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Update on NIH/FDA Pluripotent Stem Cells in Translation: Early Decisions

The event notice indicates that the meeting will be webcast at a link to be announced and many speakers indicated that their slide presentations will also be available - a significant and valuable resource to take advantage of.
Stand out meeting sessions from a legal perspective includes- Session 1 FDA Roles in the Translation Process, NIH roles in the Translation Process, in Session 2 Regulatory Challenges , in Session 3 NIH Guidelines, FDA Donor Eligibility and Cell Banking requirements, in Session 5 Here's what we expect (FDA) . Session speakers confirmed what one described: that early decisions made in research could be show stoppers at therapy stage.
Issues about donor eligibility and testing requirements were recurring subjects. Presentations revealed complex compliance issues. Attention was called to the importance of developing products with the needs of the future clinical site and staff in mind. Some of the early decisions in research/product development that could impact future clinical use included: the need to retain samples, check vendor supply chain and lot variability, check reagents for their pathogenic status, check the kinds of surfaces used for HESC growth, even the scale of vessels containing the growing cells is important since both nutrients and oxygenation make big differences for growth. Having a pure product is important but beyond that, knowing fully what the composition of the product is is as important. Each day ended with Wrap-ups/ Summaries -great ways to pull together and recap highlights of the day's sessions. FDA and NIH wrap ups noted that best practices were emerging- what are the early decisions in R&D that will impact clinical products, the control of cell and reagent sources, donor testing, knowing about any inherent characteristics incompatible with commercial scale product, stability and issues in scalability in stem cell manufacturing, and informed consent requirements. Another challenge: IVF practices in regard to their testing of couples among other issues.
The presentations also provide references for exploration -a few are listed below.

-http://ACD.OD.NIH.GOV/HESC.ASP- The ACD working Group for HESCs review and advise the NIH Director about their findings in regard to specific stem cell line applications, about the NIH stem cell registry and info about eligible lines, including if any, a variety of specific donor restrictions on the use of that line. The latest info for informed consent requirements, such as the new no exculpatory language requirement, may also be found on the NIH Stem Cell webpage side bar
-http://www.niaid.nih.gov/Volunteer/HIVandInfectious/Pages/pact.aspx--The Partnership for Access to Clinical Trials (PACT) program brings together community-based health care providers, their patients, and research clinicians from NIAID.
-http://www.ukstemcellbank.org.uk/ The UK bank to establish and make available fully characterized and quality-controlled cell banks. These will be supplied to scientific research teams and eventually to pharmaceutical companies, to enable the development of broad-ranging cell therapies
-http://www.scripps.edu/newsandviews/e_20100111/loring.html- In Session 6, ( Emerging Technology) the new 'pluritest' technology was described, see the 'pluritest.org' website for more info. Also discussed during the presentation, and described in more detail in The Scripps Institute news, excerpt: "an international team of scientists led by researchers at The Scripps Research Institute has developed a straightforward technique to determine the ethnic origin of stem cells. They initiated the study--published in the January 2010 edition of the prestigious journal Nature Methods--because the availability of genetically diverse cell lines for cell replacement therapy and drug development could have important medical consequences. Research has shown that discordance between the ethnic origin of organ donors and recipients can influence medical outcomes for tissue transplantation, and that the safety and effectiveness of specific drugs can vary widely depending on ethnic background." Vol 10. Issue 1 /January 11, 2010, News and Views, Online weekly of The Scripps Research institute

Of note:FDA Regulatory activities include regulatory review and policy but also include outreach via the Guidances, Advisory Committees and workshops like this one. Especially important to keep in mind about this developing subject area are the different roles of the NIH and FDA.. FDA oversees clinical investigation under IND reviews, regardless of funding and FDA enforces rules about the sources of stem cells for that purpose. NIH funded research is also subject to rules about the sources of stem cells but they are for a different purposes and so differ in certain ways. Another point made- while there is still a scientific question about the definitive characteristics of what a pluripotent cell is, speakers reiterated that pluripotency itself is not a regulatory requirement . In conducting its risk assessment in IND review , FDA noted that the cell type of the product would play an important role, also important in their review: how the patient route of administration might affect the dose and bio distribution and survival of cells. Regulatory authority cites were provided throughout presentations. FDA called attention to two recent Guidances on cell therapy and product potency. As an overview, the roadmap to translation for cell therapy to clinic overview starts with isolating cells, isolating , expanding and quantifying them, storing and maintaining the cells- tests are needed for each step.
Guidance for Industry Potency Tests for Cellular And Gene Therapy Products, and Guidance for Industry, Cellular Therapy for Cardiac Disease. http://www.fda.gov/BiologicsbloodVaccines/GuidanceComplianceRegulatroyInformation/guidances/default.htm

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