The governing board of the $3 billion California stem cell agency this morning began its meeting at 9:08 a.m. with an immediate comment on the Trounson Affair.
Board Chairman Jonathan Thomas reiterated the steps taken by the agency so far and said maintaining the public trust in the agency is paramount. CIRM President Randy Mills is expected to make further comments later in the meeting.
With more than 3.0 million page views and more than 5,000 items, this blog provides news and commentary on public policy, business and economic issues related to the $3 billion California stem cell agency. David Jensen, a retired California newsman, has published this blog since January 2005. His email address is djensen@californiastemcellreport.com.
Thursday, July 24, 2014
Coming Up: Live Coverage of Today's California Stem Cell Meeting
The California Stem Cell Report will provide live, gavel-to-gavel coverage of today's meeting of the 29 members of the governing board of the $3 billion California stem Cell agency, which is expected to lop $15 million from its ambitious Alpha stem cell clinic proposal.
Not on the agenda, however, is the so-called Trounson Affair, although the matter (see here and here) may well come up at the session, which begins at 9 a.m. PDT.
Not on the agenda, however, is the so-called Trounson Affair, although the matter (see here and here) may well come up at the session, which begins at 9 a.m. PDT.
Interested parties in Canada will be able to participate in the meeting from the W Montreal Hotel, where one of the CIRM directors is staying. However, the agenda does not mention a room number so our advice is to check with the agency for specifics. The room is public by law for CIRM meeting purposes.
Other teleconference locations are available in Los Angeles, which has two, and one each in La Jolla and Napa.
The meeting, which will be in Millbrae, Ca., can be heard through the Internet via an audiocast. Directions for logging in can be found on the agenda.
A Brief Look at the $12 Million California Stem Cell Bridging Program
The directors of the California stem cell agency later today
are expected to approve $1.2 million for two University of California stem cell
researchers as part of what the agency calls a “bridging” program.
The effort has received little attention but could be a key
for some research as scientists try to turn more basic efforts into something
that could reach the marketplace.
Patricia Olson, executive director of scientific activities
, has prepared some slides for today’s meeting in Millbrae, Ca., that lay out a
bit of the scope of the effort.
She reported that the goal of the program is to accelerate
development of stem cell therapies. She said the bridging program is aimed at
providing an “efficient and seamless advancement of promising CIRM-funded
translation and development projects towards and through clinical development.”
No RFA is used for the effort. Instead scientists must
submit a brief proposal that could lead to further consideration. Up to $12
million has been allotted to the effort. Olson did not present information on
how many proposals have been submitted.
Wednesday, July 23, 2014
California Stem Cell Agency Director Prieto Defends Agency in Trounson Affair
One of the long-standing directors of the $3 billion
California stem cell agency has taken issue with an item earlier today
headlined “Fallout From the Trounson Affair: A Taint on the California Stem Cell Agency.”
In an email, Francisco Prieto, a Sacramento
physician, said, among other things, that it is “grossly unfair” to say that none
of the media coverage and other reaction in the matter reflects well the
agency. Prieto, who has served as a CIRM director since 2004, said “the agency
had nothing to do with it.”
The item this morning discussed reaction to the news
earlier this month that the agency’s former president, Alan Trounson, was
appointed to the board of StemCells, Inc., which is the recipient of $19.4
million in funding from the stem cell agency. Concerned about a conflict of
interest, the agency has announced a “full review” of all StemCells, Inc.,
activities. Trounson was appointed seven days after he left the agency.
The item also contained a comment from scientist Jeanne Loring
of Scripps that said that the Trounson Affair detracts from the value of CIRM’s
good work.
Here is the text of Prieto’s comment, the essence of
which is certain to be shared by many CIRM board members,
“I think Jeanne Loring is right: CIRM has been a remarkable driver of this research, and it would be a shame if the actions of Dr. Trounson and StemCells Inc. (What were they thinking?) obscures this. I think it is grossly unfair to say that ‘none of this reflects well on the agency,’ when the agency had nothing to do with it. Most of us on the board – and the staff, I think it’s safe to say – felt blindsided by this. I was gratified to see Randy Mills’ prompt and appropriate response to this, and I expect we’ll hear more from him on the subject. I think it’s a bit disingenuous of Michael Hiltzik (of the Los Angeles Times) to say that ‘so many members had to recuse themselves that only nine were left to vote,’ when the new voting procedures (that prohibited members from grant-receiving institutions from discussing or voting on those grants) were a direct response to the reform recommendations in the IOM report, and were lauded by most at the time. I don’t think that included Mr. Hiltzik, who I believe has never had anything good to say about the agency or its work. I’m curious whether that will change as stem cell treatments we’ve funded actually start moving into clinical trials, but I won’t hold my breath.”
The California Stem Cell Report has great respect
for Prieto and the other 28 members of the agency’s board and its staff.
However, there is no escaping the impact of the news
and the resultant commentary, which will be around virtually forever, embedded
in every Internet search that is performed about the stem cell agency. Today,
for example, a Google search on the term “California stem cell agency” turned
up eight hits on the first page of search returns. Five dealt with the Trounson
Affair.
Moreover, conflict of interest concerns were aired very
early on in CIRM’s history, dating back to 2004, before the ballot proposal
creating the agency was even approved by voters. Revolving door issues also
came up years ago, including in 2007 when Richard Murphy, a former member of
the board, was hired as interim president at a salary of $300,000 for six
months work. The Little Hoover Commission mentioned the issue briefly in its
88-page report in 2009.
Additionally, given that financing of the agency was
limited to 10 years, revolving door problems were always likely to surface. It
was an issue that could have been dealt with by the board years ago, avoiding
the situation with Trounson today. Revolving door restrictions could have and
should have been part of his conditions of employment.
The IOM’s recommendations for dealing with
conflict-of-interest problems at the agency were far-reaching. The steps taken
by the agency do little to comply with the IOM recommendations. The strange case of having only nine out of 29
members eligible to vote is not all that uncommon. Indeed, the agency has
worked hard to keep its 12 patient advocate members in attendance at board meetings
because sometimes they are the only ones who can vote without legal
conflicts-of-interest.
The situation with Trounson is certainly
unpleasant. Whether board members think
the reaction is unfair is not the main point. It is up to them to take action
to respond to those public concerns and ensure that the agency’s integrity is
reinforced and that its work is not impugned by conflicts of interest, real or
perceived.
Fallout From the Trounson Affair: A Taint on the California Stem Cell Agency
Directors of the $3 billion California stem cell agency meet
tomorrow in the San Francisco Bay Area, but the hottest topic is not on
the agenda, and that is the Trounson Affair.
Alan Trounson SF Business Times photo |
The term is shorthand for the host of questions raised
by the appointment July 7 of Alan Trounson, former president of the agency, to the board of StemCells, Inc. The company is the recipient of $19.4
million from the agency, cash that was awarded under unusual circumstances on a narrow 7-5 vote.(See here and here.)
Two days after Trounson’s appointment, the agency’s
new president, Randy Mills, announced a “full review” of the activities
involving StemCells, Inc., a publicly traded firm based in Newark, Ca.
News and commentary about the matter has slowly
emerged since the appointment. None of it reflects well on the agency, which is
trying to devise some way to secure public or private funding beyond 2017 when
the cash for new awards runs out.
The most visible article appeared in the Sunday Los
Angeles Times, which has a readership of 4 million in print and online. Michael Hiltzik wrote that cronyism is rife at the California Institute for Regenerative Medicine(CIRM),
as the agency is formally known. He
said,
“How bad are the conflicts? When the (29-member) board considered a proposal earlier this year to spend $16 million to attract three star scientists to California, so many members had to recuse themselves that only nine were left to vote. (Six ended up voting in favor.)
“When conflicts of interest are so rife that only one-third of your board can weigh in on a major policy issue, that's tantamount to not having any board at all.”
The tone was echoed in other pieces, including one
in the San Francisco Business Times, where the headline said,
“'Trounson affair' another strike against California stem cell agency.”
Other articles appeared in the San Diego U-T and the
San Francisco Chronicle. The Orange County Register published an editorial that
said the agency has “operated as a cash cow for a tiny circle of well-connected
individuals and institutions.”
The Trounson appointment “reignites charges of
cronyism,” said a headline on allgov.com. Pete Shanks of the Biopolitical Times wrote,
"Let's be blunt: This looks like a pay-off. Technically, what Trounson and (Irv) Weissman (of Stanford) and StemCells, Inc., just did may not be illegal. But it's shameless.”
The Scientist magazine wrapped the Trounson appointment into a piece that included news about a lawsuit against StemCells, Inc., that
charged the company was endangering patients in clinical trials.
The talk ricocheted around California’s stem
cell community. One longtime reader of the California Stem Cell Report, who is
a patient advocate, said in an email that Trounson’s conflicts of interest are “now
even more transparent and make the previous grant decisions even more suspect.”
“Self-dealing” is how this supporter of stem cell research described the
situation.
|
“No one seems to understand what conflict of interest means. I don't understand how they can even think that it is OK to do this.”
We should note that these remarks come from persons
who back stem cell research and the agency.
In some ways, however, public attention to the
matter could be described as minimal. The stem cell agency is little known to most
people, and the Trounson Affair did not garner front page headlines. But like some
sort of immortal cell, the stories will live on. Twenty years ago, the
individual stories would have faded, buried in the morgues of the mainstream
media. However, today, given Google searches, they will become embedded in all
future reporting about the agency – not to mention knife-edged opposition
research should another bond measure be placed before voters to fund the
agency.
The Trounson Affair also highlights the need for the
agency to stiffen its revolving door restrictions – the regulations that would
have prevented his immediate appointment to a post at an enterprise that has benefited
from the agency’s largess. It is a problem that will grow as the agency nears
its financial demise. CIRM staffers will naturally be looking for places where
they can find future employment.
The state has some minimal laws restricting future
employment by agency personnel. But the agency needs to do more, a difficult
task given that such action basically will change the terms of employment for
staffers. But failure to confront the issue will only lead to more debacles.
CIRM’s “full review” of the situation is well-taken.
However, the agency has not publicly defined even generally what a full review entails nor
has it responded to questions from the California Stem Cell Report about the
nature of the inquiry. The agency also has not indicated whether it is seeking an
outside, independent entity to conduct the review. One possibility would be
State Controller John Chiang, who is the chairman of the only state body (the
Citizens Finanancial Accountability and Oversight Committee) charged with
oversight of the agency. Another would be the state’s Fair Political Practices
Commission, the state department charged with enforcing conflict of interest
laws.
One California researcher and CIRM grant recipient has
noted that the flap obscures the work that the stem cell agency has done. In a
comment filed on an item on the California Stem Cell Report, Jeanne Loring,
director of the Center for Regenerative Medicine at The Scripps Research
Institute, said that despite “the errors in judgment” at the agency,
“CIRM is at the heart a remarkably effective driver of cutting edge research. By focusing on a narrow area of research and encouraging international collaboration, CIRM has singlehandedly pushed the whole world's stem cell research forward. If CIRM were a drug company, it would be considered miraculous that so many promising treatments are in the pipeline just eight years after it started."
Loring makes a good point. And it would be a shame
if the good work of CIRM is discredited because of a failure to deal
forthrightly, quickly and publicly with conflict of interest issues at the
agency.
Cloaking Informed Consent in Clinical Trials: A California Stem Cell Case
Patients come first. That’s the rhetoric from
biotech companies and agencies like the FDA, which has oversight responsibilities
for clinical trials.
But when it comes down to specifics, the doors may close
and little revealed publicly.
The case in point involves StemCells, Inc., which
was charged last week in a lawsuit with endangering patients involved in its clinical
trials. The suit was filed by an unhappy manager who was fired by the Newark,
Ca., firm. StemCells, Inc., said the charges lack merit.
But the firm is mum when it comes to the question of
whether it has informed its patients about the allegations and its response.
Those are the folks whose brains are being injected with what is alleged to be
a product that is being manufactured improperly. They are supposed to give
informed consent to the treatment and obviously need to know when serious
questions are raised about their therapy.
Queried by the California Stem Cell Report about
whether the patients in its clinical trials had been informed of the allegations,
Ken Stratton, general counsel for StemCells, Inc., said,
“StemCells, Inc. has no further comment on the ongoing litigation or Mr. Williams’ allegations.”
The FDA and the attorney for the fired manager responded
in much the same fashion. Asked whether his client, Rob Williams, or he has
notified the FDA about the alleged problems, attorney Daniel Velton said,
“We can't comment on pending cases.”
The FDA said,
“As a matter of policy FDA cannot comment on whether or not we are investigating or have plans to investigate any allegations.”
Questioned further about the federal rules for giving patients the information they need to provide informed consent, Paul
Richards, a spokesman for the FDA said,
“Participation in any clinical trial is associated with some level of risk as the safety of investigational products has not been fully assessed. FDA’s primary responsibility is to determine whether the theoretical risks of the proposed study are reasonable and acceptable in order for the study to proceed “It is the responsibility of the study sponsor to conduct an investigation properly, to ensure proper monitoring of the investigation and to ensure that the investigation is conducted in accordance with the general investigational plan and protocols contained in the Investigational New Drug (IND) application. The sponsor also ensures that FDA and all participating investigators are promptly informed of significant new adverse effects or risks with respect to the product being studied.
“Additionally, the individual who actually conducts the clinical investigation (i.e. the investigator) is responsible for ensuring that the trial is conducted according to the signed investigator statement, the investigational plan, and applicable regulations. The investigator is also responsible for protecting the rights, safety, and welfare of subjects under the investigator's care; and for the control of drugs under investigation.
“In certain situations in which FDA alleges a clinical investigator has violated applicable regulations, FDA may initiate a clinical investigator disqualification proceeding. FDA does maintain a publicly available database that provides information about disqualification actions. Further background related to this topic is available at: http://www.fda.gov/ICECI/EnforcementActions/ucm321308.htm”
No doubt seems to exist that the patients should be
informed about the allegations in the lawsuit. However, implementation of the requirement is considerably less than transparent. How does the public know whether
patients have actually been informed? How
do the patients themselves know whether they have been adequately informed? Trust
us is the operative and dubious response.
One must ask whether these tight-lipped non-responses are in the best interests of patients, biomedical research, the government or
even the companies. Patients and the public deserve more than lip-service to
the process of informed consent. The parties involved in the StemCells, Inc.,
litigation, as well as the government, can do better.
What possible harm could
result from simply saying, “Yes, the patients have been told of the lawsuit and
the company’s response.” Such a response might help to inspire confidence among
persons considering clinical trials and help recruit the patients needed to
test possible new therapies.
Labels:
clinical trials,
fda,
informed consent,
openness
Tuesday, July 22, 2014
Coming Up: Live Coverage of Thursday's California Stem Cell Board Meeting
The California Stem Cell Report will provide live, gavel-to-gavel
coverage of this Thursday’s meeting of the 29 members of the governing board of
the $3 billion California stem Cell agency, which is expected to lop $15
million from its ambitious Alpha stem cell clinic proposal.
Interested parties in Canada will be able to
participate in the meeting from the W Montreal Hotel, where one of the CIRM
directors is staying. However, the agenda does not mention a room number so our
advice is to check with the agency for specifics. The room is public by law for
CIRM meeting purposes.
Other teleconference locations are available in Los
Angeles, which has two, and one each in La Jolla and Napa.
The meeting, which will be in Millbrae, Ca., can be
heard through the Internet via an audiocast. Directions for logging in can be
found on the agenda.
California Stem Cell CEO Randy Mills on Focus and Four-Part Tests
It was a case of CEOs interviewing CEOs.
More specifically, it was Robin Smith, head of Neostem,
Inc., “grilling” Randy Mills, the new CEO of the $3 billion California
stem cell agency. The venue
was the Huffington Post.
It wasn’t exactly hard ball stuff, as one might
expect. Nonetheless it was the lengthiest such exchange with Mills, the former
president of Osiris Therapeutics of Maryland, since he was named
president of the stem cell agency in April.
Neostem, by the way, does not hold any awards from
the agency, although it does have operations in Mountain View and Irvine. The
Southern California location is the site of the former California Stem
Cell, Inc., which was purchased
by Neostem for $126 million earlier this year, obviously making Neostem a potential applicant for CIRM funds.
Here are some excerpts from Smith’s interview with
Mills.
On leaving Osiris and coming to California, Mills,
“I started discussing with my family what might be next, including taking a break to spend more time with them. However, fate had other plans.
“In February I received a call from CIRM asking if I was interested in the President and CEO position. Having spent the past five years as a grant reviewer for CIRM, I was already quite familiar with the stem cell agency. If you believe in the potential of regenerative medicine and cell therapy as I do, there is no place in the world you could go to have a bigger impact. No company, no other state, not even a country can have the impact California can have in bringing these treatments to patients. And so with that, I accepted the challenge.”
Mills’ four-part test for CIRM projects:
“First, will what we are doing speed up the development of stem cell treatments for patients? Second, will it increase the likelihood of a successful treatment reaching patients? Third, is it for an unmet medical need? And lastly is it efficient?”
Mills’ focus on “focus,” something he mentioned in
slicing $15 million from the $70 million Alpha stem cell clinic plan.
“For CIRM to achieve its full potential, I firmly believe we need to remain focused on bringing treatments to patients, fast. Everyone loves that word, ‘focus.’ However, what they may not love is living with its reciprocal, which is everything else we don't do. Without focus, you never have to have the hard conversation. You never have to say ‘no.’ However, without focus, you also tend to not get things done. I came to CIRM to get stem cell treatments to patients in need, and that means focus.”
Mills on funding projects that “otherwise will get
done.”
“CIRM exists under the principle of ‘If not for us....’ California stepped up when, and most importantly because, others would not. Funding something here-and-now that will otherwise get done without CIRM is not consistent with what the people of California wanted to accomplish with Proposition 71(which created the agency). It also violates my third rule, because it wouldn't really be an unmet need if it were going to happen in any event.
“We are here to help get stem cell treatments that, if not for us, would take longer to reach patients or might not happen at all. If you look at the projects we support that are now heading into clinical trials, many would never have even gotten off the ground if it hadn't been for us.”
Mills did not mention this, but funding such projects
is also high risk. That’s why they haven’t been funded. So the reasonable
expectation is to see a few failures – perhaps more than a few – among the CIRM
efforts.
Verastem Bid for Nearly $10 Million from California Nixed
A Massachusetts firm called Verastem, Inc., heard some bad
news recently about its pitch for $9.9 million from the $3 billion California
stem cell agency.
The Cambridge firm sought the cash from the agency to help
out with a clinical trial dealing with breast cancer.
However, the agency’s directors were told in a memo on the
CIRM Web site,
“Weaknesses in the scientific merit of the proposal combined with portfolio considerations led to a staff recommendation NOT to fund.“
By portfolio considerations, the staff seemed to mean that
the agency had already awarded funds in the same scientific area.
The agency’s reviewers, who come from outside California,
gave the application of score of 74. Formal action will be taken on the application at Thursday’sboard meeting in the San Francisco Bay Area.
Monday, July 21, 2014
Scripps Confirms Marletta's Plan to Leave as President
The Scripps Research Institute has confirmed the departure of Michael Marletta as its president in a 72-word statement, but details about who will replace him and when are yet to emerge.
The move was announced this morning at a meeting at the 2,900-employee institute in La Jolla. Later, Scripps released its terse statement. Not mentioned were any proposals to deal with the financial plight of the renown biomedical research organization, which is the issue at the root of Marletta's resignation.
Bradley Fikes and Gary Robbins of the San Diego U-T have more details and background. Here is the Scripps statement, which is oddly worded, seeming to leave open the possibility of Marletta remaining at Scripps. Fikes and Robbins nonetheless report that four scientists at the morning meeting said Marletta is resigning.
The move was announced this morning at a meeting at the 2,900-employee institute in La Jolla. Later, Scripps released its terse statement. Not mentioned were any proposals to deal with the financial plight of the renown biomedical research organization, which is the issue at the root of Marletta's resignation.
Bradley Fikes and Gary Robbins of the San Diego U-T have more details and background. Here is the Scripps statement, which is oddly worded, seeming to leave open the possibility of Marletta remaining at Scripps. Fikes and Robbins nonetheless report that four scientists at the morning meeting said Marletta is resigning.
"The Scripps Research Institute (TSRI) Board of Trustees has announced that Dr. Michael Marletta has indicated his desire to leave TSRI and the Board is working with Dr. Marletta on a possible transition plan. Should Dr. Marletta depart TSRI, the Board will work to make any transition to new leadership the highest priority. Any such transition will engage all key constituencies in a dialogue about the future direction of this storied institution."
Scripps Research President Resigns Following Financial Problems at the Institute
Michael Marletta today resigned as head of The Scripps
Research Institute, one of nation’s leading biomedical research organizations,
in the wake of an aborted, $600 million merger with the University of Southern
California.
Bradley Fikes and Gary Robbins of the San Diego U-T reported that the announcement of his resignation came at a meeting this morning at the La Jolla facility. They wrote
that no successor has been named.
Marletta joined Scripps in January 2012. His resignation
came in the wake of financial problems at the institute, which is operating
this year under a $21 million deficit. Leading members of the faculty called
for his removal after the proposed merger came to light.
Scripps has not yet made a formal announcement on its Web Site.
Scripps has not yet made a formal announcement on its Web Site.
California's Far-Reaching Alpha Stem Cell Clinic Plan Likely to Take a Hit
The new president of the California stem cell agency today recommended cutting $5 million from the agency’s ambitious Alpha stem cell
clinic plan, declaring that the proposal’s $70 million cost is “not clearly
justified.”
If the agency’s board goes along with the
recommendation at Thursday’s meeting, it will be the first time the $3 billion agency
has so radically and retroactively reduced a proposal in its nearly 10-year
history.
Randy Mills |
In a memo posted early today on the agency’s Web
site, Randy Mills, who joined the agency in May, said,
“While all of the aims of the concept plan are individually laudable, it is my firm belief that the proposal as written is too broad and overly complex to be successful. In a word, it lacks focus. As a result of its overly wide-ranging scope, I also believe that there is a real possibility of incurring significant duplicative costs. Following a thorough review and conversations with senior members of the CIRM team, it is my opinion that the $70 million price tag is not clearly justified in terms of the benefits it will deliver to the people of California.”
The Alpha stem cell clinic program was heavily
promoted by the former president of the stem cell agency, Alan Trounson. It is
aimed at making the Golden State the world’s “go-to” location for stem cell
therapies.
Mills said, however, that a $15 million component
creating a data/information center should be stripped from the plan and whittled down to $10 million.
He said the center’s efforts to combine aspects of
clinical operations, data sharing, education/marketing and lobbying for
insurance coverage of stem cell therapies would create an “unwieldy program.”
Instead, he said the Alpha effort should focus on early stage clinical
operations – “efforts directly related
to high quality stem cell clinical trials.”
Mills’ proposal comes late in the game for the Alpha
awards. Applicants have already submitted their proposals. The review of the
applications was originally scheduled to occur last month. However, in one of
his first public acts, Mills postponed the review, which is now scheduled for September. The reason for the delay, the agency said in June,
was the difficulty in finding reviewers.
However, the information center component is
contained in a separate RFA and could be easily discarded. The only damage
would be the time that the five applicants spent on preparing their
applications, which is no small task, and the work that the CIRM staff has done so
far.
Mills told the board in the memo,
“I realize that making this recommendation after the submission of applications for the CIMC (the data center) is not optimal. However, given the magnitude of the spending proposed, not sharing this critical assessment with the board would be a greater disservice. I believe that these recommendations are not only fiscally responsible, but more importantly, give the program the greatest chance of success to accomplish our mission of getting stem cell treatments to patients in need.”
Mills is also recommending alterations in the
clinical portion of the Alpha plan, which involves eight separate applications, all of which are likely to have come from institutions with representation on the stem cell board. Mills said he wanted “a staged approach to funding, where we evaluate
with proper metrics, the effectiveness of the program in a limited number of
sites before expanding.”
Mills’ Alpha memo does not appear to envision
modifying the RFA, so presumably the staged funding would be dealt with in
negotiations following board action on the applications, which will probably
occur later this year. Up to five awards
are scheduled to be made.
Mills’ Alpha cuts are virtually certain to be
approved by the board. Rejection of his recommendations would amount to a vote
of no-confidence in Mills and would likely result in his resignation. (On Thursday, however, he assured the board he would not quit if it did not go along.)
Even earlier this year, Mills, who has made his
career in business, demonstrated his parsimony. Trounson’s proposed operations 2014-15
budget for the agency was submitted in early May at $17.9 million, an increase
of 9.5 percent increase over estimated 2013-14 spending. Mills sliced the
spending plan to $17.3 million, maintaining it at virtually same level as last
year – lower if inflation is factored in.
(An earlier version of this item incorrectly stated that Mills wanted to cut the program by $15 million.)
(An earlier version of this item incorrectly stated that Mills wanted to cut the program by $15 million.)
Labels:
alpha clinics,
cirm future,
ICOC,
mills parsimony
Sunday, July 20, 2014
Creating Critical Mass at a California Stem Cell Consortium
News about the California stem cell agency is
dominated by such matters as $70 million Alpha stem cell rounds and clinical
trials but other significant activities occur below that high level surface.
One such event is the creation of a state-of-the-art
imaging facility at the Sanford Consortium in the San Diego, a facility that
was built with the help of a $43 million award from the stem cell agency.
According to Terri Somers, a spokeswoman for the imaging
facility,
“It is the only commercial location within California where these high-powered imaging modalities are available to researchers under one roof, along with pharmacology expertise and a deep reference library.”
The facility was opened last fall by Molecular
Imaging, Inc., of Ann Arbor, Mich. While it provides services to the stem cell
researchers at the consortium, its services are also available to researchers
throughout the state, both private and academic.
Edward Holmes, president of the Sanford Consortium,
said in a statement,
“We believe that in vivo imaging will play a critical role in tracking the effects of stem cells, and accelerate first-of-their-kind therapies to treat and cure some of the world’s most debilitating diseases.”
The facility includes one of the world’s most
powerful MRIs, a PET, SPECT, CT, ultrasound and other molecular imaging technologies.
It additionally provides as access to hundreds of disease models and a decades-deep
expertise in pharmacology, according to the company.
Somers said,
“These technologies have been developed and leveraged in academic settings. Some global pharmaceutical companies have embraced the technologies as well, creating core-imaging facilities. However, drug companies don’t have this capacity on all their campuses, and none are in Southern California, making access for researchers here problematic.”
With the facility at Sanford, these services are
more readily available to cash-strapped biotech companies on the entire West
Coast.
Backers of the stem cell agency argue that one of
its benefits has been to help build the critical mass in California that is
necessary to support and attract stem cell research. The addition of the
Molecular Imaging Center at the Sanford Consortium appears to be part that
continuum.
Saturday, July 19, 2014
Los Angeles Times: California Stem Cell Agency Rife with Conflicts and Unrealistic Expectations
The Los Angeles Times, California’s largest
circulation newspaper, is carrying a piece this weekend about “cronyism,”
conflicts of interest and “inflated expectations” at the state’s $3 billion
stem cell agency.
The column by Pulitzer Prize-winning writer and author
Michael Hiltzik used this month’s Trounson Affair as a starting point to
dissect the situation at the California Institute for
Regenerative Medicine (CIRM), as the agency is formally known.
Trounson was appointed to the board of StemCells,
Inc., of Newark, Ca., on July 7, just seven days after leaving the
agency. StemCells, Inc., holds a $19.4 million award from the agency. CIRM has ordered a full review of the
situation and barred its staff from communicating about StemCells, Inc.,
matters with Trounson.
But even before Trounson’s appointment, there were
issues involving StemCells, Inc.(See here and here.) Hiltzik said,
“The relationship already reeked of cronyism.”
Hiltzik wrote,
“Trounson's move comes as CIRM must begin looking to the future, but any discussions about extending the agency's life span will have to address the flaws created by Proposition 71 (the ballot initiative that created the agency in 2004). Among them is the program's very structure, and even its scientific goals.”
Hiltzik continued,
“How bad are the conflicts? When the board considered a proposal earlier this year to spend $16 million to attract three star scientists to California, so many members had to recuse themselves that only nine were left to vote. (Six ended up voting in favor.)
“When conflicts of interest are so rife that only one-third of your board can weigh in on a major policy issue, that's tantamount to not having any board at all.”
Some of the issues at the agency have to do with the
ballot campaign that created it in 2004, an election in which California voters
were led to believe that miraculous stem cell therapies were imminent.
Hiltzik wrote,
“Programs like CIRM are always susceptible to inflated expectations.
"”Since Big Science needs great public support it thrives on publicity,’ the physicist Alvin Weinberg, a veteran of the Manhattan Project, wrote in a famous 1961 article in "Science" about the drawbacks of big-money scientific research. He added: ‘The inevitable result is the injection of a journalistic flavor into Big Science which is fundamentally in conflict with the scientific method.... The spectacular rather than the perceptive becomes the scientific standard.’"
Hiltzik acknowledged the contributions that CIRM has
made to stem cell science.
“CIRM-funded labs have produced genuine achievements. But the agency tends to delineate its progress in buildings built, papers published, and big-name scientists lured to California. But the specific cures promised by the Proposition 71 campaign haven't materialized, which doesn't surprise anyone steeped in the realities of the scientific method.”
Hiltzik concluded,
“Even if one believes the need for California to devote $3 billion to a narrow, extremely speculative field of science, the Trounson case and other CIRM administrative missteps have made clear that Proposition 71 created the wrong framework to manage a complex research effort. The initiative left the public with no way to tell if its money has been well spent, and no accountability if it hasn't.
“Moreover, the program deprived potentially more promising research efforts of resources and contributed to the general impoverishment of California's entire higher-education system. If its sponsors have the audacity to ask taxpayers for even more money under the same terms as Proposition 71, the reply should be a resounding ‘no.’ If the voters are gullible enough to repeat the same mistake they made in 2004, there's no cure for them.”
The Hiltzik column appeared online last night. It is
scheduled to appear in the Sunday print edition of the Times, which says it has
a combined print and online reach of 4 million readers.
Friday, July 18, 2014
California-funded Stem Cell Diabetes Treatment Edges Forward
CIRM video
The California stem cell agency’s $39 million investment
in a possible therapy for diabetes moved forward this week with the
announcement that the treatment could enter clinical trials as early as next
month.
ViaCyte, a San Diego, Ca., firm, and the agency announced
yesterday that the firm has applied with the FDA to start the testing to determine whether
the product is safe in human beings. The
Juvenile Diabetes Research Foundation, which is also funding the ViaCyte
product, said the therapy was “potentially transformative.”
Randy Mills, president of the $3 billion stem cell
agency, said in a press release,
“This is good news for ViaCyte and is a clear sign of the progress they are making. Filing for an IND is a crucial step along the path to bringing a stem cell treatment to patients. CIRM will be working with them and supporting them every step of the way to try and make this happen as quickly, and as safely, as possible.”
The agency described the treatment like this:
“Viacyte’s approach uses a thin plastic pouch, containing an immature form of pancreatic cells that, when implanted under the skin, are designed to mature and become insulin-producing and other cells needed to regulate blood glucose levels. These cells are able to sense when blood glucose is high, and then secrete insulin to restore it to a healthy level. In effect this is designed to mimic the glucose regulating functions of the pancreas, which, in people with T1D(type one diabetes), no longer works. This approach was shown to be effective in extensive preclinical testing in models of the disease.”
Paul Laikind, president of ViaCyte, told Bradley
Fikes of the San Diego U-T that if “all goes smoothly” the initial stage of the
clinical trials could begin next month or in September. Fikes also quoted the first chairman of the stem cell
agency, Robert Klein, whose son has diabetes, as saying,
"This is an exciting day for the father of any son or daughter who has Type 1 diabetes."
The therapy, which is based on human embryonic stem cells, could take years to successfully
complete all of the necessary clinical trials. Only one out of 10 possible
therapies that enter clinical trials enters the marketplace.
Labels:
cirm future,
clinical trials,
diabetes,
viacyte,
video
Wednesday, July 16, 2014
StemCells, Inc., Says Patient Endangerment Charges Lack Merit
StemCells, Inc., said late today that charges that
is endangering patients in clinical trials have no merit and that the company’s
primary concern is the safety of its patients.
The Newark, Ca., company was responding to a lawsuit filed earlier this week that alleges the firm is manufacturing human cells in a process that puts “patients at risk of infection or death.”
The Newark, Ca., company was responding to a lawsuit filed earlier this week that alleges the firm is manufacturing human cells in a process that puts “patients at risk of infection or death.”
The suit was filed by Rob Williams, a former senior
manager at the publicly traded company. Williams said he was unlawfully fired
by StemCells, Inc., after bringing the stem cell quality issues to the attention
of senior management.
Earlier today, the California Stem Cell Report asked
the company for comment. Ken Stratton, general counsel, responded. Here is the
text of his reply.
“As you know, StemCells, Inc. is engaged in the research and development of cell-based therapeutics and is currently sponsoring clinical studies of potential therapies for spinal cord injury, AMD and pediatric neurological disorders as well as conducting pre-clinical studies in Alzheimer’s disease. The pre-clinical AD studies, but none of the clinical studies, are partly funded by CIRM.
“The Company has reviewed the complaint filed by Mr. Williams, a former employee whose employment was terminated for performance deficiencies, and finds no merit to the allegations. The Company has retained Littler Mendelson as its litigation counsel and intends to defend itself vigorously in court.
“You should know, the elements of manufacturing practices that concerned Mr. Williams were immediately and carefully reviewed by the Company. The Company’s primary concern has always been, and will continue to be, the safety and tolerability of stem cell transplantation in its clinical trials. Over the years, we have consulted with multiple experts in the field and we believe our processes, procedures and controls, as fully described in our regulatory filings, are appropriate for a company at our early stage of clinical development and comport with applicable guidelines and regulations. To date, no patients participating in the Company’s clinical studies have experienced any product related safety concerns.
“We hope you find this information helpful.”
California Stem Cell Agency Examining Lawsuit Alleging Patient Endangerment and 'False Certification'
The California stem cell agency today
said it is reviewing a lawsuit involving the recipient of a $19.4 million award that alleges the firm is manufacturing human cells in a process
that puts “patients at risk of infection or death.”
The most
serious allegations against StemCells, Inc., of Newark, Ca., involve its clinical trials. The stem cell agency’s $19.4 million research award
to StemCells, Inc., does not involve human beings, only initial development of
a possible therapy for Alzheimer’s over the next three years. Both the clinical
trials and the Alzheimer’s research use the same proprietary cells,
HuCNS-SC.
The company
said in a 2013 press release,
"We know from the preclinical work that our proprietary HuCNS-SC cells survive in the toxic environment of the Alzheimer's disease brain and restore memory under the regulation of the host."
The lawsuit
was filed by a former senior manager, Rob Williams, of the publicly traded
StemCells, Inc. He alleged he was fired after complaining about the cell
problem to senior management.
In addition
to possible injury, the lawsuit said that “the use of adulterated stem cells
lots could skew patient test results, effectively jeopardizing data behind
years of clinical trials and research.”
The lawsuit
said that StemCells, Inc., receives
“…millions of dollars in government funding, including grants from the California Institute for Regenerative Medicine (CIRM). As part of certifications that Defendants made and, on information and belief, continue to make, to the State of California in order to obtain such funding, Defendants represent that their manufacturing processes yield stem cells that are ‘safe for human stem cell transplantation.’ Additionally, in order to secure CIRM funding, Defendants represented and represent that the company follows current Good Manufacturing Practices (cGMPs) promulgated by the U.S. Food and Drug Administration (FDA), a set of standards designed to protect the public from dangers to consumer/patient health and safety. Plaintiffs protected activity, as described above, included efforts to stop, complaints about and refusal to engage in or cover up violations of these standards, and by extension the false certifications submitted to the government, certifications that the company used and uses in order to secure substantial funding.”
The lawsuit
also alleged that StemCells, Inc., violated FDA standards and provided false
information to the FDA, which has not yet responded to queries from the
California Stem Cell Report.
In response
to a query, Kevin McCormack, spokesman for the $3 billion stem cell agency,
said the agency was carefully reviewing the lawsuit.
Williams
attorney, Daniel Velton, has not responded to queries about whether he or
Williams informed the FDA of the issues raised in the lawsuit.
StemCells,
Inc., did not respond to queries about the matter. (Late yesterday, the firm said the allegations have no merit. See full text here.)
The company was founded by Stanford researcher, Irv Weissman, who sits on its board and is chairman of its scientific advisory board. His wife, Ann Tsukamoto, is executive vice president. Last week the stem cell agency announced that it was conducting a full review of its activities with the firm after it appointed the agency's former president, Alan Trounson, to its board seven days after he left the agency.
Here is a
copy of the lawsuit, which was first reported by Elizabeth Warmerdam of
Courthouse News Service.
California Lawsuit Charges StemCells, Inc., with Putting Patients at Risk
A former senior manager at StemCells, Inc., which holds
a $19.4 million award from the California stem cell agency, has filed a lawsuit
alleging that “deficiencies in the company's cell lines put patients at risk of
infection or death during clinical trials.”
The charges were contained in a suit by Rob
Williams in Alameda County court, according to an article on Courthouse News Service written by Elizabeth Warmerdam. Williams is suing for wrongful termination,
retaliation and violation of the California False Claims Act.
According to the article, the complaint said that
StemCells, Inc., of Newark, Ca., says its stem cells are safe for human
transplantation. Warmerdam continued,
“Williams says he was hired as the company's senior manager of manufacturing in December 2013 to oversee its manufacturing facility, where stem cell cultures are cultivated for use in clinical trials.
“'Shortly after beginning his employment, plaintiff noted poor sterile technique, failure to adhere to current Good Manufacturing Practices in the company's manufacturing process, and substantial deficiencies in the company's Manual Aseptic Processing of HuCNS-SC (Human Central Nervous System Stem Cells) cell lines - failure and deficiencies that put patients at risk of infection or death during ongoing clinical trials,’ Williams says.
“Williams claims he also saw manufacturing deviations during cryopreservation of Working Cell Bank lots, leading to numerous stem cell lots with dangerously high numbers of damaged cells.
“'Knowing that these cells were to be injected into human patients, and that the high level of damaged cells and the possibility of contaminating microorganisms could cause serious harm to patients, plaintiff immediately took his concerns to upper management. He also noted that the use of adulterated stem cells lots could skew patient test results, effectively jeopardizing data behind years of clinical trials and research,’ the complaint states."
It was not immediately clear whether Williams’
allegations directly involve the work being funded by the California stem cell
agency(see here, here and here), which has been asked for comment on the lawsuit. The California Stem Cell Report has also asked
the publicly traded company for a comment, although the article said the firm did not respond
to a query by Courthouse News Service.
(Late yesterday, the firm said the allegations have no merit. See full text here.)
(Late yesterday, the firm said the allegations have no merit. See full text here.)
The lawsuit said that Williams was told to conceal
his finding from unspecified reports and that he was suspended shortly thereafter. It
said that he sent emails to upper level management about his concerns and that
he was fired a few weeks later.
Williams’ Linked In profile said that he has 15
years industry experience, including nearly six years as a senior manager at
Alvine Pharmaceuticals and three at Johnson&Johnson.
Williams is seeking unspecified punitive damages
from StemCells, Inc.
Courthouse News Service is a Pasadena-based national news service for lawyers and the news media.
A Nature Post Mortem on the Scripps-USC deal and Faculty Rebellion
The journal Nature took a look this week at the vicissitudes
at The Scripps Research Institute, including its now defunct, $600 million
merger with USC as well as the Scripps faculty uprising.
The July 15 piece was written by Erika Check Hayden, who
reported,
“Scripps faculty members…felt that the (USC) deal sold them short. In interviews, they noted Scripps’ coveted ocean-front location: La Jolla is one of the priciest zip codes in the United States. The $15-million annual payments over 40 years offered by USC would be the equivalent of a $250-million mortgage, they say. That would not even cover one year of the institute’s operating expenses, which were $400 million in 2013.
“’It didn’t make a lot of sense financially,’ (Scripps researcher Martin) Friedlander says ‘You can’t ignore a $20-million deficit, but there are many other creative ways of addressing the financial shortfall. We certainly do not have our backs against the wall.’”The nearly done deal with USC came about because of Scripps’ financial plight. The faculty took umbrage when they learned about it late in the game and called for the removal of President Michael Marletta. The deal then collapsed, and Scripps said it is going to look at unspecified alternatives.
Hayden has interviews with a number of folks, including both from within and without Scripps. She concluded,
“Marletta has said that he is seeking more donations for Scripps, but is disadvantaged by being a relatively recent arrival; he was chair of the chemistry department at the University of California, Berkeley, until 2010.
“’Philanthropy is about long-term relationships with your donors; it’s not something where you just turn the spigot and say, ‘OK, we’ll go out and raise a billion dollars’,’ says Salk president William Brody, who initiated his institute’s fund-raising campaign soon after arriving in 2009.
“Still, Brody and other observers say that Scripps should be able to find a way out of its current dilemma that does not involve dissolution or losing its independence.
“’If they can stick to their knitting and stay the course, they will be successful,’ Brody predicts.”
Labels:
big pharma,
biomedical research,
nih funding,
uscscripps
Tuesday, July 15, 2014
California Stem Cell Agency to Alter $70 Million Alpha Clinic Proposal
The California stem cell agency is moving to revise a
significant component of its $70 million plan to create one-stop Alpha clinics aimed at establishing the Golden State as the leading location worldwide for stem cell
therapies.
Details of the changes that will be considered at the July 24 meeting of the agency’s board are not yet available. However, they deal with
creation of a $15 million data and information management center that would be
involved in clinical trial support, outreach and education and “development of
healthcare economic resources.” The last area would involve efforts to convince
insurance companies and the government to pay for what are likely to be very
expensive treatments.
The Alpha clinic applications were scheduled to be reviewed
last month behind closed doors by the agency’s out-of-state reviewers. However,
the June review was postponed shortly after Randy Mills became the new
president of the agency. The agency said
it was having difficulty getting qualified reviewers. The review is now set for mid-September.
The Alpha clinic proposal has been championed for several
years by the agency’s former president, Alan Trounson, who is now involved in a
conflict-of-interest flap. Last week, he was appointed to the board of StemCells,
Inc., of Newark, Ca., which is the recipient of a $19.4 million award from
CIRM. The agency has launched a review of all activities involving the publicly
traded firm and banned CIRM employees from speaking to Trounson about
StemCells, Inc., matters.
Mills began his presidential duties May 15 at which time Trounson
was designated as a senior scientific advisor. Early in May, Kevin McCormack,
CIRM spokesman, said that Trounson would remain with the agency until June 30. McCormack said that Trounson would “help shepherd through a number of projects and commitments he has made,” which some assumed included the review of the
Alpha applications in June.
McCormack did not response to a question on June 6 about whether
Trounson would be participating in the review.
Eight, unidentified major institutions are competing for the
Alpha clinic awards. Five are specifically shooting for the information
management center. StemCells, Inc., is
not expected to be involved because of the terms of the RFA, but Stanford
University is quite likely to be among the applicants.
Stanford scientist Irv Weissman, founder of StemCells, Inc.,
and currently a member of its board and chairman of its scientific advisory
board, is head of the Palo Alto school’s Institute of Stem Cell Biology and Regenerative Medicine.
The CIRM board meeting will be held in Millbrae but two teleconference
locations, where the public can participate, will be available in Los Angeles and one in La Jolla. Specific locations can be
found on the agenda.
Labels:
alpha clinics,
conflicts,
grantmaking,
revolving door,
trounson affair
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