1. ¹Johns Hopkins Berman Institute of Bioethics, Johns Hopkins University, Baltimore, Maryland, USA
2. ²Department of Psychiatry and Behavioral Sciences, Johns Hopkins University, Baltimore, Maryland, USA
3. ³Abarbanel Mental Health Center, Bat Yam, Israel, affiliated with the Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
4. ⁴Department of Psychiatry, Harvard Medical School, Belmont, Massachusetts, USA
5. ⁵Institute for Cell Engineering, Johns Hopkins University, Baltimore, Maryland, USA
6. ⁶Department of Neurology, Johns Hopkins University, Baltimore, Maryland, USA
7. ⁷Department of Molecular Microbiology and Immunology, Johns Hopkins University, Baltimore, Maryland, USA
8. ⁸Division of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, Maryland, USA
9. ⁹Center for Bioethics, University of Minnesota, Minneapolis, Minnesota, USA
10. ¹⁰Johns Hopkins Transverse Myelopathy Center, Johns Hopkins University, Baltimore, Maryland, USA
11. ¹¹Georgetown University Law School, Georgetown University, Washington, District of Columbia, USA
12. ¹²Division of Pediatric Blood and Marrow Transplantation, Duke University Medical Center, Durham, North Carolina, USA
13. ¹³Faculty of Philosophy, Oxford University, Oxford, UK
14. ¹⁴International Center for Spinal Cord Injury, Kennedy Krieger Institute, Baltimore, Maryland, USA
15. ¹⁵Zanvyl/Krieger Mind Brain Institute, Johns Hopkins University, Baltimore, Maryland, USA
16. ¹⁶National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA
17. ¹⁷Invitrogen Corporation, Carlsbad, California, USA
18. ¹⁸Institute of Medical Biology, A*STAR, National University of Singapore, Singapore
19. ¹⁹Department of Biological Sciences and Biotechnology, University of Milan, Milan, Italy
20. ²⁰WM Keck Center for Collaborative Neuroscience, Rutgers University, Piscataway, New Jersey, USA
21. ²¹Stanford Law School, Stanford University, Stanford, California, USA
22. ²²University of Colorado at Denver, Denver, Colorado, USA

Correspondence: A Regenberg, Johns Hopkins Berman Institute of Bioethics, Johns Hopkins University, 100 N. Charles Street, Suite 740, Baltimore, MD 21201, USA. E-mail: aregenbe@jhsph.edu

Received 21 March 2008; Revised 30 June 2008; Accepted 5 August 2008; Published online 27 August 2008.

Abstract

Progress in regenerative medicine seems likely to produce new treatments for neurologic conditions that use human cells as therapeutic agents; at least one trial for such an intervention is already under way. The development of cell-based interventions for neurologic conditions (CBI-NCs) will likely include preclinical studies using animals as models for humans with conditions of interest. This paper explores predictive validity challenges and the proper role for animal models in developing CBI-NCs. In spite of limitations, animal models are and will remain an essential tool for gathering data in advance of first-in-human clinical trials. The goal of this paper is to provide a realistic lens for viewing the role of animal models in the context of CBI-NCs and to provide recommendations for moving forward through this challenging terrain.