The progress made in stem cell biology over the past decade has opened up exciting new opportunities for basic and translational scientists worldwide. Induced pluripotent stem cells (iPSCs) are particularly useful because scientists can differentiate them into many different cell types that are relevant for drug discovery and clinical therapies. Stem Cell Translation Laboratory (SCTL) scientists investigate iPSCs that have been derived by reprogramming adult cells (e.g. skin cells) into embryonic-like stem cells. These patient and disease-specific cells have the potential to become any cell type of the human body. The SCTL is developing the most rigorous and efficient iPSC differentiation protocols for dopamine neurons (e.g. Parkinsons disease), motor neurons (e.g. Lou Gehrigs diseases), insulin-producing cells (type 1 diabetes), hepatocytes (e.g. liver failure), nociceptors (e.g. opioid research), and other cell types relevant for both understanding disease mechanisms as well as cell therapy applications. We are also developing new quality control measures for quality control, genome stability, and cell maturation. One recent breakthrough finding is the discovery of new small molecules that dramatically improve viability of iPSCs for cryopreservation or single cell cloning, which now allows efficient and streamlined genome editing with CRISPR/Cas in pluripotent stem cell lines. To bring iPSC-based therapies more efficiently to patients, it is pivotal to continue addressing the key knowledge gaps and roadblocks in a coordinated multidisciplinary fashion. On September 26, 2017, NCATS hosted the Workshop on Translational Challenges of Induced Pluripotent Stem Cells (iPSC) on the NIH campus in Bethesda, Maryland. This workshop focused on defining collaborative opportunities, addressing challenges and applying best practices in translational iPSC research. Topics of interest included quality control standards for pluripotency, safety, experimental reproducibility, cost-efficient scalability and manufacturing, improved and efficient differentiation protocols, advances in -omics and functional characterization of cell type identities, and other relevant questions. Workshop goals included: 1) Highlighting the biological and technological challenges of advancing iPSCs into clinical applications and drug discovery; 2) Evaluating the state-of-the field of iPSC translation and discussing critical steps for quality control, standardization and reproducibility; 3) Discussing methods of protocol development/optimization and molecular and functional analyses; and 4) Seeking input from experts on areas of prioritization and tractable strategies for the iPSC field. Ongoing Collaborations: 1) Joseph Wu, MD, PhD (Stanford University): iPSCs for isolating human lineage-specific cardiovascular cells 2) Mark Schurdak, PhD (University of Pittsburgh): Optimization of protocols to terminally differentiat hiPSCs from normal and Huntingtons disease patients for quantitative systems pharmacology drug discovery 3) NIH LINCS Consortium (Cedars Sinai & Mount Sinai): A library of signatures from iPSCs and their derived cardiomyocytes and neurons
| García-Castro, Javier; Singeç, Ilyas (2017) Prospects of Pluripotent and Adult Stem Cells for Rare Diseases. Adv Exp Med Biol 1031:371-386 |
| Singeç, Ilyas; Simeonov, Anton (2016) Translating Stem Cell Biology Into Drug Discovery. Drug Target Rev 3:34-38 |
