Stem Cell Directors Order More Consideration on $20 Million UCLA Research Application

Directors of the California stem cell agency today deferred action on a $20 million proposal that was rejected by its grant reviewers and sent it back for more consideration. 

The move involved an application by Stanley Nelson and M. Carrie Miceli of UCLA dealing with Duchenne muscular dystrophy. They are parents of a child with the affliction, which is usually fatal by age 25. The CIRM review summary said it is “a devastating and incurable muscle-wasting disease caused by genetic mutations in the gene that codes for dystrophin, a protein that plays a key role in muscle cell health.”

Nelson had filed an appeal seeking to overturn reviewers' action. Five  mothers and two fathers with children suffering from the disease made emotional appeals to CIRM directors on behalf of the application. Following their presentation, Art Torres, co vice chairman of the CIRM board, responded equally emotionally that the board is dedicated to finding therapies for such afflictions as Duchenne muscular dystrophy

The board approved more review for the application after it was disclosed that a company issued a press release two days ago that showed that  a drug involved in the proposal was more effective than reviewers believed. Philip Pizzo, a member of the board and dean of the Stanford medical school, expressed caution about the press release, given its timing and source. His comments came prior to the appearance of the parents.

The application is scheduled to be brought back to the full board in September for further action.

From Patient Advocates to Presidents: Decision-making Roles on Billions in Research

Directors of the California stem cell agency are moving to spell out the roles of the players who make the de facto decisions on its $3 billion in research funding.

This Wednesday evening in Burlingame, Ca., the directors' Science Subcommittee is scheduled to consider delineating the functions of members of the grant review group, also known as the Grants Working Group, along with CIRM staff in connection with grant reviews. Although the full CIRM board has legal authority to approve or reject grants, it almost never overturns a positive decision by the Grants Working Group on an application.

The agenda for Wednesay's meeting contains the full text of the addition to the working group's bylaws, but it does not say why changes are needed in the reviewers' closed-door procedures. However, from time to time, CIRM directors have commented during their public meetings that it is not always clear to the scientific reviewers what the full process entails.

Among other things, the bylaws addition spells out the role of the CIRM president, Alan Trounson, and other CIRM staff. It says the president can participate in discussions but cannot assign scores or vote. The president, however, has the top decision authority on grant pre-applications, which are a separate process. 

The new language makes it clear that the high-powered scientific reviewers can be expected, from time to time, to be asked by staff to explain themselves, or as the new bylaw phrases it, to “clarify their views or address specific issues in order to present a complete and useful” public review summary report to the CIRM governing board.

Also added to thebylaws would be this language,

“Prior to governing board consideration of GWG recommendations, the president and scientific staff should consider whether there are applications which they believe warrant particularly close review by the board, or whether specific modifications may be needed to successfully execute a particular proposal.”

The bylaw change also deals the roles of the eight patient advocates on the grant review group -- all of whom are also members of the CIRM governing board. They play a key role during “programmatic review,” an extremely broad-ranging process.

The bylaws addition states:

“Programmatic review is led by one of the GWG (Grants Working Group) vice chairs, who is a patient advocate, or a patient advocate member designated by the vice chairs. During programmatic review, patient advocate members of the GWG join the scientist members to make and vote on programmatic motions and funding recommendations to the gtoverning board. Programmatic review is intended to allow consideration of issues beyond scientific merit, such as disease representation and societal impact. In making funding recommendations to the governing board, GWG members consider the scientific merit of each application, as reflected in the scientific score, as well as any programmatic issues raised.”

The chairman of the CIRM Scientific Subcommittee is a patient advocate, Jeff Sheehy, who is also vice chair of the review group and plays the leading patient advocate role in the CIRM working group. Additionally, Sheehy, a communications manager at UCSF and nationally known HIV/AIDs advocate, almost invariably leads the full board discussion prior to action on grant applications.

In addition to the meeting location, the public can participate in the session at a teleconference site at UC Irvine. The address can be found on the agenda.

Royalty Rules at the California Stem Cell Agency: Business Friendly Changes Proposed

If you are looking to follow the money trail at the $3 billion California stem cell agency, next Thursday's meeting of its 29-member board of directors is a good place to start.

On the agenda are revisions in its intellectual property rules, which are all about who gets paid and how much and when – should an agency-financed product generate significant cash.
The key question about the proposed changes is whether they will generate an appropriate return for the state, given its $6 billion investment, including interest on the bonds that finance CIRM. The impact of the changes is not crystal clear. And the staff memo does not mention two important definition changes that appear to be quite business friendly.

During the 2004 ballot campaign that created the stem cell agency, California voters were told that the state would share as much as $1 billion or more in royalties. Eight years later, no royalties have materialized since CIRM research has not yet resulted in a commercial therapy. 

At next week's meeting in Burlingame, directors will be asked to modify CIRM rules for royalties that CIRM staff said "could be a disincentive" for business. A staff memo said the proposals would alter provisions that create "administrative challenges and uncertainty." The memo asserted the proposed changes would ensure "a comparable economic return to California" equal to the existing provisions. However, the memo provided no explanation or evidence for how that result would come about. The proposed changes could also be applied retroactively with the agreement of CIRM and the grantee.

Currently CIRM grantees and collaborators must share as much as 25 percent of their licensing revenue in excess of $500,000, depending on the proportion of agency funding for the product. The IP rules also contain a provision for payments in the event of development of a "blockbuster" therapy. The staff memo described how that would work.

“It provides that grantees and collaborators must share revenues resulting from CIRM funded research as follows: after revenues exceed $500,000, three times the grant award, paid at a rate of 3% per year, plus upon earning $250M(million) in a single calendar year, a onetime payment of three times the award, plus upon earning revenues of $500M in a single calendar year, an additional onetime payment of three times the award and, finally, in the instance where a patented CIRM funded invention or CIRM funded technology contributed to the creation of net commercial revenue greater than $500M in a single calendar year, and where CIRM awarded $5 million or more, an additional 1% royalty on revenues in excess of $500 million annually over the life of the patents.”

The proposed changes would exempt "pre-commercial revenues" from the state's revenue sharing, the memo said, in order to maximize the amount businesses can "re-invest in product development." The proportionality payment provision would be changed to require only 15 percent of licensing revenues if CIRM's investment is less than 50 percent and 25 percent if it is more than 50 percent. Revenue sharing would be extended to "commercializing entities." No definition of "commercializing entities" was provided in the board agenda material, but a June version of the changes defined them as "A For-Profit Grantee and its Collaborator or Licensee that sells, offers for sale or transfers a Drug, product(s) or services resulting in whole or in part from CIRM-Funded Research."

Not mentioned in the CIRM staff memo were two new provisions in the rules involving the definition of licensing revenue and the sale of a therapy. Both could be construed as quite favorable to businesses. According to the June version of the changes, licensing revenues are defined as a figure minus "a proportion of expenses reasonably incurred in prosecuting, defending and enforcing related patent rights equal to CIRM’s percentage of support for development."  The sale provision says that royalties on "net commercial revenue" are not due until received from sales in the United States or Europe. That provision would appear to exclude California from receiving royalties on product sales in most of the world, where it is easier to receive regulatory approval for sale of new therapies and drugs. (See here -- page 2 -- for royalty provision and here for definition of "first commercial sale"-- page 3.)

The existing IP regulations are enshrined in a 2011 state law. However, the law also provided that they can be altered by the agency, the CIRM memo said, “if it determined that it was necessary to do so either to ensure that research and therapy development are not unreasonably hindered as a result of CIRM’s regulations or to ensure that the State of California has an opportunity to share in the revenues derived from such research and therapy development.”

The memo continued,

"The proposed amendments re-strike the balance both to ensure that industry will partner with CIRM and to ensure that the State has the opportunity to benefit from successful therapy development."

Board action next week will give the go-ahead for posting the proposals as part of the official state administrative rules process. They are subject to additional changes in that process. 

The agenda originally contained the full text of the changes. However, that material has been dropped from the board agenda. An earlier version can be found here and here. We have queried the agency about the reason for dropping the text in the board agenda.

(Editor's note: The agency has now reposted the version of the text of the changes that was on the agenda earlier, saying that it was having problems with its web site. For the definitions of terms, however, it is still necessary to refer to the June documents.)

Stem Cell Confab Attracts Modest Media Attention

The World Stem Cell Summit generated light news coverage on the first day of its meeting in Pasadena, Ca., while the California stem cell agency posted on the Internet six videos filmed during the three-day conference.

Our search turned up only three news stories this morning on the meeting: One in the Pasadena Star News, one on the Chinese news agency Xinhua and one on radio station KPCC. The first two focused largely on efforts to turn research into cures.

Beige Luciano-Adams of the Pasadena paper wrote,

"Hundreds of pioneers and leaders in stem cell research are in Pasadena this week for the World Stem Cell Summit. They're discussing stem cell breakthroughs – and roadblocks."

She quoted Andy Grove, former CEO of Intel and now a patient advocate, as saying,

"'Today patents are used to keep products off the market. This is lethal. This is not what the founders of the (U.S.) patent office envisioned."

Xinhua wrote,

"How do you take the phenomenal scientific research going on in labs and translate it into medical treatments?' said Bernie Siegel, the founder and co-chair of the summit and executive director of the Genetic Policy Institute, which organized the event."

CIRM's communications manager, Amy Adams, posted six videos of interviews with attendees at the conference. One 2-minute, 30-second spot featured Jonathan Thomas, chairman of the California stem cell agency.

He said the single thing he hoped attendees would take away from the conference was the "grand scale of things we currently have in the pipeline." He said the agency has funded research on 26 different diseases. Overall, CIRM has given away $1.3 billion to 453 recipients since 2005.

More videos are likely to be posted in the next two days by CIRM, which has a booth at the meeting and provided $175,000 to support the conference, including funding to send as many 125 persons to the affair.

(Editor's note: An earlier version of this item carried an incorrect identification of the Pasadena Star News reporter.)

Andy Grove: More on Speeding Cures

Recently we carried an item on Andy Grove's views on translational medicine, a subject that now deeply engages the California stem cell community. Today, the San Jose Mercury News ran an interview with Grove, a Silicon Valley pioneer, former CEO of Intel and currently a patient advocate. The piece dealt with his campaign to generate therapies more quickly as well providing a glimpse into his current life.

The article by Lisa Krieger is well worth reading. Here are some excerpts.

"He handles (Parkinson's) disease the way he handles everything else: open and direct. He adopts none of the common tricks employed by sufferers -- clutching a pen, or stuffing hands into pockets -- often used to minimize its visibility. His torso keels with each step, a distorted but determined gait. His arms bob; his fingers fidget.

"Underneath is keen intellect and a growing sense of frustration that medical science has offered him and other patients so little after decades of experimentation."

Krieger continued,

"'He's turned his own encounters into problems to be solved,' said Robert A. Burgelman, professor of management at the Stanford University Graduate School of Business. "He takes a strategic approach.

"'He's an extraordinary man, one of the most focused persons I have every come across. Once he comes to an insight, he follows through on it, turning it into action,' he said. 'And he has an ability to reflect on his own experience and draw insights from that, which is an unusual and powerful thing for leaders to have.'"

Stem Cell Blogging from Toronto

For a close-up, on-the-scene look at the biggest stem cell conference in the world, check out the blog on the web site of the California stem cell agency.

Don Gibbons, CIRM's chief communications officer, has filed one blog item already about and is Tweeting a couple of times a day. He wrote,

"What a rapt full house of attendees heard was an impassioned evening of hope and excitement balanced nicely with restraint and patient persistence."

Gibbons said in an email this morning,

"I am on the ISSCR education committee that plans the opening public session that I wrote about in the blog and made an oral presentation the following day on public education and (the CIRM) high school curriculum project."

CIRM has also sent 20 patient advocates to the meeting in Toronto of the International Society for Stem Cell Research as part of a $200,000 program. Don Reed of Fremont, Ca., is one of them. He is also filing reports on his blog.

World Stem Cell Summit Garners Support from State Stem Cell Agency

The California stem cell agency board today approved $125,000 to send as many 125 persons to the World Stem Cell Summit in Pasadena in October.

Up to 75 would be patient advocates, a group that will be key in drumming up support for a new $3 billion to $5 billion bond ballot measure that has been proposed by by CIRM Chairman Robert Klein. The other 50 would be researchers and others involved in CIRM grant programs. The subsidies would be paid with funds donated to the agency by private parties.

The stem cell meeting is sponsored by a stem cell advocacy organization, the Genetics Policy Institute.

Board members raised questions about the cost of the registration -- $495 -- at the convention. They also asked whether any speakers or parts of the program would be controlled by industry sponsors. A representative of the convention said sponsors cannot "pay to play."

During the board discussion, CIRM staff disclosed that the agency also expected to receive a request for a $50,000 conference grant to support the meeting from Caltech, one of the convention sponsors.

Massive, $243 Million Disease Round Gets Okay from Stem Cell Agency

Directors of the California stem cell agency today gave the go ahead to a $243 million disease team grant round aimed at generating a “development candidate” for a clinical trial, doubling the size of the original proposal by the CIRM staff.

The huge round would fund up to 30 planning grants and 12 full grants of up to $20 million. It would come on top of the initial disease team round of $250 million last October. The application process for the latest round would begin this November with planning grants, which are required to be eligible for full grants. Full grants are scheduled to be approved in 2012.


The size of today's round was doubled at the suggestion of CIRM Director Jeff Sheehy. He said most of the disease team efforts are risky and expected to fail. He said it was important to get more in the pipeline.

Sheehy first proposed 45 planning grants and 15 full grants. CIRM President Alan Trounson resisted the increase in the number of grants, declaring it would overtax staff and reviewers. After some discussion, Sheehy agreed to 30 planning grants and 12 full grants.

The board also approved a more than $600,000 study of its operations by the prestigous Institute of Medicine that would be expected to completed by the general election of November 2012. Chairman Robert Klein said the study would be key to winning voter approval of more billions in state bonds to fund stem cell research.

Sheehy supported the proposal, declaring it would be taken “very seriously” by editorial boards and other segments of the media as well as the public.

Duane Roth, co-vice chairman of the stem cell board, dissented. He said the study will require considerable work on the part of staff. He also said the board should not have “blind trust” that the findings would be ones desired by the board.

The study will be funded with part of the $3.5 million that CIRM has in private donor funds. The cost could increase beyond the $600,000 range because CIRM directors indicated they wanted some changes in the scope of the study. CIRM is currently involved in an expensive external review of its strategic plan and will be subject to a performance audit($400,000 or so), which it will also pay for, under the terms of legislation expected to be signed into law.

The board also approved paying its patient advocate members up to $15,000 annually. That action came during a session late yesterday, which CIRM had assured us would be solely an executive session.

In other actions, the board approved procedures for selection of a new chair and vice chairman. Klein has said he will step down as chairman in December. However, if the board does not vote on a replacement, he will continue in office.

Francisco Prieto, a Sacramento physician, was named chairman of the directors' Evaluation Subcommittee, and Ted Love, executive vice president of Onyx Pharmaceuticals, Inc., of Richmond, Ca. , was named vice chairman. The panel evaluates the performance of the chairman, vice chairman and president of the agency.

Here is a link to the CIRM press release on the meeting.

CIRM Directors Pay Proposal Advances; Another Openness Failure on Grants Management System

The California stem cell agency is set to pay 1/3 of its 29-member board of directors as much as $15,000 a year.

The action, however, is contingent on passage of legislation (SB1064) that is now in the “suspense” file in the Assembly Appropriations committee. Legislation in “suspense” is usually on hold pending action on the state budget, which is not likely to be adopted any time soon.

According to a CIRM spokesman, the agency's directors' Governance Subcommittee on Tuesday approved the new pay policy, which is aimed at compensating the 10 patient advocate members of the board for the extra time that they must put in. The policy will go before the entire board later this month.

A large part of the burden placed on patient advocate members of the board stems from the conflicts of interests built into the board by Prop. 71,  drafted by CIRM Chairman Robert Klein and a handful of others. The ballot initiative requires a super-majority (65 percent) for a CIRM governing board quorum, which is based on the number of board members eligible to vote. Since many of the board members have conflicts of interest that prevent them voting on matters before the board, the presence of patient advocates is necessary to take action – much more so than many other members of the board.

According to Don Gibbons, CIRM's chief communications officer, the subcommittee took no action on staff reports on CIRM's ongoing efforts to build a custom system to deal with its $3 billion in grants. Directors asked for a report after expressing concerns the effort could be an expensive failure. Details on their discussions will have to wait for release of the transcript.

However, once again, CIRM failed in its responsibility to the public by not posting the reports publicly in a timely fashion. The failure effectively bars the public from being able to make informed comments and violates CIRM's promise of highest standards of openness and transparency. Two reports were prepared. One did not appear until one business day before the Governance Subcommittee meeting. The other appears to have been posted on CIRM's Web site only minutes before the meeting was scheduled to begin.

We will have more on the grants management system when the transcript is posted.

Pay Proposal for Patient Advocates on CIRM Board

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

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

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

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

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

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

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

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

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

CIRM Airs Powerful Patient Video

With some regularity, the board of the California stem cell agency hears directly from some of those afflicted with diseases and conditions that are among the targets of its $3 billion in research.

All of their stories are emotional, sometimes heart-rending.

The CIRM staff recently captured on video one of the more powerful presentations at its meeting in San Diego last June. Called “Spotlight on Leukemia,” it is now available via CIRM's YouTube site.

The video is a fine piece of work that tells a strong human story and well supports CIRM's mission.

Don Gibbons, CIRM's chief communications officer, aired the video at last month's CIRM board meeting. He said,

“Amy Adams led the story direction and Todd Dubnicoff did the amazing video editing.”

It is narrated by Geoff Lomax, senior officer for the CIRM Standards Working Group.

Patient Advocate Slams CIRM Reforms as Ludicrous

Patient advocate Don Reed recently presented his views on the Little Hoover Commission's preliminary recommendations for improvements in the operations of the California stem cell agency.

The piece on his blog, stemcellbattles.com, used terms like “worst fears,” “disaster,” “ludicrous” and “politicizing.” Reed said the the agency is highly successful. “Why gut it?” he asked.

Reed has followed the agency since the campaign to create it in 2004. He is vice president for public policy of Americans for Cures, which is the private stem cell lobbying group of the chairman of stem cell agency, Robert Klein. He is only one of two staff members currently listed on the group's Web site.

We do not agree with Reed's analysis of what the Little Hoover Commission is considering, and we think that some of his information is off target. For example, Reed asserts that five audits have determined that CIRM is “open and honest.” In fact, all but of one of those audits had a quite limited scope involving compliance with accepted accounting practices. And as many persons know, compliance with accounting standards does not signify a healthy or open enterprise. One recent case in point is General Motors.

The only audit to go beyond such limited scope was a “performance audit” by the state's auditor, but it also did not examine CIRM's openness or honesty.

Like Reed, we support CIRM's endeavors. But we believe that it is hampered by unnecessarily restrictive provisions in Prop. 71, which are now codified in state law and the state Constitution and virtually impossible to change. Many of those provisions are management minutia that should have been left to the CIRM board to decide. Those include the dual CEO structure, super-majority quorum requirements and a poorly thought-out cap on the number of CIRM employees. That is not to mention the conflicts of interest on the board that were built into the initiative in order to win political campaign support from all the key institutions and businesses.

Reed is attempting to drum up a letter-writing campaign from patient advocates and “true believers” in stem cell research to persuade the Little Hoover Commission to pull back on its recommendations.

While we respect Reed's beliefs, the Little Hoover Commission should pay no more attention to “true believer” letters than the NIH should to the thousands of letters it has received from the religious right opposing its proposed rules on human embryonic stem cell research – at least as far as both rely on faith-based reasoning.

The California stem cell agency is giving away $3 billion in taxpayer funds at a cost of another $3 to $4 billion in interest. Evaluation and support of the effort must be based on hard facts and conditioned on how well it actually meets its public responsibilities and its much-touted adherence to the highest standards of openness and transparency.

Rutgers' Young on Geron Trials

Wise Young, the director of Rutgers Center for Collaborative Neuroscience and a grant reviewer for CIRM, has pulled together a fine overview of the science and media coverage of the Geron clinical trial.

In a piece complete with numbered footnotes, Young (see photo) wrote,

"The media response was massive [18]. The story was carried by almost every news source [18-20]. The community response was initially strongly positive. Coming on the 3rd day after President Barack Obama’s inauguration, some thought that the approval of the first HESC trial was due to Obama’s coming to power.

"The exuberance faded as people read the fine print. First, the trial is not for people with chronic spinal cord injury. It is intended to be used within 2 weeks after injury for people with complete thoracic spinal cord injury. Second, the goal of the trial is to show safety and feasibility, not necessarily efficacy. Third, the cells have been differentiated to the point that they are no longer acting as stem cells but only as oligodendroglia."

Young also wrote,

"Geron’s web site and news reports indicate that the trial will treat 8-10 patients who are within 2 weeks after “complete” thoracic spinal cord injury. It will probably start in July 2009. However, many details are unclear. Before the FDA placed a hold on the trial application in May 2008, Geron had said that the cells would be transplanted into the spinal cord of patients undergoing spinal cord decompressive surgery and all the patients will receive a 2-month period of pharmacological immunosuppression . It is not clear that the same regimen will be used.

"In the meantime, the reaction of the spinal cord injury community has ranged from exuberance over the approval of the first HESC trial [22] to deep pessimism over comments by Okarma, who said that people with “complete” spinal cord injury have no chance of recovering any function, or something to this effect. Many people in the spinal cord injury community [23] were disappointed at being excluded from the study which is only for the newly injured."

From the Reed Family: A Thankful Perspective on the Geron Trials

Don Reed of Fremont, Ca., is a nearly tireless and good-hearted advocate for human embryonic stem cell research. Today's announcement concerning the Geron clinical trials has special meaning for him and his family. We asked him for comment. Here is what he sent.

"MAGNIFICENT BEGINNING

"Don, Roman, and the entire Reed family would like to congratulate Geron Corporation and the stem cell research community for today's wonderful news.

Years ago, California's Roman Reed Spinal Cord Injury Research Act of 1999 funded Dr. Hans Keirstead's pioneering work, just approved for human trials by the FDA. These particular trials, to re-insulate nerves in the damaged spine, will involve only newly injured individuals. But they are forerunners of the day when paralysis will no longer be an automatic life sentence in a prison of immobility.

"Roman was asked by a reporter: what was the first thing he would do on the day when regenerative medicine allows him to walk again?

"He replied: 'I want to walk down the beach, hand in hand with my wife Terri, and toss a ball around with my children.'

"A small dream -- and an enormous one-- because if we can cure paralysis, the very symbol of that which has been called incurable, we can do anything.

"Congratulations to all, and may the New Year, and the new administration, live up to this magnificent beginning.

"Thank you,"

Don C. Reed

Advocate Reed Says No Need for Independent Look at CIRM

On Sept. 5, 2008, we carried an item dealing with a proposal for an examination of the California Institute for Regenerative Medicine by a bipartisan state department charged with improving government operations.

We said that corporations pay hundreds of thousands of dollars or more for independent assessments of their operations and that CIRM should welcome the opportunity to have a similar study performed for free. The springboard for our comments was an item written by patient advocate Don Reed on his blog, stemcellbattles.com. Reed has responded to our item. Here is what he sent us.

"Several days ago David Jensen printed a thoughtful article inquiring why I was encouraging people to write to Governor Arnold Schwarzenegger ( State Capitol Building, Sacramento, CA 95814), urging him to veto Senate Bill 1565.
 
"The reason is simple. If people like our stem cell program as it is (I do), they should defend it. The Governor will be deciding in the next few days whether to accept SB 1565, or veto it.  This is the only chance we have of preventing SB 1565 from becoming law.
 
"SB 1565 could completely restructure California’s stem cell research program, as well as removing our state’s official preference for embryonic stem cell research.
 
"The article criticized advocates like myself who are allegedly “blind to blemishes… (and who) regard any evaluation or analysis of the effort as destructive.”
 
"I don't see it that way. As someone who attends and participates in most of the meetings of the stem cell program, I see a constant and vigorous process of adjustment and correction.  The CIRM has had (by my count) four in-depth official audits; the California Court system has examined our (California’s) structure.  Evaluation and analysis is ongoing, and the public is involved in every step of the way. That’s good; it’s democratic; it’s necessary.
 
"What I do object to is throwing out something magnificent after the decision has been made.  California fought for our stem cell program, debated over it, and voted.
 
"Why did we have to use the initiative process?
 
"Practicality!
 
"I fought for three years trying to pass the Roman Reed Spinal Cord Injury Research Act—and California ended up with only $1.5 million a year for that program. It is a good program, spending $12 million, but bringing in more than $50 million dollars in matching grants, new money for the economy—but it is so little, compared to the need.
 
"As a grass roots organizer I helped with Senator Deborah Ortiz’s groundbreaking legislation. She did a terrific job, gaining permission for the (then) controversial new medical procedure. To me, she will always be the Mother of California stem cell research. But those bills came with no money. It took a real fight to get a lousy hundred thousand bucks to run a committee, and no research money whatsoever.
 
"I remember a group of advocates meeting with Senator Barbara Boxer, a true friend of research, and she told us: “What you need is billions of dollars, and we can’t get that for you in Sacramento.” 
 
"So Sacramento couldn’t raise the money to fund the research. Washington dropped the ball for political reasons.
 
"But California came through.
 
"Embryonic stem cell research offers hope for cure not only for my paralyzed son, but also for Sarah Palin’s boy who has Down’s Syndrome, and for millions more who suffer with incurable illness or injury.  
 
"SB 1565 would remove California’s official preference for embryonic stem cell research, which was the reason we passed the program in the first place. Unfortunately the new Republican platform calls a complete ban on embryonic stem cell research, both public and private, criminalizing it completely. SB 1565 is co-authored by Senator George Runner, a long-time opponent of embryonic stem cell research, and if that bill passes, it could provide ammunition to the opponents of the research.
 
"And the program itself? The California structure--with decisions made by patient advocates and experts-- was designed specifically to keep the focus on the quest for cure. No one wanted it to become a self-serving bureaucracy, rambling around in search of reasons to justify its existence, making endless speeches and not actually doing a lot.
 
"We wanted intelligent action, and we got it.
 
"So of course we are going to defend it. Can someone point me to a better program, anywhere in the world?
 
"If so, let me know. In the meantime, I think we should protect what we won.
 
"Anybody that cares about stem cell research should write Governor Schwarzenegger today. (tip: on the outside of the envelope, put VETO S.B. 1565, that helps them."

A Partial Case for a Deep Outside Look at CIRM

California patient advocate Don Reed has embarked on a letter-writing campaign to have the governor of the state veto legislation aimed at ensuring affordable access to taxpayer-financed stem cell therapies.

Of course, Reed, a longtime and effective advocate for stem cell research, does not see the legislation that way. In an appeal on his blog to the patient advocate community, he warns that SB1565 "intends to restructure" the board of directors of the $3 billion California stem cell research effort. With all due respect to Reed, he has overstated the case in a way that does not necessarily well-serve the best interests of the California Institute for Regenerative Medicine.

His assertion is based on the measure's request for a study of CIRM by the state's bipartisan Little Hoover Commission. Even if the commission performs the study and recommends structural changes, their enactment is remote. They would have surmount a huge barrier, including possible alterations in the state Constitution. That would require a two-thirds vote in both house of the legislature and a vote of the people.

Reed's latest outpouring against the legislation embodies in some ways the deep-seated concerns among some stem cell advocates that somehow CIRM could be thwarted. Many supporters of the state research effort are wary of any public scrutiny of the agency. They are blind to blemishes. They regard any evaluation or analysis of the effort as destructive. The true believers also sometimes seem to be bent on funding hESC research regardless of whether the state of California benefits significantly from the expenditure of $6 billion in public funds, including interest.

Prop. 71 created the California Institute for Regenerative Medicine. The ballot measure was not hammered out in a public process. It was written behind closed doors with no public input. It was put before the people because someone ponied up $1 million-plus to gather the necessary signatures to place it on the ballot. The initiative placed representatives of the beneficiaries of the $3 billion in grants in control of the rules for giving away the money, a built-in conflict-of-interest that naturally raises concerns. Prop. 71 also created difficulties that have repeatedly hampered CIRM's operations, including an unusual quorum arrangement that forced directors last month to fill out their panel by picking a member from the audience. All of which is legal under Prop. 71. And then there is the dual executive arrangement that helped to create unhealthy management tension early on.

All public agencies need and deserve outside scrutiny. It is one of the basic principles of American government and is embodied in the concept of checks and balances amid the three branches of government. CIRM operates largely without those checks. For all practical purposes, it is free from fiddling by the executive or legislative branches of California government.

CIRM is an extraordinary experiment, unprecedented in California history. Some believe it could serve as a model for successfully tackling other difficult social problems. It is important that it be successful, fulfilling its mission efficiently and in a manner that recognizes its first responsibility is to the public – not the scientific community, not patient advocates and not industry. They are all exceedingly important constituencies, but CIRM is first a public endeavor. If CIRM is perceived to be under the control of those groups, its credibility will be damaged. Public trust can be mercurial. It can easily vanish overnight, and the whole field of hESC research can become besmirched.

Corporations regularly pay hundreds of thousands of dollars – if not millions – to outside consultants to analyze and critique their operations as they strive to remain efficient and competitive. The Little Hoover Commission will basically do the same job for free for the California Institute for Regenerative Medicine. CIRM should welcome the commission and use the opportunity to build support for making some of the difficult changes that could make it more successful in its mission.

Stemcellbattles.com Is Offline; Webmaster Hospitalized

We received the following from patient advocate Don Reed of Hayward, Ca.

"Karen Miner’s and my weblog, www.stemcellbattles.com, is down.

"Webmaster Karen(pictured) is in hospital, recovering from bladder augmentation surgery. The operation was done in hopes of freeing her from many problems of a personal nature, related to her spinal cord injury of fourteen years.

"We are in hopes she will recover fully, but for the foreseeable future, our small column, www.stemcellbattles.com, will not go forward. Karen was the webmaster; now her health will not permit her to carry on this unpaid chore.

"Selfishly, I am devastated.

"Not only did Karen’s webmastery give me a voice, but she and I have worked closely together for more than ten years. She is a part of everything I have done in research advocacy. Co-chair of Californians for Cures, Karen worked hard on the Roman Reed Spinal Cord Injury Research Act, Senator Deborah Ortiz’s stem cell laws, Bob Klein’s magnificent and unparalleled Proposition 71, and the four years continual defense of it afterward, in the unending attacks the California Institute for Regenerative Medicine has endured; always, Karen was there.

"Paralyzed in body but never in spirit, Karen Miner is a constant source of inspiration to me and all who have the blessing of knowing her.

"Anyone wishing to send Karen a get-well-soon email may send it me at: diverdonreed@pacbell.net. I will print them out and take them to her in the hospital.

"Don’t send anything electronically complicated or fancy, no e-cards, please. I don’t know how to work that stuff. Thanks.

"Don C. Reed
"Co-chair, Californians for Cures"

The Harsher Reality of the FDA Hold on Geron

At least one analyst is minimizing the impact of the FDA's hold on Geron's clinical trials for its human embryonic stem cell product.

Ren Benjamin, an analyst with Rodman & Renshaw in New York, told Bloomberg News earlier this week said that he thinks the company will "be able to work through the issues with the FDA."

However, the reality is likely to be a tad harsher. The hold is the second pronounced signal in two months from the FDA that it wants to proceed with extreme caution on hESC trials. And the action has stirred some concerns in the patient advocate community, which has pressed for more speed on the research.

Even prior to the Geron hold, Don Reed, vice president for public policy for Americans for Cures, worried about the FDA posture on his blog, stemcellbattles.com. He wrote on May 6,

"The FDA hearings on embryonic stem cells have been (in my view) deeply politicized, setting the stage to block human trials, an enormous setback."

He continued,

"A laundry list of new conditions may send...Geron, Advanced Cell Technology, and Novocell, and the entire embryonic stem cell research field back to the drawing board, conceivably for
decades."

Reed referred to the FDA hearings last month during which the bugaboo of teratomas was raised by the FDA. Monya Baker of Nature's Niche stem cell blog covered the meeting. On April 11, she wrote that the consensus from attendees at the meeting was that the FDA "seems cautious about moving forward, but not spooked."

But then came the FDA hold this week. It had to be a bitter pill for Geron. Thomas Okarma, CEO of the company, said that the company had spent four years working with the FDA and amassed 21,000 pages of documents to alleviate the agency's concerns.

Geron's stock took a big hit on the day the hold was announced, dropping to a 52-week low of $3.76. It stood at $3.97 at the time of this writing, compared to a 52-week high of $9.85.

Geron has been mum since Okarma's statement along with Novocell and Advanced Cell Technology, although we have asked for comments from all three. We are likely to hear more from Geron after it receives the FDA's written concerns.

As for analyst Benjamin's somewhat optimistic position, his firm is a market maker in Geron stock, which means that it has a stake in the company's well-being.

Affordable Access to CIRM Therapies: A Patient Advocate's Position

For a patient advocate's take on legislation aimed at ensuring affordable access to CIRM-financed therapies, take a gander at the May 13 posting by Don Reed on his blog, stemcellbattles.com.

Earlier this week, Reed (see photo) was the only person to testify before the state Senate Appropriations Committee on the measure, aside from the bill's author, Sen. Sheila Kuehl, D-Santa Monica.

Reed's view is that CIRM's regulations, which can be easily changed unilaterally by CIRM, are sufficient to provide affordability. He also contends that an outside study of CIRM's structure is unnecessary and that its built-in conflicts are only a "convergence of expertise." We should note that last week most of the experts on the panel were barred by law from even discussing -- much less voting on -- $271 million in grants because of their conflicts of interest.

Reed is a regular at meetings of the board of directors of the stem cell agency and is vice president for public policy for Americans for Cures, the private advocacy group directed by CIRM Chairman Robert Klein.

Kuehl's bill, SB 1565, was approved 14-0 by the committee and is now on the Senate floor. In addition to codifying in state law affordable access, the measure would require an independent study of CIRM by a state agency known as the Little Hoover Commission with recommendations for reform.

A note on the analysis from the Appropriations Committee says that the Little Hoover staff contends that the legislature does not have the legal authority to direct its work. An opinion as been requested from the Legislative Counsel's office on the question. However, the Legislative Counsel works for the legislature and is inclined to find ways for lawmakers to do what they want.

Here is a link to the floor analysis available to California state senators as they consider the measure.

(Editor's note: After this item was posted, we learned that the bill passed the Senate on Thursday on a 40-0 vote. It nows goes to the Assembly.)

Corn, Ham and the Women of CIRM

From Hollywood's Sherry Lansing to Davis' Claire Pomeroy, patient advocate Don Reed covered them all. Wonder Women, he called them.

Reed is the irrepressible patient advocate who doggedly attends almost every meeting involving the California stem cell agency and then goes on to fight the stem cell fight nationwide via the Internet.

On his blog – stemcellbattles.com – the July 31 posting involved the women on CIRM's Oversight Committee. Reed did not mention the women who staff the agency, including the two who are now in charge of the effort. But he probably will.

With Lansing, Reed reached into the past and rented a movie, Rio Lobo, in which she appeared with John Wayne. Lansing's heart-of-gold character did away with a bad guy who had done her wrong, but then helped Wayne to walk again.

Reed himself is a goodhearted fellow. Some people might think his writing style a little corny, but I once had a high school speech teacher who constantly reminded me, “Corn is better than ham any day of the week.”