Nobody likes a busy signal. And for U.S. stem-cell researchers, none has been more frustrating than the one on the National Institutes of Health's (NIH) Human Embryonic Stem Cell Registry home page. That's where the government agency lists all of the embryonic-stem-cell lines that scientists are allowed to study using taxpayer dollars. For months, the page has been depressingly static. "None are available at this time," it read. "Please check back later."
That message changed Dec. 2 at 12:30 p.m. ET, when the government finally made available the first 13 stem-cell lines that researchers can study with federal funds. Researchers had been awaiting the announcement since March 9, when President Obama signed an Executive Order lifting the ban that former President George W. Bush had placed on government support of human-embryonic-stem-cell research. The previous Administration had restricted federally funded studies to only the dozen or so stem-cell lines that had been created before Aug. 9, 2001. The new policy allows scientists to experiment with any existing stem-cell line, regardless of when it was created, as long it meets specific criteria showing it was derived in an ethically and scientifically responsible manner.
Already, the Federal Government has given out 31 grants totaling about $21 million for research involving the larger pool of human embryonic stem cells. But the recipients of that money have been waiting to use it since September, when the NIH, charged with establishing and applying the stem-cell vetting criteria, began reviewing potentially eligible cells. In addition to the 13 lines approved on Wednesday, another 96 lines are waiting for the green light, 20 of which may get it by Friday.
"It's exciting to be able to say that, after what clearly has been a time of frustration on the part of the scientific community over their inability to gain access to federally funded cell lines, that's now changing," Dr. Francis Collins, director of NIH, told reporters during a telephone briefing. "Because the vast majority of basic biomedical research that goes on in the U.S. is supported by NIH, the fact that researchers who are our grantees could not work on the new lines was seen by many people as a significant deterrent to rapid progress in the field."
Progress will certainly accelerate as more stem-cell lines are added to the government registry. A larger pool of available stem cells is a more accurate reflection not only of the diversity of the population but also of the variety of forms that treatable diseases can take. That translates into more opportunities for researchers to study basic human development and disease development, screen new drugs for their effectiveness against disease and create entirely new therapies. The ultimate goal is to use stem cells, which can morph into any of the body's hundreds of different cell types, to cure disease by repairing or even replacing damaged or defective cells.
Of the 13 newly approved lines, 11 came from the lab of Dr. George Daley, director of the Stem Cell Transplantation Program at Children's Hospital Boston and a member of the Harvard Stem Cell Institute; the other two came from Dr. Ali Brivanlou, an embryologist at Rockefeller University. Daley's submission for NIH review was 130 pages long, he says, including a 16-page informed-consent document signed by each of the donors of the embryos from which the stem-cell lines were derived, ensuring that the donors were aware of where their embryos were going and what they would be used for. "Our documents were very exhaustive," Daley says. "Probably too exhaustive. But I stand behind that process. I think it does need to be scrupulous and done with great care."
That kind of meticulousness probably helped expedite the NIH's approval of the new lines, a process that involves applying a checklist of criteria spelled out by the agency and providing documentation that the cells meet all of the requirements exactly. This review, says Collins, boils down to NIH staff agreeing that all the necessary criteria for inclusion have been met. Approval of some lines may be less straightforward if certain requirements have not been met to the letter. For instance, since stem-cell lines are drawn from unused embryos donated to research by couples undergoing the IVF procedure, researchers must offer proof that each couple was fully informed of all their options for discarding excess embryos. If the proper documentation doesn't exist, an NIH working group would have to determine whether the spirit of the requirement was met.
Some experts worry that the stringent vetting and documentation processes may place an undue burden on labs that have painstakingly created human-embryonic-stem-cell lines using their own hard-earned private funds. (Researchers are still prohibited from using federal money to create new stem-cell lines because of a congressional ban on harming or destroying embryos.) According to some estimates, as many as 780 such lines may exist worldwide, but not all labs may be willing to subject themselves to the scrutiny and administrative hassle of registering their lines with the NIH. Even among the handful of stem-cell lines that were eligible for federally funded study under President Bush, only one has so far been resubmitted for NIH review and inclusion in the government registry.
In many cases, researchers studying existing stem-cell lines do so free of any monetary strings, which means they are also entitled to any potential commercial windfall that may come from the application of the cells to a treatment or therapy. "Any discoveries they make using the lines will be theirs," says Amy Wilkerson, associate vice president for research support at Rockefeller University, who oversaw the submission of the university's lines.
But despite the relatively slow start for American stem-cell research, Rockefeller's Brivanlou is hopeful that the NIH approvals mark the beginning of a new era in our understanding of human development. "I consider it a shame that at the beginning of the 21st century, we know more about how development works in the worm, the fruit fly and the mouse than we know about our own development. And it's not because of scientific limitations or technological limitations," he says. "It would be nice if someday people are allowed to ask basic questions simply about where we come from as human beings. I'm optimistic that we are experiencing the first steps in the right direction."