NEW YORK, Aug. 17, 2022 /PRNewswire/ --As per Zion Market Research study, The global esoteric testing market size was worth USD 20,163.10 million in 2021 and is estimated to grow to USD 37917.67 million by 2028, with a compound annual growth rate (CAGR) of approximately 11.10 percent over the forecast period.

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Esoteric Testing Market: Overview

Esoteric testing involves the scientific examination of uncommon molecules and substances that are not often done in a clinical laboratory. These tests are carried out when a doctor needs more comprehensive information regarding the patient's condition. However, as laboratory testing technology develops, tests that are currently thought to be esoteric may end up being common in a short period of time. These tests are recommended by doctors when they require more detailed information than routine lab testing to finish a diagnosis, determine a prognosis, or select and track a treatment plan. Esoteric testing frequently requires the use of specialized tools and materials, as well as skilled personnel to conduct the test and analyze the results. These tests are typically more expensive and ordered less frequently than standard tests. Esoteric tests include those in endocrinology, genealogy, immunology, microbiological, molecular diagnostics, cancer, serology, and toxicity.

The rising esoteric testing market value for innovatively reasonable tests has resulted in significant initiatives in R&D, which will strengthen market development potential in the future. Planned and improved novel diagnostic techniques with diverse benefits for a wide range of intriguing infections will drive global market demand. The key factor influencing the growth of the global esoteric testing market is insufficient payback. Medicaid and Medicare coverage of proper medicine and tailored medication is extremely limited.

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Key Industry Insights & Findings of the Esoteric Testing Market Reports:

As per the analysis shared by our research analyst, the Esoteric Testing Market is expected to grow annually at a CAGR of around 11.10 % (2022-2028).

Through the primary research, it was established that the Esoteric Testing Market was valued at approximately USD 20163.1 million in 2021 and is projected to reach roughly USD 37917.67 million by 2028.

New advances in widespread proteomics, as well as stronger collaboration between researchers and physicians, contribute to understanding and detecting complicated illness characteristics.

Rapid growth for genomics and proteomics, as well as greater investment in the identification of innovative solutions to detect rare biomolecules, will drive overall market growth. Advancement of novel test methods with significant advantages for a variety of chronic illnesses will drive global esoteric testing market demand.

Targeted therapy and personalized medicine are only covered in a few areas under Medicare. Diagnostic test reimbursement has fallen in recent years, reducing the volume of tests performed.

The global esoteric testing market is expected to be driven by several developing technologies and integrated innovations such as genetic studies, digital PCR, next-generation sequencing & Pyrosequencing, microfluidic platforms, and enhanced molecular phenotyping technologies.

North America will continue to dominate the global esoteric testing market during the forecast period due to an increase in chronic illness, an expansion in government diagnostic activities, and an overall increase in community awareness of self-diagnosis.

The increasing prevalence of chronic diseases and an expanded focus on early detection and treatment of unusual infections will also drive the regional market growth.

Zion Market Research published the latest report titled 'Esoteric Testing Market By Type (Infectious Disease Testing, Endocrinology Testing, Oncology Testing), Technology (Enzyme-Linked Immunosorbent Assay, Chemiluminescence Immunoassay, Mass-Spectrometry, Real-Time Polymerase Chain Reaction, DNA Sequencing, Flow Cytometry, and Other Technologies), By End-Use (Hospital-based Laboratories and Independent & Reference Laboratories), and By Region - Global and Regional Industry Overview, Market Intelligence, Comprehensive Analysis, Historical Data, and Forecasts 2022 2028.'into their research database.

Industry Dynamics:

Esoteric Testing Market: Growth Drivers

Proteomics testing on a broad scale is fundamental to clinical translation and biological research. New advances in widespread proteomics, as well as stronger collaboration between researchers and physicians, contribute to understanding and detecting complicated illness characteristics. Rapid growth for genomics and proteomics, as well as greater investment in the identification of innovative solutions to detect rare biomolecules, will drive overall market growth. Advancement of novel test methods with significant advantages for a variety of chronic illnesses will drive global esoteric testing market demand

Esoteric Testing Market: Restraints

Limited reimbursements will hinder the expansion of the esoteric testing sector during the forecast period. Targeted therapy and personalized medicine are only covered in a few areas under Medicare. Diagnostic test reimbursement has fallen in recent years, reducing the volume of tests performed.

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Global Esoteric Testing Market: Opportunities

The global esoteric testing market is expected to be driven by several developing technologies and integrated innovations such as genetic studies, digital PCR, next-generation sequencing & Pyrosequencing, microfluidic platforms, and enhanced molecular phenotyping technologies. The increased usage of information technology has enabled faster and more effective data transfer. Industry participants in the esoteric testing market should take advantage of the prospects provided by growing economies such as China and India, which is predicted to determine the market's speedy growth over the forecast period.

Global Esoteric Testing Market: Segmentation

The global esoteric testing market is segregated based on technology, type, end-user, and region.

By type, the market is divided into infectious disease testing, oncology testing, endocrinology testing, genetic testing, toxicology testing, immunology testing, neurology testing, and other testing. Among these, the infectious disease testing segment dominated the market in 2021. The increased incidences of infectious diseases, combined with rising demand for specialty testing, will drive up overall sector test volumes. Furthermore, the increasing efficacy of esoteric testing in the identification of immunological illnesses is a significant component driving the segment's rapid rise.

By technology, the market is classified into enzyme-linked immunosorbent assay, chemiluminescence immunoassay, mass-spectrometry, real-time polymerase chain reaction, DNA sequencing, flow cytometry, and other technologies. Over the forecast period, chemiluminescence is expected to develop at the fastest rate. The rapid expansion in the use of chemiluminescence in esoteric testing due to its capacity to identify chemicals in small production test samples is driving the market growth.

By end-users, the market is segmented into hospital-based laboratories and independent & reference laboratories. The independent & reference laboratories held a significant share in the market in 2021. Growth is being fueled by the ongoing automation technology of diagnostic laboratories, the rise in accredited laboratories, broad reimbursement acceptance, and attractive benefits provided by these providers. The segment is expanding because of continuous diagnostic laboratory automation & digitization, an increase in accredited laboratories, extensive financing support, and aggressive incentives provided by these providers.

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List of Key Players in Esoteric Testing Market:

LabCorp

Quest Diagnostics

OPKO Health

H.U. Group Holdings Inc.

Helios Limited

Sonic Healthcare

Mayo Foundation for Medical Education and Research (MFMER US)

Eurofins Scientific

Stanford Clinical Pathology

Foundation Medicine

Kind star Global Technology Inc.

ARUP Laboratories

Georgia Esoteric & Molecular Laboratory LLC

Thorofare Technologies Ltd.

ACM Global Laboratories

Biaxiality Labs

National Medical Services Inc. (NMS)

Baylor Esoteric and Molecular Laboratory

Cerda Expert

Health Quest Esoteric

BUHLMANN Diagnostics Corp (BDC US)

BP Diagnostic Centre SDN BHD

Flow Health

Leo Labs Inc.

Key questions answered in this report:

What are the growth rate forecast and market size for Esoteric Testing Market?

What are the key driving factors propelling the Esoteric Testing Market forward?

What are the most important companies in the Esoteric Testing Market Industry?

What segments does the Esoteric Testing Market cover?

How can I receive a free copy of the Esoteric Testing Market sample report and company profiles?

Report Scope:

Report Attribute

Details

Market size value in 2021

USD 20163.1 Million

Revenue forecast in 2028

USD 37917.67 Million

Growth Rate

CAGR of almost 11.10 % 2022-2028

Base Year

2020

Historic Years

2016 2021

Forecast Years

2022 2028

Segments Covered

By Product Type, By Application, And By End Use

Forecast Units

Value (USD Billion), and Volume (Units)

Quantitative Units

Revenue in USD million/billion and CAGR from 2022 to 2028

Regions Covered

North America, Europe, Asia Pacific, Latin America, and Middle East & Africa, and Rest of World

Countries Covered

U.S., Canada, Mexico, U.K., Germany, France, Italy, Spain, China, India, Japan, South Korea, Brazil, Argentina, GCC Countries, and South Africa, among others

Companies Covered

LabCorp , Quest Diagnostics , and OPKO Health . H.U. Group Holdings, Inc. , Helios Limited , Sonic Healthcare , Mayo Foundation for Medical Education and Research (MFMER, US), Eurofins Scientific, Stanford Clinical Pathology , Foundation Medicine , Kind star Global Technology, Inc. , ARUP Laboratories , Georgia Esoteric & Molecular Laboratory, LLC , Thorofare Technologies Ltd. , ACM Global Laboratories, Biaxiality Labs , National Medical Services Inc. (NMS) , Baylor Esoteric and Molecular Laboratory , Cerda Expert , Health Quest Esoteric , BUHLMANN Diagnostics Corp (BDC, US), BP Diagnostic Centre SDN BHD , Flow Health , and Leo Labs, Inc. .

Report Coverage

Market growth drivers, restraints, opportunities, Porter's five forces analysis, PEST analysis, value chain analysis, regulatory landscape, market attractiveness analysis by segments and region, company market share analysis, and COVID-19 impact analysis.

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Statistics on Global Esoteric Testing Market Size & Share to Surpass USD 37917.67 Million by 2028, Exhibit a CAGR of 11.10% | Industry Trends,...

Recommendation and review posted by Bethany Smith

Each week we highlight the noteworthy titles that have recently hit streaming platforms in the United States. Check out this weeks selections below and past round-upshere.

Costa Brava, Lebanon (Mounia Akl)

What can you do when your homelands falling apart? The easy answer is stay or leave, but both options carry too much complexity to simply choose and be done. For starters, not everyone has that choicewhether due to finances, family, or myriad other reasons. And those who are able must dig deep within themselves to rationalize why. Do you leave because of greater opportunity? Do you stay because you want to be part of the solution? Or do you find yourself in a sort of purgatoryone foot planted on each side, only to discover your fear of losing out on the benefits of one for the potential of the other has you locked in stasis? Thats where Walid (Saleh Bakri) currently exists. Jared M. (full review)

Where to Stream: Kino Now

The Ken Jacobs Collection

Filmatique is exclusively streaming work by Ken Jacobs, one of the most wildly creative and influential artists in cinema history. The 20-title curation spans Jacobss nearly 70-year career: early films using New York City as a poetic landscape (Orchard Street) and as a setting for Smiths carnivalesque performances (Little Stabs at Happiness and Blonde Cobra); experiments with found footage (Tom, Tom, the Pipers Son); and an embracing digital tools to create stroboscopic effects that turn silent shorts and Victorian stereoscopic photographs into mind-expanding 3D investigations (Capitalism: Child Labor).

Where to Stream: Filmatique

The Legend of Molly Johnson (Leah Purcell)

A favorite of Leah Purcells as a child, Henry Lawsons short story The Drovers Wife was always at the front of her mind when growing into adulthood as an artist. It only makes sense, then, that she would take that 1892 tale and reimagine it as an Australian western that would bring her own ancestral history as a fair-skinned Aboriginal woman to light. First she had to give the titular wife a name: Molly Johnson. Next it was fleshing out a dramatic narrative beyond that of a devoted mother staying up all night to protect her children from a hidden snake while reminiscing about all the other times for which she did the same (fire and flood) with her husband consistently away. A legend was born. Jared M. (full review)

Where to Stream: VOD

Media Man (Danny Lyon)

Over the next two weeks Le Cinma Club will stream, for free, Media Man. Filmed by Danny Lyon over the course of five years with his wife Nancy Weiss,it barrels across cities and countrysides, quilting together a warm study of Americas many faces. This patchwork portrait of overlooked people and their passions encapsulates Lyons ability to immortalize fleeting histories through momentous encounters.

Where to Stream: Le Cinma Club

On the Count of Three (Jerrod Carmichael)

Considering the raw, uncomfortable truths found in Jerrod Carmichaels comedy, the logline of his directorial debut shouldnt come as a surprise: two friends make a pact to end their lives and experience one final day together before plans to carry through with the dual deeds. Though not scripted by Carmichael himself,The Carmichael Showwriter-producer Ari Katcher and hisRamyco-writer Ryan Welch have crafted a character-focused story with layers of necessary darkness and pathos while still injecting humor that mostly feels like a natural fit considering the subject matter. As to be expected,Taste of Cherrythis is not, but with its layers of despair and dark comedy mixed with genuine friendship, Carmichael owes a bit toMikey and Nickyin this ride-or-die, last-day-in-a-life outing. Even if the last act doesnt succeed as intended,On the Count of Threethreads the difficult task of finding the humor in hopelessness while not exploiting the genuine pain of severe depression. Jordan R. (full review)

Where to Stream: Hulu

Orphan: First Kill (William Brent Bell)

Screenwriters Alex Mace and David Leslie Johnson-McGoldrick gave their character Leena Klammer, aka Esther Albright, a complete back story at the end of Jaume Collet-SerrasOrphan. A victim of a rare hormone disorder known as hypopituitarism, causing proportional dwarfism, had made it so this 33-year-old woman looked as though she were only nine. The condition obviously prevented her from being seen as a mature adult; thus she used it to her manipulative advantage. What began as thieving, however, eventually escalated to murder once her desire to sleep with her adopted fathers reinforced that finding love, while unquestionably difficult, proved impossible when her targets initially believed themselves to be herdad. At least seven people were left dead in her wake alongside their homes charred remains. Jared M. (full review)

Where to Stream: VOD

The Princess (Ed Perkins)

By design, Ed PerkinsThe Princesskeeps a healthy, mediated distance from its subject, the late Princess of Wales. After all, the news is the first draft of history, and the film restricts its view to what we knew at the time. In doing so Perkins orchestrates a film that demystifies the lore and media obsession with Princess Diana, in essence pointing its gaze inwardtowards the media covering the adoring fans in the moment. They sometimes turn against the media, defending the Peoples Princess in shouting matches on talk shows and sometimes in the streets, yet the economic incentives for rabid paparazzi persist. John F. (full review)

Where to Stream: HBO Max

Shadow (Zhang Yimou)

With its gorgeously choreographed sword duels, sabers slicing through paddles of blood and rain, watercolor bi-chromatic palettes and sumptuous costumes, Zhang YimousShadow(Ying) is a film of visual charms. To enter into the Fifth Generation maestros latest period piece is to be invited to marvel at a 116-minute long dance a stunning return to form from a director whod previously ventured into semi-autobiographical terrain with the 2014 movingComing Home, and later veered into the bombastic Chinese-cum-Matt Damon blockbuster epic letdownThe Great Wall(2016).Shadowbrings heart and spectacle together, and the result is a bombastic martial artswuxiareplete with duels of breath-taking beauty that will please longtime Zhang acolytes and newbies alike. Leonardo G. (full review)

Where to Stream: MUBI (free for 30 days)

Spin Me Round (Jeff Baena)

There are a number of reasons to recommendSpin Me Round, a winning comedy about the manager of an Olive Garden-style restaurant who gets chosen to attend a training program in Italy. The film features a shockingly stacked supporting cast of comedy titans: Aubrey Plaza, Tim Heidecker, Molly Shannon, Fred Armisen, Lil Rel Howery,The Offices Zach Woods,Lookings Lauren Weedman, andHigh Maintenances Ben Sinclair. The latest from director Jeff Baena, whose strange and wildly diverse filmography includesLife After BethandThe Little Hours,Spin Me Roundstood out as one of the better comedies in a strong SXSW lineup earlier this yeareven its Harlequin romance novel-apingposteris a gem. Chris S. (full review)

Where to Stream: VOD

Vengeance (B.J. Novak)

Despite its generic title, B.J. Novaks feature-directing debutVengeanceis a smart, subversive fish-out-of-water comedy about a stereotypical coastal elite that gets intellectually catfished into traveling to a remote Texas town complete with a rodeo and Whataburger, seemingly the defacto town meeting place. Novak plays Ben Manalowitz, a Brooklyn-based writer for theNew Yorkerand Bumble power user who dreams of branching out into podcasting, though he just hasnt had his big break. After trading witty quips with famed ladies man John Mayer at a rooftop party about the first-world problem of having too many beautiful women in your phone to keep track of, he meets Eloise (Issa Rae), a producer at a podcast network modeled on NPR. John F. (full review)

Where to Stream: VOD

Also New to Streaming

Hulu

Red Cliff

MUBI (free for 30 days)

I Am LoveBetween Two DawnsCycling the FrameEaster EggsCaballerangoBabysitter

Prime Video

Lucky

VOD

Get Away If You CanThe Immaculate RoomLost Illusions

Excerpt from:
New to Streaming: Ken Jacobs, The Legend of Molly Johnson, Spin Me Round, The Princess & More - The Film Stage

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Global Metabolic Partnering 2015-2022: Deal trends, players and financials" report has been added to ResearchAndMarkets.com's offering.

Global Metabolic Partnering 2015 to 2022 provides the full collection of 1100+ Metabolic disease deals signed between the world's pharmaceutical and biotechnology companies since 2015.

Most of the deals included within the report occur when a licensee obtains a right or an option right to license a licensor's product or technology. More often these days these deals tend to be multi-component including both a collaborative R&D and a commercialization of outcomes element.

The report takes readers through the comprehensive Metabolic disease deal trends, key players and top deal values allowing the understanding of how, why and under what terms companies are currently entering Metabolic deals.

The report presents financial deal terms values for Metabolic deals, where available listing by overall headline values, upfront payments, milestones and royalties enabling readers to analyse and benchmark the value of current deals.

The initial chapters of this report provide an orientation of Metabolic dealmaking trends.

Chapter 1 provides an introduction to the report.

Chapter 2 provides an overview of the trends in Metabolic dealmaking since 2015 covering trends by year, deal type, stage of development, technology type and therapeutic indication.

Chapter 3 includes an analysis of financial deal terms covering headline value, upfront payment, milestone payments and royalty rates.

Chapter 4 provides a review of the leading Metabolic deals since 2015. Deals are listed by headline value. The chapter includes the top 25 most active Metabolic dealmakers, together with a full listing of deals to which they are a party. Where the deal has an agreement contract published at the SEC a link provides online access to the contract.

Chapter 5 provides comprehensive access to Metabolic deals since 2015 where a deal contract is available, providing the user with direct access to contracts as filed with the SEC regulatory authorities. Each deal title links via Weblink to an online version of the deal record contract document, providing easy access to each contract document on demand.

Chapter 6 provides a comprehensive directory of all Metabolic partnering deals by specific Metabolic target announced since 2015. The chapter is organized by specific Metabolic therapeutic target. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand.

In addition, a comprehensive appendix is provided with each report of all Metabolic partnering deals signed and announced since 2015. The appendices are organized by company A-Z, stage of development at signing, deal type (collaborative R&D, co-promotion, licensing etc) and technology type. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand.

The report includes deals for the following indications: Acromegaly, Addison's disease, Cirrhosis, Cushing's syndrome, Diabetes, Type 1, Type 2, Insipidus, Fatty liver, Gallstones, Goitre, Growth hormone disorders, Gynaecomastia, Inborn errors of metabolism, Phenylketonuria, Hyperaldosteronism, Hypercalcaemia, Hyperthyroidism, Hypocalcaemia, Hypogonadism, Hypopituitarism, Hypothyroidism, Liver disease, Nonalcoholic steatohepatitis (NASH), Lysosomal storage disorders, Nutrition and vitamins, Rickets, Pheochromocytoma, Primary bilary cirrhosis, Prolactinemia, Short stature, Syndrome of Inappropriate Antidiuretic Hormone (SIADH), Thyroid disease, plus other metabolic indications.

Report scope

Global Metabolic Partnering 2015 to 2022 includes:

In Global Metabolic Partnering 2015 to 2022, available deals and contracts are listed by:

Analyzing actual contract agreements allows assessment of the following:

Key Topics Covered:

Executive Summary

Chapter 1 - Introduction

Chapter 2 - Trends in Metabolic dealmaking

2.1. Introduction

2.2. Metabolic partnering over the years

2.3. Metabolic partnering by deal type

2.4. Metabolic partnering by industry sector

2.5. Metabolic partnering by stage of development

2.6. Metabolic partnering by technology type

2.7. Metabolic partnering by therapeutic indication

Chapter 3 - Financial deal terms for Metabolic partnering

3.1. Introduction

3.2. Disclosed financials terms for Metabolic partnering

3.3. Metabolic partnering headline values

3.4. Metabolic deal upfront payments

3.5. Metabolic deal milestone payments

3.6. Metabolic royalty rates

Chapter 4 - Leading Metabolic deals and dealmakers

4.1. Introduction

4.2. Most active in Metabolic partnering

4.3. List of most active dealmakers in Metabolic

4.4. Top Metabolic deals by value

Chapter 5 - Metabolic contract document directory

5.1. Introduction

5.2. Metabolic partnering deals where contract document available

Chapter 6 - Metabolic dealmaking by therapeutic target

6.1. Introduction

6.2. Deals by Metabolic therapeutic target

Appendices

Appendix 1 - Directory of Metabolic deals by company A-Z since 2015

Appendix 2 - Directory of Metabolic deals by deal type since 2015

Appendix 3 - Directory of Metabolic deals by stage of development since 2015

Appendix 4 - Directory of Metabolic deals by technology type since 2015

For more information about this report visit https://www.researchandmarkets.com/r/7b8gbu

Originally posted here:
Global Metabolic Partnering Report 2022: Deal Trends, Players and Financials Analysis of 1100+ Deals Signed Since 2015 - ResearchAndMarkets.com -...

Recommendation and review posted by Bethany Smith

Non-alcoholic fatty liver disease (NAFLD) is a form of chronic liver disease increasingly arising among children and adults. It ranges from simple steatosis and non-alcoholic steatohepatitis (NASH) to advanced fibrosis and cirrhosis with chronic liver failure. Non-alcoholic fatty liver disease is a condition marked by the deposition of lipids in the liver cells in patients who consume very little or no alcohol. The histological picture is similar to alcohol-induced liver injury, yet it occurs in people who do not drink or consume very little. Liver enzymes such as alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), and other markers of liver injury are increased in NAFLD. However, those levels are normal in a large percentage of patients with NAFLD [1,2].

According to a meta-analysis that included more than eight and a half million people from 22 countries, the worldwide prevalence of fatty liver is 24%, whereas, in the United States, the prevalence of NAFLD is also24% [3]. Compared to a worldwide population, patients with NAFLD and non-alcoholic steatohepatitis have an increased death rate due to liver-related diseases. These patients are strongly associated with extrahepatic diseases such as endocrinopathies like diabetes mellitus, metabolic syndrome, thyroid dysfunction, insulin resistance, cardiovascular diseases, and chronic kidney diseases [3].

Subclinical hypothyroidism is characterized by increased plasma thyroid-stimulating hormone (TSH) and plasma thyroid hormone levels within the reference range and without obvious clinical symptoms. Overt hypothyroidism has obvious clinical symptoms and low free Thyroxine (fT4) [4]. Thyroid hormones are crucial in multiple physiological processes like homeostasis, mineral, lipid, carbohydrates, and protein metabolisms. Lipid metabolism has been reported, such as increased metabolic rate, weight loss, lipolysis, and lowering serum cholesterol levels. The physiological process of thyroid hormones (TH) affects almost every organ, and the liver is one of the most critical targets of TH [5]. Low thyroid hormone function may cause hypercholesterolemia which plays a fundamental role in the pathophysiology of hypothyroidism-induced NAFLD [6].

In recent years, more attention has been brought to hypothyroidism-induced NAFLD. The pathogenesis of non-alcoholic fatty liver disease and thyroid hormones may have close correlations, as research in the last ten years showed that disruptions of cellular TH signaling, trigger chronic hepatic disease, including non-alcoholic fatty liver disease, alcoholic fatty liver disease, and hepatocellular carcinoma. This disruption results in decreased hepatic lipid utilization and secondary lipid accumulation [3,5,7].

The present review will discuss associations between hypothyroidism and NAFLD and the importance of thyroxine (T4), triiodothyronine (T3), and thyroid-stimulating hormone (TSH) in its pathophysiology. The association between NAFLD and hypothyroidism is still controversial. At present, there are multiple studies showing an association. Therefore, it is necessary to conduct more studies in order to answer this question [8,9].

Since discovering alcohol-related liver disease in 1845, fatty liver disease (FLD) has been primarily linked to excessive alcohol consumption. Non-alcoholic fatty liver disease was first described in 1981, and it includes a wide variety of hepatic conditions that include simple steatosis to steatohepatitis, advanced fibrosis, and cirrhosis. Non-alcoholic steatohepatitis and NAFLD have a similar presentation on histology, but in the case of NAFLD, there is different pathophysiology [10].

The prevalence of NAFLD among the population drastically increased in the last twenty years. NAFLD manifests itself in various ways in people worldwide, affecting both the female and male sex. The global prevalence of NAFLD is 25 percent, which is approximately as high as one billion and growing. One of the most common causes of chronic liver disease in the United States is NAFLD. It affects 80 to 100 million people, and approximately 25% of the cases progress to NASH. NAFLD includes a wide range of histopathological conditions such as non-alcoholic fatty liver (NAFL), non-alcoholic steatohepatitis (NASH), fibrosis, NASH cirrhosis, and NASH-related hepatocellular carcinoma (HCC). NAFLD is a diagnosis of exclusion. The number of NASH patients with cirrhosis is rising, resulting in an increase in liver transplantation for end-stage cirrhosis [11-13].

NAFLD is characterized by the accumulation of more than five percent of hepatic fat in the absence of any secondary causes. NAFLD can manifest in different ways, ranging from a simple accumulation of fat that is a metabolic condition with no symptoms and no inflammation of the liver (non-alcoholic fatty liver) to symptomatic non-alcoholic steatohepatitis with inflammation of the liver. It has the capacity to progress into severe fibrosis, end-stage liver disease, and HCC depending on NAFLD subtypes. While 10 to 20% of NAFLD patients develop NASH, only zero to four percent develop cirrhosis within 10 to 20 years. The rising prevalence of NAFLD with advanced fibrosis is concerning because individuals with advanced fibrosis tend to have an increased death rate of liver-related and non-liver-related diseases than the general population [11,14-17].

NAFLD is divided into four stages: 1. Simple steatosis (NAFL) - accumulation of fat in the liver without inflammation or damage to the liver cells; 2. NASH - accumulation of fat in the liver with the presence of inflammation and liver celldamage - hepatitis; 3. Fibrosis - scarring of the liver tissue (excess of fibrous tissue) in the inflamed liver. Fibrosis is categorized into stages - mild, moderate, and advanced, or into four stages (0-4) based on the progression of scaring; 4. Liver cirrhosis - permanently damage to the liver with nodules of damaged liver cells surrounded by scar tissue; 5. Progression to HCC [18].

To diagnose NAFLD, we must exclude other causes such as alcohol abuse (which is defined as consumption of >20 g/day for women or >30 g/day for men), drug abuse, Hepatitis C and Hepatitis B, Wilson's disease, hemochromatosis, celiac disease, and autoimmune liver disease, because NAFLD is a diagnosis of exclusion [19,20].

Insulin resistance and obesity are the two most important risk factors for NAFLD. However, NAFLD is associated with other extrahepatic manifestations such as obstructive sleep apnea, hypertension, gut microbiota alterations, dyslipidemia, sedentary lifestyle, overconsumption of carbohydrates (leading to de novo lipogenesis), and genetic predisposition. The prevalence of NAFLD in obese patients is 51%, patients with dyslipidemia are 69%, Type 2 diabetes 22,5%, and patients with hypertension are 39,3%, respectively. Nonetheless, there are other endocrine diseases that are associated with NAFLD: hypopituitarism, hypogonadism, polycystic ovarian syndrome, and hypothyroidism [11,14,19].

Thyroxine (T4 or 3,3,5,5-tetraiodo-l-thyronine) and triiodothyronine (T3 or 3,5,3-triiodo-l-thyronine) are thyroid hormones. The thyroid gland produces predominantly T4, but deiodination of T4 in peripheral tissues creates the majority of systemic T3, which is the most potent thyroid hormone. The hypothalamic-pituitary axis controls the secretion of TH from the thyroid gland. Thyrotropin-releasing hormone (TRH) is a hormone released by the hypothalamus that acts on the pituitary gland by binding to G protein-coupled TRH receptors on the thyrotrope, causing an increase in intracellularcyclic adenosine monophosphate (cAMP) and the production of thyrotropin. TSH stimulates the generation and release of TH by binding to a G protein-coupled TSH receptor on the thyroid follicular cell [21,22].

The thyroid gland regulates many biological activities in the liver, adipose tissue, central nervous, cardiovascular, and musculoskeletal systems by producing and releasing thyroid hormones such as thyroxine and triiodothyronine into the blood. One of the main physiological functions of TH is to maintain basal energy expenditure by glucose and lipid metabolism modulation. Thyroid hormones increase the production of fatty acids, modulate the sensitivity of the insulin in the hepatic tissue, and decrease hepatic gluconeogenesis, in addition to increasing lipid export and oxidation. The TH receptor (TR) regulates lipid metabolism (synthesis, mobilization, and degradation) as well as the metabolism of glucose by regulating the expression of several other nuclear receptors.HMG-CoA reductase (3-hydroxy-3methylglutarylcoenzyme A), which initiates cholesterol biosynthesis, can be stimulated by thyroid hormones. Also, triiodothyronine (T3) has the ability to bind to certain thyroid hormone-responsive elements and activate the LDL receptor gene, thereby upregulating LDL receptors. Also, thyroid hormones regulate HDL metabolism. Thyroid hormones increase the expression of the regulatory sterol element-binding protein-2, which regulates cholesterol metabolism (SREBP-2). T3 also stimulates lipoprotein lipase, which catabolizes triglyceride (TG)-rich lipoproteins, resulting in a reduction in TG levels [15,22-25].

Both T3 and T4 act via TRs, when thyroid hormones binding to the genes of thyroid receptors, which then help facilitate the transport of free fatty acids in the cells of the hepatic tissue, with the help of protein transporters like liver fatty acid-binding proteins (L-FABPs), fatty acid transporter proteins (FATPs), and fatty acid translocases (FAT). THs can increase intrahepatic lipolysis through lipophagy in hepatocytes via THR-, resulting in decreased TG clearance and increased TG hepatic uptake [10,26]. This type of lipophagy is linked to triiodothyronine physiology. T3 plays a significant role in fatty acid transport to mitochondria and mitochondrial metabolism by altering the range of hepatic lipid-related metabolites T3. T3 stimulates lipophagy in cultured hepatic cell lines, which leads to hepatic autophagy and ketogenesis. These results suggest that T3 regulates hepatic autophagy, which is an important step in the management of NAFLD [27].

We know that hypothyroidism is linked to hypometabolism. It is characterized by increasing weight, decreasing resting energy expenditure, and decreasing gluconeogenesis and lipolysis. Obesity, impaired lipid metabolism, and insulin resistance can be caused by THs dysfunction, which are symptoms of metabolic syndrome that are also present in NAFLD [28]. Overt hypothyroidism and subclinical hypothyroidism are both related to NAFLD [8].

In subclinical hypothyroidism, there are several factors that contribute to the progression of NAFLD, such as the physiology of TSH on the hepatocytes cell membrane, impairing hepatic triglyceride metabolism (promoting hepatic lipogenesis) through the upregulation of SREBP-1c activity provoked by stimulation of TSH receptors. Also, in the case of low thyroid hormone, diminished glucose-sensing receptors of the beta cells of the pancreas. In this manner, it reduces insulin secretion and decreases lipolysis in the adipose tissue, increasing the traffic of FFA to the hepatic tissue [19,22,29].

Patients with hypothyroidism often have atherogenic dyslipidemia. It has been established that the pathophysiology of hypothyroidism-induced hyperlipidemia is due to a reduction in cholesterol excretion and a marked rise in apoB lipoproteins as a result of insufficient catabolism and turnover brought on by a reduction in the number of low-density lipoprotein (LDL) receptors on the surface of the hepatic cells. In this manner, elevated total and LDL cholesterol levels are common in hypothyroid patients. Also, decreased clearance level of triglycerides from plasma and the buildup of intermediate density lipoproteins were noted in hypothyroidism. Hypothyroidism-induced NAFLD may develop because of the rising LDL and accumulation of triglycerides in the hepatic tissue [22,27]. Accumulation of the lipids causes oxidative stress and inflammatory response in the liver [8].Another factor that may be involved in the thyroid-liver complex is leptin. Leptin is elevated in hypothyroid patients and is also elevated in NAFLD patients. Leptin can promote hepatic insulin resistance and play a role in hepatic fibrogenesis [7].

In the meta-analysis from 2018 that involved 26 studies and 61,548 participants, 11 studies with a total of 47,217 patients with NAFLD/NASH had significantly higher thyroid-stimulating hormones than healthy controls, this difference remains significant. With the progression of NAFLD, the level of TSH increased as well. The research found that hypothyroidism raised the probability of non-alcoholic fatty liver disease or non-alcoholic steatohepatitis. These results were disputed in subsequent evaluations based on the degree of hypothyroidism. The risk of non-alcoholic steatohepatitis was substantially correlated with subclinical hypothyroidism but not with the risk of NAFLD. On the other hand, the risk of non-alcoholic fatty liver disease is substantially correlated with overt hypothyroidism in adults but not with the risk of NASH. These results might be inconsistent due to the small number of included studies. This meta-analysis also discovered that the relationship between NAFLD and free T3 (FT3) and free T4 (FT4) may vary by the number of people that live in the area and that non-alcoholic fatty liver disease is perhaps unrelated to FT3 or FT4. These results might be evidence that TSH, rather than thyroid hormones, plays a key role in the onset and progression of NAFLD [8].

In another meta-analysis from 2021 that involved 17 articles and 14,514 participants included, elevated TSH levels maybe be a risk factor that is independently associated with NAFLD. FT4 was significantly associated with NAFLD when FT3 was not associated [30].

There is no drug therapy for hypothyroidism-induced NAFLD that is currently approved. Steatosis can be reduced through structured lifestyle changes such as weight loss, dietary changes such as reduced drinking of alcohol, decreasing intake of food and drinks that have a high level of fructose, and increased daily activities and workouts [16,29].

In a case report of a patient with NASH who was diagnosed with Graves' disease (GD), the liver enzyme levels improved after the onset of GD and subsequent hyperthyroidism. They worsened after starting treatment and returning to a euthyroid state [31].

A small-molecule liver-directed thyroid hormone receptor agonist (Resmetirom) with high liver uptake is administered orally and is under development (currently in Phase 3 of trial) for the treatment of NAFLD/NASH and familial hypercholesterolemia [4,32].

Continued here:
Nonalcoholic Fatty Liver Disease and Hypothyroidism: What You Need to Know - Cureus

Recommendation and review posted by Bethany Smith

Dr. Julie Russak is a board-certified dermatologist and founder of Russak Dermatology Clinic and Russak+ Aesthetic Center.

NEW YORK (PRWEB) August 10, 2022

About three months after a person recovers from COVID-19, excess clumps of hair come out in ones hand, stick to the back or clog the shower drain. A brush contains much more hair than normal, or hair litters the bathroom floor or a bed pillow in disturbing amounts.

Some hair loss about 100 hairs per day is natural and normal. When that increases to 300 or more hairs per day, something is not right. Post-COVID, if hair thins perceptibly, the condition is called telogen effluvium.

Telogen effluvium is not specific to COVID. It can happen several months after any time the body undergoes stress, such as after a high fever, the flu or another serious illness, so its no surprise that it happens to COVID patients. Its also common during pregnancy and after giving birth, and certain medications can trigger it. However, since the beginning of the pandemic, the incidence of telogen effluvium has increased 400 percent. The hair loss associated with telogen effluvium occurs primarily on the head and excessive shedding can continue for many months.

Scientists have yet to link hair-loss directly to the COVID virus. Most experts agree that the stress related to contracting COVID, anxiety over the disease, job loss, feelings of isolation and depression, and changes in lifestyle due to the pandemic are the real culprits. The good news is that in most cases of telogen effluvium, hair growth and loss return to normal, but it takes a long time, up to a year.

Dr. Julie Russaks signature Hair Restoration Therapy is a safe, nonsurgical way to stimulate hair growth. The treatment combines Platelet-Rich Plasma (PRP) Therapy, Exosome Stem Cell injections, diffused microneedling and topical and oral supplements. Its a unique, clinically-proven approach that is more proactive because it delivers these regenerative ingredients into the scalp, awakening hair follicles and stimulating hair growth.

PRP TherapyBlood platelets contain hundreds of healing proteins called growth factors, which are known for their regenerative qualities. Normally, platelets make up about 10 percent of the bloods cellular components. In PRP Therapy, that percentage is flipped to 90 percent. When platelet-rich plasma is infused into the scalp, it encourages hair growth at an accelerated rate.

During a Hair Restoration Therapy treatment, first a phlebotomist draws a small sample of the patients blood, which is spun in a centrifuge designed to concentrate platelets. In essence, the red and white blood cells are separated from the platelets and plasma (clear-gold fluid), creating PRP. The concentration of platelets in PRP, and thus the concentration of growth factors, can be up to ten times greater than in normal blood.

Once the PRP is isolated, the clinician injects and microneedles the PRP into the scalp. Microneedling ensures the valuable growth factors in the PRP penetrate directly into the hair follicles, and injections allow the PRP to get even deeper into the skin. The growth factors in the PRP then stimulate stem cells around the hair follicles into an active growth phase.

In addition, non-platelet components of the blood plasma are present in lower amounts, and contains nutrients, vitamins, hormones, electrolytes and proteins that are essential to hair health. As a result, hair grows back and at a faster rate than if left only to Mother Nature.

Exosome BoosterExosomes are a new breakthrough in regenerative hair therapy that triggers healing, cell stimulation and regeneration. Exosomes are extracellular vesicles, or small bubbles, released from stem cells. They contain genetic signals that stimulate cells, in this case healthy hair follicles.

The exosomes released by regenerative cells such as stem cells are potent drivers of repair, regeneration and healing. When hair loss occurs, exosomes signal the follicles to heal themselves which stimulates new hair growth. Exosomes contain bioactive molecules that play a crucial role in cell-to-cell communications and are vital in all cellular regenerative processes. When they are infused directly into the scalp, the result is intensive hair rejuvenation, especially when combined with PRP Therapy.

Reversing Other Types of Hair LossThinning hair can result from many different factors in addition to COVID and stress, including family history, hormonal changes, certain disease and medications, scalp imbalance and more. PRP Therapy enhanced with an Exosome Booster delivers a comprehensive approach to treating hair loss in many situations. Any person who has experienced miniaturization, or hair thinning, is an ideal candidate for this proven hair restoration therapy. Its customized for each patient with the goal of reactivating the hair follicles.

The protocol consists of four sessions spaced one month apart, with maintenance treatments every four to six months after that to keep hair follicles stimulated and to maintain hair growth. These in-clinic treatments are augmented by oral supplements and Russak Dermatologys proprietary topical spray, called Hair Fortifying RX.

Hair Fortifying RX is a concentrated blend of vitamins and hair regrowth stimulants, including 10% minoxidil, which reduces inflammation around the follicle and stimulates hair regrowth on its own. When combined with Hair Restoration Therapy, the result is fuller, denser hair!

Selected by the New York Times as a New York Super Doctor, Dr. Julie Russak, M.D., FAAD, is a board-certified dermatologist, Fellow of the American Academy of Dermatology and the founder/CEO of Russak Dermatology Clinic in New York City. In addition to providing comprehensive care in cosmetic and medical dermatology through the clinic, she serves on the faculty of Mount Sinai Hospital and was awarded Teacher of the Year in 2022. Her expertise includes general, cosmetic and pediatric dermatology, skin cancer and dermatologic surgery. For more information, go to http://www.russakdermatology.com.

The rest is here:
Curbing COVID-Related Hair Loss: Russak Dermatology's Hair Restoration Therapy - PR Web

Recommendation and review posted by Bethany Smith

Suggest participates in affiliate programs with various companies. Links originating on Suggests website that lead to purchases or reservations on affiliate sites generate revenue for Suggest . This means that Suggest may earn a commission if/when you click on or make purchases via affiliate links.

One of the many perks of being a Hollywood celebrityother than making millions of dollars to play pretend on a movie or TV setis access to products that can help them look their very best. Thats especially the case when it comes to skincare. And there is one product on the market that countless A-listers swear byAugustinus Bader The Rich Cream.

Of course, celebrity status doesnt make you an expert. But regular shoppers do agree that this luxury moisturizer has completely changed their skin for the better. Yes, its pricey. But, many are certain that this high-dollar skin cream is absolutely worth it.

Augustinus Bader The Rich Cream has been popping up all over social media in recent months. Everyone from Jennifer Aniston to Lily Aldridge to Victoria Beckham to Sandra Oh is swearing by this essential high-end moisturizer.

Supermodel Karen Elsona self-proclaimed high-end beauty addicthas called Augustinus Bader The Rich Cream the secret elixir for her skin that tends to be her go-to for all things.

RELATED: Elizabeth Olsen Swears By This Jennifer Aniston-Approved Device To Tighten And Brighten Skin

The brand credits TFC-8 technology as the reason this moisturizer is so effective. They say it sends your stem cells into repair mode with a mix of ceramides, peptides, amino acids, vitamins C and E, and cholesterol. Also helping to achieve that plump and silky skin is hyaluronic acid, squalane, and shea butter.

According to a 55-year-old cellular biologist on the Nordstrom website, this mix of ingredients has created permanent changes in her skin that has left it unbelievably even-toned and hydrated.

Other reviews indicate that it takes just a tiny amount to see visibly smoother wrinkles and way less prominent dry patches.

I thought it might be too heavy and thick for my skin. I was wrong. It is a thick, rich cream but it does go into the skin easily, and honestly, the first time I used it I could instantly see my skin had plumped, one reviewer shared.

Another added, Hands down the best cream I have ever tried and Ive tried them all. Yes it is expensive but worth every penny. Your skin is smooth, hydrated, and glowing. It just works and you dont need much.

Augustinus Bader The Rich Cream is available in multiple sizes, from a 0.5 ounce bottle, the standard 1 ounce bottle, or a larger 1.7 ounce bottle. Buying the larger size will definitely help you save a few bucks, and it will be an investment that pays off immediately.

Read more:
Jennifer Aniston And Sandra Oh Swear By This Moisturizer That Shoppers Say Created Permanent Changes To Their Skin - Suggest

Recommendation and review posted by Bethany Smith

Professor Augustinus Baders skincare products contain the patented TFC8 technology, backed by 30 years of science and research - and results have shown an increase by 110% of more elasticity in the skin as well!

Image: Augustinus Bader)

When we hear on the grapevine that celebrities are obsessing over skincare products or with a beauty brand - we too are equally eager to hear the secret behind their gorgeous, glowing skin.

Augustinus Bader, whos earned a cult-beauty status thanks to his rejuvenating skin care products, is the man whom Jennifer Aniston, Kim Kardashian and Victoria Beckham all love too. And its not just celebrities who hail his namesake products as the secret weapon behind nourished and renewed skin, but beauty editors and dermatologists too. Not to mention contain the patented TFC8 technology, which is backed by 30 years of science and research.

And we have a way to you can get 20% off your next order, thanks to the auto-replenish programme! Customers are able to save 20% on each order when they subscribe to regular, customisable, delivery cycles. How cool is that?

Augustinus Bader

Augustinus Bader

Augustinus Bader

And its so simple too!

The skincare formulas are hand crafted by Professor Augustinus Bader in his own laboratory. Hes a globally recognised biomedical scientist, physician and one of the foremost experts in the field of stem cell biology and regenerative medicine. So its no wonder why celebs are quick to reach for his products before hitting the red carpet.

Not to mention his products have received 90 industry awards in just four years - and products have been voted The Greatest Skincare Of All Time.

Best of all? The results of Augustinus Bader products are proven through extensive clinical trials - and who wouldnt want younger looking skin in as little as four weeks?

Based on a 4-week clinical trial, with participants using hero product The Rich Cream: Forehead wrinkles visibly reduced by 37%, crow's feet wrinkles visibly reduced by 54%, crow's feet fine lines visibly reduced by 46% and of those testers, skin felt 92% firmer and 110% more elasticity in the skin - in just 4 weeks!

So what are you waiting for? Give Augustinus Bader products a go and see how your skin can change in four weeks too!

Have you used any of the Augustinus Bader skincare products before? Or are you keen to give them a try and see what they could do for you? Let us know your thoughts in the comments section below.

Originally posted here:
Victoria Beckham and Kim Kardashian are fans of Augustinus Baders skincare range - and you can get 20% off - The Mirror

Recommendation and review posted by Bethany Smith

A bone marrow transplant is a medical procedure that replacesthe bone marrow with healthy cells. Replacement cells might come from either ones own body or from a donor. A stem cell transplant, or more specifically, a hematopoietic stem cell transplant, is another name for a bone marrow transplant. Transplantation can be used to treat leukemia, myeloma, and lymphoma, as well as other blood and immune system illnesses that impact the bone marrow. Cancer and cancer treatment can damage the hematopoietic stem cells. Hematopoietic stem cells are blood-forming stem cells. Hematopoietic stem cells that are damaged may not develop into red blood cells, white blood cells, or platelets. These blood cells are vital, and each one serves a specific purpose. A bone marrow transplant can help the body regenerate the red blood cells, white blood cells, and platelets it requires.

The global Bone Marrow Transplant market is estimated to be valued at $10,356.1 Mn Mn in 2021 and is expected to exhibit a CAGR of 4.0% over the forecast period (2022-2028).

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The study provides data on the most exact revenue estimates for the complete market and its segments to aid industry leaders and new participants in this market. The purpose of this study is to help stakeholders better understand the competitive landscape and design suitable go-to-market strategies. The market size, features, and growth of theBone Marrow Transplantindustry are segmented by type, application, and consumption area in this study. Furthermore, key sections of the GlobalBone Marrow Transplantmarket are evaluated based on their performance, such as cost of production, dispatch, application, volume of usage, and arrangement.

Competitive Analysis: Global Bone Marrow Transplant Market

Detailed Segmentation:

By Type:

By Treatment Type:

:

: United States, Canada, and Mexico & : Argentina, Chile, Brazil and Others & : Saudi Arabia, UAE, Israel, Turkey, Egypt, South Africa & Rest of MEA. : UK, France, Italy, Germany, Spain, BeNeLux, Russia, NORDIC Nations and Rest of Europe. -: India, China, Japan, South Korea, Indonesia, Thailand, Singapore, Australia and Rest of APAC.

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This report has looked at high-impact rendering elements and causes to help readers comprehend the overall trend. Furthermore, the report contains constraints and obstacles that may operate as roadblocks for the players. This will enable people to pay attention and make well-informed business judgments. Specialists have also focused on future business opportunities.

Competitive Outlook:

Company profiles, revenue sharing, and SWOT analyses of the major players in theBone Marrow TransplantMarket are also included in the research. TheBone Marrow Transplantindustry research offers a thorough examination of the key aspects that are changing, allowing you to stay ahead of the competition. These market measuring methods assist in the identification of market drivers, constraints, weaknesses, opportunities, and threats in the global market.

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Youll be able to quickly pinpoint the information you need thanks to the concise analysis, clear graph, and table style.

Denotes the area and market segment that is likely to expand the fastest and dominate the market.

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Provides industry understanding via Value Chain Market Dynamics scenario, as well as market development potential in the next years

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Increasing efforts to set up centers for Bone Marrow Transplant is expected to Boost the growth of the market, Top Key players | Lonza, Merck KgaA,...

Recommendation and review posted by Bethany Smith

Are you age 18-40? Did you know thousands of people are waiting to find a lifesaving donor match for marrow or stem cells, many of them children?

Lord of New Life Lutheran Church of Midland is hosting a Be The Match donor registry drive at the Monday, Aug. 15 through Saturday, Aug. 20 at Midland County Fairgrounds. The drive will be 11 a.m.-10 p.m. Monday through Friday and 10 a.m.-8 p.m. Saturday at building 25, space 47.

Be The Match, operated by the National Marrow Donor Program, is a global leader in marrow/stem cell transplantation. They also host an international registry that matches patients who need a lifesaving transplant with eligible donors.

A marrow/stem cell donation is often the only cure for leukemia, lymphoma, sickle cell and other deadly diseases.

Matches are determined by genetic traits, not blood type, and can be difficult to find. Only 30% of patients have matches within their own family. Thousands of patients are waiting to find a match.

Many are not even aware of the program and that they can literally save someones life,said Jamie Fiste, drive organizer. If anyone is interested, we encourage them to stop by our booth. We will have everything they need to get placed on the registry. People can also register right from home.

If contacted by Be The Match, the organization arranges the entire donation process.

For more information about Be The Match, the donation process, or to register from home, visit http://www.bethematch.org.

For more information about the registry drive, visit http://www.lordofnewlife.org/be-the-match-bone-marrow-registry-drive.

See the original post:
Save a life by joining Be The Match registry at Midland Fair - Midland Daily News

Recommendation and review posted by Bethany Smith

By UNC Health Talk

In the first few minutes after a baby is born, the babys umbilical cord (which carries oxygen and nutrients from the placenta to the growing fetus) is cut and usually discarded.

But many people dont realize that extracting and donating the nutrient-rich blood from the cord can save someone elses life, as it did for 1-year-old Cole Baranowski.

Thanks to a cord blood transplant, Cole is now in remission from leukemia. He received an umbilical cord blood unit after his unrelated adult donor fell through.

UNC Health pediatric hematologist-oncologist Kimberly Kasow, DO, who specializes in bone marrow transplantation, explains how umbilical cord blood donation works and how it can give critically ill patients a second chance at life.

Umbilical cord blood is the blood left in the umbilical cord and placenta after a baby is delivered. It is rich in young blood-making cells, called hematopoietic stem cells.

Most cells can only make copies of themselves; for example, a skin cell can only make another skin cell. However, hematopoietic stem cells can mature into different types of blood cells in the body that will grow up to support the immune system and blood function. These stem cells can be used to treat more than 70 different diseases, including blood and bone marrow cancers such as leukemia and lymphoma; other blood disorders, such as sickle cell disease; bone marrow failures such as aplastic anemia; and disorders of the immune system, such as severe combined immunodeficiency.

Many patients with one of these conditions will require a lifesaving blood or marrow transplant to replace the unhealthy blood-forming cells with healthy ones. (Bone marrow is the spongy tissue inside bones that contains stem cells that can develop into blood cells.)

As bone marrow transplant doctors, we will consider using umbilical cord blood for transplant because it is enriched in young cells that will grow and mature to be the healthy red and white blood cells and platelets that our patients need to survive, Dr. Kasow says.

When a patient needs a lifesaving blood or marrow transplant, umbilical cord blood is one of three sources that providers use, in addition to bone marrow and blood from adult donors. Cord blood may be a preferred option; it doesnt have to match the recipient as perfectly as a marrow or blood product from an adult donor should, since umbilical cord cells are not as mature. Cord blood transplant can be used in both children and adults.

Theres a lot to consider when selecting the right stem cell product and donor for our patients, Dr. Kasow says. We want the closest match possible to keep their body from rejecting these new cells and to prevent undesirable side effects. Oftentimes, these young cells in cord blood may help minimize those risks.

The Baranowskis experienced the benefits of a cord blood donation firsthand.

Cole had been scheduled to receive a bone marrow transplant from an adult donor, but when he was only a week from his scheduled transplant, the Baranowskis learned that his donor was not able to donate.

Cole had already been through two rounds of chemotherapy and CAR T-cell therapy (the process of training the patients own immune cells to destroy cancer cells) to get him healthy enough to receive the transplant.

To make matters worse, his parents had just learned that his cancer was starting to come back. Frantic to get him the healthy blood he needed, they felt they were back at square one. Thankfully, an umbilical cord blood match provided a quick solution.

The day we found out the donor fell through was as bad as the day we found out Cole had leukemia, says Allison Baranowski, Coles mom.

But were so thankful someone was so thoughtful and generous to donate their babys cord blood it saved Coles life. We didnt have to jump through as many hoops as if we had decided to pursue another adult donor.

Cole received his cord blood transplant March 4. His care team at the UNC Childrens decorated his room in a Batman theme and celebrated his second birthday his second chance at life.

Awareness about the ability to donate your babys umbilical cord blood is so important. Theres really no downside to it, and it can literally save a life, Baranowski says.

There are two ways to preserve umbilical cord blood:

Donate it to a public cord blood bank.

If you want to donate your babys cord blood, make sure your hospital is a collection site. For example, UNC Rex Hospital in Raleigh and N.C. Womens Hospital in Chapel Hill are collection sites for the Carolinas Cord Blood Bank, one of the largest and most respected cord blood banks in the world. Youll need to let your care team know that you want to donate before you go into labor.

If you choose to donate, youll be asked to provide your familys medical history, similar to when you donate blood as an adult. Your blood will be screened for genetic disorders and infections, per U.S. Food and Drug Administration guidelines. The babys umbilical cord blood will be tested to make sure it is healthy and has enough cells. If its viable, it will be frozen and listed in a registry, available for anyone in need of a transplant. Cord blood units that do not qualify can often be used for research, which is also important for future lifesaving discoveries.

Cord blood donations are an especially important option for ethnic minorities in need of a transplant, as it is usually harder for them to find adult donors who are a match when a sibling match is not an option.

Pay a third-party company to store it in a private cord blood bank to be used by the child or a family member later in life.

Some families choose to bank their childs umbilical cord blood privately, in the event that the child or another family member might need it in the future. If you choose to bank your childs cord blood, numerous vendors offer kits that you can purchase before delivery. Make sure you ask how much it will cost to collect and process the cord blood, and to store it annually.

Banking cord blood is a good option for parents who already know of a medical issue in the family that might require a blood or marrow transplant. For example, it might be recommended to a parent who already has a child with leukemia. However, theres no guarantee the childs cord blood will match the family members needs, Dr. Kasow says.

If you dont already have a family member in need of a blood transplant, chances are very small that you would need to use cord blood thats been banked privately, she says. And on the flip side, if you donate the cord blood to a public bank and find later that you need it for your child or a family member, your provider can check to see if it made it into the registry and if it is still available.

If you have questions about whether to donate or preserve your babys umbilical cord blood, talk to your doctor. For more information on cord blood preservation, visit BeTheMatch.org or the American College of Obstetricians and Gynecologists.

Editor's note: This story originally appeared on the UNC Health Talk blog.

Read the original here:
Umbilical cord blood donation saved 1-year-old boy's life - WRAL News

Recommendation and review posted by Bethany Smith

Cell membrane-coated nanoparticles, applied in targeted drug delivery strategies, combine the intrinsic advantages of synthetic nanoparticles and cell membranes. Although stem cell-based delivery systems were highlighted for their targeting capability in tumor therapy, inappropriate stem cells may promote tumor growth.

Study:Stem cell membrane-camouflaged targeted delivery system in tumor. Image Credit:pinkeyes/Shutterstock.com

A review published in the journalMaterials Today Biosummarized the role of stem cell membrane-camouflaged targeted delivery system in tumor therapy and focused on the underlying mechanisms of stem cell homing toward target tumors. Nanoparticle-coated stem cell membranes have enhanced targetability, biocompatibility, and drug loading capacity.

Furthermore, the clinical applications of induced pluripotent stem cells (iPSCs) and mesenchymal stem cells (MSCs) were investigated as membrane-camouflaged targeted delivery systems for their anti-tumor therapies. In concurrence, the stem cell membrane-coated nanoparticles have immense prospects in tumor therapy.

Cell-based targeted delivery systems have low immunogenicity and toxicity, innate targeting capability, ability to integrate receptors, and long circulation time. Cells such as red blood cells, platelets, stem cells, tumor cells, immune cells, and even viral/bacterial cells can serve as effective natural vesicles.

MSCs derived from the umbilical cord (UC-MSCs), bone marrow (BM-MSCs), and adipose tissue (ATMSCs) are utilized in clinical applications. However, iPSCs are preferable over MSCs in clinical applications due to their easy fetch by transcription factor-based reprogramming of differentiation of somatic cells.

Stem cells (MSCs/ iPSCs) can be easily isolated and used as drug delivery systems for tumor therapy. Stem cell-based delivery systems have inflammation or tumor lesions targeting capacity. However, stem cells are often entrapped in the lung due to their size, resulting in microembolism.

Cell membrane-coated nanoparticles are applied in targeted delivery strategies. To this end, stem cell membrane-coated nanoparticles have tremendous prospects in biomedical applications. Although previous reports mentioned the role of cell membrane-coated nanocarriers in tumor therapy, delivery systems based on stem cell membranes have not been explored extensively.

Stem cell membrane-coated nanoparticles obtained from stem cells have complex functioning and can achieve biological interfacing. Consequently, stem cell membrane-coated nanoparticles served as novel drug delivery systems that could effectively target the tumor.

Previous reports mentioned the preparation of doxorubicin (DOX) loaded, poly (lactic-co-glycolic acid) (PLGA) coated MSC membrane-based nanovesicles, which showed higher cellular uptake than their PLGA uncoated counterparts. Similarly, the DOX-loaded MSC membrane-coated gelatin nanogels showed enhanced storage stability and sustained drug release.

Thus, the stem cell membrane-coated nanoparticles served as novel carriers for stem cells and facilitated the targeted delivery of the drugs at the tumor site. Since the stem cell membrane-coated nanoparticles had good targeting and penetration abilities, they enhanced the efficiency of chemotherapeutic agents in tumor therapy and minimized the side effects.

Reactive oxygen species (ROS) based photodynamic therapy (PDT) is mediated by photosensitizers with laser irradiations. Previous reports mentioned the development of MSC membrane-based mesoporous silica up-conversion ([emailprotected]2) nanoparticles that efficiently targeted the tumor due to their high affinity after being coated with MSC membrane.

These cell membrane-coated nanoparticles showed high cytocompatibility (with hepatocyte cells) and hemocompatibility (with blood). Moreover, the [emailprotected]2 nanoparticles-based PDT therapy under 980-nanometer laser irradiations could inhibit the tumors in vivo and in vitro. Consequently, the stem cell membrane-coated nanoparticles had circulation for an extended time and escaped the immune system, thereby increasing their accumulation at the tumor site.

Stem cell membrane-coated nanoparticles were also applied to deliver small interfering RNA (siRNA) via magnetic hyperthermia therapy and imaging. Previous reports mentioned the preparation of superparamagnetic iron oxide (SPIO) nanoparticles using an MSC membrane that reduced the immune response.

Additionally, the CD44 adhesion receptors were preserved on the surface of the MSC membrane during preparation. These prepared nanovesicles were unrecognized by macrophages, which enabled their stability in blood circulation. The nanosize and tumor homing capacity of MSCs helped the nanovesicles generate a dark contrast in T2-weight magnetic resonance imaging (MRI).

Cell membrane-coated nanoparticles helped fabricate various targeted delivery strategies. Especially, stem cell membrane-coated nanoparticles have the following advantages: stem cells are easy to isolate and expand in vitro. Thus, multilineage potential and phenotypes could be preserved for more than 50 population doublings in vitro.

Stem cell membrane-coated nanoparticles also have an intrinsic capacity to target inflammation or tumor lesions. Hence, these nanoparticles were established for tumor therapy, building a strong foundation for stem cell membrane-mediated delivery systems.

On the other hand, stem cell membrane-coated nanoparticles have the following drawbacks: Despite various sources for collecting MSCs (UC-MSCs/BM-MSCs/ATMSCs), the number of cells obtained is limited, although iPSCs are relatively easy to fetch by reprogramming differentiated somatic cells, the reprogramming is a high-cost step, restricting the clinical applications of iPSCs.

Zhang, W., Huang, X. (2022). Stem cell membrane-camouflaged targeted delivery system in tumor. Materials Today Bio.https://www.sciencedirect.com/science/article/pii/S2590006422001752

Disclaimer: The views expressed here are those of the author expressed in their private capacity and do not necessarily represent the views of AZoM.com Limited T/A AZoNetwork the owner and operator of this website. This disclaimer forms part of the Terms and conditions of use of this website.

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Stem Cell Membrane-Coated Nanoparticles in Tumor Therapy - AZoNano

Recommendation and review posted by Bethany Smith

The publication of proposals for new European Union rules on the use of substances of human origin (SoHO) has elicited largely positive responses from industryso far. But it is early days yet. The draft text, released in mid-July, will now enter that curious black box that is the EUs legislative machinery, and exactly when and how it emerges is, at the moment, anyones guess. What can be said at this stage is that there is wide relief right across the healthcare community in Europe that these proposals allow the chance of at last dragging the current legal frameworkcreated in the hugely different scientific and technological circumstances of 20 years agokicking and screaming into the second quarter of the 21st century (which it will be by the time any new rule proposed comes into force).

Expectations are highand diverse. The scope of the products covered by the legislation is wide, from blood transfusions to plasma collection, from IVF to plasma-derived medicines (PDMPs) and to transplants of bone marrow or stem cells or corneas, and extending as far as breast milk and fecal microbiota. The proposed new rules imply big changes for companies working on advanced therapy medicinal products (ATMPs) and on PDMPsand are accordingly provoking anxieties as well as hopes, not least because an EU-wide authorization procedure is foreseen, with upfront risk assessment and clinical outcome data collection requirements. Even if the proposals insist these requirements will be proportionate to the identified risks, these are the sort of suggestions that cause company bosses to lose sleep over yet another round of new regulation.

EuropaBio, representing many European biotech companies, welcomed the proposal as an avenue that can support the growth of ATMPs and allow Europe to ensure it reclaims its title as a global leader in ATMP innovation. According to Claire Skentelbery, its director general, establishing a predictable, future-proof, and fair SoHO framework is critical for the sector, as requirements for donation, procurement, and testing apply to blood, tissues, and cells used in the production of ATMPs.

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), with many member companies involved in ATMP development, was also glad to see the proposal emerge. But its early reaction highlighted concerns over the risk that the new rules could unhelpfully spill over into regulation of ATMPs, where, for instance, borderline issues frequently emerge when blood cells are used as starting materials for ATMP manufacture. As starting material for ATMPs, maintaining the clear classification between blood, tissues and cells, and ATMPs is crucial to provide clarity and appropriate regulatory standards when developing these transformative treatments, said EUCOPE. And in a clear hint of more reserved position, it added: We will stay active around the new SoHO regulation discussion.

The industry-backed European Alliance for Transformative Therapies (TRANSFORM) says it expects the outcome to reflect exigence of high quality standards, and, like EUCOPE, insists on retaining a clear distinction between advanced therapies and blood, tissues, and cells.The US-based Alliance for Regenerative Medicine greeted the proposal as holding the promise of improving patient safety while establishing greater legal and regulatory certainty for patients and developersagain with an emphasis on maintaining clear regulatory distinctions between starting materials and ATMPs. The EUs ATMP classification has established the region as a global leader in the regulation of cell and gene therapies, it saysurging that determining the classification of borderline cases between SoHOs and ATMPs should be based on the advice of the European Medicines Agency.

Another industry grouping engaged in PDMPs also took a conspicuously conditional stance on the proposals. The Plasma Protein Therapeutics Association said it welcomes positive developments but regrets missed opportunities to support both donors and patients. In particular, it fears not enough will be done to increase supplies of the plasma at the heart of the crucial and often irreplaceable rare disease treatments its members manufacture.

The declared aims of the EUs proposal are to increase the safety and quality of the processes in which these substances are donated and used, and to boost their supply and to ease their availability. The EU says it wants to offer support for innovation. But how far any such support will turn out to be balanced with new obstacles is what is behind the industry caution.

Reflector is Pharmaceutical Executives correspondent in Brussels

Read the rest here:
Optimism Abounds On New EU Blood And Tissue Rules - Pharmaceutical Executive

Recommendation and review posted by Bethany Smith

HAEMOGLOBIN IS a protein in your red blood cells. Your red blood cells carry oxygen throughout your body. If you have a condition that affects your bodys ability to make red blood cells, your haemoglobin levels may drop. Low haemoglobin levels may be a symptom of several conditions, including different kinds of anaemia and cancer.

If a disease or condition affects your bodys ability to produce red blood cells, your haemoglobin levels may drop. When your haemoglobin level is low, it means your body is not getting enough oxygen, making you feel very tired and weak.

Normal haemoglobin levels are different for men and women. For men, a normal level ranges between 14.0 grams per decilitre (gm/dL) and 17.5 gm/dL. For women, a normal level ranges between 12.3 gm/dL and 15.3 gm/dL. A severe low-haemoglobin level for men is 13.5 gm/dL or lower. For women, a severe low haemoglobin level is 12 gm/dL.

Your doctor diagnoses low haemoglobin by taking samples of your blood and measuring the amount of haemoglobin in it. This is a haemoglobin test. They may also analyse different types of haemoglobin in your red blood cells, or haemoglobin electrophoresis.

Several factors affect haemoglobin levels and the following situations may be among them:

Your body produces red blood cells and white blood cells in your bone marrow. Sometimes, conditions and diseases affect your bone marrows ability to produce or support enough red blood cells.

Your body produces enough red blood cells, but the cells are dying faster than your body can replace them.

You are losing blood from injury or illness. You lose iron any time you lose blood. Sometimes, women have low haemoglobin levels when they have their periods. You may also lose blood if you have internal bleeding, such as a bleeding ulcer.

Your body cannot absorb iron, which affects your bodys ability to develop red blood cells.

You are not getting enough essential nutrients like iron and vitamins B12 and B9.

Your bone marrow produces red blood cells. Diseases, conditions and other factors that affect red blood cell production include:

Lymphoma: This is a term for cancers in your lymphatic system. If you have lymphoma cells in your bone marrow, those cells can crowd out red blood cells, reducing the number of red blood cells.

Leukaemia: This is cancer of your blood and bone marrow. Leukaemia cells in your bone marrow can limit the number of red blood cells your bone marrow produces.

Anaemia: There are many kinds of anaemias involving low-haemoglobin levels. For example, if you have aplastic anaemia, the stem cells in your bone marrow dont create enough blood cells. In pernicious anaemia, an autoimmune disorder keeps your body from absorbing vitamin B12. Without enough B12, your body produces fewer red blood cells.

Multiple Myeloma: This causes your body to develop abnormal plasma cells that may displace red blood cells.

Chronic Kidney Disease: Your kidneys dont produce the hormone that signals to your bone marrow to make red blood cells. Chronic kidney disease affects this process.

Antiretroviral medications: These medications treat certain viruses. Sometimes these medications damage your bone marrow, affecting its ability to make enough red blood cells.

Chemotherapy: Chemotherapy may affect bone marrow cells, reducing the number of red blood cells your bone marrow produces.

Doctors treat low haemoglobin by diagnosing the underlying cause. For example, if your haemoglobin levels are low, your healthcare provider may do tests that reveal you have iron-deficiency anaemia. If that is your situation, they will treat your anaemia with supplements. They may recommend that you try to follow an iron-rich diet. In most cases, treating the underlying cause of anaemia will bring the haemoglobin level up.

Many things can cause low haemoglobin, and most of the time you cannot manage low haemoglobin on your own. But eating a vitamin-rich diet can help maintain your red blood cells. Generally, a balanced diet with a focus on important nutrients is the best way to maintain healthy red blood cells and haemoglobin.

keisha.hill@gleanerjm.comSOURCE: Centres for Disease Control and Prevention

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Factors that affect haemoglobin levels and how to detect when it's low - Jamaica Gleaner

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Country

United States of AmericaUS Virgin IslandsUnited States Minor Outlying IslandsCanadaMexico, United Mexican StatesBahamas, Commonwealth of theCuba, Republic ofDominican RepublicHaiti, Republic ofJamaicaAfghanistanAlbania, People's Socialist Republic ofAlgeria, People's Democratic Republic ofAmerican SamoaAndorra, Principality ofAngola, Republic ofAnguillaAntarctica (the territory South of 60 deg S)Antigua and BarbudaArgentina, Argentine RepublicArmeniaArubaAustralia, Commonwealth ofAustria, Republic ofAzerbaijan, Republic ofBahrain, Kingdom ofBangladesh, People's Republic ofBarbadosBelarusBelgium, Kingdom ofBelizeBenin, People's Republic ofBermudaBhutan, Kingdom ofBolivia, Republic ofBosnia and HerzegovinaBotswana, Republic ofBouvet Island (Bouvetoya)Brazil, Federative Republic ofBritish Indian Ocean Territory (Chagos Archipelago)British Virgin IslandsBrunei DarussalamBulgaria, People's Republic ofBurkina FasoBurundi, Republic ofCambodia, Kingdom ofCameroon, United Republic ofCape Verde, Republic ofCayman IslandsCentral African RepublicChad, Republic ofChile, Republic ofChina, People's Republic ofChristmas IslandCocos (Keeling) IslandsColombia, Republic ofComoros, Union of theCongo, Democratic Republic ofCongo, People's Republic ofCook IslandsCosta Rica, Republic ofCote D'Ivoire, Ivory Coast, Republic of theCyprus, Republic ofCzech RepublicDenmark, Kingdom ofDjibouti, Republic ofDominica, Commonwealth ofEcuador, Republic ofEgypt, Arab Republic ofEl Salvador, Republic ofEquatorial Guinea, Republic ofEritreaEstoniaEthiopiaFaeroe IslandsFalkland Islands (Malvinas)Fiji, Republic of the Fiji IslandsFinland, Republic ofFrance, French RepublicFrench GuianaFrench PolynesiaFrench Southern TerritoriesGabon, Gabonese RepublicGambia, Republic of theGeorgiaGermanyGhana, Republic ofGibraltarGreece, Hellenic RepublicGreenlandGrenadaGuadaloupeGuamGuatemala, Republic ofGuinea, RevolutionaryPeople's Rep'c ofGuinea-Bissau, Republic ofGuyana, Republic ofHeard and McDonald IslandsHoly See (Vatican City State)Honduras, Republic ofHong Kong, Special Administrative Region of ChinaHrvatska (Croatia)Hungary, Hungarian People's RepublicIceland, Republic ofIndia, Republic ofIndonesia, Republic ofIran, Islamic Republic ofIraq, Republic ofIrelandIsrael, State ofItaly, Italian RepublicJapanJordan, Hashemite Kingdom ofKazakhstan, Republic ofKenya, Republic ofKiribati, Republic ofKorea, Democratic People's Republic ofKorea, Republic ofKuwait, State ofKyrgyz RepublicLao People's Democratic RepublicLatviaLebanon, Lebanese RepublicLesotho, Kingdom ofLiberia, Republic ofLibyan Arab JamahiriyaLiechtenstein, Principality ofLithuaniaLuxembourg, Grand Duchy ofMacao, Special Administrative Region of ChinaMacedonia, the former Yugoslav Republic ofMadagascar, Republic ofMalawi, Republic ofMalaysiaMaldives, Republic ofMali, Republic ofMalta, Republic ofMarshall IslandsMartiniqueMauritania, Islamic Republic ofMauritiusMayotteMicronesia, Federated States ofMoldova, Republic ofMonaco, Principality ofMongolia, Mongolian People's RepublicMontserratMorocco, Kingdom ofMozambique, People's Republic ofMyanmarNamibiaNauru, Republic ofNepal, Kingdom ofNetherlands AntillesNetherlands, Kingdom of theNew CaledoniaNew ZealandNicaragua, Republic ofNiger, Republic of theNigeria, Federal Republic ofNiue, Republic ofNorfolk IslandNorthern Mariana IslandsNorway, Kingdom ofOman, Sultanate ofPakistan, Islamic Republic ofPalauPalestinian Territory, OccupiedPanama, Republic ofPapua New GuineaParaguay, Republic ofPeru, Republic ofPhilippines, Republic of thePitcairn IslandPoland, Polish People's RepublicPortugal, Portuguese RepublicPuerto RicoQatar, State ofReunionRomania, Socialist Republic ofRussian FederationRwanda, Rwandese RepublicSamoa, Independent State ofSan Marino, Republic ofSao Tome and Principe, Democratic Republic ofSaudi Arabia, Kingdom ofSenegal, Republic ofSerbia and MontenegroSeychelles, Republic ofSierra Leone, Republic ofSingapore, Republic ofSlovakia (Slovak Republic)SloveniaSolomon IslandsSomalia, Somali RepublicSouth Africa, Republic ofSouth Georgia and the South Sandwich IslandsSpain, Spanish StateSri Lanka, Democratic Socialist Republic ofSt. HelenaSt. Kitts and NevisSt. LuciaSt. Pierre and MiquelonSt. Vincent and the GrenadinesSudan, Democratic Republic of theSuriname, Republic ofSvalbard & Jan Mayen IslandsSwaziland, Kingdom ofSweden, Kingdom ofSwitzerland, Swiss ConfederationSyrian Arab RepublicTaiwan, Province of ChinaTajikistanTanzania, United Republic ofThailand, Kingdom ofTimor-Leste, Democratic Republic ofTogo, Togolese RepublicTokelau (Tokelau Islands)Tonga, Kingdom ofTrinidad and Tobago, Republic ofTunisia, Republic ofTurkey, Republic ofTurkmenistanTurks and Caicos IslandsTuvaluUganda, Republic ofUkraineUnited Arab EmiratesUnited Kingdom of Great Britain & N. IrelandUruguay, Eastern Republic ofUzbekistanVanuatuVenezuela, Bolivarian Republic ofViet Nam, Socialist Republic ofWallis and Futuna IslandsWestern SaharaYemenZambia, Republic ofZimbabwe

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BUDDY SCOTT: Love stems from the Father | Brazos Living | thefacts.com - Brazosport Facts

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The media continues the one-handed count of patients that seem to be cured of HIV as a man who has lived with the disease since the 1980s has been in remission for 17 months.

The story is always the samethey seem to be cured, and they get a cool nicknamein this case the City of Hope Patient, after Duarte, California, where he was treated.

The difference in this case was the treatmenta bone marrow transplant to treat blood cancer leukemia from a donor who was naturally resistant to the virus.

The most remarkable difference however, is that he is only patient cured of HIV by coincidence.

The man had developed leukemia, and took the bone marrow transplant for that reason. As it happened, the donor was resistant to HIV, and taught the mans body to create an immune response against the virus.

RELATED: Worlds Second Person Cured of HIV: 40-Year-old Man is Confirmed to Be 30 Months Virus-Free

This is also the first one who got it during the epidemic of HIV/AIDS that took so many lives.

When I was diagnosed with HIV in 1988, like many others, I thought it was a death sentence, said the City of Hope Patient. I never thought I would live to see the day that I no longer have HIV.

SIMILAR: Two Patients Make History After Essentially Being Cured of HIV Using Stem Cell Transplant

So far, only three people have been seemingly cured of human immunodeficiency virus (HIV) which weakens the bodys immune system and leads to the more severe AIDS (autoimmune deficiency syndrome) which can be lethal.

The man no longer takes antiretroviral drugs, the only treatment for HIV. A bone marrow transplant is not a likely future cure, do to it being a tricky and side-effectual procedure.

Nevertheless, all cure cases have been those where a patient is given a transplant of some kind, mostly stem cells, that contain the very rarely occurring natural immunity to the virus.

The case was reported at the AIDS 2022 conference in Montreal, Canada.

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See the original post here:
Fourth Patient Seemingly Cured of HIV Through Wild Coincidence - Good News Network

Recommendation and review posted by Bethany Smith

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announces dosing of the first patient in a company-sponsored Phase 1/2 study evaluating a cryopreserved, readily available formulation of GDA-201 for the treatment of follicular and diffuse large B cell lymphomas (NCT05296525).

We are excited to further advance the development of GDA-201, a NAM-enabled natural killer (NK) cell therapy candidate which we believe has the potential to be a new readily available, cryopreserved treatment option for cancer patients with relapsed/refractory lymphoma, said Ronit Simantov, M.D., chief medical and scientific officer of Gamida Cell. Our NK cells elicited an adaptive immune response in patients in the previous investigator-sponsored study with the fresh formulation of GDA-201, potentially leading to durable remissions. We are truly grateful for the contribution of all the participants and clinical collaborators who will allow us to continue studying GDA-201 in this multi-center open label trial.

The Phase 1 portion of the study is a dose escalation phase, designed to evaluate the safety of GDA-201, and will include patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma, marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in 63 patients comprised of two cohorts of patients with either FL or DLBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR-T or stem cell transplant.

Interest in NK cell therapies has increased in recent years as a potential alternative to current cell therapies, as NK cells have the potential to be effective in hematological and solid tumors while avoiding common safety issues, said Veronika Bachanova, M.D., Ph.D., University of Minnesota. We are particularly excited about Gamidas cryopreserved formulation of GDA-201, which has potential as a new treatment option for patients.

GDA-201 leverages Gamida Cells proprietary NAM (nicotinamide) technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity (ADCC). In an investigator-sponsored Phase 1/2 study in patients with relapsed or refractory lymphoma, treatment with the fresh formulation of GDA-201 with rituximab demonstrated significant clinical activity. Of the 19 patients with non-Hodgkin lymphoma (NHL), 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%. Two-year data on outcomes and cytokine biomarkers associated with survival data demonstrated a median duration of response of 16 months (range 5-36 months) and an overall survival at two years of 78% (95% CI, 51%91%). In this study, GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 19 patients with NHL and 16 patients with multiple myeloma. The most common Grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia. There were no incidents of cytokine release syndrome, neurotoxic events, graft versus host disease or marrow aplasia.

About NAM Technology

Our NAM-enabled technology, supported by positive Phase 3 data for omidubicel, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM, we can expand and metabolically modulate multiple cell types including stem cells and NK cells with appropriate growth factors to maintain the cells active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About GDA-201

Gamida Cell applied the capabilities of its NAM-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves ADCC and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the US and EU, which is the patient population that will be studied in the currently ongoing GDA-201 Phase 1/2 clinical trial.

For more information about GDA-201, please visit https://www.gamida-cell.com. For more information on the Phase 1/2 clinical trial of GDA-201, please visit http://www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapy candidates with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to: the timing of initiation of the expansion portion of the currently ongoing Phase 1/2 clinical trial of GDA-201, as well as the progress of, and data reported from, this clinical trial; the potentially life-saving or curative therapeutic and commercial potential of Gamida Cells product candidates (including omidubicel and GDA-201); and Gamida Cells expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cells goals, expectations, or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including statements related to: the impact that the COVID-19 pandemic could have on our business; the scope, progress and expansion of Gamida Cells clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics; and the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission (SEC) on May 12, 2022, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cells forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.

Read more:
Gamida Cell Announces Dosing of First Patient in Company-Sponsored Phase 1/2 Study of NK Cell Therapy Candidate GDA-201 - Business Wire

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Forward-Looking Statements

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Forward-looking statements are subject to significant risks and uncertainties,including those discussed in Part I Item 1A, "Risk Factors," of our AnnualReport on Form 10-K for the year ended December 31, 2021, as supplemented by thesection entitled "Risk Factors" in this Quarterly Report on Form 10-Q, thatcould cause actual results to differ materially from those projected in suchforward-looking statements due to various factors, including our ability tocontinue as a going concern; our ability to comply with Nasdaq's continuedlisting requirements; impacts resulting from or relating to the COVID-19pandemic and actions taken to contain it; anticipated use of capital and itseffects; our ability to increase the volume of our product sales; failures bythird-party payers to reimburse for our products and services or changes toreimbursement rates; our ability to continue developing existing technologiesand to develop, protect and promote our proprietary technologies; plans todevelop and perform LDTs; our ability to comply with Food and DrugAdministration ("FDA") regulations that relate to our products and to obtain anyFDA clearance or approval required to develop and commercialize medical devices;our ability to develop and commercialize additional diagnostic products andachieve market acceptance with respect to these products; our ability to competesuccessfully; our ability to obtain any regulatory approval required for ourfuture diagnostic products; or our suppliers' ability to comply with FDArequirements

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for production, marketing and post-market monitoring of our products; ourability to maintain sufficient or acceptable supplies of immunoassay kits fromour suppliers; in the event that we succeed in commercializing our productsoutside the United States, the political, economic and other conditionsaffecting other countries; changes in healthcare policy; our ability to complywith environmental laws; our ability to comply with the additional laws andregulations that apply to us in connection with the operation of ASPiRA LABS;our ability to use our net operating loss carryforwards; our ability to useintellectual property; our ability to successfully defend our proprietarytechnology against third parties; our ability to obtain licenses in the event athird party successfully asserts proprietary rights; the liquidity and tradingvolume of our common stock; the concentration of ownership of our common stock;our ability to retain key employees; our ability to secure additional capital onacceptable terms to execute our business plan; business interruptions; theeffectiveness and availability of our information systems; our ability tointegrate and achieve anticipated results from any acquisitions or strategicalliances; future litigation against us, including infringement of intellectualproperty and product liability exposure; and additional costs that may berequired to make further improvements to our laboratory operations.Company Overview

?Growth. In 2022, we have continued to grow the top line in terms of bothproduct volume and revenue. Our focus has been on OVA1plus, and, in the secondhalf of 2022, we plan to drive OVA1plus sales volume not only through our owncommercial team but also through our collaboration with BioReference Health,LLC, formerly known as BioReference Laboratories, Inc. ("BRL"). We believeAspira GenetiX and Aspira Synergy should also contribute to increased revenue.In addition, positive trends in the tenure of our sales professionals shouldlead to volume growth. As of June 30, 2022, 67% of our sales professionals hadbeen with us for more than three months and 58% had been with us for more thansix months. We aim not only to increase the number of physicians ordering forthe first time but also to increase repeat orders from existing physiciancustomers.

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In parallel to building our OVA platform offering and our commercial deployment,we have been working on several key publications and product extensions.The OVAWatch manuscript, "Analytical Validation of a Deep Neural NetworkAlgorithm for the Detection of Ovarian Cancer," has been published online in theJournal of Clinical Oncology Clinical Cancer Informatics. The Company hasprepared an application for a Proprietary Laboratory Analyses code with the

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American Medical Association for the OVAWatch test to distinguish it fromOVA1plus with an expectation that Novitas and other payers will apply theOVA1plus Centers for Medicare & Medicaid Services fee to OVAWatch, ensuringconsistent coverage and pricing for both OVA products.COVID-19 Pandemic

In December 2019, a novel strain of coronavirus was reported to have surfaced inWuhan, China. The novel coronavirus has since spread to over 100 countries,including every state in the United States. In March 2020, the World HealthOrganization declared COVID-19, the disease caused by the novel coronavirus, apandemic, and the United States declared a national emergency with respect tothe coronavirus outbreak. This outbreak has severely impacted global economicactivity, and many countries and many states in the United States have reactedto the outbreak by instituting quarantines, mandating business and schoolclosures and restricting travel periodically throughout the pandemic. In orderto reduce the impact of limitations on visiting physician offices due toclosures and quarantines, we implemented other mechanisms for reachingphysicians such as virtual sales representative meetings, Key Opinion Leaderpresentations, and increased digital sales and marketing. Patient enrollment forour planned clinical research studies has been slower than originally planneddue to the impact of clinic closures and patients not seeking medical care insome states, which has led to delays in the completion of such studies.Given the potential for future resurgences of COVID-19 cases and the variety offederal and state actions taken to contain them, we are unable to estimate thepotential future impact of the COVID-19 pandemic on our business, results ofoperations or cash flows as of the date of the filing of this Form 10-Q.In addition, as of the date of the filing of this Form 10-Q, we haveapproximately four months of reagents, one of our key testing supplies, instock, depending on volume of tests performed, and we are working with themanufacturer to ensure a consistent supply over the next six months. Aspreviously disclosed, we have put in place staffing and reagent contingencyplans to ensure there is no down time at our lab. We believe the lab couldcontinue to operate in the event any isolated infection were to impact a portionof the workforce. The full impact of the COVID-19 pandemic continues to evolveas of the date of the filing of this Form 10-QCritical Accounting Policies and Estimates

Our product revenue is generated by performing diagnostic services using ourOVA1, OVERA, OVA1plus or Aspira GenetiX tests, and the service is completed uponthe delivery of the test result to the prescribing physician. The entiretransaction price is allocated to the single performance obligation contained ina contract with a patient. Under ASC Topic 606, Revenue from Contracts withCustomers, all revenue is recognized upon completion of the OVA1, OVERA,OVA1plus or Aspira GenetiX test and delivery of test results to the physicianbased on estimates of amounts that will ultimately be realized. In determiningthe amount of revenue to be recognized for a delivered test result, we considerfactors such as payment history and amount, payer coverage, whether there is areimbursement contract between the payer and us, and any developments or changesthat could impact reimbursement. These estimates require significant judgment bymanagement. For OVA1, OVERA, OVA1plus and Aspira GenetiX tests, we also reviewour patient account population and determine an appropriate distribution ofpatient accounts by payer (i.e., Medicare, patient pay, other third-party payer,etc.) into portfolios with similar collection experience. When evaluated forcollectability, this results in a materially consistent revenue amount for suchportfolios as if each patient account were evaluated on an individual contractbasis.

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Results of Operations - Three Months Ended June 30, 2022 Compared to ThreeMonths Ended June 30, 2021The selected summary financial and operating data of the Company for the threemonths ended June 30, 2022 and 2021 were as follows:

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Results of Operations - Six Months Ended June 30, 2022 Compared to Six MonthsEnded June 30, 2021The selected summary financial and operating data of the Company for the sixmonths ended June 30, 2022 and 2021 were as follows:

Product Revenue. Product revenue was $3,853,000 for the six months ended June30, 2022, compared to $3,136,000 for the same period in 2021. Revenue for ASPiRALABS is recognized when the OVA1, OVERA, or OVA1plus test is completed based onestimates of what we expect to ultimately realize. The 23% product revenueincrease is primarily due to an increase in OVA1 test volume compared to theprior year, partially offset by a modest decrease in AUP, which decreased from$377 in the first half of 2021 to $376 in the first half of 2022.Medicaid represents approximately 11.8% of volume in the six months ended June30, 2022, at an AUP of $90. This is compared to 11.7% of volume in the firsthalf of 2021, at an AUP of $90. Our OVA1plus AUP without Medicaid was $415 forthe six months ended June 30, 2022, compared to $417 for the same period in2021.The number of Product tests performed increased 23% to 10,257 during the sixmonths ended June 30, 2022, compared to 8,328 Product tests for the same periodin 2021. This increase was due to increased access to provider offices andincreased investment in our current commercial channel.Genetics Revenue. Genetics revenue was $106,000 for the six months ended June30, 2022, compared to $159,000 for the same period in 2021. Revenue for AspiraGenetiX is recognized when the Aspira GenetiX test is completed based onestimates of what we expect to ultimately realize. The 33% genetics revenuedecrease is primarily due to decreased volumes as compared to the same period in2021, in addition to the AUP decreasing to $424 from $483 from the same periodin 2021.

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Liquidity and Capital ResourcesWe plan to continue to expend resources selling and marketing OVA1, OVERA,OVA1plus and Aspira GenetiX and developing additional diagnostic tests andservice capabilities. We plan to launch our next generation ovarian cancer riskassessment test, OVAWatch, in the second half of 2022.We have incurred significant net losses and negative cash flows from operationssince inception, and as a result have an accumulated deficit of approximately$489,239,000 as of June 30, 2022. We also expect to incur a net loss andnegative cash flows from operations for 2022. Working capital levels may not besufficient to fund operations as currently planned through the next twelvemonths, absent a significant increase in revenue over historicrevenue or additional financing. Given the above conditions, there issubstantial doubt about our ability to continue as a going concern.We expect to raise capital through sources that may include public or privateequity offerings, debt financings, collaborations, licensing arrangements,grants and government funding and strategic alliances. However, additionalfunding may not be available when needed or on terms acceptable to us. If we areunable to obtain additional capital, we may not be able to continue sales andmarketing, research and development, or other operations on the scope or scaleof current activity, and that could have a material adverse effect on ourbusiness, results of operations and financial condition.As discussed in Note 2 to the condensed consolidated financial statements, inMarch 2016, we entered into a loan agreement (as amended on March 7, 2018 andApril 3, 2020, the "DECD Loan Agreement") with the State of ConnecticutDepartment of Economic and Community Development (the "DECD"), pursuant to whichwe may borrow up to $4,000,000 from the DECD.

As discussed in Note 3 to the condensed consolidated financial statements, onFebruary 8, 2021, the Company completed a public offering (the "2021 Offering")resulting in net proceeds of approximately $47,720,000, after deductingunderwriting discounts and offering expenses. There was a change in estimate inthe third quarter of 2021 in the amount of $138,000 relating to an expensereversal of offering costs.

In connection with a private placement offering of common stock and warrants wecompleted in May 2013, we entered into a stockholders agreement which, amongother things, gives two of the primary investors in that offering the right toparticipate in any future equity offerings by the Company on the same price andterms as

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Off-Balance Sheet ArrangementsAs of June 30, 2022, we had no off-balance sheet arrangements that arereasonably likely to have a current or future material effect on our condensedconsolidated financial condition, results of operations, liquidity, capitalexpenditures or capital resources.

Edgar Online, source Glimpses

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Recommendation and review posted by Bethany Smith

Although once ridiculed, Lovelock's theory that the earth's natural cycles are living, self-regulating organisms underpins much of climate science

Although most of the world knew James Lovelock as an independent scientist and originator of the Gaia hypothesis, he had a slightly different take. Im not a scientist really. Im an inventor or a mechanic. Its a different thing. The Gaia theory is just engineering written very large indeed, hetold theGuardianin 2020.

Regardless of labels, theres no denying the significant influence of Lovelock, whodied July 26on his 103rdbirthday. Although many of his discoveries and ideas on subjects ranging from cryonics to chlorofluorocarbons, and climate to nuclear power were controversial, most gained acceptance as the world caught up.

Named for the Greek Earth goddess, hisGaia theory developed with evolutionary biologist Lynn Margulis during the 1960s when he was working for NASAs moon and Mars programs saw the world with its natural cycles as a living, self-regulating organism. When one cycle is knocked out of equilibrium, others work to restore balance.

At the time, many prominent scientists ridiculed the hypothesis, but its continued to gain acceptance because it helps to explain the chemical and physical balances in air, land and water that make life possible. It underpins much of climate science. The idea isnt that Earth is conscious of these processes; just that the cycles work together to keep the planet healthy and able to support life.

Its similar to the ways in which many Indigenous Peoples worldwide view the living Earth. Everything is interconnected. He understood that human activities that destroy rainforests and reduce biodiversity, for example, hinder Gaias ability to minimize the impacts of runaway greenhouse gases in the atmosphere.

Lovelock wasnt afraid to change his views in the face of evolving evidence, but he also refused to ever soften his message, something I learned from interviewing him several times.

His research revealed the effects of CFCs on the ozone layer, and he warned that burning fossil fuels was changing the climate before these issues were on most peoples radar. His electron capture device, invented in the late 1960s, detected rising CFC levels in the atmosphere as well as pollutants like PCBs in air, soil and water and led to the discovery that this was causing ozone depletion. That eventually resulted in theMontreal Protocol on Substances that Deplete the Ozone Layer, adopted in 1987 by all countries helping the ozone layer to recover and preventing millions of cases of skin and other cancers and eye cataracts.

Like many who clearly see the environmental predicaments weve created, Lovelock wasnt always optimistic, despite his knowledge of the many available and emerging solutions. I would say the biosphere and I are both in the last 1% or our lives, he told theGuardiantwo years ago.

Lovelock, who started out in medicine, even thought pandemics such as COVID-19 could be related to planetary self-regulation: I could easily make you a model and demonstrate that as the human population on the planet grew larger and larger, the probability of a virus evolving that would cut back the population is quite marked.

He said opposition to the Gaia hypothesis surprised him: Im wondering to what extent you can put that down to the coal and oil industries who fought against any kind of message that would be bad for them.

As for solutions to the climate crisis, he advocated for technologies that havent always been popular, including nuclear energy and Edward Tellers suggestion of a sunshade in a heliocentric orbit that would diffuse a few percent of sunlight from the Earth. However, he cautioned, I dont think we should start messing about with the Gaia system until we know a hell of a lot more about it. It is beginning to look as if renewable energy wind and solar if properly used, may be the answer to the energy problems of humanity.

James Lovelock continued to work, write and speak until his final days. My main reason for not relaxing into contented retirement is that like most of you I am deeply concerned about the probability of massively harmful climate change and the need to do something about it now, he said.

Lovelock may have left Gaia, but the knowledge he left endures and is essential to understanding our place, predicament and future.

David Suzuki is a scientist, broadcaster, author and co-founder of the David Suzuki Foundation. Written with contributions from David Suzuki Foundation Senior Writer and Editor Ian Hanington.

@nowtoronto

David Suzuki

David Suzuki is a scientist, broadcaster, author and cofounder of the David Suzuki Foundation.

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David Suzuki: Gaia theorist James Lovelock was always ahead of the times - NOW Toronto

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Recommendation and review posted by Bethany Smith

Immediate cord clamping (ICC), within a few seconds after birth, became routine in the latter half of the 20th century, as part of a tranche of medical birth-related interventions that collectively, undoubtedly improved maternal and neonatal survival and outcomes [1]. The trend to ICC (within 15-20 seconds after birth) was partly driven by some early studies suggesting that the most benefit in terms of blood volume is achieved within this time frame [2], and that deferred cord clamping (DCC) increased rates of polycythemia and jaundice [1]. It may also have been partly driven by increased rates of operative deliveries and consequent pressure to minimize surgical times, as well as the increased availability and effectiveness of neonatal resuscitation. Furthermore, ICC was proposed as a means to reduce the risk of maternal exposure to fetal blood group antigens at a time (before RhD immunoprophylaxis) when hemolytic disease of the fetus and newborn was far more common than it is now.

Formal evidence that ICC was beneficial was never sought, and recent research summarized in systematic reviews [3-6] has suggested that it may be harmful when compared with DCC for various intervals from 30 seconds until when the cord stops pulsating (defined in some studies as physiological cord clamping). ICC before the onset of breathing exposes the newborn baby to a period of significantly restricted cardiac function, whereas DCC until after the onset of breathing (which often does not occur until late in the first minute after birth) may mean that the expanding pulmonary circulation is able to fill with blood from the placenta, rather than by reverse flow across the ductus arteriosus [7]. This may improve left ventricular preload and stabilize pressures and flows in major vessels [7].

In addition, when cord clamping is deferred, babies may receive a transfusion of blood from the umbilical cord and placenta. A recent systematic review demonstrated that DCC in preterm babies improves peak hematocrit in the first week by 2.7% (95% confidence intervals (CI) 1.88-3.52) and reduced the proportion of babies receiving any subsequent blood transfusion (RD: -0.07, 95%CI -0.11 to -0.04) [6]. Some studies have found a weight increase in the first two minutes after birth when the cord is not clamped, supporting the hypothesis of placental transfusion [8]. Yet, recent evidence shows that placental transfusion may not always occur (Conference abstract: Vijayaselvi R, Abraham A, Kumar M, Kuruvilla A, Mathews J, Duley L. Measuring Umbilical Flow and Placental Transfusion for Preterm Births: Weighing Babies at 33-36 Weeks Gestation with Cord Intact. 1st Congress of Joint European Neonatal Societies; 2015).

The relative roles of cardiovascular stabilization at birth versus placental transfusion in improving outcomes have not been established. Understanding the contributions of these two mechanisms has significant implications for research and practice: for example, if the size of placental transfusion is more important, then prescribing a top-up transfusion soon after birth for babies with lower than average hemoglobin (who are known to be at higher risk of various adverse outcomes) [9] may be justified, especially for the babies for whom DCC has been precluded by maternal or fetal conditions. These include significant maternal bleeding, and monochorionic twins, where deferred cord clamping in the first twin could lead to one twin losing blood to the other. However, if it is the effects on improving cardiovascular stability in the first minutes (with consequential benefits for cardiorespiratory function and reducing severity of illness during the subsequent neonatal intensive care unit (NICU) stay), regardless of the magnitude of transfusion, then early top-up transfusion is unlikely to be helpful.

Observational studies suggest that exposure to blood transfusion itself is harmful to preterm babies, increasing the risk of adverse outcomes [10]. However, this suggestion has not been supported by the small number (to date) of randomized controlled trials of blood (red cell) transfusion thresholds [11-14]. It is unlikely to be the means by which DCC reduced deaths in the largest trial to date of deferred cord clamping in preterm babies, the Australian Placental Transfusion Study (APTS), and in the most recent systematic review on this, because neither showed a difference in rates of other adverse outcomes [6,15].

Another possibility is that it is the umbilical cord blood stem cells received by the baby are the main reason for the observed benefits to both survival and reduced requirement for later blood transfusion [16]. Umbilical cord blood has been demonstrated to be such a good contributor to hematopoiesis that it is a recognized stem cell resource for pediatric and adult hematopoietic stem cell transplant [17]. In addition, umbilical cord blood is a potential regenerative and immunomodulatory agent for a variety of clinical conditions [18], so in this case, the extent of placental transfusion would be critical to the improvement of outcomes, and transfusion with adult red cells would not suffice. There are no established methods to quantify the contribution of umbilical cord stem cells to placental transfusion. However, a larger volume of placental transfusion results in the baby receiving more nucleated cells [19], including more umbilical cord stem cells.

Discerning whether these effects (initial enhanced cardiovascular stability leading to early and sustained reduction in severity of illness or volume of placental transfusion) appear to be the main driver of improved outcomes is likely to contribute to practice change, as well as to informing the design of future research studies into methods to improve outcomes of high-risk newborn babies and reduce their transfusion dependence.

The causal mechanisms of reduced transfusion requirements found in DCC relative to ICC are yet to be resolved. The aim of the study is to address the question; In preterm infants (P) does DCC (I) compared to ICC (C) reduce dependence on red cell transfusion via enhanced cardiovascular stability (mediator 1, M1) or via an increased volume of placental transfusion (M2).

The study is a nested retrospective study, called the Transfusions in the APTS Newborns Study (TITANS) (study registration: ACTRN12620000195954), of the cohort of babies who were enrolled and randomly assigned to ICC or DCC in the Australian and New Zealand (NZ) sites for APTS (study registration: ACTRN12610000633088). This design has been developed to take advantage of the comprehensive dataset already collected for APTS, and because there is currently no suitable prospective study that could address the same research questions in such a large group of participants.

Babies had been considered eligible for APTS if obstetricians or maternal-fetal medicine specialists anticipated that delivery would occur before 30 weeks of gestation. Exclusion criteria included fetal hemolytic disease, hydrops fetalis, twin-twin transfusion, genetic syndromes, and potentially lethal malformations. Further details are available in the original APTS publication [15]. In the present TITANS analysis, we will also exclude any baby with a diagnosis of hemolytic anemia or aplastic/hypoplastic anemia.

There were 1401 babies enrolled for APTS from the 13 Australian and 5 NZ hospital sites [15]. APTS data was provided to the TITANS team on 31 July, 2020. It is planned to collect additional data from Australian and NZ APTS sites using a customised, secure web-based database application (REDCap) [20], which is maintained by the University of Sydney, Sydney, Australia. Data will be obtained from source documents (patient hospital records and laboratory reports) using the electronic data collection application from each study site. The individual participant data collected will correspond to the minimum data required to answer the research questions. Baby identification (ID) and other babies details from APTS will be used to re-identify participants and link them to hospital records. Identified data will be collected, in order to allow linkage between the data newly collected from patient records and hospital laboratories and the existing APTS dataset. The data will be checked with respect to range, internal consistency, consistency with published reports and missing items. After data cleaning and analysis, data will be stored in re-identifiable form, with each participants data being identified with the same study numbering system as used for the APTS study.

We will combine the data already extracted, stored and cleaned from APTS with the additional data obtained from study sites for each participating baby, to determine which factors are most influential in reducing transfusion requirements. The specific objectives are, after adjustment for prior risk factors (listed below), to determine:

1.Whether the effect of the intervention (cord clamping) on the outcome (blood transfusions) is mediated by placental transfusion (measured by hematocrit (Hct)) as seen in Figure 1 (a, c) following the causal path X M1 Y, where X is the intervention, ICC or DCC, Y is the outcome, mediator M1 is placental transfusion, and M2 is initial severity of illness stability

2.Whether the effect of the intervention (cord clamping) on the outcome (blood transfusions) is mediated by initial severity of illness (respiratory support, sampling line yes/no and total duration number, blood pressure, cumulative blood sample volume) as seen in Figure 1 (b, c) following the causal path X M2 Y

3.Whether the effect of cord clamping intervention on the outcome (blood transfusions) is driven by multiple mediators (placental transfusion and initial severity of illness) as seen in Figure 1 (c)

4.Whether cording clamping intervention (ICC or DCC) has a direct effect on the outcome after accounting for the mediators as seen in all panels of Figure 1: X Y.

The protocol was approved by the Northern Sydney Local Health District Human Research Ethics Committee in November 2019 (Version 3.0, Reference 2019/ETH12819), the Mater Misericordiae Ltd Human Research Ethics Committee (Version 1.0, Reference HREC/MML/56247), the Mercy Health Human Research Ethics Committee (Version 2.0, Reference 2020-078), and the Southern Health and Disability Ethics Committee (Version 1.0, Reference 19/STH/195). The ethics committees have granted a waiver of consent. The study is conducted in accordance with the National Health and Medical Research Council Statement on Ethical Conduct in Research Involving Humans.

Intervention

The intervention consisted of either immediate or delayed cord clamping (as assigned in APTS). Immediate clamping was defined as clamping the cord within 10 seconds of delivery. Delayed clamping was defined as clamping the cord at least 60 seconds after delivery, with the infant held as low as possible, below the introitus or placenta, and with no palpation of the cord. Variations in the protocol were allowed if they would aid the mother, baby, or both. If the baby was non-vigorous (heart rate <100 beats per minute, low muscle tone, or lack of breathing, or crying), clinicians were allowed to break protocol using their discretion. Cord milking was not part of the protocol for either intervention. Further details may be sourced from the original APTS publication [15].

Outcomes

The primary outcome is the proportion of babies receiving red cell transfusion (for restoration of hemoglobin or blood volume). The secondary outcomes are number of transfusions per baby, cumulative transfusion volume (mL/kg) per baby, and primary reasons for each transfusion.

Putative Mediators

M1: Indicators of placental transfusion to be assessed will be hematocrit (on admission, highest on the first day, highest in the first week collected before any postnatal transfusion).

M2: Indicators of initial severity of illness to be assessed will be cumulative blood sample volume collected throughout hospital stay (number of blood tests multiplied by hospitals usual sample volume for each type of test), sampling line (umbilical arterial line or peripheral arterial line) - yes/no and total duration, mechanical ventilation or inspired O2, and blood pressure.

Sensitivity Analyses (For the Primary Outcome Analysis Only)

Sensitivity analyses will adjust for the following variables: gender, birth <27 weeks vs. 27 weeks, method of delivery (vaginal versus cesarean), intraventricular hemorrhage (IVH) (yes/no and grade III/IV yes/no), surgery for patent ductus arteriosus (PDA), necrotizing enterocolitis (NEC), and sodium in the first 24 hours of life. We will also test model assumptions relating to sequential ignorability and post-randomization confounding (discussed further in the data analysis plan).

Potential Confounders (Covariates)

The following covariates may be used for adjustment in the analysis: gestational age at randomization before birth and any oral iron supplement pre-transfusion.

Timing of Assessments

Putative mediating variables will only be analyzed if they have been measured before the outcome and will be excluded if there is not adequate time and date information available. If the multiple mediator model is applied, careful consideration of timing information will be evaluated. If there is insufficient empirical information to conclude the causal ordering of mediators (M1 causes M2), we will adjust our analytic approach (as discussed in the analysis plan) and discuss any limitations.

Data Analysis Plan

The analysis will include all babies who were initially randomized in the APTS trial for whom we were able to obtain the relevant data and be based on intention-to-treat. All statistical analyses will be conducted in R version 4.1.3 (2022-03-10; R Foundation for Statistical Computing, Vienna, Austria). Descriptive characteristics for continuous data will be presented as means or medians, as appropriate, and categorical data will be presented as frequencies and percentages.

A model-based inference approach will be applied to estimate the average causal mediation effect (ACME), average direct effect (ADE), and the average total effect as recommended [23-25]. This approach will be applied with the R mediation package [26]. We will initially fit two models, one model with mediation as the dependent variable and intervention as the independent variable (mediator model), and a second model with the outcome as the dependent variable, and both mediation and intervention as independent variables (outcome model). To account for the clustering of multiples, estimates will be calculated with generalized estimating equations with a compound symmetric correlation structure to account for within subject correlations. Depending on the outcome (binary, count, skew) these will be modelled with the appropriate family and link functions.

A counterfactual framework will be applied to the mediator and outcome models to simulate the values of the mediator and outcome to estimate the potential values of the mediator. This process is used to estimate the ACME, ADE, and average total effects; 95%CI will be estimated with 1000 bootstrap simulations.

We will apply single mediator models on both placental transfusion variables and initial severity of illness variables if mediators are statistically independent, as seen in Table 1. Independence will be tested using linear regression and any appropriate link functions. If both mediators are not statistically independent, we will investigate the possibility of multiple mediator models, which require an expanded framework for analysis [21]. Here we assume that initial severity of illness is causally related to placental transfusion. For this process, we will use the method developed by Imai and Yamamoto [21] to estimate the ACME and ADE. Following this, 95%CI will be estimated with 1000 bootstrap simulations. If theoretical and empirical timing data and sensitivity analyses suggest that M1 and M2 have non-causal correlation and may be affected by an unmeasured latent mediator, we will adjust our approach to estimate interventional direct and path-specific indirect effects [27,28].

Sensitivity analyses have been limited to a set of biologically plausible and clinically meaningful groups that will be explored by including them for adjustment with covariates, and with the introduction of interaction terms if appropriate. Missing data will be described, reasons for missing data will be explored, and the impact of missing data on conclusions about the treatment effect on the primary outcome will also be explored where possible (e.g., using sensitivity analyses and multiple imputation techniques).

Methodological Assumptions

The causal mediation approach assumes sequential ignorability: that the treatment effect on the outcome is not confounding and that the mediator effect on the outcome is not confounded. As treatment was randomly allocated to neonates, we will assume that the treatment-mediator relationship is not confounded. However, the mediator itself has not been randomized. Thus, unknown confounders may be driving a spurious effect in the mediator-outcome relationship. We will employ additional sensitivity analyses to estimate whether any mediation effects are sensitive to the violation of the assumption of sequential ignorability. To test the possibility of unmeasured confounders we will examine the correlation between residuals in the mediator model and the outcome model. If there is no correlation this would suggest there is no unmeasured confounding, if there is correlation between the residuals, an unmeasured mediator may be affecting both the measured mediator and the outcome. We will apply the method developed by Imai et al. andTingley et al. [23,26] that uses sensitivity analyses to evaluate if the ACME estimate is sensitive to unmeasured confounding.

Post-randomization confounders are dependent on the treatment allocated, affect both mediator and outcome, and can corrupt the mediation estimate. In the context of the present trial, it is possible that non-adherence to the intervention is a post-randomization confounder. We are analyzing our data based on intention to treat principles; however, a sensitivity analysis based on the actual time of cord clamping to assess the influence of non-adherence with the treatment protocol on our estimates may be performed.

Blood transfusions of neonates have been associated with a number of serious adverse outcomes [29]. Nevertheless, there are few evidence-based methods to reduce transfusion exposure [30]. The APTS study found that DCC was associated with a statistically significant reduced need for red cell transfusions by about 10% compared to ICC [15]. However, the mechanism remains unclear.

The study will, at a minimum, provide further information that should increase clinicians understanding of the pathways by which DCC (or other methods to accomplish placental transfusion) results in beneficial patient outcomes. Since one of the main barriers to implementation is lack of understanding about the mechanisms by which such a simple practice change should have such dramatic effects, this should improve adherence to recommendations to defer cord clamping for most babies, thereby reducing mortality and transfusion incidence.

By elaborating on the mechanisms, it may also provide good evidence for how other routine neonatal intensive care practices and interventions affect likelihood of needing to transfuse. Better understanding of these effects may lead to other testable hypotheses or improvements in other aspects of practice, further reducing transfusion exposure and improving other outcomes.

Potential limitations of the study include the dependence on some routinely collected clinical data, which were not collected at the time by the original study according to predefined research definitions. However, we have no reason to think that potential problems of data quality would have been influenced by study group allocation and so do not anticipate that this will be a source of bias.

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Protocol for a Nested, Retrospective Study of the Australian Placental Transfusion Study Cohort - Cureus

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Market (5th Edition) report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035.

Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, LibmeldyT, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors).

Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain.

In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods.

For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics.

Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation.

Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

3.1. Context and Background

3.2. Evolution of Gene Therapies

3.3. Classification of Gene Therapies

3.3.1. Somatic and Germline Gene Therapies

3.3.2. Ex Vivo and In Vivo Gene Therapies

3.4. Routes of Administration

3.5. Mechanism of Action

3.6. Overview of Gene Editing

3.6.1. Evolution of Genome Editing

3.6.2. Applications of Genome Editing

3.6.3. Available Genome Editing Techniques

3.7. Advantages and Disadvantages of Gene Therapies

3.7.1 Ethical and Social Concerns Related to Gene Therapies

3.7.2. Constraints and Challenges Related to Gene Therapies

3.7.3. Therapy Development Concerns

3.7.4. Manufacturing Concerns

3.7.5. Commercial Viability Concerns

4. GENE DELIVERY VECTORS

4.1. Chapter Overview

4.2. Viral and Non-Viral Methods of Gene Transfer

4.3. Viral Vectors for Genetically Modified Therapies

4.4. Types of Viral Vectors

4.5. Types of Non-Viral Vectors

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

5.1. Chapter Overview

5.2. Regulatory Guidelines in North America

5.3. Regulatory Guidelines in Europe

5.4. Regulatory Guidelines in Asia-Pacific

5.5. Reimbursement Scenario

5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW

6.1. Chapter Overview

6.2. Gene Therapy Market: Clinical and Commercial Pipeline

6.3. Gene Therapy Market: Early-Stage Pipeline

6.4. Gene Therapy Market: Special Drug Designations

6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)

7. COMPETITIVE LANDSCAPE

7.1. Chapter Overview

7.2. Gene Therapy Market: List of Developers

7.3. Key Players: Analysis by Number of Pipeline Candidates

8. MARKETED GENE THERAPIES

8.1. Chapter Overview

8.2. Gendicine (Shenzhen Sibiono GeneTech)

8.3. Oncorine (Shanghai Sunway Biotech)

8.4. Rexin-G (Epeius Biotechnologies)

8.5. Neovasculgen (Human Stem Cells Institute)

8.6. Imlygic (Amgen)

8.7. Strimvelis (Orchard Therapeutics)

8.8. LuxturnaT (Spark Therapeutics)

8.9. ZolgensmaT (Novartis)

8.10. Collategene (AnGes)

8.11. ZyntelgoT (bluebird bio)

8.12. LibmeldyT (Orchard Therapeutics)

9. KEY COMMERCIALIZATION STRATEGIES

9.1. Chapter Overview

9.2. Successful Drug Launch Strategy: ROOTS Framework

9.3. Successful Drug Launch Strategy: Product Differentiation

9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product

9.5. List of Currently Approved Gene Therapies

9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers

9.6.1. Strategies Adopted Before Therapy Approval

9.6.1.1. Participation in Global Events

9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms

9.6.1.3. Indication Expansion

9.6.2. Strategies Adopted During/Post Therapy Approval

9.6.2.1. Geographical Expansion

9.6.2.2. Participation in Global Events

9.6.2.3. Patience Assistance Programs

9.6.2.4. Awareness through Product Websites

9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms

9.7. Concluding Remarks

10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES

10.1. Chapter Overview

10.2. Lumevoq (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

10.3. OTL-103

10.4. PTC-AADC

10.5. BMN 270

10.6. rAd-IFN/Syn3

10.7. beti-cel

10.8. eli-cel

10.9. lovo-cel

10.10. SRP-9001

10.11. EB-101

10.12. ProstAtak

10.13. D-Fi

Continued here:
Gene Therapy Market Research Report 2022 - ResearchAndMarkets.com - Business Wire

Recommendation and review posted by Bethany Smith

Published: Published Date - 09:21 PM, Wed - 10 August 22

Hyderabad: This concept may seem quite fictional and even futuristic. However, this is what geneticists worldwide through gene therapy are pursuing, while trying to find cure for a wide range of diseases that challenge modern medicine including cancers, heart diseases, diabetes, haemophilia, AIDS, genetic disorders, among others.

Gene therapy involves altering the genes inside the cells of the human body, in order to treat or prevent the disease progression. Essentially, geneticists worldwide are exploring ways to utilise gene therapy to alter genetic composition of cells that are responsible for causing diseases and in the process find a long term cure for diseases. The potential to unlock the cure for a wide range of diseases has become a major driving force for researchers and pharma giants worldwide to focus their energies and resources on gene therapy.

So what exactly is gene and gene therapy?

The Gene Therapy Advisory and Evaluation Committee (GTAEC), which monitors clinical trials across India on gene therapies, defines Gene is the most basic and functional unit of heredity and inheritance and consists of a specific sequence of nucleotides in DNA or RNA located on chromosomes that encodes for specific proteins. The human genome comprises more than 20,000 genes. Gene therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

The genetic material that has to be introduced to the diseased cell is done through a vector, whch is usually a virus. Viruses are the preferred vectors or vehicles as they are adaptable and efficient in delivering genetic material, the GTAEC, said.

While worldwide major pharmaceutical companies are developing gene therapies for treatment of single gene defects like haemophilia and muscular dystrophy, the Department of Biotechnology (DBT), Government of India, Tata Memorial Hospital, Mumbai and IIT-Mumbai have collaborated to start clinical trials of gene therapy on cancer in India.

Gene therapy in cancer:

In the last few years, CAR- (Chimeric Antigen Receptor) T therapy, a form of gene therapy has emerged as a breakthrough treatment for cancer, especially for leukemia, lymphoma (cancer of the lymphatic system) and multiple myeloma or the cancer of the plasma cells.

The CAR-T cells are genetically engineered in a laboratory and they bind with the cancer cells and kill them. The therapy is available in a few cancer research centres (on clinical trials basis) in US and cost of treatment ranges anywhere from Rs 3 crore to Rs. 4 crore.

To reduce treatment costs, promote and support development CAR-T cell technology against cancers, for the first time in India, Biotechnology Industry Research Assistance Council (BIRAC), established by DBT, Tata Memorial Hospital and IIT Bombay, have launched clincal trials of CAR-T gene therapy to treat cancers. The CAR-T cells were designed and manufactured at Bioscience and Bioengineering (BSBE) department of IIT Bombay. The gene therapy study on cancers is in early phase clinical trials at Tata Memorial in Mumbai.

Regulation of gene therapy:

Realising the potential of gene therapies in treating complex diseases, the GOI is providing financial and even technical guidance to researchers through ICMR, DBT and DST. To ensure gene therapies are introduced in India and clinical trials for gene therapies are performed in an ethical, scientific and safe manner, the ICMR has also framed National Guidelines for Gene Therapy Product Development and Clinical Trials document.

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Health and Tech: The promise of gene therapy to cure cancers - Telangana Today

Recommendation and review posted by Bethany Smith

DUBLIN, Aug. 10, 2022 /PRNewswire/ -- The "Stem Cell Therapy Global Market Opportunities And Strategies To 2031" report has been added to ResearchAndMarkets.com's offering.

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The global stem cell therapy market reached a value of nearly $4,019.6 million in 2021, having increased at a compound annual growth rate (CAGR) of 70.9% since 2016. The market is expected to grow from $4,019.6 million in 2021 to $10,600.2 million in 2026 at a rate of 21.4%. The market is then expected to grow at a CAGR of 11.4% from 2026 and reach $18,175.4 million in 2031.

Growth in the historic period in the stem cell therapy market resulted from rising prevalence of chronic diseases, a rise in funding from governments and private organizations, rapid growth in emerging markets, an increase in investments in cell and gene therapies, surge in healthcare expenditure, and an increase in pharmaceutical R&D expenditure. The market was restrained by low healthcare access in developing countries, limited reimbursements, and ethical concerns related to the use of embryonic stem cells in the research and development.

Going forward, increasing government support, rapid increase in the aging population, rising research and development spending, and increasing healthcare expenditure will drive market growth. Factors that could hinder the growth of the market in the future include high cost of stem cell therapy, stringent regulations imposed by regulators, and high cost of storage of stem cells.

The stem cell therapy market is segmented by type into allogeneic stem cell therapy and autologous stem cell therapy. The autologous stem cell therapy segment was the largest segment of the stem cell therapy market segmented by type, accounting for 100% of the total in 2021.

The stem cell therapy market is also segmented by cell source into adult stem cells, induced pluripotent stem cells, and embryonic stem cells. The induced pluripotent stem cells was the largest segment of the stem cell therapy market segmented by cell source, accounting for 77.2% of the total in 2021. Going forward, the adult stem cells segment is expected to be the fastest growing segment in the stem cell therapy market segmented by cell source, at a CAGR of 21.7% during 2021-2026.

Story continues

The stem cell therapy market is also segmented by application into musculoskeletal disorders and wounds & injuries, cancer, autoimmune disorders, and others. The cancer segment was the largest segment of the stem cell therapy market segmented by application, accounting for 49.7% of the total in 2021. Going forward, musculoskeletal disorders and wounds & injuries segment is expected to be the fastest growing segment in the stem cell therapy market segmented by application, at a CAGR of 22.1% during 2021-2026.

The stem cell therapy market is also segmented by end-users into hospitals and clinics, research centers, and others. The hospitals and clinics segment was the largest segment of the stem cell therapy market segmented by end-users, accounting for 66.0% of the total in 2021. Going forward, hospitals and clinics segment is expected to be the fastest growing segment in the stem cell therapy market segmented by end-users, at a CAGR of 22.0% during 2021-2026.

Scope:

Markets Covered:

By Type: Allogeneic Stem Cell Therapy; Autologous Stem Cell Therapy

By Cell Source: Adult Stem Cells; Induced Pluripotent Stem Cells; Embryonic Stem Cells

By Application: Musculoskeletal Disorders and Wounds & Injuries; Cancer; Autoimmune Disorders; Others

By End-Users: Hospitals And Clinics; Research Centers; Others

Key Topics Covered:

1. Stem Cell Therapy Market Executive Summary

2. Table of Contents

3. List of Figures

4. List of Tables

5. Report Structure

6. Introduction

7. Stem Cell Therapy Market Characteristics

8. Stem Cell Therapy Trends And Strategies

9. Impact Of Covid-19 On Stem Cell Therapy Market

10. Global Stem Cell Therapy Market Size And Growth

11. Global Stem Cell Therapy Market Segmentation

12. Stem Cell Therapy Market, Regional And Country Analysis

13. Asia-Pacific Stem Cell Therapy Market

14. Western Europe Stem Cell Therapy Market

15. Eastern Europe Stem Cell Therapy Market

16. North America Stem Cell Therapy Market

17. South America Stem Cell Therapy Market

18. Middle East Stem Cell Therapy Market

19. Africa Stem Cell Therapy Market

20. Stem Cell Therapy Global Market Competitive Landscape

21. Stem Cell Therapy Market Pipeline Analysis

22. Key Mergers And Acquisitions In The Stem Cell Therapy Market

23. Stem Cell Therapy Market Opportunities And Strategies

24. Stem Cell Therapy Market, Conclusions And Recommendations

25. Appendix

Companies Mentioned

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Stem Cell Therapy Global Market Report 2022: Rising Research and Development Spending, and Increasing Healthcare Expenditure Present Future...

Recommendation and review posted by Bethany Smith

When coronary arteries are blocked, starving the heart of blood, there are good medications and treatments to deploy, from statins to stents. Not so for heart failure, the leading factor involved in heart disease, the top cause of death worldwide.

Its whats on death certificates, said cardiologist Christine Seidman.

Seidman has long been interested in heart muscle disorders and their genetic drivers. She studies heart failure and other conditions that affect the myocardium the muscular tissue of the heart not the blood vessels where atherosclerosis and heart attacks come from, although their consequences are also felt in the myocardium, including heart failure.

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With her colleagues at Brigham and Womens Hospital and Harvard Medical School, she and a long list of international collaborators have been exploring the genetic underpinnings of heart failure. Based on experiments deploying a new technique called single-nucleus RNA sequencing on samples from heart patients, on Thursday they reported in Science their discovery of how genotypes change the way the heart functions.

Their work raises the possibility that some of the molecular pathways that lead to heart failure could be precisely targeted, in contrast to treating heart failure as a disease with only one final outcome.

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Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting, she said. To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

Seidman talked with STAT about the research, including how snRNAseq solves the smoothie problem, and what it might mean for patients. The conversation has been edited for clarity and brevity.

What happens in heart failure?

The heart becomes misshapen in one of two ways. It either becomes hypertrophied, where the walls of heart muscle become thickened and the volume within the heart is diminished, in what we call hypertrophic cardiomyopathy. Or it becomes dilated, when the volume in the heart is expanded and the walls become stretched. I think of it as an overinflated balloon, and that is called dilated cardiomyopathy.

Hypertrophy and dilatation are known to cause the heart over time to have profoundly diminished functional capacity. And clinically, we call that heart failure, much more commonly arising from dilated cardiomyopathy.

What does it feel like to patients?

When we see patients clinically, theyre short of breath, they have fluid retention. When we look at their hearts, we see that the pump function is diminished. That has led to a hypothesis of heart failure as sort of the end stage of many different disorders, but eventually the heart walks down a final common pathway. Then you need a transplant or a left ventricular assist device, or youre going to die prematurely.

What can be done?

Heart failure is a truly devastating condition, and it can arise early in life, in middle age, and in older people. There is no treatment for it, no cure for it, except cardiac transplantation, of course, which provides a whole host of other problems.

How did you approach this problem?

One of the questions we wanted to answer is, are there signals that we can discern that say there are different pathways and there are molecules that are functioning in those pathways that ultimately converge for failure, but through different strategies of your heart?

We treat every patient with heart failure with diuretics. We give them a series of different medications to reduce the pressure against which the heart has to contract. Im clinically a cardiologist, but molecularly Im a geneticist, so it doesnt make sense. If your house is falling down because the bricks are sticking together or if its falling down because the roof leaks and the water is pooling, you do things differently.

Tell me how you used single-cell RNA sequencing to learn more.

Looking at RNA molecules gives us a snapshot of how much a gene is active or inactive at a particular time point. Until recently, we couldnt do that in the heart because the approach had been to take heart tissue, grind it all up, and look at the RNAs that are up or down. But that gives you what we call a smoothie: Its all the different component cells those strawberries, blueberries, bananas mixed together.

But theres a technology now called single-cell RNA sequencing. And that says, what are the RNAs that are up or down in the cardiomyocytes as compared to the smooth muscle cells, as compared to the fibroblasts, all of which are in the cells? You get a much more precise look at whats changing in a different cell type. And thats the approach that we use, because cardiomyocytes [the cells in the heart that make it contract] are very large. Theyre at least three times bigger than other cells. We cant capture the single cell it literally does not fit through the microfluidic device. And so we sequenced the nuclei, which is where the RNA emanates from.

What did you find?

There were some similarities, but what was remarkable was the degree of differences that we saw in cardiomyocytes, in endothelial cells, in fibroblasts. Theres a signature thats telling us I walked down this pathway as compared to a different one that caused the heart to fail, but through activation or lack of activation of different signals along the way.

And that to me is the excitement, because if we can say that, we can then go back and say, OK, what happens if we were to have tweaked the pathway in this genotype and a different pathway in a different genotype? Thats really what precision therapy could be about, and thats where we aim to get to.

Whats the next step?

It may be that several genotypes will have more similarities as compared to other genotypes. But understanding that, I think, will allow us to test in experimental models, largely in mice, but increasingly in cellular models of disease, in iPS [induced pluripotent stem] cells that we can now begin to use molecular technologies to silence a pathway and see what that does to the cardiomyocytes, or silence the fibroblast molecule and see what that does in that particular genotype.

To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

What might this mean for patients?

If we knew that an intervention would make a difference thats where the experiments are we would intervene when we saw manifestations of disease. So the reason I can tell you with confidence that certain genes cause dilated cardiomyopathy is theres a long time between the onset of that expansion of the ventricle until you develop heart failure. So theres years for us to be able to stop it in its tracks or potentially revert the pathology, if we can do that.

What else can you say?

I would be foolish not to mention the genetic cause of dilated cardiomyopathy. Ultimately, if you know the genetic cause of dilated cardiomyopathy, this is where gene therapy may be the ultimate cure. Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting.

My colleagues have estimated that approximately 1 in 250 to 1 in 500 people may have an important genetic driver of heart muscle disease, cardiomyopathy. Thats a huge number, but not all of them will progress to heart failure, thank goodness. Around the world, there are 23 million people with heart failure. Its what ends up on most peoples death certificate. It is the most common cause of death.

Its a huge, huge burden. And there really is no cure for it except transplantation. We dont have a reparative capacity, so were going to have to know a cause and be able to intervene precisely for that cause.

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New research digs into the genetic drivers of heart failure, with an eye to precision treatments - STAT

Recommendation and review posted by Bethany Smith

New York, Aug. 08, 2022 (GLOBE NEWSWIRE) -- The global intracranial therapeutic delivery market is currently valued at around US$ 1.6 Bn and is anticipated to progress at an impressive CAGR of 7.9% over the 2022-2032 study period.

Cell and gene therapies are at the forefront of innovation in treating severe diseases, such as cancer, as well as rare diseases, accounting for around 12 percent of the pharmaceutical industrys clinical pipeline. However, the growing focus on effective therapy has impacted positive financial grades for cell and gene therapy throughout the clinical and social spectrum; intracranial therapeutic administration has been gaining favor in the biopharma industry.

The progressive development of CRISPR and next-generation sequencing has led to a surge in the interest in gene therapy and cell treatment in the past few years. The manufacturing community for cell and gene therapies, including pharmaceutical companies, contract development and manufacturing organizations (CDMOs), and suppliers of lab supplies and equipment, are looking into ways to strengthen supply chains and address process bottlenecks.

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Sales footprint expansion, which has been gaining more and more traction among key participants, calls for the desired assistance, based on financial approvals and consolidated activities. Additionally, several clinical trials have been carried out in association with research institutes.

Key Takeaways from Market Study

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Rising prevalence of neurological disorders and increasing research activities for the development of regenerative medicine to drive market growth over the coming years, says an analyst of Persistence Market Research.

Market Competition

The therapeutic delivery for intracranial is a highly consolidated market with limited key manufacturers operating in the industry. A majority of market players are focused on offering a limited range of cell, gene, and enzyme replacement therapy used for neurological disorder indications.

To strengthen their position in the global market, key players are focusing on strategic approaches such as mergers and collaborations to improve their production capabilities and expand their portfolios in various clinical and research fields.

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What Does the Report Cover?

Persistence Market Research offers a unique perspective and actionable insights on the intracranial therapeutic delivery market in its latest study, presenting a historical demand assessment of 2017 2021 and projections for 2022 2032.

The research study is based on the therapy (cell-based therapy, gene therapy, and enzyme replacement therapy) and indication (spinal muscular atrophy (SMA), multiple sclerosis, batten disease), and amyotrophic lateral sclerosis, across three key regions of the world considered in the taxonomy.

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Intracranial Therapeutic Delivery Market revenue will climb to US$ 4.2 Bn by the end of 2032 Persistence Market Research - GlobeNewswire

Recommendation and review posted by Bethany Smith