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New CEO for tilapia genetics firm – Fish Update

June 6th, 2017 11:48 pm

GENOMAR Genetics, which specialises in the tilapia industry, has appointed Alejandro Tola Alvarez as its CEO.

Alvarez (pictured), who took up his new role on June 1, will be responsible for innovation, operations and business development within the company, which is part of the EW Group.

He hasbeen part of the Genomar group since 2006, based in South-East Asia as chief operational officer and in Norway as chief technical officer.

We were very pleased to find a highly qualified internal candidate for the CEO position, said chairman Odd Magne Rdseth.

Alejandro has played a major role in both R&D and commercial development of the most reputable and professional genetic brands in global tilapia aquaculture.

He comes with a deep understanding of the tilapia operating environments and the opportunities of modern breeding technologies, such as genomics, to improve economic and environmental performance of the industry.

Alvarez is a qualified vet and has masters degrees in aquaculture and business administration.

GenoMar Genetics, based in Oslo with its main operation in Luzon, Philippines, has developed the Genomar Supreme Tilapia strain (GST) through more than 25 years of selective breeding.

The company was part of the Norway Fresh Group until March 2017 when EW Group concluded an agreement to acquire 100 per cent of GenoMar Genetics shares.

EW Group, based in Visbek, Germany, is a family owned holding company with more than 120 subsidiaries in over 30 countries.

The core business of the group, which has 9,000 employees worldwide, is animal breeding, animal nutrition and animal health.

Over the past 10 years, the group has expanded into the aquaculture sector and includes companies such as AquaGen, Aquabel, GenoMar Genetics and Vaxxinova.

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Why Herbalife, JinkoSolar, and NewLink Genetics Slumped Today … – Motley Fool

June 6th, 2017 11:48 pm

The stock market closed Monday with modest losses, sending the Dow, S&P 500, and Nasdaq Composite lower from their record closes last Friday. Nevertheless, the declines were all less than 0.2%, and investors appeared to be in a wait-and-see mode as they look forward to more momentous news due out later this month. Among the top potential market movers for June will be the U.K. elections later this week and the Federal Reserve's meeting to determine the future course of interest rates. Yet company-specific items were in the spotlight today, and some stocks posted significant losses. Herbalife (NYSE:HLF), JinkoSolar (NYSE:JKS), and NewLink Genetics (NASDAQ:NLNK) were among the worst performers on the day. Below, we'll look more closely at these stocks to tell you why they did so poorly.

Shares of Herbalife dropped nearly 7% after the company said this morning that it would have to revise its financial expectations. Citing the need for its distributors to learn, teach, and implement new technology and processes, Herbalife said that it now expects net sales to fall 2% to 6% in the second quarter, with volume falling 4% to 8%. For the full year, Herbalife thinks it will be able to grow revenue 0.5% to 3.5% despite seeing volume come in a range between -1% and +2%. Upward adjustments to earnings guidance weren't enough to satisfy shareholders, and activist investor Bill Ackman spoke out against the company's news. Herbalife is a controversial company, but when negative things that get said about the business pan out in its financials, the seller of nutritional supplements and other consumer products needs to take steps to remedy the situation.

Image source: Herbalife.

JinkoSolar stock fell 8% in the wake of the company's first-quarter financial results. The Chinese solar company said that solar shipments jumped by nearly 30% from year-ago levels, topping the 2-gigawatt mark. Revenue was also up by double-digit percentages, but adjusted net income was down sharply, falling more than 80%. CEO Kangping Chen said that falling selling prices of solar modules led to gross margin contraction, which in turn resulted in bottom-line weakness. Chen remained optimistic about JinkoSolar's prospects for the remainder of the year, but investors didn't seem as confident that the company would be able to improve margins and capitalize on building demand in China. With JinkoSolar having been involved in big projects in the Persian Gulf region, it's possible that diplomatic tensions in the area also weighed on the stock.

Finally, shares of NewLink Genetics finished down 12%. The company said over the weekend that a phase 2 study of its breast cancer candidate treatment indoximod in combination with taxane chemotherapy failed to reach its intended endpoints. In particular, NewLink was trying to establish a statistically significant difference as to progression-free survival, overall survival, and objective response rate. Without achieving those goals, investors aren't certain what the next step forward is for NewLink. Still, with other studies having shown more encouraging results, NewLink might still end up being a potential takeover target from larger players in the biotech space.

Dan Caplinger has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.

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Oxford Genetics gets 500000 from Mercia Technologies – Tech City News

June 6th, 2017 11:48 pm

BioTech firm Oxford Genetics has raised 500,000 from Mercia Technologies.

The news comes after the company, which specialises in synthetic biology and DNA design, raised 1m from Mercia, which has a direct equity stake of 47.9% in the firm in October last year.

Oxford Genetics has so far raised 5.8m through a combination of grants and external investments and says it will use this latest round to expand its reach in the US market and further its growth.

Oxfords AI firm Oxbotica gets 8.6m to lead driverless car consortium

Dr Ryan Cawood, CEO of Oxford Genetics, commented on the raise: Mercias continued support has been instrumental in helping us to achieve the significant progress to date.

Our turnover has doubled in the last year and with this additional capital, we will be able to further expand the team, giving us the ability to build the most innovative technologies in the DNA and protein design market.

Dr Mark Payton, CEO of Mercia Technologies PLC, spoke about the companys trajectory over the past year.

Oxford Genetics has clearly demonstrated its ability to create market leading technologies and has been bolstered by an industry leading research and development team.

Payton went on to note that life sciences and bio-sciences continued to be a key sector for Mercia and one which they believe would deliver significant shareholder value over the medium term.

Follow Yessi Bello Perez on Twitter @yessibelloperez

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What’s In Your Genes? – Pacific Northwest Inlander

June 6th, 2017 11:47 pm

Picture a time in the not-too-distant future when whole genome sequencing is routine. A time when, before babies even learn to talk, their parents will have the ability to learn what the future may have in store for their offspring: Is their little girl predisposed to getting breast cancer? Will their happy-go-lucky son one day develop Alzheimer's?

"There is no doubt in my mind that, in addition to going in and having blood chemistry done, you're gonna have DNA sequencing done, too. It will be there at some point," says Nicholas Schork, a quantitative geneticist at the J. Craig Venter Institute in La Jolla, California, who has studied genomic medicine for more than three decades. "We can debate about the timeline, but it'll become routine."

The hope is that genetic testing will make health care more effective by allowing doctors and patients to focus on areas that need attention the patient's genetic "vulnerabilities." At the same time, patients may learn of areas where they won't need to be quite as vigilant. And treatments could, in turn, be perfectly tailored to a patient's specific needs.

But as with any significant and broadly applicable medical advance, there are questions. For example, should patients learn that they carry markers for currently incurable genetic diseases, or that they are at high risk for developing a condition like Alzheimer's, which has no effective treatment? And just who owns all that genetic data? Who will have access to it?

Even with important questions left unanswered, health educators are moving forward to take advantage of the promises genetic testing offers. Washington State University's new Elson S. Floyd College of Medicine has announced it is partnering with Arivale, a Seattle-based company that conducts whole genome sequencing, to help complete a portrait of a person a "portrait" that can be used to promote wellness over that individual's entire lifespan. Every member of the school's inaugural class will have the opportunity to undergo testing, which will also include blood tests and a lifestyle evaluation. Then, over the next year, Arivale's team of nurses and dietitians will provide individually tailored follow-up, based on each individual's risks and goals. It's a unique partnership, made possible in large part because the medical school is new, with its first class of students starting in 2017.

Allowing the medical students to experience genetic testing firsthand is just part of the goal. "We need physicians that understand it well enough that they can make it better going forward," says John Tomkowiak, founding dean of WSU's College of Medicine. "That's where our students are going to be uniquely positioned."

WHAT GENES TELL US

Genetic testing already provides important information about a person's health or their heritage. Hospitals screen newborn babies for certain genetic disorders, and in some cases, tests can detect disorders before birth. And diagnostic testing can confirm, or rule out, many disorders in adults.

Testing doesn't have to be ordered by a physician. For $200, you can provide a saliva sample, mail it back to 23andMe.com and find out not only your ancestry, but also your risks for a number of diseases, including Alzheimer's and Parkinson's. Ancestry.com offers a glimpse into your heritage for $99. Color.com claims to reveal your risk for the most common hereditary cancers, and even offers "complimentary genetic counseling" for a $249 fee.

But if genetic testing is to revolutionize the health care industry, as many have promised, there's still a ways to go. "The technology is at the beginning stages," says Thomas May, a faculty researcher for the HudsonAlpha Institute for Biotechnology.

Companies like 23andMe offer genetic tests that may provide information about some genetic disorders from currently known genetic variants. But whole genome sequencing is different; it will reveal all your individual genetic variants.

How valuable is that information? There are a relatively small number of conditions that researchers are confident result from a specific genetic variant, May says. For example, there is one variant that researchers have found is associated with an increased risk of developing breast or ovarian cancer. A genetic test that shows an increased risk for breast cancer is considered an "actionable" outcome, meaning there are things you can do to prevent the outcome, like beginning mammograms earlier. Though there are more than 50 actionable outcomes like that, it's still a relatively small number.

Adding to the confusion is the fact that not everyone who develops breast cancer actually has the genetic variant in fact, May says only about 10 percent do. So even if testing shows that you don't have the "breast cancer gene," that doesn't mean it's OK to stop getting mammograms.

"Most variants and correlations are of that type: We can't say for certain if you're gonna get a disease," May says.

Doctors are mixed about whether genetic testing is currently having a real impact on patients. In a May survey conducted by the Medscape Physician Oncology Report on Genomics Testing, 71 percent of oncologists surveyed felt that genetic testing was either "very" or "extremely" important to the oncology field. At the same time, 61 percent said that, currently, fewer than a quarter of their patients would actually benefit from genetic testing.

The number of diseases with "actionable" outcomes will inevitably grow, as more people are tested and more data becomes available. But this leaves deeper questions, says Schork, the quantitative geneticist. A company or health care provider would likely give patients information about diseases that can be prevented or cured. If someone is predisposed to obesity, for instance, then he or she can elect to receive targeted care to reduce that risk.

But what about diseases that, right now, are incurable?

Take Huntington's disease, a genetic disorder that breaks down nerve cells in the brain. It's rare, but it's a "hideous way to die," Schork says. A person can be screened at the age of 25 and be found to carry the Huntington's gene, but there's debate about whether or not that information should be shared with a client or not. The same goes for genetic variants related to Alzheimer's disease.

"If there's nothing they can do about it, then there's a concern about whether or not that information should be imparted," Schork says.

When the Food and Drug Administration ordered 23andMe to stop telling customers their odds of contracting diseases in 2013, Harvard Medical School genetics professor Robert Green and Laura Beskow, a professor at Duke University's Institute for Genome Sciences and Policy, argued against the FDA. They cited a number of studies showing that direct-to-consumer genetic testing does not cause a large percentage of customers despair. In an interview with the New York Times in April, Green said the potential for distress based on results of a genetic test for Alzheimer's was "much smaller than anticipated."

Another question: Who really owns the DNA data that is being collected from willing users of genetic testing? Consider Myriad, a company that offers genetic testing both to help determine cancer risk and design better treatment plans for patients who already have cancer. The company has something that "others do not," Schork says: insight into which genetic variants predispose women to breast cancer.

What Myriad is really selling, then, is not the genetic test itself, but access to insights it has gained through mining its database, insights that can be leveraged into whatever level of payment the company decides to charge.

It's potentially critical information that could help save a life, and some argue that the data should be in the public domain not held by a private company.

"There have been huge debates about whether the community should challenge the monopoly that Myriad has," Schork says. "There are many groups out there that would like to counteract the monopoly Myriad has, by building public domain data sets."

JUST ONE TOOL

"Genetic testing is not a blueprint. It's really not," says Jennifer Lovejoy, chief translational science officer for Arivale. "Genes are really just one factor the environment, diet, exercise, pollutants and even emotional state have a big impact on genes."

That's why Arivale not only collects genetic information on each client, but also evaluates various blood tests and lifestyle factors to create a "dense data cloud" of information about a patient.

"That is the grand vision: that everybody would have these dense, dynamic data clouds, and understand the choices that will be optimal to optimize wellness and avoid disease," says Lovejoy.

Arivale touts the success stories among its nearly 2,000 clients. One client found out he had a gene associated with high sensitivity to saturated fat, giving him a better indication of an appropriate diet that helped him lose weight. Another client discovered that his genes may have an impact on his cholesterol. Another learned he was at risk of developing diabetes.

Ideally, this type of preventive care will soon be covered by insurance, Lovejoy says. The thinking is that preventing disease will bring down the cost of health care overall, making insurers likely to cover more preventive care, "but we have to prove it," Lovejoy says. Researchers are conducting studies and trials to do just that, and if they can prove it, then genetic testing could soon be routine in health care.

"If you think about what health care should mean, it should mean, one, the ability to deal with disease and that's what everyone does today," Arivale co-founder Leroy Hood said at a press conference in April announcing the company's partnership with WSU. "But two, it should mean the ability to optimize wellness for each individual. That is, improving their health and/or letting them avoid disease." That's a concept Hood calls "scientific wellness, and he thinks it could lead to "a whole new health care industry in the future."

Tomkowiak, of WSU's College of Medicine, agrees: "The concept of scientific wellness has the potential to disrupt the entire industry by shifting the cost curve, by keeping people healthier and reducing the cost of health care overall."

Regardless of whether or not Arivale becomes an industry leader, Tomkowiak believes that the practice of medicine will be fundamentally altered in the near future.

"We absolutely believe that seven years from now, the practice of scientific medicine and scientific wellness will be common," he says. "Instead of being behind the curve, we want... to be leading this effort."

For about $3,500, clients can sign up for Arivale's program. The fee includes whole genome sequencing, which is also available from other sources. So how do Arivale clients achieve "scientific wellness"? Here are the elements of their program:

Welcome package: Clients get a welcome package with a Fitbit to track sleep, activity and heart rate. The package asks for information to help understand a client's bacteria in their gut, and asks for a sample of saliva to measure a person's stress level.

Online test: Clients take a series of online assessments about their goals, health history, lifestyle, stress, personality and happiness.

Call from coach: You'll talk to a coach who will get to know what you want to accomplish and give you a personalized action plan.

Labs: You'll take blood tests so your coach can understand your current health. While you're there, they'll take your vital signs.

A picture emerges: The various test create a picture of you, which an Arivale coach will use to provide a step-by-step plan to "optimize your wellness," according to the company.

Follow-up: You're not done yet. You'll be contacted by your coach regularly to review your action plan, and Arivale will provide reports on how you're progressing. Every six months, you'll complete another set of clinical labs.

Source: arivale.com/your-journey

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New cancer medicine targets rare genetic flaw – Press TV

June 6th, 2017 11:47 pm

This file photo shows doctors at Memorial Sloan Kettering Cancer Center in New York City.

An experimental cancer medicine called larotrectinib has shown promise treating a diverse range of cancers in people young and old, researchers said at a major cancer conference in the United States.

The treatment targets a genetic abnormality which is often found in rare cancers - including salivary gland cancer, juvenile breast cancer, and a soft tissue cancer known as infantile fibrosarcoma - which are particularly difficult to treat.

This abnormality also occurs in about 0.5 percent to one percent of many common cancers.

In the study released at the American Society of Clinical Oncology conference, 76 percent of cancer patients - both children and adults with 17 different kinds of cancer - responded well to the medicine.

A total of 79 percent were alive after one year. The study is ongoing.

Twelve percent went into complete remission from their cancer.

The clinical trial included 55 patients - 43 adults and 12 children. All had advanced cancers in various organs, including the colon, pancreas and lung, as well as melanoma.

"These findings embody the original promise of precision oncology: treating a patient based on the type of mutation, regardless of where the cancer originated," said lead study author David Hyman, chief of early drug development at Memorial Sloan Kettering Cancer Center in New York.

"We believe that the dramatic response of tumors with TRK fusions to larotrectinib supports widespread genetic testing in patients with advanced cancer to see if they have this abnormality."

Made by Loxo Oncology Inc., larotrectinib is a selective inhibitor of tropomyosin receptor kinase (TRK) fusion proteins.

TRK proteins are a product of a genetic abnormality when a TRK gene in a cancer cell fuses with one of many other genes, researchers said.

The US Food and Drug Administration has not yet approved the treatment for widespread use.

The treatment was well tolerated by patients, and the most common side effects were fatigue and mild dizziness.

"If approved, larotrectinib could become the first therapy of any kind to be developed and approved simultaneously in adults and children, and the first targeted therapy to be indicated for a molecular definition of cancer that spans all traditionally-defined types of tumors," said Hyman.

(Source:AFP)

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The Future of Medicine Depends on Protections for Pre-Existing Conditions – Pacific Standard

June 6th, 2017 11:47 pm

Pacific Standard
The Future of Medicine Depends on Protections for Pre-Existing Conditions
Pacific Standard
Biomedical researchers can see a future where genetic tests are used to treat and prevent many diseases before major symptoms even present themselves. But that future won't be possible without strong insurance protections for pre-existing conditions.

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Drug Helps Fight Breast Tumors Tied to ‘Cancer Genes’ – Sioux City Journal

June 6th, 2017 11:47 pm

SUNDAY, June 4, 2017 (HealthDay News) -- A twice-daily pill could help some advanced breast cancer patients avoid or delay follow-up sessions of chemotherapy, a new clinical trial reports.

The drug olaparib (Lynparza) reduced the chances of cancer progression by about 42 percent in women with breast cancer linked to BRCA1 and BRCA2 gene mutations, according to the study.

Olaparib delayed cancer progression by about three months. The drug also caused tumors to shrink in three out of five patients who received the medication, the researchers reported.

"Clearly the drug was more effective than traditional chemotherapy," said Dr. Len Lichtenfeld, deputy chief medical officer for the American Cancer Society.

"This is a group where a response is more difficult to obtain -- a young group with a more aggressive form of cancer -- and nonetheless we saw a close to 60 percent objective response rate," he said.

The study was funded by AstraZeneca, the maker of Lynparza.

Olaparib works by cutting off the avenues that malignant cancer cells use to stay alive, said lead researcher Dr. Mark Robson. He's a medical oncologist and clinic director of Clinical Genetics Service at Memorial Sloan Kettering Cancer Center in New York City.

The drug inhibits PARP, an enzyme that helps cells repair damaged DNA, Robson said.

Normal cells denied access to PARP will turn to the BRCA genes for help, since they also support the repair of damaged DNA, Robson said.

But that "backup capability" is not available to breast cancer cells in women with BRCA gene mutations, Robson said.

"When you inhibit PARP, the cell can't rescue itself," Robson said. "In theory, you should have a very targeted approach, one specifically directed at the cancers in people who have this particular inherited predisposition."

Olaparib already has been approved by the U.S. Food and Drug Administration for use in women with BRCA-related ovarian cancer. Robson and his colleagues figured that it also should be helpful in treating women with breast cancer linked to this genetic mutation.

The study included 302 patients who had breast cancer that had spread to other areas of their body (metastatic breast cancer). All of the women had an inherited BRCA mutation.

They were randomly assigned to either take olaparib twice a day or receive standard chemotherapy. All of the patients had received as many as two prior rounds of chemotherapy for their breast cancer. Women who had hormone receptor-positive cancer also had been given hormone therapy.

After 14 months of treatment, on average, people taking olaparib had a 42 percent lower risk of having their cancer progress compared with those who received another round of chemotherapy, Robson said.

The average time of cancer progression was about seven months with olaparib compared with 4.2 months with chemotherapy.

Tumors also shrank in about 60 percent of patients given olaparib. That compared with a 29 percent reduction for those on chemotherapy, the researchers said.

Severe side effects also were less common with olaparib. The drug's side effects bothered 37 percent of patients compared with half of those on chemo. The drug's most common side effects were nausea and anemia.

"There were fewer patients who discontinued treatment because of toxicity compared to those who received chemotherapy," Robson said. "Generally it was pretty well tolerated."

Only about 3 percent of breast cancers occur in people with BRCA1 and BRCA2 mutations, the researchers said in background notes.

Despite this, the results are "quite exciting," said Dr. Julie Fasano, an assistant professor of hematology and medical oncology at the Icahn School of Medicine at Mount Sinai in New York City.

Olaparib could wind up being used early in the treatment of metastatic breast cancer as an alternative to chemotherapy, and future studies might find that the drug is effective against other forms of breast cancer, Fasano said.

"It may be a practice-changing study, in terms of being able to postpone IV chemotherapy and its associated side effects" like hair loss and low white blood cell counts, Fasano said.

Lichtenfeld noted that olaparib also places less burden on patients.

"It may be easier for women to take two pills a day rather than go in for regular chemotherapy," Lichtenfeld said. "Clearly, this is a treatment that will garner considerable interest.

The findings were scheduled to be presented Sunday at the American Society of Clinical Oncology's annual meeting, in Chicago. The study was also published June 4 in the New England Journal of Medicine.

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Scientists are finding more genes linked to IQ. This doesn’t mean we can predict intelligence. – Vox

June 6th, 2017 11:47 pm

Last month, researchers announced some astonishing findings in Nature Genetics: Theyd found 40 genes that play a role in shaping human intelligence, bringing the total number of known intelligence genes up to 52.

This study was a big deal because while weve known intelligence is largely heritable, we havent understood the specifics of the biology of IQ why it can be so different between people, and why we can lose it near the end of life.

The Nature Genetics study was a key early step toward understanding this, hailed as an enormous success in the New York Times.

And there are many more insights like this to come. The researchers used a design called a genome-wide association study. In it, computers comb through enormous data sets of human genomes to find variations among them that point to disease or traits like intelligence. As more people have their genomes sequenced, and as computers become more sophisticated at seeking out patterns in data, these types of studies will proliferate.

But theres also a deep uneasiness at the heart of this research it is easily misused by people who want to make claims about racial superiority and differences between groups. Such concerns prompted Nature to run an editorial stressing that the new science of genetics and intelligence comes to no such conclusions. Environment is crucial, too, Nature emphasized. The existence of genes for intelligence would not imply that education is wasted on people without those genes. Geneticists burned down that straw man long ago.

Also, nothing in this work suggests there are genetic difference in intelligence when comparing people of different ancestries. If anything, it suggests that the genetics that give rise to IQ are more subtle and intricate than we can ever really understand.

Were going to keep getting better at mapping the genes that make us smart, make us sick, or even make us lose our hair. But old fears and myths about genetics and determinism will rear their heads. So will fears about mapping ideal human genes that will lead to designer babies, where parents can pick traits for their children la carte.

To walk through the science, and to bust its myths, I spoke to Danielle Posthuma, a statistical geneticist at Vrije Universiteit in Amsterdam, who was the senior author on the latest Nature study.

Theres a simple understanding of genetics were all taught in high school. We learn, as Gregor Mendel discovered with pea plants, that we can inherit multiple forms of the same gene. One variation of the gene makes wrinkled peas; the other makes for round peas. Its true, but its hardly the whole story.

In humans, a few traits and illnesses work like this. Whether the bottom of your earlobes stick to the side of your face or hang free is the result of one gene. Huntingtons disease which deteriorates nerve cells in the brain is the result of a single gene.

But most of the traits that make you you your height, your personality, your intellect arise out of a complex constellation of genes. There might be 1,000 genes that influence intelligence, for example. Same goes for the genes that lead to certain disorders. Theres no one gene for schizophrenia, for obesity, for depression.

A single gene for one of these things also wont have an appreciable impact on behavior. If you have the bad variant of one gene for IQ, maybe your IQ score ... is 0.001 percent lower than it would have been, Posthuma says.

But if you have 100 bad variants, or 1,000, then that might make a meaningful difference.

Genome-wide association studies allow scientists to start to see how combinations of many, many genes interact in complicated ways. And it takes huge data sets to sort through all the genetic noise and find variants that truly make a difference on traits like intelligence.

The researchers had one: the UK Biobank, a library that contains genetic, health, and behavioral information on 500,000 Britons. For the study, they pulled complete genome information on 78,000 individuals who had also undergone intelligence testing. Then a computer program combed through millions of sites on the gene code where people tend to variate from one another, and singled out the areas that correlated with smarts.

The computer processing power needed for this kind of research this study had to crunch 9.3 million DNA letters from 78,000 people hasnt been available very long. But now that it is, researchers have been starting to piece together the puzzle that links genes to behaviors.

A recent genome-wide analysis effort identified 250 gene sites that predicted male pattern baldness in a sample of 52,000 men. (Would you really want to know if you had them?) And theres been progress identifying genes that signal risk for diabetes, schizophrenia, and depression.

And these studies dont just look at traits, diseases, and behavior. Theyre also starting to analyze genetic associations to life outcomes. A 2016 paper in Nature reported on 74 gene sites that correlate with educational attainment. (These genes, the study authors note, seem to have something to do with the formation of neurons.) Again, these associations are tiny the study found that these 74 gene variants could only explain 3 percent of the difference between any two people on what level of education they achieve. Its hardly set in stone that youll flunk school if you dont have these gene variants.

But still, they make a small significant difference once you start looking at huge numbers of people.

Its important to note that Posthumas study was only on people of European ancestry. Whatever we find for Europeans doesnt necessarily [extrapolate] for Asians or South Americans, [or any other group] she says. Those things are often misused.

Which is to say: The gene variations that produce the differences between Europeans arent necessarily the same variations that produce differences among groups of different ancestry. So if you were to test the DNA of someone of African origin, and saw they lacked these genes, it would be incredibly irresponsible to conclude they had a lower capacity for intelligence. (Again, there are also likely hundreds of more genetic sites that have something to do with intellect that have yet to be discovered.)

Posthumas work identifying genes associated with intelligence isnt about making predictions about how smart a baby might grow up to be. She doesnt think you can reliably predict educational or intelligence outcomes from DNA alone. This is all really about reverse-engineering the biology of intelligence.

Genes code for proteins. Proteins then interact with other proteins. Researchers can trace this pathway all the way up to the level of behavior. And somewhere along that path, there just might be a place where we can intervene and stop age-related cognitive decline, for instance, and Alzheimers.

We're finally starting to see robust reliable associations from genes with their behavior, she says. The next step is how do we prove that this gene is actually evolved in a disorder, and how does it work?

Understanding the biology of intelligence could also lead the way for personalized approaches to treating neurodegenerative diseases. Its possible that two people with Alzheimers may have different underlying genetic causes. Knowing which genes are causing the disease, then, you might be able to tailor the treatment, Posthuma says.

As more and more genome-wide studies are conducted, the more researchers will be able to assign people polygenic risk scores for how susceptible they might be for certain traits and diseases. That can lead to early interventions. (Or, perhaps in the wrong hands, a cruel and unfair sorting of society. Have you seen the movie Gattaca?)

And there are some worries about abusing this data, especially as more and more people get their genomes analyzed by commercial companies like 23&Me.

Many people are concerned that insurance companies will use it, she says. That they will look into people's DNA and say, Well, you have a very high risk of being a nicotine addict. So we want you to pay more. Or, You have a high risk of dying early from cancer. So you have to pay more early in life. And of course, that's all nonsense. Its still too complicated to make such precise predictions.

We now have powerful tools to edit genes. CRISPR/Cas9 makes it possible to cut out any specific gene and replace it with another. Genetic engineering has advanced to the point where scientists are building whole organisms from the ground up with custom DNA.

Its easy to indulge our imaginations here: Genome-wide studies are going to make it easier to predict what set of genes leads to certain life outcomes. Genetic engineering is making it easier to assemble whatever genes we want in an individual. Is this the perfect recipe for designer babies?

Posthuma urges caution here, and says this conclusion is far afield from the actual state of the research.

Lets say you wanted to design a human with superior intelligence. Could you just select the right variants of the 52 intelligence genes, and wham-o, we have our next Einstein?

No. Genetics is so, so much more complicated than that.

For one, there could be thousands of genes that influence intelligence that have yet to be discovered. And they interact with each other in unpredictable ways. A gene that increases your smarts could also increase your risk for schizophrenia. Or change some other trait slightly. There are trade-offs and feedback loops everywhere you look in the genome.

If you would have to start constructing a human being from scratch, and you would have to build in all these little effects, I think we wouldn't be able to do that, Posthuma says. It's very difficult to understand the dynamics.

There are about 20,000 human genes, made up of around 3 billion base pairs. We will never be able to fully predict how a person will turn out based on the DNA, she says. Its just too intricate, too complicated, and also influenced heavily by our environment.

So you could have a very high liability for depression, but it will only happen if you go through a divorce, she says. And who can predict that?

And, Posthuma cautions, there are some things that genome-wide studies cant do. They cant, for instance, find very, very rare gene variations. (Think about it: If one person in 50,000 has a gene that causes a disease, its just going to look like noise.) For schizophrenia, she says, we know that there's some [gene] variants that decrease or increase your risk of schizophrenia 20-fold, but they're very rare in the population.

And they cant be used to make generalizations about differences between large groups of people.

Last year, I interviewed Paul Glimcher, a New York University social scientist whose research floored me. Glimcher plans to recruit 10,000 New Yorkers and track everything about them for decades. Everything: full genome data, medical records, diet, credit card transactions, physical activity, personality test scores, you name it. The idea, he says, is to create a dense, longitudinal database of human life that machine learning programs can mine for insights. Its possible this approach will elucidate the complex interactions of genetics, behavior, and environment that put us at risk for diseases like Alzheimers.

Computer science and biology are converging to make these audacious projects easier. And to some degree, the results of these projects may help us align our genes and our environments for optimal well-being.

Again, Posthuma cautions: Not all the predictions this research makes will be meaningful.

Do we care if we find a gene that only increases our height or our BMI or our intelligence with less than 0.0001 percent? she asks. It doesn't have any clinical relevance. But it will aid our scientific understanding of how intellect arises nonetheless.

And thats the bottom line. The scientists doing this work arent in it to become fortune tellers. Theyre in it to understand basic science.

What most people focus on, when they hear about genes for IQ, they say: Oh, no. You can look at my DNA. You can tell me what my IQ score will be, Posthuma says. They probably dont know its much better if you just take the IQ test. Much faster.

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Scientists are finding more genes linked to IQ. This doesn't mean we can predict intelligence. - Vox

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New therapy offers hope against incurable form of breast cancer – The Guardian

June 6th, 2017 11:46 pm

A consultant studies a mammogram. The drug olaparib could slow cancer growth by three months, researchers have found. Photograph: Rui Vieira/PA

A type of inherited and incurable breast cancer that tends to affect younger women could be targeted by a new therapy, researchers have found.

A small study presented at the worlds largest cancer conference found treating patients with the drug olaparib could slow cancer growth by three months and be less toxic for patients with inherited BRCA-related breast cancer.

Researchers said there was not enough data to say whether patients survived longer as a result of the treatment.

We are in our infancy, said Dr Daniel Hayes, president of the American Society of Clinical Oncology and professor of breast cancer research at the University of Michigan. This is clearly an advance; this is clearly proof of concept these can work with breast cancer.

Does it look like its going to extend life? We dont know yet, he said.

The drug is part of the developing field of precision medicine, which targets patients genes to tailor treatment.

It is a perfect example of how understanding a patients genetics and the biology of their tumor can be used to target its weaknesses and personalize treatment, said Andrew Tutt, director of the Breast Cancer Now Research Centre at The Institute of Cancer Research.

Olaparib is already available for women with BRCA-mutant advanced ovarian cancer, and is the first drug to be approved that is directed against an inherited genetic mutation. The study was the first to show olaparib can slow growth of inherited BRCA-related breast cancer. The drug is not yet approved for that use.

People with inherited mutations in the BRCA gene make up about 3% of all breast cancer patients, and tend to be younger. The median age of women in the olaparib trial was 44 years old.

BRCA genes are part of a pathway to keep cells reproducing normally. An inherited defect can fail to stop abnormal growth, thus increasing the risk of cancer. The study examined the effectiveness of olaparib against a class of BRCA-related cancers called triple negative. Olaparib is part of a class of four drugs called PARP-inhibitors that work by shutting down a pathway cancer cells use to reproduce.

The study from Memorial Sloan Kettering Cancer Center in New York randomly treated 300 women with advanced, BRCA-mutated cancer with olaparib or chemotherapy. All the participants had already received two rounds of chemotherapy.

About 60% of patients who received olaparib saw tumors shrink, compared with 29% of patients who received chemotherapy. That meant patients who received olaparib saw cancer advance in seven months, versus four months for only chemotherapy.

Researchers cautioned it is unclear whether olaparib extended life for these patients, and that more research was needed to find out which subset of patients benefit most from olaparib.

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A New Gene Therapy Could Hold the Key to Curing Allergies and Asthma – Futurism

June 6th, 2017 11:46 pm

In Brief Researchers have successfully used immunotherapy to "turn off" asthma and allergic responses in animals. This work will eventually be used to create one-shot treatments that permanently silence allergies. Erasing Asthma

Scientists from the University of Queensland have used gene therapy to turn off the immune response responsible for asthma. The team believes their technique may also be able to permanently silence severe allergy responses to common allergens such as bee venom, peanuts, and shellfish. Thus far, the research has been successful in animal trials, and if it can be replicated in human trials, it may provide a one-time treatment for asthma and allergy patients.

The technique erases the memory of the cells which cause allergic reactions using genetically modified stem cells that are resistant to allergens. We have now been able wipe the memory of these T-cells in animals with gene therapy, de-sensitizing the immune system so that it tolerates the [allergen] protein, lead researcher Ray Steptoe said in a press release. We take blood stem cells, insert a gene which regulates the allergen protein and we put that into the recipient. Those engineered cells produce new blood cells programmed to express the protein and target specific immune cells, which turn off the allergic response.

According to the Centers for Disease Control (CDC), about 1 in 12 people (25 million) in the U.S.have asthma, and these numbers are increasing annually. As of 2007, the last year for which the CDC has data, asthma cost the U.S. approximately $56 billion in costs for medical bills, lost work and school days, and early deaths. According to the World Health Organization (WHO), 235 million people worldwide have asthma, which is the most common chronic childhood disease, occurring in all countries regardless of level of development.

The researchers findings must now besubjected to further pre-clinical investigation, with the aim of replicating the results in the laboratory using human cells. The longer term goal will be a one-time gene therapy injection that would replace short-term allergy treatments, which vary in their effectiveness. We havent quite got it to the point where its as simple as getting a flu jab so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals, Dr. Steptoe said in the press release.

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A first: All respond to gene therapy in a blood cancer study – ABC News

June 6th, 2017 11:46 pm

Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer that's on the rise. Although it's early and the study is small 35 people every patient responded and all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

Experts at an American Society of Clinical Oncology conference in Chicago, where the results were announced Monday, say it's a first for multiple myeloma and rare for any cancer treatment to have such success.

Chemotherapy helps 10 to 30 percent of patients; immune system drugs, 35 to 40 percent at best, and some gene-targeting drugs, 70 to 80 percent, "but you don't get to 100," said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

"These are impressive results" but time will tell if they last, he said.

ABOUT THE DISEASE

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, and more than 115,000 worldwide. It's the second fastest growing cancer for men and the third for women, rising 2 to 3 percent per year, according to the National Cancer Institute. About 60,000 to 70,000 Americans have it now.

Nine new drugs have been approved for it since 2000 but they're not cures; only about half of U.S. patients live five years after diagnosis.

With cell therapy, "I can't say we may get a cure but at least we bring hope of that possibility," said Dr. Frank Fan. He is chief scientific officer of Nanjing Legend Biotech, a Chinese company that tested the treatment with doctors at Xi'an Jiaotong University.

HOW IT WORKS

The treatment, called CAR-T therapy , involves filtering a patient's blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer and then given back to the patient intravenously.

Doctors call it a "living drug" a one-time treatment to permanently alter cells that multiply in the body into an army to fight cancer. It's shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma, in patients whose cancer worsened despite many other treatments.

THE STUDIES

In the Chinese study, 19 of 35 patients are long enough past treatment to judge whether they are in complete remission, and 14 are. The other five had at least a partial remission, with their cancer greatly diminished. Some are more than a year past treatment with no sign of disease.

Most patients had a group of side effects common with this treatment, including fever, low blood pressure and trouble breathing. Only two cases were severe and all were treatable and temporary, doctors said.

The second study was done in the U.S. by Bluebird Bio and Celgene, using a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients. Eighteen are long enough from treatment to judge effectiveness, and all 15 who got an adequate amount of cells had a response. Four have reached full remission so far, and some are more than a year past treatment.

WHAT EXPERTS SAY

The results are "very remarkable" not just for how many responded but how well, said Dr. Kenneth Anderson of Dana-Farber Cancer Institute in Boston.

"We need to be looking for how long these cells persist" and keep the cancer under control, he said.

Dr. Carl June, a University of Pennsylvania researcher who received the conference's top science award for his early work on CAR-T therapy, said "it's very rare" to see everyone respond to a treatment. His lab also had this happen all 22 children testing a new version of CAR-T for leukemia responded, his colleagues reported at the conference.

"The first patients we treated in 2010 haven't relapsed," June said.

Dr. Michael Sabel of the University of Michigan called the treatment "revolutionary."

"This is really the epitome of personalized medicine," extending immune therapy to more types of patients, he said.

NEXT STEPS

Legend Biotech plans to continue the study in up to 100 people in China and plans a study in the U.S. early next year. The treatment is expected to cost $200,000 to $300,000, and "who's going to pay for that is a big issue," Fan said.

"The manufacturing process is very expensive and you can't scale up. It's individualized. You cannot make a batch" as is done with a drug, he said.

Nick Leschly, Bluebird's chief executive, said the next phase of his company's study will test what seems the ideal dose in 20 more people.

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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IT’S A START: Newly approved gene therapy may help 4 percent of cancer patients – Sarasota Herald-Tribune

June 6th, 2017 11:46 pm

By Laurie McginleyThe Washington Post

The oncologist was blunt: Stefanie Joho's colon cancer was raging out of control and there was nothing more she could do. Flanked by her parents and sister, the 23-year-old felt something wet on her shoulder. She looked up to see her father weeping.

"I felt dead inside, utterly demoralized, ready to be done," Joho remembers.

But her younger sister couldn't accept that. When the family got back to Joho's apartment in New York's Flatiron district, Jess opened her laptop and began searching frantically for clinical trials, using medical words she'd heard but not fully understood. An hour later, she came into her sister's room and showed her what she'd found.

"I'm not letting you give up," she told Stefanie. "This is not the end."

That search led to a contact at Johns Hopkins University, and a few days later, Joho got a call from a cancer geneticist co-leading a study there.

"Get down here as fast as you can!" Luis Diaz said. "We are having tremendous success with patients like you."

What followed is an illuminating tale of how one woman's intersection with experimental research helped open a new frontier in cancer treatment with approval of a drug that, for the first time, targets a genetic feature in a tumor rather than the disease's location in the body.

The breakthrough, now made official by the Food and Drug Administration, immediately could benefit some patients with certain kinds of advanced cancer that aren't responding to chemotherapy. Each should be tested for that genetic signature, scientists stress.

"These are people facing death sentences," said Hopkins geneticist Bert Vogelstein. "This treatment might keep some of them in remission for a long time."

A pivotal small trial

In August 2014, Joho stumbled into Hopkins for her first infusion of the immunotherapy drug Keytruda. She was in agony from a malignant mass in her midsection, and even with the copious amounts of OxyContin she was swallowing, she needed a new fentanyl patch on her arm every 48 hours. Yet within just days, the excruciating back pain had eased. Then an unfamiliar sensation hunger returned. She burst into tears when she realized what it was.

As months went by, her tumor shrank and ultimately disappeared. She stopped treatment this past August, free from all signs of disease.

The small trial in Baltimore was pivotal, and not only for the young marketing professional. It showed that immunotherapy could attack colon and other cancers thought to be unstoppable. The key was their tumors' genetic defect, known as mismatch repair (MMR) deficiency akin to a missing spell-check on their DNA. As the DNA copies itself, the abnormality prevents any errors from being fixed. In the cancer cells, that means huge numbers of mutations that are good targets for immunotherapy.

The treatment approach isn't a panacea, however. The glitch under scrutiny which can arise spontaneously or be inherited is found in just 4 percent of cancers overall. But bore in on a few specific types, and the scenario changes dramatically. The problem occurs in up to 20 percent of colon cancers and about 40 percent of endometrial malignancies cancer in the lining of the uterus.

In the United States, researchers estimate that initially about 15,000 people with this defect may be helped by this immunotherapy. That number is likely to rise sharply as doctors begin using it earlier on eligible patients.

Joho was among the first.

Even before Joho got sick, cancer had cast a long shadow on her family. Her mother has Lynch syndrome, a hereditary disorder that sharply raises the risk of certain cancers, and since 2003, Priscilla Joho has suffered colon cancer, uterine cancer and squamous cell carcinoma of the skin.

Stefanie's older sister, Vanessa, had already tested positive for Lynch syndrome, and Stefanie planned to get tested when she turned 25. But at 22, several months after she graduated from New York University, she began feeling unusually tired. She blamed the fatigue on her demanding job. Her primary-care physician, aware of her mother's medical history, ordered a colonoscopy.

When Joho woke up from the procedure, the gastroenterologist looked "like a ghost," she said. A subsequent CT scan revealed a very large tumor in her colon. She'd definitely inherited Lynch syndrome.

She underwent surgery in January 2013 at Philadelphia's Fox Chase Cancer Center, where her mother had been treated. The news was good: The cancer didn't appear to have spread, so she could skip chemotherapy and follow up with scans every three months.

By August of that year, though, Joho started having relentless back pain. Tests detected the invasive tumor in her abdomen. Another operation, and now she started chemo. Once again, in spring 2014, the cancer roared back. Her doctors in New York, where she now was living, switched to a more aggressive chemo regimen.

"This thing is going to kill me," Joho remembered thinking. "It was eating me alive."

Genetics meets immunology

Joho began planning to move to her parents' home in suburban Philadelphia: "I thought, 'I'm dying, and I'd like to breathe fresh air and be around the green and the trees.' "

Her younger sister wasn't ready for her to give up. Jess searched for clinical trials, typing in "immunotherapy" and other terms she'd heard the doctors use. Up popped a trial at Hopkins, where doctors were testing a drug called pembrolizumab.

"Pembro" is part of a class of new medications called checkpoint inhibitors that disable the brakes that keep the immune system from attacking tumors. In September 2014, the treatment was approved by the FDA for advanced melanoma and marketed as Keytruda. The medication made headlines in 2015 when it helped treat former President Jimmy Carter for melanoma that had spread to his brain and liver. It later was cleared for several other malignancies.

Yet researchers still don't know why immunotherapy, once hailed as a game changer, works in only a minority of patients. Figuring that out is important for clinical as well as financial reasons. Keytruda, for example, costs about $150,000 a year.

By the time Joho arrived at Hopkins, the trial had been underway for a year. While an earlier study had shown a similar immunotherapy drug to be effective for a significant proportion of patients with advanced melanoma or lung or kidney cancer, checkpoint inhibitors weren't making headway with colon cancer. A single patient out of 20 had responded in a couple of trials.

Why did some tumors shrink while others didn't? What was different about the single colon cancer patient who benefited? Drew Pardoll, director of the Bloomberg-Kimmel Institute for Cancer Immunotherapy at Hopkins, and top researcher Suzanne Topalian took the unusual step of consulting with the cancer geneticists who worked one floor up.

"This was the first date in what became the marriage of cancer genetics and cancer immunology," Pardoll said.

In a brainstorming session, the geneticists were quick to offer their theories. They suggested that the melanoma and lung cancer patients had done best because those cancers have lots of mutations, a consequence of exposure to sunlight and cigarette smoke. The mutations produce proteins recognized by the immune system as foreign and ripe for attack, and the drug boosts the system's response.

And that one colon-cancer patient? As Vogelstein recalls, "We all said in unison, 'He must have MMR deficiency!' " because such a genetic glitch would spawn even more mutations.

When the patient's tumor tissue was tested, it was indeed positive for the defect.

The researchers decided to run a small trial, led by Hopkins immunologist Dung Le and geneticist Diaz, to determine whether the defect could predict a patient's response to immunotherapy. The pharmaceutical company Merck provided its still-experimental drug pembrolizumab. Three groups of volunteers were recruited: 10 colon cancer patients whose tumors had the genetic problem; 18 colon cancer patients without it; and 7 patients with other malignancies with the defect.

The first results, published in 2015 in the New England Journal of Medicine, were striking. Four out of the 10 colon cancer patients with the defect and 5 out of the other 7 cancer patients with the abnormality responded to the drug. In the remaining group, nothing. Since then, updated numbers have reinforced that a high proportion of patients with the genetic feature benefit from the drug, often for a lengthy period. Other trials by pharmaceutical companies have shown similar results.

The Hopkins investigators found that tumors with the defect had, on average, 1,700 mutations, compared with only 70 for tumors without the problem. That confirmed the theory that high numbers of mutations make it more likely the immune system will recognize and attack cancer if it gets assistance from immunotherapy.

For Joho, now 27 and living in suburban Philadelphia, the hard lesson from the past few years is clear: The cancer field is changing so rapidly that patients can't rely on their doctors to find them the best treatments.

"Oncologists can barely keep up," she said. "My sister found a trial I was a perfect candidate for, and my doctors didn't even know it existed."

Her first several weeks on the trial were rough, and she still has some lasting side effects today joint pain in her knees, minor nausea and fatigue.

"I have had to adapt to some new limits," she acknowledged. "But I still feel better than I have in five years."

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Gene therapy has been used to ‘switch off’ asthma symptoms – ScienceAlert

June 6th, 2017 11:46 pm

Scientists have used gene therapy to 'switch off' the immune response that causes asthma, and are hopeful that the same technique could be used to target other severe allergies to peanuts, bee venom, and shellfish,keeping them at bay for life.

The research, which has so far seen success in animal trials, works byerasing the memory of the cells responsible for causing an allergic reaction, and if replicated in humans, could offer a one-off treatment for allergy patients.

"The challenge in asthma and allergies is that these immune cells, known as T-cells, develop a form of immune 'memory', and become very resistant to treatments,"says lead researcherRay Steptoefrom the University of Queensland (UQ) in Australia.

"We have now been able 'wipe' the memory of these T-cells in animals with gene therapy, de-sensitising the immune system so that it tolerates the [allergen] protein."

An allergic response is a hypersensitive immune reaction to a substance that is normally harmless. When people are exposed to their allergic trigger, it can cause anything from itchy eyes and a runny nose to - in the most extreme cases -death.

Asthma is a common allergic response of the airways affecting 2.5 million Australiansand hundreds of millions around the world. About 80 percent of people who experience asthma in Australia are susceptible to hay fever - an allergic response to rye grass pollen.

"When someone has an allergy or asthma flare-up, the symptoms they experience results from immune cells reacting to protein in the allergen," says lead researcherRay Steptoefrom the University of Queensland (UQ) in Australia.

While previous research has looked into using nanoparticle 'trojan horses' to smuggle the allergen past the immune system, and at new immunotherapy approaches, right now, the most effective treatment for people suffering from allergies is to simply avoid all known triggers.

To figure out a better way, Steptoe and his teamtook bone marrow from mice that had been genetically modified to have a resistance against asthma caused by rye grass pollen, and transplanted the bone marrow into unmodified mice.

"We take blood stem cells, insert a gene which regulates the allergen protein, and we put that into the recipient," says Steptoe.

"Those engineered cells produce new blood cells programmed to express the protein and target specific immune cells, which 'turn off' the allergic response."

Even though this study only looked at asthma, the researchers hope that the same approach could be used to provide protection against other common allergies - food and otherwise.

"Our work used an experimental asthma allergen, but this research could be applied to treat those who have severe allergies to peanuts, bee venom, shellfish and the like," Steptoe said.

But before we start throwing our puffers in the bin, the studies still have to be replicated in human trials, and that's where things get much more complicated.

"In the real world, unfortunately, it's not just usually a single allergen protein [that causes an immune response]. There might be several proteins that you might be allergic to and you'd have to target each of those proteins," Steptoe told ScienceAlert.

"We're currently doing experiments to see if we can turn off multiple response at the same time."

The research is published in JCI Insight.

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Orchard Therapeutics hits up Hitachi CDMO to make autologous gene therapy – BioPharma-Reporter.com

June 6th, 2017 11:46 pm

By Staff ReporterStaff Reporter , 06-Jun-20172017-06-06T00:00:00Z Last updated on 06-Jun-2017 at 10:36 GMT2017-06-06T10:36:21Z

Hitachi Group subsidiary PCT Cell Therapy Services will provide clinical manufacturing services for Orchards autologous gene therapy for the treatment of adenosine deaminase deficiency.

Under terms of the deal, PCT will make clinical supplies of OTL-101, an autologous ex-vivo gene therapy for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID).

The Manufacturing Services Agreement (MSA) is an extension of a previous contract between the UK-based Orchard Therapeutics and its contract development and manufacturing organisation (CDMO).

We are very pleased to extend our relationship with PCT into a full GMP manufacturing services agreement for OTL-101, Orchards chief manufacturing officer Stewart Craig said in a statement.

As a world-leading CDMO for cell-based therapeutic products, this is an important step in advancing our lead program for the treatment of children afflicted with ADA-SCID.

PCTs CEO Robert Preti added the expansion will now include clinical manufacturing in support of [Orchards] ADA-SCID gene therapy is testament to our successful collaboration and our dedicated stewardship of this important program.

The clinical results are cause for hope among this patient population and we look forward to helping advance this important new therapeutic towards commercialisation.

The deal comes after Orchard announced it had contracted Dutch CMO PharmaCell to manufacture products from its gene therapy pipeline earlier this year, and seven months after the firm inked an alliance with gene and cell therapy firm Oxford BioMedica for process development services and cGMP-grade manufacture of lentiviral vectors.

Orchard did not supply further information when contacted by this publication.

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Do you really need to be worried about all that screen time? – Well+Good

June 6th, 2017 11:45 pm

Photo: Stocksy/Vegterfoto

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It feels great tobe one of the first people to Instagrammermaid toastor your early-morning workout with your furry BFF. And when its Bachelorette time, does an episodeeven happen if you dont live-tweet it?

All that time looking at screens (and the potentially eye-damaging blue lightthey emit) adds up, thoughespecially sinceyoublink 66 percent less than normal while checkingyour iPhone or watching videoson your laptop,studies show.

Think about how much of your day is spent looking at illuminated digital data: To start off, theres (at least) eight hours at the officeusing your work computer. Plus,the average American looks at their phone 76 timesper dayfor a total of 145 minutes. (A really good argument for ditching your phone on vacation, BTW.) And that doesnt even figure in Netflix binges(hello, nightly hit of Friends, a la Emma Watson).

So how do you know how much is too much? Is it even such a big deal?

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Symptoms like blurred or double vision, fatigue, or recurring headaches are a tipoff. But the best way to assess things is to visit your optometristtheres no hiding that late-night Instagram scrolling habit from them. They can tell if screens are hurting your eyes, even if you dont have any of the tell-tale signs, says Linda Chous, MD,optometrist and chief eye care officer of UnitedHealthcare.

There are muscles inside the eyes that have to work for us to see up close, and Dr. Chous says they have all a few tests to see how those muscles are workingwhich is a good reason to keep optometrists on your medical calendar (along with the gyno, dentist, and dermatologist). Theres no need to see your eye doctor as often as your main MD, but booking some face time every two years is ideal, Dr. Chous says.

Just because you have 20/20 vision doesnt mean you have healthy eyes. It can give you a false sense of security.

Just because you have 20/20 vision doesnt mean you have healthy eyes, she says. It can give you a false sense of security. Dr. Chous explains that the eyes truly are windows to the rest of the body, and often they can providecues for things to look out for, like diabetes or high-blood pressure.

The takeaway: Make a regular date with your optometrist toavoid stressing out about your screen time year-round. Best-case scenario? Youre all good. Or maybe you just need your prescription tweaked. Worst-case? You come up with a plan together.

3/3

Maybe you already know staring at a computer all day at work is messing with your visionyou can feel it. Or your eye doctor literally told you that you needed to chill with the screen time. (There isnt a hard-and-fast rule when it comes to how many hours is bad, so let your doctorand your eyesbe your guide.) So what do you do? James Stringham, PhD, who specializes in neurology and vision, says the first thing to do is fill up on more greens.

Leafy greens are full of luteinand zeaxanthin, two specific types of antioxidantsthat are vital for vision, he says. Dr. Stringham explains that theyre highly concentrated in eye tissue, which the blue light from screens can damage. When that happens, people can start experiencing headaches or migraines, he says.

Leafy greens are full of luteinand zeaxanthin, two specific types of antioxidantsthat are vital for vision.

Ideally, Dr. Stringham says people need 26 milligrams of the two antioxidants each dayabout two cups of spinach. But most people dont get that, and may opt to pop a supplement. (In fact, Dr. Stringham has been helping develop one, Blutein, which will be available next month at Vitamin Shoppe.) He says some professional athletes even take the two so-called eye vitaminsto speed up how quickly their eyes react to a moving target. Some other foods withlutein and zeaxanthin: eggs, zucchini, broccoli, and Brussels sprouts.

What about those blue light-fighting glasses that are gaining traction?Dr. Stringham says if youre getting enough bluetein and zeaxanthin, they arent necessary. Wearing glasses doesnt give you the other benefits filling up on the healthy foods wouldlutein and zeaxanthin is in your brain too, so getting enough is important for cognitive function as well, he says.

A breakdown in vision, he points out, is yet another sign of inflammation. So when you fill up on foods that fight that, it does your whole body good.

This inflammation-fighting food pyramid can help you build the ultimate healthy meal planand here are a few recipes, if you need guidance.

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Blind man wants provincial government to pay for his chance to regain sight – CBC.ca

June 6th, 2017 11:45 pm

ReginaldArseneau was 29 years old when he started losing his eyesight.

It didn't stop him from completing four years of university and building a house and a life. But now the 69-year-old has a chance to improve his vision, if he can only find a way to pay for it.

The Argus II, a new type of implant, captures images on a camera that's attached to glasses worn by the patient. The image is then transmitted to the eye's retina with the help of a small computer worn on a belt.

Arseneau, who suffers from retinitis pigmentosa, a condition that changes the way the retina responds to light, causing slow vision loss over time, could regain about two per cent of his eye sight, his opthamologist said.

But while the cost of the surgery will be covered, he needs help paying for the chip.

"It costs $150,000just for theArgusII. It's the chip they put in the eyes with the computers so just for that, it costs $150,000," said Arseneau. "After that it's sure I'll need a surgery but the government should pay the surgery. I will need also a follow-up for the first three months."

The New Brunswick government, however, said it's not paying.

Reginal and Marie-Ange Arseneau built their home in the 1980s, when Reginald was already blind. He has also published two books - one is a memoir, written in French. (Bridget Yard/CBC)

In anemail,Departmentof Health spokespersonGeneviveMallet-Chiassonsaid the department will "not consider coverage for this prosthesis at this time."

"According to the New Brunswick Regulation 84-20 under the Medical Services Payment Act, schedule 2b,prosthetic devices are not insured services," she wrote.

Arseneau said he'll continue to lobby for financial aid.

He said he's lived withretinitispigmentosafor over 40 years, but he is also losing his hearing now, and wears two hearing aids.

At home, he can still "see" with his hands, feeling his way around, even working on carpentry projects with the help of a braille ruler and measuring tape. But when he leaves the house, or goes on vacation, his wife, Marie-Ange, has to guide him, he said.

He hopes the implant will help him regain one of his senses, and his independence.

"I will see black and white and shapes," he said. "I could distinguish maybe a door, or windows, and that will really help me with my mobility."

Arseneau still has the ruler made for him at Universite de Moncton decades ago. He uses it in his woodworking projects. He finds simple ways to hold on to his favourite hobbies and independence. (Bridget Yard/CBC)

DrFalvioRezende, of TheMaisonneuve-RosementHospital in Montreal, said he'simplanted theArgusII in two patients.

"To transform an illness that would seem to have no cure, and re-engage people into society, it's enormous," he told Radio-Canada. "It's possibly the most touching experience I've had in my career."

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Diabetes Study in Indiana – MyWabashValley

June 6th, 2017 11:45 pm

Indianapolis, IN - INDIANAPOLIS (WISH) From awareness, to education, Indiana lawmakers will soon study diabetes. And it's a personal fight for the Indiana lawmaker spearheading the committee.

For millions of Americans, a quick prick, and sugar reading, is a daily routine. But it's something State Representative Vanessa Summers (D-Indianapolis) would like to see go away. "It's just an unnecessary disease that has grown rapid and is at epidemic portions in our country, and especially in Indiana, State Rep. Summers said.

A disease the American Diabetes Association said impacts nearly 30 million Americans by attacking cells and how bodies produce insulin. To fix this problem, Representative Summers is behind a state study. "It's time to put a face and a name, and action to a condition that is treatable, State Rep. Summers said.

The Indiana Department of Health said more than half a million Hoosiers suffer from diabetes. Nearly 300,000 may not know they have it, and it's the seventh leading cause of death across the state.

A problem representative Summers knows well, because she's had the disease for a decade. "I started out with an A1C of 13. Your A1C should be under 6, State Rep. Summers said. A disease that's hit her family hard. "I've had one cousin to die, State Rep. Summers said. He had a foot amputated, and then he died from complications of diabetes."

To save her life, she's made major changes, including her diet, and teaching others as well. "They just have got to eat right, State Rep. Summers said. They've got to rainbow their colors. You need red, green, purple, yellow."

She's gone from four shots a day, to one. A success story she hopes will inspire her fellow lawmakers, and other Hoosiers as they tackle this issue because she knows how tempting it can be to veer off course.

Sometimes I take a bite because I'm human, State Rep. Summers said. I want to taste that cake."

The study committee was announced about a week ago, but it may take some time before the group may not meet until later this summer. Lawmakers use these off-season meetings to learn information that can help them draft bills for when the 2018 session starts.

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Griffin to hosts talks on diabetes – CT Post

June 6th, 2017 11:45 pm

Photo: Contributed / Contributed

Griffin to hosts talks on diabetes

The Diabetes Education & Support Group at Griffin Hospital will host two free presentations on managing diabetes during the summer holidays on Tuesdau, June 13 at 2:30 p.m. and 6:30 p.m. at the hospital, 130 Division St., Derby.

Certified Diabetes Educator Mary Swansiger will lead a discussion on managing diabetes during holidays, vacation and special events during the summer, including meal planning and strategies for making the summer happier and healthier.

Both presentations will be in Childbirth Education Classroom A. There will be free valet parking for the 2:30 p.m. presentation.

The Diabetes Education & Support Group meets September through June on the second Tuesday of each month to discuss the management of diabetes, its challenges, and day-to-day dietary concerns. Individuals with diabetes and their caregivers are welcome to attend.

No registration is required. For more information, call 203-732-1137.

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Month-Long Diabetes Control Possible with New Injection | GEN – Genetic Engineering & Biotechnology News

June 6th, 2017 11:45 pm

The daily or once-weekly insulin shotnecessary for the control of type 2 diabetescould be replaced by a twice- or even once-a-month shot. A new, longer-lasting injectable formulation has been developed that combines a familiar diabetes-control molecule, glucagon-like peptide-1 (GLP1), with a heat-sensitive elastin-like polypeptide (ELP). Once a solution containing the GLP1ELP combo passes through a standard needle and penetrates the skin, it reacts to body heat, forming a biodegradable gel-like depot that slowly releases the drug as it dissolves.

The novel drug-delivery mechanism was developed by scientists based at Duke University, who assert that it could be used to enhance therapeutic outcomes by eliminating peak-and-valley pharmacokinetics and improving overall safety and tolerability. The scientists, led by Ashutosh Chilkoti, Ph.D., chair of the department of biomedical engineering at Duke, suggest that their work could be broadly applicable; that is, it could improve the pharmacological performance of peptides and protein therapeutics besides GLP1.

Details of the work appeared June 5 in the journal Nature Biomedical Engineering, in an article entitled One-Week Glucose Control via Zero-Order Release Kinetics from an Injectable Depot of Glucagon-Like Peptide-1 Fused to a Thermosensitive Biopolymer. The one week indicated in the title refers to the drug depots performance in mice. Glucose control, the scientists found, was more durable in rhesus monkeys, and even longer glucose control, the scientists suggested, could be achieved in humans, since humans have slower metabolisms than mice or monkeys.

A subcutaneous depot formed after a single injection of GLP1 recombinantly fused to a thermosensitive elastin-like polypeptide results in zero-order release kinetics and circulation times of up to 10 days in mice and 17 days in monkeys, the authors of the article indicated. The optimized pharmacokinetics lead to 10 days of glycaemic control in three different mouse models of diabetes, as well as the reduction of glycosylated haemoglobin levels and weight gain in ob/ob mice treated once weekly for 8 weeks.

Many current treatments for type 2 diabetes use GLP1, a signaling molecule that causes the pancreas to release insulin to control blood sugar. However, this peptide has a short half-life and is cleared from the body quickly.

To make treatments last longer, researchers have previously fused GLP1 with synthetic microspheres and biomolecules like antibodies, making them active for 2 to 3 days in mice and up to a week in humans. Currently, the longest-acting glucose control treatment on the market, dulaglutide, requires a once-weekly injection, while standard insulin therapies often have to be injected twice or more every day. Despite improvements such as these, many treatments don't include a mechanism to control the rate of the peptide's release, and treatment effectiveness can plateau after prolonged use.

The Duke researchers persisted with their ongoing experiments, which focused on thermosensitive delivery biopolymers. By varying the design of their delivery biopolymers at the molecular level, they found a sweet spot that maximized the duration of the drug's delivery from a single injection, noted Dr. Chilkoti. "By doing so, he continued, we managed to triple the duration of this short-acting drug for type 2 diabetes, outperforming other competing designs."

Building upon their previous work with the drug and delivery system, researchers in the Dr. Chilkotis laboratory optimized their solution to regulate glucose levels in mice for 10 days after a single injection, up from the previous standard of 2 to 3 days.

In further tests, the Duke team found that the optimized formulation improved glucose control in rhesus monkeys for more than 14 days after a single injection, while also releasing the drug at a constant rate for the duration of the trial.

"What's exciting about this work was our ability to demonstrate that the drug could last over 2 weeks in nonhuman primates," remarked Kelli Luginbuhl, a Ph.D. student in Dr. Chilkotis laboratory and co-author of the study. "Because our metabolism is slower than monkeys and mice, the treatment should theoretically last even longer in humans, so our hope is that this will be the first biweekly or once-a-month formulation for people with type 2 diabetes."

Despite a variety of treatment options, managing type 2 diabetes still poses a problem. Patients don't always reach their glycemic targets, and adherence to a treatment plan that relies on frequent, meal-specific dosing leaves room for human error. By limiting the number of injections a person will need to control their glucose levels, the researchers hope this new tool will improve treatment options for the disease.

The researchers now plan to study the immune response to repeated injections and test the material with other animal models. They are also considering additional applications for this controlled-release system, such as delivering pain medication.

Dr. Chilkoti also indicated that because the drug is synthesized inside Escherichia coli bacterial cultures instead of mammalian cells, it is cheaper and faster to produce, making it a potential target for use in developing countries once it's commercialized.

According to a report issued last year by Grand View Research, the global insulin market is expected to reach $53.04 billion by 2022. Grand View anticipates that the most lucrative segment will consist of long-acting analogs. The segments high growth rate, estimated at 15.0%/year, is accounted for by fast-selling products such as Lantus by Sanofi Aventis. Moreover, the addition of new products such as Novo Nordisks Tresiba ultra-long-acting analog is expected to further drive segment growth. Tresiba is administered subcutaneously once daily at any time of day. Even longer-lasting formulations, such as those contemplated by Dr. Chilkotis team, may contribute to yet more growth in the segment.

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Month-Long Diabetes Control Possible with New Injection | GEN - Genetic Engineering & Biotechnology News

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Health tourism’s economic impact grows in Yucatan – The Yucatan Times

June 6th, 2017 11:45 pm

Officials announce the creation in Merida of a dental cryopreservation laboratory.

MERIDA With the increase in the exchange rate of the dollar, health tourism in Yucatan will generate even more than the three million dollars per year previously received. Mrida receives and treatsbetween three thousand and four thousand patients, said Armando Noguera Aguilar, general director of Dental Perfect, a company that will invest 25 million pesos in the city during 2017.

Armando Noguera Aguilar, general director of Dental Perfect (Photo: La Jornada Maya)

In a press conference at El Gran Caf, held in February, Noguer Aguilar said that dental health tourism treats seven million people each year in Mexico, so as Mrida turns into a cluster for the medical sector, there is an enormous potential to attract new customers.

The reason why there is health tourism in Merida, he explained, is because in the United States a dental implant can cost between five thousand and six thousand dollars, while in Yucatan only one thousand dollars.

To the state also come visitors from Europe, for the same economic benefit, he said. People prefer to go to Mexico, because they find the same quality with better prices, he said.

The investment of 25 million pesos to be held in Merida has as its objective the creation of three clinics and a laboratory for cryopreservation of dental stem cells, spaces that will create 150 direct jobs for skilled workers and 450 indirect ones; Its presence will increase dental health tourism by 25 percent, he predicted.

In about 10 years, dental stem cells will regenerate neuronal, bone, muscle, heart and some organs to treat problems such as Alzheimers or brain tumors.

With this scenario, he said that the quality of life of the human being is expected to increase in 10 years over the next decade, due to research in the branch worldwide, in places such as Germany, Switzerland and even Mexico.

Source: http://www.lajornadamaya.mx

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