NEWARK, Calif., Sept. 6, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the California Institute for Regenerative Medicine (CIRM) has approved an award to the Company for up to $20 million under CIRM's Disease Team Therapy Development Award program (RFA 10-05). The award is to fund preclinical development of StemCells' proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in Alzheimer's disease over a maximum four-year period, with the goal of filing an investigational new drug (IND) application for a clinical trial in that time. In July, CIRM approved a separate award to the Company under RFA 10-05 for up to $20 million to fund preclinical development of HuCNS-SC cells in cervical spinal cord injury.

"With the recent spate of late-stage clinical failures in Alzheimer's disease, it is clear that the field could benefit from alternative approaches to lessen the huge burden on families, caregivers and our healthcare system," commented Martin McGlynn, President and CEO of StemCells, Inc. "Our recently reported preclinical data, which showed that our neural stem cells restored memory and enhanced synaptic function in two animal models relevant to Alzheimer's disease, shows our approach has promise. We greatly appreciate the support from CIRM, which should help us accelerate our efforts to test our HuCNS-SC cells in Alzheimer's disease."

StemCells will evaluate its HuCNS-SC cells as a potential treatment for Alzheimer's disease in collaboration with Frank LaFerla, Ph.D., a world-renowned researcher in the field. Dr. LaFerla is Director of the University of California, Irvine (UCI) Institute for Memory Impairments and Neurological Disorders (UCI MIND), and Chancellor's Professor, Neurobiology and Behavior in the School of Biological Sciences at UCI.

Mr. McGlynn added, "CIRM's approval of two awards to StemCells illustrates the tremendous promise of our neural stem cell technology and the high degree of confidence in the world class team of scientists and clinicians who will be working to translate this technology into potential treatments and cures for these devastating diseases."

About CIRM

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding.

About StemCells, Inc.

StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company has shown preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The Company is also conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and recently reported positive interim data for the first patient cohort. The Company has also initiated a Phase I/II clinical trial in dry age-related macular degeneration (AMD), and is pursuing preclinical studies in Alzheimer's disease. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand. Further information about StemCells is available at http://www.stemcellsinc.com.

The StemCells, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7014

Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the Securities Act of 1933, as amended, and the Securities Exchange Act of 1934, as amended, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding; the potential of the Company's HuCNS-SC cells to treat a broad range of central nervous system disorders such as Alzheimer's disease and spinal cord injury; the prospect of initiating a clinical trial in Alzheimer's disease or cervical spinal cord injury; the timing and prospects for funding by the California Institute for Regenerative Medicine; and the future business operations of the Company, including its ability to conduct clinical trials as well as its other research and product development efforts. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including the fact that additional trials will be required to demonstrate the safety and efficacy of the Company's HuCNS-SC cells for the treatment of any disease or disorder; uncertainty as to whether the results of the Company's preclinical studies will be replicated in humans; uncertainties about the prospect and timing of entering into the agreements necessary to receive funding from CIRM and whether the Company will satisfy, and continue to satisfy, all preconditions for such funding; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current and planned research and development operations; uncertainty as to whether HuCNS-SC cells and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; and other factors that are described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2011, and in its subsequent reports on Forms 10-Q and 8-K.

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StemCells, Inc. Awarded $20 Million From the California Institute for Regenerative Medicine for Alzheimer's Disease ...

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SILVER SPRING, Md. and SINGAPORE, Singapore, Sept. 6, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced today its wholly-owned subsidiary, Austrianova Singapore Pte Ltd (ASPL) has just received notice that the U.S. based Personal Care Products Council (PCPC) has assigned the International Nomenclature Cosmetic Ingredient (INCI) name Gel8 (pronounced gelate), to the company's proprietary cell encapsulation materials.

The PCPC determined, through the International Nomenclature Committee, to provide the Gel8 name assignment to ASPL. As a result, Gel8 will now be included as a new product in the International Cosmetic Ingredient Dictionary & Handbook as well as in the next update of the Cosmetic industry's International Buyer's Guide. The combined Dictionary/Handbook contains more than 19,100 INCI labeling names for the United States, European Union, Japan and other countries. There are many aspects of cosmetics safety and labeling overseen by the FDA and other regulatory bodies around the world. Receiving the INCI designation allows for greater potential use in cosmetics throughout the US and the world.

Dr. John Dangerfield, Chief Operating Officer of ASPL, said, "We are thrilled that our work has enabled us to receive this designation. Most importantly, holding the INCI name is necessary for product safety and visibility as well as a significant step forward for using the Cell-in-a-Box(R) and Bac-in-a-Box(R) technology throughout the cosmetics industry. We see applications of the technology that expand from the cancer field well into other areas of medicine, cosmetics, and nutrition, among others, and see this designation as one of the first important steps."

The Chief Executive of Nuvilex, Dr. Robert Ryan, stated, "I was extremely impressed by what I saw while visiting ASPL in Singapore last week. I'm certain the effort they put forward to receive assignment of the INCI name Gel8 for our live cell components from our Cell-in-a-Box(R) and Bac-in-a-Box(R) patented encapsulation technology from the Personal Care Products Council is a significant move forward. All of the effort by the lab over the past year will allow us to expand the use of the technology and ultimately be able to produce products for other markets including the cosmetics market. The ability to receive the INCI designation also demonstrates how broad this platform technology we are developing together with ASPL is."

About Nuvilex

Nuvilex, Inc. (NVLX) is an international biotechnology provider of live therapeutically valuable, encapsulated cells and services for research and medicine. A great deal of work is ongoing to move Nuvilex and its Austrianova Singapore subsidiary forward. This was clearly apparent during Dr. Ryan's recent trip to Singapore completed last week. Our company's clinical offerings will include cancer, diabetes and other treatments using the company's cell and gene therapy expertise and live-cell encapsulation technology.

The Nuvilex, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13494

Safe Harbor Statement

This press release contains forward-looking statements described within the 1995 Private Securities Litigation Reform Act involving risks and uncertainties including product demand, market competition, and meeting current or future plans which may cause actual results, events, and performances, expressed or implied, to vary and/or differ from those contemplated or predicted. Investors should study and understand all risks before making an investment decision. Readers are recommended not to place undue reliance on forward-looking statements or information. Nuvilex is not obliged to publicly release revisions to any forward-looking statement, reflect events or circumstances afterward, or disclose unanticipated occurrences, except as required under applicable laws.

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Nuvilex Cleared to Enter Multi Billion Dollar Cosmetics Industry

Recommendation and review posted by Bethany Smith

05-09-2012 16:14 Hank brings us breaking news from a team of geneticists working on figuring out what all that "junk DNA" in the human genome really is - turns out it's not junk after all. Like SciShow: Follow SciShow: References for this episode can be found in the Google document here: scishow, news, breaking, hank green, gene, genetics, DNA, junk DNA, ENCODE project, human genome project, code, instruction, protein, encyclopedia of DNA elements, biomedical, research, function, regulatory gene, regulator, cell, medicine, tissue sample, disease, autoimmune diseases, type 1 diabetes, illness, learn, discovery, immune system, predict, treat, noncoding DNA

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The Secret of Your "Junk," Revealed! - Video

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TUESDAY, Sept. 4 (HealthDay News) -- At least seven antibiotic-resistant genes have recently passed between soil bacteria and bacteria that cause human disease, according to a new study.

Further research is needed to determine how widespread this sharing is, and to what extent it could make disease-causing bacteria harder to control, said the researchers at Washington University School of Medicine in St. Louis.

"It is commonplace for antibiotics to make their way into the environment. Our results suggest that this may enhance drug resistance in soil bacteria in ways that could one day be shared with bacteria that cause human disease," first author and graduate student Kevin Forsberg said in a university news release.

For this study, the researchers analyzed the DNA of bacteria in soil samples collected at various locations in the United States. The findings were published recently in the journal Science.

The researchers said it's important to find the answers to many questions, such as: Did the genes pass from soil bacteria to human pathogens or vice versa? Are the genes just the tip of a vast reservoir of shared resistance? Did some combination of luck and a new technique for studying genes across entire bacterial communities lead to the discovery of the shared resistance genes?

While humans only mix their genes when they have children, bacteria regularly exchange genes throughout their lifecycles. That means that when a strain of bacteria develops resistance to antibiotics, it can share this ability not only with its descendants but also with other bacteria, the researchers explained.

"I suspect the soil is not a teeming reservoir of resistance genes. But if factory farms or medical clinics continue to release antibiotics into the environment, it may enrich that reservoir, potentially making resistance genes more accessible to infectious bacteria," study senior author Gautam Dantas, an assistant professor of pathology and immunology, said in the news release.

-- Robert Preidt

Copyright 2012 HealthDay. All rights reserved.

SOURCE: Washington University School of Medicine in St. Louis, news release, Aug. 30, 2012

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Environmental 'Gene-Swapping' May Play Part in Antibiotic Resistance

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DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/b9kb5p/rnai_technologie) has announced the addition of Jain PharmaBiotech's new report "RNAi - Technologies, Markets and Companies" to their offering.

RNA interference (RNAi) or gene silencing involves the use of double stranded RNA (dsRNA). Once inside the cell, this material is processed into short 21-23 nucleotide RNAs termed siRNAs that are used in a sequence-specific manner to recognize and destroy complementary RNA. The report compares RNAi with other antisense approaches using oligonucleotides, aptamers, ribozymes, peptide nucleic acid and locked nucleic acid.

Various RNAi technologies are described, along with design and methods of manufacture of siRNA reagents. These include chemical synthesis by in vitro transcription and use of plasmid or viral vectors. Other approaches to RNAi include DNA-directed RNAi (ddRNAi) that is used to produce dsRNA inside the cell, which is cleaved into siRNA by the action of Dicer, a specific type of RNAse III. MicroRNAs are derived by processing of short hairpins that can inhibit the mRNAs. Expressed interfering RNA (eiRNA) is used to express dsRNA intracellularly from DNA plasmids.

Delivery of therapeutics to the target tissues is an important consideration. siRNAs can be delivered to cells in culture by electroporation or by transfection using plasmid or viral vectors. In vivo delivery of siRNAs can be carried out by injection into tissues or blood vessels or use of synthetic and viral vectors.

Because of its ability to silence any gene once the sequence is known, RNAi has been adopted as the research tool to discriminate gene function. After the genome of an organism is sequenced, RNAi can be designed to target every gene in the genome and target for specific phenotypes. Several methods of gene expression analysis are available and there is still need for sensitive methods of detection of gene expression as a baseline and measurement after gene silencing. RNAi microarray has been devised and can be tailored to meet the needs for high throughput screens for identifying appropriate RNAi probes. RNAi is an important method for analyzing gene function and identifying new drug targets that uses double-stranded RNA to knock down or silence specific genes. With the advent of vector-mediated siRNA delivery methods it is now possible to make transgenic animals that can silence gene expression stably. These technologies point to the usefulness of RNAi for drug discovery.

RNAi can be rationally designed to block the expression of any target gene, including genes for which traditional small molecule inhibitors cannot be found. Areas of therapeutic applications include virus infections, cancer, genetic disorders and neurological diseases. Research at academic centers that is relevant to RNAi-based therapeutics is mentioned.

Regulatory, safety and patent issues are discussed. Side effects can result from unintended interaction between an siRNA compound and an unrelated host gene. If RNAi compounds are designed poorly, there is an increased chance for non-specific interaction with host genes that may cause adverse effects in the host. However, there are no major safety concerns and regulations are in preliminary stages as the clinical trials are still ongoing and there are no marketed products. Many of the patents are still pending.

The markets for RNAi are difficult to define as no RNAi-based product is approved yet but several are in clinical trials. The major use of RNAi reagents is in research but it partially overlaps that of drug discovery and therapeutic development. Various markets relevant to RNAi are analyzed from 2011 to 2021. Markets are also analyzed according to breakdown of technologies and use of siRNAs, miRNAs, etc.

Profiles of 161 companies involved in developing RNAi technologies are presented along with 226 collaborations. They are a mix of companies that supply reagents and technologies (nearly half of all) and companies that use the technologies for drug discovery. Out of these, 33 are developing RNAi-based therapeutics and 34 are involved in microRNAs. The bibliography contains selected 600 publications that are cited in the report. The text is supplemented with 34 tables and 10 figures.

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Research and Markets: RNAi - Technologies, Markets and Companies - Updated 2012 Report

Recommendation and review posted by Bethany Smith

ScienceDaily (Sep. 5, 2012) The first integrated understanding of how the human genome functions will be published this week -- the triumphant result of a collaborative five-year project involving more than 440 researchers working in 32 labs worldwide. The Encyclopedia of DNA Elements project, known as ENCODE, will publish simultaneously on Sept. 6 a massive number of scientific papers, including one main integrative paper and five others in Nature; 18 in Genome Research; six in Genome Biology; and other affiliated papers in Science, Cell, and other scientific journals.

During the ENCODE study, researchers found that more than 80 percent of the human genome sequence is linked to biological function. They also mapped more than 4 million regulatory regions where proteins interact with the DNA with exquisite specificity. These findings are a significant advance in understanding the precise and complex controls over the expression of genetic information within a cell.

"Penn State's contribution to the ENCODE project involves using the new ENCODE data to help explain how genetic variants that do not affect the structure of encoded proteins could affect a person's susceptibility to disease," said Ross Hardison, the T. Ming Chu Professor of Biochemistry and Molecular Biology at Penn State and a member of the ENCODE research team. The research, led by Hardison, is highlighted in the main integrative ENCODE paper to be published in the journal Nature.

"Genome-wide association studies can map with high resolution the places on our genomes where variation in the DNA sequence among individual persons affects their likelihood of having diabetes, cardiac disease, any of a large number of autoimmune diseases such as Crohn's disease, and other common diseases," Hardison said. Because most of these genetic variations are not in regions of the DNA that contain the codes for producing proteins, scientists suspected that some of these noncoding regions might have an important role in controlling the expression of genes.

Hardison's team at Penn State worked with others in the ENCODE Consortium to show, on a genome-wide scale, that many of the DNA regions that do not hold codes for proteins do, indeed, have an important role in controlling which genes are turned on and which are turned off. "Moreover, our research has made it possible to generate specific molecular hypotheses for how genetic variants in these DNA regions that control gene expression could affect the susceptibility to disease," Hardison said. "We demonstrate this process using, as an example, a locus associated with Crohn's and a few other autoimmune diseases. It is exciting to see our basic research revealing insights that help the progress of medical science, potentially facilitating a more personalized approach to medical practice."

In addition to Hardison, other Penn State scientists whose work on the ENCODE project is featured among the papers to be published on Sept. 6 include Programmer/Analyst Belinda Giardine, postdoctoral scholars Robert S. Harris and Weisheng Wu, and Professor of Biology and of Computer Science and Engineering Webb Miller.

The overall ENCODE findings bring into much sharper focus the continually active genome in which proteins routinely turn genes on and off using sites that are sometimes at great distances from the genes they regulate; where sites on a chromosome interact with each other, also sometimes at great distances; where chemical modifications of DNA influence gene expression; and where various functional forms of RNA, a form of nucleic acid related to DNA, help regulate the whole system. "The ENCODE catalog is like Google Maps for the human genome," said Elise Feingold, a program director at the National Institutes of Health National Human Genome Research Institute (NHGRI), who helped to start the ENCODE Project. "The ENCODE maps allow researchers to inspect the chromosomes, genes, functional elements and individual nucleotides in the human genome in much the same way."

"During the early debates about the Human Genome Project, researchers had calculated that only a few percent of the sequence encoded proteins, the workhorses of the cell," said Eric D. Green, director of NHGRI. "Early on, some scientists even argued that most of the genome was 'junk.' ENCODE now gives us much more appreciation of the complex molecular ballet that converts genetic information into living cells and organisms, and we can now say that there is very little, if any, junk DNA."

Hundreds of researchers in the United States, United Kingdom, Spain, Singapore and Japan performed more than 1,600 sets of experiments on 147 types of tissue with technologies standardized across the consortium. The experiments relied on innovative uses of new next-generation sequencing technologies enabled, in part, by NHGRI's technology initiative for DNA sequencing. In total, ENCODE generated more than 15-trillion bytes of raw data and its analysis consumed the equivalent of more than 300 years of computer time.

The ENCODE project received principal funding from the National Human Genome Research Institute at the National Institutes of Health. Computation was enabled, in part, through the Penn State Cyberstar Computer, funded by the National Science Foundation (grant OCI-0821527).

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Huge human gene study includes Penn State University research

Recommendation and review posted by Bethany Smith

Researchers have discovered a gene mutation that protects people in Southeast Asia against malaria in much the same fashion that a sickle cell trait protects Africans from the disease.

But while the sickle cell protects against the frequently lethal form of the disease caused by the parasite Plasmodium falciparum, the newly discovered gene mutation protects against Plasmodium vivax, which is generally thought to be more benign.

Malaria causes an estimated 1 million deaths per year worldwide, and at least half the world's population lives in areas at risk for the disease.

The sickle cell trait developed in Africa, where malaria is epidemic. People with one mutated gene have red blood cells that may occasionally assume a characteristic sickled shape under stress, generally with few or no severe effects.

People carrying two copies of the mutated gene, however, show much more severe sickling under similar conditions, and this can impede blood flow to organs, causing severe pain and organ deterioration.

The disease can be fatal. The trait has survived in the population, however, because carriers have a decreased vulnerability to P. falciparum malaria. They contract the disease, but symptoms are less severe.

The mechanism by which the trait provides protection is not yet clear, but it may involve changes in the membranes of red blood cells, which are attacked by the parasite.

A team of researchers led by geneticist Ivo Mueller of the Walter and Eliza Hall Institute in Parkville, Australia, and the Barcelona Center for International Health Research in Spain studied a genetic disorder called Southeast Asian Ovalocytosis or SAO, which causes red blood cells to become elliptical in shape or rod-shaped.

One copy of the gene produces mild disease, while two copies are generally lethal. Previous studies have shown that the carrier or trait form of the disorder, with one mutated gene, provides some protection against P. falciparum. Now Mueller and his colleagues have demonstrated that the mutation provides significant protection against P. vivax.

The team reported in the journal PLoS Medicine that they studied 1,975 children under the age of 15 in three independent epidemiological studies in Papua, New Guinea. About 10% to 15% of the population in that area carries the mutation.

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Newly discovered gene mutation offers malaria defense like sickle cell

Recommendation and review posted by Bethany Smith

September 5, 2012

Connie K. Ho for redOrbit.com Your Universe Online

A group of scientists from Singapore recently discovered three new genes related to Primary Angle Closure Glaucoma (PACG), which is one of the main causes of blindness for Chinese people.

The new findings, recently published in the scientific journal Nature Genetics, will assist 15 million people throughout the globe who suffer from the disease.

This provides further evidence that genetic factors play a role in development of PACG, explained lead principal investigator Aung Tin, a professor of ophthalmology at NUH and deputy executive director at SERI who has studied PACG for over 10 years, in a prepared statement. It is a major achievement for our Singapore team leading the largest international consortium of doctors and scientists involved in glaucoma research. The results may lead to new insights into disease understanding and open the possibility of novel treatments in the future as well as the potential of early identification of people at risk of the disease.

80 percent of these 15 million who have PACG reside in Asia and the results can be very beneficial to medical experts who work with this particular population.

This is a landmark study identifying three genes that contribute to angle-closure glaucoma, a form of glaucoma that is particularly common in Asians. These data are the first critical steps toward a better understanding of the underlying molecular events responsible for this blinding disease, mentioned Janey Wiggs, the Paul Austin Chandler Associate Professor of Ophthalmology at Harvard Medical School, in the statement.

The study was a collaborative effort by scientists hailing from the Singapore Eye Research Institute (SERI), Singapore National Eye Centre (SNEC), the Genome Institute of Singapore (GIS), the National University of Singapore (NUS), National University Hospitals (NUH) Department of Ophthalmology and the Tan Tock Seng Hospital (TTSH).

This is a landmark finding, and may potentially change how we view PACG as a disease with genetic links. It highlights how a collective effort from scientists and clinicians and clinician-scientists can unravel diseases of major importance to Singapore. Because this disease is more common in Asians than in the Western populations, such studies will not be done in the US/Europe. This study has to be done in Asia as it is a disease with more implication for Asians. As such, Singapore has led the way forward, noted Wong Tien Yin, executive director of SERI as well as head and chair professor of the Department of Ophthalmology at NUHS, in the statement.

The group of investigators completed a genome-wide association study (GWAS) of 1,854 PACG cases and 9,608 controls of more than five sample collections throughout Asia. Another 1,917 PACG cases received validation experiments and 8,943 controls were taken from another six sample collections on a global scale. 1,293 Singapore residents with PACG and 8,025 Singaporean controls were part of the study.

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Gene Found In Glaucoma Largely Affects Asian Populations

Recommendation and review posted by Bethany Smith

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Linda Aagard 801-587-7639 University of Utah Health Sciences

SALT LAKE CITY For the first time, a mutation in HIF2, a specific group of genes known as transcription factors that is involved in red blood cell production and cell metabolism, has been identified in cancer tumor cells.

Researchers from Huntsman Cancer Institute (HCI) at the University of Utah and the National Institutes of Health found the mutation in tumor cells of two patients with the rare cancers paraganglioma/pheochromocytoma and somatostatinoma. The mutation was previously identified in connection with a non-cancerous hereditary condition, but never before in spontaneously arising cancers. The research results appear in the September 6, 2012 issue of the New England Journal of Medicine.

Transcription factors are proteins that bind to specific sequences of DNA to regulate cell functions. Hypoxia-inducible factors (HIFs) are transcription factors that control a wide range of functions connected to the way cells respond to oxygen, including metabolism (the way energy is produced) and the creation of red blood cells. Increased amounts of HIF in cancer cells was found to be responsible for the unique way they generate energy, referred to as the Warburg effect, named for Otto Warburg, a German physiologist who received the 1931 Nobel Prize in Physiology for this research.

"These HIF pathway mutations were first discovered while studying people with a condition called polycythemia that makes the body overproduce red blood cells," said one of the senior authors, Josef Prchal, M.D., professor in the Division of Hematology and Hematologic Malignancies at the University of Utah School of Medicine, an HCI investigator, and an expert on polycythemic disorders who has previously discovered other mutations in other HIF pathway genes.

The findings raise some important questions for future research. "The patients in which we found these mutations have a rare combination of diseases, paraganglioma/pheochromocytoma and polycythemia, yet they shared similar mutations," said Prchal. "Learning whether HIF pathway mutations are present in other cancers could increase understanding of the mechanisms of cancer cell metabolism and offer a possible new target for cancer treatment development."

###

Felipe Lorenzo, M.D., Ph.D., postdoctoral fellow in the University of Utah Division of Hematology and Hematologic Malignancies, collaborated on this article. Other co-authors are associated with various entities of the National Institutes of Health. The research was supported, in part, by the Intramural Research Program of the National Institutes of Health, the National Institute of Child Health and Human Development, and the National Institute of Neurological Disorders and Stroke and VAH Merit Award to Dr. Prchal. The National Institutes of Health support Huntsman Cancer Institute through P30 CA042014.

The mission of Huntsman Cancer Institute (HCI) at the University of Utah is to understand cancer from its beginnings, to use that knowledge in the creation and improvement of cancer treatments, to relieve the suffering of cancer patients, and to provide education about cancer risk, prevention, and care. HCI is a National Cancer Institute-Designated Cancer Center, which means that it meets the highest national standards for cancer care and research and receives support for its scientific endeavors. HCI is also a member of the National Comprehensive Cancer Network (NCCN), a not-for-profit alliance of the world's leading cancer centers that is dedicated to improving the quality and effectiveness of care provided to patients with cancer. For more information about HCI, please visit http://www.huntsmancancer.org.

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HIF gene mutation found in tumor cells offers new clues about cancer metabolism

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Editor's Choice Main Category: Cystic Fibrosis Article Date: 05 Sep 2012 - 10:00 PDT

Current ratings for: Ivacaftor Improves Lung Function And Symptoms In Cystic Fibrosis Patients With Specific Genetic Mutation

The STRIVE study randomised 161 patients aged 12 and over with cystic fibrosis and at least one copy of the G551D mutation in the CFTR gene to ivacaftor (150mg every 12 hours) or placebo. Results showed a mean absolute improvement of 10.6% in predicted FEV1 after 24 weeks' treatment with ivacaftor compared to placebo (p<0.0001), sustained at 10.5% at 48 weeks.

The ENVISION study, which included 52 children aged 6-11 years, showed similar absolute improvement of 12.5% in predicted FEV1 with ivacaftor at 24 weeks compared to placebo (p<0.0001), with a 10.0% improvement maintained at 48 weeks.

STRIVE showed a significant reduction in pulmonary exacerbations and clinically significant improvement in the respiratory domain of patients' quality of life with ivacaftor. Both studies showed an increase in body weight (mean increases of 2.7kg at 48 weeks in STRIVE and 2.8kg in ENVISION).

"Ivacaftor is the first medicine to treat the underlying cause of cystic fibrosis in people with the G551D mutation, a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene," said Stuart Elborn, Professor of Respiratory Medicine at Queen's University, Belfast, Northern Ireland and a principal investigator. "These data showing the consistent and sustained benefit of this medicine confirm that ivacaftor has the potential to make a significant difference to the lives of children, adolescents and adults with this form of CF."

He added: "The data don't capture the full benefit for patients. It's been very noticeable in the patients I look after that they are able to do things they previously couldn't after starting treatment with ivacaftor. They feel better and more able to plan for the future."

Fewer patients in the ivacaftor treatment groups discontinued treatment due to adverse events compared to placebo in both trials. Most adverse events associated with ivacaftor were mild to moderate, with some of the commonest including headache, upper respiratory tract infection, rash, diarrhea and abdominal pain.

Data reported from the follow-up PERSIST study showed that improvements in lung function with ivacaftor were sustained for patients continuing treatment from the STRIVE and ENVISION trials for up to 96 weeks. Those switched from placebo to open-label ivacaftor showed similar improvements in FEV1 to those seen in the patients starting on the drug during the placebo-controlled trials.

Reporting the findings, Dr Edward McKone, Consultant Respiratory Physician at St Vincent's University Hospital, Dublin, Ireland, said:

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Ivacaftor Improves Lung Function And Symptoms In Cystic Fibrosis Patients With Specific Genetic Mutation

Recommendation and review posted by Bethany Smith

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Haley Bridger hbridger@broadinstitute.org 617-714-7968 Broad Institute of MIT and Harvard

Cambridge, MA. Wed. September 5, 2012 Most of the DNA alterations that are tied to disease do not alter protein-coding genes, but rather the "switches" that control them. Characterizing these switches is one of many goals of the ENCODE project a sweeping, international effort to create a compendium of all of the working parts of the human genome that have not been well studied or well understood.

The function of the vast majority of the human genome has remained largely unknown, but the Encyclopedia of DNA Elements (ENCODE) project, launched in 2003, set out to change that. Comprised of more than 30 participating institutions, including the Broad Institute, the ENCODE Project Consortium has helped to ascribe potential biochemical function to a large fraction of the non-coding genome. This work has revealed elements that act like dimmer switches, subtly turning up or down a gene's activity and influencing what parts of the genome are utilized in different kinds of cells. The team characterized and mapped out the locations of thousands of these switches and signals. More than 30 papers detailing these results appear online in Nature, Science, Genome Research, and Genome Biology this week.

"With these maps in hand, we can begin to understand why genetic variants that land in the annotated regions may predispose people to disease," said Brad Bernstein, a senior associate member at the Broad Institute and an associate professor of pathology at Massachusetts General Hospital (MGH) and Harvard Medical School. Bernstein is also a principal investigator in the ENCODE Consortium. "It turns out that many of the variants that genetic researchers have tied to various diseases lupus, Crohn's disease, metabolic diseases, high cholesterol, and much more sit in these regions that alter how genes are expressed in specific kinds of cells."

Researchers from the Broad, MIT, and MGH found that variants associated with autoimmune diseases such as lupus and rheumatoid arthritis sit in regions that are active only in immune cells, whereas variants tied to cholesterol and metabolic diseases sit in regions active in liver cells.

In order to generate detailed maps of the switches that lie between and even nestled within genes, data collection centers in the consortium amassed high-quality and comprehensive datasets detailing the function of elements in the genome. By looking across more than 140 cell types, they generated more than 1,500 datasets.

Several groups at the Broad Institute contributed to this work, including the Genome Sequencing and Analysis Program and the Epigenomics Program, which helped generate many of the datasets for the project. The term "epigenome" refers to a layer of chemical information on top of the genetic code, which helps determine when and where (and in what types of cells) genes will be active. This layer of information includes a suite of chemical changes that appear across the genetic landscape of every cell, and can differ dramatically between cell types. Researchers at the Broad and at the other ENCODE data collection centers developed ways to characterize these epigenetic "marks" across cell types.

"By bringing together computational groups from across the world and gathering all of the data generated, we can get at much more complex questions," said Manolis Kellis, an associate member of the Broad Institute, principal investigator at the MIT Computer Science and Artificial Intelligence Lab (CSAIL), and an associate professor of Computer Science at MIT. Kellis is also head of the MIT Computational Biology Group and a principal investigator in the ENCODE Consortium.

Maps and data generated through the ENCODE project have been publicly released as they have become available. With these maps, Bernstein, Kellis, and their Broad colleagues:

Original post:
Mapping a genetic world beyond genes

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/96czlk/gene_therapy_tec) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2011, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2011-2021. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

The voluminous literature on gene therapy was reviewed and selected 700 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 72 tables and 14 figures.

Profiles of 187 companies involved in developing gene therapy are presented along with 208 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Read more:
Research and Markets: Gene Therapy - Technologies, Markets and Companies - Updated 2012 Report

Recommendation and review posted by Bethany Smith

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Carolann Murphy, PA CMurphy@KesslerFoundation.org 973-324-8382 Kessler Foundation

West Orange, NJ. September 5, 2012. Kessler Foundation has released preliminary research findings from its clinical study of the wearable robotic exoskeletal device, Ekso (Ekso Bionics). Gail Forrest, PhD, assistant director of Human Performance and Engineering Research, presented the Ekso research data on September 3, at the meeting of the Academy of Spinal Cord Injury Professionals at the Rio Suites in Las Vegas. Dr. Forrest directs mobility research at the Foundation, including activity-based locomotor therapy, functional electrical stimulation, and treadmill training with the LokomatPro v6, as well as Ekso. Her research focuses on new ways to improve function and restore mobility for people with disabilities and reduce their long-term risks for complications.

Ekso, has been undergoing clinical investigation in patients with spinal cord injury at Kessler since October 2011, when the research team received the second commercial unit distributed by Ekso Bionics. "Our initial research results are promising for the potential application of Ekso-assisted walking in rehabilitation, in exercise/wellness programs, in the community and for home use," said Dr. Forrest.

Dr. Forrest not only studies the mechanics of how people with paralysis stand and walk in Ekso, she looks at the impact of these activities on their muscles, hearts and lungs. Long-term studies are needed to evaluate the effects on common secondary complications such as cardiovascular disease, loss of bone and muscle, pressure ulcers, depression, chronic pain, and loss of bladder/bowel control.

Dr. Forrest reported on data collected in 13 patients (12 with paraplegia and 1 with tetraplegia) with spinal cord injury (complete and incomplete injuries) enrolled in the Ekso study. Thus far, walking and standing with Ekso is feasible for people with a range of spinal cord disorders that cause paraplegia. Individuals with higher levels of spinal cord injury may also benefit, but require more time to learn Ekso-assisted walking.

Gait and balance data indicate positive results/progress, ie, for individuals engaging in Ekso-assisted training sessions, walking speed and distance, fluidity, gait and balance improve with training on the exoskeleton. Metabolic and cardiovascular responses were evidenced by increases in oxygen consumption, ventilation and heart rate. These increases occurred with changes from resting to standing position and increased further with changes from standing to walking. Dr. Forrest confirmed this effect by comparing the responses of a patient skilled at Ekso-assisted walking (30 sessions of training) with those of a novice walker. Oxygen consumption returned to baseline much faster in the skilled walker, indicating a training effect. "These are only preliminary data," emphasized Dr. Forrest. "The mechanisms underlying these responses need further investigation. These findings are indicative of potential benefits for the heart, lungs, and the circulation, an important finding in this high-risk population."

Another interesting finding was increased muscle firing in the lower leg muscles during Ekso-assisted walking. More detailed research is needed to evaluate the potential health benefits of this muscle activity, according to Dr. Forrest.

Advances in engineering are enabling advances in Ekso research. Auto-control for greater flexibility and maximal independence is a new feature in the upgraded device, Ekso 1.1, being tested at Kessler Foundation. Data collection is now automated, which will aid the expansion of Ekso's capabilities in the future.

###

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Kessler Foundation releases preliminary research findings for Ekso in spinal cord injury

Recommendation and review posted by sam

WINSTON-SALEM, N.C., Sept. 5, 2012 /PRNewswire/ -- Tengion, Inc. (TNGN), a leader in regenerative medicine, today announced that its common stock will begin trading on the OTCQB tier of the OTC Marketplace ("OTCQB") effective Thursday, September 6, 2012. The OTCQB is the venture marketplace for 3,500 companies that are current in their reporting with the Securities and Exchange Commission. Investors will find Real-Time quotes and market information for the Company on http://www.otcmarkets.com. The Company's shares of common stock will continue to trade under the symbol TNGN.

On September 4, 2012, Tengion received a notice from NASDAQ stating that the Company has not regained compliance with NASDAQ Listing Rule 5550(b)(1) and that its common stock will cease trading on the NASDAQ Capital Market effective on September 6, 2012.

About TengionTengion, a clinical-stage regenerative medicine company, is focused on developing its Organ Regeneration Platform to harness the intrinsic regenerative pathways of the body to regenerate a range of native-like organs and tissues with the goal of delaying or eliminating the need for chronic disease therapies, organ transplantation, and the administration of anti-rejection medications. An initial clinical trial is ongoing for the Company's most advanced product candidate, the Neo-Urinary Conduit, an autologous implant that is intended to catalyze regeneration of native-like urinary tissue for bladder cancer patients requiring a urinary diversion following bladder removal. The Company's lead preclinical candidate is the Neo-Kidney Augment, which is designed to prevent or delay dialysis kidney transplantation by increasing renal function in patients with advanced chronic kidney disease. Tengion has worldwide rights to its product candidates.

Forward-Looking Statements Certain statements set forth above may constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including the Company's plan to transition from the NASDAQ Capital Market to the OTC Marketplace and the Company's ability to continue trading on the OTCQB. Although the Company believes that these statements are based upon reasonable assumptions within the bounds of its knowledge of its business and operations, there are a number of factors that may cause actual results to differ from these statements including actions by the OTCQB Marketplace and the OTC Bulletin Board and the market makers issuing quotations, if any, in the Company's common stock, the Company's immediate need for capital and the Company's ability to remain timely in its reporting requirements with the SEC. For additional factors which could cause actual results to differ from expectations, reference is made to the reports filed by the Company with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended. The forward looking statements in this release are made only as of the date hereof and the Company disclaims any intention or responsibility for updating predictions or expectations in this release.

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Tengion Common Stock to Begin Trading on The OTCQB™ Tier of the OTC Marketplace on September 6, 2012

Recommendation and review posted by sam

NEW YORK, Sept. 5, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NBS) ("NeoStem" or the "Company"), a cell therapy company, today announced that Company management of a NeoStem company, Progenitor Cell Therapy ("PCT"), an internationally recognized contract development and manufacturing organization (CDMO), has been invited to present on its core expertise in development of commercial manufacturing processes for cell therapy at two cell therapy conferences in September. At each, PCT will offer its unique perspective as an industry leader in contract development and manufacturing of cell therapy products, with over 12 years of exclusive cell-therapy focused experience.

Timothy Fong, Ph.D, M.B.A, PCT's Vice President, Technology and Product Development, will be sharing PCT's expertise in cell therapy manufacturing with a focus on commercialization. At IBC Life Sciences' Cell Therapy Bioprocessing Conference, he will chair a panel on quality assurance and controls and will give a presentation entitled "From Concept to Product: Considerations for Developing a Robust Commercial Manufacturing Process", which will include considerations for developing a robust commercial manufacturing process. He will also speak at the Stem Cells USA and Regenerative Medicine Congress on "Cell manufacturing considerations for first-in-world stem cell therapeutics".

Dr. Fong stated, "As a cell therapeutic progresses from concept to product, the development of a commercial manufacturing process may contain unexpected technical and quality issues. The development path should follow several defined steps. My presentations will discuss the key steps in the process and highlight critical areas that need to be addressed to develop a successful commercial manufacturing process. PCT helps clients bridge the gap between discovery and patient care through efficient transfer of cell-based therapies from laboratory into clinical practice."

NeoStem and PCT invite you to attend the conference(s), see Dr. Fong's talks, and connect with the PCT team at PCT's booths. If you are a colleague of PCT or NeoStem, PCT can offer you a registration discount. Please contact PCT at bdm@pctcelltherapy.com for more details.

IBC Life Sciences' 2nd Annual Cell Therapy Bioprocessing Conference

Terrapinn 4th Annual Stem Cells USA and Regenerative Medicine Congress

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT") with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cells product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a patent and patent pending (IP) portfolio, we believe we are well positioned to succeed.

For more information on NeoStem, please visit http://www.neostem.com. For more information on PCT, please visit http://www.pctcelltherapy.com.

See the rest here:
Progenitor Cell Therapy, a NeoStem Company, Invited to Present at Two Conferences in September

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/9f72rt/drug_delivery_in_c) has announced the addition of Jain PharmaBiotech's new report "Drug Delivery in Cancer - Technologies, Markets and Companies" to their offering.

Drug delivery remains a challenge in management of cancer. Approximately 12.5 million new cases of cancer are being diagnosed worldwide each year and considerable research is in progress for drug discovery for cancer. Cancer drug delivery is no longer simply wrapping up cancer drugs in a new formulations for different routes of delivery. The focus is on targeted cancer therapy. The newer approaches to cancer treatment not only supplement the conventional chemotherapy and radiotherapy but also prevent damage to normal tissues and prevent drug resistance.

Innovative cancer therapies are based on current concepts of molecular biology of cancer. These include antiangiogenic agents, immunotherapy, bacterial agents, viral oncolysis, targeting of cyclic-dependent kinases and tyrosine kinase receptors, antisense approaches, gene therapy and combination of various methods. Important methods of immunotherapy in cancer involve use of cytokines, monoclonal antibodies, cancer vaccines and immunogene therapy.

Several innovative methods of drug delivery are used in cancer. These include use of microparticles as carriers of anticancer agents. These may be injected into the arterial circulation and guided to the tumor by magnetic field for targeted drug delivery. Polyethylene glycol (PEG) technology has been used to overcome some of the barriers to anticancer drug delivery. Encapsulating anticancer drugs in liposomes enables targeted drug delivery to tumor tissues and prevents damage to the normal surrounding tissues. Monoclonal antibodies can be used for the delivery of anticancer payloads such as radionucleotides, toxins and chemotherapeutic agents to the tumors.

Antisense oligonucleotides have been in clinical trials for cancer for some time now. RNAi has also been applied in oncology. Small interfering RNAs (siRNAs) can be targeted to tumors and one example is suppression of H-ras gene expression indicating the potential for application in therapy of ovarian cancer. Cancer gene therapy is a sophisticated form of drug delivery for cancer. Various technologies and companies developing them are described. Nucleic acid-based cancer vaccines are also described.

Drug delivery strategies vary according to the type and location of cancer. Role of drug delivery in the management of cancers of the brain, the bladder, the breast, the ovaries and the prostate are used as examples to illustrate different approaches both experimental and clinical. Biodegradable implants of carmustine are already used in the treatment of malignant brain tumors.

The market value of drug delivery technologies and the anticancer drugs are difficult to separate. Cancer market estimates from 2011-2021 are given according to organs involved and the types of cancer as well as according to technologies. Distribution of the into major regions is also described.

Profiles of 213 companies involved in developing innovative cancer therapies and methods of delivery are presented along with their 242 collaborations. The bibliography contains over 650 publications that are cited in the report.The report is supplemented with 58 tables and 9 figures.

Key Topics Covered:

See the article here:
Research and Markets: Drug Delivery in Cancer - Technologies, Markets and Companies - Updated 2012 Report

Recommendation and review posted by Bethany Smith

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 5, 2012According to the Centers for Disease Control and Prevention (CDC), 1 in 88 children in the U.S. has autism spectrum disorder (ASD), a broad group of neurodevelopmental disorders. Children and adolescents with ASD are typically fascinated by screen-based technology such as videogames and these can be used for educational and treatment purposes as described in an insightful Roundtable Discussion published in Games for Health Journal: Research Development, and Clinical Applications, a peer-reviewed publication from Mary Ann Liebert, Inc.. The article is available free on the Games for Health Journal website.

Individuals with ASD have difficulty with communication and social interaction, but they often have particularly good visual perceptual skills and respond well to visual stimuli. Videogames offer opportunities for successful learning, motivation to improve skills such as planning, organization, and self-monitoring, and reinforcement of desired behaviors without the need for direct human-to-human interaction.

Autism is a growing area of interest for the gamification community, and Games for Health Journal continues to explore various aspects of how videogame technology can be beneficial in treating this complex spectrum of disorders. In a previous issue of the Journal, the article "Comparing Energy Expenditure in Adolescents with and without Autism while Playing Nintendo Wii Games" described how gaming might help individuals with ASD increase their daily physical activity to prevent obesity.

"Children and young adults with ASD have unique opportunities to capitalize on their interest and aptitude in videogames as a resource to develop desired social behaviors and life skills and to increase their physical activity," says Games for Health Journal Editor-in-Chief Bill Ferguson, PhD, who moderated the Roundtable.

###

About the Journal

Games for Health Journal (http://www.liebertpub.com/g4h) breaks new ground as the first journal to address this emerging and increasingly important area of health care. The Journal provides a bimonthly forum in print and online for academic and clinical researchers, game designers and developers, health care providers, insurers, and information technology leaders. Articles explore the use of game technology in a variety of clinical applications. These include disease prevention and monitoring, nutrition, weight management, and medication adherence. Gaming can play an important role in the care of patients with diabetes, post-traumatic stress disorder, Alzheimer's disease, and cognitive, mental, emotional, and behavioral health disorders.

About the Publisher

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Can videogaming benefit young people with autism spectrum disorder?

Recommendation and review posted by Bethany Smith

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Cathia Falvey cfalvey@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 5, 2012A group of experts has developed consensus recommendations for future research directions to determine why nearly two-thirds of Americans with Alzheimer's disease (AD) are women. The recommendations are published in a Roundtable discussion in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://www.liebertpub.com/jwh.

An estimated 5.4 million Americans are affected by AD and related dementias, and that number will likely rise to 11-16 million people by the year 2050 if no effective cures or preventive measures are developed. The main risk factors for AD are age and sex, with affected women outnumbering men 2 to 1. This may be due at least in part to the fact that women tend to live longer.

An interdisciplinary roundtable of experts convened by the Society for Women's Health Research (Washington, DC) led to a set of recommendations to help guide future AD research and make the evaluation of sex and gender differences a component of future studies. The consensus recommendations encompass seven themes, including the need to assess the link between sex and AD incidence, raise awareness of sex differences among the research community, and to take into account sex-based differences in the experimental design and data analysis of studies on disease risk, early diagnosis, and drug discovery.

"There are still major gaps in our knowledge of the role of sex and gender in the onset and progression of Alzheimer's disease, and these recommendations will provide a useful guide for future research in this area," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

###

About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the Official Journal of the Academy of Women's Health.

About the Academy

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Why does Alzheimer's disease affect twice as many women as men?

Recommendation and review posted by Bethany Smith

~Varying Perspectives Represented and Best Practices Shared by Experts~

Newswise The 4th Annual Consumer Genetics Conference, a yearly forum designed to spark influential conversation on the current state of personal genomics and to shape the future of the field, is being held October 3-5 at the Seaport Hotel in Boston, Massachusetts. This event will bring together the fields preeminent researchers, clinicians, government regulators and industry leaders, who will provide their perspectives on the direction of this field, and the crucial topics surrounding its implications to clinical health and medicine.

In this highly interactive forum, panel participants will represent a broad spectrum of viewpoints, and will engage in an open discussion on best practices and policies, as well as new advancements and challenges. Through the course of this dynamic exchange, the future of consumer-based genetics will be explored, examined, and shaped.

The deadline to take advantage of discounted early bird registration rates is Friday, September 7.

Major themes to be covered at this conference will include: Personal Genomics, Third-Generation Sequencing, Molecular Diagnostics, Investment & Funding Opportunities, Genome Interpretation, The Future of Personalized Medicine, Big Data, Prenatal/Neonatal & Disease Diagnostics, Empowering Patients, Nutrition, Food Genetics & Cosmetics

Keynote speakers and featured presenters will include:

Diana Bianchi, M.D., Executive Director, Mother Infant Research Institute, Tufts Medical Center Kenneth Chahine, Ph.D., J.D., Senior Vice President and General Manager, DNA, Ancestry.com Brian T. Naughton, Ph.D., Founding Scientist, 23andMe Nathan Pearson, Ph.D., Director of Research, Knome, Inc. John Quackenbush, Ph.D., Professor, Biostatistics and Computational Biology, Cancer Biology Center for Cancer Computational Biology, Dana-Farber Cancer Institute Heidi L. Rehm, Ph.D., FACMG, Chief Laboratory Director, Molecular Medicine, Partners HealthCare Center for Personalized Genetic Medicine; Assistant Professor of Pathology, Harvard Medical School Lee Silver, Ph.D., Professor of Molecular Biology and Public Affairs, Woodrow Wilson School, Princeton University

For more information on the 4th Annual Consumer Genetics Conference, including registration and a full program schedule, please visit http://www.consumergeneticsconference.com.

Media are invited to attend this conference and meet and interview speakers on-site. Registration can be obtained by emailing Lynn Blenkhorn at lynn.blenkhorn@fkhealth.com, or by calling: 508-851-0930. If you cannot attend but you would like to speak with one of the presenters, we would be pleased to organize a phone interview on the day of their presentation.

See the article here:
Consumer Genetics Conference 2012 Features Preeminent Leaders in Industry, Academia, Medicine and Government to ...

Recommendation and review posted by Bethany Smith

ScienceDaily (Sep. 5, 2012) That low bone density causes osteoporosis and a risk of fracture is common knowledge. But an excessively high bone density is also harmful. The most serious form of excessively high bone density is a rare, hereditary disease which can lead to the patient's death by the age of only five. Researchers at Lund University in Sweden are now trying to develop gene therapy against this disease.

In order for the body to function, a balance is necessary between the cells that build up the bones in our skeletons and the cells that break them down. In the disease malignant infantile osteopetrosis, MIOP, the cells that break down the bone tissue do not function as they should, resulting in the skeleton not having sufficient cavities for bone-marrow and nerves.

"Optic and auditory nerves are compressed, causing blindness and deafness in these children. Finally the bone marrow ceases to function and, without treatment, the child dies of anemia and infections," explains Carmen Flores Bjurstrm. She has just completed a thesis which presents some of the research at the division for Molecular Medicine and Gene Therapy in Lund.

The researchers' work focuses on finding alternatives to the only treatment currently available against MIOP, namely a bone-marrow transplant. This treatment can be effective, but it is both risky and dependent on finding a suitable donor.

Gene therapy requires no donor, as stem cells are taken from the patients themselves. Once the cells' non-functioning gene has been replaced with a healthy copy of itself, the stem cells are put back into the patient.

Great hopes have been placed on gene therapy as a treatment method but the work has proven to be more difficult than expected. The method is used today for certain immunodeficiency diseases, and has also been applied to a blood disorder called thalassemia.

"So far, the method is not risk-free. Since it is impossible to control where the introduced gene ends up, there is a certain risk of it ending up in the wrong place and giving rise to leukemia. This is why gene therapy is only used for serious diseases for which there is no good treatment," says Carmen Flores Bjurstrm.

The Lund researchers have conducted experiments with gene therapy in both patient cells and laboratory animals. The next step is to conduct trials on patients. The trials will probably take place at the hospital in Ulm, Germany, which currently treats the majority of children in Europe suffering from MIOP.

MIOP is a rare disease: in Sweden a child is born with the condition approximately once every three years. Worldwide, the incidence of the disease is one case for every 300 000 births. It is, however, more common in Costa Rica where 3-4 children per 100 000 births have the disease.

"But there are several other genetic mutations that lead to other osteopetrosis diseases. If we manage to treat MIOP, it may become possible to treat these other conditions as well," hopes Carmen Flores Bjurstrm along with her supervisor, Professor Johan Richter.

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Can gene therapy cure fatal diseases in children?

Recommendation and review posted by Bethany Smith

NEW YORK, Sept. 5, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NBS) ("NeoStem" or the "Company"), a cell therapy company, today announced that Company management of a NeoStem company, Progenitor Cell Therapy ("PCT"), an internationally recognized contract development and manufacturing organization (CDMO), has been invited to present on its core expertise in development of commercial manufacturing processes for cell therapy at two cell therapy conferences in September. At each, PCT will offer its unique perspective as an industry leader in contract development and manufacturing of cell therapy products, with over 12 years of exclusive cell-therapy focused experience.

Timothy Fong, Ph.D, M.B.A, PCT's Vice President, Technology and Product Development, will be sharing PCT's expertise in cell therapy manufacturing with a focus on commercialization. At IBC Life Sciences' Cell Therapy Bioprocessing Conference, he will chair a panel on quality assurance and controls and will give a presentation entitled "From Concept to Product: Considerations for Developing a Robust Commercial Manufacturing Process", which will include considerations for developing a robust commercial manufacturing process. He will also speak at the Stem Cells USA and Regenerative Medicine Congress on "Cell manufacturing considerations for first-in-world stem cell therapeutics".

Dr. Fong stated, "As a cell therapeutic progresses from concept to product, the development of a commercial manufacturing process may contain unexpected technical and quality issues. The development path should follow several defined steps. My presentations will discuss the key steps in the process and highlight critical areas that need to be addressed to develop a successful commercial manufacturing process. PCT helps clients bridge the gap between discovery and patient care through efficient transfer of cell-based therapies from laboratory into clinical practice."

NeoStem and PCT invite you to attend the conference(s), see Dr. Fong's talks, and connect with the PCT team at PCT's booths. If you are a colleague of PCT or NeoStem, PCT can offer you a registration discount. Please contact PCT at bdm@pctcelltherapy.com for more details.

IBC Life Sciences' 2nd Annual Cell Therapy Bioprocessing Conference

Terrapinn 4th Annual Stem Cells USA and Regenerative Medicine Congress

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT") with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cells product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a patent and patent pending (IP) portfolio, we believe we are well positioned to succeed.

For more information on NeoStem, please visit http://www.neostem.com. For more information on PCT, please visit http://www.pctcelltherapy.com.

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Progenitor Cell Therapy, a NeoStem Company, Invited to Present at Two Conferences in September

Recommendation and review posted by simmons

LONDON (Reuters) - Roche Holding AG, the world's biggest maker of cancer drugs, said it would build on its drive into personalized medicine to hold onto its long-term growth momentum and said it would keep up spending on research and development.

In a statement ahead of a presentation to investors in London on Wednesday, the Swiss drugmaker said it expected 19 late stage trials to read out over the next 18 months.

"More than 60 percent of our pharmaceutical pipeline projects are coupled with the development of companion diagnostics in order to make treatments more effective," Chief Executive Severin Schwan said in a statement.

Roche said it was committed to a stable R&D budget. It spent 8.3 billion Swiss francs ($8.71 billion)on R&D in 2011. The company is also targeting growth in emerging markets as austerity-hit European markets slash healthcare budgets.

(Reporting by Caroline Copley)

Original post:
Roche’s personalized medicine push: 60% of drug pipeline includes companion diagnostic

Recommendation and review posted by sam

GAITHERSBURG, MD--(Marketwire -09/05/12)- Cytomedix, Inc. (CMXI), a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell technologies for wound and tissue repair, today announced that the Company's AutoloGel System will be highlighted in an oral abstract and a poster presentation at the 4th Congress of the World Union of Wound Healing Societies (WUWHS 2012) being held from September 2-6, in Yokohama, Japan.

The AutoloGel System is a device for the production of autologous platelet rich plasma ("PRP") gel, and is the only PRP device cleared by the U.S. Food and Drug Administration ("FDA") for use in wound management.

"The Impact of Autologous Platelet Rich Plasma (PRP) Gel on Chronic Wounds" will be presented September 5th from 3:00 to 4:30 p.m. JST as part of the Tissue Engineering and Regenerative Medicines in Wound Healing session. The poster, OR 206, will be presented by Laura Parnell, MSc, CWS, Precision Consulting on behalf of Carelyn P. Fylling, RN, MSN, CWS, CLNC, Vice President of Professional Services of Cytomedix and lead author on the poster.

Key Study Findings (all data reflects mean outcomes)

The study concluded that AutoloGel PRP Gel "initiated rapid size reduction in long-standing non-healing wounds of multiple etiologies in multiple health care sites even in patients with compromised status."

In addition, Dr. Chugo Rinoie, DPM, ABPO, CWS, Chief of Podiatric Surgery, Wound Healing Center, Methodist Hospital of Southern California, Arcadia, Calif., and Medical Director, Millennia Wound Management, Inc., Los Angeles, will present Poster 125, entitled "Healing Complex, Severe Diabetic and Ischemic Wounds in Japan Using Platelet-Rich Plasma Gel" in the Exhibit Hall as part of the Diabetic Foot, Critical Limb Ischemia and Foot Care session.

"We are honored to have these two presentations of positive data in support of the use of AutoloGel to accelerate wound healing in a variety of chronic wounds selected for presentation at WUWHS 2012, as more than 1,200 abstracts were submitted for inclusion at this prestigious international Congress," noted Martin P. Rosendale, Chief Executive Officer of Cytomedix. "The data from these studies support and validate previous studies showing that AutoloGel significantly and reliably improves the rate of healing, speed and progress to healing as compared with previous experience with standard wound care alone. The complexity and co-morbidities associated with the wounds treated in these studies would have excluded them from any randomized controlled trial, making the findings from these real-world studies even more compelling."

"We believe we will continue to generate data such as these through the comprehensive collection of evidence we are undertaking through the Centers for Medicare and Medicaid Services' Coverage with Evidence Development program. We are confident such data will continue to strongly support the ongoing coverage for autologous PRP gel for the benefit of the various stakeholders in improving clinical wound care outcomes while lowering overall costs," added Mr. Rosendale.

About the Congress of the World Union of Wound Healing SocietyThe Congress of the World Union of Wound Healing Societies is held once every four years and provides an international forum for announcement of the latest research relating to wound healing that draws between 3,500 to 5,000 clinicians, researchers and professionals who serve the wound care markets around the world. The Congress also helps to ensure the exchange of information, the improvement and development of education, international person-to-person support and the promotion of industrial collaboration. The ultimate aim is to develop the field of wound healing. Despite the fact that "wounds" are a fundamental component and important target for surgery, there are still many factors that have yet to be clarified or fully understood.

About Cytomedix, Inc. Cytomedix, Inc. is a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell separation products that enhance the body's natural healing processes. The Company's advanced autologous technologies offer clinicians a new treatment paradigm for wound and tissue repair. The Company's patient-derived PRP systems are marketed by Cytomedix in the U.S. and distributed internationally. Our commercial products include the AutoloGel System, cleared by the FDA for wound care and the Angel Whole Blood Separation System. The Company is developing novel regenerative therapies using our proprietary ALDH Bright Cell ("ALDHbr") technology to isolate a unique, biologically active population of a patient's own stem cells. A Phase 2 trial evaluating the use of ALDHbr for the treatment of ischemic stroke is underway. For additional information please visit http://www.cytomedix.com.

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Cytomedix's AutoloGel System Highlighted in Two Presentations at the 4th Congress of the World Union of Wound Healing ...

Recommendation and review posted by sam

SAN DIEGO, September 5, 2012 /PRNewswire/ --

Shire plc (LSE: SHP, NASDAQ: SHPG), today announced that its lead regenerative medicine product, DERMAGRAFT (human fibroblast-derived dermal substitute) has received regulatory approval from Health Canada as a class IV medical device for the treatment of diabetic foot ulcers (DFUs), a complication of diabetes.

This approval gives Shire Regenerative Medicine the potential to extend availability of DERMAGRAFT to people with DFUs beyond the U.S., where it is currently available, and is an important first step for the company as it continues to develop its international expansion strategy.

"We're thrilled to have the opportunity to bring DERMAGRAFT to Canada, as we believe it plays an important role in the treatment of DFUs," said Matt Pauls, Vice President of Global Commercial Operations for Shire Regenerative Medicine. "This approval represents an important milestone in our strategic growth plan to build a global business that provides regenerative medicine solutions for people around the world with life-altering conditions."

This announcement follows Shire's recent commitment to build a new regenerative medicine campus in San Diego to gain increased capacity to meet future demand for DERMAGRAFT, as well as additional space and infrastructure to develop and manufacture new regenerative medicine products.

Shire Regenerative Medicine intends to make DERMAGRAFT available in Canada in Q1 2013, and will leverage Shire's current infrastructure and commercial knowledge of the Canadian healthcare system, where the total population with diabetes was estimated to be 2.7 million people (7.6% of the total Canadian population) in 2010, and is projected to rise to 4.2 million people (10.8% of the total Canadian population) by 2020[i]. In 2008, the Canadian Association of Wound Care estimated that 345,000 people with diabetes will develop a DFU in their lifetime and that DFUs were costing Canada's healthcare system more than $150 million annually.[ii]

Health Canada's approval of DERMAGRAFT on August 21, 2012 is based on a Canadian Device License Application submitted by Shire Regenerative Medicine in 2011.

About DFUs

DFUs are chronic sores that can develop on the feet of people with diabetes. Among people with diabetes, up to 25% experience a DFU in their lifetime,[iii,iv] and approximately 2% develop a DFU each year.[v] If not properly treated, DFUs may result in serious complications.

About DERMAGRAFT

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DERMAGRAFT® Approved in Canada for Treatment of Diabetic Foot Ulcers

Recommendation and review posted by sam

LOS ANGELES UCLA researchers have discovered a type of cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.

The research was done using human bone marrow, which contains all the stem cells that produce blood during post-natal life.

"We felt it was especially important to do these studies using human bone marrow, as most research into the development of the immune system has used mouse bone marrow," said the study's senior author, Dr. Gay Crooks, co-director of UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and a co-director of the cancer and stem-cell biology program at UCLA's Jonsson Comprehensive Cancer Center. "The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of post-natal life."

The research team was "intrigued to find this particular bone marrow cell, because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life," said Crooks, a professor of pathology and pediatrics.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, cancers of the blood.

The findings appeared Sept. 2 in the early online edition of the journal Nature Immunology.

Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce cells in intermediate stages of development called progenitors, which make various blood lineages, like red blood cells or platelets.

Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.

"Like the stem cells, the progenitor cells are also very rare, so before we can study them, we needed to find the needle in the haystack," said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author of the study.

Previous work had found a fairly mature type of lymphocyte progenitor with a limited ability to differentiate, but the new work describes a more primitive type of progenitor primed to produce the entire immune system, Kohn said.

Excerpt from:
'Missing link' ties blood stem cells, immune system

Recommendation and review posted by Bethany Smith