Candidate Barack Obama promised to lift Bush Administration restrictions on embryonic stem cell research. Two days after he became President Obama, the government gave its approval for the first-ever human trials using therapies derived from this controversial area of science.
Geron, a Menlo Park, Calif.-based biotechnology firm, was given the go-ahead by the Food and Drug Administration Thursday. While officials at both the company and the agency are calling the timing of the announcement coincidental, the news reignites the debate between those who see the promise of miracle-like cures in human stem cells and opponents who say the harvesting of cells from embryos is akin to murder.
Geron will choose 10 patients who have suffered spinal cord injuries resulting in paralysis. The patients will be given the treatments between 7 to 14 days after being injured, and will be studied for up to a year. If the patients remain safe, then Geron will ask for permission to expand the study and increase the dosage.
“This marks the beginning of what is potentially a new chapter in medical therapeutics, one that reaches beyond pills to a new level of healing: the restoration of organ and tissue function achieved by the injection of healthy replacement cells,” Geron president and CEO Dr. Thomas Okarma said.
A Shift in Research Priorities
“It’s a bold step, and the hard part of this is going to be managing expectations,” Chuck Murry, Ph.D., codirector of the University of Washington’s Institute for Stem Cell and Regenerative Medicine, told TechNewsWorld. “People have very high hopes for stem cells to do something remarkable. We have to keep in mind — the wonder with working with embryonic stem cells is that they can turn into anything, and the hardest thing with working with stem cells is that they can turn into anything.”
Making sure the embryonic cells turn into targeted, healthy spinal cord cells and don’t become disorganized rogue cells will be the focus of the trials. “Based on what we know, there’s very good reason to think this can be done safely,” Murry said.
Murry knows some of Geron’s researchers and has asked them about the timing of the approval. Last spring Geron submitted a 21,000-page application for testing, “which may be the largest in the history of the FDA,” he said. “It seems more than coincidental, but they assured me it was just coincidence. This [approval] was going to come either way, it just happened to be this week.”
The arrival of the Obama administration is rekindling enthusiasm among researchers, who have seen countries like Israel, Italy, South Korea and the UK take the lead over the U.S. in embryonic stem cell science since August 2001, when then-President George W. Bush allowed limited testing of existing cell lines while placing restrictions on further research. The research community is aware that the political and ethical landscape remains filled with potential obstacles — Congress must still weigh in — and a new hurdle has arisen since 2001: a weak economy and its impact on federal funding, Murry said.
“We don’t have our heads in the sand,” Murry said. “Everybody thinks we’re going to get expanded permission, but will there be resources to do so? In order to make up for lost time, will resources be made available?”
The Difference Between Ethics and Safety
The Bush administration’s policies may have resulted in federal researchers leaving the field for private or overseas work, while states like California, New York and Massachusetts kept up the scientific pace, said Summer Johnson, executive managing editor of the American Journal of Bioethics. “What we’ve seen now announced is that stem cells are still moving forward and the private sector is doing well,” she told TechNewsWorld. “I think even with the polices and the brain drain that we saw over the last eight years, companies like Geron were still able to have some progress.”
Johnson reminds those who are excited about Obama’s campaign promises that he recently said he may leave it to Congress to lift the previous administration’s restrictions. And there is also the question of whether or not the research community has thought through the bioethics issues involved, now that stem cell research is back on the agenda.
“Safety is not the same thing as bioethics,” she said. “There’s simply more to it. It’s making sure that we’re picking the right diseases for studying stem cell clinical trials, the right patient population, making sure once we’ve picked the right patients that we’re telling them all the information they need to know. Once we’ve enrolled people, are we gathering data appropriately, and if there are adverse events, are we disclosing that information?
“If we’ve learned anything from gene therapy experimentation and other things we’ve done in human research, it’s that a momentary pause sometimes makes the difference between research going well and research going not so well.”
That takes the point back to managing expectations. The patients selected for Geron’s trials will have suffered the worst kinds of spinal cord transection injuries, Murry said. “These are the most difficult patients to help. Usually in animal models where these cells have been shown to be effective, the injuries were not the severest, not through-and-through transections. You always start where you do the least harm, but sometimes you also end up starting where you do the least good. It wouldn’t surprise me if the initial study would show modest benefits.”