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Archive for March, 2020

The Top 3 Gene-Editing Stocks to Own in 2020 – Investorplace.com

Thursday, March 12th, 2020

The gene-editing revolution is already here, and these stocks will help you capitalize on the movement.

Gene-editing therapies allow you to remove cells from the body, modify them and reintroduce them. With this technique, theres hope for a cure for cancer, blood disorders, blindness, AIDS, cystic fibrosis, muscular dystrophy, Huntingtons disease and a host of other diseases.

For example, gene-editing has already been used to eliminate HIV in mice.

According to the National Institute on Drug Abuse, HIV-1 could be eliminated in mice using a combination of two antiviral technologies long-acting viral reservoir-targeted antiretroviral therapy and CRISPR/Cas-9 gene editing.

Doctors are even attempting to cure blindness after hacking a patients genes.

A patient recently had the procedure done at the Casey Eye Institute at Oregon Health & Science University for an inherited form of blindness.

We literally have the potential to take people who are essentially blind and make them see, said Charles Albright, chief scientific officer at Editas Medicine. We think it could open up a whole new set of medicines to go in and change your DNA.

If the procedure is found to be a success, doctors plan on testing it on more children and adults. Success for one company could also create a sizable opportunity in the sector for related stocks as well.

Source: Catalin Rusnac/ShutterStock.com

CRISPR Therapeutics (NASDAQ:CRSP) is one of the top names in the gene-editing market with nine drug candidates.

One candidate is CTX001, a drug that targets sickle cell and transfusion-dependent beta-thalassemia (TBT). With both, patients have poorly formed red blood cells that just cant delivery oxygen throughout the body well. In Nov. 2019, CTX001 successfully helped to eliminate symptoms in a patient with TBT, and another with sickle cell.

Overall, while still very early, the results provide the first suggestion of curative potential for this cutting-edge technology in such genetic diseases, and with potential for further safety refinement of Crispr/Cas9 administration, could suggest broad long-term potential of the many early-stage gene editing therapeutic tools VRTX has accumulated, RBC Capital Markets Brian Abrahams wrote.

Other candidates (CTX110, CTX120, CTX130) are also candidates for a cancer treatment known as chimeric antigen receptor T cell (CAR-T) therapy.

Source: vxhal/ShutterStock.com

Editas Medicine (NASDAQ:EDIT) along with Allergan (NYSE:AGN) just treated a blind patient with EDIT-101 as part of a Brilliance phase clinical trial for the treatment of Leber congenital amaurosis (LCA). With this study, its the first time a patients genes are being modified in the body itself, which is known as in vivo treatment.

Editas is also working on a sickle-cell disease and transfusion-dependent beta-thalassemia (TDT) drug, EDIT-301. EDiT-102 is being developed for Usher Syndrome 2a, a genetic condition characterized by hearing loss and vision loss that begins in adolescence or adulthood.

Source: CI Photos/ShutterStock.com

Intellia Therapeutics (NASDAQ:NTLA) is also working on a sickle-cell disease drug. Its also working on NTLA-5001, a drug that could help treat acute myeloid leukemia (AML). This is a rare type of cancer found in the bone marrow, which leads to the production of abnormal red and white blood cells.

In addition, the company is working on NTLA-2001 for transthyretin amyloidosis, a rare condition characterized by buildups of protein deposits called amyloids throughout the body, which can lead to the loss of sensation in extremities, and in internal organs.

Better, according to the company, In 2019, we advanced our full-spectrum strategy, guiding both ourin vivoandex vivo lead programs toward the clinic. We also continued to build on our genome editing and delivery capabilities to enable a rapid succession of candidates, said Intellia President and Chief Executive Officer, John Leonard, M.D., adding:

We are off to a productive start in 2020. We announced the nomination of NTLA-5001, a WT1-directed TCR-T cell therapy for the treatment of AML, and plan to select our third development candidate in the first half of this year, which will be for the treatment of HAE. In addition, in the second half of the year, we expect to begin dosing ATTR patients with NTLA-2001, a potential single-course treatment for ATTR patients. This is anticipated to be the first-ever systemically delivered CRISPR/Cas9-based therapy to enter the clinic, representing an important milestone in our mission to deliver potentially curative therapies from our proprietary modular platform.

Ian Cooper, an InvestorPlace.com contributor, has been analyzing stocks and options for web-based advisories since 1999. As of this writing, Ian Cooper did not hold a position in any of the aforementioned securities.

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Assistant professor says he’s been fired because he dared to talk about human population variation – Inside Higher Ed

Thursday, March 12th, 2020

An assistant professor of psychology at Marietta College says his contract isnt being renewed because of what hes said and was alleged to have said about differences between ethnic groups.

Many academics believe that race is mere social construct -- that there is no meaning behind being black, white or anything else, beyond what society assigns to it. Others say that that is mere orthodoxy and that race is real; this group often points to research demonstrating group-based differences in complex traits such as intelligence.

Scientists at the cutting edge of studying race and complex traits, meanwhile, say that these traits are always a mix between genetics and environment. And as of now, these experts add, its impossible to tell in any genuine way just what the mix is, because babies cant be raised exactly the same way over two generations, as such experiments would require.

Bo Winegard falls in the middle camp and believes that purposely not talking about race-based differences is disingenuous and dangerous. The "rich, variegated tapestry of humanity" and its evolution have long interested him and ought to be among the truths that academics pursue, he said in a recent interview. Otherwise, he added, "literal racists" will fill the information void.

I do think theres an informational embargo on human population variation and certainly on race and IQ, he said. People have opinions, and they dont want those to get out publicly.

Whatever you think of Winegards ideas, he said in a recent essay in the conservative academic publication Quillette, you should care that hes effectively being fired for them.

If it can happen to me, then it can happen to any academic who challenges the prevailing views of their discipline, he wrote. You may disagree with everything I believe, say, and write, but it is in everyones interests that you support my freedom to believe, say and write it.

Trouble Begins

Winegard, who is in his second year at Marietta and is scheduled to leave at the end of the academic year, says the trouble started in October. That's when he was invited to address the University of Alabamas Evolution Working Group, which is affiliated with the universitys evolution studies program. Both parties agreed that Winegard would talk about population variation, or, in his words, the hypothesis that human biological differences are at least partially produced by different environments selecting for different physical and psychological traits in their populations over time.

The idea was to link the theory with natural selection, in line with a recent article Winegard co-wrote for Personality and Individual Differences. The article, called "Dodging Darwin: Race, Evolution and the Hereditarian Hypothesis," says, "Like most hereditarians (those who believe it likely that genes contribute to differences in psychological traits among human populations), we do not believe there is decisive evidence about the causes of differences in cognitive ability." Yet the "partial genetic hypothesis is most consistent with the Darwinian research tradition."

One class visit with students went well, Winegard recalled in Quillette. Then he received a number of texts from a campus host expressing concern about Winegards entry on the website RationalWiki. The website, like Wikipedia, is edited by volunteers, but is dedicated to debunking what it sees as junk science. And Winegard, according to RationalWiki, is guilty of writing racist bullshit for the right-wing online magazine Quillette.

Winegard told his hosts that he disagreed with the characterization. He has previously argued, for example, that racism isnt wrong because there arent races; it is wrong because it violates basic human decency and modern moral ideals.

This, of course, contradicts a broad literature asserting that race is a social construct, not a biological one, but it doesnt endorse racism. As Winegard said in the same co-written article, In fact, pinning a message of tolerance to the claim that all humans are essentially the same underneath the skin is dangerous. It suggests that if there were real differences, racism would be justified.

Despite the texts, Winegards main talk at Alabama went on as scheduled, followed by what he described as a rowdy question-and-answer period. Someone yelled that he was a racist, and another accused him of promoting phrenology, a discredited pseudoscience having to do with skull shape.

But Winegard said via telephone that that he never spoke about phrenology or on race and IQ at Alabama. The most controversial thing he said was that psychology may someday, in the aggregate, provide some explanation as to why East Asian societies tend toward collectivism, he added.

One of his slides, however, did say that groups may vary on socially significant traits (on average) such as intelligence, agreeableness, athleticism, cooperativeness [and] criminality.

Alabamas student newspaper published an article on the talk, vaguely linking the subject matter to eugenics, or reproduction to promote certain heritable traits. It also published an apology from the group that hosted him.

Winegard said this week that he never mentioned eugenics, and that he finds things such as forced sterilization morally repugnant. He didn't preclude having mentioned embryo selection once or twice on Twitter, he said, but he's never made a sustained argument.

Back at Marietta, Winegard was summoned to a meeting with his president and provost to discuss the article. While they werent pleased, Winegard wrote in Quillette, they told [him] to be more strategic in my navigation of such a sensitive topic. I agreed that I would try.

Months later, someone began emailing Winegards department and administration about things hes written and said on Twitter. One tweet, in particular, read, The greatest challenge to affluent societies is dealing openly, honestly, and humanely with biological (genetic) inequality. If we dont meet this challenge, I suspect our countries will be torn apart from the inside like a tree destroyed by parasites.

At a second, consequent meeting with his supervisors, Winegard explained (as he recapped in Quillette) that his tweet was not about groups, but rather about individual genetic differences, and the need to create a humane society for everyone, not just for the cognitive elite and hyper-educated (a theme I discuss often). The simile about parasites was a reference to political conflict and not a reference to some group of humans or another, he also said.

Winegard recalled his bosses expressing disappointment in me and particular dismay about the tweet I had deleted, which they said evoked anti-black and anti-Semitic tropes. He agreed and apologized but said he would continue to pursue potentially controversial research topics.

Termination

Termination never came up, even after Winegard published a co-written article on human population variation -- until two weeks ago.

My boss informed me, without any warning, that the college was not renewing my contract, he wrote in Quillette. I dont know if my paper was the proximate cause of my firing, but in the light of the foregoing weeks tumult, it was plausibly the last straw.

Did Winegard see it coming? I had worried vaguely about such an eventuality, but didnt really think it would happen, he wrote. I naively assumed that the norms of academic freedom would prevail. They did not.

Winegard told Inside Higher Ed that hes had strong teaching evaluations and high research productivity since hes been at Marietta. He sees no apparent reason for his effective termination, apart from the controversy surrounding what he has said and, more to the point, is alleged to have said.

In response to his Quillette article, some have argued that one should wait until tenure to pursue certain topics. But Winegard reiterated that he, perhaps navely, took academic freedom seriously. Beyond that, he said, if academics follow "pragmatic" advice about waiting until tenure to discuss controversial issues, it means waiting 10 or more years, through graduate school and the tenure track.

Im perplexed by the response, he said of Mariettas actions. The best response would have been to come out with a bold, affirmative statement for academic freedom, even if the college distanced itself from Winegards views in doing so.

Otherwise, he said, Youre incentivizing this trollish behavior. Trollish here refers to those Winegard says emailed his institution about him anonymously.

Marietta declined comment, saying Winegards case was a private personnel issue.

Relevant, widely followed American Association of University Professors policy says that even professors on probationary appointments should enjoy the same academic freedom as those with tenure, even if they don't have the same due process protections. Winegard said he's unaware of any paths to appeal, but AAUP policy also holds that a faculty committee should evaluate any concerns about non-reappointment related to a possible violation of academic freedom.

Winegard's department chair did not respond to a request for comment. Marietta's Faculty Council chair also did not respond to questions about the case.

Facts and Feelings

Attempts to link cognition to race have for decades happened mostly in academe's fringes. That's because it's either dog-whistle racist junk science or there is a conspiracy of silence surrounding it, depending on what you believe. In 1994, Richard Herrnstein and Charles Murray's The Bell Curve: Intelligence and Class Structure in American Life was immediately controversial, stirring concerns about lack of peer review and whether it represented mainstream science.

Race-based science debates don't just happen in psychology. In January, for example, Philosophical Psychology faced a boycott for publishing an article in defense of race-based research on intelligence. The gist of that article, written by Nathan Cofnas, a Ph.D. candidate in philosophy at the University of Oxford, was that when advances in science reveal genetic variants underlying individual differences in intelligence, we wont be ready for it.

One of the main criticisms of Cofnas's piece was that it speculated that these breakthroughs are close. They are not. So postulating about them is, in a sense, pseudoscience, critics maintain.

Cofnas said at the time that those "who argue that we should wait for the genetics and neuroscience of intelligence to become more advanced before we attempt to study this issue often claim that, in the meantime, we should accept the environmental explanation for the purpose of policy making" and more. But that is a "political, not a scientific, position."

Journalist Angela Saini, author of the 2019 book Superior: The Return of Race Science (which Winegard has reviewed), said that her research demonstrates there is simply "no conspiracy against talking about race and IQ in academia, largely because this matter was settled 70 years ago -- and reinforced by genetics since -- by the universal understanding that race is a social construct."

It's "impossible to say that any differences in attainment we may see between socially defined groups must be biological in origin," Saini added. "Scientists are overwhelmingly in consensus on this."

That a "few academics like to claim otherwise," she said, "in particular, a small number of social scientists on the margins of respectable academia, does nothing to undermine the scientific facts. The facts, Im afraid, dont care about their feelings."

Intelligence researcher Richard Haier, professor emeritus in the pediatric neurology division at the School of Medicine at the University of California, Irvine, said that the questions Winegard is working on are controversial and emotional -- and well within the bounds of reasonable debate.

What happened at Marietta is, therefore, an apparent violation of academic freedom, Haier said. I dont know all the details, but I do know that it is very hard to defend academic freedom for issues that are not just controversial but also extremely emotional. And a lot of people in academia are happy to say that they support academic freedom but there are many examples of occurrences that appear to violate academic freedom, and the local academic community has not stood up for academic freedom.

Haier added, The hard thing about science is to go where the data take you. Without tenure and even with tenure, its becoming increasingly difficult to address controversial ideas, where some points of view do not acknowledge the legitimacy of other points of view, and therefore shut down discussion. Thats not how science works.

Lee Jussim, distinguished professor of psychology at Rutgers University and co-author of a recent paper on political bias in social science research, said that the topic of race and IQ "is poison." Further, he said, "I see no reason to believe the methods are capable of answering the question of how much race differences in intelligence are genetic versus environmental versus some combination.

That doesn't mean that Winegard or anyone else should be fired for trying to do so, however, Jussim said. Of course he has a right to pursue the line of inquiry.

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Interpace Diagnostics Issues Update Regarding Announced Contract with BCBS of Massachusetts – GlobeNewswire

Thursday, March 12th, 2020

Parsippany, NJ, March 11, 2020 (GLOBE NEWSWIRE) -- Interpace Diagnostics, a subsidiary of Interpace Biosciences (NASDAQ: IDXG) announced today that it has entered into a contract with Blue Cross Blue Shield of Massachusetts. While terms of this Agreement are not disclosed, Interpace is an in-network lab with Blue Cross Blue Shield of Massachusetts for all product lines.

Interpace continues to expand in-network agreements and medical coverage for its services with leading National and Regional health plans, while maintaining Medicare coverage through Novitas Solutions.

According to Jack Stover, CEO of Interpace, This contract with Blue Cross Blue Shield of Massachusetts continues our trend of establishing improved reimbursement through participation as an in-network provider. Im pleased to announce that this is the first contract secured by our new VP of Managed Care and Payer Relations, Jeff Salzman.

AboutThyroid Nodules, ThyGeNEXT and ThyraMIR Testing

According to theAmerican Thyroid Association, approximately 20% of the 525,000 thyroid fine needle aspirations (FNAs) performed on an annual basis in the U.S. are indeterminate for malignancy based on standard cytological evaluation, and thus are candidates for ThyGenX and ThyraMIR.

ThyGenX and ThyraMIR reflex testing yields high predictive value in determining the presence and absence of cancer in thyroid nodules. The combination of both tests can improve risk stratification and surgical decision-making when standard cytopathology does not provide a clear diagnosis for the presence of cancer.

ThyGenX utilizes state-of-the-art next-generation sequencing (NGS) to identify more than 100 genetic alterations associated with papillary and follicular thyroid carcinomas, the two most common forms of thyroid cancer. ThyraMIR is the first microRNA gene expression classifier. MicroRNAs are small, non-coding RNAs that bind to messenger RNA and regulate expression of genes involved in human cancers, including every subtype of thyroid cancer. ThyraMIR measures the expression of 10 microRNAs. Both ThyGenX and ThyraMIR are covered by both Medicare and Commercial insurers.

AboutInterpace Biosciences

Interpace Biosciences is a leader in enabling personalized medicine, offering specialized services along the therapeutic value chain from early diagnosis and prognostic planning to targeted therapeutic applications.

The Interpace Diagnostics division provides clinically useful molecular diagnostic tests, bioinformatics and pathology services for evaluating risk of cancer by leveraging the latest technology in personalized medicine for improved patient diagnosis and management. Interpace has four commercialized molecular tests and one test in a clinical evaluation process (CEP): PancraGEN for the diagnosis and prognosis of pancreatic cancer from pancreatic cysts; ThyGeNEXT for the diagnosis of thyroid cancer from thyroid nodules utilizing a next generation sequencing assay; ThyraMIR for the diagnosis of thyroid cancer from thyroid nodules utilizing a proprietary gene expression assay; and RespriDX that differentiates lung cancer of primary vs. metastatic origin. In addition, BarreGEN for Barretts Esophagus, is currently in a clinical evaluation program whereby we gather information from physicians using BarreGEN to assist us in positioning the product for full launch, partnering and potentially supporting reimbursement with payers.

The Interpace Pharma Solutions division provides pharmacogenomics testing, genotyping, biorepository and other customized services to the pharmaceutical and biotech industries. The Pharma Solutions Business also advances personalized medicine by partnering with pharmaceutical, academic, and technology leaders to effectively integrate pharmacogenomics into their drug development and clinical trial programs with the goals of delivering safer, more effective drugs to market more quickly, and improving patient care.

For more information, please visit Interpace Biosciences website at http://www.interpace.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, relating to the Company's future financial and operating performance. The Company has attempted to identify forward looking statements by terminology including "believes," "estimates," "anticipates," "expects," "plans," "projects," "intends," "potential," "may," "could," "might," "will," "should," "approximately" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are based on current expectations, assumptions and uncertainties involving judgments about, among other things, future economic, competitive and market conditions and future business decisions, all of which are difficult or impossible to predict accurately and many of which are beyond the Company's control. These statements also involve known and unknown risks, uncertainties and other factors that may cause the Company's actual results to be materially different from those expressed or implied by any forward-looking statement. Additionally, all forward-looking statements are subject to the risk factors detailed from time to time in the Company's filings with the SEC, including without limitation, the Annual Report on Form 10-K and the companys Quarterly Reports filed with the SEC. Because of these and other risks, uncertainties and assumptions, undue reliance should not be placed on these forward-looking statements. In addition, these statements speak only as of the date of this press release and, except as may be required by law, the Company undertakes no obligation to revise or update publicly any forward-looking statements for any reason.

CONTACTS:

Interpace DiagnosticsInvestor Relations:Joe Green(646) 653-7030jgreen@edisongroup.comEdison Group

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Podcast: A family on the frontier of hyper-personalized medicine – MIT Technology Review

Wednesday, March 11th, 2020

Deep Tech is a new subscriber-only podcast that brings alive the people and ideas in our print magazine. Episodes will be released every two weeks. Were making the first four installments, built around our 10 Breakthrough Technologies issue, available for free.

Three-year-old Ipek Kuzu has an extremely rare genetic mutation that disrupts a protein needed for DNA repair, causing the loss of brain cells. Now shes become only the second person in the world to receive a customized antisense oligonucleotide drug designed to compensate for the DNA mistake by allowing her cells to splice together a functional version of the protein. The drug took Boston-based pediatrician and geneticist Tim Yu only months to create, heralding a new era of individualized genomic medicine. But it cost $2 million to manufacture and testleading to questions about how soon hyper-personalized treatments for rare genetic disorders can be made accessible and affordable. Journalist Erika Check Hayden got to know the Kuzu family, and in this episode she chronicles Ipeks journey, with help from Ipeks father Mehmet and Technology Review biomedicine editor Antonio Regalado.

Show notes and links:

If DNA is like software, can we just fix the code?, from the March/April 2020 print issue, p. 46

Hyper-personalized medicine, from the March/April 2020 print issue, p. 18

Sign up for The Download your daily dose of what's up in emerging technology

Two sick children and a $1.5 million bill: One family's race for a gene therapy cure, from the November/December 2018 print issue, October 23, 2018

Episode Transcript

Audio ID: This is MIT Technology Review.

Mehmet Kuzu: Around five to six months, they said she has something called ataxia telangectasia. And they said this doesnt have any cure. The initial days were very tough. We were crying all the time. So then after a while, we started investigating what can be done.

Wade Roush: Mehmet Kuzus three-year-old daughter, Ipek, has a rare genetic mutation that could end her life by age 25. But now shes getting a so-called antisense drug that her doctors engineered specifically for her. Which makes Ipek one of the first patients being swept up in a new wave of hyperpersonalized medicine. Journalist Erika Check Hayden wrote about the Kuzu family in the latest issue of Technology Review. And today, she helps us understand where this breakthrough came from, and how soon it might be scaled up. Im Wade Roush, and this is Deep Tech.

[Theme music]

Were right at the beginning of a revolution in individualized genomic medicine. And if you want to know what that revolution sounds like, this is a good place to start.

[Sound of Illumina sequencing machines]

Thats one of the hundreds of high-speed gene sequencing machines at the Broad Institute of MIT and Harvard. Here at the Broads genomics platform in Cambridge there are so many of these machines that the institute can read the equivalent of 30 whole human genomes every 10 minutes.

There arent a lot of research centers with that kind of power. But in many places around the world its now possible to scan a babys full genome for just a few hundred dollars, and locate DNA coding errors that can cause rare conditions like ataxia telangectasia.

Thats how doctors diagnosed Ipek Kuzu when she was just six months old. The mistake in her DNA means her cells cant make a protein called ATM thats essential for DNA repair. Over the long run that causes the loss of brain cells, which means Ipek has some trouble walking and doesnt talk as much as a typical three-year-old.

Today Ipek is receiving an antisense drug made just for her. Its designed to compensate for the DNA mistake and restore production of ATM. Which makes her only the second person in the world to get this kind of treatment. The first was another little girl named Mila Makovec. She has different genetic disorder called Batten disease that causes blindness, seizures, and other neurodegenerative problems. And Mila got her own customized antisense drug starting in 2018.

But to understand how her doctors came up with these two medicines, and why this whole field of hyperpersonalized medicine is so hot that the editors of Technology Review decided to put it on this years list of 10 breakthrough technologies, we first have to jump back a few years, to 2016.

[CNBC Squawk Box news clip]

CNBC male anchor: Ionis Pharmaceuticals, in pre-market trading, is higher. The FDA has approved a drug called Spinraza. Spinraza.

CNBC female anchor: Its not Spine-raza?

CNBC male anchor: Maybe it is. Because its for spinal muscular atrophy. Its the first drug approved to treat the rare and fatal disease.

Wade Roush: Spinal muscular atrophy affects about 1 in 10,000 babies. So its not nearly as rare as Batten Disease or ataxia telangectasia. But Spinraza is literally the key to all of the more recent work to make customized antisense drugs for Mila and Ipek. So lets take a minute to go over how it works.

What made Spinraza a big deal is that it was one of the first successful medicines made using an antisense oligonucleotide. In other words, a customized strand of RNA.

Antonio Regalado: If you can imagine, inside a cell, there's the DNA.

Wade Roush: This is Antonio Regalado, the editor for biomedicine at Technology Review.

Antonio Regalado: And it kind of sends out these messages into the nucleus made of RNA and those are used as the templates to make proteins. And so antisense is a drug that acts at the level of RNA. They're going to stick to that RNA message and they could block it.

Wade Roush: Keep it from being translated.

Antonio Regalado: Keep it from being translated, or modify the translation in some fashion.

Wade Roush: In the cells of healthy people, theres a protein called SMN that helps motor neurons survive and grow. A gene called SMN1 carries the instructions for making that protein, and people with spinal muscular atrophy have a mutation that disables that gene. But it just so happens that human DNA also contains a second copy of the gene, called SMN2. This second copy is typically inactive, thanks to a small error that keeps the RNA message from being spliced together into a proper template. The Spinraza molecule contains a short segment of antisense RNA that prevents the splicing error. And that allows the body to start making the motor neuron protein.

Ionis Pharmaceuticals is the company that makes Spinraza, and they put a lot of work into figuring out how to get their molecule into cells in the brain and the nervous system, where it can do its work.

Antonio Regalado: And they finally mastered it and came up with pretty much kind of a miracle drug for one of these rare brain diseases that affects kids, spinal muscular atrophy. And so from that example, people then said, well, why can't we use antisense for other diseases that are similar?

And what we learned was that there was a doctor in Boston named Timothy Yu, who was an expert in sequencing genomes of sick children. And there was one girl named Mila Makovec. And her parents had come to him. He'd sequenced the genome. And then he just realized, I don't have to stop here. Once I've identified this defect, I don't have to stop. I could potentially make a drug. And so that's exactly what he did.

Wade Roush: It turned out that Milas disease was caused by a splicing error very similar to the one that causes spinal muscular atrophy, except that in Milas case it disrupts a different protein called CLN7. Tim Yus idea was to take the backbone of the Spinraza molecule and attach a customized strand of antisense RNA. With this new business end, so to speak, the drug would enable Milas cells to start making functional copies of the CLN7 protein.

Antonio Regalado: That was probably at that point just the clearest, starkest, most stunning example of this hyper personalized medicine. Because in this case, it was really for one person. So we were very interested in this phenomenon, because it's a reflection of what technology can do. And then in the middle of last year, a pretty prominent journalist, Erika Check Hayden, came to us and she was also interested and wanted to do some work to find the cases, find the families and write more stories about it. And as it developed, we decided, well, let's put this on our list of breakthrough technologies, because it really is. And so Erika ended up writing the piece and she did a lot of work to find the patients. One of the great things she did was to find this Kuzu family, which happens to be right here in Cambridge.

Wade Roush: Erika, could you introduce yourself and tell us a little bit about you?

Erika Check Hayden: Sure. My name is Erika Check Hayden. I'm a journalist based in San Francisco. And I also run the science communication program at the University of California, Santa Cruz.

Wade Roush: When you set out to start reporting this piece, did you feel like it was important to go beyond the first sort of headline-making case of Mila Makovec and look for additional patients who were going through this process to see how broadly applicable the whole idea is?

Erika Check Hayden: I do think that while people have been very impressed by Mila's case and by the drug that Tim Yu made for her, which is called milasen, I think there's also been this question of are we gonna be able to do this for other patients? And if so, you know, who is going to be treatable via this method? And so if I'm going out and finding other families that are hopefully replicating that success, I think is a really important statement about how impactful this approach might eventually be.

Wade Roush: So this is where the Kuzu family comes in. So could you tell us a little bit about them and how you got in touch with them?

Erika Check Hayden: So the Kuzu family, they originally came from Turkey and the father in the family, Mehmet Kuzu, is now a software engineer at Google. And they were living in Silicon Valley when their daughter Ipek was born. And soon after she was born, she was diagnosed with this disease called ataxia telangectasia, which is also called A-T disease. And when that happened, they set about trying to understand if there was anything they could do to treat the disease or slow the disease. And that's what led Mehmet down this path that eventually led him to work with Tim Yu.

Mehmet Kuzu: I sent the genetic report of our daughter. Then he said, oh, there's a potential here, but there are two main problems. He said this might cost around like two million, and the insurance will not cover it. The second problem, it might cause damage because, we have a theoretical idea, but biology is complicated. So at the end of the day, it might be worse than what is expected.

Wade Roush: Right. So for the Kuzu family, while it was obviously bad news that your kid is getting diagnosed with A-T disease, there is this amazing foundation or non-profit led by Brag Margus, the A-T Children's Project, that has all this data and also apparently has some fundraising clout. And they wind up helping to finance a lot of this research and even finance Ipeks treatment.

Erika Check Hayden: Right. And I think that's part of why this particular project was able to move so fast, because Brad Margus and the A-T Children's Project had done a lot of work over the years to fundraise and educate their community about the potential for treating this disease, so that when they found something that he actually thought could work, they were able to raise $1.4 million in a relatively short amount of time to fund the development of this unique drug.

Mehmet Kuzu: I think he understood to the promise of it. And then he agreed to financially support us. But the problem is this money in the pool is coming from many families. So we should have a fair selection. Then they found three kids that young in age, like three, two, two, three, four, with the right mutation type, and they got skin samples from all of them, and tested it. They were able to do it quickly.

Wade Roush: Mehmet can recount all these events pretty calmly. But I think its worth underscoring what a roller coaster the familys been on. The backing of the AT Childrens Project opened a window for Tim Yu to design and manufacture an antisense drug. But the required safety testing is so expensive that only there was only enough money to do that for one patient. There was a two in three chance that Ipek would not be that patient. And even if she did get selected, there was no way to know whether the treatment would be effective. Mila Makovec had been having fewer seizures since she started getting her antisense treatment, but doctors still werent 100 percent sure that it was because of the medicine. On top of all that, there was still the risk of unintended side effects.

Mehmet Kuzu: and then at the end of the day, Ipeks cells responded the best among these three candidates. Now, once we know we are selected, now we concentrate on second issue: do we really want to take this risk of, like, making things worse? And then I thought, like, most probably something good will happen. Of course there is a probability of, a possibility [of failure]. But imagine if that happens: science will learn from this. And her kind of sacrifice, and that would help, too, many other people.

Erika Check Hayden: It's been just incredible over the past few years to meet these families, understand what they're doing, how they're doing it. I've just been really struck by everything they've been able to accomplish. And also the mindset that they bring to this where, you know, you'll talk to, or I will talk to, parents who are doing this for their kids and they've had scientists tell them, 'You've got to be prepared for the possibility that this isn't going to help your kid. You know, you might be doing all of this work on behalf of some other future child. This might not come in time to help your own child.' And they persist and are really driven.

Wade Roush: Ok. So in the same way that Tim Yu helped to create this unique drug called milasen for Mila Makovec, he's created a drug called atipeksen for Ipek. If that drug if that drug works, how will it help Ipek?

Erika Check Hayden: If this drug works, basically what it's going to do is correct the way that Ipek's cells interpret her genetic information so that she will make a functioning copy of the ATM protein. Now, how we will know if this is working is a bit of a tricky question. So, Tim Yu and other doctors are going to try a variety of methods to see if they can tell whether the drug is actually helping her. So, for instance, they will look at things like can they see evidence in Ipek's body that the drug is actually making corrected versions of the protein? They will look for evidence that she isn't declining in the ways that we might expect her to if she wasn't getting treatment to help control her disease. But it might be tricky to tell whether it actually works or not.

Mehmet Kuzu: She had three injections until this point because they are starting with very low dose and escalating itAnd fortunately, we haven't seen any adverse effects in the first three. But like, of course, knowing if this is really working or not, they told us that it will take time. Maybe we need a year to understand if it's really working. But at least we have seen that no bad thing happened. At hospital she's going on the full anesthesia. They're putting on a mask. And after the injection they are taking bloods every four hours, three or four times. These are very stressful for her. She's fighting not to have this mask. She's crying a lot. Uh, but once discharge happens, once we come home, she forgets about everything. She just plays with her toys.

Wade Roush: Right. And this is one of the things you mentioned in your piece. Not only will it be tricky to see whether it's working or not, but we're talking about by definition an n of one study where there's only one patient. So you don't get the kinds of large numbers that help researchers feel more confident that a drug is safe and effective.

Erika Check Hayden: I think what we still don't know very well yet is which diseases are going to be helped most by this approach, or even if any of these individual customized treatments can cure a patient. So if you talk to Mila's mom, Julia Vitarello, she is very convinced that that drug has helped Mila. But I think accumulating that data to the level where we really know that this is a worthwhile approach, you know, that's probably going to take a while.

And to take a step back, I think that's part of the reason why these drugs are only being used right now in patients that have really severe progressive diseases, because you are taking a certain risk by giving a treatment to a patient when you haven't done the kinds of safety testing that we might be used to for a drug that would normally go through an FDA approval process. In fact, there are some people who object to even using the word treatment because we don't necessarily know that these drugs are going to cure the patients.

So in the meantime, I think everybody would like to see far more patients at least be able to try this. And so there's this question as to whether it's only going to be patients who have the resources to raise that money or access that money that are going to benefit. And I don't think anybody wants that to be the case.

Wade Roush: Are there any signs that the drug industry is looking at how to scale up some of these treatments? And, you know, maybe create a pipeline for hyper personalized drugs?

Erika Check Hayden: So we're seeing things like Ionis, their co-founder Stan Crooke has started a foundation called the n-Lorem Foundation that's going to try to develop these treatments for patients. The reason is that developing a drug for one patient that costs millions of dollars and doesn't really have a very large market is not something that's necessarily going to be attractive to a company. But I think people think there is a direction that could evolve where, you know, if the drug industry is better able to manufacture these drug templates or backbones and more easily switch out the part of the drug that's the business end that's doing the targeting of different genetic diseases to where that becomes much more large scale, much more customizable, much cheaper. You know, then you might see a model where this is much more economical, affordable, reimbursed by insurance companies, because right now this is not and obviously that's a major cost barrier.

Wade Roush: Do you think this is a time for patients with rare genetic disorders and families of those patients to feel more hopeful? Or is it just too early realistically for this to affect lots of people who are already suffering from these conditions?

Antonio Regalado: Right. It goes back to the question, should this be a breakthrough technology? Because right now, it's not helping that many people. We're talking about helping one person. Or we're talking about helping two or three very few people. Very few. And that's a strike against the idea, frankly. Like, why? Why should we invest resources into this when it helps so few people? Why should we call it a breakthrough technology? Well, the reason to is, it's sweet. Technically, it's sweet. And it paints a path towards a future where it like you can do a lot more with genetic drugs.

Wade Roush: So you can imagine a future not 100 years away, but maybe 10 years away, where this can be scaled up and broadened out to more patients.

Antonio Regalado: Yeah, absolutely. I mean, will the drugs work? How well will they work? It's kind of an open question. But yeah, we've already gone from one case to five cases next year no doubt it it'll be 10 and then a hundred and then thousands. Most likely. I want to raise something else, which this whole scenario is not fair. Because there's a lot of people with rare diseases and a lot of kids dying of rare disease in every neighborhood and every corner and every precinct of the country, of the world. So who has the opportunity to have this chance?

Wade Roush: Well, who does so far?

Antonio Regalado: Well, it is a very small subset of parents who for whatever reason have the ability to wrap their head around the science, to find where the opportunity is, and to raise quite a lot of money. And this is not bake sale money. This is two million dollars. Three million dollars. You have to really have a way to do that, and it favors people with a big network. That's why we're seeing people, you know, entrepreneurs from Silicon Valley or other people who just for whatever reason, manage to pull it off.

Wade Roush: If this kind of inequity persisted, it would definitely become a huge point of criticism around this whole area of therapy. But maybe you could look at these parents as the pioneers.

Antonio Regalado: Right. Exactly. A lot of the parents will say, well, in addition to trying to help my child, I also want to invest and try and create the process by which everybody else can be helped because they also have a lot of empathy for the next person. The idea is to help everybody. The pathway to doing that is not clear yet.

Wade Roush: All right. Well, whether this is a breakthrough or not, it raises so many interesting and thorny questions that it's perfect fodder for Technology Review.

Antonio: It's definitely a breakthrough, man. It's definitely a breakthrough.

Wade Roush: Okay. Thanks Antonio.

[Theme music]

Thats it for this edition of Deep Tech. This is a podcast were making exclusively for MIT Technology Review subscribers,to help bring alive some of the people and ideas youll find in the pages of our website and our print magazine. But the first four episodes cover our annual 10 breakthrough technologies issue, and were making those episodes free for everyone.

Deep Tech is written and produced by me and edited by Michael Reilly, with editorial help this week from Jennifer Strong. Our theme is by Titlecard Music and Sound in Boston. Special thanks this week to David Cameron, Howard Gelman, Erika Check Hayden, Mehmet Kuzu, Antonio Regalado, and Jane Wilkinson. Im Wade Roush. Thanks for listening, and we hope to see you back here for our next episode in two weeks.

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Henry Ford Receives $25M Grant to Expand Precision Medicine Program – HealthITAnalytics.com

Wednesday, March 11th, 2020

March 11, 2020 -Henry Ford Health System has received a $25 million grant to accelerate its precision medicine program, with the ultimate goal of creating a Precision Health Center.

With the donation, Henry Ford will focus on advancing cancer research and treatment, as well as precision therapies for behavioral health, cardiovascular conditions, and metabolic diseases.

Henry Ford received the gift from developer Chris Jeffries and his wife Lisa. The donation is the largest single gift from an individual in the health systems 105-year history.

We are incredibly grateful to Lisa and Chris Jeffries for their generosity, saidWright Lassiter, III, president and CEO of Henry Ford Health System.

We are experiencing a momentous era in medicine, a radical shift from the traditional approach to cancer care. This gift will help us consolidate and advance our collective efforts to create unprecedented access to advanced, highly personalized treatments for our patients and members.

The grant will significantly boost the health systems translational research efforts, which quickly transforms the most innovative discoveries in the lab into new treatments for patients.

Translational research is a significant differentiator of our clinical programs at Henry Ford and is a critical element to help us treat many of the most challenging conditions our patients face, saidAdnan Munkarah, MD, executive vice president and chief clinical officer of Henry Ford Health System.

Translational research is bench-to-bedside, meaning it allows patients to benefit from discoveries in real time. That is an essential part of our history and commitment to medicine and academics not only offering the latest innovations in medicine, but also playing a leading role in their development.

The donation will build on the organizations past work to advance precision medicine and personalized care. In October 2017, Henry Ford Health System launched the Henry Ford Cancer Institute, a facility focused on ambulatory cancer treatment, precision medicine, clinical trials and research, and enhanced support services for cancer patients.

With the grant, researchers will be able to continue to develop individualized therapies for cancer and other conditions.

By analyzing genetic and non-genetic factors, we can gain a better understanding of how a disease forms, progresses and can be treated in a specific patient, saidTom Mikkelsen, MD, medical director of the Precision Medicine Program andClinical Trials Officeat Henry Ford Health System.

As of now, we can check for more than 500 genomic markers, which helps us understand the pattern of changes in a patients tumor cells that influence how cancer grows and spreads. Im confident this gift will lead to advancements that provide hope for patients with even the most complex diagnoses.

The Henry Ford Cancer Institute has one unified team of cancer specialists working to deliver personalized cancer treatments. The Institute includes five hospital locations, six additional outpatient cancer centers, and dozens of aligned doctors offices.

Even a decade ago, our approach to treating brain cancer was Precision Medicine before anyone knew what Precision Medicine was, said Steven Kalkanis, MD, CEO of Henry Ford Medical Group and Henry Fords chief academic officer.

In the time since, weve seen a significant increase in the number of brain cancer patients who are outliving their prognoses, due in large part to clinical innovation. Our relentless pursuit of clinical breakthroughs has more momentum now than at any other point in history.

The new grant will only serve to accelerate precision medicine in care delivery.

The support of our donors is the fuel behind our clinical innovations and the breakthroughs that are improving peoples lives, saidMary Jane Vogt, senior vice president and chief development officer at Henry Ford Health System. It is remarkable to work with donors who believe in a better tomorrow and the power of a unified approach to medicine.

The donation is expected to help drive innovations in treating brain, lung, pancreatic, and colon cancers, as well as other chronic diseases like cystic fibrosis, asthma, and heart disease.

The team at Henry Ford is second to none, said Chris Jeffries. We believe this gift will lead to other families having more time together. Defeating cancer requires a concerted effort from everyone and we hope to make as big an impact on that goal as possible.

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DNA Sequencing Industry Insights and Outlook to 2028 – Analysis of Applications in Healthcare, Oncology and Infections – Benzinga

Wednesday, March 11th, 2020

Dublin, March 11, 2020 (GLOBE NEWSWIRE) -- The "DNA Sequencing - Technologies, Markets & Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

The value of DNA sequencer market in 2018 is described with estimates for 2023 and 2028. Various methods and factors on which market estimates depend are described briefly. Markets are tabulated according to geographical areas as well as applications. Small sequencers form the basis of SWOT (strengths, weaknesses, opportunities, threats) analysis. Several marketing strategies have been outlined. The report includes profiles of 147 companies involved in sequencing and their 173 collaborations.

The report briefly reviews basics of human genome variations, development of sequencing technologies, and their applications. Current large and small sequencers are described as well as companies developing them. Various applications of sequencing are described including those for genetics, medical diagnostics, drug discovery and cancer.

Next generation sequencing technologies, both second and third generations, are reviewed. Companies developing software for analysis of sequencing data are also included. Selected academic institutes conducting research in sequencing are also listed.

Current market is mostly for research applications and future markets will be other applications related to healthcare.

Key Topics Covered

Executive Summary 1. Introduction2. DNA Sequencing Technologies3. Role of Bioinformatics in Sequencing4. Comparative Analysis of Sequencing Technologies5. Sequencing for Research6. Applications of Sequencing in Healthcare7. Applications of Sequencing in Oncology8. Sequencing in Genetic Disorders9. Sequencing in Neurological and Psychiatric Disorders10. Applications of Sequencing in Infections11. Role of Sequencing in Personalized Medicine12. Current Status and Future Prospects13. Markets for Sequencers14. Companies Involved in Sequencing15. References

For more information about this report visit https://www.researchandmarkets.com/r/8bubjr

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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DNA Sequencing Industry Insights and Outlook to 2028 - Analysis of Applications in Healthcare, Oncology and Infections - Benzinga

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Ethos Therapy Continues Global Expansion – PRNewswire

Wednesday, March 11th, 2020

PALO ALTO, Calif., March 10, 2020 /PRNewswire/ -- Varian (NYSE: VAR) today announced Medisch Spectrum Twente Hospital in The Netherlands, and Icon Cancer Centre Wahroonga and Royal North Shore Hospital (RNSH) in Australia treated their first patients with Ethos therapy. Thisartificial intelligence (AI)-driven holistic adaptive therapy solution is designed to deliver an entire adapted treatment in a typical 15-minute timeslot.

Adaptive therapy provides the ability to personalize the patient's treatment based on their anatomy and position at the time of treatment. The goal is to better target the tumor, reduce dose to healthy tissue, and potentially improve overall outcomes.

At Medisch Spectrum Twente Hospital, the first two patients treated were for prostate cancer, at RNSH the first treatment was for head and neck cancer and at Icon Cancer Centre Wahroonga, the first treatment was for prostate cancer. Additionally, since delivering the first Ethos therapy in the world in September 2019, Herlev and Gentofte Hospitalin Denmark has already delivered 100 adaptive fractions for bladder cancer patients.

"Since the launch of Ethos therapy, the response from clinicians globally has been very strong," said Chris Toth, president Varian Oncology Systems. "Ethos therapy was designed to launch a new era of personalized adaptive radiation therapy and we are very pleased to see patients in Australia and The Netherlands now have access to these adaptive treatments. With Ethos therapy recently receiving 510(k) clearance, the first installation in the US is in process and will be treating patients soon."

Clinician Experience

"The future is adaptive," said Erik Van Dieren, head of Medical Physics, Medisch Spectrum Twente Hospital."With Ethos we know adaptive radiotherapy on a daily basis is achievable for a large number of patients due to high accuracy and excellent sparing of the healthy tissue in about 15 minutes treatment time."

"Early Ethos therapy experience from Icon is showing promising results," said Amy Teh, MD, radiation oncologist at Icon Cancer Centre, Wahroonga."In a prostate patient, where the target volume is highly dependent on bladder and rectal positioning, we have used the AI-driven online adaptive workflow on the Ethos platform to effectively and efficiently adapt to the new position of the bladder and rectum each day. This has allowed superior coverage of the true target. This technology marks another step forward in the advancement of radiation therapy taking personalized medicine to another level allowing us to ensure more dose to the tumor target, and less dose to surrounding healthy organs."

"RNSH is very pleased to enter the world of Ethos therapy after recently treating our first patient," said Jeremy Booth, head of Medical Physics, Northern Sydney Cancer Centre, RNSH. "The patient treatment for head and neck cancer was an exceptional experience, uniting our expert team of radiation therapists, medical physicists and radiation oncologists at the console to ensure we safely delivered the best treatment."

"We've found that, with bladder cancer patients, we are seeing the most impact using online adaptation," said Poul Geertsen, MD, PhD, head of Radiotherapy, Department of Oncology at Herlev and Gentofte Hospital. "With Ethos therapy, we are seeing treatment margin reductions of up to 50 percent, which is impressive."

The streamlined workflow of Ethos therapy is enabled by its AI-driven planning and contouring capabilities. Physicians define their clinical intent from pre-defined templates and the initial treatment plan is generated based on the physician's pre-defined clinical objectives. The treatment is adapted in response to changes in the patient's anatomy and the tumor's shape and position, at the time of treatment. The ability of Ethos to enable on-couch adaptive treatment puts the patient at the center of care.

Ethos therapy offers the use of multimodality images (MR, PET, CT) registered with daily iterative CBCT images at the console. By providing an up-to-date view of the patient's anatomy in multiple imaging modality views, Ethos therapy provides clinicians the confidence to make more informed adaptive treatment decisions. The solution is built on Varian's latest treatment delivery technology andprovides fast imaging and treatment delivery without compromising quality.

For more information on Ethos, visit http://www.varian.com/ethos.

About Varian

At Varian, we envision a world without fear of cancer. For more than 70 years, we have developed, built and delivered innovative cancer care technologies and solutions for our clinical partners around the globe to help them treat millions of patients each year. With an Intelligent Cancer Care approach, we are harnessing advanced technologies like artificial intelligence, machine learning and data analytics to enhance cancer treatment and expand access to care. Our 10,000 employees across 70 locations keep the patient and our clinical partners at the center of our thinking as we power new victories in cancer care. Because, for cancer patients everywhere, their fight is our fight. For more information, visit http://www.varian.comand follow @VarianMedSys on Twitter.

Customers were not paid for their testimonials. Individual results may vary

Press Contact

Mark PlungyDirector, Global Public Relations+1 (650) 424-5630 [emailprotected]

Investor Relations Contact

Anshul MaheshwariVice President, Investor Relations+1 (650) 424-5631 [emailprotected]

SOURCE Varian

https://www.varian.com/

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Global Therapeutic Drug Monitoring Industry Insights, 2018-2028: Unmet Needs and Strategies for Development – MENAFN.COM

Wednesday, March 11th, 2020

(MENAFN - GlobeNewsWire - Nasdaq) Dublin, March 11, 2020 (GLOBE NEWSWIRE) -- The "Therapeutic Drug Monitoring (TDM) Technologies, Markets & Companies to 2028" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

This report deals with therapeutic drug monitoring, a multi-disciplinary clinical specialty, aimed at improving patient care by monitoring drug levels in the blood to individually adjust the dose of drugs for improving outcome. TDM is viewed as a component of personalized medicine that interacts with several other disciplines including pharmacokinetics and pharmacogenetics. One chapter is devoted to the monitoring of drugs of abuse (DoA). Various technologies used for well-known DoA are described. A section on drug abuse describes methods of detection of performance-enhancing drugs.

TDM market is analyzed from 2018 to 2028 according to technologies as well as geographical distribution. The global market for DoA testing was also analyzed from 2018 to 2028 and divided according to the area of application. Unmet needs and strategies for development of markets for TDM are discussed. The report contains profiles of 35 companies involved in developing tests and equipment for drug monitoring along with their collaborations. The text is supplemented with 21 tables, 9 figures and 210 selected references from the literature.

Benefits of the Report

The report contains information on the following:

Key Topics Covered Executive Summary

1. Introduction

2. Technologies for TDM

3. Drug Monitoring Instruments

4. Applications of TDM

5. Drugs Requiring Monitoring

6. Monitoring of Biological Therapies

7. Monitoring of Drug Abuse

8. Markets for TDM

9. Companies

10. References

For more information about this report visit https://www.researchandmarkets.com/r/pclxni

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

MENAFN1103202000703653ID1099832888

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Scientists developed the worlds most sophisticated lab model of the human body – Tech Explorist

Wednesday, March 11th, 2020

Current practices in drug development have led to therapeutic compounds being approved for widespread use in humans, only to be later withdrawn due to unanticipated toxicity. These occurrences are mostly the result of erroneous data generated by in vivo and in vitro preclinical models that do not accurately recapitulate human physiology.

To speed up new drugs to market and reduce animal testing, scientists from the Wake Forest Institute for Regenerative Medicine (WFIRM) have come up with a mindblowing solution. They have developed the worlds most sophisticated laboratory model of the human body, a system of miniaturized organs that can be used to detect harmful and adverse effects of drugs before they are prescribed to patients.

Scientists developed this system from many human cell types that are combined into human tissues representing a majority of the organs in the human body, such as the heart, liver, and lungs. Each of these miniature organs is tiny 3D tissue-like structures about one-millionth the size of an adult human organ.

Anthony Atala, MD, of the Wake Forest Institute for Regenerative Medicine and the studys senior author said,The most important capability of the human organ tissue system is the ability to determine whether or not a drug is toxic to humans very early in development and its potential use in personalized medicine. Weeding out problematic drugs early in the development or therapy process can save billions of dollars and potentially save lives.

During the experiment, this new model shows the potential of quantifying toxicity measure toxicity in many drugs approved for human use. Although toxicity from the recalled drugs was not found initially using standard 2D cell culture systems and animal testing models, and adverse effects were not detected throughout three levels of human clinical trials, this new system can readily detect toxicity, replicating the damage seen in humans.

To create the model, scientists isolated tiny samples of human tissue cells and engineered them into miniature versions of the human organ. These tissue cells can contain blood vessel cells, immune system cells, and even fibroblasts.

Each of these organs, also known as organ tissue equivalents, performs the same functions that they do in the human body. For example, the heart beats about 60 times each minute, the lung breaths the air from the surrounding environment, and the liver breaks down toxic compounds into harmless waste products.

Co-author Aleks Skardal, Ph.D., formerly of WFIRM and now at Ohio State University, said,We knew very early on that we needed to include all of the major cell types that were present in the original organ. To model the bodys different responses to toxic compounds, we needed to include all of the cell types that produce these responses.

Another exciting thing about the model that each system contains media, a substance containing nutrients and oxygen that is circulated among all the organ types, delivering oxygen, and removing waste. The small blood system n these devices use a technology called microfluidics to recirculate test compounds through the organ system and remove the drug breakdown products that each organ is producing.

Co-author Thomas Shupe, Ph.D., of WFIRM, said,Creating little human organs for drug testing was a logical extension of the work we have accomplished in building human-scale organs. Many of the same technologies we have developed at the human-scale level, like including a very natural environment for the cells to live in, also produced excellent results when brought down to the microscopic level.

Additional co-authors include Julio Aleman, Steven Forsythe, Shiny Rajan, Sean Murphy, Mahesh Devarasetty, Nima Pourhabibi Zarandi, Goodwell Nzou, Robert Wicks, Hooman Sadri-Ardekani, Colin Bishop, Shay Soker, and Adam Hall.

Authors Skardal, Shupe, Soker, Murphy, Bishop, and Atala are inventors on patent rights related to this work owned by Wake Forest University Health Sciences. The patents, whose value may be affected by publication, have the potential to generate royalty income in which the inventors would share.

The study is published in the journal Biofabrication.

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3 steps to add annual Medicare wellness visits in your practice – American Medical Association

Wednesday, March 11th, 2020

Medicare now recognizes the important work that primary care physicians do when it comes to preventive screenings in older patients. So instead of only paying doctors for sick visits, the program will pay physicians to perform a preventive annual Medicare wellness visit (AWV).

But physicians should make some adjustments to their practices to ensure they adapt to what is the first of several Medicare changes intended to promote preventive care and improve care coordination and chronic disease management for Medicare patients.

The AMA STEPS Forward module Medicare Annual Wellness Visit (AWV): Streamline Workflow to Perform a Thorough AWV helps physicians understand the AWV, communicate with patients to set expectations about the visit and map out an AWV workflow.

TheAMAsSTEPS Forward open-access modules offerinnovative strategies that allow physicians and their staff to thrive in the new health care environment. These courses can help you prevent physician burnout, create the organizational foundation for joy in medicine and improvepractice efficiency.

The AWV is a primary care visit that involves preventive care, advanced care planning and depression and dementia screening. It gives physicians a chance to focus on safety issues, such as falls, and social needs, such as food insecurity and transportation. Physicians and their teams can update information in a patients chart, such as a medication list, or they can create and maintain a personalized screening and prevention plan.

The Centers for Medicare and Medicaid Services (CMS) is recognizing that these visits help enhance a patients quality of life and that they are different from traditional sick visits. Identifying mental health concerns, cognitive impairments and other factors often involves in-depth conversations and non-face-to-face work. And physician offices can set up a system where all members of the care team contribute to the effort, maximizing patient benefit, practice pay and time savings.

Here are three steps to optimize annual wellness visits in your practice.

An annual wellness visit is different from the initial preventive physical examination, known as the IPPE. AWVs are offered to patients 12 months after they enroll in Medicare Part B and they are then covered once every 12 months.

Numerous components are part of the initial AWV, including screening for cognitive impairment and reviewing functional ability and level of safety. Nonphysician members of the care team can perform most of the components; the physicians role is to synthesize the findings and provide recommendations. During subsequent visits, the information is reviewed and updated.

Make clear this visit is not the same as an annual physical and doesnt include a physical exam. Medicare covers the AWV 100%, but any evaluation and management (E/M) work done during the visit is subject to copays, deductibles and coinsurance.

An AWV is covered only once every 12 months, so if a patient has had one elsewhere in that timeframe, they are not eligible for another one from your office.

First, physician offices will need to decide whether to combine AWV and problem-based visits. The STEPS Forward module then offers a sample process map as guidance on how to map out each step of the visit.

For example, if an office chooses to combine an AWV with E&M, the module outlines how to conduct and document the visit using both AWV and E&M templates.

TheCMEmodule, Medicare Annual Wellness Visit (AWV) Streamline Workflow to Perform a Thorough AWV,is enduringmaterial and designated by the AMA for a maximum of0.5AMA PRA Category 1 Credit.

The module is part of theAMA EdHub,anonline platformwith top-quality CME and education that supports the professional development needs of physicians and other health professionals. With topics relevant to you, it also offers an easy, streamlined way to find, take, track and report educational activities.

Learn more aboutAMA CME accreditation.

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Podcast: Treating blindness with CRISPR; customized cancer drugs; Beyond Meat takes on critics; and saving bananas from extinction – Genetic Literacy…

Wednesday, March 11th, 2020

For the first time ever, CRISPR has been used to edit DNA inside a living human being. Scientists have also tapped the gene-editing tool to accelerate DNA sequencing in hopes of customizing cancer treatments. Plant-based burger startup Beyond Meat blasts critics who claim its products are ultra-processed. Genetic engineering may save the worlds favorite banana from extinction. But how does the public feel about all this genetic tinkering?

On this episode of Science Facts & Fallacies, plant geneticist Kevin Folta and GLP editor Cameron English go beyond the headlines to break down the latest developments from the world of genetics and biotechnology.

Podcast: Play in new window | Download

Subscribe: Apple Podcasts | Android |

Can CRISPR gene editing save the Cavendish banana from extinction?

The Cavendish bananathat delicious, yellow tropical fruit currently populating the produce sections of our grocery storesmay not be available for much longer. A fungal disease known as Tropical Race 4 (TR-4) is wreaking havoc on banana plantations across South America, threatening to wipe out the Cavendish for good. TR-4 spreads rapidly and isnt easily controlled with pesticide applications. Thats why scientists are working feverishly to immunize the banana by cutting a segment of DNA out of its genome that makes it susceptible to TR-4.

More precise cancer treatments may be possible by pairing CRISPR with genetic sequencing

Researchers at Johns Hopkins School of Medicine have used CRISPR to rapidly sequence particular genes involved in the development of breast cancer, eliminating the DNA replication process usually required for genome sequencing. The development could enable the selection of customized cancer drugs that treat the disease based on the genetic makeup of individual patients.

Beyond Meat goes on the offensive, blasting critics who claim plant-based burgers are ultra-processed

Plant-based burgers have been a hit with consumers so far, achieving nearly a $1 billion in sales in 2019. This development has made the meat industry nervous, and theyve launched expensive marketing campaigns to dissuade the public from chowing down on the beef-free alternatives. The industrys biggest criticism: plant-based meats are ultra-processed, and presumably less nutritious than traditional burgers.

Beyond Meat, maker of the wildly popular Beyond Burger, is having none of this. The company announced in early March it was going on the offensive to counter the marketing assault on its products, arguing that plant-based foods may actually be healthier than meat in some cases.

Targeting blindness with CRISPR: Doctors attempt first editing of genes inside a human body

Gene editing has yielded dozens of important medical treatments for deadly diseases, including cancers like leukemia and lymphoma. Typically, doctors extract immune cells from a patient, edit their DNA, then infuse them back into the persons body to attack the disease. Scientists have now taken this approach a step further by injecting a virus carrying the instructions to produce CRISPR-Cas9 directly into a patients eye, where it is expected to edit out a mutation involved in Leber congenital amaurosis, a genetic condition that causes blindness. Will this groundbreaking procedure work? Is it safe?

Infographic: What the US public thinks about tinkering with human genetics

As all this genetic engineering work begins reshaping intimate aspects of our lives, scientists and policy makers are eager to find out how consumers feel about the technology. Is the public on board, or do they fear a loss of human control? Both.

A majority of people surveyed by Pew (60%) said genetic engineering should be used to prevent serious diseases and produce organs for people who need them (57%), but they were also concerned about using the technology to enhance human performance. 69 percent, for example, said implanting brain chips to improve memory and information processing would be a step too far.

Kevin M. Folta is a professor in the Horticultural Sciences Department at the University of Florida. Follow Professor Folta on Twitter @kevinfolta

Cameron J. English is the GLPs senior agricultural genetics and special projects editor. BIO. Follow him on Twitter @camjenglish

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Genetic Engineering Co. Says Its COVID-19 Vaccine Could Be Approved By End of the Year – American Greatness

Wednesday, March 11th, 2020

A Texas-based genetic engineering company claims to have created a vaccine to prevent the coronavirus (COVID-19) and is hoping to have the drug approved and available to the public by the end of the year.

John Price, the CEO of Greffex, told Fox News Monday that he was completely confident in his companys new vaccine.

Were confident in the vaccine, the quality of the vaccine completely. The end result will be what the government wants to do in terms of testing, Price said.

The company had previously created a vaccine to combat MERS and that research helped them develop the new vaccine. MERS has a tremendous number of similarities to the coronavirus, Price explained.

The vaccine is still in the testing stage, and if approved, could be available to the public by years end, he said.

When asked whether there was a way to fast track the approval process, Price answered that it would be a policy decision for the government.

Thats always the $100 million question. The earliest that we think would be the end of the year. The latest would be 18 months. But we think that we could depending on the approval process of the government get something in 2020, he said.

Yesterday was the first time I heard people say its a pandemic, Price added. If its truly a pandemic, then you can pretty much do whatever you want. The process is roughly four weeks for the first animal testing and then you go into human trials. And thats the part that will be determined by the government.

National Institute of Allergy and Infectious Diseases Director Dr. Anthony Fauci and his team, meanwhile, are working on a separate vaccine which could take up to 18 months to prove safety and effectiveness. The FDA has granted approval for the National Institutes of Health to begin the first stage of clinical testing in that vaccine.

Media-driven panic about the virus has contributed to a jittery and unnerved stock market in recent days. The Dow Jones Industrial Average plunged 1,500 points in early trading, Monday.

As of Monday morning, there were approximately 600 confirmed cases of COVID-19 in the United States and 22 deaths. There are now 111,362 cases worldwide, according to the John Hopkins tracking map.

By comparison, the CDC estimates that 35.5 million people got sick with seasonal influenza in the United States during the 20182019 season, with an estimated 16.5 million people going to a health care provider for their illness. According to the CDC, there have been 490,600 hospitalizations, and 34,200 deaths from influenza, this season.

Unfortunately, the global death rate for COVID-19 is 3.4 percent, which is much higher than the common flu, according to the World Health Organization.

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Genetic Engineering Co. Says Its COVID-19 Vaccine Could Be Approved By End of the Year - American Greatness

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US agencies launch initiative to boost understanding of GMOs – World Grain

Wednesday, March 11th, 2020

WASHINGTON, DC, US The US Food and Drug Administration, the Environmental Protection Agency and the US Department of Agriculture launched a $7.5 million consumer education initiative focused on highlighting the science behind genetically modified organisms.

The goal of the effort, called Feed Your Mind, is to answer the most common questions consumers have about GMOs, including how they are regulated and whether they are safe and healthy.

Less than a dozen genetically modified crops are grown in the United States, but they often make up an overwhelming majority of the crop grown. More than 90% of soybeans, corn and sugar beets planted in 2018 were genetically modified.

Genetic engineering has created new plants that are resistant to insects and diseases, led to products with improved nutritional profiles, as well as certain produce that dont brown or bruise as easily, said Stephen M. Hahn, MD, commissioner of the FDA.

One educational video from the FDA points out that genetically modified soybeans have healthier oils that may be used to replace oils that contain trans fats. Other materials highlight how reduced bruising and browning may help combat food waste.

Consumers, however, remain uncertain. Concerns that GMOs are unhealthy and harmful are widespread. The number of shoppers avoiding GMOs tripled over the past decade, according to The Hartman Group. Close to half of consumers surveyed last year said they avoid bioengineered ingredients, compared to 15% in 2007.

A study published last year in Nature Human Behavior found more than 90% of participants had some level of opposition to GMO foods. It also found that consumers with the strongest opposition to GMO foods thought they were more knowledgeable about the topic than other participants, despite scoring lower on an actual knowledge test.

While foods from genetically engineered plants have been available to consumers since the early 1990s and are a common part of todays food supply, there are a lot of misconceptions about them, Hahn said. This initiative is intended to help people better understand what these products are and how they are made.

The Feed Your Mind initiative will launch in phases. Materials already released include a new website, fact sheets, infographics and videos. Supplementary science curriculum for high schools, resources for health professionals and additional consumer materials will be released later this year and in 2021.

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How children can learn to balance science and religion – The Conversation UK

Wednesday, March 11th, 2020

It sometimes feels like society is permanently at loggerheads, divided over any number of issues, from genetic engineering and vaccines to euthanasia and religion, and unable to engage in productive exchanges across ideological divides.

Consequently, if education is to develop the next generation, it must nurture children as future citizens with the capacity to have productive conversations across these barriers of opinion and discipline.

We are often faced with big questions. But beyond the eternal questions concerning how life came into being and its purpose, there are more immediate concerns about which there will need to be decisions from citizens and leaders both now and in the future. How should we respond to climate change? Should government be allowed to quarantine people to prevent the spread of disease? Should euthanasia of terminally ill children be allowed?

Responses to questions such as these can be informed by science, as well as by ethics, philosophy and religion. But how can we generate a well reasoned argument using a range of diverse and often contradictory sources? And how can we develop childrens ability to do so, too? Children, after all, are the future.

First, children need to explore what an argument is, and what a good argument looks like within the subject they are studying. Put simply, an argument is a claim or set of claims supported by evidence and reasons, while a good argument is one justified by strong reasons and evidence that are relevant to the claim. But how do these arguments differ when it comes to the study of science and religious education (RE) in school?

The teaching and learning of arguments in science subjects has been extensively researched over the past 20 years. Academic textbooks and practical resources for teaching have been produced to support it.

But while RE curriculum documents often cite the need for students to produce well reasoned arguments, there has been far less research on and fewer resources for the teaching and learning of arguments within the subject.

One distinguishing feature between arguments in different subject areas is what is considered to be an acceptable reason. In the case of arguments in RE, what counts as a reason can be less defined and evidence-based than in the sciences, particularly when the focus may be on providing a safe space for expressing beliefs and respecting diversity, rather than on constructing persuasive arguments.

So what can be done about this and how can we ensure that children studying the two subject areas can better argue with one another? The Oxford Argumentation in Religion and Science (OARS) project brings the expertise of working science and RE teachers together, in collaboration with academic researchers. The project is exploring potential approaches for cross-curricular work across these disciplines, producing resources to support the teaching and learning of argument and reasoning in schools.

Our project team suggests that there are at least three good reasons to engage in cross-curricular teaching of argument and reasoning.

First, the subject groups can learn useful lessons from each other. Science teachers can draw on the skills of RE teachers for whom discussion, debate and dialogue are core features of their curriculum and daily work. RE teachers, on the other hand, could benefit by drawing on the well established resources and structure for teaching scientific arguments. They may also draw upon science teachers expertise when exploring scientific ideas and worldviews in RE.

Second, for the range of issues that might draw on both scientific and religious arguments for example, abortion, end-of-life decisions, evolution cross-curricular teaching could help develop a students capacity to discern the difference between those based on scientific evidence and those based more on faith and belief. It could also further their ability to accept and learn from other worldviews.

Finally, this work could extend across the whole school curriculum and bring greater coherence between school subjects. Learning about arguments in different subjects can make clear what is distinctive about each subject area (for example, highlighting the features of scientific arguments that make them distinctly scientific, as compared to other subjects). It can also highlight what features of arguments are common across specialities, showing how different subjects across the curriculum are related.

There is no single way that this cross-curricular collaboration could be rolled out in schools. Indeed, our participating teachers are innovative in finding approaches that work within the bounds of their busy, and often different, school lives.

In one example, an RE teacher and a science teacher are exploring the same question in their separate subject lessons: Why should we act on climate change? Students are asked to construct arguments using information that they have been learning in each subject, before combining these separate arguments from religion and science to present a convincing and coherent answer that draws on both disciplines.

We do not have all the answers and our work is ongoing. But we are convinced of the importance of learning how to argue and how to engage with others arguments for the sake of better scientific literacy, better religious literacy, and to create better citizens. Ultimately, it is about having productive discussions about what often appear to be unbridgeable divides and unanswerable dilemmas and to bring people together in the process.

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FDA, EPA and USDA launch GMO education initiative – New Food

Wednesday, March 11th, 2020

The initiative aims to educate consumers about GMOs, including their production processes, their health information and other safety-related questions.

The US Food and Drug Administration (FDA), in collaboration with the US Environmental Protection Agency (EPA) and the US Department of Agriculture (USDA), have launched a new initiative to help consumers better understand foods created through genetic engineering, commonly called GMOs or genetically modified organisms.

The initiative, Feed Your Mind, aims to answer the most common questions that consumers have about GMOs, including what GMOs are, how and why they are made, how they are regulated and to address health and safety questions that consumers may have about these products.

While foods from genetically engineered plants have been available to consumers since the early 1990s and are a common part of todays food supply, there are a lot of misconceptions about them, said FDA Commissioner, Stephen M. Hahn, M.D. This initiative is intended to help people better understand what these products are and how they are made. Genetic engineering has created new plants that are resistant to insects and diseases, led to products with improved nutritional profiles, as well as certain produce that dont brown or bruise as easily.

Farmers and ranchers are committed to producing foods in ways that meet or exceed consumer expectations for freshness, nutritional content, safety, sustainability and more. I look forward to partnering with FDA and EPA to ensure that consumers understand the value of tools like genetic engineering in meeting those expectations, said Greg Ibach, Under Secretary for Marketing and Regulatory Programs at USDA.

As EPA celebrates its 50th anniversary, we are proud to partner with FDA and USDA to push agricultural innovation forward so that Americans can continue to enjoy a protected environment and a safe, abundant and affordable food supply, said EPA Office of Chemical Safety and Pollution Prevention Assistant Administrator, Alexandra Dapolito Dunn.

The Feed Your Mind GMO initiative is launching in phases. The current materials released include a new website, as well as a selection of fact sheets, infographics and videos. Additional materials including a supplementary science curriculum for schools, resources for health professionals and additional consumer materials will be released later in 2020 and 2021.

To guide development of the Feed Your Mind initiative, the three government agencies formed a steering committee and several working groups consisting of agency leaders and subject matter experts; sought input from stakeholders through two public meetings; opened a docket to receive public comments; examined the latest science and research related to consumer understanding of genetically engineered foods; and conducted extensive formative research. Funding for Feed Your Mind was provided by Congress in the Consolidated Appropriations Act of 2017 as the Agricultural Biotechnology Education and Outreach Initiative.

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Beyond Meat rolls out frozen breakfast sausage patties, addresses pricing in plant-based meat sector – FoodNavigator-USA.com

Wednesday, March 11th, 2020

While Beyond Meat debuted in the frozen aisle with beef crumbles and chicken strips (the latter have been dropped), the brand took off after launching refrigerated burgers and sausages designed to sit in the [animal] meat case, with sales in its fresh platform growing 275% in 2019 vs 11.8% growth in frozen.

If the refrigerated plant-based meat category is growing significantly faster, however, the frozen aisle is still the largest section in grocery for plant-based meat and still represents a sizeable opportunity, chief growth officer Chuck Muth told FoodNavigator-USA. Plus, our breakfast sausage patty cooks better from frozen.

The patties (MSRP $4.99 for six) contain 11g protein per serving, with 50% less total fat, 35% less saturated fat and sodium, 33% fewer calories, and 35% less sodium than the leading brand of pork sausage patties, with a base of pea protein and brown rice protein.

Asked about pricing in the plant-based meat segment following rival Impossible Foods move to cut prices to foodservice distributors by 15%, Muth said:

Impossible is quite a bit smaller than us; they are just starting to scale and as they are scaling, they are finding efficiencies and, I assume, bringing their price down accordingly. However their frontline pricing is still significantly higher than ours, so they still have a bit to go, and our pricing is more attractive.

He added:One of our goals is to reduce our pricing, so as we are able to develop more production efficiencies and [increase] capacity, and as we engineer products, we are very much focused on bringing our prices down.

Weve made it our stated goal that at least one of our items will be as cheap or cheaper than animal meat within the next four years or so, and thats the long term goal, to be priced competitively, not just with other plant based meats but with animal meats as well.

So is Beyond Meat sustaining or growing sales velocities in high-profile restaurant chains after the initial excitement or marketing budget wears off?

I think the encouraging thing for us is seeing product expansions in existing chains where we have partnerships, because theyre seeing good results coming in, said Muth, citing the example of Carls Jr and Hardees now offering Beyond Sausage breakfast burritos and egg and cheese biscuits as well as burgers.

He also noted that Dunkin which is rolling out Beyond Sausage sandwiches nationwide after a successful trial had attracted new guests and increased check sizes in part because plant-based products are premium items, but also because customers have proved more likely to pair them with higher-priced beverages such as lattes and cold brew. Its bringing in bigger register rings.

While some big names in QSR have not yet introduced plant-based entrees or breakfast options, they are all monitoring the space closely, he said.

Its more a timing issue than anything, plus they also want something thats unique to them since they are not going to be first to market, so they are thinking about what will differentiate them from the competition. But long term if they see their competitors being successful in this space they are going to have to take a very serious look.

Asked if Beyond Meat were in a position to be able to say yes to every account thats interested, or whether supply constraints were holding the company back, Muth said the firm was expanding in-house extrusion capabilities in the near future and adding more co-packers to its network in the US, Canada, Europe and Asia for downstream patty/sausage formation and packaging.

His comments came as CEO Ethan Brown told analysts last month that Beyond Meat began the year with around $700m in gross revenue capacity, with plans to scale to over a billion by the end of the year.

On the ingredient sourcing front, while Beyond Meat has recently expanded its pea protein sourcing capabilities,it is also exploring multiple other plant-based protein sources for sensory reasons (adding new flavors), nutritional reasons (to balance out amino acid profiles), and supply chain reasons (to diversify), said Muth, who noted that the Beyond Sausage uses a small amount of faba bean protein, while Beyond Beef and Beyond Burgers utilize mung bean and rice protein as well as peas.

As for the innovation pipeline, right now, Beyond Meat is focused on beef, poultry, and pork alternatives including plant-based bacon, said Muth. But down the road wed potentially look at other things.

Quizzed about the brands decision to go on the offensivethis year to tackle the narrative that plant-based meats are highly processed and unhealthy, he said:

We believe in the category and the space and were very positive, you wont hear us bad mouth other plant-based products or brands, but there are a lot of false narratives out there about whats in our products, so we think we have an obligation to talk about whats in our foods, so to understand that things like methyl cellulose [which isused in most plant-based meat products] are in many foods, things like ice cream and baked goods.

We want to make sure that consumers are well informed and to remind people that most foods we eat are processed.

Asked whether it was disingenuous to make a virtue of Beyond Meats all-natural non-GMO credentials [which distinguish it from rival Impossible Foods] given its commitment to science-based messaging and consumer education, he said:

Its not about what we believe, its what our consumers, our shoppers, believe, so were not saying theres anything bad about it [genetic engineering in food production].

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UNL team links wild wheat gene to drought tolerance in cultivated wheat – Grand Island Independent

Wednesday, March 11th, 2020

New research from the University of NebraskaLincoln has led to the discovery of a novel gene that improves drought adaptation in wheat a breakthrough that could contribute to increased world food security.

In new research published in Plant Biotechnology Journal, Harkamal Walia, associate professor and Heuermann Chair of Agronomy and Horticulture at Nebraska, and colleagues describe a novel form of a gene obtained from wild wheat that has the potential to improve drought tolerance in cultivated wheat. Introducing this gene into cultivated wheat improved the plant root structure so that it continued to grow in search of water under dry soil conditions.

Wheat is the most widely grown crop in the world and, together with rice, provides more than 50% of the caloric intake of humans globally. Like other crops, wheat is exposed to a wide range of environmental limitations, such as high temperature, disease pressure and drought.

The scavenging nature of wheat root systems during times of drought may have been lost when wild wheats were adopted for agriculture by early humans or as cultivated wheat was bred for improved responsiveness to irrigation and fertilizers during the mid-1900s. This improved responsiveness was key to feeding a booming world population during the 1960s.

As todays producers strive for more crop per drop to feed a world population that is again in the midst of a boom and is expected to grow from about 7.5 billion today to more than 9.6 billion by 2050, it is evident that future crops will need greater drought resilience. The discovery by Walia and his colleagues could represent an important new genetic resource, enabling breeders to recapture this natural survival trait in cultivated wheat. UNL has secured a patent on the discovery via NUtech Ventures, enabling future commercialization of this technology.

The potential impact of the discovery grew substantially when the team found that adding the wild root gene also resulted in plants with larger grains in the absence of drought. Walia and his team were not expecting this, as introducing tolerance to a stress can sometimes result in lost productivity when the stress is absent.

This particular trait may have the opposite effect, which is a benefit in both conditions, Walia said. We are now working to understand the reason behind this surprising finding.

The genetic engineering of wheat plants was performed at Nebraskas Center for Biotechnology.

Walia is one of many researchers worldwide helping to develop a catalog of genes that will contribute to creating more robust plants for the future. Drought response is a complicated trait, Walia said, which involves many genes contributing to survival and productivity when water is limited. He hopes that research in this area will continue to discover new genetic resources that plant breeders and geneticists can use to develop more drought-tolerant crops.

From a genetic improvement perspective, it takes a community to make a crop more adaptive, Walia said. This finding is one piece of a very large puzzle.

The research was spearheaded by doctoral students Dante Placido and Jaspreet Sandhu in the Department of Agronomy and Horticulture. The work was supported by the Institute of Agriculture and Natural Resources and the Robert B. Daugherty Water for Food Global Institute.

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UNL team links wild wheat gene to drought tolerance in cultivated wheat - Grand Island Independent

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Cell Therapy Insights Report, 2018-2028: Markets, Technologies, Ethics, Regulations, Companies & Academic Institutions – Benzinga

Wednesday, March 11th, 2020

Dublin, March 10, 2020 (GLOBE NEWSWIRE) -- The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

The cell-based markets was analyzed for 2018, and projected to 2028. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 309 of these are profiled in part II of the report along with tabulation of 302 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 67 Tables and 25 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

This report contains information on the following:

The report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

Key Topics Covered

Part I: Technologies, Ethics & RegulationsExecutive Summary 1. Introduction to Cell Therapy2. Cell Therapy Technologies3. Stem Cells4. Clinical Applications of Cell Therapy5. Cell Therapy for Cardiovascular Disorders6. Cell Therapy for Cancer7. Cell Therapy for Neurological Disorders8. Ethical, Legal and Political Aspects of Cell therapy9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions10. Markets and Future Prospects for Cell Therapy11. Companies Involved in Cell Therapy12. Academic Institutions13. References

For more information about this report visit https://www.researchandmarkets.com/r/bzimne

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Cell Therapy Insights Report, 2018-2028: Markets, Technologies, Ethics, Regulations, Companies & Academic Institutions - Benzinga

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Team Creates Shape-Changing Material That Pushes Biological Boundaries – University of Texas at Dallas

Wednesday, March 11th, 2020

Study lead author Laura Rivera Tarazona, a biomedical engineering doctoral student, worked with Dr. Taylor Ware (left) and Dr. Zachary Campbell on her research that incorporated plant DNA into yeast to give it light-responsive traits.

Combining the powers of the living and the inanimate, an interdisciplinary team from The University of Texas at Dallas has embedded genetically modified yeast into a synthetic gel to create a novel, shape-changing material designed to grow under specific biochemical or physical conditions.

This is definitely a case where the product is more than the sum of its parts, said Dr. Taylor Ware, assistant professor of bioengineering in the Erik Jonsson School of Engineering and Computer Science and corresponding author of a paper published in January in Science Advances, the American Association for the Advancement of Sciences open-access journal.

The idea to use the reproductive growth of cells to drive shape change within an inanimate container began with an old, reliable standby: bakers yeast, or Saccharomyces cerevisiae.

Yeast was the first eukaryotic organism to have its genome totally sequenced, Ware said. Wonderful tools exist already to modify it genetically. The cells have stiff cell walls, unlike mammalian cells, which make them better for pushing outward on the gel to change its shape.

By genetically modifying the yeast in different ways, the research team created composites that responded to various stimuli.

In proof-of-concept experiments, biomedical engineering doctoral student Laura Rivera Tarazona, lead author of the paper, incorporated plant DNA into yeast to give it light-responsive traits. When the resulting yeast-hydrogel composite was exposed to light, the entire object changed shape as the growing yeast pushed outward on the boundaries of the gel.

The research team also modified the yeast to respond to biochemical stimuli, including amino acids, which are building blocks of proteins.

This combination of animate with inanimate lends itself to interacting with the body in a particularly useful way using cellular mechanisms to drive shape change, Ware said. Given the flexibility of yeast, this composite could be designed to respond to any of countless conditions.

Dr. Zachary Campbell, assistant professor of biological sciences in the School of Natural Sciences and Mathematics and a co-author of the study, said the awesome power of yeast genetics made the project possible.

Weve had the ability to make yeast do amazing biological things for a long time, but its only in the past few years that we have had the ability to create strains where gene activity is precisely controlled by light, Campbell said.

Theres a beauty to taking something thats ordinarily so static and endowing it with this capability to transform into other things.

Dr. Zachary Campbell, assistant professor of biological sciences in the School of Natural Sciences and Mathematics

The researchers believe the shape-changing response has potential applications as a type of reporter both inside and outside the body.

Where I think this research eventually goes is indicating disease states via detection of proteins and other biomolecules, Ware said.

Ware said shape change could also be used to perform mechanical work to open a container or uncover an adhesive, for example.

Our results are in the very early stage, but the fact that were taking a series of molecular events and transducing them into something mechanical is already exciting in itself, Ware said.

Rivera Tarazona uses a microscope as one of the successful projects is displayed on the monitor in the background.

Campbell added that, although the physical transformations in the composite materials are very slow, capitalizing on genetic manipulations to drive minuscule devices could have additional applications, such as releasing drugs from a capsule in response to a precise biological trigger.

Theoretically, you could use these to detect anything you can detect in nature by combining an existing genetic circuit from another cell type with the yeast, he said. This allows access to a dazzling array of physiological cues.

Theres a beauty to taking something thats ordinarily so static and endowing it with this capability to transform into other things.

Other authors of the research included biomedical engineer Hyun Kim PhD19 and Vandita Bhat, a molecular biology doctoral student graduating this spring.

The work was supported by a grant (R01NS100788) from the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, and is partially based on work supported by the National Science Foundation.

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Isaac Asimov, the candy store kid who dreamed up robots – Salon

Wednesday, March 11th, 2020

The year 2020 marks a milestone in the march of robots into popular culture: the 100th anniversary of the birth of science fiction writer Isaac Asimov. Asimov coined the word 'robotics', invented the much-quoted Three Laws governing robot behavior, and passed on many myths and misconceptions that affect the way we feel about robots today.

A compulsive writer and homebodypossibly, an agoraphobicAsimov hated to travel: ironically, for a writer who specialized in fantastic tales often set on distant worlds, he hadn't been in an airplane since being flown home from Hawaii by the US Army after being released from service just before a test blast of the atomic bomb on the Bikini Atoll. (Asimov once grimly observed that this stroke of luck probably saved his life by preventing him from getting leukemia, one of the side effects that afflicted many servicemen who were close to the blast.)

By 1956, Asimov had completed most of the stories that cemented his reputation as the grand master of science fiction, and set the ground rules for a new field of study called "robotics," a word he made up. Researchers like Marvin Minsky of MIT and William Shockley of Bell Labs had been doing pioneering work into Artificial Intelligence and Robotics since the early 1950s, but they were not well-known outside of the scientific and business communities. Asimov, on the other hand, was famous, his books so commercially successful that he quit his job as a tenured chemistry professor at Boston College to write full-time. Asimov's 1950 short story collection, I, Robot, put forward a vision of the robot as humanity's friend and protector, at a time when many humans were wondering if their own species could be trusted not to self-destruct.

Born in January 1920, or possibly October 1919the exact date was uncertain because birth records weren't kept in the little Russian village where he came fromAsimov emigrated to Brooklyn in 1922 with his parents. Making a go of life in America turned out to be tougher than they expected, until his father scraped together enough money to buy a candy store. That decision would have a seismic impact on Isaac's future, and on robotics research and the narratives we tell ourselves about human-robot relationships to this day.

As a kid, Isaac worked long hours in the store where he became interested in two attractions that pulled in customers: a slot machine that frequently needed to be dismantled for repairs; and pulp fiction magazines featuring death rays and alien worlds. Soon after the first rocket launches in the mid-1920s, scientists announced that space travel was feasible, opening the door to exciting tales of adventure in outer space. Atomic energythe source of the death rayswas also coming into public consciousness as a potential "super weapon." But both atomic bombs and space travel were still very much in the realm of fiction; few people actually believed they'd see either breakthrough within their lifetimes.

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The genre of the stories in the pulps wasn't new. Fantastical tales inspired by science and technology went back to the publication of Mary Shelley's Frankenstein in 1818, which speculated about the use of a revolutionary new energy source, electricity, to reanimate life. Jules Verne, H. P. Lovecraft, H. G. Welles, and Edgar Rice Burroughs all wrote novels touching on everything from time travel, to atomic-powered vehicles, to what we now call genetic engineering. But the actual term, "science fiction," wasn't coined by any of them: that distinction goes to Hugo Gernsbeck, editor of the technical journal, Modern Electrics, whose name would eventually be given to the HUGO, the annual award for the best science fiction writing.13

Gernsbeck's interest in the genre started with a field that was still fairly new in his time: electrical engineering. Even in 1911, the nature of electricity was not fully understood, and random electrocutions were not uncommon; electricians weren't just tradesmen, but daredevils, taking their lives in their hands every time they wired a house or lit up a city street.14 Gernsbeck, perhaps gripped by the same restless derring-do as his readers, wasn't satisfied with writing articles about induction coils. In 1911, he penned a short story set in the twenty-third century and serialized it over several issues of Modern Electrics, a decision that must have baffled some of the electricians who made up his subscribers. At first, Gernsbeck called his mash-up of science and fiction "scientifiction," mercifully changing that mouthful to "science fiction." He went on to publish a string of popular magazines, including Science Wonder Stories, Wonder Stories, Science, and Astounding. (Gernsbeck's rich imagination didn't stretch far enough to come up with more original titles.)

Asimov's father stocked Gernsbeck's magazines in the candy store because they sold like hotcakes, but he considered them out-and-out junk. Young Isaac was forbidden to waste time reading about things that didn't exist and never would, like space travel and atomic weapons.

Despite (or possibly because of) his father's objections, Isaac began secretly reading every pulp science fiction magazine that appeared in the store, handling each one so carefully that Asimov Senior never knew they had been opened. Isaac finally managed to convince his father that one of Gernsbeck's magazines, Science Wonder Stories, had educational valueafter all, the word "science" was in the title, wasn't it?15

Isaac sold his first short story when he was still an eighteen-year-old high school student, naively showing up at the offices of Amazing Stories to personally deliver it to the editor, John W. Campbell. Campbell rejected the story (eventually published by a rival Gernsbeck publication, Astounding) but encouraged Isaac to send him more. Over time, Campbell published a slew of stories that established Isaac, while still a university student, as a handsomely paid writer of science fiction.

When you read those early stories today, Asimov's weaknesses as a writer are painfully glaring. With almost no experience of the world outside of his school, the candy store, and his Brooklyn neighborhood and no exposure to contemporary writers of his time like Hemingway or FitzgeraldIsaac fell back on the flat, stereotypical characters and clichd plots of pulp fiction. Isaac did have one big thing going for him, though: a science education.

By the early 1940s, Asimov was a graduate student in chemistry at Columbia University, as well as a member of the many science fiction fan clubs springing up all over Brooklyn whose members' obsession with the minutiae of fantastical worlds would be familiar to any ComicCon fan in a Klingon costume today. Asimov wrote stories that appealed to this newly emerging geeky readership, staying close enough to the boundaries of science to be plausible, while still instinctively understanding how to create wondrous fictional worlds.

The working relationship between Asimov and his editor, Campbell, turned into a highly profitable one for both publisher and author. But as Asimov improved his writing and tackled more complex themes, he ran into a roadblock: Campbell insisted that he would only publish human- centered stories. Aliens could appear as stock villains but humans always had to come out on top. Campbell didn't just believe that people were superior to aliens, but that some peoplewhite Anglo-Saxons were superior to everyone else. Still a relatively young writer and unwilling to walk away from his lucrative gig with Campbell, Asimov looked for ways to work around his editor's prejudices. The answer: write about robots. Asimov's mechanical beings were created by humans, in their own image; as sidekicks, helpers, proxies, and, eventually, replacements. Endowed with what Asimov dubbed "positronic brains," his imaginary robots were even more cleverly constructed than the slot machine in the candy store.

Never a hands-on guy himself, Asimov was nonetheless interested in how mechanisms worked. Whenever the store's one-armed bandit had to be serviced, Isaac would watch the repairman open the machine and expose its secrets. The slot machine helped him imagine the mechanical beings in his stories.

Although Asimov can be credited with kick-starting a generation's love affair with robots, he was far from their inventor. (Even I, Robot borrowed its title from a 1939 comic book of the same name written by a pair of brothers who called themselves Eando Binder, the name eventually bestowed on the beer-swilling, cigar-smoking robot star of the TV show, Futurama.) But in writing his very first robot story, Asimov was both jumping on a new obsession of the 1920s, and mining old, deep myths going back to ancient Jewish tales of the golem, which was a man made of mud and magically brought to life, as well as stories as diverse as Pygmalion, Pinocchio, and engineering wonders like the eighteenth century, chess-playing Mechanical Turk, and other automatons.

Robots have an ancient history and a surprisingly whimsical one. Automatons have been frog marching, spinet playing, and minuet dancing their way out of the human imagination for hundreds, if not thousands, of years, but it wasn't until the machine age of the early twentieth century that robots appeared as thinking, reasoning substitute humans. The word robotCzech for "mechanical worker"wasn't coined in a patent office or on a technical blueprint, but as the title of a fantastical play by Karel Capek, Rossum's Universal Robots, which was first performed in 1920, the reputed year of Isaac Asimov's birth. In adopting robots as his main characters, and the challenges and ethics of human life in a robotic world as one of his central themes, Asimov found his voice as a writer. His robots are more sympathetic and three-dimensional than his human characters. In exploring the dynamics of human-robot partnershipsas Asimov would do particularly well in detective/robot "buddy" stories, such as his 1954 novel Caves of Steel he invented a subgenre within the broader world of science fiction.

Asimov's humanoid robots were governed by the Three Laws of Robotics. More whimsical than scientific, they established ground rules for an imaginary world where humans and mechanical beings coexisted. Eventually, the Three Laws were quoted by researchers in two academic fields that were still unnamed in the 1940s: artificial intelligence and robotics.

First published by Astounding magazine in 1942 as part of Asimov's fourth robot story "Runaround", the Three Laws stated that:

A robot may not injure a human being or, through inaction, allow a human being to come to harm.

A robot must obey the orders given it by human beings except where such orders would conflict with the First Law.

A robot must protect its own existence as long as such protection does not conflict with the First or Second Laws.

According to Asimov's biographer Michael Wilson in Isaac Asimov: A Life of the Grand Master of Science Fiction (New York, Carrol & Graff, 2005), "Asimov was flattered that he had established a set of pseudoscientific laws. Despite the fact that in the early 1940s the science of robotics was a purely fictional thing, he somehow knew that one day they would provide the foundation for a real set of laws."

The Three Laws would continue to appear not only in the world of robot-driven books and filmslike Aliens (1986), where the laws are synopsized by the synthetic human Bishop when trying to reassure the robot-phobic heroine Ellen Ripleybut by some real-world roboticists and AI researchers, who are now considering how to develop a moral code for machines that may one day have to make independent, life-or-death decisions.

Read more from the original source:
Isaac Asimov, the candy store kid who dreamed up robots - Salon

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