Syngenta Crop Protection and Insilico Medicine to Harness Artificial Intelligence to Transform Sustainable Product Innovation – BioSpace

Posted on February 4, 2021 by Dook Domini

Feb. 3, 2021 07:00 UTC

BASEL, Switzerland--(BUSINESS WIRE)-- Syngenta Crop Protection is collaborating with artificial intelligence (AI) and deep learning company Insilico Medicine to accelerate the invention and development of new, more effective crop protection solutions that protect crops from diseases, weeds and pests, while also protecting ecosystems. By bringing new solutions to farmers faster and more efficiently through innovation, Syngenta will help them meet the ongoing challenges they face, in order to enhance productivity and meet global demand for affordable, quality food.

This collaboration with Insilico Medicine means that Syngenta can harness the immense potential and scope of AI to develop the next generation of sustainable crop protection solutions as part of Syngentas $2bn commitment to innovation and sustainability, said Camilla Corsi, Head Crop Protection Research at Syngenta. This will further transform agriculture by providing farmers around the world with the tools they need to produce healthy, nutritious, affordable and sustainably grown food in the most efficient way, while also minimizing the environmental impact.

Insilico Medicine has a proven track record and has delivered significant advances in pharmaceutical research, using AI and deep learning to design, synthesize and validate new ingredients. The same approach also has the potential to transform the development of new crop protection solutions that help keep plants safe, from planting to harvesting. Working closely with Syngenta, Insilico Medicine will use their AI-powered small molecule generative chemistry technology not only to invent molecules for active ingredients faster, but also actively design molecules that are more sustainable and environmentally friendly.

We are very happy to collaborate with a company that is dedicated to developing safe and sustainable solutions for growers, said Alex Zhavoronkov, PhD, founder, and CEO, Insilico Medicine. Our artificial intelligence is designed from the ground up to produce very precise chemistry to protect human health, while ensuring short-term and long-term safety. This expertise is extremely valuable for crop sciences, and especially so for businesses whose top priority is the safety of their products. Syngenta is a progressive company with many brilliant scientists, and we will be working together to use artificial intelligence for the benefit of agriculture.

Our reputation as a global leader in innovation is built on a foundation of collaboration and our understanding of the challenges faced by growers, Camilla Corsi also noted. Working together with Insilico Medicine, combining our skills, knowledge and technologies, will help ensure that new and more effective crop protection solutions will be in the hands of farmers sooner.

About Syngenta

Syngenta is one of the worlds leading agriculture companies, comprising of Syngenta Crop Protection and Syngenta Seeds. Our ambition is to help safely feed the world while taking care of the planet. We aim to improve the sustainability, quality and safety of agriculture with world class science and innovative crop solutions. Our technologies enable millions of farmers around the world to make better use of limited agricultural resources. Syngenta Crop Protection and Syngenta Seeds are part of Syngenta Group with 49,000 people in more than 100 countries and is working to transform how crops are grown. Through partnerships, collaboration and The Good Growth Plan we are committed to accelerating innovation for farmers and nature, striving for carbon neutral agriculture, helping people stay safe and healthy and partnering for impact.

To learn more visit http://www.syngenta.com and http://www.goodgrowthplan.com

About Insilico Medicine

Insilico Medicine develops software that leverages generative models, reinforcement learning (RL), and other modern machine learning techniques for the generation of new molecular structures with specific properties. Insilico Medicine also develops software for the generation of synthetic biological data, target identification, and the prediction of clinical trials outcomes. The company integrates two business models; providing AI-powered drug discovery services and software through its Pharma.AI platform (www.insilico.com/platform/) and developing its own pipeline of preclinical programs. The preclinical program is the result of pursuing novel drug targets and novel molecules discovered through its platforms. Since its inception in 2014, Insilico Medicine has raised over $52 million and received multiple industry awards. Insilico Medicine has also published over 100 peer-reviewed papers and has applied for over 25 patents. Website http://insilico.com/

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Cautionary Statement Regarding Forward-Looking Statements

This document may contain forward-looking statements, which can be identified by terminology such as expect, would, will, potential, plans, prospects, estimated, aiming, on track and similar expressions. Such statements may be subject to risks and uncertainties that could cause the actual results to differ materially from these statements. For Syngenta, such risks and uncertainties include risks relating to legal proceedings, regulatory approvals, new product development, increasing competition, customer credit risk, general economic and market conditions, compliance and remediation, intellectual property rights, implementation of organizational changes, impairment of intangible assets, consumer perceptions of genetically modified crops and organisms or crop protection chemicals, climatic variations, fluctuations in exchange rates and/or commodity prices, single source supply arrangements, political uncertainty, natural disasters, and breaches of data security or other disruptions of information technology. Syngenta assumes no obligation to update forward-looking statements to reflect actual results, changed assumptions or other factors.

2021 Syngenta. Rosentalstrasse 67, 4002 Basel, Switzerland. The Syngenta logo are trademarks of a Syngenta Group Company. All other trademarks are the property of their respective owners.

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Exploring the Importance of Finding a Molecular Target in Lung Cancer – Targeted Oncology

Posted on February 4, 2021 by Dook Domini

Brendon Stiles, MD, discusses the current landscape for treating patients with metastatic lung cancer.

Brendon Stiles, MD, a thoracic surgeon at NewYork-Presbyterian Hospital and an associate professor of cardiothoracic surgery at Weill Cornell Medicine, discusses the current landscape for treating patients with metastatic lung cancer.

Stiles states that there is an amazing number treatment options available in the metastatic setting. Understanding the molecular subtype of cancer in patients with metastatic disease is important for their treatment. There are targets such as EGFR,ALK,ROS,BRAF,NTRK,MET, andRET.There are multiple options when targeting rare alterations in this population. He says it gets to that principle of having to look for mutations to know if they are there.

Aside from targeted therapy, some patients are eligible for immunotherapy, which is a completely different paradigm based on PD-L1 expression and combination therapy, according to Stiles.

Stiles sees many patients in the late stages of their disease as part of the initial diagnosis phase or sometimes to perform a diagnostic procedure to uncover metastatic disease. There was a time when there wasnt good prognosis for patients with lung cancer. Now that oncologists have figured out the appropriate therapy for each type of patient, Stiles explains that he would like to see these treatments move into the early-stage setting. This is slowly starting to happen, he says, but its critical to know the histologic and molecular subtype of a patient with stage IV disease.

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Exploring the Importance of Finding a Molecular Target in Lung Cancer - Targeted Oncology

Bioinformatics Services Market | Know the Latest Innovations and Future Market Scope – BioSpace

Posted on February 4, 2021 by Dook Domini

Bioinformatics is the field mainly involving molecular biology, genetics, mathematics, statistics, and computer science. The bioinformatics services include analysis of the data that can range from processing sequencing reads from instrument to data aggregation and mining data samples. Bioinformatics services can help biologists to understand the biological process with a computational intensive technique for machine learning algorithms, pattern recognition, data mining and visualization.

Bioinformatics tools can help to compare genomic and genetic data and understand evolutionary aspects of molecular biology. Bioinformatics services are finding wide application in chemoinformatics, genomics, metabolomics, RNA-seq analysis, and drug design. The database is an important part for bioinformatics research and application to cover various information types including molecular structure, protein and DNA sequences, and phenotypes in bioinformatics services.

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Bioinformatics Services Market: Notable Highlights

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Some of the most prominent competitors operating in the competitive landscape of global bioinformatics services market include

Bioinformatics Services Market Dynamics

Bioinformatics Services Finding Wide Application in Personalized Medicine Discovery

With the increasing prevalence of various diseases, new treatments and drugs are being discovered and developed. Extensive molecular biological data on patients is being included on a large scale in diagnosis and treatment. Bioinformatics services is fundamental to precision medicine as developing personalized medicine depends on accessing genetic and molecular data. In recent years, the majority of the molecularly targeted drugs have been developed based on the detected gene mutation.

Next-gen sequencing in bioinformatics services is emerging as an important tool in genomic analysis and developing personalized medicine. Next-gen sequencing along with microarrays in bioinformatics services have also paved the way for precision medicine in oncology. Meanwhile, increasing availability and decreasing the cost of next-gen sequencing is allowing worldwide cancer centers to offer next-gen sequencing based personalized oncology for clinical practice while suggesting specific medicine and treatment.

Increasing Initiatives by Governments and Private Organizations in Bioinformatics Services

With increasing application of new technologies in life science, governments and organizations across various countries are investing in the new technologies and in research and development activities in bioinformatics services. According to the Global Alliance for Genomics and Health (GA4GH), around 60 million genomes are likely to be sequenced by 2025. Moreover, with the presence of national clinical genomic initiatives worldwide, the generation of genomic data in healthcare is expected to outpace that in research in the coming years. Governments across countries are increasingly investing in the biotechnology and bioinformatics services to effectively implement new technologies and support genomic and epidemiological research.

Countries such as the US, UK, Australia, France, Japan, Saudi Arabia, Qatar, Denmark are developing new strategies for projects focusing on cancer and rare diseases, along with the use of sequencing services and genomic data. New research activities are also being conducted for application of bioinformatics services in biodefense. The Mid-Atlantic Microbiome Meet-up (M3) is focusing on the use of next-generation sequencing technologies and recent advances in biodefense, especially related to infectious diseases, and also using metagenomic methods for detection.

Shortage of Skilled Workforce and High Cost Hampering the Bioinformatics Services Market Growth

Although bioinformatics services is emerging as an important part of research in life science, lack of skills and knowledge in bioinformatics is hindering its growth. With the technological and process advancements in biotechnology, it has become imperative that bioinformatics techniques are performed by skilled personnel. However, the need for heavy investment in tool upgradation and installation training is impeding the growth of bioinformatics services. Owing to this there is a lack of skilled manpower in bioinformatics services who can adapt to the high-end bioinformatics techniques and processes.

Moreover, the lack of skilled professionals in bioinformatics services is also hampering the growth of clinical laboratories as they are focusing to automate processes. However, in recent years, governments along with healthcare institutions are focusing on strategies to provide new courses in bioinformatics as it holds a big promise in solving many health related and environmental issues.

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Bioinformatics Services Market Segmentation

Based on the type, the bioinformatics services market is segmented into

On the basis of application, the bioinformatics services market segment includes

Based on the specialty, the bioinformatics services market is segmented into

Based on the end-user, the bioinformatics services market segment includes

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Adjuvant platinum-based chemotherapy in non-small cell lung cancer: The role of relative dose-intensity and treatment delay. – Physician’s Weekly

Posted on February 4, 2021 by Dook Domini

The study investigated the association of the relative dose-intensity (RDI) of cisplatin and timing of adjuvant platinum-based chemotherapy (APC) with survival for stage I-III non-small cell lung cancer (NSCLC) patients.Real-life data of patients treated with APC (four cycles of cisplatin and vinorelbine) between 2007 and 2014 was included to analyse the association between disease-free survival (DFS) and overall survival (OS) with RDI (ratio of received to planned dose-intensity). High RDI was defined as cisplatin RDI of > 75% and low RDI 75%.Out of 198 patients, 166 were eligible. Low RDI was administered to 72 (43%) patients. In multivariate analysis, those patients had a significantly higher risk of recurrence (HR: 1.87, 95%CI 1.13-3.09, p=0.01) and death (HR: 1.91, 95%CI 1.32-3.23, p=0.01) versus patients in the high RDI group. The risk of death was significantly higher in patients with PS 1 treated with low versus high RDI (HR: 2.72, 95%CI: 1.22-6.09, p=0.014). The risk of recurrence was higher for patients with squamous cell carcinoma of low versus high RDI (HR: 3.82, 95%CI: 1.01-14.4, p=0.048). No impact of delayed APC beyond six weeks from surgery on neither DFS (HR: 0.78, 95%CI: 0.46-1.33, p=0.36) nor OS (HR 0.67, 95%CI: 0.40-1.15, p=0.15) was observed.Low cisplatin RDI 75% of APC, but not extended time from surgery to APC onset > six weeks, was associated with significantly shorter survival in NSCLC patients.

PubMed

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Adjuvant platinum-based chemotherapy in non-small cell lung cancer: The role of relative dose-intensity and treatment delay. - Physician's Weekly

Syngenta Crop Protection and Insilico Medicine to Harness Artificial Intelligence to Transform Sustainable Product Innovation – Business Wire

Posted on February 4, 2021 by Dook Domini

BASEL, Switzerland--(BUSINESS WIRE)--Syngenta Crop Protection is collaborating with artificial intelligence (AI) and deep learning company Insilico Medicine to accelerate the invention and development of new, more effective crop protection solutions that protect crops from diseases, weeds and pests, while also protecting ecosystems. By bringing new solutions to farmers faster and more efficiently through innovation, Syngenta will help them meet the ongoing challenges they face, in order to enhance productivity and meet global demand for affordable, quality food.

About Syngenta

To learn more visit http://www.syngenta.com and http://www.goodgrowthplan.com

About Insilico Medicine

Cautionary Statement Regarding Forward-Looking Statements

2021 Syngenta. Rosentalstrasse 67, 4002 Basel, Switzerland. The Syngenta logo are trademarks of a Syngenta Group Company. All other trademarks are the property of their respective owners.

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Syngenta Crop Protection and Insilico Medicine to Harness Artificial Intelligence to Transform Sustainable Product Innovation - Business Wire

M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board – BioSpace

Posted on February 4, 2021 by Dook Domini

Feb. 3, 2021 12:30 UTC

ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced its scientific advisory board (SAB) that will support the Companys mission of translating its innovative bicistronic-S1S3 technology platform into best-in-class therapies that address unmet needs within the LSD community. The Companys platform enables improved biodistribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.

As we work to advance our robust pipeline, we seek the input and support of a world-class team of scientific advisors with deep expertise in genetics, rare diseases, and lysosomal storage and metabolic disorders in particular, said Pawel Krysiak, president and chief executive officer of M6P Therapeutics. The collective insights, knowledge, commitment, and expertise of our scientific advisory board will help us translate this high science into potential medical benefit for the individuals affected by these serious conditions.

By combining the substantial expertise of the SAB with the expertise of the Companys internal R&D team in recombinant enzyme and gene therapies, M6P Therapeutics is well positioned to rapidly advance its deep pipeline of LSD programs. The members of the SAB are:

M6P Therapeutics bicistronic-S1S3 technology platform enhances mannose 6-phosphate content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, said Stuart Kornfeld, MD, M6P Therapeutics co-founder and chairman of its SAB. With promising pre-clinical data across numerous LSD programs, this innovation can potentially translate into new and more efficacious treatments, reduced immunogenicity, and more efficient dosing regimens.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board - BioSpace

Experts speak the plain truth about COVID-19 vaccine – Wellington Advertiser

Posted on February 4, 2021 by Dook Domini

WELLINGTON COUNTY The information about COVID-19 and now the vaccines to fight it has been coming fast and furious over the past year. Its no wonder people are overwhelmed with information and confused about who to trust.

Thats why the Canada Institute of Health Research (CIHR) launched a media blitz in January: to address misinformation and dispel myths head-on.

Dr. Charu Kaushic is the scientific director of the Canadian Institute of Infections and Immunity for the CIHR and a professor of molecular medicine at McMaster University.

In a broad-ranging interview with the Advertiser on Jan. 28, Kaushic answered the most-asked questions and countered some oft-held myths surrounding the vaccines.

Kaushic explained that the COVID-19 vaccines work differently than the vaccines people are accustomed to.

Traditional vaccines take a weakened or inactivated form of a virus and inject it into the body to trigger an immune response.

The new vaccines, called mRNA vaccines, take genetic information from the virus the spike protein and that is injected into the body.

The cells make the protein, they are seen as foreign, and that triggers the immune response, Kaushic said.

Its the immune response that produces antibodies, and its antibodies that protect recipients from getting infected when the real virus comes along. And since only part of the protein is made, it does not do any harm to the person vaccinated.

Once youve had two doses of the Pfizer or Moderna vaccines, you are 95% protected, Kaushic said.

Ninety-five per cent is not 100% but thats the protection. Between the first and second dose, you are not 95% protected. Thats why we still need to exercise precautions, she said.

The mRNA vaccines may be new to laypeople, but researchers have been working on the technology for at least a decade, Kaushic said.

For a long time (researchers) have anticipated there would be a pandemic and they have been preparing since 2017 for making a pandemic vaccine, she said.

When the pandemic started, researchers took the virus, used the technology, and started trials, and the first trials generated good results.

Kaushic said initial trials included 30,000 people aged 18 to 65 as well as those over 80.

Trials are ongoing now for younger age groups, pregnant women and people with underlying health conditions.

We expect to know more (about these groups) in three to four months, she said.

Kaushic said because the vaccine is a world-wide priority and governments have a lot of money on the table to have the vaccine manufactured and distributed, normal timelines have been compressed.

Personnel have been redeployed to vaccine production and its all hands on deck.

The work has been compressed at a cost to governments, but theres no compromise on safety, she said.

Kaushic said she hasnt heard of any deaths as a result of the vaccine and allergic reactions are rare.

In terms of side effects, some people feel nauseated and feverish after receiving the vaccine but that only lasts a day or two.

Kaushic said there is a host of good information out there about the virus, the vaccines, and health protection measures, and listed the World Health Organization, Health Canada and the Centres for Disease Control and Prevention as appropriate places to start.

People should not be getting their news from Facebook, she said.

Get information from people who know what they are talking about. So ask your doctor or health specialist. Check authoritative articles.

Its important to get the right information and its incumbent on people to make sure they check their sources.

Dr. Julie Ray, a family physician at the Upper Grand Family Health Team, in Elora, said her patients are anxious about the vaccine too, although most are eager to get it.

The biggest question is when do I get it and where do I get it? she said. And my biggest answer is I dont know yet.

The second question: is it safe?

Its not so much misinformation, but reservations about how quickly things have progressed, Ray said.

I personally havent had that many conversations that go that way. Most are saying Sign me up, or put me on a list.

Ray said scientists believe there will be herd immunity when approximately 70 per cent of the population is vaccinated.

The idea is if I get sick with COVID, there needs to be enough people immune to it so I cant get them sick and spread it, she said.

Right now, the vaccine is not approved by Health Canada for children, pregnant women or people who with certain underlying health conditions, but trials are ongoing, and that information will change when results are known.

Ray said people who fall in that category should talk to their healthcare provider.

You can get the vaccine if its deemed appropriate. Doctors will discuss it on a case-by-case basis, assessing the riskbenefit in each case, she said.

Ray added family doctors will be part of the broad plan to administer the vaccine, but long-term care residents and front-line healthcare workers are the priority right now.

With news of supply-chain issues for distribution of the vaccine, previous timelines are now out the window.

But that doesnt mean you shouldnt be prepared for it.

If you are in a special population, dont leave it until public health calls with an appointment. If youre vaccine-hesitant, talk to your doctor. Ask questions. Express your concerns, Ray said

Understanding is knowledge and knowledge is power.

Wellington-Dufferin-Guelph Public Health also has vaccine information on its website.

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Experts speak the plain truth about COVID-19 vaccine - Wellington Advertiser

Ross Prize Ceremony and Webinar on the Genetics of Neurological Disorders – The New York Academy of Sciences

Posted on October 30, 2020 by Dook Domini

New York, October 26, 2020 The Ross Prizein Molecular Medicine will be awarded to Adrian R. Krainer, PhD, St. Giles Foundation Professor at Cold Spring Harbor Laboratory, in a virtual ceremony and webinar hosted by the New York Academy of Sciences, Feinstein Institutes for Medical Research, and the journal Molecular Medicine on October 30. The webinar will be held 1 PM to 4:50 PM EDT.

The Feinstein Institutes for Medical Research has selected Dr. Krainer as the eighth recipient of the Ross Prize, which is awarded annually through the Feinstein Institutes peer-reviewed, open-access journal, Molecular Medicine.

The Ross Prize recognizes Dr. Krainer for his pioneering work in introducing anti-sense therapy in clinical use, and for its successful application to spinal muscular atrophy (SMA). The Ross Prize includes a $50,000 award.

After a brief award presentation at the start of the October 30 webinar, Dr. Krainer will discuss his work. This will be followed by a session on topics in the genetics of neurological disorders. Speakers for this session will include: Edward M. Kaye, MD, Stoke Therapeutics; Michelle L. Hastings, PhD, Rosalind Franklin University of Medicine and Science; and Timothy Yu, MD, PhD, Boston Children's Hospital, Harvard Medical School. The webinar will be held 1 PM 4:50 PM EDT.

The Ross Prize is made possible by the generosity of Feinstein Institutes board members Robin and Jack Ross. The Ross Prize recognizes biomedical scientists whose discoveries have transformed the way medicine is practiced. The awardees are midcareer researchers who have made a significant impact in the understanding of human disease pathogenesis and/or treatment. Moreover, it is anticipated that they will continue to make profound advances in the general field of molecular medicine.

The Ross Prize is a worthy tribute to the significance and impact of the fundamental and applied research conducted by my lab and our collaborators, which culminated in a disease-modifying therapy for spinal muscular atrophy, Dr. Krainer said. I greatly admire the seven previous Prize recipients, so I am humbled to join this distinguished group of scientists and clinicians.

Dr Krainer is the eighthrecipient of the Ross Prizein Molecular Medicinebecause his discoveries are revolutionizing treatment of a devastating, crippling pediatric illness, saidKevin J. Tracey, MD, president and CEO of the Feinstein Institutes and editor emeritus ofMolecular Medicine.His work enables children with spinal muscular atrophy to crawl, walk, and live a full life.

Dr. Krainer explained his work in more detail:

"My labs research has a long-standing focus on understanding RNA splicing, a fundamental cellular process. In addition, we are interested in how alterations in this key step in gene expression cause or contribute to disease. This basic research eventually led us to the development of mechanism-based therapies. Our main goals are to continue gaining novel insights into RNA-splicing mechanisms and regulation, and to translate these findings into new drugs or clinically useful methods. These are important goals, because they differ from the traditional path for drug development, and so they have the potential to yield effective solutions to intractable medical problems."

In addition to studying the mechanisms of RNA splicing, Dr. Krainer uses multidisciplinary approaches to examine the ways in which they go awry in disease, and the means by which faulty splicing can be corrected. He co-developed the first FDA-approved therapy for the genetic disorder SMA an illness that has been the leading genetic cause of infant death based on the biological process of RNA splicing. This life-saving drug is also the first approved splicing-corrective therapy.

To learn more about the Ross Prize celebration and symposium, and to register for the event, please visit http://www.nyas.org/RossPrize2020.

Past recipients of the Ross Prize are: Daniel Kastner, MD, PhD, scientific director of the National Institutes of Healths (NIH) National Human Genome Research Institute (NHGRI); Huda Y. Zoghbi, MD, professor, Departments of Pediatrics, Molecular and Human Genetics, Neurology and Neuroscience at Baylor College of Medicine; Jeffrey V.Ravetch, MD, PhD,the Theresa and Eugene M. Lang Professor and head of the Leonard Wagner Laboratory of Molecular Genetics and Immunology at The Rockefeller University; Charles N.Serhan, PhD, DSc, director of the Center for Experimental Therapeutics and Reperfusion Injury at Brigham and Womens Hospital, the SimonGelmanProfessor ofAnaesthesiaat Harvard Medical School and professor at Harvard School of Dental Medicine; Lewis C.Cantley, PhD, the Meyer Director of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medical College andNew York-Presbyterian Hospital; John J. OShea, MD, scientific director at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); and Dan R. Littman, MD, PhD, the Helen L. and Martin S. Kimmel Professor of Molecular Immunology in theSkirballInstitute ofBiomolecularMedicine at New York University School of Medicine.

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Ross Prize Ceremony and Webinar on the Genetics of Neurological Disorders - The New York Academy of Sciences

Should the FDA Have Approved Remdesivir to Treat COVID-19 Patients? – Healthline

Posted on October 30, 2020 by Dook Domini

Last week, the Food and Drug Administration (FDA) approved the antiviral remdesivir as a treatment for COVID-19 in adults and children 12 years and older, paving the way for wider use of the drug.

Dr. Lanny Hsieh, clinical professor of infectious diseases at UCI Health, said the FDAs move is very exciting.

Putting together all of the scientific evidence we have on remdesivir to date, it remains the standard of care for hospitalized patients with COVID-19, she said. Ultimately, it is our patients who would benefit from this FDA approval.

However, research so far on remdesivir is mixed, and the drug is far from a cure for COVID-19.

In May, the FDA issued an emergency use authorization (EUA) for remdesivir, marketed in the United States under the brand name Veklury. This allowed the drug to be used to treat those with severe COVID-19.

The agency broadened the EUA in August to allow for its use on all hospitalized patients with COVID-19, regardless of how severe their illness.

President Trump took remdesivir along with several other treatments when he was hospitalized for COVID-19 in early October.

The FDA based its decision on three randomized controlled trials.

One study of 1,062 participants with mild, moderate, or severe COVID-19 was published earlier in October in the New England Journal of Medicine.

Results from this trial show that remdesivir reduced the length of hospital stay by about 5 days from 15 down to 10.

Patients taking remdesivir also had a lower chance of dying after 28 days 11.4 percent compared with 15.2 percent in patients receiving an inactive placebo.

This [study], along with other trials reviewed by the FDA, has led to remdesivirs approval, Hsieh said, who is the principal investigator on the remdesivir clinical trial at UCI Medical Center.

The two other trials reviewed by the FDA had similar results. One of these also showed that a 5-day course of remdesivir worked just as well as taking the drug for 10 days.

However, preliminary results from the World Health Organization (WHO) Solidarity trial of more than 11,000 participants found that remdesivir had little effect on how long they stayed in the hospital and no effect on death.

This study was published as a preprint on medRxiv and hasnt yet been peer-reviewed, so the results should be viewed with some caution. The WHO plans to publish it in the New England Journal of Medicine.

Given the results of the WHO study, Dr. Eric Topol, a professor of molecular medicine at the Scripps Research Translational Institute, questioned whether the FDA should have granted remdesivir a full approval.

How can Remdesivir get a full [FDA] approval when there are such mixed data? Not supportive of this decision at all, he wrote on Twitter. Does it work early? Does it work late? Does it work anytime? So much unresolved.

However, Hsieh said the WHOs study had several limitations, including not comparing remdesivirs effects to a placebo, and looking at several potential treatments in the same study.

Although interesting, Solidaritys findings do not take away from the results of [the NEJM trial], she said, which is a study that is conducted with the most scientific rigor to date.

Without a COVID-19 vaccine approved in the United States, doctors are anxious for an effective treatment for COVID-19. Remdesivirs approval finally gives them something to work with.

Given the limited arsenal of effective or even marginally effective treatments for COVID-19, and the fact that we dont have a fully curative therapy or a vaccine, it is good to have more options, said Dr. Matthew G. Heinz, a hospital physician and internist in Tucson, Arizona.

But he said remdesivir is still difficult to get in some parts of the country, especially in rural areas.

And its expensive. A 5-day course of treatment can cost $3,120 for people with private insurance, reports Vox.

Remdesivir is also not without risks. In some people, it can cause elevated liver enzymes, which could be a sign of liver damage. The most common side effect, though, is nausea.

In specific situations for certain patients, I do think [remdesivir] is reasonable to use, Heinz said, because it can inhibit viral replication if given at the right time point.

Remdesivir blocks the coronavirus from replicating, so it works best if given early.

Remdesivir is likely going to be more useful for stopping serious progression of the disease, Heinz said. But to give it to someone whos already critical getting intubated or who has already been intubated may not work.

The drug is less effective in later stages of severe COVID-19, when the damage is caused more by an overactive immune response than by the virus itself.

At this point, doctors turn to other treatments that target the immune system. One of these is the corticosteroid dexamethasone, which dampens the immune response and has been shown to reduce deaths from COVID-19.

Although remdesivir isnt completely effective against COVID-19, many doctors on the front line are glad to have it as an option.

Given that its not shown to have significant safety concerns, and at least one good study does show some benefit, it is reasonable to have remdesivir as an available treatment while we wait for better ones, Heinz said.

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Should the FDA Have Approved Remdesivir to Treat COVID-19 Patients? - Healthline

UArizona researchers have breakthrough related to stomach cancer – Eastern Arizona Courier

Posted on October 30, 2020 by Dook Domini

TUCSON A promising new biomarker that appears in patients before stomach cancer develops may help with early detection of the disease and improve patient response to therapy, according to findings in a study led by University of Arizona Health Sciences researchers.

The biomarker can be detected through a simple blood test, saving time and lowering costs. Currently, stomach cancer diagnosis requires endoscopic collection of stomach tissue through a biopsy procedure, and then analysis by pathology.

Published in Gut, the journal of the British Society of Gastroenterology, the study was led by Juanita L. Merchant, MD, PhD, chief of the Division of Gastroenterology and Hepatology at the UArizona College of Medicine Tucson, a cancer biology program researcher at the UArizona Cancer Center and an elected member of the National Academy of Medicine.

See a video of how Drs. Juanita Merchant and Yana Zavros found a new biomarker to help diagnose stomach cancer.

The biomarker, MiR130b, is a microRNA or small non-coding RNA molecule that can play an important role in regulating gene expression, affecting disease development and progression. MiR130b can be produced by a group of immune cells called myeloid-derived suppressor cells (MDSCs), commonly associated with infections caused by Helicobacter pylori (H. pylori), a bacteria associated with ulcers. These particular cell types in the stomach correlate with early, preneoplastic changes (before a tumor develops) that can lead to gastric cancer long after an H. pylori infection has passed.

The study included collaboration with Yana Zavros, PhD, associate head for research in the College of Medicine Tucsons Department of Cellular and Molecular Medicine and the Cancer Centers shared resource director for Tissue Acquisition Cellular and Molecular Analysis.

Even though you get can get rid of the bacteria, oftentimes the infection itself already has initiated a cascade of events that inevitably may lead to cancer, Dr. Zavros said. That is why early detection is so important.

A Blood Test Instead of a Procedure

The study arose out of basic science mouse models that simulated changes in the stomach similar to that caused by H. pylori. This led the researchers to identify MiR130b in the mouse models, and they also detected the same microRNA in the plasma of human patients that either had precancerous changes or those that already had progressed to cancer.

This was a retrospective study, said Dr. Merchant, who is a member of the universitys BIO5 Institute. It is very exciting because now we can begin looking at this biomarker more prospectively in different patient populations.

Although less common in the United States, the National Cancer Institute reports gastric (stomach) cancer is the third most common cause of cancer-related deaths in the world. The findings, however, could have major implications for Arizonas rural areas and Hispanic and Native American populations, which are at greater risk for developing gastric and other gastrointestinal (GI) cancers, because these diseases often are caused by dietary and environmental factors and may go undetected for long periods.

Dr. Merchants lab has a sub-project in the Cancer Centers U54 grant (Partnership for Native American Cancer Prevention) to study detection of the microRNA described in the Gut paper in members of Native American populations with H. pylori.

This molecular signature (the microRNA MiR130b) that we discovered may help us see if patients have changes in their mucosa (the membrane that lines the stomach) related to having H. pylori, Dr. Merchant said. And a blood sample would be less invasive and then could be a way to make the decision whether we need to bring a patient in for an endoscopy.

Broader Implications for Treatment

Once diagnosed, gastric cancer can be difficult to treat. Immunotherapies with proven effectiveness in treating other types of cancer are not as successful against most GI cancers, including stomach cancer. The researchers believe these new findings in gastric cancer may help to address why other GI cancers also are resistant to therapy.

The underlying mechanism by which a patient may not respond well in gastric cancer may be applicable in other organs as well Dr. Zavros said. The way the cells interact with each other to render that patient resistant to therapy may be quite similar between gastric, pancreatic and colon cancers.

Dr. Merchant added: There may be dual-purposes. We can look at it as a biomarker to help us from a diagnostic perspective, but we also can look at therapies that can be developed based on what this microRNA itself is targeting.

Another project funded by the Cancer Centers Sparking Bench-to-Bedside Team Science Project award is building from results of this study to explore therapies for pancreatic and gastric cancer. The investigators are exploring the tumor microenvironment, in particular the immune cell MDSCs, referred to previously, that appears to dampen the chemotherapeutic response to immunotherapies.

The project relies heavily on Dr. Zavros BioDroid program, which develops miniature organs in the lab with a realistic microanatomy, also known as organoids. These are used in collaboration with the Tissue Acquisition Repository for Gastrointestinal and HEpaTic Systems (TARGHETS), created by Dr. Merchant. TARGHETS is a GI/Hepatology biorepository that collects samples from patients who undergo endoscopy.

Both Drs. Zavros and Merchant are looking to the BioDroid and TARGHETS efforts to reveal additional information that will allow them to develop new approaches to address resistance of gastric cancer to immunotherapies.

We want to find a way to reprogram the cancer cells or the immune cells within that patients tumor environment to make the patient more responsive to the therapy, Dr. Zavros said. A biomarker gives us a place to start.

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UArizona researchers have breakthrough related to stomach cancer - Eastern Arizona Courier

Aviceda Therapeutics Announces Formation of Scientific Advisory Board – BioSpace

Posted on October 30, 2020 by Dook Domini

Oct. 27, 2020 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Aviceda Therapeutics, a late-stage, pre-clinical biotech company focused on developing the next generation of immuno-modulators by harnessing the power of glycobiology to manipulate the innate immune system and chronic, non-resolving inflammation, is announcing the members of its Scientific Advisory Board who will help shape ongoing development efforts.

The Aviceda Scientific Advisory Board includes Pamela Stanley, PhD; Ajit Varki, MD; Christopher Scott, PhD; Geert-Jan Boons, PhD; Salem Chouaib, PhD; and Peng Wu, PhD.

Aviceda has assembled an extraordinary multi-disciplinary team of world-class scientists and renowned researchers to join our efforts in developing the next generation of glyco-immune therapeutics for the treatment of immune-dysfunction conditions, said Mohamed A. Genead, MD, Founder, CEO & President of Aviceda Therapeutics. Each individual offers a fresh perspective and unique strategic acumen that complements and strengthens the insights of our in-house leadership development team.

Prof. Scott, Aviceda Scientific Co-Founder, is Director of the Patrick G Johnston Centre for Cancer Research and Cell Biology at Queens University Belfast. He is internationally renowned for his work in development of novel approaches in the field of antibody and nanomedicine-based therapies for the treatment of cancer and other conditions. Prof. Scott has a background in both the pharmaceutical industry and academia and was a founding scientist of Fusion Antibodies Plc. Research in his laboratory is funded by agencies such as Medical Research Council, UK charities and various industrial sources. He also held a Royal Society Industrial Fellowship with GSK from 2012 to 2015 and won the Vice Chancellors Prize for Innovation in 2015 with his groups work on developing a novel Siglec targeting nanomedicine for the treatment of sepsis and other inflammatory conditions.

The novelty of Avicedas platform technology is its potential to affect immune responses associated with a wide range of disease states, many of which are currently unmet or underserved needs. I look forward to the continued development of Avicedas core technology and moving forward to clinical trials that will pave the way for truly disruptive therapeutic strategies to enter the clinic that will significantly impact and improve patients lives in the not-too-distant future, said Prof. Scott.

Avicedas Scientific advisory chairwoman, Prof. Stanley, is the Horace W. Goldsmith Foundation Chair; Professor, Department of Cell Biology; and Associate Director for Laboratory Research of the Albert Einstein Cancer Center, Albert Einstein College of Medicine, New York. She obtained a doctorate degree from the University of Melbourne, Australia, for studies of influenza virus, and was subsequently a postdoctoral fellow of the Medical Research Council of Canada in the laboratory of Louis Siminovitch, University of Toronto, where she studied somatic cell genetics. Prof. Stanleys laboratory is focused on identifying roles for mammalian glycans in development, cancer and Notch signaling. Among her many varied contributions, Prof. Stanleys laboratory has isolated a large panel of Chinese hamster ovary (CHO) glycosylation mutants; characterized them at the biochemical, structural and genetic levels; and used them to identify new aspects of glycan synthesis and functions. She serves on the editorial boards of Scientific Reports, Glycobiology and FASEB Bio Advances; she is an editor of the textbook Essentials of Glycobiology; and her laboratory is the recipient of grants from the National Institutes of Health. Prof. Stanley has received numerous awards, including a MERIT award from the National Institutes of Health, an American Cancer Society Faculty Research Award, the Karl Meyer Award from the Society for Glycobiology (2003) and the International Glycoconjugate Organization (IGO) Award (2003).

Working with Aviceda represents a unique opportunity to contribute to science at the cutting edge. Its pipeline contains a broad range of candidates that represents numerous first-in-class opportunities, said Prof. Stanley.

Prof. Varki is currently a distinguished professor of medicine and cellular and molecular medicine, Co-director of the Glycobiology Research and Training Center and Executive Co-director for the UCSD/Salk Center for Academic Research and Training in Anthropogeny at the University of California, San Diego; and an Adjunct Professor at the Salk Institute for Biological Studies. Dr. Varki is also the executive editor of the textbook Essentials of Glycobiology. He received basic training in physiology, medicine, biology and biochemistry at the Christian Medical College, Vellore, The University of Nebraska, and Washington University in St. Louis, as well as formal training and certification in internal medicine, hematology and oncology. Dr. Varki is the recipient of numerous awards and recognitions, including election to the American Academy of Arts and Sciences and the US National Academy of Medicine, a MERIT award from the National Institutes of Health, an American Cancer Society Faculty Research Award, the Karl Meyer Award from the Society for Glycobiology and the International Glycoconjugate Organization (IGO) Award (2007).

The Aviceda team is already building on the foundational work in the emerging field of glycobiology to develop potential therapeutics and interventional strategies. Their work could be critically important for growing the understanding of how glycobiology and glycochemistry are applicable to immunology, and more broadly, to the field of drug and therapeutic development, said Prof. Varki.

Prof. Boons is a Distinguished Professor in Biochemical Sciences at the Department of Chemistry and the Complex Carbohydrate Research Center (CCRC) of the University of Georgia (USA) and Professor and Chair of the Department of Medicinal and Biological Chemistry of Utrecht University (The Netherlands). Prof. Boons directs a research program focused on the synthesis and biological functions of carbohydrates and glycoconjugates. The diversity of topics to which his group has significantly contributed includes the development of new and better methods for synthesizing exceptionally complex carbohydrates and glycoconjugates. Highlights of his research include contributions to the understanding of immunological properties of complex oligosaccharides and glycoconjugates at the molecular level, which is being used in the development of three-component vaccine candidates for many types of epithelial cancer; development of convergent strategies for complex oligosaccharide assembly, which make it possible to synthesize large collections of compounds with a minimal effort for structure activity relationship studies; and creation of a next generation glycan microarray that can probe the importance of glycan complexity for biological recognition, which in turn led to identification of glycan ligands for various glycan binding proteins that are being further developed as glycomimetics for drug development for various diseases. Among others, Prof. Boons has received the Creativity in Carbohydrate Science Award by the European Carbohydrate Association (2003), the Horace Isbell Award by the American Chemical Society (ACS) (2004), the Roy L. Whistler International Award in Carbohydrate

Chemistry by the International Carbohydrate Organization (2014), the Hudson Award (2015) and the Cope Mid-Career Scholar Award from ACS (2016).

Aviceda is leading the field of glycoimmunology in exciting new directions. I look forward to working with the company as it pursues multiple lines of development efforts that will someday transform the way immune-inflammatory conditions are treated in the clinic, said Prof. Boons.

Prof. Chouaib is the Director of Research, Institute Gustave Roussy, Paris, where he is active in research in tumor biology. Previously, Prof. Chouaib worked at the French National Institute of Health and Biomedical Research (INSERM) where he led a research unit focused on the investigation of the functional cross talk between cytotoxic cells and tumor targets in the context of tumor microenvironment complexity and plasticity. His research was directed at the transfer of fundamental concepts in clinical application in the field of cancer vaccines and cancer immunotherapy. Prof. Chouaib is a member of the American Association of Immunologists, New York Academy of Sciences, French Society of Immunologists, International Cytokine Society, American Association for Cancer Research, International Society for Biological Therapy of Cancer and American Association of Biological Chemistry. He was awarded the cancer research prize of the French ligue against cancer in 1992 and in 2004 the presidential prize in biotechnology. He was awarded for translational research and scientific excellency by INSERM. His research has resulted in more than 310 scientific articles and several reviews in the field of human immunology, tumor biology and cancer immunotherapy; he has also been an editor for several textbooks.

Dr. Wu is an Associate Professor in the Department of Molecular Medicine at Scripps Research. The current research in the Wu laboratory integrates synthetic chemistry with glycobiology to explore the relevance of protein glycosylation in human disease and cancer immunotherapy. In 2018, Dr. Wu developed a platform to construct antibody-cell conjugates for cancer immunotherapy, which does not require genetic engineering. Previously, while working as a postdoctoral fellow in the group of Professor Carolyn R. Bertozzi at the University of California, Berkeley, Dr. Wu developed an aldehyde-tag (SMARTag) based technology for site-specific labeling of monoclonal antibodies, which served as the foundation for Redwood Biosciences Inc., a biotech company co-founded by Bertozzi. In 2014, Redwood Bioscience Inc. and the SMARTag Antibody-Drug Conjugate technology platform was acquired by Catalent Pharma Solutions.

About Aviceda Therapeutics

Founded in 2018 and based in Cambridge, Massachusetts, Aviceda Therapeutics is a late-stage, pre-clinical biotechnology company with a mission to develop the next generation of glyco-immune therapeutics (GITs) utilizing a proprietary technology platform to modulate the innate immune system and chronic, non-resolving inflammation. Aviceda has assembled a world-class, cross-disciplinary team of recognized scientists, clinicians and drug developers to tackle devastating ocular and systemic degenerative, fibrotic, oncologic and immuno-inflammatory diseases. At Aviceda, we exploit a unique family of receptors found expressed on all innate immune cells and their associated glycobiological interactions to develop transformative medicines. Combining the power of our biology with our innovative cell-based high-throughput screening platform and proprietary nanoparticle technology, we can modulate the innate immune response specifically and profoundly. Aviceda is developing a pipeline of GITs that are delivered via biodegradable nanoparticles and which safely and effectively target numerous immune-inflammatory conditions. Avicedas lead ophthalmic optimized nanoparticle, as an intravitreal formulation, AVD-104, is being developed to target various immune system responses that contribute to pathology associated with age-related macular degeneration (AMD).

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Aviceda Therapeutics Announces Formation of Scientific Advisory Board - BioSpace

Remdesivir approved by FDA to treat Covid-19, but the evidence is mixed – Vox.com

Posted on October 30, 2020 by Dook Domini

The Food and Drug Administration on Thursday gave its first full approval for a drug to treat Covid-19 to the antiviral remdesivir. But some researchers say the FDA is once again promoting a Covid-19 therapy based on shaky evidence.

Developed by Gilead Sciences and marketed under the brand name Veklury, remdesivir previously received emergency use authorization (EUA) from the FDA in May, which allowed it to be used to treat patients with severe Covid-19. In August, the FDA relaxed its guidelines to allow the drug to be used in less serious cases. President Donald Trump also took the drug as part of his treatment when he was diagnosed with Covid-19 earlier in October.

Full FDA approval promotes remdesivir to the standard of care for hospitalized patients, and other potential treatments for Covid-19 will now have to be compared to it during clinical research.

Todays approval is supported by data from multiple clinical trials that the agency has rigorously assessed and represents an important scientific milestone in the Covid-19 pandemic, FDA commissioner Stephen Hahn in a statement Thursday. The FDA based its decision on three randomized controlled trials. (The largest of those looked at 1,062 hospitalized patients.) The trials results showed that remdesivir reduced the length of hospital stays in some Covid-19 patients.

However, shortly before the approval was granted, a study from the World Health Organization announced preliminary results that found the drug had no effect on mortality and unlike the FDAs findings negligible effects on how long patients were in hospitals. The study, known as the Solidarity Trial, recruited almost 12,000 patients, making it the largest Covid-19 treatment study in the world thus far. Researchers say the findings should have given the FDA pause.

I think its really inappropriate to give this a full approval because the data dont support it, said Eric Topol, a professor of molecular medicine at the Scripps Research Translational Institute. What [the FDA] should have done instead of issuing the approval was put on the brakes.

Absent a vaccine, doctors are desperate for an effective treatment for Covid-19, and the FDAs approval of remdesivir finally gives them an option. In the United States, Covid-19 case counts are rising again, with states like Wisconsin opening field hospitals to deal with a looming surge.

But the approval of remdesivir has raised concerns, not only because of the results of the WHOs trial but also because it follows a number of questionable FDA authorizations for other Covid-19 therapies that appear to have been influenced by political pressure from the White House.

Now some researchers and doctors are concerned that remdesivir could not only be less effective than promised, but that its approval could also undermine other efforts to develop better Covid-19 therapies.

Remdesivir seems to be most effective relatively early on for hospitalized patients with severe Covid-19. To help beat back the illness, it interferes with how SARS-CoV-2, the virus that causes Covid-19, makes copies of itself. The virus uses genetic instructions in the form of RNA, written in a code made of molecules represented by the letters A, U, G, and C. The drug mimics the molecule represented by A, adenosine. The fake adenosine blocks the virus from copying itself but doesnt fool human cells. The result is the virus cant reproduce as much within a patients body.

The antiviral drug was originally developed to treat the Ebola virus, and it has received a hefty investment from the US government over almost two decades, as Ekaterina Cleary, lead data analyst and research associate at the Center for Integration of Science and Industry, wrote in a piece for Stat News:

Research from the Center for Integration of Science and Industry, with which I am affiliated, determined that between gathering knowledge behind remdesivirs chemical structure and molecular target, the NIH invested as much as $6.5 billion between 2000 and 2019.

Remdesivir treatment is not without risks. It has been shown to cause some side effects in some people, such as elevated liver enzymes, which could indicate liver damage. The drug can also trigger allergic reactions, resulting in fever, shortness of breath, wheezing, swelling, low blood oxygen, and changes in blood pressure.

For a patient with private insurance, the intravenous drug can cost $3,120 for a five-day course of treatment.

Antivirals like remdesivir are most effective early on during the progression of Covid-19, when most of the damage is being done by the virus itself. Its less effective in later stages, when the problem isnt just the virus. The severe manifestations of the disease are caused by an out-of-control immune response to the infection, said Angela Rasmussen, a virologist at the Columbia University Mailman School of Public Health.

If the immune system gets riled up, it can cause a lot more destruction than SARS-CoV-2 and require more-drastic interventions like intubation, at which point another approach is needed. Thats a big reason why corticosteroids like dexamethasone, which tamp down on the immune system, are the only drugs so far reliably demonstrated to actually reduce Covid-19 mortality.

But giving a patient steroids too early in an infection could prevent the immune system from mounting an effective response against SARS-CoV-2.

Coming up with an effective treatment regimen requires delicately balancing where a patient is in the course of their coronavirus infection and how severe their illness has become. But given how murky it is to identify an infection to begin with let alone confirming the diagnosis and starting the correct treatment during the appropriate window researchers have a hard time teasing out what interventions work best.

Thats why carefully controlled, large-scale clinical trials are so important. And with mixed results coming from the studies conducted to date, some scientists dont think the evidence for remdesivirs effectiveness is enough for the FDA to grant approval.

I was really surprised when I saw that news, Rasmussen said.

The FDA has already made some controversial decisions around Covid-19 drug treatments. The agency granted an EUA for the anti-malaria drug hydroxychloroquine in March, after Trump called it a game changer. The FDA revoked the EUA in June, saying hydroxychloroquine was unlikely to be effective and could cause lead to heart problems.

Then in August, the agency granted an EUA for convalescent plasma to treat Covid-19. But the National Institutes of Health said the evidence used by the FDA was insufficient.

There is more evidence that remdesivir works compared with that of convalescent plasma, but thats not saying much. Its not as weak as the case for plasma, but thats no standard. The case for plasma is nonexistent, said Jeremy Faust, attending physician in emergency medicine at Brigham and Womens Hospital in Boston and an instructor at Harvard Medical School. There is actually randomized controlled trial data that suggests [that] for a subset of patients, remdesivir can decrease hospital length of stay.

The strongest results in favor of remdesivir show that patients who received it had a median recovery time of 10 days, compared to 15 days for those who took the placebo. Its a significant effect, but its not huge, and its certainly not a cure for Covid-19, nor a way to guarantee fewer deaths.

Faust said one of his concerns with the FDAs remdesivir approval is a phenomenon known as indication creep, in which a treatment shown to work in only a limited set of circumstances gets prescribed to more and more people. The worry here is that remdesivir, which is approved only for Covid-19 patients over 12 years old who required hospitalization, could start being used in patients with milder Covid-19 illness, or in more severe cases past the point where it could be effective.

What will happen, I guarantee, is people will start to use the medication more than they need it, Faust said. Since the course of treatment is five days, it could also extend the length of hospital stays in patients who would otherwise be discharged earlier, saddling them with unnecessary costs.

Another concern is that the approval of remdesivir, especially with such mixed evidence for its effectiveness, could undermine further research.

Topol noted that with remdesivir now as the only fully approved drug, it becomes much more difficult to conduct studies on other therapies because they now have to be compared against remdesivir, the new standard treatment, as well as a a placebo.

That raises the cost and complexity of trials, delaying results. Such comparisons are worthwhile if the standard of care is effective, but it adds unnecessary complications if its not.

It also makes it harder to recruit people for subsequent clinical trials of the drug to better validate its effectiveness. People may be more reluctant to sign up for a trial where they could get a placebo when they know they could get the actual drug.

The biggest, most serious problem is that we wont get to the truth, Topol said.

Its worth noting that remdesivir could still be a viable treatment for Covid-19, but the evidence presented so far is contradictory and more investigation is needed to clarify its effectiveness. So why did the FDA go ahead with its approval, then?

Its hard to say, but Herschel Nachlis, a research assistant professor of government at Dartmouth College, suggested the approval might be a strategic move by the agency to deflect political pressure away from the all-important Covid-19 vaccination campaign. Trump has linked a vaccine to his election prospects and blamed the FDA for holding it back. The appearance that a Covid-19 vaccine was rushed to meet political needs could make people reluctant to get vaccinated, so regulators are keen to distance themselves from the 2020 election campaign.

If, in the short term, approving remdesivir gives the President a win and alleviates some pressure on the agency from the President about vaccines, that helps buy the FDA important time, Nachlis told Vox in an email. It might be another case, like convalescent plasma, of giving up some ground in a battle to put yourself in the position to be able to win the broader war.

Whether Nachliss hypothesis is correct isnt yet known. But what is clear is that the evidence on remdesivirs effectiveness appears to be mixed, which is why it would have been helpful for the FDA to have held a public advisory committee meeting to discuss the evidence, a step it typically takes for full pharmaceutical approvals.

Since it may be months before a vaccine for Covid-19 is available, treatments are still urgently needed and other approaches are being studied. Trump, for example, also underwent a course of an experimental monoclonal antibody therapy from the company Regeneron when he was treated for Covid-19. There are multiple clinical trials of these drugs underway, but now they have competition.

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Remdesivir approved by FDA to treat Covid-19, but the evidence is mixed - Vox.com

FDA Approves New FoundationOneLiquid CDx Companion Diagnostic Indications for Three Targeted Therapies That Treat Advanced Ovarian, Breast and…

Posted on October 30, 2020 by Dook Domini

FoundationOne Liquid CDx analyzes the largest genomic region of any FDA-approved comprehensive liquid biopsy test and was approved in August to provide tumor mutation profiling in accordance with professional guidelines for patients with any solid tumor. Concurrently, it was approved as a companion diagnostic for a poly (ADP-ribose) polymerase (PARP) inhibitor approved by the FDA for the treatment of metastatic castration-resistant prostate cancer patients with qualifying BRCA1/2 alterations, and for three first-line EGFR tyrosine kinase inhibitors (TKIs) for the treatment of non-small cell lung cancer patients.

FoundationOne Liquid CDx offers oncologists an important and minimally invasive tool to consider when making treatment decisions for their patients, regardless of the type of cancer they have, said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. These three additional companion diagnostic claims expand the tests clinical utility into breast and ovarian cancer, demonstrating our commitment to bringing precision medicine to more patients, and we plan to continue working with our biopharma partners to increase that reach.

Todays approval expands FoundationOne Liquid CDxs companion diagnostic indications to include the following targeted therapies:

PIK3CA is the most commonly mutated gene in HR+/HER2- breast cancer; approximately 40% of patients living with HR+/HER2- breast cancer have this mutation.1

An estimated one in four women with epithelial ovarian cancer have a mutation of the BRCA1 or BRCA2 gene.2

Using a blood sample, FoundationOne Liquid CDx analyzes over 300 cancer-related genes for genomic alterations. FoundationOne Liquid CDx results are delivered in an integrated report that identifies alterations matched to FDA-approved therapies. It also enables accelerated companion diagnostic development for biopharma companies developing precision therapeutics.

As a laboratory professional service which has not been reviewed or approved by the FDA, the FoundationOne Liquid CDx report delivers information about the genomic signatures microsatellite instability (MSI) and blood tumor mutational burden (bTMB), as well as single gene alterations, including NTRK fusions, to help inform the use of other therapies including immunotherapies. Also, as a laboratory professional service, the report provides relevant clinical trial information and includes interpretive content developed in accordance with professional guidelines in oncology for patients with any solid tumor.

About FoundationOne Liquid CDx

FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and mutation status confirmed using an FDA-approved tumor tissue test, if feasible. For the complete label, including companion diagnostic indications and complete risk information, please visit http://www.F1LCDxLabel.com.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

PIQRAY is a registered trademark of Novartis AG.

RUBRACA is a registered trademark of Clovis Oncology, Inc.

ALECENSA is a registered trademark of Chugai Pharmaceutical Co., Ltd., Tokyo, Japan.

Source: Foundation Medicine

1 The Cancer Genome Atlas Network. Comprehensive molecular portraits of human breast tumours. Nature. 2012;490(7418):61-70.2 Pennington et al, Clin Cancer Res. 2014; 20(3):764-7753 Dearden et al. Ann Oncol. 2013 Sep; 24(9): 23712376.

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FDA Approves New FoundationOneLiquid CDx Companion Diagnostic Indications for Three Targeted Therapies That Treat Advanced Ovarian, Breast and...

How Many Have Recovered From Covid-19 Cases? No One Knows. – msnNOW

Posted on October 30, 2020 by Dook Domini

Gabriela Bhaskar for The Wall Street Journal Coronavirus recoveries are undercounted in public-health statistics.

Health researchers collect various pieces of Covid-19 data to get a handle on the spread of the new coronavirus, but one metric has proven tough to pin down: how many infected people have recovered.

Among the 8.8 million coronavirus cases reported in the U.S. so far, some 3.5 million have recovered, according to Johns Hopkins University, which tracks the pandemic.

Yet the tally of recovered Covid-19 patients misses the mark, health experts say, while also failing to capture the many people who are struggling with lingering medical issues from their cases.

The data are so spotty, public-health authorities say they dont know what the true count is. The national figure displayed on trackers likely misses the true count by millions, estimates Ashish Jha, dean of the Brown University School of Public Health.

The spottiness stems from the absence of both an agreed-upon definition for a coronavirus recovery and a standardized way to track the numbers of patients, the health experts say. What constitutes recovery is so nebulous that some states dont even track it, and those that do probably undercount the true number.

Im not aware of anyone using the recovery figures, said Marcus Plescia, chief medical officer of the Association of State and Territorial Health Officials. Ive seen that data, and Ive not paid attention to them.

The lack of clarity means the popular picture of Covid-19s toll misses a datapoint indicating that most infected people have overcome the illness. And it highlights the challenges in assembling data that would help health authorities track the virus and help people fight it.

Like a lot of these issues with data reporting for Covid, theres really not a clear standard for how to do it, said Lauren Gardner, a Johns Hopkins University associate engineering professor who leads the team that built the schools widely cited Covid-19 dashboard.

Both the general public and many health professionals tend to consider patients recovered if they feel the way they did before they became ill. Yet many states define coronavirus recovery differently.

These states count a Covid-19 case as a recovery simply because time has passed since a person developed symptoms or was discharged from the hospital.

Michigan defines recovered cases as the number of people with a confirmed Covid-19 diagnosis who are alive 30 days after getting symptoms. Texas estimates recoveries under a complex formula that subtracts deaths and certain other cases, and assumes it takes 32 days for hospitalized patients to recover and 14 days for nonhospitalized patients.

Such measurements might indicate how many people who tested positive for the coronavirus didnt die, but might miss those who never displayed symptoms and didnt undergo testing.

The metrics also miss the viruss individual impact, according to health experts. While some patients bounce back quickly, others can struggle with side effects for weeks or even months.

There is a variable path after people get sick, said Eric Topol, a cardiologist and professor of molecular medicine at the Scripps Research Institute. There are people who are still ill months after they get sick. We know those symptoms can be very severe, and people can be debilitated.

Given the complexities, some states dont try to count how many have recovered from Covid-19.

The California Department of Public Health said assessing who is recovered is too subjective, and the persistence of side effects in a subset of Covid-19 patients known as long-haulers makes it hard to get a good count.

The Florida Department of Health also doesnt provide a specific recovery metric. It said relying on hospital discharges or estimating length of illness doesnt capture recovery.

Without data from every state and accurate figures from states reporting data, the recovery numbers given by Covid-19 trackers available online are useless for assessing how many people have recovered, said Jennifer Nuzzo, an epidemiologist and senior scholar at the Johns Hopkins Center for Health Security.

Dr. Nuzzo said she doesnt know what the true number of recovered patients is.

Covid-19 trackers get their recovery figures from states that are reporting the numbers. Alexis Madrigal, a founder of the widely cited Covid Tracking Project, said he has sometimes wanted to stop recording recoveries because of too much imprecision in what constitutes a recovery and in tallying them.

Most recovery definitions are not what they purport to be or what people expect, said Mr. Madrigal, who is also a staff writer at The Atlantic magazine.

Donna Bourne, of Shelby, Ohio, got sick and tested positive for Covid-19 in late March. Seven months later, she said, she still struggles with fatigue and severe shortness of breath climbing the stairs in her home. She uses her asthma inhaler every day, as opposed to once a week before getting Covid-19. She now also has an irregular heartbeat, recall problems and sometimes drools because the lower part of her face is partially paralyzed.

Despite her continued health issues, she fits the description of presumed recovered in Ohio, because she is still alive more than 21 days since she first developed symptoms.

Your body just does not recover from Covid the way it would from the flu, said Ms. Bourne, 55 years old, who was recently furloughed from her job assisting workers with disabilities. Its really, really hard on you.

Many state and local health departments ignore recovery tallies in gauging the progress of the pandemic.

Without a standard definition of Covid-19 recovery, there isnt a way to compare data between counties or cities, or to tell how well people living in these places are recovering, said Oscar Alleyne, chief of programs and services for the National Association of County and City Health Officials.

What health authorities could use is a figure capturing how people infected with Covid-19 are faring over time, Dr. Alleyne said. Armed with such data, public-health departments would be better equipped to write guidelines for treatment and allocate the resources that doctors and patients need, he said.

Write to Sarah Toy at sarah.toy@wsj.com

How Many Have Recovered From Covid-19 Cases? No One Knows. - msnNOW

COVID-19 in the continent’s children: UK award supports new research – University of Cape Town News

Posted on October 30, 2020 by Dook Domini

Professor Heather Zar, the chair of the Department of Paediatrics and Child Health and the director of the South African Medical Research Council Unit on Child and Adolescent Health at the University of Cape Towns(UCT) Faculty of Health Sciences, is spearheading a research project that is one of 12 globally to receive a recent COVID-19 grant.

The National Institute for Health Research / United Kingdom Research and Innovation Global Effort on COVID-19(NIHR/UKRI GECO) grant is a collaborative funding opportunity. Under Professor Zar as the project leader, the study will offer a unique opportunity to understand the determinants of severe acute respiratory syndrome coronavirus2(SARS-CoV-2) infection and coronavirus disease(COVID-19) in children in Africa in a low-middle-income country(LMIC) context. The award will enable Zar and her team to undertake a study titled Spectrum, determinants and long-term outcome of SARS-CoV-2 infection and disease in African children.

The reasons children typically develop mild illness or have asymptomatic infection are poorly understood. In LMICs, where children make up a large proportion of the population, risk factors such as malnutrition, HIV exposure, tuberculosis or prior infection with endemic coronaviruses may have an impact on the risk of infection and development of COVID-19.

This project aims to investigate the spectrum of illness in African children, the risk factors for infection or disease, and the immune or inflammatory factors protecting children against SARS-CoV-2 infection or severe COVID-19 disease.

Zar is collaborating with partners at the universities of Western Australia and Southampton in the United Kingdom for this research.

COVID-19 infection in African children

This funding provides a wonderful opportunity to better understand COVID-19 in African children in an LMIC context, she said.

This is particularly relevant because of the high burden of pneumonia, which continues to be the major single killer of children under five years of age, due to factors such as malnutrition, smoke exposure and the high burden of infectious disease in these settings.

Understanding why children are only mildly affected may be key to developing new strategies to prevent or ameliorate illness.

However, this hasnt occurred with COVID-19, said Zar, who is an affiliate member of the Institute of Infectious Disease and Molecular Medicine(IDM) at UCT. Surprisingly, children in LMICs and globally are predominantly only mildly affected by COVID-19, with relatively few severe cases or deaths occurring in young children. The current project will investigate whether prior infection with other organisms (including seasonal coronaviruses) protects children against severe disease through development of immunity.

Understanding why children are only mildly affected may be key to developing new strategies to prevent or ameliorate illness, said Zar.

Drakenstein Child Health Study

Whitney Barnett, the projects programme manager, said that this funding will offer researchers the additional focus of investigating COVID-19 across different settings, ranging from communities to hospitals. SARS-CoV-2 infection will be investigated in children who are hospitalised with pneumonia as well as in children who are part of the population-based Drakenstein Child Health Study(DCHS), anovel African birth cohort study, which is led by Zar.

The DCHS has comprehensively investigated the early-life determinants of child health, and developmental pathways to health or disease from pregnancy through childhood so it provides a unique platform to study COVID-19 in children, and the impact of the pandemic on child health.

It is especially important to be able to do this study here because children make up a high proportion of the population.

The DCHS also bridges the intersection of infectious diseases and the emergence of non-communicable diseases, Zar said.

It is especially important to be able to do this study here because children make up a high proportion of the population, and risk factors such as malnutrition, pollution, poverty and a high burden of infections may contribute to their vulnerability to developing illness.

She added that the context of the DCHS offers further understanding of COVID-19-related childhood illness, including the protective or risk factors for infection or disease that have been carefully measured from the antenatal period through childhood, and the role of inflammation.

As a child health specialist in respiratory illness, Zar said that this will inform future research and healthcare approaches and provide a unique opportunity to generate new knowledge, identify risk factors for illness and develop novel strategies for prevention and treatment.

COVID-19 in the continent's children: UK award supports new research - University of Cape Town News

FoundationOneCDx Receives FDA-Approval as a Companion Diagnostic for VITRAKVI(larotrectinib) to Identify Patients with NTRK Fusions Across All Solid…

Posted on October 30, 2020 by Dook Domini

Foundation Medicine, Inc. today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneCDx to be used as a companion diagnostic for VITRAKVI (larotrectinib), which is currently FDA-approved for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. FoundationOne CDx, the only FDA-approved tissue-based comprehensive genomic profiling (CGP) test, is now approved to detect NTRK1/2/3 fusions across all solid tumor types and identify patients who may be appropriate for treatment with VITRAKVI.

NTRK fusion positive cancer occurs when a piece of the chromosome containing the NTRK gene breaks off and binds to another chromosome. These NTRK gene fusions produce TRK fusion proteins, which may cause cancer cells to grow.1 NTRK fusions are more commonly found in rare cancer types, such as secretory carcinoma of the breast or salivary gland and infantile fibrosarcoma, but they can also occur across many more common cancer types including glioma, melanoma and carcinomas of the lung, thyroid and colon.2

"Taking a comprehensive and validated approach to genomic testing is critical for all advanced cancer patients, but especially for those harboring rare mutations that can be missed with alternative testing methods," said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. "Not only will this approval improve access to genomic testing and reinforce the role it plays in rare cancers, but it also confirms the incredible progress made toward tumor-agnostic cancer care. We're proud of the impact this will have on NTRK fusion positive cancer patients."

FoundationOne CDx is the first FDA-approved broad companion diagnostic that is clinically and analytically validated for solid tumors. The tissue-based comprehensive genomic profiling test is currently approved as a companion diagnostic for more than 20 targeted therapies.

"Many patients with rare conditions, like NTRK fusion positive cancer have limited treatment options and poor access to targeted therapies," said Susan Spinosa, co-chair and patient founder of the NTRKers. "This companion diagnostic approval is a critical step forward in addressing this challenge as it provides this patient population with broader access to comprehensive genomic testing and appropriate treatment options."

The approval of VITRAKVI was based on data from three multicenter, open-label, single-arm clinical trials: LOXO-TRK-14001 (NCT02122913), SCOUT (NCT02637687), and NAVIGATE (NCT02576431). Identification of positive NTRK gene fusion status was prospectively determined in local laboratories using next generation sequencing (NGS) or fluorescence in situ hybridization (FISH). NTRK gene fusions were inferred in three pediatric patients with infantile fibrosarcoma who had a documented ETV6 translocation by FISH. The major efficacy outcome measures were overall response rate (ORR) and response duration, as determined by a blinded independent review committee according to RECIST 1.1. The clinical validation to support the FoundationOne CDx NTRK companion diagnostic approval was then achieved through a clinical bridging study between the local clinical trial assays and FoundationOne CDx.

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed paraffin embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit http://www.foundationmedicine.com/genomic-testing/foundation-one-cdx.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patient's cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicine's molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

Vitrakvi is a registered trademark of Bayer

Source: Foundation Medicine

1 National Cancer Institute. "NTRK Gene Fusion." https://www.cancer.gov/publications/dictionaries/cancer-terms/def/ntrk-gene-fusion

2 "Annals of Oncology. "Identifying patients with NTRK fusion cancer" https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6859817/

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FoundationOneCDx Receives FDA-Approval as a Companion Diagnostic for VITRAKVI(larotrectinib) to Identify Patients with NTRK Fusions Across All Solid...

The body fires ‘blobs of fat’ packed with toxic proteins to fight bacteria – Live Science

Posted on October 19, 2020 by Dook Domini

The human body uses many tactics to fight invaders. Scientists just found another weapon in its arsenal: tiny fat blobs packed with toxic proteins that are fired at the enemy.

Complex organisms including fungi, plants and animals are made up of eukaryotic cells that contain subcellular structures called organelles. These organelles all work together to keep the cells functioning; the nucleus, for example, is the brain of the cell and lipid droplets (LDs) are teeny blobs of fat that store and provide fuel for the cell when needed.

Lipid droplets are usually attached to the mitochondria an organelle that generates most of the cell's energy and serve as a source of fuel when needed. Previous research has found that certain parasites, viruses and bacteria steal these droplets and also use them to fuel growth. So until now, scientists thought that these lipid droplets supported infection, the authors wrote in a new study published Oct. 15 in the journal Science.

Related: 5 ways gut bacteria affect your health

"It was previously thought that bacteria were merely using the lipid droplets to feed on, but we have discovered these fatty droplets are involved in the battle between the pathogens and our cells," co-author Robert Parton, head of the cell biology and molecular medicine division of the University of Queensland's Institute for Molecular Bioscience in Australia, said in a statement. Previously, scientists found that exposing fruit flies to a microbe induced formation of lipid droplets with antimicrobial activity, according to an accompanying commentary in the journal Science.

Parton and his team wanted to see whether this strange battle technique also existed in mammalian cells. They injected mice with lipopolysaccharide, a toxin that's produced by bacteria. The toxin spurred threatened cells in the mice's liver to produce more lipid droplets and increase the size of existing ones, according to the study.

In cells infected with the toxin, threatened cells packed lipid droplets with hundreds of antiviral and antibacterial proteins.The scientists also found that the droplets detached from the mitochondria and moved toward the bacterial toxin.

"Fat is part of the cell's arsenal cells manufacture toxic proteins, package them into the lipid droplets, then fire them at the intruders," Parton said. "This is a new way that cells are protecting themselves, using fats as a covert weapon, and giving us new insights into ways of fighting infection."

The researchers also saw a similar response when they exposed human macrophages a type of white blood cell that helps detect and destroy pathogens and problem cells to the bacterial toxin in the lab. Now, Parton and his team hope to figure out how the lipid droplets actually target the bacteria, he said in the statement. "By understanding the body's natural defenses, we can develop new therapies that don't rely on antibiotics to fight drug-resistant infections."

Though scientists are just discovering this defense strategy now, these droplets were first discovered more than 130 years ago, and they are present in all types of eukaryotic cells, according to the commentary.

"There is great and justifiable excitement regarding the functions of LDs and other membraneless organelles," and how they change in many cellular processes, Douglas Green, the chair of the immunology department at St. Jude Children's Research Hospital in Tennessee, wrote in the accompanying commentary. "We have much to learn about these drops of oil in cells."

Originally published on Live Science.

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The body fires 'blobs of fat' packed with toxic proteins to fight bacteria - Live Science

Study shows a molecular dance that keeps your heart beating – WSU News

Posted on October 19, 2020 by Dook Domini

A microscope photograph of a heart muscle cell. The regular green patterns show stained actin filaments.

By Tina Hilding, Voiland College of Engineering and Architecture

It might look like a little game at the molecular scale.

Filament-like proteins in heart muscle cells have to be exactly the same length so that they can coordinate perfectly to make the heart beat.

Another protein decides when the filament is the right size and puts a wee little cap on it. But, if that protein makes a mistake and puts the cap on too early, another protein, leiomodin, comes along and knocks the cap out of the way.

This little dance at the molecular scale might sound insignificant, but it plays a critical role in the development of healthy heart and other muscles. Reporting in the journal, Plos Biology,a WSU research team has proven for the first time how the mechanism works.

The finding could someday lead to improved diagnostics and medical treatments for serious and sometimes devastating hereditary heart conditions that come about from genetic mutations in the proteins. One of these conditions, cardiomyopathy, affects as many as one in 500 people around the world and can often be fatal or have lifetime health consequences. A similar condition called nemaline myopathy affects skeletal muscles throughout the body with often devastating consequences.

Mutations in these proteins are found in patients with myopathy, saidAlla Kostyukova, associate professor in the Gene and LindaVoiland School of Chemical Engineering and Bioengineeringand leader of the project. Our work is to prove that these mutations cause these problems and to propose strategies for treatment.

Heart muscle is made of tiny thick and thin filaments of proteins. With the help of electrical signals, the rope-like filaments bind and unbind in an intricate and precise architecture, allowing heart muscle to contract and beat.

The thin filaments are made of actin, the most abundant protein in the human body. Tropomysin, another protein, wraps itself around the actin filaments. Tropomyosin together with two other proteins, tropomodulin and leiomodin, at the end of the actin filaments act as a sort of cap and determine the filament length.

Its beautifully designed, said Kostyukova, whose research is focused on understanding protein structures.

And, tightly regulated.

To keep heart muscle healthy, the actin filaments, which are about a micron long, all have to be the exact same length. In families with cardiomyopathy, genetic mutations result in formation of filaments that are either too short or too long. Those affected can have significant heart problems that cause disability, illness and death.

In a project that spanned seven years, the researchers proved that leiomodin attaches to the end of the actin filament and kicks out the other protein, tropomodulin, to assure the actin filaments proper length.

This is the first time that this has been shown with the atomic-level precision, said Dmitri Tolkatchev, research assistant professor in the Voiland School and lead author on the paper. Previously, several laboratories attempted to solve this problem with very little success. With our data we finally have a direct proof.

The researchers used state-of-the-art approaches to make the key proteins and study them at the molecular and cellular level. The work entailed designing the molecules, constructing them at the gene level in a plasmid, and then producing them into bacterial or cardiac cells. The researchers used nuclear magnetic resonance, which works on the same physical principle as Magnetic Resonance Imaging (MRIs), to understand the proteins binding at the atomic level. They also used molecular dynamic simulation to model them.

The probability of being able to show this mechanism was not high, but the impact of the discovery is, said Tolkatchev, an expert in nuclear magnetic resonance. This was a very important problem to study and could have a significant impact in the field of muscle mechanics.

The researchers hope to continue the work, identifying additional components and molecular mechanisms that regulate thin filament architecture, whether diseased or healthy.

The multidisciplinary group included researchers from the University of Arizona led by Carol Gregorio, director of the Cellular and Molecular Medicine Department. WSUs group has expertise in protein structure, structural biochemistry, and properties of actin filaments and regulatory proteins, and UAs group has expertise in molecular, cellular and developmental biology of muscle assembly. The collaborative work was funded by the National Institutes of Health.

Invitae CEO on how COVID-19 testing could have a positive impact on the future of genetics testing – Report Door

Posted on October 19, 2020 by Dook Domini

Invitae CEO Sean George joins Yahoo Finances on the move to talk about how Invitae is bringing genetic testing and information into mainstream discussions.

JULIE HYMAN: Im watching shares of Invitae, which have just about tripled this year. We are joined now by the CEO, Sean George, from San Francisco. Sean, thanks for joining us. You guys do diagnostic testing and you dont do it for COVID specifically, but you do it for a host of other diseases. And weve obviously seen COVID-19 testing really expand. You can drive up and get it in places. There are now going to be at-home tests that are going to be introduced. Do you think that this then gets people more comfortable with testing for a range of different diseases? Or are you hoping that it does?

SEAN GEORGE: Well, I think it does. And I think in general, trend of the last decade or so is people are starting to understand that this diagnostic information is very useful in advance of when theres an actual problem. Obviously, people are now getting very familiar with COVID testing, what it could have done prior to, and certainly during an outbreak and to manage it.

And when you think about how fundamental in an individuals genomic information is for their health, the idea is starting to catch hold with more and more people, especially generalists, and the idea that genetic information used at the right time, ahead of time, at every stage in life, has profound implications on an individuals health.

ADAM SHAPIRO: So these kinds of tests, we keep hearing in relation to COVID, the issue of false positives. But putting that to the side, in the traditional diagnostic models, whats an accepted, if there is, metric, for when youre going to get a false positive or false negative? How does the industry deal with that issue?

SEAN GEORGE: Right, and especially in medical genetics, that threshold is very, very, very, very tight. Its exacting on 99 and a few nines of how the percentage of sensitivity and specificity these tests need to have because of the profound implications of genetics. Whether its dealing with cancer, cardiovascular disorders, pediatric disorders, battling cancer as 45 million people are per year in the markets we serve, and to answer those fundamental questions and then take concrete medical next steps. The sensitivity specificity, the precision of the tests need to be extraordinarily high.

Story continues

BRIAN CHEUNG: Hey, Sean, Brian Cheung here. So for those that maybe arent that well versed in this type of science, explain to us exactly where you fit in the business world of things that might be tangentially related to genetic testing. I mean, do you guys work with say CRISPR firms or types of, I guess, diagnostic or treatment relative to businesses in that space? Where does your business fit in that universe?

SEAN GEORGE: I think the way to think about it, the people who, certainly anybody who has had an introduction or a brush with genetics and the health care, their clinician, were the number one brand in medical genetics around the globe. Their clinician certainly knows us and has been working with us for many years. And again, whether thats to diagnose any number of 1,000 rare disorders, to help an individual understand their prognosis and next best steps for cancer treatment.

Certainly around reproductive health. You may have heard carrier screening or non-invasive prenatal screening. Those are the kind of tests that we do. And we primarily do it with your clinician. Certainly up until now, mostly the specialists of the world, the medical genetics, the various conditions. Like I said, there are thousands. Ever increasingly, as we have dramatically driven down the price of the, weve driven down the cost of this testing by almost two orders of magnitude over the last 10 years.

And ever increasingly, generalists are starting to use this information. Theyre becoming more comfortable with it. We have tools and front end capabilities to allow clinicians to use the information without the deepest of knowledge around each individual gene, around each individual condition. And have tools online to follow the patients, follow up on their results, make sure they understand what the next best step is, and really support the clinician, the patient with the use of genetics in mainstream medical care.

JULIE HYMAN: And Sean, when were talking about genetics testing, is it mostly probability testing, particularly for cancers when you talk about carrier testing that youre at X percent risk for developing a certain disorder? Or is genetics testing also increasingly being used for actual diagnosis?

SEAN GEORGE: The kind of testing we specialize, the kind of testing put into play by most clinicians, medical geneticists, is a little more precise than just a very slight probability increase over average risk. These are, carrier screens, you mentioned, this will tell you whether you and your partner are carrying different conditions. It will tell you what are the odds specifically of having a child who might be affected by the condition. Or if you were to have a child, what would the odds be.

Ill take, for example, some of the cardiovascular diseases or cancer genetic diseases. If you have some of these genes that are affected, your risk is 30 to 80 times the population average risk of these conditions. So its quite prognostic. Now like I said, when you do get an answer, it really helps to diagnose the condition far earlier than you would otherwise be able to without the use of genetics.

And then depending on the disease, there are a variety of next steps, all the way from preventive approaches to therapies. And we work with over 100 biopharmaceutical partners too, who have therapies that are specifically targeted against genetic conditions that we can introduce our patients to as early as possible.

JULIA LA ROCHE: Sean, this is such a fascinating discussion, what youve been bringing up, and youre talking about the next best action. And its such an interesting way to look at health care, especially looking at it from preventative as well, or even just having a better understanding of your genetics and what it could mean or what the outcomes can be. I guess for you, if we could just kind of step back, what is the moonshot for you when it comes to your business and the broader health care system?

SEAN GEORGE: And in fact, were kind of, Im not sure at what inning we are in of that moonshot, I think its probably early, first or second. But the real idea, and when we founded the company, it was a simple idea. Genetics is so impactful for health care on an individual basis, yet is sparingly used at all in modern health care around the globe. The moonshot is really to have an individuals genome available, managed on their behalf, so that information can be put into play at the right point, at the right time, with all of the people around them that can help put that information to use for their health.

So for example, upon birth. 3% of live births end up in the NICU or the PICU because of genetic conditions. Having that information instantaneously would dramatically improve the outcome of the children in that situation. Some 20% to 30% of populations primary health care complaint over their lives will be genetic in nature.

Knowing that ahead of time instead of after years and years and years of symptoms kind of popping up and then getting worse over time and finally getting diagnosed, knowing ahead of time can greatly improve the preventive measures taken. I think we will in five to 10 years, we will look back in horror thinking that women had children, started families without accessing the fundamental genetic information that can introduce so much risk along the way, either for the women themselves during carrying to term, and then certainly upon delivery.

And I think cancer is another example. Its one of the more advanced conditions when considering molecular medicine. I think again, in 10 years well look back, and the idea that we didnt access the fundamental information about a persons genetic background and the genomics of the cancer itself to precision tailor treatment to them, I think well look back and wonder why we didnt do it faster.

But again, a big problem with that has been cost in the past, and weve taken care of that and are continuing to work on it. So I think across all stages in life, were about to see here in the next decade this genetic information to be used almost as a medical utility. Again, and our aim is, the moonshot is to across all stages of life, all the way from birth until youre dealing with aging and senescence.

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Invitae CEO on how COVID-19 testing could have a positive impact on the future of genetics testing - Report Door

Curing the incurable: teaching an old drug new tricks to fight ovarian cancer – The Guardian

Posted on October 19, 2020 by Dook Domini

In May, PhD students who are funded by the Medical Research Council (MRC) were invited to enter the Max Perutz science writing award 2020 and to tell the general public why your research matters. From the many entries received, the 10 that made the shortlist covered diverse topics, including motor neurone disease, self-harm, babies experiences of pain, and bone loss resulting from space travel.

The essays were judged by the Observers Ian Tucker, the Science Museums Roger Highfield, Prof Fiona Watt from the MRC, Bristol Universitys Andy Ridgeway and the journalist and broadcaster Samira Ahmed.

At a virtual ceremony last Tuesday, the 1,500 prize was presented to the winner, Sarah Taylor from the MRC Institute of Genetics and Molecular Medicine at the University of Edinburgh for her article about her research into the influence of proteins on the effectiveness of chemotherapy.

Here we publish the winning article, described by Samira Ahmed as a terrifically told and intriguing story.

She sits in the small consulting room once again, waiting to hear the news of her latest scan. It has been a difficult journey since the last time she sat in this chair, before her most recent round of treatment began. Over a month of exhaustion, vomiting, soreness, sleepless nights and the inevitable hair loss. But this time, the chemotherapy has not been successful. After all the side effects, all the pain that she has endured, her tumour is still growing, a dark mass on her ovary. Where does she go from here? What can she do when the treatment shes pinned all her hopes on just stops working?

This situation is all too common for women with high grade serous ovarian cancer (HGSOC), a devastating form of ovarian cancer. Only 35% survive longer than five years following their diagnosis. While chemotherapy and surgery are highly effective at initially shrinking tumours, the cancer continues to fight back. Over time the tumour changes, with cells that survive treatment prevailing and replicating, passing on the protective traits that give them that survival edge. The tumour becomes completely resistant to chemotherapy, and no barrier remains to stop it from growing out of control and overwhelming the body.

However, there are groups of patients whose cancers are much more sensitive to chemotherapy treatment than others, who can be completely cured by chemotherapy. One key to this is DNA repair proteins, the tools that all cells use to protect their DNA from damage. Think of this DNA as the instruction manual for a cell, detailing how to build all the proteins the cell requires to live and carry out different functions. Cancer cells often have defective DNA repair proteins, as this allows them to adapt and grow rapidly. Strange as it may sound, this can be a good thing from our perspective! Chemotherapy kills cancer cells by attacking their DNA, and those which lack DNA repair proteins essentially forgot to bring a first aid kit they cannot fix themselves up and keep going. This means that the chemotherapy can completely kill off the cancer, so the patient will survive. This reveals gaps in the armour of this cancer, which we can exploit to help the women who need it most.

No two cancers are quite the same, even within a specific type like HGSOC. Some have completely functional DNA repair proteins. Some have defective proteins initially but can adapt and fix these. Others can make excessive amounts of the proteins to combat the effects of chemotherapy and survive. I hope that by learning what happens to these proteins as a cancer cell becomes resistant to chemotherapy, I can make new drugs to prevent the crucial DNA repair proteins from functioning, which will enable the chemotherapy to kill cancer cells more effectively.

The first question that I asked was which, if any, of these proteins are actually important for the way HGSOC reacts to chemotherapy. I used cells taken from HGSOC patient tumours and adapted to grow easily in the lab, called cell lines, which have similar properties to an actual tumour in a patient. By using cell lines taken from a selection of patient tumours, scientists can build up a picture of the similarities and differences between patient tumours. I started by assessing the growth of various cell lines when treated with a drug called carboplatin, the standard chemotherapy used to treat HGSOC. The slower the cells grow, the more effective it is as a treatment. I found that there was a lot of variation in sensitivity to carboplatin between the different cell lines unsurprising really since one of the main challenges in cancer research is how many differences there are between individuals tumours, and even between different parts of the same tumour.

Next, I set out to find the reason for these differences, looking for changes in the DNA repair proteins. I studied a database of ovarian cancer patients looking for clues on what could be going on, and found that it is common for the tumour cells to produce either abnormally high or low amounts of certain DNA repair proteins. So, I decided to measure the amount of repair proteins produced by my cell lines. I found that in the cell line that was most sensitive to chemotherapy, one of these repair proteins was almost entirely missing! This is a really good indicator that this protein could be an important factor behind repairing the damage caused by chemotherapy.

So, I had identified a protein potentially involved in chemotherapy effectiveness. What next? I wanted to confirm that this protein acts in the way I suspected within the cancer cells. I blocked the cell lines from producing the protein I was interested in, and again looked to see how sensitive to chemotherapy the cancer cells were. This confirmed my initial suspicions removing the protein made the cancer cells much more susceptible to chemotherapy!

As I am only in my first year of working on this project, there is still much to be done, but this is an exciting starting point. I certainly find it very exciting! I plan to study the mechanism used by these cancer cells to alter the amount of this repair protein, and see how smart the cancer cells are are they cheating the chemotherapy by producing more of this protein to prevent the cells from being killed? Does this result in a chemotherapy-resistant tumour? Most importantly, I would like to identify patients whose cancers have high levels of this repair protein, for whom conventional chemotherapy might be less effective, and focus on how I can help them. To tackle this problem, I would like to test drugs that block this protein from carrying out DNA damage repair, leaving the cancer powerless, unable to repair the damage inflicted by chemotherapy. My dream is that one day this will help more women to leave that consulting room feeling victorious, having beaten the odds, and able to shut the door for good on their way out.

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Curing the incurable: teaching an old drug new tricks to fight ovarian cancer - The Guardian

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UArizona researchers have breakthrough related to stomach cancer – Eastern Arizona Courier

Aviceda Therapeutics Announces Formation of Scientific Advisory Board – BioSpace

Remdesivir approved by FDA to treat Covid-19, but the evidence is mixed – Vox.com

FDA Approves New FoundationOneLiquid CDx Companion Diagnostic Indications for Three Targeted Therapies That Treat Advanced Ovarian, Breast and…

How Many Have Recovered From Covid-19 Cases? No One Knows. – msnNOW

COVID-19 in the continent’s children: UK award supports new research – University of Cape Town News

FoundationOneCDx Receives FDA-Approval as a Companion Diagnostic for VITRAKVI(larotrectinib) to Identify Patients with NTRK Fusions Across All Solid…

The body fires ‘blobs of fat’ packed with toxic proteins to fight bacteria – Live Science

Study shows a molecular dance that keeps your heart beating – WSU News

Invitae CEO on how COVID-19 testing could have a positive impact on the future of genetics testing – Report Door

Curing the incurable: teaching an old drug new tricks to fight ovarian cancer – The Guardian