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 ...

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Area 51 Revisited Main Show Only - secretsocietiestoday - Video

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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.

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Genomics in Society: Facts, Fictions and Cultures: Margaret Lock Keynote - Video

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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

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Strategy discovered to activate genes that suppress tumors and inhibit cancer

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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.

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

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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.

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Enginasion Partners with TransCytos to Develop Breakthrough ‘Transfection’ Technology for Genetic Engineering ...

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(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

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

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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.

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

Recommendation and review posted by Bethany Smith

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

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Intermountain Healthcare and Myriad Genetics Enter Into Research Collaboration Agreement

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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.

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60 climbers to help raise funds

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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.

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Stem cell drug approved in Canada to treat bone marrow disease

Recommendation and review posted by sam

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.

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Assessing the Landscape for Personalized Medicine

Recommendation and review posted by sam

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.

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

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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

Recommendation and review posted by simmons

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.

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Family hangs hope for boy on unproven therapy in India

Recommendation and review posted by simmons

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 ...

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

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.

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Osiris Wins Canadian Approval for First Stem-Cell Therapy

Recommendation and review posted by Bethany Smith

LONDON Scientists have mapped the complete genetic codes of 21 breast cancers and created a catalogue of the mutations that accumulate in breast cells, raising hopes that the disease may be able to be spotted earlier and treated more effectively in future.

The research, the first of its kind, untangles the genetic history of how cancer evolves, allowing scientists to identify mutational patterns that fuel the growth of breast tumors, and start to work out the processes behind them.

"These findings have implications for our understanding of how breast cancers develop over the decades before diagnosis in adults and might help to find possible targets for improved diagnosis or therapeutic intervention in the future," said Mike Stratton, who led the research team.

Breast cancer kills more than 450,000 women a year worldwide and is the most common cancer among women, accounting for 16 percent of all cases, according to the World Health Organization (WHO).

A study last year by the Institute for Health Metrics and Evaluation in the United States found that global breast cancer cases have more than doubled in just three decades, from 641,000 cases in 1980 to 1.6 million cases in 2010 - a pace that far exceeds global population growth.

"This is the first time we've been able to delve fully into breast cancer genomes in such a thorough way," said Peter Campbell, head of cancer genetics and genomics at the Wellcome Trust Sanger Institute in Cambridge, where the studies were led.

The work had given scientists "a full panoramic view of the cancer genome" and helped them identify "mutational patterns rather than individual mutations in specific genes", he added.

DNA mutations "We've known for many years now that all cancers are due to abnormalities of DNA...that occur in every single cell of the body over the course of a lifetime," said Stratton.

"But although we've known that, it's remarkable how rudimentary our knowledge is about what the processes are that cause these abnormalities, these mutations in our DNA."

Stratton's team sequenced the genomes of the 21 breast cancers and catalogued all the mutations. They found five major processes that cause one letter of code to be changed to another letter. Genetic code comes in four DNA letters, A,C,G and T.

Read the original here:
Gene changes behind breast cancer untangled

Recommendation and review posted by Bethany Smith

CRO adds TRAC to service portfolio for pre-clinical ADME-Tox studies

HELSINKI, FINLAND - Plexpress, developer of the innovative TRAC platform for high-throughput gene expression analysis, has announced it will partner with SBW to provide SBW's customers with access to Plexpress' novel technology. SBW, a contract research organisation serving the drug discovery market, will offer Plexpress' pre-validated TRAC Cytochrome P450 (CYP) family multiplex assays as part of its portfolio for investigating in vitro drug interactions and pre-clinical ADME-Tox profiles. Such a service is expected to be very popular, especially given a recent draft report from the FDA, which suggests that drug discovery researchers use CYP mRNA induction, rather than CYP activity, to assess drug metabolism and activity (1).

TRAC (Transcript Analysis with the aid of Affinity Capture) technology is a new approach to measuring gene expression changes that offers several benefits over qPCR and microarrays, including higher sample throughput and increased assay speed, while simultaneously reducing technical variation. For more information about how TRAC could boost the efficiency of your drug discovery pipeline, please visit http://www.plexpress.com. For more information about the new SBW service utilising TRAC, view the full release by visiting the SBW website or contact SBW directly at info@sbw.fi.

Reference: 1. FDA Guidance for Industry Report (Feb 2012): Drug Interaction Studies Study Design, Data Analysis, Implications for Dosing, and Labeling Recommendations

About Plexpress Plexpress was established as a spinoff from the Finnish Technology Research Center VTT in 2007. Including R&D at VTT, the company's TRAC technology has been developed continuously for nearly 10 years and it has been documented in over 15 publications. The technology was originally developed for demanding bioprocess applications, which required a very robust solution for gene expression analysis. Since starting operations in 2008, the company has carried out over 50 projects using the TRAC technology in the fields of pharmaceutical development and academic research.

For further information see: http://www.plexpress.com

About SBW SBW is an integrated contract research organization (CRO), located in Helsinki, Finland and has offices also in Germany and North-America. We operate under the brands SBW/Biodix, SBW/Toxis and SBW/novamass. We offer comprehensive services and platforms for drug discovery, functional food ingredients testing, REACH chemical safety assessment and related life science areas. Our services have more than 100 customers in aroung 20 countries in Europe, Asia and US. We offer services on a fee for service Cbasis as well as contracting early discovery projects on milestone reward basis. As a proof of our model, we bring our expertise and experience from more than 600 engagements with customers ranging from virtual Biotechs to several top 10 Pharmaceutical companies. We also promote industryacademia partnerships and are involved in many academic research projects aiming to provide novel innovation services. We are also authors in over 60 peer-reviewed research papers on drug research and technologies since 2005.

Dr Jari Rautio: CEO Plexpress t: +358 40 504 0355 e: jari.rautio@plexpress.com w: http://www.plexpress.com

Eliminate sensor failure from target impact and the harsh effects of extreme environments. Ifm's 316 stainless steel, metal face sensors resist aggressive chemicals, will not corrode, and offer a true non-flush operation with no false outputs.

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Plexpress and SBW Partner to Offer TRAC Gene Expression Analysis

Recommendation and review posted by Bethany Smith

Public release date: 17-May-2012 [ | E-mail | Share ]

Contact: Clea Desjardins clea.desjardins@concordia.ca 514-848-2424 x5068 Concordia University

Montreal, May 18, 2012 What do synthetic fuels, new treatments for malaria and genetic engineering have in common? In a word, biology. To examine the wide-reaching implications of this evolving discipline, Concordia University's Centre for Structural and Functional Genomics presents Building Biology: A Symposium on Synthetic Biology.

Held at Concordia's Loyola Campus on May 21, the symposium brings together the world's top researchers on the subject, including academics from Harvard, MIT, Berkeley and Johns Hopkins. Because this event is closely tied into the work of Concordia's cutting-edge genomics laboratory, the President and CEO of Genome Canada, Pierre Meulien, will give the welcoming address.

What: Building Biology: A Symposium for Synthetic Biology When: Monday, May 21, 8 a.m. to 6 p.m. Where: Concordia University, Loyola Campus, 7141 Sherbrooke Street West, SP S-110

The purpose of this symposium is to foster interaction between established synthetic biologists in Canada and North America. The event represents a cornerstone in the creation of a synthetic biology research hub for Canada while putting Concordia on the map as the prime location for research in synthetic biology.

Speakers include: Pierre Meulien (Genome Canada), Jay Keasling (University of California Berkeley), Jack Newman (Amyris Inc.), Peter Facchini (University of Calgary), Ron Weiss (MIT), Pamela Silver (Harvard University), Radhakrishnan Mahadevan (University of Toronto), Matthew Scott (University of Waterloo), Mads Kaern (University of Ottawa), Joel Bader (John Hopkins University) and Nathan Hillson (Joint BioEnergy Institute).

###

This symposium is generously sponsored by Concordia University, Genome Quebec, CSFG, PhytoMetaSyn, CRIBIQ, DNA 2.0 and IDT.

Related links:

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Concordia welcomes world's best synthetic biology researchers

Recommendation and review posted by Bethany Smith

On my latest blog, ThinkCreeps posted a comment quoting my statement that "we do not know why natural genetic engineering systems are as successful as they have been in generating useful evolutionary novelties in the history of life." Then he goes on to answer, "Yes we do - the good ones spread quickly through the population."

If only things were that simple! Good novelties just appear, as if by magic, and then spread due to their selective advantages. ThinkCreeps apparently shares a common illusion in evolutionary thinking that natural selection is all we need in the way of basic principles to understand the evolutionary process.

All scientific views of evolution by descent with modification envision two separate and essential steps in the establishment of living organisms with novel features:

In the absence of detailed information about the mechanisms of variation, heritable differences were widely assumed to arise randomly and accidentally. After Mendelism was rediscovered at the start of the 20th century, Mendelian segregations were added as variation modes in the neo-Darwinian "Modern Synthesis." Nonetheless, the sources of new segregating alleles (genetic differences) were still assumed to be stochastic accidents.

It was even claimed by some neo-Darwinians that evolution could be ascribed to changes in allele frequencies in populations due to natural selection acting on their fitness contributions. Many thinkers did not notice that this argument neglected the large number of cases where evolutionary differences were accompanied by other kinds of heritable change, such as alterations in chromosome structure or number.

With the advent of molecular genetics and DNA sequencing in the second half of the 20th century, it became possible to study the mechanisms of genome change in detail. It is commonly assumed that genome alterations account for the vast majority of heritable variation in living organisms. Other kinds of heritable changes are known and may also play an important role in evolution. Non-DNA changes include inheritance of self-templated cell structures and protein conformations, such as prions.

The results of the molecular studies are clear. Heritable changes can occur at the genetic level, through alterations in DNA sequences and in the structures of cell DNA molecules, and at the epigenetic level, through alterations in the way DNA is modified chemically and complexed with RNA and proteins in stable chromatin configurations.

Genetic and epigenetic changes result from the actions of cell biochemical activities, not from accidents. This is a critical fundamental discovery of molecular genetics.

There are many different activities that work directly on DNA and bring about genetic changes, ranging from single nucleotide substitutions to major restructuring of chromosomes. DNA modules can move from one place to another in the genome, RNA molecules can be reverse transcribed into DNA and inserted into the genome, and broken DNA molecules can be rejoined in novel combinations. The genome sequence record provides a rapidly growing mountain of evidence showing how important such non-random events have been in evolutionary history.

There are also many distinct activities that modify DNA and chromatin structures leading to heritable epigenetic changes. Our knowledge of these chromatin remodeling processes is younger than our acquaintance with DNA changes, and they do not leave the same kind of trace in the genome sequence record. But we do know that epigenetic changes have a profound influence on genome restructuring activities, and the same ecological challenges and stresses lead to high levels of both epigenetic and genetic variability.

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James A. Shapiro: Variation and Selection: What's the Difference? What Are the Issues?

Recommendation and review posted by Bethany Smith

SAN DIEGO, May 18, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today announced that a new publication from the large Women & Infants multi-center clinical study on the Sequenom Center for Molecular Medicine's (Sequenom CMM) MaterniT21 PLUS laboratory-developed test (LDT) has been published online in the peer-reviewed journal, Prenatal Diagnosis. Along with this week's publication, the Company announces that as of the week ended May 12, 2012, Sequenom CMM has processed more than 10,000 commercial MaterniT21 PLUS test samples in 2012.

The publication addresses the capability of the MaterniT21 PLUS LDT to accurately detect the presence of certain fetal trisomies in pregnant women carrying twins or triplets. The paper will appear in the journal's May issue and the full abstract can be found online at: http://onlinelibrary.wiley.com/doi/10.1002/pd.3892/abstract.

"The underlying biology and these positive study data provide evidence that this type of DNA-testing can be reliably employed as a clinical management option for women expecting twins or triplets who are at increased risk for fetal chromosome anomalies," said Allan Bombard, M.D., Laboratory Director for Sequenom Center for Molecular Medicine.

The published results are derived from the large international, multi-center study conducted at 27 prenatal diagnostic centers worldwide, with previous publications on trisomy 21 and trisomies 18 and 13 in Genetics in Medicine. Participating sites collected and processed maternal plasma samples from 4,664 pregnant women in the late first and early second trimester who were at increased risk for fetal aneuploidy. Blinded samples from pregnancies with trisomy 21, trisomy 18, and trisomy 13 as well as those with other abnormal karyotypes were tested.

In the same multi-center study, maternal plasma samples were tested from 25 twin and two triplet pregnancies. Of the twin pregnancies, there were no trisomies in 17 pregnancies (known as euploid), trisomy 21 in seven (two cases of trisomy 21 in both fetal twins, five cases of trisomy 21 in one fetal twin only), and trisomy 13 in one (in one fetal twin). There were two triplet pregnancies, neither of which had trisomies. The MaterniT21 PLUS technology correctly classified the eight twin pregnancies with trisomy 21 or trisomy 13, the 17 twin euploid pregnancies and both triplet euploid pregnancies.

"We know that, in the U.S., pregnant women carrying twins or higher multiples are becoming more common due to the use of assisted reproductive technologies and acknowledge that more of these women have increased risks for fetal aneuploidy, such as advanced maternal age," said Harry F. Hixson, Jr., Ph.D., Chairman and CEO, Sequenom, Inc. "This published data provides valuable evidence to specialists that Sequenom CMM's MaterniT21 PLUS LDT can provide reliable detection of certain fetal trisomies in twins, just as in single pregnancies."

The research was led by Jacob Canick, PhD, and Glenn Palomaki, PhD, of the Division of Medical Screening and Special Testing in the Department of Pathology and Laboratory Medicine at Women & Infants Hospital and The Warren Alpert Medical School of Brown University. The study also included scientists at Sequenom Center for Molecular Medicine, San Diego, CA.

As of the week ended May 12, Sequenom CMM has processed more than 10,000 MaterniT21 PLUS tests in 2012. Due to the successful rate of adoption, the Company recently announced that it has increased its internal goal to 40,000 MaterniT21 PLUS tests billed in 2012, up from the original internal goal of 25,000 tests billed for the year. As of the last week in April, the 52-week run rate had increased to more than 45,000 tests.

The MaterniT21 PLUS LDT is available solely through Sequenom CMM as a testing service to physicians. To learn more about the test, please visit Sequenomcmm.com.

About SequenomSequenom, Inc. (SQNM) is a life sciences company committed to improving healthcare through revolutionary genetic analysis solutions. Sequenom develops innovative technology, products and diagnostic tests that target and serve discovery and clinical research, and molecular diagnostics markets. The company was founded in 1994 and is headquartered in San Diego, California. Sequenom maintains a Web site at http://www.sequenom.com to which Sequenom regularly posts copies of its press releases as well as additional information about Sequenom. Interested persons can subscribe on the Sequenom Web site to email alerts or RSS feeds that are sent automatically when Sequenom issues press releases, files its reports with the Securities and Exchange Commission or posts certain other information to the Web site.

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Study In Prenatal Diagnosis Finds Sequenom CMM's MaterniT21™ PLUS Lab-developed Test Accurately Detects Fetal ...

Recommendation and review posted by Bethany Smith

Public release date: 17-May-2012 [ | E-mail | Share ]

Contact: Jane E. Rubinstein jrubinstein@rubenstein.com 212-843-8287 IntegraGen

Toronto, CANADA (May 17, 2012) By focusing on the identification of common genetic variants, researchers have identified 57 single nucleotide polymorphisms (SNPs) that predictwith a high degree of certainty--the risk that siblings of children with Autism Spectrum Disorder (ASD) will also develop the condition. The findings were presented at the International Meeting for Autism Research.

ASD is among the most common form of severe developmental disability with prevalence rates up to 1 in 88 children. Boys are greater than four times more likely to be diagnosed with ASD, while recurrence risks for the sibling of a child with ASD are estimated at 18.7%. Since multiple studies have shown that early assessment and intervention offer significantly improved long-term outcomes, early identification of children at risk of ASD has become a key goal.

Though many recent studies demonstrate that autism has a genetic basis, the inheritance pattern of ASD in most families is highly complex. While genetic testing for autism has been limited to the identification of copy number variants (CNVs), autism-associated CNVs are found only in approximately 10% of children with ASD.

Researchers seeking an alternative approach to identify biomarkers for autism have focused on a number of common genetic variants--or SNPs --that have been shown to be related to the risk of ASD. While individual SNPs do not cause ASD, recent studies have shown that the presence of a combination of autism-associated SNPs can predict with a high degree of certainty whether a child will develop ASD.

"By looking at a combination of gender-specific, risk-associated, genetic common variants, we were able to identify siblings of children with ASD who have a significantly increased risk of developing autism," says lead author Francois Liebaert, MD, Vice President of Research and Development for IntegraGen, SA, Evry, France, "Earlier identification of siblings of children with autism at increased risk may lead to faster referrals, earlier diagnosis, earlier intervention and better prognosis. We also hope to replicate these findings in families that do not have a child with autism."

These findings build upon earlier identification of eight autism-related SNPs that occur in males and females (Autism risk assessment in siblings of affected children using sex-specific genetic scores) published the February 17, 2011 edition of Molecular Autism.

To determine which SNPs were associated with autism, researchers applied techniques that have been used to analyze other complex diseases. By combining statistical results from genome wide association studies (GWAS) with biological information from multiple sources including databases and scientific literature, the researchers were able to identify and prioritize the SNPs, and develop gender-specific genetic scores to predict the risk of autism.

The study comprised greater than 1,100 families which have more than one child diagnosed with ASD, referred to as multiplex families, including nearly 2,000 affected and 600 unaffected siblings. The male to female ratio for affected children was close to 4.2:1. The discovery cohort included 545 families from the Autism Speaks Autism Genetic Resource Exchange Repository (AGRE). The findings were then replicated in a population comprising 627 families including 339 families from a separate AGRE collection and DNA samples from 288 independent families collected at the University of Washington, Seattle and currently maintained at the University of Pennsylvania.

Original post:
Common genetic variants identify autism risk in high risk siblings of children with ASD

Recommendation and review posted by Bethany Smith