References
- Flory E, Reinhardt J. European Regulatory Tools for Advanced Therapy
Medicinal Products. Transfusion Medicine and Hemotherapy.
2013;40(6):5-5.
- Committee for Medicinal Products for Human Use, European Medicines
Agency. Alofisel® EPAR Public Assessment Report EMA/CHMP/64055/2018
Corr.1
https://www.ema.europa.eu/en/documents/assessment-report/alofisel-epar-public-assessment-report_en.pdf.
Accessed May 13, 2020
- Committee for Medicinal Products for Human Use, European Medicines
Agency. Yescarta® EPAR Public Assessment Report EMA/481168/2018.
https://www.ema.europa.eu/en/documents/assessment-report/yescarta-epar-public-assessment-report_en.pdf.
Accessed May 13, 2020.
- Committee for Medicinal Products for Human Use, European Medicines
Agency. Kymriah® EPAR Public Assessment Report EMA/485563/2018.
https://www.ema.europa.eu/en/documents/assessment-report/kymriah-epar-public-assessment-report_en.pdf.
Accessed May 13, 2020.
- Committee for Medicinal Products for Human Use, European Medicines
Agency. Zynteglo® EPAR Public Assessment Report. EMA/56140/2020/Corr.1
https://www.ema.europa.eu/en/documents/assessment-report/zynteglo-epar-public-assessment-report_en.pdf.
Accessed May 13, 2020.
- European Commission. Regulation (EC) No 1394/2007. Regulation (EC) No
1394/2007. https://eur-lex.europa.eu/eli/reg/2007/1394/oj.
Accessed May 7, 2020.
- Commission for Advanced Therapies, European Medicines Agency.
Guideline on quality, non-clinical and clinical requirements for
investigational advanced therapy medicinal products in clinical trials
EMA/CAT/852602/2018.
https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-quality-non-clinical-clinical-requirements-investigational-advanced-therapy_en.pdf.
Accessed May 12, 2020.
- Pound P, Ritskes-Hoitinga M. Can prospective systematic reviews of
animal studies improve clinical translation? J Transl Med 2020; 18:15.
- Schinnerling K, Rosas C, Soto l, Thomas R and Aguillón JC, Humanized
Mouse Models of Rheumatoid Arthritis for Studies on Immunopathogenesis
and Preclinical Testing of Cell-Based Therapies. Front Immunol 2019;
Feb 1910:203.
- Perel P, Roberts I, Sena E, Wheble P, Briscoe C, Sandercock P, Macleod
M, Mignini LE, Jayaram P, Khan KS Comparison of treatment effects
between animal experiments and clinical trials: systematic review.
BMJ2007; 334:197.
- Federal Drug Agency. Preclinical Assessment of Investigational
Cellular and Gene Therapy Products FDA-2012-D-1038.
https://www.fda.gov/media/87564/download. Accessed May 12, 2020.
- Commission for Advanced Therapies, European Medicines Agency.
Guideline on human cell-based medicinal products.
EMEA/CHMP/410869/2006.
https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-human-cell-based-medicinal-products_en.pdf.
Accessed May 12, 2020.
- Reyes B, Coca MI, Codinach M, López-Lucas MD, Del Mazo-Barbara A,
Caminal M, Oliver-Vila I, Cabañas V, Lope-Piedrafita S, García-López
J, Moraleda JM, Fontecha CG, Vives J. Assessment of biodistribution
using mesenchymal stromal cells: Algorithm for study design and
challenges in detection methodologies. Cytotherapy 2017;
Sep19(9):1060-1069.
- Leahy M, Thompson K, Zafar H, Alexandrov S, Foley M, O’Flatharta C,
Dockery P. Functional imaging for regenerative medicine. Stem Cell Res
Ther 2016; Apr 19;7(1):57.
- International Conference on Harmonisation. ICH S1 Guideline on the
need for carcinogenicity studies of pharmaceuticals S1A.
https://database.ich.org/sites/default/files/S1A%20Guideline.pdf.
Accessed May 12, 2020.
- World Health Organization Technical Report Series 978 Annex 3 2013.
Recommendations for the evaluation of animal cell cultures as
substrates for the manufacture of biological medicinal products and
for the characterization of cell banks (Replacement of Annex 1 of WHO
Technical Report Series, No. 878). Appendix 2, Tumourigenicity
protocol using athymic nude mice to assess mammalian cells.
https://www.who.int/biologicals/vaccines/TRS_978_Annex_3.pdf?ua=1.
Accessed May 12, 2020.
- Barkholt L, Flory E, Jekerle V, Lucas-Samuel S, Ahnert P, Bisset
L,Büscher D, Fibbe W, Foussat A, Kwa M, Lantz O, Mačiulaitis R,
Palomäki T, Schneider CK, Sensebé L, Tachdjian G, Tarte K, Tosca L,
Salmikangas P. Risk of tumorigenicity in mesenchymal stromal
cell–based therapies—Bridging scientific observations and
regulatory viewpoints. Cytotherapy 2013; 15(7):753-759.
- Kuroda T, Yasuda S, Kusakawa S, Hirata N, Kanda Y, Suzuki K, Takahashi
M, Nishikawa S, Kawamata S, Sato Y. Highly Sensitive In vitroMethods for Detection of Residual Undifferentiated Cells in Retinal
Pigment Epithelial Cells Derived from Human iPS Cells. PLoS ONE 2012;
7(5):e37342.
- Borowicz S, Van Scoyk M, Avasarala S, Karuppusamy-Rathinam M, Tauler
J., Bikkavilli R, and Winn R. (2014). The Soft Agar Colony Formation
Assay. J Neurosurg 2009; 110(6):1189–1197.
- Helfer BM, Ponomarev V, Patrick S, Blower P, Feitel A, Fruhwirth G,
Jackman S, Pereira Mouriès L, Park M, Srinivas M, Stuckey D, Thu M,
Van den Hoorn T, Herberts CA, Shingleton W. Options for imaging
cellular therapeutics in vivo ; a multi stakeholder perspective.
(In preparation).
- Sato Y, Bando H, Di Piazza M, Gowing G, Herberts CA, Jackman S, Leoni
G, Libertini S, MacLachlan T, McBlane JW, Pereira Mouriès L, Sharpe M,
Shingleton W, Surmacz-Cordle B, Yamamoto K, van der Laan JW,
Tumorigenicity assessment of cell therapy products: The need for
global consensus and points to consider. Cytotherapy 2019;
21:1095-1111
- Vestergaard HT, Apote LD, Schneider CK, Herberts C. The evolution of
nonclinical regulatory science: Advanced therapy medicinal products as
a paradigm. Molecular Therapy 2013; 21(9):1644-1648
- Harting MT, Jimenez F, Xue H, Fischer UM, Baumgartner J, Dash PK and
Cox CS. Intravenous mesenchymal stem cell therapy for traumatic brain
injury. Laboratory investigation Journal of Neurosurgery 2014;
110(6):1189-1197
- Mount NM, Ward SJ, Kefalas P, Hyllner J. Cell-based therapy technology
classifications and translational challenges. Phil. Trans. R. Soc. B
2015; 370:20150017
- Mestas J and Hughes CCW. Of Mice and Not Men: Differences between
Mouse and Human Immunology J Immunol March 1, 2004; 172(5):2731-2738.
- Oosterwijk-Wakka JC, Tiemessen DM, Bleumer I, de Vries IJ, Jongmans W,
Adema GJ, Debruyne FM, de Mulder PH, Oosterwijk E, Mulders PF.
Vaccination of patients with metastatic renal cell carcinoma with
autologous dendritic cells pulsed with autologous tumor antigens in
combination with interleukin-2: a phase 1 study. J Immunother 2002;
25(6):500-8.
- Balsari A, Marolda R, Gambacorti-Passerini C, Sciorelli G, Tona G,
Elisabetta Cosulich E, Donatella Taramelli D, Giuseppe Fossati G,
Giorgio Parmiani G, Cascinelli N. Systemic administration of
autologous, alloactivated helper-enriched lymphocytes to patients with
metastatic melanoma of the lung - A phase I study. Cancer Immunology
Immunotherapy 1986; 21(2): 148-155.
- Fibbe WE, Noort WA, Mesenchymal stem cells and hematopoietic stem cell
transplantation Annals of the New York Academy of Sciences 2002; 996:
235-244.
- Commission Advanced Therapies, Clinical Trial Facilitation Group &
European Commission. Q&A on Good laboratory practice (GLP) principles
in relation to ATMPs
https://www.ema.europa.eu/en/documents/other/good-laboratory-practice-glp-principles-relation-advanced-therapy-medicinal-products-atmps_en.pdf.
Accessed May 12, 2020.
- Kuroda T, Yasuda S, Sato Y. In vitro detection of residual
undifferentiated cells in retinal pigment epithelial cells derived
from human induced pluripotent stem cells. Methods Mol Biol. 2014;
1210:183-92.