ISCO Science BLOG

Time 2020-12-21 23:06:49

Web Name: ISCO Science BLOG

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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 Cellsin Parkinson's Chimp Study International Stem Cell Corp. (OTCBB: ISCO) has announced the mostimportant experimental results in the history of the company. Last week, thecompany confirmed that these nonembryonic stem cells can, in fact, become partof a host primate and function as normal cells. Now I'm really feeling guilty because this story deserves a lot more attentionthan I can give it today. Nevertheless, ISCO presented the results of a blindedchimpanzee Parkinson's study at the American Academy of Neurology's 65th annualmeeting on March 20. There's been a lot of skepticism about the potential of human parthenogenicstem cells (hpSCs) for several reasons. First of all, they don't developnaturally into adult cells such as the neuronal cells that were used in thisstudy. Unlike regular cells that have the DNA of both the mother and thefather, hpSC DNA consists of two copies of the mother's genome, so they have tobe provoked to become specific cell types. As a result, these hpSCs, which come from immature ova gathered during routinein vitro fertilization procedures, cannot develop into fetuses. Obviously, thisis a big plus for those who oppose the use of embryonic stem cells. It raisedthe possibility, however, that cells derived from hpSCs would not function asnormal cells for therapeutic purposes. We now know that hpSC-derived neuronal cells will engraft, became part of thechimps they were given to and produce the dopamine needed to prevent thesymptoms 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 primatestudies before going into human studies, hopefully early next year. Theimportance of hpSC therapies, however, shouldn't be overlooked. Not only dothey provide a solution to many people's ethical problems with embryonicallyderived stem cells, they represent an entirely different path to regenerativemedicine. As you know, BioTime (AMEX: BTX) recently consolidated Geron's stem cellIP. BioTime is now the undisputed leader in embryonic and induced pluripotentstem cells. ISCO, however, owns the parthenogenic space. It uses methods ofdifferentiation and cell production that are unique to its own platform. Infact, far more information is available about those technologies in this journal article of Nature'sScientific Reports. Ultimately, when the company has created its bank of cell lines to match mostof the human species, it will be able to provide off-the-shelf therapeuticcells for many conditions that would otherwise require immune suppression oriPS cells. This is a big deal, but it's also in the future. The Parkinson'sdisease target is ideal for ISCO because there is no immune rejection behindthe blood-brain barrier where these cells are required. This enables a path torevenues much sooner than the cell line strategy. I'll have a lot more on this development in the future. Already, these resultshave generated more attention in the scientific press than the company has everbefore garnered. That will increase as more information is released. Also, by the way, the company's hpSC cosmetics line, Lifeline Skin Care, isgetting serious attention in the beauty and anti-aging press. Products have recently been featured in Elleand Prevention magazines as well as other forums. Yours for transformational profits, Patrick Cox 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 92101Session: Movement Disorders; Parkinson's Disease TherapeuticsDate and time: March 20th, 2013 at 3:30 PM PDTParkinson'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 CorporationInternational 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=0To 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/intlstemcellSafe harbor statementStatements 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 CorporationDr. Simon CrawExecutive Vice PresidentPhone: 760-940-6383Email: ir@intlstemcell.comDr. Ruslan SemechkinVice President, R DPhone: 760-940-6383Email: ras@intlstemcell.com 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 cells (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 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.The full-text of the publication can be found at http://www.nature.com/srep/2013/130315/srep01463/full/srep01463.htmlContacts:International Stem Cell CorporationDr. Simon CrawExecutive Vice PresidentPhone: 760-940-6383Email: ir@intlstemcell.comDr. Ruslan SemechkinVice President, R DPhone: 760-940-6383Email: ras@intlstemcell.com International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) ("ISCO" or "the Company") a California-based biotechnology company, today announced the conclusions from its study demonstrating the efficacy and safety of the hepatocyte-like cells (HLC) derived from human parthenogenetic stem cells (hpSC) in a well-established animal model of a congenital liver disorder associated with bilirubin metabolism. The data from this pre-clinical study indicates that implanting HLC in rodents produced both a significant initial decrease and the long-term stabilization of bilirubin levels in blood serum.Criggler-Najjar syndrome type 1 (CN1) is a rare inherited metabolic disorder in which the sufferer's liver lacks a specific enzyme -- UGT1A1, which is essential for the clearance of the toxin bilirubin. The syndrome results in unconjugated hyperbilirubinemia, a disorder characterized by severe neurological complications which, if left untreated, can lead to irreversible acute encephalopathy. Allogenic hepatocyte transplantation (HT) has been used as an alternative therapeutic option for patients with liver-based metabolic diseases including CN1. However, one of the major factors limiting the clinical advancement of human HT is a shortage of mature, functioning human hepatocytes as well as the limited repopulation capacity of grafted adult cells.The use of HLCs to treat CN1 has several potential advantages over transplantation of primary hepatocytes. Firstly, HLC would circumvent the shortage of primary cells, as they can be produced and expanded in vitro. Secondly, there is evidence to suggest that grafting HLC may yield better long-term repopulation and persistent metabolic activity than using immature fetal hepatocytes. HLCs can also be given before the onset of bilirubin encephalopathy occurs, and can thus provide sufficient amounts of UGT1A1 to allow the liver to metabolize this toxin.ISCO has previously reported how these HLC engraft in the liver of Gunn rats, a well-validated model of CN1 where the animals lack UGT1A1 and therefore accumulate toxic levels of unconjugated bilirubin. In addition to this result, no adverse safety signals were detected 16 weeks after the implantation of a therapeutic dose of HLC, and serum levels of bilirubin continued to decline, compared with the control group, up to the conclusion of the observation period at week 19. Moreover, the overall structure and morphology of the liver in all rodents in the treatment group appeared to be undamaged, with no apparent inflammation, tumorigenicity or cell rejection observed.Dr. Ruslan Semechkin, Vice President - head of R D for ISCO comments: "This study provides important evidence for the use of our HLC product as a viable source of transplantable cells for the treatment of CN1. Having completed this study, we can now discuss with the FDA the requirements for our Investigational New Drug (IND) application and phase 1 clinical trial."About International Stem Cell CorporationInternational 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=0To 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/intlstemcellSafe harbor statementStatements pertaining to anticipated developments, the potential use of technologies to develop therapeutic 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" 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.Progress in our Parkinson's disease program was showcased inBreakthroughTechnology Alert today. Read here.CARLSBAD, CA -- (Marketwire) -- 02/04/13 -- International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) ("ISCO" or "the Company") a California-based biotechnology company, today announced positive 12-week results from its pre-clinical in vivo Parkinson's disease study. The primary goal of this behavioral study was to demonstrate the therapeutic benefits of neuronal cells derived from human parthenogenetic stem cell (hpSC) line in a rat model of Parkinson's disease (PD).Parkinson's disease is a common neurodegenerative disorder caused by a progressive degeneration of dopamine-producing neurons resulting in gradual dysfunction of the motor system and can eventually lead to death. 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 cause some of the motor symptoms to worsen.Our proprietary technology is based on a homogeneous population of neuronal cells (NCs) derived from homozygous hpSCs through a scalable and efficient method, developed by ISCO's R D team. These parthenogenetically-derived NCs are cryopreservable and can become neurons once they are implanted into the brain. As such, they hold significant therapeutic potential not only because they can differentiate into dopamine-producing neurons, but also because these cells deliver trophic factors that may be able to provide a level of protection to existing neurons affected by the disease.The animal model used in this study is the 6-OHDA lesioned rat, a well-established and validated model of PD which has been used extensively in the development and testing of drugs for the treatment of PD. The pharmacological induction of rotational behavior in rats is widely used to assess the effects of lesions and potential of cell therapy to effectively replace the dopaminergic system in the rat brain and thus serves as a model of PD. The experimental rats with unilateral dopamine (6-OHDA) lesions survived the inoculation of cells into the brain and signs of improvement in rotational behavior of these animals were clearly observed. Correlational analysis of rotation intensity demonstrated a difference between the drug effects in the control group vs. experimental (transplanted) group of animals. These interim results demonstrate that a single injection of hpSC-derived neuronal cells into the striatum of rats with induced PD symptoms can lead to a significant slowdown in the progression of the disease.Dr. Ruslan Semechkin, Vice President - head of R D, comments: "This is a very important result for our pre-clinical Parkinson's program. The initial in vivo results are very encouraging and show the therapeutic promise of hpSC-derived neuronal cells in the treatment of individuals with Parkinson's disease. Results from this behavioral study will be presented and discussed together with the results of non-human primate study before the end of the first quarter of 2013"About International Stem Cell CorporationInternational 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 atwww.internationalstemcell.com.To receive ongoing corporate communications via email, visit:http://www.b2i.us/irpass.asp?BzID=1468 to=ea s=0To like our Facebook page or follow us on Twitter for company updates and industry related news, visit:www.facebook.com/InternationalStemCellCorporationandwww.twitter.com/intlstemcellSafe harbor statementStatements pertaining to anticipated developments, the potential use of technologies to develop therapeutic 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" 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.

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