StemCell Therapy

Conflict of Interest: CIRM to End Contract with Consultant Linked to Grant Recipient

May 20th, 2012 3:55 pm

The California stem cell agency will
not renew a contract with a "special advisor" who has been
nominated to the board of directors of a firm that is sharing in a $14.5 million grant from the agency.

She is Saira Ramasastry, managing
partner of LifeSciences Advisory, LLC, of Emerald Lake Hills, Ca.
Ramasastry has worked for CIRM since May of 2010. Last month, she was
nominated to the board of Sangamo BioSciences, Inc., of Richmond, Ca.
Her responsibilities with CIRM have included "industry analysis
and consultation." Sangamo cited her experience with CIRM in its
press release on her nomination. She was also employed as a
consultant by Sangamo, according to the firm.

Ramasastry's dual roles raise obvious
conflict of interest questions. The case highlights the issues
that can arise between CIRM and the biotech industry as the agency
moves to engage industry more closely. CIRM's response additionally
demonstrates a lack of awareness of the potential for serious
mischief or worse when dealing with consultants.

The California Stem Cell Report asked
CIRM on May 6 for comment on the Sangamo-Ramasastry matter. The
questions included whether Ramasastry disclosed to CIRM her work for
Sangamo and whether CIRM took any action per the agency's conflict of
interest code. CIRM did not respond to the question of whether
Ramasastry ever disclosed her ties to Sangamo, which expects to
receive $5.2 million from the CIRM grant if it runs a full four
years.

Here is the text of CIRM's reply today
from spokesman Kevin McCormack.

"Saira Ramasastry was an
independent contractor. As required by law, we do ask independent
consultants to complete Form 700s(statements of economic interests)
if they participate in an agency decision making role. Her role did
not fall into that category - she was identified as a 'special
advisor' in connection with our external review process - and so she
did not have to fill out a Form 700. Her contract with CIRM comes to
an end at the end of June, and she will not be elected to Sangamo's
board of directors until July. Obviously once she is a member of the
Sangamo board she will not be consulting or advising CIRM because of
our strict conflict of interest rules."

(Editor's note: The board election is
June 21, according to the company, not July.)

Our take: CIRM is heavily dependent on
outside contractors. Expenditures for their services are the second
largest item in CIRM's operational budget, exceeded only by salaries
and benefits of regular employees. The responsibilities of outside
contractors cover a wide range of sensitive tasks including computer
system security, development of software that deals with proprietary
information from grant recipients, analysis of confidential business
operations of grant and loan applicants and much more.

The agency needs to know who their
consultants are working for besides CIRM. Whether they make decisions
for CIRM is beside the point. Gathering information that is not
normally accessible to the public can be extremely valuable to
businesses and their competitors as well as applicants for
CIRM's $3 billion. In Ramasastry's case, she was privy to a great
deal of confidential or economically useful information during her work on CIRM's external
review and likely much more.

The use of California's Form 700 is
hardly adequate to assess conflict of interest issues involving
private consultants. The form was developed in the 1970s to deal with
elected officials primarily and provides only the grossest sort of
look at financial holdings and income.

CIRM's current move to embrace industry
requires more scrutiny of conflicting interests – not less. NextThursday the CIRM board will deal with some of its conflict ofinterest rules. It is fine opportunity to ask for a sharper analysis
of conflict issues and consultants with an eye to strengthening CIRM
regulations and ensuring protection of the agency and its grantees'
work – not to mention the interests of the people of California.

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Scripps CEO Joins Stem Cell Agency Board; Love Leaves

May 20th, 2012 3:55 pm

Michael Marletta
Scripps Photo

State Treasurer Bill Lockyer has
appointed Michael Marletta, president and CEO of the Scripps Research
Institute, to the 29-member board of directors of the $3 billion
California stem cell agency.

Marletta fills the seat of Floyd Bloom,
also a Scripps executive, who resigned last year. Scripps has
received $45.3 million in funding from CIRM.

In a letter yesterday to the stem cell
agency, Lockyer said Marletta is a member of the National Academy of
Science, American Academy of Arts and Sciences and the Institute of
Medicine. Marletta joined Scripps in 2011 and became president in
January.

Prior to that, he was at the University
of California, Berkeley, where he once served as chairman of the
department of chemistry, among other roles. An item on the Scripps
web site said Marletta "focused his research on the intersection
of chemistry and biology. He is acknowledged as a pioneer in
discovering the role of nitric oxide, a critical player in
communication between cells."

The CIRM board has another vacancy to
fill. Ted Love resigned last month after serving on the board since
its inception in December 2004. CIRM said Love, executive vice
president of Onyx Pharmaceuticals, resigned for personal reasons.
State Controller John Chiang is considering a number of candidates to
replace him. Love was the only African-American on the board.

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$95 Million in California Stem Cell Grants: Preview the Spending

May 20th, 2012 3:54 pm

For those interested in how the
California stem cell agency is going to spend its next $95 million,
you can check out short digests today of the 19 research grant applications, including reviewer comments, that are virtually certain of receiving the cash.

The applications came in what CIRM
calls its "early translational III" round, which is
scheduled to be acted on by the CIRM board May 24 in San Francisco.

Digests of reviewer comments are
part of the directors' meeting agenda. They include scientific
scores, a statement from the applicant and a summary of what
reviewers had to say during their closed door sessions. But you won't
find the names of the applicants, their institutions or businesses.
The stem cell agency conceals the names of the winners until after
the board acts. Names of the unlucky ones are not disclosed by CIRM.
The agency says it does not want to embarrass anybody including the
institutions involved.

However, persons familiar with the area
of science involved may well be able to discern at least some of the
names of applicants from the information contained in the summaries.

Scientific scores of the successful
applicants ranged from 88 to 53. Nine grants scored higher than 53
but were rejected by reviewers(the Grants Working Group). The panel
turned down 22 applications overall. The CIRM board has final
authority on applications, but has almost never rejected a positive
decision by reviewers. Sometimes, however, it will overrule a
negative decision.

One successful application that was
scored at 53 involved ALS. The $1.7 million proposal was approved
for "programmatic reasons," according to the summary.
Often, programmatic motions for approval are made by CIRM board
members sitting on the review panel. However, the summary did not
disclose who made the motion or the vote. The summary said,

"The programmatic reasons provided
were that ALS is a devastating disease that is not well-represented
in CIRM's portfolio."

The other successful application that
scored at 53 sought $6.3 million for research involving heart
disease. The summary did not clearly identify the specific reason for
approving the grant on a programmatic motion. But it said,

"The
GWG (grants working group) ... advised as a condition for funding
that the applicant consult additional vector specialists with
translational and clinical experience to select a more appropriate
vector to move this program towards the clinic."

Again CIRM withheld the vote on the
motion and the name of the person who made the motion.

Applicants who have been rejected by
reviewers can appeal to the full board. So far no appeals have been
publicly posted by CIRM. The success rate on such appeals is mixed.

The translational round was open to
both academics and businesses, which have received a tiny fraction of
CIRM's $1.3 billion in spending so far. Some businesses have
complained publicly and, as well, to a panel of the Institute of
Medicine that is evaluating CIRM's performance.

The California Stem Cell Report
yesterday asked CIRM for the number of businesses that applied in the
translational round, including the pre-application process, which is
used to whittle down the total number of applications. The request included total numbers as well. CIRM spokesman
Kevin McCormack declined to produce the figures prior to the CIRM
board meeting, saying they "won't be ready" until after the
session.

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IP to Grant Oversight: Study Calls for Host of Improvements at California Stem Cell Agency

May 20th, 2012 3:54 pm

The $3 billion California stem cell
agency is laboring under a range of problems that include protection of
its intellectual property and management of its nearly 500 grants plus an inadequate ability to track its own performance, a seven-month
study said yesterday.

The performance audit by the Moss Adams accounting
firm of Seattle, Wash., made 27 recommendations for improvements,
including more effort to ease strain connected to the agency's
controversial dual executive arrangement. The study said that the
nearly eight-year-old agency has many "opportunities" to
"enhance performance reporting and decision making, strengthen
effectiveness and efficiency, retain essential human resources and
leverage technology."

In response to the report, the stem
cell agency said, "(M)anagement concurs with the findings and
recommendations....The recommendations are focused and constructive.
CIRM is already implementing many of these recommendations, and we
will be investigating the others in the coming months."

The performance audit is the first ever
made of the California Institute for Regenerative Medicine. The
audit is required by state law and was commissioned by the agency at
a cost of $234,944. For years, the agency for years had resisted calls for a
performance audit until it sought legislative approval in 2010 for
removal of a 50-person cap on its staff. Originally, the performance
audit legislation would have put the study in the hands of the only
state body charged with oversight of the agency and its board. CIRM,
however, was successful in lobbying to have that provision removed.

The 54-page report identified once
again a number of issues that have troubled the stem cell agency for
some years. Moss made 12 top priority recommendations, many of which
dealt with information technology and grants management. Many of the
recommendations focused on providing better and faster information on
performance outcomes, which the audit said has been slow to come and
hard to generate.

The report said,

"Key performance information is
not readily available to CIRM leadership and other stakeholders on an
ongoing basis. CIRM board members and senior management do not
receive regularly updated, enterprise-level performance information.
The ability to evaluate performance against strategic goals is
critical to effective leadership and program monitoring, evaluation,
and reporting."

The audit stated,

"CIRM does not effectively
communicate outcome-based performance internally or externally. As
such, CIRM does not focus on performance metrics as part of its
(staff) meeting process."

The report additionally said,

"CIRM does not have an integrated
financial information system....The use of spreadsheets results in
labor intensive processes to generate reports and respond to
information inquiries, since data must be pulled from multiple
spreadsheets, a process that may be prone to error. ...Spreadsheets
are not linked to each other or a master report. CIRM does not have a
comprehensive list of spreadsheets or instructions for how to
maintain the files or generate reports from them."

Moss Adams said that CIRM needed to do
a better job in "bond forecasting," a reference to the
California state bonds that finance virtually every aspect of the
agency's operations. CIRM directors were caught by surprise a few
years ago when they suddenly learned the agency was up against a
major cash crunch.

Some of the recommendations will
require more work from CIRM grantees and their technology transfer
offices in an effort to track intellectual property and grant outcomes.
The report also recommended a speed-up in CIRM's review of progress
reports from grant recipients, which have been lagging completion by
several months.

The dual executive arrangement, which
was written into law by Prop. 71, has troubled CIRM since nearly day
one. CIRM's own external review panel also identified it as problem
two years ago. The executive structure is virtually impossible to
change because of the political difficulty in making alterations in
the ballot initiative.

Moss-Adams said,

"The working relationship between
the chairman’s office and the president’s office has vastly
improved over the past year, but there are still opportunities for
improvement."

The performance audit recommended,

"Make every effort to manage and
operate as one cohesive organization, while recognizing the varying
roles, responsibilities, and authorities that exist with positions in
both the chairman’s office and president’s office."

One of the top 12 recommendations
involved CIRM's public relations/communications effort. CIRM
Chairman J.T. Thomas told directors last June that the agency was in
a "communications war."

Moss-Adams said,

"CIRM does not have a
communication plan, and there is lack of clarity on how to address
mission-based communication to CIRM’s various target audiences,
especially the general public....The best way to facilitate
results-based communications is to 1) quantify goals and outcomes in
CIRM’s strategic plan and 2) report on achievement of those goals
and outcomes by enhancing CIRM’s annual report with additional
performance-based information."

Another performance assessment of the
stem cell agency is also underway. It is being conducted by the
prestigious Institute of Medicine and is costing CIRM $700,000. That
report is expected this fall.

CIRM's board of directors is scheduled
to consider the Moss Adams report at its meeting May 24.

Our take: While the findings and
recommendations of the performance audit were delicately worded in
many cases, they brought out issues that need to be addressed, many
of which have been around for a great deal of time. At their meeting
next week, CIRM directors should act very directly on the
recommendations. They can do that by requiring a written report each
month from CIRM Chairman J.T. Thomas and CIRM President Alan Trounson
on the specific steps that they are taking to implement the
performance audit's recommendations. Otherwise, the inevitable drift
will set in.

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California Budget Slashing Misses Stem Cell Agency

May 20th, 2012 3:54 pm

The $3 billion California stem cell
agency dodged the governor's financial knife today.

This morning, Gov. Jerry Brown
announced sweeping cuts throughout California state government as he
attempted to close a new, $15.7 billion deficit. A report in the Los
Angeles Times said the governor was "grabbing any spare change available." But this afternoon, in response to a
query, Kevin McCormack, CIRM's spokesman, said,

"The answer is no, we won't be
affected."

The question arose because California's
financial picture is much bleaker than it was just four months ago.
And the stem cell agency's only real source of cash is money borrowed
by the state -- general obligation bonds.

Under Prop. 71, which created the
agency in 2004, the bond funds flow directly to the agency without
intervention by the legislature or the governor. However, Brown has
been chary of additional bond sales since they create an increasing
burden in the form of interest costs. Those costs must be financed
out of money that otherwise might go to the University of
California, K-12 schools and medical help for the poor.

Under an agreement arrived at last year, CIRM has what amounts to a $225 million line of credit with the
state, which should take care of its needs until January. The cash is
coming from short-term borrowing by the state instead of bonds.

The Brown Administration has cut back
on bond borrowing and intends to cut more this fall. According to the state Department of Finance, the cost of borrowing
has declined $173 million this fiscal year, down to $5.2 billion.
CIRM's share of the debt service is more than $200,000 a day.

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Central nervous system stem cells shed light on mechanism that controls asymmetrical division

May 20th, 2012 9:12 am

Figure 1: Neuroblasts localize polarity complexes (green) on the epithelial side (top) and divide perpendicular to the epithelium in a normal Drosophila embryo (scale bar, 10 m). Credit: 1 2012 Elsevier Inc.

Animals consist of many distinct cell types, all of which originate during development from a single cell: the fertilized egg. To generate this vast cellular diversity, the egg and its descendants must divide unevenly to produce new cells with different fates. Nowhere is this process more important than in the central nervous system, where the asymmetric division of neural stem cells called neuroblasts contributes to the profusion of neurons and glial cells.

Several proteins assemble into so-called polarity complexes that localize at one end of the neuroblast to help guide this unbalanced division (Fig. 1). But the mechanism that controls the orientation of these complexes has remained elusive. Now, a team of RIKEN biologists has discovered the master regulator that directs how these proteins are laid down in the neuroblasts of developing fruit fly, or Drosophila, embryos.

Fumio Matsuzaki and his colleagues at the RIKEN Center for Developmental Biology, Kobe, screened various mutant Drosophila embryos for defects in neuroblast polarity. They uncovered an important player in this process: a gene called trapped in endoderm 1 (Tre1), which encodes a transmembrane receptor protein. In a series of experiments with fly strains in which they deleted the Tre gene, the researchers showed that this receptor is necessary to orient the polarity of the protein complexes in a perpendicular direction relative to the neighboring epithelial cell layer.

Further dissections of proteinprotein interactions revealed that Tre1 recruits and orthogonally orients a critical polarity complex, known as Par, through a cascade of apically localized protein intermediaries. First, Tre1 activates a subunit of an important signal transducing molecule to recruit the protein Pins, which regulates spindle orientation. Another protein, called Inscuteable, then acts as a molecular link between Pins and Par to ensure that every component is in the proper location.

The Par-complex is known to regulate the formation of cell polarity in various cell types including stem cells and neurons, explains team member and co-author Shigeki Yoshiura. So this process might be involved in the orientation of the polarity of various cell types during development.

With Tre1 emerging at the top of the hierarchy controlling the orientation of polarity complexes in the neuroblast, Matsuzaki and colleagues are turning their attention to finding its regulator. We still do not know which molecule or molecules act as the extrinsic signal from epithelial cells, Yoshiura says. The RIKEN team is also investigating whether this mechanism is conserved through evolution and is applicable to mammalian neural stem cells.

More information: Yoshiura, S., et al. Tre1 GPCR signaling orients stem cell divisions in the Drosophila central nervous system. Developmental Cell 22, 7991 (2012).

Provided by RIKEN

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Central nervous system stem cells shed light on mechanism that controls asymmetrical division

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Michael J. Fox Looks Past Stem Cells

May 20th, 2012 9:12 am

Michael J. Fox, whose turn from Parkinsons disease patient to scientific crusader made him one of the countrys most visible advocates for stem cell research, now believes the controversial therapy may not ultimately yield a cure for his disease, he told ABCs Diane Sawyer in an exclusive interview.

There have been problems along the way, Fox said of stem cell studies, for which he has long advocated. Instead, he said, new drug therapies are showing real promise and are closer today to providing a cure for Parkinsons disease, a degenerative illness that over time causes the body to become rigid and the brain to shut down.

Stem cells are an avenue of research that weve pursued and continue to pursue but its part of a broad portfolio of things that we look at. There have been some issues with stem cells, some problems along the way, said Fox, who suffers from the diseases telltale tics and tremors.

Its not so much that [stem cell research has] diminished in its prospects for breakthroughs as much as its the other avenues of research have grown and multiplied and become as much or more promising. So, an answer may come from stem cell research but its more than likely to come from another area, he said.

Tune in to World News with Diane Sawyer Friday at 6:30 p.m. E.T. to see more of Diane Sawyers interview with Michael J. Fox

Fox, who recently appeared in episodes of Curb Your Enthusiasm and The Good Wife, has dedicated himself to finding a cure for Parkinsons, the disease with which he was diagnosed in 1991.

Fox said he still strongly believes in stem cell research and government support of those studies, praising ongoing research at New Yorks Memorial Sloan-Kettering Hospital. When asked about earlier criticism he received from conservative talk show host Rush Limbaugh about his advocacy, Fox said it only sharpens your resolve.

Scientists are conducting research and looking for a cure on multiple fronts, Fox said, including drug therapies, experimental surgeries, and developing tests to help make earlier diagnoses.

To that end, his Michael J. Fox Foundation for Parkinsons Research, the largest private funder of Parkinsons disease research worldwide, has recently launched an online initiative to increase studies across the country by pairing patients with clinical trials in their areas.

The Fox Trial Finder(Visit FoxTrialFinder.org for more info on clinical trial participation) harnesses the power of the Internet to find patients and, based on their profile of symptoms, pair them with research scientists conducting clinical trials.

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Michael J. Fox Looks Past Stem Cells

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Stem Cells Used To Repair Damaged Teeth

May 20th, 2012 9:12 am

SALT LAKE CITY -- Utahs scandal-plagued, state-owned liquor business will have a new management scheme. After weeks of back-room negotiations, legislators and the governor have found a compromise.

Right now, in Utah the governor is mostly excluded from liquor policy. He will get a lot more power in liquor. But he will have to share power with the independent liquor commission. All of this change is in the hope of avoiding new scandals.

We have reached a compromise on the governance for alcohol policy for the state of Utah, said Sen. John Valentine. Governor Gary Herbert wanted the liquor commission to disappear. He wanted the governor to run liquor, as he runs other state departments. Prior to 1977, we had it all under the executive branch, said Valentine. But Valentine studied history. When liquor was under the governor, distilleries donated booze to the governor's fundraising gala. And in 1976, under Governor Calvin L. Rampton, there was a scandal. We ended up with five indictments. We ended up with a governor who ended up with 30 cases of booze up at the mansion that we couldnt account for, said Valentine. So the legislature created the liquor commission. But now under the commission, former Liquor Chief Dennis Cullen is accused of insider dealing with a firm owned by his son. In running the state liquor business, Utah moves from scandal to scandal. Utah has tried the governor running liquor, and tried the commission running liquor, and after this latest scandal, Utah will try a combination of the governor and the commission.

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Stem Cells Used To Repair Damaged Teeth

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Multipotent stromal stem cells from normally discarded human placental tissue demonstrate high therapeutic potential

May 20th, 2012 9:12 am

ScienceDaily (May 18, 2012) Scientists at Children's Hospital Oakland Research Institute (CHORI) led by Vladimir Serikov, MD, PhD, and Frans Kuypers, PhD, report in the current Epub issue of Stem Cells Translational Medicine that placental stem cells with important therapeutic properties can be harvested in large quantities from the fetal side of human term placentas (called the chorion).

The chorion is a part of the afterbirth and is normally discarded after delivery, but it contains stem cells of fetal origin that appear to be pluripotent -- i.e., they can differentiate into different types of human cells, such as lung, liver, or brain cells. Since these functional placental stem cells can be isolated from either fresh or frozen term human placentas, this implies that if each individual's placenta is stored at birth instead of thrown away, these cells can be harvested in the future if therapeutic need arises. This potential represents a major breakthrough in the stem cell field.

In previous work, Drs. Serikov and Kuypers reported a novel technology to harvest blood-forming stem cells from the placenta to augment cord blood cells. These cells are "siblings" of the cord blood derived stem cells. Cord blood stem cells, unlike embryonic stem cells, have been used for many hundreds of successful bone marrow transplants. These transplants are mainly performed in children, as the amount of cells that can be harvested from cord blood is usually not sufficient for a successful transplant in adults. Adding placental-derived stem cells to the cord blood stem cells could make successful adult bone marrow transplants routinely possible.

The current report demonstrates that placental stem cells have much broader therapeutic potential than bone-marrow transplants, because they are pluripotent -- i.e. able to differentiate into many different cell types -- and they also generate growth factors that help in tissue repair. These cells are shown to integrate into different tissues when transplanted into mice, but like cord blood stem cells, and in contrast to embryonic pluripotent stem cells, they do not form tumor-like structures in mice.

Placental-derived stem cells are often viewed as "adult" stem cells in contrast to "embryonic" stem cells, which are the dominant focus in the stem cell research field. However, this report shows that these fetal stem cells can be harvested in large numbers, and without the ethical concerns attached to the use of embryonic stem cells. These stem cells may thus be a more practical source for regenerative medicine, particularly since, if placentas are routinely saved instead of thrown away, each individual will be able to draw on their own fetal stem cells if future therapeutic needs arise.

Placental stem cells are only 9 months old, and in contrast to adult stem cells, do not need to be reprogrammed to become pluripotent. Placental-derived stem cells have characteristics of young and vigorous cells, including young mitochondria. Future research will be aimed to bring this to the clinic and to test their efficacy in translational therapeutic applications.

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The above story is reprinted from materials provided by Children's Hospital & Research Center Oakland, via Newswise.

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/C O R R E C T I O N — Amgen/

May 20th, 2012 9:12 am

In the news release, Amgen's BiTE Antibody Blinatumomab (AMG 103) Achieved High Rate of Complete Response in Adult Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia, issued 16-May-2012 by Amgen over PR Newswire, we are advised by the company that the third paragraph now includes additional safety information. The complete, corrected release follows:

THOUSAND OAKS, Calif., May 16, 2012 /PRNewswire/ --Amgen (AMGN) today announced updated results from a Phase 2 study that showed treatment with blinatumomab (AMG 103) helped achieve a high-rate of complete response (CR) in 72 percent of adult patients with relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL) treated in the study. Blinatumomab is the first of a new class of agents called bi-specific T cell engagers (BiTE) antibodies, designed to harness the body's cell-destroying T cells to kill cancer cells. Blinatumomab targets cells expressing CD19, a protein found on the surface of B-cell derived leukemias and lymphomas, such as ALL. Full results of the study will be presented during an oral abstract session at the 48th Annual Meeting of the American Society of Clinical Oncology (ASCO) on June 4. (Abstract Number 6500; Oral Presentation, 8:00 a.m. - 8:15 a.m. CDT, E354a).

In this Phase 2 single-arm dose-ranging trial, 26 of the 36 patients treated with blinatumomab across all of the tested doses and schedules achieved a CR or complete response with partial hematologic recovery (CRh*). All but two patients achieved a molecular response, meaning there was no evidence of leukemic cells by polymerase chain reaction.

For patients who received the selected dose and schedule, the most common adverse events were grade one or two and included pyrexia (70 percent), headache (39 percent), tremor (30 percent) and fatigue (30 percent). These were most frequently seen at the onset of treatment in cycle one. Reversible central nervous system events led to treatment interruptions in six patients with two patients permanently discontinuing treatment. Cytokine release syndrome led to treatment interruption in two patients. One patient had a fatal event of fungal infection that the investigator considered related to treatment.

At the time of the analysis, median survival was 9.0 (8.2, 15.8) months with a median follow-up period of 10.7 months. In the group of patients who received the selected dose, median survival was 8.5 months. The median duration of response in the 26 patients who responded to treatment was 8.9 months.

"For these patients with limited treatment options, the remission rate observed in the trial is a vast improvement over the current standard of care," said Professor Max Topp, Department of Internal Medicine II, University of Wuerzburg and chair of the study. "These results also represent significant progress in our research of immunotherapies; a new approach to fighting cancer that we believe could make a real difference for patients."

Phase 2 Study Design

This Phase 2 dose-ranging study evaluated the efficacy, safety and tolerability of blinatumomab in adult patients with B-precursor ALL who had relapsed following treatment with standard front-line chemotherapy or allogeneic stem cell transplant. Patients received blinatumomab for 28 days followed by two weeks off therapy over a six week treatment cycle, for up to five treatment cycles. Patients received a continuous intravenous infusion of blinatumomab at an initial dose of five or 15 micrograms per meter squared per day, ranging up to 30 micrograms for the remainder of the treatment. The primary endpoint of the study was the rate of CR/CRh*. Secondary endpoints included molecular response rate, duration of response and overall survival. As of April 13, 2012, all 36 patients were evaluable for efficacy and safety.

In addition to the results from this study, data from studies of 12 Amgen investigational molecules and marketed products will be presented at the ASCO Annual Meeting. These include results from studies of the immunotherapy talimogene laherparepvec, pipeline molecules such as rilotumumab (AMG 102) and AMG 386 and marketed products. A complete listing of Amgen abstracts of interest can be found at http://www.amgen.com/media/featured_content.html. Abstracts are available online at http://www.asco.org.

About Blinatumomab

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/C O R R E C T I O N -- Amgen/

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Advanced Cell Technology to Present at World Stem Cells & Regenerative Medicine Congress in London

May 20th, 2012 9:12 am

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that chairman and CEO Gary Rabin will be presenting at the World Stem Cells and Regenerative Medicine Conference, May 21-23, in London.

Mr. Rabins presentation, titled Successes and ongoing advancements of human clinical trials for the treatment of AMD & Stargardts Disease, will be given on Monday, May 21 at 5:05 p.m. BST (London time). Mr. Rabin will provide an update on ACTs three ongoing human clinical trials in the U.S. and E.U. for Dry Age-Related Macular Degeneration (Dry AMD) and Stargardts Macular Dystrophy (SMD).

ACT recently announced Data and Safety Monitoring Board (DSMB) approval to move forward with enrollment and treatment of additional patients with SMD in its U.S. SMD trial, and to treat the final two patients to round out the initial dosing arm in its European trial. All three of the companys ongoing clinical trials use human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells.

About SMD, Dry AMD and Degenerative Diseases of the Retina

Stargardts Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. SMD causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium or RPE cell layer.

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as thirty million people in the United States and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority the dry form of AMD which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

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Advanced Cell Technology to Present at World Stem Cells & Regenerative Medicine Congress in London

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First stem cell drug approved for systemic disease treatment

May 20th, 2012 9:10 am

Osiris Therapeutics Inc said on Thursday that Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.

Osiris shares rose 14 percent to $6.00 in extended trading after the news was announced.

Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient's body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.

The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.

Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company's chief executive, said in an interview that could take three to four years.

Some investment analysts have been skeptical about Prochymal's future. In 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.

Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD -- those who have more or less failed all other therapies -- Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 percent of patients.

In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.

The Canadian authorities approved the drug on the basis of that data, the company said.

FDA submission this year

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First stem cell drug approved for systemic disease treatment

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Prochymal approval makes Canada first country to OK stem cell therapy

May 20th, 2012 9:10 am

Toni Clarke BOSTON Globe and Mail Update Published Friday, May. 18, 2012 12:38PM EDT Last updated Friday, May. 18, 2012 12:56PM EDT

Osiris Therapeutics Inc. OSIR-Q said on Thursday that Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.

Osiris shares rose 14 per cent to $6.00 in extended trading after the news was announced.

Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient's body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.

The disease kills up to 80 per cent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.

Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company's chief executive, said in an interview that could take three to four years.

Some investment analysts have been skeptical about Prochymal's future. In 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.

Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD those who have more or less failed all other therapies Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 per cent of patients.

In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.

The Canadian authorities approved the drug on the basis of that data, the company said.

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Prochymal approval makes Canada first country to OK stem cell therapy

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Family hangs hope for boy on unproven therapy in India

May 20th, 2012 9:10 am

Indian clinic's stem cell therapy real?

STORY HIGHLIGHTS

For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.

New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

That's when Cash began walking with the aid of braces for the first time.

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Family hangs hope for boy on unproven therapy in India

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Medical success or boondoggle?