Cutting-edge cures: UC's stem cell research

Imagine a patch that could repair the tissue damage of a heart attack. Or something that could save a diabetic's limbs from being amputated or reverse the damage done by lung cancer. Cure for AIDS, Parkinson's disease or Alzheimer's? All of these possibilities and more are on the agenda at the University of California's stem cell research labs.

Hundreds of UC researchers are delving into the mysteries of both embryonic and adult stem cells, and the outcome of their work has the potential to revolutionize the practice of medicine. Every UC campus has faculty engaged in stem cell research and the equally important mission of training future researchers and doctors in this fast-developing field of regenerative medicine.

Hope for spinal cord injuries

UC research is behind the world's first embryonic stem cell study using humans. The U.S. Federal Drug Administration has just approved human trials to treat acute spinal cord injuries. The therapy is based on the research of Hans Keirstead, co-director of UC Irvin's Sue and Bill Gross Stem Cell Research. Menlo Park-based Geron Corp. is conducting the trials. Listen to Keirstead's views on using stem cells from embryos.

"This trial was approved only after rigorous safety testing and consultation of countless experts in the field," Keirstead said. "Any benefit to the patient, even an incremental one, would be a resounding victory."

Keirstead and colleague Gabriel Nistor developed a technique for prompting human embryonic stem cells to develop into spinal cord cells. When those cells were injected into rats with spinal cord injuries, they could walk again. Other UC researchers see similar successes on the horizon.

"The field is very young, and this new generation of scientists are the ones who will reap the benefits of this research," said Arnold Kriegstein, head of UC San Francisco's Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research. "We're in a transformative moment."

Kriegstein studies how embryonic and the more developed progenitor nerve cells produce the structures of the nervous system. His work has applications in the treatment of brain disorders such as autism and Parkinson's disease. This exploration of the brain and nervous system is only one of the several research pipelines active at UC campuses. The work is breaking ground in both the understanding of disease and clinical treatments.

At UC Merced, tissue engineer Kara McCloskey believes such research can produce an additional benefit for California in the form of new business ventures. McCloskey is working on what she calls a living cardiac patch that will enable damaged heart tissue to regenerate.

"The goal is to replace the need for heart transplants by repairing the damaged heart," said McCloskey. "I think we can develop the technology in five to 10 years."

The number of people who stand to benefit range from those in need of heart transplants to the thousands of patients who suffer heart attacks each year and face a lifetime of medication and impaired physical activity. The heart lacks the ability to heal its own scar tissue effectively after it's been damaged, and scarred tissue can't handle the workload of the heart. The result is congestive heart failure.

McCloskey is focusing on developing functional progenitor heart cells and finding a way to deliver the "cardiac patch" to a living heart.

"We still have a lot of the rejection factors we face with organ transplants," McCloskey said. "How do we get the host body to accept the donor cells? It involves tricking the body into accepting foreign cells."

If her efforts prove viable, McCloskey said, startup companies - maybe even one she founds herself - could license the technology to manufacture the patches.

California's pioneering effort

UC's research effort is thriving thanks to California voter approval of Proposition 71 in 2004. That ballot measure created the California Institute for Regenerative Medicine and a $3 billion pot of research funding aimed at finding cures for dozens of fatal and debilitating diseases and chronic conditions.

Collectively, the University of California's 10 campuses have received more grants from the California Institute of Regenerative Medicine than any other university or private research center in the state. UC has received a little more than half of the number of grants dispersed and the dollar amounts given out. That money is funding the building of new labs, hiring new faculty and developing technology and tools to further the understanding of how stem cells develop into different human organs. Learn more about stem cells.

UC researchers are anticipating their efforts will take another major leap forward when President Barack Obama makes good on his promise to lift the federal restrictions on stem cell research. In 2001, former President George W. Bush issued guidelines that prohibited the use of federal funds for any stem cell lines not on an approved list. Those restrictions have hampered research, said Kriegstein, head of UC San Francisco's stem cell research center, one of the largest in the nation.

"I think there has been an opportunity lost for the last eight years," he said. "That's time lost that will be difficult to regain."

The federal restrictions have cost research centers in California both time and money. Research funded with National Institutes of Health grants has had to be strictly segregated from the work being done using new stem cell lines not on the approved list. Researchers have to work in separate labs or even buildings. That often means having to spend precious grant money on buying duplicates of expensive equipment.

"You have to be careful you don't pick up even a pipette bought with NIH money," said Jan Nolta, director of the UC Davis Stem Cell Program.

If federal restrictions are lifted, she said, California researchers will be able to collaborate with more U.S. researchers in states that only have had access to the federally approved cell lines, which many researchers believe are not as potent as newly created cell lines. She's hoping more federal funding will start flowing as well.

"The ability to get NIH funding is dwindling," she said. "That completely leaves out junior investigators. We're terrified we're losing a generation of young researchers."

Training future researchers

Another important component of UC's stem cell research has been the CIRM-funded training programs. Eight UC campuses have formal programs to introduce students, postdoctoral and clinical fellows to the field of stem cell research. The training program is set to expand, Nolta said, by establishing an internship program with UC researchers for California State University students.

UC Davis has established a Good Manufacturing Practice Lab to produce cell therapy materials to use in clinical trials. That facility will offer training certificates, Nolta said, for students who want to work in the clinical trials and startup companies set to grow out of the research discoveries emerging today.