Notice Number: NOT-NS-14-032

Update: The following update relating to this announcement has been issued:

• September 5, 2014 - Notice to Inform Potential Applicants to RFA-NS-14-006. See Notice NOT-NS-14-044.

Key Dates
Release Date: May 13, 2014

Issued by
National Institute of Neurological Disorders and Stroke (NINDS)

Purpose

The purpose of this Notice is to alert the research community to the current NINDS best practices guidelines for development and distribution of human induced pluripotent stem cells (iPSC) through the NINDS Repository, also known as the NINDS Human Genetics Resource Center. The iPSC lines available through the NINDS Repository were primarily developed through the American Recovery and Reinvestment Act and collaborations with government (California Institute for Regenerative Medicine (CIRM)) and non-government organizations (the Amyotrophic Lateral Sclerosis Association, the Association for Frontotemporal Degeneration, CHDI, the Hereditary Disease Foundation, the Huntington's Disease Society of America, the Michael J. Fox Foundation, and the Parkinson's Disease Foundation). For further information on NINDS Repository banking guidance see NOT-NS-12-003.

All iPSC lines available through the NINDS Repository have a Certificate of Analysis (CofA) which provides quality control data (sterility, cell recovery, karyotype, identity match (if applicable), surface antigen expression of stem cell markers and pluripotency analysis), as well as information on the method of derivation, passage method, passage number and split ratio for each line. The NINDS Repository catalog is updated frequently, and investigators are encouraged to visit the website on a regular basis to access the availability of new human fibroblast and iPSC lines.

Based on the availability of these resources, NINDS will no longer support the development of iPSC lines (e.g. control lines and/or lines with specific mutations) that are already represented in the NINDS Repository or in similar resources such as the NIMH or NIGMS Repositories. Moreover, NINDS is not presently seeking applications that are focused primarily on developing iPSC lines solely as a resource. Investigators who propose to develop and use iPSC lines not represented in the NINDS Repository or similar resources, for hypothesis-driven research, and anticipate depositing these lines in the NINDS Repository, are strongly encouraged to consult their NINDS program director prior to submission of a grant application.

Quality Control and Freedom to Operate Requirements for iPSC lines to be banked with the NINDS Repository

In addition to requiring compliance with standard NIH resource sharing policies, NINDS may request that novel investigator-developed iPSC lines be made available through the NINDS Repository, consistent with achieving the goals of this program. Such a request for deposition of iPSC lines will be made prior to the release of a notice of grant award, and will be specified in the notice of grant award. For the human iPSC lines to be deposited with the NINDS Repository, investigators will need to meet the following requirements prior to the release of a notice of grant award:

• Patient consent must allow for de-identified and broad data and resource sharing (academic and industry investigators) including use for genetic studies, wherein part or all of the genome may be sequenced;
• When IP is applicable, the institution/facility must have all necessary legal authority for sharing, and document this, including any necessary licenses for iPSC and related (e.g. genome editing, reporter use) technologies that allow deposition and broad distribution of resulting iPSC lines through the NINDS Repository;
• All iPSC lines derived under NINDS funding mechanisms must be characterized for sterility and be free of mycoplasma contamination, have normal karyotypes, normal growth rates and colony morphology, demonstrated pluripotency through a pluritest, scorecard test or equivalent test, demonstrate surface antigen expression of stem cell markers, demonstrated ability to form embryoid bodies and demonstrated transgene silencing for the reprogramming factors used;
• A timeline must be provided for banking of available iPSC lines with the NINDS Repository.

Investigators are strongly encouraged to contact their NINDS program director prior to application submission to determine the broad applicability of the iPSC lines to be developed.

Development of isogenic iPSC Lines
For generation of isogenic lines, that are to be deposited in the NINDS Repository, and where the disease causing mutation is represented in NINDS Repository iPSC lines, investigators are encouraged to perform gene editing in the available NINDS Repository iPSC lines. Furthermore, such grant applications should budget for and propose whole genome sequencing of the parent and edited clones. The genetic data generated along with available de-identified clinical data will be deposited with the NIH database of Genotypes and Phenotypes (dbGaP).

Inquiries

Please direct all inquiries to:

Margaret Sutherland, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-5680
Email: [email protected]

David Owens, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-1447
Email: [email protected]

Please direct NINDS Repository inquiries to:

Roderick A. Corriveau, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-5680
Email: [email protected]