Parochial or Global? The California Research Spending Rule and Science

UC Davis stem cell researcher Paul Knoepfler (left) has triggered an interesting discussion on his blog centering on the question: “Does it matter where stem cell research happens.”

This is of considerable interest here in the Golden State, which has a rich, $3 billion stem cell research effort that it is basically limited to California. The reason for that is entirely political. It would have been folly to ask voters to approve the program, as they did in 2004, if the money were going to flow to Harvard, Great Britain and Singapore.

Enforcing and defining the limits on spending are not small issues. The California stem cell agency has written its rules in such a way that companies headquartered elsewhere and also with major operations elsewhere still can receive grants.

One example is the $3.7 million CIRM grant to Maine's Jackson Laboratory. The justification is that the money is largely being spent at the company's Sacramento facility. Out-of-state spending also nearly sank a $5.4 million grant to Evan Snyder at Sanford-Burnham.

However, stem cell research is global. A powerful argument can be made that funding should be global if the science is to avoid pursuing a pinched path of parochialism. Should poor or even average science be funded because it is peculiar to California? Of course, considerable differences do exist on just what is “good” science.

On his blog, Knoepfler cites the case of Advanced Cell Technology of Santa Monica, Ca., which moved its headquarters to California largely because of the passage of Prop. 71. The company, however, apparently has no significant research operations in California. All are in Massachusetts, where it was previously headquartered.

ACT has not received funds from CIRM. Has it sought any? CIRM won't say. The agency cloaks its applicants in secrecy, so it is nearly impossible to tell whether a specific company has applied for taxpayer funds. But one of ACT's researchers complained to the CIRM board about a negative decision on a grant application. The researcher said one of the reviewers had a conflict of interest, which CIRM denied.

Knoepfler and others commenting on his blog wrestle with some of the important issues of geography-based funding, which even extends to choices within California. The main argument, however, for maintenance of the ban on non-California grants is political. Support for CIRM would vanish if it were to send its billions to out-of-state researchers, regardless of the worthiness of their efforts.

(Editor's note: A very early version of this item did not contain the information about the ACT researcher's complaint about a conflict of interest.)

$50 Million Clinical Trial Effort Proposed to CIRM Directors

The California stem cell agency has released additional information on its proposed, first-ever funding round for participation in clinical trials.

The plan, expected to be approved on Thursday by directors, would provide $50 million for one or two trials and would be limited to “novel cell therapies from pluripotent stem cells.” Both businesses and non-profit institutions could compete, but businesses would be required to take a loan instead of a grant. In both cases, matching funds would be required. No match percentage was specified.

The goal, a CIRM document said, is

“completion of early stage clinical trials within three years that: 1) demonstrate preliminary safety in humans and 2) provide compelling data for proof of mechanistic concept and/or early testing for efficacy that could lead to more definitive efficacy studies.”

The clinical trial proposal is part of CIRM's aggressive push to develop therapies from stem cells. The agency has already committed one-third of its $3 billion in resources. It will need to show some high profile, tangible results if it is secure additional funding in a few years.

California voters approved the stem cell research effort five years ago, creating CIRM as a way to circumvent federal restrictions on funding for human embryonic stem cell research. Prop. 71 gave top priority to support for that area. Notably, however, the clinical trial proposal does not extend to hESC research.

The plan is part of a relatively brief agenda for the CIRM board meeting in Sacramento this week. Traditionally, board members use part of the day to discuss CIRM privately with legislators and legislative staff.

Another matter on the agenda involves a grant in which a La Jolla researcher planned to use state funds to finance research outside of California, which is barred by CIRM. The $3.6 million grant to Evan Snyder of the Burnham Institute was approved by directors last April. Apparently it was unknown at the time that Snyder planned to use state funds outside of California. After months of discussions and the hiring of an outside consultant, CIRM staff is now recommending that the grant be funded at $5.4 million to handle the increased costs of doing all the work in California.

The staff report said,

“CIRM staff was assured that the core facilities at the grantee institution were adequate and could replace all the research activities critical to this project. To address the ability of junior staff to substitute for experienced senior out-of state investigators, CIRM was reassured that, in addition to the expertise of the PI, there is an established collaborative, integrated multi-institutional network that will be actively engaged in the project and can provide scientific and technical expertise to complement the newly hired and more junior staff. CIRM also received adequate assurances with regard to the ability of the PI to commit adequate time to the project.”

Not discussed in the staff report is the question of exactly how the grant happened to win approval from CIRM directors with the non-California component. CIRM rules are explicit on the ban on the use of CIRM funds outside of California. We are querying the agency concerning the matter.

Oddly, the staff report also does not mention the names of those involved and the institutions despite the fact that they were identified to the board last April.

The grant was approved by directors after Snyder filed an “extraordinary petition,” which is usually an appeal by an applicant who has been rejected by scientific reviewers.. In Snyder's case, reviewers, however, had approved it for funding.

Also on the CIRM directors' agenda is a proposal that would apparently increase the salary for the new vice president of research and development, who would effectively be the No. 2 staff person at CIRM. Currently the top of the range is $332,000 but does not seem high enough to lure the right person. CIRM has not provided any details, however, other than a terse listing that compensation for the position will be considered by directors.

Another item to be considered involves recommendations on new state legislation affecting CIRM. As we reported earlier, the agency hopes to sidetrack the bills by asking that they be sent off to interim study for a year or so.

The latest version of the agenda contains instructions for listening to the board proceedings via the Internet.

(Editor's note: An earlier version of this item incorrectly said that the Snyder grant would have used CIRM funds to support his Australian collaborators. CIRM said that funding in that country is coming from a non-CIRM source.)