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with information that is now outdated.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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