协和医院与贝瑞和康无创产前临床试验圆满完成

2012-05-24 09:49:44  互联网  编辑：  在线咨询好大夫  由北京协和医院与北京贝瑞和康生物技术有限公司联合进行的针对胎儿染色体非整倍体疾病的大规模无创DNA产前检测临床试验已于2012年5月18号顺利完成，并取得圆满成功。该试验是国际上首个大规模前瞻性的针对胎儿染色体非整倍体疾病的无创DNA产前检测临床试验。经过双方接近1年的共同努力，通过严格的临床随访，本次临床试验的结果显示，无创DNA产前检测技术对于孕12周以上胎儿的三大染色体非整倍体疾病(唐氏综合征、爱德华氏综合征、帕陶氏综合征)的准确率接近100%，假阳性率为0.05%。该结论与2011年由湘雅医院和贝瑞和康共同完成的回顾性临床试验结果几乎完全一致。阶段性研究成果已由北京协和医院于2012年4月28日在沈阳召开的"2012盛京母胎医学论坛"上公布，其结果获得了行业内各方专家的关注与好评。该临床试验是按照国际标准设计与执行，利用贝瑞和康自主研发的无创DNA产前检测平台，对来自于北京协和医院和北京市海淀区妇幼保健院的2236例门诊随机样本进行母体外周血中游离DNA的深度测序，并结合生物信息学分析的方法，对游离DNA中包含的胎儿片段信息进行定量分析并对其遗传状态做出判断。本次前瞻性大规模临床试验揭示了无创DNA产前检测技术作为一项新型的产前检测技术，具有安全、准确、快速的特点，明显优于现在血清学筛查技术，同时由于该技术仅需抽取孕妇的外周血即可准确判断胎儿是否患有染色体非整倍体疾病，因而可有效避免侵入性产前检测带来的感染和流产风险，极大地降低孕妇及其家属的心理负担，代表了当前产前检测和防止先天性缺陷儿出生技术的最新发展方

关于北京协和医院北京协和医院是集医疗、教学、科研于一体的大型三级甲等综合医院，是北京协和医学院的临床学院、中国医学科学院的临床医学研究所，是卫生部指定的全国疑难重症诊治指导中心，也是最早承担干部保健和外宾医疗的医院之一。在2011年、2012年复旦大学医院管理研究所公布的"中国最佳医院排名榜"中名列榜首

Folic acid intake associated with reduced risk of autism: study

 June 14, 2012 in Autism spectrum disorders (Medical Xpress) -- A new study by researchers at the UC Davis MIND Institute suggests that women who consume the recommended daily dosage of folic acid, the synthetic form of folate or vitamin B-9, during the first month of pregnancy may have a reduced risk of having a child with autism. The study furthers the researchers' earlier investigations, which found that women who take prenatal vitamins around the time of conception have a reduced risk of having a child with autism. The current study sought to determine whether the folic acid consumed in those supplements was the source of the protective effect. The finding suggests that, in addition to women who already have conceived, those who are attempting to become pregnant should consider consuming folic acid supplements, the authors said. The study found that women who each day consumed the recommended amount of folic acid (600 micrograms, or .6 milligrams) during the first month of pregnancy experienced a reduced risk of having a child with autism spectrum disorder, specifically when the mother and/or her child had a specific genetic variant (MTHFR 677 C>T) associated with less efficient folate metabolism. The study will be published in the July issue of the American Journal of Clinical Nutrition. "This research is congruent with the findings of earlier studies that suggest that improved neurodevelopmental outcomes are associated with folic acid intake in early pregnancy," said lead study author Rebecca J. Schmidt, assistant professor of public health sciences in the UC Davis School of Medicine and a researcher with the UC Davis MIND Institute. "It further supports recommendations that women with any chance of becoming pregnant should consider consuming folic acid at levels of 600 micrograms or greater per day." Autism is a neurodevelopmental disorder characterized by impairments in social interaction, communication deficits and repetitive behaviors and often is accompanied by intellectual disability. An estimated 1 in 88 children born today will be diagnosed with autism spectrum disorder, according to the U.S. Centers for Disease Control and Prevention. "What's reassuring here is knowing that, by taking specific action in terms of their intake of folic acid from food or supplements, women can reduce the risk of autism spectrum disorder in their future children," said study senior author Irva Hertz-Picciotto, chief of the division of environmental and occupational health in the Department of Public Health Sciences and a MIND Institute researcher. The study authors said that folic acid offers protection against problems in embryonic brain development by facilitating DNA methylation reactions that can lead to changes in the way that the genetic code is read. An ample supply of methyl donors such as folic acid could be especially important in the period around conception, when the DNA methylation road map is set forth. For the study, the researchers collected data from approximately 835 Northern California mothers of 2- to 5-year-old children who had autism, developmental delay or typical development and who were participants in the Childhood Autism Risk from Genetics and the Environment (CHARGE) study between 2003 and 2009. Each mother's average daily folic acid intake was assessed on the basis of the amount and the frequency of consumption of folic acid-containing dietary supplements such as prenatal vitamins and multivitamins, as well as the consumption of food supplemented with folic acid such as fortified breakfast cereals or energy bars. Information was collected for the period when the women were pregnant and for the three months before they became pregnant. The study found that mothers of typically developing children reported greater-than-average intake of folic acid, and were more likely to meet intake recommendations during the first month of pregnancy than were mothers of children with autism spectrum disorder. Among study participants, as the amount of folic acid consumed increased, the associated risk for autism spectrum disorder decreased. Mothers of children with developmental delay tended to have lower estimated folic acid intake when compared with mothers of typically developing children during the three months before pregnancy. The mothers of infants who were developing normally said they consumed an average 779 micrograms of folic acid daily and 69 percent of them at least met the daily guidelines. The mothers of children with autism consumed an average of 655 micrograms of folic acid. Fifty-four percent of them consumed the recommended 600 micrograms or more per day Consuming supplemental folic acid before and during early pregnancy has been recommended for decades, after studies demonstrated its potential to prevent up to 70 percent of neural tube defects, or improper formation of the embryonic brain and spinal cord. Folic acid's protective effect on neural tube defects also was stronger when mothers and/or children carried the MTHFR 677 C>T gene variant. Early maternal folic acid supplementation has more recently been shown to improve other social, attention and behavioral outcomes in the developing child.  Journal reference: American Journal of Clinical Nutrition 

誰會承認是 patent troll? patent troll 應有新定義 !

InterDigital's Bill Merritt on patent trolls, standards development and disputes with the big boys By Daniel Cooper  posted Jun 14th 2012 10:00AM Interview You probably won't know the name, but you most certainly use its technology on a daily basis. InterDigital is a pioneering company that helped develop WCDMA, 3G and HSDPA during its 50-year history. It counts former Apple CEO Gil Amelio as one of its directors, but the only time you'll ever hear its name is when it's embroiled in litigation.  Either because it's suing, or being sued for licensing fees in the complex, murky world of wireless technologies, it's easy to get the idea that InterDigital is a patent troll. A name that, both Nokia and most recently, Huawei have barely stopped short of throwing at the company.  But what's it like being painted as the villain in the wireless business pantomime? Company president and CEO William "Bill" Merritt took the time to answer some of our questions, talk about what the company actually does, what's in the future and why they definitely aren't a patent troll. Let's first talk about "patent trolls," can you explain to our readers the distinction between yourselves and "trolls," and how you feel about the term being applied to InterDigital?  I would say I'm uncomfortable with creating a class system around patent holders, and over the past few years we've seen companies that are clearly not trolls adopt more aggressive behavior around monetizing their portfolios. Traditionally, people see trolls as companies that don't do their own development, or don't have products. 

"It's always very trying to be lumped into that category." We're very different and, given that description, it's always very trying for us to be lumped into that category: we have hundreds of engineers, we participate in and often chair standards committees, and we partner with leading operators, infrastructure companies and solution providers to develop and test new solutions. At Mobile World Congress this year, we demonstrated technologies – all of them developed in-house – with partners like Alcatel-Lucent and Spectrum Bridge. Technologies that we expect to see in the marketplace in four to five years – that's how far ahead of the curve we often work. 

Your position in the market is to wireless companies what ARM is to chip design, in so much that you license your wares to others. Do you ever feel that you're missing out by not producing your own hardware?  I don't think we do. First of all, we do build: every one of our development projects involves building out a complete system so we can test our technologies. We just don't focus on getting those products into a form factor that can be sold, because other companies are far better at that. More broadly, I think direct concern over immediate products in the market would possibly detract from our focus on absolute leading-edge technologies. Our eye, and our research budget, is always on what the wireless world will need and move towards in the future... not what it would be willing to buy at a specific price point today. So, in a sense, not having products is beneficial for us in our work on pioneering technologies, especially within standards. It might give us a perspective that other standards participants, all of whom are seeking market advantage, don't have. 

To what extent are companies like InterDigital involved when a new standard is being developed? For example, if the ITU started discussing "5G" tomorrow (for example), would you be lobbying to handle the research or is it a race between the various parties to patent as much technology as possible?  I think it's not so much a race as it is a competition. There are a certain number of technical challenges that need to be solved, and engineers compete to develop the best, clearest, most elegant and most easily implemented solution. We compete alongside them – and again, not having products frees us to examine all aspects of a new standard's requirements. InterDigital's key is that we don't wait for standards bodies to launch a project to begin research: we look at the challenges of current technology – for example, in power consumption, or interference control, or policy control from the operator perspective – and determine what solutions would be needed to solve those problems. 

Let's talk now about the creation of the patents. Is all of your research undertaken in-house or do you also purchase some in from universities or other companies?  So far, nearly all our development has been done in-house. We have more than 200 engineers, and R&D facilities in Philadelphia, New York, Montreal and San Diego. We're not against patent acquisitions. But, mainly they align with technologies in which we've built significant in-house portfolios, or where we intend to begin R&D efforts. 

Are you continuing to add to your patent portfolio, and if so, what direction have those patents taken? What's the next big innovation in the space?  As we always have, we're continuing to develop new technologies and find new solutions to issues, standards-based or otherwise, and as a result we're continuing to add to our patent portfolio, at a rate of over 1,000 patents and applications per year. From a business perspective, it's essential. The market for patents has changed, and as a public company we're focused on growing our value. One of the ways we're seeking to do this is through patent portfolio sales, and selling patents implies relying on our ability to develop new ones. So we are, absolutely: it's how we expect to draw the maximum value from our strong engineering team.  "The market for patents has changed." In terms of next big innovation, some of the key technologies we're working on right now involve bandwidth management – aggregating, segmenting and managing bandwidth, from both the device and the operator perspective. We're also doing tremendous work in spectrum aggregation, integrating TV white space and various other spectrum resources, licensed and unlicensed, into a comprehensive system. Both these technologies are rooted in our sense of a migration from fixed to dynamic networks. We're also doing work in standards-based machine-to-machine communications. We see that, and the impact standards have on driving down costs and expanding capability, as being a huge milestone in the development of wireless and the internet of things. 

Who do you work with when developing your patents? Do you have much input from the Apple & RIMs of this world?  We partner with a number of companies during the research and testing phases, so we constantly get input. And we attend more than 100 engineering and academic conferences a year, submitting contributions, presenting – we recently presented our roadmap through 2020 at the LTE World Summit in Barcelona. Whether it's standards work or efforts that we hope will underpin the wireless technologies of the future, we're in constant discussion with the wireless engineering community. 

As standards are becoming more universal, is there a greater need for licenses to be negotiated centrally rather than individually to prevent new entrants to the wireless market becoming bogged down in disputes?  One of the beautiful aspects of the wireless industry is the constant change and emergence of new products, companies, business models. Similarly, there are many, many ways to license a technology, depending on the market position of the licensee, their product, their revenue model, etc. I think companies need the freedom to talk to each other, and craft their own solutions. 

"Sometimes, disputes cannot be avoided." And bear in mind, the vast majority of our license agreements are reached quietly, with mutual agreement and minimal fanfare. I know disputes are messy, and that the engineering world looks down on them. But sometimes disputes cannot be avoided, and I personally think that companies need to maintain the independence to govern themselves the way they choose. 

How do you calculate the worth of your patents? Is there a flat rate per handset or are there multipliers based upon the profitability and quantity of handsets in the market? Is there a different set of fees based on tablets and broadband dongles compares to phones?  As I mentioned before, there is a broad range of types of agreements, depending on product type, fixed payments vs. percentage royalties, and many other variables. Our basic practice is to achieve what we feel is a fair value for our contributions, but also to put the licensee in a position to be successful. No one would gain, including us, if the royalties on a product made the product unsuccessful. 

Huawei made a complaint to the European Union, alleging that you were demanding too much money for a standards-essential patent -- echoing a claim that Nokia has made previously. Under FRAND terms, how much room is there for negotiation or do you offer a flat price for every company?  I obviously can't say a great deal about ongoing discussions with specific companies. I can only repeat what we said in our response to the announcement of Huawei's complaint: standards bodies specify commitments that participating companies should uphold, and InterDigital takes those commitments very seriously. Again, we've signed over 50 licensing agreements, the vast majority with no dispute and great mutual understanding. 

More Info Huawei files EU antitrust complaint against InterDigital InterDigital files ITC complaint against Nokia, Huawei, and ZTE for 3G patent infringement Apple licenses InterDigital tech, presumably for 3G iPhone One website petitioned for company insiders to leak details about your business dealings. Given that you largely operate "behind the scenes," has the increased attention of the internet been a boon or a burden to your day to day lives?  I think that, in the way you're asking, it's been neither – it's just the ongoing reality that all public companies must operate under. But the internet has been a boon to InterDigital, definitely, because it's driving the demand for data, and the technological struggle to provide it! Technology is about limitations – data rate, power supply, spectrum, processing – and as long as the ecosystem makes it desirable to exceed those limitations, offering great applications and new capabilities, InterDigital will be in business.  

Osiris干细胞药物第二个营销许可 (New Zealand) for GvHD in children

2012年06月15日05:40 来源：北美环球财经  Osiris Therapeutics, Inc. (NASDAQ:OSIR) 公布新西兰已经授予公司一级的干细胞疗法Prochymal?营销许可，这是用来治疗儿童急性移植物与宿主病(GvHD)的药物。为此新西兰加入加拿大的行列，上个月加拿大成为世界上第一个批准干细胞药物的国际公认的监管机构。Prochymal也是第一个获得批准的针对GvHD的药物，GvHD是毁灭性的骨髓移植并发症，80%的感染儿童都难以避免死亡，许多都是出现在诊断後的几周内。

Osiris Receives Second Approval for Life-Saving Stem Cell Drug; Prochymal Granted Marketing Consent by New Zealand June 14, 2012, 8:00 a.m. EDT COLUMBIA, Md., Jun 14, 2012 (BUSINESS WIRE) -- Osiris Therapeutics, Inc. OSIR +14.14%  , announced today it has received consent from New Zealand to market its first-in-class stem cell therapy Prochymal(R) (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. With this decision New Zealand joins Canada, which last month became the world's first internationally recognized regulatory authority to grant approval to a stem cell drug. Prochymal is also 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.  "With each of our approvals it becomes clearer that the time for life-saving stem cell therapies in the practice of medicine has arrived, and we are humbled to have a leading role," said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. "I would like to thank the professionals at Medsafe for their thoughtful and expeditious review of this complex application. I would also like to thank the team at Osiris that continues to do an outstanding job of making Prochymal available to children around the world suffering from the devastating effects of GvHD."  Osiris submitted a New Medicine Application (NMA) to Medsafe (New Zealand's medical regulatory agency) in May of 2011, and was granted Priority Review in June of 2011. Priority review provides expedited review for new drugs which offer a significant clinical advantage over current treatment options. Prochymal was granted provisional consent under Section 23 of the Medicines Act 1981.  "The incidence of GvHD is likely to rise as the demographic profile of our transplant population evolves," said Hans Klingemann, M.D., Ph.D., a Professor of Medicine and the Director of the Bone Marrow & Hematopoietic Stem Cell Transplant Program at Tufts University School of Medicine. "Effective strategies to manage the often lethal consequences of GvHD reduce the overall risk to transplantation and provide the transplant physician with better options when approaching their most difficult cases."  Clinical trials have shown that Prochymal is able to induce an objective, clinically meaningful response in 61-64 percent of children with GvHD that is otherwise refractory to treatment. Furthermore, treatment response with Prochymal resulted in a statistically significant improvement in survival.  "As a mother who watched my son Christian suffer and die from the horrifying effects of GvHD, while waiting for the regulatory approvals necessary to allow him access to Prochymal, words cannot express how happy I am that significant progress is finally being made," said Sandy Barker, President and Co-founder of the Gold Rush Cure Foundation. "We are proud to stand side-by-side with Osiris in this historic battle for our children around the world. Our motto is 'not one more child, not one more family' and when it comes to GvHD mortality, zero is the only acceptable number."  Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program (EAP). It is expected that Prochymal will be commercially available in New Zealand later this year. 

About GvHD  GvHD represents a major unmet medical need with no approved treatment until Prochymal. GvHD is the leading cause of transplant related mortality, in which immune cells contained within the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. Severe GvHD can cause blistering of the skin, intestinal hemorrhage and liver failure. Severe GvHD is extremely painful and fatal in up to 80 percent of cases. Currently, steroids are used as first-line therapy with a success rate of only 30-50 percent. When steroids fail, treatment options are limited to immunosuppressive agents used off-label with little benefit and significant toxicities. 

About Prochymal (remestemcel-L)  Prochymal is the world's first approved drug with a stem cell as its active ingredient. Developed by Osiris Therapeutics, Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor. Prochymal is truly an off-the-shelf stem cell product that is stored frozen at the point-of-care and infused through a simple intravenous line without the need to type or immunosuppress the recipient. Prochymal is approved in Canada and New Zealand for the management of acute graft-versus-host disease (GvHD) in children and is available for adults and children in eight countries including the United States, under an Expanded Access Program. Prochymal is currently in Phase 3 trials for refractory Crohn's disease and is also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes. 

About Osiris Therapeutics  Osiris Therapeutics, Inc. is the leading stem cell company, having developed the world's first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets. In Biosurgery, Osiris currently markets Grafix(R) for burns and chronic wounds and Ovation(R) for orthopedic applications. Osiris is a fully integrated company with capabilities in research, development, manufacturing and distribution of stem cell products. Osiris has developed an extensive intellectual property portfolio to protect the company's technology, including 48 U.S. and 144 foreign issued patents.  Osiris, Prochymal, Grafix and Ovation are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, www.Osiris.com . (OSIR-G) 

About Christian Barker & Gold Rush Cure Foundation  At only 13-years-old, Christian was diagnosed with a rare form of leukemia. The treatment for this cancer required a bone marrow transplant. However, due to the aggressive nature of the cancer, Christian relapsed and a second transplant was conducted. The treatment regimen consisted of extreme chemotherapy, radiation, and multiple surgeries. Despite the negative side effects from those treatments, nothing compared to the brutal suffering which occurred after he contracted graft-vs.-host disease, which painfully destroyed every part of Christian's body. The disease caused Christian's skin to peel off, and he vomited several liters of blood daily due to the destruction of his gastro intestinal tract. To make matters even worse, Christian experienced liver failure and suffered daily with extreme pain from head to toe. This condition was prevalent for the four months, and eventually lead to his death. No child should have to suffer like this, and watching Christian's body being destroyed by the disease was as horrifying for him as it was for our family and his caregivers.  After Christian died in December 2007, his mother and father, Sandy and Gary, established the Gold Rush Cure Foundation (GRCF), which is dedicated to raising public awareness and funding to help eradicate childhood cancer. GRCF is also committed to being advocates for children fighting cancer. In June, GRCF made its 11th trip to Washington, D.C., since Christian's passing to meet with lawmakers about the vital need for more funding and awareness for childhood cancer, which is the leading cause of death by disease for children. For more information, please visit, www.goldrushcure.org .  

QPS Taiwan using Pristima®Suite (preclinical data application)

Xybion and QPS Announce that QPS Taiwan has Selected Pristima® Suite as their Preclinical Data Management System Xybion's Pristima continues to accelerate high adoption and growth in the global CRO sector and announces that QPS Taiwan selects to implement Pristima Suite.  Cedar Knolls, NJ (PRWEB) May 24, 2012  Xybion Corporation and QPS LLC announced today that QPS Taiwan, a global preclinical toxicology services provider, has selected to deploy Pristima®Suite, Xybion’s premier preclinical application. QPS will leverage Pristima Suite in a ‘Software as a Service’ (SaaS) deployment model, supported by Xybion’s Singapore data center & technology services group. Pristima is an end-to-end preclinical data management system that helps companies to reduce their preclinical operational costs while decreasing time-to-market for drug discovery. QPS will be leveraging many key components of the preclinical software suite including the General Toxicology, Clinical Pathology, Necropsy, Pathology and SEND modules to capture, manage and report data from preclinical studies. Following an exhaustive selection process where all competitive products were considered, QPS Taiwan selected Pristima as a SaaS installation. Xybion will host the application in its Singapore data center. With the addition of QPS Taiwan, Xybion continues to expand its offerings in the Asia Pacific market, growing its client base to a total of 11 countries in North America, Europe and Asia. QPS Taiwan will be using the Pristima functionality to refine and expand its comprehensive GLP CRO preclinical study service capabilities to serve its clients worldwide. Carlos Frade, Vice President of Preclinical R&D Solutions at Xybion, was quoted: “QPS Taiwan selected the Software as a service model, which offers a less time consuming validation process and a more cost effective approach to achieve the required capabilities, maintenance, and support over the Internet. QPS Taiwan will leverage Xybion’s Total Preclinical Solutions package to install, validate and deploy the most modern and most well-integrated data management suite on the market.” Walter Bee, Ph.D., Vice President, Head of Global Safety Assessment and Regulatory Affairs, commented: “QPS Taiwan’s Center of Toxicology and Preclinical Sciences (CTPS) is one of the oldest and most experienced GLP laboratories in Asia. Our scientists combine training and experience from Europe, Japan, the USA and Asia in the unique environment of a first class Taiwanese laboratory. With the introduction of Pristima® as our data acquisition and management system, we are underlining our commitment to excellent service to our sponsors. This software enables our scientists to provide results quickly, reduces paperwork and increases data quality. It supports the organization, structure, and format of datasets in the Standard for Exchange of Non-Clinical Data (SEND) format for submission to the US Food and Drug Administration (FDA). The tailored solution Xybion provided fits our current needs but leaves room for expansion in line with our growth ambitions.” As an enterprise-grade solution suite for preclinical research, Pristima offers a powerful and robust toolset to improve clients’ efficiency, promote compliance and accelerate the final reporting. Dr. Pradip Banerjee, CEO, Xybion Corporation commented, “We are pleased to have QPS Taiwan as a client and look forward to assisting them in reaching their business goals. We are delighted that QPS Taiwan has decided to have Xybion host Pristima in our data center. Many of our clients find SaaS delivery to be an efficient, scalable and cost effective alternative to an onsite installation. We are committed to maintaining Pristima as the leading preclinical data management solution to meet the needs of a fast growing CRO.”

About Xybion Corporation:  Xybion Corporation is a global leader in the development and delivery of critical enterprise solutions to highly regulated industries, helping companies lower cost, mitigate risk and accelerate growth, while managing quality and compliance. Xybion is the maker of Pristima Suite, a leading preclinical data management solution for the life science sector. Pristima Suite is the only fully integrated preclinical and vivarium management solution that natively connects to enterprise quality & compliance management processes on the market today. 

About QPS  Founded in 1995, QPS is a GLP/GCP-compliant contract research organization (CRO) supporting discovery, preclinical and clinical drug development. We provide quality services to pharmaceutical and biotechnology clients worldwide. QPS operates regional laboratories and testing facilities which are located at its headquarters in Newark, DE, USA; Springfield, MO, USA; Groningen, The Netherlands; Hyderabad, India; and Taipei, Taiwan.