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Anonymous US stem cell donor saves the life of Norwich girl – Eastern Daily Press

By daniellenierenberg

PUBLISHED: 18:00 18 June 2020

Simon Parkin

Imogen Roe returned home to Norfolk after 100 days in isolation in hospital. Picture: Anna Dagless

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A nine-year-old girls life was saved by an anonymous blood stem cell donor after a brave battle with leukaemia in which she asked her mum am I going to die?

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Imogen Roe, from Norwich, had just turned six when she was treated for routine tonsillitis. But her worried parents, Anna and Dean, took her back to the doctors when they noticed abnormal bruising and a rash on her body.

They were told to take Imogen to their local hospital, and were soon confronted with the devastating diagnosis.

Mum Anna, 38, said: We suspected it was a reaction to penicillin at worst. But within an hour of being at our local hospital we were told it was leukaemia, and within 24 hours we were in Addenbrookes Hospital. It all happened so fast, I dont remember feeling much other than panic and shock.

Because of the aggressiveness of her leukaemia, young Imogen began two-and-a-half years of high dosage chemotherapy.

Anna said: Imogen was such a trouper, and very co-operative which made for a very easy patient!

I cannot believe how well she just got on with things; cannulas, nose tubes, general anaesthetics, surgery - even though at times she has been very scared about procedures.

During this time the family was divided, with Imogen and her mum in Addenbrookes, whilst Dad Dean, 39, stayed at home with their other two children, Imogens twin sister Charlotte, and older brother Liam, 11.

MORE: Esm, three, wins 19-month cancer battle but cannot celebrate traditional bell ringingIn January 2019, almost three years after her diagnosis, young Imogen finished her chemo and rang her end of treatment bell to huge applause from doctors, nurses, hospital staff - and her proud, but exhausted parents.

The young family were finally able to get their lives back on track, but unfortunately more heartbreaking news was just around the corner.

Anna said: It was July 2019, almost three years to the day since Imogens original diagnosis, and we had just bought a puppy and booked a family holiday abroad with friends. Then we noticed that familiar rash on her legs, and were told to bring her to hospital.

After undergoing tests, doctors confirmed the worst Imogen had relapsed and the leukaemia had come back.

Doctors revealed that, in addition to further chemotherapy, Imogens best chance of beating the disease was to have a blood stem cell transplant from a matching donor.

Imogens siblings were both tested, and the family were delighted to be told that her sister Charlotte was a 100% match.

However, after further tests, they were confronted with shocking news after a decade of thinking Charlotte and Imogen were non-identical twins, they were in fact identical, which meant that Charlotte would not be a suitable donor after all.

Doctors began looking for Imogens potential lifesaver elsewhere searching the worldwide register of potential blood stem cell donors, hoping to find a perfect stranger who happens to be Imogens genetic twin.

With Imogens life hanging in the balance a match was found. Cord blood, donated by a new mother in the USA and frozen nine years prior, was a perfect match for Imogen. This was the only suitable match for Imogen anywhere in the world.

MORE: How support, prayers and herbal rememdies helped nurse beat coronavirusAs the frozen cord blood was prepared to be flown from America to the UK, Imogen had 10 days of conditioning treatment prior to transplant; four days of extremely strong chemotherapy, and then eight sessions of total body irradiation, to prepare her to receive the new blood stem cells.

This is an incredibly vulnerable point in any treatment plan. The new marrow should, over a few weeks, start to regenerate within the body, but for Imogen, after 36 days of daily blood tests, there was still no sign of any new cells being manufactured.

Her mum said: It was a very serious situation as without any white cells to fight off infection, Imogen was extremely vulnerable even from her isolation room, as you can pick up bugs from your own body.

Ten days post transplant she got an infection, and she went into septic shock. This led to several serious viruses, a chest infection, and bacterial infection. We had the rapid response team on standby for a transfer to intensive care, but Imogen pulled through, just as her dad arrived after making the five hour journey from our home.

Imogen remained in isolation in Bristol for 99 days, fighting off multiple infections, whilst dad Dean travelled the five hours back and forth between home and the hospital to bring Anna clean clothes and supplies.

Finally, Imogen was transferred back to Addenbrookes to continue her recovery one step closer to home.

MORE: Wife of rugby star launches new business two years after being given a month to liveOn March 13, in the midst of the Covid-19 pandemic, she was finally discharged and returned home to her mum, dad and siblings, who are now all self isolating.

It had been 170 days since she, or mum Anna, had seen any extended family.

Speaking about Imogens anonymous donor, Anna said the family were acutely aware of the luck involved in finding a match.

She said: Imogen had just one match. There are some people we know through our time in hospitals who were fortunate enough to have a selection of matches, and many others are still waiting for a match that may never come.

If you are aged between 17 and 55 and in general good health, you take the first step to register as a blood stem cell donor by registering for a home swab kit at dkms.org.uk

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Rare Hematology Treatment Market Analysis 2020 Along with Research Report 2026 – Cole of Duty

By daniellenierenberg

The Latest Study on Rare Hematology Treatment Market Perspective, Comprehensive Analysis along with Major Segments and Forecast, 2020-2026. The Rare Hematology Treatment Market report is a valuable source of data for business strategists. It provides the industry overview with market growth analysis with a historical & futuristic perspective for the following parameters; cost, revenue, demands, and supply data (as applicable). The report explores the current outlook in global and key regions from the perspective of players, countries, product types, and end industries. This Rare Hematology Treatment Market study provides comprehensive data that enhances the understanding, scope, and application of this report.

Top Companies: Takeda, Biogen, Novo Nordisk, Bayer, CSL Behring, Pfizer, PRA Health Sciences, Celgene, Alexion Pharma, Amgen

Get a sample copy Of this Report:

https://www.marketinsightsreports.com/reports/06122088250/global-rare-hematology-treatment-market-size-status-and-forecast-2020-2026/inquiry?Mode=31

Overview: Blood develops from hematopoietic stem cells (HSC) and formation takes place in the bone marrow through a sequence of the regulated process known as hematopoiesis. Blood contains component such as plasma, RBS, WBC, and platelets. Blood disorders include bleeding disorders such as hemophilia, blood clots; blood cancers (leukemia, lymphoma, and myeloma). When the normal process of the blood development fails, abnormal blood cell type is produced, which causes blood cancer and other blood disorders. The rare hematological disease includes anemia-type red blood cell conditions, white blood cell dysfunctions, immuno-disorders and other platelet-based abnormalities.

Global Rare Hematology Treatment Market Split By Product Type And Applications:

This report segments the global Rare Hematology Treatment Market on the basis ofTypesare:

Plasma Derived FactorsRecombinant Factors

On the basis ofApplication, the Global Rare Hematology Treatment Market is segmented into:

Hemophilia AHemophilia BVon Wilebrand DiseaseOthers

Regional Analysis: For a comprehensive understanding of market dynamics, the global Rare Hematology Treatment Market is analyzed across key geographies namely: United States, China, Europe, Japan, Middle East & Africa, India, and others. Each of these regions is analyzed on the basis of market findings across major countries in these regions for a macro-level understanding of the market.

Explore Full Report With Detailed TOC Here:

https://www.marketinsightsreports.com/reports/06122088250/global-rare-hematology-treatment-market-size-status-and-forecast-2020-2026?Mode=31

Influence of the Rare Hematology Treatment Market report:-Comprehensive assessment of all opportunities and risks in the Rare Hematology Treatment Market. Rare Hematology Treatment Market recent innovations and major events.-A detailed study of business strategies for the growth of the Rare Hematology Treatment Market-leading players.-Conclusive study about the growth plot of the Rare Hematology Treatment market for forthcoming years.-In-depth understanding of Rare Hematology Treatment Market -particular drivers, constraints, and major micro markets.-Favorable impression inside vital technological and market latest trends striking the Rare Hematology Treatment Market.

What are the market factors that are explained in the report?

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

-Key Market Features: The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

Analytical Tools: The Global Rare Hematology Treatment Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

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Stem Cell Therapy Market to 2027 – Global Analysis and Forecasts – ResearchAndMarkets.com – Yahoo Finance

By daniellenierenberg

The "Stem Cell Therapy Market to 2027 - Global Analysis and Forecasts by Type; Treatment; Application; End User, and Geography" report has been added to ResearchAndMarkets.com's offering.

The stem cell therapy market was valued at US$ 1,534.55 million in 2019 and is estimated to reach US$ 5,129.66 million by 2027; it is expected to grow at a CAGR of 16.7% from 2020 to 2027.

The increasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines are the key factor driving the stem cell therapy market. However, high cost related of the stem cell therapy limits the growth of the market.

Stem cell research has been widely investigated globally for various medical applications, especially for the treatment of humans. This raises the importance of creating public awareness about stem cell research and its clinical potential. The main role of stem cells is in the replacement of dying cells and reconstruction of damaged tissues. Based on the extensive stem cell research, many scientists have claimed that these cells could probably be used in the treatment of various diseases, including cancer and cardiovascular disease.

There is a large number of potential treatment procedures that are undergoing clinical trials, and a notably few stem cell therapies have won FDA (i.e., US -Food and Drug Administration) approval for clinical usage. For instance, in 2019, the FDA approved Fedratinib for the first-line treatment for myelofibrosis. Moreover, stem cell therapies are widely used in bone marrow transplantation, and these therapies have benefited thousands of people suffering from leukemia. Hematopoietic stem cells are used for treating more than 80 medical diseases, including immune system disorders, blood disorders, neurological disorders, metabolic disorders, genetic disorders, and several types of cancers, such as leukemia and lymphoma; this is also likely to boost the demand for this treatment procedure during the forecast period. Researchers are further investigating the use of stem cell therapies in the treatment of autoimmune disorders.

The global stem cell therapy market has been segmented on the basis of type, treatment, application type, and end user. Based on type, the market has been segmented into adult stem cell therapy, induced pluripotent stem cell therapy, embryonic stem cell therapy, and others. The adult stem cell therapy held the largest share of the market in 2019; however, induced pluripotent stem cell therapy is estimated to register the highest CAGR in the market during the forecast period. Based on treatment, the stem cell therapy market has been segmented into allogeneic and autologous. The allogeneic segment held a larger share of the market in 2019; however, the market for the autologous segment is expected to grow at a higher CAGR during the forecast period.

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Global Bone Marrow-Derived Stem Cells (BMSCS) Market (COVID 19 Impact Analysis) Data Highlighting Major Vendors, Promising Regions, Anticipated Growth…

By daniellenierenberg

Global Bone Marrow-Derived Stem Cells (BMSCS) Market research report delivers comprehensive analysis of the market structure along with estimations of the various segments and sub-segments of the market. This study also analyzes the market status, market share, growth rate, sales volume, future trends, market drivers, market restraints, revenue generation, opportunities and challenges, risks and entry barriers, sales channels, and distributors. The company profiles of all the chief and dominating market players and brands who are taking steps such as product launches, joint ventures, mergers and acquisitions are mentioned in the report. With the use of SWOT analysis and Porters Five Forces analysis which are two of the standard, prominent and full-proof methods, this Global Bone Marrow-Derived Stem Cells (BMSCS) Market report is been framed.

Global Bone Marrow-Derived Stem Cells (BMSCS) Market By Service Type (Sample Preservation and Storage, Sample Analysis, Sample Processing, Sample Collection and Transportation), Application (Personalized Banking Applications, Research Applications, Clinical Applications), Country (U.S., Canada, Mexico, Germany, Italy, U.K., France, Spain, Netherland, Belgium, Switzerland, Turkey, Russia, Rest of Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Rest of Asia- Pacific, Brazil, Argentina, Rest of South America, South Africa, Saudi Arabia, UAE, Egypt, Israel, Rest of Middle East & Africa), Market Trends and Forecast to 2027

By providing an absolute overview of the market, Global Bone Marrow-Derived Stem Cells (BMSCS) Market report covers various aspects of market analysis, product definition, market segmentation, key developments, and the existing vendor landscape. Such market insights can be accomplished with this comprehensive Global Bone Marrow-Derived Stem Cells (BMSCS) Market research report which takes into account all the aspects of current and future market. The report provides wide-ranging analysis of the market structure along with the estimations of the various segments and sub-segments of the market. This Global Bone Marrow-Derived Stem Cells (BMSCS) Market research report delivers an analytical measurement of the main challenges faced bythe business currently and in the upcoming years.

Bone marrow-derivedstem cells(BMSCS) marketis expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to growing at a CAGR of 10.4% in the above-mentioned forecast period. Increasing awareness regarding the benefits associates with the preservation of bone marrow derived stem cells will boost the growth of the market.

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The major players covered in the bone marrow-derived stem cells (BMSCS) market report are CBR Systems, Inc, Cordlife Sciences India Pvt. Ltd., Cryo-Cell International, Inc.ESPERITE N.V., LifeCell International Pvt. Ltd., StemCyte India Therapeutics Pvt. Ltd, PerkinElmer Inc, Global Cord Blood Corporation., Smart Cells International Ltd., Vita 34 among other domestic and global players. Market share data is available for Global, North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Some of the factors such as introduction of novel technologies for the preservation of stem cells and their storage, surging investment that will help in research activities leading to stem cells benefits, adoption of hemotopoietic stem cell transplantation system will accelerate the growth of the bone marrow-derived stem cells (BMSCS) market in the forecast period of 2020-2027. Various factors that will create opportunities in the bone marrow-derived stem cells (BMSCS) market are increasing occurrences of various diseases along with rising applications in emerging economies.

Large cost of operation and strict regulatory framework will restrict the growth of bone marrow-derived stem cells (BMSCS) market in the above mentioned forecast period. Ethical concern leading to stem cells will become the biggest challenge in the market growth.

Global Bone Marrow-Derived Stem Cells (BMSCS) Market By Service Type (Sample Preservation and Storage, Sample Analysis, Sample Processing, Sample Collection and Transportation), Application (Personalized Banking Applications, Research Applications, Clinical Applications), Country (U.S., Canada, Mexico, Germany, Italy, U.K., France, Spain, Netherland, Belgium, Switzerland, Turkey, Russia, Rest of Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Rest of Asia- Pacific, Brazil, Argentina, Rest of South America, South Africa, Saudi Arabia, UAE, Egypt, Israel, Rest of Middle East & Africa), Market Trends and Forecast to 2027

Global Bone Marrow-Derived Stem Cells (BMSCS) Market Scope and Market Size

Bone marrow-derivedstem cells(BMSCS) market is segmented on the basis of service type and application. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Thisbonemarrow-derived stem cells (BMSCS) market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on bone marrow-derived stem cells (BMSCS) market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Bone Marrow-Derived Stem Cells (BMSCS) Market Country Level Analysis

Bone marrow-derivedstem cells(BMSCS) market is analysed and market size insights and trends are provided by country, service type and application as referenced above.

The country section of the bone marrow-derivedstem cells(BMSCS) market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Bone marrow-derived stem cells (BMSCS) market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipments, installed base of different kind of products for bone marrow-derived stem cells (BMSCS) market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the bone marrow-derived stem cells (BMSCS) market. The data is available for historic period 2010 to 2018.

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Key Highlights of Report

Competitive Landscape and Bone Marrow-Derived Stem Cells (BMSCS) Market Share Analysis

Bone marrow-derived stem cells (BMSCS) market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to bone marrow-derived stem cells (BMSCS) market.

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Healthcare innovations making waves in 2020 – Crawley Observer

By daniellenierenberg

The speed of change is fascinating

As we head into the 2020s, tech developers and medical scientists are working hard to ensure that we stay healthy and well-cared for, for as long as possible. Here are some of the big healthcare innovations making waves at the moment.

Artificial blood has been proposed for a number of years, but it is only recently that it has gained any sort of traction in medical circles. The science behind it is simple to understand while no doubt actually being incredibly complex. Artificial blood does not seek to replace blood entirely but instead to supplement red blood cells in the body, to carry oxygen where it needs to be delivered.

These blood cells are called artificial as they are generated in a lab environment instead of a human body and are grown from stem cells. Researchers typically use hematopoietic stem cells taken from bone marrow for growing such cells. With blood donation dropping, this could be a timely solution to support healthcare professionals alongside blood donation drives.

Blindness is a massive issue no matter where you live on the planet. In particular, corneal blindness is the fourth most common blindness in the world. However, there is now a solution that is cheap and easy to deliver, and that could make a massive difference to many people.

Many are now coming forward to support medical staff in their endeavours in this sector. Tej and Wendy Kohli are key voices in creating help here. Following other charitable givers like Bill and Melissa Gates and Warren Buffett, it is clear that input from top figures like this is crucial in supporting a cause and seeing it develop into a system that could genuinely make a difference.

The ability to use genetic information to come up with a treatment plan for a patient is a precise art. However, research into the world of genomics is making this a much more viable method of treatment than it has previously.

The scope for this is massive. So much of our response to certain treatments can be bound up in our genetics and research here could change medicine forever. With setbacks like organ transplant rejection and gene mutation still having a major impact on medicine as a whole, it is incredibly important that researchers work to find a solution that works around these issues. Comprehensive and detailed genomics might be it.

All three of these areas have seen some massive leaps forward in the past month. No matter what, focus needs to be kept on these key areas in the future. Only then will we see some massive improvements that could shape our healthcare for the better. These developments could lead to the complex becoming the norm in worldwide healthcare.

Developments made today might one day be celebrated as pioneering breakthroughs that helped to transform healthcare and the lives of humanity.

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Hematopoietic Stem Cell Transplantation (HSCT) Market Segments, Opportunity, Growth and Forecast by End-use Idustry 2020-2026 – Cole of Duty

By daniellenierenberg

Hematopoietic Stem Cell Transplantation (HSCT) Market report provide the COVID19 Outbreak Impact analysis of key factors influencing the growth of the market Size (Production, Value and Consumption). This Hematopoietic Stem Cell Transplantation (HSCT) industry splits the breakdown (data status 2014-2020 and Six years forecast 2020-2026), by manufacturers, region, type and application. This study also analyses the Hematopoietic Stem Cell Transplantation (HSCT) market Status, Market Share, Growth Rate, Future Trends, Market Drivers, Opportunities and Challenges, Risks and Entry Barriers, Sales Channels, Distributors and Porters Five Forces Analysis.

Hematopoietic Stem Cell Transplantation (HSCT) Market competitive landscapes provides details by topmost manufactures like (Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc), including Capacity, Production, Price, Revenue, Cost, Gross, Gross Margin, Growth Rate, Import, Export, Market Share and Technological Developments

Get Free Sample PDF (including COVID19 Impact Analysis, full TOC, Tables and Figures)of Hematopoietic Stem Cell Transplantation (HSCT)[emailprotected]https://www.researchmoz.us/enquiry.php?type=S&repid=2276986

Hematopoietic Stem Cell Transplantation (HSCT) Market Competition by Manufacturers (2020 2026): Hematopoietic Stem Cell Transplantation (HSCT) Market Share of Top 3 and Top 5 Manufacturers, Hematopoietic Stem Cell Transplantation (HSCT) Market by Capacity, Production and Share by Manufacturers, Revenue and Share by Manufacturers, Average Price by Manufacturers By Market, Manufacturers Manufacturing Base Distribution, Sales Area, Product Type, Market Competitive Situation and Trends, Market Concentration Rate.

Scope of Hematopoietic Stem Cell Transplantation (HSCT) Market:In 2019, the market size of Hematopoietic Stem Cell Transplantation (HSCT) is million US$ and it will reach million US$ in 2025, growing at a CAGR of from 2019; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.

In this report, 2018 has been considered as the base year and 2019 to 2025 as the forecast period to estimate the market size for Hematopoietic Stem Cell Transplantation (HSCT).

On the basis of product type, this report displays the shipments, revenue (Million USD), price, and market share and growth rate of each type.

Allogeneic Autologous

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, shipments, revenue (Million USD), price, and market share and growth rate foreach application.

Peripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)

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Hematopoietic Stem Cell Transplantation (HSCT) Market: Regional analysis includes:

The Study Objectives Of This Hematopoietic Stem Cell Transplantation (HSCT) Market Report Are:

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Trial Omitting Total Body Irradiation Before HSCT Stopped Early – Medscape

By daniellenierenberg

Hematopoietic stem cell transplantation (HSCT) may offer the chance of a cure for patients with leukemia and other blood cancers, but the process of preparing the body to receive such a transplant can be brutal, involving whole body irradiation as well as chemotherapy conditioning. New results show that both steps are needed: a trial that omitted whole body irradiation in young patients with acute lymphoblastic leukemia (ALL) was stopped early because of significantly poorer outcomes.

The multicenter, global FORUM (For Omitting Radiation Under Majority Age) trial involved 75 centers in 17 countries between 2013 and 2018.

"Our study shows significantly better outcomes for total body irradiation compared to myeloablative chemo-conditioning arms, with no differences between the [two]chemo-conditioning groups," concluded Christina Peters, MD, professor of pediatrics in the Department of Stem Cell Transplantation at St Anna Children's Hospital in Vienna, Austria.

The findings in favor of total body irradiation were pronounced enough that the study was halted early by a safety committee, she added.

Peters presented the findings as part of the virtual European Hematology Association (EHA) 25th Annual Congress 2020.

Describing the results as "sobering," session co-moderator Shai Izraeli, MD, director of the Department of Hematology-Oncology at Schneider Children's Medical Center, in Petah Tikva, Israel, said an online comment from the virtual meeting audience reflected the reaction to these unwelcome results: "So we are still stuck with total body irradiation?"

Peters responded the good news is that the number of patients needing to undergo stem cell transplants is low, and with research advances, may hopefully drop even further.

"Only 10% of patients under the age of 18 nowadays undergo allogeneic HSCT, and perhaps in the future that will become even less if we are able to rescue some of the groups with other immunological measures such as CAR-T cells and antibodies," she said.

"I think it is very important to better identify those who really need total body irradiation in the future," she added.

Commenting for Medscape Medical News, Izraeli agreed.

"The prognosis of children after bone marrow transplantation is excellent the majority are cured from their leukemia," he said. "And we have to remember that those who undergo bone marrow transplant have the worst leukemias."

He pointed out that, in fact, contemporary chemotherapy alone is effective in the treatment of more than 90% of patients with ALL younger than aged 18.

For the 10% of patients who do not respond to chemotherapy alone and undergo allogeneic HSCT, about 50% to 80% of pediatric patients who have resistant leukemia are cured. However, the total body irradiation used to prepare the body to receive the transplant is linked to potentially serious consequences later in life, including sterility, lung problems, growth retardation, and secondary cancer.

To determine if the irradiation component could be safely replaced with a chemotherapy-based conditioning approach, Peters and colleagues conducted the FORUM trial.

In total 413 patients undergoing HSCT were enrolled and randomized to pretransplant conditioning with total body irradiation and etoposide (n = 202) or a chemotherapy-only approach with fludarabine/thiotepa/busulfan (flu/thio/bu; n = 99) or fludarabine/thiotepa/treosulfan (treo; n = 93).

Most patients (72%) had B-cell precursor ALL and 23% had T-cell ALL. Just over half (54%) were transplanted in first complete remission (CR1), 40% in CR2, and 4% in CR3.

The source of stem cells was bone marrow for most patients (82%); peripheral blood stem cell for 12%, and cord blood for 4%.

The aim of the study was to demonstrate noninferiority with the chemotherapy approach.

However, the significantly inferior outcome observed in the chemotherapy-only group led to randomization being halted in March 2019.

The 2-year overall survival in the intent-to-treat (ITT) analysis, with a mean observation time of 2.1 years, was 0.75 0.04 for chemo-conditioning versus 0.91 0.02 for total body irradiation/etoposide (ITT P < .001).

The ITT analysis showed relapses were significantly higher in the chemo-conditioning group (2-year cumulative incidence of relapse [CIR], 0.33) compared with the total body irradiation group (CIR, 0.12; P < .001).

The 2-year event-free survival (EFS) rate was also significantly higher in the total body irradiation group (0.86 vs 0.58; P < .001), and transplant-related mortality over 2 years was lower with total body irradiation (0.02 vs 0.09; P = .02).

A per-protocol analysis showed the 2-year overall survival to be the same in the two chemotherapy groups (both 0.77 0.05) compared with 0.91 0.02 in the total body irradiation group (P = .003).

"In this cohort [the 91% overall survival rate] may even be lower than contemporary intensive frontline therapy results that are achieved nowadays," Peters said.

In looking at subgroups, there were no significant differences according to age group or cancer phenotype, while MLL rearrangement was associated with higher relapse incidence.

Remission status was found to notably influence EFS, dropping from 0.91 in CR1 patients with total body irradiation to 0.76 in CR2 patients. However, total body irradiation remained significantly higher compared with the chemo-conditioning groups in CR1 (P = .004) and CR2 (P < .001).

Transplant-related mortality was not significantly different between the total body irradiation and chemo-conditioning groups in the CR1 or CR2 groups (P = .09 and P = .18, respectively), despite the significant difference when remission status was not included.

Overall, "we tried to identify subgroups in which total body irradiation might be eliminated, however in all analyses, total body irradiation was better than chemo-conditioning in all arms," Peters said.

Meanwhile, the findings underscore that even when patients cannot receive total body irradiation, the alternative chemo-conditioning therapy in fact shows favorable efficacy on its own, Izraeli said.

"The prognosis of the chemotherapy group is also quite remarkably good, although less than the total body irradiation arm. This means that if for some reason total body irradiation cannot be given, the chemotherapy is a very reasonable alternative."

Peters has reported relationships with Amgen, Novartis, Pfizer, Medac, Jazz, and Neovii. Izraeli has reported no relevant financial relationships.

EHA 2020 Congress. Presented June 12, 2020. Abstract S102.

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Coronavirus: inside the UAE stem cell centre working to treat Covid-19 – The National

By daniellenierenberg

When Dr Yendry Ventura began work to set up the Abu Dhabi Stem Cell Centre in late 2018, there was, he says, nothing else "related to stem cell therapy in the emirate.

Fast forward to today and the situation has changed dramatically. After opening in December last year, the centre has already received international press coverage over to its research into a treatment for Covid-19.

Their groundbreaking work has involved taking stem cells from a patients blood and returning them, via a nebuliser, as a fine mist to the lungs.

There they help regenerate lung cells and improve the body's immune response by preventing an overreaction to the infection that can damage healthy cells.

What characterises the method, says Dr Ventura, is that very little manipulation of the cells is needed for the treatment to be effective.

The future for the stem cells lies in regenerative medicine, in which you can treat almost all the degenerative conditions.

Dr Yendry Ventura

We separate a specific layer of cells from the blood, Dr Ventura told The National. Were the first one to use these cells with this route with this method.

We believe this way the cells can be aimed much better to the affected organs - the upper and lower respiratory tract.

In April, the centres efforts to develop a Covid-19 treatment resulted in the recovery of all 73 patients the treatment was initially trialled on. A quarter had been in intensive care.

The results appeared so promising that this month the centre secured intellectual property rights to the technique, allowing the treatment to be widely licensed, including to facilities abroad.

The ongoing work is a case of the centres specialists applying their expertise to best effect to help in a time of crisis, Dr Ventura said.

But the research is a departure from the facilitys usual purpose, which involves developing cutting-edge stem cell treatments for conditions such as cancer and heart disease.

Stem cells were first extracted from humans and grown in laboratories less than a quarter of a century ago.

The human body is mostly made of specialised cell types, such as heart muscle cells, kidney cells or nerve cells, all of which have a particular form related to their function.

Stem cells, however, have not yet undergone the process of developing into a specialised cell type, and are able to be manipulated to perform specific a function.

In adults, stem cells are found in tissues including fat and bone marrow, and these can be turned into multiple cell types.

One technique that the Abu Dhabi Stem Cell Centre plans to implement is haematopoietic stem cell transplantation, which involves stem cells being removed from an individual who is due to have cancer treatment.

The cells are then processed in a laboratory and injected into the patient after they have undergone chemotherapy or radiotherapy.

In this way, they can replace stem cells destroyed by the treatment, allowing a patient to tolerate a higher dose of therapy.

Dr Ventura says that similar treatments were applicable to most cancers of the blood as well as cancers that produce solid tumours.

There are many of these therapies still in research stage, but if you conquer this research, you can have a programme in which you can ... treat many kinds of cancers at the same time in one centre, he said.

The reality is that cell therapy is curing cancer We need to improve this therapy and make it available for many other people.

The future for the stem cells lies in regenerative medicine, in which you can treat almost all the degenerative conditions.

You can create in the future, if you have the right technologies, even artificial organs.

Set up with private sector funding in collaboration with the UAE authorities, the Abu Dhabi Stem Cell Centre works closely with experts at Sheikh Khalifa Medical City.

But the institution has stressed it is keen to forge further partnerships with both public and private sector medical institutions.

Currently, it operates seven days a week and has more than 100 staff, including nurses, technicians and doctors specialising in immunology, haematology, pathology, orthopaedics, urology and radiology.

In another initiative, the facility has recently begun running Minimal Residual Disease tests, which look at how many malignant cells remain in a patients blood or bone marrow.

These tests are useful for people with a variety of blood cancers, including lymphoma, leukaemia and myeloma. But they require fresh samples from the patient, so the lack of UAE testing facilities has, until now, required patients to travel abroad.

We try to implement the tests here in the Abu Dhabi Stem Cell Centre so the patient does not need to travel anymore, said Dr Ventura.

Updated: June 16, 2020 12:43 PM

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COVID-19 and cancer care – Pursuit

By daniellenierenberg

People with cancer may have compromised immunity due to their disease or its treatment. Early reports suggest cancer is a risk factor for severe COVID-19 disease as a result, many patients with cancer, and their families, are concerned about the risks and impacts of COVID-19.

Their concerns and distress are likely to be compounded by extensive media coverage of the pandemic, rapid changes in information about COVID-19 and continuing uncertainty about how to contain the disease.

On top of this, community transmission of COVID-19 has threatened the capacity of cancer services to provide routine investigations and care. In some cases, this has seen a prioritisation or modification of patients cancer therapies.

For example, chemotherapy may be postponed as this can compromise the immune system and make patients more vulnerable to developing COVID-19 disease.

Patients and families may, in turn, be frightened about not receiving proper treatment.

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Another cause for concern is the change in the routine clinical management of cancer patients, partly because patients fear being infected. In Victoria, cancer hospitals have reported a 40 per cent decline in patient presentations for cancer management appointments since the stay-at-home guidelines in late March 2020.

This has raised concern among oncology health professionals about the health and wellbeing of vulnerable cancer patients whose health outcomes are likely to be negatively affected.

As patients are presenting less to hospital, it becomes crucial to develop new ways of identifying any distress in order to provide high-quality cancer management and supportive care.

The restrictions imposed by governments have been effective in limiting the spread of COVID-19, but social distancing, quarantine and visitor limitations have also reduced the opportunity for family support and connection important sources of strength and wellbeing.

These same government restrictions have also increased the use of telehealth to deliver care to cancer patients during COVID-19. Telehealth tools include simple patient health portal messages to relieve triage phone lines, e-consultation and telephone or video-based virtual visits.

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The latest data from the Victorian COVID-19 Cancer Networks Telehealth Expert Group found that all of Victorias health services were now using telehealth for cancer care. And these tools may continue to expand, allowing greater access to supportive care beyond this crisis.

However, as useful as it is, this technology may pose issues of trust, isolation, disconnectedness and worries about abandonment as patients no longer benefit from the reassuring structure of the hospital oncology setting.

Telehealth may also not be possible for patients without a computer or internet access, which may create inequities in psychosocial care.

These issues should be considered if we upscale telehealth procedures for cancer patients and want to keep the best changes after the pandemic.

With respect to COVID-19, people with blood cancer are a particularly vulnerable group and require specialised attention and care. This is because blood cancers affect the production and functions of cells created in the bone marrow, which produces all the cells of our immune system.

Additionally, treatment for blood cancers compromise the immune system further, placing patients at very high risk of opportunistic infections, often for an extended period.

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Even common respiratory viruses, like colds, can threaten survival. As a result, blood cancer specialists have recommended heightened surveillance and protective isolation for people with blood cancers.

A second major issue for blood cancer patients during the COVID-19 pandemic is the availability of donor stem cell products for patients undergoing haematopoietic stem cell transplantation.

Approximately 350 stem cell transplants are performed in Australia every year to treat blood cancers like leukaemia and multiple myeloma.

But finding a suitable stem cell donor is incredibly challenging, and about 80 per cent of donated stem cells come from overseas donors.

Unfortunately, COVID-19 has impacted donor availability due to border restrictions, flight changes and fear of becoming infected, creating very fearful scenarios for those waiting for potentially life-saving stem cell transplantation.

Victoria has already reported a case of a donor cancellation at short notice because the donor was worried about having been in contact with somebody with COVID-19.

The emotional consequences of the COVID-19 pandemic for people with blood cancers cannot be underestimated, nor can the consequences for the healthcare providers who care for them.

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The COVID-19 pandemic has had a serious and disruptive effect.

In the short term, staff and resources at many hospitals have been reassigned to manage the rush of patients with COVID-19. Healthcare providers are faced with ethical dilemmas and required to make difficult treatment decisions without evidence-based guidelines.

Estimating the risk versus the benefit of administering potentially immunosuppressive treatment to patients with haematological cancers with a scarcity of knowledge about this novel disease, and balancing the individual and societal benefits with stretched resources, poses acute ethical dilemmas.

Making these challenging decisions can create fertile grounds for burnout and trauma.

More than ever, we need to develop ways to nurture healthcare providers.

Consideration of their unique experiences and needs during these exceptionally difficult times is important and we need to flexibly develop services to respond to those needs now and beyond this crisis.

But in developing such services, as well as recognising unmet needs, we have to acknowledge the problem-solving skills, resourcefulness and innovation that patients and healthcare providers have already undertaken as they have strived to adapt to managing cancer during COVID-19.

Banner: Getty Images

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Children’s Hospital Los Angeles Ranked No. 1 Children’s Hospital in the Western U.S., No. 5 Nationally for Second Straight Year – Newswise

By daniellenierenberg

Newswise LOS ANGELES (June 16, 2020) Children's Hospital Los Angeles (CHLA) ranks again among the nations premier destinations for pediatric care, according to the U.S. News & World Report Best Children's Hospitals annual list released today.

CHLA not only retained its national No. 5 ranking in U.S. News Honor Roll of Best Childrens Hospitalswhich recognizes institutions with the most outstanding pediatric clinical careit continued its four-year streak of being the highest-scoring childrens hospital in the entire Western United States.

To make U.S. News & World Reports prestigious Best Childrens Hospitals Honor Roll, one must demonstrate the strongest achievements in clinical excellence, with a matchless team of expert, compassionate specialists committed to research and education as well as protocols that drive safety and quality and consistently lead to the best health outcomes for patients, says CHLA President and Chief Executive Officer Paul S. Viviano. "This honor affirms the work of every CHLA team member and our belief that when parents choose Childrens Hospital Los Angeles, they are choosing the best care for kids."

Every year, U.S. News scores nearly every major hospital and health system in the country and ranks them according to performance benchmarks, peer review, certifications, and other data provided by the hospital and third-party measurements of excellence. Children's hospitals are ranked separately from other facilities due to the specialized expertise, equipment and facilities required to care for infants, children and youth.

This year, U.S. News surveyed 118 pediatric medical centers, including hospitals that are freestanding or part of a larger institution. CHLA improved its ranking over last year in seven of the 10 pediatric specialty categories the survey considers, including a number two ranking for Neonatal Care. In all, the hospital earned top-10 recognition in seven of those categories:

CHLA has an organization-wide commitment to providing our patients the care they need no matter their circumstances, says CHLA Chief Medical Officer James Stein, M.D., MSc. "Our clinical teams often treat the most acute cases that are outside the scope or expertise of other childrens hospitals in California, and being named a Top-5 childrens hospital in the U.S. is a testament to the clinicians and staff who work every day to make sure each child receives the best care and experience possible.

Founded in 1901, Children's Hospital Los Angeles is a pediatric academic medical center built around its mission of creating hope and building healthier futures for children. Renowned for its world-class clinical care, leading-edge research and one of the largest and most successful pediatric training programs in the countryall while being the pediatric safety net hospital for the entire regionCHLA now sees more than 600,000 patient visits annually between its main hospital and five neighborhood care clinics.

CHLA physicians, nurses and clinical staff provide compassionate and lifesaving pediatric care for patients ranging from infants to young adults, hailing from all 50 states and more than 75 countries. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC. Many of the hospital's achievements in care are made possible through a cohesive relationship between clinical experts at the bedside and the basic, translational, and clinical research conducted in The Saban Research Institute of CHLA.

In the past year, CHLA has had several notable achievements, including:

U.S. News and World Report works with research firm RTI International to develop its annual Best Children's Hospitals list, a collaboration between hospitals and the magazine to benchmark the performance of childrens hospitals for the benefit of parents and their children. The survey evaluates hundreds of data points, including patient survival and surgical complication rates; staffing, technology and special services; infection prevention and delivery of care; reputation among peer physicians nationwide (i.e. Where would the best pediatric specialists send their kids?); how involved parents are in their childrens care; and many other measurements of excellence.

U.S. News Media Group, the parent of U.S. News & World Report, announced the 2020-21 hospital rankings online at 12:01 a.m. EST on Tuesday, June 16. For additional information, please visit the Best Childrens Hospitals Honor Roll and specialty rankings page at usnews.com/childrenshospitals.

About Children's Hospital Los Angeles Founded in 1901,Children's Hospital Los Angelesis ranked the topchildrens hospital in California andfifth in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll of childrens hospitals. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC through an affiliation dating from 1932.The hospitalalso leads thelargest pediatric residency training program at a freestanding childrens hospital of its kind in the western United States.TheSaban Research Instituteof Childrens Hospital Los Angeles encompasses basic, translational and clinical research conducted at CHLA. The hospitals Global Health Program facilitates services for international patients from more than 75 countries. To learn more, follow us on Facebook, Instagram, LinkedIn and Twitter, and visit our blog for families (CHLA.org/blog) andour research blog (ResearCHLABlog.org).

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Plinabulin Combo May Be Superior Therapy for Prevention of Chemotherapy-Induced Neutropenia – Targeted Oncology

By daniellenierenberg

Treatment with plinabulin in combination with pegfilgrastim (Neulasta) achieved the primary end point and several key secondary end points in the phase 3 PROTECTIVE (Study 106) clinical trial, which evaluated the combination compared with pegfilgrastim alone for superiority in chemotherapy-induced neutropenia prevention, according to the interim results announced by BeyondSpring, developer of the drug.

These results demonstrate significant enhancement to granulocyte colony-stimulating factors (G-CSFs) with the combination compared with pegfilgrastim alone for the prevention of grade 4 or severe neutropenia, supporting the potential for superiority with the combination compared with pegfilgrastim alone.

These interim results from the PROTECTIVE-2 Phase 3 study, which compares the Plinabulin-Neulasta combination to Neulasta alone, have the potential to be clinically meaningful for cancer patients receiving chemotherapy, stated Douglas W. Blayney, MD, professor of Medicine at Stanford Medical School and global principal investigator of Plinabulins chemotherapy-induced neutropenia studies. Since most infections, hospitalizations, and other complications of chemotherapy-induced neutropenia occur in the first week after chemotherapy, it is particularly gratifying to see the combinations clinical benefit demonstrated.

The primary end point was the rare of severe neutropenia prevention in cycle 1 (P <.01). The key secondary end points included duration of severe neutropenia in cycle 1 (P <.05) and duration of severe neutropenia in the first 8 days of cycle 1 (P <.05). The achievement of these end points demonstrates the agents ability to provide early protection against severe neutropenia induced by chemotherapy.

The interim analysis of the double-blind, active-controlled, global PROTECTIVE-2 study was pre-specified for the first 120 patients accrued. The study also has procedures in place for the prevention of potential bias after the planned interim analysis, and BeyondSpring opted to be informed by independent statisticians on whether the pre-specified P values were met rather than the exact P values.

The study was designed to assess both the safety and efficacy of plinabulin in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide (TAC) in a 21-day cycle. Plinabulin was administered in a 40 mg dose on day 1 with a 6 mg dose of pegfilgrastim on day 2, while pegfilgrastim was administered at the same 6 mg dose on day 2 in the control arm. TAC is considered a high-risk chemotherapy regimen associated with neutropenia.

PROTECTIVE-2 is a superiority study for chemotherapy-induced neutropenia efficacy compared with pegfilgrastim alone and is currently enrolling patients. Grade 4 neutropenia rate for TAC and pegfilgrastim is observed in 83% to 93% of patients, which represents a severe unmet medical need.

The study drug and G-CSF have complementary mechanisms of action for preventing chemotherapy-induced neutropenia. Plinabulin is a first-in-class differentiated immune and stem cell modulator, which is currently in late-stage clinical development as a potential treatment for increasing overall survival in patients with cancer in addition to its ability to alleviate chemotherapy-induced neutropenia.

Plinabulin is also under evaluation in a phase 3 study as a direct anticancer drug as treatment of patients with nonsmall cell lung cancer and 2 phase 3 studies for the prevention of chemotherapy-induced neutropenia. The data for this agent in preventing chemotherapy-induced neutropenia highlights its ability to boost the number of hematopoietic stem or progenitor cells (HSPcs), as well as lineage-/cKIT+/Sca1+ cells in mice. Plinabulins effects on HSPcs may explain its ability to treat chemotherapy-induced neutropenia as well as its ability to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34-positive cells.

To be included in the study, patients should be candidates for TAC in either the adjuvant or neoadjuvant setting, have early-stage breast cancer, and have received no prior chemotherapy. Patients must also have an ECOG performance status of 0 or 1 and a life expectancy of at least 3 months. Patients cannot be included in the study if they have a history of myelogenous leukemia, myelodysplastic syndrome, or sickle cell disease; use of CYP3A4, CYP2D6 or P-glycoprotein inhibitors and inducers within 14 days prior to receiving the study drug; or have received an investigational agent or tumor vaccine within 2 weeks, any concurrent anticancer therapies, or a bone marrow or stem cell transplantation.

These results could help to confirm the patient benefit of Plinabulins different mechanism of action from the G-CSF-based agents, such as Neulasta, Blayney stated. Plinabulin appears to have chemotherapy-induced neutropenia protection in Week 1, and G-CSFs have protection in Week 2 of chemotherapy cycles. The combination should logically provide significantly better protection than Neulasta alone as shown in the interim readout. We are well on our way to confirming that the combination offers protection throughout the chemotherapy cycle, which is an unmet medical need.

Reference

BeyondSpring Announces Positive Topline Interim Results from PROTECTIVE-2 (Study 106) Phase 3 Trial Evaluating Superiority of Plinabulin in Combination with Neulasta for Chemotherapy-Induced Neutropenia Prevention. NewsRelease. BeyondSpring. June 15, 2020. Accessed June 15, 2020. https://bit.ly/2YDJbiQ

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2025 Projection: Thalassemia Treatment Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2026 – Cole of Duty

By daniellenierenberg

COVID-19 impact will also be included and considered for forecast.

Global Thalassemia Treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Thalassemia Treatment Market Size Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

>>Need a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=57&RequestType=Sample

Global Thalassemia Treatment Market 2018-2024Brandessence Market Research is working on a new report titleGlobal Thalassemia Treatment Market: Global Size, Trends, Competitive, Historical & Forecast Analysis, 2018-2024?. Rise in number of altered Thalassemia genes, increase in awareness about the disease and high adoption of chelation therapy & blood transfusion for treatment by doctors as well as patients arelikely to enhance the growth of Global Thalassemia Treatment Market.

Scope of Global Thalassemia Treatment Market Reports

Thalassemia is aninherited blood disorder in which the body makes an abnormal form of hemoglobin.People having Thalassemia disease are unable to make sufficient hemoglobin which causes severe anemic conditions.Hemoglobin is found in red blood cells and transports oxygen to all parts of the body. When there is insufficient hemoglobin in the red blood cells, oxygen cannot get to all parts of the body. Organs demand oxygen and are unable to function properly.There are two primary types of Thalassemia disease such as Alpha Thalassemia disease and Beta Thalassemia disease.Alpha Thalassemia results in a formation of additional beta globins, which leads to the formation of beta-globin tetramers (4) called Hemoglobin H.

Beta Thalassemia causesadditionalformation of alpha globins, which develops alpha globin tetramers (a4) that store in the erythroblast (immature red blood cell).Thalassemia is caused by mutations in the DNA of cells that make hemoglobin.

Factors that increase risk of Thalassemia include Family history of thalassemia and certain ancestry.Possible complications of Thalassemia includeIron overload, Infections, Bone deformities, splenomegaly, slowed growth rate of child and Heart problems.

Thalassemia signs and symptoms include Fatigue, Weakness, Pale or yellowish skin, Facial bone deformities, slow growth, abdominal swelling, Dark urine, chest pain,cold hands and feet, poor feeding, greater susceptibility to infections. Diagnosis of Thalassemia includesa complete blood count (CBC), a reticulocyte count,Iron count, Genetic testing and prenatal testing. Treatment of Thalassemia depends on the type and severity of Thalassemia such as Blood transfusions, Bone marrow or stem cell transplant, Surgery and Gene therapy.

Global Thalassemia Treatment Market has been segmented on the basis ofType of Treatment, Diagnosis, End usersand Geography. On the basis of Type of TreatmentGlobal Thalassemia Treatment Market is classified into Blood Transfusion, Chelating Therapy, Bone Marrow Transplant, Stem Cell Transplant, Surgery, Gene Therapy and Others.On the basis of DiagnosisGlobal Thalassemia Treatment Market is classified into Perinatal Testing, Prenatal Testing, Pre-Implantation and Other.On the basis of the End user the Global Thalassemia Treatment Market is classified into Hospitals, Biotechnological Laboratories, Diagnostic Laboratories, Educational Research Institutes, Pharmaceutical Industries and others.

The regions covered in Global Thalassemia Treatment Market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, Global Melanoma Drug Market sub divided in to U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Thalassemia Treatment Market Reports

Global Thalassemia Treatment Market reports cover prominent players like Bluebird bio Inc., Acceleron Pharma Inc., Novartis AG, Celgene Corporation, Shire plc, Bellicum Pharmaceuticals, GlaxoSmithKline Plc, Celgene,Lonza group, Alnylam Pharmaceuticals Inc., Calimmune Inc., CRISPR Therapeutics, Editas Medicine Inc., Errant Gene Therapeutics LLC, Gamida Cell Ltd, Gilead Sciences Inc., Incyte Corp, Ionis Pharmaceuticals Inc., IRBM Science Park SpA, Johnson & Johnson, Kiadis Pharma NV, La Jolla Pharmaceutical Company, Merck & Co Inc., PharmaEssentia Corp, Protagonist Therapeutics Inc., Sangamo Therapeutics Inc., Zydus Cadila Healthcare Ltd, Genorama Ltd, HiMedia Laboratories, DiagCor Bioscience Inc. Ltd and Tosoh Bioscience Inc.

Global Thalassemia Treatment Market Dynamics

Increase in awareness about the disease and technological expansions are likely to raise the adoption of gene therapies. Also Rising Prevalence of Thalassemia, Increase in Pharmaceutical R&D Spending, Increasing Spending on Stem Cell Research, Rising Healthcare Expenditure and Rising Asian Population will boost theGlobal Thalassemia Treatment Market. Treatment of Thalassemia is mostly restricted to regular blood transfusions and iron chelation therapy.Moreover, High operation cost of sophisticated clinical and preclinical imaging systems, High cost of maintenance andless life span of accessoriesalso restraining theGlobal Thalassemia Treatment Market.Yearlyspending for treatment of Thalassemia ranged from $ 108 to 432, depending on type of treatment with average cost per blood transfusion was $ 5.22.2. Average 18.5%14.3 of the total annual income was spent on the treatment for Thalassemia. Drugs prescribed for Thalassemia mostly cures symptoms and side effects such as anemia, iron overload, slow growth of children and vitamin deficiency.Occurrence of Thalassemia is reported to increase steadily over the years across different regions. This can be due to population migration, intermarriages, genetic as well as environmental factors prompting the condition and its implications.Systematic Drugs under Pipeline, Rising Scope for Gene Therapy and increasing awareness towards Thalassemia are some opportunities in the forecast period for theGlobal Thalassemia Treatment Market.

Global Thalassemia Treatment MarketRegional Analysis

North America have largest share ofGlobal Thalassemia Treatment Market. It is mainly driven by quickly increasing immigrant population from tropical regions, rising number of population with Thalassemia carrier gene and rise in birth rates due to variation of genes among the population in the U.S.There are some prenatal tests available on the market to determine the possibility of alpha thalassemia including both invasive and non-invasive technique.

The alpha thalassemia testing market has aemergent trend in the countries with traditional groups like Mediterranean countries, African countries and few countries in Asia Pacific. Furthermore, in Asia Pacific region the growth in similar community marriage practices and high fertility ratewith alpha thalassemia patients have been detected. This is expected to raiseacceptance of blood transfusion and chelation therapy treatments during the forecast period.A latestimprovement in the testing of alpha Thalassemia may determine the risk of the disease by in vitro examination of the embryo. While there are various such tests available in theGlobal Thalassemia Treatment Market but lack of awareness leads to the neglect and delayed diagnosis of the diseased state.

Most frequently prone area for alpha thalassemia is Mediterranean countries, African countries, and Southeast Asian countries. Thalassemia trait practically affects 6% to 35% of the population in these ethnic groups. Middle East & Africa is likely to be the fastest risingGlobal Thalassemia Treatment Market during the forecast period.

Key Benefits for Global Thalassemia Treatment Market Reports

Global Thalassemia Treatment Market report covers in depth historical and forecast analysis.Global Thalassemia Treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Global market sale (K Units), Global market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.Global Thalassemia Treatment Market report helps to identify opportunities in market place.Global Thalassemia Treatment Market report covers extensive analysis of emerging trends and competitive landscape.Global Thalassemia Treatment Market Segmentation

Global Thalassemia Treatment Market: By Type of Treatment Analysis

Blood TransfusionChelating TherapyBone Marrow TransplantStem Cell TransplantSurgeryGene TherapyOtherGlobal Thalassemia Treatment Market: By Diagnosis Analysis

Perinatal TestingPrenatal TestingPre-ImplantationOtherGlobal Thalassemia Treatment Market: By End user Analysis

HospitalsBiotechnological LaboratoriesDiagnostic LaboratoriesEducational Research InstitutesPharmaceutical IndustriesOtherGlobal Thalassemia Treatment Market: By Regional & Country Analysis

North AmericaU.S.MexicoCanadaEuropeUKFranceGermanyItalyAsia PacificChinaJapanIndiaSoutheast AsiaLatin AmericaBrazilThe Middle East and AfricaGCCAfricaRest of Middle East and AfricaNeed a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=57&RequestType=Methodology

Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the Global Thalassemia Treatment Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide Global Thalassemia Treatment Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the Global Thalassemia Treatment Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Thalassemia Treatment Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Thalassemia Treatment Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the Global Thalassemia Treatment Market.

Market Forecast: Here, the report offers a complete forecast of the Global Thalassemia Treatment Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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2025 Projection: Thalassemia Treatment Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2026 - Cole of Duty

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Race Oncology confirms positive results from Bisantrene drug trial on patients with advanced acute myeloid leukaemia – Small Caps

By daniellenierenberg

Specialty pharmaceutical company Race Oncology (ASX: RAC) has reported positive data from an investigator-initiated Phase II clinical trial of historical cancer drug Bisantrene on patients with relapsed or refractory acute myeloid leukaemia.

Conducted at the Sheba Medical Centre in Israel, the open-label, single-agent trial studied 10 patients who, on average, had failed three prior lines of treatment.

Bisantrene was found to be well tolerated with no unexpected or serious toxicities, the company reported.

After a single course of treatment, the drug demonstrated an overall clinical response rate of 40%, with one patient progressing to complete remission and three achieving partial remission.

One patient was bridged to allogeneic stem cell transplantation (where cells are donated to the patient from a genetically-matched donor) and there were no removals or withdrawals from the study during treatment.

The drug also had marked activity in four patients with extramedullary (outside of the bone marrow) acute myeloid leukaemia such as leukemia cutis, chloromas, and central nervous system disease which has historically been difficult to treat.

The most frequently reported serious adverse events were thrombocytopaenia (low blood platelets) and mucositis (mouth ulcers), both of which were expected side effects of anthracyline and anthracene chemotherapeutics.

One patient experienced transient grade one kidney toxicity and there were no liver toxicities observed.

Race said there were no anaphylactoid-type reactions observed in any patient over the course of treatment. This type of reaction was a serious adverse event regularly observed in the historical trials.

Relapsed or refractory acute myeloid leukaemia remains a significant therapeutic challenge.

While meaningful gains have been achieved in recent years with the introduction of new targeted drugs, published studies claim the clinical outcomes remain unsatisfactory.

Bisantrene is a small molecule cancer drug related to anthracyclines the most widely-used class of chemotherapy drugs.

Unlike anthracyclines, it has a greatly reduced risk of cardiotoxicity (heart damage), meaning it can be used with patients who have reached their cardiotoxic limit with anthracyclines, or cannot tolerate anthracyclines due to existing heart conditions, age or other factors.

Bisantrene was tested in more than 40 phase II clinical trials during the 1980s and 1990s with up to $200 million put into its development, before it was lost in a series of big pharmaceutical mergers.

Race clinical advisory board chairman and international authority in clinical leukaemia and stem cell research, Professor Borje Andersson, said the trial confirmed historical results which used a different formulation of Bisantrene.

While Bisantrene had been demonstrated in the 1980s as an effective salvage drug against acute myeloid leukaemia, the [clinical] data we had was old, he said.

It was important for us to study it by todays standards using the current formulation, so we could confirm whether our strategy of repurposing this drug is sound.

We also wanted to confirm that Bisantrene could still generate a meaningful response rate in a highly-frail patient population with heavily pre-treated acute myeloid leukaemia, Professor Andersson added.

The study saw a reduction in the leukaemic disease burden and an overall response rate in 40% of the patients.

While we must study the drug further, it appears that with this kind of response, Bisantrene-based therapy may have the potential to serve as an important bridge to allogeneic stem cell transplantation in patients who otherwise have few therapeutic options, Professor Andersson said.

Race chief scientific officer Dr Daniel Tillett said a key focus of Phase II clinical trial was to determine Bisantrenes safety in a modern context.

These results are pleasing from a safety and activity perspective, particularly given the clinically-challenging patient population included in the trial, he said.

It was encouraging to see the drugs tolerability profile compared favourably with other commonly-used chemotherapy agents such as anthracyclines, while the side effects were in keeping with what we would expect to see with all chemotherapeutics of this class, Dr Tillett added.

Professor Andersson said the trial found Bisantrene to be an agent with an acceptable safety profile and promising anti-leukaemic activity.

As this was an open-label, single-agent trial, we can be confident that it was the Bisantrene exposure which generated the positive results, he said.

The patient cohort had advanced acute myeloid leukaemia and had previously failed an average of three lines of therapy, so they were always going to be tough to treat, but a 40% overall response rate after only a single course of treatment markedly exceeded our expectations.

It is a hugely promising result and one which reinforces our development plans for this drug, Professor Andersson added.

A follow-up study combining Bisantrene with other anti-leukaemic drugs is currently in the advanced planning stages.

At mid-morning, shares in Race Oncology were up 53.97% to $0.485.

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Hematopoietic Stem Cells Transplantation (HSCT) Market Trends and Forecast Analysis by Business Manufactures and Product Type-Kite Pharma, Thermo…

By daniellenierenberg

The Hematopoietic Stem Cells Transplantation (HSCT) Market research report enhanced worldwide Coronavirus COVID19 impact analysis on the market size (Value, Production and Consumption), splits the breakdown (Data Status 2014-2020 and 6 Year Forecast From 2020 to 2026), by region, manufacturers, type and End User/application. This Hematopoietic Stem Cells Transplantation (HSCT) market report covers the worldwide top manufacturers like (Kite Pharma, Thermo Fisher Scientific, CellGenix Technologie Transfer, Cesca Therapeutics, R&D Systems) which including information such as: Capacity, Production, Price, Sales, Revenue, Shipment, Gross, Gross Profit, Import, Export, Interview Record, Business Distribution etc., these data help the consumer know about the Hematopoietic Stem Cells Transplantation (HSCT) market competitors better. It covers Regional Segment Analysis, Type, Application, Major Manufactures, Hematopoietic Stem Cells Transplantation (HSCT) Industry Chain Analysis, Competitive Insights and Macroeconomic Analysis.

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Hematopoietic Stem Cells Transplantation (HSCT) Market report offers comprehensive assessment of 1) Executive Summary, 2) Market Overview, 3) Key Market Trends, 4) Key Success Factors, 5) Hematopoietic Stem Cells Transplantation (HSCT) Market Demand/Consumption (Value or Size in US$ Mn) Analysis, 6) Hematopoietic Stem Cells Transplantation (HSCT) Market Background, 7) Hematopoietic Stem Cells Transplantation (HSCT) industry Analysis & Forecast 20202026 by Type, Application and Region, 8) Hematopoietic Stem Cells Transplantation (HSCT) Market Structure Analysis, 9) Competition Landscape, 10) Company Share and Company Profiles, 11) Assumptions and Acronyms and, 12) Research Methodology etc.

Scope of Hematopoietic Stem Cells Transplantation (HSCT) Market:Hematopoietic stem cell transplants (HSCT) present to a valid treatment for several congenital and other hematopoietic system disorders, post chemotherapy, and immune sensitive diseases. HSCT is also preferred for replacement of cellular components and deficient cells. The indications for HSCT thus are wide; the most frequent indication as per reported by Worldwide Network for Blood and Marrow Transplantation Group (WNBT) (2013) is lymphoproliferative disorder (53.2% of all HSCT), 12% of whom received allogeneic and the rest received autologous transplant. Plasma cell disorders are the most frequent indication in this group. A multitude of literature published by researchers and organizations demonstrate that autologous transplant own a greater edge against allogeneic HSCT.

Over 30 years of studies in the field of blood-forming stem cells i.e. hematopoietic stem cells (HSC), researchers have developed significant understanding to use HSCs as a therapy. At present, no type of stem cell, adult, embryonic or fetal has attained such sufficient status. Hematopoietic stem cell transplantation (HSCT) is now routinely used for treating patients with malignant and non-malignant disorders of blood and the immune system. Currently, researchers have observed that through animal studies HSCs have the ability to form other cells such as blood vessels, muscles, and bone. Further application of this approach it may eventually be able to treat a wide array of conditions and replace ailing tissues. However, despite the vast experience with HSCs, researchers face major barriers in expanding their use beyond the replacement of immune and blood cells.

Hematopoietic stem cells are unable to proliferate and differentiate in-vitro. Researchers have yet to evolve an accurate method to differentiate stem cells from other cells derived from blood or bone marrow. Once such technical barriers are overcome, the avenues for realizing the full potential of HSCT. The type of transplant a person receives depends on several different factors, including the type and course of the disease, availability of suitable donors, and the patients overall health. There are three different sources of hematopoietic stem cells such as bone marrow, peripheral blood stem cells, and umbilical cord blood. The stem cell source used for a given transplant depends upon the underlying disease, the type of transplant (allogeneic or autologous), and size of the patient.

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, shipments, revenue (Million USD), price, and market share and growth rate foreach application.

Leukemia Lymphoproliferative Disorders Solid Tumors Non-Malignant Disorders Others

On the basis of product type, this report displays the shipments, revenue (Million USD), price, and market share and growth rate of each type.

Autologous Transplant Allogenic Transplant

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Geographically, the report includes the research on production, consumption, revenue, Hematopoietic Stem Cells Transplantation (HSCT) market share and growth rate, and forecast (2020-2026) of the following regions:

Important Hematopoietic Stem Cells Transplantation (HSCT) Market Data Available In This Report:

Strategic Recommendations, Forecast Growth Areasof the Hematopoietic Stem Cells Transplantation (HSCT) Market.

Challengesfor the New Entrants,TrendsMarketDrivers.

Emerging Opportunities,Competitive Landscape,Revenue Shareof Main Manufacturers.

This Report Discusses the Hematopoietic Stem Cells Transplantation (HSCT) MarketSummary; MarketScopeGives A BriefOutlineof theHematopoietic Stem Cells Transplantation (HSCT) Market.

Key Performing Regions (APAC, EMEA, Americas) Along With Their Major Countries Are Detailed In This Report.

Company Profiles, Product Analysis,Marketing Strategies, Emerging Market Segments and Comprehensive Analysis of Hematopoietic Stem Cells Transplantation (HSCT) Market.

Hematopoietic Stem Cells Transplantation (HSCT) Market ShareYear-Over-Year Growthof Key Players in Promising Regions.

What is the (North America, South America, Europe, Africa, Middle East, Asia, China, Japan)production, production value, consumption, consumption value, import and exportof Hematopoietic Stem Cells Transplantation (HSCT) market?

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Cell Therapy Manufacturing Market: Regional Analysis and Strategies in Forecasted Period 2020-2030 – 3rd Watch News

By daniellenierenberg

Prophecy Market Insights has recently published a Cell Therapy Manufacturing report which represents the latest industry data and future trends, allowing users to recognize the products and driving revenue growth and profitability of the market.

The report offers a broad analysis of key segments, key drivers, regions, and leading market players. The report contains an analysis of different geographical areas and presents a competitive scenario to promote leading market players, new entrants, and investors determine emerging economies. The key highlights offered in the report would benefit market players to formulate strategies for the future and gain a strong position in the Cell Therapy Manufacturing market.

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The Cell Therapy Manufacturing report begins with a brief introduction which contains a market overview of the industry followed by its market size and research scope. Further, the report provides an overview of market segmentation, for example- type, application, and region. The drivers, restraints, and opportunities for the market are also mentioned, along with current policies and trends in the industry. The Cell Therapy Manufacturing market also covers PEST analysis for the market. Thisanalysisprovides information based on four external factors (political, economic, social and technological) in relation to your business situation. Basically, it helps to understand how these factorswillaffect the performance and activities of your business in the long-term. The report describes the growth rate of each segment in-depth with the help of charts and tables. Moreover, various regions related to the growth of the Cell Therapy Manufacturing market are analyzed in the report. These regions include North America, Europe, Asia-Pacific, Middle East and Africa, and Latin America.

Segmentation Overview:

Cell Therapy Manufacturing market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry. In this research report, there is an accurate analysis of the current and upcoming opportunities in the market by explaining the fastest and largest growing segments across regions. The survey report includes vast investigation of the geographical scene of the Cell Therapy Manufacturing market, which is manifestly arranged into the localities

Australia, New Zealand, Rest of Asia-Pacific

The study presents the performance of each player active in the Cell Therapy Manufacturing market. It also provides a summary and highlights the current advancements of each player in the market along with its SWOT analysis. The information provided in the research report is a great source for study investors and stakeholders interested in the market. In addition, the report offers insights on buyers, suppliers, and merchants in the market. There is a comprehensive analysis of consumption, market share, and growth rate of each application is offered for the historic period.

Cell Therapy ManufacturingMarket Key Players:

harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

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Combined irradiation and chemotherapy better prepares children for stem cell transplantation than chemotherapy alone – DOTmed HealthCare Business News

By daniellenierenberg

THE HAGUE, Netherlands, June 12, 2020 /PRNewswire/ -- Treatment of childhood cancer is a success story, particularly for acute lymphoblastic leukemia (ALL). More than 90% of ALL patients below 18 years of age are rescued with contemporary chemotherapy. However, the remaining 10% have resistant or reoccurring leukemia and require alternative treatment regimens. One of the most powerful leukemia therapies is hematopoietic stem cell transplantation from a donor (allogeneic HSCT). Approximately 50-80% of pediatric ALL patients that receive allogeneic HSCT are cured, 20% experience leukemic reoccurrence (relapse), and 10% die from complications.

Allogeneic HSCT is a multistep procedure:

Identify a suitable donor, i.e., a compatible sibling or unrelated person.

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Therefore, a large consortium of pediatric transplant experts initiated a global study to investigate whether chemotherapy-based conditioning could substitute TBI. The study is called FORUM (For Omitting Radiation Under Majority Age) and had to be stopped because chemotherapy-based conditioning had significantly poorer outcomes (i.e., lower overall survival rates) than the combination of TBI and chemotherapy. The researchers will now perform prospective monitoring to better define the advantages and limitations of various conditioning approaches.

Presenter: Dr Christina PetersAffiliation: Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, AustriaAbstract: #S102 TBI OR CHEMOTHERAPY BASED CONDITIONING FOR CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY "FORUM" ON BEHALF OF THE AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP

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Stem Cell Banking Market will Generate Massive Revenue to $6,956 million by 2023 | Cord Blood Registry, ViaCord, Cryo-Cell, China Cord Blood…

By daniellenierenberg

The global stem cell banking market was valued at $1,986 million in 2016, and is estimated to reach $6,956 million by 2023, registering a CAGR of 19.5% from 2017 to 2023. Stem cell banking is a process where the stem cell care isolated from different sources such as umbilical cord and bone marrow that is stored and preserved for future use. These cells can be cryo-frozen and stored for decades. Private and public banks are different types of banks available to store stem cells.

Top Companies Covered in this Report: Cord Blood Registry, ViaCord, Cryo-Cell, China Cord Blood Corporation, Cryo-Save, New York Cord Blood Program, CordVida, Americord, CryoHoldco, Vita34

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Increase in R&D activities in regards with applications of stem cells and increase in prevalence of fatal chronic diseases majorly drive the growth of the global stem cell banking market. Moreover, the large number of births occurring globally and growth in GDP & disposable income help increase the number of stem cell units stored, which would help fuel the market growth. However, legal and ethical issues related to stem cell collections and high processing & storage cost are projected to hamper the market growth. The initiative taken by organizations and companies to spread awareness in regards with the benefits of stem cells and untapped market in the developing regions help to open new avenues for the growth of stem cell banking market in the near future.

The global stem cell banking market is segmented based on cell type, bank type, service type, utilization, and region. Based on cell type, the market is classified into umbilical cord stem cells, adult stem cells, and embryonic stem cells. Depending on bank type, it is bifurcated into public and private. By service type, it is categorized into collection & transportation, processing, analysis, and storage. By utilization, it is classified into used and unused. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

CHAPTER 5: STEM CELL BANKING MARKET, BY BANK TYPE

CHAPTER 6: STEM CELL BANKING MARKET, BY SERVICE TYPE

CHAPTER 7: STEM CELL BANKING MARKET, BY UTILIZATION

CHAPTER 8: STEM CELL BANKING MARKET, BY REGION

CHAPTER 9: COMPANY PROFILES

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BrainStorm Receives $500K to Support ALS Study of NurOwn Therapy – ALS News Today

By daniellenierenberg

The ALS Association and I AM ALS have awarded BrainStorm Cell Therapeutics $500,000 to support an amyotrophic lateral sclerosis (ALS) biomarker study based on the biotechnology companys pivotal trial into its NurOwn therapy.

Specifically, the combined grant $400,000 is from the ALS Association will be used to gain insights from data as well as tissue and blood samples collected from patients enrolled in the Phase 3 clinical trial (NCT03280056) that is measuring NurOwns safety and effectiveness.

As part of the award, BrainStorm agreed to share data and samples with the ALS community to potentially advance other ALS research, and to have trial results independently validated.

By assessing how NurOwn interacts with brain and spinal cord targets, the hope is the study produces a deeper understanding of crucial biomarkers associated with treatment response. A biomarker is any measurable body substance that changes over time, and that correlates with treatment response.

If it works the way its intended, the study is expected to help inform scientists broader understanding of ALS biomarkers.

This grant to BrainStorm marks an important step forward in establishing how exactly NurOwn works in the body, Calaneet Balas, ALS Association president and CEO, said in a press release. This research is also important to our overall pursuit of identification and validation of ALS biomarkers. We hope NurOwn is ultimately proven effective in treating ALS, and we stand ready to support BrainStorm in its plan to apply for a biologics license for NurOwn.

NurOwn is a cell-based therapy that usesmesenchymal stem cells (MSCs), which are extracted from a patients own bone marrow. These cells have the ability to generate different cell types. After extraction, MSCs are expanded in the lab and matured into cells that produce high levels ofneurotrophic factors, which are compounds that promote nervous tissue growth and survival. The converted cells are then reintroduced into the body via an injection into muscles or the spinal canal.

A Phase 2 trial (NCT02017912) found NurOwn to be safe and to significantly slow disease progression in a subset of ALS patients with fast-advancing disease. The Phase 3 trial is evaluating the safety and effectiveness of three administrations every two months of NurOwn into the spinal canal, when compared with a placebo.

The Phase 3 trials chief goal is to confirm the effectiveness of NurOwn as measured by the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R), a score of abilities such as swallowing, speech, handwriting, or walking.

Despite the COVID-19 pandemic, patient dosing in the trial is expected to be complete by next month. The study is fully enrolled with 200 participants across six U.S. sites. All patients have been given at least two of three doses. A summary of important findings will be announced later this year.

This critical research study involves one of the largest and most robust clinical trial collectors of [cerebrospinal fluid] biomarkers, said Chaim Lebovits, BrainStorm CEO. Data generated from this study will increase our understanding of how NurOwn therapy impacts ALS disease progression, and may identify patients who benefit the most from this form of therapy. We also hope that this research study will benefit the broader ALS community as we collectively advance toward our shared goal of delivering much-needed treatments, he said.

Danielle Carnival, CEO of I AM ALS, said the ALS community is at a pivotal time in terms of treatment research.

We need to move with urgency in all of our efforts to deliver treatments and cures for ALS, she said. This biomarker research will help us more expeditiously understand the effectiveness of NurOwn, while possibly unlocking discoveries that provide clues for other promising treatments.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone – P&T Community

By daniellenierenberg

THE HAGUE, Netherlands, June 12, 2020 /PRNewswire/ -- Treatment of childhood cancer is a success story, particularly for acute lymphoblastic leukemia (ALL). More than 90% of ALL patients below 18 years of age are rescued with contemporary chemotherapy. However, the remaining 10% have resistant or reoccurring leukemia and require alternative treatment regimens. One of the most powerful leukemia therapies is hematopoietic stem cell transplantation from a donor (allogeneic HSCT). Approximately 50-80% of pediatric ALL patients that receive allogeneic HSCT are cured, 20% experience leukemic reoccurrence (relapse), and 10% die from complications.

Allogeneic HSCT is a multistep procedure:

For high-risk leukemia, the gold standard conditioning procedure is a combination of total body irradiation (TBI) and high dose chemotherapy. This approach is very effective in controlling leukemia in the conditioning step, but patients may experience highly negative consequences of this procedure later in life: sterility, growth retardation, lung problems, and secondary cancer.

Therefore, a large consortium of pediatric transplant experts initiated a global study to investigate whether chemotherapy-based conditioning could substitute TBI. The study is called FORUM (For Omitting Radiation Under Majority Age) and had to be stopped because chemotherapy-based conditioning had significantly poorer outcomes (i.e., lower overall survival rates) than the combination of TBI and chemotherapy. The researchers will now perform prospective monitoring to better define the advantages and limitations of various conditioning approaches.

Presenter:Dr Christina PetersAffiliation:Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, AustriaAbstract:#S102 TBI OR CHEMOTHERAPY BASED CONDITIONING FOR CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY "FORUM" ON BEHALF OF THE AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP

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Westerleigh resident is alive because of stem cell therapy by his doctor — for free. Heres his story. – SILive.com

By daniellenierenberg

Stephen Raffone had difficulty breathing. He coughed up sputum and was wheezing. Doctors told him he had chronic obstructive pulmonary disease (COPD), a condition that causes blocked airflow from the lungs.

As a result, he was being treated for stage 4 COPD.

His doctor was also treating him for cellulitis, an inflammatory and painful bacterial skin infection where extremities appear red and swollen and the area can feel hot and tender to the touch, as well as poor circulation.

My legs were beginning to get ulcerated and they were breaking down, said Raffone.

He was administered the Roman Catholic Churchs Last Rites three times several years ago when he was a patient in Richmond University Medical Center.

Raffone, who is now 63, was in need of a lung transplant.

He was a heavy smoker and it took its toll. However, because he was in a weakened state due to other serious health conditions, doctors told him hed never survive the surgery.

The Westerleigh resident, who has been in need of 24-hour care for the last several years, requires the assistance of two nurses who rotate 12-hour shifts.

One, a close family friend, suggested Raffone see a medical specialist who performs stem cell therapy, a procedure where the patients own stem cells are removed, treated and returned to his or her own body after a conditioning regimen.

She contacted Dr. Alexandre M. Scheer of Scheer Medical Wellness and he agreed to see Raffone.

Dr. Alexandre M. Scheer (Courtesy/Stephen Raffone)Staten Island Advance

But since Raffone was unable to leave his home, Scheer visited Raffone for a consultation and to evaluate his condition.

Fast forward a year and a half and Scheer has continued with those visits almost every Saturday free of charge also underwriting the cost for treatments, as well as Uber rides from Manhattan to Staten Island, in order to perform the stem cell procedure.

RAFFONES NURSE SPEAKS

One of Raffones nurses recounted Raffones journey.

She explained that when they started to explore stem cell therapy she placed calls to several doctors, but the biggest thing that jumped out at her was the astronomical cost.

But there was something about Dr. Scheer. And I just knew he was the right one, said the registered nurse for more than 30 years. "He wasnt interested in money. His goal is his patients outcome. Stephen did pay for the first set of treatments, but since then, Dr. Scheer has not taken a dime.

When the patient began treatments, the first therapy was a tremendous boost and then every week after that he was treated for seven weeks. In the beginning, the doctor visited every week and brought whatever supplies was needed. The PRP (platelet rich plasma) treatments are daily.

I draw the blood, I spin the blood," she said. We have a small centrifuge here so it separates the blood. The PRP is given by a nebulizer. It takes about 30 minutes. And once a week he gets a protein enriched plasma, which takes about a half hour, she added.

He has chronic venous ulcerations of the both lower extremities from the knee down, she said.

Raffone has end stage COPD. But since he started the treatments, hes gone to the hospital only once. And he has tested negative for antibody COVID-19.

RAFFONES TREATMENT BEGINS

Raffone was required to install the centrifuge machine with needles and plasma tube, a laboratory device used for the separation of fluids, gas or liquid, based on density. Separation is achieved by spinning a vessel containing material at high speed.

Initially, Dr. Scheer sent a plastic surgeon to my home to perform liposuction, a type of fat-removal procedure used in plastic surgery, where they separate the fat and preserve the stem cells, Raffone said. They did this four times weekly at the beginning. Dr. Scheer has been visiting my home pretty much each week since Sept. 22, 2018. But right now the stem cell therapy is done once a month."

They draw blood out and spin it. Its all done through IV. Right now stem cell infusion is done once a month and daily through a nebulizer. Dr. Scheer does it on Saturday and my nurse and dear friend to Dr. Scheer does it during the week. My house looks like a hospital. Dr. Scheer is keeping me alive and everything is healing up so well, said Raffone.

Stephen Raffone's left leg before stem cell treatment. (Courtesy/Stephen Raffone)Staten Island Advance

Raffone says he wanted to come forward with his account at this time because hes so grateful and especially today when so many negative stories are in the news.

We need some good stories. There are very few people like Dr. Scheer, especially now during the COVID-19 crisis, he said.

My nurse draws the blood and puts it in a centrifuge when the doctor cant make it from the city. But Dr. Scheer is still coming to my house in spite of the COVID-19 crisis," Raffone continued.

Raffone has been confined to a bed one that he says turns you from side to side and upside down. But Dr. Scheer is confident that when restrictions are lifted and physical therapy sessions resume, Raffone will be able to walk.

The stem cell therapy is not only helping to combat Raffones COPD, but it has also helped him with cellulitis on his leg.

Stephen's Raffone healed left leg after stem cell therapy. (Courtesy/Stephen Raffone)Staten Island Advance

Scheer, a staunch supporter of stem cell therapy, has a background in neurosurgery and regenerative medicine. He performs surgery at several surgical centers in Manhattan.

It has to do with the amount of cells your bone marrow," he said. What we do is . . . saturate the body with stem cells. It suppresses the inflammatory response. COVID-19 also is an inflammatory disease. The COVID-19 kills the lungs. So you dont have oxygen going through. The stem cells protect, so you have continual oxygen transfer.

Dr. Scheer, who practices at Sheer Medical Wellness in Manhattan, says you can regenerate yourself.

I want my patients to be fine. I will pay for the patient. Im happy Stephens alive. And then my life is made. Stephen will now be able to walk after physical therapy. He was on 12 liters of oxygen daily. Hes now on two liters. I know his nurse very well and thats how we connected. The stem cell treatment is the appropriate treatment for him. I pay out of pocket because I know the right treatment for his condition," he added.

Dr. Scheer points out in China and in Israel stem cell therapy is the treatment they use for COVID- 19.

Its where you take Eastern and Western medicine and put it together. The patients body and will to live and having the right outlook on life has a lot to do with proper health. Our group is so big. We have 40 different doctors in my practice. Im the medical director, he said. Stem cell treatment is the future of medicine. At $10,000 a treatment, its very expensive. And the number depends on the issue at hand.

THE INITIAL CALL

When Scheer spoke to Raffone, He said I cant get out of bed,' the doctor said. "I drove to Staten Island and I got to know Stephen and his family very well. Its not a one-time treatment. Im seeing him on a weekly basis. There is a relationship that occurs. And thats what matters and thats what keeps people alive. Hope is what keeps them alive. And Im doing this since 2001. The treatment involves platelet enriched plasma that suppresses inflammatory reactions in the lungs. Whats happening is youre able to suppress the inflammatory reaction. His legs and his heart are getting better as well. This is a treatment until we can get him walking.

Scheer says Raffone must undergo physical therapy in oder for him to walk around freely.

And hell be able to travel to my office. Im not giving up on him. Im paying out of pocket. A quarter of my patients, I pay for. Stephen has gone through so much. Hes alive because of stem cell therapy. And due to his lung condition with COVID, he has not contracted it."

Scheer says its been a team effort, with multiple doctors coming into play.

Stephen is keeping himself alive. Im just the tool that can help. I just do the best I can for as many people as I can.

Originally posted here:
Westerleigh resident is alive because of stem cell therapy by his doctor -- for free. Heres his story. - SILive.com

To Read More: Westerleigh resident is alive because of stem cell therapy by his doctor — for free. Heres his story. – SILive.com
categoriaBone Marrow Stem Cells commentoComments Off on Westerleigh resident is alive because of stem cell therapy by his doctor — for free. Heres his story. – SILive.com | dataJune 13th, 2020
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