Archive for May, 2012

ScienceDaily (May 21, 2012) A substance in human mesenchymal stem cells that promotes growth appears to spur restoration of nerves and their function in rodent models of multiple sclerosis (MS), researchers at Case Western Reserve University School of Medicine have found.

In animals injected with hepatocyte growth factor, inflammation declined and neural cells grew. Perhaps most important, the myelin sheath, which protects nerves and their ability to gather and send information, regrew, covering lesions caused by the disease.

"The importance of this work is we think we've identified the driver of the recovery," said Robert H. Miller, professor of neurosciences at the School of Medicine and vice president for research at Case Western Reserve University.

Miller, neurosciences instructor Lianhua Bai and biology professor Arnold I. Caplan, designed the study. They worked with Project Manager Anne DeChant, and research assistants Jordan Hecker, Janet Kranso and Anita Zaremba, from the School of Medicine; and Donald P. Lennon, a research assistant from the university's Skeletal Research Center.

In MS, the immune system attacks myelin, risking injury to exposed nerves' intricate wiring. When damaged, nerve signals can be interrupted, causing loss of balance and coordination, cognitive ability and other functions. Over time, intermittent losses may become permanent.

Miller and Caplan reported in 2009 that when they injected human mesenchymal stem cells into rodent models of MS, the animals recovered from the damage wrought by the disease. Based on their work, a clinical trial is underway in which MS patients are injected with their own stem cells.

In this study, the researchers first wanted to test whether the presence of stem cells or something cells produce promotes recovery. They injected mice with the medium in which mesenchymal stem cells, culled from bone marrow, grew.

All 11 animals, which have a version of MS, showed a rapid reduction in functional deficits.

Analysis showed that the disease remained on course unless the molecules injected were of a certain size; that is, the molecular weight ranged between 50 and 100 kiloDaltons. Research by others and results of their own work indicated hepatocyte growth factor, which is secreted by mesenchymal stem cells, was a likely instigator.

The scientists injected animals with 50 or 100 nanograms of the growth factor every other day for five days. The level of signaling molecules that promote inflammation decreased while the level of signaling molecules that counter inflammation increased. Neural cells grew and nerves laid bare by MS were rewrapped with myelin. The 100-nanogram injections appeared to provide slightly better recovery.

Read the original:
Growth factor in stem cells may spur recovery from multiple sclerosis

ScienceDaily (May 21, 2012) Tissue engineers can use mesenchymal stem cells derived from fat to make cartilage, bone, or more fat. The best cells to use are ones that are already likely to become the desired tissue. Brown University researchers have discovered that the mechanical properties of the stem cells can foretell what they will become, leading to a potential method of concentrating them for use in healing.

To become better healers, tissue engineers need a timely and reliable way to obtain enough raw materials: cells that either already are or can become the tissue they need to build. In a new study, Brown University biomedical engineers show that the stiffness, viscosity, and other mechanical properties of adult stem cells derived from fat, such as liposuction waste, can predict whether they will turn into bone, cartilage, or fat.

That insight could lead to a filter capable of extracting the needed cells from a larger and more diverse tissue sample, said Eric Darling, senior author of the paper published in Proceedings of the National Academy of Sciences. Imagine a surgeon using such a filter to first extract fat from a patient with a bone injury and then to inject a high concentration of bone-making stem cells into the wound site during the same operation.

If mechanical properties of stem cells -- viscosity, stiffness, size -- predict what they will become, the next step is to develop a high-throughput testing and sorting device.In the paper, the researchers report that the stiffest adipose-derived mesenchymal stem cells tended to become bone, the ones that were biggest and softest tended to become fat, and those that were particularly viscous were most likely to end up as cartilage.

"The results are exciting because not only do the mechanical properties indicate what lineage these cells could potentially go along but also the extent of their differentiation," said Darling, assistant professor of medical science in the Department of Molecular Pharmacology, Physiology. and Biotechnology and the University's Center for Biomedical Engineering. "It tells us how good they are going to be if we differentiated them for a given tissue type."

So when tissue engineers go looking through extracted fat for cells to create bone, for instance, they can sort through the cells looking for ones with a certain level of stiffness or greater. Whether the cells are "undifferentiated" stem cells that have made no move toward becoming a specific cell type, or ones that are already bone cells, only the ones with the requisite stiffness would make the cut. That process would yield a higher population of cells for making new bone tissue.

"Can we enrich the cell populations for cells that we want to use, whether they are totally undifferentiated cell types, partially differentiated, or completely differentiated?" Darling said. "It doesn't matter as long as it's targeted for the specific tissue application."

Darling's study, led by research assistants Rafael Gonzaelz-Cruz and Vera Fonseca, involved cloning adipose-derived adult human stem cells into 32 stem cell populations. They then poked, prodded, and measured the cells with an atomic force microscope, gaining measurements of how big they were, how sturdy they were under pressure, and how the force between them and the scope's cantilevered probe changed over time. The team found the cells exhibited a wide range of stiffness, viscosity and size.

Once they had the measurements, the researchers chemically induced the cells to differentiate and analyzed the levels of key metabolites produced by the cells as they matured a few weeks later. For each population, the metabolites indicated the relative proportion that differentiated into one tissue or another. Population 28, for example, apparently responded productively to chemical cues for producing cartilage, only somewhat well for producing bone and poorly to cues for making fat.

The key moment was when the researchers correlated each cell population's mechanical measurements with its metabolite data. Did the mechanical properties predict which populations would be the most successful in turning into bone cells or cartilage cells or fat cells? Sure enough, they did. The stiffest cell populations produced more bone. The squishiest cells were the ones that produced the most fat. The ones with the highest viscosity were the ones seemingly headed toward becoming cartilage.

Read this article:
Physical properties predict stem cell outcome

NEW DELHI (CNN) Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesnt have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash theyve journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash who presents symptoms similar to Down Syndrome in 2010. I am helping improve their quality of life, Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

Thats when Cash began walking with the aid of braces for the first time.

His parents were thrilled. Before the treatments, Cash could only get around by hopping, his mother said. The results were enough to persuade Cashs mother to go back to Shroff for more help.

We saw evidence the first time that its worth trying again, Krolick said. In this particular case, with Cashs other conditions, we dont have many other options.

For four or five weeks of treatment, Shroff says she has charged her 87 American patients an average of $25,000. Its a big financial hit for Burnaman, a volunteer firefighter and property manager, and Krolick, who attends technical college in Greenville, South Carolina.

More here:
Parents Cross Globe to Try Unproven Treatment on Son

Indian clinic's stem cell therapy real?

STORY HIGHLIGHTS

For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.

New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

That's when Cash began walking with the aid of braces for the first time.

Visit link:
Family hangs hope on stem cells

THE WOODLANDS, Texas--(BUSINESS WIRE)--

Opexa Therapeutics, Inc. (NASDAQ:OPXA - News), a biotechnology company developing a novel T-cell therapy for multiple sclerosis (MS), announced today that the Company is rebranding its leading MS therapy with the new name TcelnaTM. The product, previously known as Tovaxin, will now be known as Tcelna as the company positions itself towards the treatment of patients with Secondary Progressive MS (SPMS).

"Opexa has worked diligently in the optimization of its overall manufacturing process and clinical development program while concentrating its efforts in the SPMS indication. The rebranding of our lead product as Tcelna encompasses these advancements and our continued dedication to make a difference in the treatment of MS," commented Neil K. Warma, President and Chief Executive Officer of Opexa.

The name Tcelna (pronounced Te-SELL-nuh) reflects the T-cell derivation of the product. Opexa has requested a registered trademark for the new brand name.

About Opexa

Opexa Therapeutics, Inc. is dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS). The Companys leading therapy, TcelnaTM, a personalized cellular immunotherapy treatment, is in clinical development targeting both Secondary Progressive and Relapsing Remitting MS. Tcelna is derived from T-cells isolated from peripheral blood, expanded ex vivo and reintroduced into the patients via subcutaneous injections. This process triggers a potent immune response against specific subsets of autoreactive T-cells known to attack myelin and, thereby, reduces the risk of relapse over time.

For more information, visit the Companys website at http://www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words expects, believes, anticipates, estimates, may, could, intends, and similar expressions are intended to identify forward-looking statements. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding the development of the Companys product candidate, Tcelna, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: our capital position, the ability of the Company to enter into and benefit from a partnering arrangement for the Company's product candidate, Tcelna, on reasonably satisfactory terms (if at all), our dependence (if partnered) on the resources and abilities of any partner for the further development of Tcelna, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs and to undertake and complete any further clinical studies for Tcelna, the success of our clinical trials, the efficacy of Tcelna for any particular indication, such as Relapsing Remitting MS or Secondary Progressive MS, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights (including for Tcelna), the risk of litigation regarding our intellectual property rights, the success of third party development and commercialization efforts with respect to products covered by intellectual property rights that the Company may license or transfer, our limited manufacturing capabilities, our dependence on third-party manufacturers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date made. We assume no obligation or undertaking to update any forward-looking statements to reflect any changes in expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based. You should, however, review additional disclosures we make in our reports filed with the Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2011.

Continue reading here:
Opexa Therapeutics Announces TcelnaTM as New Brand Name for MS Therapy

DEERFIELD, Ill.--(BUSINESS WIRE)--

CHDI Foundation, Inc. and Lundbeck today announced a research collaboration to investigate a targeted therapy for Huntingtons disease (HD). Currently, no treatment exists to slow or halt the progression of HD,1 a challenging hereditary neurodegenerative disease characterized by a triad of behavioral, cognitive, and motor symptoms.2

As part of the collaboration CHDI will conduct pre-clinical studies on a Lundbeck investigative compound. Research will focus on the compounds effect on P2X receptors that may be involved in HD.3 The study results will influence future research into this and other compounds for HD.

One of our important functions at CHDI is to seek out promising research ideas in the HD field, and this includes this interesting new work on Lundbecks compound. Lundbeck is well established in central nervous system drug research and development, and were looking forward to tapping their expertise, noted Ignacio Munoz-Sanjuan, Vice President, Translational Biology at CHDI. Lundbeck has a proven track record of not only bringing new therapies to market but also working to support the needs of their patient communities. We hope this research collaboration provides a stepping stone for future therapies that slow the progression of HD.

CHDI is a privately-funded, not-for-profit biomedical research organization that works with an international network of scientists to discover and develop therapies for HD. The organization actively enables HD research by collaborating with research organizations and pharmaceutical companies conducting promising research, often providing financial support. CHDI activities include exploratory biology, clinical studies and trials, and educational workshops. Cooperation is key to finding therapies for HD, which is why CHDI works with a variety of researchers within the HD research community.

We look forward to working with this distinguished group of scientists who share our dedication to the HD community, said Staffan Schberg, president of Lundbeck. CHDI is a beacon of hope for the HD community and devotes itself entirely to finding therapies that have the potential to improve the lives of HD families. Given our shared commitment, we are thrilled to partner with them and hope that our research will lead to advancements in HD therapeutic options. We are also pleased that this announcement coincides with HD Awareness Month to help draw attention to the need to find therapies for this degenerative neurological disease.

This collaboration is part of Lundbecks continued commitment to its HD Research Initiative, launched in 2010 to identify and ultimately commercialize therapies that may slow or halt the progression of the disease. The initiative is driven by collaborations with academic institutions, research organizations and companies that share Lundbecks ongoing commitment to the HD community. In 2011, Lundbeck and the University of Massachusetts Medical School began to investigate RNAi-based therapies to suppress the production of mutant huntingtin (mHtt), the abnormal protein that causes HD. Those conducting early-stage HD research and interested in exploring opportunities to collaborate with Lundbeck should send an email to HDresearch@lundbeck.com.

About Huntingtons Disease

Huntingtons disease is a hereditary neurodegenerative disease characterized by a triad of progressive behavioral, cognitive, and motor symptoms2 that vary from person to person. The survival time after the onset of symptoms can range from 10 to 30 years.1 The HD gene, whose mutation results in the disease, was localized in 1983 and isolated in 1993.4,5 For more information on HD, please visit HDBuzz (hdbuzz.net) or the Hereditary Disease Foundation (www.hdfoundation.org).

About CHDI Foundation, Inc.

The rest is here:
Lundbeck and CHDI Foundation Announce Research Collaboration to Investigate Candidate Therapy for Huntington’s Disease

20-05-2012 13:17 WATCH THE LATEST VIDEO THAT CAME OUT TODAY HERE FAIR USE NOTICE: These Videos may contain copyrighted (©) material the use of which has not always been specifically authorized by the copyright owner. Such material is made available to advance understanding of ecological, political, human rights, economic, democracy, scientific, moral, ethical, and social justice issues, etc. It is believed that this constitutes a 'fair use' of any such copyrighted material as provided for in section 107 of the US Copyright Law. In accordance with Title 17 USC Section 107, this material is distributed without profit to those who have expressed a prior general interest in receiving similar information for research and educational purposes. Date: 11-15-09 Host: George Knapp Guests: Bob Lazar, Gene Huff, John Lear George Knapp was joined by the legendary and elusive Bob Lazar, along with Gene Huff and John Lear, for a discussion on the 20th anniversary of the program UFOs: The Best Evidence which first told the story of Area 51. Appearing alone in the first hour, Lazar provided an update on his research into hydrogen power. He detailed his invention of a hydrogen fuel cell which stores enough energy to power a car up to 400 miles. While he expressed some concern about having this technology thwarted by the government, Lazar praised the state of Michigan, where he now lives, for being extremely supportive of his research. Beginning in the 2nd hour, John ...

See the article here:
Area 51 Revisited Main Show Only - secretsocietiestoday - Video

21-05-2012 05:09 Keynote 3: Margaret Lock, McGill University Genes as tools for the prevention of Alzheimer's disease A growing panic exists about the approaching "tsunami" of Alzheimer's disease and its potential impact on the global economy. This concern is heightened by a failure to develop drugs to cure AD, despite billions of dollars worth of research. In response to this situation, Alzheimer experts have recently published position pieces involving a move to the prevention of AD that will make use of biomarkers to predict who among us are at risk. Among the several biomarkers for AD being routinely used in research settings is the susceptibility gene, ApoE?4. Following a brief discussion of debates among AD experts about the concept of AD and the entanglement of aging and dementia, this talk will consider the social and ethical impact of biomarker testing, including genetic testing, with the prime objective of developing drugs to prevent AD.

Here is the original post:
Genomics in Society: Facts, Fictions and Cultures: Margaret Lock Keynote - Video

Cancer cells. The research team developed an innovative cancer-fighting strategy of suppressing the enzyme produced by the PAD4 gene. Credit: National Cancer Institute

(Medical Xpress) -- A team of scientists has developed a promising new strategy for "reactivating" genes that cause cancer tumors to shrink and die. The researchers hope that their discovery will aid in the development of an innovative anti-cancer drug that effectively targets unhealthy, cancerous tissue without damaging healthy, non-cancerous tissue and vital organs. The research will be published in the Journal of Biological Chemistry.

The team, led by Yanming Wang, a Penn State University associate professor of biochemistry and molecular biology, and Gong Chen, a Penn State assistant professor of chemistry, developed the new strategy after years of earlier research on a gene called PAD4 (peptidylarginine deiminase 4), which produces the PAD4 enzyme. Previous research by Wang and other scientists revealed that the PAD4 enzyme plays an important role in protecting the body from infection. The scientists compared normal mice with a functioning PAD4 gene to other mice that had a defective a PAD4 gene. When infected with bacteria, cells from the normal mice attacked and killed about 30 percent of the harmful bacteria, while cells from the defective mice battled a mere 10 percent. The researchers discovered that cells with a functioning PAD4 enzyme are able to build around themselves a protective, bacteria-killing web that Wang and his colleagues dubbed a NET (neutrophil extracellular trap). This NET is especially effective at fighting off flesh-eating bacteria.

Now, in their new study, Wang and his collaborators have focused on the less-desirable effects of the same PAD4 gene. While PAD4 is clearly a critical part of the body's defense strategy, the gene's over-expression may be linked to autoimmune diseases such as rheumatoid arthritis and multiple sclerosis. One situation in which the PAD4 enzyme is markedly increased is in patients with certain cancers, such as breast, lung, and bone cancers. "We know that the PAD4 gene acts to silence tumor-suppressor genes," said Wang. "So we theorized that by inhibiting the enzyme that this gene produces, the 'good guys' -- the tumor-suppressor genes -- would do a better job at destroying cancerous tissue and allowing the body to heal."

To test their theory, Wang and his colleagues treated mice that had cancerous tumors with a molecule to inhibit the PAD4 enzyme. They found that, especially when combined with additional enzyme inhibitors, the treatment worked as effectively as the most-commonly-used chemotherapy drug, doxorubicin, which shrinks tumors by about 70 percent.

Most striking, however, was that the PAD4 enzyme-inhibition strategy caused significantly less damage to healthy tissues. "Current chemotherapy drugs such as doxorubicin don't attack just tumors; unfortunately, they also attack healthy areas of the body," Wang explained. "That's why chemotherapy patients experience such terrible side effects such as weight loss, nausea, and hair loss. Because the PAD4 treatment appears to be less toxic, it could be an excellent alternative to current chemotherapy treatments."

Wang also explained that the PAD4 gene's dual personality -- on the one hand a helpful defense against bacteria, while on the other, a harmful silencer of cancer-suppressor genes -- can be understood from the perspectives of evolution and longer life spans. "Our ancestors didn't have antibiotics, so a bacterial infection could easily result in death, especially in young children," Wang explained. "So, back then, an overactive PAD4 gene was advantageous because the NET bacteria-trapping mechanism was the body's major defense against infection." Wang also explained that on the other hand, because people today have access to antibiotics, we live much longer than our ancestors did. "PAD4's bad effects -- cancer and autoimmune diseases -- tend to be illnesses that appear later in life," Wang said. "So nowadays, an overactive PAD4 gene, while still protective against bacteria, can be detrimental later in life."

This research was funded by the National Cancer Institute of the National Institutes of Health and a Penn State Clinical and Translational Science Institute Pilot Grant Award to Wang and Chen. In addition to Wang and Chen, other researchers who contributed to this project include Yuji Wang, Pingxin Li, Shu Wang, Jing Hu, Megan Fisher, Kira Oshaben, Jianhui Wu, Na Zhao, Ying Gu of Penn State's Center for Eukaryotic Gene Regulation and the Department of Biochemistry and Molecular Biology.

Journal reference: Journal of Biological Chemistry

Provided by Pennsylvania State University

View original post here:
Strategy discovered to activate genes that suppress tumors and inhibit cancer

RICHMOND, Calif., May 21, 2012 /PRNewswire/ --Sangamo BioSciences, Inc. (SGMO) announced today that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics for HIV/AIDS, monogenic diseases and stem cell applications, were described in twelve presentations given by Sangamo scientists and collaborators at the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The meeting was held in Philadelphia from May 15-19, 2012.

"Sangamo's zinc finger DNA-binding protein (ZFP) technology is enabling development of new and improved gene and cell therapy approaches," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president, research and development. "Our ZFP Nuclease (ZFN) technology provides an extremely efficient and precise process for editing any DNA sequence. This enables us to disrupt specific genes or to precisely add DNA sequences that allow a patient's own gene to be corrected and its proper function restored while preserving the natural regulation of the gene.

Sangamo has also developed technology that allows a therapeutic gene to be inserted into a specific 'safe harbor' site. Our ability to target changes to precise locations rather than randomly into the genome, avoids the challenges of traditional gene-addition approaches that can result in unintended mutations. The increased number of related presentations at this meeting demonstrates the growing adoption of ZFN-based gene editing by the field."

Presentations from Sangamo included preliminary clinical data from ongoing Phase 1 clinical trials in HIV/AIDS as well as data from preclinical and research-stage human therapeutic programs. Therapeutic areas included ZFP-based approaches for monogenic diseases such as hemophilia, hemoglobinopathies and Huntington's disease as well as adoptive T-cell therapies for oncology.

"Visibility of ZFPs in the scientific agenda at the ASGCT meeting illustrates the broad range of potential applications for ZFP Therapeutics," said Edward Lanphier, Sangamo's president and CEO. "Our technology can be used to modify any gene with singular specificity and high efficiency. As our technology functions at the DNA level, it can potentially be applied to any disease-related gene making it a versatile platform for the generation of novel therapeutic approaches for the treatment of unmet medical needs."

ZFP Therapeutics Featured at ASGCT Meeting

All abstracts for the meeting are available online at 2012 ASGCT Meeting Abstracts.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. It has ongoing Phase 2 and Phase 1/2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic for the treatment of HIV/AIDS.Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia and hemoglobinopathies such as sickle cell anemia and beta-thalassemia, and a program in Parkinson's disease. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo has entered into a strategic collaboration with Shire to develop therapeutics for hemophilia and other monogenic diseases and has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at http://www.sangamo.com.

ZFP Therapeutic is a registered trademark of Sangamo BioSciences, Inc. CompoZr is a registered trademark of Sigma-Aldrich Corporation.

See more here:
Sangamo BioSciences And Collaborators Highlight Widening Applications Of ZFP Therapeutics® In Presentations At Major ...

WEST BOYLSTON, Mass. & SOUTHBRIDGE, Mass.--(BUSINESS WIRE)--

TransCytos: NEVER BEEN DONE BEFORE

Enginasion and TransCytos announced today that their collaboration has resulted in a prototype transfection technology that is designed to have a dramatic and positive impact on the drug-research industry.

Enginasions product development partner, TransCytos, is developing a novel transfection instrument, Cytofector R1, based on a breakthrough, patent-pending hydrodynamic transfection technology. Transfection, the introduction of genetic material into living cells, is a fundamental and essential genetic engineering process in biomedical research, and drug and gene therapy development. It has revolutionized, worldwide, biotech and pharmaceutical R&D, including the research into such diseases as cancer, diabetes, arthritis, substance abuse, neurological disorders such as Parkinsons and Alzheimers, and also has applications in the study of anxiety, aging, and pain management. Furthermore, transfection is key in the production of recombinant human proteins such as insulin, hormones, antibiotics, and vaccines.

Frost & Sullivan estimates the 2010 transfection market at $350 million, with about 200 million transfections conducted per year. However, existing transfection technologies are limited to a small number of particular cell types just five cell lines make up as much as 50% of the market; in addition, low efficiency and cell viability, as well as very slow cell recovery, are slowing progress.

Because the new TransCytos transfection technology is gentle, highly effective, and does not physically damage cells, it is potentially capable of transfecting all cell types, says Dr. Otto Prohaska, CEO of TransCytos. Current transfection techniques represent a considerable bottleneck for biomedical and pharma R&D due to low efficiency, high variability, cellular toxicity, and the inability to introduce genetic material into many of the most important cell types relevant to major diseases. The majority of cells are hard or impossible to transfect, requiring lengthy, expensive procedures with low yield and poor reproducibility. Field testing of the Cytofector R1 prototype instrument showed (a) transfection of previously non-transfectable cells (e.g. neurons), and (b) better transfection efficiencies and expression of gene products in a shorter period of time, and at lower cost.

The TransCytos transfection process could contribute to a faster and more dependable path to drug discovery, a higher success rate for biotech and pharma, and better cures, added David Bonneau, CEO of Enginasion. The capability of transfecting primary cells effectively is expected to revolutionize progress in research, and especially in drug discovery, development, and production. Enginasion is very proud to be the product-development partner of TransCytos.

Click NEVER BEEN DONE BEFORE link (above) for further information about TransCytos.

About Enginasion

For more than two decades, Enginasion (formerly Industrial Automation Systems) has been an invaluable partner to companies in the Industrial, Medical Technology, Military and Pharmaceutical sectors that need a sophisticated yet affordable engineering resource to help them overcome critical hurdles related to automation, control electronics, embedded software, and/or integrated product development. Since 1995, the Company has been providing key elements of successful R&D projects, manufacturing processes, and new high-tech products at innovative companies in the Northeast U.S.

Originally posted here:
Enginasion Partners with TransCytos to Develop Breakthrough ‘Transfection’ Technology for Genetic Engineering ...

(RTTNews.com) - Viral Genetics Inc. (VRAL.PK) announced that a physician-initiated Investigational New Drug or P-IND application submitted to the FDA in late April, 2012, has cleared the FDA's screening process with the requirement for a regular IND application being waived, resulting in the company being able to begin the first of at least two proposed clinical trial sites to investigate a potential oncology treatment developed from Viral Genetics'Metabolic Disruption Technology or MDT, which is licensed exclusively to the Company.

Enrollment and treatment of patients is expected to commence upon completion of internal hospital Institutional Review Boards (IRBs), which are already underway. The UT Health Science Center portion of the study will commence when all approvals are finalized.

The company said the clinical trial will examine the safety and efficacy of one of Viral Genetics licensed MDT compounds in combination with an existing cancer drug, sorafenib (marketed as Nexavar) in the treatment of patients resistant or otherwise unsuitable for standard treatments for stage III or IV ovarian cancer and related carcinomas.

The Company expects to introduce various other drugs and drug combinations from both of its licensed MDT and Targeted Peptides Technology (TPT) platforms to the clinic throughout 2012 and in 2013 with the submission of several additional pre-IND, P-IND and regular sponsor IND applications.

For comments and feedback: contact editorial@rttnews.com

http://www.rttnews.com

More:
Viral Genetics' P-IND Clears FDA Process To Begin Clinical Trials In Humans

SAN MARINO, Calif.--(BUSINESS WIRE)--

A physician-initiated Investigational New Drug (P-IND) application submitted to the FDA in late April, 2012, has cleared the FDAs screening process with the requirement for a regular IND application being waived, resulting in the company being able to begin the first of at least two proposed clinical trial sites to investigate a potential oncology treatment developed from Viral Genetics (Pinksheets:VRAL.PK - News) Metabolic Disruption Technology (MDT), which is licensed exclusively to the Company. The P-IND part of a larger, coordinated research effort was submitted by the first test site at the Cancer Therapy & Research Center (CTRC) at The University of Texas Health Science Center at San Antonio, which includes patient enrollment at Scott and White Hospital (S&W) in Temple, Texas. Additional test sites may also be added in the future.

Enrollment and treatment of patients is expected to commence upon completion of internal hospital Institutional Review Boards (IRBs), which are already underway. The UT Health Science Center portion of the study will commence when all approvals are finalized.

This clinical trial a milestone in the Companys transition from preclinical to clinical-stage will be the first for the intellectual property developed by Dr. M. Karen Newell Rogers, Viral Genetics Chief Scientist and licensed exclusively by the Company. It will examine the safety and efficacy of one of Viral Genetics licensed MDT compounds in combination with an existing cancer drug, sorafenib (marketed as Nexavar) in the treatment of patients resistant or otherwise unsuitable for standard treatments for stage III or IV ovarian cancer and related carcinomas.

The Primary Investigator on the trial is Tyler Curiel, M.D., MPH, a medical oncologist affiliated with the CTRC at the UT Health Science Center. Dr. Curiel is leading this study as he investigates the efficacy of combining two compounds in a cancer treatment that is hoped to cause the starvation of tumor cells and enhanced anti-tumor immunity, leading to the reduction of tumor size and reduced disease progression.

An internationally recognized expert and speaker in tumor immunology and in the humanities, Dr. Curiel has been a featured scientist on the NOVANow Science Television program on PBS, and is the recipient of numerous medical and civic service awards including the Mauveny Prize for Cancer Research from Tulane University, the STARS Research Enhancement Award from the University of Texas and the Health Care Hero Award from the San Antonio Business Journal. Dr. Curiel holds membership in a multitude of professional organizations and sits on the Editorial Boards of several publications including The Journal of Immunology and The Journal of Clinical Investigation.

Discussing Dr. Curiel's role as lead investigator and study chair, Viral Genetics' Chief Scientist, Dr. M. Karen Newell Rogers said, As one of the country's foremost ovarian cancer specialists, having Dr. Curiel as our primary investigator is an honor and we are grateful for the opportunity to validate MDT science as an approach to cancer therapy. MDT shows great promise across a broad spectrum of cancers, and other disease states. A successful clinical trial for this specific carcinoma using this combination approach could allow us to offer a new treatment option for many women and relief to their families.

The Company looked to start similar clinical trials utilizing MDT compounds on certain cancers at S&W in 2011, with funding for the research provided by an anonymous donor who gifted a $1.5 million grant to the Scott and White Foundation. This $1.5 million grant remains in place specifically to fund this research, but the scope of the project has been expanded to accommodate the participation of noted oncologist Dr. Tyler Curiel at the UT Health Science Center, and to include the UT Health Science Center in both preclinical studies and organization of the P-IND protocol.

We are very proud of Drs. Newell Rogers and Curiel, their teams, and this milestone accomplishment in the Company's growth plan. As we bring our R&D pipeline from the laboratory to the clinical environment, MDT will continue to be one of two foundations the other being TPT around which we believe we can create tremendous value for our shareholders, said Haig Keledjian, Viral Genetics' President. As important as this specific ovarian cancer study may be, it is important to note that it is just one step in a much larger planned research effort relating to MDT compounds as cancer therapies. While we have targeted ovarian cancer as our first indication, there are numerous other types of cancer we intend to study in the early stages of MDT research. There is much more to MDT than just cancer, and there is much more to Viral Genetics than just MDT. As a shareholder myself, I view this as one of multiple opportunities that help us move closer to licensing fees and revenues while potentially bringing relief to people suffering from debilitating illness.

Ovarian cancer is a particularly deadly form of cancer afflicting approximately 25,000 women each year in the United States. Amongst women, it is the most lethal of the gynecological cancers and the fifth leading cause of cancer death. Viral Genetics and Scott and White Foundation are sponsoring the study that is taking place at the UTHSCSA Cancer Therapy and Research Center and at Scott and White Hospital. The primary endpoints of the study are expected to relate to safety, although efficacy will also be studied in secondary endpoints, and to reflect a dose-escalation design.

Original post:
Viral Genetics’ P-IND Clears FDA To Commence Clinical Trials in Humans

SALT LAKE CITY, May 21, 2012 /PRNewswire/ --Intermountain Healthcare and Myriad Genetics today announced they have signed a collaborative research agreement. The purpose of this agreement is to perform research and validation studies on transformative molecular diagnostic tests being developed by Myriad in an effort to improve the care and treatments for patients at Intermountain and around the world. This collaboration highlights the shared purpose of Myriad and Intermountain in improving outcomes and the quality of life for patients.

The first project under this collaboration, PRO 008, is designed to further expand the utility of the Prolaris test by analyzing biopsy samples of 200 patients diagnosed with prostate cancer. This study will assess the ability of the Prolaris test to predict which men are at a heightened risk of biochemical recurrence and therefore should be given more aggressive therapy for their disease. The goal of this, and Myriad's other Prolaris studies underway in multiple centers in the United States and Europe, is to demonstrate the prognostic ability of the Prolaris test in assessing a patient's risk of biochemical recurrence of disease and death from disease.

"We are thrilled to be partnering with Myriad to further research across a number of diseases in an effort to improve patient care," said Brent Wallace, MD, Intermountain's Chief Medical Officer. "We look forward to embarking on our prostate cancer collaboration with Myriad and hope the findings from this study will help define the clinical benefit of the Prolaris test. This will assist in helping men diagnosed with prostate cancer to understand the aggressiveness of their disease and make better informed decisions about appropriate treatment."

"Intermountain is committed to improving patient outcomes which is in perfect alignment with the core mission of Myriad," said Peter Meldrum, President and Chief Executive Officer of Myriad Genetics."This research collaboration will have great potential to help patients by furthering research on molecular diagnostic tests which can assist healthcare providers to effectively guide treatment decisions and determine the risk of disease progression and recurrence."

About Intermountain Healthcare

Intermountain Healthcare, a nonprofit healthcare system based in Salt Lake City, Utah, serves the healthcare needs of Utah and southeastern Idaho residents. Its mission is to provideclinically excellent medical care at affordable rates in a healing environment. For more information visit intermountainhealthcare.org

About Myriad Genetics

Myriad Genetics, Inc., an internationally recognized leader in molecular diagnostics, is dedicated to making a difference in patient's lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's portfolio of molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BRACAnalysis, Colaris, Colaris AP, Melaris, TheraGuide, Prezeon, OnDose, Panexia and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries.

Safe Harbor Statement

More here:
Intermountain Healthcare and Myriad Genetics Enter Into Research Collaboration Agreement

Catherine Jayasuria at the peak of Mount Kinabalu during the inaugural climb last year to raise awareness of Duchenne Muscle Dystrophy.

KOTA KINABALU: A WOMAN who climbed Mount Kinabalu last year to raise awareness of a disease known as Duchenne will be back with a bigger team this year.

This year, the Coalition Duchenne expedition will see US-based Malaysian Catherine Jayasuria leading 60 international climbers to the 4,095m peak on Aug 25.

Last year, there were 35 climbers in the team.

Duchenne muscular dystrophy is a progressive muscle-wasting disease and those who have it often do not live beyond their 20s. Experts estimate at least 20,000 boys are born with the disease each year.

Jayasuria's 19-year-old son, Dusty Brandon, has Duchenne and it was for that reason she returned to her roots here to climb Mount Kinabalu as it was a symbol of stability, strength and hope.

At the summit, the team will again raise the Coalition Duchenne flag in honour of the hundreds of thousands of boys and young men worldwide who have Duchenne.

"Duchenne is a difficult road, and presents significant challenges along the way. Duchenne steals many things, the ability to walk, hug, move, talk and breathe, but there is that something inside of you, that it can never get to, that it can never take -- hope."

Coalition Duchenne also helped raise funds for research to treat the disease.

In March, the non-profit organisation announced the funding of a RM75,000 research agreement with a Michigan-based biotechnical company.

View original post here:
60 climbers to help raise funds

Canadian regulators have approved the world's first stem cell drug.

The drug, Prochymal, will be used to treat a deadly side effect of bone marrow transplants called acute graft-versus host disease (GvHD), which occurs in children.

Acute graft-versus host disease kills about 80 percent of children affected.

Prochymal uses stem cells from healthy adult donors, with one donation able to create 10,000 doses of the drug, reported the New York Times.

The manufacturer, Maryland-based Osiris Therapeutics Inc., saw their shares climb 5.5 percent to $5.55 after losing 24 percent in the last year, reported Bloomberg.

In extended trading, stocks rose 14 percent.

The drug was approved, said Reuters, on the condition that further clinical tests are carried out.

There has been debate about the effectiveness of the drug in recent years.

Late stage clinical trials three years ago failed to show results but more recent tests have shown the drug to be relatively effective about a month into therapy.

Osiris says that it plans to seek approval from the US Food and Drug Administration this year.

Original post:
Stem cell drug approved in Canada to treat bone marrow disease

In complex therapeutic areas such as oncology, thought leaders have long sought improvements to lengthy trials, hit-or-miss treatments and soaring research costs. While there is still much to be resolved, the rise of personalized medicine has set its sights on these and other obstacles in the pursuit of more targeted and efficient therapies.

The one-size-fits-all approach that has dominated clinical drug development has been the most appropriate route when costs are considered, but this strategy has failed to meet the needs of the patient. Guesswork has dominated drug development for far too long, leading to a push to make drugs mechanistic through molecular target validation, biomarker identification and other techniques.

Researchers are only now beginning to make headway into the importance of gene variation, which highlights the importance of close analysis of individual patients in the pursuit of the right type of patient-oriented therapy needed. Scientists are also making important breakthroughs in the understanding of variables such as genetic changes and drug sensitivity, which have dramatic implications for determining how effective specific medications might be for unique individuals. The promise of smaller, faster studies that could allow for the testing of a much higher volume of treatments is becoming more and more difficult to ignore.

However, a host of obstacles have stood in the way of a more personalized approach:

The questions listed above are not the only challenges that threaten a more widespread adoption of personalized medicine. A move toward personalized therapies will necessitate an overhaul of the way research is considered and executed, including the conduct of clinical studies. Study administrators will have to acknowledge and work around long timeframes for reaching trial endpoints.

Overcoming the entrenched risk aversion that is part of the culture of regulatory and statistical mindsets is also necessary. In an industry that is leery of any element of risk, it will be a challenge to accept and embrace the risk that is inherent within personalized medicine.

Adoption of New Technology, Better Collaboration is Needed In the face of many obstacles, there is still optimism surrounding the path forward for personalized medicine. Some researchers feel nanotechnology could offer access to data that can help narrow the focus of personalized treatments, and others hold high hopes for sequencing and translational bioinformatics.

Of course, in order to capitalize on the possibilities of personalized medicine, strides must be made in key areas. Advocates are calling for the FDA to deliver more clarity surrounding personalized therapies, while others feel that collaboration among a host of healthcare disciplines is needed in order to reach the customization and individualization that could mark successful personalized treatments.

While the road ahead may be rocky and the task may seem daunting, industry observers not only recognize the immense promise of personalized medicine, but feel that it is a necessary evolution in clinical research and drug development.

Despite its unfulfilled promises, wrote Paul Thomas of Pharma QbD, personalized medicine still provides the greatest hope for change and for Big Pharma to rediscover success.

Read this article:
Assessing the Landscape for Personalized Medicine

As Dan Via lay face down in the surf in Corolla, unable to move his arms and legs, the thought flashed through his mind: "I'll probably never ride my bike again."

It was Labor Day weekend and the Williamsburg pediatrician, then 51, was relaxing at the North Carolina beach with his wife, Susan, and their younger son, Nathan. The family had had an emotionally grueling summer with the acute illness of his 90-year-old mother-in-law; and a week earlier, his older son, Forrest, had left for his freshman year at Radford University.

At midday, in slightly choppy chest-deep water, Via was lying on a boogie board, when a strong wave caught the board and propelled him forward face-first into the sand. He remembers the blow. His neck hyper-extended, the force of the water tore the ligaments and ruptured a disc in his upper neck. But he never lost consciousness not as strangers dragged him to safety, not while the local emergency crew stabilized him, and not in the Nightingale helicopter as it transported him to a hospital in Norfolk. Later that same day, after extensive screening tests, he had a bone graft and a steel plate and screws inserted in his spine to fuse discs C3 and C4.

He was effectively a quadriplegic with an uncertain future. His doctors weren't optimistic that he'd walk again. They thought he might learn to feed himself.

Via has defied the odds. Last weekend, as part of the 100-mile Cap2Cap ride from Williamsburg to Richmond, familiar territory for the cycling enthusiast, he rode a full 25 miles in a recumbent bike. "It eliminates the balance issue," he says, somewhat ruefully of his low-to-the-ground three-wheeler.

Family and community support

In January, just four months after the accident, he returned part-time to his medical practice, Sentara Pediatric Physicians. "I think he has increased the demands on himself and is now probably seeing the same number of patients as before," says Jennifer Altman, one of six physicians in the practice.

Initially, she says, his colleagues were concerned about how the emotional and physical demands dealing with a squirming 15-month-old, for example would affect his healing process. The fears proved unfounded. "It sounds trite, but he has been inspiring to us in the office and to his patients as well. Children don't notice his disability, they see past it," she says. "I'm able to see his improvements day to day and week to week. He has become much more his old self. This is his second home."

Slowly, but surely, Via is defying his doctors' gloomy prognosis and regaining full function. "When I look at him now, it's just like he has arthritis," says his wife, Susan, who took a leave of absence from her job teaching violin at the College of William and Mary to look after him. "She was steadfastly positive," he says.

The couple met as musicians for the Virginia Symphony, where Via played the double bass for 7 years prior to returning to his undergraduate alma mater Duke for his medical degree. "He's a really, really talented natural musician," says Susan, noting that he went to Juilliard, a top-ranked music school.

Read the original:
Exclusive: 'On the road again' — Dr. Dan revels in return to (recumbent) biking

COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics Inc. (NASDAQ:OSIR - News) announced today it has received market authorization from Health Canada to market its stem cell therapy Prochymal (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. The historic decision marks the worlds first regulatory approval of a manufactured stem cell product and the first therapy approved for GvHD a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis.

"I am very proud of the leadership role Canada has taken in advancing stem cell therapy and particularly gratified that this historic decision benefits children who would otherwise have little hope," said Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada and Principal Investigator in the phase 3 clinical program for Prochymal. "As a result of Health Canada's comprehensive review, physicians now have an off-the-shelf stem cell therapy in their arsenal to fight GvHD. Much like the introduction of antibiotics in the late 1920's, with stem cells we have now officially taken the first step into this new paradigm of medicine."

Prochymal was authorized under Health Canada's Notice of Compliance with conditions (NOC/c) pathway, which provides access to therapeutic products that address unmet medical conditions and which have demonstrated a favorable risk/benefit profile in clinical trials. Under the NOC/c pathway, the sponsor must agree to carry out confirmatory clinical testing.

Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies, said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. Most importantly, today is a great day for children and their families who bravely face this horrific disease. While today marks the first approval of a stem cell drug, now that the door has been opened, it will surely not be the last.

Health Canadas authorization was made following the recommendation of an independent expert advisory panel, commissioned to evaluate Prochymal's safety and efficacy. In Canada, Prochymal is now authorized for the management of acute GvHD in children who fail to respond to steroids. The approval was based on the results from clinical studies evaluating Prochymal in patients with severe refractory acute GvHD. Prochymal demonstrated a clinically meaningful response at 28 days post initiation of therapy in 61-64 percent of patients treated. Furthermore, treatment with Prochymal resulted in a statistically significant improvement in survival when compared to a historical control population of pediatric patients with refractory GvHD (p=0.028). The survival benefit was most pronounced in patients with the most severe forms of GvHD. As a condition of approval, the clinical benefit of Prochymal will be further evaluated in a case matched confirmatory trial and all patients receiving Prochymal will be encouraged to participate in a registry that will monitor the long-term effects of the therapy.

Refractory GvHD is not just deadly to the patients it afflicts, but is devastating for the family, friends, and caregivers who watch helplessly as the disease progresses, said Joanne Kurtzberg, MD, Head of the Pediatric Bone Marrow Transplant Program at Duke University and Lead Investigator for Prochymal. "I have personally seen Prochymal reverse the debilitating effects of severe GvHD in many of my patients and now, after nearly two decades of research, the data demonstrating consistently high response rates, a strong safety profile and improved survival clearly support the use of Prochymal in the management of refractory GvHD."

Prochymal is currently available in several countries, including the United States, under an Expanded Access Program (EAP). Prochymal will be commercially available in Canada later this year.

Today Osiris turns the promise of stem cell research into reality, delivering on decades of medical and scientific research, said Peter Friedli, Chairman and Co-founder of Osiris. It took 20 years of hard work and perseverance and I want to personally thank everyone involved for their dedication to this important mission.

In addition to the extensive intellectual property protection Osiris has around Prochymal, which includes 48 issued patents, Health Canada's decision will also provide Prochymal with regulatory exclusivity within the territory. Canada affords eight years of exclusivity to Innovative Drugs such as Prochymal, and an additional six-month extension is available since it addresses a pediatric population.

Read the original:
World's First Approved Stem Cell Drug; Osiris Receives Marketing Clearance from Health Canada for Prochymal

Indian clinic's stem cell therapy real?

STORY HIGHLIGHTS

For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.

New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

That's when Cash began walking with the aid of braces for the first time.

Go here to see the original:
Family hangs hope for boy on unproven therapy in India

MONT-SAINT-GUIBERT, Belgium, May 18, 2012 /PRNewswire/ --

The Belgian biotechnology company, Cardio3 BioSciences (C3BS), a leader in the discovery and development of regenerative and protective therapies for the treatment of cardiovascular diseases, today announces that the final results of its Phase II clinical trial of C3BS-CQR-1 is will be presented at the late breaking clinical trial session at the European Society of Cardiology 2012 Heart Failure Congress in Belgrade, Serbia taking place on May 19-22.

Andr Terzic, M.D., Ph.D, Director at Center of Regenerative Medicine, Mayo Clinic, the co-lead investigator on the trial, will present new final follow up data on the Company's stem cell therapy for heart failure, C3BS-CQR-1, which is based on "Cardiopoiesis" proprietary technology. The presentation will be held on Sunday, May 20th in Belgrade, Serbia.

Dr. Christian Homsy, CEO of Cardio3 BioSciences, said: "Being selected to present the final follow-up data in the late breaking clinical trial session at this prestigious cardiology congress highlights the quality of our technology and reiterates our belief in C3BS-CQR-1 as a potential treatment for patients with heart failure, a condition with a significant unmet medical need. We look forward to advancing the product into Phase III."

About Cardio3 BioSciences

Cardio3 BioSciences is a Belgian leading biotechnology company focused on the discovery and development of regenerative and protective therapies for the treatment of cardiac diseases. The company was founded in 2007 and is based in the Walloon region of Belgium. Cardio3 BioSciences leverages research collaborations in the US and in Europe with Mayo Clinic and the Cardiovascular Center Aalst, Belgium.

The Company's lead product candidate C3BS-CQR-1 is an innovative pharmaceutical product consisting of autologous cardiac progenitor stem cells. C3BS-CQR-1 is based on ground breaking research conducted at Mayo Clinic that allowed discovery of cardiopoiesis, a process to mimic in adult stem cells the natural signals triggered in the early stages of life during the cardiac tissue development. Cardio3 BioSciences has also developed C-Cath, the next-generation injection catheter with superior efficiency of delivery of bio therapeutic agents into the myocardium.

C3BS-CQR-1, C-Cure, C-Cath, Cardio3 BioSciences and the Cardio3 BioSciences and C-Cath logos are trademarks or registered trademarks of Cardio3 BioSciences SA, in Belgium, other countries, or both. Mayo Clinic holds equity in Cardio3 BioSciences as a result of intellectual property licensed to the company. In addition to historical facts or statements of current condition, this press release contains forward-looking statements, which reflect our current expectations and projections about future events, and involve certain known and unknown risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. These forward-looking statements are further qualified by important factors, which could cause actual results to differ materially from those in the forward-looking statements, including timely submission and approval of anticipated regulatory filings; the successful initiation and completion of required Phase III studies; additional clinical results validating the use of adult autologous stem cells to treat heart failure; satisfaction of regulatory and other requirements; and actions of regulatory bodies and other governmental authorities. As a result, of these factors investors and prospective investors are cautioned not to rely on any forward-looking statements. We disclaim any intention or obligation to update or review any forward-looking statement, whether as a result of new information, future events or otherwise.

For more information contact:

Cardio3 BioSciences: http://www.c3bs.com Dr Christian Homsy, CEOTel : +32-10-39-41-00 Anne Portzenheim, Communication Manager aportzenheim@c3bs.com

The rest is here:
Cardio3 BioSciences Has Been Selected to Present C3BS-CQR-1 Trial Data in Late Breaking Clinical Trial Session at ...

A Columbia-based biotechnology company said this week it received the worlds first government approval to market a stem cell drug, in Canada.

Osiris Therapeutics, founded in 1992, spent 17 years developing a stem cell therapy that offers anti-inflammatory and tissue-regeneration properties. The first treatment it has received approval for this week will help treat children whove received bone marrow transplants that their bodies have rejected. The condition, known as acute graft-versus-host disease, or GvHD, is fatal to 80 percent of the children who contract it, the company said.

C. Randal Mills, president and CEO of Osiris, said in a conference call Friday morning that the company has spent the past eight years navigating clinical trials and regulatory paperwork in a mission to be the first approved stem cell treatment in the world.

During the past eight years, we have not wavered from that mission, Mills said. We now need a new mission.

The two-decade path to market for Osiris drug, Prochymal, is par for the course in the biotechnology industry, where a new pharmaceutical is measured in multi-million dollar clinical trials and reviews that take years.

Prochymal is the first off-the-shelf stem cell drug approved for sale, and the first approved for GvHD, the company said. It derives its stem cells, it said, from the bone marrow of healthy adult donors between 18 and 30 years old.

Osiris is a small biotech company, with around 50 employees, in an industry where far larger competitors, with thousands of employees, usually grab the headlines with blockbuster drugs.

Yet Osiris is a key player in the states nascent stem cell therapies industry. Osiris is one of the worlds largest and most advanced stem cell firms, according to testimony provided by the leaders of the Maryland Stem Cell Research Fund this year in the General Assembly.

The taxpayer-subsidized fund doles out millions of dollars a year in grants to promote stem cell research; Osiris, however, has never received a grant from the fund, according to TEDCO.

This week, the fund said it will award $12.4 million in research grants to 40 projects led by university researchers from Johns Hopkins, University of Maryland and other institutions.

Original post:
Columbia firm is world's first to market with stem cell drug

COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics Inc. (NASDAQ:OSIR - News) announced today it has received market authorization from Health Canada to market its stem cell therapy Prochymal (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. The historic decision marks the worlds first regulatory approval of a manufactured stem cell product and the first therapy approved for GvHD a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis.

"I am very proud of the leadership role Canada has taken in advancing stem cell therapy and particularly gratified that this historic decision benefits children who would otherwise have little hope," said Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada and Principal Investigator in the phase 3 clinical program for Prochymal. "As a result of Health Canada's comprehensive review, physicians now have an off-the-shelf stem cell therapy in their arsenal to fight GvHD. Much like the introduction of antibiotics in the late 1920's, with stem cells we have now officially taken the first step into this new paradigm of medicine."

Prochymal was authorized under Health Canada's Notice of Compliance with conditions (NOC/c) pathway, which provides access to therapeutic products that address unmet medical conditions and which have demonstrated a favorable risk/benefit profile in clinical trials. Under the NOC/c pathway, the sponsor must agree to carry out confirmatory clinical testing.

Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies, said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. Most importantly, today is a great day for children and their families who bravely face this horrific disease. While today marks the first approval of a stem cell drug, now that the door has been opened, it will surely not be the last.

Health Canadas authorization was made following the recommendation of an independent expert advisory panel, commissioned to evaluate Prochymal's safety and efficacy. In Canada, Prochymal is now authorized for the management of acute GvHD in children who fail to respond to steroids. The approval was based on the results from clinical studies evaluating Prochymal in patients with severe refractory acute GvHD. Prochymal demonstrated a clinically meaningful response at 28 days post initiation of therapy in 61-64 percent of patients treated. Furthermore, treatment with Prochymal resulted in a statistically significant improvement in survival when compared to a historical control population of pediatric patients with refractory GvHD (p=0.028). The survival benefit was most pronounced in patients with the most severe forms of GvHD. As a condition of approval, the clinical benefit of Prochymal will be further evaluated in a case matched confirmatory trial and all patients receiving Prochymal will be encouraged to participate in a registry that will monitor the long-term effects of the therapy.

Refractory GvHD is not just deadly to the patients it afflicts, but is devastating for the family, friends, and caregivers who watch helplessly as the disease progresses, said Joanne Kurtzberg, MD, Head of the Pediatric Bone Marrow Transplant Program at Duke University and Lead Investigator for Prochymal. "I have personally seen Prochymal reverse the debilitating effects of severe GvHD in many of my patients and now, after nearly two decades of research, the data demonstrating consistently high response rates, a strong safety profile and improved survival clearly support the use of Prochymal in the management of refractory GvHD."

Prochymal is currently available in several countries, including the United States, under an Expanded Access Program (EAP). Prochymal will be commercially available in Canada later this year.

Today Osiris turns the promise of stem cell research into reality, delivering on decades of medical and scientific research, said Peter Friedli, Chairman and Co-founder of Osiris. It took 20 years of hard work and perseverance and I want to personally thank everyone involved for their dedication to this important mission.

In addition to the extensive intellectual property protection Osiris has around Prochymal, which includes 48 issued patents, Health Canada's decision will also provide Prochymal with regulatory exclusivity within the territory. Canada affords eight years of exclusivity to Innovative Drugs such as Prochymal, and an additional six-month extension is available since it addresses a pediatric population.

See more here:
World's First Approved Stem Cell Drug; Osiris Receives Marketing Clearance from Health Canada for Prochymal

Osiris Therapeutics Inc says Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.

Osiris shares rose 14 percent to $6.00 in extended trading after the news was announced.

Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient's body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.

The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.

Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company's chief executive, said in an interview that could take three to four years.

Some investment analysts have been skeptical about Prochymal's future. In 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.

Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD -- those who have more or less failed all other therapies -- Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 percent of patients.

In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.

The Canadian authorities approved the drug on the basis of that data, the company said.

FDA SUBMISSION THIS YEAR

Read the original here:
Canada approves stem cell therapy

By Meg Tirrell - 2012-05-18T20:13:19Z

Osiris Therapeutics Inc. (OSIR) rose after the company said it won the worlds first approval for a stem- cell drug, gaining clearance in Canada to sell Prochymal for a disease that can attack patients who received bone-marrow transplants.

Osiris climbed 5.5 percent to $5.55 at 4 p.m. New York time. The shares have lost 24 percent in the last 12 months.

Prochymal was approved for the treatment of acute graft versus host disease in children for whom steroids havent worked, the Columbia, Maryland-based company said yesterday in a statement. Steroids have a 30 percent to 50 percent success rate, and severe GvHD can be fatal in 80 percent of cases, according to the company.

The therapy uses mesenchymal stem cells derived from bone marrow that can take on different forms to combat the immune reaction that causes patients to literally peel out of their skin and shed their intestinal lining, Osiris Chief Executive Officer Randal Mills said in a telephone interview. The disease has no equal.

The company hasnt sought approval for this indication in the U.S., where regulators asked for more data before considering whether to allow sales of the drug, Mills said. Prochymal is used in eight countries, including the U.S., on an expanded-access program basis, which allows patients to receive experimental medicines without participating in clinical trials.

This is the first regulatory approval of a stem-cell drug -- where the active ingredient of the drug is a stem cell -- in the world, Mills said. Its a huge deal for us and a huge deal for the entire field of stem-cell therapy.

Osiris shares declined from an all-time high of $28.56 in 2007 as the biotechnology company faced clinical setbacks, including two studies in 2009 that failed to show statistical improvement of Prochymal versus placebo.

The Canadian approval was based on data showing a clinically meaningful response 28 days after starting therapy for 61 percent to 64 percent of patients treated, Osiris said in the statement.

Prochymal may draw $16.7 million in revenue next year with Canadian approval, estimated Edward Tenthoff, an analyst with Piper Jaffray & Co., before the companys announcement. He said that while Prochymal would be the first stem-cell drug to receive approval, other regenerative products used for wound- healing that employ stem cells are already on the market, such as Carticel from Sanofis Genzyme unit.

See the original post here:
Osiris Wins Canadian Approval for First Stem-Cell Therapy