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California Stem Cell Agency First: Big Pharma Hook Up

Posted: October 28, 2012 at 7:59 am


BURLINGAME, Ca. – For the first
time, a Big Pharma company has hooked into the $3 billion California
stem cell agency, a move that the agency described as a “watershed”
in its efforts to commercialize stem cell research.

The involvement of GlaxoSmithKline
comes via a partnership with ViaCyte, Inc., of San Diego, Ca., in a
clinical trial, partially financed with a $10.1 million grant today
from the stem cell agency. The trial involves a human embryonic stem
cell product that has “the potential to essentially cure patients
with type 1 diabetes and provide a powerful new treatment for those
with type 2 disease,” ViaCyte said. Scientific reviewers for the agency, formally known as the California Institute for Regenerative Medicine(CIRM),  “characterized the goal of the proposed therapy as as the 'holy grail' of diabetes treatments.”
CIRM Director Jeff Sheehy, who is co
vice chair of the agency's grant review group, said the ViaCyte product
could be manufactured on a large scale and basically involves “taking
(small) pouches and popping them into patients.”
The stem cell agency's award triggered
arrangements between ViaCyte and Glaxo that will bring in financial
and other support from Glaxo. The exact amount of cash was not
disclosed. CIRM said Glaxo will “co-fund and, assuming success,
conduct the pivotal trial and commercialize the product.” Under the terms of the grant, Glaxo and ViaCyte will have to meet CIRM milestones in order to secure continued funding. 
Following board approval, Jason
Gardner
, head of the Glaxo stem cell unit, characterized the
arrangement as a partnership. He told the board that the company
intends to develop a “sustainable pipeline.”
Gardner credited CIRM President Alan
Trounson
with being instrumental in helping to put the arrangement
together, beginning with their first meeting three years ago.
Trounson said the deal will resonate not only in California but
throughout the world.
Paul Laikind, president of ViaCyte,
also addressed the board, stressing the importance of CIRM's
financial support for his company over past years. It has received
$26.3 million (not including the latest grant) from California taxpayers at a time when stem cell
funding was nearly dried up. He noted that small companies such as ViaCyte do not have the resources to carry a product through the
final stages of clinical trials and subsequent production. Gardner also said,

“When the commercial funding avenues
have become much more risk averse, CIRM support (has ensured) that
promising, innovative cell therapy technologies are fully explored.”

In comments to the California Stem Cell
Report,
Elona Baum, CIRM's general counsel and vice president for
business development, described the award as a “watershed” for
the eight-year-old agency, linking the agency with Big Phama for the
first time. Much of CIRM's current efforts are aimed at stimulating
financial commitments from large companies, which are necessary to
commercialize stem cell research.
Arrangements between Big Pharma and
small companies are not unusual and can vanish quickly. However, the
CIRM-ViaCyte-Glaxo deal sends a message to other Big Pharma companies
and smaller ones, perhaps clearing away concerns that have hindered
other deals that could involve the stem cell agency.
The stem cell agency is pushing hard to
fulfill the promises of the 2004 ballot campaign that created CIRM.
Voters were led to believe that stem cell cures were virtually around
the corner. None have been developed to date.

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Trounson Going Halftime in January and February

Posted: October 28, 2012 at 7:59 am


BURLINGAME, Ca. -- The president of the $3 billion California stem cell agency, Alan Trounson, will be working half-time while living in Australia during January and February of next year.

Trounson told the governing board of the agency of his plans at the beginning of its meeting here morning. He said he needs to spend more time with his family, which lives in Melbourne.

Trounson has an 11-year-old son with whom Trounson said he hasn't spend much time in the last 18 months.  Trounson said he intends to teach his son to surf. Trounson's daughter also will be getting married in February.

Meanwhile, directors are currently discussing approval of grants in its $20 million-plus strategic partnership round.

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Texas Science Flap Cited as California Stem Cell Agency Eyes its Own Processes

Posted: October 28, 2012 at 7:59 am


OAKLAND, Ca. – Meeting against a
backdrop from Texas that involves conflicts of interest and mass
resignations of grant reviewers, a task force of the $3 billion
California stem cell agency today began a partial examination of its
own grant approval process, specifically focusing on appeals by
rejected applicants.

The president of the California
organization, Alan Trounson, told the task force that it was dealing
with a “very serious matter” that in some ways is similar to what
happened in Texas. He said the science community is “very much
concerned.”
The situation in Texas involves the
five-year-old Cancer Prevention and Research Institute, which like
the California stem cell agency, formally known as the California
Institute of Regenerative Medicine (CIRM)
, has $3 billion of borrowed
money to use to finance research.
The chief scientific officer of the
Texas organization, Nobel laureate Alfred Gilman, resigned Oct. 12
during a flap about its attempts “to simultaneously support basic
research and nurture companies.”
Gilman's departure was triggered by a
$20 million award made without scientific review. Reviewer
resignations followed with letters that accused the Texas group of
“hucksterism” and dishonoring the peer review process. (Writer Monya Baker has a good overview today in Nature.)
The situation in Texas came to a head
AFTER the governing board of the California research group created
its task force. The problems in Texas are bigger and not identical to
those in California, which mainly involve the free-wheeling nature of the appeal process, not an entire lack of scientific review.
Nonetheless, this past summer, directors of the California agency for
the first time approved an award that was rejected twice by
reviewers. The award went to StemCells, Inc., of Newark, Ca., which
now has won $40 million, ranking the company No. 1 in
awards to business from CIRM.
Earlier this month, Los Angeles Times
business columnist Michael Hiltzik characterized the StemCells, Inc.,
award as “redolent of cronyism.”
Today's session of the CIRM task force
focused primarily on an aspect of the agency's appeals process that
CIRM labels as “extraordinary petitions.” They are letters which
rejected applicants use to challenge decisions by grant reviewers.
The researchers follow up with public appearances before the
governing board, often trailing squads of patients making emotional
appeals.
Both researchers and patients have a
right under state law to appear before the CIRM board to discuss any
matter. CIRM, however, is trying to come up with changes in the
appeal process that will make it clear to researchers on what the
grounds the board might overturn reviewers' decisions. The agency is
also defining those grounds narrowly and aiming at eliminating
appeals based on differences in scientific opinion.
At today's meeting, CIRM Director Jeff
Sheehy
, a patient advocate and co-vice chair of the grants review
group, said peer review is an “extraordinary way of analyzing
science, but it is not always perfect.” However, he also said that
“as a board we are not respecting input” from scientists and thus
allow the perception that we can be “persuaded against the judgment
of scientists.”
CIRM Director Oswald Steward, director
of the Reeve-Irvine Research Center at UC Irvine, agreed with a
suggestion by Sheehy that board must act with “discipline” when
faced with appeals by rejected applicants. Steward said, 

“The
process has gotten a little out of hand.”

It was a sentiment that drew no dissent
at today's 90-minute meeting.
Missing from today's meeting, which had
teleconference locations in San Francisco, Irvine, La Jolla and Palo
Alto, were any of the hundreds of California scientists whose
livelihoods are likely to be affected by changes in the grant
approval process. Also absent were California biotech businesses,
along with the only representative on the task force from CIRM's
scientific reviewers.
Our comment? When researchers and
businesses that have millions at stake fail to show up for key
sessions that set the terms on how they can get the money, it is a
sad commentary on their professional and business acumen.
Bert Lubin, a CIRM director and
chairman of the task force, indicated he would like to have two more
meetings of the task force prior to making recommendations to a full
board workshop in January with possible final action later that
month. Lubin, CEO of Children's Hospital in Oakland, said the matter
is “really important for the credibility of our whole
organization.”

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Ootent growth factor for blood stem cells identified

Posted: October 28, 2012 at 6:45 am

ScienceDaily (Oct. 22, 2012) Duke Medicine researchers studying the interaction of blood stem cells and the niche where they reside have identified a protein that may be a long-sought growth factor for blood stem cells.

The protein is called pleiotrophin, and is produced by cells that line the blood vessels in bone marrow. In mouse studies conducted by the Duke researchers, the protein helps transplanted blood stem cells locate to the bone marrow, where they produce mature red and white blood cells in the body.

The finding, reported in the Oct. 18, 2012, issue of the journal Cell Reports, could lead to new treatments that speed recovery of healthy blood levels for patients receiving chemotherapy or undergoing bone marrow and cord blood transplants.

"Our hypothesis is that pleitrophin has the potential to promote blood stem cell growth in the manner that erythropoietin stimulates red blood cell precursors," said principal investigator John Chute, M.D., professor of Medicine, Pharmacology & Cancer Biology.

Many patients have benefitted from the discovery of erythropoietin (EPO), which stimulates the body to produce mature red blood cells. A synthetic form of EPO is commonly used to treat patients with anemia. Similarly, granulocyte colony stimulating factor (Neupogen), a growth factor for white blood cells, is used to remedy low white blood cell counts that often result from chemotherapy or radiation treatments for cancer.

"A principle objective in hematology for several decades is to identify a growth factor capable of promoting blood stem cells to grow without differentiating," Chute said.

Pleiotrophin may be one such growth factor. Pleiotrophin, which means "many forms," appears to make blood stem cells grow and promote production of all the mature blood lineages that are derived from the blood stem cell. Previously, Chute and his colleagues had shown that treatment with pleiotrophin promoted the expansion of mouse and human blood stems cells in cultures that were capable of engrafting in transplanted mice.

In the new research, lead researcher Heather Himburg, Ph.D., assistant professor of medicine, and Chute's research team showed that cells lining blood vessels in the bone marrow produce pleiotrophin, where it acts as a homing device to attract and retain stem cells. The researchers then demonstrated that genetically engineered mice missing the gene encoding pleiotrophin had decreased numbers of stem cells in their bone marrow, and had difficulty making new blood cells if depleted.

When the researchers treated normal mice with an anti-pleiotrophin antibody, it had the surprising effect of causing existing blood stem cells to be released from bone marrow and enter the blood stream. The finding was particularly exciting to the researchers, as the effect was similar to that observed when granulocyte-colony stimulating factor is used clinically to mobilize stem cells from a donor's bone marrow for use in blood stem cell transplants.

"The discoveries together suggest two possible therapeutic uses," said Chute. "Treatment with pleiotrophin may prove useful in helping patients more quickly regenerate their own blood forming cells after chemotherapy or bone marrow transplant. Second, anti-pleiotrophin antibodies may be useful in mobilizing stem cells to the peripheral blood."

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NIH-CRM Awards Cellular Dynamics up to $7 Million to Produce Human Induced Pluripotent Stem Cells and Differentiated …

Posted: October 28, 2012 at 6:45 am

MADISON, Wis., Oct. 24, 2012 /PRNewswire/ --The National Institutes of Health Center for Regenerative Medicine (NIH-CRM) has awarded Cellular Dynamics International, Inc. (CDI), the world's largest commercial producer of human induced pluripotent stem (iPS) cell lines and tissue cells, a contract to provide human iPS cell lines and terminally differentiated tissue cells from normal or specified patient populations. The contract is worth up to $7.0 million for the three year life of the agreement. This contract follows on two NIH-CRM contracts awarded in September 2011, whereby CDI will generate and genetically engineer iPS cell lines; the company has delivered on several of these cell lines already.

This contract meets a primary goal of the NIH-CRM: to provide researchers with the support needed to move stem cell technology forward. It is expected that these cells will be utilized for preclinical research, including screening compound libraries. As one of the only companies in the world capable of providing iPSC-derived tissue cells in the quality, quantity, and purity required for preclinical research, CDI currently manufactures iCell Cardiomyocytes, iCell Neurons, iCell Endothelial Cells, and iCell Hepatocytes. In addition, the company launched its MyCell Services in June this year, whereby researchers can contract CDI to manufacture iPS cells and tissue cells, as well as do genetic engineering, for patient samples they supply.

"This contract enables NIH researchers to easily access our human iPS cell-derived cells and accelerate the pace of their research," said Chris Parker, chief commercial officer of CDI. "Scientists can now concentrate on their experiments and the data they generate instead of trying to manufacture a consistent and pure supply of human cells. Our expertise is providing the necessary tools human cells so that researchers can focus on discoveries."

Robert Palay, chief executive officer of CDI, continued, "CDI was founded on Dr. James Thomson's vision that the industrial manufacture of human iPS cells and the somatic cells derived from them is critical for discoveries and benefits to human health to be realized. This NIH contract award recognizes CDI's leadership in developing iPSC technology and providing high quality cellular tools to the biological research community in their drive toward improving human health."

About Cellular Dynamics International, Inc.Cellular Dynamics International, Inc. (CDI) is a leading developer of next-generation stem cell technologies for drug development, cell therapy, tissue engineering and organ regeneration. CDI harnesses its unique manufacturing technology to produce differentiated tissue cells from any individual's stem cell line in industrial quality, quantity and purity. CDI is accelerating the adoption of pluripotent stem cell technology, adapting its methods to fit into standard clinical practice by the creation of individual stem cell lines from a standard blood draw. CDI was founded in 2004 by Dr. James Thomson, a pioneer in human pluripotent stem cell research at the University of Wisconsin-Madison. CDI's facilities are located in Madison, Wisconsin. See http://www.cellulardynamics.com.

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Medistem CEO Dr. Alan Lewis to Present at the Stem Cell Meeting on the Mesa

Posted: October 28, 2012 at 6:45 am

SAN DIEGO--(BUSINESS WIRE)--

Medistem announced today Dr. Alan Lewis, the recently appointed CEO of Medistem, will be presenting at the Stem Cells on the Mesa meeting. The company will discuss its ongoing programs at creating effective treatments for cardiovascular diseases using the Endometrial Regenerative Cell (ERC), a stem cell derived from menstrual blood.

The lining of the uterus is unique in its ability to create large numbers of new blood vessels monthly. We found that the cells responsible for creation of new blood vessels, the ER, are shed into the menstrual blood. The ERC have now been cleared by the FDA for clinical trials, said Dr. Lewis. Currently we are pursuing clinical trials in patients with heart failure and critical limb ischemia, an advanced form of peripheral artery disease. In both of these conditions we anticipate the production of new blood vessels will lead to therapeutic responses.

The NIH has recently published that Medistem's ERC possess a unique genetic profile associated with stimulation of new blood vessel growth. The paper may be found at http://www.translational-medicine.com/content/pdf/1479-5876-10-207.pdf

We are excited about the commercialization potential of these cells, given that one donor can generated 20,000 treatment doses, said Thomas Ichim, President and Chief Scientific Officer of Medistem.

Medistems presentation will be at 5:30 PM, Monday, October 29th, 2012, Location: Sanford Consortium, 2880 Torrey Pines Scenic Drive, La Jolla, CA.

The 2012 Stem Cell Meeting on the Mesa is aimed at bringing together senior members of the business development and scientific research communities in regenerative medicine to advance stem cell science into cures for debilitating diseases and injuries. The meeting features a nationally recognized Scientific Symposium attended by more than 600 leading scientists and researchers alongside the regenerative medicine industry's premier annual Investor and Partnering.

Cautionary Statement

This press release does not constitute an offer to sell or a solicitation of an offer to buy any of our securities. This press release may contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking information. Factors which may cause actual results to differ from our forward-looking statements are discussed in our Form 10-K for the year ended December 31, 2007 as filed with the Securities and Exchange Commission.

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Clinical trial attempts to cure autism with cord blood

Posted: October 28, 2012 at 6:45 am

Researchers recently announced the beginning of a FDA-approved clinical trial that uses umbilical cord blood stem cells to cure autism.

Dr. Michael Chez, director of pediatric neurology at Sutter Neuroscience Institute in Sacramento, Calif., said he and his colleagues have been processing the trial for more than a year now, and they have high hopes it will succeed.

- Dr. Michael Chez, director of pediatric neurology at Sutter Neuroscience Institute in Sacramento, Calif.

What we are looking at, is cases that dont have an obvious genetic link, Chez told FoxNews.com. Patients that we presume something went wrong with their brains, which caused a change to autistic features.

Related: Cord blood cures baby's grapefruit-sized tumor

In other words, the trials patients will essentially have no reason to have autism or at least no genetic markers for the disease. This means they must have presumably developed it through another factor, such as the environment or exposure to an infection.

Chez got the idea to treat autism with cord blood stem cells when he observed the cells make a big difference for a little boy who had cerebral palsy.

Related: Cord blood reverses cerebral palsy in Colorado girl

Now, Chez wants to put this boys experience to work for children with autism.

Autism is a developmental disorder that appears in a childs first three years of life, according to the National Institutes of Health. One in 88 U.S. children have it, and it affects one in 54 boys. The condition impacts the brains normal development of social and communication skills sometimes mildly, sometimes extremely.

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Solving stem cell mysteries

Posted: October 28, 2012 at 6:45 am

Public release date: 26-Oct-2012 [ | E-mail | Share ]

Contact: Yixian Zheng zheng@ciwemb.edu 410-246-3032 Carnegie Institution

Baltimore, MD The ability of embryonic stem cells to differentiate into different types of cells with different functions is regulated and maintained by a complex series of chemical interactions, which are not well understood. Learning more about this process could prove useful for stem cell-based therapies down the road. New research from a team led by Carnegie's Yixian Zheng zeroes in on the process by which stem cells maintain their proper undifferentiated state. Their results are published in Cell October 26.

Embryonic stem cells go through a process called self-renewal, wherein they undergo multiple cycles of division while not differentiating into any other type of cells. This process is dependent on three protein networks, which guide both self-renewal and eventual differentiation. But the integration of these three networks has remained a mystery.

Using a combination of genetic, protein-oriented and physiological approaches involving mouse embryonic stem cells, the teamwhich also included current and former Carnegie scientists Junling Jia, Xiaobin Zheng, Junqi Zhang, Anying Zhang, and Hao Jianguncovered a mechanism that integrates all three networks involved in embryonic stem cell self-renewal and provide a critical missing link to understanding this process.

The key is a protein called Utf1. It serves three important roles. First, it balances between activating and deactivating the necessary genes to direct the cell toward differentiation. At the same time, it acts on messenger RNA that is the transcription product of the genes when they're activated by tagging it for degradation, rather than allowing it to continue to serve its cellular function. Lastly, it blocks a genetic feedback loop that normally inhibits cellular proliferation, allowing it to occur in the rapid nature characteristic of embryonic stem cells.

"We are slowly but surely growing to understand the physiology of embryonic stem cells," Zheng said. "It is crucial that we continue to carrying out basic research on how these cells function."

###

Non-Carnegie co-authors on the paper include Gangquing Hu, Kairong Cui, Chengyu Liu and Keji Zhao of the National Institutes of Health; and John Yates III and Bingwen Lu of the Scripps Research Institute, the latter of whom is now at Pfizer.

This research was supported by NIH, NHLBI intramural research, HHMI, and the Cystic Fibrosis Foundation Therapeutics Inc.

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National Press Club Celebration of the Publication of “The Grace of Stem Cells” by Jeong Chan Ra, Chairman of RNL BIO

Posted: October 28, 2012 at 6:45 am

SEOUL, South Korea, Oct. 25, 2012 /PRNewswire/ -- The Grace of Stem Cells byDr. Jeong Chan Ra, biomedical scientist and Chairman of RNL BIO, was published by October 24, 2012.At the National Press Club, leaders from around the world and the media gathered to discuss Dr. Ra's claim that huge amounts of peer reviewed data is systematically ignored in order that opponents of ethical stem cell research are able to promote unsuccessful, unsafe embryonic strategies. Dr. Ra is Chairman of RNL BIO, itself a leader in stem cell translational research.

The Grace of Stem Cells is the first comprehensive analysis of adult stem cell interventions to be published in the English language, and was hailed by leaders for its equally profound story of the relationship between Christian faith and a scientific approach to stem cell research.

Dr. Ra's previous work includes dozens of scientific papers and a book that has sold over 30,000 copies and is ranked one of the best-sellers in science books in Asia.

Emeritus President of the Southern Baptist Convention and one of America's most influential religious leaders Dr. Morris Chapman joined others from America and abroad to celebrate the work as, to quote Dr. Chapman, "the most important book for pro-life advocates on this subject to be published ever."

Patients who had received treatment for incurable diseases using their own (autologous) stem cells were both in attendance and spoke of Dr. Ra's story. Chloe Sohl, who lost her hearing to autoimmune hearing loss, told her story. But the discussion pivoted not on the stories of individuals but the huge amount of published data of recent origin and its effect on the huge demand for stem cells.

Dr. Jeong Chan Ra stated, "As much controversy and issues of safety are concerned in the field of stem cells or embryonic stem cells, autologous adult stem cells are derived from cells that already exist in our bodies to help us treat all kinds of diseases, which is a reminder of God's omnipotence."

Dr. Ra added, "I sincerely hope The Grace of Stem Cells instills faith to people with incurable diseases to be treated." He described the complete commitment of RNL BIO and its partners and friends to bioethics, to protection of life, and to aggressive and systematic research and translational medicine and educational programs. He also discussed the recent and extraordinary growth of RNL BIO and what his plans include for the future.

All royalties from the publications in the United States will be donated to Bethesda Life Foundation in Tennessee, USA. The book is now available in paper as well as e-book at amazon.com.

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Cellular Therapeutics And Regenerative Medicine: Preclinical Assessments For Proof Of Concept And Safety

Posted: October 28, 2012 at 6:44 am

By Thomas Vihtelic, Director, Experimental Therapeutics, MPI Research

Regenerative medicine is attracting significant attention as a medical technology capable of treating what were previously determined to be untreatable diseases. Scientists worldwide are engaged in stem cell research that may enable the repair of damaged heart muscle after a heart attack, restore movement after spinal cord injury, regenerate pancreatic tissue to produce insulin for people with diabetes, and achieve long-sought medical remedies. Animal studies with cell-based therapeutics have demonstrated success, although technological and scientific hurdles remain. Preclinical assessments of cell-based therapies to demonstrate proof of concept and evaluate the risk-benefit ratio for human use present unique challenges requiring expertise and specialized technical capabilities.

This paper presents an overview of stem cell types, their therapeutic risk factors, and preclinical research objectives. The applications of cell-based therapies for selected diseases based on animal models and the qualities to look for when choosing a contract research organization (CRO) for evaluating stem cell-based products are also discussed.

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