Archive for May, 2014

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Newswise LA JOLLARonald M. Evans, director of the Gene Expression Laboratory at Salk and Howard Hughes Medical Institute investigator, is one of three scientists chosen to receive $5 million in research funding as part of The Lustgarten Foundation's new "Distinguished Scholars" program, which recognizes individuals who have made outstanding achievements in research to focus their efforts on finding a cure for pancreatic cancer.

The Lustgarten Foundation, the nation's largest private funder of pancreatic cancer research, established the new Distinguished Scholars initiative to identify and fund the best minds in research today to engage in pancreatic cancer research. The three scientists will each receive $5 million in research funding over the next five years. The grantees were selected by The Lustgarten Foundation's Scientific Advisory Board due to their historical accomplishments of breakthrough research.

"I am deeply honored by The Lustgarten Foundation's support and belief that this research will pave the road to a cure," says Evans. "We are excited to tackle the challenge and know that this funding will help us pioneer new advances toward understanding and treating this devastating disease."

"The Foundation's Scientific Advisory Board has selected these outstanding scientists because each one is a leader in their field with the greatest potential for developing an early detection test and more effective therapies for the nation's most lethal cancer," says Kerri Kaplan, executive director of The Lustgarten Foundation. "Together, we will pursue our mutual goals of improving survival rates for people with pancreatic cancer and eradicating this deadly disease."

Douglas Fearon, of Cold Spring Harbor Laboratory and Weill Cornell Medical College, and Bert Vogelstein, of Johns Hopkins Kimmel Cancer Center, will also both receive funding as part of the new program.

Evans, who is also the holder of the March of Dimes Chair in Molecular and Developmental Biology, focuses on hormones and how they communicate signals within the body. Several of the hormone signals Evans discovered are primary targets in the treatment of breast cancer, prostate cancer, pancreatic cancer and leukemia, as well as osteoporosis and asthma. Most recently he has been studying the use of Vitamin D in the treatment of pancreatic cancer in the laboratory. As a Lustgarten Foundation Distinguished Scholar, he will expand these studies to conduct clinical trials in pancreatic cancer patients using Vitamin D therapies.

About The Lustgarten Foundation and Pancreatic Cancer: Pancreatic cancer is swift and silent, often undetected until it's too late. More than 39,000 people will die from it this year. The overall five-year survival rate for pancreatic cancer is six percent and most with advanced cancer die within a year. There are no early detection tests, no effective long-term treatments and, unless the cancer is surgically removed in its earliest stages, no cure. It is the fourth-leading cause of cancer deaths in the United States.

The Lustgarten Foundation is America's largest private foundation dedicated to funding pancreatic cancer research. Based in Bethpage, New York, the Foundation supports focused research to find a cure for pancreatic cancer, facilitates dialogue within the medical and scientific community, and educates the public about the disease through awareness campaigns and fundraising events. The Foundation has provided millions of research dollars and assembled the best scientific minds with the hope that one day, a cure can be found. Cablevision Systems Corporation, a leading media and telecommunications company, underwrites The Lustgarten Foundation's administrative costs, so 100 percent of every dollar donated to the Foundation goes directly to pancreatic cancer research. Additional information about The Lustgarten Foundation is available at http://www.lustgarten.org.

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Salk Professor Named Grantee in New Pancreatic Cancer Research Program

PUBLIC RELEASE DATE:

29-May-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 29, 2014Women have accounted for half the students in U.S. medical schools for nearly two decades, but as professors, deans, and department chairs in medical schools their numbers still lag far behind those of men. Why long-held gender stereotypes are keeping women from achieving career advancement in academic medicine and what can be done to change the institutional culture are explored in an article in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website.

In "Stuck in the Out-Group: Jennifer Can't Grow Up, Jane's Invisible, and Janet's Over the Hill," Anna Kaatz, PhD, MPH and Molly Carnes, MD, MS, University of Wisconsin-Madison, present examples of three women at different stages of their careers to illustrate the ways in which gender stereotypes can influence people's judgment and negatively affect women in social interactions, causing them to be in the out-group and lose out on opportunities for professional advancement.

"Challenging cultural stereotypes about women and men is a critical step toward achieving gender equity in academic medicine," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website. Journal of Women's Health is the official journal of the Academy of Women's Health and the Society for Women's Health Research.

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Gender stereotypes keep women in the out-group

WEDNESDAY, May 28, 2014 (HealthDay News) -- A team of international researchers has identified nearly 85 percent of proteins in the human body.

Proteins are the substances that provide structure, function and regulation of the body's tissues and organs. Human genes contain instructions (encoding) that direct the production of proteins, according to the U.S. National Institutes of Health.

In addition to finding the majority of the body's proteins, the researchers also identified 193 new proteins on the human genome. The proteins were found in areas of DNA that were believed to be "noncoding," or regions that do not encode proteins.

Finding proteins in areas with genes that weren't believed to code means the human genome could be more complex than previously believed, the researchers concluded.

"This was the most exciting part of this study, finding further complexities in the genome. The fact that 193 of the proteins came from DNA sequences predicted to be noncoding means that we don't fully understand how cells read DNA, because clearly those sequences do code for proteins," Dr. Akhilesh Pandey, a professor at the McKusick-Nathans Institute of Genetic Medicine and of biological chemistry, pathology and oncology at Johns Hopkins University in Baltimore, said in a news release.

More than 10 years ago, researchers identified all of the nearly 25,000 genes in human DNA. Known as the Human Genome Project, the research provided scientists with genetic information that helped them figure out how changes in certain genes could trigger some diseases.

The current researchers set out to create an initial catalog of all the proteins in the human body, or the human "proteome." The team identified proteins originating from more than 17,000 genes, which is about 84 percent of all of the genes in the human genome predicted to encode proteins.

Cataloging human proteins and where they can be found in the body may provide scientists even more insight than a catalog of all the genes in the human genome, the researchers pointed out. They explained that the characteristics of an organism depend on its genes. These genes, however, provide directions for making proteins, which are the building blocks of all cells in the body.

"You can think of the human body as a huge library where each protein is a book," explained Pandey, who is also the founder and director of the Institute of Bioinformatics in Bangalore, India. "The difficulty is that we don't have a comprehensive catalog that gives us the titles of the available books and where to find them. We think we now have a good first draft of that comprehensive catalog."

In attempting to catalog all of the proteins in the body, the team of researchers from Johns Hopkins and the Institute of Bioinformatics conducted a broad examination of the proteins in the human body from 30 tissue samples.

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Researchers Identify New Genetic Building Blocks

Genetic testing can predict illnesses, but many don't want to know

Luke Hilger has been anticipating his 18th birthday for years, but not just for the usual reasons. Hilger has known since he was 12 years old that Huntingtons Disease runs in his family. Hes watched his mother steadily decline during the past five years, in the grip of what many in medicine believe to be among the bodys cruelest illnesses.

Huntingtons usually strikes people in their 40s. It causes nerve cells in the brain to break down. Sufferers lose control of their muscles and begin to twitch uncontrollably. Then they lose their ability to think. Eventually they develop depression and dementia. There is no cure.

There is a genetic test that will tell Hilger if he is destined to get Huntingtons. For the past five years, anxious about the possibility that he would suffer the same fate as his mother, Hilger was certain he wanted to take the test. But physicians, citing ethical considerations, told him he would have to wait until he was 18.

Hilger, who lives with his parents outside Salem, turns 18 in four months. He has attended national conferences for families of Huntingtons victims. He has looked for physical signs that he might be among those who get an early onset version of Huntingtons. And he has made a choice. He no longer wants to know his fate.

The idea was, I had to know, Hilger says. I had to know because its going to help me sleep at night.

But now, Hilger says, hes figured out that for him, the stress of not knowing is more bearable than if he takes the test and discovers he will suffer as he has seen his mother suffer.

Three weeks ago, scientists announced a promising new blood test that experts say within 10 years should be available to predict who is going to get dementia-causing Alzheimers disease. Physicians can use a brain scan to detect amyloid plaque buildup that has been associated with Alzheimers. Though the amyloid test is far from refined as a predictive tool, neurologists such as Eran Klein at Oregon Health & Science University say they are getting more inquiries from patients who want the test. Patients want to know if they are going to get Alzheimers, even though the disease has no cure and there is little therapy to ease its brain-decaying symptoms.

Hilger and Kleins fate-seeking patients are not medical oddities. They are, scientists and bioethicists say, a first wave that eventually could grow into a tsunami. As genetic testing and brain scanning become more widely used and better understood, most of us will have opportunities to know ahead of time what diseases we are likely to contract. The question is, will we want to? Only about one in five people with family histories of Huntingtons choose to take the genetic test that preoccupies Hilger.

Theres this technological paradigm that more information is always better, Klein says. But sometimes more information just complicates.

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Is there a disease in your future?

TED

Stephen Friend spoke at TED 2014 in Vancouver.

In many cases, genetic factors can explain why some people get sick, or why people are predisposed to an illness. But most of the time, knowing about a genetic predisposition for certain diseases hasn't shown us how to prevent or cure that illness.

So maybe looking at sick people is the wrong approach.

Instead, we need to find the people who are genetically predisposed to these diseases but don't get sick, say biochemist Stephen Friend, president of Sage Bionetworks, and Eric Schadt, director of the Icahn Institute for Genomics at Mount Sinai School of Medicine.

Friend and Schadt are the principle investigators for the Resilience Project, an initiative that's trying to study those rare people who have the same genetic factors that normally cause disease but who are somehow protected by either genetic mutations or environmental factors.

In a TED 2014 talkreleased online today, Friend calls these people "unexpected heroes" most people don't know they have these hidden protective traits that could perhaps help others.

It turns out, he explains, that there are precedents for finding people like this and creating therapies based on the factors that make them unique.

In 1980s and 1990s, doctors realized that a very small number of people with high levels of HIV never developed AIDS, he explains. They had certain genetic mutations that prevented them from getting sick. Now, treatments for AIDS are being developed based on those mutations.

Along the same lines, most individuals who have high lipid levels, meaning fatty acids and cholesterol, develop heart disease. But there are some who don't. This can sometimes be explained by genetic mutations or protective environmental factors. Once these are better understood, they may provide new strategies for fighting heart disease.

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How Healthy People Who Should Be Sick Could Revolutionize Medicine

Genetic Researching: Gene Therapy
Bio Video.

By: Ali Banach

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Genetic Researching: Gene Therapy - Video

PUBLIC RELEASE DATE:

28-May-2014

Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Tampa, Fla. (May 28, 2014) To be suitable for medical transplantation, one idea is that human embryonic stem cells (hESCs) need to remain "undifferentiated" i.e. they are not changing into other cell types. In determining the best way to culture hESCs so that they remain undifferentiated and also grow, proliferate and survive, researchers have used blood cell "feeder-layer" cultures using animal-derived feeder cells, often from mice (mouse embryonic fibroblasts [MEFs]). This approach has, however, been associated with a variety of contamination problems, including pathogen and viral transmission.

To avoid contamination problems, a Brazilian research team has investigated the use of human menstrual blood-derived mesenchymal cells (MBMCs) as feeder layers and found that "MBMCs can replace animal-derived feeder systems in human embryonic stem cell culture systems and support their growth in an undifferentiated stage."

The study will be published in a future issue of Cell Medicine, but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/cm/pre-prints/content-CM1019silvadosSantos.

"Human embryonic stem cells present a continuous proliferation in an undifferentiated state, resulting in an unlimited amount of cells with the potential to differentiate toward any type of cell in the human body," said study corresponding author Dr. Regina Coeli dos Santos Goldenberg of the Instituto de Biofisica Carlos Chagas Filho, Universidade Federal do Rio de Janeiro. "These characteristics make hESCs good candidates for cell based therapies."

Feeder-layers for hESCs comprised of MEFs have been efficiently used for decades but, because of the clinical drawbacks, the authors subsequently experimented with human menstrual blood cells as a potential replacement for animal-derived feeder-layers, not only for negating the contamination issues, but also because human menstrual blood is so accessible. MBMCs are without ethical encumbrances and shortages, nor are they difficult to access - a problem with other human cells, such as umbilical cord blood cells, adult bone marrow cells or placenta cells.

"Menstrual blood is derived from uterine tissues," explained the researchers. "These cells are widely available 12 times a year from women of child-bearing age. The cells are easily obtained, possess the capability of long-term proliferation and are clinically compatible with hESCs-derived cells."

The researchers found that their culture system using MBMCs as a feeder-layer for hESCs are the "closest and more suitable alternative to animal-free conditions for growing hESCs" and a "good candidate for large-expansion of cells for clinical application." They also found no difference in growth factor expression when comparing the use of growth factors in both the standard feeder system using animal cells and the feeder system they tested using hESCs.

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Brazilian researchers find human menstrual blood-derived cells 'feed' embryonic stem cells

Discredited stem-cell treatment loses in Strasbourg

(ANSA) - Strasbourg, May 28 - The European Court of Human Rights on Wednesday ruled that an Italian ban on a controversial stem-cell therapy was legitimate. The case centered around a woman suffering from a degenerative brain disease since birth who argued her rights had been violated by the State denying her Stamina treatment. The process involves extracting bone-marrow stem cells from a patient, turning them into neurons by exposing them to retinoic acid for two hours, and injecting them back into the patient. But its credibility has long been suspect, and last fall the health ministry ruled that the Stamina Foundation would no longer be allowed to test the treatment on humans. The foundation was also stripped of its non-profit status after a study found its treatment was "ignorant of stem-cell biology". Recent investigations have shown risks of the treatment range from nausea to cancer, and as many as one quarter of all patients treated have experienced "adverse effects". The head of the foundation, Davide Vannoni, may face indictment.

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European rights court says Stamina ban legit

Arany PR et al.

This image shows the structure of the tooth cells as they begin the regeneration process.

Using lasers to regenerate and grow body parts sounds like science fiction, but researchers have just demonstrated that it might be a tranformative tool in medicine or at least dentistry in the future.

A Harvard-led team just successfully used low-powered lasers to activate stem cells and stimulate the growth of teeth in rats and human dental tissue in a lab. The results were published today in the journal Science Translational Medicine.

Stem cells exist throughout the body, and they fascinate scientists because they have the ability to become different types of cells which means they have the potential to repair or replace damaged or worn out tissue. Figuring out new ways to make them useful has long been a goal of medical researchers.

Using lasers to make stem cells do their work is particularly appealing, since it's a minimally invasive technique, only requiring light once the damaged area is exposed. Scientists have theorized in the past that this was possible, since lasers have been shown to stimulate growth for unknown reasons, but this is the first time that the process has been demonstrated and observed.

The ability to naturally regrow dental tissue could transform dentistry, making it possible to regrow teeth instead of replacing them with a substitute like porcelain. But even more amazingly, once it's better understood, this same technique could potentially be used to heal wounds and regenerate bone, skin, and muscle.

The research is in its earliest stages and has not yet been tested on humans, so it's far too soon to say whether these futuristic techniques will ever make it to your local hospital. The treatment possibilities raised by these experiments, however, are exciting to contemplate.

Arany PR et al.

This is the exposed rat molar that received the laser treatment, causing it to start to grow back.

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Scientists Can Regrow Teeth With Lasers

SINGAPORE: Australia-based stem cell therapy firm Mesoblast has announced plans to accelerate commercial manufacturing operations in Singapore.

This is to prepare for new product launches in the United States and other major markets over the next couple of years.

Its existing operations in Singapore include making stem cell products for clinical trials under its contract with its partner, pharmaceutical company Lonza.

One of its key products still awaiting full approval is Prochymal, which Mesoblast says can help to more than double the survival rate of patients suffering from complications after receiving tissue transplants from donors -- known as graft versus host disease.

The global stem cell market is expected to grow at an average annual rate of 12 per cent between 2011 and 2016 to reach more than S$8 billion by 2016.

Mesoblast said commercial manufacturing requires a much larger capacity and operations must be scaled-up to meet regulatory demands.

Silviu Itescu, chief executive at Mesoblast, said: "We are now in a phase of making more investments in order to get our processes to commercial scale. That anticipates successful commercial launches.

"If we're successful in that over the next 18-24 months, then we're going to leverage the investment in our commercial facilities to be able to build up and prepare for launching of much larger opportunities in cardiovascular medicine, orthopaedics and diseases of immunity and inflammation which would require purpose-built facilities."

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Mesoblast to accelerate operations in S'pore

Washington, DC (PRWEB) May 28, 2014

Catherine M. Bollard, MBChB, MD, director of Childrens National Health Systems Program for Cell Enhancement and Technologies for Immunotherapy (CETI), and her team have performed the hospitals first treatment using T-cell therapy for a 6-month-old patient with congenital immune deficiency and a life-threatening virus infection.

Not only does this therapy offer a potentially curative treatment for patients who have failed conventional therapies for infections and cancer, the procedure sets the stage for avoiding potentially toxic drugs which can ultimately reduce inpatient stays and medical costs.

Its extremely important, offering a novel therapeutic thats not available at the majority of hospitals worldwide, said Dr. Bollard, a member of the Division of the Blood and Marrow Transplantation and senior scientist at Childrens Nationals Center for Cancer and Immunology Research at Childrens Research Institute. She is also the Principal Investigator and the Sheik Zayed Institute for Pediatric Surgical Innovation.

Childrens National is one of the few hospitals in the world to offer cellular therapy to treat life-threatening infections in patients with immune deficiencies as well as preventing or treating relapse in children with cancer. Cellular therapy uses the bodys own immune system to fight cancer and/or infections.

Patients from other hospitals and childrens facilities have been referred to Childrens National because of the uniqueness of the cell therapies we can now offer here, Dr. Bollard said. This kind of procedure reduces the amount of time for care and is not only cost effective for a hospital but also more tolerable for the patient, said Dr. Bollard. None of this could have been achieved without every one of those members within the CETI Program pulling together as a team to make it happen.

In the first of its kind cellular therapy achievement at Childrens National, Dr. Bollard and her team have shown that in the laboratory they can train nave or inexperienced immune system cells (T-cells) to kill cancer and/or viruses. In the first patient treated here, T-cells were grown from the patients mother and then injected into the young patient, who had severe combined immunodeficiency and a potentially life threatening virus infection. The T-cells the patient received (cytotoxic T lymphocytes) are a type of white blood cell that can kill virus-infected cells or cancer cells infected or cells that are damaged in other ways.

The babys immunodeficiency ailments included SCID, or severe combined immunodeficiency, a primary immune deficiency, which can result in the onset of one or more serious infections within the first months of life. Early in life, the child was infected with cytomegalovirus (CMV), a latent virus related to herpes that has significant morbidity and high mortality rates in immune compromised people. Initially, the patient had received a bone marrow transplant, but the CMV could not be cleared with the drug therapy he received after transplant, Dr. Bollard said.

Conventional treatment using antiviral agents is expensive and toxic and can be ineffective. Transfer of virus-specific T cytotoxic cells is seen as an alternative means of preventing and treating these infections. The hospital takes donor cells and manufactures them in the lab to fight specific viruses and/or cancer. The cells are given to the patients in the outpatient clinic, in a procedure that takes less than five minutes. The cytotoxic T-cells usually take within two to six weeks after which time the patient may no longer need other medications to treat or prevent infection.

We give these cells to the patient and then we hope that in a couple of weeks the CMV viral load falls to very low levels or even zero, Dr. Bollard said. This patient is 6 months old. By giving these T-cells, he can get off the drug therapy and spare his kidneys from the toxicity of the antiviral drugs.

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Catherine M. Bollard, MBCHB, MD, of Childrens National Performs Its First Treatment Using T-Cell Therapy On Child ...

By Joel Ceausu, May 28th, 2014

An East End Montreal hospital is home to a new national network on regenerative medicine and cell therapy research. CellCAN will be based at Maisonneuve-Rosemont Hospital and directed by renowned cell therapy researcher Dr. Denis Claude Roy. The objective is to unite efforts of researchers, clinicians, funders, industry, charities, government members, patient representatives and the public. Specifically, CellCAN will promote exchanges, cooperation, partnership development and innovation in regenerative medicine and cell therapy, explained Roy. As the hub of a network of cell therapy centers and labs in Toronto, Ottawa, Quebec City, Edmonton, and Vancouver, CellCAN will propel Canadian stem cell research and clinical development forward thanks to a $3 million grant over four years. Discoveries in stem cell research make their way to clinical trials bringing researchers closer to new treatments for patients with cancer, diabetes, cardiovascular and ocular diseases, neurological and blood disorders and other health issues. Regenerative cell therapies have almost unlimited possibilities, said Roy, director of the cellular therapy laboratory at Maisonneuve-Rosemonts research centre. This will transform the nature of medicine and have significant impact on our health care systems. The Universit de Montral-affiliated hospital in Rosemont is an internationally recognized leader in hematology-oncology, stem cell transplants, ophthalmology, nephrology and kidney transplants. The funds come from the federally financed Networks of Centres of Excellence, Maisonneuve-Rosemont Foundation, Ronald and Herbert Black, and various organizations across Canada.n

Click here to see the full newspaper. Updated on May 28, 2014

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East End home for cell network

Charlottesville, VA (PRWEB) May 27, 2014

Researchers at the University of Virginia School of Medicine have devised a way to detect unintended side effects of manipulating genes using a revolutionary new system that is sweeping the scientific world by storm.

The gene targeting system, called CRISPR, allows editing of genetic information at specifically targeted sites in the genome. UVAs new approach reveals the system has the potential to bind to unintended sites and cause gene mutations at some of these sites mutations that could have serious consequences for research and efforts to develop medical treatments. UVAs new approach, however, also identifies ways to help prevent those potentially dangerous off-target effects, allowing scientists to improve their results with this important new gene-editing system.

A primary goal of gene manipulation is to correct harmful mutations, so it is vital to avoid introducing mutations unintentionally, explained UVAs Mazhar Adli, PhD, of the Department of Biochemistry and Molecular Genetics. We know that genetic mutations are hallmarks of disease. The whole aim is to change these apparent genetic mutations, to go and correct these mutations, he said. We want to change this information only at the targeted space, at the targeted locus. If you change any other information, basically you are introducing mutations that you dont want. You are correcting one gene and potentially you might be introducing mutations in 10 other genes and maybe many other places in the genome.

Adlis new research sheds light on the potential off-target effects of the CRISPR/Cas9 gene editing system. The system has proved extremely popular because it allows scientists to manipulate specific sections of the genome of living mammals, making it a tool of tremendous importance for scientific research. It has been adopted quickly and widely, including for work in human cells, because it is comparatively simple and because many labs have the resources to use it. You can basically target any genomic region in living cells and change the genetic information, which has been the holy grail of research for the past several decades, Adli said. To be able to go and change the genetic information in living cells was a dream, basically.

UVAs new research helps explain the mechanism that underpins the CRISPR system and why it is vulnerable to off-target gene mutations. We not only found where it binds in the genome, we also investigated why it goes to these regions in the genome. By analyzing specific sequences underlying these off-targets, we also found out the determinants why it goes to the on-targets and also to these off-target regions, and our research shows that it goes there because of some sequence similarity to the original targeted regions, Adli said. So our results will help improve the specificity of the system so that we can minimize the off-targets.

Adlis work also showed that the naturally occurring form of a key enzyme used in the CRISPR system introduces far more mutations than an altered form of that enzyme that is less commonly used. The former cuts both strands of DNA during gene editing, allowing mutations to occur, while the latter snips only one strand, allowing cells to repair the damage without introducing mutations.

Unfortunately the wild-type form [the naturally occurring form] is much easier to deal with. Everyone in the field is using the wild type. Now with this paper, and with additional papers coming out, they will have to stop using the wild type form of the enzyme. They have to use the [altered form] to overcome the off-targets. It is much superior and the off-targets are very dangerous.

The findings have been published online by the journal Nature Biotechnology and will appear in a forthcoming print edition. The paper was authored by UVAs Cem Kuscu and Sevki Arslan, sharing credit as the lead authors; Ritambhara Singh; Jeremy Thorpe; and Adli.

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UVA Detects Unwanted Effects of Important Gene ...

Scientists armed with a supercomputer and a vast trove of newly collected data on the body's most potent "tumor suppressor" gene have created the best map yet of how the gene works, an accomplishment that could lead to new techniques for fighting cancers, which are adept at disabling the gene in order to thrive.

Scientists from the University of Colorado Cancer Center and the University of Colorado Boulder used a new technology to tease out how the p53 gene -- which is responsible for recognizing damaged DNA in cells and then marking them for death -- is actually able to suppress tumors by determining what other genes p53 regulates. The study, published in the journal eLife, describes dozens of new genes directly regulated by p53.

The study authors say further research can explore which of these genes are necessary for p53's cancer-killing effect, how cancer cells evade these p53-activated genes, and how doctors may be able to moderate cancer cells' ability to stay safe from these genetic attempts at suppression.

The exhaustively studied p53 gene -- which has been the subject of 50,000 papers over more than 30 years of research -- is the most commonly inactivated gene in cancers. When p53 acts, cells are stopped or killed before they can survive, grow, replicate and cause cancer.

As such, all cancers must deal with p53's anti-tumor effects. Generally, there are two ways that cancer cells do this: by mutating p53 directly or by making a protein called MDM2, which stops p53 from functioning

The current study explores cancer cells' second strategy of blocking p53 function by producing the protein MDM2. Researchers have reasoned that treating a patient with an MDM2 inhibitor should allow p53 to restart its anti-cancer activities.

"MDM2 inhibitors, which are through phase I human trials, effectively activate p53 but manage to kill only about one-in-20 tumors," said Joaqun Espinosa, an investigator at the CU Cancer Center, an associate professor of molecular, cellular and developmental biology at CU-Boulder, and the paper's co-senior author. "The question is why. What else is happening in these cancer cells that allow them to evade p53?"

The answer is in what are called "downstream" effects of this gene, Espinosa said. The gene p53 doesn't act against cancer alone. Instead, it is the master switch that sets in motion a cascade of genetic events that lead to the destruction of cancer cells. And until now, it was unclear exactly which other genes were directly activated by p53.

The imperfect knowledge of p53's effects isn't for lack of research interest. Researchers have written thousands of papers exploring p53's targets and, in fact, many genetic targets are previously known. Most of these studies determine genetic targets by measuring levels of RNA.

When a gene is activated, it creates a protein. But between the gene and its protein product is the measurable step of RNA -- the more gene-specific RNA, the more often a gene's informational blueprint is carried to the cell's manufacturing centers, and the more protein is eventually made. Researchers measure RNA to see which genes are being turned up or down by any other gene.

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How a cancer-killing gene may actually work

Student Work:Genetically Modified Organisms:ChiChi (G8)
After learning about genetic engineering and genetically modified organisms, grade 8 students were asked to write a script and create a GMO animation to discuss the ethical implications of...

By: Springs Pacelli

Originally posted here:
Student Work:Genetically Modified Organisms:ChiChi (G8) - Video

NEW YORK (TheStreet) --Foundation Medicine (FMI) is a good company in what many believe is an almost-impossible sector -- diagnostics. Bears believe diagnostic tests become commoditized with vanishing profit margins and no real winners. Diagnostic companies are simply providers of hardware (or a simple operator of hardware) vulnerable to relatively low barriers to entry. Even if a company roils out a successful diagnostic test, fast followers introduce similar tests and profit margins disappear. There is certainly a kernel of truth to these bearish arguments but Foundation Medicine has a comparative advantage over other diagnostic companies which investors are failing to recognize.

The price of a particular diagnostic test may fall over time (the rapidly diminishing cost to sequence the whole genome is a great example) but more important is what happens when adoption of these tests accelerates and the Big Data assembled from them are put to use treating patients.

Foundation Medicine's current FoundationOne cancer diagnostic examines about 236 genes and four types of alterations. While you can tout the advantage of a single test that comprehensively analyses information versus a series of more specific tests, the point is this is a lot of data. If you take the low end of the company's guidance -- 22,000 tests in 2014 -- this implies information on about 5.2 million genetic alterations generated in only one year. If you include how these patients are treated and the efficacy of these treatments, you can see how Foundation Medicine is putting Big Data to good use.

Foundation Medicine's advantage comes from taking its data and combining it with the latest research and clinical practices to provide information to a doctor about 1) the specific genetic makeup of their patients' cancers, and 2) potentially effective treatments using approved drugs or experimental drugs in clinical trials. The value Foundation Medicine provides is not the diagnostics, itself, but analyzing the data that comes out of the diagnostics.

These data are exceptionally useful for oncologists, who likely have little time to follow all of the clinical trials and changing treatment paradigms or new molecular targets. This means the real barrier of entry Foundation Medicine builds is not the testing but its ability to collect and analyze the data. These data are proprietary, so each additional test and refinement of the analysis puts the company further ahead and increases that barrier. A competitor might come along and create a similar test, but the diagnostic results themselves are not meaningful unless put into a useful context. It is that useful context Foundation Medicine constantly attempts to develop and improve, making it difficult for others to enter into the space and compete in a meaningful way.

Foundation Medicine is not without its challenges. Foremost, the company must convince insurance companies to pay for tests like FoundationOne. While one can talk in the abstract about the utility of having a single test that examines all potentially important genetic mutations and the utility it provides to oncologists and patients, it is something different to change the way payers reimburse for diagnostics. The reimbursement process will be slow and there is always the risk Foundation Medicine isn't successful. This weekend's American Society of Clinical Oncology (ASCO) annual meeting is important in this regard.

Neither of these challenges will happen overnight so don't expect Foundation Medicine to be a quick-hit winner. Investing in the company requires patience.Ultimately, Foundation Medicine looks like a nice risk/reward at its current valuation.

Sobek is long Foundation Medicine.

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Bull Thesis on Foundation Medicine: Using Big Data to Improve Treatment Decisions for Cancer Patients

PUBLIC RELEASE DATE:

28-May-2014

Contact: Stephanie Desmon sdesmon1@jhmi.edu 410-955-8665 Johns Hopkins Medicine

A multicenter team of researchers report that a commercial test designed to rule out the presence of genetic biomarkers of prostate cancer may be accurate enough to exclude the need for repeat prostate biopsies in many if not most men.

"Often, one biopsy is not enough to definitively rule out prostate cancer," says study researcher Jonathan Epstein, M.D., director of the Division of Surgical Pathology and a professor of pathology, urology and oncology at the Johns Hopkins University School of Medicine. "Our research finds that by looking for the presence or absence of cancer in a different way, we may be able to offer many men peace of mind without putting them through the pain, bleeding and risk of infection that can come with a repeat biopsy."

The new research, called the Detection of Cancer Using Methylated Events in Negative Tissue (DOCUMENT) study, suggests that an initial biopsy complemented with an epigenetic diagnostic test accurately rules out the existence of cancer up to 88 percent of the time. The test, developed by MDxHealth, which paid for the study, was described online in April in The Journal of Urology.

The test specifically captures the presence of chemical modifications to non-nuclear DNA sequences within cells that commonly appear when prostate cancer is present. These so-called epigenetic changes, which add a methyl group to the biochemical makeup of the DNA, alter the way genes function without changing their foundational DNA sequence. The researchers analyzed tissue from biopsies from 320 men with elevated prostate-specific antigen (PSA) levels whose results were negative for prostate cancer. The men were patients at The Johns Hopkins Hospital; the University of California, Los Angeles; the Cleveland Clinic; Eastern Virginia Medical School; and Lahey Hospital & Medical Center.

The epigenetic biomarkers the test detects reflect a process called DNA hypermethylation, in which a methyl group is chemically attached to DNA in this case, to genes called GSTP1, APC and RASSF1. These genes are known to play prominent tumor suppressive roles in key cancer-related pathways. When these genes are hypermethylated, they are commonly silenced, which can lead to a loss of this tumor-suppressing function and the emergence of cancer.

Specifically, the GSTP1 gene acts as a detoxifying agent, preventing genomic damage by carcinogens. Studies find that GSTP1 is methylated in up to 90 percent of prostate cancer cases, making it a strong indicator of the disease.

For the study, pathologists compared methylation levels between the subjects' initial tissue biopsies and later tissue samples taken from each man done within 24 months of the first biopsy. They found that average levels of APC and RASSF1 were about twice as high in the 92 subjects whose second biopsies yielded positive results, as compared to the 228 with two negative biopsies. For GSTP1, the levels were more than eight times higher in the cancerous biopsies.

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Study affirms value of epigenetic test for markers of prostate cancer

Researchers used an emerging technique to correct the gene behind a fatal immune system disorder in an infant.

A new kind of gene therapy which involves editing, rather than replacing, faulty genes in sick people, is being used experimentally in patients. The latest report shows how scientists can correct a broken gene as it sits in the patients genome. How the health of the patient, a 4-month old infant, will change is yet to be reported.

Genome editing technology is considered a promising new tool for curing disease. For decades, gene therapy has meant that a virus delivers a functional copy of a gene that is dysfunctional in a patient. The dysfunctional copy remains and the therapeutic version typically remains separate from the rest of the genome.

The technology has drawbacks. First, by sitting outside of the genome, the activity of therapeutic gene isnt regulated properly. In some cases, the therapeutic copy is delivered by a retrovirus the plunks the new gene down near randomly in the patients genome, which risks disrupting another gene, potentially causing cells to turn cancerous. Second, some diseases, such as Huntingtons, cant be treated this way because the broken copy of the gene causes harm. To treat these kinds of conditions, the original copy of the gene must be corrected. Using genome editing to repair genes could circumvent these issues (see Genome Surgery).

In the new study, published today in the journal Nature, researchers in Milan treated a condition known as Severe Combined Immunodeficiency Syndrome, or SCID (this condition is sometimes referred to as bubble boy disease because children afflicted may live in protected environments because the risk of death from infectious disease is extremely likely). Children with this genetic condition have been treated with the additive gene therapy method in the past, and some suffered leukemia-like diseases as a side effect (see The Glimmering Promise of Gene Therapy). In the new report, researchers describe treating a single infant with zinc-finger nucleases designed to repair a defective copy of an important immune system gene.

The report does not look at the long term health effects for the infant. But the team shows that the genome editing did reconstitute a functional copy of the immune system gene in a small fraction of bone marrow cells (which give rise to immune cells). This work is undoubtedly a step towards using gene repair for gene therapy, writes immunologist Alain Fischer in an accompany article also published in Nature. Fischer led the first successful gene therapy trials for SCID patients.

In March, researchers reported an even more dramatic example of gene repair. Scientists used zinc fingers to engineer the immune cells of patients with HIV to resist the virus (see Can Gene Therapy Cure HIV?). In a few patients, the amount of virus in the blood decreased and in one patient, the virus could no longer be found.

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Genome Editing to Reverse Bubble Boy Syndrome

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28-May-2014

Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Tampa, Fla. (May 28, 2014) To be suitable for medical transplantation, one idea is that human embryonic stem cells (hESCs) need to remain "undifferentiated" i.e. they are not changing into other cell types. In determining the best way to culture hESCs so that they remain undifferentiated and also grow, proliferate and survive, researchers have used blood cell "feeder-layer" cultures using animal-derived feeder cells, often from mice (mouse embryonic fibroblasts [MEFs]). This approach has, however, been associated with a variety of contamination problems, including pathogen and viral transmission.

To avoid contamination problems, a Brazilian research team has investigated the use of human menstrual blood-derived mesenchymal cells (MBMCs) as feeder layers and found that "MBMCs can replace animal-derived feeder systems in human embryonic stem cell culture systems and support their growth in an undifferentiated stage."

The study will be published in a future issue of Cell Medicine, but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/cm/pre-prints/content-CM1019silvadosSantos.

"Human embryonic stem cells present a continuous proliferation in an undifferentiated state, resulting in an unlimited amount of cells with the potential to differentiate toward any type of cell in the human body," said study corresponding author Dr. Regina Coeli dos Santos Goldenberg of the Instituto de Biofisica Carlos Chagas Filho, Universidade Federal do Rio de Janeiro. "These characteristics make hESCs good candidates for cell based therapies."

Feeder-layers for hESCs comprised of MEFs have been efficiently used for decades but, because of the clinical drawbacks, the authors subsequently experimented with human menstrual blood cells as a potential replacement for animal-derived feeder-layers, not only for negating the contamination issues, but also because human menstrual blood is so accessible. MBMCs are without ethical encumbrances and shortages, nor are they difficult to access - a problem with other human cells, such as umbilical cord blood cells, adult bone marrow cells or placenta cells.

"Menstrual blood is derived from uterine tissues," explained the researchers. "These cells are widely available 12 times a year from women of child-bearing age. The cells are easily obtained, possess the capability of long-term proliferation and are clinically compatible with hESCs-derived cells."

The researchers found that their culture system using MBMCs as a feeder-layer for hESCs are the "closest and more suitable alternative to animal-free conditions for growing hESCs" and a "good candidate for large-expansion of cells for clinical application." They also found no difference in growth factor expression when comparing the use of growth factors in both the standard feeder system using animal cells and the feeder system they tested using hESCs.

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Brazilian researchers find human menstrual blood-derived cells 'feed' embryonic stem cells

Block GmbH Centre for Living Cell Therapy
The fresh cell therapy/stem cell therapy is always a full body treatment. The improvement of the function of individual organs also affects all other organs positively.

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SINGAPORE: Australia-based stem cell therapy firm Mesoblast has announced plans to accelerate commercial manufacturing operations in Singapore.

This is to prepare for new product launches in the United States and other major markets over the next couple of years.

Its existing operations in Singapore include making stem cell products for clinical trials under its contract with its partner, pharmaceutical company Lonza.

One of its key products still awaiting full approval is Prochymal, which Mesoblast says can help to more than double the survival rate of patients suffering from complications after receiving tissue transplants from donors -- known as graft versus host disease.

The global stem cell market is expected to grow at an average annual rate of 12 per cent between 2011 and 2016 to reach more than S$8 billion by 2016.

Mesoblast said commercial manufacturing requires a much larger capacity and operations must be scaled-up to meet regulatory demands.

Silviu Itescu, chief executive at Mesoblast, said: "We are now in a phase of making more investments in order to get our processes to commercial scale. That anticipates successful commercial launches.

"If we're successful in that over the next 18-24 months, then we're going to leverage the investment in our commercial facilities to be able to build up and prepare for launching of much larger opportunities in cardiovascular medicine, orthopaedics and diseases of immunity and inflammation which would require purpose-built facilities."

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Mesoblast to accelerate operations in S'pore

Personalized Medicine Update: Dr. Annabelle Rodriguez-Oquendo
Dr. Rodriguez-Oquendo is studying the genetic link between healthy HDL cholesterol, heart disease, and infertility in women.

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Personalized Medicine Update: Dr. Annabelle Rodriguez-Oquendo - Video

Spinal Cord Injury; Director of Faculty; Staff Disability Services (Employment Videos)
This video shows an individual with a spinal cord injury describing adaptive techniques she uses for work and demonstrating job tasks she performs in her job as a director of faculty and staff...

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Family Centers President Bob Arnold and Barbara Netter.

At its annual luncheon in Darien on April 23, the Center for HOPE recognized Greenwich resident Barbara Netter for her many contributions toward the advancement of gene therapy-based cancer treatments.

As the President of the Alliance for Cancer Gene Therapy (ACGT) a nonprofit organization she founded with her late husband, Edward, in 2001 Ms. Netter has raised nearly $22.5 million for numerous gene therapy research initiatives around the world. Over the years, ACGTs support has been instrumental in launching 17 human clinical trials addressing lung, ovarian, prostate and breast cancer, as well as lymphoma and leukemia.

For her work providing millions of cancer patients with a renewed sense of optimism, Ms. Netter was presented with the Ray of HOPE Award. The Ray of HOPE is the Center for HOPEs highest honor that highlights a member of the community whose efforts assist those coping with a loss, critical illness or life-altering circumstance.

We are delighted to recognize Barbara with a Ray of HOPE Award, shinning the light on her body of work in the area of critical illness and bereavement, said Bob Arnold, Family Centers president. Barbara truly inspires us, as her bountiful efforts continue to fill our world with hope and compassion.

In addition to her work with ACGT, Ms. Netter has been involved for many years with The Den for Grieving Kids a Family Centers program providing support to children and families who have lost a loved one.

The Center for HOPE Luncheon also featured a keynote address from Huffington Post founder and editor-in-chief Arianna Huffington.

All proceeds from the event benefited the Center for HOPE and The Den for Grieving Kids.

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Center for HOPE honors Netter

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27-May-2014

Contact: Garth Sundem garth.sundem@ucdenver.edu University of Colorado Denver

Scientists armed with a supercomputer and a vast trove of newly collected data on the body's most potent "tumor suppressor" gene have created the best map yet of how the gene works, an accomplishment that could lead to new techniques for fighting cancers, which are adept at disabling the gene in order to thrive.

Scientists from the University of Colorado Cancer Center and the University of Colorado Boulder used a new technology to tease out how the p53 genewhich is responsible for recognizing damaged DNA in cells and then marking them for deathis actually able to suppress tumors by determining what other genes p53 regulates. The study, published in the journal eLife, describes dozens of new genes directly regulated by p53.

The study authors say further research can explore which of these genes are necessary for p53's cancer-killing effect, how cancer cells evade these p53-activated genes, and how doctors may be able to moderate cancer cells' ability to stay safe from these genetic attempts at suppression.

The exhaustively studied p53 genewhich has been the subject of 50,000 papers over more than 30 years of researchis the most commonly inactivated gene in cancers. When p53 acts, cells are stopped or killed before they can survive, grow, replicate and cause cancer.

As such, all cancers must deal with p53's anti-tumor effects. Generally, there are two ways that cancer cells do this: by mutating p53 directly or by making a protein called MDM2, which stops p53 from functioning.

The current study explores cancer cells' second strategy of blocking p53 function by producing the protein MDM2. Researchers have reasoned that treating a patient with an MDM2 inhibitor should allow p53 to restart its anti-cancer activities.

"MDM2 inhibitors, which are through phase I human trials, effectively activate p53 but manage to kill only about one-in-20 tumors," said Joaqun Espinosa, an investigator at the CU Cancer Center, an associate professor of molecular, cellular and developmental biology at CU-Boulder, and the paper's co-senior author. "The question is why. What else is happening in these cancer cells that allow them to evade p53?"

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New University of Colorado study illuminates how cancer-killing gene may actually work