$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.)