Archive for the ‘Gene Therapy Research’ Category

05/24/2012 . (Classic Rock) Former Iron Maiden singer Paul Di'Anno wants his ex-bandmate Clive Burr to undergo stem cell therapy, despite the costs and risks associated with the procedure.

Burr, the drummer with Maiden from 1979 until 1982, has been in a wheelchair as a result of multiple sclerosis, which has been attacking his nervous system since before he was diagnosed in 2002.

MS reduces the ability of the brain and spinal cord to communicate with each other, resulting in a wide range of potentially severe symptoms. The cause is unknown and there is no cure; but in 2009 researchers made the first breakthrough in reversing symptoms through stem cell therapy.

Di'Anno tells Talking Metal Pirate Radio Burr's condition is "not very good at all." He had a lot to say, read it here.

Classic Rock Magazine is an official news provider for antiMusic.com. Copyright Classic Rock Magazine- Excerpted here with permission.

antiMUSIC News featured on RockNews.info and Yahoo News

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Di'Anno Wants Former Iron Maiden Bandmate To Undergo Stem Cell Therapy

TRADITION Three former Treasure Coast high school students have been awarded the first $3,000 internships at the Vaccine and Gene Therapy Institute's research laboratories in the Tradition Center for Innovation.

The Summer Undergraduate Research Foundation at VGTI, known as SURF-VGTI, pairs each student with a faculty mentor who will then work on research project for six weeks starting June 11. Their research findings will be presented in late July, according to the VGTI website.

Daniel Rosenberg and Lysa Vola, both graduates of Jensen Beach High School, and Robert Tack, a graduate of Port St. Lucie High School all now college students are this year's first interns, said John Schatzle, principle investigator and director of scientific affairs at VGTI.

For the past few years, Torrey Pines Institute for Molecular Studies, also located in Tradition, has been offering similar type summer internships to area college students and public school teachers.

VGTI concentrated on offering internships to students from the Treasure Coast and providing them real-world research experience, Schatzle said. "They will be working like any other researcher would be in the lab."

VGTI opened the doors of its new $47 million, 100,000-square-foot research facility in February to develop vaccines and immunotherapies for diseases such as AIDS, cancer, tuberculosis and diseases associated with emerging viral infections.

Rosenberg, 19, who now is a biomedical engineering student at the University of Miami, will be paired with a mentor who is studying HIV, Schatzle said.

"We've actually found one of its major hiding places in the body and trying to figure out how to purge it, and effectively have a cure for HIV," he said. "(Rosenberg's) lab is working on that project."

Rosenberg said he is excited to be working on such an important study.

"The last semester I took a physiology class and I learned a lot about AIDS and now I can apply that knowledge," Rosenberg said. "It astounds me that it impacts so many people every year and we have not found a solid cure for the disease."

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Tradition-based VGTI offers its first summer research internships

Featured Article Academic Journal Main Category: Fertility Also Included In: Genetics;Men's Health Article Date: 25 May 2012 - 2:00 PDT

Current ratings for: 'Sperm Gene Discovery May Lead To Non-Hormonal Male Contraceptive'

Currently, the only male contraceptives available rely on disrupting the production of hormones like testosterone, which can cause unpleasant side effects such as acne, irritability and mood swings.

First author Dr Lee Smith is Reader in Genetic Endocrinology at the University of Edinburgh's Centre for Reproductive Health. He told the media:

"If we can find a way to target this gene in the testes, we could potentially develop a non-hormonal contraceptive."

The gene, called Katnal1, is critical to enabling sperm to mature in the testes.

Finding a way to regulate the gene could potentially stop the sperm maturing and render them ineffective.

Not only could this form the basis of a new type of male contraceptive that does not involve disrupting hormone levels, it could also lead to new treatments for male infertility caused by a faulty Katnal1 gene.

"The important thing is that the effects of such a drug would be reversible because Katnal1 only affects sperm cells in the later stages of development, so it would not hinder the early stages of sperm production and the overall ability to produce sperm," explained Smith.

"Although other research is being carried out into non-hormonal male contraceptives, identification of a gene that controls sperm production in the way Katnal1 does is a unique and significant step forward in our understanding of testis biology," he added.

Link:
Sperm Gene Discovery May Lead To Non-Hormonal Male Contraceptive

ScienceDaily (May 24, 2012) Researchers at NYU School of Medicine have, for the first time, identified a single gene that simultaneously controls inflammation, accelerated aging and cancer.

"This was certainly an unexpected finding," said principal investigator Robert J. Schneider, PhD, the Albert Sabin Professor of Molecular Pathogenesis, associate director for translational research and co-director of the Breast Cancer Program at NYU Langone Medical Center. "It is rather uncommon for one gene to have two very different and very significant functions that tie together control of aging and inflammation. The two, if not regulated properly, can eventually lead to cancer development. It's an exciting scientific find."

The study, funded by the National Institutes of Health, appears online ahead of print May 24 in Molecular Cell and is scheduled for the July 13 print issue.

For decades, the scientific community has known that inflammation, accelerated aging and cancer are somehow intertwined, but the connection between them has remained largely a mystery, Dr. Schneider said. What was known, due in part to past studies by Schneider and his team, was that a gene called AUF1 controls inflammation by turning off the inflammatory response to stop the onset of septic shock. But this finding, while significant, did not explain a connection to accelerated aging and cancer.

When the researchers deleted the AUF1 gene, accelerated aging occurred, so they continued to focus their research efforts on the gene. Now, more than a decade in the making, the mystery surrounding the connection between inflammation, advanced aging and cancer is finally being unraveled.

The current study reveals that AUF1, a family of four related genes, not only controls the inflammatory response, but also maintains the integrity of chromosomes by activating the enzyme telomerase to repair the ends of chromosomes, thereby simultaneously reducing inflammation, preventing rapid aging and the development of cancer, Dr. Schneider explained.

"AUF1 is a medical and scientific trinity," Dr. Schneider said. "Nature has designed a way to simultaneously turn off harmful inflammation and repair our chromosomes, thereby suppressing aging at the cellular level and in the whole animal."

With this new information, Dr. Schneider and colleagues are examining human populations for specific types of genetic alterations in the AUF1 gene that are associated with the co-development of certain immune diseases, increased rates of aging and higher cancer incidence in individuals to determine exactly how the alterations manifest and present themselves clinically.

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Key gene found responsible for chronic inflammation, accelerated aging and cancer

Gene Findings in African-Americans May Pave Way Toward Better Quit-Smoking Treatments

By Denise Mann WebMD Health News

Reviewed by Laura J. Martin, MD

May 22, 2012 -- A "smoking gun" gene may play a role in how many cigarettes certain smokers puff each day.

Researchers from 50 medical institutions across the country analyzed genetic material of more than 32,000 African-American smokers and non-smokers to see if certain genes predicted when they began smoking, how many cigarettes they smoked, and how easily they were able to quit.

According to the new study, a variant in a nicotine receptor gene predicts about one extra cigarette smoked per day. This same general location has been implicated in smoking behavior among white Europeans. Among African-Americans, the new genetic marker appears on a different spot on the same gene.

The findings appear in the May 22 issue of Translational Psychiatry. They are part of the Study of Tobacco in Minority Populations (STOMP) Genetics Consortium.

"This region is really important for addiction biology, regardless of race or ethnicity," says researcher Helena Furberg, PhD, an assistant attending epidemiologist at Memorial Sloan-Kettering Cancer Center in New York City.

This is somewhat surprising, she says. "Smoking behaviors differ among ethnic groups." For example, African-Americans typically start smoking at later ages than their counterparts of European descent and smoke fewer cigarettes each day. But they have a higher risk for lung cancer and are less likely to quit smoking.

The findings hold potential for tailoring smoking cessation treatments down the road, Furberg says. "The next research step would be to see if currently available smoking cessation medications would work better or differently among people who carry these variants."

See the article here:
Smoking Gene May Reveal Why Some People Smoke More

OMAHA, Neb.--(BUSINESS WIRE)--

Transgenomic, Inc. (TBIO.OB) today announced it has entered into an agreement with Gene Logic, Inc., an Ocimum Biosolutions Company, to acquire its biorepository assets for $250,000 in cash. The biorepository contains thousands of human biological samples that can be used to validate diagnostic assays developed by Transgenomic. Transgenomic will use the samples to develop and validate its REVEAL family of proprietary ICE COLD-PCR-based oncology assays, as well as its WAVE MCE System and SURVEYOR Scan Mutation Detection Kits.

This transaction significantly strengthens Transgenomics position in translational medicine services, providing a deep source of clinical samples allowing us to validate new drug-associated genetic targets, said Craig Tuttle, Chief Executive Officer of Transgenomic. The biorepository provides not only strategic and operational benefits, but also long-term cost savings. Acquiring specimens on a case-by-case basis is expensive and time-consuming; such expenditure would quickly surpass the cost of acquiring this asset.

The Gene Logic biorepository consists of a high quality, diverse collection of human tissue samples and extracted DNA specimens with linked clinical information. The 60,000 samples and specimens cover multiple disease areas, including many thousands of cancer tissue specimens.

About Transgenomic

Transgenomic, Inc. (www.transgenomic.com) is a global biotechnology company advancing personalized medicine in cancer and inherited diseases through its proprietary molecular technologies and world-class clinical and research services. The Company has three complementary business divisions: Transgenomic Pharmacogenomic Services is a contract research laboratory that specializes in supporting all phases of pre-clinical and clinical trials for oncology drugs in development. Transgenomic Clinical Laboratories specializes in molecular diagnostics for cardiology, neurology, mitochondrial disorders, and oncology. Transgenomic Diagnostic Tools produces equipment, reagents, and other consumables that empower clinical and research applications in molecular testing and cytogenetics. Transgenomic believes there is significant opportunity for continued growth across all three businesses by leveraging their synergistic capabilities, technologies, and expertise. The Company actively develops and acquires new technology and other intellectual property that strengthen its leadership in personalized medicine.

Forward-Looking Statements

Certain statements in this press release constitute forward-looking statements of Transgenomic within the meaning of the Private Securities Litigation Reform Act of 1995, which involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. Forward-looking statements include, but are not limited to, those with respect to management's current views and estimates of future economic circumstances, industry conditions, company performance and financial results, including the ability of the Company to grow its involvement in the diagnostic products and services markets. The known risks, uncertainties and other factors affecting these forward-looking statements are described from time to time in Transgenomic's filings with the Securities and Exchange Commission. Any change in such factors, risks and uncertainties may cause the actual results, events and performance to differ materially from those referred to in such statements. Accordingly, the Company claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 with respect to all statements contained in this press release. All information in this press release is as of the date of the release and Transgenomic does not undertake any duty to update this information, including any forward-looking statements, unless required by law.

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Transgenomic Acquires Clinical Sample Biorepository from Gene Logic

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Tara Womersley tara.womersley@ed.ac.uk 44-131-650-9836 University of Edinburgh

A new type of male contraceptive could be created thanks to the discovery of a key gene essential for sperm development.

The finding could lead to alternatives to conventional male contraceptives that rely on disrupting the production of hormones, such as testosterone and can cause side-effects such as irritability, mood swings and acne.

Research, led by the University of Edinburgh, has shown how a gene Katnal1 is critical to enable sperm to mature in the testes.

If scientists can regulate the Katnal1 gene in the testes, they could prevent sperm from maturing completely, making them ineffective, without changing hormone levels.

The research, which is published in the journal PLoS Genetics, could also help in finding treatments for cases of male infertility, when malfunction of the Katnal1 gene hampers sperm development.

Dr Lee Smith, Reader in Genetic Endocrinology at the Medical Research Council Centre for Reproductive Health at the University of Edinburgh, said: "If we can find a way to target this gene in the testes, we could potentially develop a non-hormonal contraceptive.

"The important thing is that the effects of such a drug would be reversible because Katnal1 only affects sperm cells in the later stages of development, so it would not hinder the early stages of sperm production and the overall ability to produce sperm.

"Although other research is being carried out into non-hormonal male contraceptives, identification of a gene that controls sperm production in the way Katnal1 does is unique and a significant step forward in our understanding of testis biology."

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Gene discovery points towards new type of male contraceptive

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Tara Womersley Tara.Womersley@ed.ac.uk 44-131-650-9836 Public Library of Science

A new type of male contraceptive could be created thanks to the discovery of a key gene essential for sperm development.

The finding could lead to alternatives to the conventional male contraceptives that rely on disrupting the production of hormones, such as testosterone. These treatments can cause side-effects such as irritability, mood swings and acne.

Research, led by the University of Edinburgh, has shown how a gene Katnal1 is critical to enable sperm to mature in the testes.

If scientists can regulate the Katnal1 gene in the testes, they could prevent sperm from maturing completely, making them ineffective without changing hormone levels.

The research, which is published in the journal PLoS Genetics, could also help in finding treatments for cases of male infertility when malfunction of the Katnal1 gene hampers sperm development.

Dr Lee Smith, Reader in Genetic Endocrinology at the University of Edinburgh's Centre for Reproductive Health, said: "If we can find a way to target this gene in the testes, we could potentially develop a non-hormonal contraceptive.

"The important thing is that the effects of such a drug would be reversible because Katnal1 only affects sperm cells in the later stages of development, so it would not hinder the early stages of sperm production and the overall ability to produce sperm.

"Although other research is being carried out into non-hormonal male contraceptives, identification of a gene that controls sperm production in the way Katnal1 does is a unique and significant step forward in our understanding of testis biology."

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Gene study could pave way for non-hormonal male contraceptive

DARPA, the science arm of the US Department of Defense, is trying to find a way to create a streamlined manufacturing process for purpose-specific engineering of plants and animals, reports Popular Science's Rebecca Boyle. This program, called Living Foundries, "sets up an assembly line paradigm for life and its constituent parts," Boyle says. "Under this program, genetic engineering would no longer be limited to modification of existing organisms instead, scientists would be able to concoct anything they wanted from scratch, using a suite of ingredients and processes that could apply in any situation." And DARPA's first grants for the program have just been announced $15.5 million spread among six institutions and companies, including the J. Craig Venter Institute. This last pick is particularly appropriate, she says, given the group's work in synthetic biology.

The purpose of the grants is to build a basic library of modularized parts that can be used in assembling various organisms, Boyle says, like wires or circuits that can be used to build electronics. "The ultimate goal is a genetic starter set that could be snapped together like so many Legos, forming any system the military might require," she adds.

Our sister publication GenomeWeb Daily News has more on the project here.

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DARPA's Synthetic Army

LOS ANGELES Understanding the genetic diversity within and between populations has important implications for studies of human disease and evolution. This includes identifying associations between genetic variants and disease, detecting genomic regions that have undergone positive selection and highlighting interesting aspects of human population history.

Now, a team of researchers from the UCLA Henry Samueli School of Engineering and Applied Science, UCLA's Department of Ecology and Evolutionary Biology and Israel's Tel Aviv University has developed an innovative approach to the study of genetic diversity called spatial ancestry analysis (SPA), which allows for the modeling of genetic variation in two- or three-dimensional space.

Their study is published online this week in the journal Nature Genetics.

With SPA, researchers can model the spatial distribution of each genetic variant by assigning a genetic variant's frequency as a continuous function in geographic space. By doing this, they show that the explicit modeling of the genetic variant frequency the proportion of individuals who carry a specific variant allows individuals to be localized on a world map on the basis of their genetic information alone.

"If we know from where each individual in our study originated, what we observe is that some variation is more common in one part of the world and less common in another part of the world," said Eleazar Eskin, an associate professor of computer science at UCLA Engineering. "How common these variants are in a specific location changes gradually as the location changes.

"In this study, we think of the frequency of variation as being defined by a specific location. This gives us a different way to think about populations, which are usually thought of as being discrete. Instead, we think about the variant frequencies changing in different locations. If you think about a person's ancestry, it is no longer about being from a specific population but instead, each person's ancestry is defined by the location they're from. Now ancestry is a continuum."

The team reports the development of a simple probabilistic model for the spatial structure of genetic variation, with which they model how the frequency of each genetic variant changes as a function of the location of the individual in geographic space (where the gene frequency is actually a function of the x and y coordinates of an individual on a map).

"If the location of an individual is unknown, our model can actually infer geographic origins for each individual using only their genetic data with surprising accuracy," said Wen-Yun Yang, a UCLA computer science graduate student.

"The model makes it possible to infer the geographic ancestry of an individual's parents, even if those parents differ in ancestry. Existing approaches falter when it comes to this task," said UCLA's John Novembre, an assistant professor in the department of ecology and evolution.

SPA is also able to model genetic variation on a globe.

Read more from the original source:
New genetic method pinpoints geographic origin

BOSTON The great promise of the Human Genome Project is that if we can crack the genetic code in each of our cells, we may be able to predict what diseases we might get and prevent them. But more than a decade into this project, no medical miracles have been produced. Now, a new study by the Harvard School of Public Health has more disappointing news. WBURs All Things Considered host Sacha Pfeiffer spoke with the studys senior author, Peter Kraft, an associate professor of epidemiology at Harvard.

Sacha Pfeiffer: Your study looked at one of the possible key reasons for why simply mapping the human genome as huge a scientific accomplishment as that is might not alone be enough to start curing or preventing diseases. What else have researchers thought might be necessary to do that?

Peter Kraft: Weve actually been fabulously successful, in the last five years especially, in finding genetic variants that are associated with disease risk. But when people looked and asked the question, Do these actually help us predict whos going to be at high risk? the answer was mostly no. And one of reasons that might have been is that people looked at these variants in isolation, one at a time. But if you considered how they work together, and how they work together with the environment, to influence cancer risk or disease risk generally, people thought that might help boost the predictive ability.

So in terms of how genes work with other genes, or how genes react if you smoke, or if youre overweight, or if youve taken hormones that kind of thing?

Right, exactly. So the models up till now have assumed that a gene is a gene and its effect is the same whether you smoke or not. But, of course, that may not be the case and in fact probably isnt the case.

And so in your study you took those factors into account environmental and lifestyle factors. What did you find?

We sort of played a thought experiment and said, What if we knew how actually these things worked together? And given that information we tried to predict who was at high risk and who was at low risk. And we found that even knowing that information, which were a long way from knowing and understanding but even if we knew it, the change in the risk estimates would not be all that great. Its giving us a 1 to 3 percent increase of our ability to detect people who are at high risk.

Is that not a very useful increase?

Well, it depends on the context, but not necessarily. It seems to be in the range where your decision as a patient and your clinicians recommendations wouldnt really change that much. So given what they knew before they drew your blood and looked at your genetics, their recommendation would probably be the same.

So your study tells us that if we get our genes mapped, we might learn a little bit more if were at risk of a disease, but not very much to help our doctors. So where does that leave us in terms of our hopes for the Humane Genome Project and this idea that we could create personalized medicine customized for every individual?

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Study: Knowing Genetic Makeup May Not Help Predict Disease Risk

May 23, 2012

Isabel Teotonio

In a landmark study examining the smoking behaviour of more than 32,300 African Americans, researchers have identified a genetic marker linked to how much a person smokes.

The findings of the study, which were published Tuesday in Translational Psychiatry, may prove useful in helping develop treatments to help smokers butt out.

This kind of research has been done in the past on white populations, but studying those who are non-European is important given their greater genetic diversity.

If we want to think about a future where we can use biological markers and psychosocial history to really tailor treatments, we need to understand the genetic architecture of smoking in multiple populations, said clinical associate professor of medicine at Stanford University Sean David, the studys lead author.

We havent found the cure to smoking with this study, said David, referring to the Study of Tobacco in Minority Populations Genetics Consortium, called STOMP. But we have found an informative genetic marker of smoking quantity that we think could inform future research to help move the field forward.

Researchers combined the findings of 13 previous studies, which provided a sample size of 32,389 men and women of African ancestry. This enabled them to better see links that may have been too subtle to spot in smaller studies. Genome-wide association studies are used to identify common genetic factors that influence health and disease.

The STOMP study, which included 78 researchers from 50 academic institutions across the United States, is the first meta-analysis of genome-wide association studies for smoking behaviours in African Americans. (Meta-analysis is a statistical technique for combining the results of independent studies.)

Investigators gathered a variety of data, including when people smoked their first cigarette, when they began smoking regularly and if they were heavy, or light, smokers.

Continue reading here:
Genetic marker predicts smoking behaviour in African Americans

Public release date: 23-May-2012 [ | E-mail | Share ]

Contact: Wileen Wong Kromhout wwkromhout@support.ucla.edu 310-206-0540 University of California - Los Angeles

Understanding the genetic diversity within and between populations has important implications for studies of human disease and evolution. This includes identifying associations between genetic variants and disease, detecting genomic regions that have undergone positive selection and highlighting interesting aspects of human population history.

Now, a team of researchers from the UCLA Henry Samueli School of Engineering and Applied Science, UCLA's Department of Ecology and Evolutionary Biology and Israel's Tel Aviv University has developed an innovative approach to the study of genetic diversity called spatial ancestry analysis (SPA), which allows for the modeling of genetic variation in two- or three-dimensional space.

Their study is published online this week in the journal Nature Genetics.

With SPA, researchers can model the spatial distribution of each genetic variant by assigning a genetic variant's frequency as a continuous function in geographic space. By doing this, they show that the explicit modeling of the genetic variant frequency the proportion of individuals who carry a specific variant allows individuals to be localized on a world map on the basis of their genetic information alone.

"If we know from where each individual in our study originated, what we observe is that some variation is more common in one part of the world and less common in another part of the world," said Eleazar Eskin, an associate professor of computer science at UCLA Engineering. "How common these variants are in a specific location changes gradually as the location changes.

"In this study, we think of the frequency of variation as being defined by a specific location. This gives us a different way to think about populations, which are usually thought of as being discrete. Instead, we think about the variant frequencies changing in different locations. If you think about a person's ancestry, it is no longer about being from a specific population but instead, each person's ancestry is defined by the location they're from. Now ancestry is a continuum."

The team reports the development of a simple probabilistic model for the spatial structure of genetic variation, with which they model how the frequency of each genetic variant changes as a function of the location of the individual in geographic space (where the gene frequency is actually a function of the x and y coordinates of an individual on a map).

"If the location of an individual is unknown, our model can actually infer geographic origins for each individual using only their genetic data with surprising accuracy," said Wen-Yun Yang, a UCLA computer science graduate student.

See original here:
Researchers develop new genetic method to pinpoint individuals' geographic origin

(CNN) -

Getting personalized genetic tests that can pinpoint your risk of developing a number of diseases like cancer, diabetes, Alzheimer's or heart disease are not yet "ready for prime time" according to a new recommendation Tuesday from the American College of Obstetricians and Gynecologists. ACOG says while these tests could be important tools down the road, right now, they should only be used in a clinical trial setting, where experts can put the information into a proper context.

The College published their opinion "Personalized Genomic Testing for Disease Risk" in the June issue of Obstetrics & Gynecology. The advocacy group says the lack of rigorous scientific evidence that the tests are valuable and improve clinical care was the basis for the opinion.

Experts are concerned that a genetic test could tell a patient they have no markers for colon cancer, which could lead someone to get the false impression that they won't get the disease and possibly forego colon screening. On the flip side, genetic testing may reveal that a woman has a 1% risk of getting breast cancer, which could get her very freaked out about getting breast cancer, even though her risk may still be minimal compared to other women without the genetic marker.

"All results require careful interpretation since the result will be affected by other factors such as medical or family history. There is also the potential hazard of a misinterpreted or inaccurate test result, " says Dr. Nancy Rose, Chairman of ACOG's Committee on Genetics.

Dr. Melissa Fries, Director of Genetics and Fetal Medicine at MedStar Washington Hospital Center in Washington, DC supports the new directive. "Genetic testing in general has great power if done for specific indications. We do not know yet the value of tests that may measure minor increases or decrease in the development of disease."

Since the human genome was mapped in 2001 the promise of personalized medicine and genetic testing has been one of medicine's holy grails. Hundreds of genetic variations have been linked to diseases like cancer, but few have been the focus of research that has translated into treatments for patients. As for when these kinds of test will be "ready for prime time?" Rose says the timeframe is currently unknown.

In 2008, ACOG announced their position discouraging people from getting the DNA tested by using home genetic tests you can buy on the internet because they were concerned about " the potential harm of misinterpreted or inaccurate results."

However ACOG does support patients get genetic testing for certain diseases like the BRCA 1 & 2 mutation that increases the risk of breast cancer, Cystic Fibrosis, Fragile X syndrome, the most common form of learning disability and cognitive impairment (occurring predominately in boys), and Tay-Sachs disease, a fatal genetic disorder.

Read more from the original source:
Personalized genetic testing not ready

CAMBRIDGE, Mass.--(BUSINESSWIRE)--

- Global studies showed significant and sustained improvements in lung function and other measures of disease among people with a specific genetic mutation -

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion by consensus recommending the approval of KALYDECO (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KALYDECO is the first medicine to treat the underlying cause of CF, a rare, genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. In people with the G551D mutation, KALYDECO helps the defective CFTR protein function more normally. An estimated 1,100 people in Europe have this mutation.

The CHMP opinion was based on positive findings from two global Phase 3 studies in which KALYDECO demonstrated unprecedented improvements in breathing and other measures of disease for people ages 6 and older with this specific genetic mutation. People treated with KALYDECO experienced significant and sustained improvements in lung function, weight gain and certain quality of life measurements compared to those on placebo. In addition, people who took KALYDECO were 55 percent less likely to have pulmonary exacerbations, or periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits, than those who received placebo. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of adverse events associated with KALYDECO were mild to moderate. Adverse events most commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea.

While there has been great progress in cystic fibrosis treatment during the last few decades, we are still only treating the symptoms and complications of the disease, said Stuart Elborn, M.D., KALYDECO investigator and President of the European Cystic Fibrosis Society. KALYDECO is a fundamentally different approach to the way we treat cystic fibrosis because it targets the underlying cause of the disease. In clinical trials, KALYDECO helped people with a specific genetic mutation breathe more easily, gain weight and generally feel better.

The CHMPs positive opinion will now be reviewed by the European Commission, which has the authority to approve medicines for the European Union. The European Commission generally follows the recommendation of the CHMP and typically issues marketing approval within three to four months.

Since 1998, Vertex has been committed to developing new medicines to treat the underlying cause of cystic fibrosis, said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. KALYDECO represents an important achievement in this ongoing effort. We look forward to working with the European Medicines Agency to bring KALYDECO, our first new medicine in Europe, to people with CF as quickly as possible.

KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 35,000 people in Europe and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years but the median age of death remains in the mid-20s. There are more than 1,800 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The absence of working CFTR proteins results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

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Vertex Receives European CHMP Positive Opinion for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying ...

ASTANA, Kazakhstan, May 24, 2012 /PRNewswire/ --Sir Richard Roberts, the eminent British biologist and Nobel Prize laureate, said today European opposition to genetically modified organisms is political rather than scientific in nature.

He also said "personal medicine" based on human genome research holds large-scale promise to improve the health of the world's people on an individualized basis.

Roberts, who won the Nobel in 1993 for his shared discovery of split genes, made his remarks at the Astana Economic Forum, a global conference of scientists, academics, multinational executives and government leaders.

"On a political level, governments must embrace genetically modified organisms (GMOs) and not give way to European prophets of doom, who oppose the use of GMOs for purely political reasons," said Roberts. "It is important to note there is a complete absence of evidence that GMOs can cause any harm. Indeed to any well-informed scientist, traditionally bred plants seem much more likely to be harmful than GMOs."

Roberts predicted growing knowledge of the human genome will yield better medical treatments and diagnostics. "It is just as important that we learn more about the bacteria that colonize our bodies since they are an essential part of what it means to be human," he said.

He also predicated synthetic biology will enable scientists to build novel microorganisms from "scratch."

"Most exciting is the promise of stem cells where the challenge is to understand how they drive their differentiation into all of the other cell types in our bodies," Roberts said. "While I do not advocate prolonging life indefinitely, I am very much in favor of ensuring that as we age, the quality of our life does not diminish."

The annual Astana Economic Forum this year has drawn thousands of participants from more than 80 nations to this rapidly growing Central Asian nation. There has been much focus at the current sessions on the Greek financial crisis and turbulence in the Euro currency, in addition to the broader economic, scientific and international trade issues that are a traditional mainstay at Astana.

Deal making is a big part of both the official and the unofficial agenda at Astana. Multinationals represented include Chevron, Toyota, Nestle, Microsoft, BASF, Total, General Electric.

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Nobelist Speaks Out on Genetic Modification, Synthetic Biology, Stem Cell Research

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Marge Dwyer mhdwyer@hsph.harvard.edu 617-432-8416 Harvard School of Public Health

Boston, MA Harvard School of Public Health (HSPH) researchers have found that detailed knowledge about your genetic makeupthe interplay between genetic variants and other genetic variants, or between genetic variants and environmental risk factorsmay only change your estimated disease prediction risk for three common diseases by a few percentage points, which is typically not enough to make a difference in prevention or treatment plans. It is the first study to revisit claims in previous research that including such information in risk models would eventually help doctors either prevent or treat diseases.

"While identifying a synergistic effect between even a single genetic variant and another risk factor is known to be extremely challenging and requires studies with a very large number of individuals, the benefit of such discovery for risk prediction purpose might be very limited," said lead author Hugues Aschard, research fellow in the Department of Epidemiology.

The study appears online May 24, 2012 and will appear in the June 8, 2012 print issue of The American Journal of Human Genetics.

Scientists have long hoped that using genetic information gleaned from the Human Genome Project and other genetic research could improve disease risk prediction enough to help aid in prevention and treatment. Others have been skeptical that such "personalized medicine" will be of clinical benefit. Still others have argued that there will be benefits in the future, but that current risk prediction algorithms underperform because they don't allow for potential synergistic effectsthe interplay of multiple genetic risk markers and environmental factorsinstead focusing only on individual genetic markers.

Aschard and his co-authors, including senior author Peter Kraft, HSPH associate professor of epidemiology, examined whether disease risk prediction would improve for breast cancer, type 2 diabetes, and rheumatoid arthritis if they included the effect of synergy in their statistical models. But they found no significant effect by doing so. "Statistical models of synergy among genetic markers are not 'game changers' in terms of risk prediction in the general population," said Aschard.

The researchers conducted a simulation study by generating a broad range of possible statistical interactions among common environmental exposures and common genetic risk markers related to each of the three diseases. Then they estimated whether such interactions would significantly boost disease prediction risk when compared with models that didn't include these interactions since, to date, using individual genetic markers in such predictions has provided only modest improvements.

For breast cancer, the researchers considered 15 common genetic variations associated with disease risk and environmental factors such as age of first menstruation, age at first birth, and number of close relatives who developed breast cancer. For type 2 diabetes, they looked at 31 genetic variations along with factors such as obesity, smoking status, physical activity, and family history of the disease. For rheumatoid arthritis, they also included 31 genetic variations, as well as two environmental factors: smoking and breastfeeding.

But, for each of these disease models, researchers calculated that the increase in risk prediction sensitivitywhen considering the potential interplay between various genetic and environmental factorswould only be between 1% and 3% at best.

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Knowing genetic makeup may not significantly improve disease risk prediction

ANN ARBOR, Mich.--(BUSINESS WIRE)--

RetroSense Therapeutics, a gene therapy company dedicated to vision restoration, announced completion of a pre-IND meeting with the Center for Biological Evaluation and Research (CBER) division of the FDA that took place on May 22nd 2012 regarding RetroSenses lead biologic, RST-001 for vision restoration in retinal degenerative conditions.

The purpose of the meeting was to obtain CBERs guidance for the clinical path to a Biologics License Application (BLA) for RST-001 in the US, and clarity on the steps required for Investigational New Drug (IND) submission. The discussions included manufacturing criteria, the scope and design of the preclinical studies, and the scope and design of Phase I and IIa clinical trials.

Sean Ainsworth, CEO, noted,"We were quite encouraged by the FDA's feedback and comments and identified nothing that will hinder our path to the clinic. RetroSense remains on schedule to bring RST-001 to the clinic safely and expeditiously.

Peter Francis, MD, PhD, Clinical Director, led the meeting. Dr. Francis stated, It was a successful, productive interaction with the FDA. We were thankful to have representation by Foundation Fighting Blindness, who have been supportive of our novel approach to vision restoration.

We are very excited about the potential for this innovative treatment to restore vision in people who are blind from retinal degenerations, says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. It is an elegant and powerful approach to overcoming devastating eye diseases.

About RetroSense Therapeutics

RetroSense Therapeutics is a biotechnology company developing a game-changing gene therapy to restore vision in patients suffering from blindness due to retinitis pigmentosa (RP) and advanced dry age-related macular degeneration (advanced dry-AMD). There are currently no FDA approved therapies to improve or restore vision in patients with these retinal degenerative conditions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information about RetroSense, visit http://www.retro-sense.com/.

About Foundation Fighting Blindness

The Foundation Fighting Blindness is a national non-profit organization driving research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect more than 10 million Americans. Since 1971, the Foundation has raised over $450 million as the leading non-governmental funder of retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a variety of retinal degenerative diseases, and proving a cure is in sight. With a network of 50 chapters, the Foundation also provides support, education and resources to affected individuals and their families in communities across the country.

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RetroSense Therapeutics Completes pre-IND Meeting for RST-001

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Sarah Jackson press_releases@the-jci.org Journal of Clinical Investigation

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response. A recent clinical trial indicated that gene therapy to insert the correct ADA gene in the patient's own bone marrow cells can also lead to a good response.

However, patients were noted to have defects in B cell tolerance, meaning that some B cells that react to antigens from the body fail to be eliminated, leading to an autoimmune response. Dr. Eric Meffre and colleages at Yale University in New Haven, Connecticut and Alessandro Aiuti in Milan, Italy joined together to better understand why patients developed B cell tolerance problems. They found that loss of the ADA gene directly contributes to B cell tolerance problems and that these defects are mostly corrected after gene therapy. Their results point to a previously unknown role for ADA in B cell response and support the use of gene therapy as an effective treatment option for ADA-deficient severe combined immunodeficiency patients.

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TITLE:

Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy

AUTHOR CONTACT:

Eric Meffre Yale University School of Medicine, New Haven, CT, USA Phone: 1-203-737-4535; Fax: 1-203-785-7903; E-mail: eric.meffre@yale.edu

ACCOMPANYING COMMENTARY

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Gene therapy can correct forms of severe combined immunodeficiency

THE WOODLANDS, Texas--(BUSINESS WIRE)--

Opexa Therapeutics, Inc. (NASDAQ:OPXA - News), a biotechnology company developing a novel T-cell therapy for multiple sclerosis (MS), announced today that the Company is rebranding its leading MS therapy with the new name TcelnaTM. The product, previously known as Tovaxin, will now be known as Tcelna as the company positions itself towards the treatment of patients with Secondary Progressive MS (SPMS).

"Opexa has worked diligently in the optimization of its overall manufacturing process and clinical development program while concentrating its efforts in the SPMS indication. The rebranding of our lead product as Tcelna encompasses these advancements and our continued dedication to make a difference in the treatment of MS," commented Neil K. Warma, President and Chief Executive Officer of Opexa.

The name Tcelna (pronounced Te-SELL-nuh) reflects the T-cell derivation of the product. Opexa has requested a registered trademark for the new brand name.

About Opexa

Opexa Therapeutics, Inc. is dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS). The Companys leading therapy, TcelnaTM, a personalized cellular immunotherapy treatment, is in clinical development targeting both Secondary Progressive and Relapsing Remitting MS. Tcelna is derived from T-cells isolated from peripheral blood, expanded ex vivo and reintroduced into the patients via subcutaneous injections. This process triggers a potent immune response against specific subsets of autoreactive T-cells known to attack myelin and, thereby, reduces the risk of relapse over time.

For more information, visit the Companys website at http://www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words expects, believes, anticipates, estimates, may, could, intends, and similar expressions are intended to identify forward-looking statements. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding the development of the Companys product candidate, Tcelna, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: our capital position, the ability of the Company to enter into and benefit from a partnering arrangement for the Company's product candidate, Tcelna, on reasonably satisfactory terms (if at all), our dependence (if partnered) on the resources and abilities of any partner for the further development of Tcelna, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs and to undertake and complete any further clinical studies for Tcelna, the success of our clinical trials, the efficacy of Tcelna for any particular indication, such as Relapsing Remitting MS or Secondary Progressive MS, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights (including for Tcelna), the risk of litigation regarding our intellectual property rights, the success of third party development and commercialization efforts with respect to products covered by intellectual property rights that the Company may license or transfer, our limited manufacturing capabilities, our dependence on third-party manufacturers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date made. We assume no obligation or undertaking to update any forward-looking statements to reflect any changes in expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based. You should, however, review additional disclosures we make in our reports filed with the Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2011.

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Opexa Therapeutics Announces TcelnaTM as New Brand Name for MS Therapy

Indian clinic's stem cell therapy real?

STORY HIGHLIGHTS

For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.

New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

That's when Cash began walking with the aid of braces for the first time.

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Family hangs hope on stem cells

DEERFIELD, Ill.--(BUSINESS WIRE)--

CHDI Foundation, Inc. and Lundbeck today announced a research collaboration to investigate a targeted therapy for Huntingtons disease (HD). Currently, no treatment exists to slow or halt the progression of HD,1 a challenging hereditary neurodegenerative disease characterized by a triad of behavioral, cognitive, and motor symptoms.2

As part of the collaboration CHDI will conduct pre-clinical studies on a Lundbeck investigative compound. Research will focus on the compounds effect on P2X receptors that may be involved in HD.3 The study results will influence future research into this and other compounds for HD.

One of our important functions at CHDI is to seek out promising research ideas in the HD field, and this includes this interesting new work on Lundbecks compound. Lundbeck is well established in central nervous system drug research and development, and were looking forward to tapping their expertise, noted Ignacio Munoz-Sanjuan, Vice President, Translational Biology at CHDI. Lundbeck has a proven track record of not only bringing new therapies to market but also working to support the needs of their patient communities. We hope this research collaboration provides a stepping stone for future therapies that slow the progression of HD.

CHDI is a privately-funded, not-for-profit biomedical research organization that works with an international network of scientists to discover and develop therapies for HD. The organization actively enables HD research by collaborating with research organizations and pharmaceutical companies conducting promising research, often providing financial support. CHDI activities include exploratory biology, clinical studies and trials, and educational workshops. Cooperation is key to finding therapies for HD, which is why CHDI works with a variety of researchers within the HD research community.

We look forward to working with this distinguished group of scientists who share our dedication to the HD community, said Staffan Schberg, president of Lundbeck. CHDI is a beacon of hope for the HD community and devotes itself entirely to finding therapies that have the potential to improve the lives of HD families. Given our shared commitment, we are thrilled to partner with them and hope that our research will lead to advancements in HD therapeutic options. We are also pleased that this announcement coincides with HD Awareness Month to help draw attention to the need to find therapies for this degenerative neurological disease.

This collaboration is part of Lundbecks continued commitment to its HD Research Initiative, launched in 2010 to identify and ultimately commercialize therapies that may slow or halt the progression of the disease. The initiative is driven by collaborations with academic institutions, research organizations and companies that share Lundbecks ongoing commitment to the HD community. In 2011, Lundbeck and the University of Massachusetts Medical School began to investigate RNAi-based therapies to suppress the production of mutant huntingtin (mHtt), the abnormal protein that causes HD. Those conducting early-stage HD research and interested in exploring opportunities to collaborate with Lundbeck should send an email to HDresearch@lundbeck.com.

About Huntingtons Disease

Huntingtons disease is a hereditary neurodegenerative disease characterized by a triad of progressive behavioral, cognitive, and motor symptoms2 that vary from person to person. The survival time after the onset of symptoms can range from 10 to 30 years.1 The HD gene, whose mutation results in the disease, was localized in 1983 and isolated in 1993.4,5 For more information on HD, please visit HDBuzz (hdbuzz.net) or the Hereditary Disease Foundation (www.hdfoundation.org).

About CHDI Foundation, Inc.

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Lundbeck and CHDI Foundation Announce Research Collaboration to Investigate Candidate Therapy for Huntington’s Disease

20-05-2012 13:17 WATCH THE LATEST VIDEO THAT CAME OUT TODAY HERE FAIR USE NOTICE: These Videos may contain copyrighted (©) material the use of which has not always been specifically authorized by the copyright owner. Such material is made available to advance understanding of ecological, political, human rights, economic, democracy, scientific, moral, ethical, and social justice issues, etc. It is believed that this constitutes a 'fair use' of any such copyrighted material as provided for in section 107 of the US Copyright Law. In accordance with Title 17 USC Section 107, this material is distributed without profit to those who have expressed a prior general interest in receiving similar information for research and educational purposes. Date: 11-15-09 Host: George Knapp Guests: Bob Lazar, Gene Huff, John Lear George Knapp was joined by the legendary and elusive Bob Lazar, along with Gene Huff and John Lear, for a discussion on the 20th anniversary of the program UFOs: The Best Evidence which first told the story of Area 51. Appearing alone in the first hour, Lazar provided an update on his research into hydrogen power. He detailed his invention of a hydrogen fuel cell which stores enough energy to power a car up to 400 miles. While he expressed some concern about having this technology thwarted by the government, Lazar praised the state of Michigan, where he now lives, for being extremely supportive of his research. Beginning in the 2nd hour, John ...

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Area 51 Revisited Main Show Only - secretsocietiestoday - Video

21-05-2012 05:09 Keynote 3: Margaret Lock, McGill University Genes as tools for the prevention of Alzheimer's disease A growing panic exists about the approaching "tsunami" of Alzheimer's disease and its potential impact on the global economy. This concern is heightened by a failure to develop drugs to cure AD, despite billions of dollars worth of research. In response to this situation, Alzheimer experts have recently published position pieces involving a move to the prevention of AD that will make use of biomarkers to predict who among us are at risk. Among the several biomarkers for AD being routinely used in research settings is the susceptibility gene, ApoE?4. Following a brief discussion of debates among AD experts about the concept of AD and the entanglement of aging and dementia, this talk will consider the social and ethical impact of biomarker testing, including genetic testing, with the prime objective of developing drugs to prevent AD.

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Genomics in Society: Facts, Fictions and Cultures: Margaret Lock Keynote - Video

Cancer cells. The research team developed an innovative cancer-fighting strategy of suppressing the enzyme produced by the PAD4 gene. Credit: National Cancer Institute

(Medical Xpress) -- A team of scientists has developed a promising new strategy for "reactivating" genes that cause cancer tumors to shrink and die. The researchers hope that their discovery will aid in the development of an innovative anti-cancer drug that effectively targets unhealthy, cancerous tissue without damaging healthy, non-cancerous tissue and vital organs. The research will be published in the Journal of Biological Chemistry.

The team, led by Yanming Wang, a Penn State University associate professor of biochemistry and molecular biology, and Gong Chen, a Penn State assistant professor of chemistry, developed the new strategy after years of earlier research on a gene called PAD4 (peptidylarginine deiminase 4), which produces the PAD4 enzyme. Previous research by Wang and other scientists revealed that the PAD4 enzyme plays an important role in protecting the body from infection. The scientists compared normal mice with a functioning PAD4 gene to other mice that had a defective a PAD4 gene. When infected with bacteria, cells from the normal mice attacked and killed about 30 percent of the harmful bacteria, while cells from the defective mice battled a mere 10 percent. The researchers discovered that cells with a functioning PAD4 enzyme are able to build around themselves a protective, bacteria-killing web that Wang and his colleagues dubbed a NET (neutrophil extracellular trap). This NET is especially effective at fighting off flesh-eating bacteria.

Now, in their new study, Wang and his collaborators have focused on the less-desirable effects of the same PAD4 gene. While PAD4 is clearly a critical part of the body's defense strategy, the gene's over-expression may be linked to autoimmune diseases such as rheumatoid arthritis and multiple sclerosis. One situation in which the PAD4 enzyme is markedly increased is in patients with certain cancers, such as breast, lung, and bone cancers. "We know that the PAD4 gene acts to silence tumor-suppressor genes," said Wang. "So we theorized that by inhibiting the enzyme that this gene produces, the 'good guys' -- the tumor-suppressor genes -- would do a better job at destroying cancerous tissue and allowing the body to heal."

To test their theory, Wang and his colleagues treated mice that had cancerous tumors with a molecule to inhibit the PAD4 enzyme. They found that, especially when combined with additional enzyme inhibitors, the treatment worked as effectively as the most-commonly-used chemotherapy drug, doxorubicin, which shrinks tumors by about 70 percent.

Most striking, however, was that the PAD4 enzyme-inhibition strategy caused significantly less damage to healthy tissues. "Current chemotherapy drugs such as doxorubicin don't attack just tumors; unfortunately, they also attack healthy areas of the body," Wang explained. "That's why chemotherapy patients experience such terrible side effects such as weight loss, nausea, and hair loss. Because the PAD4 treatment appears to be less toxic, it could be an excellent alternative to current chemotherapy treatments."

Wang also explained that the PAD4 gene's dual personality -- on the one hand a helpful defense against bacteria, while on the other, a harmful silencer of cancer-suppressor genes -- can be understood from the perspectives of evolution and longer life spans. "Our ancestors didn't have antibiotics, so a bacterial infection could easily result in death, especially in young children," Wang explained. "So, back then, an overactive PAD4 gene was advantageous because the NET bacteria-trapping mechanism was the body's major defense against infection." Wang also explained that on the other hand, because people today have access to antibiotics, we live much longer than our ancestors did. "PAD4's bad effects -- cancer and autoimmune diseases -- tend to be illnesses that appear later in life," Wang said. "So nowadays, an overactive PAD4 gene, while still protective against bacteria, can be detrimental later in life."

This research was funded by the National Cancer Institute of the National Institutes of Health and a Penn State Clinical and Translational Science Institute Pilot Grant Award to Wang and Chen. In addition to Wang and Chen, other researchers who contributed to this project include Yuji Wang, Pingxin Li, Shu Wang, Jing Hu, Megan Fisher, Kira Oshaben, Jianhui Wu, Na Zhao, Ying Gu of Penn State's Center for Eukaryotic Gene Regulation and the Department of Biochemistry and Molecular Biology.

Journal reference: Journal of Biological Chemistry

Provided by Pennsylvania State University

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Strategy discovered to activate genes that suppress tumors and inhibit cancer