Request for Information: Scientific and Regulatory Issues to be Explored at an Upcoming Pluripotent Stem Cell Workshop involving NIH and FDA

Notice Number: NOT-NS-10-016

Key Dates
Release Date:    May 6, 2010
Response Due By:  July 16, 2010

Issued by
National Institutes of Health (NIH), (http://www.nih.gov)

Purpose

The National Institutes of Health (NIH) and the Food and Drug Administration (FDA) are proposing 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 that will be useful in developing the first workshop which will focus on establishment of pluripotent stem cell lines and banks, manufacturing for early phase clinical trials, manufacturing for commercial production, and characterization of human stem cell-derived products.

Background

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?

The development of effective therapeutic products derived from pluripotent stem cells will require the ability to demonstrate safety and other characteristics of the starting cell populations. In addition, effective methods will have to be developed to produce the target cells of choice consistently through execution of controlled in vitro differentiation protocols.  Finally, analytic test methods will need to be developed that permit identification and characterization of the intended final cell type(s), as well as facilitate assessment of additional non-target cells present in the final product. These test methods should possess sufficient precision to ensure product safety and anticipate possible effectiveness. 

To enable the translation to clinical research to occur more effectively, and to increase the coordination between the research and regulatory communities, the National Institutes of Health and the Food and Drug Administration are proposing 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 first workshop will focus 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.  This workshop is expected to address the issues of  cell line generation and banking, manufacturing for early phase clinical trials (academic and industry perspectives), manufacturing for commercial production, and characterization of human stem cell-derived products (methods, limitations, and current developments).  The second workshop is expected to focus on preclinical safety and  proof of concept  testing, including, among other topics, clinical relevance of the animal models, selection of cell dose levels, impact of host microenvironment, and cell fate post-administration.  The third workshop is expected to focus on clinical trial design including safety monitoring, immunogenicity, immunosuppressant use and immune monitoring.

Information Requested

NIH is seeking information that will be useful in developing the first workshop of the series. Specifically, NIH is seeking input to the following five questions:

  1. What challenges exist in moving pluripotent cells from the laboratory into clinical trials?
  2. What key questions are posed by pluripotent stem cell biology with respect to safety and potential clinical effectiveness?
  3. Are you aware of any laboratories, research institutions, companies or countries that have experience with safety and regulatory translational stem cell research that can serve as models for discussion?
  4. What can be learned from our experience with other cell therapies that is applicable to products derived from pluripotent stem cells?
  5. What other questions should be asked at the workshop?

Responses

Responses should be submitted via email to nindsstemcell@mail.nih.gov, and will be accepted through July 16, 2010. 

Inquiries

Specific questions about this RFI should be directed to the contact listed below:

Susan Marino, PhD
Office of the Director
National Institute of Neurological Disorders and Stroke
National Institutes of Health
marinosw@ninds.nih.gov


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