Stem Cell Reviews and Reports

, Volume 5, Issue 2, pp 135–139 | Cite as

Product Regulation and the Clinical Translation of Stem Cell Research

  • Barbara von TigerstromEmail author


The recent approval by the United States Food and Drug Administration of a clinical trial involving a product derived from human embryonic stem cells, along with recent concerns about unproven stem cell therapies being offered to patients, highlight the importance of regulation at the critical stage of beginning human trials of novel therapies. The regulations governing therapeutic products (drugs and related products) are one part of the broader legal framework, but will play an increasingly prominent role as we move into clinical translation. The classification of products as drugs or biologics, on one hand, or minimally manipulated cell and tissue products for homologous use, on the other, will determine the requirements that will apply, including whether use in clinical trials requires approval. Product regulation works alongside other parts of the legal and policy framework, notably research ethics review and legal responsibilities of medical professionals, that play important though limited roles. Three key developments and challenges currently facing product regulation and related areas will affect stem cell research in this phase: regulatory reform, fragmentation, and capacity.


Legal Ethics Regulation Clinical translation Clinical trials 



This commentary is based in part on research funded by the Stem Cell Network (Canada) core grant “Towards the Clinic?: Ethical, Legal and Social Issues (ELSI) Relevant to Emerging Stem Cell Therapies” and completed with the assistance of Bobbi-Jo Cammer, Jacqueline Shaw, Jodi Roach, Erin Schroh, and Arif Chowdhury (LL.B. students, University of Saskatchewan).


  1. 1.
    Geron (2009). Geron Receives FDA Clearance to begin world’s first human clinical trial of embryonic stem cell-based therapy. News Release January 23, 2009. Accessed February 2, 2009, at
  2. 2.
    Geron (2009). FDA Places Geron’s GRNOPC1 IND on clinical hold. News Release May 14, 2008. Accessed February 2, 2009, at
  3. 3.
    Pollack, A. (2009). F.D.A. approves a stem cell trial. New York Times January 23, 2009. Accessed February 2, 2009, at
  4. 4.
    International Society for Stem Cell Research (2008). Guidelines for the clinical translation of stem cells. December 3. Accessed February 2, 2009, at
  5. 5.
    Lau, D., Ogbogu, U., Taylor, B., Stafinski, T., Menon, D., Caulfield, T. (2008). Stem cell clinics online: the direct-to-consumer portrayal of stem cell medicine. Cell Stem Cell, 3, 591–594.PubMedCrossRefGoogle Scholar
  6. 6.
    Fox, J. L. (2008). FDA scrutinizes human stem cell therapies. Nature Biotechnology, 26, 598–599.PubMedCrossRefGoogle Scholar
  7. 7.
    Scott, C. T. (2008). Weighing risks and rewards en route to the clinic. Nature Reports Stem Cells, December 18, 2008. doi: 10.1038/stemcells.2008.158.
  8. 8.
    EC. Regulation (EC) No 1394 of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004, [2007] O.J. L 324/121.Google Scholar
  9. 9.
    United States Food and Drug Administration (1998). Guidance for human somatic cell therapy and gene therapy. FDA Center for Biologics Evaluation and Research.Google Scholar
  10. 10.
    World Medical Association (2008). Declaration of Helsinki: ethical principles for medical research involving human subjects. Amended October 2008. Accessed February 2, 2009, at
  11. 11.
    United States. Food and Drugs, Regulation, 21 C.F.R., Part 312, § 312.34.Google Scholar
  12. 12.
    Canada. Food and Drug Regulations, C.R.C., c. 870, s. C.08.010-011.Google Scholar
  13. 13.
    EC. Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community Code Relating to Medicinal Products for Human Use, [2001] O.J. L 311/67.Google Scholar
  14. 14.
    Health Canada (2008). Guidance document for industry and practitioners: special access programme for drugs. Health Products and Food Branch. Accessed February 2, 2009, at
  15. 15.
    Caplan, A. (2007). Is it sound public policy to let the terminally ill access experimental medical innovations? Am J Bioethics, 7(6), 1–3.CrossRefGoogle Scholar
  16. 16.
    United Kingdom Medicines and Healthcare products Regulatory Agency (2008). Concept paper on the review of the regulation of unlicensed medicines. Accessed February 2, 2009, at
  17. 17.
    EC. Directive 2004/23/EC of the European Parliament and of the Council of 31 March 2004 on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells, [2004] O.J. L 102/48.Google Scholar
  18. 18.
    Canada. Safety of Human Cells, Tissues and Organs for Transplantation Regulations, S.O.R./2007-118.Google Scholar
  19. 19.
    United States. Human Cells, Tissues, and Cellular and Tissue-Based Products Regulations, 21 C.F.R., Part 1271.Google Scholar
  20. 20.
    Isasi, R., Nguyen, T. M. (2008). The rationale for a registry of clinical trials involving human stem cell therapies. Health Law Rev, 16(2), 56–68.Google Scholar
  21. 21.
    Experts Committee for Human Research Participant Protection in Canada (2008). Moving ahead: Final report. Accessed February 2, 2009, at
  22. 22.
    McDonald, M., et al. (2000). The governance of health research involving human subjects. Ottawa: Law Commission of Canada.Google Scholar
  23. 23.
    Canadian Institutes of Health Research (2007). Updated guidelines for human pluripotent stem cell research, June 29, 2007. Accessed February 2, 2009, at
  24. 24.
    Davies, M. (2008). Raising the bar: ethics review, governance, and accountability in community-based human health research. Saskatchewan Law Review, 71, 91–116.Google Scholar
  25. 25.
    Therapeutic Goods Administration (Australia) (2008). Summary of the human cell and tissue therapies framework. Accessed February 2, 2009, at
  26. 26.
    Yeates, N., Lee, D. K., Maher, M. (2007). Health Canada’s progressive licensing framework. CMAJ, 176, 1845–1847.PubMedGoogle Scholar
  27. 27.
    Parliament of Canada (2008). Bill C-51: an act to amend the food and drugs act and to make consequential amendments to other acts. 39th Parliament, 2nd Session.Google Scholar
  28. 28.
    Hebert, P. C. (2007). Progressive licensing needs progressive open debate. CMAJ, 176, 1801.PubMedGoogle Scholar
  29. 29.
    European Medicines Agency (2008). Guideline on safety and efficacy follow-up—risk management on advanced therapy medicinal products. London.Google Scholar
  30. 30.
    Enserink, M. (2005). Selling the stem cell dream. Science, 131, 160–163.Google Scholar
  31. 31.
    Pray, L., Robinson, S. (2007). Challenges for the FDA: the future of drug safety, Workshop summary. National Academies.Google Scholar
  32. 32.
    von Tigerstrom, B. (2007). Globalisation, harmonisation and the regulation of therapeutic products: the Australia New Zealand Therapeutic Products Authority Project in global context. Canterbury Law Rev, 13, 287–313.Google Scholar
  33. 33.
    World Health Organization (2004). WHO medicines strategy: countries at the core, 2004–2007. Geneva: World Health Organization.Google Scholar

Copyright information

© Humana Press 2009

Authors and Affiliations

  1. 1.College of LawUniversity of SaskatchewanSaskatoonCanada

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