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Scientists Hope to Renew Stem Cell Advances

January 29, 2009 at 6:30 PM EDT
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Stem cell scientists anticipate a loosening of federal research funding restrictions under the Obama administration despite otherwise shrinking budgets. Spencer Michels reports on where stem cell research stands and how scientists think it may change in the coming years.

JIM LEHRER: Now, the changing science and politics of stem-cell research. President Obama has said he will soon lift restrictions on using federal funds for such work; that would reverse the Bush administration policy.

NewsHour correspondent Spencer Michels has our Science Unit report.

SPENCER MICHELS, NewsHour correspondent: Three days into the new administration, the Geron Corporation got permission from the Food and Drug Administration to start human trials using stem cells.

THOMAS OKARMA, CEO, Geron: This is the world’s first attempt to use embryonic stem cells in people.

SPENCER MICHELS: Thomas Okarma, Geron’s CEO, said the embryonic stem cells will be used to try to repair damage to the spinal cord. Stem cells can turn into mature, specialized cells like spinal cord, or heart, or cartilage, and can replace diseased or injured ones.

In this animated video, Geron shows how it already used stem cells with rats.

THOMAS OKARMA: We take the animal under anesthesia and we open up the spinal cord. And we give the animal a very precise damage to the cord that resembles the damage in a automobile accident.

Then, within two weeks of the injury, we inject the human cells that we make from embryonic stem cells and, after several weeks, we see dramatic and sustained improvement in the animal’s locomotion, its body control, its paw placement, and that improvement is permanent.

Restrictions devastated research

SPENCER MICHELS: Okarma says he believes the FDA took a long time to approve the company's 22,500-page application to be sure the clinical trials would be safe, and not for political reasons.

But stem cells have been at the heart of a longtime political dispute.

FORMER U.S. PRESIDENT GEORGE W. BUSH: I have concluded that we should allow federal funds to be used...

SPENCER MICHELS: In 2001, the Bush administration decreed that only a few pre-existing lines of embryonic stem cells could be used for research by those who received federal funds. Although Okarma was using one of those approved lines, he still says the restrictions have had a disastrous effect on research.

THOMAS OKARMA: Well, the impact of the Bush restrictions are deeper and broader than most people realize. It has delayed investment by industry and Wall Street. It's delayed the clinical development of this technology. It's created a chaotic state-by-state policy difference.

SPENCER MICHELS: In fact, in labs receiving federal funding and in many universities throughout the country, research with embryonic stem cells came to a halt.

But eight states managed to move ahead by providing their own funding. The biggest spender was California. In 2004, voters approved Proposition 71, $3 billion in state-issued bonds to support stem-cell research.

The money attracted hundreds of scientists to the state, who pushed ahead with basic research in both adult and embryonic stem cells.

Alan Trounson now heads the California Institute for Regenerative Medicine, or CIRM, which was set up to manage the funds. He thinks stem-cell research has moved ahead surprisingly fast, despite the federal restrictions or perhaps because of them.

ALAN TROUNSON, California Institute for Regenerative Medicine: I actually don't see that, in the end, that the administration had such a strong negative effect on the field. In fact, I think it might have been the reverse. If there's good medicine to be had, then you will overcome negative politics.

Prioritizing cures for patients

SPENCER MICHELS: The way forward has not been totally smooth sailing, as different priorities had to get ironed out. The institute began by funding research tools like new labs and basic research. That brought criticism that curing patients was not a high enough priority.

Okarma and others claimed CIRM was spending hardly any money on finding drugs and therapies, so-called translational research.

THOMAS OKARMA: What money has been dispersed by CIRM in California, by other state institutions, and by the NIH has predominantly gone to basic science. And there's nothing wrong with that. But what's absent is any significant cash flow to projects that are translational, that take a cell type and enable it to be used in a clinical trial.

SPENCER MICHELS: Now the California institute has started prodding researchers to develop practical applications. It awarded cell biologist Tamara Alliston a preliminary $50,000 grant to study how damaged cartilage in a knee can be replaced or repaired by stem cells.

TAMARA ALLISTON, University of California, San Francisco: Somehow the body knows how to make cartilage at the right place at the right time in the right shape. We're taking advantage of some of the same signals in the physical environment and some of the chemical signals that are present and applying those same signals to the stem cells. And so already we've gotten really good results.

Hope for new therapies

SPENCER MICHELS: Next, she hopes to get a larger grant and try stem cells on goats and, within four years, on humans.

Alliston and other members of her team at the University of California, like orthopedic surgeon Dr. Hubert Kim, think that the process of turning basic stem-cell research into useful therapy will be spurred on by a new federal stem-cell policy.

DR. HUBERT KIM, University of California, San Francisco: We don't want this research going on in an ivory tower and never see the light of day in terms of helping patients.

SPENCER MICHELS: One of Kim's patients is 15-year-old high school athlete Nathan Cipres, who could benefit from stem-cell therapy, if it becomes a reality. The cartilage in his knee peeled off from the joint surface, causing acute pain.

NATHAN CIPRES: For about three years, it was really hard to play, because it hurt a lot and nothing helped it.

SPENCER MICHELS: Kim says traditional treatments, re-attaching the cartilage with screws, are slow.

DR. HUBERT KIM: Patients like Nathan often don't heal these injuries, and we need better treatments for these kids.

I have to be able to trust you to back off if your knee starts bugging you again, OK, because you know how seriousness this is, right? If that piece breaks off, and we're in kind of a world of hurt, then you will need this stem cell stuff.

SPENCER MICHELS: So what's ahead now for California? David Jensen thinks the state will suffer. He writes the online California Stem Cell Report, and he says the fact that California has committed so much state money may actually discourage federal funding.

DAVID JENSEN, California Stem Cell Report: When the Obama administration looks around and, in light of this economic crisis, and they see all these demands for all these additional funds for various kinds of scientific research, all extremely worthy, and they say, "Well, we're going to fund the areas that need money." And, you know, California is filling a big need. Do we need to do that?

SPENCER MICHELS: Arnold Kriegstein, who heads stem-cell research at the University of California at San Francisco, foresees more money being poured into research despite shrinking budgets.

DR. ARNOLD KRIEGSTEIN, University of California, San Francisco: Well, I think there's a kind of perfect storm, as it were. There's a leverage of the dollars that have been supported here in the state by public subscription through Prop. 71 and the relaxation of the federal policy.

New rules must be written

SPENCER MICHELS: The California Institute of Regenerative Medicine says that, even without a lot of new federal money, a new Obama policy allowing federal funding will spur advances. Private industry may provide funding, as well, since the research has advanced to the point where human therapy is near.

Meanwhile, the debate over the ethics of stem-cell research continues. Religious groups who have opposed the use of embryonic stem cells in the past are upset about any change.

And on the eve of President Obama's inauguration, the U.S. Conference of Catholic Bishops wrote to him warning that reversing Bush policies on stem cells "could introduce significant negative and divisive factors into our national life at a time when we need to come together to address the serious challenges facing our people."

Scientists like Kriegstein acknowledge those concerns could still impact federal policy.

DR. ARNOLD KRIEGSTEIN: People still have deeply held feelings. Not everybody agrees with the sort of use of these stem cells for research that we're doing here and elsewhere. But I think the public has come to realize that the long-term benefit really is on the side of science.

SPENCER MICHELS: He also has a warning for those who think stem-cell research, like that the FDA approved, will have instant results.

DR. ARNOLD KRIEGSTEIN: I think that in stem-cell research especially, there's a risk to rushing too quickly into therapy or into the clinic. Obviously, that's our goal, but I think the public has to be patient, because science is unpredictable, and it usually takes a lot longer than most of us would like.

SPENCER MICHELS: No matter what the Obama administration recommends, it still will be months before any new federally funded research can proceed, as new rules and regulations have to be written.