Shuffling the Files: Changes on the CIRM Web Site

The California stem cell agency has re-organized its cyberspace book shelves to help make its Web site easier to navigate.

Nothing has been removed, said Amy Adams, the major domo for the CIRM’s Internet operation. 

Writing on the agency’s blog, she explained one of the major changes dealing with “our funding” category.

“It turns out that people looking for information about research we've funded weren't thinking to look under 'our funding.' And since no other section of the site seemed like a likely place to find that content, people sent me helpful emails suggesting that I add pages about where our funding has gone (I politely directed them to that content).”

Most of the information on funding has now been moved to “our progress,” she said, leaving only information on how to get and manage grants under the “our funding” category. Other changes have occurred as well. You can read all about them here.

California Stem Cell Agency Blogs on Questionable Stem Cell Clinics

The California stem cell agency has taken notice of a piece in the New York Times that reports on questionable practices of enterprises that say they are stem cell clinics. 

In a blog item written yesterday by Don Gibbons, senior science and education officer at the agency, the agency said that the practices described in the article generated a cautionary advisory to patients last month by the stem cell agency and 12 other organizations. 

Gibbons noted that much of the Sept. 9 Times article by Laura Beil focused on one clinic,  the Regenerative Medicine Institute in Tijuana, which is just on the California-Mexico border. Gibbons’ item reported that one U.S. expert says that some of the clinic’s key procedures would not pass muster in this country.

Gibbons continued,

“Most important, the clinic has not produced any publishable data. We all have great hope for the promise of stem cell science, but I think it is fair to say that anyone who legitimately cares about that promise wants to find out what is the right type of cell to put in each patient. When is the right time for the transplant, and what is the best method of delivering the cells. We will never learn those things without collecting data in a well-designed clinical trial and sharing that data.”

The Times article quoted UC Davis researcher Paul Knoepfler as saying,

“There is absolutely no legitimate reason for such clinics to be not publishing their data.”

Gibbons also said that the International Society for Stem Cell Research, which backed away under threats of lawsuits a few years ago from some criticism of dubious stem cell clinics, plans to release this week a new statement on these sorts of treatments.

We should note that Times article, which is syndicated to many newspapers in this country, did not pick up the widely reported instances of deaths and injuries from these treatments. It did report that problems exist in this country as well as abroad. The article said, 

“In the United States, too, it is easy to conduct business outside government oversight, said Dr. George Q. Daley, who studies stem cells for blood diseases at Harvard Medical School. Close down one shady operation, he went on, and more seem to randomly pop up.

“Even questionable publicity does not necessarily hurt business. Regnocyte, a company in Florida, posted an unflattering CNN report about it on its Web site under the heading ‘special coverage.’”

Rise Up and Blog! Any Scientist Can!

The old saying has it that you can tell the pioneers by the arrows in their backs. 

Tell that to Paul Knoepfler, the UC Davis stem cell researcher who has been something of a pioneer in stem cell blogging and still is -- at least given that almost no other stem cell researcher blogs both as a researcher and patient advocate in addition to taking on wide-ranging business and public policy issues.

Knoepfler does have a few arrows in his back, some from enterprises that are less than happy with his vieww of their conduct and some from within the stem cell research community itself. But overall he has found the experience beneficial and rewarding. 

In an article in Nature Medicine Sept. 6, Knoepfler is recruiting more researchers for the stem cell blog brigade. To encourage them, he recounts his experiences and costs (such as $1,500 for his special domain name, although a normal domain name can be had for virtually no cost). His experiences include working late at night and on weekends. We can testify that the father of three puts in the hours, just based on the nature of the blog content and frequency of posting. 

Knoepfler continues his pitch in his piece in Nature, which is read primarily by researchers,

“To all this a reader might say, ‘Okay, this all sounds well and good, but you can't have your cake and eat it, too! Your science must have suffered from all this.’ In fact, I have lost some sleep, but I have not seen much in the way of negative scientific consequences. My lab has done very well during the past three years, and we have published many important papers, including one earlier this year that demonstrated the molecular similarities between induced pluripotency in stem cells and oncogenic transformation in cancer cells. I even secured tenure during this time.

“Reflecting on my personal transformation to the role of scientist−advocate, I have come to notice many tangible positive outcomes. In a general sense, my work has served to build bridges and stimulate new dialogue between industry and academia in the stem cell field. At the same time, I couple these efforts to accountability. Today, if someone does an Internet search for 'stem cell blog', that person will find my site, with all its educational outreach resources, at the top of the results list. A few years ago, the same search would mostly have yielded sites published by opponents of stem cell research or proponents of sketchy, for-profit endeavors to attract stem cell 'tourism'. I have also interacted with more than 100 patients and caregivers, helping them make more educated and, I believe, safer, stem cell−related medical decisions with their physicians.”

Knoepfler’s bottom line pitch? Any scientist can!

As he put it:

“Any scientist can, and should, do it. It is only logical that scientists would adapt to today's reality—a funding-poor environment that is nonetheless rich with opportunities for communication—by becoming advocates. I predict that any scientist who devotes a tiny bit of time to advocacy endeavors will find that the payoff is greatly multiplied. I will even help. Drop me an e-mail. Or better yet, leave a comment on my blog or message me on Twitter. Let's get the conversation going.”

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.