Request for Information Defining Unmet Needs for Clinical Translation of Cell-Based Therapies

Notice Number: NOT-RM-12-008

Key Dates
Release Date: March 2, 2012

Issued by
National Institutes of Health (NIH)
Office of Strategic Coordination (Common Fund)


The Common Fund’s NIH Center for Regenerative Medicine (NIH CRM) seeks comments on persistent unmet needs that currently impede technical and scientific progress in translating advances in stem-and progenitor cell-based technologies to the clinic, and where NIH investment and/or coordination can help in overcoming these impediments.


Successful clinical translation of cell-based therapies faces many practical and technical challenges. Yet, the potential pay-off of these therapies for medical practice is very high, and thus the enthusiasm remains strong. The Common Fund established NIH CRM within the NIH Intramural Research Program to serve as a resource for the scientific community (NIH Center for Regenerative Medicine). In this spirit, NIH CRM solicits your feedback on specific activities that it could carry out addressing these practical and technical challenges. NIH CRM is particularly interested in receiving feedback on the potential activities listed below but feedback is not limited to these activities:

Information Requested:

This request for information (RFI) invites feedback from the scientific and clinical research communities, other interested organizations, and the public on potential NIH CRM activities including but not limited to the following:

1. Provision of services

  • Repository for human iPS cell lines
  • Repository for human ESC lines
  • Repository of human MSCs
  • Service to generate iPS cell lines at cost, and transfer to the user for characterization and use
  • Service to generate iPS cell lines and characterize them to a minimum performance standard (e.g. differentiation along all three germ lines, karyotype, teratoma formation, epigenetic state, HLA type, others)
  • Developing and disseminating standard protocols
  • Providing standardized reporter lines for comparison as investigators develop their own hiPS or hESCs
  • Establishing a large bank of characterized cell lines that reflect the genetic diversity in the population

2. Addressing issues important to accelerating the translation of cell-based therapies

  • Defining how many cells are needed for a particular application
  • Establishing clear standards for GMP- and GLP-level outputs
  • Preservation and storage of labile materials (e.g. cells, media, engineered tissues)
  • Providing reference standards to developers
  • Providing fully synthetic cell processing platforms (i.e. xeno-free culture media and reagents)
  • Developing reliable cell labeling and tracking schemes for shipping and storage purposes
  • Establishing centralized facilities to ease standardization and compliance issues
  • Freedom to operate (i.e. centralized access to relevant patents and licenses)
  • Common human subjects consent procedures and anonymization for cell sources
  • Establishing a well-characterized common source (e.g. cord blood, bone marrow, skin) as a starting point for deriving iPS cells lines

How to Submit a Response:

The NIH is gathering input in two different ways:

1. Respond to this RFI via E-mail within one month of the release date given above to: Please include the RFI number (NOT-RM-12-008) in the subject line of your response.

2. Respond to this RFI via an interactive web-based dialogue: by submitting comments via the internet within one month of the release date. This format provides an opportunity to view and comment on other participants' responses.

All responses will be treated identically, no matter which method is used to submit comments. However, it is important that only one method is selected (i.e. E-mail or web-based dialogue) so that the NIH does not receive duplicate responses.

Response to this RFI is voluntary. All interested parties are invited to respond. Responders are free to provide feedback on any or all of the above items as well as to suggest additional NIH CRM activities not listed. Responders should be aware that the information provided will be analyzed and may appear in various reports. Any personal identifiers (e.g. names, addresses, E-mail addresses, etc.) will be removed when responses are compiled. Only the de-identified comments will be used. However, the government cannot guarantee the confidentiality of the information provided. Proprietary, classified, confidential, or sensitive information should not be included in your response. Please also note that the government will not pay for response preparation or for the use of any information contained in the response.

This RFI is for information and planning purposes only and should not be construed as a solicitation or as an obligation on the part of the Federal Government, the NIH, or the NIH Common Fund to make any awards or implement any activities suggested, recommended, or commented on. Acknowledgement of receipt of responses may not be made, nor will respondents be notified of NIH CRM's analysis of the information received. No basis for claims against the Federal Government or the NIH shall arise as a result of a response to this RFI or the NIH's use of such information. Any proprietary information should be so marked.

General Information:

Note: All of the following fields are optional. Proprietary, classified, confidential, or sensitive information should not be included in your response.

1. Please identify the nature of your interest in the area (i.e. are you a biomedical or clinical researcher, a member of an advocacy or community group, or other?).
2. Please indicate the name of your organization.
3. Please indicate your main area of research interest.
4. Your name.
5. Your E-mail address.


Please direct all inquiries to:

Scott Lipnick, Ph.D.
Tel: (301) 496-6798
NIH Center for Regenerative Medicine
50 Center Drive, Room 1139
MSC 8024
Bethesda, MD 20892
Tel: 301-402-6956
Fax: 301-480-6367