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Category Archives: Stem Cells

California Stem Cell Agency Launches Five-Year Push for Cures

Posted: May 27, 2012 at 3:56 pm


The $3 billion California stem cell
today officially embarked on a course that will mean closer ties to
the biotech industry in hopes of fulfilling the campaign promises to voters to
turn stem cells into cures.

On a unanimous voice vote, directors approved
changes in the seven-year-old agency's strategic plan. The action
will likely mean less money for some activities that enjoyed more cash in the past,  but directors put off action until at least late July.  The plan also sets the course for what may be the last years of life
for the unprecedented state research program. Authorization to borrow
more money (state bonds) for its grants will run out in about 2017.

During a brief discussion of the plan, which has been debated for some months, CIRM Director Jeff Sheehy noted that the agency has now entered "the realm of trade-offs."  Ellen Feigal, CIRM's senior vice president for research and development, told the board that the plan will require hard decisions and sharp focus on priorities. 

Among other things, for first time CIRM overtly set a goal of creating 20 programs that include outside
investment that focus on products. Another five-year goal explicitly calls for financing at least 10 therapies in early-phase
clinical trials, affecting at least five diseases. Overall, the plan seeks to achieve clinical proof-of-concept for stem cell therapies.

In contrast to the Proposition 71
campaign rhetoric, CIRM's strategic plan acknowledges that developing therapies takes a very long time,
often decades.
Two scenarios were presented to the
board for spending the agency's remaining $836 million for grants and
loans. One would allocate $506 million for development research, $195
for translational research and $135 million for basic research, but
nothing for training and "facilities/core resources."
The other scenario calls for $486
million for development research, $160 million for translational
research, $105 for basic research, $60 million for training and $25
million for "facilities/core resources."

The first scenario would mean a $85 million cut in training and shared lab programs – cash that helps to finance researchers and that benefits the many institutions that have representation on the CIRM board.

The board put off action on either scenario after CIRM President Alan Trounson said he wanted more time to prepare a complete analysis of the scenarios. 

The plan also calls for creation of a
platform to enable grantees, disease foundations, venture capitalists
and others to purse CIRM's mission when its state bond funding runs
out. The possibility exists that another bond measure would be submitted to voters. But in either case, CIRM will need a solid record to attract support. 

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CIRM Directors Pleased with Performance Audit Findings

Posted: May 27, 2012 at 3:56 pm


The $3 billion California stem cell
agency received a "very favorable" performance audit report
compared to other government agencies, CIRM directors were told
today.

Representatives of Moss Adams, which
was paid $234,944 by CIRM for the study, made the comments during a
presentation today to the agency's 29 directors. During their
comments, CIRM executives and directors focused on the favorable
aspects of the findings of the six-month study.
CIRM Chairman J.T. Thomas said the
report showed that CIRM is "doing better than being on the right
track." Co-vice chairman Art Torres said,

 "Comparatively we
have done very well."

The report praised the professionalism
of the CIRM staff – "a high caliber group" – and noted
the seven-year-old agency is both "ramping up and ramping down"
at the same time – a reference to the end of state bond funding for
CIRM in 2017.
Prior to the presentation, CIRM
President Alan Trounson said the staff would review the findings and
come up with a plan for the board at its July meeting. The agency is
already implementing some of the recommendations.
The audit was required by a recent
state law that also allowed CIRM to hire more than 50 persons, a cap
imposed by Proposition 71, which created the agency. The audit found a need for improvement in 27 areas and made recommendations. Of the 20 recommendations with the highest priority,
half involved how CIRM manages its information, much of which is
needed for good decision-making. The audit did not assess the
scientific performance of the agency.
The Moss Adams report, performed by the
Seattle firm's San Francisco office, said,

"CIRM board members and senior
management do not receive regularly updated, enterprise-level
performance information. The ability to evaluate performance against
strategic goals is critical to effective leadership and program
monitoring, evaluation, and reporting. CIRM does not currently have a
formal performance reporting program."

In addition to decision-making
information, Moss Adams called for improvements in the agency's
long-troubled grants management system, better grant outcome
tracking, development of a results-based communications plan,
creation of a comprehensive, formal business development plan,
formulation of a comprehensive information technology plan that would
include steps to establish clear responsibility for CIRM's website
and improved monitoring of invention disclosure forms from grantee
institutions.
Last week, in a long overdue move, the
agency hired a director for information technology, who is expected
to solve many of the problems cited in the audit.
State law requires another performance audit in a few years. 

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Live Coverage of Tomorrow's California Stem Cell Meeting

Posted: May 27, 2012 at 3:56 pm


The California Stem Cell Report will
provide live coverage of tomorrow's meeting of the governing board of
the $3 billion California stem cell agency. Directors are expected to
make major decisions about the agency's future direction, hear the results
of the first-ever performance audit and award about $95 million in
grants or loans.

The meeting will be held near the San
Francisco airport with another public teleconference location at UC
San Francisco. Los Angeles will also have two public teleconference
locations. Another will be in La Jolla.
The meeting will be audiocast live on
the Internet, which the California Stem Cell Report will monitor from its
base in Panama near the Pacific entrance to the Panama Canal.
Instructions for listening in on the
audiocast can be found on the agenda along with specific addresses
for the public teleconference locations. The meeting is scheduled to
begin at 9 a.m. PDT.  

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Michael J. Fox Backs Away From Stem Cell Cure for Parkinson's

Posted: May 27, 2012 at 3:56 pm


It was not exactly a case of "Back
to the Future," the hit movie starring Michael J. Fox,
but it did offer a reflection on the past.

It involves the actor's
changing views on stem cell research in connection with Parkinson's
disease, which he has had since the 1990s.
Fox's 2004 Ad
Early on, Fox was well-known for his
support of human embryonic stem cell research. ABC News
recently described him as having become "one of the country’s
most visible advocates for stem cell research." In California, Fox was a prominent promoter of the ballot initiative, Proposition 71,
that created the $3 billion California stem cell agency in 2004. "
He filmed a TV commercial that was
aired widely during the 2004 campaign to create the stem cell agency,
declaring,

 "It could save the life of someone you love."

Today he is considerably less
confident. In an interview last week with ABC, he cited "problems
along the way." Fox said,

“It’s not so much that [stem cell
research has] diminished in its prospects for breakthroughs as much
as it’s the other avenues of research have grown and multiplied and
become as much or more promising. So, an answer may come from stem
cell research but it’s more than likely to come from another area.”

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Third Researcher Appealing Grant Rejection to Stem Cell Agency Board

Posted: May 27, 2012 at 3:56 pm


The director of robotics and biosurgery
at UC Davis is appealing rejection of his application for a
$4.9 million grant from the California stem cell agency.

The scientist, W. Douglas Boyd,
noted that his proposal was given a scientific score of 67, which was
one point below the cutoff for most grants approved by CIRM's
Grants Working Group. Thirteen grants fell in the 68 to 53
range, including Boyd's. Reviewers approved four in that range,
including two with scores of 53.
Boyd's letter was brief, focusing on a
letter of support from an Indiana firm, Cook Biotech, Inc.,
that would supply the "material and technical expertise to
create a new bioengineered cardiac patch
material."
The appeal letter, along with other
appeals(see here and here), will be given to CIRM directors in the agenda material for their meeting tomorrow in San Francisco. The
board does not have to act on the petitions or discuss them.
Researchers can also appear before the board to make a case.

Claire Pomeroy, CEO of the UC
Davis Health Systems, is a member of the CIRM board. She
will be barred from taking part in any discussion of Boyd's
application or voting on it.

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Stem Cell Agency Hires Tech Chief to Solve a Myriad of Problems

Posted: May 27, 2012 at 3:56 pm


In a move that was long overdue, the $3
billion California stem cell agency last week hired a director of
information technology to straighten out key problems ranging from
its grants management system to how it handles its website.

The new hire comes as the CIRM
governing board faces the results of its first-ever performance audit, which is markedly critical of how the agency handles its
information. Half of the audit's 20 highest priority recommendations for improvement focus on information deficiencies, including
critical information necessary for CIRM executives to determine the
agency's performance.
Solving those problems will fall on
the shoulders of Bill Gimbel, who is no stranger to CIRM. He has been
working with the agency as an information technology advisor since
2010 through a contract with Infonetica, Inc., of Pleasanton, Ca., according to CIRM spokesman Kevin McCormack.  Gimbel is now the first staff person in a chief technology position at CIRM since October 2007, when about 25 percent of CIRM employees left.
Bill Gimbel
A graduate of MIT, Gimbel, who will be
paid $180,000 annually, has a broad range of experience in computer
technology and software dating back to 1992. According to his
Linkedin web site, he was most recently director of IT at Infonetica.
He lists himself as owner of aptReader, an app for reference books.
He has also worked for LearningExpress and Scholastic, Inc.
The stem cell agency has been wrestling with information technology issues for years. The critical grants
management system has been an issue at least since 2007, when
directors were told its costs would not exceed $757,000. No figures
for the total spent since then have been made public by CIRM, which
is attempting to build a custom system, but the amount clearly and
easily surpasses the 2007 estimate, based on some of the outside
consulting costs. Although the agency hopes to resolve many of the
problems by the end of this calendar year, the grant system was the
target of considerable attention by Moss Adams, the firm that
prepared the performance audit.
Over the years, CIRM directors have received
intermittent, sketchy CIRM staff reports about the grants management
system, but the Moss Adams discussion is the most
comprehensive.
Among other things, the Moss Adams report said in bureaucratically delicate language,

 "Integration
of website content management has not been an integral part of the
GMS (grants management system) development process, which could
result in suboptimal operational efficiency and effectiveness.

"Grants management system
development is effectively managed at a tactical level, but it lacks
dedicated, strategic governance and oversight, which has resulted in
an elongated development process and requirements conflicts."

Moss Adams said,

"The new grants management system
intellectual property module, currently under development, does not
include provisions to address commercialization activity."

The performance audit additionally
said,

"CIRM board members and senior
management do not receive regularly updated, enterprise-level
performance information. The ability to evaluate performance against
strategic goals is critical to effective leadership and program
monitoring, evaluation, and reporting. CIRM does not currently have a
formal performance reporting program."

Moss Adams continued on other
information technology topics:

"Data and document access are
inefficient as a result of CIRM operating without a document
management system.....In most cases, CIRM staff cannot access
information without human interface. Information is stored in
multiple locations, which are not linked or indexed."

The audit said that the agency has
tried to solve its information problems without a plan. 

 "CIRM’s
information system needs have been met by a variety of tools,
including in-house developed applications, off-the-shelf
applications, databases, and spreadsheets, most of which are not
integrated," the audit stated.

One of the effects of all this is much
wasted time when CIRM's tiny staff tries to extract information from
the hodge-podge of systems. It is time that cannot be spared as the
workload increases in the next few years, as it is certain to do. 
CIRM's 29-member board is scheduled to consider the performance audit at its meeting this Thursday. 

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Anti-psychotic drug kills cancer stem cells without side-effects: study

Posted: May 27, 2012 at 2:13 pm

Vancouver, May 27: A team of Canadian scientists have discovered that thioridazine, a drug used to treat psychotic disorder, could successfully kill cancer stem cells in humans without the toxic side-effects on normal cells.

The research, published Thursday in the science journal CELL, may pave the way for the development of anticancer drugs for treatment of various cancers.

Conventional cancer treatments, like chemotherapy, work in a way that is toxic to cells, which may also lead to side-effects such as hair loss, nausea and anemia, according to the researchers from McMaster University.

Stem cells have long been believed to be the source of many cancers. In 1997, Canadian researchers first identified cancer stem cells in certain types of leukemia. Cancer stem cells have since been identified in blood, breast, brain, lung, gastrointestinal, prostate and ovarian cancer.

"The unusual aspect of our finding is the way it kills cancer stem cells, by differentiating them and changing them into cells that are non-cancerous," said Mick Bhatia, the principal investigator for the study and scientific director of McMaster's Stem Cell and Cancer Research Institute.

"We think this lack of toxicity is why it doesn't have effects on the normal cells, which would be beneficial to the patients," Bhatia said.

Bhatia said their next step was to test thioridazine in clinical trials, focusing on patients with acute myeloid leukemia whose disease has relapsed after chemotherapy. He wants to find out if the drug can put their cancer into remission, and prevent the cancer from coming back by targeting the root of the cancer (cancer stem cells).

Bhatia's team also found that thioridazine works through the dopamine receptor on the surface of the cancer cells in both leukemia and breast cancer patients.

The finding means it may be possible to use thioridazine as a biomarker that would allow early detection and treatment of breast cancer and early signs of leukemia progression, he said.

The research team would also look into the effectiveness of the drug in other types of cancer. Bhatia said they will collaborate with academic groups as well as industry to move forward.

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Anti-psychotic drug kills cancer stem cells without side-effects: study

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Drug destroys human cancer stem cells but not healthy ones

Posted: May 27, 2012 at 2:15 am

ScienceDaily (May 24, 2012) A team of scientists at McMaster University has discovered a drug, thioridazine, successfully kills cancer stem cells in the human while avoiding the toxic side-effects of conventional cancer treatments.

"The unusual aspect of our finding is the way this human-ready drug actually kills cancer stem cells; by changing them into cells that are non-cancerous," said Mick Bhatia, the principal investigator for the study and scientific director of McMaster's Stem Cell and Cancer Research Institute in the Michael G. DeGroote School of Medicine.

Unlike chemotherapy and radiation, thioridazine appears to have no effect on normal stem cells.

The research, published May 24 in the science journal Cell, holds the promise of a new strategy and discovery pipeline for the development of anticancer drugs in the treatment of various cancers. The research team has identified another dozen drugs that have good potential for the same response.

For 15 years, some researchers have believed stem cells are the source of many cancers. In 1997, Canadian researchers first identified cancer stem cells in certain types of leukemia. Cancer stem cells have since been identified in blood, breast, brain, lung, gastrointestinal, prostate and ovarian cancer.

To test more than a dozen different compounds, McMaster researchers pioneered a fully automated robotic system to identify several drugs, including thioridazine.

"Now we can test thousands of compounds, eventually defining a candidate drug that has little effect on normal stem cells but kills the cells that start the tumor," said Bhatia.

The next step is to test thioridazine in clinical trials, focusing on patients with acute myeloid leukemia whose disease has relapsed after chemotherapy. Bhatia wants to find out if the drug can put their cancer into remission, and by targeting the root of the cancer (cancer stem cells) prevent the cancer from coming back. Researchers at McMaster have already designed how these trials would be done.

Bhatia's team found thioridazine works through the dopamine receptor on the surface of the cancer cells in both leukemia and breast cancer patients. This means it may be possible to use it as a biomarker that would allow early detection and treatment of breast cancer and early signs of leukemia progression, he said.

The research team's next step is to investigate the effectiveness of the drug in other types of cancer. In addition, the team will explore several drugs identified along with thioridazine. In the future, thousands of other compounds will be analyzed with McMaster robotic stem cell screening system in partnership with collaborations that include academic groups as well as industry.

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Drug destroys human cancer stem cells but not healthy ones

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From stem cell to brain cell: New technique mimics the brain

Posted: May 26, 2012 at 9:18 am

ScienceDaily (May 24, 2012) A new technique that converts stem cells into brain cells has been developed by researchers at Lund University. The method is simpler, quicker and safer than previous research has shown and opens the doors to a shorter route to clinical cell transplants.

By adding two different molecules, the researchers have discovered a surprisingly simple way of starting the stem cells' journey to become finished brain cells. The process mimics the brain's natural development by releasing signals that are part of the normal development process. Experiments in animal models have shown that the cells quickly adapt in the brain and behave like normal brain cells.

"This technique allows us to fine-tune our steering of stem cells to different types of brain cells. Previous studies have not always used the signals that are activated during the brain's normal development. This has caused the transplanted cells to develop tumours or function poorly in the brain," says Agnete Kirkeby, one of the authors of the study.

Since the method effectively imitates the brain's own processes, it reduces the risk of tumour formation, one of the most common obstacles in stem cell research. The quick, simple technique makes the cells mature faster, which both makes the transplant safer and helps the cells integrate better into the brain. The results of the study bring stem cell research closer to transplant trials in the human brain.

"We have used the new protocol to make dopamine neurons, the type of neuron that is affected by Parkinson's disease, and for the first time, we are seriously talking about these cells as being good enough to move forward for transplantation in patients. The next step is to test the process on a larger scale and to carry out more pre-clinical safety tests," explains Malin Parmar, research team leader.

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Fat stem cells may help treat kidney ailments

Posted: May 25, 2012 at 8:16 pm

Fat stem cells may help treat kidney ailments BS Reporter / Mumbai/ AhmedabadMarch 06, 2007 In a breakthrough in the stem cell research, scientist from Ahmedabad have developed a technique to encourage a new kind of stem cells called Mesenchymal stem cells generated from fat (adipose tissue) of donors, which can be used in treating kidney diseases. Mesenchymal stem cells generated from fat of donors hold great promise for the treatment of kidney diseases, claims H L Trivedi, director, Institute of Kidney Diseases and Research Center (IKDRC), Ahmedabad. We will soon patent the research, he added. The institute will soon convene a meeting of scientists working on the project and take a decision on securing the patent for the research. A team of scientists from the IKDRC, led by Trivedi, has clinically proved that when presented in the right physical context, certain growth factors encourage the survival and proliferation of fat mesenchymal stem cells grown outside the body. Trivedi says the research offers hope of cent per cent recovery for patients suffering from severe kidney diseases as the mesenchymal stem cells will nullify the rejection rate of the body, thus inducing the body to accept the newly transplanted kidney as part of its own body. Emphasising on the success of mesenchymal stem cells for kidney treatment, Trivedi further said mesenchymal stem cells were the best repair stem cells compared to other stem cells. The worlds first recipient of these kinds of stem cells is a kidney patient - Hetalben Mewada, a 30-year-old housewife from Palanpur in Gujarat, claims the scientist from IKDRC. Speaking about the financial aspect of the kidney treatment, Trivedi said, Mesenchymal stem cells using fat is simple and cheap. The latest invention would cut the cost of surgery drastically and make it affordable for the needy people. It will also reduce the chances of recurrence and complexity in the post surgery situation, he said. Mesenchymal stem cells are available in bone marrow and peripheral blood cells in smaller quantity, but the cells are not economically feasible. Mesenchymal stem cells can be easily derived from fat and is economically viable. Mesenchymal stem cells that are already in the fats are separated and grown through culturing technique in laboratory. IKDRCs scientists carried out a kidney transplant operation using Mesenchymal stem cells derived from fats along with adult hematopoietic stem cells infused into the transplanted kidney to create tolerance or acceptance by the patients. This eliminates the chance of rejection, and the patients would not need medication. Under this procedure, the mesenchymal stem cells act as a big brother to adult hematopoietic stem cells. Mesenchymal stem cells protect these hematopoietic stem cells and help their grafting into different organs by themselves getting grafted and making space for their younger siblings (adult hematopoietic stem cells) to live along with them in their vicinity. Fat stem cells may help treat kidney ailments BS Reporter / Mumbai/ Ahmedabad Mar 06, 2007, 22:52 IST

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