Archive for December, 2019

The history of the elephants in Borneo is the most interesting of the four sub species of elephants that exist in the wilds of Asia. The four sub species are Elephasmaximusmaximus from Sri Lanka, Elephasmaximusindicus from mainland India, and Elephasmaximussumatranus from Sumatra, Indonesia. The status of the Bornean elephant Elephasmaximusbornensis is yet to be confirmed.

For a long time there was a theory that the Bornean elephants were smaller than the other mainland sub species and were called pigmy elephants. However, now with scientific investigation, it has been established that these elephants are the same as the other Asian elephants. The authorities in Borneo however prefer to continue to refer to them as pigmy elephants. This is because they feel that pigmy elephants would be more attractive to potential tourists. There is a push to promote tourism in Sabah and Borneo in a big way.

There was much speculation as to whether the Bornean elephant is really a wild elephant or whether they were released into the wild by a former Sultan. Dr. Prithiviraj Fernando and some other researchers found that the DNA of the Bornean elephant proved that it was a wild elephant and not feral.

Two different alternative hypotheses have been suggested by researchers. One is that the elephants in Borneo are non-native to Borneo and are a recent introduction, in the 17th century. The introduction hypothesis is based on historical records suggesting that the current population represents the descendants of a domesticated herd that formerly existed on Sulu Island, Philippines, and were introduced to eastern Sabah by the Sultan of Sulu in the 17th century. The original elephants, most likely, came from the Javan elephant population, now extinct. It was further reported that only two elephants were introduced to Jolo (Sulu) Island in the late 13th century, and their descendants were transported to Sabah around 1673.

The other theory is that they are from an ancient colonization several thousand years ago. No fossils have been found, which has led to the theory that it is evidence of a very recent introduction. On the other hand it has been found by researchers, including Dr. Prithiviraj Fernando, that there is DNA divergence of the Borneo sub species in other Asian elephants. This favours the theory of early existence.

The Bornean elephant is morphologically and, in some ways, behaviourally distinct from the elephants of mainland Asia. Their genetic distinctiveness from other mainland Asian elephant subspecies makes them one of the highest priority populations for Asian elephant conservation. It is classified as endangered according to the IUCN (International Union for Conservation of Nature) Red List of threatened species.

The social behaviour of the Bornean elephant is the same as that of the other subspecies of the Asian elephant. The herd is led by the matriarch with all adult males being ejected by the herd when they attain puberty. This is to prevent inbreeding. If the male calves remain after they become adults, they will necessarily have to mate with their close relatives. The males wander around on their own or in small bull groups. Only some of the males carry tusks. The percentage of males with tusks in the Bornean population has not been ascertained as yet.

The Bornean elephant population in Sabah is about 2,000 individuals that are currently restricted mainly to a limited number of forest reserves. The main populations are in the Central Forest and Tabin Wildlife Reserve. Herds of elephants are also found in the Lower Kinabatangan Wildlife Sanctuary and the North Kinabatangan Reserve.

The main threats to the survival of the species are population fragmentation and isolation of the existing herds. These elephants do not cross from one reserve to another across human-dominated landscapes that separate forest fragments. This means that since there is no movement into new areas and no new blood flowing, there is the possibility of new species developing within the isolated herds. Conservation measures should be taken to maintain current levels of genetic diversity in fragmented habitats.

The conservation of the Bornean elephant should aim at securing connectivity between spatially distinct populations. It may not be easy but elephant corridors have to be established to facilitate the free and secure movement of these elephants from one range to another.

Pics: Benoit Gossens

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Bornean elephants are the same as other Asian elephants - Sunday Observer

It's hard to imagine that season one of The Big Bang Theory aired 11 years ago. That11 years ago, Sheldon got himself a loom, the boys'were senthome without pants and the couples we known and love were not yet formed.

When the sitcom first aired in 2008, little did audiences know what a science-comedic phenomenon it would become. Earlier this year, whenTheBig Bang Theoryendedits run, fanswere polarised.Some had continually called for the show to end,whilst others expressed sadness over its finale.

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In general, TheBig Bang Theoryreeked of the wittiest and brilliant writing in its first half, seasons one through six. That being said, season one especially is filled with underappreciated humor. Here are some of those jokes.

In the pilot episode, Sheldon and Leonard visit the 'High IQ Sperm Bank' to donate sperm for money. Originally it's Sheldon's idea because he wants to make a little extra money to get fractional T1 bandwidth in the apartment.

As the two are seated in the clinic, Sheldon comes to the conclusion that it's actually genetic fraud because there's a possibility that a toddler with the same DNA wouldn't come out as smart as him. He says "Some poor woman is going to pin her hopes on my sperm, what if she winds up with a toddler who doesn't know if he should use an integral or a differential to solve the area under a curve?"

After Leonard reassures him the mother would surely still love her child anyway, Sheldon doesn't see it that way, saying "I wouldn't."

You can never sit in Sheldon's spot, that's rule one. In the pilot episode Penny, the new neighbor, makes that mistake. Sheldon, being the gracious host that he is, gives her an earful. By earful we mean, some pretty complicated, science-jargon laced reasons as to why she should move immediately.

The now-legendary reasoning is "In the winter that seat is close enough to the radiator to remain warm, and yet not so close as to cause perspiration. In the summer its directly in the path of a cross-breeze created by open windows there, and there. It faces the television at an angle that is neither direct, thus discouraging conversation, nor so far wide to create a parallax distortion."

Did you know that the concept of Zodiac signs is a mass cultural delusion? At least according to Sheldon Cooper, it is.

In the opening episode, Penny introduces herself as a Sagittarius. Her zodiac sign, according toher, tells the guys way more than they should need to know.

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But according to Sheldon it only tells them that sheparticipates in the "mass cultural delusion that the Suns apparent position relative to arbitrarily defined constellations and the time ofher birth somehow affectsher personality." Neat.

The show established Leonard's incapacity to flirt and talk with women early on. He calls Penny's waitressing job at the Cheesecake factory as her "acting as like.. a carbohydrate delivery system."

By this point, Penny is very well accustomed to Leonard and Sheldon, so she comes up with a quick-witted answer in no time. "Call it whatever you want, but I get minimum wage."

Dr. Leslie Winkle, an experimental physicist at California Institute of Technology was one of the most quick-witted characters on the show. We are not talking of a mean sense of humor,rather intelligent humor. Remember the time she explained the chemistry of sex?

Penny: "Wow, a girl scientist!" Leslie: "Yep, come for the breasts. Stay for the brains." This was just one of the earlier of Leslie's legendary quips and insults. Every appearance was punctuated with smart comments and quick thinking.

One of the groupmates, Raj Koothrappali is from India,and he adds to the social variation in their dynamics. Audiences are given a peek into Indian culture, every time Rajesh's parents, Doctor and Mrs. Koothrappali, call from India.

In"The Grasshopper Experiment", Rajesh's parents call to inform him they have set him up with some Indian girl Lalita Gupta, practicing dentistry in L.A.

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Whilst Rajesh tries stopping them from meddling in his life, Sheldon slyly reminds him that his parents don't consider this as meddling and that, "Indian parents continue to have greater than average involvement in their children's love lives." An angry Raj retorts "Why are you telling me about my own culture?" The first of many similar exchanges.

Remember the gigantic time machine from the movie Time Machine in "The Nerdvana Annihilation"? Leonard leads the gang to buy this machine prop, which takes up half the boys' living room. Howard and Raj also pitch in on condition that everyone will take the prop home in turn.

To Howard, this time machine shall serve the purposes of a "chick magnet", something he is going to use to woo the ladies. And he's not shy about it, either. This leads Sheldon to add the rule "In addition to the expected no shoes in the time machine and no eating in the time machine, I propose that we add pants must be worn at all times in the time machine."

In "The Nerdvana Annihilation", Penny is late for work, as the guys' time machine prop has blocked her way. Unable to go down the stairs, Sheldonadvises herto go up the roof,and hop over thesmall gap between this and the otherbuilding.

The small gap turns out to be a three feet wide gap and she slips and skins her knee. And when shedoes manage to take the leap, it turns out the door to the stairwell of the second building is locked. She is thus forced to go down a fire escape which ends on an Armenian Family's floor.

This lovely Armenian family feeds her eight courses of lamb. And, it gets worse. They try to fix her up with their son.

In "The Pork Chop Indeterminacy",Mary Coopersends over her daughter and Sheldon's twinsister, Missy to Sheldon. The immediate purpose is to get Sheldon's signatures on his father'sestate papers.

Missy visits Sheldon's office, causing the males at CalTech tohover around. Because Sheldon is so disconnected from his twin sister, none of his friends have an idea who she is to him.

Leonard asks "So how do you two know each other?" To which Missy replies, "Oh, he once spent nine months with my legs wrapped around his head." This is Missy's eyebrow-raising way of describing the fact she and Sheldon are twins.

Schrdinger's Cat is referenced in "The Tangerine Factor" by Sheldon Cooper. The premise of this joke is that Leonard asks Penny out for a date, which she agrees to. Since Leonard isn't the type of guy Penny usually goes out with, she has second thoughts about the date. As does Leonard.

Before the date, Pennyand Leonard both turn to Sheldon for advice, separately though. And he advises them to go through with it. The date could turn out to be both good and bad, based on the whole premise ofSchrdinger's Cat. Leonard immediately gets it, but Penny requires some classwork.

NEXT:The Big Bang Theory: 10 Reasons Why Mary Cooper Is The Best Guest Character On The Show

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Bisma Fida thinks pop culture shapes human lives. Sitcoms, dramas, and movies have helped her survive the impact of the geopolitical conflict of her homeland. Every piece she authors is essentially a third-world feminist's take.You can connect to her at bisma.fid@yahoo.com

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The Big Bang Theory: 10 Season 1 Jokes You Didnt Notice - Screen Rant

In the vast arsenal of animal weaponry, the most exaggerated, elaborate and diverse devices such as tusks, claws and antlers have not been shaped by a need to fend off fierce predators. Rather, these impressive forms are driven by sex.

Everybody understands at a gut level that its usually males that have flashy displays or weapons like tusks and antlers, says Doug Emlen, an animal weapon expert at the University of Montana in Missoula. Biologists say that these fantastic shapesfrom the giant curved tusks of woolly mammoths to the nightmarish jaws of stag beetlesevolved to ward off competition from rival males and to impress females.

Examples of such sexually selected weapons abound throughout the animal kingdom in insects, fish, crustaceans, reptiles and mammals as varied as narwhal, rhinoceros and moose. Even extinct species such as trilobites and dinosaurs sported elaborate projections. The number and variety of examples argue that evolution has turned to weaponry time and again in the race to reproduce successfully.

Its such a common theme that Emlen had to persuade his editors to include seven detailed, full-page line drawings in a survey of natures weapons that he wrote for the 2008 Annual Review of Ecology, Evolution, and Systematics, featuring more than 280 examples of fantastical spikes, horns, antlers, pincers, tusks, claws, extended jaws, saws and spears. The illustration above offers a taste.

Scientists still debate the degree to which female choice plays a role in shaping the weapons flair and are still trying to figure out what factors drive the diversity of weapon forms seen among even closely related species. But its clear that the wild array of weapons evolved to aid successful mating.

Like the structures, types of combat vary greatly. Rhinoceros beetles, named for their rhino-like horns, guard access to the oozes of tree sap that females feed upon before laying eggs. Rival males size each other up, and if their horns sizes are similarly matched, a face-off ensues and each uses his horns to try to flip, pry and toss his rival off the tree branch.

Most species of male fiddler crabs guard their burrows, where mating takes place. Dueling males shove and tap on each others single, enlarged clawand, should the fight escalate, they lock claws, secret-handshake style, as if theyre testing the others strength. If one decides he has the upper hand, he flings his opponent away from the burrow.

The fearsome weapons seem to evolve whenever three criteria are met, Emlen says. One: Males must be competing over either resources such as food or over females. Two: Its possible for access to those resources to successfully be guarded. And three: Males of the species compete in one-on-one duels.

But the fighting is almost never to the death and rarely results in serious wounds. Scientists say that this supports the idea that these weapons are built for rivalrytheir designs optimized not for destruction but for power struggles. Indeed, variation in the size of male weaponry is huge, Emlen notes: While overall body size among adult male elk might vary by a factor of 2 at most, their antler racks can vary by a factor of more than 30, he says. And the most dazzling weapons act largely as deterrents, with actual fights breaking out only when males are closely matched.

As the weapons grow bigger and flashier, they come with the cost of producing and lugging around such big structures. (And sometimes other costs: Male fiddler crabs can only stuff algae in their mouths with one claw.) Studies show that the weapons sizes are sensitive to nutrition, parasite load, stress and overall physical conditionand so the healthiest, most fit individuals sport the most impressive weapons.

Researchers consider these ostentatious male weapons to be honest signalsadvertising the owners might and fitness accurately. And not just physical fitness. A study of nearly 200 Iberian red deer stags measured the size and complexity of the animals antlers and found that bigger and more elaborate racks correlated with both bigger testes and faster-swimming sperm. From that and other evidence, many biologists think that bigger weapons can advertise reproductive superiority, too.

And while Emlen believes that male weapons evolved primarily for the purposes of male-male rivalry battles, comparative physiologist Brook Swanson of Gonzaga University in Spokane, Washington, argues that those signals are almost certainly also being assessed by females choosing mates. Even if a male could beat up all the other males, females almost always have a choice among mates, he says.

Take those fiddler crabs. Males of more than 100 species of the crabs have enlarged claws, and research suggests that the females can be picky. Some will cruise an area of multiple male burrows and size up the weapons being waved at them, among other things, before selecting a mate. We dont know what the females thinking, but shes taking into account a bunch of complicated information, Swanson says.

Though scientists believe that the primary role for these animal weapons is in reproduction, there are cases in which the weapons also serve as deterrents or defenses against predatorslikely as an evolutionary bonus. Elk antlers are a case in point. Unlike many other North American species in the deer family, elk hang onto their antlers until March, long after the mating season has ended in October. When Matt Metz, a PhD student at the University of Montana, and his colleagues tracked wolf kills in Yellowstone National Park they found that during March, wolves are three to four times more likely to attack an antlerless male elk than one still wearing his rack.

Since elk rarely use the antlers in defense, preferring to rear up and kick predators with their front hooves, presumably the structures serve as deterrents, Metz says. Yet if antler weapons had evolved primarily as a defense against predators, it wouldnt make sense to shed them at all, he addsand females should have them, too.

Why nature came up with such a bizarre array of weapon shapes and forms remains a bit of a mystery. But as a general rule, Swanson says, evolution tended to exaggerate structures already in existence. Crabs and lobsters have pincer claws that over evolutionary time became enlarged. And arthropods (spiders, insects and crustaceans) have exoskeletonsthat genetic changes could sculpt to form projections such as the horns or giant mandibles seen in beetles.

Weapons also are probably molded by the type of fighting and where its doneas borne out by work on the shapes of rhinoceros beetle horns by evolutionary biologist Erin McCullough. As a graduate student with Emlen, she spent two summers in Taiwan videotaping battles of the Japanese horned beetle, which has a pitchfork-shaped horn. She compared its fights to those of the Hercules beetle, which sports thick, pinching horns, and a species of Golofa beetle, which has thinner, sword-like horns. Each fights in slightly different ways, all with the goal of flicking their opponent off a tree branch or bamboo shoot.

McCullough, now a postdoctoral researcher at Syracuse University in New York, first measured how much force was needed to dislodge an average-size male from a branch. Next, she CT-scanned the critters horns, built 3-D computer models of the structures and used engineering tools to calculate the stresses and strains that the structures could withstand. She found that each horn performed best under the forces of its species-specific fighting style. This is a big component for why different species have different weapons, she says.

In October, an international group of researchers used the same computer modeling techniques to suggest that the largest antlers ever to existthe 12-feet-across by 5-feet-high rack of the prehistoric Irish elkwere used for male sparring, too.

But McCullough notes that the scariest, showiest weapons are not always very lethal. Some diversity, such as curlicues and extra tines, is probably driven by the display functions of weapons, she says.

Some of the largest animal weapons ever found adorned dinosaur heads. An example is seen in the horns and frills of the triceratops, a type of ceratopsid dinosaurbig-bodied herbivores that lived in large herds in open spaces, not unlike caribou. They had the biggest skulls of land-living animals that ever lived, partly due to these big bony structures on their heads, says Scott Sampson, paleontologist and executive director of the California Academy of Sciences in San Francisco.

Of course, its tricky to study dinosaur behavior, or even determine a skeletons sex, from fossils. Paleontologists continue to debate whether such unusual face decor was used to help dinosaur species recognize their own kind, for male-male mating contests or signals to females, or for defense. But Sampson says several lines of evidence persuade him that these horns were sexual weapons or displays rather than spears to fend off predators.

Importantly, these features werent fully grown until the animals reached adult size and reproductive age. And many of the features of dinosaur horns, spikes and frills were lousy as weapons against carnivorous predators, Sampson says. Some were thin to the point of fragility or curved in seemingly the wrong direction. Take, for example, Kosmoceratops, a flamboyant fossil found in southern Utah that sported 15 horns on its face, the top of its skull and its bony frill, some of which curve back on themselves. Im quite certain this pattern is all about show, Sampson says. Ceratopsids, he says, would have been more likely to use their sheer size as a weapon against predators.

From spines and plates on late Cretaceous behemoths to horns on tiny, modern-day beetles, making and carrying flashy weapons can come at a huge energy cost. An elks antlers are akin to a 180-pound man wearing a 12-pound gold chain around his neck.

But the costs are worth it. In a lot of mating systems, if you dont produce a weapon, then you have zero success, Swanson says. You have no choice but to play the game.

This article originally appeared in Knowable Magazine, an independent journalistic endeavor from Annual Reviews. Sign up for the newsletter.

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The Weapons of Sexual Rivalry - Scientific American

Representational image: A young science enthusiast peering at the sky using a telescope

Chandrayaan-2 may have dominated popular imagination during 2019 despite the Vikram Lander failing to soft-land on the lunar surface, but the year was marked by several significant developments by Indian scientists in fields ranging from nanotechnology to climate change.

The run-up to the lunar mission with planned landing of the lander-cum-rover, the launch campaign, the journey to the lunar orbit and the landing sequence all attracted national and international attention. The year ended in triumph for citizen science when Chennai-based software engineer, Shanmuga Subramanian, discovered debris of Vikram on the lunar surface using publicly available high-resolution images of the landing site. This development comes close to a rise in citizen science initiatives in the country.

Staying with space and astronomy, star of an exoplanet was named after Indian physicist, Bibha Chowdhury. During the year, Indian software engineers got readied software that will run the Thirty Meter Telescope (TMT), which is slated to be the worlds largest ground-based telescope operating at optical and infrared wavelengths. Details about TMT and other international Big Science projects in which India is participating were on display in a roving exhibition called Vigyan Samagam which attracted huge crowds.

Climate change: Responding to climate change impacts being seen in the Himalayan region, Indian scientists developed a common framework for assessment of climate change vulnerability in all the states in the region, using an index based on socio-economic factors, demographic and health status, sensitivity of agricultural production, forest-dependent livelihoods, and access to information, services and infrastructure. This knowledge will now be applied to develop a countrywide map of climate vulnerability.

Scientists from the Indian Institute of Tropical Meteorology (IITM) found a link between warming of the Indo-Pacific Ocean and changing rainfall patterns in many parts of the globe, including India. The warming pools of the Indo-Pacific Ocean are expanding, and this, in turn, is altering a major weather phenomenon known as the Madden Julian Oscillation (MJO). The warming of Indo-Pacific Ocean is occurring due to man-made emissions. Another group from the Indian Institute of Science warned that as many as 55 percent of glaciers in the Satluj basin may disappear by 2050. and 97 percent by 2090, under extreme climate change scenario. Using ice thickness of glaciers as the basis, scientists also estimated that glaciers in the Hindu Kush Himalayas might contain 27% less ice than previously suggested.

Eco-friendly technologies: The year saw progress towards development of less polluting crackers, with the Council of Scientific and Industrial Research (CSIR) releasing first set of green crackers. A national centre to pursue R&D in clean coal technologies was also opened in Bangalore. Eight teams of innovators from different parts of the world were selected for an international competition to develop more efficient and climate-friendly cooling solutions for residential buildings. The team will get seed money to translate their ideas into prototypes. The final winner of the Global Climate Prize will be announced in November 2020.

Representational image: Microscopic bacteria

Indian genomic data: In an important development, Delhi-based Institute of Genomics and Integrative Biology (IGIB) and Hyderabad-based Centre for Cellular and Molecular Biology (CCMB) completed whole genome sequencing of 1008 Indian individuals representing diverse ethnic groups in the country. The data will act as baseline information for developing various applications in predictive and preventive medicine.

Scientists from CCMB also found underlying genetic factors for infertility among Indian men. This knowledge could help in developing a genetic test for male infertility in near future. As part of genetic studies to trace the origins of population groups in the Indian sub-continent, it had been seen that sizeable population group of Mundas in central and northeast India shares genetic ancestry with Southeast Asian populations as well. A study revealed how and when this admixture between Mundas and Southeast Asian populations took place.

The Department of Biotechnology (DBT) launched a new human atlas initiative called Manav to develop a unified database of molecular network of all the tissues in the human body, and to derive a holistic picture of the working of human body. This mega project will collate and integrate molecular information on human tissues and organs that currently lies hidden in research articles in an unstructured and disorganized form.

Developments in gene editing: Indian scientists developed a new variant of currently popular gene editing tool, CRISPR-Cas9, and showed that it can increase precision in editing genome while avoiding unintended changes in DNA. The researchers showed that this type of gene editing can be used to correct sickle cell anemia, a genetic blood disorder. The experiments were done in human-derived cells from patients of sickle cell anemia, according to findings published in Proceedings of the National Academy of Sciences (PNAS).

New nano materials: Continuing their work in nano science and technology in 2019, scientists at the Mumbai-based Tata Institute of Fundamental Research (TIFR) used gold nanoparticles, and by rearranging size and gaps between them, developed a new material with unique properties like capacity to absorb light and carbon dioxide. Gold does not have these properties, and therefore, the new material has been named black gold, dye to its black appearance.

Boosting rice productivity: Scientists at the National Institute of Plant Genome Research (NIPGR) identified a gene involved in regulating the size of rice grain. The new development represents a new approach towards developing rice varieties that produce bigger and consequently heavier grains. Scientists from the Bose Institute came up with a new salt-tolerant transgenic rice plant by over-expressing a gene from a wild rice called Porteresia coarctata into the commonly used IR 64 indica rice variety.

Other important developments during the year included a new plan to establish a museum for marine archaeology at Lothal, a new satellite-based weather information service for deep sea fishers, grand challenge for cancer research to develop affordable cancer diagnostics and treatment, a white paper on e-cigarettes that led to its ban in India, and new initiative to boost malaria research in the country.

(This article was originally published on India Science Wire.)

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Rewind 2019: A Look Back at Significant Developments in Indian Science This Year - The Weather Channel

Deep Learning

One of the factors often credited for this latest boom in artificial intelligence (AI) investment, research, and related cognitive technologies, is the emergence of deep learning neural networks as an evolution of machine algorithms, as well as the corresponding large volume of big data and computing power that makes deep learning a practical reality. While deep learning has been extremely popular and has shown real ability to solve many machine learning problems, deep learning is just one approach to machine learning (ML), that while having proven much capability across a wide range of problem areas, is still just one of many practical approaches. Increasingly, were starting to see news and research showing the limits of deep learning capabilities, as well as some of the downsides to the deep learning approach. So are peoples enthusiasm of AI tied to their enthusiasm of deep learning, and is deep learning really able to deliver on many of its promises?

The Origins of Deep Learning

AI researchers have struggled to understand how the brain learns from the very beginnings of the development of the field of artificial intelligence. It comes as no surprise that since the brain is primarily a collection of interconnected neurons, AI researchers sought to recreate the way the brain is structured through artificial neurons, and connections of those neurons in artificial neural networks. All the way back in 1940, Walter Pitts and Warren McCulloch built the first thresholded logic unit that was an attempt to mimic the way biological neurons worked. The Pitts and McCulloch model was just a proof of concept, but Frank Rosenblatt picked up on the idea in 1957 with the development of the Perceptron that took the concept to its logical extent. While primitive by todays standards, the Perceptron was still capable of remarkable feats - being able to recognize written numbers and letters, and even distinguish male from female faces. That was over 60 years ago!

Rosenblatt was so enthusiastic in 1959 about the Perceptrons promises that he remarked at the time that the perceptron is the embryo of an electronic computer that [we expect] will be able to walk, talk, see, write, reproduce itself and be conscious of its existence. Sound familiar? However, the enthusiasm didnt last. AI researcher Marvin Minsky noted how sensitive the perceptron was to small changes in the images, and also how easily it could be fooled. Maybe the perceptron wasnt really that smart at all. Minsky and AI researcher peer Seymour Papert basically took apart the whole perceptron idea in their Perceptrons book, and made the claim that perceptrons, and neural networks like it, are fundamentally flawed in their inability to handle certain kinds of problems notably, non-linear functions. That is to say, it was easy to train a neural network like a perceptron to put data into classifications, such as male/female, or types of numbers. For these simple neural networks, you can graph a bunch of data and draw a line and say things on one side of the line are in one category and things on the other side of the line are in a different category, thereby classifying them. But theres a whole bunch of problems where you cant draw lines like this, such as speech recognition or many forms of decision-making. These are nonlinear functions, which Minsky and Papert proved perceptrons incapable of solving.

During this period, while neural network approaches to ML settled to become an afterthought in AI, other approaches to ML were in the limelight including knowledge graphs, decision trees, genetic algorithms, similarity models, and other methods. In fact, during this period, IBMs DeepBlue purpose-built AI computer defeated Gary Kasparov in a chess match, the first computer to do so, using a brute-force alpha-beta search algorithm (so-called Good Old-Fashioned AI [GOFAI]) rather than new-fangled deep learning approaches. Yet, even this approach to learning didnt go far, as some said that this system wasnt even intelligent at all.

Yet, the neural network story doesnt end here. In 1986, AI researcher Geoff Hinton, along with David Rumelhart and Ronald Williams, published a research paper entitled Learning representations by back-propagating errors. In this paper, Hinton and crew detailed how you can use many hidden layers of neurons to get around the problems faced by perceptrons. With sufficient data and computing power, these layers can be calculated to identify specific features in the data sets they can classify on, and as a group, could learn nonlinear functions, something known as the universal approximation theorem. The approach works by backpropagating errors from higher layers of the network to lower ones (backprop), expediting training. Now, if you have enough layers, enough data to train those layers, and sufficient computing power to calculate all the interconnections, you can train a neural network to identify and classify almost anything. Researcher Yann Lecun developed LeNet-5 at AT&T Bell Labs in 1998, recognizing handwritten images on checks using an iteration of this approach known as Convolutional Neural Networks (CNNs), and researchers Yoshua Bengio and Jrgen Schmidhube further advanced the field.

Yet, just as things go in AI, research halted when these early neural networks couldnt scale. Surprisingly very little development happened until 2006, when Hinton re-emerged onto the scene with the ideas of unsupervised pre-training and deep belief nets. The idea here is to have a simple two-layer network whose parameters are trained in an unsupervised way, and then stack new layers on top of it, just training that layers parameters. Repeat for dozens, hundreds, even thousands of layers. Eventually you get a deep network with many layers that can learn and understand something complex. This is what deep learning is all about: using lots of layers of trained neural nets to learn just about anything, at least within certain constraints.

In 2010, Stanford researcher Fei-Fei Li published the release of ImageNet, a large database of millions of labeled images. The images were labeled with a hierarchy of classifications, such as animal or vehicle, down to very granular levels, such as husky or trimaran. This ImageNet database was paired with an annual competition called the Large Scale Visual Recognition Challenge (LSVRC) to see which computer vision system had the lowest number of classification and recognition errors. In 2012, Geoff Hinton, Alex Krizhevsky, and Ilya Sutskever, submitted their AlexNet entry that had almost half the number of errors as all previous winning entries. What made their approach win was that they moved from using ordinary computers with CPUs, to specialized graphical processing units (GPUs) that could train much larger models in reasonable amounts of time. They also introduced now-standard deep learning methods such as dropout to reduce a problem called overfitting (when the network is trained too tightly on the example data and cant generalize to broader data), and something called the rectified linear activation unit (ReLU) to speed training. After the success of their competition, it seems everyone took notice, and Deep Learning was off to the races.

Deep Learnings Shortcomings

The fuel that keeps the Deep Learning fires roaring is data and compute power. Specifically, large volumes of well-labeled data sets are needed to train Deep Learning networks. The more layers, the better the learning power, but to have layers you need to have data that is already well labeled to train those layers. Since deep neural networks are primarily a bunch of calculations that have to all be done at the same time, you need a lot of raw computing power, and specifically numerical computing power. Imagine youre tuning a million knobs at the same time to find the optimal combination that will make the system learn based on millions of pieces of data that are being fed into the system. This is why neural networks in the 1950s were not possible, but today they are. Today we finally have lots of data and lots of computing power to handle that data.

Deep learning is being applied successfully in a wide range of situations, such as natural language processing, computer vision, machine translation, bioinformatics, gaming, and many other applications where classification, pattern matching, and the use of this automatically tuned deep neural network approach works well. However, these same advantages have a number of disadvantages.

The most notable of these disadvantages is that since deep learning consists of many layers, each with many interconnected nodes, each configured with different weights and other parameters theres no way to inspect a deep learning network and understand how any particular decision, clustering, or classification is actually done. Its a black box, which means deep learning networks are inherently unexplainable. As many have written on the topic of Explainable AI (XAI), systems that are used to make decisions of significance need to have explainability to satisfy issues of trust, compliance, verifiability, and understandability. While DARPA and others are working on ways to possibly explain deep learning neural networks, the lack of explainability is a significant drawback for many.

The second disadvantage is that deep learning networks are really great at classification and clustering of information, but not really good at other decision-making or learning scenarios. Not every learning situation is one of classifying something in a category or grouping information together into a cluster. Sometimes you have to deduce what to do based on what youve learned before. Deduction and reasoning is not a fort of deep learning networks.

As mentioned earlier, deep learning is also very data and resource hungry. One measure of a neural networks complexity is the number of parameters that need to be learned and tuned. For deep learning neural networks, there can be hundreds of millions of parameters. Training models requires a significant amount of data to adjust these parameters. For example, a speech recognition neural net often requires terabytes of clean, labeled data to train on. The lack of a sufficient, clean, labeled data set would hinder the development of a deep neural net for that problem domain. And even if you have the data, you need to crunch on it to generate the model, which takes a significant amount of time and processing power.

Another challenge of deep learning is that the models produced are very specific to a problem domain. If its trained on a certain dataset of cats, then it will only recognize those cats and cant be used to generalize on animals or be used to identify non-cats. While this is not a problem of only deep learning approaches to machine learning, it can be particularly troublesome when factoring in the overfitting problem mentioned above. Deep learning neural nets can be so tightly constrained (fitted) to the training data that, for example, even small perturbations in the images can lead to wildly inaccurate classifications of images. There are well known examples of turtles being mis-recognized as guns or polar bears being mis-recognized as other animals due to just small changes in the image data. Clearly if youre using this network in mission critical situations, those mistakes would be significant.

Machine Learning is not (just) Deep Learning

Enterprises looking at using cognitive technologies in their business need to look at the whole picture. Machine learning is not just one approach, but rather a collection of different approaches of various different types that are applicable in different scenarios. Some machine learning algorithms are very simple, using small amounts of data and an understandable logic or deduction path thats very suitable for particular situations, while others are very complex and use lots of data and processing power to handle more complicated situations. The key thing to realize is that deep learning isnt all of machine learning, let alone AI. Even Geoff Hinton, the Einstein of deep learning is starting to rethink core elements of deep learning and its limitations.

The key for organizations is to understand which machine learning methods are most viable for which problem areas, and how to plan, develop, deploy, and manage that machine learning approach in practice. Since AI use in the enterprise is still continuing to gain adoption, especially these more advanced cognitive approaches, the best practices on how to employ cognitive technologies successfully are still maturing.

See the original post here:
Are We Overly Infatuated With Deep Learning? - Forbes

Prior to Payne Bauer's arrival, Sherando High School football coach Bill Hall didn't keep track of tackles for losses.

But his middle linebacker's penchant for bringing down ball carriers in the backfield at an astonishing rate changed all of that.

And this season, Bauer kept the calculators whirring at a rate even higher than before. Bauer racked up 41 tackles behind the line of scrimmage, including 12 sacks, for the Warriors, who rebounded from injuries to advance to the Region 4C playoffs.

Bauer's phenomenal play earned him several postseason accolades including his second consecutive Winchester Star Defensive Player of the Year honor.

The Payne Train racked up 93 tackles on the season with nearly half of them coming behind the opposition's line of scrimmage.

If you have somebody in the teens that raises an eyebrow at a regional or state meeting, Hall said. You don't ever hear of someone having 20 and he has 41 and he had 36 last year. Things like that are crazy.

But to hear it from Bauer, you'd be crazy to think it was all him. The humble 6-foot-2, 232-pounder gives all of the credit to the guys who play five yards in front of him, often seeming like opposing running backs fell into his lap.

It's mostly the guys in front of you, said Bauer when asked about how he racks up so many tackles behind the line of scrimmage. Everyone always talks about linebackers at Sherando, but usually it's the D-linemen. They don't get any love, but they deserve it more than anyone.

People like Caleb McLee, Skyler Taylor-Goode, he was a monster, and Brett Shockey they absorbed two blockers, he added. When two guys are blocking one dude, that always leaves linebackers free. The defensive linemen to me are the most selfless people on the football team because they eat everything for the linebackers and that's why the linebackers make plays.

While those linemen are excellent football players, Hall says the speedy and powerful Bauer often doesn't give himself enough credit in the equation to success.

He's so modest, Hall said. He just busts things off like it's not a big deal. He's been brought up the right way. He really appreciates people serving each other. He's a service type of kid. It doesn't go unnoticed on him that people are doing stuff that he can be successful. Some kids never get that, but he understands the team component of a ballgame and how it works together.

That being said, and those guys do a great job in front of him, you don't have 41 tackles for a loss without being really, really, really, really good. You just can't plug-and-play and put somebody in there and have 41 tackles for a loss. That doesn't happen.

Bauer has several schools, including Division I programs, who have offered him scholarships. He's weighing his options until February's signing date, but is sure of one thing.

My biggest thing is I want to play at a football school, Bauer said. There are some schools that are basketball schools, but I want to play where everyone loves football.

So what makes Bauer so good?

It's not just one thing, but a combination that leads to his success.

While he'd like to be stronger, Bauer is a bear. Often one arm is enough to bring down a ballcarrier. And when he hits somebody they rarely fall forward.

The thing about Payne is that he doesn't give himself credit for the type of athlete he is and the strength he possesses, Hall said. He is just naturally physical strong. He does wear his passion on his sleeve, but he applies his passion into his development to be really great. His work in the weight room is really off the charts. He doesn't take days off in the weight room.

Every lift we do in the weight room is football oriented, Bauer said. I guess I have grip strength and I took down guys with my hand. That played a big part because I wouldn't have been able to do that last year and the year before.

Bauer is willing to work at his craft.

He spends many hours watching film and working with linebackers coach John Minteer on reading offenses. And when he makes a read, he attacks.

This season, the Warriors needed Bauer to exert a little more pressure on the quarterback and he responded with his career-high sack total and forced many hurries.

I watch a lot of film, Bauer said. I like to apply myself in that. This year, I kind of became an end rusher guy. I tried watching videos of guys coming off the edge like T.J. Watt, Von Miller, J.J. Watt. Khalil Mack is my favorite. I try to watch guys like that and do what they do.

Bauer is coachable. Minteer and defensive coordinator Jake Smith challenged Bauer throughout his career and he continued to a improve.

He likes feedback, Hall said. I think that's a common characteristic of great players is that they want feedback. You can coach him hard.

And Bauer is not one to enjoy his own press clippings and accolades. While he may have several eye-popping plays during a game, Bauer knows that is not a true measure of what he accomplished.

He points to the season-opener against James Wood as a perfect example. In that contest, he registered three sacks and had a 78-yard touchdown run.

Film doesn't lie, Bauer said. I thought I played really well against James Wood because I had two or three sacks and I had a nice long touchdown run, but I looked at the film and I played terrible. It was probably one of the worst games because I played like so lazy. It was not a good game.

He's his harshest critic, Hall said. In games, he'll be like, 'Coach I'm not playing very well.' Sometimes that could be accurate to his level, but it would be really good for other people.

And while Bauer had many great moments, it's the plays that he didn't make that haunt him.

I don't think I played as well as I should have, he said of his entire season. I can remember plays where I missed. Those plays are, 'How did they get me?' I try to see what I could have done better watching film on Saturday or Monday. I try to find what I could have done better to make the play.

And Bauer carries that desire to succeed over to the practice field. He doesn't believe in doing things halfway or at half the speed. He's a gamer on Friday because of Monday-Thursday.

Payne understood that the way he practiced transferred over to the way he played in a game, Hall said. He had to play at a speed and attack level for a certain amount of time so that would transfer over so he could sustain that type of speed for that long of a time on a Friday night.

He is wide open in practice. You would have to pull him off in practice from a physicality standpoint. He was always full speed. I don't want to discredit the work he does in practice because he's one of the better practice players we've ever had. He embraced how what he does in practice transfers to what he is in the ballgame. Kids don't always make the correlation.

Bauer puts the whole package on the field and it's his intangibles that make him special.

He is really gifted at being able to make adjustments in the game, Hall said. When you watch him play football, an educated person watches him and says, 'That cat knows what he's doing.' Based of the blocking scheme that happens, he fits right. That's because of film study, being coachable, him understanding how he fits based off a blocking scheme and how we fit as a defense.

The longer you're in the business the more you appreciate it, Hall added. You realize they don't come along all of the time. We've been fortunate to have those guys. But again all of those guys, whoever you want to talk about Brian Barlow, George Aston, Dylan Rivers are cut from the same cloth that they all were with their preparation phase toward the game of football. They poured themselves into it. Then you add that with genetics, then it's a recipe for being the player that [Payne] is.

Bauer has come a long way from choosing not to play football his freshman season at Sherando. Bauer envisioned himself as a baseball player and he is a very good one having led the Warriors with a .439 average last season.

But, Hall knew he would make a good football player and tasked quarterback and baseball standout Hunter Entsminger to bring Bauer to practice. All Bauer has done since then is lead the Warriors for three consecutive seasons in tackles, racking up 309 with 99 of those being for losses.

Bauer has garnered a pair of Class 4 Northwestern District and Region 4C Defensive Player of the Year honors and has been named the the VHSL's first team defense the past two seasons.

While Hall had no doubt that Bauer had the potential to be a stellar player, others didn't and that motivated Bauer.

I think I just wanted to prove everyone wrong, he said. I can just remember people saying, 'You're not going to be good. You're not going to be on varsity or whatever. They're just going to put you on JV.' My first day, Coach Hall put me on varsity. I just wanted to prove everyone wrong that doubted me.

Bauer and Entsminger became fast friends. Bauer said he and Entsminger, now on a baseball scholarship at James Madision University, talk each day.

This season Entsminger was often on the field with Bauer symbolically. Entsminger's No. 8 was part of Bauer's attire.

Everyone knows that Hunter is my best friend, Bauer said. When they started handing out towels our first game, they were like, 'Hey, here and tossed it to me.' I guess I can blame some of my success to Hunter because I wore a little piece of him.

And like Entsminger was a big brother to him, Bauer returned the favor this season to sophomore quarterback Dylan Rodeffer. Bauer did many things together with Rodeffer, who was pressed into service because of an injury to starter Chacai Campbell.

Hall wasn't surprised. He's seen how much teammates mean to Bauer. It's the little things like eating pizza together at CiCi's on Wednesday nights that mean so much.

He wouldn't have missed that for anything, Hall said. If it had been three hours away, he would have driven three hours to get there because it was that important to him.

Bauer hardly can believe his football career is done at Sherando.

I'm going to miss it a lot, he said. It kind of makes me sad that it's over. I've got this family here and I'm going to be able to talk to the guys still. It's not even the sport I will miss the most. It's the brotherhood and the bonding we have.

When asked about how much he would miss Bauer, Hall became emotional.

All of those guys, you spend so much time with them, said a teary Hall. The other thing is that people don't know their story. You tell a story, but they don't know the rest of the story. I think the parts that I know that other people don't know if you only knew how this kid has risen to where he's at, your level of appreciation would go off the charts. He'll be an unbelievable success story when the story comes out. Down the road you will be like, 'Are you serious?' Now you know the rest of the story.

You are always going to miss the football player. Other players come, but again they are not going to be Payne. That's the nature of the business.

Read more here:
Football Defensive Player of the Year: Sherando's Payne Bauer - The Winchester Star

STARKVILLE, Miss.A Mississippi State University partnership with the Fort Worth Zoo has hatched the first of more than 30 metamorphosed toadlets produced through in vitro fertilization.

A Puerto Rican crested toad named Olaf, hatched at the Fort Worth Zoo this year, is what one might call a work of art. ART, or assisted reproductive technologies, developed by scientists in the universitys Mississippi Agricultural and Forestry Experiment Station and the Forest and Wildlife Research Center, helps amphibians like the Puerto Rican crested toad, considered a threatened species by the U.S. Fish and Wildlife Service.

The technologies include hormone therapies, sperm cryopreservation and in vitro fertilization. MSU also is home to the countrys only National Amphibian Genome Bank, a repository of cryopreserved sperm from approximately 10 of the worlds most threatened and endangered amphibian species.

Carrie Vance, assistant research professor in the Department of Biochemistry, Molecular Biology, Entomology and Plant Pathology who co-leads the project, said Olaf is an example of how ART helps increase the genetic diversity and sustainability of populations of threatened amphibians.

Olaf represents the first time we used cryopreserved sperm from a wild Puerto Rican crested toad as a new genetic line to be combined with an egg from a captive female, said Vance, who also is a MAFES scientist at MSU. Whats more is that both of Olafs parents have since died of natural causes so Olaf is truly the last of this particular genetic line.

Vance pointed out that while cryopreserving sperm from wild males, researchers have been able to use hormone therapies to assist breeding. As opposed to other methods, this technique enables researchers to collect sperm without killing the animal, which Vance believes will result in a wider adoption of the practice.

Previously to introduce genetics from wild individuals into a captive population, the animals were brought into captivity, then paired, and often would never breed anyway. If the collection of sperm from testes macerates was needed, it would require the animal be euthanized, Vance said. This new method means we can collect the sperm and release the specimen back into the wild.

Vance said ART is one facet of a larger species survival plan, which includes steps such as habitat restoration, disease control and establishing an assurance colony in captivity.

ART helps when amphibians have difficulty breeding in captivity. Typically, amphibian breeding is cued by environmental factors such as day length, rainfall and temperature, which are things that can be difficult to control in a 10-gallon aquarium. When they dont breed, the genetic lines are lost, and a zoos entire assurance colony can collapse.

Vance said it comes down to overriding the environmental cues and synchronizing the timing of the actual breeding, noting that while it takes males only hours to generate sperm, it can take weeks for females to produce eggs.

The hormone therapy overrides the environmental factors to trigger the production of reproductive hormones, which cause sperm and egg release. Sperm cryopreservation holds the sperm in perpetuity until the eggs are ready for synchronization.

Vance has partnered with Andy Kouba, professor and head of the Department of Wildlife, Fisheries and Aquaculture in MSUs College of Forest Resources and scientist in the Forest and Wildlife Research Center, for more than 20 years developing innovative reproductive technologies for threatened and endangered species.

The researchers also have applied ART to the Mississippi gopher frog, considered one of the most endangered in the U.S. Their pioneering work resulted in thousands of Mississippi gopher frogs being produced by zoos around the country and reintroduced into their native habitat.

Many of the techniques we use on species like the Puerto Rican crested toad were developed using the Mississippi gopher frog, Kouba said. The Mississippi gopher frog was the first endangered species ever produced from frozen sperm. The offspring are still alive and have subsequently produced a second generation of offspring, considered another first of its kind.

Kouba said seeing the applied conservation in action and being able to reintroduce animals back into the wild is what excites him most about the work.

Globally, it is estimated that 30-40 percent of amphibians are threatened with extinction. In the U.S. that number is closer to 50 percent, Kouba said. Our assisted reproductive technologies have led to millions of tadpoles from threatened and endangered species being released into the wild across many species.

Kouba added that amphibians serve as indicator species for the health of their surrounding ecosystems.

They are the canary in the coal mine, Kouba said. Anything happening in the environment soaks through their permeable skin. Also, they have two life stages, an early aquatic stage and a terrestrial stage, which lets scientists know what is happening in two different environments. As an indicator species, it is important to understand why amphibian populations are disappearing and to try and help the populations recover.

In addition to the Puerto Rican crested toad and the Mississippi gopher frog, other species the team focuses on include the Boreal toad, Houston toad, Chiricahua leopard frog and various species of salamanders. Graduate students on the project include doctoral student Allison Julien of Scotts Valley, California; masters student Isabella Burger of Prattville, Alabama; and Kristen Counsell, a spring 2018 masters graduate of Cedar Falls, Iowa. Masters student Amanda Gillis of Fallston, Maryland, and research associate Emmet Guy of Oxford contribute to the labs salamander research.

Support for the Olaf project includes funding from Disneys Conservation Endowment Fund and the Association of Zoos and Aquariums. Longtime funding partner, the Institute of Museums and Library Services, supported the early development of this work and currently sponsors the labs salamander research. Morris Animal Foundation also has provided previous financial support.

For more on the Mississippi Agricultural and Forestry Experiment Station, visit http://www.mafes.msstate.edu. For more on the Forest and Wildlife Research Center, visit http://www.fwrc.msstate.edu.

MSU is Mississippis leading university, available online at http://www.msstate.edu.

Read the rest here:
One-of-a-kind toad born through MSU pioneering technology that's saving threatened species - Winston County Journal

ENGLAND TOUR OF SOUTH AFRICA, 2019-20

Dean Elgar was dismissed off the first ball of the Boxing Day Test. Getty

Too much about the state of South Africa's batting was captured by a photographer staying in the frame for longer than Dean Elgar at Centurion on Thursday (December 26). With the bowler, the fielders, the batters and the umpires all locked, loaded and leaning in for the first ball of the men's Test series against England, Christiaan Kotze - a shutterbug of 30 years' professional standing - was scuttling out of view with seemly haste. He never made it. At least, not on foot.

Kotze stepped onto an upturned cover still slippery with the morning's dew, and something in his right knee gave way. So there he lay bang in the middle of the sightscreen: a snapper stricken, a picture of pain, hopelessly over-exposed and desperately trying to crawl out of the scene. He didn't get far. First Chris Gaffaney and Paul Reiffel sidled his way officiously. Then several England players moseyed over, like cats gingerly stalking a dustbin. Eventually medics arrived bearing a stretcher, and - to sympathetic applause - carried poor Kotze to his exit, stage left.

Then, four minutes later than advertised, "Jimmy, Jimmy, Jimmy, Jimmy Anderson", as per the Barmy Army, nudged his bespoke Rolls Royce engine into gear at this level for the first time in almost six months and set off towards the crafty, crusty, cussed Elgar, the bearer of South Africa's standard through so many scrapes and scraps - who dabbed his front foot inside the line of a leg-side loosener unworthy of Anderson, looked to smear it to midwicket, and instead gave Jos Buttler the kind of catch he has been taking, even in his sleep, since he was 10 years old.

Elgar's stay of one minute, one ball made him the eighth man overall and the second South African - after Jimmy Cook, who edged Kapil Dev to Sachin Tendulkar at second slip at Kingsmead in November 1992 - to be dismissed with the first delivery of a rubber. It also added a layer of lustre to Anderson's return from a calf injury to become the ninth player and the first specialist fast bowler to earn 150 Test caps.

Few players are as unconcerned with hiding their feelings as Elgar, who had taken only a few paces from the wicket before he stopped and fumed with frustration for significantly longer than he had batted. Perhaps, as a member in excellent standing of the glass-half-empty club, he was convinced of what would would happen in the ensuing hours. He had, of course, seen it all before.

Aiden Markram, Zubayr Hamza, Faf du Plessis and debutant Dwaine Pretorius all got themselves in only to get out to an England attack that recognised the ally that is a South African Highveld pitch, and bowled accordingly. Vernon Philander adhered to the first half of that equation and might yet not fall victim to the second bit, what with only one wicket standing.

Another debutant, Rassie van der Dussen, stopped at the boundary to perform an aggressive range of warm-up stretches and twirled his bat like a baton as the shiny blare of a brass band accompanied him to the wicket. After 189 first-class innings he was finally on parade. The first 33 balls he faced - for six runs - suggested he had submitted himself utterly to the philosophy of Jacques Kallis, South Africa's new batting consultant. The 34th, bowled by Sam Curran, a 1930s matinee idol of a left-armer complete with a helmet of flying hair the colour of incandescence itself, streaked across Van der Dussen, took the outside edge and flew into Joe Root's hands at first slip.

Where had we seen that before? Seven wickets fell to catches in the arc that followed tentative strokes - testament to the doubt England's bowlers caused, by using the seam effectively, in players whose minds were pre-strewn with misgivings in the wake of five consecutive Test defeats wrapped around a dismal World Cup.

Quinton de Kock was among the edgily departed, but that was all he shared with them. A widely held misconception is that de Kock is not, to put it politely, cricket's greatest thinker. Closer to the truth is that actions speak louder than either words or thoughts, and that they matter exponentially more. De Kock is a man of action. It's not that he doesn't think. It's that he has little need to think because he was born knowing what needs to be done and how to do it. His analysis of Elgar's dismissal was as pithy as it was brief: "It's not the greatest way of getting out."

There is something Kallisesque in De Kock's imperviousness to pressure, in his refusal to have his reaction to threatening deliveries and near misses dictated by his opponents, the conditions or the match situation. His reality is not what the rest of us might recognise as reality. That is what makes him play as if his team were 397/4 when he took guard and not, as they were, 97/4.

De Kock might several times have taken himself out of England's way before he reached 50, and his sixth century was there for the plucking when he steered Curran to Buttler. As he trooped off, he slung his bat across his shoulders and hung a hand from either end, which made him look like an ox under a yoke. He had tamed himself, and he knew it. But at least the theatre of competition was, from South Africa's perspective, a significantly better place when he left than when he arrived.

Overall, there wasn't a lot of Kallis' icy intent on view and even less of head coach Mark Boucher's bulldog spirit. It would be unfair to judge their impact on the evidence of their first day at the office. As De Kock said after stumps, "It's only day one with them." But judged they will be nonetheless. Too much of how South Africa have batted in recent innings was in evidence, and in this scenario continuity is not at all a good thing. So far, not so different, and thus not so good.

But, as Christiaan Kotze could have told them, what fells you today might fuel your tomorrow. Not many years ago he was shot in the side in the line of duty while covering a protest. He was unable to get up and get on with it on Thursday, but he knows what it takes to come back strong. He's taking Friday off, but look out for him around the boundary on Saturday. If his compatriots need an inspiration, he'll be there.

Cricbuzz

See the original post:
No sign of the Boucher, Kallis genetic code in SA's batting as yet - Cricbuzz - Cricbuzz

life-style,

Body odour is caused by bacteria breaking down sweat and is largely linked to the apocrine glands, said Melbourne dermatologist Dr Hope Dinh. Men have more of these glands in the armpit which make them more prone to body odour. The surge in hormones during the teenage years can affect odour output in both males and females. Some population groups even naturally produce less odour. "East Asians who have nearly complete loss of typical body odour, when compared to people of African and European descent, have significantly less of the characteristic axillary odorants and variants in the ABCC11 gene, which is expressed and localised in apocrine sweat glands," said Dr Dinh. Individuals with certain medical conditions are more susceptible to having body odour. These can include diabetes, urinary tract infections, an overactive thyroid, kidney and liver dysfunction and a genetic disorder called trimethylaminuria. Certain medications can also cause sweating in some people; including antidepressants, triptans for migraines, NSAIDs (non -steroidal anti-inflammatory medications) and opioids. Lifestyle factors in what we eat and how we live can also contribute. Dr Dinh identified common reasons for body odour which include: "With the boom in activewear as a fashion statement, it can mean more body odour due to the trapped heat and synthetic fabrics. Hence try not to sit too long in your yoga pants after a session; hit the showers quickly after exercise and have a spare change of clothes," said Dr Dinh. "I recommend changing footwear or socks regularly throughout the day if they are damp. Just this simple measure can reduce the malodour significantly." How should we be washing ourselves? Dr Dinh's tips: 1 Keep the armpits clean: Wash them regularly using anti-bacterial soap, and the number of bacteria will be kept low, resulting in less body odour. 2 Deodorant or antiperspirant: Deodorants make the skin more acidic, making it more difficult for bacteria to thrive. An antiperspirant block the sweating action of the glands, resulting in less sweating. 3 Drying thoroughly between skin folds after washing is very important, otherwise any trapped moisture can lead to bacteria or thrush thriving and hence lead to body odour. 4 People can apply corn starch or talc powder to the worst areas of sweating after bathing. Despite the tips offered for effective bathing, Dr Dinh said it can be a common misconception that body odour is a result of poor hygiene. If people are concerned they should consult their GP to rule out any kind of medical cause for their issues such as diabetes or thyroid problems. The GP can refer the patient to a dermatologist for further treatment if needed. Treatment can start with topical treatment such as prescription-strength anti-perspirant, or a deodorant. More treatments for more advanced cases include medication, surgery on the apocrine gland or nerves that feed the sweat glands, and the use of medical devices on feet, hands and underarms. "Miradry utilizes thermal energy that targets and eliminates the sweat and odour glands in your underarm. Once those glands are eliminated, they do not grow back." "Hyperhidrosis Botulinum toxin (anti-sweat) treatments are easily performed in clinic by a dermatologist. Botulinum toxin injections are approved for hyperhidrosis affecting the armpits. It is a safe and effective treatment all done in clinic," said Dr Dinh. Anti-sweating injections for excessive sweating conditions are highly effective, long lasting and receive a partial rebate from Medicare.

https://nnimgt-a.akamaihd.net/transform/v1/crop/frm/K5E4qWjbHGabfQuRuq4ELE/6422c706-5bb7-4ee9-ad03-f50eefb1d493.jpg/r3_287_5614_3457_w1200_h678_fmax.jpg

December 26 2019 - 9:18AM

Body odour is caused by bacteria breaking down sweat and is largely linked to the apocrine glands, said Melbourne dermatologist Dr Hope Dinh. Men have more of these glands in the armpit which make them more prone to body odour. The surge in hormones during the teenage years can affect odour output in both males and females. Some population groups even naturally produce less odour.

"East Asians who have nearly complete loss of typical body odour, when compared to people of African and European descent, have significantly less of the characteristic axillary odorants and variants in the ABCC11 gene, which is expressed and localised in apocrine sweat glands," said Dr Dinh.

Individuals with certain medical conditions are more susceptible to having body odour. These can include diabetes, urinary tract infections, an overactive thyroid, kidney and liver dysfunction and a genetic disorder called trimethylaminuria. Certain medications can also cause sweating in some people; including antidepressants, triptans for migraines, NSAIDs (non -steroidal anti-inflammatory medications) and opioids.

Lifestyle factors in what we eat and how we live can also contribute.

Dr Dinh identified common reasons for body odour which include:

"With the boom in activewear as a fashion statement, it can mean more body odour due to the trapped heat and synthetic fabrics. Hence try not to sit too long in your yoga pants after a session; hit the showers quickly after exercise and have a spare change of clothes," said Dr Dinh.

"I recommend changing footwear or socks regularly throughout the day if they are damp. Just this simple measure can reduce the malodour significantly."

How should we be washing ourselves? Dr Dinh's tips:

1 Keep the armpits clean: Wash them regularly using anti-bacterial soap, and the number of bacteria will be kept low, resulting in less body odour.

2 Deodorant or antiperspirant: Deodorants make the skin more acidic, making it more difficult for bacteria to thrive. An antiperspirant block the sweating action of the glands, resulting in less sweating.

3 Drying thoroughly between skin folds after washing is very important, otherwise any trapped moisture can lead to bacteria or thrush thriving and hence lead to body odour.

4 People can apply corn starch or talc powder to the worst areas of sweating after bathing.

Despite the tips offered for effective bathing, Dr Dinh said it can be a common misconception that body odour is a result of poor hygiene. If people are concerned they should consult their GP to rule out any kind of medical cause for their issues such as diabetes or thyroid problems. The GP can refer the patient to a dermatologist for further treatment if needed.

Treatment can start with topical treatment such as prescription-strength anti-perspirant, or a deodorant. More treatments for more advanced cases include medication, surgery on the apocrine gland or nerves that feed the sweat glands, and the use of medical devices on feet, hands and underarms.

OFFENSIVE: Body odour can be off-putting for co-workers.

"Miradry utilizes thermal energy that targets and eliminates the sweat and odour glands in your underarm. Once those glands are eliminated, they do not grow back."

"Hyperhidrosis Botulinum toxin (anti-sweat) treatments are easily performed in clinic by a dermatologist. Botulinum toxin injections are approved for hyperhidrosis affecting the armpits. It is a safe and effective treatment all done in clinic," said Dr Dinh.

Anti-sweating injections for excessive sweating conditions are highly effective, long lasting and receive a partial rebate from Medicare.

Read the rest here:
What causes body odour and why do some have it worse? - The Canberra Times

A new Defense Department memo warns that direct-to-consumer (DTC) genetic testing "could expose personal and genetic information, and potentially create unintended security consequences and increase risk to the joint force and mission." The memo adds that "there is increased concern in the scientific community that outside parties are exploiting the use of genetic data for questionable purposes, including mass surveillance and the ability to track individuals without their authorization or awareness. Until notified otherwise, [Department of Defense] military personnel are advised to refrain from the purchase and/or use of DTC genetic services."

Way too late.

Some 26 million Americans have already used DTC genetic testing services to gain insights into their health risks and ancestry. By one estimate, it was possible in 2018 to use these DTC databases to personally identify 140 million Americans of European descent using genetic information uploaded by themselves and their relatives. It is projected that as many as 100 million Americans will have used such genetic testing services in the next two years. At that point, almost any American could be identified by matching their DNA to that of their relatives in online databases. In other words, we users of genetic testing services have been voluntarily creating "a de facto national DNA database." To use the hoary, but apt cliche: The Pentagon is closing the barn door well after the horses have stampeded out.

Given how pervasive and much more easily deployed facial recognition technology is, I can hardly wait to read the DoD memo warning troops not to post their photos on Facebook.

While it is not possible to rein in genetic and facial recognition surveillance technologies in dictatorships like China and Russia, Americans should urgently seek to do so through legislation in Congress.

Disclosure: Any would-be criminal relatives are on notice that my DNA test results are publicly available.

See the rest here:
Pentagon Memo Warns Against Identification Risks of Consumer Genetic Testing - Reason

Nathan Anderson and two of his four sons, Griffin, 10, and Brennan, 4, have been diagnosed with a rare mutation on their Runx1 gene. Besides causing easy bruising, excessive bleeding and a tendency to develop hematomas, the mutation predisposes them to certain blood cancers. Wife and mother, Joy Anderson, has become an outspoken advocate for the condition and her family.

As Joy Anderson tucked her four boys into their beds one recent evening, she asked each of them to reflect on the difficult year the family has had.

Oldest son Griffin, 10, expressed anger and sadness, especially because he isnt allowed to play contact sports anymore.

Maxwell, 9, said hes worried about his siblings and parents.

Nolan, 8, said hes tried to be more compassionate, as you never know what someone is facing.

And then theres sweet-yet-rambunctious Brennan. At just 4 years old, he doesnt really understand whats been going on, only that hes had many doctors visits and needle pricks lately.

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A little over a year ago, the Anderson family received news that rocked their world: Brennan had tested positive for the Runx1 gene mutation.

It was the third such blow for the family, who hails from Arlington, a village in Hancock County outside of Findlay, in just a few short months. Griffin first received the same diagnosis in August 2018, followed by their father, Nathan, in November that year and now Brennan.

Maxwell and Nolan, fortunately, do not share the mutation.

"Its like a 50-50 chance of being passed down," Joy said. "In that case, I felt like we were a living statistic."

But their situation is anything but routine: Only 120 individuals in the world have been diagnosed with the mutation.

Visible symptoms of the disease, which causes platelet abnormalities, include easy bruising, excessive bleeding and a tendency to develop hematomas. But the most distressing part is its predisposition (40% to 70%) to certain blood cancers.

"I felt like I couldnt breathe, like I was having a panic attack," the 41-year-old mother said of the diagnoses. "I tried to Google everything to learn more about it, and there isnt really much out there."

Three people she loves dearly half her family now must be followed regularly by a hematologist and undergo bone marrow biopsies annually to monitor for cancer.

"Little did I know we would have such a domino effect after we all got tested," she said.

***

The Andersons had spent much of the first nine years of Griffins life trying to figure out what caused his bruising and the huge goose eggs that would form on his body.

Sure, he was an active boy, but with each small accident, his parents couldnt help notice the extreme results that sometimes landed him in the hospital to ensure his blood had clotted.

Doctors in Toledo offered a general diagnosis of low platelets and a blood disorder when Griffin was 3 and prescribed special medication to use during surgeries.

He began seeing various hematologists at Nationwide Childrens Hospital. However, the family still had few answers until a doctor suggested genetic testing in August 2018.

Thats when the Andersons met Elizabeth Varga, a genetic counselor at Childrens, who counseled them what results might yield.

Four weeks after Griffin had blood drawn for the genetic tests, Varga called to say they found something.

Runx1 is a gene involved in the making of blood cells. If there is an abnormality, it can impair the production of platelets, which help blood clot.

Patients are predisposed "to something called myelodysplastic syndrome, which is essentially a pre-leukemia state that can evolve and change to be a cancer of the blood," Varga told the family.

Typically, thats acute myeloid leukemia, which is most common in adults over 60, but the Runx1 mutation increases the risk for AML in all ages.

"So basically from the time of birth youre kind of set up to have that evolution," Varga said. "However, not all patients that have a Runx1 abnormality will ever get cancer right now we dont have a great way to gauge who will and who wont."

To deliver this news to Joy was very difficult, said Varga, who has three young sons.

Each phone call, Joy said, felt like a "sucker punch," as she struggled to learn what this would mean for their family.

***

In the days leading up to Griffin and Brennans first bone marrow biopsy in December 2018, the elder brother watched YouTube videos of the minor but uncomfortable procedure, which is the best way to monitor changes.

"I was interested in it," the fifth grader said. "I want to be an ER doctor when I grow up."

Joy and Nathan Anderson said its a bit of an odd blessing that two sons have the diagnosis as they dont have to be alone.

"I usually tell Brennan to be brave and we can do it," Griffin said, adding that his youngest brothers silly antics calm his nerves.

The mutation presents differently in Griffin and Brennan. Both bruise easily, but its much more pronounced in Griffin.

Brennan has dealt with a slew of pulmonology issues asthma, seven bouts with pneumonia that may be linked with the gene mutation.

Discovered only 20 years ago, very little research has been done on the mutation, making it difficult to discern what symptoms are caused by it, said Katrin Ericson, executive director of the Runx1 Research Program, a California-based nonprofit group that funds research and provides patient support.

Earlier this year, Joy Anderson was the first patient family representative to speak at the organizations annual conference. Shes become very active in increasing awareness of the Runx1 mutation, which is underdiagnosed, Ericson said.

Based on epidemiological estimates, between 2,000 and 18,000 people in the United States could be living with it.

Ericson said the organization is thrilled that, in May, the National Institutes of Health launched the first longitudinal, natural history study of Runx1. The Andersons are one of 25 or so families participating, and they traveled to Bethesda, Maryland, in June for testing.

"These patients have really been struggling with this most of their lives," Ericson said. "They had no idea they had this mutation. Maybe they were misdiagnosed at first."

There can be guilt felt by parents for passing it unknowingly to their children, and Joy said her husband feels this way.

Nathan his parents tested negative never experienced symptoms, he said. However, looking back, routine blood work often showed low platelet counts.

"Shortly after the diagnosis, I was at a charity event for work and I was chopping wood," Nathan said. "I noticed that I had bruises up and down my arm."

During his first bone marrow biopsy at Ohio State Universitys Arthur G. James Cancer Hospital last December, doctors discovered he had myelodysplastic syndrome (MDS), or pre-leukemia. Hes unsure what this means for the future, except continued monitoring.

Nathan and his two sons will travel to the NIH in the summer for follow-up biopsies. (Griffin and Brennan had a second one in June with zero changes.)

The boys also have blood drawn every few months.

"Our hope is if we do more frequent surveillance for MDS, that we will hopefully be able to be preemptive," Varga said. "If we do see any progression, the only cure right now is a bone marrow transplant."

However, a bone marrow transplant which requires chemotherapy and a lifetime of immunity-suppressing drugs is a procedure that wouldnt be done without good reason, Varga said.

***

One of the biggest challenges of having a genetic disorder, Joy and Nathan agreed, is explaining it to others.

"Some people think were dying and some think were getting chemotherapy," said Nathan, who has taken up running to cope with the diagnosis and keep himself healthy. "Others think its not a big deal."

People have questioned the necessity of "putting our kids through all this," Joy said. Others have said that everyone would find something wrong if they did genetic testing, she said.

"Were trying to monitor and learn more," she said. "Im all about being proactive. I feel like what if we never even did anything about it, and then one day this has progressed to leukemia and its so far into it, we cant do anything to help."

That attitude, Varga said, represents a shift shes detected.

"Previous generations, there was much more of I dont want to know," Varga said. "There was a fear of stigma or discrimination, but younger generations are more embracing (of) knowledge and being powerful."

***

For now though, the Anderson boys will continue to climb on top of their swing set and run in the field behind their house. Theyll fish and ride bikes and wrestle.

"Theyre boys still, and we want them to live a normal life," Nathan said. "We dont stop."

Griffin has recently fallen in love with playing drums and though his parents dont always appreciate the noise, theyre thankful Griffin has found an outlet other than sports.

The family is thankful for the power the knowledge of this diagnosis ultimately brings them, and with how strong it has shown the six of them to be.

"Its a reminder of just how precious life is," Nathan said. "You might live a long life or you might run into complications. Its a constant reminder to live every day to the fullest."

award@dispatch.com

@AllisonAWard

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Ohio family faces uncertainty as 2 sons, husband diagnosed with rare genetic disorder - The Columbus Dispatch

Its been a banner year for First Opinion. We published nearly 500 essays written by more than 600 authors from industry, academia, government, and private life in the United States and beyond. They took on the mundane, like the implications of the Elijah E. Cummings Lower Drug Costs Now Act (aka H.R. 3), and the fanciful, like why we need a Public Domain Day to highlight when drugs go off patent.

Authors addressed the opioid crisis, patent thickets, the personal toll imposed by drug shortages, drug costs, deaths of despair, the oxymoron of having a waiting room in an emergency department, shoddy production of generic drugs by foreign manufacturers, the burgeoning use of artificial intelligence and machine learning in new drug research and development and health care, and much, much more.

Here are the five most widely read First Opinions of 2019. If you didnt get to read them when they first appeared, now is as good a time as any:

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1. 23andMe had devastating news about my health. I wish a person had delivered it Dorothy Pomerantz thought it would be fun to have her DNA analyzed, so she sent her spit to 23andMe. A link in an email took her to the companys website, where she learned she was at very high risk of developing breast and ovarian cancer. Im not the first person to get surprising and terrible news from an at-home genetic testing company. With the increasing popularity of 23andMe, and other companies like it, I wont be the last. But she wished she had gotten the news from a person.

2. Time in range: a new way for people with diabetes to monitor blood sugar More and more people with diabetes are using continuous glucose meters, which measure blood sugar every few minutes. That information offers a new way to evaluate how well someone is controlling his or her diabetes: time in range. The University of Washingtons Dr. Lorena Alarcon-Casas Wright explains how it works.

3. Our child received a devastating diagnosis before she was born. We decided to protect her Allison Chang learned that, at 15 weeks of gestation, her daughter had severe malformations due to trisomy 18, a deadly genetic condition. She and her husband could not protect our daughter from trisomy 18, but we could shield her from any pain or agony that would come with it.

4. I have spinal muscular atrophy. Critics of the $2 million new gene therapy are missing the point Nathan Yates has lived with spinal muscular atrophy for all 30 years of his life. He answers critics upset over the $2.1 million cost of Zolgensma, a new drug to treat the condition, and asks them to take into account its long-term benefits.

5. Ghost networks of psychiatrists make money for insurance companies but hinder patients access to care When Jack Turban started his training to become a psychiatrist, he went looking for a therapist of his own. What he found were ghost networks of mental health providers.

And heres a bonus: Published in July 2018, the essay Physicians arent burning out. Theyre suffering from moral injury was the third most widely read First Opinion of 2019.

On to 2020!

Read more here:
5 most widely read First Opinions of 2019 - STAT

This article, written byMichael Mackley, Dalhousie University, originally appeared on The Conversation and is republished here with permission:

Youve likely heard about direct-to-consumer DNA testing kits. In the past few years, at-home genetic testing has been featured in the lyrics of chart-topping songs, and has helped police solve decades-old cold cases, including identifying the Golden State Killer in California.

Even if you dont find a DNA testing kit under your own Christmas tree, theres a good chance someone you know will.

Whether youre motivated to learn about your health or where your ancestors came from, it is important to understand how these tests work before you spit in the tube.

While exciting, there are things that these genetic testing kits cannot tell users and important personal implications that consumers should consider.

Health, traits and ancestry kits

My main area of research is around clinical genome sequencing, where we look through all of a persons DNA to help diagnose diseases. With a PhD in genetics, I often get questions from friends and family about which direct-to-consumer genetic test they should buy, or requests to discuss results. Most questions are about two types of products: ancestry and health kits.

The most popular ancestry kit is from AncestryDNA. These kits are aimed at giving users insight into where their ancestors might be from. They can also connect users with family members who have used the service and have opted into having their information shared. Another option is Living DNA, which has a smaller dataset but provides more precise information on the U.K. and Ireland.

The most popular health kit is from 23andMe. Depending on the users preference, results include information on predispositions for diseases such as diabetes and Alzheimers, as well as on the likelihood of having certain traits such as hair colour and taste. This company also offers ancestry analysis, as well as ancestry and trait-only kits that dont provide health information. The kit offered by the newer MyHeritage DNA also provides a combined ancestry and health option.

There are other kits out there claiming to evaluate everything from athletic potential to relationship compatibility. But gift-buyers beware: for most of these, in contrast to those above, the evidence is seriously lacking.

How these tests work

For all of these tests, customers receive a kit in the mail. The kits contain instructions for collecting a saliva sample, which you mail back to the company for analysis.

During this analysis, these popular tests do not look at the entire genome. Instead, they employ single nucleotide polymorphism (SNP) genotyping. As humans we all share 99.9 per cent of our DNA. SNPs are essentially what is left: all of the points at which we can differ from our neighbour, making us unique. SNP genotyping looks at a subset of these sites to survey the users genome.

These SNPs are then compared to reference datasets of individuals with known conditions or ancestry. Most results are based on the SNPs shared with a given group. For example, if your results say that you are 42 per cent Southeast Asian, its because 42 per cent of your SNPs were most likely to have come from a group in the reference dataset labelled Southeast Asian. The same goes for traits and health conditions.

How they differ from clinical tests

Direct-to-consumer genetic tests are not a substitute for clinical assessment. The methods used differ dramatically from what is done to diagnose genetic diseases.

In a clinical setting, when suspicion of a genetic condition is high, entire genes are often analyzed. These are genes where we understand how changes in the DNA cause cellular changes that can cause the disease. Furthermore, clinical assessment includes genetic counselling that is often key to understanding results.

In contrast, findings from direct-to-consumer genetic tests are often just statistical links; there is commonly no direct disease-causing effect from the SNPs.

Users may interpret a result as positive, when the risk increase is only minimal, or entirely false. These tests can also give false reassurance because they do not sequence genes in their entirety and can miss potentially harmful variants.

Before you spit in a tube, stop and think

These tests are exciting: they introduce new audiences to genetics and get people thinking about their health. Theyre also helping to build vast genetic databases from which medical research will be conducted.

But for individual users, there are important caveats to consider. Recent reports have questioned the accuracy of these tests: identical twins can receive different results. Furthermore, a lack of diversity in the reference data has caused particular concern regarding accuracy of results for ethnic minorities.

There are also concerns about the way these tests emphasize racial categories that science considers to be social constructs and biologically meaningless.

A recent paper in the British Medical Journal suggests four helpful questions for users to consider. First, users should ask themselves why they want the test. If it is to answer a medical question, then they should speak with their doctor. Users should also think about how they might feel when they receive results containing information they would rather not know.

Users should also consider issues around security and privacy. It is important to read the fine print of the service youre using, and determine whether youre comfortable sharing personal information, now and in the future.

In Canada, policies around genetics have not always kept up with the science. At present, direct-to-consumer genetic testing is unregulated. And, although Canadians have legislative protections against genetic discrimination, those laws are being challenged in the courts, and could change.

Finally, it may also be worth discussing DNA testing with relatives. We share half of our genome with our immediate family members, and smaller fractions with more distant relatives. Genetic results not only affect us, but our family.

Bottom line: Its all for fun

Some users may feel they learn more about themselves. For others, results may bring people closer together not a bad outcome for the holiday season.

At the end of the day, these genetic testing kits are for entertainment: they should not be used to assess health risk in any meaningful way.

If you have any questions related to your health or a genetic disease, discuss these with your family doctor or a suitable health-care professional.

Michael Mackley, Junior Fellow, MacEachen Institute for Public Policy and Governance; Medical Student, Dalhousie University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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DNA tests might be a fun holiday gift, but beware of the hype - HalifaxToday.ca

In this last full stock trading week of 2019, let's tip our collective hat to the markets. It was quite a year for equities, with major indexes rising to fresh all-time highs on a frequent basis and some of our favorite top stocks reaching the same milestone.

That said, we're seeing a few late-in-the-game stumbles from certain companies listed on the various exchanges. Here's a pair that were nursing a Santa hangover on the day after Christmas.

Image source: Getty Images

Shares of Qiagen(NYSE:QGEN) were eviscerated by almost 21% on Boxing Day, following news that a hoped-for takeover is not going to happen.

The Germany-based company announced the day before Christmas that it has opted to go it alone rather than sell itself to an outside entity. This isn't the gift many investors were hoping for from the genetic testing specialist -- in November it temptingly announced that it had been the target of "several conditional, non-binding indications of interest for a full acquisition." The stock promptly took off like a rocket.

So Thursday's fall to Earth is not surprising. It also brings back concerns investors had before the takeover speculation. After all, Qiagen's latest reported quarter indicated some bumps in the road, with a second consecutive reduction of full fiscal year sales guidance (blamed on difficulties in the Chinese market) and concerns about leadership following the departure of longtime CEO Peer Schatz.

Although the company recently signed up for a potentially promising, long-term collaboration with Illuminain next-generation sequencing diagnostics, we have no pertinent details on the arrangement. Given that, plus uncertainties over Qiagen's other business areas and the pesky matter of the CEO job, it might be best to leave the stock alone just now.

Planet Earth wasn't the only home of downer stocks on Thursday. Far up in space, Virgin Galactic Holdings (NYSE:SPCE) saw a downward trajectory of more than 4% across the day.

It's very possible that a deal announced on Thursday by SpaceX, Virgin Galactic's rival in otherworldly exploration, was the culprit. Privately held SpaceX won a contract from NASA to supply parts for exploratory vehicles.

This isn't a planet-sized deal, as it only totals about $7.5 million. Besides, although Virgin Galactic's ambitions overlap those of SpaceX, the former company is not a maker of components. However, the deal does indicate that there is revenue to be made in the private space sector, and Virgin Galactic still isn't making much (or any, if you don't count deposits for potential space flights by well-to-do tourists).

Somewhat surprisingly, for a company that could be a poster boy for speculative early-stage investments, Virgin Galactic has quite a few admirers in the investment community. Earlier this month, it racked up its third buy recommendation from a noted investment bank (Morgan Stanley, to be exact), which feels it has a lot of upside if it can manage to become the first viable space tourism operator.

That's an awfully long stretch to wait, though, given the monster costs and unproven model of this particular business. I think Virgin Galactic will be a fun stock to watch, but I personally am not about to invest in it.

Read more here:
2 Stocks That Tumbled on Thursday - The Motley Fool

According to news articles published in early December, Veritas Genetics, a Massachusetts-based company that hoped to lower the cost of whole-genome sequencing, is suspending its U.S. operations because of a lack of investment. Articles theorize that the decreased funding was driven mainly by new CFIUS regulations and heightened CFIUS scrutiny.

Early in December 2019, Veritas announced that its adverse financing situation had forced the suspension of its U.S. business. Veritas has stated that it is assessing potential paths forward, and there are rumors that one such path is the sale of the company. Veritas will no longer sell its tests, which include genetic testing for diseases and cancers (such as the BRCA test), in the United States. Veritas will continue to operate and sell its tests outside the United States.

Veritas first launched in 2014, and since 2015 it had raised $50 million in financing. Major investors included Chinese companies, such as Lilly Asia Ventures, which invested $10 million into the company, and Simcere Pharmaceutical. However, there has been increased scrutiny in the past two years for transactions that involve Chinese investors, especially when sensitive personal information, such as genetic information, is at stake. This year, for example, CFIUS forced iCarbonX, the Chinese, majority owner of U.S. company PatientsLikeMe, to divest its stake in the U.S. company.

According to news reports, recent CFIUS activity may have scared away not only Chinese investors but also non-Chinese investors reluctant to invest in a company with Chinese ownership. Non-Chinese investors may fear that Veritass Chinese ownership will lead to increased CFIUS scrutiny of any investment into Veritas, regardless of the investors nationality. Investors may also worry that CFIUS scrutiny could delay their return on investment if their firms are forced to stall business to address CFIUSs concerns.

No doubt the proposed CFIUS regulations from September also concern foreign investors: the proposed regulations explicitly target U.S. companies that maintain or collect sensitive personal data of U.S. citizens. While most sensitive personal data only triggers the proposed regulations if the U.S. business maintains or collects such data on greater than one million individuals, companies with genetic data are considered to be covered businesses no matter how many individuals are involved. Thus, companies like Veritas will always fall under CFIUS jurisdiction if a foreign person would acquire certain rights in the company. These rights include:

Several genetic and biopharmaceutical companies expressed concern in public comments to the regulations that the proposed regulations, specifically including all genetic data in the definition of sensitive personal data, would stymie foreign investment in these companies. Several companies argued that the Department of the Treasury should revise the proposed CFIUS regulations to require that genetic data be identifiable. Companies often are in possession of anonymized genetic information, which these companies argued does not pose a risk to national security. We await publication of the final regulations and whether CFIUS will make any changes to the definition of sensitive personal data, particularly as it pertains to genetic information. It is to be seen whether U.S. companies in other industries will face similar funding obstacles as foreign investors grow more wary of CFIUS.

Originally posted here:
Veritas, a US Genetic Sequencing Company, Suspends US Operations Due to Decreased Funding; CFIUS Thought to be Leading Cause - Lexology

Check out the companies making headlines before the bell:

Boeing Boeing remains on watch, after new documents reviewed by a congressional panel revealed what's being called "very disturbing" revelations regarding the grounded 737 Max jet, according to a congressional aide quoted by Reuters. Boeing issued a statement saying it had proactively brought the documents to the FAA and Congress, and said the tone and content do not reflect "the company we are and need to be".

Amazon.com Amazon said the holiday shopping season broke all prior records, with "billions" of items ordered worldwide and "tens of millions" Amazon devices purchased.

Qiagen Qiagen said it decided against a sale of the company following a review. The Netherlands-based genetic testing firm said it determined that operating as a stand-alone business is its best option. Qiagen said it had gotten several indications of interest, with reports saying one of those potential bids came from medical device maker Thermo Fisher Scientific.

PayPal PayPal will continue to pursue potential takeover targets in 2020, according to Chief Financial Officer John Rainey. He told the Wall Street Journal there are many acquisition opportunities in the payments sector, with PayPal targeting transactions in the $1 billion to $3 billion range.

Exxon Mobil, Chevron These and other oil stocks could get a boost as oil prices touch their highest in more than 3 months, boosted by a report showing lower U.S. crude inventories.

TiVo The digital video recorder maker said it would pay a termination fee of $50.8 million to technology licensing company Xperi under certain circumstances, if their planned all-stock merger does not take place. There are other termination scenarios, according to an SEC filing, in which Xperi would pay TiVo $44 million.

KKR The private equity firm is buying digital content platform Overdrive from Rakuten for an undisclosed amount. The Japanese e-commerce company purchased Overdrive in 2015 for $410 million.

Spectrum Pharmaceuticals The drug maker said its experimental treatment for non-small cell lung cancer missed its primary goal in a mid-stage trial.

Read this article:
Stocks making the biggest moves premarket: Boeing, Amazon, PayPal & more - CNBC

A research team from the University of Houston has found a way to use the stem cells found in fat and guide it to become a pacemaker-like cell, according to a new study.

We are reprogramming the cardiac progenitor cell and guiding it to become a conducting cell of the heart to conduct electrical current, said study co-author Bradley McConnell, associate professor of pharmacology, in a press release

The team, publishing the study in the Journal of Molecular and Cellular Cardiology, worked on converting adipogenic mesenchymal stem cells, which reside within fat cells, into cardia progenitor cells. The ensuing cardiac progenitor cells can be programmed to aid heartbeats as a sinoatrial node (SAN), which is part of the electrical cardiac conduction system.

The researchers used what they called a standard screening strategy to test for reprogramming factors for converting human cardiac progenitor cells into pacemaker-like cells. According to their study results, the authors observed expressions of many pacemaker-specific genes, including CX30.2, KCNN4, HCN4, HCN3, HCN1, and SCN3b. The authors wrote that SHOX2, HCN2, and TBX5 (SHT5) combinations of transcription factors were much better candidate(s) in driving cardiac progenitor cells into pacemaker-like cells than other combinations and single transcription factors.

Results of this study show that the SHT5 combination of transcription factors can reprogram CPCs into Pacemaker-like cells, they wrote in their conclusion. SHT5 may be used as a potential stem cell therapy for sick sinus syndrome (SSS) and for other cardiac conduction diseases.

Original post:
The Next Generation of Biologic Pacemakers? New Discovery in Stem Cells from Fat Creates Another Alternative Treatment - DocWire News

University of Houston's C.T. Bauer College of Business has received its second largest donation to benefit its entrepreneurship program.

The Cyvia and Melvyn Wolff Center for Entrepreneurship, which was recently ranked the top undergraduate entrepreneurship program in the country, received the $13 million gift from its namesake foundation The Cyvia and Melvyn Wolff Family Foundation and the state of Texas is expected to match an additional $2 million, bringing the total impact to $15 million.

"Our family is deeply committed to the ideals of entrepreneurship," says Cyvia Wolff in a news release. "Our business personified everything that it means to be an entrepreneur. The skills, the thinking, the mindset are fundamental to success for business leaders today and in the future. On behalf of my late husband, we are truly honored to ensure the entrepreneurial legacy not only endures but remains accessible for students. We are truly honored to be part of this program and university."

The money will be used to create three endowments for the program. The Dave Cook Leadership Endowment, named for the center's director, Dave Cook, will be created and funded with $7 million of the donation to support leadership within the organization. For $4 million, the center will create the Wolff Legacy Endowment, which aims to increase students involved in the center, as well as the companies coming out of the program. The last $2 million will be used to create the Cyvia and Melvyn Wolff Endowed Chair(s)/Professorship(s) in Entrepreneurship. This initiative will support research and community outreach.

"We are passionate about entrepreneurship and how it can forever change students' lives," says Bauer Dean Paul A. Pavlou in the release. "We seek to further promote entrepreneurship as a university-wide, even citywide effort, by collaborating within and across the university in a multitude of areas, such as technology, health care, arts and sports."

The program was created in the mid '90s and was later renamed after Cyvia and Melvyn Wolff in 2007, and has seen great success over the past decade. In that time, Wolff students have created 1,270 businesses, with identified funding of just over $268 million. According to the release, the program has been ranked in the top two spots of the Princeton Review's top undergraduate entrepreneurship programs for nine of the past 12 years.

"Entrepreneurship is crucial for the future of our country, as well as our city and state," says UH President Renu Khator in the release. "We are proud to be at the forefront of work around entrepreneurial training and research. The uniqueness of our program has and continues to make it the model program. This extraordinary gift ensures our leadership in this space will continue and will support the creation of businesses, change communities and impact our students' lives."

At UH, 2,500 students take at least one entrepreneurship course a year, and more than 700 students complete certificate programs.

"What we are doing is transformative in the lives of students, mentors and stakeholders in a way that elevates everyone towards excellence," Cook, who was named the director of the program in 2017, says in the release. "The impact of this gift allows us to remain the leader and to move forward with confidence, purpose and permanence."

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3 innovative research projects coming out of the University of Houston - InnovationMap

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10170423

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

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The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market 2019-2027 / Trends, Growth, Opportunities And Top Key - Market Research Sheets

Data Bridge Market ResearchLorea is a professional writer with expertise in industry research.

She had worked with many corporates prior to joining research

Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market,By Applications (Eurodegenerative Disorders, Autoimmune Diseases, Cancer & Tumors, Cardiovascular Diseases), By Product (Blood Pressure (BP) Monitoring Devices, Pulmonary Pressure Monitoring Devices, Intracranial Pressure (ICP) Monitoring Devices), By End User (Hospitals And Ambulatory Surgical Center), By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2025

The Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market is expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

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In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks. The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease. According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe. With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Major Market Drivers and Restraints:

Introduction of novel autologous stem cell based therapies in regenerative medicine

Reduction in transplant associated risks

Prevalence of cancer and diabetes in all age groups

High cost of autologous cellular therapies

Lack of skilled professionals

Browse Detailed TOC, Tables, Figures, Charts and Companies @https://www.databridgemarketresearch.com/toc/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market

The global autologous stem cell and non-stem cell based therapies market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of autologous stem cell and non-stem cell based therapies market for global, Europe, North America, Asia Pacific and South America.

Some of the major players operating in the global autologous stem cell and non-stem cell based therapies market are Antria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more pleaseRequest an Analyst Callor can drop down your inquiry.

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Industry Trends and Forecast to 2025 - News Distribute

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States. Meredith Rizzo/NPR hide caption

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

"My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out," Gray says. "That's when they found out that I was having my first crisis."

It was Gray's first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

"Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And it's a deep pain. I can't touch it and make it better," says Gray. "Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Gray's journey through this medical experiment, which is being watched closely for some of the first hints that changing a person's genes with CRISPR could provide a powerful new way to treat many diseases.

"This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line," says Dr. Francis Collins, director of the National Institutes of Health.

"To be able to take this new technology and give people a chance for a new life is a dream come true," Collins says. "And here we are."

Doctors removed bone marrow cells from Gray's body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that it's far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

"We have to be cautious. It's too early to celebrate," Frangoul says. "But we are very encouraged so far."

Collins agrees.

"That first person is an absolute groundbreaker. She's out on the frontier," Collins says. "Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation."

This is the story of Gray's journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe. Meredith Rizzo/NPR hide caption

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

It's July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

"Nice to meet y'all," she says.

Gray is just days away from her birthday, which she'll be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

"It's the right time to get healed," says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company. Meredith Rizzo/NPR hide caption

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

"It's horrible," Gray says. "When you can't walk or, you know, lift up a spoon to feed yourself, it gets real hard."

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells don't carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

"I didn't feel normal. I couldn't do the regular things that every other kid could do. So I had to be labeled as the sick one."

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Gray's heart and other parts of her body. Gray knows that many patients with sickle cell don't live beyond middle age.

"It's horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen," she says. "Who wouldn't worry?"

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says. Meredith Rizzo/NPR hide caption

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says.

Gray married and had children. But she hasn't been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

"It's scary. And it affected my oldest son, you know, because he's older. So he understands. He started acting out in school. And his teacher told me, 'I believe Jemarius is acting out because he really believes you're going to die,' " Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; there's no cure for most patients.

"It was just my religion that kind of kept me going," Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

"I was excited," Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

"CRISPR technology has a lot of potential use in the future," Frangoul says.

To try to treat Gray's sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. It's a protein that fetuses make in the womb to get oxygen from their mothers' blood.

"Once a baby is born, a switch will flip on. It's a gene that tells the ... bone marrow cells that produce red cells to stop making fetal hemoglobin," says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain. Ed Reschke/Getty Images hide caption

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

"We are trying to introduce enough ... fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where it's supposed to," Frangoul says.

Then on July 2, after extracting Gray's cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

"They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath," Gray says. "So that was a little scary, tough moment for me."

After that moment passed, Gray says, she cried. But her tears were "happy tears," she adds.

"It was amazing and just kind of overwhelming," she says, "after all that I had went through, to finally get what I came for."

The cells won't cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the disease's complications.

"This opens the door for many patients to potentially be treated and to have their disease modified to become mild," Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her "supercells."

"They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family," she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer. Meredith Rizzo/NPR hide caption

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Concerns about risk

Other doctors and scientists are excited about the research. But they're cautious too.

"This is an exciting moment in medicine," says Laurie Zoloth, a bioethicist at the University of Chicago. "Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases."

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

"This a brand-new technology. It seems to work really well in animals and really well in culture dishes," she says. "It's completely unknown how it works in actual human beings. So there are a lot of unknowns. It might make you sicker."

Zoloth is especially concerned because the research involves African Americans, who have been mistreated in past medical studies.

Frangoul acknowledges that there are risks with experimental treatments. But he says the research is going very slowly with close oversight by the Food and Drug Administration and others.

"We are very cautious about how we do this trial in a very systematic way to monitor the patients carefully for any complications related to the therapy," Frangoul says.

Gray says she understands the risks of being the first patient and that the study could be just a first step that benefits only other patients, years from now. But she can't help but hope it works for her.

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville. Meredith Rizzo/NPR hide caption

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville.

She imagines a day when she may "wake up and not be in pain" and "be tired because I've done something not just tired for no reason." Perhaps she could play more with her kids, she says, and look forward to watching them grow up.

"That means the world to me," Gray says.

It could be many weeks or even months before the first clues emerge about whether the edited cells are safe and might be working.

"This gives me hope if it gives me nothing else," she says in July.

Heading home at last

About two months later, Gray has recovered enough to leave the hospital. She has been living in a nearby apartment for several weeks.

Enough time has passed since Gray received the cells for any concerns about immediate side effects from the cells to have largely passed. And her gene-edited cells have started working well enough for her immune system to have resumed functioning.

So Gray is packing. She will finally go home to see her children in Mississippi for the first time in months. Gray's husband is there to drive her home.

"I'm excited," she says. "I know it's going to be emotional for me. I miss the hugs and the kisses and just everything."

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss. Meredith Rizzo/NPR hide caption

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss.

Gray is wearing bright red glittery eye shadow. It matches her red tank top, which repeats "I am important" across the front.

She unzips a suitcase and starts pulling clothes from the closet.

"My goodness. Did I really bring all this?" she says with a laugh.

Before Gray can finish packing and depart, she has to stop by the hospital again.

"Are you excited about seeing the kids?" Frangoul says as he greets her. "Are they going to have a big welcome sign for you in Mississippi?"

Turns out that Gray has decided to make her homecoming a surprise.

"I'm just going to show up tomorrow. Like, 'Mama's home,' " she says, and laughs.

After examining Gray, Frangoul tells her that she will need to come back to Nashville once a month for checkups and blood tests to see if her genetically modified cells are producing fetal hemoglobin and giving her healthier red blood cells.

"We are very hopeful that this will work for Victoria, but we don't know that yet," Frangoul says.

Gray will also keep detailed diaries about her health, including how much pain she's experiencing, how much pain medication she needs and whether she needs any blood transfusions.

"Victoria is a pioneer in this. And we are very excited. This is a big moment for Victoria and for this pivotal trial," Frangoul says. "If we can show that this therapy is safe and effective, it can potentially change the lives of many patients."

Gray hopes so too.

See more here:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Health News - NPR

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

Picture: Supplied.

WASHINGTON, United States - In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta-thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

CURES

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face gruelling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in the hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

**BIOTERRORISM **

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his ex-communication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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2019: The year gene therapy came of age - Eyewitness News

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

Meredith Rizzo/NPR

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out, Gray says. Thats when they found out that I was having my first crisis.

It was Grays first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And its a deep pain. I cant touch it and make it better, says Gray. Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Grays journey through this medical experiment, which is being watched closely for some of the first hints that changing a persons genes with CRISPR could provide a powerful new way to treat many diseases.

This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line, says Dr. Francis Collins, director of the National Institutes of Health.

To be able to take this new technology and give people a chance for a new life is a dream come true, Collins says. And here we are.

Doctors removed bone marrow cells from Grays body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

We are very, very excited, says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that its far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

We have to be cautious. Its too early to celebrate, Frangoul says. But we are very encouraged so far.

Collins agrees.

That first person is an absolute groundbreaker. Shes out on the frontier, Collins says. Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation.

This is the story of Grays journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcares Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

The study took place at HCA Healthcares Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Meredith Rizzo/NPR

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

Its July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

Nice to meet yall, she says.

Gray is just days away from her birthday, which shell be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

Its the right time to get healed, says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

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In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

Meredith Rizzo/NPR

Its horrible, Gray says. When you cant walk or, you know, lift up a spoon to feed yourself, it gets real hard.

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells dont carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

I didnt feel normal. I couldnt do the regular things that every other kid could do. So I had to be labeled as the sick one.

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Grays heart and other parts of her body. Gray knows that many patients with sickle cell dont live beyond middle age.

Its horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen, she says. Who wouldnt worry?

Gray says she understands the risks involved in the treatment. This gives me hope if it gives me nothing else, she says.

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Gray says she understands the risks involved in the treatment. This gives me hope if it gives me nothing else, she says.

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Gray married and had children. But she hasnt been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

Its scary. And it affected my oldest son, you know, because hes older. So he understands. He started acting out in school. And his teacher told me, I believe Jemarius is acting out because he really believes youre going to die, Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; theres no cure for most patients.

It was just my religion that kind of kept me going, Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

I was excited, Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

CRISPR technology has a lot of potential use in the future, Frangoul says.

To try to treat Grays sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. Its a protein that fetuses make in the womb to get oxygen from their mothers blood.

Once a baby is born, a switch will flip on. Its a gene that tells the bone marrow cells that produce red cells to stop making fetal hemoglobin, says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcares TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells dont carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

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Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells dont carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

Ed Reschke/Getty Images

We are trying to introduce enough fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where its supposed to, Frangoul says.

Then on July 2, after extracting Grays cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath, Gray says. So that was a little scary, tough moment for me.

After that moment passed, Gray says, she cried. But her tears were happy tears, she adds.

It was amazing and just kind of overwhelming, she says, after all that I had went through, to finally get what I came for.

The cells wont cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the diseases complications.

This opens the door for many patients to potentially be treated and to have their disease modified to become mild, Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her supercells.

They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family, she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

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Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Meredith Rizzo/NPR

Concerns about risk

Other doctors and scientists are excited about the research. But theyre cautious too.

This is an exciting moment in medicine, says Laurie Zoloth, a bioethicist at the University of Chicago. Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases.

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

Read more:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Invest Records

Protein Associated with Leukemia May Lead to Targeted Therapy for Currently Incurable Acute Lymphoblastic Leukemia

ALL is a form of blood cancer that primarily affects children and young people and causes large quantities of malignant progenitor cells to build in a patients blood instead of healthy white blood cells. This is often caused by 2 chromosomes fusing together to create new abnormal genes that disrupt the system controlling normal blood development. Because of this process, certain types of leukemia are extremely resistant and unable to be cured with intensive chemotherapy or stem cell transplantation.

Researchers analyzed a protein called TCF3-HLF, which is typically associated with this type of leukemia and does not occur naturally. It is produced through the fusion of 2 chromosomes and contains elements of transcription factors, which activate the transcription of certain genes.

The analysis revealed that TCF3-HLF activates a whole range of genes, but it does so in the wrong contextat the wrong point in the blood development process. The formation of malignant white blood cells is then triggered, causing leukemia.

The study authors also discovered that the abnormal protein does not act alone, but instead gathers more than 100 other proteins around it, which helps to activate the genes. The researchers investigated the function of the individual proteins in the genetic machinery and used it to identify key elements that could be targeted through therapy.

Using the CRISPR/Cas9 method, researchers detached the specific parts they had identified from the machinery and found 11 critical factors that are crucial to the build-up of malignant abnormal blood cells in leukemia.

One of the essential components now identified is the protein EP300, a cofactor that boosts gene activation. The researchers used a new kind of substance called A-485, known to bind to EP300 and inhibit its activity. When A-485 was administered to human leukemia cells, the malignant cells died off.

The study authors noted that it is possible to stop the fundamental driving force behind the leukemia directly and thus develop a targeted type of therapy. Given that other forms of leukemia are caused by similar mechanisms, it may also be possible to identify a common denominator for developing new drugs to combat cancer.

REFERENCE

New approach to treating incurable leukemia in children discovered [press release]. University of Zurich. BioPortfolio website. Published November 24, 2019. https://www.bioportfolio.com/news/article/4148041/New-approach-to-treating-incurable-leukemia-in-children-discovered.html. Accessed December 4, 2019.

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Protein Associated with Leukemia May Lead to Targeted Therapy for Currently Incurable Acute Lymphoblastic Leukemia - Pharmacy Times

Throughout the year we have published the views of company executives, government regulators, industry analysts and scientists on a variety of topics and, in our popular annual feature, we include a selection of these that paints a picture of the significant events that shaped 2019. The major talking point was on the capital markets front where investors turned their backs on the biopharmaceutical sector for most of the year returned big time in the final quarter. M&As helped sparked life, with blockbuster deals taking place throughout the year, particularly in the red hot cell and gene therapy space, with an exclamation point added this week as Roche Holding AG followed up the completion of its $4.8 billion acquisition of Spark Therapeutics Inc. with the announcement of a blockbuster deal with Sarepta Therapeutics Inc., underscoring big pharmas appetite for gene therapy technologies.

Our favorite quote from the year

The Wright brothers showed that you could fly a plane, but it wasnt very far, and it wasnt very safe. Thats where cell therapy is now.At a scientific session at the American Society of Hematology meeting on gene editing, Wendell Lim, professor and chair of cellular and molecular pharmacology at the University of California, San Francisco, and the co-founder of Cell Design Labs, discussed his laboratorys work that uses gene editing to improve cell therapies, specifically CAR T cells

Capital markets

The biopharmaceutical sector headed into 2019 on a negative note, with most of the damage inflicted in December 2018 a month that will go down as one of the worst for equities, with the Dow Jones Industrial Average stumbling by almost 9% and the Nasdaq Composite performing just as poorly, dropping 9.5%.

Summing up the situation, the Cowen and Co. biotechnology analyst team wrote in its monthly biotech thermometer report to clients: "Investors are shell-shocked by the depreciation that was so quick, so dramatic, and so close to the end of the year. Sentiment is worse than we can remember over the last five years, and in fact many investors have developed a bear market mindset."

Fast forward 12 months and the curtain is now closing on a year where, for many months, the biopharmaceutical industry languished and company executives undoubtedly were wondering, during that difficult period, whether there would be a reversal of fortunes.

The answer came in the fourth quarter. To the surprise of industry pundits, public biopharmaceutical companies turned the corner and are on a major upswing, with the sector outperforming the general market in final frame with a 23% upswing year-to-date.

Reflecting on the change, Cowen and Co. analysts, writing in their monthly Biotech Thermometer noted, "Those who stuck with the sector through the famine of Q3 have feasted on a cornucopia of returns during the first two months of Q4."

Bear to bull

Most analysts speculated at the beginning of the year that the recipe to create an upswing in the sectors fortunes would be tied to an increase in M&A activity. SVB Leerink analyst Joseph Schwartz was certainly prescient in a note to investors earlier this year pointing to gene therapy companies as being prime targets. "With increasing pricing pressure and the emphasis on 'value' from payers, we believe curative gene therapy programs represent ideal acquisition targets for large pharma or biotech companies looking to diversify their product portfolios and reduce their reliance on price hikes."

RBC Capital Markets analyst Brian Abrahams, writing in the 2019 RBC Biotech Outlook Report, struck a similar note: "For 2019, we are more neutral vs. 2018, as growth and competitive headwinds, alongside more tangible manifestations of drug cost containment, potentially mitigate increasingly attractive valuations among larger-caps and what we expect will be increasing M&A activity and enthusiasm backstopping the smids."

Industry reels from FDA commissioner resignation

The announcement from Scott Gottlieb that he was resigning as FDA commissioner to return to the American Enterprise Institute in April took the industry by surprise, and the news weighed heavily on biopharmaceutical equities, with the BioWorld Biopharmaceutical index falling almost 4% on the news.

"Scott has helped us to lower drug prices, get a record number of generic drugs approved and onto the market, and so many other things. He and his talents will be greatly missed."President Donald Trump's tweet in response to Scott Gottlieb's announcement that he was resigning as FDA commissioner

The industry had to wait until December for a replacement when the U.S. Senate voted 72-18 to confirm Stephen Hahn as the commissioner of the FDA, providing the agency with another commissioner with a deep background in oncology. Secretary of Health and Human Services Alex Azar thanked the Senate for prioritizing the candidates nomination, adding that the development will be a major boost to the already rapid pace of the Presidents aggressive public health agenda.

Setting the agenda

Looking at the BioWorld Biopharmaceutical index it reveals that it did attain an almost 12% increase in value by February. Catalyzing the jump was the positive vibe that emanated from the January J.P. Morgan 37th annual health care conference, which every year sets the industrys agenda. From the market reaction to the upcoming plans laid bare by presentations from many of the leading biotechnology and big pharma companies in attendance, it appeared that the sector came out ahead.

Business development

"Once again we're seeing a lot of billion-dollar deals and they tend to be preclinical-stage deals that have multiple assets. It's a trend that started in 2016."David Thomas, vice president of industry research and analysis for the Biotechnology Innovation Organization, on its 2019 Emerging Therapeutic Company Trend Report, which showed that the number of global R&D-stage licensing deals surged by 107% in 2018 over the prior year

"The shift away from larger market drugs to more niche indications reflects the change in R&D focus of biopharma companies."Roger Humphrey, executive managing director and leader of JLL's Life Sciences group

Despite a decade of effort to streamline discovery and development and increase productivity, the projected return on investment in R&D at the worlds leading pharmaceutical companies has hit an all-time low, according to the 10th annual analysis by management consultancy Deloitte.

No other industry would operate on such low R&D returns.Substantive change is needed to shorten R&D cycle times, according to Karen Taylor, director of Deloittes health practice

Cash flows

Despite a rough ride on the capital markets for much of the year, particularly in the second and third quarters, the biopharmaceutical sector had no difficulty in attracting capital.

[This is] "the most exciting time to be investing in life sciences in a generation."Otello Stampacchia, managing director, Omega Funds, which raised $438 million to deploy into new and existing life sciences companies

"Capital is so available, it strikes me insane that almost half the industry needs to raise money this year."Dennis Purcell, founder and senior advisor at Aisling Capital LLC, during a session at the BIO CEO & Investor Conference

"It's very much a chess game in the current market; you have to think multiple steps out."Nol Brown, managing director, health care investment banking, at Cantor Fitzgerald, on planning whether to IPO during a session at the BIO CEO & Investor Conference

"It's a great time to be an entrepreneur. There are so many sources of capital. And there's a lot of it."Chau Quang Khuong, private equity partner at Orbimed Advisors, speaking at the Biocom Global Life Science Partnering Conference on the advantages of starting a company now

M&As

Bristol-Myers Squibb Co.'s announced $74 billion bid for Celgene Corp., the largest M&A in biopharma history, made for what J.P. Morgan analyst Cory Kasimov called "one of the most significant pre-conference announcements ever."

Although the deal did not spark a wave of M&A activity at the time, transactions remained sporadic for the rest of the year. According to the 2020 RBC Biotech Outlook report, Despite the unevenness throughout the course of 2019, the forces we believed would stimulate a meaningful pickup in acquisitions coalesced to generate new highs in biopharma acquisitions, with a record number of announced $1B+ public company deals.

"Two turkeys don't make an eagle. Both businesses have seen their share of challenges, with ABBV's Humira seeing competitive pressures both in the U.S. and EU and Allergan's rather well-documented business challenges. However, financial potential does have our attention, with the deal being immediately accretive to earnings."Christopher J. Raymond, Piper Jaffray analyst, in a research note on the first reaction to the announcement that Abbvie Inc. was proposing to acquire Allergan plc in a cash and stock deal worth about $63 billion

Gene therapy is hot

Barely a day went by this year without news relating to cell and gene therapy companies being announced. Given the ongoing developments, it was not surprising that the sector continued to attract financing and business development, with biopharma companies ensuring that they secured a position in the space for themselves through partnering and acquisitions.

"Gene therapy gets around many of the drug delivery challenges that have plagued other treatment modalities like small molecules. In part, that is because it's a one-time approach and also it really enables you to target the tissue bed of interest, in some cases direct to the CNS."Shankar Ramaswamy, chief business officer, Axovant Gene Therapies Ltd.

"Gene therapies are among the most exciting medical advancements of our time. However, they also pose a great technical challenge, since the AAV particles designed to deliver genes to tissues are subject to immediate and overwhelming immune attack, specifically by complement C3, which may result in significant safety and efficacy constraints. We believe that the targeted control of C3 may prevent the C3-mediated attack on AAV particles."Cedric Francois, CEO and co-founder of Apellis Pharmaceuticals Inc.

"[Many gene therapies] are introduced with much higher levels of uncertainty about their long-term safety and effectiveness than standard treatments, and patients and insurers are being asked to pay extremely high prices up front for the promise of long-term benefit."Steven Pearson, president, The Institute for Clinical and Economic Review, which is proposing adaptations to the framework it uses in assessing the value of potential single or short-term transformative therapies (SSTs). The proposals are intended to help decision-makers conduct a more reliable and transparent evaluation of the uncertainty, value and value-based pricing of SSTs

Some of the gene therapeutic products and candidates have truncated the size of the genes and have done some pretty clever maneuvers to try and make the gene inserts smaller in order to be incorporated by AAV.Daniel Dornbusch, head of business development at Dnarx Inc., explained, while noting that the full length might be better and there are many genes that cant be truncated

"Gene editing is a rapidly developing technology that represents one of the most exciting developments in medicine. These techniques will be integral to the next generation of advanced therapeutics and we welcome their potential to provide important, and potentially life-saving, treatments for patients. As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles."Janet Lambert, CEO, The Alliance for Regenerative Medicine commenting on the release of the Alliance's Therapeutic Developers' Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications

Artificial intelligence and machine learning

There is no doubt that the next wave of drug discovery will be enabled by powerful supercomputers dining on complex algorithms to uncover potential new scientific approaches for the development of innovative therapeutics, and industry executives certainly provided BioWorld with a wide range of opinions on its value.

"The idea behind machine learning is that it's a method that should generalize and be able to make decisions or make estimates or classifications based on data it hasn't seen before."John Irvine, chief scientist for data analytics at Draper Laboratories, Siemens Healthineers. The firm is working with Brisbane's Translational Research Institute and using artificial intelligence to diagnose medical conditions ranging from breast cancer to post-traumatic stress disorder

"I think the drive in this field is that we saw the success of Google and Facebook to basically take what seemed like white noise out in the ether and squeeze out this gold; it's like the new gold rush. And in Alzheimer's we've been desperate for success, and it seems like we have this messy, noisy data it almost feels like static sometimes and we want a machine learning algorithm to come in and wrangle that up and squeeze gold out of what looks like noise. Unfortunately, I don't think it's going to be that easy."Newman Knowlton, a statistician at Millcreek, Utah-based statistical consulting firm Pentara Corp.

"When I have casual conversation about this, I think of the first cell phone and what iPhone is like today. At first, we wanted only a few functions, but we eventually realized we needed more functionality to benefit from the technology. It's the same in biomedicine."Ken Drazan, CEO, Arsenal Biosciences Inc.

"Data-driven medical innovation is growing exponentially, and our partnership with Google will help us lead the digital transformation in health care. It will empower us to solve some of the most complex medical problems; better anticipate the needs of people we serve; and meet them when, where and how they need us. We will share our knowledge and expertise globally while caring for people locally and always do it with a human touch."Gianrico Farrugia, president and CEO of Mayo Clinic

"Across the globe, clinicians spend more time doing data entry in patients' records than they do treating patients, and I think AI has a real opportunity to change that."Chris Nave, Managing Director, Brandon Capital

Editors note: See Fridays issue for Part 2.

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Perspectives on the year: The industry has its say on the markets, M&A and gene therapy - BioWorld Online