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A new day for stem cell research

March 9, 2009


Human embryonic stem cells.

President Obama has lifted restrictions on human embryonic stem cell research.

Photo: James Dutton, Stem Cell Institute

Obama's action opens possibilities for University's Stem Cell Institute

By Deane Morrison

On March 9, 2009, President Obama lifted former President Bush's executive order of August 9, 2001 restricting federal funding of human embryonic stem cell (hESC) research.

Researchers at the University of Minnesota Stem Cell Institute—the world's first institution devoted to stem cell research—welcomed the news, which comes as the institute prepares to celebrate its 10th anniversary this year.

"In the USA, it's good news for anybody involved in stem cell research," says Jonathan Slack, director of the institute. In Minnesota, he says, "we've always believed it's important to work on both embryonic and adult stem cells. We don't think any line of research should be closed down arbitrarily.

"We firmly believe this type of research should happen in public research universities with the transparency and oversight that comes with federal funding."

Obama's action opens wide the door to studying the use of hESCs to treat heart disease, neurodegenerative conditions, arthritis, cancer, and other conditions where the body's original cells are wearing out or, as in cancer, tumor growth has wreaked mechanical damage on cells and tissues, Slack adds.

University researchers will now have access to numerous lines of human embryonic stem cells that previously could only be studied using strictly private funding—a requirement that was often prohibitively difficult. The action also allows the Stem Cell Institute to supply researchers nationwide with hESC lines derived with private support after the 2001 executive order.

The backdrop

Embryonic stem cells are valued because they have the potential to give rise to virtually any type of cell or tissue found in adults. Human ESC cell lines are produced when cells are taken from the interior of embryos created, but not used, during in vitro fertilization procedures. The embryo is destroyed, but if the cells are successfully cultured they may give rise to lineages called hESC lines.

Since 1995 Congress, through the Dickey-Wicker Amendment, has forbidden the use of federal funds—which come mostly from the National Institutes of Health (NIH)—for any research in which human embryos are destroyed. In 1998 the Clinton administration allowed federal funds to be used for research on human ESCs, arguing that the cells were no longer embryos. On August 9, 2001, the Bush administration cut off federal research support except for those hESC lines derived before that date.

But of the 60 to 70 approved cell lines, only some 12 to 15 were actually available and usable, says Dan Kaufman, associate professor of medicine and associate director of the institute.

A high hurdle falls

Under the Bush administration, no one was prohibited from research on hESCs derived after August 2001, as long as the work was performed using private funds. But this restriction, says Slack, meant "a lot of red tape." For example, it's a rare medical research lab that has used NIH funds for neither equipment, nor supplies, nor staff. Even working in a building built with federal support raised sticky problems for those who wanted to use human ESCs other than the 60 approved lines.

"We firmly believe this type of research should happen in public research universities with the transparency and oversight that comes with federal funding."

Few institutions have met the challenge of separating federally and privately funded operations to the extent necessary—but the University of Minnesota has made it a priority by creating space dedicated to privately funded work within the Stem Cell Institute. Researcher Meri Firpo, an assistant professor of medicine, uses the space to derive hESC cell lines.

"The University of Minnesota has gone out of its way to allow me to have a nonfederally funded research lab," she says. "A lot of people don't have this kind of support."

With the lifting of restrictions, she'll be able to distribute the hESC lines she has developed to researchers elsewhere and collaborate with them on research. Her lines comprise both normal cells, which can be used to study basic human development, and cells containing genetic mutations associated with diseases, which may be used to model the diseases and design therapies.

She also studies the development of hESCs into pancreatic cells. Her goals: to gain a better understanding of such development and to generate cells for diabetes transplantation therapies.

For Kaufman's part, he is studying the development of normal blood cells and creating populations of "natural killer" white blood cells that specialize in attacking cancer cells or HIV. He has used hESCs from federally approved lines to make natural killer cells, but looks forward to having a wider choice of cells to work with.

"We want to make [natural killer] cells that are more efficient at killing tumors or at making cells that will be good at it," Kaufman says. "Now, in a pot of hESC cells, maybe one percent would lead to natural killer cells that kill, in particular, cancer or HIV. We'd like to get that to 10 percent. So we may use federal money to extend the work to new cell lines that may do a better job."

Researchers at the Stem Cell Institute also are studying hESCs as avenues to combating neurological and cardiovascular disease, among other conditions. Nationally, says Kaufman, clinical trials of at least three hESC-based therapies—for treating spinal cord injury, blindness, and diabetes—have been on hold awaiting FDA approval.

The action by Obama may already be benefiting the institute: Enrollment is booming in its NIH-funded course on hESCs. Taught every three months, it is for researchers who want to learn how to grow the cells.

"We allow five in the course. This time, so many applied that we're trying to accommodate eight," says Firpo. "More people are interested [in learning to do this research] and are coming here and learning about working [with hESCs] here."

"Lifting the restrictions "makes things, potentially, logistically easier for people like Meri and me and may attract people who weren't in the field before," Kaufman observes.

Despite the optimism over Obama's action, a loosening of purse strings is also necessary, says Kaufman.

"Congress will absolutely have to increase the [base] NIH budget," he says. "You can approve all the cell lines you want, but it won't do any good if the budget remains flat, as it has for four to five years." No one can predict for sure what Congress will do, but many of its members from both sides of the aisle are known to favor embryonic stem cell research.

As work on hESCs gears up, the University's Stem Cell Institute will continue to pursue other stem cell research. The institute has some of the world's pioneers in the use of adult stem cells, including those found in umbilical cord blood.

"We are also exploring using possibilities offered by a new approach called induced pluripotent stem cells, or IPS cells," says Slack. "[These] are skin cells that have been reprogrammed to go back to the original stem cell state. Then they can develop into new cells that might be used for damaged hearts or nerve cells or other diseases.

"As the nation's first established stem cell institute, we believe in exploring all pathways to scientific discovery. A lift on the stem cell restrictions is great for science."

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