Key Action Items
In an article in World Stem Cell Report 2013, Dr. Knoepfler identified several key action items for the stem cell field in 2014. These are important because they relate not only to the field of pluripotent stem cells, but also to adult stem cell-based research and development. They include the need to train a new generation of stem cell clinicians, balance advocacy for responsible reforms at the FDA with the need to work with the agency, and monitor and raise awareness of noncompliant stem cell interventions no longer only available outside the U.S. but more recently proliferating at stem cell clinics across the U.S. He also put out the call to leverage social media to educate the public about stem cells and obtain funding for stem-cell-related research.
The advantages of pluripotency can be exploited to produce a targeted population of cells for transplantation, genetic manipulation, delivery of a therapeutic agent, or regeneration of an organ. Stem cell lines are valuable research tools for studying cell biology, embryogenesis and developmental biology, and are proving increasingly useful in drug discovery and development for producing cell-based models of disease and for preclinical toxicology studies.
Pluripotency can have a dark side as well, and when pluripotency control mechanisms go awry due to genomic or epigenomic changes, cell immortality and tumorigenicity may result.
Timothy Kamp, M.D., Ph.D., professor and co-director, Stem Cell and Regenerative Medicine Center at the University of Wisconsin, Madison, presented his group’s latest work differentiating human pluripotent stem cells to create spontaneously contracting cardiomyocytes for cell therapy to repair cardiac muscle damaged as a result of ischemia (myocardial infarction), cardiomyopathies, certain arrhythmias, or valvular disease, for example.
The paradigm he described for producing iPSC involves reprogramming skin or blood cells, expanding and cryopreserving the pluripotent stem cells to create an iPSC bank, and then using those cells to make relevant cellular products for cardiac repair or a tissue engineered cardiac patch. Dr. Kamp’s group has demonstrated proof-of-principle in mice that ESC can induce regeneration of cardiomyocytes in infarcted heart muscle. iPSC technology is “rapidly evolving,” said Dr. Kamp, as he demonstrated their progress toward growing human iPS-engineered cardiac tissue in the lab as a 3-dimensional model of functional human myocardium.