StemCell Therapy

CAMBRIDGE, Mass. and NEW YORK, May 06, 2020 (GLOBE NEWSWIRE) -- Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of small molecule, tumor-agnostic therapies, today announced that its President and Chief Executive Officer, David M. Epstein, Ph.D., will present an update about the Companys business at the Bank of America Global Research Health Care Conference 2020. The presentation will take place on Tuesday, May 12, 2020, at 4:20 PM ET.

About Black DiamondBlack Diamond Therapeutics is a precision oncology medicine company pioneering the discovery of small molecule, tumor-agnostic therapies. Black Diamond targets undrugged mutations in patients with genetically defined cancers. Black Diamond is built upon a deep understanding of cancer genetics, protein structure and function, and medicinal chemistry. The Companys proprietary technology platform, Mutation-Allostery-Pharmacology, or MAP, platform, is designed to allow Black Diamond to analyze population-level genetic sequencing data to identify oncogenic mutations that promote cancer across tumor types, group these mutations into families, and develop a single small molecule therapy in a tumor-agnostic manner that targets a specific family of mutations. Black Diamond was founded by David M. Epstein, Ph.D., and Elizabeth Buck, Ph.D., and, beginning in 2017, together with Versant Ventures, began building the MAP platform and chemistry discovery engine. For more information, please visit http://www.blackdiamondtherapeutics.com.

Contacts:

For Investors:Natalie Wildenradtinvestors@bdtherapeutics.com

For Media:Kathy Vincent(310) 403-8951media@bdtherapeutics.com

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Black Diamond Therapeutics to Present at the Bank of America Global Research Health Care Conference 2020 - GlobeNewswire

FILE: Medical researchers perform serology, testing blood samples to find out whether someone already had and recovered from COVID-19, in Stanford University's Clinical Virology Lab in March 2020.

FILE: Medical researchers perform serology, testing blood samples to find out whether someone already had and recovered from COVID-19, in Stanford University's Clinical Virology Lab in March 2020.

Photo: Steve Fisch/Stanford Medicine

FILE: Medical researchers perform serology, testing blood samples to find out whether someone already had and recovered from COVID-19, in Stanford University's Clinical Virology Lab in March 2020.

FILE: Medical researchers perform serology, testing blood samples to find out whether someone already had and recovered from COVID-19, in Stanford University's Clinical Virology Lab in March 2020.

Why so many people are convinced that they had COVID-19 already

The week before Thanksgiving, Barbara O'Donnell came down with a wretched cough.

"It was just really bad, and it was constant," says O'Donnell, 62. "I would turn purple," gasping for breath. She could barely walk up the hills near her home outside of Philadelphia. Though she is a smoker, she was healthy and strong - "I don't get the flu, ever" - and had never experienced anything like this before.

It "felt like my lungs were so full that I wasn't going to make it," she says.

Two weeks of resting at home, and the illness vanished as quickly as it came. Two months later, California reported the country's first case of covid-19 that wasn't acquired via travel or direct contact with someone who had been abroad. Three weeks after that, Philadelphia closed nonessential businesses and issued a stay-at-home order. O'Donnell's job, as a privately employed aide for an elderly patient in a nursing home, was put on hold - the nursing home permitted only its own staff on the premises.

Sitting in her apartment, the thought occurred to her: "What if it was here way before they think it was?" she wondered. Was that cough covid?

The virus was here before anyone thought it was, we now know. Health officials in Santa Clara County, south of San Francisco, recently determined that at least two people who died in early and mid-February tested positive for the virus. But that doesn't answer the question that has been spreading, afflicting anyone who recently - or even kind of recently - experienced any covid symptoms:

Did I have it? I think I had it.

"I've been getting emails from hundreds, maybe thousands of people telling me, 'I'm sure I had it,' " Eran Bendavid, an associate professor of medicine specializing in infectious disease who is studying covid-19 at Stanford University.

"I am 99 percent sure I had it," says Janet Truchard, 58, who woke up in her Las Vegas home on Jan. 15 "sick as a dog" with a fever, dry cough, migraine and chest pain. She visited several doctors who prescribed various courses of antibiotics, diagnosing her with sinusitis and then allergies. The cough persisted all the way through March 25, but a chest X-ray came back clear.

Thinkihadititis is a condition afflicting people who experienced covid-like illnesses that befell them long before coronavirus was a thing. It happens when bits of news and scientific findings lodge in the parts of the brain that incubate hope - Oh hey, maybe I already beat it! - and anxiety - Oh God, maybe I gave it to a bunch of people.

Like covid-19, Thinkihadititis has infected some high-profile patients. "Sopranos" star Michael Imperioli told Page Six he was "certain" he caught the virus in early February. A star of the reality series "Love Island" thinks she "had the 'rona" while the show was filming in South Africa in January. And Patti Stanger, star of the Bravo show "Millionaire Matchmaker," was stricken with shallow breathing, a fever, fatigue and nausea after a January vacation in Miami, despite flying with a face mask at the advice of her nail technician. She had to skip the Grammys and had a panic attack when her fever spiked to 102.

"I didn't get up for three weeks," she says. "I didn't eat a thing. I lived on bone broth and crackers."

Later on, when news broke that the coronavirus had arrived in America earlier than we ever knew, "I thought, I could be one of those people," she says.

"I started hearing from all these friends saying, 'I think I had it, I think I had it.'"

- - -

No one wants to have covid-19, but everyone wants to have had it.

And recent research suggests that many people have already had it without knowing. Epidemiologists have said that the number of infections greatly exceeds the official count of cases, potentially by a factor of 10 or more, since people can be asymptomatic carriers of the illness, and because not every victim has been tested.

The World Health Organization has cautioned against the assumption that those who have already had the illness can't get it again. Researchers are still learning about the protective benefits that the disease's antibodies might bestow on survivors.

But after two long months of bad news about the painful effects and unpredictable deadliness of covid-19 - the sudden crashes, the mysterious strokes, the wide-ranging attacks on the body - who could be blamed for wondering, optimistically, about whether they've already joined the ranks of the Recovered?

JoAnna Fischer is sure she had covid-19. She lost her sense of smell, and for three months, she had a cough and chest pain so bad she needed supplemental oxygen. Her husband came down with a respiratory illness, and so did her cat.

One problem: Fischer fell ill all the way back in September, when she was living in northeast Pennsylvania. That's far earlier than epidemiologists believe the disease could have come to the United States.

"There is 0.0% probability that #SARSCoV2 was circulating with community transmission in the US in or before Nov 2019," tweeted Trevor Bedford, a computational biologist at Fred Hutchinson Cancer Research Center who has been tracking the virus' genetic code and spread.

Fischer isn't willing to give up her theory. "When you're thinking about how this thing spread so fast," the 63-year-old says, "it couldn't have just gotten here in December."

Bendavid, the Stanford professor, said one person who wrote to him believed they caught the virus in 2018. "That, I think, is stretching it," says Bendavid.

Thinkihadititis usually involves stretching the imagination to some degree. After all, covid-19 shares some symptoms with the seasonal flu and common allergies. Currently fewer than 20 percent of covid-19 tests are coming back positive, according to data reported to the Centers of Disease Control and Prevention, suggesting that the great majority of people who thought they had it - even in the middle of the pandemic - didn't actually have it.

To the extent that having already beat covid-19 is preferable to wondering if you're one of the people it's going to put in the hospital, Thinkihadititis may be a form of positive thinking. Humans are hardwired to anticipate positive outcomes, says Tali Sharot, a professor of cognitive neuroscience at the University College London who studies optimism and expectations.

"When there is something we want to believe, we are very good at interpreting the evidence in a way that would support that belief," says Sharot.

The reverse is also true. "Say there was a doctor saying that if you had it before, then the likelihood that you would get it again is higher, and it would be even more dangerous," says the professor. If that were the case, people "would probably look back to their illnesses and interpret certain symptoms as definitely not covid-19."

Whether it's a comfort or a source of anxiety, Thinkihadititis represents a state of limbo.

The good news? There's a cure.

Kind of.

- - -

"The only way to know is to get an antibody test," says Rachael Ayscue. "And I don't know anywhere around here that'll give one."

Ayscue, 47, lives in the suburbs of Raleigh, North Carolina. On Jan. 6, she felt sick, and before long she was coughing so much that it sometimes hurt to breathe. Then her daughter got sick, too. Raleigh didn't see its first confirmed covid case until March 3, but Ayscue wondered whether the tech workers of the Research Triangle had traveled to Asia over the holidays, and brought the virus back. (Epidemiologists believe the earliest American cases originated in Europe, not Asia.)

Antibody tests may provide relief from the fever of doubt. Also known as serology tests, they determine whether a patient's blood contains antibodies, which are proteins that help us fight off infection. The presence of antibodies means the patient's immune system has already been exposed to the virus.

Those tests are now becoming available nationwide, although experts warn that their accuracy can vary.

Since it began offering appointments for antibody tests, the telemedicine provider PlushCare saw "a pretty overwhelming response (from) people who are interested," especially in harder-hit areas like New York, says James Wantuck, the platform's chief medical officer and co-founder.

PlushCare's doctors remind patients that immunity is not a given and that they must continue social distancing and other protective measures. And if the tests come back negative, some patients - confident in their self-diagnosis and wary of possible testing inaccuracies - might not believe them.

"Some of the patients are certainly disappointed," says Wantuck. "I think everyone wants to have this in their rearview mirror or feel some sense of relief."

Fischer, the woman who got sick last September, says she couldn't be convinced so easily. "If I get tested," she says, "and I don't have the antibodies, I think I would ask for a different test."

Ayscue says she plans to get an antibody test if she can, but she doesn't need one to feel comfortable returning to life as usual. She does not hesitate to go to the grocery store, sometimes without a mask. She says she supports the protesters who have demonstrated against strict lockdown rules in North Carolina.

"I've chosen not to be afraid," she says.

Read more:
Why so many people are convinced that they had COVID-19 already - Chron.com

Middlefield Dr. Heng Wang, a board certified pediatrician and the Medical Director of the DDC Clinic located in Middlefield, OH, announced today that their Amish Genetic Disease Panel will now test for 160 rare conditions. This important tool allows faster diagnosis of unidentified genetic conditions at a reduced cost. DDC Clinic delivers personalized and life-changing medical care to special needs children affected by rare genetic disorders.

Were very excited to be able to now screen for an additional 40 conditions as part of this panel, said Dr. Wang. Early diagnosis leads to early treatments, and those effective treatments can be lifesaving.

Dr. Wang credited the hard work of his staff with bringing this project to fruition months ahead of schedule. This project was partially funded by the Elisabeth Severance Prentiss Foundation and the Fowler Family Foundation. Their support allows us to keep the cost of the new panel the same as the previous panel.

The improved Amish Genetic Disease Panel will be used in DDC Clinics collaboration with the Care Center in Middlefield. Parents of newborn infants at the birthing center can have a sample of their babys cord blood sent to DDC Clinic for analysis. Parents would then know if their child is affected by any of these 160 rare conditions. If a condition is identified, no additional testing would be needed and support services could be initiated early in the babys life. Thanks to our generous donors and the United Way of Geauga County, parents are asked to pay only $25.00 for this testing as part of their birthing fee.

The Amish Genetic Disease Panel has proven to be an important tool and is a great example of personalized medicine in action. It is a resource for doctors serving Amish and Mennonite patients in both Geauga County and neighboring communities.

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Amish Genetic Disease Panel created by DDC Clinic can now test for 160 rare conditions - The Weekly Villager

While researchers are working to advance drugs to treat COVID-19 and vaccines to give people immunity against the virus, the mental health impact of the pandemic will also have to be managed. This is where PGx testing may be most useful, experts in the field said.

"It is worthwhile to consider not just the utility of PGx in preventing hospitalization or changing the course of COVID-19 care," but also the impact it could have on managing "the burden on the patients that do survive a COVID-19 infection [and] those that are suffering from the isolation of social distancing, as well as the financial hardships," said David Thacker, a clinical pharmacogenetics content specialist at Translational Software.

According to a recent JAMA editorial, during the SARS outbreak in 2003, there was a greater incidence of post-traumatic stress syndrome and psychological distress among patients and doctors. In communities impacted by Hurricane Ike in 2008, around 5% of individuals met the criteria for major depressive disorder, while one in 10 adults in New York City had symptoms of the disorder after 9/11.

"In the context of the COVID-19 pandemic, it appears likely that there will be substantial increases in anxiety and depression, substance use, loneliness, and domestic violence; and with schools closed, there is a very real possibility of an epidemic of child abuse," wrote Sandro Galea from Boston University School of Public Health, Raina Merchant from the Perelman School of Medicine, and Nicole Lurie from the Coalition for Epidemic Preparedness Innovations in Norway.

A survey in March by the American Psychiatric Association found that more than a third of polled individuals said that the pandemic was seriously impacting their mental health, nearly half said they were scared about getting the virus, and 62% said they feared a loved one would get it. Meanwhile, calls to substance abuse and mental health help lines increased eightfold from February to March.

As the pandemic continues, people may increasingly turn to medications to deal with the psychological wounds left by the pandemic. Drugs to treat mental health conditions, including major depressive disorder, are some of the most widely prescribed drugs in the U.S., but they're also highly variable and associated with unwanted side effects.

As such, one of the main areas where PGx testing has seen uptake is for personalizing psychiatry drugs. Myriad Genetics recently published a meta-analysis involving more than 1,500 patients with major depressive disorder who were enrolled in four studies, which showed that patients who received treatment based on PGx information had significantly better outcomes than those who did not.

Although PGx testing in psychiatry is not without its naysayers, doctors may reach for such testing if the use of mental health drugs increases during or after the pandemic. Genomind, a mental health-focused PGx testing company, recently took a number of steps to make it easier for physicians to deliver psychiatric care during the pandemic. Doctors can order Genomind's PGx test and send a saliva collection kit to patient's homes, which can then be mailed to the lab for analysis. Through Genomind, doctors also have access to Sharecare's HIPAA-compliant telemedicine platform for free until September, which they can use to remotely see patients and discuss PGx test results, if ordered.

"The utility of PGx during the COVID-19 crisis is more important than ever," a spokesperson for the company said. "This service is helping enable critical mental health treatment during the pandemic and Genomind is doing its best to enable as many mental health professionals as possible."

This story first appeared in our sister publication, Genomeweb.

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Drug-gene testing could give experts insight into COVID-19 treatment - ModernHealthcare.com

For a few years now, scientists at Washington University have been working on techniques to turn stem cells into pancreatic beta cells as a way of addressing insulin shortages in diabetics. After some promising recent strides, the team is now reporting another exciting breakthrough, combining this technique with the CRISPR gene-editing tool to reverse the disease in mice.

The pancreas contains what are known as beta cells, which secrete insulin as a way of tempering spikes in blood-sugar levels. But in those with diabetes, these beta cells either die off or dont function as they should, which means sufferers have to rely on diet and or regular insulin injections to manage their blood-sugar levels instead.

One of the ways scientists are working to replenish these stocks of pancreatic beta cells is by making them out of human stem cells, which are versatile, blank slate-like cells that can mature into almost any type of cell in the human body. The Washington University team has operated at the vanguard of this technology with a number of key breakthroughs, most recently with a cell implantation technique that functionally cured mice with diabetes.

The researchers are continuing to press ahead in search of new and improved methods, and this led them to the CRISPR gene-editing system, which itself has shown real promise as a tool to treat diabetes. The hope was that CRISPR could be used to correct genetic defects leading to diabetes, combining with the stem cell therapy to produce even more effective results.

As a proof of concept, the scientists took skin cells from a patient with a rare genetic type of diabetes called Wolfram syndrome, which develops during childhood and typically involves multiple insulin injections each day. These skin cells were converted into induced pluripotent stem cells, which were in turn converted into insulin-secreting beta cells. But as an additional step, CRISPR was used to correct a genetic mutation that causes Wolfram syndrome.

These edited beta cells were then pitted against non-edited beta cells from the same batch in test tube experiments and in mice with a severe type of diabetes. The edited cells proved more efficient at secreting insulin and when implanted under the skin in mice, reportedly caused the diabetes to quickly disappear. The rodents that received the unedited beta cells remained diabetic.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigator Jeffrey R. Millman. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

The researchers are now continuing to work on improving the beta cell production technique, which in the future could involve cells taken form the blood or even urine, rather than the skin. They believe that further down the track this therapy could prove useful in treating both type 1 and type 2 diabetes, by correcting mutations that arise from genetic and environmental factors, and possibly be used to treat other conditions, as well.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigator Fumihiko Urano. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

The research was published in the journal Science Translational Medicine.

Source: Washington University

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CRISPR combines with stem cell therapy to reverse diabetes in mice - New Atlas

TBTAK has invested TL 2.3 billion ($ 300 million) for the development of 16 vaccine and other medicine projects over the past five years in Turkey, the president of the Scientific and Technological Research Council of Turkey (TBTAK) said Saturday.

A platform consisting of 41 Turkish institutions has mobilized to develop medicines and vaccines against COVID-19 pandemic, professor Hasan Mandal noted, adding that efforts to develop anti-coronavirus drugs and vaccines were launched late December in coordination with the Ministry of Industry and Technology and proposals to be carried out within a 9 to 12 month period were taken into account and evaluated.

There are 16 projects working in synergy both in medicine and vaccine groups thanks to the COVID-19 Turkey Platform, which includes 225 researchers from 25 universities, eight public research bodies and eight private firms, Mandal added.

For medicine in pre-clinical phases, we'll be in the production phase this summer, probably much earlier. For vaccine, pre-clinical phase will be completed within a nine-month period, Mandal said. He added that it is now time for institutions cooperating with the science body to prove their valor.

The modeling of molecules in the medicine group began with the identification of over 10,026 molecules that could provide a solution for this virus, Mandal explained, adding synthesis works regarding this and its production are now underway.

In order to fight with a virus that you recently came across, you have to know it and define it correctly. All proceeding phases are tied to this. For that, it should be isolated from all external conditions and genetic characterization of the virus should be carried out. We will have genetic characterization of this virus determined next week. This situation shows the competence of this country. We now know this virus and this will be among the most important indicators how we will fight it both on the medicine side and vaccine side, Mandal said.

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Turkey's top scientific body invests TL 2.3 billion on 16 vaccine projects over 5 years | Daily Sabah - Daily Sabah

Researchers identify molecular markers that can help in developing better varieties of black pepper

Dubbed as the 'king of spices', black pepper has a lot to offerwhether as a flavouring agent or as a traditional medicine to relieve common cold, chest congestion or sore throat. A vital ingredient in many cuisines, it is the only spice that finds its way on nearly everything on the dining table. Piperine, a chemical in the black pepper, gives it a sharp taste. It is a native plant of the Western Ghats, and the crop is of immense agronomic value in today's world trade. However, insufficient genomic resources pose as main barriers in cultivating disease-resistant varieties of this master spice.

In a recent study, researchers from ICAR-National Bureau of Plant Genetic Resources, New Delhi, have identified a type of DNA sequence, called Simple Sequence Repeats (SSRs), which aims to ease and promote the genetic analysis of black pepper. The findings of the study, published in the journal PLoS ONE, could lead to enhanced productivity with better traits of the plant.

Simple Sequence Repeats, also called microsatellites, are repeating stretches of DNA that contain one to six nucleotide bases, and are randomly present throughout the genome. These can be passed from one generation to the next. Hence, they are used as a 'signpost' to keep track of a gene of interest. They play critical roles in genetic studies and plant breeding. The location of these SSRs in the genome remain the same in related species, thus enabling cost-effectiveness of similar studies on them.

Unlike other commercial crops like watermelon, cotton or finger millets, very few genetic studies have been done on black pepper, due to lack of resources. Although the Western Ghats harbours the maximum genetic diversity of black pepper, it remained "largely untouched from genomic interventions," say the researchers. The identification of cross-species SSRs will save time, effort and resources in the development of SSRs in these species, and aid future genetic and evolutionary studies.

The researchers first sequenced the entire genome isolated from the leaf samples of black pepper (Piper nigrum) and then scanned it for the presence of SSRs. The genome was then amplified with a method called Polymerase Chain Reaction, using short nucleic acid sequences called primers, designed to identify the right SSRs. The researchers compared the results of their analysis in thirty different types of black pepper. The researchers also explored ten species of Piper (including P. nigrum) for the presence of these SSRs by looking at an online database of the genetic sequence.

In all, 69,126 SSRs were identified in the study, with the majority of SSRs composed of two nucleotide repeatsthymine and adenosine (TA). Out of 85 primers, 74 produced the required results of appropriate-sized SSRs. Genetic diversity study of the thirty cultivars reported four distinct groups. A few SSRs were found in closely-related species, implying that they could be used in studying other species of black pepper with limited genomic knowledge.

The current study seeks to fill the void in genomic knowledge of black pepper species by identifying easy-to-detect molecular markers to enable its genetic and breeding studies. The SSRs, which indicate the location of a gene related to the desired trait, can help in choosing the right plants for breeding experiments, which is otherwise a difficult activity.

"The genomic microsatellite markers identified in black pepper in this study would form valuable and long-awaited resources for researchers and plant breeders", say the researchers.

The findings can also be used for diversity studies, linkage mapping, evolutionally biology, DNA fingerprinting and trait association shortly.

Originally posted here:
Paparazzi on the black pepper - Research Matters

As many people yearn to return to some form of normalcy, states are beginning to consider what the reopening of nonessential businesses should look like. In his daily press briefing Gov. Andrew Cuomo (D-N.Y.) said a crucial first step for reopening is widespread COVID-19 testing which New York State currently lacks.

On that same day, Dr. Augustine M.K. Choi, Weill Cornell Dean, announced a new initiative to begin antibody testing employees of Weill Cornell.

Current testing efforts across the state are focused on detecting those with the SARS-CoV-2 virus, but in order to begin reopening businesses people must be tested for previous exposure to the virus.

The current diagnostic used to test patients suspected of having COVID-19 at WCM is a real time reverse transcription polymerase chain reaction, an effective and relatively fast method to detect genetic material. It can be used to detect the RNA present in the SARS-CoV-2 virus.

PCR is the gold standard because its such a highly sensitive and specific test and can deliver reliable and accurate diagnosis in as fast as 2-5 hours. Compared to other available platforms its much faster and more accurate, said Dr. Melissa Cushing, pathology, in Chois update.

However, as institutions begin to test for people who were exposed to the virus and recovered, another method is required antibody testing. Instead of testing for the genetic material of the virus itself, antibody tests search for the antibodies that the body creates in response to COVID-19. These antibodies are formed between three and 15 days after experiencing symptoms, according to Cushing.

As of April 17, testing was made available for New York Presbyterian staff that tested positive for COVID-19 or had a COVID-19-like illness and returned to work.

WCM plans to make more testing available to its staff, as it works to increase its testing capabilities. Cushing predicted that this public testing is at least several weeks away. Experiencing the brunt of statewide shortages of certain materials, WCM also requires access to reagents and more high output platforms to increase its testing capabilities.

We need to really scale up with the amount of reagents we have with our current tests. Then we are really looking to some of the commercial labs to provide the large, high frequency platforms that we already use in our labs so that the process can be much more automated, Cushing said. That is our goal to be testing as many people that need to be tested in our city.

In order to address the testing insufficiencies on a statewide level, the governor issued an executive order on April 17 that directs all public and private labs capable of conducting virology testing to coordinate with the State Department of Health to prioritize coronavirus testing.

The testing and tracing is the guideposts through this. As we are working our way through the next several months the testing, which is informing us as to who can go back to work helping us isolate people, its about testing, Cuomo said in his daily briefing on April 17. Testing is a totally new challenge. Nobody has done this and what we need to do on testing.

According to Cuomo, the lack of infrastructure to facilitate widespread testing mirrors the earlier lack of coordination between hospitals, which the Surge and Flex initiative addressed the initiative coordinated the distribution of scarce medical supplies between public and private hospitals across the state.

Besides the lack of infrastructure, another impasse to wide scale testing is the availability of the materials specifically chemical reagents necessary to run the tests.

Currently, this order will not affect the labs on Cornells Ithaca campus.

Cornell University is not offering any human testing for COVID-19 on campus at this point. We will always follow all state/federal government regulations as appropriate, John Carberry, a University spokesperson, wrote in a statement to The Sun.

Cornell is affiliated with two of the 301 laboratories and hospitals capable of performing viral testing the Allyn B Ley Clinical Laboratory housed in Cornell Health and the Hospital for Special Surgery Dept of Pathology and Laboratory Medicine in New York City.

Initially, 28 laboratories with clinical laboratory permits from the state health department and experience in molecular-based virology could conduct testing. However, this system is unable to meet the demand for the widespread testing needed to reopen New York State.

We dont have a testing system that can do this volume, or that can be ramped up to do this volume. We dont have a public health testing system, its de minimis if you look at what our government department of health have, Cuomo said.

The state has begun its efforts to perform antibody tests on 3,000 individuals to better understand what percentage of the population is currently immune to the virus. The plan is being supported financially by former New York City mayor Michael Bloomberg, who pledged more than $10 million to create a test and trace program.

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As Cuomo Issues New Executive Order, Weill Cornell Medicine Ramps Up COVID-19 Testing - Cornell University The Cornell Daily Sun

Many people dislike drug companies. Drug companies often charge a lot of money for their products. Sometimes their drugs dont work. Sometimes they have side effects. Sometimes they are addictive. Even the best drugs wont keep us alive forever.

But, during the coronavirus pandemic, we are relying on drug companies and other healthcare companies to save our lives and the lives of our loved ones.

While government plays a vital role in managing the response to the COVID-19 pandemic, its the private sector that will provide the solutions to identify and fight the disease. Who would you rather depend on profit-making private companies or bureaucratic government agencies?

The CDC response

In the United States, the Centers for Disease Control, a federal agency, was initially tasked with providing tests for COVID-19. Its test kits could not detect the difference between COVID-19 and lab-grade water.

The CDC also limited testing to patients who had recently traveled to China and were symptomatic. Whoops!

The lack of reliable test kits enabled the disease to spread throughout the United States. The U.S. Food and Drug Administration determined on Feb. 29 that certified labs, including commercial lab testing companies, could develop and distribute COVID-19 test kits.

Private companies and clinics stepped up quickly. Some examples:

The Cleveland Clinic developed an eight-hour test kit.

Hackensack Meridian Health developed a rapid response test kit that can provide results within a few hours.

On March 23, Everlywell became the first company to offer a test kit that consumers can use at home.

Roche shipped 400,000 test kits to labs across the United States beginning March 13, and an additional 400,000 the following week.

Thermo Fisher Scientific developed a test kit that can detect COVID-19 within four hours.

Drug companies in the United States and throughout the world are also working to develop a vaccine to fight COVID-19.

Cambridge-based Moderna Inc. has already begun Phase 1 testing of its mRNA-1273 vaccine that is based on the genetic sequence of COVID-19. Regeneron Pharmaceuticals of Tarrytown, New York, is working on an antibody treatment that uses the virus to build up antibodies that fight COVID-19. Inovio Pharmaceuticals of Plymouth Meeting, Pennsylvania., is developing INO-4800, a vaccine similar to Modernas that is made from optimized DNA plasmids.

But thats just a sample. At least 16 U.S. pharmaceutical companies are working on vaccines for COVID-19.

The government has been doing its part. President Trump declared a national emergency and Congress has been negotiating a massive spending bill, while the Federal Reserve Board dropped interest rates back to zero, resumed bond buying and provided liquidity to the banking system. None of these actions reassured investors and stopped the stock market from tanking. Recall that during the financial crisis, the Obama Administration invested more than $800 billion in a stimulus that stimulated the federal debt, but not the economy.

Communism vs. capitalism

While the government and private sector have both been working hard to battle the coronavirus, the Chinese government has been working hard to preserve the Communist Party by blaming the United States for the pandemic.

When Wuhan doctor Li Wenliang warned his colleagues in late December about a possible coronavirus that resembled SARS, local police reprimanded him for spreading rumors and he was called before a disciplinary council of the local Communist Party and forced to repent and confess, in writing, that he had spread rumors harmful to the glory of the Party, according to City Journal.

In early January, news of the virus started circulating on Chinese social media accounts. The government responded by shutting them down. By silencing news about the outbreak of COVID-19, Communist leaders allowed it to spread, resulting in the pandemic and thousands of deaths that could have been avoided.

Xu Zhiyong, an activist who criticized Chinese President Xi Jinpings response to the coronavirus, was jailed for subversion. Journalists Li Xehua, Fang Bin and Chen Qiushi, who tried to inform the public about the coronavirus outbreak, are all missing. And Western journalists who brought these stories to the worlds attention have been expelled from China.

Wuhan activists, professors and lawyers who had asked for President Xis resignation have all virtually disappeared. In addition to stifling the news at home, Chinas leaders failed to inform other countries about the severity of the coronavirus or to prevent its spread outside of China.

Should China be paying reparations to the rest of the world? Should it at least admit its guilt and apologize? According to an editorial in state-run media agency Xinhua, We should say righteously that the U.S. owes China an apology, the world owes China a thank you.

In addition, while the United States has shipped medical supplies to China to help fight the outbreak, China is threatening to impose export controls on pharmaceuticals needed by the U.S. to fight COVID-19.

In an article in Xinhua, Beijing bragged about its handling of COVID-19, according to Fox News. The article also claimed that China could impose pharmaceutical export controls which would plunge America into the mighty sea of coronavirus.

Without disclosing which drug or drugs are in short supply, the U.S. Food and Drug Administration announced a shortage of raw materials made in China that are needed to produce the drug.

While the United States is the world leader in medical research, China supplies 80% to 90% of antibiotics used in the United States, 70% of acetaminophen and about 40% of heparin, according to Yanzhong Huang, a senior fellow for global health at the Council on Foreign Relations.

Government vs. private sector

In other countries where health care is socialized, such as Italy and the United Kingdom, government responses have been underwhelming and politically charged.

Some 4,032 have died in Italy, more than in China, with 627 Italian deaths reported Friday (March 20) the highest daily toll for any country so far, according to The Wall Street Journal.

Which brings us back to the United States, where many Americans believe we would be better off with a socialized healthcare system. After all, many other countries have socialized medicine.

Some believe we should have price controls on drugs. Others believe that drug companies should not make profits and should make life-saving drugs available at no cost.

That belief would lead to no more life-savings drugs being developed, because there would be no incentive for companies to do so. Even if companies wanted to be able to give away free drugs, doing so would quickly put them out of business. Jobs would be eliminated and their stock would become worthless.

The average cost to develop a new drug is $2.6 billion. Post-approval research and development costs, such as the cost of monitoring effectiveness and safety, average $312 million and boost the total cost for each approved drug to almost $3 billion.

Most drugs never make it to market. Only about 12% of drugs that enter clinical testing are eventually approved for public use.

Some drug companies have made a great deal of money. Some have taken advantage of the drug-approval process and charged outrageous prices for their products.

Weve all heard about companies like Theranos, which achieved a valuation of $9 billion based on fraudulent blood-testing technology, or Mylans price gouging for its EpiPen. Companies in any industry that take advantage of their customers usually pay in the end, but in such cases, government involvement is necessary.

Regardless, even with no vaccine yet available, special interest groups that pretend that their interest is our interest are calling for President Trump to prohibit profiteering by Big Pharma during the coronavirus pandemic.

If Big Pharma develops a vaccine that works, the company that does so should make a significant profit. Is there a sector more deserving of a profit than one that saves lives? While small businesses are suffering most during the pandemic, large companies have also had to deal with major losses, a huge drop in their stock price and mounting expenses in an attempt to staunch the bleeding.

In 1900, 37% of all American deaths were from infectious diseases, George Will wrote. Today the figure is 2%.

Id rather the drug companies get a cut of my hard-earned money than the more than 70 groups that are trying to stop them from earning a profit. To my knowledge, none of the 70 groups has criticized President Xi.

Brenda P. Wenning of Newton is president of Wenning Investments LLC in Newton. She can be reached at Brenda@WenningInvestments.com or 617-965-0680. For additional information, visit her blog at http://www.WenningAdvice.com.

Link:
Drug companies are the good guys - MetroWest Daily News

CLAIM

"this coronavirus genome contained sequences of another virus [] the HIV virus (AIDS virus)"

DETAILS

Inaccurate: Genomic analyses indicate that the virus has a natural origin, and was not engineered. The so-called unique protein sequence insertions found in the 2019 novel coronavirus can be found in many other organisms, not just HIV.

KEY TAKE AWAY

Genomic analyses of the novel coronavirus show that it was not engineered. In addition, the claim that its genome contains inserted HIV sequences is based on a now-withdrawn preprint of a study that contained significant flaws in design and execution. The so-called HIV insertions identified by the authors are in fact gene sequences that can also be found in many other organisms besides HIV.

REVIEW Numerous articles published in April 2020 report that Nobel laureate Luc Montagnier claimed that SARS-CoV-2 is a manipulated virus that was accidentally released from a laboratory in Wuhan, China and that Indian researchers have already tried to publish the results of the analyses that showed that this coronavirus genome contained sequences of another virus [] the HIV virus (AIDS virus). The claim that SARS-CoV-2 contains HIV insertions began circulating in January 2020, and was propagated by outlets such as Zero Hedge and Infowars. Health Feedback covered this claim in early February 2020, and found it to be inaccurate.

Firstly, genomic analysis of the novel coronavirus, published in Nature Medicine, has demonstrated that the virus is not the product of bioengineering, but is rather of natural origin[1]. The current most likely theory, based on what scientists know about viral evolution, is that the virus first emerged in pangolins or bats (or both) and later developed the ability to infect humans. This ability to infect human cells is conferred by the so-called spike (S) protein, which is located on the surface of the enveloping membrane of SARS-CoV-2.

After the 2003-2005 SARS outbreak, researchers identified a set of key amino acids within the S protein which give SARS-CoV-1 a super-affinity for the ACE2 target receptor located on the surface of human cells[2,3]. Surprisingly, the S protein of the current SARS-CoV-2 does not contain this optimal set of amino acids[1], yet is nonetheless able to bind ACE2 with a greater affinity than SARS-CoV-1[4]. This finding suggests that SARS-CoV-2 evolved independently and undermines the claim that it was manmade[1]. Indeed, the best engineering strategy would have been to harness the known and efficient amino acid sequences already described in SARS-CoV-1 order to produce a more optimal molecular design for SARS-CoV-2. The authors of the Nature Medicine study[1] concluded that Our analyses clearly show that SARS-CoV-2 is not a laboratory construct or a purposefully manipulated virus.

Secondly, the claim that SARS-CoV-2 contains HIV insertions is based on a preprint of a research study uploaded to bioRxiv on 2 February 2020. A preprint is a study in progress that has not been peer-reviewed by other scientists. The authors of the preprint, titled Uncanny similarity of unique inserts in the 2019-nCoV spike protein to HIV-1 gp120 and Gag, claimed to have found 4 insertions in the spike glycoprotein (S) which are unique to 2019-nCoV and are not present in other coronaviruses. The authors further asserted that all of [these inserts] have identity/similarity to amino acids residues in key structural proteins of HIV-1 [which] is unlikely to be fortuitous in nature.

The work was swiftly criticized by experts. In this Forbes article, Arinjay Banerjee, a postdoctoral fellow at McMaster University who has studied coronaviruses, said that:

The authors compared very short regions of proteins in the novel coronavirus and concluded that the small segments of proteins were similar to segments in HIV proteins. Comparing very short segments can often generate false positives and it is difficult to make these conclusions using small protein segments.

Researchers also took to Twitter to demonstrate this problem first-hand. Trevor Bedford, a faculty member at the Fred Hutchinson Cancer Research Center who studies viral evolution, re-analyzed the gene and protein sequences used by the authors and found that the so-called unique inserts appeared in many other organisms, including Cryptosporidium and Plasmodium malariae, which cause cryptosporidiosis and malaria, respectively.

Assistant professor at Stanford University Silvana Konermann also checked the authors findings and came to the same conclusion, calling the similarity spurious.

This has also been independently confirmed in another published analysis[5]. In other words, these sequences are not insertions, but are rather common sequences found in numerous other organisms such as bacteria and parasites. Therefore, the existence of these sequences in SARS-CoV-2 does not provide evidence of a link to HIV, nor that scientists purposely inserted HIV sequences into the SARS-CoV-2 genome.

In summary, genomic analysis of the virus indicates that it does not contain so-called HIV insertions and that it was not engineered in a lab. Evidence points to the virus having a natural origin.

The only thing accurate about these articles is that Nobel Prize winner and virologist Luc Montagnier did in fact make these claims. Although he holds impressive scientific credentials, his claims run contrary to credible scientific evidence. And despite having won the Nobel Prize in Physiology or Medicine in 2008 for his co-discovery of the link between HIV and AIDS, Montagnier now promotes widely discredited theories such as the pseudoscience of homeopathy and that autism is caused by bacteria that emit electromagnetic waves. Articles which repeat Montagniers claims without critically evaluating their veracity exhibit the common appeal to authority fallacy, in which something is assumed to be true simply because the person saying it is considered to be an expert, thereby misleading readers into believing that this theory is scientifically credible. This demonstrates the importance of verifying scientific claims with other experts in the same field, rather than simply taking such claims from a single expert at face value.

SCIENTISTS FEEDBACK [These comments come from an evaluation of a related claim.] Aaron T. Irving, Senior Research Fellow, Duke-NUS Medical School:Its easier to believe misinformation when it is mixed with truth. The region highlighted in the pre-print is indeed an insertion in nCoV-2019 relative to its bat ancestors and indeed it has high identity to the HIV gp120/gag. However, the authors chose to align only this small region and not do a basic check on whether there were other sequences which were also homologous (showing high degree of similarity/identity). As it turned out, the region is also homologous to many unrelated sequences. As such, the conclusions drawn from the data are no longer valid and there are many open-ended questions regarding this region highlighted. I see the authors themselves agree with this criticism by other scientists and have voluntarily withdrawn their preprint pending a much deeper investigation.

The author of this article by European Scientist also compared the genome sequences of SARS-CoV-2 and HIV using the Basic Local Alignment Search Tool (BLAST), developed by the U.S. National Institutes of Health, and found no significant similarity, explaining that In plain English, SARS-CoV-2 is not made of the bat coronavirus and small bits of the HIV virus. Readers who wish to verify the level of sequence identity between the two viruses for themselves are welcome to follow the steps listed in the article.

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Nobel laureate Luc Montagnier inaccurately claims that the novel coronavirus is man-made and contains genetic material from HIV - Health Feedback

So when does the world get back to normal? It is a question that is on the mind of half of the worlds 7.8 billion people who have been asked to stay put at home to avoid a virus that has made millions sick. Not until a good vaccine is developed, Dr Anthony Fauci, Americas top infectious diseases expert, told reporters recently at a White House press briefing.

In normal times, experts say developing an effective vaccine would take anywhere between five and 10 years. But these are unprecedented times, and even scientists are being compelled to find newer ways to develop and test vaccines. Best-case scenarios for vaccine development have already shrunk the time frame to 12 to 18 months; globally, more than 100 vaccine candidates are at different stages of development. Five of these have reached the Phase 1 trials, and 18 are in pre-clinical stage, said Dr Shahid Jameel, virologist and CEO, Wellcome Trust/DBT India Alliance.

Closer home, Indian scientists, researchers and vaccine manufacturers are racing ahead, after Prime Minister Narendra Modi, in his speech on April 14, asked Indias young scientists and researchers to come forward to work on a vaccine against Covid-19.

For those working on vaccines, what has helped, said Premas Biotech cofounder and managing director Prabuddha Kundu, is that regulatory pathways are being fast-tracked and regulators are now willing to consider new scientific processes, and collaborations are happening.

Bharat Biotech is collaborating with virologists at the University of Wisconsin-Madison and vaccine company FluGen on a unique intranasal vaccine called CoroFlu.

For a country that is a leading manufacturer and supplier of vaccines, India definitely has some advantages in building one. According to the department of biotechnology, the government body that is leading the hunt for the vaccine development, four major companiesSerum Institute of India, Zydus Cadila, Bharat Biotech, Biological E Ltdhave a candidate each, besides academic research groups from the Indian Institute of Science, International Centre for Genetic Engineering and Biotechnology, National Institute of Immunology, and Translational Health Science and Technology Institute.

Vaccines are built by taking different approaches, said Jameel. They can either use inactivated viruses (for instance, the injectable polio vaccine); or live attenuated or weakened virus (the oral polio vaccine built in the 1950s) that is weakened to the point that it infects and multiplies, but does not cause disease; or subunit vaccines, where you take a part of the virus (protein), produce en masse and purify it and use it as a vaccine. The idea is to choose the antigens that best stimulate the immune system. One method of production involves isolating the specific protein from the virus or producing it using recombinant DNA technology and then administering it on its own. This reduces the likelihood of adverse reactions to the vaccine. The hepatitis-B vaccine is one example of that approach, said Jameel. Besides, vaccines being developed are also either based on DNA, RNA, or vector vaccine-based approaches.

The selection of an antigen and the antigenic design of the potential candidate will have a profound effect in generating an effective immune response. Since spike protein of the SARS-CoV-2 is a potential target, one will need to decide whether full-length spike glycoprotein or part of the protein that binds with the receptor needs to be selected, said Professor Sunit K. Singh, head, molecular biology unit, faculty of medicine, Institute of Medical Sciences, Banaras Hindu University.

For now, Premas Biotech seems to have worked around some of those issues. The Gurugram-based company, in collaboration with US-based Akers Biosciences, is working on its vaccine candidate using a mixture of three antigens produced in bakers yeast. Its cofounder and managing director, Prabuddha Kundu, said that traditionally vaccines were made by injecting heat-killed or attenuated whole viruses or bacteria, but since that had side effects, the approach of late has been to take a part of surface proteins, purify and produce it recombinantly (by rearranging genetic material) to elicit an antibody response.

In the case of SARS-CoV-2, one of the top targets is the spike protein present on the outer surface of the virus, and is understood to be the weapon with which it binds to the human cells (receptors) and gains entry. But since there were concerns about mutations in spike proteins, Kundu said that his team had created a mixture of the spike protein and two other proteins found on the outer membrane of the virus. These, said Kundu, would be replicated on its genetically engineered platform of bakers yeast (D-CryptTM). The platform has worked in the past, tooit has been successful in expressing 30 proteins similar to those in the structure of the selected Covid-19 antigens. It is also safer, and cost-effective, said Kundu, adding that the company has applied for animal trials with the Review Committee on Genetic Manipulation.

In Hyderabad, Bharat Biotech is collaborating with virologists at the University of Wisconsin (UW)-Madison and vaccine company FluGen on a unique intranasal vaccine called CoroFlu. The new vaccine is being built on the backbone of the trios flu vaccine candidate known as M2SR, developed a couple of years ago. M2SR is a self-limiting version of the influenza virus that induces an immune response against the flu, said Dr Krishna Ella, chairman and managing director, Bharat Biotech. [FluGen cofounder Yoshihiro] Kawaokas lab will insert gene sequences from SARS-CoV-2 into M2SR so that the new vaccine will also induce immunity against the novel coronavirus.

CoroFlus safety and efficacy in animal models is being assessed at the UW-Madisons influenza research institute, said Ella, which could take four to six months. Post the results of animal trials, which is crucial, Bharat Biotech will begin production scale-up for safety and efficacy testing in humans. CoroFlu could be in human clinical trials by the fall of 2020, he said.

Bharat Biotech is also working on a second candidate that will utilise the inactivated rabies vector platform, for which funding has been approved by the department of biotechnology. The department has also recommended funding support to Ahmedabad-based Zydus Cadila for advancing the development of a DNA vaccine candidate, as well as Phase 3 human clinical trials for recombinant BCG vaccine (VPM1002) planned in high-risk population by the Pune-based Serum Institute of India (SII). SII is also testing its vaccine candidate (in collaboration with US-based biotech company Codagenix) on the animal models and hopes to have a vaccine by 2021, its CEO Adar Poonawalla has said.

Despite the urgency, there are challenges in making a vaccine for SARS-CoV-2, said Kundu. The tools that are normally available to us otherwise are not available here, he said. For instance, they did not have specific antibodies to test antigens. Despite that we have been able to work through this by developing surrogate models, he said.

The amount of time that the immunity lasts in the body is also something that is still not known. In the case of SARS-CoV and MERS infections, the natural immunity does not last long, said Singh. Based on that knowledge, one has to also decide the doses of vaccination to be given in order to have immunity for a long time. The challenge to produce in huge quantities to cover the population under a mass vaccination programme will also need to be taken on. That will require a global coalition, and not just a few companies.

For those working on vaccines, what has helped, said Kundu, is that regulatory pathways are being fast-tracked and regulators are now willing to consider new scientific processes, and collaborations are happening. For instance, two global vaccine manufacturersSanofi and GSKhave come together to develop an adjuvant vaccine for Covid-19. Sanofi is providing the antigen that will be produced on its Baculovirus Expression Vector System platform, said its spokesperson. The recombinant technology produces an exact genetic match to proteins found on the surface of the virus, the spokesperson told THE WEEK. GSK will provide its novel adjuvant technologyAS03.

An adjuvant is a substance that is combined with a vaccine antigen to help stimulate a stronger and more targeted immune response. This can help provide better protection or in some instances, like a pandemic, could reduce the amount of antigen required per dose, allowing more vaccine doses to be produced and supplied, said the spokesperson. This is a critical advantage in a pandemic setting. The AS03 adjuvant will help improve the immune response to the antigen and may also be antigen sparing. Due to the critical need for a vaccine to address Covid-19, Sanofi will be testing its own adjuvant as well.

According to Jameel, the challenge in building a vaccine against Covid-19 may not be any different from making a vaccine for other diseases. The power of technology available today is evident from the fact that since the pandemic began in January, we already have more than 100 candidates, he said. For India, the opportunity will be in manufacturing the vaccines that are developed eventually and making them affordable for all.

More here:
Team spirit - THE WEEK

WASHINGTON, April 15, 2020 /PRNewswire/ --Vanda Pharmaceuticals, Inc.(Vanda) (Nasdaq: VNDA) today announced the initiation of the CALYPSO program to study the role that human genetic variations play in SARS-CoV-2 ("COVID-19") infection and disease progression. As a part of the CALYPSO program, Vanda will collaborate with University of Washington School of Medicine and its Virology Lab on a pharmacogenetics study in patients with COVID-19. The study will focus on the sequencing of the genome of individual patients, as well as the COVID-19 virus, and the identification of genetic factors that correlate with disease progression and outcomes.

In support of this study, Vanda and UW Medicine plan to collect Whole-Genome Sequencing ("WGS") data from over 1,000 patients with COVID-19 infection, and perform Viral Genome Sequencing, which should enable Vanda and the UW Medicine Virology Lab to explore host susceptibility, associations of WGS with clinical outcomes and severity of disease, as well as host-virus interactions. The study is scheduled to begin enrollment in the coming weeks and will be open to patients in hospitals and clinics around the United States.

"We look forward to the advancement of our program and the opportunity to work with and leverage the expertise of UW Medicine to expand our understanding of the COVID-19 infection mechanism," said Mihael H. Polymeropoulos, M.D., President and Chief Executive Officer of Vanda.

"The study has the potential to provide new insights into virushost interactions that could lead to more effective public health strategies and the design and development of vaccines and therapeutics," said Sandra P. Smieszek, Ph.D., Head of Genetics at Vanda. "With the vast amount of data we expect to collect, the team will aim to discern the factors associated with severity and other critical, clinical characteristics of the infected individuals."

"By leveraging our sequencing expertise and capabilities in collaboration with Vanda, we will be able to provide the necessary insight for potentially life-saving solutions for patients," said Alex Greninger M.D., Ph.D., M.S., M.Phil., Assistant Professor, Laboratory Medicine, Assistant Director, Virology Division at the University of Washington School of Medicine. "We believe this collaboration will help answer critical questions and hopefully outcomes in the fight against COVID-19."

"We are grateful to collaborate with Vanda as we try to find better ways to care for people currently suffering from COVID-19, and as we develop plans for the next phase of the national response," said Keith R. Jerome, M.D., Ph.D., Head of Virology Division at the University of Washington School of Medicine. "The approach of combining host and viral genomics to identify the most promising treatments may serve as a model for future efforts around the world. This unique agreement positions UW Medicine and Vanda for potentially changing the course of the COVID-19 pandemic."

"This is the type of collaboration we need to bring solutions to patients suffering in this time of crisis," said Dr. Greninger. "We look forward to getting this important work underway."

About Vanda Pharmaceuticals Inc.

Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit http://www.vandapharma.com and follow us on Vanda's Twitter and LinkedIn.

About UW Virology

The UW Virology is one of nine divisions comprising the Department of Laboratory Medicine at the University of Washington School of Medicine. The UW Medicine Virology Clinical Laboratories perform diagnostic testing for a full range of human pathogens including respiratory viruses, herpes group viruses, HIV, hepatitis, and enteric viruses, and was one of the earliest providers of COVID-19 testing. The Division provides the highest quality patient care and serves as a model of excellence for clinical laboratories across the nation. Its UW Virology Lab is also recognized as a worldwide leader in virology research. UW Medicine Virology's research programs integrate the latest in computational, laboratory, and clinical research methods to advance the understanding of infectious diseases. Many past and current faculty members in the Virology Division have received prestigious awards recognizing their scientific achievements.

Vanda Contact:

AJ Jones IIChief Corporate Affairs and Communications OfficerVanda Pharmaceuticals Inc.202-734-3400

pr@vandapharma.com

UW Medicine Contact:

Susan GreggDirector, Media Relations206-616-6730

sghanson@uw.edu

CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS

Various statements in this release are "forward-looking statements" under the securities laws. These forward-looking statements include, without limitation, statements regarding the design, enrollment and anticipated findings of the CALYPSO program, the promotion of more effective public health strategies and the design and development of vaccines and therapeutics. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others: Vanda's ability to enroll patients for, and successfully conduct, the study described in this press release; the ability of Vanda, either alone or with its partners, to process the data collected and subsequently develop effective vaccines or therapeutics; the ability to obtain FDA approval of any such vaccines or therapeutics; and other factors that are set forth in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's annual report on Form 10-K for the fiscal year ended December 31, 2019, which is on file with the SEC and available on the SEC's website at http://www.sec.gov. Additional factors may be set forth in those sections of Vanda's annual report on Form 10-Q for the fiscal quarter ended March 31, 2020, to be filed with the SEC in the second quarter of 2020. In addition to the risks described above and in Vanda's annual report on Form 10-K and quarterly reports on Form 10-Q, other unknown or unpredictable factors also could affect Vanda's results. There can be no assurance that the actual results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this release is provided only as of the date of this release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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SOURCE Vanda Pharmaceuticals Inc.

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Vanda Pharmaceuticals Announces Initiation of "CALYPSO" to Study the Role of Genetic Variation in COVID-19 Infections in Collaboration With...

As Americans yearn for a future with work, shopping and outings with friends, one concept may become a crucial prerequisite: the widespread use of antibody tests.

Antibody tests, also known as serology tests, are designed to detect the presence of antibodies proteins that are key elements of the bodys immune response to germs. Antibodies would be present in people who battled the coronavirus and recovered, as well as in people who had been exposed but whose infection was so mild that they didnt notice they were sick.

If someone has antibodies from exposure to the coronavirus, that person should have immunity to the virus, at least for a period of time. (More on that in a bit.) That means they can go out into the wider community without fear of getting sick themselves. Antibody tests could also be helpful for making staffing decisions in medical units treating coronavirus patients.

Anthony Fauci, a top federal official in the fight against the coronavirus, has floated the idea of "immunity certificates" based on antibody testing. "As we get to the point of considering opening the country, it is very important to understand how much that virus has penetrated society," Fauci said on CNN.

Its also a key element of Democratic presidential candidate Joe Bidens plan for reopening the country. And New York Gov. Andrew Cuomo has gone so far as to develop and start implementing an antibody testing system for New York State. "We cannot restart life as we knew it without testing," Cuomo has tweeted.

PolitiFact interviewed several experts about the promise and potential pitfalls of antibody testing.

What has been done so far?

The Food and Drug Administration has already granted an "emergency use authorization" for one particular antibody test manufactured by a company called Cellex. The FDA also green-lighted other tests for the market without the usual review process, as long as they are labeled with disclaimers that the agency has not fully reviewed them.

That enabled more than 90 tests to go to market, some from academic organizations and some from medical companies, according to Politico. The CDC itself is working to produce one. Researchers at Stanford University have produced one test, while Mount Sinais Icahn School of Medicine has produced another.

The key to developing antibody tests is to have an appropriate coronavirus protein, "because thats what antibodies recognize," said Elizabeth McNally, director of the Center for Genetic Medicine at Northwestern Universitys Feinberg School of Medicine.

The tests work by having some viral protein adhered to a surface, McNally said. "A persons blood sample is mixed with a virus protein and is tested for whether there is an interaction between antibodies in the persons blood and the antibody on the surface."

The good news is that many of the tests are not overly complicated, often requiring blood from a self-administered finger prick. Some have a turnaround time as rapid as 10 to 15 minutes.

How accurate are these antibody tests?

Thats the bad news: The tests so far have not been especially accurate.

In mid April, the FDA said the National Cancer Institute would start reviewing tests for accuracy. This move was welcomed by medical experts.

The United Kingdom has already pulled some tests for inaccuracy. Many antibody tests now being rolled out "may not be as accurate as wed like," FDA commissioner Stephen Hahn has acknowledged. Scott Becker, CEO of the Association of Public Health Laboratories, went so far as to label many of the tests "crappy," CNN reported.

"There is very limited data, almost none peer-reviewed, evaluating how well these tests perform," said Elitza S. Theel, director of the infectious diseases serology laboratory at the Mayo Clinic. "So its critical that laboratories carefully perform validation and verification studies to ensure that the test they are offering for clinical testing is accurate."

A key metric will be a tests "sensitivity," which refers to the percentage of positive tests that reflect genuine infections. "Anything less than 99% would mean too many cases in the population are wrongly identified, throwing off population estimates," said Theo Vos, a professor at the University of Washingtons Institute for Health Metrics and Evaluation.

Will antibodies actually confer immunity?

Scientists expect that a past infection will produce some immunity. But they arent entirely certain of it, and it remains unclear how long that immunity could last.

Generally speaking, the degree and duration of immunity from an infection varies depending on the germ in question. Immunity from common cold viruses last a few weeks; immunity from chickenpox can last decades. Many infectious diseases fall somewhere in between.

One hopeful sign is that an infection with the virus that causes SARS, which is similar to COVID-19, produces an antibody response for a year, maybe more. Thats not as long as some diseases, but it could buy time until a successful vaccine is produced, which experts say could happen within 12 to 18 months.

One unknown is whether that immune response from a past coronavirus infection is strong enough to actually preclude a new infection.

COVID-19 probably gives immunity to "most people, but we dont know for sure that is the case in every recovered COVID patient," said Angela Rasmussen, associate research scientist at the Center for Infection and Immunity at the Columbia University Mailman School of Public Health. "We dont know what levels of antibodies confer protection either, so we should not assume that just having antibodies guarantees that you have completely protective immunity. Theres still a lot more to learn."

What are some of the practical challenges of administering tests?

One practical concern is when to collect samples from individuals.

"We know it takes over a week in some cases to mount an immune response to the virus and to develop a detectable level of antibodies," Theel said. "The samples need to be collected at a certain time after symptom onset. Using samples collected too soon would lead to negative results simply because the patient hasnt developed an immune response to the virus yet."

By the same token, additional testing may be necessary to confirm that someone who is antibody-positive is no longer experiencing an active infection that can get others sick.

Another practical issue is where the test should be administered.

"We dont want to have people rushing to a doctors office or a hospital since that could actually worsen the spread of the virus," McNally said. "Some companies are producing small individual test kits which will produce a + or - result. Some of these tests will require a doctors order and some may become commercially available."

For instance, McNallys institution, Northwestern University, is developing a kit that is designed to be sent through the mail. Once returned via mail with a blood drop on specially treated paper, the tested individual can find out their result a few days later through a secure website.

What can antibody testing tell us about society at large, rather than just the individual?

Knowing how many people are immune will be a key element of the decision to reopen the economy. However, undertaking society-wide testing will require coordinated effort.

Already, some studies are under way. The World Health Organization is working on a study of a half-dozen nations, while one effort in the U.S. is already collecting blood samples in such cities as New York, Seattle and Minneapolis. Other studies are occurring in Michigan, Miami and Los Angeles.

One study completed in a hard-hit town in Germany recently found that 14% of those tested had antibodies to coronavirus.

McNally said research studies like these are important because they "allow us to be able to know how the population has developed immunity. We will want to know what percentage of the population has developed antibodies and how this differs across neighborhoods and areas. This is the type of information that will help to guide decisions about bringing people back to work and future safety issues."

What needs to be done to enable large-scale antibody testing?

Vos recommends focusing on a small number of the most promising tests, then scaling up production. The expected high demand for antibody tests should help push companies to do this quickly, he said. In the meantime, he said, poorly performing tests should be restricted and the FDA should accelerate its regulatory process.

"Things are happening quickly, but without strong oversight there will be a flood of undocumented tests in use, muddying the waters for anyone trying to make sense of what is going on in the community," Vos said.

Its also important to note that antibody tests are just one part of whats needed to restart the economy. Experts say it will also require a system of widely available diagnostic tests to determine if someone has an active coronavirus infection, along with "contact tracing," a method of identifying people who were in close proximity to other people who were infected, so that they can be tested and, if necessary, quarantined.

CDC director Robert R. Redfield told NBC News that large-scale antibody testing could fall into place "over the next several months."

McNally told PolitiFact that she expects antibody testing to be ramping up in May.

"We know most people take about four weeks to develop antibodies and immunity, so assuming exposure to the virus occurred in March or April, we really want testing working at scale in May," she said. "I think were on target for that."

Link:
Antibody testing: The promise and pitfalls of using them to reopen the US - PolitiFact

NEW YORK and BASEL, Switzerland, April 15, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies for neurological diseases, today announced its collaboration with Invitae, a leading medical genetics company, in the Detect Lysosomal Storage Diseases (Detect) program to facilitate faster diagnoses for children with lysosomal storage disorders (LSDs), including GM1 gangliosidosis and GM2 gangliosidosis, also known as Tay-Sachs/Sandhoff disease. Invitae offers genetic testing and counseling at no charge to patients suspected of having an LSD.

Axovant is committed to developing novel gene therapies for those living with rapidly progressive neurodegenerative diseases. We are hopeful that our collaboration with Invitae will provide families with easier access to genetic testing and bring us one step closer to identifying patients who may benefit from potential therapies, said Parag Meswani, PharmD., Axovants SVP of Commercial Strategy & Operations. Our AXO-AAV-GM1 clinical program targeting GM1 gangliosidosis is currently enrolling at the National Institutes of Health, and we are seeking IND clearance for the AXO-AAV-GM2 clinical trial targeting Tay-Sachs and Sandhoff diseases. Early intervention is ideal with potentially disease-modifying genetic therapies, and our diagnostics partnership with Invitae should allow us to identify and enroll children at even earlier stages of disease progression.

LSDs are progressive, multi-system, inherited metabolic diseases associated with premature death, and genetic testing is a crucial first step to arriving at a diagnosis. LSDs are misdiagnosed or undiagnosed in the majority of patients. The Detect program includes a specific LSD testing panel of 53 genes designed to provide patients and families accurate information quickly to preserve valuable treatment time.

Genetic testing can expedite an accurate diagnosis, facilitate earlier interventions, allow genetic counseling of family members, and support clinical research for LSDs such as GM1 and GM2 gangliosidosis, said Robert Nussbaum, M.D., chief medical officer of Invitae. Were pleased Axovant has joined the Detect program to help offer no-charge, sponsored genetic testing for those patients suspected of having the disease.

Research has shown no-charge testing programs with large well-designed panels help increase utilization of genetic testing, which can shorten the time to diagnosis by as much as 2 years in some conditions. Accurate diagnoses enable clinicians to focus on providing disease-specific care sooner, helping reduce costs and improve outcomes.

Additional details, as well as terms and conditions of the program, can be found at https://www.invitae.com/en/detectLSDs/.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

About Invitae

Invitae Corporation (NVTA) is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected. Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on February 10, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

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Media Contact:

Parag MeswaniAxovant Gene Therapies(212) 547-2523investors@axovant.commedia@axovant.com

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Axovant Announces Partnership with Invitae to Increase Access to Genetic Testing and Accelerate Diagnoses of GM1 and GM2 Gangliosidosis - Yahoo...

A team at Stanford Medicine has developed gadgets that can be fitted in an ordinary toilet to screen urine and feces and upload the encrypted health data. The technology may be particularly useful for monitoring individuals at high risk of developing particular illnesses.

Many people will be uncomfortable with the idea of cameras and sensors in their toilet. It may seem like an unthinkable intrusion into what is perhaps the most private of all activities.

But a team of developers at Stanford Medicine in Stanford, CA, believe the clinical benefits of their smart toilet could be far-reaching.

They are also confident that their toilet can safeguard the privacy of users.

Technologies that continually monitor a persons health play a growing role in healthcare.

Existing devices include smartwatches for collecting data, such as heart rate, and wearable blood pressure monitors. A skin patch is in development that tracks movement, heart rate, and breathing.

The thing about a smart toilet, though, is that unlike wearables, you cant take it off, says Prof. Sanjiv Gambhir, chair of radiology at Stanford Medicine. Everyone uses the bathroom theres really no avoiding it and that enhances its value as a disease-detecting device.

Prof. Gambhir believes the smart toilet may be particularly useful for monitoring people at high risk of conditions, such as prostate cancer, irritable bowel syndrome (IBS), and kidney failure, due to their genetic predispositions, for example.

His team developed a suite of gadgets that a person can fit in the bowl of an ordinary toilet. Its sort of like buying a bidet add-on that can be mounted right into your existing toilet, he says. And like a bidet, it has little extensions that carry out different purposes.

In a pilot study, 21 volunteers tested the device over several months.

The smart toilet is the perfect way to harness a source of data thats typically ignored and the user doesnt have to do anything differently.

Prof. Sanjiv Gambhir

A motion sensor activates the smart toilet to start capturing video data, which are then digitally analyzed.

One of the smart toilets algorithms can detect abnormal urine flow rate, stream time, and volume, which could be useful for flagging prostate problems in men, for example.

Another gauges the consistency of fecal matter from the images and classifies it according to the Bristol stool chart. This is a standardized system used by clinicians worldwide to diagnose problems such as constipation, gut inflammation, and a lack of dietary fiber.

The smart toilets software can also identify color changes in urine using urinalysis strips (dipstick tests). It can detect 10 different markers, including the number of white blood cells and the levels of specific proteins in the urine. These biomarkers can provide early warnings of diseases, such as kidney infections and bladder cancer.

According to an article describing the technology in Nature Biomedical Engineering, the toilets abilities are comparable to the performance of trained medical personnel.

Encrypted data from the toilet upload to a secure cloud server. In the future, this information could integrate with a healthcare providers record-keeping system for easy access by the individuals doctor.

The Stanford team envisages an app sending a text alert to the healthcare team if the device detects an urgent issue, such as blood in someones urine.

Identifying who is using the toilet will be critical in a household of several people.

The whole point is to provide precise, individualized health feedback, so we needed to make sure the toilet could discern between users, Prof. Gambhir said. To do so, we made a flush lever that reads fingerprints.

However, in case someone uses the toilet and another flushes it, or if the toilet has an auto-flush system, a camera captures what the article calls the distinctive features of their anoderm [skin tissue lining of the anus].

We know it seems weird, but as it turns out, your anal print is unique, says Prof. Gambhir.

The recognition system is fully automatic, which means that no human will see the scans.

Despite the teams best efforts to ensure user privacy and data confidentiality, the smart toilet may prove a hard sell.

A survey conducted by the researchers of 300 prospective users revealed that only 15% described themselves as very comfortable with the concept.

The researchers plans include recruiting more volunteers to test the toilet and individualizing the available tests. A patient with diabetes might want glucose levels in their urine checked, for example.

In addition to urine tests, the team would also like to build into their toilet the ability to carry out molecular analysis of stool samples.

Thats a bit trickier, but were working toward it, says Prof. Gambhir.

If successful, one advantage for the squeamish will be that they no longer have to collect their own stool samples and take them to a clinic for testing.

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'Smart toilet' recognizes users and checks for signs of disease - Medical News Today

As the COVID-19 pandemic continues to claim lives around the world, there are no specific treatments for the disease beyond supportive care. Several drugs already prescribed for other illnesses have shown promise against the novel coronavirus in preclinical studies. And they are now being tested in clinical trials or given to patients on a compassionate-use basis. But experts warn that these medications have yet to prove effective in treating COVID-19 patients.

As of this writing, the virus has infected more than two million people worldwide and caused more than 130,000 deaths. A vaccine and new treatments could take years to fully develop, but the World Health Organization recently launched a large international trial called Solidarity to test four existing therapies. They are the closely related malaria drugs chloroquine and hydroxychloroquine; the antiviral medication remdesivir (originally developed to treat Ebola); the antiviral combination of lopinavir and ritonavir (used for HIV); and those two HIV drugs plus the anti-inflammatory small protein interferon beta. A number of separate clinical trials of these medications and others are underway in several countries, including the U.S.

The U.S. Food and Drug Administration has approved remdesivir for treating COVID-19 patients under the compassionate-use protocol (a designation that gives patients with life-threatening illnesses access to an experimental drug). And the agency has granted an emergency use authorizationwhich allows for otherwise unapproved drugs or uses during an emergencyfor chloroquine and hydroxychloroquine.

None of these therapies are proven, says Stanley Perlman, a professor of microbiology and immunology at the University of Iowa. Only the results of randomized clinical trials can show whether they work, he adds.

Here is what scientists know so far about some of the most prominent drugs currently being tested as treatments for the potentially deadly infection.

President Donald Trump has repeatedly touted the malaria drugs chloroquine and hydroxychloroquine as a treatment for COVID-19despite a lack of clinical evidence that they work for the disease. The presidents comments set off a scramble among doctors and patients to obtain the drugswhich are frequently used to treat autoimmune diseases such as rheumatoid arthritis and lupusand there is now a shortage of them in the U.S. Also, these substances can be dangerous in healthy people: a man in Arizona died after ingesting a fish-tank cleaner containing a type of chloroquine that is not approved for human use. On March 28 the FDA issued an emergency authorization for administering chloroquine or hydroxychloroquine to COVID-19 patientsbut many experts say the widespread usage of these drugs is premature.

The clinical support is very, very minimal, says Maryam Keshtkar-Jahromi, an assistant professor of medicine at the Johns Hopkins University School of Medicine, who co-authored an article in the American Journal of Tropical Medicine and Hygiene calling for more randomized controlled trials of chloroquine and hydroxychloroquine. The drugs do not show strong evidence at this point, she adds.

A few preclinical studies have suggested these compounds could be effective at blocking infection with the novel coronavirus (officially called SARS-CoV-2), but there has been very little good evidence from clinical trials in patients with the illness it causes, COVID-19. A controversial small, nonrandomized trial of hydroxychloroquine combined with the antibiotic azithromycin in France suggested that COVID-19 patients given the treatment had less virus, compared with those who refused the drugs or those at another hospital who did not receive them. But experts have questioned the studys validity, and the society that publishes the journal in which it appeared has issued a statement of concern about the results, according to Retraction Watch. (Scientific American reached out to the papers authors for comment but did not hear back from them.) A preprint study in China also claimed to show that hydroxychloroquine benefitted COVID-19 patients, but it had significant methodology problems, Keshtkar-Jahromi says. The issues included confounding variables, such as the fact that all of the subjects received other antiviral and antibacterial treatments.

Some scientists say the preclinical evidence is strong enough to support chloroquines use, however. We know how it acts at the cellular level against the virus. We have preclinical proof, says Andrea Cortegiani, an intensivist and researcher in the departments of anesthesia and intensive care and of surgical, oncological and oral sciencesat the University of Palermo in Italy. Second, its a cheap drug, available all over world, adds Cortegiani, who is also a clinician at University Hospital Paolo Giaccone in Italy.

Chloroquine and hydroxychloroquine have been hypothesized to work against COVID-19 by changing the pH required for SARS-CoV-2 to replicate. Given their use in autoimmune disorders, these medications could also play a role in dampening the immune response to the viruswhich can be deadly in some patients.

But these drugs cardiac toxicity is a concern, Keshtkar-Jahromi says. There have been some reports of myocarditis, or inflamed heart tissue, in people with COVID-19 who have not taken chloroquine or hydroxychloroquine. If patients receiving one of these medications die of heart complicationsand are not in a clinical trialdoctors cannot know if the drug contributed to higher chance of death.*

A drug that modulates the immune response could also make someone more vulnerable to other viral or bacterial infections. Its a double-edged sword, says Sina Bavari, chief science officer and founder of Edge BioInnovation Consulting in Frederick, Md., who co-authored Keshtkar-Jahromis article in the American Journal of Tropical Medicine. Giving a drug to suppress the immune system has to be done with extreme care.

We are not saying, Dont [prescribe chloroquine], Bavari says. We are saying, More data is needed to better understand how the drug worksif it works.

This experimental antiviral drug was developed to treat Ebola, and it has been shown to be safe for use in humans. It is a broad-spectrum antiviral that blocks replication in several other coronaviruses, according to studies in mice and in cells grown in a lab. In addition to the WHO investigation, at least two trials in China and one in the U.S. are currently evaluating remdesivir in COVID-19 patients. Results for the Chinese trials are expected later this month.

As of this moment, we dont have data for remdesivir in human COVID-19 disease, says Barry Zingman, a professor of medicine at Albert Einstein College of Medicine and clinical director of infectious diseases at Montefiore Health Systems Moses Campus. The two related institutions, both located in New York City, recently joined a nationwide clinical trial of the drug. Our patients are randomized, so we dont know whos getting the drug or a placebo. [But] we have seen some patients do remarkably well, Zingman says. Trial results are on track for publication sometime in the next six to eight weeks, he adds.

As Scientific American reported previously, remdesivir works by inhibiting an enzyme called an RNA-dependent RNA polymerase, which many RNA virusesincluding SARS-CoV-2use to replicate their genetic material. Timothy Sheahan of the University of North Carolina at Chapel Hill and his colleagues have shown the drug is effective against the coronaviruses that cause severe acute respiratory syndrome (SARS) and Middle East respiratory syndrome (MERS), respectively, as well as some of the viruses behind the common cold. The team is currently in the process of testing the drugs efficacy against SARS-CoV-2. A recent study of compassionate use of remdesivir in 53 severe COVID-19 patients found that 63 percent of those taking the drug improved, but it was not a randomized controlled trial.

Remdesivir has some chance, Perlman says. If we can give [the drug] early in the disease course, it could work. To know for sure, scientists must await the results of the ongoing clinical trials.

One limitation with remdesivir is that it must be given intravenously, so patients can only get it in a hospital. Sheahan and his colleagues at Emory University have recently developed a related drug called EIDD-2801, which can be taken in pill form. Like remdesivir, the medication works as a nucleoside analogue, interfering with viral replication. It was effective at preventing SARS-Cov-2-infected lung cells from replicating in a lab dish and related viruses from doing so in mice.

The HIV drugs ritonavir and lopinavir (sold as a combination therapy by AbbVie under the brand name Kaletra) have been tested against COVID-19 in a few clinical trials. The initial data have not shown them to be effective, however. A study in the New England Journal of Medicine found they conferred no benefit beyond standard care.

The drug combination is what is known as a protease inhibitor, and it works by blocking an enzyme involved in viral replication. But its action is specific to HIV and so is unlikely to work for SARS-CoV-2, Perlman says. If you have the key to a car, and you try to put it in your car, the odds of it working are one in a million, he says. Kaletra [targets] a completely different lock than the one for COVID-19.

Nevertheless, the WHO trial includes a group of COVID-19 patients who will receive these drugs on their ownand another group that will receive them in combination with interferon beta, a small cell-signaling molecule used to treat multiple sclerosis. The molecule is a massive orchestrator of immune response, Perlman notes, so it must be used carefully. In mouse studies of the SARS and MERS coronaviruses, it halted the infections when administered early. When it was given later, he says, the mice died. Using a drug that activates the immune system could be helpful in the beginning of an infection, but giving it too late could be deadly.

Researchers are also considering a number of other therapies that tamp down the rampant immune response seen in severe COVID-19 cases. Such a flood of immune cells in the lungsknown as a cytokine stormcan lead to death. Many of the sickest patients have elevated levels of an inflammatory protein called interleukin-6 (IL-6). Research in China has suggested that Actemra (tocilizumab), an IL-6-blocking antibody drug made by Roche, shows promise against COVID-19. And Chinese authorities have recommended the drug in their treatment guidelines. Roche has since initiated a phase III randomized controlled clinical trial for the medication. In the U.S., Michelle Gongchief of the division of critical care at Montefiore and Albert Einstein and director of critical care research at Montefioreand her colleagues are among dozens of groups conducting a double-blind, placebo-controlled clinical trial of a related drug called sarilumab, which is already approved for treating rheumatoid arthritis. Sarilumab will only be given to the sickest individuals: to be part of the trial, patients must be hospitalized with COVID-19 and in severe or critical condition.

Another treatment approach involves injecting COVID-19 patients with blood plasma from people who have recovered from the illness. The FDA recently issued guidance on the investigational use of such convalescent plasma, which contains antibodies to the coronavirus, and clinical trials are underway.

Blood from disease survivors has been used as a treatment throughout historyfrom polio-infected horses in the 1930s to former Ebola patients in 2014. There is a long-lasting rationale for the use of convalescent plasma against any infectious disease, Cortegiani says. One problem, however, is that scientists do not know whether people develop strong immunity against SARS-CoV-2. And it is not easy to collect plasma containing enough antibodies, he adds. Another issue is the shortage of eligible donors. Some companies are looking into ways to produce these antibodies artificially. In the meantime, a number of hospitals are searching for volunteers to donate plasma.

None of the therapies described above have yet been proved to treat COVID-19. But some answers can be expected in the next few weeks and months as the results of clinical trials emerge. Until then, Cortegiani says, we cannot say, This drug is more promising than the other one. We can only say, There is a rationale for it.

Read more about the coronavirus outbreakhere.

*Editors Note (4/16/20): This paragraph was edited after posting to correct Maryam Keshtkar-Jahromis comments about her concerns with chloroquine and hydroxychloroquine.

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Here's What We Know about the Most Touted Drugs Tested for COVID-19 - Scientific American

At the front lineof advances in personalized medicine are cell and gene therapies. These are two overlapping fields of medical research whereby the overall aim is to treat the underlying causes of both genetic and acquired diseases.

Is the promise of personalized medicine on the edge of being delivered? Decades of research and advances in genomics, cell biology, cancer and analytical technologies have permitted exciting progress in the cell and gene therapy space recently. Technology Networks recently spoke with three of the leaders in this space, Allogene Therapeutics, bluebird bioInc. andMogrify to gain their insights on the next developments in cell and gene therapy.

Joe Foster, COO, Mogrify.

"We are on the cusp of new breakthroughs in this field and that evolution of engineered cell therapy has the potential to expand beyond cancer. Engineering, synthetic biology and gene editing has opened the door beyond allogeneic cell therapy. There is a bright future with transformative technologies that may hold the key to addressing solid tumors."

David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder, Allogene Therapeutics.

"Although gene therapies are still a nascent technology, they present a new paradigm for healthcare, offering a one-time treatment that can address the underlying genetic cause of certain severe genetic diseases and cancer. We anticipate that the three techniques being studied for gene therapy gene editing, gene addition and gene-based immunotherapy will become more distinct and that the entire treatment journey will become more efficient."

Martin Butzal, Head of Medical Europe, bluebird bio.

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Looking to the Future of Cell and Gene Therapies - Technology Networks

Cave nectar bat (Eonycteris spelaea) from Singapore. Justin Ng/Linfa Wang hide caption

Cave nectar bat (Eonycteris spelaea) from Singapore.

Updated at 11 a.m. ET

Dr. Linfa Wang, a virologist at the Duke-National University of Singapore, has been working around the clock to help Singapore fight this coronavirus. He hasn't hugged his daughter in over two months.

"We're in a war zone right now. Everything comes to me very fast," Wang said in an interview with NPR's Short Wave podcast. He's given over 100 interviews since January, when international reports first surfaced of a new coronavirus.

Since then, scientists have learned a great deal about the coronavirus, now called SARS-CoV-2. And one of the mysteries they're still trying to untangle is where the virus came from in the first place. Scientific evidence overwhelmingly points to wildlife, and to bats as the most likely origin.

Wang is an expert in emerging zoonotic diseases, or diseases hosted in animals that spread to humans. The CDC estimates that six out of ten infectious diseases in people come from animals. Among them are Lyme disease, Rabies, West Nile, and diseases caused by coronaviruses, including this coronavirus and the SARS virus.

The 2003 outbreak of SARS was eventually traced to horseshoe bats in a cave in the Yunnan province of China, confirmed by a 2017 paper published in the journal Nature. It was a detective hunt that took over a decade, sampling the feces, urine, or blood of thousands of horseshoe bats across the country and seeing if those samples are a genetic match to the virus.

The work of virus hunting of tracking an outbreak to its origin point can take years. Wang stresses that pinpointing the true origin of this coronavirus will take time as well. "Of course, the technology and everything is much more advanced 17 years later [since the 2003 SARS outbreak]. But, for us to try to solve everything in two to three months is just not feasible."

'This is a product of nature'

In early January 2020, Chinese scientists sequenced the entire genome for SARS-CoV-2 and published it online. Researchers at the Wuhan Institute of Virology in China compared its genome to a library of known viruses and found a 96% match with coronavirus samples taken from horseshoe bats from Yunnan.

"But that 4% difference is actually a pretty wide distance in evolutionary time. It could even be decades," says Dr. Robert F. Garry, professor of microbiology and immunology at Tulane University School of Medicine.

That extra 4% suggests the SARS-CoV-2 may not have evolved from bats alone, but may include viral material from another animal. In that case, the virus would have continued to evolve through natural selection in that animal. Moreover, that other animal may have acted as an "intermediate host," ultimately transmitting the virus to humans.

With this coronavirus, scientists aren't fully clear on whether an intermediate host was involved nor the chain of cross-species transmission to humans. Studies have found a genetic similarity between this coronavirus and coronaviruses found in pangolins, also called scaly anteaters, which are vulnerable to illegal wildlife trade.

Given that some China's earliest COVID-19 patients were connected to the Hunan Seafood Wholesale Market in Wuhan, it is likely the seafood market played a role in amplifying the virus. However, there is not enough evidence to prove that is where the virus transmitted from animals to humans. There is also evidence emerging that among the first 41 patients hospitalized in China, 13 had no connection to this particular marketplace. The path of the pathogen is still unknown.

As for clues the virus holds about its animal origins, Robert Garry and fellow researchers have hypothesized that SARS-CoV-2 could be a blend of viruses from bats and another animal.

"The receptor binding domain actually shares a lot of sequence similarity to a virus that's found in the pangolin" Garry said, referring to the receptor-binding mechanism that allows the virus to form a strong attachment to human cells. "So, those sequences probably did arise from a virus like the pangolin coronavirus, or maybe some other coronavirus that can circulate in pangolins or some other animals." Further genetic analysis is needed to figure this out.

In studying the genome, Garry also confirms the virus came from wildlife. "This is a product of nature. It's not a virus that has arisen in a laboratory by any scientist, purposely manipulating something that has then been released to the public," he said.

'It's A Billion Dollar Question'

So, why are bats such good hosts for viruses?

"I used to say it's a million dollar question. Now I say it's a billion dollar question," said Wang, speaking to NPR's Short Wave podcast on Tuesday.

Bats are critically important for pollinating flowers and dispersing seeds. They catch bugs, the same ones that bite us and eat some of our crops. But bats also harbor some of the toughest known zoonotic diseases.

The Rabies virus, the Marburg virus, the Hendra and Nipah viruses all find a natural reservoir in bats, meaning those viruses live in bats without harming them. The Ebola outbreak in West Africa was traced to a bat colony. The SARS virus originates in bats, along with other coronaviruses. And now, SARS-CoV-2 is linked to bats too.

Wang believes bats' high tolerance for viruses may have to do with the fact that they are the only mammal that's adapted for flight.

"During flight, their body temperature goes all the way to 42 degrees (or 108 degrees Fahrenheit). And their heartbeat goes up to 1000 beats per minute," Wang said.

Flying several hours a day, bats burn a great deal of energy. This creates toxic free radicals that damage their cells, but Wang's research has shown that bats have also evolved abilities to repair and minimize that cellular damage. Those same defensive abilities may help them not only tolerate flight, but also to fight infectious diseases in a way that human bodies cannot.

"Our hypothesis is that bats have evolved a different mechanism to get the balance right for defensive tolerance. And that favors the virus to live peacefully with bats," said Wang.

Peter Daszak, President of the U.S.-based non-profit Ecohealth Alliance, says that even if bats are the origin, they are not to blame for the pandemic.

"It's not bats. It's us. It's what we do to bats that drives this pandemic risk," Daszak said. His research demonstrates how interactions between wildlife and livestock, food and agriculture practices, as well as humans close proximity to animals in densely populated areas, create the conditions for viral outbreaks.

"One of the positive things about finding out that we're actually behind these pandemics is that it gives us the power to do something about it. We don't need to get rid of bats. We don't need to do anything with bats. We've just got to leave them alone. Let them get on, doing the good they do, flitting around at night and we will not catch their viruses," Daszak said.

Given that infectious and zoonotic diseases have been on the rise for decades, Wang is frustrated by the fact that countries around the world failed to understand the impact this novel coronavirus outbreak would have.

"I'm so angry right now. This COVID-19 outbreak, before January 20th, you could say it's China's fault. The Chinese government. But after January 20th, the rest of the world is still not taking it seriously. Our political system, our diplomatic system, our international relationship system is just not ready," Wang said.

January 20th is when Chinese health officials confirmed the new coronavirus could be transmitted between humans and the World Health Organization kicked into high gear to evaluate the global risk. There were more than 200 cases then. Now, the confirmed case count is nearing 2 million worldwide.

Email the show at shortwave@npr.org.

This episode was produced by Brit Hanson, edited by Viet Le, and fact-checked by Emily Vaughn.

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Where Did This Coronavirus Originate? Virus Hunters Find Genetic Clues In Bats - NPR

Mary Joyce of Overland Park, Kansas, lives each day as if it is a gift because, in her mind, it absolutely is. She lives with the rare Pompe disease, a degenerative muscle disease, which took her sisters life in 2013. My sister passed away only at 53," Joyce said. "I call myself in the bonus years at age 58!April 15 is International Pompe Day. Its a day to highlight the people who live with the genetic condition and to bring attention to it. Joyce said its hard to explain what Pompe has done to her. It looks like youre healthy from the outside, but its all inside," she said. "My muscles are like rubber bands -- like noodles inside. Her sister Shirley was older than her by three years, and was diagnosed first. Joyce said, When she first started having symptoms, and falling inside her house, she would call me and me and the boys would go over and help her. Years after her symptoms started, a doctor in St. Louis diagnosed Shirley. A few years later, Shirley diagnosed her sister. She said, 'You have the waddle!' Joyce said. Shirley noticed the way Marys hips moved when she walked, and urged her to get tested.The test came back positive for Pompe. Both women qualified for a clinical trial for a medication to treat Pompe. Once the double-blind study was completed and a year had passed, the sisters were told Joyce had received the medication. Shirley had not. "I felt so bad that she did not get the drug," Joyce said. "That just broke my heart. I thought why did I get it? I didn't deserve it. She was having symptoms. I was just barely starting."When asked what she misses most about her sister, Joyce answered, Everything.Joyce is now a grandmother of two, and is watching her sisters grandchildren grow. She gets enzyme replacement therapy every-other week. And she dreams, "that no one will have to endure this. That itll be somehow wiped out -- that there will be a medicine to get those newborns right away before any damage is done to the sweet babies, and that they have a wonderful, normal life.

Mary Joyce of Overland Park, Kansas, lives each day as if it is a gift because, in her mind, it absolutely is.

She lives with the rare Pompe disease, a degenerative muscle disease, which took her sisters life in 2013.

My sister passed away only at 53," Joyce said. "I call myself in the bonus years at age 58!

April 15 is International Pompe Day. Its a day to highlight the people who live with the genetic condition and to bring attention to it.

Joyce said its hard to explain what Pompe has done to her.

It looks like youre healthy from the outside, but its all inside," she said. "My muscles are like rubber bands -- like noodles inside.

Her sister Shirley was older than her by three years, and was diagnosed first.

Joyce said, When she first started having symptoms, and falling inside her house, she would call me and me and the boys would go over and help her.

Years after her symptoms started, a doctor in St. Louis diagnosed Shirley. A few years later, Shirley diagnosed her sister.

She said, 'You have the waddle!' Joyce said.

Shirley noticed the way Marys hips moved when she walked, and urged her to get tested.

The test came back positive for Pompe.

Both women qualified for a clinical trial for a medication to treat Pompe. Once the double-blind study was completed and a year had passed, the sisters were told Joyce had received the medication. Shirley had not.

"I felt so bad that she did not get the drug," Joyce said. "That just broke my heart. I thought why did I get it? I didn't deserve it. She was having symptoms. I was just barely starting."

When asked what she misses most about her sister, Joyce answered, Everything.

Joyce is now a grandmother of two, and is watching her sisters grandchildren grow.

She gets enzyme replacement therapy every-other week. And she dreams, "that no one will have to endure this. That itll be somehow wiped out -- that there will be a medicine to get those newborns right away before any damage is done to the sweet babies, and that they have a wonderful, normal life.

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International Pompe Day highlighting those who live with rare genetic condition - KMBC Kansas City

When adult brain cells are injured, they revert to an embryonic state, according to new findings published in the April 15, 2020 issue of Nature by researchers at University of California San Diego School of Medicine, with colleagues elsewhere. The scientists report that in their newly adopted immature state, the cells become capable of re-growing new connections that, under the right conditions, can help to restore lost function.

Repairing damage to the brain and spinal cord may be medical sciences most daunting challenge. Until relatively recently, it seemed an impossible task. The new study lays out a transcriptional roadmap of regeneration in the adult brain.

A cross-section of a rat brain depicts cells (in blue) expressing normal levels of the Huntingtin gene while cells (in red) have had the gene knocked out. The latter cells, without the Huntingtin gene, displayed less regeneration.

Using the incredible tools of modern neuroscience, molecular genetics, virology and computational power, we were able for the first time to identify how the entire set of genes in an adult brain cell resets itself in order to regenerate. This gives us fundamental insight into how, at a transcriptional level, regeneration happens, said senior author Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

Using a mouse model, Tuszynski and colleagues discovered that after injury, mature neurons in adult brains revert back to an embryonic state. Who would have thought, said Tuszynski. Only 20 years ago, we were thinking of the adult brain as static, terminally differentiated, fully established and immutable.

But work by Fred Rusty Gage, PhD, president and a professor at the Salk Institute for Biological Studies and an adjunct professor at UC San Diego, and others found that new brain cells are continually produced in the hippocampus and subventricular zone, replenishing these brain regions throughout life.

Our work further radicalizes this concept, Tuszynski said. The brains ability to repair or replace itself is not limited to just two areas. Instead, when an adult brain cell of the cortex is injured, it reverts (at a transcriptional level) to an embryonic cortical neuron. And in this reverted, far less mature state, it can now regrow axons if it is provided an environment to grow into. In my view, this is the most notable feature of the study and is downright shocking.

To provide an encouraging environment for regrowth, Tuszynski and colleagues investigated how damaged neurons respond after a spinal cord injury. In recent years, researchers have significantly advanced the possibility of using grafted neural stem cells to spur spinal cord injury repairs and restore lost function, essentially by inducing neurons to extend axons through and across an injury site, reconnecting severed nerves.

Last year, for example, a multi-disciplinary team led by Kobi Koffler, PhD, assistant professor of neuroscience, Tuszynski, and Shaochen Chen, PhD, professor of nanoengineering and a faculty member in the Institute of Engineering in Medicine at UC San Diego, described using 3D printed implants to promote nerve cell growth in spinal cord injuries in rats, restoring connections and lost functions.

The latest study produced a second surprise: In promoting neuronal growth and repair, one of the essential genetic pathways involves the gene Huntingtin (HTT), which, when mutated, causes Huntingtons disease, a devastating disorder characterized by the progressive breakdown of nerve cells in the brain.

Tuszynskis team found that the regenerative transcriptome the collection of messenger RNA molecules used by corticospinal neurons is sustained by the HTT gene. In mice genetically engineered to lack the HTT gene, spinal cord injuries showed significantly less neuronal sprouting and regeneration.

While a lot of work has been done on trying to understand why Huntingtin mutations cause disease, far less is understood about the normal role of Huntingtin, Tuszynski said. Our work shows that Huntingtin is essential for promoting repair of brain neurons. Thus, mutations in this gene would be predicted to result in a loss of the adult neuron to repair itself. This, in turn, might result in the slow neuronal degeneration that results in Huntingtons disease.

Co-authors include: Gunnar Poplawski, Erna Van Nierkerk, Neil Mehta, Philip Canete, Richard Lie, Jessica Meves and Binhai Zheng, all at UC San Diego; Riki Kawaguchi and Giovanni Coppola, UCLA; Paul Lu, UC San Diego and Veterans Administration Medical Center, San Diego; and Ioannis Dragatsis, University of Tennesee.

Funding for this research came, in part, from the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation, the Veterans Administration (Gordon Mansfield Consortium for Spinal Cord Regeneration), the National Institutes of Health (NS09881, EB014986), the Gerbic Family Foundation and the NINDS Informatics Center for Neurogenetics and Neurogenomics (NS062691).

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When Damaged, the Adult Brain Repairs Itself by Going Back to the Beginning - UC San Diego Health