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, May 29, 2014
Lunch Break for California Stem Cell Directors
LA JOLLA, Ca. -- Directors of the California stem cell agency have adjourned for lunch. Later this afternoon, they are scheduled to approve a $14.3 million award to Asterias Therapeutics for an historic hESC trial.
Researchers Seek to Overturn Negative Decisions by California Stem Cell Agency
LA JOLLA, Ca. -- Three rejected applicants for millions of dollars from the California stem cell agency have appealed negative reviewer decisions in the agency's business-friendly strategic partner program.
The agency's staff rejected two of the appeals but is sending one back to reviewers.
Names of the applicants and the amounts sought were withheld by the agency. The agency also withheld the contents of the appeal letters, material that was, until recently, was routinely disclosed.
The agency's staff rejected two of the appeals but is sending one back to reviewers.
Names of the applicants and the amounts sought were withheld by the agency. The agency also withheld the contents of the appeal letters, material that was, until recently, was routinely disclosed.
California Spending $16.2 Million to Lure Stem Cell Scientists
LA JOLLA, Ca. -- Directors of the California stem cell agency this morning approved $16.2 million to recruit three star researchers to UCLA, UC Berkeley and the Gladstone Institutes in San Francisco.
The move came on a 6-2-1 vote by the 29-member board. Most of the directors were disqualified from voting because of conflicts of interest.
The recipients are Todd McDevitt of the Georgia Institute of Technology, who is being recruited by Gladstone; Xavier Darzacq of the Ecole Normale Superlieure in Paris, who is being sought by UC Berkeley, and John Chute of Duke, who is being recruited by UCLA. Chute is the second researcher from Duke to be lured to California with the help of a CIRM grant.
Appeals by UC San Francisco and UC Davis were rejected by the board.
The vote on the awards followed an unsuccessful effort to scrap the entire recruitment program.
The move came on a 6-2-1 vote by the 29-member board. Most of the directors were disqualified from voting because of conflicts of interest.
The recipients are Todd McDevitt of the Georgia Institute of Technology, who is being recruited by Gladstone; Xavier Darzacq of the Ecole Normale Superlieure in Paris, who is being sought by UC Berkeley, and John Chute of Duke, who is being recruited by UCLA. Chute is the second researcher from Duke to be lured to California with the help of a CIRM grant.
Appeals by UC San Francisco and UC Davis were rejected by the board.
The vote on the awards followed an unsuccessful effort to scrap the entire recruitment program.
California Stem Cell Researcher Recruitment Program Narrowly Survives
LA JOLLA, Ca. -- Directors of the $3 billion California stem cell agency this morning rejected an attempt to reject proposals to spend $16.2 million to recruit star scientists to the Golden State.
The move failed on a 4-5 vote on the 29-member board. Most members of the board were disqualified because of conflicts of interests. The major beneficiaries of the program all have representatives on the CIRM.
Names of the researchers and the recruiting institutions were not disclosed.
The motion was made by Francisco Prieto and seconded by Steve Juelsgaard. Prieto said the grants are "not the best place to for the (agency) to put its money right now."
The discussion of the matter is continuing following the vote.
The move failed on a 4-5 vote on the 29-member board. Most members of the board were disqualified because of conflicts of interests. The major beneficiaries of the program all have representatives on the CIRM.
Names of the researchers and the recruiting institutions were not disclosed.
The motion was made by Francisco Prieto and seconded by Steve Juelsgaard. Prieto said the grants are "not the best place to for the (agency) to put its money right now."
The discussion of the matter is continuing following the vote.
UC San Francisco and UC Davis Seek Millions for Recruitment of Scientists
LA JOLLA, Ca. -- The University of California at Davis and the University of California at San Francisco are both appealing rejection of multimillion dollar proposals that would help them recruit highly regarded scientists to California.
The proposals were turned down by reviewers for the California stem cell agency. However, appeal letters were submitted to the board by Arnold Kriegstein, director of the stem cell program at UC San Francisco, and Thomas Vail, chairman of the Department of Orthopedic Surgery at the same institution.
The appeal letter from UC Davis was signed by Frederick Meyers, vice dean of its medical school; Jan Nolta, head of the school's stem cell program, and Diana Farmer, chair of the Department of Surgery.
The letters were not available online at the time of this writing.
The proposals were turned down by reviewers for the California stem cell agency. However, appeal letters were submitted to the board by Arnold Kriegstein, director of the stem cell program at UC San Francisco, and Thomas Vail, chairman of the Department of Orthopedic Surgery at the same institution.
The appeal letter from UC Davis was signed by Frederick Meyers, vice dean of its medical school; Jan Nolta, head of the school's stem cell program, and Diana Farmer, chair of the Department of Surgery.
The letters were not available online at the time of this writing.
Samuelson Resigns from California Stem Cell Agency Board
LA JOLLA, Ca. -- Longtime CIRM Director Joan Samuelson has resigned from her position on the governing board of the $3 billion California stem cell agency, it was announced this morning.
Samuelson has served on the board since its inception in 2004 and has been a strong advocate for potential treatments for Parkinson's Disease. Samuelson also has Parkinson's Disease and has not been able to attend a number of recent meetings.
The vacancy will be filled by State Controller John Chiang, who is soliciting applications and recommendations.
Samuelson has served on the board since its inception in 2004 and has been a strong advocate for potential treatments for Parkinson's Disease. Samuelson also has Parkinson's Disease and has not been able to attend a number of recent meetings.
The vacancy will be filled by State Controller John Chiang, who is soliciting applications and recommendations.
California Stem Cell Directors Open Meeting This Moning
LA JOLLA, Ca. -- Directors of the $3 billion California stem cell agency opened their meeting today at 9:09 a.m. PDT. It is the first meeting for Randy Mills, the new president of the research program. Alan Trounson, his predecessor, was not at the meeting when it began although he is still an advisor to the agency.
CIRM Chairman Jonathan Thomas welcomed Mills, declaring that he was "passionate about patients."
Wednesday, May 28, 2014
Check This Web Site Tomorrow for Results of the California Stem Cell Agency Meeting
The California Stem Cell Report will
provide live, gavel-to-gavel coverage of tomorrow's session of the
governing board of the $3 billion California stem cell agency from
the meeting location in La Jolla, Ca.
On the agenda is a $14.3 million award
to Asterias Therapeutics to continue the hESC clinical trial begun by
Geron. Directors are scheduled to receive an update on CIRM finances.
The agency is scheduled to run out of money for new awards in 2017
and is attempting to find new sources of funding.
For those who want to listen to the
daylong meeting on the Internet, instructions can be found on the meeting agenda. A live teleconference location is also available in
San Francisco. The address can be found also on the agenda.
Tuesday, May 27, 2014
Results Heralded in Asterias hESC Clinical Trial
Two prominent stem cell patient
advocates are hailing as a “giant leap forward” the initial
results of a clinical trial involving a human embryonic stem cell
therapy for spinal cord injuries.
Their remarks came in the wake of last week's announcement by Asterias Therapeutics of Menlo Park, Ca., that
the trial had cleared its first safety hurdle. Asterias picked up
the trial from Geron, which began it in 2010. Asterias and its
parent, BioTime of Alameda, Ca., purchased Geron's stem cell assets
after Geron abandoned the trial in 2011.
Asked for comment on the Asterias announcement,
Roman Reed of Fremont, Ca., who was honored for his advocacy work in
2013 by the Genetics Policy Institute, said,
“Having started this groundbreaking research with Roman's Law funding of pioneer Dr. Hans Keirstead, I am extremely excited about the possibility of a paralysis treatment.
“My goal is paralysis cure. This research brings that dream closer to fruition for all of my paralyzed brethren. Onward & Upwards!”
![]() |
| Then California Gov. Schwarzenegger (left) with Don Reed (center) and Roman Reed at 2007 stem cell agency press conference AP photo |
A
major moment came, the elder Reed said “with the famous paralyzed
'rats-that-walked-again'” feature on '60 Minutes,' which made
Christopher Reeve say, 'Oh, to be a rat today!' Today, the dream
he embodied is a giant leap closer.”
Reeve was an actor who was famous for
his screen portrayal of Superman and was paralyzed as the result of a horse-riding accident. He died Oct. 10, 2004, about one
month before the California voters created the state's $3 billion
stem cell agency.
Roman Reed, who is a candidate for the California state Senate in next week's election, is paralyzed as the
result of a football injury to his spine in 1994. He coined
the California stem cell agency's motto, “Turning stem cells into cures.”
Asterias is expected to receive a $14.3 million award from the California stem cell agency on Thursday to
help continue the trial.
Labels:
asterias,
clinical trials,
don reed,
romanreed
Asterias-Geron hESC Trial and Funding Reported in The Scientist
The Scientist magazine today picked up
on the item on the California Stem Cell Report that said that Asterias
Therapeutics would receive $14.3 million to continue the human
embryonic stem cell trial that was abandoned by Geron.
The Scientist article by Jef Akst was headlined,
“Geron
hESC Trial to Resume? Nearly
three years after Geron shuttered its stem cell program, BioTime
receives funding to relaunch a Phase 1 trial for spinal cord injury.”
The brief piece largely cited
information contained in our May 22 item. Akst also said that
Asterias, a subsidiary of Biotime, and the California stem cell
agency, which is expected to provide the cash, declined to comment.
Asterias also has reported successful results from the first phase of the trial.
Where California's Stem Cell Spending is Going: Less Than a Half-Billion Left
| Breakdown of projected spending by the California stem cell agency CIRM graphic |
California's stem cell research effort
appears to be down to its last $468 million after nearly 10 years and
630 awards to scientists and their institutions.
Nearly half-a-billion dollars sounds like a lot, but when grant rounds hit $70 million, as is expected in
the Alpha stem cell clinic round later this summer, the money
vanishes quickly.
A caveat does exist on the $468 million
figure. It does not include $409 million for award rounds approved
only in concept by the stem cell agency's governing board. The agency has not yet solicited
applications for those rounds. Its governing board, at any time,
could cancel those proposed rounds.
In 2004, the agency started with $3
billion but really did not get rolling until 2007. Funds for new
awards are scheduled to run out sometime in 2017. The agency has been
working on plans for future financing, which may be discussed at
Thursday's governing board meeting in San Diego. At least the agenda
contains an item called “finance update.”
The latest figures on the agency's
research spending are contained in a presentation for the meeting by
Patricia Olson, executive director of scientific activities and one
of the veterans of the agency.
Under current funding plans, the
presentation shows that 38 percent of the agency's research funding
will go for “development and clinical trials” compared to 15
percent for basic research. Development and clinical trials is
defined as “preparation for and conduct of clinical testing of stem
cell-based therapeutic candidates, safety and proof- of–concept in
humans” and “infrastructure for clinical applications of cell
therapies.”
Readers should be aware of some
additional caveats on the charts in the presentation. They show
$1.869 billion awarded by the agency. However, the CIRM Web site shows $1.784 billion awarded, an $85 million difference.
A presentation chart labeled “future
funding” shows $467.9 million potentially available, meaning that
no RFAs have been issued in those areas. But another chart apparently
based on board decisions last December and called 'future funding
approved” shows the same figures plus additional sums for a total
of $664.8 million. We are querying the agency concerning the figures.
A separate question late last week
generated another perspective on research spending in relation to
administrative and other costs. Kevin McCormack, senior director for
public communications, replied in an email that the agency is expected to spend
$2.746 billion for research and facilities (buildings and
laboratories) during its current projected lifetime. He said $180
million is allotted for operational and grant administration
expenses. Legal expenses are expected to run at $26.9 million with
$12.5 million having been spent through April of this year.
By law, the agency can spend only 6
percent of the amount of the awards for administrative expenses.
Legal costs have no cap.
Another $47.1 million has been budgeted
for “capitalized interest,” although total interest costs are
expected to be in the neighborhood of $3 billion or so. That will be
paid by through the state budget. The agency operates on money
borrowed by the state (bonds). The interest costs are associated with
the bonds.
Monday, May 26, 2014
$37 Million Slated for California Stem Cell Research
Directors of the California stem cell
agency this week are expected to hand out $37 million to businesses
and scientists for projects involving human embryonic stem
cells(hESC), spinal injury and urinary incontinence.
About $20 million will go to
enterprises involved in later stage research. A $14.3 million award
is expected to go to Asterias Therapeutics of Menlo Park, Ca., which
has purchased the stem cell program that originated with Geron Corp.
Asterias plans to move forward with the hESC clinical trial that
Geron began amid much ballyhoo. However, Geron abandoned the effort
in 2011 for financial reasons. (See here and here.)
A $5.6 million award is expected to go
a previous but unidentified grant recipient for work involving
HIV/AIDS. (Late today, an anonymous reader said in a comment at the end of this item that the award is for Sangamo Biosciences in Richmond, Ca.)
At their meeting Thursday in San Diego, directors of the agency are also
expected to approve $16.2 million to help recruit three out-of-state
scientists. The academic recruitment program was created to help
California lure star scientists to the Golden State. The awards range
from $6.4 million to $4.6 million. Two additional applicants were
classified as by reviewers as additional possibilities for funding
depending on the druthers of the board. The names of the applicants
are being withheld by the
agency
The recruitment program was originally
budgeted some years ago for $44 million. Twenty-three million dollars was added in
2013. Prior to this week, the awards helped to finance the recruitment of seven scientists.
Information about the $900,000 urinary
incontinence award can be found here.
Next Action on Stem Cell Agency Budget on June 5
Final decisions on the proposed, $18 million
operational budget for the California stem cell agency has been
delayed until some time in June in order to give the agency 's new
president, Randy Mills, time to put his mark on it.
The governing board's Finance
Subcommittee has scheduled a meeting for June 5 to re-examine the
plan. The panel took a first look at it earlier this month. Following
action by the subcommittee, the spending plan will go to the full
board. No date has been set for that session.
Details of the new version of the
budget are not publicly available. When they are posted on the agenda
for the June 5 meeting, they may give some sense of Mills' priorities.
Friday, May 23, 2014
Correction
The "major step forward" item earlier today incorrectly identified the Asterias vice president as Mary Lebkowski. Her correct first name is Jane.
Major Step Forward on hESC Stem Cell Treatment for Spinal Cord Injury
The “first-in-man” clinical trial
of a human embryonic stem cell therapy has cleared its first safety
hurdle, Asterias Biotherapeutics, Inc., reported yesterday.
The Menlo Park, Ca., firm said the
spinal cord injury treatment caused “no serious adverse” events
in its phase one safety trial. The therapy was originally developed
by Geron Corp., which abandoned the trial in 2011 and sold its stem
cell business to Asterias, which is a subsidiary of Biotime, Inc., of
Alameda, Ca.
Asterias also said in a press release,
“In four of the five subjects, serial MRI scans performed throughout the 2-3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration. This effect was seen in the animal model testing of AST-OPC1.”
![]() |
| Jane Lebkowski Asterias photo |
Jane Lebkowski, president of research
and development at Asterias, said,
“The safety demonstrated in this trial positions Asterias to start a new phase 1/2a clinical trial in 2014, subject to clearance from the FDA .”
The California stem cell agency appears ready to award $14.3 million to Asterias to assist in that new
clinical trial, as reported yesterday by the California Stem Cell
Report. The agency also had loaned Geron $25 million for its clinical
trial. The money has since been repaid.
(Asked to clarify the genealogy of the Asterias clinical trial, Katy Spink, vice president of Asterias, said in an email,
"AST-OPC1 is the former GRNOPC1. The results presented by Jane Lebkowski at ASGCT and referred to in (the) press release are from the clinical trial that was started by Geron in 2010. Slides from that presentation are on our website. We are unfortunately not at liberty to comment on the question of potential CIRM funding, or on details of our future development plans beyond what is described on our website and in our SEC filings.")
Stephen McKenna, director of the
Rehabilitation Trauma Center at the Santa Clara Valley Medical
Center, said,
“Spinal cord injury represents a tremendous unmet medical need that not only results in severe disability, but can also significantly shorten the projected lifespan of affected individuals. There are no approved therapies that can repair spinal cord injuries.”
Asterias said 12,000 persons suffer a
spinal cord injury each year in the United States and about 1.3
million Americans are estimated to be living with a spinal cord
injury.
(An earlier version of this item incorrectly said that Asterias announced the results in a press release today. The press release was dated yesterday. An earlier version also carried an incorrect first name for Jane Lebkowski. The material about the genealogy of the treatment was added shortly after the original version of this item was posted.)
(An earlier version of this item incorrectly said that Asterias announced the results in a press release today. The press release was dated yesterday. An earlier version also carried an incorrect first name for Jane Lebkowski. The material about the genealogy of the treatment was added shortly after the original version of this item was posted.)
Thursday, May 22, 2014
California Stem Cell Agency Expected to Pump $14 Million into Revival of hESC Clinical Trial Dropped by Geron
Geron's abandoned clinical trial for a
spinal cord therapy based on human embryonic stem cells appears to be
back on again but at a different company and with $14.3 million in
help from the California stem cell agency.
An eagle-eyed anonymous reader of the
California Stem Cell Report pointed out the upcoming award to
Asterias Biotherapeutics/Biotime, which purchased the stem cell
operations of Geron Corp. Geron ditched the trial in November 2011
for what it said were financial reasons.
Here is what the reader said in a
comment posted on the “Trounson" item that appeared recently on this blog.
“The Strategic Partnership III Award reviews are in: Asterias Biotherapeutics / BioTime will get $14 million to restart spinal cord injury trial with GRNOPC1.”
The reference is to an item on the agenda for the May 29 meeting of the governing board of the $3
billion agency.
The exact title of the application is
“A Phase I/IIa Dose Escalation Safety Study of [REDACTED] in
Patients with Cervical Sensorimotor Complete Spinal Cord Injury.”
The information provided by the
anonymous reader goes beyond what is currently available on the CIRM
Web site, and he or she seems to have special knowledge of the nature
of the application. That includes the names of the applicants,
normally kept secret by CIRM, and the nature of the material involved
in the proposal, GRNOPC1.
The staff recommendation is virtually
certain to be approved by the CIRM governing board, which rarely
rejects such positions.
Another $9.8 million proposal for a
spinal cord therapy project was rejected by the staff. The
applicants, however, may make an appeal directly to the board on May
29.
CIRM was also involved with the original Geron
proposal, which was the first clinical trial for a therapy based on
human embryonic stem cells. The stem cell agency loaned the company $25 million only
three months before Geron bailed out on the project. It was an
unpleasant surprise for the agency and shock to many patients. Geron
paid back the loan with interest.
Asterias was headed by Tom Okarma, once
the CEO of Geron, and BioTime is headed by Michael West, who founded
Geron many years ago.
Our thanks to the anonymous reader for pointing out the information on the award.
(The day following publication of this item, Asterias reported clearing the first safety hurdle on the trial.)
(Okarma left Asterias in April. An earlier version of this item said Okarma was still head of the company.)
Our thanks to the anonymous reader for pointing out the information on the award.
(The day following publication of this item, Asterias reported clearing the first safety hurdle on the trial.)
(Okarma left Asterias in April. An earlier version of this item said Okarma was still head of the company.)
Labels:
asterias,
biotime,
clinical trials,
geron,
hesc
Tuesday, May 20, 2014
Art Caplan on Fake Stem Cell Research: 'Off-the-Rails Syndrome?'
One of the more prominent bioethicists
in the nation, Art Caplan, is asking,
“Why so Much Fake, Unduplicable Stem Cell Research?”
Caplan weighed in on Medscape.com today, citing the STAP flap involving Japanese and American
researchers as well as a scandal involving a false but widely
accepted – for a time -- claim that a Korean scientist had cloned
human embryos.
Caplan said,
“Stem cell research seems again and again to go off the rails when it comes to the ethics of research.”
He continued,
“I think there are a couple of reasons why this particular area has gotten itself in so much hot water. One is that there is a relative shortage of funding. Because of the controversial nature of cloning -- getting stem cells from human embryos -- some avenues of funding have dried up, and it puts pressure on people to come up with other ways to try to make human stem cells. With less funding, there is more pressure. Sometimes people cut corners. I think that can lead to trouble.
“Another problem in the stem cell field is that if you can come up with a way to produce human stem cells without sacrificing or cloning embryos from humans, you are going to find yourself being a hero to the world. That is what happened in Japan.”
Caplan said,
“There is a lesson here: Until somebody replicates and until somebody can show that they can also do what has been alleged, there isn't a breakthrough. There is only confirmation and then breakthroughs. I think we have to be a lot more careful -- both in science and in media coverage -- before we start saying, 'Aha -- here is a single study, a single report, a presentation. Now we have shown that something can be done.'”
Caplan said,
“Another major problem in the stem cell field is that the number of people doing research in this area has shrunk. It is obviously of keen interest to come up with regenerative medicine solutions to all kinds of healthcare problems. I think a lot of post-docs and graduate students are saying, "I am not sure that I want to set my career track into a field that is sometimes controversial and where funding may be dipping." That may mean that there are fewer people to watch one another. It is not a big field, so maybe part of the reason that it keeps getting in trouble is less ability to do peer review.”
Our thoughts: There is no doubt some
spectacular fraud has surfaced in stem cell research. But the problem
of replication within stem cell research may not be entirely out of
line with problems elsewhere in science. Los Angeles Times columnist
Michael Hiltzik wrote last fall about a study by Amgen that examined
53 “landmark papers” in cancer research and blood biology. Only
five could be proved valid, a shocking result, according to Amgen.
Similar results were turned up by Bayer in Germany, Hiltzik said.
Additionally, as Caplan notes, the stem
cell community is small and the hype is grand. With stupendous
expectations come the likelihood that every missed shot –
fraudulent or not – becomes an equally stupendous failure that is
colored by suspicions of scientific misdeeds. Plus there are truly
bogus practitioners at work, charging desperate people large sums for
untested and potentially harmful treatments.
The mavens of science, those who
control the medical schools and set the standards, may have some
responsibility for some of the problems with replication. Writing May 8 on the blog of the Global Biological Standards Institute, scientist
Beth Schachter touched on this in a post headlined,
“What are science trainees learning about reproducibility?”
She asked one Ph.D. student at
UC San Francisco what sort of training are he and his fellow students receiving on reproducibility and standards. The response?
“None.”
Labels:
ethics,
fraud,
scientific culture,
stem cell hype
'Immortal' Stem Cells and Informed Consent in California
The California stem cell agency is
looking for some help in connection with a $32 million stem cell banking program to be sure that people who donate tissue understand
that their cells will be transformed and made “immortal.”
The agency has issued a $150,000 request for proposals (RFP) for a 12-month study dealing with
informed consent matters in the banking effort. The agency's RFP
said it is “a unique program because it involves the creation of
9,000 iPS cell lines, from 3,000 individuals” that could increase
to 5,000.
The RFP requests a report that will
provide “in-depth analysis of donors' current understanding with
respect to the informed consent process” and “recommendations for
education materials that may improve program performance over time.”
Issues dealing with informed consent and research were highlighted a few years ago in a book, "The Immortal Life of Henrietta Lacks," that was on the New York Times hardcover bestseller list for more than 40 weeks.
The California stem cell agency's RFP said,
“Unique aspects of the tissue collection for disease modeling awards that is critical for donors to understand include that researchers intend to:
“• Test donors blood and skin samples for infectious disease
“• Transform donor cells and make them into immortal iPSC lines
“• Record the genetic sequence of donor their cells
“• Distribute donor cells and associated medical and genetic information widely
“• Use cells and associated information to develop commercial medical products with no financial compensation to donors.
“In addition, minors and/or individuals with cognitive impairments will be recruited for this research and consent may be obtained from their legal guardian."
The proposed California study is linked to a $4 million segment of its $32 million stem cell banking effort approved in
March of 2013. Former CIRM President Alan Trounson said at the time,
“This initiative will provide scientists with access to multiple cell lines that should have much of the genetic variations that represent the variety within any human disease such as Alzheimer’s, heart disease, lung fibrosis and autism.”
The deadline for applications is June
1. Here is a link to a 2010 CIRM report on issues involved in the stem cell banking program, which was only a proposal at the time.
Labels:
bioethics,
ethics,
informed consent,
ips,
stem cell bank
Monday, May 19, 2014
Capricor and California Stem Cell Agency Say All is Well With $27 Million Investment
One recent headline in Forbes
magazine read,
“Stem Cell Therapy To Fix The Heart: A House Of Cards About To Fall?”
Over at the Boston Globe, another story
this month said,
“A provocative new study calls into question the rationale for using stem cells to repair the heart — a much-hyped experimental therapy that grew out of insights from a groundbreaking Boston researcher’s laboratory.”
![]() |
| Eduardo Marban Cedars-Sinai photo |
But it appears that heart stem cell
regeneration is like cheese, you might say. There are many different
varieties. And in the case of a $27 million investment in heart cell regeneration by the California stem cell agency, all the players say
things are just fine with their project.
The state's research effort involves a
publicly traded company called Capricor Therapeutics. The Beverly
Hills firm and one of its founders. Eduardo Marban, director of the
Cedars-Sinai Heart Institute in Los Angeles, received the $27 million
from the state stem cell agency.
In the wake of the news reports
involving heart stem cell regeneration, the California Stem Cell
Report queried both Marban and the stem cell agency. Marban replied
within 37 minutes last week. Here is the text of what he had to say
in his email.
“All of the critiques have been leveled at competing technologies. Indeed, I have been among the more vocal critics (for example see http://www.tctmd.com/show.aspx?id=124193).
“Although some alarmist headlines have inappropriately questioned all cardiac stem cell work, the issues have to do with only one specific subtype of cardiac stem cell, which is marked by the expression of an antigen called c-kit. This is NOT the cell type being developed by Capricor; in fact, my laboratory has found that c-kit-positive cells play no role in Capricor’s cell product. That product, known as CAP-1002, contains a small fraction (less than 5 percent) of c-kit-positive cells which can be completely removed with no loss in potency. CAP-1002 is believed to work by indirect 'paracrine' mechanisms that differ fundamentally from those postulated for the c-kit-positive cells.
“By way of background, the critiques of c-kit-positive heart cells have come in two forms: first, in concerns expressed by the editors of the Lancet regarding data integrity in the SCIPIO trial; this was conducted by Piero Anversa and colleagues using c-kit-positive cells. Second, a recent Nature paper (attached in case you do not have a copy), of which I am an author, questions the claim that c-kit-positive cells are true cardiac progenitors.
“These concerns in no way undermine Capricor’s technology. In fact, CAP-1002 remains the only heart-derived cell product in commercial development. I am proceeding full speed ahead with mechanistic and translational work on CAP-1002, and Capricor continues to feel bullish about the product, which is now in phase 2 trials (the furthest along of any CIRM-funded project).”
Kevin McCormack, a spokesman for the
stem cell agency, also said that Capricor is using a different method
and a different stem cell than the one being called into question.
You can hear the May 15 Capricor
conference call on its first quarter results here. Its stock price
jumped from $6.15 on May 14 to as high as $7.65 during the day May 15. Its 52-week low was $1.15 and its
52-week high $17.15.
(Editor's note: If you would like a copy of the Nature paper that Marban referenced, please send an email to djensen@californiastemcellreport.com.)
Friday, May 16, 2014
Trounson Calls for Closer Ties Between Biotech Industry and Academic Research
Alan Trounson, the departing president
of the California stem cell agency, was in Australia earlier this
month beating the drum for tighter links between science and
industry.
Trounson, a native Australian, said researchers are
“starving” in Australia and did not have much good to say either
about the amount of the federal research funding in the United States.
According to an account on
probonoaustralia, Trounson, who is now working as a senior adviser to
the California agency, declared,
"What is hampering our smartness in converting high quality science discoveries to community benefit? Is Australian science research sustainable without the ability to expand beyond the publicly funded institutional base of universities, medical centers and (the national science agency)? Where is our biotech industry? Who spans the (funding) valley of death? There is a distinct scarcity of industry connecting with the Australian medical research community and a lack of venture investment in the biosciences."
He continued,
“We need to really encourage the very smart academics to become interested in applying their discoveries for the community benefit – universities have to change how they recognize and reward scientists who enter this pathway – not penalize them.
“It isn’t about number of publications or grants... – these are not the measure for making the innovative contributions needed. They probably handicap the conversion to a more effective system that the community will recognize and endorse.
“The current federal grant system is broken in the USA...and would not be any better here. Money is trickling down an unsustainable pipeline that fails to encourage developments of use to the community.
“The system must be fixed despite the inertia that exists even among the majority of scientists. We will have no viable research environment soon if we continue to look away from the problems that are evident and are turning away the smart young scientists.”
Trounson spoke during the 10th
anniversary symposium of veski, an Australian group he helped to
found. It is designed to foster an “innovation economy.”
Trounson's portrayal of Australian
science was not all bleak. He cited major achievements scored by
researchers down under. He said,
“We might have cause to be optimistic about the next 20 years! Maybe even expect Australia to have a minister of science driving high tech developments and a strong biotechnology industry employing scientists and medical graduates maturing in universities and research centers.”
Trounson's last day at CIRM is June 30.
He is returning to his family in Australia.
Subscribe to:
Comments (Atom)


