Capricor and California Stem Cell Agency Say All is Well With $27 Million Investment

One recent headline in Forbes magazine read, 

“Stem Cell Therapy To Fix The Heart: A House Of Cards About To Fall?”

Over at the Boston Globe, another story this month said,

 “A provocative new study calls into question the rationale for using stem cells to repair the heart — a much-hyped experimental therapy that grew out of insights from a groundbreaking Boston researcher’s laboratory.”

Eduardo Marban
Cedars-Sinai photo

But it appears that heart stem cell regeneration is like cheese, you might say. There are many different varieties. And in the case of a $27 million investment in heart cell regeneration by the California stem cell agency, all the players say things are just fine with their project.

The state's research effort involves a publicly traded company called Capricor Therapeutics. The Beverly Hills firm and one of its founders. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, received the $27 million from the state stem cell agency.

In the wake of the news reports involving heart stem cell regeneration, the California Stem Cell Report queried both Marban and the stem cell agency. Marban replied within 37 minutes last week. Here is the text of what he had to say in his email.

“All of the critiques have been leveled at competing technologies. Indeed, I have been among the more vocal critics (for example see http://www.tctmd.com/show.aspx?id=124193).

“Although some alarmist headlines have inappropriately questioned all cardiac stem cell work, the issues have to do with only one specific subtype of cardiac stem cell, which is marked by the expression of an antigen called c-kit. This is NOT the cell type being developed by Capricor; in fact, my laboratory has found that c-kit-positive cells play no role in Capricor’s cell product. That product, known as CAP-1002, contains a small fraction (less than 5 percent) of c-kit-positive cells which can be completely removed with no loss in potency. CAP-1002 is believed to work by indirect 'paracrine' mechanisms that differ fundamentally from those postulated for the c-kit-positive cells.

“By way of background, the critiques of c-kit-positive heart cells have come in two forms: first, in concerns expressed by the editors of the Lancet regarding data integrity in the SCIPIO trial; this was conducted by Piero Anversa and colleagues using c-kit-positive cells. Second, a recent Nature paper (attached in case you do not have a copy), of which I am an author, questions the claim that c-kit-positive cells are true cardiac progenitors.

“These concerns in no way undermine Capricor’s technology. In fact, CAP-1002 remains the only heart-derived cell product in commercial development. I am proceeding full speed ahead with mechanistic and translational work on CAP-1002, and Capricor continues to feel bullish about the product, which is now in phase 2 trials (the furthest along of any CIRM-funded project).”

Kevin McCormack, a spokesman for the stem cell agency, also said that Capricor is using a different method and a different stem cell than the one being called into question.

You can hear the May 15 Capricor conference call on its first quarter results here. Its stock price jumped from $6.15 on May 14 to as high as $7.65 during the day May 15. Its 52-week low was $1.15 and its 52-week high $17.15.

(Editor's note: If you would like a copy of the Nature paper that Marban referenced, please send an email to djensen@californiastemcellreport.com.)

$61 Million Approved for Stem Cell Clinical Trials

Directors of the California stem cell agency today handed out $61 million in its ambitious disease team program that is designed to push stem cell research into clinical trials.

Action on the six awards followed yesterday's move by the board to set aside $200 million to beef up its efforts for stage one and two clinical trials. The agency is focusing ever more intently on commercializing its research.

The $3 billion program is expected to run out of cash for new grants in 2017. Its efforts to develop a new stream of funding depend a great deal on showing results that resonate with the public and possible funding sources.

This grant round was originally budgeted for $100 million, but directors today were loath to approve additional funding. They noted that the agency is now living an era of financial limits.

Today's awards are aimed at cancer (three grants), sickle cell anemia, macular degeneration and severe airway obstruction. Size of the awards ranges from $20 million to $4.4 million. Terms range up to four years.

The agency did not immediately release the names of the winners, in keeping with its policy of secrecy until after the board votes. However, the names of the applicants could be determined by a knowledgeable person searching articles in the various fields. A CIRM news release is expected shortly with the names of the researchers and institutions. (Here is a link to the press release. All of the recipients are employed at institutions represented on the governing board.)

Thirteen applications, including one from a business, were considered by reviewers.

Here are links to the information provided by CIRM staff on the grant applications: summaries of the grant reviews, staff justification for funding tier two applications, Power Point presentations to be made to directors on the applications, including the range of scores on individual applications. However, the range of scores on the rejected applications was not provided.

Stem Cell Board Nixes $13.4 Million to Help Cure Cartilage Disorders

Directors of the California stem cell agency today rejected a $13.4 million proposal that would have been the first disease team research that it has backed that would deal with cartilage disorders.

The project would have built on earlier research funded by CIRM. The agency staff cited work at UC Davis  by Kyriacos Athanasiou and at Scripps by Peter Schultz. Funding of the application was supported by the CIRM staff, which did not identify the applicant.

However,  the governing board, however, went along, on a 10-1 vote, with arguments by Director Jeff Sheehy and others that the agency had finite resources and had to make difficult decisions. He noted that many alternative treatments exist for cartilage problems. Sheehy said,

"We are here to make progress on serious diseases and conditions."  

Former Chairman Robert Klein, in an unusual appearance as a member of the public, urged approval of the grant. He said that it would address conditions "very prevalent in the older population."  Klein told the board that the research would be a "very important milestone that will help you get more money in the future."

While Klein did not mention it, older persons participate in elections to a much greater degree than younger persons. They would be an important group in winning support for another bond issue to fund CIRM, which runs out of cash for new grants in 2017.

$13.9 Million Approved for Sickle Cell Research at UCLA

The California stem cell agency today effectively approved a $13.9 million grant involving UCLA to develop stem cell gene therapy for sickle cell anemia.

CIRM directors acted on a staff recommendation to place the grant in a category that is automatically approved by the board.

(Here is a press release that was issued later by UCLA. The investigator is Donald Kohn.)

CIRM Directors Reject $16 Million Eye Research Application

Directors of the California stem cell agency today rejected a $16 million application from Cedars-Sinai for research involving retinitis pigmentosa.

The action came on a split vote despite a personal appearance by the principal investigator, Kent Small, and a patient advocate.

CIRM staff had recommended rejecting the grant because the agency is funding a number of other eye research projects. The vote was 7-4 to approve the staff recommendation.

Small and Clive Svendsen, also of Cedars-Sinai, noted that other diseases have many grants from CIRM and have not been subjected to the same sort of objection. They noted that the eye is a particularly good target for stem cell therapy compared to other areas of the body.

$4.4 Million Grant Approved for Severe Airway Obstruction

The governing board of the California stem cell agency today effectively approved a $4.4 million grant for research involving severe airway obstruction.

The board approved a staff recommendation to place the grant in a category that the board automatically votes to fund. Names of the applicant and employer have not been released by the agency.

$98 Million for Stem Cell Therapies for Cancer, Arthritis and More

Directors of the California stem cell agency this week are expected to approve nearly $98 million in awards for early stage clinical trials for treatments of diseases ranging from sickle cell anemia to cancer.

The awards will involve the $3 billion agency's signature disease team program, which is aimed at pushing stem cell research into the marketplace.

In addition to sickle cell anemia and cancer(see here, here and here), the applications involve therapies for macular degeneration, arthritis and severe airway obstruction. Names of the applicants were withheld by the agency. The identities of successful applicants will be released following board action. Names of the rejected applicants, however, are not released by the agency. The number of business applications was also not immediately released.

The goal of the latest round in the disease team program is completion of an early phase clinical trial during the award period, which can be up to four years. Amounts of the proposed awards range from $20 million to $4.4 million. CIRM originally budgeted $100 million for this round.

Four applications won outright approval from the agency's grant reviewers. CIRM staff recommended that three out of four “tier two” grants be approved. The scientific scores of all seven of those applications range from 79 to 67 on a scale of 100.

Some of the five rejected applicants and sympathetic patient advocates may well appear at the board meeting in Los Angeles on Wednesday and Thursday to lobby for their proposals. The CIRM board can fund any of the applications, regardless of their scientific scores.

Here are links to the ample information provided by CIRM staff on the grant applications: summaries of the grant reviews, staff justification for funding tier two applications, Power Point presentations to be made to directors on the applications, including the range of scores on individual applications. However, the range of scores on the rejected applications was not provided.

Searching for CIRM's Financial Future, Plus a $100 Million California Stem Cell Round

The California stem cell agency next week is expected to give away as much as $100 million as it pushes aggressively to generate therapies that will benefit the Golden State as well as the world.

At the same time, the agency will decide whether it will focus more intensely on a handful of projects as it tries to come up with a plan to finance its future. The $3 billion agency is currently funded by money that the state borrows, but cash for new grants will run out in 2017.

The agency is looking for a way to continue operations through some sort of public-private partnership. A consultant's report was due on that subject on Nov. 30. At its meeting Dec. 11 and Dec. 12 in Los Angeles, the board is scheduled to spend one hour hearing a presentation on the report at the end of a lengthy examination of recommendations from the agency's new Scientific Advisory Board(SAB), the panel that proposed that CIRM sharpen its focus. The California Stem Cell Report has asked for a copy of the document, which is a public record.

The SAB report plays into the agency's need to come up with results that will stimulate both private and public interest in supporting the agency's future operations. So far, CIRM has lacked the research results that resonate widely with the public.
 
The agency has prepared a tightly organized schedule for examining the SAB proposals, which could mean that the agency's shared labs, bridges and training grants programs would be phased out. However, CIRM staff is only recommending an end to the shared labs program.

The staff has proposed creation of an “accelerated development pathway” for six to eight projects, as recommended by the SAB. The staff document did not describe what that would entail or how much it would cost.

Also on the staff list is a proposal to advance the most promising early translational research programs and a $10 million proposal by CIRM President Alan Trounson to participate in a global effort “to create (a) Haplotype Bank and GMP library.” No further details on that plan were available at the time of this writing.

For the stem cell community, the immediate big news next week will be board action on applications in its signature disease team program. This is the third round, and awards could run as high as $20 million each for a total of $100 million. The round also involves possible collaborators in England, Spain and China, a country that has been pushing hard on stem cell research. No state funds will be used overseas.

According to the RFA, the purpose of the disease team effort is “to advance early clinical development of novel therapies derived from or targeting stem cells, potentially offering unique benefit with well-considered risk to persons with disease or serious injury.”

The summaries of the grant review findings have not yet been posted on the CIRM Web site, but are likely to come later this week. The CIRM governing board almost never overturns a positive recommendation from its reviewers. Sometimes it will approve an application rejected by reviewers. However, directors are feeling squeezed financially and may not be as open to elevating rejected applications as they have been in the past. Look for rejected applicants to personally petition the board for approval along with affected patient advocates.

Also on the agenda is the topic of the search for a new president for the agency to replace Trounson, who is resigning to return to Australia and his family. An item was posted on that yesterday.

Next week's meetings are open to the public in Los Angeles and at teleconference locations in La Jolla and Pleasanton. Addresses can be found on the agenda. The board also usually provides an Internet audiocast and a Webinar link to PowerPoint presentations used at the meeting. Directions for using those tools are likely to be posted on the CIRM Web site later this week.

California Stem Cell Merger: Capricor and Niles Therapeutics

Capricor, Inc., a Beverly Hills company benefiting from $27 million from the California stem cell agency, this week announced that it is merging with Niles Therapeutic, Inc., of San Mateo.

Linda Marban
Capricor photo

The Capricor story and its treatment for heart disease have been highlighted (see here and here) by the $3 billion state research agency, which is partially funding a clinical trial for the firm. The firm sprang from work by Eduardo Marban of Cedars-Sinai in Los Angeles, one of Capricor's founders. He received $6.9 million for his early and current work. Capricor was awarded $19.8 million more.

Capricor, a privately held firm, and the publicly traded Niles announced on Monday that they were merging. The new company will be known as Capricor Therapeutics, Inc., and will be based in San Mateo.

The new firm will be publicly traded with Capricor CEO Linda Marban as the new CEO.

The new board of directors will have two members from Niles and seven from Capricor, including its executive chairman, Frank Litvack, who was an unsuccessful candidate for chairman of the stem cell agency board in 2011.

The merger press release said that the new company “should have better access to capital, more potential for steady pipeline development and more risk diversification."

On completion of the merger, a joint press release said,

“Nile will issue to Capricor stockholders shares of Nile common stock such that Capricor stockholders will own approximately 90% of the combined company's outstanding shares, and Nile stockholders will own approximately 10%, calculated in each case on a fully-diluted basis assuming the issuance of shares underlying options and warrants. Options of Capricor will be assumed by Nile and become options to acquire stock of Nile.”

Linda Marban said,

"Capricor's and Nile's product portfolios complement each other well, as our therapies will address both the underlying causes and debilitating effects of heart disease. Capricor's CDCs are allogeneic cardiac derived stem cells that aim to attenuate and potentially improve damage to the heart that can result in heart failure, while Nile's cenderitide is intended to treat patients following hospital discharge from an acute episode of heart failure."

Niles' stock price stood at $0.04 recently. Its 52 week high was $0.20 and the 52-week low was $0.02.

More Researchers Eligible for $100 Million Stem Cell Round

Directors of the California stem cell agency today expanded eligibility for the $100 million disease team round to include some earlier recipients in its early translational rounds.

Ellen Feigal, vice president for research and development, said some of those grantees are likely to be ready for more funding as they move closer to clinical trials. She said if they cannot apply in this round, they may have to wait for another year.

Directors agreed that it was important to avoid gaps in funding on successful programs. The RFA could be posted very soon.

California Stem Cell Agency Boosting Disease Team Program to $543 Million

Directors of the California stem cell agency are set to give away $20 million next Thursday and authorize a handsome addition to their signature disease team effort, bringing its total to $543 million.

It is all part of the $3 billion agency's push to develop therapies prior to running out of money for new grants in 2017.

The $20 million is expected to go to the first two winners in the agency's new strategic partnership program. CIRM says the effort is aimed at creating “incentives and processes that will: (i) enhance the likelihood that CIRM funded projects will obtain funding for Phase III clinical trials (e.g. follow-on financing), (ii) provide a source of co-funding in the earlier stages of clinical development, and (iii) enable CIRM funded projects to access expertise within pharmaceutical and large biotechnology partners in the areas of discovery, preclinical, regulatory, clinical trial design and manufacturing process development.”

CIRM reviewed six applications with two winning approval. The agency's governing board is expected to ratify the decision next week. None of the applicants have been identified by the agency, which routinely withholds that information prior to board action even when applicants have identified themselves.

Addition of a new $100 million disease team round will come on top of the second, $213 million disease team awards approved last this summer. The first round, awarded in 2009, totaled $230 million.  The size of the new round could be altered by CIRM directors prior to approval. Also before the board is a $40 million proposal to expand the industry-friendly strategic partnership effort into a second round.

The thrust of the disease team effort is to speed the process of establishing clinical trials and to finance efforts that might founder in what the biotech industry calls a valley of death – a high risk financial location, so to speak, where conventional financiers fear to tread.

The new disease team round will require “co-funding” from applicants but the agency did not specify what it means by the term. The matter of matching funds has become an issue in awards to StemCells, Inc., of Newark, Ca., in this summer's disease team round.

Next week's agenda additionally contains a plan to tighten review of proposed research budgets in grant applications, making it clear that CIRM staff will be negotiating such matters even after the board approves grants and loans.

So far no researchers have testified in public on the budget plan although it could well have a significant impact on their future efforts.

Additional matters will discussed as well at the meeting in Burlingame, which also has a teleconference location in La Jolla that will be open to the public. The address and additional material can be found on the agenda.  

Two Additional Appeals Rejected in Disease Team Round

The governing board of the California stem cell agency last night rejected appeals by two applicants -- OncoMed Pharmaceuticals of Redwood City and Albert Wong of Stanford -- in the $200 million disease team round.

Both petitions generated little discussion. You can find more on their petitions here and here.

The board also approved changes in its intellectual property and grant administration rules. Both proposals will now enter the state's official administrative law process for more comment and possible change.

StemCells, Inc., Wins Another $20 Million From California Stem Cell Agency

Following a second impassioned pitch by its former chairman, Robert Klein, the governing board of the California stem cell agency approved a $20 million award to a financially strapped biotech firm, StemCells, Inc., of Newark, Ca.

Approval came on a 7-5 vote with the condition that the company demonstrate it has access to $20 million in matching funds prior to funding.  It is the second $20 million award that the company has received in the disease team round, which now totals $214 million. Another disease team application has been tabled and will not be considered until October.

The current CIRM chairman, J.T. Thomas, a Los Angeles bond financier, asked for the financial proof because he said some concerns were expressed during an executive session that CIRM would now "account for such a large part of the assets of the company." Martin McGlynn, CEO of StemCells, Inc., also told the board that the company might have to drop its Alzheimer's research if it did not receive the CIRM award.

The StemCells, Inc., application was rejected twice by reviewers. The original rejection came before the July meeting at which Klein first appeared (see here and here). The proposal was then sent back for re-review, during which it was rejected again.

However, the 29-member board narrowly approved the application following discussion tonight and following its rejection of another Alzheimer's research proposal from USC. Both applicants produced a number of witnesses, including patients, on behalf of their appeals.

The re-review on the StemCells, Inc., application said in reference to a statement by Klein to board in July,

“The reviewers did not feel there was compelling data for neuron migration in the submitted manuscript. This is the manuscript specifically referenced at the ICOC (CIRM governing board) meeting (in July) that prompted the call for additional analysis. The manuscript is not yet accepted, it is 'potentially acceptable' but requires 'major revisions' according to the journal editor note. In addition, however, the studies in this manuscript used mouse NSCs, not the human NSCs proposed for the disease team award....”

In July, Klein said, “....(W)e have brand-new data that demonstrates and totally contradicts the key weakness on which it was downgraded.” 

A footnote: The CIRM staff said that as a result of two StemCells application, a proposal is being prepared to limit applications to one per entity in later rounds.

CIRM Executive Session on StemCells, Inc., Appeal

The governing board of the California stem cell agency is meeting in executive session to consider proprietary information on an appeal by StemCells, Inc., following a second public pitch by the agency's former chairman, Robert Klein, on behalf of the financially strapped business.

Martin McGlynn, CEO of the publicly traded firm, indicated to the board that it may have to drop its Alzheimer's research program if it does not receive the $20 million from CIRM.

 The board's executive session followed rejection of another Alzheimer's related application, this one from researchers at USC.

USC Loses Appeal on $20 Million Proposal

Directors of the California stem cell agency this evening rejected an application for a $20 million research grant to a team at the University of the Southern California. 

The team, led by Roberta Diaz Brinton, filed an appeal last month in the $200 million disease team round, which first came up for consideration in July. CIRM staff pointed out that normally such appeals are considered at the time the round is first considered by the board.

The vote was split on the application, but actual tally was not announced by the agency following the vote. (Later, at the request of the California Stem Cell Report, a board spokesman said the vote was 4-10 on the 29-member board.)

Brinton and other investigators spoke to the board along with several patient advocates.

(An earlier version of this item did not contain the 4-10 vote.)

UCLA's Nelson and Miceli Win $6 Million from California Stem Cell Agency

Directors of the California stem cell agency today approved a $6 million research grant for Stanley F. Nelson and M. Carrie Miceli  of UCLA, who appealed a negative decision  by grant reviewers on its earlier $20 million request.

Following a re-review of the proposal, a special CIRM panel recommended the lower amount and conversion of the proposal to an early translational grant instead of a disease team. The UCLA team will be asked to send a revised proposal to CIRM prior to funding.

Nelson told the board that he was "okay" with the revision and said his team would be back later for more funding for a larger project.

Capricor Wins $20 Million from California Stem Cell Agency

Directors of the California stem cell agency this evening approved an award of up to $20 million to Capricor, Inc., of Los Angeles, rejecting a negative decision on the application by agency reviewers.

Approval of the application came after a re-review triggered by Capricor's appeal in July. The governing board also declined to impose a staff-recommended condition dealing with the patient cohort.

Appeals in $243 Million Stem Cell Round Climb to a Record 11

A Stanford researcher is seeking to overturn reviewer rejection of his application for $20 million in the California stem cell agency's largest ever research round and bringing to a record 11 the number of appeals in the round.

The previous record number of appeals in a round was nine in 2010. In the current $243 million disease team round, 11 of 15 applicants rejected by reviewers filed appeals, nine in July and two since then, including the Stanford researcher. Two of the appeals were successful in July. And it appears that three more appeals will be approved, albeit with conditions, later today. 

In his petition to the agency's governing board, Albert Wong of Stanford said,

“This science has been favorably reviewed by CIRM in the past and was on the cusp of funding. Moreover, three recent publications support our approach.”

He said that his team can “refute nearly every negative comment made either through referencing available scientific information or our application.”  Additionally he indicated that his team did not fully understand the opportunities for appeals during the application review process.

Wong and CIRM did not disclose the score on the application (5373), but it was listed next to last on the CIRM list of reviewer summaries.  Generally, those summaries are listed in order of their scientific score ranking.

Wong's appeal is on the agenda for today's meeting of the CIRM governing board, which is expected to act tonight on all the appeals filed in the $243 million disease team round. The $35 million basic biology round is expected to be acted on tomorrow.

Good News on Three Appeals; Not-so-Good News for StemCells Inc. and Klein

A Los Angeles biotech firm, Capricor, Inc., and a UC Irvine researcher, Henry Klassen, appear to be assured tonight of winning their appeals for nearly $20 million each from the California stem cell agency.

A research team at UCLA – Stanley F. Nelson and M. Carrie Miceli – also apparently won its appeal on its application but only on a substantially revised basis, according to a CIRM document. The agency indicated it would fund the grant but at reduced scope and cost – $6 million instead of $20 million.

However, StemCells, Inc., which was publicly supported by the former chairman of the stem cell agency, Robert Klein, lost its appeal for $20 million. The CIRM document said research cited by Klein as contradicting what reviewers identified as a key weakness did not contain “compelling data.”

A fifth applicant who appealed, Tim Hoey of OncoMed Pharmaceutical of Redwod City, Ca., was also rejected during the re-review process on a $20 million application.

The CIRM governing board, in July, sent all five applications back for reconsideration as a result of appeals of negative decisions by reviewers. The move followed a record-breaking level of appeals by researchers during an emotional meeting filled with testimony from patient advocates. The appeals came in a round that was budgeted originally for $243 million and that represents one of the agency's key efforts to commercialize stem cell research.

It was also the first time the agency's governing board has engaged in such an extensive re-review process on applications.

The revised recommendations for funding are scheduled to be acted on tonight at a meeting of the CIRM board in Burlingame, Ca. The panel has almost never rejected positive decisions by its review group and is likely to accept the latest recommendations. The board is deeply concerned about maintaining the integrity of the review process and not rejecting reviewer decisions without ample consideration.

The recommendations for funding on Capricor's application by Linda Marban, CEO of the firm, and the one from UC Irvine by Henry Klassen both contain conditions, but those probably will not stand in the way of acceptance by the applicants. (The executive chairman of Capricor is Frank Litvack, who last year was a candidate for chairman of the California stem cell agency.)

StemCells, Inc., of Newark, Ca., which is a publicly traded firm, had two applications in the disease team round. One dealing with spinal injuries was approved. However, the agency in its re-review of the second, dealing with Alzheimer's, said,

“The reviewers did not feel there was compelling data for neuron migration in the submitted manuscript. This is the manuscript specifically referenced at the ICOC (CIRM governing board) meeting (in July) that prompted the call for additional analysis. The manuscript is not yet accepted, it is 'potentially acceptable' but requires 'major revisions' according to the journal editor note. In addition, however, the studies in this manuscript used mouse NSCs, not the human NSCs proposed for the disease team award....”

In his pitch to the CIRM board, Klein said, “....(W)e have brand-new data that demonstrates and totally contradicts the key weakness on which it was downgraded.”

In the document prepared for the CIRM board, Ellen Feigal, senior vice president for research and development, discussed the re-review process and gave more details on the decisions. She said,

“In consultation with the Chair of the ICOC and CIRM scientific staff, the President and the Co-Vice Chair determined that the additional analysis should be conducted by the Review Chair of the GWG(grant review group), another scientific member of the review panel, and a patient advocate member of the GWG. The additional scientist reviewer was selected based on the expertise necessary to assess the new information. Each of the 3 individuals (chair, scientist, and patient advocate) voted on whether the information changed the funding recommendation by the GWG. A new score was not assigned."

Feigal continued,

“For each application, the information provided or referenced at the board meeting, and associated specific additional material were requested from the applicant. The new information was evaluated in all cases by the GWG Review Chair as well as one of the originally assigned reviewers and a patient advocate.”

Feigal's report does not identify the applicants by name – only by application number. Here is the number and name for those who do not want to wade through the CIRM web site to determine who is who: 5735 Capricor, 5739 Klassen, 5426 UCLA, 5352 Oncomed, 5416 StemCells, Inc.

The California Stem Cell Report will provide gavel-to-gavel coverage of tonight's and tomorrow's meeting of the CIRM board. The session will be audiocast live on the Internet. Interested parties can participate in the meeting at teleconference locations in Pleasanton, Los Angeles and La Jolla. The agency has added another way of listening to the proceedings -- a dial-in method using an 800 number. Details are on the agenda. 

USC Researchers Appeal Rejection of $20 Million Proposal

Researchers from the University of Southern California are making a pitch to overturn rejection of their $20 million grant application by reviewers in one of the signature commercialization rounds of the California stem cell agency.

The appeal by Roberta Diaz Brinton and Lon Schneider will be taken up one week from tomorrow by the governing board of the $3 billion state enterprise.

The USC application deals with Alzheimer's. It came in the $243 million disease team round that was considered last month during a record-breaking outpouring of appeals and a day of emotion-filled appearances by patients. CIRM directors adjourned their meeting without completing action on a number of items, leaving open the possibility of additional appeals such as the one from USC.

The Brinton-Schneider application received a score of 63 from reviewers. They said in a letter to the board,

“We are submitting the petition at this time as we are new to the CIRM ICOC(governing board) process and after listening to the July 26 ICOC meeting deliberations now understand that the petition process allows the ICOC to further consider our proposal. We noted that the proposal scored one point above ours and another two points below ours, each utilized the extraordinary petition strategy to gain ICOC review which resulted in funding approval in the former, and reconsideration in the latter instance.”

Their statement reinforced a concern expressed by CIRM Director Oswald Steward, director of the Reeve Center at UC Irvine,  at last month's board meeting about fairness in the grant process. He said,

“I'm not really quire sure that all of the applicants clearly understood that they could come back to us to address the criticisms(of reviewers).”

Concerns about whether all applicants fully understand the appeal process have surfaced on a number of occasions over the last several years. The CIRM board, however, is generally reluctant to overturn negative recommendations by reviewers. It also almost never reverses positive recommendations.

Next week the board is scheduled to make unspecified changes in the appeal process. No further details on those changes have yet been released by the agency although the meeting is just four business days away.

In the Brinton-Schneider letter to the CIRM board, the scientists defended their scientific approach and responded to criticism by reviewers, especially those related to sedation. Reviewers expressed reservations about over-sedation, which the researchers said were erroneous.

It is not clear whether other scientists will be making appeals during next week's board meeting.