StemCell Therapy

Public release date: 25-Jun-2012 [ | E-mail | Share ]

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

New Rochelle, NY, June 25, 2012Gene therapy to replace the protein missing in Pompe disease can be effective if the patient's immune system does not react against the therapy. Targeted delivery of the gene to the liver, instead of throughout the body,suppresses the immune response, improving the therapeutic effect, according to an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. The article is available free online at the Human Gene Therapy website.

"The current unmet medical need in Pompe disease is for prevention of immune responses against standard-of-care enzyme replacement therapy," says coauthor Dwight Koeberl, MD, PhD. "However, we foresee a future application of the dual vector strategy described in this paper, including a liver-expressing vector along with a ubiquitously expressing vector, which might achieve much higher efficacy than either vector alone."

In the article "Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease," Ping Zhang and coauthors from Duke University Medical Center (Durham, NC), targeted a gene delivery vector carrying the therapeutic gene to the livers of mice with Pompe disease. Not only did the liver-specific expression of the protein induce immune tolerance, but when combined with non-targeted delivery of the therapeutic gene it also boosted the overall effectiveness of the treatment.

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About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of content and a free sample issue may be viewed online at the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc. is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at the Mary Ann Liebert, Inc. website.

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Targeted gene therapy enhances treatment for Pompe disease

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/skdhnn/translational_rege) has announced the addition of the "Translational Regenerative Medicine - Oncology, CNS and Cardiovascular-Rich Pipeline Features Innovative Stem Cell and Gene Therapy Applications" report to their offering.

More Guidelines Needed to Grow Regenerative Medicine Market, Report Finds

Standardized research guidelines are needed to control and encourage the development of gene therapy and stem cell treatments, according to a new report by healthcare experts GBI Research.

The new report* shows how regenerative medicine is seen as an area with high future potential, as countries need ways to cope with the burden of an aging population.

The stem cell market alone is predicted to grow to around $5.1 billion by 2014, while gene therapy has also shown promise despite poor understanding of some areas of regenerative medicine and a lack of major approvals (the only approvals to date being made in Asia).

Up until now, securing research within clinics has been difficult, with a high number of failures and discontinuations throughout all phases of clinical study. Stem cell therapy uses bone marrow transplants as an established treatment method, but the development of the therapy into further applications and has not yet become common practice.

Similarly, tissue engineering has been successful in the areas of skin and bone grafts, but translation into more complex therapies has been an issue for researchers. Although scientific possibilities are ever-increasing, the true potential of regenerative medicine has yet to be demonstrated fully.

A desire to discover new and innovative technologies has encouraged governments in the UK and Singapore to focus directly on regenerative medicine as a future potential economy booster.

Companies Mentioned:

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Research and Markets: Translational Regenerative Medicine - Oncology, CNS and Cardiovascular-Rich Pipeline Features ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patients own CD34+ hematopoietic stem cells transduced with bluebird bios lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.

Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families, said David Davidson, M.D., chief medical officer of bluebird bio. We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients.

Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.

About ALD

Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological disorder that, in its most severe form, causes damage to the myelin sheath (an insulating layer of membranes that surrounds nerve cells in the brain) and progressive dysfunction of the adrenal glands. Also known as Lorenzo's Oil disease, ALD is estimated to affect one in every 21,000 boys worldwide. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 4 and 10. Boys afflicted with this form of ALD develop normally until the onset of symptoms. The symptoms of this disorder often progress rapidly and, in a matter of years, can lead to a vegetative state and, ultimately, death. Current treatment options are limited to allogeneic stem cell transplantation when there is an appropriate donor. Allogeneic transplants carry a significant risk of serious morbidity and death.

About bluebird bio's CCALD Product Development

bluebird bios CCALD product program has the potential to halt the progression of CCALD by providing a functional ABCD1 gene to the patients own stem cells. These stem cells proliferate, and some of the progeny cells travel to the brain where they become microglial cells incorporating the corrective gene. Data from the first clinical study treating X-linked CCALD patients with the companys lentiviral gene therapy product demonstrated continued stable expression of the transgene and the corresponding ABCD-1 protein for over four years in two CCALD patients, resulting in prolonged disease stabilization. bluebird bio plans to initiate a Phase 2/3 clinical study in CCALD in both the United States and Europe in 2013.

About bluebird bio

bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bios product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The companys novel approach uses stem cells harvested from the patients own bone marrow into which a healthy version of the disease causing gene is inserted. bluebird bios approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and presenting a one-time potentially transformative therapy using a patients own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. Led by a world-class team, bluebird bio is privately held and backed by top-tier life sciences investors, including Third Rock Ventures, TVM Capital, ARCH Venture Partners, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its operations are located in Cambridge, Mass. and Paris, France. For more information, please visit http://www.bluebirdbio.com.

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bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy

Public release date: 18-Jun-2012 [ | E-mail | Share ]

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

New Rochelle, NY, June 18, 2012A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

"This is a very novel approach for addressing the huge medical problem of cocaine addiction," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

In the article "AAVrh.10-Mediated Expression of an Anti-Cocaine Antibody Mediates Persistent Passive Immunization That Suppresses Cocaine-Induced Behavior," (http://online.liebertpub.com/doi/pdfplus/10.1089/hum.2011.178) a team of researchers from Weill Cornell Medical College (New York, NY), The Scripps Research Institute (La Jolla, CA), and Cornell University (Ithaca, NY) used a virus-based delivery vehicle in mice to transfer a gene that produces a protein capable of binding to cocaine present in the blood, preventing the cocaine from crossing into the brain. The protein is a monoclonal antibody that sequesters cocaine, making the vaccinated mice resistant to the drug's effects. Whereas unvaccinated mice exhibited hyperactivity when exposed to intravenous cocaine, the immunized mice showed no effects, according to authors Jonathan Rosenberg, et al.

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About the Journal

Human Gene Therapy (http://www.liebertpub.com/hum), the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of contents and a free sample issue may be viewed online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

About the Publisher

Mary Ann Liebert, Inc. (http://www.liebertpub.com) is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at the Mary Ann Liebert, Inc. website (http://www.liebertpub.com).

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Anti-cocaine vaccine described in Human Gene Therapy Journal

Editor's Note: Read our blog series on psychiatry's new rulebook, the DSM-5.

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Editor's Note: Read our blog series on psychiatry's new rulebook, the DSM-5.

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http://rss.sciam.com/sciam/topic/gene-therapy

By Ewen Callaway of Nature magazine

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The Advances news section in April's issue of Scientific American included stories on digital textbooks, the promise of using gene therapy to fight blindness and how fragile orchids survive. To learn more about any of the stories, follow these links.

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By Ewen Callaway of Nature magazine

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Public release date: 2-May-2012 [ | E-mail | Share ]

Contact: Holly Auer holly.auer@uphs.upenn.edu 215-200-2313 University of Pennsylvania School of Medicine

PHILADELPHIA -- HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

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Genetically modified T cell therapy shown to be safe, lasting in decade-long study of HIV patients

A little more than three years ago a medical team from Berlin published the results of a unique experiment that astonished HIV researchers. The German group had taken bone marrow--the source of the body’s immune cells--from an anonymous donor whose genetic inheritance made him or her naturally resistant to HIV. Then the researchers transplanted the cells into a man with leukemia who had been HIV-positive for more than 10 years. Although treatment of the patient’s leukemia was the rationale for the bone marrow transplant therapy, the group also hoped that the transplant would provide enough HIV-resistant cells to control the man’s infection. The therapy exceeded the team’s expectations. Instead of just decreasing the amount of HIV in the patient’s blood, the transplant wiped out all detectable traces of the virus from his body, including in multiple tissues where it could have lain dormant. The German researchers were so surprised by the spectacularly positive results that they waited nearly two years before publishing their data.

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Antidepressants restore well-being to many people, but sometimes at the cost of such side effects as weight gain or loss of interest in sex. And these side effects can be just part of the frustration. As Robin Marantz Henig wrote in " Lifting the Black Cloud ," in the March issue of Scientific American , the drugs that have long dominated the market--the selective serotonin reuptake inhibitors (SSRIs) and the serotonin and norepinephrine reuptake inhibitors (SNRIs)--"do not help everyone and eventually fail in more than a third of users. A pill that seems to be working today might well stop helping tomorrow. And the drugs can take several weeks to start having a marked effect." Equally disturbing, some major pharmaceutical houses, such as GlaxoSmithKline , are pulling back from developing psychiatric medicines.

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A young woman who calls herself blue­berryoctopus had been taking anti­depressants for three years, mostly for anxiety and panic attacks, when she recounted her struggles with them on the Web site Experience Project. She said she had spent a year on Paxil, one of the popular SSRIs (selective serotonin reuptake inhibitors), but finally stopped because it destroyed her sex drive. She switched to Xanax, an ­antianxiety drug , which brought back her libido but at the cost of renewed symptoms. Then Paxil again, then Lexapro (another SSRI), then Pristiq, a member of a related class of antidepressants, the SNRIs (serotonin and norepinephrine reuptake inhibitors). At the time of the post, she was on yet another SSRI, Zoloft, plus Wellbutrin (a cousin of SNRIs that affects the activity of dopamine as well as norepinephrine), which was intended to counteract the sexual side effects of Zoloft. “I don’t notice much of a difference with the Wellbutrin, but I’m on the lowest dose now,” she wrote. “I’m going back to my psychiatrist next week, so maybe he’ll up it. Who knows.”

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http://rss.sciam.com/sciam/topic/gene-therapy

A little more than three years ago a medical team from Berlin published the results of a unique experiment that astonished HIV researchers. The German group had taken bone marrow--the source of the body’s immune cells--from an anonymous donor whose genetic inheritance made him or her naturally resistant to HIV. Then the researchers transplanted the cells into a man with leukemia who had been HIV-positive for more than 10 years. Although treatment of the patient’s leukemia was the rationale for the bone marrow transplant therapy, the group also hoped that the transplant would provide enough HIV-resistant cells to control the man’s infection. The therapy exceeded the team’s expectations. Instead of just decreasing the amount of HIV in the patient’s blood, the transplant wiped out all detectable traces of the virus from his body, including in multiple tissues where it could have lain dormant. The German researchers were so surprised by the spectacularly positive results that they waited nearly two years before publishing their data.

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

Antidepressants restore well-being to many people, but sometimes at the cost of such side effects as weight gain or loss of interest in sex. And these side effects can be just part of the frustration. As Robin Marantz Henig wrote in " Lifting the Black Cloud ," in the March issue of Scientific American , the drugs that have long dominated the market--the selective serotonin reuptake inhibitors (SSRIs) and the serotonin and norepinephrine reuptake inhibitors (SNRIs)--"do not help everyone and eventually fail in more than a third of users. A pill that seems to be working today might well stop helping tomorrow. And the drugs can take several weeks to start having a marked effect." Equally disturbing, some major pharmaceutical houses, such as GlaxoSmithKline , are pulling back from developing psychiatric medicines.

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

A young woman who calls herself blue­berryoctopus had been taking anti­depressants for three years, mostly for anxiety and panic attacks, when she recounted her struggles with them on the Web site Experience Project. She said she had spent a year on Paxil, one of the popular SSRIs (selective serotonin reuptake inhibitors), but finally stopped because it destroyed her sex drive. She switched to Xanax, an ­antianxiety drug , which brought back her libido but at the cost of renewed symptoms. Then Paxil again, then Lexapro (another SSRI), then Pristiq, a member of a related class of antidepressants, the SNRIs (serotonin and norepinephrine reuptake inhibitors). At the time of the post, she was on yet another SSRI, Zoloft, plus Wellbutrin (a cousin of SNRIs that affects the activity of dopamine as well as norepinephrine), which was intended to counteract the sexual side effects of Zoloft. “I don’t notice much of a difference with the Wellbutrin, but I’m on the lowest dose now,” she wrote. “I’m going back to my psychiatrist next week, so maybe he’ll up it. Who knows.”

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

ScienceDaily (Mar. 27, 2012) A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

This study's findings, published in PLoS ONE, represents a new approach to treating these related, and serious, red blood cells disorders, say the investigators.

"This gene therapy technique has the potential to cure many patients, especially if we prescreen them to predict their response using just a few of their cells in a test tube," says the study's lead investigator, Dr. Stefano Rivella, Ph.D., an associate professor of genetic medicine at Weill Cornell Medical College. He led a team of 17 researchers in three countries.

Dr. Rivella says this is the first time investigators have been able to correlate the outcome of transferring a healthy beta-globin gene into diseased cells with increased production of normal hemoglobin -- which has long been a barrier to effective treatment of these disease.

So far, only one patient in France has been treated with gene therapy for beta thalassemia, and Dr. Rivella and his colleagues believe the new treatment they developed will be a significant improvement. No known patient has received gene therapy yet to treat sickle cell anemia.

A Fresh Approach to Gene Therapy

Beta-thalassemia is an inherited disease caused by defects in the beta-globin gene. This gene produces an essential part of the hemoglobin protein, which, in the form of red blood cells, carries life-sustaining oxygen throughout the body.

The new gene transfer technique developed by Dr. Rivella and his colleagues ensures that the beta-globin gene that is delivered will be active, and that it will also provide more curative beta-globin protein. "Since the defect in thalassemia is lack of production of beta-globin protein in red blood cells, this is very important," Dr. Rivella says.

The researchers achieved this advance by hooking an "ankyrin insulator" to the beta-globin gene that is carried by a lentivirus vector. During the gene transfer, this vector would be inserted into bone marrow stem cells taken from patients, and then delivered back via a bone marrow transplant. The stem cells would then produce healthy beta-globin protein and hemoglobin.

This ankyrin insulator achieves two goals. First, it protects delivery of the normal beta-globin gene. "In many gene therapy applications, a curative gene is introduced into the cells of patients in an indiscriminate fashion," Dr. Rivella explains. "The gene lands randomly in the genome of the patient, but where it lands is very important because not all regions of the genome are the same." For example, some therapeutic genes may land in an area of the genome that is normally silenced -- meaning the genes in this area are not expressed. "The role of ankyrin insulator is to create an active area in the genome where the new gene can work efficiently no matter where it lands," Dr. Rivella says. He adds that the small insulator used in his vector should eliminate the kind of side effects seen in the French patient treated with beta-thalassemia gene therapy.

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New gene therapy approach developed for red blood cell disorders

SILVER SPRING, Md.--(BUSINESS WIRE)--

Nuvilex, Inc. (OTCQB:NVLX), an emerging biotechnology provider of cell and gene therapy solutions through its acquisition of the SG Austria assets, today discussed the value of encapsulation, freezing, storage, survivability and localization of human stem cells once implanted using the proprietary Cell-in-a-Box technology.

The encapsulation of human stem cells is enabled by the Cell-in-a-Box technology, which can then be frozen, stored and later implanted into target tissues. The benefits of encapsulation are several: first, the process allows for freezing of stem cells for long-term storage without appreciably affecting viability. Second, encapsulation protects the stem cells from stress factors caused by direct aeration and sheer forces associated with bioreactors. Third, Cell-in-a-Box encapsulated stem cells are held in place at the site of implantation, maximizing their potential efficacy as they have the potential to stimulate growth of surrounding new, healthy tissue. Finally, encapsulated cells may prevent any potential side effects associated with direct injection since they remain localized to the area of treatment when encapsulated.

Dr. Robert Ryan, Chief Executive Officer of Nuvilex, commented, For many years it was assumed stem cells existed only to replace cells that had died or were damaged. Recent studies suggest factors stem cells secrete provide signals to surrounding tissue that can stimulate regeneration. The potential therefore, is that if stem cells can be maintained at a particular site where damaged, removed or non-functional tissue was through some sort of holding mechanism, this may aid in a positive growth response in that tissue. In addition, the stem cells themselves have the potential to undergo development into the appropriate cell type at that location, potentially creating miniature organs. The Cell-in-a-Box technology is designed specifically for those purposes. Thus, encapsulated stem cells would be implanted and remain in place, ultimately being able to serve a broad number of medical applications entirely dependent on where in the body they are placed.

About Nuvilex

Nuvilex, Inc. (OTCQB:NVLX) is an emerging international biotechnology provider of live clinically useful, therapeutically valuable, encapsulated cells, as well as services for encapsulating live cells for the research and medical communities. Through substantial effort, the aspects of our corporate activities alone and in concert with SG Austria continue to move toward agreement completion and ultimately a strong future together. Our companys ultimate clinical offerings will include cancer, diabetes and other treatments using the companys industry-leading cell and gene therapy expertise and cutting edge, live-cell encapsulation technology.

Safe Harbor Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 involving risks and uncertainties, including product demand, market competition, and Nuvilexs ability to meet 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, to reflect events or circumstances afterward, or to disclose unanticipated occurrences, except as required under applicable laws.

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Cell-in-a-Box® Encapsulation Technology Creates Extensive Applications within the Stem Cell Arena

London, March 19 (ANI): Scientists have now found answer to why some patients fail to respond to some of the most successful cancer drugs.

Tyrosine kinase inhibitor drugs (TKIs) work effectively in most patients to fight certain blood cell cancers, such as chronic myelogenous leukemia (CML), and non-small-cell lung cancers (NSCLC) with mutations in the EGFR gene.

These precisely targeted drugs shut down molecular pathways that keep these cancers flourishing and include TKIs for treating CML, and the form of NSCLC with EGFR genetic mutations.

Now, a multi-national research team led by scientists at Duke-NUS Graduate Medical School in Singapore, working with the Genome Institute of Singapore (GIS), Singapore General Hospital and the National Cancer Centre Singapore, has discovered that there is a common variation in the BIM gene in people of East Asian descent that contributes to some patients' failure to benefit from these tyrosine kinase inhibitor drugs.

"Because we could determine in cells how the BIM gene variant caused TKI resistance, we were able to devise a strategy to overcome it," said S. Tiong Ong, M.B.B. Ch., senior author of the study and associate professor in the Cancer and Stem Cell Biology Signature Research Programme at Duke-NUS and Division of Medical Oncology, Department of Medicine, at Duke University Medical Center.

"A novel class of drugs called the BH3-mimetics provided the answer," he said.

"When the BH3 drugs were added to the TKI therapy in experiments conducted on cancer cells with the BIM gene variant, we were able to overcome the resistance conferred by the gene. Our next step will be to bring this to clinical trials with patients," Ong added.

Yijun Ruan, Ph.D., a co-senior author of this study and associate director for Genome Technology and Biology at GIS said: "We used a genome-wide sequencing approach to specifically look for structural changes in the DNA of patient samples. This helped in the discovery of the East Asian BIM gene variant. What's more gratifying is that this collaboration validates the use of basic genomic technology to make clinically important discoveries."

If the drug combination does override TKI resistance in people, this will be good news for those with the BIM gene variant, which occurs in about 15 percent of the typical East Asian population. By contrast, no people of European or African ancestry were found to have this gene variant.

"While it's interesting to learn about this ethnic difference for the mutation, the greater significance of the finding is that the same principle may apply for other populations," said Patrick Casey, Ph.D., senior vice dean for research at Duke-NUS and James B. Duke Professor of Pharmacology and Cancer Biology.

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Gene variant in East Asians could explain resistance to cancer drugs

Public release date: 6-Mar-2012 [ | E-mail | Share ]

Contact: Cody Mooneyhan cmooneyhan@faseb.org 301-634-7104 Federation of American Societies for Experimental Biology

Bethesda, MDA new research discovery by a team of Stanford and European scientists offers hope that people with atherosclerotic disease may one day be able to avoid limb amputation related to ischemia. A new research report appearing online in the FASEB Journal suggests that the delivery of genes for two molecules naturally produced by the body, called "PDGF-BB" and "VEGF" may successfully cause the body to grow new blood vessels that can save ischemic limbs.

"We hope that our findings will ultimately develop into a safe and effective therapy for the many patients, suffering from blocked arteries in the limbs, who are currently not adequately treated by surgery or drugs," said Helen M. Blau, Ph.D., a senior researcher involved in the work and Associate Editor of the FASEB Journal from the Baxter Laboratory for Stem Cell Biology at the Institute for Regenerative Medicine and Stem Cell Biology at Stanford. "This could help avoid the devastating consequences of limb amputations for both patients and their families."

To make this discovery, Blau and colleagues, including Andrea Banfi (now at Basel University), introduced the genes for PDGF-BB and VEGF into the muscles of mice, either independently or together. When high doses of VEGF alone were produced, they caused the growth of vascular tumors. When the two factors were produced in unbalanced amounts, tumor growth also occurred. When VEGF and PDGF were delivered in a fixed ratio relative to one another, however, no tumors occurred, and blood flow was restored to ischemic muscle tissue and damage repaired without any toxic effects. To achieve a "balanced" delivery of PDGF-BB and VEGF, scientists placed both genes in a single gene therapy delivery mechanism, called a "vector."

Although the report shows the feasibility of growing robust and safe new blood vessels that restore blood flow to diseased tissues, Blau points out that "there are multiple challenges to correcting peripheral vasculature disease by using proangiogenic gene therapy strategies. Two important challenges are what to deliver and how to get it to where it can have beneficial effects. Clinical success will require both delivering a gene therapy construct that encodes for effective angiogenic factors and ensuring that the sites of delivery are where the construct can have the greatest clinical benefit."

"This ingenious work, based on the latest techniques of molecular biology, tells us that it is possible to reinvigorate parts of our body that can't get enough blood to keep them going," said Gerald Weissmann, M.D., Editor-in-Chief of the FASEB Journal. "The next question is whether this approach will work in humans and exactly how to deliver the new treatment to places that need it the most."

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Receive monthly highlights from the FASEB Journal by e-mail. Sign up at http://www.faseb.org/fjupdate.aspx. The FASEB Journal is published by the Federation of the American Societies for Experimental Biology (FASEB) and is the most cited biology journal worldwide according to the Institute for Scientific Information. In 2010, the journal was recognized by the Special Libraries Association as one of the top 100 most influential biomedical journals of the past century. FASEB is composed of 26 societies with more than 100,000 members, making it the largest coalition of biomedical research associations in the United States. Celebrating 100 Years of Advancing the Life Sciences in 2012, FASEB is rededicating its efforts to advance health and well-being by promoting progress and education in biological and biomedical sciences through service to our member societies and collaborative advocacy.

Details: Andrea Banfi, Georges von Degenfeld, Roberto Gianni-Barrera, Silvia Reginato, Milton J. Merchant, Donald M. McDonald, and Helen M. Blau. Therapeutic angiogenesis due to balanced single-vector delivery of VEGF and PDGF-BB. FASEB J. doi:10.1096/fj.11-197400 ; http://www.fasebj.org/content/early/2012/03/05/fj.11-197400.abstract

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Stanford scientists develop gene therapy approach to grow blood vessels in ischemic limbs