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Back to the future of stem cells

Jul 7, 2009 4:00 PM

With President Barack Obama’s recent easing of funding restrictions on embryonic stem cell research, and the support of key longtime supporters of stem cell research, such as Utah’s senior U.S. senator, Orrin G. Hatch, the University of Utah is poised to make its mark in cell therapy and regenerative medicine.

Several large vats of liquid nitrogen at the University’s Cell Therapy Facility keep stem cells frozen at 190 degrees Celsius. “It shuts down the cells’ metabolism so they don’t age,” says Linda L. Kelley, Ph.D., director of the University’s Cell Therapy Facility. “At that temperature, stem cells could last 40 or 50 years.”

The Cell Therapy Facility is the hub for much of the University’s stem cell research, processing and purifying the cells for transplanting in leukemia and lymphoma patients and for clinical trials to treat diseases of the heart, kidneys, and peripheral arteries. Kelley, professor of internal medicine, who has researched stem cells from the beginning of her career in the early 1990s, says clinical research is just catching up with the breakthroughs in basic science research over the past couple of decades.

But she expects President Barack Obama’s economic stimulus package, which funnels more money into clinical research and trials, to accelerate translational research using stem cells. The U School of Medicine’s Program in Cell Therapy and Regenerative Medicine is poised to build on this momentum. Lorris Betz, M.D., Ph.D., senior vice president for health sciences, executive dean of the medical school, and University of Utah Health Care CEO, says stem cell research and therapy “represents a totally new approach to treating many chronic and debilitating diseases. It could transform the way we treat disease.”

Mario R. Capecchi, Ph.D., 2007 Nobel Laureate in Physiology and Medicine agrees. “The therapeutic potential for embryonic stem cells and induced  pluripotent stem cells (adult stem cells that can be induced to return to their embryonic state) for many major human diseases is enormous. Now the hard work needs to be done so that this potential may be reached,” says Capecchi, distinguished professor of human genetics and biology.

Scientists long believed stem cells existed, but it wasn’t until the 1960s that their existence was proven. Embryonic stem cells (ESCs), which are taken from frozen embryos in the earliest stages of human development, are considered particularly valuable because they have the ability to turn into just about any cell type in the human body. But the use of ESCs harvested from frozen embryos has created a heated ethical controversy, prompting former President George W. Bush to ban federal funding for such research. In recent years, however, the discovery of induced pluripotent stem cells and the finding that regular skin cells can be “reprogrammed” to act like ESCs may defuse the controversy.

These developments have increased potential for using all types of stem cells in research and treatments, according to Kelley. “There probably won’t be one best stem cell source,” she says. “Many doors will open.”

Along with basic science investigation, current stem cell research at the University includes three clinical trials in which physician/researchers are investigating whether patients’ own adult stem cells can help regenerate new tissue in damaged hearts and kidneys, as well as blood vessels in the legs of people with peripheral artery disease.

In addition to directing the stem cell facility, Kelley conducts her own research. She’s the principal investigator on a new $5 million National Institutes of Health grant aimed at bringing a stem cell therapy for amyotrophic lateral sclerosis (AL S) to clinical trials within five years. The grant, in partnership with University of Utah spinoff Q Therapeutics Inc., will enable critical manufacturing and testing necessary for U.S. Food and Drug Administration approval to bring a cell-based therapy for AL S, also called Lou Gehrig’s disease, to human clinical trials.

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