Using 3D printers, researchers have collaborated from around the globe to develop nanoclay-based 3D bioprinted scaffolds which could be used to aid skeletal regeneration.

Hailing from the University of Southampton, the Istituto Italiano di Tecnologia in Rome, University Hospital Carl Gustav Carus and Technische Universitt in Dresden, and China Medical University in Taiwan, the researchers 3D bioprinted implantable nanocomposite scaffolds, laden with human bone marrow stromal cells (HBMSCs) and human umbilical vein endothelial cells (HUVECs), which have the potential to facilitate bone formation.

3D bioprinting for orthopedics

Ideally, 3D printed implants of this nature should sustain cell viability and promote cells to multiply, in addition to generating functional constructs shortly after printing and stimulating the host microenvironment to aid tissue growth.

Realistically, bioprinting is in its infancy and the prospect of whole 3D printed transplant organs is still probably decades away. However, there have been a number of innovative developments in the field thus far, such asa novel bio-ink enabling scientists at the University of Minnesota to create a functional 3D printed beating human heart.

Similarly, researchers fromTsinghua Universityhave3D bioprinted brain-like tissue structures capable of nurturing neural cells, while in May microdispensing specialistnScryptand aerospace companyTechShotsuccessfully completed the first functional 3D bioprinting experiment in space a human knee meniscus.

Most recently, researchers from theUniversity of Montrealhave developed a new method of cell bioprinting based on a drop-on-demand technique, called Laser Induced Side Transfer, which utilizes a low energy nanosecond laser and the laws of microfluidic dynamics to jet living cells onto each other. The team believes their work could be adapted for applications such as 3D drug screening models and artificial tissues.

The nanoclay-based method

According to the report,nanoclay-based bioink formulations are particularly attractive for implant applications given their ability, even at low concentrations, to shear while being extruded and regain their shape upon deposition, while shielding cells from potential damage from the printing process.

During the study, scientists harnessed the physiochemical properties of Laponite (LAP), a smectite nanoclay suspension, and combined it with HBMSCs, bone morphogenic protein-2 (BMP-2), and vascular endothelial growth factor to produce LAP-alginate-methylcellulose bioink. HBMSCs, collected from patients undergoing routine hip surgery, and HUVECs, obtained from the umbilical cords of healthy mothers after normal, full-term deliveries, were encapsulated in the bioink and printed using an in-house built bioprinter.

After printing, the scaffolds were incubated for 10 minutes in a sterile calcium chloride solution to enable crosslinking.

The skeletal functionality of the HBMSCs-laden 3D bioprinted scaffolds was investigated in vitro, ex vivo, and in vivo. The results demonstrated significant improvements in mineralized tissue formation with the addition of HBMSCs in 3DP, but not in mold-cast bulk scaffolds.

Significance of the findings

According to the researchers, the printing of bioinks laden with cells that can act as building blocks for the generation of tissue-like structures represents a simple and effective approach to produce readily implantable constructs.

The potential to print stem cells, preserving cell viability, proliferation, and functionality, is currently a key unmet challenge for the biofabrication approach to regenerative medicine. Clay-based bioinks, such as the one looked at in this study, are now proven to offer an attractive vehicle for printing HBMSCs in three-dimensional constructs due to their shear-thinning and inherent functional properties.

Further details of the study can be found in the article titledNanoclay-based 3D printed scaffolds promote vascular ingrowth ex vivo and generate bone mineral tissue in vitro and in vivo, published in the Biofabrication journal. The article is co-authored by Gianluca Cidonio, Michael Glinka, Yang-Hee Kim, Janos Kanczler, Stuart Lanham, Tilman Ahlfeld, Anja Lode, Jonathan Dawson, Michael Gelinsky, and Richard Oreffo.

The 4th annual 3D Printing Industry Awards are coming up in November 2020 and we need a trophy. To be in with a chance of winning a brand newCraftbot Flow IDEX XL3D printer,enter the MyMiniFactory trophy design competition here. Were happy to accept submissions until the 30th of September 2020.

Subscribe to the3D Printing Industry newsletterfor the latest news in additive manufacturing. You can also stay connected by following us onTwitterand liking us onFacebook.

Looking for a career in additive manufacturing? Visit3D Printing Jobsfor a selection of roles in the industry.

Featured image shows functional investigation of 3D printed scaffold vascularisation in a CAM model. Image via Biofabrication journal.

Read this article:
Researchers develop nanoclay-based bioprinting method to produce functional bone implants - 3D Printing Industry

Bone Marrow Stem Cells Comments Off on Researchers develop nanoclay-based bioprinting method to produce functional bone implants – 3D Printing Industry | September 18th, 2020

After Coach Paul Trosclair won a Louisiana high school football championship in December 2018, he and his family walked from the Superdome in New Orleans to celebrate with a bowl of late-night gumbo. He mused about retiring, but no one took him seriously.

For five seasons, Trosclair had endured fatigue and other effects of multiple myeloma, a treatable but incurable blood cancer, missing only a single game. He coached from a golf cart when the burning sensation in his feet made it too painful to stand. And when he was sidelined that one Friday night after a blood clot required surgery, he phoned his players from his hospital bed to wish them luck.

With a state title at Eunice High School, after runner-up finishes there in 1997 and 1998, Trosclair had reached the pinnacle of a long, successful career. He was one of Louisianas winningest coaches. Back home on the Cajun prairie, he rode in a convertible during the towns victory parade, holding the championship trophy. He had nothing left to prove, but he stayed on for the 2019 season, elevating his career record to 247 victories even as medication left him with muscle cramps so severe at times that his fork fell from his fingers.

I couldnt pull the trigger, Trosclair, 64, said in a telephone interview. Its hard to walk away.

Now, he feels compelled, becoming one of a number of older coaches across the country who are choosing to retire rather than risk their health in the coronavirus pandemic.

In June, he gave his retirement notice after 40 years of coaching, the last 25 years at Eunice High. His cancer was in remission but his immune system was compromised. He did not think he could remain safe when a new school year and a new football season began. Not in a locker room where his players dress shoulder to shoulder. Not in the weight room. Not in crowded school hallways.

My doctors thought it was in my best interest not to coach, Trosclair said. I was on the edge; the coronavirus got me to jump over.

While young athletes are considered less vulnerable to Covid-19, the disease caused by the coronavirus, aging coaches are at higher risk of infection and having a severe response. At least 30 high school and club team coaches have died of coronavirus-related causes, according to a search of online obituaries. Though some were in their 70s, one was 27, another 30.

Countless other coaches have been forced to reconsider whether it is worth risking their health to continue their careers.

It remains unclear how many coaches have retired for reasons related to Covid-19. The N.C.A.A., the National Federation of State High School Associations, state athletic associations and coaching organizations said they have not kept such figures.

But a number of states have reported an uptick in teacher retirements, even if it is uncertain how many are related to the coronavirus. Louisiana, for instance, reported 335 retirements in August compared with 196 that month in 2019. In Ohio, the retirement rate more than doubled from July 1 through mid-August, compared to that period a year ago.

More so than in previous years, we are hearing about coaching staff retirees, said Jennifer Mann, a data technician with the Clell Wade Coaches Directory, a well-regarded national networking tool for coaches that tracks collegiate, high school and junior high school sports.

Even so, they may represent a fraction of coaches, though their departures often are deeply felt in their communities.

There are hundreds of thousands of high school coaches across the country in various sports, so even if there are hundreds who have retired, it is a pretty small number, Bruce Howard, a spokesman for the national high school federation, said in an email.

Some coaches who walked away said the pandemic had led them into deep introspection about their safety and their lifes direction.

Norm Ogilvie, 60, Duke Universitys longtime track and field coach, said in a statement that he felt there needs to be a final meaningful chapter for the remaining years I have on our rapidly changing planet.

Mike Fox, 64, retired after 22 years and seven trips to the College World Series as the baseball coach at the University of North Carolina. The coronavirus, he told the school, made him realize it is time for me to be a full-time husband, father and grandfather and do other things with my life.

Updated Sept. 18, 2020

Heres whats happening as the world of sports slowly comes back to life:

Joe Bustos, 57, who won two Arizona state basketball championships in 23 seasons coaching at North High School in Phoenix, stepped down, expressing frustration with virtual teaching and concern after two Arizona teachers died over the summer of Covid-19, including a 61-year-old high school swimming coach.

Im just afraid; I dont want to be playing Russian roulette, Bustos said in an interview. I love coaching and teaching, but at the end of the day youve got to look out for yourself.

Peter Kingsley, 54, taught middle school for nearly three decades in Boulder, Colo., and coached football, basketball, wrestling and track. But he has epilepsy and a circulatory condition that leaves him predisposed to strokes. His wife urged him to retire because of the pandemic. And he was influenced by spending 22 days in hospice with his father, who died this summer of bone cancer.

I had a choice to make whether to potentially die or keep coaching and teaching, Kingsley said in a telephone interview. I just needed to stay safe.

Trosclairs decision to leave coaching in Louisiana came reluctantly, after battling a cancer that he had never heard of until he learned he had it.

In spring 2014, he began to experience dizzy spells and fatigue. His blood pressure rose and his kidneys began to fail. The diagnosis was multiple myeloma, which begins in the bone marrow and limits the bodys ability to fight off infections, weakens bones, reduces kidney function and lowers a persons red blood cell count.

Trosclair began chemotherapy and taking a corticosteroid called Decadron, which left him intensely focused, insatiably hungry and agitated from extreme insomnia followed by bouts of crashing. He recalls his oncologist at the M.D. Anderson Cancer Center in Houston saying that he might lose his job in such a severe state. He jokingly replied, They already think Im crazy, so theyll give me a pass.

He asked one of his assistant coaches to remain vigilant in case his temper flared. Some days he felt 20 years old, he told a Louisiana reporter. Other days he felt 100. Still, Trosclair coached every game in the 2014 football season. In early 2015, he underwent a stem cell transplant. His own blood-making stem cells were harvested, frozen, then reintroduced after chemotherapy to produce new, healthy blood cells. He spent six weeks in Houston for the treatment and recovery.

People in Eunice raised some money and it was a big help, Trosclair said.

Months later, though, a mix-up over blood thinners during the 2015 football season led to a blood clot in his left leg and forced him into intensive care at a hospital in Lafayette, La. His left foot swelled to three times its normal size. Three surgeries were required, causing him to miss his only game in 25 seasons. Trosclair spoke to his team beforehand by phone, saying, I love you. Go out and play.

Irma Trosclair, his wife and the superintendent of schools in Lafayette Parish, one of Louisianas largest school districts, still keeps a video of the bedside pep talk.

When I saw him doing that, with all those tubes he had going, I knew that coaching wasnt just work, she said. It was what was going to pull him through.

In 2018, Eunice High unexpectedly reached the Class 3A state championship game and prevailed, 59-47, with Trosclairs Wing-T offense, an intricate symphony of misdirection and strategic passing. After a quarter century at the school and five seasons of fighting cancer, he claimed his biggest football victory. Trosclair told a television interviewer, It was like the universe opened its doors and said, here you go, heres a gift for you.

The high school and its football team confirm that Eunice still measures up, even as its population and student enrollment continue to shrink and a third of its 9,800 residents live in poverty. It is the only traditional public high school in St. Landry Parish to carry an A-rating of academic performance from the state and has maintained its diversity a half century after desegregation.

When you think of Eunice High, you think of Coach Trosclair, said the principal, Mitch Fontenot. Everybody looks up to him. He has a real calming effect. Its a big loss.

Sixteen starters were to return for the 2020 season. Another deep playoff run seemed possible. But the coronavirus shuttered Louisiana schools in March and the state became a hot spot. Trosclair no longer felt he could protect himself and manage his team safely at the same time. Retirement began to seem inevitable.

On June 18, Trosclair saw Dr. Donna M. Weber, his oncologist at M.D. Anderson, who wrote in a letter that he was at particular risk of infection during the pandemic and that she advised him not to return to work.

Irma Trosclair said, He needed his doctor to tell him he absolutely had no other option. I think hes very much at peace with it.

There has been sobering validation of Trosclairs decision. The athletic director of an area high school also retired with multiple myeloma. The father of one of Trosclairs former players died of Covid-19. Trosclairs replacement at Eunice High, interim coach Andre Vige, 41, tested positive, along with two Eunice High players, one of whom was hospitalized. All have recovered. Two teenage brothers in the area, the youngest a football player, also contracted the virus. The elder brother died at age 19.

Its possible for young people to die, Trosclair said. Thats the scary thing.

He spent the summer playing golf, taking 6 a.m. walks around the Eunice High track and lifting weights at the school when no one was around. He takes Revlimid, a maintenance medication, three weeks of every four. And while fatigue and muscle spasms persist, acupuncture has helped relieve the burning feeling in his feet. His red blood cell count and other markers are encouraging. Still, he has avoided large gatherings.

When the Eunice city council honored him, his wife went in his place. His grandchildren have not visited since March. When his youngest son, Trenon, 26, got married in June, Trosclair sat in an isolated section of the church, then left through a side door and skipped the reception.

Im really sad right now, he texted his wife.

Louisianas delayed high school football season is set to begin on Oct. 1. Trosclair would like to remain involved with the team in some manner. He has studied plays at the dining room table with Trenon, the teams secondary coach. Perhaps he will help with game planning. He would like to attend games, if he can stand away from everyone, but his wife is skeptical. She has another idea.

Hopefully theyll let me keep my same parking spot, Irma Trosclair said. Then we can watch the whole game from my vehicle. Surely theyll grant that for Coach.

Gillian Brassil contributed reporting. Sheelagh McNeill and Susan Beachy contributed research.

Read the original post:
The Pandemic Is Chasing Aging Coaches From the Field - The New York Times

Bone Marrow Stem Cells Comments Off on The Pandemic Is Chasing Aging Coaches From the Field – The New York Times | September 18th, 2020

Life expectancy for people with sickle cell disease appears to be declining, and the same systemic racism being protested around the nation could be at least partly responsible, a local expert says.

If you compare other orphan diseases, like cystic fibrosis or hemophilia, which have less cases than sickle cell, and then compare funding and research, its a night-and-day difference, said Dr. Alan R. Anderson, director of Prisma Health Upstates Comprehensive Sickle Cell Disease Program.

The U.S. Food and Drug Administration defines an orphan disease as a condition that affects fewer than 200,000 people nationwide.

That directly goes along with this same prejudice, racial disparities that we see in our political-social landscape, he said. Its about time we recognize this is a serious problem.

Anderson points to a National Institutes of Health study that concluded that life expectancy dropped from 42 for men and 48 for women in the early 1990s to 42 for women and 38 for men in 2005 even as life expectancy increased for people with other chronic conditions.

And a Sept. 1 New England Journal of Medicine article reports that while cystic fibrosis affects a third fewer than sickle cell, it receives seven to 11 times the funding. The authors added that the development of disease-modifying therapies has stagnated because of inadequate research funding, attributable at least in part to structural racism.

Sickle cell disease (SCD) is a genetic disorder of the red blood cells that primarily affects African Americans, though people from Hispanic, southern European, Middle Eastern, southern Asia, or Asian Indian backgrounds can also get the disease. Both parents must carry the genes for their child to get the disease.

While healthy cells are round, in people with SCD they resemble a sickle, or C shape, and are also hard and sticky, clogging the flow of blood through the vessels, according to the U.S. Centers for Disease Control and Prevention. Symptoms include pain, infections and stroke, and typically begin when a child is around 5 months old.

Treatments include medicines that can reduce complications and extend life, the agency reports, but the only cure is a risky bone marrow or stem cell transplant from a close match, like a brother or sister.

The state Department of Health and Environmental Control says that despite improvements in treatments, SCD remains a life-limiting disease with multi-organ complications that reduces the quality of life of impacted individuals especially as the person ages.

DHEC also reports that SCD has suffered from decades of poor disease awareness and lack of funding compared to other inherited disorders.

Historically, Anderson said, while children are cared for via Medicaid, adults often lack insurance so they have no medical home. And along with the funding disparity, there is a lack of training for medical professionals about SCD.

This is not rocket science, he said. If you focus on sickle cell disease like you do on diabetes and other chronic diseases, we will see reductions in acute care needs that will ultimately manifest in life expectancy.

DHEC has an SCD plan that calls for improving access to care, sustainable funding and increasing the educational awareness of medical professionals about SCD.

Its going to take more research and more advocacy and, I believe, policy changes, said the Rev. Sean Dogan of Long Branch Baptist Church in Greenville. We need that same momentum for SCD as well.

Anderson said that Prismas program has stepped up preventive health strategies and treatments for people of all ages while reducing ER visits and hospitalizations by about 50%, which will hopefully increase life expectancy.

Last year, it launched Camp Crescent to provide SCD patients and their families a respite from the disease. But the pandemic prevented that this year.

So organizers are holding a block party from 4-6 p.m. on Sept. 19 where people can safely gather for some fun and to raise awareness, said Dogan.

Its going to be an exciting event to raise awareness [and] celebrate those who have SCD and their caregivers, he said. The more awareness the community has, the more support it will give. And our community is a very generous community.

Modeled on the drive-by birthday parties so popular during the pandemic, it will feature patients and their families driving by the entrance of Prismas Cancer Center on Faris Road, he said. There, theyll find encouragement, well wishes and fun activities like quizzes with prizes, he said.

Meanwhile, on Sunday, blood drives to benefit patients will be held in the area, including one at Long Branch from 10 a.m. to 2 p.m., he said.

To learn more about the event, go to http://events.r20.constantcontact.com/register/event?oeidk=a07eh9btc8wd9af19f2&llr=76g5y7tab or https://www.ghschildrens.org/programs/camp-crescent/.

While the exact number of people with SCD is unknown, its estimated they number about 100,000 nationwide.

South Carolina doesnt track SCD numbers, but between 1991 and 2017, 1,884 infants were born with it, DHEC said. Another 56,607 were born with sickle cell trait, which means they dont have symptoms but can pass the gene on to their children.

Read the original:
Block party planned to raise awareness of sickle cell disease - Greenville Journal

Bone Marrow Stem Cells Comments Off on Block party planned to raise awareness of sickle cell disease – Greenville Journal | September 18th, 2020

After Graham Weston caught the coronavirus from his son, who hadnt shown any symptoms, the San Antonio tech entrepreneur realized that the role of silent spreaders demanded more attention.

He and other prominent philanthropic leaders in the city formed a new nonprofit with the express purpose of screening hundreds and eventually thousands of people to identify who is infected and asymptomatic and keep them from unknowingly spreading the virus.

The larger goal is even more ambitious: deliver an effective way of supporting societys recovery from the pandemic.

We can never really suppress the virus and give people the confidence to go back (to school or work) when we have silent spreaders walking through our population, said Weston, founder of the 80 | 20 Foundation and former CEO and chairman of Rackspace Technology.

The nonprofit, called Community Labs, has adopted a new approach to testing that focuses on micro populations in shared places, such as area schools and businesses.

It is using an existing test developed by Thermo Fisher Scientific. The polymerase chain reaction, or PCR, test relies on a sample taken from the front of the nostril easier on the person getting tested. Results will be available within 24 hours, far faster than many providers of coronavirus tests, the nonprofits leaders said Thursday during their announcement of the initiative.

The nonprofit will pilot the testing effort in the Somerset Independent School District. It has the capacity to process 600 tests a day and aims to scale up to 12,000 tests a day by November, with the goal of lowering the price to $35 per test.

Community Labs is working with BioBridge Global, which houses the nonprofit and conducts the testing in collaboration with UT Health San Antonio. BioBridge, which operates San Antonios blood bank, owns its own lab off Interstate 10 near Vance Jackson on the West Side.

Experts estimate that up to half of people who contract the coronavirus may display no symptoms, said bank executive J. Bruce Bugg Jr., chairman and trustee of the Tobin Endowment and co-founder of Community Labs. While hospitals have widely screened patients for the coronavirus when they are scheduled for surgery, federal and local health officials have largely prioritized testing people with symptoms.

On ExpressNews.com: New nonprofit aims to improve COVID-19 testing

Its an approach that has persisted since early in the pandemic, when testing for the coronavirus was severely limited by regulatory, processing and supply chain bottlenecks. Health departments, including the San Antonio Metropolitan Health District, initially focused their efforts on people who were severely ill, those with classic symptoms and front-line workers.

Metro Health briefly tested asymptomatic people in the community earlier this year but halted the effort when a surge of cases over the summer caused demand for testing to skyrocket.

While testing those with symptoms may help diagnose people with COVID-19, it does little to halt chains of transmission that stem from asymptomatic carriers. To fill that testing gap, Community Labs is taking the exact opposite approach, Bugg said.

He said the goal is to create a strong testing model that screens for asymptomatic carriers and that can be replicated and applied in cities across the state.

Community Labs approach hinges on quick turnaround times, which are not typical with the traditional testing. Waiting a week for results would render the value of testing asymptomatic people moot, Weston said, as they wont know to isolate themselves and already could have spread the virus to others by the time they learn they are infected.

Dr. Rachel Beddard, chief medical officer of BioBridge, said the company already conducts fast, high-volume testing for communicable diseases on blood products collected by the South Texas Blood and Tissue Center and on tissue, bone marrow and stem cells gathered by GenCure, another subsidiary.

Earlier this year, federal regulators granted an emergency use authorization for the coronavirus test that BioBridge is using.

On ExpressNews.com: San Antonio students are back in classrooms very different classrooms

Somerset ISD, a seven-school system in rural Southwest Bexar County, was selected as a demonstration site after its superintendent, Saul Hinojosa, agreed to participate.

With about 40 percent of all students having returned to school for in-person learning, the district will begin testing students and staff Wednesday, starting with students involved in extracurricular activities, all high school staff and all nurses and police officers. Only students whose parents sign a consent form will be tested at school, Hinojosa said.

The goal is to test as many students as possible so they, their parents and staff feel comfortable returning to school. If everything goes according to plan and enough students agree to get tested, Hinojosa said, the entire district could be back learning face to face earlier than expected.

We need students in the classroom because thats where they learn best, and we hope that this strategy will lessen the concern on parents, along with the anxiety, to where they feel confident that the school campus is a safe environment for the kids, he said.

Unlike the deep nasal swabs that have been used to collect samples for many coronavirus tests, the test used by Community Labs uses a sample thats simpler and far less invasive to collect. Participants rub a small Q-tip inside each nostril for five seconds before dropping it into a test tube.

Community Labs was co-founded by Weston, Bugg and J. Tullos Wells, managing director of the Kronkosky Charitable Foundation. Weston is serving as chairman, while Bugg and Wells are vice chairmen.

The Kronkosky and 80 | 20 foundations and the Tobin Endowment have contributed a combined $2.5 million to start the nonprofit.

Lauren Caruba covers health care and medicine in the San Antonio and Bexar County area. To read more from Lauren, become a subscriber. lcaruba@express-news.net | Twitter: @LaurenCaruba

See more here:
Silent spreaders of the coronavirus are the target of a new testing venture in San Antonio - San Antonio Express-News

Bone Marrow Stem Cells Comments Off on Silent spreaders of the coronavirus are the target of a new testing venture in San Antonio – San Antonio Express-News | September 18th, 2020

The European Medicines Agency is on the verge of releasing revised guidance for advanced therapy medicinal products containing genetically modified cells, which includes chimeric antigen receptor (CAR)-T cell therapies.

The Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells was originally issued in 2012 but underwent revision and consultation from July 2018-July 2019. The revised version is expected to be adopted in October and published in November, according to Ana Hidalgo-Simon, MD, PhD, head of advanced therapies at EMA. She previewed the major changes at RAPS Convergence 2020.

There were an enormous number of comments on the document, Hidalgo-Simon said.The agency is also working on a Q&A document on principles of good manufacturing practices (GMP) for Advanced Therapy Medicinal Products (ATMP) starting material. There will likely be consultation on the document in 2021, she said. (RELATED: Regulation of advanced therapy medicinal products in the EU, Regulatory Focus, 16 July 2020.)

Major changesEMA chose to update the guidance to reflect the increase in clinical experience with these therapies, particularly chimeric antigen receptor-T (CAR-T) cells; to cover new categories of products, such as induced pluripotent stem (iPS) cells; and to allow for consideration of new tools for genetic modification of cells, such as genome editing technologies, she said.

The main quality updates are related to starting materials, the manufacturing process, and characterization and release. For example, the starting materials guidance will now include genome editing tools, while the manufacturing process includes a new section on comparability. The characterization and release portion of the guidance includes specific advice for CAR-T cells.

Additionally, the guidance calls for dose-finding studies to explore safety, toxicity, and anti-tumor activity at different dose levels, to define the threshold dose required for anti-tumor effect, and to define the recommended dose or range for Phase 2 studies. She said sponsors need to show a solid rationale for the criteria being used to find the dose.

The guidance also calls for Phase 3 confirmatory trials to follow a randomized controlled design, comparing the CAR-T cell therapy to a reference regimen, unless otherwise scientifically justified. Single-arm studies will continue to be allowed, but they will be the exception, Dr. Hidalgo-Simon said.

Be very careful with the design of the trials, she advised. The assumptions need to be really, very well backed.

When it comes to safety, the guidance calls for a 15-year follow period. While sponsors wont have all the answers at the time of submission, Hidalgo-Simon said they should have a plan that includes monitoring during the post-authorization period.

Hidalgo-Simon also advised sponsors to think beyond the approval process and consider what evidence will be needed to convince other stakeholders -- from patients to payers -- about the safety and efficacy of the therapy.

Avoiding development pitfallsRichard Dennett, PhD, the senior director of chemistry, manufacturing and controls regulatory affairs at PPD, also participated in the RAPS Convergence 2020 session on advanced therapies. He reviewed development points where companies can run into trouble with advanced therapies, particularly CAR-T cell products.Dennett recommended that product sponsors keep the end in mind when developing advanced therapies by focusing on the target product profile at the beginning of development. That profile includes the indication for which approval will be sought and the incidence of that indication; other considerations include mode of action, demographics, how much of the product needs to be produced, and market access and reimbursement considerations.

He also outlined several areas where developers should focus to create a watertight regulatory package, including sufficient product characterization, potency assay, impurities, formulation, stability, lack of sufficient development batches, and validation strategy.

Dennett urged developers to dive into the growing number of regulatory guidance documents for advanced therapies. In addition to the European guidance documents, developers should consultthe US Food and Drug Administrations Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), which was released in January 2020. (RELATED: Advanced therapies: Trip hazards on the development pathway, Regulatory Focus, 02 August 2020)

Live and breathe the guidances that are out there, Dennett advised. They allow us to understand what expectations we need to meet.

The key to success in advancing CAR-T cell therapies is the mitigation of risk, Dennett said: The biggest risk is the one that you havent thought of.RAPS 2020 Convergence

View original post here:
Convergence: EMA close to finalizing guidance for advanced therapies - Regulatory Focus

IPS Cell Therapy Comments Off on Convergence: EMA close to finalizing guidance for advanced therapies – Regulatory Focus | September 18th, 2020

Global stem cell banking market is set to witness a substantial CAGR of 11.03% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. The increased market growth can be identified by the increasing procedures of hematopoietic stem cell transplantation (HSCT), emerging technologies for stem cell processing, storage and preservation. Increasing birth rates, awareness of stem cell therapies and higher treatment done viva stem cell technology.

Get Sample Report + All Related Graphs & Charts (with COVID 19 Analysis) @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-stem-cell-banking-market&pm

Competitive Analysis:

Global stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of inflammatory disease drug delivery market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Competitors:

Few of the major competitors currently working in global inflammatory disease drug delivery market are: NSPERITE N.V, Caladrius, ViaCord, CBR Systems, Inc, SMART CELLS PLUS, LifeCell International, Global Cord Blood Corporation, Cryo-Cell International, Inc., StemCyte India Therapeutics Pvt. Ltd, Cordvida, ViaCord, Cryoviva India, Vita34 AG, CryoHoldco, PromoCell GmbH, Celgene Corporation, BIOTIME, Inc., BrainStorm Cell Therapeutics and others

Market Definition:Global Stem Cell Banking Market

Stem cells are cells which have self-renewing abilities and segregation into numerous cell lineages. Stem cells are found in all human beings from an early stage to the end stage. The stem cell banking process includes the storage of stem cells from different sources and they are being used for research and clinical purposes. The goal of stem cell banking is that if any persons tissue is badly damaged the stem cell therapy is the cure for that. Skin transplants, brain cell transplantations are some of the treatments which are cured by stem cell technique.

Cord Stem Cell Banking MarketDevelopment and Acquisitions in 2019

In September 2019, a notable acquisition was witnessed between CBR and Natera. This merger will develop the new chances of growth in the cord stem blood banking by empowering the Nateras Evercord branch for storing and preserving cord blood. The advancement will focus upon research and development of the therapeutic outcomes, biogenetics experiment, and their commercialization among the global pharma and health sector.

Cord Stem Cell Banking MarketScope

Cord Stem Cell Banking Marketis segmented on the basis of countries into U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

All country based analysis of the cord stem cell banking marketis further analyzed based on maximum granularity into further segmentation. On the basis of storage type, the market is segmented into private banking, public banking. On the basis of product type, the market is bifurcated into cord blood, cord blood & cord tissue. On the basis of services type, the market is segmented into collection & transportation, processing, analysis, storage. On the basis of source, market is bifurcated into umbilical cord blood, bone marrow, peripheral blood stem, menstrual blood. On the basis of indication, the market is fragmented into cerebral palsy, thalassemia, leukemia, diabetes, autism.

Cord stem cell trading is nothing but the banking of the vinculum plasma cell enclosed in the placenta and umbilical muscle of an infant. This ligament plasma comprises the stem blocks which can be employed in the forthcoming time to tackle illnesses such as autoimmune diseases, leukemia, inherited metabolic disorders, and thalassemia and many others.

Market Drivers

Increasing rate of diseases such as cancers, skin diseases and othersPublic awareness associated to the therapeutic prospective of stem cellsGrowing number of hematopoietic stem cell transplantations (HSCTs)Increasing birth rate worldwide

Market Restraint

High operating cost for the therapy is one reason which hinders the marketIntense competition among the stem cell companiesSometimes the changes are made from government such as legal regulations

Key Pointers Covered in the Cord Stem CellBanking MarketIndustry Trends and Forecast to 2026

Market SizeMarket New Sales VolumesMarket Replacement Sales VolumesMarket Installed BaseMarket By BrandsMarket Procedure VolumesMarket Product Price AnalysisMarket Healthcare OutcomesMarket Cost of Care AnalysisMarket Regulatory Framework and ChangesMarket Prices and Reimbursement AnalysisMarket Shares in Different RegionsRecent Developments for Market CompetitorsMarket Upcoming ApplicationsMarket Innovators Study

Key Developments in the Market:

In August, 2019, Bayer bought BlueRock for USD 600 million to become the leader in stem cell therapies. Bayer is paying USD 600 million for getting full control of cell therapy developer BlueRock Therapeutics, promising new medical area to revive its drug development pipeline and evolving engineered cell therapies in the fields of immunology, cardiology and neurology, using a registered induced pluripotent stem cell (iPSC) platform.In August 2018, LifeCell acquired Fetomed Laboratories, a provider of clinical diagnostics services. The acquisition is for enhancement in mother & baby diagnostic services that strongly complements stem cell banking business. This acquisition was funded by the internal accruals which is aimed to be the Indias largest mother & baby preventive healthcare organization.

For More Insights Get FREE Detailed TOC @https://www.databridgemarketresearch.com/toc/?dbmr=global-stem-cell-banking-market&pm

Research objectives

To perceive the most influencing pivoting and hindering forces in Cord Stem Cell Banking Market and its footprint in the international market.Learn about the market policies that are being endorsed by ruling respective organizations.To gain a perceptive survey of the market and have an extensive interpretation of the Cord Stem Cell Banking Market and its materialistic landscape.To understand the structure of Cord Stem Cell Banking Market by identifying its various sub segments.Focuses on the key global Cord Stem Cell Banking Market players, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).To project the consumption of Cord Stem Cell Banking Market submarkets, with respect to key regions (along with their respective key countries).To strategically profile the key players and comprehensively analyze their growth strategiesTo analyze the Cord Stem Cell Banking Market with respect to individual growth trends, future prospects, and their contribution to the total market.

Customization of the Report:

All segmentation provided above in this report is represented at country levelAll products covered in the market, product volume and average selling prices will be included as customizable options which may incur no or minimal additional cost (depends on customization)

Contact:

Data Bridge Market Research

US: +1 888 387 2818

UK: +44 208 089 1725

Hong Kong: +852 8192 7475

Email @[emailprotected]

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.

Read the original:
Cord Stem Cell Banking Market Analysis 2020 With COVID 19 Impact Analysis| Leading Players, Business Prospects, In-depth Analysis Research Report...

Skin Stem Cells Comments Off on Cord Stem Cell Banking Market Analysis 2020 With COVID 19 Impact Analysis| Leading Players, Business Prospects, In-depth Analysis Research Report… | September 18th, 2020

With September being Sickle Cell Disease Awareness Month, Loma Linda University Childrens Health wants to help educate the community about SCD one of the most common yet overlooked genetic disorders in the world.

Each year, approximately 1,000 babies in the U.S. and 500,000 worldwide are born with the disease, according to the Sickle Cell Disease Association of America.

Akshat Jain, MD, MPH, a global sickle cell disease expert at Childrens Hospital, is passionate about establishing awareness and proper care for children suffering from SCD and Sickle Cell Trait, especially the diverse patient population in San Bernardino County.

There are many barriers to receiving care for those with SCD in our community, Jain says. One barrier specifically is lack of awareness surrounding the disease coupled with lack of awareness surrounding the treatment options available at Childrens Hospital.

In sickle cell disease, a persons red blood cells have an irregular cell shape, Jain says. Instead of round discs, theyre in a crescent or sickle shape.

Due to their shape, texture and inflexibility, the cells become clumped together. This grouping causes a blockage in a childs blood vessels, hindering blood-flow. This blockage may cause varying levels of pain and potentially organ damage long-term.

Jain says some of the signs and symptoms of SCD include:

Jain says that many children with SCD develop symptoms in their first year of life. SCD is commonly diagnosed during newborn screening tests, which check for the abnormal hemoglobin found in SCD. Additionally, if both parents of a child are known carriers of a SCD trait, their child will have a 25% chance of having the disease, Jain says.

Some of the emergent issues needing immediate medical care in kids with SCD disease include:

Treatments for SCD include pain medicines for pain management, adequate hydration, blood transfusions, vaccines and antibiotics, and some medicines. Currently, stem cell transplant from bone marrow is the recognized cure for SCD.

Childrens Hospital, with Jain working as a lead on the team, performed the institutions first stem cell transplant in 2019, curing a then 11-year-old girl who had suffered from SCD since birth. Since then, the team has successfully performed the transplant on several pediatric patients.

Patients with SCD at Childrens Hospital are placed into a treatment and care program where Jain and his team offer non-traditional services such as individualized patient treatment plans and direct access to the care team in case of an emergent event. Additionally, the program is working toward offering curative gene therapy for both sickle cell and hemophilia patients.

The bottom line is children and families suffering from this disease need to know that theyre not alone, Jain says. Here at Childrens Hospital, we are here to manage and fight this disease alongside of you.

Learn more about our treatments for sickle cell disease at our Specialty Team Centers.

See the article here:
Here's what to know about Sickle Cell Disease in kids - Loma Linda University Health

Skin Stem Cells Comments Off on Here’s what to know about Sickle Cell Disease in kids – Loma Linda University Health | September 18th, 2020

Setting the tempo for development

Many animals display similarities in their organization (body axis, organ systems, and so on). However, they can display vastly different life spans and thus must accommodate different developmental time scales. Two studies now compare human and mouse development (see the Perspective by Iwata and Vanderhaeghen). Matsuda et al. studied the mechanism by which the human segmentation clock displays an oscillation period of 5 to 6 hours, whereas the mouse period is 2 to 3 hours. They found that biochemical reactions, including protein degradation and delays in gene expression processes, were slower in human cells compared with their mouse counterparts. Rayon et al. looked at the developmental tempo of mouse and human embryonic stem cells as they differentiate to motor neurons in vitro. Neither the sensitivity of cells to signals nor the sequence of gene-regulatory elements could explain the differing pace of differentiation. Instead, a twofold increase in protein stability and cell cycle duration in human cells compared with mouse cells was correlated with the twofold slower rate of human differentiation. These studies show that global biochemical rates play a major role in setting the pace of development.

Science, this issue p. 1450, p. eaba7667; see also p. 1431

What determines the pace of embryonic development? Although the molecular and cellular mechanisms of many developmental processes are evolutionarily conserved, the pace at which these operate varies considerably between species. The tempo of embryonic development controls the rate of individual differentiation processes and determines the overall duration of development. Despite its importance, however, the mechanisms that control developmental tempo remain elusive.

Comparing highly conserved and well-characterized developmental processes in different species permits a search for mechanisms that explain differences in tempo. The specification of neuronal subtype identity in the vertebrate spinal cord is a prominent example, lasting less than a day in zebrafish, 3 to 4 days in mouse, and around 2 weeks in human. The development of the spinal cord involves a well-defined gene regulatory program comprising a series of stereotypic changes in gene expression, regulated by extrinsic signaling as cells differentiate from neural progenitors to postmitotic neurons. The regulatory program and resulting neuronal cell types are highly similar in different vertebrates, despite the difference in tempo between species. We therefore set out to characterize the pace of differentiation of one specific neuronal subtypemotor neuronsin human and mouse and to identify molecular differences that explain differences in pace. To this end, we took advantage of the in vitro recapitulation of in vivo developmental programs using the directed differentiation of human and mouse embryonic stem cells.

We found that all stages of the developmental progression from neural progenitor to motor neuron were proportionally prolonged in human compared with mouse, resulting in human motor neuron differentiation taking about 2.5 times longer than mouse. Differences in tempo were not due to differences in the sensitivity of cells to signals, nor could they be attributed to differences in the sequence of the key genes or their regulatory elements. Instead, the data revealed that changes in protein stability correlated with developmental tempo, such that slower temporal progression in human corresponded to increased protein stability. An in silico model indicated that increased protein stability could account for the slower tempo of development in human compared with mouse.

The results suggest that differences in protein turnover play a role in interspecies differences in the pace of motor neuron differentiation. The identification of a molecular mechanism that can explain differences in the pace of embryonic development between species focuses attention on the role of protein stability in tempo control. This suggests a parsimonious explanation for the substantial variation in the tempo of development between species and indicates how the overall dynamics of developmental processes can be influenced by kinetic properties of gene regulation. What determines species-specific rates of protein turnover remains to be determined, but the availability of in vitro systems that mimic in vivo developmental tempo opens up the possibility of exploring this issue.

Different animal species develop at different tempos, and equivalent developmental stages can be matched between mouse and human at different developmental time points. Neural progenitors in the spinal cord progress through the same succession of gene expression to generate motor neurons in mouse and human, and this serves as a model to study tempo differences. The in vitro directed differentiation of mouse embryonic stem cells to motor neurons advances at greater than twice the speed of human embryonic stem cell differentiation. The equivalent progression of development at different rates is shown for the transcription factors PAX6 (green), OLIG2 (red), and NKX2.2 (blue). E, embryonic day; W, embryonic week; CS, Carnegie stage. Scale bars are 50 m.

Although many molecular mechanisms controlling developmental processes are evolutionarily conserved, the speed at which the embryo develops can vary substantially between species. For example, the same genetic program, comprising sequential changes in transcriptional states, governs the differentiation of motor neurons in mouse and human, but the tempo at which it operates differs between species. Using in vitro directed differentiation of embryonic stem cells to motor neurons, we show that the program runs more than twice as fast in mouse as in human. This is not due to differences in signaling, nor the genomic sequence of genes or their regulatory elements. Instead, there is an approximately two-fold increase in protein stability and cell cycle duration in human cells compared with mouse cells. This can account for the slower pace of human development and suggests that differences in protein turnover play a role in interspecies differences in developmental tempo.

Go here to see the original:
Species-specific pace of development is associated with differences in protein stability - Science Magazine

Spinal Cord Stem Cells Comments Off on Species-specific pace of development is associated with differences in protein stability – Science Magazine | September 18th, 2020

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

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