I woke up on Saturday to a heartbreaking front-page article in the New York Times about a terminally ill young woman who chooses to freeze her brain. She is drawn into a cottage industry spurred by transhumanist principles that offers to preserve people in liquid nitrogen immediately after death and store their bodies (or at least their heads) in hopes that they can be reanimated or digitally replicated in a technologically advanced future.

Proponents have added a patina of scientific plausibility to this idea by citing the promise of new technologies in neuroscience, particularly recent work in connectomicsa field that maps the connections between neurons. The suggestion is that a detailed map of neural connections could be enough to restore a persons mind, memories, and personality by uploading it into a computer simulation.

Science tells us that a map of connections is not sufficient to simulate, let alone replicate, a nervous system, and that there are enormous barriers to achieving immortality in silico. First, what information is required to replicate a human mind? Second, do current or foreseeable freezing methods preserve the necessary information, and how will this information be recovered? Third, and most confounding to our intuition, would a simulation really be you?

I study a small roundworm, Caenorhabditis elegans, which is by far the best-described animal in all of biology. We know all of its genes and all of its cells (a little over 1,000). We know the identity and complete synaptic connectivity of its 302 neurons, and we have known it for 30 years.

If we could upload or roughly simulate any brain, it should be that of C. elegans. Yet even with the full connectome in hand, a static model of this network of connections lacks most of the information necessary to simulate the mind of the worm. In short, brain activity cannot be inferred from synaptic neuroanatomy.

Synapses are the physical contacts between neurons where a special form of chemoelectric signalingneurotransmissionoccurs, and they come in many varieties. They are complex molecular machines made of thousands of proteins and specialized lipid structures. It is the precise molecular composition of synapses and the membranes they are embedded in that confers their properties. The presence or absence of a synapse, which is all that current connectomics methods tell us, suggests that a possible functional relationship between two neurons exists, but little or nothing about the nature of this relationshipprecisely what you need to know to simulate it.

Additionally, neurons and other cells in the brain are in constant communication through signaling pathways that do not act through synapses. Many of the signals that regulate fundamental behaviors such as eating, sleeping, mood, mating, and social bonding are mediated by chemical cues acting through networks that are invisible to us anatomically. We know that the same set of synaptic connections can function very differently depending on what mix of these signals is present at a given time. These issues highlight an important distinction: the colossally hard problem of simulating any brain as opposed to the stupendously more difficult task of replicating a particular brain, which is required for the promised personal immortality of uploading.

The features of your neurons (and other cells) and synapses that make you you are not generic. The vast array of subtle chemical modifications, states of gene regulation, and subcellular distributions of molecular complexes are all part of the dynamic flux of a living brain. These things are not details that average out in a large nervous system; rather, they are the very things that engrams (the physical constituents of memories) are made of.

While it might be theoretically possible to preserve these features in dead tissue, that certainly is not happening now. The technology to do so, let alone the ability to read this information back out of such a specimen, does not yet exist even in principle. It is this purposeful conflation of what is theoretically conceivable with what is ever practically possible that exploits peoples vulnerability.

Finally, would an upload really be you? This is unanswerable, but we can dip our toes in. Whatever our subjective sense of self is, lets assume it arises from the operation of the physical matter of the brain. We could also tentatively conclude that such awareness is substrate-neutral: if brains can be conscious, a computer program that does everything a brain does should be conscious, too. If one is also willing to imagine arbitrarily complex technology, then we can also think about simulating a brain down to the synaptic or molecular or (why not?) atomic or quantum level.

But what is this replica? Is it subjectively you or is it a new, separate being? The idea that you can be conscious in two places at the same time defies our intuition. Parsimony suggests that replication will result in two different conscious entities. Simulation, if it were to occur, would result in a new person who is like you but whose conscious experience you dont have access to.

That means that any suggestion that you can come back to life is simply snake oil. Transhumanists have responses to these issues. In my experience, they consist of alternating demands that we trust our intuition about nonexistent technology (uploading could work) but deny our intuition about consciousness (it would not be me).

No one who has experienced the disbelief of losing a loved one can help but sympathize with someone who pays $80,000 to freeze their brain. But reanimation or simulation is an abjectly false hope that is beyond the promise of technology and is certainly impossible with the frozen, dead tissue offered by the cryonics industry. Those who profit from this hope deserve our anger and contempt.

Michael Hendricks is a neuroscientist and assistant professor of biology at McGill University.

Read more:
The False Science of Cryonics - MIT Technology Review

Recommendation and review posted by Bethany Smith

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Cryonics Technology Market Application Analysis(Animal husbandry, Fishery science) and Forecast 2020-2029 - TheLoop21

Recommendation and review posted by Bethany Smith

While testing for the coronavirus has been slow and scarce across the U.S., one Bay Area laboratory has created what they say is a faster, more accurate COVID-19 test, which is already attracting long lines of patients hoping to find out if they are infected.

The virus mutates every day, every week

We were able totweak some parameters and make sure that we give accurate results, but with afaster turnaround time, said Dr. Connie Chao-Shern, a molecularbiologist who helped create the new test with her team at Avellino Labs inMenlo Park. The virus mutates every day, every week, and this [test] actuallyis following the virus in all the mutations.

Dr. Chao-Shern, who serves as the chief lab officer forAvellino Labs, says the new test provides quicker and more accurate results thanother tests, including those created by the CDC.

The quicker wedetect the patients who are positive, the better they we can stop the spread,said Dr. Chao-Shern. The longerwe wait, the more chance of spreading the virus to somebody else.

FDA Authorizes Bay Area Lab to Begin Testing for Coronavirus

After receiving emergency authorization from the FDA, AvellinoLabs received its first batch of tests to process Monday morning. The NBC Bay Area Investigate Unit was theonly media outlet at the headquarters when those initial 33 samples arrived forprocessing.

Avellinos new coronavirus test can be processed within fourto seven hours, which would allow patients to be notified of their resultswithin one day.

Lab Focused on Eye Diseases Pivots to Coronavirus

COVID-19 tests are a new direction for the lab, which, upuntil this week, only focused on making tests to diagnose eye diseases.

We suspended testing of those, said John Hong, who heads the legal department and human resources at Avellino Labs. We've pivoted completely to testing for coronavirus, explaining that because both tests require genetic testing, the technology is similar.

We quickly realized, almost immediately, that this is something that may be able to benefit everyone

The company began developing the COVID-19 test in Januaryafter employees at their Shanghai laboratory complained about a lack of tests inChina. Soon after, Avellino Labs created their own test in California, whichthey then shipped abroad to their coworkers.

We were thinking of that just being a resource internally, Hong said. Then, we quickly realized, almost immediately, that this is something that may be able to benefit everyone.

Firefighters Trade Hoses for Nose Swabs

As the coronavirus began to spread across California, HaywardFire Chief Garrett Contreras began searching for test supplies that his firefightersand paramedics could utilize to care for communities in the Bay Area. Itwasnt long before the Chief connected with executives at Avellino Labs.

One of our vendors...had called me and said that ChiefContreras was looking for the test, said Scott Korney Chief Operating Officerat Avellino. [Contreras] was thrilled that we had availability of testing.

The partnership allowed Hayward to open its first drive-upand walk-up testing site Monday at Hayward Fire Station #7, 28270 HuntwoodAvenue.

Suppression, through isolation after testing, or SIT, as we call it, is an approach that has proven to be most effective in countries on the leading edge of this pandemic, said Chief Garrett Contreras.

His firefighters armed with swabs, thermometers, andtesting kits are now on the front line of the pandemic. In opening the testing site, the department declaredthat sick people, first responders, and health-care workers with recentexposures to the novel coronavirus can be tested for infection.

Coronavirus Tests Remain Scarce

About 60 percent of the coronavirus testing done in the U.S. took place in just about the last week, according to the White House.

"We now have 370,000 tests that have been done," said Dr. Deborah Birx, coordinator of the White House coronavirus task force, who made those comments during a press conference on Tuesday. "The majority of those over 220,000 in the last eight days, which, those of you who have been tracking the South Korea numbers, put us equivalent to what they did in eight weeks that we did in eight days."

Tests, however, remain scarce in the Bay Area and across the country, so only about 10% of those in line at the Hayward site have been tested -- the sick and elderly continue to be prioritized. Yet, to understand the impact of the virus, some argue far more people need to be tested and even retested.

Testing once - it lets you know whether you're infected at that point in time, said John Hong. But without the second test, without the third, fourth test in the future, you can't you can't be assured of anything except for when you got tested that first time.

We will stop this pandemic

Lab employees spent roughly a month, working throughout the night, to develop the test.

It was exhilarating, said Dr. Chao-Shern. We cheered...we, at that time, actually, we hugged!

Currently, the lab can process about 350 tests per day. Over the next two months, however, Avellino plans to triple its staffing in order to increase its processing capacity to 7,000 tests per day.

The number of tests we could actually run will be determined by how quickly we can bring in partners to help us rapidly expand, said Eric Bernabei, Chief Sales and Marketing Officer for Avellino Labs. We have a plan now that we could get close to 200,000 tests per month, he said.

The demand for tests, like the virus, is spreading. Withevery batch of used tests dropped off, new tests are going out. Avellino Labshas been inundated with calls from all over the world, asking if the lab canproduce tests for their communities.

We will prevail, said Dr. Chao-Shern. We will stop the spread and we will stop this pandemic.

Read the original:
Bay Area Lab Aims to Distribute Millions of Coronavirus Tests After Creating Their Own - NBC Bay Area

Recommendation and review posted by Bethany Smith

Sixteen years ago, in the very early days of medical genetic testing, I received a positive result for one of the BRCA mutations, which are correlated with a vastly increased risk of breast and ovarian cancers. I began interviewing doctors and genetic counsellors, in order to decide whether to have preventive surgeriesand, if so, which ones. I quickly understood that the science of cancer prevention had a tunnel-vision problem: I was counselled to undergo surgeries that would lower my risk of cancer but vastly increase other health risks. I joked that cancer prevention would be the more successful the sooner I died of something else.

What made my predicament more difficult was that the medical advice I got wasnt wrong. Preventive surgeries were the best options available for me to live a longer life. But the advice was dispensed without regard for, or at times even acknowledgment of, the damage that these surgeries would do to my health. I agonized over the risks, the uncertainties, and the irreversible nature of my decisions. In the end, I chose to have the surgeries, albeit not in the order and not nearly on the time frame that was recommended back then. I am now older than my mother or her aunt, who also had the mutation, ever lived to be. But I was profoundly changed by the surgeries. My life now is not fully continuous with my life before the interventions. I like some of the changes and regret the others. I also know that, if I had not been mindful of the unintended consequences of my decisions, my health and my quality of life today would be the worse for it.

In the United States, we are in a similarly terrible predicament now, as a society, as I was as a person with a body. The measures we are taking to save ourselves from a global pandemic of the novel coronavirus are changing us in fundamental, possibly irreparable ways. By instituting lockdowns and deploying a variety of emergency powers across the country, we are destroying our economy, our social fabric, and our political system. We will never be the same. Whether we change for both the better and the worse, as opposed to the solely catastrophic, will depend on how mindful we remain of the damage we are doing as we attempt to save ourselves from the pandemic.

The economy has already taken the biggest sudden hit in memory. Hundreds of thousands of people have lost their jobs, and many more will become unemployed in the coming weeks. Inequalities in wealth, opportunity, and access to health care have become even more glaring than they were just a couple of weeks ago.

The social fabric is being torn in unprecedented ways, owing to school closings, a widespread shift to working from home, social distancing, sheltering in place. Whereas we used to share dozens of experiences a day with friends, acquaintances, and strangersfrom riding the subway to working in an office, standing in line at lunch, going to a concert, eating at a restaurant, chatting to an Uber drivermany of us have been reduced to sharing only isolation and the fear of chance encounters, if either of those can be said to be shared.

Our political system, frayed as it was, is under extraordinary stress. The Supreme Court has delayed cases. The Justice Department is seeking extreme powers. The Trump Administration is using the crisis as an opportunity to push through a more extreme version of its agenda. The President now lies to the nation daily ,not only on Twitter but also on live television, during briefings that he has turned into versions of his rallies. The election campaign is in a state of suspended animation. The borders are effectively closed. At the federal level, there is a real possibility that the coronavirus will paralyze the work of Congress, leaving the White House without check. At the local level, quarantine measures either have stopped or will stop all town councils, school boards, and community meetings. Local news media, an endangered species before the crisis, may have been dealt a final, fatal blow by the coronavirus.

In the past week, several high-profile writers have raised the possibility that emergency measures taken against the pandemic are too drastic. The founding director of the Yale-Griffin Prevention Research Center, David Katz, writing in the Times, has argued that the threat of the pandemic is overestimated. The papers opinion columnist Thomas Friedman echoed this argument. But the problem is that, after the Trump Administration wasted the time that was available to prepare for the pandemics spread, by instituting widespread testing and creating additional hospital facilities, todays Draconian measures are both necessary and probably insufficient. As the President careens toward lifting preventive measures, in order to help the economy at the expense of human lives, we will increasingly find ourselves in the absurd position of demanding that the government drastically curtail all manner of freedom.

The low bar set by the incompetent, self-obsessed, lying President makes any halfway-competent public servant sound brilliant. In New York, Governor Andrew Cuomos popularity seems to have spiked simply because he is acting and speaking rationally. In this time of crisis, with little concrete information available, I need Cuomos measured bullying, Rebecca Fishbein wrote on Jezebel, in a piece called Help, I Think Im in Love with Andrew Cuomo???

We tend to throw the word authoritarian around a lot, usually to mean anything that we perceive as less than democratic. But one of the technical definitions of authoritarianism is a regime in which one person or a single group of people make all governmental decisions, denying the public participation in political life. (This distinguishes authoritarianism from totalitarianism, where people are forced to participate all the time.) When we virtuously retreat to our homes, deserting public space and delegating all authority to one man armed with emergency powers, we are creating a society as close to the textbook definition of authoritarianism as has ever actually existed.

So what do we do now that so much economic, social, and political damage has already been done? We have to start talking about the damage, and thinking about tomorrow. We have to recognize that what we are doing to avoid being killed by a virus is also killing us as a society. We have to make it a priority to restore the social fabric.

One tool that will be necessary for this project is an antibody test, which will tell people whether theyve already had the coronavirus and recovered from it. (Antibody tests for the novel coronavirus exist, but the tests that are currently availableor, for most people, not availableare tests that check for the presence of the virus. People who have already recovered will test negative.) It is currently assumed that people who have recovered from the infection might have immunity to it, at least for a period of time. Provided theyve been quarantined for enough time, these could be the people who can volunteer at hospitals, with food and service delivery, at schools. A large enough number of people with immunity, mobilized intelligently, could not only help prevent new infections but also help remedy some of the inequalities that the crisis has exacerbated.

See more here:
In the Midst of the Coronavirus Crisis, We Must Start Envisioning the Future Now - The New Yorker

Recommendation and review posted by Bethany Smith

The story of Britains most successful Covid-19 app yes, there is such a thing started with twins.

Tim Spector, a professor of genetic epidemiology at Kings College London, has been probing and observing identical twins for a long time and in Just 1992 he launched the registry TwinsUK to aid research on the subject. His interest was investigating how people with identical genes could grow up to develop radically different traits and illnesses. Take nutrition: identical twins might eat the same food, but react very differently maybe one will develop an allergy while the other wont; or their blood sugar level would draw dissimilar curves, with different repercussions on each siblings metabolism.

After decades spent researching twins at Kings College, in 2018 Spector teamed up with technologists Jonathan Wolf and George Hadjigeorgiou to launch a startup called ZOE. In partnership with research institutions and universities in various countries, the company started carrying out its own studies about twins and nutrition. Its first study, gauged how over 1,000 people (mostly twins) responded to various kinds of food. A similar study, with a larger sample, is currently underway.

ZOE's business plan was to combine the data gathered through these studies with machine learning, and eventually develop a consumer product able to predict every persons individual response to what they eat. According to a company spokesperson, the product would be like [genetic testing service] 23andme, but for eating.

Then the coronavirus outbreak started. As the virus ripped through the globe, on March 20 Spector resolved to turn to TwinsUKs volunteers to track whether they were showing any coronavirus symptoms, and how those symptoms progressed over time. He roped in ZOE, too which was tasked with creating an app through which twins could report their symptoms. The initial plan was to have all volunteers tested for Covid-19, but the spokesperson says that it has since proved impossible to get hold of the testing kits.

While designing the app, the ZOE team thought that its utility could go well beyond keeping tabs on twins. We realised that this was something that we could make even more useful overall by getting everybody in the public to participate, ZOE president George Hadjigeorgiou says. [This can] help understand how the virus is progressing [in the UK] and also to help understand whether a particular area is at a high risk.

Hadjigeorgiou says the app was developed and inaugurated with a sense of extreme urgency. We just put together a SWAT team and we worked through the weekend to get it live. The result? The Covid Symptom Tracker. The app went live on Tuesday, both for Android and iOS phones. By Wednesday, Britons had downloaded it over a million times.

The app is extremely simple. Users are asked to create an account, and provide information, including age, sex at birth, height, weight, postcode of residence, pre-existing health conditions, and habits such as smoking. Then, theyre asked daily to report any symptom that could be associated with Covid-19. The list currently includes fever, persistent cough, fatigue, shortness of breath, loss of smell or taste, hoarseness, chest pain, abdominal pain, diarrhoea, drowsiness and confusion, and lack of appetite. Hadjigeorgiou says that as more symptoms get flagged by the international research community as potentially relevant to Covid-19 detection, they will be added to the app.

The data gathered this way will be shared with researchers at Kings College and at the Guys and St Thomas' Hospitals, to get a better sense of how the pandemic is spreading through the UK, and which symptoms should be regarded as telltale signs of a novel coronavirus infection as opposed to the common cold or the seasonal flu. Hadjigeorgiou says that the company has also been trying to get in touch with the UK government and the NHS in order to share their data with them, although whether that would result in a partnership was unclear as of Wednesday. The company spokesperson says that ZOE is talking with NHSX, the NHSs digital unit.

According to Hadjigeorgiou, information about how symptoms are being reported in various parts of the UK might be even made public. What we will try to do [Wednesday night] or [Thursday] at the latest is publish the first set of data that we collected, he says. That will be geographical data but whether we'll be able to publish down to the postcode level maybe not yet. But the intent is to share all this information in a way that's useful for people.

Yet, a banner published on the apps website this morning reads: We are not sharing any analysis or data from the app except with researchers at King's College London and the NHS. As soon as they have analysis they are happy with, they will share it. It is unclear whether that will affect the timetable of the publication of the location data.

The company is planning to launch the app in the US in the near future. Hadjigeorgiou says that partner institutions there might include high-profile universities and hospitals with which ZOE which opened a Boston office last year had already been working for its nutrition studies.

The app privacy policy mentions that anonymised data might be shared with US-based organisations including Harvard University, Stanford University, Massachusetts General Hospital, Tufts University, and Berkeley University. The spokesperson also says that the company has been in touch with American hospitals. One paragraph in the apps privacy policy underlines that the data will be handled in accordance with GDPR, but that they might not enjoy similar levels of protection when transferred to countries like the US.

Beyond that, Hadjigeorgiou cannot bring himself to make prediction about Covid Symptom Trackers future. I don't think anybody will know the answer of how the world will look like in two weeks, he says. It would be ideal if this app can be part of the NHSs efforts because that's the only way we can really deliver something that will have everything it needs to be integrated with what public health systems are doing.

As of Thursday morning, the app ranked first on Google Play Store's list of most downloaded health apps in the UK. That is despite the fact that, currently, searching for Covid on Google Play returns no result apart from an app run by the World Health Organisation.

Gian Volpicelli is WIRED's politics editor. He tweets from @Gmvolpi

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Original post:
The inside story of the UK's biggest coronavirus symptom tracker app - Wired.co.uk

Recommendation and review posted by Bethany Smith

Scientists around the globe are testing existing drugs to treat Covid-19 patients. Some of these drugs are already approved for treating other diseases, such as Malaria, HIV and arthritis.

Novel Coronavirus (SARS-Cov-2) has disrupted the entire globe, every country is being affected with this coronavirus disease (Covid-19). Since there is no confirmed treatment, scientists are testing existing drugs to treat this disease. Following are brief about some drugs which are shortlisted to treat Covid-19 patients.

Chloroquine has been used to treat patients with malaria for nearly a century. It was discovered in 1934 by an Italian scientist. The active ingredient in the malaria drug Resochin. It is on the World Health Organizations List of Essential Medicines, the safest and most effective medicines needed in a health system.

Chloroquine is a synthetic version of quinine. Quinine is a natural compound found in the bark of cinchona trees. Chloroquine works by essentially slowing down virus entry to cells, which can slow replication rate. It is already known to be safe for humans, as well as cost effective.

Hydroxychloroquine is closely related to chloroquine. It is regarded as less toxic metabolite of the malaria drug.

Hydroxychloroquine is used to treat certain autoimmune diseases, like lupus and rheumatoid arthritis. It has been on the market since the 1950s. It works by disrupting communications between cells in the immune system. Scientists hope it might help mitigate cytokine storms.

Kaletra is a combination of two antiviral drugs, lopinavir and ritonavir, which are used to combat HIV. It is widely available and clinical trials are underway.

These two drugs complement each other. Lopinavir prevents viral enzymes from cutting up important proteins, that are key to HIVs reproduction. Whereas, Ritonavir helps boost lopinavirs concentrations in cells. But a recent study suggested it has no benefits for Covid-19 severe cases.

Remdesivir was developed to fight Ebola virus but failed to prove effective. In case of SARS and MERS, Remdesivir showed some positive results. Both of these diseases are caused by coronaviruses. So that, it is considered to be effective against this novel coronavirus (SARS-Cov-2).

It is unclear yet that how Remdesivir works, but a new study shows that it appears to block RNA replication during the reproductive cycle of a coronavirus.

Losartan is a hypertension drug that reduces blood pressure by preventing a hormone called angiotensin from binding to receptors on blood vessels.

Losartan works by blocking a receptor, or doorway into cells that the chemical called angiotensin II uses to enter the cells and raise blood pressure. SARS-CoV-2 binds to the angiotensin-converting enzyme 2 (ACE2) receptor.

Favipiravir or Avigan is a flu drug developed in Japan. It has shown promising results in treating mild to moderate cases of Covid-19. This antiviral drug has been used in Japan to treat influenza. It was approved last month as an experimental to treat Covid-19 infections.

Actemra is an immunosuppressant that is used to quiet Cytokine storms. Actemra or Tocilizumab is approved to treat rheumatoid arthritis and juvenile rheumatoid arthritis. It blocks a cell receptor that binds interleukin 6 (IL-6). Interlukin 6 is a cytokine or a type of protein released by the immune system, that can trigger dangerous inflammatory cascades.

See the rest here:
7 powerful drugs being explored to treat Covid-19 patients - - Technology Times Pakistan

Recommendation and review posted by Bethany Smith

During this process the cells not only shed any memories of their previous identities, but they revert to a younger state. They accomplish this transformation by wiping their DNA clean of the molecular tags that not only differentiate, say, a skin cell from a heart muscle cell, but of other tags that accumulate as a cell ages.

Recently researchers have begun to wonder whether exposing the adult cells to Yamanaka proteins for days rather than weeks could trigger this youthful reversion without inducing full-on pluripotency. In fact, researchers at the Salk Institute for Biological Studies found in 2016 that briefly expressing the four Yamanaka factors in mice with a form of premature aging extended the animals life span by about 20%. But it wasnt clear whether this approach would work in humans.

Sarkar and Sebastiano wondered whether old human cells would respond in a similar fashion, and whether the response would be limited to just a few cell types or generalizable for many tissues. They devised a way to use genetic material called messenger RNA to temporarily express six reprogramming factors the four Yamanaka factors plus two additional proteins in human skin and blood vessel cells. Messenger RNA rapidly degrades in cells, allowing the researchers to tightly control the duration of the signal.

The researchers then compared the gene-expression patterns of treated cells and control cells, both obtained from elderly adults, with those of untreated cells from younger people. They found that cells from elderly people exhibited signs of aging reversal after just four days of exposure to the reprogramming factors. Whereas untreated elderly cells expressed higher levels of genes associated with known aging pathways, treated elderly cells more closely resembled younger cells in their patterns of gene expression.

When the researchers studied the patterns of aging-associated chemical tags called methyl groups, which serve as an indicator of a cells chronological age, they found that the treated cells appeared to be about 1 to 3 years younger on average than untreated cells from elderly people, with peaks of 3 years (in skin cells) and 7 years (in cells that line blood vessels).

Next they compared several hallmarks of aging including how cells sense nutrients, metabolize compounds to create energy and dispose of cellular trash among cells from young people, treated cells from old people and untreated cells from old people.

We saw a dramatic rejuvenation across all hallmarks but one in all the cell types tested, Sebastiano said. But our last and most important experiment was done on muscle stem cells. Although they are naturally endowed with the ability to self-renew, this capacity wanes with age. We wondered, Can we also rejuvenate stem cells and have a long-term effect?

When the researchers transplanted old mouse muscle stem cells that had been treated back into elderly mice, the animals regained the muscle strength of younger mice, they found.

Finally, the researchers isolated cells from the cartilage of people with and without osteoarthritis. They found that the temporary exposure of the osteoarthritic cells to the reprogramming factors reduced the secretion of inflammatory molecules and improved the cells ability to divide and function.

The researchers are now optimizing the panel of reprogramming proteins needed to rejuvenate human cells and are exploring the possibility of treating cells or tissues without removing them from the body.

Although much more work needs to be done, we are hopeful that we may one day have the opportunity to reboot entire tissues, Sebastiano said. But first we want to make sure that this is rigorously tested in the lab and found to be safe.

Other Stanford co-authors are former postdoctoral scholar Marco Quarta, PhD; postdoctoral scholar Shravani Mukherjee, PhD; graduate student Alex Colville; research assistants Patrick Paine, Linda Doan and Christopher Tran; Constance Chu, MD, professor of orthopaedic surgery; Stanley Qi, PhD, assistant professor of bioengineering and of chemical and systems biology; and Nidhi Bhutani, PhD, associate professor of orthopaedic surgery.

Researchers from the Veterans Affairs Palo Alto Health Care System, the University of California-Los Angeles and the Molecular Medicine Research Institute in Sunnyvale, California, also contributed to the study.

The research was supported by the National Institutes of Health (grants R01 AR070865, R01 AR070864, P01 AG036695, R01 AG23806, R01 AG057433 and R01 AG047820), the Glenn Foundation for Medical Research, the American Federation for Aging Research and the Department of Veterans Affairs.

Sarkar, Quarta and Sebastiano are co-founders of the startup Turn Biotechnologies, a company that is applying the technology described in the paper to treat aging-associated conditions. Rando is a member of the scientific advisory board.

Excerpt from:
Old human cells rejuvenated with stem cell technology - Stanford Medical Center Report

Recommendation and review posted by Bethany Smith

Global Cell-Based Immunotherapy Market By Application (Prostate Cancer, Breast Cancer, Skin Cancer, Ovarian Cancer, Brain Tumor, Lung Cancer, Other), End- User (Hospitals, Ambulatory Surgical Centers, Specialized Cancer Institutes), Geography (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Industry Trends and Forecast to 2026

Market Analysis:

Global cell-based immunotherapy market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Improvement in healthcare infrastructure and rising healthcare expenditure are the factor for the market growth. Few of the major competitors currently working in the global cell-based immunotherapy market are AbbVie Inc., Genentech USA, Inc., Amgen Inc, AstraZeneca, Bayer AG, Boehringer Ingelheim International GmbH., Eli Lilly and Company, GlaxoSmithKline plc., Novartis AG, Pfizer Inc, Bio-Rad Laboratories, Inc., F. Hoffmann-La Roche Ltd, Takara Bio Inc., Bausch Health, Lonza Group AG, Precision Biosciences., Marker Therapeutics, Inc., Kiadis Pharma, Lyell Immunopharma, Inc., among others.

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Key Developments in the Market:

Competitive Analysis:

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

Market Definition:Global Cell-Based Immunotherapy Market

Cell-based immunotherapy is a potential future-oriented cancer treatment approach. It is evolving quickly as an alternative to traditional cancer treatment based on chemotherapy. Stem cells are used for the diagnosis of different types of cancer in cell-based immunotherapy. These cells have the ability to create extra placental or embryonic cells to cure cancer. This therapy is widely used in application such as breast cancer, skin cancer, ovarian cancer, lung cancer and prostate cancer.

Market Drivers

Market Restraints

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Segmentation: Global Cell-Based Immunotherapy Market

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By End-User

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Global Cell-Based Immunotherapy Market Future Growth Analysis, Business Demand and Opportunities to 2027 | AbbVie Inc., Genentech USA, Inc., Amgen...

Recommendation and review posted by Bethany Smith

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Know the Growth Opportunities in Emerging Markets

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market to Witness Growth Acceleration During 2017 2025 - Daily Science

Recommendation and review posted by Bethany Smith

The electronic newsletter I Love My Freedom on a regular basis blasts out e-mails hawking ostentatious rip-offs as well as serpent oil, such as a dementia-reversing miracle therapy, a diabetes destroyer material as well as a life-saving cancer cells treatment that a Nazi drug store allegedly established at Hitlers command. Peppered in between these messages funded by third-party hucksters are main advertisements from Donald Trumps governmental reelection campaign.

For months, participants of the head of states internal circle consisting of Donald Trump Jr., Senate Majority Leader Mitch McConnell (R-Ky), previous House Speaker Newt Gingrich as well as also Trump himself have actually been releasing require contributions with the newsletter, which usually heads out 5 or even more times each day. Many receivers were most likely unintentionally subscribed; I Love My Freedom, the team that runs the eponymous newsletter, has actually obtained an expanding checklist of Americans call info with a concealed e-mail collecting system including an internet of pro-Trump Facebook web pages.

Right- wing political leaders, companies as well as media electrical outlets have a background of dealing with unethical entities behind the scenes to earn money as well as press their programs. The Trump campaigns service with I Love My Freedom is no exemption.

I Love My Freedom (e-mail information multiplied by HuffPost).

By layout, its e-mails seem theyre sent out straight from Trump as well as his allies, though theyre in fact dispersed by means of [emailprotected] At all-time low, they birth please notes noting they were spent for by either the National Republican Senatorial Committee, its House equivalent, the McConnell Senate Committee, or the Trump Make America Great Again Committee (which is collectively run by the Republican National Committee as well as Trumps reelection campaign).

Renting out accessibility to collected e-mail listings is a typical as well as very profitable method worked out by traditional as well as liberal teams alike, commonly for political fundraising objectives. But points obtain morally dirty when e-mail representatives do not veterinarian their enrollers which can lead to e-mail receivers being flooded with ripoffs as well as scams, like the counterfeit cancer cells treatment. In this instance, nonetheless, the genuine inquiry is whether Trumps group troubled to veterinarian I Love My Freedom.

The campaign did not react to HuffPosts ask for remark.

I Love My Freedom, which makes up the newsletter as well as a conservative blog site called Trending Politics, belongs to Making Web LLC, a rare advertising company thats signed up in Minnesota to a 51- year-old male called Allan G. Ferretti.

Through the newsletter alone, I Love My Freedom has actually enhanced a list of aggressive rip-offs consisting of some that misleadingly link Trump, in spite of its negotiations with his campaign. Hours prior to dispersing a fundraising e-mail from the McConnell Senate Committee this month, I Love My Freedom discharged off a funded message advertising a breakthrough stem cell therapy which it baselessly suggested Trump is getting.

This has got Liberals jumping out of their seats, the e-mail checks out. President Trump is in perfect health how is it that hes so seemingly immune to old age? Well in recent years, billionaires like President Trump have increasingly turned to the power of Stem Cells.

Titled Trump Health Bombshell, the e-mail web links to a rambling item pitch that promotes $67 containers of stem cell tablets as the Holy Grail of aging backwards. These tablets make cells inside your body become physically younger, it asserts, without supplying a shred of clinical proof. I Love My Freedom has likewise spammed its customers with enrollers get-rich-quick plans consisting of a secret IRS loophole, as well as has actually routed them to video clips recommending elders ought to exchange their recommended medicines for tricksters supplements also advising that they can pass away if they do not.

Recent newsletter versions have actually circulated actual phony information, as well, installing advertisements camouflaged as write-ups that connect to internet sites stealthily copying genuine media electrical outlets.

I Love My Freedom e-newsletters have actually included misleading advertisements resulting in phony information websites copying genuine electrical outlets..

One such advertisement includes the message Royal Family Mourns As Tragedy Is Confirmed, along with a picture of Meghan Markle, the Duchess ofSussex Clicking on it causes a web site imitating U.S.A. Today that goes crazy regarding a skin care line, which it incorrectly asserts Markle released. Another, birthing the heading [BREAKING NEWS] Prayers Go Out to Oprah Winfrey, web links to a fraudulent information website marketing a Brain Booster supplement, which it brazenly states is in charge of Winfreys occupation success.

In enhancement to Trump, his oldest kid, McConnell as well as Gingrich, I Love My Freedom has actually likewise sent fundraising e-mails in behalf of previous White House press assistant Sarah Huckabee Sanders, House Minority Leader Kevin McCarthy (R-Calif), House Republican Whip Steve Scalise (R-La), previous United Nations Ambassador Nikki Haley,Rep Elise Stefanik (R-N.Y.),Rep David Joyce (R-Ohio), previous White House Deputy Chief of Staff Karl Rove as well as National Republican Congressional Committee Chairman TomEmmer

All were spent for by the NRCC, NRSC, McConnell Senate Committee or the Trump Make America Great AgainCommittee Only the NRCC reacted to an ask for remark.

We rented this list to prospect new donors. We do our best to vet each vendor, but similar to renting a car, it is impossible to know or control what every other renter does with a list they too are renting, spokesperson Chris Pack informed HuffPost.

We will not be using this vendor going forward.

I Love My Freedom constructed its cash-cow e-mail realm by spending virtually $2 million right into Facebook advertisements, which attract individuals in with clickbait surveys or pledges of free MAGA equipment, as well as result in web pages advising them to send their e-mail addresses. This immediately indications them as much as obtain the newsletter, in addition to its numerous funded messages.

The team likewise earns money by marketing Trump- themed product such as Make Liberals Cry Again hats, as well as organizing third-party advertisements on Trending Politics which was seen a million times last month alone, according to electronic analytics device ComparableWeb

I Love My Freedom did not accept HuffPosts ask for a meeting.

I Love My Freedom makes use of clickbait Facebook advertisements to gather individualss e-mails, after that subscribes them to its newsletter.

Ferretti released ilovemyfreedom.org in addition to the Facebook web page Trump for President Fan Club (currently President Donald Trump Fan Club, which has 1.6 million fans) in the summertime of 2015, as reporter April Glaser reported last loss in a write-up regarding I Love My Freedoms viral development on Facebook.

Its among greater than a lots preferred web pages the team runs to run its countless Facebook advertisements, consisting of Donald Trump Is My President, Donald Trump 2020 Voters, President Trump Has My Vote, Donald Trumps Americans, Team Trump Fan Club, The President Trump Fan Club, We Need Trump 2020 as well as President Trumps Patriot Army.

These web pages create a stream of hyper-partisan memes as well as Trending Politics post to their target market of millions. Boosted by Facebooks formula, the advertisements which largely target elders are in some cases seen thousands of countless times each.

This looks like an operation thats got a very highly engaged audience that would be a prime target for a lot of conservative politicians to try to raise grassroots money from, stated Michael Beckel, research study supervisor at the political reform team Issue One.

When you run a pro-Trump Facebook team with greater than a million participants, that can make your [email] checklist an appealing possession.

Trumps campaign has actually currently gathered a citizen e-mail checklist thats so big it leases it out to outdoors celebrations. So why would certainly his group wish to fundraise with I Love My Freedoms checklist particularly provided the teams historical connections to grifters as well as scam artist?

Its type of striking that the Trump campaign is making a financial investment in [I Love My Freedoms] checklist, kept in mindBeckel But a t completion of the day, he stated, much more e-mails still means more potential voters or donors for them.

Calling all HuffPost superfans!

Sign up for subscription to come to be a starting participant as well as aid form HuffPosts following phase

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Donald Trump Is Using An Insanely Sketchy Newsletter To Find Campaign Donors - The Union Journal

Recommendation and review posted by Bethany Smith

Texas resident Hunter Howard shares journey ater testing positive for coronavirus.

Texas resident Hunter Howard shares journey ater testing positive for coronavirus.

Texas resident Hunter Howard shares journey ater testing positive for coronavirus.

Texas resident Hunter Howard shares journey ater testing positive for coronavirus.

Texas resident shares personal journey of testing positive for coronavirus

It has been 14 days since Hunter Howard first suspected he had contracted COVID-19.

Ten days since it was confirmed.

Now Howard, a 50-year-old Dallas resident, is recovering and out of quarantine. Yet his breathing is labored at times, his sense of taste is altered and intestinal problems ensue.

Coronavirus: How Harris County's stay-at-home order compares to other counties, states

I went for a walk yesterday and after about five blocks, I had to sit down. I couldnt breathe, Howard said Wednesday.

Howard, who has no underlying health conditions, believes he caught the virus via an infected passenger seated behind him on a flight home from Aspen, Colo.,on March 9. Upon his return to Dallas, it was life as normal.

Until March 12.

I had a slight fever and a dry cough. It is a light tickle in the back of your throat. There was no phlegm or mucus, said Howard, who is the president of Dallas-based Hormone Therapeutics.

By the next day, he began experiencing head and body aches, fatigue and night sweats. His fever was also rising, but he had no way to measure his temperature due to a shortage of thermometersamid the coronavirus pandemic.

Houston: Rapper Scarface says he tested positive for coronavirus

He relied on Gatorade to help replenish his electrolytes. I was incredibly thirsty. I was surprised by how much Gatorade I needed, Howard said.

After calling his doctor, Howard decided not to seek additional medical attention.

My physician told me there were only 42 testing units allocated to Dallas at that time and unless I needed a ventilator, I should stay home and rest, said Howard. He also said if I felt like I needed help to come in.

Two days later, his symptoms worsened considerably.

My fever was much higher and my breathing was really labored. I felt like my lungs were two paper bags. I could hear them crackling every time I would take a breath, said Howard.

He also experienced intense body pains, including discomfort in his back and chest.

I felt like I had a strap around my chest. The pain felt like it was coming out of the bottom lobes of my lungs and rib cage. It was uncomfortable to lie down, he said.

But the most alarming thing was the fear of the unknown.

The symptoms were worsening and I did not know how much worse it might get. I was being told to self-quarantine unless I needed ICU breathing support, he said. When do you make a decision to go in? At what point do you need to take better care of yourself?

After taking physician-advised doses of 1600mg of Tylenol, his situation improved. My fever broke and I have been feeling better ever since, said Howard.

On March 16, four days after he originally suspected he may be afflicted, Howard received a physician referral to get tested for COVID-19 at Baylor Scott & White Medical Center in Greenville.

The test requires a swab from a hard-to-reach area of the throat. To get there, technicians insert a nasopharyngeal swab essentially a giant Q-tip deep into the patients nose.

It stings the nostrils but only takes around 10 seconds, Howard said. A day later, it was confirmed that Howard had the coronavirus.

Howard was released from quarantine this week, but complications from the virus remain. His sense of smell and taste buds are still slightly off, and he has ongoing nausea and intestinal problems.

Still, he believes the worst is behind him. Now, hes dishing out advice to others.

Most of us will get the coronavirus. Safeguard your immune system, eat well and rest, Howard said.

We need to all protect the elderly and those with immunosuppression or lung and breathing issues by following social guidelines, and with the lack of beds and ventilators and medication, I believe we need shelter-in-place to slow down the transmission of this virus.

@marcydeluna

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Texas resident shares personal journey of testing positive for coronavirus - Chron

Recommendation and review posted by Bethany Smith

After teasing positive top-line results last November, a deeper dive into Merck & Co. and Bayers heart failure drug vericiguat helped kick off this years scientific meeting of the American College of Cardiology, presented virtually as a late-breaking trial.

The data was highly anticipated, especially since the two Big Pharma partners decided to continue with clinical testingand enroll over 5,000 high-risk participants worldwideafter the novel compound missed its primary endpoints in two previous phase 2 studies.

The phase 3 VICTORIA trial saw a 10% drop in a composite rate of cardiovascular deaths and related hospitalizations, among patients with recently worsening heart failure and reduced ejection fraction. Compared to placebo, the treatment difference with vericiguat started to become apparent after three months.

With a median follow-up period of 10.8 months, patients showed significant reductions in hospitalizations when looked at specifically, however the reduction in cardiovascular-related deaths alone was not statistically significant.

The study, simultaneously published in the New England Journal of Medicine, said the overall difference between the drug and placebo translated to an absolute risk reduction of 4.2 events per 100 patient-yearsor, that it would take about 24 to 28 people treated with vericiguat for one year to prevent one cardiovascular-related death or hospitalization.

For a group of patients with this form of high-risk heart failure, where other heart failure drugs have rarely been studied, vericiguat provides a significant novel addition to usual treatment, said the studys lead author and presenter, Paul Armstrong, a distinguished university professor of medicine at the University of Albertas Canadian VIGOUR Centre.

This is a sick population that has a significant unmet need, Armstrong added. Of the 6.5 million adults in the U.S. suffering from heart failure, many have decompensating conditions leading to repeat hospital visits, and few options for slowing the progression of the disease.

RELATED:Bayer, Merck's vericiguat hits endpoint in heart failure phase 3

Vericiguat is a novel guanylate cyclase stimulator, designed to boost a cell signaling pathway that helps relax blood vessels and increase blood flow, as well as improve heart muscle function.

Its two phase 2 studies enrolled chronic heart failure patients with reduced ejection fraction and preserved ejection fraction, respectively. Vericiguat didnt meet eithers primary endpoints measuring the hormone biomarker N-terminal pro B-type natriuretic peptide, or NT-proBNP, which is released by the heart in response to pressure changes and associated with worsening heart failure.

Still, Bayer and Merck felt there were clear dose responses when looking at the studies subgroups individually, with vericiguats effect sizes on par with clinical trials of other heart failure drugs, according to Mercks vice president and head of global clinical cardiovascular development, Joerg Koglin. This, as well as the drugs safety data, pushed the two companies to forge ahead with the larger trial.

Of course now, seeing the phase 3 results, we felt that it was the right decision, Koglin said in an interview with FierceBiotech. But Bayer and Merck were very closely aligned on this...I wouldnt put it into big gamble category.

This is not just another chronic heart failure study, he said. It is differentiated because it really studied a clinical problem that has not been studied before in other big heart failure trials. It didnt look at broad chronic heart failureit specifically focused on what we believed was a huge unmet clinical need.

Vericiguats safety profile included similar rates of serious adverse events compared to placebo, as well as no adverse effects related to electrolytes or kidney function. That gave the study the unique chance to enroll decompensating patients with higher levels of renal impairment at baseline compared to other drugs, Koglin said.

Now the companies are currently discussing the VICTORIA data with regulators, and expect to finalize plans for follow-up studies over the next six months.

This study is not only answering questionsdoes it work, is it safe, and were we able to enroll a unique patient populationbut the study also generates a number of additional hypotheses, he said, which includes vericiguats relationship with NT-proBNP.

Patients with lower levels had shown greater treatment effect sizes, but those in the highest quartile at the start of the study trended more toward placebo. That will be interesting for us to study, to figure out what it means and why, and how it should or should not inform the use of the drug.

Meanwhile, the ACCs flagship meeting, presented together with the World Congress of Cardiology, will continue virtually this year after being forced to cancel its in-person plans at Chicagos McCormick Place due to the spreading coronavirus pandemic. The large annual confabs of the American Association for Cancer Research and the American Society for Clinical Oncology, among several other medical meetings, have also postponed or gone online.

As a physician-scientist, if you work for three-and-a-half to four years on a program like this, its a little bit of a bummer that youre not able to present the results face-to-face in front of 15,000 cardiologists, Koglin said. But it is what it isand Im glad that we can still get the data out. And being able to do so with a simultaneous publication in the New England Journal I think will do the trick.

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ACC: Bayer and Merck's vericiguat leads off virtual meeting with positive phase 3 heart failure data - FierceBiotech

Recommendation and review posted by Bethany Smith

Say youre feeling depressed, distressed, helpless or anxious. Whom should you turn to for professional help? A cognitive behavioral therapist, whod challenge your dysfunctional thoughts? An old-fashioned Freudian analyst, who might have you lie on a couch, spending months, maybe years, and thousands of dollars delving into your unconscious responses to the ways your parents raised you? But what if all you need is a much less expensive social worker or even a life coach?

These questions have fueled a fierce debate among researchers and clinical practitioners. The argument isnt whether talk therapy is helpful. Hundreds of clinical trials have shown that various mainstream forms of psychotherapy can help treat many mental afflictions, including depression, anxiety, post-traumatic stress, obsessive-compulsive disorder and eating disorders. Indeed, most Americans prefer talk therapy to medication, and talk therapy alone may work just as well and in some cases last longer than medication for some mental health problems. The argument is more about how and why these treatments work, and, accordingly, which therapeutic approaches are most likely to achieve the best results at the lowest cost.

On one side are those who say choosing the right approach is essential, implying that some strategies are clearly better than others. The other side argues that any good therapist will do, because factors common to most psychotherapies especially a strong bond between patient and therapist are whats most important. In between these two camps, but just as involved in the debate, are agnostics who argue that its too soon to choose sides we still just dont know enough about how therapy works.

There are high stakes in this dispute. This isnt just about a bunch of academics fighting each other, says Bruce Wampold, a psychologist now retired from the University of WisconsinMadison. There are tremendous implications for how mental health care is delivered and managed. More and more mental health patients are being treated solely with medication prescribed by general practitioners, with many talk therapists voicing concern that insurance firms and health plans are threatening their profession in their efforts to cut costs. And even when patients are given access to talk therapy, insurers tend to push for the cheapest, most time-limited strategies, which are often not in the interest of patients, Wampold says.

Antidepressants have replaced psychotherapy as the main treatment for depression in the United States. Use of psychotherapy continues to decline.

People have been trying to talk other people into feeling better for more than 3,000 years. Ancient Greeks counseled patients suffering from addictions and psychoses. For nearly 2,500 years, Buddhists have handed down a canon of advice to people struggling with day-to-day emotional suffering. But it was only at the end of the nineteenth century that the Austrian neurologist Sigmund Freud popularized the talking cure.

These days, more than 130,000 licensed US psychiatrists and psychologists provide what is often a blend of Freudian-ish psychodynamic therapy (focused on childhood experiences) and cognitive behavioral therapy (focused on challenging negative thought patterns). But you can also receive some version of talk therapy from a social worker, counselor or marriage and family therapist or conceivably even from a life coach, mentor, hypnotist, neurofeedback practitioner or shaman (see sidebar below).

In 1936, the eminent US psychologist Saul Rosenzweig proposed that a therapists preferred method was all but irrelevant. Instead, he wrote, factors common to most therapies will determine the outcome more strongly than any of the therapys specific features.

Given a therapist who has an effective personality and who consistently adheres in his treatment to a system of concepts which he has mastered and which is in one significant way or another adapted to the problems of the sick personality, then it is of comparatively little consequence what particular method that therapist uses, he wrote.

Rosenzweig began his iconic paper with a passage from Lewis Carrolls Alices Adventures in Wonderland in which a dodo bird, judging contestants in a race, declares that all have won and all must have prizes. He thus set the stage for the long-running debate over what has since been known as the dodo bird verdict.

All must have prizes, the dodo told Alice after a race in Wonderland. Are all psychotherapy methods also winners? An influential twentieth century scholar proposed the idea.

CREDIT: BRYAN SATALINO

In subsequent years, researchers identified several potential common factors, ranging from the therapists office environment to the placebo effect, which has a strong role in both talk therapy and medication. Yet at the top of the list of factors is the relationship, whats called the therapeutic alliance, between patient and therapist.

The reasoning is that this positive emotional bond, made up of trust, respect, affection and high expectations for improvement, inspires each member of the pair to keep showing up and doing the hard work that therapy demands. Specifically, researchers have found, a strong alliance will help the therapist and patient reach consensus on their goals, as well as on what sort of tasks such as free association, Socratic questioning, or homework will help achieve them.

Sixty years after Rosenzweig put his stake in the ground, Wampold and colleagues reviewed more than 200 scientific studies comparing the effectiveness of mainstream therapies, mostly variants of psychodynamic and cognitive-therapy tactics used by well-trained practitioners. They found only minimal differences between outcomes, concluding that bona fide treatments are roughly equivalent. (That conclusion may not extend to outlier therapies such as shamanism, Wampold and other researchers warn, which havent been sufficiently evaluated.)

The bottom line, according to Wampold, is: You cant have psychotherapy without a relationship. Every patient knows that. And every psychotherapist knows it.

The bond between therapist and patient has a bigger impact on the success of psychotherapy than the type of therapy, studies find. Other factors include everything that happens outside the therapists office, including other events in the patients life and society as a whole.

In recent decades, researchers have published scores of papers emphasizing the importance of the bond between patient and therapist. In 2018, a task force for the American Psychological Association concluded: The psychotherapy relationship makes substantial and consistent contributions to outcome independent of the type of treatment.

In 2019, however, psychologist Pim Cuijpers, a leading agnostic, weighed in with a paper in the Annual Review of Clinical Psychology arguing that were still far from knowing how talk therapy works.

CREDIT: RODHO / SHUTTERSTOCK

Forty-five million Americans more than 18 percent of the nations adult population are currently dealing with a mental illness, according to the nonprofit Mental Health America. Perhaps never before have they had so many options for treatment. Psychologist John Norcross at the University of Scranton in Pennsylvania has counted more than 500 different types of psychotherapies. The diversity is also impressive. Websites advertise such arcanely titled brands as Multi-Faceted Intuitive Wellness, Vedic Astrology and Transpersonal Counseling and Personal Totem Pole Process.

In 2018, a pair of worried researchers published a list of therapies practiced by social workers billing themselves as specialists in such alleged treatments as angel card readings, cuddle party facilitations, ancestral healing and bilateral stimulation. In Amsterdam, research psychologist Pim Cuijpers is keeping his own list of offbeat treatments for anxiety and depression, both individualized and therapist-led, including studies on the impact of Chinese calligraphy practice, ingesting saffron, chewing gum and participating in karaoke. Theres even an entire book on the psychotherapeutic value of constricting ones anus 100 times a day.

Few of the outlier treatments have been studied in depth, so while theres little evidence that any of them work, theres also no clear proof that they dont. Still, for years, mainstream psychotherapists and researchers have warned that theyre part of a dangerous trend.

Of course, some critics have just as vehemently attacked Freudian psychoanalysis, which only a few decades ago was considered the gold standard. In 1975, Nobel Prizewinning biologist Peter Medawar famously dismissed psychoanalytic theory as the most stupendous intellectual confidence trick of the twentieth century.

As the debate rages on as to how psychotherapy works, new therapies are sure to keep emerging. Buyers beware.

Katherine Ellison

Earlier studies have been inconclusive, wrote Cuijpers and two colleagues at Vrije University Amsterdam. They cited flaws including researcher bias in favor of particular therapies and an excess of papers that show an association between a treatment and an outcome without proving which facet of the treatment caused the outcome. Whats more, they suggested, some studies purporting to compare different therapy types tip the scales by comparing the researchers favorite with another form of therapy designed to fail in other words not a tested, bona fide strategy.

Solving the mystery once and for all will require much more work, they wrote, adding: It is as if we have been in a pilot phase of research for five decades.

In a 2007 review, Yale psychologist and child psychiatry researcher Alan Kazdin voiced a similar complaint, urging researchers to focus their studies on the ways that the patient-therapist relationship may be helpful rather than simply determining that it helps. How does one get from my therapist and I are bonding to my marriage, anxiety, and tics are better? Kazdin asked in his paper. This is a leap with the intervening steps unspecified or untested, at least to my knowledge. Reached by Knowable, Kazdin said that after 13 years, he still sees this gap in the research.

In recent years, some professional organizations have vouched for some psychotherapies over others, based on a preponderance of evidence that certain methods lead to good outcomes for specific diagnoses. The treatments most often touted as having strong research support have been versions of cognitive behavioral therapy (CBT), which one organization, the Society of Clinical Psychology, recommends for everything from adult attention-deficit hyperactivity disorder to obsessive-compulsive disorder to irritable bowel syndrome.

Critics note, however, that CBTs clear rules and comparatively brief time frames have made it easier to study experimentally than more complex, open-ended therapies, thus yielding more net evidence that it gets results without proving that it is better or worse than any other therapy. Indeed, in recent years, some experts have challenged CBTs reputation as the gold standard, with research suggesting that longer-term, psychoanalytic therapy can be more effective in some cases.

Wampold argues that a greater diversity of options will allow therapists and patients to find the approach that works best for them. Unless theres convincing evidence that one treatment is superior, he says, you should allow therapists to give the treatment they can deliver most effectively and patients should have the right to choose the treatments they prefer.

Patient preferences do make a difference. In a 2018 meta-analysis of 53 studies involving more than 16,000 patients, researchers called such preferences crucial to outcomes. The authors advised that psychotherapists should educate patients about different types of talk therapy and elicit their preferences as to the therapists strategies.

Any resolution to this long-running conflict may ultimately come from the lab, and its so-far elusive promise of delivering indisputable biological evidence of the most effective treatments.

Some researchers are now using functional magnetic resonance imaging scans to track subtle changes in certain brain regions during and after psychotherapy. One day, perhaps, therapists will be able to post before-and-after scans of patients brains, highlighting the improvements, much as plastic surgeons do today. But not yet. The studies so far have mainly focused on laying the groundwork by determining which areas of the brain are affected by therapy, says University of Pittsburgh psychiatrist Jay Fournier. Researchers want to pinpoint how the brain changes in each patient as a result of different treatments and what sorts of changes best predict long-term recovery. They also hope to gain a more nuanced understanding of which characteristics in a patient might predict response to different sorts of treatments.

Analyzing results of 22 studies that scanned patients brains before and after undergoing therapy, researchers found some consistent changes in brain activity. Blue highlights indicate brain regions where activity decreased after therapy, including the cingulate cortex (rostral ACC) and insula, which are involved in emotional processing and anxiety. IFG refers to the inferior frontal gyrus, which is involved in language processing.

CREDIT: L. MARWOOD ET AL / NEUROSCIENCE & BIOBEHAVIORAL REVIEWS 2018

Others searching for concrete markers of a patients progress have been measuring neurochemical changes during therapy. We dont just ask the depressed patient if hes feeling better, says Sigal Zilcha-Mano, a clinical psychologist and expert on the patient-therapist bond at the University of Haifa in Israel. She and her team also measure levels of the stress hormone cortisol, the bonding hormone oxytocin, body temperatures, and the synchrony of speech and movement between patient and therapist.

These measurements, which capture changes many patients and therapists arent aware of in the moment, may eventually present a detailed portrait of what can be among the most intense of human relationships. One day, Zilcha-Mano says, they may also enable more precise, individualized, tailored treatments for each patient. Its a matter of traits and states, she explains. Patients may come to therapy with the trait of being more or less able to trust and form relationships. But depending on the skill of the therapist, they may enter a state of being more open to such a bond. The real-time portrait of chemical changes and movements could someday illuminate this process, particularly in the case of oxytocin, considered to be an important biological marker of trust.

In whatever way it might happen, breaking through the current impasse over what makes psychotherapy effective could help practitioners do a better job, Cuijpers and his colleagues argue. Psychotherapeutic treatments for depression havent improved in decades, they note.

They also hope that more cogent explanations for how talk therapy works could help revive the approach, which other critics say has long suffered from a reputation of lacking scientific credibility.

Understanding how therapies work, write Cuijpers and his colleagues, may make it possible to develop treatments that focus on the core processes and are, therefore, more effective and efficient, and more acceptable to patients.

Skepticism about how psychotherapy works was a problem long before Freud came on the scene. Its at least as old as the charismatic German physician Franz Anton Mesmer, born in 1734. His practice of mesmerism relied on claims that he could manipulate an invisible bodily fluid through animal magnetism. Mesmer established a strong rapport with his patients with nervous disorders, many of whom ended up feeling they had been cured.

But he alienated other doctors, and in 1784, while Mesmer was practicing in Paris, King Louis XVI appointed an international commission of scientists and physicians, including Benjamin Franklin, to investigate his methods. They concluded that he couldnt support his claims of invisible fluids or animal magnetism, ruining his reputation.

Mesmers treatment worked at least for some but not his explanation of how it did so. Psychotherapys quest for legitimacy continues.

Excerpt from:
Psychotherapy on the couch - Knowable Magazine

Recommendation and review posted by Bethany Smith

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Theres a stranger at the door.

At every home around the world from Milan to Modestoas well as from Santa Cruz to Watsonville. It is a cousin to a more familiar presence in our lives, the persistent low-grade angst endemic to contemporary life. But this stranger is exponentially more unsettling and ominous. No one knows the toll it will demand.

That stranger is not so much the COVID-19 virus now threatening the worlds health and economy as it is the dread and anxiety that attends it. Fear of the virus has reached every neighborhood, if not every household, in the world.

How do we take the measure of this frightening new mutation of social anxiety? Most people are already negotiating with the stranger. Denying it, like a Florida spring breaker, is not the responsible option. Facing it, adapting to it, even listening to it is likely to lead to a better outcome for health and peace of mind.

Still, there is an emerging consensus on how to deal with pandemic anxiety that reflects a ritual familiar to anyone who has traveled on a commercial flight. In case of turbulence, when the oxygen masks drop, weve been told hundreds of times, secure your mask first. Then help others with theirs.

To survive the crisis will likely require a renewed sense of community and helping others. But it makes sense that to be an effective helper, everyone will need to come to some kind of self-assessment on their mental and emotional health. Spreading panic or denial will help no one and will probably lead to more suffering. Before engaging with the world, at least in an effective and helpful manner, all of us will have to come to an understanding with the stranger at the door.

First off, advises Santa Cruz physician Dawn Motyka, its crucial to understand the distinction between the viral infection itself and the anxiety of avoiding it. Motyka says she has seen patients who are convinced that they have contracted the illnessand that they may be developing pneumoniabecause they are experiencing the inability to draw a deep breath.

This I feel like I cant get a full breath of air thing is a very common symptom of anxiety, Motyka says. I tell people to walk up and down the stairs a couple of times. If you can manage it, you dont have pneumonia. If you have some kind of pulmonary thing, any amount of exertion will just wipe you out.

Motyka defines anxiety as a state of persistent heightened physiological arousal. Anxiety releases adrenaline, the bodys go-to hormone in fight-or-flight situations of immediate threat. The human psyche is equipped to deal with environmental threats, but not so much a constant state of uncertainty and dread. The metaphor Motyka likes is that of a car revving its engine but not necessarily going anywhere.

Our society is so overstimulating that so many people get trapped with the accelerator slightly pressed at all times, she says.

Anxiety, she insists, is not solely a psychological stateit has physiological dimensions in the body. Its a physiological state that can be generated by a thought, and often is, she says. But it can also be generated by a pattern of physical stimulation in the environment, like a cellphone alert going off all the time.

Especially in an environment where shelter in place and social distancing have become the new normal, managing anxiety is always a question of balance. Your body, as well as your mind, needs down timediversions such as reading, listening to music, or gardening.

I always think of Gone With the Wind, Motyka says. Scarlett OHara says, I just cant think about this right now. Ill think about it tomorrow. Thats good advice sometimes. Budget the amount of time you spend catastrophizing.

Leigh Anne Jasheway is a wellness and stress-management expert, and a stand-up comedian. Jasheway has published more than two dozen books, and has keynoted events across the country with the message that a healthy sense of humor is one of the keys to a sane and balanced life. While emphasizing that there is nothing inherently funny about COVID-19, Jasheway says cultivating humor is a valuable coping mechanism.

The reason we have a sense of humor is for arousal relief, she says, which is the release of negative emotional states, as well as for social bonding. Rather than saying just change your attitudetheres a little bit of privilege in that kind of languageits more like changing the prism through which you look at things.

Spirituality is another realm that has lessons of facing dread and intense fears and their effects on mental health. Much of what Buddhist meditation has taught, for example, has now been embraced in the medical world, most prominently, the benefits of focusing on breathing as a means of managing stress and anxiety.

Catherine Toldi is a Zen priest and teacher at the Santa Cruz Zen Center. One of her studentsa single parent working multiple jobs and living with stress pre-pandemictold her recently about the meditation practice shes been teaching, This is exactly what weve been practicing for all these years. Here it is, the ultimate practice conversation. In a way, its almost a relief to face a real fear instead of the fears that I make up in my head.

Zen practice, Toldi says, is a way to take a step to the side to watch how the brain works. If youre a person who, over the years, has painstakingly been looking at your mind, you can take that step back and say, Hmm, how do I want to think about this right now? Rather than just being like a fish on a hook that immediately goes wherever your mind is telling you to go. Were not under the illusion that were in control of this thing going on. But ultimately, we are the ones who choose what radio station were going to listen to in our brain.

Toldi also points to the Zen paradox that working on ones self is the same as loving others. Meditation, she says, is not about my awakeningthis is not about me going off on some mountaintop somewhere. Its about you. What do you need?

The evolution of the pandemic might mean that once people are more secure or certain in their own situation, then they might focus on another impulse: to care for the community.

In Santa Cruz County, there are enormous and immediate needs in the nonprofit sector. Many nonprofits, whether they are arts-oriented or health and human services, typically hold their annual fundraisers in the spring. This year, those events have uniformly been cancelled, abruptly cutting off lifeblood financing.

You really have this triple whammy, says Karen Delaney, the executive director of the Santa Cruz Volunteer Center. Everybody has had to cancel their fundraisers while trying to cope with both elevated risk and elevated need.

Suzanne Willis of Second Harvest Food Bank says her organization is receiving about 10 times the call volume for their services from a year ago, at the same time that many of their volunteers are retirees and in the demographic most vulnerable to the virus.

Second Harvest is converting many of their farmers-market distribution points to a more grab-and-go style, which means an even greater demand for volunteers to bag food for pickup.

The most important thing to hear in Santa Cruz County, says Willis, is that the food bank is here, and we have food. We have a pipeline coming in and were not going to run out.

But Second Harvest needs volunteers to work in either of two shifts daily, working mild physical labor in a warehouse with safety protocols in place.

The situation is similar at Grey Bears, which delivers food to seniors and other clients at 150 sites around the county. Gov. Gavin Newsoms stay-at-home orders last week reduced the number of 65-and-over volunteers able to work at Grey Bears.

Were all kind of moving through this blindfolded, says Tim Brattan of Grey Bears, who calculates that theyve lost about half of their volunteer force. (Those who) are receiving our services, I would urge them to stick with us. Were committed to this. Were going to keep providing this essential service.

Most nonprofits are in critical need of both volunteer energy and financial donations. Susan True, the CEO of the Community Foundation Santa Cruz County, says donors are beginning to step up.

Were seeing an incredible desire to act, says True, who has established a quick response fund at the foundation. Donors to the fund include many of the dependable names the foundation has depended on in the past, as well as many new people looking to give. The nature of the philanthropy suggests that people are only beginning to focus on the community.

When you think about the [2019] wildfires in Sonoma County, most of the donations in that situation came within the first week, says True. But this is a really different situation where the effects are multiplying so quickly that we anticipate people understanding the depth of crisis much later, when they actually see it. We dont have that visibility yet.

The Volunteer Center is gearing up for a bigger push for raising funds for local nonprofits by re-imagining their annual Human Race fundraiser. The race, now in its 40th year, has historically brought together the countys nonprofits for a May walkathon. This years event has been converted to an online fundraising effort.

Theres probably not going to be an actual walkathon on May 9, says Delaney, though we hope there will be some sort of celebration, depending on what happens.

Instead, the Human Race is now a six-week GoFundMe-style fund-raising campaign, which kicks off this week, to fill the hole created by the cancellation of the various spring fundraisers around the county.

Not every nonprofit knows how to do online fundraising, Delaney says. But the way the web-based Human Race (humanracesc.org) will go is that any person can pick their favorite charity, create their own fundraising page and raise money for the causes they care about.

Charitable donation is often a function of habit. But this year, Delaney says, a new kind of thinking and new kind of action is required.

Its not an event, she says. Its a campaign. In the past, weve asked people to gather some money from friends, show up at the race, and walk. Now were asking people, as youre sheltering in place, to connect the way youre connecting otherwise, online or over the phone, and spend a little time stepping up for the community.

Still, she says the Human Race will commit to one sign of normal times: There will still be T-shirts.

The Volunteer Center of Santa Cruz Countys Human Race will be fundraising online March 25-May 9. Go to humanracesc.org to donate or begin fundraising for your favorite cause.

For continuing in-depth coverage of the new coronavirus and its effects locally, visit goodtimes.sc/category/santa-cruz-news/coronavirus.

To learn about action you can take now, whether youre seeking assistance or want to find ways of supporting the community, visit goodtimes.sc/santa-cruz-coronavirus-resources.

Wallace Baine has been an arts writer, film critic, columnist and editor in Santa Cruz for more than 25 years. He is the author of A Light in the Midst of Darkness, a cultural history of the independent bookseller Bookshop Santa Cruz, as well as the book Rhymes with Vain: Belabored Humor and Attempted Profundity, and the story collection The Last Temptation of Lincoln. He is a staff writer for Good Times, Metro Silicon Valley and San Benito/South Valley magazine.

Read more here:
Giving Back to the Santa Cruz Community Amid Coronavirus - Good Times Weekly

Recommendation and review posted by Bethany Smith

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Alicia Morgans: Hi, I am so excited to have here with me today, Dr. Declan Murphy and Dr. Michael Hofman, two professors from the Peter MacCallum Cancer Center in Melbourne, Australia, also with the University of Melbourne there in Australia. I am so excited to have you here to talk with me today about the ProPSMA trial and the results that are being presented at EAU 2020 as well as being published in the Lancet this year.

So Michael and Declan, can you tell me a little bit about why this trial, why now, what are the issues that we have as clinicians trying to understand and stage patients with high risk localized disease and why was this work so important?

Declan Murphy: Well, thank you Alicia and I'm good day from Melbourne. It's very nice to be connecting with you all today. The reason we did this study was, first of all, we know that patients with high-risk localized prostate cancer are at higher risk of biochemical occurrence or biochemical persistence following treatment of the primary, whether that's surgery or radiotherapy. And we all know that one of the reasons why patients have a higher rate of biochemical recurrence is that standard conventional imaging fails to identify the true state of disease in these patients.

We know that many of these patients will have lymph node disease or even distant disease that's not apparent on conventional imaging using CT bone scan and even MRI scan. So that's the background to the patient population that we studied in this study. And here in Australia and in a number of other countries around the world, we have had really quite a lot of encouraging experience using novel imaging, PSMA PET/CT to be precise over the past five or six years.

And we already know from retrospective or community experience actually in Australia that PSMA PET/CT appears to have better performance characteristics in staging these higher-risk patients. So with that in mind, we designed this study and before we go into the detail of that, I'll hand over to Michael who leads our PSMA PET/CT program here at Peter MacCallum Cancer Center to just explain a little about PSMA PET/CT.

Michael Hofman: Thank you. So PSMA is prostate-specific membrane antigen. This is a cell surface glycoprotein overexpressed in prostate cancer. And to do the scan, we injected intravenously a small radiolabeled peptide that binds to this receptor. It's then taken up into the prostate cancer cells and we put the patient on the PET scanner, a positron emission tomography scanner. And this enables us to image the whole body and see where that radiation has gone to.

And it's combined at the same time with a CT on the PET/CT scanner so that we can see the anatomic background. And this allows us to localize both the primary prostate cancer and any distant metastatic disease in a way that bone scan and CT have never really been able to do before. It's a very sensitive test. We can detect deposits down to around three millimeters in size. And with the CT we can localize very precisely where that abnormality is.

Alicia Morgans: Wonderful. So as we think about this, particularly from the United States where we don't have routine availability of things like PSMA PET/CTs. I always think about how hard it might be to replace our conventional imaging strategy. So our CTs and our bone scans with something new. There's going to have to be a bar that we meet that shows that a new imaging strategy is at least as good, if not better, than the strategy that we already have.

And it seems that this study was ... really one of its goals, was really to help to define that or to meet that bar, at least to help us understand if it could meet that bar. But I'd love to hear about the study and how did you design it? What were its goals and objectives and what were the arms in the trial?

Michael Hofman: Yeah so this is a multicenter, randomized study conducted at 10 large centers around Australia. These were centers that had nuclear medicine facilities that could do the gallium PSMA PET scans, but also multidisciplinary uro-oncology teams with both urologic surgeons and radiation oncologists treating these patients. And what we did is the primary aim was to see whether the PSMA PET/CT was more accurate than the combined findings of conventional staging. That being bone scan and CT and we randomized patients to receive either the PSMA PET or the current standard of care imaging, bone scan or CT.

Then there was a selective crossover because we did not want to deny patients with a normal CT and bone scan access to this new technology. So patients with fewer than three unequivocal distant metastases crossed over and had the other imaging arm. And then importantly, we followed patients up carefully for six months and at six months patients selectively had repeat imaging and then the principal investigators at each site used all the information up until that six-month time point, including the repeat imaging to define the ground truth.

That may have been histopathologic evaluation through a biopsy of bone metastasis or it might've been the fact that a bone metastasis went from being absent on CT to a sclerotic lesion at six months combined with appropriate changes in PSA. So we had a predefined criteria system that enabled us to define the ground truth and this enabled us to very cleanly define the accuracy of the staging investigations and accurately compare at bone scan to CT.

We also had a number of key secondary outcome measures, the main one being management impact. The referring physicians were asked to record their management intent prior to randomization, then after first-line imaging, after second-line imaging so we could see whether these results resulted in a change in patient management. We also looked at the difference in equivocal results between the two arms. These are the number of uncertain findings as the result of a scan report.

We looked at the radiation dose of the two imaging and modalities between the two arms. And we also looked at findings in the patients that crossed over to second-line and that gave us an assessment of the incremental accuracy of both conventional imaging or PSMA PET after first-line imaging.

Alicia Morgans: Wonderful. So can you tell me and just remind us specifically who were these patients? We talked about high-risk patients at the start, but I think this was high-risk patients. And how many did you end up enrolling in each of your arms?

Declan Murphy: So the patients we selected for this study were newly diagnosed patients with high-risk features. So predominantly, this is around what we consider to be traditional high-risk features by D'Amico or NCCN or EAU criteria. So PSA greater than 20 or clinically T3 cancer or ISUP grade group, four or five, which are the classic high risks. But we also chose to include ISUP group three so that's old fashioned Gleason 4+3=7. Because we're aware within that higher intermediate risk category, these men have a higher chance of biochemical recurrence. So we thought it would be of value to include these higher intermediate risks as well as the classic high risks.

So these were newly diagnosed patients. They were allowed to have PSA levels and up to 100 and in other words, they were being considered for curative-intent treatment either with surgery or radiotherapy. And as Michael pointed out, management intent was a key secondary endpoint of the study. So once we diagnosed these patients that had their biopsy, we hadn't done any staging, we then recorded the management intent, whether that may be surgery or radiotherapy or so on. And then we submit them to the study and I'll hand over to Michael and he'll describe at the study schema, which we can show to you as well on the screens.

Michael Hofman: Yeah, so this was a 300 patient study. We randomized 302 patients, 152 in the conventional arm and the 150 in the PSMA PET/CT arm. They were followed up at six months. We had very few drop-offs. We had two patients in the conventional arm and three patients in the PSMA arm. And that means we were able to analyze the primary endpoint in 150 patients in the conventional arm and 145 patients in the PSMA PET arm and the results, in summary, the reference standard showed that 30% of these men had nodal or distant metastatic disease. And what we showed is that PSMA PET/CT had a 27% greater accuracy than conventional imaging. So that's an accuracy of 92% for PSMA PET compared to 65 for bone scan CT. And this is one scan, the PSMA PET compared to two scans or two visits, the CT and the bone scan.So we must remember that the advantage of a PSMA PET is that it's a one-stop-shop.

And we also broke that down into nodal or distant metastatic subgroups. So end-stage or end-stage and PSMA PET was as similarly superior. And we also performed a sensitivity analysis where equivocal lesions were considered positive for metastatic disease rather than at negative. And the superiority of PSMA PET persisted in that sensitivity analysis as well. So I think the take-home message here is that the primary endpoint of accuracy showed a large difference in favor of PSMA PET/CT.

Alicia Morgans: So really interesting. And Declan, as an urologic oncologist, I'd love to hear your thoughts on those patients who ended up going forward with surgery. Because presumably some of them would have. Were there patients who had nodal positivity, for example, at the time of surgery that were missed by the PSMA or what was the false positive and false negative rate at the time of surgery?Because I think that this is something that people talk about, "Oh, PSMA could have a lot of false positives or certainly miss things if they're too small." So what was your experience?

Declan Murphy: Yeah, so yes, a significant proportion of these patients did go on and have surgery. So, therefore, the radical prostatectomy and lymph nodes become the ground truth, if you like, for those patients. But let me tell you, as we've seen in other retrospective series and in big systematic reviews that we've done on experience of PSMA PET/CT. We see first of all that the specificity is extremely high, well over 90% in other words, if you see high avidity in a lesion, a lymph node for example, on PSMA PET/CT, it's almost always right.

And that's a really important point. I think that ProPSMA will help emphasize previous data predominantly retrospective open to selection bias, but in this prospective study, we show with very tight control of multicenter on the PET imaging, we show very high specificity. If these things light up, it's almost always right.

On the other hand, on the sensitivity side, we always know that every imaging modality, even advanced molecular imaging like PSMA PET/CT will always be incapable of detecting tiny lesions below a certain threshold. But as Michael mentioned at the start, we do see extraordinary sensitivity once lesions get to about three millimeters in size or thereabouts compared to, for example, as we know with conventional imaging with CT and we cannot define a node as being positive until it gets to 10 millimeters in size.

You know that is the threshold because of the lack of specificity in conventional imaging. But what we see is once you're down to, once you're at about three millimeters or above, you will see avidity in many lesions, below that you will not. So a PSMA PET/CT from a surgeon's perspective will never replace a lymph node dissection if you really value seeing whether these nodes are positive or not.

But as we will see in the management impact from this study, this imaging has moved the conversation forward for a lot of clinicians managing these patients. So what do you do in your high-risk patient who has a negative PSMA PET/CT for nodes, do we still need to go and do a node dissection? Well, if you really value the staging, many will still do a node dissection, but we also know that lymph node dissection does not improve survival in high-risk prostate cancer and those lead to complications in a proportion of patients, again by current EAU guidelines.

And I think what we see in Australia and other countries where there's a lot of PSMA PET/CT is people will counsel their patients and say, "Well, look your PET/CT is negative for the nodes." If we did a node dissection, a proportion of patients will still have microscopic disease, but it's not going to improve your survival and it does lead to an increase in complications. And I believe that we will see less lymph node dissection in patients who have negative PET scans and whether that's right or wrong.

And one of the other aspects about novel imaging for staging these patients, Alicia, is that you do see lymph nodes in places that are outside the standard template for an extended pelvic lymph node dissection. And this explains to me why these patients do not have a survival benefit. It's because we are seeing lymph nodes in the presacral spaces, the mesorectal spaces, left supraclavicular lymph nodes and so on without necessarily having nodes in the classic internal-external iliac regions there for example.

So I think the management impact is very significant as Michael will come to in a second when we speak about this, but an obvious flow on from that, which I'm sure we'll discuss is does this improve outcomes for our patients? It certainly has higher accuracy as we've just demonstrated. It has a high management impact. But this study is not designed to show whether that will improve longer-term outcomes for our patients.

Alicia Morgans: And maybe the next study will be an adaptive surgical approach around the PSMA findings. And you can see if you do a dissection in the mesorectal area or if you address more distant disease either by not operating on them or by using SBRT for something that perhaps you can change outcomes. But the first step may be in identifying the extent of disease and its location. So certainly this seems to be a step in the right direction.

And I'd love to hear from Michael, from a nuclear medicine perspective. This was a group of patients who all had a PSA that was greater than or equal to 20 nanograms per milliliter or as I understand, although that, please correct me if I'm wrong. If patients have high-risk features by other criteria, perhaps they didn't need to have a PSA that high. But if you're trying to apply this data in a clinical space, you may have a PSA that's lower. What is your PSA threshold for thinking about using PSMA PET in this setting?

Michael Hofman: Yeah, so in this study, you did not have to have a PSA greater than 20 which was one of the measures of high-risk disease. But you might just have had Gleason grade group three, four or five or clinical T stage three or greater.

Alicia Morgans: Very good. But if you were applying this data, I usually think about this cutoff of around two or sometimes people say down to one or 1.5. Where is your cutoff when you're thinking about a PSMA PET? For those of us who don't have access to these scanners at this point, where do you see the highest accuracy? And I think we all recognize and you should tell us that this is a curve, right? So there's no absolute value of PSA that's going to be the cutoff above which or below which will have an accurate scan. But where do you think about things being generally more accurate versus less?

Michael Hofman: Yeah, look, it's a great question. In this study, most of the patients are actually selected on the basis of histopathology, so the Gleason grade group and PSA, therefore, did not play a part. So we have not actually teased out some of these subgroup analyses, but I think your PSA question is a very interesting one to go back and look at the data and see if there's a threshold below, which even despite having a Gleason grade group three perhaps if your PSA is 1.5, do you need a PSMA PET? These are some further analyses that we can look at.

Declan Murphy: But I think we could say to clinicians out there, Alicia, that if you're looking at this data and trying to think how does it apply to my practice or how might it apply if I have access to PSMA PET/CT? I would make two points.

First of all, they had these inclusion criteria. That's what we use in practice nowadays. So if you have these high-risk or higher intermediate-risk, in other words, that grade group three at least in 4+3 at diagnosis, then what this trial shows is first of all, and the accuracy is much higher and if you have a PSMA PET/CT, so that's our routine staging, we might as well get that accurate information.

And the second point, which will I hope have global implications is that we see this trial as being a vehicle to help get PSMA PET/CT standardized around the world and reimbursed around the world. Unlike a lot of the oncology trials that readout as positive endpoints, the survival benefit, et cetera. But sometimes the drug is so expensive in countries it's really not going to be impactful.

PSMA PET/CT, at least in our experience is not an expensive imaging tool. And we see this as a platform for people all around the world to work with their local nuclear medicine people with their regulators, with their funding bodies to say, "Hey, come on, the cost, we'll show this in this study of doing a PSMA PET/CT" for us is about equivalent to doing a CT and a bone scan in our environment.

So why would we do that when we have higher accuracy and, and perhaps Michael, we'll talk about the equivocal studies and the radiation dose, which were important secondary endpoints, which I think feed into an argument that we would like to see carried on around the world about making this scan available on a widespread basis?

Michael Hofman: Yeah, and I think it's worth adding that a key feature of this study is that we assist PSMA PET as a replacement for CT and bone scan. So many imaging studies for new tests look at the additional value of adding a new test. This study was saying, "Well, if you were scheduled for a CT and bone scan" and not everyone needs imaging. If you're low risk, you don't need imaging. But if you are a patient that needs a CT and bone scan by current guidelines, then you are better off having a PSMA PET as singles test rather than two tests." Instead, you no longer need the CT and the bone scan.

And just want to go back and focus on some of the secondary objectives of the study. So the primary objective being accuracy, but then management impact. We looked at management impact and we defined a medium or high impact as a change in therapeutic modality. So let's say surgery to hormone therapy or a change within the therapy. So for example, Declan's looked at the scan and is now going to extend his pelvic nodal dissection a little bit or the radiation oncologist is going to change his radiation field. And that's encompassed a significant management change. And that occurred in 15% of patients who underwent a CT in bone scan and 27% in patients with a PSMA PET/CT. So that's almost like doubling of the high to medium management impact of the study.

Another key secondary objective was looking at equivocal or uncertain findings between the imaging arms because no one likes to get a bone scan or CT scan back saying that there is an equivocal abnormality, it could be a metastasis, but we really don't know. And the conventional arm had equivocal findings in 23% so almost a quarter of scans versus PSMA PET that had equivocal findings in 7%, so that's a significant reduction and that's attributed to the high tumor contrast.

These tumors light up very brightly on a PSMA PET scan. It's easy for the readers to look at these scans. We also looked at reporter agreement, so all the PSMA PET scans were read a second time by a core imaging laboratory of experts and we compared it to the local review at the 10 sites around Australia. And it's important that PSMA PET was found to have a very high reporter agreement, a Kappa score of .88 so experts were agreeing amongst each other. This is really important because it's very hard to have an accurate test if five people look at the scan and come up with different conclusions.

And the last key secondary endpoint that I'd like to mention was radiation dose. Now because these are often well men that are going to be cured and we don't want to expose them to large amounts of radiation from diagnostic imaging and PET scanning might sound a little bit scary to people out there who aren't familiar with the technology, but in fact, the PSMA PET scan approach exposed men to half the radiation of a CT and bone scan. So that was eight millisieverts that's for a PSMA PET/CT compared to 19 millisieverts with a combination of a CT and bone scan.

Alicia Morgans: So when we think about it from a nuclear medicine perspective in terms of rolling out technology, there's high agreement between the local readers and these experts suggesting that with some training this is something that can be disseminated or implemented at sites around the world with really good accuracy. And for the patients to understand the importance of radiation. They all know it anyway, but they would actually receive half of the dose of radiation with this assessment as compared to the combination of bone scans and CTs that we actually re routinely use.

That's very, very critically important information. So as we wrap this up, and this is again, phenomenal work, gentlemen and to your entire teams. Because this has been something that I think the world has been waiting for, to think about trying to broaden the spectrum of applicability of PSMA PET. What would your take-home message be to audiences around the world as they're trying to think about how do we get this technology? How do we use your data in our day to day practices?

Declan Murphy: Well, thank you. Alicia. I'll give you my perspective on it as someone who's worked closely with Michael and Rod Hicks and his other colleagues in here and at other centers is I think there's a great opportunity space for clinicians like me and you working in prostate cancer to buddy up with talented nuclear medicine physicians all around the world. And often there's a bit of a disconnect there. Before all this came along, nuc med didn't come into our tumor board into our multidisciplinary team meetings.

But when all the PSMA PET started arriving, we started saying, "Hey folks, come and sit in our meeting. We'd all learn from each other." And now we have this really productive environment that's leading to lots of trials and also trials in therapies like lutetium PSMA. So I say to people out there, if you don't have a close working relationship with the nuc meds in your hospital, you should go down and find where they live. A knock on their door and say, "Hello, I'm a prostate cancer clinician, are you interested in PSMA PET?" And they're going to go, "Oh yes, I've just been to a conference and everyone's talking about PSMA PET" or "Do you have any prostate cancer patients?".

Because that community is very interested just as we are as prostate cancer clinicians and my messages to encourage people to go and work with each other. Encourage yourselves to share the tumor boards together or have virtual meetings together because I think there's a great opportunity space as this trial shows that for us to hopefully make a difference for our patients by collaborating more with our nuc med physicians.

Michael Hofman: Yeah. I share that key message as part of this study we set up a Tencent network around Australia with really multidisciplinary expertise. That network's now being used for several further studies both in the PSMA PET imaging space, but also in lutetium PSMA therapy space. And maybe I'll leave you with the key message of the trial, which is really that the data that we've produced here as support PSMA PET/CT as a replacement to the current standard of CT and bone scan, and we think this is beneficial for patients as a one-stop-shop investigation and also improving that patient navigation through the imaging pathway with fewer hospital visits and less radiation dose, more accurate imaging.

Alicia Morgans: Wonderful. Well, I sincerely appreciate your time. I know that everyone viewing and listening will be on the lookout for that Lancet paper, which is coming out in March, which is really going to detail the findings from this ProPSMA Phase III trial that looked at PSMA PET/CT as a replacement for CT and bone scan in the high-risk localized prostate cancer population. And certainly, I'm sure that they will be on the lookout for your presentation at EAU.

Thank you both so much for your time and again, congratulations on this excellent work.

Declan Murphy: Thank you.

Michael Hofman: Thank you so much.

Originally posted here:
PSMA PET/CT Imaging for Staging High-risk Prostate Cancer Prior to Curative-intent Surgery or Radiotherapy (proPSMA) - Michael Hofman and Declan...

Recommendation and review posted by Bethany Smith

On a Friday evening last month, Fernando Tatis Jr. sauntered toward an area beside the home dugout at Peoria Stadium. A spring training game against the Chicago Cubs was nearing its start. Tatis, the Padres magnetic young shortstop, had finished warming up on the field. With time to spare, he approached the barrier between players and fans.

Baseballs, pens and various other items were thrust in his direction. Hopeful pleas filled the air. The assembled throng appeared to be a typical collection of autograph-seekers, with one exception: A woman with a shaved head stood in the front row. In one hand, Gina Grosso held a baseball. In the other, she clutched a poster board advertising an unusual request.

All I want for CHEMO is for TATIS to sign my BALD HEAD!

Within seconds, the message had been spotted by its intended recipient. Tatis briefly stopped to sign for a couple of young children.

And then, Tatis recalled, I went straight to...

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Fernando Tatis Jr., a signature and the moment a fan hopes will help save lives - The Athletic

Recommendation and review posted by Bethany Smith

LONDON--(BUSINESS WIRE)--The global autologous cell therapy market is poised to grow by USD 1.97 billion during 2020-2024, progressing at a CAGR of almost 22% during the forecast period. Request free sample pages

Read the 120-page report with TOC on "Autologous Cell Therapy Market Analysis Report by Therapy (Autologous stem cell therapy and Autologous cellular immunotherapies), Application (Oncology, Musculoskeletal disorders, and Dermatology), Geography (North America, APAC, Europe, South America, and MEA), and the Segment Forecasts, 2020-2024".

https://www.technavio.com/report/autologous-cell-therapy-market-industry-analysis

The market is driven by the increasing demand for effective drugs for cardiac and degenerative disorders. In addition, the limitations in traditional organ transplantations are fueling the demand for stem cell therapies. All these factors are anticipated to boost the growth of the autologous cell therapy market.

The demand for effective drugs for cardiac and degenerative disorders has been increasing across the world. In addition, the discovery of possible cardiac autologous cells has enabled vendors to develop novel drugs for the treatment of various cardiac diseases. For instance, Mesoblast is developing MPC-150-IM. It is a Phase III candidate for the treatment of advanced and end-stage chronic heart failure. Similarly, Shire has been developing autologous stem cell therapies for chronic myocardial ischemia. These products are expected to be launched during the forecast period and will have a positive impact on the growth of the global autologous cell therapy market.

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Major Five Autologous Cell Therapy Market Companies:

Bayer AG

Bayer AG operates its business through segments such as Pharmaceuticals, Crop Science, Consumer Health, and Animal Health. The company offers induced pluripotent stem cells. They are developed by reprogramming mature body cells to behave like embryonic stem cells that are injected to restore diseased tissue in patients.

Brainstorm Cell Therapeutics Inc.

Brainstorm Cell Therapeutics Inc. operates its business through an unified business segment. NurOwn is the key offering of the company. It is a cell therapy platform, which develops mesenchymal stem cells for the treatment of human diseases such as immune and inflammatory diseases.

Daiichi Sankyo Co. Ltd.

Daiichi Sankyo Co. Ltd. operates its business through segments such as Innovative Pharmaceuticals, Generic, Vaccine, and OTC Related. Heartcel is the key offering of the company. It is an immune-modulatory progenitor cell therapeutic agent, which is used for ischemic heart failure.

FUJIFILM Holdings Corp.

FUJIFILM Holdings Corp. operates its business through segments such as Imaging solutions, Healthcare and material solutions, and Document solutions. The company uses induced pluripotent stem cells to derive differentiated cells, which are used in researching various diseases and conditions.

Holostem Terapie Avanzate Srl

Holostem Terapie Avanzate Srl operates its business through an unified business segment. Holoclar is the key offering of the company. It is an advanced therapy medicinal product containing stem cells indicated to repair the cornea after injury.

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Autologous Cell Therapy Market Therapy Outlook (Revenue, USD Billion, 2020-2024)

Autologous Cell Therapy Market Application Outlook (Revenue, USD Billion, 2020-2024)

Autologous Cell Therapy Market Regional Outlook (Revenue, USD Billion, 2020-2024)

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Related Reports on Healthcare Include:

Global Cancer Stem Cell Therapeutics Market Global cancer stem cell therapy market by type (allogeneic stem cell transplant and autologous stem cell transplant) and geography (Asia, Europe, North America, and ROW).

Global Mantle Cell Lymphoma Therapeutics Market Global mantle cell lymphoma therapeutics market by product (combination therapy and monotherapy) and geography (Asia, Europe, North America, and ROW).

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Global Autologous Cell Therapy Market 2020-2024 | Evolving Opportunities with Bayer AG and Brainstorm Cell Therapeutics Inc. | Technavio - Business...

Recommendation and review posted by Bethany Smith

While many of us are practicing social distancing, working from home or living in quarantine-like and isolated situations, life goes on as normal for the space station-dwelling astronauts.

They're aware of the pandemic and have been sharing their support for people across the globe through their Twitter accounts. NASA astronaut Jessica Meir shared her perspective: "From up here, it is easy to see that we are truly all in this together. #EarthStrong."

But the astronauts aren't just floating around and taking cool pictures of Earth. Each week, hundreds of science experiments are in progress on the station. In addition to working on these experiments, the astronauts study themselves to better understand the human body in space.

Here's a look at the cool science they've been doing 254 miles from Earth.

Space pants

Living in space is an adjustment for the human body as it adapts to the lack of gravity.

Over the years, astronauts have noticed changes in their vision as a response to the headward fluid shift they experience. This also increases pressure in the head.

Last week, NASA astronauts Jessica Meir and Andrew Morgan, as well as Russian cosmonaut Oleg Skripochka, tested out the Russian Chibis hardware, also known as the Russian Space Agency's Lower Body Negative Pressure experiment.

It's basically a pair of pants housed in the Russian Orbital Segment of the space station.

The rubber pants use suction to draw fluids back down towards the legs and feet, just like we experience walking on Earth.

Researchers hope that hardware to reverse the fluid shift astronauts experience in space could also help with their vision changes.

While Morgan was wearing the Chibis pants, Meir used a tonometer to measure his eye pressure, with doctors on Earth watching in real time. Morgan's head and chest were also scanned to monitor blood flow.

The astronauts also tested their hearing as part of the European Space Agency's Acoustic Diagnostics experiment to monitor if the astronauts' hearing changes in response to noise and lack of gravity on the station.

Heart, muscle and bone

Multiple experiments are currently occurring on the station that could not only benefit the health of astronauts, but human life on Earth as well.

These cells could treat astronauts who experience heart abnormalities and be used to treat people and children with cardiac diseases and disorders on Earth. The cells can also be used to investigate the development of new pharmaceuticals.

One experiment, called Engineered Heart Tissues, allows the astronauts to watch heart cell muscle contractions in real time.

Meir and Morgan have been taking care of the heart cells, watching how they react to the lack of gravity. When the heart cells return to Earth, the results of the space experiment will be compared with a similar control experiment on Earth.

The astronauts have also been studying bone samples to understand and develop bone treatments for astronauts who suffer bone loss in space, as well as people diagnosed with osteoporosis on Earth. The goal is to determine new treatments for both.

Mice are also sharing space on the station with the astronauts in a mouse habitat so they can study how the mice and their gene expression reacts to zero gravity.

Understanding how their gene expression is altered can help NASA better prepare for long-term human spaceflight. The study also serves a secondary purpose of allowing them to determine countermeasures for muscle atrophy, which can occur in space or for patients on bed rest.

It's all in your gut

Astronauts don't get much of a chance to vary their diets in space. That means they could also be missing out on vital nutrients and other added benefits of the fresh food we consume on Earth.

The Japanese space agency's Probiotics investigation is studying how good gut bacteria could improve the human microbiome on long-term missions.

Meanwhile, the astronauts are also participating in an experiment called Food Acceptability, looking at the "menu fatigue" that happens when they eat based on limited options over months on the station. This usually causes them to lose weight by the time they return to Earth.

Continued here:
While we were stockpiling, here's what astronauts were up to in space last week - CNN

Recommendation and review posted by Bethany Smith

Regenerative Medicine Market: Snapshot

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

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Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

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Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe areVericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc.A large number of players are anticipated to enter the global market throughout the forecast period.

About TMR Research:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

See original here:
Regenerative Medicine Market Demand, Growth, Opportunities and Analysis Of Top Key Player Forecast To 2025 - Daily Science

Recommendation and review posted by Bethany Smith

With approximately 1 in every 18 people in the world using drugs, theres a growing global prevalence for substance use, according to the latest World Drug Report by the United Nations. While many people are able to use certain substances recreationally, some abuse substances, and others fall into the addiction category.

Addiction, however, doesnt come down to a lack of willpower or a series of bad choices. Whether you become an addict or not depends on a combination of factors, including genetic loading, early childhood trauma, an adverse home environment, and specific personality traits such as impulsivity, poor emotional regulation and distress tolerance. These all increase your risk dramatically, says Jean Coetzee, the Clinical Manager at the Harmony Addiction and Psychiatric Clinic in Hout Bay.

At the basis of every addiction, regardless the type, lie similar drivers, as just mentioned. The drug of choice is just the symptom, but the addiction is the actual disease, says Siobhan Alford, Hospital Manager at the Harmony Addiction and Psychiatric Clinic.

Neuroplasticity: The hope in the fight against addiction

Addictive behaviour is a result of the brains ability to adapt and form patterns, according to a study published in the journal Dialogues in Clinical Neuroscience. Most drugs that are abused activate dopamine (the happy hormone) in the brains reward system. This novel experience produces patterns in the brain that link external cues; to the use of drugs; to the reward system. Over time, the reward system is automatically triggered by these cues alone, leading to an urge to take drugs.

Luckily, every problem has in it the seeds of its own solution. Coetzee explains that when you start adopting healthy coping mechanisms, rather than unhealthy ones like substance abuse, positive neural pathways can also become entrenched in the brain. The two ways in which you can rewire the brain and entrench positive coping mechanisms is through repetition and novel experiences, Coetzee says. There is hope in recovering from addiction because it is possible to change the real estate (physical structure) of your brain and consequently your behaviour and thinking too.

How to identify addictive behaviour

Addictive behaviour usually develops in vulnerable individuals, but how do you know if you or a loved one is at risk of addiction? Although using substances that activate the reward system can contribute to the development of addiction, vulnerability to addiction is influenced by a complex set of variables such as genetic predisposition, environmental circumstances, psychological factors, social pressures or trauma. A strong connection between trauma, post-traumatic stress disorder (PTSD) and addiction has been found in studies by renowned expert Prof. Alexander McFarlane.

A simple way of identifying addictive behaviour in a loved one or yourself is through the presence of the 4 Cs:

In South Africa, alcohol is still the number one abused substance followed by cannabis, methamphetamine and then opioids (which includes codeine, heroine, nyaope and wonga), according to the SACENDU Project. Besides substances, process addictions like gambling, gaming and sex are also very prevalent and treated with the same gravitas as any other drug.

The road to recovery

The first step to recovering from addiction is taking responsibility to become clean. You dont choose to be an addict, but you can choose to become sober, Alford says. Making an appointment at a registered rehabilitation centre is one of the first steps that you can take to come clean. Some centres, like Harmony Clinic, work with medical aids to cover drug and alcohol rehabilitation treatment.

Harmony Clinic takes an evidence-based scientific and medical approach to treating addiction through a biopsychosocialspiritual model. As a dual diagnosis hospital, patients who are admitted to the clinic see a psychiatrist within 48 hours and a general practitioner within 24 hours of their admission, Alford says.

Alongside the medical approach, DBT (Dialectal Behavioural Therapy) skills such as mindfulness are a strong foundation of evidence-based addiction treatment. After eight weeks of daily mindfulness practice, the real estate of the brain can already start to change, Coetzee says. Although the brain rewires quite rapidly, individuals can still be vulnerable years after theyve started their recovery process which is why abstinence (or harm reduction where abstinence is not possible) is strongly recommended.

The road to recovery is equally as important for family members as for addicts themselves. Patients usually show significant progress in therapy and then they go home to where circumstances havent changed, Alford says, which is why experts advise family members of recovering addicts to get their own therapy and to join support groups. Popular networks for family support groups in South Africa include Al-Anon and Nar-Anon.

This post is sponsored by Harmony Clinic produced by Brandstudio24 for Health24.

Anina Visser - PARTNER CONTENT

See more here:
You are not your addiction - Health24

Recommendation and review posted by Bethany Smith

When it comes to squids, you just can't keep them down.

Not just because they're slippery, but also because they have an incredible genetic editing ability - it lets them tweak their own RNA long after it's left the nucleus.

Here's what that means. Genes, in humans at least, mostly stay unchanging until they're recombined and passed onto the next generation.

This is the same for our messenger RNA (mRNA). Helpful molecules read our DNA, create short little RNA messages, and send them outside the nucleus to tell the rest of the cell which proteins need to be built.

Once that mRNA has exited the nucleus, it's thought the genetic information it carries can't be messed with much - but new research has shown that in squid nerves, this isn't the case.

"We are showing that squid can modify the RNAs out in the periphery of the cell," says Marine Biological Laboratory (MBL), Woods Hole geneticist Joshua Rosenthal.

"It works by this massive tweaking of its nervous system," Rosenthal told Wired. "Which is a really novel way of going through life."

The team took nerves from specimens of adult male longfin inshore squid (Doryteuthis pealeii), and analysed the protein expression, as well as the squids' transcriptome, which is similar to a genome, but for mRNA.

They found that in squid nerves (or neurons), the mRNA was being edited outside of the nucleus, in a part of the cell called the axon.

This mRNA editing allows the squids to finely tune the proteins they produce at local sites (see diagram below). With this finding, squids have become the only creatures we know of that can do this.

(Vallecillo-Viejo et al., Nucleic Acids Research, 2020)

This isn't the first time squids have shown off their genetic editing prowess, though. Back in 2015, a similar team at MBL discovered that squids edit their mRNA inside their nucleus to an incredibly large degree orders of magnitude more than what happens in humans.

"We thought all the RNA editing happened in the nucleus, and then the modified messenger RNAs are exported out to the cell," Rosenthal explains.

But the team showed that although editing is happening in both, it occurs significantly more outside the nucleus in the axon, rather than inside the nucleus.

So, why do squids bother? Why do they need to change their mRNA so much?Well, we don't yet know, but the research team has some ideas.

Octopus, cuttlefish and squids all use mRNA editing to diversify the proteins produced in the nervous system. This could be one of the reasons why these creatures are so much smarter than other invertebrates.

"The idea that genetic information can be differentially edited within a cell is novel and extends our ideas about how a single blueprint of genetic information can give rise to spatial complexity,"the team writes in their new paper.

"Such a process could fine-tune protein function to help meet the specific physiological demands of different cellular regions."

Although right now this is just an interesting genetics study into squids, the researchers think that eventually, this type of system might be able to help treat neurological disorders that include axon dysfunction.

CRISPR has completely changed the game when it comes to editing the DNA inside our cells, and RNA is significantly less permanent and therefore editing it could be less dangerous.

"RNA editing is a hell of a lot safer than DNA editing," Rosenthal told Wired.

"If you make a mistake, the RNA just turns over and goes away."

The research has been published in Nucleic Acids Research.

Continue reading here:
Squids Can Edit Their RNA in an Unprecedented Way, Scientists Discover - ScienceAlert

Recommendation and review posted by Bethany Smith

Scientists at the Lewis Katz School of Medicine at Temple University (LKSOM) and Fox Chase Cancer Center show that EGR4, known mainly for its role in male fertility, serves as a critical brake on immune activation. The new study Suppression of Ca2+signals by EGR4 controls Th1 differentiation and anticancer immunityin vivo,published online inEMBO Reports, demonstrates that taking EGR4 away, thus effectively releasing the brake, promotes the activation of killer T cells, which infiltrate and attack tumors and thereby boost anticancer immunity.

While the zinc finger transcription factors EGR1, EGR2, and EGR3 are recognized as critical for Tcell function, the role of EGR4 remains unstudied. Here, we show that EGR4 is rapidly upregulated upon TCR engagement, serving as a critical brake on Tcell activation. Hence, TCR engagement of EGR4/T cells leads to enhanced Ca2+responses, driving sustained NFAT activation and hyperproliferation. This causes profound increases in IFN production under resting and diverse polarizing conditions that could be reversed by pharmacological attenuation of Ca2+ entry, wrote the investigators.

Finally, aninvivomelanoma lung colonization assay reveals enhanced antitumor immunity in EGR4/mice, attributable to Th1 bias, Treg loss, and increased CTL generation in the tumor microenvironment. Overall, these observations reveal for the first time that EGR4 is a key regulator of Tcell differentiation and function.

Other early growth response proteins, or EGRs, are important to T cell activity, but whether EGR4 also has a role in immunity has been largely overlooked, explained Jonathan Soboloff, Ph.D., professor of Medical Genetics and Molecular Biochemistry at the Fels Institute for Cancer Research and Molecular Biology at LKSOM. Our study reveals a new side to the importance of EGR4.

Soboloffs team examined the influence of EGR4 expression in immune cells in collaboration with Dietmar J. Kappes, Ph.D., professor of Blood Cell Development and Cancer at Fox Chase Cancer Center. In initial experiments, the researchers found that T cell activation is associated with EGR4 upregulation. They then showed that knocking-out, or eliminating, EGR4 from immune cells results in a dramatic increase in calcium signaling and expansion of T helper type 1 (Th1) cell populations. Th1 cells, in response to the presence of foreign entities, including tumor cells, activate cytotoxic, or killer, T cells, which then wipe out the invader.

We know from our previous work that T cells control calcium signaling and that when intracellular calcium levels are elevated, calcium signaling can drive T cell activation, Soboloff said.

The Soboloff and Kappes labs next studied the functional importance of EGR4 in cancer immunity by utilizing an adoptive mouse model of melanoma in which some host animals lacked EGR4 expression. Compared to mice with typical EGR4 levels, EGR4 knockout animals showed evidence of expanded populations of Th1 cells and enhanced anticancer immunity. In particular, EGR4 knockout mice had reduced lung tumor burden and fewer metastases than mice with normal EGR4 expression.

In future work, the Soboloff and Kappes groups plan to further explore strategies for EGR4 targeting. The development of an agent to target EGR4 specifically may be difficult, due to the diverse actions of EGR pathways. But eliminating EGR4 specifically from a patients T cells, and then putting those cells back into the patient, may be a viable immunotherapeutic approach, Kappes said.

More:
Removing EGR4 "Brake" on Immune Activation May Be Viable I/O Therapeutic Approach - Clinical OMICs News

Recommendation and review posted by Bethany Smith

The "Stem Cell and Gene Therapy Biological Testing Market, Size, Share, Outlook and Growth Opportunities 2019-2025" report has been added to ResearchAndMarkets.com's offering.

Stem Cell and Gene Therapy Biological Testing market outlook report presents key insights into global Stem Cell and Gene Therapy Biological Testing markets and companies with emphasis on market size, growth factors, R&D investments, market shares and 2019 market dynamics.

The report is structured to understand the key strategies of Stem Cell and Gene Therapy Biological Testing companies in terms of their investments, product portfolio, potential opportunities and future plans. The Stem Cell and Gene Therapy Biological Testing report provides 7-year outlook on market size across different Stem Cell and Gene Therapy Biological Testing types, applications, end-user industries and countries.

Scope of the report

Key Topics Covered:

1. Table of Contents

2. Global Stem Cell and Gene Therapy Biological Testing Industry Overview

2.1 Key Findings

2.2 Market Definition

2.3 Industry Overview

2.4 Report Guide and Research Methodology

3. Executive Summary

3.1 Key Trends Shaping the Future of Stem Cell and Gene Therapy Biological Testing Market to 2025

3.2 Key focus areas of Leading Manufacturers in the market

3.3 Potential Application Segments with strong growth prospects, 2019-2025

3.4 Key Emerging Markets vital for growth of Stem Cell and Gene Therapy Biological Testing Market

3.5 Prominent Types of Stem Cell and Gene Therapy Biological Testing set to Gain Market Shares, 2019-2025

4. Stem Cell and Gene Therapy Biological Testing Market Strategic Analysis Review

4.1 Porter's Five Forces Analysis of Global Stem Cell and Gene Therapy Biological Testing Market

4.2 Supply Chain Analysis of Stem Cell and Gene Therapy Biological Testing Industry

4.3 Pricing Analysis and Forecasts

4.4 SWOT Analysis

5. Global Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

5.1 Global Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

5.2 Global Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

5.3 Global Stem Cell and Gene Therapy Biological Testing Market Outlook by Region, 2019-2025

6. Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

6.1 Introduction

6.2 Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

6.3 Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

6.4 Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

6.5 Leading Companies

7. Europe Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

7.1 Introduction

7.2 Europe Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

7.3 Europe Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

7.4 Europe Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

7.5 Leading Companies

8. North America Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

8.1 Introduction

8.2 North America Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

8.3 North America Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

8.4 North America Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

8.5 Leading Companies

9. Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

9.1 Introduction

9.2 Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

9.3 Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

9.4 Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

9.5 Leading Companies

10. Competitive Landscape

10.1 Leading Players

10.2 Market Shares of Top Companies in Revenue Terms, 2018

10.3 Company Benchmarking (Peer-to-Peer Comparison)

10.4 Financial Analysis

11. Business Profiles of Leading Stem Cell and Gene Therapy Biological Testing Companies

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12. Stem Cell and Gene Therapy Biological Testing Market Recent News and Deals Landscape

12.1 Mergers and Acquisitions

12.2 Stem Cell and Gene Therapy Biological Testing Market New Product Launches

12.3 Asset Transactions

12.4 Financial Announcements

13. Appendix

For more information about this report visit https://www.researchandmarkets.com/r/ol7uf2

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Global Stem Cell and Gene Therapy Biological Testing Market (2019 to 2025) - Potential Growth Opportunities and Areas of Focus -...

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Three years after a brain cancer failure sent the company reeling, VBL Therapeutics is touting its first hint of positive Phase III results.

The Israeli biotech announced its lead drug, VB111, met an interim efficacy benchmark in a trial testing it against standard-of-care alone in recurrent, chemotherapy-resistant ovarian cancer. The patients in the treatment arm had a CA-125 response rate a measure of cancer antigen often used as a proxy in ovarian cancer studies and in detecting ovarian cancer at least 10% higher than those in the control, the independent review determined.

The response rate in the first 60 enrolled and evaluable patients was 53%. That indicated a treatment response rate of at least 58% an encouraging number, the company said, because of the CA-125 data from their earlier Phase II trial.

We are very pleased by the outcome of this interim analysis, which demonstrates the potential benefit of VB-111 over standard-of-care in a randomized-controlled study, VBL CEO Dror Harats said. The OVAL Phase 3 interim data are at least as good as the CA-125 response results observed in our VB-111 Phase 2 study.

In that earlier study, which indicated a dose-dependent response, patients who showed a CA-125 response ultimately had an overall survival rate of808 days, versus 351 days for those who did not. The primary endpoint for this study, which is set to be completed in 2022, is overall survival.

The data come two years after VBL announced the combo of VB-111 and Avastin had failed to beat out Avastin alone in a Phase III study of glioblastoma, a notoriously hard-to-treat indication. It was their first Phase III trial after a smattering of Phase II trials in different solid tumors had turned up mixed results.

VB-111 is what the company deems a gene therapy agent. It uses an adenovirus the viral vector traditionally used for gene therapy that carries a gene to cause explosive cell death in tumors, rupturing blood cells. They have anti-inflammatory and other cancer programs in development.

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VBL and its gene therapy cancer treatment are back with a peek at PhIII potential - Endpoints News

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Basel, March 27, 2020 AveXis, a Novartis company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)has adopted a positive opinion recommending conditional marketing authorization of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. A rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, SMA results in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.2,3 Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworkingSMN1gene. Zolgensma is administered during a single intravenous (IV) infusion, delivering a new working copy of the SMN gene into a patients cells, halting disease progression. The positive opinion is an important step towards offering a new treatment option in Europe for babies and young children with SMA.

The European Commission (EC) reviews the CHMP recommendation and usually delivers its final decision in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway, Liechtenstein and the United Kingdom.

Todays positive CHMP opinion for Zolgensma marks a critical step closer to EC approval and to bringing the only gene therapy for SMA to Europe, helping to address the devastating impact the disease has on patients and their families, said Dave Lennon, president of AveXis. Zolgensma provides a transformational new way to treat this rare but debilitating disease delivering a potentially life-saving medicine with a one-time administered treatment. Given the urgency to treat SMA and the novel nature of gene therapy, we need to be equally innovative in advancing access, so we are offering governments and reimbursement bodies a Day One access program to enable rapid access to Zolgensma upon approval.

In the most severe forms of the disease, children who are not treated are unable to lift their heads, sit, stand, or even swallow, and typically do not survive beyond two years of age unless permanently ventilated, said Dr. Francesco Muntoni, Professor and Pediatric Neurologist at Great Ormond Street Hospital for Children, London. The results we have seen for Zolgensma to date from the STR1VE clinical trial show an impressive survival rate at the conclusion of the study, with the majority of patients being able to sit without support. And through follow-up on the START trial, an average of 4.5 years later, we can see the long-term potential this significant gene therapy may have for children with this rare disease.

The CHMP positive opinion is based on the completed Phase 3 STR1VE-US and Phase 1 START trials that evaluated the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients <6 months of age at dosing, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. STR1VE-EU, a comparable Phase 3 study is ongoing. Zolgensma demonstrated prolonged event-free survival; rapid motor function improvement, often within one month of dosing; and, sustained milestone achievement, including the ability to sit without support, a milestone never achieved in untreated Type 1 patients.4

Additional supportive data included interim results from the ongoing SPR1NT trial, a Phase 3, open-label, single-arm study of a single, one-time IV infusion of Zolgensma in presymptomatic patients (<6 weeks at age of dosing) genetically defined by bi-allelic deletion of SMN1 with 2 or 3 copies of SMN2. These data demonstrate rapid, ageappropriate major milestone gain, reinforcing the critical importance of early intervention in SMA patients.4

The most commonly observed side effects after treatment were elevated liver enzymes and vomiting. Acute serious liver injury and elevated aminotransferases can occur. Patients with pre-existing liver impairment may be at higher risk. Prior to infusion, physicians should assess liver function of all patients by clinical examination and laboratory testing. And, they should administer systemic corticosteroid to all patients before and after treatment, and then continue to monitor liver function for at least 3 months after infusion.4

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We are delighted to know that EMA considers a new treatment effective to fight SMA and that it can benefit a part of our community. We rely on all relevant stakeholders, to work at their best to get it to patients without any delay. SMA Europe will continue working to ensure that all patients living with SMA in Europe have the possibility to access any treatment that can be beneficial for them in a timely and sustainable way, said Menca de Lemus, President of SMA Europe.

Zolgensma Day One access program

SMA is a significant burden to the healthcare system in Europe with cumulative estimated healthcare costs per child ranging between 2.5 to 4 million within the first 10 years alone.1Designed to work within existing pricing and reimbursement frameworks, yet recognizing the novel nature of a one-time gene therapy for a devastating and progressive disease, the Day One access program offers ministries of health and reimbursement bodies (in countries without pre-existing early access pathways) a variety of flexible options that can be implemented immediately at time of approval. The program is designed to ensure that the cost of patients treated before national pricing and reimbursement agreements are in place, are aligned with the value-based prices negotiated following clinical and economic assessments. The program is meant to ensure the continued integrity of the local pricing and reimbursement framework. AveXis is already in advanced discussions with multiple countries in Europe to agree on terms of the program. The following options can be customized for each country:

About Spinal Muscular AtrophySMA is the leading genetic cause of infant death.2,3 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.5In Europe each year, approximately 550600 infants are born with SMA.6,7 SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.2 It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.8 This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.9 More than 30% of patients with SMA Type 2 will die by age 25.10

Novartis will conduct a conference call with investors to discuss this news release on Monday, March 30, 2020 at 3 p.m. Central European Time and 9 a.m. Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Novartis website. A replay will be available after the live webcast by visiting https://www.novartis.com/investors/event-calendar.

About Zolgensma (onasemnogene abeparvovec)Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma represents the first approved therapeutic in the companys proprietary platform to treat rare, monogenic diseases using gene therapy.6 Approximately 400 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program in the U.S. AveXis is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada and Australia in late 2020 or early 2021.6

AveXis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Genethon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

InMay 2019, the U.S. Food and Drug Administration approved Zolgensma for the treatment of pediatric patients less than two years of age with SMA with bi-allelic mutations in the SMN1 gene. 11 In the U.S. nearly all on-label patients have been approved by their payer for access to Zolgensma. On March 19, 2020, Zolgensma was approved by Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis. Reimbursement with MHLW is expected by the end of 1H20, pending agreement Zolgensma will be available at that time.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as positive CHMP opinion, offering, recommending, step towards, step closer, potential, can, will, plan, may, could, would, expect, anticipate, pipeline, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Zolgensma, or regarding potential future revenues from Zolgensma. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Zolgensma, will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Zolgensma will be commercially successful in the future. In particular, our expectations regarding Zolgensma could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.About AveXisAveXis, a Novartis company, is the worlds leading gene therapy company, redefining the possibilities for patients and families affected by life-threatening genetic diseases through our innovative gene therapy platform. Founded in 2013 and headquartered in Bannockburn, IL, the goal of AveXis cutting-edge science is to address the underlying, genetic root cause of diseases. AveXis pioneered foundational research, establishing AAV9 as an ideal vector for gene transfer in diseases affecting the central nervous system, laying the groundwork to build a best-in-class, transformational gene therapy pipeline. AveXis received its first U.S. Food and Drug Administration approval in May 2019 for the treatment of spinal muscular atrophy (SMA). AveXis is also developing therapies for other genetic diseases, including Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) SOD1 and Friedreichs ataxia. For additional information, please visitwww.avexis.com.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more atwww.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisor follow @NovartisNews for the latest News & Media Updates at https://twitter.com/novartisnews.For Novartis multimedia content, please visit https://www.novartis.com/news/media-library.For questions about the site or required registration, please contact media.relations@novartis.com

References1. Estim. 10-year cumulative SMA costs for major EU markets based on available data including local healthcare resource utilizations studies, local databases and public information from previous SMA therapy economic assessments, as of February 21, 2020.2. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895-908.3. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed October 9, 20184. STR1VE-US, START and SPR1NT clinical data on file5. Finkel RS, et al. Neurology. 2014;83(9):810-817.6. Data on file.7. Verhaart IEC, et al. J Neurol. 2017;264(7):1465-1473.8. SolerBotija C, et al. Brain. 2002;125(7):1624-1634.9. Wang CH, et al. J Child Neurol. 2007;22(8):1027-1049.10. Darras BT, Finkel RS Spinal Muscular Atrophy. Chapter 25 - Natural History of Spinal Muscular Atrophy. October 2017.11. FDA approval: https://www.fda.gov/vaccines-blood-biologics/zolgensma

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AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA) - Yahoo Finance

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