Tuesday, August 18, 2009

CIRM Directors Wrestle with Strategy and Ties to Business

The Chinese calendar calls this the year of the ox. At the California stem cell agency, however, this is the year of cozier relationships with the biotech industry.

At a two-day San Francisco meeting that begins tomorrow, the $3 billion state enterprise will re-examine its strategic plan with an eye to strengthening its ties to industry. CIRM's intent is to push taxpayer-financed stem cell therapies into the marketplace and actually treat people – but not necessarily right away. That probably won't happen for 10 to 14 years, according to its plan. Science and regulators are slow.

The plan (here and here) covers a lot of ground, ranging from CIRM's goal of a creating a “stem cell culture” in California to beefing up the management of its burgeoning portfolio of grants, which is expected to hit $1 billion this year.

But the overriding theme is closer ties to industry. A new and highly paid vice president will be handling them. CIRM will lobby increasingly on behalf of industry. The agency will implement an unprecedented, $500 million lending program for the riskiest biotech firms. And it will recruit scientific grant reviewers who understand “what is necessary for commercial success.”

Nothing is wrong with this, in principle. In fact, we think CIRM early on was not aggressive enough in engaging business. Only tiny numbers of industry representatives have appeared at CIRM meetings. Few companies have been successful in winning CIRM grants. And rejected business applicants have complained bitterly about the process.

Business must be firmly engaged with CIRM in order to develop therapies. But the marriage of business and government is fraught with peril. The best of intentions can go awry. They seemed to have done so with proposed creation this week of “a tremendous loophole” in the agency's earlier regulations to ensure affordable access to taxpayer-financed therapies.

John M. Simpson
has observed CIRM for several years as the stem cell project director of Consumer Watchdog of Santa Monica, Ca. He and others we talked to have expressed concern about the vagueness of the strategic plan's proposals about industry. In response to a query, he said,
“The strategic plan envisions greater ties with the business community without providing enough specifics of how it all will work. Implicit in the plan seems to be the idea that if businesses are not taking CIRM's money it is a failure on CIRM's part and CIRM needs to loosen its rules.

“This loses sight of the fact that CIRM plays a regulatory role. In fact, if businesses want taxpayer dollars, businesses need to abide by CIRM's well-crafted regulations.

“CIRM seems to be saying we'll keep changing the rules, until you come and play with us. CIRM needs to understand if you go too far down a path of accommodation, the game isn't worth it.”
As part of the plan, another observer says that it would be useful for CIRM to survey activity in California's private sector concerning stem cell research. And, he said, more details are needed on CIRM's role in clinical trials and just exactly how it will help industry in connection with the FDA.

Earlier this year, CIRM's apparent move away from basic research triggered concerns among some scientists. They argued that it is too early to push most research into clinical trials. For example, the agency this year will approve a $210 million disease team grant round, its largest ever single research grant round. CIRM's plan reaffirms a commitment to basic science, but whether that allays fears of critics remains to be seen.

One matter that may trouble many scientists is what may be a reluctance to share information outside of the CIRM research community. The strategic plan discusses sharing “instructive negative research” results within its own community. And it says,
“To manage the flow of information, CIRM is developing and implementing a categorization system and database to store information according to disease relevance, cell types and technologies employed, research results, questions raised and answered and possible next steps.”
In neither of those two examples does CIRM specifically say the information will be shared outside the CIRM community or with the public. That may be an oversight, but it would useful to have some assurance that the information will be publicly accessible.

The latest revision to the strategic plan also presents scientifically justified movement away from its original charter – funding work related almost entirely to hESC research. In fact, the word “embryonic” only appears 20 times in the plan's 37 pages. That may be disappointing to some patient advocates, who may also not be pleased with the 10 to 14 year timetable for therapies.

Simpson additionally identified one objective in the report that he took issue found dubious. He said,
“As a scientific goal CIRM pledges to 'encourage the development of a 'stem cell culture' in California...'

“That makes about as much sense as if NASA said it planned to encourage a rocket science culture in the United States.

“The notion of a 'stem cell culture' becomes even more troublesome when you consider the hype that has been all too common in the field.”
(The strategic plan will be taken up at a two-day meeting of the CIRM board that begins Wednesday in San Francisco. The public can participate in Southern California at a location at the City of Hope in Duarte. A Web audiocast is also available without the possibility of participation. See the agenda for details.) Sphere: Related Content

1 comment:

  1. Anonymous4:27 PM

    If CIRM wants to encourage the advance of stem cell science into medicines, it needs to fund studies that provide the backbone needed to support that advance as well as training for grantees in the application of science. Not all researchers will be interested in doing this type of preclinical research but there are likely to be many that will, and through support and interaction with them, not only can CIRM grow the stem cell culture they want but also develop some knowledgeable stem cell entrepreneurs that will start or populate companies to take their therapies forward.Seeing science come to something can be a strong motivator across the spectrum and based on history, it can propel a field forward more effectively than probably anything else.

    Investing in the riskiest companies may not be wise unless they are willing to accept some guidance with the investment. Geron's difficulty getting to the clinic, stalled yet again today, is evidence that there is much to be learned.