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Pluristem stem cell therapy saves a patient, shares jump

By Dr. Matthew Watson

Wed May 9, 2012 3:35pm BST

(Reuters) - Pluristem Therapeutics Inc said a 7-year old girl suffering from a bone marrow disease experienced a reversal of her condition after receiving its experimental stem cell therapy, sending the Israeli company's shares up 32 percent.

The girl, suffering from aplastic bone marrow in which the patient has no blood-forming stem cells, had a significant rise in her red cells, white cells and platelets following an injection of Pluristem's therapy -- PLacental eXpanded cells.

"The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow," Reuven Or, the child's physician at Hadassah Medical Center, was quoted in a statement by Pluristem.

Last September, the company said animal studies showed that the therapy had the potential to treat blood tissue complications related with acute radiation syndrome, commonly called radiation sickness.

Last month, the U.S. health regulators gave a go ahead to the company to start a mid-stage trial of the therapy for treating Intermittent Claudication -- a subset of peripheral artery disease.

Pluristem shares, which have gained 5 percent since receiving the FDA nod for the mid-stage trial, were up 15 percent at $2.70 in morning trade on the Nasdaq. They touched a high of $3.10 earlier.

(Reporting by Esha Dey in Bangalore; Editing by Gopakumar Warrier)

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Iranian researcher helps treating muscular dystrophy using stem cells

By daniellenierenberg

Source: ISNA, Tehran

Iranian researcher and lecturer Radbod Darabi jointly with his collogues from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process which for the first time makes the production of human muscle cells from stem cells efficient and effective.

Radbod Darabi, MD, PhD with Rita Perlingeiro, PhD. (Credit: Image courtesy of University of Minnesota Academic Health Center)

The research outlines the strategy for the development of a rapidly dividing population of muscle-forming cells derived from induced pluripotent (iPS) cells.

IPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

According to the researchers, there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells.

This lag has dramatically limited the development of cell therapies or clinical trials for human patients.

The latest research from the University of Minnesota provides the proof-of-principle for treating muscular dystrophy with human iPS cells, setting the stage for future human clinical trials.

As the researchers noted one of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response.

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Iranian researcher helps treating muscular dystrophy using stem cells

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Single cell triggers fibroid uterine tumor

By Sykes24Tracey

CHICAGO, May 5 (UPI) -- U.S. researchers say they've identified the molecular trigger of fibroid uterine tumors -- a single stem cell develops a mutation and grows uncontrollably.

Dr. Serdar Bulun, the chairman of obstetrics and gynecology at Northwestern University Feinberg School of Medicine and Northwestern Memorial Hospital, said the single cell activates other cells to join its frenzied expansion.

"It loses its way and goes wild," Bulun said in a statement. "No one knew how these came about before. The stem cells make up only 1.5 percent of the cells in the tumor, yet they are the essential drivers of its growth."

Dr. Masanori Ono, a post-doctoral student in Bulun's laboratory who was the study's lead author, said the stem cell that initiated the tumor carries a mutation called MED12.

Recently, mutations in the MED12 gene have been reported in the majority of uterine fibroid tissues. Once the mutation kicks off the abnormal expansion, the tumors grow in response to steroid hormones, particularly progesterone, Bulun said.

"Understanding how this mutation directs the tumor growth gives us a new direction to develop therapies," Bulun said in a statement.

The paper is published in the journal PLoS ONE.

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Single cell triggers fibroid uterine tumor

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Complex cancer industry trial literature is too confusing for patients to understand

By Dr. Matthew Watson

By J. D. Heyes

Have you ever read something so complex and confusing that it frustrated you to
the point of distraction? Well, a new study has found that cancer trial
literature causes that kind of frustration - and may be misleading to patients
as well.

According to Prof. Mary Dixon-Woods, professor of Medical Sociology at the
University of Leicester Department of Health Sciences in Great Britain, a
number of cancer patients found information leaflets describing cancer trials
too long, too incomprehensible and too intimidating.

"These information sheets are poorly aligned with patients' information
needs and how they really make decisions about whether to join a cancer
trial," said Dixon-Woods, lead author of the research http://www.eurekalert.org/pub_releases/2012-03/uol-cti032612.php,
which was published in the international journal Sociology of Health and
Illness.

"Some patients did find them very useful, but many others paid them little
attention. They preferred to rely on discussions they had with their doctor to
make up their minds," she said. Read more…

Source:
http://feeds.feedburner.com/integratedmedicine

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Johnson & Johnson (China) Investment Ltd. Acquires Guangzhou Bioseal … – MarketWatch (press release)

By Dr. Matthew Watson


The Star-Ledger - NJ.com
Johnson & Johnson (China) Investment Ltd. Acquires Guangzhou Bioseal ...
MarketWatch (press release)
GUANGZHOU, China, May 3, 2012 /PRNewswire via COMTEX/ -- Johnson & Johnson (China) Investment Ltd. today announced it has acquired Guangzhou Bioseal Biotechnology Co., Ltd. (Bioseal) a privately held biopharmaceutical company specializing in the design ...
Johnson & Johnson buys Chinese sealant makerBioscience Technology

all 4 news articles »

Source:
http://news.google.com/news?q=biotechnology&output=rss

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UAB Professor Louise Chow elected to National Academy of Science – UAB News

By Dr. Matthew Watson


UAB News
UAB Professor Louise Chow elected to National Academy of Science
UAB News
By Beena Thannickal Louise Chow, Ph.D., professor in the Department of Biochemistry and Molecular Genetics at the University of Alabama at Birmingham and senior scientist at the UAB Comprehensive Cancer Center, was elected a member of the National ...
UAB researcher elected to prestigious National Academy of Sciencesal.com (blog)
Six Stanford faculty elected to National Academy of SciencesStanford University News

all 11 news articles »

Source:
http://news.google.com/news?q=molecular-genetics&output=rss

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Another > $100M month for companies in the cell therapy space

By Dr. Matthew Watson

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Last month we reported here on this blog that March was more than a $100M month for companies in the stem cell and cell-based regenerative medicine space in terms of monies raised.  

What we missed was a $15M grant from Cancer Prevention and Research Institute of Texas (CPRIT) for UK-based CellMedica.  This pumps last month's total to just under $140M.

This month, according to our sources, betters even March's better numbers by coming in at just over $170M though that is largely on the back of one large deal in Asia.  Here's how the numbers break down.

Allocure kicked off the month with a decent $25M Series B round from new syndicate member Lundbeckfond Ventures, as well as previous investors SV Life Sciences and Novo A/S.  Allocure is headed into phase 2 for acute kidney injury with an allogeneic mesenchymal stem cell therapeutic they currently call AC607.  


Little-known Canadian-based, Sernova then announced a $3.6M PIPE to fund continued development of its proprietary Cell Pouch System(TM), and, in particular, to fund the upcoming first-in-man clinical trial for patients with diabetes receiving an islet transplant.  The application to proceed with this trial is currently under review by Health Canada.


Next up was NeoStem closing a $6.8M public offering for "expanding" their contract manufacturing business, Progenitor Cell Therapy, and "enrolling the PreSERVE AMR-001 Phase 2 clinical trial for preserving heart function after a heart attack".  


The biggest deal of the month was a $65M convertible debt financing of China Cord Blood by none other than global powerhouse Kohlberg Kravis Roberts (KKR) through it KKR China Growth Fund L.P., a China-focused investment fund managed by KKR.  We believe this is deal is certainly an investment in the future of China's healthcare market potential but that it is bigger than that.  We believe a significant driver for this deal may likely have been the opportunity to consolidate this sector globally - to use a significant operation and 'war chest' to fund mergers and acquisitions on both the public and private cord blood banking sector worldwide.


The only classic first-round venture raise this month was a milestone-based $5M Series A by Bay City Capital into Phil Coelho's new company, SynGen, to fund his latest iteration of stem cell processing devices.


Forbion Capital then announced that it was leading a series D round, joined by fellow existing investors TVM Capital, Lumira Capital, Intersouth Partners, Caisse de depot et placement du Quebec, Morningside Group, and Aurora Funds, of $25M into Argos Therapeutics in order to kick them into their phase 3.  The hope here is that with some early phase 3 data they may be able to attract the elusive partner they couldn't land with a mere bucket of phase 2 data.


Innovacell landed the only European deal by announcing an 8.3M Euro (~$11M) investment by Buschier, Fides, HYBAG, and Uni Venture.  This will be used for the continued clinical development of its cell-therapy (ICES13) for the treatment of stress-urinary incontinence currently in a ph 3 study in several European countries.


ReNeuron announced a private placement also open to existing shareholders that brought in just under $10M (£6.1M) to support their phase 1 trial in stroke and other pre-clinical, clinical, and regulatory milestones. 


Finally, the Bio-Matrix Scientific Group, in an apparent ongoing quest to continuously reinvent itself, announced at month's end that they had formed a new subsidiary named Regen BioPharma and that they had raised $20M in a financing commitment from Southridge Partners II to purchase its common stock as required over the term of the agreement at a price set by an agreed formula.  This money is said to be dedicated to the acquisition of discovery-stage intellectual property and driving it through to phase 2 trials in an exercise of maximum value creation over a period they claim to be as short as 18-24 months.


..


So in the end, the month saw companies in the space raise just over $170M and even if you back out the stem cell banking deal its still over $100M for cell therapy companies.  


Over the 2 months, then, we've seen just over $311M raised through a variety of means by companies at every stage of maturity and for intended purposes ranging from acquisition, consolidation, early stage clinical development, and phase 3 testing.


--Lee


p.s. If you are aware of other deals in the sector this month, let us know and we'll update this accordingly.


Source:
http://feeds.feedburner.com/CellTherapyBlog

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Gene Hunt Is On for Mental Disabilities in Children

By Dr. Matthew Watson

By Ewen Callaway of Nature magazine

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April 2012 Advances: Additional Resources

By Dr. Matthew Watson

The Advances news section in April's issue of Scientific American included stories on digital textbooks, the promise of using gene therapy to fight blindness and how fragile orchids survive. To learn more about any of the stories, follow these links.

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Processed EHR text can be used to develop CDS tools Read more: Processed EHR text can be used to develop Clinical decision support tools with automated text processing

By Dr. Matthew Watson

See on Scoop.itinPharmatics

Free text in electronic health records, with the help of natural language processing (NLP) technology, can be used to create accurate clinical decision support (CDS) tools, according to a study published this week in the Journal of the American Medical Informatics Association

See on jamia.bmj.com

Source:
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Why mHealth hasn’t created an Instagram (yet)

By Dr. Matthew Watson

See on Scoop.itinPharmatics

Read about why a Qualcomm Life executive says mobile health doesn’t yet have an Instagram, and why it eventually will.

See on http://www.medcitynews.com

Source:
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Kudos to CIRM: Stem Cell Agency Sticks with Full Financial Disclosure

By Dr. Matthew Watson


A key panel of directors of the $3
billion California stem cell agency yesterday voted unanimously to
retain full public disclosure of the financial interests of its
directors and top executives.
The director's Governance Subcommittee
bypassed a proposal that would have substantially weakened disclosure at a time when the agency is moving closer to industry in an effort
to develop cures.
"Because of CIRM's unique mission
and the agency's longstanding commitment to transparency," said
Kevin McCormack, the agency's spokesman, "they believed that
CIRM should continue to set an example by requiring the broadest
disclosure of members of the board and high level staff."
Currently CIRM board members and top
executives must disclose all their investments and income – in a
general way – along with California real property that they hold.
Under the rejected changes, disclosures would have instead been
required only "if the business entity or source of income is of
the type to receive grants or other monies from or through
the California Institute for Regenerative Medicine." 
The proposed changes would also have
relieved CIRM officials of reporting investment in or income from
venture capital or other firms that may be engaged in financing
biotech or stem cell enterprises, since the firms do not receive cash
from CIRM or engage in biomedical research.

The subcommittee's action will go before the full CIRM board later this month, where it is expected to be ratified. 

Our take? The Governance Subcommittee
took the right action and is to be commended for going beyond the
letter of the law. The integrity and credibility of CIRM are
paramount. As the California Stem Cell Report wrote last week, narrowing disclosure would only have engendered suspicion and
unnecessarily raised questions about the conduct of the agency as it
embarks on an aggressive push for stem cell cures.

Source:
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Researcher Alert: First Look at Proposed Rules for California’s Stem Cell Bank

By Dr. Matthew Watson


The California
stem cell agency today unveiled initial details of how it plans to
run its $30 million bank of reprogrammed adult stem cells.

The proposed
regulations are the first step this year in the $3 billion agency's
project to make IPS cells available worldwide at low cost. It is part
of an effort to stimulate the science and develop commercial cures by
removing research roadblocks.
As Amy Adams,
CIRM's communications manager wrote earlier this year on the agency's research blog,

"One way for CIRM to accelerate research is by creating more of a library system
for stem cells – except we don’t want the cells back."

The agency expects
to issue its first RFA next month in the stem cell banking initiative, which consists of three grant rounds to be approved by
the CIRM board no later than Feburary of next year.

To clear the way
for the first round, CIRM plans to revise its IP regulations to
ensure that they don't hamper the distribution of stem cells in its bank and their wide use.  The revisions will come before the CIRM directors'
IP/Industry Subcommittee next Tuesday. The six-member panel is
co-chaired by co-chaired by Stephen Juelsgaard, former executive vice
president of Genentech, and Duane Roth, CEO of Connect in San Diego,
a nonprofit that supports tech and life sciences entrepreneuers.
Sites where the public can participate in the meeting will be
available in San Francisco, La Jolla, Los Angeles and two in Irvine.
Under the new IP rules, CIRM will retain ownership of the cells in its bank instead
of the grantee, as the current IP rules state.
In a memo to
directors, Elona Baum, general counsel for the agency, said,

"This permits
CIRM to have complete control of this valuable resource and is
consistent with the practice of NIH’s Center for Regenerative
Medicine
which is also creating a repository for iPSC lines and
derived materials."

Baum also said,

"The (current) IP
regulations were drafted to address conventional drug discovery
activities and did not contemplate creation of a comprehensive
repository of cell lines intended for broad distribution. As a
result, the IP regulations contain a number of provisions which are
either not applicable or worse could impede the success of the hiPSC
bank. For instance, IP regulations permit the exclusive licensing of
CIRM funded inventions and technology. This would be
counterproductive to the goals of the hiPSC repository which are
predicated on wide spread access."

Baum provided the
following summary of the $30 million banking initiative:

"These lines
will serve as valuable tools in drug discovery and will be available
to researchers worldwide. The Tissue Collection RFA No. 12-02 will
fund clinicians and other scientists to identify, recruit and consent
sufficient numbers of affected individuals within a disease
population so as to effectively represent the disease’s
manifestations. Tissues will be collected and appropriate clinical,
medical or diagnostic information, will be obtained to enable
informed discovery of disease-related phenotypes and drug development
activities using hiPSC-based models. These tissue samples will be
provided (without charge) to the recipient of the CIRM hiPSC
Derivation Award (RFA No. 12-03) for the production of the hiPSC
lines. Once derived, characterized and released, the lines will be
deposited in the CIRM hiPSC bank funded under RFA No. 12-04."

Specific addresses
for the public meeting locations can be found on the agenda.

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

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ACT and CIRM Together on Eye Research Webinar

By Dr. Matthew Watson


Executives of Advanced Cell Technology,
which has been repeatedly rejected for funding by the $3 billion
California stem cell agency, will participate this week in the agency's Internet "webinar" on research involving the human eye.
ACT, which is engaged in the only hESC
clinical trial in the nation, was held up earlier this month (see
here and here) at an Institute of Medicine hearing as a prime example
of the California's agency's failure to fund the stem cell industry
in any significant way.
Gary Rabin, CEO of ACT
Nonetheless, three ACT execs are scheduled to be online for the CIRM session on Wednesday: Gary Rabin, the CEO of
ACT; Matthew Vincent, director of business development, and Edmund
Mickunas
, vice president of regulatory affairs. The webinar will deal with regulatory issues
with the FDA and clinical trials involving the eye.
Also on the panel are Samuel Barone,
medical officer with the FDA, and Mark Humayun, professor of
opthamology at USC, who is the PI on a $16 million macular
degeneration grant from CIRM.
So what is the significance of ACT's
participation in the CIRM event? If the relationship between ACT and
CIRM has been touchy, this sort of cooperation is probably a good
sign for both. For one, CIRM could have hardly staged the webinar
without ACT, given the subject matter. But if the agency did not
want to engage ACT, it could have simply done nothing about setting
up a webinar in which the firm would participate.
Does this mean that ACT is going to
receive a handsome grant or loan from CIRM? CIRM has established
procedures (RFAs, peer review, etc.) for approval of funding, and ACT
would have to go through that process unless CIRM does something very
unusual.
Wednesday's event is aimed at researchers and
is likely to be technical. Persons interested in taking part must
register in advance.

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

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Oxford Scientists Seek to Test Genetic Solution to Dengue-Carrying Mosquitoes … – OregonLive.com

By Dr. Matthew Watson


Bloomberg
Oxford Scientists Seek to Test Genetic Solution to Dengue-Carrying Mosquitoes ...
OregonLive.com
LONDON — British biotechnology start-up Oxitec wants to start US tests of a new weapon in the war on dengue fever: genetically modifying mosquitoes that carry the disease so that their progeny self-destruct. Dengue, endemic in more than 100 countries, ...
Scientists modifying mosquitoes to prevent dengueVaccine News Daily (blog)

all 6 news articles »

Source:
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Kashi is latest food to go anti-biotechnology – Oregon Natural Resources Report

By Dr. Matthew Watson


Oregon Natural Resources Report
Kashi is latest food to go anti-biotechnology
Oregon Natural Resources Report
Tellingly, the press release issued by Kashi comes quickly on the heels of an agenda-driven campaign to “out” the health food maker's use of foods produced with biotechnology. The declaration of the cowardly cereal creator's about-face on biotechnology ...

Source:
http://news.google.com/news?q=biotechnology&output=rss

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REGION: Surprise cord-blood find is 'godsend' for ailing boy

By Sykes24Tracey

After half a year of blood transfusions to treat life-threatening anemia, 9-year-old Ricky Martinez was running out of time.

The Murrieta boy needed a bone marrow transplant to save his life. Although his parents had held numerous drives seeking a match for their son, the perfect donor eluded them.

Then another option appeared ---- doctors found Ricky's own blood from his umbilical cord, banked at birth, and stored in a medical facility.

"I had donated it at birth, when I delivered," said Ricky's mother, Cynthia Martinez. "I had no idea that I'd be using it for him nine years later."

The cord blood discovery represents a "godsend" for the family, Martinez said, because Ricky's body began rejecting the transfusions that keep him alive.

Cord blood contains stem cells ---- undifferentiated cells that can spur production of healthy tissue to help treat various diseases. Doctors believe it could jump-start Ricky's bone marrow, allowing his body to resume normal blood production.

But it's not a guarantee.

Ricky's condition, aplastic anemia, is an extremely rare disease, and cord blood transplantation is an experimental procedure for the condition, said David Buchbinder, a hematologist and transplant physician who is treating Ricky at Children's Hospital Orange County, in the city of Orange.

Although the procedure offers few risks of complications, it also pushes the boundaries of medical practice, placing Ricky in a realm of mixed medical opinions and uncertain results, Buchbinder said.

His parents say they're willing to go there to save their son's life.

Visit link:
REGION: Surprise cord-blood find is 'godsend' for ailing boy

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Researchers Rejuvenate Blood-Forming Stem Cells in Mice

By NEVAGiles23

Latest Senior Health News

THURSDAY, May 3 (HealthDay News) -- Researchers who found a way to rejuvenate aged blood-forming cells in mice say their achievement offers clues about how it may be possible to combat health problems associated with old age.

The study by scientists at Cincinnati Children's Hospital Medical Center and Ulm University Medicine in Germany appeared online May 3 in the journal Cell Stem Cell.

Hematopoietic (meaning "to make blood") stem cells, which originate in the bone marrow, produce all of the body's red and white blood cells and platelets. As people age, these cells increase in number but become but less effective at generating new blood cells and immune cells. This makes older people more susceptible to infections and diseases, including leukemia.

In laboratory experiments with mouse cells, the researchers found that a specific protein that regulates cell aging also controls a process that causes blood-making stem cells to age. Using drugs to inhibit the action of this protein (called Cdc42) reversed aging of the hematopoietic stem cells and restored their function to a level similar to that of younger stem cells.

It had been believed that the aging of hematopoietic stem cells was locked in by nature and could not be reversed by using drugs, according to a hospital news release.

"Our findings suggest a novel and important role for Cdc42, and identify its activity as a target for ameliorating natural [hematopoietic stem cell] aging," principal investigator Hartmut Geiger, of the University of Ulm, said in the release. "We know the aging of [these stem cells] reduces in part the response of the immune system response in older people, which contributes to diseases such as anemia and may be the cause of tissue attrition in certain systems of the body."

Researchers say the next step is to test a protein inhibitor in mice to see how hematopoietic stem cells and various tissues respond. The researchers also are gathering samples of human blood-making stem cells for future lab tests.

Although studies involving animals can be useful, they frequently fail to produce similar results in humans.

-- Robert Preidt

Go here to see the original:
Researchers Rejuvenate Blood-Forming Stem Cells in Mice

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4-Hour, Whole-Body 'Face-Lift' Uses Patient's Own Fat, Stem Cells

By JoanneRUSSELL25

May 4, 2012 9:27am

ABC News Paula Faris reports:

It is a medical claim that sounds like science fiction. Walk into a plastic surgeons office for a face-lift and walk out roughly four hours later with a whole-body makeover that required no incision and leaves you with no scars.

But some doctors say that fiction is now reality in the form of a stem-cell makeover, a procedure that uses the fat and stem cells from one part of the body to revamp another part of the body, all in a single office visit.

Such a claim convinced Debra Kerr to try the procedure herself in hopes of achieving a younger look. My eyes are looking heavier, and the lines are so pronounced and gravitys really taken over, Kerr, 55, said. I want to look as good and as young as I really feel.

Kerr, a skin-care specialist from Ohio, underwent a stem-cell makeover in which fat was removed from her waist via liposuction. The fat was then spun in the lab to concentrate its stem cells and, hours later, injected into Kerrs face and breasts.

Were taking a patients own fatty tissue, and we are just repositioning it in another part of their body, said Dr. Sharon McQuillan, a physician and founder of the Ageless Institute in Aventura, Fla., where Kerr had her procedure done.

Courtesy Dr. Sharon McQuillan

Because the makeover uses a patients own stem cells, there is virtually no risk that the body will reject the transfer, according to doctors like McQuillan who perform the procedure.

This enhancement will be enough to make her [Kerr] happy, McQuillan said. She wont have any scars. She doesnt really have any of the risks associated with general anesthesia or a full face lift.

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4-Hour, Whole-Body 'Face-Lift' Uses Patient's Own Fat, Stem Cells

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U of M researchers develop new muscular dystrophy treatment approach using human stem cells

By JoanneRUSSELL25

Public release date: 3-May-2012 [ | E-mail | Share ]

Contact: Caroline Marin crmarin@umn.edu 612-624-5680 University of Minnesota Academic Health Center

MINNEAPOLIS/SAINT PAUL (May 4, 2012) Researchers from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that for the first time makes the production of human muscle cells from stem cells efficient and effective.

The research, published today in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

According to U of M researchers who were also the first to use ES cells from mice to treat muscular dystrophy there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells. This lag has dramatically limited the development of cell therapies or clinical trials for human patients.

The latest research from the U of M provides the proof-of-principle for treating muscular dystrophy with human iPS cells, setting the stage for future human clinical trials.

"One of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response," said principal investigator Rita Perlingeiro, Ph.D., associate professor of medicine in the Medical School's Division of Cardiology. "Up until now, deriving engraftable skeletal muscle stem cells from human pluripotent stem cells hasn't been possible. Our results demonstrate that it is indeed possible and sets the stage for the development of a clinically meaningful treatment approach."

Upon transplantation into mice suffering from muscular dystrophy, human skeletal myogenic progenitor cells provided both extensive and long-term muscle regeneration which resulted in improved muscle function.

To achieve their results, U of M researchers genetically modified two well-characterized human iPS cell lines and an existing human ES cell line with the PAX7 gene. This allowed them to regulate levels of the Pax7 protein, which is essential for the regeneration of skeletal muscle tissue after damage. The researchers found this regulation could prompt nave ES and iPS cells to differentiate into muscle-forming cells.

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