Author Archives: Fredricko

America Stem Cell, Inc. Awarded a Phase I STTR to Explore the Therapeutic Potential of Its Platform Technology (ASC …

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SAN ANTONIO--(BUSINESS WIRE)--

America Stem Cell, Inc. (ASC) today announced that it has been awarded an Advanced Technology Small Business Technology Transfer Research (STTR) grant from the National Heart Lung and Blood Institute at the National Institutes of Health. This grant will be conducted in collaboration with scientists at the Wake Forest Institute of Regenerative Medicine (WFIRM) in Winston-Salem, NC, and will explore the combination of two technologies: ASC-101 developed by America Stem Cell and amniotic fluid-derived stem cells discovered and pioneered by Dr. Shay Soker and colleagues at WFIRM. We will examine the effect of ASC-101-treated amniotic fluid-derived stem cells in an experimental model of compartment syndrome. Compartment syndrome results from a variety of injuries such as fractures, contusions, burns, trauma, post-ischemic swelling and blast injuries such as gunshot wounds. If not addressed quickly, it can lead to considerable loss of muscle tissue. Musculoskeletal disorders are the primary cause of disability in the United States with associated costs of more than $800 billion annually. In addition to civilian injuries, more than 42,000 soldiers have been injured since the beginning of the Iraq and Afghanistan wars: the majority of these injuries were musculoskeletal in nature.

America Stem Cell has demonstrated that ASC-101 enhances the ability of stem cells to migrate to their target tissue. While most companies are concerned with the type of cells used for cell therapy (i.e. the hardware), America Stem Cell addresses how to get the cells to go where they are needed most (i.e. the software). With this award, America Stem Cell will expand the potential for therapeutic application of ASC-101 with amniotic fluid-derived stem cells. According to Dr. Leonard Miller, the Co-Principal Investigator on the grant, The successful combination of ASC-101 with amniotic fluid-derived stem cells would be directly relevant to improving the treatment of muscle damage that occurs following compartment syndrome as well as multiple other types of injuries.

America Stem Cell, Inc. is a clinical stage company that is in clinical trials at the University of Texas M.D. Anderson Cancer Center for improving clinical outcomes for cancer patients undergoing hematopoietic stem cell transplantation. This award enables America Stem Cell to expand the development of ASC-101 to yet another cell type. Lynnet Koh, CEO of America Stem Cell, noted, The combination of ASC-101 with amniotic fluid-derived stem cells could synergistically enhance the therapeutic and regenerative capacity of these cells and most importantly provide an off-the-shelf, effective solution for tissue damage due to multiple types of injuries or diseases. ASC-101 is a transformative technology with the potential to improve clinical outcomes for patients undergoing a wide variety of cell therapies for the treatment of diseases such as graft versus host disease, diabetic complications, and ischemic diseases such as myocardial infarctions, retinopathy and critical limb ischemia. America Stem Cell has established a number of collaborations examining the potential of ASC-101 to improve cell therapies for multiple clinical conditions using a wide variety of cell types.

About America Stem Cell, Inc.

America Stem Cell is a privately held biotechnology company based in San Antonio, TX, with offices in San Diego, CA, and is dedicated to the development and commercialization of enabling technologies to enhance and expand the therapeutic potential of cell therapies. The key technology platforms (ASC-101 and ASC-102) are designed to improve the homing and engraftment of cells to target organs. ASC-101 is currently in clinical trials to improve the therapeutic potential of hematopoietic stem cells for patients in need of hematopoietic stem cell transplantation. Additionally, these technologies have the potential to enhance the efficacy of cell therapies for the treatment of inflammation from chemotherapy/radiation, autoimmune diseases, and ischemic diseases including myocardial infarction and stroke. America Stem Cell has partnerships and collaborations with Kyowa Hakko Kirin, Spectrum Medical Innvoations, Florida Biologix, and various medical research institutions including the University of Texas M.D. Anderson Cancer Center, Oklahoma Medical Research Foundation, Fred Hutchinson Cancer Center,,University of California San Diego, Sanford-Burnham Institute, Indiana University, Juvenile Diabetes Research Foundation, as well as corporate partnerships. For additional information, please contact Lynnet Koh at 210-410-6427, or view http://www.americastemcell.com.

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America Stem Cell, Inc. Awarded a Phase I STTR to Explore the Therapeutic Potential of Its Platform Technology (ASC ...

South Korean lawmakers one step from rigorous new system for stem cell Advancement: scientists, physicians and …

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SEOUL, South Korea, Sept. 19, 2012 /PRNewswire/ -- Seung-Jo Yang, a Parliament member, prepared a bill proposing new law for the management and transplantation of stem cells. On September 17, the National Health and Welfare Committee referred the bill to the Conference for review. The previous 18th National Assembly proposed a similar stem cell bill when much of its term had already passed so it was not fully discussed until the period ended, disappointing many patients with rare diseases. The Bill was referred to the 19th National Assembly, though, which shows the willingness of South Korean leaders to meet the expectations that this new law will be passed coming from the medical community and patient groups throughout South Korea.

Dr. Jeong-Chan Ra, president of RNL BIO's stem cell technology institute said "This effort for new stem cell bill will advance the use and sophistication of autologous adult stem cell technology as a powerful solution for overcoming incurable diseases." Dr. Ra, whose pioneering efforts in Korean stem cell research are known worldwide, is equally known in Korea as an ardent advocate for governmental investment in regenerative medicine. Perhaps no scientist has pushed harder for rigorous standards for stem cell banking, which this proposal may at last bring to fruition.

In South Korea stem cell banks have not been operated under a specific legal structure, so development and progress has been limited. The proposed law suggests that the harvesting and preservation of stem cells must be controlled by national regulation, specifically a management system for stem cell harvesting, storage and implantation. Through this, stem cell experts expect growth in responsible stem cell research and faster, even safer development of therapeutics.

Additionally, the current Korean policy requires stem cell programs - regardless of whether there are available therapies for patients with any particular condition - to complete clinical trial phase III for market approval. Had such rules been in place, for example, for the treatment of H.I.V., tens of thousands of people would have died. Many treatments for those with incurable diseases have been approved without completion of Phase III including stem cell treatments in other nations, even for the treatment of HIV, which stem cells have now cured. The bill under consideration proposes that physicians can use their own stem cells to treat conditions under their discretion if those stem cells are properly expanded, managed, handled and provided to clinicians for them at or above the proposed standards, which is the best news patients with incurable diseases for which no existing cure is available by current medicine have had in a long time.

When the bill is passed, high standards will be established and the better methods will immediately be made obvious to both government and patients. Through this the stem cell community expects a leap in industrial growth, and a leap in the ethical adherence of physicians to do no harm to patients and to provide remedies where possible for the aid of their patients.

The Korean medical community also expects not only to see an influx of domestic patients but also many patients from other nations that lack standards for the growth of patients' own stem cells. South Korea, many economists predict, could become the Mecca for stem cell therapeutics.

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South Korean lawmakers one step from rigorous new system for stem cell Advancement: scientists, physicians and ...

Destruction of Senescent Cells May Not Be Sufficient

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Senescent cells build up in our tissues with age. These cells have become damaged or passed the Hayflick limit and thus fallen out of the normal cell cycle of division. They should either self-destruct or be destroyed by the immune system, and until that happens they secrete all sorts of undesirable signaling compounds that tend to harm surrounding tissues. The more senescent cells you have, the more harm they cause - and the growth in their numbers with passing years is one of the root contributing causes of aging.

Given this outline, plans for dealing with the problem tend to involve identifying and destroying senescent cells - removing the cells from the picture is fairly clearly the way to go. The destroying part is pretty easy (there is no shortage of methods to kill cells) but the identification part is still a challenge, despite considerable progress from the cancer research community in building ways to target specific cell populations via aspects of their surface chemistry or other characteristics. At this point the state of the art demonstration of improved health in mice through destruction of senescent cells requires a combination approach of gene engineering and a targeted therapy, which isn't terribly practical as the basis for a human therapy.

Progress will be made nonetheless, and a near-future brace of therapies that remove the contribution of senescent cells to aging seems to be very plausible at this point. Yet this all assumes that senescent cells can be wiped out on an ongoing basis without consequence: a fair enough assumption for most tissues, made up of cells that are replaced and replenished on an ongoing basis. Recent research suggests, however, that cells that are far less readily replaced or are normally not replaced at all in the life span of an individual also turn senescent with age - such as those in the brain.

Postmitotic neurons develop a p21-dependent senescence-like phenotype driven by a DNA damage response:

In senescent cells, a DNA damage response drives not only irreversible loss of replicative capacity but also production and secretion of reactive oxygen species (ROS) and bioactive peptides including pro-inflammatory cytokines. This makes senescent cells a potential cause of tissue functional decline in aging.

To our knowledge, we show here for the first time evidence suggesting that DNA damage induces a senescence-like state in mature postmitotic neurons in vivo. About 40-80% of Purkinje neurons and 20-40% of cortical, hippocampal and peripheral neurons in the myenteric plexus from old [mice showed inceasing senescence-like characteristics] with age.

...

We conclude that a senescence-like phenotype is possibly not restricted to proliferation-competent cells. Rather, dysfunctional telomeres and/or accumulated DNA damage can induce a DNA damage response leading to a phenotype in postmitotic neurons that resembles cell senescence in multiple features. Senescence-like neurons might be a source of oxidative and inflammatory stress and a contributor to brain aging.

So if this research holds up we can't just rampage through the body and destroy everything that looks like a senescent cell. More discrimination is needed, which in turn means more complex therapies and a greater understanding of differences in biochemistry between the cell populations of interest. More to the point, we will also need some method of reversing this senescence-like state in the brain and nervous system cells that we want to keep around. Will a general repair of all of the known forms of cellular damage be sufficient for that? Is neural dysfunction absolutely a consequence of the damage modes described by the Strategies for Engineered Negligible Senescence? It seems unlikely that we'll get a solid answer to that question until SENS version 1.0 is implemented in mice, but the initial expectation would be that yes, it is.

And what about the mice that were treated with a method to destroy senescent cells? They didn't appear to have their brain function markedly impacted, or the researchers would have noted as much. However: (a) it was a study using mice engineered to age rapidly, and thus may not have lasted long enough to uncover issues of that sort, and (b) the method used to destroy senescent cells was very narrow and specific in its targeting, and may or may not have reached these neurons that fall into a senescence-like state.

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Vote for SENS Foundation at Chase Community Giving

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SENS Foundation manages a program of research, development, and advocacy for rejuvenation biotechnology - building the foundation for therapies that will reverse aging in the old by repairing the cellular and biochemical damage that causes it. At present the Chase Community Giving event at Facebook is winding to a close on the 19th of this month, with $10,000 grants provided to those charities given the most votes by the community. So if you have a Facebook account, take a few moments to head on over to the SENS Foundation page and add your vote. Similar past events have demonstrated that there are more than enough SENS supporters out there to win any charitable popularity measure like this; so vote before the 19th and pass it on to your friends.

Link: https://www.facebook.com/ChaseCommunityGiving/app_162065369655?cv=2&app_data=location|/charity/view/ein/94-3473864

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Spurring Regeneration of Axons in Spinal Injury

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Researchers continue to make progress in induced nerve regeneration: "researchers were able to regenerate 'an astonishing degree' of axonal growth at the site of severe spinal cord injury in rats. Their research revealed that early stage neurons have the ability to survive and extend axons to form new, functional neuronal relays across an injury site in the adult central nervous system (CNS). The study also proved that at least some types of adult CNS axons can overcome a normally inhibitory growth environment to grow over long distances. Importantly, stem cells across species exhibit these properties. ... The scientists embedded neural stem cells in a matrix of fibrin [mixed] with growth factors to form a gel. The gel was then applied to the injury site in rats with completely severed spinal cords. ... Using this method, after six weeks, the number of axons emerging from the injury site exceeded by 200-fold what had ever been seen before. The axons also grew 10 times the length of axons in any previous study and, importantly, the regeneration of these axons resulted in significant functional improvement. ... The grafting procedure resulted in significant functional improvement: On a 21-point walking scale, without treatment, the rats score was only 1.5; following the stem cell therapy, it rose to 7 - a score reflecting the animals' ability to move all joints of affected legs. Results were then replicated using two human stem cell lines, one already in human trials for ALS. ... We obtained the exact results using human cells as we had in the rat cells."

Link: http://www.eurekalert.org/pub_releases/2012-09/uoc--nsc091012.php

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Progress in LysoSENS: Bacterial Enzymes Deployed in Cell Culture to Break Down 7-Ketocholesterol

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LysoSENS is the oldest extant research program of the SENS Foundation, started back when the SENS program ran under the auspices of the Methuselah Foundation. In brief, LysoSENS is the development of a means of biomedical remediation. A whole range of harmful metabolic byproducts build up in human tissue with age, and we lack the means to break them down, or break them down fast enough. Some of these compounds simply cause harm, while others actually progressively impair the ability of cells to remove any unwanted chemicals, leading to what is known as the garbage catastrophe in aging - cells overwhelmed with broken protein machinery and waste products.

To do something about this issue we need ways to break down these waste products, such as those that make up lipofuscin, a mix of compounds that bloat and degrade the cellular recycling machinery known as lysosomes. Lipofuscin is implicated in a range of age-related diseases (as well as a class of genetic conditions known as lysosomal storage diseases). The LysoSENS project aims to discover bacterial enzymes capable of breaking down lipofuscin constituents and other important damaging compounds, and which can safely be introduced to human tissue. Researchers will then build a therapy to deliver these enzymes to where they are needed in our cells.

We have long known that such enzymes must exist, because places such as graveyards and battlefields do not exhibit a buildup of lipofuscin - something is eating it all. So the LysoSENS project started out by sifting through bacteria in soil samples, testing to see which of the bacterial species in the samples could consume harmful compounds such as 7-ketocholesterol, and then isolating the responsible enzymes.

This has been going on for a few years now, of course, and progress has been made - even at the all-too-low levels of funding available for this work. At this stage in the project a number of candidate enzymes that break down 7-ketocholesterol have been identified, and researchers are now putting them through their paces in cell cultures. One enzyme at least is worthy of a published paper.

Increased resistance to oxysterol cytotoxicity in fibroblasts transfected with a lysosomally targeted Chromobacterium oxidase

7-Ketocholesterol (7KC) is a cytotoxic oxysterol that plays a role in many age-related degenerative diseases. 7KC formation and accumulation often occurs in the lysosome, which hinders enzymatic transformations that reduce its toxicity and increase the sensitivity to lysosomal membrane permeabilization.

We assayed the potential to mitigate 7KC cytotoxicity and enhance cell viability by overexpressing 7KC-active enzymes in human fibroblasts. One of the enzymes tested, a cholesterol oxidase engineered for lysosomal targeting, significantly increased cell viability in the short term upon treatment with up to 50?µM 7KC relative to controls. These results suggest targeting the lysosome for optimal treatment of oxysterol-mediated cytotoxicity, and support the use of introducing novel catalytic function into the lysosome for therapeutic and research applications.

Some comments at the SENS Foundation:

The success of the approach employed by the team at Rice makes this enzyme, Chromobacterium sp. DS1 cholesterol oxidase, an important step toward a true rejuvenation biotechnology - a therapy that can target and repair damage that underlies the diseases and disabilities of the aging process. SENS Foundation is pleased to continue backing Dr. Mathieu's research, so that further work can move us closer to making such treatments a reality.

Given that many different harmful metabolic waste products exist, the field of biomedical remediation has enormous scope for growth - and certainly for more funding, which should hopefully start to arrive in the wake of proof of concept work like this. There is no need to slow down after finding one or more enzymes that break down 7-ketocholesterol, as firstly there could still be far better enzymes out there for this job, and secondly there remain numerous other damaging waste compounds in our cells and tissues that are worthy of biomedical remediation.

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Struggling With the Separation of Aging and Disease

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There is a school of thought that declares the average pace of degenerative aging as "normal" and states that any faster degenerations should be broken out and called "disease." This is somewhat manageable at the level of taxonomy, where you are only cataloging and describing the various ways in which bodily parts and systems break down, but as a system of thought it falls down badly once you have the ability to look under the hood to see what is going in our biochemistry. All of aging and age-related disease descend from the same collection of damage-causing processes, which like rust in a metal construction can lead to any number of different forms of ultimate structural failure - but all stemming from the same root causes. So trying to draw a dividing line between aging and disease produces issues and unnecessary additional work, especially if the researcher is trying to treat only "disease" but let "aging" progress, as you can see from the opening paragraphs in this paper: "Aging of the musculoskeletal system starts early and is detrimental to multiple functions of the whole organism, since it leads to disability and degenerative diseases. The age-related musculoskeletal changes are important in medical risk assessment and care because they influence the responses to treatment and outcomes of therapy. ... There are two major problems that one faces while trying to disentangle the biological complexity of the musculoskeletal aging: (a) it is a systemic, rather than 'compartmental,' problem, which should be dealt with accordingly, (b) the aging per se is neither well defined nor reliably measurable. A unique challenge of studying any age-related process is a need of distinguishing between the 'norm' and 'pathology,' which are interwoven in the aging. When another dimension is added, namely genetics underlying the system's functioning, even less is known about this aspect, and attempts to decipher genetic relationships between the system's components are few. ... To disentangle the aging-related pathology from the homeostasis particular for aging steady-state, is a challenging task. Despite the multiple definitions of the aging process were proposed, there is no single agreed upon and reliable measurement, therefore underlying molecular mechanism of aging is still not fully understood. The definition of aging is complicated by the occurrence of various diseases that modify body functions and tissue structures; these disease-related changes that are common in older persons are often hard to delineate from the aging process per se."

Link: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3429074/

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Two lessons I learned this week.

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I learned two valuable things this week I thought I'd pass on in a Friday afternoon post.  Actually strictly speaking these are likely things I've learned before but needed to re-learn or to be 'reminded' of their importance.
Please pardon a little stroll away from the typically strict focus on cell therapy -- but in a way that's the theme of today's post.
1.  Take time each week to read something from outside your specific profession or job focus.  
I'm not talking here about the importance of escaping in the evening with a fiction novel (also very important) but rather reading something professional but from well outside your area of focus.  Here are my examples.

I always read WIRED magazine.  Aside from GEN it's the only magazine I read.  Just reading something outside of cell therapy or biotech often infuses me with an idea that otherwise would have never occurred to me like the need for a cell therapy X Prize or cellular aggregates as microcarriers or tissue-engineered memory and processing devices or even just the conviction to better represent cell therapy to the broader world out there of scientists, engineers, journalists, policy-makers, or perhaps people with too much money looking to be inspired and wanting to make a difference.

Similarly, on a flight this week I reached into the seat pocket in front of me and discovered a recent copy of the Journal of the American Medical Association.  I read a fascinating article that has me excited about an idea for how we as a cell therapy industry might lead the way in addressing clinical trial and data transparency that would put our sector in a leadership position, lend the industry a much-needed spotlight, and has the potential to facilitate the kind of meta-analysis and data-mining that could only be done through data aggregation.  I believe the concept has the potential to be disproportionately significant for a sector defined by so many small, under-powered trials.
The idea may never see the light of day but the point is the source of the inspiration.  In order to 'think' outside the box one typically has to 'be' outside the box.  Lesson?  Spend some time outside your box.
2. It often takes something very small to make a disproportionately significant impact on someone.  
I was reminded recently through an exchange of simple kindnesses just how little it sometimes takes to make a big difference in someone's life.  For you what might be so easy to give might be of unparalleled value to someone for whom that is so unattainable.  
Lesson?  When the opportunity knocks for you to give something small or simple, take it.  This kind of charity almost always has the potential to be mre impactful than you might ever imagine.

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

Nonviral gene therapy targeting cardiovascular system

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The goal of gene therapy is either to introduce a therapeutic gene into or replace a defective gene in an individual's cells and tissues. Gene therapy has been urged as a potential method to induce therapeutic angiogenesis in ischemic myocardium and peripheral tissues after extensive investigation in recent preclinical and clinical studies. A successful gene therapy mainly relies on the development of the gene delivery vector. Developments in viral and nonviral vector technology including cell-based gene transfer will further improve transgene delivery and expression efficiency. Nonviral approaches as alternative gene delivery vehicles to viral vectors have received significant attention. Recently, a simple and safe approach of gene delivery into target cells using naked DNA has been impro...

MedWorm Sponsor Message: Please support the Doctors In Chains campaign for the medics tortured and sentenced for up to 15 years in Bahrain. #FreeDoctors

Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

StemCells, Inc., Discloses How it Will Generate $40 Million in Matching Funds

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StemCells, Inc., said yesterday that it
will come up with the $40 million needed to match loans from the California
stem cell agency through “existing infrastructure and overhead”
and will not be issuing stocks or warrants to the agency.

In a statement to shareholders, Martin
McGlynn, CEO of the publicly traded firm, discussed the $40 million in loans awarded by agency this summer, including $20 million last
week. The stem cell agency's governing board, after it emerged from
an executive session on the matter, last Wednesday night adopted a
motion requiring the company to demonstrate that it has the matching
funds.
CIRM Chairman J.T. Thomas, a Los Angeles bond financier, said that
concerns were expressed during the executive session that the agency
“would account for such a large part of the assets of the company.”
At his suggestion, the board approved the loan on the condition that
“it show it has access” to the $20 million in matching funds that
company offered during the application process. StemCells, Inc., also
offered a $20 million match on another loan approved in July by CIRM.
The latest financial reports from
StemCells, Inc., which is based in Newark, Ca., show that it had
assets of $17 million as of June 30 and liabilities of $11.6 million.
The company reported net income for the second quarter of $833,522
compared to a loss of $4 million for the same period a year ago.
In its filing with the SEC, the company
said,

“We have incurred significant
operating losses since inception. We expect to incur additional
operating losses over the foreseeable future. We have very limited
liquidity and capital resources and must obtain significant
additional capital and other resources in order to provide funding
for our product development efforts....”

In his statement yesterday, McGlynn
said the California stem cell agency had “doubled down” on
StemCells, Inc., in approving the two loans. He said the company is
not concerned about meeting the matching requirements. McGlynn said, 
Martin McGlynn
StemCells, Inc., Photo

“To be clear, we do not interpret the
diligence requirement as an obligation to raise a specific amount of
money in a particular period of time, and we wish to correct the
misstatements made by some uninformed third parties that the ICOC is
requiring us to raise $20 million in matching funds. In
point of fact, we expect that a substantial amount of our
contribution towards these projects will come from existing
infrastructure and overhead, salaries for our existing personnel, and
other contributions in kind. Furthermore, we will soon be
reviewing the budgets for both projects in detail with CIRM
staff. Because each disease team budget was prepared on a
stand-alone basis, we expect to see significant economies and
efficiencies now that the company has in fact been awarded funding
for both.”

McGlynn also said,

"Under this particular CIRM
program (RFA 10-05), funding for companies will be in the form of
unsecured, non-recourse, interest-bearing, term loans, which will be
forgivable in the event the funded research fails to result in a
commercialized product. On the other hand, should the product be
successfully commercialized, CIRM would earn milestone payments
depending on how successful the product becomes. Because CIRM
shares the downside risk, and could participate handsomely on the
upside, the structure makes the loan about as close to 'equity' as one could, without having to dilute existing shareholders in order
to gain access to significant amounts of capital.  The company
will not issue stock, warrants or other equity to CIRM in connection
with these awards. 

"Of course, we realize that CIRM
prefers that applicants from industry provide evidence of their
ability to secure whatever additional funds may be needed to complete
any CIRM-funded project, in this case the filing of an IND for each
indication. This is stated in the text of RFA 10-05 itself and
was repeated in various comments by CIRM staff during the application
process. When making the second award on September 5, the
ICOC naturally recognized the sizeable commitment it was making
to StemCells, so it instructed CIRM staff to satisfy themselves
of the company's ability to access the capital needed to fund the
project, namely the Alzheimer's program through to the filing of the
IND.”

McGlynn also said firm's bid for
another $10 million from CIRM could come in the form of a grant
instead of a loan. He said,

"Finally, I can confirm that in
June of this year the Company applied for up to $10
million under CIRM's Strategic Partnership I program
(RFA 12-05). Unlike the disease team awards under RFA
10-05, if companies are approved for funding under RFA 12-05, they
may elect to take such funding in the form of a grant, not a
loan. Our application under RFA 12-05 is for a controlled Phase
II clinical trial of HuCNS-SC cells in Pelizaeus-Merzbacher disease
(PMD), a rare myelination disorder. StemCells completed a Phase
I study in PMD in February 2012 and in April announced that
all of the patients from that study showed evidence of cell-derived
myelination and three of the four patients in the study showed
measurable gains in motor and/or cognitive function.”

According to CIRM, the awards in the strategic partner round will be approved either next month or in December. 
StemCells, Inc. stock was trading at
$1.85 at the time of this writing. Last week, it rose to $2.43.
During the last 12 months, its high was $2.67 and its low was 59
cents.

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

Road to Commercialization: California Stem Cell Agency Seeking Top Level Product Development Execs

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Looking for a good job at an
enterprise that is on the cutting edge of biotechnology?

Nine positions are open at the $3
billion California stem cell agency, headquartered in San Francisco.
Some of them could pay more than $200,000 a year.
Several of the new jobs are closely
aligned with the agency's fresh focus on commercializing stem cell
research and driving therapies into the clinic. Scientists and
lawyers are being recruited along with a business development
officer. For some of the positions, travel is required.
One new, high-level position is
described as a senior development officer. The job posting calls for
“expertise in product development for stem cell therapies.” The
person would “directly interact with investigators on CIRM’s
clinically applicable research programs to help provide product
development guidance from preclinical, manufacturing, and first in
human to early phase clinical regulatory perspectives.” An M.D. or
Ph.D. degree in a biomedical science is required. Pay tops out at
$232,891. This person would report to Ellen Feigal, senior vice
president for research and development.
A second, high-level position reporting to Feigal is senior medical officer, who would manage the
agency's portfolio aimed at commercialization of stem cell research,
specifically “focused on IND enabling and clinical development
projects.” This also requires an M.D. or Ph.D. and substantial professional experience in development of biomedical research and
products. Pay also could run as high $232.891 annually.
A third new job at CIRM is
business development officer. That person would help generate
“outside investment in stem cell research in California for both
CIRM-funded and not currently CIRM-funded programs by
biopharmaceutical strategic partners; equity investors (venture
capital and others); and disease foundations.” The salary range
hits $216,270 annually. It wouldn't be surprising if the person in
this job also became involved in developing a funding mechanism for
CIRM after it runs out of state cash in 2017 or so. 
This position reports to Elona Baum,
general counsel and vice president, business development.
And yet another new position is called
director of alliance management. The job deals with the agency's
extensive collaborative funding partnerships, many of which are
abroad. CIRM wants somebody with a law degree, experience in
intellectual property and business law along with strong negotiating
skills. The pay range for the post tops out at $232,891 annually. This position reports to CIRM President
Alan Trounson.
Other open positions include: deputy
general counsel, two science officers and office manager.

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

California Stem Cell Firsts: From Emotional Appeals to $40 Million Awards

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During the last few months, the $3 billion California stem cell
agency, which is approaching its eight-year anniversary, has chalked up a
number of important firsts.

Most of them came during the July and
September meetings of its 29-member governing board and were related to strenuous efforts by researchers to win approval of awards of up to
$20 million each. Several firsts involved the agency's former
chairman, Robert Klein, who could be considered the father of the
state's stem cell research effort.
So here is the California Stem Cell
Report's list of firsts at the California Institute of Regenerative
Medicine (as CIRM, the stem cell agency, is formally known) for the
summer of 2012.
It was the first time that a single
company – in this case, StemCells, Inc. , of Newark, Ca. –
received two awards in the same round.
It was the first time any company has
been awarded as much as $40 million. Again, StemCells, Inc.
It was the first time that Klein has lobbied his former board (see here and here) on
behalf of a particular grant application. That occurred in both July
and September with one of StemCells, Inc.'s application.
It was the first time that the board
has approved an application that has been rejected twice by
reviewers, again the StemCells, Inc., proposal backed by Klein.
It was the first time that board has
received such a large outpouring of appeals by rejected applicants.
It was the first time that the board
has received such lengthy presentations of emotional appeals by
patient advocates on behalf of rejected applicants.
It was the first time that action on a
grant round has been extended over three months(see here and here). The disease team
round began in July. Action will not be completed until the end of
October.
It was the first time that the
governing board has sent so many applications back for re-review –
five, six if the one to be acted on in October is included.
It was also the first time that the
board has ordered a full-blown review of its grant appeal process
with an eye to making making major changes in it.
Several reasons exist for the number of
firsts racked up by CIRM. One is the high stakes involved in the
disease team round that began in July and the low number approved by reviewers – six compared to the 12 approved by the board, as of
today, out of 21 applications. Another reason involves the
increasing understanding on the part of many scientists that they can
appeal directly to the board when reviewers reject their
applications. However, it is also clear that not all applicants
grasp the full range of appeal possibilities. A third reason involves
the agency's muddled appeal process, which has been a problem for
years. And a fourth reason involves the board's push to drive research into
the clinic and commercialization, which applicants are quickly
learning how to exploit.
Readers should feel free to add their
own firsts to this list. They can do so – even, anonymously – by
clicking on the word “comments” at the end of this item.

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Projected Growth in $3 Billion Parkinson’s Treatment Market Gives RBCC a Bright Outlook

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NOKOMIS, Fla.--(BUSINESS WIRE)--

The market for effective new treatments for Parkinsons disease and other degenerative neurological disorders is strong and projected to grow sharply in coming years. Thats good news for Rainbow Coral Corp.s (RBCC) biotech subsidiary, Rainbow Biosciences, as it closes in on a deal with Amarantus Biosciences, Inc. (AMBS)

The increasing market potential for Parkinsons therapies is what initially attracted RBCCs attention to Amarantus work. The Parkinson's Disease Foundation estimates that as many as one million Americans suffer from Parkinson's disease, and 60,000 new patients are diagnosed each year. Medication costs per person are believed to be around $2,500 each year and the total economic impact is estimated to be around $25 billion in the U.S. alone.

Most encouraging of all, the market for Parkinsons drugs could grow to a value of $3.75 billion by 2015, according to a report last year by Visiongain.

Growth in the Parkinson's treatment market is being driven by new breakthroughs, and RBCC is working to help advance the timeline for a cure for the debilitating disease. RBCC is currently negotiating a potential definitive agreement with Amarantus, a company that could be on the verge of promising new diagnostic and therapeutic tools for Parkinsons patients and their doctors.

Amarantus owns the rights to a promising therapeutic protein known as MANF that prevents a type of cell death called apoptosis that could be the beginning of a cure for the disease. The company also owns the license to a groundbreaking diagnostic platform called NuroPro for Parkinsons that allows neurologists to accurately diagnose and track the progression of Parkinsons disease in patients. This groundbreaking test could potentially be on market in certain regions as early as 2013.

For more information on RBCCs biotechnology initiatives, please visit http://www.rainbowbiosciences.com/investors.html.

About Rainbow BioSciences

Rainbow BioSciences, LLC, is a wholly owned subsidiary of Rainbow Coral Corp. (OTCBB:RBCC). The company continually seeks out new partnerships with biotechnology developers to deliver profitable new medical technologies and innovations. For more information on our growth-oriented business initiatives, please visit our website at [www.RainbowBioSciences.com]. For investment information and performance data on the company, please visit http://www.RainbowBioSciences.com/investors.html.

Notice Regarding Forward-Looking Statements

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Moving Day to benefit National Parkinson Foundation held Sunday at MCC

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University of Rochester neurologists as well as families touched by Parkinsons disease will be hitting the pavement this weekend to raise awareness and funds to help fight the disease.

The local Moving Day celebration, which includes a walk and other activities aimed at Parkinsons disease, is this Sunday, Sept. 16, on the grounds of Monroe Community College. While the actual walk begins at 10:30 a.m. and ends at noon, registration opens at 8:30 a.m. In addition to the walk, several events revolving around movement a key difficulty for patients with the disease will be held throughout the morning, including dance, yoga, Tai Chi, vocal and other physical exercises.

Moving Day is sponsored by the National Parkinson Foundation, which works to improve the quality of life for people with the disease through research, education and outreach. The goal of the Rochester Chapter of NPF this year is $65,000; so far the group has raised $26,000, already more than the total achieved last year. Funds will go to support the National Parkinson Foundation and its Rochester chapter, as well as Parkinsons research at the Medical Center.

Vicki Aspridy, registrar at the Simon School, is a member of the local chapter and part of the planning committee for the event. She is helping to organize Moving Day in honor of her mother, who had the disease late in life and passed away four years ago. A University team is being organized by neurologist Michelle Burack, M.D., Ph.D., who treats patients with the disease and also does research aimed at understanding and reducing the symptoms that patients experience.

For more information, visit http://MovingDayRochester.org.

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Moving Day to benefit National Parkinson Foundation held Sunday at MCC

Researchers battle Parkinson’s disease using mouse model

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Parkinsons disease may prove to be no match for a team of UH researchers at the Center of Nuclear Receptors and Cell Signaling.

CNRCS Director Jan-ke Gustafsson and professor Margaret Warner, along with their team of researchers, have found a link between beta-sitosterol present in many plants and good for preventing cholesterol absorption from ones diet and ALS-Parkinsons disease. This discovery may aid in the fight against Parkinsons and was recently published in Proceedings of the National Academy of Sciences.

Parkinsons disease is a chronic progressive neurological disease linked to a decrease in dopamine, production in the substantia nigra and marked by tremors in resting muscles, rigidity, slowness of movement, impaired balance and a shuffling gait.

If ALS-Parkinsons patients do have a defect in LXRbeta signaling, they will benefit from pharmaceuticals which target signaling of this receptor. Many such drugs are being developed at present, Warner said.

Authors Yubing Dai and Wanfu Wu researching in the CNRCS lab. | Amanda Hilow/The Daily Cougar

Gustafsson has had a long-standing interest in nuclear receptors because they are activated by the small molecules like hormones, and medication can be developed to increase or decrease their activity. In 1995, Gustafssons lab discovered two novel nuclear receptors one was LXRbeta. An efficient way to unmask the function of the newly discovered genes is to use gene technology to eliminate the hormone in mice and see what happens.

The receptor continues to show promise as a potential therapeutic target for this disease, as well as other neurological disorders, Gustafsson said in a press release for HealthNewsDigest.com. LXRbeta performs an important function in the development of the central nervous system, and our work indicates that the presence of LXRbeta promotes the survival of dopaminergic neurons, which are the main source of dopamine in the central nervous system.

During World War II, ALS-Parkinsons disease was prevalent in the Pacific Islands and Guam where there was a shortage of wheat. During that time, people had to make bread from cycad seeds, which contain a high level of beta-sitosterol.

The reason for the susceptibility of certain people in Guam to toxicity from beta-sitosterol remained a mystery until we developed the LXRbeta knockout mice, Warner said. These mice develop ALS-Parkinsons disease spontaneously as they age, and the disease is made worse if beta-sitosterol is added to their diet.

However, it was discovered that LXRbeta knockout mice did not need to be fed beta-sitosterol to develop Parkinsons disease. Meaning UH researchers have to look for the possibility that people develop ALS- Parkinsons disease because of defective LXRbeta signaling.

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Pittsburgh-area couple awarded for work with Parkinson's disease patients

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Tom and Carol Reid, of Plum, have been living with Parkinsons disease for 17 years, but its how they are helping others with the disorder that has earned them a big honor.

They will be awarded with the Local Hero award Saturday from the Davis Phinney Foundation.

Carol Reid remembers when she started noticing a change in her husband.

He was losing facial expression. I didn't know what that meant. I just felt like you know, you're not responding to me, she said. He was drooling. His eyes were watering. He was falling often.

Tom Reid went to the family doctor and was given Claritin for his watery eyes.

It took two years to get a second opinion and a diagnosis of Parkinsons, a neurodegenerative disease.

I was in a classical case of denial, said Tom.

Symptoms involve loss of motor control, affecting speech and movement.

Tom was a captain in the Army before becoming a corporate attorney. He enjoyed public speaking and using what he and his wife called his command voice.

Parkinsons has changed that.

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Pittsburgh-area couple awarded for work with Parkinson's disease patients

Pittsburgh-area couple awarded for work with Parkinson’s disease patients

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Tom and Carol Reid, of Plum, have been living with Parkinsons disease for 17 years, but its how they are helping others with the disorder that has earned them a big honor.

They will be awarded with the Local Hero award Saturday from the Davis Phinney Foundation.

Carol Reid remembers when she started noticing a change in her husband.

He was losing facial expression. I didn't know what that meant. I just felt like you know, you're not responding to me, she said. He was drooling. His eyes were watering. He was falling often.

Tom Reid went to the family doctor and was given Claritin for his watery eyes.

It took two years to get a second opinion and a diagnosis of Parkinsons, a neurodegenerative disease.

I was in a classical case of denial, said Tom.

Symptoms involve loss of motor control, affecting speech and movement.

Tom was a captain in the Army before becoming a corporate attorney. He enjoyed public speaking and using what he and his wife called his command voice.

Parkinsons has changed that.

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Pittsburgh-area couple awarded for work with Parkinson's disease patients

Brain Games Help Parkinson’s Patients

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PARKINSONS & MEMORY: In addition to the motor symptoms in Parkinson's disease, there are also cognitive symptoms which may be evident even in the early stages of the disease. These may include deficits in executive function (especially planning and attention), set-shifting (ability to alternate between two or more tasks), and memory. Approximately 25%-30% of Parkinson's patients develop dementia. It is not yet known whether dementia is actually a symptom of Parkinson's disease or whether patients with Parkinson's disease are for some reason also at higher risk for dementia. A large number of Parkinson's patients also experience psychiatric disorders such as depression, anxiety, or sleep disorders.

Because Parkinson's disease damages neurons in the substantia nigra which produce dopamine, treatment usually involves drugs which work to counteract this shortage of dopamine. Parkinson's patients may benefit from treatment with several kinds of drugs simultaneously. These drugs can often combat the motor symptoms for a long time, but as the disease progresses and the substantia nigra continues to degrade, the drugs eventually become less effective.

Some patients whose motor symptoms cannot be controlled by medication undergo brain surgery to destroy portions of the brain regions responsible for some of the motor symptoms in Parkinson's disease, or benefit from deep brain stimulation (DBS) -- using thin wires to stimulate electrical activity in the brain. There has also been controversial research involving implantation of cells from aborted fetuses into the brains of Parkinson's patients in an attempt to regrow neurons in the substantia nigra; more recently, scientists are exploring the possibility of using stem cells isolated from healthy adults or grown in the laboratory. As yet, this work is still highly experimental. (Source: Memorylossonline.com)

UNIVERSITY OF MARLYAND STUDY: Researchers are looking to study the benefits of exercise for fitness, walking, balance, and memory. They are still enrolling participants, and those eligible for the study are people who have been diagnosed with Parkinsons disease and have mild to moderate gait or balance difficulty. The study is being conducted at the Veterans Affairs Medical Center at the University of Maryland School of Medicine in Baltimore, Maryland.

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Brain Games Help Parkinson’s Patients

Multiple Sclerosis Pill Approved by U.S. FDA

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The U.S. FDA this week approved a once-a-day pill for the treatment of multiple sclerosis (MS). The pill, being branded as Aubagio by Sanofi S.A., is specifically for the treatment of adults with relapsing forms of MS.

In a clinical trial, the relapse rate for patients using Aubagio was about 30 percent lower than the rate for those taking a placebo, said Dr. Russell Katz, director of the division of neurology products in the FDAs Center for Drug Evaluation and Research. Multiple sclerosis can impair movement, sensation, and thinking, so it is important to have a variety of treatment options available to patients.

According to the National Institutes of Health, MS is a chronic autoimmune disease that affects the central nervous system. It disrupts the communication between the brain and the body, causing motor skill disruption for nearly every part of the body, depending on which nerves in the brain are damaged.

As for the drug itself, side effects seen during drug trials included diarrhea, abnormal liver tests, nausea, and hair loss. Also, the box warnings for the drug warn of possible liver problems and fetal harm, including the risk of birth defects. Doctors will have to check patients liver function and give a pregnancy test before starting treatment with Aubagio.

Many people living with MS struggle with the additional burden of injectable therapies administered daily to weekly, said Dr. Aaron Miller, medical director at the center for multiple sclerosis at Mount Sinai Medical Center. The FDAs approval of Aubagio, a new oral treatment option, is an encouraging advancement for the MS community and may be a valuable treatment for people living with this often debilitating disease.

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Sanofi multiple sclerosis pill gets US approval

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Sanofi SA has won U.S. approval for its multiple sclerosis pill Aubagio - one of the two treatments for the chronic disease that could return the French drugmaker to growth after several blockbuster drugs lost patent protection.

The drug has been shown to be less effective than some rivals but has milder side effects and analysts say it could find favor among newly diagnosed patients. Around 35 percent to 40 percent of multiple sclerosis (MS) sufferers prefer to take no medication rather than face unwanted side effects.

"In a clinical trial, the relapse rate for patients using Aubagio was about 30 percent lower than the rate for those taking a placebo," Russell Katz, director of the Division of Neurology Products at the Food and Drug Administration, said in a statement on Wednesday.

Aubagio is expected to launch on the U.S. market in a few weeks, a spokeswoman for Sanofi unit Genzyme said.

Multiple sclerosis, which has no cure, affects 2.5 million people worldwide. It is a chronic, often disabling disease that attacks the central nervous system and can lead to numbness, paralysis and loss of vision.

MS drugs Gilenya by Novartis and Biogen Idec Inc's BG-12 are expected to dominate a market that JPMorgan analysts predict growing to $14 billion in 2015 from $9.6 billion last year.

Aubagio is seen grabbing a much smaller chunk of this market, reaching modest sales of $353 million in the United States and five major European countries by 2020, according to business intelligence firm Datamonitor.

Cheuvreux analyst Marcel Brand, who has a more optimistic forecast, predicts peak sales of Aubagio of 1.48 billion euros by 2018. "Although Aubagio is not as effective on relapse rates as Gilenya, it's free of its longer-term side effects," he said.

Patients taking Gilenya have to be monitored because the drug causes the heart rate to slow down in the first hours after ingestion.

European regulators are expected to give their response to Aubagio in the first quarter of 2013.

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Sanofi multiple sclerosis pill gets US approval