LONDON (Reuters) - Taking a sample or biopsy from just one part of a tumour might not give a full picture of its genetic diversity and may explain why doctors, despite using genetically targeted drugs, are often unable to save patients whose cancer has spread, scientists said.

A study by British researchers found there are more genetic differences than similarities between biopsies taken from separate areas of the same tumour, and yet further gene differences in samples taken from secondary tumours.

That might help explain why, despite recent development of a wave of highly targeted drugs designed to tackle cancers of specific genetic types, the prognosis remains poor for many patients with so-called solid-tumour disease like breast, lung, or kidney cancer that has spread to others parts of the body.

But the researchers, whose study was partly funded by charity Cancer Research UK and published in the New England Journal of Medicine, said it also pointed to a way forward.

The team carried out the first ever genome-wide analysis of the genetic changes or faults in different regions of the same tumour.

They looked at four patients with cancer in their kidneys, taking samples from different regions of the primary tumour and also from other organs where the tumour had spread.

They found that the majority of gene faults, around two-thirds, were not the same in one sample as in another, even when the biopsies were taken from the same tumour.

Samples taken from secondary tumours - which are a result of the disease spreading to other parts of the body - had yet more different genetic faults, suggesting that basing treatment decisions on just one primary tumour sample is not sufficient.

"We've known for some time that tumours are a patchwork of faults, but this is the first time we've been able to use cutting-edge genome sequencing technology to map out the genetic landscape of a tumour in such exquisite detail," said Charles Swanton, of University College London's cancer institute, who led the study and presented its results at a briefing in London on Tuesday.

He said they had uncovered "an extraordinary amount of diversity" at a genetic level both within tumours and within a single patient, with more differences between biopsies from the same tumour than similarities.

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Cancer gene mutation more complex than previously thought -study

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Public release date: 7-Mar-2012 [ | E-mail | Share ]

Contact: Cathia Falvey cfalvey@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, Mar 07, 2012--More than 2 million teenagers suffer from depression in the U.S. Recent drug warnings and study results have led to increased controversy surrounding the treatment of adolescent depression. A state-of-the-art issue reporting on the latest research findings on antidepressant medications combined with appropriate therapeutic strategies has been published by Journal of Child and Adolescent Psychopharmacology, a peer-reviewed journal from Mary Ann Liebert, Inc. The special issue on psychopharmacology of adolescent depression is available free on the Journal of Child and Adolescent Psychopharmacology website.

"There are no radically new treatments on the horizon for the treatment of depression, and so we have to do better with the treatments we have available," says Graham J. Emslie, MD, Guest Editor of the issue and Director of Child Psychiatry at University of Texas Southwestern Medical Center, Dallas. "Few youths with depression receive adequate treatment."

The issue focuses on the controversy, the data, and the challenges and opportunities in the care of adolescents with major depressive illness. The articles cover a wide range of issues that all contribute to the goal of improving outcomes. Included in the issue, Greg Clarke, PhD et al., Kaiser Permanente Center for Health Research (Portland, OR), present an evaluation of new and refill antidepressant prescribing practices among physicians before and after warnings related to teen suicide risk were issued. Kenneth Wells, MD, MPH and colleagues from University of California, Los Angeles, and Stony Brook University (NY), explore the effectiveness of appropriate care delivered in a primary care setting. Dr. Emslie and colleagues from UT Southwestern Medical Center examine the common problem of insomnia in youths with depression and its impact on treatment.

"Depression is a major public health concern among young people, particularly teens, but many people have a hard time talking about it," says Harold S. Koplewicz, MD, Editor-in-Chief of Journal of Child and Adolescent Psychopharmacology, and President, Child Mind Institute, New York, NY. "Advancing research is one way we can work to change a culture of denial that too often stands in the way of effective and sometimes life-saving treatment."

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About the Journal

Journal of Child and Adolescent Psychopharmacology is an authoritative peer-reviewed journal published bimonthly in print and online. The journal is dedicated to child and adolescent psychiatry and behavioral pediatrics, covering clinical and biological aspects of child and adolescent psychopharmacology and developmental neurobiology. Complete tables of content and a sample issue may be viewed online at the Journal of Child and Adolescent Psychopharmacology website.

About the Company

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More effective treatments urgently needed for adolescent depression

Recommendation and review posted by Bethany Smith

Public release date: 7-Mar-2012 [ | E-mail | Share ]

Contact: Michael Bernstein m_bernstein@acs.org 202-872-6042 American Chemical Society

With the "Refrigerator Mother" notion about the cause of autism a distant and discredited memory, scientists are making remarkable progress in untangling the genetic roots of the condition, which affects millions of children and adults, according to an article in the current edition of Chemical & Engineering News. C&EN is the weekly newsmagazine of the American Chemical Society, the world's largest scientific society.

In the story, C&EN Associate Editor Lauren K. Wolf points out that most people in the 1960s believed autism resulted from a lack of maternal warmth and emotional attachment. It was a hypothesis popularized by Austrian-born American child psychologist and writer Bruno Bettelheim. Now scientists around the globe are focusing on genes that have been implicated in autism and related conditions, collectively termed "autism spectrum disorders." That research may solve mysteries about autism, which affects 1 in 110 children in the U.S. Among them: what causes autism, why does it affect more boys than girls and what can be done to prevent and treat it?

C&EN explains that scientists now have solidly implicated certain genes as being involved in autism. Most of those genes play a role in the transmission of signals through the junctions or "synapses" between nerve cells. Synapses are the territory where one nerve releases a chemical signal that hands off messages to an adjoining nerve. The human brain has an estimated 1,000 trillion synapses, and they are hot spots for miscommunications that underpin neurological disorders like autism. Scientists now are gleaning information on what those genes do, what brain circuits they affect and how the proteins they produce function. In doing so, they are paving the way for future medications for autism spectrum disorders.

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The American Chemical Society is a nonprofit organization chartered by the U.S. Congress. With more than 164,000 members, ACS is the world's largest scientific society and a global leader in providing access to chemistry-related research through its multiple databases, peer-reviewed journals and scientific conferences. Its main offices are in Washington, D.C., and Columbus, Ohio.

To automatically receive news releases from the American Chemical Society contact newsroom@acs.org.

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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From 'Refrigerator Mothers' to untangling the genetic roots of autism

Recommendation and review posted by Bethany Smith

CHESHIRE, Conn.--(BUSINESS WIRE)--

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN - News) today announced that asfotase alfa (formerly known as ENB-0040), a highly innovative investigational targeted enzyme replacement therapy, was shown to improve skeletal abnormalities, pulmonary and physical function, and cognitive development in a Phase 2 study of infants and young children with life-threatening hypophosphatasia (HPP). Alexion is developing asfotase alfa as a potential treatment for patients with HPP, an ultra-rare, genetic, life-threatening metabolic disease for which there are currently no approved or effective treatment options. Findings from the study are published in the March 8th issue of the New England Journal of Medicine.

Due to a genetic enzyme deficiency, symptomatic patients with HPP face progressive damage to multiple vital organs including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure.1,2,3,4 About half of newborns with HPP do not survive past one year of age.1

This inborn error of metabolism can cause progressive skeletal deterioration and muscle weakness in severely affected infants and very young children with HPP, leading to respiratory insufficiency and significant mortality, said lead study author Michael P. Whyte, M.D., Medical-Scientific Director, Center for Metabolic Bone Disease and Molecular Research, Shriners Hospitals for Children, St. Louis. In this study of patients with severe perinatal and infantile forms of HPP, we saw in nearly all patients striking skeletal healing that included improved bone formation and reduced deformity, as well as improved pulmonary function and motor development. These findings are remarkable given the historically grim outlook for patients with life-threatening HPP.

About the Study

The multinational, open-label Phase 2 study of asfotase alfa enrolled 11 patients with HPP ages 3 years or younger whose symptoms began before the age of 6 months. Patients in the study received asfotase alfa for six months and then had the opportunity to enroll in an open-label extension study.

The primary efficacy endpoint was change in the skeletal manifestations of HPP, as assessed by radiography. Response to treatment was defined as a mean improvement of two or more points, as rated by a panel of three independent radiologists, on a seven-point scale known as the radiographic global impression of change (RGI-C). Skeletal changes were also assessed using a 10-point scale that measured skeletal abnormalities at the wrists and knees. Additional efficacy assessments included evaluations of respiratory status, gross motor function, and cognitive development (Bayley-III scale).

Ten patients completed the six-month study and nine patients are currently participating in the extension study. All patients treated with asfotase alfa demonstrated an improvement in two key biochemical indicators of HPP: blood levels of PPi (inorganic pyrophosphate) and PLP (pyridoxal 5 phosphate). For the primary efficacy endpoint, nine of 10 patients (90%) met the criterion for treatment response by week 24, and eight of nine (89%) achieved treatment response by week 48. Skeletal healing became apparent as early as week 3.

Respiratory function improved in all patients. These improvements were evident as early as week 12. Compared to the 10 of 11 patients who required respiratory support at baseline, at week 48 only three of nine patients required any respiratory support and only one patient remained on full mechanical ventilation. In addition to the improvements in bone mineralization and respiratory function, there were improvements in fine motor, gross motor and cognitive development, as assessed by the Bayley-III instrument, for seven of the eight patients who were evaluated. Whereas at baseline, no patients were able to bear weight through their legs owing to skeletal abnormalities and muscle weakness, at 48 weeks of treatment, seven of nine patients were able to bear weight through their legs.

The most common treatment-related adverse event observed in the study was mild injection-site reaction. Severe adverse events observed in the study were generally consistent with the symptoms expected of patients with severe HPP, including infection, respiratory disorders, and nervous-system complications. One patient died, and this was determined to not be related to study drug.

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New England Journal of Medicine Publishes Data from Phase 2 Study of Asfotase Alfa in Life-Threatening Hypophosphatasia

Recommendation and review posted by Bethany Smith

NEW YORK (Reuters) - An experimental therapy for a rare, often fatal genetic disorder appears to offer hope for infants and very young children with the condition, according to data from a small clinical trial reported in the New England Journal of Medicine on Wednesday.

The enzyme-replacement drug, asfotase alfa, acquired by Alexion Pharmaceuticals Inc with its $610 million purchase of Canada-based Enobia Pharma, could become the first approved treatment for the metabolic disease hypophosphatasia, or HPP.

The condition is a genetic enzyme deficiency that causes bone softening and muscle weakness and can lead to severe lung problems and damage to other vital organs. It affects about 1 in 100,000 newborns worldwide, according to the National Institutes of Health. About half of infants with a severe form of the disease do not survive beyond one year.

In the study of 11 babies and toddlers under three with life-threatening HPP, treatment with asfotase alfa resulted in "striking" improvements in skeletal problems and dramatic improvements in lung function and mobility, researchers reported.

"I'm thrilled to see babies who were really doomed responding to the treatment," Dr Michael Whyte, the study's lead investigator, said in a telephone interview.

"The therapy is proving not only life-saving but also health-restoring," added Whyte, of Shriners Hospitals for Children in St. Louis, who has been working on this ultra-rare disease for more than 30 years.

Videos accompanying the online version of the study in the New England Journal of Medicine show dramatic motor skill improvements of two of the trial subjects. In one, a three-year-old who was unable to stand prior to therapy is shown climbing the steps of a small plastic slide after 24 weeks of treatment.

Respiratory function improved in all patients, researchers said. Ten of the 11 needed breathing support before treatment. After 48 weeks of treatment, only three of the nine children who remained on therapy needed help breathing.

Breathing problems arise as the shape of the chest becomes deformed due to soft bones in the thorax, which compromises lung function, Whyte explained.

'SHE CAN STAND ON HER OWN'

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Alexion drug offers hope for rare, deadly disorder

Recommendation and review posted by Bethany Smith

SAN DIEGO, March 7, 2012 /PRNewswire/ -- Sequenom, Inc. (NASDAQ: SQNM - News), a life sciences company providing innovative genetic analysis solutions, today reported total revenues of $15.5 million and $55.9 million for the fourth quarter and full-year of 2011, respectively. Net loss was $22.2 million, or $0.22 per share, and $74.2 million, or $0.75 per share, for the fourth quarter and full-year, respectively.

"2011 was a pivotal year for Sequenom as the Sequenom Center for Molecular Medicine launched its cornerstone MaterniT21(TM) prenatal laboratory-developed test and advanced a number of other important programs," said Harry F. Hixson, Chairman and CEO of Sequenom, Inc. "The positive uptake from the launch of the MaterniT21 LDT and increasing early volumes for testing services since has set the tone for our expected growth and expansion during 2012."

Fourth Quarter 2011 Performance

Fourth quarter revenues of $15.5 million in 2011 increased 13% over revenues of $13.8 million for the comparable period in 2010. Fourth quarter 2011 revenues from the genetic analysis operating segment were essentially flat year-over-year, while revenues from the Sequenom Center for Molecular Medicine (Sequenom CMM) diagnostics services operating segment grew more than 130% in the fourth quarter of 2011 from the prior year period.

Gross margin for the fourth quarter of 2011 was 46% as compared to gross margin of 62% for the fourth quarter of 2010. This difference reflects the increased costs associated with the nationwide launch of the MaterniT21(TM) LDT during the fourth quarter, in October of 2011.

Total operating expenses for the fourth quarter of 2011 were $29.0 million, as compared to total operating expenses of $30.7 million for the fourth quarter of 2010. Research and development expenses increased $2.6 million to $13.1 million for the fourth quarter of 2011, a change associated primarily with higher labor costs and an increase in consumables associated with additional T21 clinical studies. Selling and marketing expenses increased by $1.1 million to $9.1 million for the fourth quarter of 2011, resulting primarily from higher labor costs associated with the expansion of the Sequenom CMM sales force and the CLIA laboratory. Total stock-based compensation expense was $2.8 million for the fourth quarter of 2011, consistent with the $2.8 million in stock-based compensation recorded for the fourth quarter of 2010.

"Our 2011 year end results are demonstrative of our commitment to delivering on the priorities set early in the year. We met the major goals of our strategic plan, managing costs while establishing the groundwork for continued commercial expansion," said Paul V. Maier, Sequenom's CFO. "We have set new goals and are focusing on accelerating our growth and expansion in a number of important areas to maintain this positive trajectory in 2012. As a result of our recent financing, we are well capitalized to implement our expansion plans."

Full-Year 2011 Performance

The Company reported revenues of $55.9 million for the full-year 2011, an increase of 18% compared to revenues of $47.5 million for the full-year 2010. The Company will continue to account for product revenue from our diagnostic testing services on a cash basis until further experience is gained and additional internal and third party controls are established that will allow a reasonable estimate of collectable amounts to be made before moving to the accrual method of accounting.

Net loss for the full-year 2011 was $74.2 million or $0.75 per share, as compared to net loss of $120.8 million, or $1.69 per share for 2010.

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Sequenom, Inc. Reports Financial Results for the Fourth Quarter and Full-Year of 2011

Recommendation and review posted by Bethany Smith

WEDNESDAY, March 7 (HealthDay News) -- The genetic makeup of cancer cells differs significantly from region to region within a single tumor, according to new research that raises questions about the true potential of personalized cancer medicine.

With this treatment approach, doctors study a tumor's genetic makeup to determine which drugs would work best in a particular patient. But if the genetic mutations driving the cancer cells vary widely, a single tissue sample won't necessarily give the full picture.

This "targeted therapy" involves "sticking a needle into the primary tumor site and taking a small sliver of a tumor, doing a gene analysis, and creating a genetic profile of the tumor to predict how the tumor will behave," explained Dr. Dan Longo, an oncologist and deputy editor at the New England Journal of Medicine.

"What this paper tells us is that is an oversimplification of the complexity of tumors and their heterogeneity," he said. "If you look at different sites of the very same tumor and the very same person, one site might tell you a gene profile associated with a good prognosis and the other site will tell you a gene profile associated with a bad prognosis."

Longo wrote an editorial accompanying the new study, published in the March 8 issue of the New England Journal of Medicine.

In the study, scientists from Cancer Research UK London Research Institute took 13 biopsies, or tissue samples, from a patient whose kidney cancer had spread. The biopsies were from eight regions of the kidney tumor and four tumors in the chest and lungs.

Researchers also took normal tissue, sequenced the patient's genome and compared that to what they found in the biopsies.

Genetic analysis turned up 128 mutations in the tumors. But only about one-third, or about 40 of those mutations, were present in all of the biopsies.

"The majority of mutations are not shared in every biopsy," said senior study author Charles Swanton, a professor of cancer medicine at the research institute.

Swanton and his colleagues also analyzed tumor tissue samples from another three patients with kidney cancer. From a total of 30 biopsies from all four patients, 26 tissue samples had mutations that were highly heterogenous, or varied, from one another.

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Is Cancer Outwitting 'Personalized Medicine'?

Recommendation and review posted by Bethany Smith

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

Interleukin Genetics, Inc. (OTCQB: ILIU.PK - News) announced today the publication of a peer-reviewed study which found that Interleukin-1 (IL-1) gene variations are associated with increased risk of periodontal disease. The study, which appears on the Journal of Periodontologys website, in advance of appearing in the print edition, was led by Nadeem Y. Karimbux, D.M.D., Associate Professor of Oral Medicine, Infection and Immunity, Harvard School of Dental Medicine.

The study assessed the potential value of IL-1 genetic variations in the risk for developing severe periodontal disease. The IL-1 genetic variations in the published study are included in Interleukin Genetics PST Genetic Test, the first genetic test to analyze genes for variations that identify an individuals predisposition for over-expression of inflammation and risk for periodontal disease. Researchers reviewed 27 published studies on IL-1 genetics and periodontal disease from 1997 through June 2011 which examined Caucasian adults, 35 years or older with adult periodontal disease, to determine whether there was a significant association between the presence of the IL-1 gene variations and the severity and progression of periodontal disease. Thirteen studies qualified for the quantitative meta-analysis, which found significant effects for the two individual gene variations (IL1A OR=1.48; and IL1B OR= 1.54) and for a composite genotype that combines minor alleles at each locus (OR= 1.51). Some heterogeneity was evident, but there was no indication of publication bias.

This review and meta-analysis show that IL1A and IL1B genetic variations are significant contributors to chronic periodontitis in Caucasians, said Dr. Karimbux. Having this actionable information can assist dentists in establishing more aggressive treatment protocols for patients at increased risk.

Periodontal disease is caused by a chronic bacterial infection that activates inflammation which destroys the gums and bone supporting the teeth. If left untreated, periodontitis leads to tooth loss. Studies have shown that people with chronic and prolonged inflammatory periodontal disease are at an increased risk of several systemic conditions, such as heart disease, strokes, rheumatoid arthritis and certain chronic pulmonary diseases.

Periodontal disease is one of the most common chronic diseases worldwide, but fortunately most individuals develop only a mild form of periodontitis that when caught early, can be easily treated. We now know that approximately 8 to 13 percent of the adult population will develop more destructive forms of periodontitis, and most of those at risk can be identified early based on smoking, IL-1 genetics and diabetes, said Kenneth Kornman D.D.S., PhD., study author and Chief Scientific Officer of Interleukin Genetics. While additional studies should be undertaken to look at specific periodontal conditions and additional ethnicities, this study reaffirms the role genetics plays in adult oral and overall health.

About Interleukin Genetics, Inc. Interleukin Genetics, Inc. (OTCQB: ILIU.PK - News) develops and markets a line of genetic tests under the Inherent Health and PST brands.The products empower individuals to prevent certain chronic conditions and manage their existing health and wellness through genetic-based insights with actionable guidance. Interleukin Genetics leverages its research, intellectual property and genetic panel development expertise in metabolism and inflammation to facilitate the emerging personalized healthcare market. The Company markets its tests through partnerships with health and wellness companies, healthcare professionals and other distribution channels. Interleukin Genetics flagship products include its proprietary PST genetic risk panel for periodontal disease and tooth loss susceptibility sold through dentists and the Inherent Health Weight Management Genetic Test that identifies the most effective diet and exercise program for an individual based on genetics. Interleukin Genetics is headquartered in Waltham, Mass. and operates an on-site, state-of-the-art DNA testing laboratory certified under the Clinical Laboratory Improvement Amendments (CLIA). For more information, please visit http://www.ilgenetics.com.

About PST The PST Genetic Test identifies individuals with increased risk for severe and progressive periodontal disease and significant tooth loss based on a proprietary panel of genetic variations that predispose an individual to over-express inflammation. In August 2010, Interleukin Genetics announced the initiation of a landmark clinical study on risk factors predictive of periodontal disease progression to tooth loss using a new version of the PST Genetic Test. This clinical studybeing conducted at the University of Michigan School of Dentistry and led by Dr. William Giannobile, Director of the Michigan Center for Oral Health Researchis designed to test whether risk factors, including genetic information, can guide more successful intervention and thus reduce the adverse outcomes of periodontal disease, such as tooth loss.

Certain statements contained herein are forward-looking statements, including statements that the clinical studies have the potential to expand the use of the PST Genetic Test. Because such statements include risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that could cause actual results to differ materially from those expressed or implied by such forward-looking statements include, but are not limited to, those risks and uncertainties described in the Interleukin Genetics annual report on Form 10-K for the year ended December 31, 2010 and other filings with the Securities and Exchange Commission. Interleukin Genetics disclaims any obligation or intention to update these forward-looking statements.

Link:
Newly Published Meta-Analysis Study Finds that IL-1 Gene Variations Contained in Interleukin Genetics’ PST Test are ...

Recommendation and review posted by Bethany Smith

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

Viral Genetics (Pinksheets: VRAL.PK - News) announced today that it has submitted a pre-IND briefing document to the US Food and Drug Administration (FDA) for its Lyme Disease drug candidate, VGV-L, marking important milestones for both the Company and its supporters in the Lyme community.

To our knowledge, this is the first novel drug candidate that has been proposed for study in the treatment of chronic Lyme Disease post-infection in quite some time. We are equally pleased that it represents the second drug candidate we have developed from our licensed Targeted Peptides platform, said Haig Keledjian, President of Viral Genetics. Our shareholders should be proud that our team managed to bring a drug candidate to this step of preliminary FDA review within about 30 months. Within the single Targeted Peptides platform, we are also developing candidates for treatment of sepsis, staphylococcus and streptococcus infection, multiple sclerosis and other conditions, while we continue to complete IND-enabling preclinical testing for our HIV/AIDS candidate.

The pre-IND submission provides extensive research information gathered by Viral Genetics researchers over a 2 year period of rigorous and detailed testing which resulted in positive results, to the FDA, along with a protocol for a proposed US human clinical trial designed under the guidance of a leading Lyme clinician at one of the nations top medical centers. Testing to date was conducted at the University of Colorado, Texas A&M University, Scott & White Hospital, and has been led by Viral Genetics Chief Scientist, Dr. M. Karen Newell-Rogers, with significant contributions from several clinicians.

The Company anticipates that the response to the pre-IND submission will be received in March-April 2012. While the FDAs written responses to pre-IND submissions are typically comprehensive, in some cases the need for additional clarification or discussion necessitates a meeting in person or by teleconference.

The written pre-IND response typically provides detailed insight into the FDA's concerns about available information on a particular drug being proposed for human testing in a particular patient population, and helps preempt any potential deficiencies that the FDA may find upon submission of the full IND application. This feedback acts as a kind of blueprint that guides the sponsors completion of the full IND towards attainment of FDA clearance to proceed with the proposed clinical trial. Post-submission of an IND, FDA reviewers may need clarifications or additional information before making a decision. FDA requirements for an IND include detailed information on all aspects of the proposed product such as manufacturing, preclinical and clinical testing, scientific background, proposed clinical development plan, clinical protocol, etc. This information needs to be presented in a format aimed towards clarifying the rationale of the proposed clinical trial, and for ease of review by the FDA reviewers.

Funding for some of the pre-clinical trial studies leading to the filing was initiated by Viral Genetics advisor, Richard Gerstner, former head of IBMs Asia operations and later the companys personal computer division. In April of 2011, Mr. Gerstner, who faced a long battle with Lyme during his tenure at IBM and his subsequent career in the venture capital arena, was a recipient, along with Dr. M. Karen Newell Rogers, of the Lauren F. Brooks Hope Award, given by the Time for Lyme Foundation. Since the commencement of Time for Lymes fundraising efforts in 2001, it has raised nearly $5 million and partnered with Columbia University Medical Center to create its Lyme and Tick-Borne Disease Research Center. Past recipients of the award include Nobel Laureate Dr. Luc Montagnier, also an advisory board member of Viral Genetics, and world-renowned pediatrician Dr. Charles Ray Jones.

This research was further supported by grants from the Time for Lyme and the Turn the Corner Foundation who both saw the promise in Dr. Newell-Rogers approach, helping it to reach this phase of development and the pre-IND filing. The proposed therapy, like several others in Viral Genetics R&D pipeline, is based on Targeted Peptide technology (TPT) and uses synthetic peptides to "trick" cells that may be responsible for harmful symptoms, making them vulnerable to the body's natural immune response mechanism.

Emphasizing the platform nature of the TPT approach, Dr. Newell-Rogers expanded on how TPT potentially may be targeted to a number of potential diseases or indications that have proved stubbornly resistant to more traditional approaches such as, in this case, chronic Lyme Disease. The idea behind our research is that those with a genetic blueprint that does not allow certain self-peptides to be processed or removed tend to mount a chronic inflammatory immune response that is not properly controlled. In terms of drug development, we believe that many diseases and chronic illnesses may be dependent in important ways on this harmful type of inflammation, Dr. Newell stated. Her theory proposes a targeted peptide to replace or remove certain self-peptides and hopefully restore a less harmful and more specific immune response in patients. The studies conducted by Viral Genetics aim to shed light on this chronic inflammatory response and symptoms shared by a significant subset of Lyme disease patients.

Currently there is no treatment for Lyme Disease once it has developed into its chronic, long-term state, other than antibiotics regimens which, while managing the disease for some of those infected, leaves untouched some of the symptoms for a significant portion of those suffering from this debilitating condition.

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Viral Genetics Submits Pre-IND Document for Lyme Disease Drug Candidate to FDA

Recommendation and review posted by Bethany Smith

Yesterday, the Mesothelioma Applied Research Foundation hosted a teleconference featuring Dr. Dan Sterman of the University of Pennsylvania Medical Center, who discussed the recent advances in mesothelioma gene therapy.

Washington, DC (PRWEB) March 07, 2012

The call was attended by nearly 100 people interested in learning more about progress recently made in mesothelioma treatment. The recording of the call is available on the Meso Foundations website, at http://www.curemeso.org/calls.

This call is part of Meso Foundations Meet the Experts series, which was created to provide mesothelioma patients, their families, and others interested with the most up-to-date mesothelioma information. Previous calls have included an interview with Dr. Lee Krug of Memorial Sloan-Kettering about using vaccines and manipulations of the immune system to prevent a mesothelioma recurrence.

Mesothelioma is a malignant tumor of the lining of the lung, abdomen, or heart known to be caused by exposure to asbestos. Medical experts consider it one of the most aggressive and deadly of all cancers. Approximately 3,000 Americans are diagnosed with mesothelioma every year, but accurate treatment information and help are very difficult to find.

The Meso Foundation is the leading organization dedicated to eradicating mesothelioma as a life-ending cancer by funding peer-reviewed mesothelioma research, providing patient support services and education, and advocating Congress for increased federal funding for research. Mesothelioma funding, per death, has historically been extremely low, and even as recently as 2010, the NCI reported that mesothelioma receives as little as 7 times less funding than other cancers. The Meso Foundation was founded in 2000 to address this imbalance and since then has independently funded over $7.6 million in peer-reviewed mesothelioma research. More information is available at http://www.curemeso.org.

For more information about the topics discussed in the Meet the Experts series or for information about mesothelioma treatment, patients and their loved ones are encouraged to contact Mary Hesdorffer, NP at mhesdorffer(at)curemeso(dot)org or by phone at (703) 879-3820.

Maja Belamaric Mesothelioma Applied Research Foundation (703) 879-3822 Email Information

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Meso Foundation Covers Recent Advances in Mesothelioma Gene Therapy in Teleconference

Recommendation and review posted by Bethany Smith

BOSTON - Scientists are reporting what could be very bad news for efforts to customize cancer treatment based on each person's genes.

They have discovered big differences from place to place in the same tumour as to which genes are active or mutated. They also found differences in the genetics of the main tumour and places where the cancer has spread.

This means that the single biopsies that doctors rely on to choose drugs are probably not giving a true view of the cancer's biology. It also means that treating cancer won't be as simple as many had hoped.

By analyzing tumours in unprecedented detail, "we're finding that the deeper you go, the more you find," said one study leader, Dr. Charles Swanton of the Cancer Research UK London Research Institute in England. "It's like going from a black-and-white television with four pixels to a colour television with thousands of pixels."

Yet the result is a fuzzier picture of how to treat the disease.

The study is reported in Thursday's New England Journal of Medicine.

It is a reality check for "overoptimism" in the field devoted to conquering cancer with new gene-targeting drugs, Dr. Dan Longo, a deputy editor at the journal, wrote in an editorial.

About 15 of these medicines are on the market now and hundreds more are in testing, but they have had only limited success. And the new study may help explain why.

The scientists used gene sequencing to a degree that has not been done before to study primary tumours and places where they spread in four patients with advanced kidney cancer. They found that two-thirds of gene mutations they detected were not present in all areas of the same tumour. They also were stunned to see different mutations in the same gene from one part of a tumour to another.

That means a single biopsy would reveal only a minority of mutations. Still, it's not clear whether doing more biopsies would improve accuracy, or how many or how often they should be done.

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Gene study suggests treating cancer is more complex than many had hoped

Recommendation and review posted by Bethany Smith

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

Contact: Megan Fellman fellman@northwestern.edu 847-491-3115 Northwestern University

Northwestern University scientists have developed a powerful analytical method that they have used to direct stem cell differentiation. Out of millions of possibilities, they rapidly identified the chemical and physical structures that can cue stem cells to become osteocytes, cells found in mature bone.

Researchers can use the method, called nanocombinatorics, to build enormous libraries of physical structures varying in size from a few nanometers to many micrometers for addressing problems within and outside biology.

Those in the fields of chemistry, materials engineering and nanotechnology could use this invaluable tool to assess which chemical and physical structures -- including size, shape and composition -- work best for a desired process or function.

Nanocombinatorics holds promise for screening catalysts for energy conversion, understanding properties conferred by nanostructures, identifying active molecules for drug discovery or even optimizing materials for tissue regeneration, among other applications.

Details of the method and proof of concept is published in the Proceedings of the National Academy of Sciences.

"With further development, researchers might be able to use this approach to prepare cells of any lineage on command," said Chad A. Mirkin, who led the work. "Insight into such a process is important for understanding cancer development and for developing novel cancer treatment methodologies."

Mirkin is the George B. Rathmann Professor of Chemistry in the Weinberg College of Arts and Sciences and professor of medicine, chemical and biological engineering, biomedical engineering and materials science and engineering. He also is the director of Northwestern's International Institute for Nanotechnology (IIN).

The new analytical method utilizes a technique invented at Northwestern called polymer pen lithography, where basically a rubber stamp having as many as 11 million sharp pyramids is mounted on a transparent glass backing and precisely controlled by an atomic force microscope to generate desired patterns on a surface. Each pyramid -- a polymeric pen -- is coated with molecules for a particular purpose.

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Influencing stem cell fate

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ScienceDaily (Mar. 6, 2012) Northwestern University scientists have developed a powerful analytical method that they have used to direct stem cell differentiation. Out of millions of possibilities, they rapidly identified the chemical and physical structures that can cue stem cells to become osteocytes, cells found in mature bone.

Researchers can use the method, called nanocombinatorics, to build enormous libraries of physical structures varying in size from a few nanometers to many micrometers for addressing problems within and outside biology.

Those in the fields of chemistry, materials engineering and nanotechnology could use this invaluable tool to assess which chemical and physical structures -- including size, shape and composition -- work best for a desired process or function.

Nanocombinatorics holds promise for screening catalysts for energy conversion, understanding properties conferred by nanostructures, identifying active molecules for drug discovery or even optimizing materials for tissue regeneration, among other applications.

Details of the method and proof of concept is published in the Proceedings of the National Academy of Sciences.

"With further development, researchers might be able to use this approach to prepare cells of any lineage on command," said Chad A. Mirkin, who led the work. "Insight into such a process is important for understanding cancer development and for developing novel cancer treatment methodologies."

Mirkin is the George B. Rathmann Professor of Chemistry in the Weinberg College of Arts and Sciences and professor of medicine, chemical and biological engineering, biomedical engineering and materials science and engineering. He also is the director of Northwestern's International Institute for Nanotechnology (IIN).

The new analytical method utilizes a technique invented at Northwestern called polymer pen lithography, where basically a rubber stamp having as many as 11 million sharp pyramids is mounted on a transparent glass backing and precisely controlled by an atomic force microscope to generate desired patterns on a surface. Each pyramid -- a polymeric pen -- is coated with molecules for a particular purpose.

In this work, the researchers used molecules that bind proteins found in the natural cell environment, such as fibronectin, which could then be attached onto a substrate in various patterns. (Fibronectin is a protein that mediates cell adhesion.) The team rapidly prepared millions of textured features over a large area, which they call a library. The library consisted of approximately 10,000 fibronectin patterns having as many as 25 million features ranging in size from a couple hundred nanometers to several micrometers.

To make these surfaces, they intentionally tilt the stamp and its array of pens as the stamp is brought down onto the substrate, each pen delivering a spot of molecules that could then bind fibronectin. The tilt results in different amounts of pressure on the polymeric pens, which dictates the feature size of each spot. Because the pressure varies across a broad range, so does the feature size.

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GAYVILLE When it came to helping his brother, Jason Selchert was willing to do a lot more than give the shirt off his back. In January, he became a bone marrow donor for his sibling, Jeff Selchert.

Jason, 39, superintendent of the Gayville-Volin School, said he had plenty of time to prepare for the procedure.

A little over two years ago, my brother, who is four years older than I am, was diagnosed with leukemia, he stated. When he was diagnosed, they determined that probably at some point in his illness, he was going to have to undergo a bone marrow transplant. Luckily enough, he has five siblings, and we were all tested. Two of the five were a match. I was one of the two.

Since the chance that a sibling will be a match is about one in four, the fact that two were a match was fortuitous, Jason said.

Jason and his oldest brother were both perfect matches, according to the blood and DNA testing.

However, Jason was selected as the best candidate.

After undergoing treatment following the initial diagnosis, Jeffs leukemia went into remission. But a few days prior to last Thanksgiving, the cancer had returned.

It was pretty well advanced, and they determined that the only way to treat it was going to be a transplant, Jason said. I had known for more than two years that it was maybe going to be an option. Its a pretty lengthy psychological process you go through to make sure that you are mentally stable enough to go through it, and understand what is going to happen and what could be the end result for me and my brother.

Jason donated his bone marrow stem cells Jan. 30. A successful transplant to his brother occurred the next day.

The worst part of the process was the time leading up to the donation, according to Jason. During the four previous days, he was given medication that caused his body to overproduce bone marrow stem cells. The process made Jason ache and feel nauseated, similar to the flu.

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alan bernstein From Wednesday's Globe and Mail Published Wednesday, Mar. 07, 2012 2:00AM EST

Most Canadians are unaware that two of their own a lanky physics whiz from Alberta and a rumpled Shakespeare-quoting MD from Toronto made a discovery 50 years ago that transformed the understanding of human biology and opened new doors to the treatment of cancer and other diseases.

Toiling away in labs atop Torontos old Princess Margaret Hospital, James Edgar Till and Ernest Armstrong (Bun) McCulloch proved that a single rare cell could produce the red blood cells, white blood cells and platelets needed to make blood, while simultaneously reproducing itself. Dr. Till and Dr. McCulloch originally called the cell a colony-forming unit. Today, its better known as a stem cell.

A great new book, Dreams and Due Diligence, by Joe Sornberger, tells the story. Still, that so few of us know let alone celebrate the fact that the stem cell is a Canadian discovery is baffling. Canada founded the entire field of stem-cell science. We have done much of the heavy lifting for decades: discovering neural stem cells, skin stem cells and cancer stem cells. If hockey is Canadas game, stem-cell science is Canadas science. Not knowing about Dr. Till and Dr. McCulloch is not knowing about Maurice Richard and Wayne Gretzky.

The way it happened didnt help. Their original paper was published in an obscure journal, Radiation Research, in 1961. Public interest went viral only after American James Thomson isolated human embryonic stem cells in 1998, which simultaneously raised hopes that stem cells could be used to repair any damaged cell in the body and ethical concerns that doing so would encourage the destruction of human embryos.

In 2002, the Canadian Institutes of Health Research developed guidelines for all stem-cell research carried out in Canada with its funds. These guidelines have become the gold standard for other countries, including the United States.

Whats even more remarkable is that Canada does such groundbreaking research on a dime. The all in investment in stem-cell research in Canada public, private and charitable funding is about $75-million. This support is provided by Canadians through taxes, donations to health charities and the generosity of community leaders individuals such as Robert and Cheryl McEwen of Toronto and the late Harley Hotchkiss of Calgary. But we still seriously lag behind California, which, with roughly the same population as Canada, has committed $3-billion over 10 years for stem-cell research.

How much further Canadas star scientists can go, however, is in doubt. According to the Stem Cell Network, there are 40 to 50 early-phase clinical trials using transplanted cells ready to roll out over the next four years. All are currently unfunded.

Prime Minister Stephen Harper has said his government will continue to make the key investments in science and technology but bemoaned Canadas less-than-optimal results for those investments. Stem-cell research has already proved itself a sound investment: Dr. Till and Dr. McCullochs work formed the basis of the bone marrow transplantation program at Princess Margaret Hospital that alone has saved thousands of lives. But it will take more than government funding: Private industry and private citizens also need to support life-saving research.

Canadians have good reason to be proud of our countrys contributions to health research and medicine. Two stand out as landmarks: the discovery of insulin in the 1920s and the discovery of stem cells in the 1960s. On Wednesday, at a dinner that brings together many of the countrys leading figures in business, the arts, entertainment, sports and science, the Canadian Stem Cell Foundation will be launched. The event will look back at that great discovery 50 years ago and look forward to ensure that Canadians continue to contribute to stem-cell research and its application to human disease.

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If Canada's game is hockey, its science is stem cells

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Washington, Mar 7 (ANI): Researchers have offered a potential insight into a known anti-cancer gene, which may also open the door to new therapeutic options that may aid weight loss and longevity in humans.

Mice with an extra dose of a known anti-cancer gene lose weight even as their appetites grow. Not only that, but according to the report, the animals also live longer, and that isn't just because they aren't getting cancer, either.

One of the animals' youthful secrets is hyperactive brown fat, which burns energy instead of storing it.

The findings add to evidence that tumour suppressors aren't designed only to protect us against cancer, the researchers say. They also point to new treatment strategies aimed to boost brown fat and fight aging.

"Tumor suppressors are actually genes that have been used by evolution to protect us from all kinds of abnormalities," said Manuel Serrano of the Spanish National Cancer Research Center.

In this case, the researchers studied a tumour suppressor commonly lost in human cancers. Mice with an extra copy of the gene known as Pten didn't get cancer, but that's not the half of it.

Those mice were also leaner, even as they ate more than controls, Serrano said. That suggested that the animals were experiencing some sort of metabolic imbalance-and a beneficial one at that.

Cancer protection aside, the animals lived longer than usual. They were also less prone to insulin resistance and had less fat in their livers.

Those benefits seem to trace back to the fact that those Pten mice were burning more calories thanks to overactive brown fat.

Studies in isolated brown fat cells confirmed that a boost in Pten increases the activity of those cells. Pten also made it easier for brown fat to form.

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One of the world's leading researchers into spinal cord injuries says China could hold the key to a cure that he has been searching for since he met late actor Christopher Reeve in the 1990s.

US-based Doctor Wise Young first used the word "cure" in relation to his work after a conversation with Reeve, the Superman hero who became a quadriplegic in an equestrian accident in 1995.

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Reeve contacted him looking for help and the two became close friends. The actor died of heart failure in 2004 at the age of 52, having devoted his life to raising awareness about spinal cord injuries and stem-cell research.

But it was a star of a different sort, Chinese gymnast Sang Lan, who set Young on the path he believes has brought a cure closer than ever, thanks to ground-breaking clinical trials of stem-cell therapy he is conducting in China.

"Everybody assumed that I'm doing this in China because I wanted to escape George W. Bush, but that's not the case at all," Young told AFP, recalling the former US president's 2001 decision to effectively stop federal funding of embryonic stem cell research.

"I started the clinical trials in 2005 here in Hong Kong . . . mainly because of a promise that I made to a young woman. Her name is Sang Lan."

Sang crushed her spine during a routine warm-up exercise at the Goodwill Games in New York in 1998. She met Young as she underwent treatment and rehabilitation in the US over the next 12 months.

"Her parents came to me and asked whether or not there would ever be a cure for her, and I said we're working very hard on it," said Young, who was by then one of the leading US experts on spinal cord injuries.

"When she went back to China after doing her rehabilitation in New York she cried and asked how would therapies go from the United States to China.

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by Stephen Coates

HONG KONG, March 7, 2012 (AFP) - One of the world's leading researchers into spinal cord injuries says China could hold the key to a cure that he has been searching for since he met late actor Christopher Reeve in the 1990s.

US-based Doctor Wise Young first used the word "cure" in relation to his work after a conversation with Reeve, the "Superman" hero who became quadriplegic in an equestrian accident in 1995.

Reeve contacted him looking for help and the two became close friends. The actor died of heart failure in 2004 at the age of 52, having devoted his life to raising awareness about spinal cord injuries and stem-cell research.

But it was a star of a different sort, Chinese gymnast Sang Lan, who set Young on the path he believes has brought a cure closer than ever, thanks to ground-breaking clinical trials of stem-cell therapy he is conducting in China.

"Everybody assumed that I'm doing this in China because I wanted to escape George W. Bush, but that's not the case at all," Young told AFP in an interview, recalling the former US president's 2001 decision to effectively stop Federal funding of embryonic stem cell research.

"I started the clinical trials in 2005 here in Hong Kong ... mainly because of a promise that I made to a young woman. Her name is Sang Lan."

Sang crushed her spine during a routine warm-up exercise at the Goodwill Games in New York in 1998. She met Young as she underwent treatment and rehabilitation in the United States over the next 12 months.

"Her parents came to me and asked whether or not there would ever be a cure for her, and I said we're working very hard on it," recalled Young, who was by then one of the leading US experts on spinal cord injuries.

"When she went back to China after doing her rehabilitation in New York she cried and asked how would therapies go from the United States to China.

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Doctor looks to China for spinal injury 'cure'

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Newswise (PHILADELPHIA) Researchers at the Rothman Institute at Jefferson have shown that patients who receive surgery less than 24 hours after a traumatic cervical spine injury suffer less neural tissue destruction and improved clinical outcomes. The results of their study, the Surgical Timing in Acute Spinal Cord Injury Study (STASCIS) are available in PLoS One.

This practice-changing study is the first to show that the timing of surgery after traumatic spinal cord injury (SCI) matters, says Alexander Vaccaro, MD, PhD, professor of Orthopaedics and Neurosurgery at Jefferson Medical College of Thomas Jefferson University and attending surgeon at Thomas Jefferson University Hospital, the largest spinal cord injury center in the country.

The multicenter study recruited 313 patients; 182 of whom underwent surgery less than 24 hours after traumatic cervical SCI and 131 of whom underwent surgery at or after 24 hours post-SCI.

For both groups, the degree of neurologic improvement was measured by change in American Spinal Injury Associations (ASIAs) ASIA Impairment Scale (AIS). A two-grade improvement in AIS scores post-surgery was associated with improved neurologic outcomes. Baseline neurological assessments were performed within 24 hours of injury on all subjects.

A total of 222 patients were followed to six months post-surgery.

In the early surgery group (surgery performed less than 24 hours post-injury), 42.7 percent showed no improvement, 36.6 percent had a one grade improvement, 16.8 percent had a two-grade improvement and 3.1 percent had a three grade improvement. Comparatively, in the late surgery group (surgery performed at 24 hours or more post-injury), 50 percent showed no improvement, 40.7 percent had a one grade improvement and 8.8 percent had a two grade improvement.

What this tells us is that the odds of a significant (at least two grade) improvement in neurologic status is 2.8 times higher when surgery is performed within 24 hours post-injury. This can be the difference between walking and not for the rest of ones life, says Vaccaro.

Complications occurred in 24.2 percent of early surgery patients versus 30.5 percent of late surgery patients.

Previous research has been inconclusive on the issue, with the common thought among most surgeons that you can wait up to five days post-injury and have the same outcomes. We should not practice that way anymore armed with this new information, says Vaccaro.

Research was performed in collaboration with the University of Toronto; University of Virginia; University of Maryland, Baltimore; University of British Columbia; and the University of Kansas.

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Decompression Surgery Performed Less than 24 Hours After Traumatic Cervical Spinal Cord Injury Leads to Improved ...

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

Contact: Lee-Ann Landis leeann.landis@jefferson.edu 215-955-2240 Thomas Jefferson University

(PHILADELPHIA) Researchers at the Rothman Institute at Jefferson have shown that patients who receive surgery less than 24 hours after a traumatic cervical spine injury suffer less neural tissue destruction and improved clinical outcomes. The results of their study, the Surgical Timing in Acute Spinal Cord Injury Study (STASCIS) are available in PLoS One.

"This practice-changing study is the first to show that the timing of surgery after traumatic spinal cord injury (SCI) matters," says Alexander Vaccaro, MD, PhD, professor of Orthopaedics and Neurosurgery at Jefferson Medical College of Thomas Jefferson University and attending surgeon at Thomas Jefferson University Hospital, the largest spinal cord injury center in the country.

The multicenter study recruited 313 patients; 182 of whom underwent surgery less than 24 hours after traumatic cervical SCI and 131 of whom underwent surgery at or after 24 hours post-SCI.

For both groups, the degree of neurologic improvement was measured by change in American Spinal Injury Association's (ASIA's) ASIA Impairment Scale (AIS). A two-grade improvement in AIS scores post-surgery was associated with improved neurologic outcomes. Baseline neurological assessments were performed within 24 hours of injury on all subjects.

A total of 222 patients were followed to six months post-surgery.

In the early surgery group (surgery performed less than 24 hours post-injury), 42.7 percent showed no improvement, 36.6 percent had a one grade improvement, 16.8 percent had a two-grade improvement and 3.1 percent had a three grade improvement. Comparatively, in the late surgery group (surgery performed at 24 hours or more post-injury), 50 percent showed no improvement, 40.7 percent had a one grade improvement and 8.8 percent had a two grade improvement.

"What this tells us is that the odds of a significant (at least two grade) improvement in neurologic status is 2.8 times higher when surgery is performed within 24 hours post-injury. This can be the difference between walking and not for the rest of one's life," says Vaccaro.

Complications occurred in 24.2 percent of early surgery patients versus 30.5 percent of late surgery patients.

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Surgery less than 24 hours after traumatic cervical spinal cord injury leads to improved outcomes

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WASHINGTON, DC--(Marketwire -03/05/12)- The Alliance for Regenerative Medicine issued the following statement today: "An article about stem cell treatments taking place in Texas published by Nature last week is extremely troubling. The article suggests that patients are being administered stem cell treatments that have not been systematically demonstrated to be safe and effective therapies through the established FDA regulatory process.

"Cell therapy treatments, including those using adult stem cells, hold the promise of providing patients with treatments and cures for numerous diseases and disabilities. However, FDA regulation is key to ensuring that the treatments available to patients are safe and effective.

"The Alliance for Regenerative Medicine (ARM), a non-profit organization whose mission is to promote increased funding and development of regenerative medicine products, believes cell therapy and regenerative medicine products, including autologous cell therapy products, must go through the rigorous safety testing that is part of the FDA regulatory process before they can be marketed to the public. These regulations are designed to promote safe collection, manufacture, storage, and use of human cells, and cellular and tissue based products. ARM members comply with these rules because they know that FDA oversight helps to prevent patients from exposure to potentially unsafe products.

"We urge all companies developing stem cell treatments to follow FDA rules governing research and product development. ARM remains committed to working with all stakeholders to ensure that safe and effective products reach patients as soon as possible."

About The Alliance for Regenerative Medicine (ARM) The Alliance for Regenerative Medicine (ARM) is a Washington, DC-based non-profit organization that promotes legislative, regulatory, reimbursement, and financing initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, and provides services to support the growth of its member companies and organizations. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

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The Alliance for Regenerative Medicine Statement on Use of Cell Therapies Not Approved by the Federal Drug ...

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Released: 3/5/2012 12:00 PM EST Embargo expired: 3/6/2012 2:30 PM EST Source: Laurantis Pharma

Newswise (Ventura, CA, March 6, 2012) Lymphedema Awareness Day was established to support the needs of patients with lymphedema and to raise awareness for the need to develop new treatment options. Today, researchers have presented investigational pre-clinical data on the first potential pharmacologic agent used in combination with surgery for the treatment of breast cancer associated lymphedema at the Gordon Research Conference for Molecular Mechanisms in Lymphatic Function & Disease.

Currently there is no advanced pharmacologically-based therapeutic for lymphedema, said Stanley G. Rockson, MD, Director, Center for Lymphatic and Venous Disorders, Stanford University School of Medicine, and Principal Investigator of the upcoming first in human trial for Lymfactin. Although the research Is still early, the preliminary data show a remarkable improvement in the ability to successfully conduct lymph node transplant surgery with the benefit of Lymfactin.

Lymphedema occurs in about 20 percent of the three million patients with breast cancer.The condition occurs from trauma to the lymph nodes, and blockages in the lymphatic system as a result of breast cancer treatment. The symptoms include painful swelling and inflammation of the limbs.

Turku, Finland-based Laurantis Pharma Oy is developing Lymfactin, a vascular endothelial growth factor C (VEGF-C) in an adenoviral vector, for the treatment of secondary lymphedema associated with breast cancer. The therapy with Lymfactin involves a surgical operation where a lymph node flap is harvested from the patients lower abdominal wall and injected with Lymfactin, which leads to the transient presence of the adenovirus containing the VEGF-C gene. The lymph node is then transferred to the axillary region.

This new treatment is very promising, said Wendy Chaite, Founder, Lymphatic Research Foundation, a national organization devoted to advancing lymphatic research and to finding improved treatments and cures for lymphedema and lymphatic diseases. Lymphedema is one of those seldom discussed but far too common conditions that biotech and pharmaceutical companies have yet to explore. Although this research is at an early stage, the lack of any advanced treatments makes this research all the more important for those who suffer from this condition.

Professor Kari Alitalo of the University of Helsinki who made the discovery that the growth factor VEGF-C regulates the growth and development of the lymphatic system in humans and other mammals, presented the findings. From this discovery and in association with his co-workers Dr. Tuomas Tammela and Dr. Anne Saaristo, they identified that if VEGF-C is injected into tissues in mice and subsequently in pigs, growth of new lymphatic vessels and the restoration of the lymphatic architecture is catalyzed. Previous studies have shown that transferring lymph nodes from the inguinal region to the axillary region in patients with secondary lymphedema following their treatment for breast cancer was found to offer a slight improvement in their condition. Removal of old scar tissue from the axilla is considered an important step of this procedure.

The team in Finland then went a step further and showed that by combining VEGF-C injections with lymph node transfer in animal models using mice and then pigs, the response seen was even better than lymph node transfer alone. The results in mice indicated that lymphedema treated with lymph node transfer alone resulted in about 20% improvement. However, when combined with the administration of VEGF-C, the overall response was increased to around 80%.

In collaboration with Dr Rockson, Dr Anne Saaristo, MD and Prof. Kari Alitalo, Laurantis researchers are developing a treatment to potentially enable much higher transfer success. The Company plans to start a phase I/II study in patients with breast cancer associated lymphedema in early 2013. Lymfactin is an investigational therapy and has not been approved by the U.S. Food and Drug Administration.

Target Indication: Breast Cancer Associated Lymphedema The impairment of lymphatic drainage caused by dysfunction of the lymphatic vasculature leads to an accumulation of proteins and associated fluid, and finally to lymphedema-a chronic progressive swelling of the affected tissues. Lymphedema can either be hereditary - in which case it is diagnosed as primary lymphedema - or it can occur as a consequence of a disease, trauma, surgery, or radiotherapy and-thereby diagnosed as secondary lymphedema.

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On Lymphedema Awareness Day, Researchers Present Pre-Clinical Data Offering Cautious Hope for Breast Cancer Patients ...

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Research Explores the Role of the SRY Gene in the Male Fight-Or-Flight Response.

The pulse quickens, the heart pounds and adrenalin courses through the veins, but in stressful situations is our reaction controlled by our genes, and does it differ between the sexes?

Australian scientists, writing in BioEssays, believe the SRY gene, which directs male development, may promote aggression and other traditionally male behavioural traits resulting in the fight-or-flight reaction to stress.

Research has shown how the body reacts to stress by activating the adrenal glands which secrete catecholamine hormones into the bloodstream and trigger the aggressive fight-or-flight response. However, the majority of studies into this process have focused on men and have not considered different responses between the sexes.

Historically males and females have been under different selection pressures which are reflected by biochemical and behavioural differences between the sexes, said Dr Joohyung Lee, from the Prince Henrys Institute in Melbourne. The aggressive fight-or-flight reaction is more dominant in men, while women predominantly adopt a less aggressive tend-and-befriend response.

Dr Lee and co-author Professor Vincent Harley, propose that the Y-chromosome gene SRY reveals a genetic underpinning for this difference due to its role in controlling a group of neurotransmitters known as catecholamines. Professor Harleys earlier research had shown that SRY is a sex-determining gene which directs the prenatal development of the testes, which in turn secrete hormones which masculinise the developing body.

If the SRY gene is absent the testes do not form and the foetus develops as a female. People long thought that SRYs only function was to form the testes said Professor Harley. Then we found SRY protein in the human brain and with UCLA researchers led by Professor Eric Vilain, showed that the protein controls movement in males via dopamine.

Besides the testes, SRY protein is present in a number of vital organs in the male body, including the heart, lungs and brain, indicating it has a role beyond early sex determination, said Dr Lee. This suggests SRY exerts male-specific effects in tissues outside the testis, such as regulating cardiovascular function and neural activity, both of which play a vital role in our response to stress.

The authors propose that SRY may prime organs in the male body to respond to stress through increased release of catecholamine and blood flow to organs, as well as promoting aggression and increased movement which drive fight-or-flight in males. In females oestrogen and the activation of internal opiates, which the body uses to control pain, may prevent aggressive responses.

The role of SRY regulation of catecholamines also suggests the gene may have a role in male-biased disorders such as Parkinsons disease.

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Men Respond More Aggressively Than Women to Stress, and it’s All Down to a Single Gene

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

Contact: Dr. Hilary Glover hilary.glover@biomedcentral.com 44-203-192-2370 BioMed Central

Spinal muscular atrophy (SMA) is an incurable, and progressive, disease caused by an inheritable defect in the gene SMN1. Depending on the severity of the mutation it can result in the loss of spinal cord motor neurons, muscle wasting (atrophy) and even death of an affected child. A new study published in Biomed Central's open access journal BMC Medicine shows that Fasudil, a ROCK inhibitor, can improve both the size of muscle fibers and their connection to motor neurons. Fasudil also increased the lifespan and improved the movement of SMA mice.

SMA affects 1 in 6,000 births and is the leading cause of death in young children. In its less severe form the muscle wasting of SMA traps bright young children within their bodies. Researchers from the Ottawa Hospital Research Institute and the University of Ottawa realized that SMA caused problems in regulation of the ROCK intracellular signaling pathway and that inhibiting this pathway could increase the lifespan of SMA mice.

By targeting the ROCK pathway in spinal cord and muscles, Fasudil bypasses the genetic defect SMN1. Dr Kothary, who led the team, explained, "Fasudil increased the lifespan of SMA mice from 30 to 300 days, allowing them to survive well into adulthood. Although it had no apparent effect on the damaged neurons themselves, Fasudil increased muscle size and the endplate junction between muscles and their motor neurons. Consequently, the mice were also better coordinated, better groomed, and could move about more freely than untreated SMA mice."

Melissa Bowerman from the Ottawa Hospital Research Institute continued, "Finding a cure for SMA is still a long way off, however we hope that treatment with drugs like Fasudil, which goes some way towards restoring normal developmental, or HDAC inhibitors, which alter how genes are regulated, along with nutrition and physiotherapy will provide a package of therapy to improve the quality and length of life of SMA children."

###

Notes to Editors

1. Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy Melissa Bowerman, Lyndsay M Murray, Justin G Boyer, Carrie L Anderson and Rashmi Kothary BMC Medicine (in press)

Please name the journal in any story you write. If you are writing for the web, please link to the article. All articles are available free of charge, according to BioMed Central's open access policy.

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Fasudil bypasses genetic cause of spinal birth defect

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ScienceDaily (Mar. 6, 2012) Scientists from the Ottawa Hospital Research Institute (OHRI) and the University of Ottawa (uOttawa) have discovered that a drug called fasudil can extend the average lifespan of mice with Spinal muscular atrophy (SMA) from 30.5 days to more than 300 days.

The study is published March 6 in BioMed Central's open access journal BMC Medicine, by Dr. Rashmi Kothary, his graduate student Melissa Bowerman and others.

SMA is the leading inherited cause of death in infants and toddlers, affecting approximately 25,000 people in Canada and the United States. Scientists have known for many years that this disease is caused by inherited mutations in a gene called survival motor neuron 1 (SMN1). Most early attempts at developing treatments for SMA focused on replacing this gene, however, Dr. Kothary's group has focused on understanding and targeting the physiological defects present in certain nerve cells with SMA. These cells have a weakened internal scaffold, which hinders their ability to connect with muscle cells and contributes to the severe muscle weakness associated with SMA.

Two years ago, Dr. Kothary and his team showed that a laboratory compound called Y-27632, which targets an enzyme that is involved in maintaining the cellular scaffold, could greatly increase lifespan in a certain mouse model of SMA. In this new study, they tested a compound called fasudil, which is similar to Y-27632, but has the advantage that it has already been approved for human clinical trials for other conditions, meaning that it could possibly be re-targeted to use in clinical trials for SMA more quickly than a completely new drug.

The Kothary group found that fasudil-treated SMA mice survived for an average of more than 300 days, compared to just 30.5 days for untreated SMA mice. However, the average lifespan of fasudil-treated SMA mice was still only about half as long as that of normal mice. Fasudil-treated SMA mice also had larger muscle fibres than the untreated SMA mice, and they behaved more normally with respect to grooming and other regular activities. However, they did not perform any better in strength and balance tests and they still had low numbers of motor neurons, which is typical for SMA.

"Our study is important because it expands a new area of research into SMA, which could lead to the development of new treatments," said Melissa Bowerman. "Of course, this research is still at the early stages and although we found that fasudil could significantly increase lifespan in a mouse model of SMA, this drug couldn't correct all the problems in these mice, and it had serious side effects when used at higher doses."

"A number of groups are working to develop fasudil-like compounds with fewer side effects, and we're very excited to see how these perform in our models, and hopefully in human SMA clinical trials some day," said Dr. Kothary "However, we continue to believe that SMA is a disease that will best be addressed using multiple strategies together, including nutrition and possibly drug, cell and gene therapies."

"Dr. Kothary's group has been a pioneer in SMA research, both in characterizing the impact of SMA on tissues and organs, and in discovering a novel therapeutic pathway involving enzymes that target the cell scaffold," said Dr. Alex MacKenzie, an expert in SMA at CHEO Research Institute and the University of Ottawa, who was not involved in the study. "It has to be said that this approach was not intuitively obvious and Dr. Kothary and his team are to be commended for their creativity in its discovery. It represents an important addition to the armamentarium of experimental SMA treatments."

Although fasudil has been approved by the U.S. Food and Drug Administration for use in certain adult human clinical trials, it is still considered experimental, and has not been approved for the treatment of any human condition in the United States or Canada. Individuals who are interested in experimental therapies should discuss this with their health care professional.

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New approach for treating genetic muscle wasting disease shows promise in mice

Recommendation and review posted by Bethany Smith