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Category Archives: Stem Cells

Pay-for-Eggs Legislation: Strange Bedfellows and Existential Questions

Posted: July 10, 2013 at 8:26 pm

 The California pay-for-eggs bill
today generated a feature article that said the legislation has
“sparked an unusual lineup of partisans on both sides and resonates
far beyond” the Golden State.
The piece by Alex Mathews on Capitol
Weekly
, a news service specializing in California government and
political coverage, said,

“(C)omplicating the issue is
California’s role as a national leader in stem cell research, the
existential question of who or what constitutes a research subject,
and finally, the fact that compensation for fertility purposes is and
has been legal for years in California.”

Mathews was writing about the measure
(AB926) by Assemblywoman Susan Bonilla, D-Concord, that removes a ban
in California on paying women for eggs for scientific research.
Currently women can be paid in California for providing eggs for IVF.
The measure would not alter a ban on compensation for eggs in
research financed by the $3 billion California stem cell agency.
However, later this month, the agency will consider modifying its position somewhat.
The bill has passed the legislature and
is on its way to Gov. Jerry Brown. The industry association
sponsoring the bill expects the governor to sign it later this month
although the governor, as a general rule, does not make public
commitments on legislation.
Mathews' article covered the background
and arguments on the bill and noted that it has received little
mainstream media attention.
Lisa Ikemoto
UC Davis photo
She also quoted Lisa Ikemoto, a law
professor and bioethicist at UC Davis, on the sensitive nature of the
issue. Ikemoto said,

“On the fertility side, it’s
politically hard to touch because it’s all around family formation.
Nobody wants to restrict family formation. On the research side, when
the issue of payment for eggs came up, it was connected with human
embryonic stem cell research, and human embryonic stem cell research
was politicized from the outset.”

Mathews also wrote about the strange
bedfellows opposing the bill. She said,

“Groups that fundamentally oppose
stem cell research such as the California Catholic Conference and
other pro-life groups are natural opponents of the bill, but they are
joined by a number of pro-choice groups who expressed concerns over
the limited research on the effects of egg donation on women’s
health.”

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California Stem Cell Merger: Capricor and Niles Therapeutics

Posted: July 10, 2013 at 11:52 am

Capricor, Inc., a Beverly Hills company
benefiting from $27 million from the California stem cell agency,
this week announced that it is merging with Niles Therapeutic, Inc.,
of San Mateo.
Linda Marban
Capricor photo
The Capricor story and its treatment
for heart disease have been highlighted (see here and here) by the $3
billion state research agency, which is partially funding a clinical
trial for the firm. The firm sprang from work by Eduardo Marban of
Cedars-Sinai in Los Angeles, one of Capricor's founders. He received
$6.9 million for his early and current work. Capricor was awarded
$19.8 million more.
Capricor, a privately held firm, and
the publicly traded Niles announced on Monday that they were merging.
The new company will be known as Capricor Therapeutics, Inc., and will
be based in San Mateo.
The new firm will be publicly traded
with Capricor CEO Linda Marban as the new CEO.
The new board of directors will have
two members from Niles and seven from Capricor, including its
executive chairman, Frank Litvack, who was an unsuccessful candidate for chairman of the stem cell agency board in 2011.
The merger press release said that the
new company “should
have better access to capital, more potential for steady pipeline
development and more risk diversification."
On completion of the merger, a joint
press release said,

Nile
will issue to Capricor stockholders shares of Nile common stock such
that Capricor stockholders will own approximately 90% of the combined
company's outstanding shares, and Nile stockholders will own
approximately 10%, calculated in each case on a fully-diluted basis
assuming the issuance of shares underlying options and warrants.
Options of Capricor will be assumed by Nile and become options to
acquire stock of Nile.”

Linda Marban said,

"Capricor's
and Nile's product portfolios complement each other well, as our
therapies will address both the underlying causes and debilitating
effects of heart disease. Capricor's CDCs are allogeneic cardiac
derived stem cells that aim to attenuate and potentially improve
damage to the heart that can result in heart failure, while Nile's
cenderitide is intended to treat patients following hospital
discharge from an acute episode of heart failure."

Niles' stock price stood at $0.04
recently. Its 52 week high was $0.20 and the 52-week low was $0.02.

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Newly identified bone marrow stem cells reveal markers for ALS

Posted: July 10, 2013 at 3:43 am

Public release date: 9-Jul-2013 [ | E-mail | Share ]

Contact: George Hunka ghunka@aftau.org 212-742-9070 American Friends of Tel Aviv University

Amyotrophic Lateral Sclerosis (ALS) is a devastating motor neuron disease that rapidly atrophies the muscles, leading to complete paralysis. Despite its high profile established when it afflicted the New York Yankees' Lou Gehrig ALS remains a disease that scientists are unable to predict, prevent, or cure.

Although several genetic ALS mutations have been identified, they only apply to a small number of cases. The ongoing challenge is to identify the mechanisms behind the non-genetic form of the disease and draw useful comparisons with the genetic forms.

Now, using samples of stem cells derived from the bone marrow of non-genetic ALS patients, Prof. Miguel Weil of Tel Aviv University's Laboratory for Neurodegenerative Diseases and Personalized Medicine in the Department of Cell Research and Immunology and his team of researchers have uncovered four different biomarkers that characterize the non-genetic form of the disease. Each sample shows similar biological abnormalities to four specific genes, and further research could reveal additional commonalities. "Because these genes and their functions are already known, they give us a specific direction for research into non-genetic ALS diagnostics and therapeutics," Prof. Weil says. His initial findings were reported in the journal Disease Markers.

Giving in to stress

To hunt for these biomarkers, Prof. Weil and his colleagues turned to samples of bone marrow collected from ALS patients. Though more difficult to collect than blood, bone marrow's stem cells are easy to isolate and grow in a consistent manner. In the lab, he used these cells as cellular models for the disease. He ultimately discovered that cells from different ALS patients shared the same abnormal characteristics of four different genes that may act as biomarkers of the disease. And because the characteristics appear in tissues that are related to ALS including in muscle, brain, and spinal cord tissues in mouse models of genetic ALS they may well be connected to the degenerative process of the disease in humans, he believes.

Searching for the biological significance of these abnormalities, Prof. Weil put the cells under stress, applying toxins to induce the cells' defense mechanisms. Healthy cells will try to fight off threats and often prove quite resilient, but ALS cells were found to be overwhelmingly sensitive to stress, with the vast majority choosing to die rather than fight. Because this is such an ingrained response, it can be used as a feature for drug screening for the disease, he adds.

The hunt for therapeutics

Whether these biomarkers are a cause or consequence of ALS is still unknown. However, this finding remains an important step towards uncovering the mechanisms of the disease. Because these genes have already been identified, it gives scientists a clear direction for future research. In addition, these biomarkers could lead to earlier and more accurate diagnostics.

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Microparticles Create Localized Control of Stem Cells

Posted: July 10, 2013 at 3:43 am

Newswise Before scientists and engineers can realize the dream of using stem cells to create replacements for worn out organs and battle damaged body parts, theyll have to develop ways to grow complex three-dimensional structures in large volumes and at costs that wont bankrupt health care systems.

Researchers are now reporting advances in these areas by using gelatin-based microparticles to deliver growth factors to specific areas of embryoid bodies, aggregates of differentiating stem cells. The localized delivery technique provides spatial control of cell differentiation within the cultures, potentially enabling the creation of complex three-dimensional tissues. The local control also dramatically reduces the amount of growth factor required, an important cost consideration for manufacturing stem cells for therapeutic applications.

The microparticle technique, which was demonstrated in pluripotent mouse embryonic cells, also offers better control over the kinetics of cell differentiation by delivering molecules that can either promote or inhibit the process. Based on research sponsored by the National Institutes of Health and the National Science Foundation, the developments were reported online July 1 in the journal Biomaterials and were presented at the 11th Annual International Society for Stem Cell Research meeting held in Boston June 12-15, 2013 .

By trapping these growth factors within microparticle materials first, we are concentrating the signal they provide to the stem cells, said Todd McDevitt, an associate professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. We can then put the microparticle materials physically inside the multicellular aggregate system that we use for differentiation of the stem cells. We have good evidence that this technique can work, and that we can use it to provide advantages in several different areas.

The differentiation of stem cells is largely controlled by external cues, including morphogenic growth factors, in the three-dimensional environment that surrounds the cells. Most stem cell researchers currently deliver the growth factors into liquid solutions surrounding the stem cell cultures with a goal of creating homogenous cultures of cells. Delivering the growth factors from microparticles, however, provides better control of the spatial and temporal presentation of the molecules that govern the growth and differentiation of the stem cells, potentially allowing formation of heterogeneous structures formed from different cells.

Groups of stem cells stick together as they develop, forming multicellular aggregates that form spheroids as they grow. The researchers took advantage of that by driving microparticles containing growth factor BMP4 or noggin which inhibits BMP4 signaling into layers of stem cells using centrifugation. When the cell aggregates formed, the microparticles became trapped inside.

The researchers used confocal imaging and flow cytometry to observe the differentiation process and found that growth factors in the microparticles directed the cells toward mesoderm and ectoderm tissues just as they do in solution-based techniques. But because the BMP4 and noggin molecules were directly in contact with the cells, much less growth factor was needed to spur the differentiation approximately 12 times less than what would be required by conventional solution-based techniques.

One of the major advantages, in a practical sense, is that we are using much less growth factor, said McDevitt, who is also director of the Stem Cell Engineering Center at Georgia Tech. From a bioprocessing standpoint, a lot of the cost involved in making stem cell products is related to the cost of the molecules that must be added to make the stem cells differentiate.

Beyond more focused signaling, the microparticles also provided a localized control not available through any other technique. That allowed the researchers to create spatial differences in the aggregates a possible first step toward forming more complex structures with different tissue types such as vasculature and stromal cells.

To build tissues, we need to be able to take stem cells and use them to make many different cell types which are grouped together in particular spatial patterns, explained Andres M. Bratt-Leal, the papers first author and a former graduate student in McDevitts lab. This spatial patterning is what gives tissues the ability to perform higher order functions.

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Sports Medicine and Stem Cells: Athletes are ready for New Era of Treatment

Posted: July 10, 2013 at 3:43 am

TAMPA, Fla., July 9, 2013 /PRNewswire/--As new medical frontiers are forged, their place in sports medicine will be assured if athletes can recover or heal faster. This concept has not been lost in the dawn of Regenerative Medicine. This new age has emerged in which regenerative stem cell treatments are being applied to blindness, spinal cord injuries and congestive heart failure. It is no wonder that elite athletes such as Peyton Manning turned to stem cells when his neck wasn't healing. http://www.drlox.com

So what is it that makes the allure of stem cells attractive to high caliber athletes? According to Dr. Dennis Lox, a Sports Medicine and Regenerative specialist, in the Tampa Bay, Florida area, stem cells have unique capabilities for injured athletes. First the stem cells are very effective at alleviating inflammatory responses seen in chronic injuries. By blocking and altering the mechanisms in which inflammation occurs, some chronic injuries may heal. Dr. Lox then comments that, the stem cells are also at the same time regenerative cells that may heal by releasing factors that allow healing to occur, or trophic effects. Lastly, the stem cells may allow regeneration of injured tissue. All these mechanisms, Dr. Lox stresses, cannot be achieved by cortisone injections, or commonly prescribed anti-inflammatory medications. http://www.drlox.com

The unique way in which regenerative therapies such as Platelet Rich Plasma (PRP) and stem cells, exert effects on injured tissues will always be seen positively as athletes search for better and quicker healing treatments. Also, athletes need to be concerned with career longevity. The possible fountain of youth that repairing injured muscles and joints may hold with stem cell therapy makes it an attractive option. No adverse effects have been reported in clinical trials using stem cells for knee arthritis. The same cannot be said for knee surgery. The upside to minimizing further joint injury by avoiding surgery, may deter accelerated arthritis development. Dr. Lox notes a player's career may be lengthened by early repair of knee injury, and preventing arthritis development. These are two goals of regenerative medicine, and in some athletes mind two reasons to consider stem cell therapy.

About Dr. Dennis Lox Dr. Lox practices in the Tampa Bay Florida area. Dr. Lox is a Sports and Regenerative Medicine Physician, who specializes in the use of regenerative and restorative medicine to assist in treating athletic and arthritis conditions. Dr. Lox may be reached at (727) 462-5582 or visit Drlox.com.

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HIV Clinical Trial Hailed by California Stem Cell Agency

Posted: July 9, 2013 at 4:03 pm

The
California stem cell agency today scored what it called an “important
milestone” with the announcement of the start of a clinical trial
involving a therapy to help protect persons infected with HIV from
the effects of the virus.
The
trial is partially funded from a $20 million award from the stem cell
agency, which is known as CIRM, to researchers
at UCLA and Calimmune, a Tucson, Az.,
company. Calimmune's share of the award was $8.2 million.
Alan
Trounson
, president of the $3 billion state agency, said in a
statement,

CIRM
funding of this Phase l/ll trial is an important milestone for us.
One of our goals is to support research that moves the most promising
science out of the lab and into clinical trials in people. To be able
to do that with a disease as devastating as HIV/AIDS highlights the
importance of our funding and the potential impact it could have on
the health of people around the world.”

The
trial was announced by Calimmune this morning. The company said,

The
first patient has begun treatment in a Phase I/II clinical trial
designed to determine whether a pioneering genetic medicine approach
can help to protect individuals infected with HIV from the effects of
the virus. The study, “
Safety
Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1
Infection
,”
utilizes a gene medicine called Cal-1, developed in the lab of Nobel
Laureate Dr. 
David
Baltimore
 and
by Calimmune.”

Baltimore
served on the CIRM board from 2004 until June 6, 2007. He 
resigned
from the board 
about
18 months before the application process began for the grant round
that ultimately funded Calimmune, a company he helped to found. He is currently chairman of the Calimmune board. 
Asked
for comment, Jeff Sheehy, a member of the CIRM governing
board and communications director for AIDS research at UC San
Francisco
, said,

"This
trial will hopefully offer several important insights into the safety
and feasibility of genetically modifying blood forming stem cells in
an HIV patient as a potential therapy.  We are very early in
this research, and with this Phase I trial's goal of establishing
safety and the risks involved, I applaud the courage and altruism
demonstrated by the patients who are willing to participate in this
study."

The Calimmune
press release
 said
the principal investigators on the clinical trial are 
Ron
Mitsayasu
 of
UCLA and 
Jacob
P. Lalezari
 of Quest
Clinical Research
 of
San Francisco. Quest is currently soliciting patients for the
clinical trial as well as UCLA. (Persons interested in participating
in the trial can find email contacts 
at
this website
.
Twelve are needed.)
The
principal investigators on the CIRM award are Irvin Chen of
UCLA and Geoff Symonds of Calimmune.  

Here are links to the CIRM press release on the subject and the agency's blog item.

(An earlier version of this story did not include the fact that Calimmune's share of the CIRM award was $8.2 million or the links to the agency press release and blog.)

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Girl who received windpipe made from stem cells dies

Posted: July 8, 2013 at 10:48 pm

July 8, 2013 (PEORIA, Ill.) (WLS) -- Hannah Warren, the toddler who underwent a successful trachea transplant surgery in April, died over the weekend. She would have turned 3 in August.

Hannah, who died on July 6, 2013, was "unable to overcome additional health issues that were identified as her care progressed," according to Children's Hospital of Illinois in Peoria. On April 9, 2013, a windpipe made from her own stem cells was implanted into Hannah's trachea. Until the surgery, she was unable to breathe, eat or swallow on her own, and had spent her entire life in a hospital in South Korea.

The Children's Hospital of Illinois released a statement July 8 that read in part, "Although regenerative medicine remains in the early stages for pediatric patients, progress is being made. Hannah, and the physicians caring for her, helped advance this area of medical practice which is only at its very beginning stages. Even at this time of loss and grief, we, and Hannah's family, take comfort in the knowledge that the efforts of her physicians and the care team working with them will benefit and serve other children and adults in the years to come."

(Copyright 2013 WLS-TV/DT. All Rights Reserved.)

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Scéil(TM) : A New Offering for the General Public to Turn Adult Cells into Stem Cells and Store Them

Posted: July 8, 2013 at 10:47 pm

Regulatory News :

The Cellectis Group (Paris:ALCLS) (Alternext: ALCLS) announced today that it is launching Scil, an offering for the general public that involves storing induced pluripotent stem cells (iPS) generated from a skin sample so that people can benefit, if needed, from future regenerative medicine treatments as soon as they become available.

Cellectis is a biotechnology industry group with 13 years experience in genome engineering and stem cells. It has a strong background in handling induced pluripotent stem cells on a large scale up through their maturation and differentiation into functional cell types.

The Group has developed wide-reaching projects based on iPS cells, including the iPS Engineering Hub, a service that helps match new drugs to patient needs. Regenerative medicine is another area of development for the Group. One ongoing project, in partnership with worldwide diabetes market leader Novo Nordisk, is focused on developing a treatment for type 1 diabetes using engineered stem cells. Cellectis has also been working since 2010 with the CiRA laboratory, run by Pr Shinya Yamanaka, winner of the 2012 Nobel prize in medicine for his work on induced pluripotent stem cells.

Scil is part of the Groups strategic focus on therapeutics. While not itself a therapeutics solution, Scil naturally complements the Groups offering in this area.

Scil will initially be marketed by a new wholly owned subsidiary of Cellectis SA, Scil Private Limited, in Singapore. Another Scil subsidiary is being set up in Dubai. These locations accord with existing national laws and regulatory frameworks.

In a meeting with US press at which Scil was unveiled, Andr Choulika announced, "We are proud to be the first in the world to make the major scientific breakthrough of iPS cell technology available to the public. Scil represents a real economic opportunity, one of the many steps forward to come in regenerative medicine.

About Cellectis Founded in France in 1999, the Cellectis Group bases its work on highly specific DNA engineering technologies. Its application sectors are human health, agriculture and bio-energies. Cellectis was co-founded by Andr Choulika, its Chairman and CEO, and is now one of the worlds top companies in the field of genome engineering, with revenue of $27million in 2012. Leading the field of pluripotent stem cells, Cellectis has developed expertise in drug discovery, toxicity testing, and regenerative medicine. Cellectis has a solid background in the large-scale handling of stem cells up until their maturation and differentiation into functional cell types. We employ a workforce of 230 people at 5 sites worldwide: New Brighton (Minnesota) & Cambridge (Massachusetts) in the United States, Gothenburg in Sweden, and Paris & Evry in France. The Group has signed more than 100 industry agreements with pharmaceutical, agrochemical, and biotechnology companies. Our clients and partners include University College London (UCL), the National Institutes of Health (NIH), Novo Nordisk, the Center for iPS Cell Research and Application (CiRA) of Kyoto University, AFM, Novartis, BASF, Bayer, and Limagrain. Since 2007, Cellectis has been listed on the NYSE Euronext Alternext market (code: ALCLS) in Paris. For more information, visit our website: http://www.cellectis.com.

Disclaimer This press release and the information contained herein do not constitute an offer to sell or subscribe, or a solicitation of an offer to buy or subscribe, for shares in Cellectis in any country.

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Eggs and Cash: Stem Cell Agency Considering Easing Restrictions on Stem Cell Lines Derived Using Payments

Posted: July 7, 2013 at 10:12 pm

The California stem cell agency is
moving to remove an absolute ban on use of stem cell lines derived
from eggs from women who have been paid to provide them.
The action comes as state legislation
is headed for Gov. Jerry Brown's desk that would permit payments for
eggs to be used in research that is not funded by the agency. The measure (AB926) would not alter the separate ban on egg payments
involving research funded by the $3 billion stem cell agency.
Under a proposal that will come before the agency's standards group July 24, CIRM's governing board could
approve the use of stem cell lines derived as a result of payment to
women. Board action would be based on whether stem cell lines would
“advance CIRM's mission” and would follow a staff evaluation
involving scientific and ethical issues.
Over recent years, stem cell
researchers around the country have reported that they are not able
to obtain sufficient eggs without payment. And earlier this year,
paid egg providers were used in research in Oregon that cloned human stem cells, a feat that researchers have struggled with for years.
A CIRM staff report said that the
Oregon research has “generated scientific interest among CIRM
grantees and the desire to utilize derived SCNT lines. CIRM’s
current policy prohibits the use of the (Oregon) SCNT lines because
oocyte donors were financially compensated. CIRM requests the Medical
and Ethical Standards Working Group (SWG) revaluate this prohibition
with regard to CIRM grantees ability to utilize the resulting lines
in light of recent scientific and policy developments.”
Last month, the California Stem Cell
Report
queried the agency concerning earlier, sketchy information onthe CIRM blog about a possible change in its compensation rules. We
asked whether the agency was considering “sidestepping” the ban
on compensation. Kevin McCormack, a CIRM spokesman, said, “No, not
at all.” He said it would be premature to elaborate until a firm
proposal was ready.
The staff proposal to be considered on
July 24 said,

“Proposition 71’s 'prohibition on
compensation' compels the ICOC(the agency's governing board) to adopt
standards 'prohibiting compensation to research donors.' This
requirement has been consistently interpreted to prohibit the use of
CIRM funds to financially compensate oocyte (or other cell or tissue)
donors. In 2006, this interpretation was extended to exclude from
use, in CIRM-funded research, any stem cell line where research
donors were financially compensated, even if the derivation was done
without the use of CIRM funds. Proposition 71, however, does not
compel the ICOC (the agency's governing board) to prohibit the use of
stem cell lines where financial compensation is provided to the
oocyte donors, provided that CIRM funds are not used to compensate
the donors or derive the lines.”

The July 24 meeting will be held in San
Francisco. No remote teleconference locations have been announced.  If approved, the changes would likely be considered July 25 by the full agency board.

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California Legislation Removing Ban on Payments for Eggs for Research Heads to Governor

Posted: July 7, 2013 at 8:40 pm

Legislation to allow women in
California to be paid for their eggs for scientific research is on
its way to Gov. Jerry Brown following final legislative approval last
week.
Sponsors of the bill, a national
fertility industry organization, expect the governor later this month
to sign the measure, which would go into effect next year.
The measure, AB 926 by Assemblywoman
Susan Bonilla, D-Concord, would repeal a ban on payments to women who
provide eggs for scientific research. However, the measure would not
affect the ban on payments to egg providers in research funded by the
$3 billion California stem cell agency. That ban is covered by a
separate legal provision. Stem cell researchers around the country have complained that they they cannot get eggs without payment.
Women in California can be paid for
providing eggs for reproductive purposes. According to a legislative analysis, payments can run as high as $50,000 for women with special
characteristics but average around $9,000 for each session, which can
generate more than one egg.
The sponsor of the legislation is the
American Society for Reproductive Medicine of Alabama, whose members
represent a wide swath of the $5 billion-a-year fertility business.
The measure would open new business avenues for the industry.
Bonilla argues that the measure allows women to be treated on the same footing as men who provide sperm for
research and would encourage more research into reproductive health issues.
Opponents argue that the safety of the
egg production procedures has not been well-established including
their long-term impact. They also argue that allowing payment would
lead to exploitation of poor and minority women.
The bill received its final
legislative approval on July 1 when the Senate passed it on a 24-9
vote.

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