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Category Archives: Stem Cells
Reproducing Research Results: Removing a Scientific Roadblock
Posted: September 23, 2012 at 3:54 pm
The California stem cell agency faces
no easy task in trying to translate basic research findings into
something that can be used to treat patients and be sold commercially.
Even clinical trials, which only begin
long after the basic research is done and which involve more ordinary
therapeutic treatments than stem cells, fail at an astonishing rate.
Only one out of five that enter the clinical trial gauntlet
successfully finish the second stage, according to industry data
cited last spring by Pat Olson, executive director of scientific activities at the stem cell agency. And
then come even more challenges.
long after the basic research is done and which involve more ordinary
therapeutic treatments than stem cells, fail at an astonishing rate.
Only one out of five that enter the clinical trial gauntlet
successfully finish the second stage, according to industry data
cited last spring by Pat Olson, executive director of scientific activities at the stem cell agency. And
then come even more challenges.
But at a much earlier stage of
research there is the “problem of irreproducible results,” in the
words of writer Monya Baker of the journal Nature. Baker last month reported on
moves by a firm called Science Exchange in Palo Alto, Ca., to
do something to ease the problem and speed up preclinical research.
The effort is called the Reproducibility Initiative and also involves
PLOS and figshare, an open science Internet project.
research there is the “problem of irreproducible results,” in the
words of writer Monya Baker of the journal Nature. Baker last month reported on
moves by a firm called Science Exchange in Palo Alto, Ca., to
do something to ease the problem and speed up preclinical research.
The effort is called the Reproducibility Initiative and also involves
PLOS and figshare, an open science Internet project.
Elizabeth Iorns Science Exchange Photo |
Science Exchange is headed by Elizabeth
Iorns, a scientist and co-founder of the firm. She wrote about test-tube-to-clinic translation issues in a recent article in New
Scientist that was headlined, “Is medical science built on shaky
foundations?”
Iorns, a scientist and co-founder of the firm. She wrote about test-tube-to-clinic translation issues in a recent article in New
Scientist that was headlined, “Is medical science built on shaky
foundations?”
Iorns said,
“One goal of scientific publication
is to share results in enough detail to allow other research teams to
reproduce them and build on them. However, many recent reports have
raised the alarm that a shocking amount of the published literature
in fields ranging from cancer biology to psychology is not
reproducible.”
Iorns cited studies in Nature that
reported that Bayer cannot “replicate about two-thirds of published
studies identifying possible drug targets” and that Amgen failed at
even a higher rate. It could not “replicate 47 of 53 highly
promising results they examined.”
reported that Bayer cannot “replicate about two-thirds of published
studies identifying possible drug targets” and that Amgen failed at
even a higher rate. It could not “replicate 47 of 53 highly
promising results they examined.”
The California Stem Cell Report earlier
this week asked Iorns for her thoughts on the implications for the
California stem cell agency, whose motto is "Turning stem cells into cures." Here is the full text of her response.
this week asked Iorns for her thoughts on the implications for the
California stem cell agency, whose motto is "Turning stem cells into cures." Here is the full text of her response.
“First, I think it is important to
accept that there is a crisis affecting preclinical research. Recent
studies estimate that 70% of preclinical research cannot be
reproduced. This is the research that should form the foundation upon
which new discoveries can be made to enhance health, lengthen life,
and reduce the burdens of illness and disability. The
irreproducibility of preclinical research is a significant impediment
to the achievement of these goals. To solve this problem requires
immediate and concrete action. It is not enough to make
recommendations and issue guidelines to researchers. Funders must act
to ensure they fund researchers to produce high quality reproducible
research. One such way to do so, is to reward, or require,
independent validation of results. The reproducibility initiative
provides a mechanism for independent validation, allowing the
identification of high quality reproducible research. It is vital
that funders act now to address this problem, to prevent the wasted
time and money that is currently spent funding non-reproducible
research and to prevent the erosion of public trust and support for
research.”
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Research suggests promise of cell therapy for bowel disease
Posted: September 19, 2012 at 11:13 pm
Public release date: 19-Sep-2012 [ | E-mail | Share ]
Contact: Karen Richardson krchrdsn@wakehealth.edu 336-716-4453 Wake Forest Baptist Medical Center
WINSTON-SALEM, N.C. Sept. 19, 2012 New research shows that a special population of stem cells found in cord blood has the innate ability to migrate to the intestine and contribute to the cell population there, suggesting the cells' potential to treat inflammatory bowel disease (IBD).
"These cells are involved in the formation of blood vessels and may prove to be a tool for improving the vessel abnormalities found in IBD," said lead author Graca Almeida-Porada, M.D., Ph.D., a professor at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine. The research is published in the current print issue of the journal Hepatology.
Up to 1 million Americans have IBD, which is characterized by frequent diarrhea and abdominal pain. IBD actually refers to two conditions ulcerative colitis and Crohn's disease in which the intestines become red and swollen and develop ulcers. With IBD, blood vessels in the intestine leak and contribute to inflammation.
While there is currently no cure for IBD, there are drug therapies aimed at reducing inflammation and preventing the immune response. However, these therapies aren't always effective. The long-term aim of the research is to develop an injectable cell therapy to induce tissue recovery.
The work, performed while Almeida-Porada was at the University of Nevada, also involved colleagues from Indiana University School of Medicine. The researchers studied a special population of cells, known as endothelial colony-forming cells, found in cord blood, bone marrow and circulating blood. The finding in 1997 that the cells can contribute to blood vessel formation in adults, not just embryos, initiated the notion of using them for therapy. Studies in humans have validated the ability of these cells to improve reduced blood flow to the limbs and to treat heart diseases.
However, there have been few studies to explore the inherent biologic ability of these cells to home to different organs and contribute to tissue-specific cell populations. Evaluating their potential to migrate to the intestine was an obvious choice, said Almeida-Porada, because dysfunctional blood vessels are a hallmark of IBD. Not only are circulating levels of vessel-forming cells reduced in patients with IBD, but a key factor in IBD progression is the development of abnormal or immature blood vessels, which leads to chronic inflammation.
The cells were injected into fetal sheep at 59 to 65 days gestation. About 11 weeks later, intestinal tissue was analyzed to detect the presence of the human cells. The researchers found that the human cells had migrated to the intestine and contributed significantly to the cell population there.
"This study shows that the cells can migrate to and survive in a healthy intestine and have the potential to support vascular health," said Almeida-Porada. "Our next step will be to determine whether the cells can survive in the 'war' environment of an inflamed intestine."
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Research suggests promise of cell therapy for bowel disease
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America Stem Cell, Inc. Awarded a Phase I STTR to Explore the Therapeutic Potential of Its Platform Technology (ASC …
Posted: September 17, 2012 at 3:15 pm
SAN ANTONIO--(BUSINESS WIRE)--America Stem Cell, Inc. (ASC) today announced that it has been awarded an Advanced Technology Small Business Technology Transfer Research (STTR) grant from the National Heart Lung and Blood Institute at the National Institutes of Health. This grant will be conducted in collaboration with scientists at the Wake Forest Institute of Regenerative Medicine (WFIRM) in Winston-Salem, NC, and will explore the combination of two technologies: ASC-101 developed by America Stem Cell and amniotic fluid-derived stem cells discovered and pioneered by Dr. Shay Soker and colleagues at WFIRM. We will examine the effect of ASC-101-treated amniotic fluid-derived stem cells in an experimental model of compartment syndrome. Compartment syndrome results from a variety of injuries such as fractures, contusions, burns, trauma, post-ischemic swelling and blast injuries such as gunshot wounds. If not addressed quickly, it can lead to considerable loss of muscle tissue. Musculoskeletal disorders are the primary cause of disability in the United States with associated costs of more than $800 billion annually. In addition to civilian injuries, more than 42,000 soldiers have been injured since the beginning of the Iraq and Afghanistan wars: the majority of these injuries were musculoskeletal in nature.
The successful combination of ASC-101 with amniotic fluid-derived stem cells would be directly relevant to improving the treatment of muscle damage that occurs following compartment syndrome as well as multiple other types of injuries.
America Stem Cell has demonstrated that ASC-101 enhances the ability of stem cells to migrate to their target tissue. While most companies are concerned with the type of cells used for cell therapy (i.e. the hardware), America Stem Cell addresses how to get the cells to go where they are needed most (i.e. the software). With this award, America Stem Cell will expand the potential for therapeutic application of ASC-101 with amniotic fluid-derived stem cells. According to Dr. Leonard Miller, the Co-Principal Investigator on the grant, The successful combination of ASC-101 with amniotic fluid-derived stem cells would be directly relevant to improving the treatment of muscle damage that occurs following compartment syndrome as well as multiple other types of injuries.
America Stem Cell, Inc. is a clinical stage company that is in clinical trials at the University of Texas M.D. Anderson Cancer Center for improving clinical outcomes for cancer patients undergoing hematopoietic stem cell transplantation. This award enables America Stem Cell to expand the development of ASC-101 to yet another cell type. Lynnet Koh, CEO of America Stem Cell, noted, The combination of ASC-101 with amniotic fluid-derived stem cells could synergistically enhance the therapeutic and regenerative capacity of these cells and most importantly provide an off-the-shelf, effective solution for tissue damage due to multiple types of injuries or diseases. ASC-101 is a transformative technology with the potential to improve clinical outcomes for patients undergoing a wide variety of cell therapies for the treatment of diseases such as graft versus host disease, diabetic complications, and ischemic diseases such as myocardial infarctions, retinopathy and critical limb ischemia. America Stem Cell has established a number of collaborations examining the potential of ASC-101 to improve cell therapies for multiple clinical conditions using a wide variety of cell types.
About America Stem Cell, Inc.
America Stem Cell is a privately held biotechnology company based in San Antonio, TX, with offices in San Diego, CA, and is dedicated to the development and commercialization of enabling technologies to enhance and expand the therapeutic potential of cell therapies. The key technology platforms (ASC-101 and ASC-102) are designed to improve the homing and engraftment of cells to target organs. ASC-101 is currently in clinical trials to improve the therapeutic potential of hematopoietic stem cells for patients in need of hematopoietic stem cell transplantation. Additionally, these technologies have the potential to enhance the efficacy of cell therapies for the treatment of inflammation from chemotherapy/radiation, autoimmune diseases, and ischemic diseases including myocardial infarction and stroke. America Stem Cell has partnerships and collaborations with Kyowa Hakko Kirin, Spectrum Medical Innvoations, Florida Biologix, and various medical research institutions including the University of Texas M.D. Anderson Cancer Center, Oklahoma Medical Research Foundation, Fred Hutchinson Cancer Center,,University of California San Diego, Sanford-Burnham Institute, Indiana University, Juvenile Diabetes Research Foundation, as well as corporate partnerships. For additional information, please contact Lynnet Koh at 210-410-6427, or view http://www.americastemcell.com.
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Upstate Medical breaking ground today on $15 million cord blood bank
Posted: September 17, 2012 at 3:15 pm
Syracuse, N.Y. -- Luca Vassallo went through five rounds of chemotherapy, localized radiation and multiple surgeries to deal with the aftermath of his cancer treatment, all before he turned 1 year old.
In the end, the procedure that sent him into remission involved an intravenous drip and a strangers donated blood cells.
Its like a transfusion, said Lucas mother, Manal Vassallo, of Camillus, explaining how donated blood from a strangers umbilical cord filled her boys body with healthy cells.
They take a vial and hook it up through the IV and drip the cord blood in, Vassallo said. It was a really peaceful day.
Blood transplants like the one that helped Luca could become much more prevalent throughout New York. Today, Upstate Medical University will break ground on a $15 million, state-owned public cord blood bank, the second of its kind in New York.
The long-delayed Upstate Cord Blood Bank, at the former Community General campus in Onondaga, will store blood donations collected from umbilical cords and placentas, which most often are thrown out after deliveries.
Those materials contain stem cells, building blocks of the immune system that generate new blood and can even change a recipients blood type. Cord blood stem cells are used in the treatment of more than 60 malignant, genetic and acquired blood diseases such as leukemia, lymphoma and sickle cell anemia.
Through the donations of cord blood from families all across our region, we have the ability to save lives through transplantation and further fuel biomedical research that may move us closer in finding breakthroughs for dozens of diseases, said Dr. David Smith, president of Upstate.
The facility is part of a nationwide push to grow cord blood collections and solicit more donations from women of different races and ethnicities to increase the chance for successful transplants.
We want to develop in this country an inventory so that anyone who qualifies, by their disease, could have this available, said Tom Quinn, the senior vice president for health system development at Upstate. Right now, (the pool is) not as genetically diverse as it ought to be.
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Upstate Medical breaking ground today on $15 million cord blood bank
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At the Right Place at the Right Time – New Insights into Muscle Stem Cells
Posted: September 17, 2012 at 3:15 pm
17.09.2012 - (idw) Max-Delbrck-Centrum fr Molekulare Medizin (MDC) Berlin-Buch
Muscles have a pool of stem cells which provides a source for muscle growth and for regeneration of injured muscles. The stem cells must reside in special niches of the muscle for efficient growth and repair. The developmental biologists Dr. Dominique Brhl and Prof. Carmen Birchmeier of the Max Delbrck Center for Molecular Medicine (MDC) Berlin-Buch have elucidated how these stem cells colonize these niches. At the same time, they show that the stem cells weaken when, due to a mutation, they locate outside of the muscle fibers instead of in their stem cell niches (Developmental Cell, http://dx.doi.org/10.1016/j.devcel.2012.07.014)*. Muscle stem cells, also called satellite cells, colonize a niche that is located between the plasma membrane of the muscle cell and the surrounding basal lamina. Already in newborns these niches contain satellite cells from which both muscle cells and new stem cells can be generated.
Weakened stem cells In the present study Dr. Brhl and Professor Birchmeier showed that mouse muscle progenitor cells lacking components of the Notch signaling pathway cannot colonize their niche. Instead the muscle progenitor cells locate in tissue between the muscle fibers. The developmental biologists view this as the cause for the weakening of the muscles. The stem cells that are in the wrong place are no longer as potent as they originally were and hardly contribute to muscle growth.
In addition, the Notch signaling pathway has a second function in muscle development. It prevents the differentiation of stem cells into muscle cells through suppression of the muscle developmental factor MyoD and thus ensures that there will always be a pool of stem cells for muscle repair and regeneration. In the future this work could gain in importance for research on muscle regeneration and muscle weakness.
Contact: Barbara Bachtler Press Department Max Delbrck Center for Molecular Medicine (MDC) Berlin-Buch in the Helmholtz Association Robert-Rssle-Strae 10; 13125 Berlin, Germany Phone: +49 (0) 30 94 06 - 38 96; Fax: +49 (0) 30 94 06 - 38 33 e-mail: presse@mdc-berlin.de http://www.mdc-berlin.de/ function fbs_click() {u=location.href;t=document.title;window.open('http://www.facebook.com/sharer.php?u='+encodeURIComponent(u)+'&t='+encodeURIComponent(t),'sharer','toolbar=0,status=0,width=626,height=436');return false;} html .fb_share_link { padding:2px 0 0 20px; height:16px; background:url(http://static.ak.facebook.com/images/share/facebook_share_icon.gif?6:26981) no-repeat top left; } Share on Facebook
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At the Right Place at the Right Time - New Insights into Muscle Stem Cells
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StemCells, Inc., Discloses How it Will Generate $40 Million in Matching Funds
Posted: September 16, 2012 at 3:56 pm
StemCells, Inc., said yesterday that it
will come up with the $40 million needed to match loans from the California
stem cell agency through “existing infrastructure and overhead”
and will not be issuing stocks or warrants to the agency.
In a statement to shareholders, Martin
McGlynn, CEO of the publicly traded firm, discussed the $40 million in loans awarded by agency this summer, including $20 million last
week. The stem cell agency's governing board, after it emerged from
an executive session on the matter, last Wednesday night adopted a
motion requiring the company to demonstrate that it has the matching
funds.
McGlynn, CEO of the publicly traded firm, discussed the $40 million in loans awarded by agency this summer, including $20 million last
week. The stem cell agency's governing board, after it emerged from
an executive session on the matter, last Wednesday night adopted a
motion requiring the company to demonstrate that it has the matching
funds.
CIRM Chairman J.T. Thomas, a Los Angeles bond financier, said that
concerns were expressed during the executive session that the agency
“would account for such a large part of the assets of the company.”
At his suggestion, the board approved the loan on the condition that
“it show it has access” to the $20 million in matching funds that
company offered during the application process. StemCells, Inc., also
offered a $20 million match on another loan approved in July by CIRM.
concerns were expressed during the executive session that the agency
“would account for such a large part of the assets of the company.”
At his suggestion, the board approved the loan on the condition that
“it show it has access” to the $20 million in matching funds that
company offered during the application process. StemCells, Inc., also
offered a $20 million match on another loan approved in July by CIRM.
The latest financial reports from
StemCells, Inc., which is based in Newark, Ca., show that it had
assets of $17 million as of June 30 and liabilities of $11.6 million.
The company reported net income for the second quarter of $833,522
compared to a loss of $4 million for the same period a year ago.
StemCells, Inc., which is based in Newark, Ca., show that it had
assets of $17 million as of June 30 and liabilities of $11.6 million.
The company reported net income for the second quarter of $833,522
compared to a loss of $4 million for the same period a year ago.
In its filing with the SEC, the company
said,
said,
“We have incurred significant
operating losses since inception. We expect to incur additional
operating losses over the foreseeable future. We have very limited
liquidity and capital resources and must obtain significant
additional capital and other resources in order to provide funding
for our product development efforts....”
In his statement yesterday, McGlynn
said the California stem cell agency had “doubled down” on
StemCells, Inc., in approving the two loans. He said the company is
not concerned about meeting the matching requirements. McGlynn said,
said the California stem cell agency had “doubled down” on
StemCells, Inc., in approving the two loans. He said the company is
not concerned about meeting the matching requirements. McGlynn said,
Martin McGlynn StemCells, Inc., Photo |
“To be clear, we do not interpret the
diligence requirement as an obligation to raise a specific amount of
money in a particular period of time, and we wish to correct the
misstatements made by some uninformed third parties that the ICOC is
requiring us to raise $20 million in matching funds. In
point of fact, we expect that a substantial amount of our
contribution towards these projects will come from existing
infrastructure and overhead, salaries for our existing personnel, and
other contributions in kind. Furthermore, we will soon be
reviewing the budgets for both projects in detail with CIRM
staff. Because each disease team budget was prepared on a
stand-alone basis, we expect to see significant economies and
efficiencies now that the company has in fact been awarded funding
for both.”
McGlynn also said,
"Under this particular CIRM
program (RFA 10-05), funding for companies will be in the form of
unsecured, non-recourse, interest-bearing, term loans, which will be
forgivable in the event the funded research fails to result in a
commercialized product. On the other hand, should the product be
successfully commercialized, CIRM would earn milestone payments
depending on how successful the product becomes. Because CIRM
shares the downside risk, and could participate handsomely on the
upside, the structure makes the loan about as close to 'equity' as one could, without having to dilute existing shareholders in order
to gain access to significant amounts of capital. The company
will not issue stock, warrants or other equity to CIRM in connection
with these awards.
"Of course, we realize that CIRM
prefers that applicants from industry provide evidence of their
ability to secure whatever additional funds may be needed to complete
any CIRM-funded project, in this case the filing of an IND for each
indication. This is stated in the text of RFA 10-05 itself and
was repeated in various comments by CIRM staff during the application
process. When making the second award on September 5, the
ICOC naturally recognized the sizeable commitment it was making
to StemCells, so it instructed CIRM staff to satisfy themselves
of the company's ability to access the capital needed to fund the
project, namely the Alzheimer's program through to the filing of the
IND.”
McGlynn also said firm's bid for
another $10 million from CIRM could come in the form of a grant
instead of a loan. He said,
another $10 million from CIRM could come in the form of a grant
instead of a loan. He said,
"Finally, I can confirm that in
June of this year the Company applied for up to $10
million under CIRM's Strategic Partnership I program
(RFA 12-05). Unlike the disease team awards under RFA
10-05, if companies are approved for funding under RFA 12-05, they
may elect to take such funding in the form of a grant, not a
loan. Our application under RFA 12-05 is for a controlled Phase
II clinical trial of HuCNS-SC cells in Pelizaeus-Merzbacher disease
(PMD), a rare myelination disorder. StemCells completed a Phase
I study in PMD in February 2012 and in April announced that
all of the patients from that study showed evidence of cell-derived
myelination and three of the four patients in the study showed
measurable gains in motor and/or cognitive function.”
According to CIRM, the awards in the strategic partner round will be approved either next month or in December.
StemCells, Inc. stock was trading at
$1.85 at the time of this writing. Last week, it rose to $2.43.
During the last 12 months, its high was $2.67 and its low was 59
cents.
$1.85 at the time of this writing. Last week, it rose to $2.43.
During the last 12 months, its high was $2.67 and its low was 59
cents.
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Road to Commercialization: California Stem Cell Agency Seeking Top Level Product Development Execs
Posted: September 16, 2012 at 3:56 pm
Looking for a good job at an
enterprise that is on the cutting edge of biotechnology?
Nine positions are open at the $3
billion California stem cell agency, headquartered in San Francisco.
Some of them could pay more than $200,000 a year.
billion California stem cell agency, headquartered in San Francisco.
Some of them could pay more than $200,000 a year.
Several of the new jobs are closely
aligned with the agency's fresh focus on commercializing stem cell
research and driving therapies into the clinic. Scientists and
lawyers are being recruited along with a business development
officer. For some of the positions, travel is required.
aligned with the agency's fresh focus on commercializing stem cell
research and driving therapies into the clinic. Scientists and
lawyers are being recruited along with a business development
officer. For some of the positions, travel is required.
One new, high-level position is
described as a senior development officer. The job posting calls for
“expertise in product development for stem cell therapies.” The
person would “directly interact with investigators on CIRM’s
clinically applicable research programs to help provide product
development guidance from preclinical, manufacturing, and first in
human to early phase clinical regulatory perspectives.” An M.D. or
Ph.D. degree in a biomedical science is required. Pay tops out at
$232,891. This person would report to Ellen Feigal, senior vice
president for research and development.
described as a senior development officer. The job posting calls for
“expertise in product development for stem cell therapies.” The
person would “directly interact with investigators on CIRM’s
clinically applicable research programs to help provide product
development guidance from preclinical, manufacturing, and first in
human to early phase clinical regulatory perspectives.” An M.D. or
Ph.D. degree in a biomedical science is required. Pay tops out at
$232,891. This person would report to Ellen Feigal, senior vice
president for research and development.
A second, high-level position reporting to Feigal is senior medical officer, who would manage the
agency's portfolio aimed at commercialization of stem cell research,
specifically “focused on IND enabling and clinical development
projects.” This also requires an M.D. or Ph.D. and substantial professional experience in development of biomedical research and
products. Pay also could run as high $232.891 annually.
agency's portfolio aimed at commercialization of stem cell research,
specifically “focused on IND enabling and clinical development
projects.” This also requires an M.D. or Ph.D. and substantial professional experience in development of biomedical research and
products. Pay also could run as high $232.891 annually.
A third new job at CIRM is
business development officer. That person would help generate
“outside investment in stem cell research in California for both
CIRM-funded and not currently CIRM-funded programs by
biopharmaceutical strategic partners; equity investors (venture
capital and others); and disease foundations.” The salary range
hits $216,270 annually. It wouldn't be surprising if the person in
this job also became involved in developing a funding mechanism for
CIRM after it runs out of state cash in 2017 or so.
business development officer. That person would help generate
“outside investment in stem cell research in California for both
CIRM-funded and not currently CIRM-funded programs by
biopharmaceutical strategic partners; equity investors (venture
capital and others); and disease foundations.” The salary range
hits $216,270 annually. It wouldn't be surprising if the person in
this job also became involved in developing a funding mechanism for
CIRM after it runs out of state cash in 2017 or so.
This position reports to Elona Baum,
general counsel and vice president, business development.
general counsel and vice president, business development.
And yet another new position is called
director of alliance management. The job deals with the agency's
extensive collaborative funding partnerships, many of which are
abroad. CIRM wants somebody with a law degree, experience in
intellectual property and business law along with strong negotiating
skills. The pay range for the post tops out at $232,891 annually. This position reports to CIRM President
Alan Trounson.
director of alliance management. The job deals with the agency's
extensive collaborative funding partnerships, many of which are
abroad. CIRM wants somebody with a law degree, experience in
intellectual property and business law along with strong negotiating
skills. The pay range for the post tops out at $232,891 annually. This position reports to CIRM President
Alan Trounson.
Other open positions include: deputy
general counsel, two science officers and office manager.
general counsel, two science officers and office manager.
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California Stem Cell Firsts: From Emotional Appeals to $40 Million Awards
Posted: September 16, 2012 at 3:56 pm
During the last few months, the $3 billion California stem cell
agency, which is approaching its eight-year anniversary, has chalked up a
number of important firsts.
Most of them came during the July and
September meetings of its 29-member governing board and were related to strenuous efforts by researchers to win approval of awards of up to
$20 million each. Several firsts involved the agency's former
chairman, Robert Klein, who could be considered the father of the
state's stem cell research effort.
September meetings of its 29-member governing board and were related to strenuous efforts by researchers to win approval of awards of up to
$20 million each. Several firsts involved the agency's former
chairman, Robert Klein, who could be considered the father of the
state's stem cell research effort.
So here is the California Stem Cell
Report's list of firsts at the California Institute of Regenerative
Medicine (as CIRM, the stem cell agency, is formally known) for the
summer of 2012.
Report's list of firsts at the California Institute of Regenerative
Medicine (as CIRM, the stem cell agency, is formally known) for the
summer of 2012.
It was the first time that a single
company – in this case, StemCells, Inc. , of Newark, Ca. –
received two awards in the same round.
company – in this case, StemCells, Inc. , of Newark, Ca. –
received two awards in the same round.
It was the first time any company has
been awarded as much as $40 million. Again, StemCells, Inc.
been awarded as much as $40 million. Again, StemCells, Inc.
It was the first time that Klein has lobbied his former board (see here and here) on
behalf of a particular grant application. That occurred in both July
and September with one of StemCells, Inc.'s application.
behalf of a particular grant application. That occurred in both July
and September with one of StemCells, Inc.'s application.
It was the first time that the board
has approved an application that has been rejected twice by
reviewers, again the StemCells, Inc., proposal backed by Klein.
has approved an application that has been rejected twice by
reviewers, again the StemCells, Inc., proposal backed by Klein.
It was the first time that board has
received such a large outpouring of appeals by rejected applicants.
received such a large outpouring of appeals by rejected applicants.
It was the first time that the board
has received such lengthy presentations of emotional appeals by
patient advocates on behalf of rejected applicants.
has received such lengthy presentations of emotional appeals by
patient advocates on behalf of rejected applicants.
It was the first time that action on a
grant round has been extended over three months(see here and here). The disease team
round began in July. Action will not be completed until the end of
October.
grant round has been extended over three months(see here and here). The disease team
round began in July. Action will not be completed until the end of
October.
It was the first time that the
governing board has sent so many applications back for re-review –
five, six if the one to be acted on in October is included.
governing board has sent so many applications back for re-review –
five, six if the one to be acted on in October is included.
It was also the first time that the
board has ordered a full-blown review of its grant appeal process
with an eye to making making major changes in it.
board has ordered a full-blown review of its grant appeal process
with an eye to making making major changes in it.
Several reasons exist for the number of
firsts racked up by CIRM. One is the high stakes involved in the
disease team round that began in July and the low number approved by reviewers – six compared to the 12 approved by the board, as of
today, out of 21 applications. Another reason involves the
increasing understanding on the part of many scientists that they can
appeal directly to the board when reviewers reject their
applications. However, it is also clear that not all applicants
grasp the full range of appeal possibilities. A third reason involves
the agency's muddled appeal process, which has been a problem for
years. And a fourth reason involves the board's push to drive research into
the clinic and commercialization, which applicants are quickly
learning how to exploit.
firsts racked up by CIRM. One is the high stakes involved in the
disease team round that began in July and the low number approved by reviewers – six compared to the 12 approved by the board, as of
today, out of 21 applications. Another reason involves the
increasing understanding on the part of many scientists that they can
appeal directly to the board when reviewers reject their
applications. However, it is also clear that not all applicants
grasp the full range of appeal possibilities. A third reason involves
the agency's muddled appeal process, which has been a problem for
years. And a fourth reason involves the board's push to drive research into
the clinic and commercialization, which applicants are quickly
learning how to exploit.
Readers should feel free to add their
own firsts to this list. They can do so – even, anonymously – by
clicking on the word “comments” at the end of this item.
own firsts to this list. They can do so – even, anonymously – by
clicking on the word “comments” at the end of this item.
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Road to Commercialization: California Stem Cell Agency Seeking Top Level Product Development Execs
Posted: September 16, 2012 at 3:55 pm
Looking for a good job at an
enterprise that is on the cutting edge of biotechnology?
Nine positions are open at the $3
billion California stem cell agency, headquartered in San Francisco.
Some of them could pay more than $200,000 a year.
billion California stem cell agency, headquartered in San Francisco.
Some of them could pay more than $200,000 a year.
Several of the new jobs are closely
aligned with the agency's fresh focus on commercializing stem cell
research and driving therapies into the clinic. Scientists and
lawyers are being recruited along with a business development
officer. For some of the positions, travel is required.
aligned with the agency's fresh focus on commercializing stem cell
research and driving therapies into the clinic. Scientists and
lawyers are being recruited along with a business development
officer. For some of the positions, travel is required.
One new, high-level position is
described as a senior development officer. The job posting calls for
“expertise in product development for stem cell therapies.” The
person would “directly interact with investigators on CIRM’s
clinically applicable research programs to help provide product
development guidance from preclinical, manufacturing, and first in
human to early phase clinical regulatory perspectives.” An M.D. or
Ph.D. degree in a biomedical science is required. Pay tops out at
$232,891. This person would report to Ellen Feigal, senior vice
president for research and development.
described as a senior development officer. The job posting calls for
“expertise in product development for stem cell therapies.” The
person would “directly interact with investigators on CIRM’s
clinically applicable research programs to help provide product
development guidance from preclinical, manufacturing, and first in
human to early phase clinical regulatory perspectives.” An M.D. or
Ph.D. degree in a biomedical science is required. Pay tops out at
$232,891. This person would report to Ellen Feigal, senior vice
president for research and development.
A second, high-level position reporting to Feigal is senior medical officer, who would manage the
agency's portfolio aimed at commercialization of stem cell research,
specifically “focused on IND enabling and clinical development
projects.” This also requires an M.D. or Ph.D. and substantial professional experience in development of biomedical research and
products. Pay also could run as high $232.891 annually.
agency's portfolio aimed at commercialization of stem cell research,
specifically “focused on IND enabling and clinical development
projects.” This also requires an M.D. or Ph.D. and substantial professional experience in development of biomedical research and
products. Pay also could run as high $232.891 annually.
A third new job at CIRM is
business development officer. That person would help generate
“outside investment in stem cell research in California for both
CIRM-funded and not currently CIRM-funded programs by
biopharmaceutical strategic partners; equity investors (venture
capital and others); and disease foundations.” The salary range
hits $216,270 annually. It wouldn't be surprising if the person in
this job also became involved in developing a funding mechanism for
CIRM after it runs out of state cash in 2017 or so.
business development officer. That person would help generate
“outside investment in stem cell research in California for both
CIRM-funded and not currently CIRM-funded programs by
biopharmaceutical strategic partners; equity investors (venture
capital and others); and disease foundations.” The salary range
hits $216,270 annually. It wouldn't be surprising if the person in
this job also became involved in developing a funding mechanism for
CIRM after it runs out of state cash in 2017 or so.
This position reports to Elona Baum,
general counsel and vice president, business development.
general counsel and vice president, business development.
And yet another new position is called
director of alliance management. The job deals with the agency's
extensive collaborative funding partnerships, many of which are
abroad. CIRM wants somebody with a law degree, experience in
intellectual property and business law along with strong negotiating
skills. The pay range for the post tops out at $232,891 annually. This position reports to CIRM President
Alan Trounson.
director of alliance management. The job deals with the agency's
extensive collaborative funding partnerships, many of which are
abroad. CIRM wants somebody with a law degree, experience in
intellectual property and business law along with strong negotiating
skills. The pay range for the post tops out at $232,891 annually. This position reports to CIRM President
Alan Trounson.
Other open positions include: deputy
general counsel, two science officers and office manager.
general counsel, two science officers and office manager.
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StemCells, Inc., Discloses How it Will Generate $40 Million in Matching Funds
Posted: September 16, 2012 at 3:55 pm
StemCells, Inc., said yesterday that it
will come up with the $40 million needed to match loans from the California
stem cell agency through “existing infrastructure and overhead”
and will not be issuing stocks or warrants to the agency.
In a statement to shareholders, Martin
McGlynn, CEO of the publicly traded firm, discussed the $40 million in loans awarded by agency this summer, including $20 million last
week. The stem cell agency's governing board, after it emerged from
an executive session on the matter, last Wednesday night adopted a
motion requiring the company to demonstrate that it has the matching
funds.
McGlynn, CEO of the publicly traded firm, discussed the $40 million in loans awarded by agency this summer, including $20 million last
week. The stem cell agency's governing board, after it emerged from
an executive session on the matter, last Wednesday night adopted a
motion requiring the company to demonstrate that it has the matching
funds.
CIRM Chairman J.T. Thomas, a Los Angeles bond financier, said that
concerns were expressed during the executive session that the agency
“would account for such a large part of the assets of the company.”
At his suggestion, the board approved the loan on the condition that
“it show it has access” to the $20 million in matching funds that
company offered during the application process. StemCells, Inc., also
offered a $20 million match on another loan approved in July by CIRM.
concerns were expressed during the executive session that the agency
“would account for such a large part of the assets of the company.”
At his suggestion, the board approved the loan on the condition that
“it show it has access” to the $20 million in matching funds that
company offered during the application process. StemCells, Inc., also
offered a $20 million match on another loan approved in July by CIRM.
The latest financial reports from
StemCells, Inc., which is based in Newark, Ca., show that it had
assets of $17 million as of June 30 and liabilities of $11.6 million.
The company reported net income for the second quarter of $833,522
compared to a loss of $4 million for the same period a year ago.
StemCells, Inc., which is based in Newark, Ca., show that it had
assets of $17 million as of June 30 and liabilities of $11.6 million.
The company reported net income for the second quarter of $833,522
compared to a loss of $4 million for the same period a year ago.
In its filing with the SEC, the company
said,
said,
“We have incurred significant
operating losses since inception. We expect to incur additional
operating losses over the foreseeable future. We have very limited
liquidity and capital resources and must obtain significant
additional capital and other resources in order to provide funding
for our product development efforts....”
In his statement yesterday, McGlynn
said the California stem cell agency had “doubled down” on
StemCells, Inc., in approving the two loans. He said the company is
not concerned about meeting the matching requirements. McGlynn said,
said the California stem cell agency had “doubled down” on
StemCells, Inc., in approving the two loans. He said the company is
not concerned about meeting the matching requirements. McGlynn said,
Martin McGlynn StemCells, Inc., Photo |
“To be clear, we do not interpret the
diligence requirement as an obligation to raise a specific amount of
money in a particular period of time, and we wish to correct the
misstatements made by some uninformed third parties that the ICOC is
requiring us to raise $20 million in matching funds. In
point of fact, we expect that a substantial amount of our
contribution towards these projects will come from existing
infrastructure and overhead, salaries for our existing personnel, and
other contributions in kind. Furthermore, we will soon be
reviewing the budgets for both projects in detail with CIRM
staff. Because each disease team budget was prepared on a
stand-alone basis, we expect to see significant economies and
efficiencies now that the company has in fact been awarded funding
for both.”
McGlynn also said,
"Under this particular CIRM
program (RFA 10-05), funding for companies will be in the form of
unsecured, non-recourse, interest-bearing, term loans, which will be
forgivable in the event the funded research fails to result in a
commercialized product. On the other hand, should the product be
successfully commercialized, CIRM would earn milestone payments
depending on how successful the product becomes. Because CIRM
shares the downside risk, and could participate handsomely on the
upside, the structure makes the loan about as close to 'equity' as one could, without having to dilute existing shareholders in order
to gain access to significant amounts of capital. The company
will not issue stock, warrants or other equity to CIRM in connection
with these awards.
"Of course, we realize that CIRM
prefers that applicants from industry provide evidence of their
ability to secure whatever additional funds may be needed to complete
any CIRM-funded project, in this case the filing of an IND for each
indication. This is stated in the text of RFA 10-05 itself and
was repeated in various comments by CIRM staff during the application
process. When making the second award on September 5, the
ICOC naturally recognized the sizeable commitment it was making
to StemCells, so it instructed CIRM staff to satisfy themselves
of the company's ability to access the capital needed to fund the
project, namely the Alzheimer's program through to the filing of the
IND.”
McGlynn also said firm's bid for
another $10 million from CIRM could come in the form of a grant
instead of a loan. He said,
another $10 million from CIRM could come in the form of a grant
instead of a loan. He said,
"Finally, I can confirm that in
June of this year the Company applied for up to $10
million under CIRM's Strategic Partnership I program
(RFA 12-05). Unlike the disease team awards under RFA
10-05, if companies are approved for funding under RFA 12-05, they
may elect to take such funding in the form of a grant, not a
loan. Our application under RFA 12-05 is for a controlled Phase
II clinical trial of HuCNS-SC cells in Pelizaeus-Merzbacher disease
(PMD), a rare myelination disorder. StemCells completed a Phase
I study in PMD in February 2012 and in April announced that
all of the patients from that study showed evidence of cell-derived
myelination and three of the four patients in the study showed
measurable gains in motor and/or cognitive function.”
According to CIRM, the awards in the strategic partner round will be approved either next month or in December.
StemCells, Inc. stock was trading at
$1.85 at the time of this writing. Last week, it rose to $2.43.
During the last 12 months, its high was $2.67 and its low was 59
cents.
$1.85 at the time of this writing. Last week, it rose to $2.43.
During the last 12 months, its high was $2.67 and its low was 59
cents.
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