Geron's Clinical Trial: Risk, Front-loading and Costs

The Vatican doesn't particularly care for the idea. "Unacceptable" was the word it used.

Over at Bioworld Today, it was "the dawn of a new era."

A reader of the New York Times warned of the danger of "front-loaded" arguments that can be detrimental to stem cell research.

What they were talking about is Geron and its re-vitalized clinical trial of an hESC therapy for spinal injuries. The FDA late this week lifted its hold on the effort. Andy Pollack of the New York Times said "cysts were discovered in some mice injected with the cells. Geron had to do another mouse study and develop better ways to check the purity of its cells."

Pollack wrote,

"Years of further testing will be required before the therapy would be proven and ready for widespread use, assuming it works."

Among the readers of Pollack's story was a person from Seattle who said,

"As in the case of most things in science, we just do not know enough to say which scientific modalities will bear fruit first if at all and when and hence multi-pronged approaches are needed. And the field of stem cell biology painfully realized a few years ago, some of these discoveries are fads that cannot be replicated by others ( I am referring to Catherine Verfaillie's assertion that adult stem cells can do most of what embryonic stem cells can do, a claim that has since been refuted/ withdrawn or generally agreed to be highly exaggerated at best). The damage done to stem cell biology from that fiasco is still evident. Hence most scientists would agree that front-loaded arguments for and against particular technologies are dangerous and can be detrimental to general scientific progress."

(Verfaillie is a CIRM grant reviewer.)

Over at UC Davis, stem cell researcher Paul Knoepfler, writing on his blog, called it a “milestone,” but noted that while the potential is high, the risk is very high as well.

Donna Young, Washington editor of Bioworld Today carried a detailed account concerning the trial. Her story began,

"The FDA's blessing Friday of the world's first clinical trial of a human embryonic stem cell (hESC) therapy has not only launched what may be 'the dawn of a new era' for medical treatment, but if successful, the therapies could alleviate much of the burdensome costs plaguing the nation's health care system, insisted Geron Corp. CEO Thomas Okarma."

Most of the news reports did not mention the seminal role of UC Irvine in the research leading to the clinical trial. You can find more details about that here on the Califoria Stem Cell Report.

Geron Go-Ahead Rouses Industry and Researchers

Geron Corp. said today that it hopes to begin within the next two months a clinical trial for an hESC therapy for spinal injuries, triggering a wave of ebullience from scientists, investors and patient advocates.

The company disclosed its plans following the lifting by the FDA of a hold on the effort.

Clive Cookson of the Financial Times of London caught the global significance. He wrote,

“The world’s first treatment based on human embryonic stem cells is set to begin a clinical trial within the next two months, in patients with acute spinal cord injury.”

For patient advocate Don Reed of Fremont, Ca., it was an emotional event. Writing on his blog, Reed said,

“The research behind this ground-breaking event was paid for a California law named after my paralyzed son, the Roman Reed Spinal Cord Injury Research Act of 1999.”
Reed continued,

“Christopher 'Superman' Reeve gave us his blessing, saying, 'One day, Roman and I will stand up from our wheelchairs, and walk away from them forever.' Cure did not come in time for our paralyzed champion, but we still believe in that great dream.”

Shares of Geron, a firm in Menlo Park, Ca., shot up 17 percent or 83 cents to $5.63. Analysts predicted skyrocketing share prices if the clinical trial proves successful.

Rob Waters and Molly Peterson of Bloomberg News interviewed Thomas Okarma, CEO of Geron. Bloomberg said,

"The FDA action strengthens Geron’s ability to start similar trials in the future, Okarma said. It also leaves regulators with a better grasp of how to review a completely new type of therapy.

"'It’s helped both of us to get up the learning curve together,' Okarma said. 'We’ve brought the FDA to a higher level of understanding which has been very positive and mutually beneficial. And phew, it’s about time.'"

Peterson and Bloomberg continued:

"Two other U.S. companies are working with embryonic stem cells. Advanced Cell Technology;of Marlborough, Massachusetts(and Los Angeles), is awaiting FDA approval to start a clinical trial in patients with Stargardt’s macular dystrophy, an eye disorder that causes severe vision loss.

"Viacyte, formerly known as Novocell Inc., a closely held San Diego company, is working on methods of turning embryonic stem cells into insulin-secreting cells for the treatment of diabetes."

Paul Knoepfler, a stem cell researcher at UC Davis, had predicted on his blog on July 1 that the FDA hold on Geron would be lifted “very soon.” Today he said,

"The hold was placed because of a 'higher frequency of cysts.' However many of us were wondering why the serious concern (and seemingly long hold) about non-tumorigenic cysts that were somewhat unremarkable in appearance. That remains unclear except that perhaps it was the "higher frequency" part. Despite being a bit puzzled about the length of the hold, I think in the long run caution makes sense."

CIRM President Alan Trounson said in a statement,

“This is an important milestone for the whole field to have an embryonic stem cell therapeutic in clinical trials. We are looking with hope and expectation that the transplant will be safe and effective.”

Geron is expected to be one of three firms (including Advanced Cell Technology) seeking funding from the California stem cell agency in its upcoming $50 million clinical trial round. The RFA was originally scheduled for May but was rescheduled for this month. CIRM appears likely to miss that target.

Pay Proposal for Patient Advocates on CIRM Board

The California stem cell agency is proposing to pay the 10 patient advocate members on its 29-member board of directors up to $15,000 a year for work performed in connection with their responsibilities for CIRM.

The move would implement some provisions of legislation that also removes the 50 person cap on the size of the CIRM staff. That legislation (SB1064) seems likely to be enacted this year. It is up for consideration in Sacamento next Wednesday by the Assembly Appropriations Committee.

Both the cap and the problem being addressed by the patient advocate proposal stem from Prop. 71, the ballot initiative that created CIRM and also wrote variety of minutia into law. The proposition requires a super-majority (65 percent) for a CIRM governing board quorum, which is based on the number of board members eligible to vote. Since many of the board members have conflicts of interest that prevent them voting on matters before the board, the presence of patient advocates is necessary to take action – much more so than many other members of the board.

A memo by James Harrison, outside counsel to the CIRM board, said,

"As a result of the requirements in Proposition 71, the Patient Advocate members of CIRM’s Governing Board are required to devote a substantial amount of time to the review of applications for research and facilities funding and the development of the standards pursuant to which research must be conducted. The time devoted to service on the Working Groups is above and beyond the time devoted to Board, subcommittee and task force meetings. In the aggregate, this service can seriously affect members’ ability to serve while simultaneously carrying out their other responsibilities, including their current occupations.

“Under the proposed bylaw, the Board would have the authority to set a daily consulting rate to compensate Patient Advocate members of the Grants Working Group and the Vice Chairs of the Facilities and Standards Working Groups for their service on the Working Groups. The Patient Advocates would continue to be limited to a per diem of approximately $116 per day for their service on the Board, Board subcommittees, and task forces. The proposed bylaw also addresses concerns that SB 1064 imposes no cap on the daily consulting rate the Board could set for Patient Advocate members of the Working Groups. Thus, it would impose a $15,000 annual cap and it would limit the daily rate to no more than 75% of the rate paid to scientific members of the Grants Working Group. In addition, the bylaw would require the Board to find that service on the Working Groups requires an extraordinary commitment of time.”

The CIRM directors' Governance Subcommittee will take up the pay plan at its meeting next Tuesday. Also on tap for the session are changes in outside contracting procedures. The agency is heavily reliant on contractors because of the 50 person staff limit.

The panel is additionally slated to discuss CIRM's grants management system, which has been a critical issue for the agency since 2007. A number of directors expressed concern at their June meeting about the staff decision to build a custom system, declaring that such efforts often have unfortunate outcomes. The staff report on the matter is not yet available to the public.

The public can take part in the subcommittee meeting at a number of teleconference locations throughout the state. The specific addresses can be found on the agenda.

Managing More than 300 Grants: CIRM's Thin Ice Question

A key panel of directors of the California stem cell agency on Tuesday will wrestle with ongoing issues involving its critical grants management system and consider changes in its policies concerning the many outside contractors CIRM is forced to rely on.

The agency has already posted some background information for the Governance Subcommittee meeting including a list of current contracts and the proposed changes in contracting. However, still missing is a report on the grants management system, which stirred strong concern from directors at their June meeting.

One director, Michael Friedman, CEO of the City of Hope, warned that CIRM was skating on “thin ice” with its plans to create its own software to monitor and track grants. Friedman said,

“Everybody has seen horrible examples of custom-designed systems that go bad.”

He continued,

“I have to express in the strongest possible terms my discomfort” with the CIRM staff decision to build a custom system.

Friedman's probing led CIRM directors to seek the report on the grants system for next week's meeting. CIRM plans to use the system for the entire grant process from application to termination. CIRM has awarded more than 300 grants involving more than $1 billion. It plans to hand out another $2 billion over the next seven years or so.

The grants management effort has a long history, dating back to 2007 when directors were told it would cost only $757,000. By our count, the agency has spent $1.2 million so far, not including staff time, with much more coming this year. CIRM has not publicly provided its own total nor has it forecast how much more will needed to be spent until the system is fully developed. Also missing are projected annual maintenance and service costs following that point.

For the current fiscal year, the CIRM budget forecasts that spending on information technology will jump by 53 percent, from $817,000 to $1.2 million, an increase of about $433,000. Most of that goes for the grant system.

Also on tap Tuesday are unspecified changes in the bylaws for the CIRM governing board.

Tuesday's meeting will have teleconference locations in San Francisco, Los Angeles, Sacramento, La Jolla, Irvine and Palo Alto. If you would like to attend any of those sessions, you should check the agenda for specific addresses. In some cases, you will need to call CIRM because it has not provided enough information on the agenda to let the public know exactly where the meetings will be held.

Jonny and His Stem Cell Breakdown

We all owe a debt of gratitude to the reader who posted an anonymous comment yesterday concerning the reality of life in stem cell labs.

The commentator poor-mouthed his brief note as “bit light.” But we think it is just what the field needs – a well-seasoned researcher engaged in educating the unwashed – sort of.

The matter involves Jonathan Garlick of Tufts. A light-hearted fellow, he is given to using an unusual method to discuss science. In this case, stem cell science.

Known as Dr. Jonny Cool J, Garlick has put together a six-minute rap on the virtues of the field. It has been viewed nearly 22,000 times.

“This Guy RULEEEES!!!” said one viewer. And now, here's Jonny.

Despite New CEO Job, Alan Lewis to Continue to Consult for CIRM

The California stem cell agency said this morning that Alan Lewis will continue to work as a consultant to the agency although he has what appears to be a full-time job as the new CEO of Ambit Biosciences of San Diego.

In the wake of the announcement of the new job, we queried CIRM earlier today about Lewis' status as the interim vice president for research and development for the $3 billion stem cell agency, an arrangement that began only on June 21.

Don Gibbons, CIRM's chief of communications, replied via a comment filed on our item that reported that Lewis had a new job. Gibbons said,

“CIRM's president was informed of Mr. Lewis' new position Friday. From the outset the $250,000 salary was to be prorated based on days actually worked with the full amount assuming a five-day week. With his new position Mr. Lewis will scale back his time with CIRM from a planned two to three days a week to 2 to 3 days a month, and the salary will be prorated accordingly, while we continue the search for a VP for R&D. “

Gibbons did not mention whether Lewis would continue to hold the title of interim vice president. Ambit and Lewis have not yet responded to our queries concerning his job with CIRM.

CIRM's New Interim VP for Research Takes Job with San Diego Firm

Only a little more than a month into his $250,000, six-month contract with CIRM, Alan Lewis has taken a job as CEO of the Ambit Biosciences of San Diego.

An anonymous reader alerted us this morning to Lewis' new post. (See the reader's comment to the left of this item under the heading “recent comments” or via the comments function on the “$250,000 Contract” item.) The reader makes the reasonable assumption that Lewis, former head of Novocell, will no longer be working part-time with the California stem cell agency. However, the full press release on Lewis' sort-of-new job does not mention the CIRM contract. We are asking both Lewis and the stem cell agency to clarify his status.

Lewis has served as executive chairman of Ambit since March, a fact not mentioned by CIRM when it announced his appointment as interim vice president of research and development. (Lewis told us that his executive chairmanship began in May. Today's Ambit news release says March.)

The anonymous reader also makes a good point concerning the amounts paid to CIRM contractors. The best talent costs money. But failing to effectively manage a $3 billion investment costs a lot more. Also readers should understand that the figures paid to independent contractors should be discounted by 35 to 50 percent if they are to be compared to conventional salaries. Standard pay figures do not include the cost of fringe benefits paid by employers, such as health insurance, retirement contributions, Social Security payments, etc. Depending on the company or government, those fringe benefits can run to 35 to 50 percent. In the case of individual contractors with CIRM, they must pay for those benefits themselves.

But regardless of the justification for spending large sums for outside contractors, California voters can be outraged by the amounts. Their reaction is visceral and emotional, a phenomena we discussed in this item. You can see examples of it on this blog on items dealing with salaries at CIRM and some outside contracts.

We also want to extend our personal thanks to the anonymous reader who brought Lewis' new job to our attention and that of our readers. We encourage comments from all readers, especially those bring fresh information to California stem cell matters.

A Matter for CIRM to Ponder: California's Visceral Reaction to High Salaries for Public Servants

Lavish government or quasi-government salaries are an anathema to California voters.

They reacted predictably with outrage last week when news surfaced about what they considered egregious greed. Jail time was urged. “Nauseating” was another word that was used.

The response is something to be considered by directors of the California stem cell agency, which has its own set of generous executive salaries. What is important here is what the voters perceive as lavish or greedy – not the perceptions of recipients or the perceptions of those who approve the pay.

One non-CIRM instance involved the city of Bell, a tiny, not particularly well-off enclave not too far south of downtown Los Angeles. It was there that the city manager resigned after it was disclosed by the Los Angeles Times that he was earning $800,000 a year. And that left him with a pension estimated at $600,000, going on $700,000. Times columnist Steve Lopez wrote,

“The sad reality, dear Californian, is that depending on where you live, you may be personally contributing to the insultingly fat pension of ousted Bell city administrator Robert "Ratso" Rizzo.”

The other instance involved the executive director of the non-profit California School Boards Association, which is based in Sacramento and supported by tax dollars that are paid to the association by school boards. The executive, who is mostly a lobbyist, retired after Sacramento television station KCRA disclosed that he made more than $540,000 two years ago, the most recent figure available.

“Piggies,” “unconscionable,” “retire him to the county jail” were some of the 129 comments filed on a Sacramento Bee story about the matter.

The salary scale at the $3 billion California stem cell agency tops out at more than $500,000. Amounts paid to its top executives have triggered harsh comments from a handful of observers. CIRM's contracts with outsiders have also been targeted. But the pay and contracts have received no widespread notice.

A good case can be made that the salaries at the highest levels of the stem cell agency are necessary and appropriate. But that makes little difference to citizens struggling with layoffs, cutbacks and wage roll backs. Even before the current dismal economic climate, they would froth and foam reflexively at what they regarded as excessive pay for public servants.

As CIRM aims at another pitch to the voters for billions more for research, its directors and executives should be preparing for a visceral and emotional outburst from citizens concerning its executives' pay.

CIRM Defends Legality of Barring Public From Marriott Meeting

The California stem cell agency says it did not violate state open meeting laws when it barred two members of the public from a meeting in June at a San Francisco hotel.

In an unsolicited note earlier this month to the California Stem Cell Report, Ian Sweedler, deputy legal counsel for CIRM, said,

“The Bagley-Keene Open Meeting Act applies to multimember boards or commissions, like the ICOC (CIRM directors) and its subcommittees, not to agency staff, or to other meetings that staff attend or organize in the operation of the agency, including the SCNT workshop.  We are always mindful of Bagley-Keene, and careful to follow it when it applies.”

We asked him to clarify his response, including whether CIRM's actions in this case violated recent changes in the California Constitution that guarantee the public a broadly construed right to information about the taxpayer's business.

Sweedler said,

“Sorry if I wasn’t clear.  Yes, I am stating, without qualification, that the SCNT workshop did not violate Bagley-Keene or Proposition 59(the change in the state Constitution), or any other law that I am aware of.  You had stated that a specific CIRM activity violated a specific law, and I thought it was important to correct that.  Thanks.”

We understand and respect Sweedler's opinion. However, it would take a court decision to determine whether he is correct. Our research indicates that a strong case can be made that such meetings must be open. Additionally, the state's open meeting laws were written well before CIRM, an agency unlike any other in state history, came into being. They must be reinterpreted not only in the wake of the Prop. 71 but also in the wake of a new, much broader constitutional guarantee of right of public access. The most important point, however, is that CIRM has pledged to go beyond the mere compliance with open meeting laws. As we noted earlier,

“Legalities aside, it is not in CIRM's best interest to bar persons from any of its sessions – not to mention that it is not in the best interests of the people of California. CIRM needs to do more than meet the minimum standards of the state's sunshine laws. To fail to do so will create a record that will surely harm CIRM's public support and hamper its efforts to secure more funding after it runs out of the $2 billion it has left to spend.”

As to whether CIRM adhered to the law, Sweedler confirmed that the meeting in question included the attendance of at least one alternate for a CIRM director, Jeannie Fontana. We also understand that some persons were in attendance who had not been invited directly by CIRM.

Those facts have an impact on whether the ban on the public is strictly legal. The First Amendment Coalition of California watches over the application of sunshine laws in California. The group says,

“California law doesn’t exactly ban deal-making by politicians in smoke-filled rooms, but it comes close to doing that at the local level (under the Brown Act) and among state agencies (under the Bagley-Keene Act). The fundamental idea behind these statutes is that the full process of political deliberating and decision-making—justly likened to the making of sausage—should be conducted in the open, in public meetings for all to see. Although the laws allow for certain matters to be considered in 'executive session,' they are the rare exception (in theory, at any rate).”

The coalition's Web site discusses cases (see here, here and here) that have application concerning the incident at the Marriott Hotel. They deal with the presence of a board member (or alternate in this case) at meetings such as those held by CIRM, as well as the possibility that they could lead to an illegal “serial meeting” of the CIRM board of directors even if only CIRM staffers were involved at the initial meeting.

In one case involving local open meeting laws, the First Amendment Coalition quoted the state attorney general as saying,

“In construing these terms, one should be mindful of the ultimate purposes of the Act — to provide the public with an opportunity to monitor and participate in decision-making processes of boards and commissions.  Conversations which advance or clarify a member’s understanding of an issue, or facilitate an agreement or compromise among members, or advance the ultimate resolution of an issue, are all examples of communications which contribute to the development of a concurrence as to action to be taken by the legislative body.”

In other words, such meetings should be open to the public.

Sweedler's statements go beyond CIRM's earlier comments. The stem cell agency previously contended that the meeting was closed to protect proprietary information, but failed to argue that open meeting laws did not apply. Don Gibbons, CIRM communications chief, said the meeting was designed “to gather information for critical decisions regarding the direction our funding should take.” To us, that sounds like something the public should have access to.

CIRM has virtually nothing to gain by barring the public. It is not as if hoards will descend on the sessions. Even full CIRM board meetings rarely attract more than 10 members of the public. Indeed, the agency is likely to benefit from comments that come from a different angle.

Correction

In two recent items, we misspelled the last name of a Duke University researcher. The correct spelling is Rob Wechsler-Reya. We had it incorrectly as Weschler.

Questions and Commentary on Stem Cell Reprogramming

The California stem cell agency and a UC Davis stem cell researcher are blogging on the implications of research that indicates that reprogrammed adult stem cells may not be the magic bullet that avoids the problems associated with human embryonic stem cells.

You can find a summary of some of the research and discussion from CIRM's Amy Adams here. Paul Knoepfler of UC Davis said,

"So if iPS cells are not that similar to ESC, what does this mean?

"First, it is not the end of the world. Many of us had assumed that iPS cells are similar to ESC in some ways, but distinct in others. These studies simply validate that notion.

"Second, iPS cells are still very useful even if they are not quite so close siblings of ESC. In fact, iPS cells do not need to be extremely similar to ESC to be useful. The pluripotency and self-renewal of iPS cells makes them a powerful tool regardless.

"Third, iPS cells are a distinct type of stem cell from ESC and should be considered as such.

"Fourth, these studies also highlight that every iPS cell line made is distinct from the others made. The heterogeneity amongst iPS cell lines is probably more substantial than the differences between ESC lines, but ESC lines also vary between each other quite a lot as well.

"Finally, the tumorigenicity of iPS cells is a serious concern. A remaining open question is how prone iPS cells are to form malignant tumors (although don't forget that teratoma can be malignant!). We predict that iPS cells are dramatically more prone to produce malignant tumors compared to ESC."

(Editor's note: An earlier version of this item contained typos in the material that was inserted from Knoepfler's original post. He corrected those typos on his blog, and we have altered his quote above to reflect those changes.)

Day 153 of the Wechsler-Reya Affair: Near Silence from All Quarters

California's $6 million, long-running courtship of a pair of Duke Blue Devils has apparently not been consummated.

But, like many other consummations, it is not easy to tell. Silence is the watchword on the part of those who know.

Last April, the California stem cell agency offered a $6 million signing bonus to Robert Wechsler-Reya, a researcher at Duke University, to move to La Jolla's Sanford-Burnham Institute. The award is part of a two-year, $44 million taxpayer-financed effort to help recruit scientists to California.

Sanford-Burnham is also providing “an extremely generous commitment of laboratory space, matching funds for equipment, additional support for relocation and research, and access to excellent core facilities,” CIRM grant reviewers said.

But the multimillion dollar enticement did not make the Duke pair jump with alacrity. Wechsler-Reya told the California Stem Cell Report last spring he had not made a decision. That situation seems to be unchanged today.

On Monday, we queried Wechsler-Reya about whether he and his wife, Tannishtha Reya, also a stem cell researcher, will take their work to Sanford-Burnham. So far he has not responded. CIRM remains mum as well, failing to answer our questions from Monday.

Andrea Moser, vice president for communications at Sanford-Burnham, however, did say something.

“At the current time, we do not have an update on the Wechsler-Reya recruitment.”

In April, the $3 billion stem cell agency tried to impose a June 30 deadline on the courtship. CIRM President Alan Trounson told directors that Sanford-Burnham had assured him the deal would be done by then.

One reason that directors wanted the matter settled was competition for more of the signing bonus cash. If the Duke researcher was not coming to California, more money would be available to help other institutions lure scientists to the Golden State. CIRM reviewers held a closed-door session last week to make decisions on those applications, but no news was forthcoming from that event.

So we are now into Day 153 of the Wechsler-Reya Affair. Stay tuned for all the developments.

(Editor's note: We started counting the days beginning with the Feb. 18 application deadline for the grant.)

(Wechsler-Reya photo)

Stem Cell Directors Postpone Action on Troubled Appeals Process

Directors of the California stem cell agency today delayed action on moves to reshape the “broken” grant appeal process at CIRM.

In response to a query about the outcome of the directors' Science Subcommittee, Don Gibbons, chief of CIRM communications, said,

“A lengthy discussion resulted in agreement to continue the discussion rather than take action at this time. It was generally agreed that any re-scoring request from the board should be a rarely used option.”

CIRM's board of directors has been bedeviled by problems with the appeals process for 2 1/2 years. The directors' new Science Subcommittee began to address the issue late in May.

More details on the directors' thinking on the problem will have to await the unveiling of the meeting transcript in about two weeks.

Biopolitical Times Questions Rationale for Future CIRM Funding

The case that California stem cell Chairman Robert Klein is trying to make for more funding for the $3 billion research effort rings hollow, according to the Center for Genetics and Society.

Writing on the group's Biopolitical Times blog, Jesse Reynolds, the Berkeley center's project director on biotechnology accountability, said that another ballot measure for stem cell research would fail. He said,

“...(I)nstead of the promised cures of the Proposition 71 campaign, reality-based voters see dramatic cuts in the state's essential services, liberalized federal funding of embryonic stem cell research, and a potential alternative in cellular reprogramming.”

Reynolds made his comments in the wake of a cover story in the San Francisco Examiner about the state of CIRM. Reynolds wrote that Klein “unsurprisingly defends its continued funding.”

Reynolds continued,

“But Klein’s arguments ring hollow. First, he cites an economic study that concludes the program has generated significant tax revenue. But that study's conclusions were controversial, and in any case $100 million is far less than the billion dollars the CIRM has already spent. The program is certainly not paying for itself, as he suggests now and as he claimed before the vote on Proposition 71.

“Second, Klein cites reduced health care costs. He goes so far as to say, 'First of all, we’re saving lives.' While I sincerely hope that embryonic stem cell research leads to therapies, that is not yet the case. Clinical trials are yet to begin. (Maybe next year?) Until there are genuine therapies, such savings remain hypothetical.”

CIRM Seeks Communications Coordinator

The California stem cell agency is still looking for a “communications outreach coordinator” to help beef up its public relations efforts.

The agency has re-posted the RFP for the $85,000, 12-month job after apparently not being satisfied with the earlier round of applicants. The part-time position also includes a $10,000 travel budget. The winning applicant will be expected to work 20 to 30 hours a week.

Among the responsibilities for the position are three CIRM town forums, Stem Cell Awareness Day, relations with patient advocacy groups and assistance in developing material for use in advocacy group meetings.

Deadline for applications is July 30.

UC Davis Scientist Praises CIRM Appeals Change

A stem cell researcher at UC Davis today said a change in the CIRM grant appeals procedure makes “a lot of sense.”

Writing on his blog in regard to "extraordinary petitions," Paul Knoepfler said,

“I think the proposed change makes a lot of sense and would greatly improve the process. Sometimes the reasons in the petitions are clearly not meritorious and as it now stands, they end up wasting CIRM's time. The last time CIRM received 9 petitions as well, which represented a remarkably large fraction of the total applications. A stricter process would discourage the submission of large numbers of petitions, an important issue given that the number of petitions received by CIRM continues to grow.

“As someone who has submitted dozens of grant applications over the years to many different funding agencies, I know how it feels to get applications unfunded and receive critiques I may not agree with. It is not fun. It is especially difficult when one is near but below the funding cutoff. However, it happens all the time.

“I think CIRM might consider a bit stronger language in their policy on appeals making it clear that appeals should only be filed in very limited circumstances and that appeals found to be without merit will be frowned upon.

“In other words, don't file an appeal simply because the rules theoretically allow you to and/or you are grumpy about how your grant application was received. Such frivolous appeals may affect your reputation.”

Knoepfler also received a comment from Jeff Sheehy, chairman of the CIRM Science Subcommittee, which will be considering the changes. Sheehy said,

“One thing that I think confuses folks is the lack of control we have over what comes to the Board.  As a public body, we're governed by State open government-Bagley-Keene rules (which I strongly support, btw).

“On any matter under consideration by the Board, any member of the public can speak to the matter or provide documents to Boardmembers.  Within that legal reality, we can only channel and not reject 'extraordinary petitions.'”

Whither Wechsler-Reya? A $6 Million Stem Cell Tale

Is a high-flying stem cell researcher from Duke coming to California to claim his $6 million grant from the state's $3 billion stem cell agency?

Can the prestigious Sanford-Burnham Institute in tony La Jolla, Ca., lure him and his wife away from their spots at the Gothic Wonderland, as Duke University is sometimes known?

The answers to these and other questions are still a mystery despite a quasi-deadline for the giveaway imposed by the 29 directors of the Californa Institute for Regenerative Medicine.

The will-they or won't-they matter involves Robert Wechsler-Reya and Tannishtha Reya, another Duke researcher.

The couple is being or has been recruited by Sanford-Burnham with the help of the multimillion dollar grant from California taxpayers. Directors of the state stem cell agency approved the award in April.

At the time, directors appeared to be initially unaware that the deal was not done. Wechsler-Reya told the California Stem Cell Report that he had not made up his mind on whether he would become a latter-day California argonaut.

According to the transcript of the April 29 CIRM board meeting, Chairman Robert Klein said he wanted a “definitive time” for conclusion of the Wechsler-Reya recruitment. CIRM President Alan Trounson said that Sanford-Burnham said it would be finished by the end of June. He told directors,

“We're reassured that that will be the case.”

Sanford-Burnham has announced other hires this month, but did not mention the Duke researchers. Earlier this month, we asked CIRM about the situation.

Amy Adams, communications manager for the agency, replied on July 1,

“The deadline in the RFA is for funding to start within six months after ICOC approval. At the ICOC meeting, there was agreement that it would be desirable to know Dr. Wechsler-Reya's decision before the next review so that we know how many slots remain, and the hiring institution agreed that would be reasonable. It was also stated that we hoped to have the recruitment resolved by the end of June, which has not happened.”

Wechsler-Reya has not responded to a request for comment. We are asking both him and CIRM again about the matter.

CIRM Finally Discloses Grant Appeal Proposals

The California stem cell agency early today belatedly posted a two-page memo on proposed changes in how it will deal with appeals by scientists whose grant applications have been rejected by reviewers.

The information comes only one business day before a meeting of the CIRM directors' Science Subcommittee to consider alterations in the appeals process. The changes deal with an issue that has been publicly troubling the CIRM board for 2 1/2 years. Many CIRM directors are unhappy with a situation that finds researchers publicly appearing before the board to press their cases. One director has described the process as “broken,” a description that others would likely use.

One proposed change provides for “re-scoring” of applications when directors are unable to make an "informed decision" at the board meeting at which an application is considered. The re-scoring would occur when “a material dispute of fact exists or when new information that is not available at the time of the initial review has come to light.”

The staff memo said,

“Programmatic issues, such as whether the agency’s portfolio is well-balanced among diseases, should not be a justification for re-scoring, nor should clear errors in the review of an application that have been identified by staff and presented to the board.”

Other changes would stipulate that CIRM staff would not respond in writing to the board on “extraordinary petitions” unless they believe the petition has merit.

Applicants would be advised that they may submit a petition – which is tantamount to an appeal – five days before the CIRM board is to consider their application “if they believe a material error or omission in the review of their application may have affected the recommendation made by the Grants Working Group to the board.”

Staff summaries of reviewers' recommendations would also cover the “majority views and the minority views, when there is a major difference of scientific opinion between large blocks of reviewers (e.g. eight speaking against and five in favor).”

As we have noted, it is a dismal commentary on CIRM that it has taken the agency until now to post the two-page document on the proposed changes. The agency cannot expect well-considered comments or questions from the hundreds of affected California scientists when it discloses information only one business day prior to the meeting at which a matter is to be considered. Indeed, we suspect that most California stem cell researchers are not even aware that CIRM is considering changes tomorrow that could affect their livelihood and work. That is not to mention that CIRM's belated posting does not come close to meeting the pledge of CIRM Chairman Robert Klein that the $3 billion research effort will adhere to the highest standards of openness and transparency.

Sticky, Troubling Appeals by Rejected Researchers Targeted by Stem Cell Agency

A key step in the process for awarding billions of dollars in research grants is “broken,” according to many directors of the California stem cell agency, and major changes are looming that will affect hundreds of scientists.

Just what those changes are likely to be is unclear. The stem cell agency has not made available to the public any drafts of likely proposals or other related material for a meeting on the matter that begins in only one business day. The only information on the agenda for the directors' Science Subcommittee says: “consideration of changes to extraordinary petition and grant administration appeals processes.”

(Editor's note: Hours after this item was posted, CIRM added material to its agenda that outlined the proposed changes. You can find more on the plan here.)

Nonetheless, specific proposals are virtually certain to come out of the session. Those would then go to the full board meeting on Aug. 18 for ratification and perhaps modification.

Many CIRM directors have long been uncomfortable dealing with public appeals by applicants rejected by the agency's grant review group. On the first such occasion in January 2008, Gerald Levey, a CIRM director and then dean of the UCLA Medical School, said, "I don't think we can run a board this way. If we do, it would be chaos."

Eight months later, directors approved the use of “extraordinary petitions” by applicants. However, that has not eased the situation. Last month directors were hit with a record nine petitions and overturned negative decisions by reviewers on four grants, also a record.

The Science Subcommittee first dealt with the appeals process on May 25. According to the transcript, the panel's chairman, Jeff Sheehy, a patient advocate and communications manager at UC San Francisco, said during the brief discussion,

“This is a broken process. I think almost everybody on the board would agree that it is.”

Duane Roth, co-vice chairman of the CIRM board and a San Diego businessman, said,

"This is one that I think really needs to be rethought completely because what we have right now doesn't seem to be working. Even the extraordinary petitions, I think with the exception potentially of one which we responded to, we agreed with the reviewers. And then in the board meetings turned around and disagreed with our own disagreement. And I think we just don't want to do that.

"Second, I think we're inviting very bad precedent behavior by opening ourselves up to the kinds of things that we went through at the last meeting. We just invite people to continue to do that. I'd like to see us find a solution for grievances on the reviews that everybody can get behind and endorse and embrace."

Among the suggestions in May was one from Sheehy to create a “re-review” option. Facing an appeal, directors could send an application back to the review group for re-evaluation in the light of comments from an applicant. CIRM directors almost certainly already have that authority although it has never been used or formally articulated. Prop. 71 makes the CIRM board the final arbiter on applications. Directors can do anything they want with an application. In practice, however, the grant review group makes the de facto decisions with the board intervening in only a tiny percentage of cases.

Sheehy, who is also a member of the grants review group, additionally said he would like the bifurcated appeal process consolidated. Currently, an “appeal” can only be made on the grounds of a conflict of interest. However, those reviews are done behind closed doors and applicants do not know the identity of reviewers charged with close evaluation of their proposals. Conflict appeals are handled privately by CIRM staff and do not normally come before the full board. Extraordinary petitions are technically not appeals, according to CIRM, but in reality serve that purpose.

Complicating the whole matter is the fact that any scientist or member of the public can speak directly to the board at its meetings on any matter, including rejection of applications. They can also write or email the directors. That is a matter of law and cannot be changed by CIRM rules.

At the May subcommittee meeting, CIRM President Alan Trounson discussed the public appearances of researchers. He said,

"It makes it very difficult on the spot to try and provide the board with the kind of information that they need."

Trounson also said the schedule for petitions is tight and comes only days before a board meeting, making it difficult for CIRM staff to evaluate the issues that are raised. However, some petitions are being presented to the board even though they have not met the deadline for submission.

Art Torres, co-vice chairman of the board and a former state lawmaker, suggested that applicants sign a document stating that they have read the rules for filing petitions. CIRM Chairman Robert Klein concurred with that suggestion.

Whatever comes out of Tuesday's meeting, it is a dismal commentary on CIRM that it cannot or will not provide timely information to the public and its stakeholders about how it plans to handle this portion of $3 billion in taxpayers' business.

Here is a link to a reading list, prepared by the California Stem Cell Report, of articles and CIRM documents on appeals and extraordinary petitions.

New CIRM VP Lewis and Ambit Biosciences

The new, part-time interim vice president for research and development at the California stem cell agency, Alan Lewis, also is executive chairman of Ambit Biosciences, a privately held biotech firm in San Diego. That information was not included in CIRM 's news release on his role at the agency.

We asked Lewis by email about his work at Ambit and told him we would carry his responses verbatim. He responded directly and quickly. Here are the questions and his answers.

California Stem Cell Report (CSCR): When did you become executive chairman of Ambit?
Lewis: “Approx May, 2010."

CSCR: What responsibilities does that entail?
Lewis: “It is similar to being board chair except Ambit is seeking a new CEO so I am the 'sign off' person who speaks once a week to three key staff. Also responsible for helping to create board agendas, and I am a member of the compensation committee.”

CSCR:Is the position compensated? If so, how much?
Lewis: “Typical of biotech boards--approx $25k/year. This consists of an annual fee plus board meeting fee.”

CSCR: Do you plan to continue as executive chairman while you are serving as interim VP at CIRM?
Lewis: “Yes. It involves approx five meetings per year in San Diego, and will have less responsibility as soon as the new CEO joins, probably mid-later July this year.”

CSCR: Do you see any conflicts of interest of any sort involving CIRM and your role at Ambit?
Lewis: "No. Ambit has no interest in regenerative medicines or cancer stem cells. They are focused on kinase inhibitors for cancer and inflammatatory diseases.”

CSCR: Were you a candidate for the VP position as a fulltime employee at CIRM?
Lewis: “No.”

CSCR: Do you plan to seek any other position at CIRM or on the CIRM board in the future?
Lewis: “Not given this any thought – probably not.”