Taking Stem Cell Technology to the Clinic

More than 160 scientists from Cedars-Sinai and the University of Wisconsin – Madison explored ways to nudge stem-cell technology closer to the clinic at a Nov. 20-22 symposium hosted by the Cedars-Sinai Board of Governors Regenerative Medicine Institute at the Harvey Morse Conference Center.

William L. Murphy, PhD, co-director of the Stem Cell and Regenerative Medicine Center at the University of Wisconsin – Madison, addresses scientists at November symposium.

Symposium attendees browse scientific posters at Cedars-Sinai.

"Stem cells can really become a transformative biomedical tool," William L. Murphy, PhD, co-director of the Stem Cell and Regenerative Medicine Center at the University of Wisconsin – Madison, said in the opening keynote address. Already, he explained, more than 300 clinical trials utilizing stem cells are under way, and the world's 12 largest pharmaceutical corporations all maintain active programs in this area.

The stem-cell technique consists of engineering adult human skin cells to return to an embryonic state. Once altered, these so-called Induced pluripotent stem cells, or iPSCs, can produce virtually any cell of the human body for use in modeling disease processes, studying human development and testing potential drug therapies.

Dhruv Sareen, PhD, director of the Cedars-Sinai Induced Pluripotent Stem Cell Core and assistant professor of biomedical sciences, said the next step in this still-young science is to assemble stem cells into "complex systems" that "more faithfully model human disease."

Murphy said he foresees a future in which tissues self-assemble on computer chips and researchers transform stem cells into functioning human organs.

But as the symposium made clear, this remarkable technology must be made more efficient, economical and sophisticated to advance to the next level and into the medical marketplace. Among the issues:

  • Laboratory staffs face a "daily headache" of maintaining iPSCs cells, which do not always differentiate into the desired cell types and must be screened to ensure normal chromosomal status, said Sareen. These imperfections could pose safety risks for future therapeutic use of these cells, he explained.
  • Mass-producing iPSCs and their products can be prohibitively expensive, given the high-priced steroid and hormone supplements that are added to influence cell growth and differentiation, Murphy said. He and his colleagues are seeking cheaper ways to achieve these goals, such as harnessing existing biomaterials, including endogenous growth factors, and altering the substrate on which cells are cultured.
  • Tissues produced through iPSCs need to be more specialized, said Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute and professor of biomedical sciences and medicine. It is not enough to generate a generic motor neuron, he explained in his closing keynote address; the neuron must function within a system. And to study adult-onset diseases, such as amyotrophic lateral sclerosis (Lou Gehrig's disease), scientists must be able to "age" the cells and tissues.

A key purpose of this year's annual gathering, "Emerging Technologies in Regenerative Medicine," was to foster "collaborations and synergies" between Cedars-Sinai and the University of Wisconsin – Madison, both major centers of stem-cell research, Sareen said. "This was a wonderful symposium," he added, that produced many actionable items.

Murphy was equally enthusiastic. "This has been an absolutely fantastic meeting," he told the Harvey Morse Auditorium audience before heading back to Wisconsin.