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  • Microscopic images from the California Institute for Regenerative Medicine

    Two neurospheres derived from human embryonic stem cells, as they begin forming connections.

  • Microscopic images from the California Institute for Regenerative Medicine

    Retinal pigment epithelial cells derived from human embryonic stem cells.

  • Microscopic images from the California Institute for Regenerative Medicine

    Mouse neonatal heart cells.

  • Microscopic images from the California Institute for Regenerative Medicine

    Human embryonic stem cells in various stages of differentiation into neurons.

  • California Institute for Regenerative Medicine file, 2008

    This microscopic image shows a neurosphere, a ball of human embryonic stem cells giving rise to nerve cells. The miracle of stem cells, as discovered in 1998, is their ability to grow into any cell type. Stem cell experiments in mice have had impressive results.

  • Alan Trounson, president of the state’s stem cell agency, expects some projects will succeed in the next five years.

  • David Lesher is a veteran journalist in Sacramento who works at the Public Policy Institute of California.

The Conversation: These cells could save lives. But when? And at what cost?

Published: Sunday, Feb. 12, 2012 - 12:00 am | Page 1E
Last Modified: Sunday, Feb. 12, 2012 - 11:57 am

Should California maintain its leadership in stem cell research and continue funding the California Institute for Regenerative Medicine? To comment, please use our comments section at the end of the story or go our facebook page at www.facebook.com/sacramentobee.

In the future, scientists tell us, stem cells could make the blind see again, help the crippled to walk again and perhaps cure some of our most debilitating diseases like cancer, Alzheimer's and AIDS.

It sounds like science fiction. Doctors wouldn't just manage your damaged or deteriorating body parts, as they largely do today. They could actually repair or replace them, perhaps using your own cells. Hundreds of billions of dollars in annual medical care is at stake as well as millions of lives that might be saved or vastly improved.

The good news is that it's not science fiction. Just last month, the only human trial under way in the United States involving embryonic stem cells reported that eyesight was improved for two legally blind women. And in California, where voters approved a $3 billion bond to support stem cell science in 2004, the president of the state's stem cell agency said he is "optimistic" that at least a few California treatments will prove successful in humans in the next five years.

The bad news in the stem cell field is the growing uncertainty about money and the time it will take to complete a cautious and evolving regulatory process - even after "proof" in human trials is achieved.

Those who speculate say that the most advanced stem cell treatments are still probably a decade away from becoming available to patients. And the cost to get them there will far exceed California's $3 billion investment. The hope was that California's bond would jump-start a biotech industry by building the laboratories and seeding early research to a point where private support would take over.

But that point of commercial viability is a moving target as private investors have grown more risk averse and the regulatory path for such radical new therapies is unpredictable. So the biggest question today in the stem cell field is not whether the science will work someday. The big questions are how will we pay for it, how will regulators know when it's ready and when will it happen?

"We are at a time when venture capital doesn't invest as early as it used to," said Larry Goldstein, a leading stem cell scientist at UC San Diego. "So the public has to do it. You may not like the system, but that's the system."

Alan Trounson, president of the state's stem cell agency, adds that the "astronomical" cost of getting treatments through the regulatory process will diminish opportunities if the system isn't changed. But even with the challenges, he told me last week, he expects at least a few California projects will prove successful in humans within the next five years.

That may mean a genetically modified stem cell treatment to cure AIDS, he said; it may mean a treatment that eliminates the need for some diabetics to monitor or inject insulin; there might be a treatment to restore eyesight to those suffering from a major cause of blindness.

"These are the kind of things we need to get through," he said. "I hope that we have a number of them showing proof by 2015 or 2016. I'm optimistic. The caveat is that nothing is guaranteed."

The miracle of stem cells, as discovered in 1998, is their ability to grow into any cell type. Goldstein said you can see their potential in his area of study, Alzheimer's disease. In California, a 2009 study predicted that Alzheimer's cases will nearly double by 2030 and the cost to employers and the state could soar to $100 billion a year.

Meanwhile, stem cell experiments in mice are impressive. After stem cell treatments, elderly mice with severe memory loss have restored their ability to navigate a maze that was long forgotten. Autopsies on the mice found their brains still clogged with plaque and other signs of Alzheimer's, but the stem cells had rewired the brain's learning center, the hippocampus.

The most successful stem cell experiment so far is one that Californians might remember because it gained attention when Proposition 71 authorized the stem cell bond in 2004. Back then, as "60 Minutes" later reported, a paralyzed rat injected with a stem cell treatment was able to walk again. And Christopher Reeve, the late actor who was paralyzed in a horse riding accident, appeared in campaign commercials to tell voters, "You know, stem cells have already cured paralysis in animals."

In 2009, the spinal cord experiment was the first treatment involving human embryonic stem cells to be approved for human trials. Since October 2010, five patients have received the stem cell treatment, the most recent being a 23-year-old woman from Pleasanton who was paralyzed in a car accident in November.

Today, that experiment is the leading example of the challenges facing stem cell science because the sponsor of the project, Geron Corp. of Menlo Park, announced last November that it was getting out of the stem cell business and shutting down its landmark study on spinal cord injuries.

Early indications from the human trials have been positive, and Geron said that it was not withdrawing from the field because of doubts about the science. Instead, it attributed its decision to "uncertain economic conditions" and a review of cost, timelines and "clinical, manufacturing and regulatory complexities."

Geron and the state's stem cell agency - known as the California Institute for Regenerative Medicine - are working to find another sponsor for the spinal cord trial. But this setback came at a pivotal time for the agency. About half of the bond funds have been spent and there is considerable discussion today about how to spend the second half.

In its early years, the agency spent time and money to build infrastructure for this nascent science, successfully completing 12 laboratories and recruiting more than 100 scientists, many from out of state. That early investment has been credited with establishing California as an international leader in stem cell science and for developing an infrastructure that will continue to benefit the state.

But Proposition 71 also made some pretty bold boasts about the achievements the initiative would accomplish within 10 years. For the agency, that clock started ticking in 2007 when a court challenge was resolved and bonds were sold. Now the agency's board is focused on work that is most likely to produce the quickest result.

In August, the board of directors heard a presentation about the new "Translational Portfolio" - a list of 43 experiments considered the best hopes for a breakthrough. The projects target 26 different conditions including sickle cell, diabetes, Parkinson's, Huntington's, Alzheimer's, autism, damaged heart tissue and AIDS.

"This is what the taxpayers are waiting to see," Sherry Lansing, a member of the board, said after the presentation. "You keep reading these articles - 'Where are the results? Where is the beef?' Now I think we can really answer that."

The Translational Portfolio is a fascinating look into the future and what a new era in medicine might look like someday. It uses technologies that are just a few years old, like the ability to map a patient's DNA and target specific genes, or to take an adult cell back to its formative state and reprogram it to avoid a previous malfunction.

The problem is that even the most advanced experiments in the Translational Portfolio are still a couple of years away from the same point in the regulatory pipeline where high cost and uncertainty forced Geron out of the field. And there is still no clear answer about how to resolve those same challenges, although the cost-benefit calculation will be different for other treatments.

Trounson said he is optimistic some experiments will move further than the Geron project and be recognized as successful human treatments before the agency's 10-year deadline in 2017. In a discussion in October, though, some board members had doubts and they discussed the idea of asking voters to approve another bond. But with a state budget crisis and no major scientific breakthrough to brag about, that idea was set aside.

This spring, the board will prepare a strategic plan that will explore alternate funding sources and other plans to either maintain the agency's operations or to phase it out with a successful conclusion. Stakes are high, though, and some board members worried openly about a "cliff," where the agency shuts down abruptly, jeopardizing projects under way and risking the criticism of a "squandered" investment.

For California voters, it should not be a surprise that their stem cell investment was risky. When it passed by a whopping 19-percentage-point margin the topic was a raging national controversy, the state budget was in deficit and, as a state report said later, "Nothing like it had ever been attempted before."

But the stem cell vote also reflects an essential truth about California. For all of its diversity, the state has always been defined by newcomers who - compared to the nation - are optimistic about the future and willing to take risks. It's a thread that runs from the Gold Rush to Silicon Valley.

Four years after Proposition 71, California voters did it again when they passed a $10 billion bond to pay for high-speed rail. Ironically, that entrepreneurial risk will also be debated this spring now that the project's estimated cost has risen to about $100 billion.

Unlike high-speed rail, which continues to have strong support from the governor, the stakes surrounding California's stem cell investment have been largely invisible. That's too bad, because stem cell science is a much smaller investment for taxpayers with a greater possible return.

California's voter-entrepreneurs need to know that. It was pretty audacious of them in 2004 to try to create another economic driver like Silicon Valley and save lives at the same time. What's remarkable today is not that there is still risk or challenge; it's that the audacious vision is still possible.

© Copyright The Sacramento Bee. All rights reserved.

Read more articles by David Lesher



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