Jan. 26, 2009 -- As the dust settles from the U.S. Food and Drug Administration's approval of the first-ever study of a treatment based on human embryonic stem cells, researchers are now assessing what this trial may mean for the years to come in stem cell research -- and how the politics of the past decade may have damaged their progress.
The study, for which California based Geron Corp. won FDA approval on Friday, will examine the potential of an embryonic stem cell treatment in fixing severe spinal cord injuries in humans.
For proponents of stem cell research, the double impact of the first-of-its-kind trial and an administration that appears open to exploiting the potential of embryonic stem cells is a promising sign that progress is finally on its way.
"I am in favor of anything that will bring us closer to a cure for diseases like Alzheimer's and diabetes," said former first lady Nancy Reagan in a statement issued Friday in response to news of the study. Reagan emerged as a prominent supporter of stem cell research after her husband, President Ronald Reagan, passed away in 2004 after a 10-year battle with Alzheimer's disease.
"I am very pleased to hear that human trials of embryonic stem cell therapy will begin soon and am very hopeful that it will be successful so that further trials can move forward," the statement reads.
And where proponents were hopeful, some stem cells researchers were ecstatic. One such researcher is Dr. Robert Lanza of the Institute for Regenerative Medicine at Wake Forest University School of Medicine. Lanza is also chief scientific officer for Advanced Cell Technology, which is planning its own FDA-approved study this summer which would test a technique using embryonic stem cells to prevent blindness.
"This is what we've all been waiting for," Lanza said. "It has been over a decade since embryonic stem cells were first discovered; this sends a message that we're ready at last to start helping people."
Still, others are more cautious in their appraisal. Dr. George Daley, immediate past president of the International Society for Stem Cell Research, paradoxically termed the approval "[a] huge first step, but only a tiny one."
And Lorraine Iacovitti, interim director of the Farber Institute for Neurosciences at Thomas Jefferson University Medical College, said much work remains in order to make up for lost time.
"It will of course require a significant infusion of new federal money into the field to attract back many researchers who were frightened away over the last eight years under President Bush," Iacovitti said. "Despite the difficulties during that period, much progress was made understanding which stem cells work, how they work and in what ways they can be modified to improve their therapeutic potential."
Did Obama Win Influence Approval?
Naomi Kleitman, director of the Extramural Research Program for the National Institutes of Health, told ABC News that Obama's presidential victory had nothing to do with the FDA approval of Geron's trial. But most researchers agree that the presence of the Obama administration is a welcome sign for a field of research that after a decade and a half of political roadblocks is finally beginning to hit its stride.
Embryonic stem cell research has been a field plagued by a dearth of federal funds since the mid-1990s, when President Bill Clinton made the first executive branch move to block federal funding for the creation of embryos for stem cell research. In August 2001, President George W. Bush strengthened the ban on federal funding dramatically by barring federal funds for research on all but a few existing embryonic stem cell lines.
What has traditionally made embryonic stem cells such a hot-button issue is the fact that, in order to obtain them, researchers must destroy human embryos -- a step that some say violates the sanctity of human life.
But Geron's research uses discarded embryos from in-vitro fertilization procedures, which in all likelihood would have been destroyed anyway.
Ruth Macklin, a professor of bioethics at the Albert Einstein College of Medicine, said that the influx of federal funds through the NIH will also come attached with rigorous ethical oversight, as it the case with other federally funded research.
"With a more favorable funding environment -- meaning, specifically, NIH funding -- stem cell research can proceed more expeditiously and more efficiently," Macklin said. "It will be more efficient since there will be no need to engage in elaborate ways to segregate stem cell research from federally funded research within research institutions, as has been the case under the Bush ban."
The Risks of First Steps
But with so much riding on the success of this first step, some wonder if the spinal cord trial puts the best foot forward. Even Lanza's optimism is tempered by the possibility that such a trial will go wrong, an outcome he said would be "a disaster." And Daley noted, "There is some controversy regarding whether spinal cord injury is the appropriate patient population to test the first embryonic stem cell products; some feel only patients with fatal diseases should be treated."
Thoru Pederson, professor of biochemistry and molecular pharmacology at the University of Massachusetts Medical School, countered that "[researchers] have to start clinical evaluation somehow, after all."
But all agree that a setback at this point -- the death of an experimental subject or an unexpected negative side effect -- could do more to hurt the beleaguered field of research than even the eight-year stranglehold on federal funds. Bryon Petersen, associate professor in the department of pathology at the University of Florida, terms the trial a "hail Mary pass" that he feels may be destined for disaster.
"Pushing the envelope of medicine is what we do, but we have to be very cautious," he warned. "There are new therapies waiting to be discovered, but if this trial fails in a big way it will push the field 10 years back."