California Stem Cell Agency Eyes Changes in Funding Decisions; Possible Impact on Bond Election, Development of Different Therapies

A CIRM slide outlining current programmatic criteria. GWG
refers to the group that reviews applications.  The
 subcommittee reference is to the panel of directors  who
ratify reviewers' decisions. ICOC is the abbreviation for
the name of the governing board.

The $3 billion California stem cell agency is re-examining its criteria for awarding hundreds of millions of dollars with an eye to placing more emphasis on what could be called non-scientific criteria.

The move could have an impact on hundreds of researchers in the state and the development of stem cell therapies that could benefit untold numbers of patients afflicted with a host of deadly and debilitating diseases. It could also have an impact on a possible ballot measure to provide an additional $5 billion for the 13-year-old stem cell program.

The changes could be acted on as early as tomorrow at a meeting of the governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. The meeting is in Oakland, but Internet access is available for those who wish to comment and hear the proceedings.

The move comes under the rubric of "programmatic review" of applications for funding. It has been an ill-defined term for years at the agency. But more specificity was disclosed yesterday in a series of 20 slides scheduled to be shown at tomorrow's meeting of the agency's 29 directors. The posting of the slides came less than two days prior to the meeting.

The agency's staff has laid out seven possible areas where changes might be made:

• "Annual Program Budget and Goals
• "Value Proposition of Proposed Project
• "Patient population, competitive landscape
• "Relevance of Project to Stem Cells
• "Contribution to CIRM Portfolio
• "Disease area, current award overlap
• "Previous CIRM Support of Project"

The full impact of increased use of any or all of those criteria was not clear from the slides provided by the agency. But it could mean that an application that received a high scientific score could be sidelined in favor of one that fills a void or bolsters a weak spot in the CIRM award portfolio.

CIRM slide on possible new award criteria 

Over the years, many CIRM board members have expressed frustration with how the scoring on some applications works. In the case of some applications, only one point separates those receive millions and those who receive none. The concern has been that a one point difference is less than meaningful.

The slides do not flesh out all the likely reasons for putting more emphasis on non-scientific issues, but the agency is approaching the end of its life. It expects to run out of cash for new awards at the end of next year.

A private fundraising effort is underway to tide the agency over until, it is hoped, voters approve $5 billion more for the agency in November 2020.

Changes in award criteria could lead to approval of research whose results are more likely to resonate with voters in time for a ballot measure campaign in two years.

CIRM was created in 2004 by voters who were swayed by a campaign that raised expectations that stem cell cures were just around the corner. The agency has yet to produce a therapy that is available for widespread use. However, it has helped to fund 49 clinical trials, which are the last stages before a therapy is approved by the federal government for general use.

$95 Million in California Stem Cell Grants: Preview the Spending

For those interested in how the California stem cell agency is going to spend its next $95 million, you can check out short digests today of the 19 research grant applications, including reviewer comments, that are virtually certain of receiving the cash. 

The applications came in what CIRM calls its "early translational III" round, which is scheduled to be acted on by the CIRM board May 24 in San Francisco.

Digests of reviewer comments are part of the directors' meeting agenda. They include scientific scores, a statement from the applicant and a summary of what reviewers had to say during their closed door sessions. But you won't find the names of the applicants, their institutions or businesses. The stem cell agency conceals the names of the winners until after the board acts. Names of the unlucky ones are not disclosed by CIRM. The agency says it does not want to embarrass anybody including the institutions involved.

However, persons familiar with the area of science involved may well be able to discern at least some of the names of applicants from the information contained in the summaries.

Scientific scores of the successful applicants ranged from 88 to 53. Nine grants scored higher than 53 but were rejected by reviewers(the Grants Working Group). The panel turned down 22 applications overall. The CIRM board has final authority on applications, but has almost never rejected a positive decision by reviewers. Sometimes, however, it will overrule a negative decision.

One successful application that was scored at 53 involved ALS. The $1.7 million proposal was approved for "programmatic reasons," according to the summary. Often, programmatic motions for approval are made by CIRM board members sitting on the review panel. However, the summary did not disclose who made the motion or the vote. The summary said,

"The programmatic reasons provided were that ALS is a devastating disease that is not well-represented in CIRM's portfolio."

The other successful application that scored at 53 sought $6.3 million for research involving heart disease. The summary did not clearly identify the specific reason for approving the grant on a programmatic motion. But it said,

 "The GWG (grants working group) ... advised as a condition for funding that the applicant consult additional vector specialists with translational and clinical experience to select a more appropriate vector to move this program towards the clinic." 

Again CIRM withheld the vote on the motion and the name of the person who made the motion.

Applicants who have been rejected by reviewers can appeal to the full board. So far no appeals have been publicly posted by CIRM. The success rate on such appeals is mixed.

The translational round was open to both academics and businesses, which have received a tiny fraction of CIRM's $1.3 billion in spending so far. Some businesses have complained publicly and, as well, to a panel of the Institute of Medicine that is evaluating CIRM's performance.

The California Stem Cell Report yesterday asked CIRM for the number of businesses that applied in the translational round, including the pre-application process, which is used to whittle down the total number of applications. The request included total numbers as well. CIRM spokesman Kevin McCormack declined to produce the figures prior to the CIRM board meeting, saying they "won't be ready" until after the session.