California's $25 Million Loan to Geron: Approval Came Only After Major Departure from Longstanding Procedures

The state of California chalked up a historic first last May when its stem cell research agency approved a $25 million loan to a corporation engaged in another first – a clinical trial for a treatment created from human embryonic stem cells.

It was the first time that the state has funded a clinical trial -- one watched by untold numbers of persons globally who hope that stem cells will ease their pain and cure their suffering. The funding is also critical to Geron Corp. of Menlo Park, Ca., which initiated the safety trial for its spinal injury stem cell therapy.

CIRM President Alan Trounson told agency directors in May,

"It's just possible that this trial might have faltered without our backing."

Approval of the loan, however, came after the $3 billion stem cell agency publicly deviated significantly from its normal funding procedures. The Geron application was not given a public scientific score, standard practice for all the other 433 applications that the agency has approved over the last six years. The usual summary of grant reviewer comments was not provided to the public or the board. The three other applicants in the $50 million round all withdrew prior to presentation to the CIRM board – another first in CIRM's grant program. And no public explanation was provided at the time for the departures from long-established procedures.

In response to questions from the California Stem Cell Report, CIRM defended its actions. James Harrison, the board's outside counsel, said the unusual handling of the Geron application was necessary to protect confidential information. Harrison said,

"CIRM has a significant challenge and responsibility to protect the confidentiality of the companies’ submissions as any violation could have adverse consequences for the companies, including a material disclosure, particularly for those companies that are publicly traded."

Regarding the failure to provide a summary of grant reviewer comments, Harrison said a summary was indeed available, pointing to a document that contained only a 67-word listing of the criteria used by evaluators. By contrast, the top-scoring applicant for a $14.6 million disease team grant in 2009 had a 1,219 word summary review.

Regarding withholding the scientific score of the Geron application by reviewers, Harrison said publishing it would have been "confusing" because all the other applicants had dropped out.

Asked whether the applicants were encouraged by CIRM in any manner to drop their applications. Harrison replied,

"In this case, three applicants, on their own volition, withdrew their applications before the board meeting and therefore the applications were not presented to the board."

Asked whether the other applicants had any sort of assurances that they would have a better chance later, Harrison said,

"CIRM has made no assurances, either directly or indirectly, to any applicants, including the applicants for the Targeted Clinical Development Awards, regarding future funding.  CIRM does try to assist applicants by providing feedback, where appropriate."

As to the lack of an explanation to the public and interested parties for the departure from longstanding procedures, Harrison did not respond directly. He said,

"This was CIRM's first clinical trial review and we expect to strengthen and refine the procedures for the next round, including explaining to the public how and why clinical review summaries differ from other CIRM grant review summaries."

(The full text of Harrison's statement can be found here.)

Geron's application was approved in May by directors on a 16-1 vote. The dissent came from Joan Samuelson, a patient advocate member of the board and a member of the grant review group.

According to the transcript of the directors meeting, she said the trial was not ready and CIRM was not ready.

"There were lots of -- this is based on the peer review and the comments by the scientist members of the grants working group. There were many concerns that many of the scientist members felt should be satisfied before embarking on a clinical trial and they weren't."

Ellen Feigal, CIRM's vice president for research and development, responded following Samuelson's remarks. Feigal said,

"I want to make it clear, making the decision to move towards and into a clinical trial is a very complicated decision. It's not black and white. It's based on judgment, on experience, on science, and the data, and there's not really a right or wrong answer. And I just think that the tenor of the discussions that we've had, the tenor of the discussions of the grant review group had were appropriately deliberative and considered all the different issues."

(Editor's note: Discussion of the Geron application begins on page 142 of the May transcript. It picks up again on page 153. The 16-1 roll call vote on the application by the 29-member board can be found here. Five board members did not vote because of conflicts of interest. The others either were not present or did not answer the roll call. Votes by the grant review group are not disclosed. The names of scientists specifically evaluating applications are not disclosed except for the membership of the grant review group.)