WASHINGTON (AP) Too often, newborns die of genetic diseases before doctors even know what's to blame. Now scientists have found a way to decode those babies' DNA in just days instead of weeks, moving gene-mapping closer to routine medical care.

The idea: Combine faster gene-analyzing machinery with new computer software that, at the push of a few buttons, uses a baby's symptoms to zero in on the most suspicious mutations. The hope would be to start treatment earlier, or avoid futile care for lethal illnesses.

Wednesday's study is a tentative first step: Researchers at Children's Mercy Hospital in Kansas City, Mo., mapped the DNA of just five children, and the study wasn't done in time to help most of them.

But the hospital finds the results promising enough that by year's end, it plans to begin routine gene-mapping in its neonatal intensive care unit and may offer testing for babies elsewhere, too while further studies continue, said Dr. Stephen Kingsmore, director of the pediatric genome center at Children's Mercy.

"For the first time, we can actually deliver genome information in time to make a difference," predicted Kingsmore, whose team reported the method in the journal Science Translational Medicine.

Even if the diagnosis is a lethal disease, "the family will at least have an answer. They won't have false hope," he added.

More than 20 percent of infant deaths are due to a birth defect or genetic diseases, the kind caused by a problem with a single gene. While there are thousands of such diseases from Tay-Sachs to the lesser known Pompe disease, standard newborn screening tests detect only a few of them. And once a baby shows symptoms, fast diagnosis becomes crucial.

Sequencing whole genomes all of a person's DNA can help when it's not clear what gene to suspect. But so far it has been used mainly for research, in part because it takes four to six weeks to complete and is very expensive.

Wednesday, researchers reported that the new process for whole-genome sequencing can take just 50 hours half that time to perform the decoding from a drop of the baby's blood, and the rest to analyze which of the DNA variations uncovered can explain the child's condition.

That's an estimate: The study counted only the time the blood was being decoded or analyzed, not the days needed to ship the blood to Essex, England, home of a speedy new DNA decoding machine made by Illumina, Inc. or to ship back the results for Children's Mercy's computer program to analyze. Kingsmore said the hospital is awaiting arrival of its own decoder, when 50 hours should become the true start-to-finish time.

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Two-day test can spot gene diseases in newborns

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KANSAS CITY, Mo., Oct. 3, 2012 /PRNewswire/ --Today investigators at Children's Mercy Hospitals and Clinics in Kansas City reported the first use of whole genome information for diagnosing critically ill infants. As reported in Science Translational Medicine, the team describes STAT-Seq, a whole genome sequencing approach - from blood sample to returning results to a physician - in about 50 hours. Currently, testing even a single gene takes six weeks or more.

Speed of diagnosis is most critical in acute care situations, as in a neonatal intensive care unit (NICU), where medical decision-making is made in hours not weeks. Using STAT-Seq, with consent from parents, the investigators diagnosed acutely ill infants from the hospital's NICU. By casting a broad net over the entire set of about 3,500 genetic diseases, STAT-Seq demonstrates for the first time the potential for genome sequencing to influence therapeutic decisions in the immediate needs of NICU patients.

"Up to one third of babies admitted to a NICU in the U.S. have genetic diseases," said Stephen Kingsmore, M.B. Ch.B., D.Sc., FRCPath, Director of the Center for Pediatric Genomic Medicine at Children's Mercy. "By obtaining an interpreted genome in about two days, physicians can make practical use of diagnostic results to tailor treatments to individual infants and children."

Genetic diseases affect about three percent of children and account for 15 percent of childhood hospitalizations. Treatments are currently available for more than 500 genetic diseases. In about 70 of these, such as infantile Pompe disease and Krabbe disease, initiation of therapy in newborns can help prevent disabilities and life-threatening illnesses.

STAT-Seq uses software that translates physician-entered clinical features in individual patients into a comprehensive set of relevant diseases. Developed at Children's Mercy, this software substantially automates identification of the DNA variations that can explain the child's condition. The team uses Illumina's HiSeq 2500 system, which sequences an entire genome at high coverage in about 25 hours.

Although further research is needed, STAT-Seq also has the potential to offer cost-saving benefits. "By shortening the time-to-diagnosis, we may markedly reduce the number of other tests performed and reduce delays to a diagnosis," said Kingsmore. "Reaching an accurate diagnosis quickly can help to shorten hospitalization and reduce costs and stress for families."

About Children's Mercy Hospitals and Clinics Children's Mercy Hospitals and Clinics, located in Kansas City, Mo., is one of the nation's top pediatric medical centers. The 333-bed hospital provides care for children from birth through the age of 21, and has been ranked by U.S. News & World Report as one of "America's Best Children's Hospitals" and recognized by the American Nurses Credentialing Center with Magnet designation for excellence in nursing services. Its faculty of 600 pediatricians and researchers across more than 40 subspecialties are actively involved in clinical care, pediatric research, and educating the next generation of pediatric subspecialists. For more information about Children's Mercy and its research, visit childrensmercy.org or download our mobile phone app CMH4YOU for all phone types. For breaking news and videos, follow us on Twitter, YouTube and Facebook.

About The Center for Pediatric Genomic Medicine at Children's Mercy Hospital The first of its kind in a pediatric setting, The Center for Pediatric Genomic Medicine combines genome, computational and analytical capabilities to bring new diagnostic and treatment options to children with genetic diseases. For more information about STAT-Seq, diagnostic tests and current research, visit http://www.pediatricgenomicmedicine.com.

Melissa Novak Phone: (816) 346-1341 E-mail: mdnovak@cmh.edu

Carin Ganz Phone: (212) 373-6002 E-mail: cganz@golinharris.com

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50-Hour Whole Genome Sequencing Provides Rapid Diagnosis for Children With Genetic Disorders

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Medicine appears poised to begin sequencing the entire genetic scripts of newborn babies with serious illnesses, a revolutionary change that was set in motion three years ago when scientists and doctors in Wisconsin used a similar technique to diagnose and treat a young Monona boy with a mysterious illness.

In a study released Wednesday in the journal Science Translational Medicine, researchers at Children's Mercy Hospitals and Clinics in Kansas City report that they used whole genome sequencing to diagnose babies born with serious genetic illnesses. Of the seven cases in which doctors used genome sequencing, six resulted in diagnoses.

Moreover, researchers said a diagnosis can be returned as quickly as 50 hours after a blood sample is taken from a baby, an important finding given that many of the diseases that afflict infants require very rapid treatment. That's much faster than the four to six weeks it had taken previously to go from sequencing to diagnosis.

Doctors at the Kansas City hospital said the test and accompanying analysis cost about $13,500 for each child and could present an appealing cost savings to health insurers. In the United States, thousands of babies each year with serious unknown diseases end up in the neonatal intensive care unit; there, beds cost some $8,000 a night, and total expenses for one child can easily run to $250,000 or more.

"We think this is going to transform the world of neonatology," said Stephen Kingsmore, an author of the new paper and director of the Center for Pediatric Genomic Medicine at Children's Mercy Hospitals and Clinics. Kingsmore said his hospital will be using sequencing routinely for seriously ill newborns by the end of the year and will perform the same service for other hospitals around the country.

"This is a dramatic, even miraculous development," said Philip M. Farrell, former dean of the University of Wisconsin-Madison Medical School. "It's the equivalent of putting a man on the moon as far as I'm concerned."

At Children's Hospital of Wisconsin and the Medical College of Wisconsin, where a similar newborn sequencing program quietly began two months ago, one of the doctors involved read the new paper and declared it "a huge leap forward.

"This is going to revolutionize our ability to take care of kids," added David Dimmock, a pediatric genetics specialist who worked on the team that sequenced young Nic Volker of Monona and crafted the treatment that appears to have saved the boy's life.

"The aim of this is to replace conventional testing with something that is faster and more comprehensive."

While the sequencing of Nic's genes in 2009 was used as a last resort after many other tests had been tried, the technology is now assuming a far more significant role in medicine. The hospital in Kansas City and Children's in Wisconsin are now using sequencing as a "first-line test," one that will save time and money over the current practice in which doctors hunt through a forest of individual tests for different diseases and mutations.

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Genetic sequencing gets faster, cheaper - and routine

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A new method of genetic testing appears to be able to help doctors diagnose critically ill babies more quickly than ever before, according to a new study.

The method allows doctors for decode a baby's entire genome in two days -- breathtakingly fast compared to current methods that can take six weeks or more.

In the new study, the researchers report using the approach to decode the entire genomes of six acutely ill newborns admitted to neonatal intensive care units, two of whom had already been determined to have genetic diseases. What they found in this proof of concept, they said, could be used in the future to more quickly diagnose sick newborns and treat them early.

The study was published Wednesday in the journal Science Translational Medicine.

"We think that we have come up with a solution for the tragic families who have a baby who's born and the doctors are not sure of what the cause of the baby's illness is," said the study's senior author, Dr. Stephen F. Kingsmore, director of the Center for Pediatric Genomic Medicine at Children's Mercy Hospitals and Clinics in Kansas City, Mo.

Many of the 3,500 known genetic diseases cause medical problems during the first month of life, the researchers wrote in their study. In the United States, over 20 percent of infant deaths are caused by genetic disorders and birth defects.

"Up to one third of babies admitted to a neonatal intensive care unit in the United States have genetic diseases," Kingsmore said, adding that babies with genetic problems often die or are sent home before a diagnosis is made.

For families coping with the tragedy of a sick newborn, the test may make a big difference.

"The family doesn't know what's going on," Kingsmore said. "The doctors are working heroically to figure out what's wrong. That can go on for weeks."

Armed with an early genetic diagnosis, Kingsmore said that doctors can communicate more clearly with the family.

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New, Faster Genetic Screen May Help Sick Babies

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For babies born with the rare genetic disorder phenylketonuria (PKU), their bodies are unable to break down a certain amino acid, which can lead to brain damage and seizures. If found early enough, however, PKU is easily treated, and children with the condition can go on to live a normal life. But sometimes, genetic testing for disorders such as this one come too late, and narrow windows of opportunity for treatment can close up for good.

But now, parents and physicians can have answers regarding a babys genetic abnormalities in only a few short days. Researchers from Childrens Mercy Hospitals & Clinics in Kansas City, Mo., have developed a new whole-genome sequencing technology capable of diagnosing genetic disorders in ICU newborns in just 50 hours a significantly less amount of time than the 12 to 14 days needed for current screening techniques.

The ability to diagnose infants in such a short amount of time could help to speed up available treatments as well as provide relief or knowledge to anxious parents.

There are about 500 diseases that can present in a baby for which theres a treatment, Dr. Stephen Kingsmore, director of the Center for Pediatric Genomic Medicine at Childrens Mercy Hospitals and Clinics and lead author of the study, told FoxNews.com. But for diseases that dont have treatment, this info can still be useful. It gives parents and physicians an answer. You can stop doing additional testing or stop giving futile treatments. Parents can get counseling about whether this can recur in a future child and get advice about how intense treatments can be.

Currently, there are more than 3,500 known genetic disorders conditions caused by a mutation in a single gene and the definitive method diagnose them is to sequence the mutated gene. However, a big problem with gene sequencing up until now has been knowing exactly which gene to sequence, according to the researchers. Each genome contains more than 3.1 billion nucleotides, and of those, three to four million variants exist. In order to diagnose a condition, all of those variants need to be analyzed a task that can take quite a long time.

To speed up this process, Kingsmore, along with fellow Childrens Mercy Hospital researcher Neil Miller, teamed up with the company Illumina a group dedicated to technologies that analyze genetic variations. Having announced in January the Illumina high-speed 2,500 a high-speed sequencing device, the company approached Kingsmore and Miller to develop software that would go hand-in-hand with their new instrument.

That was how SAGA and RUNE were born. After the Illumina high-speed 2,500 sequences the entire genome in less than 30 hours, the software applications then come into play. First, SAGA, which stands for sign-assisted genome analysis, helps physicians to determine which parts of the genome are significant depending on the patients symptoms.

It allows them to click on buttons of symptoms that are corresponding in the baby such as difficulty breathing, etc, Kingsmore said. The computer then matches those particular symptoms and signs to the right parts of the genome and selects of those 3,500 genetic diseases, which ones are appropriate to test. So it allows us to test the variants that are likely to cause a disease.

To determine how effective SAGA was in determining a diagnosis, the researchers used the program on over 500 previously diagnosed cases, and the software was 99 percent accurate in selecting the right gene according to the patients symptoms.

RUNE solves the second part of the puzzle, which is determining how these variants impact the gene in which they occur. Standing for rapid understanding of nucleotide-variant effect, RUNE essentially ranks the order of diseases that are on possibly on target for the variants that were found.

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Test can identify genetic disorders in newborns in days

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A new technology can diagnose rare genetic disorders in critically ill newborns within a few days, rather than the weeks that are needed with current methods, researchers say.

The technology involves sequencing the infant's genome, and then using new software to hone in on the genes most likely to be disease culprits.

In a new study, researchers identified the genetic cause of a newborn's illness in three out of four babies tested. The whole process takes about 50 hours, they said.

The speed of the new test is what could make it useful for sick babies in neonatal intensive care units (NICUs), the researchers said. Currently, it can take weeks for doctors to diagnose a genetic disorder in an ill infant, and many babies die before their test results are available, said study researcher Stephen Kingsmore, director of the Center for Pediatric Genomic Medicine at Children's Mercy Hospital in Kansas City.

A faster diagnosis for genetic conditions would allow doctors to provide earlier treatments if there are any or to give parents an earlier warning, and potentially more time together with their child, if the condition is untreatable and fatal, the researchers say.

Doctors already routinely screen newborns for a few genetic disorders that have effective treatments. But these tests look for single genes, rather than at the entire genome. There about 3,500 diseases known to be caused by mutations in a single gene, and 500 of these have some type of treatment available, Kingsmore said.

"By obtaining an interpreted genome in about two days, physicians can make practical use of diagnostic results to tailor treatments to individual infants and children," Kingsmore said.

However, critics point out that the diseases identified by new technology are rare, and extra genetic information is not always helpful. In fact, some are worried the genetic testing could deliver more information than researchers know what to do with.

Diagnosing genetic diseases

To begin a diagnosis with the new technology, the researchers take a drop of the baby's blood so that his or her genome can be sequenced.

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Newborn Genetic Test Catches Rare Diseases Earlier

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October 3, 2012

April Flowers for redOrbit.com Your Universe Online

Your intelligence like almost all other traits is a gift from your parents, at least in part. Scientists have known for a long time that intelligence is at least partially inherited through genetics. According to psychological scientist Christopher Chabris, however, it may be some time before researchers can identify the specific genetic roots of intelligence.

A new study from Union College shows that the genes long thought to be linked to intellectual prowess actually appear to have no bearing on ones IQ, complicating scientific endeavors to get to the root of the genetics of intelligence.

An international team of researchers including Harvard economist David Laibson used large data sets that included both intelligence testing and genetic data to examine a dozen genes. In almost every case, the team found that IQ could not be linked to the specific genes that were tested.

In all of our tests we only found one gene that appeared to be associated with intelligence, and it was a very small effect. This does not mean intelligence does not have a genetic component. It means its a lot harder to find the particular genes, or the particular genetic variants, that influence the differences in intelligence, said Chabris. The results of this new study were published online in the journal Psychological Science.

Previous studies of identical and fraternal twins informed and bolstered the notion that intelligence is a heritable trait. This new research validates that conclusion, yet the exact parameters of the genetics of intelligence remain a mystery. The team asserts that the older studies, which picked out specific genes, had flaws because of the technological limits of the time. Those limits prevented researchers from probing more than a few locations in the human genome to find genes that affected intelligence.

We want to emphasize that we are not saying the people who did earlier research in this area were foolish or wrong, Chabris said. They were using the best technology and information they had available. At the time, it was believed that individual genes would have a much larger effect they were expecting to find genes that might each account for several IQ points.

The team says that much more research is needed to determine the exact role that genes play in intelligence.

As is the case with other traits, like height, there are probably thousands of genes and their variants that are associated with intelligence, he said. And there may be other genetic effects beyond the single gene effects. There could be interactions among genes, or interactions between genes and the environment. Our results show that the way researchers have been looking for genes that may be related to intelligence the candidate gene method is fairly likely to result in false positives, so other methods should be used.

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Study Says Genetics Of Intelligence Remains A Riddle, For Now

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AMES, Iowa, Oct. 3, 2012 /PRNewswire/ --NewLink Genetics Corporation (NLNK) announces the launching of an open-label, randomized, multi-institutional adaptive design Phase2B/3 study to evaluate efficacy of its tergenpumatucel-L (HyperAcute Lung) product candidate in patients with progressive or relapsed Stage-IIIB/IV non-small cell lung cancer (NSCLC).

The Phase 2B portion of the study will evaluate two dosing schedules for tergenpumatucel-L versus docetaxel and the Phase 3 portion of the study will further assess efficacy of the selected dose against docetaxel. The primary endpoint of the study will be to evaluate survival in second-line therapy for patients with advanced non-small cell (stage IIIB/IV) lung cancer. Secondary objectives include progression free survival, evaluation of tumor response, and immunological response in treated patients.

"We are pleased to move another promising HyperAcute product candidate with encouraging survival data from Phase 2 into advanced-stage studies," commented Dr. Charles Link, Chairman and Chief Executive Officer of NewLink. "Non-small cell lung cancer remains the leading cause of cancer death in the United States."

"Immunotherapies are emerging as one of the most promising next treatment paradigms for cancer patients by allowing the patient's immune system to fight their disease without significant new toxicities. We are excited to participate in this advanced study to evaluate NewLink's innovative HyperAcute Lung immunotherapy in NSCLC," said principal investigator of the study Dr. Ramaswamy Govindan, Professor of Medicine, Co-Director Section of Medical Oncology at the Alvin J Siteman Cancer Center, Washington University School of Medicine, St. Louis, MO.

"This trial design is based on Phase 2 non-small cell lung cancer data presented at the recent ASCO meeting demonstrating 11.2 months median survival in 2nd and 3rd line patients who failed prior treatment. Our correlative immunological data showing 21.9 vs. 7 months survival in certain patients capable of generating IFN-gamma responses versus patients who did not mount this response, suggest patients with the best immune responses may have significantly greater long term overall survival. If these types of data can be confirmed in the new larger, randomized study an important novel therapy will be made available for patients with very limited options. We are delighted to be one of the lead centers," commented principal investigator for the Phase -2 study and Co-PI for the Phase 3 study Dr. John C. Morris, Professor of Medicine, Director of Experimental Therapeutics, Thoracic Cancer and Head & Neck Cancer Programs at University of Cincinnati, Cincinnati, OH.

Although a number of therapies have been approved in lung cancer, the prognoses for patients remain poor. "This study is designed to test the hypothesis that patients treated with HyperAcute immunotherapies may be sensitized to subsequent treatments with chemotherapy while also evaluating whether survival benefits observed in our Phase 2 study can be reproduced in a large controlled Phase 3 study," commented Dr. Nick Vahanian, President, Chief Medical Officer, NewLink Genetics.

Adaptive Study Design

This Phase 2B/3 study will enroll patients having a better baseline immune system status relative to the patient population in the earlier Phase 2 study. In order to be eligible for the study, patients must have Stage IIIB or Stage IV recurrent or treatment refractory non-small cell lung cancer with good performance status (ECOG <2) and no more than one prior chemotherapy failure. A lymphocyte count of >/= 1000/L, platelets >/= 100,000/L, hemoglobin >10.0 gm/dL, albumin >/= 3.0 gm/dL and acceptable hepatic and renal function are required for enrollment.

Two hundred forty (240) patients will be randomized (2:1:1) to receive: Arm 1: Docetaxel 75 mg/m2 intravenously given every 3 weeks for 4 doses; Arm 2a: Tergenpumatucel-L at 300 million cells given by intradermal injection weekly for 11 weeks then every 2 months for 5 additional doses (up to a total of 16 immunizations); Arm 2b: Tergenpumatucel-L at 300 million cells given by intradermal injection every 2 weeks for 6 doses and then every month for 10 additional doses (up to a total of 16 immunizations).

Phase 3 Study Design

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NewLink Genetics Launches Adaptive Design Phase 2B/3 Clinical Trial of tergenpumatucel-L Immunotherapy in Patients ...

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SALT LAKE CITY, Oct. 3, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced that, in support of National Hereditary Breast and Ovarian Cancer (HBOC) Week and National Previvor Day, it has launched an online quiz to help people assess their risk for hereditary cancers. The Hereditary Cancer Quiz is available online at http://www.hereditarycancerquiz.com. In addition, the company is providing financial support toward educational and awareness initiatives to three advocacy organizations-Bright Pink, the National Ovarian Cancer Coalition (NOCC) and Living Beyond Breast Cancer (LBBC).

HBOC Week marks the transition between National Ovarian Cancer Awareness Month and National Breast Cancer Awareness Month and was established by U.S. Congressional resolution in 2010 to raise awareness about hereditary cancer. National Previvor Day raises awareness for those individuals who have a known gene mutation or a strong family history of cancer but have not yet developed cancer.

"Understanding their risk for hereditary cancers, such as breast and ovarian cancer, is critical to helping patients make informed decisions about treatment and prevention. Our hereditary cancer risk quiz empowers patients to understand their family history and provides a framework for an informative discussion with a healthcare professional," said Mark Capone, President, Myriad Genetic Laboratories. "In addition, organizations such as Bright Pink, NOCC and Living Beyond Breast Cancer offer hereditary cancer patients and their families vital support and information in their fight against these diseases, and we are proud to support their efforts."

"Funding from Myriad and our other partner companies assists our organization in offering better and more valuable resources to hereditary cancer patients and their families," said David Barley, Chief Executive Officer, National Ovarian Cancer Coalition. "We are proud to work with Myriad, as they play a major role in the understanding and diagnosis of a person's hereditary risk for cancer."

About Hereditary Cancer

Hereditary cancers, also called inherited cancers, are those caused by genetic mutations that are passed from parent to child. These mutations predispose people to developing a particular type of cancer. Mutations in BRCA1 and BRCA2 genes are the most common cause of hereditary breast and ovarian cancers and can lead to male breast cancer, pancreatic cancer, prostate cancer and others. Women with a BRCA mutation are five times more likely to develop breast cancer than those without the mutation and more than ten times as likely to develop ovarian cancer1. Approximately 7%2 of breast cancer and approximately 14% 3,4,5 of invasive ovarian cancer result from inherited gene mutations.

DNA testing for BRCA mutations is done through a blood or saliva test and can indicate whether a person carries a BRCA gene mutation. Testing is recommended for people with certain personal and/or family history pattern, including:

Myriad Genetics is a pioneer in hereditary cancer testing and offers tests for a variety of hereditary cancer syndromes, including BRACAnalysis(R), which detects mutations in the BRCA1 and BRCA2 genes. This test has become the standard of care in identification of individuals with hereditary breast and ovarian cancer. Nearly one million patients have benefited from Myriad's hereditary cancer testing.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patients' 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

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Myriad Genetics Sponsors Cancer Awareness Initiatives in Support of National Hereditary Breast and Ovarian Cancer Week

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Newly published obesity research coming out of the University of Alberta has touched upon a possible avenue for obesity reduction.

Jason Dyck, a U of A medical researcher and professor, is the lead researcher for the study, which may be close to finding a new way to combat obesity.

In this study, mice are fed a high-fat, high-sugar diet the type of diet that generally leads to obesity. They then have adiponectin DNA injected into their leg muscles.

Adiponectin, secreted by fat cells, is known to have heart-protective and weight-regulating properties. If fat cells get too large, they no longer secrete normal amounts of the hormone-like substance.

What Dyck and his research team have found is the mice receiving this gene therapy start to produce adiponectin in higher levels, thus exhibiting weight loss.

Regardless of its high fat diet, the mouse is acting like a skinny mouse, Dyck said.

Treating obesity as an illness has raised some eyebrows in the Edmonton community, including radio show host Yukon Jack who said, if being fat is a disease, then playing bingo is a professional sport.

Dyck says this viewpoint probably reflects the majority of opinions about obesity. However, many doctors and medical professonals consider obesity a disease.

But this type of research has broader applications than just exercise-free weight loss. Obesity is closely linked with the loss of insulin sensitivity observed in Type 2 Diabetes.

What we are seeing with this gene therapy is a significant, but modest effect on weight gain. Despite only a slight reduction in weight gain, we are still seeing almost a complete restoration of insulin sensitivity, Dyck said.

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Gene therapy a possible option for obesity

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Is gene therapy finally becoming a reality? The European Commission is poised to authorize, for the first time in the Western world, the commercialization of a gene-therapy product. Called Glybera (alipogene tiparvovec), it is designed to treat a rare genetic defect involved in fat metabolism.

Success has been a long time coming. Gene therapy was first administered more than 20 years ago, to a child who had a rare disorder of the immune system called adenosine deaminase (ADA) deficiency. Since then, it has struggled to find its place in medicine amid a roller coaster of successes and setbacks, hype and scepticism that has little precedent in modern times. Although the approval of Glybera is a positive move, it is unlikely to herald a new age of gene therapies not without significant changes to the system. It is no coincidence that no gene therapy has yet been approved in the United States and that no other gene-therapy product is being considered by regulators in Europe.

Here is why. The design, development and manufacture of products such as Glybera a virus engineered to carry a correct copy of the defective gene is complex and done mostly in academic centres. Yet legislation introduced in the past decade in Europe and the United States demands that these products be produced under the same rules that cover conventional drugs, in establishments operated with industry-like standards and certified by government agencies.

This is a formidable challenge for academic centres, which tend to lack the necessary human and financial resources. So why is the development of gene therapy focused there, and not in industry, which seems better suited?

The first reason is the financial uncertainty generated by the complex, confused and poorly harmonized regulatory environment as the history of Glybera shows. At first, the application for its authorization received a negative opinion from two committees at the European Medicines Agency (EMA): the Committee for Advanced Therapies (CAT) and the Committee for Human Medicinal Products for Human Use (CHMP). Only when another body, the Standing Committee of the European Commission, asked the EMA to reconsider the application in a restricted indication did the CHMP eventually recommend approval under exceptional circumstances, requiring post-marketing studies and the set-up of a restricted-access programme. The Dutch firm Amsterdam Molecular Therapeutics, the inventor of Glybera, did not survive the process, and became known as uniQure after refinancing.

Lack of resources is a second reason. For many years, the drug industry stayed away from gene therapy, perceiving it as a dangerous technology of dubious efficacy that was too complex to develop and targeted too small a market.

There are some positive signs, because this last perception, at least, is changing: the industry now recognizes that rare diseases and orphan-drug legislation provide attractive opportunities. Some recombinant proteins and monoclonal antibodies originally developed as orphan drugs have been repurposed for larger indications.

The industry now recognizes that rare diseases and orphan-drug legislation provide attractive opportunities.

An example of how academia and industry could cooperate comes from the recent alliance between the drug giant GlaxoSmithKline (GSK) in London, and the charity-funded San Rafaelle Telethon Institute for Gene Therapy (TIGET) in Milan, Italy. GSK gained an exclusive licence to develop and commercialize the ADA treatment, and will co-develop with TIGET gene therapies for six more genetic diseases. The contribution of public or charity-funded organizations in early development phases lowers the cost and risk of investing in diseases with a tiny market, and gives the industry access to technologies that can be expanded to more profitable applications, thereby repaying the investment and allowing resources to be fed back into rare diseases. Unfortunately, promising therapies for hundreds of orphan diseases are unlikely to attract similar industrial interest.

So, how do we ensure that scientists will continue to develop such treatments? Should they all turn to the hospital exemption, which permits experimental therapies to be manufactured and used under the responsibility of a physician without regulatory supervision?

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Gene therapies need new development models

Recommendation and review posted by Bethany Smith

EMERYVILLE, Calif.--(BUSINESS WIRE)--

KineMed, Inc. (www.kinemed.com) announced today that David Fineman, KineMed, Inc.s President and CEO will present at the 2012 BIO Investor Forum on Wednesday, October 10th at 11:00 AM PST at the Palace Hotel in San Francisco, CA.

In a talk entitled Pairing Diagnostics with Therapeutics: Biomarker Solutions for Personalized Medicine, Mr. Fineman will share recent developments and breakthrough results from KineMeds powerful kinetic biomarker platform for interrogating the dynamic behavior of the human proteome and lipidome, and discuss how KineMeds exquisitely targeted translational biomarker repertoire is being used as the first functionally interpretable and clinically predictive systems biology platform that can be translated directly into humans.

The timing could not be better for a revolution in biomarker technology, commented Mr. Fineman, The rapid, inexorable shift of pharmaceutical development and patient care towards the Personalized Medicine model means that drugs must be paired with diagnostic biomarkers to enable the right drug to be selected for the right patient at the right time. This requires better targeting of disease subclasses: patient-specific diagnostics, as well as more disease subset-specific therapeutics. KineMeds dynamic signatures translate directly into humans and by virtue of being functionally interpretable and much closer to phenotype than other omics technologies, our measurements offer perhaps the most clinically predictive class of biomarkers yet developed, enabling greater public health benefits and better reimbursement rates for commercial providers.

A webcast of the presentation will be available on Wednesday, October 10th at 12:00 PM PST, one hour after Mr. Finemans live presentation via KineMeds website at http://www.kinemed.com/webcast

About KineMed, Inc.

KineMed, Inc., based in Emeryville, CA, is a drug and disease diagnostic developer, serving pharmaceutical clients and foundations with decision-critical information that accelerates, guides and de-risks drug development, to reduce cost and avoid attrition.KineMed is a partner of choice for drug developers seeking to develop the next generation of medicines that target the underlying causes rather than expressed symptoms of disease and is a field leader in pairing drugs with diagnostic biomarkers that offer Yes/No answers to critical patient management questions: Is this the right drug? Is the drug working?

KineMeds biomarkers are qualitatively and quantitatively different in the answers that they give, compared with other molecular diagnostics, because they are based on kinetic measurements - active measures that compare the rate of biochemical changes between healthy and disease states in a living animal or human. The companys unique, patented isotopic tracer technology tracks dynamic changes in key biochemical pathways that identify the complex biological processes underlying disease to predict clinical outcome. KineMeds biomarkers are fully translational from animal to man, seamlessly harmonizing data across pre-clinical and clinical phases of development, through to the diagnosis and management of diseases including fibrotic, metabolic, cardiovascular disease, and cancer.

KineMeds platform addresses key industry needs:

In addition to assisting clients, KineMed also has an active pipeline of therapeutics and diagnostics in development.

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Pairing Diagnostics with Therapeutics: KineMed to Present Biomarker Solutions for Personalized Medicine at the 2012 ...

Recommendation and review posted by sam

NEW YORK & PARIS--(BUSINESS WIRE)--

Bionest Partners, a strategy and management consulting firm for the life science industries, and a leader in personalized medicine (PM) strategy consulting, was invited to participate in the 8th Burrill Personalized Medicine Meeting, a key annual event in the world of PM held at San Francisco on September 13 and 14, 2012.

At the event, Dr. Sean X. Hu, Head of Bionest USA and Managing Partner, the invited speaker from Bionest, hosted a group of senior managers from leading molecular diagnostics (MDx) companies in a panel entitled First Generation PM Companies Where are they now? What can we learn? In addition, Dr. Hu served as an expert host during the Conversations with Experts Luncheon session and shared his expertise on how best to evaluate PM drug and diagnostic product strategies, and, optimize decision-making and implementation.

The panelists shared how their company strategies led to their success to-date, and discussed their views on the key trends and factors in the MDx industry that would impact their future success. Dr. Hu commented: The trend towards PM is inevitable yet remains highly challenging. A great deal of valuable learning emerged from our discussions at this Burrill conference.

For diagnostic companies, it is critical to leverage their core strengths and carefully place their bets by targeting specific unmet clinical needs before, at the time of, or after medical / drug intervention decisions. Depending on whether an MDx company develops a Laboratory-developed test (LDT) or an IVD kit, one must understand and effectively address specific challenges related to its business model, as well as regulatory, pricing, market access and other commercialization aspects across key geo markets.

Dr. Hu continued, For pharmaceutical companies, there is more to PM than simply knowing whether or not and how to incorporate patient stratification biomarkers (as companion diagnostics) into their drugs clinical development and commercialization strategies. Some cutting edge players have already begun to think beyond companion diagnostics and leverage other kinds of Dx products (e.g. disease diagnosis / definition, prognosis etc.) to enhance the value of their drug assets.

Bionest is a powerhouse in PM strategy consulting, serving the pharmaceutical, diagnostics, biotech and medical device industries. It is experienced in addressing a broad spectrum of challenges, from development and commercialization strategies for individual drug assets, diagnostics and other related products, to corporate level PM business models, commercialization capability building, R&D and commercialization business processes, and organizational structure.

Bionest has been driving thought leadership on PM, with many publications on the strategic, commercial and scientific aspects of PM. For more details, please visit http://www.bionest.com, and navigate to section Strategic/Practices/Personalized Medicine Strategy.

For further discussions with Dr. Hu and his Bionest team on PM strategies, please contact him at shu@bionest.com.

About Bionest Partners

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Bionest Hosted Discussions at the Burrill Personalized Medicine Conference

Recommendation and review posted by sam

BOSTON, Oct. 3, 2012 /PRNewswire-USNewswire/ -- Spaulding Rehabilitation Hospital announced today that both its Traumatic Brain Injury (TBI) Program and Burn Injury Rehabilitation Program have been selected as Model System sites by the National Institute on Disability and Rehabilitation Research (NIDRR). These selections combined with last year's selection of Spaulding as a Spinal Cord Injury Model System Site make Spaulding one of only two providers nationally to be selected as a Model System in all three specialties at the same time.

These grants are awarded in five year cycles by NIDRR through an extremely selective process with only 21 national sites for TBI and only 5 for Burn Injury. NIDRR awards Model Systems grants to institutions that are national leaders in medical research and patient care. Each site provides the highest level of comprehensive specialty services, from the point of injury through rehabilitation and community reentry. Dr. Joe Giacino will serve as Program Director for the TBI Model System Site and Dr. Jeffrey Schneider will serve as Program Director for the Burn Injury Model System site. Dr. Ross Zafonte will serve as Administrative Co-Director for both.

"Our selection by NIDRR as only the second provider in the nation to serve as a Model Systems Site in Burn Injury, TBI and SCI at the same time is a tremendous recognition for Spaulding and the Harvard Department of Physical Medicine and Rehabilitation. These grants will have an important impact on the communities Spaulding serves by allowing our talented clinicians to explore new avenues of research and care," said Dr. Ross Zafonte, VP of Research, Education and Medical Affairs, Spaulding Rehabilitation Network and Chairman of the Harvard Medical School Department of PM&R at Spaulding.

This funding will allow Spaulding clinicians and researchers to improve both regional and national understanding of TBI and Burn Injury treatment models. Each Model Systems site contributes to the national Model Systems Database for a better understanding of long-term health outcomes. Spaulding will also expand its participation in collaborative research and education as well as outreach efforts to inform individuals with TBI and Burn Injuries, their families and care givers, health care professionals and the general public about its work.

"These Model Systems recognitions by NIDRR are another acknowledgment of Spaulding's standing as a national leader in rehabilitative care and research. Most importantly for those recovering from or caring for someone with a traumatic brain injury or burn injury, these grants will greatly enhance the chance to improve their daily lives by empowering our researchers and clinicians with significant resources and support," said David Storto, President, Spaulding Rehabilitation Network.

About the Spaulding Rehabilitation Network

A member of Partners HealthCare, The SpauldingRehabilitation Network includes Spaulding Rehabilitation Hospital-Boston (main campus), a 196-bed facility, as well as Spaulding Rehabilitation Hospital Cape Cod. Additional locations include the two long-term care facilities Spaulding Hospital Cambridge and Spaulding Hospital North Shore andtwo skilled nursing facilities, as well as twenty-three outpatient sites throughout the Greater Boston area. Spaulding is a teaching hospital of Harvard Medical School as well as the official rehabilitation hospital of the New England Patriots. Spaulding is the only rehabilitation hospital in New England continually ranked since 1995 by U.S. News and World Report in its Best Hospitals survey with a #5 ranking in 2012. For more information, please visit http://www.spauldingrehab.org.

SOURCE Spaulding Rehabilitation Hospital

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Spaulding Selected by NIDRR as both TBI and Burn Injury Model System Sites

Recommendation and review posted by sam

NEW YORK, Oct. 3, 2012 /PRNewswire-USNewswire/ --United Spinal Association's membership division, National Spinal Cord Injury Association (NSCIA), will host two free webinars in October in recognition of National Disability Employment Awareness Month to provide people with disabilities useful information and resources for career building.

(Logo: http://photos.prnewswire.com/prnh/20110413/MM82757LOGO)

"Social Security work incentives, resume building and job interviewing skills for people with disabilities are webinar topics designed to equip job seekers with a solid foundation to realize their career goals," said Marlene Perkins, VP of Corporate and Community Relations at United Spinal.

The first webinar Understanding Social Security Work Incentives and Ticket to WorkOct. 11th at 3 p.m. to 4:30 p.m. EDT, will discuss Social Security work programs and incentives with featured speaker, Marlene A. Ulisky, Area Work Incentives Coordinator; North Florida; Social Security Administration (SSA).

Social Security's Ticket to Work Program is a free and voluntary program available to people who are ages 18 through 64 and receive Social Security Disability Insurance (SSDI) or Supplemental Security Income (SSI) benefits because they are disabled or blind.

The webinar will cover the benefits of employment, the responsibilities of a working beneficiary, how the Social Security Administration (SSA) recognizes work which is not reported, and how SSA applies the work incentives.

Attendees will also learn how to maintain cash benefits, and get the most out of Medicare and/or Medicaid.

The second webinar Employment Etiquette: Resume Building and Job Interview TipsOct. 24th at 3 p.m. to 4 p.m. EDT, will provide practical information on building the perfect resume and how to make the best impression on potential employers.

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United Spinal Presents Free Webinars For National Disability Employment Awareness Month

Recommendation and review posted by sam

CHICAGO, IL and PITTSBURGH, PA--(Marketwire - Oct 3, 2012) - Today, KineMatik announced that Stemnion, Inc., a global regenerative medicine company focused on the research, development and clinical use of its proprietary technology platform derived from human placental cells, has selected KineMatik's Electronic Laboratory Notebook (ELN) to deliver a research data management platform across its research organization. KineMatik is a leading global provider of a fully integrated solution that helps manage R&D at all levels of the product development lifecycle, keeping projects on budget and on schedule, while simultaneously providing the ability to meet stringent regulatory requirements for training and quality.

Stemnion will deploy KineMatik's ELN across several research divisions to streamline data capture, eliminate data silos, facilitate collaboration and information sharing, reduce complexity and speed time to research.

"After evaluating several ELN options available on the market, we chose KineMatik because of its extensive capability to perform document management in a traceable and compliant manner," commented George L. Sing, CEO."KineMatik's ELN will provide Stemnion an infrastructure for centralized research data management, enabling researchers to easily collaborate, and protect sensitive intellectual capital."

"We are pleased that Stemnion has joined the KineMatik family of very satisfied customers," remarked Michael Price, VP of Sales at KineMatik."We are certain that Stemnion will realize the same high-value as that of our other clients, including increased collaboration on research processes, reduced R&D costs through improved efficiencies, and enhanced protection of vital Intellectual Property."

KineMatik's ELN is a flexible and extensible platform based on world-standard ECM technology for the capture, searching and publishing of experimental data, capable of being fully validated. With superior integration capabilities to LIMS, CDS, instrumentation, SAP and SharePoint, the KineMatik ELN increases research productivity and efficiency by improving data accessibility, promoting knowledge sharing and reuse of data across an organization, minimizing duplication of efforts.

About Stemnion

Stemnion, Inc. is a privately held regenerative medicine company focused on the research, development and clinical use of its proprietary technology platform, derived from human placental cells. This platform provides an exciting new approach to wound healing and tissue repair in that it promotes rapid and better quality healing in a wide range of injuries. Stemnion's proprietary technology platform is derived from a unique population of cells isolated from full-term placental tissue that is normally discarded following delivery of a baby. There is no interruption in the development of the baby to procure this tissue, and therefore there are no ethical, religious, moral or political issues associated with their use. The Company has completed a Phase I human safety trial for the treatment of burns, and is currently planning additional Phase I and Phase II clinical trials.http://www.stemnion.com.

About KineMatik

KineMatik is a leading global provider of an Electronic Laboratory Notebook (ELN), Quality Management System (QMS), Laboratory Resource Management System (LRMS) and Project & Program Management (PPM) solutions based on industry standard software platforms including Open Text Content Server and Microsoft SharePoint Enterprise Content Management (ECM) technologies.KineMatik, with our expert Ph.D. Scientific Advisors, helps people in industry, government and academic organizations operate more collaboratively, manage their data more efficiently and be more productive in their daily tasks whether they are bench scientists, laboratory directors, project managers or quality and audit teams. KineMatik has been in business for over 12 years focused on helping make R&D organizations more efficient. For further information please visit us at http://www.kinematik.com.

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Stemnion Selects KineMatik ELN as Their Enterprise Research Data Management Platform

Recommendation and review posted by sam

ScienceDaily (Oct. 2, 2012) Regenerative-medicine researchers have moved a promising step closer to helping infertile, premenopausal women produce enough eggs to become pregnant. Surgeons at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine in Winston-Salem, NC, have now reported that they were able to stimulate ovarian cell production using an in vitro rat model, and observed as the cells matured into very early-stage eggs that could possibly be fertilized.

Results from this novel study were presented at the 2012 American College of Surgeons Annual Clinical Congress.

"While conventional hormone replacement therapy is able to maintain female sexual characteristics, it's unable to restore ovarian tissue function, which includes the production of eggs," the study's authors reported. Ovarian tissue function is critical for premenopausal women who desire to conceive.

Several fertility disorders can leave premenopausal women without an adequate amount of eggs. These disorders can also prevent a woman's ovaries from secreting enough of the hormones that stimulate egg production. Events such as ovarian operations, an injury, or radiation therapy for cancer can interfere with ovarian function, according to Anthony Atala, MD, FACS, director of the Wake Forest Institute for Regenerative Medicine and chair of the department of urology at the Wake Forest Baptist Medical Center.

Although the causes may vary, about 10 percent of childbearing-age women struggle with infertility, meaning that these women try for at least one year but are not able to conceive. The U.S. Centers for Disease Control and Prevention says that the most common cause of infertility in premenopausal women is polycystic ovarian syndrome -- an imbalance of sex hormones. This disorder causes irregular ovulation and higher levels of male hormones in affected women.

According to Dr. Atala, the goal of this study was to spur the ovaries to produce the female sex hormones estrogen and progesterone as well as stimulate egg production. The surgeons extracted ovarian cells from three-week old female rats, which would be equiva- lent to about 25 years old in humans. The cells were isolated in a culture of nutrient-dense growth factors for one week. Next, the cells were placed under a collagen gel that allows them to grow three dimensionally instead of in a single layer. The researchers then assessed cell growth, hormone production, and gene expression in the specimens.

In their early observations, the surgeons found immature oocytes protruding from clusters of ovarian cells. To help the oocytes mature, the surgeons developed a microwell system to keep oocytes inside clusters of ovarian cells. In humans, primordial germ cells or oogonium are the first stage of development into ovums, or mature eggs. The researchers also found that the cells expressed germ cell markers consistent with those of early stage eggs. They observed that the oocytes began to develop zona pellucida, a membrane that forms around an ovum as it develops, and showed a capacity to produce steroids similar to those produced by early stage eggs or follicles.

"Now, the goal is creating more mature structures that could actually be used for fertilization," Dr. Atala explained.

Dr. Atala and his colleagues believe that the newly generated oocytes would be able to mature to a certain stage in humans. The oocytes would then be put back into the female patient to go through natural ovulation and conception, or the oocytes would be fertilized in vitro and then implanted in the uterus. Dr. Atala said because ovarian cell function is restored, a woman using this procedure may be able to produce the necessary hormones and would not need addi-tional hormone replacement therapy.

Although the surgeons were able to generate early stage eggs in vitro, Dr. Atala cautions that the procedure has a while to go before it can be applied to humans: "This study represents the elementary, first stages of the research process," he said. "But we're showing the principle signs that this approach is a potential strategy for infertile women who want to have children," he concluded.

Continued here:
Surgeons recreate eggs in vitro to treat infertility

Recommendation and review posted by sam

LOS ANGELES--(BUSINESS WIRE)--

The producers of the longest running television health series American Health Journal, Windsor Broadcast Productions, are launching Innovations in Medicine, a new series to air on PBS SoCal. Produced by Windsor Broadcast Productions, the series will feature new developments, technology, procedures, and products in healthcare. The company is currently in production of its first six segments for the premiere 30-minute episode.

"Audiences have been demanding for this type of programming for years," said Executive Producer Roland Perez. "We regularly receive great feedback from stories we've produced on new medical equipment in beta testing that's not even FDA approved. People want to know whats going to be available to them."

With Innovations in Medicine Windsor will offer the first weekly show devoted to revealing compelling healthcare information previously available only from trade shows, healthcare insiders, medical journals and research newsletters.

Segments featured in the premiere episode include the glucose sensor company Dexcom and AVIIR Labs which focuses on advancing cardiovascular disease risk assessment, monitoring and an international stem cell story. The first episode of Innovations in Medicine is slated to premiere on SoCal PBS in November of 2012.

About Windsor Broadcast Productions

Founded in 1976, Windsor Broadcast Productions is located in Palm Desert, California. In 1988, they launched the nationwide syndicated program The American Health Journal which now reaches over 30 million homes. The American Health Journal has received over 92 national and international awards. The show is sponsored by Toshiba America and HF Healthcare.

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Windsor Broadcast Productions Launches New 30 Minute Series “Innovations in Medicine”

Recommendation and review posted by simmons

ST. LOUIS, Oct. 3, 2012 /PRNewswire/ --Sigma-Aldrich Corporation (SIAL) announced today that Sigma Life Science, its innovative biological products and services research business, has launched Stemline Pluripotent Culture Medium, a novel human pluripotent stem cell culture medium that provides a consistent environment for the long-term maintenance and growth of healthy pluripotent stem cells. The new medium performs equivalently to the industry's leading medium and provides academic and pharmaceutical stem cell research labs with a substantially lower cost alternative to higher priced media. Additional information and sample requests of the Stemline Pluripotent Culture Medium are available at http://www.sigma.com/stemlinepsc.

"The exorbitant cost of media for pluripotent stem cells is a universal complaint from the stem cell research community. Our Stemline Pluripotent Culture Medium performs equivalently to the leading medium for maintaining pluripotency and optimal growth rates, and is produced more efficiently than traditional media, resulting in lower costs. For example, a typical academic lab that consumes three 500 mL bottles of media per week could save at least $12,000 annually using our new Stemline medium. A high-throughput pharmaceutical development team that consumes 20 liters of media weekly could save more than $160,000 annually," said John Listello, Market Segment Manager for Regenerative Medicine at Sigma Life Science.

Culturing pluripotent stem cells can be challenging as many media's undefined, heterogenous mixtures can cause inconsistent growth rates and undesired spontaneous differentiation. The Stemline Pluripotent Stem Cell Culture Medium is serum-free, composed of fully-defined components and has 80% less basic fibroblast growth factor than the leading pluripotent stem cell culture medium. This provides a consistent environment for long-term maintenance of optimal growth rates, viability and pluripotency. Rigorous characterization of the Stemline Pluripotent Stem Cell Culture Medium has demonstrated that cultured pluripotent stem cells display all established pluripotency markers and maintain proper karyotype and the ability to differentiate into each of the three germ layers. The feeder-independent medium also enables culturing with synthetic matricies, thereby eliminating a source of variability that would prohibit later clinical applications.

"Academic and pharmaceutical groups performing toxicology screens, disease-specific stem cell research or studies of the basic mechanisms behind pluripotency and differentiation depend upon a steady supply of consistent, high-performance cell culture medium. This novel Stemline medium extends Sigma's existing position as one of the largest global providers of cell culture media," said Listello.

Existing Stemline stem cell culture media include specialized formulations for expansion of six human adult stem cell and progenitor cell types: hematopoietic, neural, dendritic, mesenchymal, T-cells, and keratinocytes. These six Stemline media are produced under good manufacturing practices (GMP) and have Device Master File certificates from the U.S. Food and Drug Administration.

Sigma Life Science's comprehensive stem cell product portfolio includes custom iPS cell CompoZr ZFN-mediated genetic engineering, Stemgent Reprogramming Lentiviruses, the MISSION shRNA Library with the latest content release from The RNAi Consortium, 3D matrices, growth factors, small molecules, other cell culture media and the industry's most validated antibodies. Sigma Life Science acquired a worldwide license to Kyoto University's iPS cell patent portfolio in February, 2012.

For more information and to request pricing, visit http://www.sigma.com/stemlinepsc.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "could," "could expect," "can be," "predictive" or similar expressions, or by expressed or implied discussions regarding potential future revenues from products derived there from. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that pluripotent stem cells, pluripotent stem cell media, or related custom services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated with pluripotent stem cells, pluripotent stem cell media, or related custom services could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; the Company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Company's assets and liabilities as recorded in its consolidated balance sheet, and other risks and factors referred to in Sigma-Aldrich's current Form 10-K on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Sigma-Aldrich is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Sigma Life Science: Sigma Life Science is a Sigma-Aldrich business that represents the Company's leadership in innovative biological products and services for the global life science market and offers an array of biologically-rich products and reagents that researchers use in scientific investigation. Product areas include biomolecules, genomics and functional genomics, cells and cell-based assays, transgenics, protein assays, stem cell research, epigenetics and custom services/oligonucleotides. Sigma Life Science also provides an extensive range critical bioessentials like biochemicals, antibiotics, buffers, carbohydrates, enzymes, forensic tools, hematology and histology, nucleotides, amino acids and their derivatives, and cell culture media.

About Sigma-Aldrich: Sigma-Aldrich is a leading Life Science and High Technology company whose biochemical, organic chemical products, kits and services are used in scientific research, including genomic and proteomic research, biotechnology, pharmaceutical development, the diagnosis of disease and as key components in pharmaceutical, diagnostics and high technology manufacturing. Sigma-Aldrich customers include more than 1.3 million scientists and technologists in life science companies, university and government institutions, hospitals and industry. The Company operates in 38 countries and has nearly 9,100 employees whose objective is to provide excellent service worldwide. Sigma-Aldrich is committed to accelerating customer success through innovation and leadership in Life Science and High Technology. For more information about Sigma-Aldrich, please visit its website at http://www.sigma-aldrich.com.

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Sigma® Life Science Launches Novel, Affordable Pluripotent Stem Cell Culture Medium

Recommendation and review posted by simmons

The Human Frontier Science Program Organization has awarded Stephen Quake the 2013 Nakasone Award for his work developing technologies in biophysics, biological automation, genome analysis, and personalized medicine. Quake is a professor of bioengineering and a Howard Hughes Medical Institute investigator at Stanford University. He is also a co-founder of Helicos Biosciences and Fluidigm.

Personalized medicine and molecular diagnostics firm Nodality has appointed pharmaceutical, biotech, and venture capital executive Laura Brege to serve as company president and CEO. Brege formerly was executive VP and COO at Onyx Pharmaceuticals, and she was a general partner at Red Rock Capital Management. She also was senior VP and CFO at COR Therapeutics, CFO at Flextronics, and treasurer of The Cooper Companies.

The National Center for Advancing Translational Sciences has named Chris Austin to be the first director of the new center, which launched at the beginning of the year and has so far been run by Acting Director Thomas Insel. Austin began his National Institutes of Health career in 2002, after working as a genomics researcher at Merck, as a senior advisor to the director for translational research at the National Human Genome Research Institute.

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NantHealth, Blue Shield Partner on Personalized Medicine Venture

Recommendation and review posted by sam

BOSTON--(BUSINESS WIRE)--

Third Rock Ventures, LLC, a venture capital firm focused on building life sciences companies, today announced that Felix W. Frueh, Ph.D., has joined Third Rock as an entrepreneur-in-residence (EIR). As an EIR, Dr. Frueh will provide strategic input on new and existing portfolio companies with a particular focus on personalized medicine.

We are pleased to welcome Felix to our growing team of industry leaders, said Alexis Borisy, partner of Third Rock Ventures. Since the founding of Third Rock, we have been dedicated to launching companies focused on personalized medicine, and Felix is the ideal person to help us continue to grow and develop those companies in our portfolio and discover new opportunities to invest in the space.

Dr. Frueh is a thought leader in personalized medicine with 15 years of R&D, management and policy experience. Most recently, Dr. Frueh served as president of the Medco Research Institute, leading Medcos real-world, outcomes-oriented research initiatives and collaborations after having formed Medcos personalized medicine research and development organization. Prior to joining Medco, Dr. Frueh was associate director for genomics at the U.S. Food and Drug Administration (FDA), where he built and led the core genomics review team in the Center for Drug Evaluation and Research (CDER) and chaired the first FDA-wide, interdisciplinary pharmacogenomics review group (IPRG). Before joining the FDA, he held senior positions at several biotechnology companies. Dr. Frueh has been a member of various working groups on genetics and genomics at the FDA and Department of Health and Human Services (DHHS). He serves on the board of the Personalized Medicine Coalition and is also a board member at Enterome Biosciences. He is an adjunct faculty member at the Institute for Pharmacogenomics and Individualized Therapy (IPIT) at the University of North Carolina (UNC) and held faculty appointments in the Departments of Pharmacology and Medicine at Georgetown University in Washington, D.C. He was a postdoctoral fellow at Stanford University and the University of Basel, Switzerland, where he also received his Ph.D. in biochemistry.

Throughout my career, I have had the opportunity to see new technologies and innovative approaches in personalized medicine make an impact on the lives of patients, said Dr. Frueh. I believe that Third Rocks approach to investing in cutting edge science has the potential to make a tremendous difference, and I look forward to being a part of that effort.

About Third Rock Ventures

Third Rock Ventures is a venture capital firm founded in 2007 with the mission to launch transformative life sciences companies. With more than $800 million and two funds under management, the firm is focused on working with passionate entrepreneurs to build exceptional companies working in areas of disruptive science that will make a difference in the lives of patients. The firm has assembled a team with deep expertise and a proven track record of building respected and successful life sciences companies. With decades of complementary, cross-functional operational and leadership experience, the Third Rock team actively engages with its portfolio companies to provide hands-on strategy and experience to successfully launch companies with the best vision, science, operations, people and culture. With offices in Boston, MA and San Francisco, CA, Third Rock is well positioned geographically to closely collaborate with its portfolio companies to achieve their goals. To learn more about Third Rock and its portfolio companies, please visit http://www.thirdrockventures.com.

Current Third Rock Portfolio Companies

Ablexis, Afferent Pharmaceuticals, Agios Pharmaceuticals, Alcresta, Allena Pharmaceuticals, Alnara Pharmaceuticals (acquired by Eli Lilly & Co. in 2010), bluebird bio, Blueprint Medicines, Constellation Pharmaceuticals, CytomX Therapeutics, DC Devices, Edimer Pharmaceuticals, Eleven Biotherapeutics, Ember Therapeutics, Foundation Medicine, Global Blood Therapeutics, Igenica, Kala Pharmaceuticals, Lotus Tissue Repair, MyoKardia, Nine Point Medical, PanOptica Pharmaceuticals, Rhythm Pharmaceuticals, SAGE Therapeutics, Seventh Sense Biosystems, Taris Biomedical, Topica Pharmaceuticals, Warp Drive Bio and Zafgen.

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Third Rock Ventures Appoints Personalized Medicine Expert Felix W. Frueh, Ph.D., as Entrepreneur-in-Residence

Recommendation and review posted by sam

IRVINE, Calif.--(BUSINESS WIRE)--

Unite 2 Fight Paralysis (U2FP) is pleased to offer an outstanding list of speakers for its 7th annual Working 2 Walk Science & Advocacy Symposium, a leading educational conference focused on research aimed at repairing the chronic spinal cord injury (SCI). The 2012 conference will be held November 1-2, 2012, at the Hilton Irvine/Orange County Airport in Irvine, California.

The Reeve-Irvine Research Center is a co-organizer of the Symposium, and its team of top-notch research scientists will be presenting the results of their latest work. Speakers from RIRC include Drs. Aileen Anderson, Leif Havton, Hans Keirstead, and the Centers Director, Dr. Oswald Steward.

In addition, scientists from around the country who are working to solve various pieces of the SCI neurological puzzle will share their progress. Included are: Drs. Justin Brown and Mark Tuszynski, University of California, San Diego; Dr. Jerry Silver, Case Western Reserve University; Dr. Ravi Bellamkonda, Georgia Institute of Technology; and Dr. Murray Blackmore, Marquette University.

The Working 2 Walk symposium offers a unique opportunity in the spinal cord injury community, bringing scientists together with investors, practitioners, and advocates in a supportive and strategic environment. Speakers from these segments of the community include: Jonathon Thomas, JD, PhD, California Institute for Regenerative Medicine; Stephen Huhn, MD, Stem Cells, Inc.; Ida Cahill, President and CEO, Sam Schmidt Paralysis Foundation; Damodar Thapa, PT, Kathmandu, Nepal; Frank Reynolds, InVivo Therapeutics; and Anthony Caggiano, MD, PhD, Acorda Therapeutics.

Unite 2 Fight Paralysis works hard to build the knowledge base and resources for those living with SCI, leading to informed advocacy efforts and wise investment in research. Advocates Dennis Tesolat, Bob Yant, and Roman Reed will speak to the importance of community advocacy during the conference.

Unite 2 Fight Paralysis was founded and is led by people who have a personal connection to spinal cord injury. U2FPs staff and Board of Directors are comprised of SCI survivors and family members who are determined to accelerate the progress of science toward achieving regeneration and repair of the injured spinal cord.

For more information: http://www.u2fp.org or http://www.working2walk.org.

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Unite 2 Fight Paralysis Announces Working 2 Walk Symposium Agenda

Recommendation and review posted by sam

Tuesday, October 2, 2012 - 09:00

A Thunder Bay regenerative medicine company is asking for another $300,000 from the city to help it survive as it continues to seek sustainable provincial funding. ReGen Medicine, which provides bone and tissue for use in transplants, is requesting the money flow at a rate of $50,000 a month for the next six months. And, board chairwoman Judy Sander said Monday, this is the last time theyll ask. If the $300,000 requested Monday night is provided, the city will have given $1.5 million to the company since 2005, including an interest-free loan of $250,000 in May. The money stopped flowing from the May loan last month. I had hoped at that time that . . . I would never be back in this seat again, Sander told council. This has definitely been a long haul. It is definitely not a risk-free operation. This environment, the health care field, is extremely challenging, she said. It definitely takes time and money and energy to reach success. Were very close, but were not there yet. The money would help expand the business more sales people are needed so the company can compete with other tissue banks, and more senior managerial support is required, board member Yves Fricot said while ReGen Medicine works to secure government funding. The company is working on a business sustainability report at the request of the Ministry of Health; the report is to be completed later this month. The report, Sander and Fricot said, is intended to identify what the company needs to be sustainable, and build its case for provincial funding. And if after all that the government is still not involved, Sander said it may be time to give up on the dream. But Fricot said the company is providing an essential service, and the government is simply trying to figure out where ReGen Medicine will fit within the provincial system. The donor list is growing, and the fact that ReGen Medicine is providing the service in Canada is saving the Ontario health care system $16-20 million a year. Plus, theyre helping head off what he said is a looming shortage of tissue and bone for transplant. If you dont work to develop and ultimately support this organization, youre going to have a problem that you shouldnt have, Fricot said. Its true on a personal level for people who need help and cant find it in Canada, (and its) going to be true in the long run as the American economy changes and (U.S. tissue banks) go back to filling their own needs. Council didnt vote on the request, but asked city administration prepare a report on the request. Also Monday night, council heard a presentation from the family of the late Suzan Labine who asked to have the playground portion of Junot Park renamed Suzan Labine Childrens Playground. Labine died Dec. 28, 2010, at age 58. She was a highly respected member of the community, serving on the board of many community organizations and agencies, including Westminster United Church and St. Josephs Care Group. Labine was also chairwoman of the Lakehead Public School Board for six years, and was co-ordinator of the United Ways Leaders of the Way campaign. Council directed administration to examine the request and report back at a future meeting.

Continued here:
ReGen Medicine asks city council for another $300,000

Recommendation and review posted by sam

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

Contact: Sandy Van sandy@prpacific.com 808-526-1708 Cedars-Sinai Medical Center

LOS ANGELES (Oct. 2, 2012) Researchers at Cedars-Sinai's Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLOS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetic's own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods transplantation into the organ or injection into the blood have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells pancreas cells that produce insulin or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

"Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes," said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yu's lab as an endocrinology fellow, is the article's first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

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Cedars-Sinai study sheds light on bone marrow stem cell therapy for pancreatic recovery

Recommendation and review posted by Bethany Smith

ScienceDaily (Oct. 2, 2012) Researchers at Cedars-Sinai's Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLoS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetic's own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods -- transplantation into the organ or injection into the blood -- have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells -- pancreas cells that produce insulin -- or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

"Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes," said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yu's lab as an endocrinology fellow, is the article's first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

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New study sheds light on bone marrow stem cell therapy for pancreatic recovery

Recommendation and review posted by Bethany Smith