Page 877«..1020..876877878879..890900..»

Trio wins Nobel Prize in medicine for discovery of hepatitis C virus – STAT

Posted: October 5, 2020 at 2:51 pm

Two Americans and one British-born scientist now working in Canada have won the 2020 Nobel Prize in medicine or physiology on Monday for their discovery of the hepatitis C virus.

Thomas Perlmann, secretary of the Nobel Committee and professor of molecular development biology at the Karolinska Institute, announced the award at a ceremony in Stockholm.

Harvey Alter of the National Institutes of Health in Bethesda, Md., Michael Houghton the University of Alberta in Canada, and Charles Rice of Rockefeller University in New York City share the prize.

advertisement

Their work has helped speed the fight against blood-borne hepatitis, a major global health problem that causes cirrhosis and liver cancer in people around the world. Their discovery has also allowed for the rapid development of antiviral drugs directed at hepatitis C.

The discovery of hepatitis C virus was important for public health in two ways, biochemist Jeremy Berg, professor of computational and systems biology at the University at Pittsburgh and former director of the NIHs National Institute of General Medical Sciences, told STAT. First, the discovery enabled testing for screening blood for the agent that was responsible for blood-borne transmission of hepatitis and liver cancer risk. Second, knowledge of the causative agent facilitated drug development, particularly protease inhibitors that can treat existing hepatitis C virus infections and cure the disease in many cases.

advertisement

Thanks to their discovery, highly sensitive blood tests for the virus are now available and these have essentially eliminated post-transfusion hepatitis in many parts of the world, greatly improving global health, the Nobel Prize committee said in its announcement. For the first time in history, the disease can now be cured, raising hopes of eradicating Hepatitis C virus from the world population.

The three laureates will share 10million Swedish kronor, or about $1.07 million. Their names are added to a list of medicine Nobel winners that includes 222 men and 12 women.

Hepatitis, defined as liver inflammation, is usually caused by a viral infection, although alcohol misuse, environmental toxins, and autoimmune disease can also lead to the same condition. Hepatitis A comes from contaminated food or polluted water, but the illness it causes is relatively brief in duration. Hepatitis C, like hepatitis B, is a blood-borne infection, bringing acute illness to some people but chronic disease to 71 million people around the world, according to WHO estimates.

This is the second Nobel Prize awarded for a hepatitis discovery. In the 1960s, Baruch Blumberg, who identified what became known as the blood-borne hepatitis B virus, won the prize in 1976. Baruch laid the groundwork for the development of diagnostic tests and an effective vaccine.

Alter had also been studying hepatitis in patients who had received blood transfusions. Tests based on Baruchs work could diagnose transfusion-related hepatitis B, but they still couldnt explain a large number of cases. Tests for hepatitis A virus infection were also developed around this time, but could not account for these cases. Alter and his NIH colleagues proved that this was something different when they showed that blood from these hepatitis patients could transmit the disease to chimpanzees, the only susceptible host besides humans. Later studies determined that this infectious agent was indeed a virus and the disease was a new, distinct form of chronic viral hepatitis that became known as non-A, non-B hepatitis.

Michael Houghton, then working for the pharmaceutical firm Chiron, took the next steps to isolate the genetic sequence of the virus. That meant collecting DNA fragments from nucleic acids found in the blood of an infected chimpanzee, on the theory that they would contain some genetic material from the unknown virus. Houghton and his team used sera from hepatitis patients to identify the virus based on their suspicion that the patients blood would contain antibodies to the virus.

They did find one clone and later showed that it was derived from a novel RNA virus belonging to the flavivirus family and it was named hepatitis C virus. The antibodies in chronic hepatitis patients were the smoking gun pointing toward this virus as the missing agent.

Rice, while a researcher at Washington University in St. Louis, then worked to answer an essential question: Could this clone alone cause hepatitis C? Rice and other groups had zeroed in on one end of the cloned viral genome that hadnt been characterized. There were some variations that made it difficult for the virus to replicate, so he genetically engineered an RNA variant of hepatitis C virus that included the newly defined region of the viral genome and subtracted the inactivating genetic variations. When this RNA was injected into the liver of chimpanzees, virus was detected in the blood, as were the kind of pathological changes seen in humans with the chronic disease. This was the final proof that hepatitis C virus alone could cause the unexplained cases of transfusion-mediated hepatitis.

Rice was previously honored for his studies of hepatitis C replication with the 2016 Lasker Award, often called a precursor to the Nobel. He shared that prize with Ralf Bartenschlager of the University of Heidelberg and Michael Sofia, formerly at Pharmasset and now at Arbutus Biopharma.

And Alter and Houghton shared an earlier Lasker Award, in 2000, for their hepatitis C work, furthering the Laskers reputation as a Nobel predictor. The prize recognized Alters work identifying non-A, non-B hepatitis and Houghtons use of then-early molecular biology methods to isolate the virus.

In 2013, Houghton turned down $100,000 in prize money from the Gairdner Foundation for a prize recognizing the same work because it did not include two colleagues: Qui-Lim Choo and George Kuo, who worked with him at Chiron when they identified and cloned the hepatitis C virus.

I agonized over it, Houghton told The Globe and Mail of accepting another prize in which his colleagues were not named. And I decided I didnt want to do that again.

The three of us worked closely together for almost seven years to discover this very elusive and challenging virus using a novel approach in the field of infectious disease, he told the Canadian Press at the time of the Gairdner prize. The Canadian award is also considered a Nobel bellwether.

In a conference call with reporters Monday morning, Houghton said he would accept the Nobel Prize and his share of the money. It would be really too presumptuous of me to turn down a Nobel, he said, citing the Nobels traditions and regulations drawn from the will of its creator, Alfred Nobel. Most big inventions, not all of them, but most involve many people, and I worked for six or seven years with two colleagues at Chiron Corp. in California, and without their input I doubt I would have succeeded.

This story has been updated with detail on the laureates research.

Sharon Begley contributed to this report.

Read the original:
Trio wins Nobel Prize in medicine for discovery of hepatitis C virus - STAT

Posted in Molecular Medicine | Comments Off on Trio wins Nobel Prize in medicine for discovery of hepatitis C virus – STAT

American College Health Association and Scripps Research Translational Institute Partner to Bring the NIH All of Us Research Program to College…

Posted: October 5, 2020 at 2:51 pm

SILVER SPRING, Md., Oct. 5, 2020 /PRNewswire/ --The American College Health Association (ACHA) and Scripps Research Translational Institute have announced a partnership to bring the National Institutes of Health (NIH) All of Us Research Program to five campuses this academic year in a pilot program.

All of Us is building the largest, most diverse health resource of its kind by asking one million or more participants to share their health information. Data from such a large and diverse group of people will enable scientists to see patterns in how different factorsfrom genetics to lifestyle habitsimpact a person's health, why some people respond differently to the same condition or treatment, and ultimately how to treat each person based on their unique health story.

Scripps Research is heading up key aspects of the initiative that make it possible for anyone anywhere to participate in research, including integrating mobile health technologies such as wearable devices into the research program, and digital enrollment and engagement of volunteers.

"All of Us represents a far-reaching initiative for what's possible in medical research today and in the futurehow each individual can generate useful data about their own health and what makes them tick," says Eric Topol, MD, Founder and Director of Scripps Research Translational Institute, Professor of Molecular Medicine and Executive Vice-President of Scripps Research. "The initiative will provide an unprecedented window into individual differences in biology, physiology, lifestyle, and environment that shape human health and ultimately will enable us to more effectively prevent and treat illness."

The partnership with ACHA will bring awareness about the program to young, diverse students who are eager to address health inequities on and off campus.

"Many students are seeing health inequities play out in real time as their families have been greatly impacted by COVID-19. Participation in the All of Us program is its own form of health activism, and we think students are ready to take on that challenge," says Devin Jopp, EdD, ACHA's CEO.

The five participating schools include Albion College, California State Polytechnic University-Pomona, Florida International University, Texas Southern University, and University of Louisville. These schools will enlist the help of student engagement associates to bring awareness and education to other students.

For more information on the All of Us Research Program, visit JoinAllofUs.org/students.

About ACHA

The American College Health Association (www.acha.org) is a national nonprofit association and the nation's principal leadership organization for advancing the health of college students and campus communities through advocacy, education, and research. ACHA's diverse membership provides and supports the delivery of healthcare, prevention, and wellness services for the nation's 20 million college students. ACHA advocates for integrating the critical role of college health into the mission of higher education.

About the Scripps Research Translational Institute

The Scripps Research Translational Institute, formerly named Scripps Translational Science Institute, was founded in 2007 with one essential aimto individualize healthcare by leveraging the remarkable progress being made in human genomics and combining it with the power of wireless digital technologies. Bringing together basic scientists and clinical investigators, the Translational Institute fosters highly collaborative multidisciplinary research with the greatest potential to transform the practice of healthcare and improve human health.

About the All of Us Research Program

The mission of the All of Us Research Program is to accelerate health research and medical breakthroughs, enabling individualized prevention, treatment, and care for all of us. The program will partner with one million or more people across the U.S. to build the most diverse biomedical data resource of its kind, to help researchers gain better insights into the biological, environmental, and behavioral factors that influence health. For more information, visit http://www.JoinAllofUs.org/students.

Media Contact

Rachel Mack

443-270-4560

[emailprotected]

SOURCE American College Health Association

Originally posted here:
American College Health Association and Scripps Research Translational Institute Partner to Bring the NIH All of Us Research Program to College...

Posted in Molecular Medicine | Comments Off on American College Health Association and Scripps Research Translational Institute Partner to Bring the NIH All of Us Research Program to College…

Medicure announces an agreement with Reliance Life Sciences for the marketing rights of a cardiovascular biosimilar – BioSpace

Posted: October 5, 2020 at 2:51 pm

WINNIPEG, AB, Oct. 5, 2020 /PRNewswire/ -Medicure Inc.("Medicure" or the "Company") (TSXV: MPH) (OTC: MCUJF), a pharmaceutical company, announces that through its wholly-owned subsidiary, Medicure International Inc., it has entered into a License, Manufacture and Supply Agreement (the "Agreement") with Reliance Life Sciences Private Limited ("RLS") for a cardiovascular biosimilar (the "Product"). Medicure is responsible for the regulatory approval process for the Product. A biosimilar is a biological product that is highly similar to and has no clinically meaningful differences from an approved reference product. The Agreement grants an exclusive right to Medicure to market and sell the Product in the United States of America, Canada and the European Union.

"We are very pleased with the agreement we have reached with RLS. The Product fits well with Medicure's mission of being a significant cardiovascular company focused on the U.S. market." commented Dr. Albert Friesen, Chief Executive Officer for Medicure. "We look forward to the growth of our portfolio of cardiovascular products."

About Medicure Inc. Medicure is a pharmaceutical company focused on the development and commercialization of therapies for the U.S. cardiovascular market. The present focus of the Company is the marketing and distribution of AGGRASTAT(tirofiban hydrochloride) injection and ZYPITAMAGTM (pitavastatin) tablets in the United States, where they are sold through the Company's U.S. subsidiary, Medicure Pharma Inc. For more information on Medicure please visit http://www.medicure.com. For additional information about ZYPITAMAGTM, refer to the full Prescribing Information.

About Reliance Life Sciences Private Limited Reliance Life Sciences Private Limited (RLS) is part of the Promoter Group of Reliance Industries Limited. RLS is a research driven organization developing business opportunities in bio-therapeutics (plasma proteins, biosimilars and novel proteins), pharmaceuticals, regenerative medicine, clinical research services, and molecular medicine. The Reliance Group isIndia'slargest private sector enterprise, with annual revenues of$ 86 billion USD. The Group's flagship company, Reliance Industries Limited isIndia'slargest private sector company and a Fortune Global 100 company. RLS is a fully integrated life sciences industry player with in-house capabilities in research, pre-clinical and clinical development, process development, quality management, commercial-scale manufacturing, and marketing. For further information on Reliance Life Sciences please visithttp://www.rellife.com/

To be added to Medicure's e-mail list, please visit: http://medicure.mediaroom.com/alerts

Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward Looking Information: Statements contained in this press release that are not statements of historical fact, including, without limitation, statements containing the words "believes", "may", "plans", "will", "estimates", "continues", "anticipates", "intends", "expects" and similar expressions, may constitute "forward-looking information" within the meaning of applicable Canadian and U.S. federal securities laws (such forward-looking information and forward-looking statements are hereinafter collectively referred to as "forward-looking statements"). Forward-looking statements, include estimates, analysis and opinions of management of the Company made in light of its experience and its perception of trends, current conditions and expected developments, as well as other factors which the Company believes to be relevant and reasonable in the circumstances. Inherent in forward-looking statements are known and unknown risks, uncertainties and other factors beyond the Company's ability to predict or control that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements, and as such, readers are cautioned not to place undue reliance on forward-looking statements. Such risk factors include, among others, the Company's future product revenues, the ability of AGGRASTATto provide benefits to COVID-19 patients, expected future growth in revenues, stage of development, additional capital requirements, risks associated with the completion and timing of clinical trials and obtaining regulatory approval to market the Company's products, the ability to protect its intellectual property, dependence upon collaborative partners, changes in government regulation or regulatory approval processes, and rapid technological change in the industry. Such statements are based on a number of assumptions which may prove to be incorrect, including, but not limited to, assumptions about: general business and economic conditions; the impact of changes in Canadian-US dollar and other foreign exchange rates on the Company's revenues, costs and results; the timing of the receipt of regulatory and governmental approvals for the Company's research and development projects; the availability of financing for the Company's commercial operations and/or research and development projects, or the availability of financing on reasonable terms; results of current and future clinical trials; the uncertainties associated with the acceptance and demand for new products and market competition. The foregoing list of important factors and assumptions is not exhaustive. The Company undertakes no obligation to update publicly or otherwise revise any forward-looking statements or the foregoing list of factors, other than as may be required by applicable legislation. Additional discussion regarding the risks and uncertainties relating to the Company and its business can be found in the Company's other filings with the applicable Canadian securities regulatory authorities or the US Securities and Exchange Commission, and in the "Risk Factors" section of its Form 20F for the year ended December 31, 2019.

View original content:http://www.prnewswire.com/news-releases/medicure-announces-an-agreement-with-reliance-life-sciences-for-the-marketing-rights-of-a-cardiovascular-biosimilar-301145432.html

SOURCE Medicure Inc.

Read more from the original source:
Medicure announces an agreement with Reliance Life Sciences for the marketing rights of a cardiovascular biosimilar - BioSpace

Posted in Molecular Medicine | Comments Off on Medicure announces an agreement with Reliance Life Sciences for the marketing rights of a cardiovascular biosimilar – BioSpace

The secretive group at the center of the nation’s largest vaccine trials – WKTV

Posted: October 5, 2020 at 2:51 pm

A group you've probably never heard of holds powerful sway over which coronavirus vaccines will end up on the market.

It's known as the DSMB.

Members of a Data and Safety Monitoring Board are the only ones who get to look under the hood while a trial is ongoing. They know who has been given a Covid-19 vaccine, and who has gotten a placebo. The very doctors running the trials, the pharmaceutical companies that developed the vaccines, and even the US Food and Drug Administration don't know.

Armed with that secret, only the DSMB can monitor how safe and effective a vaccine is shaping up to be.

One word from the DSMB, and a trial can be stopped. That's what happened to the AstraZeneca trial in early September after a study participant developed neurological symptoms. Shortly after, it came to light that the same trial had been paused briefly in July for similar reasons. While the vaccine trial resumed in the UK, it is still on pause in the US.

"They're very powerful. They're key guardians of science and safety and are as important if not more important than the FDA," said bioethicist Art Caplan.

Earlier this year, the National Institutes of Health appointed a common DSMB to monitor Covid-19 vaccine clinical trials that are being funded by the federal government under Operation Warp Speed. This DSMB has 10 to 15 members with specialties including vaccine development, statistics and ethics.

It's not a glamorous or public-facing job. They're paid only a modest honorarium by the NIH -- just $200 per meeting -- and there are no press conferences, no TV interviews, no fame and no glory.

That's because members' names aren't typically revealed while trials are in progress to shield them from external pressures.

Caplan, who has served on about 20 DSMBs, said there's a good reason members' names are kept secret.

"You wouldn't want some investor calling a DSMB member and saying 'Hey, how's this clinical trial looking? If you tell me, I'll give you 10% of whatever I make,'" said Caplan.

Carrie Wolinetz, associate director for science policy at the National Institutes of Health, said various types of people might try to influence DSMB members.

"It doesn't have to be nefarious. Parents of a very ill child might be anxious about how the trial of a drug that could help their child is going, and they might contact the folks at the DSMB. Keeping their names private is a way to preserve independence of the group," she said.

There's a lot at stake. They scrutinize the data carefully. One word from them, and a vaccine's chances of coming to market could be squashed. Millions of dollars spent on research and development could all be for naught.

While there are good arguments for secrecy, Caplan said he disagrees with the confidentiality that currently shrouds the DSMBs for Covid-19 vaccine candidates.

"We need to know if we can trust the vaccine, so the more transparency the better," Caplan said.

In order to reach population immunity through a vaccine, a large proportion of the US public needs to get vaccinated. But confidence in a potential vaccine is low -- 49% of Americans say they definitely or probably would not get a vaccine if one were available now, according to a recent poll by the Pew Research Center.

"We want to know they're fully independent, that they have no prior relationships with the company. So they're not conflicted in any way," said Dr. Eric Topol, professor of molecular medicine at Scripps Research. "We want to know about their expertise. It's important to know who they are."

The job of the DSMB, as the name suggests, is to monitor the data that comes out of clinical trials.

In clinical trials, there can be thousands, or tens of thousands, of study participants. Some are randomly assigned to receive an intervention -- in this case, the vaccine -- and some receive a placebo.

The studies are what's called "double-blinded." The participants don't know which they're getting, and neither do the doctors running the trials.

If a study volunteer has what appears to be a side effect or "adverse event," the DSMB can look and see if they received the vaccine or the placebo.

"If it was a placebo, then it's one of these random things," Susan Ellenberg, a member of Covid-19-related DSMBs, told CNN's Chief Medical Correspondent Dr. Sanjay Gupta. "If it was the vaccine, it could still have been a random thing. But then people have to wring their hands and try and consider how likely is it that the vaccine could cause this kind of event?"

If these events are concerning enough, the DSMB can recommend that the trial be stopped for safety reasons. The stakes are especially high in Covid-19 vaccine trials, which may ultimately be administered to millions of healthy people -- unlike drug trials intended for those who are already sick and may have few options.

"Even an adverse event that happens as infrequently as one in 10,000 people or one in 20,000 people -- that would be a lot of people who would have a serious adverse event," explained Ellenberg, a professor of biostatistics at the Perelman School of Medicine at the University of Pennsylvania.

At pre-determined intervals, the DSMB also checks on efficacy. If people receiving the vaccine get sick roughly as often as those who get the placebo, that's not a good sign. The board can recommend that the trial be stopped due to "futility."

They may also look at the quality of the data, Ellenberg said. If there's missing data, participants who drop out, or if the trial is being conducted poorly, it's the DSMB that can weigh in.

"Most of the time, a data monitoring committee will say, 'Everything looks fine, keep going,' " Ellenberg said. "But sometimes -- you never know when ... a hard decision is going to have to be made. And that's the value of these committees."

Conversely, if it looks like the vaccine is working exceptionally well, the DSMB may recommend that the study sponsor submit an application to the FDA before the official end of the trial, in order to get it more quickly to market.

"The people who serve on these committees are thoroughly vetted for conflicts of interest," Ellenberg said.

Members are screened to make sure they don't have a financial interest in the pharmaceutical company that's sponsoring the vaccine trial.

"DSMB members or their family members should have no professional, proprietary, or financial relationship with the sponsoring companies," according to a statement from the National Institute of Allergy and Infectious Diseases, which organized the common DSMB for the Covid-19 vaccine candidates under Operation Warp Speed -- including Moderna, AstraZeneca and Johnson & Johnson. "Selected DSMB members and their family members were not allowed to work for other companies developing COVID-19 vaccines."

Topol, of Scripps Research, said it's "unprecedented to have a DSMB with that much authority." Typically, each clinical trial has its own DSMB.

Such is the case with Pfizer, whose trial is not neither under the common DSMB nor funded by the government. Pfizer's DSMB comprises "a chairperson and 4 additional members that meets on a weekly basis," according to a spokeswoman.

Topol considers that small for a trial that aims to enroll up to 44,000 participants. "The trials that I ran always had six or seven at least, sometimes eight or nine," he said. "In large trials, you got to have a bioethicist, virologist, an immunologist, epidemiologist... You have all the critical areas covered."

It's a big honor to be named to a DSMB.

But it's a no-no to brag about it, as one university recently found out.

The university proudly posted that one of its professors was named chair of the DSMB for the government-supported trials of coronavirus vaccines.

When CNN called to ask why the professor was publicly identified, the university quickly removed the press release.

"It looks like a staff member shared that news and was unaware that it was not for public consumption," a university spokesperson wrote to CNN.

CNN is not revealing the professor's name or the name of the university.

Despite the lack of public recognition, fame and glory, Ellenberg says there's plenty of motivation to serve on these boards.

"You feel a great responsibility when you're on these trials," she said. "Everybody's trusting you with these data."

She remains faithful in the DSMB process. If it goes as it's supposed to, "I would take the vaccine myself, and I would recommend that other people take it," she said.

Still, downstream from the DSMB, Ellenberg acknowledges "we're in uncharted territory."

Last week, President Trump claimed the White House can overrule the FDA's attempt to toughen its Covid-19 vaccine guidelines -- guidelines that could push hopes of a vaccine authorization past Election Day.

"It never occurred to anybody that anybody outside the FDA would would try and interfere with that," Ellenberg said. "And I'm hopeful that they won't."

More here:
The secretive group at the center of the nation's largest vaccine trials - WKTV

Posted in Molecular Medicine | Comments Off on The secretive group at the center of the nation’s largest vaccine trials – WKTV

News > Science > Understanding the way proteins shapeshift – University of Leeds

Posted: October 5, 2020 at 2:51 pm

The University of Leeds has secured a 5.4 million grant to identify new techniques for investigating and manipulating the chemical building blocks of life - proteins.

The five-year project - in collaboration with the University of Oxford - will lead to a better understanding of fundamental biochemical processes and will identify new research strategies for tackling cancer and other diseases.

Andy Wilson, Professor of Organic Chemistry in the School of Chemistry at Leeds and principal investigator, said: The goal of this project is not only to get a better understanding of the way proteins work - but to establish how to disarm the rogue behaviour of some proteins that lead to disease.

Why proteins are so important

Proteins are the chemical workhorses that give cells their shape, structure and function.They are large molecules which can perform a range of cellular operations.

Although many regions of proteins adopt a fixed 3-D structure, many proteins found in the human body are able to change shape. This shape-shifting enables proteins to perform a range of different functions at different times.

The shape-shifting is linked to a part of the protein structure known as an intrinsically disordered region (IDR). The IDR changes shape and therefore its local interactions, dependent on the role it has in the cell at that particular moment.

The focus of the research project is to understand the mechanisms by which groups of IDRs change shape, revealing the role they play in a healthy cell and in the development of disease.

Understanding Aurora-A

The research project will investigate how a protein called Aurora-A is controlled by interactions involving IDRs. Aurora-A plays a role in several cellular processes that are relevant to human disease, including cell division, gene expression and the function of a hair-like projection from the cell surface called the primary cilium.

The involvement of Aurora-A in each process is dependent on a different shape-shifting protein interacting with it, but it is unclear how most of these interactions serve to control Aurora-A or how these different roles are coordinated.

Aurora-A is of major interest of Richard Bayliss, Professor of Molecular Medicine at Leeds and a co-investigator on the research project.

Professor Bayliss said: My team and others have been studying these individual interactions one-by-one for a long time.

This transformative project will enable us to understand how they fit together to produce a network that governs Aurora-As many cellular roles.

Professor Wilson added: We will deepen our understanding of the way Aurora-A is affected by changes in the shape of the proteins that interact with it.

Our aim is to develop new chemical and biological tools that will allow us to regulate the interaction of specific shape shifting proteins so we can identify the role they play in controlling Aurora-A.

By establishing the molecular processes that are most relevant to disease development and which shape-shifting proteins control these processes, targeted drug discovery efforts could be developed.

Collaborative research

In addition to ProfessorWilson and ProfessorBayliss, the research team includes other investigators based at Leeds who bring a wide range of expertise to the project: Dr Megan Wright, Dr Takashi Ochi, Dr Darren Tomlinson and Professor Sheena Radford, from the Astbury Centre for Structural Molecular Biology,andProfessorColin Johnson, in the School of Medicine.The final team member is Dr Fanni Gergely, a senior researcher and a leading cancer cell biologist based at the University of Oxford.

Professor Bayliss said: To tackle challenging scientific projects needs great team work, and we are fortunate to have so many outstanding colleagues who will contribute to our effort.

The academic scientists will work closely with industrial partners AstraZeneca and LifeArc to ensure rapid translation of research findings into drug discovery.

The researchers believe a better understanding of IDRs will have impact beyond cancer biology in many areas of biology including regulation of crop growth, cardiovascular biology and ageing.

Further information

Image credit: Professor Richard Bayliss. Illustration shows Aurora-A (light blue) and its three protein partners.

For further details, please contact David Lewis in the University of Leeds press office: d.lewis@leeds.ac.uk

Go here to read the rest:
News > Science > Understanding the way proteins shapeshift - University of Leeds

Posted in Molecular Medicine | Comments Off on News > Science > Understanding the way proteins shapeshift – University of Leeds

New COVID-19 Test Doesn’t Use Scarce Reagents, Catches All But the Least Infectious – Newswise

Posted: October 5, 2020 at 2:51 pm

Newswise A major roadblock to large scale testing for coronavirus infection in the developing world is a shortage of key chemicals, or reagents, needed for the test, specifically the ones used to extract the viruss genetic material, or RNA.

A team of scientists at the University of Vermont, working in partnership with a group at the University of Washington, has developed a method of testing for the COVID-19 virus that doesnt make use of these chemicals but still delivers an accurate result, paving the way for inexpensive, widely available testing in both developing countries and industrialized nations like the United States, where reagent supplies are again in short supply.

The method for the test, published on Oct. 2 in PLOS Biology, omits the step in the widely used reverse transcription polymerase chain reaction (RT-PCR) test where the scarce reagents are needed.

92% accuracy, missing only lowest viral loads

The accuracy of the new test was evaluated by a team of researchers at the University of Washington led by Keith Jerome, director of the universitys Molecular Virology Lab, using 215 COVID-19 samples that RT-PCR tests had shown were positive, with a range of viral loads, and 30 that were negative.

It correctly identified 92% of the positive samples and 100% of the negatives.

The positive samples the new test failed to catch had very low levels of the virus. Public health experts increasingly believe that ultra-sensitive tests that identify individuals with even the smallest viral loads are not needed to slow spread of the disease.

It was a very positive result, said Jason Botten, an expert on pathogenic RNA viruses at the University of Vermonts Larner College of Medicine and senior author on the PLOS Biology paper. Bottens colleague Emily A. Bruce is the papers first author.

You can go for the perfect test, or you can use the one that's going to pick up the great majority of people and stop transmission, Botten said. If the game now is focused on trying to find people who are infectious, there's no reason why this test shouldn't be front and center, especially in developing countries where there are often limited testing programs because of reagent and other supply shortages.

Skipping a step

The standard PCR test has three steps, while this simpler version of the standard test has only two, Botten said.

In step 1 of the RT-PCR test, you take the swab with the nasal sample, clip the end and place it in a vial of liquid, or medium. Any virus on the swab will transfer from the swab into the medium, he said. In step 2, you take a small sample of the virus-containing medium and use chemical reagents, the ones that are often in short supply, to extract the viral RNA. In step 3, you use other chemicals to greatly amplify any viral genetic material that might be there. If virus was present, youll get a positive signal.

The new test skips the second step.

It takes a sample of the medium that held the nasal swab and goes directly to the third, amplification step, Botten said, removing the need for scarce RNA extraction reagents as well as significantly reducing the time, labor and costs required to extract viral RNA from the medium in step 2.

Botten said the test is ideally suited to screening programs, in both developed and developing countries, since it is inexpensive, takes much less processing time and reliably identifies those who are likely to spread the disease.

Its low cost and efficiency could extend testing capacity to groups not currently being tested, Botten said, including the asymptomatic, nursing home residents, essential workers and school children. The standard RT-PCR test could be reserved for groups, like health care workers, where close to 100% accuracy is essential.

An influential pre-print points way to widespread adoption of test

The two-step test developed by the University of Vermont team first caught the attention of the scientific community in March, when preliminary results that accurately identified six positive and three negative Vermont samples were published as a preprint in bioRxiv, an open access repository for the biological sciences. The preprint was downloaded 18,000 times in its first week, it ranked 17th among 15 million papers the site had published and the abstract was viewed 40,000 times.

Botten heard from labs around the world who had seen the preprint and wanted to learn more about the new test.

They said, I'm from Nigeria or the West Indies. We can't test, and people's lives are at stake. Can you help us?

Botten also heard from Syril Pettit, the director of HESI, the Health and Environmental Sciences Institute, a non-profit that marshals scientific expertise and methods to address a range of global health challenges, who had also seen the preprint.

Pettit asked Botten to join a think tank of likeminded scientists she was organizing whose goal was to increase global testing capacity for COVID-19. The test developed by the University of Vermont and University of Washington teams would serve as a centerpiece.

To catalyze a global response, the group published a call to action in EMBO Molecular Medicine.

And it took action, reaching out to 10 laboratories in seven countries, including Brazil, Chile, Malawi, Nigeria and Trinidad/Tobago, as well as the U.S. and France, to see if they would be interested in giving the two-step test a trial run.

Universally, the response was yes, Pettit said.

The outreach led to a new HESI program called PROPAGATE. Each of the labs in the PROPAGATE Network will use the two-step test on a series of positive and negative samples sent to them by the University of Washington to see if they can replicate the results the university achieved.

The study has already shown promising results. One of the labs in Chile has also used the test on its own samples from the community and got accurate results.

Assuming all goes well, Pettit and her colleagues at the University of Vermont and the University of Washington as well as scientists from the 10 partner sites plan to publish the results.

The goal is the make the two-step test accessible to any lab in the world facing these hurdles and see a broad uptake, she said.

See more here:
New COVID-19 Test Doesn't Use Scarce Reagents, Catches All But the Least Infectious - Newswise

Posted in Molecular Medicine | Comments Off on New COVID-19 Test Doesn’t Use Scarce Reagents, Catches All But the Least Infectious – Newswise

Nuclear Medicine/Radiopharmaceuticals – Global Market Forecast to 2027: Production of Radiopharmaceuticals from Cyclotrons Gaining Momentum -…

Posted: October 5, 2020 at 2:51 pm

DUBLIN--(BUSINESS WIRE)--The "Nuclear Medicine/Radiopharmaceuticals Global Market - Forecast To 2027" report has been added to ResearchAndMarkets.com's offering.

The nuclear medicine global market is poised to grow at a high single digit CAGR from 2020 to 2027 to reach $10,742.7 million by 2027.

Over the past 50 years, the nuclear medicine field has displayed a strong link between investments in chemistry and the development of radionuclide and radio-labeled compounds which have impacted the healthcare practice. Nuclear medicine comprises diagnostic and therapeutic techniques that use radioisotopes for applications like oncology, cardiovascular and neurological disorders to provide information at both molecular and cellular levels for probing, tracking tissue function, study disease progression and assessing treatment responses.

Increasing radioisotopes applications, rise in public awareness, use of SPECT/CT and PET/CT imaging scans, the abundance of radiopharmaceuticals, advancement in imaging technology (hybrid imaging) and alpha therapy based targeted cancer treatment is boosting nuclear medicine market growth. In addition, increasing need in emerging markets, production of radiopharmaceuticals from cyclotrons, efficient diagnosis and treatments, emerging radio isotopes and replacement of old/traditional equipment are the opportunities likely to propel the growth of the nuclear medicine market.

The nuclear medicinal market is classified based on modality into diagnosis and therapeutics. The diagnostics market commanded the largest market revenue in 2020 and is expected to grow at a mid single digit CAGR from 2020 to 2027 due to an increase in SPECT and PET procedures. The therapeutics segment is projected to grow at high teen CAGR from 2020 to 2027 due to technological advancements in the targeted treatment of cancers.

Potential new radioisotopes in the pipeline and advancement in neurological treatments are the key factors driving the growth of the therapeutics market. Diagnosis by products is segmented into SPECT and PET. SPECT market commanded the largest revenue in 2020 and is expected to grow at low single digit CAGR from 2020 to 2027 due to an increase in TC-99m isotope applications and product approvals.

Among SPECT is segmented based on isotopes into Technetium (Tc-99m), Thallium (Tl-201), Gallium (Ga-67), Iodine (I-123), Samarium (Sm-153), Xenon (Xe-133), Rhenium (Re-186) and others. Technetium (Tc-99m) accounted for the largest share in 2020 and is projected to grow at a mid single digit CAGR from 2020 to 2027 due to its extensive usage in various diagnostic applications and emerging sources to meet the demand. SPECT market by application is segmented into cardiology, pulmonary, oncology, nephrology, neurology, inflammation, thyroid gland, lymphology and others.

Cardiology accounted for the largest share in 2020 and is expected to grow at mid single digit CAGR from 2020 to 2027 due to an increase in the number of cardiac imaging cases using Tc-99m. Oncology is expected to grow at mid single digit CAGR from 2020 to 2027 due to increasing expanding usage in early screening tests in vulnerable populations in various developed countries.

PET is the fastest-growing segment with mid single digit CAGR from 2020 to 2027 due to an increase in the adoption of cyclotron for the production of PET isotopes increasing its availability. The PET isotopes include Fluorodeoxyglucose (18F-FDG), Gallium (Ga-68), Rubidium (Rb-82) and others.

Fluorodeoxyglucose (18F-FDG) accounted for the largest share in 2020 and the market is expected to grow at mid single digit CAGR from 2020 to 2027. Gallium (Ga-68) is expected to grow at high double digit CAGR from 2020 to 2027 due to an increase in usage as theranostic pair in assessing the suitability of patient for Lutathera and many emerging targeted radiotherapy agents.

PET by applications is segmented into cardiology, oncology, neurology, inflammation and others. Oncology accounted for the largest share in 2020 and is projected to grow at high single digit CAGR from 2020 to 2027 due to an increase in the patient pool of lung, thyroid, brain breast cancer and dementia related conditions.

Some of the key players of the nuclear medicine market are

Key Topics Covered:

1 Executive Summary

2 Introduction

3 Market Analysis

4 Nuclear Medicine Global Market, by Modality

5 Nuclear Medicine Global Market, by End-Users

6 Stable Isotopes

7 Nuclear Medicine Global Market by Region

8 Competitive Landscape

9 Major Player Profiles

For more information about this report visit https://www.researchandmarkets.com/r/so14du

Read this article:
Nuclear Medicine/Radiopharmaceuticals - Global Market Forecast to 2027: Production of Radiopharmaceuticals from Cyclotrons Gaining Momentum -...

Posted in Molecular Medicine | Comments Off on Nuclear Medicine/Radiopharmaceuticals – Global Market Forecast to 2027: Production of Radiopharmaceuticals from Cyclotrons Gaining Momentum -…

Smith’s 2005 stem cell law to be reauthorized by House – InsiderNJ

Posted: October 4, 2020 at 6:00 am

Smiths 2005 stem cell law to be reauthorized by House

Rep. Chris Smith (R-NJ)statement submitted during debate in the House of Representatives

on the Stem Cell Therapeutic and Research ActSeptember 29, 2020

Margaret Hahnmy mother-in-lawpassed away on Friday and a Mass of Christian burial will be held today at St. Mary Church in South Amboy, New Jersey. She was 96 and was deeply loved and will be deeply missed.

MargaretPegwas a great womanwife, mother, grandmother, and great-grandmother. She selflessly devoted her life to public service including her amazing work as Sayreville Borough Clerk for twenty years. She had an incredible reputation for getting things done for the people. No matter who served as mayor or on Council, everyone knew she was the power.

My wife Marie and I will join family and friends today at her funeral and internment making it impossible for me to speak today during the debate on the reauthorization of a law I originally authored fifteen years agothe Stem Cell Therapeutic and Research Act of 2005and the Stem Cell Therapeutic and Research Act of 2015.

So, I submit these comments for the Congressional Record.

Madam Speaker, today the House of Representatives will vote to reauthorize the Stem Cell Therapeutic and Research Act.

This was an original idea of mine 20 years ago. Joined by 70 cosponsors, I introduced it in 2001 and again in 2003.

After five long years of hard work and numerous setbacks, my bill was finally enacted into law in 2005.

Beginning in 2001, Dr. Joanne Kurtzberg, who is President of the Cord Blood Association, helped draft my original law.Dr. Kurtzberg has said, Cord blood transplantation is now an established field with enormous potential. In the future, it may emerge as a source of cells for cellular therapies focused on tissue repair and regeneration.

The new law created a nationwide umbilical cord blood stem cell program, designed to collect, derive, type, and freeze cord blood units for transplantation into patients to mitigate and to even cure serious disease. Pursuant to the law, it also provided stem cells for research. The new cord blood program was combined in our 2005 law with an expanded bone marrow initiative, which was crafted over several years by our distinguished colleague, CongressmanBill Young.

I was the prime sponsor again when it was reauthorized in 2015.

Umbilical cord blood stem cells, obtained after the birth of a child, have proved highly efficacious in treating 70 diseases, including sickle-cell disease, lymphoma, and leukemia. And scientists are continuing to study and better understand the regenerative effects of cord blood cell therapies for other diseases and conditions. Bone marrow donations provide lifesaving transplants to treat diseases like blood cancer, sickle cell anemia, or inherited metabolic or immune system disorders.

The National Cord Blood Inventory (NCBI) provides funding to public cord blood banks participating in the program to allow them to expand the national inventory of cord blood units available for transplant. These units are then listed on the registry by the Be the Match Program. The funds appropriated thus far have led to an important increase in the overall number of high-quality cord blood units available through the national registry, including 150,000 NCBI units. Within the Be the Match registry, there are more than 783,000 NCBI units worldwide.

The Program registry allows patients and physicians to locate matching cord blood units, as well as adult donors for marrow and peripheral blood stem cells, when a family donor is not available. The Program is the worlds largest, most diverse donor registry, with more than 22 million volunteers and more than 300,000 public cord blood units. To date, the National Marrow Donor Program/Be The Match (NMDP), through its operation of the Program, has facilitated more than 100,000 transplants. More than 45,000 patients have receivedcord bloodtransplants, according Dr. Joanne Kurtzberg.

The reauthorization before us authorizes $23 million to be appropriated for fiscal year 2021 through fiscal year 2025. It also authorizes $30 million to be appropriated for fiscal years 2021 through 2025 for the bone marrow transplant program. This continues funding at the same levels authorized in the 2015 authorization bill.

Madam Speaker, each year nearly 4 million babies are born in America. In the past, virtually every placenta and umbilical cord was tossed as medical waste. Today, doctors have turned this medical waste into medical miracles.

Not only has God in His wisdom and goodness created a placenta and umbilical cord to nurture and protect the precious life of an unborn child, but now we know that another gift awaits us immediately after birth. Something very special is left behindcord blood that is teeming with lifesaving stem cells. Indeed, it remains one of the best kept secrets in America that umbilical cord blood stem cells and adult stem cells in general are curing people of a myriad of terrible conditions and diseasesover 70 diseases in adults as well as in children.

The legislation that is before us will enable even more patients to receive the treatments that they so desperately need.

(Visited 154 times, 1 visits today)

Continued here:
Smith's 2005 stem cell law to be reauthorized by House - InsiderNJ

Posted in New Jersey Stem Cells | Comments Off on Smith’s 2005 stem cell law to be reauthorized by House – InsiderNJ

This years SN 10 scientists aim to solve some of sciences biggest challenges – Science News

Posted: October 4, 2020 at 5:57 am

In the midst of a pandemic that has brought so much worry and loss, its natural to want to helpto do some small part to solve a problem, to counter pain, or to, importantly, remind others that there is beauty and wonder in the world. Scientists have long been doing just that. Many are chasing answers to the myriad challenges that people face every day, and revealing the rewards in the pursuit of knowledge itself. Its in that spirit that we present this years SN 10: Scientists to Watch.

For the sixth consecutive year, Science News is featuring 10 early- and mid-career scientists who are pushing the boundaries of scientific inquiry. Some of the researchers are asking questions with huge societal importance: How do we prevent teen suicide? What are the ingredients in wildfire smoke that are damaging to health? Is there a better way to monitor earthquakes to save lives? What about finding new ways to diagnose and treat diseases?

Others are trying to grasp how weird and wonderful the natural world isfrom exploring how many supermassive black holes are out there in space to understanding the minuscule genetic details that drive evolution. For instance, SaraH Zanders, one of this years SN10, is unveiling the drama that unfolds when life divvies up its genetic material.

A couple of the scientists on this years list have also taken steps to support people from groups that are underrepresented in the sciences. These researchers see how science benefits when people from diverse backgrounds contribute to the pursuit of answers.

Headlines and summaries of the latest Science News articles, delivered to your inbox

All of this years honorees are age 40 and under, and all were nominated by Nobel laureates, recently elected members of the U.S. National Academy of Sciences or previous SN 10 scientists. The world feels very different than it did at the start of 2020, when we first put out our call for SN 10 nominations, but the passion these scientists have for their work endures. The curiosity, creativity and drive of this crew offers hope that we can overcome some of our biggest challenges.

Though it often takes time, out of crisis comes action. Also out of crisis comes a renewed appreciation for small pleasures that give life meaning. These researchers find joy in the search for scientific answers. Heres how Zanders describes what motivates her work: Its just I like to solve puzzles. ElizabethQuill

Affiliation: Dartmouth CollegeHometown: Dhaka, BangladeshFavorite black hole: Cygnus X-1

Tonima Tasnim Ananna is bringing the heaviest black holes out of hiding. She has drawn the most complete picture yet of black holes across the universe where they are, how they grow and how they affect their environments. And she did it with the help of artificial intelligence.

As far as astronomers can tell, nearly every galaxy stows a black hole at its center, weighing millions or billions of times the mass of the sun. Though these supermassive black holes can heat surrounding material until it glows brighter than all the galaxys stars combined, the light can be concealed by gas and dust also drawn in by the black holes pull. High-energy X-rays cut through that dusty veil. So for her Ph.D., completed in 2019, Ananna gathered surveys from four X-ray telescopes, more datasets than any previous study had used. Her goal was to create a model of how black holes grow and change across cosmic history. It was supposed to be a short paper, Ananna says. But models that explained one or a few of the datasets didnt work for the full sample. It stumped us for some time.

To break the gridlock, she developed a neural network, a type of artificial intelligence, to find a description of the black hole population that explained what all the observatories saw. She just went off and taught herself machine learning, says astrophysicist Meg Urry of Yale University, Anannas Ph.D. adviser. She doesnt say, Oh, I cant do this. She just figures out a way to learn it and do it. One early result of the model suggests that there are many more active black holes out there than previously realized.

Black holes could be gobbling down gas as fast as theoretically possible.

Galaxies live and die by their black holes. When a black hole puts out energy into the galaxy, it can cause stars to form, Ananna says. Or it could blow gas away, shutting down star formation and stunting the galaxys growth (SN: 3/31/20). So understanding black holes is key to understanding how cosmic structures everything from galaxy clusters down to planets and perhaps even life came to be. Anannas model is built on data describing black holes at different cosmic distances. Because looking far in space is like looking back in time, the model shows how black holes grow and change over time. It could also help figure out how efficiently black holes eat. Early hints suggest black holes could be gobbling down gas as fast as theoretically possible, which may help explain how some got so big so fast (SN: 3/16/18).

When Ananna was a 5-year-old in Dhaka, Bangladesh, her mother told her about the Pathfinder spacecraft landing on Mars. Her mother was a homemaker, she says, but was curious about science and encouraged Anannas curiosity, too. Thats when I realized there were other worlds, she says. Thats when I wanted to study astronomy. There were not a lot of opportunities to study space in Bangladesh, so she came to the United States for undergrad, attending Bryn Mawr College in Pennsylvania. She chose an all-womens school not known for a lot of drinking to reassure her parents that she was not going abroad to party. Although Ananna intended to keep her head down and study, she was surprised by the social opportunities she found. The women at Bryn Mawr were fiercely feminist, articulate, opinionated and independent, she says. It really helped me grow a lot. Traveling for internships at NASA and CERN, the European particle physics laboratory near Geneva, and a year at the University of Cambridge, boosted her confidence. (She did end up going to some parties no alcohol for me, though.)

Now, Ananna is giving back. She cofounded Wi-STEM (pronounced wisdom), a mentorship network for girls and young women who are interested in science. She and four other Bangladeshi scientists who studied in the United States mentor a group of 20 female high school and college students in Bangladesh, helping them find paths to pursue science. LisaGrossman

Affiliation: Texas Tech UniversityHometown: Rome, ItalyFavorite telescope: Very Large Array, New Mexico

On September 3, 2017, Alessandra Corsi finally saw what she had been waiting for since mid-August: a small dot in her telescope images that was the radio afterglow of a neutron star collision. That stellar clash, discovered by the Advanced Laser Interferometer Gravitational-Wave Observatory team, or LIGO, which included Corsi, was the first direct sighting of a neutron star collision (SN: 10/16/17). The event, dubbed GW170817, was also the first of any kind seen in both gravitational waves and light waves.

Telescopes around the world spotted all kinds of light from the crash site, but one particular kind, the radio waves, took their sweet time showing up. Corsi had been waiting since August17, when the gravitational waves were spotted. Longest two weeks of my life, Corsi says. The radio waves were key to understanding a superfast particle jet launched by the colliding stars.

Early on, the jet appeared to have been smothered by a plume of debris from the collision (SN: 12/20/17). But follow-up radio observations made by Corsis team and others confirmed that the jet had punched through the wreckage (SN: 2/22/19). This jet was the first of its kind to be seen from the side, allowing Corsi and colleagues to probe its structure. The jet almost certainly would have gone unnoticed if the gravitational waves hadnt clued astronomers in.

Corsi is a pioneer in the new field of multimessenger astronomy, which pairs observations of light waves with spacetime ripples, or gravitational waves. The pairing is like having eyes and ears on the cosmos, Corsi says. You cannot learn all that you could with only one of the two. In the case of GW170817, gravitational waves revealed how the neutron stars danced around each other as they spiraled toward collision, and light waves unveiled the type of material left in the aftermath (SN: 10/23/19). Using this multimessenger approach could also give astronomers a more complete picture of other cataclysms, such as smashups between neutron stars and black holes, and the explosive deaths of massive stars. Such spectacular events reveal some of the most fundamental physics in our universe, Corsi says.

If gravitational wave signals were converted into sound, they would create their own kind of music.

Most researchers specialize in either gravitational waves or light, but Corsi is very well-versed in both messengers, says Wen-fai Fong, an astrophysicist at Northwestern University in Evanston, Ill. That makes her extremely versatile in terms of the types of multimessenger science she can study.

Corsi has now built a computational tool to scan LIGO data for gravitational waves stirred up by whatever is left behind in a neutron star merger. The tool is based on a paper she published in 2009 years before LIGO scored its first gravitational wave detection (SN: 2/11/16). The paper describes the gravitational wave pattern that would signal the presence of one possible remnant: a rapidly spinning, elongated neutron star. Alternatively, a neutron star smashup could leave behind a black hole. Knowing which tells us a lot about how matter behaves at densities way higher than we could ever explore in a lab, Corsi says.

Corsi taught herself to play the piano in high school, and now enjoys playing both classical music and tunes from favorite childhood movies, like Beauty and the Beast. The audio frequencies of piano notes are similar to the frequencies of spacetime tremors picked up by LIGO. If gravitational wave signals were converted into sound, they would create their own kind of music. Thats the thing I like to think of when Im playing, she says. MariaTemming

Affiliation: Colorado State UniversityHometown: Richmond, R.I.Favorite outdoor activities: Cross-country skiing and gardening

Emily Fischer has always cared about air pollution. Its innate. Its a calling, she says. Exposure to air pollution raises your risk for many common ailments, such as cardiovascular disease, asthma, diabetes and obesity. But unlike some other risk factors for these diseases, you cant choose not to breathe, right? You have to have clean air for everyone. In her youth, she organized rallies to clean up the cigarette smokefilled air of her Rhode Island high school. That interest led Fischer to study atmospheric chemistry and motivates her current work as a self-described air pollution detective. Air pollution may conjure images of thick black plumes billowing from smokestacks, but Fischer says most air pollution is invisible and poorly understood. She combines analytical chemistry with high-flying techniques to understand where air pollution comes from and how it changes as it moves through the air.

Wildfire smoke like that filling the skies in the American West this season is a major, but still mysterious, source of air pollution. Thousands of different solids, liquids and gases swirl together to form wildfire smoke, and its chemical composition changes as it blows through the atmosphere. This dynamic mixture, which is also affected by whats burning on the ground, is tricky to measure, since each of its many components requires highly specialized equipment and expertise to assess. The equipment also has to be airborne, typically lofted into the air via planes or balloons. There has been beautiful work on wildfire smoke, Fischer says, but in most studies, we just have not had all the measurements needed to really interpret things.

You cant choose not to breathe, right? You have to have clean air for everyone.

To get a fuller view, she dreamed big: Why not try to measure everything, and measure it systematically? She pulled together a diverse team of 10 lead researchers, and scores more graduate students and postdocs, to pull off the most comprehensive analysis of wildfire smoke ever attempted, a project dubbed WE-CAN. During the summer of 2018, Fischer led over a dozen six-hour flights over the West, chasing wildfire smoke plumes and systematically measuring the air in and around smoke plumes with nearly 30 different instruments crammed into the cargo hold of a C-130 plane.

[WE-CAN] is a big collaboration, says Ronald Cohen, an atmospheric chemist at the University of California, Berkeley. He says success stemmed in large part from the team that came together.

Making an environment for successful collaboration is really satisfying to me, Fischer says.

While team members are still analyzing the data, the project is already revealing some of the smokes secrets. For example, formaldehyde and hydrogen cyanide two chemicals linked to cancer and other health problems are abundant in wildfire smoke. Recent wildfires show how important it is to understand the role of climate change in fires, Fischer says, and who is most vulnerable in our society, and how we can best prepare and protect those communities.

Fisher is also planning to adapt some of what shes learned from WE-CAN to track ammonia emissions from farms and feed lots, which are another major source of air pollution.

Fischer is deeply committed to bringing more undergraduate women, especially women of color, into the geosciences. And shes using science to figure out how. She brought a team of social scientists and geoscientists together to study how different interventions can help. She and colleagues found that for every female role model a student has, her probability of continuing on in her geosciences major roughly doubles. Having someone to look up to who looks like them is key to building a sense of belonging and identity as a scientist, Fischer says. To help build that network, Fischer started PROGRESS, a workshop and mentorship program that aims to support undergraduate women in the geosciences. Started at Colorado State University in 2014, the program has since expanded, reaching over 300 women at institutions across the United States.

For her own mentees, Fischer tries to instill a willingness to take risks and go after big, bold questions. The easy things are done, she says. Pushing forward our understanding of pressing questions means chasing research projects that might lead nowhere, she says, or might crack open a new field of research. Its OK to be wrong, and its OK to take risks. Thats what science needs right now. JonathanLambert

Affiliation: University of Illinois at Urbana-ChampaignHometown: Mumbai, IndiaFavorite element:Gold

Prashant Jain explores how light interacts with matter such as how plants use sunlight to photosynthesize and applies that knowledge to new problems. He recently took lessons from nature to convert carbon dioxide into other useful molecules. In a paper last year in Nature Communications, Jain and Sungju Yu, also at Illinois at the time, reported using gold nanoparticles as a catalyst to drive chemical reactions between carbon dioxide and water.

When light hit the nanoparticles, it set off a series of reactions that converted carbon dioxide into hydrocarbon fuels such as methane and propane. In essence, the process not only sucked carbon dioxide a greenhouse gas out of the air, but it also made that carbon into fuel. No wonder the oil giant Shell is funding Jains work. The whole process isnt very efficient, so Jain is working to improve how much carbon dioxide gets used and how much fuel gets produced. But along the way he hopes to learn more about how nature uses energy to make matter and to inspire his lab to create more sustainable and renewable energy technologies.

I am myself still a student.

In another example of using chemistry to push toward future technologies, Jain and colleagues shined light on gold and platinum nanoparticles and triggered reactions that liberated hydrogen from ammonia molecules. Hydrogen is important in many industries fuel cells for zero-carbon vehicles use it, for example but it can be dangerous to transport because its flammable. Jains discovery could allow workers to transport ammonia instead, which is safer, and then free the hydrogen from the ammonia once it has arrived wheres it needed. The work was reported online in July in Angewandte Chemie.

Jain has a remarkable ability and optimism to see unsuccessful laboratory experiments as successful steps toward understanding the natural world, says Karthish Manthiram, a chemical engineer at MIT. As a first-year graduate student at the University of California, Berkeley, Manthiram remembers being frustrated that his experiments werent turning out as expected. But Jain, a postdoctoral fellow in the same lab, stepped in to helpand recast the problematic results. Hes always viewed what others see as failure as moments of clarity that build up to moments when things make more sense, Manthiram says. For me that was an important lesson in how to be a scientist.

Growing up in a family that worked mostly in business and finance, Jain fell in love with science as a preteen inspired in part by watching the movie Jurassic Park and its fictional depiction of what might be possible through understanding the molecular world. Soon he spotted a physics textbook for sale from a street vendor and bought it. I tried to read the book, nothing much made sense, he says. I wanted to be the one to figure out all these mysteries of nature. He chose to major in chemical engineering in college (inspired in part by a magazine published by the chemical company DuPont), and then switched to physical chemistry when he moved to the United States to get a Ph.D.

Promoted this year to full professor, Jain has never stopped pushing to acquire new knowledge; when he finished teaching this last spring semester, he enrolled in an online MIT course on quantum information science. I am myself still a student, he says. AlexandraWitze

Affiliation: Indiana UniversityHometown: Houston, TexasFavorite fieldwork: Observing rituals

Between 2000 and 2015, at a high school of about 2,000 students in the town of Poplar Grove (a pseudonym), 16 former and current students died by suicide; three other similar-aged individuals in the community, mostly at private schools, also took their own lives. A clinician who had grown up in the town reached out to Anna Mueller for help breaking the cruel cycle. Before that e-mail in fall 2013, Mueller was using big data to understand why teen and young adult suicide rates in the United States were spiking. The U.S. Centers for Disease Control and Prevention estimates that suicides among 10- to 24-year-olds jumped 56 percent between 2007 and 2017.

Scholars theorized that suicidal people attracted other suicidal people. But Muellers work undercut that idea. In 2015 in the Journal of Health and Social Behavior, for instance, she reported that merely having a suicidal friend did not increase a teens suicide risk. A teens risk only went up with awareness that a teenage friend had made a suicide attempt. Knowledge of the attempt matters to transformingrisk, Mueller says. She carried an understanding of that contagion effect to Poplar Grove, where she worked with sociologist Seth Abrutyn of the University of British Columbia in Vancouver, the half of the duo who is more focused on the theoretical.

Anna Muellers long-term goal is to create a sort of litmus test that identifies schools that could be at risk of a suicide cluster.

The team conducted 110 interviews and focus group meetings, lasting from 45 minutes to four hours, with Poplar Grove residents, plus some individuals outside the community for comparison. The teams research revealed that teens felt an intense pressure to achieve in their affluent, mostly white town, where everybody seemed to know everyone else. While teens and young adults in a first wave of suicides might have had mental health problems, peers and community members often attributed those deaths to the towns pressure cooker environment. That narrative, however incomplete, was especially strong when the youth who killed themselves were classic overachievers. Tragically, over time, that script became embedded in the local culture, making even youth who werent previously suicidal see suicide as a viable option (SN: 4/3/19), Mueller says.

Mueller and Abrutyn were among the first researchers to start chipping away at the underlying reasons for why suicide rates have been rising in high schoolers, particularly overachieving girls without obvious underlying mental health problems, says Bernice Pescosolido, a sociologist at Indiana University in Bloomington who helped bring Mueller into the schools sociology department. What Anna and Seth have really been able to show is how imitation works and what the contagion effect looks like on the ground.

Muellers long-term goal is to create a sort of litmus test that identifies schools that could be at risk of a suicide cluster. That way, school and community leaders can intervene before the first suicide and its resulting firestorm. Since fall 2018, she has been researching suicide trends in school districts in Colorado that are more diverse than Poplar Grove. When it comes to school culture, her early work shows, theres often a trade-off between academic or athletic excellence and a supportive environment.

In anticipation of her work in Poplar Grove, Mueller knew she needed a more boots-on-the-ground approach than her big data training allowed. So she trained in qualitative methods, including how to design a study; interview techniques, such as how to write questions to elicit desired conversations; and the detailed data analysis required for this research tactic.

Mueller also sees the value in observing interactions, a common sociological approach. This spring, with the pandemic in full swing, she spent a lot of time on her home computer watching socially distant graduation ceremonies in her Colorado schools. She found that a schools culture showed in the details, such as whether valedictorians addressed hot-button issues, such as the Black Lives Matter movement, in their speeches. Of all of my moments in the field, rituals are the ones that tug at my own heartstrings because Im watching kids graduate and thats just inherently beautiful, but it also is a very powerful data moment, she says. SujataGupta

The National Suicide Prevention Lifeline can be reached at 1-800-273-TALK (8255).

Affiliation: MITHometown: Adelaide, AustraliaFavorite subatomic particle: The gluon

When Phiala Shanahan was a graduate student, she was shocked to learn that experiments disagreed on the size of the proton (SN: 9/10/19). Protons and neutrons are the key building blocks of 99 percent of the visible matter in the universe, she says. And we know, in some sense, surprisingly little about their internal structure.

If theres something I dont understand, Im extremely stubborn when it comes to figuring out the answer.

That ignorance inspires her studies. She aims to calculate the characteristics of protons and neutrons based on fundamental physics. That includes not just their size, but also their mass and the nature of their components how, for example, the quarks and gluons that make them up are sprinkled around inside. Such calculations can help scientists put the standard model, the theory that governs elementary particles and their interactions, to the test.

Shanahan is known for her prowess calculating the influence of gluons, particles that carry the strong force, which binds the proton together. For example, when gluons contributions are included, the proton is squeezed to a pressure greater than estimated to exist within incredibly dense neutron stars, she and a coauthor reported in Physical Review Letters in 2019. Its a very remarkable calculation, says physicist Volker Burkert of the Thomas Jefferson National Accelerator Facility in Newport News, Va. Thats very fundamental, and its the first time it has been done. Because they have no electric charge, gluons tend to elude experimental measurements, and that has left the particles neglected in theoretical calculations as well. Shanahans gluon results should be testable at a new particle collider, the Electron-Ion Collider, planned to be built at Brookhaven National Lab in Upton, N.Y. (SN: 4/18/17).

Persistence. I hate not knowing something, she says. So if theres something I dont understand, Im extremely stubborn when it comes to figuring out the answer.

A technique called lattice QCD is the foundation for Shanahans work. Its named for quantum chromodynamics, the piece of the standard model that describes the behavior of quarks and gluons. QCD should allow scientists to predict the properties of protons and neutrons from the bottom up, but the theory is incredibly complex, making full calculations impossible to perform even on the best available supercomputers. Lattice QCD is a shortcut. It breaks up space and time into a grid on which particles reside, simplifying calculations. Shanahan is leading efforts to use machine learning to rev up lattice QCD calculations putting her persistence to good use. We dont have to rely on computers getting better. We can have smarter algorithms for exploiting those computers, she says. She hopes to speed up calculations enough that she can go beyond protons and neutrons, working her way up to the properties of atomic nuclei. EmilyConover

Affiliation: CaltechHometown: Kolomna, RussiaFavorite protein: He cant pick just one

Mikhail Shapiro believes that in the future, were going to have smart biological devices that are roaming our bodies, diagnosing and treating disease something akin to the submarine in the 1966 classic sci-fi film Fantastic Voyage. As the shrunken sub entered and repaired the body of a sick scientist, commanders on the outside helped control it. Similarly, were going to want to talk to the cells that we are going to send into the body to treat cancer, or inflammation, or neurological diseases, Shapiro says.

Shapiro and his colleagues are working on building, watching and controlling such cellular submarines in the real world. Such a deep view inside the body might offer clues to basic science questions, such as how communities of gut bacteria grow, how immune cells migrate through the body or how brains are built cell by cell.

Despite his futuristic visions, Shapiro is often drawn to the past. I like science history a lot, he says. Right now, hes in the middle of rereading the Pulitzer Prizewinning The Making of the Atomic Bomb. Just before that, he read a biography of Marie Curie.

There is not a protein that I learn about that I dont think about ways to misuse it, Shapiro says. But hes especially fond of the proteins that build the outer shell of gas vesicles in certain kinds of bacteria. These microscopic air bags have so many uses that were totally unanticipated, Shapiro says.

In addition to letting bacteria sink or float, these bubbles provide a communication system, Shapiro and colleagues have found. Over the last several years, they have coaxed both bacterial cells and human cells to make gas vesicles and have placed such cells within mice. Because the air-filled pockets reflect sound, the engineered cells can be tracked from outside a mouses body. Using patterns of sound waves, the researchers can also drive bacterial cells around in lab dishes.

There is not a protein that I learn about that I dont think about ways to misuse it.

In another nod to Fantastic Voyage, scientists can weaponize these cellular submarines. Weve essentially turned cells into suicide agents triggered by ultrasound, Shapiro says. This explosion could release chemicals into the surroundings and destroy nearby cells. This sort of targeted detonation could be damaging to tumors, for instance. Complete warfare is possible, he says.

By seeing the potential in these esoteric gas vesicles, Shapiro was ahead of his time and hugely innovative, says Jason Lewis, a molecular imaging scientist at Memorial Sloan Kettering Cancer Center in New York City. I think weve only scratched the surface of what his work will do in terms of a greater impact.

Frustration, Shapiro says, is what made him switch to engineering after studying neuroscience as an undergraduate at Brown University in Providence, R.I. He realized that existing tools for studying processes inside the brain fell short. And I didnt see enough people making better tools.

But he didnt stop at developing new neuroscience technologies. Oddly enough, once I got into the engineering part of things, I got so fascinated with weird proteins, and magnetic fields, and sound waves, and all the more physics-y side of things. Thats become as much, if not more, of my passion as the original neuroscience. In his Twitter bio, Shapiro describes his expertise as succinctly as possible: Bio-Acousto-Magneto-Neuro-Chemical Engineer at Caltech. LauraSanders

Affiliation: Stanford UniversityHometown: Nanjing, ChinaFavorite organism: Planarian

Planarians are the most charismatic of all flatworms, Bo Wang says. They have this childish cuteness that people just love. But the adorable facade isnt what drew Wang to study the deceptively simple worms, which resemble little arrows with eyes. It was planarians superpower: regeneration. Slice a planarian into pieces and, within a week or two, each chunk will grow into a new flatworm head and all. Studying the cells that drive this process could offer lessons for turning on regeneration in human tissues, to treat various diseases, regrow limbs and grow organs for next-generation transplants.

Wang uses statistical physics to figure out how planarians regenerate entire organs cell by cell. Newly formed brain cells, for instance, must physically position themselves to avoid turning into amorphous aggregates, Wang says. His interest in how things fit together began in graduate school at the University of Illinois at Urbana-Champaign. There, Wang trained as a physicist and worked on self-assembling materials. Wang now works to uncover the physical rules that living cells follow. Im fascinated by how molecules arrange themselves seemingly randomly, but there are still statistical rules that those molecules will follow, he says.

Bo Wang works to uncover the physical rules that living cells follow.

His physics-based approach is raising new questions and unveiling biological processes that would be hard for biologists to come by using traditional methods alone, says regeneration biologist Alejandro Snchez Alvarado of the Stowers Institute for Medical Research in Kansas City, Mo. Wang is a new breed of flatworm biologist, Snchez Alvarado says. He is occupying a very unique niche in the community of developmental biology.

Wang and colleagues recently found that nerve cells, or neurons, in regenerating planarian brains form a predictable pattern dictated by the types of cells in their midst. Planarians brains are akin to cities made up of neighborhoods of neurons. Within each neighborhood, no two neurons that do the same job will live next to each other; those cells repulse each other but stay close enough to communicate, the researchers reported in the May Nature Physics. Because of this behavior, increasing the types of neurons in a neighborhood limits the ways cells can pack together. The team dubbed this packing process chromatic jamming, after a famous mathematical puzzle called the four-color problem (SN: 3/6/09).

The finding is surprising and challenges what we think we understand about organogenesis and about organization of cells within an organ, says Snchez Alvarado. Chromatic jamming appears to be key to how the planarian brain comes together, guiding single cells into neighborhoods that are a driving force in organ development, he says. If similar physical rules apply to human cells, that could help scientists sketch blueprints for engineering and growing artificial organs. CassieMartin

Affiliation: Stowers Institute for Medical ResearchHometown: Glenwood, IowaFavorite organism: Fission yeast

An invitation to work in the lab of her genetics professor Robert Malone at the University of Iowa in Iowa City set SaraH Zanders on the path to becoming a scientist. It was a turning point in my life, Zanders says. Before that, she didnt really know how she would put her biology degree to use, or what it meant to be a scientist. In Malones lab, she fell in love with meiosis, the process by which organisms divvy up genetic information to pass on to future generations. The first step is julienning the genome and swapping pieces of chromosomes. That just seems like such a bad idea to basically shred your [DNA] in the process of getting it from one generation to the next, she says. She started studying the proteins involved in making the cuts. It was like I was born to do that. I never would have known without that push.

A different kind of push led Zanders to spell her first name with a capital H: An elementary school teacher kept leaving the letter off. Zanders has capitalized it for emphasis ever since. If I write it without the big H, it doesnt look like my name anymore, she says. It feels like somebody else.

Meiosis is full of conflict. For her postdoctoral work, Zanders focused on a particular type of dustup caused by some selfish genesgenes that propagate themselves even if it hurts the host. As the monk Gregor Mendel laid out in his study of pea plants, a particular version of a gene typically has a 50-50 chance of being passed on to the next generation. But the selfish genes Zanders was studying, a type called meiotic drivers because they propel themselves during meiosis, manage to get themselves inherited far more often. These kinds of systems do a complete end run around Mendels laws, says Daniel Barbash, an evolutionary geneticist at Cornell University.

In Schizosaccharomyces pombe, also called fission yeast, Zanders discovered, a family of selfish genes makes moves that would be right at home in a Game of Thrones story line. Zanders and colleagues were the first to work out the molecular tricks that thesegenes use to skirt Mendels laws, reporting the findings in eLife in 2017. The genes, known as wtf genes, produce both a poison and an antidote. All of the spores the yeasts gametes get the poison, but only those that inherit certain gene versions also get an antidote. Spores that dont get the antidote die, ensuring that only offspring with specific wtf gene versions survive to pass their genes on to the next generation. For the fission yeast, such predatory tactics can have big consequences, even driving two nearly identical strains toward becoming different species. Some selfish genes have made themselves essential for proper development (SN: 7/3/18). In humans and other animals, genetic conflicts may lead to infertility.

For the fission yeast, such predatory tactics can have big consequences, even driving two nearly identical strains toward becoming different species.

This extremely important family of meiotic cheaters has been just sitting in plain sight waiting for somebody who had the right kind of lens and the care to discover them, says Harmit Malik, an evolutionary geneticist at the Fred Hutchinson Cancer Research Center in Seattle and Zanders postdoctoral mentor. Zanders helped build a case that the skewed inheritance in these yeast was a real effect, not just fluctuations in the data. Before she began her work, virtually nothing was known about meiotic drivers in yeast. Now the wtf genes are among the best known meiotic drivers studied in any lab organism. Some selfish genes in worms also use the poison-antidote trick to beat the competition (SN: 5/11/17). Meiotic drivers in fruit flies, mice and maybe humans win genetic conflicts by other means (SN: 10/31/17; SN: 2/24/16).

Zanders is now on the lookout for other genetic fights in yeast. Understanding such conflicts more generally may help answer big questions in evolution, as well as shedding light on human infertility. As for what motivates her, Its just I like to solve puzzles, Zanders laughs. I wish it was a deep desire to help people, but its definitely not that. TinaHesmanSaey

Affiliation: CaltechHometown: Jinzhai County, ChinaFavorite hobby: Carpentry

As the Rose Parade wound through Pasadena, Calif., on January 1, 2020, Zhongwen Zhan listened to the underground echoes of the marching bands and dancers. With a sensitive technology known as distributed acoustic sensing, or DAS, Zhan tracked the parades progress. He even identified the most ground-shaking band. (It was the Southern University and A&M Colleges Human Jukebox.)

The study was a small but elegant proof of concept, revealing how DAS is capable of mapping out and distinguishing among small seismic sources that span just a few meters: zigzagging motorcycles, the heavy press of floats on the road, the steady pace of a marching band. But Zhan seeks to use the technology for bigger-picture scientific questions, including developing early warning systems for earthquakes, studying the forces that control the slow slide of glaciers and exploring seismic signals on other worlds.

Zhan has a crystal-clear vision of DAS scientific possibilities, says Nate Lindsey, a geophysicist at Stanford University who is also part of the small community of researchers exploring the uses of DAS. When you get such a cool new tool, you like to just apply it to everything, he adds. But Zhans expertise is very deep, and it goes into many different areas. He knows whats important.

So far, Zhan and other researchers have used the technology to study aftershocks following the 2019 Ridgecrest earthquakes in Southern California (SN: 7/12/19), to demonstrate that interactions between ocean waves produce tiny quakes beneath the North Sea, and to examine the structure of glaciers.

DAS piggybacks off the millions of fiber-optic cables that run beneath the ground, ferrying data for internet service, phones and televisions (SN: 6/14/18). Not all of the glass cables are in use all of the time, and these strands of dark fiber can be temporarily repurposed as seismic sensors. When pulses of light are fired into the fibers ends, defects in the glass reflect the light back to its source. As vibrations within the Earth shift and stretch the fibers, a pulses travel time also shifts.

Whole networks of seismic sensors could be deployed in places currently difficult or impossible to monitorat the ocean bottom, atop Antarctic glaciers, on other planets.

Over the last few years, scientists have begun testing the effectiveness of these dark fibers as inexpensive, dense seismic arrays which researchers call DAS to help monitor earthquakes and create fine-scale images of the subsurface. In these settings, Zhan notes, DAS is proving to be a very useful supplement to existing seismograph networks. But the potential is far greater. Whole networks of sensors could be deployed in places currently difficult or impossible to monitor at the bottom of the ocean, atop Antarctic glaciers, on other planets. Seismology is a very observation-based field, so a seismic network is a fundamental tool, he says.

Ive been interested in science since I was young, but wasnt sure what kind of science I wanted to do, Zhan says. In China, students usually have to decide on a field before they go to college, he adds, but I was fortunate. At age 15, Zhan was admitted to a special class for younger kids within the University of Science and Technology of China in Hefei. The program allowed him to try out different research fields. A nature lover, Zhan gravitated toward the earth sciences. Environmental science, chemistry, atmospheric science I tried all of them.

Then, in late 2004, a magnitude 9.1 earthquake ruptured the seafloor under the Indian Ocean, spawning deadly tsunamis (SN: 1/5/05). After hearing from a researcher studying the quake, Zhan knew he wanted to study seismology. I was amazed by how seismologists can study very remote things by monitoring vibrations in the Earth, Zhan says. The data are just wiggles, complicated wiggles, but so much info can be extracted. And when we do it fast, it can provide a lot of benefit to society. CarolynGramling

Scientists and journalists share a core belief in questioning, observing and verifying to reach the truth. Science News reports on crucial research and discovery across science disciplines. We need your financial support to make it happen every contribution makes a difference.

Go here to read the rest:
This years SN 10 scientists aim to solve some of sciences biggest challenges - Science News

Posted in Rhode Island Stem Cells | Comments Off on This years SN 10 scientists aim to solve some of sciences biggest challenges – Science News

Mesoblast Receives Complete Response Letter From the FDA for Biologics License Application for Steroid-Refractory Acute Graft Versus Host Disease in…

Posted: October 2, 2020 at 12:58 am

NEW YORK, Oct. 01, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (NASDAQ:MESO, ASX:MSB)), global leader in allogeneic cellular medicines for inflammatory diseases, announced today that the US Food and Drug Administration (FDA) has issued a Complete Response Letter to its Biologics License Application (BLA) for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). While the Oncologic Drugs Advisory Committee (ODAC)1 of the FDA voted 9:1 that the available data support the efficacy of remestemcel-L in pediatric patients with SR-aGVHD, the FDA recommended that Mesoblast conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD. As there are currently no approved treatments for this life-threatening condition in children under 12, Mesoblast will urgently request a Type A meeting with the FDA, expected within 30 days, to discuss a potential accelerated approval with a post-approval condition for an additional study.

Joanne Kurtzberg, MD, Jerome Harris Distinguished Professor of Pediatrics, Director, Pediatric Blood and Marrow Transplant Program, and Co-Director, Stem Cell Transplant Laboratory Duke University Medical Center, said: "The Phase 3 trial results showed that remestemcel-L provides a meaningful treatment for children with SR-aGVHD who have a very dismal prognosis. I look forward to having this much-needed therapy available to our patients."

Mesoblast is currently conducting a randomized, controlled Phase 3 trial evaluating remestemcel-L in up to 300 ventilator-dependent adults with moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19. A second interim analysis by the trial's independent Data Safety Monitoring Board is expected in early November, with completion of patient enrollment expected in December. COVID-19 ARDS is an inflammatory disease with a similar profile of damaging inflammatory cytokines as is seen in children with SR-aGVHD, and is the primary cause of death in COVID-19 infection. The trial's primary endpoint is reduction of all-cause mortality within 30 days of randomization.

The FDA also identified a need for further scientific rationale to demonstrate the relationship of potency measurements to the product's biologic activity. Assays measuring the potency of remestemcel-L will continue to be refined to provide further scientific rationale for its use in severe inflammatory diseases with high mortality risk, such as SR-aGVHD and COVID-19 ARDS.

Mesoblast Chief Executive Dr Silviu Itescu stated: "We are working tirelessly to bring remestemcel-L to patients with life threatening inflammatory conditions, including SR-aGVHD and COVID-19 ARDS."

About Acute Graft Versus Host DiseaseAcute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.2In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.3,4There are currently no FDA-approved treatments in the United States for children under 12 with SR-aGVHD, a potentially life-threatening complication of an allogeneic bone marrow transplant for blood cancer.

About Remestemcel-LMesoblast's lead allogeneic cell therapy product candidate, remestemcel-L, is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. Remestemcel-L is thought to have immunomodulatory properties to counteract the cytokine storms that are implicated in various inflammatory conditions by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. This vote includes a change to the original vote by one of the ODAC panel members after electronic voting closed.2. Niederwieser D, Baldomero H, Szer J. Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. Bone Marrow Transplant 2016; 51(6):778-85.3. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology 2011;2011:601953.4. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation 2019;54(11):1805-1814

Conference CallAn audio webcast will begin at 9.15am Friday, October 2 AEST / 7.15pm Thursday, October 1, 2020 EDT. The audio webcast can be accessed viahttps://webcast.boardroom.media/mesoblast-limited/20200930/NaN5f7147e5581a8100190f7687orWebcast link

The archived webcast will be available on the Investor page of the Company's website http://www.mesoblast.com

About MesoblastMesoblast Limited (NASDAQ:MESO, ASX:MSB)) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblast has a strong and extensive global intellectual property (IP) portfolio with protection extending through to at least 2040 in all major markets. The Company's proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid-refractory acute graft versus host disease and moderate to severe acute respiratory distress syndrome. Mesoblast is completing Phase 3 trials for its product candidates for advanced heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblast's licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "likely," "look forward to," "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions and variations thereof. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. The risks, uncertainties and other factors that may impact our forward-looking statements include, but are not limited to: the timing, progress and results of Mesoblast's preclinical and clinical studies; Mesoblast's ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals (including our request to have a Type A meeting with the FDA, the outcome of such a meeting, and any future decision that the FDA may make on the BLA for remestemcel-L for pediatric patients with SR-aGVHD); and the pricing and reimbursement of Mesoblast's product candidates, if approved; Mesoblast's ability to establish and maintain intellectual property on its product candidates and Mesoblast's ability to successfully defend these in cases of alleged infringement. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast's actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. Unless required by law, we do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorizedby the Board.

For further information, please contact:

Read more:
Mesoblast Receives Complete Response Letter From the FDA for Biologics License Application for Steroid-Refractory Acute Graft Versus Host Disease in...

Posted in New York Stem Cells | Comments Off on Mesoblast Receives Complete Response Letter From the FDA for Biologics License Application for Steroid-Refractory Acute Graft Versus Host Disease in…

Page 877«..1020..876877878879..890900..»