Published Jan 1, 2007 8:00 AM

13. State of Inquiry: Stem Cells

By Dan Gordon '85

Three decades ago, UC researchers opened a new frontier in the life sciences and created the biotechnology industry. For many scientists, there is a sense that history is repeating itself in stem cell research.

This new frontier, heavier in politics, morality, and heady talk of cures for dozens of diseases, is arguably more monumental than the first. Although federal law limits stem cell research at the national level, states and universities across the U.S. are rushing to create stem cell centers or pass laws to capitalize on an expected biotech windfall. Once again, California is betting on its university system to keep it out front in that race.

Last August, for example, top state politicians, including Gov. Arnold Schwarzenegger, Sen. Dianne Feinstein and Los Angeles Mayor Antonio Villaraigosa '77, chose the UCLA campus to announce a bipartisan drive to block the U.S. Senate bill that would place new limits on human embryonic stem cell (HESC) research.

"The study of stem cells has created a whole new area of biology that bridges people from many different fields," says Harley Kornblum, director of UCLA's Neural Stem Cell Research Center. "With very good scientists working together on a problem in which there is so much potential, a lot of things are going to come out of this that we don't even imagine yet."

In 2004 the state passed Proposition 71, providing $3 billion in funding to create the California Institute for Regenerative Medicine (CIRM), a new state agency that would award grants and loans to support stem cell science at institutions across the state — including research on new HESC lines. Lawsuits challenging the constitutionality of the CIRM have constrained government funding, but the initiative has generated substantial contributions from private donors. The favorable environment has also put the state's leading institutions at an advantage in the competition for the brightest young scientists.

UCLA, which in 2005 committed $20 million over five years to establish the cross-disciplinary Institute for Stem Cell Biology and Medicine, was able to land five highly sought-after stem cell researchers in its initial wave of recruiting in October. The scientists, all under 40 years old, came from such respected East Coast institutions as Harvard, MIT and Johns Hopkins University.

When the new faculty was announced, Owen Witte, the UCLA institute's director, noted that "These scientists [all had] multiple offers from other institutions and could have gone just about anywhere."

While adult stem cells have been studied and used clinically for many years, access to HESC lines has been restricted by presidential order for almost all researchers receiving federal funds. Scientists argue that HESC's unique potential to regenerate and form into any of the body's 200 tissues might one day lead to revolutionary new approaches to treating diseases that have baffled them for decades. Already, a company in Menlo Park, Calif., working with UC Irvine scientist Hans Keirstead, hopes to begin a clinical trial in 2007 on an HESC-based treatment for spinal cord injury.

But scientists caution that when it comes to using HESCs to repair diseased tissues, researchers are still learning the fundamentals, including how to grow cells that will remain unspecialized while replicating, and what signals cause HESCs to become specialized cells. They must learn how to deliver the cells to the desired place in the body. They must learn how to protect them against an attack by the body's immune system — and ensure that the cells won't continue to reproduce unchecked, promoting cancer.

There is another opening created by the new science, and it appears to be much less daunting. HESC research could be used to establish laboratory models able to reveal the root causes of diseases by recapitulating their processes under the microscope. Following tried-and-true methods of discovery, new drugs based on these observations could be screened in cultures with far greater efficiency for their potential utility in humans.

"That's a more traditional match for the pharmaceutical industry, and if I were to speculate, that will occupy the next decades of work with these cells," says Randy Schekman '71, campus program director of UC Berkeley's Stem Cell Center. And, like so many others in the stem cell story, he has a personal stake: His wife has Parkinson's.

"We all have family and friends who suffer from these diseases," Schekman says. "Now, we have the prospect of not just palliative care ... but cures."