Date: December 12, 2011
Response Due Date: February 9, 2012
National Institutes of Health (NIH)
The National Institutes of Health (NIH) and the Food and Drug Administration (FDA) are organizing a series of workshops that will engage the broader research community regarding requirements necessary for translation of pluripotent stem cell-derived products into the clinic. The purpose of this RFI is to solicit information pertinent to developing the second workshop, which will focus on preclinical safety and proof-of-concept evaluation including, among other topics: clinical relevance of the animal models of disease, translation of cell dose levels from animal to human, assessment of cell engraftment/survival in animals, effect of delivery site on cell behavior, and cell disposition post-administration.
Human pluripotent stem cells have the potential to both self-renew and differentiate into specialized cell populations that can be used to repair, replace, and regenerate damaged tissue. These characteristics offer substantial promise for development into therapeutic products to address unmet medical needs. The state of stem cell science is advancing rapidly and research activities in this area, including those directed to clinical application, are expected to multiply in the next few years. Will pluripotent stem cells pose additional challenges to therapeutic development beyond those encountered with other types of somatic cellular products?
To enable more effective translation to clinical research and increase the coordination between the research and regulatory communities, the National Institutes of Health and the Food and Drug Administration are planning a workshop series to engage the broader research community regarding prerequisites necessary for translation of investigational pluripotent stem cell-derived products into the clinic.
The first workshop, held March 21-22, 2011, focused on establishment of stem cell lines and banks, covering safety and regulatory issues related to how the original cell line derivation is done in the case of embryonic stem cells, and how the cells are reprogrammed in the case of iPS cells (Day 1: http://videocast.nih.gov/Summary.asp?File=16558; Day 2: http://videocast.nih.gov/Summary.asp?File=16557). The second workshop will focus on preclinical safety and proof -of -concept assessment, including, among other topics, clinical relevance of the animal models of disease, translation of cell dose levels from animal to human, assessment of cell engraftment/survival in animals, effect of delivery site on cell behavior, and cell disposition post-administration,. The third, and final, workshop is expected to focus on clinical trial design, including safety monitoring, immunogenicity, immunosuppressant use and immune monitoring.
NIH is seeking information that will be useful in developing the second workshop of the series. Specifically, NIH is seeking comment on the following five items:
1. Describe the challenges that exist in the preclinical evaluation of pluripotent stem cell-derived products to enable their translation from the laboratory into clinical trials.
2. Present key questions posed by pluripotent stem cell biology that should be addressed using animal models to assess safety and potential clinical effectiveness.
3. Identify any laboratories, research institutions, companies or countries that have experience with translational stem cell research that can serve as models for discussion.
4. Describe what can be learned from our experience with other cell therapies that is applicable to products derived from pluripotent stem cells.
5. Present other questions that should be addressed at the workshop.
Note: Responses to this RFI are voluntary. The information provided will be analyzed and may appear in various reports. Any personal identifiers (e.g., names, addresses, email addresses, etc.) will be removed when responses are compiled. Proprietary, classified, confidential, or sensitive information should not be included in your response.
Responses should be submitted via email to firstname.lastname@example.org, and will be accepted through February 9, 2012.
This Request for Information (RFI) is for planning purposes only and is not a solicitation for applications or an obligation on the part of the United States (U.S.) Government to provide support for any ideas identified in response to it. Please note that the U.S. Government will not pay for the preparation of any comment submitted or for its use of that comment.
Please direct all inquiries to:
Marino, Ph.D., Office of the Director
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