Pluripotent Stem Cells in Translation: A Food and Drug Administration‐National Institutes of Health Collaboration

• Published in: Stem cells translational medicine 2013-07, Vol.2 (7), p.483-487
• Main Authors: Kleitman, Naomi, Rao, Mahendra S., Owens, David F.
• Format: Article
• Language: English
• Subjects: Animals; Clinical translation; Clinical trials; Clinical Trials as Topic - trends; Collaboration; Cooperative Behavior; Drug dosages; Humans; Manufacturing; Medical equipment; Meeting Reports; Models, Animal; National Institutes of Health (U.S.); Pluripotency; Pluripotent stem cells; Pluripotent Stem Cells - cytology; Process controls; Product testing; Stem Cell Transplantation - trends; Stem cells; Translational Medical Research - trends; United States; United States Food and Drug Administration; Workshops
• ISSN: 2157-6564; 2157-6580
• DOI: 10.5966/sctm.2013-0042

Recently, the U.S. Food and Drug Administration (FDA), the U.S. National Institutes of Health, and the stem cell research community have collaborated on a series of workshops that address moving pluripotent stem cell therapies into the clinic. The first two workshops in the series focused on preclinical science, and a third, future workshop will focus on clinical trials. This summary addresses major points from both of the recent preclinically focused meetings. Recently, the U.S. Food and Drug Administration (FDA), the U.S. National Institutes of Health, and the stem cell research community have collaborated on a series of workshops that address moving pluripotent stem cell therapies into the clinic. The first two workshops in the series focused on preclinical science, and a third, future workshop will focus on clinical trials. This summary addresses major points from both of the recent preclinically focused meetings. When entering into a therapeutics developmental program based on pluripotent cells, investigators must make decisions at the very early stages that will have major ramifications during later phases of development. Presentations and discussions from both invited participants and FDA staff described the need to characterize and document the quality, variability, and suitability of the cells and commercial reagents used at every translational stage. This requires consideration of future regulatory requirements, ranging from donor eligibility of the original source material to the late‐stage manufacturing protocols. Federal, industrial, and academic participants agreed that planning backward is the best way to anticipate what evidence will be needed to justify human testing of novel therapeutics and to eliminate wasted efforts.