Stem cell ruling puts researcher's project in limbo

(This story was originally published Aug. 31, and was updated Sept. 10.)

Joanna Wysocka

Joanna Wysocka, PhD, has had a spectacular year. The young Stanford University School of Medicine stem cell researcher published her research in one of the world’s most respected scientific journals, won a top award from an international stem cell society and received the highest of scores on a National Institutes of Health grant proposal that promises to help children with a rare but devastating developmental disorder.

But like many other Stanford researchers, her work has now been thrown into an uncertain limbo by an Aug. 23 injunction issued by U.S. District Judge Royce Lamberth suspending federal funding for human embryonic stem cell research. Although an appeals court on Sept. 9 placed a temporary stay on the injunction, it remains to be seen what the final outcome will be.

Nonetheless, Stanford law professor Hank Greely, director of the Stanford Center for Law and the Biosciences, called the stay a welcome development for those who support embryonic stem cell research as it allows NIH funding for such work to resume. “It is a good sign that the court of appeals’ action recognizes the urgency and importance of the case,” he said, while adding that a final decision may be months away.

Lamberth’s ruling had prompted strong criticism from scientists. Irving Weissman, MD, director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine, criticized the Aug. 23 ruling as “profound and disturbing,” and warned that the potential impact of the ruling could be “a massive halt to most embryonic stem cell research in the United States.” Indeed, the ruling continues to trouble other Stanford researchers worried about their ability to pursue this type of work going forward.

Wysocka’s work is primarily focused on CHARGE syndrome, a congenital disorder that affects one in 10,000 children and subjects them to a host of potentially life-threatening complications. Her initial work on the syndrome’s mechanism was impressive enough to be published in Nature, one of the world’s most prestigious science journals. This year she was also given the 2010 Outstanding Young Investigator Award by the International Society for Stem Cell Research.

For Wysocka, assistant professor of chemical and systems biology and of developmental biology, the injunction put in doubt NIH funding that seemed sure to come this year. She had applied for an RO1 grant from the NIH to continue the work, and was delighted when the NIH rated her proposed project as more worthy than 99 percent of the proposals received. “I was confident that the application would be funded,” she said.

Then she heard about the Lamberth ruling, and the trajectory of her skyrocketing research took a dive. Her initial work was funded by a seed grant from the California Institute for Regenerative Medicine, but that grant has ended. “I am currently funding this project largely from leftovers of my start-up funds and relatively unrestricted junior investigator awards, but we need more money to continue,” she said. While the latest ruling means that the NIH can fund grants like hers, there is uncertainty among researchers nationwide due to the unsettled state of the law.

Lamberth’s ruling arose from a challenge to the Obama administration guidelines, which in March 2009 expanded federal funding for research on human embryonic stem cells. Those guidelines replaced the Bush administration guidelines, which allowed federal funding of research on a more limited number of embryonic stem cell lines.

The Lamberth injunction rejects the Obama administration guidelines, but the judge’s reasoning — that any human embryonic stem cell research necessarily requires the destruction of human embryos and therefore contravenes existing law — also seems to make it illegal to use federal funding even for research on cell lines OK’d by the Bush administration.

Take, for instance, the work of Michael Snyder, PhD, professor and chair of genetics. His team studies the genes that transform human embryonic stem cells to mature nerves. He hopes the research will one day help cure neurodegenerative diseases such as Alzheimer’s and Parkinson’s. Prior to the Aug. 23 injunction, there was not much controversy about the tools his lab used.

“Our work uses standard, well-established cell lines that have been studied with federal dollars for quite some time,” Snyder said. The cell lines are among those approved by the Bush administration. But after Judge Lamberth’s injunction, Snyder was told by the NIH that an award he was supposed to be receiving for this research had to be withheld.

On Sept. 10, the day after the appeals court stayed the injunction, the NIH notified Snyder that his funding was back on track. Still, he worries about another reversal. “For us it would be a disaster to switch to other cell types — a waste of enormous resources that have already been invested in our work,” he said.

The Lamberth decision also affected  radiology professor Brian Rutt, PhD.  He is is in the initial stages of creating advanced imaging methods for stem cells, but now questions that course of inquiry. “I wouldn’t want to change my focus too quickly from conventional imaging research toward stem-cell-specific imaging if the prospects for future NIH funding are dim,” Rutt said.

Wysocka, too, is concerned. Although the NIH now can move forward on her grant, she’s not sure how fast it can act — and whether the next court ruling could deal another setback before that happens. She noted that the NIH committee that must give final approval for her funding met while Lamberth’s ruling was in effect, so it did not consider her grant. That committee meets only three times a year, she said, so now she may have to wait.

“The review process was disrupted — it’s not something that can change in a day,” she said. “I have no clue when my grant will get back on track.”

Writers Christopher Vaughan, Erin Digitale and Jonathan Rabinovitz contributed to this story.