CIRM Okays First Clinical Trial Round; $50 Million Earmarked

Directors of the California stem cell agency this morning unanimously approved a $50 million foray – its first-ever -- into the financing of clinical trials.

The program appears likely to be of benefit to only a handful of organizations – particularly the three that have pluripotent proposals that are now on hold with the FDA. The names of the enterprises were not disclosed during the board meeting because to do so would raise conflict of interest questions.

The CIRM staff proposed one or two trials that 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. During the board discussion, the CIRM staff provided two examples of matching requirements. CIRM would provide a $20 million grant on a $40 million trial with the rest to be provided by the applicant or $25 million on a $75 million trial.

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.”

Several board members said the clinical proposal, which could lead to awards by the end of this year, represented exactly what CIRM should be doing. Ted Love, a board member and executive vice president of Onyx Pharmaceuticals of Emeryville, Ca., said,

“We don't have the luxury of waiting.”

He and others, such as patient advocate board member Jeff Sheehy, cited the need to bring therapies to the point where they can actually be used to treat suffering persons.

Some board members did express concern about the possible appearance of favoritism and the need to ensure that the proposal contain sufficient funds to monitor clinical trial patients for long periods, perhaps their lifetimes.

CIRM President Alan Trounson also said that he understands that some of the potential applicants are facing serious cash problems and need to find funding soon. He said the clinical trial proposal also might bring out more applicants than the three possibilities he is aware of.

Under the plan, Trounson would be given significant discretion to deal with the needs of applicants.

The ambitious proposal is part of CIRM's drive to bring therapies to market. More funding for clinical trials is expected to be provided by CIRM within the next year or so. The agency has already committed one-third of its $3 billion in resources. It will need to show tangible, publicly appealing results if it is secure additional funding in a few years.

Here is a link to the CIRM press release on the program.