StemCell Therapy

December 23, 2019• Physics 12, 149

Experiments capture the motion of an ionized molecule with an unprecedented combination of spatial and temporal resolution.

The ionization of a molecule by light sounds simplethe light comes in, electrons get kicked out. But ask about the details of this processsuch as how the molecule reconfigures in responseand the theoretical picture is murky for all but a few textbook cases. A set of techniques for controlling and imaging molecules has now reached a new level of temporal and spatial resolution to provide some clarity. Markus Kitzler-Zeiler of Vienna University of Technology (TU Vienna) and colleagues imaged the wave function of a simple molecule as it responded to the loss of an electron [1]. Combining two state-of-the-art techniques, they produced a movie of the molecule that captured the movements of the molecules protons in 1 picometer ( 1012 m) incrementsa small fraction of the width of an atomand with 70 attosecond ( 71017s) resolution. The approach allows a test of ionization models in H2 and may lead to a better understanding of chemical processes in more complex molecules.

The researchers focus on the simple motion of protons in singly ionized molecular hydrogen ( H2+). With a simple structure of just two protons bound together by an electron, H2+is for molecular physics what the fruit fly is for genetics. In an experiment, the team prepares an ensemble of cold H2 molecules by injecting gas through a supersonic nozzle and into a vacuum chamber. When an H2 molecule is intercepted by a sufficiently strong laser pulse, the lasers electric field removes one electron, which sets the now weakly bound protons into an oscillatory motion mediated by the remaining electron. This oscillation would continue indefinitely, were it not for the still-present laser field, which causes the second electron to be ejected at some random time ( t) after the first. With nothing left to keep the protons together, their mutual repulsion pushes them apart in a Coulomb explosion [2].

The time between the first and second ionization is anywhere between 0 and about 5 fs, and it is the increasing separation between the two protons during this interval that the researchers image by combining two techniques (Fig. 1). In the first technique, they use their laser pulse as a sort of stopwatch that starts when the first ejected electron is detected and stops when the second ejected electron is detected. In fact, the pulse is just two cycles of elliptically polarized, 750-nm light, and its rotating electrical field vector serves as the ticking hand of a watch that makes a full rotation every 2.5 fs. The researchers can infer the ticks that dislodge the first and second electrons by measuring the direction that they escapea technique known as angular streaking [3].

The second experimental ingredient is a picoscope, which extracts the distance between the protons when the molecule explodes by measuring their kinetic energy. When the laser pulse strips off the second electron, each proton feels a 1r repulsive Coulomb potential. Each proton then slides down this potential by moving further away from its partner, gaining kinetic energy by an amount that depends on its initial distance from the other proton. The team measured this kinetic energy at the time of the explosion and compared it to the known potential, achieving an effective magnifying glass that sees the proton separation when the molecule breaks apart. Much like particle physicists, the researchers measured the kinetic energy of the protons by following their trajectories in electric and magnetic fields [4].

Each timed Coulomb explosion serves as a mini experiment. After roughly 10 million such experiments, the researchers reconstructed the proton-proton distance at several time intervals after the first ionization (Fig. 2). When viewed together, these reconstructions essentially provide a time-lapsed, picometer-resolution image of the molecular wave functionboth the separation between the protons and the protons quantum-mechanically smeared out position. Although this demonstration is primarily a proof-of-principle, the researchers were also able to use their precise mapping of the wave function to test and rule out two ionization scenarios for H2, known as shake-up and resonant multiphoton electronic excitations.

This is the first time an ionized molecule has been viewed with such high spatial and temporal resolution at once. Having validated this approach with the molecular fruit fly, the next step would be to apply it to more complicated and less studied species, particularly those that are of interest to chemistry, molecular biology, and medicine. Applying the picoscope to more complicated molecules will require knowledge of more complicated molecular potential surfaces. However, Kitzler-Zeiler and colleagues contend this is not a huge problem since such surfaces can be readily calculated, even if the molecular dynamics preceding the Coulomb explosion cannot. From my view, an interesting avenue to explore would be the process of quantum tunneling. When an electron is removed from the H2+ ion by the strong laser field, it spends time under the barrier of the ions potential [5]. But the length of time and the behavior of the ion during that time are fascinating unknowns that the new technique might potentially explore.

The art of molecular movie making has advanced significantly since the 1980s and 1990s, when Ahmed Zewail and his colleagues first used femtosecond laser pulses to make movies of molecular oscillations and chemical reactionsan accomplishment that was recognized with the 1999 Nobel Prize in Chemistry. Zewail adopted a pump-probe technique, where one pulse (the pump) excites a collection of molecules and a subsequent weaker pulse (the probe) samples changes in the molecular state. The inherent time resolution of his approach was on the order of the fs duration of the envelope of the laser pulsethe smooth rise and fall in intensity that modulates the much more rapidly varying optical cycles. By contrast, Kitzler-Zeiler and colleagues achieved a sub-fs probe by tapping into the carrier electric field, which rotates with the actual cycles of elliptically polarized light [6]. A further improvement might come from using available techniques to stabilize and exploit the relative phase between the carrier and the envelope [7]. This capability would allow the determination of not only the relative time between two ionization events, t, but also the absolute timing of the events. Nevertheless, with the relative timekeeping already in hand, the researchers have obtained an unprecedented view of both the position of a molecular wavepacket and its shape.

This research is published in Physical Review Letters.

Alexei Sokolov is a professor of Physics and Astronomy at Texas A&M, where he holds the Stephen Harris Professorship in Quantum Optics. He obtained an M.S. from Moscow Institute of Physics and Technology in 1994 and a Ph.D. in physics from Stanford University in 2001.His general expertise is laser physics, nonlinear optics, ultrafast science, and spectroscopy. Specific areas of interest include the generation of subcycle optical pulses with prescribed temporal shape; ultrafast atomic, molecular, and nuclear processes; and applications of molecular coherence in biological, medical, and securityoriented areas.

Vclav Hanus, Sarayoo Kangaparambil, Seyedreza Larimian, Martin Dorner-Kirchner, Xinhua Xie (), Markus S. Schffler, Gerhard G. Paulus, Andrius Baltuka, Andr Staudte, and Markus Kitzler-Zeiler

Phys. Rev. Lett. 123, 263201 (2019)

Published December 23, 2019

Read more from the original source:
Viewpoint: Timing Molecular Motion with an Optical Stopwatch - Physics

A team of researchers from the Hebrew University of Jerusalem have successfully associated the Bacillus Calmette-Guerin (BCG) vaccine with reducing the risk of Alzheimers.The disease affects one in 10 adults over the age of 65 a number that is expected to triple by 2030, according to HU.BCG is the vaccine used to treat tuberculosis worldwide. Though in the past it was administered routinely in the United States, it is today mainly used in developing countries or with high-risk populations in Western countries.However, the vaccine has another use, according to Herv Bercovier of HUs Department of Microbiology and Molecular Genetics. Bercovier, who led the BCG study with colleagues Charles Greenblatt and Benjamin Klein, said that BCG is also used to treat and cure certain types of bladder cancer.Hebrew University Professor Herv Bercovier (Credit: Lior Mizrahi)People with bladder cancer tend to be 70 years or older, he explained to The Jerusalem Post, so they are close to the population that has an increased risk for developing Alzheimers.Furthermore, Bercovier explained, BCG has been shown to lower general chronic inflammation. Patients with Alzheimers suffer from chronic inflammation in the brain as a result of proteins that improperly unfold inside and outside of the brain.As such, the HU team followed 1,371 bladder cancer patients receiving treatment at HUs Hadassah-University Medical Center. During follow-up visits, 65 cancer patients had developed Alzheimers, a release explained. Those who had not received BCG as part of their treatment had a significantly higher risk of developing Alzheimers than did BCG-treated patients: 8.9% (44 patients) as opposed to 2.4% (21).Moreover, when compared with the general non-bladder cancer population, people who had never been treated with BCG had a four-fold higher risk for developing Alzheimers than did those who were treated with BCG.Theres data reaching back to the 1960s that shows that countries treating bladder cancer patients with the BCG vaccine had a lower prevalence of Alzheimers disease but it hadnt been properly analyzed, said Bercovier.With this study, he said he believes we are closer to understanding the vaccines impact.Bercovier noted that the next steps are to reproduce the study in different populations. Then, he said, to prove causality, the team will conduct a formal clinical trial.The findings of this study were published in PLOS ONE.

Originally posted here:
Could a vaccine reduce the rate of Alzheimer's? These Israelis think so - The Jerusalem Post

Youve definitely heard of the ketogenic diet starving your body of carbs to force it to burn fat and produce the mind-clarifying, brain-healing compounds known as ketones. You may have even heard of people and athletes ingesting ketone salts or drinks to propel them into or keep them in a state of ketosis. And if you were paying close attention during the Tour de France this year, you may have spied Team Jumbo-Visma openly drinking ketones mid-race.

The funny thing is, these athletes are not on a ketogenic diet. They are not fat adapted.

For the last three years or so, weve seen Tour athletes fueling with carbs and then supplementing with exogenous ketones to score a two to three percent boost in performance from dual-fueling, says Matt Johnson, a former competitive cyclist and co-founder of The Feed, an online sports nutrition shop and leading supplier of exogenous ketones in the U.S. June was insane with teams placing $10,000 to $20,000 orders for ketone esters and rush shipping them to France. We could barely keep up with it.

Elite athletes biohacking to score a tiny edge? Nothing new.

But this is: a study in the Journal of Physiology says everyday athletes who arent on a keto diet, who arent fat-adapted, may improve their recovery by a whopping 15 percent just from drinking exogenous ketones after intense training days. And the news is spreading.

We have also had a huge spike in individual athletes ordering the product that seems to be only growing, Johnson adds.

Now, will this approach work for you? Heres everything you need to know.

First, a quick biology lesson slash crash course in the trendiest diet of the twenty-teens: in an ideal world, your body breaks down carbohydrates into glucose, which is then transported and used or stored as energy for your muscles, organs and, most importantly, your brain.

Your brain is at the top of the pecking order it gobbles about 20 percent of your total energy expenditure, a lot for a single organ and if its not fueled, everything else stops functioning. When you deprive your body of carbohydrates, your muscles can use fat for fuel, but your brain cant. Instead, your body has a fail-safe to prevent total shutdown: the liver starts converting fat into a superfood designed to save your starving brain: ketones.

Even if your body can adapt to burning fat quickly to fuel long runs and rides, it would still prefer to burn carbs. Which is why the notion of pro athletes downing exogenous ketone drinks without having to give up carbs is completely bonkers.

Ketones are essentially a fourth macronutrient your blood sugar is stable, your body is burning fat and your brain has entered an almost elevated state of functionality. In ketosis the state you reach when adhering to a keto diet your brain starts producing more mitochondria (the little powerhouses of energy in your body) and better regulating neurons. Staying in a state of ketosis has been shown to help clear the brain of proteins that can lead to and worsen Alzheimers disease, reduce seizures in about half of people with epilepsy and even extend the lifespan of mice.

In athletes, staying in ketosis via a ketogenic diet can increase fat utilization during exercise (great, considering your body can store way more fat for fuel than carbs), help reduce body fat and sometimes improve endurance time trials and sprint peak power.

The catch: it all rides on you steering clear of carbs with no slip-ups. If you eat more than your allotted count typically 50 grams, which is one cup of pasta or just two bananas your body falls out of ketosis and you dont get any of these benefits. And pretty much all nutritionists agree that even if your body can adapt to burning fat quickly to fuel long runs and rides, it would still prefer to burn carbs.

Which is why the notion of professional athletes downing exogenous ketone drinks without having to give up carbs is completely bonkers.

In the early 2000s, as part of a DARPA program to enhance U.S. soldier performance, Oxford professors Kieran Clarke and Richard Veech set out to distill the exact molecular structure of one of the ketones our body produces. The resulting ketone ester is a specific molecule, butanedial, that converts directly to beta hydroxybutyrate, the ketone our liver naturally produces in the ketogenic state, when you digest it, explains Geoffrey Woo, co-founder and CEO of HVMN.

HVMN is currently the only company to produce ketone esters, as they lease the patent to Clarke and Veechs molecular structure.

Now, keto followers are probably familiar with other brands of keto drinks (usually based on MCT oil) and ketone salts. But esters are different than these aids. MCT oils dont produce ketones; they help put your body in a state of ketosis so it can start producing its own but since that requires carbohydrate starvation, thats not an option for dual-fueling athletes, Johnson explains.

Ketone salts, meanwhile, use beta hydroxybutyrate as well, but by their nature, theyre bound to a mineral. Because you have to take so much ketone to raise your blood levels enough to see an effect, youre also gaining a lot of mineral load. This leads to a lot of GI issues in athletes, explains Woo. That, plus the fact that the salts dont raise your ketone levels that much, leaves a lot of room for a superior product. There has been minimal testing on the aids but the HVMN esters have been tested and verified, Johnson says.

Ketone esters are a way to eat ketones directly thats going to convert 100 percent to ketones in your body, Woo adds.

Woo says professional athletes drinking exogenous ketones during a race report about a two to three percent increase in performance. That matters in an event like the Tour but the real benefit for athletes, especially everyone other than Egan Bernal or Geraint Thomas, seems to be in downing a bottle once the race is over.

The aforementioned Journal of Physiology study, conducted by seemingly impartial Belgian researchers, simulated a Tour with everyday athletes: 20 fit men trained twice a day (HIIT or intermittent endurance training in the morning, then 1.5- to 3-hour endurance sessions at night), six days a week for three weeks. Half drank a ketone ester after each workout while half drank a placebo.

After three weeks, the guys were shredded everyone showed signs of cardiovascular, hormonal and perceptual overreaching. But those who had taken ketone esters regularly had significantly less damage in all these areas, and on a two-hour endurance test, they were able to ride at a higher sustainable pace and produce more power in the final 30 minutes compared to guys who recovered regularly. All in all, researchers estimated the ketone esters helped improve recovery by 15 percent.

Mainly, its providing your body with another option for fuel, says Jonathan Scott, Ph.D., R.D., an assistant professor at the Uniformed Services University in Bethesda, Maryland who researches performance nutrition and dietary supplements.

You can still have your cake and eat it too. Athletes dont need to consume a diet thats extremely restrictive, and they can then consume exogenous ketones to introduce yet another fuel source the body can use.

Your brain is either going to use glycogen or ketones for power. If ketones are available, glycogen is spared and your muscles can instead use that energy to fuel fiber repair and metabolic cleanup. Whats more, now your body isnt going to break down other structures like muscle fiber to get your brain the fuel, saving your body extra damage.

And, because ketones keep your blood glucose stable, your body is steadily producing insulin, which sweeps glucose into your cells, continuously topping off the pool of energy as its being used and at a much faster rate than youre able to with food, Scott explains.

In addition to faster post-exercise glycogen replenishment, a 2018 Italian study in Current Sports Medicine Report foundthat exogenous ketones decrease proteolysis (the breakdown of proteins into amino acids) and act as metabolic modulators and signaling metabolites.

Theres also some chemistry research to suggest exogenous ketones may help realign your hormone production, adds Krista Austin, Ph.D., C.S.C.S., a sports scientist, exercise physiologist and nutritionist. The anterior pituitary produces hormones that become dysregulated if youre overtraining, dont sleep well at night, have a poor heat tolerance, or experience something like a traumatic brain injury, she explains. Exogenous ketones seem to help realign the production of susceptible hormones like prolactin, which can otherwise prevent proper sleep and recovery.

To top it off, it takes very minimal effort for athletes to earn all these gains: You can still have your cake and eat it too literally and figuratively, Scott explains. Athletes dont need to be consuming a diet thats extremely restrictive on food choices or energy sources during exercise, and they can then consume exogenous ketones to introduce yet another fuel source the body can use.

Johnson says its only a matter of time before major American sports stars pick up the training aid and that well definitely see it in the Olympics. Basketball and hockey especially have some grueling schedules. Imagine the benefit in-season for back-to-back games on the road?

Johnson estimates that roughly 80 percent of the interest in exogenous ketones on The Feed comes from Europe and about 60 percent of that is from non-elites.

For most amateur athletes, that 15 percent improvement in recovery means youll simply feel better after a grueling workout youll have less muscle soreness and stiffness, more energy, better range of motion and sleep better, says Austin.

But thats not necessarily the score it sounds like. If you dont feel terrible after a series of tough training days or a hard race, youre much more likely to get back out, sooner, Austin says. But you might do more harm than good. Until we understand better how exogenous ketones affect the body and recovery, numbing the alarm doesnt change the need for rest.

And will they even work for you like they do for the pros? Jurys still out. Everyday athletes are likely going to respond differently to exogenous ketones, considering just the impact of genetics and training on energy substrate metabolism (how well your body burns other fuel sources) alone, Scott says. And, as with all supplements or performance aids, there are very clearly responders and non-responders. It simply doesnt work for everyone, he adds.

But most importantly, there are so many other aspects of performance that everyday athletes would be better served to focus on, Scott points out, including but not limited to sleep, diet composition, diet quality, nutrient timing, hydration, training program, rest days, stress management, meditation, visualization and even social relationship quality. For elites, all these things are taken into consideration and already optimized, he says. But I would hate for an amateur athlete to start taking ketones to improve sleep for better recovery when its really their stress management that needs to be tweaked.

The upside: as long as you monitor everything above, all our experts agree, theres close to no risk in trying.

Pretty much everyone agrees you shouldnt be using exogenous ketones to enhance recovery after every hard workout or race. This isnt meant for a long weekend ride, Johnson cautions. Even if it was really hard and I came home completely bonked and exhausted, I dont need a ketone ester to feel better at work the next few days.

Not only will drinking it post-ride regularly lead to overtraining, but, at $37 a bottle, a few bottles a week doesnt make economic sense for most of us. The effects of exogenous ketones last roughly an hour after ingestion and youre intended to drink a whole bottle immediately after moving for recovery.

If a client is having trouble sleeping, Ill have them drink ketones before bed so their body can catch up on repairs. But its important to address the underlying issues of why theyre not sleeping in the bigger picture.

But when marathon training gets serious and youre logging 15K, 18K and 12K all within a few days? Thats when you want to take it. Harder training weeks, multi-day endurance competitions, multi-stage races I would absolutely be using it after every stage. That level of benefit is enormous, Johnson adds.

Austin agrees, but adds shell also use it sparingly to disrupt recovery inhibitors. If a client is having trouble sleeping, Ill have them drink ketones before bed for just a few nights so their body can catch up on repairs, she says. But its important to address the underlying issues of why theyre not sleeping in the bigger picture.

And while we have no studies on microdosing (which would be more approachable and more wallet-friendly), Austin says shes seen some results. If someone is new to training, that mid-morning fatigue can be debilitating in terms of getting work done, but taking 10 milliliters of ketones can give them an energy boost, she explains.

Everyone agrees, given the current state of research, exogenous ketones are generally safe. And the one high-quality product we have on the market now (HVMN) is good to go.

But its worth noting that exogenous ketones are currently sold as dietary supplements, which means theres no oversight by the FDA. As ketones become more popular and more formulas come to market, well inevitably see products packed with both other enhancements and other cost-cutting, potentially dangerous ingredients, Scott says. (The upside: the hefty price of formulas like HVMN will likely come down, too.)

We also dont know the effects or risks of using it long term is there a threshold after which exogenous ketones stop being as effective? If your body gets used to the aid in recovery, could it eventually stop being as efficient at rebuilding without it? Do you get any of the neuroprotective benefits of naturally going into ketosis? And, perhaps most importantly, if youre an ultra-runner or frequent multi-day racer using exogenous ketones for recovery, what nuanced alarm bells are you overlooking?

There are definitely a lot of unanswered questions when it comes to exogenous ketones. But with minimal risk and serious potential gains, we wouldnt knock anyone for giving a sip.

Continued here:
How Athletes Are Reaping the Benefits of Keto Without Actually Giving Up Carbs - Gear Patrol

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."

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

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This post was originally published on Market Reports Observer

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Absorbable and Non-Absorbable Sutures Market Global Industry Analysis, Size, Share, Growth, Trends and Forecast 2025 - Market Reports Observer

Delaware Technical Community College alumna and adjunct instructor Autumn Cooper of Selbyville recently received the inaugural Delaware Veterinary Technician of the Year Award from the Delaware Veterinary Medical Association.

I am so honored to receive this award, Cooper said. This has been the most humbling and gratifying moment of my technician career.

The award is presented to a licensed veterinary technician who is a member in good standing of the Delaware Veterinary Medical Association, and has shown a positive contribution to the advancement of the profession and veterinary technicians in Delaware.

Cooper graduated from Delaware Tech in 2013 with a degree in veterinary technology. She earned her bachelor of science degree in business management from Wilmington University in 2018. Cooper began working at Savannah Animal Hospital in Lewes in 2013, and worked as an educational laboratory specialist at Delaware Tech from 2013-16. She has been an adjunct instructor for Delaware Tech since 2016, teaching anatomy and physiology, veterinary nursing, surgical and anesthesia nursing, and dental procedure/imaging.

Autumn is compassionate, smart, hardworking, and driven, said Lisa Garrison, veterinary technology instructor. Her passion for learning and education, and elevating licensed veterinary technologists in this profession, is commendable.I am honored to have been part of her journey from the first time I met her in 2010 as a student to now as a leader in the profession.

Veterinary medicine has been a lifelong passion for Cooper.

Like many in the veterinary field, I was blessed to grow up in a home with a variety of animals, Cooper said. I quickly learned, through routine pet care and pet emergencies, that an important part of loving an animal is making sure that it receives veterinary care.

Cooper said the education she received at Delaware Tech helped her get to where she is today.

The instructional team at Delaware Tech has been vital in propelling me to success in the veterinary field, she said. I am so grateful to my instructors, mentors, co-workers, family, friends, and God for my continued success and future opportunities.

For more information on the veterinary technology program, go to http://www.dtcc.edu/programs.

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Delaware Tech's Autumn Cooper named vet tech of the year - CapeGazette.com

PHILADELPHIA (CBS) It was a medical first in Philadelphia as human technology was used to save a dog with heart disease. Its a high-tech hearth procedure that is routinely performed on human patients. Now, for the first time in the region, its been successfully used for a dog.

Her family says Sophie has a heart of gold. Now, she also has a heart thats making history.

Shes just a joy, Sophie s owner Karen Cortellino said. It was love at first sight.

But their love story was suddenly threatened when the 9-year-old boxer fainted.

We were told even with medicine theres a high risk of sudden death, Cortellino said.

Cortellino learned her beloved rescue had an arrhythmia, the same type of erratic heartbeat thats diagnosed in people.

She had arrhythmogenic right ventricle cardiomyopathy, said Dr. Anna Gelzer, a cardiologist at Penns School of Veterinary Medicine.

Gelzer figured the human treatment could also work for dogs.

Sophie was the first case where weve tried to ablate ventricular tachycardia, Gelzer said.

For that, they turned to colleagues at Penn Medicine, where human heart patients are treated.

We were able to use the exact same equipment, Gelzer said.

When she explained to us that Sophie was going to have this procedure at the human hospital at HUP, I couldnt believe it, Cortellino said.

During ablation, a high energy catheter tip burns tiny portions of damaged heart tissue to restore normal rhythms.

The red dots are dots where we ablated, Gelzer said.

High-tech mapping helped guide the ablation, but it was the first-of-its-kind on a dog.

Were fortunate that things went smoothly, Gelzer said.

Cortellino says Sophie was pretty quickly back to normal with the human intervention saving her dogs life.

Its amazing. It is just completely amazing, Cortellino said.

Cortellino is hoping the risk she took with Sophie having the procedure will help other dogs in the future.

The treatment is experimental for now with a grant. The heart disease that Sophie has is common for boxers and is also prevalent in American bulldogs.

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Shes Just A Joy: Penn Doctors Save 9-Year-Old Dogs Life With First-Of-Its-Kind Heart Surgery - CBS Philly

GENETICS is published by the Genetics Society of America, a scholarly society that seeks to deepen our understanding of the living world by advancing our understanding of genetics. Since 1916, GENETICS has published high-quality, original research presenting novel findings bearing on genetics and genomics. The journal publishes empirical studies of organisms ranging from microbes to humans, as well as theoretical work.

While it has an illustrious history, GENETICS has changed along with the communities it serves: it is not your mentor's journal.

The editors make decisions quickly in around 35 days without sacrificing the excellence and scholarship for which the journal has long been known. GENETICS is a peer reviewed, peer-edited journal, with an international reach and increasing visibility and impact. All editorial decisions are made through collaboration of at least two editors who are practicing scientists.

GENETICS is constantly innovating: expanded types of content include Reviews, Commentary (current issues of interest to geneticists), Perspectives (historical), Primers (to introduce primary literature into the classroom), Toolbox Reviews, plus YeastBook, FlyBook, and WormBook . For particularly time-sensitive results, we publish Communications. As part of our mission to serve our communities, we've published thematic collections, including Genomic Prediction, Multiparental Populations, Genetics of Immunity, and Genetics of Sex.

More than just a publisher, the Genetics Society of America is mission-driven and places a high priority on responding to community needs. GENETICS and G3 have long been committed to supporting resources that serve scientists. We were the first journals to partner with Cold Spring Harbor Laboratories to enable seamless deposits of manuscripts from our submission systems straight into the preprint server bioRxiv, as well as from bioRxiv to GENETICS and G3, and we have accepted submissions posted for preprint servers since 2012. Articles feature links to model organism databases like SGD, FlyBase, and WormBase. We have also partnered with Overleaf to provide custom templates for authors who use LaTex, saving them time at submission. Our collaboration with protocols.io, encourages authors to freely share methods from GENETICS articles, helping to increase research reproducibility. The annotation tool Remarq is available on both the GENETICS and G3 websites and allows for collaborative commenting and article sharing. Our latest collaboration with Figshare ensures that supplemental material and data files are permanently associated with an articleand that authors arent limited by file type or size when providing data that support their work. Early online publication means that research investigations are freely accessible and in PubMed within days of acceptance which eliminates delays in discovering the latest science.

For information on the Genetics Society of America, please visit the GSA Home Page.

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About GENETICS | Genetics

Learn about the building blocks of life and the future of medicine.

The way we diagnose and treat diseases is changing, with new technologies enabled by a deeper understanding of the human genome and its relationship to health and disease.

In HMX Fundamentals Genetics, youll get an overview of key concepts behind the evolving fields of human genetics, genomics, and precision medicine.

For upcoming course dates and application information, visit our Courses page.

Experience a full genetics lesson in the HMX Preview course.

Overview

The Central Dogma and Genetic Variation

Mendelian Inheritance of Disease

Identifying Mendelian Disease Genes

Chromosomal Aberrations

The Genetics of Cancer

Common Complex Traits

Human Population Genetics

Beyond the Genome Sequence

Genetics and Precision Medicine

Lecturer in Genetics, Harvard Medical School

As DNA sequencing becomes cheaper and more readily available, the role of genetics in medicine is expanding. With our increasing understanding of the variation in the human genome, we can start to predict how specific changes in DNA sequence will affect an individuals health. Human genetics is extremely complicated, but it is apparent that many human diseases are influenced by genetics. From a medical perspective, this includes everything from rare DNA sequence variants that drastically increase risk of a disease, to common variants that lead to small changes in risk.

Historically, genetics specialists have helped patients to navigate these situations, but genetics is beginning to permeate medicine as a whole. This means that health care professionals across many fields are faced with new questions. When do you recommend genetic testing to a patient? What kind of testing will provide the most conclusive results? How do you help the patient to understand those results, and use them to make decisions about his or her care? Moving forward, an understanding of the principles of genetics and an ability to apply them in todays medical landscape will be an enormous asset for any health care professional.

Even outside of medicine, genetics has a very important presence in our world today, so, fundamentally, what I would like students to take away from this course is a level of genetic literacy that will allow them to navigate the questions and decisions that they will face in their own lives.

Direct-to-consumer genetic testing now allows everyone to learn about their own genetics; these tests can potentially include information about health and disease risk and ancestry that have significant impacts for individuals and their families. When making decisions about genetic testing, it is important to be informed about exactly what you will learn and what the consequences of the results might be down the line.In this course, we put the fundamental principles of human genetics into the context of the world today, which will provide students with the tools and resources to ask the right questions and make informed genetic decisions.

Professor of Medicine, Harvard Medical SchoolAssociate Physician, Brigham and Womens HospitalGeneticist, Brigham and Womens HospitalDirector, Genomes2People Research Program at Brigham and Womens Hospital

Genomics is inherently exciting, and you can see that because of its relationship to forensic law enforcement, reproduction, ancestry, cancer and all sorts of medical developments, and all of the controversy related to direct-to-consumer genetic testing.

The skeleton of genetics is sequencing, and people are building on that with gene expression, proteomics, metabolomics. So there are multiple layers of knowledge and exploration that are generating tremendous excitement in the world, and motivating enormous investment scientifically, educationally, financially, and entrepreneurially. So the question isnt really why Im interested in genomics, the question is why isnt everybody interested in genomics because its so fabulously interesting.

Senior Genetic Counselor and Project Manager, Brigham & Womens Hospital

Genetics is really a subspecialty of all specialties. The more we understand the genetic basis of biology, the more we will understand human health and disease. Having a strong background in genetics will be important as genomic technologies continue to expand and are implemented more and more into clinical care. Understanding how to look for patterns of disease and when to refer a patient to other specialists is an important tool for every health care provider, and as more patients have genetic results in their medical records, having the ability to understand how this is or is not important to your patients medical care will be of the utmost importance. Overall a global knowledge of genetics will help you to provide better care to your future patients.

I hope students are able to grasp some of the basic concepts about genetics and to understand why it is a vital part of medical care. I hope that this background knowledge will be useful to them if and when they care for patients who come to them with a genetic test result, a family history of genetic disease or a presenting genetic diagnosis. I hope this course excites them about the concept of genetics and genomics and encourages them to want to learn more as their career progresses.

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Genetics - HMX | Harvard Medical School

Experimental breeding

Genetically diverse lines of organisms can be crossed in such a way to produce different combinations of alleles in one line. For example, parental lines are crossed, producing an F1 generation, which is then allowed to undergo random mating to produce offspring that have purebreeding genotypes (i.e., AA, bb, cc, or DD). This type of experimental breeding is the origin of new plant and animal lines, which are an important part of making laboratory stocks for basic research. When applied to commerce, transgenic commercial lines produced experimentally are called genetically modified organisms (GMOs). Many of the plants and animals used by humans today (e.g., cows, pigs, chickens, sheep, wheat, corn (maize), potatoes, and rice) have been bred in this way.

Britannica Quiz

Branches of Genetics

Which is the study of the influence that genes and traits have on habits and actions?

Cytogenetics focuses on the microscopic examination of genetic components of the cell, including chromosomes, genes, and gene products. Older cytogenetic techniques involve placing cells in paraffin wax, slicing thin sections, and preparing them for microscopic study. The newer and faster squash technique involves squashing entire cells and studying their contents. Dyes that selectively stain various parts of the cell are used; the genes, for example, may be located by selectively staining the DNA of which they are composed. Radioactive and fluorescent tags are valuable in determining the location of various genes and gene products in the cell. Tissue-culture techniques may be used to grow cells before squashing; white blood cells can be grown from samples of human blood and studied with the squash technique. One major application of cytogenetics in humans is in diagnosing abnormal chromosomal complements such as Down syndrome (caused by an extra copy of chromosome 21) and Klinefelter syndrome (occurring in males with an extra X chromosome). Some diagnosis is prenatal, performed on cell samples from amniotic fluid or the placenta.

Biochemistry is carried out at the cellular or subcellular level, generally on cell extracts. Biochemical methods are applied to the main chemical compounds of geneticsnotably DNA, RNA, and protein. Biochemical techniques are used to determine the activities of genes within cells and to analyze substrates and products of gene-controlled reactions. In one approach, cells are ground up and the substituent chemicals are fractionated for further analysis. Special techniques (e.g., chromatography and electrophoresis) are used to separate the components of proteins so that inherited differences in their structures can be revealed. For example, more than 100 different kinds of human hemoglobin molecules have been identified. Radioactively tagged compounds are valuable in studying the biochemistry of whole cells. For example, thymine is a compound found only in DNA; if radioactive thymine is placed in a tissue-culture medium in which cells are growing, genes use it to duplicate themselves. When cells containing radioactive thymine are analyzed, the results show that, during duplication, the DNA molecule splits in half, and each half synthesizes its missing components.

Chemical tests are used to distinguish certain inherited conditions of humans; e.g., urinalysis and blood analysis reveal the presence of certain inherited abnormalitiesphenylketonuria (PKU), cystinuria, alkaptonuria, gout, and galactosemia. Genomics has provided a battery of diagnostic tests that can be carried out on an individuals DNA. Some of these tests can be applied to fetuses in utero.

Physiological techniques, directed at exploring functional properties or organisms, are also used in genetic investigations. In microorganisms, most genetic variations involve some important cell function. Some strains of one bacterium (Escherichia coli), for example, are able to synthesize the vitamin thiamin from simple compounds; others, which lack an enzyme necessary for this synthesis, cannot survive unless thiamin is already present. The two strains can be distinguished by placing them on a thiamin-free mixture: those that grow have the gene for the enzyme, those that fail to grow do not. The technique also is applied to human cells, since many inherited human abnormalities are caused by a faulty gene that fails to produce a vital enzyme; albinism, which results from an inability to produce the pigment melanin in the skin, hair, or iris of the eyes, is an example of an enzyme deficiency in man.

Although overlapping with biochemical techniques, molecular genetics techniques are deeply involved with the direct study of DNA. This field has been revolutionized by the invention of recombinant DNA technology. The DNA of any gene of interest from a donor organism (such as a human) can be cut out of a chromosome and inserted into a vector to make recombinant DNA, which can then be amplified and manipulated, studied, or used to modify the genomes of other organisms by transgenesis. A fundamental step in recombinant DNA technology is amplification. This is carried out by inserting the recombinant DNA molecule into a bacterial cell, which replicates and produces many copies of the bacterial genome and the recombinant DNA molecule (constituting a DNA clone). A collection of large numbers of clones of recombinant donor DNA molecules is called a genomic library. Such libraries are the starting point for sequencing entire genomes such as the human genome. Today genomes can be scanned for small molecular variants called single nucleotide polymorphisms, or SNPs (snips), which act as chromosomal tags to associated specific regions of DNA that have a property of interest and may be involved in a human disease or disorder.

Many substances (e.g., proteins) are antigenic; i.e., when introduced into a vertebrate body, they stimulate the production of specific proteins called antibodies. Various antigens exist in red blood cells, including those that make up the major blood groups of man (A, B, AB, O). These and other antigens are genetically determined; their study constitutes immunogenetics. Blood antigens of man include inherited variations, and the particular combination of antigens in an individual is almost as unique as fingerprints and has been used in such areas as paternity testing (although this approach has been largely supplanted by DNA-based techniques).

Immunological techniques are used in blood group determinations in blood transfusions, in organ transplants, and in determining Rhesus incompatibility in childbirth. Specific antigens of the human leukocyte antigen (HLA) genes are correlated with human diseases and disease predispositions. Antibodies also have a genetic basis, and their seemingly endless ability to match any antigen presented is based on special types of DNA shuffling processes between antibody genes. Immunology is also useful in identifying specific recombinant DNA clones that synthesize a specific protein of interest.

Because much of genetics is based on quantitative data, mathematical techniques are used extensively in genetics. The laws of probability are applicable to crossbreeding and are used to predict frequencies of specific genetic constitutions in offspring. Geneticists also use statistical methods to determine the significance of deviations from expected results in experimental analyses. In addition, population genetics is based largely on mathematical logicfor example, the Hardy-Weinberg equilibrium and its derivatives (see above).

Bioinformatics uses computer-centred statistical techniques to handle and analyze the vast amounts of information accumulating from genome sequencing projects. The computer program scans the DNA looking for genes, determining their probable function based on other similar genes, and comparing different DNA molecules for evolutionary analysis. Bioinformatics has made possible the discipline of systems biology, treating and analyzing the genes and gene products of cells as a complete and integrated system.

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Genetics - Methods in genetics | Britannica

If, then, progress was to be made in Genetics, work of a different kind was required.

It sprang from genetics and bears the mark of an implicit Darwinian mechanism.

The metaphors of genetics and evolutionary models can be applied.

I've been studying up on biology and genetics; talking to Chang got me interested.

Lindstrom of Iowa has led in research on the genetics of tomatoes, chromosome relations and mode of inheritance.

Most students of genetics realize that a factor difference usually affects more than a single character.

The formalism of memetics reminds many of us of formal languages, as well as of the shorthand used in genetics.

General biology and the science of Genetics are bringing to light much that must be incorporated in Sociology.

The terminology is based on today's fashionable lingo of genetics, and of memetics, its counterpart.

They also opened new horizons for hypotheses in astronomy, genetics, anthropology.

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Genetics Synonyms, Genetics Antonyms | Thesaurus.com

Genetics is a field of scientific study focused on heredity and DNA variation. Genetics professionals need a strong background in biological science to support their work, and most positions in this field require graduate degrees. Learn more about genetics and relevant career options here.

Genes are composed of DNA, the molecule that determines a living thing's unique physical characteristics, such as hair color or immunity to certain diseases. Geneticists are the biological scientists who study genes and how genetic variations affect the body. The job duties of genetics professionals vary by setting. While clinical geneticists and genetics doctors work directly with patients, genetic scientists spend most of their time in research or laboratory settings.

Many careers in this field require doctoral degrees in genetics or closely related fields; however, options are available to those who hold relevant bachelor's or master's degrees. Many geneticists focus their careers on research and laboratory study and typically hold Ph.D.s in genetics, molecular biology or related fields of study.

Geneticists may also serve as clinical geneticists, physicians who provide medical care to patients suffering from hereditary diseases. Clinical geneticists must complete medical school and obtain licensure to practice as physicians. Below is a list of Study.com articles to help you choose the degree program that's right for you.

While online degree programs in genetics are rare, some schools offer correspondence courses that lead to college credit. Students may be able to complete courses or earn degrees in biology and other genetics-related subjects.

While becoming a geneticist is the obvious career path, a variety of other career options are also available for students who do not possess a doctoral degree. Individuals with a master's degree in a related branch of counseling may become genetic counselors, providing therapeutic services to patients who have been diagnosed with hereditary diseases.

With a master's degree in genetics, a student might find employment as a laboratory research assistant. A bachelor's degree related to genetics may also qualify a graduate for a job as a laboratory technician. Here are a few links to articles that may help you discover which career you want to pursue.

According to the U.S. Bureau of Labor Statistics (BLS), jobs for biological scientists were expected to increase 19 percent from 2012-2022, faster than the average for other occupations (www.bls.gov). Advances in research have uncovered new information on genes, and more genetics professionals will be needed to develop medical treatments out of this new information. Although employment rates in this field fluctuate according to government funding and economic climate, the BLS reports that biological scientists, such as geneticists, are less prone to job loss caused by recessions.

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Genetics - Study.com

It hasn't been the best quarter for Myriad Genetics, Inc. (NASDAQ:MYGN) shareholders, since the share price has fallen 20% in that time. In contrast the stock is up over the last three years. However, it's unlikely many shareholders are elated with the share price gain of 47% over that time, given the rising market.

Check out our latest analysis for Myriad Genetics

While markets are a powerful pricing mechanism, share prices reflect investor sentiment, not just underlying business performance. One flawed but reasonable way to assess how sentiment around a company has changed is to compare the earnings per share (EPS) with the share price.

Over the last three years, Myriad Genetics failed to grow earnings per share, which fell 37% (annualized).

Thus, it seems unlikely that the market is focussed on EPS growth at the moment. Therefore, we think it's worth considering other metrics as well.

It could be that the revenue growth of 4.2% per year is viewed as evidence that Myriad Genetics is growing. In that case, the company may be sacrificing current earnings per share to drive growth, and maybe shareholder's faith in better days ahead will be rewarded.

You can see below how earnings and revenue have changed over time (discover the exact values by clicking on the image).

NasdaqGS:MYGN Income Statement, December 24th 2019

It's good to see that there was some significant insider buying in the last three months. That's a positive. On the other hand, we think the revenue and earnings trends are much more meaningful measures of the business. So we recommend checking out this free report showing consensus forecasts

Investors in Myriad Genetics had a tough year, with a total loss of 8.9%, against a market gain of about 40%. Even the share prices of good stocks drop sometimes, but we want to see improvements in the fundamental metrics of a business, before getting too interested. Unfortunately, last year's performance may indicate unresolved challenges, given that it was worse than the annualised loss of 6.9% over the last half decade. Generally speaking long term share price weakness can be a bad sign, though contrarian investors might want to research the stock in hope of a turnaround. If you want to research this stock further, the data on insider buying is an obvious place to start. You can click here to see who has been buying shares - and the price they paid.

Myriad Genetics is not the only stock insiders are buying. So take a peek at this free list of growing companies with insider buying.

Please note, the market returns quoted in this article reflect the market weighted average returns of stocks that currently trade on US exchanges.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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Does Myriad Genetics's (NASDAQ:MYGN) Share Price Gain of 47% Match Its Business Performance? - Yahoo Finance

DetailsCategory: Small MoleculesPublished on Monday, 23 December 2019 16:05Hits: 504

NEW YORK, NY, USA and LONDON, UK I December 23, 2019 I Akari Therapeutics, Plc (Nasdaq:AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announces that a U.S. Food and Drug Administration (FDA) investigational new drug application (IND) is open for its multicenter Phase III study for the treatment of pediatric HSCT-TMA with nomacopan, allowing clinical sites to open in the first quarter of 2020.

With the pediatric HSCT-TMA IND now open we look forward to starting the pivotal Phase III study of nomacopan in HSCT-TMA, a potential treatment for a high risk pediatric population that suffer very high death rates and for which there are currently no approved therapies. If successful, we expect HSCT-TMA to be a gateway into a range of other poorly treated orphan TMAs, commented Clive Richardson, CEO of Akari Therapeutics. In addition, following the recent successful completion of our Phase II bullous pemphigoid study, we expect data from our Phase I/II atopic keratoconjunctivitis trial in early 2020 and interim data from our Phase III paroxysmal nocturnal hemoglobinuria trial in the first half of 2020.

HSCT-TMA is an orphan hematological condition that occurs in up to 30% of patients who have received a hematopoietic stem cell transplant (HSCT). There are no approved treatments for pediatric HSCT-TMA, and it has an estimated mortality rate of more than 80% in children with the severe form of the disease1. It is this severe form that is being targeted with nomacopan which is a bifunctional inhibitor of complement C5 and leukotriene B4 (LTB4). Following the recent end-of-Phase II meeting with the FDA, Akari has now opened an IND to initiate its pivotal pediatric HSCT-TMA study based on a single arm responder-based design. Recruitment will be focused on specialist pediatric sites in the U.S. and Europe where treatment tends to be concentrated in specialist centres.

Whilst the role of complement inhibition is understood to play an important role in pediatric HSCT-TMA, the Company believes LTB4 may also be an important target in reducing epithelial activation in both TMA and graft versus-host disease2 (GVHD) which often occur simultaneously. The Company believes daily dosing with nomacopan may also be of particular advantage in facilitating more complete complement suppression, especially in HSCT-TMA patients with high transfusion requirements.

As previously announced, this two-part pivotal Phase III study of nomacopan in pediatric patients with HSCT-TMA is based on guidance from the Companys end-of-Phase II meeting with the FDA. Part A of the trial is a dose confirmation study. Part B of the trial is a single arm responder-based efficacy study that will follow an interim analysis of Part A and a meeting with the FDA. Akari has both FDA fast track and orphan status for this program.

1 Sonata Jodele, et al. New approaches in the diagnosis, pathophysiology, and treatment of pediatric hematopoietic stem cell transplantation associated thrombotic microangiopathy. Transfus Apher Sci . 2016 April; 54(2): 181190

2 Takatsuka, et al. Predicting the severity of intestinal graft-versus-host disease from leukotriene B4 levels after bone marrow transplantation. Transplantation 2000, 26: 1313-1316

About Akari Therapeutics

Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari's lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4). Nomacopan is currently being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal hemoglobinuria (PNH). Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in AKC and BP. Akari is also developing other tick derived proteins, including longer acting versions.

SOURCE: Akari Therapeutics

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Akari Therapeutics Announces Initiation of Pivotal Phase III Trial of Nomacopan in Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic...

Of all blood cancers, leukaemia and lymphoma are among the most curable.

However, many people, including doctors, still believe the disease leads to immediate death.

This is no longer true today as they are not fatal.

With optimal treatment, the majority of patients go into remission and are considered cured.

These two cancers have been more extensively studied than other forms of cancer, due to the ease in obtaining samples from blood, bone marrow or lymph nodes, spurring the advent of novel targeted therapies for a cure, says consultant haematologist Dr Ng Soo Chin.

Most blood cancers start in the bone marrow, where blood is produced.

Bone marrow contains stem cells, which mature and develop into red blood cells, white blood cells or platelets.

In most blood cancers, normal cell development is interrupted by the uncontrolled growth of an abnormal type of a particular blood cell.

These abnormal blood cells, which are cancerous, prevent your blood from performing many of its functions, like fighting off infections or preventing serious bleeding.

Leukaemia or white blood is classified into acute and chronic disease, which is then divided further into subtypes: acute lymphocytic leukaemia, acute myeloid leukaemia, chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML).

The presentation between acute and chronic leukaemia differs.

The acute person will tell you he was well a week ago and is now down with symptoms such as lethargy, anaemia and recurrent infection.

Suddenly, he may look pale, so we check his blood count for any abnormalities. A bone marrow exam will further confirm whether it is acute.

With chronic leukaemia, the patient can be unwell for a couple of months.

We are increasingly picking up cases early because of blood test availability.

The survival rate has improved tremendously for acute leukaemia, with more than 50% fully cured because bone marrow transplants are easily available in the country.

For CLL and CML, 95% of patients are alive at the 10-year mark, says Dr Ng.

Generally, chronic leukaemia patients belong to the older age group (50 years and above), but acute leukaemia can occur in all ages.

Leukaemia symptoms are often vague and not specific, so its easy to overlook them as they may resemble symptoms of the flu and other common illnesses.

In fact, chronic leukaemia may initially produce no symptoms and can go unnoticed or undiagnosed for years.

Lymphomas, a type of blood cancer that begins in a subset of white blood cells called lymphocytes, can be classified into Hodgkins and non-Hodgkins.

The main difference between Hodgkins and non-Hodgkins lymphoma is the specific lymphocyte each involves.

Lymphocytes are an integral part of your immune system, which protects you from germs.

Five-year survival rates are high with Hodgkins lymphoma at 86% and non-Hodgkins lymphoma at 70%.

You can beat the disease even if it is detected at a late stage.

Multiple myeloma, which is the third kind of blood cancer, forms in a type of white blood cell called a plasma cell.

Patients often complain of bone pain, and unfortunately, this type of cancer has no cure.

Blood cancers typically involve abnormal white blood cells and can affect paople of all ages, depending on the type of cancer. 123rf.com

Fear of treatment

Chemotherapy is a much dreaded word among cancer patients.

But with advances in medicine, newer chemotherapy-free treatments are now available.

Dr Ng says, Traditionally, cancer is treated via surgery or radiation the layman says we fry and poison them, which is not far from the truth!

Radiation means burning the cancerous area, but a lot of times, the cancer can also be present elsewhere, so there is limitation to this treatment.

With chemotherapy, we use cytotoxic (cell-killing) drugs they go in and knock off both cancer and normal cells.

The short-term effects include vomiting, hair loss, appetite loss and weight loss.

But as doctors, we are looking at a different perspective. We are more worried about white cells dropping (neutropenia) because the patient can pick up an infection that can potentially kill him.

Neutropenia is a condition that results when the body does not have enough neutrophils, a type of white blood cell that is an essential first line of defence against infections.

Thats one risk of chemotherapy, although we can now improve neutropenia by giving a growth factor injection.

But for certain cancers, we need to step up the drugs.

He adds: We are scared of neutropenia, but patients are more concerned about bodily changes.

The older ones get upset over losing hair because they cannot take it when others ask them what has happened to their hair.

Young people are not as concerned with hair loss because it can be trendy.

We understand that chemotherapy is less than pleasant and strong doses can impair fertility in young patients, especially women.

Despite current technology, only one-third of patients are successful in freezing their eggs.

What he is concerned about is that chemotherapy can actually increase the patients risk of getting another cancer, especially blood cancer.

It can happen the day after! says Dr Ng.

Most experts believe chemotherapy damages stem cells, so if youre unlucky, you might get acute myeloid leukaemia after undergoing chemotherapy for breast cancer.

Its just like crossing the road there is always a risk of being knocked down.

All our cells have a biological clock and there is an orderly exchange of old and new cells.

But with blood cancers such as leukaemia, there is a clone of abnormal cells.

Cancer cells have an advantage over normal cells because they can survive longer.

Chemotherapy is still needed to treat most acute blood cancers, although if the mutation is known, targeted therapies can be applied.

For chronic blood cancers, there is no need for chemotherapy. Oral drugs are enough to combat the disease.

Eventually, many patients are able to wean off the drugs.

As we may be aware, immunotherapy is the buzzword in cancer treatment today.

Also called biologic therapy, it is a type of cancer treatment that boosts the bodys natural defences to fight cancer.

It uses substances made by the body or in a laboratory to improve or restore immune system function.

One of the latest treatment modalities is the CAR T-cell therapy, a form of immunotherapy that uses specially altered T cells a part of the immune system to fight cancer.

A sample of a patients T cells are collected from the blood, then modified to produce special structures called chimeric antigen receptors (CARs) on their surface.

When these CAR T-cells are reinfused into the patient, the new receptors enable them to latch onto a specific antigen on the patients tumour cells and kill the cells.

At the moment, this intravenous therapy is available in the United States and hasnt reached our shores yet. It has to be properly regulated first, says Dr Ng.

A volunteer is having his head shaved to donate hair to make wigs for cancer patients in this filepic. Hair loss is one of the side effects of chemotherapy that affect patients the most.

Following natural remedies

The consultant haematologist errs on the side of caution when patients ask about natural cancer remedies, or the dos and donts during treatment.

We always believe there should be a scientific approach to the problem.

If patients are doing okay while undergoing treatment and there is no weight loss, I tell them to go ahead and do what they always do.

However, just be particular about food hygiene, as there is a chance you may get food poisoning.

If youre undergoing chemotherapy, then youll land yourself in hospital, and if your luck is bad, you may even land up in the ICU (intensive care unit).

So make sure the food is cooked and not left overnight to reduce your chances of infection.

Eat a balanced diet, he advises.

When it comes to exercise, he says to work out within your limit.

Instead of pushing the body and running marathons or climbing mountains, go for walks.

Dr Ng says, Life should go on, but be sensible.

Dont go to crowded places because you may pick up an infection, but dont be withdrawn either. All humans need social interaction.

With the billion-dollar dietary supplements industry, companies are constantly trying to lure customers into buying their products.

A lot of supplements are just glorified vitamins in different packaging.

The more expensive they are, the more people will buy them, thinking they are good.

There are people with good intentions, but unfortunately, there are also a lot of scammers out there that is life.

For the amount you spend on supplements, why not keep the money aside and go for a trip once your treatment is over? he suggests.

Often, the late diagnosis is due to preference for alternative treatment.

These alternative treatments are like fashion shows, after some time, they go out of trend.

For me, youre wasting valuable time because cancer is not your friend.

Yes, chemotherapy is tough, but with the latest chemo-free regimen, patients are more willing to come forward.

The earlier it is treated, the higher your chances of recovering, he says.

To share his 30-odd years of knowledge and experience in the field, Dr Ng has written his third book titled Understanding Blood Disorders.

Intended for patients, caregivers and healthcare professionals, proceeds from the sales of the 270-page book will go to the newly set-up Faith Hope Love Hospice Care Malaysia in Petaling Jaya, Selangor.

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Leukaemia and lymphoma have a good survival rate - The Star Online

Cell and gene therapy manufacturing may never be standardized but the whole industry would gain if firms collaborated to develop common methods for some processes according to an expert.

Manufacturing cell and gene therapies is an expensive business, partly because no two products are made the same way.

A recent study in the journal Nature suggested the average cost of making an autologous cell therapy is between $100,000 and $300,000 per patient.1

The authors attributed the high cost to the use of novel and specialized manufacturing processes [which] make scaling to meet commercial demand a significant challenge for all.

A separate study in the Journal of Clinical Oncology also concluded that difficulties scaling-up the bespoke manufacturing processes and technologies used to make cell and gene therapies significantly increases production costs.2

Market tensions

And high costs are a problem, according to Maria Whitman, managing principal at consulting firm, ZS Associates, who said cell and gene therapy firms need to find more economic ways of making products.

Standardization in manufacturing across the industry is not likely to be the priority for standardization in the short termHowever, the in-market cell and gene therapies have illuminated a number of tensions in the U.S. healthcare system which was designed for pills and biologics.

With over 200 CAR-TCR trials alone in the United States, there is need for standardization of aspects of the process to enable scale and commercial viability of these technologies. The challenge is that, today, each manufacturer is in part by necessity establishing their own process and protocols, she said.

The key is to look for similarities in processes, according to Whitman.

Potential areas for manufacturing and logistical standardization include apheresis protocols, labeling and information management, tracking processes, and training certifications, she said.

Whitman suggested contract manufacturers could help to identify common manufacturing challenges if customers are willing to work together and share information about noncompetitive areas of production.

The process question we should be asking as an industry is this: what is really competitive IP, and what is not? If we answer that, we can identify and solve for more systemic needs.

Logistics is another area where standardization would benefit the sector, Whitman added, citing developers of autologous therapies as the obvious example.

Autologous cell therapies are produced from the patients own cells. Typically the cells are harvested at a clinic and transported to the manufacturing facility before being returned to the patient. Ensuring such therapies are delivered in a timely fashion is vital.

According to Whitman, Manufacturers are trying multiple approaches to streamline the logistics of distance between manufacturing and patient administration. Some are developing in-house solutions and technology or leveraging partnerships to minimize risks and timing.

There is also a new industry emerging of companies forming to solve specific issues including apheresis networks, product manufacturers, as well as companies that create ordering portals, supply chain management systems.

One approach is to localize manufacture. Whitman said, There are already a number of manufacturers working on technologies to make point-of-care cell therapy a reality. Some academics are also creating their own CAR-TCRs, for example, and running trials in parallel with traditional manufacturer trials.

Ultimately the growth of the cell and gene therapy sector will depend on manufacturers ability to balance production and logistics costs with product prices. And the desire to find such a balance is clear, Whitman said.

Manufacturers will look for ways to optimize and automate the process where possible to reduce the cost of skilled human labor and continue to remove risk and drive efficiency in the system.

References1. http://www.nature.com/articles/s41434-019-0074-72. hascopubs.org/doi/10.1200/JCO.18.02079

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IP or Not IP: That Is the Question for Cell and Gene Therapy Sector - Genetic Engineering & Biotechnology News

For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, there's no getting around the fact that it's been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360% over the last 10 years to the S&P 500's 190%. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, it's worth highlighting how far we've come in the past 10 years in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in health-care costs without breaking an ecosystem that incentivizes the search for new discoveries. Here are some of the decades biggest medical breakthroughs:

Cell therapies: First approved in the U.S. two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumors, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Siences Inc. and Novartis AGs Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldn't be looking so far into the future without this decades extraordinary progress.

Gene therapies: Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

RNA revolution: The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the past two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology IPO of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Cancer immunotherapy: Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the body's defense mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got a hold of Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the U.S. alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumors is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

Conquering hepatitis C: From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good new is, treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health impact.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programs and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are likely to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will likely come with steeper prices. Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible. That would really be a breakthrough.

Max Nisen at mnisen@bloomberg.net

@2019Bloomberg

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Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard

NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.

FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann

The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.

At $2.1 million per patient, Zolgensma is the worlds costliest single-dose treatment.

Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartiss AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.

Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.

There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January, Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a Russian roulette.

The company said it developed the plan with the help of bioethicists with an eye toward fairness.

This may feel like youre blindly passing it out, but it may be the best we can do, said Alan Regenberg, who is on the faculty at Johns Hopkins Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2, he said.

According to Rucinski, the parties will continue their discussion in January to see what can be improved in the design of the program.

Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogens Spinraza.

Not all of the SMA community are opposed to Novartis program.

Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.

The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who receives the treatment, he said.

Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler

Originally posted here:
Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters

The European Medicines Agency has validated BioMarins application to market its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.

As such, the company said it expects the agencys review of the therapy in January next year under accelerated assessment.

The EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 for valoctocogene roxaparvovec and recently granted BioMarin's request for accelerated assessment of the MAA, potentially shortening the review period.

The submission is based on an interim analysis of study participants treated in an ongoing Phase III study with material from the to-be-commercialised process and updated three-year Phase I/II data.

It marks the first marketing application to be filed in Europe for a gene therapy product for any type of hemophilia.

BioMarin also announced the filing of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the treatment, with the review expected to being in February.

"We are pleased that the agency has recognised the potential scientific advancement that valoctocogene roxaparvovec could bring to people with severe hemophilia A," said Hank Fuchs, president, Global Research and Development at BioMarin.

"We continue to move thoughtfully and urgently through the regulatory review process to deliver a treatment that we believe has the potential to make a meaningful difference to people with hemophilia A.

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BioMarin's haemophilia gene therapy moves forward in the EU - PharmaTimes

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.

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