By Rob Stein
Washington Post Staff Writer
Tuesday, October 12, 2010; A2
Doctors in Atlanta are monitoring the first patient to receive an injection of millions of human embryonic stem cells in a government-sanctioned attempt to test the promising but ethically fraught therapy.
Surgeons at the Shepherd Center, which specializes in injuries to the spinal cord and brain, infused the laboratory-grown cells into the patient's damaged spine on Friday, the hospital and Geron Corp., which is sponsoring the research, announced on Monday.
A team of doctors planned to test the patient to look for safety problems as well as any indications that the treatment is helping to restore sensation and movement, officials said. They stressed that it would be some time before any results are released. No additional details about the patient were made public.
The highly anticipated step was welcomed by scientists who are eager to move the research from the laboratory to the clinic. Human embryonic stem cells have been tested in animals, and clinics in other countries claim to offer therapies using the cells. But the trial is the first vetted by any government entity to try to evaluate the approach. After repeated delays, the Food and Drug Administration gave the go-ahead in July.
"This is a huge milestone for the field," said George Q. Daley, a Harvard Medical School stem cell researcher who is not involved in the study. "But we must be cautious. This is only the first attempt and we should anticipate lots of failures before we learn how to deliver cells safely and effectively. I wish this brave patient well," he added.
The development, however, was criticized by those with moral objections to research using the cells because days-old embryos are destroyed to obtain them.
"Geron is helping their stock price, not science and especially not patients," said David Prentice, senior fellow for life sciences at the Family Research Council.
The study is also raising alarm among many proponents of the research. Some argue that the experiment is premature; others question whether it is ethical, and many fear that the trials risk disaster for the field should something go awry. If patients are hurt by the cells - or even if there is no hint that the cells help - it could be devastating to the field, they argue.
"Everybody is just holding their breath and hoping there are no bad outcomes," said Evan Y. Snyder, director of the stem cell program at the Sanford-Burnham Medical Research Institute in La Jolla, Calif.
Supporters of the privately funded research are confident that it has been adequately vetted. The FDA has demanded extensive experiments in the laboratory and on animals to provide evidence that the cells hold promise and are safe to test in people.
"This accomplishment results from extensive research and development and a succession of inventive steps," Thomas B. Okarma, Geron's president and chief executive, said in a statement.
Safety worries - most prominently fears that the cells could cause tumors - prompted the FDA to demand additional data, including, most recently, assurance that cysts that developed in mice injected with the cells posed no threat.
Although Geron eventually hopes to test the cells for many medical problems, the first trial is being conducted at seven sites and will involve only 10 patients who were partially paralyzed by a spinal cord injury in the previous one to two weeks.
Surgeons injected the first patient with about 2 million "oligodendrocyte progenitor cells," created from embryonic stem cells in the hopes that the cells will form a restorative coating around the damaged spinal cord. In tests in hundreds of rats, partially paralyzed animals walked. The human study is design primarily to find out whether the therapy is safe, but doctors will look for evidence that it helps patients recover.
Spinal cord injuries, however, are highly unpredictable. Patients often improve on their own, and that complicates trying to gauge whether the cells had any effect. Some also wonder whether trauma victims who have so recently suffered a life-altering injury will agree to the experiments out of desperation without fully understanding the risks. There is also concern that the therapy may worsen the patients' conditions.
But company officials said they are confident. Even if problems occur, research shows that the cells do not leave the site of the injury, indicating that patients would not experience any negative effects, they said. Each subject also will be assigned an independent advocate to try to make sure that volunteers fully understand their decisions.
"There are a lot of people who want to do everything they can to get better," said Daniel Heumann, a paraplegic who serves on the board of the Christopher and Dana Reeve Foundation. "God bless them if they want to take the risk to do this. I give them all the respect in the world. Without risk, there is no reward."
The announcement comes as the future of federal funding for embryonic stem cell research remains in doubt. A federal judge ruled in August that the Obama administration's more permissive policy for funding the research violates a federal law that prohibits taxpayer money being used for research that involves the destruction of human embryos. The Justice Department is appealing the decision.