CARLSBAD, Calif.--(BUSINESS WIRE)--Accelerated Biosciences, a regenerative medicine innovator, announced today new data that further demonstrates statistically significant cytolysis with induced pluripotent stem cell (iPSC)-derived natural killer (NK) cells programmed from its ethically sourced human trophoblast stem cells (hTSCs). Pluristyx, a Seattle-based firm supporting drug development, regenerative medicine, and cell and gene therapies, further confirmed Accelerated Biosciences hTSC line offers before-unrealized opportunities in cell-specific therapeutics. Along with this recent data on successful iPSC differentiation, Accelerated Biosciences has already demonstrated efficient differentiation of its pluripotent stem cells with remarkable doubling times and growth characteristics to programmed NK, cartilage, bone, fat, neuron, pancreas, liver, and secretome cells.

This new data validates our findings, explains Yuta Lee, President and Founder of Accelerated Biosciences. We know the properties of our trophoblast stem cells have been long-sought by the medical science community because of the potential to speed and amplify the development of life-saving therapeutics; theyre immune privileged, chromosomally stable (not tumorigenic), pathogen free, pluripotent, easy to scale and manufacturer, and of special interest, they are ethically sourced from the chorionic villi (pre-placental tissue) of non-viable and often life-threatening tubal ectopic pregnancies. Mr. Lees father, Professor Jau-Nan Lee, MB, MD, PhD, an obstetrics and gynecologic physician and researcher in Taiwan, first isolated hTSC in 2003. Mr. Lee created Accelerated Biosciences to elevate the visibility of this pluripotent human trophoblast stem cell platform to those engaged in developing allogeneic cell therapeutics and has been instrumental in the filing and prosecution of intellectual property to protect the companys hTSC platform to date holding 34 patents.

Benjamin Fryer, PhD, Co-founder and CEO of Pluristyx, worked closely with Accelerated Biosciences to prepare much of its key hTSC data. Dr. Fryer, a trophoblast expert who was previously a research scientist at Janssen Research & Development of Johnson & Johnson, now serves on Accelerated Biosciences Scientific Advisory Board. Initially I was skeptical these cells were what they said they were. If we hadnt grown and characterized them in our lab, I might have remained skeptical. These are indeed trophoblast stem cells, explained Dr. Fryer. The potential of these cells is enormous. One of the industrys largest challenges is that its almost impossible to scale primary cells. These cells are scalable. With these cells you can make the amount required for millions of patients and theyre sourced compliant to regulatory requirements. Weve made IPS cells (induced pluripotent stem cells) and NK (natural killer) cells from them, which is the next wave of cells for cell therapies. For therapeutic developers, because these cells are not sourced from a person or viable embryo, these cells deliver the trifecta of legal, ethical, and IP advantages.

As the biotechnology industry works toward developing therapies that target only diseased cells without harming healthy cells and tissues, cell-based therapies draw increasing interest, explains industry expert, Martina Molsbergen, CEO of C14 Consulting, who has partnered with Accelerated Biosciences in a business development role. With all the promise that cell therapies hold, the biotechnology industry also remains concerned that the therapeutics are derived in a socially and ethically responsible manner. Accelerated Biosciences has discovered and is now offering what scientists see as the holy grail of stem cell sources.

Prominent biosciences experts have been drawn to Accelerated Biosciences cell breakthrough. Protein chemist and molecular biologist Igor Fisch, PhD, former President and CEO of Selexis, Geneva, Switzerland, recognizes the impact that Accelerated Biosciences hTSCs will have on human health: Not only are these cells politically correct, but they can also differentiate. Because they are sourced from pre-placenta material, theyre immune privileged, which means that are not seen as foreign by the human body. With these cells, we can create a cell bank a single source for a wide range of patients.

Peter Hudson, FTSE, BSc Hons, PhD, Chief Scientist and a senior advisor to Avipep P/L in Melbourne, Australia, and an adjunct professor at the University of Queensland, led a large oncology consortium to complete the first Phase 1 clinical trial of a novel engineered antibody targeting prostate and ovarian cancer. Hudsons interest in Accelerated Biosciences hTSCs has evolved into a role on its Scientific Advisory Board. Trophoblast stem cells are likely to be the next wave of cancer-targeting therapeutics, explains Dr. Hudson. The ability to ethically source trophoblast stem cells and program them to target only diseased, cancerous cells is very powerful technology.

Why are scientists so interested in stem cell-based therapies?

The human body constantly produces specialized cells from its own stem cells (undifferentiated cells) to renew and repair itself. Current therapies harness this power in autologous cell therapies in which the patients own cells are removed, differentiated into disease-fighting cells, and reinserted.

What makes the human trophoblast stem cell so important to medical science?

The human trophoblast stem cell (hTSC) comes from placental tissue and has special properties that make it extremely desirable to therapeutic developers. The hTSC is such an early stem cell that it has much more capacity for growth than a stem cell taken from an adult, for example. This means that one cell can become millions. The hTSC also carries with it the same immune-privilege that a growing embryo has inside its mother: its not seen as foreign although its genetically different than its mother. Unlike other foreign materials, the hTSC is not rejected by the human body, which means that it can be used with many different patients (allogeneic cell therapy). With these benefits, the scientific community holds a high regard for hTSCs, but it also faces socio-ethical concerns about how those stem cells are typically sourced.

Accelerated Biosciences sidesteps hTSC sourcing concerns in a profoundly elegant way. Dr. Jau-Nan Lee, an OB-GYN in Taiwan, found inspiration in what was considered medical waste. When surgical intervention was necessary to remove an ectopic pregnancy that would otherwise risk the womans life, the non-viable embryo and pre-placental tissue lodged in the fallopian tube was removed, sent to pathology, and discarded. Gaining permission from institutional colleagues and sampling the pre-placental tissue, Dr. Lee isolated hTSC that offered all the benefits of hTSC pluripotency, immune privilege, and scalability without pathogens and without ethical compromises.

About Accelerated Biosciences

Founded in 2013, Accelerated Biosciences is a private company focused on regenerative medicine and built around the hTSC discoveries of obstetrics and gynecology physician and researcher, Professor Jau-Nan Lee, MB, MD, PhD. Accelerated Biosciences holds a large and robust patent portfolio and an encumbrance-free intellectual property (IP) estate. Accelerated Biosciences mission is to leverage its renewable, immune-privileged human cell source to fuel breakthrough cell therapies that effectively target the most challenging diseases of the human body. For more information about Accelerated Biosciences, visit acceleratedbio.com or email mmolsbergen@c14consultinggroup.com.

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Accelerated Biosciences' Immune-Privileged Human Trophoblast Stem Cells (hTSCs) Offer Breakthrough Opportunities in Cancer-Targeting Therapeutics and...

IPS Cell Therapy Comments Off on Accelerated Biosciences’ Immune-Privileged Human Trophoblast Stem Cells (hTSCs) Offer Breakthrough Opportunities in Cancer-Targeting Therapeutics and… | May 15th, 2021

Small dreams have no power to move hearts, and in a new six-year strategic plan, St. Jude Childrens Research Hospital is thinking very big.

What would it take to drastically increase cure rates for childhood cancer worldwide?

St. Judes answer: $11.5 billion and an additional 1,400 jobs.

To get a rough sense of scale, work it out with a pencil:

Spread over six yearsat $1.916 billion each yearits just under a third of the NCIs annual spend, fourfold this years projected revenues of the American Cancer Society, and more than seventyfold the budget of the World Health Organizations International Agency for Research on Cancer.

Its a broad and ambitious plan that will allow the institution to grow at an almost 8% compound annual growth rate, James Downing, president and CEO of St. Jude, said to The Cancer Letter.

At a global level, we also want to see identifiable increases in cure rates. We are watching those very carefully. Our goal is to move toward cure rates of 60% for diseases like acute lymphoblastic leukemia, Hodgkins lymphoma and Wilms tumor, Downing said. As we look at a global population, survival rates hover around 20%, and wed like to see those go up year by year.

A lot of our efforts are based on implementation science, looking at what works and what doesnt work. Workforce, drug distribution and true advancements in cure rates are what were seeking over the next six years.

The plan, rolled out on April 27, calls for an acceleration of research and treatment globallynot just for pediatric cancer, but also other illnesses, including blood disorders, neurological diseases, and infectious diseases.

Not surprisingly, this amount represents the largest investment the Memphis, Tenn. hospital has made in its nearly 60-year history. The previous strategic plan, the largest expansion in the institutions history, resulted in $7 billion in investments (The Cancer Letter, May 19, 2017).

The multi-phase expansion plan is funded almost entirely by steadily increasing donor contributions generated by ALSAC, the fundraising and awareness organization for St. Jude.

It is an ambitious plan. But were going to have lots of new personnel, new investments, new technology and new partnerships. We have formal partnerships with many U.N. associate agencies and organizations around the world.

Within the past six years, St. Jude has advanced fundamental, clinical, and translational research, Downing said.

Two years ago, we began strategically looking at the most pressing issues in the field of pediatric cancer, Downing said. As we developed the strategic plan over those two years, there were many ideas we critically assessed, and we often said, Its not really best for St. Jude to pursue that.

In the end, we aligned on goals that collectively bring the prospect of remarkable benefits to the field of childhood cancer, and to children with cancer everywhere.

On campus, St. Jude accepted nearly 20% more new cancer patients; increased faculty by 30% and staff by 23%; and embarked on several large-scale construction projects.

The new strategic plan focuses on five areas: fundamental science, childhood cancer, pediatric catastrophic diseases, global impact, and workforce and workplace culture.

Were coming out of a six-year strategic plan in which we increased our number of cancer patients by 20%, with 30% new faculty, 23% more staff, many large-scale construction projects, said Charles Roberts, executive vice president of St. Jude and director of the hospitals Comprehensive Cancer Center. And were now going into a new strategic plan that is 60% larger than our prior plan.

Under the plan, St. Jude will hire nearly 70 new faculty members, plus supporting laboratory staff, to work in basic, translational, and clinical research across 22 departments.

These investigators will have the freedom to pursue the type of conceptually driven research that leads to tomorrows clinical advances.

As we launch a strategic plan, weve identified the most exciting opportunities and challenges at that point in time, Roberts said to The Cancer Letter. However, we fully realize that we dont know whats coming next. New discoveries will be made, and new opportunities will emerge. Via the blue-sky process, weve set aside substantial funds every year to invest in the pursuit of emerging opportunities suggested by faculty and staff.

Part of what brought me here from Boston was the last strategic plan, and its so exciting to be a part of this. But just looking at the numbers, 1,400 new positions, average salary of $90,000. Six hundred and forty of those positions are in research, 266 are in clinical, 100 are in global pediatric medicine, and 394 in support.

Those are the kinds of numbers that you need to make these things real, and I think its exciting for St. Jude and for the field of cancer research, as we bring in all of these new folks.

During the next six years, St. Jude will invest more than $250 million to expand technology and resources available to scientists and clinicians in their search to understand why pediatric catastrophic diseases arise, spread and resist treatments. These investments will include:

St. Jude will invest $3.7 billion during the next six years to expand cancer-focused research and related clinical care. These efforts will center on raising survival rates for the highest-risk cancers and for children with relapsed diseases, while simultaneously improving quality of life for pediatric cancer survivors. The investments will include:

In the U.S., more than 80% of children diagnosed with cancer will be cured. In contrast, 80% of children with cancer live in limited-resource countries, where a mere 20% survive their disease. To address this, St. Jude will more than triple its investment in its international efforts coordinated through St. Jude Global and the St. Jude Global Alliance during the next six years.

This represents an investment of more than $470 million. Global initiatives include:

Under the plan, St. Jude will expand research and treatment programs to advance cures for childhood catastrophic diseases. The $1.1 billion, six-year investment includes work in nonmalignant hematological diseases, such as sickle cell disease; a new laboratory-based research program in infectious diseases that affect children worldwide; and a new research and clinical program to better understand and treat pediatric neurological diseases.

The plan outlines several strategies to encourage teamwork, and internal and external collaboration. These will include:

It is estimated that 87% of funds to sustain and grow St. Jude over the next six years will come from public donations.

Patients at St. Jude do not receive a bill for treatment, travel, housing or fooda model established by ALSAC and St. Jude founder Danny Thomas, who believed in equal access to medical care and driving research advances.

There are an incredible number of donors across the United States who support St. Jude, Downing said. This carries a great responsibility for us to seek the maximum possible impact to improve outcomes for childhood cancer.

Downing and Roberts spoke with Matthew Ong, associate editor of The Cancer Letter.

Matthew Ong: Congratulations on the official launch of St. Judes second six-year strategic plan. Could you briefly walk us through whats in it?

James Downing: It is an exciting time for St. Jude Childrens Research Hospital. Were finishing our prior six-year strategic plan, which started in Fiscal Year 2016. That $7 billion investment in the organization spanned fundamental science, clinical and translational research, clinical operations, and our global efforts. During the course of the plan, we increased faculty by 30% and staff by 23% and accelerated progress against pediatric catastrophic diseases.

About two years ago, we started discussing the next strategic plan. We looked critically at what we had accomplished under the previous plan, the expertise we had assembled, and the major problems in the field of pediatric catastrophic diseases, including cancer, infectious diseases, nonmalignant hematologic diseases and pediatric neurologic diseases. During that period, we involved more than 200 individuals across the institution to develop the new strategic plan.

This plan, at its core, focuses on accelerating progress against pediatric catastrophic diseases on a global scale. It outlines a $11.5 billion investment during the next six years, which includes the addition of 1,400 jobs and $1.9 billion in new capital investments, construction and renovations. Its a broad and ambitious plan that will allow the institution to grow at an almost 8% compound annual growth rate.

The plan has 11 goals, divided among five major areas: fundamental science, pediatric cancer, other childhood catastrophic diseases, global impact, and a focus on people and place. In each of these areas, were recruiting new individuals, investing in new technology, and expanding collaborations across campus, across the United States, and globally.

Ill start with fundamental science. In our last strategic plan, we invested heavily in increasing basic science programs on campus by expanding faculty numbers, technology and institutional data infrastructurein the belief that expanding fundamental science leads to new knowledge that helps advance cures. This is investigator-initiated science, often not related to diseases, but rather to the fundamental questions of biology.

In this new plan, were again investing heavily in expanding fundamental science at St. Jude. Weve committed more than $1 billion to fundamental science. This includes increasing laboratory-based faculty by more than 33% during the next six years, and more than $250 million dedicated to investments in technology.

Our goal is to make sure every dollar is spent wisely and effectively in pursuit of our missionto advance cures and means of prevention for pediatric catastrophic diseases through research and treatment.

The $250 million will fund multiple shared resources, department-based technology centers and new centers of excellence. Some of the faculty are being recruited to the centers of excellence, including those in data-driven discovery, innate immunity and inflammation, leukemia and advanced microscopy. These individuals will also have homes in academic departments.

On the technology front, were investing in shared resources. Well bring online some new ones, as well as some (Center for Modeling Pediatric Diseases and the Center for High-Content Screening) created at the end of the last strategic plan. The newest is focused on spatial transcriptomics. It will allow scientists across campus to look at the expression of genes in tissue context and at the single-cell level.

A new effort in structural biology is to create a $20 million Cryo-Electron Tomography Center. This is the next level of cryo-electron microscopy, which allows the identification of the structure of molecules or molecular machines within the context of cells. Its a developing technology that will feed other investments weve made in structural biology, such as the installation of one of the largest magnets in the world in our NMR facility, our Cryo-Electron Microscopy Center and single-cell analysis capabilities. The plan brings those tools to bear on defining normal biology and disease states.

Another effort is a Center of Excellence in Advanced Microscopy. Over the last six years, weve become one of the leading centers in the application of advanced microscopy to fundamental biology. This has been led by investigators in our Developmental Neurobiology, Cell and Molecular Biology, and Immunology departments.

Were positioned to build the next generation of microscopes to explore biology in ways never dreamed. With new faculty recruitments and collaborations with commercial companies and other institutions, we seek to develop the next generation of microscopes, and apply that to normal biology and to pediatric catastrophic diseases.

Another area were investing in heavily is data science. Over the last six years, and even before that, we expanded data sciences across campus. This initially started with the Pediatric Cancer Genome Project in 2010. Since then, we recruited many data scientists, and coalesced them into our Computational Biology, Biostatistics, and Epidemiology and Cancer Control departments, and into shared resources that provide bioinformatics support.

But over the last several years, weve seen the explosion of data, from structural biology to microscopy.

As we look to the future and the capabilities weve amassed, were poised to significantly increase our investment in data science and become a leading institution in the application of data science to biologic discovery. This is a $40 million investment with 30 full-time employees.

We have a task force led by faculty members to develop the roadmap for how were going to move forward. As data is accumulated and we look across those disparate data types, we can gain knowledge through true data scienceexploring that data and advancing our understanding of biology.

The last area in fundamental research is our graduate school. During the last strategic plan, we developed the St. Jude Graduate School of Biomedical Sciences, which offers a PhD and two masters programs.

Were going to expand that over the coming six years by increasing the number of students in the Biomedical Sciences PhD, the Master of Science in Global Child Health and the Master of Science in Clinical Investigations programs. We will also create a new masters program in data science. That will bring a new population of students to campus, which will further enhance our scientific enterprise.

Pediatric cancer is our next area of focus. This has always been our institutions major focus. This area encompasses $3.7 billion of the operating dollars we will spend over the next six years. Although weve invested heavily in this effort in the past, were going to expand it significantly.

Were going to focus on pediatric cancers where the least progress has been madecancers that are incurable and relapsed diseaseand gain insights into how we can change the outlook for those cancers.

The first area of investment is new faculty10 laboratory-based individuals who will expand our research efforts in understanding the biology of cancer. Some of those faculty will go into the Center of Excellence in Leukemia, but others will focus on solid tumors, brain tumors, or on biologic aspects that cut across cancer types.

Our second area for expansion will focus on assessing new therapeutic approaches for the highest-risk cancers. We need to access and evaluate more new therapies in a rigorous manner to identify those which should be moved forward into frontline clinical trials. Pediatric cancer encompasses many different types of cancer.

To run clinical trials, you need a sufficient number of patients to be able to answer questions in a reasonable time frame. We need a way to identify the best new agents to move into clinical trials.

Our investment in preclinical testing will help us set up that infrastructure. Much of it was established in the last strategic plan, but it must be expanded so that we have the best pediatric cancer models and can assess single and combination therapies to see which are worth moving forward into clinical trials.

On the clinical trial front, we want to expand our infrastructure to run those clinical trialsnot only on our campus, but in collaborations across the United States and around the globe. To make progress in some of these high-risk pediatric cancers, we need many patients. For many of the high-risk cancers, there are not a sufficient number of patients in the United States to conduct the trials. We, therefore, need to set up global collaborative networks that can address these high-risk cancers.

So, were investing in our ability to access drugs through commercial sources, to rigorously assess these in preclinical models and to establish the global infrastructure to run these clinical trials with an associated translational science infrastructure second to none.

Our third emphasis under the cancer focus is cancer immunotherapy. We began our work in cancer immunotherapy decades ago. We developed the chimeric antigen receptor, or CAR, against CD-19. That is the basis for the FDA-approved therapy that is being used right now on a variety of different fronts. Over the last several years, weve also invested heavily in expanding our cancer immunotherapy efforts, primarily focused on CAR-modified T cells.

As part of this new strategic plan, we are creating a new program, the Translational Immunology and Immunotherapy Initiative. It will facilitate cross-departmental efforts focused on cancer immunotherapy and will explore the fundamental biology of chimeric antigen receptor approaches to cancer immunotherapy.

What makes an ideal antigen that can be attacked by a chimeric antigen receptor? How does one manipulate CAR T cells so that they undergo exhaustion and stop killing the tumor? How do we change that? And what are the features of the microenvironment that decrease the killing potency of CAR T cells? These will require significant investments, including additional faculty.

Another emphasis will be looking at long-term toxicities of the therapies we use to treat children with cancer. As we cure more and more pediatric cancers, we must continually look at the toxicities from therapy and figure out how to reduce those without sacrificing the ability to be cured. Part of that is precision medicine, and so we are continuing to invest in our genomic and pharmacogenomic efforts and our proton therapy center.

Part of reducing toxicities comes from learning from long-term survivors. So, we will continue to invest in St. Jude LIFE, our long-term, follow-up study. We will expand that to some of the newer pediatric cancer therapies, including immunotherapy and targeted agents. We will assess long-term complications from these therapeutic approaches and try to define which patients will be susceptible to these toxicities.

MO: I have to mention the obvious: $11.5 billion is quite the budget. Your new strategic plan is work that, one could argue, might be on par or exceeds the coordination and budget required to realize multiple projects, say, at the World Health Organization or even at some U.S. federal agencies. What did it take for you and your team at St. Jude to get to this point?

JD: There are an incredible number of donors across the United States who support St. Jude. Our goal is to make sure every dollar is spent wisely and effectively in pursuit of our missionto advance cures and means of prevention for pediatric catastrophic diseases through research and treatment. This carries a great responsibility for us to seek the maximum possible impact to improve outcomes for childhood cancer.

We have the ideal team at St. Jude to execute this. Our leadership meets multiple times each week. Two years ago, we began strategically looking at the most pressing issues in the field of pediatric cancer. We discussed which areas represented the greatest opportunities for St. Jude to contribute. We talked to many experts inside and outside of the institutionaround the globeand made hard decisions as we went forward.

Strategic planning is deeply engrained at St. Jude as a rigorous process that is part of our scientific culture. We knew it was going to take two years to develop this plan. We dont hire consultants; we do it all ourselves. Faculty across the institution participated in the development of priorities and goals for this strategic plan via structured meetings.

As we developed the strategic plan over those two years, there were many ideas we critically assessed, and we often said, Its not really best for St. Jude to pursue that. In the end, we aligned on goals that collectively bring the prospect of remarkable benefits to the field of childhood cancer, and to children with cancer everywhere.

Every child who comes on this campus is part of our mission. We provide them with the best care possible. We do that in the context of research studies, so that were learning from every single patient. That means were not only helping children today; were also advancing cures for children tomorrow.

Weve rolled out the new strategic plan across campus during the last month, and the excitement is palpable. Our commitment continues long after the strategic plans launch.

We have routine strategic planning retreats, where we assess the goals for the year, evaluate progress against the prior years goals, perform talent assessments and proactively seek out emerging opportunities. Every employee on campus will develop yearly goals that cascade down from the goals of this plan.

As we develop this roadmap, we know there are going to be new ideas. Charlie can tell you about a process incorporated into the strategic plan that allows us to not only move forward on this roadmap, but also add initiatives as new ideas emerge.

Charles Roberts: Its a process we began with the last strategic plan, called our blue-sky process. As we launch a strategic plan, weve identified the most exciting opportunities and challenges at that point in time.

However, we fully realize that we dont know whats coming next. New discoveries will be made, and new opportunities will emerge. Via the blue-sky process, weve set aside substantial funds every year to invest in the pursuit of emerging opportunities suggested by faculty and staff.

Ideas that have emerged from the blue-sky process have been phenomenal. Our engagement with World Health Organization (WHO)a collaboration to raise childhood cancer survival rates internationallyis one example.

The Center for Modeling Pediatric Diseases is another example. This center makes iPS cells that come from patients so that we can investigate mechanisms that underlie cancer predisposition.

In another blue-sky project, were looking at DNA methylation to characterize pediatric solid tumors with the goal of identifying new therapeutic opportunities. Some of our immunotherapy initiatives also came out of the blue-sky process. Were looking forward to growing our blue-sky endeavors as we go forward.

Were coming out of a six-year strategic plan in which we increased our number of cancer patients by 20%, with 30% new faculty, 23% more staff, many large-scale construction projects. And were now going into a new strategic plan that is 60% larger than our prior plan.

The other central part of our strategic planning process focuses on the importance of collaboration. We have systematically incorporated a focus upon collaboration into our entire strategic planning and execution process. Our strategic planning efforts began by bringing together the intellectual resources of faculty and staff at St. Jude. This yielded projects that have interactions between many investigators on campus.

We recognize, however, that were still a relatively small institution, and theres a lot of expertise outside. We asked: How can we engage top scientists to tackle problems related to cancer and other catastrophic illnesses of childhood?

In pursuit of this, during our last strategic plan, we created the St. Jude Research Collaboratives, in which we fund investigators from multiple institutions who collaborate with investigators at St. Jude.

Initially, we were planning to fund two or three Collaboratives. However, they were remarkably successful, and top scientists eagerly joined.

Consequently, weve grown the program to five St. Jude Research Collaboratives already. These teams are each funded at an average of $8 million over 5 years, so each investigator is getting R01-level funding, or a little bit better. This has been a phenomenal success.

In the new strategic plan, were going to grow the program to a steady state of 11 funded collaboratives, representing close to a $90 million investment. So far, three of the Collaboratives are directly focused on childhood cancer. A fourth is a basic science-focused project relevant to childhood cancer. Were excited about the growth of this collaboration-focused program.

Lastly, Id like to address global collaboration. If you look across the globe, in low- and middle-income countries, the cure rates for childhood cancer are less than 20%.

This is a problem we know we can solve. Weve proven in the United States we can drive the cure rate to 80%. How can we help the rest of the world?

Because of the resources brought to us by our donors, we are able to think about these things, and so were now collaborating around the globe to drive cure rates forward for childhood cancer worldwide.

JD: As an example of new ideas and how rapidly we can act on them, Id like to tell you about a new blue-sky proposal that came up at the end of the last strategic plan. This idea was precipitated at a faculty retreat. One of our senior investigators was presenting, and during a coffee break, someone said, Well, what if you did this? That emerged into a blue-sky proposal, Seeing the Invisible in Protein Kinases. This was work from Dr. Babis Kalodimos, our Structural Biology department chair. He had a Science paper that came out several months ago, where he used the high-field NMR spectroscopy to look at the structure of the ABL kinase. He was able to identify transient conformational states that help to explain how resistant mutants work.

This gave us new insights into transient states that exist in molecules that can only be seen under high-field NMR, not with other structural biology approaches.

Based on that, we started thinking, Well, what if you did this on all kinases? What if you just did it against tyrosine kinases, serine kinases, receptor tyrosine kinases? What new rules would emerge from this? What would it tell us about families of kinases? What would it tell us about mechanisms of inhibition to kinase inhibitors? What might it tell us about new approaches to developing drugs against protein tyrosine kinases?

And since kinases are a major focus for targeted therapy, there was great excitement about pursuing these studies. Dr. Kalodimos developed the proposal and brought it forward; however, it was clear that this effort would be beyond the scope of our blue-sky process.

Blue-sky initiatives are usually somewhere in the $12 million range, and this was north of $30 million. Yet, in the end after thorough internal and external reviews, the project will move forward as part of the new strategic plan..

This is an approach that will give us fundamental knowledge and can have a profound impact on our understanding of a major class of targets for next-generation therapy.

MO: If I recall accurately, St. Jude has a network of partnerships with a few dozen countries worldwide. Does this plan call for an expansion of efforts within each of those countries? And how many of them?

JD: When I took over in 2014, we had what we called the International Outreach Program, which was 24 programs in 17 countries. During the programs 25-year history, we had made great progress. We were making significant impact and changing the outlook for children with cancer in those 17 countries. But we were affecting only about 3% of children with cancer across the globe, and the International Outreach Program was not scalable.

So, at the beginning of the last strategic plan, we recruited Dr. Carlos Rodriguez-Galindo. He developed a vision that after assessing, we decided to move forward on. This new effort encompasses the Department of Pediatric Global Medicine, St. Jude Global and the St. Jude Global Alliance.

These are all integrated. We developed a model that we think is scalable around the world, and we think this model ultimately can affect children with cancer everywhere.

The idea is that first we must train a workforce to treat children with cancer around the globe. We cant train the workforce ourselves, but we can train the trainers, who will then train the workforce.

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St. Jude's $11.5B, six-year plan aims to improve global outcomes for children with cancer and catastrophic diseases - The Cancer Letter

IPS Cell Therapy Comments Off on St. Jude’s $11.5B, six-year plan aims to improve global outcomes for children with cancer and catastrophic diseases – The Cancer Letter | May 15th, 2021

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.

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.

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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.

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.

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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.

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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.

This study provides a particularized anatomy according to the L.E.A.P mechanism

The regional analysis offers market assays across:

The study, prepared through the L.E.A.P mechanism adds a dimension of infallibility and assures precise information on all the growth dynamics.

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Stem Cell Therapy Market Report | Know the Cutting-Edge Innovations and Future Trends of Market - BioSpace

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market Report | Know the Cutting-Edge Innovations and Future Trends of Market – BioSpace | May 15th, 2021

Researchers used a pig model of heart attacks, which more closely resembles the human heart in size and physiology, and thus has high clinical relevance to human disease.

Researchers used a pig model of heart attacks, which more closely resembles the human heart in size and physiology, and thus has high clinical relevance to human disease.In a large-animal study, researchers have shown that heart attack recovery is aided by injection of heart muscle cells derived from human induced pluripotent stem cell line, or hiPSCs, that overexpress cyclin D2. This research, published in the journal Circulation, used a pig model of heart attacks, which more closely resembles the human heart in size and physiology, and thus has higher clinical relevance to human disease, compared to studies in mice.

An enduring challenge for bioengineering researchers is the failure of the heart to regenerate muscle tissue after a heart attack has killed part of its muscle wall. That dead tissue can strain the surrounding muscle, leading to a lethal heart enlargement.

Heart experts thus have sought to create new tissue applying a patch of heart muscle cells or injecting heart cells to replace damaged muscle. Similarly, they have tried to stimulate division of existing heart muscle cells near the damaged area. This current study, led by researchers at the University of Alabama at Birmingham, shows progress in both goals.

After the experimental heart attack, heart tissue around the infarction site was injected with about 30 million bioengineered human cardiomyocytes that were differentiated from hiPSCs. These cells also overexpress cyclin D2, part of a family of proteins involved in cell division.

Compared to control human cardiomyocytes, the cyclin D2-cardiomyocytes showed enhanced potency to repair the heart. They proliferated after injection, and by four weeks, the hearts had less pathogenic enlargement, reduced size of dead muscle tissue and improved heart function.

Intriguingly, the cyclin D2-cardiomyocytes stimulated not only their own proliferation, but also proliferation of existing heart muscle cells around the infarction site of the pig heart, as well as showing angiogenesis, the development of new blood vessels.

These results suggest that the cyclin D2-cardiomyocyte transplantation may be a potential therapeutic strategy for the repair of infarcted hearts, said study leader Jianyi Jay Zhang, M.D., Ph.D., the chair of Biomedical Engineering, a joint department of the UAB School of Medicine and the UAB School of Engineering.

This ability of the graft cyclin D2-cardiomyocytes to stimulate the proliferation of nearby existing heart cells suggested paracrine signaling, a type of cellular communication where a cell produces a signal that induces changes in nearby cells.

Exosomes small blebs or tiny vesicles that are released by human or animal cells and contain proteins and RNA from the cells that release them are one common form of paracrine signaling.

Zhang and colleagues found that exosomes that they purified from the cyclin D2-cardiomyocyte growth media indeed promoted proliferation of cultured cardiomyocytes. In addition, the treated cardiomyocytes were more resistant to programmed cell death, called apoptosis, induced by low oxygen levels. The exosomes also induced proliferation of various other cell types, including human umbilical vein endothelial cells, human vascular smooth muscle cells and 7-day-old rat cardiomyocytes that have almost undetectable proliferation.

Part of the cargo that exosomes carry are microRNAs, or miRNAs. These short pieces of RNA have the ability to interact with messenger RNA in target cells, and they are robust players of gene regulation in cells. Humans have more than 2,000 miRNAs with different RNA sequences, and these are thought to regulate a third of the genes in the genome.

So, the researchers documented which microRNAs were present in exosomes from the cyclin D2-overexpressing cardiomyocytes and in exosomes from non-overexpressing cardiomyocytes. As expected, they found differences.

Jianyi Jay Zhang, M.D., Ph.D.Together, the exosomes from both types of cells contained 1,072 different miRNAs, and 651 were common to the two exosome groups. However, 332 miRNAs were found only in the cyclin D2-overexpressing cardiomyocytes, and 89 miRNAs were specific for the non-overexpressing cardiomyocytes. In preliminary work of characterizing the effects of specific miRNAs, one particular miRNA from the cyclin D2-overexpressing exosomes was shown to stimulate proliferation when delivered into rat cardiomyocytes.

Thus, as the therapeutic potential of exosomes for improving cardiac function becomes more evident, combining an exosome-mediated delivery of proliferative miRNAs with transplantation of cyclin D2-overexpressing cardiomyocytes, or cell products, could become a new promising strategy for upregulating proliferation of the recipient cardiomyocytes and reducing cardiac fibrosis, Zhang said. Altogether, our data suggest that cardiac cell therapy, involving cardiomyocytes with enhanced proliferation capacity, may become an efficacious future strategy for myocardial repair and prevention of congestive heart failure in patients with acute myocardial infarctions.

UAB Department of Biomedical Engineering co-authors with Zhang, in the study Cyclin D2 overexpression enhances the efficacy of human induced pluripotent stem cell-derived cardiomyocytes for myocardial repair in a swine model of myocardial infarction, are Meng Zhao, Yuji Nakada, Yuhua Wei, Weihua Bian, Anton V. Borovjagin, Yang Zhou and Gregory P. Walcott.

Additional co-authors are Yuxin Chu and Min Xie, Division of Cardiovascular Disease, UAB Department of Medicine; Wuqiang Zhu, Department of Cardiovascular Diseases, Physiology and Biomedical Engineering, Mayo Clinic Arizona, Scottsdale; Thanh Nguyen, UAB Informatics Institute; and Vahid Serpooshan, Emory University and Georgia Institute of Technology, Atlanta.

Support came from National Institutes of Health grants HL114120, HL131017, HL149137 and HL134764.

At UAB, Zhang holds the T. Michael and Gillian Goodrich Endowed Chair of Engineering Leadership.

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Heart attack recovery aided by injecting heart muscle cells that overexpress cyclin D2 - The Mix

Cardiac Stem Cells Comments Off on Heart attack recovery aided by injecting heart muscle cells that overexpress cyclin D2 – The Mix | May 15th, 2021

The report on the Rheumatoid Arthritis Stem Cell Therapy market provides a birds eye view of the current proceedings and the impact on the COVID-19 pandemic. Further, the report ponders over the various factors that are likely to impact the overall dynamics of the market once the COVID-19 pandemic subsides. The current trends, growth opportunities, restraining factors, and drivers are discussed in the report in detail.

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The growing prevalence and recurrence of rheumatoid arthritis is expected to be the major factor driving the growth of the rheumatoid arthritis stem cell therapy market over the forecast period. Although doctors do not know the exact cause of rheumatoid arthritis, but certain risk factors are observed to be associated with it.

These risk factors include age (most common between the age of 40 and 60), family history, gender, environment (a toxic chemical in the environment can up the odds), obesity and smoking. Changes in lifestyle and eating habits are contributing to the growing prevalence of rheumatoid arthritis.

Rheumatoid Arthritis Stem Cell Therapy Market: Segmentation

Tentatively, the global rheumatoid arthritis stem cell therapy market can be segmented on the basis of treatment type, application, end user and geography.

Based on treatment type, the global rheumatoid arthritis stem cell therapy market can be segmented into:

Based on application, the global rheumatoid arthritis stem cell therapy market can be segmented into:

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Based on distribution channel, the global rheumatoid arthritis stem cell therapy market can be segmented into:

Based on geography, the global rheumatoid arthritis stem cell therapy market can be segmented into:

Rheumatoid Arthritis Stem Cell Therapy Market: Regional Outlook

Geographically, the global rheumatoid arthritis stem cell therapy market can be segmented into viz. North America, Latin America, Europe, Asia-Pacific excluding Japan (APEJ), Japan and the Middle East and Africa (MEA). North America is expected to be the dominant region in the global rheumatoid arthritis stem cell therapy market, owing to the presence of various key players.

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The rheumatoid arthritis stem cell therapy market in Asia Pacific excluding Japan is expected to grow at a significant CAGR due to the expansion of product offerings by key players. Europe is expected to have the second large share in the global rheumatoid arthritis stem cell therapy market throughout the forecast period.

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Allogeneic Mesenchymal Stem Cell Segment Is Expected To Lead In the Global Rheumatoid Arthritis Stem Cell Therapy Market over the Forecast Period,...

Cardiac Stem Cells Comments Off on Allogeneic Mesenchymal Stem Cell Segment Is Expected To Lead In the Global Rheumatoid Arthritis Stem Cell Therapy Market over the Forecast Period,… | May 15th, 2021

Mice, rats and pigs all share a secret superpower: They can all use their intestines to breathe, and scientists discovered this by pumping oxygen up the animals' butts.

Why run such experiments, you ask? The research team wanted to find a potential alternative to mechanical ventilation, a medical treatment where a machine pushes air into a patient's lungs through the windpipe. Ventilators deliver oxygen to the lungs and help remove carbon dioxide from the blood, but the machines aren't always available.

Early in the COVID-19 pandemic, for example, hospitals faced a severe shortage of ventilators, The New York Times reported. Although doctors can also use a technique called extracorporeal membrane oxygenation (ECMO), where blood is pumped out of the body and reoxygenated with a machine, the procedure carries inherent risks, such as bleeding and blood clots; and it's often less readily available than ventilators, according to Mayo Clinic.

Related: The 10 weirdest medical cases in the animal kingdom

In search of another solution, the study authors drew inspiration from aquatic animals like sea cucumbers and freshwater fish called loaches (Misgumus anguillicandatus), which use their intestines for respiration. It was unclear whether humans and other mammals have similar capabilities, although some scientists attempted to answer that question in the 1950s and 1960s.

"We initially looked at a mouse model system to see if we could deliver oxygen gas intra-anusly," said senior author Dr. Takanori Takebe, a professor at the Tokyo Medical and Dental University and a director at the Center for Stem Cell and Organoid Research and Medicine at Cincinnati Children's Hospital Medical Center.

"Every time we performed experiments, we were quite surprised," Takebe told Live Science.

Without intestinal ventilation, mice placed in a low-oxygen environment survived for only about 11 minutes; with ventilation into their anuses, 75% survived for 50 minutes, thanks to an infusion of oxygen that reached their hearts. The team then tried using oxygenated liquid, rather than gas, in mice, rats and pigs, and they found similarly promising results. The team noted that more work still needs to be done to see if the approach is safe and effective in humans, according to a paper on their findings published May 14 in the journal Med.

"The pandemic has highlighted the need to expand options for ventilation and oxygenation in critical illness, and this niche will persist even as the pandemic subsides," as there will be times when mechanical ventilation is unavailable or inadequate on its own, Dr. Caleb Kelly, a clinical fellow and physician-scientist at Yale School of Medicine, wrote in a commentary of the study. If, after further evaluation, intestinal ventilation eventually becomes common practice in intensive care units, this new study "will be marked by historians as a key scientific contribution," he wrote.

Before starting their experiments in rodents, the team got very familiar with loach guts. The fish take in oxygen mostly through their gills, but occasionally, when exposed to low-oxygen conditions, loaches instead use a portion of their intestines for gas exchange, Takebe said. In fact, in response to the lack of oxygen, the structure of gut tissues near the anus changes such that the density of nearby blood vessels increases and secretion of fluids related to digestion decreases.

These subtle changes allow loaches to "suck up the oxygen more efficiently," Takebe said. In addition, the outermost lining of the loach gut the epithelium is very thin, meaning oxygen can easily permeate the tissue to reach the blood vessels beneath, he added. To simulate this structure in their mouse models, the team thinned out the gut epithelium of the rodents using chemicals and various mechanical procedures.

They then placed the mice under extremely low-oxygen conditions and used a tube to pump oxygen gas up the animals' bums and into their large intestines.

Related: 8 bizarre animal surprises from 'True or Poo' Can you tell fact from myth?

Compared with mice whose gut epithelium had not been thinned, the mice with thin epitheliums survived significantly longer in the experiment with most surviving 50 minutes as compared with about 18 minutes. Again, mice not given any oxygen only survived for about 11 minutes. In addition to surviving longer, the group with thinned-out gut linings showed signs that they were no longer starved for oxygen; they stopped gasping for air or showing signs of cardiac arrest, and the oxygen pressure in their major blood vessels improved.

Although this initial experiment suggested that oxygen could pass through the intestine and into circulation, thinning out the gut epithelium would likely not be feasible in human patients, Takebe said.

Particularly in critically ill patients, "I think additional damage to the gut would be really dangerous, for the treatment perspective," Takebe said. But "over the course of the experiments, we realized that even the intact gut has some, not really efficient, but some capacity to exchange the gas," he noted, meaning there may be a way to introduce oxygen through the gut without first thinning out the tissues.

So in another experiment, rather than using oxygen gas, the team tried perfluorodecalin (PFD), a liquid fluorocarbon that can be infused with a large amount of oxygen. The liquid is already used in people, such as for use in the lungs of infants with severe respiratory distress, the authors noted in their report.

The liquid also acts as a surfactant a substance that reduces surface tension; since a surfactant lines the air sacs of the lungs and helps boost gas exchange in the organ, PFD may fulfill a similar purpose in the intestines, Takebe said.

Much like in the oxygen-gas experiments, the oxygenated PFD rescued mice from the effects of being placed in a low-oxygen chamber, enabling the rodents to meander about their cage more than mice not given the treatment. After just one injection of 0.03 ounces (1 milliliter) of the liquid, the rodents' improvements persisted for about 60 minutes.

"We are not quite sure why this improvement is persisting much longer than the original expectations," Takebe noted, as the authors expected the effects to wear off in just a couple minutes. "But the observation is really reproducible and very robust."

Related: Gasp! 11 surprising facts about the respiratory system

The team then moved on to a pig model of respiratory failure, where they placed pigs on ventilators and only provided a low level of oxygen and then injected PDF into the pigs' posteriors with a long tube. Compared with pigs not given the PFD treatment, pigs given PFD improved in terms of the oxygen saturation of their blood, and the color and warmth returned to their skin. A 13.5 oz (400 ml) infusion sustained these improvements for about 18 to 19 minutes, and the team found that they could give additional doses to the pigs without noticeable side effects.

The team also tested the safety of repeat dosing in rats and found that, while their oxygen levels rose, the animals showed no notable side effects, markers of organ damage or stray PFD lingering in their cells.

Following this success in animal models, Takebe said that his team hopes to start a clinical trial of the treatment in humans sometime next year. They would likely start by testing the safety of the approach in healthy volunteers and beginning to work out what dose levels would be reasonable, he said. However, to make the jump from animals to human patients, the team will need to address a number of critical questions.

For instance, the treatment could potentially stimulate the vagus nerve a long nerve that connects the gut and brain so trial organizers should likely be on the lookout for side effects like falling blood pressure or fainting, Takebe noted. Also, the lower gut contains relatively little oxygen compared with other organs in the body, he added. The community of bacteria and viruses that live in the gut are adapted to these low-oxygen conditions, and a sudden infusion of oxygen might disrupt those microbes, he said.

"The consequence of reversing this so-called 'physiologic hypoxia' is unknown," Kelly noted in his commentary, echoing Takebe's sentiments. In humans, it will be important to determine how many doses of oxygenated liquid could be safely administered into the gut without causing unintended changes to the intestinal environment, he wrote.

In addition, the animal models in the study don't fully reflect what critically ill patients experience during respiratory failure, a condition that often coincides with infection, inflammation and low blood flow, Kelly noted. So there may be additional factors to consider in critically ill patients that weren't relevant in rodents and pigs. And depending on a given patient's condition, they may need a higher or lower dose of PFD all of these fine details will need to be carefully assessed in future trials, Takebe said.

Originally published on Live Science.

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Pigs can breathe through their butts. Can humans? - Livescience.com

Cardiac Stem Cells Comments Off on Pigs can breathe through their butts. Can humans? – Livescience.com | May 15th, 2021

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck & Co. (NYSE: MRK), known as MSD outside the United States and Canada, today announced positive results from the pivotal neoadjuvant/adjuvant Phase 3 KEYNOTE-522 trial investigating KEYTRUDA, Mercks anti-PD-1 therapy, in combination with chemotherapy as pre-operative (neoadjuvant) treatment and then continuing as a single agent (adjuvant) treatment after surgery. KEYNOTE-522 met its dual primary endpoint of event-free survival (EFS) for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC). Based on an interim analysis conducted by the independent Data Monitoring Committee (DMC), neoadjuvant KEYTRUDA plus chemotherapy followed by adjuvant KEYTRUDA as monotherapy showed a statistically significant and clinically meaningful improvement in EFS compared with neoadjuvant chemotherapy alone. As previously communicated, KEYNOTE-522 met its other dual primary endpoint of pathological complete response (pCR). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified.

KEYTRUDA is the first immunotherapy to show positive results for event-free survival in patients with high-risk early-stage TNBC, a particularly aggressive form of breast cancer, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. The improvement in pathological complete response rates initially observed following pre-operative treatment was encouraging, and now that we are seeing the data mature after four years to include a statistically significant improvement in event-free survival, we look forward to working with the FDA and other global authorities to bring this new option to patients as quickly as possible. We are grateful to the study participants who are critical to our efforts to advance potential treatment options for patients with TNBC.

An analysis of pCR from KEYNOTE-522 was presented at the European Society for Medical Oncology (ESMO) 2019 Congress and published in the New England Journal of Medicine. Findings showed a statistically significant increase in pCR for KEYTRUDA plus chemotherapy versus chemotherapy alone as neoadjuvant therapy in patients with early-stage TNBC, regardless of PD-L1 status. As previously announced, the company received a Complete Response Letter (CRL) from the FDA in March 2021 regarding Mercks supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA for the treatment of patients with high-risk early-stage TNBC based on these pCR data and early interim EFS findings. The CRL followed the FDAs Oncologic Drugs Advisory Committee meeting that voted 10-0 that a regulatory decision should be deferred until further data were available from KEYNOTE-522.

The KEYTRUDA clinical development program for TNBC encompasses several internal studies and external collaborative trials, including the ongoing studies KEYNOTE-242 and KEYNOTE-355.

Merck has an expansive clinical development program investigating KEYTRUDA in earlier lines of therapy including in neoadjuvant, adjuvant and locally advanced settings, with approximately 20 registrational studies ongoing.

About KEYNOTE-522

KEYNOTE-522 is a Phase 3, randomized, double-blind trial (ClinicalTrials.gov, NCT03036488), evaluating a regimen of neoadjuvant KEYTRUDA in combination with chemotherapy followed by adjuvant KEYTRUDA as monotherapy versus a regimen of neoadjuvant chemotherapy followed by adjuvant placebo. The dual primary endpoints are pCR and EFS. The secondary endpoints include pCR rate using alternative definitions (i.e., no invasive or noninvasive residual cancer in breast or nodes) at the time of definitive surgery, overall survival, EFS in patients whose tumors express PD-L1 (Combined Positive Score [CPS] 1), safety and patient-reported outcomes. The study enrolled 1,174 patients who were randomized 2:1 to receive either:

About Triple-Negative Breast Cancer (TNBC)

Triple-negative breast cancer is an aggressive type of breast cancer that characteristically has a high recurrence rate within the first five years after diagnosis. While some breast cancers may test positive for estrogen receptors, progesterone receptors or overexpression of human epidermal growth factor receptor 2 (HER2), TNBC tests negative for all three. Approximately 15-20% of patients with breast cancer are diagnosed with TNBC. TNBC tends to be more common in women who are younger than 40 years of age, who are African American or who have a BRCA1 mutation.

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 1,400 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 in the U.S.

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.

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 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 (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 Carcinoma

KEYTRUDA, in combination with trastuzumab, and fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. 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.

KEYTRUDA, as a single agent, 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 Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:

Cervical Carcinoma

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] 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.

Triple-Negative Breast Cancer

KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or the programmed death ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of antiPD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% of these patients interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA with Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently 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. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen, which was at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT 3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT 3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT 3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients 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 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.

Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis

Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1). All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Immune-Mediated Nephritis With Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.

Immune-Mediated Dermatologic Adverse Reactions

KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with antiPD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other antiPD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% of 2799 patients receiving KEYTRUDA. Monitor for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or Grade 2 reactions. For Grade 3 or Grade 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 HSCT before or after antiPD-1/PD-L1 treatment. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute and chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between antiPD-1/PD-L1 treatment and allogeneic HSCT. Follow patients closely for evidence of these complications and intervene promptly. Consider the benefit vs risks of using antiPD-1/PD-L1 treatments 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 an antiPD-1/PD-L1 treatment 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-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%).

Excerpt from:
Merck Announces Phase 3 KEYNOTE-522 Trial Met Dual Primary Endpoint of Event-Free Survival (EFS) in Patients With High-Risk Early-Stage...

Cardiac Stem Cells Comments Off on Merck Announces Phase 3 KEYNOTE-522 Trial Met Dual Primary Endpoint of Event-Free Survival (EFS) in Patients With High-Risk Early-Stage… | May 15th, 2021

A groundbreaking new study led by the University of Minnesota, Twin Cities has shown for the first time that lab-created heart valves implanted in young lambs for a year were capable of growth within the recipient.

The valves also showed reduced calcification and improved blood flow function compared to animal-derived valves currently used when tested in the same growing lamb model.

Currently, researchers have not been able to develop a heart valve that can grow and maintain function for paediatric patients. The only accepted options for these children with heart defects are valves made from chemically treated animal tissues that often become dysfunctional due to calcification and require replacement because they dont grow with the child. These children will often need to endure up to five (or more) open heart surgeries until a mechanical valve is implanted in adulthood. This requires them to take blood thinners the rest of their lives.

Minnesota Professor Robert Tranquillo and his colleagues used a hybrid of tissue engineering and regenerative medicine to create the growing heart valves. Over an eight-week period, they used a specialised tissue engineering technique they previously developed to generate vessel-like tubes in the lab from a post-natal donors skin cells. To develop the tubes, the researchers combined the donor sheep skin cells in a gelatine-like material, called fibrin, in the form of a tube and then provided nutrients necessary for cell growth using a bioreactor.

The researchers then used special detergents to wash away all the sheep cells from the tissue-like tubes, leaving behind a cell-free collagenous matrix that does not cause immune reaction when implanted. This means the tubes can be stored and implanted without requiring customised growth using the recipients cells.

The next step was to precisely sew three of these tubes (about 16 mm in diameter) together into a closed ring. The researchers then trimmed them slightly to create leaflets to replicate a structure similar to a heart valve about 19 mm in diameter.

After these initial steps, it looked like a heart valve, but the question then became if it could work like a heart valve and if it could grow, Prof Tranquillo said. Our findings confirmed both.

This second generation of tri-tube valves was implanted into the pulmonary artery of three lambs. After 52 weeks, the valve regenerated as its matrix became populated by cells from the recipient lamb, and the diameter increased from 19 mm to a physiologically normal valve about 25 mm. The researchers also saw a 17 to 34% increase in the length of the valve leaflets as measured from ultrasound images. In addition, researchers showed that the tri-tube valves worked better than current animal-derived valves, with almost none of the calcification or blood clotting that the other valves showed after being implanted in lambs of the same age.

We knew from previous studies that the engineered tubes have the capacity to regenerate and grow in a growing lamb model, but the biggest challenge was how to maintain leaflet function in a growing valved conduit that goes through 40 million cycles in a year, said Zeeshan Syedain, a senior research associate in Prof Tranquillos lab. When we saw how well the valves functioned for an entire year from young lamb to adult sheep, it was very exciting.

This is a huge step forward in paediatric heart research, Tranquillo said. This is the first demonstration that a valve implanted into a large animal model, in our case a lamb, can grow with the animal into adulthood.

If confirmed in humans, the new heart valves could prevent the need for repeated valve replacement surgeries in thousands of children born each year with congenital heart defects. The valves can also be stored for at least six months, which means they could provide surgeons with an off the shelf option for treatment.

The study has now been published in the journal Science Translational Medicine, while the valve-making procedure has been patented and licensed to University of Minnesota start-up company Vascudyne. Prof Tranquillo said the next steps are to implant the tri-tube valve directly into the right ventricle of the heart to emulate the most common surgical repair and then start the process of requesting approval from the US Food and Drug Administration (FDA) for human clinical trials over the next few years.

If we can get these valves approved someday for children, it would have such a big impact on the children who suffer from heart defects and their families who have to deal with the immense stress of multiple surgeries, Prof Tranquillo said. We could potentially reduce the number of surgeries these children would have to endure from five to one. Thats the dream.

Image credit: Syedain, et al, Tranquillo Lab, University of Minnesota.

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Lab-created heart valves grow with the recipient - Lab + Life Scientist

Skin Stem Cells Comments Off on Lab-created heart valves grow with the recipient – Lab + Life Scientist | May 3rd, 2021

CRISPR is revolutionary. Its also a total brute.

The classic version of the gene editing wunderkind literally slices a gene to bits just to turn it off. Its effective, yes. But its like putting an electrical wire through a paper shredder to turn off a misbehaving light bulb. Once the wires are cut, theres no going back.

Why not add a light switch instead?

This month, a team from the University of California, San Francisco (UCSF) reimagined CRISPR to do just that. Rather than directly acting on genesirrevocably dicing away or swapping genetic lettersthe new CRISPR variant targets the biological machinery that naturally turns genes on or off.

Translation? CRISPR can now flip a light switch to control geneswithout ever touching them directly. It gets better. The new tool, CRISPRoff, can cause a gene to stay silent for hundreds of generations, even when its host cells morph from stem cells into more mature cells, such as neurons. Once the sleeping beauty genes are ready to wake up, a complementary tool, CRISPRon, flips the light switch back on.

This new technology changes the game so now youre basically writing a change [into genes] that is passed down, said author Dr. Luke Gilbert. In some ways we can learn to create a version 2.0 of CRISPR-Cas9 that is safer and just as effective.

The crux is something called epigenetics. Its a whole system of chemicals and proteins that controls whether a gene is turned on or off.

If that sounds confusing, lets start with what genes actually look like inside a cell and how they turn on. By turning on, I mean that genes are made into proteinsthe stuff that builds our physical form, controls our metabolism, and makes us tick along as living, breathing humans.

Genes are embedded inside DNA chains that wrap very tightly around a core proteinkind of like bacon-wrapped asparagus. For genes to turn on, the first step is that they need a bunch of proteins to gently yank the DNA chain off the asparagus, so that the genes are now free-floating inside their cellular space capsule, called the nucleus.

Once that chunk of bacon-y DNA is free, more proteins rush over to grab onto the gene. Theyll then roll down the genes nucleotides (A, T, C, and G) like a lawn mower. Instead of mulch, however, this biological machine spews out a messenger that tells the cell to start making proteinsmRNAs. (Yup, the same stuff that makes some of our Covid-19 vaccines.) mRNA directs our cells protein factory to start production, and voil, that gene is now turned on!

Anything that disrupts this process nukes the genes ability to turn into proteins, essentially shutting it off. Its enormously powerfulbecause one single epigenetic machine can control hundreds or thousands of genes. Its a master light switch for the genome.

The team started with a CRISPR system that has a neutered Cas9. This means that the protein normally involved in cutting a gene, Cas9, can no longer snip DNA, even when tethered to the correct spot by the other component, the guide RNA bloodhound. They then tacked on a protein thats involved in switching off genes to this version of CRISPR.

Heres the clever part: the protein is designed to hijack a natural epigenetic process for switching genes off. Genes are often shut down through a natural process called methylation. Normally, the process is transient and reversible on a gene. CRISPRoff commandeers this process, in turn shutting down any targeted gene but for a far longer period of timewithout physically ripping the gene apart.

Thanks to epigenetics enhancing power, CRISPRoff lets researchers go big. In one experiment targeting over 20,000 genes inside immortalized human kidney cells with CRISPRoff, the team was able to reliably shut those genes off.

Not satisfied with a one-way street, the team next engineered a similar CRISPR variant, with a different epigenetics-related protein, dubbed CRISPRon. In cells inside petri dishes, CRISPRon was able to override CRISPRoff, and in turn, flip the genes back on.

We now have a simple tool that can silence the vast majority of genes, said study author Dr. Jonathan Weissman. We can do this for multiple genes at the same time without any DNA damage and in a way that can be reversed.

Even crazier, the off switch lasted through generations. When the team turned off a gene related to the immune system, it persisted for 15 monthsafter about 450 cellular generations.

The edits also lasted through a fundamental transformation, that is, a cells journey from an induced pluripotent stem cell (iPSC) to a neuron. iPSCs often start as skin cells, and are rejuvenated into stem cells through a chemical bath, when they then take a second voyage to become neurons. This process often wipes away epigenetic changes. But to the authors surprise, CRISPRoffs influence remained through the transformations. In one experiment, the team found that shutting off a gene related to Alzheimers in iPSCs also reduced the amount of subsequently encoded toxic proteins in the resulting neurons.

What we showed is that this is a viable strategy for silencing Tau and preventing that protein from being expressed, said Weissman, highlighting just one way CRISPRoffand controlling the epigenome in generalcan alter medicine.

This isnt the first time someones tried to target the epigenome with CRISPR. The same team previously experimented with another set of CRISPR variants that tried the same thing. The difference between the two is time and stability. With the previous setup, scientists struggled to keep the light switch off for a single generation. The new one has no trouble maintaining any changes through multiple divisionsand transformationsin the genome.

A reliable CRISPR tool for epigenetics is insanely powerful. Although we have drugs that work in similar ways, theyre far less accurate and come with a dose of side effects. For now, however, CRISPRoff and CRISPRon only work in cells in petri dishes, and the next step towards genomic supremacy would be to ensure they work in living beings.

If thats the case, it could change genetic editing forever. From reprogramming biological circuits in synthetic biology to hijacking or reversing ones to prevent disease, epigenetic reprogramming offers a way to do it all without ever touching a gene, nixing the threat of mutationswhile leading to lasting effects through generations.

I think our tool really allows us to begin to study the mechanism of heritability, especially epigenetic heritability, which is a huge question in the biomedical sciences, said study author Dr. James Nuez.

Image Credit: nobeastsofierce/Shutterstock.com

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A New CRISPR Tool Flips Genes On and Off Like a Light Switch - Singularity Hub

Skin Stem Cells Comments Off on A New CRISPR Tool Flips Genes On and Off Like a Light Switch – Singularity Hub | May 3rd, 2021

New research report on Autologous Stem Cell Based Therapies market size by In4Research provides the latest developments in the market has attained and the latest news regarding the market and its building blocks. Some of the most accurate topics mentioned in the report include market drivers and restraints, challenges, opportunities, COVID-19 information related to the market, key players of the market, and the entire market segmentation.

This report provides data for the base year 2020 as well as the forecast period (2021-2026) along with the CAGR. This report contains detailed research of the market from the industry level to the market players and how the market is functioning at present along with all the relevant data and information. Thereby, the Autologous Stem Cell Based Therapies research report has been formulated encompassing all the key points that are present in the form of tables and graphs to make it more specific for decision-makers.

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The segmental analysis offered in the report pinpoints key opportunities available in the Autologous Stem Cell Based Therapies market through leading segments. The regional study of the Autologous Stem Cell Based Therapies market included in the report helps decision-makers to gain a sound understanding of the development of different geographical markets in recent years and going forth.

By Type:

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To comprehend Global Autologous Stem Cell Based Therapies market dynamics in the world mainly, the worldwide Autologous Stem Cell Based Therapies market is analyzed across major regions. A customized study by region and country can be provided considering the below splits.

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The report provides in-depth information about profitable showing markets and examines the markets for the global Autologous Stem Cell Based Therapies market with business strategies of the key players and the new entering market industries are considered in detail and it presents the deep-dive eyesight of this Autologous Stem Cell Based Therapies market from 2021 to 2026 and prospective prediction market trends.

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Key Trends of Autologous Stem Cell Based Therapies Market 2021 Business Opportunities, Market Dynamics, Growth Size and Forecasts to 2026 - Clark...

Cardiac Stem Cells Comments Off on Key Trends of Autologous Stem Cell Based Therapies Market 2021 Business Opportunities, Market Dynamics, Growth Size and Forecasts to 2026 – Clark… | May 3rd, 2021

CHICAGO, April 28, 2021 /PRNewswire/ -- According to the new market research report "Stem Cell Therapy Marketby Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) - Global Forecasts to 2026", published by MarketsandMarkets, the global market is projected to reach USD 401 million by 2026 from USD 187 million in 2021, at a CAGR of 16.5% during the forecast period.

Browse and in-depth TOC on"Stem Cell Therapy Market"142 - Tables45 - Figures160 - Pages

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The Market growth is driven mainly by factors such as increasing investment in stem cell research and the rising number of GMP-certified stem cell manufacturing plants. However, factors such as ethical concerns and the high cost of stem cell research and manufacturing process likely to hinder the growth of this market.

The adipose tissue-derived MSCs segment accounted for the largest share of the cell source segment in the Stem Cell Therapy Market in 2020.

Based on the cell source from which stem cells are obtained, the global market is segmented into four sources. These include adipose tissue-derived MSCs (mesenchymal stem cells), bone marrow-derived MSCs, placenta/umbilical cord-derived MSCs, and other cell sources (which includes human corneal epithelium stem cells, peripheral arterial-derived stem cells, and induced pluripotent stem cell lines). In 2020, adipose tissue-derived MSCs accounted for the markets largest share due to their increasing utilization in treating inflammatory diseases and wounds & injuries. There are several associated advantages, such as ease of harvesting stem cells by minimally invasive methods, simplicity of the isolation procedure, and better quality & proliferation capacity of adipose tissue-derived stem cells.

The musculoskeletal disorders segment accounted for the largest share of the therapeutic application segment in the Stem Cell Therapy Market in 2020

Based on therapeutic application, the global market is segmented into musculoskeletal disorders, wounds & injuries, cardiovascular diseases, surgeries, inflammatory & autoimmune diseases, neurological disorders, and other therapeutic applications (which include ocular diseases, fat loss, and peripheral arterial diseases). In 2020, the musculoskeletal disorders segment accounted for the largest share of the therapeutic application segment. The large market share of this segment is attributed to the increasing prevalence of musculoskeletal disorders such as osteoarthritis, bone repair, and regeneration

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The Asia Pacific region is the fastest-growing region of the Stem Cell Therapy Market in 2020.

The Asia Pacific region is estimated to grow at the highest CAGR in the market during the forecast period. Some of the major factors fueling the growth of the APAC market include regulatory approvals and guidelines for product approvals and the presence of major stem cell players in countries such as South Korea, Japan, India, and Australia.

Key players in the Stem Cell Therapy Market include Smith & Nephew (UK), MEDIPOST Co., Ltd. (South Korea), Anterogen Co., Ltd. (South Korea), PHARMICELL Co., Ltd. (South Korea), JCR Pharmaceuticals Co., Ltd. (Japan), and NuVasive, Inc. (US).

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Stem Cell Therapy Market worth $401 million by 2026 - Exclusive Report by MarketsandMarkets - PRNewswire

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy Market worth $401 million by 2026 – Exclusive Report by MarketsandMarkets – PRNewswire | May 3rd, 2021

Lorraine LaRosa faced a seemingly impossible decision.

She knew how fortunate she was to have not one, but three perfect matches for a bone marrow transplant, a procedure used to treat several cancerous and noncancerous diseases, such as leukemia and Hodgkins lymphoma. The statistics for finding a perfect match can be grim. The best odds rest in an immediate family member. Otherwise, a patient must rely on the bone marrow registry and the slim chance of matching with a stranger.

Ms. LaRosa had the benefit of a large family. Out of seven siblings, four were healthy enough to be tested. And three came back as matches: her sisters Jennifer Lappe and Melissa Senatore, who live in Calverton, and her brother Jason Klinge of Southampton.

I was in tears because I didnt know what to do and who was the better choice, Ms. LaRosa said.

A sense of urgency had arrived in early 2020. Ms. LaRosa, 62, who lives in Mattituck, was undergoing frequent blood transfusions due to a lack of platelets and low red blood cells.

Things were getting pretty bad at that point, she said.

She didnt want to put the burden on any of her siblings. She called her doctor to talk through her concerns. The time had come to move forward, so the doctor took the decision out of her hands.

After careful evaluation of all three siblings health and medical histories and considering Ms. LaRosas worsening situation the doctors choice became clear. The donor would be her sister Jennifer.

Ms. Lappe understood her sisters hesitancy to ask forsuch a serious commitment. But there was never a moment of doubt. She had seen her sister struggle for years with her illness and have to endure the uncertainty of misdiagnosis and multiple procedures.

I knew she was scared, Ms. Lappe said. Id be scared with what she had to go through. But shes a lot stronger than I think she thinks she is.

Ms. LaRosa texted her sister with the news that unfortunately she would be the donor.

To me there was never a question, said Ms. Lappe, 60. Ill do whatever you need. Im in a million percent. I said, Im selfish, I love you. I want you to be around forever.

The sisters, who were always so close from a young age and grew up in a tight-knit family, would soon form an unbreakable bond one saving the others life.

Before Ms. LaRosa received an ultimate diagnosis of myelodysplastic syndrome, or MDS, in February 2020, she endured years of joint pain and symptoms that seemed to mystify her doctors. One time when her shoulder hurt, she was told it was a torn rotator cuff, which turned out to be inaccurate. Before that, when she was struggling to walk with swollen feet, a podiatrist said she had a torn Achilles tendon, but she hadnt done anything that seemed to warrant an injury typically seen in athletes. Lupus was also incorrectly diagnosed.

She struggled on a continual journey from one doctor to another.

She ended up in an emergency room on Feb. 27, 2017, and a doctor there noticed that her platelets the smallest blood cells seemed low.

Two months later, a doctor at New York Cancer & Blood Specialists diagnosed large granular leukemia, a rare cancer of white blood cells. As time went on, however, her condition did not improve.

Ms. LaRosas daughter, Taylor, described her mom as a fighter who was always optimistic and never overly concerned about her health issues.

I was more so the worrywart, said Taylor, 29. I kind of forced her to go to all these appointments and all of these doctors chasing all of these kind of vague symptoms. No one could kind of come up with what was going on.

A 2009 Mattituck High School graduate, Taylor works as a physician assistant at Weill Cornell Medical Center in New York City. She connected her mom with Dr. Gail Roboz, who specializes in hematology/oncology, in November 2017. Dr. Roboz became well known as the doctor for Robin Roberts, an anchor of ABCs Good Morning America, who was diagnosed with MDS in 2012.

[Dr. Roboz] kind of took on my case and was monitoring me and was saying my blood didnt really make any sense, Ms. LaRosa said. There were mutations in my blood that werent making sense for the large granular leukemia diagnosis.

Extensive testing revealed that Ms. LaRosa had a predisposition to MDS, a bone barrow disorder and blood cancer that often goes unrecognized and under-diagnosed, according to the MDS Foundation.

Then it was kind of a weird watching and waiting game, Taylor said. I think we all hoped it couldnt turn into this [MDS], but we knew it could.

Low-risk patients who do not receive a bone marrow transplant face an average survival rate of up to six years, according to the MDS Foundation. High-risk patients face as little as five months.

Taylor said she braced herself for the possibility that her mother would need a bone marrow transplant at some point. Each December, her mother would undergo a checkup with her oncologist.

Taylor examined her mothers lab work after the December 2019 appointment and could see the results were abnormal. The family had booked a cruise around Christmastime, so Taylor reached out to her mothers oncologist to see if it would be safe to travel.

Dr. Roboz gave them the OK and said theyd deal with it when they returned.

We went on this cruise and I didnt know anything, Ms. LaRosa said. My husband didnt know anything, but my daughter had all this information. She had some emotional moments on the cruise. Now, looking back, I know why.

Taylor recalled the trip to the Bahamas like something out of a movie, where nothing went wrong.

It was like a perfect trip, she said.

When Ms. LaRosa returned to her doctor for a follow-up, the reality of the situation set in. Dr. Roboz referred her to Dr. Tsiporah Shore, who has expertise in bone marrow transplants, at Weill Cornell Medicine. They met on March 14, 2020.

She basically said we need to do this right away, Ms. LaRosa said. Things were really progressing.

Ms. Lappe could see her sisters health was declining.

Nothing they did was making her better and I know this was something she dreaded doing, she said.

Before determining who would be the match, Ms. Lappe said she underwent the most extensive testing of her life. To find a match, doctors analyze the patients tissue type, specifically the human leukocyte antigen, or HLA, tissue, the proteins found on most cells in the body, according to the nonprofit organization Be the Match.

Finding the match is just the initial step in assuring that the donor is suitable for the transplant and there are no other potential ailments that could be passed on.

Ms. Lappe had assumed her brother Jason would be the pick since she had an autoimmune disease and he did not. When she found out she had been selected, it coincided with the early stage of the pandemic. That added another layer of stress, since Ms. Lappe knew if she came down with the virus, it could upend the entire process.

Other questions loomed over her.

Youre worrying, is my body going to do what it needs to do? Is it going to work? Will her body reject it? she said.

To begin the donation last July, Ms. Lappe received injections to increase her white blood cell count. At the same time, her sister was undergoing radiation and chemotherapy to essentially wipe her immune system clear, eliminating a lifetime of protections that had been built up.

Ms. Lappe said she had been warned shed feel pain in her bones from the shots. When she didnt feel anything after the first shot, she worried it might not be working.

By the third and fourth shot, there was no mistaking the odd sensation.

You have these bone pains, she said. Ive never had that happen.

On the fifth day, the doctors did a blood test as the final determination to begin the donation process.

To read more about bone marrow donation, visit BeTheMatch.org.

The procedure, called peripheral blood stem cell donation, required Ms. Lappe to be connected to a machine for six hours as blood was removed via a port in her chest to separate out the blood-forming cells. The remaining blood circulated back into her body.

At the end of it, one bag of the pinkish liquid that would be used to save her sister had been accumulated.

I said to her afterwards, it was so emotional, Ms. Lappe said, adding that she knew she would feel an overwhelming sense of guilt if the procedure didnt work.

She took a picture of the bag and its label, which read, Donor: Jennifer Lappe and Recipient: Lorraine LaRosa. She texted the picture to her sister and said, Oh, my gosh.

Ms. Lappe finished her donation on a Wednesday and her sister began to receive her bone marrow the next morning, once the doctors had determined they had a sufficient number of stem cells to start the process.

Then the waiting game began.

The day of a transplant is Day Zero. Every day afterward continues an upward count toward engraftment, when the blood-forming cells received during the transplant begin to grow and create healthy blood cells.

I would say those days were the hardest, just waiting, Taylor said. They would draw her labs every morning at 4 a.m. and the results would be back at 6 a.m.

A nurse would write the number on a board, and for several days it remained at zero. To pass the time, Ms. LaRosa would play games like Yahtzee with her husband, Mark, who commuted each day into the city. Taylor would watch Netflix shows like Jane the Virgin with her mom. The days were largely a blur for Ms. LaRosa.

Taylor knew it could take one to two weeks for engraftment to begin. It was Day 11 when they saw the first sign of hope as a nurse wrote .1 on the board, signaling the first sign of growth.

I remember that day being like a huge relief and huge turn, Taylor said.

Ms. LaRosa spent over a month in the hospital for close monitoring as her counts continued to climb. Even after she was released, she had to stay at a nearby hotel for another week because of daily checkups. She set her sights on Day 100, another milestone moment in the recovery.

If you make past Day 100, its a good thing, she said.

Even after a successful procedure with a 100% match, theres never a moment of being entirely in the clear. Ms. LaRosa will continue to be monitored for the rest of her life and setbacks are always possible. Shes faced one setback already, with graft vs. host disease, which can be common after a bone marrow transplant. Shes also endured blood clots.

But the biggest thing is that shes now clear of MDS and feeling better than before the procedure. She still, however, faces residual effects from chemotherapy. Shes often tired.

When she returned home, she mostly stayed inside, unable to venture out with the threat of COVID-19 still hanging over everything. Her immune system was rebuilding from scratch. She remains on a special diet. She cant have plants in the house, which put her at risk of exposure to pathogens that can cause disease. She cant have alcohol.

I said, God, I really want a glass of wine, Ms. LaRosa said with a laugh.

Taylor said there are constant signs of progress. Her mother just recently had a port removed from her chest wall after close to nine months. She received her COVID-19 vaccine. Her hair is growing back.

Shes starting to like the style, Taylor said.

She looks forward to the next steps of returning to normal: going to a movie theater and eating dinner at their favorite restaurant, Grana in Jamesport. When they sit together and toast their wine glasses, Taylor said she knows shell cry. They have always shared a close bond, particularly since Taylor was adopted at around 3 months old after her mother endured years of infertility issues.

Shes been my best friend and my rock for my whole life, Taylor said.

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Two local sisters share an unbreakable bond after bone marrow donation - Riverhead News Review - Riverhead News Review

Bone Marrow Stem Cells Comments Off on Two local sisters share an unbreakable bond after bone marrow donation – Riverhead News Review – Riverhead News Review | May 3rd, 2021

Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

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Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

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Bone Marrow Processing Systems Market Quantitative Market Analysis, Current and Future Trends - Good News Gum

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing Systems Market Quantitative Market Analysis, Current and Future Trends – Good News Gum | May 3rd, 2021

THE FAMILY of a nine-year-old leukaemia patient have been given 10 days to raise funds for life-saving treatment.

Nathaniel Nabenas family are appealing as he "clings on to life" after they were told they have until May 12 to find 201,000 for a stem cell transplant.

2

Without the operation, his cancer will be terminal.

Nathaniel is not entitled to free NHS treatment because he is not a British national.

He flew to the UK to have a 5,000 prosthetic eye fitted privately after losing it to a tumour in his home country Nigeria.

Doctors at Londons Great Ormond Street Hospital, moved by Nathaniels plight, have revealed they have waived their private consultant fees to help.

And Paul OGrady, who presents ITV series Little Heroes at the childrens hospital, has voiced his support.

It was only when he arrived here in November that doctors discovered he had acute myeloid leukaemia, a cancer so aggressive that he could have died within weeks without chemotherapy.

A stem cell match has been found but the family now have to find 201,103.

This money goes to the NHS for the cost of the transplant, treatment and after-care, based on a typical in-patient admission of eight weeks and a three-month follow-up as an outpatient.

2

Nathaniels cancer is in remission after six rounds of chemo but his consultant says it could return at any time.

If they raise enough, then a transplant will go ahead after tests due to take place on May 14.

Parents Ebisidor, a business analyst, and wife Modupe, 38, who are staying with family in Croydon, South London, were initially told the hospital bill could be as much as 825,000.

Ebisidor, 45, told the Mirror: Weve seen a dramatic turnaround from the hopeless situation we were in six months ago and we cant thank Sunday People readers enough.

Its incredible that the doctors are treating him in their private work without charging. They are wonderful. We are so grateful to everyone for giving us hope but at the same time asking people to help Nathaniel cling on to life. We know its a lot to ask.

Professor Ajay Vora, a consultant paediatric haematologist at GOSH, said the superhero fan had been incredibly brave.

But he warned: The cancer could come back at any time and the longer we wait the more likely it will return. Then Nathaniel will only have the option of palliative care.

"The tests we are doing in two weeks will reassure us it hasnt started to come back before we give him the transplant.

Prof Vora added: All the consultants involved in his care are working in a private capacity and have waived their fee because they want to help him.

Our time is not borrowed from the NHS because we are treating Nathaniel in our private service in our time.

Doctors had hoped Nathaniel would be able to have a bone marrow transplant from one of his two sisters Nadia, 11, and Nicole, 21 months. But they were not a match.

Instead, stem cells from a stored umbilical cord will be used to save him.

Doctors will give Nathaniel high doses of chemotherapy to kill off his stem cells and replace them with healthy ones.

Dad Ebisidor said: The faster we can do this transplant the more chance Nathaniel has of survival.

"We dont have this sort of treatment back home. We didnt bring him to the UK sick. He got poorly while he was here. If the operation doesnt work our only option will be to take him to a hospice.

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By law, non-UK residents get free emergency care but are charged for operations if they are admitted to hospital.

They pay for treatment at 150% of the NHS national tariff the cost normally incurred for eligible patients.

A Great Ormond Street spokesman said: Nathaniel has responded well to treatment, with our clinical teams working to provide the best care for him including looking at taking advantage of the short window of time for receiving a transplant.

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Desperate family of boy, 9, with leukaemia have 10 days to save his life... - The Sun

Bone Marrow Stem Cells Comments Off on Desperate family of boy, 9, with leukaemia have 10 days to save his life… – The Sun | May 3rd, 2021

The objective of the study is to define market sizes of different segments and countries in previous years and to forecast the values to the next Five years. The report is designed to incorporate both qualify qualitative and quantitative aspects of the industry with respect to each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as drivers and restraining factors which will define the future growth of the Hematopoietic Stem Cell Transplantation (HSCT) market.

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Major Participators LandscapeThese market players enjoyed broad industry coverage, outstanding operational ability, and strong financial resources. Manufacturers are focusing on product innovation, brand extension, and the introduction of new brands to cater to the preferences of consumers. Some of them will be endowed with vital future while others will show a weak growth during the prospective timeframe.Major market participators covered in our report are:Cryo-Save AG ViaCord Inc Lonza Group Ltd Pluristem Therapeutics Inc China Cord Blood Corp CBR Systems Inc Regen Biopharma Inc Escape Therapeutics Inc

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Hematopoietic Stem Cell Transplantation (HSCT) Market: Application OutlookPeripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)

By Type:Allogeneic Autologous

Table of Content1 Report Overview1.1 Product Definition and Scope1.2 PEST (Political, Economic, Social and Technological) Analysis of Hematopoietic Stem Cell Transplantation (HSCT) Market2 Market Trends and Competitive Landscape3 Segmentation of Hematopoietic Stem Cell Transplantation (HSCT) Market by Types4 Segmentation of Hematopoietic Stem Cell Transplantation (HSCT) Market by End-Users5 Market Analysis by Major Regions6 Product Commodity of Hematopoietic Stem Cell Transplantation (HSCT) Market in Major Countries7 North America Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis8 Europe Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis9 Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis10 Latin America, Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis 11 Major Players Profile

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Regional Segment AnalysisThe report focuses on detailed analysis of major regions like North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Columbia), and Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa).

Report Key AudienceHematopoietic Stem Cell Transplantation (HSCT) manufacturersDownstream vendors and end-usersTraders, distributors, and resellers of Hematopoietic Stem Cell Transplantation (HSCT)Hematopoietic Stem Cell Transplantation (HSCT) industry associations and research organizationsProduct managers, Hematopoietic Stem Cell Transplantation (HSCT) industry administrator, C-level executives of the industriesMarket Research and consulting firms

Hematopoietic Stem Cell Transplantation (HSCT) Report Provide:Potential opportunities and challenges analysis in Hematopoietic Stem Cell Transplantation (HSCT) market.Current and future market outlook in the developed and emerging regional markets.Detailed analysis of the segment that is expected to dominate the market.Regions that are expected to witness the fastest growth during the forecast period.Identify the latest developments, market shares, and strategies employed by the major market players.Comprehensive & in-depth research and after-sales warranty by Global Market Monitor.Analysis of Influences of COVID-19 to the present and future Hematopoietic Stem Cell Transplantation (HSCT) market and related industry.

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Hematopoietic Stem Cell Transplantation (HSCT) Global Market Report (2020-2027) Segmented by Type, Application and region (NA, EU, and etc.) The...

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Global Market Report (2020-2027) Segmented by Type, Application and region (NA, EU, and etc.) The… | May 3rd, 2021

Cell and gene therapies are overlapping fields of research and treatments. While both aim to treat and potentially cure diseases, they have slightly differing approaches and have different historical backgrounds. Due to growing interest surrounding this field, the general public still has much to learn and understand about each of these potentially life-saving therapies.

Below, we provide a general overview and brief historical context for each type of therapy.

Cell therapyis the process of replacing damaged or dysfunctional cells with new, healthy ones by transferring live cells into a patient. These can be autologous (also known as self-to-self, using cells from the patient receiving the treatment) or allogeneic (using cells from a donor for the treatment). While this field of treatment has recently begun to expand, some forms of cell therapy like the cancer-treating hematopoietic stem cell transplantation(HSCT) have been in practice for decades.

While many people have heard of bone marrow transplants, few realize that this procedure is a stem cell therapy. While stem cells can be derived from many sources, such as umbilical cord blood and mobilized peripheral blood, bone marrow derived stem cell therapy is the most commonly used today and has been for more than 50 years.

The first transfusion of human bone marrow was given to a patient with aplastic anemia in 1939. After World War II researchers diligently worked to restore bone marrow function in aplasia patients caused by exposure to radiation produced by the atomic bomb. After a decade of work they were able to show, in a mouse model, that aplasia could be overcome by bone marrow treatment.

The first allogeneic HSCT, which led the way to current protocols, was pioneered by E. Donnall Thomas and his team at the Fred Hutchinson Cancer Research Center and reported in the New England Journal of Medicine in 1957. In this study six patients were treated with radiation and chemotherapy and then received intravenous infusion of bone marrow rich stem cells from a normal donor to reestablish the damaged or defective cells. Since then the field has evolved and expanded worldwide. While almost half of HSCT are allogeneic, the majority of HSCT are autologous, the patient's own stem cells are used for treatment, which carries less risk to the patient.

In 1988, scientists discovered that they could derive stem cells from human embryos and grow the cells in a laboratory. These newly derived stem cells, referred to as embryonic stem cells (hESCs), were found to be pluripotent, meaning they can give rise to virtually any other type of cell in the body. This versatility allows hESCs cells to potentially regenerate or repair diseased tissue and organs. Two decades after they were discovered, treatments based on hESCs have been slow in coming because of controversy over their source and concerns that they could turn into tumours once implanted. Only recently, testing has begun as a treatment for two major diseases: heart failure and type 1 diabetes.

In 2006, researchers made a groundbreaking discovery by identifying conditions that would allow some cells to be 'reprogrammed' genetically. This new type of stem cell became known as induced pluripotent stem cells (iPSCs). Since this discovery, the field has expanded tremendously in the past two decades. Stem cell therapies have expanded in use and have been used to treat diseases such as type 1 diabetes, Parkinson's and even spinal cord injuries.

There has also been a growing focus on using other immune cells to treat cancer. Therapies such as CAR T-cellare dependent upon a patient's T-cells, which play a critical role in managing the immune response and killing cells affected by harmful pathogens. These cells are then reengineered to target and kill certain cancerous cells. Several CAR T-cell therapies have been FDA approved, with the first approval being given in 2017 for Yescarta and Kymriah, to be used for the treatment of B-cell leukemia in children and young adults.

Gene therapyis a process that modifies the expression of a gene or alters the biological process of living cells for therapeutic use. This process can take the form of replacing a disease-causing gene with a new, healthy one, inactivating the mutated gene, or introducing a new gene to help the patient's body fight a disease.

While the use of gene therapy to treat humans is fairly new, the science behind it has been used in science for decades. Farmers and geneticists have collaborated for years on crop improvement using cross pollination, genetic engineering and microinjection techniques to create stronger, more resilient crops.

The first human patient to be treated with gene therapy was a four-year old girlsuffering from severe combined immunodeficiencyin 1990. She received treatment for a congenital disease called adenosine deaminase (ADA). Since then, gene therapies have been used to treat diseases such as cancer, cystic fibrosis and hemophilia.In 2017, the FDA gave its first approval of a gene therapy called Luxturna, which is used to treat patients with established genetic vision loss that may result in blindness. Gene therapies are still being studied and developed, with over 1,000 clinical trialscurrently underway.

ThermoGenesis Holdings Inc., is a pioneer and market leader in the development and commercialization of automated cell processing technologies for the cell and gene therapy fields. We market a full suite of solutions for automated clinical biobanking, point-of-care applications and large-scale cell processing and manufacturing with a special emphasis on the emerging CAR-T immunotherapy market. We are committed to making the world a healthier place by creating innovative solutions for those in need.

For more information on the CAR-TXpress multi-system platform, please contact our Sales team.

Disclaimer

Thermogenesis Holdings Inc. published this content on 13 April 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 April 2021 07:10:03 UTC.

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ThermoGenesis : The History of Cell and Gene Therapy - marketscreener.com

Spinal Cord Stem Cells Comments Off on ThermoGenesis : The History of Cell and Gene Therapy – marketscreener.com | April 19th, 2021

LCol Laura Laycock on deployment.

LCol Laura Laycock

It was Oct. 7, 2019, and life was not just good, it was amazing.

My career in the Royal Canadian Air Force was going great. I loved my job and was getting promoted. Throughout my Canadian Armed Forces career of over 20years, I had represented Canada around the world with NORAD, NATO and the UN. I had married the most incredible man. We relocated to Ottawa, started to travel the world together, and were ready to start a family.

Then, on Oct. 8, 2019, everything changed.

I was diagnosed with Chronic Myeloid Leukemia(CML) after blood work for vertigo showed extremely elevated white blood cell counts. CML is a blood cancer where the bone marrow overproduces white blood cells, which eventually impairs the development of white and red blood cells and platelets. Its usually caused by a spontaneous mutation in DNA, which contains our genetic code.

LCol Laycock

Twenty years ago, researchers developed a new line of drugs that combat this overproduction of white blood cells. These targeted oral chemotherapy pills have been revolutionary in the fight against CML. Most people who take them do so for the rest of their lives and have good survival rates; however, a stem cell transplant remains the only actual cure. But its risky and not needed for most people.

Its now been about 17months since my diagnosis and my body has not tolerated this targeted chemotherapy. I fall into that small fraction of people who get debilitating or life-threatening side effects from this medication. My doctors are discussing other treatment options, one of which is a stem cell transplant, but my mixed ethnicity (European/Middle Eastern) has made it difficult to find a donor match.

My journey since my diagnosis has been to slow down and educate myself so that I can heal and advocate for my care; to appreciate every little moment of joy; and to do my best to overcome each challenge that arises. I have found strength in the extraordinary support Ive received from my family, my friends and my community, both old and new.

With the help of family and friends, I recently began a social media campaign to increase stem cell donor education and registration in Canada and around the world. Many people are unaware of the potentially lifesaving role they can play by registering to become stem cell donors. Stem cell transplants are vital treatment options for people with a range of medical conditions including spinal cord injuries, heart disease, diabetes, and some cancers.

The process to donate is simple. First, you register online with Canadian Blood Services or Hma-Qubec and do a mail-in cheek swab., and then you wait. It could be months or years before you are identified as a match. During this waiting period, you should update your contact information with the registry if it changes.

When you are matched, you will be contacted to continue with the donation process. This process is similar to giving blood, but it has its differences. The cells are usually collected intravenously from peripheral blood in a non-surgical procedure but, in rare cases, they are collected directly from the bone marrow in a surgical procedure. In either case, the risks associated with donating are minor.

In Canada, individuals aged17 to 35 can register to become stem cell donors (ages18 to 35 in Quebec). Both CBS and Hma-Qubec are part of an international network of donor registries from over 50countries. This network has a pool of over 38million donors but, unfortunately, matches are rare.

Your stem cells could potentially help others around the world, and throughout this process donor privacy is assured at all times.

LCol Laycock on her wedding day.

Stem cell matching relies on Human Leukocyte Antigen typing, which is highly influenced by ethnicity. This means that a patients best chance of finding a matching donor is from those who share similar ethnic backgrounds. Research conducted by Gragert et al.(2014) has shown that the likelihood of finding a match for certain ethnic groups can be as low as 16 percent and as high as 75 percent for others. This disparity highlights the need for more ethnically diverse stem cell donors in our registries.

Today, I am calling on my DND and CAF families to register as stem cell donors to help people, like me, who are fighting for our lives. If you arent able to register, please share this call with those who can. You, or someone you know, could be the match that saves a life a simple swab is all it takes to be a hero.

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From an insight perspective, this research report has focused on various levels of analysis industry trends analysis, top players analysis, company profiles, which discuss the basic views on the competitive landscape, emerging and high-growth segments of Autologous Stem Cell Based Therapies market, and high-growth regions. Besides, drivers, restraints, challenges, and opportunities pertaining to Autologous Stem Cell Based Therapies market are also predicted in this report.

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Major Participators LandscapeThese market players enjoyed broad industry coverage, outstanding operational ability, and strong financial resources. Manufacturers are focusing on product innovation, brand extension, and the introduction of new brands to cater to the preferences of consumers. Some of them will be endowed with vital future while others will show a weak growth during the prospective timeframe.Major market participators covered in our report are:US STEM CELL, INC. Med cell Europe Pluristem Therapeutics Inc Mesoblast Tigenix Brainstorm Cell Therapeutics Regeneus

To Get More Information on The Regional Analysis Of Autologous Stem Cell Based Therapies Market, Click Here:https://www.globalmarketmonitor.com/reports/643098-autologous-stem-cell-based-therapies-market-report.html

Autologous Stem Cell Based Therapies Application AbstractThe Autologous Stem Cell Based Therapies is commonly used into:Neurodegenerative Disorders Autoimmune Diseases Cardiovascular Diseases

Autologous Stem Cell Based Therapies Type AbstractBased on the basis of the type, the Autologous Stem Cell Based Therapies can be segmented into:Embryonic Stem Cell Resident Cardiac Stem Cells Umbilical Cord Blood Stem Cells

Table of Content1 Report Overview1.1 Product Definition and Scope1.2 PEST (Political, Economic, Social and Technological) Analysis of Autologous Stem Cell Based Therapies Market2 Market Trends and Competitive Landscape3 Segmentation of Autologous Stem Cell Based Therapies Market by Types4 Segmentation of Autologous Stem Cell Based Therapies Market by End-Users5 Market Analysis by Major Regions6 Product Commodity of Autologous Stem Cell Based Therapies Market in Major Countries7 North America Autologous Stem Cell Based Therapies Landscape Analysis8 Europe Autologous Stem Cell Based Therapies Landscape Analysis9 Asia Pacific Autologous Stem Cell Based Therapies Landscape Analysis10 Latin America, Middle East & Africa Autologous Stem Cell Based Therapies Landscape Analysis 11 Major Players Profile

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Major countries of North America, Europe, Asia Pacific, and the rest of the world are all exhaustive analyzed in the report. Apart from this, policy mobilization, social dynamics, development trends, and economic development in these countries are also taken into consideration.

Target Audience for this Report Autologous Stem Cell Based Therapies manufacturers Autologous Stem Cell Based Therapies traders, distributors, and suppliers Autologous Stem Cell Based Therapies industry associations Product managers, Autologous Stem Cell Based Therapies industry administrator, C-level executives of the industries Market Research and consulting firms Research & Clinical Laboratories

Report SpotlightsDetailed overview of marketChanging market dynamics in the industryIn-depth market segmentationHistorical, current and projected market size in terms of volume and valueRecent industry trends and developmentsCompetitive landscapeStrategies of key players and products offeredPotential and niche segments, geographical regions exhibiting promising growthA neutral perspective on market performanceMust-have information for market players to sustain and enhance their market footprints

About Global Market MonitorGlobal Market Monitor is a professional modern consulting company, engaged in three major business categories such as market research services, business advisory, technology consulting.We always maintain the win-win spirit, reliable quality and the vision of keeping pace with The Times, to help enterprises achieve revenue growth, cost reduction, and efficiency improvement, and significantly avoid operational risks, to achieve lean growth. Global Market Monitor has provided professional market research, investment consulting, and competitive intelligence services to thousands of organizations, including start-ups, government agencies, banks, research institutes, industry associations, consulting firms, and investment firms.ContactGlobal Market MonitorOne Pierrepont Plaza, 300 Cadman Plaza W, Brooklyn,NY 11201, USAName: Rebecca HallPhone: + 1 (347) 467 7721Email: info@globalmarketmonitor.comWeb Site: https://www.globalmarketmonitor.com

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Global Autologous Stem Cell Based Therapies Market Survey Report, 2020-2027 KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper

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Children and young adults who underwent an allogeneic hematopoietic stem cell transplant (alloHSCT) after achieving complete response with CD19 CAR T-cell therapy experienced durable B-cell acute lymphoblastic leukemia (B-ALL) control, according to the results of a phase 1 trial (ClinicalTrials.gov Identifier: NCT01593696) published in the Journal of Clinical Oncology.

Although a proportion of patients who undergo CAR T-cell therapy go on to receive alloHSCT, the study authors stated that The role for [alloHSCT] following CD19-CAR T-cell therapy to improve long-term outcomes in [children and young adults] has not been examined.

The phase 1 trial evaluated 50 children and young adults with B-ALL who received CD19.28 CAR T-cell therapy. The primary objective was to determine the maximum tolerated dose of CAR T cells, toxicity, and feasibility of generating CAR T cells in the study population. In addition, this analysis retrospectively evaluated the effect of alloHSCT on survival after CAR T-cell therapy.

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At baseline, the median age was 13.5 years (range, 4.3-30.4), and 40 (80%) of the patients were male. The median number of prior regimens was 4 (range, 4.3-30.4); 22 (44%) patients had at least 1 prior HSCT, 2 (4%) had prior CD19-targeted therapy, and 5 (10%) of the patients had prior treatment with blinatumomab.

Complete response was achieved in 31 (62%) of the patients. Among these patients, 28 (90.3%) were negative for minimal residual disease. Higher rates of complete response were associated with primary refractory disease, fewer prior lines of therapy, M1 marrow, or fludarabine/cytarabine-based lymphodepletion. The median overall survival was 10.5 months (95% CI, 6.3-29.2) during a median follow-up of 4.8 years.

Of the 28 patients who achieved complete response, 21 (75%) proceeded to undergo consolidative alloHSCT. The median overall survival for these patients was 70.2 months (95% CI, 10.4-not estimable), with an event-free survival not yet reached. The rate of relapse after alloHSCT was 4.8% (95% CI, 0.3-20.3) at 12 months and 9.5% (95% CI, 1.5-26.8) at 24 months.

Any grade cytokine release syndrome (CRS) developed among 35 (70%) patients, with 9 (18%) experiencing grade 3 to 4 CRS. Of the 10 patients (20%) who developed neurotoxicity, 4 cases were severe. One cardiac arrest occurred during CRS. All patients with CRS, neurotoxicity, and cardiac arrest recovered.

The authors concluded that CD19.28 CAR T cells followed by a consolidative alloHSCT can provide long-term durable disease control in [children and young adults] with relapsed or refractory B-ALL.

Disclosure: Please see the original reference for a full disclosure of authors affiliations.

Reference

Shah NN, Lee DW, Yates B, et al. Long-term follow-up of CD19-CAR T-cell therapy in children and young adults with B-ALL. J Clin Oncol. Published online March 25, 2021. doi:org/10.1200/JCO.20.02262c

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Durable B-ALL Control With Allogeneic Transplant After CAR T-Cell Therapy - Cancer Therapy Advisor

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Kaytlyn Gerbin, left, runs the Wonderland Trail around Mount Rainier. She completed the 93-mile loop in just under 19 hours. Her friend Tara Fraga helped with pacing between miles 30-55. (Ryan Thrower Photo)

When Kaytlyn Gerbin moved to Seattle 10 years ago to attend graduate school at the University of Washington, a friend took her to Kerry Park in the Queen Anne neighborhood on her first visit. The celebrated viewpoint offered Gerbin a glimpse of Mount Rainier that ignited an ongoing passion.

At the time, I had absolutely no idea there was a trail all the way around it, and didnt know the first thing that went into climbing to the summit or running even a few miles on the trails, Gerbin said. Since then, Ive climbed Rainier 10 times, and spent countless hours on the mountain and trails in that park.

Along with her drive to get to know Washington states most famous landmark more intimately, Gerbin achieved her PhD in bioengineering at UW, where her research was focused on the therapeutic and regenerative potential of cardiac cells. For the past four years shes been a scientist at Allen Institute for Cell Science, where she studies stem cells and cardiomyocytes, or cardiac muscle cells.

Our latest Geek of the Week, Gerbin is an accomplished ultrarunner, and she now knows a lot more about that trail that encircles Mount Rainier.

With COVID-19 lockdowns impacting her international race season last summer, Gerbin, a sponsored athlete for The North Face, went after the fastest known time, or FKT, for a run around the Wonderland Trail. Together with teammate Dylan Bowman of Portland and a small crew of local filmmakers, they made Summer of Wonder, a short film about the experience, which you can watch in full here:

The average thru-hiker takes 10-14 days to complete the 93-mile Wonderland Trail, with its 24,000 feet of elevation gain. Gerbin did it in 18 hours, 41 minutes, 53 seconds, and the film is a breathtaking look at her endurance feat.

Gerbins passion for running started with 3-mile commutes back and forth between her apartment, her research lab, and campus during grad school. Eventually she started trail running,essentially as a life hack to see if she could squeeze a five-day backpacking route into a weekend between experiments.

It turned out I was actually pretty good at that, and that opened up opportunities to start racing at some of the most competitive trail races in the U.S. and Europe, Gerbin said.

Shes since raced with Team USA at the Trail World Championships, reached the podium at the iconic Western States 100, and won races such as the Canary Islands Transgrancanaria and Cascade Crest 100 in Washington. She also still holds the womens self-supported FKT for the Rainier Infinity Loop (set in 2019), which combines the Wonderland Trail with two summits and descents of Mount Rainier.

Her preferred racing distance is anything between 50-100 miles long, the more elevation gain and technical the trail, the better. During peak training, Gerbin is usually hitting between 70-90 miles with over 20,000 feet of elevation gain each week. She calls the Pacific Northwest the best outdoor playground there is.

Although I love running fast, Im also really excited about pushing myself on more challenging terrain. So many of my other FKT goals and route ideas are along these lines, with more technical traveling than actual running, she said.

COVID permitting, her highest race priority this year is Ultra Trail du Mont Blanc, which is the most competitive world-stage for ultrarunning, at the end of August. The race circumnavigates Mont Blanc, passing through France, Italy, and Switzerland and covering around 105 miles and 33,000 feet of elevation gain.

While Gerbins experience as a scientist does inform her appreciation for what shes putting her body through during ultrarunning, shes equally passionate in the lab. At the Allen Institute shes seeking answers to broad questions about how cells work, including how single cells and all of their components are integrated into a functional system, while using imaging to build predictive models of cell behavior.

I get the opportunity to work with a multidisciplinary team of badass scientists, biologists, and engineers on really cool problems in cell biology, she said.

Learn more about our latest Geek of the Week, Kaytlyn Gerbin:

What do you do, and why do you do it? Science and ultrarunning for me have always come down to problem solving.

As a scientist, problem solving is inherent to experimental design, data analysis, and interpreting results. By asking hard questions, Im interested in pushing the field of cell biology forward, and challenging the current way of thinking.

As an ultrarunner, its a different kind of problem solving, but I lean on the same mindset to figure out how to push my athletic limits further and faster.

One thing that always amazes me is how adaptable the human body is. My training in cell science gives me context for how all of these stressors and inputs were putting on our bodies are fundamentally happening at the single cell level, and it keeps me thinking about the cells response to external cues in my research.

Whats the single most important thing people should know about your field? Yes, I do think about science and when Im running, and no, I do not geek out on heart rate monitors and training zones and all those numbers when Im running.

Where do you find your inspiration? Im inspired by brilliant women that are pushing whats possible in both science and in sports. I think we often set boundaries for ourselves about what we think is possible, without ever letting ourselves really hit that limit. Im inspired by women who set bold goals and bring others up and along for the ride, redefining whats possible.

Whats the one piece of technology you couldnt live without, and why? My Garmin 935. I use this watch daily to track miles run, elevation gain, etc. The battery life has lasted me for 100 miles of running and ~24 hrs, but its small enough to wear every day.

Whats your workspace like, and why does it work for you? Prior to 2020, I was splitting my time between the tissue culture hood (passaging cells, differentiating cardiomyocytes, setting up experiments), conference rooms (team science and collaboration means a lot of group discussions!), and my computer for writing and analysis. Since then, Ive shifted my work to be more remote while I work on a few different manuscripts. I have an office set up at home with a window, some good tunes, plenty of coffee, and a chair for my dog to wait impatiently on.

Your best tip or trick for managing everyday work and life. (Help us out, we need it.) I have always been a to-do list person. Most mornings start with me listing out tasks (and breaking those down into many sub-tasks). I feel productive as I cross things off, and it also helps me prioritize and plan ahead to make sure I can also fit my training runs in.

Mac, Windows or Linux? Mac as a personal preference, Windows for my work computer (I do work at the Paul Allen Institute 🙂

Transporter, Time Machine or Cloak of Invisibility? Transporter. I just promise not to use it in races.

Greatest game in history: Lode Runner. I havent played it since I was a kid, but the memories of yelling at the computer with my sister frantically hitting up-down-up-down arrows make me feel like it was just yesterday.

Best gadget ever: Garmin inReach mini satellite messaging and SOS call, all in a device small enough to throw in the bottom of a pack (or shorts pocket) and forget its there. I bring this with me anytime Im headed out into the wilderness/mountains, but I hope I never need to use it.

First computer: iMac G3.

Current phone: iPhone 11.

Favorite app: I have a love/hate relationship with Strava. Ive also been using DuoLingo during the pandemic and have a strong daily streak going!

Most important technology of 2021: COVID vaccines!!

Most important technology of 2023: Advancements in remote/low-resource medical care.

Final words of advice for your fellow geeks: Most problems can be solved with more snacks and some time (works for science and running).

Twitter: @kaytlyn_gerbin

LinkedIn: Kaytlyn Gerbin

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Kaytlyn Gerbin is blazing trails in cell science and as an ultrarunner who has conquered Mount Rainier - GeekWire

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