An international collaboration involving Monash University and Duke-NUS researchers have made an unexpected world-first stem cell discovery that may lead to new treatments for placenta complications during pregnancy.

While it is widely known that adult skin cells can be reprogrammed into cells similar to human embryonic stem cells that can then be used to develop tissue from human organs - known as induced pluripotent stem cells (iPSCs) - the same process could not create placenta tissue.

iPSCs opened up the potential for personalised cell therapies and new opportunities for regenerative medicine, safe drug testing and toxicity assessments, however little was known about exactly how they were made.

An international team led by ARC Future Fellow Professor Jose Polo from Monash University's Biomedicine Discovery Institute and the Australian Research Medicine Institute, together with Assistant Professor Owen Rackham from Duke-NUS in Singapore, examined the molecular changes the adult skin cells went through to become iPSCs. It was during the study of this process that they discovered a new way to create induced trophoblast stem cells (iTSCs) that can be used to make placenta cells.

This exciting discovery, also involving the expertise of three first authors, Dr. Xiaodong Liu, Dr. John Ouyang and Dr. Fernando Rossello, will enable further research into new treatments for placenta complications and the measurement of drug toxicity to placenta cells, which has implications during pregnancy.

"This is really important because iPSCs cannot give rise to placenta, thus all the advances in disease modelling and cell therapy that iPSCs have brought about did not translate to the placenta," Professor Polo said.

"When I started my PhD five years ago our goal was to understand the nuts and bolts of how iPSCs are made, however along the way we also discovered how to make iTSCs," said Dr Liu.

"This discovery will provide the capacity to model human placenta in vitro and enable a pathway to future cell therapies," commented Dr Ouyang.

"This study demonstrates how by successfully combining both cutting edge experimental and computational tools, basic science leads to unexpected discoveries that can be transformative," Professor Rackham said.

Professors Polo and Rackham said many other groups from Australian and international universities contributed to the study over the years, making it a truly international endeavour.

Reference:Liu, X., Ouyang, J.F., Rossello, F.J. et al. Reprogramming roadmap reveals route to human induced trophoblast stem cells. Nature (2020). https://doi.org/10.1038/s41586-020-2734-6

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Continued here:
Researchers Discover a Way To Create Induced Tropoblast Stem Cells - Technology Networks

Cardiac Stem Cells Comments Off on Researchers Discover a Way To Create Induced Tropoblast Stem Cells – Technology Networks | September 18th, 2020

Sept. 15, 2020 16:00 UTC

Curis Platforms are Accelerating Drug Discovery with Human-Relevant 3D Engineered Muscle Tissue Analysis

SEATTLE--(BUSINESS WIRE)-- Curi Bio, a leading developer of human stem cell-based platforms for drug discovery, today announced the Mantarray platform for human-relevant 3D engineered muscle tissue (EMT) analysis. Curis Mantarray platform enables the discovery, safety, and efficacy testing of new therapeutics by providing parallel analysis of 3D EMTs with adult human-like functional profiles. By providing drug developers human-relevant tissue-specific biosystems in the preclinical stage of drug development, Curi aims to help pharmaceutical partners develop safer and more effective therapeutics in less time, at lower cost. Curi Bio is currently partnering with several leading pharmaceutical companies to accelerate the development of the Mantarray platform and to apply it to drug discovery and development projects.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200915005905/en/

A 3D engineered muscle tissue in Curis Mantarray platform. (Graphic: Business Wire)

Cardiovascular diseases often involve a gradual loss of cardiac contractile strength and function, ultimately leading to heart failure. Cardiomyocytes derived from human induced pluripotent stem cells (iPSC-CMs) offer a promising route to model the contractile deficiencies seen in the hearts of patients with cardiovascular diseases. However, 2D cell models lack the physiologically relevant structure and function of 3D models. As a result, 3D engineered muscle tissues have been growing in use in the drug development industry. Yet existing 3D EMT solutions are complex and low throughput, often relying on laborious serial optical imaging of each tissue to measure contractility.

Curi will make the Mantarray platform available to pharmaceutical and research customers as a standalone bioscience instrument together with multiwell consumable plates for casting and assaying EMTs. Curi will also offer service contracts and partnerships leveraging the Mantarray technology for applications in drug discovery, disease modeling, and safety and efficacy screening. Curis Mantarray platform leverages a proprietary, label-free, non-optical, electromagnetic measurement system for direct contractility assessment of up to 24 parallel iPSC-derived 3D engineered muscle tissues simultaneously. With the Mantarray platform, scientists can achieve clinically relevant functional measurements of human iPSC-derived engineered muscle tissue contractility, with a throughput and reproducibility compatible with higher-throughput screening workflows. Mantarray brings clinically relevant functional data into the earliest stages of preclinical testing of new medicines.

Leveraging human iPSC-derived cells, Mantarray 3D tissues can be used to create high-fidelity models of human diseases. For example, Mantarray 3D EMTs can be gene-edited with a CRISPR/Cas9 system to model human diseases such as Duchenne muscular dystrophy and various cardiomyopathies. Multi-modal Mantarray data show enhanced disease stratification providing researchers with more physiological data for the discovery and validation of new therapeutics.

The Mantarray platform also provides a breakthrough cardiotoxicity safety and efficacy testing platform with novel magnetic detection of drug-induced contractile changes. The magnetic detection approach can measure both acute and chronic drug responses. Drugs can be measured on the order of seconds to minutes with enough sensitivity to measure dose-response-like behavior. Alternatively, longer-term chronic experiments can be performed over the course of days. Applications include acute and chronic structural cardiotoxicity evaluation.

At Curi Bio, our goal is to provide researchers with innovative human-relevant cells, systems, and data to accelerate the discovery of new medicines, said Curi CEO Michael Cho. By providing drug developers unprecedented access to clinically-relevant preclinical models that more closely recapitulate human cardiac and skeletal muscle tissue, Curi is closing the gap between preclinical results and clinical impact.

Dr. Nicholas Geisse, Chief Science Officer of Curi Bio, will present Curis Mantarray platform and Curis recently announced ComboMat platform in a presentation at the Discovery on Target 2020 Virtual Conference.

Event: Discovery on Target 2020Date: Thursday, September 17, 2020Time: 11:15 AM EDTSession: Disease ModelingTitle: Structural Maturation in the Development of hiPSC-Cardiomyocyte Models for Preclinical Safety, Efficacy, and Discovery

Curis Mantarray platform integrates proprietary methods and IP exclusively licensed to Curi Bio by the University of Washington.

To learn more about how the Mantarray platform can improve the predictive power of 3D EMTs, or about Curis other human-relevant preclinical platform technologies and services, please reach out at http://www.curibio.com/contact.

About Curi Bio

Curi Bios preclinical discovery platform combines human stem cells, systems, and data to accelerate the discovery of new medicines. The Curi Engine is a seamless, bioengineered platform that integrates human iPSC-derived cell models, tissue-specific biosystems, and AI/ML-enabled phenotypic screening data. Curis suite of human stem cell-based products and services enable scientists to build more mature and predictive human iPSC-derived tissueswith a focus on cardiac, musculoskeletal, and neuromuscular modelsfor the discovery, safety testing, and efficacy testing of new drugs in development. By offering drug developers an integrated preclinical platform comprising highly predictive human stem cell models to generate clinically-relevant data, Curi is closing the gap between preclinical data and human results, accelerating the discovery and development of safer, more effective medicines.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200915005905/en/

Read more:
Curi Bio Announces Mantarray Platform for Analysis of 3D Engineered Muscle Tissues for Discovery of New Therapeutics - BioSpace

Cardiac Stem Cells Comments Off on Curi Bio Announces Mantarray Platform for Analysis of 3D Engineered Muscle Tissues for Discovery of New Therapeutics – BioSpace | September 18th, 2020

TOKYO and BOTHELL, Wash., Sept. 18, 2020 /PRNewswire/ --Astellas Pharma Inc.(TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Seattle Genetics, Inc. (Nasdaq:SGEN) today announced that a phase 3 trial of PADCEV (enfortumab vedotin-ejfv) met its primary endpoint of overall survival compared to chemotherapy. The results were reviewed by an independent Data Monitoring Committee following a planned interim analysis. The global EV-301 clinical trial compared PADCEV to chemotherapy in adult patients with locally advanced or metastatic urothelial cancer who were previously treated with platinum-based chemotherapy and a PD-1/L1 inhibitor.

In the trial, PADCEV significantly improved overall survival (OS), with a 30 percent reduction in risk of death (Hazard Ratio [HR]=0.70; [95% Confidence Interval (CI): 0.56, 0.89]; p=0.001). PADCEV also significantly improved progression-free survival (PFS), a secondary endpoint, with a 39 percent reduction in risk of disease progression or death (HR=0.61 [95% CI: 0.50, 0.75]; p<0.00001).

For patients in the PADCEV arm of the trial, adverse events were consistent with those listed in the U.S. Prescribing Information, with rash, hyperglycemia, decreased neutrophil count, fatigue, anemia and decreased appetite as the most frequent Grade 3 or greater adverse event(s) occurring in more than 5 percent of patients.Data from EV-301 will be submitted for presentation at an upcoming scientific congress. Patients in the chemotherapy arm of the trial will be offered the opportunity to receive PADCEV.

The results will be submitted to the U.S. Food and Drug Administration (FDA) as the confirmatory trial following the drug's accelerated approval in 2019. EV-301 is also intended to support global registrations.

"EV-301 is the first randomized trial to show overall survival results compared to chemotherapy in patients with locally advanced or metastatic urothelial cancer who previously have received platinum-based treatment and a PD-1 or PD-L1 inhibitor, and we are encouraged by the potential this may have in helping patients who have otherwise limited alternatives," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas. "We look forward to discussing these results with global health authorities."

"These survival results from the confirmatory trial for PADCEV are welcome news for patients whose cancer has progressed after platinum-based chemotherapy and immunotherapy," said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics. "We continue to explore PADCEV's activity across the spectrum of urothelial cancer including its potential for use in earlier lines of therapy."

Globally, approximately 580,000 people will be diagnosed with bladder cancer in 2020.1Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis (where urine collects inside the kidney), ureter (tube that connects the kidneys to the bladder) and urethra.2Approximately 80 percent of people do not respond to PD-1 or PD-L1 inhibitors after a platinum-containing therapy has failed as an initial treatment for advanced disease.3

About the EV-301 TrialThe EV-301 trial (NCT03474107) is a global, multicenter, open-label, randomized phase 3 trial designed to evaluate PADCEV versus physician's choice of chemotherapy (docetaxel, paclitaxel or vinflunine) in approximately 600 patients with locally advanced or metastatic urothelial cancer who were previously treated with a PD-1 or PD-L1 inhibitor and platinum-based therapies. The primary endpoint is overall survival of participants treated with PADCEV compared to those treated with chemotherapy. Secondary endpoints include progression-free survival, duration of response, and overall response rate, as well as assessment of safety/tolerability and quality-of-life parameters.

For more information about the EV-301 clinical trial, please visit http://www.clinicaltrials.gov.

About PADCEV (enfortumab vedotin-ejfv)PADCEV was approved by the U.S. Food and Drug Administration (FDA) in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDA's Accelerated Approval Program based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.4

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.4,5 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).4 PADCEV is co-developed by Astellas and Seattle Genetics.

PADCEV Important Safety Information

Warnings and Precautions

Adverse ReactionsSerious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab AbnormalitiesIn one clinical trial, Grade 3-4 laboratory abnormalities reported in 5% were: lymphocytes decreased (10%), hemoglobin decreased (10%), phosphate decreased (10%), lipase increased (9%), sodium decreased (8%), glucose increased (8%), urate increased (7%), neutrophils decreased (5%).

Drug Interactions

Specific Populations

For more information, please see the full Prescribing Information for PADCEV here.

About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+ healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en/.

About Seattle Genetics Seattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in people's lives. The company is headquartered in the Seattle, Washington area, with locations in California, Switzerland and the European Union. For more information on our robust pipeline, visit http://www.seattlegenetics.comand follow @SeattleGeneticson Twitter.

About the Astellas and Seattle Genetics CollaborationAstellas and Seattle Genetics are co-developing PADCEV (enfortumab vedotin-ejfv) under a 50:50 worldwide development and commercialization collaboration that was entered into in 2007 and expanded in 2009.

Astellas Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties.

Information about pharmaceutical products (including products currently in development), which is included in this press release is not intended to constitute an advertisement or medical advice.

Seattle Genetics Forward Looking Statements Certain statements made in this press release are forward looking, such as those, among others, relating to the submission of data from the EV-301 trial for presentation at an upcoming scientific congress; intended regulatory actions, including plans to submit the results of the EV-301 trial to the FDA as the confirmatory trial following the drug's accelerated approval in the U.S. and plans to discuss the results with global health authorities and seek global registrations; conduct of a comprehensive clinical development program for PADCEV, which includes exploring PADCEV's activity in other types of urothelial cancer and its potential for use in earlier lines of therapy;the therapeutic potential of PADCEV,including its efficacy, safety and therapeutic uses, and anticipated development activities, including ongoing and future clinical trials. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include that the data from the EV-301 trial may not be selected for presentation at scientific congresses; the possibility of delays in the submission of results to the FDA; that the results from the EV-301 trial may not be enough to convert PADCEV's accelerated approval in the U.S. to regular approval or to support any other global registrations; that, even if PADCEV receives regular approval in the U.S. or any other global registrations, the product labeling may not be as broad or desirable as anticipated; the possibility that ongoing and subsequent clinical trials may fail to establish sufficient activity; the risk of adverse events or safety signals; and the possibility that adverse regulatory actions may occur. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption "Risk Factors" included in the company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

1 International Agency for Research on Cancer. Cancer Tomorrow: Bladder. http://gco.iarc.fr/tomorrow. Accessed 07-31-2020.2 American Society of Clinical Oncology. Bladder cancer: introduction (10-2017).3 Shah, Manasee V., et al "Targeted Literature Review of the Burden of Illness in UC" (PCN108), Nov 2018.4PADCEV [package insert] Northbrook, IL: Astellas, Inc.5Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.

SOURCE Astellas Pharma Inc.

https://www.astellas.com/en/

More here:
Astellas and Seattle Genetics Announce PADCEV (enfortumab vedotin-ejfv) Significantly Improved Overall Survival in Phase 3 Trial in Previously Treated...

Cardiac Stem Cells Comments Off on Astellas and Seattle Genetics Announce PADCEV (enfortumab vedotin-ejfv) Significantly Improved Overall Survival in Phase 3 Trial in Previously Treated… | September 18th, 2020

NEW YORK (PRWEB) September 15, 2020

Derm Institute Introduces New Product To Erase Age Spots(New CB Spot Treatment AddressesDark Blemishes)Derm Institute, producer of an award-winning skincare line that blends DNA and stem cell technology with traditional therapeutic herbs, has introduced a new product, CellularBrightening Spot Treatment (a.k.a., CB Spot Treatment), that eraces age spots. Also called liver spots or sun spots, these dark blemishes often appear as we age and tend to affect the hands, face, backs and shoulders.Derm Institute Operations Director Annie Hsu says thatCB Spot Treatment was developed for all skin types and is formulated not only to fade age spots but to promote more radiant, firmer skin.

Though most often caused by repeated exposure to the sun and/or tanning beds, age spots also can be caused by hormonal changes, side effects of certain medications (e.g., non-steroidal anti-inflammatories, tetracyclines and psychotropic drugs), wound healing and irritation caused by some cosmetics and hair products.

Getting rid of age spots can be one of the most frustrating and challenging aspects of caring for our skin and achieving a more youthful appearance, says Hsu. Our new CB Spot Treatment is a departure from other products on the market because it contains LightninC Complex, a proprietary mix of ingredients that inhibit the production of tyrosinase and melanogenesis, which are known for producing age spots.

Hsu says that LightninC Complex contains 48% vitamin C which is 30 times more effective than Kojic acid in reducing age spots, increasing collagen synthesis and protecting collagen to improve firmness. In addition, CB Spot Treatment is hydroquinone free.

Our formulation does not harm or irritate the skin like some other products on the market, she said. Rather, it significantly protects, regenerates and hydrates damaged skin.

Hsu says that CB Spot Treatment contains Vitamin A, a natural antioxidant that helps stimulate collagen, reverses the signs of prematurely-induced photo-aging and increases skin elasticity as well as vitamin E that scavenges damaging free radicals and protects the skin from UV damage.

The 30 ml CB Spot Treatment is priced at $290 and will be available exclusively at Space NK Apothecary, Space NK counters in Bloomingdales, Nordstrom, Bergdorf Goodman and online at DISkinCare.com.DERM INSTITUTES BACKGROUND:Derm Institute was founded in 2009 by renowned skincare researcher Young Lin who created a product to improve his father's skin, which was badly scarred and hypersensitive following chemo treatments. He used powerful herbal antioxidants to create a therapeutic ointment to reverse the damage and restore his skin back to its original health. Today, Derm Institute continues to use medical-grade ingredients as well as Eastern therapeutic herbs and plant stem cells as the foundation for its line of skin care products. The ingredients are derived from the stems and seeds of plants, which are the most durable part of the plant and least affected by pollution. In addition, its team of scientists employ the use of a microarray, a high-tech device used in the pharmaceutical industry, to test ingredients and examine gene activity. Made in America, Derm Institute products contain a potent combination of antioxidants, groundbreaking peptides and advanced delivery systems designed to achieve instant and cumulative results. For more information, visithttp://www.DISkincare.com.*****CONTACT (323) 378-1403

Share article on social media or email:

Visit link:
Derm Institute's Newest Product Erases Age Spots (Users Can Rid Their Skin Of Ugly Dark Blemishes With New CB Spot Treatment) - PR Web

Skin Stem Cells Comments Off on Derm Institute’s Newest Product Erases Age Spots (Users Can Rid Their Skin Of Ugly Dark Blemishes With New CB Spot Treatment) – PR Web | September 16th, 2020

Brady Feeney hadnt even taken any classes at Indiana University when he fell ill with Covid-19. Three weeks after he moved to Bloomington, the incoming freshman was in the emergency room, struggling to breathe. Before his illness, Feeney had been a perfectly healthy teenager, with no preexisting conditions. In high school, he was a three-time all-state football player and won two state titles in Missouri. But after two weeks of hell fighting the virus, his mother said, his bloodwork indicated possible heart problems.

When SARS-CoV-2 first struck the United States, the medical community had two working assumptions: First, this was primarily a respiratory disease, and second, it seemed to hit older people much harder than younger people, with eight out of 10 confirmed Covid-19 deaths in the U.S. happening in adults 65 or older. But now, new research is challenging both of these assumptions.

Growing evidence suggests that SARS-CoV-2 doesnt only infect the lungs. It also affects the brain, kidneys, and heart. At first, doctors and researchers wondered if these issues beyond the lungs came just from the stress of having Covid-19 and being on a ventilator or life support. But increasingly, research indicates that the virus may be attacking other organs in the body directlyand this may be more common than previously thought, even among those who arent sick enough to be hospitalized. Some have suggested that Covid-19 is actually a blood vessel disease; the lungs are merely the way the virus enters the body, but from there it gets into the bloodstream and takes up residence in major organs, leaving patients with complex, long-lasting symptoms. Moreover, experts now believe, healthy young people can get mild cases of the coronaviruseven not knowing they were sickthat could leave them with lasting cardiovascular damage. Even those who seem to have recovered from the deadly respiratory illness are not free of its complications.

Heart failure could be the next chapter of the coronavirus illness, Dr. Gregg C. Fonarow, interim chief of UCLAs Division of Cardiology, recently argued in a co-authored editorial in the journal JAMA Cardiology. Even if in younger adults Covid-19 may not be fatal, there still may be important health consequences, he told me.

Myocarditis, or inflammation of the heart, is usually a rare condition that can occur with viral infections, including the flu. But from the start of the pandemic, doctors were seeing heart inflammation among patients hospitalized with serious cases of Covid-19, Fonarow said: Early research showed that 20 to 30 percent of those hospitalized had heart issues. Left untreated, myocarditis can damage the heart and lead to heart attacks and arrhythmias, among other complications.

Read more here:
What Is Covid-19 Doing to Our Hearts? - The New Republic

Cardiac Stem Cells Comments Off on What Is Covid-19 Doing to Our Hearts? – The New Republic | September 16th, 2020

It is September of a presidential election year, with a divided government and a deeply polarized electorate. Many congressional watchers would tell you that absolutely nothing could pass in this environment. Earlier this month, however, in defiance of the naysayers and in the spirit of all too rare bipartisanship, the House Energy and Commerce Committee overwhelmingly voted to reauthorize the C.W. Bill Young Cell Transplantation Program (the Program).

We must extend this demonstration of bipartisanship and willingness to work together to save American lives to the full House and the Senate and ensure that the Program is reauthorized before it expires at the end of September.

For 30 years, Congress has authorized and re-authorized the Program, which provides access to life-saving bone marrow, peripheral blood stem cell and cord blood transplants for patients living with one of the more than 70 blood cancers or blood disorders for which transplant is the only curative option.

The Program was first established through a partnership with the Navy in 1986, transferred to the NIH for oversight the following year, then authorized by Congress in 1994 and has been reauthorized in 1998, 2005, 2010, and 2015.

During those 30 years, the National Marrow Donor Program/Be The Match (NMDP) has facilitated more than 100,000 lifesaving and life-extending transplants.

While the Program enjoys broad bipartisan and bicameral support, as it has during every other reauthorization, it is still in jeopardy which is why we strongly urge Congress to act swiftly to protect the Program and the patients who rely on it.

Because of its unassailable mission, because of its great successes over three decades, and because the Program is relied upon by more patients than ever before, reauthorization of the national registry remains non-controversial.

Reauthorization was critical before the pandemic, but now because of the wild unpredictability of COVID-19, and its downstream impacts that create life-threatening risk to bone marrow transplant patients, it is even more urgent that Congress reauthorize the Program before adjourning for the election.

Thanks in large part to the national program status afforded to NMDP under the authorizing statue, we were:

These critical victories during the pandemic share two common, and extraordinarily important, threads. First, we would not have been able to secure these victories without NMDPs national program status. And, second, patients in the U.S. would have died had these things not happened.

While we have no way to predict exactly what the next emergencies will look like, we nevertheless know with certainty that they will happen. Based upon the unfathomable experience of the last six months, we know that the lynch pin to the solution will be our national program status.

This is exactly why reauthorization now is so critical.

No lapse in the Program means no lapse in our national program status. And that means no lapse in our ability to move mountains in our efforts to leave no patient without the transplant that they so desperately need.

The members of the House Energy & Commerce Committee proved that even in these most trying political times, Congress can come together and work across the aisle to save the lives of Americans in need. We hope the full House and the Senate will follow suit and act quickly to reauthorize the Program.

Brian Lindberg is the chief legal officer and chief policy officer for the National Marrow Donor Program/Be The Match.

Read more:
Full House and Senate should reauthorize the C.W. Bill Young Cell Transplantation Program | TheHill - The Hill

Bone Marrow Stem Cells Comments Off on Full House and Senate should reauthorize the C.W. Bill Young Cell Transplantation Program | TheHill – The Hill | September 16th, 2020

The parents of a cancer-stricken toddler have launched a desperate search for a bone marrow donor to step forward and save their daughters life.

Three-year-old Adeline Davidson, from Alness, suffers with a rare form of blood cancer which affects only one in every 250,000 children.

Her only hope of survival is finding a suitable bone marrow donor.

Earlier this year her parents Steph, 26, and Jordan, 28, were ecstatic to have found two viable donors taking them one step closer to saving the happy go lucky youngster.

However, their world came crumbling down on Monday when consultants told them that both were no longer viable and there were no matching donors left on the register.

The heartbroken mum said the news has left them at a loss and right back to where they were almost two years ago.

She said: Its absolutely devastating.

We got a phone call and I thought it was a date for a transplant, so this was the last thing I was expecting.

I didnt actually process anything she said until I was off the phone and I just thought what on earth does that even mean for us now?

It felt like someone had just stabbed me in the gut. It was horrendous.

We have literally no idea how long we are going to be waiting for.

I cant put into words how heartbroken we are after finding out Adeline no longer has a donor available and we are back

Posted by Steph Davidson onMonday, 14 September 2020

Sharing the heartbreaking news on social media, the 26-year-old mother of-three said she couldnt explain the pain she felt daily knowing Adeline wasnt experiencing life as a normal healthy three-year-old.

She added: We were ecstatic when we first found out there even was a match because its a long process and a long wait.

But to have that taken away from you and to be left with nothing after all this time, I honestly cant explain it.

Adeline is so happy and a part of me is happy for that, but also it saddens me because I just think she has no idea what she is missing out on.

Mrs Davidson says for Adeline, everyday health problems such as a common cold, virus or sickness bug could be fatal.

If her bone marrow failure is left, it could develop leading her to become unwell and unstable or develop conditions such as leukaemia if her body begins producing abnormal cells.

The Ross-shire family is now pleading with individuals to sign up to become a stem cell or bone marrow donor to help save terminally ill children such as Adeline.

Mrs Davidson said: The only thing that we can do is try and search for this match.

Only 0.04% of the population are on the register so the facts are against us but I just want more hope.

I just really hope that people are convinced to sign up and share the word elsewhere.

It means the world when people say I have signed up because of your story.

However, many individuals that are signing up, they possibly cant be the match for Adeline but they possibly could be for someone else. There is so many people like Adeline that are waiting for this.

Continue reading here:
Highland family issue urgent plea to help find life-saving bone marrow donor for three-year-old daughter - Press and Journal

Bone Marrow Stem Cells Comments Off on Highland family issue urgent plea to help find life-saving bone marrow donor for three-year-old daughter – Press and Journal | September 16th, 2020

Differences in biological sex can dictate lifelong disease patterns, says a new study by Michigan State University researchers that links connections between specific hormones present before and after birth with immune response and lifelong immunological disease development.

Published in the most recent edition of the Proceedings of the National Academy of Sciences, the study answers questions about why females are at increased risk for common diseases that involve or target the immune system like asthma, allergies, migraines and irritable bowel syndrome. The findings by Adam Moeser, Emily Mackey and Cynthia Jordan also open the door for new therapies and preventatives.

This research shows that its our perinatal hormones, not our adult sex hormones, that have a greater influence on our risk of developing mast cell-associated disorders throughout the lifespan, says Moeser, Matilda R. Wilson Endowed Chair, professor in the Department of Large Animal Clinical Sciences and the studys principle investigator. A better understanding of how perinatal sex hormones shape lifelong mast cell activity could lead to sex-specific preventatives and therapies for mast cell-associated diseases."

Mast cells are white blood cells that play beneficial roles in the body. They orchestrate the first line of defense against infections and toxin exposure and play an important role in wound healing, according to the study, Perinatal Androgens Organize Sex Differences in Mast Cells and Attenuate Anaphylaxis Severity into Adulthood.

However, when mast cells become overreactive, they can initiate chronic inflammatory diseases and, in certain cases, death. Moesersprior research linked psychological stress to a specific mast cell receptor and overreactive immune responses.

Moeser also previously discovered sex differences in mast cells.Female mast cells store and release more inflammatory substanceslike proteases, histamine and serotonin, compared with males. Thus, female mast cells are more likely than male mast cells to kick-start aggressive immune responses. While this may offer females the upper hand in surviving infections, it also can put females at higher risk for inflammatory and autoimmune diseases.

IBS is an example of this, says Mackey, whose doctoral research is part of this new publication.

While approximately 25% of the U.S. population is affected by IBS,women are up to four times more likelyto develop this disease than men.

Moeser, Mackey and Jordans latest research explains why these sex-biased disease patterns are observed in both adults and prepubertal children. They found that lower levels of serum histamine and less-severe anaphylactic responses occur in males because of their naturally higher levels of perinatal androgens, which are specific sex hormones present shortly before and after birth.

Mast cells are created from stem cells in our bone marrow, Moeser said. High levels of perinatal androgens program the mast cell stem cells to house and release lower levels of inflammatory substances, resulting in a significantly reduced severity of anaphylactic responses in male newborns and adults.

We then confirmed that the androgens played a role by studying males who lack functional androgen receptors, says Jordan, professor of Neuroscience and an expert in thebiology of sex differences.

While high perinatal androgen levels are specific to males, the researchers found that while in utero, females exposed to male levels of perinatal androgens develop mast cells that behave more like those of males.

For these females, exposure to the perinatal androgens reduced their histamine levels and they also exhibited less-severe anaphylactic responses as adults, says Mackey, who is currently a veterinary medical student at North Carolina State University.

In addition to paving the way for improved and potentially novel therapies for sex-biased immunological and other diseases, future research based will help researchers understand how physiological and environmental factors that occur early in life can shape lifetime disease risk, particularly mast cell-mediated disease patterns.

While biological sex and adult sex hormones are known to have a major influence on immunological diseases between the sexes, were learning that the hormones that we are exposed to in utero may play a larger role in determining sex differences in mast cell-associated disease risk, both as adults and as children, Moeser said.

Reference: Mackey E, Thelen KM, Bali V, et al. Perinatal androgens organize sex differences in mast cells and attenuate anaphylaxis severity into adulthood. Proc Natl Acad Sci USA. Published online September 11, 2020:201915075. doi:10.1073/pnas.1915075117.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Excerpt from:
How the Hormones You're Born With Influence Disease Risk - Technology Networks

Bone Marrow Stem Cells Comments Off on How the Hormones You’re Born With Influence Disease Risk – Technology Networks | September 16th, 2020

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

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

To remain ahead of your competitors, request for a sample[emailprotected]

https://www.persistencemarketresearch.com/samples/14563

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

To receive extensive list of important regions, Request Methodology here @

https://www.persistencemarketresearch.com/methodology/14563

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

Explore Extensive Coverage of PMR`s

Life Sciences & Transformational HealthLandscape

About us:

Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics andmarket research methodologyto 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.

Our client success stories feature a range of clients from Fortune 500 companies to fast-growing startups. PMRs collaborative environment is committed to building industry-specific solutions by transforming data from multiple streams into a strategic asset.

Contact us:

Naved BegPersistence Market ResearchAddress 305 Broadway, 7th Floor, New York City,NY 10007 United StatesU.S. Ph. +1-646-568-7751USA-Canada Toll-free +1 800-961-0353Sales[emailprotected]Websitehttps://www.persistencemarketresearch.com

View post:
Hematopoietic Stem Cells Transplantation Market to Witness a Substantiation between 2017 and 2025 - Scientect

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cells Transplantation Market to Witness a Substantiation between 2017 and 2025 – Scientect | September 16th, 2020

NEW YORK, Sept. 15, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that its lead product candidate remestemcel-L has been selected as the winner of the Fierce Innovation Awards - Life Sciences Edition 2020 for Biotech Innovation.The Fierce Innovation Awards is a peer-reviewed program from the publisher ofFierceBiotech and FiercePharma.

Mesoblast Chief Executive Dr Silviu Itescu stated: This important award is recognition of Mesoblasts leadership as an innovator in the cell therapy industry, and of the potential for remestemcel-L to profoundly impact the lives of children suffering with steroid-refractory acute graft versus host disease (SR-aGVHD).

Remestemcel-L is under priority review by the United States Food and Drug Administration (FDA) for pediatric SR-aGVHD and, if approved, product launch in the United States is expected in 2020. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2020.

Remestemcel-L is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is thought to have immunomodulatory properties to counteract the cytokine storms that are implicated in various inflammatory conditions by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

Given the extensive inflammatory response in COVID-19 infection, remestemcel-L is also being evaluated in a randomized, controlled Phase 3 trial in up to 300 ventilator-dependent adults with moderate to severe acute respiratory distress syndrome (ARDS), the primary cause of mortality in COVID-19 patients. The trial aims to confirm results from a pilot study at New Yorks Mt Sinai hospital which showed that nine of 12 patients (75%) were successfully discharged from hospital a median of 10 days after receiving two intravenous doses of remestemcel-L within five days.The trials independent Data Safety Monitoring Board (DSMB) recently completed an interim analysis of the trials first 30% enrolled patients and recommended that the trial should continue as planned after reviewing all safety data and results for the trials primary endpoint of all-cause mortality within 30 days of randomization.The DSMB will perform a second interim analysis when 45% of the enrollment target has completed 30 days of follow-up. About Fierce Innovation Awards Life Sciences Edition 2020These awards highlight companies that demonstrate innovative solutions, technologies, and services that have the potential to make the greatest impact for biotech and pharma companies.The evaluation criteria are effectiveness, technical innovation, competitive advantage, financial impact, and true innovation.The awards programs applications were reviewed by a panel of executives from majorbiotech and pharmacompanies includingAstellas, Accenture, AstraZeneca, Angiocrine Bioscience, Biotech Research Group, NIHR Clinical Research Network, Medidata Solutions and PPD.

About MesoblastMesoblast Limited (Nasdaq:MESO; ASX:MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblast has a strong and extensive global intellectual property (IP) portfolio with protection extending through to at least 2040 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblasts Biologics License Application to seek approval of its product candidate RYONCIL (remestemcel-L) for pediatric steroid-refractory acute graft versus host disease has been accepted for priority review by the United States Food and Drug Administration (FDA), and if approved, product launch in the United States is expected in 2020. Remestemcel-L is also being developed for other inflammatory diseases in children and adults including moderate to severe acute respiratory distress syndrome (ARDS). Mesoblast is completing Phase 3 trials for its product candidates for advanced heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the timing, progress and results of Mesoblasts preclinical and clinical studies; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts product candidates, if approved; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

For further information, please contact:

Go here to read the rest:
Mesoblast Wins 2020 Fierce Biotech Innovation of the Year Award for remestemcel-L - GlobeNewswire

Bone Marrow Stem Cells Comments Off on Mesoblast Wins 2020 Fierce Biotech Innovation of the Year Award for remestemcel-L – GlobeNewswire | September 16th, 2020

Written by Ralph Alex Arakal | Bengaluru | Updated: September 14, 2020 8:12:24 amAhmed and his family after the successful bone marrow transplant (Express photo)

A three-year-old girl from Iraq became a lifesaver for her 18-year-old brother after she donated her bone marrow for a successful transplantation that took place in Bengaluru.

Ahmed had undergone splenectomy in his native country and was referred to Manipal Hospitals in Bengaluru since only optimal treatment is available in Iraq. According to doctors at the hospital, the teenager was also suffering from symptomatic anemia (needing frequent blood transfusions) and jaundice.

Dr Mallikarjun Kalashetty, consultant Haematology, Haemato-Oncology & Bone Marrow Transplantation at Manipal Hospitals, said Ahmed required an allogeneic bone marrow transplantation.

The best donors for such patients are the human leukocyte antigen (HLA)-matched siblings who are normal or with a minor form of haemoglobinopathy (a hereditary condition involving an abnormality in the structure of haemoglobin) or thalassaemia (a blood disorder involving lower-than-normal amounts of an oxygen-carrying protein), Dr Kalashetty explained.

However, things were not easy for the medical team at the hospital considering the age of the donor the patients three-year-old younger sister and the obvious language barrier. Transfusion experts at the hospital soon realised the process was challenging as they required the processing of 8-10 litres of blood from the donor aged three, weighing 18 kilograms, who had only a blood volume of about 1.3 litres.

Considering her age, the donor had to be sedated to elicit co-operation during apheresis (extracting blood and separating components) in multiple sittings and preserve the stem cells through cryopreservation. To counter the low volume of blood going into the apheresis machine, we filled the dead spaces with compatible RBC, and to reduce the fluid overload, we determined and monitored the volume of the fluid going into the body of the child, Dr C Shivaram, consultant transfusion medicine said.

However, the allogeneic bone marrow transplantation was successful and Ahmed has now recovered from the sickle-cell disease. He did have few complications after transplantation like mucositis, febrile neutropenia, and viral reactivation, which were successfully managed, Dr Kalashetty said.

Ahmeds quality of life has improved significantly and his parents are overjoyed to see their son doing so well after suffering from the illness for several years. The satisfaction of seeing the joy on the faces of the patient and his family is unmatched, he said.

The Indian Express is now on Telegram. Click here to join our channel (@indianexpress) and stay updated with the latest headlines

For all the latest Bangalore News, download Indian Express App.

Link:
Three-year-old bone marrow donor, Bengaluru doctors give Iraqi boy a new lease of life - The Indian Express

Bone Marrow Stem Cells Comments Off on Three-year-old bone marrow donor, Bengaluru doctors give Iraqi boy a new lease of life – The Indian Express | September 14th, 2020

TOKYO--(BUSINESS WIRE)--SanBio Co., Ltd. (headquarters: Chuo-ku, Tokyo, Representative Director and President: Keita Mori, hereafter SanBio) hereby announces that it has obtained new analytical results from the Phase 2b clinical trial (the trial) of SB623 for the treatment of chronic motor deficit resulting from ischemic stroke the SanBio Group (SanBio Co., Ltd. and its subsidiary SanBio, Inc.) conducted in the US. It also announces that based on the newly obtained results, it has updated its development plans, including in regard to late-stage clinical trials for the ischemic stroke and hemorrhagic stroke programs of SB623 in Japan.

The trial evaluated efficacy and safety of SB623 in 163 patients suffering from chronic motor dysfunction from ischemic stroke. On January 29, 2019, SanBio announced that the trial did not meet its primary endpoint, as it failed to demonstrate statistical significance in the difference in the proportion of patients whose Fugl-Meyer Motor Scale (FMMS) score improved by 10 or more points from the baseline (primary endpoint) between the treatment group that received SB623 and the control group. Since then, the SanBio Group had continued to work on additional analysis of the trial data, and results of the additional analysis are as follows.

In conducting the additional analysis, from the perspective of minimal clinically important difference (MCID, or the minimal change in scores or other metrics that could be interpreted to mean the change in a patient is clinically meaningful) and based on the results of the Phase 2 clinical trial of SB623 for the treatment of chronic motor deficit from traumatic brain injury (TBI; STEMTRA trial), the company reevaluated trial data using composite FMMS. Of the total 163 patients enrolled in the trial, the company specifically looked at 77 patients who had infarct areas smaller than a certain size (47% of all patients enrolled in this trial). The SanBio Group evaluated the proportion of patients that met one or more of the following FMMS score improvement criteria 24 weeks after treatment: 6-point improvement on FMMS score for upper extremity, 4-point improvement on FMMS score for lower extremity, and 9-point improvement on FMMS total score (all from the baseline). Of the 51 patients in the treatment group that received SB623, improvement was seen in 49%, versus in 19% of 26 patients in the control group that received sham surgery, the difference between the two groups being statistically significant (p-value of 0.02). SanBio Group thinks that even compared to the primary endpointthe proportion of patients whose FMMS score improved by 10 or more points over the baseline six months after treatmentthe endpoint using composite FMMS can adequately explain clinical significance of the treatment efficacy. Details of the additional analysis results will be announced at the financial results briefing for institutional investors and the media held on September 15, 2020. The briefing video will be made available to the public on our website on the 16th of September or thereafter.

Based on the above results, the SanBio Group has begun preparations for the next late-stage clinical trials in the ischemic stroke and hemorrhagic stroke programs of SB623. 2021. Specific designs of the clinical trials and the contents of development for those two programs will be announced promptly upon being finalized. To maximize the value of SB623 at an early stage by selecting areas to focus the Groups management resources on, the SanBio Group plans to prioritize the development of the ischemic stroke and hemorrhagic stroke programs in Japan at the same time as it prepares to file for approval of SB623 for the treatment of chronic motor deficit resulting from TBI in Japan by the end of the current fiscal year (ending January 2021). The Group, however, postponed the global Phase 3 clinical trial for the TBI program of SB623 it had planned to commence this fiscal year to the next or subsequent fiscal years.

Many patients suffering from the chronic effects of ischemic stroke are said to be regularly taking drugs to prevent recurrence. However, because there is no drug that can fundamentally cure motor dysfunction, there is high unmet need for therapeutic drugs to restore motor functions for patients in the chronic phase of stroke. The SanBio Group aims to contribute to improving the lives of these patients, as well as of their family members, suffering from motor impairment and difficulties it causes in carrying out their daily lives through SB623.

About SB623

SB623 is an allogeneic mesenchymal stem cell produced by modifying and culturing bone marrow derived from healthy donors. Implantation of SB623 cells into nerve tissues is expected to promote regeneration of damaged nerve cells. Because SB623 is made from allogeneic cells, large-scale production is possible and there is no need for complex cell processing required for treatments using autologous cells, e.g., cell preparation for each patient at medical institutions. Hence, pharmaceutical products made from allogeneic cells, such as SB623, can be provided to many patients in uniform quality.

About SanBio Co., Ltd. and SanBio, Inc.

SanBio Group is engaged in the regenerative cell medicine business, spanning research, development, manufacture, and sales of regenerative cell medicines. The Companys propriety regenerative cell medicine product, SB623, is currently being investigated for the treatment of several conditions including chronic neurological motor deficit resulting from traumatic brain injury and ischemic stroke. The Company is headquartered in Tokyo, Japan and Mountain View, California, and additional information about SanBio Group is available at https://sanbio.com.

See more here:
Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit...

Bone Marrow Stem Cells Comments Off on Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit… | September 14th, 2020

BOSTON & LOUISVILLE, Ky.--(BUSINESS WIRE)--Talaris Therapeutics, Inc., a privately held biotechnology company developing transformative cell therapies that have the potential to induce durable immune tolerance across a range of indications, today announced a collaboration with Kentucky Organ Donor Affiliates (KODA). KODA is an independent, non-profit organ and tissue procurement organization that facilitates deceased donor transplants throughout Kentucky, southern Indiana and western West Virginia. The collaboration will advance preclinical studies of the potential of Talaris allogeneic cell therapy to induce immune tolerance to an organ from a deceased donor.

Organ donation from deceased donors makes possible more than 80 percent of solid organ transplants in the U.S. These transplants are frequently lifesaving, but they bring the burden of lifelong immunosuppression for organ recipients, which puts patients at heightened risk of infection as well as a number of other potentially serious side effects. Additionally, immunosuppressant drugs are toxic to the kidneys over time, which can result in declining kidney function and necessitate another organ transplant.

Talaris novel cell therapy, FCR001, has shown promising potential to eliminate the need for immunosuppression among recipients of kidney transplants from living donors. Our collaboration with KODA will be an important step toward potentially extending this promise to recipients of organ transplants from deceased donors as well, said Scott Requadt, Chief Executive Officer of Talaris.

Within the collaboration, KODA, after authorization from the donors family, will recover vertebrae from deceased organ donors. Researchers at Talaris will then isolate stem cells from the vertebrae, with the goal of demonstrating the feasibility of producing FCR001 from vertebral bone marrow. These preclinical studies will serve as a first step toward enabling future clinical studies to evaluate whether FCR001 administered alongside organ transplantation can induce durable immune tolerance to an organ from a deceased donor.

The generosity organ donors and their families display is measureless. By initiating this collaboration with Talaris, we hope to further honor their gift by advancing an important treatment for transplant recipients with the potential to improve many individuals lives, said Julie Bergin, RN, BSN, MHA, President & Chief Executive Officer of KODA.

About Talaris Therapeutics

Talaris Therapeutics, Inc. is a late-clinical stage biotechnology company that is developing transformative cell therapies with the potential to eliminate the burden of chronic immunosuppression for organ transplant recipients as well as induce durable remissions in patients with severe auto-immune and immune-mediated disorders. Talaris was founded on technology discovered and developed by Dr. Suzanne Ildstad and operates its own cell processing facility in Louisville, KY. Talaris is backed by leading life sciences investors Blackstone Life Sciences, Longitude Capital and Qiming Venture Partners USA and maintains corporate offices in Boston, MA and Louisville, KY. http://www.TalarisTx.com.

About Kentucky Organ Donor Affiliates (KODA)

Kentucky Organ Donor Affiliates is dedicated to saving lives through organ and tissue donation and transplantation. KODA is an independent, federally designated, non-profit organ and tissue procurement organization formed in 1987. KODA was recognized by Louisville Business First as the Nonprofit of the Year in 2011. KODAs mission is to provide organ and tissues to those in need and to maintain a profound respect for those who gave. KODA serves 114 counties in Kentucky, 4 counties in southern Indiana and 2 counties in western West Virginia. For more information visit donatelifeky.org or call 1-800-525-3456.

Read the rest here:
Talaris Therapeutics Announces Collaboration With Kentucky Organ Donor Affiliates to Advance Preclinical Study of Tolerance Induction to Organs From...

Bone Marrow Stem Cells Comments Off on Talaris Therapeutics Announces Collaboration With Kentucky Organ Donor Affiliates to Advance Preclinical Study of Tolerance Induction to Organs From… | September 14th, 2020

Rosh Hashanah is the day of pregnancy of the world. Hayom Harat Olam (The day of creation of the world). What does it mean?Medical research today is focusing on a fascinating and visionary field, which is the study of "stem cells."The uniqueness of stem cells comes from two main features which distinguish them from other cells. The first is that they are still 'neutral cells', meaning, they have not yet received their final identity and purpose in the body, so they have not yet finalized their differentiation process. As a result of this, they are able to differentiate and can develop into one of many types of mature cells, such as a skin cell, a muscle cell or a blood cell. By doing so, new cells are created, which can regenerate various tissues and organs.The second unique feature of stem cells is that they are able to divide and multiply infinitely and produce identical cells. Thus, a constant pool of unsorted stem cells that have not acquired a final designation is preserved.The possibilities of healing, cell regeneration and the human body in general are fascinating, and the field of stem cells is one of the leading fields in the world. Its healing potentials are so vast, not only when it comes to repairing the damaged, but also recreating something more true and precise.When I think of Rosh Hashanah from a spiritual standpoint, it feels like today is the birth of the world. We have before us 353 days (because some Jewish months arent full) which have not yet been defined by how they will play out and how they will look. It is unknown how we will succeed as individuals, and how we will succeed as nation. Interestingly, the second feature that exists with stem cells also connects to Rosh Hashanah. On this holy day, we are praying and asking Hashem from a place of "anything is possible" there are no boundaries, no glass barriers, no one is stopping us from simply praying and asking. cnxps.cmd.push(function () { cnxps({ playerId: '36af7c51-0caf-4741-9824-2c941fc6c17b' }).render('4c4d856e0e6f4e3d808bbc1715e132f6'); });Hashem, everything is completely in our hands! We can change the world and create something entirely new. Rosh Hashanah gives us the opportunity to create and build anew; its the source of rebirth for the relationship between man and his friend, man and Hashem, and of course, man as one who is part of a nation and of the entire world.It's not for nothing that it is written in the Talmud Yerushalmi that whoever spends his time on Rosh Hashanah sleeping, his luck will also sleep. It's the time to act, ask, beg and change.The cells will divide, the decisions made will be in place, and the abundance from shamayim (heaven) will come down. We now have a very great ability and opportunity to influence that. Please dont miss out on this opportunity!Be that as it may, today is Harat Olam the pregnancy of the world. It is our responsibility to pray that this pregnancy will be the best it can be. Have a happy and sweet New Year.Rabbi Yitzak Neriya is the head of the Torah Betzion yeshiva and founder of the Echad Lechad (One to One) foundation.

See the original post here:
Stem cells, like Rosh Hashanah, give the opportunity to be reborn - The Jerusalem Post

Skin Stem Cells Comments Off on Stem cells, like Rosh Hashanah, give the opportunity to be reborn – The Jerusalem Post | September 14th, 2020

Venus pokes Uranus, provoking your mother, sister or aunt to ruffle your feathers. Stand your ground and dont allow anyone to sway you from your purpose. Although well-intentioned, this person isnt fully informed, and most importantly,she isnt living your life. Theres no need for harsh words, just gently remind her thatyoure capable of making your own decisions. And thats that!

Although this week brings a breath of fresh air in the form of a new project or chapter, someone is intent on undermining you. Their doubts stem from jealousy, so dont allow them to project their negativity onto you. Stay your course, Pisces. Come up with your own facts rather than listening to biased opinions. Believe in yourself and toss the naysayers to the curb.

A self-confessed astrology nerd, Natashas horoscopes, research and articles have been published in Todays Astrologer, in addition to international publications across the globe. A senior member of the Australian Academy of Astrology and Cosmobiology and a member of the American Federation of Astrologers, she has presented cosmic updates for Your Life Naturally and has appeared as a special guest on podcasts, including Sivana and Healthy-ish.

Feature Image: Supplied.

Go here to see the original:
HOROSCOPES: How Thursday's new moon will affect your week, according to your star sign. - Mamamia

Skin Stem Cells Comments Off on HOROSCOPES: How Thursday’s new moon will affect your week, according to your star sign. – Mamamia | September 14th, 2020

Sherrie Hewson faced up to turning 70 this week by giving herself a real lift.

The Benidorm and Corrie star, who hits the milestone birthday on Thursday, has had a second facelift to boost her confidence after a really tough year that saw her beloved brother die and left her lonely in lockdown.

Sherrie says she was completely broken after her older sibling Brett Hutchinson passed away in April and she couldnt visit him because of coronavirus restrictions.

Brett died of a brain tumour and the shock of his death has left Sherrie more determined than ever to live life to the full.

She told the Sunday Mirror: I am still not very strong, I still have moments. Whenever I hear any Motown music, which he loved, I just cry. The pain is so terrible, my heart is broken.

Now I look at my life and think, Hang on a minute, what have I got, how long have I got?

I mean, look at my brother. Two years ago he was the healthiest thing on this planet.

So none of us know whats going to happen.

In ten years time I am going to be 80, so I need to be healthy and well for my grandchildren.

I wish I had planned more when I was younger. Instead I just went steaming ahead, thinking I was going to live forever.

Brett was only a couple of years older than me, so as I go into my seventies his death has made me want to live every day to the max.

I want to do everything now everything crazy, mad and wonderful. Everything that is possible to do I want to do it now.

And that all started with her facelift.

Dubbed the love handle facelift or LHF Sherries procedure took just minutes and she went out to lunch straight afterwards.

The procedure involved removing fat from around her hips and stomach. This was then combined with her blood, to release stem cells from the fat.

The mixture, a thick serum, was then injected into layers into her face without even drawing blood. The results will last five years.

Sherrie said: The LHF was a quick treatment and its given me such a boost. I feel like its rejuvenated my skin and restored all the volume its natural looking with no cutting, and it was all done in under an hour.

I absolutely love the results.

She adds: I had a facelift when I was 50 and Ive had bits and bobs done throughout the years a bit of botox and filler. But I havent had anything done for a long time now.

But as I approached 70. I just thought, What will I do for myself at this age? I thought I would give myself a last kind of boost so I can look in the mirror and think, Youre not bad for your age.

Its to give myself a lift after all the stuff I have been through, particularly in the last 18 months.

I need to give my self-confidence and self-worth a big kick up the a**e so can I feel good about myself and hold my head up high.

The actress , who lives alone, says she found lockdown awful and even turned to drinking wine every night to get through it.

She says: It was just terrifying being alone. Thank God for the internet! I have been doing the Wonderbirds show and Ive been presenting courses on Zoom. That kept my mind busy.

I did put on weight and I am trying to be more sensible now. I started drinking, and I was drinking every night which I would never do. Then I thought, I must stop this.

So I stopped for two months and I didnt even miss it. Thats when I realised I didnt have a problem. I was just bored and lonely.

Now Sherrie is hoping to spend the next decade healthy and happy and maybe even in love.

Her Corrie character Maureen Webster had four husbands, including Reg Holdsworth, played by Ken Morley. But Sherrie says she hasnt even had a proper date since her marriage to Ken Boyd broke down in 2001, after he had an affair.

Now she hopes her facelift will give her the boost she needs to start going out with people again.

She says: Dating is harder as you get older - the older you get, the more you lack confidence. I sometimes look in the mirror and dont know who that much older woman is.

Sherrie would be more than happy with a kiss and cuddle with someone with their arms around you.

She says: I cant imagine any more what that feels like that warmth and that someone who wants to be with me. Just that alone would be enough.

Sherrie, who played flirty hotel manager Joyce Temple-Savage in ITV sitcom Benidorm, says she has had a couple of potential love interests.

One was a younger American man she met on dating site Plenty of Fish. But they came to nothing.

She says: I havent really dated since I broke up with my husband except I did go for a meal with a man and I ordered a bottle of wine. He put the cork in the bottle and said he was driving and I said, Im bloody not! and took the cork out.

We got back to my house and he tried kissing me and I just thought, No!

But passion is still on Sherries wishlist as shes still open to an unbelievable affair with the right man.

She also has dreams of launching a travel programme for older people showing that they can still do crazy, mad and wonderful things.

Because coronavirus restrictions are set to change again tomorrow, with the new Rule of Six, Sherrie wont be able to celebrate her birthday as she had hoped with a big bash. But she will spend the day with daughter Keeley and youngest grand-daughter Rosie, who turned one in May.

Reflecting on her own age, the gran-of-three says: I have been on this earth for 70 years. Doesnt that sound weird?

The thing is how the hell did I get to number seven? When I got to number six I was gobsmacked. But you know what? A lot of people dont get to number seven, so how lucky am I?

Ive got a wonderful family and Ive had a fabulous career. My family are my life blood, I breathe for them.

And Im healthy, thank God. When you moan and groan about getting older and I do, all the time I tell myself my great grandmother, grandmother and my mum all lived until their eighties and nineties. So I am on a good wicket there arent I?

Sherrie admits she sometimes envies her daughters family life.

She says: Ive been on stage since I was four and, when I look back, my whole life has been my career.

I have no regrets, except that I would have had more children, given the chance. Keeley is at home with her three and I never had the privilege to do that. I was working so much.

But instead of dwelling on the past, Sherrie is determined to look ahead from now on. She says: At 70, I am still finding out who I am and what I want to do with my life.

Fat Transfer and Stromal Vascular fraction

We are increasingly using fat along with concentrated platelet rich plasma as a natural lipo filler to add volume where there has been for fat loss and to help with skin rejuvenation.

Fat is the bodys major source of mesenchymal stem cells, which are considered multi-potent, as they have the ability to differentiate themselves into a number of different types of stem cells that are useful for regeneration, tissue repair and wound healing. PRP is used to promote cell growth and collagen production, so is extremely useful for Creping and thinning skin.

Combined they make the perfect treatment to regenerate and rejuvenate and replace lost volume due to ageing.

What is it? An all-natural alternative incision free facelift

Downtime? 0-24 hours

How long does it last? Up to five years

Where can I get it? http://www.harleystreetskinclinic.com 0207 436 4441

How much is it? FROM 3,950

Follow this link:
Sherrie Hewson turns 70 with second facelift after tough year left her 'broken' - Mirror Online

Skin Stem Cells Comments Off on Sherrie Hewson turns 70 with second facelift after tough year left her ‘broken’ – Mirror Online | September 14th, 2020

Sept. 14, 2020 12:00 UTC

BOSTON & BORDENTOWN, N.J.--(BUSINESS WIRE)-- Alexion Pharmaceuticals Inc.. (NASDAQ:ALXN) and Caelum Biosciences, Inc. today announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis. The CARES clinical program includes two parallel Phase 3 studies one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200914005234/en/

In AL amyloidosis, misfolded amyloid proteins can build up in many organs throughout the body, including the heart and kidneys, causing significant damage to these organs and impairing their function. While current treatments address the bone marrow disorder that creates the misfolded amyloid proteins, there are no approved therapies for the significant organ damage the disease causes, said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from these organs. Data from Phase 1 studies suggest that this treatment approach may improve organ function and long-term survival. We look forward to investigating this further in the Phase 3 clinical program.

AL amyloidosis is particularly devastating when it affects the heart, with median survival in these patients of less than one year following diagnosis, said Michael Spector, President and Chief Executive Officer of Caelum. Long-term survival data from AL amyloidosis patients treated with CAEL-101 in the Phase 1a/1b study showed that 78 percent were still alive after a median follow-up time of more than three years. We recognize the urgent need for new treatments that address the organ damage caused by AL amyloidosis and are working together with the AL amyloidosis community and Alexion to advance the Phase 3 clinical program as quickly as possible.

About the CARES Phase 3 Clinical Program

The CARES clinical program consists of two parallel double-blind, randomized, event-driven global Phase 3 studies, which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and nave to standard of care (SoC) treatment (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy). One study is enrolling approximately 260 patients with Mayo stage IIIa disease and one study is enrolling approximately 110 patients with Mayo stage IIIb disease. The studies will be conducted at approximately 70 sites across North America, the United Kingdom, Europe, Israel, Japan, and Australia.

In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks.

The primary study objectives are overall survival and the safety and tolerability of CAEL-101. Key secondary objectives will assess functional improvement in the six-minute walk test (6MWT), quality of life measures (Kansas City Cardiomyopathy Questionnaire Overall Score & Short Form 36 version 2 Physical Component Score) and cardiac improvement (Global Longitudinal Strain, or GLS).

Phase 2 Study Results

The Phase 2 open-label dose escalation study was conducted to investigate higher doses of CAEL-101 than had been evaluated in Phase 1 studies with a primary objective to identify the best dose to advance into Phase 3 development. The study evaluated the safety and tolerability of CAEL-101 in 13 AL amyloidosis patients at three study sites who received up to 1000 mg/m2 of CAEL-101 (two times the Phase 1 dose) administered in combination with SoC treatment. The study met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the Phase 3 study.

Phase 1a/1b Long-Term Follow-Up Results Presented at ISA 2020

As previously reported, the Phase 1a/1b study of CAEL-101 was the first clinical trial to demonstrate improvement in cardiac function via GLS after treatment with an amyloid fibril targeted therapy in AL amyloidosis patients with amyloid cardiac involvement. New long-term follow-up data from the Phase 1a/1b study will be presented at the virtual International Symposium on Amyloidosis (ISA), September 14 to 18, 2020, in the poster titled, Long term follow-up of patients with AL amyloidosis treated on a phase 1 study of Anti-Amyloid Monoclonal Antibody CAEL-101 (Abstract #342, Divaya Bhutani, M.D., et. al, Columbia University Medical Center). These data demonstrate 78 percent survival (15/19) at a median follow-up of more than three years (37 months) in AL amyloidosis patients treated with CAEL-101 as well as durable organ response among evaluable patients, further supporting the advancement of CAEL-101 into Phase 3 development.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: http://www.alexion.com.

[ALXN-P]

About Caelum Biosciences

Caelum Biosciences, Inc. (Caelum) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelums lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (AL) amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.caelumbio.com.

Forward-Looking Statement

This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion and Caelum, including statements related to: the safety and efficacy CAEL-101 as a treatment for AL amyloidosis; CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from the heart, kidney and other organs; data from the Phase 1 studies suggest that the treatment approach may improve organ function and long-term survival and enrollment of the Phase 3 trials. Forward-looking statements are subject to factors that may cause Alexion's and Caelums results and plans to differ materially from those expected by these forward looking statements, including for example: the anticipated safety profile and the benefits of the CAEL-101 may not be realized (and the results of the clinical trials may not be indicative of future results); the inability to enroll and complete the Phase 3 trial; results of clinical trials may not be sufficient to satisfy regulatory authorities; results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexions or Caelums business, including on commercial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us; the risk that third party payors (including governmental agencies) will not reimburse for the use of our products at acceptable rates or at all; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including COVID-19 and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended June 30, 2020 and in their other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200914005234/en/

See the original post here:
Alexion and Caelum Biosciences Announce Start of Phase 3 Studies of CAEL-101 in AL Amyloidosis - BioSpace

Cardiac Stem Cells Comments Off on Alexion and Caelum Biosciences Announce Start of Phase 3 Studies of CAEL-101 in AL Amyloidosis – BioSpace | September 14th, 2020

Sept. 14, 2020 10:45 UTC

BOTHELL, Wash. & KENILWORTH, N.J.--(BUSINESS WIRE)-- Seattle Genetics, Inc. (Nasdaq: SGEN) and Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced two new strategic oncology collaborations.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200914005237/en/

The companies will globally develop and commercialize Seattle Genetics ladiratuzumab vedotin, an investigational antibody-drug conjugate (ADC) targeting LIV-1, which is currently in phase 2 clinical trials for breast cancer and other solid tumors. The collaboration will pursue a broad joint development program evaluating ladiratuzumab vedotin as monotherapy and in combination with Mercks anti-PD-1 therapy KEYTRUDA (pembrolizumab) in triple-negative breast cancer, hormone receptor-positive breast cancer and other LIV-1-expressing solid tumors. Under the terms of the agreement, Seattle Genetics will receive a $600 million upfront payment and Merck will make a $1.0 billion equity investment in 5.0 million shares of Seattle Genetics common stock at a price of $200 per share. In addition, Seattle Genetics is eligible for progress-dependent milestone payments of up to $2.6 billion.

Separately, Seattle Genetics has granted Merck an exclusive license to commercialize TUKYSA (tucatinib), a small molecule tyrosine kinase inhibitor, for the treatment of HER2-positive cancers, in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics will receive $125 million from Merck as an upfront payment and is eligible for progress-dependent milestones of up to $65 million.

Collaborating with Merck on ladiratuzumab vedotin will allow us to accelerate and broaden its development program in breast cancer and other solid tumors, including in combination with Mercks KEYTRUDA, while also positioning us to leverage our U.S. and European commercial operations, said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. The strategic collaboration for TUKYSA will help us reach more patients globally and benefit from the established commercial strength of one of the worlds premier pharmaceutical companies.

These two strategic collaborations will enable us to further diversify Mercks broad oncology portfolio and pipeline, and to continue our efforts to extend and improve the lives of as many patients with cancer as possible, said Dr. Roger M. Perlmutter, President, Merck Research Laboratories. We look forward to working with the team at Seattle Genetics to advance the clinical program for ladiratuzumab vedotin, which has shown compelling signals of efficacy in early studies, and to bring TUKYSA to even more patients with cancer around the world.

Ladiratuzumab Vedotin Collaboration Details

Under the terms of the agreement, Seattle Genetics and Merck will collaborate and equally share costs on the global development of ladiratuzumab vedotin and other LIV-1-targeting ADCs. The companies have agreed to jointly develop and share future costs and profits for ladiratuzumab vedotin on a 50:50 basis worldwide. Merck will pay Seattle Genetics $600 million upfront and make a $1.0 billion equity investment in 5.0 million shares of Seattle Genetics common stock at a price of $200 per share. In addition, Seattle Genetics will be eligible to receive up to $2.6 billion in milestone payments, including $850 million in development milestones and $1.75 billion in sales milestones.

The companies will jointly develop and commercialize ladiratuzumab vedotin and equally share profits worldwide. The companies will co-commercialize in the U.S. and Europe. Seattle Genetics will be responsible for marketing applications for approval in the U.S. and Canada, and will record sales in the U.S., Canada and Europe. Merck will be responsible for marketing applications for approval in Europe and in countries outside the U.S. and Canada, and will record sales in countries outside the U.S., Europe and Canada. Including the upfront payment, equity investment proceeds and potential milestone payments, Seattle Genetics is eligible to receive up to $4.2 billion.

The closing of the equity investment is contingent on completion of review under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act).

TUKYSA Collaboration Details

Under the terms of the agreement, Merck has been granted exclusive rights to commercialize TUKYSA in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics retains commercial rights and will record sales in the U.S., Canada and Europe. Merck will be responsible for marketing applications for approval in its territory, supported by the positive results from the HER2CLIMB clinical trial.

Merck will also co-fund a portion of the TUKYSA global development plan, which encompasses several ongoing and planned trials across HER2-positive cancers, including breast, colorectal, gastric and other cancers set forth in a global product development plan. Seattle Genetics will continue to lead ongoing TUKYSA global development planning and operational execution. Merck will solely fund and conduct country-specific clinical trials necessary to support anticipated regulatory applications in its territory.

Seattle Genetics will receive from Merck $125 million as an upfront payment and is eligible to receive progress-dependent milestones of up to $65 million. Seattle Genetics will also receive $85 million in prepaid research and development payments to be applied to Mercks global development funding obligations. In addition, Seattle Genetics would receive tiered royalties on sales of TUKYSA in Mercks territory.

The financial impact of these collaborations is not included in Seattle Genetics 2020 guidance.

Seattle Genetics Conference Call Details

Seattle Genetics management will host a conference call to discuss these collaborations today at 6:00 a.m. Pacific Time (PT); 9:00 a.m. Eastern Time (ET). The event will be simultaneously webcast and available for replay from the Seattle Genetics website at http://www.seattlegenetics.com, under the Investors section. Investors may also participate in the conference call by calling 844-763-8274 (domestic) or +1 412-717-9224 (international). The conference ID is 10147850.

About Ladiratuzumab Vedotin

Ladiratuzumab vedotin is a novel investigational ADC targeted to LIV-1. Most metastatic breast cancers express LIV-1, which also has been detected in several other cancers, including lung, head and neck, esophageal and gastric. Ladiratuzumab vedotin utilizes Seattle Genetics proprietary ADC technology and consists of a LIV-1-targeted monoclonal antibody linked to a potent microtubule-disrupting agent, monomethyl auristatin E (MMAE) by a protease-cleavable linker. This novel ADC is designed to bind to LIV-1 on cancer cells and release the cell-killing agent into target cells upon internalization. Ladiratuzumab vedotin may also cause antitumor activity through other mechanisms, including activation of an immune response by induction of immunogenic cell death.

About TUKYSA (tucatinib)

TUKYSA is an oral, small molecule tyrosine kinase inhibitor (TKI) of HER2, a protein that contributes to cancer cell growth. TUKYSA in combination with trastuzumab and capecitabine was approved by the U.S. Food and Drug Administration (FDA) in April 2020 for adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting. In addition, TUKYSA received approval in Canada, Singapore, Australia and Switzerland under the Project Orbis initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners. A marketing application is under review in the European Union.

TUKYSA is being evaluated in several ongoing clinical trials and additional studies are planned. Current trials include the following:

For additional information, visit http://www.clinicaltrials.gov.

TUKYSA Important Safety Information

Warnings and Precautions

If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Based on the severity of the diarrhea, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Monitor ALT, AST, and bilirubin prior to starting TUKYSA, every 3 weeks during treatment, and as clinically indicated. Based on the severity of hepatoxicity, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Adverse Reactions

Serious adverse reactions occurred in 26% of patients who received TUKYSA. Serious adverse reactions in 2% of patients who received TUKYSA were diarrhea (4%), vomiting (2.5%), nausea (2%), abdominal pain (2%), and seizure (2%). Fatal adverse reactions occurred in 2% of patients who received TUKYSA including sudden death, sepsis, dehydration, and cardiogenic shock.

Adverse reactions led to treatment discontinuation in 6% of patients who received TUKYSA; those occurring in 1% of patients were hepatotoxicity (1.5%) and diarrhea (1%). Adverse reactions led to dose reduction in 21% of patients who received TUKYSA; those occurring in 2% of patients were hepatotoxicity (8%) and diarrhea (6%).

The most common adverse reactions in patients who received TUKYSA (20%) were diarrhea, palmar-plantar erythrodysesthesia, nausea, fatigue, hepatotoxicity, vomiting, stomatitis, decreased appetite, abdominal pain, headache, anemia, and rash.

Lab Abnormalities

In HER2CLIMB, Grade 3 laboratory abnormalities reported in 5% of patients who received TUKYSA were: decreased phosphate, increased ALT, decreased potassium, and increased AST. The mean increase in serum creatinine was 32% within the first 21 days of treatment with TUKYSA. The serum creatinine increases persisted throughout treatment and were reversible upon treatment completion. Consider alternative markers of renal function if persistent elevations in serum creatinine are observed.

Drug Interactions

Use in Specific Populations

For more information, please see the full Prescribing Information for TUKYSA here.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

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 1,200 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 patient's 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 1 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.

KEYTRUDA is indicated for the treatment of 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 are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient 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.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

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).

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. 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 TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

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 adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). 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 adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or 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.

Read more:
Seattle Genetics and Merck Announce Two Strategic Oncology Collaborations - BioSpace

Cardiac Stem Cells Comments Off on Seattle Genetics and Merck Announce Two Strategic Oncology Collaborations – BioSpace | September 14th, 2020

Global Exosome Therapeutic Market By Type (Natural Exosomes, Hybrid Exosomes), Source (Dendritic Cells, Mesenchymal Stem Cells, Blood, Milk, Body Fluids, Saliva, Urine Others), Therapy (Immunotherapy, Gene Therapy, Chemotherapy), Transporting Capacity (Bio Macromolecules, Small Molecules), Application (Oncology, Neurology, Metabolic Disorders, Cardiac Disorders, Blood Disorders, Inflammatory Disorders, Gynecology Disorders, Organ Transplantation, Others), Route of administration (Oral, Parenteral), End User (Hospitals, Diagnostic Centers, Research & Academic Institutes), Geography (North America, Europe, Asia-Pacific and Latin America)

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

To Remain Ahead Of Your Competitors, Request for a FREE Sample Here (with covid 19 Impact Analysis) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-exosome-therapeutic-market&DW

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for an Analyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

Make an Enquiry before Buying @ https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-exosome-therapeutic-market&DW

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process.

The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

The major players covered in the report are evox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global. Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Read More@ https://www.databridgemarketresearch.com/reports/global-exosome-therapeutic-market?DW

About Data Bridge Market Research:

An absolute way to forecast what future holds is to comprehend the trend today!

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

Contact:

Data Bridge Market ResearchUS: +1 888 387 2818UK: +44 208 089 1725Hong Kong: +852 8192 7475Email @ Corporatesales@databridgemarketresearch.com

Original post:
Exosome Therapeutic Market (Covid 19 Impact Analysis) Data Highlighting Major Vendors, Promising Regions, Anticipated Growth Forecast To 2027 - Good...

Cardiac Stem Cells Comments Off on Exosome Therapeutic Market (Covid 19 Impact Analysis) Data Highlighting Major Vendors, Promising Regions, Anticipated Growth Forecast To 2027 – Good… | September 14th, 2020

If someone were to stop me in the street and ask me to give a limb to save a life, I wouldnt hesitate, says Rosenberg passionately. What good is a hand when it comes to an entire life?

By Mendel Super Chabad.org

Rabbi Yigal Rosenberga well-loved community rabbi in California and co-director ofChabad of Santa Clarawith his wife, Elanaisnt new to saving lives. From domestic violence to drug addiction, the rabbi doesnt shy away from involvement in lifesaving activities, but instead finds himself propelled towards them. But seldom does one get the opportunity to directly save a life in a very physical way.

In mid-February, before the emerging pandemic would dominate the headlines, as well as the lives of him and his community members, Rosenberg hosted a cooking demonstration at a local Lexus dealership featuring local gourmet chefTanyaSolomon, coupled with a bone marrow donor swabbing drive on behalf of the Gift of Life registryone of two private U.S. stem-cell registries.

Donor turnout was high, with virtually all attendees getting swabbed, a brief process in which a long cotton swab collects cells from the inside of the cheek, where they are then shipped off to a lab to determine their human leukocyte antigens, or HLA markers. Once the tissue type is determined, potential donors are added to a worldwide registry, which is made available to transplant centers and physicians.

Since only 30 percent of patients find a match within their immediate family, the majority need to search the registries. As bone marrow matches are very much dependent on the donor and recipient sharing specific genetic markers, the next best shot after family is ones ethnic group. While Gift of Life services the general community, many of their matches are betweenAshkenazicJews due to the relatively small size of their gene pool.

Days after the swabbing drive, Rosenberg received a call from Gift of Life officials. Assuming that they were calling about his recent donor drive, he was stunned when they told him he was a match. Your stem cells can save the life of a 40-year-old man with leukemia, they said. Taken aback, Rosenberg informed them that while he was happy to help, among all the hustle and bustle of the event he hadnt actually had a chance to join the registry and be swabbed. They told me I was on the registry from a drive at the Rabbinical College of America in Morristown, N.J., more than 10 years before! hetold Chabad.org.

The rabbi doesnt see this as mere coincidence. When we help others,Gdhelps us, he reflects. What greater opportunity can one have than to save the life of another? Its the embodiment of ourshlichusto help our fellow in every way possible.

If someone were to stop me in the street and ask me to give a limb to save a life, I wouldnt hesitate, says Rosenberg passionately. What good is a hand when it comes to an entire life? And giving bone marrow was far easier.

To give the life-giving serum, Rosenberg would have to drive to Los Angeles for several days to LifeStream Blood Bank in San Bernardino, a transplant center nearly eight hours away by car. Gift of Life arranged all the accommodation, but nobody could replace the rabbi at home. We have three children of our own, the rabbi explains, and now, because schools are closed for in-person learning, were accommodating local Jewish kids atChabadfor their virtual school. Fourteen children spend eight hours a day at theChabad center, keeping up with their general virtual studies in a safe environment. During breaks, they enjoy an array of in-person Jewish educational activities. We just had ashofarfactory and made thank you cards for the firefighters battling Californias wildfires, shares Rosenberg.

Before traveling to Los Angeles, the rabbi had to be conditioned for the transplant. He received several shots of Neupogen dailyeven onShabbat, after consulting a senior rabbito stimulate his bone marrow into producing extra stem cells.

At the transplant center in Los Angeles, Rosenberg was hooked up to a machine for eight hours, extracting the stem cells from his blood. All my blood left my body three times during the procedure, he says, noting that it was mostly painless.

The precious stem cells were immediately hand-delivered by special courier to the patient in N.J., whose identity is legally withheld from the donor for five years.

Rosenberg, who is also a chaplain with the Santa Clara police department, received well-wishes and support from many in the broader community, including the mayor, deputy mayor and various faith leaders he works with.

Left to hold down the fort in Santa Clara for three days, Elana Rosenberg was very supportive of her husbands decision. I felt grateful for the opportunity that my husband had to be able to help someone in a way that no one else could, she says, noting that out of 7 billion people in the world, only he was a match for that young man; Im just praying that the recipient of the donation takes it well and can continue to lead a life of purpose in good health.

(Reprinted with permission from Chabad.org)

View post:
Bone Marrow Donor Drive Saves a Life in an Unexpected Way - Anash.org - Good News

Bone Marrow Stem Cells Comments Off on Bone Marrow Donor Drive Saves a Life in an Unexpected Way – Anash.org – Good News | September 12th, 2020