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.

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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.

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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

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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

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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.

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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

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Plexpress and SBW Partner to Offer TRAC Gene Expression Analysis

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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.

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

Recommendation and review posted by Bethany Smith

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

Contact: Raymond MacDougall macdougallr@mail.nih.gov 301-402-0911 NIH/National Human Genome Research Institute

People have increasing opportunities to participate in genetic testing that can indicate their range of risk for developing a disease. Receiving these results does not appreciably drive up or diminish test recipients' demand for potentially costly follow-up health services, according to a study performed by researchers at the National Institutes of Health and colleagues at other institutions.

The study in the May 17, 2012 early online issue of Genetics in Medicine was done by investigators with the Multiplex Initiative, a multi-center collaborative initiative involving investigators from the National Institutes of Health's Intramural Research Program, Group Health Cooperative in Seattle, and the Henry Ford Health System in Detroit.

The tests are available from a growing number of commercial producers, and health care providers have been uncertain whether people who received information only about risk would follow up by demanding diagnostic testing to monitor for predicted illnesses.

The study is the first to use electronic health records -- rather than self-reported behavior -- to measure the impact of genetic testing on the subsequent consumption of health services by commercially insured, healthy adults. Self reports, which can be affected by memory lapses and other problems, tend to be less accurate.

"We need to understand the impact of genomic discoveries on the health care system if these powerful technologies are going to improve human health," said Dan Kastner, M.D., Ph.D., scientific director and head of the National Human Genome Research Institute's (NHGRI) Division of Intramural Research. "We are still learning how to integrate new genomic discoveries into clinical care effectively and efficiently."

"There are a lot of unanswered questions about how genetic test results can be used to guide people towards making positive lifestyle and health behavior changes," said Colleen McBride, Ph.D., chief of NHGRI's Social and Behavioral Research Branch. "This study goes a long way towards bringing data to these debates and shows that people are not likely to make inappropriate demands of health delivery systems if they are properly informed about the limitations of genetic tests."

Genetic tests, such as those used in this study, can detect common variants of genes associated with modest alterations in the chances of developing particular diseases. The term multiplex refers to simultaneously performing multiple genetic tests on a single blood sample.

The study included 217 healthy people between the ages of 25 and 40 who elected to participate in genetic susceptibility testing offered by their health plan. The researchers analyzed health care usage by the participants in the 12 months before genetic testing and the 12 months following the testing. They also compared the test group's behavior with a group of about 400 similar plan members who declined the testing offer.

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NIH-led study finds genetic test results do not trigger increased use of health services

Recommendation and review posted by Bethany Smith

Medical experts feared personal genetic test results might drive overuse of expensive medical care

The study in the May 17, 2012 early online issue of Genetics in Medicine was done by investigators with the Multiplex Initiative, a multi-center collaborative initiative involving investigators from the National Institutes of Health's Intramural Research Program, Group Health Cooperative in Seattle, and the Henry Ford Health System in Detroit.

The tests are available from a growing number of commercial producers, and health care providers have been uncertain whether people who received information only about risk would follow up by demanding diagnostic testing to monitor for predicted illnesses.

The study is the first to use electronic health records -- rather than self-reported behavior -- to measure the impact of genetic testing on the subsequent consumption of health services by commercially insured, healthy adults. Self reports, which can be affected by memory lapses and other problems, tend to be less accurate.

"We need to understand the impact of genomic discoveries on the health care system if these powerful technologies are going to improve human health," said Dan Kastner, M.D., Ph.D., scientific director and head of the National Human Genome Research Institute's (NHGRI) Division of Intramural Research. "We are still learning how to integrate new genomic discoveries into clinical care effectively and efficiently."

"There are a lot of unanswered questions about how genetic test results can be used to guide people towards making positive lifestyle and health behavior changes," said Colleen McBride, Ph.D., chief of NHGRI's Social and Behavioral Research Branch. "This study goes a long way towards bringing data to these debates and shows that people are not likely to make inappropriate demands of health delivery systems if they are properly informed about the limitations of genetic tests."

Genetic tests, such as those used in this study, can detect common variants of genes associated with modest alterations in the chances of developing particular diseases. The term multiplex refers to simultaneously performing multiple genetic tests on a single blood sample.

The study included 217 healthy people between the ages of 25 and 40 who elected to participate in genetic susceptibility testing offered by their health plan. The researchers analyzed health care usage by the participants in the 12 months before genetic testing and the 12 months following the testing. They also compared the test group's behavior with a group of about 400 similar plan members who declined the testing offer.

The researchers counted the number of physician visits and laboratory tests or procedures the people received, particularly those services associated with four of the eight conditions tested by the multiplex panel. Most of the procedures or screening tests that were counted are not among those currently recommended for people in this age group who don't have symptoms. The researchers found that participants in genetic testing did not change their overall use of health care services compared with those not tested.

All of the individuals who elected to undergo the multiplex test carried at least one at-risk genetic marker, with the majority carrying an average of nine at-risk variants. The tests performed for the Multiplex Initiative include a set of genetic variants reliably associated with an increase in disease risk and for which some corrective health behavior has been shown to prevent illness.

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Genetic Test Does Not Trigger Increased Use of Health Services

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May 18, 2012

Connie K. Ho for RedOrbit.com

Recently, genetic tests have been on the rise and are offered by more and more commercial producers. While there are many opportunities for people to participate in genetic testing, this doesnt necessarily mean that people are following up on those services. These are the results found in a study that is published in the May 17 issue of Genetics in Medicine.

The study was a collaborative project by the Multiplex Initiative, which includes researchers from the National Institutes of Healths Intramural Research Program, the Group Health Cooperative, and the Henry Ford Health System. Researchers analyzed variants of genes related to diseases such as colorectal cancer, coronary heart disease, high blood cholesterol, hypertension, lung cancer, melanoma, osteoporosis, and type 2 diabetes. The study allowed researchers to better understand patients health care needs.

Our study was a best-case scenario, because we chose 15 genes reliably associated with relatively small risks for eight common diseases that health behaviors can affect, remarked lead author Dr. Robert J. Reid in a prepared statement. We hope that testing positive activates patients to make behavior changes that could lower their risk, such as quitting smoking without causing them to make many extra visits to their doctors.

The report was one of the first studies that looked at electronic health records, as opposed to self-reported behavior, to quantify the impact of genetic testing on health services chosen by adults.

We need to understand the impact of genomic discoveries on the health care system if these powerful technologies are going to improve human health, explained Dr. Dan Kastner, scientific director and head of the National Human Genome Research Institutes (NHGRI) Division of Intramural Research, in a statement. We are still learning how to integrate new genomic discoveries into clinical care effectively and efficiently.

Genetic tests are also important because they can find variants of genes related to the changes in the risk of developing a particular disease.

Understanding personalized genetic information is important because it is becoming more readily available and we need to figure out how to integrate it effectively and efficiently into the clinical care we provide, noted coauthor Dr. Eric B. Larson in a prepared statement.

The scientists hope to continue to research consumer interest of genetic testing and how that affects patients long-term health care goals.

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Few Use Follow Up Services After Genetic Testing

Recommendation and review posted by Bethany Smith

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

Contact: Rebecca Hughes hughes.r@ghc.org 206-287-2055 Group Health Research Institute

SEATTLEPeople have more and more chances to participate in genetic testing that can indicate their range of risk for developing a disease. Receiving these results does not appreciably drive up or diminishtest recipients' demand for potentially costly follow-up health services, according to a new study in the May 17, 2012 early online issue of Genetics in Medicine.

The study was done by researchers with the Multiplex Initiative, a multi-center collaborative initiative involving investigators from the National Institutes of Health's Intramural Research Program, Group Health Cooperative in Seattle, and the Henry Ford Health System in Detroit.

The tests are available from a growing number of commercial producers, and health care providers have been uncertain whether people who received information only about risk would follow up by demanding diagnostic testing to check for predicted illnesses.

The study is the first to use electronic health recordsrather than self-reported behaviorto measure the impact of genetic testing on the subsequent use of health services by commercially insured, healthy adults. Self-reports, which can be affected by memory lapses and other problems, tend to be less accurate.

"Our study was a best-case scenario, because we chose 15 genes reliably associated with relatively small risks for eight common diseases that health behaviors can affect," said the study's first author Robert J. Reid, MD, PhD. Dr. Reid is Group Health's associate medical director of research translation and an associate investigator at Group Health Research Institute. Those diseases were type 2 diabetes, coronary heart disease, high blood cholesterol, hypertension, osteoporosis, lung cancer, colorectal cancer, and melanoma. "We hope that testing positive activates patients to make behavior changes that could lower their risk, such as quitting smoking," he added, "without causing them to make many extra visits to their doctors."

"Understanding personalized genetic information is important because it is becoming more readily available and we need to figure out how to integrate it effectively and efficiently into the clinical care we provide," said coauthor Eric B. Larson, MD, MPH, Group Health Cooperative's vice president for research and Group Health Research Institute's executive director.

"There are a lot of unanswered questions about how genetic test results can be used to guide people toward making positive lifestyle and health behavior changes," said Colleen McBride, PhD, chief of the Social and Behavioral Research Branch at the National Human Genome Research Institute (NHGRI). "This study goes a long way toward bringing data to these debates and shows that people are not likely to make inappropriate demands of health delivery systems if they are properly informed about the limitations of genetic tests."

Genetic tests, such as those used in this study, can detect common variants of genes associated with modest changes in the chances of developing particular diseases. "Multiplex" means simultaneously performing many genetic tests on one blood sample.

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Genetic testing may not trigger more use of health services

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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

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

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(Phys.org) -- By investigating the existence of an unusual four-stranded structure of DNA in human cells, scientists have opened the door to novel cancer therapeutics and a new era for personalised medicine.

When Watson and Crick discovered the double helix structure of DNA in 1953, they declared they had found the secret of life. However, as in all pursuits of science, the story did not end there. Less than 60 years later, a team led by chemist Professor Shankar Balasubramanian and cancer biologist Professor Steve Jackson has found that an unusual four-stranded configuration of DNA also forms at sites across the human genome in living cells.

Although known about by scientists for decades, the structure was considered to be something of a structural curiosity rather than a feature found in nature. It forms in regions of DNA that are rich in one of its building blocks, guanine (G), when a single strand of the double-stranded DNA loops out and doubles back on itself, forming a four-stranded handle in the genome.

G-quadruplexes have been known to occur at the ends of chromosomes in the regions known as telomeres, but it wasnt until a strong association had been noticed with genes responsible for cell proliferation that Balasubramanian and others began to suspect that G-quadruplexes might be a potential target for cancer therapy. If you synthesize a quadruplex-binding molecule and put it into cancer cells, it can impair the growth of these cells, he said. Weve come such a long way from thinking that we understand the genome and it appeared that this structure could tell us something new.

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Protecting the genetic code

At the heart of the new discovery is an innovative way of locating the structures in living cells and then capturing them for further examination. The scientists discovered that when pyridostatin binds to G-quadruplex DNA it causes a double-strand break in the double helix when the cell tries to replicate and copy its genes: Pyridostatin binding to G-quadruplexes is a major impediment to copying the genome so if a cell tries to replicate, this will generate breaks in the DNA, said Jackson.

Over the years, Jacksons lab has found that there are certain proteins in the cell that act as molecular policemen, patrolling the nucleus of the cell looking for damaged DNA. If they detect damage, they start making repairs, and at the same time set off alarm signals to alert the rest of the cell that theres a problem.

When there is no DNA damage, these molecular policemen are distributed evenly throughout each cells nucleus. But when cells are treated with pyridostatin, they congregate in specific locations on the DNA, indicating regions of damage, and showing up as dots under the microscope. The field really jumped on the idea that these dots represent telomeres that have G-rich sequences and in vitro have the potential to form G-quadruplexes, said Jackson. But we stained the dots for telomere proteins and found there was only a small amount of overlap. So clearly, this pyridostatin compound is inducing DNA damage in lots of other places, and we were faced with the issue: if they are not telomeres, what are they?

This confirmed an earlier finding in Balasubramanians lab by Julian Huppert, then a PhD student, who devised a computational search algorithm to map out every spot in the entire human genome that had potential to fold into a G-quadruplex. He found there were close to 350,000 of them.

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A new dimension to DNA and personalized medicine of the future

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

Bionest Partners, a premier strategy and management consulting firm for the life science industries, and a recognized leader in personalized medicine (PM) strategy consulting, announces the publication of an article in a recent issue of Nature Reviews Drug Discovery (NRDD).

This article, a result of the FDA Personalized Medicine Initiative a consortium effort across the FDA, industry and academia analyzes the key value drivers of PM development and commercialization strategies, elucidates a comprehensive approach for evaluating these strategies, and employs various modeling tools to support decision-making.

The publication of this article is timely, given the significant and increasing commitment to PM by pharmaceutical companies, highlighted by recent PM launches such as Xalkori and Zelboraf, and notable PM candidates in the pipeline including T-DM1 (Roche/Genentech, for Her2 positive breast cancer), Dabrafenib (GSK, for BRAF V600E positive melanoma), and Afatinib (BI, for EGFR mutation positive NSCLC).

Dr. Sean X. Hu, Head of Bionest USA and Managing Partner, North America, a co-lead author of the article, and the only representative from the management consulting industry serving in the FDA PM Initiative consortium, points out that the key to successful decision-making around whether and how best to incorporate biomarkers into drug development and commercialization depends on an in-depth understanding of multiple disciplines: the underlying science, clinical trial design and development paths, regulatory requirements, partnership and coordination between drug and diagnostic partners, drug and diagnostic commercialization, pricing and market access, and decision-making in the context of many uncertainties. This article identifies key value drivers and risk factors associated with PM drug and diagnostic development and commercialization. In addition, it presents a coherent approach to tie these factors together, and describes the use of financial modeling tools to arrive at such metrics as risk-adjusted net present value (NPV) and return on investment (ROI) metrics that companies often use to make key investment decisions for their products.

Dr. Federico Goodsaid, who co-authored the article while at the FDA and is currently VP of Strategic Regulatory Intelligence at Vertex Pharmaceuticals, adds: This consortium, with the participation of FDA, academic institutions and many pharmaceutical companies, was a unique opportunity to advance the field of PM by tackling one of the bottlenecks in optimizing decision-making on PM development and commercialization strategies how to quantify the commercial value of a potential biomarker-based strategy. Sean and his team made this work possible with their knowledge about PM, advanced modeling tools, and rigor in the application of decision-analysis methods to assess the value of PM strategic options and to account for their corresponding risks and uncertainties.

Bionest is attending the upcoming ASCO 2012 Annual Meeting in Chicago, June 1 5, 2012. For further discussions with Dr. Hu and his Bionest team on PM strategies, either at ASCO 2012 or another occasion, please contact Dr. Rachel Laing (rlaing@bionest.com).

Bionest is a powerhouse in PM strategy consulting, experienced in a broad spectrum of project types, from corporate level PM business models, commercialization capability building, R&D and commercialization business processes, and organizational structure, to development and commercialization strategies for individual drug assets and companion diagnostics.

In addition, Bionest has been driving thought leadership on PM, with many articles published or in development on the strategic, commercial and technical aspects of PM, including a recent article in collaboration with the Personalized Medicine Coalition (PMC) in the December 2011 supplement issue of Science which describes the approaches for optimization of PM decision-making.

For more details, please visit http://www.bionest.com, and navigate to section Strategic/Practices/Personalized Medicine Strategy.

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Bionest Publishes Landmark Article on Quantitative Financial Analysis of Personalized Medicine Strategies

Recommendation and review posted by sam

NEWARK, Calif., May 17, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM - News) today announced completion of the first planned interim safety review of the Company's Phase I/II spinal cord injury clinical trial, which indicated that the surgery, immunosuppression and the cell transplants have been well-tolerated. The trial, which is designed to evaluate the safety and preliminary efficacy of the Company's proprietary HuCNS-SC(R) cells (purified human neural stem cells), represents the first time that neural stem cells have been transplanted as a potential therapeutic agent for spinal cord injury. A summary of the data will be presented by Armin Curt, M.D., principal investigator for the clinical trial, at the Interdependence 2012 Global SCI Conference, which is being held in Vancouver, British Columbia, from May 15 to 17, 2012.

The interim data is from the first cohort of patients, all of whom suffered a complete spinal cord injury in which there is no neurological function below the level of the injury. All patients enrolled were transplanted with a dose of 20 million cells at the site of injury in the thoracic spinal cord. There were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first four months following transplantation of the cells. Changes in sensitivity to touch were observed in two of the patients. The data from multiple evaluations of the patients during this four month period have been reviewed by an independent Data Safety Monitoring Committee, which has recommended that the study advance to enrollment of patients with incomplete neurological injury. Enrollment is now underway and is open to patients in Europe, the United States and Canada with incomplete spinal cord injury. The trial, which is being conducted at Balgrist University Hospital, Zurich, Switzerland, is the only ongoing clinical trial evaluating neural stem cell transplantation in spinal cord injury.

"We are very encouraged by the interim safety outcomes for the first cohort," said Dr. Curt, who is Professor and Chairman of the Spinal Cord Injury Center at the University of Zurich, and Medical Director of the Paraplegic Center at Balgrist University Hospital. "The patients in the trial are being closely monitored and undergo frequent clinical examinations, radiological assessments by MRI and sophisticated electrophysiology testing of spinal cord function. The comprehensive battery of tests provides important safety data and is very reassuring as we progress to the next stage of the trial."

The Interdependence 2012 Global SCI Conference is intended to bring together international healthcare and research facilities to showcase their work through presentations, workshops and exhibits and to discuss how to advance research, implement new best practices and shape the next generation of spinal cord injury research. Interdependence 2012 is jointly organized by the Rick Hansen Institute, a Canadian not-for-profit organization committed to accelerating the translation of discoveries and best practices into improved treatments for people with spinal cord injuries, and the Rick Hansen Foundation.

About the Spinal Cord Injury Clinical Trial

The Phase I/II clinical trial of StemCells, Inc.'s HuCNS-SC(R) purified human adult neural stem cells is designed to assess both safety and preliminary efficacy. Twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level are planned for enrollment. The Company has dosed the first three patients all of whom have injuries classified as AIS A, in which there is no neurological function below the injury level. The second and third cohorts will be patients classified as AIS B and AIS C, those with less severe injury, in which there is some preservation of sensory or motor function. The injuries are classified according to the American Spinal Injury Association Impairment Scale (AIS). In addition to assessing safety, the trial will assess preliminary efficacy based on defined clinical endpoints, such as changes in sensation, motor and bowel/bladder function.

All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord and will be temporarily immunosuppressed. Patients will be evaluated regularly in the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any recovery of neurological function below the injury site. The Company intends to follow the effects of this therapy long-term, and a separate four-year observational study will be initiated at the conclusion of this trial.

The trial is being conducted at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation, and is open for enrollment to patients in Europe, Canada and the United States. If you believe you may qualify and are interested in participating in the study, please contact the study nurse either by phone at +41 44 386 39 01 or by email at stemcells.pz@balgrist.ch.

Additional information about the Company's spinal cord injury program can be found on the StemCells, Inc. website at http://www.stemcellsinc.com/Therapeutic-Programs/Clinical-Trials.htm and at http://www.stemcellsinc.com/Therapeutic-Programs/Spinal-Cord-Injury.htm, including video interviews with Company executives and independent collaborators.

About Balgrist University Hospital

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StemCells, Inc. Reports Positive Interim Safety Data From Spinal Cord Injury Trial

Recommendation and review posted by sam

SAN DIEGO, May 17, 2012 /PRNewswire/ -- Cardium Therapeutics (NYSE MKT: CXM) today announced that Christopher J. Reinhard, Chairman & CEO will present a corporate overview and participate in a Regenerative Medicine Panel being held at the Sachs 2012 Science, Partner, and Investment Forum: Gateway for the Global Life Sciences in West Palm Beach, FL May 21 22, 2012. The Regenerative Medicine Panel, chaired by Bernard Siegel, Executive Director, Genetics Policy Institute and Jeffrey Cohen, Director, Ladenburg Thalmann & Co, Inc., will take place on May 22 at 9:15 a.m. ET and Cardium's investor presentation is scheduled for 10:40 a.m. ET on the same day in Ballroom B.

(Logo: http://photos.prnewswire.com/prnh/20051018/CARDIUMLOGO)

An updated investor presentation is now available on Cardium's website at http://phx.corporate-ir.net/phoenix.zhtml?c=77949&p=irol-presentations.

About Cardium

Cardium is a health sciences and regenerative medicine company focused on the acquisition and strategic development of new and innovative bio-medical product opportunities and businesses with the potential to address significant unmet medical needs that have definable pathways to commercialization, partnering and other economic monetizations. Cardium's current medical opportunities portfolio, which is focused on health sciences and regenerative medicine, includes the Tissue Repair Company, Cardium Biologics, and the Company's in-house MedPodium Health Sciences healthy lifestyle product platform. The Company's lead commercial product Excellagen topical gel for wound care management recently received FDA clearance for marketing and sale in the United States. Cardium's lead clinical development product candidate Generx is a DNA-based angiogenic biologic intended for the treatment of patients with myocardial ischemia due to coronary artery disease. In addition, consistent with its capital-efficient business model, Cardium continues to actively evaluate new technologies and business opportunities. In July 2009, Cardium completed the sale of its InnerCool Therapies medical device business to Royal Philips Electronics, the first asset monetization from the Company's biomedical investment portfolio. News from Cardium is located at http://www.cardiumthx.com.

Forward-Looking Statements

Except for statements of historical fact, the matters discussed in this press release or the referenced investor presentation are forward looking and reflect numerous assumptions and involve a variety of risks and uncertainties, many of which are beyond our control and may cause actual results to differ materially from expectations. For example, there can be no assurance that results or trends observed in one clinical study or procedure will be reproduced in subsequent studies or in actual use; that new clinical studies will be successful or will lead to approvals or clearances from health regulatory authorities, or that approvals in one jurisdiction will help to support studies or approvals elsewhere; that the company can attract suitable commercialization partners for our products or that we or partners can successfully commercialize them; that our product or product candidates will not be unfavorably compared to competitive products that may be regarded as safer, more effective, easier to use or less expensive or blocked by third party proprietary rights or other means; that the products and product candidates referred to in this report or in our other reports will be successfully commercialized or will enhance our market value; that new product opportunities or commercialization efforts will be successfully established; that third parties on whom we depend will perform as anticipated; that we can raise sufficient capital from partnering, monetization or other fundraising transactions to maintain our stock exchange listing or adequately fund ongoing operations; or that we will not be adversely affected by these or other risks and uncertainties that could impact our operations, business or other matters, as described in more detail in our filings with the Securities and Exchange Commission. We undertake no obligation to release publicly the results of any revisions to these forward-looking statements to reflect events or circumstances arising after the date hereof.

Copyright 2012 Cardium Therapeutics, Inc. All rights reserved.

For Terms of Use Privacy Policy, please visit http://www.cardiumthx.com.

Cardium Therapeutics, Generx, Cardionovo, Tissue Repair, Gene Activated Matrix, GAM, Excellagen, Excellarate, Osteorate, MedPodium, Appexium, Linee, Alena, Cerex, D-Sorb, Neo-Energy, Neo-Carb Bloc, Neo-Chill, and Nutra-Apps are trademarks of Cardium Therapeutics, Inc. or Tissue Repair Company.

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Cardium to Present at Sachs Associates 2012 Science, Partner, and Investment Forum: Gateway for the Global Life Sciences

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Posted May 17, 2012

Awards to advance regenerative medicine address diverse, debilitating diseases and conditions

COLUMBIA, Md. - The Maryland Stem Cell Research Commissionhas completed its review of the 179 applications received in response to its three FY 2012 Requests for Applications (RFAs). The board of directors of the Maryland Technology Development Corporation (TEDCO) approved the Commission's recommendation to fund 40 new proposals with the Maryland Stem Cell Research Fund's (MSCRF) $12.4 million FY 2012 budget.

"We are pleased to announce our grant awards for FY 2012," said Margaret Conn Himelfarb, MPH, chair of the Commission. "These projects address a diverse array of debilitating and costly diseases and conditions, some of which are traditionally underfunded. Maryland's investment in cutting-edge stem cell research continues to advance the field and strengthens our State's national leadership position in the life sciences. We are grateful to Governor Martin O'Malley and the Maryland General Assembly for recognizing the tremendous economic and humanitarian benefits of this pioneering research."

This funding cycle, the Commission gave priority to proposals that focus on advancing regenerative medicine by selecting promising research that targets sickle cell anemia, schizophrenia, type 1 diabetes, nerve injury, Parkinson's disease, Crohn's disease, multiple sclerosis (MS), heart disease, osteoarthritis, Lou Gehrig's disease (ALS), retinal disease, and other debilitating medical conditions. Continuing the collaboration initiated last year with the California Institute of Regenerative Medicine (CIRM), the MSCRF will also support a Maryland researcher working with CIRM-funded scientists to study stem cell differentiation and bone repair.

This year's MSCRF awards include:

9 Investigator-Initiated Research Grants (RFA-MD-12-1) - providing up to $600,000 in direct costs over a maximum of three years to investigators with preliminary data to support their hypotheses.

17 Exploratory Research Grants (RFA-MD-12-2) - providing up to $200,000 in direct costs over a maximum of two years to researchers with novel approaches, mechanisms or models that may differ from current thinking in the field and/or new hypotheses that have little or no preliminary data.

14 Post-Doctoral Fellowship Research Grants (RFA-MD-12-3) - providing post-doctoral fellows up to $55,000 per year, for up to two years.

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Maryland Stem Cell Research Commission Funds 40 New Proposals in FY 2012

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

California Stem Cell, Inc. (CSC) announced today that well-known stem cell & regenerative medicine industry veteran Gregory A. Bonfiglio, J.D. has joined its Board of Directors.

Gregory Bonfiglio has over 25 years of experience working with technology companies, and was an early investor in the stem cell industry. He is Managing Partner of Proteus Venture Partners, an investment & advisory firm he founded in early 2006 to provide venture funding and strategic advisory services in the stem cell & regenerative medicine space. Mr. Bonfiglio is on the Boards of VistaGen Therapeutics and StemCyte, Inc.; he is the Chairman of the Board of the Centre for Commercialization of Regenerative Medicine (RM Translation Center in Toronto, Canada). In addition, Mr. Bonfiglio sits on the Advisory Board and Finance Committee of the International Society for Stem Cell Research (ISSCR); he is on the Commercialization Committee of the International Society for Cellular Therapy (ISCT).

Mr. Bonfiglio brings to CSC an extensive background in strategic consulting, having held partnership positions with various legal and venture firms, and having successfully led a team that took pioneering stem cell company Advanced Cell Technology public in early 2005. Were thrilled to welcome to our board someone with the breadth of industry experience that Greg has, and are very much looking forward to his participation in the continued growth of this Company, said COO Chris Airriess.

This appointment coincides with a ramp up of commercial product sales as well as advancements of CSCs active Phase II clinical trial in metastatic melanoma.

About California Stem Cell

California Stem Cell Inc. (CSC) is an Irvine, CA based company which has developed proprietary methods to generate human stem cell lines, expand them to clinically and commercially useful numbers, and differentiate them at extremely high purity using fully-defined, proprietary media and GMP processes. CSC is able to supply its human cell populations to companies and institutions worldwide for use in the development of therapies, efficacy screening or the creation of toxicity profiles for candidate drugs, and experimental research tools.

CSC is focused on the development of stem cell based therapies for spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS, or Lou Gehrigs Disease), and metastatic cancers.

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Industry Consultant Gregory Bonfiglio Joins California Stem Cell Board of Directors

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ANN ARBOR, Mich., May 17, 2012 (GLOBE NEWSWIRE) -- Aastrom Biosciences, Inc. (Nasdaq:ASTM - News), the leading developer of patient-specific expanded multicellular therapies for the treatment of severe chronic cardiovascular diseases, today announced that company president and CEO Tim Mayleben will be presenting at the World Stem Cells & Regenerative Medicine Congress at the Park Plaza hotel in London, UK. The presentation entitled "Phase 3 Development of a Cellular Therapy Product" will take place on Monday, May 21, 2012 at 4:40 pm (BST).

The Aastrom presentation will cover the benefits of a special protocol assessment and offer insights on achieving manufacturing readiness. The presentation will also address the role of clinicians and patients in the Phase 3 development process.

About Aastrom Biosciences

Aastrom Biosciences is the leader in developing patient-specific, expanded multicellular therapies for use in the treatment of patients with severe, chronic cardiovascular diseases. The company's proprietary cell-processing technology enables the manufacture of ixmyelocel-T, a patient-specific multicellular therapy expanded from a patient's own bone marrow and delivered directly to damaged tissues. Aastrom has advanced ixmyelocel-T into late-stage clinical development, including a Phase 3 clinical program to study patients with critical limb ischemia and a planned Phase 2b clinical trial in patients with ischemic dilated cardiomyopathy. For more information, please visit Aastrom's website at http://www.aastrom.com. For more information on the pivotal REVIVE Phase 3 clinical trial, please visit the trial website at http://www.revivecli.com.

The Aastrom Biosciences, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=3663

This document contains forward-looking statements, including, without limitation, statements concerning clinical trial plans and progress, objectives and expectations, clinical activity timing, intended product development, the performance and contribution of certain individuals and expected timing of collecting and analyzing treatment data, all of which involve certain risks and uncertainties. These statements are often, but are not always, made through the use of words or phrases such as "anticipates," "intends," "estimates," "plans," "expects," "we believe," "we intend," and similar words or phrases, or future or conditional verbs such as "will," "would," "should," "potential," "could," "may," or similar expressions. Actual results may differ significantly from the expectations contained in the forward-looking statements. Among the factors that may result in differences are the inherent uncertainties associated with clinical trial and product development activities, regulatory approval requirements, competitive developments, and the availability of resources and the allocation of resources among different potential uses. These and other significant factors are discussed in greater detail in Aastrom's Annual or Transition Report on Form 10-K or 10-K/T, Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. These forward-looking statements reflect management's current views and Aastrom does not undertake to update any of these forward-looking statements to reflect a change in its views or events or circumstances that occur after the date of this release except as required by law.

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Aastrom Biosciences to Present at World Stem Cells & Regenerative Medicine Congress

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

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that chairman and CEO Gary Rabin will be presenting at the World Stem Cells and Regenerative Medicine Conference, May 21-23, in London.

Mr. Rabins presentation, titled Successes and ongoing advancements of human clinical trials for the treatment of AMD & Stargardts Disease, will be given on Monday, May 21 at 5:05 p.m. BST (London time). Mr. Rabin will provide an update on ACTs three ongoing human clinical trials in the U.S. and E.U. for Dry Age-Related Macular Degeneration (Dry AMD) and Stargardts Macular Dystrophy (SMD).

ACT recently announced Data and Safety Monitoring Board (DSMB) approval to move forward with enrollment and treatment of additional patients with SMD in its U.S. SMD trial, and to treat the final two patients to round out the initial dosing arm in its European trial. All three of the companys ongoing clinical trials use human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells.

About SMD, Dry AMD and Degenerative Diseases of the Retina

Stargardts Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. SMD causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium or RPE cell layer.

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as thirty million people in the United States and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority the dry form of AMD which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

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Advanced Cell Technology to Present at World Stem Cells & Regenerative Medicine Congress in London

Recommendation and review posted by sam

Pluristem Therapeutics Ltd. (Nasdaq:PSTI; DAX: PJT: PLTR) today reported that the cardiac function in a diabetic-induced diastolic dysfunction in animals improved following PLacental eXpanded (PLX cells) administration.

The study was conducted as part of the European Commission's Seventh Framework Program (FP7) in collaboration with Prof. Doctor Carsten Tschope and his staff at the Charite Universitaetsmedizin Berlin, Berlin-Bradenburg Center for Regenerative Therapies (BCRT), Berlin, Germany.

Dr. Tschope said, "Currently, there are limited treatment options for diastolic dysfunction and even fewer options for diabetic induced diastolic dysfunction. This study holds promise that PLX cells might be able to inhibit diabetic induced diastolic dysfunction progression as well as possibly repair the existing damage, hypotheses that will be further explored in future studies."

Diabetes was induced in thirty-six mice resulting in the development of diastolic heart failure. After seven days, the animals received either PLX cells from two separate batches or placebo (12 subjects in each of the three groups). Ten mice were not treated (controls).

After three weeks, several cardiac parameters were assessed and found to be significantly improved following the treatment with PLX cells. Important measurements included the cardiac ejection fraction and the left ventricular (LV) relaxation time constant, believed to be the best index of LV diastolic function and a determination of the stiffness of the ventricle. Cardiac ejection fraction improved 19%, the left ventricular relaxation time constant fell 16% and stiffness of the ventricle fell 19%.

Administration of either batch of PLX cells also resulted in a significant anti-inflammatory effect.

Pluristem chairman and CEO Zami Alberman said, "As we demonstrated last week with the announcement that our cells successfully treated the seven year old patient suffering from aplastic bone marrow disease, our strategy is to develop a minimally invasive cell therapy solution that can be used to treat a wide range of life-threatening diseases. Our initial testing of a treatment for diastolic heart disease opens a new potential indication where our cells can be used and potentially positions Pluristem as a "first-line of defense" for diastolic dysfunction."

Pluristem's share price jumped 5.6% in pre-market trading on Nasdaq to $3.01, giving a market cap of $126.33 million. The share rose 10.6% on the TASE today to NIS 11.50.

Published by Globes [online], Israel business news - http://www.globes-online.com - on May 15, 2012

Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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Pluristem trial finds stem cells improve cardiac dysfunction

Recommendation and review posted by Bethany Smith

Cell surface protein blows potent cells cover; targeted drug works in preclinical tests

Newswise HOUSTON Breast cancer stem cells wear a cell surface protein that is part nametag and part bulls eye, identifying them as potent tumor-generating cells and flagging their vulnerability to a drug, researchers at The University of Texas MD Anderson Cancer Center report online in Journal of Clinical Investigation.

Weve discovered a single marker for breast cancer stem cells and also found that its targetable with a small molecule drug that inhibits an enzyme crucial to its synthesis, said co-senior author Michael Andreeff, M.D., Ph.D., professor in MD Andersons Departments of Leukemia and Stem Cell Transplantation and Cellular Therapy.

Andreeff and colleagues are refining the drug as a potential targeted therapy for breast cancer stem cells, which are thought to be crucial to therapy resistance, disease progression and spread to other organs.

Its been difficult to identify cancer stem cells in solid tumors, Andreeff said. And nobody has managed to target these cells very well.

The marker is the cell surface protein ganglioside GD2. The drug is triptolide, an experimental drug that Andreeff has used in preclinical leukemia research. The team found triptolide blocks expression of GD3 synthase, which is essential to GD2production.

Triptolide stymied cancer growth in cell line experiments and resulted in smaller tumors and prolonged survival in mouse experiments. Drug development for human trials probably will take several years.

Cancer stem cells are similar to normal stem cells

Research in several types of cancer has shown cancer stem cells are a small subpopulation of cancer cells that are capable of long-term self-renewal and generation of new tumors. More recent research shows they resist treatment and promote metastasis.

Cancer stem cells are similar to normal stem cells that renew specialized tissues. The breast cancer findings grew out of Andreeffs long-term research in mesenchymal stem cells, which can divide into one copy of themselves and one differentiated copy of a bone, muscle, fat or cartilage cell.

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Scientists Discover Marker to Identify, Attack Breast Cancer Stem Cells

Recommendation and review posted by Bethany Smith