Hematopoietic stem cells are young or immature blood cells found to be living in bone marrow. These blood cells on mature in bone marrow and only a small number of these cells get to enter blood stream. These cells that enter blood stream are called as peripheral blood stems cells. Hematopoietic stem cells transplantation is replacement of absent, diseased or damaged hematopoietic stem cells due to chemotherapy or radiation, with healthy hematopoietic stem cells. Over last 30 years hematopoietic stem cells transplantation market seen rapid expansion and constant expansion with lifesaving technological advances. Hematopoietic stem cells transplantation is also known blood and marrow transplantation which brings about reestablishment of the patients immune and medullary function while treating varied range of about 70 hematological and non-hematological disorders. In general hematopoietic stem cells transplantation is used in treatment of hereditary, oncological, immunological and malignant and non-malignant hematological diseases.

There are two types of peripheral blood stem cell transplants mainly autologous and allogeneic transplantation. In autologous transplants patients own hematopoietic stem cells are harvested or removed before the high-dose treatment that might destroy the patients hematopoietic stem cells. While in allogeneic transplants stem cells are obtained from a tissue type of matched or mismatched donor. Hematopoietic stem cells are harvested from blood or bone marrow and is then frozen to use later. Depending upon the source of hematopoietic stem cells, worldwide there are three types of hematopoietic stem cells transplants namely bone marrow transplant (BMT), peripheral blood stem cell transplant and cord blood transplant. Major drivers in the hematopoietic stem cells transplantation market are establishment of strong and well developed network of hematopoietic stem cells transplantation organizations having global reach and presence has recognized NGO named Worldwide Network for Blood and Marrow Transplantation Group (WBMT) in official relation with World Health Organization (WHO) and rapid increase in number of transplants. Major restraints in hematopoietic stem cells transplantation market is high cost of transplantation and lack of funding for WBMT and other organizations such as regional, national and donor.

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The global market for Hematopoietic stem cells transplantation market is segmented on basis of transplant type, application, disease indication, end user and geography:

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Based on transplantation type, hematopoietic stem cells transplantation market is segmented into allogeneic and autologous. Hematopoietic stem cells transplantation market is also segmented by application type into bone marrow transplant (BMT), peripheral blood stem cell transplant and cord blood transplant. The market for hematopoietic stem cells transplantation is majorly driven by bone marrow transplant (BMT) segment. Based on end user hematopoietic stem cells transplantation market is segmented into hospitals and specialty centers. Peripheral blood stem cell transplant type holds the largest market for hematopoietic stem cells transplantation. Hematopoietic stem cells transplantation market is further segmented by disease indication into three main categories i.e. lymphoproliferative disorders, leukemia, and non-malignant disorders. Segment lymphoproliferative disorder holds largest share amongst the three in Hematopoietic stem cells transplantation market. On the basis of regional presence, global hematopoietic stem cells transplantation market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. Europe leads the global hematopoietic stem cells transplantation market followed by U.S. due to easy technological applications, funding and high income populations. Other reasons for rise in hematopoietic stem cells transplantation market is high prevalence of lymphoproliferative disorders and leukemia; demand for better treatment options; and easy accessibility and acceptance of population to new technological advances. Transplantation rates in high income countries are increasing at a greater extent but continued rise is also seen in low income countries and expected to rise more. Hematopoietic stem cells transplantation market will have its potential in near future as being a perfect alternative to traditional system in many congenital and acquired hematopoietic disorders management. While India, China and Japan will be emerging as potential markets. An excellent and long term alternative to relief by side effects of chemotherapy, radiotherapy and immune-sensitive malignancies is another driver for hematopoietic stem cells transplantation market. The key players in global hematopoietic stem cells transplantation market are Lonza, Escape Therapeutics, Cesca Therapeutics Inc., Regen BioPharma, Inc., Invitrx Inc, StemGenex, Lion Biotechnologies, Inc., CellGenix GmbH, Actinium Pharmaceuticals, Inc., Pluristem, Kite Pharma, Novartis AG.You Can Request for TOC Here @https://www.persistencemarketresearch.com/toc/14563

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The Hematopoietic Stem Cells Transplantation market to Undergo positive Transformation between 2017 and 2025 - Crypto Daily

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press release: The UW has a long history of pioneering medical advancements that have transformed the world. From performing the first bone marrow transplant in the United States to cultivating the first laboratory-derived human embryonic stem cells. Now, where will UW medical research go next?

On the next Wisconsin Medicine Livestream, meet trailblazing doctors, researchers, and medical leaders who are charting a bold course to completely alter the health care landscape. During this insightful panel discussion, well explore how gene therapy and cell replacements could hold the keys to treating inherited and acquired blindness. Youll also discover the remarkable potential in xenotransplantation where nonhuman animal source organs are transplanted into human recipients. In addition, you will learn about UW Healths journey to build a multidisciplinary program to serve the community. These, and other, fascinating developments in treatment and care are happening right now at the UW and are the future of medicine. The presentation will be moderated by Robert Golden, the dean of the University of WisconsinMadisons School of Medicine and Public Health.

Our Guests:

David Gamm, professor, Department of Ophthalmology and Visual Sciences; Emmett A. Humble Distinguished Director, McPherson Eye Research Institute; Sandra Lemke Trout Chair in Eye Research

Dr. Gamms lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases (RDDs). As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. The Gamm Lab uses induced pluripotent stem cells to create retinal tissues composed of authentic human photoreceptor cells rods and cones that can detect light and initiate visual signals in a dish. The aims of his laboratory are to investigate the cellular and molecular events that occur during human retinal development and to generate cells for use in retinal disease modeling and cell replacement therapies. In collaboration with other researchers at UWMadison and around the world, the lab is developing methods to produce and transplant photoreceptors and/or retinal pigment epithelium (RPE) in preparation for future clinical trials. At the same time, the Gamm Lab uses lab-grown photoreceptor and RPE cells to test and advance a host of other experimental treatments, including gene therapies. In so doing, the lab seeks to delay or reverse the effects of blinding disorders, such as retinitis pigmentosa and age-related macular degeneration, and to develop or codevelop effective interventions for these RDDs at all stages of disease.

Dhanansayan Shanmuganayagam, assistant professor, Department of Surgery, School of Medicine and Public Health; Department of Animal and Dairy Sciences, UWMadison; director, Biomedical, and Genomic Research Group

Dr. Shanmuganayagams research focuses on the development and utilization of pigs as homologous models to close the translational gap in human disease research, taking advantage of the overwhelming similarities between pigs and humans in terms of genetics, anatomy, physiology, and immunology. He and his colleagues created the human-sized Wisconsin Miniature Swine breed that is unique to the university. The breed exhibits greater physiological similarity to humans, particularly in vascular biology and in modeling metabolic disorders and obesity. He currently leads genetic engineering of swine at the UW. His team has created more than 15 genetic porcine models including several of pediatric genetic cancer-predisposition disorders such as neurofibromatosis type 1 (NF1). In the context of NF1, his lab is studying the role of alternative splicing of the nf1 gene on the tissue-specific function of neurofibromin and whether gene therapy to modulate the regulation of this splicing can be used as a viable treatment strategy for children with the disorder.

Dr. Shanmuganayagam is also currently leading the efforts to establish the University of Wisconsin Center for Biomedical Swine Research and Innovation (CBSRI) that will leverage the translatability of research in pig models and UWMadisons unique swine and biomedical research infrastructure, resources, and expertise to conduct innovative basic and translational research on human diseases. The central mission of CBSRI is to innovate and accelerate the discovery and development of clinically relevant therapies and technologies. The center will also serve to innovate graduate and medical training. As the only center of its kind in the United States, CBSRI will make UWMadison a hub of translational research and industry-partnered biomedical innovation.

Petros Anagnostopoulos, surgeon in chief, American Family Childrens Hospital; chief, Section of Pediatric Cardiothoracic Surgery; professor, Department of Surgery, Division of Cardiothoracic Surgery

Dr. Anagnostopoulos is certified by the American Board of Thoracic Surgery and the American Board of Surgery. He completed two fellowships, one in cardiothoracic surgery at the University of Pittsburgh School of Medicine and a second in pediatric cardiac surgery at the University of California, San Francisco School of Medicine. He completed his general surgery residency at Henry Ford Hospital in Detroit. Dr. Anagnostopoulos received his MD from the University of Athens Medical School, Greece. His clinical interests include pediatric congenital heart surgery and minimally invasive heart surgery.

Dr. Anagnostopoulos specializes in complex neonatal and infant cardiac reconstructive surgery, pediatric heart surgery, adult congenital cardiac surgery, single ventricle palliation, extracorporeal life support, extracorporeal membrane oxygenation, ventricular assist devices, minimally invasive cardiac surgery, hybrid surgical-catheterization cardiac surgery, off-pump cardiac surgery, complex mitral and tricuspid valve repair, aortic root surgery, tetralogy of Fallot, coronary artery anomalies, Ross operations, obstructive cardiomyopathy, and heart transplantation.

When: Tuesday, Sept. 29, at 7 p.m. CDT

Where: Wisconsin Medicine Livestream: wiscmedicine.org/programs/ending-alzheimers

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ONLINE: The Future of Medicine - Isthmus

Fort Collins, Colorado The Stem Cell Therapy Market is growing at a rapid pace and contributes significantly to the global economy in terms of turnover, growth rate, sales, market share and size. The Stem Cell Therapy Market Report is a comprehensive research paper that provides readers with valuable information to understand the basics of the Stem Cell Therapy Report. The report describes business strategies, market needs, dominant market players and a futuristic view of the market.

The report has been updated to reflect the most recent economic scenario and market size regarding the ongoing COVID-19 pandemic. The report looks at the growth outlook as well as current and futuristic earnings expectations in a post-COVID scenario. The report also covers changing market trends and dynamics as a result of the pandemic and provides an accurate analysis of the impact of the crisis on the market as a whole.

Global Stem Cell TherapyMarketwas valued at 117.66 million in 2019 and is projected to reach USD255.37 million by 2027, growing at a CAGR of 10.97% from 2020 to 2027.

Industry Stem Cell Therapy Study provides an in-depth analysis of key market drivers, opportunities, challenges and their impact on market performance. The report also highlights technological advancements and product developments that drive market needs.

The report contains a detailed analysis of the major players in the market, as well as their business overview, expansion plans and strategies. Key players explored in the report include:

The report provides comprehensive analysis in an organized manner in the form of tables, graphs, charts, pictures and diagrams. Organized data paves the way for research and exploration of current and future market outlooks.

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The report provides comprehensive data on the Stem Cell Therapy market and its trends to help the reader formulate solutions to accelerate business growth. The report provides a comprehensive overview of the economic scenario of the market, as well as its benefits and limitations.

The Stem Cell Therapy Market Report includes production chain analysis and value chain analysis to provide a comprehensive picture of the Stem Cell Therapy market. The research consists of market analysis and detailed analysis of application segments, product types, market size, growth rates, and current and emerging industry trends.

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

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The market is geographically spread across several key geographic regions and the report includes regional analysis as well as production, consumption, revenue and market share in these regions for the 2020-2027 forecast period. Regions include North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa.

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Key Questions Addressed in the Report:

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Stem Cell Therapy Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

"Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease," said Geoff MacKay, AVROBIOs president and CEO. "Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder."

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

Story continues

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as "aims," "anticipates," "believes," "could," "designed to," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform; the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200928005176/en/

Contacts

Investor Contact: Christopher F. BrinzeyWestwicke, an ICR Company339-970-2843chris.brinzey@westwicke.com

Media Contact: Tom DonovanTen Bridge Communications857-559-3397tom@tenbridgecommunications.com

See the article here:
AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease - Yahoo...

COMEDIAN Al Murray told Lorraine Kelly today his seven-year-old nephew is "very ill" but is "hanging in there" amid his battle with leukemia.

The Pub Landlord star, 52, appeared on the ITV lunchtime show to urge viewers to donate the price of a round of drinks to cancer charity DKMS, and help them boost their stem cell register.

3

Finley Relf has a rare and aggressive form ofleukaemiaand his only hope of survival is a bone marrow transplant.

Al's partner Eleanor Relf is the sister of Finley's dad Ben. Finley's family had been tested to be a donor, but are sadly not a match.

Speaking to Lorraine, 60, Al said: "Hes an incredibly brave little lad.

"I cant tell you hes well, hes very, very ill, but hes hanging in there.

"They load him up with treatments and he takes it. But he's seven, he wants to be out there playing footballwith his friends."

3

Al went on: "People can get on this register and then be a life saver in waiting. That's what we're trying to do.

"Theres been a decline in donors and contributions. The thing about blood stem cell donations is its dead easy.

"They send you a pack with swabs and you swab some cheek tissue and send it back to them."

And Lorraine joked: "It's just like picking your nose which we all do!"

It takes just a few minutes to become a donorbut Al explained that processing the swabs to add people to the register costs DKMS 40 per person.

3

Finley, who lives in Haywards Heath, West Sussex, with four-year-old brother Jacob, is having chemotherapy at Londons Great Ormond Street Hospital.

Finleys family are hoping another stranger will be identified as his genetic twin with similar tissue. Al said Finley has tried two donors but their stem cells didn't work.

He said: Its not like blood types. Its much more fundamental to your DNA."

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"Its not quite fingerprints but its on that level, so thats why we need more people to join the register."

Finleys ordeal started last year when his parents Ben and Nicky noticed he was losing weight.

Doctors discovered his spleen was enlarged and he underwent numerous tests, which led them to diagnosing him with blood cancer.

Visit link:
Al Murray says his seven-year-old nephew is very ill but hanging on in there amid battle with rare form of L - The Irish Sun

News Release

Monday, September 28, 2020

Findings show deficits in the electrical activity of cortical cells; possible targets for treatment for 22q11.2 deletion syndrome.

A genetic study has identified neuronal abnormalities in the electrical activity of cortical cells derived from people with a rare genetic disorder called 22q11.2 deletion syndrome. The overexpression of a specific gene and exposure to several antipsychotic drugs helped restore normal cellular functioning. The study, funded by the National Institutes of Health (NIH) and published in Nature Medicine, sheds light on factors that may contribute to the development of mental illnesses in 22q11.2 deletion syndrome and may help identify possible targets for treatment development.

22q11.2 deletion syndrome is a genetic disorder caused by the deletion of a piece of genetic material at location q11.2 on chromosome 22. People with 22q11.2 deletion syndrome can experience heart abnormalities, poor immune functioning, abnormal palate development, skeletal differences, and developmental delays. In addition, this deletion confers a 20-30% risk for autism spectrum disorder (ASD) and an up to 30-fold increase in risk for psychosis. 22q11.2 deletion syndrome is the most common genetic copy number variant found in those with ASD, and up to a quarter of people with this genetic syndrome develop a schizophrenia spectrum disorder.

This is the largest study of its type in terms of the number of patients who donated cells, and it is significant for its focus on a key genetic risk factor for mental illnesses, said David Panchision, Ph.D., chief of the Developmental Neurobiology Program at the NIHs National Institute of Mental Health. Importantly, this study shows consistent, specific patient-control differences in neuronal function and a potential mechanistic target for developing new therapies for treating this disorder.

While some effects of this genetic syndrome, such as cardiovascular and immune concerns, can be successfully managed, the associated psychiatric effects have been more challenging to address. This is partly because the underlying cellular deficits in the central nervous system that contribute to mental illnesses in this syndrome are not well understood. While recent studies of 22q11.2 deletion syndrome in rodent models have provided some important insights into possible brain circuit-level abnormalities associated with the syndrome, more needs to be understood about the neuronal pathways in humans.

To investigate the neural pathways associated with mental illnesses in those with 22q11.2 deletion syndrome, Sergiu Pasca, M.D., associate professor of psychiatry and behavioral sciences at Stanford University, Stanford, California, along with a team of researchers from several other universities and institutes, created induced pluripotent stems cells cells derived from adult skin cells reprogramed into an immature stem-cell-like state from 15 people with 22q11.2 deletion and 15 people without the syndrome. The researchers used these cells to create, in a dish, three-dimensional brain organoids that recapitulate key features of the developing human cerebral cortex.

What is exciting is that these 3D cellular models of the brain self-organize and, if guided to resemble the cerebral cortex, for instance, contain functional glutamatergic neurons of deep and superficial layers and non-reactive astrocytes and can be maintained for years in culture. So, there is a lot of excitement about the potential of these patient-derived models to study neuropsychiatric disease, said Dr. Pasca.

The researchers analyzed gene expression in the organoids across 100 days of development. They found changes in the expression of genes linked to neuronal excitability in the organoids that were created using cells from individuals with 22q11.2 deletion syndrome. These changes prompted the researchers to take a closer look at the properties associated with electrical signaling and communication in these neurons. One way neurons communicate is electrically, through controlled changes in the positive or negative charge of the cell membrane. This electrical charge is created when ions, such as calcium, move into or out of the cell through small channels in the cells membrane. The researchers imaged thousands of cells and recorded the electrical activity of hundreds of neurons derived from individuals with 22q11.2 deletion syndrome and found abnormalities in the way calcium was moved into and out of the cells that were related to a defect in the resting electrical potential of the cell membrane.

A gene called DGCR8 is part of the genetic material deleted in 22q11.2 deletion syndrome, and it has been previously associated with neuronal abnormalities in rodent models of this syndrome. The researchers found that heterozygous loss of this gene was sufficient to induce the changes in excitability they had observed in 22q11.2-derived neurons and that overexpression of DGCR8 led to partial restoration of normal cellular functioning. In addition, treating 22q11.2 deletion syndrome neurons with one of three antipsychotic drugs (raclopride, sulpiride, or olanzapine) restored the observed deficits in resting membrane potential of the neurons within minutes.

We were surprised to see that loss in control neurons and restoration in patient neurons of the DGCR8 gene can induce and, respectively, restore the excitability, membrane potential, and calcium defects, said Pasca. Moving forward, this gene or the downstream microRNA(s) or the ion channel/transporter they regulate may represent novel therapeutic avenues in 22q11.2 deletion syndrome.

Grants:MH107800; MH100900; MH085953; MH060233; MH094714

About the National Institute of Mental Health (NIMH):The mission of theNIMHis to transform the understanding and treatment of mental illnesses through basic and clinical research, paving the way for prevention, recovery and cure. For more information, visit theNIMH website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Khan, T. A., Revah, O., Gordon, A., Yoon, S., Krawisz, A. K., Goold, C., Sun, Y., Kim, C., Tian, Y., Li, M., Schaepe, J. M., Ikeda, K., Amin, N. D., Sakai, N., Yazawa, M., Kushan, L., Nishino, S., Porteus, M. H., Rapoport, J. L. Paca, S. (2020). Neuronal defects in a human cellular model of 22q11.2 deletion syndrome. Nature Medicine. doi: 10.1038/s41591-020-1043-9

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The rest is here:
funded study sheds light on abnormal neural function in rare genetic disorder - National Institutes of Health

Creative minds from Yokohama National University have developed an efficient method of successfully generating hair growth. Find out more here, and what this could mean for future progress.

Published in the journal Biomaterials, the research team based in Japan focused on a research method that shows "great potential".

Led by Dr Tatsuto Kageyama and Professor Junji Fukuda, they've proposed a new approach to regenerate hair.

Previous studies have utilised a three-dimensional tissue culture called hair follicle germ (HFG).

HFG is composed of hair follicle stem cells derived from both the epithelial (outer layer of skin) and mesenchymal tissue (embryonic connective tissue).

This approach requires manually merging the stem cells derived from these different origins under a microscope.

Thus, it becomes a challenge to produce the 5,000 or more HFG needed per transplant patient.

Dr Kageyama and Professor Fukuda wanted to perform research that was scalable - something that would make hair regeneration easier.

READ MORE:Losing hair fast? Applying this natural solution to the scalp resulted in new hair growth

The team "fabricated hair beads (HBs) in U-shaped wells in a plate array using hair follicle stem cells".

The hair follicle stem cells were encapsulated in collagen - a structural protein.

In fact, collagen is believed to play an important role in hair follicle generation throughout a person's life.

"A suspension of mouse epithelial cells was then added into the wells containing the gel encapsulated hair beads," detailed the researchers.

DON'T MISS

After 24 hours, the epithelial cells adhered to the collagen gel, which then contracted to form "a bead-based hair follicle germ".

To test the efficiency of the hair bead approach, the scientists transplanted the bead-based hair follicle germ onto the back of mice.

They also used other methods, such as the one outlined earlier, to compare the results.

The collagen-enriched hair bead approach produced a "high rate of hair generation four weeks after being transplanted onto the skin of mice".

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It was noted how this method produced more hair than the other approaches, suggesting the collagen enrichment plays a vital role in hair growth.

Professor Fukuda commented on his findings: "Using an automated spotter, this approach was scalable to prepare a large number of hair follicle germs.

"[This] is important for human treatment because thousands of tissue grafts are necessary for a single patient."

The next step is to "find a way to expand the number of hair follicle stem cells".

Fukuda explained: "In this study, we worked on how to prepare tissue grafts.

"However, to deliver this approach to hair loss patients, we need a proper approach to obtain a sufficient number of hair follicle stem cells".

The researchers believe their results "show great potential for clinical applications in hair regenerative therapy".

Until then, Minoxidil has been approved as suitable treatment to help slow down hair loss.

Read more:
Hair loss treatment: Breakthrough research links one ingredient to hair growth - Express

You can print a lot of things with a 3D printer. A gun, a home, a dinner. Soon, you could even print new pieces of yourself.

While most uses of 3D printing involve extruding layers of plastic through a nozzle to create a three-dimensional structure, before too long, similar technology could let physicians print structures made of human cells -- from tiny structures like 'organs on a chip', to huge ones like whole replacement organs.

"Bioprinting has a great promise -- it has a lot of advantages and capabilities. Of course, it's not really perfect yet, but despite that, we have all these good things going on in the field," says Dr Ibrahim Ozbolat, principal investigator at Penn State University's bioprinting-focused Ozbolat Lab.

SEE: Guide to Becoming a Digital Transformation Champion (TechRepublic Premium)

One of those things is making replacement organs. The process of bioprinting human tissue is a bit more involved than, say, 3D printing a new desk toy. First, you have to get some stem cells from the person who needs the new organ, culture them in the right biochemical soup until you've got enough, then turn them into a bioink that can be extruded through a nozzle that's two microns thick (or one 80th the size of a human hair). The bioink will be pushed through the printer, usually onto a scaffold made of hydrogel. A bit more culturing, and you could have a useable tissue that can either be printed directly onto the patient in an operating theatre, or built in a lab and then implanted.

Bioprinting isn't a fast process, but it could make a substantial impact in healthcare, not least in offering a solution to the chronic shortage of donor organs. In the US, for example, there are over 112,000 people still on transplant waiting lists.

Other than there simply aren't enough of them, another problem with donated organs is that recipients' immune systems can attack them, causing the donor organ to fail. If that doesn't happen in the first few days or weeks, it will eventually -- kidneys donated from living donors tend to last around 12 to 20 years. People with transplanted organs also need to take medication to suppress their immune system long-term. While those drugs lessen the chance of organ rejection, it also leaves those taking them at risk of other diseases such as certain cancers.

Bioprinted organs made from an individuals' own tissue won't be rejected by their body, will last far longer, won't need anti-rejection meds, and can be custom made to the individual's exact measurements -- whether they're a four year old or a NFL linebacker.

That's the goal, anyway. So far, most human organs that have been made in the lab and not got as far as being implanted into people. Not all human organs are created equally -- or can be created by bioprinting, for that matter. Flat tissues, like skin, and hollow ones, like the stomach or bladder, are relatively easy to print, whereas complex solid organs -- the heart, liver, or pancreas -- would be far harder to recreate with printing due to the rich blood supply they need.

The problem, according to Dr Anthony Atala, director of the Wake Forest Institute for Regenerative Medicine, is the blood vessels of larger bioprinted organs.

"Vascularity still remains a challenge because there's so many cells per centimetre [in large organs like the heart] that you really need a lot of vascular supply and nutrition. So to create the large structures is still a challenge, even though the printer is definitely helping in that area, but we're not yet ready."

The bigger an organ is, the more blood supply it needs to bring organs and nutrients to the tissue. Large organs need a complex web of interconnected, different-sized arteries, capillaries, and veins. The walls of the vessels need to be strong enough to withstand the normal flow of blood through them without causing clots, and need to be made of specific layers. For now, it's too much complexity for 3D bioprinters to manage.

While researchers are working on how to print full-size organs, the tiniest bioprinted structures are already helping researchers. Bioprinting can also be used to make 'organs on a chip' -- tiny samples of tissue that mimic the functions and structures of their full-grown counterparts. These mini organs allow pharmaceutical companies to test drugs on versions of human tissues, and assess their effectiveness or toxicity instead of using unreliable and ethically difficult animal models.

One day, organs on a chip could be made using individuals' own cells to test potential therapies. Rather than using the same standard treatments for every patient, by taking some cells, culturing them and printing them onto the chip, physicians can have a unique view into how their patient will react to a particular drug without having to start them on a whole course of it.

"These miniature human organs we can use for drug discovery, direct toxicity testing, and personalised medicine, BCS modelling and personalised medicine. We've taken the same strategy, and by using the same printers, we can print miniature structures that replicate the normal human response," Atala says.

SEE: Coronavirus and 3D printing: How makers are stepping up to supply vital medical kit

As well as printing healthy tissue, bioprinters can make samples of diseased tissues -- like cancers -- to investigate current and future therapies.

"We're trying to find a way to create an effective treatment for solid tumours. I work with immunologists [who] engineer immune cells and make cytotoxic cells against the cancer cells. Now we're trying the immune cells in different 3D models that we print or we build. This could be used as a pre-screening tool for immunotherapy: so rather than directly going and checking things on the patient, this will be an intermediary step to screen the effectiveness of the therapy," Penn State's Ozbolat says.

And that's not the only way that bioprinting -- or rather, BioPrinters -- could help develop new therapies for common health conditions.

While HP's printers are more associated with offices rather than labs, HP also sells printers to the life sciences industry with its D300 BioPrinter line which prints drugs instead of documents. The machines are typically used in small to medium pharmaceutical companies in secondary drug discovery, where compounds thought to be effective against a particular disease are tested to see if they have any affect against the condition, and if so, at what dose.

The life sciences printer came into being, according to Annette Friskopp, global head and general manager of Specialty Printing Systems at HP Inc, after a group of HP engineers began researching the highest value fluid in the world that could be jetted through a thermal inkjet print head or similar. Was it perfume, jet fuel, rare chemical compounds?

"One of the highest value fluids that they discovered in this exploration was drug compounds If you've ever printed a photograph on an HP printer, just think of how many small droplets of ink have to directionally be sprayed down onto that piece of paper, so when the photograph comes out of your printer, there's your friends and family. Leveraging exactly that same technology, that is [drug] dispensing using thermal inkjet technology," she says.

Viiv Healthcare's David Irlbeck has been using HP's D300 in his work to create latency reversal drugs for HIV. (Latency reversal involves changing an HIV infection from its latent phase to an active one, the body can mount an immune response against it, and is thought to be a promising avenue in the search for a cure for HIV.)

SEE: 3D printing during COVID-19 shows potential, but financial realities bite

Viiv uses HP's D300 bioprinter for measuring out very small, very accurate amounts of particular compounds to see how their effectiveness changes at different doses. The machine is, says Irlbeck, "very user friendly, very easy to program, and it can do titrations that would be extremely difficult to do manually". By having a printer print out the drugs, researchers are able to get them down to finer and finer concentrations that human researchers simply wouldn't be able to make by doing the same process by hand, and would likely be less accurate if they did. And, because printers can measure out doses of drugs at tiny and highly accurate concentrations, it allows pharmaceutical researchers to reduce any wastage of the (very expensive) compounds.

Bioprinters also enable researchers to combine two drugs in very precise concentrations to see if they might have a synergistic effect -- where the two drugs together might have a greater effect than that which both produce individually. "It's very, very technically demanding to do synergy work without an instrument like the D300," Irlbeck adds.

As well as working on HIV medications, the D300 was recently co-opted into working on treatment for SARS-CoV-2, the virus that causes COVID-19, prepping drug plates for a lab at the University of North Carolina, which was looking into their antiviral potential.

The bioprinters are also being used elsewhere in the fight against coronavirus. HP has donated four of the D300e BioPrinters to four research facilities working on COVID-19: the Spanish National Research Council, the Monoclonal Antibody Discovery Laboratory at Fondazione Toscana Life Sciences in Italy, the US Center for Nuclear Receptors and Cell Signaling (CNRCS) at University of Houston, and France's Grenoble Alpes University Hospital. Between them, the facilities are using the machines for research into the fundamental biology of COVID-19, monoclonal antibodies and other potential therapeutic candidates, and work on immunisation: the future of healthcare is likely to be 3D printed.

Read more here:
3D printing is making a giant leap into health. That could change everything - ZDNet

Doctors believe they are closer to a treatment for multiple sclerosis after discovering a drug that repairs the coatings around nerves that are damaged by the disease.

A clinical trial of the cancer drug bexarotene showed that it repaired the protective myelin sheaths that MS destroys. The loss of myelin causes a range of neurological problems including balance, vision and muscle disorders, and ultimately, disability.

While bexarotene cannot be used as a treatment, because the side-effects are too serious, doctors behind the trial said the results showed remyelination was possible in humans, suggesting other drugs or drug combinations will halt MS.

Its disappointing that this is not the drug well use, but its exciting that repair is achievable and it gives us great hope for another trial we hope to start this year, said Prof Alasdair Coles, who led the research at the University of Cambridge.

MS arises when the immune system mistakenly attacks the fatty myelin coating that wraps around nerves in the brain and spinal cord. Without the lipid-rich substance, signals travel more slowly along nerves, are disrupted, or fail to get through at all. About 100,000 people in the UK live with the condition.

Funded by the MS Society, bexarotene was assessed in a phase 2a trial that used brain scans to monitor changes to damaged neurons in patients with relapsing MS. This is an early stage of the condition that precedes secondary progressive disease, where neurons die off and cause permanent disability.

The drug had some serious side-effects, from thyroid disease to raised levels of fats in the blood, which can lead to dangerous inflammation of the pancreas. But brain scans revealed that neurons had regrown their myelin sheaths, a finding confirmed by tests that showed signals sent from the retina to the visual cortex at the back of the brain had quickened. That can only be achieved through remyelination, said Coles.

Details of the work were presented on Friday at MSVirtual2020, a joint meeting of the European Committee for Treatment and Research in Multiple Sclerosis and its Americas counterpart.

While bexarotene will not go into phase 3 trials for MS, the finding that the nervous system can be stimulated to resheath damaged neurons has given scientists fresh hopes for another trial they hope to launch later this year. That trial will monitor the effects of the diabetes drug metformin along with clemastine, an antihistamine, a combination that Prof Robin Franklin at the Wellcome-MRC Cambridge Stem Cell Institute showed last year could drive remyelination in animals.

Metformin seems to work by rejuvenating stem cells in the central nervous system, which then go on to become myelin-producing cells called oligodendrocytes. These churn out fresh myelin to replace that destroyed by MS. The researchers hope the drug combination will at least slow the progression of the disease, but there is a chance it will prevent further damage to neurons completely.

The results of this trial give us confidence that medicines that promote myelin regeneration will have a real impact on the treatment of MS, and we look forward to the outcome of future trials with increased optimism, said Franklin.

Dr Emma Gray, at the MS Society, said: Finding treatments to stop MS progression is our number one priority, and to do that we need ways to protect nerves from damage and repair lost myelin. This new research is a major milestone in our plan to stop MS and were incredibly excited about the potential its shown for future studies.

Continue reading here:
MS treatment a step closer after drug shown to repair nerve coating - The Guardian

Answers Tatyana Donskikh, head of the clinical diagnostic department, including a day hospital, neurologist of the Federal Center for Medical Sciences of the Federal Medical and Biological Agency of Russia:

Despite all the efforts of modern medicine, no remedy has yet been found that can cure dementia. But it is possible to slow down the development of the disease! And these chances must be used.

Modern treatment is carried out mainly in two directions:

1. Drug therapy.

2. Optimal care that supports mental initiative and a sense of security.

The drugs available to people with dementia today can be divided into three groups:

This group includes a drug used to treat dementia of all severity. Since the binding sites of glutamate are present only in the brain and spinal cord, the agent is well tolerated and has practically no contraindications for administration. This is very important for elderly patients who often have many concomitant diseases.

This group of drugs includes a number of drugs. They prevent the breakdown of acetylcholine already formed in the brain. They are prescribed for mild to moderate severity of the disease. Since acetylcholine is often found outside the brain, acetylcholinesterase inhibitors can cause a number of side effects.

Treatment is carried out for a long time, first as monotherapy, then in combination.

Non-drug approaches to treatment are important, in particular, psychological support for patients and their relatives, neuropsychological training, music therapy, phototherapy, art therapy, aromatherapy and other methods of additional sensory stimulation, therapeutic gymnastics, etc.

Since the prevalence of Alzheimers disease is expected to grow rapidly in the world and the existing therapeutic approaches are rather modest, the search for new forms of action and methods of treatment continues constantly. There are many of these directions. These include, for example, the development of new neuroprotective drugs, neuroreparation technologies using stem cells. Particular hopes were pinned on immunological approaches associated with the use of amyloid vaccines and immunoglobulins in attempts to remove -amyloid from the brain. Unfortunately, clinical trials of amyloid vaccines have shown an unacceptably high risk of developing encephalitis or leukoencephalopathy.

The maximum benefit from any effective remedy is possible only when applied at an early, pre-demented stage of the pathological process. Therefore, it is so important to develop approaches to the earliest possible diagnosis of Alzheimers disease.

There are contraindications, you need to consult a doctor

Read more from the original source:
What treatments can prolong the life of someone with Alzheimer's? - Pledge Times

This press release was orginally distributed by SBWire

Albany, NY -- (SBWIRE) -- 09/23/2020 -- Transparency Market Research (TMR) has published a new report on the global cell separation technology market for the forecast period of 20192027. According to the report, the global cell separation technology market was valued at ~ US$ 5 Bn in 2018, and is projected to expand at a double-digit CAGR during the forecast period.

Overview

Cell separation, also known as cell sorting or cell isolation, is the process of removing cells from biological samples such as tissue or whole blood. Cell separation is a powerful technology that assists biological research. Rising incidences of chronic illnesses across the globe are likely to boost the development of regenerative medicines or tissue engineering, which further boosts the adoption of cell separation technologies by researchers.

Expansion of the global cell separation technology market is attributed to an increase in technological advancements and surge in investments in research & development, such as stem cell research and cancer research. The rising geriatric population is another factor boosting the need for cell separation technologies Moreover, the geriatric population, globally, is more prone to long-term neurological and other chronic illnesses, which, in turn, is driving research to develop treatment for chronic illnesses. Furthermore, increase in the awareness about innovative technologies, such as microfluidics, fluorescent-activated cells sorting, and magnetic activated cells sorting is expected to propel the global cell separation technology market.

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North America dominated the global cell separation technology market in 2018, and the trend is anticipated to continue during the forecast period. This is attributed to technological advancements in offering cell separation solutions, presence of key players, and increased initiatives by governments for advancing the cell separation process. However, insufficient funding for the development of cell separation technologies is likely to hamper the global cell separation technology market during the forecast period. Asia Pacific is expected to be a highly lucrative market for cell separation technology during the forecast period, owing to improving healthcare infrastructure along with rising investments in research & development in the region.

Rising Incidences of Chronic Diseases, Worldwide, Boosting the Demand for Cell Therapy

Incidences of chronic diseases such as diabetes, obesity, arthritis, cardiac diseases, and cancer are increasing due to sedentary lifestyles, aging population, and increased alcohol consumption and cigarette smoking. According to the World Health Organization (WHO), by 2020, the mortality rate from chronic diseases is expected to reach 73%, and in developing counties, 70% deaths are estimated to be caused by chronic diseases. Southeast Asia, Eastern Mediterranean, and Africa are expected to be greatly affected by chronic diseases. Thus, the increasing burden of chronic diseases around the world is fuelling the demand for cellular therapies to treat chronic diseases. This, in turn, is driving focus and investments on research to develop effective treatments. Thus, increase in cellular research activities is boosting the global cell separation technology market.

Increase in Geriatric Population Boosting the Demand for Surgeries

The geriatric population is likely to suffer from chronic diseases such as cancer and neurological disorders more than the younger population. Moreover, the geriatric population is increasing at a rapid pace as compared to that of the younger population. Increase in the geriatric population aged above 65 years is projected to drive the incidences of Alzheimer's, dementia, cancer, and immune diseases, which, in turn, is anticipated to boost the need for corrective treatment of these disorders. This is estimated to further drive the demand for clinical trials and research that require cell separation products. These factors are likely to boost the global cell separation technology market.

According to the United Nations, the geriatric population aged above 60 is expected to double by 2050 and triple by 2100, an increase from 962 million in 2017 to 2.1 billion in 2050 and 3.1 billion by 2100.

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Productive Partnerships in Microfluidics Likely to Boost the Cell Separation Technology Market

Technological advancements are prompting companies to innovate in microfluidics cell separation technology. Strategic partnerships and collaborations is an ongoing trend, which is boosting the innovation and development of microfluidics-based products. Governments and stakeholders look upon the potential in single cell separation technology and its analysis, which drives them to invest in the development of microfluidics. Companies are striving to build a platform by utilizing their expertise and experience to further offer enhanced solutions to end users.

Stem Cell Research to Account for a Prominent Share

Stem cell is a prominent cell therapy utilized in the development of regenerative medicine, which is employed in the replacement of tissues or organs, rather than treating them. Thus, stem cell accounted for a prominent share of the global market. The geriatric population is likely to increase at a rapid pace as compared to the adult population, by 2030, which is likely to attract the use of stem cell therapy for treatment. Stem cells require considerably higher number of clinical trials, which is likely to drive the demand for cell separation technology, globally. Rising stem cell research is likely to attract government and private funding, which, in turn, is estimated to offer significant opportunity for stem cell therapies.

Biotechnology & Pharmaceuticals Companies to Dominate the Market

The number of biotechnology companies operating across the globe is rising, especially in developing countries. Pharmaceutical companies are likely to use cells separation techniques to develop drugs and continue contributing through innovation. Growing research in stem cell has prompted companies to own large separate units to boost the same. Thus, advancements in developing drugs and treatments, such as CAR-T through cell separation technologies, are likely to drive the segment.

As per research, 449 public biotech companies operate in the U.S., which is expected to boost the biotechnology & pharmaceutical companies segment. In developing countries such as China, China Food and Drug Administration (CFDA) reforms pave the way for innovation to further boost biotechnology & pharmaceutical companies in the country.

Global Cell Separation Technology Market: Prominent Regions

North America to Dominate Global Market, While Asia Pacific to Offer Significant Opportunity

In terms of region, the global cell separation technology market has been segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. North America dominated the global market in 2018, followed by Europe. North America accounted for a major share of the global cell separation technology market in 2018, owing to the development of cell separation advanced technologies, well-defined regulatory framework, and initiatives by governments in the region to further encourage the research industry. The U.S. is a major investor in stem cell research, which accelerates the development of regenerative medicines for the treatment of various long-term illnesses.

The cell separation technology market in Asia Pacific is projected to expand at a high CAGR from 2019 to 2027. This can be attributed to an increase in healthcare expenditure and large patient population, especially in countries such as India and China. Rising medical tourism in the region and technological advancements are likely to drive the cell separation technology market in the region.

Launching Innovative Products, and Acquisitions & Collaborations by Key Players Driving Global Cell Separation Technology Market

The global cell separation technology market is highly competitive in terms of number of players. Key players operating in the global cell separation technology market include Akadeum Life Sciences, STEMCELL Technologies, Inc., BD, Bio-Rad Laboratories, Inc., Miltenyi Biotech, 10X Genomics, Thermo Fisher Scientific, Inc., Zeiss, GE Healthcare Life Sciences, PerkinElmer, Inc., and QIAGEN.

These players have adopted various strategies such as expanding their product portfolios by launching new cell separation kits and devices, and participation in acquisitions, establishing strong distribution networks. Companies are expanding their geographic presence in order sustain in the global cell separation technology market. For instance, in May 2019, Akadeum Life Sciences launched seven new microbubble-based products at a conference. In July 2017, BD received the U.S. FDA's clearance for its BD FACS Lyric flow cytometer system, which is used in the diagnosis of immunological disorders.

For more information on this press release visit: http://www.sbwire.com/press-releases/cell-separation-technology-mar/release-1305278.htm

Read more from the original source:
Cell Separation Technology Market: Productive Partnerships in Microfluidics Likely to Boost the Market Growth - Press Release - Digital Journal

MINNEAPOLIS, Sept. 24, 2020 /PRNewswire/ --Today, Senator Jack Reed (D-RI) introduced the TRANSPLANT Act (S. 4762), which will provide for the timely reauthorization of the C.W. Bill Young Transplantation Program (Program). The National Marrow Donor Program(NMDP)/Be The Match applauds the leadership of Senator Reed and urges Congress to act quickly to reauthorize the Program, which expires on September 30th.

"We appreciate the leadership of the bill's sponsor Sen. Jack Reed (D-RI), and the bipartisan support of the bill's lead cosponsors Sens. Richard Burr (R-NC), Tina Smith (D-MN), and Tim Scott (R-SC)," said NMDP/Be The Match Chief Policy Officer Brian Lindberg.

The Program provides access to live-saving bone marrow, peripheral blood stem cell (PBSC) and cord blood transplants to those living with one of the more than 70 blood cancers or blood disorders for which a transplant is the only curative option.

Since its inception in the mid-1980s, NMDP/Be The Match has been entrusted by Congress to operate the Program and has facilitated more than 100,000 life-saving transplants to give patients with otherwise fatal blood cancers or blood disorders a second chance at life.

The COVID-19 pandemic with increasingly restrictive travel bans, border closings, and the declining availability of scheduled commercial passenger aviation service on international and domestic routes - has created unprecedented obstacles for NMDP/ Be The Match in delivering life-saving cells to the patients who need them.

This new reality creates a web of increasingly complex challenges in facilitating the timely collection and delivery of bone marrow products to patients who are in the midst of treatment protocols or whose conditions have deteriorated to the point that a bone marrow transplant is the only course of treatment that will save their life.

"During the pandemic, we have been able to mitigate any disruption to our mission and complete more than 3,000 lifesaving therapies without missing a single delivery," continued Lindberg. "We have been able to do that, in large part, because of our designation in Federal law as the nation's bone marrow registry. Unfortunately, that designation will lapse with the expiration of our Federal authorization on September 30, 2020."

This designation has helped Be The Match to care for our patients during the pandemic by:

"Our ability to call upon our Federal partners in these extreme circumstances, and our standing in the eyes of foreign governments where we must operate, could be compromised after September 30th if our authorization expires and we are no longer technically designated as the Nation's registry," said Lindberg. "This could put at risk our ability to continue to ensure the timely delivery of life-saving cellular products to otherwise terminally ill patients here and throughout the world.

"Ensuring access to cellular therapy is critical for the patients we serve," continued Lindberg. "Congress must move immediately to reauthorize these programs to expand the number of adult volunteer donors and cord blood units on the national registry so that every American who needs a transplant can find a match."

About Be The MatchFor people with life-threatening blood cancerslike leukemia and lymphomaor other diseases, a cure exists. Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant. People can contribute to the cure as a member of the Be The Match Registry, financial contributor or volunteer. Be The Match provides patients and their families one-on-one support, education, and guidancebefore, during and after transplant.

Be The Match is operated by the National Marrow Donor Program (NMDP), a nonprofit organization that matches patients with donors, educates health care professionals and conducts research through its research program, CIBMTR (Center for International Blood and Marrow Transplant Research), so more lives can be saved. To learn more about the cure, visit BeTheMatch.org or call 1 (800) MARROW-2.

SOURCE Be The Match

https://bethematch.org

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National Marrow Donor Program/Be The Match Urges Congress to Quickly Pass Reauthorization Bill to Protect Access to Life Saving Bone Marrow and Cord...

Latest Research Study on Stem Cell Therapy Market published by The Insight Partners, offers a detailed overview of the factors influencing the global business scope. The research report provides deep insights into the global market revenue, parent market trends, macro-economic indicators, and governing factors, along with market attractiveness per market segment. The report provides an overview of the growth rate of the Stem Cell Therapy market during the forecast period, i.e., 20202027. Most importantly, the report further identifies the qualitative impact of various market factors on market segments and geographies. The research segments the market on the basis of product type, application, technology, and region. To offer more clarity regarding the industry, the report takes a closer look at the current status of various factors including but not limited to supply chain management, niche markets, distribution channel, trade, supply, and demand and production capability across different countries. Some of the key players profiled in the study are MEDIPOST, Pharmicell Co., Inc., RichSource, BioTime Inc. (Lineage Cell Therapeutics, Inc.), Mesoblast Limited, Holostem Terapie Avanzate Srl, U.S. Stem Cell, Inc., Caladrius Biosciences, Inc., TiGenix NV, AlloSource, etc.

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

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Stem Cell Therapy Market In-Depth Analysis

Stem cells are preliminary body cells from which all other cells with specialized functions are generated. Under controlled environment in the body or a clinical laboratory, these cells divide to form more cells called daughter cells. Due to the advent of modern health science, these cells play a major role in understanding the occurrence of diseases, generation of advanced regenerative medicines, and drug discovery. There are certain sources such as embryo, bone marrow, body fats, and umbilical cord blood amongst others, where stem cells are generated. The global stem cell therapy market is driven by factors such asincreasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines. However, high cost related with stem cell therapy is likely to obstruct the growth of the stem cell therapymarket during the forecast period. The growing research and development activities in Asia Pacific region is expected to offer huge growth opportunity for stem cell therapy market.

End User Insights

Based on end user, the Stem Cell Therapy market is segmented into academic and research institutes, hospitals and specialty clinics. The academic and research institutes held the largest share of end user segment in the global market and is expected to grow at the fastest rate during the forecast period.

Key Benefits

The report profiles the key players in the industry, along with a detailed analysis of their individual positions against the global landscape. The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Stem Cell Therapy market. The researcher provides an extensive analysis of the Stem Cell Therapy market size, share, trends, overall earnings, gross revenue, and profit margin to accurately draw a forecast and provide expert insights to investors to keep them updated with the trends in the market.

Competitive scenario:

The study assesses factors such as segmentation, description, and applications of Stem Cell Therapy industries. It derives accurate insights to give a holistic view of the dynamic features of the business, including shares, profit generation, thereby directing focus on the critical aspects of the business.

Scope of the Report

The research on the Stem Cell Therapy market focuses on mining out valuable data on investment pockets, growth opportunities, and major market vendors to help clients understand their competitors methodologies. The research also segments the Stem Cell Therapy market on the basis of end user, product type, application, and demography for the forecast period 20212027. Comprehensive analysis of critical aspects such as impacting factors and competitive landscape are showcased with the help of vital resources, such as charts, tables, and infographics.

Global Stem Cell Therapy Market By Type

Global Stem Cell Therapy Market By Treatment

Global Stem Cell Therapy Market ByApplication

Global Stem Cell Therapy Market By End User

Promising Regions & Countries Mentioned in TheStem Cell Therapy Market Report:

Major highlights of the report:

All-inclusive evaluation of the parent market

Evolution of significant market aspects

Industry-wide investigation of market segments

Assessment of market value and volume in past, present, and forecast years

Evaluation of market share

Study of niche industrial sectors

Tactical approaches of market leaders

Lucrative strategies to help companies strengthen their position in the market

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Massive Growth Seen in Stem Cell Therapy Market 2020-2027 | In Depth Analysis with Top Key Players RichSource, Mesoblast Limited, TiGenix NV,...

Los Angeles, United State, The report titledGlobal Hematopoietic Stem Cell Transplantation (HSCT) Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Hematopoietic Stem Cell Transplantation (HSCT) market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Hematopoietic Stem Cell Transplantation (HSCT) market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Hematopoietic Stem Cell Transplantation (HSCT) market is carefully analyzed and researched about by the market analysts.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

Top Key Players of the Global Hematopoietic Stem Cell Transplantation (HSCT) Market : Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT)

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The Essential Content Covered in the Global Hematopoietic Stem Cell Transplantation (HSCT) Market Report :

Top Key Company Profiles.Main Business and Rival InformationSWOT Analysis and PESTEL AnalysisProduction, Sales, Revenue, Price and Gross MarginMarket Size And Growth RateCompany Market Share

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Segmentation By Product:, Allogeneic, Autologous Hematopoietic Stem Cell Transplantation (HSCT)

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Segmentation By Application: Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

In terms of region, this research report covers almost all the major regions across the globe such asNorth America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. WhileHematopoietic Stem Cell Transplantation (HSCT) Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets.Hematopoietic Stem Cell Transplantation (HSCT) Market in South, America region is also expected to grow in near future.

Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?*Which segment is currently leading the market?*In which region will the market find its highest growth?*Which players will take the lead in the market?*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Hematopoietic Stem Cell Transplantation (HSCT) market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

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Table of Contents

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Hematopoietic Stem Cell Transplantation (HSCT) Revenue1.4 Market by Type1.4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Growth Rate by Type: 2020 VS 20261.4.2 Allogeneic1.4.3 Autologous1.5 Market by Application1.5.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Share by Application: 2020 VS 20261.5.2 Peripheral Blood Stem Cells Transplant (PBSCT)1.5.3 Bone Marrow Transplant (BMT)1.5.4 Cord Blood Transplant (CBT)1.6 Study Objectives1.7 Years Considered 2 Global Growth Trends2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Perspective (2015-2026)2.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Growth Trends by Regions2.2.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Share by Regions (2015-2020)2.2.3 Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Hematopoietic Stem Cell Transplantation (HSCT) Market Growth Strategy2.3.6 Primary Interviews with Key Hematopoietic Stem Cell Transplantation (HSCT) Players (Opinion Leaders) 3 Competition Landscape by Key Players3.1 Global Top Hematopoietic Stem Cell Transplantation (HSCT) Players by Market Size3.1.1 Global Top Hematopoietic Stem Cell Transplantation (HSCT) Players by Revenue (2015-2020)3.1.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Revenue Market Share by Players (2015-2020)3.1.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Ratio3.2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Hematopoietic Stem Cell Transplantation (HSCT) Revenue in 20193.3 Hematopoietic Stem Cell Transplantation (HSCT) Key Players Head office and Area Served3.4 Key Players Hematopoietic Stem Cell Transplantation (HSCT) Product Solution and Service3.5 Date of Enter into Hematopoietic Stem Cell Transplantation (HSCT) Market3.6 Mergers & Acquisitions, Expansion Plans 4 Market Size by Type (2015-2026)4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Type (2015-2020)4.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Type (2021-2026) 5 Market Size by Application (2015-2026)5.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020)5.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)6.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in North America (2019-2020)6.3 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)6.4 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 7 Europe7.1 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)7.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Europe (2019-2020)7.3 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)7.4 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 8 China8.1 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)8.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in China (2019-2020)8.3 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)8.4 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 9 Japan9.1 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)9.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Japan (2019-2020)9.3 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)9.4 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 10 Southeast Asia10.1 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)10.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)10.4 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 11 India11.1 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)11.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in India (2019-2020)11.3 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)11.4 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 12 Central & South America12.1 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)12.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Central & South America (2019-2020)12.3 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)12.4 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 13 Key Players Profiles13.1 Regen Biopharma Inc13.1.1 Regen Biopharma Inc Company Details13.1.2 Regen Biopharma Inc Business Overview13.1.3 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.1.4 Regen Biopharma Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020))13.1.5 Regen Biopharma Inc Recent Development13.2 China Cord Blood Corp13.2.1 China Cord Blood Corp Company Details13.2.2 China Cord Blood Corp Business Overview13.2.3 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.2.4 China Cord Blood Corp Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.2.5 China Cord Blood Corp Recent Development13.3 CBR Systems Inc13.3.1 CBR Systems Inc Company Details13.3.2 CBR Systems Inc Business Overview13.3.3 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.3.4 CBR Systems Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.3.5 CBR Systems Inc Recent Development13.4 Escape Therapeutics Inc13.4.1 Escape Therapeutics Inc Company Details13.4.2 Escape Therapeutics Inc Business Overview13.4.3 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.4.4 Escape Therapeutics Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.4.5 Escape Therapeutics Inc Recent Development13.5 Cryo-Save AG13.5.1 Cryo-Save AG Company Details13.5.2 Cryo-Save AG Business Overview13.5.3 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.5.4 Cryo-Save AG Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.5.5 Cryo-Save AG Recent Development13.6 Lonza Group Ltd13.6.1 Lonza Group Ltd Company Details13.6.2 Lonza Group Ltd Business Overview13.6.3 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.6.4 Lonza Group Ltd Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.6.5 Lonza Group Ltd Recent Development13.7 Pluristem Therapeutics Inc13.7.1 Pluristem Therapeutics Inc Company Details13.7.2 Pluristem Therapeutics Inc Business Overview13.7.3 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.7.4 Pluristem Therapeutics Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.7.5 Pluristem Therapeutics Inc Recent Development13.8 ViaCord Inc13.8.1 ViaCord Inc Company Details13.8.2 ViaCord Inc Business Overview13.8.3 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.8.4 ViaCord Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.8.5 ViaCord Inc Recent Development 14 Analysts Viewpoints/Conclusions 15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Hematopoietic Stem Cell Transplantation (HSCT) Market is Thriving Worldwide with Top Companies like: China Cord Blood Corp, CBR Systems Inc, Escape...

Alyssa Mulvey in the Great North Children's Hospital at the RVI.

The total brought in by the man who walked 21 laps around Elizabethan walls earlier this month reached 2,000 earlier this week.

He was accompanied by Stewart Brown for the whole 26.2-mile marathon distance and family, friends and other supporters did some of the laps during the event in aid of wards in the Great North Children's Hospital (GNCH) at the RVI in Newcastle.

On his fund-raising page, Derek who lives in Highcliffe, Spittal specifically mentioned two local girls from Berwick (Alyssa Mulvey and Kelis Bloomfield) who are currently receiving treatment at the GNCH.

Alyssa has been poorly since birth and was diagnosed with A20 haploinsufficiency, a very rare auto-immune condition, when she was 10.

She has been on ward 3, a bone marrow transplant ward that deals with children who were born with zero immune systems or severe immune conditions. She had recovered well from a stem cell transplant last year, using stem cells donated by her dad Shane, but then contracted Covid-19.

Fortunately, Alyssa made an excellent recovery. However, a check-up weeks later revealed her blood pressure was extremely high and tests found that she had a serious kidney disease called thrombotic microangiopathy (TMA).

Although the condition was not caused by Covid-19, medics believe the virus ignited it in her system and that it had been dormant beforehand. She has now been at the GNCH for 21 consecutive weeks.

Kelis receives treatment on ward 2a. She has eosinophilic esophagitis (EoE), which is a chronic allergic inflammatory disease of the esophagus. She is allergic to things such as grass and pollen, which can cause respiratory failure.

Ward 2a specialises in respiratory and long-term ventilation, and diabetes.

Derek has previously supported the GNCH by doing the Great North Run half marathon his way of giving back to the team who saved the life of his daughter, Saskia, in 2018.

As the run was not held this year due to the coronavirus pandemic, he still wanted to do something in 2020 and so came up with the local challenge, which took place earlier this month before the extra local and national restrictions were announced.

He said: We set off just before 6am and after 8am, Deva (his wife) and Saskia joined us and walked the opposite way. Friends and supporters did some of the laps with us.

"We kept a brisk pace and when we had a break, we had a bacon roll and coffee courtesy of The Mule on Rouge.

"Many of the visitors on the day asked us why were doing the walk and a combination of visitors, friends and supporters saw a lot of people put money into the charity bucket that we had with us when we counted it later, the total in it was 255.

In the afternoon, Deva and Saskia completed half marathon distance and with six laps to go we were joined by the Plundering Pirates of the North East, dressed in costume, who raised money for their own charities. The last lap was done with Stewarts wife Amanda and their children, Mathew and Mia. Me and Stewart were pleased to complete the marathon distance in 10 hours.

Also thanks to all those who have donated online gf.me/u/yny5p2 its great that Ill be giving more than 1,000 to the two wards.

Alyssas mum Kirstie said that her daughter, 14, has infusions meds going in through a central line every day and has the dialysis procedure four to five times a week.

In relation to Dereks fund-raising, she said: He is amazing and we think he is an absolute superhero for helping to raise funds for and awareness of these wards.

The money will be vital for them because they are not as well known as others such as the cancer wards.

Other support for Alyssa and her family includes Kirsties best friend Julie Newton organising a raffle (1,000) and piping events on Thursday evenings at Mordington Avenue until last week (about 700 in total from the bucket donations each week).

Denise Lody from Facebook group Isolation Berwick upon Tweed and Surrounding Area set-up a GoFundMe page that raised 2,651.

Sean Ryan and Dave Smith of 2SPT raised 2,623.55 by running private physical training sessions through the lockdown period.

Further funds came from a beautiful cake raffled by Ashley McKnight and Berwick landscape postcards sold by Julie.

Kirstie said: The response has been unbelievable. Alyssa said to me why are they doing this for me, but I reminded her of how brave she is and told her how much people love her and they wanted to show just how much they care.

We were unable to speak in depth with Kelis mum Stacey, but she did send us a message that said: What Derek has done for these wards is absolutely amazing.

"Kelis has helped out by doing an online raffle, which raised 70 to the cause.

Originally posted here:
Marathon effort in aid of hospital wards - Berwick Advertiser

Medical skin care products are used for beautifying or to address some other skin care problems. The cosmetic industry is booming and skin care forms a very huge part of this industry. The aesthetic appearance is so important that people spend a lot on skin care products and treatment. People being more technologically aware of the various new skin care products trending in the market. In addition to the aesthetic application, the medical skin care products are also used to address issues such as acne, pimples or scars.

Medical Skin Care Products Market: Drivers and Restraints

The medical skin care products is primarily driven by the need of natural based active ingredients products which are now trending in the market. Consumers demand medical skin care products which favor health and environment. Moreover, the consumers are updated with the trends so that various companies end up providing such products to satisfy the customers. For instance, a single product face mask has thousands of different variants. This offers consumers different options to select the product depending on the skin type. Moreover, the market players catering to the medical skin care products are offering products with advanced technologies. For instance, Santinov launched the CICABEL mask using stem cell material based on advanced technologies. The stem cells used in the skin care product helps to to protect and activate the cells and promote the proliferation of skin epidermal cells and the anagenesis of skin fibrosis.

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Medical Skin Care Products Market: Segmentation

On the basis of product type the medical skin care products market can be segmented as:

On the basis of application, the medical skin care products market can be segment as:

On the basis of distribution channel, the medical skin care products market can be segment as:

Medical Skin Care Products Market: Overview

Medical skin care products are used to address basic skin problems ranging from acne to scars. There are various advancements in the ingredients used to offer skin care products to the consumers. For instance, the use of hyaluronic acid and retinoids is the latest development in the industry. The anti-aging creams are at the forefront as the help treating issues such as wrinkles, scars, acne, and sun damage. Another, product in demand is the probiotic skincare which include lactobacillus and bifidobacterium.

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Medical Skin Care Products Market: Region-wise Outlook

In terms of geography, medical skin care products market has been divided into five regions including North- America, Asia- Pacific, Middle-East & Africa, Latin America and Europe. North America dominated the global medical skin care products market as international players are acquiring domestic companies to make their hold strong in the U.S. LOral is accelerating its U.S. market by signing a definitive agreement with Valeant Pharmaceuticals International Inc. to acquire CeraVe, AcneFree and Ambi skin-care brands for US$ 1.3 billion. The acquisition is expected LOreal to get hold of the brands in the price-accessible segment. Asia Pacific is expected to be the fastest growing region owing to the increasing disposable income and rising awareness towards the skin care products.

Medical Skin Care Products Market: Key Market Participants

Some of the medical skin care products market participants are Avon Products Inc., Beiersdorf AG, Colgate-Palmolive Company, Kao Corporation, LOral S.A., Procter & Gamble, Shiseido Company, The Estee Lauder Companies Inc., Unilever PLC, Revlon, Clinique Laboratories, llc., Murad, LLC., SkinCeuticals, RMS Beauty, J.R. Watkins and 100% PURE.

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Europe to Show Consistency in the Medical Skin Care market from 2017 to 2025 - Crypto Daily

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy - Business Wire

NEW YORK, Sept. 23, 2020 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (the "Company" or "Actinium") today announced that it has successfully completed the first dosing cohort in the Actimab-A and venetoclax combination, multi-center Phase 1 trial for patients with Relapsed or Refractory ("R/R") Acute Myeloid Leukemia (AML) age 18 and above. All patients from the first dosing cohort (0.50 uCi/kg of Actimab-A) completed treatment and cleared their initial safety evaluation, thus allowing the study to proceed to the second dose cohort of 1.0 uCi/kg Actimab-A added to venetoclax. In a poster presentation at the American Association of Cancer Research (AACR) Annual Meeting 2019, Actimab-A was shown to be synergistic with venetoclax in venetoclax resistant cell lines, by depleting MCL-1, a protein shown to mediate resistance to venetoclax. The ongoing Phase 1 study was planned to replicate this synergy in a clinical setting. Actinium plans to report study proof of concept results in 2021.

Venetoclax is a B-Cell Lymphoma 2 (BCL-2) inhibitor jointly developed and marketed by AbbVie and Genentech that is approved in combination with hypomethylating agents ("HMAs") for patients with AML. The use of venetoclax has become widespread in the treatment of fit and unfit patients with R/R AML following its inclusion in the recently expanded National Comprehensive Cancer Network ("NCCN") guidelines. Actinium's preclinical research has demonstrated that by adding Actimab-A to venetoclax, the targeted internalized radiation from Actimab-A can deliver potent AML cell killing, as well as effectively deplete MCL-1 levels. The overexpression of MCL-1, a member of the BCL-2 family which venetoclax does not inhibit, promotes resistance to venetoclax. Thus, Actimab-A reverses resistance to venetoclax and has independent anti-leukemic activity mediated by CD33 as well.

"We are pleased to confirm that the second combination trial in our CD33 program is advancing through the dose escalation study as planned. Despite approval in multiple blood cancers, including AML, most AML patients are not cured with venetoclax regimens and eventually relapse. Based on the preclinical data, synergy with venetoclax and Actimab-A should lead to higher remission rates in R/R AML," said Dr. Mark Berger, Actinium's Chief Medical Officer. "We continue to generate promising data from our broader combination program. For example, the Actimab-A combination trial with chemotherapy agent CLAG-M increased the complete response rate compared to CLAG-M alone in R/R AML patients by 60%. We expect to complete the proof of concept Actimab-A venetoclax combination trial in 2021."

This Phase 1 study is a multicenter, open label trial of Actimab-A added to venetoclax for patients with CD33 positive R/R AML. The study will continue to enroll patients that have been previously treated with venetoclax as well as venetoclax nave patients. Gary Schiller, MD, Professor, Hematology-Oncology and Director, Hematologic Malignancy/Stem Cell Transplant Program at the UCLA Medical Center is the Principal Investigator for this study. The trial is also active at the University of Louisville.

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "We continue to advance the CD33 program for fit and unfit R/R AML patients as there is still a significant unmet need despite multiple recently approved agents. These therapeutic agents are not curative and patients continue to experience low response rates and/or high relapse rates. Our CD33 program, which also includes the Actimab-A CLAG-M combination trial, is anchored in leveraging mechanistic synergies of Actimab-A with approved or novel therapeutic agents in order to improve patient outcomes. We look forward to multiple clinical trial updates by year-end from our three ongoing trials in R/R AML, including our Iomab-B SIERRA Phase 3 pivotal trial."

Rationale for Actimab-A Venetoclax Combination Trial

This Phase 1/2 trial is a multicenter, open label trial of Actimab-A (lintuzumab-Ac225) added to venetoclax for patients with CD33 positive relapsed/refractory (R/R) Acute Myeloid Leukemia. The Phase 1 portion of the study is designed to determine the maximum tolerated dose (MTD) of Actimab-A added to venetoclax for R/R AML. The Phase 2 portion of the trial will assess the percentage of patients with CR, CRh, or Overall Response (CR + CRh), up to six months after the start of the treatment without receiving other AML therapies. The trial will enroll R/R AML patients who have been treated with venetoclax as well as venetoclax-nave patients. At the 1.0 uCi/kg dose, Actimab-A is administered on Day 1 of each cycle for four cycles and venetoclax is taken on Days 1-21 of each cycle for up to 4 cycles. Each cycle is 28 days, with a potential to expand to 42 days to allow for full hematologic recovery. Gary Schiller, MD, Professor, Hematology-Oncology and Director, Hematologic Malignancy/Stem Cell Transplant Program at the UCLA Medical Center is the Principal Investigator for this study.

More information on the clinical trial design is available at clinicaltrials.gov (NCT03867682).

About Actinium's CD33 Program (Actimab-A)

Antibody Radiation Conjugate (ARC) Actimab-A targets the CD33 antigen that is expressed on virtually all AML cells with the antibody lintuzumab which delivers potent alpha radiation via its Actinium-225 radioisotope payload. Blood cancers like AML are highly sensitive to radiation but cannot treated with the current standard of external beam delivery because the disease is too widespread throughout the body. The combination of targeted radiation with Actimab-A potentially allows for greater cancer cell death than a standalone chemotherapy regimen such as CLAG-M or venetoclax, which are frequently used in the treatment of fit and unfit patients with relapsed or refractory AML per National Comprehensive Cancer Network (NCCN) guidelines. Prior clinical results in over 100 patients treated with Actimab-A, including a Phase 1/2 trial of 58 patients, demonstrated a safety profile with minimal non-hematologic toxicities and an unmatched ability to deliver attenuated doses of radiation internally to CD33 expressing cancer cells. In the Phase 1/2 trial, Actimab-A as a single agent produced a 69% remission rate (CR, CRi, CRp) at high doses in patients with newly diagnosed AML but Actinium elected to pursue low dose combination trials for therapeutic development based on observed myelosuppression. In the Actimab-A CLAG-M Phase 1 combination trial, the second cohort with CLAG-M plus the 0.50 uCi/kg dose showed that 86% (6/7) of patients achieved complete remission (CR/CRi) after receiving the 0.50 uCi/kg dose of Actimab-A. This is a nearly 60% increase over the remission rate reported in a trial of seventy-four patients with relapsed or refractory AML who received CLAG-M alone. The company expects trial results, including the third dose cohort, in 2020. The Actimab-A Venetoclax Phase 1 trial continues to enroll patients in a maximum tolerated dose and expects to announce proof-of-concept results in 2021.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

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

Contacts:

Investors:Clayton RobertsonActinium Pharmaceuticals, Inc.crobertson@actiniumpharma.com

Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

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SOURCE Actinium Pharmaceuticals, Inc.

Company Codes: AMEX:ATNM

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Actinium Pharmaceuticals Successfully Completes First Dosing Cohort in the Phase 1 Study of Actimab-A and Venetoclax Combination Therapy in...

Imagine if you could print yourself a new skin. Wrinkles and wounds begone with the flick of a switch. I appreciate that this sounds farfetched, but I have been looking into bioprinting and anti-aging applications are not so far off into the future.

So what is bioprinting?

Bioprinting is a type of 3D printing that uses actual cells and other biological materials as inks to fabricate 3D biological structures. Bioprinted materials have the potential to repair damaged organs, cells, and tissues in the human body (source). All sorts of cell types are being studied for bioprinting including stem cells, muscle cells, and endothelial cells. When printed, each layer of cells will cool and stick to one another (due to the collagen), creating a solid, stable structure.

Researchers have used bioprinting to introduce cells to help repair the heart after a heart attack as well as deposit cells into wounded skin.

A couple of years ago, researchers in Singapore mimicked human skin pigmentation using a 3-D bioprinter. he pigmented skin was constructed in vitro using three types of skin cells: keratinocytes, melanocytes and fibroblasts. Apparently, this was a breakthrough for the making biometic skin because while the skin itself isnt a problem, uniform pigmentation had been a real challenge.

A review of scientific literature, covering studies that mostly used collagen and hydrosol gels as the biometric ink, found that there had been a positive impact of natural bioinks in promoting wound healing. These have all been animal tests, but some researchers think that human wound healing with bioink will happen in the next 3-5 years.

A clue that we could see cosmetic skincare applications one day came when LOreal partnered with a 3-D bioprinting company to develop 3-D printed tissue for product evaluation and research (source).

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Is Bioprinting the Future of Anti-aging Skincare? - Truth In Aging

Later that night, Fiona was searching her godfather's books on Amazon when she came across an unusual review.

"Dr Walker was my biological father and sperm donor," it read, and the commenter was trying to find out further information about him.

Fiona, curious, emailed the reviewer, Anne: She explained how she had letters and photographs, and she'd be willing to share them.

Through back-and-forth she'd discovered Dr Walker had assisted more than 100 families by using his own sperm.

By this point, Fiona was almost certain she was the result of this.

"When I fished out his letters, I found a drivers license of his that he had given me from when he was 16. And I showed it to my husband and said, 'Who does this look like?' and he just laughed, because it looks exactly like our youngest daughter," Fionasaid on SBS Insight.

"I went and had a chat to my mum and I said, 'Look, something's going on here and I'd like you to tell me the truth.'"

Fiona's mother told her they'd used a sperm donor. She said Dr Walker was her gynaecologist, and sperm donations usually come from medical students.

Fiona, then 53, asked her mother if Dr Walker was the donor, but her mother couldn't give her a straight answer.

Fiona and her mother. Image: Supplied.

She told Insight she was devastated.

"It made a lot ofsense, but at the same timeI was really angry that I'd been lied to. It was a bit of a rollercoaster, I'd be thinking it was prettycool one moment and then prettyhorrible the next moment. It's been like that eversince," she said.

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'Who does this look like?' Fiona was scrolling online when she discovered a family secret. - Mamamia