References
Bear, A. S., R. A. Morgan, K. Cornetta, C. H. June, G. Binder-Scholl, M. E. Dudley, S. A. Feldman, S. A. Rosenberg, S. A. Shurtleff, C. M. Rooney, H. E. Heslop, and G. Dotti. 2012. Replication-competent retroviruses in gene-modified T cells used in clinical trials: Is it time to revise the testing requirements? Molecular Therapy 20(2):246–249.
Bersenev, A. 2017. Historical analysis of cell-based therapeutic products marketed and approved worldwide. Cell Trials: Current Trends in Cell Therapy. April 7. http://celltrials.info/2017/04/07/marketed-approved (accessed August 1, 2017).
Buechner, J., S. A. Grupp, S. L. Maude, M. Boyer, H. Bittencourt, T. W. Laetsch, P. Bader, M. R. Verneris, J. Stefanski, G. D. Myers, M. Qayed, M. A. Pulsipher, B. D. Moerloose, H. Hiramatsu, K. Schlis, K. Davis, P. L. Martin, E. R. Nemecek, C. Peters, P. Wood, T. Taran, K. T. Mueller, Y. Zhang, and S. Rives. 2017. Global registration trial of efficacy and safety of CTL019 in pediatric and young adult patients with relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL): Update to the interim analysis. Presented at European Hematology Association Annual Meeting, June 24, 2017, Madrid.
Crabbe, M. A., K. Gijbels, A. Visser, D. Craeye, S. Walbers, J. Pinxteren, R. J. Deans, W. Annaert, and B. L. Vaes. 2016. Using miRNA-mRNA interaction analysis to link biologically relevant miRNAs to stem cell identity testing for next-generation culturing development. Stem Cells Translational Medicine 5(6):709–722.
Dahl, S. L., A. P. Kypson, J. H. Lawson, J. L. Blum, J. T. Strader, Y. Li, R. J. Manson, W. E. Tente, L. DiBernardo, M. T. Hensley, R. Carter, T. P., Williams, H. L. Prichard, M. S. Dey, K. G. Begelman, and L. E. Niklason. 2011. Readily available tissue-engineered vascular grafts. Science Translational Medicine 3(68)68ra9.
FDA (U.S. Food and Drug Administration). 2012. The road to the biotech revolution: Highlights of 100 years of biologics regulation. https://www.fda.gov/aboutfda/whatwedo/history/forgshistory/cber/ucm135758.htm (accessed August 28, 2017).
Grupp, S. A., M. Kalos, D. Barrett, R. Aplenc, D. L. Porter, S. R. Rheingold, D. T. Teachey, A. Chew, B. Hauck, J. F. Wright, M. C. Milone, B. L. Levine, and C. H. June. 2013. Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. New England Journal of Medicine 368(16):1509–1518.
Haddock, R., S. Lin-Gibson, N. Lumelsky, R. McFarland, K. Roy, K. Saha, J. Zhang, and C. Zylberberg. 2017. Manufacturing cell therapies: The paradigm shift in health care of this century. NAM Perspectives. Discussion Paper. National Academy of Medicine, Washington, DC. https://nam.edu/manufacturing-cell-therapies-the-paradigm-shift-in-healthcare-of-this-century (accessed September 12, 2017).
Heslop, H. E., K. S. Slobod, M. A. Pule, G. A. Hale, A. Rousseau, C. A. Smith, C. M. Bollard, H. Liu, M. F. Wu, R. J. Rochester, P. J. Amrolia, J. L. Hurwitz, M. K. Brenner, and C. M. Rooney. 2010. Long-term outcome of EBV-specific T-cell infusions to prevent or treat EBV-related lymphoproliferative disease in transplant recipients. Blood 115(5):925–935.
Hollyman, D., J. Stefanski, M. Przybylowski, S. Bartido, O. Borquez-Ojeda, C. Taylor, R. Yeh, V. Capacio, M. Olszewska, J. Hosey, M. Sadelain, R. J. Brentjens, and I. Rivière. 2009. Manufacturing validation of biologically functional T cells targeted to CD19 antigen for autologous adoptive cell therapy. Journal of Immunotherapy 32(2):169–180.
Kalos, M., B. L. Levine, D. L. Porter, S. Katz, S. A. Grupp, A. Bagg, and C. H. June. 2011. T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia. Science Translational Medicine 3(95):95ra73.
Kelly Scientific Publications. 2017. Global regenerative medicine market analysis and forecast to 2021; stem cells, tissue engineering, biobanking & CAR-T industries. https://www.reportlinker.com/p04876076/Global-Regenerative-Medicine-Market-Analysis-Forecast-to-Stem-Cells-Tissue-Engineering-BioBanking-CAR-T-Industries.html (accessed September 12, 2017).
Klinker, M. W., R. A. Marklein, J. L. Lo Surdo, C. H. Wei, and S. R. Bauer. 2017. Morphological features of IFN-gamma-stimulated mesenchymal stromal cells predict overall immunosuppressive capacity. Proceedings of the National Academy of Sciences of the United States of America 114(13):E2598–E2607.
Lawson, J. H., M. H. Glickman, M. Ilzecki, T. Jakimowicz, A. Jaroszynski, E. K. Peden, A. J. Pilgrim, H. L. Prichard, M. Guziewicz, S. Przywara, J. Szmidt, J. Turek, W. Witkiewicz, N. Zapotoczny, T. Zubilewicz, and L. E. Niklason. 2016. Bioengineered human acellular vessels for dialysis access in patients with end-stage renal disease: Two Phase 2 single-arm trials. Lancet 387(10032):2026–2034.
Lehman, N., R. Cutrone, A. Raber, R. Perry, W. Van’t Hof, R. Deans, A. E. Ting, and J. Woda. 2012. Development of a surrogate angiogenic potency assay for clinical-grade stem cell production. Cytotherapy 14(8):994–1004.
Lo Surdo, J. L., B. A. Millis, and S. R. Bauer. 2013. Automated microscopy as a quantitative method to measure differences in adipogenic differentiation in preparations of human mesenchymal stromal cells. Cytotherapy 15(12):1527–1540.
Louis, C. U., K. Straathof, C. M. Bollard, S. Ennamuri, C. Gerken, T. T. Lopez, M. H. Huls, A. Sheehan, M. F. Wu, H. Liu, A. Gee, M. K. Brenner, C. M. Rooney, H. E. Heslop, and S. Gottschalk. 2010. Adoptive transfer of EBV-specific T cells results in sustained clinical responses in patients with locoregional nasopharyngeal carcinoma. Journal of Immunotherapy 33(9):983–990.
Malik, N. 2012. Allogeneic versus autologous stem-cell therapy. BioPharm International 25(7). Epub date: July 1, 2012. http://www.biopharminternational.com/allogeneic-versus-autologous-stem-cell-therapy (accessed August 29, 2017).
Marklein, R. A., J. L. Lo Surdo, I. H. Bellayr, S. A. Godil, R. K. Puri, and S. R. Bauer. 2016. High content imaging of early morphological signatures predicts long term mineralization capacity of human mesenchymal stem cells upon osteogenic induction. Stem Cells 34(4):935–947.
Maude, S. L., N. Frey, P. A. Shaw, R. Aplenc, D. M. Barrett, N. J. Bunin, A. Chew, V. E. Gonzalez, Z. Zheng, S. F. Lacey, Y. D. Mahnke, J. J. Melenhorst, S. R. Rheingold, A. Shen, D. T. Teachey, B. L. Levine, C. H. June, D. L. Porter, and S. A. Grupp. 2014. Chimeric antigen receptor T cells for sustained remissions in leukemia. New England Journal of Medicine 371(16):1507–1517.
Mendicino, M., A. M. Bailey, K. Wonnacott, R. K. Puri, and S. R. Bauer. 2014. MSC-based product characterization for clinical trials: An FDA perspective. Cell Stem Cell 14(2):141–145.
Mitsuyasu, R. T., P. A. Anton, S. G. Deeks, D. T. Scadden, E. Connick, M. T. Downs, A. Bakker, M. R. Roberts, C. H. June, S. Jalali, A. A. Lin, R. Pennathur-Das, and K. M. Hege. 2000. Prolonged survival and tissue trafficking following adoptive transfer of CD4zeta gene-modified autologous CD4(+) and CD8(+) T cells in human immunodeficiency virus-infected subjects. Blood 96(3):785–793.
Muraro, P. A., R. Martin, G. L. Mancardi, R. Nicholas, M. P. Sormani, and R. Saccardi. 2017a. Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis. Nature Reviews: Neurology 13(7):391–405.
Muraro, P. A., M. Pasquini, H. L. Atkins, J. D. Bowen, D. Farge, A. Fassas, M. S. Freedman, G. E. Georges, F. Gualandi, N. Hamerschlak, E. Havrdova, V. K. Kimiskidis, T. Kozak, G. L. Mancardi, L. Massacesi, D. A. Moraes, R. A. Nash, S. Pavletic, J. Ouyang, M. Rovira, A. Saiz, B. Simoes, M. Trneny, L. Zhu, M. Badoglio, X. Zhong, M. P. Sormani, and R. Saccardi. 2017b. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurology 74(4):459–469.
NASEM (National Academies of Sciences, Engineering, and Medicine). 2017. Exploring the state of the science in the field of regenerative medicine: Challenges of and opportunities for cellular therapies: Proceedings of a workshop. Washington, DC: The National Academies Press.
NIH (National Institutes of Health). 2013. Regenerative medicine. https://www.report.nih.gov/NIHfactsheets/ViewFactSheet.aspx?csid=62 (accessed September 13, 2017).
Niklason, L. E., J. Gao, W. M. Abbott, K. K. Hirschi, S. Houser, R. Marini, and R. Langer. 1999. Functional arteries grown in vitro. Science 284(5413):489–493.
Palberg, T., J. Johnson, S. Probst, P. Gil, J. Rogalewicz, S. Chalk, M. Kennedy, and K. Green. 2011. Challenging the cleanroom paradigm for biopharmaceutical manufacturing of bulk drug substances. BioPharm International 24(8):44–60.
Park, J. H., I. Rivière, X. Wang, T. Purdon, M. Sadelain, and R. J. Brentjens. 2016. Impact of disease burden on long-term outcome of 19-28z CAR modified t cells in adult patients with relapsed B-ALL. Journal of Clinical Oncology 34(15 Suppl):7003.
Porter, D. L., B. L. Levine, M. Kalos, A. Bagg, and C. H. June. 2011. Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia. New England Journal of Medicine 365(8):725–733.
Przybylowski, M., A. Hakakha, J. Stefanski, J. Hodges, M. Sadelain, and I. Rivière. 2006. Production scale-up and validation of packaging cell clearance of clinical-grade retroviral vector stocks produced in cell factories. Gene Therapy 13(1):95–100.
Roh, K. H., R. M. Nerem, and K. Roy. 2016. Biomanufacturing of therapeutic cells: State of the art, current challenges, and future perspectives. Annual Reviews of Chemical and Biomolecular Engineering 7:455–478.
Schuster, S. J., M. R. Bishop, C. Tam, E. K. Waller, P. Borchmann, J. McGuirk, U. Jäger, S. Jaglowski, C. Andreadis, J. Westin, I. Fleury, V. Bachanova, S. R. Foley, P. J. Ho, S. Mielke, H. Holte, O. Anak, L. Pacaud, R. Awasthi, F. Tai, G. Salles, and R. Maziarz. 2017. Global pivotal phase 2 trial of the cd19-targeted therapy ctl019 in adult patients with relapsed or refractory (r/r) diffuse large b-cell lymphoma (DLBCL)—an interim analysis. Hematological Oncology 35:27.
Sormani, M. P., P. A. Muraro, R. Saccardi, and G. Mancardi. 2017. NEDA status in highly active MS can be more easily obtained with autologous hematopoietic stem cell transplantation than other drugs. Multiple Sclerosis 23(2):201–204.
Themeli, M., I. Rivière, and M. Sadelain. 2015. New cell sources for T cell engineering and adoptive immunotherapy. Cell Stem Cell 16(4):357–366.
Turner, L., and P. Knoepfler. 2016. Selling stem cells in the USA: Assessing the direct-to-consumer industry. Cell Stem Cell 19(2):154–157.
Walker, R. E., C. M. Bechtel, V. Natarajan, M. Baseler, K. M. Hege, J. A. Metcalf, R. Stevens, A. Hazen, R. M. Blaese, C. C. Chen, S. F. Leitman, J. Palensky, J. Wittes, R. T. Davey, Jr., J. Falloon, M. A. Polis, J. A. Kovacs, D. F. Broad, B. L. Levine, M. R. Roberts, H. Masur, and H. C. Lane. 2000. Long-term in vivo survival of receptor-modified syngeneic T cells in patients with human immunodeficiency virus infection. Blood 96(2):467–474.
