As per new Study on Stem Cell Therapy Market 2019 Future Strategy, Analysis and Prediction by Leading Manufacturers, its Application and Types…

A new business intelligence report released by Garner Insights with title Global Stem Cell Therapy Market Research Report 2019 that targets and provides comprehensive market analysis with prospects to 2024. The analysts of the study have garnered extensive research methodologies and data sources (i.e. Secondary & Primary Sources) in order to generate collective and useful information that delivers latest market undercurrents and industry trends.

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

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Some of key competitors or manufacturers included in the study are: Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCRPharmaceutical, Pharmicell, Medi-post, Anterogen, Molmed, Takeda (TiGenix),

If you are involved in the Global Stem Cell Therapy industry or intend to be, then this study will provide you comprehensive outlook. Its vital you keep your market knowledge up to date segmented by major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports, we can provide customization according to your requirement.

Market Segment by Type, covers: Autologous, Allogeneic

Market Segment by Applications, can be divided into: Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, Others,

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

What are the affecting elements that are made reference to in the report?

Market Scenario:The report further highlights the development trends in the global Stem Cell Therapy market. Factors that are driving the market growth and fueling its segments are also analyzed in the report. The report also highlights on its applications, types, deployments, components, developments of this market.

Key Market Highlights:The Stem Cell Therapy report gives a top to bottom examination on a portion of the key elements, involving income, cost, limit, limit usage rate, creation, generation rate, utilization, import/send out, supply/request, net, piece of the pie, CAGR, and gross edge. Furthermore, the report shows a far reaching investigation of the market development factors and their most recent patterns, alongside important market fragments and sub-portions.

Analytical Tools:The Global Stem Cell Therapy Market report incorporates the decisively examined and assessed information of the significant market members and their market scope utilizing various investigative devices. The diagnostic apparatuses incorporate Porters five powers examination, SWOT investigation, achievability study, and venture return investigation, which have been utilized to consider the development of the key players working in the market.

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Some of the Points cover in Global Stem Cell Therapy Market Research Report is:

Chapter 1: Overview of Global Stem Cell Therapy Market (2019-2024) Definition Specifications Classification Applications Regions

Chapter 2: Market Competition by Players/Suppliers 2019 and 2024 Manufacturing Cost Structure Raw Material and Suppliers Manufacturing Process Industry Chain Structure. Continued

The main points which are answered and covered in this Report are-

What will be the total Stem Cell Therapy Market in the coming years till 2024?What will be the key factors which will be overall affecting the industry?What are the various challenges addressed?Which are the major companies included?

Thank You For Visiting Our Report : you can likewise get singular part astute segment or locale insightful report form like Asia, United States, Europe.

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As per new Study on Stem Cell Therapy Market 2019 Future Strategy, Analysis and Prediction by Leading Manufacturers, its Application and Types...

Stem Cell Therapy Market Size, Share, Trends, and Opportunity Analysis by 2017 – 2025 – The Market Expedition

Global Stem Cell Therapy Market Analysis

The recent report published by TMRR on the global Stem Cell Therapy market is an in-depth analysis of the overall prospects of the Stem Cell Therapy market in the upcoming years. The data collected from credible primary and secondary sources is accurately represented in the report backed up by relevant figures, graphs, and tables. The report includes a quantitative and qualitative analysis of the various aspects of the market by collecting data from the key participants in the Stem Cell Therapy market value chain.

The report reveals that the global Stem Cell Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and surpass the value of ~US$XX by the end of 2029. The presented study also includes a thorough analysis of the micro and macroeconomic factors, regulatory framework, and current trends that are expected to influence the growth of the Stem Cell Therapy market during the assessment period.

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Vital Information Enclosed in the Stem Cell Therapy Market Report:

Important Queries Addressed in the Report

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Stem Cell Therapy Market Segmentation

The market study put forward by TMRR segments the global Stem Cell Therapy market to offer a microscopic understanding of the various aspects of the Stem Cell Therapy market. The Stem Cell Therapy market is segmented on the basis of region, product type, end-user, and more.

The study offers a Y-o-Y growth projection of each market segment and sub-segment over the stipulated timeframe of the study.

Key Trends

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

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

Global Stem Cell Therapy Market: Market Potential

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

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

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

Global Stem Cell Therapy Market: Regional Outlook

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

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

Global Stem Cell Therapy Market: Competitive Analysis

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

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

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Stem Cell Therapy Market Size, Share, Trends, and Opportunity Analysis by 2017 - 2025 - The Market Expedition

Newly approved sickle cell drug ushers in an era of therapeutic breakthroughs – Worcester Telegram

Terri Booker's worst sickle cell crisis almost killed her.

Her sticky, stiff, crescent-shaped red blood cells formed a blockage in her bloodstream, starving her of oxygen, triggering unbearable pain, and sending her, at 20 years old, into kidney and lung failure.

Now 36, the Philadelphia lawyer is thrilled by the U.S. Food and Drug Administration's recent approval of Novartis' sickle cell drug Adakveo, a monthly intravenous infusion for patients over age 15 that has been shown to dramatically reduce vessel-obstruction crises and hospitalizations.

The new drug, which targets a specific protein involved in cell stickiness, is part of a wave of novel therapies that could transform the outlook for patients with the devastating illness, the most common inherited blood disorder. In February, the FDA is expected to approve Global Blood Therapeutics' daily pill Voxelotor, which treats the underlying cause by helping to prevent red blood cells from deforming.

More than 30 other therapies are in development, including some that may offer a cure by repairing the genetic mutation that hinders hemoglobin's ability to carry oxygen the defect that causes red blood cells to sickle. Just last month, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the first treatment made with genetically edited cells is producing a crucial protein in the inaugural sickle cell patient, four months after her infusion.

"I'm actually excited for the future of sickle cell treatment," Booker said. "Not only treatments but cures are in the pipeline."

That optimism was echoed by Alexis A. Thompson, head of hematology at Lurie Children's Hospital of Chicago.

"For such a challenging disease, we have had so few tools to treat it," said Thompson, past president of the American Society of Hematology. Adakveo "is terribly exciting. It represents not just an additional tool, but one that was designed specifically for sickle cell disease. These new agents moving toward approval address the disease process in a rational way."

An estimated 100,000 Americans, most of them African American, have sickle cell disease. Worldwide, millions are affected.

For decades, doctors could give them nothing more than symptom relief using blood transfusions, painkilling opioids, and hydroxyurea, a drug that increases the hemoglobin in red blood cells. Developed as a cancer drug, hydroxyurea was approved for sickle cell disease in 1998. Almost two decades passed before a second drug, Endari, an amino acid that reduces oxidative stress on deformed cells, was approved in 2016.

Despite limited treatments, improvements in medical care have lengthened patients' lifespan to about 50 years.

"This used to be a pediatric disease because patients didn't survive into adulthood," said David J. Axelrod, the physician who co-directs the sickle cell program at Jefferson University Hospitals. "Now it's more of a chronic disease, so patients are dying from end-stage organ disease."

Living with the disorder is a constant struggle. Zemoria Brandon sees it every day as an administrator and social worker with the Philadelphia-Delaware chapter of the Sickle Cell Disease Association of America, cofounded by her husband, who died in 1998.

"We have children on morphine to relieve their pain," Brandon said. "We've had children who have had strokes."

Like others, Brandon is happy about Adakveo, which cut the rate of obstructed-vessel crises by nearly half to 1.63 episodes a year, compared with 2.98 a year for patients on a placebo.

"But," Brandon added, "we're hoping that a universal cure is on the horizon."

The only proven cure for children with the disease is a stem-cell transplant from a tissue-matched sibling. The risky procedure which destroys the patient's own immune system, then restores it using the donor's cells is an option for only about 10% of children because matched siblings are so rare.

Recently, however, researchers have begun to expand the pool of eligible patients by using less perfectly matched donor stem cells, along with techniques that enrich the transplanted cells and reduce the chances of infection and rejection. Even adults have been successfully treated.

"The toxicity of the transplant used to be too much for adults. There was 100% mortality," said Thompson at Lurie Children's Hospital. "We've identified ways to minimize the risks."

Another promising approach involves gene therapy using an inactivated virus to ferry a corrective gene into the patient's cells. Bluebird bio's experimental gene therapy LentiGlobin adds a gene that can enable patients to make healthy red blood cells, with the goal of replacing sickled cells as they die off. Early results from a small group of patients are encouraging.

Cost, as always, is a looming issue. Novartis said Adakveo will be priced at $2,357 per vial, so a year's treatment would cost between about $84,000 and $113,000.

Booker has personal experience with the cost barrier. She wanted to try Endari, but couldn't afford the $300 monthly co-pay. She applied to Emmaus Medical Inc.'s financial assistance program and recently began taking the company's drug.

"I do keep abreast of research. I need to because sometimes even doctors don't know what's going on," Booker said. "I worry that these new drugs will be too expensive for patients to afford or, worse, they won't know about them."

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Newly approved sickle cell drug ushers in an era of therapeutic breakthroughs - Worcester Telegram

A new stem cell therapy promises to heal injured heart – TheHealthSite

This study published in the journal Nature reported that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.

Mediated by macrophage cells of the immune system, the secondary healing process provided a modest benefit to heart function after a heart attack, according to the principal investigator Jeffery Molkentin, PhD, director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center and a professor of the Howard Hughes Medical Institute (HHMI).

The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimized scar and improved contractile properties, Molkentin said.

The findings build on a 2014 study published by the same research team. As in that earlier study, the current paper shows that injecting c-kit positive heart stem cells into damaged hearts as a strategy to regenerate cardiomyocytes doesnt work.

The findings prompted Molkentin and his colleagues to conclude that there is a need to re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work.

Researchers worked with two types of heart stem cells currently used in the clinical trials bone marrow mononuclear cells and cardiac progenitor cells.

As they went through the process of testing and re-verifying their data under different conditions, they were surprised to discover that in addition to the two types of stem cells, injecting dead cells or even an inert chemical called zymosan also provided benefit to the heart by optimizing the healing process. Zymosan is a substance designed to induce an innate immune response.

They reported that stem cells or zymosan therapies tested in this study altered immune cell responses that significantly decreased the formation of extracellular matrix connective tissue in the injury areas, while also improving the mechanical properties of the scar itself.

Researchers also found that stem cells and other therapeutic substances like zymosan have to be injected directly into the hearts surrounding the area of infarction injury.

Most of the current trials were also incorrectly designed because they infuse cells into the vasculature. Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic, Molkentin explained.

Published : November 30, 2019 11:06 am

Original post:
A new stem cell therapy promises to heal injured heart - TheHealthSite

Harnessing Gamma T Cells To Bring Effective Therapies to Patients – Technology Networks

GammaDelta Therapeutics is a company that focusses on utilizing the unique properties of gamma delta () T cells to develop novel immunotherapies for patients.Through their research, the companys scientists have discovered a number of targets and antibodies that have the potential to modulate the activity of T-cells in situ. Therefore, GammaDelta Therapeutics recently announced the formation of Adaptate Biotherapeutics, a spin-out company that will focus on research in this area.

Technology Networks spoke with Natalie Mount, CEO of Adaptate BioTherapeutics, to learn more about the company's aims and the challenges faced when developing immunotherapies and advancing them into clinical studies.

Molly Campbell (MC) Please can you tell us more about T-cell based cell therapy products and their potential applications?Natalie Mount (NM): T cells play an increasingly appreciated critical role in immune surveillance, being able to recognize malignant/transformed cells through a pattern of stress markers. The recognition mechanism is not major histocompatibility complex (MHC) restricted and not dependent on a single antigen.

T cells therefore have potential in a range of disease indications, including both hematological and solid malignancies and a positive correlation between T cell infiltration and prognosis/survival in patients has been determined in a range of oncology indications in studies published in the literature by other groups. Additionally, as a cell therapy, T cells can be used in an allogeneic setting (ie, T cells can be used for unrelated recipients without a requirement for matching).

Both Adaptate Biotherapeutics and GammaDelta Therapeutics are focussed on harnessing the potential of T cells, in particular the V1 subtype which is the predominant T cell type in tissue.This is based on data originating from the labs of Professor Adrian Hayday of Kings College London and the Crick Institute, supported by Cancer Research Technology and also from Professor Bruno Silva Santos of Institute for Molecular Medicine at the University of Lisbon, Portugal.

Previous clinical trials conducted by other groups/companies targeting or using T cells in cancer have focussed on the V2 subtype which is predominant in the blood. These trials have demonstrated safety, but efficacy has been limited.Compared to V2 cells, V1 cells, which are the focus of work at Adaptate Biotherapeutics and GammaDelta Therapeutics, are less susceptible to exhaustion and activation induced cell death. Expansion of donor derived V1 has been shown to be a positive prognostic indicator for acute myeloid leukemia patients following hematopoietic stem cell transplant.

MC: Why are current immunotherapy treatment approaches limited?NM: Immunotherapy approaches have had very significant success and impact in Oncology recently, however, challenges and unmet needs remain.One challenge is effective treatment of solid tumors. The hypoxic, low nutrient tumor environment provides a challenge for successful infiltration and activation of T cells. However, V1 T cells have real potential as they are naturally tissue resident and hence primed for this environment. In addition, their ability to recognize malignant cells by a pattern of markers expressed by dysregulated, transformed cells rather than one specific antigen presented by the MHC provides an additional advantage for both specificity of response and maintenance of efficacy.

T cells act as orchestrators of an immune response and, following recognition of a cell as malignant, they induce maturation of monocytes and signal to alpha beta T cells, hence increasing immunogenicity of the tumor and providing a sustained response, with potential even in tumors with low mutational load which have proven challenging with other immunotherapies.

MC: The new spin-out company, Adaptate Biotherapeutics, will build on GammaDelta's knowledge to modulate T-cell activity using therapeutic antibodies. Why have you decided to create a spin-out focusing on this area of research?NM: GammaDelta Therapeutics was formed in 2016 to harness the unique properties of T cells, and since then has gained extensive knowledge of T-cell biology. In addition to gaining insight into cell growth and isolation, the companys scientists have also discovered a number of targets and antibodies that have potential to modulate the activity of T-cells in situ.

GammaDelta Therapeutics now has a pipeline of cell therapy products progressing into clinical development under the guidance of CEO, Dr Paolo Paoletti.

Adaptate Biotherapeutics will be developing antibodies which will be administered to cancer patients to modulate activity of the patient's gamma delta T cells in situ.

Delivery of cell therapy and antibody therapeutics each needs focus and specific skillsets and formation of two independent entities will facilitate this. The two companies share a common goal to harness the potential of T cells to bring effective therapies to patients. Both benefit from support of the scientific founding team and have common investors, Abingworth and Takeda Pharmaceuticals.MC; Your goal is to develop targets and antibodies that can modulate the activity of T-cells and advance them into clinical studies. What challenges exist here, and how do you hope to overcome them?

Our assets at Adaptate Biotherapeutics are currently at the pre-clinical stage and therefore face the non-clinical development risks for a novel therapy. However, these risks are mitigated by biology understanding from our scientific founders and the work at GammaDelta Therapeutics to date.

One of our challenges is in selecting the most suitable patient population for initial trials. There is potential for opportunity for our therapeutics in multiple indications but the utility of animal models in modelling the human immune compartment and human tumor setting is limited. Therefore in vitro and ex vivo models are important, in addition to the learnings from other clinical studies.

MC: GammaDelta Therapeutics formed in 2016 to gain extensive knowledge of T-cell biology and to developing a portfolio of investigational cell therapies. Some of these cell therapies are poised to enter clinical development. Can you tell us any further information about these therapies?NM: GammaDelta was set up to develop cell-based therapy utilizing ex-vivo expanded tissue resident gd T cells. Subsequent acquisition of Lymphact SAS allowed GammaDelta to augment its capabilities with a platform for ex-vivo expansion of blood derived V1 cells. GammaDelta is focussed on progressing ex-vivo expanded skin and blood derived V1 cells to the clinic both in unengineered and engineered formats. Clinical trials are currently on track to commence in the next 12-18 months.

MC: Your press release states: "The two companies will continue sharing their insights into T-cell biology as they work towards developing different therapeutic modalities". How will you continue to share insights here?NM: Antibodies and cells represent complementary approaches to realizing the potential of T cell activity for patients with solid and haematological malignancies.

The two companies will work together in areas of common interest in the biology of these fascinating cells, such as understanding the phenotype and behavior of T cells in tumors and mechanisms of cell regulation as well as the effects of antibody on the T cells.

We have deliberately established a contractual framework that allows efficient collaboration between scientists of both the companies via formal and informal meetings.

MC: What are your hopes for the future of Adaptate Biotherapeutics?NM: This is a remarkable time in the development of new immune therapies, and the role of "non-conventional" cell types of the immune system is coming to the fore as we recognize the successes achieved to date and the needs of patients and related scientific challenges that remain.

Both GammaDelta Therapeutics and Adaptate Biotherapeutics are at the lead of translating our increasing understanding of T cell biology and its potential into therapies to address these unmet needs.

Adaptate Biotherapeutics has a fantastic opportunity to build and accelerate a portfolio of antibody-based approaches in this novel area and I look forward to the successful translation of this science into therapies with the support of our investors at Abingworth and Takeda Pharmaceuticals.

Dr Natalie Mount, CEO of Adaptate Biotherapeutics was speaking with Molly Campbell, Science Writer, Technology Networks.

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Harnessing Gamma T Cells To Bring Effective Therapies to Patients - Technology Networks

Phase 2 Trial Data on ALS NurOwn Therapy, Supporting Safety And Early Efficacy, Published – ALS News Today

NurOwn showed a good safety profile, as well as potential efficacy in a Phase 2 clinical trial that included people with rapidly progressing amyotrophic lateral sclerosis (ALS).

Trial results, which have been previously reported, have now been published in the journalNeurology in a paper titled, NurOwn, phase 2, randomized, clinical trial in patients with ALS.

NurOwn, which is being developed by BrainStorm Cell Therapeutics, is a stem cell-based therapy. It involves taking mesenchymal stem cells (MSCs), a type of cell capable of differentiating into other cell types, from a person.

These MSCs are modified so they produce more neurotrophic factors (NTFs) compounds that help drive the growth and survival of nervous tissue. The cells are then re-introduced to the body by injection into muscles and/or the spinal canal (termed intramuscular and intrathecal injection, respectively).

In the Phase 2 trial (NCT02017912), which was funded by BrainStorm, 48 people with ALS were enrolled; 36 were treated with NurOwn, and 12 were given a placebo. Participants received the treatment after a three-month pretransplant period, and were followed for six months after treatment.

The participants were predominantly (72.9%) male, and their average age was 51.1 years.

The studys primary goal of the study was to evaluate safety, measured by number of patients with adverse events to treatment, and this goal was met. The use of NurOwn was found to be safe and well-tolerated.

Eleven participants nine in the treatment group and two in the placebo group developed 16 serious adverse events (SAEs).

All treatment-emergent SAEs [those that occurred after start of treatment] were deemed to be related to ALS disease progression, and none was considered possibly, probably, or definitely related to study treatment, the researchers noted.

Data were also analyzed for early indications of treatment efficacy. Researchers specifically looked at rate of disease progression, as measured by the slope (that is, the change over time) in scores on the Revised ALS Functional Rating Scale (ALSFRS-R).

Overall, these rates were not significantly different between the NurOwn-treated and placebo groups.

However, in a subset of 21 patients with particularly rapid disease progression (15 given NurOwn and six a placebo), the average rate of disease progression showed a significantly improvement at two weeks (+3.3 vs. 1.3) and four weeks (+2.0 vs. 0.1) following treatment for those that got NurOwn.

Rapid progressors were defined in this study, at enrollment, as those with a decline of more than 2 points in ALSFRS-R scores during the pretreatment period.

This positive trend continued for all study time points, but it was not statistically significant after four weeks.

The researchers also looked at the proportion of patients with an improvement of at least 1.5 points each month, based on the reasoning that, responder analyses may more accurately capture individual treatment responses than changes in mean slope alone. That is, because each individual with ALS is different, some might be more likely to respond to treatment than others.

At four weeks post-treatment, a significantly greater proportion of those given NurOwn compared to placebo met this responder threshold (47% vs. 9%). In the rapid progression group, there were significant differences at both week four and week twelve (80% vs. 0%, and 53% vs. 0%, respectively).

For all of the above efficacy measurements, the greatest response was seen shortly following the injection, with decreasing response over time. This may suggest the need for repeated treatments to maintain a sustained therapeutic effect, the researchers wrote.

Cerebrospinal fluid (CSF) was collected just before and two weeks after the injection. Analysis of this fluid, broadly, showed an increase in levels of NTFs and a decrease in inflammatory markers, which suggests that NurOwn works as intended. (CSFfluid surrounds the brain and spinal cord.)

Specifically, the levels of monocyte chemoattractant protein-1 (MCP-1), a marker of immune cell infiltration and neuroinflammation, were significantly lower post-treatment in patients given NurOwn, while no significant change was observed in the placebo group. This correlated with ALSFRS-R slope improvement at all time points.

[W]e observed a clear biological effect of the treatment on CSF biomarkers to support its proposed mechanism of action in ALS, Robert H. Brown Jr., PhD, MD, of the University of Massachusetts Medical School and a study co-author, said in a BrainStorm press release.

We met our primary endpoint and demonstrated that a single dose of NurOwn was safe and well-tolerated while supporting NurOwns mechanism of action on neuroprotection and neuroinflammation pathways in ALS, added Ralph Kern, MD, MHSc, chief operating officer and chief medical officer of BrainStorm.

We look forward to completing the current Phase 3 study to confirm the promising Phase 2 findings and expand our understanding of the potential of MSC-NTF cell therapy in ALS, Kern added.

A fully enrolled, placebo-controlled Phase 3 study (NCT03280056) is underway in the U.S. in 200 ALS patients, and a secondary safety analysis found no new concerns. The trial is expected to conclude in late 2020, with results announced shortly thereafter.

Results from the [Phase 2] study underscore the importance of conducting a larger Phase 3 clinical trial that will build upon the data collected in our Phase 2 study, said Chaim Lebovits, Brainstorms president and chief executive officer. We look forward to reporting our clinical results in the scientific literature and through corporate announcements.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Total Posts: 279

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Phase 2 Trial Data on ALS NurOwn Therapy, Supporting Safety And Early Efficacy, Published - ALS News Today

Proof-of-Concept Study of CAR-NK Cell Therapy with Engineered Persistence Shows Potential – Cancer Network

A first-of-kind multi-antigen targeted off-the-shelf chimeric antigen receptor (CAR)-natural killer (NK) cell with engineered persistence has the potential to be a readily available treatment option for patients, Robert A. Brodsky, MD, said in a preview of the 61st American Society of Hematology (ASH) Annual Meeting & Exposition.

As most of you are well aware, CAR T-cell (therapy has) captured the imagination of physician scientists and patients alike, mainly for their incredible efficacy in treating B-cell malignancies like acute lymphocytic leukemia and non-Hodgkin lymphomas, said Brodsky, who serves as secretary of ASH and is also the director of the division of hematology at Johns Hopkins Medicine.

However, he added, this treatment option does not come without its drawbacks: namely, time, expenses, toxicity.

Only about two-thirds of patients enrolled in CAR T-cell trials will actually see infusion because often the disease will progress during the time it takes to make a successful product, Brodsky said.

Therefore, there is a need to develop a more timely infusion that can be associated with lower costs, and hopefully, less toxicity.

At the upcoming ASH Annual Meeting & Exposition, being held from December 7-10 in Orlando, Florida, Jode P. Goodridge, PhD, will present on his teams proof-of-concept study of induced pluripotent stem cell (iPSC)-derived effector cells.

iPSC-derived effector cells offer distinct advantages for immune therapy over existing patient- or donor- derived platforms, both in terms of scalable manufacturing from a renewable starting cellular material and precision genetic engineering that is performed at the single-cell level, the researchers wrote in their abstract. iPSC derived natural killer (iNK) cells offer the further advantage of innate reactivity to stress ligands and MHC downregulation and the potential to recruit downstream adaptive responses.

The candidate, called FT596, is consistently manufactured from a master iPSC line engineered to uniformly express an NK cell-calibrated CD19-targeting CAR, an enhanced functioning high-affinity, non-cleavable CD16, and a recombinant fusion of IL-15 and IL-15 receptor alpha for cytokine-autonomous persistence, according to the abstract.

What the authors here did is take advantage of the use of induced pluripotent stem cells and differentiated them to natural killer cells. Natural killer cells are not T cells but they are another form of lymphocytes that can be very effective in killing cancer cells. What they did is they engineered these pluripotent stem cells to target B cells, and they are specifically targeting the CD19 antigen on B cells and showing that these are very effective in cell line models and animal models, explained Brodsky.

However, of note, this product has not been tested in humans yet.

The big advance here is that this offers the potential of having a readily available source of basically CAR-NK cells that wouldnt need time to grow them up before they would be infused, Brodsky concluded.

Goodridge JP, Mahmood S, Zhu H, et al. FT596: Translation of First-of-Kind Multi-Antigen Targeted Off-the-Shelf CAR-NK Cell with Engineered Persistence for the Treatment of B Cell Malignancies. Presented at: 61st ASH Annual Meeting & Exposition Meeting preview; to be presented December 7, 2019; Orlando, Fla. Abstract 301.

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Proof-of-Concept Study of CAR-NK Cell Therapy with Engineered Persistence Shows Potential - Cancer Network

Mum shares heartbreaking photo of toddler sobbing through gruelling cancer treatment – The Sun

THIS is the heartbreaking moment a tiny toddler sobs her way through agonising cancer treatment.

Sophia Soto's devastated mum took the photo while her little 14-month-old was having a lead put on her chest.

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Sophia was diagnosed with stage 4 neuroblastomaafter tumours were discovered behind her eyes and on her kidney.

And her mum Rosie, 40, is now sharing the harrowing image in a bid to highlight the traumatic reality of childhood cancer.

Speaking about the poignant picture, Rosie, from Florida, USA, said: "The picture of Sophia upset really does home in on the reality of childhood cancer.

"She was having a lead put on her chest for her treatment - which she didn't want - hence why Sophia was so upset.

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"I look back at the picture now and wonder how I did it; it was so hard watching my little girl so ill."

Rosie first suspected something was wrong with Sophia after she began developing bruising around her eyes - something she claims doctors repeatedly dismissed as being from bump or fall.

It wasn't until Rosie took Sophia to see an eye specialist that they spotted what they suspected tumours behind her eyes were causing the bruising.

Rosie added: "Sophia kept getting bruising on her eyes and I didn't recall her falling over or anything, so I didn't understand where they were come from.

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"I kept taking her to the doctors because the bruising wasn't going away, but they just said it must have been from a bump or something.

"Sophia wasn't referred for a scan or biopsy until I went to see an eye specialist with her who knew straight away that it was caused by a tumour.

"She was sent for an MRI where black spots appeared on the scans confirming the tumours behind her eyes.

"It was then the biopsy which found the tumours on one of her kidneys as well which led to her stage 4 neuroblastoma diagnosis."

Neuroblastoma is a rare type of cancer that mostly affects babies and children and develops from specialised nerve cells left behind from a baby's development in the womb.

I kept taking her to the doctors because the bruising wasn't going away, but they just said it must have been from a bump or something

After being diagnosed in March 2014, the then 14-month-old endured 60 rounds of chemotherapy, 20 rounds of radiation and a stem cell transplant over a six months period.

Thankfully, Sophia, now six, has been in remission for five years and now looks like a completely different child compared to the one in the heart wrenching photograph.

She was told she was in remission in November 2014 and has been medication free for two years.

Sophia isn't yet classified as 'cancer free' so still goes for check ups every six months with specialists.

What are the symptoms of cancer in children?

Cancer symptoms can be very similar to those of other childhood illnesses - and they vary between children.

According toCancer Research UK, there are 15 signs to look out for:

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The brave six-year-old still has tumours behind her eyes which cannot be removed due to the placement of them, but doctors believe the tumours are benign and therefore not causing Sophia too much harm.

Rosie added: "Doctors are reluctant to remove the tumours Sophia currently has behind her eyes as they've said it would be likely the surgery to disfigure her face.

"Whilst they are tumours, doctors are reasonably confident that they are not cancerous so we have decided to not have the surgery right now, but it may be something she has when she's older."

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Sophia now looks like any other fun-loving child and from her appearance, you would never know she had cancer.

The six-year-old loves to dance and hopes of becoming a vet one day.

Rosie said: "No one can imagine what she went through looking at her now - she just looks like a normal regular child.

"Sophia has her moments when she asks about when she was sick and has questions about her treatment scars, but over all she's a pretty happy girl.

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SURGE IN CASES Every 90 secs someone is told they have cancer - 10 signs you need to know

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"If I was to say anything to other parents with children battling cancer, I'd say to them to not give up, stay positive and keep your faith.

"It's really important not to compare your child's process to anyone else as everyone battles illnesses differently as every situation is different.

"We're over the moon that Sophia is now doing so well - we're really blessed that she's such a fighter."

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Mum shares heartbreaking photo of toddler sobbing through gruelling cancer treatment - The Sun

Mesenchymal Stem Cells Market Key Trends, Key Players, Challenges And Standardization, Analysis Of Key Players, And Forecast To 2026 – WindStreetz

The Global Mesenchymal Stem Cells Market covers explicit data considering the advancement rate, advertise gauges, drivers, confinements, future based interest, and income during the figure time frame. Further, the report comprises information amassed from various primary and secondary sources.

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The mesenchymal stem cells market is anticipated to grow at a CAGR of 7.0% from 2018 to 2026 according to a new research report published by Alexa Reports Research. The market was valued at USD 1,335.1 million in 2017 and is estimated to reach USD 2,518.5 Million by 2026. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.

Mesenchymal stem cells are adult stem cells, which are traditionally found in the bone marrow. However, they can also be parted from other available tissues including peripheral blood, cord blood, fallopian tube. These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.

The global mesenchymal stem cells market growth is primarily driven by the increasing demand for these stem cells as an effective treatment alternative for knee replacement in the recent past.

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Furthermore, increasing elderly population across the globe, and rising prevalence of various chronic diseases including cancer, autoimmune diseases, bone and cartilage diseases are factors expected to boost the market growth during the forecast period. In addition, effective government policies, and funding for research and development would positively influence the market growth over coming years. However, some of the political point of views, and higher cost of treatment by using mesenchymal stem cells might restraint the growth during the forecast period.

Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.

The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.

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About Us:Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data. Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data.

Contact Us:Alexa ReportsPh no: +1-408-844-4624 / +91- 7030626939Email: sales@alexareports.comSite: https://www.alexareports.com

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Mesenchymal Stem Cells Market Key Trends, Key Players, Challenges And Standardization, Analysis Of Key Players, And Forecast To 2026 - WindStreetz

Bone Therapeutics SA: Information on the total number of voting rights and shares – GlobeNewswire

Regulated information

Gosselies, Belgium, 29November 2019, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopaedics and bone diseases, today announces an increase in the total number of voting rights and shares as a result of the issuance of new shares on 14November 2019 following the conversion of convertible bonds issued on the private placement on 7March 2018. The following information is published in accordance with Article15 of the Belgian Law of 2May 2007 on the publication of major shareholdings in issuers whose shares are admitted to trading on regulated market.

(1) Based on the conversion price of EUR3.3971 (92% of the Volume-Weighted-Averaged-Price of Bone Therapeutics shares on 14November 2019).

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy and an innovative biological product in later-stage clinical development across a number of disease areas, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its allogeneic cell therapy platform (ALLOB) which uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, cutting-edge manufacturing process.

The Companys ALLOB product pipeline includes a cell therapy product candidate that is expected to enter PhaseIIb clinical development for the treatment of difficult-to-heal fractures and a PhaseII asset in patients undergoing a spinal fusion procedure. In addition, the Company is also developing an off-the-shelf protein solution, JTA-004, which is expected to enter PhaseIII development for the treatment of pain in knee osteoarthritis.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. Further information is available at http://www.bonetherapeutics.com.

Contacts

Bone Therapeutics SAThomas Lienard, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10 00investorrelations@bonetherapeutics.com

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709 5701bonetherapeutics@consilium-comms.com

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics SA: Information on the total number of voting rights and shares - GlobeNewswire