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Roadmap to overcome obstacles for using induced pluripotent stem cells

June 27, 2017

Another concern with using either iPSCs or embryonic stem cells is that cells with the ability to turn into many different cell types may grow out of control, producing cancerous tumors. Knoepfler points out those studies involved implanting large numbers of undifferentiated stem cells into mice that were treated with immunosuppressant drugs to reject transplants, making the conditions ideal for cancers to arise. This scenario, he argues, is unlikely to be applicable when treating humans for actual diseases. In such cases, the stem cells would be induced to have a specific function, and the body's natural immune defenses would be present.

The "pluripotent" nature of stem cells, which potentially allow their use to repair almost any tissue, is only beginning to be harnessed for human therapies. Stem cell therapy has already been successfully used for years to treat leukemia and related bone and blood cancers. The use of iPSCs could vastly increase the spectrum of diseases that might be treated with stem cells, without the safety and ethical concerns inherent in using embryonic stem cells.

"Dr. Barrilleaux and I argue for a shift in research priorities," said Knoepfler. "Future studies of iPSCs should increasingly focus on issues most relevant to the eventual clinical use of the cells, offering the fastest pathway to treating patients with this potentially powerful therapeutic tool."

Knoepfler's own research focuses on determining how stem cell behavior is controlled during normal embryonic development as well as during healing and regeneration. He also studies how control systems go awry in developmental disorders and cancer. One key direction for the Knoepfler lab is using leading genomics technology to better understand why stem cells behave the way they do and how to change that behavior for clinical use.

Funding for Knoepfler's article was provided by a grant from the California Institute for Regenerative Medicine.

Source: University of California - Davis Health System