Translational Stem Cell Research in Pediatrics: Ethical Issues

  • Michael Fuchs
Part of the Stem Cell Biology and Regenerative Medicine book series (STEMCELL)


The ethics of medical research constitutes one part within the field of medical ethics and bioethics where a far reaching consensus could be found at least as far as the principles and necessary procedures are concerned. Much more moral conflict is, however, prevalent in other parts of bioethics. The consensus achieved in the ethics of medical research was built upon the concept of informed consent, which is deemed a central aspect in the ethics of research on human beings. The focus on informed consent, however, causes problems in cases where research subjects are unable to give consent for whatever reason. Minors make up for one of the groups where the capability to give free and informed consent is, at least, problematic.

The lack of informed consent in the case of minors creates a dilemma in that paediatric research is needed for good paediatric practice and for the benefit of future minor individuals.

Against this background, alternative concepts such as assumed consent (McCormick), educational benefit or minimal risk have been developed.

With reference to the clinical translation of stem cells, detailed ethical and regulatory guidelines have been discussed. Nevertheless, no special recommendations for the clinical translation of stem cells to minors are in place at the current moment. The article examines the helpfulness of the distinct alternative concepts.


Gene therapy Instrumentalization Minimal risk Pediatric research Therapeutic attempt 


  1. 1.
    Plett P. Peter Plett und die übrigen Entdecker der Kuhpockenimpfung vor Edward Jenner. Sudhofs Archiv 2006; 2:219–32.Google Scholar
  2. 2.
    Fleischmann AR, Collogan LK. Research with children. In: Emanuel E, Grady C, Crouch RA, Lie RK, Miller, FG, Wendler D (Ed.): The Oxford Textbook of Clinical Research Ethics. New York: Oxford University Press 2008; 446–60, 447Google Scholar
  3. 3.
    Faden RR, Beauchamp TL. A History and Theory of Informed Consent. New York: Oxford University Press 1986.Google Scholar
  4. 4.
    Heinrichs B. Forschung am Menschen. Elemente einer ethischen Theorie biomedizinischer Humanexperimente. Berlin, New York: Walter de Gruyter 2006.Google Scholar
  5. 5.
    Magnus D. Medizinische Forschung an Kindern. Rechtliche, ethische und rechtsver­gleichende Aspekte der Arzneimittelforschung an Kindern. Tübingen: Mohr Siebeck 2006.Google Scholar
  6. 6.
    Stötter H. Paediatric drug development. Historical background of regulatory initiatives. In: Rose K, van den Anker JN (Ed.): Guide to Paediatric Clinical Research. Basel: Karger 2007; 25–32.Google Scholar
  7. 7.
    Rose K, Stötter H. ICH E 11: clinical investigation of medicinal products in the paediatric population. The international guidance on clinical drug development in children. In: Rose K, van den Anker JN (Ed.): Guide to Paediatric Clinical Research. Basel: Karger 2007; 33–7.Google Scholar
  8. 8.
    Varma S, Wendler D. Risk-benefit assessment in pediatric Research. In: Emanuel E, Grady C, Crouch R A, Lie RK, Miller, FG, Wendler D. (Ed.): The Oxford Textbook of Clinical Research Ethics. New York: Oxford University Press 2008; 527–38.Google Scholar
  9. 9.
    Council of Europe–Directorate of Legal Affairs. Explanatory Report to the Convention for the Protection of Human Rights and Dignity of the Human Being with Regard to the Application of Biology and Medicine: Convention on Human Rights and Biomedicine, Strasbourg 1997–DIR/JUR (97) 5.Google Scholar
  10. 10.
    Ramsey P. The Patient as Person. Exploration in Medical Ethics, 2nd Edition. New Haven: Yale University Press 2002, 39.Google Scholar
  11. 11.
    McCormick R. Experimentation in children: Sharing in sociality. A reply to Paul Ramsey. In: The Hastings Center Report 1976; 6:41–6.Google Scholar
  12. 12.
    Brock DW. Ethical issues in exposing children to risks in research. In: Grodin MA, Glantz LH (Ed.): Children as Research Subjects. Science, Ethics, and Law. Oxford, New York: Oxford University Press 1994; 81–101.Google Scholar
  13. 13.
    Osieka TO. Das Recht der Humanforschung : unter besonderer Berücksichtigung der 12. Arzneimittelgesetz-Novelle. – Hamburg : Kovac 2006; 246.Google Scholar
  14. 14.
    Neubauer D, Laitinen-Parkkonen P, Matthys D. Ethical challenges of clinical research in children. Protection from risks vs. access to benefits. In: Rose K, van den Anker JN (Ed.): Guide to Paediatric Clinical Research. Basel: Karger 2007; 38–46.Google Scholar
  15. 15.
    International Society for Stem Cell Research (ISSCR) (2008): Guidelines for the Clinical Translation of Stem Cells. URL [September 1, 2009].
  16. 16.
    Lau D, Ogbogu U, Taylor B, Stafinski T, Menon D, Caulfield T. Stem cell clinics online: the direct-to-consumer portrayal of stem cell medicine. Cell Stem Cell 2008; 3:591–4.PubMedCrossRefGoogle Scholar
  17. 17.
    Heyer M (2010). Do therapies based on the application of the body’s own adult stem cells need authorization or approval? An evaluation. (; June 15, 2010).
  18. 18.
    Honnefelder L. Ethical, legal and social evaluation of recent stem cell research, North-Rhine Westphalias’s approach. Brussels, Lecture, October 1st 2009.Google Scholar
  19. 19.
    Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 1995; 270:4705. PubMedCrossRefGoogle Scholar
  20. 20.
    Hoogerbrugge PM, van Beusechem VW, Fischer A, Debree M, le Deist F, Perignon JL et al. Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Ther 1996; 3:179–83.PubMedGoogle Scholar
  21. 21.
    Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296:2410–3.PubMedCrossRefGoogle Scholar
  22. 22.
    Aiuti A, Vai S, Mortellaro A, Casorati G, Ficara F, Andolfi G et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med 2002; 8:423–5.PubMedCrossRefGoogle Scholar
  23. 23.
    Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288:669–72.PubMedCrossRefGoogle Scholar
  24. 24.
    Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New Engl J Med 2002; 346:1185–93.PubMedCrossRefGoogle Scholar
  25. 25.
    Walters L, Palmer JG. The Ethics of Human Gene Therapy. New York, Oxford: Oxford University Press 1997.Google Scholar
  26. 26.
    Heinemann T, Heinrichs B, Klein C, Fuchs M, Hübner D. Der “kontrollierte individuelle Heilversuch” als neues Instrument bei der klinischen Erstanwendung risikoreicher Therapieformen. Ethische Analyse einer somatischen Gentherapie für das Wiskott-Aldrich-Syndrom. Jahrbuch für Wissenschaft und Ethik 2006; 11:153–99.Google Scholar
  27. 27.
    Appelbaum PS, Lidz CW. The therapeutic misconception. In: Emanuel E, Grady C, Crouch R A, Lie RK, Miller, FG, Wendler D. (Ed.): The Oxford Textbook of Clinical Research Ethics. New York: Oxford University Press 2008; 633–44.Google Scholar
  28. 28.
    Hermerén G. The role of the expert in ethics committees. In: Council of Europe (Ed.): Standing Conference of European Ethics Committees. Proceedings, Stockholm 1994; 42–8.Google Scholar

Copyright information

© Springer Science+Business Media, LLC 2011

Authors and Affiliations

  • Michael Fuchs
    • 1
  1. 1.Institut für Wissenschaft und EthikUniversität BonnBonnGermany

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