SAN FRANCISCO, Dec. 17, 2019 /PRNewswire/ -- The genome of human cells looks a lot like a tangled ball of yarn, with tightly wound clumps from which myriad loose strands escape and loop out. But there is order to this tangleand growing evidence that the genome's 3D architecture influences the activity of its genes. Understanding the rules that control gene activity has been the object of a long collaboration between Gladstone investigators Deepak Srivastava, Benoit Bruneau, Katherine Pollard, Bruce Conklin, and Nevan Krogan, and their UC San Francisco (UCSF) partner Brian Black. Together, they have already found many key regulators of gene activity in the heart.

Now, their collaboration has received a strong shot in the arm from the National Institute of Health with the recent award of a Program Project Grant totaling $13 million between the labs for the next five years.

With this new support, the researchers will carry out a comprehensive probe into gene activity in heart cells and its intersection with the genome's 3D organization during heart formation.

"It is truly gratifying to see our long collaboration supported in this way by the National Institute of Health,"says Srivastava, president of Gladstone Institutes and project leader on this multi-investigator grant. "This funding will allow us to dig deep into processes that are fundamental to heart cell biology, but that will also directly inform our efforts to design therapies for congenital heart disease, heart failure, and other heart diseases."

Heart failure is the most common cause of death in adults, and congenital heart defects the most common form of birth defects. These defects have been traced to mutations in a number of proteins that regulate gene activity in heart cells, including the proteins at the core of the researchers' new proposal.

"However, the investigation of the 3D organization of the genome is a relatively new area, particularly in the heart," says Srivastava, who is also a pediatric cardiologist and has devoted much of his career to understanding heart formation and congenital heart defects.

The work outlined in this grant is therefore expected to yield novel insight into heart disease and spur the design of new therapies. It will also help the researchers improve their ability to coax human cells into becoming various types of heart cells. This technology could eventually be used to regenerate failing heart tissue.

Gladstone Senior InvestigatorBruce Conklinwill lend his expertise in cardiac stem cell biology and CRISPR gene-editing technology to the project.

The researchers' plan is to correlate gene activity and genome organization at the whole-genome scale and during multiple stages of heart formation. This will require enormous technological power. It will also require massive computing power and statistical analysis to store and sift through the large data sets the group will generate.

But the team is well-positioned to take on this challenge.

"Our studies are facilitated by extraordinary new technology,"says Bruneau, also a cardiovascular development specialist and the director of the Gladstone Institute of Cardiovascular Disease.

The $13 million proposal will leverage Srivastava, Bruneau, and Black's deep understanding of heart development and disease, and enlist the state-of-the-art technologies and analytic tools that Pollard and Krogan have developed to collect and analyze information about biological networks on a grand scale.

"Our team combines a remarkable array of expertise and technologies," says Srivastava, who is also director of the Roddenberry Stem Cell Center at Gladstone. "It would be impossible for any one or two labs in isolation to pursue the complex goals we set out to achieve with this project."

Dynamic Protein Networks

The project focuses on a small set of proteins the team has previously shown to be crucial for the formation of a functional heart. These proteins, known as transcription factors, activate or silence genes by binding to specific DNA sequences in the genes' vicinity.

The scientists have shown that cardiac transcription factors can associate with each other and with other proteins. "Depending on the associations they form, they turn genes on, off, or somewhere in between, and different types of heart cells may form," says Black.

But for a transcription factor to turn a gene on or off, it needs to access the gene's DNA sequence. That's not as easy as it sounds, as much of the genome is wound up in tight coils that give no foothold to transcription factors.

Bruneau's team studies proteins that modulate the accessibility of DNA sequences along the genome, a process known as chromatin remodeling. These proteins unspool segments of the genome from the tightly wound coils, opening up stretches of DNA that transcription factors can bind.

Like transcription factors, chromatin remodeling proteins associate with each other and with other proteins, forming associations that vary depending on the cell type or the stage of heart formation.

Interestingly, Srivastava's group recently discovered that cardiac transcription factors may have long-range effects on the 3D organization of the genome. The genome is housed in a separate compartment of the cell, a spherical structure called the nucleus. Srivastava's team found that cardiac transcription factors may pull genome loops all the way to proteins lining the edges of the nucleus.

The picture that emerges from these findings is that of a vast network of proteins that coordinate gene activity and genome architecture, and change as the heart forms.

Now the researchers want to know how these networks form, how many proteins they entail, and what genes they affect.

Dynamic Lab Partnerships

To answer these questions, the team will analyze the associations between cardiac transcription factors, chromatin remodeling proteins, and their various partners during heart development. They will pair this analysis with a genome-wide survey of the genes these proteins target and of these genes' activity.

"Our overarching goal is to understand all the levels of gene regulation in developing hearts, from genes and transcription factors to chromatin remodeling and to genome organization within the nucleus," says Bruneau, who is also a professor of pediatrics at UCSF.

The researchers will use a battery of sophisticated techniques to capture the complexes that proteins form with each other or with DNA sequences and to record which genes are active or inactive in different types of heart cells.

They will leverage various models of heart development, including human induced pluripotent stem cells (hiPS cells) that can give rise to heart tissue in the dish, or cells from the developing heart of mouse embryos. They will also use CRISPR technology and other genetic tools to insert mutations in heart cells and evaluate the impact of these mutations on the protein-genome networks.

Their success will depend on high-throughput data collection and analysis, and powerful statistics to guarantee the validity of the findings. That's where Krogan and Pollard come in.

Krogan's labwill contribute technology his lab developed to determine how proteins interact with one another in the celland how those interactions affect the interaction of proteins with DNA.

Pollard's groupwill devise statistical methods to rigorously analyze the protein networks and gene activity profiles the researchers uncover through the lens of genetic causes of heart disease.

"The biggest challenge will be to develop novel computational methods, including artificial intelligence tools," says Pollard, who directs the Gladstone Institute for Data Science and Biotechnology. "This is the first time that scientists will integrate such diverse kinds of data to understand a disease."

Together, these tools will allow the researchers to reliably identify connections between protein networks and gene activity at all stages of heart formation, in the context of disease or healthy heart formation.

"This project crystallizes a more than a decade-long collaboration across our labs, with a laser focus on fundamental concepts of gene regulation," says Bruneau.

"We will learn how these concepts apply to the heart and to heart diseases," he adds, "but we think they will also be relevant to other organs and sets of diseases."

Media Contact:Megan McDevittmegan.mcdevitt@Gladstone.ucsf.edu

Related Images

team-of-researchers-who-received.jpg Team of Researchers who Received the Grant New funding from the NIH fuels collaboration between UCSF's Brian Black and Gladstone's Deepak Srivastava, Benoit Bruneau (front row, left to right), Katie Pollard, Bruce Conklin (back row, left to right), and Nevan Krogan (not shown).

Related Links

Gladstone Release

SOURCE Gladstone Institutes

https://gladstone.org

Read the original post:
$13 Million Grant to Probe the Genome of Heart Cells - PRNewswire

Cardiac Stem Cells Comments Off on $13 Million Grant to Probe the Genome of Heart Cells – PRNewswire | December 18th, 2019

NEW DELHI: In a first, researchers, including one of Indianorigin, have reprogrammed the human bodys fat cells into those similar to the hearts pacemaker cells which control heartbeat by creating rhythmic electrical impulses an advance that may lead to new therapies for cardiac failure. The study, published in the Journal of Molecular and Cellular Cardiology, noted that the new pacemaker-like cell may become a useful alternative treatment for heart conduction system disorders, and to bridge the limitations of current treatments such as artificial pacemakers. '; var randomNumber = Math.random(); var isIndia = (window.geoinfo && window.geoinfo.CountryCode === 'IN') && (window.location.href.indexOf('outsideindia') === -1 ); console.log(isIndia && randomNumber Artificial pacemakers need to be regularly examined and over time can stop working properly. In the study, researchers, including Suchi Raghunathan from the University of Houston, tweaked unspecialised stem cells to turn them into conducting cells of the heart that could carry electrical current. Batteries will die. This biologic pacemaker is better able to adapt to the body and would not have to be maintained by a physician, said study co-author Bradley McConnell.

Read the original here:
Human fat cells tweaked to function like a pacemaker - Times of India

Cardiac Stem Cells Comments Off on Human fat cells tweaked to function like a pacemaker – Times of India | December 18th, 2019

Transparency Market Research (TMR)has published a new report on the globalcell separation technology marketfor the forecast period of 20192027. According to the report, the global cell separation technology market was valued at ~US$ 5 Bnin 2018, and is projected to expand at a double-digit CAGR during the forecast period.

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 asstem cellresearch 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.

Request a Brochure of Cell Separation Technology Market Report

https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=1925

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 reach73%, 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.

For More Actionable Insights into The Competitive Landscape of Cell Separation Technology Market , Pre Book This Report

https://www.transparencymarketresearch.com/checkout.php?rep_id=1925&ltype=S

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 Alzheimers, 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.

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 includeAkadeum 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.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

ContactTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY 12207United StatesTel:+1-518-618-1030USA Canada Toll Free:866-552-3453Email:sales@transparencymarketresearch.comWebsite:http://www.transparencymarketresearch.com

More:
Cell Separation Technology Market is Estimated to Record Highest CAGR by 2027 - Techi Labs

Cardiac Stem Cells Comments Off on Cell Separation Technology Market is Estimated to Record Highest CAGR by 2027 – Techi Labs | December 18th, 2019

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

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.Europe and North America spearheaded the market as of 2016, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

Get More Information:https://www.trendsmarketresearch.com/report/sample/3184

In 2016, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy.

Request For Table of Contents:https://www.trendsmarketresearch.com/report/requesttoc/3184

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

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

Report Description:https://www.trendsmarketresearch.com/report/bone-marrow-processing-system-market

See the original post:
Bone Marrow Processing System Market Expected to Witness an Imperishable Growth over 2025 - Guru Online News

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing System Market Expected to Witness an Imperishable Growth over 2025 – Guru Online News | December 18th, 2019

Gene therapy is fighting to enter mainstream medicine. With sickle cell disease, the fight is heating up.

Roughly two years ago, the FDA made the historic decision to approve the first gene therapy in the US, finally realizing the therapeutic potential of hacking our biological base code after decades of cycles of hope and despair. Other approvals soon followed, including Luxturna to target inherited blindness and Zolgensma, a single injection that could save children with a degenerative disease from their muscles wasting away and dying before the age of two.

Yet despite their transformative potential, gene therapy has only targeted relatively rareand often fataldisorders. Thats about to change.

This year, a handful of companies deployed gene therapy against sickle-cell anemia, a condition that affects over 20 million people worldwide and 100,000 Americans. With over a dozen therapies in the run, sickle-cell disease could be the indication that allows gene therapy to enter the mainstream. Yet because of its unique nature, sickle-cell could also be the indication that shines an unflinching spotlight on challenges to the nascent breakthrough, both ethically and technologically.

You see, sickle-cell anemia, while being one of the worlds best-known genetic diseases, and one of the best understood, also predominantly affects third-world countries and marginalized people of color in the US. So far, gene therapy has come with a hefty bill exceeding millions; few people afflicted by the condition can carry that amount. The potential treatments are enormously complex, further upping costs to include lengthy hospital stays, and increasing potential side effects. To muddy the waters even more, the disorder, though causing tremendous pain and risk of stroke, already has approved pharmaceutical treatments and isnt necessarily considered life-threatening.

How we handle gene therapies for sickle-cell could inform many other similar therapies to come. With nearly 400 clinical trials in the making and two dozen nearing approval, theres no doubt that hacking our genes will become one of the most transformative medical wonders of the new decade. The question is: will it ever be available for everyone in need?

Even those uninterested in biology have likely heard of the disorder. Sickle-cell anemia holds the crown as the first genetic disorder to be traced to its molecular roots nearly a hundred years ago.

The root of the disorder is a single genetic mutation that drastically changes the structure of the oxygen-carrying protein, beta-globin, in red blood cells. The result is that the cells, rather than forming their usual slick disc-shape, turn into jagged, sickle-shaped daggers that damage blood vessels or block them altogether. The symptoms arent always uniform; rather, they come in crisis episodes during which the pain becomes nearly intolerable.

Kids with sickle-cell disorder usually die before the age of five; those who survive suffer a lifetime of debilitating pain and increased risk of stroke and infection. The symptoms can be managed to a degree with a cocktail of drugsantibiotics, painkillers, and a drug that reduces crisis episodes but ups infection risksand frequent blood transfusions or bone marrow transplants. More recently, the FDA approved a drug that helps prevent sickled-shaped cells from forming clumps in the vessels to further combat the disorder.

To Dr. David Williams at Boston Childrens Hospital in Massachusetts, the availability of these treatmentshowever inadequatesuggests that gene therapy remains too risky for sickle-cell disease. Its not an immediately lethal diseaseit wouldnt be ethical to treat those patients with a highly risky experimental approach, he said to Nature.

Others disagree. Freeing patients from a lifetime of risks and pain seems worthy, regardless of the price tag. Inspired by recent FDA approvals, companies have jumped onto three different treatments in a bitter fight to be the first to win approval.

The complexity of sickle-cell disease also opens the door to competing ideas about how to best treat it.

The most direct approach, backed by Bluebird Bio in Cambridge, Massachusetts, uses a virus to insert a functional copy of the broken beta-globin gene into blood cells. This approach seems to be on track for winning the first FDA approval for the disorder.

The second idea is to add a beneficial oxygen-carrying protein, rather than fixing the broken one. Here, viruses carry gamma-globin, which is a variant mostly present in fetal blood cells, but shuts off production soon after birth. Gamma-globin acts as a repellent that prevents clotting, a main trigger for strokes and other dangerous vascular diseases.

Yet another idea also focuses on gamma-globin, the good guy oxygen-carrier. Here, rather than inserting genes to produce the protein, the key is to remove the breaks that halt its production after birth. Both Bluebird Bio and Sangamo Therapeutics, based in Richmond, California, are pursing this approach. The rise of CRISPR-oriented companies is especially giving the idea new promise, in which CRISPR can theoretically shut off the break without too many side effects.

But there are complications. All three approaches also tap into cell therapy: blood-producing cells are removed from the body through chemotherapy, genetically edited, and re-infused into the bone marrow to reconstruct the entire blood system.

Its a risky, costly, and lengthy solution. Nevertheless, there have already been signs of success in the US. One person in a Bluebird Bio trial remained symptom-free for a year; another, using a CRISPR-based approach, hasnt experienced a crisis in four months since leaving the hospital. For about a year, Bluebird Bio has monitored a dozen treated patients. So far, according to the company, none has reported episodes of severe pain.

Despite these early successes, advocates worry about the actual impact of a genetic approach to sickle-cell disease.

Similar to other gene therapies, the treatment is considered a last-line, hail Mary solution for the most difficult cases of sickle cell disease because of its inherent risks and costly nature. Yet end-of-the-line patients often suffer from kidney, liver, and heart damages that make chemotherapy far too dangerous.

Then theres the problem of global access. Some developing countries, where sickle-cell disease is more prevalent, dont even have consistent access to safe blood transfusions, not to mention the laboratory equipment needed for altering blood-producing stem cells. Recent efforts in education, early screening, and prevention have also allowed people to live longer and reduce the stigma of the disorder.

Is a $1 million price tag ever attainable? To combat exhorbitant costs, Bluebird Bio is offering an installment payment plan for five years, which can be terminated anytime the treatment stops working. Yet for patients in South Africa, India, or Cambodia, the costs far exceed the $3 per month price tag for standard treatment. Even hydroxyurea, the newly-approved FDA drug to reduce crisis pain episodes, is just a fraction of the price tag that comes with gene therapy.

As gene therapy technologies are further refined and their base cost reduced, its possible that overall costs will drop. Yet whether these treatments will be affordable in the long run remains questionable. Even as scientists focus on efficacy rather than price tag, NIH director Dr. Francis Collins believes not thinking about global access is almost unethical. There are historical examples for optimism: vaccines, once rather fringe, now touch almost every corner of our world with the help of scientific knowledge, advocacy groups, andfundamentallyproven efficacy.

With the rise of gene therapy, were now in an age of personalized medicine beyond imagination. Its true that perhaps sickle-cell disease genetic therapies arent quite there yet in terms of safety and efficacy; but without tackling access issues, the therapy will be stymied in its impact for global good. As genetic editing tools become more powerful, gene therapy has the potential to save even more livesif its made accessible to those who need it most.

Image Credit: Image by Narupon Promvichai from Pixabay

The rest is here:
Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub

Bone Marrow Stem Cells Comments Off on Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It’s Riddled With Controversy – Singularity Hub | December 18th, 2019

The "Hematopoietic Stem Cell Transplantation (HSCT) Market" report contains data that has been carefully analyzed in the various models and factors that influence the industrial expansion of the Hematopoietic Stem Cell Transplantation (HSCT) market. An assessment of the impact of current market trends and conditions is also included to provide information on the future market expansion. The report contains comprehensive information on the global dynamics of Hematopoietic Stem Cell Transplantation (HSCT), which provides a better prediction of the progress of the market and its main competitors [Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc]. The report provides detailed information on the future impact of the various schemes adopted by governments in different sectors of the world market.

The Hematopoietic Stem Cell Transplantation (HSCT) market report is crafted with figures, charts, tables, and facts to clarify, revealing the position of the specific sector at the regional and global level. The report also provides a brief summary of all major segments, such as [Autologous], with more detailed market share data in terms of supply, demand, and revenue from trading processes and after-sales.

Grab the sample of Hematopoietic Stem Cell Transplantation (HSCT) market here: http://www.marketsnresearch.com/request-for-sample.html?repid=62938

The Hematopoietic Stem Cell Transplantation (HSCT) report rates the market according to different segments, including geographic areas [Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)] and current market trends. The market report contains information about different companies, manufacturers and traders.

The market report comprises an analysis of the latest developments in the field of innovative technologies, detailed profiles of the industry's top competitors, and an excellent business model. The report also contains information on market expectations for the coming years. The Hematopoietic Stem Cell Transplantation (HSCT) report also provides a detailed summary of the macro and microelement estimations that are important to market participants and newly developed companies.

For more enquires regarding Hematopoietic Stem Cell Transplantation (HSCT) market, click here: http://www.marketsnresearch.com/inquiry-for-buying.html?repid=62938

The different characteristics and performance of Hematopoietic Stem Cell Transplantation (HSCT) are analyzed based on subjective and quantitative techniques to give a clear picture of current and future evaluation.

Research Objective :

Our board of exchange givers additionally as exchange experts over the value chain have taken immense endeavors in doing this gathering activity and hard work add request to deliver the key players with helpful essential and optional information concerning the world Hematopoietic Stem Cell Transplantation (HSCT) advertise. moreover, the report furthermore contains contributions from our exchange experts that may encourage the key players in sparing their time from the inside examination half. firms WHO get and utilize this report will be totally benefitted with the derivations conveyed in it. but this, the report furthermore gives top to bottom investigation on Hematopoietic Stem Cell Transplantation (HSCT) deal in addition on the grounds that the elements that impact the customers additionally as undertakings towards this technique.

Thanks for reading this article; you'll be able to additionally get individual chapter wise section or region wise report versions like North America, Europe, Asia-Pacific, South America, geographic area and continent.

Sorry! The Author has not filled his profile.

See the original post:
Hematopoietic Stem Cell Transplantation (HSCT) Market Expected to Deliver Dynamic Progression until 2028| Regen Biopharma Inc - The World Industry...

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Market Expected to Deliver Dynamic Progression until 2028| Regen Biopharma Inc – The World Industry… | December 18th, 2019

In exchange for the exclusive right to market and distribute leronlimab in the U.S. for HIV-related indications, Vyera will pay upfront and regulatory and sales-based milestone payments of up to $87.5 million, as well as a royalty of 50 percent on net sales. Vyera will also make an investment in CytoDyn of $4 million in the form of registered CytoDyn common stock

CytoDyn will maintain responsibility for the development and FDA approval of leronlimab for all HIV-related and other indications

VANCOUVER, Washington and NEW YORK, Dec. 17, 2019 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn) and Vyera Pharmaceuticals, LLC (Vyera), today announced that they have entered into a Commercialization and License Agreement (CLA) and a related Supply Agreement to commercialize leronlimab (PRO 140) in the U.S. for the treatment of HIV.

Under theterms of the CLA, CytoDyn will maintain responsibility for the development and FDA approval of leronlimab for all HIV-related and other indications, while Vyera has been granted an exclusive license to market and distribute leronlimab in the U.S. for the treatment of HIV. In exchange for such exclusive license, Vyera has agreed to pay upfront and regulatory and sales-based milestone payments of up to $87.5 million, as well as a royalty of 50 percent on net sales. Vyera also agreed to make an investment in CytoDyn of $4 million in the form of registered CytoDyn common stock.

It is anticipated that these agreements will enable CytoDyn to leverage Vyeras well-established commercial infrastructure and highly-experienced sales team for the launch and commercialization of leronlimab and provide Vyera with a complimentary and novel product to bolster its pipeline of therapies for the treatment of infectious diseases.

This agreement helps complete the strategic objective to further establish CytoDyn as a leader in efforts to enhance the lives of patients through target-specific medicine, said Nader Pourhassan, Ph.D., CytoDyns President and Chief Executive Officer. Vyeras focus on developing therapies for patients living with serious and neglected diseases make them an ideal partner for this collaboration. We are excited to work with Vyera to leverage their platforms and capabilities to potentially offer a more effective treatment option for this HIV population.

Averill L. Powers, Chief Executive Officer of Phoenixus AG, Vyeras parent company, noted: Vyeras collaboration with CytoDyn demonstrates our commitment to address the needs of significant patient populations across our group companies generally and, in particular, a new level of our commitment to supporting patients living with HIV.

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) has granted a "Fast Track" designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients, and the second is for metastatic triple-negative breast cancer (mTNBC). Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in Highly Treatment Experienced (HTE) Multi-Drug Resistant (MDR) HIV Patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98 percent in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additional clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of graft-versus-host disease (GvHD).

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as graft-vs-host disease (GvHD) and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab (PRO 140) as a once-weekly monotherapy for HIV-infected patients and, plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab (PRO 140) can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and has received clearance to initiate a clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

About VyeraVyera is a United States based biopharmaceutical company committed to developing and commercializing treatments that address serious and rare diseases with high unmet medical needs. Vyera supports programs that offer financial assistance to patients in need and gives discounts to organizations that provide care to underserved populations. Vyeras research and development efforts focus on novel treatment options for toxoplasmosis and other rare or serious health conditions. https://www.vyera.com/.

Forward-Looking StatementsThis press release contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995, that involve risks, uncertainties, and assumptions that are difficult to predict. CytoDyn and Vyera (collectively, the Companies) intend that such forward-looking statements be subject to the safe harbors created thereby. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companies forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Companies cash position, (ii) the Companies ability to raise additional capital to fund its operations, (iii) the Companies ability to meet its debt obligations, if any, (iv) the Companies ability to enter into partnership or licensing arrangements with third parties, (v) the Companies ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Companies ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Companies clinical trials, (viii) the results of the Companies clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companies products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companies control. CytoDyn urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, neither Company the Company undertakes any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CytoDyn Contacts:

Media:Grace FotiadesLifeSci Public Relationsgfotiades@lifescipublicrelations.com(646) 876-5026

Investors:Deanna Ebenhahndebenhahn@cytodyn.com

Vyera Contacts:

Media:media@vyera.com

Investors:ir@vyera.com

Visit link:
CytoDyn Signs Definitive Agreements with Vyera Pharmaceuticals to Commercialize Leronlimab in the U.S. for the Treatment of HIV - GlobeNewswire

Bone Marrow Stem Cells Comments Off on CytoDyn Signs Definitive Agreements with Vyera Pharmaceuticals to Commercialize Leronlimab in the U.S. for the Treatment of HIV – GlobeNewswire | December 18th, 2019

Overall score: 85/100

Tested August 2019

This face mask is formulated with ingredients such as ovine placenta and Phyto stem cells, a blend that claims to repair the skin and boost the production of collagen and elastin. It aims to leave skin hydrated, firmer and looking younger overnight these effects should be long-lasting.

250.00

Available from: net-a-porter.com

As many as 91% of our testers agreed that this product delivered on its claims. It left skin feeling more hydrated by morning and the effects were long-lasting.

It plumped and firmed the skin, especially around the neck and dcolletage. Our testers also noted improvements to the appearance of eye bags and fine lines around the eyes. The face mask absorbed, blended nicely and left the skin feeling soft. The panel described it as an intensive product, which reduced the size of pores and left the complexion smoother and more even.

All product information provided by the manufacturer is correct at time of publication.

View post:
MZ Skin Replenish and Restore Overnight Face Masque Review - goodhousekeeping.com

Skin Stem Cells Comments Off on MZ Skin Replenish and Restore Overnight Face Masque Review – goodhousekeeping.com | December 18th, 2019

Company closes $5.6 million in funding and secures distinguished board of directors as it seeks to empower individual health ownership with personalized biological analysis and storage

GoodCell ("LifeVault Bio"), the personal biobanking company with the health indicators to inform actionable next steps in your health journey, today announced it has secured a $2.6 million price round under LifeVault Bio and a $3 million convertible note, and brought on renowned technology executive Anthony Bay as its newest board member. The capital will be used to expand GoodCell Diagnostics, the companys commercial application, as it pioneers a cell quality test to measure the DNA damage to somatic cells over time, as well as fuel the formation of strategic partnerships across the healthcare and life sciences sectors, and grow the team at its headquarters in Waltham, Mass.

GoodCell helps individuals take control of their health through personalized biobanking of cells, DNA and blood plasma, with the belief that medical science will continue to progress, bringing forth new ways of preventing, detecting and treating diseases. Research continues to prove that cells are an essential starting material for the treatments of tomorrow. DNA and plasma are widely validated as critical information sources for monitoring and tracking health risk and informing lifestyle decisions. GoodCell aims to empower individuals with personal health information and storage resources to take full advantage of breakthrough medical science as it emerges.

"Stem cells are among the most promising areas of medical research because they are the starting materials from which all other cells originate," said Brad Hamilton, co-founder and chief science officer at GoodCell. "Some of these cells, specifically induced pluripotent stem (iPS) cells which can be derived from a persons own skin or blood, can be programmed to produce virtually any type of cell in the human body. This versatility has made them an instrumental tool, helping scientists understand and fight some of the biggest health threats of our time, such as Parkinsons disease, Type 1 diabetes and heart disease. GoodCell exists to help people preserve their access to these potentially lifesaving cells."

After GoodCell sends members a sample collection kit to their doorstep, they are prompted to schedule a convenient blood-draw with a certified phlebotomist, who then safely packages and ships the sample for processing. Once received, GoodCell isolates and preserves three components of the blood sample: cells, DNA and blood plasma. The DNA sample is then tested to inform genetic predisposition to disease, such as metabolic, neurologic and cardiac disorders, as well as certain cancers. Armed with deep insight into a members biology, the GoodCell Dashboard displays their health information as a comprehensive overview, designed to inform the next best action in their health journey. Samples are stored in a state-of-the-art, FDA-registered CLIA/CAP certified lab and biorepository that is trusted by larger biotechnology companies and the National Institutes of Health. Since it is the change in health indicators that indicates risk, recurrent sampling is possible to enable measuring the trajectory of change in plasma components or DNA. Since the samples belong to GoodCell members, they can decide whether or not to share their information with their doctor or allow researchers to use it in clinical studies.

"To me, GoodCell represents the ultimate in personalized medicine. Individuals can now have their own biobank and their own biodata. These wont be owned by a hospital or in the case of your cells, by no one at all. These will be stored for you, accessible only on your instruction. As new tests come online or as cells become a broader therapy source, you will be able to tap into your own earlier, preserved self in the form of your blood," said David Scadden, MD, co-founder and chair of the Scientific Advisory Board at GoodCell. "Imagine two scenarios. First, a new blood test becomes available for Alzheimers disease. You get the test, but just like current tests for things like prostate cancer, it is only meaningful in light of how it is changing. Your doctor will likely advise waiting months or a year to re-test. With a GoodCell sample, we envision the test can be done on your blood from a previous time. Then you can know how things are changing without the prolonged wait and the anxiety it engenders. Second, lets say the stem cell field delivers on the therapies it is currently testing for diabetes, heart failure, Parkinsons disease and macular degeneration. Those therapies will likely be as cells derived from you. Would you want those to be from you at a younger age since we know our cells accumulate genetic damage with age? I think most people would, and would want cells from their blood, which the bones have shielded from radiation, rather than their skin as is currently done. GoodCell will have those blood cells for you and has shown they can be made into stem cells (iPSC) with high efficiency."

Story continues

GoodCell is focused on continuing to grow its customer base and building up its talent pool at its new headquarters in Waltham, Mass. The company, which is poised to expand its headcount in early 2020, will also be exploring strategic partnerships with cell and gene therapy companies and interest groups that could benefit from GoodCell members deciding whether to opt-in to allow access to stored cells, DNA and plasma. GoodCell will also continue to recruit pioneers in business, science and technology to its board positions. Most recently, it welcomed Anthony Bay, former Global Head of Digital Video for Amazon and a veteran senior executive at other technology powerhouses, including Apple and Microsoft.

"Ive devoted my career to creating scalable and differentiated technology platforms and unique digital experiences in many industries, and am excited to lend my expertise and perspectives to GoodCell," said Bay. "I am delighted to play a role in helping the GoodCell team scale and expand to match the size of our opportunity to change peoples lives."

Bay joins an already robust and diverse group of consumer technology and life science leaders, including John Goscha, Lucidity Lights founder and Chairman of the Board of Directors, Finally Light Bulb Company founder and entrepreneur; David Scadden, MD, professor of medicine at Harvard Universitys Department of Stem Cell and Regenerative Biology; Daniel Marshak, principal consultant in therapeutics, diagnostics and medical devices; Avi Ellman, managing partner of Delta Global Investment Services; and Trevor Perry, co-founder and chief executive officer at GoodCell.

"Up until now, existing genetics offerings can only go so far as to inform your genetic makeup. GoodCell is taking that a step further today by combining genetics, health indicator testing and personal biobanking into one solution, and then turning this information right back to the individual so they can understand the story of their health and leverage actionable data at any age," said Perry. "We are taking advantage of leading scientific innovation to help people take control of their health through personalized biobanking of cells, DNA, and blood plasma, and we believe the tremendous amount of support we received during this initial funding round will further allow us to be a true enabler of and partner in this process. Our goal is to set a new standard for personal biobanking as an individual health milestone, and our mission is to ensure our members feel confident and prepared to own their aging experience, and we look forward to accelerating our efforts in the months ahead."

For more information about GoodCell, visit https://www.goodcell.com. To order your starter kit, visit https://www.goodcell.com/shop/.

About GoodCell

GoodCell helps you take control of your health through personalized biobanking of cells, DNA and blood plasma. Leveraging the best science, the technology provides health indicators for a comprehensive and proactive approach to self-care. Through the GoodCell Dashboard, the company informs the next best action in your health journey, offering access for you and for your doctor to actionable data and insights that relate to all aspects of your health through genetic reporting and blood analysis. Driven by mounting evidence in support of cellular therapy and united in the belief that you should be empowered to take control of your health, GoodCell is led by a founding team of scientific advisors with a diverse set of medical research and clinical expertise. By backing up your starting materials, GoodCell is setting a new standard of personal biobanking today for a healthier future. Learn more at: https://www.goodcell.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191217005485/en/

Contacts

PAN CommunicationsStaci Didner407 734 7325Goodcell@pancomm.com

Go here to see the original:
GoodCell Oversubscribes Upon Debut, Fueling Expansion of Health Tracking and Personal Biobanking Services; Adds Former Amazon and Microsoft Executive...

Skin Stem Cells Comments Off on GoodCell Oversubscribes Upon Debut, Fueling Expansion of Health Tracking and Personal Biobanking Services; Adds Former Amazon and Microsoft Executive… | December 18th, 2019

Though the research was intended as a proof of concept, the experimental gene therapy slowed tumour growth and prolonged survival in mice with gliomas, which constitute about 80% of malignant brain tumours in humans.

The technique takes advantage of exosomes, fluid-filled sacs that cells release as a way to communicate with other cells.

The research was carried out by scientists at the Ohio State University and published in the journal Nature Biomedical Engineering.

While exosomes are gaining ground as biologically friendly carriers of therapeutic materials because there are a lot of them and they dont prompt an immune response the trick with gene therapy is finding a way to fit those comparatively large genetic instructions inside their tiny bodies on a scale that will have a therapeutic effect.

This new method relies on patented technology that prompts donated human cells such as adult stem cells to spit out millions of exosomes that, after being collected and purified, function as nanocarriers containing a drug.

When they are injected into the bloodstream, they know exactly where in the body to find their target even if its in the brain.

Senior study author L. James Lee, professor emeritus of chemical and biomolecular engineering at Ohio State University, said: Think of them like Christmas gifts: the gift is inside a wrapped container that is postage paid and ready to go. This is a Mother Nature-induced therapeutic nanoparticle.

In 2017, Lee and colleagues made waves with news of a regenerative medicine discovery called tissue nanotransfection (TNT). The technique uses a nanotechnology-based chip to deliver biological cargo directly into skin, an action that converts adult cells into any cell type of interest for treatment within a patients own body.

By looking further into the mechanism behind TNTs success, scientists in Lees lab discovered that exosomes were the secret to delivering regenerative goods to tissue far below the skins surface.

The scientists placed about one million donated cells on a nano-engineered silicon wafer and used an electrical stimulus to inject synthetic DNA into the donor cells. As a result of this DNA force-feeding, as Lee described it, the cells need to eject unwanted material as part of DNA transcribed messenger RNA and repair holes that have been poked in their membranes.

The electrical stimulation had a bonus effect of a thousand-fold increase of therapeutic genes in a large number of exosomes released by the cells, a sign that the technology is scalable to produce enough nanoparticles for use in humans.

Essential to any gene therapy is knowing what genes need to be delivered to fix a medical problem. For this work, the researchers chose to test the results on glioma brain tumours by delivering a gene called PTEN, a cancer-suppressor gene. Mutations of PTEN that turn off that suppression role can allow cancer cells to grow unchecked.

For Lee, founder of Ohio States Center for Affordable Nanoengineering of Polymeric Biomedical Devices, producing the gene is the easy part. The synthetic DNA force-fed to donor cells is copied into a new molecule consisting of messenger RNA, which contains the instructions needed to produce a specific protein. Each exosome bubble containing messenger RNA is transformed into a nanoparticle ready for transport, with no blood-brain barrier to worry about.

The testing in mice showed the labelled exosomes were far more likely to travel to the brain tumours and slow their growth compared to substances used as controls.

Because of exosomes safe access to the brain, Lee said, this drug-delivery system has promise for future applications in neurological diseases such as Alzheimers and Parkinsons disease.

Read more from the original source:
Mother Nature provides new gene therapy strategy to reverse disease - Health Europa

Skin Stem Cells Comments Off on Mother Nature provides new gene therapy strategy to reverse disease – Health Europa | December 18th, 2019

A Wisconsin boy with a uncommon illness that causes his pores and skin to blister on the slightest contact has now been recognized with most cancers.

Charlie Knuth, 13, from Darboy, was adopted by his mother and father after he was deserted on the hospital as a child, reported WFRV.

He suffers from epidermolysis bullosa (EB), a uncommon genetic tissue dysfunction that causes the pores and skin to blister and burst, leaving uncooked sores which might be inclined to infections.

Charlie has lived most of his life wrapped in bandages and has to take particular baths on daily basis to deal with his sores and maintain them from getting contaminated.

However the teenager is now going through a brand new battle after being recognized earlier this 12 months with lymphoma, a most cancers of the immune system.

Charlie Knuth, 13, from Darboy, Wisconsin, was born with a uncommon pores and skin dysfunction. Pictured: Charlie, proper, along with his father, Kevin

The dysfunction, often known as epidermolysis bullosa, causes the pores and skin to blister and burst on the slightest contact and leaves uncooked sores. Pictured: Charlie within the hospital)

Victims of EB are lacking sort VII collagen, a protein that enables the highest layer of pores and skin to bind with the underside layers.

The slightest motion can causes the pores and skin to instantly and constantly fall off.

The dysfunction could be very uncommon, and is estimated to happen in 20 newborns per a million reside births within the US, based on Stanford Kidss Hospital.

About 87 % of youngsters born with EB die throughout their first 12 months of life.

There isnt any remedy for EB so remedy goals at stopping blisters from changing into contaminated.

Charlies mom, Trisha Knuth, stated she and his father, Kevin, have tried a number of measures to assist deal with her son, together with lotions, lotions and gloves.

He is additionally undergone two stem cell transplants, during which new sheets of pores and skin grown and graft over the injuries.

In 2017, Charlie underwent surgical procedure to revive the usage of his palms, which had been degenerating because of his situation.

With no surgical procedure, his palms can be lined in scar tissue.

The scar tissue will really construct up between all the net areas between every finger, and the palms, and develop proper excessive of the hand so persons are left with simply nothing however mitts, Trisha advised WFRV.

In keeping with the station, throughout the surgical procedure, pores and skin was taken from Charlies thighs to make use of on his palms.

Titanium rods had been additionally inserted intoevery of his fingers, and saved there for 5 weeks, to stop them from curling into his palms.

Charlie (left and proper) was deserted at a hospital earlier than being adopted by his present mother and father. Earlier this month, he was recognized with lymphoma, a most cancers of immune system cells

His mother and father stated theyre touring to Minnesota to determine what stage his most cancers is at and what remedy hell bear. Pictured: Charlie, proper, along with his mom, Trisha

On Wednesday, Trisha posted on Fb that her son was recognized with lymphoma.

Lymphoma is most cancers that begins within the lymphocytes, that are immune system cells that struggle an infection.

There are two kinds of the most cancers, Non-Hodgkins and Hodgkins, nevertheless its not clear which kind Charlie has.

Indicators and signs embody swelling of the lymph nodes, fever, fatigue, shortness of breath and sudden weight reduction.

Therapy varies and may embody chemotherapy, radiation remedy and immunotherapy.

Its estimated that 82,310 folks will likely be recognized with lymphoma in 2019 and that 20,970 will die, based on the American Most cancers Society.

Within the Fb put up, Trisha wrote: My head is spinning and my coronary heart is breaking. My candy boy.

Charlies mom added the household will likely be touring to Minnesota so his most cancers can get staged and so they can assess remedy choices.

View original post here:
Boy abandoned at the hospital after being born with butterfly skin disease diagnosed with cancer - Herald Publicist

Skin Stem Cells Comments Off on Boy abandoned at the hospital after being born with butterfly skin disease diagnosed with cancer – Herald Publicist | December 18th, 2019

The ODAC discussions were based on the supplemental Biologics License Application (sBLA), currently under priority review at the FDA, seeking approval of KEYTRUDA monotherapy for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, NMIBC with carcinoma in-situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy (removal of bladder). This application is based on results from the Phase 2 KEYNOTE-057 trial.

The positive vote from todays ODAC meeting supports the potential for KEYTRUDA in certain patients with high-risk, non-muscle invasive bladder cancer, who currently have limited non-surgical treatment options approved by the FDA, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. We are encouraged by todays productive discussion and look forward to working with the FDA as they continue their review of our supplemental application for KEYTRUDA in this patient population.

The ODAC provides the FDA with independent, expert advice and recommendations on marketed and investigational medicines for use in the treatment of cancer. The FDA is not bound by the committees guidance but takes its advice into consideration. Merck anticipates a Prescription Drug User Fee Act (PDUFA), or target action date, in January 2020, based on priority review.

About Bladder Cancer

Bladder cancer begins when cells in the urinary bladder start to grow uncontrollably. As more cancer cells develop, they can form a tumor and spread to other areas of the body. Bladder cancers are described based on how far they have invaded into the wall of the bladder. NMIBC occurs when the cancer has not grown into the main muscle layer of the bladder. It is estimated that more than 80,000 new cases of bladder cancer will be diagnosed in 2019 in the United States. Approximately 75% of patients with bladder cancer are diagnosed with non-muscle invasive bladder cancer (NMIBC). For high-risk NMIBC patients who are BCG-unresponsive with persistent or recurrent disease, treatment guidelines recommend radical cystectomy, a surgery to remove the entire bladder that often requires removal of other surrounding organs and tissues. In men, removal of the prostate is common, and in women, surgeons may also remove the uterus, fallopian tubes, ovaries and cervix, and occasionally a portion of the vagina.

About KEYNOTE-057

The filing was based on data from KEYNOTE-057 (NCT02625961), a Phase 2, multicenter, open-label, single-arm trial in 102 patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in-situ (CIS) with or without papillary tumors who were ineligible for or had elected not to undergo cystectomy (Cohort A). In this study, BCG-unresponsive high-risk NMIBC is defined as persistent disease despite adequate BCG therapy, disease recurrence after an initial tumor-free state following adequate BCG therapy, or T1 disease following a single induction course of BCG. Patients received KEYTRUDA 200 mg every three weeks until unacceptable toxicity, persistent or recurrent high-risk NMIBC or progressive disease. Assessment of tumor status was performed every 12 weeks, and patients without disease progression could be treated for up to 24 months. The major efficacy outcome measures were complete response (as defined by negative results for cystoscopy [with transurethral resection of bladder tumor (TURBT)/biopsies as applicable], urine cytology, and computed tomography urography [CTU] imaging) and duration of response.

About KEYTRUDA (pembrolizumab) Injection, 100mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10] as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency), thyroid function (prior to and periodically during treatment), and hyperglycemia. For hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 and withhold or discontinue for Grade 3 or 4 hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL. Serious adverse reactions occurred in 16% of patients; those 1% included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; 1 from GVHD after subsequent allogeneic HSCT and 1 from septic shock. The most common adverse reactions (20%) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-170, KEYTRUDA was discontinued due to adverse reactions in 8% of 53 patients with PMBCL. Serious adverse reactions occurred in 26% of patients and included arrhythmia (4%), cardiac tamponade (2%), myocardial infarction (2%), pericardial effusion (2%), and pericarditis (2%). Six (11%) patients died within 30 days of start of treatment. The most common adverse reactions (20%) were musculoskeletal pain (30%), upper respiratory tract infection and pyrexia (28% each), cough (26%), fatigue (23%), and dyspnea (21%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. Serious adverse reactions occurred in 42% of patients; those 2% were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis. The most common adverse reactions (20%) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%).

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.9%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients; those 2% were urinary tract infection, pneumonia, anemia, and pneumonitis. The most common adverse reactions (20%) in patients who received KEYTRUDA were fatigue (38%), musculoskeletal pain (32%), pruritus (23%), decreased appetite (21%), nausea (21%), and rash (20%).

Adverse reactions occurring in patients with gastric cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

Adverse reactions occurring in patients with esophageal cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

In KEYNOTE-158, KEYTRUDA was discontinued due to adverse reactions in 8% of 98 patients with recurrent or metastatic cervical cancer. Serious adverse reactions occurred in 39% of patients receiving KEYTRUDA; the most frequent included anemia (7%), fistula, hemorrhage, and infections [except urinary tract infections] (4.1% each). The most common adverse reactions (20%) were fatigue (43%), musculoskeletal pain (27%), diarrhea (23%), pain and abdominal pain (22% each), and decreased appetite (21%).

Adverse reactions occurring in patients with hepatocellular carcinoma (HCC) were generally similar to those in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of ascites (8% Grades 34) and immune-mediated hepatitis (2.9%). Laboratory abnormalities (Grades 34) that occurred at a higher incidence were elevated AST (20%), ALT (9%), and hyperbilirubinemia (10%).

Among the 50 patients with MCC enrolled in study KEYNOTE-017, adverse reactions occurring in patients with MCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy. Laboratory abnormalities (Grades 34) that occurred at a higher incidence were elevated AST (11%) and hyperglycemia (19%).

In KEYNOTE-426, when KEYTRUDA was administered in combination with axitinib, fatal adverse reactions occurred in 3.3% of 429 patients. Serious adverse reactions occurred in 40% of patients, the most frequent (1%) were hepatotoxicity (7%), diarrhea (4.2%), acute kidney injury (2.3%), dehydration (1%), and pneumonitis (1%). Permanent discontinuation due to an adverse reaction occurred in 31% of patients; KEYTRUDA only (13%), axitinib only (13%), and the combination (8%); the most common were hepatotoxicity (13%), diarrhea/colitis (1.9%), acute kidney injury (1.6%), and cerebrovascular accident (1.2%). The most common adverse reactions (20%) were diarrhea (56%), fatigue/asthenia (52%), hypertension (48%), hepatotoxicity (39%), hypothyroidism (35%), decreased appetite (30%), palmar-plantar erythrodysesthesia (28%), nausea (28%), stomatitis/mucosal inflammation (27%), dysphonia (25%), rash (25%), cough (21%), and constipation (21%).

Read this article:
FDA Oncologic Drugs Advisory Committee (ODAC) Recommends KEYTRUDA (pembrolizumab) for the Treatment of Certain Patients with High-Risk, Non-Muscle...

Skin Stem Cells Comments Off on FDA Oncologic Drugs Advisory Committee (ODAC) Recommends KEYTRUDA (pembrolizumab) for the Treatment of Certain Patients with High-Risk, Non-Muscle… | December 18th, 2019

MELVILLE, N.Y., Dec. 16, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, announced today feature coverage in the news outlet, IEEE Pulse, a magazine of the IEEE Engineering in Medicine and Biology Society. According to IEEE, it is the worlds largest technical professional organization for the advancement of technology.

To view the IEEE Pulse Magazines article featuring BioRestorative, click here.

The published cover-story article features commentary from Francisco Silva, Chief Scientist and Vice President of Research and Development for BioRestorative, regarding BRTX-100, the Companys lead therapeutic candidate for chronic lumbar disc disease. Once the U.S. Food and Drug Administration (FDA) authorizes the sale of BRTX-100, we would ship it to your doctor, and with a 30-minute procedure the material would be injected into your disc in a 1.5 ml solution, explains Silva. He elaborates on the product, discussing growing and expanding stem cells from the patients bone marrow under hypoxic conditions that mimic those in the normal intervertebral space. We are enriching the cells to be able to survive in this harsh environment, says Silva.

In addition to BRTX-100, the magazine article also highlights BioRestoratives other research pursuit, its ThermoStem program, utilizing brown adipose (fat) derived stem cells to target treatment of metabolic diseases and disorders, like diabetes, obesity and hypertension.

About BioRestorative Therapies, Inc.

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

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

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

Forward-Looking Statements

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

CONTACT:Email: ir@biorestorative.com

Excerpt from:
BioRestorative Therapies Featured in IEEE Pulse Magazine's Cover Story About Stem Cell Therapies for Low Back Pain - GlobeNewswire

Bone Marrow Stem Cells Comments Off on BioRestorative Therapies Featured in IEEE Pulse Magazine’s Cover Story About Stem Cell Therapies for Low Back Pain – GlobeNewswire | December 16th, 2019

News Release

Tuesday, December 10, 2019

New findings from a clinical trial show that treatment with the immunotherapy drug blinatumomab is superior to standard chemotherapy for children and young adults with high- or intermediate-risk B-cell acute lymphoblastic leukemia (B-ALL) that has relapsed. Those treated with blinatumomab had longer survival, experienced fewer severe side effects, had a higher rate of undetectable residual disease, and were more likely to proceed to a stem cell transplant.

Our study demonstrates that immunotherapy with blinatumomab is more effective and less toxic than chemotherapy as a bridge to curative bone marrow transplant for children and young adults with very aggressive relapse of B-ALL, said Patrick Brown, M.D., who chaired the trial and is director of the Pediatric Leukemia Program at the Johns Hopkins Kimmel Cancer Center, Baltimore. We are thrilled that these patients, whose survival has not substantially improved for decades, now have a new and better standard of care.

The findings were presented as a late-breaking abstract at the American Society of Hematology (ASH) annual meeting on Dec. 10, 2019. The trial was led by the Childrens Oncology Group (COG), part of the National Cancer Institute (NCI)sponsored National Clinical Trials Network. NCI is part of the National Institutes of Health. Amgen reviewed the trial protocol and amendments and provided the study drug under a Cooperative Research and Development Agreement with NCI.

These findings will likely have immediate impact on the treatment of this group of children and young adults with relapsed B-ALL, said Malcolm Smith, M.D., Ph.D., associate branch chief for pediatric oncology in NCIs Cancer Therapy Evaluation Program, which sponsored the trial. These results also reinforce the important role that federally funded clinical trials play in developing more effective treatments for children with cancer.

When children have B-ALL that relapses after their initial treatment, they are typically given chemotherapy. The first four to six weeks of chemotherapy, the reinduction phase, is commonly followed by additional intensive chemotherapy, or consolidation treatment, to further reduce disease levels. Following this, hematopoietic stem cell transplant is considered the best treatment for approximately half of patients, based on factors such as whether relapse occurred during initial treatment or shortly after it was completed.

However, chemotherapy can produce severe side effects in some patients and is sometimes ineffective in reducing leukemia levels to the low levels needed prior to transplant. As a result, patients may not be able to proceed to transplant or transplant may be delayed, which increases the risk that the leukemia will return.

The COG study investigated blinatumomab as an alternative type of consolidation treatment to follow the reinduction phase. Blinatumomab is a type of immunotherapy that works by binding to two different molecules: CD19, a protein, or antigen, expressed on the surface of B-ALL cells, and CD3, an antigen expressed on T cells. By bringing T cells close to leukemia cells, the immunotherapy helps the T cells recognize and kill the cancer cells.

Blinatumomab has been approved by the U.S. Food and Drug Administration (FDA) for adults and children with B-ALL that has returned or has not responded to treatment. FDA has also granted accelerated approval to the drugmeaning confirmatory trials must show it has clinical benefitfor some adults and children undergoing treatment for B-ALL who achieve complete remission but still have small amounts of leukemia detectable using very sensitive methods.

Investigators in this study wanted to see if blinatumomab could increase rates of survival free from leukemia and be less toxic than intensive chemotherapy in children and young adults undergoing consolidation treatment.

The trial report was based on 208 children and young adults aged 130 with relapsed B-ALL who had received reinduction chemotherapy and were considered to have high- or intermediate-risk disease. They were randomly assigned to receive either two rounds of intensive chemotherapy or two 4-week rounds of treatment with blinatumomab before proceeding to a transplant. (A separate part of the study addressed children with low-risk disease.)

After a median follow-up time of 1.4 years, those in the blinatumomab group had higher rates of 2-year disease-free survival, the primary outcome of the study, than those who received intensive chemotherapy (59.3 5.4% vs. 41 6.2%). Those treated with blinatumomab also had higher rates of overall survival (79.4 4.5% vs. 59.2 6%), fewer severe side effects, a higher rate of undetectable residual disease (79% vs. 21%), and a higher rate of proceeding to stem cell transplant (73% vs. 45%).

At a planned interim analysis, an independent data safety monitoring committee concluded that the outcome for children treated with blinatumomab was superior to that of children treated with chemotherapy only and recommended that enrollment to the high- and intermediate-risk part of the trial be stopped.

Future clinical trials will study whether blinatumomabs effects in relapsed B-ALL can be enhanced by combining it with other immunotherapy and will test whether adding the drug to standard chemotherapy for children and young adults with newly diagnosed B-ALL is beneficial.

About the National Cancer Institute (NCI):NCIleads the National Cancer Program and NIHs efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website atcancer.govor call NCIs contact center, the Cancer Information Service, at 1-800-4-CANCER (1-800-422-6237).

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

###

See the original post here:
Immunotherapy drug improves outcomes for some children with relapsed leukemia - National Institutes of Health

Bone Marrow Stem Cells Comments Off on Immunotherapy drug improves outcomes for some children with relapsed leukemia – National Institutes of Health | December 16th, 2019

Aspen Neuroscience, a new biotech company, has raised $6.5 million to develop cell therapies for Parkinsons disease using patients own cells.

The company was co-founded by renowned stem cell scientists Jeanne F. Loring, PhD, and Andres Bratt-Leal, PhD, and initially supported by Summit for Stem Cell, a non-profit organization that provides a variety of services for Parkinsons patients.

Parkinsons hallmark motor symptomsinclude tremor, slowness of movement (bradykinesia), stiffness (rigidity), uncontrollable movements (dyskinesia), and poor balance.

As the disease progresses, patients typically need to gradually increase their dopaminergic therapeutic dose for maximum benefit. Even after that they might sometimes experience reappearance or worsening of symptoms due to diminishing effects of dopaminergic therapy, known was off periods.

Importantly, dopaminergic therapy is delivered to areas of the brain other than the striatum, a key motor control region severely affected in Parkinsons disease. Because of the therapys off-target behavior, patients also may experience side effects such as hallucinations or cognitive impairment.

Aspen wants to combine its expertise in stem cell biology, genomics and neurology and develop the first autologous (self) stem cell-based therapy for Parkinsons disease.

In this type of cell therapy, a patients own cells (usually skin cells) are reprogrammed back into a stem cell-like state, which allows the development of an unlimited source of almost any type of human cell needed, including dopamine-producing neurons, which are those mainly affected by this disorder.

Because these cells are derived from patients, they do not carry the risk of being rejected once re-implanted, eliminating the need for immunosuppressive complementary therapies, which carry serious side effects such as infections and possibly limiting therapeutic potential.

In theory, replacing lost dopaminergic neurons with new stem cell-derived dopamine-producing ones could potentially ease or reverse motor symptoms associated with the disease.

Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinsons disease, Howard J. Federoff, MD, PhD, Aspens CEO, said in a press release.

We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist, Federoff said.

The companys lead product (ANPD001) is undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinsons disease. Aspen experts also are developing a gene-editing treatment (ANPD002) for familial forms of Parkinsons, starting with the most common genetic variant in the GBAgene, which provides instructions to make the enzyme beta-glucocerebrosidase.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32, according to the press release.

With over three years of experience in the medical communications business, Catarina holds a BSc. in Biomedical Sciences and a MSc. in Neurosciences. Apart from writing, she has been involved in patient-oriented translational and clinical research.

Total Posts: 208

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

The rest is here:
Aspen Neuroscience Receives $6.5M for Parkinson's Stem Cell Therapy - Parkinson's News Today

Skin Stem Cells Comments Off on Aspen Neuroscience Receives $6.5M for Parkinson’s Stem Cell Therapy – Parkinson’s News Today | December 16th, 2019

LOUISVILLE, Colo., Dec. 16, 2019 /PRNewswire/ --French Transit, LLC brands MyChelle Dermaceuticalsand CRYSTAL Deodoranttoday announced plans to continue and expand existing partnerships with several key charities and non-profits in 2020. These initiatives are part of an extensive corporate social responsibility program spearheaded by French Transit CEO Martin Floreani.

"It is no longer enough to be 'clean' or 'cruelty-free' in a passive manner," says Floreani. "We also need to be proactive in our stewardship of the planet and its resources. We need to put our time, money and full commitment behind our mission which we define as Clean & Caring." Floreani will be a key panelist during a Natural Products Expo West education seminar in March dedicated to helping other companies define and grow their own corporate social responsibility programs.

"Brands need to embrace cruelty-free research processes, but our company goes beyond by supporting organizations like The Gentle Barn that foster vegan and cruelty-free lifestyles." The Gentle Barn is a non-profit organization dedicated to ending animal cruelty and animal testing that is unfortunately still a reality in the beauty and personal care category. It provides sanctuary for abused and neglected animals and allows inner-city and at-risk children to interact with them, promoting respect and responsibility. MyChelle and CRYSTAL brands supported the Gentle Barn's "Gentle 12 program" for the month of August, and served as title sponsors for the charity's 20th Anniversary Gala in September and have confirmed their renewed commitment for 2020.

MyChelle Dermaceuticals will also continue and expand its support of The Coral Restoration Foundation, the world's largest nonprofit marine-conservation organization dedicatedto restoring our planet's coral reefs to a healthy state. MyChelle was one of the first cosmetic manufacturers to reject marine-toxic ingredients, including oxybenzone, octinoxate, butylparaben, retinyl palmitate and 4-methylbenzylidene camphor. "MyChelle is dedicated not only to creating reef-friendly sun care but also helping organizations such as the Coral Restoration Foundation to preserve and grow reefs," said Floreani. Last July, the company donated 1% of all net proceeds from online sales to the Foundation. This year MyChelle will continue its support of the Coral Restoration Foundation but is also working to be the first brand in its category to incorporate ocean recycled plastic into its packaging.

French Transit is also a recurring sponsor of Breast Cancer Prevention Partners (BCPP), an organization that raises awareness of toxic chemicals used in many personal care products. In 2019, during Breast Cancer Awareness Month, French Transit donated 1% of net sales from their websites to the organization, a commitment that will be repeated in 2020.

"We look forward to building upon our work with these amazing and truly impactful organizations in 2020," said Floreani. "And we are excited and proud to be a leader in a growing community of personal care companies who actively go beyond their claims to foster positive change and real results."

http://www.mychelle.com (PRNewsFoto/MyChelle Dermaceuticals)" alt="Founded in 2000, the Colorado-based skin care company is credited as the first to successfully develop and market natural skin care products using a combination of anti-aging peptides, plant stem cells, and clinically proven dermatological ingredients. Learn more at http://www.mychelle.com (PRNewsFoto/MyChelle Dermaceuticals)">

View original content to download multimedia:http://www.prnewswire.com/news-releases/french-transit-brands-to-expand-their-corporate-social-responsibility-programs-in-2020-300974956.html

SOURCE French Transit

Read more here:
French Transit Brands to Expand their Corporate Social Responsibility Programs in 2020 - Benzinga

Skin Stem Cells Comments Off on French Transit Brands to Expand their Corporate Social Responsibility Programs in 2020 – Benzinga | December 16th, 2019

According to a recent report published by Persistence Market Research, the Stem Cell-Derived Cells Market is expected to witness a CAGR growth of ~XX% over the forecast period 2019 2029 and reach a value of ~US$ by the end of 2029. Furthermore, the micro and macro-economic elements that are forecasted to influence the trajectory of the Stem Cell-Derived Cells Market are thoroughly analyzed in the presented market study.

The report throws light on the various raw material suppliers, vendors, manufacturers, and market players in the value chain of the Stem Cell-Derived Cells Market. Further, the economic and political scenarios of various regions and its impact on the Stem Cell-Derived Cells Market are thoroughly discussed in the report.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/28780

The report studies and provides key analysis in the shape of Stem Cell-Derived Cells Market dynamics such as the drivers, restraints, trends, opportunities, forecast factors and value chain that impact the Stem Cell-Derived Cells Market.

Critical insights enclosed in the report:

The competitive outlook chapter throws light on the business prospects of prominent players operating in the Stem Cell-Derived Cells Market. The product pricing strategies, preferred marketing channels, product portfolio of prominent players, and market presence of each company is included in the report.

The report caters to the following queries pertaining to the Stem Cell-Derived Cells Market:

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/28780

The presented market study sheds light on the market scenario in different regional markets. In addition, the impact of the regulatory and governmental policies on the prospects of the Stem Cell-Derived Cells Market in each region is analyzed in the report. The report offers comprehensive data on the growth parameters and market dynamics for each of these regions

key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/28780

Reasons why you should choose PMR

About us:

Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

Contact us:

Persistence Market Research305 Broadway, 7th FloorNew York City, NY 10007United StatesPh.no. +1-646-568-7751

This post was originally published on Downey Magazine

Read more:
Stem Cell-Derived Cells Market To Boost Demand And Forecast In Upcoming Year 2019 2029 - Downey Magazine

IPS Cell Therapy Comments Off on Stem Cell-Derived Cells Market To Boost Demand And Forecast In Upcoming Year 2019 2029 – Downey Magazine | December 16th, 2019

Gary L. Dunbar, executive director of the Field Neurosciences Institute, part of Ascension St. Mary's, was recently presented with the Gusi Peace Prize International Award.

Dr. Dunbar recently traveled to Manila, Philippines to accept this honor at the Gusi Peace Prize International 20th Annual Awards Night.

The Gusi Peace Prize award is given by the Gusi Peace Prize Foundation to recognize individuals and organizations who contribute to global peace and progress through a wide variety of fields. Dunbar was one of 18 international recipients selected for the award and chosen because of his global contributions in both the educational and the research domains of neuroscience. Similarly, his contributions in research, especially for developing new strategies for treating damage to the nervous system, including transplantation of genetically altered adult stem cells as a potential therapy for injury to the brain and spinal cord as well as neurological deficits in Huntington's, Parkinson's and Alzheimer's diseases, has earned international recognition and a prominent leadership role in the American Society for Neural Therapy and Repair.

"I felt both honored and humbled to be selected for the Gusi Peace Prize, especially after meeting and hearing, first-hand, what the other 2019 Gusi Laureates have accomplished in the context of helping others, which was humbling to me," shared Dunbar. "The prize is given to those whose efforts have provided significant improvements to the lives of others through education, research, politics, and/or the arts, along with a strong commitment to humanitarian commitments, so I felt deeply honored to be included in this group of people."

Dunbar has been the executive director for the Field Neurosciences Institute since 2008. Martha Ann Joseph, Chair of the institute's board of directors, was thrilled to hear that Dunbar was a recipient of the Gusi Peace Prize International Award.

"(Field Neurosciences Institute) is blessed to have Dr. Dunbar as our executive director for the past 11 years as he has always embraced the mission of the organization, to help others in terms of preventing brain injury and searching for cures for neurological problems emanating from trauma and disease to the nervous system," Joseph said.

"Dr. Dunbar embodies the very premise of the Gusi award in attaining peace and dignity for fellow humans by his tireless devotion to finding new ways to treat a variety of neurological disorders and for his dedicated efforts in educating the next generation of neuroscientists, physicians, and health-care providers at the highest level," added E. Malcolm Field, Field Neurosciences Institute director.

Presently, Dunbar holds the John G. Kulhavi Professorship in Neuroscience, as well as the E. Malcolm Field Endowed Chair in Neuroscience at CMU.

The Gusi Peace Prize was founded by the Honorable Ambassador Barry Gusi, to honor and continue the work of his late father, Captain Gemeniano Javier Gusi, who fought against Japanese oppression during World War II and later championed human rights in the Philippines. For 20 years, the Gusi Peace Prize Award has been awarded to prominent individuals from all over the world who have made significant contributions to the betterment of humankind.

Here is the original post:
International Peace Prize awarded to Ascension St. Mary's executive director - Midland Daily News

Spinal Cord Stem Cells Comments Off on International Peace Prize awarded to Ascension St. Mary’s executive director – Midland Daily News | December 16th, 2019

Sometimes when hes asleep, Nick Hernandez has this dream.

The 54-year-old Homer Glen man is playing baseball for a professional team. He hits a ball down the line. He smells the grass. Everything is normal, vividly normal.

And then

Hernandez wakes up in a body that had a C-6 spinal injury 37 years ago.

Before the accident, Hernandez played basketball and baseball for the former Joliet Catholic High School (now Joliet Catholic Academy). He was a college prospect for both sports and was to serve as team captain for both sports his senior year.

Hernandez also was a scholar, ranking 30th out of 205 students. His grade-point average was 4.03

I loved Joliet Catholic, Hernandez said, even though I felt like a fish out of water freshman year because all my friends went to Lockport.

Hernandez has remained close to his Catholic High family through the years. And like a good family, this close group of his friends is raising $75,000 to buy Hernandez a new special needs van, which will keep him mobile and independent.

His current van is 19 years old, and Hernandezs mechanic said it soon will be unsafe to drive, Hernandez said.

Hernandez appreciates their friendship and expressed it more than once.

Im the luckiest guy on the face of the earth, he said, despite the injury.

The injury that changed his life happened Aug. 4, 1982.

It was 9:30 p.m. and Hernandez was driving home from a friends house. He was on 143rd Street, less than a mile from home, when two horses jumped out. He remembers the impact, the horses coming down on top of my vehicle before slipping into unconsciousness.

I woke up a few minutes later in the vehicle, Hernandez said. My rearview mirror was bent and facing me, and I had blood streaming down my face. I could not move, and I was in a great deal of pain, neck pain Id never felt before. I kind of knew immediately. I just knew I had a spinal cord injury.

Oddly enough, he said, a friend of the family recently had a neck injury while swimming, an incident Hernandez had only vaguely noted at the time, but something he recalled in that moment, with his vehicle on the opposite side of the road and the nice cut in the top of my head, which would leave a permanent scar.

But a scar was not Hernandezs concern at the time.

My job was just to fight, to try to stay alive, quite honestly, Hernandez said.

His parents, Nick Sr. and Madeleine, soon arrived on the scene; a priest gave last rites on the spot. Hernandez recalled thinking, Well, this is how it ends.

Hernandez said he was taken to Silver Cross to be stabilized and was transferred to Northwestern Memorial Hospital that night, his home until December. He spent three days in the intensive care unit, where he was given morphine to help him sleep, Hernandez said.

He wore a brace that was drilled into his temples to stabilize his spine You feel like Frankenstein, Hernandez said, and he was periodically rotated every two hours, as if he was on a rotisserie, he said.

It was a do-or-die situation, Hernandez said. I guess something inside me said, Youre still alive. You have to. Too many people just cared about me, I guess this was long before Christopher Reeve. I was just a kid who fought to stay alive.

Hernandezs weight dropped from 180 to 130 pounds as his muscles atrophied, he said. He learned to breathe through his diaphragm.

The experience forced him to grow up fast, he said, which he did, with the help of family, friends, teammates and coaches (Mike Gillespie coached Hernandez for basketball ,and Jack Schimanski coached Hernandez for baseball) who visited him.

I never wrapped my arms around how it could be so emotional for someone else, Hernandez said. Its the ripple effect I didnt understand at the time.

When physical therapy was completed, Hernandez returned to Catholic High to complete his senior year and graduated with honors. While there, he attended school events prom, as well as baseball and basketball games.

He eventually relearned to drive, using hand controls in a modified van. It was frightening at first, until Hernandez moved past that fear.

I wanted to be on the road again, Hernandez said. Thats the one thing about driving; it gives you that freedom.

He worked for medical supply companies until he wound up with pressure ulcers in 2010 that led to a bone infection and a flap surgery to correct it.

It was not a good time in my life, Hernandez said.

But it gave him time to reevaluate his life. He moved back to his parents house, where he now lives in a basement apartment. He now focuses his energy on taking better care of himself and spending time with loved ones, including going to movies with his girlfriend.

He still deals with nerve pain, although some days are better than others, he said.

Hernandez exercises at home with the use of a standing table (which puts pressure on his bones and reminds people just how tall he is at 6-foot-4) and swims using a modified butterfly stroke all summer in the family pool.

I just drag my legs behind me, Hernandez said. I may do 30 to 40 laps in the pool. Its really, really helpful for one with a spinal cord injury, or anybody really, when rehabbing. The water is a good way to exercise without putting too much pressure on the body.

Watching athletes, especially his nieces and nephews who play sports, from the sidelines has given Hernandez an appreciation of the capabilities of the human body when all parts work in unison.

And he thinks, I used to do that

But Hernandez has learned to deal with it, he said.

Because the other options arent pretty, Hernandez said. So if not for my family and friends, and obviously my belief in God you have doubts here and there, but I still have my faith I can hang in there if you can laugh with this injury, you can make it another day. As long as the good Lord wants you around, youll still be there. Im not preaching to anybody, but this is how I choose to live.

Hernandez still clings to his dream, his dream of walking one day.

Hes extremely excited about a Nov. 27 report from Mayo Clinic about how STEM cells from a patients fat can improve sensory motor function in people with spinal cord injuries.

If I just keep myself in shape, maybe, if Im here in another five years, maybe I can still walk, Hernandez said. It kind of keeps you motivated.

HOW TO HELP

Visit gofundme.com/f/new-wheels-for-nick-in-2020 or make checks payable to Nick Hernandez and mail to Al Petro, 4712 Galway Road, Joliet, IL 60431.

Visit link:
'I was just a kid trying to stay alive' - The Herald-News

Spinal Cord Stem Cells Comments Off on ‘I was just a kid trying to stay alive’ – The Herald-News | December 16th, 2019

Cell processing involves a series of activities ranging from cells collection from donor, cell extraction till the storage, and cells distribution to receiver for cell therapy. Cell processing includes various processes of cell collection, cell isolation, cell analysis, cell expansion, washing and concentration, preservation, and distribution.

Cell therapy refers to administering of living whole cells in a patient for treating a disease. Cells origin can be from the same individual, known as autologous source or from another individual, known as allogeneic source. For cell therapy, different types of cells can be used, including hematopoietic stem cells, skeletal muscle cells, embryonic stem cells, neural cells, and mesenchymal cells. Cell therapy is used for the treatment of autoimmune diseases, cancers, infectious and urinary diseases, repairmen of spinal cord injuries, rebuilding damaged cartilage in joints, improvement of a weakened immune system, and aiding patients with neurological disorders.

Key Drivers of Global Cell Processing Technologies Market

Increase in demand for cell therapy, as it is the only curative treatment for several diseases, such as autoimmune disease, cancer, and neural disease. This factor is responsible for growth of the global cell processing technologies market.

There is a rise in prevalence of various diseases, such as cancer, immune diseases, neurological disorders, cardiovascular disease, etc. According to the National Cancer Institute, in 2016, there were an estimated 15.5 million cancer survivors in the U.S. The number of cancer survivors is anticipated to increase to 20.3 million by 2026.

Request a PDF Sample on Cell Processing Technologies Market Report

https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=73602

Personalized medicines or precision medicines with advanced treatments such as gene therapy and cell therapy are witnessing a surge in their adoption, as most of the key biotechnology and pharmaceutical players are heavily investing in these technologies

Government investments in cell-based researches, rise in the number of GMP-certified production facilities, and increase in clinical trials of various diseases are key factors propelling the growth of the global cell processing technologies market

Key Restraints of Global Cell Processing Technologies Market

Cell processing is a complex procedure with high probabilities of failure. Thus, its complexity and accuracy demand technologically advanced and high-tech infrastructure, along with a skilled operating staff. These incur high infrastructural and operating costs, which limit its adoption on a large scale.

High operating cost also leads to high cell therapy cost to patients. However, at present, the cell therapy is approved for a limited number of conditions, which further limits the usage of cell processing technologies and services

Equipment Segment of Cell Processing Technologies Market to Witness Strong Growth

Complexity in cell processing steps and accuracy required for procedure has led to the development of advanced automated cell processing systems. Key players have been heavily investing in the development of advanced cell processing systems. Advancements in software for managing these systems are projected to expand the application areas for cell processing units.

In May 2018, GE Healthcare introduced the Sefia S-2000 cell processing system, which is advanced than its predecessor Sefia S-1000 cell processing system. This advanced system was developed with focus on chimeric antigen receptor (CAR) T-cell therapy.

Investments in CAR-T and other cell and gene therapy products are projected to drive the adoption of cell processing equipment. As the installed base for cell processing systems is projected to surge, the demand for consumables is likely to grow during the forecast period.

Pre Book this Report

https://www.transparencymarketresearch.com/checkout.php?rep_id=73602&ltype=S

Oncology is projected to be the most promising application area of cell processing technology, considering high investments and ongoing research in cell therapies for cancer treatment and high investment made by key biotechnology and pharmaceutical companies in this area.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving method-ologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of re-search is the key to help enterprises reach right decision.

ContactMr. Rohit BhiseyTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY 12207United StatesTel: +1-518-618-1030USA Canada Toll Free: 866-552-3453Email: sales@transparencymarketresearch.comWebsite: http://www.transparencymarketresearch.comBlog: https://theglobalhealthnews.com/

Read the original here:
Cell Processing Technologies Market Segments, Leading Player, Application and Forecast Analysis - Techi Labs

Spinal Cord Stem Cells Comments Off on Cell Processing Technologies Market Segments, Leading Player, Application and Forecast Analysis – Techi Labs | December 16th, 2019