Gold nanoparticles capable of 'Unzipping' DNA
ScienceDaily (June 20, 2012) New research from North Carolina State University finds that gold nanoparticles with a slight positive charge work collectively to unravel DNA's double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.
"We began this work with the goal of improving methods of packaging genetic material for use in gene therapy," says Dr. Anatoli Melechko, an associate professor of materials science and engineering at NC State and co-author of a paper describing the research. Gene therapy is an approach for addressing certain medical conditions by modifying the DNA in relevant cells.
The research team introduced gold nanoparticles, approximately 1.5 nanometers in diameter, into a solution containing double-stranded DNA. The nanoparticles were coated with organic molecules called ligands. Some of the ligands held a positive charge, while others were hydrophobic -- meaning they were repelled by water.
Because the gold nanoparticles had a slight positive charge from the ligands, and DNA is always negatively charged, the DNA and nanoparticles were pulled together into complex packages.
"However, we found that the DNA was actually being unzipped by the gold nanoparticles," Melechko says. The positively-charged ligands on the nanoparticles attached to the DNA as predicted, but the hydrophobic ligands of the nanoparticles became tangled with each other. As this tangling pulled the nanoparticles into clusters, the nanoparticles pulled the DNA apart.
"We think gold nanoparticles still hold promise for gene therapy," says Dr. Yaroslava Yingling, an assistant professor of materials science and engineering at NC State and co-author of the paper. "But it's clear that we need to tailor the ligands, charge and chemistry of these materials to ensure the DNA's structural integrity is not compromised."
The finding is also relevant to research on DNA-based electronics, which hopes to use DNA as a template for creating nanoelectronic circuits. Because some work in that field involves placing metal nanoparticles on DNA, this finding indicates that researchers will have to pay close attention to the characteristics of those nanoparticles -- or risk undermining the structural integrity of the DNA.
The research was supported by the National Science Foundation.
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Healthpoint Biotherapeutics To Create State-of-the-Art Cell Sciences Manufacturing Facility
FORT WORTH, Texas, June 20, 2012 /PRNewswire/ --Healthpoint Biotherapeutics today announced the company is planning a significant upgrade to its 80,000 square foot research and manufacturing facility in Ft. Worth. The new, state-of-the-art facilities will comprise 25,000 square feet of manufacturing infrastructure, labs and clean room space for the production of cell-based therapies.
"We are very pleased to be embarking on this important and innovative initiative at our headquarters here in Fort Worth," noted Travis E. Baugh, President and Chief Operating Officer of Healthpoint Biotherapeutics. "The planned, ultra-modern facility will create an efficient platform to support our lead pipeline candidate, HP802-247, which recently completed a successful phase 2b study in venous leg ulcers. Importantly, the facility will also add critical capabilities as we look toward the promise of regenerative medicine."
The facility upgrade comes as the company is engaged in developing a pipeline of next generation wound care therapies based on cell and cell-matrix biology. The advanced nature of the facility will provide efficient and scalable manufacturing capabilities to support both development and future commercialization, as well as create a unique environment for novel collaboration and identification of new technologies.
Healthpoint Biotherapeutics is planning to invest approximately $60 million to build out and staff the research and manufacturing facility over the next few years. The company plans to add a total of 51 new employees in research and development (R&D), quality assurance and quality control, operations and information technology (IT), with 31 of these hires planned for 2012.
"We are thrilled to have Healthpoint Biotherapeutics locate their state-of-the-art manufacturing facility in our great city," said Fort Worth mayor Betsy Price. "This is the latest example of how life science related businesses are becoming a much bigger part of our diversified economy. "
"The establishment of this contemporary cell biology facility represents a significant advance for the Fort Worth life sciences footprint," added David Berzina, Executive Vice President of Economic Development with the Fort Worth Chamber of Commerce. "As such, we would like to emphasize the importance of this investment for the greater Ft. Worth community."
The project will be built out in two phases to coincide with current pipeline requirements. The first phase will include advanced tissue processing capabilities, master cell banking, production of clinical supplies for phase III trials and process development to support future manufacturing scale-up. Important features of the second, commercialization phase include automated closed system cell culturing, automated vial filling and the use of radio frequency identification (RFID) technology allowing efficient, robotic packaging in -80 degrees C freezers and product traceability in sealed dry ice shippers.
Construction of the new facilities is slated to begin in the second quarter, with the first phase anticipated to be operational by the end of 2012. Full validation and commissioning of the facility to release product for clinical trials is expected in 2013. Completion of the second phase will be timed to meet the requirements for the manufacture and ultimate commercialization of the investigational product HP802-247, following regulatory approvals.
Healthpoint Biotherapeutics already operates advanced, biologic manufacturing facilities worldwide, including its facility in Lausanne, Switzerlandwhich has received manufacturing authorization from the Swiss Regulatory Agency, Swissmedic, for the production of cell-based therapiesas well as a biologic manufacturing plant in Curacao.
About HP802-247 HP802-247 is an investigational allogeneic living human cell suspension that consists of two components that are sprayed sequentially on the wound bed at the time of treatment: a fibrinogen solution and a cell preparation containing a mixture of growth arrested, living, allogeneic epidermal keratinocytes and dermal fibroblasts. Based on in vitro studies, HP802-247 is believed to release various growth factors and cytokines into the micro-environment of the wound.
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CBR – World’s Largest Stem Cell Bank – Applies Two Decades of Experience to Advance Regenerative Medicine
SAN BRUNO, Calif., June 20, 2012 /PRNewswire/ -- Twenty years ago this month, CBR (Cord Blood Registry) in partnership with the University of Arizona, processed the first cord blood stem cell sample in the world to be stored specifically for family use. Since 1992, the number of conditions treated with cord blood stem cells has greatly expanded, and so has CBR. Today, CBR is the largest family cord blood bank in the world with more than 425,000 samples in storage a population the size of a major city like Miami. What distinguishes the "city of individuals" with newborn stem cells banked at CBR is the exclusive opportunity to participate in a growing number of ground-breaking clinical trials.
(Photo: http://photos.prnewswire.com/prnh/20120620/SF27549-INFO)
(Logo: http://photos.prnewswire.com/prnh/20120216/AQ54476LOGO)
"As the leader and innovator in family banking, we believe every newborn deserves a healthy future and that we have a responsibility to lead the way," said Heather Brown, vice president of scientific & medical affairs at CBR. "Looking back, the creation of our bank allowed families for the first time to preserve a genetically-related source of newborn stem cells, ready and available if needed for a lifesaving transplant to regenerate a person's immune system after radiation or chemotherapy. As we look to the future, we are helping shape new areas of regenerative medicine. We are the only family bank actively pioneering clinical trials evaluating new therapeutic uses of cord blood stem cells for unexpected injuries and conditions with no current cure."
Expanding Areas of Clinical Research: Helping the Body Heal Injured Nerves Until very recently, the prevailing medical opinion in neurology has been that damage to the central nervous system caused by injuries like birth trauma, accidents or stroke is often permanent. Currently, intervention after injury focuses on stabilizing the patient to minimize damage. However, data from animal research in recent years has challenged this assumption, leading to cord blood stem cell clinical research to study whether these cells may stimulate neural cell and tissue repair to restore function and alleviate neurological impairments.
CBR is taking the lead in moving animal research rapidly into the clinic to investigate the ability for cord blood stem cells to trigger the body's own mechanisms to initiate nerve repair by establishing specific clinical trials at leading medical institutions across the country. By pairing researchers with children who have been diagnosed with chronic conditions like cerebral palsy, traumatic brain injury or hearing loss-- and who also have access to their own cord blood stem cells -- CBR is helping physicians move beyond surgery and drugs to evaluate how newborn stem cells may help the body repair itself.
Celebrating a History of Firsts Throughout its history, CBR has taken many of the first steps to create and advance the notion of preserving and ensuring access to high quality newborn stem cells that are viable for use. Among the company's contributions to stem cell medicine and science, CBR was:
"CBR continuously improves our systems and technology to maintain the highest published cell recovery rate in the industry of 99%, every single time. We treat every sample as if it belongs to our own child or grandchild," says Tom Moore, CEO and founder of CBR. "That care and precision is what we offer clinical researchers, who are partnering exclusively with CBR to evaluate the use of a child's own cord blood stem cells to help treat chronic diseases like cerebral palsy, hearing loss and traumatic brain injury."
About Cord Blood RegistryCBR (Cord Blood Registry) is the world's largest and most experienced cord blood bank.The company has consistently led the industry in technical innovations and safeguards more than 425,000 cord blood collections for individuals and their families. CBR was the first family bank accredited by AABB and the company's quality standards have been recognized through ISO 9001:2008 certificationthe global business standard for quality. CBR has also released more client cord blood units for specific therapeutic use than any other family cord blood bank. Our research and development efforts are focused on helping the world's leading clinical researchers advance regenerative medical therapies.For more information, visit http://www.cordblood.com.
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Long-term pesticide exposure is harmful: STM study
Calcutta News.net Wednesday 20th June, 2012
Long-time exposure to pesticides via inhalation may cause moderate to severe blood toxicity and reduction in the total number of bone marrow cells, leading to several degenerative diseases like aplastic anaemia, researchers at the School of Tropical Medicine (STM) here say.
The researches arrived at the conclusion from procedures performed on mice.
"As a whole, exposure to pesticides reduced the total number of bone marrow cells or, in other words, suppressed them," Sujata Law, assistant professor (Stem Cell Biology) at STM's Department of Medical Biotechnology, told IANS.
Bone marrow is the soft, flexible tissue found in long bones such as the thigh bone and the hip bone that contain immature cells called stem cells.
Stem cells, particularly the haematopoeitic stem cells (HSC) or the blood-forming stem cells can develop into the following types - red blood cells that carry oxygen, white blood cells that fight infection and platelets that help to clot blood.
So, in effect, bone marrow is the birthplace of these important cells.
"Bone marrow suppression leads to a number of degenerative diseases like aplastic anaemia, where the deficiency in the number of cells in the circulating blood (peripheral cytopenia) is the main feature," Law said.
The exact underlying mechanism is unknown but it has been concluded from the research published in the Journal of Environmental Toxicology that the microenvironment of the stem cells, in which they develop, is somehow deranged and this prevents their development into the various types of cells.
"In order to prevent degenerative diseases related to pesticide exposure, it is of prime importance that those handling pesticides take precautions like wearing protective clothing, including masks and gloves," she said.
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Next Generation: The Heart Camera
A new camera system allows researchers to measure multiple cardiac signals at once to understand how they interact to control heart function.
THE DEVICE: A complex interplay of signals governs the hearts rhythm. Voltage changes and calcium flux are both important in controlling heart muscle function, with each signal influencing the others dynamics. Scientists at the University of Oxford have created a single camera system that can capture the dynamics of these signals simultaneously, yielding important insight into their relationship.
Peter Lee and colleagues combined several colors of light emitting diodes (LEDs) with a multi-band emission filter so that one very high speed camera could capture the different wavelengths of light emitted by various fluorescent dyes. By using different colors of LEDs, they were able to stimulate different dyes to measure changes in calcium and voltage across cardiac tissue or single layers of human cardiomyocytes (created from induced pluripotent stem cells).
WHATS NEW:The new setup took advantage of advances in lighting technology, explained Lee. While many older systems used xenon lamps, LEDs are cheap, cover the spectrum from infrared to ultraviolet, and reach peak intensity almost immediatelyallowing for ultra-rapid switching between excitation colors. Many previous systems also relied on a moving wheel to switch between colors, and thus measure different signals, explained Guy Salama, who researches cardiac arrhythmias at the University of Pittsburgh, but was not involved in the new cameras development. The wheels needed to move uniformly without wobbling, which would throw off its precision measurements, said Salama, and meant that each parameter had to be recorded for exactly the same amount of time. But Lees system, which uses electronics to control the length of time each LED shines, allows for different excitation times for each parameter of interestwhich is important as not all physiological changes happen on the same time scale, said Salama. Lees system has also jettisoned the need for moving parts, which can require careful alignment.
Single camera and LED system. Peter Lee
IMPORTANCE: Because calcium and voltage changes interact to control cardiac function, and perturbations in either leading to dysfunctions like arrhythmia, Lees camera system provides researchers with a tool to further investigate the interaction between the two signals, and thus gain a deeper understanding of cardiac function.
Using a single camera with multiple emission filters also allowed Lee and his collaborators to measure calcium properly, Lee explained. Many previous experiments used high-affinity calcium dyes, which bound strongly but could perturb the signal. The strong LEDs allowed for weaker-binding dyes, and ratiometric calcium measurement, meaning the dyes display shifts in emission wavelength upon binding calcium. Researchers can then quantify the concentration of calcium based on the light emissions they detect and calcium flux simultaneously.
Additionally, explained Lee, the simplicity of the system makes it more easily scalable. LEDs are cheap and perform well, and the lack of moving parts makes setup much easier than multi-camera systems that need careful calibration.
NEEDS IMPROVEMENT: As appealingly simple as a one-camera setup is, a single camera and multiple light sources can also introduce new hurdles, explained Salama. Because one camera is being used to capture multiple parameters, this cuts down on the number of image frames that can be devoted to each signal, noted Salama. For example, if a camera is running at 1,000 frames per second, but imaging four signals, only 250 of those frames would capture each parameter.
Salama also feared that lining up the LEDs and camera might result in the different light sources hitting the cardiac tissue at different angles, and bouncing off at different angles, making it difficult for the camera to capture them all. When visualizing the voltage and calcium propagations over a single layer of cells, scientists need to make sure the emissions theyre comparing are coming from the same locationso they arent trying to match voltage changes in one set of cells with calcium fluxes in another. When imaging microscopic-scale changes, Lee works around this problem by merging the lights into one path and using an optical fiber to direct all the colors to one site.
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Next Generation: The Heart Camera
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French scientists revive stem cells of dead people
French scientists revive stem cells of dead people
A group from the Pasteur Institute was able to reactivate muscle stem cells from deceased persons after 17 days, which functioned normally after transplant...
by Fabrice Chretien
French scientists were able to revive stem cells of muscle and bone marrow from persons who were already dead for 17 days, reports the journal Nature Communications in a paper released on Wednesday (13th) in France.
A team of researchers from the Pasteur Institute demonstrated that it is possible to reactivate the muscle stem cells from human cadavers and transplant them to make new ones born in perfect condition.
The scientists found that these cells did not die with the person. That's because they reduced their activity to a minimum and, after discarding the mitochondria (small bodies that help with breathing), were in a state of hibernation.
Thus, cells could survive even in an environment so hostile, without oxygen and in the middle of an acid bath, as well as in the case of a muscle injury, "sleeping and waiting out the storm," as Professor Fabrice Chrtien affirmed to the newspaper Libration.
"This reserve of stem cells could serve to make bone marrow transplants used to treat leukemia and blood diseases, among other conditions. They could also address the lack of donors," said Chretien, who led the study alongside researcher, Shahragim Tajbakhsh.
Despite the advances that have also been successfully tested in rats, the experiment showed an increase of one type of substance called ROS, which, in turn, has an incompatibility with the cells and genome, Professor Jean-Marc Lemaitre pointed out to the paper, Le Figaro. Due to this fact, the study still needs to determine whether these new cells, even in perfect condition, can hide still undetected malformations.
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French scientists revive stem cells of dead people
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New method generates cardiac muscle patches from stem cells
ScienceDaily (June 19, 2012) A cutting-edge method developed at the University of Michigan Center for Arrhythmia Research successfully uses stem cells to create heart cells capable of mimicking the heart's crucial squeezing action.
The cells displayed activity similar to most people's resting heart rate. At 60 beats per minute, the rhythmic electrical impulse transmission of the engineered cells in the U-M study is 10 times faster than in most other reported stem cell studies.
An image of the electrically stimulated cardiac cells is displayed on the cover of the current issue of Circulation Research, a publication of the American Heart Association.
For those suffering from common, but deadly heart diseases, stem cell biology represents a new medical frontier.
The U-M team of researchers is using stem cells in hopes of helping the 2.5 million people with an arrhythmia, an irregularity in the heart's electrical impulses that can impair the heart's ability to pump blood.
"To date, the majority of studies using induced pluripotent stem cell-derived cardiac muscle cells have focused on single cell functional analysis," says senior author Todd J. Herron, Ph.D., an assistant research professor in the Departments of Internal Medicine and Molecular & Integrative Physiology at the U-M.
"For potential stem cell-based cardiac regeneration therapies for heart disease, however, it is critical to develop multi-cellular tissue like constructs that beat as a single unit," says Herron.
Their objective, working with researchers at the University of Oxford, Imperial College and University of Wisconsin, included developing a bioengineering approach, using stem cells generated from skin biopsies, which can be used to create large numbers of cardiac muscle cells that can transmit uniform electrical impulses and function as a unit.
Furthermore, the team designed a fluorescent imaging platform using light emitting diode (LED) illumination to measure the electrical activity of the cells.
"Action potential and calcium wave impulse propogation trigger each normal heart beat, so it is imperative to record each parameter in bioengineered human cardiac patches," Herron says.
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Animal Stem Cell Therapy
BYRON, MN--It's a dream for many in the medical field, to use a person's own stem cells to help them heal. And it's a reality already happening in our area.
But it's not humans who are being treated. In this case, dogs are the ones being treated.
Animal Stem Cell Regenerative Therapy has been performed a few thousand times now across the U.S. Doctors harvest stem cells and re-enter them where the animal is having problems.
Both Marley and Vinnie have bad ligaments in their legs, and like many dogs suffering from arthritis, they are subject to monthly doses of expensive drugs.
That is until today.
Dr. Garren Kelly, D.V.M. at Meadow View Veterinary Clinic just outside Rochester says, "If you'd of asked me 5 years ago if I would be doing anything like this, I would have said no. But then as soon as I saw it i'm like 'Yeah that's for me'. I kind of like staying on the cutting edge of technology and surgeries".
The two are undergoing a first of its kind surgery in minnesota, using regenerative stem cells.
Blood is taken from the dogs, as well as fat tissue.
Then stem cells are separated out from the fat, activated with an led light, and injected back into the affected area. All in the same day.
MediVet America trainer Jordan Smith says, "It's a better quality of life, we're not promising to give them 10 years or 5 years but we are promising that the years that they do have remaining are a lot more enjoyable".
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Animal Stem Cell Therapy
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Pluristem Therapeutics' Cell Therapy Broadens Addressable Markets – Demonstrates Systemic Effectiveness of …
HAIFA, Israel, June 19, 2012 (GLOBE NEWSWIRE) -- Pluristem Therapeutics, Inc. (PSTI) (TASE:PLTR) announced today at the 2012 Bio International Convention the results of a pre clinical study it conducted measuring the effectiveness of its Placental eXpanded (PLX) cells when administered intramuscularly(IM). Cell therapies are traditionally delivered through intravenous (IV) injections for systemic effect. However, Pluristem's latest findings show that its PLX cells can be effective when injected by needle, into the muscle. Avoiding the use of an IV is simple and more cost-effective. This opens far larger markets for treatments in a wide range of potential outpatient settings and local clinics.
"The ability for IM injections of PLX cells has significant market implications that potentially broaden the indications and frequency with which our cell therapy can be used. We look forward to conducting additional testing of this very promising approach," said Zami Aberman, Chairman and CEO of Pluristem.
The study found that Intramuscularly administered PLX cells are safe, effective, easy to inject and provided systemic therapeutic benefits in a wide range of hematological disorders, as well as primary and secondary bone marrow failure, such as in radiation sickness and possibly for some complications from chemotherapy and radiotherapy.
The results of the study demonstrated a significant survival and recovery rate of bone marrow and peripheral blood counts in animals pre-irradiated by high lethal doses. These findings indicate that the IM route of administration of PLX cells stimulate the hematopoietic stem cells (HSCs) of the bone marrow to produce red and white blood cells as well as platelets crucial for the treatment of hematological disorders. The study was conducted in cooperation with the Sharett Institute of Oncology at Hadassah Hospital in Jerusalem.
"Pluristem is extremely pleased at how convincingly this study's data demonstrates that our PLX cells have the ability to stimulate the HSCs involved in rescuing bone marrow. With PLX cells, we may be able to reverse the traditional mindset that if you want to get a systemic effect, you need to inject the cells intravenously," said Liat Flaishon, MD. PhD. BD Director and the Head of the Radiation project at Pluristem.
"We had announced on May 9, 2012 the successful treatment of a pediatric patient whose bone marrow graft was rescued using our PLX cells. This data demonstrates the basis for the successful treatment. In the treatment conducted by Professor Reuven Or from the Bone Marrow Transplantation Unit at Hadassah, PLX cells were given to this patient intramuscularly as well," added Dr. Flaishon.
Prof. Raphael Gorodetsky, Head of the Laboratory of Biotechnology and Radiobiology in the Cancer Research Laboratories of Sharett Institute of Oncology at Hadassah Hospital, has been conducting the animal studies of Pluristem's PLX cells in the past several months. In these studies PLX cells and control medium were administered intramuscularly to C3H mice previously irradiated by a total body dose of 770cGy. The company previously reported initial results from these studies with respect to Acute Radiation Syndrome.
The key results of the Study include:
- After an initial sharp fall, a significant increase in the total number of bone marrow cells extracted from the major bones at 23 days was recorded: from~16million cells/mouse to ~32 million cells/mouse in the PLX treated (p<0.001). Non-irradiated animals had an average of 40 million cells.
- at 23 days a significant increase in the total number of red blood cells was recorded from 3.5 in the surviving controls to 6 million cells/microliter, in comparing the PLX (p<0.001). Non-irradiated animals had an average of 7 million cells/microliter.
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Tips from the Journals of the American Society for Microbiology
Public release date: 19-Jun-2012 [ | E-mail | Share ]
Contact: Jim Sliwa jsliwa@asmusa.org 202-942-9297 American Society for Microbiology
Human Insulin Suppresses Mosquito Immune System: Increasing Cases of Type II Diabetes Could Abet Malaria's Spread
Human insulin suppresses the mosquito immune system, according to a paper in the June Infection and Immunity. And while mosquitoes and malaria might seem to go together like baseball and hotdogs, mosquitoes' immunological resistance to the malaria parasite actually slows its spread among H. sapiens.
"A fair portion actually fight off the infection," says first author Nazzy Pakpour of the University of California, Davis.
But now the rate of type 2 diabetes is climbing in Africa as in most of the rest of the world, to the point where by 2030, one in five adults there are predicted to be so-afflicted. More diabetes means more hyperinsulinemiamore human insulin to inhibit mosquitoes' immune response to Plasmodium falciparum, thus aiding and abetting transmission of this dread disease.
As horrific as the medical consequences of all this might be, the science is intriguing. "It's crazy to think something in our blood could change how mosquitoes respond to parasites," says Pakpour.
In earlier work, Pakpour and collaborators showed that ingested human insulin activates the insulin/IGF-1 signaling pathway in Anopheles stephensi mosquitoes, making them more vulnerable to invasion by P. falciparum. The new study showed that insulin signaling reduced expression of certain mosquito immunity genes that are under the same regulatory control, and that human insulin suppressed mosquito immunity by activating the so-called PI3K signaling pathway, and that artificially inhibiting that pathway could reverse the immunosuppressive effects of human insulin.
(N. Pakpour, V. Corby-Harris, G.P. Green, H.M. Smithers, K.W. Cheung, M.A.Riehle, and S. Luckhart, 2012. Ingested human insulin inhibits the mosquito NF-KAPPAB-dependent immune response to Plasmodium falciparum. Infect. Immun. 80:2141-2149.)
Download the journal article at http://bit.ly/asm061912a
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Abnormal gene product associated with prostate cancer generated by unusual mechanism
Public release date: 19-Jun-2012 [ | E-mail | Share ]
Contact: Jeremy Moore jeremy.moore@aacr.org 215-446-7109 American Association for Cancer Research
PHILADELPHIA Researchers have identified a potential new pathway in prostate cancer cells by which cancer-driving gene products can be generated, according to a study published in Cancer Discovery, a journal of the American Association for Cancer Research.
"Our work shows that cancers have many more tricks than we thought to generate potential cancer-driving genes or gene products," said Hui Li, Ph.D., assistant professor of pathology at the University of Virginia in Charlottesville, and a recipient of an Innovative Research Grant from Stand Up To Cancer (SU2C). The AACR is the scientific partner of SU2C.
Gene fusion is a common characteristic of human cancers. In many cases, the protein products of these gene fusions, which are generated via an RNA intermediate, have a key role in the genesis of the cancer. A well-characterized example of this is the protein that drives chronic myeloid leukemia, BCR-ABL, which is generated via RNA intermediates from a fusion gene formed by chromosomal translocation an event involving exchange of genomic DNA between two distinct chromosomes.
"For many years, chromosomal translocation was considered the sole way in which single RNAs consisting of copies of parts of two genes, so-called fusion RNAs, could be generated," said Li. "We have shown that fusion RNAs can be generated without changes to DNA by a new mechanism that we are calling cis-SAGe [cis-splicing of adjacent genes]." Recently, a fusion RNA formed from parts of the SLC45A3 and ELK4 genes was identified in prostate cancer cells in the absence of any DNA alterations. Li and his colleagues confirmed in two prostate cancer cells lines that the SLC45A3-ELK4 fusion RNA could be detected even though there was no evidence of genomic DNA rearrangement.
Detailed molecular analysis of the prostate cancer cell lines indicated that the SLC45A3-ELK4 fusion RNA was generated by cis-SAGe. SLC45A3 and ELK4 are neighboring genes, and cis-SAGe occurred when an RNA that crossed the boundary between the two genes was formed.
The protein CCCTC-binding factor normally acts to insulate SLC45A3 and ELK4 from each other. Li and his colleagues found that levels of this protein at the gene boundary inversely correlated with the amount of SLC45A3-ELK4 fusion RNA generated, providing molecular insight into how the quantity of this fusion RNA could be regulated.
A functional role for the SLC45A3-ELK4 fusion RNA in prostate cancer was suggested by two observations. First, it promoted the growth of the two prostate cancer cell lines in culture. Second, its levels in human prostate samples correlated with prostate cancer disease progression normal prostate tissue expressed the lowest levels and prostate cancer specimens from men with metastatic disease expressed the highest levels.
"These data are not sufficient to say that the SLC45A3-ELK4 fusion RNA has a causal role in prostate cancer," said Li. "But they are highly suggestive, and I am very excited that this high-risk project, which I would not have been able to pursue without the grant from Stand Up To Cancer, has uncovered what seems to be a new way in which cancer can be driven."
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The Hospital for Sick Children in Toronto Adopts Life Technologies' Ion Protonâ„¢ Sequencer to Launch New Centre for …
CARLSBAD, Calif., June 19, 2012 /PRNewswire/ -- Life Technologies Corporation (LIFE) today announced it has partnered with The Hospital for Sick Children (SickKids) to advance pediatric genomic research on the Ion Proton Sequencer. Under the agreement, the semiconductor-based platform will be the primary instrument on which multiple clinical research samples will be mapped daily on four sequencers in the hospital's newly launched Centre for Genetic Medicine.
SickKids and Life Technologies will collaborate on developing sequencing workflows and protocols for the Ion Proton System that are tailored for studies of interest to researchers in the Centre. The first collaborative project will focus on sequencing clinical research samples to better understand the genetics behind autism, with a long-term goal to sequence up to 10,000 genomes per year to study various diseases in children.
"The perfect storm of unparalleled advances in genome sequencing technology and information science, and a captivated hospital striving for new ways to move forward in medical treatment, bring us to this important day," says the new Centre's Co-Director, Dr. Stephen Scherer, who also leads The Centre for Applied Genomics at SickKids and the University of Toronto's McLaughlin Centre. "We are very excited to work with Life Technologies to enhance our sequencing capabilities, such that 'genomic surveillance' may soon become the first line of investigation in all clinical research studies ongoing at our institution."
"Since the first published draft sequence of the human genome, our knowledge in genetics has exponentially increased," says Dr. Ronald Cohn, Co-Director of the SickKids Centre for Genetic Medicine. "With the help of this new technology, we will be able to further deepen our understanding of the genetic basis of human disease and translate this directly into daily clinical practice. We have finally reached a point, where individualized medicine is not just a theoretical concept, but will become an integral part of clinical care and management."
The Ion Proton Sequencer is designed to sequence an entire human genome in a day for $1,000. Unlike traditional next generation systems, it relies on semiconductor chips to map human exomes and genomes, making it much faster and less expensive to analyze DNA at unprecedented throughput levels and generate accurate sequencing data.
The Ion Proton Systemis based on the same proven technology as its predecessor, the Ion Personal Genome Machine (PGM), which is designed for sequencing small genomes or sets of genes. Combined with Life Technologies' AmpliSeq targeted sequencing technology, researchers can sequence panels of genes associated with disease on the PGM or exomes and genomes on the Ion Proton Sequencer in just a few hours.
"SickKids has a rich history of being at the forefront of pediatric medicine and we are pleased that its leaders have chosen the Ion Proton Sequencer as the Centre's primary technology to push the boundaries of genomics," said Mark Stevenson, President and Chief Operating Officer of Life Technologies. "Ion semiconductor technology's speed, simplicity and scalability are democratizing sequencing, and it will now be applied in disease research to benefit children."
The above mentioned technology is for research use only and not intended for human diagnostic or therapeutic use.
About Life Technologies Life Technologies Corporation (LIFE) is a global biotechnology company with customers in more than 160 countries using its innovative solutions to solve some of today's most difficult scientific challenges. Quality and innovation are accessible to every lab with its reliable and easy-to-use solutions spanning the biological spectrum with more than 50,000 products for translational research, molecular medicine and diagnostics, stem cell-based therapies, forensics, food safety and animal health. Its systems, reagents and consumables represent some of the most cited brands in scientific research including: Ion Torrent, Applied Biosystems, Invitrogen, GIBCO, Ambion, Molecular Probes, Novex, and TaqMan. Life Technologies employs approximately 10,400 people and upholds its ongoing commitment to innovation with more than 4,000 patents and exclusive licenses. LIFE had sales of $3.7 billion in 2011. Visit us at our website: http://www.lifetechnologies.com.
Life Technologies' Safe Harbor StatementThis press release includes forward-looking statements about our anticipated results that involve risks and uncertainties. Some of the information contained in this press release, including, but not limited to, statements as to industry trends and Life Technologies' plans, objectives, expectations and strategy for its business, contains forward-looking statements that are subject to risks and uncertainties that could cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Any statements that are not statements of historical fact are forward-looking statements. When used, the words "believe," "plan," "intend," "anticipate," "target," "estimate," "expect" and the like, and/or future tense or conditional constructions ("will," "may," "could," "should," etc.), or similar expressions, identify certain of these forward-looking statements. Important factors which could cause actual results to differ materially from those in the forward-looking statements are detailed in filings made byLife Technologies with the Securities and Exchange Commission.Life Technologies undertakes no obligation to update or revise any such forward-looking statements to reflect subsequent events or circumstances.
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Genetic testing to choose the right antidepressant
Psychiatrists often try two or more medications in a patient suffering with major depression before settling on the one that seems to work best for that individual. Sometimes, after several are tried and abandoned, two (or even three) are used in combination.
Medication selection is part of the art of psychiatry, but, now, testing is available that promises to make it more of a science. A company called AssureRx Health now offers what it calls GeneSightRxpharmacogenomic laboratory testing that helps identify which antidepressants are a good match for a persons genetic makeup, and which are not so good a match.
Sometimes, the testing reveals why three or four antidepressants havent worked for a patient, while pointing in the direction of one that might.
This is extremely good news, because psychiatrists have several different kinds of antidepressants to choose fromsome which increase the activity of the brain chemical messenger serotonin, some which increase the activity of the brain chemical messenger norepinephrine and some which increase both. And they do so by varying mechanisms, requiring the activity of different enzymes.
The technology behind GeneSightRx actually determines which genetic variantsin terms of the enzymes that are activated by antidepressantsa person possesses.
Different antidepressants affect the enzymes very differently. Hence, the testing can literally predict with some accuracy which antidepressants are likely to work in a particular person, and which are likely to cause the fewest side effects.
Recent studies have revealed that antidepressants dont work much better than placebo medications (sugar pills) for many patients. But those studies werent conducted by first selecting patients who are more likely (as determined by GeneSightRx) to respond to the particular medicine being studied.
Its very possible that patients given medications suggested by such testing would do far better than those given placebosbecause they arent being lumped together and given one medicine, regardless of their individual genetic makeup.
Moreover, since many patients discontinue their antidepressants due to side effects like sexual dysfunction and sleeplessness, choosing a medication that is metabolically and genetically less likely to cause these and other side effects makes good sense.
GeneSightRx also predicts which ADHD medications, antipsychotics and pain medications patients are likely to respond to and from which they are likely to experience fewer side effects.
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Genetic testing to choose the right antidepressant
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NewLink Updates Enrollment Information for Its Pivotal Phase 3 HyperAcute(R) Pancreas Immunotherapy (algenpantucel-L …
AMES, Iowa, June 19, 2012 (GLOBE NEWSWIRE) -- NewLink Genetics Corporation (NLNK) today announced that in the second quarter of 2012 it passed the midpoint in enrollment in its Phase 3 HyperAcute Pancreas immunotherapy product candidate (algenpantucel-L) trial as number of participating institutions reached to 74 sites around the US.
"Now that we have passed the midway point of trial enrollment, we are confident that the trial will be fully enrolled before the end of 2013," said Dr. Nick Vahanian, President and Chief Medical Officer of NewLink Genetics. He added, "We are very thankful for the support we have received from our patients and investigators and based on currently available data we anticipate reaching the trigger event for the first interim analysis in early 2013."
The current Phase 3 study is designed to evaluate the benefit of HyperAcute Pancreas in up to 722 patients who have undergone resection of pancreatic cancer. The clinical trial design was approved by the FDA under Special Protocol Assessment prior to opening in May 2010 and the HyperAcute Pancreas was granted Fast Track and Orphan Drug designation in the fourth quarter of 2010.
This Phase 3 trial was initiated based on encouraging results from the Company's Phase 2 study of HyperAcute Pancreas in patients who had undergone resection of pancreatic cancer. The Phase 2 results were recently updated and showed one, two and three year survival of 86% , 51% and 42% respectively, based on Kaplan-Meier analysis with a 33 months of median follow up. Demonstrating improvements of 37%, 59% and 121% over predicted one, two and three outcomes respectively based upon nomogram analysis of these same patients.
About algenpantucel-L
NewLink's algenpantucel-L immunotherapy product candidate consists of a group of two allogeneic pancreatic cancer tumor cell lines that were modified to express Alpha-Gal. These cell lines were chosen to provide a broad coverage of pancreatic cancer antigens. Each of the modified cell lines is grown in large cultures, harvested, irradiated and packaged. Approximately 150 million cells of each HyperAcute Pancreas cell line are given by intradermal injection with each treatment. A series of up to 14 treatments using both cell lines over a period of six months was used in NewLink's Phase 2 clinical trial. In NewLink's Phase 3 trial protocol, NewLink is adding an additional series of monthly, maintenance treatments, to be given during the next six months.
About algenpantucel-L clinical trials
About the Phase 2 Study
The multi-institutional, open-label, dose-finding, Phase 2 trial evaluated the use of algenpantucel-L in addition to chemotherapy with chemoradiotherapy in the adjuvant setting for resected pancreatic cancer. Adjuvant therapy was to start within seven weeks after surgery. The first cycle of treatment consisted of vaccination with either 100 million or 300 million cells per dose given intradermally on days 1 and 8. One week after the second vaccination, gemcitabine was administered at 1000mg/m2/week for three weeks, on days one, eight, and 15, in conjunction with HyperAcute Pancreas immunotherapy dosed on days 1 and 15 of cycle two. Chemoradiotherapy was initiated one to two weeks after the completion of cycle two. Continuous infusion 5-FU was administered at 250 mg/m2/day for the entire duration of radiation therapy. HyperAcute Pancreas immunotherapy was administered on days 1, 15, 29, and 43 of the chemoradiotherapy stage. A total of up to 14 vaccinations were dosed for patients who completed the entire study treatment.
About the Phase 3 Study
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NewLink Updates Enrollment Information for Its Pivotal Phase 3 HyperAcute(R) Pancreas Immunotherapy (algenpantucel-L ...
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bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patients own CD34+ hematopoietic stem cells transduced with bluebird bios lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.
Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families, said David Davidson, M.D., chief medical officer of bluebird bio. We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients.
Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.
About ALD
Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological disorder that, in its most severe form, causes damage to the myelin sheath (an insulating layer of membranes that surrounds nerve cells in the brain) and progressive dysfunction of the adrenal glands. Also known as Lorenzo's Oil disease, ALD is estimated to affect one in every 21,000 boys worldwide. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 4 and 10. Boys afflicted with this form of ALD develop normally until the onset of symptoms. The symptoms of this disorder often progress rapidly and, in a matter of years, can lead to a vegetative state and, ultimately, death. Current treatment options are limited to allogeneic stem cell transplantation when there is an appropriate donor. Allogeneic transplants carry a significant risk of serious morbidity and death.
About bluebird bio's CCALD Product Development
bluebird bios CCALD product program has the potential to halt the progression of CCALD by providing a functional ABCD1 gene to the patients own stem cells. These stem cells proliferate, and some of the progeny cells travel to the brain where they become microglial cells incorporating the corrective gene. Data from the first clinical study treating X-linked CCALD patients with the companys lentiviral gene therapy product demonstrated continued stable expression of the transgene and the corresponding ABCD-1 protein for over four years in two CCALD patients, resulting in prolonged disease stabilization. bluebird bio plans to initiate a Phase 2/3 clinical study in CCALD in both the United States and Europe in 2013.
About bluebird bio
bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bios product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The companys novel approach uses stem cells harvested from the patients own bone marrow into which a healthy version of the disease causing gene is inserted. bluebird bios approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and presenting a one-time potentially transformative therapy using a patients own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. Led by a world-class team, bluebird bio is privately held and backed by top-tier life sciences investors, including Third Rock Ventures, TVM Capital, ARCH Venture Partners, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its operations are located in Cambridge, Mass. and Paris, France. For more information, please visit http://www.bluebirdbio.com.
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bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy
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Cryopraxis, Sponsor of Stem Cell Research is Represented at Bio2012 in Boston
RIO DE JANEIRO--(BUSINESS WIRE)--
Cryopraxis established in 2001 as the pioneer private umbilical cord blood bank in Brazil will be present at Bio 2012 in Boston. Eduardo Cruz, chairman of the board, will be a speaker at the Brazilian break-out session speaking about The Brazilian Biotechnology Sector and showing the results of the company's commitment to R&D. Cryopraxis has already collected and processed more than 25000 cord blood units (CBU) and is actively involved in several R&D projects in Brazil and abroad.
A spin-off of Cryopraxis, Cellpraxis, has recently finished one of the world's first cell therapy project clinical trials in Brazil: ReACT. ReACT is a stem cell formulation. This regenerative medicine pioneer product aims on treating an orphan disease condition called refractory angina. Refractory angina patients suffer from untreatable severe chest pain and the results of the clinical trial in a 5 years follow up proved ReACT to positively interfere in the course of the pathology. Most of the individuals treated experienced relief in pain and better quality of life. ReACT will be presented at Bio2012 as an example of Brazil's dynamic biotechnology research.
Cryopraxis is accredited by the American Association of Blood Bank since 2009.
According to Tatiana Lima, Technical Director at Cryopraxis, "extensive training and strict adherence to good laboratory practices are basic principles in Cryopraxis' corporate strategy." Janaina Machado, cell lab director describes the company's primary mission: "maximizing safety and efficiency of collection procedures to make sure our clients get what they look for: the highest quality standards."
Cryopraxis is part of Axis Biotec (www.axisbiotec.com.br) and it has the largest biological cryogenic storage facility in Brazil and one of the largest in the World. It is the largest umbilical cord blood bank in Brazil. The company is involved in several research projects in Brazil and abroad.For more information, visitwww.cryopraxis.com.brand http://www.cellpraxis.com
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Cryopraxis, Sponsor of Stem Cell Research is Represented at Bio2012 in Boston
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Chicago woman cured of sickle cell disease
ScienceDaily (June 18, 2012) Chicagoan Ieshea Thomas is the first Midwest patient to receive a successful stem cell transplant to cure her sickle cell disease without chemotherapy in preparation for the transplant.
University of Illinois Hospital & Health Sciences System physicians performed the procedure using medication to suppress her immune system and one small dose of total body radiation right before the transplant.
The transplant technique is relatively uncommon and is a much more tolerable treatment for patients with aggressive sickle cell disease who often have underlying organ disease and other complications, says Dr. Damiano Rondelli, professor of medicine at UIC, who performed Thomas's transplant.
The procedure initially allows a patient's own bone marrow to coexist with that of the donor. Since the patient's bone marrow is not completely destroyed by chemotherapy or radiation prior to transplant, part of the immune defense survives, lessening the risk of infection. The goal is for the transplanted stem cells to gradually take over the bone marrow's role to produce red blood cells -- normal, healthy ones.
Thomas, 33, had her first sickle cell crisis when she was just 8 months old. Her disease became progressively worse as an adult, particularly after the birth of her daughter. She has spent most of her adult life in and out of hospitals with severe pain and has relied on repeated red blood cell transfusions. Her sickle cell disease also caused bone damage requiring two hip replacements.
"I just want to be at home with my daughter every day and every night," said Thomas, who depends on family to help care for her daughter during her frequent hospitalizations.
This type of stem cell transplant is only possible for patients who have a healthy sibling who is a compatible donor.
Thomas' sister was a match and agreed to donate blood stem cells through a process called leukapheresis. Several days prior to leukapheresis, Thomas' sister was given drugs to increase the number of stem cells released into the bloodstream. Her blood was then processed through a machine that collects white cells, including stem cells. The stem cells were frozen until the transplant.
Last Nov. 23, four bags of frozen stem cells were delivered to the hospital's blood and marrow transplant unit. One by one, the bags were thawed and hung on an IV pole for infusion into Thomas. The procedure took approximately one hour. Her 13-year-old daughter, Miayatha, was at her bedside.
Six months after the transplant, Thomas is cured of sickle cell disease and no longer requires blood transfusions.
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Next Generation: The Heart Camera
A new camera system allows researchers to measure multiple cardiac signals at once to understand how they interact to control heart function.
THE DEVICE: A complex interplay of signals governs the hearts rhythm. Voltage changes and calcium flux are both important in controlling heart muscle function, with each signal influencing the others dynamics. Scientists at the University of Oxford have created a single camera system that can capture the dynamics of these signals simultaneously, yielding important insight into their relationship.
Peter Lee and colleagues combined several colors of light emitting diodes (LEDs) with a multi-band emission filter so that one very high speed camera could capture the different wavelengths of light emitted by various fluorescent dyes. By using different colors of LEDs, they were able to stimulate different dyes to measure changes in calcium and voltage across cardiac tissue or single layers of human cardiomyocytes (created from induced pluripotent stem cells).
WHATS NEW:The new setup took advantage of advances in lighting technology, explained Lee. While many older systems used xenon lamps, LEDs are cheap, cover the spectrum from infrared to ultraviolet, and reach peak intensity almost immediatelyallowing for ultra-rapid switching between excitation colors. Many previous systems also relied on a moving wheel to switch between colors, and thus measure different signals, explained Guy Salama, who researches cardiac arrhythmias at the University of Pittsburgh, but was not involved in the new cameras development. The wheels needed to move uniformly without wobbling, which would throw off its precision measurements, said Salama, and meant that each parameter had to be recorded for exactly the same amount of time. But Lees system, which uses electronics to control the length of time each LED shines, allows for different excitation times for each parameter of interestwhich is important as not all physiological changes happen on the same time scale, said Salama. Lees system has also jettisoned the need for moving parts, which can require careful alignment.
Single camera and LED system. Peter Lee
IMPORTANCE: Because calcium and voltage changes interact to control cardiac function, and perturbations in either leading to dysfunctions like arrhythmia, Lees camera system provides researchers with a tool to further investigate the interaction between the two signals, and thus gain a deeper understanding of cardiac function.
Using a single camera with multiple emission filters also allowed Lee and his collaborators to measure calcium properly, Lee explained. Many previous experiments used high-affinity calcium dyes, which bound strongly but could perturb the signal. The strong LEDs allowed for weaker-binding dyes, and ratiometric calcium measurement, meaning the dyes display shifts in emission wavelength upon binding calcium. Researchers can then quantify the concentration of calcium based on the light emissions they detect and calcium flux simultaneously.
Additionally, explained Lee, the simplicity of the system makes it more easily scalable. LEDs are cheap and perform well, and the lack of moving parts makes setup much easier than multi-camera systems that need careful calibration.
NEEDS IMPROVEMENT: As appealingly simple as a one-camera setup is, a single camera and multiple light sources can also introduce new hurdles, explained Salama. Because one camera is being used to capture multiple parameters, this cuts down on the number of image frames that can be devoted to each signal, noted Salama. For example, if a camera is running at 1,000 frames per second, but imaging four signals, only 250 of those frames would capture each parameter.
Salama also feared that lining up the LEDs and camera might result in the different light sources hitting the cardiac tissue at different angles, and bouncing off at different angles, making it difficult for the camera to capture them all. When visualizing the voltage and calcium propagations over a single layer of cells, scientists need to make sure the emissions theyre comparing are coming from the same locationso they arent trying to match voltage changes in one set of cells with calcium fluxes in another. When imaging microscopic-scale changes, Lee works around this problem by merging the lights into one path and using an optical fiber to direct all the colors to one site.
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Next Generation: The Heart Camera
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Labceutics Launches "Labceutics Connect" Platform
LONDON--(Marketwire - Jun 19, 2012) - Labceutics (www.labceutics.com), the leading personalized medicine laboratory network in the EU, has launched the Labceutics Connect platform. Labceutics Connect provides its network members with a secure portal to share information, promote training and best practices, and discuss personalized medicine partnering opportunities. The platform will also provide members with the ability to introduce new and innovative personalized medicine tests and technologies within the network.
Labceutics has partnered with more than 120 leading EU laboratories to provide pharmaceutical and diagnostic companies with a one-stop personalized medicine laboratory partner. The network's focus is on the development and implementation of companion laboratory testing and related services that enable a high return on investment for personalized therapies.
"Being part of a personalized medicine network and having the ability to use a tool like Labceutics Connect enables our biocluster to be on the cutting edge of personalized medicine and to interact more effectively with our peers across the EU," said Jesus Mara Izco PhD, of CIBER-BBN (Spain). "We are seeing new tests and technology coming to the market every day and we want to ensure the services we provide our community are of the highest quality."
Given the innovation and evolution of personalized medicine on a global level, Labceutics is also partnering with the key stakeholders in the industry including pharmaceutical companies, diagnostic companies, personalized medicine-focused associations, biobanks, biotechnology companies and other healthcare organizations.
Labceutics will have a booth at the ESMO Congress (www.esmo.org), which takes place in Vienna (Austria) between 28 September and 2 October 2012. There will be on-site Labceutics Connect demos, information about the benefits of joining and leveraging the Labceutics network, and details on the launch of the Labceutics network in Asia.
To obtain more information about the connectivity platform or to meet us at the congress, please contact Jose Luis Ferreiro at: joseluis.ferreiro@labceutics.com.
About Labceutics Labceutics (www.labceutics.com), a division of Diaceutics, supports the integration of pharmaceutical and diagnostic companies through its personalized medicine laboratory network that offers early testing access, quality standards, education and real-time communication across stakeholders to help physicians diagnose, prescribe and monitor care effectively.
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Labceutics Launches "Labceutics Connect" Platform
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Sixteenth Patient Dosed In Neuralstem ALS Stem Cell Trial
ROCKVILLE, Md., June 19, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the first patient to receive stem cell transplantation in both regions of the spinal cord has been treated in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). This is also the 16th patient to be treated in the trial altogether and the first patient returning to the trial for a second treatment. In this treatment, the patient received five injections in the cervical (upper back) region of the spinal cord, in addition to the ten he received previously in the lumbar (lower back) region of the spine, for a total of 15 injections. This is the highest number of injections in the trial so far. Patient 16 is also the first patient in the world to receive stem cell transplants in both the lumbar and cervical regions of the spinal cord in an FDA-approved trial. Two additional previously-treated patients are expected to return to the trial this summer in this cohort, provided they continue to meet the inclusion requirements. The trial is taking place at Emory University Hospital in Atlanta, Georgia.
(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )
"Transplanting the first of the returning patients represents a major milestone in the trial," said Dr. Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "The ability to safely administer multiple dosings to these patients is a key enabling step in administering the maximum safe dose. Not only are we dosing patients for a second time in this cohort, we are now dosing in both the lumbar and cervical regions of the spinal cord for the first time, where the stem cell therapy could support both walking and breathing."
About the Trial
The Phase I trial to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in ALS patients has been underway since January 2010. The trial is designed to enroll up to 18 patients. The first 12 patients were each transplanted in the lumbar (lower back) region of the spine, beginning with non-ambulatory and advancing to ambulatory cohorts.
The trial then advanced to transplantation in the cervical (upper back) region of the spine. The first cohort of three was treated in the cervical region only. The current cohort of three will receive injections in both the cervical and lumbar regions of the spinal cord. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously-treated patients to this cohort. The first of these returning patients was just treated. The entire 18-patient trial concludes six months after the final surgery.
About Neuralstem
Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem is in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.
In addition to ALS, the company is also targeting major central nervous system conditions with its cell therapy platform, including spinal cord injury, ischemic spastic paraplegia and chronic stroke. The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in chronic spinal cord injury.
Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company has received approval from the FDA to conduct a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include CTE (chronic traumatic encephalopathy), Alzheimer's disease, anxiety, and memory disorders.
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Sixteenth Patient Dosed In Neuralstem ALS Stem Cell Trial
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Bethel woman waits for marrow match
BETHEL, Vt. -
Amelia Lincoln loves to garden. But so far this planting season, she has had to sit it out.
"It's been a long haul and we try to keep a pretty positive attitude about everything. So, I generally could feel worse right now," she said.
Lincoln's immune system is fragile. For the past six weeks, she has been undergoing chemotherapy for cancer.
"I have acute myelogenous leukemia," she said. "I have a leukemia that came back after a stem cell transplant two years ago."
She had been in remission, but the aggressive cancer in her bone marrow is back.
"It's a change of priorities, but what would anyone say if their spouse was sick," husband James Patterson said.
Lincoln needs another transplant at the Norris Cotton Cancer Center. But right now, she has yet to find the perfect match.
"We used to use bone marrow specifically for a bone marrow transplant. Nowadays, we can use medicines to stimulate a patient's bone marrow cells into the blood. We can collect those bone marrow cells in the blood-- called peripheral blood stem cells-- and use those cells for the transplant," said Dr. Kenneth Meehan of the Norris Cotton Cancer Center.
A donor drive Tuesday in Randolph Center could increase Amelia's odds. No needles-- just a swab.
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Bethel woman waits for marrow match
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Stem Cell Therapy for CMT-Gary B-part 2.mp4 – Video
17-06-2012 23:59 Gary B Stem Cell Therapy for CMT Part 2 - For more info. visit
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Stem Cell Therapy for CMT-Gary B-part 2.mp4 - Video
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Full Spectrum Genetics Enters Strategic Collaboration With a Leading Global Pharmaceutical Company
SOUTH SAN FRANCISCO, CA--(Marketwire -06/19/12)- Full Spectrum Genetics, Inc., a privately-held protein analysis and engineering platform and product company, today announced a strategic collaboration with an undisclosed leading global pharmaceutical company for the purpose of generating multiple novel therapeutic protein and antibody product candidates.
Under the terms of the agreement, Full Spectrum Genetics will be responsible for engineering and analyzing variants of specified molecules using its MapEng platform. The pharmaceutical company will receive worldwide rights to develop and commercialize product candidates arising from the collaboration. Full Spectrum Genetics will receive an upfront payment to initiate the collaboration and is eligible for additional financial consideration. Additional terms were not disclosed.
About Full Spectrum Genetics
Founded in 2010, Full Spectrum Genetics, Inc. is a privately-held protein analysis and engineering platform and product company. The Company's MapEng platform enables the ultra-high throughput quantification of the effect on binding of every possible single amino acid substitution within a protein binding site. The MapEng platform provides a comprehensive analysis of protein structure-function relationships, with multiple applications for generating better biotherapeutics and diagnostics. For more information on Full Spectrum Genetics and its MapEng platform, visit http://www.fsgene.com.
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Full Spectrum Genetics Enters Strategic Collaboration With a Leading Global Pharmaceutical Company
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Bethel woman waits for marrow match
BETHEL, Vt. -
Amelia Lincoln loves to garden. But so far this planting season, she has had to sit it out.
"It's been a long haul and we try to keep a pretty positive attitude about everything. So, I generally could feel worse right now," she said.
Lincoln's immune system is fragile. For the past six weeks, she has been undergoing chemotherapy for cancer.
"I have acute myelogenous leukemia," she said. "I have a leukemia that came back after a stem cell transplant two years ago."
She had been in remission, but the aggressive cancer in her bone marrow is back.
"It's a change of priorities, but what would anyone say if their spouse was sick," husband James Patterson said.
Lincoln needs another transplant at the Norris Cotton Cancer Center. But right now, she has yet to find the perfect match.
"We used to use bone marrow specifically for a bone marrow transplant. Nowadays, we can use medicines to stimulate a patient's bone marrow cells into the blood. We can collect those bone marrow cells in the blood-- called peripheral blood stem cells-- and use those cells for the transplant," said Dr. Kenneth Meehan of the Norris Cotton Cancer Center.
A donor drive Tuesday in Randolph Center could increase Amelia's odds. No needles-- just a swab.
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Bethel woman waits for marrow match
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The WIN 4th SYMPOSIUM, on June 28-29 in Paris, Scientific Program Recognized by Leading Oncology Society
PARIS--(BUSINESS WIRE)--
The scientific program of the 4th WIN Symposium, on Cancer personalized medicine and efficacy of biomarkers, a major event where hundreds of representatives from 20 countries will gather on next June 28 and 29, 2012 in Paris (France), has been endorsed by the American Society of Clinical Oncology (ASCO), the worlds leading professional organization representing physicians who care for people with cancer.
John Mendelsohn, Chairman of WIN Consortium and Director of the Institute for personalized therapy, UT M. D. Anderson Cancer Center, says: we have been proud to receive the endorsement of ASCO for the quality of educational content of the WIN Symposium Program 2012. This is a great achievement and recognition of the scientific value of the WIN Program Symposium and the major impact this symposium may bring.
The WIN Consortium meeting reflects the vision and goals of ASCOs blueprint for transforming cancer research (www.ASCO.org/Blueprint) and the potential that molecularly-driven therapies have to transform clinical research, said Sandra Swain, MD, President of ASCO. ASCO is pleased to endorse this meeting because of the opportunities it provides for international collaboration.
Organized by the WIN Consortium, this congress gathers the main opinion leaders in cancer care and drug development for two days of debates and exchanges and offers an excellent opportunity to advance research in Cancer and biomarkers at international level, facilitating the translation of scientific results into clinical practice. Advanced and innovative concepts to increase efficacy of personalized cancer therapeutics and molecular diagnostics will be disclosed during WIN Symposium: launch of WINTHER, the most advanced study in personalized medicine selected by the European Community EU FP7.
About WIN Consortium: The WIN Consortium is an initiative of the Cancer Institute Gustave Roussy, Villejuif, IGR (France) and University of Texas MD Anderson Cancer Center (USA), which brings together 22 international cancer centers, three technology partners (Agilent Technologies, GE Healthcare and LifeTechnologies) and associations such as Sage Bionetworks and the National Breast Cancer Foundation as part of a legal entity operating non-profit. Countries participating in the symposium: U.S., France, Hungary, Romania, Singapore, Brazil, Spain, Italy, India, China, Israel, Jordan, UK, Canada... The american Foundation Medicine is an official member of the WIN Consortium. The quality of the WIN 2012 Symposium program has been also recognized by several major international organizations: ESMO, INCa and UICC.
ASCO is a registered trademark of the American Society of Clinical Oncology. Used with permission. This is not an ASCO sponsored event.
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The WIN 4th SYMPOSIUM, on June 28-29 in Paris, Scientific Program Recognized by Leading Oncology Society
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