Stem Cell Conflict-of-Interest Rules Sent Back for More Work

BURLINGAME, Ca. – Directors of the California stem cell agency today sidetracked minor changes in its conflict of interest rules for the scientists who review applications for millions of dollar in state funds.

The scientists score the applications, and their decisions have determined the fate of 98 percent of thousands of applications over the last nine years. The agency has given out $1.9 billion.

The proposed conflict changes were referred to the directors' Governance Subcommittee for possible alteration. The action came after two directors, Jeff Sheehy and Steve Juelsgaard, found fault with them for different reasons.

Sheehy objected to the proposal after the staff said the changes would  allow two situations at the agency which have been determined to be conflicts of interest. Juelsgaard was concerned about ambiguous definitions of personal and professional conflicts as well as protection of privacy.

Today's proposal was largely triggered by a conflict of interest earlier this year involving two internationally renown scientists: Lee Hood, president of the Institute for Systems Biology in Seattle, Wash., and Irv Weissman of Stanford, who are close friends and own a ranch together in Montana.

Hood was recruited by CIRM President Alan Trounson to serve as a scientific reviewer in a $40 million grant round involving Weissman. Trounson has been a guest of Weissman's at the ranch. One of the applications in the round involved Weissman, who could have received a payment of a few thousand dollars, and Stanford,which would would have been the site of a $24 million genomics facility.

The agency said it did not detect the relationship between Hood and Weissman until it was called to their attention by another reviewer who was also participating in the closed-door review of applications. The agency is conducting another review of the applications later this fall.

The other conflict case involved John Sladek of the University of Colorado.

The changes proposed today would create a threshold of $5,000 a year for conflicts involving salary or consulting fees. Less than that would not trigger a conflict situation. Other proposed changes to be taken up by the agency board involve personal and professional conflicts along with the nature of the economic disclosures that reviewers, all of whom are from out-of-state, must disclose privately to a handful of agency officials.

Wide-ranging Recommendations for California Stem Cell Agency

The California stem cell agency this morning released a Power Point summary of wide-ranging recommendations from its newly created scientific advisory board(SAB).

The recommendations will be discussed later today and again in December. Here is an excerpt from the lead recommendation.  The complete Power Point presentation can be found at the end of this item.

"SAB advises CIRM to identify, through a prioritization process, the top 6 to 8 projects, with clear relevance to the remit of CIRM’s stem cell mission, and to setaside the funding to ensure the projects can proceed to phase 1 and 2a clinical trials as rapidly as possible, without financial impediments. – Achieving clinical proof of concept is a key goal to achieve, to attract future potential investors and supporters of stem cell research, and has a strong chance of success, as long as CIRM advances the most promising clinical candidates “at speed”; this will require careful assessment / prioritization of portfolio.

"Preliminary management response: Management accepts this recommendation and will need to identify a process for selection of these projects that would include representatives from GWG(grant review group), CDAP(another agency advisory panel), and other external expertise as needed, and the amount of funding that would need to be set aside by the ICOC(CIRM directors). Recommendations will be developed for this priority group of projects as to where expertise and approach need to be modified to maximize the potential and to ensure rapid and effective progress. Management will provide separately a process to select these priority projects."

CIRM Scientific Advisory Board Recommendations

CIRM Directors Open Today's Meeting

Directors of the California stem cell agency have just begun their meeting. The Power Point presentation of the recommendations of the newly created scientific advisory board is now available and will be posted on this web site shortly.

Upcoming: Live Coverage of Today's California Stem Cell Meeting

BURLINGAME, Ca. -- The California Stem Cell Report will provide live, gavel-to-gavel coverage today of the meeting of  the governing board of the $3 billion California stem cell agency.

The meeting begins at 9 a.m. PDT. Stories will be filed as warranted throughout the day. On the agenda are the first recommendations from the agency's new scientific advisory board, an update on the agency's critical translational research program and changes in its conflict-of-interest rules for grant reviewers. At the time of this writing, neither the recommendations of the science advisors or the report on translational grants is available to the public.

For those who wish to follow the board action live, the agency is providing an Internet audiocast and a WebEx Internet connection. Full details are on the meeting agenda. For those who have not used either in the past, we recommend that you log on in advance of the 9 a.m. PDT meeting to work out any kinks.

Stem Cell Person of the Year Award: Nominees Now Named

Voting is underway around the world for the semi-finalists for the Stem Cell Person of 2013 award, and one of the top staff members of the California stem cell agency is in the running.

She is Patricia Olson, executive director of scientific activities for the $3 billion California Institute for Regenerative Medicine(CIRM). Olson is the only member of the unique agency's staff to have been nominated for the award, which is made by UC Davis stem cell researcher Paul Knoepfler. The award carries a $1,000 prize that comes out of Knoepfler's pocket.

Olson's name caught my eye because in many ways she represents the entire, tiny staff of the nine-year-old agency. Only a little more than 50 people work at the San Francisco enterprise, which is an unusual combination of science, academia, business and government. In its early years, the number of employees was even smaller, less than it would take to staff your average Burger King. Yet, the agency has given out $1.9 billion, acted on thousands of grant applications and overseen 570 awards. More are to come.

From the agency's start, many worried that the size of the staff would not be adequate to keep up, a question that can still be raised justifiably. But the CIRM staff has forged on, scrutinizing grantee work with more care than the mighty NIH, as we have noted in the past.

Olson is one of the few longtime veterans of the agency, joining it in 2006. She has acquired a set of skills and institutional knowledge that has well-served both CIRM and the stem cell field. Like many of us, she has a bit of a sharp edge at times. But it is exercised all for the sake of focusing diligently on stem cell science and efficiently executing the agency's mission.

Only 12 semi-finalists will emerge from the current voting, which concludes Oct. 16. Many worthwhile candidates are on the list. But as a representative of the CIRM staff, a vote for Pat Olson could provide a big high-five for her and all the “stem cellists” at their King Street headquarters and all the work that they have done -- not only this year, but for the last nine years.

You can cast your votes by going to this page on Knoepfler's blog.

New Grant Reviewers for California Stem Cell Agency

Five new scientific grant reviewers are expected to be approved tomorrow by directors of the California stem cell agency, and at least four have backgrounds that might make them judges in the upcoming $40 million stem cell genomics round.

They include one scientist who once held $7.4 million in grants from the California stem cell agency. He is Martin Pera of the University of Melbourne in Australia. Pera was the first head of the USC stem cell program, serving from 2006 to 2011. The program was launched in the wake of the passage of Prop. 71, which created California's $3 billion stem cell program.

According to the University of Melbourne, Pera has done a significant amount of research in the area of cytogentics and genome mapping.

Scientific grant reviewers for CIRM all come from out-of-state. Pera is likely the first former CIRM grant recipient to be selected as one of the reviewers who make 98 percent of the decisions on the agency's grant applications. None are required to disclose publicly their financial or professional interests.

Other proposed scientific reviewers include:
Bradley Bernstein of Massachusetts General Hospital and Harvard, who is co-director of the epigenomics program at the Broad Institute.

Richard Gibbs, director of the human genome sequencing center at the Baylor College of Medicine.

Barry Rosen of the Wellcome Trust in the United Kingdom, who has done work in genetics and genome engineering.

Steven Jon Russell of the Harvard Medical School, who works in diabetes and completed the first outpatient trial of a treatment device described as a closed-loop artificial pancreas blood glucose control system.

Others being reappointed are Shelly Heimfeld of Hutchinson Cancer Research Center, Ihor Lemischka of Mount Sinai and Thomas Zwaka, also of Baylor.

The CIRM staff document prepared for tomorrow's meeting contains additional information on all the scientists.

Live Coverage Tomorrow for Meeting of Directors of California Stem Cell Agency

The California Stem Cell Report will provide live, gavel-to-gavel coverage tomorrow from the Burlingame, Ca., meeting location of the governing board of the $3 billion California stem cell agency.

Stories will be filed as warranted throughout the day. On the agenda are the first recommendations from the agency's new scientific advisory board, an update on the agency's critical translational research program and changes in its conflict-of-interest rules for grant reviewers. At the time of this writing, neither the recommendations of the science advisors or the report on translational grants is available to the public.

For those who wish to follow the board action live, the agency is providing an Internet audiocast and a WebEx Internet connection. Full details are on the meeting agenda. For those who have not used either in the past, we recommend that you log on in advance of the 9 a.m. PDT meeting to work out any kinks.

Nobel Winner Ties to California Stem Cell Agency

The California stem cell agency can claim a connection to two of the winners today of the Nobel Prize in medicine, including one who was involved as far back as 2005.

Writing today on the agency's blog, Amy Adams, the agency's communications manager, said,

“Randy Schekman established the stem cell training program at UC Berkeley, which supports graduate and post-doctoral students working in stem cell labs.”

That grant was approved in September 2005 although the agency did not immediately have the cash to fund it.

Adams also wrote,

“Thomas Sudhoff (of Stanford) is a collaborator on a Tools and Technologies Award to Marius Wernig...In the collaboration with Wernig, the group intends to study defects in the normal signaling between neurons in people with autism, schizophrenia, depression and other diseases. Starting with stem cells developed from people with those diseases, they’ll be able to study the defects in cellular communication and then try to find ways of fixing those defects.” 

Brown Veto of Spinal Research Funds Triggers Ire

California Gov. Jerry Brown's veto of legislation backed by some stem cell patient advocates has triggered a sharp and negative reaction. One person said he was “disgusted” by the veto. Another person called the governor a “stingy rat.”

It was Roman Reed, a California patient advocate, who said he was disgusted and now planned to run for the state legislature to help find cures for paralyzed persons. Reed said in an interview with the blog ipscell.com:

“Curing the paralyzed and finding medical cures is the most important task of our lives for the health of our loved ones and the economy.  Some fights we just have to win and together we can.”

The legislation in question is AB714 by Assemblyman Bob Wieckowski, D-Fremont. It would have provided $1 million to UC Irvine's Reeve-Irvine Research Center, which has received $15.1 million from similar state appropriations over past years. The money has gone for research into spinal cord injuries and paralysis and has involved human embryonic stem cells. However, funding of the act has expired.

Brown said in his veto message that the measure “strives to do only good.” But the governor said that he has proposed $511 million in additional support for the University of California over the next four years. He indicated that it is now up to the UC system to decide whether it wants to use some of that additional cash for the spinal cord research.

UC Davis researcher Paul Knoepfler wrote about the veto on his blog and carried Reed's remarks. One of the persons commenting on the item identified himself as Brian Sanderson and said,

“I recall reading that Christopher Reeve(the actor who played Superman) said 'I wish I was a rat' when he heard of the progress that had been made repairing spinal cords of rats. Personally, I think that the Governor is behaving like a stingy rat.”

Reed was named as the Knoepfler Lab Stem Cell Person of 2012 for his lobbying efforts here and in Alabama as well as for his mentoring of other advocates. Reed is the son of another stem cell activist, Don Reed, who wrote about the veto on his blog, Stem Cell Battles. The legislation involved in the governor's veto is named after the younger Reed, who was paralyzed as the result of a football accident some years ago.

The legislation indirectly involves directors of the California stem cell agency. The head of the UCI Reeve Center, Oswald Steward, sits on the agency's governing board, along with Sue Bryant, interim provost of UCI, and Sherry Lansing, a former chair of the UC Board of Regents. Roman Reed also came up with the motto of the stem cell agency: “Turning stem cells into cures.”

Trounson to Make London Appearance

Alan Trounson, president of the California stem cell agency, is scheduled to speak later this month in London at King's College.

The event is slated for Oct. 28. Trounson will be hosted by Fiona Watt, who is a member of the stem cell agency's newly created scientific advisory board and director of the Center for Stem Cells and Regenerative Medicine at the school.

Trounson's topic is “The California Institute for Regenerative Medicine: How to invest $3 billion in stem cell research.”

Scientific Advisors Take First Look at $3 Billion California Stem Cell Agency

Directors of the California stem cell agency on Wednesday will hear the first recommendations from a newly formed scientific advisory board, created to provide “cohesive” advice that a blue-ribbon study said has been lacking at the agency.

Formation of the panel was recommended last December by the prestigious Institute of Medicine (IOM) in its $700,000 report on the performance­ of the stem cell agency. The IOM said a scientific advisory board would be invaluable in helping the agency to “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.”

The report from the eight-member panel is not yet available to the public although only two business days remain before the agency's governing board meeting in Burlingame, Ca.

The panel was created last July and has held at least one meeting, which was not noticed publicly. The members include only one Californian, Corey Goodman, co-founder of venBio, a San Francisco biotech venture capital firm. According to an article by Bernadette Tansey on Xconomy, his current advice to biotech industry executives is:

 “Don’t do what I did. That worked then—it won’t work now.”

The other scientific advisors are: Sir John Bell, Oxford University, Great Britain; Christine Mummery, Leiden University Medical Center, The Netherlands; Sean Morrison, Children’s Research Institute at UTSW, Texas; Stu Orkin, Harvard Medical School, Dana Farber Cancer Institute, Mass., and also a member of the IOM panel that studied CIRM; Fiona Watt, Centre for Stem Cells and Regenerative Medicine, King's College London; John Wagner, University of Minnesota Stem Cell Institute,who is also a member of the CIRM standards group, and Maria Grazia Roncarolo, San Raffaele-Telethon Institute for Gene Therapy (TIGET).
(More information on each member is available by clicking on their names.)

In response to queries from the California Stem Cell Report, Kevin McCormack, senior director of public communications, said that the members of the board will not receive compensation but will be reimbursed for their expenses. McCormack said that they have been screened for conflicts of interest.

CIRM, as the $3 billion stem cell agency is known, has not yet prepared a written description of its new advisors' duties. McCormack instead referred to the IOM's description of what the board should be doing.

The IOM recommended formation of “a single Scientific Advisory Board, as opposed to multiple advisory boards as proposed in the 2012 strategic plan, (that) would provide cohesive, longitudinal, and integrated advice to the president regarding strategic priorities, which is lacking in the current CIRM organizational structure."

At its July 25 meeting, the CIRM governing board was told by President Alan Trounson that the scientific advisory board “may or may not be supportive completely of our strategic plan, but it will be a recommendation we'll bring to the board for further discussions about how we orient ourselves.”

The IOM cited several areas where CIRM has “made strategic decisions that resulted in the omission of some important areas.” They included “addressing the novel ethical and regulatory aspects of clinical applications of potential stem cell therapies” and preparation of “academic institutions in California for collaboration with the private biotechnology or large pharmaceutical sectors.”

The IOM report said,

“(T)he notable absence of industry representatives on most disease teams demonstrates the inadequate emphasis of CIRM’s translational/development RFAs on what is needed to enable regulatory approval for cell-based therapies.”

Also scheduled for Wednesday's governing board meeting is a review of its translational grant portfolio. That report is also not yet available publicly. In the past, such reports were often limited to a Power Point outline and not available to the public until their presentations were underway during the board meeting.

Here is the text of the IOM's summary of its recommendation for creation of the scientific advisory board.

“CIRM proposes to create a Clinical Advisory Panel and Industry Advisory Board. Although the committee supports CIRM’s intent to establish advisory boards, it recommends that one Scientific Advisory Board be established. Striking the proper balance in research across the portfolio of basic, translational, and clinical studies will require that CIRM solicit broad input in executing its strategic plan. The committee believes the proposed Scientific Advisory Board could serve an invaluable role in this process.

“Recommendation 4-1. Establish a Scientific Advisory Board. CIRM should establish a single Scientific Advisory Board comprising individuals with expertise in the scientific, clinical, ethical, industry, and regulatory aspects of stem cell biology and cell-based therapies. A single Scientific Advisory Board, as opposed to multiple advisory boards as proposed in the 2012 strategic plan, would provide cohesive, longitudinal, and integrated advice to the president regarding strategic priorities, which is lacking in the current CIRM organizational structure. The majority of the members of the Scientific Advisory Board should be external to California, appointed by and reporting to the CIRM president. 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. The board’s reports and the president’s response to those reports should be delivered to the ICOC(the CIRM board) and discussed in sessions open to the public.”

Groundbreaking Ahead? Geron's Stem Cell Program Officially Goes to Biotime/Asteria

Geron's once-heralded stem cell therapy program -- financed at one point with $25 million from the California stem cell agency – has officially landed in the hands of an Alameda, Ca., enterprise involving two former CEOs of Geron.

Completion of the transaction was announced Tuesday by BioTime, which acquired the Geron assets. The move “could jump start groundbreaking but not yet clinically successful stem cell programs,” according to an article by Ron Leuty of the San Francisco Business Times. Leuty also outlined the complicated financial terms.

Geron, based in Menlo Park, Ca., was the first in the nation to launch a clinical trial for a human embryonic stem cell therapy. In 2011, in a major departure from its usual procedures, the stem cell agency loaned Geron $25 million to help with the trial. About three months after the loan agreement was signed on Aug 1, 2011, Geron announced it was abandoning the trial for financial reasons. Four patients had been enrolled in the trial.

Geron repaid the loan with $36,732.33 in interest, laid off 38 percent of its staff (66 persons connected to the stem cell program) and began an effort to sell off the stem cell effort.

About a year later, BioTime, which is headed by Michael West, who founded Geron, began expressing an interest. Tom Okarma, another Geron CEO, hooked up with West on the effort and is now head of Asteria Biotherapeutics, a BioTime subsidiary that is taking control of the old Geron stem cell program.

Geron let Okarma go in February 2011. He was head of the company as it plowed through the arduous FDA process to begin the clinical trial of the stem cell treatment for spinal injuries.

The stem cell agency has said that the loan to Geron is not transferable automatically to BioTime. The firm will have to compete for funding under the agency's established rules.

Both Geron and BioTime are publicly traded. In the last 12 months, Geron stock has traded at a range from 91 cents to $3.95, closing at $3.40 yesterday. BioTime has traded in a range from $2.67 to $5.02, closing at $3.88 yesterday.

As part of the deal with Geron, the stem cell agency received 537,893 warrants to buy Geron stock at $3.98 each. The warrants expire in 2021.

Here are other news articles on completion of the transaction, which was announced earlier this year: by Jef Akst at The Scientist, by Damian Garde at Fierce Biotech.

Trivializing Conflicts of Interest at a $3 Billion California State Agency

The California stem cell agency is planning minor changes in its conflict-of-interest rules that narrowly target a violation that arose last spring involving two internationally known scientists.

The proposal was triggered by a situation in which Lee Hood of Seattle, Wash., was recruited to serve as a scientist-member of the group that was reviewing applications in a $40 million grant round last spring. One of the applications involved stem cell scientist Irv Weissman of Stanford University, a close friend of Hood. The men also own a ranch in Montana together and have scientific and professional links. Alan Trounson, the stem cell agency's president, has been a guest of Weissman's at the ranch and recruited Hood, an expert in genomics, as a grant reviewer.

CIRM, as the $3 billion stem cell agency is known, said it did not detect the relationship between Hood and Weissman until it was called to their attention by another reviewer who was also participating in the closed-door review of applications.

A staff memo prepared for next week's meeting  of the agency governing board described the violation as “inadvertent and highly technical.”

On Monday, the California Stem Cell Report reported that unspecified changes were being considered in the conflict rules. Following publication of the item, more information on the proposal was posted on the agency's Web site in preparation for next Wednesday's meeting of the agency's governing board in Burlingame, Ca.

The memo, prepared by the agency's attorneys, said, 

“In order to prevent both the reality and appearance of a conflict, while preserving CIRM's ability to attract the best reviewers available, the rules should flag only those interests that could genuinely be deemed material.”

The changes would create a threshold of $5,000 a year for conflicts involving salary or consulting fees. Less than that would not trigger a conflict situation. Other proposed changes to be taken up by the agency board involve personal and professional conflicts along with the nature of the economic disclosures that reviewers, all of whom are from out-of-state, must disclose privately to a handful of agency officials.

The agency's scientific reviewers do not have to disclose publicly their financial and professional interests despite the fact that they have made all of the decisions on 98 percent of the applications for the $1.8 billion the agency has handed out. The interests of the reviewers are also withheld from applicants, many of whom may have competing or professional interests. The agency requires that reviewers who have conflicts must be removed from consideration of applications where conflicts exist. However, there is no way to determine whether that is actually done because the applications are reviewed behind closed doors and the economic and professional disclosures are withheld from the public.

Our take:
The agency's position on the conflict involving Weissman and Hood is disingenuous. To say that it is “highly technical” trivializes the entire matter. The conflict problem does not necessarily arise because Hood's friend (Weissman) could have received a few thousand dollars through approval of a grant. It involves much, much more. The application was for $24 million to create the first-ever stem cell genomics center in California. Should Weissman's employer, Stanford University, have been selected for the facility, it would have accrued to the great benefit of that institution, both monetarily and otherwise. Indirectly, it would have also enhanced Weissman's already substantial reputation and prestige as a person whose name can sway actions by California's state research effort. In the world of science, reputation and prestige often count for more than money.
­

Conflict-of-Interest Changes Coming at California Stem Cell Agency

Directors of the $3 billion California stem cell agency meet next week to consider changes in conflict of interest rules for the persons who make virtually all the decisions on the hundreds of applications  for the cash that it hands out for research.

Details of the changes and their justification are not yet publicly available for the meeting in Burlingame, Ca., on Oct. 9. The only information that was posted on the CIRM Web site as of this morning was this brief note from the meeting agenda:

“Request for consent to initiate rule-making to amend conflict of interest regulations for non-ICOC members of the Grants Working Group.”

ICOC is the abbreviation for the stem cell governing board, whose members are required to publicly disclose many of their financial interests.

The changes would affect the out-of-state scientists who score grant applications during closed-door deliberations. The agency's governing board, which has the ultimate legal authority for application approval, has ratified 98 percent of the decisions by the grant working group, according to the agency's own calculations.

The agency does not require the scientists to disclose publicly either their economic or professional interests and has resisted proposals for more transparency for years. The interests of reviewers instead are disclosed privately to a limited number of persons within the agency There does not appear to be a significant effort to audit the disclosures for accuracy.

Questions about the legal necessity for public disclosure of grant reviewers' interests have arisen as far back as 2007, including a recommendation by the state auditor that the agency seek an opinion from the state attorney general on the matter. The auditor said,

 “The FPPC (the state's government ethics agency) believes that members of working groups, who perform duties such as advising the committee on standards and policy or evaluating grant applications and making award recommendations to the committee, may need to be included in the conflict-of-interest code. Specifically, the FPPC believes that, under state regulations, working group members may act as decision makers if they make substantive recommendations that are, over an extended period, regularly approved without significant amendment or modification by the committee. Thus, as decision makers, working group members would need to be subject to the conflict-of-interest code.”

However, the agency said, also  in 2007,

  “The recommendations of the CIRM working groups have never been routinely and/or regularly adopted by the ICOC. Until the time that such a pattern is detected, the question you suggest we raise with the attorney general is entirely hypothetical, and is therefore not appropriate for submission. We will, however, continue to monitor approvals for such a pattern and will reconsider our decision if one emerges."

Because of the closed door nature of the grant application review process, conflict questions rarely surface publicly. Last spring, however, the California Stem Cell Report reported conflict violations involving an internationally reknown scientist-reviewer in a $40 million grant round. The scientist, Lee Hood of Seattle, Wash., is a close friend of one of the applicants, Irv Weissman of Stanford. They also own property together in Montana.

As we reported at the time,

“The conflict was not discovered by the agency during the review. It was raised by another reviewer at the end of the review, which, for the first time in CIRM history, failed to conclude with a decision supporting any of the proposals. Reviewers' comments have been sent back to applicants with another review scheduled for November. The agency said Hood will not take part in that session.”

Hood was recruited as a reviewer by CIRM President Alan Trounson, who has been a guest of Weissman at the Hood-Weissman ranch.

Midnight Deadline for Stem Cell Person of the Year

Today is more than showdown day for the functioning of the federal government. It is the last day to submit nominations for the Stem Cell Person of 2013.


This is the second year for the contest, which carries a $1,000 prize provided by UC Davis stem cell researcher Paul Knoepfler. He runs the contest and is the ultimate arbiter on who wins.


You have until midnight to submit your nominations. Here are links to more details.

California's $70 Million Alpha Stem Cell Clinic Plan Edging Forward

The $3 billion California stem cell agency is predicting that “human healthcare will be greatly improved” by stem cell research by 2024 and this week is fleshing out some of the details of an ambitious plan to help make that happen.

The effort involves what the agency calls “Alpha” stem cell clinics. The agency said its proposed $70 million, Alpha network will support its own projects along with products developed worldwide and brought to California.

The plan will come before the stem cell agency's standards group tomorrow at a meeting in San Francisco. The agency's staff has recommended alteration of some of the agency's regulations to avoid duplication in connection with review and approval of clinical trials.

The staff document said a workshop earlier this year developed a “consensus that it would be beneficial if the CIRM (the stem cell agency) regulations reflected existing state and federal requirements with regard to delegation of responsibility and avoid unnecessary duplication of effort, such as the IRB (institutional review board) responsibility for the risk and benefit assessment.”

The staff is recommending that “CIRM’s regulatory requirements for clinical research should be modified to avoid duplication of IRB’s responsibility for review and approval of clinical trials.”

The staff also said,

“CIRM’s existing regulatory requirements for notification, review and approval of basic and pre-clinical research appear effective at this time without creating undue burdens. In fact, mature systems appear to be in place to efficiently incorporate ESCRO (embryonic stem cell research oversight) operations into institutional compliance programs.”

Also on the standards group agenda are issues dealing with informed consent by providers of somatic cells “obtained under general (biomedical) research protocols" in connection with iPSC (induced pluripotent stem cell) derivation.

Dubbed the “Discuss Project,” its goal is “to initiate a process designed to develop consensus for the use of previously collected specimens for iPSC research" and to publish "final considerations" in early 2014.

Another item on the agenda tomorrow involves an update on the “progress of CIRM iPSC bank and donor consent protocol.”

Deadline Approaching on Stem Cell Person of the Year Awards

Only six more days remain to submit nominations for the Stem Cell Person of the Year.

This is the contest run by UC Davis stem cell researcher Paul Knoepfler, who will give the winner $1,000 of his own, personal cash.

The criteria are nearly wide open although Knoepfler is looking for the “most influential” person in the field. He wrote on his blog,

“Nominees can be almost anyone ranging from an advocate to an academic or industry scientist to a stem cell biotech leader to a policy maker to a physician…think outside the box! Surprise me!”

Knoepfler initiated the contest last year. The 2012 winner was Roman Reed, a San Francisco Bay Area patient advocate.

Knoepfler reported today on his blog that he has received two dozen nominations so far. He is planning on opening up the selection of the semi-finalists to online voting from the list that will be published in early October. Last year he selected the semi-finalists himself.

As for the names he has received so far, Knoepfler said,

“This group of nominees is very diverse, far more so than last year, and includes more scientists and physicians.”

Nominations should be sent to knoefpler@ucdavis.edu. More information about the rules can be found here.

Surgeon Expected to be Added to California Stem Cell Agency Board

Julie Freischlag, UC Davis Photo

The next new member of the governing board of the California stem cell agency is likely to be a surgeon from Johns Hopkins, Julie Freischlag.

Freischlag was named this week as the new vice chancellor for human health services and dean of the UC Davis medical school, replacing Claire Pomeroy, who resigned to become president of the Lasker Foundation.

Pomeroy had served on the stem cell board since 2005. She was named to the board by the UC Davis chancellor because of her position as dean of the medical school.

Freischlag, who is scheduled to begin her new job Feb. 10, is no ordinary surgeon. Currently she is chair of the Department of Surgery at Johns Hopkins. Prior to that she was chief of vascular surgery at UCLA. A Baltimore Sun profile of her in 2003 said she stands out because of her stellar career in “perhaps the most macho specialty in medicine.” The article said women surgeons are outnumbered 5 to 1 by men.

Responding to a query from the California Stem Cell Report, a spokeswoman for the UC Davis chancellor was noncommital about whether Freischlag would be appointed to the stem cell agency board. Luanne Lawrence, associate chancellor for strategic communications at Davis, said,

“This is one of the important and transitional decisions that hasn’t been addressed quite yet.”

However, the deans of the other major California medical schools sit on the stem cell agency board, which gives away $300 million a year for research. Given that context, it would seem prudent for the dean of the UC Davis medical school to serve on the board as well.

For more on Freischlag's background, see here and here.

Inside Stem Cells: A Guide from a UC Davis Researcher

The Sacramento Bee today published an article on UC Davis stem cell researcher Paul Knoepfler and his new book, which explores the basics of stem cells and how to make good judgments concerning possible treatments.

The book, “Stem Cells: An Insider's Guide,” is aimed at a general audience while at the same time exploring the nuances of stem cell science.

Below is a copy of the article, which appeared on the main page of The Bee Web site early today and which was written by yours truly. In the print version of The Bee, the Knoepfler piece appeared on the same page as a New York Times story dealing with dubious stem cell treatments(Here is a link to the original version of the Times' piece.).

The Knoepfler story was also the No. 1 article that turned up this afternoon when the Google news category was searched using the term “stem cell.” It is likely to be published elsewhere in the coming weeks as it is circulated by the McClatchy News Service. 

UC Davis stem cell researcher warns consumers to beware of unproven or dangerous stem cell treatments

By David Jensen

Bee correspondent

PUBLISHED SUNDAY, SEP. 15, 2013

In his day job, UC Davis scientist Paul Knoepfler probes the inner workings of stem cells and cancer cells and what makes them behave the way they do.

On the side, the father of three daughters blogs about costly, unproven stem cell treatments and provides guidance for those seeking experimental therapies.

Knoepfler is a rare stem cell researcher who regularly explores the most problematic aspects of stem cell therapies on the Internet in full public gaze. He considers himself an advocate for patients as well as a scientist, having survived an aggressive form of prostate cancer at the age of 42.

Now 46, Knoepfler began his blog in 2010, shortly after his cancer was diagnosed. His blogging has encountered resistance from some colleagues, who are uncomfortable with such public endeavors. But he has polished and expanded the blog to the point that it has received international recognition. He will receive an award in December at the World Stem Cell Summit in San Diego for his advocacy efforts.

This month he moved beyond cyberspace and published “Stem Cells: An Insider’s Guide” (World Scientific Publishing). The book covers stem cells from A to Z and is aimed at the general reader, especially people considering stem cell therapy for themselves, a family member or friend.

The book comes during what has been described by Christopher Scott, a senior research scholar at Stanford University, as “an epidemic of transplant clinics offering so-called cures and therapies” both here and abroad. The international stem cell medical tourism business, widely promoted on the Internet, is taking in roughly $1 billion annually, according to an estimate in Stanford Medicine, a publication of the Stanford medical school.

Knoepfler is a believer in the potential and power of stem cells. But he says that some of the dubious stem cell treatments now being offered have resulted in deaths and injuries. Other unpleasant issues arise as well in stem cell research, including the tendency of some stem cells to generate cancer cells, and Knoepfler wants to talk about them.

“Too often in the academic section of the stem cell field, people pussyfoot around the most important issues or do not even dare talk about them at all,” says Knoepfler.

He is “not on some crusade to dissuade people from getting risky stem cell procedures,” but says safety and training need to be encouraged. Reckless behavior endangers the entire field, he says.

“There is no better illustration of the risks of unlicensed stem cell treatments administered by untrained doctors than the recently reported case of a woman who received a stem cell facelift, only to have bone grow in her eye,” he said, referring to a Scientific American report involving a Beverly Hills clinic.

To help patients, Knoepfler’s book begins with the basics: What are stem cells? He moves on to topics ranging from whether stem cells can treat baldness to whether they can help with afflictions such as Alzheimer’s, autism and arthritis. Along the way, he discusses the potential for regenerating limbs, made-to-order organ transplants and curing spinal paralysis, none of which have reached the stage where patients can be assured of using the techniques safely and effectively.

Stem cell treatments are not exactly new. They were first used in the 1950s in bone marrow transplants. More recently, what has excited researchers and the public are pluripotent stem cells, which have the ability to transform themselves into any part of the body. The full range of stem cells, however, includes adult, fetal, embryonic and induced pluripotent cells (iPS), which are also known as reprogrammed adult stem cells.

“Each type of stem cell has clinical promise for specific diseases, but also certain weaknesses. Some stem cells also stir controversy. For example, embryonic stem cells have been at the center of ethical debates for more than a dozen years,” Knoepfler writes.

Embryonic stem cells are derived from an embryo only a few days after fertilization and require destruction of the embryo. It was that type of process that triggered the federal restrictions – now rescinded by President Barack Obama – on federal funding of research using human embryonic stem cells. Some people believe that the process amounts to killing a human being, while others do not.

Knoepfler has come up with a battery of questions and “rights” that persons considering a stem cell treatment should address as they contemplate spending tens of thousands of dollars for treatments.

His “patient bill of rights” includes the right to treatment by a trained provider, the right to continuing follow-up and the right not to be charged to participate in a clinical trial.

Just this month, he added on his blog, ipscell.com, the top 10 questions that patients should ask. They include such areas as the details of the methods to be used and costs, the cell numbers and types, regulatory compliance by the provider, data supporting the cellular product’s effectiveness and data supporting its safety.

As for mainstream media reports and research papers that seem to promise miraculous cures, Knoepler says, “My advice to patients is to be cautious when reading papers on the clinical use of stem cells. Do not believe everything you read and avoid placing too much weight on any one paper. If something is real, it should be reproducible by multiple groups.”

One of the areas of Knoepfler’s research involves cancer stem cells, a topic of special interest to him because of his own encounter with the disease. “Even with something as wonderful as stem cells, there also can too much of a good thing. Stem cells can cause cancer and in certain conditions they can make cancer especially hard to cure,” he explains.

In the case of embryonic stem cells, he cites their ability to form an unusual tumor called a teratoma. Knoepfler, who was an English literature major as an undergraduate, says, “The name ‘teratoma‘ literally means ‘monster tumor,’ and there is good reason for that nomenclature. These tumors look monstrous when observed by eye, akin to an animal put into a developmental blender.”

Knoepfler did not use stem cell therapy to treat his cancer, but he says that as a cancer survivor he understands all too well why patients turn to it when they’re dealing with a life-threatening or life-changing medical condition.

“If you feel that your disease has put you in a place for which patience is not an option,” he says, “talk with your physician. Get second and maybe even third and fourth opinions before deciding whether or not to proceed.”

Knoepfler’s advice may not be appreciated by some of the firms he writes about, but others think well of him.

Jonathan Thomas, chairman of the $3 billion California stem cell agency, says the field needs more scientists like Knoepfler.

“It's hard to overstate the value of what Paul does,” Thomas said in an email.
“Paul is a powerful advocate for helping the public understand what research is being done, and why it is important. He is gifted at taking complex science and turning it into plain English so that anyone can understand what he's talking about.”

Knoepfler has received $2.2 million from the stem cell agency for his research, which is also funded by the federal government and other sources. He has been at UC Davis since 2006, part of the campus’ stem cell program, which has received $131 million in grants from the state stem cell agency since 2005, generating 333 research-related jobs.

Knoepfler, whose father was a physician and mother a counselor, sees a bright future for stem cells, indeed a “medical revolution.”

“Stem cells are today’s new frontier of medicine that will no doubt have an unimaginable impact on our lives, but even more so on the lives of many of our kids and grandchildren,” he said.

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Jensen is a retired Sacramento Bee journalist and has produced the California Stem Cell Report (californiastemcellreport.blogspot.com) on the Internet since 2005, writing nearly 3,600 items.

Selling Stem Cell Sizzle: The Future of a $3 Billion Effort

All stem cell research is not created equal, a truism that found fresh validity this week.  Particularly research that could play a role in whether the California stem cell agency can find more cash to continue its operations. 

The difference was highlighted yesterday by articles on the stem cell agency’s blog. The pieces dealt with findings – some esoteric and some not-so-esoteric -- that received international attention. The articles written by CIRM staffers Amy Adams and Don Gibbons were fine as far as they went. But it is one thing to deal with the nuts and bolts of research and another to look at it from the perspective of whether it resonates with the public. 

The research in question is from Spain and Stanford. Researchers in Spain  reprogrammed adult cells in a living mouse to become like embryonic stem cells. Those results received much “gee whiz” attention in the mainstream media, most of which overlooked problematic aspects  involving its cancer-linked results(see researcher Paul Knoepfler's take here and Gibbons' item here).  

The other findings out of Stanford dealt with people and Down syndrome, along with cognitive function, aging and Alzheimer’s.

The press release by Krista Conger from Stanford said,

"'Conceptually, this study suggests that drug-based strategies to slow the rate of stem cell use could have profound effects on cognitive function, aging and risk for Alzheimer’s disease in people with Down syndrome,' said co-author Craig Garner, PhD, who is the co-director of Stanford’s Center for Research and Treatment of Down Syndrome and a professor of psychiatry and behavioral sciences.”

Both the press release and the CIRM blog item briefly noted that funding from CIRM helped to sustain the research. Stanford buried the information at the end of its release. CIRM mentioned it much higher in its item. 

In neither case were specific funding figures mentioned. Nor was there any attempt to say whether this research would have been slow in coming or not coming at all without CIRM help. 

Why does that matter? The $3 billion state stem cell agency will run out of funds for new grants in about three years, not very long given the length of time it takes to develop major funding sources and the rather deliberate pace at which CIRM works on some matters. 

Currently the agency spends about $300 million year on research and is not likely to be able to renew its funding at that level. But if it wants to play at even the $50 million level, it will have to generate some sizzle from the research that it has funded. 

Sizzle is what the Stanford research has. It resonates with people. We all know somebody or a family with issues such Alzheimer’s, Down syndrome or cognitive problems. Missing largely, however, from the press releases, media stories and even the CIRM blog is some sort of way of assessing whether CIRM funding played a KEY role. 

And that is the clincher for agency. That is the sizzle that will sell the agency as absolutely necessary if it truly wishes to turn stem cells into cures.

(Editor's note: Shortly after this item was posted, we searched the agency's Web site. One of the results disclosed that agency gave $1.4 million to Michael Clarke of Stanford for the research. He has filed two progress reports on his findings.) The research received additional support from CIRM as well, but the amounts were not readily apparent.)