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Category Archives: Stem Cells

Some California Stem Cell Board Members Bristle at IOM Recommendations

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Some directors of the $3 billion California stem cell agency today sharply challenged recommendations by the Institute of Medicine(IOM) concerning conflicts of interest and the role of patient advocates on the governing board.

Much  of the criticism came from the 10 patient advocate members on the board, whose roles would be significantly altered  under the IOM recommendations. Jeff Sheehy, a patient advocate for HIV/AIDs and vice chairman of the agency's grant review group, said he saw no evidence in the IOM report for its recommendations regarding patient advocates. He said,

 "If you had some here, I would be more comfortable." 

He continued,

"We are not all powerful. We are a minority on the (29-member) board." 

Jonathan Shestack, another patient/adovcate director, said the IOM's conclusions on "conflicts of interest could not possibly be more incorrect than they are." He said,

"Advocates are here to advocate."

Harold Shapiro, chairman of the IOM study, said, "We are not against patient advocates." He said that the IOM supports advocates and that its recommendations could increase the role of patient advocates, albeit in a different manner.

Shapiro added that conflicts of interest do not necessary bar participation by board members. He said, however, they must be disclosed and managed.

Director Robert Price of UC Berkeley said,

 "We have gone to great lengths to manage conflicts of interest."

The IOM recommended that all board members be removed from the grant review process, which would be turned over to the CIRM president. The board would only vote on a slate of applications, not individual grants. More disclosure would be required of personal conflicts of interest, including health matters, that the board said research has shown can create bias.

 The IOM report said,

 “Far too many board mem­bers
represent organizations that receive CIRM funding or benefit from
that funding. These com­peting personal and professional
interests com­promise the perceived independence of the ICOC(the
CIRM governing board), introduce potential bias into the board’s
decision making, and threaten to undermine confidence in the board.”

More than 90 percent of the $1.7 billion that the CIRM board has awarded has gone to institutions that are represented on the CIRM governing board.

J.T. Thomas, chairman of the CIRM board, said that the agency takes the recommendations "very seriously" and that they would discussed further at a board workshop in early January. The workshop is scheduled to be public but the date and location has not been announced. Thomas said the recommendations will receive "lengthy discussion" thereafter and review by appropriate subcommittees of directors.

No one from the public commented during the roughly 90 minute discussion.  Eighteen out of 29 board members were present at the beginning of today's meeting.

Source:
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A Singular Scientific Story: Duchenne's Disease, a Family and the California Stem Cell Agency

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Unusual and personal conditions,
including a tie to the $3 billion California stem cell agency,
surround a promising scientific development reported today by a
husband and wife research team at UCLA. 
Their research involves Duchenne
muscular dystrophy (DMD), an inherited disease that afflicts about
one in 3,600 boys and results in muscle degeneration and, eventually,
death.
Carrie Miceli
UCLA photo
The researchers, Stan Nelson and Carrie
Miceli, said they have discovered a promising FDA-approved drug that
could advance the fight against the affliction.
Miceli and Nelson have an 11-year-old
son, Dylan, with the disease. They have been studying the affliction
for some time, but their most recent and ambitious research plan was
rejected earlier this year by stem cell agency grant reviewers, a process that
normally kills an application. Undaunted, Miceli and Nelson appealed
to the full stem cell agency board last July. Backed by an emotional
presentation involving patient advocates, they won approval of a $6
million grant.
Adding to all this, their appeal used a
process known as an “extraordinary petition,” which the Institute
of Medicine (IOM) last week said should be abandoned because it undermines
the integrity of the CIRM grant review process.
Stan Nelson
UCLA photo
Even prior to the IOM recommendation,
the CIRM board was moving to restrict its free-wheeling
appeals procedures.
As for the $6 million CIRM grant, it will carry the important burden of advancing the findings that were published in the
journal Science Translational Medicine. However, the money was not used in the study reported today.
The research showed that dantrolene, the FDA-approved drug, “provides a powerful boost to
the (Duchenne) therapy currently being tested in clinical trials,”
according to a UCLA press release.
The news release said,

“(The researchers) hope this one-two
punch used in combination will overcome the genetic mutations that
cause DMD, restore a missing protein needed for proper muscle
function and allow those affected by the disease to lead relatively
normal lives.”

“Their youngest son, Dylan, 11, was
diagnosed with DMD in 2004. While he’s still ambulatory – many
DMD patients require the use of wheelchairs by about age 10 – Dylan
can no longer run or climb stairs and he can’t shoot a basketball
over his head like other boys his age.  Despite these
challenges, Miceli said Dylan remains a happy, funny and engaged boy,
full of life and passion.

“'We entered into this field because
of the diagnosis of our son, but we hope our research can help many
others,' she said. 'There are drugs that can help manage the symptoms
of the disease, but nothing that changes its course dramatically.
We’re trying to correct the defect that causes DMD with highly
personalized genetic medicine.'” 

UCLA said the grant from CIRM will be
used for “longer term studies of their drug combination therapy in
mouse models to ensure it can restore dystrophin levels to normal or
near normal levels. They also will explore whether DMD patients with
other mutations can benefit from the combination therapy.”

Asked for comment, Kevin McCormack, a
spokesman for the stem cell agency, said today's findings "are certainly
very encouraging." He continued,

"Clearly there is still a long way
to go before we know if this approach will work in people but we're
delighted that funding from the stem cell agency is helping  the
researchers move their work forward....This is what voters set out to
do when they approved Proposition 71 to create the stem cell agency."

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/aAacD9507GI/a-singular-scientific-story-duchennes.html

Constitutional Questions Raised on Sweeping Changes at the California Stem Cell Agency

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The outside counsel to the governing board of the California stem cell agency is preparing an opinion on whether some of the major changes recommended by the blue-ribbon Institute of Medicine(IOM) study might require a vote of the people.

More than three years ago, the same issue was raised  and used by the agency to resist unwanted changes.

Kevin McCormack, agency spokesman, said today that James Harrison of Remcho Johansen & Purcell of San Leandro will perform the analysis. Harrison has been counsel to the CIRM board since its inception. He also wrote part of Proposition 71, which created the stem cell agency in 2004.

Harrison's analysis was disclosed after CIRM Director Sherry Lansing, who is also chairwoman of the University of California Board of Regents, said this morning that the board's "hands are tied" concerning some of the IOM proposals because they could require a vote of the people. Other members of the board bristled at the IOM recommendations.

In 2009, Harrison tackled a similar task in connection with related, proposed structural changes at the $3 billion stem cell research effort. In reaction to proposals by the Little Hoover Commission, the state's good government agency, Harrison said,

“The Little Hoover Commission’s proposals would effect drastic and disruptive changes to CIRM’s governance and operating systems. Such changes run counter to the voters’ intent, and do not further Proposition 71’s purposes.”

The California Stem Cell Report wrote at the time, 

"The 10-page legal memo hung most of its arguments on a provision in Prop. 71 that states that it can only be amended by the legislature if the changes 'enhance the ability of the institute to further the purposes of the grant and loan programs.'
"Harrison's memo said the Hoover proposals (in question) could only be enacted through another ballot measure...."

The Little Hoover proposals dealt with the structure of the board and the conflicting responsibilities of the president and the chairman. The IOM has recommended major changes in both areas and approvingly cited the Hoover study .

Harrison's analysis will also delineate which IOM recommendations can be implemented by board action and which will require legislative approval.

The IOM report, which cost the stem
cell agency $700,000, recommended a host of changes that critics for
years have said are needed. But the 17-month study also went beyond
what the critics had proposed. The IOM said that the 29-member
governing board should be stripped of power to approve individual grants.
Instead, the board would be limited to voting for or against a slate
of applications.
The IOM also proposed far-reaching
changes to remove conflict of interest problems, clean up a troubling
dual-executive arrangement and fundamentally change the nature of the
governing board. The recommendations would greatly strengthen the
role of the agency's president, significantly alter the role of
patient advocates on the governing board and engage the biotech
industry more vigorously.
CIRM's governing board and its first chairman, Robert Klein, an attorney who directed the writing of the stem cell initiative and wrote parts of it, have mightily resisted related proposals. In 2009, Klein even warned of lawsuits if legislative action were initiated for reforms (see here and here). 
In an editorial today, The Sacramento Bee said changes are long overdue at the agency. The Bee said CIRM has "been consumed by a siege mentality that has prevented any real introspection.”  In another editorial earlier this week,
the San Francisco Chronicle said that prompt and major changes are
needed at the agency.
Many of the more significant
recommendations clearly require either a rare, super, supermajority vote of
the legislature (70 percent) and the signature of the governor or
another ballot initiative, which is very unlikely. Achieving the 70
percent vote is exceedingly difficult except on the most
noncontroversial matters before the legislature. The requirement
permits only 13 members of the 40-member Senate to block any CIRM
legislation, giving minority viewpoints extraordinary power over the
content of any CIRM legislation.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/6UqNIAVe5JY/constitutional-questions-raised-on.html

Stem Cell Agency Chairman Says IOM Report 'Quite Complimentary'

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Jonathan Thomas
CIRM Photo

Jonathan Thomas, the Los Angeles bond
financier who is chairman of the $3 billion California stem cell agency, has
weighed in at more length on the sweeping recommendations from the
Institute of Medicine for changes at the agency.

He wrote a piece for the agency's blog
that said the 124-page report was “quite complimentary.” Thomas' article carried forward the theme of the stem cell agency's press
release yesterday that said the IOM “praises the agency as a 'bold
social innovation.'”
Thomas did acknowledge that the report
“highlighted some areas and made some recommendations about where
and how we might improve our performance.” 
Thomas concluded by saying the agency
takes the report seriously and will, over the next few months,
consider how best to respond.
Nearly needless to say, other observers
of the agency differ with Thomas' characterization of the report as
“quite complimentary.(See here and here.)

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/HmbrFtBzcs4/stem-cell-agency-chairman-says-iom.html

IOM Report: Many Major Changes at Stem Cell Agency Require Legislation

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As the Institute of Medicine began its presentation today to the governing board of the $3 billion California stem cell agency, one of its key directors, Sherry Lansing, pointed out that some of the IOM recommendations require legislative action and perhaps a vote of the people.

Her essential point was that the agency is hamstrung by the very measure that created it in 2004. She did not elaborate on her comment, but here is a look at some of matters she was talking about. (Lansing is chairwoman of the UC board of regents and former CEO of a Hollywood film studio.)

The ballot measure locked in management minutia and more, and then slapped on tight restrictions that make it nearly politically impossible to alter even such matters as who is in charge of production of the annual report. (By order of law, the chairman, in case you are wondering.)

And if the 29-member governing board -- so sized and specified to every nit and nat by the ballot measure -- wants to make the sweeping changes recommended by the IOM, agency directors face a formidable task in California's Capitol.

Many of
the most important recommendations for the California stem cell
agency require a vote of the California legislature and
signature of the governor. The IOM's choice of words – that these proposals “may” need legislation – is conservative. There is little doubt that legislative action would be needed to fully implement the recommendations below.

The vote needed is no simple majority. It is a rare, super,
super-majority (70 percent) of each house. The requirement was also written into state law by Proposition 71, the 10,000-word ballot initiative
that created the stem cell agency. The 70 percent requirement gives extraordinary power to minority viewpoints that must be dealt with to win passage of legislation. In the state Senate, for example, it would take only  13 senators to block a bill.  
With that background, here are direct quotes from the IOM report on its legislative recommendations.
Separate Operations from Oversight
The board should focus on strategic
planning, oversee financial performance and legal compliance, assess
the performance of the president and the board, and develop a plan
for transitioning CIRM to sustainability. The board should oversee
senior management but should not be involved in day-to-day
management. The chair and the board should delegate day-to-day
management responsibilities to the president. Each of the three
working groups should report to management rather than to the ICOC.
Change the Composition and Structure of
the Board and Working Groups
CIRM should put systems in place to
restructure the board to have a majority of independent members,
without increasing the size of the board. It should include
representatives of the diverse constituencies with interests in stem
cell research, but no institution or organization should be
guaranteed a seat on the board. Consideration should be given to
adding members from the business community. The terms of board
members should be staggered to balance fresh perspectives with
continuity.
The chair and other ICOC members should
be prohibited from serving on the working groups. During the
reconstitution of the working groups, the current level of
representation of disease advocates should be maintained, such board
members being replaced with other disease advocates who are not board
members.
Revise Conflict of Interest
Definitions and Policies
CIRM should revise its definitions of
conflict of interest to recognize conflicts arising from nonfinancial
interests, such as the potential for conflict arising from an
individual’s interest in a specific disease, and should reassess
its policies for managing conflict of interest in light of this
broader definition.
Restructure the Grant Review and
Funding Process
CIRM should restructure the grant
review and funding process to separate oversight and strategic
planning from day-to-day operations. The ICOC should remain
responsible for oversight and articulation of an overall strategic
plan. However, grant management, funding recommendations, and grant
administration should be the responsibility of the CIRM scientific
staff, reporting to the president. This restructuring would help
mitigate concerns related to conflicts of interest and would also put
the review and funding process in the hands of those best equipped to
make those decisions.
The committee recommends several
changes pertaining to the development and approval of RFAs,
composition of the Grants Working Group, reordering of rankings by
CIRM staff, notification of applicants, and process for making final
decisions.
Enhance Industry Representation in Key
Aspects of CIRM Organization
Industry representation on the ICOC,
the Scientific Advisory Board, the Standards Working Group, and the
Grants Working Group should be enhanced to leverage industry’s
expertise and resources in product development, manufacturing, and
regulatory approval in support of the ultimate goal of bringing
therapies to patients.
Consider Harmonizing Intellectual
Property Policies with Policies of Bayh-Dole Act
 As
other sources of funding for stem cell research become available and
as the field of regenerative medicine advances from the laboratory to
the clinic, the ICOC should reconsider whether its goal of developing
cures would be better served by harmonizing CIRM’s IP policies
wherever possible with the more familiar policies of the Bayh-Dole
Act.  

Source:
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More IOM-CIRM Coverage: One Story Notes Major 2007 Conflict Flaps at Stem Cell Agency

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Additional coverage emerged this morning,
including stories in the Los Angeles Times, the Nature web site and
Businessweek. on a blue-ribbon report that recommended sweeping changes
at the the $3 billion California stem cell agency

In the Times, California's largest
circulation newspaper, Eryn Brown's story was headlined,

"Stem cell
agency board criticized for conflicts of interest."

The article began,

"The board of California's stem cell
funding agency is rife with conflicts of interest and should be
restructured to improve the integrity of its grant-making process,
according to a new report from independent experts convened by the
national Institute of Medicine."“

In the San Diego U-T, reporter Bradley
Fikes' article was the only piece in all the coverage to mention two major conflict-of-interest flaps at the agency in 2007.
One involved then CIRM board member
John Reed, head of Sanford-Burnham in La Jolla, who tried to influence CIRM staff
in connection with a grant to his organization, triggering
an investigation by the state's political ethics commission. (Reed's
actions were first disclosed by the California Stem Cell Report.) The
other case involved inappropriate actions by four members of the
29-member board in an $85 million round. Ten applications were dumped
from the round because of the directors' actions. The conflict
issues were so rampant that only eight of the directors present at a
December 2007 meeting could discuss the issues.
(See here, here and here.)
On the Nature news blog, Monya Baker
had a thorough piece that said the agency “received a mixture of
praise and hard-to-enact recommendations from an august scientific
body.” She also wrote,

“It’s unclear what effect the
report will have. Many of these recommendations run counter to
requirements enshrined in the legislation that created CIRM, and the
board of CIRM has heard similar recommendations before and failed to
act on them.”

On the web site of the journal Science,
Greg Miller wrote that IOM report "praises the California Institute for
Regenerative Medicine (CIRM) as a 'bold social innovation' that
provided a creative new source of funding that has turned the state
into an international hub of stem cell research. But the IOM panel
authoring the report also concluded that the funding agency’s
organization and governance is not optimal."
Businessweek carried the AP story by
Alicia Chang mentioned yesterday. The AP story also appeared on the San
Francisco Chronicle and Sacramento Bee web sites and was also carried internationally on other web sites.  The Chronicle also had a staff story by Erin Allday.  
(An earlier version of this item did not contain the last sentence regarding the Allday story.)

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/yk8zuHF6IKA/more-iom-cirm-coverage-one-story-notes.html

Text of Comments Reacting to IOM Report on California Stem Cell Agency

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Here is the full text or the essential
elements of comments sought by the California Stem Cell Report on the
Institute of Medicine report that recommended sweeping changes at the
California stem cell agency. Consumer Watchdog and the Center for
Genetics and Society put their comments in the form of news releases,
which contained redundant material.

From Stuart Drown, executive director
of California's Little Hoover Commission:

“CIRM initiated the Institute of
Medicine review, which is to its credit.  The Institute of
Medicine took a scrupulous and rigorous approach to its review of the
California Institute of Regenerative Medicine and in its report,
notes CIRM’s many achievements and accomplishments.

“The Institute’s recommendations
for much-needed changes to CIRM’s governance structure to provide
greater efficiency, clarity and accountability reinforce the
recommendations the Little Hoover Commission made in 2009. The
institute graciously acknowledged the commission’s work, which
clearly is as relevant now as it was in 2009.

“Then and now, the Commission’s
recommendations are aimed at improving CIRM’s ability to meet its
goals for the good of all who can benefit from stem cell research,
and to ensure that California taxpayers’ dollars are put to their
most efficient use to that end.”

From John M. Simpson, stem cell project
director at Consumer Watchdog of Santa Monica, Ca.(full press release here):

“Consumer Watchdog Thursday welcomed
a report from the prestigious Institute of Medicine (IOM) calling for
sweeping reforms in governance at California’s stem cell agency and
an end to the board’s built-in conflicts of interest. 

“The report said that 'far too many
board members represent organizations' that receive funding or
benefit from the stem cell agency. The IOM said that the board’s
oversight function should be separated from the day-to-day management
of the California Institute for Regenerative Medicine (CIRM). 

“'The IOM's critical report echoes
what every independent evaluator has said in the past,' said John
M. Simpson, Consumer Watchdog’s Stem Cell Project director.
'As we have repeated from the beginning, CIRM suffers from built-in
conflicts of interest and needs to separate the board's oversight
function from day-to-day management.'

“'It's long past time to make the
changes the report calls for, but given the spin the agency put on
its response -- saying the report praises the 'agency as a bold
innovation' -- shows it's business as usual. This sort of behavior
will only ensure that CIRM doesn't get another round of public
funding,' Simpson said.”

From Marcy Darnovsky, associate
executive director of the Center for Genetics and Society in
Berkeley, Ca.(full press release here):

“The Center for Genetics and Society,
a nonprofit policy research and advocacy organization, welcomed the
report on the California stem cell agency released today by the
Institute of Medicine and called for stronger protections for  the interests of Californians as
the agency continues its disbursement of public funds.

“CIRM is nearing the end of the
billions of dollars of public funding allocated to it in
2004. The agency is currently considering how to extend
its operations after the money runs out. CGS Associate Executive
Director Marcy Darnovskysaid, “Given the agency’s
shortcomings and the state’s budgetary problems, it would be wrong
to ask Californians to give it more public money. If the agency
acquires new funds from industry sources or venture firms, it must
recognize that it has ongoing obligations to the people of
California.”

“She continued, 'CIRM has not
responded in a meaningful way to many previous public interest
suggestions or to independent reviews, including the one in 2008 by
the state’s Little Hoover Commission. We hope the agency will not
continue that pattern.' 

“'Today’s report from the IOM
reaffirms the significance of the conflicts of interest and
structural flaws that were built into the stem cell program from the
beginning, and that continue to threaten its credibility and
effectiveness. These are serious problems that the Center for
Genetics and Society and other public interest voices pointed out
even before the agency was approved by the 2004 ballot measure on
which backers spent some $35 million. 

“'Many aspects of these early
concerns remain directly relevant,' Darnovsky said. 'There is
still no way for elected officials to provide oversight because the
measure that created CIRM requires a 70% vote by both houses – more
than a supermajority. The agency’s governing board is still tainted
by its built-in conflicts of interest, and still includes no
representation of the public beyond disease advocates. Members of the
agency’s powerful Working Groups, including the one that reviews
grant applications, are still not required to publicly disclose their
individual financial interests.'”

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/iu4DrhoewYo/text-of-comments-reacting-to-iom-report.html

IOM Proposals for Overhaul at CIRM Win High Marks

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The Institute of Medicine's
recommendations for major changes at the California stem cell agency
today received generally high marks from independent observers and
critics.

Many of the proposals echoed
suggestions from California's Little Hoover Commission, the
state's good government agency. Asked for comment, Stuart Drown, the
commission's executive director, said,

“The institute’s recommendations
for much-needed changes to CIRM’s governance structure to provide
greater efficiency, clarity and accountability reinforce the
recommendations the Little Hoover Commission made in 2009."

He continued,

“Then and now, the Commission’s
recommendations are aimed at improving CIRM’s ability to meet its
goals for the good of all who can benefit from stem cell research,
and to ensure that California taxpayers’ dollars are put to their
most efficient use to that end.”

The California Stem Cell Report also
asked the agency's first president, Zach Hall, for his thoughts. Here
is the full text of what Hall, who was one of the peer reviewers on
the IOM study, had to say,

“The IOM Committee and its staff have
done an impressive job.  The report recognizes the scientific
value and achievements of the CIRM and, at the same time, makes
cogent recommendations that, if taken seriously, will further improve
the quality and the public credibility of the Institute. The
committee and staff deserve the thanks of the scientific community
and all California citizens for their careful and thoughtful work.” 

John M. Simpson, stem cell project
director for Consumer Watchdog of Santa Monica, Ca., said,

“It's long past time to make the
changes the report calls for, but given the spin the agency put on
its response -- saying the report praises the 'agency as a bold
innovation' -- shows it's business as usual. This sort of behavior
will only ensure that CIRM doesn't get another round of public
funding,” 

Marcy Darnovsky, associate executive
director of the Center for Genetics and Society of Berkeley, Ca.,
welcomed the recommendations. But she said,

“Given the agency’s shortcomings
and the state’s budgetary problems, it would be wrong to ask
Californians to give it more public money. If the agency acquires new
funds from industry sources or venture firms, it must recognize that
it has ongoing obligations to the people of California.” 

She continued,

 “CIRM has not
responded in a meaningful way to many previous public interest
suggestions or to independent reviews, including the one in 2009 by
the state’s Little Hoover Commission. We hope the agency will not
continue that pattern.” 

The California Stem Cell Report also
queried most of the 10 patient advocates on the agency's governing
board for comment. Their roles could be altered in a major way by the
IOM recommendations. None of the advocates have yet responded.
(The full text or nearly
full text of all the above comments is available here.)  

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/yyZ0TAisMZQ/iom-proposals-for-overhaul-at-cirm-win.html

Coverage of the IOM Report: Light but a Column with a Cutting Edge

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News coverage has been light so far
today of the Institute of Medicine's recommendations for an overhaul
at the $3 billion California stem cell agency. But a Pulitzer Prize-winning columnist from the Los Angeles Times took a sharp knife to the agency's press release on the IOM report.

Michael Hiltzik, who is a regular critic of the agency, asked,

 "So how did CIRM react to the report? Even before the review panel's conference call with the press was completed, the agency issued a news release stating that the panel had 'praised' the agency 'for its ground breaking work in helping advance the science of stem cell research.'"If you wanted to know about the committee's criticisms, the first mention of those was in paragraph 9 of the news release. It quoted board Chairman Jonathan Thomas as promising to 'work on establishing a process to enable us to consider how best to proceed with reviewing the recommendations.' 

"By my count, that's seven steps it will take before actually acting on the recommendations. 

"As it happens, the panel's recommendations, which include creating a majority of independent board members without any potential conflicts of interest, track very closely to recommendations made by several previous outside reviews of CIRM, especially a 2009 study by the state's Little Hoover Commission.

"CIRM rejected almost every one, and it looks to be preparing to circle the wagons again against sensible improvements in the way it does business."

The Associated Press story by Alicia
Chang popped up in two different forms on the Washington
Post web site and in Ottowa and Spokane, among other places. Chang
was on board for the IOM news conference and had this to say about
CIRM from one of the IOM study group members.

“'They’re not broken but they’re
bent,' said Sharon Terry, president of the nonprofit Genetic Alliance
who was part of the panel. 'They need some correction.'”

Chang's story originally began,

“California has transformed into a
powerhouse player in stem cell research, but the taxpayer-funded
institute responsible for that needs an overhaul, a report released
Thursday found.”

Another version, that appeared in
Ottowa and Spokane and beyond, started this way,
“A report says California’s stem
cell agency needs more independent oversight and recommends a
restructuring to avoid the appearance of conflict of interest.”
Ron Leuty of the San Francisco
Business, who is one of perhaps two reporters who regularly cover
the stem cell agency, wrote,

“A review of California's stem cell
research funding agency proposed changes to the agency's governing
structure and commercial goals while praising its results so far. The
124-page report from the Institute
of Medicine recycles many conflict of interest and
intellectual property concerns that have dogged the San
Francisco-based” agency.

Stephanie O'Neill at KPCC radio in Los
Angeles also had a story.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/XpN8S2N2-iw/coverage-of-iom-report-light-but-column.html

Collapse of Big Pharma Deal Involving California Stem Cell Agency

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A ballyhooed deal has blown apart that
would have hooked up – for the first time – Big Pharma and the $3
billion California stem cell agency.

The breakdown of the arrangement was
quietly disclosed yesterday in background material prepared for the
Dec. 12 meeting of the stem cell agency's governing board.
The deal was first announced Oct. 25
when Viacyte, Inc., of San Diego, received a $10.1 million award to
help finance a clinical trial for a diabetes treatment involving
Viacyte and GlaxoSmithKline.
The CIRM background memo said this week, however,

“We have recently been informed that
GSK was not able to obtain the final approval required due to
business reasons in the context of GSK's overall research and
development portfolio and investment needs and not as a result of any
scientific or technical assessment of ViaCyte's program.”

The memo gave no further details about
the Glaxo decision.
CIRM staff proposed that Viacyte, which
has received $36 million from CIRM, be given another $3 million
because Glaxo has exited the trial.
The arrangement involving Glaxo,
Viacyte and CIRM was trumpeted in October, when Viacyte was awarded
the $10 million. Officials of the stem cell agency said the award
was a “watershed” for CIRM. Jason Gardner, head of the Glaxo stem
cell unit and who attended the meeting, told the California Stem Cell
Report that the arrangement was a partnership and that the company
intended to develop a sustainable pipeline.
It was the second significant
business-connected deal that has collapsed for the $3 billion agency
within the last 13 months. In November 2011, Geron abandoned its
clinical trial for spinal injuries. CIRM had loaned Geron $25 million
for the trial just three months earlier. The company paid the money
back with interest.
CIRM staff said that advisors to the
agency remain “extremely positive” about the Viacyte research and
“strongly recommended” that the company receive the additional $3
million. The memo said that trial has a “strong potential” to be
commercialized.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/p-E0ivzGfr0/collapse-of-big-pharma-deal-involving.html

Excerpts from the IOM Report on the California Stem Cell agency

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Here are excerpts from the $700,000 Institute of Medicine (IOM) report on the $3 billion California stem cell
agency -- the California Institute of Regenerative Medicine (CIRM).

Overall Comments
“Improvements to CIRM’s governance
structure, scientific program, and policies are critical to bet­ter
serving California taxpayers who elected to devote funding to promote
stem cell research in the state. The necessary changes outlined by
the IOM committee, if enacted by the state and/or the institute,
would help to instill confidence among the scientific community and
California residents in the vital work that CIRM is accomplishing.”
“It is the committee’s judgment
that overall, CIRM has done a very good job of initially establishing
and then updating the strategic plans that have set priorities for
and guided its programs, and of taking advantage of its guaranteed
flow of $300 million a year for 10 years to establish a sustainable
position in regenerative medicine for California. 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.
“Despite its demonstrable
achievements to date, as well as the largely positive independent
reports covering various aspects of its operations, no one would
claim that CIRM is a perfect organization or that it should adhere
slavishly to its initial form of organization, set of regulations, or
pattern of priorities. The field of regenerative medicine has
advanced rapidly since November 2004, and CIRM itself has seen the
need to alter its activities and approaches in some areas. The
committee believes the same should be true of its governance
structure, some of its administrative practices, and its use of
external perspectives on strategic scientific priorities and on the
evaluation of other key policies, such as intellectual property, to
ensure that they continue to encourage the development and deployment
of new treatments.”
“While the restrictions on amending
the administrative structure of CIRM established in Proposition 71
had the advantage of protecting the institute’s ongoing operations
from outside interference in an ethically controversial arena, they
also made it difficult to modify the organization’s structure in
response to experience and/or changing circumstances. Moreover, these
protections, whatever their benefits, appear to some to shield CIRM
from the normal accountability mechanisms in place for state
agencies.”
Conflicts of Interest
“Far too many board mem­bers
represent organizations that receive CIRM funding or benefit from
that funding. These com­peting personal and professional
interests com­promise the perceived independence of the ICOC,
introduce potential bias into the board’s decision making, and
threaten to undermine confidence in the board. Neither the board
chair nor board members should serve on any working group. The board
itself should include representatives of the diverse constituencies
that have an interest in stem cell research, but no institution or
organiza­tion should be guaranteed a seat.”
“The problematic perception of
conflicts of interest has persisted for as long as CIRM has existed.
The IOM committee would be less concerned about individual board
members with actual or perceived conflicts of interest if the board
membership included more truly independent members. The majority of
board members should be independent, with no competing or conflicting
personal or professional interest. Broader representation from a
wider variety of stakeholders will inject new perspectives into the
panel and will help to dispel the perception of conflicts of
interest.
“CIRM also should revise its conflict
of interest definitions to include non-financial interests, such as
the potential for personal conflicts of interest to arise from one’s
own affliction with a disease or personal advocacy on behalf of that
disease. CIRM policies for managing conflicts of interest should
apply to that broader definition.”
Structure and Governance
“Currently, the ICOC (the agency's
governing board) functions both as an executor and as an
overseer—competing duties that compromise the ICOC’s critical
role of pro­viding independent oversight and strategic
direc­tion. The IOM committee recommends that CIRM’s operations
be separated from its over­sight. The board should delegate more
author­ity and responsibility for day-to-day affairs to the
president and senior management, and the ICOC’s three working
groups should report to senior management within CIRM, rather than to
the ICOC. The moves would permit the board to better focus its energy
and collective talent on strategic planning, overseeing financial
perfor­mance, ensuring legal compliance, assessing the
president’s performance, and devising a plan for preserving and
expanding its considerable assets to permit the institute to continue
its important work after the bond measures end.”
Unrealistic Goals
“While the latest round of awards
challenge teams to have filed a request to begin clinical trials or
to have completed early-stage trials in patients within four years,
the committee feels these ambi­tious goals are unrealistic. New
therapies take more time to progress to federal approval, and
early-stage clinical trials are beset by a stagger­ingly high
failure rate. Rather than judging suc­cess by simply tallying the
number of active clini­cal trials, the IOM committee suggests
that CIRM also continue its focus on underlying biological mechanisms
that drive the success or failure of a promising therapy and on
careful design of clini­cal trials. Advances in these areas will
help the entire field progress, even if a specific drug candi­date
is not approved." 
Economic Impact
“In the short term, CIRM’s
expenditures are supporting approximately 3,400 jobs and their
innovative efforts have also attracted substantial additional private
and institutional resources to this research arena in California
CIRM’s long-term impact on such critical aspects of the California
economy as state tax revenues and health care costs beyond the
shorter-term and temporary impact of its direct expenditures cannot
be reliably estimated at this point in CIRM’s history.... (T)he
estimate of the Analysis Group (2008) that the CIRM program alone
would support about 3,400 jobs as long as it was allocating about
$300 million per year in research and development grants appears
quite reasonable to the committee. To put this estimate in context,
however, total employment in California is roughly 16 million, and
NIH alone provides more than $3.5 billion per year to California
research institutions.”
Intellectual Property
“CIRM should propose regulations that
specify who will have the power and authority to assert and enforce
in the future rights retained by the state in CIRM-funded
intellectual property. CIRM should seek to clarify which state
agencies and actors will be responsible for the exercise of
discretion currently allocated to CIRM and the ICOC (the CIRM
governing board) over future determinations on issues regarding
march-in rights, access plans, and revenue-sharing rights that might
arise years after CIRM's initial funding period has passed.... (T)he
ICOC should reconsider whether its goal of developing cures would be
better served by harmonizing CIRM’s IP policies wherever possible
with the more familiar policies of the Bayh-Dole Act(federal IP law).

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/Td6TGOVGYNo/excerpts-from-iom-report-on-california.html

Text of CIRM Chairman's Comments on the IOM Report

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Here is the text of comments on the IOM study of CIRM from J.T. Thomas, chairman of the agency. 

"We deeply appreciate all the hard work of the IOM committee in compiling  long and detailed report and the IOM clearly put considerable thought into compiling it. This has just been released so our Board and our staff has not had a chance to look at it yet, let alone digest its findings and recommendations, so it’s premature for us to offer any opinions. We are looking forward to the IOM presentation at the next meeting of our board, the Independent Citizens Oversight Committee (ICOC) where we’ll have a chance to talk with the IOM directly about the report. After that we’ll put together a process on how best to proceed so that we can respond in as thoughtful a manner to the recommendations as the IOM did in making them."

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/YkRTcgnXNqU/text-of-cirm-comments-on-iom-report.html

$80 Million in Grants, Money for Viacyte and Blue-Ribbon Report on California Stem Cell Agency

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Directors of the California stem cell
agency are expected to give away $80 million next week to 20
fortunate researchers in addition to exploring a “commercialization
and industry engagement plan.”

The subjects are on tap for the Dec. 12 meeting in Los Angeles of the governing board of the $3 billion
research effort.
The $80 million grant round is aimed at
“career development of physician scientists working in
translational stem cell research.”

“This award will fund promising
physician scientists in the critical early stages of their careers as
independent investigators and faculty members establishing their own
laboratories and programs.” 

Summaries of the grant reviewers
comments and application scores should be available sometime this
week. The bare-bones agenda lacked elaboration on the
commercialization plan.
Directors are additionally scheduled to
hear a presentation on the blue-ribbon report by the Institute of
Medicine for which the agency is paying $700,000. The report has been
17 months in the making and is scheduled to be released this
Thursday.
Other interesting matters are on the
table, although the agency has yet to produce background material
laying out any details. The subjects include:
  • More money – no amount yet specified
    – for Viacyte, Inc, of San Diego, which has received more than $36
    million from CIRM.
  • An update of the agency's response to
    the only performance audit conducted at the agency. The audit
    identified 27 areas where improvement is needed, but the governing
    board has not discussed the results publicly since they were
    disclosed last May.
  • Approval of the concept plan for
    another round of basic biology grants and adoption of conflict of
    interest code changes.

Interested parties will be able to take part at the meeting location in Los Angeles and teleconference locations in La Jolla, Oakland and UCSF. If you are interested in
the teleconference locations, you will need to contact the agency for more specific directions than are provided on the agenda.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/RU_xC86oAyw/80-million-in-grants-money-for-viacyte.html

Extra, Extra! CIRM Staffers Blog the World Stem Cell Summit

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Years ago, I worked with an editor who
used to advise his lagging scribes to put their noses in their
typewriters and peck.

Well, the folks at the California stem cell agency have their
noses in what passes today for typewriters and are pecking away
furiously. Their subject is the World Stem Cell Summit, which has
received only slight coverage in the mainstream media.
Today, the stem “cellists” from San
Francisco's King Street filed -- on the agency's blog -- three fulsome
items on doings at the summit, which is taking place in West Palm
Beach, Fla. Yesterday they filed four. Photos and charts were
included. More coverage is expected tomorrow.
CIRM staffers blogging the World Stem Cell Summit
covered UC Davis researcher Paul Knoepfler discussing
patient advocacy and its role in funding stem cell research. 

The CIRM writers are doing double-duty
in at least one case. Geoff Lomax, the agency's senior officer for
its standards group, is additionally speaking on a panel at the session. A
handful of other CIRM officials are also appearing at the conference,
which ends tomorrow.

The primary purpose, we presume, of
sending state employees across the country is to gather the latest
information on stem cell science and issues and to make contacts. It
is a bit of a bonus for the public to have the CIRM attendees also
file stories on the sessions.
A couple of the items caught my
attention. One dealt with patient advocates and their role in
energizing and helping to drive funding for research. Another item
discussed what appear to be growing issues with dubious stem cell treatments and the damage they can do to the field in general.
Lomax summarized the signs of a stem
cell scam like this:
  • “Claims of miracle cures for
    diseases
  • “Single treatments or cells that
    can treat any type of disease
  • “Lack of objective information,
    evidence (such as published medical reports) that a treatment is
    effective
  • “Treatment by a doctor who is
    not trained or certified to treat the specific disease
  • “No system exists to collect
    information and follow up with patients”

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/usBXfuFwqy4/extra-extra-cirm-staffers-blog-world.html

BioTime Makes Bid for Geron's Stem Cell Assets

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Biotime, Inc., and two men who were
leading players in history of Geron Corp. today made a surprise,
public bid for the stem cell assets of their former firm.

Michael West
West photo
Tom Okarma
AP file photo
The men are Michael West and Thomas
Okarma. West founded Geron in 1990 and was its first CEO. West is
now CEO of Biotime. Okarma was CEO of Geron from 1999 to 2011.
Okarma joined Biotime on Sept. 28 to lead its acquistion efforts.
Both Geron, based in Menlo Park, Ca., and Biotime, based in Alameda,
Ca., are publicly traded.
West and Okarma sent an open letter this morning to Geron shareholders and issued a press release making
a pitch for the Geron's stem cell assets. Geron jettisoned its hESC
program nearly a year ago and closed its clinical trial program for
spinal injuries. The move shocked the California stem cell agency,
which just a few months earlier had signed an agreement to loan the
firm $25 million to help fund the clinical trial. The portion of the
loan that was distributed was repaid with interest.
At the time, Geron said it would try to
sell off the hESC program, but no buyers have surfaced publicly.
Personnel in the program have been laid off or found employment
elsewhere.
The West-Okarma letter to shareholders
said that under the deal,

“Geron would transfer its stem cell
assets to BAC(a new subsidiary of Biotime headed by Okarma), in
exchange for which you along with the other Geron shareholders would
receive shares of BAC common stock representing approximately 21.4%
of the outstanding BAC capital stock. BioTime would contribute to BAC
the following assets in exchange for the balance of outstanding BAC
capital stock:

  • “$40 million in BioTime common
    shares;
  • “Warrants to purchase BioTime
    common shares (“BioTime Warrants”);
  • “Rights to certain stem cell
    assets of BioTime, and shares of two BioTime subsidiaries engaged in
    the development of therapeutic products from stem cells.”
The letter asked Geron shareholders to
write the firm's board of directors to urge them to approve the
offer.
Geron had no immediate response to the
proposal. Asked for comment, Kevin McCormack, spokesman for the
California stem cell agency, said the deal “had nothing to do with
us.” However, in the past, CIRM has indicated that it could find a
way to transfer the loan to an entity that would continue spinal
injury clinical trial. CIRM President Alan Trounson was also involved
at one point in trying to assist in a deal.
Geron's shares rose 12 cents to $1.54
today while Biotime's shares lost four cents to $3.95.
Here are links to the two news stories
that have appeared so far on the proposed deal: Associated Press, Marketwatch.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/v1bas6eGZF0/biotime-makes-bid-for-gerons-stem-cell.html

Update on Move To Curb Researcher Appeals at California Stem Cell Agency

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Directors of the $3 billion California
stem cell agency are still mulling details of changes in their
free-wheeling and sometimes emotional appeals process for grant applications
that are rejected by the agency's reviewers.

A special task force of directors met last week
for the second time to discuss the likely alterations. Kevin McCormack, spokesman for the agency, said
the group made no decisions. Another meeting will be held later at a
date to be determined. The task force's recommendations will then go
before the full board, probably in late January.
McCormack said members of the panel
have asked for “more details regarding the process that would be
employed if the appeals and extraordinary petition processes were
merged.”
The agency has an odd, bifurcated
appeals process. Early in its existence, the agency said appeals of
reviewer decisions could be based only on conflicts of interest.
However, researchers have a right under state law to speak to the
governing board in public on any issue whatsoever. As some
researchers began to use that avenue to ask for reconsideration of
their applications, the CIRM board created what it called
“extraordinary petitions” in an effort to control the process and
limit appeals. Both the “appeals” and “extraordinary petitions”
are, in fact, appeals but on different grounds and employing different
mechanisms.
The task force was created in September
after directors complained about “arm-twisting” and “emotionally charged presentations” in connection with a record number of
appeals earlier this year.
Here is a link to an item about the task force's first meeting. Here is a link to an agency summary of the task force's deliberations prior to last week's meeting. The
transcript of the session should be available on the CIRM web site
within the next two weeks. It will be found under the meetings
section of the web and then under the heading for the task force's
November session.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/Hkq7zcXyMsQ/update-on-move-to-curb-researcher.html

Diane Winokur, Veteran ALS Patient Advocate, Named to California Stem Cell Agency Board

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Longtime ALS patient advocate Diane
Winokur of San Francisco, who has lost two sons to the disease, has
been appointed to the governing board of the $3 billion California
stem cell agency.

She fills the vacancy left by David
Serrano Sewell, who resigned from the 29-member panel earlier this
year after serving since the agency's inception. CIRM has 10 patient advocates
on its board.
Diane Winokur
Photo -- Legal Momentum
Winokur is well-known in ALS circles.
She sat for five years on the governing board of the national ALS
advocacy group and currently serves on the board for the California
state group, the Golden West chapter. She also served for six years on the board of
trustees for the Sanford- Burnham Institute in La Jolla, which has received $37 million from the stem cell agency. She left Sanford in 2011.
Last summer Winokur appeared before the
CIRM board to successfully seek approval of an $18 million ALS grant
that was rejected by the agency's reviewers. Researcher Clive
Svendsen of Cedars-Sinai in Los Angeles appealed the denial to the
full board and was supported in emotional testimony by other patient
advocates as well, including persons with the affliction.(See here for video of some of the testimony.)
The agency has awarded about $30.6
million, including the Svendsen grant, for research directly related
to ALS.
Golden
West issued a press release Nov. 21, lauding the Winokur appointment
by California Lt. Gov. Gavin Newsom. The release quoted Lucie Bruijn,
chief scientist of The ALS Association, as saying,

"Her
contributions have been invaluable and she will be a tremendous asset
in moving the ALS research field forward through CIRM
funding."

The stem cell agency posted a blog item
on Winokur's appointment in addition to a press release. CIRM
Chairman J.T. Thomas said,

“Her knowledge, expertise and
leadership will be a tremendous addition to the ICOC (the stem cell
agency governing board) and help guide us in our work.”

Patient advocate Don Reed of Fremont,
Ca., described Winokur in a 2008 blog item as “small, elegant, full
of energy, an exclamation point of a person.”

(Editor's note: Based on information from CIRM and Golden West, an earlier version of this item incorrectly stated that Winokur is a current member of the Sanford-Burnham board.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/dITcyP7WJbs/diane-winokur-veteran-als-patient.html

Stem Cell Board Members Lubin and Sheehy Honored

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A couple of members of the governing
board of the $3 billion California stem cell agency were honored for
their work this month.

Jeff Sheehy
UCSF Photo
One is Jeff Sheehy, a UCSF
communications manager and nationally known HIV/AIDS advocate, who
has served on the CIRM board since its inception. He was named by POZ
magazine as one of the top 100 “soldiers” in the fight against
HIV/AIDS.
CIRM's Amy Adams filed an item on
Sheehy on the agency's blog yesterday. She said, 

“Jeff once told me
that when he joined CIRM’s board eight years ago, he didn’t see a
role for stem cells in an HIV/AIDS treatment. Now, CIRM has committed
$40 million toward HIV/AIDS projects and two teams of researchers
from City
of Hope and UCLA are
working toward clinical trials.”

Sheehy was also invited to the White
House to commemorate World AIDs day Dec. 1.
Bert Lubin
Childrens Hospital Photo
Also honored was Bert Lubin, CEO of
Childrens Hospital in Oakland, where he has worked since 1973, a
remarkable achievement in today's world of transitory employment. The
San Francisco Business Times named Lubin as the “most admiredCEO” in the San Francisco Bay Area. The newspaper said that since
he took charge at Childrens in 2009,

“He recruited a new senior management
team, chopped away at the pediatric hospital’s operating deficit
and worked to heal relationships with the local community and
governmental and political leaders that were deeply frayed...”

On a personal note, a friend who has
long volunteered at Childrens gives him high marks as well, citing
several encounters where he exceeded the usual CEO effort.

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/9b9s3mW2ebM/stem-cell-board-members-lubin-and.html

California Stem Cell Agency Boosting Disease Team Program to $543 Million

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Directors of the California stem cell
agency are set to give away $20 million next Thursday and authorize
a handsome addition to their signature disease team effort, bringing
its total to $543 million.

It is all part of the $3 billion
agency's push to develop therapies prior to running out of money for
new grants in 2017.
The $20 million is expected to go to
the first two winners in the agency's new strategic partnership
program. CIRM says the effort is aimed at
creating “incentives and processes that will: (i) enhance the
likelihood that CIRM funded projects will obtain funding for Phase
III clinical trials (e.g. follow-on financing), (ii) provide a source
of co-funding in the earlier stages of clinical development, and
(iii) enable CIRM funded projects to access expertise within
pharmaceutical and large biotechnology partners in the areas of
discovery, preclinical, regulatory, clinical trial design and
manufacturing process development.”
CIRM reviewed six applications with two winning approval. The agency's governing board is expected to ratify the decision next week. None of the applicants have been identified by the agency, which routinely withholds that information prior to
board action even when applicants have identified themselves.
Addition of a new $100 million
disease team round will come on top of the second, $213 million disease
team awards approved last this summer. The first round, awarded in
2009, totaled $230 million.  The size of the new round could be altered by CIRM directors prior to approval. Also before the board is a $40 million
proposal to expand the industry-friendly strategic partnership effort
into a second round.
The thrust of the disease team effort
is to speed the process of establishing clinical trials and to finance
efforts that might founder in what the biotech industry calls a
valley of death – a high risk financial location, so to speak,
where conventional financiers fear to tread.
The new disease team round will require
“co-funding” from applicants but the agency did not specify what
it means by the term. The matter of matching funds has become an issue in awards to StemCells, Inc., of Newark, Ca., in this summer's
disease team round.
Next week's agenda additionally
contains a plan to tighten review of proposed research budgets in
grant applications, making it clear that CIRM staff will be
negotiating such matters even after the board approves grants and
loans.
So far no researchers have testified in
public on the budget plan although it could well have a significant
impact on their future efforts.
Additional matters will discussed as
well at the meeting in Burlingame, which also has a teleconference
location in La Jolla that will be open to the public. The address
and additional material can be found on the agenda.  

Source:
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/1gFmBSDEYCU/california-stem-cell-agency-boosting.html


  1. Stem Cells