Archive for the ‘Bone Marrow Stem Cells’ Category

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. Moesers prior 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 substances like 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 likely to 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 the biology 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.

For more information on Moesers research, go to the Gastrointestinal Stress Biology Laboratory. Also, visit the MSU College of Veterinary Medicines website for more about its research efforts.

(Note for media: Please include the following link to the study in all online media coverage: https://www.pnas.org/content/early/2020/09/10/1915075117)

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New research connects the hormones we're born with to lifetime risk for immunological diseases - MSUToday

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.

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

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

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.

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Block party planned to raise awareness of sickle cell disease - Greenville Journal

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

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

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.

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The Pandemic Is Chasing Aging Coaches From the Field - The New York Times

WASHINGTON, Sept. 4, 2020 /PRNewswire/ -- During the COVID-19 pandemic, many stem cell transplant centers including guidance from the National Marrow Donor Program (NMDP) recommend that stem cell products be frozen for preservation. However, a new study in Blood Advances suggests that the freezing process can decrease the quality of stem cells, particularly if they were manipulated before being preserved, if they had high white blood cell content, or if they were stored for a long period of time.

Stem cells can develop into many different types of cells, so they are often used in treatment to replace or repair damaged organs or tissues. Allogeneic stem cell transplantation, which involves transferring stem cells from a healthy donor to a patient, can treat a variety of diseases, including leukemia, lymphoma, myeloma, thalassemia, and sickle cell disease.

Before the coronavirus outbreak, it was not common to freeze allogeneic donor stem cells prior to infusion. However, due to COVID-19's effects on donor and hospital availability, as well as new travel and transportation restrictions, more transplant centers including the NMDP are recommending cryopreservation.

"Prior to COVID-19, the donor and transplant systems were well coordinated and effective. Now, with irregular flights and closed borders, travel and transportation are not assured," said lead study author Duncan Purtill, MD, of Fiona Stanley Hospital in Western Australia. "Five to seven days before stem cell transplant, the recipient usually starts chemotherapy to remove all their bone marrow cells. Without a healthy transplant to replace the cells on the same day, they would be left with no functioning bone marrow, which would of course be very high risk and carry a poor prognosis. Life literally depends on the safe arrival and immediate infusion of stem cells."

Dr. Purtill and his team analyzed 305 samples of allogeneic stem cell products that were cryopreserved at participating Australian cell processing labs between 2015 and 2019. They found that, on average, the recovery of the stem cell products was 74%. This is considered an acceptable, viable recovery, enabling the cells to be used in transplantation. However, some products did not recover to that level: around 15% of the surveyed products had a cell recovery of less than 50%. In fact, the study found that quality recovery could range as low as just 6%. Such a significant cell loss after thawing may mean that the remaining cells may be too few, or too damaged, to achieve timely bone marrow recovery in the patient after infusion.

"It seems that there is variability in recovery and more work needs to be done to determine why," said Dr. Purtill. "When we freeze stem cells and then thaw them afterwards, we sometimes get unexpected results. In this study we identified some possible factors influencing that variability."

The research team pointed to three possible reasons for the loss of quality in some of the stem cells products they analyzed. First, they noted that prolonged transportation and storage time prior to cryopreservation was associated with a loss of quality. They also found that higher white cell concentration of the product affected its quality. It was thought that the presence of other white cells could adversely affect the stem cells, either by releasing damaging enzymes or chemicals, or else by consuming nutritional elements within the product and resulting in less healthy stem cells. And finally, they pointed out that a small proportion of cells which underwent complex manipulation before being frozen also suffered quality loss for instance, when the cell processing lab removed lymphocytes or washed the product to remove plasma and other noncellular components.

Dr. Purtill and his collaborators expressed hope that their findings could serve to inform and improve stem cell transplantation, collection, and processing procedures. "Our findings could be a note of caution for transplant centers to not take for granted that the frozen product they have received will show perfect recovery once thawed," said Dr. Purtill. "I hope centers will insist on receiving a pilot vial which has been frozen and transported in the same way. They can assess the pilot vial to determine its viability before they use the full product and start chemotherapy for the patient."

Blood Advancesis a peer-reviewed, online only, open access journal of the American Society of Hematology (ASH), the world's largest professional society concerned with the causes and treatment of blood disorders.

Blood Advances is a registered trademark of the American Society of Hematology.

SOURCE Blood Advances

http://www.bloodadvances.org

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Study: Cryopreservation Associated with Loss of Quality in Donor Stem Cell Products - PRNewswire

PHOENIX, Sept. 8, 2020 /PRNewswire/ --(OTC CELZ) -- Creative Medical Technology Holdings Inc. announced today receipt of a Notice of Allowance from the United States Patent and Trademark Office for its patent application, "Perispinal Perfusion by Administration of T Regulatory Cells Alone or in Combination with Angiogenic Cell Therapies."

The patent covers the use of activated T regulatory cells for inducing an increase in blood circulation in areas surrounding the disc of patients with lower back pain. It is believed that a significant proportion of patients suffering from lower back pain have abnormally poor circulation, which does not effectively remove waste products and irritants. Restoration of circulation in the lower back is associated with reduction of pain.

The Company acquired a previously granted US Patent # 9,598,673 covering use of various types of stem cells, autologous and allogeneic, for treating lower back pain. The Company has completed an autologous cell therapy pilot study in the area of lower back pain utilizing this patented technology and is currently in the process of assembling data for publication.

"Creative Medical Technology Holdings is developing a critical mass of issued intellectual property covering multiple cell therapy treatments of lower back pain as well as other indications," said Timothy Warbington, President and CEO of the Company. "Testimony to the size of the lower back pain market is the $1 Billion Mesoblast-Grunenthal deal for a pre-review cell therapy product1. We are enthusiastic to add this new therapy to our expanding portfolio of rapid-to-commercialize cellular therapies."

Creative Medical Technology Holdings has previously commercialized its CaverStemR technology involving personalized bone marrow cellular therapy for erectile dysfunction. This technology is covered by issued patent # 8,372,797 and a clinical trial demonstrating safety with signals of efficacy published in the peer-reviewed literature2.

"Immunotherapy is one segment of the biotechnology industry that is expanding at an exponential rate," said Donald Dickerson, CFO of the Company. "The recent Nobel Prize in the area of Immunotherapy of Cancer, as well as the current valuations of immunotherapy companies, validates the approaches that we have been developing, and now patenting. Essentially our approach is to use stem cells, or immunotherapy to enable the body to heal itself."

"The Company welcomes the biotechnology/life sciences community and key opinion leaders to contact us to discuss potential collaboration on our patented technologies in this amazing space," Mr. Warbington said further.

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in stem cell technology in the fields of urology, neurology and orthopedics and trades on the OTC under the ticker symbol CELZ. For further information about the company, please visit http://www.creativemedicaltechnology.com.

Forward Looking Statements

OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website at http://www.sec.gov.

Creativemedicaltechnology.comwww.StemSpine.com http://www.Caverstem.com http://www.Femcelz.com

1 https://www.biopharma-reporter.com/Article/2019/09/12/Gruenenthal-partners-with-Mesoblast-for-back-pain-cell-therapy#:~:text=Gr%C3%BCnenthal%20agrees%20deal%20with%20Mesoblast,disease%20in%20previously%20treated%20patients.

2 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6958721/

SOURCE Creative Medical Technology Holdings, Inc.

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Application of Immunotherapy to $7 Billion Lower Back Pain Market Patented by Creative Medical Technology Holdings - PRNewswire

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

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

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

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

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Global Hematopoietic Stem Cells Transplantation Market to Witness Rapid Development During the Period 2017 2025 - The News Brok

A novel Bcl-2 inhibitor, APG-2575, has been granted an Orphan Drug designation (ODD) by the FDA for the treatment of patients with chronic lymphocytic leukemia (CLL), announced Ascentage Pharma in a press release.1

This marks the second ODD for APG-2575, after 1 was granted to the drug in July 2020 for the treatment of Waldenstrm Macroglobulinemia (WM).2

At present, CLL still presents considerable unmet medical needs. APG-2575 is a key drug candidate in Ascentage Pharma's pipeline targeting apoptosis. The APG-2575 received this ODD from the FDA shortly after the first ODD in WM, and this designation will be helpful in enhancing our communication with the FDA and expediting our development of APG-2575 in these rare cancer diseases," said Yifan Zhai, MD, PhD, chief medical officer, Ascentage Pharma, in a statement.1 All the policy support and incentives as a result of this ODD will help us accelerate the global clinical development of APG-2575, which we hope will soon offer additional treatment options for patients with CLL."

In hematologic malignancies, APG-2575 may selectively block Bcl-2 as a way to renew the apoptosis process in cancer cells. The first study of APG-2575 in CLL, as well as in small lymphocytic leukemia (SLL), is currently recruiting 35 patients with relapsed or refractory disease. In the phase 1b dose-escalation study (NCT04215809), patients will receive APG-2575 alone or in combination with other therapeutic agents. The primary end point of the study is dose-limiting toxicity, and the secondary end point is the maximum tolerated dose of APG-2575.

The study will follow a non-randomized 3 + 3 design at a starting dose of 200 mg given on day 1 of a 28-day cycle. The dose will be increased to 400 mg, followed by 600 mg, 800 mg, and 1200 mg.

To be included in the trial, patients must be 18 years or older with a histologically confirmed diagnosis of CLL/SLL, and ECOG performance status of 2 or lower, adequate bone marrow function, and a serum creatinine level of 1.5upper limit of normal. In part 1, patients will be eligible for dose escalation if they have received 3 or fewer prior lines of systemic therapy. Female patients are required to be postmenopausal for 2 years or surgically sterile prior to beginning treatment in the study.

Patients are excluded from this study if they have undergone allogeneic stem cell transplant within 90 days of joining the study, have active graft-versus-host-disease or are in need of immunosuppressive therapy, and/or have Richter's syndrome. The study also excludes patients with certain prior therapies and comorbidities that may interfere with APG-2575 treatment.

Multiple cancer centers in the United States are involved in the phase 1b study of APG-2575 including the Mayo Clinic in Scottsdale Arizona, City of Hope in Duarte, California, Dana-Farber Cancer Institute in Boston, Massachusetts, Novant Health in Charlotte, North Carolina, Grabrail Cancer Center in Canton, Ohio, Cleveland Clinic in Cleveland, Ohio, and Swedish Health in Seattle, Washington.

Outside of the realm CLL/SLL, APG-2575 is being investigated in other hematologic malignancies like WM, AML, and T-cell prolymphocytic leukemia. Studies of APG-2575 in these disease states are currently recruiting patients in centers in the United States, Australia, and China.

References:

1. Ascentage Pharma's Bcl-2 inhibitor apg-2575 granted Orphan Drug designation by the FDA for the treatment of chronic lymphocytic leukemia. News release. Ascentage Pharmaceuticals. September 7, 2020. Accessed September 8, 2020. https://prn.to/2ZiOqFJ

2. Ascentage Pharmas Bcl-2 inhibitor apg-2575 granted Orphan Drug Designation by the FDA for the treatment of waldenstrm macroglobulinemia. News release. Ascentage Pharmaceuticals. July 15, 2020. Accessed September 8, 2020. https://bit.ly/329A5gL

Excerpt from:
FDA Grants Orphan Drug Designation to Novel Bcl-2 inhibitor in CLL - Targeted Oncology

WASHINGTON, Sept. 4, 2020 /PRNewswire/ -- During the COVID-19 pandemic, many stem cell transplant centers including guidance from the National Marrow Donor Program (NMDP) recommend that stem cell products be frozen for preservation. However, a new study in Blood Advances suggests that the freezing process can decrease the quality of stem cells, particularly if they were manipulated before being preserved, if they had high white blood cell content, or if they were stored for a long period of time.

Stem cells can develop into many different types of cells, so they are often used in treatment to replace or repair damaged organs or tissues. Allogeneic stem cell transplantation, which involves transferring stem cells from a healthy donor to a patient, can treat a variety of diseases, including leukemia, lymphoma, myeloma, thalassemia, and sickle cell disease.

Before the coronavirus outbreak, it was not common to freeze allogeneic donor stem cells prior to infusion. However, due to COVID-19's effects on donor and hospital availability, as well as new travel and transportation restrictions, more transplant centers including the NMDP are recommending cryopreservation.

"Prior to COVID-19, the donor and transplant systems were well coordinated and effective. Now, with irregular flights and closed borders, travel and transportation are not assured," said lead study author Duncan Purtill, MD, of Fiona Stanley Hospital in Western Australia. "Five to seven days before stem cell transplant, the recipient usually starts chemotherapy to remove all their bone marrow cells. Without a healthy transplant to replace the cells on the same day, they would be left with no functioning bone marrow, which would of course be very high risk and carry a poor prognosis. Life literally depends on the safe arrival and immediate infusion of stem cells."

Dr. Purtill and his team analyzed 305 samples of allogeneic stem cell products that were cryopreserved at participating Australian cell processing labs between 2015 and 2019. They found that, on average, the recovery of the stem cell products was 74%. This is considered an acceptable, viable recovery, enabling the cells to be used in transplantation. However, some products did not recover to that level: around 15% of the surveyed products had a cell recovery of less than 50%. In fact, the study found that quality recovery could range as low as just 6%. Such a significant cell loss after thawing may mean that the remaining cells may be too few, or too damaged, to achieve timely bone marrow recovery in the patient after infusion.

"It seems that there is variability in recovery and more work needs to be done to determine why," said Dr. Purtill. "When we freeze stem cells and then thaw them afterwards, we sometimes get unexpected results. In this study we identified some possible factors influencing that variability."

The research team pointed to three possible reasons for the loss of quality in some of the stem cells products they analyzed. First, they noted that prolonged transportation and storage time prior to cryopreservation was associated with a loss of quality. They also found that higher white cell concentration of the product affected its quality. It was thought that the presence of other white cells could adversely affect the stem cells, either by releasing damaging enzymes or chemicals, or else by consuming nutritional elements within the product and resulting in less healthy stem cells. And finally, they pointed out that a small proportion of cells which underwent complex manipulation before being frozen also suffered quality loss for instance, when the cell processing lab removed lymphocytes or washed the product to remove plasma and other noncellular components.

Dr. Purtill and his collaborators expressed hope that their findings could serve to inform and improve stem cell transplantation, collection, and processing procedures. "Our findings could be a note of caution for transplant centers to not take for granted that the frozen product they have received will show perfect recovery once thawed," said Dr. Purtill. "I hope centers will insist on receiving a pilot vial which has been frozen and transported in the same way. They can assess the pilot vial to determine its viability before they use the full product and start chemotherapy for the patient."

Blood Advancesis a peer-reviewed, online only, open access journal of the American Society of Hematology (ASH), the world's largest professional society concerned with the causes and treatment of blood disorders.

Blood Advances is a registered trademark of the American Society of Hematology.

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Study: Cryopreservation Associated with Loss of Quality in Donor Stem Cell Products - WFMZ Allentown

Could gene therapy provide a solution to HIV? A new research project aims to find out.

The National Institutes of Health(NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that. According to an announcement from the Keck School of Medicine at USC, the goal will be to develop a therapy that prepares patients for a stem cell transplantation using their own cells with little to no toxicity, engineers their own stem cells to fight HIV and stimulates those cells to quickly produce new and engineered immune cells once they're reintroduced into the patient. The hematopoietic stem cell transplants, also known as bone marrow transplants, have been used to treat some blood cancers. The idea is to infuse an HIV patient withhealthy donor blood stem cells that can grow into any type of blood or immune cell.

The gene therapy strategy has been inspired by three cases where leukemia patients who also had HIV received blood stem cell transplants from donors who also carried a mutation that confers immunity to HIV. The mutation was in the CCR5 gene, which encodes a receptor that HIV uses to infect immune cells and is present in about 1 percent of the population, USC said.

The program will engineer blood cells to remove CCR5 from a patient's own stem cells.That will be combined with other genetic changes so that the progeny of engineered stem cells will release antibodies and antibody-like molecules that block HIV.

In addition to the potential gene therapy treatment, researchers are also assessing whether or not CAR-T treatments will benefit HIV patients. Researchers from Harvard University developed a Dual CAR T-cell immunotherapy that can potentially help fight HIV infection. First reported by Drug Target Review, the HIV-specific CAR-T cell is being developed to not only target and eliminated HIV-infected cells, but also reproduce in vivo to enable the patients to fight off the infection. HIVs primary target it T cells, which are part of the bodys natural immune response.

Todd Allen, a professor of Medicine at Harvard Medical School, said the Dual CAR-T cell immunotherapy has so far provided a strong, long-lasting response against HIV-infection while being resistant to the virus itself.

According to the report, theDual CAR T cell was developed through the engineering of two CARs into a single T cell. Each of the CARs contained a CD4 protein that allowed it to target HIV-infected cells and a costimulatory domain, which signaled the CAR T cell to increase its immune functions. As DTR reported, the first CAR contained the 4-1BB co-stimulatory domain, which stimulates cell proliferation and persistence, while the second has the CD28 co-stimulatory domain, which increases its ability to kill infected cells.

To protect the CAR-T cells from HIV, the team added the protein C34-CXCR4, which prevents HIV from attaching to and infecting cells. When that was added, the researchers found in animal models that the treatment was long-lived, replicated in response to HIV infection, killed infected cells effectively and was partially resistant to HIV infection.

Still, other researchers are looking to those rare individuals who are infected with HIV but somehow on their own are able to suppress the virus without the need for any treatment. Researchers have sought to replicate what this small percentage of patients can naturally do in other patients who require those daily regimens of medications. Through the sequencing of the genetic material of those rare individuals, researchers made an interesting discovery.

The team discovered large numbers of intact viral sequences in the elite controllers chromosomes. But in this group, the genetic material was restricted to inactive regions, where DNA is not transcribed into RNA to make proteins, MedNewsToday reported.

Now the race is on to determine how this can be replicated and used to treat the nearly 38 million people across the globe who have been diagnosed with HIV.

Original post:
New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients - BioSpace

Global Human Mesenchymal Stem Cells (hMSC) Market report forecast 2020-2024 investigate the market size, manufactures, types, applications and key regions like North America, Europe, Asia Pacific, Central & South America and Middle East & Africa, focuses on the consumption of Human Mesenchymal Stem Cells (hMSC) in these regions. This report also studies the global Human Mesenchymal Stem Cells (hMSC) market share, competition landscape, status share, growth rate, future trends, market drivers, opportunities and challenges, sales channels and distributors.

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Human Mesenchymal Stem Cells (hMSC) Market Manufactures:

Human Mesenchymal Stem Cells (hMSC) Market Types:

Human Mesenchymal Stem Cells (hMSC) Market Applications:

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Scope of this Report:

The content of the study subjects, includes a total of 15 chapters:

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Table of Contents of Human Mesenchymal Stem Cells (hMSC) Market:

1 Market Overview

1.1 Human Mesenchymal Stem Cells (hMSC) Introduction

1.2 Market Analysis by Type

1.2.1 Type 1

1.2.2 Type 2

1.3 Market Analysis by Applications

1.3.1 Application 1

1.3.2 Application 2

1.4 Market Analysis by Regions

1.4.1 North America (United States, Canada and Mexico)

1.4.2 Europe (Germany, France, UK, Russia and Italy)

1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia)

1.4.4 South America, Middle East and Africa

1.4.4.5 Turkey Market States and Outlook (2014-2024)

1.5 Market Dynamics

1.5.1 Market Opportunities

1.5.2 Market Risk

1.5.3 Market Driving Force

2 Manufacturers Profiles

2.1 Manufacture

2.1.1 Business Overview

2.1.2 Human Mesenchymal Stem Cells (hMSC) Type and Applications

2.1.2.1 Product A

2.1.2.2 Product B

2.1.3 Manufacture Human Mesenchymal Stem Cells (hMSC) Sales, Price, Revenue, Gross Margin and Market Share (2017-2018)

3 Global Human Mesenchymal Stem Cells (hMSC) Sales, Revenue, Market Share and Competition by Manufacturer (2017-2018)

3.1 Global Human Mesenchymal Stem Cells (hMSC) Sales and Market Share by Manufacturer (2017-2018)

3.2 Global Human Mesenchymal Stem Cells (hMSC) Revenue and Market Share by Manufacturer (2017-2018)

3.3 Market Concentration Rate

3.3.1 Top 3 Human Mesenchymal Stem Cells (hMSC) Manufacturer Market Share in 2018

3.3.2 Top 6 Human Mesenchymal Stem Cells (hMSC) Manufacturer Market Share in 2018

3.4 Market Competition Trend

13 Sales Channel, Distributors, Traders and Dealers

13.1 Sales Channel

13.1.1 Direct Marketing

13.1.2 Indirect Marketing

13.1.3 Marketing Channel Future Trend

13.2 Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

15.1 Methodology

15.2 Data Source

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Global Human Mesenchymal Stem Cells (hMSC) Market 2020, Impact of Covid-19 on Leading Vendors, Types, Applications, Regions and Forecast to 2024 - The...

In 2016, Danny Wade, a successful marketing professional and an active and doting father to his three young children, aged 11, 8 and 6, checked himself into the emergency department when he began experiencing severe, inexplicable bone pain and unusual fatigue.

Two days later, after undergoing a battery of tests, Danny was diagnosed with multiple myeloma, a little-known and incurable cancer of the plasma cells. He was just 42 years old.

"I was shocked when I got the news, Danny recalls. What upset me most was when the doctor told me that the average life expectancy for myeloma patients was only five to seven years. The thought that I would not see my children grow up was devastating. I knew I had to fight for my life.

Thats exactly what Danny has been doing. Within six months of being diagnosed, Danny went through a difficult high-dose chemotherapy regimen to prepare for an autologous stem cell transplant using his own stem cells. Then in 2017 after further tests, Dannys doctors recommended that his best option for survival was to undergo another transplant with stem cells from a healthy donor. He took his doctors advice and underwent the procedure. Fortunately, he was eligible to participate in a clinical trial at Maisonneuve-Rosemont Hospital where he received a breakthrough therapy involving bi-weekly injections that he will continue to take for a year.

Dannys condition is relatively stable at this time, and he extremely thankful to still be alive. He is thrilled to watch his children grow and to resume being an active part of their lives. He credits his survival to the life-saving treatments that he has access to and the love and support he receives from his partner, Anik. With my beloved Anik by my side, Ive had the courage to get through this nightmare and to have faith that I can get through whatever else the future holds.

Danny is eager to do what he can to help others living with myeloma. I made a promise that once I was doing well, I would do everything in my power to help find a cure so that other patients dont have to live through the horrors I have," says Danny. Danny is a member of the organizing committee of the Montreal Support Group, and recently co-founded the South Shore Myeloma Support Group.

Over the past four years, Danny has seen, first-hand, the life-changing impact that advances in myeloma research are having on the lives of those living with this incurable cancer. Thats why he and his family are more intent than ever to raise as much awareness and funds for myeloma as they can, and will be participating in Myeloma Canadas 12th annual Montreal Multiple Myeloma March on Sunday, September 20, at 10 am.

This years Montreal March has been modified to help stop the spread of COVID-19. In compliance with physical distancing measures, participants are encouraged to hold their own walk in their neighbourhood at the same time as the regularly scheduled March on September 20. Danny and his fellow Montreal Marchers have set their fundraising goal at $60,000 to help further crucial research for this deadly blood cancer that affects nine new Canadians every day.

Myeloma research has produced extremely promising results over the past two decades. In fact, for the first time, theres a cure in sight, says Dr Richard LeBlanc, Medical Hematologist and Oncologist, and holder of the Myeloma Canada Chair in Multiple Myeloma Research at the Universit de Montral. We cant afford to let the current situation stop the progress weve made and put vulnerable people living with myeloma at risk, which is why its more crucial than ever to invest in research and find a cure.

The Multiple Myeloma March, Myeloma Canadas flagship fundraiser is now in its 12th year. The annual five-kilometer event brings Canadian communities together to raise essential funds for research and to help improve the lives of all Canadians impacted by myeloma. Montreal is one of a record 33 communities across the country to be included in this years Multiple Myeloma March. The national fundraising goal is set at $650,000. To learn more about how this event will be working, please click here.

While this years March will undoubtedly be different because of the pandemic, its crucial to stay positive, says Martine Elias, Executive Director of Myeloma Canada. Fundraising has taken a huge hit for many organizations. We need to do all we can to increase awareness and raise essential funds for research that will improve the lives of Canadians impacted by myeloma, and bring us closer to a cure, Martine added. As we mark Myeloma Canadas 15th anniversary, we celebrate the strength of our incredible community. More than ever, were counting on our supporters to help us achieve our goal of $650,000. Canadians impacted by this incurable cancer are depending on us.

This year, a minimum of 50% of funds raised by the Multiple Myeloma March will go directly to support Myeloma Canadas Myeloma Research Priority Setting Partnership (PSP), the first program of its kind in myeloma. The PSP will use input provided by the Canadian myeloma community to identify and define investments in myeloma research over the next 18 months. The balance raised will go toward supporting various myeloma research projects and initiatives that are pivotal for improving quality of life and moving the needle toward a cure.

Multiple myeloma, also known as myeloma, is the second most common form of blood cancer. Myeloma affects a type of immune cell called the plasma cell, found in the bone marrow. Every day, nine Canadians are diagnosed, yet in spite of its growing prevalence, the disease remains relatively unknown. While there is no cure, people with myeloma are living longer and better lives, thanks to recent breakthroughs in treatment. To find the cure, more funding and research are required. To learn more, or to donate, please visit http://www.myeloma.ca

Myeloma Canada

http://www.myeloma.ca

AB

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Father of Three with Incurable Cancer is Helping Researchers Get One Step Closer to a Cure - The Suburban Newspaper

The Stem Cell Market report 2020 provides comprehensive analysis of Stem Cell industry by types, applications, regions. It shows Stem Cell market 2020-2024 by production, consumption, supply, gross margin, revenue of key players in the industry. Additionally, Stem Cell market report covers market size, share, and growth rate of industry with respect to regions.

Stem Cell market includes following leading manufacturers on the basis of sales, revenue, price, and gross margin.

Stem Cell market report presents in-depth analysis regarding the development, current trends, industry policies, and regulations implemented in each of the geographical regions. It includes analysis of upstream raw materials, downstream demand, and current market dynamics. Furthermore, the Stem Cell market report provides an in-depth insight into Stem Cell industry during 2020-2024.

Scope of the Report:

The scope of this market is limited to tracking the stem cell market. As per the scope of this report, stem cells are biological cells that can differentiate into other types of cells. Also, various types of stem cells are used for therapeutic purposes.

Inquire or Share Your Questions If Any Before the Purchasing This Report https://www.industryresearch.co/enquiry/pre-order-enquiry/13999718

Key Market Trends:

Oncology Disorders Segment is Expected to Exhibit Fastest Growth Rate Over the Forecast Period

Cancer has a major impact on society in the United States and across the world. As per the estimation of National Cancer Institute, in 2018, 1,735,350 new cases of cancer were anticipated to get diagnosed in the United States, and 609,640 deaths were expected from the disease. This increasing medical burden is due to population growth. Bone marrow transplant or stem cell transplant is a treatment for some types of cancers, like leukemia, multiple myeloma, multiple myeloma, neuroblastoma, or some types of lymphoma.

Embryonic stem cells (ESC) are the major source of stem cells for therapeutic purposes, due to their higher totipotency and indefinite lifespan, as compared to adult stem cells with lower totipotency and restricted lifespan. However, the use of ESCs for research and therapeutic purposes is restricted and prohibited in many countries throughout the world, due to some ethical constraints. Scientists from the University of California, Irvine, created the stem cell-based approach to kill cancerous tissue while preventing some toxic side effects of chemotherapy by treating the disease in a more localized way.

Although the market shows positive growth, due to the growing focus of stem cell-based research that can further strengthen the clinical application, its expensive nature for stem cell therapy may still hamper its growth.

North America Captured The Largest Market Share and is Expected to Retain its Dominance

North America dominated the overall stem cell market with the United States contributing to the largest share in the market. In 2014, the Sanford Stem Cell Clinical Center at the University of California, San Diego (UCSD) Health System, announced the launch of a clinical trial, in order to assess the safety of neural stem cell-based therapy in patients with chronic spinal cord injury. Researchers hoped that the transplanted stem cells may develop into new neurons that could replace severed or lost nerve connections, and restore at least some motor and sensory functions. Such numerous stem cell studies across the United States have helped in the growth of the stem cell market.

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Stem Cell Market Report Covers the Following Questions:

Detailed TOC of Stem Cell Market 2020-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Increased Awareness about Umbilical Stem Cell4.2.2 Increase in the Approval for Clinical Trials in Stem Cell Research4.2.3 Growing Demand for Regenerative Treatment Option4.2.4 Rising R&D Initiatives to Develop Therapeutic Options for Chronic Diseases4.3 Market Restraints4.3.1 Expensive Procedures4.3.2 Regulatory Complications4.3.3 Ethical and Moral Framework4.4 Industry Attractiveness- Porters Five Forces Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Product Type5.1.1 Adult Stem Cell5.1.2 Human Embryonic Cell5.1.3 Pluripotent Stem Cell5.1.4 Other Product Types5.2 By Therapeutic Application5.2.1 Neurological Disorders5.2.2 Orthopedic Treatments5.2.3 Oncology Disorders5.2.4 Diabetes5.2.5 Injuries and Wounds5.2.6 Cardiovascular Disorders5.2.7 Other Therapeutic Applications5.3 By Treatment Type5.3.1 Allogeneic Stem Cell Therapy5.3.2 Auto logic Stem Cell Therapy5.3.3 Syngeneic Stem Cell Therapy5.4 By Banking Service and Technology5.4.1 Stem Cell Acquisition and Testing5.4.2 Cell Production5.4.3 Expansion5.4.4 Sub-culture5.4.5 Cryopreservation5.5 By Type of Banking5.5.1 Public5.5.2 Private5.6 Geography5.6.1 North America5.6.1.1 US5.6.1.2 Canada5.6.1.3 Mexico5.6.2 Europe5.6.2.1 UK5.6.2.2 Germany5.6.2.3 France5.6.2.4 Italy5.6.2.5 Spain5.6.2.6 Rest of Europe5.6.3 Asia-Pacific5.6.3.1 China5.6.3.2 Japan5.6.3.3 India5.6.3.4 Australia5.6.3.5 South Korea5.6.3.6 Rest of Asia-Pacific5.6.4 Middle East & Africa5.6.4.1 GCC5.6.4.2 South Africa5.6.4.3 Rest of Middle East & Africa5.6.5 South America5.6.5.1 Brazil5.6.5.2 Argentina5.6.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Osiris Therapeutics Inc.6.1.2 Pluristem Therapeutics Inc.6.1.3 Thermo Fisher Scientific6.1.4 Qiagen NV6.1.5 Sigma Aldrich Corporation6.1.6 Becton, Dickinson and Company6.1.7 Stem Cell Technologies Inc.6.1.8 AllCells LLC6.1.9 Miltenyi Biotec6.1.10 International Stem Cell Corporation

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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Stem Cell Market 2020: Share Value Analysis of Top Key Players, Global Size, Consumption Analysis, Trends Forecast to 2024 | Industry Research.co -...

Compiled by Gabi Zietsman | Health24

04 Sep 2020, 02:45

Two hikers are going to great heights to increase awareness of blood disorders, and the urgent need for bone marrow donors in South Africa.

In a symbolic hike up the country's highest peak in the Drakensberg range, adventurer and bone marrow donor Clayton Coetzee and expert mountaineer and author Gavin Raubenheimer will be tackling Mafadi from 7 to 9 September in honour of those who have helped save the lives of countless people suffering from blood disorders.

Bone marrow donation is close to my heart as one of my dearest friends got sick with Acute Myeloid Leukaemia (AML) several years ago," says Coetzee. "Its then when I heard about the SA Bone Marrow Registry and the life-saving work that they do, and I decided to sign up as a donor."

READ | Could smoking lead to this blood disorder?

Increase in cases

Blood cancer is one of the most common of these disorders and affects children the most, while other blood disorders include non-Hodgkin lymphoma (NHL).

Unfortunately, according to the Search Coordinator for the SA Bone Marrow Registry (SABMR) Alicia Venter, these diseases have increased in the last decade 45% in NHL cases and 26% in leukaemia cases.

"While recommended, prevention efforts, such as lifestyle changes, tend to be less effective for hematologic malignancies than for other cancers, which makes a blood stem cell transplant a patients only hope for survival," says Venter.

In order to be a suitable bone marrow donor, your human leukocyte antigen (HLA) needs to match someone in need. HLAs are genes in a human's DNA that help regulate immunity and affect whether or not a recipient's body will reject a transplant.

READ MORE | Bone marrow transplants less risky now

Need more non-white donors

Finding a match, however, isn't as easy as swiping on a dating app and SAMBR has a serious lack of diversity in the donor database. Currently, there are only about 74 000 local donors on the South African Bone Marrow Registry.

In South Africa, there is a dire need for donors of colour," explains Venter.

"When it comes to matching HLA types, a patients ethnicity plays an important role as HLA markers are inherited. Some ethnic groups have more complex tissue types than others, therefore finding a match is most likely to come from someone of the same ethnic group."

'As easy as giving blood'

The hike is supposed to be symbolic of the uphill battle that faces those suffering from blood disorders, including finding a suitable donor to increase their chances of survival. Besides the gruelling experience, SAMBR will also be doing a cheek swab drive at the same time in Agulhas, Western Cape, and Musina, Limpopo, to help boost donor registrations.

According to Coetzee, it's easy to be registered as a donor all it takes is a cheek swab. If you do match with someone, it's "almost as easy as giving blood".

If a match is found, a donor will undergo a full medical exam to look for any exclusionary factors like obesity, HIV status, other chronic conditions and viral infections. Once cleared, the donor goes on a five-day treatment of injections to increase the number of stem cells in the bloodstream.

On the fifth day, the donor will be admitted to a hospital and connect to a cell separator machine, where the bone marrow donation would be made. The collected samples then has 72 hours to be transplanted to the receiving patient.

In South Africa, doctors will seldom collect bone marrow straight from the source.Possible side effects to receiving the injections may include headache, bone pain and flu-like symptoms.

I want people to know that blood diseases can affect anyone, regardless of ethnicity or gender. Extending beyond our boundaries or comfort zones like climbing Mafadi can be hard, but imagine the indelible difference the act of becoming a donor can have on someone elses life and their loved ones, says Coetzee.

You can follow the hikers' journey onSAMBR's Facebook page.

If you are between the ages of 18 and 45 and want to become a donor, contact the SABMR on021 447 8638 oremail:donors@sabmr.co.za.For more info and how to donate, visit their website.

READ MORE | Leukaemia survivor stories

Image credit: Pixabay

View original post here:
There is a dire need for 'donors of colour' in the fight against blood disorders - Health24

The parents of a girl battling a deadly blood disorder are begging people to join the stem cell donor register to save her life after her only match in the world pulled out at the last minute.

Evie Hodgson, eight, who suffers from aplastic anaemia, was due to have a bone marrow transplant this month but her donor backed out at the last possible moment.

Her mum, Tina, says the chances of finding another donor are so slim that doctors are now planning a different course of treatment. But, in future, a stem call transplant is Evies best hope of being cured.

The schoolgirl, from Whitby, North Yorks, was first taken to hospital with a rash and was diagnosed with aplastic anaemia in May.

After a global donor search was launched, a 10/10 match was found and the anonymous donor agreed to the procedure. In preparation, Evie had to have dental work and one of her ovaries was removed. But on August 14 the donor pulled out.

Tina, 37, who works at RAF Flyingdales, in Pickering, North Yorks, said: We were devastated, it was a huge blow. We have no idea why the donor changed their mind. Evie has already been through so much. She thought she had a donor and now she doesnt.

The donor pulling out is quite hard-hitting, but we want to raise awareness of the stem cell register. Its so easy to be a donor. Its just like giving blood, but you could save a childs life. Its so easy to join but only 1% of the UK population is registered.

Evie said: I need this transplant to save my life. Please sign the register to help.

Tina added: The condition Evie has is life-threatening. She wont survive without a transplant. We are desperately appealing for people to sign the stem cell register.

Evie was diagnosed with the condition after she developed a pin-prick rash on her back, which didnt fade. Tests revealed she had low blood platelet levels and she was told she needed a bone marrow transplant.

Aplastic anaemia is a rare life-threatening condition where the bone marrow fails to produce enough blood cells. Around 100-150 people are diagnosed in the UK each year.

Treatment can include immunosuppressants, chemotherapy, blood transfusions, or blood and bone marrow transplants.

Neither Tina, dad Andy, 49, or brother William, five, were a match and so an international search was launched.

Tina said: Our world crumbled when Evie was diagnosed. Evie knew shed need chemotherapy. She donated her hair to The Little Princess Trust, after making friends with poorly children who have lost all their hair.

Evie will be treated with immunosuppressants while the search for a donor continues.

See the original post:
Parents plea for stem cell help to save life of daughter with rare blood disorder - Mirror Online

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and Metagenomi, a gene editing company discovering breakthrough systems for curing genetic disease, today announced that Vor will evaluate the potential use of Metagenomis gene editing technology to develop engineered hematopoietic stem cell-based therapies for the treatment of blood cancers, such as acute myeloid leukemia.

Cancer patients deserve therapies with strong effects on cancer cells and minimal effects on all other cells, said Tirtha Chakraborty, Ph.D., Vors VP and Head of Research. Our new partnership with Metagenomi will help us achieve this goal by engineering hematopoietic stem cells using precise yet flexible gene editing thereby ensuring that targeted therapies can live up to their name."

The collaboration is non-exclusive and applies to pre-clinical research only. Further terms of the agreement are not being disclosed.

This partnership unites two transformative technologies our proprietary gene editing enzymes, and Vors platform for engineering hematopoietic stem cells such that they are inherently treatment-resistant, said Brian C. Thomas, Metagenomis CEO and co-founder. We are excited to be working together to bring both of these cutting-edge approaches into the clinic.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

About Metagenomi

Metagenomi is harnessing the vast information found in life on Earth to develop cures for genetic disease. Using proprietary data collected from around the world, Metagenomi has developed novel gene editing tools that enable next-generation gene and cell therapies.

Metagenomi is based out of Emeryville, California, and was founded by pioneers in the field of metagenomics, Jill Banfield and Brian C. Thomas. Metagenomi generates massive quantities of data from natural environments, producing complete genomes from organisms that are otherwise unknown. Metagenomi then unlocks the information captured in these genomes to develop game-changing in vivo and ex vivo therapeutics.

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Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies - Business Wire

A California woman may be the first person to be cured of HIV without a bone marrow transplant, according to a recent report in Nature. More than 60 other so-called elite controllers, who have unusually potent immune responses to HIV, were found to have their virus sequestered in parts of their genome where it is unable to replicate.

The unusual case involves Loreen Willenberg, who acquired HIV in 1992. Her immune system has maintained control of the virus for decades without the use of antiretroviral treatment, and researchers have been unable to find any intact virus in more than 1.5 billion of her cells. Elite controllers are thought to make up less than half a percent of all people living with HIV.

I believe Loreen might indeed meet anyones definition of a cure, study coauthor Steven Deeks, MD, of the University of California at San Francisco, told POZ. Despite heroic efforts, we just could not find any virus that is able to replicate. Her immune system seems completely normal. Even her HIV antibodies levels are low, which is unprecedented in an untreated person.

Although antiretroviral therapy can keep HIV replication suppressed, the virus inserts its genetic material into the chromosomes of human cells, making it very difficult to eradicate. HIV can lie dormant in a reservoir of resting immune cells indefinitely, but when antiretrovirals are stopped and the cells become activated, they can start churning out new virus.

Previously, only two people were known to have been cured of HIV: Timothy Ray Brown, formerly know as the Berlin Patient, and a man in London. Both received bone marrow stem cell transplants from a donor with a rare genetic mutation that makes cells resistant to HIV entry. But this procedure is far too dangerous for people who dont need it to treat advanced cancer.

The new research suggests that Willenberg and some five dozen other people with long-term untreated HIV have their virus hidden away in their cells genomes in such a way that the viral genetic blueprint (known as a provirus) cant be used to produce new viral particles that can go on to infect other cells.

Xu Yu, MD, of the Ragon Institute of Massachusetts General Hospital, MIT and Harvard analyzed integrated HIV in millions of cells from 64 elite controllers and 41 typical HIV-positive people on antiretroviral therapy recruited at Mass General and San Francisco General Hospital.

In both groups, about 20% were women, the average age was approximately 56 and they had been living with HIV for an average of 17 years and had undetectable virus according to standard tests for nine years. Overall, the elite controllers had a higher average CD4 count (about 900 versus 70, respectively).

The researchers used next-generation gene sequencing to characterize the participants viral blueprints, including where they were inserted into human chromosomes. They found that the elite controllers had fewer integrated proviruses, but a larger proportion of them were intact, or potentially capable of replicating. The virus in these individuals was highly consistent, without the wide variety of mutations seen in most people with HIV.

Whats more, their proviruses were integrated at distinct sites in the human genome, farther away from elements that enable viral replication. Specifically, the integrated DNA was not located near sites that switch on transcription or close to accessible chromatin, which contains histone proteins that package long DNA strands into a more compact form. The DNA must then be unwound from these proteins before it can be used to produce new virus.

These data suggest that a distinct configuration of the proviral reservoir represents a structural correlate of natural viral control and that the quality, rather than the quantity, of viral reservoirs can be an important distinguishing feature for a functional cure of HIV-1 infection, Yu and colleagues wrote.

In a commentary accompanying the report, Nicolas Chomont, PhD, of the University of Montreal, characterized the proviruses in these elite controllers as being in a state of deep sleep compared with latent virus in typical people with HIV. This has only now become apparent because researchers have more sophisticated tools to pinpoint the location of proviruses within the genome.

It is unclear why this block and lock phenomenon happens in only a small proportion of people with HIV. Its possible that the virus ends up sequestered in these locations by chance. But the researchers think its more likely that the integrated proviruses at these sites are evolutionarily selected over time as the ones in locations more conducive to viral replication are eliminated by the immune system.

In Willenbergs case, the research team analyzed more than 1.5billion of her peripheral blood immune cells, including samples from gut tissue, where the virus often hides. Theycould not find any intact proviruses that could be used to produce new HIV. Given her lack of intact proviruses, the researcherswere unable to determine whether she ever fit the pattern of having latent HIV locked away in inaccessible locations.

Another 11 people, dubbed exceptional controllers, only had detectable provirusesatremote sites in the genome where it could not replicate.Since this study, the researchers have discovered a couple more elite controllers who may qualify as additional cures, according to The New York Times.

This raises the possibility that a sterilizing cure of HIVmeaning complete eradicationmay be feasible in rare instances, the study authors suggested. A similar but less complete process may be at play in the subset of about 10% of people with HIV who maintain viral suppression after stopping antiretroviral therapy but who still have detectable proviruses (known as post-treatment controllers).

This research was first presented at the International AIDS Society Conference on HIV Science last summer, where Willenberg was referred to as the San Francisco Patient. Willenberg later went public with her status, and she and Yu discussed the study findings during a webinar with HIV cure advocates last November.

I broke out in tears when I saw Dr. Yus final slide, said Willenberg, who over the years has participated in more than a dozen studies. I can only hope and pray that with continued dedication we can figure out how I have dumped the virus into the DNA junkyard.

The question now is whether its possible to develop treatments that could enable the millions of typical people with progressive HIV to become elite controllers like Willenberg. Chomont suggested that immune-based therapiesincluding CAR-T cellsmight be able to shrink the viral reservoir until it consists only of deeply latent proviruses that are unable to replicate.

The key question is, How did her immune system achieve this remarkable state? Deeks said. We do not know. We need to find more people who are exceptional controllers like Loreen and get to work on figuring out the mechanism.

In this video fromamfAR, the Foundation for AIDS Research,Loreen Willenbergtalks about living as an elite controller of HIV.

Click here to read the Nature article.Click here for more news about HIV cure research.

Read the original:
First Woman May Be Cured of HIV Without a Bone Marrow Transplant - POZ

SAN DIEGO Cystinosis is a disease that slowly and aggressively attacks your organs, tissues, muscles, bones, eyes, even your brain.

It's a genetic disorder with no cure.

And right now, the only option for treatment is an army of pills to slow it down but missing even one dose can be devastating.

Now one man is "patient one", the first to try a new treatment that may save thousands of lives.

21-year old Jordan Janz is living with the rare, unrelenting disorder.

"I was diagnosed at eight months old and basically have been living with it my whole life."

In Jordan, cystine, an amino acid, gets trapped in his cells.

When cystine levels rise, crystals build up all over the body leaving a trail of damage... even causing him to vomit up to 13 times a day.

"It's not how strong you are physically," he said.

"I think it's how strong you are mentally when you come into this."

Traditional cystinosis treatments aim to slow the build up of cystine inside cells.

In order to do that Jordan takes 56 pills each day, but now he hopes to change that, Jordan is the first patient to test a unique gene therapy.

UC San Diego professor Stephanie Cherquie's took stem cells from Jordan's bone marrow, re-engineered the cells, introduced genes that will produce cystinosin, then reinfused Jordan with his own cystinosin-producing cells.

"So, then these cells become a source of healthy stem cells for the rest of the life of the patient," said Stephanie.

Jordan had to take chemo twice a day, but he hasn't let that scare him away.

"I'm doing this obviously for other cystinosis families, right?," said Jordan.

Hoping that many others after him will now get the chance at a better, longer life.

For those born with cystinosis who make it into adulthood, the average lifespan is around 28 years old.

We're told Jordan Janz is making a good recovery. though it is still too soon to tell his long-term prognosis.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens atjim.mertens@wqad.comor Marjorie Bekaert Thomas atmthomas@ivanhoe.com.

Continued here:
YOUR HEALTH: When the body turns to crystals - WQAD.com

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Researchers have found a way, in mice and human tissue, to regenerate the cartilage that eases movement between bones.

Loss of this slippery and shock-absorbing tissue layer, called articular cartilage, is responsible for many cases of joint pain and arthritis, which afflicts more than 55 million Americans.

The researchers can envision a time when people are able to avoid getting arthritis in the first place by rejuvenating their cartilage before it is badly degraded.

Nearly 1 in 4 adult Americans suffer from arthritis, and far more are burdened by joint pain and inflammation generally.

The researchers figured out how to regrow articular cartilage by first causing slight injury to the joint tissue, then using chemical signals to steer the growth of skeletal stem cells as the injuries heal.

Cartilage has practically zero regenerative potential in adulthood, so once its injured or gone, what we can do for patients has been very limited, says co-senior author Charles K.F. Chan, assistant professor of surgery at Stanford Universitys School of Medicine.

Its extremely gratifying to find a way to help the body regrow this important tissue, Chan says.

The work builds on previous research that resulted in isolation of the skeletal stem cell, a self-renewing cell that is also responsible for the production of bone, cartilage and a special type of cell that helps blood cells develop in bone marrow.

Articular cartilage is a complex and specialized tissue that provides a slick and bouncy cushion between bones at the joints. When this cartilage is damaged by trauma, disease, or simply thins with age, bones can rub directly against each other, causing pain and inflammation, which can eventually result in arthritis.

Damaged cartilage can be treated through a technique called microfracture, in which tiny holes are drilled in the surface of a joint. The microfracture technique prompts the body to create new tissue in the joint, but the new tissue is not much like cartilage.

I realized the only way to understand the process was to look at what stem cells are doing after microfracture.

Microfracture results in what is called fibrocartilage, which is really more like scar tissue than natural cartilage, says Chan. It covers the bone and is better than nothing, but it doesnt have the bounce and elasticity of natural cartilage, and it tends to degrade relatively quickly.

The most recent research arose, in part, through the work of surgeon and lead author Matthew Murphy, a visiting researcher at Stanford who is now at the University of Manchester.

I never felt anyone really understood how microfracture really worked, Murphy says. I realized the only way to understand the process was to look at what stem cells are doing after microfracture.

For a long time, Chan says, people assumed that adult cartilage did not regenerate after injury because the tissue did not have many skeletal stem cells that could be activated. Working in a mouse model, the team documented that microfracture did activate skeletal stem cells. Left to their own devices, however, those activated skeletal stem cells regenerated fibrocartilage in the joint.

But what if the healing process after microfracture could be steered toward development of cartilage and away from fibrocartilage?

The researchers knew that as bone develops, cells must first go through a cartilage stage before turning into bone. They had the idea that they might encourage the skeletal stem cells in the joint to start along a path toward becoming bone, but stop the process at the cartilage stage.

The researchers used a powerful molecule called bone morphogenetic protein 2 (BMP2) to initiate bone formation after microfracture, but then stopped the process midway with a molecule that blocked another signaling molecule important in bone formation, called vascular endothelial growth factor (VEGF).

What we ended up with was cartilage that is made of the same sort of cells as natural cartilage with comparable mechanical properties, unlike the fibrocartilage that we usually get, Chan says. It also restored mobility to osteoarthritic mice and significantly reduced their pain.

As a proof of principle that this might also work in humans, the researchers transferred human tissue into mice that were bred to not reject the tissue, and were able to show that human skeletal stem cells could be steered toward bone development but stopped at the cartilage stage.

The next stage of research is to conduct similar experiments in larger animals before starting human clinical trials. Murphy points out that because of the difficulty in working with very small mouse joints, there might be some improvements to the system they could make as they move into relatively larger joints.

The first human clinical trials might be for people who have arthritis in their fingers and toes. We might start with small joints, and if that works we would move up to larger joints like knees, Murphy says.

Right now, one of the most common surgeries for arthritis in the fingers is to have the bone at the base of the thumb taken out. In such cases we might try this to save the joint, and if it doesnt work we just take out the bone as we would have anyway. Theres a big potential for improvement, and the downside is that we would be back to where we were before.

One advantage of their discovery is that the main components of a potential therapy are approved as safe and effective by the FDA, says co-senior author Michael Longaker, professor of surgery.

BMP2 has already been approved for helping bone heal, and VEGF inhibitors are already used as anti-cancer therapies, he says. This would help speed the approval of any therapy we develop.

Joint replacement surgery has revolutionized how doctors treat arthritis and is very common: By age 80, 1 in 10 people will have a hip replacement and 1 in 20 will have a knee replaced. But such joint replacement is extremely invasive, has a limited lifespan and is performed only after arthritis hits and patients endure lasting pain.

The researchers say they can envision a time when people are able to avoid getting arthritis in the first place by rejuvenating their cartilage in their joints before it is badly degraded.

One idea is to follow a Jiffy Lube model of cartilage replenishment, Longaker says. You dont wait for damage to accumulateyou go in periodically and use this technique to boost your articular cartilage before you have a problem.

The work appears in the journal Nature Medicine.

Support for the research came from the National Institutes of Health, the California Institute for Regenerative Medicine, the Oak Foundation, the Pitch Johnson Fund, the Gunn/Olivier Research Fund, the Stinehart/Reed Foundation, The Siebel Foundation, the Howard Hughes Medical Institute, the German Research Foundation, the PSRF National Endowment, National Center for Research Resources, the Prostate Cancer Research Foundation, the American Federation of Aging Research, and the Arthritis National Research Foundation.

Source: Stanford University

Read more:
Method regrows cartilage to cushion bones - Futurity: Research News

Mesoblast's (MESO) stock has grown over 300% since we last made out an investment case in March 2019, and an IOM runthrough six months prior, based on phase 2 trial data in acute graft versus host disease (aGVHD). Presently, the United States Food and Drug Administration (FDA) has accepted the companys Biologics License Application (BLA) for priority review of its phase 3 product candidate RYONCIL (remestemcel-L) for steroid-refractory aGVHD in children. The Prescription Drug User Fee Act (PDUFA) action date is set at 9/30/2020, and if approved, Mesoblast is geared up to make RYONCIL available immediately in the U.S. mid-August, an FDA advisory committee has already backed approval with a 9-1 vote.

Speaking at the 2020 Full-Year Financial Results and Corporate Highlights Webcast on 8/27/2020, CEO Dr. Silviu Itescu said the lead product candidate RYONCIL has a targeted market. If approved, the launch for this product is planned in 2020, and we have industrial scale manufacturing in place to meet the commercial demand as we expect to move forward.

The company has well-established plans for life cycle expansion of remestemcel in pediatric and adult inflammatory diseases, including a Phase 3 trial ongoing in patients with acute respiratory distress syndrome from COVID-19, and two additional product candidates in Phase 3 trials for heart failure and back pain with near-term U.S. readouts.

Cash-wise, Mesoblast is presently in a comfortable position. It has sufficient runway for transitioning to a commercial organization with its own product sales.

Mesoblast uses a proprietary technology platform developing mesenchymal lineage cells that are found in every vascularized tissue around blood vessels. They express a number of receptors for various inflammatory cytokines, including TNF-alpha receptor, IL-1 receptor, IL-6 receptor, interferon gamma receptor. They are activated when introduced in the middle of a cytokine storm, and release anti-inflammatory mediators to regulate and control multiple arms of the immune system thereby switching off the severe inflammatory response that is responsible for the damage to tissues forming the basis of the diseases that the company is targeting.

Mesenchymal lineage cells are collected from the bone marrow of healthy adult donors and proprietary processes are utilized to expand them to a uniform, well characterized, and highly reproducible cell population. This enables manufacturing at industrial scale for commercial purposes. Another key feature of Mesoblasts cells is they can be administered to patients without the need for donorrecipient matching or recipient immune suppression. (from company website)

Remestemcel is the companys most advanced product candidate being developed for pediatric and adult systemic inflammatory conditions. The most advanced indication is for pediatric steroid-refractory aGVHD. It is also being developed for adult steroid-refractory aGVHD and chronic GVHD. A phase 3 trial is ongoing for acute respiratory distress syndrome (ARDS) caused by COVID-19. Its also being developed for biologic refractory Crohns disease.

Image source: company website

The companys products for localized inflammatory diseases are based on the rexlemestrocel platform. These include REVASCOR for advanced chronic heart failure and MPC-06-ID for chronic low back pain due to degenerative disc disease, which are in phase 3 with readouts expected in the upcoming quarter.

Trial data from the three phase 3 trials of RYONCIL for pediatric steroid-refractory aGVHD is summarized below. (Source: news release dated 5/25/2020.)

Three trials evaluated the same treatment regimen of RYONCIL, with patients receiving twice weekly intravenous infusions of 2 million cells per kg body weight over a four-week period.

Study 275: An Expanded Access Program in 241 children across 50 centers in eight countries where RYONCIL was used as salvage therapy for steroid-refractory acute GVHD in patients who failed to respond to steroid therapy as well as multiple other agents.

Study GVHD001/002: A Phase 3 single-arm trial in 55 children across 20 centers in the United States where RYONCIL was used as the first line of treatment for children who failed to respond to steroids for acute GVHD.

Study 280: A Phase 3 randomized placebo-controlled trial in 260 patients, including 28 children, across 72 centers in seven countries where RYONCIL or placebo were added to second line therapy in patients with steroid-refractory acute GVHD who failed to respond to steroid treatment.

Study 275: Day 28 Overall Response (OR), the primary endpoint, was achieved in 65% of subjects. Survival through 100 days was significantly greater in patients who achieved a day 28 OR (82%) compared with patients that did not achieve day 28 OR (39%), with 67% overall day 100 survival.

Study GVHD001/002:

Consistent with the findings in Study 275, Day 28 OR was achieved in 70% of children. This was statistically significant compared to the pre-specified control value of 45% (70.4% versus 45%, P =0.0003). As in study 275, clinical response at day 28 was highly predictive of improved survival through day 100 (87% compared to 47% in patients that did not achieve day 28 OR P = 0.0001). Similar predictive value of day 28 was also seen in survival through day 180 (79% vs. 43.8%, P= 0.003). Overall survival was 74.1% at day 100 and 68.5% at day 180.

These results were significantly higher than those from matched control pediatric subjects from the contemporaneous database of the Mount Sinai Acute GVHD International Consortium (MAGIC), accessed to provide an unbiased and independent estimate of response rates and outcomes in matched pediatric control patients treated with institutional standard of care. In the MAGIC controls, Day 28 OR was 43% and Day 100 survival was 57%.

Study 280:

Among high-risk children and adults who had the most severe disease stages, day 28 OR was significantly greater in the RYONCIL treated group (58% versus 37%; P = 0.03) compared to placebo. Among the standard risk patients there was no significant benefit of RYONCIL treatment. Within the pediatric patients in this study (n=28) day 28 OR was significantly greater in the RYONCIL group compared with the placebo group (64% vs. 36%, respectively, P=0.05).

These Phase 3 results provide prospective, randomized controlled data which are supportive for the use of RYONCIL in children and high-risk adults with steroid-refractory acute GVHD.

RYONCIL was well-tolerated in all studies with no identified safety concerns.

52-week range: $3.12-$21.28

Last close: $18.58 (8/27/2020)

Market cap.: $2.17B

Cash balance: $129.3M (Jun-2020)

Cash burn: $82.3M (FY-2020)

Cash runway: 1.57 excl. revenue; 3.01 incl. revenue

Revenue: ~$32.2M (Jun-2020); estimated: ~$119M (Jun-2021), ~$314M (Jun-2022)

The company raised $90 million in May 2020 from existing and new institutional investors. The proceeds from the offering will be used for commercial launch of RYONCIL for acute GVHD, will also support a scale-up of manufacturing for... maturing pipeline, including GVHD label extensions and for COVID-19 ARDS and also support the clinical programs underlying those. Additionally, up to $67.5 million may be available through existing strategic partnerships and ongoing financing facilities over the next 12 months.

The company has an extensive portfolio of over 1,100 patents and patent applications that cover compositions of matter, manufacturing and therapeutic applications of mesenchymal lineage cells with protection extending through 2040 in all major markets.

The company has licensing and royalty agreements with Japan based JCR Pharmaceuticals (OTCPK:JCRRF) for their product, TEMCELL, with Grunenthal for MPC-06-ID for the Europe and Latin America regions, and with Tasly for REVASCOR for the China region. The company garnered revenue of ~$32 million in fiscal 2020 from these arrangements.

Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing. In patients with the most severe form of acute GVHD (Grade C/D or III/IV), mortality is as high as 90% despite optimal institutional standard of care.

There are currently no FDA-approved treatments in the United States for children under 12 with steroid-refractory acute GVHD.

Mesenchymal Stem Cells (MSC) based therapy looks like the most promising of the various third-line treatment options in consideration, and is at the most advanced stage towards approval.

Since I first recommended MESO to our subscribers, the stock has multiplied many times over. Now, right before PDUFA, there does not seem to be much opportunity, especially since the positive adcom has probably priced in the PDUFA as well. Sure, there is always some spike before the major date, but I would rather buy after the approval, when stocks almost always go down. There may even be a dilution given the not-so-strong cash position of the company. But after that, the company predicts a steady revenue stream and so on; buyout offers are also not unlikely. Given that, this would be the right time to purchase the stock in my opinion.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Mesoblast Before The PDUFA - Seeking Alpha

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

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As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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Demand for Myelofibrosis Treatment Market to Witness Rapid Surge During the Period 2016 2022 - Scientect

Interim analysis to commence after 28 days; results anticipated by mid-October

VANCOUVER, Washington, Aug. 25, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn” or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today the Company has reached the requisite number of enrolled patients in its Phase 3 trial for COVID-19 patients with severe-to-critical symptoms to perform an interim analysis following the 28-day phase of the trial.

This Phase 3 trial is a two-arm, randomized, double blind, placebo controlled, adaptive design multicenter study to evaluate the safety and efficacy of leronlimab in patients with severe-to-critical symptoms of respiratory illness caused by COVID-19. Patients are randomized to receive weekly doses of 700 mg leronlimab or placebo, administered via weekly subcutaneous injection for two weeks. The study has three phases lasting 28 days: Screening Period, Treatment Period, and Follow-Up Period. The primary outcome measured in this study is: all-cause mortality at Day 28. Secondary outcomes measured are: (1) all-cause mortality at Day 14, (2) change in clinical status of subject at Day 14, (3) change in clinical status of subject at Day 28, and (4) change from baseline in Sequential Organ Failure Assessment (SOFA) score at Day 14. Recently, the Data Safety Monitoring Committee (DSMC”) completed its first safety review of patients in the Phase 3 trial and reported it saw no cause to modify the study. The DSMC reviewed safety data from 149 of the 169 patients enrolled at the time of their review. The DSMC did not raise any concerns regarding safety and recommended the trial continue as planned.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, stated, We are very thankful for the many clinicians and their staff who have worked tirelessly to advance enrollment this quickly and for their care of these seriously ill patients. We are eager to perform an interim analysis of the data and remain optimistic the interim results will be consistent with those experienced by patients who received leronlimab through multiple EINDs (over 60) previously authorized by the FDA. And, in the event we are successful, we are well positioned with our distribution partner to accelerate distribution of leronlimab to patients throughout the U.S.”

About Coronavirus Disease 2019 CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a randomized clinical trial for mild-to-moderate patients in the U.S. Enrollment continues in its Phase 3 randomized clinical trial for the severe-to-critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 is believed to typically transmit person-to-person through respiratory droplets. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

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

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

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

About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA has agreed to provide written responses to the Company’s questions concerning its recent Biologics License Application by September 4, 2020, in lieu of a Type A teleconference meeting for this HIV combination therapy.

CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years.

CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking Statements This press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes,” hopes,” intends,” estimates,” expects,” projects,” plans,” anticipates” and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Company’s forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Company’s cash position, (ii) the Company’s ability to raise additional capital to fund its operations, (iii) the Company’s ability to meet its debt obligations, if any, (iv) the Company’s ability to enter into partnership or licensing arrangements with third parties, (v) the Company’s ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Company’s ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Company’s clinical trials, (viii) the results of the Company’s clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Company’s products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Company’s control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTS Investors: Michael Mulholland Office: 360.980.8524, ext. 102 Mobile: 503.341.3514 mmulholland@cytodyn.com

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CytoDyn Reaches Enrollment of 195 Patients in its Phase 3 Trial for COVID-19 Patients with Severe-to-Critical Symptoms - Stockhouse

This article was originally published here

Stem Cell Rev Rep. 2020 Aug 22. doi: 10.1007/s12015-020-10033-6. Online ahead of print.

ABSTRACT

Therapeutic clinical and preclinical studies using cultured cells are on the rise, especially now that the World Health Organization (WHO) declared coronavirus disease 2019 (COVID-19) a public health emergency of international concern, in January, 2020. Thus, this study aims to review the outcomes of ongoing clinical studies on stem cells in Severe Acute Respiratory Syndrome (SARS), Acute Respiratory Distress Syndrome (ARDS), and Middle East Respiratory Syndrome (MERS). The results will be associated with possible applications to COVID-19. Only three clinical trials related to stem cells are considered complete, whereby two are in Phase 1 and one is in Phase 2. Basically, the ongoing studies on coronavirus are using mesenchymal stem cells (MSCs) derived from bone marrow or the umbilical cord to demonstrate their feasibility, safety, and tolerability. The studies not related to coronavirus are all in ARDS conditions; four of them are in Phase 1 and three in Phase 2. With the COVID-19 boom, many clinical trials are being carried out using different sources with an emphasis on MSC-based therapy used to inhibit inflammation. One of the biggest challenges in the current treatment of COVID-19 is the cytokine storm, however MSCs can prevent or mitigate this cytokine storm through their immunomodulatory capacity. We look forward to the results of the ongoing clinical trials to find a treatment for the disease. Researchers around the world are joining forces to help fight COVID-19. Stem cells used in the current clinical studies are a new therapeutic promise for COVID-19 where pharmacological treatments seem insufficient.Graphical Abstract.

PMID:32827081 | DOI:10.1007/s12015-020-10033-6

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Review of Trials Currently Testing Stem Cells for Treatment of Respiratory Diseases: Facts Known to Date and Possible Applications to COVID-19 -...

Neutrophils are the warriors of the immune system. They are always ready to spring to action to help heal injuries or fight off disease. Unless, that is, something goes wrong in their developmental process.Immature neutrophils arent all warriors they can be dangerous turncoats. High levels of immature neutrophils in the bloodstream can be a tell-tale sign of cancer and may even be a biomarker for COVID-19.

Now scientists at La Jolla Institute for Immunology (LJI) have tracked down the rare stem cells that generate neutrophils in human bone marrow. This research, published in Immunity, gives researchers a potential path for intervening in diseases where neutrophil development goes awry.

We have identified the stem cells that are the early origins of neutrophils, the most abundant blood cell type in humans, says Huy Dinh, Ph.D., a former LJI postdoctoral associate who recently moved to a faculty position at The University of WisconsinMadison. Dinh led the study with LJI Professor Catherine C. Hedrick, Ph.D. Knowing how human neutrophils develop is especially relevant today because immature neutrophils have been found to be elevated in both the blood and lungs of severe COVID-19 patients.

Despite their importance, neutrophils have proven very hard to study. They dont hold up well outside the body, and the stem cells that make them are even harder to investigate because they only live in bone marrow.

In 2018, the Hedrick Lab reported the discovery of a group of progenitor stem cells that give rise to mature neutrophils. These progenitors sole job was to generate neutrophils, yet they appeared to also promote tumor growth. The researchers believed that detecting these progenitors could give doctors a better way to catch early cancer cases. But first, the team needed to know a lot more about neutrophil development.

The new research revealed a progenitor cell type that exists even earlier in human neutrophil development. Dinh, a past SPARK Award recipient, together with Tobias Eggert, Ph.D., a LJI visiting scientist and Melissa Meyer, Ph.D., a LJI postdoc, who served as the co-first authors in the study, spearheaded the effort to use a tool called cytometry by time-of-flight (CyTOF) to distinguish these rare cells from other types of immune progenitor cells. This work also made it possible for the researchers to identify more specific protein markers on this early progenitor cell surface.

The discovery of these protein markers was important because until now, scientists have used only a few of markers to track neutrophils over time. The new study gives scientists specific markers for tracking neutrophil development from day one.

The researchers also found that cases of skin and lung cancers are often accompanied by a flood of immature neutrophils including the early progenitor cells into the bloodstream. These immature neutrophils change as they interact with tumor cells, though the researchers arent sure yet how these changes affect cancer progression.

Dinh likens the stages of neutrophil development to the cars on a train. The early progenitors are like the train engine, keeping everything going smoothly along the track to maturity. Cancer shakes everything up, and immature neutrophils jump off the track before they reach maturity. Its like the train is falling apart, Dinh says.

Neutrophil development has been in the news recently due to the COVID-19 pandemic, as studies have shown immature neutrophils are also more abundant in some patients with COVID-19. Dinh and Hedrick think perhaps the threat of the virus prompts the body to churn out neutrophils too quickly, again forcing immature cells off the track to maturity.

We need to study this phenomenon further to see if these neutrophils can be tied to case prognosis or if they can be a drug target for COVID-19, says Dinh.

The researchers hope to continue their work to discover the exact mechanisms that stop neutrophils from reaching maturity. Knowing the earliest cell that gives rise to neutrophils is really critical for trying to target and control these cells, says Hedrick. But we dont know exactly how to do that yet.

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.

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Researchers Get First-Ever Look at a Rare but Vital Stem Cell in Humans - Technology Networks