IOM and California Stem Cell Agency: Study Lacks Key Perspective


The prestigious Institute of Medicine earlier this month kicked off its $700,000 study of the California stem cell agency minus an important perspective – the view directly from California.

None of the persons on the 13-member panel evaluating the performance of the $3 billion enterprise comes from California. The reasons for that are not clear. The IOM is all but mum on the matter.

One could argue that it is not necessary to be geographically located in California to determine whether CIRM is working at peak performance. However, some conditions do exist in California that are difficult for many others to grasp. They include its state budget crisis that has now placed the once Golden State at the bottom of the heap in terms of its credit. Some even liken it to Greece. Obviously that situation can be understood in the abstract by reading The Sacramento Bee and the Los Angeles Times. But the intensity and emotion surrounding that issue and others are difficult to comprehend for many folks living in more blessed states.

Count among the other volatile issues the cutbacks in the state's once vaunted higher education system, including the University of California, which showed its back to students by increasing tuition by nearly 18 percent this fall. Couple that with a visceral antipathy -- and that is putting it mildly -- among some Californians to what they regard as execessive state salaries, including those at the stem cell agency.

What does all this have to with financing stem cell research through an agency that was supposed to have a guaranteed stream of income isolated from mischief that could be wreaked by the governor or legislature. It turns out that CIRM's cash flow is not as guaranteed as its backers believed. Instead of issuing bonds, the state is going to finance the agency over the next 18 months with commercial paper, if necessary. That's because Gov. Jerry Brown wants to reduce the interest costs on state borrowing, which have risen sharply and now consume 8 percent of the state budget along with funds that could otherwise go to educate California's children, among other things.

Brown's parsimony is famous. During his first term in office, he denied pay raises to state college professors, saying they are amply rewarded through "psychic income." More recently, he objected to out-of-state travel by CIRM staff. Too much "lollygagging in London on the taxpayer's dime," a Brown spokesman said. CIRM Chairman Jonathan Thomas promptly cut travel in the chairman's office by 50 percent and asked CIRM President Alan Trounson to do the same for the rest of the staff.

It is an environment that can be difficult to navigate under the best of circumstances. It places limitations on the stem cell agency and tends to focus its operations and funding in different directions than might be the case if California's economic climate were rosier.

The IOM has no real response to the question of why no Californian is on the panel. The California Stem Cell Report last week asked the institute whether an overt decision had been made to exclude persons from California. The IOM did not answer directly. Instead it referred to a generic description of how panel members are selected. The institute's unwillingness to address the specific question does not speak well for the openness and transparency of the IOM examination of CIRM.

Currently the IOM does have a comment period available on the selection of the panel members, who will not become official for another three days. Interested parties can make their views known to the IOM by using this link.

A Californian or two on the IOM panel would help to bring a valuable, broader perspective to this important study, which is sure to affect the future of the state's stem cell research and voter approval of another possible multibillion dollar bond issue in the next few years.

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

International Stem Cell Corporation and The Automation Partnership Enter into Strategic Alliance to Automate Cornea Tissue Production

OCEANSIDE, CA – May 18, 2010 – International Stem Cell Corporation (ISCO.OB), http://www.intlstemcell.com, announced today that it had entered into a strategic alliance with The Automation Partnership (TAP), http://www.automationpartnership.com, to automate and scale up the production of stem cell-derived human corneal tissue. The alliance has been formed to create instrumentation for ISCO and its partners and affiliates to produce development and commercial volumes of donor tissue for cornea transplantation and to reduce the use of animals and animal eyes in safety testing of drugs, chemicals and consumer products.

Cornea-related loss or reduction of vision can be caused by physical injury, infections and degenerative diseases. In cases where cornea replacement is indicated, current medical practice typically involves a one-two hour outpatient procedure under local anesthesia using full or partial corneas from healthy human cadavers. 10 million people worldwide are candidates for such treatment, primarily in Asia and Europe where there is significant quantitative and qualitative shortage of human cornea donation.

Global efforts are underway to transition from the use of live animals and excised animal eyes to test drugs, chemicals and consumer products. For example, Europe’s Registration, Evaluation, Authorisation and Restriction of Chemicals (REACH) estimates a need to spend €270M and use 160,000 animals for eye safety testing alone to catch up with the back-log of insufficiently tested agents. In the US, the National Institutes of Health (NIH) and the Environmental Protection Agency (EPA) have launched a five-year program dedicated to finding new, non-animal technologies for toxicity testing of chemical compounds.

ISCO has discovered and filed for patents on a cell culture process for the synthesis of standardized, human, corneal tissue using stem cells. Histology, permeability and optical testing has demonstrated compatibility with natural corneas. Efforts are ongoing to further characterize this tissue and standardize and scale up its synthesis. Automation is necessary to produce sufficient, reproducible tissue for development and commercialization of the therapeutic and toxicity testing applications.

Brian Lundstrom, ISCO’s President, says: “Given the substantial unmet therapeutic and toxicology testing needs for human corneal tissue, ISCO has embarked on a focused effort to advance this technology with international investors, eye clinics, and development and commercialization partners. After reviewing a range of potential cell culture automation companies, we are very pleased that TAP has agreed to contribute their over twenty years of experience towards this goal.”

David Newble, TAP’s CEO says: “TAP has successfully installed and supported over 160 automated cell culture systems worldwide and continues to design new and customize existing equipment for cell culture and other emerging life science applications. The opportunity to join forces with ISCO in the cornea tissue area will enable us to contribute solutions for clear biomedical needs while also creating new technology and knowhow that will be useful in other applications downstream.”

ABOUT INTERNATIONAL STEM CELL CORPORATION (ISCO.OB):
International Stem Cell Corporation is a California-based biotechnology company focused on therapeutic and research products. ISCO’s core technology, parthenogenesis, results in creation of pluripotent human stem cells (hpSCs) from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenic, homozygous stem cell line that can be a source of therapeutic cells with minimal immune rejection after transplantation into hundreds of millions of individuals of differing sexes, ages and racial groups. This offers the potential to create the first true stem cell bank, UniStemCell™, while avoiding the ethical issue of using fertilized eggs. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology. More information is available at ISCO’s website,
http://www.internationalstemcell.com

To subscribe to receive ongoing corporate communications please click on the following link: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0.

About The Automation Partnership
The Automation Partnership (TAP) provides advanced automation systems and services to improve productivity in life science research, development and production.


For over 20 years TAP has been the leader in the design and development of automated cell culture and processing systems with applications in regenerative medicine, discovery research, bioprocess development and biologics production. Systems include SelecT, CompacT SelecT and Cellmate.

In addition, TAP has a strong track record in providing a range of custom automation solutions for biobanking, biological sample management, compound management and advanced high throughput screening applications.

TAP is a privately-owned company with headquarters near Cambridge, UK and a sales and support facility near Wilmington, Delaware, USA.

FORWARD-LOOKING STATEMENTS
Statements pertaining to anticipated technological developments and therapeutic applications, and other opportunities for the company and its subsidiary, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "should," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update these forward-looking statements.

Key Words: Stem Cells, Biotechnology, Parthenogenesis

CONTACTS:
International Stem Cell Corporation
Kenneth C. Aldrich, Chairman
760-940-6383
kaldrich@intlstemcell.com
Or
Brian Lundstrom, President
760-940-6383
bl@intlstemcell.com
Or
The Automation Partnership
Matthew Walker
Head of Communications
+44 1763 227 200
Matthew.walker@automationpartnership.com

Phase I clinical trial of ICT-107

Immune response correlation with progression-free survival in glioblastoma following dendritic cell immunotherapy (ICT-107) by Surasak Phuphanich and 9 co-authors, including Manish Singh, Keith Black and John Wu, J Clin Oncol 28:7s, 2010 (suppl; abstr 2097). To be presented at the 2010 ASCO Annual Meeting, June 06, 2010.

Related news releases:

ImmunoCellular Therapeutics Ltd. (IMUC) to Present Cancer Vaccine Candidate, International Business Times, June 02, 2010. Excerpt:

Data from the company’s recent clinical trial of ICT-107, the company’s dendritic cell-based cancer vaccine candidate, will be presented at the 46th Annual Meeting of the American Society of Clinical Oncology (ASCO) June 4-8 in Chicago.

See also: Immunocellular brain cancer vaccine shows promise, Reuters, June 02, 2010. Excerpt:

"We are targeting specific antigens that are on cancer stem cells ... the only population of cells that can really propagate a tumor," said Dr. John Yu, director of surgical neuro-oncology at Cedars-Sinai Medical Center in Los Angeles and ImmunoCellular's chief scientific officer.

Another related news release: Immunocellular Therapeutics Enters into Research Agreement with University of Pennsylvania to Support Phase II Clinical Trial of ICT-107, Business Wire, April 21, 2010.

Putative tumor-initiating progenitor cells predict poor lung cancer prognosis

Adult lung stem cells, vital to injury repair, associated with poor cancer prognosis, News release, UCLA Newsroom, August 17, 2010. Excerpts:

Adult stem cells that are vital for airway repair in the lung but that persist in areas where pre-cancerous lesions are found are associated with a poor prognosis in patients who develop cancer, even those with early-stage disease, researchers at UCLA's Jonsson Comprehensive Cancer Center have found.

.....

In this study, Gomperts and her team screened around 900 tumors removed from patients with non-small cell lung cancer at UCLA and the University of Texas' MD Anderson Cancer Center, looking to see whether the adult stem cells could be found in the tumor. In her lab, Gomperts is now studying the pre-cancerous lesions where the adult stem cells persist in an attempt to uncover the cascade of molecular events that may transform these cells into lung cancer stem cells.

The news release is based on this publication: Presence of a Putative Tumor-Initiating Progenitor Cell Population Predicts Poor Prognosis in Smokers with Non–Small Cell Lung Cancer by Aik T Ooi and 19 co-authors, including Brigitte N Gomperts, Cancer Res 2010(Aug 15); 70(16): 6639-48. [PubMed citation][Full text]. Abstract:

Smoking is the most important known risk factor for the development of lung cancer. Tobacco exposure results in chronic inflammation, tissue injury, and repair. A recent hypothesis argues for a stem/progenitor cell involved in airway epithelial repair that may be a tumor-initiating cell in lung cancer and which may be associated with recurrence and metastasis. We used immunostaining, quantitative real-time PCR, Western blots, and lung cancer tissue microarrays to identify subpopulations of airway epithelial stem/progenitor cells under steady-state conditions, normal repair, aberrant repair with premalignant lesions and lung cancer, and their correlation with injury and prognosis. We identified a population of keratin 14 (K14)-expressing progenitor epithelial cells that was involved in repair after injury. Dysregulated repair resulted in the persistence of K14+ cells in the airway epithelium in potentially premalignant lesions. The presence of K14+ progenitor airway epithelial cells in NSCLC predicted a poor prognosis, and this predictive value was strongest in smokers, in which it also correlated with metastasis. This suggests that reparative K14+ progenitor cells may be tumor-initiating cells in this subgroup of smokers with NSCLC.

On the low frequency of tumor-initiating cells

Tumor-Initiating Cells Are Rare in Many Human Tumors by Kota Ishizawa and 16 co-authors, including Benjamin G Neel and William Matsui, Cell Stem Cell 2010(Sep 3); 7(3): 279-282. [FriendFeed entry][PubMed citation]. Abstract:

Tumor-initiating cells (TICs) are defined by their ability to form tumors after xenotransplantation in immunodeficient mice and appear to be relatively rare in most human cancers. Recent data in melanoma indicate that the frequency of TICs increases dramatically via more permissive xenotransplantation conditions, raising the possibility that the true frequency of TICs has been greatly underestimated in most human tumors. We compared the growth of human pancreatic, non-small cell lung, and head and neck carcinomas in NOD/SCID and NSG mice. Although TIC frequency was detected up to 10-fold higher in NSG mice, it remained low (<1 in 2500 cells) in all cases. Moreover, aldehyde dehydrogenase-positive (ALDH(+)) and CD44(+)CD24(+) cells, phenotypically distinct cells enriched in TICs, were equally tumorigenic in NOD/SCID and NSG mice. Our findings demonstrate that TICs are rare in these cancers and that the identification of TICs and their frequency in other human malignancies should be validated via primary tumors and highly permissive xenotransplantation conditions.

International Stem Cell Corporation Announces Commercial Sales to General Public of Its Breakthrough Stem Cell-Based Lines of Skin Care Products


International Stem Cell Corporation (OTCBB:ISCO), http://www.internationalstemcell.com, announced today that the first new topical skin care products of its subsidiary, Lifeline Skin Care, are now available to the general public within the United States at http://www.lifelineskincare.com. Lifeline Skin Care's first two products consist of a Defensive Day Moisture Serum and Recovery Night Moisture Serum. Previously, these breakthrough serums, containing extracts from "parthenogenetic" stem cells, were available only to individuals and organizations formerly identified and selected for their interest in innovative approaches to skin care.

According to Lifeline Skin Care CEO, Dr. Ruslan Semechkin, "Because the quality products Lifeline Skin Care offers are experiencing strong demand and the human stem cell extracts require innovative manufacturing processes, we chose to develop our sales channels gradually and incrementally. We are very pleased with the customer feedback from our first limited launch and are delighted to now be able to expand production and make the products available to the general public within the US."

Purchases that could previously be made only by phone, can now be made directly through the company's website,http://www.lifelineskincare.com. "Enabling electronic order processing though our website provides the benefit of both lowering our transaction costs and increasing our scalability, while simultaneously simplifying the ordering process for our customers," said ISCO VP, Dr. Simon Craw.

ABOUT INTERNATIONAL STEM CELL CORPORATION (ISCO.OB)

International Stem Cell Corporation is a California-based biotechnology company focused on therapeutic and research products. ISCO's core technology, parthenogenesis, results in creation of pluripotent human stem cells from unfertilized oocytes (eggs). These proprietary cells avoid ethical issues associated with use or destruction of viable human embryos and, unlike most other major stem cell types, can be immune matched and be a source of therapeutic cells with minimal rejection after transplantation into hundreds of millions of individuals of differing racial groups. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary, Lifeline Cell Technology, and is developing a line of cosmeceutical products via its subsidiary, Lifeline Skin Care. ISCO is advancing novel human stem cell-based therapies where cells have been proven to be efficacious but traditional small molecule and protein therapeutics have not. More information is available on ISCO's website, http://www.internationalstemcell.com.

To subscribe to receive ongoing corporate communications please click on the following link: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0.

FORWARD-LOOKING STATEMENTS

Statements pertaining to anticipated developments, product introduction plans and related support, the potential benefits of planned products, anticipated sales growth for recently introduced products, and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products and the management of collaborations, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.

Key Words: Stem cells, parthenogenesis, biotechnology, skin care, anti-aging

International Stem Cell Corporation

Kenneth C. Aldrich, Chairman

1-760-940-6383

kaldrich@intlstemcell.com

or

Lifeline Skin Care, Inc.

Ruslan Semechkin, PhD, President & CEO Vice President, ISCO

ras@intlstemcell.com

Two More Disease Team Applications Sent Back for More Review


Directors of the California stem cell agency today referred two additional research applications for $37 million in disease team funding back to reviewers for further consideration.

Both were the subjects of appeals by researchers whose proposals were rejected by grant reviewers.

One was from Timothy Hoey of OncoMed Pharmaceuticals in Redwood City, who sought $20 million.  The other was from Henry Klassen of UC Irvine, who sought $17 million. (See here and here for their appeals.)

The  board began the day by directing staff to come back to the board in early September. But with the large number of grants to be reassessed, it was acknowledged some might not be acted on until the board's meeting in late October.

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

News Coverage of CIRM Awards: Substantial but Not Extensive


The California stem cell agency today
enjoyed substantial, if sparse, news coverage of the

$151 million in research funding
approved by its board yesterday.
Both the San Francisco Chronicle and
The Sacramento Bee carried solid stories on the grant awards. In the
case of the Chronicle, the story included compelling photos of
patients who spoke during emotional, tear-filled presentations.
The stories represent a modest change
from the past, when media outlets all but ignored the agency's
awards.
The Bee's Richard Chang wrote,

“For Melissa Biliardi of Santa
Maria,
 the (CIRM board) vote symbolizes hope. Her son, James
Birdsall
, 32, was diagnosed four years ago with Huntington's disease.
The degenerative brain disorder could prove fatal over the next 10 to
15 years. There is currently no cure or treatment, but with the
grant, UC Davis researchers hope to deliver an effective therapy in
four years.
"'This is the most hope we've ever
had for a cure or treatment,' Biliardi said.”

The Chronicle's Erin Allday wrote,

“California's stem cell funding
agency on Thursday approved nearly $100 million in grants for
research into heart disease, cancer and spinal cord injuries, and to
the cheers of dozens of patients and their supporters, it also
awarded money to rare but devastating diseases with no cure.”

The articles demonstrated the
effectiveness of patients and patient advocates in telling the CIRM
story. Reporters are always looking for a warm human dimension –
especially to enhance a dry, bare-bones science and government story.
Responding to a question from the
California Stem Cell Report, Kevin McCormack, spokesman for CIRM,
also mentioned radio news coverage in the San Francisco Bay Area. He
said the awards were covered in “two different stories on KCBS-AM
radio, one that ran several times yesterday and another that ran
several times today. KGO-AM radio also ran a story several tim, and,
of course, the best of all, today's KQED-FM Forum.”
The KQED show, which was also carried
nationally on Sirius radio, consisted of an hour-long look at CIRM,
with some calls from listeners. Guests on the show were CIRM
President, Alan Trounson, UC Davis stem cell researcher Jan Nolta and
yours truly, David Jensen.
Other stories appeared in the SanFrancisco Business Times and Genetic Engineering News. The Bee's
story appeared in the Modesto Bee as well.

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

California's $12.4 Million Stem Cell Recruitment Lure


Directors of the California stem cell
agency next Thursday are likely to approve spending $12.4 million to
lure a couple of stem cell stars to the Golden State.

It is part of a $44 million recruitment
program that has brought three highly regarded scientists to three
California research institutions, all of which have representatives
on the CIRM board. (See here, here and here.)
As usual, the $3 billion stem cell agency does not
identify the potential recipients in advance of the meeting or the
institutions that are recruiting them. However, if you have a modicum
of knowledge about the specific fields involved, it is likely that
you can identify them based on the information in CIRM's review summaries and some Internet searching.
One of the proposed research grants–a
$5.7 million award--would go a scientist who won raves from CIRM's
reviewers. The researcher was described as an “exceptional
scientist and one of the leading young developmental biologists.”
Reviewers gave his proposal a score of 90 and, in summary, said,

“Major strengths include the
candidate's exceptional productivity and contributions to the fields
of mammalian embryology and kidney development, the significance and
potential of the research program, the PI's proven leadership
capabilities, and the outstanding institutional commitment.”

 The other grant was larger–$6.7
million–but reviewers raised a number of questions about the
candidate although they recommended it for funding. The
review summary ranked the application at 57 and said,

“In summary, this is an application
from an established leader in NSC biology to pursue research focused
on disease mechanisms in PD. Strengths of the proposal include the
quality of the PI, the focus of the project on an interesting
hypothesis, and the leadership in basic science that the candidate
would bring to the applicant institution. Weaknesses included
deficiencies in the research plan, the limited track-record of the PI
in PD research and an institutional environment lacking adequate
support for basic science investigations.“

Last January, in a rare move, CIRM
directors rejected a $6.3 million recruitment grant with a score of
76 sought by the Buck Institute, which is not represented on the
board.
The proposals are scheduled to be acted
on at a public CIRM board meeting in Burlingame, Ca.

(Editor's note: an earlier version of this item incorrectly said the total of both grants was $13.4 million.)

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

Disease Team Round Hits $151 Million with Final Action


Directors of the California stem cell
agency approved an 
additional
$20 million disease team grant
 today before adjourning their
meeting. The grant brought the disease team round to a total of $151 million.

The award went to Judith  Shizuru 
of Stanford. Scientist Irv Weissman and Robert
Klein
, former chairman of the stem cell agency, both spoke on behalf
of her
appeal of a negative decision
 by grant reviewers. 
At the suggestion of the current board
chairman, J.T. Thomas, the board placed conditions on the grant
would stipulate Stanford pick up certain unknown, additional costs if
necessary. 
Here is a link to the CIRM press release on today's action.

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

Stem Cell Agency Board Sticks with More Financial Disclosure


The governing board of the $3 billion
California stem cell agency today rejected a proposal that would have
restricted transparency surrounding the financial interests of its
directors and top executives.

On a unanimous voice vote, the board
decided it would stick with the more complete disclosure rules that it
has operated under since 2005. CIRM staff had offered changes that would have narrowed the amount of economic
information that the board members and the executives would have been
required to disclose.
The directors' Governance Subcommittee, however, on May 3 rejected the plan. Sherry Lansing, a former
Hollywood film studio CEO and chair of the subcommittee, said at the time,

"I personally feel strongly that
because of CIRM's unique mission and the agency's incredibly
long-standing commitment to transparency, i believe that we should
continue to set an example by requiring the broadest disclosure for
members of the board and high level staff."

Retention of existing disclosure
rules comes at a time when more conflicts may arise. The agency is
moving to engage the biotech industry more closely as it pushes to
develop stem cell therapies. Already one case of conflict has arisen this
year dealing with industry. It involves a "special advisor"
to CIRM who was nominated to become director of a firm sharing in a
$14.5 million grant. She also was working for the firm. (See here and
here.)
The CIRM board also has built-in
conflicts of interest, written into the law by Proposition 71, which
created the agency. About 92 percent of the $1.3 billion awarded so
far has gone to institutions tied to members of the CIRM governing
board. Board members are not permitted, however, to vote on or
discuss grants to their institutions. But it is fair to say that if
California voters had foreseen that nearly all of the grants would
have gone to directors' institutions, they would not have
approved creation of the stem cell agency.
As the California Stem Cell Report remarked earlier, it is a good
move for CIRM to retain more transparency rather than less. As one of
the Moss Adams staffers said today – in a different context –
during the presentation of the first-ever performance audit of CIRM,

"When people have to fill a void
in information, they assume the worst."

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

The California Stem Cell Agency and an HIV Cure: Pushing for a Clinical Trial in 2014


The California stem cell agency's
leading efforts to find a cure for HIV – one tied to the famous "Berlin Patient" – received a plug today in a piece in the
state capital's largest circulation newspaper, The Sacramento Bee.

The article by David Lesher focused on
a $14 million CIRM grant to the City of Hope in Los Angeles that also
involves Sangamo BioSciences of Richmond, Ca. The team hopes to
launch a clinical trial by the end of next year.
The Berlin Patient is Timothy Brown,
now of San Francisco, who is the only person in the world known to
have been cured of HIV/AIDs. It came about as a side effect of a
blood transfusion carrying a rare mutation of a gene found almost
entirely among northern Europeans. Lesher, director of governmental
affairs for the Public Policy Institute in Sacramento, wrote,

"The
possibility of curing a global pandemic like AIDS with funding from
the California bond is exactly the kind of exciting potential that
inspired voters to approve Proposition 71
 by
a wide margin. But the HIV research is also a good example of the
challenge facing the state's s
tem cell agency
as it tries to show voters that they made a good investment.
 

None
of the research under way will reach patients until long after the 10
years of funding by the ballot measure runs out. With the HIV
project, researchers hope to be in human trials by 2014, but it is
likely to be at least 10 years before they can show it might work in
humans. And in the case of a stem cell
 cure
for AIDS, it would be many years after that before a treatment is
widely available.”

Jeff
Sheehy
, a prominent AIDS activist and a board member at the 
stem
cell
 agency,
described the effort as "the global home run. That's not in 10
years. … But this could be the beginning of something really
amazing."
Lesher also wrote,

"Nobody
thought stem cells 
might
be used to cure HIV when the bond (funding for the stem cell agency)
passed. Far from the embryonic stem cell 
treatments
that inspired the ballot measure, the HIV research involves a new and
growing integration of stem cell 
and
genetic science."

Indeed,
the ballot initiative that created the $3 billion California stem
cell agency trumpeted its devotion to human embryonic stem cell
research, which had been throttled by the Bush Administration. The
agency has veered away from hESC research, which now amounts
to less than $450 million out of the $1.4 billion in grants approved
since 2004. 

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

UC Davis Researchers Score Big in $113 Million Stem Cell Award Round


Scientists at the University of
California at Davis
are set to win nearly half of $113 million
expected to be awarded next week by the California stem cell agency
as it pushes aggressively to turn research into marketplace cures.

Directors of the $3 billion agency are
virtually certain to approve awards to three researchers at UC Davis,
which operates its medical school and other research facilities in
nearby Sacramento. The other three expected winners are from UCLA,
Stanford
and StemCells, Inc., of Newark, Ca., a publicly traded firm.
The $113 million round is the second largest research round in CIRM's history, surpassed only by an
another, earlier $211 million “disease team” round. The latest
effort is aimed at bringing proposed clinical trials to the FDA for approval or possibly starting trials within four years. That deadline
is close to the time when CIRM is scheduled to run out of cash unless
new funding sources are developed.
CIRM is currently exploring seeking
private financing. It could also ask voters to approve another state
bond issue. (Bonds currently provide the only real source of cash for
CIRM.)  In either case, the agency needs strong, positive results from
its grantees to support a bid for continued funding.
The CIRM board is scheduled to approve
the latest awards one week from tomorrow at a public meeting in Burlingame in the San Francisco area. The agency's policy is to
withhold the identities of applicants and winners until after formal
board action. The California Stem Cell Report, however, has pieced
together their identities from public records.
Here are the winners and links to the
grant review summaries, listed in order of the CIRM scientific
scores:
  • Vicki Wheelock, UC Davis, $19 million,
    for development of a genetically modified cell therapy for
    Huntington's disease, an inherited neurodegenerative disorder.
    Scientific score 87.
  • Antoni Ribas, UCLA, $20 million, for
    genetic reprogramming of cells to fight cancer. Scientific score 84.
  • Nancy Lane, UC Davis, $20 million, for
    development of a small molecule to promote bone growth for the
    treatment of osteoporosis. Scientific score 80.
  • John Laird, UC Davis, $14.2 million,
    for development of mesenchymal stem cells genetically modified for
    treatment of critical limb ischemia, which restricts blood flow in
    the lower leg and can lead to amputation. Scientific score 79.
  • StemCells, Inc., (principal
    investigator not yet known), $20 million, for development of human
    neural stem cells to treat chronic cervical spinal cord injury. The
    company, founded by Stanford scientist Irv Weissman, who serves on
    its board, said earlier this year that it had filed two applications
    in this round, one of which dealt with cervical cord spinal injury.
    No other applicants filed a proposal for such research. Scientific score 79.
  • Robert Robbins, Stanford, $20 million,
    development of a human embryonic stem cell treatment for end-stage
    heart failure.
    Scientific score 68.

In the case of businesses, the awards
come in the form of loans. Grants go to nonprofits. One of the
reasons behind the varying mechanisms is the difference in CIRM's
intellectual property rules for businesses and nonprofits.

CIRM's Grant Working Group earlier this
year approved the applications during closed door sessions. The full
CIRM board has ultimate authority on the applications, but it has
almost never rejected a positive action by the grant reviewers.
The board originally allotted $243 million for this round. Directors could reach into the 15
applications rejected by reviewers and approve any of them, which the
board has done in other rounds. In this round, three rejected
applications scored within seven points of the lowest rated
application approved by reviewers, which could lead some directors
to argue that the scores are not significantly different. One of the
three came from Alexandra Capela of StemCells, Inc., and was scored at 61. The other two and their scores are Clive Svendsen of
Cedars-Sinai, score 64, for ALS research, and Roberta Brinton of
USC, score 63, for an Alzheimer's project.
Rejected applicants also can appeal
reviewer decisions to the full CIRM board in writing and in public
appearances before directors.
Twenty-three researchers were eligible
to apply for funding, CIRM told the California Stem Cell Report.
Applicants qualified by either winning a related planning grant from
CIRM last year or by being granted an exception to that requirement
by CIRM staff. Of the 22 researchers who ultimately applied(one
nonprofit dropped out), six came from biotech businesses. Three of
those qualified through exceptions. Three other businesses won
planning grants last year out of the eight businesses that applied.
CIRM has come under fire for its
negligible funding of stem cell firms and is moving to embrace
industry more warmly.
Only one of the grants approved by
reviewers involves research with human embryonic stem cells, which
was the critical key to creation of the California stem cell agency.
California voters established the agency in 2004 on the basis that it
was needed because the Bush Administration had restricted federal
funding of human embryonic stem cell research.  

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

CIRM Directors Pleased with Performance Audit Findings


The $3 billion California stem cell
agency received a "very favorable" performance audit report
compared to other government agencies, CIRM directors were told
today.

Representatives of Moss Adams, which
was paid $234,944 by CIRM for the study, made the comments during a
presentation today to the agency's 29 directors. During their
comments, CIRM executives and directors focused on the favorable
aspects of the findings of the six-month study.
CIRM Chairman J.T. Thomas said the
report showed that CIRM is "doing better than being on the right
track." Co-vice chairman Art Torres said,

 "Comparatively we
have done very well."

The report praised the professionalism
of the CIRM staff – "a high caliber group" – and noted
the seven-year-old agency is both "ramping up and ramping down"
at the same time – a reference to the end of state bond funding for
CIRM in 2017.
Prior to the presentation, CIRM
President Alan Trounson said the staff would review the findings and
come up with a plan for the board at its July meeting. The agency is
already implementing some of the recommendations.
The audit was required by a recent
state law that also allowed CIRM to hire more than 50 persons, a cap
imposed by Proposition 71, which created the agency. The audit found a need for improvement in 27 areas and made recommendations. Of the 20 recommendations with the highest priority,
half involved how CIRM manages its information, much of which is
needed for good decision-making. The audit did not assess the
scientific performance of the agency.
The Moss Adams report, performed by the
Seattle firm's San Francisco office, said,

"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. CIRM does not currently have a
formal performance reporting program."

In addition to decision-making
information, Moss Adams called for improvements in the agency's
long-troubled grants management system, better grant outcome
tracking, development of a results-based communications plan,
creation of a comprehensive, formal business development plan,
formulation of a comprehensive information technology plan that would
include steps to establish clear responsibility for CIRM's website
and improved monitoring of invention disclosure forms from grantee
institutions.
Last week, in a long overdue move, the
agency hired a director for information technology, who is expected
to solve many of the problems cited in the audit.
State law requires another performance audit in a few years. 

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

California Stem Cell Agency Launches Five-Year Push for Cures


The $3 billion California stem cell
today officially embarked on a course that will mean closer ties to
the biotech industry in hopes of fulfilling the campaign promises to voters to
turn stem cells into cures.

On a unanimous voice vote, directors approved
changes in the seven-year-old agency's strategic plan. The action
will likely mean less money for some activities that enjoyed more cash in the past,  but directors put off action until at least late July.  The plan also sets the course for what may be the last years of life
for the unprecedented state research program. Authorization to borrow
more money (state bonds) for its grants will run out in about 2017.

During a brief discussion of the plan, which has been debated for some months, CIRM Director Jeff Sheehy noted that the agency has now entered "the realm of trade-offs."  Ellen Feigal, CIRM's senior vice president for research and development, told the board that the plan will require hard decisions and sharp focus on priorities. 

Among other things, for first time CIRM overtly set a goal of creating 20 programs that include outside
investment that focus on products. Another five-year goal explicitly calls for financing at least 10 therapies in early-phase
clinical trials, affecting at least five diseases. Overall, the plan seeks to achieve clinical proof-of-concept for stem cell therapies.

In contrast to the Proposition 71
campaign rhetoric, CIRM's strategic plan acknowledges that developing therapies takes a very long time,
often decades.
Two scenarios were presented to the
board for spending the agency's remaining $836 million for grants and
loans. One would allocate $506 million for development research, $195
for translational research and $135 million for basic research, but
nothing for training and "facilities/core resources."
The other scenario calls for $486
million for development research, $160 million for translational
research, $105 for basic research, $60 million for training and $25
million for "facilities/core resources."

The first scenario would mean a $85 million cut in training and shared lab programs – cash that helps to finance researchers and that benefits the many institutions that have representation on the CIRM board.

The board put off action on either scenario after CIRM President Alan Trounson said he wanted more time to prepare a complete analysis of the scenarios. 

The plan also calls for creation of a
platform to enable grantees, disease foundations, venture capitalists
and others to purse CIRM's mission when its state bond funding runs
out. The possibility exists that another bond measure would be submitted to voters. But in either case, CIRM will need a solid record to attract support. 

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

CIRM Chairman on Geron: Agency in for the Long Haul


Commenting on Geron's decision to abandon hESC research, the chairman of the California stem cell agency, Jonathan Thomas, says the agency knew "the road to new therapies would be arduous, and that for each success, there would be setbacks as well."

In a memo Monday to the agency's 29 board members, Thomas reiterated that Geron maintains the decision had nothing to do with safety concerns. He said the company's action underscores the agency's commitment to long-term development of stem cell therapies.

Thomas also said the $3 billion agency will retain the stock warrants it received from Geron, but did not disclose their numbers. We have queried CIRM for more details. CIRM loaned Geron $25 million, which was paid back by Geron on Monday.

A copy of the Thomas memo was provided to the California Stem Cell Report. Here is the full text.

"Dear Board Members:

"Earlier this afternoon, we learned that Geron made a decision to discontinue its stem cell research program, including the CIRM-funded clinical trial involving spinal cord injury. Geron made this decision in order to shift its focus to its oncology program. Geron has assured us that its decision to discontinue the trial had nothing to do with safety concerns; the cells have been well-tolerated and the patients have experienced no adverse effects. Geron will continue to follow all enrolled patients and has committed to accrue data and update the FDA and the medical community regarding the patients’ progress.

"In addition, Geron has returned CIRM’s funds, with accrued interest. CIRM will maintain the warrants it received.

"Of course, we remain committed to funding clinical development of stem cell therapies. We have always recognized that the road to new therapies would be arduous, and that for each success, there would be setbacks as well. We also know that companies make decisions for business reasons, and that it is not unusual for a company to pursue a new strategy, as Geron has done. CIRM, by contrast, is focused on its long-term goal of delivering therapies and cure to patients, and today’s events underscore the importance of CIRM’s long-term commitment to funding therapy development.

"This trial represented the first-ever clinical trial of human embryonic stem cells and we expected that it would be challenging. We share the frustration of all the patients for whom this trial offered great hope. I spoke personally with both Don and Roman Reed to express my commitment to the on-going search for therapies.

"Although we are deeply disappointed by Geron’s decision, we are grateful that Geron has established a regulatory pathway for human embryonic stem cell therapies and we are confident in the potential of stem cells to treat patients suffering from chronic disease and injury.

"Here is a link to CIRM’s press release: http://www.cirm.ca.gov/PressRelease_2011-11-14"

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

Geron hESC Withdrawal 'Real Blow' to California Stem Cell Agency, Says CGS


In a sharp-edged analysis of Geron's abandonment of stem cell research, the Center for Genetics and Society yesterday described the action as a "real blow" to the California stem cell agency.

Written by Pete Shanks, a regular contributor to the Berkeley center's Biopolitical Times, the piece chronicled some of the history and hype involving Geron and hESC research. The center has long taken a skeptical view of the California stem cell agency and Geron. Shanks wrote,

"Geron has been in trouble for a while. Former CEO Thomas Okarma, who left abruptly in February, was the subject of ridicule for his repeated announcements that ESC-based clinical trials would begin "next year" — that is, 2005, 2006, 2007, 2008 — and the trial they eventually came up with was so dubious that Arthur Caplan called it "nuts and hugely risky." Even experts in the field thought that targeting spinal cord injury in the first ESC trial was dubious, though some seem to be more willing to be critical now it has ended.

"And that was on the scientific and perhaps commercial merits. The ethical problems were much worse, since the trial was intended for people who had recently suffered damage to their spinal cords. Bioethicist Laurie Zoloth (who was once on Geron's ethics advisory board, and basically approved of the study), noted at the time of its announcement that:

"'True informed consent in this very vulnerable population, people who have suffered a devastating and life-changing injury a week prior to being asked to enter the first clinical trial for such long-awaited, highly publicized and desperately needed treatment, is hard to obtain and will need to be carefully thought through.'"

Shanks also wrote,

"What of CIRM's role? After Geron's announcement, they issued a remarkably bland press statement, followed by an internal memo that expressed deep disappointment (the California Stem Cell Report has the text). It's a real blow to them: Geron was the first private company to receive funds from CIRM to run a clinical trial using ESCs.

"This was a loan, not a grant, and was only made, as the indefatigable David Jensen (publisher of the California Stem Cell Report) discovered, after a 'major departure from longstanding procedures.' The proposal received a low score (66/100) that was not publicly revealed until Jensen specifically asked for it. And the other applicants who might have competed for those funds rather surprisingly all withdrew.

"The suspicion arises that CIRM, or some people within it, badly wanted the trial to proceed in the hope that it would give them a therapeutic success to boast about. If so, the decision just backfired."

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

Geron Quits Stem Cell Research; Move Deemed Setback to the Field


In a surprise move, Geron today said it was terminating its stem cell business and what was the first-ever clinical trial of an hESC therapy, a potential product backed by a $25 million loan made by the California stem cell agency only six months ago.

Geron's move has "stark implications" for development of stem cell therapies in the United States, The Associated Press said. Andrew Pollack of the New York Times reported,

"The move is expected to be widely seen as a setback for the field, because of Geron’s central role."

The Menlo Park, Ca., company said it will fire 66 employees, 38 percent of its workforce, and will try to find buyers for its stem cell business.

The company's CEO, John Scarlett, cited the need to "focus our resources" given the "current environment of capital scarcity and uncertain economic conditions." The clinical trial of its spinal cord treatment will be closed to further enrollment, although it will continue to follow all four enrolled patients. The company said it now plans to focus on cancer drugs.

The company did not mention CIRM or the $25 million loan in its statement. The stem cell agency issued a press release that said,

"Geron had received $6.42 million of their loan, which the company today repaid in full with accrued interest."

In the news release, CIRM's Ellen Feigal, senior vice president for research and development, did not comment directly on Geron's move. She said CIRM remained optimistic about "many exciting stem cell programs in California." She said moving a therapy into the clinic is a "highly complicated process."

Absent from CIRM's press release were any comments from its new chairman, Jonathan Thomas, and CIRM President Alan Trounson.

Dropping stem cell research will improve Geron's cash situation. The New York Times' Pollack said,

"Geron will be able to last without needing to raise new money until it receives results of clinical trials of its cancer drugs over the next 18 months. By contrast, Dr. Scarlett said, given all the precautions in the stem cell field, he did not think there would be results from the stem cell trial until 2014."

The AP quoted stock analyst Steve Brozak of WBB Securities as saying Geron had encountered difficulty in finding partners for its stem cell program. He said potential partners wanted to see "later stage results." Of the clinical trial, Brozak said,

"It could be outsourced to a place like China very easily. In that case, this would be the de facto abdication of U.S. leadership in biotechnology."

With Geron's departure, Advanced Cell Technology of Santa Monica, Ca., is the only company with a clinical trial involving hESC, one targeting macular degeneration. ACT has never received an award from the California stem cell agency although it relocated its headquarters to California in the wake of the passage of Prop. 71, which created the state's $3 billion research program.

ACT was widely believed to have applied in CIRM's financing round last spring involving Geron.

CIRM directors approved the loan to Geron last May in a meeting that departed radically from its normal awards process.

As reported by the California Stem Cell Report in August,

"The Geron application was not given a public scientific score, standard practice for all the other 433 applications that the agency has approved over the last six years. The usual summary of grant reviewer comments was not provided to the public or the board. The three other applicants in the $50 million round all withdrew prior to presentation to the CIRM board – another first in CIRM's grant program. And no public explanation was provided at the time for the departures from long-established procedures." 

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

Geron Flight from hESC Research is 'Powerful Blow'


The news about Geron's abrupt departure from hESC research is rippling around the world this morning, casting a pall over the entire field.

The Financial Times of London, in a story by Andrew Jacksaid,

"Geron, the pioneering stem cell therapy company, has dealt a powerful blow to one of the most hyped areas of medicinal research by withdrawing entirely from the field."

Ron Leuty at the San Francisco Business Times said in an analysis,

"Just why would I want to invest in a space where one of the most promising companies just called it quits."

In a Washington Post article by Rob Stein, a patient advocate expressed bitterness.

"'I’m disgusted. It makes me sick,' said Daniel Heumann, who is on the board of the Christopher and Dana Reeve Foundation. 'To get people’s hopes up and then do this for financial reasons is despicable. They’re treating us like lab rats.'"

Geron, based in the San Francisco peninsula city of Menlo Park, cited financial problems when it announced yesterday that it was giving up on its stem cell efforts in favor of its cancer drug program. Geron is laying off 38 percent of its staff and says it will try to find a buyer for the stem cell program. Geron also settled its accounts with the California stem cell agency, which loaned it $25 million last May amidst considerable publicity.

The stem cell agency and other advocates expressed optimism about the long term potential of the field and noted the difficulties of bringing any therapy to production. Geron produced 21,000 pages of material over a years-long period to get FDA permission to begin only the first step in the long clinical trial process.

But the dominant tone of the news stories was negative. Heidi Ledford of Nature said Geron is "walking out on the field." Steve Johnson of the San Jose Mercury News said the decision casts "a cloud over the commercial viability of stem cell treatments."

Here are other excerpts:

San Francisco Business Times:

"What does this mean for companies that the California Institute for Regenerative Medicine, the state’s stem cell research funding agency, is pushing toward clinical trials? Will they only get so far before they, too, exit the business?" 

Fergus Walsh, medical correspondent for the BBC(see here), wrote:

"The decision does seem to be extraordinary given the huge investment of time and resources. When I visited Geron nearly three years ago, the then chief executive claimed the technology had an incredible future. 

"John Martin, professor of cardiovascular medicine at University College London said: 'The Geron trial had no real chance of success because of the design and the disease targeted. It was an intrinsically flawed study. And for that reasons we should not be describing this as a set back. 

"The first trials of stem cell that will give an answer are our own in the heart. The heart is an organ that can give quantitative data of quality.'"

Ryan Flinn of Business Week wrote,

"Geron fell 16 percent to $1.86 in extended trading. The shares have declined 58 percent this year.  

Robert Lanza, chief scientific officer of Advanced Cell Technology Inc.(of Santa Monica, Ca.), the second company to win permission from the U.S. Food and Drug Administration to test human embryonic stem cells in people, said the news wasn’t surprising, given the small patient population affected with spinal cord injuries. 

"'It was a very difficult choice to go in and treat spinal cord injury,' Lanza said in an interview. 'There was considerable concern in the scientific community that that might not have been the ideal first indication."

As first reported by the California Stem Cell Report, Geron's loan application was scored as a 66 on a scale 100 last May by scientific reviewers for the California stem cell agency. The score was not disclosed publicly at the time CIRM directors approved the award as has been the practice for all the other 400-plus awards that the agency has granted. The Geron approval process departed radically from the agency's regular procedures.

The news coverage in California of the Geron decision was marked by the absence of a story in the Los Angeles Times, the state's largest circulation newspaper. The San Francisco Chronicle carried only a brief story buried on page 6 of its business section. The California stem cell agency is based in San Francisco and the Bay Area contains one of the larger and more important biotech business and academic research communities in the world.

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