The Top 5 Most-Read Oncology Stories of 2020 – AJMC.com Managed Markets Network

Our 5 most-read oncology stories and most-watched videos this year highlighted cancer care policy, diversity, and the growing practice of remote cancer care.

Our 5 most-read oncology stories and most-watched videos this year highlighted cancer care policy, diversity, and the growing practice of remote cancer care. It was a dynamic year from a policy perspective, with delays and accommodations from CMS amid a global pandemic. Here are the top stories in oncology this year.

5. Dr Kerin Adelson on Changes in Care Coordination Precipitated by OCM

The Oncology Care Model (OCM) has impacted the way oncologists practice, said Kerin Adelson, MD, associate professor, chief quality officer, and deputy chief medical officer for Smilow Cancer Hospital at Yale New Haven/Yale Cancer Center. With the model focused on reducing unnecessary utilization, Adelson said that the resources it provides have enhanced care coordination and led to the formation of novel infrastructure and clinical pathways.

Watch the video.

4. Dr Karen Winkfield Discusses Importance of Workforce Diversity in Oncology

Disparities in health care have been highlighted more than ever this year, with the pandemic bringing to light and exacerbating longstanding racial and economic divides in those who receive the care they need. Karen Winkfield, MD, PhD, incoming executive director of the Meharry-Vanderbilt Alliance, spotlighted the need for minority representation in the radiation oncology workforce.

Watch the video.

3. How Federal Policy Decisions Are Influencing Cancer Care

The cost of cancer is driven up by novel therapies, with unprecedented progress fostering cancers evolvement into a chronic disease. But federal policy decisions are influencing oncologists ability to provide the best possible therapies to every patient. Debra Patt, MD, PhD, MBA, MPH, of Texas Oncology, addressed the predicament at The American Journal of Managed Cares 2020 Patient-Centered Oncology Care meeting.

Read the full article.

2. Dr Sara Douglas: Distance Caregivers of Patients With Cancer Have Distinct Needs

The high degree of uncertainty associated with providing remote care means that distance caregivers of patients with cancer have unique needs for information and require customized education, explained Sara L. Douglas, PhD, RN, the Gertrude Perkins Oliva Professor in Oncology Nursing and associate dean for research at the Frances Payne Bolton School of Nursing at Case Western Reserve University in Cleveland, Ohio.

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1. Tafasitamab Combination Approved for Adults With R/R DLBCL

Tafasitamab-cxix was approved by the FDA on July 31 this year for use in combination with lenalidomide for second-line treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), and the news took the top spot in our list of most-read oncology stories this year. The approval, which was based on phase 2 results for 80 patients in the L-MIND trial, meets the needs of patients not eligible for an autologous stem cell transplant.

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The Top 5 Most-Read Oncology Stories of 2020 - AJMC.com Managed Markets Network

Adipose Tissue-derived Stem Cell Therapy Market Share Analysis, Application, Strategies of Key Players & Forecast to 2025 – Factory Gate

The Global Adipose Tissue-derived Stem Cell Therapy Market report offers users the detailed study of the market and its main aspects. The study on Global Adipose Tissue-derived Stem Cell Therapy Market, offers profound understandings about the Adipose Tissue-derived Stem Cell Therapy Market covering all the essential aspects of the market. The report provides competitive pipeline landscape of the Global Factors like production, market share, revenue rate, regions and key players define a market study start to end. This report gives an overview of market valued in the year 2019 and its growth in the coming years till 2025.

This study covers following key players: The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report: Breakdown data in in Chapter 3. AlloCure Tissue Genesis Antria Cellleris Corestem Celgene Corporation Mesoblast Cytori Therapeutics Pluristem Therapeutics Intrexon Lonza BioRestorative Therapies Pluristem Therapeutics iXCells Biotechnologies Cyagen Celltex Therapeutics Corporation

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The study is done with the help of analysis such as SWOT analysis and PESTEL analysis. It consists of the detailed study of current market trends along with the past statistics. The past years are considered as reference to get the predicted data for the forecast period. The report covers complete analysis of the Adipose Tissue-derived Stem Cell Therapy Market on the basis of regional and Global level. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry.

There are different marketing strategies that every marketer looks up to in order to ace the competition in the Global market. Some of the primary marketing strategies that is needed for every business to be successful are Passion, Focus, Watching the Data, Communicating the value To Your Customers, Your Understanding of Your Target Market. There is a target set in market that every marketing strategy has to reach. In addition, it also covers political and social factors which is likely to affect the growth of the market. It also covers and analysis several segments which are present in the market. A significant development has been recorded by the market of Adipose Tissue-derived Stem Cell Therapy, in past few years. It is also for it to grow further. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry.

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Market segment by Type, the product can be split into Segmentation by type: breakdown data from 2015 to 2020 in Section 2.3; and forecast to 2025 in section 10.7. Autologous Stem Cells Allogeneic Stem Cells

Segmentation by application: breakdown data from 2015 to 2020, in Section 2.4; and forecast to 2025 in section 10.8. Therapeutic Application Research Application

This report also splits the market by region: Breakdown data in Chapter 4, 5, 6, 7 and 8. Americas United States Canada Mexico Brazil APAC China Japan Korea Southeast Asia India Australia Europe Germany France UK Italy Russia Middle East & Africa Egypt South Africa Israel Turkey GCC Countries

The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report: Breakdown data in in Chapter 3. AlloCure Tissue Genesis Antria Cellleris Corestem Celgene Corporation Mesoblast Cytori Therapeutics Pluristem Therapeutics Intrexon Lonza BioRestorative Therapies Pluristem Therapeutics iXCells Biotechnologies Cyagen Celltex Therapeutics Corporation

Market segment by Application, split into Segmentation by application: breakdown data from 2015 to 2020, in Section 2.4; and forecast to 2025 in section 10.8. Therapeutic Application Research Application

One of the ways for the estimation for the growth of the market is estimation of the market share by the regions which is likely to contribute to the growth of the market in the estimated forecast period. In this, the growth and fall of each region is covered which is likely to boost the growth of the Adipose Tissue-derived Stem Cell Therapy Market. In addition, to determine and use precise methods, research methodology such as the qualitative and quantitative data is used for the estimation and determination of the Global Adipose Tissue-derived Stem Cell Therapy Market.

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Adipose Tissue-derived Stem Cell Therapy Market Share Analysis, Application, Strategies of Key Players & Forecast to 2025 - Factory Gate

Study clarifies the impact of getting old on hematopoietic stem cells – Microbioz India

By shifting mouse elderly hematopoietic stem cells (aged HSCs) to the environment of young mice (bone marrow niche), it was shown that the pattern of stem cell gene expression was rejuvenated to that of young hematopoietic stem cells. On the other hand, the function of elderly HSCs failed to recover in the young bone marrow niche. The epigenome (DNA methylation) of aged HSCs didnt change significantly even in the young bone marrow niche, and DNA methylation profiles were found to be a better index than the gene expression pattern of aged HSCs.

A research group headed by Professor Atsushi Iwama in the Division of Stem Cell and Molecular Medicine, The Institute of Medical Science, The University of Tokyo (IMSUT) declared these world-first Outcomes and was published in the Journal of Experimental Medicine (online) on November 24th.

The results will contribute to the development of treatments for age-related blood diseases.

Professor Atsushi Iwama, Lead Scientist, IMSUT

The research group investigated whether rejuvenating aged HSCs in a young bone marrow market environment would rejuvenate.

Tens of thousands of elderly hematopoietic stem/progenitor cells gathered from 20-month-old mice were transplanted into 8-week-old young mice without pretreatment like irradiation. After two months of follow-up, they collected bone marrow cells and performed flow cytometric analysis.

The research team also transplanted 10-week-old young mouse HSCs for comparison. In addition, engrafted aged HSCs were fractionated and RNA sequence analysis and DNA methylation analysis were conducted.

They discovered that engrafted elderly HSCs were less capable of producing hematopoietic cells compared to younger HSCs. They also showed that differentiation of aged HSCs into multipotent progenitor cells was persistently impaired even in the young bone marrow market, and that the direction of differentiation was biased. It was found that the transfer of aged HSCs into the young bone marrow market does not enhance their stem cell function.

A more detailed analysis may reveal mechanisms that irreversibly affect aged HSC function Aging studies focusing on HSCs have been chased in mice with a bone marrow transfer model. However, the effect of aging on HSCs remains to be clarified.

Professor Iwama says as follows. This analysis has a substantial impact because it clarified the effect of aging on HSCs. Our results are expected to contribute to further elucidation of the mechanism of aging in HSCs and comprehension of the pathogenic mechanism of age-related blood disorders.

Source:

Journal reference:

Kuribayashi, W.,et al.(2020) Limited rejuvenation of aged hematopoietic stem cells in young bone marrow niche.Journal of Experimental Medicine.doi.org/10.1084/jem.20192283.

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Study clarifies the impact of getting old on hematopoietic stem cells - Microbioz India

Report On Canine Stem Cell Therapy Market to 2026: (Industry Insights, Company Overview and Investment Analysis) – Farming Sector

Canine Stem Cell Therapy Market report shows the Industry Chain Structure as well as Macroeconomic Environment Analysis and Development Trend. The Canine Stem Cell Therapy Market report also provides the market impact and new opportunities created due to the COVID19/CORONA Virus catastrophe. The total market is further divided by company, by country, and by application/types for the competitive landscape analysis. The report then estimates 2020-2026 market development trends of Canine Stem Cell Therapy Industry.

TheCanine Stem Cell Therapy market report gives CAGR value, Industry Chains, Upstream, Geography, End-user, Application, Competitor analysis, SWOT Analysis, Sales, Revenue, Price, Gross Margin, Market Share, Import-Export, Trends and Forecast. The report also gives insight on entry and exit barriers of the industry.

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The Canine Stem Cell Therapy Market Report Covers Major Players:

Canine Stem Cell Therapy Market Breakdown Data by its type

Canine Stem Cell Therapy Market Breakdown Data by its Application

Canine Stem Cell Therapy Consumption Breakdown Data by Region: North America, United States, Canada, Mexico, Asia-Pacific, China, India, Japan, South Korea, Australia, Indonesia, Malaysia, Philippines, Thailand, Vietnam, Europe, Germany, France, UK, Italy, Russia, Rest of Europe, Central & South America, Brazil, Rest of South America, Middle East & Africa, GCC Countries, Turkey, Egypt, South Africa, Rest of Middle East & Africa.

The Study Objectives of Canine Stem Cell Therapy Market Report are:

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Major Points from Table of Contents:

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Report On Canine Stem Cell Therapy Market to 2026: (Industry Insights, Company Overview and Investment Analysis) - Farming Sector

Six Benefits of All-on-4 Implants Compared to Regular Dentures – Magazine of Santa Clarita

1. Bone is preserved: Dental implants preserve the bone in your jaw so that it can stay strong thanks to the stimulation when chewing. Dentures actually accelerate any bone loss or weakening in your jaw because they simply sit on your gums and dont stimulate your jawbone, which leads to bone loss with time. 2. More Comfortable: Your teeth are supported by solid implants, not gum tissue, so you wont get contact sores or friction marks on your gums. 3. Diet Benefits: Dentures limit what you can eat because you cant have anything too hard or too crunchy as it could break your dentures. They also cover your palate, which means you wont taste things as much. Implants allow you to eat almost anything you want and taste everything as it is meant to be tasted. 4. Self-Confidence: Implants dont move around when you speak or when you eat, so you dont have to be worried about embarrassment. They also help with spitting problems and speech concerns. 5. Improved Aesthetics: Tooth and bone loss can actually influence the way your face looks because you dont have the bone to support it. All-on-4s help to replace the original height between your upper and lower jaws so that your lips and cheeks are fuller. Say goodbye to the gaunt look! 6. Application: Implants permanently fix your teeth in your mouth, so you dont have to worry about messy, expensive adhesives. Schedule a Complimentary Consultation at 661-290-2825.

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Six Benefits of All-on-4 Implants Compared to Regular Dentures - Magazine of Santa Clarita

Allogeneic SCT Benefits Children and Adolescents With Relapsed Anaplastic Large Cell Lymphoma – OncLive

Findings from the International, Prospective ALCL-Relapse trial [NCT00317408] showed that allogeneic SCT after reinduction chemotherapy can lead to survival improvements in children and adolescents with high-risk relapsed or refractory anaplastic large cell lymphoma (ALCL), provide more insight into how to treat this population.

For patients with early-relapsed ALCL, data showed that autologous SCT wasnt effective.

Current standard chemotherapy reaches an event-free survival (EFS) of 70% at 5 years in children with ALCL, the study authors wrote. Retrospective data on the outcome of pediatric relapsed ALCL show a survival after relapse of more than 50%. In addition, there is no consensus on the optimal treatment approach in relapse, they explained.

Designed by the European Inter-Group for Childhood Non-Hodgkin Lymphoma, the prospective, stratified, multinational clinical trial was opened for patients at sites in 5 countriesthe United Kingdom, Germany, Austria, Switzerland, and the Czech Republic.

Patients were stratified according to the time of relapse, CD3 expression, and prior vinblastine therapy to 3 different consolidation approaches: allogeneic hematopoietic stem cell transplantation (SCT), autologous SCT, or vinblastine monotherapy.

Those whose disease progressed during frontline therapy (very high risk) or with a CD3-positive relapse (high risk) received allogeneic SCT after reinduction chemotherapy. Patients with a CD3-negative relapse within 1 year after initial diagnosis or prior exposure to vinblastine (intermediate risk) received autologous SCT after carmustine, etoposide, cytarabine, and melphalan (BEAM). However, this arm was terminated prematurely, and patients received vinblastine monotherapy instead. Patients with a CD3-negative relapse more than 1 year after initial diagnosis (low risk) received by weekly vinblastine monotherapy (6 mg/m2; maximum, 10 mg) for 24 months.

Investigators analyzed 105 patients; most were male and had ALK-positive tumors. The median age was 12.4 years and median time from initial diagnosis to relapsed/refractory disease was 8.5 months. Patients were recruited from April 2004 to February 2014 and the median follow-up time was 8.1 years.

At 5 years, overall survival (OS) in patients with central nervous systemnegative disease was 78% 4% and EFS, the primary endpoint, was 53% 5%.

Before termination of autologous SCT, EFS rates of patients in the very-high- (n = 17), high- (n = 26), intermediate- (n = 32), and low- (n = 21) risk groups were 41% 12%, 62% 10%, 44% 9%, and 81% 9%. The respective OS rates were 59% 12%, 73% 9%, 78% 7%, and 90% 6%.

Analyzing only the patients in the intermediate-risk group consolidated per protocol by autologous SCT, EFS and OS of 23 patients were 30% 10% and 78% 9%, respectively. The 5 patients with intermediate risk receiving vinblastine monotherapy experienced relapse again.

Compared with data from retrospective analyses, the survival of patients with refractory or relapsed ALCL reached 75% in our prospective trial, demonstrating that relapsed ALCL remains a curable disease, the study authors wrote.

The main limitation to the study was the implementation of the trial as treatment recommendation only in some countries, noted the authors. While recommendations were followed in approximately 90% of patients in the very high risk and high-risk groups, only 70% of patients in the intermediate risk group received autologous SCT, they explained.

Overall, investigators determined that patients with high-risk relapsed disease can benefit from allogeneic SCT and offer a chance for cure.

A long-term remission rate of 81% by outpatient vinblastine monotherapy, with low risk for late effects in patients with a late relapse was also observed by investigators. However, the monotherapy wasnt effective for early relapses.

Patients with early relapsed ALCL dont benefit from consolidation by autologous SCT or vinblastine monotherapy, the authors wrote. However, they may benefit from clinical trials testing a consolidation approach including new targeted therapies. Targeted agents should be tested as reinduction for all but late relapses. Given the efficacy of vinblastine in relapse, this shift-of-paradigm approach should be tested for low-risk patients front line.

Link:
Allogeneic SCT Benefits Children and Adolescents With Relapsed Anaplastic Large Cell Lymphoma - OncLive

Stem cells in the brain remain active and divide over months – News Landed

Stem cells are the most advanced and promising method, which could appear as a boon for the future generation. It serves as a repair system for the body, especially in adults. In general, they have the ability to renew different types of cells and can promote growth.

Scientists from the University of Zurichs Brain Research Institute found a cue stating that the mouse brain stem cell can undergo division even after months. Since now, there is no approved therapy for Alzheimers disease that results in memory loss. Eventually, the lead of the study identified that the brain cells divide in the region of the hippocampus, which is essential for learning and memory.

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Read Also: Lockdowns are an Opportunity, Not a Punishment

This evident conclusion was made by the usage of art microscopy and single-cell RNA sequencing. Researches used such techniques and analyzed the specific population of stem cells was emerging even after months. Besides, only single-cell RNA sequencing can depict the stem cells differences with their daughter cells in the molecular characteristics and the level of expression of genes.

With this approach, they are trying to increase the division number of stem cells. Once it happened, we could develop a novel therapy for neurodegenerative diseases. The future endeavors have the ability to recreate several models that would help in the medical field enormously.

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Source: Medical Xpress

+ Doctors who perform surgery on their birthday are likely lethargic and kill more patients + First case of COVID-19 in a wild animal

+ New threatening skin disease in dolphins is tied with changing global climate + Sturgill Simpson Stops Struggling

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Stem cells in the brain remain active and divide over months - News Landed

Exclusive Research on Stem Cell Therapy Products Market 2020 Segments, Regions, Strategy Analysis: Mesoblast, TiGenix, Osiris – LionLowdown

A research report on Stem Cell Therapy Products Market compiled byWorldwide Market Reports features a succinct analysis of the latest market trends. The report also includes detailed abstracts about statistics, revenue forecasts, and market valuation, which additionally highlights its status in the competitive landscape and growth trends accepted by major industry players.

Stem Cell Therapy Products Market reports provide a comprehensive overview of the global market size and share. Stem Cell Therapy Products market data reports also provide a 5 year pre-historic and forecast for the sector and include data on socio-economic data of global. Key stakeholders can consider statistics, tables & figures mentioned in this report for strategic planning which leads to the success of the organization.

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The major players profiled in this report include

New business opportunities and rising demand for Stem Cell Therapy Products market activities are the primary factors expected to drive the growth of the Stem Cell Therapy Products market in the United States, EU, Japan, China, India, Africa, Southeast Asia are the major region leading the Stem Cell Therapy Products market.

Impact of COVID-19:

Stem Cell Therapy Products Market report analyses the impact of Coronavirus (COVID-19) on the Stem Cell Therapy Products industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Stem Cell Therapy Products market in 2020.

COVID-19 can affect the global economy in 3 main ways: by directly affecting production and demand, by creating supply chain and market disturbance, and by its financial impact on firms and financial markets.

Further in the Stem Cell Therapy Products Market research reports, following points are included along with in-depth study of each point:

Production Analysis: Production of the Stem Cell Therapy Products is analyzed with respect to different regions, types, and applications. Here, price analysis of various Stem Cell Therapy Products Market key players is also covered.

Supply and Consumption: In continuation with sales, this section studies supply and consumption for the Stem Cell Therapy Products Market. This part also sheds light on the gap between supply and consumption. Import and export figures are also given in this report.

Key Strategic Developments: The study also includes the key strategic developments of the Stem Cell Therapy Products market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, Global and regional growth of the leading competitors operating in the market on a global and regional scale.

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In this report, the global Stem Cell Therapy Products market is valued at USD XX million in 2019 and is expected to reach USD XX million by the end of 2026, growing at a CAGR of XX% between 2019 and 2026.

History Year: 2013-2019

Base Year:2020

Estimated Year:2021

Forecast Year 2020to2026

The report provides an in-detail list of drivers and restraints in the market, which are influencing the market growth. Additionally, provides expected opportunities and emerging trends in the market.

The report includes a detailed study of Porters Five Forces model to analyze the different factors affecting the growth of the market. Moreover, the study also covers a market attractiveness analysis, PESTLE analysis, Value Chain Analysis, and SWOT analysis.

The report is a collection of first-hand information, qualitative assessment by industry analysts, and inputs from industry experts. Moreover, the report also maps the qualitative impact of various market factors on market segments and geographies. The major factor driving the adoption of Stem Cell Therapy Products is the formation of the major industry changing factors for the market segments are explored in this report. This report also covers the growth factors of the global market based on end-users.

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This report includes the estimation of market size for value (million US$) and volume (K Units). Both top-down and bottom-up approaches have been used to estimate and validate the market size of Stem Cell Therapy Products market, to estimate the size of various other dependent submarkets in the overall market. Key players in the market have been identified through secondary research, and their market shares have been determined through primary and secondary research. All percentage shares split, and breakdowns have been determined using secondary sources and verified primary sources.

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Exclusive Research on Stem Cell Therapy Products Market 2020 Segments, Regions, Strategy Analysis: Mesoblast, TiGenix, Osiris - LionLowdown

Canine Stem Cell Therapy Market 2026 by Product Type, Applications, Manufacturers, Revenue, Price, Share and Growth Rate & Forecast -…

The report entitled Canine Stem Cell Therapy market also gives a detailed idea of various technologies used by the manufacturers and industry experts to enhance the technological intervention in this market. An in-depth study in terms of production, Canine Stem Cell Therapy market revenue share and price are also a major factor of focus in this report. The company profile section also focusses on companies planning expansions. This can be very well studied through regional segmentation as all the key market players play an important role in uplifting the region, they operate in.

Furthermore, the report also gives the factors that might hamper the market growth over the forecast period. The detailed report on Canine Stem Cell Therapy market gives its readers a summary of the market movements and helps them to analyze all the other factors that can affect the market.

Leading Players Covered in Canine Stem Cell Therapy Market Report

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Market segmentation Canine Stem Cell Therapy market is split by Type and by Application. For the period 2016-2026, the growth among segments provide accurate calculations and forecasts for sales by Type and by Application in terms of volume and value. This analysis can help you expand your business by targeting qualified niche markets.

By Type

By Application

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Highlights of the report:

Table of Content: Canine Stem Cell Therapy Market Chapter 1. Research Objective 1.1 Objective, Definition & Scope 1.2 Methodology 1.2.1 Primary Research 1.2.2 Secondary Research 1.2.3 Market Forecast Estimation & Approach 1.2.4 Assumptions & Assessments 1.3 Insights and Growth Relevancy Mapping 1.3.1 FABRIC Platform 1.4 Data mining & efficiency

Chapter 2. Executive Summary 2.1 Plastics Additives and Master Batches Market Overview 2.2 Interconnectivity & Related markets 2.3 Ecosystem Map 2.4 Plastics Additives and Master Batches Market Business Segmentation 2.5 Plastics Additives and Master Batches Market Geographic Segmentation 2.6 Competition Outlook 2.7 Key Statistics

Chapter 3. Strategic Analysis 3.1 Plastics Additives and Master Batches Market Revenue Opportunities 3.2 Cost Optimization 3.3 Covid19 aftermath Analyst view 3.4 Plastics Additives And Master Batches Market Digital Transformation

Chapter 4. Market Dynamics 4.1 DROC 4.1.1 Drivers 4.1.2 Restraints 4.1.3 Opportunities 4.1.4 Challenges 4.2 PEST Analysis 4.2.1 Political 4.2.2 Economic 4.2.3 Social 4.2.4 Technological 4.3 Market Impacting Trends 4.3.1 Positive Impact Trends 4.3.2 Adverse Impact Trends 4.4 Porters 5-force Analysis 4.5 Market News By Segments 4.5.1 Organic News 4.5.2 Inorganic New

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Heterogenous Nature of MDS Warrants Further Research, Review Finds – AJMC.com Managed Markets Network

MDS is a complex disease with a fascinating origin. A plethora of molecular pathway disruptions have been postulated to explain the heterogeneity of the disease phenotype, but no exclusive genetic drivers are capable of recapitulating all its aspects, wrote the researchers, noting the heterogeneity of MDS introduces challenges when trying to fully understand the pathogenesis of the group of disorders.

They added, Seminal experimental studies have clarified the role of diverse gene mutations in the context of disease phenotypes, but the lack of faithful murine models and/or cell lines spontaneously carrying certain gene mutations have hampered the knowledge on how and why specific pathways are associated with MDS pathogenesis.

Recently, a gene expression-based classification was applied to RNA-sequencing data from CD34+ cells of 100 patients with MDS, resulting in the identification of 2 subcellular populations. The populations were associated and characterized by increased expression of genes related to erythroid/megakaryocytic lineages and those related to immature progenitor cells.

In addition, several genes in the iron pathway have been implicated in the pathogenesis of MDS, including:

Other recent research involved identifying differences in downstream pathways, particularly for genes of the spliceosomal complex, with all studies showing convergent functions in the 3 splice site. Some studies have shown that splicing factor mutations could result in the build-up of R-loops, including one that showed an increase in R-loops in an induced pluripotent stem cell clone harboring a SF3B1 mutation compared with a clone lacking SF3B1 mutations from a patient with MDS.

For patients with an SF3B1 mutation, luspatercept has been shown to improve response in low-grade MDS through its ability to increase erythroid maturation and hemoglobin levels.

More recently, splicing factor mutations were found to lead to the activation of inflammation and innate immunity pathways (e.g., NF-kB) or indirectly by deregulating histone acetylase (e.g., sirtuins), detailed the researchers. In fact, sirtuin 1 (SIRT1) was found to be involved in HSPC maintenance. U2AF1 mutant patients had IRAK4, a serine/threonine that activates NF-kB in the Toll-like receptor and T-cell receptor signaling pathways, that is aberrantly spliced.

Treatments targeting splicing factor mutations have mainly included developing pan-splicing modulators, including bacterially derived products and analogs that have been shown to bind the SF3B complex and disrupt spliceosome assembly.

Research focused on novel treatments for mutant p53 has also found that PRIMA-1Met, an investigational small molecule, restores the conformation of p53 and rescues p53 function. Another treatment, APR-246, has shown promise either as monotherapy or in combination with 5-azacitidine in reactivating p53 and inducing apoptosis in MDS cases carrying TP53 mutations.

Reference: Awada H, Thapa B, and Visconte V. The genomics of myelodysplastic syndrome: origins of disease evolution, biological pathways, and prognostic implications. Cells.2020;9(11):2512. doi:10.3390/cells9112512

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Heterogenous Nature of MDS Warrants Further Research, Review Finds - AJMC.com Managed Markets Network