Request Information: Scientific Regulatory Issues be Explored an Upcoming Pluripotent Stem Cell Workshop involving NIH FDA Notice Number: NOT-NS-12-004 Key Dates Release Date: December 12, 2011 Response Due Date: February 9, 2012 Related Notices NOT-NS-10-016 Issued National Institutes Health NIH) Purpose National Institutes Health NIH) the Food Drug Administration FDA) organizing series workshops will engage broader research community regarding requirements necessary translation pluripotent stem cell-derived products the clinic. purpose this RFI to solicit information pertinent developing second workshop, will focus preclinical safety proof-of-concept evaluation including, among topics: clinical relevance the animal models disease, translation cell dose levels animal human, assessment cell engraftment/survival animals, effect delivery site cell behavior, cell disposition post-administration. Background Human pluripotent stem cells the potential both self-renew differentiate specialized cell populations can used repair, replace, regenerate damaged tissue. characteristics offer substantial promise development therapeutic products address unmet medical needs. state stem cell science advancing rapidly research activities this area, including those directed clinical application, expected multiply the next few years. pluripotent stem cells pose additional challenges therapeutic development beyond those encountered other types somatic cellular products? enable effective translation clinical research increase coordination between research regulatory communities, National Institutes Health the Food Drug Administration planning workshop series engage broader research community regarding prerequisites necessary translation investigational pluripotent stem cell-derived products the clinic. first workshop, held March 21-22, 2011, focused establishment stem cell lines banks, covering safety regulatory issues related how original cell line derivation done the case embryonic stem cells, how cells reprogrammed the case iPS cells Day 1:
http://videocast.nih.gov/Summary.asp?File=16558; Day 2:
http://videocast.nih.gov/Summary.asp?File=16557). second workshop focus preclinical safety proof concept assessment, including, among topics, clinical relevance the animal models disease, translation cell dose levels animal human, assessment cell engraftment/survival animals, effect delivery site cell behavior, cell disposition post-administration,. third, final, workshop expected focus clinical trial design, including safety monitoring, immunogenicity, immunosuppressant and immune monitoring. Information Requested NIH seeking information will useful developing second workshop the series. Specifically, NIH seeking comment the following five items: 1. Describe challenges exist the preclinical evaluation pluripotent stem cell-derived products enable translation the laboratory clinical trials. 2. Present key questions posed pluripotent stem cell biology should addressed using animal models assess safety potential clinical effectiveness. 3. Identify any laboratories, research institutions, companies countries have experience translational stem cell research can serve models discussion. 4. Describe can learned our experience other cell therapies is applicable products derived pluripotent stem cells. 5. Present questions should addressed the workshop. Note: Responses this RFI voluntary. information provided be analyzed may appear various reports. Any personal identifiers e.g., names, addresses, email addresses, etc.) be removed responses compiled. Proprietary, classified, confidential, sensitive information should be included your response. Responses Responses should submitted via email
nindsstemcell@mail.nih.gov, will accepted through February 9, 2012. Request Information RFI) for planning purposes only is a solicitation applications an obligation the part the United States U.S.) Government provide support any ideas identified response it. Please note the U.S. Government not pay the preparation any comment submitted for use that comment. Inquiries Please direct inquiries to: Susan Marino, Ph.D., Office the Director National Institute Neurological Disorders Stroke National Institutes Health Phone: 301-594-4470 FAX: 301-496-0296 Email: marinosw@ninds/nih.gov