Thursday, March 28, 2013

Patrick Cox Features ISCO's Stem Cell Parkinson's Study

Patrick Cox blogs about ISCO's success in primate study (data presented at American Academy of Neurology Annual Meeting, March 20th 2013).  Reproduced with the permission of P.Cox.







ISCO Shows Functioning Parthenogenic Stem Cells in Parkinson's Chimp Study

International Stem Cell Corp. (OTCBB: ISCO) has announced the most important experimental results in the history of the company. Last week, the company confirmed that these nonembryonic stem cells can, in fact, become part of a host primate and function as normal cells.

Now I'm really feeling guilty because this story deserves a lot more attention than I can give it today. Nevertheless, ISCO presented the results of a blinded chimpanzee Parkinson's study at the American Academy of Neurology's 65th annual meeting on March 20.

There's been a lot of skepticism about the potential of human parthenogenic stem cells (hpSCs) for several reasons. First of all, they don't develop naturally into adult cells such as the neuronal cells that were used in this study. Unlike regular cells that have the DNA of both the mother and the father, hpSC DNA consists of two copies of the mother's genome, so they have to be provoked to become specific cell types.

As a result, these hpSCs, which come from immature ova gathered during routine in vitro fertilization procedures, cannot develop into fetuses. Obviously, this is a big plus for those who oppose the use of embryonic stem cells. It raised the possibility, however, that cells derived from hpSCs would not function as normal cells for therapeutic purposes.

We now know that hpSC-derived neuronal cells will engraft, became part of the chimps they were given to and produce the dopamine needed to prevent the symptoms of Parkinson's disease. This is powerful evidence of hpSC efficacy, though I'm not at all surprised by these results.

You should read the press release, which states:
"‘This pilot study represents a first essential step in bringing cell-based therapies for Parkinson's disease to clinical trials,’ commented co-author of the study Evan Y. Snyder, MD, Ph.D., director of Stem Cells and Regenerative Biology Program at Sanford Burnham Medical Research Institute.

“These placebo-controlled studies were designed to demonstrate the viability, fate and functional efficacy of the stem cell-derived neural cells after implantation to the brain. Highly pure populations of neuronal cells were differentiated from human parthenogenetic stem cells (hpSC) according to the protocol developed by International Stem Cell Corp. and recently published in the Nature Publishing Group's Scientific Reports.

“The studies employ MPTP-lesioned African green monkeys and 6-OHDA-lesioned rats, the principal models used to study Parkinson's disease. The duration of the primate study was four months and the rodent study six months. In the nonhuman primate model, behavioral endpoints were assessed with Parkinsonian scores. These scores, based on a standardized rating scale, were recorded by observers blinded to whether the primates were in the treatment or control group. Observations were done twice per day, five days per week. In the rodent model, behavioral improvement was assessed using the cylinder test, amphetamine and apomorphine-induced rotation tests. Cell engraftment, viability and phenotype of the implanted cells were determined histologically at the end of the studies. Tumorigenicity and safety of the therapy was assessed at the end of both the rodent and primate studies by gross necropsy, and brain histology.

“The primate study consisted of eight asymptomatic monkeys which have the pathology of the disease, low levels of dopamine induced by bilateral injections of the neurotoxin MPTP and lack clinical symptoms. Four of the monkeys were transplanted with hpSC-derived neuronal cells, two monkeys sham transplanted with an equivalent volume of cell-less media thus serving as the placebo control group and two healthy monkeys serving as a positive control. Behavioral endpoints were recorded to evaluate possible adverse effects.

“Subsequent to implantation of the neuronal cells, all monkeys in the treatment group had higher levels of dopamine in the brain compared with the control group. Additionally, the rats in the treatment group showed gradual improvements in motor symptoms consistent with cells survival, engraftment and dopamine release. No adverse events, including dyskinesia, deformations, tumors or overgrowth, were observed in the rat or monkey treatment groups. Overall, these results provide evidence to support the hypothesis that hpSC-derived neuronal cells can be safe and have a disease modifying effect. These results, although preliminary, are a strong indication that our approach to Parkinson's disease can succeed.

"‘These results are pivotal for our preclinical Parkinson's program showing, as they do, that the hpSC-derived neuronal cells can potentially ameliorate the behavioral symptoms without triggering dyskinesias. This data form the foundation of our discussions with the FDA as we move toward our IND in 2013,’ said Dr. Ruslan Semechkin, principal investigator of this study, head of R&D for International Stem Cell Corp. and Member of the American Academy of Neurology.

“These results will be presented and discussed at the 65th American Academy of Neurology annual meeting, one of the world's most important annual events for neurologists and neuroscience professionals and the largest such international meeting of its kind, with more than 12,000 attendees at last year's meeting.”

Obviously, the company intends to bolster this study with larger primate studies before going into human studies, hopefully early next year. The importance of hpSC therapies, however, shouldn't be overlooked. Not only do they provide a solution to many people's ethical problems with embryonically derived stem cells, they represent an entirely different path to regenerative medicine.

As you know, BioTime (AMEX: BTX) recently consolidated Geron's stem cell IP. BioTime is now the undisputed leader in embryonic and induced pluripotent stem cells. ISCO, however, owns the parthenogenic space. It uses methods of differentiation and cell production that are unique to its own platform. In fact, far more information is available about those technologies in this journal article of Nature's Scientific Reports.

Ultimately, when the company has created its bank of cell lines to match most of the human species, it will be able to provide off-the-shelf therapeutic cells for many conditions that would otherwise require immune suppression or iPS cells. This is a big deal, but it's also in the future. The Parkinson's disease target is ideal for ISCO because there is no immune rejection behind the blood-brain barrier where these cells are required. This enables a path to revenues much sooner than the cell line strategy.

I'll have a lot more on this development in the future. Already, these results have generated more attention in the scientific press than the company has ever before garnered. That will increase as more information is released.

Also, by the way, the company's hpSC cosmetics line, Lifeline Skin Care, is getting serious attention in the beauty and anti-aging press. Products have recently been featured in Elle and Prevention magazines as well as other forums.

Yours for transformational profits,

Patrick Cox

International Stem Cell features in Breakthrough Technology Alerts


Once again Patrick Cos writes about ISCO's latest breakthrough in Parkinson's diease therapy.  The full post is reproduced below with permission from P. Cox.

Breakthrough Technology Alert
 March 19, 2013

ISCO Publishes Evidence Regarding Parthenogenetic Parkinson's Therapy

Dear Breakthrough Technology Alert Reader,

This will be short. My time this month is filled with appointments for my cataract surgeries, all over an hour north from this halcyon island that I call home. To complicate things further, my son's vehicle was totaled a few days ago and I've got to get him another SUV as soon as possible.

Yes, I said SUV. As I've said here before, I believe steel is the best insurance policy. The incident that sent my son's transportation to the wrecking yard eloquently demonstrates this.

He was stopped, waiting second in line at a red light in a major intersection. A drunk driver, probably passed out, hit him at high speed from behind. Fortunately, the weight and strength of the SUV was such that my son and his passenger were uninjured. The drunk driver's vehicle careened off my son's and hit the next car in line. As it was much lighter, it was pushed across the intersection and into opposing traffic.

Fortunately, other drivers avoided the mess, but the lesson is clear. Ceteris paribus, bigger and heavier is better. Yes, smaller cars with modern safety technologies are safer than older smaller cars, but a big heavy vehicle with those same safety innovations is best of all. It is irrational to buy expensive insurance, which is paid out after the harm done by an accident, while driving a smaller car. Paying less for fuel also increases the risk of greater injury in accidents, which is a bad trade-off in my book.

Coincidentally, the password for the leaked Climategate email file has been sent to a select group of scientists, giving them access to the behind-the-scenes conversations among the world's top advocates of the man-made global warming theory. I bring this up only because this group of global warming activists and their supporters have targeted SUVs for extinction.

I'm open to persuasive evidence that CO2 needs to be controlled, but it hasn't yet been produced. Persuasive evidence is needed because, as the Climategate leaker says, policies that would cut CO2 production would have a calamitous impact on the poor.

ISCO Publishes Major Article on Parthenogenetic Stem Cell Science

It's great to see International Stem Cell Corp. (OTCBB: ISCO) moving ahead. Their technology is of enormous importance and potential, so this evidence that parthenogenetic stem cells can be used to counter Parkinson's is a major breakthrough.

This is a fascinating branch of science because, as you know, these cells do not occur naturally. They are created using oocytes, immature ova gathered during routine fertility procedures. ISCO is clearly taking a different path in the creation of therapeutic cells. From the press release:
“The paper describes the technology, developed by ISCO's R&D team, for producing highly pure populations of GMP-grade neuronal cells suitable for preclinical studies and clinical trials. The neuronal cells created using this approach are further characterized using a number of analytical methods and shown to function in a similar manner to adult cells. Moreover, neuronal cells produced using this new method express greater levels of dopamine, the neurotransmitter central to PD, than previously reported approaches. Furthermore, these hpSC-derived neuronal cells are cryopreservable and can be stored frozen, and provide a practical route to creating neurons of sufficient quality to be used to treat Parkinson's disease patients.

"'It is important that our research is reviewed and validated by the scientific community, and being able to publish in a Nature-branded journal provides confirmation of the quality of ISCO's scientific research,' commented Dr. Andrey Semechkin, CEO and co-chairman.”

Read the press release here. The journal paper is here.

Early this morning, ISCO announced positive results for their primate Parkinson’s study. This is huge. I will have more for you in a future alert. In the meantime, you can find the press release here.

MIPS Technologies (Inc.) Complete

Well, MIPS is now part of Imagination Technologies Group plc (LSE: IMG). Shares of MIPS will receive $6.21 in cash and 0.226276 shares of MIPS' common stock, a consideration worth $1.80, totaling $7.31. Our acquisition cost was $6.42, so this is a decent, but somewhat disappointing return.

Yours for transformational profits,

Patrick Cox

Wednesday, March 20, 2013

ISCO Featured in San Diego Union Tribune


The San Diego Union Tribune wrote a nice piece about ISCO discussing our advances with creating neuronal stem cells and how we can use them to treat Parkinson's disease. The article appeared today and can be viewed using the link below.

http://www.utsandiego.com/news/2013/mar/19/international-stem-cell-parkinsons-study/

International Stem Cell Corporation Announces Positive Stem Cell Data in Parkinson’s Disease

Data Presented at the American Academy of Neurology 65th Annual Meeting 

CARLSBAD, Calif. – (Marketwire) -- March 20, 2013 -- International Stem Cell Corporation (ISCC) (OTCQB: ISCO) (www.internationalstemcell.com) a California-based biotechnology company focused on the therapeutic applications of human parthenogenetic stem cells today announced positive results demonstrating the safety and efficacy of stem cell engraftment in a primate model of Parkinson’s disease. The results were presented during the American Academy of Neurology (AAN) 65th Annual Meeting, Scientific Platform Session: Parkinson’s Disease Therapeutics on Wednesday, March 20, 2013 in San Diego.

"This pilot study represents a first essential step in bringing cell-based therapies for Parkinson's disease to clinical trials," commented co-author of the study Evan Y. Snyder, MD, PhD, Director of Stem Cells and Regenerative Biology Program at Sanford Burnham Medical Research Institute.

These placebo-controlled studies were designed to demonstrate the viability, fate and functional efficacy of the stem cell derived neural cells after implantation to the brain. Highly pure populations of neuronal cells were differentiated from human parthenogenetic stem cells (hpSC) according to the protocol developed by International Stem Cell Corporation and recently published in the Nature Publishing Group’s Scientific Reports.

The studies employ MPTP-lesioned African Green monkeys and 6-OHDA-lesioned rats, the principle models used to study Parkinson's disease. The duration of the primate study was four months and the rodent study six months. In the non-human primate model, behavioral endpoints were assessed with parkinsonian scores. These scores, based on a standardized rating scale, were recorded by observers blinded to whether the primates were in the treatment or control group. Observations were done twice per day, five days per week. In the rodent model, behavioral improvement was assessed using the cylinder test, amphetamine and apomorphine induced rotation tests. Cell engraftment, viability and phenotype of the implanted cells were determined histologically at the end of the studies. Tumorigenicity and safety of the therapy was assessed at the end of both the rodent and primate studies by gross necropsy, and brain histology.

The primate study consisted of eight asymptomatic monkeys which have the pathology of the disease, low levels of dopamine induced by bilateral injections of the neurotoxin MPTP, and lack clinical symptoms. Four of the monkeys were transplanted with hpSC-derived neuronal cells, two monkeys sham transplanted with an equivalent volume of cell-less media thus serving as the placebo control group and two healthy monkeys serving as a positive control. Behavioral endpoints were recorded to evaluate possible adverse effects.

Subsequent to implantation of the neuronal cells, all monkeys in the treatment group had higher levels of dopamine in the brain compared with the control group. Additionally, the rats in the treatment group showed gradual improvements in motor symptoms consistent with cells survival, engraftment and dopamine release. No adverse events, including dyskinesia, deformations, tumors or overgrowth, were observed in the rat or monkey treatment groups. Overall, these results provide evidence to support the hypothesis that hpSC-derived neuronal cells can be safe and have a disease modifying effect. These results, although preliminary, are a strong indication that our approach to Parkinson’s disease can succeed.

"These results are pivotal for our pre-clinical Parkinson's program showing, as they do, that the hpSC-derived neuronal cells can potentially ameliorate the behavioral symptoms without triggering dyskinesias. This data forms the foundation of our discussions with the FDA as we move towards our IND in 2013,” said Dr. Ruslan Semechkin, Principal Investigator of this study, head of R&D for International Stem Cell Corporation and Member of the American Academy of Neurology.

These results will be presented and discussed at the 65th American Academy of Neurology Annual Meeting, one of the world's most important annual events for neurologists and neuroscience professionals and the largest such international meeting of its kind with more than 12,000 attendees at last year's meeting.

Location: San Diego Convention Center, 111 W Harbor Dr., San Diego, CA 92101
Session: Movement Disorders; Parkinson's Disease Therapeutics

Date and time: March 20th, 2013 at 3:30 PM PDT

Parkinson’s disease (PD) is a debilitating neurodegenerative disorder characterized by a progressive degeneration of dopamine-producing neurons in the central nervous system. Approximately 60,000 American’s are diagnosed with PD every year, world-wide there are thought to be as many as ten million sufferers. Current PD treatments, including small molecule such as Levadopa which replaces the lost dopamine, are useful in the relatively early stage of the disease. As symptoms grow worse, the efficacy of such therapies declines, leaving many patients severely disabled.


About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.

To receive ongoing corporate communications via email, visit: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0

To like our Facebook page or follow us on Twitter for company updates and industry related news, visit: www.facebook.com/InternationalStemCellCorporation and www.twitter.com/intlstemcell

Safe harbor statement

Statements pertaining to anticipated developments, the potential use of technologies to develop therapeutic products, potential 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" or "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.

Contacts:

International Stem Cell Corporation

Dr. Simon Craw, Executive Vice President

Phone: 760-940-6383

Email: ir@intlstemcell.com



Dr. Ruslan Semechkin, Vice President, R&D

Phone: 760-940-6383

Email: ras@intlstemcell.com

ISCO Featured in FierceBiotech

FierceBiotech on of the most respected and popular daily news feeds featured ISCO's breakthrough in Parkinson's disease yesterday under the title "Stem cell therapy shows promise for Parkinson's treatment"

Here's the link to the full article




The data is being presented today at the 65th Annual Meeting of the American Academy of Neurology

Friday, March 15, 2013

International Stem Cell Corporation Technology for Deriving Neuronal Cell Published by Nature Publishing Group

CARLSBAD, Calif. – (Marketwire) -- March 15, 2013 -- International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) a California-based biotechnology company focused on the therapeutic applications of human parthenogenetic stem cells announced today the publication of its breakthrough method of deriving neuronal cells for the treatment of Parkinson’s disease in Scientific Reports, a primary research publication from the publishers of Nature.

Parkinson's disease (PD) is a common neurodegenerative disorder caused by a progressive loss of dopamine-producing neurons resulting in gradual dysfunction of the motor system.  Pharmacological treatments are useful in the relatively early stage of the disease, but the continuous use of pro-dopaminergic medication eventually becomes ineffective and can worsen some of the motor symptoms.  ISCO is developing a treatment for PD based on replacing the lost neurons with new neuronal cells derived from human parthenogenetic stem cell (hpSC).  To this end ISCO has developed a sophisticated and efficient manufacturing process for neuronal cells which has now been published in the peer-reviewed journal Scientific Reports.

The paper describes the technology, developed by ISCO's R&D team, for producing highly pure populations of GMP-grade neuronal cells suitable for pre-clinical studies and clinical trials.  The neuronal cells created using this approach are further characterized using a number of analytical methods and shown to function in a similar manner to adult cells.  Moreover, neuronal cells produced using this new method, express greater levels of dopamine, the neurotransmitter central to PD, than previously reported approaches.  Furthermore, these hpSC-derived neuronal cells are cryopreservable and can be stored frozen, and provides a practical route to creating neurons of sufficient quality to be used to treat Parkinson’s disease patients. 
“It is important that our research is reviewed and validated by the scientific community”, commented Dr. Andrey Semechkin, CEO and Co-Chairman, “and being able to publish in a Nature-branded, journal provides confirmation of the quality of ISCO’s scientific research”
The full-text of the publication can be found at http://www.nature.com/srep/2013/130315/srep01463/full/srep01463.html


About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products.  ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos.  ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.
To receive ongoing corporate communications via email, visit:  http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0

To like our Facebook page or follow us on Twitter for company updates and industry related news, visit: www.facebook.com/InternationalStemCellCorporation and www.twitter.com/intlstemcell

Contacts:
International Stem Cell Corporation
Dr. Simon Craw, Executive Vice President
Phone: 760-940-6383
Email: ir@intlstemcell.com

Dr. Ruslan Semechkin, Vice President, R&D
Phone: 760-940-6383
Email: ras@intlstemcell.com

Investor Relations:
MZ Group
Mark McPartland
Senior Vice President
Phone: 212-301-7130
Email: markmcp@mzgroup.us
Web: www.mzgroup.com

International Stem Cell Corporation Raises Additional Capital

CARLSBAD, Calif. – (Marketwire) -- March 14, 2013 -- International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) a California-based biotechnology company focused on the therapeutic applications of human parthenogenetic stem cells announced today that it raised $1 million from a number of existing long-standing investors through a private placement on the same terms as the transaction with management reported on January 22, 2013.

The funds, along with those received in January 2013 from members of management, will help advance the Company’s therapeutic programs towards clinical stage, building upon recently announced promising pre-clinical data for both the Parkinson’s and Liver disease programs. 

“To have a number of existing shareholders increase their positions is gratifying and shows a high level of confidence in our ability to deliver milestones and build shareholder value”, commented Dr. Andrey Semechkin, CEO and Co-Chairman of ISCO,  “It is very important to us that we have the trust of some of our largest shareholders and that they have a strong belief in both management’s ability to execute and the technical, medical and immunological advantages of human parthenogenetic stem cells.  We believe passionately that hpSC will play an important part in the development of treatments for a number of incurable diseases and this investment brings this vision closer to reality”.

About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products.  ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos.  ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.
To receive ongoing corporate communications via email, visit:  http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0
To like our Facebook page or follow us on Twitter for company updates and industry related news, visit: www.facebook.com/InternationalStemCellCorporation and www.twitter.com/intlstemcell

Statements pertaining to anticipated developments, the potential use of technologies to develop therapeutic products, potential 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" or "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. 

Contacts:

International Stem Cell Corporation
Dr. Simon Craw, Executive Vice President
Phone: 760-940-6383


Investor Relations:
MZ Group
Mark McPartland
Senior Vice President
Phone: 212-301-7130
Email: markmcp@mzgroup.us