The IOM and the CIRM Point of View

The California stem cell agency today looked at the study that it paid $700,000 for and decided the glass was half full.

The agency's press release on the 124-page report by the prestigious Institute of Medicine was headlined, “Institute of Medicine Report Praises Stem Cell Agency as a 'Bold Social Innovation.'”

The report also recommended sweeping changes at the agency, including stripping its governing board of the power to approve individual grants, overhauling the board to install a majority of independent directors and fundamentally altering the role of the 10 patient advocates on the 29-member board.  

IOM Recommends Sweeping Changes at California Stem Cell Agency

A blue-ribbon study of the $3 billionCalifornia stem cell agency today said the program has “achieved many notable results,” but recommended sweeping changes to remove conflict of interest problems, clean up a troubling dual-executive arrangement and fundamentally change the nature of the governing board.

The recommendations from the 17-month study by the Institute of Medicine (IOM) would strip the board of its ability to approve individual grants, greatly strengthen the role of the agency's president, significantly alter the role of patient advocates on the governing board and engage the biotech industry more vigorously.

Harold Shapiro, chairman of
IOM-CIRM  panel
Princeton University Photo

Harold Shapiro, former president of Princeton University and chairman of the IOM study panel, said, “Overall, CIRM (the California Institute for Regenerative Medicine) has done a remarkably good job” in giving the state a prominent position in regenerative medicine. But he said the stem cell field has “evolved”and CIRM needs to change with it.

As for turning research into cures, the report said,

 “The challenge of moving its research programs closer to the clinic and California’s large biotechnology sector is certainly on CIRM’s agenda, but substantial achievements in this arena remain to be made.”

Asked for comment, J.T. Thomas, chairman of the CIRM governing board, said it was premature to offer an opinion on the report, which will be presented to directors Dec. 12 at their Los Angeles meeting. (See here for the full text of Thomas' remarks.)

The study was conducted at the behest of CIRM, which paid the IOM $700,000. The IOM is a prestigious non-profit organization that was created in 1970 to provide authoritative advice to policy makers and the public.

In 2010, when directors authorized the study, Robert Klein, then chairman of the CIRM board, and other board members said that they hoped the study would lead to another multibillion dollar state bond issue to support the agency(see here and here). Duane Roth, a San Diego businessman and co-vice chairman of the CIRM governing board, was the lone no vote on the study. He warned directors that that they could not “go in just sort of blind trust that (the IOM is) going to reach the conclusion you want them to reach.”

The agency will run out cash for new grants in four years. Currently California remains in the throes of state budgetary problems, and the agency has put on hold talk of another bond election. It has also broached the possibility of seeking private funding.

The IOM report said the agency should develop a full-blown “sustainability platform” and plans that would spell out its likely financial structure and future rules on grants and their administration, including intellectual property.

The study echoed concerns and complaints about CIRM's operation that were aired even before the agency was officially created by voters in 2004. One of those involves the built-in conflicts of interests on the CIRM governing board. As of September, 92 percent of the $1.7 billion handed out by the agency had gone to institutions linked to persons serving on the 29-member board.

The report said,

“Far too many board mem­bers represent organizations that receive CIRM funding or benefit from that funding. These com­peting personal and professional interests com­promise the perceived independence of the ICOC(the CIRM governing board), introduce potential bias into the board’s decision making, and threaten to undermine confidence in the board.”

The IOM cited an ongoing scandal in Texas dealing with that state's $3 billion cancer agency. The flap has led to mass resignations of the agency's grant reviewers. The IOM said,

“Recent controversy surrounding the Cancer Prevention and Research Institute of Texas grants process illustrates the importance of rigorous scientific review free from inherent or perceived conflict and the consequences when these boundaries appear to be breached.”

However, the IOM press release said,

“Because the committee was not charged with reviewing CIRM's past funding decisions, it did not identify any specific cases of conflict.”

The IOM surveyed members of the board (ICOC) about conflicts of interest. The report said,

 "While a majority of respondents stated that personal interests did not play a role in their work on the ICOC, some responses were more equivocal. One respondent replied that it was 'hard to tell' given that 'so many decisions take place off camera in secret meetings,' while another acknowledged that ICOC members are human, and of course their decisions are influenced by personal beliefs and interests."

To help deal with conflicts of interest, the IOM recommended that the CIRM governing board not be allowed to approve individual grants. Instead, the board would be given a slate of applications that would be approved as a block. All CIRM board members would be removed from the grant review committee and the grant review process would be turned over to the president of the agency, currently Alan Trounson.

The IOM recommended that a majority of the board consist of “independent” members and said that the board should not be increased beyond its current 29 members, although it could be shrunk.

Conflict of interest rules should be revised to deal with personal conflicts, which could have a major impact on the 10 patient advocate members of the board but also other directors and possibly staff who have family members with health issues. The report said,

“California law focuses primarily on financial conflicts of interest, but the committee believes that personal conflicts of interest arising from one’s own or a family member’s affliction with a particular disease or advocacy on behalf of a particular disease also can create bias for board members. Studies in psychology and behavioral economics show that conflict of interest leads to unconscious and unintentional 'self-serving bias' and to a 'bias blind spot' that prevents recognition of one’s own bias. Bias distorts evaluation of evidence and assessment of what is fair.”

The IOM said that the board is much too involved in operational matters, including the chair and the two vice-chairs. The report said,

“The board should transfer management responsibilities to management so it can provide truly independent oversight and evaluation of management, strategic planning, and broad direction for resource allocation.”

The IOM repeatedly and favorably cited a 2009 study by California's Little Hoover Commission, the state's good government agency. It noted that CIRM rejected most of the commission's recommendations. The IOM also cited recommendations by the agency's own “external review” panel in 2010 and suggestions this year from the first performance audit of the agency, which cost CIRM $234,944.

Many of the IOM's recommendations would require either legislative approval or another ballot initiative. However, changes in the Prop. 71, the ballot initiative that created CIRM, require a politically difficult super, supermajority vote (70 percent) of the both houses of the legislature and the signature of the governor. The requirement was written into the 10,000-word initiative and has been used by CIRM to block legislation that it did not favor.

Here is brief rundown on some of the other IOM recommendations:

• Greater engagement with industry to commercialize stem cell research. Noting that industry has received only 6 percent of the agency grants, the report said business representation on CIRM working groups and other committees “should be enhanced to leverage industry’s expertise and resources in product development, manufacturing, and regulatory approval in support of the ultimate goal of bringing therapies to patients.”
• Elimination of the current process in which applicants rejected by reviewers appeal publicly to the governing board. Noting that 32 percent of “extraordinary petitions” have been successful, the report said they “undermine the credibility and independent work” of grant reviewers. Instead appeals would heard only by staff behind closed doors.
• Creation of a new scientific advisory board, appointed by the CIRM president with a majority from outside of California, instead of multiple advisory groups. The report said,“Such an external board would be invaluable in vetting ideas for new RFAs, suggesting RFAs that otherwise would not have been considered, and helping CIRM maintain an appropriate balance in its research portfolio. Input from this board would help CIRM make fundamental decisions about dealing with challenges that cut across particular diseases, decide which discoveries should progress toward the clinic, and determine how best to engage industry partners in developing new therapies.”
• Funding of programs on bioethics and regulatory problems. The report said,“It is difficult for researchers to find appropriate funding for stem cell-specific ethics and policy work, and filling this funding gap is well within CIRM’s budget.”

One final note: As mentioned earlier, Duane Roth, co-vice chairman of the agency, was the only no vote on the board when it authorized the IOM study in 2010. The IOM today said,

“The critical tasks performed by the vice chairs should be reassigned to management. In particular, the important tasks of government relations and corporate relations both should be carried out by staff reporting to the president rather than by the vice chairs of the board.”

For more excerpts from the report, see this item.

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Text of CIRM Chairman's Comments on the IOM Report

Here is the text of comments on the IOM study of CIRM from J.T. Thomas, chairman of the agency. 

"We deeply appreciate all the hard work of the IOM committee in compiling  long and detailed report and the IOM clearly put considerable thought into compiling it. This has just been released so our Board and our staff has not had a chance to look at it yet, let alone digest its findings and recommendations, so it’s premature for us to offer any opinions. We are looking forward to the IOM presentation at the next meeting of our board, the Independent Citizens Oversight Committee (ICOC) where we’ll have a chance to talk with the IOM directly about the report. After that we’ll put together a process on how best to proceed so that we can respond in as thoughtful a manner to the recommendations as the IOM did in making them."

Excerpts from the IOM Report on the California Stem Cell agency

Here are excerpts from the $700,000 Institute of Medicine (IOM) report on the $3 billion California stem cell agency -- the California Institute of Regenerative Medicine (CIRM).

Overall Comments

“Improvements to CIRM’s governance structure, scientific program, and policies are critical to bet­ter serving California taxpayers who elected to devote funding to promote stem cell research in the state. The necessary changes outlined by the IOM committee, if enacted by the state and/or the institute, would help to instill confidence among the scientific community and California residents in the vital work that CIRM is accomplishing.”

“It is the committee’s judgment that overall, CIRM has done a very good job of initially establishing and then updating the strategic plans that have set priorities for and guided its programs, and of taking advantage of its guaranteed flow of $300 million a year for 10 years to establish a sustainable position in regenerative medicine for California. The challenge of moving its research programs closer to the clinic and California’s large biotechnology sector is certainly on CIRM’s agenda, but substantial achievements in this arena remain to be made.

“Despite its demonstrable achievements to date, as well as the largely positive independent reports covering various aspects of its operations, no one would claim that CIRM is a perfect organization or that it should adhere slavishly to its initial form of organization, set of regulations, or pattern of priorities. The field of regenerative medicine has advanced rapidly since November 2004, and CIRM itself has seen the need to alter its activities and approaches in some areas. The committee believes the same should be true of its governance structure, some of its administrative practices, and its use of external perspectives on strategic scientific priorities and on the evaluation of other key policies, such as intellectual property, to ensure that they continue to encourage the development and deployment of new treatments.”

“While the restrictions on amending the administrative structure of CIRM established in Proposition 71 had the advantage of protecting the institute’s ongoing operations from outside interference in an ethically controversial arena, they also made it difficult to modify the organization’s structure in response to experience and/or changing circumstances. Moreover, these protections, whatever their benefits, appear to some to shield CIRM from the normal accountability mechanisms in place for state agencies.”

Conflicts of Interest

“Far too many board mem­bers represent organizations that receive CIRM funding or benefit from that funding. These com­peting personal and professional interests com­promise the perceived independence of the ICOC, introduce potential bias into the board’s decision making, and threaten to undermine confidence in the board. Neither the board chair nor board members should serve on any working group. The board itself should include representatives of the diverse constituencies that have an interest in stem cell research, but no institution or organiza­tion should be guaranteed a seat.”

“The problematic perception of conflicts of interest has persisted for as long as CIRM has existed. The IOM committee would be less concerned about individual board members with actual or perceived conflicts of interest if the board membership included more truly independent members. The majority of board members should be independent, with no competing or conflicting personal or professional interest. Broader representation from a wider variety of stakeholders will inject new perspectives into the panel and will help to dispel the perception of conflicts of interest.

“CIRM also should revise its conflict of interest definitions to include non-financial interests, such as the potential for personal conflicts of interest to arise from one’s own affliction with a disease or personal advocacy on behalf of that disease. CIRM policies for managing conflicts of interest should apply to that broader definition.”

Structure and Governance

“Currently, the ICOC (the agency's governing board) functions both as an executor and as an overseer—competing duties that compromise the ICOC’s critical role of pro­viding independent oversight and strategic direc­tion. The IOM committee recommends that CIRM’s operations be separated from its over­sight. The board should delegate more author­ity and responsibility for day-to-day affairs to the president and senior management, and the ICOC’s three working groups should report to senior management within CIRM, rather than to the ICOC. The moves would permit the board to better focus its energy and collective talent on strategic planning, overseeing financial perfor­mance, ensuring legal compliance, assessing the president’s performance, and devising a plan for preserving and expanding its considerable assets to permit the institute to continue its important work after the bond measures end.”

Unrealistic Goals

“While the latest round of awards challenge teams to have filed a request to begin clinical trials or to have completed early-stage trials in patients within four years, the committee feels these ambi­tious goals are unrealistic. New therapies take more time to progress to federal approval, and early-stage clinical trials are beset by a stagger­ingly high failure rate. Rather than judging suc­cess by simply tallying the number of active clini­cal trials, the IOM committee suggests that CIRM also continue its focus on underlying biological mechanisms that drive the success or failure of a promising therapy and on careful design of clini­cal trials. Advances in these areas will help the entire field progress, even if a specific drug candi­date is not approved." 

Economic Impact

“In the short term, CIRM’s expenditures are supporting approximately 3,400 jobs and their innovative efforts have also attracted substantial additional private and institutional resources to this research arena in California CIRM’s long-term impact on such critical aspects of the California economy as state tax revenues and health care costs beyond the shorter-term and temporary impact of its direct expenditures cannot be reliably estimated at this point in CIRM’s history.... (T)he estimate of the Analysis Group (2008) that the CIRM program alone would support about 3,400 jobs as long as it was allocating about $300 million per year in research and development grants appears quite reasonable to the committee. To put this estimate in context, however, total employment in California is roughly 16 million, and NIH alone provides more than $3.5 billion per year to California research institutions.”

Intellectual Property

“CIRM should propose regulations that specify who will have the power and authority to assert and enforce in the future rights retained by the state in CIRM-funded intellectual property. CIRM should seek to clarify which state agencies and actors will be responsible for the exercise of discretion currently allocated to CIRM and the ICOC (the CIRM governing board) over future determinations on issues regarding march-in rights, access plans, and revenue-sharing rights that might arise years after CIRM's initial funding period has passed.... (T)he ICOC should reconsider whether its goal of developing cures would be better served by harmonizing CIRM’s IP policies wherever possible with the more familiar policies of the Bayh-Dole Act(federal IP law).

Upcoming Tomorrow Morning: IOM Performance Review of the California Stem Cell

The $700,000 Institute of Medicine study of the California stem cell agency is scheduled to be released tomorrow at 10:30 a.m. Pacific Standard Time. The California Stem Cell Report will have full coverage of the 17-month study, including reaction from the stem cell agency and excerpts. Look for the stories here tomorrow morning.

Collapse of Big Pharma Deal Involving California Stem Cell Agency

A ballyhooed deal has blown apart that would have hooked up – for the first time – Big Pharma and the $3 billion California stem cell agency.

The breakdown of the arrangement was quietly disclosed yesterday in background material prepared for the Dec. 12 meeting of the stem cell agency's governing board.

The deal was first announced Oct. 25 when Viacyte, Inc., of San Diego, received a $10.1 million award to help finance a clinical trial for a diabetes treatment involving Viacyte and GlaxoSmithKline.

The CIRM background memo said this week, however,

“We have recently been informed that GSK was not able to obtain the final approval required due to business reasons in the context of GSK's overall research and development portfolio and investment needs and not as a result of any scientific or technical assessment of ViaCyte's program.”

The memo gave no further details about the Glaxo decision.

CIRM staff proposed that Viacyte, which has received $36 million from CIRM, be given another $3 million because Glaxo has exited the trial.

The arrangement involving Glaxo, Viacyte and CIRM was trumpeted in October, when Viacyte was awarded the $10 million. Officials of the stem cell agency said the award was a “watershed” for CIRM. Jason Gardner, head of the Glaxo stem cell unit and who attended the meeting, told the California Stem Cell Report that the arrangement was a partnership and that the company intended to develop a sustainable pipeline.

It was the second significant business-connected deal that has collapsed for the $3 billion agency within the last 13 months. In November 2011, Geron abandoned its clinical trial for spinal injuries. CIRM had loaned Geron $25 million for the trial just three months earlier. The company paid the money back with interest.

CIRM staff said that advisors to the agency remain “extremely positive” about the Viacyte research and “strongly recommended” that the company receive the additional $3 million. The memo said that trial has a “strong potential” to be commercialized.

Extra, Extra! CIRM Staffers Blog the World Stem Cell Summit

Years ago, I worked with an editor who used to advise his lagging scribes to put their noses in their typewriters and peck.

Well, the folks at the California stem cell agency have their noses in what passes today for typewriters and are pecking away furiously. Their subject is the World Stem Cell Summit, which has received only slight coverage in the mainstream media.

Today, the stem “cellists” from San Francisco's King Street filed -- on the agency's blog -- three fulsome items on doings at the summit, which is taking place in West Palm Beach, Fla. Yesterday they filed four. Photos and charts were included. More coverage is expected tomorrow.

CIRM staffers blogging the World Stem Cell Summit
covered UC Davis researcher Paul Knoepfler discussing
patient advocacy and its role in funding stem cell research. 

The CIRM writers are doing double-duty in at least one case. Geoff Lomax, the agency's senior officer for its standards group, is additionally speaking on a panel at the session. A handful of other CIRM officials are also appearing at the conference, which ends tomorrow.

The primary purpose, we presume, of sending state employees across the country is to gather the latest information on stem cell science and issues and to make contacts. It is a bit of a bonus for the public to have the CIRM attendees also file stories on the sessions.

A couple of the items caught my attention. One dealt with patient advocates and their role in energizing and helping to drive funding for research. Another item discussed what appear to be growing issues with dubious stem cell treatments and the damage they can do to the field in general.

Lomax summarized the signs of a stem cell scam like this:

• “Claims of miracle cures for diseases
• “Single treatments or cells that can treat any type of disease
• “Lack of objective information, evidence (such as published medical reports) that a treatment is effective
• “Treatment by a doctor who is not trained or certified to treat the specific disease
• “No system exists to collect information and follow up with patients”

$80 Million in Grants, Money for Viacyte and Blue-Ribbon Report on California Stem Cell Agency

Directors of the California stem cell agency are expected to give away $80 million next week to 20 fortunate researchers in addition to exploring a “commercialization and industry engagement plan.”

The subjects are on tap for the Dec. 12 meeting in Los Angeles of the governing board of the $3 billion research effort.

The $80 million grant round is aimed at “career development of physician scientists working in translational stem cell research.”

The RFA said

“This award will fund promising physician scientists in the critical early stages of their careers as independent investigators and faculty members establishing their own laboratories and programs.” 

Summaries of the grant reviewers comments and application scores should be available sometime this week. The bare-bones agenda lacked elaboration on the commercialization plan.

Directors are additionally scheduled to hear a presentation on the blue-ribbon report by the Institute of Medicine for which the agency is paying $700,000. The report has been 17 months in the making and is scheduled to be released this Thursday.

Other interesting matters are on the table, although the agency has yet to produce background material laying out any details. The subjects include:

• More money – no amount yet specified – for Viacyte, Inc, of San Diego, which has received more than $36 million from CIRM.
• An update of the agency's response to the only performance audit conducted at the agency. The audit identified 27 areas where improvement is needed, but the governing board has not discussed the results publicly since they were disclosed last May.
• Approval of the concept plan for another round of basic biology grants and adoption of conflict of interest code changes.

Interested parties will be able to take part at the meeting location in Los Angeles and teleconference locations in La Jolla, Oakland and UCSF. If you are interested in the teleconference locations, you will need to contact the agency for more specific directions than are provided on the agenda.

Update on Move To Curb Researcher Appeals at California Stem Cell Agency

Directors of the $3 billion California stem cell agency are still mulling details of changes in their free-wheeling and sometimes emotional appeals process for grant applications that are rejected by the agency's reviewers.

A special task force of directors met last week for the second time to discuss the likely alterations. Kevin McCormack, spokesman for the agency, said the group made no decisions. Another meeting will be held later at a date to be determined. The task force's recommendations will then go before the full board, probably in late January.

McCormack said members of the panel have asked for “more details regarding the process that would be employed if the appeals and extraordinary petition processes were merged.”

The agency has an odd, bifurcated appeals process. Early in its existence, the agency said appeals of reviewer decisions could be based only on conflicts of interest. However, researchers have a right under state law to speak to the governing board in public on any issue whatsoever. As some researchers began to use that avenue to ask for reconsideration of their applications, the CIRM board created what it called “extraordinary petitions” in an effort to control the process and limit appeals. Both the “appeals” and “extraordinary petitions” are, in fact, appeals but on different grounds and employing different mechanisms.

The task force was created in September after directors complained about “arm-twisting” and “emotionally charged presentations” in connection with a record number of appeals earlier this year.

Here is a link to an item about the task force's first meeting. Here is a link to an agency summary of the task force's deliberations prior to last week's meeting. The transcript of the session should be available on the CIRM web site within the next two weeks. It will be found under the meetings section of the web and then under the heading for the task force's November session.

Stem Cell Board Members Lubin and Sheehy Honored

A couple of members of the governing board of the $3 billion California stem cell agency were honored for their work this month.

Jeff Sheehy
UCSF Photo

One is Jeff Sheehy, a UCSF communications manager and nationally known HIV/AIDS advocate, who has served on the CIRM board since its inception. He was named by POZ magazine as one of the top 100 “soldiers” in the fight against HIV/AIDS.

CIRM's Amy Adams filed an item on Sheehy on the agency's blog yesterday. She said, 

“Jeff once told me that when he joined CIRM’s board eight years ago, he didn’t see a role for stem cells in an HIV/AIDS treatment. Now, CIRM has committed $40 million toward HIV/AIDS projects and two teams of researchers from City of Hope and UCLA are working toward clinical trials.”

Sheehy was also invited to the White House to commemorate World AIDs day Dec. 1.

Bert Lubin
Childrens Hospital Photo

Also honored was Bert Lubin, CEO of Childrens Hospital in Oakland, where he has worked since 1973, a remarkable achievement in today's world of transitory employment. The San Francisco Business Times named Lubin as the “most admiredCEO” in the San Francisco Bay Area. The newspaper said that since he took charge at Childrens in 2009,

“He recruited a new senior management team, chopped away at the pediatric hospital’s operating deficit and worked to heal relationships with the local community and governmental and political leaders that were deeply frayed...”

On a personal note, a friend who has long volunteered at Childrens gives him high marks as well, citing several encounters where he exceeded the usual CEO effort.

Correction

The Diane Winokur appointment item yesterday incorrectly stated that she is a current member of the governing board of the Sanford-Burnham Institute. That statement was based on information provided by the California stem cell agency and Golden West ALS chapter. Sanford-Burnham today said, however, that Winokur has not been on the board since last year. She served from 2005 to 2011.

Diane Winokur, Veteran ALS Patient Advocate, Named to California Stem Cell Agency Board

Longtime ALS patient advocate Diane Winokur of San Francisco, who has lost two sons to the disease, has been appointed to the governing board of the $3 billion California stem cell agency.

She fills the vacancy left by David Serrano Sewell, who resigned from the 29-member panel earlier this year after serving since the agency's inception. CIRM has 10 patient advocates on its board.

Diane Winokur
Photo -- Legal Momentum

Winokur is well-known in ALS circles. She sat for five years on the governing board of the national ALS advocacy group and currently serves on the board for the California state group, the Golden West chapter. She also served for six years on the board of trustees for the Sanford- Burnham Institute in La Jolla, which has received $37 million from the stem cell agency. She left Sanford in 2011.

Last summer Winokur appeared before the CIRM board to successfully seek approval of an $18 million ALS grant that was rejected by the agency's reviewers. Researcher Clive Svendsen of Cedars-Sinai in Los Angeles appealed the denial to the full board and was supported in emotional testimony by other patient advocates as well, including persons with the affliction.(See here for video of some of the testimony.)

The agency has awarded about $30.6 million, including the Svendsen grant, for research directly related to ALS.

Golden West issued a press release Nov. 21, lauding the Winokur appointment by California Lt. Gov. Gavin Newsom. The release quoted Lucie Bruijn, chief scientist of The ALS Association, as saying,

"Her contributions have been invaluable and she will be a tremendous asset in moving the ALS research field forward through CIRM funding."

The stem cell agency posted a blog item on Winokur's appointment in addition to a press release. CIRM Chairman J.T. Thomas said,

“Her knowledge, expertise and leadership will be a tremendous addition to the ICOC (the stem cell agency governing board) and help guide us in our work.”

Patient advocate Don Reed of Fremont, Ca., described Winokur in a 2008 blog item as “small, elegant, full of energy, an exclamation point of a person.”

(Editor's note: Based on information from CIRM and Golden West, an earlier version of this item incorrectly stated that Winokur is a current member of the Sanford-Burnham board.

Knoepfler Award Update: More than a Baker's Dozen Nominated

As of this week, 14 persons have been recommended for the Knoepfler “Stem Cell Person of the Year” Award.

The total was reported by Paul Knoepfler, the UC Davis stem cell scientist and blogger, who is putting up $1,000 for the winner of the honor.

Knoepfler announced his award plans earlier this month, declaring that he wanted to recognize scientists or others who “truly made a difference” in the stem field. Risk-taking is one important criteria.

Knoepfler said,

“It’s something that I’m hoping I can do every year. It would also be a reward for risk taking, creativity and be breaking with tradition and be something new in that regard.” 

The award has drawn some modest attention outside of Knoepfler's blog. A few days after he introduced the award on his blog, UC Davis decided to put out a press release and video on it. The California Stem Cell Report followed with an item. Then CIRM blogged it as well.

So far we have not detected any stories about the award in the mainstream media, but things could change.

Deadline for entries is Dec. 17.

California Stem Cell Agency Still in Talks on $40 Million for StemCells, Inc.

The California stem cell agency and StemCells, Inc., are still trying to reach agreement on a deal in which the company would receive $40 million from the state's taxpayer-funded research effort.

The sticking point is the $40 million in matching funds required from the Newark, Ca., firm under the terms of the two awards approved in July and September. The latter award was okayed on a 7-5 vote by the governing board after it was rejected twice by the agency's reviewers.

On Monday the California Stem Cell Report queried the agency about the status of the awards. Kevin McCormack, the agency's spokesman, replied,

“We are still in talks with them over the terms of the funding. Hopefully, we'll have an agreement soon.”

He did not elaborate further.

CIRM staff normally reviews applications after they are approved by the agency's governing board to be sure that all conditions are being met. However, in the case of the September award to StemCells, Inc., CIRM's governing board took the unusual step of publicly stating that the firm must demonstrate it has the $20 million in matching funds before it receives any payments from CIRM. The board did not take that sort of public position on the $20 million grant approved earlier in the summer, although matching funds are required in that case as well.

The former chairman of the $3 billion stem cell agency, Robert Klein, appeared twice before the board to lobby for approval of the second award to StemCells, Inc., which is a publicly traded firm.. It was the first such appearance by Klein before his former colleagues since leaving the agency in June 2011.

Action on the StemCells, Inc., awards attracted attention from the Los Angeles Times last month. Pulitzer Prize-winning columnist Michael Hiltzik wrote that the process was “redolent of cronyism.” He said a “charmed relationship” existed among StemCells, Inc., its “powerful friends” and the stem cell agency.

Texas Flap Looms Over California Stem Cell Agency's Grant Appeals

In nine days, the California stem cell agency plans to take another crack at finding ways to curb its free-wheeling appeal process involving scientists whose applications for millions have been rejected by reviewers.

It is a matter of considerable interest to researchers who need the cash to keep their labs running and remain in good standing with their host institutions.

The stem cell agency's governing board this fall created a task force to deal with the appeals issue after a record-breaking number of researchers made public appeals featuring emotional patient advocates. Even the former chairman of the agency, Robert Klein, made a two-time pitch for one applicant. Board members later complained publicly about “arm-twisting,” lobbying and“emotionally charged presentations.”

The agenda for the Nov. 30 task force meeting in Oakland -- with teleconferencing sites in San Francisco, Irvine, Palo Alto, Seattle and Rochester, N.Y. -- contains few clues on what the panel is hoping to specifically accomplish in next week's 90-minute session.

But interested researchers can check the transcript from the Oct. 24 meeting, during which CIRM President Alan Trounson described the problem as “very critical.” He said,

“I think this is a very serious matter that could really bite us very hard in a similar way to what's happened in Texas. Unless we come up with some kind of process that really addresses the science, it's a very large concern.”

Trounson's Texas reference was to the mass resignations of reviewers at that state's $3 billion cancer research effort. Questions have been raised about integrity of its grant review process and the program's political and biotech industry relationships. James Drew of the Dallas Morning News produced a bit of an overview this week. In another piece, Eric Berger of the Houston Chronicle provided quotes from emails from the infighting on a controversial $18 million grant.

Changes in California's grant appeal process may well be also discussed at the agency's board meeting Dec. 12 in Los Angeles. The board hopes to wrap up its action by late January.

Here is a link to an item with more specifics on material presented to the task force in October. Here is a link to an August 2012 list of articles and documents related to the CIRM appeals process.

Interested parties can address comments to the agency at info@cirm.ca.gov.   

Pomeroy Moving On, Will Leave Stem Cell Board

Claire Pomeroy, one of the longtime members of the governing board of the $3 billion California stem cell agency, will be leaving her position at UC Davis and the stem cell board next June.

Pomeroy yesterday announced her departure from Davis as vice chancellor for human health services and dean of the medical school. In a telephone interview, she told the California Stem Cell Report that she is examining a “few select opportunities” to work at a national level on health reform and health policy issues.

Claire Pomeroy
UC Davis photo

Pomeroy, 57, will be spending time in Washington, D.C., working on health issues on behalf of the University of California during the transition period before she leaves her position in California.

Pomeroy came to UC Davis 10 years ago, shortly before the Golden State's stem cell agency was created in 2004. At that time, UC Davis had what she called a “fledgling” stem cell research effort. Today the school has chalked up $128 million in grants from the stem cell agency, ranking fifth among institutions funded by the agency.

She said that creation of the stem cell agency “catalyzed development of our program,” which she said has risen to “national prominence.”

Pomeroy's service on the stem cell agency board was also instrumental in attracting a $100 million grant from the Moore Foundation to start a new school of nursing at UC Davis in 2009. Through her service on the board, she met Ed Penhoet, who also served on the board and was one of the co-founders of Chiron and then president of the Moore Foundation. Subsequently, Penhoet called her for lunch to discuss her thoughts on nursing education, and developments moved on from there.

The $100 million commitment was the nation's largest grant for nursing education, according to the Moore Foundation.

'The Knoepfler Award:' Recognizing Risk and Those Who Make a Difference

A UC Davis stem cell researcher-blogger has announced a “stem cell person of the year” award complete with a $1,000 cash prize that he is putting up himself.

Paul Knoepfler, who may be the only stem cell scientist in the U.S. actively blogging on the subject, said he has decided to put his money where his mouth is. 

Since announcing the contest in a Nov.13 blog item, Knoepfler has already received eight nominations, including one for a scientist. Three days after the item aappeared, UC Davis featured Knoepfler in a press release that included a video of Knoepfler explaining the effort.

Paul Knoepfler
UC Davis photo

He said he wanted to go beyond “old fashioned awards” given by “stodgy committees.” Knoepfler said he is seeking to recognize that stem cell research is “transcending the lab.”

The goal of the award, Knoepfler said, is “to advance the stem cell field and give credit to those who make a real difference.”

Knoepfler wrote,

“The criteria are that the person made a truly outstanding difference in the stem cell field for 2012. The winner could be a scientist, a patient advocate, someone in industry, a student, a physician…really anyone who has made the field better. For non-scientist nominees I’m particularly interested in those who took personal risks or gave of themselves to help others. For scientists I am looking for outstanding scientific achievement and in particular out-of-the-box thinking. Folks in any country are eligible.”

Deadline for nominations is Dec. 17. Self-nominations are permissible. Knoepfler plans to pick five finalists and interview them by phone. He also plans an online vote that he said  “may” influence his decision.

Complete details are available on Knoepfler's blog. 

California Stem Cell Agency Blogs on Geron Clinical Trial

The California stem cell agency published an article online last week concerning the hESC clinical trial that Geron abandoned last year, dealing mainly with one of the participants in the program.

The piece was studiously non-committal about whether the $3 billion research program is likely to fund the trial once again, should BioTime, Inc., of Alameda, Ca., be successful in acquiring the assets of once was the first hESC clinical trial in the United States. The agency loaned Geron $25 million a few months before the company cancelled the trial.

Amy Adams, CIRM's communications manager, simply wrote,

“They (BioTime) would need to apply for a loan if they want CIRM to financially support the continued trial.”

The latest round of funding that BioTime could apply for has a deadline of Dec. 18 for letters of intent. In addition to a loan, a grant is also a possibility.

Adams focused on Katie Sharify, who was enrolled in the clinical trial shortly before Geron said it was dropping the effort for financial reasons. Adams interviewed Sharify before an audience of scientists.

Adams wrote,

“Katie told me that it would be impossible not to hope that a trial would help her, but that by the time she made the decision to participate she knew she was doing it to further science, not necessarily to further her own recovery. She told the audience, 'I was part of something that was bigger than me, and bigger than all of you.'”

Stem cell scientist Paul Knoepfler of UC Davis also wrote about the BioTime-Geron deal last week. Noting that Geron's decision a year ago left many “upset to put it mildly,” Knoepfler said the “idea of BioTime buying the Geron stem cell program is a great one that provides new hope on many levels.”

Researcher Alert: Keeping Tabs on the Stem Cell Exchequer

The California stem cell agency has posted the dates for meetings of its board of directors for 2013 with most of the sessions scheduled for the San Francisco Bay Area.

One is expected to take place in San Diego in August, and another in Los Angeles 13 months from now. The other five are in Northern California. Not yet on the schedule is a board workshop in early January that will be open to the public.

Why is this of interest to researchers and others? The  board controls the purse strings to $3 billion for research grants and determines what areas are to be funded. Astute scientists would do well to take in the sessions. They offer insights into board thinking and opportunities to deal with the agency staff and directors on an informal basis. Only a handful of researchers – or less – attend the meetings on a regular basis, but have been well-served by the time spent. 

BioTime Will Have to Compete for California Cash for Geron's Dormant Clinical Trial

The California stem cell agency said today it does not plan to reactivate the $25 million loan to assist in Geron's spinal injury clinical trial despite an impending deal that would turn the effort over to BioTime, Inc.

Kevin McCormack, senior director for public communications for the agency, said BioTime will have to compete in an upcoming award round if it wants to win California dollars.

Responding to a question from the California Stem Cell Report, McCormack said,

“That (earlier) loan was specific to Geron and when the trial was ended the loan ended too. Of course if Biotime and Geron do complete their deal then Biotime would be free to apply to us for a new disease team grant.”

McCormack later added that BioTime could also compete in other appropriate rounds, including the strategic partnership round just posted by CIRM. It provides for four awards of up to $15 million. Funding could come as early as October of next year. The strategic partnership round is a business-friendly effort that is aimed at attracting “industry engagement and investment.” The deadline for letters of intent is Dec. 18.

The stem cell agency made its $25 million loan to Geron in 2011 just a few months before the Menlo Park firm abandoned its human embryonic stem cell trial for financial reasons. (The full text of the loan agreement can be found here.) The company has repaid the loan with interest.

The company has tried to sell the assets associated with the clinical trial since last November. The only public interest that has surfaced has come from BioTime, Inc., of Alameda, Ca. Michael West, founder of Geron, is the CEO of BioTime. Tom Okarma, CEO of Geron from 1999 to 2011, is CEO of the BioTime subsidiary that would assume the clinical trial.

News from clinical trial is expected to be published soon, according to a story in the San Francisco Business Times by Ron Leuty. He quoted CIRM President Alan Trounson as saying that “some findings” from the trial would be published next month in a medical journal.

Geron's stock traded at $1.24 at the time of this writing today, up from $1.21 yesterday. BioTime's stock stood at $2.99, up from $2.97.