The Niche

With NIH back in the game, CIRM leans toward translation

Cross-posted from The Great BeyondObama’s decision made scientists cheer, but raised a question for CIRM: how would its leaders make the case that the agency is still necessary – especially when the state is trying to crawl out of a fiscal crisis? The California voters created CIRM in 2004 in part because the federal government wasn’t funding much human embryonic stem cell research. But with the hands of the National Institutes of Health no longer tied, long-time critics have said CIRM should “adopt a secondary role to NIH” or even close shop entirely.

In response, CIRM is repositioning itself more firmly as a translational agency, preparing for NIH to take on some of the burden of funding basic research. At its 12 March board meeting, the agency reordered some of its programs and cut or delayed funding for others to ensure that the agency will have enough money to support its clinically focused disease team grants, which are expected to be awarded this fall.

CIRM will run out of money this September because the state’s fiscal crisis stopped bond funding from flowing to the agency. The state has authorized CIRM to sell up to $400 million in private bonds to fill the gap. The board members were asked to vote on recommendations prepared by CIRM staff on ways to cut back on expenses in anticipation that $100 million of these bonds will be sold this year. The board endorsed some of the CIRM staff’s recommendations, including funding one set of grants that it awarded in January – $17.5 million to train undergraduates and master’s level students to work with stem cells – and delaying funding for twelve months a second set of grants awarded in January that will give $40.6 million to graduate students, postdoctoral and clinical fellows already working in stem cell research labs.

But the board also voted to rejigger a list of priorities drawn up by CIRM staff. The board’s vote lowered the priority of a set of basic biology grants that were to be awarded this year and boosted the priority of part of the funding for the disease team grants, which will be worth a total of $210 million.

The move protects the full amount of funding for the disease team grants, potentially worth up to $20 million apiece, which will be given to 10 to 12 groups of scientists that aim to put stem cell-based therapies into clinical trials within four years. The disease team program is designed to be a jewel in CIRM’s crown, demonstrating the agency’s special role in translating stem cell science into treatments. The move to prioritize the grants came after patient advocates on the board argued that federal funding can now flow to basic biology research: “The NIH funds that [basic] work,” said board member Joan Samuelson of the Parkinson’s Action Network.

CIRM chief communications officer Don Gibbons said the board’s action answers the critics who said that Obama’s policy change has made the agency less necessary: “This action shows that, yes, there is a place for CIRM, and we are doing something different from NIH,” Gibbons said.

The board also voted to elect two vice-chairs: current board member Duane Roth and California Democratic party leader Art Torres. Torres will draw a $75,000 salary, far less than the $332,000 salary discussed last fall, when CIRM drew some flak for awarding board chair Robert Klein a $150,000 salary on the same day that Governor Arnold Schwarzenegger warned that the state was facing “financial Armageddon”.

CIRM President Alan Trounson had the day before the board meeting endorsed the idea that CIRM should carve out a special role for itself as a translational research agency. “Wouldn’t our natural niche be somewhere between the basic science and the clinic? I think this is where we should be,” Trounson said at an 11 March meeting in San Francisco meeting to discuss the future of the agency.

But some scientists at that meeting were worried by that idea. “This push of getting out there to the clinic has some risks,” warned Arnold Kriegstein of the University of California San Francisco at the meeting. “There’s a risk that little will be learned at great cost” if patients are harmed in poorly designed early clinical trials, Kriegstein said.

And Warner Greene of UCSF’s Gladstone Institute of Virology and Immunology cautioned against “turning over the reins” of basic science to the NIH: “It’s foolish to expect now that the disease teams will succeed,” he said.

CIRM chief scientific officer Marie Csete sought to ease such fears at the 11 March meeting: “We are not abandoning basic science,” she said, adding that any CIRM-funded clinical trials will proceed with caution. “No one wants another name like Jesse Gelsinger in this field,” she said, referring to the 18-year-old who died in 1999 as a result of his participation in a gene therapy trial.

But, Csete pointed out that CIRM has “a shorter timeline” than the NIH,” and feels a special responsibility to California taxpayers who created CIRM hoping to get a real return on their investment. “First and foremost, we want to get to the end goal, which is cures,” Csete said.

by Erika Check Hayden

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