell processing, collection, and transplantation. Joint Accreditation Committee of ISHAGE-Europe and the EBMT, March 1998.
Guide to safety and quality assurance for organs, tissues, and cells. Council of Europe. Publishing, June 2002 (available at http://book.coe.int)
European Commission, Enterprise Directorate-General. Need for a legislative framework for human tissue engineering and tissue-engineered products, June 2002 (available at http://europa.eu.int/comm/enterprise/medical_devices/).
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Read, E.J. (2003). The New Regulatory Environment for Cellular Therapy Products: Challenges for Academic-Based Manufacturing Facilities. In: Sibinga, C.T.S., De Leij, L.F.M.H. (eds) Cellular Engineering and Cellular Therapies. Developments in Hematology and Immunology, vol 38. Springer, Boston, MA. https://doi.org/10.1007/978-1-4757-3718-9_5
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DOI: https://doi.org/10.1007/978-1-4757-3718-9_5
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