Newswise Princess Margaret scientists have revealed how stem cells are able to generate new blood cells throughout our life by looking at vast, uncharted regions of our genetic material that hold important clues to subtle biological changes in these cells.

The finding, obtained from studying normal blood, can be used to enhance methods for stem cell transplantation, and may also shed light into processes that occur in cancer cells that allow them to survive chemotherapy and relapse into cancer growth many years after treatment.

Using state-of-the art sequencing technology to perform genome-wide profiling of the epigenetic landscape of human stem cells, the research revealed important information about how genes are regulated through the three-dimensional folding of chromatin.

Chromatin is composed of DNA and proteins, the latter which package DNA into compact structures, and is found in the nucleus of cells. Changes in chromatin structure are linked to DNA replication, repair and gene expression (turning genes on or off).

The research by Princess Margaret Cancer Centre Senior Scientists Drs. Mathieu Lupien and John Dick is published in Cell Stem Cell, Wednesday, November 25, 2020.

We dont have a comprehensive view of what makes a stem cell function in a specific way or what makes it tick, says Dr. Dick, who is also a Professor in the Department of Molecular Genetics, University of Toronto.

Stem cells are normally dormant but they need to occasionally become activated to keep the blood system going. Understanding this transition into activation is key to be able to harness the power of stem cells for therapy, but also to understand how malignant cells change this balance.

Stem cells are powerful, potent and rare. But its a knifes edge as to whether they get activated to replenish new blood cells on demand, or go rogue to divide rapidly and develop mutations, or lie dormant quietly, in a pristine state.

Understanding what turns that knifes edge into these various stem cell states has perplexed scientists for decades. Now, with this research, we have a better understanding of what defines a stem cell and makes it function in a particular way.

We are exploring uncharted territory, says Dr. Mathieu Lupien, who is also an Associate Professor in the Department of Medical Biophysics, University of Toronto. We had to look into the origami of the genome of cells to understand why some can self-renew throughout our life while others lose that ability. We had to look beyond what genetics alone can tell us.

In this research, scientists focused on the often overlooked noncoding regions of the genome: vast stretches of DNA that are free of genes (i.e. that do not code for proteins), but nonetheless harbour important regulatory elements that determine if genes are turned on or off.

Hidden amongst this noncoding DNA which comprise about 98% of the genome - are crucial elements that not only control the activity of thousands of genes, but also play a role in many diseases.

The researchers examined two distinct human hematopoietic stem cells or immature cells that go through several steps in order to develop into different types of blood cells, such as white or red blood cells, or platelets.

They looked at long-term hematopoietic stem cells (HSCs) and short-term HSCs found in the bone marrow of humans. The researchers wanted to map out the cellular machinery involved in the dormancy state of long-term cells, with their continuous self-renewing ability, as compared to the more primed, activated and ready-to-go short-term cells which can transition quickly into various blood cells.

The researchers found differences in the three-dimensional chromatin structures between the two stem cell types, which is significant since the ways in which chromatin is arranged or folded and looped impacts how genes and other parts of our genome are expressed and regulated.

Using state-of-the-art 3D mapping techniques, the scientists were able to analyze and link the long-term stem cell types with the activity of the chromatin folding protein CTCF and its ability to regulate the expression of 300 genes to control long-term, self-renewal.

Until now, we have not had a comprehensive view of what makes a stem cell function in a particular way, says Dr. Dick, adding that the 300 genes represent what scientists now think is the essence of a long-term stem cell.

He adds that long-term dormant cells are a protection against malignancy, because they can survive for long periods and evade treatment, potentially causing relapse many years later.

However, a short-term stem cell that is poised to become active, dividing and reproducing more quickly than a long-term one, can gather up many more mutations, and sometimes these can progress to blood cancers, he adds.

This research gives us insight into aspects of how cancer starts and how some cancer cells can retain stem-cell like properties that allow them to survive long-term, says Dr. Dick.

He adds that a deeper understanding of stem cells can also help with stem cells transplants for the treatment of blood cancers in the future, by potentially stimulating and growing these cells ex vivo (out of the body) for improved transplantation.

The research was supported by The Princess Margaret Cancer Foundation, Ontario Institute for Cancer Research, Canadian Institutes for Health Research (CIHR), Medicine by Design, University of Toronto, Canadian Cancer Society Research Institute, and the Terry Fox Research Institute.

Competing Interests

Dr. John Dick served on the SAB at Trillium Therapeutics, and has ownership interest (including patents) in Trillium Therapeutics. He also reports receiving a commercial research grant from Celgene.

About Princess Margaret Cancer Centre

Princess Margaret Cancer Centre has achieved an international reputation as a global leader in the fight against cancer and delivering personalized cancer medicine. The Princess Margaret, one of the top five international cancer research centres, is a member of the University Health Network, which also includes Toronto General Hospital, Toronto Western Hospital, Toronto Rehabilitation Institute and the Michener Institute for Education at UHN. All are research hospitals affiliated with the University of Toronto. For more information: http://www.theprincessmargaret.ca

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Mapping out the mystery of blood stem cells - Newswise

Recommendation and review posted by Bethany Smith

Novartis has licensed a new stem cell therapy from Mesoblast, just weeks after the FDA rejected the Australian biotechs pitch for an approval on a separate indication.

The Swiss pharma announced Thursday afternoon it is partnering with Mesoblast $MESO to develop remestemcel-L for the treatment of acute respiratory distress syndrome, including ARDS related to Covid-19. As part of the deal, Novartis will shell out $25 million in upfront cash and take a $25 million stake in the biotech, while offering up to $1.255 billion in potential milestone payments.

Mesoblast investors embraced the news, sending shares up 11% on the Australian stock exchange Friday. The companys stock was also up roughly 17% on the Nasdaq before Fridays opening bell.

The milestone payments are split as such, per Mesoblast: $505 million will be available pre-commercialization, with an additional $750 million set aside for hitting certain sales targets and double-digit royalties.

Remestemcel-L, or Ryoncil, acts as an anti-inflammatory and consists of culture-expanded mesenchymal stem cells derived from a bone marrow donor. Currently, the drug is being evaluated in a Phase III study for Covid-19-related ARDS with 300 patients, and the first cut of data is expected in early 2021.

Should that outcome prove successful, Novartis will launch a Phase III in non-Covid ARDS after the deal closes. The companies highlighted Novartis ability to rapidly scale up cell-based therapies from the clinic to the commercial phase as a motivator for the collaboration.

The drug had been examined in a small compassionate use program for Covid-19 ARDS back in March, which included 12 patients requiring ventilators. Remestemcel-L treatment demonstrated an 83% survival rate in that program and was the basis for the ongoing Phase III.

Thursdays deal comes less than two months after the FDA issued a CRL for remestemcel-L in Mesoblasts pediatric acute graft-versus-host disease program. The rejection, which denied the company an accelerated approval, came after an ODAC adcomm in August voted 9 to 1 in favor of approval as panel members struggled to envision what a pivotal trial might look like.

During both the adcomm and in their CRL, regulators took issue with Mesoblasts study design given that the company submitted its application on the basis of one, single-arm and open-label trial. In the study, Remestemcel-L demonstrated a statistically significant benefit in its primary endpoint against the historical control rate.

But because many parents and pediatricians are reluctant to risk putting children into the placebo arm of a randomized study, Mesoblast argued that key opinion leaders said an additional study was not feasible. The veto came despite the FDA approving a similar drug Incyte and Novartis Jakafi based on one single-arm trial, something for which ODAC members chastised the FDA.

Earlier this week, Mesoblast met with the agency for its Type A meeting, and the company reported in its third quarter earnings that it does not expect the FDA to reverse its decision for accelerated approval. Mesoblast is still waiting to receive final meeting minutes to know whether thats indeed the case. The CRL set back potential approval in GvHD from 2021 to 2024, per analysts.

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Novartis bags Mesoblast's stem cell therapy for ARDS, including in Covid-19, in a deal worth up to $1.2B+ - Endpoints News

Recommendation and review posted by Bethany Smith

When a compatible bone marrow donor is found for someone in need, it must reach them in fewer than 48 hours no mean feat if they are on the other side of the world.

The COVID-19 crisis bought the planet spinning to a halt, grounding planes, yet it has failed to stop the process of stem cell delivery.

Transplants can be a matter of life and death for those with a blood disease so transplant centres have been forced to take decisive action during the pandemic.

The donations are no longer transported fresh but are cryogenically frozen in anticipation of longer delivery times.

Relays of couriers were organised at borders to move the precious bone marrow, while the crew members of planes were asked to escort the cooler bags to their destinations.

Some of the couriers have not been home for months, flying from one end of the globe to the other and sleeping in airport lounges.

In Spain, the airport civil guard facilitated one exchange between transporters who were unable to meet and in another, military planes collected stem cells from Turkey to save a child in Rome.

Nonetheless, the impact of COVID-19 has been felt in the sector.

Volunteers on donor registers have practically halved in Europe during 2020 and when looking at the situation in the EU's largest countries, everywhere tells the same story.

Compared to last year, the new donors recorded in Italy were down 49%, 37% in Spain and 40% in Germany, while France is hoping to sign up 58% of the people it did in 2019.

In the UK, the situation is far from rosy; in Leeds, for example, 50 people have registered so far in November compared to 950 the previous year.

In Italy, the Bone Marrow Donors Association (ADMO) was forced to stop events aimed at raising awareness for the cause in schools and universities, as well as meetings in sports centres and town squares. The same happened in France.

In France and Spain, during the first wave of the coronavirus, new registrations were suspended for four months, meanwhile, in Germany, events dedicated to blood donations were put on standby due to a ban on gatherings.

Luckily, however, the decline in the number of new registrations has not caused a reduction in the number of donations and there have been very few occasions when a life-saving transplant has been postponed or cancelled.

Some countries, like Germany, managed to register 827,000 new donors in 2019, while others, such as Italy and Spain - that have fragmented regional healthcare systems - managed to log 49,000 and 36,000 new names respectively.

But even in some countries where the healthcare system is public and centralised, like in France, they still haven't managed to match the German numbers, with just 27,000 signing up.

In Germany a registration costs between 35 and 45, while in Italy this figure rises to 250 per sample and that's excluding the costs of healthcare staff.

Why are there such marked differences between European countries? In Italy, NGOs like ADMO, as well as Adoces and Adisco, are responsible for registering and taking samples. The only register in Italy is found in Genoa.

Add to this limits on the number of registrations that are funded by the state, as well as the high cost of the activity that precedes the collection of the kits, such as those of promotion, which Rome is not able to finance.

Germany is home to the main pool of donors in Europe, where a tenth of the eligible population (between 18 and 55 years) is on the register and is able to export 70% of bone marrow harvested from its citizens to help patients outside of Germany.

The German system relies on 26 independent donor centres, which are mostly private or connected to universities or transfusion centres.

Each is in charge of its own funding and recruitment strategies, has no relationship with local authorities and is able to reinvest the money raised through private donations in registering new donors.

In France, the 29 centres that recruit volunteers are linked to transplant centres, often encapsulated in structures that facilitate blood donation or public hospitals.

Compared to Germany, says Dr Evelyne Marry, director of blood marrow collection and transplant at the French Biomedicine Agency, "we made a choice on profiles based on quality, rather than quantity, focusing on young people (the younger the donor, the higher the chances of successful transplantation) and different geographical areas".

The strategy is decided by Paris, while in the German Lnder there is no central coordination.

Then there Spain, which since 2018 has decided to focus on donors under 40 a choice that has inevitably reduced the number of registrations compared to previous years.

Here the system is entirely public; the numerous Spanish centres are governed by the country's 17 autonomous communities, but each follows its own strategy for registering new donors.

"Nowhere, except in Galicia, do we use mouth swabs. Blood samples are used everywhere. Why? For so many reasons, even if none of them has anything to do with science," said the Dr Enric Carreras, director of the Spanish bone marrow donor programme, managed by the Josep Carreras Foundation.

Carreras said no communication campaigns have been carried out since the beginning of the pandemic by his private non-profit foundation, which manages registrations for the state.

"For now, people are not thinking about donations, but they are worried about saving themselves and not getting infected," he said.

"This year there has been some decline in donations and transplants but not because of a lack of donors, but because hospitals have had to cancel planned operations due to a lack of beds," according to Carreras. "If there is no bed in the ICU, in case of post-transplant complications, the surgery must be cancelled."

During the first wave, "those from Girona could not go to Barcelona to make the donation," he said, but added the foundation had found solutions to the problems posed by the pandemic "in one way or another".

All the donors registered in the various countries of the world end up in the WMDA, based in the Netherlands, a sort of world database that brings together 135 interconnected sources and registers. It has over 37 million potential donors.

"Thirty years ago, before the internet, it was basically a huge telephone directory with the names of potential donors," says Joannis Mytilineos, director of the German register ZKRD.

Whenever a hospital makes a request, a search is activated globally the marrow is sold by one transplant centre and purchased by another, with the national registry acting as an intermediary.

Countries set their own prices; the cost of one unit from Germany is between 14,000 and 17,000 euros. A marrow that comes from the USA, on the other hand, can cost as much as 30,000 dollars.

About 37% of stem cells transplanted worldwide from donors other than family members come from Germany.

Mytilineos, who has Greek origins, says German donors are compatible with so many people abroad "because of our migratory past, which DNA keeps track of".

Obviously, it is cheaper for national health systems to find donors at home and not just for greater genetic compatibility between subjects. "A transplant from Spain to Spain costs 4,500, less than a third of the marrow purchased from abroad," said Carreras.

But Marry points out: "We are not competing between registers. Each country has its own rules, there are those who have public funding, those who are private, but the philosophy of registers around the world is to raise awareness and the machine of international solidarity work."

Bone marrow donors are "an international community, the inefficiency of one country is overcome by other countries, but every country strives for the best," concludes Mytilineos.

Every weekday at 1900 CET, Uncovering Europe brings you a European story that goes beyond the headlines. Download the Euronews app to get an alert for this and other breaking news. It's available on Apple and Android devices.

Continued here:
How even the COVID-19 crisis could not stop bone marrow donations getting through - Euronews

Recommendation and review posted by Bethany Smith

NEW YORK, Nov. 25, 2020 /PRNewswire/ --Amid the COVID-19 crisis, the global market for Cell Harvesting estimated at US$233.2 Million in the year 2020, is projected to reach a revised size of US$381.4 Million by 2027, growing at a CAGR of 7.3% over the period 2020-2027.Manual, one of the segments analyzed in the report, is projected to grow at a 7.9% CAGR to reach US$284.4 Million by the end of the analysis period. After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the Automated segment is readjusted to a revised 5.6% CAGR for the next 7-year period. This segment currently accounts for a 28.3% share of the global Cell Harvesting market.

Read the full report: https://www.reportlinker.com/p05798117/?utm_source=PRN

The U.S. Accounts for Over 30.9% of Global Market Size in 2020, While China is Forecast to Grow at a 10.4% CAGR for the Period of 2020-2027

The Cell Harvesting market in the U.S. is estimated at US$72 Million in the year 2020. The country currently accounts for a 30.86% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$34.9 Million in the year 2027 trailing a CAGR of 10.4% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 6.1% and 7% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 6.6% CAGR while Rest of European market (as defined in the study) will reach US$34.9 Million by the year 2027.We bring years of research experience to this 5th edition of our report. The 226-page report presents concise insights into how the pandemic has impacted production and the buy side for 2020 and 2021. A short-term phased recovery by key geography is also addressed.

Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05798117/?utm_source=PRN

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE I-1

II. EXECUTIVE SUMMARY II-1

1. MARKET OVERVIEW II-1 Cell Harvesting - A Prelude II-1 Impact of Covid-19 and a Looming Global Recession II-1 With Stem Cells Holding Potential to Emerge as Savior for Healthcare System Struggling with COVID-19 Crisis, Demand for Cell Harvesting to Grow II-1 Select Clinical Trials in Progress for MSCs in the Treatment of COVID-19 II-2 Lack of Antiviral Therapy Brings Spotlight on MSCs as Potential Option to Treat Severe Cases of COVID-19 II-3 Stem Cells Garner Significant Attention amid COVID-19 Crisis II-3 Growing R&D Investments & Rising Incidence of Chronic Diseases to Drive the Global Cell Harvesting Market over the Long-term II-3 US Dominates the Global Market, Asia-Pacific to Experience Lucrative Growth Rate II-4 Biopharmaceutical & Biotechnology Firms to Remain Key End-User II-4 Remarkable Progress in Stem Cell Research Unleashes Unlimited Avenues for Regenerative Medicine and Drug Development II-4 Drug Development II-5 Therapeutic Potential II-5

2. FOCUS ON SELECT PLAYERS II-6 Recent Market Activity II-7 Innovations and Advancements II-7

3. MARKET TRENDS & DRIVERS II-8 Development of Regenerative Medicine Accelerates Demand for Cell Harvesting II-8 The Use of Mesenchymal Stem Cells in Regenerative Medicine to Drive the Cell Harvesting Market II-8 Rise in Volume of Orthopedic Procedures Boosts Prospects for Stem Cell, Driving the Cell Harvesting II-9 Exhibit 1: Global Orthopedic Surgical Procedure Volume (2010- 2020) (in Million) II-11 Increasing Demand for Stem Cell Based Bone Grafts: Promising Growth Ahead for Cell Harvesting II-11 Spectacular Advances in Stem Cell R&D Open New Horizons for Regenerative Medicine II-12 Exhibit 2: Global Regenerative Medicines Market by Category (2019): Percentage Breakdown for Biomaterials, Stem Cell Therapies and Tissue Engineering II-13 Stem Cell Transplants Drive the Demand for Cell Harvesting II-13 Rise in Number of Hematopoietic Stem Cell Transplantation Procedures Propels Market Expansion II-15 Growing Incidence of Chronic Diseases to Boost the Demand for Cell Harvesting II-16 Exhibit 3: Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 II-17 Exhibit 4: Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 II-18 Exhibit 5: Number of New Cancer Cases and Deaths (in Million) by Region for 2018 II-19 Exhibit 6: Fatalities by Heart Conditions: Estimated Percentage Breakdown for Cardiovascular Disease, Ischemic Heart Disease, Stroke, and Others II-19 Exhibit 7: Rising Diabetes Prevalence Presents Opportunity for Cell Harvesting: Number of Adults (20-79) with Diabetes (in Millions) by Region for 2017 and 2045 II-20 Ageing Demographics to Drive Demand for Stem Cell Banking II-20 Global Aging Population Statistics - Opportunity Indicators II-21 Exhibit 8: Expanding Elderly Population Worldwide: Breakdown of Number of People Aged 65+ Years in Million by Geographic Region for the Years 2019 and 2030 II-21 Exhibit 9: Life Expectancy for Select Countries in Number of Years: 2019 II-22 High Cell Density as Major Bottleneck Leads to Innovative Cell Harvesting Methods II-22 Advanced Harvesting Systems to Overcome Centrifugation Issues II-23 Sophisticated Filters for Filtration Challenges II-23 Innovations in Closed Systems Boost Efficiency & Productivity of Cell Harvesting II-23 Enhanced Harvesting and Separation of Micro-Carrier Beads II-24

4. GLOBAL MARKET PERSPECTIVE II-25 Table 1: World Current & Future Analysis for Cell Harvesting by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-25

Table 2: World Historic Review for Cell Harvesting by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-26

Table 3: World 15-Year Perspective for Cell Harvesting by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2012, 2020 & 2027 II-27

Table 4: World Current & Future Analysis for Manual by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-28

Table 5: World Historic Review for Manual by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-29

Table 6: World 15-Year Perspective for Manual by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-30

Table 7: World Current & Future Analysis for Automated by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-31

Table 8: World Historic Review for Automated by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-32

Table 9: World 15-Year Perspective for Automated by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-33

Table 10: World Current & Future Analysis for Peripheral Blood by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-34

Table 11: World Historic Review for Peripheral Blood by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-35

Table 12: World 15-Year Perspective for Peripheral Blood by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-36

Table 13: World Current & Future Analysis for Bone Marrow by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-37

Table 14: World Historic Review for Bone Marrow by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-38

Table 15: World 15-Year Perspective for Bone Marrow by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-39

Table 16: World Current & Future Analysis for Umbilical Cord by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-40

Table 17: World Historic Review for Umbilical Cord by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-41

Table 18: World 15-Year Perspective for Umbilical Cord by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-42

Table 19: World Current & Future Analysis for Adipose Tissue by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-43

Table 20: World Historic Review for Adipose Tissue by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-44

Table 21: World 15-Year Perspective for Adipose Tissue by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-45

Table 22: World Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-46

Table 23: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-47

Table 24: World 15-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-48

Table 25: World Current & Future Analysis for Biotech & Biopharma Companies by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-49

Table 26: World Historic Review for Biotech & Biopharma Companies by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-50

Table 27: World 15-Year Perspective for Biotech & Biopharma Companies by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-51

Table 28: World Current & Future Analysis for Research Institutes by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-52

Table 29: World Historic Review for Research Institutes by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-53

Table 30: World 15-Year Perspective for Research Institutes by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-54

Table 31: World Current & Future Analysis for Other End-Uses by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-55

Table 32: World Historic Review for Other End-Uses by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-56

Table 33: World 15-Year Perspective for Other End-Uses by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-57

III. MARKET ANALYSIS III-1

GEOGRAPHIC MARKET ANALYSIS III-1

UNITED STATES III-1 Increasing Research on Stem Cells for Treating COVID-19 to drive the Cell Harvesting Market III-1 Rising Investments in Stem Cell-based Research Favors Cell Harvesting Market III-1 Exhibit 10: Stem Cell Research Funding in the US (in US$ Million) for the Years 2011 through 2017 III-2 A Strong Regenerative Medicine Market Drives Cell Harvesting Demand III-2 Arthritis III-3 Exhibit 11: Percentage of Population Diagnosed with Arthritis by Age Group III-3 Rapidly Ageing Population: A Major Driving Demand for Cell Harvesting Market III-4 Exhibit 12: North American Elderly Population by Age Group (1975-2050) III-4 Increasing Incidence of Chronic Diseases Drives Focus onto Cell Harvesting III-5 Exhibit 13: CVD in the US: Cardiovascular Disease* Prevalence in Adults by Gender & Age Group III-5 Rising Cancer Cases Spur Growth in Cell Harvesting Market III-5 Exhibit 14: Estimated Number of New Cancer Cases and Deaths in the US (2019) III-6 Exhibit 15: Estimated New Cases of Blood Cancers in the US (2020) - Lymphoma, Leukemia, Myeloma III-7 Exhibit 16: Estimated New Cases of Leukemia in the US: 2020 III-7 Market Analytics III-8 Table 34: USA Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-8

Table 35: USA Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-9

Table 36: USA 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-10

Table 37: USA Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-11

Table 38: USA Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-12

Table 39: USA 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-13

Table 40: USA Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-14

Table 41: USA Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-15

Table 42: USA 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-16

CANADA III-17 Market Overview III-17 Exhibit 17: Number of New Cancer Cases in Canada: 2019 III-17 Market Analytics III-18 Table 43: Canada Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-18

Table 44: Canada Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-19

Table 45: Canada 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-20

Table 46: Canada Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-21

Table 47: Canada Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-22

Table 48: Canada 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-23

Table 49: Canada Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-24

Table 50: Canada Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-25

Table 51: Canada 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-26

JAPAN III-27 Increasing Demand for Regenerative Medicine in Geriatric Healthcare and Cancer Care to Drive Demand for Cell Harvesting III-27 Exhibit 18: Japanese Population by Age Group (2015 & 2040): Percentage Share Breakdown of Population for 0-14, 15-64 and 65 & Above Age Groups III-27 Exhibit 19: Cancer Related Incidence and Deaths by Site in Japan: 2018 III-28 Market Analytics III-29 Table 52: Japan Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-29

Table 53: Japan Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-30

Table 54: Japan 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-31

Table 55: Japan Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-32

Table 56: Japan Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-33

Table 57: Japan 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-34

Table 58: Japan Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-35

Table 59: Japan Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-36

Table 60: Japan 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-37

CHINA III-38 Rising Incidence of Cancer Drives Cell Harvesting Market III-38 Exhibit 20: Number of New Cancer Cases Diagnosed (in Thousands) in China: 2018 III-38 Market Analytics III-39 Table 61: China Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-39

Table 62: China Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-40

Table 63: China 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-41

Table 64: China Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-42

Table 65: China Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-43

Table 66: China 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-44

Table 67: China Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-45

Table 68: China Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-46

Table 69: China 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-47

EUROPE III-48 Cancer in Europe: Key Statistics III-48 Exhibit 21: Cancer Incidence in Europe: Number of New Cancer Cases (in Thousands) by Site for 2018 III-48 Ageing Population to Drive Demand for Cell Harvesting Market III-49 Exhibit 22: European Population by Age Group (2016, 2030 & 2050): Percentage Share Breakdown by Age Group for 0-14, 15- 64, and 65 & Above III-49 Market Analytics III-50 Table 70: Europe Current & Future Analysis for Cell Harvesting by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 III-50

Table 71: Europe Historic Review for Cell Harvesting by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-51

Table 72: Europe 15-Year Perspective for Cell Harvesting by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2012, 2020 & 2027 III-52

Table 73: Europe Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-53

Table 74: Europe Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-54

Table 75: Europe 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-55

Table 76: Europe Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-56

Table 77: Europe Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-57

Table 78: Europe 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-58

Table 79: Europe Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-59

Table 80: Europe Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-60

Table 81: Europe 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-61

FRANCE III-62 Table 82: France Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-62

Table 83: France Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-63

Table 84: France 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-64

Table 85: France Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-65

Table 86: France Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-66

Table 87: France 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-67

Go here to read the rest:
Global Cell Harvesting Market to Reach US$381,4 Million by the Year 2027 - PRNewswire

Recommendation and review posted by Bethany Smith

Since losing her cousin to leukemia during childhood, Heather Lynn made it her mission to ensure others battling blood cancers get a second chance at life. Earlier this year, the St. Louis native fulfilled that life purpose: saving a stranger by donating her stem cells.

Five years ago, Lynn became the director of global special events for DKMS, the worlds largest bone marrow and blood stem cell donor center, and registered as a potential donor with the hope that someday she could give a blood cancer patient what her cousin didnt have a second chance at life and more time with the patients family.

Amid this years coronaviral pandemic, Lynn received the life-changing call from a colleague that she was a match for a 58-year-old man battling acute myeloid leukemia. I screamed with joy, Lynn recalls. I was a match for someone with blood cancer and was about to be the first employee at DKMS to donate and ultimately save someones life. After the call, Lynn realized she would be giving more than stem cells: I was giving something much bigger: hope.

Despite the uncertainty surrounding COVID-19, Lynn felt a strong sense of purpose to help this man and donated her stem cells to save his life. I have seen how much someones life can change with a blood cancer diagnosis, Lynn says. The fear, the pain, the loss it can be devastating. I have spent the past five years working to elevate the message about donating and how easy it is to sign up and give back it simply requires swabbing the inside of each cheek for 60 seconds.

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St. Louis Native Heather Lynn Discusses Motivation for Donating Stem Cells - Ladue News

Recommendation and review posted by Bethany Smith

Lurbinectedin is being studied in a number of diseases, but in lung cancer it has a more favorable side effect profile compared with topotecan, said Apar Kishor Ganti, MD, University of Nebraska Medical Center.

Lurbinectedin is being studied in a number of diseases, but in lung cancer it has a more favorable side effect profile compared with topotecan, said Apar Kishor Ganti, MD, professor of internal medicine, Division of Oncology & Hematology, University of Nebraska Medical Center.

Are there other tumor types where lurbinectedin seems to hold promise?

So, lurbinectedin is being studied in other diseases like breast cancer, mesothelioma, chronic lymphocytic leukemia, among others. But the difference in these other conditions compared to small cell [lung cancer] is there are other treatment options that are reasonably effective in these other cancers, unlike in small cell, so that's where it becomes much more important in in this particular setting.

One other reason why lurbinectedin may be effective is, like I told you earlier, there is a group of cells that seem to be shielded from chemotherapy. We call them cancer stem cells. And there are some lab data that suggests that lurbinectedin may inhibit cancer stem cells, as well. Again, this is all preliminary data. And we don't necessarily know if that occurs in humans or not, but those are some of the hypothesized mechanisms of action.

What other advantages are there of lurbinectedin over topotecan?

One of the other advantages of lurbinectedin over topotecan is that topotecan has to be given 5 days in a row, whereas lurbinectedin is given just once every 3 weeks. And the side effect profile of lurbinectedin seems to be favorable. The main side effect of lurbinectedin is bone marrow suppression, anemia, leukopenia, neutropenia, [and] thrombocytopenia, but they seem to occur in about 5% to 10% of patients. And so, that's another possible advantage of lurbinectedin over for some of the other drugs that are available.

As far as small cell lung cancer itself is concerned, even though there is a lot of research going on in small cell, multiple different drugs have been triedtargeted therapies, immunotherapythere is some evidence to suggest that immunotherapy helps with chemotherapy in the frontline setting. But immunotherapy by itself in patients who have failed chemotherapy does not seem to be much more effective. People have tried targeted therapies, again, not one of them has shown to have any meaningful benefit for these patients. So that has been very disappointing.

There have been multiple drugs that have been studied. Unfortunately, none of them have had a significant benefit so far. So, it's a fairly difficult to treat disease. And like I mentioned earlier, even though it seems to respond quite well to initial chemotherapymost patients relapse and very few are cured even if they present with very early stage disease. And that's why it's a very challenging disease to treat.

Read more:
Dr Apar Kishor Ganti Outlines the Effectiveness of Lurbinectedin and Benefits Over Competition - AJMC.com Managed Markets Network

Recommendation and review posted by Bethany Smith

Summary

MSK researchers have uncoveredan important finding about the relationship between the microbiota and the immune system, showing for the first time that the concentration of different types of immune cells in the blood changes in relation to the presence of different bacterial strains in the gut.

In recent years, the microbiota the community of bacteria and other microorganisms that live on and in the human body has captured the attention of scientists and the public, in part because its become easier to study. It has been linked to many aspects of human health.

A multidisciplinary team from Memorial Sloan Kettering has shown for the first time that the gut microbiota directly shapes the makeup of the human immune system. Specifically, their research demonstrated that the concentration of different types of immune cells in the blood changed in relation to the presence of different bacterial strains in the gut. The results of their study, which used more than ten years of data collected from more than 2,000 patients, is being published November 25, 2020, in Nature.

The scientific community had already accepted the idea that the gut microbiota was important for the health of the human immune system, but the data they used to make that assumption came from animal studies, says Sloan Kettering Institute systems biologist Joao Xavier, co-senior author of the paper together with his former postdoc Jonas Schluter, who is now an assistant professor at NYU Langone Health. At MSK, we have a remarkable opportunity to follow how the composition of the microbiota changes in people being treated for blood cancers, Dr. Xavier adds.

(From left) Researchers Emily Fontana, Luigi Amoretti, Joao Xavier, Roberta Wright, and Jonas Schluter in the lab.

The data that were used in the study came from people receiving allogeneic stem cell and bone marrow transplants (BMTs). After strong chemotherapy or radiation therapy is used to destroy cancerous blood cells, the patients blood-forming system is replaced with stem cells from a donor. For the first few weeks until the donors blood cells including the white blood cells that make up the immune system have established themselves, the patients are extremely vulnerable to infections. To protect them during this time, patients are given antibiotics.

But many of these antibiotics have the unwanted side effect of destroying healthy microbiota that live in the gut, allowing dangerous strains to take over. When the patients immune system has reconstituted, the antibiotics are discontinued, and the gut microbiota slowly starts to grow back.

The parallel recoveries of the immune system and the microbiota, both of which are damaged and then restored, gives us a unique opportunity to analyze the associations between these two systems, Dr. Schluter says.

For more than ten years, members of MSKs BMT service have regularly collected and analyzed blood and fecal samples from patients throughout the BMT process. The bacterial DNA were processed by the staff at MSKs Lucille Castori Center for Microbes, Inflammation, and Cancer, which played a key role in creating the massive microbiota dataset. Our study shows that we can learn a lot from stool biological samples that literally would be flushed down the toilet, Dr. Xavier notes. The result of collecting them is that we have a unique dataset with thousands of datapoints that we can use to ask questions about the dynamics of this relationship.

This wider effort has been led by Marcel van den Brink, Head of the Division of Hematologic Malignancies, and a team of infectious disease specialists, BMT doctors, and scientists. For a fair number of patients, we collected daily samples so we could really see what was happening day to day, Dr. van den Brink says. The changes in the microbiota are rapid and dramatic, and there is almost no other setting in which you would be able to see them.

Our study shows that we can learn a lot from stool biological samples that literally would be flushed down the toilet.

Previous research using samples collected from this work has looked at how the gut microbiota affects patients health during the BMT process. A study published in February 2020 reported that having a greater diversity of species in the intestinal microbiota is associated with a lower risk of death after a BMT. It also found that having a lower diversity of microbiota before transplant resulted in a higher incidence of graft-versus-host disease, a potentially fatal complication in which the donor immune cells attack healthy tissue.

The databank that the MSK team created contains details about the types of microbes that live in the patients guts at various times. The computational team, including Drs. Schluter and Xavier, then used machine learning algorithms to mine electronic health records for meaningful data. The data from the health records included the types of immune cells present in the blood, information about the medications that patients were given, and the side effects patients experienced. This research could eventually suggest ways to make BMTs safer by more closely regulating the microbiota, Dr. van den Brink says.

Analyzing this much data was a huge undertaking. Dr. Schluter, who at the time was a postdoctoral fellow in Dr. Xaviers lab, developed new statistical techniques for this. Because experiments with people are often impossible, we are left with what we can observe, Dr. Schluter says. But because we have so many data collected over a period of time when the immune system of patients as well as the microbiome shift dramatically, we can start to see patterns. This gives us a good start toward understanding the forces that the microbiota exerts on the rebuilding of the immune system.

This research could eventually suggest ways to make BMTs safer by more closely regulating the microbiota.

The purpose of this study was not to say whether certain kinds of microbes are good or bad for the immune system, Dr. Xavier explains, adding that this will be a focus of future research. Its a complicated relationship. The subtypes of immune cells we would want to increase or decrease vary from day to day, depending on what else is going on in the body. Whats important is that now we have a way to study this complex ecosystem.

The researchers say they also plan to apply their data to studying the immune system in patients receiving other cancer treatments.

Originally posted here:
MSK Study Is the First to Link Microbiota to Dynamics of the Human Immune System - On Cancer - Memorial Sloan Kettering

Recommendation and review posted by Bethany Smith

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Global Hematopoietic Stem Cell Transplantation (HSCT) Market Trends, Opportunities, Drivers, Challenges and Forecast to 2026 - The Market Feed

Recommendation and review posted by Bethany Smith

Severe infections like malaria cause short and long-term damage to precursor blood cells in mice, but some damage could be reversed, find researchers.

A team led by researchers from Imperial College London and The Francis Crick Institute have discovered that severe infections caused by malaria disrupt the processes that form blood cells in mice. This potentially causes long-term damage that could mean people who have recovered from severe infections are vulnerable to new infections or to developing blood cancers.

The team also discovered that the damage could be reduced or partially reversed in mice with a hormone treatment that regulates bone calcium coupled with an antioxidant. The research could lead to new ways of preventing long-term damage from severe infections including malaria, TB and COVID-19.

The research is published today in Nature Cell Biology.

First author Dr Myriam Haltalli, who completed the work while at the Department of Life Sciences at Imperial, said: We discovered that malaria infection reprograms the process of blood cell production in mice and significantly affects the function of precursor blood cells. These changes could cause long-term alterations, but we also found a way to significantly reduce the amount of damage and potentially rescue the healthy production of blood cells.

Blood is made up of several different cell types, that all originate as haematopoietic stem cells (HSCs) in the bone marrow. During severe infection, the production of all blood cells ramps up to help the body fight the infection, depleting the HSCs.

Now, the team has shown how infections also damage the bone marrow environment that is crucial for healthy HSC production and function. They discovered this using advanced microscopy technologies at Imperial and the Crick, RNA analyses led by the Gottgens group at Cambridge University, and mathematical modelling led by Professor Ken Duffy at Maynooth University.

The mice developed malaria naturally, following bites from mosquitoes carrying Plasmodium parasites, provided by Dr Andrew Blagborough at Cambridge University. The researchers subsequently observed the changes in the bone marrow environment and the effect on HSC function.

Within days of infection, blood vessels became leaky and there was a dramatic loss in bone-forming cells called osteoblasts. These changes appear strongly linked to the decline in the pool of HSCs during infection.

Lead author Professor Cristina Lo Celso, from the Department of Life Sciences at Imperial, said: We were surprised at the speed of the changes, which was completely unexpected. We may think of bone as an impenetrable fortress, but the bone marrow environment is incredibly dynamic and susceptible to damage.

Reducing the pool of HSCs can have several consequences. In the short-term, it appears to particularly affect the production of neutrophils white blood cells that form an essential part of the immune system. This can leave patients vulnerable to further infections, with potentially long-term consequences for the functioning of the immune system.

In the long term, the pool of HSCs may remain below normal levels, which can increase the chances of the patient developing blood cancers like leukaemia.

By injecting fluorescent molecules (magenta) that would normally remain in circulation and taking a series of images over time, intravital microscopy revealed that infected mice had very leaky vessels with the contents of the bone marrow blood vessels, lined by endothelial cells (green), escaping into the surrounding tissue. The red boxes highlight the areas compared in the analysis and the white lines mark the bone.

After determining in detail how severe infection affects the bone marrow environment and HSC function, the team tested a way to prevent the damage. Before infecting the mice, they treated them with a hormone that regulates bone calcium and an antioxidant to counter cellular oxidative stress, and then again after infection.

This process led to a tenfold increase in HSC function following infection compared to mice that received no treatment (around 20-40 per cent function compared to two percent function, respectively). Although this is not a complete recovery, the vast increase in function is a positive sign.

The team note that the requirement to start the hormone treatment before infection, combined with its expense and need to be refrigerated, make it unviable as a solution, especially in many parts of the world where severe infections like malaria and TB are prevalent.

However, they hope that proof that the impact of severe infection on HSC function can be significantly lessened will lead to the development of new treatments that can be widely administered.

Professor Lo Celso said: The long-term impacts of COVID-19 infection are just starting to be known. The impact on HSC function appears similar across multiple severe infections, suggesting our work on malaria could shed light on the possible long-term consequences of COVID-19, and how we might mitigate them.

Dr Haltalli concluded: Protecting HSC function while still developing strong immune responses is key for healthy ageing.

-

Manipulating niche composition limits damage to haematopoietic stem cells during Plasmodium infection by Myriam L.R. Haltalli et al. is published in Nature Cell Biology.

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Severe infections wreak havoc on mouse blood cell production | Imperial News - Imperial College London

Recommendation and review posted by Bethany Smith

MELVILLE, N.Y., Nov. 20, 2020 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, announced today that its amended joint plan of reorganization has become effective and it has emerged from Chapter 11 reorganization. Pursuant to the confirmed plan of reorganization, the Company has received $3,848,000 in financing. The confirmed plan of reorganization also provides for additional funding, subject to certain conditions, of $3,500,000 less the sum of the debtor-in-possession financing provided to the Company during the reorganization (approximately $1,227,000) and the costs incurred by the debtor-in-possession lender.

In connection with the reorganization, Lance Alstodt has been appointed the Companys President, Chief Executive Officer and Chairman of the Board. Mr. Alstodt said, This process has been a long and challenging journey for the Company. Im inspired by the great resolve and execution from our employees, professionals and investors. We are very pleased that all requirements have been met for us to emerge. Allowed creditor claims have been fully satisfied and, as importantly, our equity holders have retained their shares in this exciting new opportunity. We were able to preserve all of our intellectual property assets and look forward to initiating our Phase 2 clinical trial.

Based upon the Companys emergence from Chapter 11 reorganization, FINRA has removed the Q at the end of its trading symbol. Shareholders do not need to exchange their shares for new shares.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs.

Metabolic Program (ThermoStem): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Emerges from Chapter 11 Reorganization Other OTC:BRTX - GlobeNewswire

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Databridgemarketresearch.com Present Global Stem Cell Banking Market Industry Trends and Forecast to 2027 new report to its research database. The report spread No of pages: 350 No of Figures: 60 No of Tables: 220 in it. This Global Stem Cell Banking Market report takes into consideration diverse segments of the market analysis that todays business ask for. The Global Stem Cell Banking Market report provides estimations of CAGR values, market drivers and market restraints about the industry which are helpful for the businesses in deciding upon numerous strategies. The base year for calculation in the report is taken as 2017 whereas the historic year is 2016 which will tell you how the Global Stem Cell Banking Market is going to perform in the forecast years by informing you what the market definition, classifications, applications, and engagements are. The report helps you to be there on the right track by making you focus on the data and realities of the industry.

The research studies of this Global Stem Cell Banking Market report helps to evaluate several important parameters that can be mentioned as investment in a rising market, success of a new product, and expansion of market share. Market estimations along with the statistical nuances included in this market report give an insightful view of the market. The market analysis serves present as well as future aspects of the market primarily depending upon factors on which the companies contribute in the market growth, crucial trends and segmentation analysis. This Global Stem Cell Banking Market research report also gives widespread study about different market segments and regions.

Global stem cell banking market is set to witness a substantial CAGR of 11.03% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. The increased market growth can be identified by the increasing procedures of hematopoietic stem cell transplantation (HSCT), emerging technologies for stem cell processing, storage and preservation. Increasing birth rates, awareness of stem cell therapies and higher treatment done viva stem cell technology.

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Competitive Analysis:

Global stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of inflammatory disease drug delivery market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Competitors:

Few of the major competitors currently working in global inflammatory disease drug delivery market are: NSPERITE N.V, Caladrius, ViaCord, CBR Systems, Inc, SMART CELLS PLUS, LifeCell International, Global Cord Blood Corporation, Cryo-Cell International, Inc., StemCyte India Therapeutics Pvt. Ltd, Cordvida, ViaCord, Cryoviva India, Vita34 AG, CryoHoldco, PromoCell GmbH, Celgene Corporation, BIOTIME, Inc., BrainStorm Cell Therapeutics and others

Market Definition:Global Stem Cell Banking Market

Stem cells are cells which have self-renewing abilities and segregation into numerous cell lineages. Stem cells are found in all human beings from an early stage to the end stage. The stem cell banking process includes the storage of stem cells from different sources and they are being used for research and clinical purposes. The goal of stem cell banking is that if any persons tissue is badly damaged the stem cell therapy is the cure for that. Skin transplants, brain cell transplantations are some of the treatments which are cured by stem cell technique.

Cord Stem Cell Banking MarketDevelopment and Acquisitions in 2019

In September 2019, a notable acquisition was witnessed between CBR and Natera. This merger will develop the new chances of growth in the cord stem blood banking by empowering the Nateras Evercord branch for storing and preserving cord blood. The advancement will focus upon research and development of the therapeutic outcomes, biogenetics experiment, and their commercialization among the global pharma and health sector.

Cord Stem Cell Banking MarketScope

Cord Stem Cell Banking Marketis segmented on the basis of countries into U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

All country based analysis of the cord stem cell banking marketis further analyzed based on maximum granularity into further segmentation. On the basis of storage type, the market is segmented into private banking, public banking. On the basis of product type, the market is bifurcated into cord blood, cord blood & cord tissue. On the basis of services type, the market is segmented into collection & transportation, processing, analysis, storage. On the basis of source, market is bifurcated into umbilical cord blood, bone marrow, peripheral blood stem, menstrual blood. On the basis of indication, the market is fragmented into cerebral palsy, thalassemia, leukemia, diabetes, autism.

Cord stem cell trading is nothing but the banking of the vinculum plasma cell enclosed in the placenta and umbilical muscle of an infant. This ligament plasma comprises the stem blocks which can be employed in the forthcoming time to tackle illnesses such as autoimmune diseases, leukemia, inherited metabolic disorders, and thalassemia and many others.

Market Drivers

Increasing rate of diseases such as cancers, skin diseases and othersPublic awareness associated to the therapeutic prospective of stem cellsGrowing number of hematopoietic stem cell transplantations (HSCTs)Increasing birth rate worldwide

Market Restraint

High operating cost for the therapy is one reason which hinders the marketIntense competition among the stem cell companiesSometimes the changes are made from government such as legal regulations

Key Pointers Covered in the Cord Stem CellBanking MarketIndustry Trends and Forecast to 2026

Market SizeMarket New Sales VolumesMarket Replacement Sales VolumesMarket Installed BaseMarket By BrandsMarket Procedure VolumesMarket Product Price AnalysisMarket Healthcare OutcomesMarket Cost of Care AnalysisMarket Regulatory Framework and ChangesMarket Prices and Reimbursement AnalysisMarket Shares in Different RegionsRecent Developments for Market CompetitorsMarket Upcoming ApplicationsMarket Innovators Study

Key Developments in the Market:

In August, 2019, Bayer bought BlueRock for USD 600 million to become the leader in stem cell therapies. Bayer is paying USD 600 million for getting full control of cell therapy developer BlueRock Therapeutics, promising new medical area to revive its drug development pipeline and evolving engineered cell therapies in the fields of immunology, cardiology and neurology, using a registered induced pluripotent stem cell (iPSC) platform.In August 2018, LifeCell acquired Fetomed Laboratories, a provider of clinical diagnostics services. The acquisition is for enhancement in mother & baby diagnostic services that strongly complements stem cell banking business. This acquisition was funded by the internal accruals which is aimed to be the Indias largest mother & baby preventive healthcare organization.

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Research objectives

To perceive the most influencing pivoting and hindering forces in Cord Stem Cell Banking Market and its footprint in the international market.Learn about the market policies that are being endorsed by ruling respective organizations.To gain a perceptive survey of the market and have an extensive interpretation of the Cord Stem Cell Banking Market and its materialistic landscape.To understand the structure of Cord Stem Cell Banking Market by identifying its various sub segments.Focuses on the key global Cord Stem Cell Banking Market players, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).To project the consumption of Cord Stem Cell Banking Market submarkets, with respect to key regions (along with their respective key countries).To strategically profile the key players and comprehensively analyze their growth strategiesTo analyze the Cord Stem Cell Banking Market with respect to individual growth trends, future prospects, and their contribution to the total market.

Customization of the Report:

All segmentation provided above in this report is represented at country levelAll products covered in the market, product volume and average selling prices will be included as customizable options which may incur no or minimal additional cost (depends on customization)

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An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

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Global Stem Cell Banking Market to Get Expansion admist COVID 19, Scope With Advanced Technologies Top Key Players and Forecast 2020-2027 - The...

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This past February, Easton Lindeman then age 2, son ofCorey and Samantha was diagnosed with high-risk neuroblastoma, a type of cancer that affects his adrenal glands, spinal column, and bone marrow.Neuroblastoma is a cancer that develops from immature nerve cells found in several areas of the body. Treatment for Easton started right away. This was followed by hospitalizations for both treatments and illnesses. So far he has gone through 3 rounds of chemotherapy and IMIBG therapy at the University of Wisconsin. IMIBG Therapy is a form of radiation treatment. This was followed by more chemotherapy and surgery to try to remove as much of the tumor as possible.

The last time we talked with Easton's mom he was finishing up with his 6th round of high dose chemotherapy and stem cell transplant.

He has had countless blood and platelet transfusions, ECHO's, hearing tests, MRI's, CT scans, X-Rays, bone marrow biopsies, surgeries and sedations. PICC line placements and Port Access placement. Earlier this month, Easton took a trip to the Mayo Clinic for more treatment.

On Saturday, Easton had a very special treat. The pink firetruck, overseen by Dennis and Diana Brammow made a stop to see Easton.

Easton is 3 years old now and his mom said that he loves to pass out candy to the doctors in and nurse friends while he's in the hospital. Like most 3 year old, there isn't a lot that slows him down. At 3 he's a family man already. Tagging around after his big brother Hunter and cousin Eleanor and he's always thrilled to visit his Grandpa and Grandma Dough and Dawn Yenser.

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Easton Lindeman gets a visit from the Pink Fire Truck - News - vintontoday.com

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NEW YORK, Nov. 23, 2020 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today highlighted its intellectual property portfolio for apamistamab, a CD45 targeting antibody, and the Antibody Radiation Conjugate (ARC) comprised of apamistamab and the radioisotope iodine-131 used in the Company's lead Phase 3 candidate, Iomab-B, and its Iomab-ACT programs. Actinium owns issued or pending patents within the United States and globally covering composition of matter, formulation, methods of use, and methods of administration with potential coverage for 19 years or longer. Importantly, Actinium owns an issued patent in the US covering composition of matter, for which the Company expects validity until 2037.In addition, the Company owns a second issued US patent that further covers composition of matter, methods of use, and methods of administration for Iomab-B. The company has also received a notice of allowance in Europe for this second patent and expects it to be in force until 2036.

Iomab-B is currently being investigated in the ongoing pivotal Phase 3 SIERRA trial, which is over 75% enrolled, for targeted conditioning prior to potentially curative bone marrow transplant (BMT) for patients with relapsed or refractory Acute Myeloid Leukemia ("R/R AML"). In addition, Actinium is utilizing apamistamab with lower doses of iodine-131, known as Iomab-ACT, for targeted conditioning prior to gene therapy and adoptive cell therapy ("ACT"), namely CAR-T, including in its recently announced collaboration with Memorial Sloan Kettering Cancer Center that is supported by NIH STTR Fast Track grant funding.

"The continued protection of our lead asset Iomab-B, our Iomab-ACT program and apamistamab by a strong patent position is an important component of our development efforts, particularly as we approach the conclusion of our pivotal Phase 3 SIERRA trial for BMT conditioning in R/R AML The growth of BMT, ACT and Gene Therapy has highlighted the importance of conditioning and the need to move beyond non-targeted chemotherapy to increase the number of patients that could benefit from these potentially curative therapies.CD45 is an ideal target for conditioning applications given its unique expression on blood cancer cells and blood forming stem and immune cells and with no expression outside the hematopoietic or blood system," said Dr. Dale Ludwig, Actinium's Chief Scientific Officer. "Apamistamab is well characterized and its use in conditioning is supported by extensive clinical data across multiple clinical trials and indications. Our robust data shows that apamistamab has a favorable biodistribution profile that, together with our ARC technology, has significant advantages over other approaches such antibody drug conjugates that require payload internalization, making them impractical for targeting CD45.Further, our ARC approach allows us to use varying intensities of targeted radiation to achieve our desired conditioning outcome.With these important patents in place, and continued expansion of our patent portfolio in the US, EU and other select countries, we look forward to continuing to build out our targeted conditioning strategic business unit."

About Iomab-B

Iomab-B is Actinium's lead product candidate that is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, a pan-leukocytic antigen widely expressed on white blood cells with the monoclonal antibody, apamistamab (formerly BC8), labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects of radiation on most healthy tissues while effectively killing the patient's cancer and marrow cells. In a Phase 1/2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, Iomab-B produced enabled 100% of patients to proceed to transplant with all patients achieving transplant engraftment by day 28. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 300 patients in a number of blood cancer indications, including acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, Hodgkin's disease, Non-Hodgkin lymphomas and multiple myeloma. Actinium obtained the worldwide, exclusive rights to apamistamab (BC8) and Iomab-B from the Fred Hutchinson Cancer Research Center. Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation.Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities.With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently.Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning.The SIERRA trial is over seventy-five percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings.More information on this Phase 3 clinical trial can be found at sierratrial.com. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1 study with a CD19 CAR T-cell Therapy and Phase 1/2 anti-HIV stem cell gene therapy with UC Davis. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform.This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes.Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Clayton Robertson Actinium Pharmaceuticals, Inc. [emailprotected]

Hans VitzthumLifeSci Advisors, LLC[emailprotected](617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com

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Actinium Highlights Foundational Patents Covering the Composition of Apamistamab Antibody and Iomab-B Antibody Radiation Conjugate for Targeted...

Recommendation and review posted by Bethany Smith

The arrival every October of a survey from the long-term follow-up care unit (LTFU) at the Fred Hutchinson Cancer Care Alliance in Seattle sets in motion a routine. I walk it back from the mailbox, putting it on the kitchen table where it sits unopened for four or five days. Then its moved to the dining room table where it sits for another few days. Then its taken downstairs, still unopened, where its put in a stack of other stuff for a week-plus. The routine continues when a follow-up to the follow-up comes in the mail.

Paraphrasing, the second LTFU letter reads: cmon, man. Take 15 minutes, fill it out, mail it back. Which I do. Could this be avoidance behavior, as per psychologists? Quoting Bill Murray, the casual philosopher, (Stripes, 1981), thats a fact, Jack!

Ive been in remission for 17 years after a stem cell transplant from the Hutch a day after my 51st birthday. The LTFU began following patients in 1990, citing improvement in the mortality of transplantation.

There are about 6,800 people enrolled currently in LTFU. In 2019, 4,764 former patients received the survey and about half of them returned it, said Molly McElroy, the communications director for the Hutch. She added how theres one transplant patient ambulatory and upright from 1971; 100-plus from before 1980.

I was diagnosed in 1998 with a friendly leukemia, but during an annual checkup a few years later, the diagnosis changed, and: the prognosis isnt as promising.

Conversations like that are ineradicable, along with related reminiscences of hallway walks with chemo poles and technicians who warn, youre going to feel a little prick. This is before they draw blood or remove bone marrow. (The first time I heard it, I thought it funny; after the umpteenth, not so much.) Treatment of mantle cell lymphoma required difficult, four-cocktail, hospitalized chemotherapy at St. Marys in Grand Junction. Soon thereafter, we left for the Hutch and transplant.

Hokey and contrived as it sounds, remission from cancer is indeed about some of the people you meet along the way.

Lee Bradley fruit farmer, wine maker, country marketer, located in the northern suburbs of Paonia calmed the hothouse of my mind after diagnosis. We talked about what was coming, sharing common stories about doctors, the Hutch, the determination of it all. Last month, Bradley celebrated 20 years of good health. (His familys market, which sits alongside the north fork of the Gunnison river, is a good place to grab an easy chair and enjoy a summers day under the cottonwoods.)

Early on at the Hutch, a physician who looked like she had just left a Grateful Dead concert with her long hair, granny-glasses and leather-fringed jacket, calmly explained options. Including a clinical trial for which she had written the protocol. As a result, Im still getting mail from the LTFU. Dr. Leona Holmberg, MD, PhD., continues to get it done as a physician, researcher and oncology professor at the U. of Washington.

We also met a nattily dressed doctor with great humor. He was placing into my chest a dual Hickman catheter which would expeditiously transport stem cells, chemo, nutrition, blood products and whatever else. (A terrific invention, but not a good look on a beach. It flops around a lot.) He flirted good-naturedly with Susan, who was maybe 10 inches taller. Are you the same guy whose name is on this? I cracked wise about him getting a commission; smiling, he replied that hed done all right. Dr. Robert O. Hickman, who was a founding member of the Hutch bone marrow transplant team, died last year at 92, his invention still widely used.

After transplant, my blood counts were stubbornly stuck at zero. The body language of the doctor-posse when they rolled through my room in the mornings wasnt encouraging. Then I caught an infection and spent 10 days in isolation. My nurse recognized despair. On a rainy Saturday afternoon, she sat on the bed and without saying a word, pulled me close. For less than a minute. She likely broke a slew of HR rules, but Corinne Vaniers humanity was the tonic. The next day, my counts started to move north. Ten years after the transplant, we had lunch in Seattle and continue to be in touch.

Julia Vega, the daughter of longtime friends and a newspaper publisher pal, was a year younger than our son. She had a pelvic cancer and enjoyed some initial remission. Her dad and I shared stories about the Hutch, the Pete Gross House (apartments) and Dr. Holmberg, also her physician.

Vega was a theater student at Arizona State U. when diagnosed and instead of dropping out and taking it easy, she forged through doing the chemo, going to classes, participating in productions, graduating. I enjoyed her company as a middle schooler in North Carolina, and again at a newspaper convention where she accompanied her father, fragile thin and wearing a wig. We have a photo together from that meeting. Whenever I hear one of her favorite songs, Bob Dylans Shooting Star, I recall her grace.

A story about Chris Mesaric was framed and placed along the walls of a Hutch examination clinic. My brother and I had flown to Seattle to nail down the second opinion and I happened on it while he was wrapping up a conversation with yet another oncologist. Mesaric had grown up here, MHS Class of 1987, and had worked as an airframe mechanic for Boeing in Seattle before returning to Montrose. The docs had given him little chance in 1992 of surviving aplastic anemia, a rare bone marrow disease. He was also told that there would be no children, either. Yet he and Robin are the parents of two lovely daughters.

Mesarics brave fight went on for 11 years, receiving a Hutch transplant in 2001. He was inspirational, although we never met. We left the Hutch after five months, arriving home the day before Thanksgiving, 2003; Mesaric, 35, passed three weeks later. I know his folks, Frank and Linda Mesaric, and whenever I see Frank around town, his big, welcoming voice is at once a comfort and a knowing.

There were good times, of course, during the procedures, before the transplant. Routines that got us away from the grimness. After morning bloodwork, we had a daily 16-block hike to the Barnes & Noble bookstore in downtown Seattle. One day, former Sen. Gary Hart was spotted in the shelves, browsing, and we chatted. There were plenty of windows to look into, galleries of all sorts, and all the bits and bobs and fish tossing from the Pike Place Market. Even a free monorail to ride. We enjoyed a couple of nights out with live music at Dimitrious Jazz Alley. William visited from Alaska and the three of us caught a Mariners/White Sox game at Safeco Field. Ryanns down-the-elevator, in-the-building interim school provided friendships and field trips. There was retail therapy. The downtown flagship Nordstroms store provided almost 400,000 square feet of distraction and attraction. (After we returned to Montrose, they had to lay off the lobbys pianist. Jes kidding.)

Theres a picture of the four of us on the refrigerator from Day One. Bald and bloated, Im unrecognizable. After Susan declared we can do this, upon turning that apartments front door key, thats when the recovery, like the new bone marrow, began to graft itself.

The piece of mail in October confirms it. Im thankful for those follow-up people who still have my address.

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Detroit Lions senior VP of business development Kelly Kozole works with her daughter, Morgan, who has a rare neurological disorder called beta-propeller protein-associated neurodegeneration, or BPAN.Michael Rothstein

TROY, Mich. -- Wearing a white T-shirt with a massive star in sparkling shades of pink, yellow and seafoam green on the front, Morgan Kozole sits in front of a fold-up chalkboard in the living room of her family's Detroit-area home and starts to draw.

Using pink and yellow chalk, she sketches Mickey and Minnie Mouse. The Disney characters are dominant fixtures in the 5-year-old's life and therefore become a soundtrack for the Kozole family: Morgan constantly saying "Mickey," with her long, blond ponytail bouncing to whatever song happens to be playing on the Mickey Mouse Club.

"These are the two Mickeys," Morgan says, pointing to the chalkboard. Her mother, Detroit Lions senior vice president of business development Kelly Kozole, explains that this is her way of communicating that she would like a visitor to draw Mickey too. If it's close, Morgan accepts it. Another Mickey to fawn over.

For Morgan's birthday earlier this year, the family went to Disney World. On this trip, the Kozoles saw what they had longed for: the potential of progress.

"She knew where we were. She knew Mickey Mouse," Kelly said. "Before, she wouldn't go to the characters, and now she's jumping up and down, hugging. She really, along those lines, is also really into birthdays.

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"The 'Happy Birthday' song. Before that, she was just kind of looking. Sometimes it was too much for her with everyone singing -- sometimes loud noises are too much. This year, we had to sing 'Happy Birthday' to her three times."

Birthdays, for children, are happy occasions -- reasons for grand celebrations of progress toward adulthood. For the rest of Morgan's family it is more complicated.

Morgan has a rare neurological disease called beta-propeller protein-associated neurodegeneration, known as BPAN. It's a disorder, more prevalent in girls than boys, that causes delayed development and seizures, communication issues and, sometimes, motor dysfunction. It's unclear exactly how many people are living with BPAN worldwide due to its rarity, although Dr. Sami Barmada, a scientist at the University of Michigan studying BPAN, estimates roughly 500 to 600 people.

It's rare enough that Dr. Henry Paulson, the director of the Michigan Alzheimer's Disease Center, said there are experts in neurodegeneration who are unfamiliar with BPAN. While Kelly is trying to advocate for her daughter and others with BPAN through fundraising for research, science moves only so fast.

The Kozoles understand that. So birthdays for the family aren't always happy. They are a reminder of what could come.

"That ticking time clock," Kelly said. "Every birthday isn't exciting for me for her. Because it's one year closer to when this bomb is going to go off."

BPAN's rarity makes the reality heartbreakingly simple: There are very few effective treatments, little research and no cure. As Morgan learns how to organize her Peppa Pig characters and learns new words on her iPad -- her future looms.

At some unpredictable point in Morgan's teen and adult years -- the average is around age 25, according to Barmada -- development will just stop. Progress will decline and, in some cases, disappear. Those afflicted with BPAN begin suffering from progressive dystonia parkinsonism -- making it difficult to walk, talk or stand.

"Any day," Kelly said, "it could be like, 'Oh, your daughter's gone.'"

WHEN MORGAN WAS born on Jan. 12, 2015, she was, largely, a healthy baby. She was a little jaundiced but nothing worrisome.

When she would go to the doctor's office for shots, Morgan didn't cry. It was a little abnormal, but when you're a parent of a young child, no crying is viewed as a minor miracle. Kelly and her husband, Kevin, took this as a sign of a tough kid. Nurses even said how great it was.

Looking back, it was a warning sign that something was wrong. BPAN causes a high pain tolerance. Before long, more concerns popped up. Morgan wasn't crawling at nine months, wasn't walking at a year. Expected milestones passed without Morgan reaching them. Kevin and Kelly put her in therapy in late 2016 to work up to these childhood progressive traits and began researching potential causes. They wouldn't find an answer for more than two years.

"She was diagnosed with cerebral palsy at first. One doctor diagnosed her with that, and then another, our neurologist, said she doesn't have that," Kelly said. "Then there was speculation but not a full diagnosis she had autism, so we did all the tests for that.

"So through this kind of journey of trying to find out what was wrong, it was exciting that she didn't have something that you were going to this test for, but you still had so many more questions as you were eliminating all these potential diseases that she could have."

Befuddled, they began genetic testing and in November 2018 received a letter about a mutation on Morgan's WDR45 gene. Kelly Googled it, stumbled upon BPAN and freaked out, calling their neurologist. The neurologist told Kelly not to worry -- BPAN was very rare, and Morgan didn't have it.

Doctors diagnosed her with epilepsy because of seizures. Morgan took Keppra, which helped accelerate her vocabulary to about 50 words, typical for a 1-year-old, when she was 3. Then doctors said no, it wasn't epilepsy either.

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Another meeting with another neurologist led to a different diagnosis. Three days after she and Kevin returned to Michigan from Super Bowl LIII in February 2019, they received a call. Doctors figured out what was wrong.

It was BPAN.

"In my mind, it's worse than cancer," Kelly said. "How is this even possible? That this can even be so painful for kids later on in life. You try so hard to gain all these abilities, and then early adolescence or early adulthood, it's just [gone] one day, and I've seen a lot of these stories.

"There's a BPAN Facebook website, and that's where the doctors sent us. There's no cure. There's no therapy. 'Go to this website.' That's what I was told."

FOR MONTHS KELLY cried, angry and heartbroken. The Kozoles initially told their families and no one else.

In May 2019, Kelly went to her first Neurodegeneration with Brain Iron Accumulation (NBIA) conference. She met other parents, heard their stories and began the new normal.

She used her skills -- organization, fundraising and business -- to brainstorm ways to help. Hardly anyone had researched BPAN. Without it, there would be no chance for a cure -- not in Morgan's lifetime, which could reach her 40s, and not in the lifetime of those who might come after.

She shared what was happening with her boss, Detroit Lions president Rod Wood, and his wife, Susan, using a website link to explain BPAN. Wood knew something was wrong because of texts and emails saying they had to take Morgan to this specialist or that appointment.

"As that was confirmed and became her reality, she is now able to talk about it, in a way," Wood said. "Because she's full bore on trying to help generate awareness and financial resources to find a cure for it.

"She went from the unknown to the very tragic known to, 'OK, what are we going to do about it?'"

Kelly consulted her aunts, both of whom worked in medicine. Linda Narhi worked in biotechnology for Amgen for more than 30 years; Dr. Diane Narhi was the first female chief of staff at Simi Valley (California) Hospital. From talking with another group of fundraising BPAN parents -- BPAN Warriors -- Kelly found a guide.

If her aunts had not been resources, she might have joined BPAN Warriors. But Kelly admittedly needs to be in control, and this was her daughter. She needed to manage this herself. She created a nonprofit called Don't Forget Morgan.

Kelly's aunts provided guidance, and Wood offered contacts he had in the finance industry and Silicon Valley. Wood and Lions general counsel Jay Colvin sit on the board. Other Lions coworkers -- with Wood's blessing -- built the website, designed the logo and created social media plans and the first pitch video for Don't Forget Morgan's rollout in 2020.

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Progress started with a $15,000 grant to help with a mouse model study at Sanford Research in South Dakota, with another, larger, potential grant to come. In recent months, Kelly has focused largely on fundraising, and another parent of a child with BPAN, Christina Mascarenhas Ftikas, has focused on the medical side of the nonprofit.

"This is why I'm here," Kelly said. "I'm supposed to be a vehicle to get all of this awareness and hopefully a cure for BPAN so the child one, two, three, five years from now, there is hope.

"There is no, 'Go to Facebook.' There is something where you can actually give a parent, 'Here's the symptoms to look for.'"

ABOUT AN HOUR away in Ann Arbor, Michigan, Kaci Kegler and her husband, Brian, had been in the same Facebook community. Kelly, new to the group and looking for a nearby connection, wrote Kaci a message.

"Hey, my daughter was just diagnosed, could we connect?"

Kaci understood. She did the same thing, reaching out without success in 2016 after her daughter, Elle, was diagnosed. Kaci wanted to be a resource.

They talked for an hour. There wasn't much Kaci could say to soothe her. Kelly pinged a year later with another message: I'm starting a nonprofit. Kaci offered to help.Despite suffering from BPAN, Morgan is like any other 5-year-old who enjoys playing with her brother, Connor.Michael Rothstein

Days later, on Feb. 28, Kaci and her husband, Brian, an assistant athletic director for development at the University of Michigan, had their yearly fundraiser for BPAN research on Rare Disease Day at Pizza House in Ann Arbor. They met a doctor who had a connection to researchers at Michigan.

"I literally came home and texted [Kelly] and was like, 'Oh my gosh, we may have inroads,'" Kaci said. "We just started texting. I have never met Kelly face-to-face. We still haven't. But we've texted a lot and we've emailed quite a bit.

"It just kind of started."

By summer, they went from nothing to putting pieces in place for a full-fledged research project with a two-year, $140,000 grant for Barmada and Dr. Jason Chua to help start to solve BPAN.

Chua was working on the regulation of autophagy, which is the cleaning out of damaged cells, and studying BPAN became a natural extension of the work he had already been putting in. BPAN alters that in neurons. Barmada said Chua's research provided a "rare win-win situation" to potentially help with BPAN and other diseases too.

"There are a set of questions in BPAN that nobody has the answer to," Barmada said. "And Jason and myself, we just seem to be in the right position, the right place to be able to help out."

The goal is to understand what is happening within BPAN itself and how people end up with it, while also trying to find therapies for existing patients. Within a year, they are hoping to grow stem cells from people with BPAN in their lab, allowing for the creation of their own stem cells missing the WDR45 gene. Then they will try to either replace the gene or "stimulate autophagy through genetic or pharmacologic means," Barmada said. The hope is this can prevent neurodegeneration.

So far, they've hired a research assistant to work with Chua, developed tools to manipulate the gene using the genome-editing tool CRISPR and applied for approval from Michigan and the institutional review board to get skin biopsies to obtain stem cells from BPAN patients.

It's a process, but it's also a start.

Lions VP tries to save daughter from rare disease Fantasy fallout: Keep rolling with rookies? Tua's benching a lesson for young QBs Big Ben making push for first MVP award Should Chiefs worry about pass-rush woes?

After partnering with Michigan and Sanford, Don't Forget Morgan also began working with Dr. Kathrin Meyer, a researcher at the Center for Gene Therapy at Nationwide Children's Hospital at Ohio State.

"Solving this disease is going to require more than Jason and Sami," Paulson said. "It's going to be a first shot across the bow, but it's going to require more than that. I'll say this, being in the field for a long time. Scientists who are coming up the pike say they want to look at Alzheimer's, want to look at epilepsy. They don't say, 'I want to look at a rare disease.'

"The only way to solve a rare disease is to get someone hooked. Sometimes when you hook a really good one, as I think we have with Jason here, you hook them for life and they make a difference."

MORGAN IS BOUNCING around the Kozoles' suburban Detroit home on this late August day. They just returned from northern Michigan, and having two kids, especially one with special needs, makes tidiness unrealistic.

COVID-19 changed things. Morgan hadn't been to many of her therapies for months. Online school barely kept her attention. There was concern she would have regression in her learning. Instead, her speech advanced by being around Kelly, Kevin and her older brother, Connor, all day. She has sung more songs recently to help increase her vocabulary. Sometimes, she'll listen 20 times in a row.

"Even more than that," Connor said. They aren't sure how much she's truly learning versus memorization. But it is something.Morgan Kozole has inspired her mother, Detroit Lions VP Kelly Kozole, to marshal researchers and other advocates to develop a cure for BPAN, and perhaps help future generations of children who live with the disorder.Michael Rothstein

The family gathers inside Morgan's bedroom -- complete with a special Haven Bed with a zipper to keep her safe from wandering around at night, when she could accidentally turn on the stove and hurt herself or others -- sleep disorders are another BPAN issue. She sits on the floor and starts playing with her small, yellow dollhouse and a fake ice-cream maker. Kelly asks for an ice cream. Morgan makes one for herself instead and pretends to eat it.

Later, outside, Morgan kicks a soccer ball and plays a modified game of catch with a squishy football. Football, no surprise, is big. She says "hike" a lot. "She knows that term," Kevin says, laughing.

In these moments, Morgan seems like any other young child. She attends St. Hugo of the Hills Parish School in Bloomfield Hills, Michigan, but has a one-on-one para nanny to help. She interacts with people, often overly affectionate.

Sitting at the kitchen table after playtime outside, she plays with Starfall, a children's learning app, on her iPad. They hope it accelerates her word recognition. Morgan is entranced watching "Farmer in the Dell" and using her hands to eat orange slices and Cheerios. She needs a mirror in front of her to provide her a target for her mouth. She listens to books, another way to try absorbing information.

Morgan can now count to 20 and say three sentences in a row. Kelly and Kevin have tried to give Morgan a normal life in an abnormal situation, but they worry about the future -- what she won't have and won't be able to experience.

But Morgan has changed some of that outlook too.

"Focus on how she is so loving and has so much pure joy. A lot of parents of special needs [kids] say you can learn so much from these kids, and you really can," Kelly said. "She is, every morning, just so happy, and 'Mama!' Hugs and kisses to strangers. She has none of those behaviors you learn as an adult where you're not kind to people or you don't want to talk to someone.

"She is just open arms, will give you a hug and is so loving, and it's like, 'Wow, this is really what life is about.'"

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'This is why I'm here': A Detroit Lions VP tries to save her daughter from rare disease - ESPN

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Clinical data from three ongoing trials of Ad-RTS-hIL-12 plus veledimex (referred to as Controlled IL-12) for the treatment of recurrent or progressive glioblastoma multiforme (rGBM) and diffuse intrinsic pontine glioma (DIPG) were presented at the annual (virtual) meeting of the 2020 Society of NeuroOncology (SNO).

The data for the new treatment option, being developed by Ziopharm Oncology, included the first discussion of interim results of a phase II study of Controlled IL-12 in combination with cemiplimab (Libtayo; Regeneron Pharmaceuticals and Sanofi-aventis) for the treatment of rGBM that has recently completed enrollment, and updated interim data from the phase I study of Controlled IL-12 in combination with nivolumab for the treatment of rGBM and data from the first patient enrolled in the ongoing phase I/II study of Controlled IL-12 monotherapy for the treatment of DIPG.

Weve reported data for the first time from the ongoing phase II study of Controlled IL-12 in combination with PD-1 inhibitor cemiplimab, showing activation of the immune system across patients. These data are highly encouraging and underscore the potential of Controlled IL-12 to transform the treatment landscape of recurrent glioblastoma.

Interleukin-12Interleukin 12 or IL-12, a cytokine that is produced by dendritic cells, monocytes, and macrophages, and to a lesser extent by B-cells, induces TH1 differentiation in T-lymphocytes and the subsequent expression of interferon (INF). The cytokine belongs to the family of interleukin-12 which comprises the only heterodimeric cytokines, including IL-12, IL-23, IL-27, and IL-35.

IL-12 also promotes the expansion and survival of activated T-cells and NK cells and modulates the cytotoxic activity of CTLs and NK cells. During the adaptive immune response, IL-12 is known to primes antigen-specific T-cells for high IFN-g production, which drives the differentiation toward the Th-1 pathway. IL-12 can also act as an adjuvant for humoral immunity by enhancing the production of IgG2a and IgG2b antibodies, and it may enhance antibody production by B-cells.

Ziopharms Ad-RTS-hIL-12 plus veledimex is a gene therapy with an Adenoviral vector (Ad) providing the vehicle engineered to express IL-12 under the control of the RheoSwitch Therapeutic System (RTS). Ad-RTS-hIL-12 + veledimex has demonstrated a dose-related increase in tumor IL-12 mRNA and IL-12 protein expression. Discontinuation of veledimex resulted in a return to baseline IL-12 mRNA and protein expression in numerous syngeneic mouse tumor models. Image courtesy Ziopharm Oncology.[1]Although interleukin-12 (hIL-12) has anticancer activity, the systemic application is limited as a result of a toxic inflammatory responses. To solve the problem, scientists at Ziopharm assessed the safety and biological effects of an hIL-12 gene, transcriptionally regulated by an oral activator. [1]

Ziopharms Controlled IL-12 platform turns on the expression of IL-12 on demand, signaling for T-cells to attack and destroy cancerous tissue. As part of the treatment, genes coded to produce IL-12 are delivered to tumor sites. Following this step, patients are given a dose of veledimex to trigger the implanted genes to produce IL-12. This approach allows treating physicians to increase or decrease expression levels of IL-12 or turn it off altogether.[1]

PD-1 inhibitorThe updated data on combining Controlled IL-12 with nivolumab reveal a subset of patients with rGBM that demonstrate very encouraging survival at 16 months. This observation reveals that immune modulation with IL-12 and anti-PD-1 is well tolerated with an apparent survival benefit that will need further confirmation in upcoming more advanced clinical trials, said E. Antonio Chiocca, M.D., Ph.D., study investigator, Chairman of Neurosurgery at Brigham and Womens Hospital, Professor of Neurosurgery at Harvard Medical School, and Surgical Director of the Center for Neuro-oncology at Dana-Farber Cancer Institute.

These survival data in conjunction with previously reported MRIs showing partial responses is consistent with immune-mediated anti-tumor effects, Chiocca added.

CemiplimabThe results from the ongoing Phase II study of Controlled IL-12 in combination with PD-1 inhibitor cemiplimab for the treatment of adult patients with recurrent or progressive glioblastoma multiforme (rGBM), showed an activation of the immune system across patients with rGBM.

The combination trial demonstrated promising initial survival data, with median overall survival not yet reached. The data was comparable to results from a Controlled IL-12 monotherapy study previously presented at the 2020 annual meeting of the American Society of Clinical Oncology (ASCO).

Even with the best available therapies, the median overall survival for patients with rGBM is capped at 12 months. The Controlled IL-12 monotherapy has shown it can bring median overall survival to 16+ months.

Across its Controlled IL-12 studies to date, Ziopharm has identified a total of six partial responses, highlighting the promising potential of this therapy for the treatment of rGBM.

Remote-controlled therapyThe drug itself works like a remote-controlled therapy. The vector is injected directly into the tumor, but the gene of interest, IL-12, is under a transcriptional regulator, meaning that IL-12 is only expressed when the patient takes a drug that activates its transcription. Physicians can remotely control IL-12 expression by telling the patient when to take this pill.

Platform technologyAs we reflect on the growing body of evidence across our efforts utilizing our Controlled IL-12 platform, we are encouraged by the signs of efficacy we are seeing in these very hard-to-treat cancers. Not only are we observing cytokine production, increases in intra-tumoral T-cells, and predictable safety after treatment with Controlled IL-12 as a monotherapy and in combination with PD-1 inhibitors, but we have reported at least one partial response in each rGBM trial we have conducted to date, for a total of six, noted Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm

These MRI data, along with IL-12-driven immune response complement our encouraging survival data and we look forward to future data read-outs in 2021. Further, the initial look at data from the first patient in our phase 1/2 pediatric glioma study supports Controlled IL-12s safety profile and continued development, Cooper added.

Key studies: Controlled IL-12 + cemiplimabControlled IL-12 in combination with PD-1 inhibitor cemiplimab is currently being examined in a phase 2 study for the treatment rGBM (NCT04006119). Preliminary data in the on-demand presentation include:

Key studies: Controlled IL-12 + nivolumabControlled IL-12 in combination with the PD-1 inhibitor nivolumab is currently being examined in a phase I study for the treatment of rGBM. Interim data highlights shared in an oral on-demand presentation include:

As a follow up to our prior readout (ASCO 2020) for this combination which reported partial responses by MRI, the two patients had meaningful improvements in survival with one patient on 20 mg veledimex surviving 17.4 months and the other (10 mg veledimex) surviving 21.0 months (in follow up).

Diffuse Intrinsic Pontine GliomaControlled IL-12 monotherapy is being studied in a phase 1/2 dose-escalation study for the treatment of children with gliomas, including DIPG. Data highlights from the first patient in the study shared in a poster discussion, included:

It is important to note that these trials, including our previously disclosed monotherapy study, now consist of over 125 patients with rGBM. These provide deep learning that is ongoing and is part of the efforts to develop Controlled IL-12 as a potential therapy for brain cancers. We will continue to monitor the data across both the monotherapy and checkpoint inhibitor combination studies in the coming months. We believe there are multiple potential paths to registration for our Controlled IL-12 program, either as a monotherapy therapy or in combination with other agents, Cooper concluded.

Clinical trialsEvaluation of Ad-RTS-hIL-12 + Veledimex in Subjects With Recurrent or Progressive Glioblastoma, a Substudy to ATI001-102 NCT03679754A Study of Ad-RTS-hIL-12 With Veledimex in Subjects With Glioblastoma or Malignant Glioma NCT02026271Study of Ad-RTS-hIL-12 + Veledimex in Combination With Cemiplimab in Subjects With Recurrent or Progressive Glioblastoma NCT04006119A Study of Ad-RTS-hIL-12 With Veledimex in Combination With Nivolumab in Subjects With Glioblastoma; a Substudy to ATI001-102 NCT03636477A Study of Ad-RTS-hIL-12 + Veledimex in Pediatric Subjects With Brain Tumors Including DIPG NCT03330197

Highlights of Prescribing informationCemiplimab (Libtayo; Regeneron Pharmaceuticals and Sanofi-aventis) [Prescribing Information]Nivolumab (Opdivo; Bristol-Myers Squibb) [Prescribing Information]

Meeting presentationsLukas RV, Chiocca EA, OberheimBush NA, Landolfi J, Cavaliere R, Yu J, Kurz SC, Demars N, et al. Phase 2 Trial of Controlled IL-12 in Combination with PD-1 Inhibitor in Adult Subjects with Recurrent Glioblastoma (Abstract #901183) SNO 2020 Annual Meeting; Presented by: Rimas V. Lukas, MD [Presentation]Chiocca EA, Lukas RV, Chen CC, Rao G, Reardon D, Wen P, Bi WL, Peruzzi P, et al. Combination of Controlled Interleukin-12 Gene Therapy with Immune Checkpoint Blockade in Recurrent Glioblastoma: Updated Results of a Multi-Institutional, Open-Label Phase 1 Trial(Abstract #901050) SNO 2020 Annual Meeting; Presented by: Antonio Chiocca, MD.[Presentation]Goldman S, Mueller S, Chi S, Saratsis A, Allen R, Buck J, Demars N, Hadar N, Estupinan T, et al. Phase I/II Study of Controlled IL-12 as Immunotherapy for Diffuse Intrinsic Pontine Glioma (DIPG) (Abstract #901123), presented by Stewart Goldman, M.D., Division Head Hematology-Oncology, Neuro-Oncology & Stem Cell Transplantation at Lurie Childrens Hospital. [Poster]

Reference

[1] Barrett JA, Cai H, Miao J, Khare PD, Gonzalez P, Dalsing-Hernandez J, Sharma G, Chan T, Cooper LJN, Lebel F. Regulated intratumoral expression of IL-12 using a RheoSwitch Therapeutic System (RTS) gene switch as gene therapy for the treatment of glioma. Cancer Gene Ther. 2018 Jun;25(5-6):106-116. doi: 10.1038/s41417-018-0019-0. Epub 2018 May 14. PMID: 29755109; PMCID: PMC6021367.

Featured Image: Doctor report and recommend a method with patient treatment, results on brain x-ray film. Photo courtesy: 2020 Fotolia/Adobe. Used with permission.

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Encouraging Clinical Data for Controlled IL-12 for the Treatment of Glioblastoma and DIPG - OncoZine

Recommendation and review posted by Bethany Smith

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics (SwanBio), a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced the appointments of Patricia Patty Allen, former Chief Financial Officer of Zafgen, Inc.; Danny Bar-Zohar, M.D., Global Head of Development at Merck KGaA; and Alex Hamilton, Ph.D., Partner at Syncona, to the companys Board of Directors. In addition to her role as a non-executive Director, Ms. Allen will serve as the Chair of SwanBios Audit Committee.

SwanBio has been making tremendous progress in the advancement of our AAV-based pipeline of therapeutics for the treatment of neurological diseases, and we are thrilled to expand our Board of Directors with these key appointments, said Tom Anderson, Chief Executive Officer of SwanBio. Patty, Danny and Alex each bring a wealth of knowledge and expertise within their individual focus-areas, and their insights will be invaluable as we advance toward becoming a clinical company. We look forward to benefiting from their experience and collaborating on the important work ahead to deliver new medicines to patients with devastating diseases.

Im pleased to join the SwanBio Board of Directors, along with Danny and Alex, at this pivotal moment in the companys evolution, said Ms. Allen. SwanBio is driven by an exceptional team and founded based on innovative science, with a gene therapy approach that could make a significant difference for a number of people who suffer from genetic neurological diseases. I am excited for the future of this company and the opportunity to help guide them forward.

Ms. Allen is a business finance and operations leader with more than 25 years of experience leading private and public companies through initial public offerings, equity and debt financings, SEC reporting, investor relations, sell-side and buy-side, strategic and long-range planning, FP&A, treasury, risk management and business development. Most recently, she served as Chief Financial Officer of Zafgen, Inc. (now Larimar Therapeutics) from 2013-2020. Prior to Zafgen, Ms. Allen was an independent financial consultant from 2011-2012; served as Vice President of Finance, Treasurer and Principal Financial Officer of Alnylam Pharmaceuticals, Inc from 2004-2011; and as Director of Finance at Alkermes from 1992-2004. Ms. Allen also serves as a Director on the Board of Directors of several biotechnology companies and serves as the Chair of their Audit Committees. Ms. Allen graduated summa cum laude from Bryant College with a B.S. in business administration.

Dr. Bar-Zohar is a certified physician with proven expertise in drug development and a personal commitment to change peoples lives by developing sustainable, transformative healthcare solutions using both traditional and emerging technologies. He was recently appointed Global Head of Development at Merck KGaA in Darmstadt, Germany. Prior to Merck KGaA, Dr. Bar-Zohar was a Partner at Syncona and held various roles at Novartis Pharma AG from 2013-2020, most recently serving as Global Head, Clinical Development and Analytics. Before Novartis, Dr. Bar-Zohar held various clinical and medical affairs roles at Teva Pharmaceuticals Industries from 2006-2012. He obtained his medical doctor degree at the Sackler Faculty of Medicine, Tel-Aviv University and was trained in general surgery at the Tel-Aviv Medical Center.

Dr. Hamilton is an experienced financial leader within the biotech and pharmaceutical industries. Since SwanBios founding, Dr. Hamilton has played a key role in helping shape the companys strategy as a Board Observer. Before joining Syncona, he was a member of the Healthcare Investment Banking team at Jefferies International, where he worked on a range of financings and mergers and acquisitions across the biotechnology, pharmaceutical and healthcare sectors. Dr. Hamilton received his Ph.D. in immunology from the University of Cambridge.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of AAV-based gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTherapeutics.com.

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SwanBio Therapeutics Expands Board of Directors with Appointments of Proven Industry Leaders - Business Wire

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This new research report compilation added as an assessment overview of the global Russia Cryonics Technology market is directed to unravel crucial details about market developments, encompassing various factors such as market trends, lingering barrier implications as well as dominant drivers that effectively carve a favorable growth route for global Russia Cryonics Technology market progression and growth. The report specifically underpins superlative reader comprehension about multiple market developments by gauging into regional growth spots.

Access the PDF sample of the Russia Cryonics Technology Market report @ https://www.orbisresearch.com/contacts/request-sample/2441773?utm_source=Atish

Key Players Mentioned in the Report:

Alcor Life Extension FoundationBiocisionCellulisCesca TherapeuticsCryologicsCryonics Asia Ltd.Cryonics InstituteCryothermGE HealthcareHumaiKriorusOregon CryonicsOsirisPanasonic BiomedicalPraxair TechnologySigma-AldrichSouthern CryonicsThermo Fisher ScientificVWR

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Make an enquiry of Russia Cryonics Technology Market report @ https://www.orbisresearch.com/contacts/enquiry-before-buying/2441773?utm_source=Atish

Some of the most crucial market relevant information drawn in the report is aimed at equipping market players with a crisp overview of fast transitioning vendor landscape. The report is also designed to influence lucrative decision making amongst prominent players in terms of their investment discretion towards most appropriate investment decisions pertaining to dynamic product and pricing mix to initiate user acceptance. A close review of the sub-segmentation has also been tagged in the report, aimed at unveiling novel growth opportunities, offsetting market saturation. Minute classification of the growth hubs, encompassing details on global and local developments alike to entice critical decision making.

Types Covered in Report:

Slow FreezingVitrificationUltra-Rapid

Application Covered in Report:

Animal HusbandryFishery ScienceMedical SciencePreservation Of Microbiology CultureConserving Plant BiodiversityEnd user SegmentationLife Science And Healthcare FacilitiesResearch Laboratories

Browse the complete Russia Cryonics Technology Market report @ https://www.orbisresearch.com/reports/index/russia-cryonics-technology-market-report-2018?utm_source=Atish

About Us:Orbis Research (orbisresearch.com) is a single point aid for all your Market research requirements. We have vast database of reports from the leading publishers and authors across the globe. We specialize in delivering customized reports as per the requirements of our clients. We have complete information about our publishers and hence are sure about the accuracy of the industries and verticals of their specialization. This helps our clients to map their needs and we produce the perfect required Market research study for our clients.

Contact Us:Hector CostelloSenior Manager Client Engagements4144N Central Expressway,Suite 600, Dallas,Texas 75204, U.S.A.Phone No.: +1 (972)-362-8199 ; +91 895 659 515

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Global Russia Cryonics Technology Market 2020: Classification, Application And Specifications, Industry Overview, Analysis Of The Main Key Regions And...

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This new research report compilation added as an assessment overview of the global Europe Cryonics Technology market is directed to unravel crucial details about market developments, encompassing various factors such as market trends, lingering barrier implications as well as dominant drivers that effectively carve a favorable growth route for global Europe Cryonics Technology market progression and growth. The report specifically underpins superlative reader comprehension about multiple market developments by gauging into regional growth spots.

Access the PDF sample of the Europe Cryonics Technology Market report @ https://www.orbisresearch.com/contacts/request-sample/2441441?utm_source=Atish

Key Players Mentioned in the Report:

Alcor Life Extension FoundationBiocisionCellulisCesca TherapeuticsCryologicsCryonics Asia Ltd.Cryonics InstituteCryothermGE HealthcareHumaiKriorusOregon CryonicsOsirisPanasonic BiomedicalPraxair TechnologySigma-AldrichSouthern CryonicsThermo Fisher ScientificVWR

A keen observation and evaluation of the Europe Cryonics Technology market developments based on qualitative and quantitative research practices have been meticulously compiled to understand dynamics such as drivers, restraints, challenges and threats that closely influence holistic growth in global Europe Cryonics Technology market.

Make an enquiry of Europe Cryonics Technology Market report @ https://www.orbisresearch.com/contacts/enquiry-before-buying/2441441?utm_source=Atish

Some of the most crucial market relevant information drawn in the report is aimed at equipping market players with a crisp overview of fast transitioning vendor landscape. The report is also designed to influence lucrative decision making amongst prominent players in terms of their investment discretion towards most appropriate investment decisions pertaining to dynamic product and pricing mix to initiate user acceptance. A close review of the sub-segmentation has also been tagged in the report, aimed at unveiling novel growth opportunities, offsetting market saturation. Minute classification of the growth hubs, encompassing details on global and local developments alike to entice critical decision making.

Types Covered in Report:

Slow FreezingVitrificationUltra-Rapid

Application Covered in Report:

Animal HusbandryFishery ScienceMedical SciencePreservation Of Microbiology CultureConserving Plant BiodiversityEnd user SegmentationLife Science And Healthcare FacilitiesResearch Laboratories

Browse the complete Europe Cryonics Technology Market report @ https://www.orbisresearch.com/reports/index/europe-cryonics-technology-market-report-2018?utm_source=Atish

About Us:Orbis Research (orbisresearch.com) is a single point aid for all your Market research requirements. We have vast database of reports from the leading publishers and authors across the globe. We specialize in delivering customized reports as per the requirements of our clients. We have complete information about our publishers and hence are sure about the accuracy of the industries and verticals of their specialization. This helps our clients to map their needs and we produce the perfect required Market research study for our clients.

Contact Us:Hector CostelloSenior Manager Client Engagements4144N Central Expressway,Suite 600, Dallas,Texas 75204, U.S.A.Phone No.: +1 (972)-362-8199 ; +91 895 659 515

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Dublin, Nov. 24, 2020 (GLOBE NEWSWIRE) -- The "Acromegaly and Gigantism - Epidemiology Forecast to 2029" report has been added to ResearchAndMarkets.com's offering.

Acromegaly and gigantism are rare disorders of the pituitary gland, characterized by the hypersecretion of growth hormone (GH). In 98% of the cases of acromegaly and gigantism, the hypersecretion of GH results from a benign GH- secreting pituitary adenoma (Sesmilo, 2013). A common sign of acromegaly is enlarged hands and feet, and the disease can also cause gradual changes in the shape of the face, such as a protruding lower jaw and brow, an enlarged nose, thickened lips, and wider spacing between the teeth. Progression of acromegaly can result in major health problems such as diabetes mellitus, cardiovascular disease, hypertension, sleep apnea, carpal tunnel syndrome, and spinal cord compression (Mayo Clinic, 2019). While both acromegaly and gigantism are complications of uncontrolled GH levels, the presentations of the two hormonal diseases are very different; most notably, acromegaly occurs in adulthood and gigantism occurs during childhood (Eugster and Pescovitz, 1999). In 2019, the 7MM combined had 43,220 diagnosed prevalent cases of acromegaly in both sexes for ages 15 years and older.

The US accounted for the majority of these cases with 26,549 diagnosed prevalent cases, while Spain accounted for the fewest cases with 1,775 cases in 2019. The publisher epidemiologists forecast an increase in the diagnosed prevalent cases of acromegaly to 47,340 cases in 2029 in the 7MM at an Annual Growth Rate (AGR) of 0.95% during the forecast period. In 2019, the 7MM combined had 643 diagnosed prevalent cases of gigantism in both sexes for all ages. The publisher epidemiologists forecast an increase in the diagnosed prevalent cases of gigantism to 675 cases in 2029 in the 7MM at an AGR of 0.50% during the forecast period. Any change in the diagnosed prevalent cases of acromegaly and gigantism in the 7MM is attributable to changing population demographics and changing diagnosed prevalence rates in the respective markets.

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Reasons to Buy

Key Topics Covered:

1 Table of Contents1.1 List of Tables1.2 List of Figures

2 Acromegaly and Gigantism: Executive Summary2.1 Catalyst2.2 Related Reports2.3 Upcoming Reports

3 Epidemiology3.1 Disease Background3.2 Risk Factors and Comorbidities3.3 Global and Historical Trends3.4 Forecast Methodology3.4.1 Sources3.4.2 Forecast Assumptions and Methods3.4.3 Diagnosed Prevalent Cases of Acromegaly and Gigantism3.4.4 Diagnosed Prevalent Cases of Acromegaly and Gigantism with Macroadenoma3.4.5 Diagnosed Prevalent Cases of Acromegaly and Gigantism with Diabetes3.4.6 Diagnosed Prevalent Cases of Acromegaly and Gigantism with Cardiovascular Disease3.4.7 Diagnosed Prevalent Cases of Acromegaly and Gigantism with Hypertension3.5 Epidemiological Forecast for Acromegaly, 2019-20293.5.1 Diagnosed Prevalent Cases of Acromegaly3.5.2 Sex-Specific Diagnosed Prevalent Cases of Acromegaly3.5.3 Age-Specific Diagnosed Prevalent Cases of Acromegaly3.5.4 Diagnosed Prevalent Cases of Acromegaly with Macroadenoma3.5.5 Diagnosed Prevalent Cases of Acromegaly with Comorbidities3.5.6 Diagnosed Prevalent Cases of Gigantism3.5.7 Sex-Specific Diagnosed Prevalent Cases of Gigantism3.5.8 Age-Specific Diagnosed Prevalent Cases of Gigantism3.5.9 Diagnosed Prevalent Cases of Gigantism with Macroadenoma3.5.10 Diagnosed Prevalent Cases of Gigantism with Comorbidities3.6 Discussion3.6.1 Epidemiological Forecast Insight3.6.2 Limitations of the Analysis3.6.3 Strengths of the Analysis

4 Appendix4.1 Bibliography4.2 About the Authors4.2.1 Epidemiologist4.2.2 Reviewers4.2.3 Global Director of Therapy Analysis and Epidemiology4.2.4 Global Head and EVP of Healthcare Operations and Strategy4.3 About the Publisher4.4 Contact4.5 Disclaimer

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Epidemiology Forecast on Acromegaly and Gigantism to 2029 - - GlobeNewswire

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Rachel Scanlon's lucky toilet came through for her not once, but twice.What you dont have a lucky toilet? Maybe Rachels story will convince you to reconsider.

On TuesdaysRyan Tubridy Show, Rachelwhos beenthrough IVF an extraordinary thirteen times started by telling Ryanthat before deciding on going down the road of IVF, she and her partner Kevin had experienced a lot ofdisappointment:

"We had a lot of miscarriages. And a lot of early miscarriages and a lot of later miscarriages. But, yes, we had a lot and so, we sought treatment."

Ryan asked Rachel to give an outline of what the IVF process involves and she madesomethings clear at the start: its a long process and different womenhave different experiences.

Different clinicstreat people differently and every womans body is different. So, no two women will have the exact same IVF experience.There are different protocols involved and Rachel found herself, more than once,on a protocol where she downregulated.

"Your body is kind ofsupressedinto a menopause-like state.And I suppose its to allow the clinic to take over and to take over the regulation of your body and so you stopproducingeggs so that the drugs can takeover andproduce the eggs for you."

The menopause-like stateprobablylasts for a couple of weeks, Rachelcontinued,beforehormone injections begin. The injections are intended to produce follicles which will eventually produce eggs. She was injecting herself every evening and sometimes more than once a dayand she was scanned regularly to ensure that the hormones are overor under-stimulating follicle production.

Pumping your body full of hormones has other effects besides follicle production, though:"Your moods are up and down, youre all over the placeI mean, youre hard to live with. Its difficult. And youre bloated as well.Your stomach is bloated. You actually look like youre pregnant because your ovaries are just bulging. Youre just a hot mess, really."

Thats one way of putting it.Of course, the long, gruelling, expensiveprocess is worth it when you get a baby at the end of it. But when you dont,when the process doesnt work at all, it can be extremely disheartening.

"Looking back, one of the hardest periods for me was when that first IVF didnt work."

Rachel reckons she was naive in the beginning because she had so much hopeinvested in that first attempt, only for it to fail:

"You eventually get to a pointwhere youre taking a test. My first time, I didnt even get to take the test, my period just arrived, like nothing had happened. Like we hadnt spent all this money, like I hadnt taken all these injections. I was absolutely heartbroken the first time it didnt work."

That first time wasjust the start of a six-year IVFjourney, that took in thirteen proceduresin four clinicsacrossthreecountriesand eventually resulted in two joyouspositivetests in that lucky toilet,in a cafcalled Wilde and Green in Milltown, Dublin,two years apart.

Little Eve and hersisterErin are thetwo happy endings toRachel and Kevinsjourney.

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IVF: "I was absolutely heartbroken the first time it didn't work" - RTE.ie

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Photo Courtesy of Unsplash.

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

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

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

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

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

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

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

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

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Research may lead to identifying novel therapies for cardiac patients

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

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

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

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

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

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

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

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

About the Mount Sinai Health System

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

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

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

Recommendation and review posted by Bethany Smith

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

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

Get a Sample Copy of the Report at: https://i2iresearch.com/report/global-autologous-stem-cell-based-therapies-market-2020-market-size-share-growth-trends-forecast-2025/

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

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

Key applications:Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

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

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

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

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

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Global 3D Cardiac Mapping Systems Market: Overview

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

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

Global 3D Cardiac Mapping Systems Market: Notable Developments

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

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

Global 3D Cardiac Mapping Systems Market: Key Growth Drivers

Rising Number of Patients with Cardiac Disorders and Arrhythmia Fillips Market

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

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

Global 3D Cardiac Mapping Systems Market: Regional Outlook

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

About TMR Research:

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

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

Recommendation and review posted by Bethany Smith