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'Hope' for the paralysed?

By Dr. Matthew Watson

1 June 2012 Last updated at 12:19 ET

Seven years ago I stood on a bridge over the M40 doing a "piece to camera" for a report about spinal repair. The aim was to come up with a metaphor for how researchers at University College London were trying to overcome spinal cord paralysis.

It went something like this: "Imagine your spinal cord as a motorway, the cars travelling up and down are the nerve fibres carrying messages from your brain to all parts of the body. If this gets damaged the cars can't travel. The messages are blocked, the patient is paralysed.

"Normally there is no way of repairing a severed spinal cord. But the team at UCL took nasal stem cells, and implanted them into the area of damage. These formed a bridge, along which the nerve fibres re-grew and re-connected."

This is the World-Cup of neurorehabilitation. Our rats have become athletes when just weeks before they were completely paralysed.

The research at the Spinal Repair Unit at UCL involved rats, not humans. In my TV report we showed rats unable to climb a metal ladder after one of their front paws had been paralysed to mimic a spinal cord injury. But after an injection of stem cells, the rats were able to move nearly as well as uninjured animals.

The hope then - and now - is that such animal experiments will translate into similar breakthroughs with patients. Seven years on and the team at UCL led by Professor Geoff Raisman are still working on translating this into a proven therapy for patients. He told me "This is difficult and complex work and we want to ensure we get things right."

So it was with a sense of caution that I approached some Swiss research in the latest edition of the journal Science in which paralysed rats were able to walk again after a combination of electrical-chemical stimulation and rehabilitation training.

The research prompted some newspaper reports talking of "new hope" for paralysed patients. The lead researcher, Professor Gregoire Courtine enthused: "This is the World-Cup of neurorehabilitation. Our rats have become athletes when just weeks before they were completely paralysed."

My colleague James Gallagher has reported on the research and you can read his copy here.

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'Hope' for the paralysed?

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Paralyzed rats walk again in Swiss study

By Dr. Matthew Watson

LONDON Scientists in Switzerland have restored full movement to rats paralyzed by spinal cord injuries in a study that spurs hope that the techniques may hold promise for someday treating people with similar injuries.

Gregoire Courtine and his team at Ecole Polytechnique Federale de Lausanne saw rats with severe paralysis walking and running again after a couple of weeks following a combination of electrical and chemical stimulation of the spinal cord together with robotic support.

"Our rats are not only voluntarily initiating a walking gait, but they are soon sprinting, climbing up stairs and avoiding obstacles," said Courtine, whose results from the five-year study will be published in the journal Science on Friday.

Courtine is quick to point out that it remains unclear if a similar technique could help people with spinal cord damage but he adds the technique does hint at new ways of treating paralysis.

Other scientists agree.

"This is ground-breaking research and offers great hope for the future of restoring function to spinal injured patients," said Elizabeth Bradbury, a Medical Research Council senior fellow at King's College London.

But Bradbury notes that very few human spinal cord injuries are the result of a direct cut through the cord, which is what the rats had. Human injuries are most often the result of bruising or compression and it is unclear if the technique could be translated across to this type of injury.

It is also unclear if this kind of electro-chemical "kick-start" could help a spinal cord that has been damaged for a long time, with complications like scar tissue, holes and where a large number of nerve cells and fibres have died or degenerated.

Nevertheless, Courtine's work does demonstrate a way of encouraging and increasing the innate ability of the spinal cord to repair itself, a quality known as neuroplasticity.

Other attempts to repair spinal cords have focused on stem cell therapy, although Geron, the world's leading embryonic stem cell company, last year closed its pioneering work in the field.

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Fat-Derived Stem Cells Show Promise for Regenerative Medicine, Says Review in Plastic and Reconstructive Surgery(R)

By Dr. Matthew Watson

ARLINGTON HEIGHTS, Ill., May 29, 2012 (GLOBE NEWSWIRE) -- Adipose stem cells (ACSs)--stem cells derived from fat--are a promising source of cells for use in plastic surgery and regenerative medicine, according to a special review in the June issue of Plastic and Reconstructive Surgery(R), the official medical journal of the American Society of Plastic Surgeons (ASPS).

But much more research is needed to establish the safety and effectiveness of any type of ASC therapy in human patients, according to the article by ASPS Member Surgeon Rod Rohrich, MD of University of Texas Southwestern Medical Center, Dallas, and colleagues. Dr. Rohrich is Editor-in-Chief of Plastic and Reconstructive Surgery.

Adipose Stem Cells--Exciting Possibilities, but Proceed with Caution

The authors present an up-to-date review of research on the science and clinical uses of ASCs. Relatively easily derived from human fat, ASCs are "multipotent" cells that can be induced to develop into other kinds of cells--not only fat cells, but also bone, cartilage and muscle cells.

Adipose stem cells promote the development of new blood vessels (angiogenesis) and seem to represent an "immune privileged" set of cells that blocks inflammation. "Clinicians and patients alike have high expectations that ASCs may well be the answer to curing many recalcitrant diseases or to reconstruct anatomical defects," according to Dr. Rohrich and co-authors.

However, even as the number of studies using ASCs increases, there is continued concern about their "true clinical potential." The reviewers write, "For example, there are questions related to isolation and purification of ASCs, their effect on tumor growth, and the enforcement of FDA regulations."

Dr. Rohrich and co-authors performed an in-depth review to identify all known clinical trials of ASCs. So far, most studies have been performed in Europe and Korea; reflecting stringent FDA regulations, only three ASC studies have been performed in the United States to date.

Many Different Uses, But Little Experience So Far

Most ASC clinical trials to date have been performed in plastic surgery--a field with "unique privileged access to adipose tissues." Plastic surgeon-researchers have used ASCs for several types of soft tissue augmentation, such as breast augmentation (including after implant removal) and regeneration of fat in patients with abnormal fat loss (lipodystrophy). Studies exploring the use of ASCs to promote healing of difficult wounds have been reported as well. They have also been used as a method of soft tissue engineering or tissue regeneration, with inconclusive results.

In other specialties, ASCs have been studied for use in treating certain blood and immunologic disorders, heart and vascular problems, and fistulas. Some studies have explored the use of ASCs for generating new bone for use in reconstructive surgery. A few studies have reported promising preliminary results in the treatment of diabetes, multiple sclerosis, and spinal cord injury. No serious adverse events related to ASCs were reported in either group of studies.

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Stem cell hope for hip replacement procedure

By Dr. Matthew Watson

Hip replacements for some patients could be a thing of the past after surgeons pioneered a new stem cell procedure to tackle a bone disease that leads to arthritis.

Doctors at Southampton General Hospital are extracting stem cells from the bone marrow of patients in need of hip repair due to osteonecrosis - a condition where poor blood supply causes significant bone damage leading to severe arthritis.

These cells are mixed with cleaned, crushed bone from another patient who has had their own hip replaced and used to fill the hole made by surgeons after dead and damaged tissue has been removed from the joint.

The procedure has been developed by Doug Dunlop, a consultant orthopaedic surgeon at Southampton General Hospital, and Professor Richard Oreffo, a specialist in musculoskeletal science at the University of Southampton.

"Although this work is still ongoing, several patients who have had the procedure have reacted very well and, if we get the results we are hoping for, these patients won't need to have their hip joints replaced - they should be fixed completely," said Mr Dunlop.

Professor Oreffo added: "By using stem cells to send out chemical signals to blood vessels, we hope the body will continue to create new vessels in the hip which supply enough nutrients to maintain bone strength."

Osteonecrosis is on the rise in the UK with around 4,000 cases a year but it is much more widespread in Asia where it is the most common form of arthritis of the hip, the hospital said.

It can also be treated with drugs to help avoid arthritis and usually strikes between 30 and 50 years of age.

Osteonecrosis is one of the three main causes of arthritis alongside osteoarthritis and rheumatoid arthritis.

Arthritis in general affects one in five people in the UK.

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Fatty liver disease – Choline provides a nutritional solution for a silent epidemic

By Dr. Matthew Watson

by: Helmut Beierbeck

Fatty liver disease used to be associated with alcoholism, but it is no longer
restricted to heavy drinkers. Our calorie-rich but nutrient-poor diet has led
to an epidemic of nonalcoholic fatty liver disease (NAFLD) that tracks our
rising obesity and diabetes rates (1). Autopsies and ultrasound studies have
shown that up to 75% of the obese and 70-85% of type 2 diabetics have fatty
livers. And the low-profile but essential nutrient choline appears to provide
the solution to the problem (1, 2).

What is NAFLD?

NAFLD develops in two stages (1). In the first stage fat accumulates in the
liver. This fat can come from several sources: free fatty acids released into
the blood by fat tissue, lipogenesis in the liver from carbohydrates
(especially fructose from HFCS or table sugar), and dietary fats carried to the
liver by chylomicron remnants. Fatty liver disease is a silent epidemic because
its first stage, fat accumulation, generally doesn't produce overt symptoms. Readmore…

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Industry-sponsored cardiovascular cell therapies. Some metrics.

By Dr. Matthew Watson

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Cell therapies for cardiovascular-related conditions is a closely watched, much studied, oft-discussed, and hotly contested segment of the cell therapy industry.


The data to-date are admittedly confusing.  From a clinical perspective, the studies for which we have data have been relatively small involving a mish-mash of indications, endpoints, eligibility criterion, methods and/or route of administration, as well as the time of administration relative to event or disease progression.


Further compounding any interpretation of the data, from a technical perspective, is the fact the products have been widely varied in terms of being autologous vs allogeneic, expanded and not, genetically modified and not, from a plethora of different sources, and utilizing a wide variety of cell types from skelatal myoblasts, cardiomyocytes, mesenchymal stromal cells, mononuclear cells, etc. 


All this makes it extremely difficult to draw any conclusions with respect to what's working and what's not.  We will not attempt to do so.


All we do below is attempt to give a snapshot of the industry-sponsored cell therapy trials currently ongoing for cardiovascular-related conditions.  So here it is:


Commercial:
Pharmicell's Heartcelligram is the only cell therapy to have received regulatory approval for commercial distribution for the treatment of a cardiac-related indication.  Heartcelligram is an autologous cell therapy approved in 2011 by the Korean Food and Drug Administration (KFDA) for the treatment of Acute Mycardial Infarction (AMI).  The price is reportedly $19,000 and the trial data behind the approval has not yet been published in a peer-reviewed journal.


Phase III or II/III:
There are currently only 3 active and recruiting cardiac-related, industry-sponsored cell therapy trials.  Interestingly they all involve autologous products, two involve devices, two involve centralized manufacturing, two involve bone marrow cells as a source, two are only in European clinical sites, and two are targeting ischemic-related conditions.

  • Baxter Therapeutics' Auto-CD34+ cells
  • Cytori
Two companies warrant particular mention at this stage as they appear to be in transition between phases II and III.

Cardio3 Biosciences initially designed a trial of their autologous C-Cure in heart failure secondary to ischemic cardiomyopathy to be a phase II/III trial enrolling 240 patients.  While the trial began in late 2008 and is still registered as active but no longer recruiting on ClinicalTrials.gov the entry has not been updated for almost a year.  

In 2010 the company announced that after enrolling 45 patients - of which 21 were in the treatment arm (24 in the control arm) - they decided to close the study to future enrollment and prepare for a phase III trial.  This decision was reportedly based on "very encouraging data". 

Dr. Christian Homsy, CEO of Cardio3 BioSciences provided the following guidance: “The highly promising data we report today build on the favourable safety profile we have observed through this Phase II trial and documents in patients our belief that we have with  C-Cure a product candidate with the potential to make a real difference in the treatment of heart failure... As noted in the company’s press release of 29 June 2010, with the Phase II stage completed and to allow for potential modifications to the trial protocol, Cardio3 BioSciences has not proceeded to Phase III recruitment into the trial but has continued to gather all data for the six month analysis. Through the Phase II trial, we gained significant  experience in working with a highly innovative stem cell therapy in a clinical setting, and we are using this acquired knowledge in the design of our planned Phase III programme."  The phase III trial of C-Cure is expected to commence in the second half of 2012.

Mesoblast has also announced with its strategic partner, Teva, that they are proceeding with plans to conduct a phase III study of its allogeneic cell therapy product, Revascor, in chronic heart failure.  Most anticipate this clinical trial application to be filed sometime in late 2012.


Phase I or II:
There are over 20 active, industry-sponsored earlier-stage trials (phase I, I/II or II) for cardiovascular-related conditions.  At least 5 of these are expected to have clinical readouts this year.   



Hope this is useful.

--

This post has been brought to you by your friends at CTG.  All cell therapy. All the time. ðŸ™‚  

-- Lee @celltherapy

p.s.  As always we welcome your feedback, comments, and corrections.  
























































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Psychiatry’s "Bible" Gets an Overhaul (preview)

By Dr. Matthew Watson

Editor's Note: Read our blog series on psychiatry's new rulebook, the DSM-5.

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A Look at the ‘Son of CIRM’ Proposal on the June California Ballot

By Dr. Matthew Watson


In the last couple of weeks, two
well-respected Los Angeles Times columnists have visited what
might be called the "Son of CIRM" initiative on the
June ballot in California. It is aimed at fighting cancer by spending
$800 million or so annually on research with the money coming from a
$1-a-pack tax on cigarettes.

One of the columnists, Michael
Hiltzik
, said the measure, Proposition 29, is another
example of why California is a world leader in "paving the road to hell with good intentions." The other writer, George
Skelton, said,

"Prop. 29 would increase cancer
research. Reduce smoking. Save lives. Hurt the lying tobacco
companies. Good plan."

In his work at the Times, Hiltzik deals primarily with business
and financial news. He has written from time to time critically about
the $3 billion California stem cell agency.  Skelton is a longtime
observer of the Califorrnia political scene and has been around since
Pat Brown was governor.
In a column slated for publication
Sunday, Hiltzik said that the drafters of the cancer measure closely
examined Proposition 71, which created the stem cell agency in
2004, and "managed to reproduce the earlier measure's worst
features."
He said the Proposition 71 "retired
the trophy for doing the wrong thing in the wrong way for what sounds
like the right reasons." Hiltzik wrote,

"Proposition 71, you may recall, was sold to a gullible
public via candy-coated images of Christopher Reeve walking
again and Michael J. Fox cured of Parkinson's.
The implication was that these miracles would happen if voters
approved a $3-billion bond issue for stem cell research. Who could be
against that? 

 "As it turned out, the stem cell
measure created an unwieldy bureaucracy and etched conflicts of
interest into the state Constitution. By last count about 85% of the
$1.3 billion in grants handed out by the program, or some $1.1
billion, has gone to institutions with representatives on the stem
cell board. The program is virtually immune to oversight by the
Legislature or other elected officials. For these reasons and others,
it has grappled with only mixed success with changes in stem cell
science and politics that have called its original rationale into
question."

Hiltzik continued,

"Proposition
29, similarly, places most spending from the tobacco tax in the hands
of a nine-member board that must comprise one cardiovascular
physician affiliated with a California academic medical center; the
chancellors of UC Berkeley, UC San Francisco and UC
Santa Cruz
; two representatives of lobbying groups devoted to
tobacco-related illness (including one who has been treated for such
a disease); and three representatives from National Cancer
Institute
-designated cancer centers in the state. There are
10 of the latter, including five UC campuses and the City of Hope.
Plainly, every member of the board will represent an employer that
thinks it's in line for some of the money."

Skelton took a different approach on May 14. Using the words of a federal judge,
he lambasted the tobacco industry for its "a
certified history of deception, distortion and lying. And let's not
forget fraud and racketeering."
Skelton dealt with the current TV ads
being aired in California against the initiative. They criticize the
measure for its conflicts of interest and also say that the money
would be spent out of state.
Skelton wrote,

"The anti-29 side is hitting this
hard: that the research money generated in California could be spent
out of state. And the politest thing possible to say about that claim
is that it's disingenuous. It's stretching something that's
conceivable into a virtual certainty."

Skelton continued,

"The anti-29 camp charges that
(the structure of the board) would allow a conflict of interest in
awarding contracts. But there are state laws that protect against
such conflicts.

"Anyway, the tobacco crowd can't have it
both ways: complaining that the money could be spent outside
California and also griping when the system is set up to practically
guarantee that it will be spent in California."

Our take:
Ballot box budgeting – which is at
the heart of both the stem cell and cancer initiatives -- is one of the
reasons that California is staggering from one year to the next in a
perennial financial mess. Initiatives also sometimes create nasty
blowback that can damage the effort that they ostensibly serve. Such
is the case with the California stem cell agency, which suffers from
management and other minutia embedded in Proposition 71 that is virtually
politically impossible to change.
Hiltzik wrote,

"Gov. Brown's latest budget
proposal calls for cuts of $1.2 billion in Medi-Cal and
$900 million in CalWorks (a relief program for families with
children) and steep cuts in financial aid for college students and in
court budgets. The University of California and Cal State systems are
becoming crippled by 20 years of cutbacks in state funding,
leading to soaring tuition charges. Tobacco-related illnesses create
some of the burden on Medi-Cal and other public healthcare programs,
yet a minimal portion of Proposition 29 revenue, if any, would go to
helping taxpayers carry that burden. 

"With the overall state budget gap
approaching $16 billion, how can anyone make the case for diverting a
huge chunk of $800 million a year in new revenue to long-term
scientific research, whether in California or not? Even if you
believe that case can be made, the proper place to make it is in the
Legislature, where all these demands on the budget can be weighed and
balanced against one another — not at the ballot box, where the
only choice is to spend it the way the initiative's drafters choose
or not to raise it at all."

The California Stem Cell Report agrees
wholeheartedly.
(A personal disclosure: I worked for
Skelton when he was bureau chief for United Press International in
Sacramento some decades ago and consider him a friend. I am also
acquainted with Hiltzik but have not known him as long. I hold both
men in high regard.)

Source:
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‘Sun Never Sets on CIRM’ – California Agency Awards $69 Million to Researchers

By Dr. Matthew Watson


The California stem cell agency today
awarded $69 million in grants, including the first involving a collaboration with researchers in China, but none of the awards went to California
biotech businesses.
The awards were made in the agency's
third translational round, which funds projects that are in the
initial stage of identifying drugs or cell types that could become
drug therapies.
CIRM originally allocated $95 million
for the round, but CIRM spokesman Kevin McCormack said that grant
reviewers determined that no applications beyond $69 million were
worthy of funding.
The CIRM governing board overturned a negative
reviewer decision on one grant after the scientist – W. Douglas
Boyd
of UC Davis -- filed an appeal. The appeals of two other
researchers, including one from a San Diego business, were not successful (see here
and here).
CIRM did not disclose the number of
applications from businesses. The agency has been sharply
criticized for failing to fund businesses in a substantial way.
The approved grants involve
collaboration with researchers in Australia and Germany as well as
China. The collaborations are based on agreements worked out earlier
by CIRM with overseas groups, which fund their own countries' researchers. No CIRM cash is involved, according to the agency.
CIRM President Alan Trounson, a native
of Australia and researcher there until joining the stem cell agency,
said in a press release,

"The sun now never sets on the
CIRM collaborative projects..."

The news release also said,

 "The
Chinese Ministry of Science and Technology has committed roughly
$850,000 in collaboration with a team at UCSF to study liver failure.
This is the stem cell agency’s first joint effort with scientists
in China, which is home to a fast-growing stem cell research
community."

The UCSF liver team is led by Holger
Willenbring
, whose goal is "to develop a source of autologous
therapeutic cells for patients with liver disease who otherwise would
require a liver transplant," according to the CIRM review summary.
The agency did not spell out the details of how the collaboration
would work.
All of the winning applicants, with the
exception of a Salk researcher, work for institutions linked to at
least one of the 29-members of the CIRM governing board. CIRM
directors, however, are barred from voting or even discussing applications in which CIRM attorneys have determined there is a conflict
of interest.
You can find the names of all the successful applicants in the CIRM news release.    

Source:
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Thin Coverage of California Stem Cell Board Meeting

By Dr. Matthew Watson


Media coverage of yesterday's $69
million in research awards and other matters involving the California
stem cell agency was nearly non-existent today.
That is not unusual, however, since the
$3 billion enterprise is not within the attention span of the
mainstream press and electronic outlets.
The California Stem Report could find
only two stories involving yesterday's actions. One by Ron Leuty
appeared in the San Francisco Business Times and was a look at the grant awards. The other appeared on Nature's website.
Unfortunately, Nature's lead was incorrect.
It said,

 "The California Institute of Regenerative Medicine
(CIRM)
voted on 24 May to accept a new strategic planwhich
shrinks or eliminates support for basic research, facilities and
training, while funneling more of its funds toward clinical
development."

The CIRM governing board actually put
off until at least July decisions on which programs to cut and which
to expand. Basic research is not likely, however, to take a major
hit, for a variety of reasons.

Source:
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Stem Cell Agency Board Sticks with More Financial Disclosure

By Dr. Matthew Watson


The governing board of the $3 billion
California stem cell agency today rejected a proposal that would have
restricted transparency surrounding the financial interests of its
directors and top executives.

On a unanimous voice vote, the board
decided it would stick with the more complete disclosure rules that it
has operated under since 2005. CIRM staff had offered changes that would have narrowed the amount of economic
information that the board members and the executives would have been
required to disclose.
The directors' Governance Subcommittee, however, on May 3 rejected the plan. Sherry Lansing, a former
Hollywood film studio CEO and chair of the subcommittee, said at the time,

"I personally feel strongly that
because of CIRM's unique mission and the agency's incredibly
long-standing commitment to transparency, i believe that we should
continue to set an example by requiring the broadest disclosure for
members of the board and high level staff."

Retention of existing disclosure
rules comes at a time when more conflicts may arise. The agency is
moving to engage the biotech industry more closely as it pushes to
develop stem cell therapies. Already one case of conflict has arisen this
year dealing with industry. It involves a "special advisor"
to CIRM who was nominated to become director of a firm sharing in a
$14.5 million grant. She also was working for the firm. (See here and
here.)
The CIRM board also has built-in
conflicts of interest, written into the law by Proposition 71, which
created the agency. About 92 percent of the $1.3 billion awarded so
far has gone to institutions tied to members of the CIRM governing
board. Board members are not permitted, however, to vote on or
discuss grants to their institutions. But it is fair to say that if
California voters had foreseen that nearly all of the grants would
have gone to directors' institutions, they would not have
approved creation of the stem cell agency.
As the California Stem Cell Report remarked earlier, it is a good
move for CIRM to retain more transparency rather than less. As one of
the Moss Adams staffers said today – in a different context –
during the presentation of the first-ever performance audit of CIRM,

"When people have to fill a void
in information, they assume the worst."

Source:
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California Stem Cell Agency Launches Five-Year Push for Cures

By Dr. Matthew Watson


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

By Dr. Matthew Watson


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. 

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

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Live Coverage of Tomorrow’s California Stem Cell Meeting

By Dr. Matthew Watson


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.  

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

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

By Dr. Matthew Watson


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.”

Source:
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Third Researcher Appealing Grant Rejection to Stem Cell Agency Board

By Dr. Matthew Watson


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.

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

By Dr. Matthew Watson


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. 

Source:
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Former Zumba instructor with cancer encourages Hispanics to donate bone marrow

By Dr. Matthew Watson

Photo by Rachel Denny Clow, Corpus Christi Caller-Times // Buy this photo

Rachel Denny Clow/Caller-Times Cristina Rodriguez sits with her dogs Coby (left) and Flower at her home Thursday. Rodriguez, who has non-Hodgkin lymphoma, is having a Zumba benefit on Sunday and inviting people to register to donate bone marrow. Rodriguez is a former Zumba instructor.

CORPUS CHRISTI Had Cristina Rodriguez's cancer been more aggressive, had it penetrated her bones, things might have been different.

And while she has had chemotherapy, she has lost her hair and needs a stem cell treatment, but she doesn't need a bone-marrow transplant.

And for that, she's lucky.

Hispanics needing bone marrow have a harder time finding matching donors than do other ethnicities because few Hispanics have registered to donate.

"That could've easily been me," Rodriguez said.

That's why Rodriguez, 31, is trying to raise awareness about the importance for Hispanics to give bone marrow. The former Zumba instructor is hosting a Zumba event Sunday afternoon that partly is a fundraiser for her ongoing cancer treatments and partly a campaign to encourage more people to become donors.

Among the 8 million people signed up as bone marrow donors, 800,000 or 10 percent, identify themselves as Hispanic or Latino. Though Hispanics comprise more than one-third of Texas' population, only 17 percent of registered bone marrow donors in the state are Hispanic.

Overall, Hispanics have a 72 percent chance of finding a bone marrow donor, compared with whites, who have a 93 percent chance, according to the donor program. Only blacks fare worse, with a 63 percent likelihood of finding a donor.

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Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells

By Dr. Matthew Watson

Editor's Choice Main Category: Cardiovascular / Cardiology Article Date: 25 May 2012 - 0:00 PDT

Current ratings for: 'Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells'

4 (1 votes)

This achievement is significant, as it opens up the prospect of treating heart failure patients with their own, human-induced pluripotent stem cells (hiPSCs) to fix their damaged hearts.

Furthermore, the cells would avoid being rejected as foreign as they would be derived from the patients themselves. The study is published in the European Heart Journal. However, the researchers state that it could take a minimum of 5 to 10 years before clinical trials could start due to the many obstacles that must be overcome before using hiPSCs in humans is possible.

Although there has been advances in stem cell biology and tissue engineering, one of the major problems scientists have faced has been lack of good sources of human heart muscle cells and rejection by the immune system. Furthermore, until now, scientific have been unable to demonstrate that heart cells created from hiPSCs could integrate with existing cardiovascular tissue.

"What is new and exciting about our research is that we have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are health and young - the equivalent to the stage of his heart cells when he was just born," said Professor Lior Gepstein, Professor of Medicine (Cardiology) and Physiology at the Sohnis Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine, Technion-Israel Institute of Technology and Rambam Medical Center in Haifa, Israel, who led the study.

In the study, Professor Gepstein, Ms Limor Zwi-Dantsis, and their colleagues retrieved skin cells from two male heart failure patients, aged 51 and 61 years, and reprogrammed the cells by delivering 3 transcription factors (Sox2, Oct4, and Klf4) in addition to a small molecule called valproic acid, to the cell nucleus. The team did not include a transcription factor called c-Myc as it is a known cancer-causing gene.

Professor Gepstein said:

In addition, the team used an alternative strategy involving a virus transferred reprogramming data to the cell nucleus. However, the team removed the virus after the information had been transferred in order to avoid insertional oncogenesis.

Read more here:
Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells

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Frozen cord could save a life

By Dr. Matthew Watson

Tim and Padma Vellaichamy of Parramatta have had their new born child's umbilical cord stored cryogenically for future treatment. Pictured with their as yet unnamed three week old daughter. Picture: Adam Ward Source: The Daily Telegraph

IT'S current preservation for future regeneration - and now umbilical cord tissue is going on ice in Australia for the first time.

Usually discarded after birth, umbilical tissue from newborn babies is being collected and cryogenically frozen to be used one day for regenerative and stem cell medicine. And it doesn't just have potential for the babies involved, either. Experts say stem cells could also be used for family members who are genetically compatible.

It is hoped the cells will eventually be able to be used to repair damaged tissues and organs, with researchers investigating its uses for treating diseases like multiple sclerosis, cerebral palsy and diabetes, as well as for bone and cartilage repair.

Although cord blood storage has been available for many years, Cell Care Australia has added cord tissue storage in anticipation of new discoveries in the regenerative medicine field.

Cell Care Australia medical director associate professor Mark Kirkland said the storage process - already popular in the US, Europe and Southeast Asia - was long overdue for Australian shores.

"The science is developing around the world and we're really behind the rest of the world in providing parents the option to store these cells and we thought it was about time it was brought here," he said.

"It's finding a way to take what would otherwise be waste tissue and turning it into something of potential future value for not only your child but also potentially for other family members.'

Parramatta couple Tim and Padma Vellaichamy are among the first to use the service in Australia.

Mr Vellaichamy, 31, said he heard of the technology while working as a dentist in India and decided to store their daughter's cord cell tissue after birth three weeks ago.

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