Tuesday, July 24, 2012

Record Appeals by Researchers in Huge California Stem Cell Agency Round

The California stem cell agency's latest grant round – which is budgeted for $243 million – has drawn an extraordinary and record outpouring of appeals from more than half of the scientists rejected by the grant reviewers.

Nine of the 15 applicants who were turned down have filed appeals to the governing board for its meeting Thursday in Burlingame. No other CIRM grant round has drawn as high a percentage of appeals, formally known as extraordinary petitions. (See here for a story on the previous record for percentage of appeals.)

Only six applicants were approved for funding in the second of the agency's signature disease team rounds. Their applications totaled $113 million, although $243 million was allotted by the board, which would make it the largest in the agency's history. The round is aimed at bringing proposed clinical trials to the FDA for approval or possibly starting trials within four years.

The rejected applicants come from both biotech firms and nonprofit institutions, including at least two that are ready to begin clinical trials next year, a much-sought goal of the stem cell agency.

The appeals, posted late yesterday on the CIRM web site, deal mainly with the details of the research although they also say, in some cases, that reviewers did not grasp the facts or were dealing with information that is now outdated. Here are some samples of what the applicants had to say in their petitions.

Linda Marban, CEO of Capricor, Inc., said her firm's heart disease therapy proposal, which was rejected by reviewers, is set for a clinical trial beginning next year. She said that since the time of the review last April the firm has “made major advances in both our clinical development program and our management and operational team.”

Marban noted that Frank Litvack, former CEO of Conor Medsystems Inc. of Menlo Park, Ca., is now executive chairman of the Capricor and will provide “experienced operational leadership.” In June 2011, Litvack was nominated to chair the CIRM board. However, directors chose J.T. Thomas, a Los Angeles bond financier who has filled that post since then.

In another appeal, researcher Stuart Lipton of Sanford-Burnham said,
 “Some reviewers were concerned about the PI's (principal investigator's) lack of experience in developing a cell therapy for clinical transplantation. Reply: Since there is currently no FDA-approved stem cell product for transplantation therapy for (the) brain, no PI could possibly have the experience requested here.”
Alexandra Capela of StemCells, Inc. said reviewer comments on her application “were inconsistent with previous guidance provided by CIRM.” She said reviewers “objected to a clinical approach that was already supported by the CIRM in a successful early translational research grant and the planning grant(for her application).” Capela said this “contradicts previous acceptance of this strategy by CIRM and constitutes a central reason for our appeal to the (CIRM board).”

Henry Klassen of UC Irvine said reviewers “overlooked facts” in his application and “had no real way of gauging the extraordinary rate of our progress.” He said trials are already scheduled for next year, far earlier than time frames set up in the RFA for this round.

In addition to the written appeals, scientists can appear before the full board at its meeting and often have. Sometimes they bring patients, who make emotional presentations. The board does not have to discuss any of the appeals. Unless a board member makes a motion on an appeal, it does not even come up for a vote.  Directors are generally reluctant to approve an appeal. Here is a link to a CIRM description of the petition process.

Here is a list of researchers appealing their rejections along with links to their appeals. The list is in the order in which CIRM posted them on the board agenda. The review summaries for all the grants can be found here.

For more on the CIRM grant appeal process, see here.
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