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Archive for the ‘Cardiac Stem Cells’ Category

Breakthroughs in Stem Cell Based Treatment of Heart Disease – The Connecticut College Voice

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In the United States alone, one person dies every 36 seconds from cardiovascular disease. Globally, it is also the leading cause of death, claiming over 17 million lives each year. In cases of severe illness, heart transplants have shown great promise in increasing the life expectancy of patients with heart disease. About 75% of heart transplant recipients survive for 5 more years and about 56% survive for 10 more years. However, the average wait times for heart transplants are long, often exceeding 6 months, and some patients simply cannot afford to wait that long.

Therefore, scientists tend to refer to other modes of treatment which rely on managing chronic symptoms, such as hypertension (high blood pressure), diabetes mellitus, obesity, and high cholesterol. This approach, however, does not address the root cause of the problem, which is impaired heart functioning. Since heart cells do not have a mechanism to replace damaged tissue, scientists have become increasingly excited about the possibility of repairing or replacing damaged heart tissue using stem cells (unique cells that have the ability to divide for an extended period of time and differentiate into specialized cells, such as cardiac cells or nerve cells).

Regenerative medicine has been a topic of excitement among researchers for decades. In 1999, Anthony Atala, director of the Wake Forest Institute for Regenerative Medicine, was the first to implant lab-grown organs into several patients between 4 and 19 years old. In his method, he obtained bladder cells from the children and coaxed those cells into dividing on a scaffold (a structure that mimics the normal organ). The engineered bladders functioned normally and no ill effects were reported. Pretty much I was able to live a normal life after, said Luke, one of Atalas patients.

More recently, Yoshiki Sawa, a professor of cardiovascular surgery at the University of Osakas medical school, and his team of Japanese researchers successfully transplanted lab-grown cardiac muscles into a human patient. The researchers first extracted adult stem cells from the patients blood or skin and genetically reprogrammed them into induced pluripotent stem (iPS) cells. They were then coaxed into 0.1-millimeter-thick sheets of cardiac tissue and grafted onto the diseased human hearts. According to Sawa, the cells do not seem to integrate into the heart tissue but rather release growth factors (proteins) that help regenerate blood vessels in the damaged muscle tissue and improve cardiac function. The team has conducted an operation on a patient in January 2020, marking the worlds first transplant of cardiac muscle cells.

The United States is also home to major breakthroughs in regenerative medicine. For decades, scientists have utilized embryonic stem cells to engineer heart muscle cells that are able to maintain synchronous breathing in a dish for hours. Despite this major feat, the creation of a working heart called for a more sophisticated technique. Doris Taylor, director of regenerative medicine research at the Texas Heart Institute (THI), has grown in her lab over 100 ghost hearts using protein scaffolds. She creates these scaffolds by first obtaining an animal heart and then decellularizing it by pumping a detergent through its blood vessels to strip away lipids, DNA, soluble proteins, sugars and almost all the other cellular material from the heart, leaving only a pale mesh of collagen, laminins, and the extracellular matrix. This heart does not necessarily have to be a human heart. She often finds pig hearts to be promising tissue because of their considerable safety and unlimited supply. She then recellularizes the heart by injecting it with millions of stem cells and attaching it to artificial lungs and a blood pump. Although her technique has only been used so far for growing animal hearts, she believes that it will eventually be used to create human heart transplants, thus, revolutionizing cardiovascular surgery and putting an end to organ shortage and anti-rejection drugs.

These groundbreaking results in regenerative medicine altogether have taken years of painstaking research to achieve. Taylor believes that her research is exceptionally close to building a working, human-sized heart, and Sawa says that his technique of grafting healthy cardiac muscle sheets onto the patients diseased heart tissue has already helped one of his patients move out of intensive care in just a few days. As the researchers gain more knowledge and get closer to the solution, however, they encounter more challenging obstacles. Sawa, for instance, has found that grafted cells do not always beat in synchrony. Researchers are also split on how these grafts work. On the other hand, investigating the best way to deliver cells still remains a challenge in Taylors research.

Stem cell research in tissue engineering could save millions of lives around the world; therefore, Taylor believes that a coordinated approach among the researchers, clinicians, industry, regulatory bodies and, finally, society should be invigorated to catapult the field forward. For instance, the Twenty-first Century Cures Act can help advance her work by facilitating cooperation among experts and regulatory bodies, providing for accelerated approvals for therapeutic tools in regenerative medicine, and improving the regulation of biologics products. She also maintains that tissue engineering efforts remain poorly funded and believes that more resources must be allocated before her studies can come to life. There is a lot of dependence on societal benevolence, she said. In an interview with RedMedNet, she also said that intense collaboration on a national and an international level is crucial and should be a priority, even though it could be challenging due to scheduling issues and differences in time zones.

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Breakthroughs in Stem Cell Based Treatment of Heart Disease - The Connecticut College Voice

Mount Sinai Cardiologist Awarded $2.9 Million NIH Grant to Advance Work with Stem Cells and Heart Repair after Heart Attack – Cath Lab Digest

Research may lead to identifying novel therapies for cardiac patients

(New York, NY November 19, 2020) Human placental stem cells may have the potential to regenerate heart tissue after a heart attack, according to Mount Sinai researchers who have received a $2.9 million grant from the National Institutes of Health to study them. Their findings could lead to new therapies for repairing the heart and other organs.

Hina W. Chaudhry, MD,Director of Cardiovascular Regenerative Medicine at the Icahn School of Medicine at Mount Sinai, is the Principal Investigator for this four-year award.

This is very exciting. These cells may represent the ideal cell type for heart repair, which has been very challenging because clinical trials of other cell types did not find much benefit, says Dr. Chaudhry. Weve never before seen a stem cell type that can be harvested from an adult organthe placentaand has the ability to travel through the circulation and not be attacked by the immune system.

Dr. Chaudhry and a team of investigators previously discovered thatmouse placental stem cells can help the hearts of mice recover from injury that could otherwise lead to heart failure. They identified a specific type of placental stem cells, called Cdx2 cells, as the most effective in making heart cells regenerate. They discovered this by inducing heart attacks in groups of male mice and then injecting the placental Cdx2 cells isolated from females into their bloodstream. Imaging showed that the mice with Cdx2 stem cell treatments had significant improvement in cardiac function and regeneration of healthy tissue in the heart. The mice without this stem cell therapy went into heart failure and their hearts had no evidence of regeneration.

This team also found that the mouse Cdx2 cells have all the proteins of embryonic stem cells, which are known to generate all organs of the body, but also additional proteins, giving them the ability to travel directly to the injury site, which is something embryonic stem cells cannot do, and the Cdx2 cells appear to avoid the host immune response.

The new grant allows the researchers to build upon this discovery by isolating human Cdx2 cells from human placentas and studying their ability to grow heart cells. They also plan to expand into other organs and tissues in the future.

This was a serendipitous discovery based on clinical observations of patients with peripartum cardiomyopathy. We surmised that stem cells originating from the placenta may be assisting in repair of the mothers heart and designed studies to identify the cell types involved. We then showed that they work very well in male mice also when isolated from female placentas and now we hope to design a human cell therapy strategy for heart regeneration with this grant. Given that these cells maintain all the stem properties of embryonic stem cells, we are hopeful to utilize them for other types of organ repair as well, adds Dr. Chaudhry.

The grant is being used in collaboration with the Departments of Obstetrics and Gynecology and Pathology at Cedars-Sinai Medical Center in Los Angeles.

About the Mount Sinai Health System

The Mount Sinai Health System is New York City's largest academic medical system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai is a national and international source of unrivaled education, translational research and discovery, and collaborative clinical leadership ensuring that we deliver the highest quality carefrom prevention to treatment of the most serious and complex human diseases. The Health System includes more than 7,200 physicians and features a robust and continually expanding network of multispecialty services, including more than 400 ambulatory practice locations throughout the five boroughs of New York City, Westchester, and Long Island.Mount Sinai Heart at The Mount Sinai Hospital is within the nations No. 6-ranked heart center, and The Mount Sinai Hospital is ranked No. 14on U.S. News & World Report's "Honor Roll" of the Top 20 Best Hospitals in the country and the Icahn School of Medicine as one of the Top 20 Best Medical Schools in country. Mount Sinai Health System hospitals are consistently ranked regionally by specialty and our physicians in the top 1% of all physicians nationally by U.S. News & World Report.

For more information, visithttps://www.mountsinai.orgor find Mount Sinai on Facebook, Twitter and YouTube.

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Mount Sinai Cardiologist Awarded $2.9 Million NIH Grant to Advance Work with Stem Cells and Heart Repair after Heart Attack - Cath Lab Digest

Autologous Stem Cell Based Therapies Market Share, Growth by Top Company, Region, Application, Driver, Trends & Forecasts by 2026 – PRnews Leader

The Autologous Stem Cell Based Therapies Market was valued at US$ XX million in 2019 and is projected to reach US$ XX million by 2025, at a CAGR of XX percentage during the forecast period. In this study, 2019 has been considered as the base and 2020 to 2025 as the forecast period to estimate the market size for Autologous Stem Cell Based Therapies Market

Deep analysis about market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2025), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry have been analysed scientifically. This report will help you to establish comprehensive overview of the Autologous Stem Cell Based Therapies Market

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The Autologous Stem Cell Based Therapies Market is analysed based on product types, major applications and key players

Key product type:Embryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Key applications:Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

Key players or companies covered are:RegeneusMesoblastPluristem Therapeutics IncU.S. STEM CELL, INC.Brainstorm Cell TherapeuticsTigenixMed cell Europe

The report provides analysis & data at a regional level (North America, Europe, Asia Pacific, Middle East & Africa , Rest of the world) & Country level (13 key countries The U.S, Canada, Germany, France, UK, Italy, China, Japan, India, Middle East, Africa, South America)

Inquire or share your questions, if any: https://i2iresearch.com/report/global-autologous-stem-cell-based-therapies-market-2020-market-size-share-growth-trends-forecast-2025/

Key questions answered in the report:1. What is the current size of the Autologous Stem Cell Based Therapies Market, at a global, regional & country level?2. How is the market segmented, who are the key end user segments?3. What are the key drivers, challenges & trends that is likely to impact businesses in the Autologous Stem Cell Based Therapies Market?4. What is the likely market forecast & how will be Autologous Stem Cell Based Therapies Market impacted?5. What is the competitive landscape, who are the key players?6. What are some of the recent M&A, PE / VC deals that have happened in the Autologous Stem Cell Based Therapies Market?

The report also analysis the impact of COVID 19 based on a scenario-based modelling. This provides a clear view of how has COVID impacted the growth cycle & when is the likely recovery of the industry is expected to pre-covid levels.

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3D Cardiac Mapping Systems Market Competitive Landscape Analysis with Forecast by 2028 – The Haitian-Caribbean News Network

Global 3D Cardiac Mapping Systems Market: Overview

Cardiac mapping is a special type of technique which helps in gathering and displaying the information from cardiac electrograms. Such technique is mainly used in the diagnosis of heart rhythms. Therefore, cardiac mapping technique has gained immense popularity in case of arrhythmia. The cardiac mapping procedure involves the percutaneous insertion of catheter into the heart chamber and recording the cardiac electrograms sequentially. Such procedure helps in correlating the cardiac anatomy with the electrograms. The latest 3D cardiac mapping systems provide the three dimensional model of hearts chamber, which further helps in tracking the exact location of the catheter. Such advantages are majorly driving the global 3D cardiac mapping systems market.

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From the perspective of technology, the global 3D cardiac mapping systems market is segmented into basket catheter mapping, electroanatomical mapping, and real-time positional management (Cardiac pathways) EP system. Among these segments, electroanatomical mapping segment accounts for the maximum share in the global 3D cardiac mapping systems market. This mapping are extensively used in several healthcare industry due to its potential in increasing the safety, accuracy, and efficiency of catheter. A research report by TMR Research (TMR) thoroughly explains the new growth opportunities in the global 3D cardiac mapping systems market. Additionally, the report also provides a comprehensive analysis of the markets competitive landscape.

Global 3D Cardiac Mapping Systems Market: Notable Developments

Some of the recent developments are contouring the shape of the global 3D cardiac mapping systems market in a big way:

Key players operating in the global 3D cardiac mapping systems market include BioScience Webster, Boston Scientific Corporation, and Abbott.

Global 3D Cardiac Mapping Systems Market: Key Growth Drivers

Rising Number of Patients with Cardiac Disorders and Arrhythmia Fillips Market

The global 3D cardiac mapping systems market has grown steadily over the years, owing to the convenience it provides to the patients with heart problem. Growing number of people with cardiovascular diseases and rising cases of arrhythmia are the major factors fueling growth in the global 3D cardiac mapping systems market. Along with this, increasing pressure for reducing diagnosis errors and rapidly rising healthcare expenditure are also responsible for boosting the global 3D cardiac mapping systems market. However, above all such factors, the global 3D cardiac mapping systems market is majorly fueled by the accuracy and patient safety provided through real-time monitoring. Such 3D cardiac mapping systems are mainly designed to improve the resolution. This system also helps in gaining prompt of cardiac activation maps. All such advantages are also providing impetus to the growth of the global 3D cardiac mapping systems market.

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Furthermore, rising ageing population who are prone to heart-attack and several chronic heart disorders and increasing diagnosis rate of cardiac illness are the factors stoking demand in the global 3D cardiac mapping systems market. Moreover, this 3D cardiac mapping helps in reducing the diagnosis time. Such factor is also contributing to the growth of the global 3D cardiac mapping systems market.

Global 3D Cardiac Mapping Systems Market: Regional Outlook

On the regional front, North America is leading the global 3D cardiac mapping systems market as the region has seen rapid growth in healthcare industry. Along with this, increasing prevalence of heart attacks, rising healthcare expenditure, and burgeoning population is also responsible for fueling growth in the 3D cardiac mapping systems market in this region.

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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3D Cardiac Mapping Systems Market Competitive Landscape Analysis with Forecast by 2028 - The Haitian-Caribbean News Network

Organoids mimic the early development of the heart in mouse embryos – BioNews

16 November 2020

Organoids can be used to study early stages of heart development in mouse embryos, a new study shows.

Researchers from the cole Polytechnique Fdrale de Lausanne, Switzerland, have reported that they were able to produce a mouse heart organoid from embryonic stem cells, which displayed essential features of an early developing heart. They suggested that this reveals a novel application of organoids for studying early embryonic stages of development.

'One of the advantages of embryonic organoids is that, through the co-development of multiple tissues, they preserve crucial interactions that are necessary for embryonic organogenesis,' said Dr Giuliana Rossi, lead author of the study. 'The emerging cardiac cells are thus exposed to a context similar to the one that they encounter in the embryo.'

In their study, published in Cell Stem Cell, the team exposed mouse embryonic stem cells to a mix of three factors involved in promoting heart growth. One week later, the stem cells self-organised into so-called gastruloids:organoids with an embryo-like organisation, which displayed signs of early heart development. The cell aggregatesnot only expressed several genes known to regulate cardiovascular development, but also generated a structure resembling a vascular network. Furthermore, the researchers found an 'anterior cardiac crescent-like domain' in the gastruloids, which even produced a beating heart tissue. Similar to the muscle cells of the embryonic heart, this area was also sensitive to calcium ions.

Organoids have been mostly the focus of research into the generation of adult tissues and organs for pharmaceutical and medical research. In their new publication, Professor Matthias Ltolf and his team suggested that they can also provide a system to study early embryonic stages of the developing heart and other organs, as they preserve important tissue-tissue interactions.

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Organoids mimic the early development of the heart in mouse embryos - BioNews

Mimicking the early development of the heart – Tech Explorist

Once upon a time, growing organs in the lab were science fiction. But now, methods such as stem cell biology and tissue engineering have turned that fiction into reality with the advent of organoids.

Organoids are tiny lab-grown tissues and organs that are anatomically correct and physiologically functional.

Recently, the lab of Matthias Ltolf at the School of Life Sciences at EPFL has successfully produced a mouse heart organoid in its early embryonic stages. Scientists grew organoids from mouse embryonic stem cells, which, under the right conditions, can self-organize into structures that mimic aspects of the architecture, cellular composition, and function of tissues found in real organs.

Placed in cell-culture under specific conditions, the embryonic stem cells from a three-dimensional aggregate called a gastruloid, which can follow the mouse embryos developmental phases.

This studys idea was that the mouse gastruloid could be utilized to mimic the beginning phases of heart development in the embryo. This is a new use of organoids, which are commonly developed to mimic adult tissues and organs.

Also, there are three features of mouse gastruloids that make them a suitable template for mimicking embryonic development: they establish a body plan like real embryos. They show similar gene expression patterns. And when it comes to the heart, which is the first organ to form and function in the embryo, the mouse gastruloid also preserves important tissue-tissue interactions necessary to grow one.

Equipped with this, the scientists exposed mouse embryonic stem cells to a cocktail of three factors known to promote heart growth. Following 168 hours, the subsequent gastruloids gave early heart development indications: they expressed several genes that regulate cardiovascular development in the embryo. They even generated what resembled a vascular network.

Importantly, scientists found that the gastruloids developed what they call an anterior cardiac crescent-like domain. This structure produced a beating heart tissue, similar to the embryonic heart. As the muscle cells of the embryonic heart, the beating compartment was also sensitive to calcium ions.

Giuliana Rossi, a post-doctoral researcher from Ltolfs laboratory, said,Opening up an entirely new dimension to organoids, the breakthrough work shows they can also be used to mimic embryonic stages of development. One of the advantages of embryonic organoids is that, through the co-development of multiple tissues, they preserve crucial interactions that are necessary for embryonic organogenesis.

The emerging cardiac cells are thus exposed to a context similar to the one that they encounter in the embryo.

The study was conducted in collaboration with Viventis Microscopy, EPFL Bioimaging and Optics Platform, Institut de Biologie du Dveloppement de Marseille, Johns Hopkins University School of Medicine, EPFL Institute of Chemical Sciences and Engineering.

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Cell Therapy and Gene Therapy Markets, 2019-2020 & Forecast to 2025 and 2030 – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy and Gene Therapy Markets" report has been added to ResearchAndMarkets.com's offering.

This is an exciting and interesting time in the cell and gene therapy industry. The science is moving ahead as industry industrializes and standardizes the manufacturing and commercialization of products. Cell and gene therapy products are transforming the treatment of cancers and genetic diseases, as well as expanding into other areas of medicine including autoimmune diseases, cardiovascular diseases, musculoskeletal disease, dermatological diseases, and many others.

Cell Therapy and Gene Therapy Markets presents the market in segments that provide an overview of disease epidemiology, market estimates and forecasts, and competitive summary of leading providers:

The report examines developments in cell and gene therapy markets by condition/disorder, including principal products, trends in research and development, market breakdown of cell and gene therapies, regional market summary, and competitor summary.

The following conditions/disorders are covered:

Dermatology, including:

Oncology, including:

Ophthalmic Conditions, including:

Other Conditions, including:

The report comments on the current COVID-19 cell and gene therapy pipeline. There are a number of companies that are responding to the call to develop a therapeutic or vaccine for the coronavirus, including:

The leading influencers in the market are those which have become first-to-market participants in the cell and gene therapy segment, have new developments which may disrupt current market conditions, and/or have an extensive pipeline sure to impact the market in the long-term forecast:

Because gene therapies are currently not available in any wide capacity, there is little precedent upon which to base forecasts. Dollar figures represent the estimated global market for 2019 and the expected market for 2020 based on first-quarter company reports and are expressed in current dollars. Forecasts are provided through 2025 and an extended forecast for 2030. The size of each market segment refers to manufacturers' revenues.

For more information about this report visit https://www.researchandmarkets.com/r/ek1qqb

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Cell Therapy and Gene Therapy Markets, 2019-2020 & Forecast to 2025 and 2030 - ResearchAndMarkets.com - Business Wire

UPDATED: Merck’s Keytruda nets another approval, this time in triple negative breast cancer. Can it catch up to Tecentriq? – Endpoints News

Another day, another win for Mercks blockbuster Keytruda.

The FDA has granted accelerated approval for the cash cow combined with chemotherapy in triple negative breast cancer, giving the drug the green light in its 18th different cancer. Mondays new indication comes for patients with PD-L1-expressing tumors with a Combined Positive Score of at least 10.

Merck noted that due to the nature of the accelerated approval, the thumbs up is contingent upon confirmatory trials.

Data for the approval first came back in February, when the Keynote-355 trial demonstrated Keytruda plus chemo significantly improved progression-free survival compared to chemo by itself. The study showed that, in the target population with a CPS of at least 10, the combination reduced the risk of disease progression or death by 35% with a median PFS of 9.7 months, against 5.6 months in the placebo arm.

On safety, the February data showed 2.5% of all patients in the drug arm saw fatal adverse events, including cardiac arrest and septic shock, with serious side effects appearing in 30% of patients. Keytruda was discontinued due to adverse events in 11% of patients.

Frontline triple negative breast cancer is a particularly difficult indication to treat, as the growth of the cancer is not fueled by the hormones estrogen and progesterone, or by the HER2 protein. Its one of the rare fields in which Roches PD-L1 Tecentriq has enjoyed a head start over Keytruda and Opdivo, the leaders in the checkpoint race, as Tecentriq is approved in combination with Abraxane for this indication.

Back in May 2019, Merck conceded a failure in the arena after a Phase III study flopped on overall survival. But a few months later, the pharma turned things around after discovering a neoadjuvant regimen of Keytruda and chemo followed by Keytruda monotherapy after surgery induced a higher pathological complete response rate.

Though execs presented that as a positive, some analysts didnt paint as sunny a picture. This past February, when the Keynote-355 topline data was first published, SVB Leerinks Daina Graybosch pointed out that because only patients with a CPS of at least 10 appeared to benefit, instead of a score of at least 1, it wont be able to treat as broad a population as Tecentriq. Roche, she noted, also has about a two-year head start.

A Merck spokesperson also had this to say about the CPS and IC percentages:

In TNBC, we measure PD-L1 with a combined positive score (CPS). The CPS includes staining for tumor cells, as well as tumor-infiltrating immune cells and it is not a percentage. We believe CPS 10 is roughly equivalent to how Roche scores PD-L1+ patients (IC>=1% based on the SP142 assay) on tumor-infiltrating immune cells (IC). The prevalence of the PD-L1 positive population in TNBC whether by CPS of greater than or equal to 10 or IC of 1% is both about 40%.

Keytruda is already one of the best-selling drugs in the world, having notched roughly $3.9 billion in the first half of 2020 alone. Some have predicted the drug may overtake AbbVies Humira as the top seller within the next few years, with the most optimistic estimate pegged for $22.2 billion in sales by 2025.

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UPDATED: Merck's Keytruda nets another approval, this time in triple negative breast cancer. Can it catch up to Tecentriq? - Endpoints News

Can Hearts Repair Themselves Via Stem Cells – The Niche

Can hearts repair themselves via their own stem cells?

Sometimes what we scientists all know to be true turns out later on to be wrong and there are clear instancesof this in the stem cell field.

For example for decades the dogma was that the adult mammalianbrain did not have stem cells, but now most researchers believethat the adult brain does have stem cells, although for humans this is still being debated.

What we perceive as factual can change over time.

Yamanaka disproved the entrenched notion that differentiated cells were permanently locked into that differentiated state with his revolutionary findings on induced pluripotent stem cells. The new reality, which seemed revolutionary in some ways in 2006-2007, now is established fact.

So what about the idea of the human heart have resident populations of stem cells that can fix problems, perhaps as severe as damage from heart attacks? One of the first stem cell talks I ever saw way back around IPS cells were discovered was by a guy who assumed the factual answer to this question was Yes!

What about now in 2020? I have an ongoing Twitter poll on this question as of Nov. 5, 2020 so check it out below.

Today the cardiac regenerative field finds itself at an interesting crossroads.

A few say yes there are cardiac stem cells and that they can mediate repair. However, most heart researchers that Ive talked to in recent years feel just as strongly that there are no such cells. Some also have added that even if there are at least a handful of such cells or they arise due to damage, they cant do anything meaningful about serious heart damage.

I asked cardiac stem cell expert, Deepak Srivastava for his thoughts on this in a previous post and foundhis answercompelling. Because that was a fairly long time ago, I got an update to the same kind of question just now from two leading cardiac regenerative medicine and stem cell researchers.

Associate Professor of Medicine and Director of Cardiovascular Regenerative Medicine at Mt. Sinai, Hina Chaudhry had this to say:

One thing is certain: As both a clinical cardiologist who has cared for patients with heart attacks and a stem cell biologist, I can tell you that no scientific data supports an endogenous stem cell population in the adult heart and that no adult with a transmural myocardial infarction ever loses the resulting scar throughout their lifetime.

Professor Chuck Murry of the UW sent this:

Our current best evidence suggests that there are no stem cells in the adult heart that can give rise to new cardiomyocytes. This has been studied intensivelyfrom the bottom up, by tracing candidate stem cells and following their differentiated progeny, and from the top down by marking pre-existing cardiomyocytes and looking for their dilution as unmarked stem cells enter the pool. Both have shown the same thing: if this happens at all, its frequency is on the order of 10e-4 per year, which by any measure is next to nothing. There is slow turnover of cardiomyocytes in the adult mammalian heart, at ~1% per year, and this can be accounted for entirely by replication of pre-existing cardiomyocytes. One has to wonder, why has Nature done this? Why would such a vital organ have no stem cells for replenishment, along with such a low rate of endogenous replication?

I believe that Drs. Murry and Chaudhry are right. Chucks last question there is one for long discussions and is similar to discussions Ive had about the few stem cells/potential for endogenous repair in the adult human brain.

Still, you can find a diversity of papers now in 2020 in PubMed with Heart Regeneration or Cardiac Regeneration or Cardiac stem cells in their titles. However, many of the papers relate to stem cell infusions rather than invoking endogenous resident cells.

If not in humans, what about other mammals? There are glimpses of interesting possible stem cell activity in the mammalian heart, even if not in humans

A November 2014Cell Stem Cell paper from the lab of Juan Carlos Izpisua Belmonte, entitled InVivo Activation of a Conserved MicroRNA Program Induces Mammalian Heart Regeneration, argues for endogenous mammalian heart regeneration in part via dedifferentiation of cells into stem-like cells. This raises the interesting notion that while the mammalian heart does not normally have many (or any?) resident stem cells, damage can change some other cells into stem cell-like cells.

One of the biggest advocates of endogenous cardiac stem cells and repair, Piero Anversa formerly of Harvard and Brigham and Womens Hospital, has become one of the most controversial as well. His papers have come under fire and some have been retracted. Anversa was the subject of a Harvard investigation and was suing Harvard for how it has conducted the investigation and other matters related to his work.

In my view, his situation has raised even more skepticism about the idea of endogenous heart stem cells in people.

Even if the endogenous stem cell-like activity in the heart is absent or not enough to mediate clinically significant repair in humans, by deciphering the molecular basis of this kind of activity in other animals could the field still open the door to powerful new treatments for heart disease? For instance, if some adult mammalian hearts naturally replace 1 in 200 cells per year, perhaps cardiac researchers can find a way to boost that by an order of magnitude with a drug and have a meaningful impact for human patients.

Or if dedifferentiation of non-stem cells in the heart into stem cell-like cells can be induced by damage, could a drug therapy trigger that same effect even if damage occurred long ago or in the context of relatively minor damage?

Many researchers are focusing more on using injections of stem cells into the heart to repair damage. The types of stem cells being used for research attempts at heart repair are very diverse, including both placental cells and indirect use of IPS cells in Japan via a recently approved trial there.

Even the area of stem cell transplants into the heart generates its share of debate and well have to see in the long run how the clinical trial data turn out. I hope there can be positive impact in the future given the overwhelming number of people with heart damage.

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Can Hearts Repair Themselves Via Stem Cells - The Niche

Stem cells as therapy for heart disease: iPSCs, ESCs, CSCs …

Heart Diseases are serious and global public health concern. In spite of remarkable therapeutic developments, the prediction of patients with Heart Failure (HF) is weak, and present therapeutic attitudes do not report the fundamental problem of the cardiac tissue loss. Innovative therapies are required to reduce mortality and limit or abolish the necessity for cardiac transplantation. Stem cell-based therapies applied to the treatment of heart disease is according to the understanding that natural self-renewing procedures are inherent to the myocardium, nonetheless may not be adequate to recover the infarcted heart muscle. Following the first account of cell therapy in heart diseases, examination has kept up to rapidity; besides, several animals and human clinical trials have been conducted to preserve the capacity of numerous stem cell population in advance cardiac function and decrease infarct size. The purpose of this study was to censoriously evaluate the works performed regarding the usage of four major subgroups of stem cells, including induced Pluripotent Stem Cells (iPSC), Embryonic Stem Cells (ESCs), Cardiac Stem Cells (CDC), and Skeletal Myoblasts, in heart diseases, at the preclinical and clinical studies. Moreover, it is aimed to argue the existing disagreements, unsolved problems, and prospect directions.

Keywords: Heart disease; Myocardial regeneration; Stem cells; Tissue repair.

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Stem cells as therapy for heart disease: iPSCs, ESCs, CSCs ...

Global Autologous Stem Cell Based Therapies Market 2020 Segmentation, Statistics, Top Manufacturers, Regional Analysis and Forecast to 2025 – The…

.biz has announced a business intelligence study on Global Autologous Stem Cell Based Therapies Market 2020 by Company, Type and Application, Forecast to 2025 that reveals diverse information allowing keen market participants to understand the measures of the market. The report sheds light on market developments, noteworthy trends as well as competitive vendor activities and performance analysis. The report is aimed at offering readers real-time data vital to drive future-ready investment decisions. The research focuses on the dominant trends, persistent challenges, and threats, as well as budding opportunities influencing growth scenarios in the global Autologous Stem Cell Based Therapies market. The market report is a comprehensive research that demonstrates overall consumption structure, development trends, well-known providers, and market segments.

Executive Summary:

The report assesses the historical and future timelines, accurate growth predictions, and forecast estimations, and fast-changing market forces. The report draws references for an extensive analysis of the global Autologous Stem Cell Based Therapies market, entailing important details about key market players, with a broad overview of expansion probability and expansion strategies. The report has been designed and presented in the form of tables and figures and other statistical to generate higher reader perception. Later in the report, details on manufacturer information, leading market participants as well as other key players have also been added.

NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

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Understanding Scope:

Leading companies covered in the report include: Regeneus, US STEM CELL, INC., Mesoblast, Med cell Europe, Pluristem Therapeutics Inc, Tigenix, Brainstorm Cell Therapeutics

By the product type, the market is primarily split into: Embryonic Stem Cell, Resident Cardiac Stem Cells, Umbilical Cord Blood Stem Cells

By the end-users/application, this report covers the following segments: Neurodegenerative Disorders, Autoimmune Diseases, Cardiovascular Diseases

The report contains detailed market size and forecast for the following countries and regions: North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

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Moreover, upstream raw materials, downstream demand analysis, and end-user industry listings have been studied with vendors in this global Autologous Stem Cell Based Therapies market. Product flows and distribution channels were also presented in this research report. The report includes broad market segmentation based on the different product types, a wide application spectrum, the key regions, and the existing competition among players. In addition, the report reviews pricing analysis, profit margins, cost and demand volatility, import/export dynamics, gross revenue, and various other aspects of the market.

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Global Autologous Stem Cell Based Therapies Market 2020 Segmentation, Statistics, Top Manufacturers, Regional Analysis and Forecast to 2025 - The...

The Amniotic Fluid Stem Cell Therapy market to be in conjunction to growth from 2018 to 2026 – PRnews Leader

Stem cells are biological cells which have the ability to distinguish into specialized cells, which are capable of cell division through mitosis. Amniotic fluid stem cells are a collective mixture of stem cells obtained from amniotic tissues and fluid. Amniotic fluid is clear, slightly yellowish liquid which surrounds the fetus during pregnancy and is discarded as medical waste during caesarean section deliveries. Amniotic fluid is a source of valuable biological material which includes stem cells which can be potentially used in cell therapy and regenerative therapies. Amniotic fluid stem cells can be developed into a different type of tissues such as cartilage, skin, cardiac nerves, bone, and muscles. Amniotic fluid stem cells are able to find the damaged joint caused by rheumatoid arthritis and differentiate tissues which are damaged.

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Medical conditions where no drug is able to lessen the symptoms and begin the healing process are the major target for amniotic fluid stem cell therapy. Amniotic fluid stem cells therapy is a solution to those patients who do not want to undergo surgery. Amniotic fluid has a high concentration of stem cells, cytokines, proteins and other important components. Amniotic fluid stem cell therapy is safe and effective treatment which contain growth factor helps to stimulate tissue growth, naturally reduce inflammation. Amniotic fluid also contains hyaluronic acid which acts as a lubricant and promotes cartilage growth.

With increasing technological advancement in the healthcare, amniotic fluid stem cell therapy has more advantage over the other therapy. Amniotic fluid stem cell therapy eliminates the chances of surgery and organs are regenerated, without causing any damage. These are some of the factors driving the growth of amniotic fluid stem cell therapy market over the forecast period. Increasing prevalence of chronic diseases which can be treated with the amniotic fluid stem cell therapy propel the market growth for amniotic fluid stem cell therapy, globally. Increasing funding by the government in research and development of stem cell therapy may drive the amniotic fluid stem cell therapy market growth. But, high procedure cost, difficulties in collecting the amniotic fluid and lack of reimbursement policies hinder the growth of amniotic fluid stem cell therapy market.

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The global amniotic fluid stem cell therapy market is segmented on basis of treatment, application, end user and geography:

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Rapid technological advancement in healthcare, and favorable results of the amniotic fluid stem cells therapy will increase the market for amniotic fluid stem cell therapy over the forecast period. Increasing public-private investment for stem cells in managing disease and improving healthcare infrastructure are expected to propel the growth of the amniotic fluid stem cell therapy market.

However, on the basis of geography, global Amniotic Fluid Stem Cell Therapy Market is segmented into six key regionsviz. North America, Latin America, Europe, Asia Pacific Excluding China, China and Middle East & Africa. North America captured the largest shares in global Amniotic Fluid Stem Cell Therapy Market and is projected to continue over the forecast period owing to technological advancement in the healthcare and growing awareness among the population towards the new research and development in the stem cell therapy. Europe is expected to account for the second largest revenue share in the amniotic fluid stem cell therapy market. The Asia Pacific is anticipated to have rapid growth in near future owing to increasing healthcare set up and improving healthcare expenditure. Latin America and the Middle East and Africa account for slow growth in the market of amniotic fluid stem cell therapy due to lack of medical facilities and technical knowledge.

Some of the key players operating in global amniotic fluid stem cell therapy market are Stem Shot, Provia Laboratories LLC, Thermo Fisher Scientific Inc. Mesoblast Ltd., Roslin Cells, Regeneus Ltd. etc. among others.

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The Amniotic Fluid Stem Cell Therapy market to be in conjunction to growth from 2018 to 2026 - PRnews Leader

BioCardia Reports Third Quarter 2020 Financial Results and Business Highlights – GlobeNewswire

SAN CARLOS, Calif., Nov. 10, 2020 (GLOBE NEWSWIRE) -- BioCardia, Inc.[NASDAQ: BCDA], a leader in the development of autologous and allogenic cell therapies, today reported financial results and business highlights for the third quarter of 2020 and filed its quarterly report on Form 10-Q for the three and nine months ended September 30, 2020 with the Securities and Exchange Commission on November 10, 2020.

The Company is advancing its autologous and allogenic bone marrow-derived cell therapies for three cardiovascular indications and one respiratory indication.

Third Quarter 2020 Business Highlights:

Autologous Cell Therapies

Allogenic Cell Therapies

Corporate Developments

We are reaching critical milestones in our cardiovascular and respiratory cell therapy development programs at a time when patients are increasingly presenting with heart damage due to COVID-19, said BioCardia CEO Peter Altman, PhD. We believe that the clinical data supports patient benefit through paracrine mechanisms, which differs from those attempting to transform cells into new heart cells, and believe that the approach has tremendous promise to help patients suffering from severe heart and respiratory diseases.

Third Quarter 2020 Financial Results:

Anticipated Upcoming Milestones in Q4 2020:

About BioCardiaBioCardia, Inc., headquartered in San Carlos, California, is developing regenerative biologic therapies to treat cardiovascular and respiratory disease. CardiAMP autologous and Neurokinin-1 Receptor Positive allogenic cell therapies are the Companys biotherapeutic platforms in clinical development. The Company's products include the Helix Biotherapeutic Delivery System and its steerable guide and sheath catheter portfolio. BioCardia also partners with other biotherapeutic companies to provide its Helix system and clinical support for their programs studying therapies for the treatment of heart failure, chronic myocardial ischemia and acute myocardial infarction. For more information, visit http://www.BioCardia.com.

Forward Looking StatementsThis press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, references to the enrollment of our clinical trials, the availability of data from our clinical trials, filings with the FDA, FDA product clearances, the efficacy and safety of our products and therapies, anticipated milestones, and other statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations. Such risks and uncertainties include, among others, the inherent uncertainties associated with developing new products or technologies, regulatory approvals, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardias business and product development plans and overall market conditions.We may find it difficult to enroll patients in our clinical trials due to many factors, some of which are outside of our control.Slower than targeted enrollment could delay completion of our clinical trials and delay or prevent development of our therapeutic candidates.These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

We may use terms such as believes, estimates, anticipates, expects, plans, intends, may, could, might, will, should, approximately or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate.Additional factors that could materially affect actual results can be found in our documents filed with the SEC, including our recent filings on Form 8-K, Form 10-K and Form 10-Q, particularly any statements under the caption entitled Risk Factors Therein. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

Media Contact:Michelle McAdam, Chronic Communications, Inc.michelle@chronic-comm.com(310) 902-1274

Investor Contact:David McClung, Chief Financial OfficerInvestors@BioCardia.com(650) 226-0120

BIOCARDIA, INC.Condensed Statements of Operations(Unaudited In thousands, except share and per share amounts)

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BioCardia Reports Third Quarter 2020 Financial Results and Business Highlights - GlobeNewswire

Exploring Genetic Variation and COVID-19 Clinical Variability – Technology Networks

One of the biggest challenges that scientists and healthcare professionals are facing during the COVID-19 pandemic is the high rate of clinical variability. Whilst some patients present as asymptomatic, others are developing more severe symptoms such as pneumonia, and some cases are ultimately proving fatal. Why?The answer remains elusive; however, extensive research is exploring the possible contribution our genetics may be having. Genetic variation differences in the DNA sequences that make up our genome can impact our response to infectious diseases.

GoodCell uniquely measures and monitors inherited and acquired genetic variations in stem cells and other nucleated cells in our blood over time. Technology Networks recently spoke with Dr Salvatore Viscomi, chief medical officer at GoodCell, and attending physical at Baystate Health, to explore factors that might influence COVID-19 risk, and to discuss how the company is working to identify at-risk individuals through genetic variation analysis.

Molly Campbell (MC): For our readers that may be unfamiliar, can you discuss why medicine is moving towards a personalized approach, and why this is important considering genetic variation?Salvatore Viscomi (SV): Healthcare has traditionally taken the approach of one size fits all in defining individual risk for a disease and prescribing therapy for it. Understanding the differences between individuals on a molecular level optimizes assessment of an individuals susceptibility to a certain disease and predicting response to pharmacological therapy. Genomics plays the most important role in the emergence of personalized therapy. Identifying the inherited and acquired genetic variation will direct personalized screening and prevention plans and inform bespoke medical therapies.

MC: We know that there is high clinical variability across COVID-19 patients. How might genetic variation be contributing here, and what published evidence exists to support this?SV: Understanding immune response is critical to identifying individuals at high risk of severe morbidity and mortality. Emerging research suggests that accumulated genetic variation in our blood cells may be associated with a dysfunctional inflammatory response to COVID-19 leading to its pulmonary, cardiac and coagulopathic complications.

In a recent study published by JAMA Cardiology, researchers demonstrated an association between the presence of accumulated genetic change in our blood cells and a pro-inflammatory immune response that resembles the exaggerated cytokine release syndrome (CRS) manifested in COVID-19-positive patients. Direct evidence has emerged more recently; a study published in Cancers examined patients hospitalized with COVID-19 and found a significantly higher prevalence of accumulated genetic variation in all age groups compared to age-matched control groups.

MC: What impact might genetic variation in COVID-19 patients have on efforts to develop therapeutics or preventives, such as vaccines?SV: Identifying highly susceptible individuals through blood testing could have many applications. As an initial wave of vaccines move through Phase III trials and potentially come to market, we would have the data to determine prioritization of vaccinations when one is available. Business and government sectors need insight into risk factors that can inform inoculation strategies for societys most vulnerable, inform decisions around who should and should not be on the front lines, and give people more control when making personal decisions about how to mitigate individual risk. The broader field of genetics offers a window into the potential to correlate inherited and acquired gene mutations with immune response for the betterment of society, providing a more robust and accurate set of risk factors unique to every individual.

Furthermore, in high-risk individuals, targeting inflammation may be a clinical strategy to mitigate its clinical consequencesin COVID-19. For example, we may identify patients who are most responsive to pro-inflammatory inhibitors. Implementing measures intended to reduce subjects exposure to the infection or likelihood of contracting such infection through self-isolation, quarantine or social distancing may be advised.

MC: Can you explain the aims of GoodCell, and what the company does in terms of "banking blood for life"?SV: GoodCells mission is to extend and improve the quality of life through technology powered by our own cells. Blood is the author of our bodies, and can both cure as well as cause disease. Through our proprietary data aggregation and analytics technology platform, which aims to decode our blood cells and harness their insights to advance population and personal health, we empower individuals to identify, track and mitigate health risks. By getting ahead of their health risks, we enable the potential for a better life. In addition, through our personal biobanking service, long-term storage of your healthiest cells provides the opportunity for potential use in future therapeutics if you need them you are your best donor.

MC: Does GoodCell measure other "omics" parameters outside of genomics (DNA measurements and analysis), such as proteomics or metabolomics?SV: GoodCells platform leverages the power of blood to assess risk as such, we of course look at acquired and inherited genetic changes, but there are many more opportunities afforded by blood to understand and assess risk including routine blood chemistry tests, tests for biomarkers of disease, including emerging capabilities in liquid biopsy for earlier detection of solid tumor cancers. Ultimately, we are always looking to incorporate novel health and data insights into our product platform to better inform both an individuals health, as well as population-based health. Transcriptomics, epigenomics and metabolomics are but a few of the opportunities we are evaluating.

MC: What work is GoodCell currently conducting in the COVID-19 space?SV: GoodCell is currently engaged in a research collaboration with the New York Blood Center to evaluate how specific acquired and inherited genetic variation contribute to COVID-19 severity and recovery. We are analyzing genetic variation in asymptomatic/mildly symptomatic patients compared to hospitalized/ICU patients. GoodCell will evaluate the genetic variation in the collected samples using our proprietary assay platform to identify and validate their association with COVID-19 morbidity and mortality.

Salvatore Viscomi was speaking to Molly Campbell, Science Writer, Technology Networks.

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Exploring Genetic Variation and COVID-19 Clinical Variability - Technology Networks

Autologous Stem Cell Based Therapies Market 2020 Emerging Trend and Advancement – News by aeresearch

The recent study on the Autologous Stem Cell Based Therapies market offers a competitive advantage to organizations operating in this industry vertical through a comprehensive assessment of the present and future growth prospects.

The report explicates important facets such as primary growth catalysts, and opportunities that will ensure the revenue flow in the coming years. Further, it lists the challenges and limitations along with solutions to overcome them. Insights germane to the market share and growth rate estimates of the industry segments are also provided as well.

Apart from this, the study delves into the business scenario across the various regional markets and profiles the companies that have reigned in these geographies. Further, it highlights the prevalent strategies adopted by leading companies while simultaneously suggesting changes and new tactics for adapting to the uncertainties brought in by the Covid-19 pandemic.

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Key pointers from the TOC of the Autologous Stem Cell Based Therapies market report:

Product gamut

Application scope

Regional outlook

Competitive landscape

In conclusion, the study systematically investigates the Autologous Stem Cell Based Therapies market through various segments to provide a broad view of this business sphere. In addition, it expounds the supply chain in terms of distributors, downstream consumers, and upstream material and equipment traders in this industry.

Reasons to access this Report:

The key questions answered in this report:

Significant Point Mentioned in theResearch report:

Table of Contents for market shares by application, research objectives, market sections by type and forecast years considered:

Autologous Stem Cell Based Therapies Market Share by Key Players: Here, capital, revenue, and price analysis by the business are included along with other sections such as development plans, areas served, products offered by key players, alliance and acquisition and headquarters distribution.

Global Growth Trends: Industry trends, the growth rate of major producers, and production analysis are the segments included in this chapter.

Market Size by Application: This segment includes Autologous Stem Cell Based Therapies market consumption analysis by application.

Autologous Stem Cell Based Therapies market Size by Type: It includes analysis of value, product utility, market percentage, and production market share by type.

Profiles of Manufacturers: Here, commanding players of the global Autologous Stem Cell Based Therapies market are studied based on sales area, key products, gross margin, revenue, price, and production.

Autologous Stem Cell Based Therapies Market Value Chain and Sales Channel Analysis: It includes customer, distributor, market value chain, and sales channel analysis.

Market Forecast: This section is focused on production and production value forecast, key producers forecast by type, application, and regions

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Autologous Stem Cell Based Therapies Market 2020 Emerging Trend and Advancement - News by aeresearch

KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Demonstrated Statistically Significant Improvement in Progression-Free Survival (PFS), Overall…

KENILWORTH, N.J., & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)--Nov 10, 2020--

Merck (NYSE: MRK):

KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Demonstrated Statistically Significant Improvement inProgression-Free Survival (PFS), Overall Survival (OS) and Objective Response Rate (ORR) Versus Sunitinib as First-Line Treatment for Patients With Advanced Renal Cell Carcinoma

LENVIMA Plus Everolimus Also Showed Statistically Significant Improvement in PFS and ORR Endpoints Versus Sunitinib

Results of Investigational Phase 3 KEYNOTE-581/CLEAR Trial (Study 307) to be Presented at Upcoming Medical Meeting

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced new investigational data demonstrating positive top-line results from the pivotal Phase 3 KEYNOTE-581/CLEAR trial (Study 307). In the trial, the combinations of KEYTRUDA, Mercks anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, and LENVIMA plus everolimus were evaluated versus sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC). KEYTRUDA plus LENVIMA met the trials primary endpoint of progression-free survival (PFS) and its key secondary endpoints of overall survival (OS) and objective response rate (ORR), demonstrating a statistically significant and clinically meaningful improvement in PFS, OS and ORR versus sunitinib in the intention-to-treat (ITT) study population. LENVIMA plus everolimus also met the trials primary endpoint of PFS and a key secondary endpoint of ORR, demonstrating a statistically significant and clinically meaningful improvement in PFS and ORR versus sunitinib in the ITT study population. The ITT population included patients across all Memorial Sloan Kettering Cancer Center (MSKCC) risk groups (favorable, intermediate and poor). The safety profiles of both KEYTRUDA plus LENVIMA and LENVIMA plus everolimus were consistent with previously reported studies. Merck and Eisai will discuss these data with regulatory authorities worldwide, with the intent to submit marketing authorization applications based on these results, which will be presented at an upcoming medical meeting.

The results for KEYTRUDA plus LENVIMA versus sunitinib, which showed a statistically significant improvement in progression-free survival, overall survival and objective response rate, build on the growing scientific evidence that supports the investigation of KEYTRUDA-based combinations for the first-line treatment of advanced renal cell carcinoma, said Dr. Gregory Lubiniecki, Associate Vice President, Oncology Clinical Research, Merck Research Laboratories. Merck and Eisai are committed to working together to continue to explore the potential of the KEYTRUDA plus LENVIMA combination, particularly in areas of great unmet need such as renal cell carcinoma.

The results from KEYNOTE-581/CLEAR (Study 307) support the potential use of KEYTRUDA plus LENVIMA for the first-line treatment of advanced RCC. These data also support the potential first-line use of LENVIMA plus everolimus, which is already approved in advanced RCC following prior antiangiogenic therapy, said Dr. Takashi Owa, Vice President, Chief Medicine Creation and Chief Discovery Officer, Oncology Business Group at Eisai. These findings energize our efforts as we continue to advance our understanding and address the unmet needs of patients with difficult-to-treat cancers.

Merck and Eisai are continuing to study the KEYTRUDA plus LENVIMA combination through the LEAP (LEnvatinib And Pembrolizumab) clinical program across 19 trials in 13 different tumor types (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, RCC, squamous cell carcinoma of the head and neck, urothelial cancer, biliary tract cancer, colorectal cancer, gastric cancer, glioblastoma, ovarian cancer and triple-negative breast cancer).

About KEYNOTE-581/CLEAR (Study 307)

KEYNOTE-581/CLEAR (Study 307) is a multi-center, randomized, open-label, Phase 3 trial (ClinicalTrials.gov, NCT02811861 ) evaluating LENVIMA in combination with KEYTRUDA or in combination with everolimus versus sunitinib for the first-line treatment of patients with advanced RCC. The primary endpoint is PFS by independent review per RECIST v1.1 criteria. Key secondary endpoints include OS, ORR and safety. The study enrolled approximately 1,050 patients who were randomized to one of three treatment arms to receive:

About Renal Cell Carcinoma (RCC)

Worldwide, it is estimated there were more than 403,000 new cases of kidney cancer diagnosed and more than 175,000 deaths from the disease in 2018. In the U.S. alone, it is estimated there will be nearly 74,000 new cases of kidney cancer diagnosed and almost 15,000 deaths from the disease in 2020. Renal cell carcinoma is by far the most common type of kidney cancer; about nine out of 10 kidney cancers are RCCs. Renal cell carcinoma is about twice as common in men as in women. Most cases of RCC are discovered incidentally during imaging tests for other abdominal diseases. Approximately 30% of patients with RCC will have metastatic disease at diagnosis, and as many as 40% will develop metastases after primary surgical treatment for localized RCC. Survival is highly dependent on the stage at diagnosis, and with a five-year survival rate of 12% for metastatic disease, the prognosis for these patients is poor.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA (pembrolizumab)

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

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KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Demonstrated Statistically Significant Improvement in Progression-Free Survival (PFS), Overall...

Global Progenitor Cell Product Professional Survey 2020 by Manufacturers, Regions, Types and Applications, Forecast to 2026 – Zenit News

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Global Progenitor Cell Product Professional Survey 2020 by Manufacturers, Regions, Types and Applications, Forecast to 2026 - Zenit News

Merck Announces KEYNOTE-598 Trial Evaluating KEYTRUDA (pembrolizumab) in Combination With Ipilimumab Versus KEYTRUDA Monotherapy in Certain Patients…

KENILWORTH, N.J.--(BUSINESS WIRE)--Nov 9, 2020--

Merck Announces KEYNOTE-598 Trial Evaluating KEYTRUDA (pembrolizumab) in Combination With Ipilimumab Versus KEYTRUDA Monotherapy in Certain Patients With Metastatic Non-Small Cell Lung Cancer To Stop for Futility and Patients to Discontinue Ipilimumab

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that it will be stopping KEYNOTE-598, a Phase 3 trial investigating KEYTRUDA, Mercks anti-PD-1 therapy, in combination with ipilimumab (Yervoy ), compared with KEYTRUDA monotherapy, for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (tumor proportion score [TPS] 50%) with no EGFR or ALK genomic tumor aberrations. Merck is discontinuing the study following the recommendation of an independent Data Monitoring Committee (DMC), which determined the benefit/risk profile of the combination did not support continuing the trial. At an interim analysis, the combination of KEYTRUDA and ipilimumab showed no incremental benefit in overall survival (OS) or progression-free survival (PFS), the studys dual primary endpoints, compared with KEYTRUDA alone and crossed futility boundaries. No new safety signals for KEYTRUDA monotherapy were observed, however the combination of KEYTRUDA and ipilimumab was associated with a higher incidence of grade 3-5 adverse events (AEs), serious AEs, and AEs leading to discontinuation or death, compared with KEYTRUDA monotherapy. Merck will inform study investigators of the recommendation from the DMC and the DMC is advising that patients in the study discontinue treatment with ipilimumab/placebo. Data from this study will be submitted for presentation at an upcoming scientific congress and communicated to regulatory agencies.

We conducted KEYNOTE-598 in order to explicitly explore whether combining our anti-PD-1 therapy, KEYTRUDA, with ipilimumab provided additional benefits beyond treatment with KEYTRUDA alone in the metastatic non-small cell lung cancer setting, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. It is very clear that in this study, the addition of ipilimumab did not add clinical benefit but did add toxicity. KEYTRUDA monotherapy remains a standard of care for the treatment of certain patients with metastatic non-small cell lung cancer whose tumors express PD-L1.

While the combination of an anti-PD-1 therapy plus ipilimumab has been approved in certain indications, studies supporting these approvals have, for the most part, not compared the combination directly with anti-PD-1 monotherapy. Bristol Myers Squibb has reported topline results of CheckMate-915, a Phase 3 study in adjuvant melanoma that directly compared treatment with ipilimumab in combination with an anti-PD-1 therapy versus the anti-PD-1 therapy alone. In two separate news releases issued over the last year, the company announced the study did not meet its co-primary endpoints in the all-comer population or in patients whose tumors expressed PD-L1 <1%. These data have not yet been presented.

Merck has an extensive clinical development program in lung cancer and is advancing multiple registration-enabling studies with KEYTRUDA in combination with other treatments and as monotherapy. The lung program is evaluating KEYTRUDA across all stages of disease and lines of therapy in over 200 trials with more than 10,000 patients.

About KEYNOTE-598

KEYNOTE-598 (ClinicalTrials.gov, NCT03302234 ) is a randomized, double-blind, Phase 3 trial investigating KEYTRUDA in combination with ipilimumab compared to KEYTRUDA monotherapy for the first-line treatment of patients with metastatic NSCLC whose tumors express PDL1 (TPS 50%) with no EGFR or ALK genomic tumor aberrations. The dual primary endpoints are OS and PFS. Secondary endpoints include objective response rate, duration of response and safety. The study enrolled 568 patients who were randomized (1:1) to receive:

About Lung Cancer

Lung cancer, which forms in the tissues of the lungs, usually within cells lining the air passages, is the leading cause of cancer death worldwide. Each year, more people die of lung cancer than die of colon and breast cancers combined. The two main types of lung cancer are non-small cell and small cell. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for about 85% of all cases. Small cell lung cancer (SCLC) accounts for about 10% to 15% of all lung cancers. Before 2014, the five-year survival rate for patients diagnosed in the U.S. with NSCLC and SCLC was estimated to be 5% and 6%, respectively.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients. Pneumonitis occurred in 8% (31/389) of patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and post-marketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/PD-L1 blocking antibody. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/PD-L1 blockade and allogeneic HSCT. Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risk of treatment with a PD-1/PD-L1 blocking antibody prior to or after an allogeneic HSCT.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients with advanced NSCLC; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

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Merck Announces KEYNOTE-598 Trial Evaluating KEYTRUDA (pembrolizumab) in Combination With Ipilimumab Versus KEYTRUDA Monotherapy in Certain Patients...

Advancement: Know the Rapid Growth Factors of Biopreservation Market| Stay Up-To-Date With Emerging – PharmiWeb.com

Pune, Maharashtra,India, November 9 2020 (Wiredrelease) Origius Systems Private Limited :The increasing occurrence of chronic ailments and obesity all over the world is driving the global biopreservation market growth. The North America region is expected to lead the market growth in the projected period.

A latest report by Research Dive on the global biopreservation market reveals that the market is projected to hit $13,576.3 million by 2027, rising at a CAGR of 13.8% from 2020 to 2027. The report states the current outlook and future growth of the market. The research report is a perfect source of guidance for companies and individuals interested in investing in the market.

The report covers the following aspects:

A brief introduction of the market with its definition, advantages, and application areas. Inclusive insights on the market situation, dynamics, statistics, growth rate, revenues, market shares, and future predictions. Major market segments, drivers, limitations, and investment suitability. Current scenario of the global and regional market from the perspective of companies, countries, and end industries. Insights on foremost market players, current market trends & developments, SWOT Analysis, Porter Five Analysis, and winning business strategies.

Connect with Our Analyst to Contextualize Our Insights for Your Business:https://www.researchdive.com/connect-to-analyst/369

Factors Impacting the Market Growth:

According to the report, growing occurrence of chronic ailments like diabetes, cardiac, degenerative conditions affecting the joints, nerves, bones, and others as well as obesity all over the world are thrusting the demand for biopreservation techniques, which is fueling the growth of the market. Additionally, the increasing investments in R&D and development of advanced biopreservation products is expected to unlock rewarding opportunities for the market growth. However, greater costs involved in biopreservation processes, reliability concerns, and invention of low-priced processes are likely to detain the biopreservation market growth.

Segment Analysis:

The report segments the biopreservation market into type, biospecimen, application, end use, and region.

Based on biospecimen, the report divides the market into: Stem Cells Human Tissue Organs

Among these, the human tissue segment is expected to witness highest growth in the biopreservation market all through the projected period; mainly due to rising cases of chronic ailments, degenerative disorders, and obesity.

Based on application, the report classifies the market into: Therapeutic Research Clinical Trials

Among these, the therapeutic segment is projected to show noteworthy growth during the forecast period. This is mainly owing to the developments in treatment techniques, personalized drugs, regenerative drugs, increasing trend for cord blood banking, and increasing occurrence of chronic ailments worldwide.

Download Exclusive Free Sample Copy of the Report at:https://www.researchdive.com/request-toc-and-sample/369

Regional Analysis:

The report states the scenario of the global biopreservation market across several regions including: Europe LAMEA North America Asia Pacific

Among these, the North America region is projected to grab the highest market share of the biopreservation market in the forecast period. This is attributed to the growing awareness about personalized drugs, rising R&D in regenerative medicines, increasing prevalence of obesity, and chronic ailments.

Market Players and Business Strategies:

The report cites some of the leading players in the global biopreservation market which includes: Avantor, Inc. Bio-Techne Exact Sciences Corporation Merck KGaA ThermoGenesis Corp. BioLifeSolutions Inc. BioCision Chart Industries Thermo Fisher Scientific Inc. Worthington Industries, Inc.

The report highlights some of the wining business strategies of the players such as mergers and acquisitions, ground-breaking advances, geographical expansions, new product inventions, and many more.Quick Download Top Companies Development Strategies Summary Report

Abouat Us:

Research Dive is a market research firm based in Pune, India. Maintaining the integrity and authenticity of the services, the firm provides the services that are solely based on its exclusive data model, compelled by the 360-degree research methodology, which guarantees comprehensive and accurate analysis. With unprecedented access to several paid data resources, team of expert researchers, and strict work ethic, the firm offers insights that are extremely precise and reliable. Scrutinizing relevant news releases, government publications, decades of trade data, and technical & white papers, Research dive deliver the required services to its clients well within the required timeframe. Its expertise is focused on examining niche markets, targeting its major driving factors, and spotting threatening hindrances. Complementarily, it also has a seamless collaboration with the major industry aficionado that further offers its research an edge.

Contact Us:

Mr. Abhishek PaliwalResearch Dive30 Wall St. 8th Floor, New YorkNY 10005 (P)+ 91 (788) 802-9103 (India)+1 (917) 444-1262 (US) TollFree : +1 -888-961-4454Email:support@researchdive.comLinkedIn:https://www.linkedin.com/company/research-diveTwitter:https://twitter.com/ResearchDiveFacebook:https://www.facebook.com/Research-DiveBlog:https://www.researchdive.com/blogFollow us on:https://covid-19-market-insights.blogspot.com

This content has been published by Origius Systems Private Limited company. The WiredRelease News Department was not involved in the creation of this content. For press release service enquiry, please reach us at contact@wiredrelease.com.

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Advancement: Know the Rapid Growth Factors of Biopreservation Market| Stay Up-To-Date With Emerging - PharmiWeb.com

Stem Cell Therapy Market is estimated to be worth USD 8.5 Billion by 2030 – PRnews Leader

The success of approved stem cell therapies has caused a surge in interest of biopharma developers in this field; many innovator companies are currently progressing proprietary leads across different phases of clinical development, with cautious optimism

Roots Analysis has announced the addition of Global Stem Cells Market: Focus on Clinical Therapies, 20202030 (Based on Source (Allogeneic, Autologous); Origin (Adult, Embryonic); Type (Hematopoietic, Mesenchymal, Progenitor); Lineage (Amniotic Fluid, Adipose Tissue, Bone Marrow, Cardiosphere, Chondrocytes, Corneal Tissue, Cord Blood, Dental Pulp, Neural Tissue Placenta, Peripheral Blood, Stromal Cells); and Potency (Multipotent, Pluripotent)) report to its list of offerings.

There is a growing body of evidence supporting the vast applicability and superiority of treatment outcomes of stem cell therapies, compared to conventional treatment options. In fact, the unmet needs within this domain have spurred the establishment of many start-ups in recent years.

To order this 500+ page report, which features 185+ figures and 220+ tables, please visit this link

Key Market Insights

Over 280 stem cell therapies are under development, most of which are allogeneic productsMore than 50% of the pipeline candidates are in the mid to late phase trials (phase II and above), and allogenic therapies (majority of which are derived from the bone marrow) make up 65% of the pipeline.

70% of pipeline candidates are based on mesenchymal stem cellsIt is worth highlighting that the abovementioned therapies are designed to treat musculoskeletal (22%), neurological (21%) and cardiovascular (15%) disorders. On the other hand, hematopoietic stem cell-based products are mostly being evaluated for the treatment of oncological disorders, primarily hematological malignancies.

Close to 85% stem cell therapy developers are based in North America and Asia-Pacific regionsWithin these regions, the US, China, South Korea and Japan, have emerged as key R&D hubs for stem cell therapies. It is worth noting that majority of the initiatives in this domain are driven by small / mid-sized companies

Over 1,500 grants were awarded for stem cell research, since 2015More than 45% of the total amount was awarded under the R01 mechanism (which supports research projects). The NCI, NHLBI, NICHD, NIDDK, NIGMS and OD emerged as key organizations that have offered financial support for time periods exceeding 25 years as well.

Outsourcing has become indispensable to R&D and manufacturing activity in this domainPresently, more than 80 industry / non-industry players, based in different regions across the globe, claim to provide contract development and manufacturing services to cater to the unmet needs of therapy developers. Examples include (in alphabetical order) Bio Elpida, Cell and Gene Therapy Catapult, Cell Tech Pharmed, GenCure, KBI Biopharma, Lonza, MEDINET, Nikon CeLL innovation, Roslin Cell Therapies, WuXi Advanced Therapies and YposKesi.

North America and Asia-Pacific markets are anticipated to capture over 80% share by 2030The stem cell therapies market is anticipated to witness an annualized growth rate of over 30% during the next decade. Interestingly, the market in China / broader Asia-Pacific region is anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 8.5 billion (by 2030) financial opportunity within the stem cell therapies market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom stem cell therapies are currently considered to be a promising alternatives for the treatment of a myriad of disease indications, with the potential to overcome challenges associated with conventional treatment options. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles of several companies (including those listed below); each profile features an overview of the company, financial information (if available), stem cell therapy portfolio and an informed future outlook.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/stem-cells-market/296.html

or email [emailprotected]

You may also be interested in the following titles:

Contact:

Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Stem Cell Therapy Market is estimated to be worth USD 8.5 Billion by 2030 - PRnews Leader

California Prop 14 would keep taxpayer funding of stem cell therapy research, which can change lives: Meet the Langenhop kids – AZCentral.com

18-year-old Keely Campbell pays it forward by donating her stem cells 10 years after her dad's transplant. USA TODAY

Three-year-old Ava was constantly sick. Her gums were inflamed, and every time she got a scraped knee, it turned into a dangerous infection.

Her parents, Alicia and Jon Langenhop, were months pregnant with their third child when they learned that Ava's constellation of symptoms added up to an extremely rare, inherited disorder of the white blood cells, called leukocyte adhesiondeficiency-1. Although antibiotics and antivirals could prolong her life, the disease was considered fatal, usually before kindergarten.

Ava's primary hope, doctors told the Langenhops, was a bone marrow transplant from someone who was a good match, probably a brother or a sister.

Two-year-old Olivia had inherited the same disease as her big sister. She had been hospitalized with infections, too.

The baby in Alicias belly would be the girls best hope. Since both parents were carriers of the rare genetic mutation, the new baby, a boy, had a 25% chance of inheriting it, too.

Alicia was still in the hospital last October when they found out baby Landon had the mutation. Around the same time, the couple learned of a research trial in California.

Children Ava, Olivia and Landon Langenhop were diagnosed with an extremely rare, inherited disorder of the white blood cells, called leukocyte adhesiondeficiency-1. California Proposition 14, a citizen-initiated ballot measure, authorizes bonds continuing stem cell research.(Photo: Harrison Hill, USA TODAY)

Doctors would take each childs blood cells, fix the mutationand return them. It should be a permanent fix, with less risk than a bone marrow transplantbecause the healthy cells would be their own, so their bodies wouldnt reject them as foreign.

The approach had been tried in only one child, though.

This is the type of research reaching patients nearly two decades after President George W. Bush banned federal funding of stem cell researchand 16 years after California residents approved a tax increase on themselves to support research.

Proposition 14 on Tuesday's ballot askswhether Californians want to continue this work, providing $5.5 billion for stem cell research over the next three decades.

In the early 2000s, stem cell research was controversial because it often required the destruction of human embryos. Though embryonic stem cells remain essential for some therapies, in cases such as the Langenhops', treatment focuses on manipulating a persons own cells.

Stem cell science has made tremendous progress, but as in most new fields, the pace remains painstakingly slow. Every treatment has to be the subject of years of trial-and-error research, and many scientific hurdles linger.

Stem cells have been used to treat rare diseases, such as severe combined immunodeficiency, also known as "bubble boy disease," and they are being tested in more common conditions such as Parkinson's disease, macular degeneration, Type 1 diabetesand even heart disease.

"Even if a subset of stuff in the pipeline goes all the way, it will change the world for patients who currently don't have other good options," said Sean Morrison, a stem cell biologist in Dallas.

"It's a pivotal time in the field," said Dr. Deepak Srivastava, president of the Gladstone Institutes, a nonprofit research organization based in San Francisco. "We can feel that we're at an inflection point, where after years of discovery and creating a foundation of knowledge, we're finally at a point where a number of approaches are reaching clinical trials."

The Langenhop Family temporarily relocated from Canton, Ohio, to Los Angeles, so their children could receive stem cell treatment for leukocyte adhesion deficiency-1.(Photo: Harrison Hill, USA TODAY)

Stem cell therapies haven't advanced as far as some clinics offering expensive treatments claim.

Getting stem cells injected into your knee, for example, hasn't been scientifically proven safe or effective but some clinics charge thousands of dollars for the procedure.

"There are many clinics throughout the world,including in the United States, that continue to market unproven stem cell therapies," said Timothy Caulfield, a professor of law and public health at the University of Alberta in Canada. "They leverage the excitement that has surrounded this area to push products that aren't supported by good science. Indeed, there still aren't many stem cell therapies that are clearly ready for broad clinical application."

One way to distinguish the good science from the scams, Morrison said, is to look at what's promised. Anyone who offers to treat differentailments with the same "multipurpose" cells is selling snake oil, he said.

There are a wide variety of stem cells embryonic stem cells, induced pluripotent stem cells, somatic cells, etc, he said and it's challenging to figure out which to use against which disease.

"Every disease is a different set of challenges, and we spend years trying to develop therapies that are safe and effective," said Morrison, who chairs the public policy committee of the International Society for Stem Cell Research, a scientific organization. "When a company comes out making claims about a one-size-fits-all therapy that can treat everything, that's a sure sign that they're just trying to take your money."

Several researchers interviewed by USA TODAY said state funding helped put California at the forefront of global stem cell research.

George Daley, a stem cell biologist who is dean of Harvard Medical School, said he's envious of the California researchers who have access to this pot of money.

"California has always been a very exciting place to pursue science, but prior to (the taxpayer funding), it wasn't exactly the place that was the first on the tip of your tongue as a powerhouse community for stem cell science," he said. "But there's no way that today it wouldn't be listed in the top three."

Daley, a global leader in the field, said he thinks the funding spurred enthusiasm for stem cell research more broadly. "The entire excitement around the field of stem cell biology is part of the reason you're seeing cures in cancer and impending cures in devastating diseases like sickle cell anemia,"he said.

Although stem cell therapy hasn't gotten anyone out of a wheelchair yet, he said important research continues that may eventually transform paralyzed patients' lives. "If it's taken us longer than people anticipated, that's just the pace of science," he said.

Research begun in the Harvard lab of Doug Melton may soon revolutionize the treatment of Type 1 diabetes, he said, liberating patients from needing to inject themselves daily with insulin.

Srivastava, a cardiologist, admittedthat the field, backed by federal funding, spent more than a decade chasing cardiac stem cell treatments that never succeeded, possibly because they weren't stem cells at all.

Now, his lab and others have modified true stem cells into beating heart cells, he said, which should one day be able to restore tissue damaged by a heart attack.

The big bottleneck in the field, Srivastava said, is getting cells to fully mature in a dish. Scientists can turn stem cells into immature cells of many different types neurons, heart cells, retinal cells but they get stuck in this fetal-like state.

Beating heart cells derived from stem cells can create rhythm disturbances because they're electrically different from normal, mature cells, he said.

"It would be beneficial to have a cell that could function more like a cell after birth," Srivastava said.

Donald Kohn, the Langenhops' doctor at the University of California, Los Angeles, has been involved in stem cell research since 1985.

"I've seen it go from a dream, a vision, to now just various degrees of how it's working," said Kohn, also a member of the UCLA Broad Stem Cell Research Center. About a dozen blood diseases such as LAD-1 have shown "very good clinical responses" to the type of treatment the Langenhop children received.

The children are missing a proteinin their white blood cells that would allow the cellsto reach a cut or damaged tissue. The children can't clear infections and suffer from inflammation and other immune-related problems.

Kohn's study of kids with LAD-1 mutations will eventually include nine patients. Depending on the results, the companysponsoring the trial, Rocket Pharmaceutics of New York, will ask the U.S. Food and Drug Administration for a license to sell the therapy.

Donald Kohn, the Langenhops' UCLA doctor, calls the parents "heroic."(Photo: Harrison Hill, USA TODAY)

He said he's been blown away by how well the Langenhops have dealt with the trials and tribulations of the past year.

"I just think they're heroic, what these parents are doing," Kohn said.

At first, the Langenhops asked themselves how a disease that strikes fewer than 1 in a million children could have struck them three times.

But now, more than a year after Ava's diagnosis and six months after temporarily moving to California for the treatments, Alicia and Jon just want to bring three healthy kids home to Canton, Ohio.

They flew to California for the first time last December so doctors could collect bone marrow cells from Ava. They'd planned to come back in February for the treatment, but not enough cells had been repaired, so the February trip was just a repeat of December.

By the time they were ready to come back to UCLA Mattel Children's Hospital for her therapy in late April, the pandemic had struck. The airplane was nice and empty.

Ava Langenhop receives a stem cell treatment for an extremely rare, inherited disorder of the white blood cells, called leukocyte adhesion deficiency-1.(Photo: Courtesy of UCLA)

Ava got chemotherapy twice a day for five days to kill off her faulty white blood cells. After a day of rest, she was given back her own blood stem cells, gene-edited to remove her damaging LAD-1 mutation.

Her one complaint: the therapy smelled like creamed corn and made her gag.

She recovered quickly, and after about a month in the hospital, enough white blood cells had grown back for her to safely return to the family's rental apartment, just off the UCLA campus, where her mom has turned the living room into a classroom for the girls.

Although she lost all her hair from chemo, it's slowly growing back, and she's very proud of her cropped hairstyle, Alicia said. She picks out a bow every day to match her outfit.

At Ava's three-month checkup this summer, doctors found her bone marrow was producing healthy white blood cells. Her LAD-1 is considered eliminated and she'll never need another treatment.

"She'll probably be healthy forever," Kohn said.

Landon, age 1, completed his treatment about a month ago and was allowed out of the hospital last week. He's bald, but his blue eyes still sparkle, and as he wavedthrough the camera on a recent Zoom call, it's hard to imagine a cuter, healthier-looking baby.

Although originally slated for treatment before her brother, Olivia, 3, ended up last because, as with her sister, the processing of her cells didn't work well the first time. She went to the hospital Sunday and starts her chemo Monday night. She is scheduled to receive the corrected cells next week.

Landon Langenhop receives his stem cell treatment for leukocyte adhesion deficiency-1.(Photo: Courtesy of UCLA)

Olivia was excited to start her therapy, to be "part of the club" with her sister and brother, Alicia said. "She can't wait to turn bald."

The cost of all three children'streatments and the rental apartment and plane fare are being picked up by Rocket Pharmaceuticals. That's the upside of volunteering for clinical trials: the treatment is paid for by the sponsor.

Jon was a bank branch manager before the pandemic and now works remotely for the same bank, reviewing loan applications. "It gives me the chance to be at the hospital with whichever child is in the hospital at that time," Jon said, chuckling.

Alicia, a former teacher and day care center assistant manager, has been home-schooling the kids since the start of the pandemic.

Their doctor hopes to have the family back in Canton for Christmas or at least New Year's. The weather won't be as nice as in Southern California, but they'll be home, surrounded by their supportive family and friends. And Jon and Alicia won't have to worry anymore about every scrape and sniffle.

They don't focus much on the details of how the science works. They're just happy it has.

"We feel so fortunate to be living in a time when this is possible," Alicia said.

If they had been in this situation even a few years ago, the treatment wouldn't have existed, Jon said. If they had found out about Ava's mutation when she was younger, they wouldn't have had two more children.

"It's kind of crazy how it's all worked out for us," he said.

The strength of their kids has given them strength. "They just go with the flow. It's kind of incredible how resilient they are and can just bounce back from these hospitalizations," Jon said.

"Like nothing ever happened," Alicia added.

The three children will probably have few if any memories of their disease and treatment. For Jon and Alicia, however, this year is seared into their memory.

"It'll take us a while to realize they don't have this anymore," Jon said.

Karen Weintraub can be reached at kweintraub@usatoday.com.

Health and patient safety coverage at USA TODAY is made possible in part by a grant from the Masimo Foundation for Ethics, Innovation and Competition in Healthcare. The Masimo Foundation does not provide editorial input.

The Langenhop Family plays at the Franklin D. Murphy Sculpture Garden at UCLA. Children Ava, Olivia and Landon were diagnosed with an extremely rare, inherited disorder of the white blood cells, called leukocyte adhesiondeficiency-1. California Proposition 14, a citizen-initiated ballot measure that will appear on the ballot in the 2020, authorizes Bonds continuing Stem Cell Research. A Yes vote will allow researchers to continue studying and working towards finding cures for individuals like Ava, Olivia and Landon.(Photo: Harrison Hill, USA TODAY)

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California Prop 14 would keep taxpayer funding of stem cell therapy research, which can change lives: Meet the Langenhop kids - AZCentral.com

Caelum and Alexion Announce Upcoming Data Presentations at the 62nd American Society of Hematology Annual Meeting and Exposition – Business Wire

BORDENTOWN, N.J. & BOSTON--(BUSINESS WIRE)--Caelum Biosciences and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that two abstracts on CAEL-101, a first-in-class amyloid fibril targeted therapy, have been accepted for presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5 to 8, 2020. New data, from Cleveland Clinic, will be presented on the safety, efficacy and tolerability of CAEL-101 in combination with standard-of-care therapy in AL amyloidosis from the Phase 2 open-label dose escalation study that suggest early evidence of organ response. Data, from Caelum, that further demonstrate the safety and tolerability of CAEL-101 and support the selection of the 1000 mg/m2 dose for the Phase 3 study will also be presented.

The accepted abstracts are listed below and are now available on the ASH website:

Oral Presentation

Safety, Tolerability and Efficacy of CAEL-101 in AL Amyloidosis Patients Treated on a Phase 2, Open-Label, Dose Selection Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis. Abstract #729. An oral symposium presentation is scheduled for December 7, 2020, 2:45 p.m. PST.

ePoster Presentation

CAEL-101 is Well-Tolerated in AL Amyloidosis Patients Receiving Concomitant Cyclophosphamide-Bortezomib-Dexamethasone (CyborD): A Phase 2 Dose-Finding Study (NCT04304144), Abstract #2277 poster presentation, poster session II, December 6, 2020, 7:00 a.m. - 3:30 p.m. PST.

As was previously announced, the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101 in combination with standard-of-care (SoC) therapy in AL amyloidosis has begun. Enrollment is underway in two parallel Phase 3 studies one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease and will collectively enroll approximately 370 patients globally. The Phase 2 program continues with the addition of a study arm to evaluate CAEL-101 in combination with SoC therapy plus daratumumab.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a potential therapy for patients with AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of systemic amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About Caelum Biosciences

Caelum Biosciences, Inc. (Caelum) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelums lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (AL) amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.caelumbio.com.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: http://www.alexion.com.

[ALXN-P]

Forward-Looking Statement

This press release may contain forward-looking statements, including as such term is defined within Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to Caelums or Alexions growth strategy and our respective product development programs, plans related to clinical trials (including, commencement, completion and future patient enrollment), the anticipated benefits of CAEL-101 and any other statements that are not historical facts. Forward-looking statements are based on Caelum and Alexion managements current expectations and are subject to risks and uncertainties that could negatively affect each of our businesses, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks that CAEL-101 is not shown to be safe and effective in clinical trials or does not receive regulatory approval to be marketed, risks relating to each companys growth strategy; ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing (including, commencement, completion and future patient enrollment); risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; risks relating to the COVID-19 outbreak and its potential impact on each Companys employees and consultants ability to complete work in a timely manner and on the ability to obtain additional financing on favorable terms or at all; our ability to attract, integrate and retain key personnel; the early stage of products under development; Caelums need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in each Companys SEC filings. Alexion and Caelum expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our respective expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 or any other protections afforded by applicable law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

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Caelum and Alexion Announce Upcoming Data Presentations at the 62nd American Society of Hematology Annual Meeting and Exposition - Business Wire

Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over2017 2025 – Royal Sutton News

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over2017 2025 - Royal Sutton News

Autologous Stem Cell Based Therapies Market to Witness Increase in Revenues by 2020-2026 – PRnews Leader

Beathan Report has released the International report on The Autologous Stem Cell Based Therapies market, which is made up of advice about each of the essential parameters of this market like ingestion and the manufacturing patterns coupled with all the earnings patterns for the prediction period. Concerning creation aspect, the report provides complete detailed analysis about the manufacturing procedures combined with the gross financials accumulated by the very best most producers working within this business. The main facet of this Autologous Stem Cell Based Therapies market thats covered in the report helps the customers and the associations to better comprehend the company profile concerning drivers, restraints, challenges, and opportunities affecting and pertaining the market dynamics.

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Key market players

Major competitors identified in this market include Regeneus, Mesoblast, Pluristem Therapeutics Inc, US STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe, etc.

Based on the Region:

Asia-Pacific (China, Japan, South Korea, India and ASEAN)

North America (US and Canada)

Europe (Germany, France, UK and Italy)

Rest of World (Latin America, Middle East & Africa)

COVID-19 has affected the Overall worldwide companies and itll have a enormous time for the company recovery. Vast majority of the business sectors have realigned their company plans, priorities, and have amended their economic planning so as to stay in the company and keep their standing on the international platform. The thorough evaluation of this Autologous Stem Cell Based Therapies market will enable the brand new market entrants to acquire reliable market approaches and strategy powerful action plans for the prediction period.

Based on the Type:

Embryonic Stem Cell

Resident Cardiac Stem Cells

Umbilical Cord Blood Stem Cells

Based on the Application:

Neurodegenerative Disorders

Autoimmune Diseases

Cardiovascular Diseases

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Important highlights of this Autologous Stem Cell Based Therapies market report:

* COVID-19 effect on the earnings Streams of the Autologous Stem Cell Based Therapies market players.

* Statistics of the overall sales quantity And general market earnings.

* Business trends breakdowns.

* Estimated expansion rate of this Autologous Stem Cell Based Therapies Market.

* In-depth Information Regarding the important Distributors, traders, and dealers.

Key Benefits of the report:

-This report provides an extensive analysis of the current and emerging market trends and dynamics in the global Autologous Stem Cell Based Therapies market.

-In-depth analysis is conducted by constructing market estimations for the key market segments between 2020 and 2027.

-This report entails the detailed quantitative analysis of the current market and estimations through 2020-2027, which assists in identifying the prevailing market opportunities.

-Extensive analysis of the market is conducted by following key product positioning and monitoring the top competitors within the market framework

-Comprehensive analysis of all regions is provided that determines the prevailing opportunities in these geographies.

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Autologous Stem Cell Based Therapies Market to Witness Increase in Revenues by 2020-2026 - PRnews Leader

Orphan Drug Designation Granted for CSL Behring’s Investigational Plasma-Derived Hemopexin Therapy for Sickle Cell Disease – PRNewswire

KING OF PRUSSIA, Pa., Nov. 2, 2020 /PRNewswire/ --Global biotherapeutics leader CSL Behring announced today that its investigational, plasma-derived hemopexin therapy (CSL889) received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of sickle cell disease (SCD). These designations grant special status to drugs and biological products intended to treat a rare disease, affecting less than 200,000 patients in the US or affecting not more than five in 10,000 people in the European Union.

CSL889 is a form of plasma-derived hemopexin, an important, naturally occurring protein produced in the body whose levels are decreased in patients with SCD. Low levels of hemopexin have been associated with increased symptoms in SCD, particularly acute vaso-occlusive crises (VOC). VOC, the most common manifestation in SCD, are severe, debilitating episodes characterized by severe pain. There is no approved treatment for acute VOC, so episodes can only be managed with supportive measures such as fluids and pain killers.

"Having treated hundreds of adults and children living with sickle cell disease over 30 years, I'm intensely aware of the need for novel and effective therapies, especially to relieve the tremendous pain from VOC," said Professor Greg Kato, who is leading the clinical development of CSL 889 at CSL Behring. "This newly granted orphan status recognizes the urgency for progressing new treatment options into the clinic."

CSL Behring has two Phase I SCD programs poised to evolve the treatment paradigm for patients: CSL889 hemopexin therapy for the treatment of VOC and CSL200 lentiviral stem cell gene therapy for long-term disease management.

About Sickle Cell Disease

Sickle Cell Disease is a hereditary blood disorder in which red blood cells contain an abnormal type of hemoglobin, causing some of the cells to become distorted into a crescent, or sickle-shape. These misshapen red blood cells have difficulty passing through small blood vessels, slowing and blocking blood flow to areas of the body, damaging tissue that isn't receiving a normal flow of blood. Sickle Cell Disease can lead to episodes of severe pain, strokes, kidney, lung and heart problems, slow growth, vision problems and infection vulnerability. While frequency of Sickle Cell Disease varies globally, it is estimated to impact 100,000 people in the US and 1 in 10,000 persons in the European Union.

About CSL Behring

CSL Behringis a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 27,000 people, anddelivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vita and follow us on Twitter.com/CSLBehring.

SOURCE CSL Behring

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