TELL US WHAT YOU THINK!
NIH Seeks Public Input on Defining Unmet Needs for Clinical Translation of Cell-Based Therapies
The National Institutes of Health (NIH) Common Fund’s NIH Center for Regenerative Medicine (NIH CRM) seeks public comment 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.
This interactive web-based dialogue provides an opportunity for you to provide comments and engage in an open discussion on this topic. This site is referenced in the NIH Request for Information (RFI): Defining Unmet Needs for Clinical Translation of Cell-Based Therapies (NOT-RM-12-008
What is the NIH Common Fund?
The Common Fund supports goal-driven, research networks in which investigators work in a coordinated and synergistic way to generate data, solve technological problems, and/or otherwise create resources and tools that will be stimulatory to the broader research community. These programs typically involve a series of integrated funding initiatives that address scientific areas that are broadly relevant to human health and disease. Funding for each program is limited to 5-10 years, so goals for each program must be achievable within that timeframe. More information on the Common Fund can be found at http://commonfund.nih.gov/
How Can I Participate?
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. In this spirit, NIH CRM would like to solicit your feedback on specific activities that it could carry out that would address these practical and technical challenges. NIH CRM is particularly interested in receiving public feedback on the potential activities listed below, but feedback is not limited to these activities.
To weigh in
on a specific activity, simply click on the name of the activity to open a “dialogue box” where you can leave a comment, and also view and comment on others’ comments. You don’t have to respond to every topic, but you may.
ACTIVITIES FOR DISCUSSION:
1. Provision of services
- Repository for human induced pluripotent stem (iPS) cell lines
- Repository for human embryonic stem cell (ESC) lines
- Repository of human mesenchymal stem cells (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 human iPS (hiPS) cells or human ESCs (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 good management practice (GMP)- and good laboratory practice (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
Comments submitted via this site will be accepted until one month after the release date for the Request for Information (NOT-RM-12-008), also referenced above.
This interactive web-based dialogue site is only one way the NIH is gathering public input on this topic. Comments may also be submitted via Email according to instructions provided in the Request for Information (NOT-RM-12-008). Comments on the RFI received via this interactive web-based dialogue site will be treated identically to those received via Email. Therefore, it is important that you select only one method to use (i.e., E-mail or web-based dialogue) so that the NIH does not receive duplicate responses.
Response to the RFI via this interactive web-based dialogue site 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.