Category Archives: Cell Medicine

ROSEMONT, Pa., Oct. 19, 2020 /PRNewswire/ - Medunik USA is proud to announce that Siklos (hydroxyurea), indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises1, is now available in a 100 mg scored tablet in addition to the 1,000 mg triple-scored tablets.

Siklos has a Boxed Warning regarding low blood cell counts and cancer; please read Important Safety Information below.

"Medunik USA is proud to offer pediatric SCA patients a new option that helps optimize daily dosing. With the newly available Siklos 100 mg scored tablets, dose adjustments can now be made in 50 mg increments. Coupled with Siklos 1,000 mg triple-scored tablets (4 x 250 mg), this will offer more accurate dose adjustmentsand may make it more convenient than compounded hydroxyurea," affirmed Tanya Carro, General Manager, at Medunik USA.

The importance of optimal dosing of hydroxyurea in sickle cell anemia (SCA) patients, based on patient body weight and biological and clinical response, has been well established.2,3 This is particularly relevant in pediatric populations, where patient weight is constantly changing.

In addition to its flexible dosing, Siklos may help increase patient compliance, as it is dissolvable in water for patients who are unable to swallow tablets whole.4 Siklos tablets should be taken once daily, at the same time every day, with a glass of water. For patients who are not able to swallow the tablets, they can be dispersed immediately before use in a small quantity of water in a teaspoon.

"I can't stress enough the importance of treating pediatric patients as early as possible. Why wait for painful episodes to occur and risk complications when you can give a child Siklos and reduce the frequency of recurrent painful crises once they turn 2? There's no reason to wait. Physicians and parents need to know about Siklos!," said Dr. Corey Hebert, MD, Chief Medical Officer at Dillard University and well-known medical broadcast journalist.

Medunik is committed to providing Siklos at the lowest possible cost to all patients. That is why the company continues to offer cost savings and free home delivery through the Siklos At Home program.

For more information about prescribing Siklos tablets, please visit siklosusa.com.

SIKLOS (hydroxyurea) tablets, for oral use

WHAT IS SIKLOS?

SIKLOS is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises.

It is not known if SIKLOS is safe and effective in children less than 2 years of age.

IMPORTANT SAFETY INFORMATION

WARNING: LOW BLOOD CELL COUNT and CANCERSee full prescribing information for complete Boxed Warning.

WHAT IS THE MOST IMPORTANT INFORMATION YOU SHOULD KNOW ABOUT SIKLOS?

WHO SHOULD NOT TAKE SIKLOSDo not take SIKLOS if you are allergic to hydroxyurea or any of the ingredients inSIKLOS.See the Medication Guide for a list of the ingredients inSIKLOS.

WHAT SHOULD YOU TELL YOUR HEALTH CARE PROVIDER BEFORE TAKING SIKLOS?Tell your healthcare provider about all of your medical conditions, including if you:

Tell your healthcare provider about all the medicines you take,including prescription and over-the-counter medicines, vitamins, and herbal supplements.

WHAT ARE THE POSSIBLE SIDE EFFECTS OF SIKLOS?

SIKLOS may cause serious side effects, including:

See "What is the most important information I should know about SIKLOS?"

The most common side effects of SIKLOS include:

These are not all the possible side effects of SIKLOS.

You are encouraged to report negative side effects of prescription drugs to the FDA at http://www.fda.gov/medwatch, or 1-800-FDA-1088.

Please read the Full Prescribing Information, including Boxed Warning, Medication Guide and Instructions for Use, at http://www.SIKLOSusa.com.

For more information about SIKLOS, we invite you to contact our Medical Information Service at 1 844-884-5520 or https://www.medunikusa.com/en/medical-information-service.

About Medunik USABased in Pennsylvania, Medunik USA works to improve the health and quality of life of Americans living with rare diseases by making orphan drug therapies available in the United States. With strategic partnerships at the global level, the company has critical experience in approval and market access processes as well as the marketing of orphan drug therapies. Medunik USA makes critical medications to treat rare diseases available to American patients who might not otherwise have access to these medications. For more information, visit: http://www.medunikusa.com.

References1. Siklos (hydroxyurea) tablets, for oral use [Prescribing Information]. Addmedica, May 20182. Optimizing hydroxyurea therapy for sickle cell anemia, Ware et al. Hematology Am Soc Hematol Educ Program. 2015;2015:436-432.3. Hydroxyurea for the Treatment of Sickle Cell Disease: Efficacy, Barriers, Toxicity, and Management in Children, Strouse et al. Pediatric Blood Cancer. 2012 August; 59(2): 365371.4. Parental and Other Factors Associated with Hydroxyurea Use for Pediatric Sickle Cell Disease, Oyeku et al. Pediatr Blood Cancer. 2013 April ; 60(4): 653658.

SOURCE Medunik USA

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Siklos, the first and only hydroxyurea-based treatment for pediatric patients with sickle cell anemia, now available in 100 mg scored tablets to help...

When first- and second-line treatments have been exhausted, few options remain for patients with advanced head and neck cancer.

A new phase 2 clinical trial by researchers at the University of Michigan Rogel Cancer Center found the drug axitinib was able to extend the lives of these patients by several months, and also identified a subset of patients with a specific mutation for whom the drug is likely to work best.

Survival increased from less than 6 months with the current standard treatments to nearly 10 months in the 28 patients enrolled in the trial, the research team reported in the journal Cancer. Additionally, 75% of patients with alterations in the PI3K signaling pathway, which is involved in cell cycle regulation, had a good response to the therapy, versus 17% of those without the alterations.

These are patients with metastatic cancer for whom there are no good options outside of clinical trials, says study first author Paul Swiecicki, M.D., an oncologist at Michigan Medicine, U-Ms academic medical center. And its a very timely study because tyrosine kinase inhibitors like axitinib, which target tumors blood supply, have shown considerable synergy when combined with immunotherapy.

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One of the stumbling blocks for this type of combination therapy has been the significant side effects that occur when the two approaches are used at the same time, he says. The current study, however, showed that axitinib may actually prime the body in a way that makes subsequent immunotherapy more effective.

Although the number of patients was small, our study was able to look at what happened to those who received immunotherapy after axitinib and found these patients cancers responded extremely well similar to what weve seen from other studies where people received the treatments simultaneously, Swiecicki says. This supports the idea that we may be able to combine the two approaches in a new way by giving them sequentially rather than at the same time, which should cut down on the severity of the side effects.

Axitinib is currently approved for the treatment of renal cell carcinoma.

Based on the mechanism of action of the drug, and what we know about how head and neck cancer grows, we were optimistic it could make a difference for head and neck cancer patients, Swiecicki says.

The study is innovative in another way: it applies new criteria for measuring the effectiveness of axitinib.

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Axitinib Improves Survival for Patients with Incurable Head and Neck Cancer - Michigan Medicine

Paris, October 20, 2020, 9.15pm

AB Science announces positive top-line Phase 3 resultsfor oral masitinib in severe asthma

This is the second Phase 3 study to demonstrate efficacy for masitinib in severe asthma

AB Science SA (Euronext - FR0010557264 - AB) today announced that the Phase 3 study (AB14001) evaluating oral masitinib in severe asthma uncontrolled by high-dose inhaled corticosteroids (ICS) and with eosinophil level >150 cells/L met its primary endpoint.

The pre-specified primary analysis was rate of severe asthma exacerbations, with masitinib demonstrating a statistically significant 29% reduction in severe exacerbations relative to placebo (p=0.022). The frequency of severe asthma exacerbations was 0.43 in the masitinib arm, versus 0.62 in the placebo arm. Duration of exposure was well-balanced between the treatment-arms (16 months in the masitinib arm and 17 months in the placebo arm). Sensitivity analysis based on the rate of moderate and severe asthma exacerbations was consistent with the primary analysis and detected a statistically significant 31% reduction in exacerbations (p=0.005) between masitinib and placebo. The frequency of moderate and severe asthma exacerbations was 0.55 in the masitinib arm, versus 0.80 in the placebo arm.

This is the second time that masitinib has demonstrated efficacy in reducing severe asthma exacerbations in patients with severe asthma. The treatment effect observed in study AB14001 is comparable with the effect previously reported for study AB07015. In that first phase 3 study, which evaluated masitinib in severe asthma uncontrolled by oral corticosteroids (OCS), masitinib significantly (p=0.010) reduced the rate of severe asthma exacerbations by 35% as compared with placebo. The frequency of severe asthma exacerbations in study AB07015 was 0.34 in the masitinib arm, versus 0.45 in the placebo arm. Duration of exposure was also well-balanced between the treatment arms (13 months in both treatment arms).Safety was consistent with the known tolerability profile for masitinib.

Detailed results will be presented at an upcoming medical meeting.

The study AB14001 enrolled patients with blood eosinophil level >150 cells/L, which differs from the population usually addressed by biological treatments, targeting patients with high eosinophils (>300 cells/L or above) defined as Th2-high eosinophilic asthma.

Masitinib is a first in class drug in severe asthma, distinct from biological treatments targeting type-2 high eosinophilic phenotypes of asthma. Masitinib has a dual mechanism of action, targeting mast cells and PDGFR signaling that are both involved in airway remodeling associated with severe asthma. It has also been shown that increased mast cell activity is associated with both eosinophilic (Th2-high) and non-eosinophilic (Th2-low) asthma phenotypes. Furthermore, masitinib is orally administered, whereas biologics are sub-cutaneous, which is an advantage because oral administration is less of a burden for patients and facilitates compliance for long-term use.

There is still a need for effective therapy of patients with severe asthma. Biologics are established in first line treatment in severe asthma patients with blood eosinophil levels of 300 cells/L. However, these therapies have limited efficacy in reducing severe asthma exacerbations for severe asthmatics with blood eosinophil levels of <300 cells/L. In addition, an estimated 33% to 60% of severe eosinophilic (Th2-high) asthma patients have sub-optimal response or are in failure to type 2 targeted therapeutics.

Asthma uncontrolled by high dose inhaled corticosteroid is estimated at 1,500,000 people1,2 in the USA and in the EU. Among these patients, it is estimated that 75% (i.e. 1,125,000) have blood eosinophil levels of 150 cells/L.

We are very pleased that this study demonstrated efficacy of masitinib in severe asthma uncontrolled by high dose inhaled corticosteroids. After the first positive results of masitinib in severe asthma uncontrolled by oral corticosteroids, this study confirms the efficacy of masitinib in severe asthma population. Taken together, we now have two pieces of evidence that masitinib is effective in severe asthma with an eosinophil level above 150 cells/L, which represents a broader population that the one usually addressed by biologic therapies. These two results seem sufficiently robust to claim that masitinib is a serious candidate as a new oral treatment option for severe asthma, said Lavinia Davidescu, MD, PhD, principal coordinating investigator of the study.

This is indeed the second positive large-scale study with masitinib in patients with severe asthma not restricted to Th2-high asthma phenotypes, which represents an unmet medical need population. In addition, it is important to highlight that masitinib offers a totally new mechanism of action as compared with available treatment options in asthma, said Pascal Chanez, Professor of Respiratory Diseases at Aix-Marseille University, France.

Intellectual Property for masitinib is secured in severe asthma until 2032. The U.S. Patent and Trademark Office has granted a patent (13/983626) relating to methods of treating severe persistent asthma with masitinib. This patent, protects the use of masitinib in the treatment of severe persistent corticosteroid-dependent asthma and severe persistent corticosteroid-resistant asthma.

Phase 3 studies in asthma

Study AB14001 was a prospective, multicenter, randomized, double-blind, placebo-controlled, 2-parallel groups, phase 3 study evaluating the efficacy and safety of masitinib in asthma uncontrolled by high-dose inhaled corticosteroids and with eosinophil level (>150 cells/L.

Eligible patients were patients with eosinophil level related to asthma at baseline 0.15 K/uL and with a physician diagnosis of persistent asthma for at least 12 months based on GINA 2009 Guidelines whose asthma is partially controlled or uncontrolled on ICS/LABA combination therapy based on the following criteria:

Participants received masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration was subjected to a safety control.

The primary endpoint of this study was the annualized severe asthma exacerbation rate for the overall time on treatment, as for study AB07015 in severe asthma uncontrolled with OCS.

References1. Respir Med. 2006 Jul;100(7):1139-51. Epub 2006 May 18. Prevalence ranges from 7% (France, Germany) to 11% (USA) and 18% (UK). Average 10%. Rising incidence2. J Investig Allergol Clin Immunol 2012; Vol. 22(7): 460-475 20% of asthma patients have asthma requiring high dose inhaled or oral corticosteroids 20% of these asthma patients are uncontrolled. Only 55% of patients initially suspected of having asthma uncontrolled by high dose ICS or OCS receive a confirmed diagnosis

About masitinibMasitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglia and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases.

About AB ScienceFounded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment.

AB Science has developed a proprietary portfolio of molecules and the Companys lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, and inflammatory diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Sciences website: http://www.ab-science.com.

Forward-looking Statements - AB ScienceThis press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance.

These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents filed by AB Science with the Autorit des Marchs Financiers (AMF), including those listed in the Chapter 4 "Risk Factors" of AB Science reference document filed with the AMF on November 22, 2016, under the number R. 16-078. AB Science disclaims any obligationor undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations.

For additional information, please contact:

AB ScienceFinancial Communication & Media Relations investors@ab-science.com

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AB Science announces positive top-line Phase 3 results for oral masitinib in severe asthma - GlobeNewswire

An analysis report published by IndustryGrowthInsights (IGI) is an in-depth study and detailed information regarding the market size, market performance and market dynamics of the Regenerative Medicine. The report offers a robust assessment of the Global Regenerative Medicine Market to understand the current trend of the market and deduces the expected market trend for the Regenerative Medicine market for the forecast period. Providing a concrete assessment of the potential impact of the ongoing COVID-19 in the next coming years, the report covers key strategies and plans prepared by the major players to ensure their presence intact in the global competition. With the availability of this comprehensive report, the clients can easily make an informed decision about their business investments in the market.

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DermatologyCardiovascularCNSOrthopedicOthers

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Cell TherapyTissue EngineeringBiomaterialOtherRegenerative Medicin

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Executive Summary

Assumptions and Acronyms Used

Research Methodology

Regenerative Medicine Market Overview

Regenerative Medicine Supply Chain Analysis

Regenerative Medicine Pricing Analysis

Global Regenerative Medicine Market Analysis and Forecast by Type

Global Regenerative Medicine Market Analysis and Forecast by Application

Global Regenerative Medicine Market Analysis and Forecast by Sales Channel

Global Regenerative Medicine Market Analysis and Forecast by Region

North America Regenerative Medicine Market Analysis and Forecast

Latin America Regenerative Medicine Market Analysis and Forecast

Europe Regenerative Medicine Market Analysis and Forecast

Asia Pacific Regenerative Medicine Market Analysis and Forecast

Middle East & Africa Regenerative Medicine Market Analysis and Forecast

Competition Landscape

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2027 Projections: Regenerative Medicine Market Report By Type, Application And Regional Outlook - The Think Curiouser

Immunology through a human lens

The coronavirus disease 2019 (COVID-19) pandemic has underscored the critical need to better understand the human immune system and how to unleash its power to develop vaccines and therapeutics. Much of our knowledge of the immune system has accrued from studies in mice, yet vaccines and drugs that work effectively in mice do not always translate into humans. Pulendran and Davis review recent technological advances that have facilitated the study of the immune system in humans. They discuss new insights and how these can affect the development of drugs and vaccines in the modern era.

Science, this issue p. eaay4014

The mammalian immune system is a remarkable sensory system for the detection and neutralization of pathogens. History is replete with the devastating effects of plagues, and the coronavirus disease 2019 (COVID-19) pandemic is a defining global health crisis of our time. Although the development of effective vaccines has saved many lives, the basic workings of the immune system are complex and require the development of animal models, such as inbred mice. Indeed, research in mice has been enormously productive, and the tremendous insights gleaned have resulted in many Nobel prizes and other accolades. However, past results are not necessarily a reliable guide to the future, and a notable limitation of animal models has been their failure to accurately model some human diseases and their inability to predict human immune responses in many cases. With regard to inbred mice, which have been the principal model of choice for immunology, this is likely due to the compromises that were necessary to create a more tractable and reproducible system for experimentation, such as genetic uniformity and lack of pathogen exposure, as well as the fact that mice are evolutionarily quite distinct. These considerations suggest that direct studies of the human immune system are likely to be extremely rewarding, both from a scientific and a medical perspective.

In the past decade there has been an explosion of new approaches and technologies to explore the human immune system with unprecedented precision. Insights into the human immune response to vaccination, cancers, and viral infections such as COVID-19 have come from high-throughput omics technologies that measure the behavior of genes, mRNA (single-cell transcriptomics), proteins (proteomics), metabolites (metabolomics), cells (mass cytometry), and epigenetic modifications (ATAC-seq), coupled with computational approaches.

Sydney Brenner remarked in 2008, We dont have to look for a model organism anymore. Because we are the model organisms. We propose that studying the immune system in humans, who are genetically diverse and afflicted by a multitude of diseases, offers both a direct link to medicine (i.e., translation) and the very real prospect of discovering fundamentally new human biology. New approaches and technology are now making this area much more approachable, but profiling immunity in humans is but the first step. Computational mining of the data and biological validation in animal models or human organoids are essential next steps, in an iterative cycle that seeks to bridge fundamental and applied science, as well as mouse and human immunology, in a seamless continuum of scientific discovery and translational medicine. This will represent a new paradigm for accelerating the development of vaccines and therapeutics.

Systems biology techniques can be used to probe the human immune response to viral infections and can define molecular signatures that predict disease severity and illuminate the underlying mechanisms of disease.

Although the development of effective vaccines has saved countless lives from infectious diseases, the basic workings of the human immune system are complex and have required the development of animal models, such as inbred mice, to define mechanisms of immunity. More recently, new strategies and technologies have been developed to directly explore the human immune system with unprecedented precision. We discuss how these approaches are advancing our mechanistic understanding of human immunology and are facilitating the development of vaccines and therapeutics for infection, autoimmune diseases, and cancer.

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The science and medicine of human immunology - Science Magazine

Scientists have mapped and described the function of cells in six regions of the adult heart, providing a new foundation for studying heart disease.

Ka-thump. Ka-thump. Ka-thump. Though we barely notice it most of the time, the steady beating of a human heart is an amazingly complex performance. Like an orchestra, thousands of cells have to master their individual performances as well as work together.

Now a team of scientists has created the first atlas of human heart cells, a collection of maps showing nearly half a million heart cells and identifying the role of each in the hearts symphony. The researchers examined six regions in 14 healthy donor hearts, creating a detailed database that provides a new basis of comparison for studying heart disease, the leading cause of death worldwide.

To understand whats going wrong in various forms of heart disease, first we need to know what is normal, says Howard Hughes Medical Institute Investigator Christine Seidman, a cardiovascular geneticist at Harvard University and director of the Cardiovascular Genetics Center at Brigham and Womens Hospital. Seidman and colleagues describe the new heart atlas September 24, 2020, in the journal Nature.

I can summarize my thoughts in one word: monumental, says cardiologist Douglas Mann of Washington University School of Medicine in St. Louis, who was not involved in the study. I think its a really big accomplishment and will be a tremendous source of reference for the field.

Heart cells have proven particularly difficult to study. Unlike some cancer cells and other tissues, there are no heart cells that can be grown indefinitely in the laboratory and studied. Instead, much cardiac research is done using mice, whose hearts have important differences from human hearts.

And healthy human hearts can be hard to find (most are used in transplants). Seidmans team relied on those unusual instances in which healthy hearts were rejected for transplantation and could be frozen for use in research. First, the researchers used a high-throughput sequencing method to define individual characteristics of every heart cell. They then mapped those cells in six regions of 14 human hearts, seven from men and seven from women. For the first time, we have a zip code for each cell to know what population it belongs to, Seidman says.

The team also analyzed heart cells RNA levels using fluorescent markers to glean molecular details of their function. Identifying not only where cells are, but which proteins theyre producing, will be a particular boon for research, Mann says. For instance, by comparing cells in diseased hearts to those in healthy hearts using the atlas, researchers might pinpoint differences and target new therapies for heart disease.

For the first time, we have a zip code for each cell to know what population it belongs to.

Christine Seidman, HHMI Investigator at Brigham and Women's Hospital

Though the researchers studied a relatively small group of hearts (fourteen people cannot replicate the worlds population, Seidman says), the new atlas revealed some biological surprises. The team found previously unknown cell diversity in various parts of the heart. They also uncovered differences between the healthy hearts of males and females; females had a greater proportion of heart muscle cells, called cardiomyocytes, than males. That warrants more research, Seidman says, as those cells might hold clues to differences in heart disease between the sexes.

Still, what we see is striking heterogeneity in terms of the diverse cell types that we now know make up the tissue of the human heart, and in terms of the regional differences within the heart, says cardiologist Hugh Watkins of Oxford University in England, who was not part of the study team. Its certainly a much more complicated organ than many might have imagined!

The atlas is part of the Human Cell Atlas initiative, an effort funded by the Chan Zuckerberg Initiative to map all the cell types in the human body. It takes a big village to do this, Seidman says. Her group worked with an international team of experts on everything from heart surgery to computational biology in order to create the database atlas. All of the data are available at http://www.heartcellatlas.org.

Next, Seidman and her colleagues hope to expand the atlas to a more diverse population (the initial hearts were all from white donors). They are also beginning to compare the proteins made in healthy heart cells to those affected by heart disease.

In due course, what we really want to know is how the different cell types fit together at the microscopic and functional level, Watkins says. Thats another ambitious goal, but the atlas provided here is an exciting start.

###

Citation

Monika Litviukov et al. Cells of the adult human heart. Nature. Published online September 24, 2020. doi: 10.1038/s41586-020-2797-4

Link:
Mapping the Human Heart, Cell by Cell - Howard Hughes Medical Institute

As Americans begin pulling up their sleeves for an annual flu vaccine, researchers at the University of WisconsinMadison have provided new insights into an alternative vaccine approach that provides broader protection against seasonal influenza.

In a study published in Cell Reports Medicine today (Sept. 22), scientists describe a T-cell-based vaccine strategy that is effective against multiple strains of influenza virus. The experimental vaccine, administered through the nose, delivered long-lasting, multi-pronged protection in the lungs of mice by rallying T-cells, specialist white blood cells that quickly eliminate viral invaders through an immune response.

This three-dimensional, semi-transparent rendering of a whole influenza virus shows both the clover-like surface proteins on the outside of the virus, as well as the internal ribonucleoproteins on the inside. Existing influenza vaccines introduce proteins found on the surface of flu viruses to help induce immune protection. A new study by researchers at the UW School of Veterinary Medicine uses an internal nucleoprotein to stimulate the immune system in an effort to create a universal flu vaccine. Centers for Disease Control and Prevention

The research suggests a potential strategy for developing a universal flu vaccine, so you dont have to make a new vaccine every year, explains Marulasiddappa Suresh, a professor of immunology in the School of Veterinary Medicine who led the research. The findings also aid understanding of how to induce and maintain T-cell immunity in the respiratory tract, a knowledge gap that has constrained the development of immunization strategies. The researchers believe the same approach can be applied to several other respiratory pathogens, including the novel coronavirus that causes COVID-19.

We dont currently have any vaccine for humans on the market that can be given into the mucosa and stimulate T-cell immunity like this, says Suresh, a veterinarian with specialty training in studying T-cell responses to viral infections.

The strategy addresses the Achilles heel of flu vaccines, which is to achieve specific antibody responses to different circulating influenza strains annually, by harnessing T-cell immunity against multiple strains. In particular, the new approach calls into action tissue-resident memory T-cells, or TRM cells, which reside in the airways and lining of lung epithelial cells and combat invading pathogens. Like elite soldiers, TRM cells serve as front line defense against infection.

Marulasiddappa Suresh

We didnt previously know how to elicit these tissue-resident memory cells with a safe protein vaccine, but we now have a strategy to stimulate them in the lungs that will protect against influenza, explains Suresh. As soon as a cell gets infected, these memory cells will kill the infected cells and the infection will be stopped in its tracks before it goes further.

Flu vaccines work by arming the immune system with an enhanced ability to recognize and fight off the flu virus. Vaccines introduce proteins found on the surface of flu viruses, prompting the immune system to produce antibodies that are primed to react should the virus attack.

However, because strains must be predicted ahead of flu season in order to produce vaccines, the vaccine in any given year may not completely match the viral strains in circulation that season. Flu viruses frequently mutate and can differ across time and from region to region. In addition, protection is neither long-lasting nor universal.

Even though current vaccines that people get annually stimulate antibody responses, these antibodies dont cross-protect, notes Suresh. If there is a new flu strain not found in that years vaccine, the antibodies that we generated last year wont be able to protect. Thats when pandemics happen because there is a completely new strain for which we have no antibodies. That is a really big problem in the field.

The vaccine developed by Suresh and his team is directed against an internal protein of influenza specifically, nucleoprotein. This protein is conserved between flu strains, meaning its genetic sequences are similar across different strains of flu.

The vaccine also utilizes a special combination of ingredients, or adjuvants, that enhance an immune response, which the researchers developed to stimulate protective T-cells in the lungs. These adjuvants spur T-cells to form into different subtypes in the case of the experimental flu vaccine, memory helper T-cells and killer T-cells. By doing so, the vaccine leverages multiple modes of immunity.

Killer T-cells hunt down and kill influenza virus-infected cells. Helper T-cells assist killer T-cells and produce molecules to promote influenza control. In laboratory studies, the team found that both T-cell types were needed to protect against flu.

Researchers demonstrated in a mouse model of influenza that the vaccine provides long-lasting immunity at least 400 days after vaccination against multiple flu strains. They will next test the vaccine in ferrets and nonhuman primates, two animal models of influenza research more biologically similar to human infection and transmission.

The vaccines combination of adjuvants makes it adaptable to other pathogens and expands the toolbox for vaccine research, notes Suresh. He and his team have devised ways to program immunity to target multiple respiratory viruses. They are currently testing the same vaccine strategy against tuberculosis, which infects more than 10 million people globally each year, and human respiratory syncytial virus, or RSV, a major cause of lower respiratory tract infections during infancy and childhood.

The researchers believe the same vaccine technology can applied against SARS-CoV-2, the coronavirus that causes COVID-19. Based on the COVID-19 immunology, we know this vaccine strategy would most likely work, says Suresh.

The team is now developing an experimental vaccine against COVID-19 and conducting laboratory tests to measure its effectiveness in mice and hamsters, animal models for COVID-19. Initial unpublished studies in mice show that the vaccine stimulates strong T-cell immunity against COVID-19 in the lungs.

Along with its adaptability, this vaccine approach may harbor important safety benefits. Typically, long-lasting T-cell immune responses are stimulated by live vaccines. For instance, the measles, mumps and chickenpox vaccines administered worldwide are live, replicating vaccines essentially benign versions of the pathogenic organism. These live vaccines stimulate strong, almost lifelong immunity. However, they cant typically be given to pregnant or immunocompromised individuals due to health risks.

In the case of the UWMadison teams vaccine, because it is a protein vaccine and not a live vaccine, it should be safe for delivery to those who are pregnant or immunocompromised an advantage in delivering protection to a wider patient population. Suresh says that in recent years, vaccine development efforts have shifted away from live vaccines toward protein vaccines because an increasing number of people are living with compromised immune systems due to chemotherapy, radiation treatments or conditions such as HIV/AIDS.

Previously, we didnt know how to induce T-cell immunity in the lung without live viruses, says Suresh. If we cleverly use a combination adjuvant, which we have developed, you can induce T-cell immunity that should stay in the lungs and protect longer.

THIS WORK WAS supported by THE NATIONAL INSTITUTES of HEALTH (GRANT UO1124299).

Originally posted here:
New vaccine strategy harnesses 'foot soldier' T-cells to provide protection against influenza - University of Wisconsin-Madison

By: Explained Desk | New Delhi | Updated: September 27, 2020 7:47:46 amThis electron microscope image shows novel coronavirus SARS-CoV-2 virus particles, orange, isolated from a patient. (NIAID/National Institutes of Health via AP)

Statins are a widely used medication for lowering cholesterol. Recent research from US San Diego (University of California at San Diego) School of Medicine associated statins with reduced risk of developing severe Covid-19 disease, as well as faster recovery times.

Now another research team, also from UC San Diego School of Medicine, has explained why this happens. The first study is published in The EMBO Journal, and the new one in the American Journal of Cardiology. In short, statins remove cholesterol from cell membranes, which in turn prevents the coronavirus from getting in.

We know that SARS-CoV-2, the virus that causes Covid-19,enters the human cell by using a protein known as ACE2 on the cell surface. ACE2 can be affected by prescription statins.

Researchers retrospectively analysed the electronic medical records of 170 patients with Covid-19 and 5,281 Covid-negative control patients hospitalised at UC San Diego Health between February and June 2020. Among the patients with Covid-19, 27 per cent were taking statins on admission.

Statin use was associated with a more than 50 per cent lower risk of developing severe Covid-19. Patients taking statins also recovered faster than those not taking statins.

Source: UC San Diego Heath

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New research finds statins associated with severity of Covid-19 disease - The Indian Express

Cell Reports Medicine is a premium open-access journal from Cell Press, publishing cutting-edge research in translational and clinical biomedical sciences that inform and influence human health and medicine. The content published in Cell Reports Medicine reaches a broad range of scientists and clinicians...

Cell Reports Medicine is a premium open-access journal from Cell Press, publishing cutting-edge research in translational and clinical biomedical sciences that inform and influence human health and medicine. The content published in Cell Reports Medicine reaches a broad range of scientists and clinicians across the spectrum of medical disciplines, ensuring that your work will be both visible and accessible. The journal publishes original research that ranges from exciting concepts in human biology, health, and disease to all phases of clinical work. We encourage submissions featuring innovative ideas that open up new directions in clinical research and practice, as well as studies that provide critical information that enriches our understanding of current standards of care in diagnosis, treatment, and prognosis. This includes, but is not limited to, translational studies, clinical trials in all areas of medicine including long-term trial follow-ups as well as work in genomics, biomarker discovery, and developments in technology that contribute to diagnostics, treatment, and healthcare. Studies based on vertebrate model organisms also fall within the scope of the journal, provided that the results are directly relevant to human health and disease.

We will consider impactful, single-point papers called Reports, in addition to longer Articles. Large datasets, techniques, and tools for the community can also be considered under the Article format. We will also publish Reviews covering recent literature in emerging and active fields. The professional in-house scientific editors at Cell Reports Medicine work closely with authors, reviewers, and the journal's editorial board to ensure exemplary editorial process, fair and robust peer review, and rapid dissemination and communication of your research.

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Cell Reports Medicine - Journal - Elsevier

Joanna had just turned 62when she noticed that she couldnt stand very long before her right leg wouldhurt. She thought it was from an old injury, when her dog had slammed into herthigh. When the ache moved to her wrist, she went to a doctor who said shemight be getting arthritis.

Thepain quickly intensified. It just happened so rapidly, and I couldnt figureout why, says Joanna, who lives in a Houston suburb. Her doctors chalked it upto wear and tear. Youre getting older, she remembers them telling her.This was in early 2018.

Then she got an e-mail with a link to a video about stem cells and the conditions they could cure, including arthritis. I started watching it and then I just turned it off for a while because I thought, Im afraid Im going to get my hopes up too high, says Joanna, who asked that her last name not be used to protect her medical privacy.

Shestarted seeing full-page ads for stem cell seminars in the newspaper. Sheattended one at a local hotel, and the presenter announced that thousands ofpatients had benefited from stem cell injections. It was natural, the womansaid. No one had ever been harmed. The idea that the treatment wasnt a drugreassured Joanna.

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Shemade an appointment for the next day. It sounded too good to be true, but Iwas desperate, she says. She received injections into her back, neck andshoulder of stem cells from donated umbilical cord blood followed by an IV ofthe product the next day. The cost was $30,000, siphoned from her husbandspension. She knew she was taking a risk, but she felt hopeful.

Twodays later, her face began to burn and itch. Then her feet. She had pain inplaces that had never hurt before, like the joints of her fingers. Her hairstarted falling out, and she descended into a deep depression. Im totallymiserable, she says, months later. Im just agonizing in pain. Now I dontsee any hope.

Stem cells sold at clinics are driving whats thought to be a $2 billion global industry. Facebook pages announce seminars. Local newspapers are wrapped in ads vowing relief without surgery. Stem cells are billed as treatments for everything from autism to multiple sclerosis to baldness. Most commonly, the ads focus on orthopedic issues, especially aching knees.

Animportant point gets left out of the cheery ads: Theres not enough science tojustify using stem cells for any of the advertised conditions, including jointpain. None of the treatments advertised have been approved by the U.S. Food andDrug Administration. (The only approved stem cell treatments are for certaincancers and blood disorders.) Very few of the orthopedic studies in humans havebeen scientifically rigorous, and none have shown stem cells regrowingcartilage.

Itsnot even clear whether treatments being touted as stem cells contain viablestem cells or whether the contents should be defined as stem cells at all. Asthe stem cell industry grows rapidly, many researchers who are studying stemcells for their potential to regenerate tissues worry that the boomingmarketplace, which conflates hype with reality, might ultimately damageresearch progress.

The scientific and medical community ishaving to play defense, says Shane Shapiro of the Mayo Clinic Jacksonville inFlorida, who has conducted one of the very few published trials that comparedstem cells with a placebo for people with osteoarthritis. Misinformation andmisunderstanding about how cells are used to treat disease is proliferating.

The explosion of advertising reflects a dramatic turnaround from the controversy over stem cells that occupied the public in the early 2000s. At the time, scientists had learned how to generate lines of cells from embryos that were left over from in vitro fertilization and donated for research. In theory, the embryonic cells had the potential to treat disease by becoming a slew of different tissues, but their use became entangled with the politics of abortion. Then in 2001, President George W. Bush banned federal funding for research on embryonic stem cell lines not already in labs. Embryonic stem cell research has ridden the political tides since then: Restrictions were eased under Barack Obamas presidency, then Donald Trumps administration added restrictions on fetal tissue use.

Scientists tried to persuade the public to support the research by focusing on the great promise. They argued that stem cells might one day cure diseases by naturally repairing lost or damaged tissue. Actor Michael J. Fox, the most famous Parkinsons patient of his generation, testified to Congress in 1999 that stem cells could one day cure degenerative brain diseases. Joanna remembers Foxs passion. Thats what kind of made us aware of what stem cells were at the time, she says.

Broadly speaking, stem cells are cells capable of renewing themselves and taking on the identity of the tissue around them (SN: 3/19/16, p. 22). The early controversy about using embryonic cells has largely quieted down. The stem cells being marketed today are not embryonic; they come from bone marrow, fat tissue or birth products such as umbilical cord blood or amniotic fluid, all advertised as being able to regenerate cartilage. Clinic websites usually feature earnest testimonials with no hint of any possible negative side effects.

For the first part of the 2000s, stem cell treatments were largely sought through medical tourism. U.S. patients would travel to other countries for experimental treatments to cure diseases such as multiple sclerosis or spinal cord injury.

BioethicistLeigh Turner of the University of Minnesota in Minneapolis noticed a shiftaround 2012. One of the businesses that was part of that marketplace, acompany in South Korea, ended up popping up in the United States, he says. Thecompany, operating under the name Celltex, offered to remove stem cells from apatients own fat tissue, grow the cells in a lab and then reinject them.

Whenthe FDA sent a warning letter to the company in September 2012 stating that itsproducts must be approved before use in patients, the company moved itstreatments to Mexico. Regulations there are less stringent, though the businessremains headquartered in Houston.

Today, many clinics have learned how to operate just inside the margins of federal regulations, or simply ignore them. Turner and Paul Knoepfler of the University of California, Davis mapped the rise in U.S. stem cell clinics that market unapproved therapies, reporting in 2016 in Cell Stem Cell. The first few clinics emerged about 10 years ago, Turner says. By 2014, 2015, companies are pouring into the marketplace at a very rapid rate.

Knoepfler estimates that today more than 1,000 clinics across the country offer stem cells, though there are probably more because many doctors and chiropractors have simply added stem cells as a sideline to their main services. For some, stem cells are lucrative enough to support a business on its own. One in 4 stem cell providers in the Southwest offers the treatment exclusively, researchers at Arizona State University reported in August in Stem Cell Reports.

Thebusiness is extremely profitable, and the treatments are rarely covered byinsurance. Patients pay cash sometimes draining their life savings, takingout loans or drawing down retirement funds like Joanna did.

Often,when you go to business websites, theres not this kind of sober, frank,judicious accounting of risks and benefits or the possibility that theremight be no benefit, that someone could be harmed, Turner says. The websitestend to frame risks and benefits in a very misleading kind of way.

Andbecause each year tens of thousands of U.S. patients by Knoepflers estimate are getting cells in clinics outside of clinical trials, its difficult to knowexactly what the risks of the direct-to-consumer marketplace are. No one iskeeping track.

In 2018, researchers writing in Stem Cells Translational Medicine resorted to searching Google and the scientific literature, where they found 35 reports of serious consequences. Some patients in Florida lost their sight after getting stem cell injections into their eyes. In December, the FDA warned of serious illnesses in Nebraska linked to treatments with exosomes, products taken from placentas that are offered by some stem cell clinics.

A recent survey of neurologists, presented in March 2019 in Dallas at a meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis, asked doctors how their patients had fared after receiving unapproved stem cell treatments. About 25 percent of the 204 neurologists who responded said patients had suffered serious consequences such as strokes and seizures. Three doctors reported that patients had died. Without study, its impossible to know why.

It makes sense that treatments for knee pain appear to dominate the industry the potential customer base is large and growing. More than 600,000 people in the United States had knee replacement operations in 2014, according to data released in 2018 by the American Academy of Orthopaedic Surgeons. That number is expected to rise as baby boomers age and obesity rates climb. As the body ages, shock-absorbing cartilage in the joints wears away, which can lead to painful bone-on-bone rubbing. Stem cells are advertised as an easy way to avoid surgery.

Sofar, though, its not clear thats true. Recently, Maarten Moen, a sports medicinephysician at Bergman Clinics in Naarden, Netherlands, and his colleaguesreviewed every clinical trial he could find that examined using stem cells forknee osteoarthritis. Stem cells are prohibited for use in Holland, says Moen,a member of the medical staff of the Dutch Olympic Committee. Thats why wewere conducting this: to see if we could convince people in our country topossibly start using this therapy. But only if we answer these two questions:Is it helpful? And also, is it safe?

The groups results appeared in 2017 in the British Journal of Sports Medicine. The team found only six human studies testing stem cells for knees, and none were large trials that included a placebo comparison. While the treatment appeared safe, effectiveness couldnt be determined. Every study had methodological problems. As a result, the authors didnt recommend stem cells for knee osteoarthritis. Moen has recently updated his review, but those results have not yet been published. He offers a preview: The evidence didnt get any stronger. He found only two clinical reports that had compared the treatment with a placebo.

Bothwere from Shapiro, of the Mayo Clinic. Its been nearly three years since wefirst published our early results, Shapiro says. With his trial as the firstpiece of the puzzle, he expected that, like anything else in science, wewould be followed by a bunch of other trials. So far, other published resultshave not been pouring in.

Shapiroand colleagues from the Mayo Clinic and Yale University School of Medicine hadtested 25 patients with mild knee osteoarthritis in both legs. The researcherstook about 50 milliliters of cells from each patients bone marrow,concentrated the cells in the laboratory and then injected them back into oneknee of each patient. The other knee got an injection of saline as a placebo.The patients did not know which knee got the experimental treatment.

Both the stem cell knee and the placebo knee improved by about an equal degree about 50 to 75 percent on a pain scale, the team reported in 2017 in the American Journal of Sports Medicine. We werent able to conclude that the stem cell product was any superior in pain relief to anything else, he says. Additionally, we did not see any cartilage regrowth. A 12-month follow-up published in October in Cartilage found similar results.

The fact that the saline alone helped patients feel better was not surprising. A meta-analysis published in 2017 in the American Journal of Sports Medicine examined whether simple saline injections helped knee pain. That review, by researchers at Rush University Medical Center in Chicago and the University of Toronto, found that saltwater alone provided relief on the level of some drugs. But why the knees that got the bone marrow cells in Shapiros study improved to an equal degree is still unclear, he says. So hes not yet ready to say the stem cells dont work.

WhatI think actually happened is that we injected a therapeutic substance in one oftheir knees, and we injected a nonharmful substance, which is the saline, inthe other knee, and the patients were able to get back to their life for aperiod of time that overall made them feel better, he says.

The Mayo study tested stem cells from the patients own bone marrow. But clinics also offer cells from the bodys fat tissue, extracted by liposuction. Doctors can use enzymes to strip away extraneous cells from the fat, leaving only the regenerative cells. But that therapy is also unproven. One study, conducted by researchers in Australia and published in February 2019 in Regenerative Medicine, involved 30 patients with knee osteoarthritis. Patients who received stem cells from fat reported a 69 percent improvement in their pain, compared with no change in a comparison group that did not receive the treatment. But that study offered no placebo injection for comparison.

A second study, by a South Korean team, had a placebo, but a small number of patients. Twelve patients who received stem cells from their own fat had a 55 percent improvement in pain (based on their responses to a questionnaire) compared with no substantial improvement among 12 patients who got a placebo shot, the researchers reported in March 2019 in Stem Cells Translational Medicine. Cartilage didnt regrow with stem cells, but it didnt shrink, which it did in the placebo group.

Butthe treatments in those studies differ from what is actually offered at stemcell clinics. In both of those studies, the researchers expanded orconcentrated the cells in the laboratory before injecting them into patients apractice that is allowed in the United States only in a scientific study. UnderFDA rules, U.S. stem cell centers are only allowed to move a patients owntissue from one place to another, with little manipulation of the cells,otherwise the treatment may be considered an unapproved drug.

Sports medicine physician Kenneth Mautner and colleagues at Emory University in Atlanta compared outcomes for 76 patients with arthritis who received a treatment that was closer to what doctors can do in their offices. Each patient received either cells taken from their own bone marrow or from fat tissue. In both cases, the cells were simply moved to another place within the patients own body. After six months, both groups showed pain reductions and neither treatment was better than the other, the researchers reported in the November 2019 Stem Cells Translational Medicine. There was about 70 to 75 percent improvement for those who actually did improve, Mautner says. About one-quarter of the patients did not get better. Patients with more advanced arthritis were less likely to benefit.

Buthis study had that common shortcoming: no placebo comparison. When yourepaying a lot of money, theres obviously a placebo effect, Mautner says. Itsnot just your mind convincing you that you feel better. The placebo effect canactually be chemicals and cytokines that then produce anti-inflammatory effectsin your joints.

Inaddition to bone marrow and fat tissue, a growing number of clinics areoffering products made from donated umbilical cord blood or other birthproducts, Knoepfler says. Those cells are easy to administer and dont requirethe expertise to extract cells from the body.

Butif there is little evidence for the effectiveness of stem cells from fat andbone marrow, Shapiro says, there is zero support for umbilical products inhuman studies. Im not even studying them yet, he says.

Hardly any evidencesupports the idea that treatments marketed as stem cells can regenerate worntissue, which is what many patients think they are buying. Theres very littleevidence that it will regrow your cartilage, says orthopedic surgeon JasonDragoo of the University of Colorado Denver.

Hisresearch team is conducting a study to see whether there are treatments thatmight increase cartilage thickness. One study pairs the cellular treatment withsurgery. The existing tissue may be more receptive to regrowth, he says, ifyou clear away the debris and all the other things, get it as cleaned up aspossible, then give the cells. Hes also conducting a study comparing theability of cells from fat to repair tiny tears in cartilage that is otherwisemostly healthy, a process he compares with filling potholes.

Buteven if cartilage wont regrow, he and others say, the procedure may stillreduce inflammation, which could quiet a painful knee. Theres also earlyevidence from animal studies that cells from bone marrow or fat might sendchemical signals that jump-start a persons own healing.

Biomaterials scientist Sowmya Viswanathan of the University of Toronto and colleagues reported a study of 12 patients receiving bone marrow cells in August in Stem Cells Translational Medicine. The study had no control group. We saw improvement in symptoms, in pain, in quality of life and in joint stiffness for all the patients. These are the things that the patients care about. The fact that it doesnt regenerate cartilage doesnt disprove its ability to still be a functional, useful cell therapy, she says. It might work, but maybe not in the way that patients expect.

Viswanathan worries thatthe current stem cell market is exploiting the work of scientists, piggybackingoff of the legitimate but early studies for immediate commercial gain, shesays.

Everythinggets called stem cells. Nomenclature is still very important because if youcant name it properly, then you dont even know that youre talking about twodifferent or three or four different things, she adds.

Many clinics call the cells in their products mesenchymal stem cells, a term taken from a 1991 paper by biologist Arnold Caplan of Case Western Reserve University in Cleveland. Yet in 2017 in Stem Cells Translational Medicine, Caplan advocated for a name change: Stem cell misconceptions have led some practitioners in the United States and worldwide to advertise the availability of stem cell treatments (i.e., MSCs can cure the blind, make the lame walk and make old tissue young again).

Viswanathan and other members of the International Society for Cellular Therapy published a position statement in October in Cytotherapy that cells commonly identified as mesenchymal stem cells should more precisely be called mesenchymal stromal cells in the scientific literature to reflect the lack of evidence that, when used as a medical treatment, those cells can renew themselves and form different tissues. (Stromal cells form the bodys connective tissue.) As long as everything is called stem cells, she says, clinics focused on profits will be able to exploit legitimate research for marketing purposes.

Andthere are so many questions left to answer. She worries about what happens whenpeople have bad reactions, like Joanna did. We dont fully understand repeatinjections. We dont know the dosing. If there is an adverse event, then what?she says. Then it sets back the kind of legitimate work thats being donebecause the difference isnt apparent to the funders and to the lay publicbecause everyones calling it exactly the same thing.

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