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An Unseemly Performance: Former Chair of Stem Cell Agency Promotes $20 Million Research Proposal

August 26th, 2012 3:54 pm


Bob Klein is nearly an icon in the
history of the $3 billion California stem cell. And when he appeared
before its governing board last month and aggressively touted a $20
million grant proposal already rejected by agency reviewers, his
actions raised eyebrows.

Robert Klein
Elie Dolgin/Nature photo
Klein's comments carried unusual
weight, given that they were supported by his unique and influential
relationship with the California Institute for Regenerative
Medicine(CIRM)
. He and his associates wrote the 10,000-word ballot
initiative that created the stem cell agency in 2004. He ran the $35
million electoral campaign that convinced voters to buy into the
idea. Klein raised millions on behalf of the effort. He personally
provided the campaign $3 million. And he was the first chairman of
the agency, leaving that office only 13 months ago, when he was
designated chairman emeritus.
The meeting last month marked Klein's
first public appearance before the board on behalf of a specific
application.. He heralded the applicant, StemCells, Inc., as unique
and the “best” in United States with a “huge body of
experience.”

(The full text of his testimony can be found here.)
Irv Weissman
Stanford Photo
StemCells Inc. is a publicly traded company based in Newark, Ca., that was founded by renown Stanford
scientist Irv Weissman, who sits on its board. Weissman also played
an important role in the Prop. 71 ballot campaign that created the
stem cell agency. StemCells, Inc.'s application was turned down by
CIRM reviewers who gave it a score of 61, but the company appealed the action to the agency's governing board. Following the appearance by Klein, Weissman and others, the CIRM board sent the application back for more review.
The board will reconsider it next month or in October.
One California stem cell researcher,
who requested anonymity, said it is “highly inappropriate for Bob
Klein to be advocating for any grant application from a public
company.”
The scientist said,

 “He has
considerable influence with the ICOC(the CIRM governing board), and
is closely associated with biotech in the Bay Area. Even if he
doesn't make a lot of money himself from this, then he certainly has
friends who will.  Irv Weissman would be one of those friends."

In response to questions asked on Aug. 7 by the
California Stem Cell Report, Klein today defended his actions.  He was asked if he had “any sort of
financial ties” to firms or individuals that would benefit from
approval of the award. Klein, who is a real estate investment banker and also an attorney, said he has
“no financial interest” in the firm or individuals that might
benefit.
Klein also indicated his appearance was
entirely appropriate. He defined his role as a patient advocate –
not as a lobbyist who is paid for advocating on behalf of a company.
Klein said he had “a particular responsibility to contribute my
background knowledge and experience.”
Klein said he hoped other former board
members would follow his example. He said,

“(I)t would be a tragedy if the
expertise of board members built up over six or more years is lost.”

(The full text of his response can be found here.)
Klein's appearance came at a propitious
time for financially strapped StemCells, Inc. The company's
financial information shows that it is losing $5.4 million a quarter
as of the end of June and had only $9 million in cash on hand. It
also had liabilities of $11.6 million, up substantially from $8.5
million in September of last year.
The researcher who criticized Klein's efforts as inappropriate also said,

"StemCells Inc has been on the
stock market for 20 years, without producing anything of value for
the investors.  The stock price has been sinking fast:  it
was 60 cents this June; last year at this time, it was around $5 a
share.   

“On July 17, when the CIRM Disease
Team Award review results became available, the stock rose from 87
cents to $1.80 – a person who could anticipate the outcome of the
CIRM applications could have made considerable money in that 24 hour
period.”

Weissman's role
with the StemCells, Inc., is more than scientific. According to the
company's financial statements, he holds 88,612 shares. His wife,
Ann Tsukamoto
, is executive vice president of the firm. She holds
185,209 shares in the firm.
Weissman played a significant role in
the Prop. 71 campaign. He did the “billionaire circuit,” raising
money for the initiative, according to an article by Diana Kapp in
San Francisco magazine. Among other things, Weissman worked the
exclusive Bohemian Grove in Northern California and “pitched”
Bill Bowes, a co-founder of Amgen, who, along with his wife, gave
$1.3 million to the campaign. Weissman was the key to securing a
$400,000 contribution from Microsoft's Bill Gates. Weissman also plumped for Prop. 71 in a TV campaign ad.
In addition to StemCells, Inc., Klein
and Weissman supported a successful attempt last month to overturn
reviewers' rejection of another $20 million application by Judith Shizuru
of Stanford. The application received a score of 53 from reviewers.
One of the application's problems cited
by reviewers was the availability of antibodies for the study. The
antibodies were developed by Systemix, a company founded by Weissman.
Systemix was acquired by Novartis in 1997 for about $70 million.
Weissman said he has “negotiated back” rights to key antibodies,
which he said are now held by Stanford.
Klein said that reviewers believed the
research was “a showstopper” but did not think the documentation
was adequate. He told the CIRM directors that they now have a letter
with proprietary information that supports the grant application.
Our take: The stem cell agency has long
labored under the perception that it is something of an insiders'
club. Even the prestigious journal Nature warned in 2008 about what
it called “cronyism” at CIRM. If anything, the situation is worse today,  four years later. Enterprises associated with persons on the CIRM board of directors have received more than 90 percent of the funds handed out by the agency. Klein's efforts last month
reinforce the not-so-pleasant image of the stem cell agency as an
old boy's club and create an impression – at the very least – of
unseemly insider influence.

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

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Text of Klein’s Response to Questions Concerning His Advocacy on Rejected Grant Applications

August 26th, 2012 3:54 pm


Here is the text of Robert Klein's response today to the California Stem Cell Report concerning his appearance before the governing board of the California stem cell agency July 26, 2012. Klein, former chairman of the agency, real estate investment banker and attorney, promoted two applications seeking $20 million each from the agency. Both applications had been rejected by the agency's reviewers. Here is a link to an item on the subject.

"Dear David,
"You have posed two
questions related to my continuing role as a Patient Advocate in
contributing information to the Board of the California Institute for
Regenerative Medicine
, in an effort to optimize decisions on medical
and scientific grants and loans for research that could mitigate
and/or cure chronic diseases or injuries. 

"Q: Do you have any sort
of financial ties to StemCells Inc. or any of the individuals or
firms that would benefit from approval of those awards by the ICOC(the CIRM governing board)?
"A: I have no financial
interest in StemCells Inc. or any of the individuals or firms that
would benefit from approval of those awards by the ICOC. In fact, I
have no financial interest in any biomedical research company.

"Q: Do you think it is
appropriate for the former chairman of the ICOC to lobby that body on
behalf of awards to specific companies or individuals?
"A: First, it is
fundamental that the terms be defined to properly respond to your
question. A “Patient Advocate” is a member of a patient family or
a medical/scientific care /support group who advocates for medical
and scientific advances that might potentially mitigate and/or cure a
patient’s chronic disease or injury. A “Patient Advocate” is
not paid for his/her advocacy, unless they are staff members of a
non-profit institution dedicated to a specific disease or group of
diseases or injuries. 

"Second, a “lobbyist”
is a paid representative of a company or a for-profit institution(s)
with a financial interest in the outcome of a governmental decision. 

"I am serving as a
Patient Advocate in my presentations to the Board of the California
Institute for Regenerative Medicine. As the former Chairman of the
Board, I have a particular responsibility to contribute my background
knowledge and experience for the Board to consider, along with all
new information, in reaching their best decision. I hope other former
Board members, who possess a wealth of scientific, medical, and
institutional knowledge that can benefit the Board, would consider
the value they can contribute to future decisions. As Board terms
expire, it will be important not to lose that institutional knowledge
and medical/scientific expertise that has been built up over the last
seven plus years of the Agency’s existence. 

"In an outline format,
I would suggest the following areas where the knowledge of former
Board members can be especially valuable in optimizing the input for
Board decisions in the future. 

"A number of Board
members have participated in up to 20 or more Peer Review meetings,
some of which cover multiple days. Current grant or loan requests
represent the result of scientific and medical advancement that has
been intensely vetted in prior peer reviews; the information gained
in those peer reviews should not be lost, when a subsequent grant or
loan request – built on the earlier research outcomes – is
considered. Each peer review session has the benefit of different
specialists and scientists and/or biotech representatives with
unique backgrounds and areas of expertise. The value of the prior
contributions may be pivotal, in considering a later application,
developed from the earlier medical or research advances funded
through CIRM’s grants or loans. The current peer review,
scientific staff presentation, and Board expertise, is not the limit
of the Board’s information, in reaching the best current decision.
To the extent the Board can draw from prior peer reviews (unique
insights), prior scientific staff presentations, and prior Board
expertise, additional information that can enhance a potential
decision, the Board has the opportunity to optimize its decision
making process. This is particularly valuable, when there is a high
standard deviation – a substantial split – in the scoring
positions from the current peer review. 

"Beyond peer review
participation, Board members have intensely engaged in another 35
plus Working Group sessions on Facilities and Standards, in addition
to more than 70 Board meetings and over 125 Subcommittee meetings,
as of August 2012. Retiring Board members possess a treasury of
information on policy development, process, federal and state laws
and regulations, and the regulations of the agency, as well as in
depth information on research facilities and capabilities throughout
California, the nation, and the world. It takes a substantial length
of time for a new Board member to gain a comprehensive knowledge in
all of these areas and each Board member will develop unique
insights, which it would be a tragedy to lose. As Chairman, I
frequently reached back to consult with former Board members on
areas of their special expertise and I would hope that all current
and future Board members utilize the significant asset in developed
knowledge of the prior Board members. To the extent prior members
can be available for public meetings, this would be a substantial
benefit to the agency to broadly inform the Board, the scientific
staff, and the public. 

"The Board has a
unique contribution to make on programmatic resource allocations and
risk management of the research and clinical investments in each
disease area. The opportunities in some disease areas for major
advancement are numerous, whereas there are major diseases and/or
critical research areas where the potential, high-value advancement
options are relatively limited. For Board members who have
participated in over 20 peer reviews and 70 Board meetings, the
programmatic perspective on the opportunities in each disease area
has been highly developed. Concurrently, those Board members or
former Board members have substantial knowledge that is of critical
value in reaching programmatic decisions on the number of
opportunities for advancement in any specific disease area and the
relative risk that needs to be taken to accomplish meaningful
breakthroughs in advancing the research and clinical opportunities
in a disease and/or injury area. 

"I hope these examples
of how former Board members can contribute to the current Board’s
information in reaching decisions on the best medical/scientific
grants and loans are helpful. As I stated earlier, it would be a
tragedy if the expertise of Board members built up over six or more
years is lost. The field is extremely complicated and the Board needs
the opportunity to consider all of the information available. The
Board can choose to accept or reject any past advice or opinions
gained from prior peer review sessions or Board meetings, but the
Board should have access to the full spectrum of information and the
treasury of scientific and medical advice the agency has received
since its inception.

"There are areas that I
have not addresses in this short response, such as the institutional
value of applicants being able to rely upon prior scientific and/or
policy direction, in their current applications. From a historical
perspective, prior Board members and/or the Chairman can have
significant information that is relevant to these evaluations,
especially if the individual Board member served on a special Task
Force , Subcommittee or peer review. These more complicated areas of
individual contribution by former Board members I can address in a
future communication; but, this specific subject – alone – could
comprise several pages and I would like to obtain critical advice and
perspective from other former Board members and the scientific
community before discussing this area in greater detail.
"Bob Klein
"Chair Emeritus
"California Institute
for Regenerative Medicine"

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

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Text of Klein’s Comments Supporting Rejected Applications

August 26th, 2012 3:54 pm


Robert Klein, who served 6 ½ years as the first chairman
of the $3 billion California stem cell agency,  testified before the agency's board for the first time on July 26, 2012. Klein, a real
estate investment banker and attorney, spoke on behalf of two applicants whose
grants had been rejected by the agency's reviewers. The appearance
has raised questions about the propriety of Klein's actions.

Here is a link to an item on his appearance. Here is the text of
his comments as reported in the transcript of the meeting.

“As the board knows, I've never addressed any grant from the
floor. It is critical here to understand that we have here
StemCells, Inc., which is the only company in North America
and, for that matter, maybe in the world, that has had two stem cell
therapies in the brain with these specific neural stem cells. They
have a huge body of experience here. 

“Secondly, one of the fundamental issues here that it (the
company's grant application) was downgraded on was the issue of the
fundamental concept, the platform concept, of injecting two focal
injections in the brain, in the hippocampus of the brain. It's
important to note that I've sat on three (CIRM)peer reviews where the
scientists really affirmed this specific approach with extremely high
scores, three different views. All right.

“So it's very important to realize we have a standard deviation
here of 12 (on the review scores). These scientists were completely
split. With some recusals on that panel, if you have 12 or 13 that
can really vote, three or four very low scores can bring it out of
the funding category all the way down. It is in the region where
this board is looking where the other three peer reviews, right,
early translation, the one before that was the planning grant review,
that the hippocampus was a good platform.

“Then they said the key weakness was you can't show migration.
Dr. Laferla (a co-PI on the application) has told me that
today the Journal of Neuroscience accepted the publication of
the data demonstrating migration. It was stated previously in the
application, but it wasn't accepted for publication. It now is.
That is the fundamental weakness that they identified in this
approach.  

“So we have a reaffirmed approach to the hippocampus by three
different peer review groups and a substantial portion of these
reviewers along with data dealing with the weak point. I'm sorry it
happened today. The data was out there, accepted for publication
today, means that it should definitely fall into this category. And,
of course, Dr. (Alan) Trounson (president of CIRM) wouldn't
have been able to review that in process because he was recused from
this grant by his own voluntary recusal. So the progress of this
data being accepted for publication is new information today. 

“If I look at the entire history of CIRM, as Leeza (Gibbons,
a CIRM director) says, building up to this point, we have reaffirmed
this approach from the very beginning with Dr. Laferla, with multiple
scientific approvals, and board approval, and we have the best
company in North America with the greatest experience with these
neural stem cells, with the best researcher we have for the potential
to address this disease, and we have brand-new data that demonstrates
and totally contradicts the key weakness on which it was downgraded.”

Here is the text of Klein's remarks on
behalf of a second application, also rejected by CIRM reviewers.

“This is the only other disease team grant I will address. Very
specifically, this was a disease team grant that I was on the peer
review in the planning grant stage. There are some fundamental
issues here. Is the international company on which the one antibody
that's not coming from Stanford, the two for sorting are
coming from Stanford, is the other antibody coming from this
international company a commitment that you can rely on? 

“The reviewers said this was a showstopper. That's the word they
used. They made a decision this was a showstopper because they did
not believe the company because they thought that the documentation
was inadequate. You now have a letter that goes into great
proprietary depth about the depth of this company's commitments
written by the head of development and translation internationally
for the company. 

“If we cannot depend on company commitments of this type, and
you will review the letter in executive session, if you have one, I
will not understand how we'll be able to collaborate with companies
with proprietary products and processes where they're making
commitments to academic institutions of the highest standard. I
believe this company is going to perform. I was on an hour call to
confirm with eight members of that company their level of commitment,
and I am completely convinced by that point. 

“The review is completely factually wrong on this issue about
the other two antibodies for sorting this. Dr. (Irv) Weissman
has just said they have not only been developed, they have been used
in clinical trials. There's data on them. And they are, in fact,
being thawed under FDA direction to reuse in this trial. 

“So I believe there's a major factual difference. Remember with
Karen Aboody there was a major factual error that was pivotal
in elevating that, and we found tremendous performance on that grant
by Karen Aboody of City of Hope

“So you have a decision to make. As a risk issue, do we believe
this company? Finally, this is broader than SCID. 

Donald Kohn has written a letter that's in the public
domain that I suggest you read. It makes it very clear that opening
the niche for repopulating the immune system without chemotherapy and
radiation is a key contribution to every form of genetically modified
stem cells for an entire range of childhood diseases and other
genetic diseases in addition to therapies like sickle cell or aids. 

“I suggest that that profound contribution that can be made to
the field is a risk that is worth taking early on because of his
contribution to so many other areas. You have 12 other letters from
North America's leading pediatric geneticists that fundamentally
provide extraordinary support for this position and this approach.”

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

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Hormel Institute receives $1.7 million for skin cancer study

August 25th, 2012 6:12 am

By Kay Fate The Post-Bulletin, Austin MN

AUSTIN A five-year federal grant totaling more than $1.7 million has been awarded for skin cancer research led by Dr. Rebecca Morris, leader of the stem cells and cancer section at the Hormel Institute.

Morris, whose research uses adult, non-human stem cells, has been working to identify stem cell-regulating genes for 10 years. She now has found a gene that appears to be linked to stem cell numbers and helping to protect against skin tumor development.

The gene also might have an immune function in the skin that can fight bacteria and protect against environmental damage. As stem cell research leads to discoveries to improve health, these cells can be obtained through various ways such as from adult tissue stem cells or through bio-engineering to avoid the ethical issues of how to secure stem cells for treatment.

The new project, funded through 2017, will further Morris' research on that gene and the idea that, through using the gene, stem cells could be used to function as a way to protect the body.

The down side of stem cells is that they seem to be targets for cancer development, Morris said.

This is exciting for us because a gene whose function is to protect may do double duty by regulating stem cell activity related to the immune system, she said. If we can identify this gene, then maybe we could find a way to turn off the stem cells if theyre growing too fast, such as in cancer and other diseases involving too rapid cell growth.

With the title Identification of a Keratinocyte Stem Cell Regulatory Gene in the KSC2 Locus, the project is funded through the National Institute of Arthritis and Musculoskeletal and Skin Diseases, part of the National Institutes of Health.

The key is to find the genes that control the number and growth potential of epidermal stem cells, Morris said. Then scientists could create more stem cells or make them grow faster, for example, to heal an ulcer or the thinning of skin from aging.

Morris, who joined the institute in 2008, focuses her research on stem cells responsible for healing wounds, maintaining normal tissue integrity and cancer. Her lab uses adult stems cells isolated from the skin through a technique Morris developed when she was a post-doctorate at MD Anderson Cancer Center in Houston.

See the rest here:
Hormel Institute receives $1.7 million for skin cancer study

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New partnership to drive mass production of life-saving stem cells – Commercialization Impact Prize is first of its …

August 25th, 2012 6:12 am

TORONTO, ON Stem cells hold great promise for treating and curing numerous diseases; however, a major challenge facing scientists is how to produce stem cells in the massive quantities required for clinical use. The McEwen Centre for Regenerative Medicine (McEwen Centre) and the University of Toronto-based Centre for Commercialization of Regenerative Medicine (CCRM) are partnering to establish a fund that will drive research in this area. Several University of Toronto regenerative medicine scientists are affiliated with CCRM and the Scientific Director is Dr. Peter Zandstra of the Institute of Biomaterials and Biomedical Engineering.

The McEwen Centre-CCRM Commercialization Impact Prize launches today, and will solicit innovative ideas from regenerative medicine scientists working in labs throughout the McEwen Centre. The winning team(s) will be awarded up to $600,000 to pursue research that will determine how to manufacture stem cells for clinical use and drug screening.

This private-public funding partnership is an important step forward to accelerating the advance of a discovery from a lab bench to the patient and onto the global market. Scientists at the McEwen Centre are making significant progress towards finding a cure for diseases such as Type 1 diabetes and heart disease. Collaborative partnerships are the key to discovering the cures sooner! says Rob McEwen, co-founder of the McEwen Centre, and Chief Owner, McEwen Mining.

Deadline for submissions is October 15, 2012. The Prize will fund up to two, 2-year projects that address the following challenges:

Making the transition from pre-clinical to clinical mass production; and, Scaling up stem cell manufacturing for high throughput drug screening.

Overcoming the scale-up and manufacturing challenge of stem cells would be a huge advancement for the regenerative medicine [RM] industry and this initiative fits in perfectly with our mandate to bridge the RM commercialization gap, explains Dr. Michael May, CEO of the Centre for Commercialization of Regenerative Medicine. Were very pleased to be working with the McEwen Centre, already a partner of ours, to make this happen.

The Commercialization Impact Prize budget template and application form can be found here: http://ccrm.ca/Commercialization-Impact-Prize or http://mcewencentre.com/ccrm

About McEwen Centre for Regenerative Medicine The McEwen Centre for Regenerative Medicine was founded by Rob and Cheryl McEwen in 2003 and opened its doors in 2006. The McEwen Centre for Regenerative Medicine, part of Toronto-based University Health Network, is a world leading centre for stem cell research, facilitating collaboration between renowned scientists from 5 major hospitals in Toronto, the University of Toronto and around the world. Supported by philanthropic contributions and research grants, McEwen Centre scientists strive to introduce novel regenerative therapies for debilitating and life threatening illnesses including heart disease, spinal cord injury, diabetes, diseases of the blood, liver and arthritis.

About Centre for Commercialization of Regenerative Medicine (CCRM) CCRM, a Canadian not-for-profit organization funded by the Government of Canadas Networks of Centres of Excellence program and six institutional partners, supports the development of technologies that accelerate the commercialization of stem cell- and biomaterials-based technologies and therapies. A network of academics, industry and entrepreneurs, CCRM translates scientific discoveries into marketable products for patients. CCRM launched in Torontos Discovery District on June 14, 2011.

Read this article:
New partnership to drive mass production of life-saving stem cells - Commercialization Impact Prize is first of its ...

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Institute receives funding for skin cancer study

August 24th, 2012 8:20 pm

By Kay Fate The Post-Bulletin, Austin MN

A five-year federal grant totaling more than $1.7 million has been awarded for skin cancer research led by Dr. Rebecca Morris, leader of the stem cells and cancer section at the Hormel Institute.

Morris, whose research uses adult, non-human stem cells, has been working to identify stem cell-regulating genes for 10 years. She now has found a gene that appears to be linked to stem cell numbers and helping to protect against skin tumor development.

The gene also might have an immune function in the skin that can fight bacteria and protect against environmental damage. As stem cell research leads to discoveries to improve health, these cells can be obtained through various ways such as from adult tissue stem cells or through bio-engineering to avoid the ethical issues of how to secure stem cells for treatment.

The new project, funded through 2017, will further Morris' research on that gene and the idea that, through using the gene, stem cells could be used to function as a way to protect the body.

The down side of stem cells is that they seem to be targets for cancer development, Morris said.

This is exciting for us because a gene whose function is to protect may do double duty by regulating stem cell activity related to the immune system, she said. If we can identify this gene, then maybe we could find a way to turn off the stem cells if theyre growing too fast, such as in cancer and other diseases involving too rapid cell growth.

With the title Identification of a Keratinocyte Stem Cell Regulatory Gene in the KSC2 Locus, the project is funded through the National Institute of Arthritis and Musculoskeletal and Skin Diseases, part of the National Institutes of Health.

The key is to find the genes that control the number and growth potential of epidermal stem cells, Morris said. Then scientists could create more stem cells or make them grow faster, for example, to heal an ulcer or the thinning of skin from aging.

Morris, who joined the institute in 2008, focuses her research on stem cells responsible for healing wounds, maintaining normal tissue integrity and cancer. Her lab uses adult stems cells isolated from the skin through a technique Morris developed when she was a post-doctorate at MD Anderson Cancer Center in Houston.

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Institute receives funding for skin cancer study

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Autism And Cord Blood Stem Cells: FDA Gives Green Light For Groundbreaking Clinical Trial

August 24th, 2012 9:18 am

Sacramento, CA /PRNewswire/ Sutter Neuroscience Institute, a recognized Center of Excellence, andCBR(Cord Blood Registry), the worlds largest stem cell bank, are launching the firstFDA- approved clinical trial to assess the use of a childs own cord blood stem cells to treat select patients with autism. This first-of-its-kind placebo controlled study will evaluate the ability of an infusion of cord blood stem cells to help improve language and behavior. The study is in conjunction with the Sutter Institute for Medical Research.

According to the Centers for Disease Control and Prevention, autism spectrum disorders impact one in 88 children in the U.S., and one in 54 boys.1The condition is thought to have multiple risk factors including genetic, environmental and immunological components.

This is the start of a new age of research in stem cell therapies for chronic diseases such as autism, and a natural step to determine whether patients receive some benefit from an infusion of their own cord blood stem cells, said Michael Chez, M.D., director of Pediatric Neurology with the Sutter Neuroscience and principal study investigator. I will focus on a select portion of children diagnosed with autism who have no obvious cause for the condition, such as known genetic syndromes or brain injury.

The study will enroll 30 children between the ages of two and seven, with a diagnosis of autism who meet theinclusion criteria for the study. Enrolled participants will receive two infusionsone of the childs own cord blood stem cells and one of a placeboover the course of 13 months. Both the participants and the lead investigators will be blinded from knowing the content of each infusion. To ensure the highest quality and consistency in cord blood stem cell processing, storage and release for infusion,CBRis the only family stem cell bank providing units from clients for the study.

For information on study, visithttp://www.cordblood.com/autism.

Study Rationale A newborns umbilical cord blood contains a unique population of stem cells that have been used for more than 20 years in medical practice to treat certain cancers, blood diseases and immune disorders. When patients undergo a stem cell transplant for these conditions, the stem cells effectively rebuild the blood and immune systems.

A focus of my research has been the complex relationship between a childs immune system and central nervous system. We have evidence to suggest that certain children with autism have dysfunctional immune systems that may be damaging or delaying the development of the nervous system, continued Dr. Chez. Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients which would also improve language and some behavior in children who have no obvious reason to have become autistic. The study is similar to otherFDA-approved clinical trials looking at cord blood stem cells as a therapy for cerebral palsy.

Its exciting to partner with thought-leading medical researchers and clinicians, like Dr. Chez, who are pursuing a scientifically-sound approach in evaluating new therapeutic uses for cord blood stem cells for conditions that currently have no cures, said Heather Brown, vice president of scientific & medical affairs atCBR. Families who made the decision to bank their stem cells to cover the unknowns and what ifs in life are gaining access to this and other important clinical trials while playing an important role in the advancement of science.

The co-investigator of the study is Michael Carroll, M.D., medical director of the Blood and Marrow Transplantation and Hematological Malignancies Program at Sutter Medical Center, Sacramento.

There is a vast amount of unchartered territory when it comes to how stem cell therapies may help patients living with these conditions, said Dr. Carroll. Ive seen how stem cell therapy has changed my field of medicine and how I care for my blood cancer patients. I am eager to see how our work can open new doors for patients and families dealing with autism.

Excerpt from:
Autism And Cord Blood Stem Cells: FDA Gives Green Light For Groundbreaking Clinical Trial

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Human melanoma stem cells identified

August 24th, 2012 9:18 am

ScienceDaily (Aug. 23, 2012) Cancer stem cells are defined by three abilities: differentiation, self-renewal and their ability to seed a tumor. These stem cells resist chemotherapy and many researchers posit their role in relapse. A University of Colorado Cancer Center study recently published in the journal Stem Cells, shows that melanoma cells with these abilities are marked by the enzyme ALDH, and imagines new therapies to target high-ALDH cells, potentially weeding the body of these most dangerous cancer creators.

"We've seen ALDH as a stem cell marker in other cancer types, but not in melanoma, and until now its function has been largely unknown," says the paper's senior author, Mayumi Fujita, MD, PhD, investigator at the CU Cancer Center and associate professor in the Department of Dermatology at the CU School of Medicine.

Fujita's group transplanted ALDH+ and ALDH- melanoma cells into animal models, showing the ALDH+ cells were much more powerfully tumorigenic. In the same ALDH+ cells, the group then silenced the gene that creates this protein, finding that with ALDH knocked down, melanoma cells died in cultures and lost their ability to form tumors in animal models. In cell cultures, silencing this ALDH gene also sensitized melanoma cells to existing chemotherapies. When the group explored human tumor samples, they found distinct subpopulations of these ALDH+ cells, which made up about 0.1-0.2 percent of patients' primary tumors. In samples of metastatic melanoma -- the most aggressive form of the disease -- the percentage of ALDH+ cells was greater, even over 10 percent in some tumors, further implying the powerful danger of these cells.

"In these same ALDH+ cells, we find the markers of stem cells are upregulated and those of cell differentiation are downregulated. In addition to these clues, ALDH+ cells generate the heterogeneous cell types seen in the original tumor," says Fujita, meaning that in addition to self-renewal and tumorigenesis, ALDH+ cells fulfill the third criteria for a cancer stem cell: the ability to differentiate.

The study also shows how the ALDH gene and its protein act to create a cell's stem-like properties.

"One way ALDH makes a cancer stem cell is through the retinoic acid signaling pathway," Fujita says. The protein ALDH leads to the overproduction of retinoic acid, which in turn binds to a cell's nuclear receptors and influences the expression of many of the cell's genes -- for example, genes involved in regulating cell survival, repair, and proliferation, all of which combine to confer chemoresistance. Target cells with high ALDH and you target all the downstream effects, including the retinoic acid signaling pathway.

"Our hope is that we can intervene in this signaling, either at the level of ALDH or elsewhere in the pathway, especially to re-sensitize cells to chemotherapy. Using a new drug to take away a melanoma stem cell's chemoresistance could boost the effectiveness of existing drugs," Fujita says.

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Astrocytes control the generation of new neurons from neural stem cells

August 24th, 2012 9:18 am

24.08.2012 - (idw) University of Gothenburg

Researchers from the Laboratory of astrocyte biology and CNS regeneration headed by Prof. Milos Pekny just published a research article in a prestigious journal Stem Cells on the molecular mechanism that controls generation of new neurons in the brain. Astrocytes are cells that have many functions in the central nervous system, such as the control of neuronal synapses, blood flow, or the brains response to neurotrauma or stroke. Reduces brain tissue damage

Prof. Peknys laboratory together with collaborators have earlier demonstrated that astrocytes reduce the brain tissue damage after stroke and that the integration of transplanted neural stem cells can be largely improved by modulating the activity of astrocytes. Generation of new neurons

In their current study, the Sahlgrenska Academy researchers show how astrocytes control the generation of new neurons in the brain. An important contribution to this project came from bo Academy, one of Sahlgrenskas traditional collaborative partners.

In the brain, astrocytes control how many new neurons are formed from neural stem cells and survive to integrate into the existing neuronal networks. Astrocytes do this by secreting specific molecules but also by much less understood direct cell-cell interactions with stem cells, says Prof. Milos Pekny. Important regulator

Astrocytes are in physical contact with neural stem cells and we have shown that they signal through the Notch pathway to stem cells to keep the birth rate of new neurons low. We have also shown that the intermediate filament system of astrocytes is an important regulator of this process. It seems that astrocyte intermediate filaments can be used as a target to increase the birthrate of new neurons. Target for future therapies

The article Astrocytes Negatively Regulate Neurogenesis through the Jagged1-Mediated Notch Pathway is published in Stem Cells. 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 Weitere Informationen: http://bit.ly/NCJEdI - article

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New Partnership to Drive Mass Production of Life-Saving Stem Cells

August 24th, 2012 9:18 am

TORONTO, ONTARIO--(Marketwire -08/23/12)- Stem cells hold great promise for treating and curing numerous diseases; however, a major challenge facing scientists is how to produce stem cells in the massive quantities required for clinical use. The McEwen Centre for Regenerative Medicine (McEwen Centre) and the Centre for Commercialization of Regenerative Medicine (CCRM) are partnering to establish a fund that will drive research in this area.

The McEwen Centre-CCRM Commercialization Impact Prize launches today, and will solicit innovative ideas from regenerative medicine scientists working in labs throughout the McEwen Centre. The winning team(s) will be awarded up to $600,000 to pursue research that will determine how to manufacture stem cells for clinical use and drug screening.

"This private-public funding partnership is an important step forward to accelerating the advance of a discovery from a lab bench to the patient and onto the global market. Scientists at the McEwen Centre are making significant progress towards finding a cure for diseases such as Type 1 diabetes and heart disease. Collaborative partnerships are the key to discovering the cures sooner!" says Rob McEwen, co-founder of the McEwen Centre, and Chief Owner, McEwen Mining.

Deadline for submissions is October 15, 2012. The Prize will fund up to two, 2-year projects that address the following challenges:

"Overcoming the scale-up and manufacturing challenge of stem cells would be a huge advancement for the regenerative medicine (RM) industry and this initiative fits in perfectly with our mandate to bridge the RM commercialization gap," explains Dr. Michael May, CEO of the Centre for Commercialization of Regenerative Medicine. "We're very pleased to be working with the McEwen Centre, already a partner of ours, to make this happen."

The Commercialization Impact Prize budget template and application form can be found here: http://ccrm.ca/Commercialization-Impact-Prize or http://mcewencentre.com/ccrm.

About McEwen Centre for Regenerative Medicine

The McEwen Centre for Regenerative Medicine was founded by Rob and Cheryl McEwen in 2003 and opened its doors in 2006. The McEwen Centre for Regenerative Medicine, part of Toronto-based University Health Network, is a world leading centre for stem cell research, facilitating collaboration between renowned scientists from 5 major hospitals in Toronto, the University of Toronto and around the world. Supported by philanthropic contributions and research grants, McEwen Centre scientists strive to introduce novel regenerative therapies for debilitating and life threatening illnesses including heart disease, spinal cord injury, diabetes, diseases of the blood, liver and arthritis.

About Centre for Commercialization of Regenerative Medicine (CCRM)

CCRM, a Canadian not-for-profit organization funded by the Government of Canada's Networks of Centres of Excellence program and six institutional partners, supports the development of technologies that accelerate the commercialization of stem cell- and biomaterials-based technologies and therapies. A network of academics, industry and entrepreneurs, CCRM translates scientific discoveries into marketable products for patients. CCRM launched in Toronto's Discovery District on June 14, 2011.

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Repairing cartilage with fat: Problems and potential solutions

August 24th, 2012 9:18 am

Public release date: 23-Aug-2012 [ | E-mail | Share ]

Contact: Hilary Glover hilary.glover@biomedcentral.com 44-020-319-22370 BioMed Central

Stem cells isolated from fat are being considered as an option for treating tissue damage and diseases because of their accessibility and lack of rejection. New research published in BioMed Central's open access journal Stem Cell Research & Therapy shows that this is not as straightforward as previously believed, and that fat-derived stem cells secrete VEGF and other factors, which can inhibit cartilage regeneration. However pre-treating the cells with antibodies against VEGF and growing them in nutrients specifically designed to promote chondrocytes can neutralize these effects.

Chondrocytes make and maintain healthy cartilage but damage and disease including osteoarthritis can destroy cartilage resulting in pain and lack of mobility. Stem cell therapy using cells isolated from adult tissue (such as fat) are being investigated as a way of repairing this damage. Stem cells have the ability to become many different types of tissue so the real trick is persuading them to become cartilage rather than bone, or blood vessels, for example.

Researchers from the Georgia Institute of Technology found that adipose (fat) stem cells (ASCs) secrete large amounts of factors, especially the growth factor VEGF, which prevent cartilage regeneration and actually causes the death (apoptosis) of chondrocytes along with the formation of blood vessels. Treating ASCs with medium designed to encourage their differentiation into cartilage cells was able to reduce the amount of these secreted factors and also prevented the growth of blood vessels. Specifically, an antibody designed to neutralize VEGF prevented chondrocyte apoptosis.

Prof Barbara Boyan, who led this research, explained, "Non-treated ASCs actually impeded healing of hyaline cartilage defects, and although treating ASCs improved the situation they added no benefit to compared to cartilage allowed to heal on its own. However we only looked at cartilage repair for a week after treatment, and other people have shown that two to six weeks is required before the positive effect of ASCs on influence cartilage regeneration is seen."

So while stem cells from fat may be able to help repair damaged cartilage, careful handling and pre-treatment may be required to ensure a positive result.

###

Media contact

Dr Hilary Glover Scientific Press Officer, BioMed Central Tel: +44 (0) 20 3192 2370 Mob: +44 (0) 778 698 1967 Email: hilary.glover@biomedcentral.com

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Study Identifies Human Melanoma Stem Cells

August 24th, 2012 9:18 am

Cancer stem cells are defined by three abilities: differentiation, self-renewal and their ability to seed a tumor. These stem cells resist chemotherapy and many researchers posit their role in relapse. A University of Colorado Cancer Center study recently published in the journal Stem Cells, shows that melanoma cells with these abilities are marked by the enzyme ALDH, and imagines new therapies to target high-ALDH cells, potentially weeding the body of these most dangerous cancer creators.

Weve seen ALDH as a stem cell marker in other cancer types, but not in melanoma, and until now its function has been largely unknown, says the papers senior author, Mayumi Fujita, MD, PhD, investigator at the CU Cancer Center and associate professor in the Department of Dermatology at the CU School of Medicine.

KEY POINTS:

Fujitas group transplanted ALDH+ and ALDH- melanoma cells into animal models, showing the ALDH+ cells were much more powerfully tumorigenic. In the same ALDH+ cells, the group then silenced the gene that creates this protein, finding that with ALDH knocked down, melanoma cells died in cultures and lost their ability to form tumors in animal models. In cell cultures, silencing this ALDH gene also sensitized melanoma cells to existing chemotherapies. When the group explored human tumor samples, they found distinct subpopulations of these ALDH+ cells, which made up about 0.1-0.2 percent of patients primary tumors. In samples of metastatic melanoma the most aggressive form of the disease the percentage of ALDH+ cells was greater, even over 10 percent in some tumors, further implying the powerful danger of these cells.

In these same ALDH+ cells, we find the markers of stem cells are upregulated and those of cell differentiation are downregulated. In addition to these clues, ALDH+ cells generate the heterogeneous cell types seen in the original tumor, says Fujita, meaning that in addition to self-renewal and tumorigenesis, ALDH+ cells fulfill the third criteria for a cancer stem cell: the ability to differentiate.

The study also shows how the ALDH gene and its protein act to create a cells stem-like properties.

One way ALDH makes a cancer stem cell is through the retinoic acid signaling pathway, Fujita says. The protein ALDH leads to the overproduction of retinoic acid, which in turn binds to a cells nuclear receptors and influences the expression of many of the cells genes for example, genes involved in regulating cell survival, repair, and proliferation, all of which combine to confer chemoresistance. Target cells with high ALDH and you target all the downstream effects, including the retinoic acid signaling pathway.

Our hope is that we can intervene in this signaling, either at the level of ALDH or elsewhere in the pathway, especially to re-sensitize cells to chemotherapy. Using a new drug to take away a melanoma stem cells chemoresistance could boost the effectiveness of existing drugs, Fujita says.

SOURCE: University of Colorado Denver

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Companion Diagnostics and Personalized Medicine Market Report 2012: Twease.org

August 24th, 2012 9:18 am

ALBANY, New York, Aug. 23, 2012 /PRNewswire/ -- Twease.org - New Report Added in Pharmaceuticals Reports Database Companion Diagnostics and Personalized Medicine Market Report 2012 [http://www.twease.org/report/companion-diagnostics-and-personalized-medicine-market-report-2012.htm]

This is the latest and most up-to-date Market Report from Select Biosciences addressing the companion diagnostics (CDx) and personalized medicine marketplace. Personalized medicine is a broad field with several stakeholders all of which must be aligned in order to capture the immense potential value in targeting therapeutics to the correct patient populationthe field of stratified medicine.

To Browse Full Toc Visit: http://www.twease.org/report/companion-diagnostics-and-personalized-medicine-market-report-2012.htm [http://www.twease.org/report/companion-diagnostics-and-personalized-medicine-market-report-2012.htm]

Companion Diagnostics has been rapidly expanding over the past 3 years and in this market report we describe the current state of the marketplace from the following perspectives:

Related Reports:

Personalized Medicine Market [http://www.twease.org/report/companion-diagnostics-and-personalized-medicine-market-report-2012.htm]

MicroRNAs and Exosomes Market [http://www.twease.org/report/micrornas-and-exosomes-market-report-2012.htm]

MicroRNA Market [http://www.twease.org/report/microrna-market-trends-2011.htm]

MicroRNA Research and Disease Associations [http://www.twease.org/report/microrna-research-and-disease-associations-2010-market-report.htm]

Circulating Tumor Cells [http://www.twease.org/report/circulating-tumor-cells-ctcs-and-cancer-stem-cells-cscs-market-global-industry-size-market-share-trends-analysis-and-forecasts-2012-2018.htm]

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Stem Cell Research: Heart Stem Cells May Help Heal Hearts After Heart Attack

August 22nd, 2012 5:14 pm

Two men in landmark heart stem cell study tell their stories.

Jim Dearing of Louisville, Ky., one of the first men in the world to receive heart stem cells, might have helped start a medical revolution that could lead to a cure for heart failure.

Three years after getting the experimental stem cell procedure, following two heart attacks and heart failure, Dearings heart is working normally.

2012 WebMD, LLC. All rights reserved.

The difference is clear and dramatic -- and it's lasting, according to findings now being made public for the first time.

Dearing first showed "completely normal heart function" on an echocardiogram done in 2011, says Roberto Bolli, MD, who is leading the stem cell trial at the University of Louisville. Those results have not been published before.

That was still true in July 2012, when Dearing again showed normal heart function on another echocardiogram.

Based on those tests, Bolli says, "Anyone who looks at his heart now would not imagine that this patient was in heart failure, that he had a heart attack, that he was in the hospital, that he had surgery, and everything else."

It's not just Dearing who has benefited. His friend, Mike Jones, who had even more severe heart damage, also got the stem cell procedure in 2009. Since then, scarred regions of his heart have shrunk. His heart now appears leaner and stronger than it was before.

"What's striking and exciting is that we're seeing what appears to be a long-lasting improvement in function," Bolli says. If larger studies confirm the findings, "potentially, we have a cure for heart failure because we have something that for the first time can actually regenerate dead tissue."

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Stem Cells Could Reverse Incontinence

August 22nd, 2012 5:14 pm

Researchers report that stem cells have reversed bladder leakage in mice, and that the discovery could pave the way for new treatments against urinary incontinence.

The study, conducted at Kyungpook National University in South Korea, found that weakened pelvic-floor muscles in mice were repaired with stem cells made from amniotic fluid. The stem cells also kept the condition from recurring, even though the cells disappeared after 14 days in the body.

Urinary incontinence will affect one out of every three women after age 40. Although men may also have the condition, the frequency is much lower. Treatments for urinary incontinence include surgery, lifestyle changes like weight loss, and exercises to strengthen pelvic muscles.

Previously, stem cell therapy has been suggested as a possibility for treating urinary incontinence, but the only way to gather the cells was through invasive procedures. Collecting stem cells from amniotic fluid is easier during a routine procedure of amniocentesis.

"These stem cells ... have the ability to become muscle cells when grown under the right conditions," study leaders James Yoo and Tae Gyun Kwon said in a statement.

Testing on people, though, is needed to back up the researchers findings.

The study was published in the journal BMC Medicine.

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Research and Markets: Circulating Tumor Cells (Ctcs) And Cancer Stem Cells (Cscs) Market – Global Industry Size …

August 22nd, 2012 5:14 pm

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/khwrrx/circulating_tumor) has announced the addition of the "Circulating Tumor Cells (Ctcs) And Cancer Stem Cells (Cscs) Market - Global Industry Size, Market Share, Trends, Analysis, And Forecasts 2012 - 2018" report to their offering.

The rising prevalence of diseases like cancer and the reimbursement support by regulatory bodies in developed countries like United States and Europe are the major factors driving the growth of the CTCs and CSCs market. Though the currently used detection method lacks sensitivity or specificity to track all CTCs particularly the ones that have lost characteristic epithelial features, there is still good scope for pharmaceutical companies in the CTCs and CSCs field. The various sub-types of cancer may have their own classes and it creates an opportunity in the future.

Increase in cancer mortality rate in the past few years and an increase in number of cancer patients offers an opportunity for pharmaceutical companies to enter this sector. Every one person out of eight has the potential of getting affected by cancer and it is estimated that 12 to 37 lives can be saved daily with the help of CTCs and CSCs.

The major geographic markets for CTCs and CSCs are the U.S. and Europe. The U.S. accounted for more than 50% of the worldwide CTCs and CSC market in 2011.

This research is specially designed to estimate and analyze the demand and performance of CTCs and CSC products in a global scenario. The report covers all the major segments of the global CTC and CSC market and provides in-depth analysis, historical data and statistically refined forecast for the segments covered. The study presents a comprehensive assessment of the stakeholder strategies and winning imperatives for them by segmenting the global CTC and CSC market.

Key Topics Covered:

1. Introduction

2. Executive Summary

3. Market Overview

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Doctors to study newborn stem cells as treatment for autism

August 22nd, 2012 5:13 pm

SACRAMENTO, CA - At 4-years-old Rydr Rudgers is able to eat, speak, and walk --all thingshis family wasn't sure he'd ever do after being diagnosed with cerebral palsy as an infant.

"He was born without any brain stem functions; no sucking, no swallowing, no breathing," said Rydr's mother Elisa.

When Rydr was 15-months-old, he began stem cell infusions from his cord blood that was saved in a stem cell bank.Rydris making great progress after three infusionsand can even feed himself.

"These are like huge milestones that people don't think about, but actually being able to hold a fork and eat a sandwich is, in our world, an unanticipated milestone and it's amazing," Elisa Rudgers explained.

"Like autism, cerebral palsy or brain injuries of that nature are a diffused population, it's not one cause,"said Dr. Michael Chez, who is the Medical Director of Pediatric Neurology at the Sutter Neuroscience Institute.

Doctors at the Sutter Neuroscience Institute are now beginning research to evaluate cord blood stem cells to help improve language and behavior in autism patients.

The announcement was made on Tuesday morning at Sutter Medical Plaza.It's the first FDA-approved clinical trial that uses a newborn's stem cells from cord blood to treat autism patients.

Doctors will infuse umbilical cord stem cells into the bloodstreams of 30 children diagnosed with autism.

"We feel it will offer a safe and effective answer to the question of whether the cord blood is an effective intervention as a way to introduce stem cell therapy for autism," Chez said.

Autism impacts one in 88 children and one in 54 boys. According to Sutter doctors, a newborn's umbilical cord blood contains a unique population of stem cells that have been used for more than 20 years in medical practice.

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Stem Cells Fix Bladder Leakage in Mice, Study Finds

August 22nd, 2012 5:13 pm

TUESDAY, Aug. 21 (HealthDay News) -- Stem cells from amniotic fluid might one day help treat stress urinary incontinence, a condition caused by damaged pelvic floor muscles in which bladder leakage is brought on by exercise, coughing or simply laughing, a new study involving mice suggests.

Researchers in Korea found that this new technique repaired the damaged pelvic floor muscles in the mice, and kept the condition from recurring.

Results obtained in animal studies do not necessarily apply to humans, however, and much more research is needed before this might be considered a viable treatment for people.

Looking for an alternative to surgery to treat stress urinary incontinence, the scientists explored the use of stem cells to repair the weak muscles that cause bladder leakage. To do this in a noninvasive way, they used stem cells from amniotic fluid collected during routine amniocentesis (prenatal testing of amniotic fluid).

"These stem cells ... have the ability to become muscle cells when grown under the right conditions," explained the study's leaders, James Yoo and Tae Gyun Kwon, from Kyungpook National University, in a news release. "We found that the stem cells were able to survive for seven days inside the mice but by 14 days they had all disappeared. Nevertheless, they were able to induce regeneration of the mouse's own urethral sphincter muscle."

The study revealed the stem cells were able to strengthen the muscles of the pelvic floor. This regenerated muscle also had the right nerve connections. The researchers added that amniotic stem cells do not appear to cause an immune response, such as rejection or tumor growth. Exactly how the stem cells are able to regenerate the muscles remains unclear, they noted.

Stress urinary incontinence is common among women during and after pregnancy and in women aged 40 and older. The study authors noted that men also can develop the condition, particularly those who have undergone prostate surgery.

Current treatments for stress urinary incontinence include a combination of surgery, weight loss, pelvic floor exercises and bladder training.

The study was published Aug. 20 in the journal BMC Medicine.

-- Mary Elizabeth Dallas

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Tissue engineering: The brainmaker

August 22nd, 2012 5:13 pm

HANS SAUTTER

In December 2010, Robin Ali became suddenly excited by the usually mundane task of reviewing a scientific paper. I was running around my room, waving the manuscript, he recalls. The paper described how a clump of embryonic stem cells had grown into a rounded goblet of retinal tissue. The structure, called an optic cup, forms the back of the eye in a growing embryo. But this one was in a dish, and videos accompanying the paper showed the structure slowly sprouting and blossoming. For Ali, an ophthalmologist at University College London who has devoted two decades to repairing vision, the implications were immediate. It was clear to me it was a landmark paper, he says. He has transformed the field.

'He' is Yoshiki Sasai, a stem-cell biologist at the RIKEN Center for Developmental Biology in Kobe, Japan. Sasai has impressed many researchers with his green-fingered talent for coaxing neural stem cells to grow into elaborate structures. As well as the optic cup1, he has cultivated the delicate tissue layers of the cerebral cortex2 and a rudimentary, hormone-making pituitary gland3. He is now well on the way to growing a cerebellum4 the brain structure that coordinates movement and balance. These papers make for the most addictive series of stem-cell papers in recent years, says Luc Leyns, a stem-cell scientist at the Free University of Brussels.

Sasai's work is more than tissue engineering: it tackles questions that have puzzled developmental biologists for decades. How do the proliferating stem cells of an embryo organize themselves seamlessly into the complex structures of the body and brain? And is tissue formation driven by a genetic program intrinsic to cells, or shaped by external cues from neighbouring tissues? By combining intuition with patient trial and error, Sasai has found that it takes a delicate balance of both: he concocts controlled environments that feed cells physical and chemical signals, but also gives them free rein to 'do their thing' and organize themselves into issues. He sometimes refers to himself as a Japanese matchmaker who knows that, having been brought together, two strangers need to be left alone. They know what to do, he says. They interact in a delicate manner, and if the external cues are too strong, it will override the internal ones.

Sasai's work could find medical applications. Recapitulating embryonic development in three dimensions, it turns out, generates clinically useful cells such as photoreceptors more abundantly and efficiently than two-dimensional culture can, and houses them in an architecture that mirrors that of the human body. Sasai and his collaborators are now racing to implant lab-grown retinas into mice, monkeys and humans. The way Sasai sees it, maturing stem cells in two-dimensional culture may lead to 'next generation' therapy but his methods will lead to 'next, next generation' therapy.

A bit stiff in movement and reserved in manner, Sasai nevertheless puts on a theatrical show with a cocktail shaker at parties held by his institute after international symposia. My second job is bartender, he says, without a trace of a smile. It is, however, the cocktails he mixes in 96-well culture plates that have earned him scientific acclaim.

Like many members of his family, Sasai studied medicine. But he soon became frustrated by the lack of basic understanding in the field, especially when it came to neurological conditions. Without knowing the brain, a doctor cannot do much for the patient and therapeutics will always be superficial, he recalls thinking. There seemed no better way to know the brain than to study how it emerges and folds in the embryo. It's complex and usually complex systems are messy, says Sasai. But it's one of the most ordered. He wanted to know how this elaborate system was controlled.

We set up the permissive conditions. But after that we don't do anything. Keep them growing and let them do their job.

One piece of the puzzle was well known: the Spemann organizer, a node in vertebrate embryos that induces surrounding cells to become neural tissue. How the organizer works had been a mystery since its discovery in 1924; to find out, Sasai accepted a postdoctoral position at the University of California, Los Angeles. The post got off to a difficult start when Sasai was robbed of his money and passports at the airport on his way to California. But his scientific efforts were soon rewarded. He replaced the passports and within a month produced the clones that gave us the famous gene chordin, says his supervisor, developmental biologist Eddy De Robertis.

Sasai and his colleagues discovered that the chordin protein is a key developmental signal released by the Spemann organizer5. Rather than pushing nearby cells to become neurons, they found, chordin blocks signals that would turn them into other cell types6, 7. The work helped to establish the default model of neural induction: the idea that, without other signals, embryonic cells will follow an internal program to become neural cells.

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Sutter Neuroscience Institute launching trial of cord blood stem cells in autistic children

August 21st, 2012 4:14 pm

The Sutter Neuroscience Institute in Sacramento plans to launch groundbreaking research today to discover whether infusing umbilical cord stem cells into the bloodstreams of autistic children will help them overcome debilitating characteristics of the condition.

The clinical trial is the first of its kind to be approved by the U.S. Food and Drug Administration, said Dr. Michael Chez, the principal investigator and Sutter's director of pediatric neurology.

One of the world's foremost autism experts at the institute, which is recognized for its practice of pediatric neuroscience, Chez said the research may help identify a valuable new tool in the struggle against autism spectrum disorders, which now affect about one in 88 children nationwide, and one in 54 boys.

"This is an exciting trial, because it's exposing us to the new frontier of stem cells and whether they may have some positive effect on this disease," Chez said. "This is the start of a new age of research in stem cell therapies for chronic diseases such as autism."

Recent research has revealed robust new uses for stem cells. Stem cells, for example, can now be developed from heart muscle in cardiac patients and injected back into the heart for improved functioning. Doctors are also having success using stem cells to treat leukemia and bone marrow diseases.

Autism is the leading cause of delayed development in children, typically surfacing before 3 years of age. The condition is characterized by impaired communication, repetitive thoughts and behavior and difficulty in socialization.

The likelihood of a child's being given a diagnosis of an autism spectrum disorder increased more than 20 percent from 2006 to 2008, according to a report this year by the Centers for Disease Control and Prevention. Locally, more than 3,830 students in the four-county Sacramento region were counted as autistic in December 2011, up 13 percent from the previous year, according to state data.

The Sutter clinical trial follows promising research in using cord blood stem cells to help children with cerebral palsy, a brain disorder that shares some characteristics with autism, to improve motor function.

Chez became the point man for developing protocols for the clinical trial over a year ago, after being contacted by Cord Blood Registry, the world's largest cord blood bank. The Bay Area company has collected and frozen 400,000 samples of umbilical cord blood for individuals and families.

The umbilical cord that links a mother's placenta to her fetus is imbued with blood stem cells. Since the 1990s, parents increasingly have opted to have their babies' umbilical cords frozen in storage as a sort of insurance against future disease. The trend now extends to about 5 percent of parents.

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