WARF Stem Cell Challenge: Appeal Says Patent Involves Cells Not 'Markedly Different' Than Found in Human Body

The battle over whether excessive protection of stem cell IP stifles research that can lead to cures was engaged once more today with a broadside against the powerful Wisconsin Alumni Research Foundation(WARF).

The attack came from California’s Consumer Watchdog organization and New York’s Public Patent Foundation which have been tussling with WARF for seven years. The dispute over intellectual property (IP) centers on a patent on human embryonic stem cells held by WARF and which the other organizations are challenging in a federal appellate court in Washington, D.C.

More specifically, the patent involves research by Jamie Thomson of the University of Wisconsin, and now also of UC Santa Barbara, in which he isolated human embryonic stem cells.

Consumer Watchdog of Santa Monica, Ca., this morning issued a news release concerning the organizations’ appellate brief that was filed last week. It cited the U.S. Supreme Court ruling earlier this year that said genes cannot be patented because they exist in nature.  The lead attorney in that successful case, Dan Ravicher of the Public Patent Foundation, is also handling the challenge to WARF.

The news release said that Thomson deserved credit for being first to isolate and maintain human embryonic stem cells, but “his achievement was not the result of his having created a patentable invention.” The brief said that the work involved was “obvious.” One of the main reasons for Thomson’s achievement, the news release said, was that “he had access to human embryos and financial support that other researchers did not have.”

The brief said,

“The claims at issue here cover human embryonic stem (hES) cells that are not markedly different from those in our bodies. Thus, the claims are invalid under 35 U.S.C. § 101 for covering ineligible subject matter, an issue the Court may and, as a matter of judicial economy and public policy, should address.”

The challenge to the WARF patent has drawn impressive support in the scientific community, including  Jeanne Loring, now director of the Center for Regenerative Medicine at The Scripps Research Institute, who was involved from the start. In 2007, Loring wrote in Nature that she became involved in the case because “scientists have an obligation not only to perform research but to make sure that our research can benefit the society that supports it.”

The news release said,

“Later in the case Dr. Alan Trounson, then of Australia’s Monash University and now president of the California Institute for Regenerative Medicine, Dr. Douglas Melton of Harvard and Dr. Chad Cowan of Harvard filed affidavits supporting the challenge.”

Here is a link to legal documents and other material in the case.

Tracking the Fruits of California Stem Cell Agency Research

The California stem cell agency yesterday shed some interesting light on the awards in its $41 million round this week and their pathway to actually producing a product that can be used to treat persons who are suffering from diseases.

It is a difficult and long journey to generate usable therapies, a process poorly understood by the public, which was promised in 2004 that the stem cell agency would produce cures for ailments afflicting half the population of the state.

Writing on the agency’s blog, Amy Adams, CIRM communications manager, dealt with the issue indirectly. She said,

“Many scientists who receive our early translation awards first got their idea for a therapy while carrying out research with one of our other awards. In fact, eight of the scientists in this round of funding had previous CIRM funding for an earlier stage of research. If a scientist's early translation award provides good results, the scientists are then able to apply for one of our disease team awards, which fund the effort of compiling data to convince the Food and Drug Administration to allow them to test it in people.  Other organizations fund only early discovery research or only preclinical research. Under those conditions, researchers continually pause their projects to look for new sources of funding as the project moves through the phases toward clinical trial.”

One of the virtues of the California stem cell agency is its promise of a continued stream of funding. Former Chairman Robert Klein used to tout that particular aspect of the agency, particularly in light of limited federal resources.

Adams’ comments implicitly raise important questions concerning CIRM’s entire portfolio. How many CIRM grants have led to additional funding from CIRM? How many are basically one-off shots that have not led to research that has advanced the development of stem cell therapies, either via CIRM or other funding. What is the therapeutic and scientific significance of the research that is linked by more than one CIRM award?  What previously funded CIRM research could be fruitfully funded again to advance the science and not necessarily through the traditional grant rounds, which sometimes have awkward timing?

Unmentioned in Adams’ item is an application from a UC Irvine researcher that came up at Wednesday’s meeting of the governing board of the stem cell agency. The woman, whose name was not clearly audible on the Internet audiocast, publicly appealed rejection of her application by reviewers. She noted that it was an extension of work that was previously funded by the agency. She also noted that the score on her review was all but identical to work that was funded. The board, however, turned her appeal aside, which had already been rejected behind closed doors by CIRM staff.

Hers is not the only such case in CIRM history. But they are virtually impossible to track systematically because of the structure of the CIRM grant-making progress. It is also not clear whether the agency itself is tracking its research awards to determine if they result in continuing, fruitful research in a specific area. Nonetheless, the matter deserves some public attention. 

UCLA Takes Four of 13 Awards Today; One Business Wins

The California stem cell agency has made it official, sending out its press release on the $41 million in grants approved today for institutions throughout the state. 

Most of the 13 awards, as usual, went to organizations represented on the governing board of the agency. Individual board members, however, are barred from voting on specific grants to their organizations. 

UCLA topped the list with four grants. No other institution received more than one, including only one business, Numerate, Inc., of San Bruno, via John Griffin, the firm's chief scientific officer. The lack of awards to businesses has long been a sore subject in the biotech community.  

The only news story so far was written by Bradley Fikes of the San Diego U-T, which circulates in an area that is a hotbed of biotech research. Institutions there snagged $12.6 million in four grants. Fikes also identified one of the five researchers who lost their appeals on negative grant review decisions. He is Evan Snyder, leader of stem cell research at Sanford-Burnham Medical Research Institute in La Jolla, who had a $5 million request before the agency.

The agency's press release can be found here with more information on the proposed research here.  

CIRM Board Meeting Adjourns

Today's meeting of the directors of the California stem cell agency concluded at 4:11 p.m. PDT. We do not anticipate posting additional news items today.

Stem Cell Agency Seeks Stronger Ties with Possible Industry Funding Partners

The California stem cell agency today triggered a new program aimed at recruiting major biotech and venture capital firms to assist in providing tens of millions of dollars for research by California enterprises.

The effort, part of an $80 million business-friendly initiative, was approved by the agency's governing board on a voice vote.

Participating companies will have a special relationship with the state agency, according to a staff document. The "industry collaborators" will have early input into concept funding proposals prior to their presentation to the agency's governing board. The companies will also be able to attend agency workshops and meetings involving hundreds of grant recipients. 

Other aspects of the proposal call for special event-hosting arrangements aimed at creating more collaborations along with posting of information from the selected collaborators on the CIRM website.

California Stem Directors Still in Closed Session

Directors of the California stem cell agency today are still huddled behind closed doors, discussing an evaluation of CIRM President Alan Trounson. It is not clear when the public meeting will resume. Normally, little is publicly disclosed about such private personnel sessions, which are permitted under state law.

UCLA Snags $3.6 Million from California Stem Cell Agency

UCLA scored today with at least two grants, totaling $3.6 million, from the California stem cell agency. 

Seeking the cash were Donald Kohn,  application 6823, and Gerald Lipshutz, application 6831. Both of the  grants are for $1.8 million each. 

Their applications were initially in the agency's tier two category, which means that CIRM's reviewers did not approve them outright for funding.  CIRM staff, however, did under a new procedure, and the agency's governing ratified the recommendation. 

Lipshutz also appeared before the board along with several patient advocates who made emotional appeals for funding. Lipshutz's research deals with urea cycle disorders, which occur in one out of 8,200 births. Current treatment is arduous and can involve liver transplants.  

Kohn's research deals with sickle cell disease, which afflicts primarily African-Americans. His efforts are aimed at correcting the sickle gene defect in the blood stem cells before transplanting them back into the patient.

$41 Million in California Stem Cell Grants Virtually Approved

Directors of the California stem cell agency today all but approved about $41 million in early translational grants, rejecting all appeals by applicants and accepting staff recommendations on marginal grants.

The roll call vote was held open this morning to record a vote by one board member who was not present at the time. It is virtually certain that the member will vote in favor of affirmative action on the applications in question.

One member of the board, Joan Samuelson, abstained from voting on any of the applications. She said she did not think the board had adequate information on its total grant portfolio, particularly in view of the declining amount of money available.

The agency has about $600 million in uncommitted funds and is scheduled to run out of cash for new grants in 2017.

The research acted on today is aimed at “proof of concept for development of a therapy candidate and/or studies to select a development candidate. The approved grants can be found on this CIRM website page and are listed in tier one and tier two.  Identities of the applicants are withheld by CIRM to avoid embarrassing rejected candidates and to avoid disclosing the names of applicants to board members before they vote. However, applicants often appear before the board, as they did today, and identify themselves.

Five applicants appealed negative decisions on their applications by grant reviewers. The agency declined to disclose the appeal letters or identify the applicants, information that was a public record under the previous appeal procedures. New processes were put in place this spring that moved the appeals behind closed doors and made them subject to staff instead of board review. Nonetheless, rejected researchers have a legal right to address the board on appeals or any other matters.

At the request of the California Stem Cell Report, the agency provided the numbers of the grants on which appeals were filed. They are: 06787, 06888, 06761, 06793 and 06830. Review summaries on the applications can be found here. 

We have asked the agency to provide its legal and policy justification for now withholding information that was once a public record.

Stem Cell Board Moves into Closed Door Lunch Session

Today's meeting of the California stem cell agency governing board is now in recess for a closed-door lunch session involving the evaluation of CIRM President Alan Trounson. It is not clear when the open session will resume, but probably it will be at least 90 minutes.

UCLA Application for $1.8 Million Moves Ahead at Stem Cell Board Meeting

Directors of the California stem cell agency this morning moved forward on a $1.8 million application from UCLA that the agency's reviewers declined to approve outright.

The application (No. 6831) was moved into a category that will be approved routinely later today by the board. CIRM staff recommended that the application be approved.

Advocates speaking on behalf of the application identified UCLA as the institution involved. A researcher also spoke, but his name was not clearly audible on the Internet audiocast.

California Stem Cell Board Moves Positively on 'Wobbler' Grant Application

Directors of the California stem cell agency this morning ratified a staff recommendation for funding of a $1.8 million application that was a "wobbler" when it was examined behind closed doors by agency reviewers.

The CIRM staff had recommended funding of the application (No. 6823) with the condition that the agency staff be satisfied with the support to be provided by a collaborator. Reviewers had declined to approve the application outright.

The agency has not disclosed the identity of the applicant.

Discussion Begins in $70 Million Grant Round

Directors of the California stem cell agency have begun discussion of applications in its $70 million early translational round. Above is the Power Point presentation to the board of some of the applications including the range on the scientific scores in the round.

Stem Cell Agency Pays Tribute to the Late Duane Roth

The governing board of the California stem cell agency today paid tribute to the late Duane Roth, co- vice chairman of the agency, who died recently as the result of a bicycle accident.

With members of Roth's family present, CIRM Chairman J.T. Thomas characterized Roth, 63, as a "voice of reason" on the 29-member board. The video included testimonials from both staff and board members.

He was described as a "kind person" who could find "common ground" on difficult issues. Roth was deeply involved in San Diego affairs that went well beyond the stem cell agency. More than 1,000 persons attended memorial services for Roth earlier this month in San Diego.

CIRM President Alan Trounson said following the video that he will "miss (Roth) terribly" and expressed  "hope that his memory will lighten and brighten the day for all of us."

Jeanne Loring, a stem cell researcher at Scripps, said Roth "inspired us to do more than we thought we could do." She said he was an unusual kind of businessman who respected science.  "I wish I could thank him one more time," she said.

California Stem Cell Agency Board Opens Meeting

The California stem cell agency today started its meeting this morning at 9:11 a.m. with the announcement that the board is expecting the arrival of a "very important guest." The board began its business by dealing with routine business. The board may have difficulty with maintaining a quorum later in the day, it was announced.

Technical Problems with Stem Cell Audiocast

This morning's meeting of the governing board of the California stem cell agency was scheduled to begin five minutes ago, but technical problems are reported with its Internet audiocast. We will let you know when the audiocast begins.

Skin in California’s Stem Cell Game

The California stem cell agency’s road map to its financial future makes a big, $200 million assumption.

The amount would be the agency’s skin in the game for a new, public-private partnership to continue with the agency’s work after 2017, when its cash basically runs out.

The $200 million figure is contained in the assumptions for development of the proposed partnership, which is now in the very early stages of being crafted by a Marin County consultant, James Gollub. He was told that whatever he comes up with can assume a onetime, $50 million to $200 million public contribution.

The sixty-four-dollar question – to use a term from the 1940s -- is how to raise that sort of cash. Consider two unappetizing possibilities. The 29 members of the agency’s governing board could go to Sacramento and ask lawmakers and the governor to give them the money, a prospect that most of them would not relish. Such a move would open the door to tinkering or more with the agency’s structure and operations.  Or the board could seek more bond financing via a statewide election, requiring an electoral campaign that would cost many millions to mount. In both cases, there is no guarantee that funds would be forthcoming. Money is still tight in California government, and voters may not fancy spending more on stem cell research, especially if the agency has not delivered on the promises of the 2004 ballot campaign that created the $3 billion program.

A third possibility, however, exists, but it also could be difficult considering pressures to spend all that the agency has. The board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, has about $600 million in uncommitted cash. It could take $200 million off the table and reserve it as seed money for whatever future plans would involve. Or the board could simply roll back commitments it has made for lower priority grant rounds – ones that have not yet been initiated. Some are in concept stages, and others have not yet been posted as RFAs.

Scrimping on existing efforts is not going to suit the condition of all board members. The question of priorities on spending came up last month in connection with the agency’s generous, $69 million researcher recruitment effort that benefits many institutions represented on the agency’s board. Jeff Sheehy, who is a patient advocate member of the board but also a communications manager at UC San Francisco, and others bridled at adding more money to the recruitment program. Sheehy cited scarcity of funds and said it was a “distraction” from more important efforts. His view, however, did not prevail.

Today the board is scheduled to act on a grant round that is budgeted for $70 million. However, grant reviewers have approved grants totaling only $37 million. Board members, if they wish, could indicate that the surplus $33 million be designated as a down payment on the future of the agency – an organization in which they take great pride.

Live Coverage for Tomorrow's California Stem Cell Meeting

The California Stem Cell Report will provide live coverage of tomorrow’s meeting of the governing board of the $3 billion California stem cell agency. Stories will be filed as warranted during the daylong session, which begins at 9 a.m. PDT in La Jolla, Ca. Coverage will be provided via the Internet audiocast.

Those wishing to listen to the event live on the Internet can find instructions on the meeting agenda. Also available is a Webex transmission, which provides viewing access to the many documents that are used during the meeting but have not been made available yet to the public.

In addition to the La Jolla location, those wishing to participate in the meeting can do so at teleconference locations in Menlo Park and Duarte, both in California. Directions for the audiocast and Webex broadcast can be found on the meeting agenda.

Future Financing Plan for California Stem Cell Agency Coming Up in December

The California stem cell agency today released the $150,000 contract and proposal from James Gollub Associates to create a “strategic road map” for the $3 billion state program, which is scheduled to run out of cash for new grants sometime in 2017.

The goal of the road map, according to the contract, is to develop a “preferred model for a public-private sponsored entity to fund the most desirable translational projects in the CIRM portfolio.” That would appear to rule out future funding for basic research and training, which the agency has supported well during its nine-year history.  It would also be an acknowledgement that the agency cannot count on $300 million a year for grants as it has in the past.

Under the terms of Prop. 71, which created the agency in 2004, the effort is currently financed by state bonds, money borrowed by the state of California that flows directly to the agency without oversight by the governor or legislature. Authority to issue the bonds expires in 2017.

The contract calls for an initial report from the Gollub by Nov. 30 with the agency to decide by March 1, 2014, on whether it wants an operations and business plan from the Tiburon, Ca., firm.  Presumably the agency’s governing board would hear recommendations from Gollub at its December meeting.

The contract, signed July 16, specifies that Gollub will conduct at least 20 interviews with stakeholders to evaluate four possible models that it will develop. The stakeholders will include “investment, academic and government groups” and possibly others to be determined later.

The proposal presented by Gollub contains more information on the firm and the persons who will be working on the project. In addition to Gollub, they include Steve Marshall, Amy Rassen and Annika Barnes, all Gollub employees. The contract and proposal can be found below.

  Gollub Proposal by DavidJensen

  Gollub-CIRM Strategic Roadmap Contract by DavidJensen

Stem Cell Grant Applicants Identities and Secrecy

The California stem cell agency today took issue with a statement in an item yesterday that said the agency withheld the names of grant applicants prior to action on their applications by the agency’s governing board to avoid embarrassing the researchers.

The agency said that the reason was to avoid identifying applicants to members of the governing board prior to its vote on the applications.

The agency’s official practice of withholding the identities of institutions and individuals seeking public funds has a long history.  The agency itself has breached the policy on more than one occasion.  While it also does not officially release the names of applicants denied approval, that practice is also breached when appeals are filed.

Interested readers can find a short discussion of the policy and links to additional material at the end of this item. 

Correction

The "New Procedures" item on Aug. 26, 2013, incorrectly stated, based on information provided by the stem cell agency, that six applicants in this week’s early translational round had filed appeals to overturn negative decisions by grant reviewers.  The agency today said the correct number is five. 

New Procedures at CIRM: California Stem Cell Agency Staff Nixes Grant Application

For the first time in its nine-year history, the staff of the $3 billion California stem cell agency this week  formally and publicly weighed in on grant approval actions by its prestigious reviewers, recommending that one application be rejected and another approved with conditions.

The staff, led by CIRM President Alan Trounson and Patricia Olson, the agency’s executive director of scientific activities, made recommendations to the agency governing board on three applications that can be described as “wobblers.” In other words, the board could go either way on the proposals when it considers them at its meeting in La Jolla on Wednesday.

Trounson recommended that the governing board reject one of the wobblers  (No. 6666),  a $2.0 million proposal, even though it received a higher scientific score – 70 – than the other two applications. Trounson recommended approval of two $1.8 million projects (Nos. 6831 with a scientific score of 66 and 6832 with a scientific score of 69), with staff-imposed conditions on one.

 Trounson said the agency is or 

 "will be funding 2 similar approaches to address photoreceptor degenerative disorders so addi-tional investment in an earlier stage project is harder to justify.”

All three fall into a newly defined category, called tier 2, for ranking of applications. The CIRM web site said tier 2 proposals are now ones that possess “moderate scientific quality, or consensus on scientific merit cannot be reached and may be suitable for programmatic consideration by the ICOC(the governing board).”

A fourth application  (No. 6648) for $4.3 million that scored below all three at 64 was approved, however, by reviewers after they imposed a condition on the proposal.  CIRM staff did not publicly address that application. The application review summary said that the researcher – who was not identified – must “demonstrate, within 12 months, the ability to make the hESC–derived 3-D sheets. This is a go no/go milestone for the project.”

The agency’s standard practice is withhold the identities of applicants prior to board action because they might be embarrassed.

In the other instance where conditions are to be imposed, they appeared to deal with an in-kind contribution of “essential services, technology and expertise.”

The new process for evaluating marginal or wobbler applications was established last March in response to an Institute of Medicine study last year that made a host of recommendations for improvements at the stem cell agency.

The staff recommendations on applications came in a $70 million early translational round that is aimed at “proof of concept for development of a therapy candidate and/or studies to select a development candidate.

In all, including staff and reviewer actions, 13 applications were recommended for funding, although the board has almost never rejected reviewer decisions. The 11 grants initially approved by reviewers total $37 million. With the two more recommended by staff, the figure would be about $41 million. A total of 39 applications were considered for funding.

Five applicants filed appeals of reviewer rejections, a CIRM spokesman said today. The  California Stem Cell Report has asked for copies of those appeals and CIRM staff action on them. Appeals are also being conducted under a new staff-dominated procedure, although all applicants have the right under state law to appear before the board to address any subject.

Budgeted grant funds that are unused are available to the board for future grant rounds in any area they so desire.

(An earlier version of this item incorrectly said six applicants had filed appeals, based on information from the agency. The correct figure is five.)

(Editor's note: Kevin McCormick, CIRM's spokesman, later commented on the agency's practice of withholding the identities of applicants to avoid embarrassment to applicants who are not approved. He said,  "Actually they are withheld so that the board doesn't know the identities of the researchers or the institutions whose applications they are voting on." 
(We should note that official CIRM policy is to withhold all applicant names until board action, but it does not release any of the names of denied applicants even after board action. However, it has in the past released in advance of board action the names of applicants when it suits its purposes . Also, the names of many applicants can be discerned based on information provided in the review summaries of the applications. The identities of applicant institutions can also be determined based on which board members are allowed to participate in discussion of specific applications as well as being allowed to vote. For more on the practice of withholding names, see here, here, here and here. )

California Stem Cell Agency's $150,000 Search for Its Financial Future

A San Francisco consultant, who is often known as an “economic therapist,” has been selected to devise a “strategic road map” for the financial future of the $3 billion California stem cell agency.

James Gollub: 'economic therapist'
Gollub Associates photo

James Gollub, managing director of the firm bearing his name, is under a $150,000 contract to lay out by this fall a detailed plan for the agency. The nine-year-old research effort is scheduled to run out of money for new awards in 2017.

Gollub was selected after the agency posted a request for proposals (RFP) last spring. The RFP assumed an additional $50 million to $200 million in a onetime “public investment.” The RFP also assumed additional private funding of a yet-to-be-determined nature.

Gollub is described as a co-founder of the field of cluster-based economic development. One web site said he is known as an “economic therapist.” The website for his current firm says,

“A leading expert in innovation bridge building....
“Global experience assisting universities, institutes, government agencies and public-private partnerships link innovation sources to innovation seekers.
“Committed to the goal of increasing flow of needed solutions, optimizing financial returns and sustainable economic impacts from innovation.”

Gollub's current firm dates back to March of this year. His Linked In profile says,

“James Gollub Associates (JGA) LLC was launched to build on 36 years of Gollub’s professional research and consulting experience. That experience began with 16 years at SRI International, three years at DRI/McGraw-Hill, five years at IDeA, nine years at ICF International and three years with E-Cubed Ventures LLC. During that time Gollub has worked globally to deliver economic strategies for over 30 national, state and metropolitan regions, develop strategies to accelerate growth of new industries (clusters), plan public and private R&D institutes and advise on over 15 science and technology parks.”

The need for a financial transition plan for CIRM was publicly identified as long ago as 2009 by the Little Hoover Commission in its lengthy study and has been reiterated periodically by other bodies since then. Under the terms of Prop. 71, which created the agency, CIRM has only  a 10-year authority to issue state bonds, the borrowed funds that have sustained the research effort. Legal maneuvering blocked the issuance of bonds until 2007.

The California Stem Cell Report asked the stem cell agency on May 31 for a copy of Gollub's response to the RFP. Yesterday we asked for a copy of the contract with Gollub. Those documents will be published when they are received.

New Ties to Big Pharma and Venture Capital Proposed at California Stem Cell Agency

The $3 billion California stem cell agency wants to recruit major biotech and venture capital firms to help provide tens of millions of dollars in research awards to California enterprises.

It's part of a move to “jump start” partnerships in a relatively new, $80 million, business-friendly program that is aimed at pushing therapies into the marketplace. The recruitment plan will come before the agency's governing board at its meeting next Wednesday in San Diego. 

Participating companies will have a special relationship with the state agency, including early input into concept funding proposals prior to their being presented to the agency's governing board. The “industry collaborators” will also be able to attend agency workshops and meetings involving hundreds of grant recipients. Presumably other, non-collaborating firms would be barred.

Other provisions of the plan call for special event-hosting arrangements aimed at creating more collaborations and posting of information from the selected collaborators on the CIRM website.

According to a CIRM staff document, the initiative would be limited to biotech and pharmaceutical firms with a market capitalization of at least $500 million and “qualified venture capital firms.” The document did not define what a “qualified a venture capital firm” is. The document also appeared to bar participation of privately held firms because of the “market capitalization” criteria, which typically uses a formula involving publicly traded shares.

Elona Baum, the agency's general counsel and vice president, business development, said in a statement provided to the California Stem Cell Report,

“This is aimed at trying to jump start the creation of the partnerships that are required to satisfy the commercial validation requirements of the Strategic Partnership Funding Initiative so that timelines are better synced-up as between our review and approval cycles for the Strategic Partnership RFAs and the lengthy time required for investors to conduct due diligence and negotiate an agreement with prospective applicants to Strategic Partnership RFAs.  CIRM's independent review and approval remains the same and is wholly independent. While there may be input given to a particular RFA it only at the high level concept stage and of course CIRM has no obligation to agree. In the context of the Strategic Partnership awards, CIRM wants to fund innovative high quality science that has attracted additional  investors.  Investors will help leverage CIRM fund and will be an important source of future funding to further the project.”  

A $2 Million Bill: Outside Contracting by California Stem Cell Agency

The $3 billion California stem cell agency this week posted a list of its outside contractors, who range from a a $25,000 stem cell licensing consultant to a $550,000-a-year law firm.

The agency expects to spend $2 million during 2013-14 on outside contracting, down from $2.9 million in 2012-13. This week's report covers the 2012-13 year.

Outside contracting is the second largest item in the agency's budget, which is slated to spend $17.4 million this fiscal year for operational expenses, up 5 percent from last year's spending. The largest amount, $12.2 million, goes for salaries and benefits. (For more on the budget, see here, here and here.)

Topping the contractor list is the law firm of Remcho, Johansen & Purcell of San Leandro, Ca., which had the $550,000 contract. CIRM, as the agency is known, reported that Remcho came in under budget by $95,595. That contrasts to some previous years when the firm, which has represented the stem cell agency since its inception, required additional cash on top of its original contract. James Harrison of the Remcho firm is its face at the agency and is designated as the outside counsel to the agency's governing board. In all, the agency is slated to spend $2.2 million on legal expenses, including in-house work.

David Earp is the stem cell licensing contractor. He was paid only $13,125 on his $25,000 contract during 2012-13. It is unclear whether he will be paid the $11,875 balance. Earp was chief patent counsel and senior vice president for business development for Geron before it dropped its stem cell program. Earp was heavily involved in the $25 million loan that CIRM made to Geron in 2011. In February 2008, he testified before CIRM about its then proposed loan program.

The list of contractors included $200,000 to the AlphaMed Press of Durham, N.C., as seed funding for a stem cells translational journal, $156,434 to Hyatt Hotels for the meeting of CIRM grant recipients, $250,000 to Kutir Corp. of Newark, Ca., for informational technology services and $290,000 to the Mitchell Group of Woodland Hills, Ca., also for information technology services.

The list of contractors will be presented to the CIRM governing board at its meeting next week. The list does not usually trigger any significant discussion.

Correction

The stem cell spending item yesterday incorrectly stated that the California stem cell agency would run out of funds for new grants in 2013. The correct year is 2017.

California Stem Cell Agency Spending: Where the Money Is Going

Analysis of CIRM funding by Pat Olson, executive director of CIRM scientific activities July 2013

The California stem cell agency will have committed $472 million to translational research – a key to commercializing stem cell therapies – if it awards the full $70 million in new grants and loans slated to come before its governing board next week.

The nearly $500 million will amount to about 17 percent of its funding so far, according to an analysis last month by Pat Olson, the agency's executive director of scientific activities. The largest percentage of the agency's cash, however, will be going for “development” – 35 percent or $970 million. Olson defined “development” as “essentially our IND enabling, our preclinical development programs and our clinical development programs.”

Basic research is to receive 17 percent or about $469 million with buildings and facilities taking up $443 million or 16 percent. Training and career development has consumed about 15 percent or $414 million.

However, those calculations include $577 million in funds that have been allocated but not yet awarded. Another $491 million is “concept approved” but also not awarded. The agency's governing board could change those allocations or withdraw approval of concepts, although it has not yet shown signs that it might do so.

The agency will run out of money for new grants in 2017 and is examining the possibility of generating more cash through some sort of public-private partnership. To develop support for continued funding, the agency is under pressure to generate results that will resonate with the public and potential private funding sources. Those results are most likely to come from a late stage translational/clinical trial effort.

Here is a link to CIRM's translational portfolio as of September 2012.

(An earlier version of this item incorrectly said that the agency would run out of money for new grants in 2013. The correct year is 2017,.)

Flim-Flam Stem Cell Artists Targeted by CIRM

The California stem cell agency has joined with other prestigious stem cell organizations to help put a stop to the flim-flam artists that prey on desperate people by promising miracle cures from stem cell treatments.

The agency announced the action today on its blog, declaring that it has posted a new patient advisory document that provides a “ robust and detailed set of issues patients should consider when making treatment decisions.”

Don Gibbons, CIRM's senior science and education communications officer and author of the blog item, said the document addresses one of his special concerns: Internet “ads that come up on web searches and seem to be offering everything to everyone.”

Some of those ads can be found on many stem cell-related web sites, including this one, that carry ads that are placed there automatically by Google.

California Stem Cell Researchers to Receive $70 Million Next Week

The California stem cell agency is scheduled to give away $70 million next week as it moves forward on its efforts to turn research into cures.

The awards will be for efforts that will result in proof of concept for development of a therapy candidate and/or studies to select a development candidate.

As many as 20 grants and loans are projected to be awarded in the early translation round. The awards will range up to $3.5 million over a three year period.  The round was open to both businesses and academic institutions. Collaborators from Germany were involved, although funding for research in that country is not provided by the state stem cell agency.

The applications will come before the $3 billion agency's governing board at its Aug. 28 meeting in La Jolla. After next week's awards, the agency, which is known as CIRM,  will have about $500 million left to hand out before cash for new awards runs out in 2017. The agency is currently examining ways to continue its awards with some sort of public-private partnership.

Also on the board's agenda is a proposed announcement for a CIRM/industry co-funding agreement. No further details on that program were available early today on the agenda. Other matters to be considered include final approval of the changes in the agency's IP regulations, appointment of new members to the grant review group and the latest report on the outside contracts held by the agency. Details on those matters are yet to be posted by the agency.

A tribute to the late Duane Roth, co-vice chairman of the agency, is also scheduled. The board will additionally meet behind closed doors to evaluate the performance of CIRM President Alan Trounson.

The California Stem Cell Report will carry more information on the meeting as it becomes available.

In addition to the La Jolla location for the meeting, other locations where the public can take part in the meeting are in Menlo Park and Duarte. Specific addresses can be found on the agenda.

'Butter and Eggs Money" and a Gubernatorial Veto

Nancy Scheper-Hughes, professor of medical anthropology at UC Berkeley and director of Organs Watch, is one of the opponents of the legislation that would have permitted women to sell their eggs for research. Today she filed the following comment on the “troubling mindset” item on the California Stem Cell Report.

“Jerry Brown's veto of AB926 which would allow young women to be paid for multiple egg extractions for scientific research is one for the gals.  In western Ireland women secreted away their 'butter and eggs' money in anticipation of hard times. In my day every smart girl had her 'mad money' to escape a bad situation. Secret cash for young women is a great idea, but not when it turns on multiple cycles of pumping powerful hormones associated (in other contexts) with ovarian cancer into young women's bodies to produce 30 or 60 eggs a month. That's not promoting gender equity no matter what some of our best Democratic women leaders have to say. Selling sperm and selling eggs are a totally different matter. One  is pleasurable and safe, the other is a complicated and invasive procedure. We need good science and good research and  freedom of choice and action. We also need protection from false advertising. There are no evidence based, long term studies of the effects of these hormone injections on women ten or twenty years after the fact. Let's fund those needed longitudinal and cohort studies and hope for the best. In the meantime, women had best stick to 'butter and eggs' money. It doesn't pay a lot, but it's less painful and a heck of a lot safer.”

Bonilla: Veto of Pay-for-Eggs Bill Shows Troubling Mindset

A Democratic state legislator today assailed Democratic Gov. Jerry Brown's “mindset” as “particularly troubling” in his veto of legislation that would have allowed women to sell their eggs for scientific research.

The statement came from Assemblywoman Susan Bonilla, D-Concord, in response to Brown's action on her fertility-industry sponsored bill, AB926, which would have removed a ban on compensation for women who provide eggs for research.

Susan Bonilla
Photo from California Legislature

Brown cited health risks and other issues and said in his veto message,

“Not everything in life is for sale nor should it be.”

Alex Matthews, writing on Capitol Weekly, quoted Bonilla as saying,

“It (the governor's veto) shows a glaring inconsistency...The veto statement was very overreaching in the fact that it was making very broad statements about what women should be able to do, and while it's not legislation it certainly goes to a mindset that the governor has that I find particularly troubling.”

Bonilla continued,

“Market-driven compensation of donors by donor agencies and prospective parents continues unchecked.”

In a statement on her website, Bonilla said the governor's veto “is a regressive action that denies thousands of women the prospect of medical fertility breakthroughs.” She said,

“Many women...will be denied hope and the possibility of giving birth to a child because research on their behalf has been halted in California.”

Bonilla has argued that women involved in egg-related research, such as that involving stem cells, should be compensated, just as men are for their sperm. Women who provide eggs for fertility purposes can be legally compensated up to any amount. The current market runs about $10,000 or so per egg cycle but can be much higher.

Bonilla's measure would not have affected a ban on compensation involving research funded by the $3 billion California stem cell agency. It would have taken a 70 percent vote of each house to alter that restriction, compared to a simple majority for Bonilla's bill. The super, super-majority requirement was written into state law by Proposition 71, the measure that created the stem cell agency.

Bonilla did not indicate whether she would attempt to override the governor's veto, which would require a 2/3 vote of each house.

One of the opponents of the bill, the Center for Genetics and Society in Berkeley, called the veto a “welcome development.”

Diane Tober, associate executive director of the center, said,

“It would be unconscionable to expand the commercial market in women’s eggs without obtaining significantly more information about the risks of retrieving them.” 

Here are links to other stories today on the veto of the bill: Los Angeles Times, Sacramento Bee, an additional story from late yesterday on Capitol Weekly, TheAssociated Press and National Review.

California Gov. Jerry Brown Vetoes Pay-for-Eggs Legislation

California Gov. Jerry Brown today vetoed a fertility industry-backed measure that would have permitted women to sell their eggs for the purposes of scientific research.

In his veto message, Brown said,

“Not everything in life is for sale nor should it be.”

The bill would have repealed a ban on compensation of women who provide their eggs for scientific purposes. The measure would not have changed existing law that allows women to be paid for their eggs for IVF purposes with fees that range up to $50,000. The bill also would not have affected the ban on compensation for eggs for research that is financed by the $3 billion California stem cell agency.

The legislation (AB926) by Assemblywoman Susan Bonilla, D-Concord, was sponsored by the American Society for Reproductive Medicine and easily swept through the Democratic-dominated legislature. Bonilla said the measure would have placed women on an equal footing with men, who are paid for their sperm contributions for research. She also said that it would help to encourage more research into fertility issues.

Some stem cell scientists have complained that not enough women are willing to donate eggs without compensation, but stem cell researchers were not publicly involved in supporting the bill.

The fertility industry group had confidently predicted that Brown, a Democrat like Bonilla, would sign the bill. The governor's action could be overridden by a 2/3 vote of each house of the Legislature. It is not clear whether Bonilla will make such an attempt.

Here is the text of Brown's veto message:

"Not everything in life is for sale nor should it be.

"This bill would legalize the payment of money in exchange for a woman submitting to invasive procedures to stimulate, extract and harvest her eggs for scientific research.

"The questions raised here are not simple; they touch matters that are both personal and philosophical.

"In medical procedures of this kind, genuinely informed consent is difficult because the long-term risks are not adequately known. Putting thousands of dollars on the table only compounds the problem.

"Six years ago the Legislature, by near unanimity, enacted the prohibition that this bill now seeks to reverse. After careful review of the materials which both supporters and opponents submitted, I do not find sufficient reason to change course.

"I am returning this bill without my signature."

You can read more about the bill and its history here, here, here and here.

Duane Roth: Ecumenical Innovator for San Diego and Biotech

The Xconomy news service today carried a sterling look at the contributions that Duane Roth, co-vice chairman of the California stem cell agency, made before his untimely death as the result of a bicycle accident.

Reporter Bruce Bigelow pulled together a host of comments concerning Roth's involvement in the San Diego community, ranging from biotech to action sports companies. The headline on the piece read, “The Connector Who Wired up a Regional Innovation Economy.”

At the time of his death at the age of 63, Roth was CEO of Connect, a nonprofit organization that supported technology and innovation and one that he was credited with reviving. Bigelow also wrote,

“Once California voters approved a 2004 ballot proposition that authorized the issuance of $3 billion in grants for stem cell R&D, (Mary) Walshok (associate vice chancellor for public programs at UC San Diego) said Roth also played a key role in bringing together UCSD, Scripps, Salk, and Sanford-Burnham to create the Sanford Consortium for Regenerative Medicine. In fact, Walshok doubts whether anyone but Duane Roth could have brought the four major research centers together.”

Another speaker at the memorial services Friday attended by about 1,000 persons was Bill Walton, the former UCLA and NBA great, who grew up in San Diego.

Bigelow wrote,

“Walton, the NBA Hall of Famer who has led San Diego Sports Innovators as a division of Connect since 2010, said Roth became a business mentor to him. In his comments Friday afternoon, Walton said Roth inspired him to be a better person, and he counted Roth among the people who had the biggest influence on his life—a list that included his own father, UCLA coach John Wooden, sportscaster Chick Hearn, author David Halberstam, and Jerry Garcia of the Grateful Dead.”

Bigelow described Roth as an ecumenical and pragmatic advocate for innovation who could work with persons who did not always agree with him on all issues. He was a conservative and active Republican, but his co-vice chair at at the stem cell agency, Art Torres, former chairman of the state Democratic Party, on more than one occasion has lauded Roth's ability to work together.

Bigelow wrote about similar remarks Friday by Don Rosenberg, an executive vice president and general counsel at Qualcomm.

“'Duane and I were as different as two people can be,' Rosenberg said during his eulogy at the Church of the Immaculata. 'Duane was born in Iowa, baptized in the Mennonite church, a Republican. And me, raised in Brooklyn, Jewish, a Democrat. We quickly learned we had more in common. We were kindred spirits. We liked the same things: Bikes, biking, cars, and people.'”

A $6 Billion Question: Progress of the California Stem Cell Agency

The headlines march like legions across the Internet and throughout the world.

“New type of stem cell helps your fingers regenerate”

“Stem cell technology can mass-produce cancer-killing cells to target tumours”

"Stem-cell treatment restores sight to blind man”

“Special stem cells could heal hearts”

But then there is this extraordinarily rare headline that sounds a harshly different note:

“Stem cells: what happened to the radical breakthroughs?”

All these headlines go to address, in one form or another, a request/question posed last month by an anonymous reader of the California Stem Cell Report. The comment came on an item about the California stem cell agency's $70 million plan to establish a network of “Alpha” stem cell clinics in California.

The reader said,

“It would be nice to have an overall update on how much as been spent on California's stem cell research project and what progress has been made.”

On the surface, the answer is easy. The agency has given away $1.8 billion. The agency says it has made tremendous progress and expects to make even more with the about $600 million it has left. The prestigious Institute of Medicine has said the agency has “achieved many notable results.”

However, no thorough, rigorous evaluation has been made of the details of the agency's scientific contributions, specific grant awards or its impact on the field of regenerative medicine. No one has attempted to genuinely assess whether the work of the agency is or will be worth the roughly $6 billion(including interest) that California taxpayers will have paid for the agency's ambitious efforts.

Then there is the question of “progress towards what?” Is the progress to be measured against the promises of the 2004 ballot campaign that resulted in creation of the stem cell agency or more modest goals that eschew the hype of the campaign?

The stem cell agency is burdened in a way that most science is not. The 2004 campaign created a sort of contract with voters. They were led to believe nine years ago that the cures for diseases that the campaign said afflict nearly one-half of all California families were, in fact, right around the corner. Few, if any California stem cell researchers were publicly warning that a hard and long, long slog remained before therapies reached patients.

Last week, however, Simon Roach of the British newspapers, The Guardian and Observer, shed some light on the early, rosy promises of stem cell science compared to the world as it exists today.

He wrote that in 1998,

“(B)iomedical engineer Professor Michael Sefton declared that within 10 years, scientists would have grown an entire heart, fit for transplant. 'We're shooting big,' he said. 'Our vision is that we'll be able to pop out a damaged heart and replace it as easily as you would replace a carburetor in a car.'

“Fifteen years on, however, we've had some liver cells, eye cells, even a lab-grown burger, but no whole human organs. We could be forgiven for asking: where's our heart? It does seem strange that a field stoking so much excitement could be so far off the mark. Speaking last week about the vision that he and his colleagues outlined in 1998, Sefton said they had been 'hopelessly naïve.' As time plodded on and an understanding of the biological complexity increased, the task seemed bigger and bigger. Even now, a cacophony of headlines later, we are not much further ahead.

“Chris Mason is a professor of regenerative medicine at University College London and believes that concentrating on organ regeneration is missing a trick. 'These organs are immensely complex,' he said. 'They've got nerves, blood vessels, in the case of the liver, a bile system – there are huge degrees of complexity. These things take a long time to grow in humans, let alone in the lab without all the natural cues that occur in the growing embryo.'"

The final paragraph in Roach's article said,

“There's a tension in medical research between the glory of the big discovery and the assiduous commitment to real application. 'We're hoping the scope and possibilities of this project will catch the public's imagination,' Sefton concluded in 1998. It did, but perhaps the public's imagination isn't always what science should be vying for.”

Little doubt exists that the California stem cell agency has made a significant contribution to stem cell science, although the size of that contribution – beyond dollars – remains to be measured. For now, the key for the agency and the public is to focus on activities that will generate the greatest value over the next few years and advance the science that has already been financed by the agency.

As the $700,000 Institute of Medicine report said,

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

Stem Cell Agency's Duane Roth Eulogized at Memorial Services

An estimated 1,000 persons attended services last week for Duane Roth, co-vice chairman of the California stem cell agency, who died at the age of 63 following a bicycle accident.

The San Diego U-T reported,

“A Who’s Who of San Diego’s technology, business and civic community gathered Friday to bid farewell to Duane Roth. The biotech entrepreneur, community leader and director of Connect died last weekend of injuries sustained in a cycling accident.

“Among attendees were Gayle and former Gov. Pete Wilson, who had just celebrated his upcoming 80th birthday with former colleagues and friends in Sacramento, county Supervisor Ron Roberts, former Assemblyman Nathan Fletcher, Chamber head Jerry Sanders, Preuss School benefactors Peggy and Peter Preuss, SDG&E CEO Jessie Knight, and many biotech and high-tech leaders. These included Irwin Jacobs, Ted Waitt and Denny Sanford.”

'Paradigm Shift' -- Researchers as Patient Advocates?

It is not easy for a stem cell scientist to break into “print” in that bastion of American capitalism, Forbes magazine. Much less one from that perceived antithesis of free enterprise, Reed College.

But researcher Paul Knoepfler of UC Davis has done just that.

John Farrell of Forbes wrote about Knoepfler on Friday in the wake of the announcement Knoepfler will be honored with a “national advocacy” award by the Genetics Policy Institute.

Paul Knoepfler -- Advocate for a "new ethos"
UC Davis photo

Knoepfler, who received a degree in English literature from Reed in 1989, is being recognized for his activities as a stem cell blogger since 2009.

Farrell quoted Knoepfler as saying in an email,

“With many stumbles, face plants, land mines, and even a few threats of litigation and career retaliation along the way for the last three and half a years, I turned my crazy idea into a reality.”

Farrell continued,

“But it was only possible, (Knoepfler) added, with guidance from many patient advocates and bloggers in other fields who generously helped him learn the ropes of blogging.

“'I see this award as a validation of the notion that advocacy by scientists has become a valued part of the stem cell field,' he said.

“'My hope is to catalyze a continuing paradigm shift whereby stem cell scientists and biomedical scientists more generally have a new ethos that not just accepts, but also deeply values advocacy.'”

California Stem Cell Agency on Lacks: Informed Consent Cannot Remove All Questions

(Photo and caption from the stem cell agency blog item this morning.)

The $3 billion California stem cell agency today weighed in on the Henrietta Lacks-NIH arrangement restricting the use of her cell lines in research.

Writing on the agency's blog, Geoff Lomax, the agency's senior officer for its standards group, noted that the DNA sequence of her cell line was published without the knowledge of her descendants. Lomax said,

“The family was understandably upset by the lack of consultation and in response the research team removed the genome data from public access.”

Lomax continued,

“CIRM has benefited from these efforts. We are currently supporting an initiative to collect tissue samples from thousands of people with a range of incurable diseases and create reprogrammed iPS cells from those tissues (here's more about that initiative). These cells will be a resource for scientists worldwide working to understand and treat diseases. Part of this initiative includes a consent process to make sure people who donate fully understand how their cells will be used. (This process is formally called informed consent.) 

“The informed consent process includes a form that identifies the purposes of the research and describes the way cells will be used. We are also developing education materials that will help potential donors quickly and easily understand the basic aspects of research that will be conducted with those cells. The end result of this collaboration with our grantees will be a process that is truly informative to donors.

“The informed consent process can’t entirely eliminate all future questions on the part of the donor, but it does ensure that donors have a chance to understand how their cells will be used and what information will be made public—something Henrietta Lacks and her family never had.”