Geron, BioTime Deal Moves Forward with Letter of Intent
By Dr. Matthew Watson
Geron Corp., which once pioneered human
embryonic stem cell research, is close to selling off its hESC business in a complicated deal involving two former CEOs of the company and
BioTime, Inc., of Alameda, Ca.
announced a “letter of intent” involving a transaction in which
BioTime would acquire the assets of Geron's hESC clinical trial that
the company suddenly abandoned last year. The firm also laid off 66 people,
about 40 percent of its staff.
few months after the $3 billion California stem cell agency loaned Geron $25 million to assist in the trial. The agency could
restore the loan for the trial, but the Geron-BioTime announcement
did not mention that possibility. The California Stem Cell Report has
asked the agency for comment.
one day after Geron announced that it was giving up the hESC spinal injury trial because of financial reasons. The Menlo Park, Ca., firm
has been trying to sell its hESC assets since then. BioTime has
been the only firm to express public interest. The Geron trial was
the first hESC trial approved by the FDA.
West, who founded Geron and is now head of Biotime, and Tom Okarma,
who was CEO of Geron from 1999 to 2011. Okarma is now head of BioTime
Acquisition Corp.,(BAC) a subsidiary of BioTime.
described the deal in which BioTime would acquire Geron's
“intellectual property and other assets related to Geron’s
discontinued human embryonic stem cell programs.”
“ BioTime would contribute
to BAC $5 million in cash, $30 million of BioTime common
shares, warrants to purchase eight (8) million common shares
of BioTime at a pre-specified price, rights to use certain
human embryonic stem cell lines, and minority stakes in two of
BioTime’s subsidiaries. In addition, a private investor would
invest $5 million in cash in BAC.
“Following consummation of the
potential transaction, Geron stockholders would receive
shares representing 21.4% of the common stock of BAC as well as
warrants to purchase 8 million shares of BioTime common
stock at a pre-specified price. BioTime would own
approximately 71.6%, and a private investor would own approximately
7.0% of the outstanding BAC common stock for their $5
million investment. BioTime would also receive
warrants that would enable it to increase its ownership in BAC by
approximately 2%, which would reduce the Geron stockholders’
ownership in BAC to 19.2%. BAC would also be committed to pay
to Geron royalties on the sale of products that are
commercialized in reliance upon Geron patents acquired by
BAC.”
intent, an article earlier this week by Vickie Brower in The Scientist said,
“The offer couldn’t come at a
better time for Geron, which in recent months has started to feel
pressure from its shareholders to boost its stock price and move
products through the pipeline. Since last November, when the company
announced its decision to shutter its hESC and regenerative medicine
business and funnel its resources into developing telomerase-related
treatments for cancer, the stock price has dropped more than 50
percent to $1.30 a share. Geron claimed the move was simply to save
money, but many took the decision—which effectively terminated a
clinical trial of an hESC treatment for spinal cord injury—as a
setback for the entire field."
News coverage of yesterday's
announcement was light. Here is a link to a piece by Ryan McBride on
Fierce Biotech.
yesterday and rose to $1.24 in after hours trading. BioTime closed at
$2.97. No after hours trading was reported for BioTime.
Source:
Stem Cell Therapy
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November 18th, 2012
BioTime Will Have to Compete for California Cash for Geron’s Dormant Clinical Trial
By Dr. Matthew Watson
The California stem cell agency said
today it does not plan to reactivate the $25 million loan to assist
in Geron's spinal injury clinical trial despite an impending deal that would turn the effort over to BioTime, Inc.
public communications for the agency, said BioTime will have to
compete in an upcoming award round if it wants to win California
dollars.
California Stem Cell Report, McCormack said,
“That (earlier) loan was specific to
Geron and when the trial was ended the loan ended too. Of course if
Biotime and Geron do complete their deal then Biotime would be free
to apply to us for a new disease team grant.”
could also compete in other appropriate rounds, including the
strategic partnership round just posted by CIRM. It provides for four
awards of up to $15 million. Funding could come as early as October
of next year. The strategic partnership round is a business-friendly
effort that is aimed at attracting “industry engagement and
investment.” The deadline for letters of intent is Dec. 18.
million loan to Geron in 2011 just a few months before the Menlo Park
firm abandoned its human embryonic stem cell trial for financial
reasons. (The full text of the loan agreement can be found here.) The company has repaid the loan with interest.
assets associated with the clinical trial since last November. The only public
interest that has surfaced has come from BioTime, Inc., of Alameda,
Ca. Michael West, founder of Geron, is the CEO of BioTime. Tom
Okarma, CEO of Geron from 1999 to 2011, is CEO of the BioTime
subsidiary that would assume the clinical trial.
be published soon, according to a story in the San Francisco Business
Times by Ron Leuty. He quoted CIRM President Alan Trounson as saying
that “some findings” from the trial would be published next month
in a medical journal.
time of this writing today, up from $1.21 yesterday. BioTime's stock
stood at $2.99, up from $2.97.
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/DS-6n2DoRy0/biotime-will-have-to-compete-for.html
Stem Cell Therapy
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November 18th, 2012
Study of California Stem Agency Likely to be Released in About a Month
By Dr. Matthew Watson
The $700,000, Institute of Medicine performance study of the $3 billion California stem cell agency is expected to be
released in late November or early December, the IOM said today.
from the California Stem Cell Report, Christine Stencel, senior media
relations officer for the IOM in Washington, D.C., briefly discussed
the release plans and the impact of the East Coast super-storm.
“The DC area escaped the worst of
Sandy’s thumping but nonetheless our schedules and planning have
been somewhat thrown off as we’re playing catch up after two days
of being shut down and some of our committee members and reviewers
are in the areas that got the brunt of the storm. We’re not sure
whether the storm will cause any delays in peer review, but we’re
working toward the goal of publicly releasing the report in late
November or early December. The study staff is working with committee
members to determine the best release format but I anticipate there
will be a press briefing. I’ll send a media advisory when we’ve
got all the details worked out.”
report, which is examining the performance of the agency. The IOM
began its work in the summer of 2011.
Source:Stem Cell Therapy
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November 4th, 2012
Geron Weighs Biotime Bid for hESC Biz
By Dr. Matthew Watson
today it is assessing an offer by two of its former executives to buy
the human embryonic stem cell program that it abandoned nearly a year
ago.
including the $3 billion California stem cell agency, when it
jettisoned the first clinical trial of an hESC therapy for financial
reasons. The agency had loaned the company $25 million just a few
months earlier. Geron repaid the loan with interest.
by Michael West, who founded Geron in 1990. Tom Okarma, president of
Geron from 1999 to 2011, is involved with West on the deal and is now
working at Biotime.
conference call on its third quarter earnings. A spokesman said the
company is working with Biotime to “assess the feasibility” of
the proposal. He said the proposed transaction is complex and the
company is seeking “additional important details.”
additional comments on the Biotime proposal when questioned following
his initial statement.
Source:Biotime-Geron Deal Attracts Interest from Brit Investor
By Dr. Matthew Watson
A British investment trust that has
invested in Geron says it is going to take an advantage of an offer
by an Alameda firm that is seeking to acquire Geron's human embryonic
stem cell assets.
of British & American Investment Trust PLC, said last week in a letter to its
shareholders that it is disappointed in Geron's performance and the abandonment of its hESC program last November. The sudden halt to the
program and its historic clinical trial also surprised the California
stem cell agency, which had loaned Geron $25 million just a few
months earlier. The agency has expressed an interest in continuing the trial.
“We have been highly critical of
Geron management's decisions and strategy over the past 20 months, in
particular the decision in November 2011 to abruptly exit Geron's
regenerative medicine (stem cell) business in which it was the
acknowledged world leader. Since that time, Geron management has
attempted to sell or partner this business but to date has been
unable to announce any progress on this.”
Geron shareholder by Morningstar, but Woolf said 17 percent of his firm's
investments are in the Menlo Park, Ca., company. The
specific size of the trust's holdings in Geron was not immediately
known.
Inc., of Alameda, Ca., as a way for Geron shareholders to benefit. On
Oct. 18, Biotime proposed a complicated deal in which it would
acquire Geron's hESC program. Biotime's president, Michael West,
founded Geron in 1990. The head of the Biotime subsidiary that would acquire
the Geron assets is Tom Okarma, who was CEO of Geron from 1999 to 2011. (Here are links to brief stories on the offer: Fiercebiotech, New Scientist.)
declining stock performance. He said he is “seriously concerned”
that Geron has failed to find a buyer for the assets. Woolf said,
“These now dormant and untended
assets are inevitably losing value as competitors make progress in
Geron's absence from the field and patent protection periods
decline.”
“We believe BioTime's proposals would
make Geron's stem cell assets in combination with those of BioTime
once again the world's leading stem cell business with sufficient
resources to recommence the discontinued programmes and develop the
business further into the medium term.”
Geron shareholders to work with Biotime to complete the deal. Geron
has not commented on the offer.
Source:Stem Cell Therapy
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November 4th, 2012
California Stem Cell Agency First: Big Pharma Hook Up
By Dr. Matthew Watson
BURLINGAME, Ca. – For the first
time, a Big Pharma company has hooked into the $3 billion California
stem cell agency, a move that the agency described as a “watershed”
in its efforts to commercialize stem cell research.
comes via a partnership with ViaCyte, Inc., of San Diego, Ca., in a
clinical trial, partially financed with a $10.1 million grant today
from the stem cell agency. The trial involves a human embryonic stem
cell product that has “the potential to essentially cure patients
with type 1 diabetes and provide a powerful new treatment for those
with type 2 disease,” ViaCyte said. Scientific reviewers for the agency, formally known as the California Institute for Regenerative Medicine(CIRM), “characterized the goal of the proposed therapy as as the 'holy grail' of diabetes treatments.”
vice chair of the agency's grant review group, said the ViaCyte product
could be manufactured on a large scale and basically involves “taking
(small) pouches and popping them into patients.”
arrangements between ViaCyte and Glaxo that will bring in financial
and other support from Glaxo. The exact amount of cash was not
disclosed. CIRM said Glaxo will “co-fund and, assuming success,
conduct the pivotal trial and commercialize the product.” Under the terms of the grant, Glaxo and ViaCyte will have to meet CIRM milestones in order to secure continued funding.
Gardner, head of the Glaxo stem cell unit, characterized the
arrangement as a partnership. He told the board that the company
intends to develop a “sustainable pipeline.”
Trounson with being instrumental in helping to put the arrangement
together, beginning with their first meeting three years ago.
Trounson said the deal will resonate not only in California but
throughout the world.
also addressed the board, stressing the importance of CIRM's
financial support for his company over past years. It has received
$26.3 million (not including the latest grant) from California taxpayers at a time when stem cell
funding was nearly dried up. He noted that small companies such as ViaCyte do not have the resources to carry a product through the
final stages of clinical trials and subsequent production. Gardner also said,
“When the commercial funding avenues
have become much more risk averse, CIRM support (has ensured) that
promising, innovative cell therapy technologies are fully explored.”
Report, Elona Baum, CIRM's general counsel and vice president for
business development, described the award as a “watershed” for
the eight-year-old agency, linking the agency with Big Phama for the
first time. Much of CIRM's current efforts are aimed at stimulating
financial commitments from large companies, which are necessary to
commercialize stem cell research.
small companies are not unusual and can vanish quickly. However, the
CIRM-ViaCyte-Glaxo deal sends a message to other Big Pharma companies
and smaller ones, perhaps clearing away concerns that have hindered
other deals that could involve the stem cell agency.
fulfill the promises of the 2004 ballot campaign that created CIRM.
Voters were led to believe that stem cell cures were virtually around
the corner. None have been developed to date.
Source:Stem Cell Therapy
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October 28th, 2012
Trounson Going Halftime in January and February
By Dr. Matthew Watson
BURLINGAME, Ca. -- The president of the $3 billion California stem cell agency, Alan Trounson, will be working half-time while living in Australia during January and February of next year.
Trounson told the governing board of the agency of his plans at the beginning of its meeting here morning. He said he needs to spend more time with his family, which lives in Melbourne.
Trounson has an 11-year-old son with whom Trounson said he hasn't spend much time in the last 18 months. Trounson said he intends to teach his son to surf. Trounson's daughter also will be getting married in February.
Meanwhile, directors are currently discussing approval of grants in its $20 million-plus strategic partnership round.
Source:Stem Cell Therapy
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October 28th, 2012
Texas Science Flap Cited as California Stem Cell Agency Eyes its Own Processes
By Dr. Matthew Watson
OAKLAND, Ca. – Meeting against a
backdrop from Texas that involves conflicts of interest and mass
resignations of grant reviewers, a task force of the $3 billion
California stem cell agency today began a partial examination of its
own grant approval process, specifically focusing on appeals by
rejected applicants.
organization, Alan Trounson, told the task force that it was dealing
with a “very serious matter” that in some ways is similar to what
happened in Texas. He said the science community is “very much
concerned.”
five-year-old Cancer Prevention and Research Institute, which like
the California stem cell agency, formally known as the California
Institute of Regenerative Medicine (CIRM), has $3 billion of borrowed
money to use to finance research.
Texas organization, Nobel laureate Alfred Gilman, resigned Oct. 12
during a flap about its attempts “to simultaneously support basic
research and nurture companies.”
$20 million award made without scientific review. Reviewer
resignations followed with letters that accused the Texas group of
“hucksterism” and dishonoring the peer review process. (Writer Monya Baker has a good overview today in Nature.)
AFTER the governing board of the California research group created
its task force. The problems in Texas are bigger and not identical to
those in California, which mainly involve the free-wheeling nature of the appeal process, not an entire lack of scientific review.
Nonetheless, this past summer, directors of the California agency for
the first time approved an award that was rejected twice by
reviewers. The award went to StemCells, Inc., of Newark, Ca., which
now has won $40 million, ranking the company No. 1 in
awards to business from CIRM.
business columnist Michael Hiltzik characterized the StemCells, Inc.,
award as “redolent of cronyism.”
focused primarily on an aspect of the agency's appeals process that
CIRM labels as “extraordinary petitions.” They are letters which
rejected applicants use to challenge decisions by grant reviewers.
The researchers follow up with public appearances before the
governing board, often trailing squads of patients making emotional
appeals.
right under state law to appear before the CIRM board to discuss any
matter. CIRM, however, is trying to come up with changes in the
appeal process that will make it clear to researchers on what the
grounds the board might overturn reviewers' decisions. The agency is
also defining those grounds narrowly and aiming at eliminating
appeals based on differences in scientific opinion.
Sheehy, a patient advocate and co-vice chair of the grants review
group, said peer review is an “extraordinary way of analyzing
science, but it is not always perfect.” However, he also said that
“as a board we are not respecting input” from scientists and thus
allow the perception that we can be “persuaded against the judgment
of scientists.”
of the Reeve-Irvine Research Center at UC Irvine, agreed with a
suggestion by Sheehy that board must act with “discipline” when
faced with appeals by rejected applicants. Steward said,
“The
process has gotten a little out of hand.”
at today's 90-minute meeting.
teleconference locations in San Francisco, Irvine, La Jolla and Palo
Alto, were any of the hundreds of California scientists whose
livelihoods are likely to be affected by changes in the grant
approval process. Also absent were California biotech businesses,
along with the only representative on the task force from CIRM's
scientific reviewers.
businesses that have millions at stake fail to show up for key
sessions that set the terms on how they can get the money, it is a
sad commentary on their professional and business acumen.
chairman of the task force, indicated he would like to have two more
meetings of the task force prior to making recommendations to a full
board workshop in January with possible final action later that
month. Lubin, CEO of Children's Hospital in Oakland, said the matter
is “really important for the credibility of our whole
organization.”
Source:Stem Cell Therapy
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October 28th, 2012
BioTime Makes Bid for Geron’s Stem Cell Assets
By Dr. Matthew Watson
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 |
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.
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.
sell off the hESC program, but no buyers have surfaced publicly.
Personnel in the program have been laid off or found employment
elsewhere.
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.”
write the firm's board of directors to urge them to approve the
offer.
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.
today while Biotime's shares lost four cents to $3.95.
that have appeared so far on the proposed deal: Associated Press, Marketwatch.
Source:Stem Cell Therapy
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October 21st, 2012
Los Angeles Times: StemCells, Inc., Award ‘Redolent of Cronyism’
By Dr. Matthew Watson
The Los Angeles Times this
morning carried a column about the “charmed relationship” between
StemCells, Inc., its “powerful friends” and the $3 billion
California stem cell agency.
Pulitzer prize winner and author Michael Hiltzik, who has been
critical of the agency in the past. The piece was the first in the major
mainstream media about a $20 million award to StemCells, Inc., that was approved in September by the agency's board. The bottom line of the
article? The award was “redolent of cronyism.”
StemCells, Inc., now ranks as the leading corporate recipient of cash
from the agency with $40 million approved during the last few months.
on September's $20 million award, which was approved despite being
rejected twice by grant reviewers – “a particularly
impressive” performance, according to Hiltzik. It was the first
time that the board has approved an award that was rejected twice by
reviewers.
“What was the company's
secret? StemCells says it's addressing 'a serious unmet medical need'
in Alzheimer's research. But it doesn't hurt that the company also
had powerful friends going to bat for it, including two guys who were
instrumental in getting CIRM off the ground in the first place.”
who led the ballot campaign that created the agency and became its
first chairman, and Irv Weissman of Stanford, who co-founded
StemCells, Inc., and sits on its board. Weissman, an internationally
known stem cell researcher, also was an important supporter of the
campaign, raising millions of dollars and appearing in TV ads. Klein,
who left the agency last year, appeared twice before the CIRM board
this summer to lobby his former colleagues on behalf of Weissman's
company. It was Klein's first appearance before the board on behalf
of a specific application.
“But private enterprise
is new territory for CIRM, which has steered almost all its grants
thus far to nonprofit institutions. Those efforts haven't been
trouble-free: With some 90% of the agency's grants having gone to
institutions with representatives on its board, the agency has long
been vulnerable to charges of conflicts of interest. The last thing
it needed was to show a similar flaw in its dealings with private
companies too.”
“(Weissman) has also
been a leading beneficiary of CIRM funding, listed as the principal
researcher on three grants worth a total of $24.5 million. The agency
also contributed $43.6 million toward the construction of his
institute's glittering $200-million research building on the Stanford
campus.”
$20 million for StemCells, Inc., came on 7-5 vote that also required
the firm to prove that it had a promised $20 million in matching
funds prior to distribution of state cash.
“The problem is that
StemCells doesn't have $20 million in spare funds. Its quarterly
report for the period ended June 30 listed about $10.4
million in liquid assets, and shows it's burning about $5 million per
quarter. Its prospects of raising significant cash from investors
are, shall we say, conjectural.
“As it happens, within
days of the board's vote, the
firm downplayed any pledge 'to raise a specific amount of
money in a particular period of time.' The idea that CIRM 'is
requiring us to raise $20 million in matching funds' is a
'misimpression,' it said. Indeed, it suggested that it might count
its existing spending on salaries and other 'infrastructure and
overhead' as part of the match. StemCells declined my request that it
expand on its statement.
“CIRM spokesman Kevin
McCormack says the agency is currently scrutinizing StemCells'
finances 'to see what it is they have and whether it meets the
requirements and expectations of the board.' The goal is to set
'terms and conditions that provide maximum protection for taxpayer
dollars.' He says, 'If we can't agree on a plan, the award will
not be funded.'"
“The agency shouldn't be
deciding on the spot what does or doesn't qualify as matching funds.
It should have clear guidelines in advance.
“Nor should the board
overturn the judgment of its scientific review panels without
clear-cut reasons....The record suggests that the handling of the
StemCells appeal was at best haphazard and at worst redolent of
cronyism.”
Source:Stem Cell Therapy
Comments Off on Los Angeles Times: StemCells, Inc., Award ‘Redolent of Cronyism’ |
October 21st, 2012
California Stem Cell Agency Boosting Disease Team Program to $543 Million
By Dr. Matthew Watson
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.
agency's push to develop therapies prior to running out of money for
new grants in 2017.
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.”
board action even when applicants have identified themselves.
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.
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.
“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.
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.
public on the budget plan although it could well have a significant
impact on their future efforts.
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:Stem Cell Therapy
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October 21st, 2012
Researcher Alert: Stem Cell Agency to Take Up Grant Appeal Restrictions
By Dr. Matthew Watson
The move by the $3 billion California
stem cell agency to curtail its free-wheeling grant appeal process
will undergo its first public hearing next week.
whose applications are rejected by reviewers will have fewer avenues
to pursue to overturn those decisions. The changes could take effect
as early as next year.
number of appeals this summer that left the board complaining about
“arm-twisting,” lobbying and “emotionally charged presentations.”
criteria for re-review – “additional analysis” – of
applications involved in appeals, also called “extraordinary
petitions.” The plan states that re-review should occur only in
the case of a material dispute of fact or material new information.
(See the end of this item for agency's proposed definitions.)
appeal process, the CIRM directors' Application Review Task Force
will take up questions involving “ex parte communications.” The
agenda for the Oct. 24 meeting did not contain any additional
information on the issue but it likely deals with lobbying efforts on
grants outside of public meetings of the agency. We understand that
such efforts surfaced last summer involving the $$214 million disease
team round and Robert Klein, the former chairman of the stem cell
agency.
the board on one, $20 million application by StemCells, Inc., the
first time a former governing board member has publicly lobbied his former
colleagues on an application. The application was rejected twice by reviewers – once
on the initial review and again later on a re-review – but it was
ultimately approved by directors in September on a 7-5 vote.
its appeal process but last summer's events brought the matter to a
new head. The issue is difficult to deal with because state law
allows anyone to address the CIRM governing board on any subject when
it meets. That includes applicants who can ask the board to approve
grants for any reason whatsoever, not withstanding CIRM rules. The board can also approve a grant
for virtually any reason although it has generally relied on
scientific scores from reviewers.
designed to make it clear to scientists whose applications are
rejected by reviewers that the board is not going to look with favor
on those who depart from the normal appeals procedure.
overturned a positive decision by reviewers, in nearly every round it approves some applications that have been rejected by reviewers. That has
occurred as the result of appeals and as the result of motions by
board members that did not result from public appeals.
diseases that they have been involved with. Sometimes that means
seeking approval of applications with low scientific scores.
“material dispute of fact:”
“A material dispute of fact should
meet five criteria:(1) An applicant disputes the accuracy of a
statement in the review summary;(2) the disputed fact was significant
in the scoring or recommendation of the GWG(grant review group); (3) the dispute pertains
to an objectively verifiable fact, rather than a matter of scientific
judgment or opinion;(4) the discrepancy was not addressed through the
Supplemental Information Process and cannot be resolved at the
meeting at which the application is being considered; and
(5) resolution of the dispute could affect the outcome of the board’s
funding decision."
define “material new information:”
“New information should: (1)be
verifiable through external sources; (2) have arisen since the
Grants Working Group(grant review group) meeting at which the application
was considered; (3) respond directly to a specific criticism or
question identified in the Grants Working Group’s review; and (4)
be submitted as part of an extraordinary petition filed five business
days before the board meeting at which the application is
being considered."
Children's Hospital in Oakland with a teleconference location at UC
Irvine. Addresses can be found on the agenda.
Source:Stem Cell Therapy
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October 21st, 2012
Yamanaka and the Frailty of Peer Review
By Dr. Matthew Watson
More than one back story exists on
Shinya Yamanaka and his Nobel Prize, but one that has received little
attention this week also raises questions about hoary practice of
peer review and publication of research – not to mention the
awarding of billions of dollars in taxpayer dollars.
article in the New Scientist magazine by Peter Aldous in which the
publication examined more than 200 stem cell papers published from
“2006 onwards.” The study showed an apparent favoritism towards
U.S. scientists. Also specifically reported were long delays in
publication of Yamanaka's papers, including in one case 295 days.
“All's fair in love and war, they
say, but science is supposed to obey more noble ideals. New findings
are submitted for publication, the studies are farmed out to experts
for objective 'peer review' and the best research appears promptly
in the most prestigious journals.
“Some stem cell biologists are crying
foul, however. Last year(2009), 14 researchers in this notoriously
competitive field wrote
to leading journals complaining of "unreasonable or
obstructive reviews". The result, they claimed, is that
'publication of truly original findings may be delayed or rejected.'
“Triggered by this protest, New
Scientist scrutinised the dynamics of publication in the most
exciting and competitive area of stem
cell research, in which cells are 'reprogrammed' to
acquire the versatility of those of an early-stage embryo. In this
fast-moving field, where a Nobel prize is arguably at stake,
biologists are racing feverishly to publish their findings in top
journals.
“Our analysis of more than 200
research papers from 2006 onwards reveals that US-based scientists
are enjoying a significant advantage, getting their papers published
faster and in more prominent journals (find
our data, methods and analyses here).
“More mysterious, given his standing
in the field, is why two of Yamanaka's papers were among the 10 with
the longest lags. In the most delayed of all, Yamanaka reported that
the tumour-suppressing gene p53 inhibits the formation of
iPS cells. The paper took 295 days to be accepted. It was eventually
published by Nature in August 2009 alongside four similar
studies. 'Yamanaka's paper was submitted months before any of the
others,' complains Austin
Smith at the University of Cambridge, UK, who coordinated
the letter sent to leading journals.
“Yamanaka suggests that editors may
be less excited by papers from non-US scientists, but may change
their minds when they receive similar work from leading labs in the
US. In this case, Hochedlinger submitted a paper similar to
Yamanaka's, but nearly six months after him. Ritu
Dhand, Nature's chief biology editor, says that each paper
is assessed on its own merits. Hochedlinger says he was unaware of
Yamanaka's research on p53 before publication.”
wrote of other issues dealing with peer review, but coincidentally
also dealing with iPS cells. What New Scientist and Knoepfler are
discussing is not an isolated situation. It is part of a continuum of
complaints, both serious and self-interested but exceedingly
pervasive. A Google search today on the term “problems with peer
review” turned up 10.1 million references. Writing on Ars Technica last year, Jonathan Gitlin, science policy analyst at the National
Human Genome Research Institute, summarized many of the issues, citing a “published” (our quotation marks)
study that said peer review doesn't work “any better than chance.”
Gitlin said,
“A common criticism is that peer
review is biased towards well-established research groups and the
scientific status quo. Reviewers are unwilling to reject papers from
big names in their fields out of fear, and they can be hostile to
ideas that challenge their own, even if the supporting data is good.
Unscrupulous reviewers can reject papers and then quickly publish
similar work themselves.”
agency, peer review is undergoing some modest, indirect examination
nowadays. The agency is moving towards tighter scrutiny of budgets
proposed by applicants. And, following a record wave of appeals this
summer by disgruntled applicants rejected during peer review, it is
also moving to bring the appeal process under more control.
more successfully towards development of commercial therapies, it may
do well to consider also the frailties of its peer review process and the
perils of scientific orthodoxy.
Source:Tighter Controls on Stem Cell Grant Budgets Hits Quorum Bump
By Dr. Matthew Watson
SAN FRANCISCO – A move to tighten
budget controls on grants from the $3 billion California stem cell
agency stalled Monday, but it appears that the plan is headed for
ultimate approval.
by the agency's directors' Science Subcommittee, which could not act
on it after it lost its quorum.
the stronger budget controls, but had questions about the specifics
of implementing the plan during closed-door reviews of grant
applications. The proposal is likely to be altered to respond to
those concerns. It would then either come back to the Science
Subcommittee or go to the full board.
recipients of large grants that approval of an application by the
agency's governing board does not provide a carte blanche to
researchers. Ellen Feigal, senior vice president for research and
development, said it can be “extremely difficult” for CIRM staff
to deal with budget problems in grants following board approval.
speed the application process on its next disease team round, which
is aimed at driving research into the clinic. The concept proposal
for that round is scheduled to come before directors later this
month. The round will be limited to “more mature stage” research
that is close to a clinical trial, if not in one. Feigal said 10 to
15 applications are expected.
to CIRM's strategic partnership program was also approved.
Source:Stem Cell Therapy
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October 14th, 2012
Yamanaka: ‘Rejected, Slow and Clumsy’
By Dr. Matthew Watson
This week's announcement of the Nobel
Prize for Shinya Yamanaka brought along some interesting
tidbits, including who was “snubbed” as well as recollections
from the recipient.
wrote the “snubbed” piece and quoted Christopher Scott of
Stanford and Paul Knoepfler of UC Davis about the selection issues.
Bardin's piece mentioned Jamie Thomson and Ian Wilmut as scientists
who also could have been considered for the award but were not named.
Ultimately, Bardin wrote that the award committee was looking for a
“singular, paradigm shifting discovery,” which he concluded was
not the case with Thomson or Wilmut.
was another topic in the news coverage, much of it dry as dust.
However, Lisa Krieger of the San Jose Mercury News began her story
with Yamanaka's travails some 20 years ago. At the time, no one was returning his phone
calls as he looked for work, and he was rejected by
50 apparently not-so-farsighted American labs.
decided he was less than successful as an orthopedic surgeon,
according to an account in JapanRealTime. “Slow and clumsy” was
how Yamanaka described himself.
again he reported stumbling. In this case, he found a way to reduce
“bad cholesterol” but with a tiny complication – liver cancer.
That in turn sent him on a journey to learn how cells proliferate and
develop, which led him to the work that won the Nobel Prize.
orthopedic medicine was stimulated by his father along with the treatments
for injuries young Yamanaka received while playing rugby and learning judo. The JapanRealTime account continued,
“'My father probably still thinks in
heaven that I’m a doctor,' he said in the interview(with Asahi
Shimbun last April). 'IPS cells are still at a research phase and
have not treated a single patient. I hope to link it to actual
treatment soon so I will be not embarrassed when I meet my father
someday.'”
report from a high school biology teacher that said the 15-year-old
Gurdon's desire to become a scientist was “quite ridiculous.”
The teacher, who is unnamed, wrote,
“If he can’t learn simple
biological facts he would have no chance of doing the work of a
specialist, and it would be a sheer waste of time, both on his part
and of those who would have to teach him.”
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/7J31SRIukpg/yamanaka-rejected-slow-and-clumsy.html
Stem Cell Orthodoxy and Peer Review
By Dr. Matthew Watson
Going against the grain can be
difficult as UC Davis stem cell scientist Paul Knoepfler learned
again in connection with his research that dealt with similarities
between cancer and iPS cells.
some scientists who reviewed his paper prior to its publication in
September in Stem Cells and Development. (See here and here.)
reprogrammed adult cells are currently a hot research avenue in stem
cell research because they avoid many of the ticklish ethical and
political problems connected with human embryonic stem cells.
publication and peer review process on his blog last week. He wrote,
“Not surprisingly...there are certain
members of the stem cell field who would rather focus away from the
ideas that iPS cells are similar in some respects to cancer.”
in part by the California stem cell agency, wrote,
“Once we had a manuscript together
comparing iPS cells to cancer cells, we sent it to several high
profile journals without much luck. We thought that the fact that our
data indicated that iPS cells are similar to cancer cells might make
reviewers and editors excited. We thought that the paper was novel
and thought provoking in a number of ways. At the same time I
realized the theme of the paper would be controversial.
“I would say two general things about
the review process at the two journals that turned down the paper.
First, the reviewers at these journals were enormously helpful with
their suggestions and helped us improve the paper substantially.
Second, they were clearly very uncomfortable with the notion that iPS
cells are related in some ways to cancer so unsettled in fact that I
believe it influenced their reviews.”
findings were either “not sufficiently novel” or “trivial.”
“Little useful insights” said another. And a third said, “many
unsettling results....”
“Yeah, it may be unsettling that iPS
cells share traits with cancer cells, but if that is the reality,
isn’t it important that people know that and think about it, talk
about it, and address the issue with eyes open?”
from other researchers that can found elsewhere on the Internet
indirectly raise questions about the California stem cell agency's process
of peer review of applications for hundreds of millions of dollars in
funding, especially in the wake of this summer's unprecedented rash of appeals of decisions by grant reviewers.
amounts to scientific conventional wisdom. Obviously, no researcher
likes to see a paper rejected or a grant denied. But the record
number of appeals at CIRM and other private complaints could well indicate
that potentially profitable proposals are receiving a less than
welcome reception behind closed doors from agency reviewers.
hard-pressed to make such determinations. It is hamstrung by
procedures that do not permit it to expand an application directly –
only a staff-written summary. Names of applicants and institutions
are censored, although the board is required by law to discuss in
public most aspects of a research proposal. Exceptions are permitted for proprietary information. Additionally, a handful of the 29 members of the governing board do participate in the reviews, which come before final action by the board.
commercialize stem cell research and fulfill at least some of the
promises to voters that were made in 2004. To do that, the agency may
well have to step outside of the normal comfort zone of the good
burghers of stem cell science.
Source:Yamanaka and the Golden State
By Dr. Matthew Watson
The iPierian biopharmaceutical company
in South San Francisco was quick to make a change in its web site
this morning after the Nobel Prize for medicine was announced.
scientific advisors, Shinya Yamanaka, to note that he had won the
Nobel. The bio is tucked away on the site, but it is likely that the
company, which specializes in iPS work, will figure out how to put
the news out front on its home page as well as issue a press release.
It was all part of the reaction todayin California to the Nobel for Yamanaka, who has substantial links to
the Golden State, including UCSF and the Gladstone Institutes.
deftness than iPierian. Yamanaka is a professor at UCSF and a senior
investigator at Gladstone, and the organizations quickly put together a news conference this morning that featured Yamanaka on a video
hook-up from Japan.
issued a press release that quoted the president of Gladstone, R.
Sanders Williams, who also mentioned the California stem cell agency.
Williams said,
“Dr. Yamanaka’s story is a
thrilling tale of creative genius, focused dedication and successful
cross-disciplinary science. These traits, nurtured during Dr.
Yamanaka’s postdoctoral training at Gladstone, have led to a
breakthrough that has helped propel the San Francisco Bay Area to the
forefront of stem cell research. Dozens of labs — often supported
by organizations such as the California Institute for Regenerative
Medicine (CIRM) and the Roddenberry Foundation–have adopted his
technology.”
stem cell effort, published an item on its blog quoting CIRM
President Alan Trounson. He said,
"There are few moments in science
that are undisputed as genuine elegant creativity and simplicity.
Shinya Yamanaka is responsible for one of those. The induced
pluripotent stem cells he created will allow us to interrogate and
understand the full extent and variation of human disease, will
enable us to develop new medicines and will forever change the way
science and medicine will be conducted for the benefit of mankind. An
extraordinary accomplishment by a genuinely modest and brilliant
scientist. He absolutely deserves a Nobel award.”
agency's communications manager, said that just five years after
Yamanaka's research,
“CIRM alone is funding almost $190
million in awards developing better ways of creating iPS cells and
using those cells to develop new therapies (the
full list of iPS grants is on our website).”
cash is the well-connected iPierian, which has taken in $7.1 million.
Yamanaka, however, has never received a grant from the agency, and
it is not known whether he ever applied since CIRM releases only the
names of researchers whose applications were approved.
http://feedproxy.google.com/~r/blogspot/uqpFc/~3/RbQ09EsO8Qc/yamanaka-and-golden-state.html
Researcher Alert: California Stem Cell Agency Tightening Budget Oversight on Grants
By Dr. Matthew Watson
Some of California's top stem cell
researchers are going to have to sharpen their spreadsheets if they
want to win money from the state's $3 billion stem cell agency.
scrutiny of the high-profile, big-ticket grant applications
that it will consider during the next several years. The effort may well extend to all grant programs. The move also makes
it clear to researchers that the CIRM staff is in the driver's seat
when it comes to budgeting on research projects.
president for research and development. She said,
“Increasing the importance of
budgetary review will encourage applicants to propose rigorous,
realistic and vetted budgets, and will further our mission to be good
stewards of taxpayer dollars. These additions will not significantly
increase the workload burden on GWG members (grant reviewers) and
explicitly acknowledge that program goals, scientific plans, accurate budgeting and prudent spending are inextricably linked.”
directors' Science Subcommittee next Monday and would alter the
closed-door grant review process in the following manner, according
to Feigal's memo.
• “To assist GWG review,
appropriate expertise on budget and financial matters (e.g., this
could be in the form of a specialist reviewer, or can also be
assigned to a GWG reviewer with the appropriate background and
expertise), will review applications for sound budgeting and provide
comments or questions to the GWG for consideration by the reviewers
before the reviewer’s final scores are entered.
• “If the financial/budgetary
matter potentially directly impacts on the design or feasibility of
conducting the project, the GWG may consider this issue in the
scoring; otherwise, budgetary and financial issues and questions will
not contribute to the scientific score.
• “As appropriate, review summaries
sent to the ICOC (the CIRM governing board) will identify scientific
as well as budget or other issues. To the extent endorsed by the
GWG, the review summaries will also identify potential resolution
should the ICOC approve a given award with budget issues.
• “CIRM officers should be provided
explicit discretion to consider the budget comments, as well as
budget or other issues. To the extent endorsed by the GWG, the
review summaries will also identify potential resolution should the
ICOC approve a given award with budget issues.”
CIRM staff has experienced push-back from recalcitrant researchers
when efforts have been made to bring costs under control. She noted that
the agency's staff examines a research project's budget during the
“prefunding” review that follows board approval. However, Feigal
said, at that stage, “It is often challenging to make substantive
changes to the budget, based on appropriateness of study activities
and costs, given the ICOC approval at a given budget amount.”
budgets prior to board approval. One grant review in a $200
million-plus round this summer, for example, declared that costs to
prepare regulation packages had “overlap” and were “excessive,”
along with costs dealing with manufacturing and per patient expenses.
That was for a high-scoring application by Antoni Ribas of UCLA, and
he was not alone.
of budgetary shortcomings in recent applications:,
• “Budget does not align with the
program deliverables and milestones. For example, the budget
includes activities not relevant to project objective(s) or that are
out of scope.
•”Budget does not contain adequate
expenses for known costs. For example, an applicant may budget
$100,000 for a GMP manufacturing run of a biologic in which it is
generally accepted knowledge that the actual expenses are typically
much greater.
•“Budget item significantly exceeds
a known cost or seems excessive without adequate justification. For
example, an applicant may propose a surgical expense of $100,000 per
patient for a procedure with Medicare reimbursement set at $15,000.
•“Cost allocations are not done
properly. For example, an applicant is developing the same
therapeutic candidate for 3 indications, and is applying for CIRM
funding for 1 of the 3, but is charging CIRM for the cost of the
entire manufacturing run.”
be used in disease team, early translational, strategic partnership
rounds, and any new rounds “as deemed appropriate.” Feigal said,
however, that “all applications for CIRM awards should be
carefully examined for budgetary appropriateness.”
well-advised move, albeit one that is not likely to find favor with
researchers accustomed to loose oversight. It moves budgetary review
to an earlier stage and gives the CIRM directors a chance to weigh in
on those matters prior to approval of grants, instead of creating a
sense of entitlement on the part of recipients that may pop up
following board approval of their applications. Indeed, the plan
makes such good sense that it raises the question why it was not in
place years ago.
excellent example of the type of information that clarifies issues
and helps CIRM directors make the best possible decisions. It
provides some history, good evidence for a change and an explanation
of benefits. Additionally, the memo is timely, having been posted on
the CIRM website sufficiently in advance of next week's meeting to give affected parties and others time to comment
and make constructive suggestions. The memo is also far superior to
the Power Point presentations that are often submitted to the board
minus any nuanced, written discussion of the issue at hand.
San Francisco but also has teleconference locations in Irvine (2), La
Jolla, Stanford, Pleasanton, Oakland and Los Angeles where the public
and researchers can participate. The specific addresses can be found on the agenda.
Source:Stem Cell Therapy
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October 7th, 2012
UCD’s Knoepfler’s ‘Somewhat Provocative Paper’ on iPS
By Dr. Matthew Watson
UC Davis researcher Paul Knoepfler is
the rare stem cell scientist who blogs about his work as well as
writing about issues in the field.
what he described as a “somewhat provocative paper” published by his lab in
“Stem Cells and Development.” He said the paper argued
that iPS cells “are very similar in some ways to cancer cells.”
technical details and background of the research. But at the end of
this item, Knoepfler wrote,
“So what does this mean in the big
picture?
“I believe that iPS cells and cancer
cells are, while not the same, close enough to be called siblings. As
such, the clinical use of iPS cells should wait for a lot more study.
Even if scientists do not use iPS cells themselves for transplants,
but instead use differentiated derivatives of iPS cells, the risk of
patients getting malignant cancers cannot be ignored.
“At the same time, the studies
suggest possible ways to make iPS cells safer and support the notion
of reprogramming cancer cells as an innovative new cancer therapy.
“Stay tuned in the next few days for
part 2 where I will discuss what this paper went through in terms of
review, etc. to get published. It wasn’t a popular story for some
folks.”
research, which was financed by the California stem cell agency and the NIH, was picked up by several online sites, including Redorbit,
Medicalexpress and geekosystem.
Source:Stem Cell Therapy
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October 7th, 2012
$700,000 Blue-ribbon Study of CIRM All But Finished
By Dr. Matthew Watson
The $700,000 study of the $3 billion
California stem cell agency is nearly concluded and is expected to be
released sometime in November.
for “peer review” and no additional public meetings are
scheduled, according to a spokeswoman for the Institute of
Medicine(IOM), which is conducting the study. The IOM did not respond
to questions from the California Stem Cell Report about the number of peer reviewers or how they were selected.
part, because agency directors hoped the findings by the blue-ribbon
panel would bolster efforts to win voter approval of another multi-billion dollar state bond issue. More recently the agency has
explored the possibility of private financing to continue operations.
funds for new awards in 2017. It currently has something in the
neighborhood of $700 million for awards that is not already committed
in one fashion or another.
relations officer for the IOM, said in an email,
“There will be no
further information-gathering meetings. The committee members have
finished drafting their report and it is now undergoing peer review.
Reviewers are anonymous to study staff and committee members; they
will be listed in the front matter of the report when it’s finished
and released.”
cell agency will not be given an opportunity to comment further.
Stencel said,
“Sponsors are not
treated as peer reviewers; that is, they’re not afforded an
opportunity to comment on IOM draft reports prior to public release.
IOM is aiming for a public release in November (the exact time frame
will hinge on the duration of the peer review, which is influenced by
people’s schedules and adherence to deadlines). IOM is looking at
options for how best to hold this release, whether there will be an
event of some sort. Once plans are set, they’ll be noted on the
project web pages and IOM will alert the various stakeholders and
interested parties of the plans. The study is moving along and we’re
looking forward to the report’s debut in the not too distant
future.”
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss
Stem Cell Therapy
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September 30th, 2012