Regulatory considerations in production of a cell therapy medicinal product in Europe to clinical research

Clinical and Experimental Medicine

Volumn 14, Issue 1, 2014, Pages 25-33

  Martín, Patricia Gálvez ^a   Martinez, Adolfina Ruiz ^b   Lara, Visitación Gallardo ^b   Naveros, Beatriz Clares ^b  

^a CABIMER Andalusian Center for Molecular Biology and Regenerative Medicine CSIC University of Seville UPO   (Spain)

^b Faculty of Pharmacy   (Spain)

Author keywords

Advanced therapy; Cell therapy; Clinical trial; Stem cells

Indexed keywords

BIOMEDICAL RESEARCH; CELL- AND TISSUE-BASED THERAPY; DRUG APPROVAL; EUROPEAN UNION; HUMANS; STEM CELL TRANSPLANTATION;

EID: 84895107011     PISSN: 15918890     EISSN: 15919528     Source Type: Journal    
DOI: 10.1007/s10238-012-0213-6     Document Type: Article

References (35)

1. Mason C, Manzotti E (2009) Regen: the industry responsible for cell-based therapies. Regen Med 4:783-785
2. Committee for Advanced Therapies (CAT) (2010) Challenges with advanced therapy medicinal products and how to meet them. Nat Rev Drug Discov 9:195-201
3. Belardelli F, Rizza P, Moretti F, Carella C, Galli MC, Migliaccio G (2009) Translational research on advanced therapies. Ann Ist Super Sanita 47:72-78
4. Hughes-Wilson W, Mackay D (2007) European approval system for advanced therapies: good news for patients and innovators alike. Regen Med 2:5-6
5. Ottria G, Dallera M, Aresu O, Manniello MA, Parodi B, Spagnolo AM, Cristina ML (2010) Environmental monitoring programme in the cell therapy facility of a research centre: preliminary investigation. J Prev Med Hyg 51:133-138
6. Hogarth S, Salter B (2010) Regenerative medicine in Europe: global competition and innovation governance. Regen Med 5:971-985
7. Chamberlain G, Fox J, Ashton B et al (2007) Concise review: mesenchymal stem cells: their phenotype, differentiation capacity, immunological features, and potential for homing. Stem Cells 25:2739-2749
8. Verfaillie CM, Pera A, Lansdorp PM (2002) Stem cells: hype and reality. Hematol Am Soc Hematol Educ Program 1:369-391
9. Magee JA, Piskounova E, Morrison SJ (2012) Cancer stem cells: impact, heterogeneity, and uncertainty. Cancer Cell 20:283-296
10. Choi WY, Poss KD (2012) Cardiac regeneration. Curr Top Dev Biol 100:319-344
11. Naujok O, Lenzen S (2012) Pluripotent stem cells for cell replacement therapy of diabetes. Dtsch Med Wochenschr 137:1062-1066
12. Cohen-Haguenauer O (2011) Gene therapy and rare diseases. Rev Med Interne 32:S210-S212
13. Singh MS, MacLaren RE (2011) Stem cells as a therapeutic tool for the blind: biology and future prospects. Proc Biol Sci 278:3009-3016
14. Volarevic V, Ljujic B, Stojkovic P, Lukic A, Arsenijevic N, Stojkovic M (2011) Human stem cell research and regenerative medicine: present and future. Br Med Bull 99:155-168
15. Terzic A, Edwards BS, McKee KC, Nelson TJ (2011) Regenerative medicine: a reality of stem cell technology. Minn Med 94:44-47
16. Sokal EM (2011) From hepatocytes to stem and progenitor cells for liver regenerative medicine: advances and clinical perspectives. Cell Prolif 44:39-43
17. Parenteau NL (2009) Commercial development of cell-based therapeutics: strategic considerations along the drug to tissue spectrum. Regen Med 4:601-611
18. Dietz AB, Padley DJ, Gastineau DA (2007) Infrastructure development for human cell therapy translation. Clin Pharmacol Ther 82:320-324
19. Hampton JR (1983) The end of clinical freedom. Br Med J 287:237-1238
20. Patel SA, King CC, Lim PK et al (2010) Personalizing stem cell research and therapy: the arduous road ahead or missed opportunity? Curr Pharmacogen Person Med 8:25-36
21. Directive 2004/10/EC of the European parliament and of the council of 11 February 2004 on the harmonisation of laws, regulations and administrative provisions relating to the application of the principles of good laboratory practice and the verification of their applications for tests on chemical substances. Official J Eur Union L 50:44-59
22. Directive 2004/9/EC of the European parliament and of the council of 11 February 2004 on the inspection and verification of good laboratory practice (GLP). Official J Eur Union L 50:28-43
23. Directive 2001/20/EC the European parliament and of the council of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use. Official J Eur Union L 121:34-44
24. Yim R (2005) Administrative and research policies required to bring cellular therapies from the research laboratory to the patient's bedside. Transfusion 45:144-148
25. Trounson A, Thakar RG, Lomax G, Gibbons D (2011) Clinical trials for stem cell therapies. BMC Med 9:52
26. Commission directive 2005/28/EC of 8 April 2005 laying down principles and detailed guidelines for good clinical practice as regards investigational medicinal products for human use, as well as the requirements for authorisation of the manufacturing or importation of such products. Official J Eur Union L 91:13-19
27. Commission directive 2003/94/EC of 8 October 2003 laying down the principles and guidelines of good manufacturing practice in respect of medicinal products for human use and investigational medicinal products for human use. Official J Eur Union L 262:22-26
28. EudraLex - EU GMP Volume 4 (2009) Good manufacturing practice (GMP) guidelines-annex 1: manufacture of sterile medicinal products.EU GMP-Annex 1: Manufacture of Sterile Medicinal Products http://ec.europa.eu/health/documents/ eudralex/vol-4/index-en.htm. Accessed 15 April 2012
29. ICH Harmonised Tripartite Guideline Q2(R1) (1994) Validation of analytical procedures: text and methodology. http://www.ich.org/fileadmin/ Public-Web-Site/ICH-Products/Guidelines/Quality/Q2-R1/Step4/Q2-R1-Guideline.pdf. Accessed 15 April 2012
30. EudraLex - EU GMP Volume 4 (2001) Good manufacturing practice (GMP) guidelines-annex 15: qualification and validation http://ec.europa.eu/health/ documents/eudralex/vol-4/index-en.htm. Accessed 15 April 2012
31. Soncin S, Lo Cicero V, Astori G et al (2009) A practical approach for the validation of sterility, endotoxin and potency testing of bone marrow mononucleated cells used in cardiac regeneration in compliance with good manufacturing practice. J Transl Med 7:78
32. Regulation (EC) No 726/2004 of the European parliament and of the council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. Official J Eur Union L 136:1-51
33. Ruiz S, Abad-Santos F (2010) Regulation and evaluation of clinical trials of cell therapy. Med Clin 135:35-39
34. Celis P (2010) CAT-the new committee for advanced therapies at the European Medicines Agency. Bundesgesundheitsblatt-Gesund 53:9-13
35. Woods EJ, Bagchi A, Goebel WS et al (2010) Container system for enabling commercial production of cryopreserved cell therapy products. Regen Med 5:659-667