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Be a Bone Marrow Hero – University of Utah Health Care

By daniellenierenberg

Feb 25, 2020 12:00 AM

Every ten minutes, someone passes away from a blood disorder. Thats 148 people a day. There is a way to prevent many of these deathsa bone marrow transplant. DNA matching has the power to help thousands of people waiting for a life-saving bone marrow donation, but this special donor list depends entirely upon the willingness of individuals to sign up. Could your unique DNA hold the match that helps one person live to see tomorrow? Heres how you can find out.

Be The Match is a global hub for bone marrow donor registry working with hundreds of partners to support the transplant community. Signing up is easy online. You provide registration information, receive a kit in the mail, use the DNA swab as directed, and send it back for DNA typing. Your potentially life-saving information is secure and becomes available to specialized doctors around the world.

Even if you arent a match right away, the fact that every three minutes a person is diagnosed with a blood disorder means you could be called at any time to be a hero in someones time of need. Paloma Cariello, MD, MPH, says, Its absolutely a life-saving procedure. Its a new life that people getwe call it a new birthday, and at many hospitals they give it as a new birthday date in their chart. We sing Happy Birthday. Its a big event.

To find a close enough match to help fortify a patients immune system, doctors have to be precise. They first reach out to family, but even then, only 30% of patients find a good match. The odds of finding a match in an unrelated donor can be as low as 18%, especially with minorities.

The need for more individuals of every background cannot be overstated, says University of Utah Health Hematologist Sagar Patel, MD. He emphasizes the need for ethnic minorities to register. Every ethnicity is represented in the pool of patients, so the donor pool likewise needs to be diversified to improve the availability of similar DNA typing.

If a doctor finds you to be a suitable match, they select the ideal method for their patient and prepare you for donation. There are two donation methods: peripheral blood stem cell (PBSC) donation and bone marrow donation. Because every donor is carefully screened and prepared, and because a small amount of fluid is ultimately needed, neither procedure method impacts the performance of your own immune system, says Cariello.

With PBSC retrieval, you receive a stimulant for five days to increase the presence of blood-forming cells in your blood stream. Then a refined process of extraction occurs: Your blood is drawn, a machine collects just the cells the patient needs, and your remaining fluids are safely returned to you. This process can usually be done in one eight-hour session. Most donors report a full recovery within a week to 10 days, but you will be followed-up with until your full recovery.

If the doctor determines that the patient needs bone marrow, your procedure is a bit different. Marrow needs to be drawn from your pelvic bones. It happens in a hospital and under anesthesia, and you will feel no pain as the donation is collected. You can go back to routine activity the same day, and your system fully replenishes within four to six weeks.

Even with thousands of people in need, only about one in 430 donors in the Be The Match system are called in as a match. And the simple processes and expert professional care you receive minimize potential risk. A common side effect is bruising at the procedure sites, and some donors occasionally experience mild pain, fatigue, or dizziness. Reactions related to the use of anesthesia might also occur.

With such little risk, it shouldnt be a question as to whether you sign up, but when. And today is a perfect day. The low odds of finding a cure that these patients face are as extreme as the high rewards that await themand youwhen you make the choice to become a donor. Visit BeTheMatch.org to learn more and to become the one who initiates the miraculous call: We found a match.

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YOUR HEALTH: Knee relief can be found in the womb – WQAD.com

By daniellenierenberg

CHICAGO About 54 million Americans suffer from the aches and pains of arthritis.

Treatments range from pain medications to injections to surgery.

None of it seemed to work for 77-year-old Marty Ciesielczyk.

And it jeopardized something he loved: jogging.

"For me, it's just enjoyable, and if you're not a runner, then you would have no idea what I'm talking about."

But Marty's active lifestyle was in jeopardy when knee pain took over.

"When you got to lay on the floor to get dressed, it's tough."

It happens when there's a loss of cartilage in the joint.

"It's like a tire, and as you slowly lose rubber on the tire, it wears away," explained Dr. Adam Yanke, a surgeon with Midwest Orthopaedics at Rush University.

"You might need to have the tire replaced at some point."

Marty's arthritis was too advanced for a scope procedure but not bad enough for a joint replacement.

So he enrolled in a study testing whether amniotic fluid, which surrounds a growing baby in the uterus, could help his pain.

"Amniotic products come from patients that are having healthy, elective C-sections, and they choose to donate these products at the time of the delivery," said Dr. Yanke.

It's thought to increase tissue healing and lower inflammation.

Doctor-diagnosed arthritisis more common in womenthan in men. Arthritis and other joint disorders are among the five most costly conditions among adults 18 and older.

Your bone marrow makes mesenchymal stem cells, or MSCs. They are known to grow into new tissues, including cartilage.

By gathering these cells and injecting them into the knee joint, the hope is that they will give growth to new cartilage and reduce inflammation.

Marty received a placebo during the study, but then chose to have the amniotic fluid when the study ended.

"I mean I didn't care if it was Pixie dust, as long as my knee was going to feel better."

He went from not being able to get dressed to jogging about a week after having the injection.

"This morning, I ran three, three miles, and I had no problem at all."

Amniotic fluid is also being used to treat ulcers in the eye.

Rush University will be enrolling patients for a larger follow-up study on amniotic fluid for joint pain in the future.

Clinical trialsare still going on and most studies are still early.

A review published in 2016 in BMC Musculoskeletal Disorders concluded that MSC-based therapies offer an "exciting possibility" for treatment, but further studies need to be done on how they can best be used and how well they work.

They are also known to be very expensive.

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.

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Mum meets the stem cell donor who saved her four-year-old son’s life – Nottinghamshire Live

By daniellenierenberg

A mum has shared the emotional experience of meeting the stranger who saved her son's life.

Alfie Commons, now aged four, was just seven months old when he was diagnosed with leukaemia in 2016.

After three rounds of chemotherapy failed, Alfie received a life-saving bone marrow donation from a school teacher in Germany, who recently made the trip to the UK to meet him.

Alfie's mum, Lorna Commons, 40, of Toton has spoken about the experience in the hope it will encourage more people to sign up to become potential donors.

Looking back to the day of diagnosis, she said Alfie had been to his GP for a third time in February 2016 after suffering a cold since Christmas.

She said: "The GP told us to go to A&E for further tests as he was a little concerned.

"We got to Queen's Medical Centre in the morning and by early evening, we had the diagnosis; Alfie had infant acute lymphoblastic leukaemia (ALL)."

Ms Commons, who works in HR, added: "Even now, four years down the line, I still feel the emotions of that day. Nothing can prepare you."

The plan was to treat Alfie with chemotherapy, but after the first round failed, Ms Commons was told his only chance of survival was to get a bone marrow transplant.

The family was told Alfie was unlikely to leave hospital for the next six months.

She added: "Worse was to follow, his second course also failed and on the same day, we were told that Alfies nine-year-old brother, Billy, wasnt a bloodstemcellmatch for him either.

"The fear of losing Alfie was overwhelming, I felt helpless but I had to carry on for Alfies sake.

The transplant could not go ahead without the cancer being near enough eradicated and even when the good news came that a donor had been located, Alfie still had a mountain to climb.

After a third failed round of chemotherapy, Alfie was put on a trial immunotherapy drug as a '"last ditch attempt". Against all the odds, it worked.

"I think at that point all the doctors and nurses were preparing us for the worst. Your head has to go there," Ms Commons said.

"But then the cancer went, and it was enough to give us the bridge to getting the transplant done."

While the transplant was a success, Alfie suffered for months with Graft versus Host Disease (GvHD) on his skin and in his gut, which is the body's reaction to the new stem cells.

However, doctor's were encouraged the body was gradually accepting the cells and beginning to produce cells of their own.

On February 19, Alfie and his mum were able to meet the woman who saved his life after she made the 600-mile trip.

Christin Bouvier, 34, from Schwerin in Germany, was matched with Alfie after she registered in 2010 with DKMS, a charity dedicated to the fight against blood cancer.

The school teacher had been on the bloodstemcellregister for a number of years before she was contacted and tested as a match for Alfie.

Ms Bouvier said: When they told me that the recipient was a baby I just cried.

"Its a moment that is always with me and whenever I feel a bit down, I think back to it as it always brings me so much happiness!"

Ms Commons said she had been able to contact Ms Bouvier anonymously, as per UK law, but they were permitted to meet two years after the transplant.

Ms Bouvier added: It was always a dream to meet Lorna and Alfie and I never thought it would happen I was so delighted when Lorna invited me. I was very nervous but also very excited to meet them both in person.

"I knew the meeting would be one of those very special moments in my life."

Ms Commons feels the meeting has meant a new chapter has begun in both hers and Alfie's life and she is now focussed on the positives.

She added: "For something so small, there really is no greater gift than being a donor - I get to see my child grow up. To meet Christin, I was able to say 'this is what you've done'.

"We will be in each other's lives forever now - Alfie has her DNA in his blood. But Christin and I also share a special bond, we're just so similar and some people say we even look like sisters.

Alfie is such a special little boy and I truly believe that this story can make a real difference and save more lives.

"There is a match out there for everyone with blood cancer, people just need to come forward and register."

Anyone aged between 17-55 and in general good health can go on standby as a potential lifesaver.

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Humans soon able to regrow spines as body given ‘new power to heal itself’ – Daily Star

By daniellenierenberg

Scientists believe that humans will soon be able to recover from injuries such as broken spines, as treatment looks to boost the body's ability to heal itself.

A new study in the journal Regenerative Medicine describes how scientists were able to stimulate the self-repair response in rats.

Rats in the study were given two drugs, one of which is usually given to bone marrow transplant patients, and another which is used for bladder control.

This cocktail caused the rats' bone marrow to produce a greater number of mesenchymal stem cells, the cells which can develop into bone tissue.

As a result, enhanced calcium binding was seen at the site of the rats' spinal injuries, speeding up the production of new bone as well as healing wounds.

The study's authors hope that one day, such treatments will work on humans.

"We know that when bones break they will heal, and this requires the activation of stem cells in the bone," study co-author Sara Rankin from the National Heart and Lung Institute at Imperial College London, said in a statement.

"However, when the damage is severe, there are limits to what the body can do of its own accord.

"We hope that by using these existing medications to mobilise stem cells, as we were able to do in rats in our new study, we could potentially call up extra numbers of these stem cells, in order to boost our bodies' own ability to mend itself and accelerate the repair process."

Both drugs tested on rats are already widely used, so researchers are hopeful human trails can begin soon.

If these trials produce the same results as those seen in rats, then it's hoped the treatment could help to not only repair spinal injuries but also speed up the rate at which broken bones heal and mend damaged tissues in other organs.

Dr Tariq Fellous, first author of the research, said: "We first need to see if these medications release the stem cells in healthy volunteers before we can test them in patients with fractures.

"We have the drugs and know they are safe to use in humans we just need the funding for the human trials."

Dr Andia Redpath, who also co-authored the paper, added that repurposing existing medicines - so-called Regenerative Pharmacology - could have major potential as an efficient and cheaper way of treating diseases.

"Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the body's own stem cells, using existing drugs.

"We already know the treatments in our study are safe, it's now just a matter of exploring further if they help our bodies heal."

Stem cells are providing incredible new medical breakthroughs all the time.

Earlier this month, scientists trialled 3D-printed skin containing stem cells to treat burns victims .

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On the Road to 3-D Printed Organs – The Scientist

By daniellenierenberg

For years, scientists have predicted that 3-D printingwhich has been used it to make toys, homes, scientific tools and even a plastic bunny that contained a DNA code for its own replicationcould one day be harnessed to print live, human body parts to mitigate a shortage of donor organs. So far, researchers also used 3-D printing in medicine and dentistry to create dental implants, prosthetics, and models for surgeons to practice on before they make cuts on a patient. But many researchers have moved beyond printing with plastics and metalsprinting with cells that then form living human tissues.

No one has printed fully functional, transplantable human organs just yet, but scientists are getting closer, making pieces of tissue that can be used to test drugs and designing methods to overcome the challenges of recreating the bodys complex biology.

A confocal microscopy image showing 3-Dprinted stem cells differentiating into bone cells

The first 3-D printer was developed in the late 1980s. It could print small objects designed using computer-aided design (CAD) software. A design would be virtually sliced into layers only three-thousandths of a millimeter thick. Then, the printer would piece that design into the complete product.

There were two main strategies a printer might use to lay down the pattern: it could extrude a paste through a very fine tip, printing the design starting with the bottom layer and working upward with each layer being supported by the previous layers. Alternatively, it could start with a container filled with resin and use a pointed laser to solidify portions of that resin to create a solid object from the top down, which would be lifted and removed from the surrounding resin.

When it comes to printing cells and biomaterials to make replicas of body parts and organs, these same two strategies apply, but the ability to work with biological materials in this way has required input from cell biologists, engineers, developmental biologists, materials scientists, and others.

So far, scientists have printed mini organoids and microfluidics models of tissues, also known as organs on chips. Both have yielded practical and theoretical insights into the function of the human body. Some of these models are used by pharmaceutical companies to test drugs before moving on to animal studies and eventually clinical trials. One group, for example, printed cardiac cells on a chip and connected it to a bioreactor before using it to test the cardiac toxicity of a well-known cancer drug, doxorubicin. The team showed that the cells beating rate decreased dramatically after exposure to the drug.

However, scientists have yet to construct organs that truly replicate the myriad structural characteristics and functions of human tissues. There are a number of companies who are attempting to do things like 3-D print ears, and researchers have already reported transplanting 3-D printed ears onto children who had birth defects that left their ears underdeveloped, notes Robby Bowles, a bioengineer at the University of Utah. The ear transplants are, he says, kind of the first proof of concept of 3-D printing for medicine.

THE SCIENTIST STAFF

Bowles adds that researchers are still a ways away from printing more-complex tissues and organs that can be transplanted into living organisms. But, for many scientists, thats precisely the goal. As of February 2020, more than 112,000 people in the US are waiting for an organ transplant, according to the United Network for Organ Sharing. About 20 of them die each day.

For many years, biological engineers have tried to build 3-D scaffolds that they could seed with stem cells that would eventually differentiate and grow into the shapes of organs, but to a large extent those techniques dont allow you to introduce kind of the organization of gradients and the patterning that is in the tissue, says Bowles. There is no control over where the cells go in that tissue. By contrast, 3-D printing enables researchers with to very precisely direct the placement of cellsa feat that could lead to better control over organ development.

Ideally, 3-D printed organs would be built from cells that a patients immune system could recognize as its own, to avoid immune rejection and the need for patients to take immunosuppressive drugs. Such organs could potentially be built from patient-specific induced pluripotent stem cells, but one challenge is getting the cells to differentiate into the subtype of mature cell thats needed to build a particular organ. The difficulty is kind of coming together and producing complex patternings of cells and biomaterials together to produce different functions of the different tissues and organs, says Bowles.

To imitate the patterns seen in vivo, scientists print cells into hydrogels or other environments with molecular signals and gradients designed to coax the cells into organizing themselves into lifelike organs. Scientists can use 3-D printing to build these hydrogels as well. With other techniques, the patterns achieved have typically been two-dimensional, Eben Alsberg, a bioengineer at the University of Illinois, tells The Scientist in an email. Three-dimensional bioprinting permits much more control over signal presentation in 3D.

So far, researchers have created patches of tissue that mimic portions of certain organs but havent managed to replicate the complexity or cell density of a full organ. But its possible that in some patients, even a patch would be an effective treatment. At the end of 2016, a company called Organovo announced the start of a program to develop 3-D printed liver tissue for human transplants after a study showed that transplanted patches of 3-D printed liver cells successfully engrafted in a mouse model of a genetic liver disease and boosted several biomarkers that suggested an improvement in liver function.

Only in the past few years have researchers started to make headway with one of the biggest challenges in printing 3-D organs: creating vasculature. After the patches were engrafted into the mouses liver in the Organovo study, blood was delivered to it by the surrounding liver tissue, but an entire organ would need to come prepared for blood flow.

For any cells to stay alive, [the organ] needs that blood supply, so it cant just be this huge chunk of tissue, says Courtney Gegg, a senior director of tissue engineering at Prellis Biologics, which makes and sells scaffolds to support 3-D printed tissue. Thats been recognized as one of the key issues.

Mark Skylar-Scott, a bioengineer at the Wyss Institute, says that the problem has held back tissue engineering for decades. But in 2018, Sbastian Uzel, Skylar-Scott, and a team at the Wyss Institute managed to 3-D print a tiny, beating heart ventricle complete with blood vessels. A few days after printing the tissue, Uzel says he came into the lab to find a piece of twitching tissue, which was both very terrifying and exciting.

For any cells to stay alive, [the organ] needs that blood supply, so it cant just be this huge chunk of tissue.

Courtney Gegg, Prellis Biologics

Instead of printing the veins in layers, the team used embedded printinga technique in which, instead of building from the bottom of a slide upwards, material is extruded directly into a bath, or matrix. This strategy, which allows the researchers to print free form in 3-D, says Skylar-Scott, rather having to print each layer one on top of the other to support the structure, is a more efficient way to print a vascular tree. The matrix in this case was the cellular material that made up the heart ventricle. A gelatin-like ink pushed these cells gently out of the way to create a network of channels. Once printing was finished, the combination was warmed up. This heat caused the cellular matrix to solidify, but the gelatin to liquify so it could then be rinsed out, leaving space for blood to flow through.

But that doesnt mean the problem is completely solved. The Wyss Institute teams ventricle had blood vessels, but not nearly as many as a full-sized heart. Gegg points out that to truly imitate human biology, an individual cell will have to be within 200 microns of your nearest blood supply. . . . Everything has to be very, very close. Thats far more intricate than what researchers have printed so far.

Due to hurdles with adding vasculature and many other challenges that still face 3-Dprinted tissues, laboratory-built organs wont be available for transplant anytime soon. In the meantime, 3-D printing portions of tissue is helping accelerate both basic and clinical research about the human body.

Emma Yasinski is a Florida-based freelance reporter. Follow her on Twitter@EmmaYas24.

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Progenitor Cell Product Market Report by Manufacturers, Regions, Type and Application Forecast 2019 2026 – News Times

By daniellenierenberg

Global Progenitor Cell Product Market research report gives a comprehensive outlook of the markets 2019-2026 and offers an in-depth summary of the current market status, historic, and expected way forward for the Progenitor Cell Product Market. Additionally, to this, the report provides data on the restraints negatively impacting the markets growth. The report includes valuable information to assist new entrants, as well as established players, to understand the prevailing trends in the Market.

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Key Objectives of Progenitor Cell Product Market Report: Study of the annual revenues and market developments of the major players that supply Progenitor Cell Product Analysis of the demand for Progenitor Cell Product by component Assessment of future trends and growth of architecture in the Progenitor Cell Product Market Assessment of the Progenitor Cell Product Market with respect to the type of application Study of the market trends in various regions and countries, by component, of the Progenitor Cell Product Market Study of contracts and developments related to the Progenitor Cell Product Market by key players across different regions Finalization of overall market sizes by triangulating the supply-side data, which includes product developments, supply chain, and annual revenues of companies supplying Progenitor Cell Product across the globe

Major Players included in this report are as follows NeuroNova ABStemCellsReNeuron LimitedAsterias BiotherapeuticsThermo Fisher ScientificSTEMCELL TechnologiesAxol BioR&D SystemsLonzaATCCIrvine ScientificCDI

Progenitor Cell Product Market can be segmented into Product Types as Pancreatic progenitor cellsCardiac Progenitor CellsIntermediate progenitor cellsNeural progenitor cells (NPCs)Endothelial progenitor cells (EPC)Others

Progenitor Cell Product Market can be segmented into Applications as Medical careHospitalLaboratory

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Progenitor Cell Product Market: Regional analysis includes: Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia) Europe (Turkey, Germany, Russia UK, Italy, France, etc.) North America (United States, Mexico, and Canada.) South America (Brazil etc.) The Middle East and Africa (GCC Countries and Egypt.)

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Report structure: In the recently published report, UpMarketResearch.com has provided a unique insight into the Progenitor Cell Product Industry over the forecasted period. The report has covered the significant aspects which are contributing to the growth of the global Progenitor Cell Product Market. The primary objective of this report is to highlight the various key market dynamics listed as drivers, trends, and restraints.

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UpMarketResearch has offered a comprehensive analysis of the Progenitor Cell Product industry. The report has provided crucial information about the elements that are impacting and driving the sales of the Progenitor Cell Product Market. The section of competitive landscape keeps utmost importance in the reports published by UpMarketResearch. Competitive landscape section consists of key market players functioning in the worldwide industry of Progenitor Cell Product.

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Table of Contents 1 Industry Overview of Progenitor Cell Product 2 Manufacturing Cost Structure Analysis 3 Development and Manufacturing Plants Analysis of Progenitor Cell Product 4 Key Figures of Major Manufacturers 5 Progenitor Cell Product Regional Market Analysis 6 Progenitor Cell Product Segment Market Analysis (by Type) 7 Progenitor Cell Product Segment Market Analysis (by Application) 8 Progenitor Cell Product Major Manufacturers Analysis 9 Development Trend of Analysis of Progenitor Cell Product Market 10 Marketing Channel 11 Market Dynamics 12 Conclusion 13 Appendix

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD – Yahoo Finance

By daniellenierenberg

NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute 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.1 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.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

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

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

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD - Yahoo Finance

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Provides An In-Depth Insight Of Sales Analysis -Regenexx, Genzyme – Fashion…

By daniellenierenberg

Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020-2025 Research Report is spread throughout 100+ pages and offers exclusive important statistics, informative data, key traits and competitive landscape details on this area of interest sector.

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U.S. STEM CELL, INC.Brainstorm Cell TherapeuticsCytoriDendreon CorporationFibrocellLion BiotechnologiesCaladrius BiosciencesOpexa TherapeuticsOrgenesisRegenexxGenzymeAntriaRegeneusMesoblastPluristem Therapeutics IncTigenixMed cell EuropeHolostemMiltenyi Biotec

By Types:

Embryonic Stem CellResident Cardiac Stem CellsAdult Bone MarrowDerived Stem CellsUmbilical Cord Blood Stem Cells

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Neurodegenerative DisordersAutoimmune DiseasesCancer and TumorsCardiovascular Diseases

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Doctors Are Injecting This Naturally-Derived Substance to Restore Hair Thicknessand Its Not PRP – NewBeauty Magazine

By daniellenierenberg

The most emotional issue my patients have is hair loss, says New York dermatologist Cheryl Karcher, MD below a jaw-dropping before-and-after photo shared to her Instagram page. On the left half of the photo shared is a young womans exposed hairlinethe hair is so thin and sparse, the entire scalp is visible wherever your eye is drawn. On the right side of the photo, the same woman, but with an almost unbelievable amount of thicker hair, and, somehow, a sense of renewed confidence.

The secret? A little thing called nanofat.

In the past we only had PRP to offer that had to be done three times or more. Sometimes it would work, sometimes it didnt. Now we have nanofat hair restoration, which needs to be done just once, and is much more effective way to treat hair loss and grow hair, explains Dr. Karcher.

You May Also Like: How Low Level Laser Therapy Actually Works to Thicken Hair

So what is nanofat? According to Dr. Karcher, its derived from our own adipose tissue, whereas the ever popular PRP is derived from our blood. Nanofat includes adipose-derived stromal vascular fraction, which contains stem cells as well as growth factors. PRP contains the growth factors released from platelets in the blood, she adds. The procedure itself involves extracting anywhere from 20 to 40 millilitersof fat, usually from the abdomen, then processing it through mechanical filters, before injecting.

Like PRP, the possibilities of what nanofat can help with doesnt stop at the hairline. After the nanofat is processed to the point where there is no fat left, only stem cells and growth factors, it is injected into the scalp, the face, the neck, the decollete, or to improve sun damage, skin pigmentation, decrease wrinkles, and of course grow hair, says Dr. Karcher.

When nanofat is used for hair restoration, Dr. Karcher says she first injects the nanofat, then injects the patients PRP on top of it to act as a fertilizer for the nanofat. Perhaps the best part? Theres little to no painDr. Karcher says the most pain patients feel is during the PRP injections, so the scalp is numbed topicallyand no downtime. When nanofat is used on the face, chest or other areas, Dr. Karcher warns there may be some downtime of erythema and swelling or bruising. If injected for [skin] rejuvenation via microneedling the downtime is only about 48 hours.

While Dr. Karcher has seen unparalleled results from nanofat hair restoration, it is only ideal for patients who have some hair still present on the scalppatients who are completely bald may not be ideal candidates for the procedure. The only time I ever use PRP for hair restoration now is in a patient that doesnt have enough fat to harvest. The nanofat is just one treatment and the results seem to be superior. However, as La Jolla, CA plastic surgeon Robert Singer, MD notes, there is no safety or efficacy data surrounding nanofat treatment as of press time.

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Here are all the science projects that SpaceX will deliver to the ISS – Digital Trends

By daniellenierenberg

In a couple of weeks, SpaceX will be launching a Dragon cargo spacecraft bound for the International Space Station (ISS), carrying not only supplies for the astronauts but also a range of scientific equipment and research technology. The cargo includes tools for researching everything from growing human heart cells to making more comfortable sneakers.

One of the largest additions to the ISS will be the Bartolomeo facility, a European Space Agency project to provide more room for scientific experiments by attaching to the outside of the space station. Potential uses for the extended space include Earth observation, robotics, material science, and astrophysics, according to NASA.

Other projects include one by Adidas to test out its molding process in which thousands of pellets are blown together until they fuse, creating a midsole for shoes to make them more cushioned for high-performance athletes. Theres also a study into how water droplets form in low gravity which could help reduce the amount of water used by showers here on Earth, assisting the important project of water conservation. And theres a project to test improvements in 3D printing which could be used to print spare parts and repair tools for future space voyages.

Finally, there are also two biomedical experiments being taken to the ISS. One will look at how microgravity affects biotechnology like the Organ Chip which simulates the responses of human tissue on a small chip. And the other will investigate whether it is possible to grow human heart cells from stem cells in microgravity. The researchers believe the development of these heart cells could eventually be used to treat cardiac problems here on Earth, especially among children as their cardiac issues are particularly hard to treat.

The mission is scheduled to launch at 10:45 p.m. PT on Sunday, March 1, from Space Launch Complex 40 at Cape Canaveral Air Force Station in Florida. This will be the 20th mission as part of NASAs Commercial Resupply Services contract, in which private companies like SpaceX and Boeing take over some duties for delivering supplies to the ISS.

In the future, SpaceX will be taking a larger part in ISS operations as well. It will be delivering astronauts to and from the space station as part of NASAs Commercial Crew program, using its Crew Dragon capsule. The first manned Crew Dragon mission is targeted for May 7.

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Breakthrough, Non-Invasive Treatment Called Axol Therapy For Erectile Dysfunction And Enhanced Sexual Performance Now Available At Colorado Urology -…

By daniellenierenberg

- Axol Softwave Therapy is a new treatment for erectile dysfunction (ED) and for men who want enhanced sexual performance

- The in-office treatment is non-invasive, safe, and effective with virtually no side effects

- Axol Therapy uses low-intensity sound waves

- Axol Therapy is an alternative to ED medications, surgical implants, penile pumps, and injections

DENVER, Feb. 24, 2020 /PRNewswire/ -- The men's sexual health specialists at Colorado Urology now offer an exciting new treatment option for men living with erectile dysfunction (ED) called Axol Softwave Therapy. This safe and non-invasive treatment option is helping many men with ED achieve spontaneous and natural erections without the help of medications. The therapy can also be used to enhance a man's sexual performance.

Colorado Urology (PRNewsfoto/Colorado Urology)

About 5 in 10 men experience erectile dysfunction (ED) at some point in their lives. First-line therapies often include oral medication to help men achieve an erection. Now, Axol Therapy is providing a safe and effective alternative.

This non-invasive procedure uses gentle full-spectrum, low-intensity sound waves that stimulate revascularization, a process in which new blood vessels form. Axol Therapy promotes improved blood flow to the penis, reduces inflammation, and stimulates the migration of the body's stem cells for long-term healing. The new treatment is helping men to achieve natural erections without ED medications, pumps, injections, or penile implants.

Learn about Axol Softwave Therapy at Colorado Urology: https://www.coloradouro.com/specialties/axol-softwave-therapy/.

Axol Therapy How it Works

Axol Therapy is a modern approach to healing the body by using four types of energy: Heat, Electrohydraulic, Acoustic, and Light (HEAL). Unfocused acoustic waves are delivered to the shaft of the penis using a treatment wand that features a patented unfocused electrohydraulic acoustic wave.

The pulsed acoustic waves are delivered through the skin into the tissue to open and repair aging blood vessels, stimulate new blood vessel growth, restore blood flow, and improve erectile quality. Axol Therapy typically takes only 20 minutes, once a week, for a total of six sessions in the physician's office.

How Well Does Axol Therapy Work?

For men who are the right candidates, Axol Therapy is a safe and effective option without the side effects often experienced with oral medications. Most patients can get the quality, rigid erections they once had with Axol Therapy's gentle acoustic pulse treatment within just six office visits. Incremental improvement in erectile function may be seen after just a few sessions.

Restoring Vitality and Quality of Life

There are a number of significant benefits to Axol Therapy. For men who are candidates for this treatment option, a future without erectile dysfunction is perhaps the biggest one. The restoration of a man's vitality and spontaneous active sex life are also major benefits of this exciting new treatment.

Learn more about Axol Softwave Therapy, the benefits, and how to schedule a consultation. Visit https://www.coloradouro.com/specialties/axol-softwave-therapy/or call 888-401-7149.

About Colorado Urology

Colorado Urology, an affiliate of United Urology Group, is Eastern Colorado's premier urology practice, which was formed when Advanced Urology, Alpine Urology, and Foothills Urology became one urology group in April 2019. The group provides a broad array of urologic services, and its integrated approach to urologic care provides patients with access to experienced specialists, a comprehensive support team of healthcare professionals, innovative diagnostic tools, and highly advanced treatments and therapies. Colorado Urology operates 12 medical offices throughout the Denver metro and Boulder area, has 18 urologists, 9 advanced practice providers, and more than 130 employees.

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About United Urology Group

United Urology Group is a national management services organization whose member groups of urology practices include: Arizona Urology Specialists with locations across the greater Phoenix area; Chesapeake Urology, with offices located throughout Maryland and Delaware; Tennessee Urology, based in Knoxville, TN; and Colorado Urology, located in the greater Denver, Boulder and Front Range areas. United Urology Group members' collective staff today number more than 1,400 employees, including 150 physicians. United Urology's vision is to support the creation of a national network of urology affiliates, which will enable urologists to better meet the needs of their patients and provide the highest level of urological care.

Media Contact:

Patricia Schnably, Senior Vice President, Marketing & Communications United Urology Group25 Crossroads Drive, Suite 306, Owings Mills, MD 21117443-738-8107 pschnably@uniteduro.com

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Breakthrough, Non-Invasive Treatment Called Axol Therapy For Erectile Dysfunction And Enhanced Sexual Performance Now Available At Colorado Urology -...

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Searching for the ‘big break’ that could turn stem cells into a weapon against dementia – Genetic Literacy Project

By daniellenierenberg

Recent developments in the field of stem cell research are paving a path towards a radical shift in the way we diagnose and treat dementia. Stem cells have excited scientists for years and research groups across the globe are using them to advance modern medicine. Using stem cells to aid the fight against dementia is perhaps one of the most critical applications of the technology. Dementia is the leading cause of death in the UK, sixth in US and fifth globally, with an estimated 50 million people currently affected.

The term dementia does not relate to a single disease, but more an array of symptoms that can arise from multiple conditions. The most common is Alzheimers disease (AD) which accounts for up to 80% of all cases. Dementia itself is caused by the death of cells that make up the complex circuitry of our brains and an eventual loss of large portions of the brain. Patients suffering with dementia often exhibit the same general symptoms such as confusion, memory loss and an inability to perform day to day functions. It is a debilitating condition that often strikes the most vulnerable members of society and, consequently, many research groups around the globe work to try to understand dementia-causing diseases to provide better diagnostic and treatment platforms.

In 2007, a research group at Kyoto University in Japan published a study with the potential to change the face of research into dementia along with many other fields. Professor Shinya Yamanaka and his research team developed a method whereby stem cells (cells that can be transformed/differentiated into cells from any tissue) could be generated from a sample of skin. The study, which resulted in a 2012 Nobel Prize for Prof. Yamanaka, demonstrated that skin cells could be isolated from a patient and genetically reprogrammed into induced pluripotent stem cells (iPSCs). In short, this technology made it possible to generate and study brain cells from a patient with dementia without having to remove any of their brain. All they would need to do is provide scientists with a sample of skin.

Since this development, research groups around the globe have started using iPSCs from many patients with dementia in order to understand the biological mechanisms that underlie disease. Dr Eric Hill runs a research group at Aston University in the UK that specializes in iPSCs for dementia research and he had the following to say about the technology:

Its really exciting because it allows us to study cells with genetic mutations that are patient specific. We can get a much better picture of what is actually happening in the brains of these patients. We can now generate all the different cell types found in the human brain and understand how they function together and map the changes that result in disease.

The latter was perhaps most powerfully demonstrated in a study published by a team at the University of North Carolina, led by Professor Hansang Cho. The team was able to generate three key cell subtypes that play important roles in brain function; study the impact of mutations associated with Alzheimers disease; and even replicate some of the core malfunctions found to trigger disease in the brains of patients.

Studies like this are of significance because a large part of the focus in dementia research is on trying to understand how such changes in function arise. When a patient is diagnosed with a disease such as Alzheimers it is often too late for effective treatment. Scientists, instead, seek to elucidate those early changes in brain cell function in order to diagnose patients earlier to give more time for treatment. It is very much a case of prevention being better than a cure. Dr Hill provided an encouraging statement regarding this:

When we generate brain cells from iPSCs the cells we get are developmentally very young. What is interesting is the fact we still see differences between cells from dementia patients versus healthy patients suggesting we could find markers to help us detect and prevent disease some years before it develops.

Despite such promise, however, iPSCs have yet to provide the field of dementia research with that big break. Multiple treatments have progressed into clinical trials since the technology first emerged but no therapies have been approved. Drugs that show promise in the lab fail to deliver on their potential in patient clinical trials, sending researchers back to square one.

We should not be disheartened by this, however, and should instead view it as space into which the technology of using iPSCs to study dementia can grow. A lot of drugs fail in clinical trials because the platforms used to run initial tests dont provide scientists with a wide enough perspective of how those drugs will influence human cells. Additionally, many preclinical studies use animals with dementia-causing disease artificially induced into them. Studies like this often fail to translate into humans because the initial data is not from a human perspective. This is where researchers like Dr. Hill think iPSCs can provide us with an advantage:

iPSCs could provide us with much better platforms for screening drugs to treat and prevent these diseases. They can really add to what we already have, and while we might not be able to grow a full human brain, we can generate the cells that provide the building blocks for one. They give us the chance to screen new therapies more efficiently, better test their effectiveness and reduce the amount of animal use in dementia research.

Dr Hill is not alone in seeing the promise of using iPSCs to find better treatments for preventing the progression of dementia. Multiple research groups around the world have shown the potential of iPSC-derived brain cells for studying the effectiveness of new therapies.

In the last 12 months we have observed a wave of new studies using iPSCs to try to develop better treatments for diseases like Alzheimers, Parkinsons, Huntingtons disease and ALS. From studies in the University of California identifying cholesterol metabolism as a potential target to treating Alzheimers to studies in Luxembourg helping us find better treatments for Parkinsons, it is easy to see why the global effort to get that big break from iPSCs continues to gain interest. We might still be waiting for that next Noble Prize-winning discovery that will improve the lives of millions of patients but the collective effort of iPSC research groups across the world brings us a step closer with every study they publish. Dementia may, one day, be a thing of the past and iPSC research will likely be a significant part in getting us there.

Sam Moxon has a PhD in regenerative medicine and is currently involved in dementia research. He is a freelance writer with an interest in the development of new technologies to diagnose and treat degenerative diseases. Follow him on Twitter @DrSamMoxon

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Cumberland County family turns to non-FDA approved stem cell treatment to help two-year-old son with cerebral palsy – FOX43.com

By daniellenierenberg

Lance was diagnosed with cerebral palsy a year ago. His family hopes non-FDA approved stem cell treatment for the disease can help him walk and talk.

CAMP HILL, Pa. A family in Cumberland County has turned to stem cells to treat their two-year-old son diagnosed with cerebral palsy. The only problem: stem cell treatment for the disease hasn't been approved by the FDA.

The day he was born, when he wheeled him down the hall and he was only a pound, and I started to cry and said, will he live? And he said, of course Hes only small," said Danielle Maxwell, Lance's mom.

The words, "he's only small," are what Lance's mom and father Rob have lived by since the day he was born. The preemie, born three months early, has been through several surgeries and complications along the way. But, Lance has always been a fighter.

Lance fought so hard just to survive the beginning of life, and come home with us," said Danielle. "And he is just so happy and loving and amazing.

About a year ago, Lance was diagnosed with cerebral palsy. Doctors told his family, he will never walk, talk or take care of himself.

We just dont believe that," said Danielle. "We dont.

Lance receives a lot of different therapies but, his parents did not want to just stop there.

We both overwhelmingly feel, he never gave up, he never gave up on us, he never gave up on himself," said Rob. "So, we owe it to him to give him the opportunity. Its really that simple, he deserves the opportunity."

Danielle began researching stem cell therapies, even speaking to doctors in countries overseas where treatment with stem cells is more readily accessible than in the U.S. The FDA has approved stem cell treatments for some conditions but not cerebral palsy. However, trials to determine the effectiveness of stem cell treatment for the disease are underway.

What weve seen is a small but real appearing improvement in motor function," said Doctor Charles Cox with University of Texas Health in Houston, began a trial in 2013 on the safety and effectiveness of banked cord blood or bone marrow stem cells in children with cerebral palsy, and is now just wrapping up the results from the trial.

The overall results of this study depend if youre a glass half full or half empty kind of person," said Dr. Cox. "It is not a compelling miraculous result. Its not, Oh my God, this child was treated and look at this profound benefit.'"

Because stem cell treatment for cerebral palsy is still in trial phases, it's not approved treatment by the FDA. However, the Maxwells did find a doctor in Harrisburg willing to transfer stem cells from a full-term baby's umbilical cord to Lance. But, since it isn't FDA approved, we were not allowed to be there to show Lance receiving the stem cells. The Maxwells are hopeful following this procedure Lance may someday walk and more importantly be able to communicate with them.

He wants to be involved," said Rob. "You can tell hes trying to communicate he just cant get over that hump. We believe stem cells could be that bridge to help him move a little faster.

Danielle says, it will take about six months to see if the stem cells will have any definitive benefits for Lance. But, already says she's seeing progress. She says Lance is not able to stand on his own.

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Stem Cell Therapy Market Scope and Opportunities Analysis 2017 2025 – Instant Tech News

By daniellenierenberg

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD – BioSpace

By daniellenierenberg

NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute 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.1 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.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

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

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

Release authorized by the Chief Executive.

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Scientists May Have Found A Way To Boost The Body’s Ability To Heal Itself – IFLScience

By daniellenierenberg

Its not just Wolverine that has the ability to rebuild and restore wounded tissue. In fact, we all have a quite remarkable capacity to heal when we suffer an injury, thanks to our ability to produce new stem cells. Obviously, there is a limit to how much damage our bodies can repair, although researchers may have just discovered a way to enhance our powers of restoration by increasing the rate at which these stem cells are generated.

A new study in the journal Regenerative Medicine describes how scientists were able to stimulate the self-repair response of rats in order to rebuild broken spines. Healing similar injuries in humans is currently not possible, and the study authors are hopeful that their technique could one day help people recover from a range of previously untreatable injuries.

Rats in the study were given a cocktail of two drugs, one of which is normally administered during bone marrow transplants while the other is used for bladder control. This caused the rats bone marrow to produce an elevated number of mesenchymal stem cells, which are stem cells that can develop into bone tissue.

As a consequence, enhanced calcium binding was seen at the site of the rats spinal injuries, speeding up the formation of new bone and healing the wounds.

The figure on the right shows the level of healing with no treatment, while the figure on the left shows the effect of the two drugs in combination. The red coloring indicates calcium incorporating into the bone, which is associated with enhanced healing. Image: Imperial College London

We know that when bones break they will heal, and this requires the activation of stem cells in the bone, explained study co-author Sara Rankin in a statement. However, when the damage is severe, there are limits to what the body can do of its own accord.

We hope that by using these existing medications to mobilize stem cells, as we were able to do in rats in our new study, we could potentially call up extra numbers of these stem cells, in order to boost our bodies own ability to mend itself and accelerate the repair process.

Because the drugs involved are already widely used, the researchers are hopeful that human trials can proceed without the need for extensive safety testing. If these trials produce the same results as those seen in rats, then this treatment could help to not only repair spinal injuries, but to speed up the rate at which broken bones heal and even mend damaged tissues in other organs.

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New sickle cell disease treatments are helping people live longer and giving them a higher quality of life – PhillyVoice.com

By daniellenierenberg

Treatment for sickle cell disease has come a long way since the 1970s when the life expectancy of people living with it was less than 20 years.

People with sickle cell disease are not only living longer life expectancy is now 42 to 47 years of age but are enjoying a better quality of life, too.

"In the Philadelphia area, there has been great pediatric care for sickle cells disease and because of that people who have it are living very well," said Dr. Farzana Sayani, a hematologist at Penn Medicine.

Sayani is the director of a comprehensive sickle cell program focusing on adults living with the disease. Penn also has an active transition program for youth transitioning from a pediatric institution to adult care.

Sickle cell disease is an inherited red blood cell disorder that affects about 100,000 Americans.It is most often found in people of African or Hispanic descent.About 1 in 365 African-American babies are born with sickle cell disease, according to Sayani.

People who have the disease inherit an abnormal type of hemoglobin in their red blood cells, called Hemoglobin S, from both their mother and father.When only one parent has the hemoglobin S gene, a child will have the sickle cell trait, but usually does not develop the disease. But they may pass it on to their children.

Hemoglobin is the protein in the blood responsible for carrying oxygen to the rest of the body. Hemoglobin S causes red blood cells to become stiff and sickle-shaped. Instead of being round in shape, they look like crescent moons.

Sickle cells are sticky and can bind together, blocking the flow of blood and preventing oxygen from getting where it needs to go in the body. This causes sudden attacks of pain referred to as a pain crisis.

There are severaldifferent types of sickle cell disease.Hemoglobin SS, also known as sickle cell anemia, is the most common and most severe type of sickle cell disease.

Anemia occurs when red blood cells die at a rate faster than the body can replace them. Normal red blood cells generally live for 90 to 120 days. Sickled cells only live for 10 to 20 days. This shorter life-to-death cycle is harder for the body to sustain.

Another form,Hemoglobin SC, is not as severe as sickle cell anemia, but it can still cause significant complications, Sayani said.Other forms include Hemoglobin S0 thalassemia, Hemoglobin S+ thalassemia, Hemoglobin SD and Hemoglobin SE.

Sickle cell disease screening is a mandatory part of newborn screenings in Pennsylvania.

If the screening is positive, the family is informed and plugged into the health care system in order to receive the proper care.

If the disease is not diagnosed at birth, a blood test can confirm it at any age in which symptoms start to surface.

The severity of sickle cell disease can vary.

Each individual is affected differently, making it difficult to predict who will get what complications, Sayani said. That is why a comprehensive sickle cell program is so important.

Early signs include a yellowish tint to the skin or jaundice, fatigue and a painful swelling of the hands and feet.

"Young children with sickle cell disease may be tired, not eat very well and have delayed growth," Sayani said. "They may also develop anemia, be at greater risk of infection and start to experience pain crises."

Acute pain crises, also known as vaso-occlusive crises, can lead to long stays in the hospital to manage the crippling pain. Children with sickle cell disease also tend to experience delayed growth and puberty.

As a person with sickle cell disease grows older, the sickled red blood cells start to affect various organs, bones and joints.

This can lead to acute chest syndrome, which occurs when damaged lung tissues makes it difficult to breathe. Brain complications, including stroke, are possible.People with sickle cell disease are also prone to heart damage, eye problems, and infections like chlamydia, salmonella and staphylococcus. Chronic and acute pain is common.

There are different types of medicine that can help manage sickle cell disease.

Last year, an oral medicine was approved that makes sickle cells less likely to sickle. So was an intravenous medicine that has been shown to reduce pain crises and hospitalizations by 50%. Some people living with sickle cell disease also may need regular blood transfusions.

Hydroxyurea has also been used successfully for many years to reduce pain crises and the need for blood transfusions and hospitalizations.

Currently, blood and bone marrow transplant is the only way to cure the disease. But it is not an option for everyone because of the difficulty of finding a well-matched stem cell donor.

A related donor is best but only about a third of sickle cell patients have a donor that is related and fully-matched, Sayani said.

While these transplants have a 85% or more success rate, they also are associated with significant risks, including organ dysfunction, infection and graft vs. host disease which can be quite debilitating.

Transplants completed in children have the best results, Sayani said. But because of the risks involved, doctors only suggest it for patients with severe forms of the disease.

Early clinical trials with gene therapy are also showing promise, she added.

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CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells – Pharmacy Times

By daniellenierenberg

CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells

T cells and B cells are 2 primary cell types in our adaptive immune system, the source of immunological memory protecting us from subsequent pathogen exposure. B cells secrete pathogen-specific antibodies, which neutralize pathogens directly, or tag them for attack by other immune cells. T cells destroy pathogenic cells directly as well as secrete cytokines to attract additional immune cells.

Chimeric antigen receptor (CAR) T cells are patient derived T cells genetically manipulated to express an artificial transmembrane receptor. The artificial receptor is engineered from modular parts to bind to a surface protein (also called an antigen) on malignant cells and activate the T cell via engineered T cell signaling switches on the CAR.

Current FDA-approved CAR-T cell therapies express CARs recognizing CD19, which is expressed on the surface of almost all B cells, making these therapies specific for B-cell malignancies. Following binding with a CD19-expressing cell, the CAR T cell is activated to proliferate, eliminate the CD19-expressing cell, and persist within the patient.

Tisagenlecleucel (Kymriah, Novartis) is approved to treat pediatric and young adult patients (up to age 25) with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (R/R ALL, ELIANA trial) and adult patients with R/R diffuse large B-cell lymphoma (R/R DLBCL, JULIET trial) after 2 or more lines of systemic therapy. Axicabtagene ciloleucel (Yescarta, Kite Pharma) is approved for adult patients with R/R large B-cell lymphoma (R/R DLBCL, ZUMA-1 trial, NCT02348216) after 2 or more lines of systemic therapy. There are approximately 700 new cases of pediatric and young adult R/R ALL annually in the United States. New cases of R/R DLBCL are approximately 7000 annually in the United States.

CARs can be engineered to recognize virtually any cell surface antigen and can be expressed in a variety of immune cells, suggesting that product development will result in many modular CAR units with vast application versatility. Many different antigen and cell type combinations are already currently in development to address several cancers, such as CAR T cells for pancreatic cancer (NCT03323944).

The first step in creating these personalized genemodified cell therapies is collecting patient lymphocytes via leukapheresis at a clinic or infusion center. Lymphocytes include T cells, B cells, and natural killer cells. The leukapheresis process lasts up to 4 hours and must be coordinated with the patients continuing care regimen to ensure sufficient T cells. The lymphocytes are cryopreserved and immediately shipped to a centralized manufacturing facility.

T cells are separated from the other cells in the leukapheresis product and genetically manipulated, typically using a lentiviral gene delivery method to carry DNA encoding the CAR protein, resulting in CAR T cells. The CAR T cells are cultured to a patient-specific appropriate dose. As this process is finishing, the manufacturer coordinates with the patients health care team to ensure the patient and team are prepared to infuse the CAR T cells. The manufacturing process from the apheresis process to the clinical CAR-T cell product varies widely from patient to patient, from 14 days up to a few months. The limiting step is typically reaching the appropriate CAR-T cell dose.

About a week before the scheduled CAR-T cell infusion, the patient receives multiple days of low-dose conditioning chemotherapy. This step serves to deplete lymphocytes before administration of the CAR T cells, improving the efficacy and persistence of therapy. The CAR T cells are then administered intravenously, and the patient is monitored for adverse events (AEs). The most common AEs with both currently approved products include cytokine release syndrome (CRS), neurological toxicity (NT), hypersensitivity reactions, serious infection, prolonged cytopenias, and hypogammaglobulinemia.3,4 Both products caution that therapy could cause hepatitis B viral reactivation.

The most severe reactions are CRS and NT, both of which can be life threatening. CRS, a common immune reaction following infusion of monoclonal antibodies and CAR T cells, is characterized by fever, nausea, chills, hypotension, tachycardia, asthenia, headache, rash, and dyspnea.5 Mild cases are easily managed, whereas severe cases require more aggressive and invasive therapy, such as mechanical ventilation and intravenous administration of tocilizumab. NT associated with CAR-T cell therapy is characterized by encephalopathy, headache, aphasia, delirium, insomnia, anxiety, tremor, dizziness, seizures, and peripheral neuropathy.

During the clinical trials of Kymriah and Yescarta, CRS and NT occurred in most patients with more than 10% experiencing severe CRS and more than 20% with severe NT.3,4 Initially, the 2 AEs appeared to be independent, but data are beginning to emerge suggesting a correlation. CRS might be a predictor of neurological events; however, neurological events do not predict CRS.6

Real-world evidence from patients treated with Kymriah, presented at the 2019 Society of Hematologic Oncology annual meeting, reported that slightly more than half experienced either of these conditions and less than 20% had severe cases.7 Two-year followup data regarding Yescarta reported severe CRS cases in 11% of patients and severe NT cases in 32%.8 Due to the high rate of occurrence and severity of CRS and NT, both Yescarta and Kymriah have restricted availability through Risk Evaluation and Mitigation Strategies.3,4 Both medications must be administered at a Risk Evaluation and Mitigation Strategiescertified health care facility with health care providers trained in the management and treatment of CRS and 2 doses of tocilizumab available for each patient before CART cell infusion.

Regardless of the potential for severe adverse events (AEs), the benefit of both Yescarta and Kymriah outweigh the risks, as they are highly effective singleadministration therapies. Despite the aggressive nature of the cancers treated with CAR T-cell therapy, meaningful clinical benefit can be achieved within 1 month. A summary of clinical trial primary response rates as well as 2-year data and published real-world data can be found in Table.

Despite differences between the 2 clinically available products, their safety and efficacy profiles in patients with R/R DLBCL are comparable. Differences between the therapies range from the molecular units comprising the CARs, manufacturing differences, lymphodepletion regimen, number of CAR T cells and volume infused, and whether the infusion and short-term patient monitoring occurs in an inpatient or outpatient setting.

Patient-specific genetically modified cell therapies can present many manufacturing challenges. One stark difference between the available therapies, relevant to the patient and clinician experience, has been the manufacturing time and failure rate. During ZUMA-1, of the 101 patients treated with Yescarta, the median time from leukapheresis to product delivery was 17 days (range, 14-51 days), and a 1% manufacture failure rate was reported.4 The ELIANA trial of Kymriah in patients with R/R ALL reported a 9% manufacture failure rate, whereas the JULIET R/R DLBCL trial reported a failure rate of 6.9%, and of the 106 patients receiving Kymriah, the median manufacture time was 113 days (range, 47-196 days).3

Commercial manufacture of Kymriah for DLBCL has struggled to meet specifications.11 While addressing the production issue, Novartis has initiated a safety study evaluating out-of-specification product (NCT04094311) and a managed-access program (NCT03601442).

Known causes of CAR-T cell therapy failure are T cell exhaustion and antigen escape. T cell exhaustion is characterized by a loss of responsive T cells due to changes in gene expression and can be prevented by immune checkpoint inhibitors PD-1, PD-L1, or CTLA- 4. Antigen escape describes a condition in which some cancer cells do not express the CAR-targeted antigen; therefore, they escape immune activation and survive within the patient. Engineering a secondary CAR to a different antigen, such as CD22 in the case of ALL, increases the likelihood of targeting all malignant cells. Solutions to both of these inhibitory mechanisms are currently under clinical trial investigation.12,13

The therapeutic success of CAR T cells ensures gene-modified immune cell therapy will be refined, optimized, and broadly applied until limits are reached. Many clinical groups are investigating biomarkers associated with severe AEs to provide an additional layer of precision care to the CAR-T cell therapy model.6,14 In addition, clinical trials are underway evaluating combination therapies to enhance the efficacy and improve the safety of CART cell therapy. Early-stage research is evaluating the possibility of off-the-shelf CAR-T cell therapy, not a patient-unique manufactured product, to reduce the time to treatment and achieve manufacturing efficiencies and consistencies.15,16 Gene-modified cell therapy, such as CAR T-cell therapy, is revolutionizing oncology, and this living drug model is breathing life into the hopes of patients with cancer and caregivers.

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Stress and Gray Hair – ThirdAge

By daniellenierenberg

Medical ResearchThe belief that acute stress can turn hair gray is a popular one, but until now it hasnt been scientifically proven.

But findings that appeared in the publication Nature indicate that the belief may be more than a myth. The study, which used mice as models, was funded in part by the National Institutes of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other NIH components.

Hair color is determined by cells called melanocytes, which produce the pigment melanin. New melanocytes are made from melanocyte stem cells that live within the hair follicle at the base of the hair strand. As we age, these stem cells gradually disappear. The hair that regrows from hair follicles that have lost melanocyte stem cells has less pigment and appears gray.

A research team, led by Dr. Ya-Chieh Hsu of Harvard University, used mice to examine stress and hair graying. The mice were exposed to three types of stress involving mild, short-term pain, psychological stress, and restricted movement. All caused noticeable loss of melanocyte stem cells and hair graying.

Having established a link between stress and graying, the scientists then explored several potential causes, including the role of the stress hormone corticosterone, but altering its levels didnt affect stress-related graying.

The researchers eventually turned to the neurotransmitter noradrenaline, which, along with corticosterone, was elevated in the stressed mice. They found that noradrenaline, also known as norepinephrine, was key to stress-induced hair graying. By injecting noradrenaline under the skin of unstressed mice, the researchers were able to cause melanocyte stem cell loss and hair graying.

Noradrenaline is produced mostly by the adrenal glands. However, mice without adrenal glands still showed stress-related graying. Noradrenaline is also the main neurotransmitter of the sympathetic nervous system, which is responsible for the fight-or-flight reaction in response to stress.

Ultimately, the team discovered that signaling from the sympathetic nervous system plays a critical role in stress-induced graying. Sympathetic nerves extend into each hair follicle and release noradrenaline in response to stress. Normally, the melanocyte stem cells in the follicle are dormant until a new hair is grown. Noradrenaline causes the stem cells to activate.

Using fluorescent labelling, the researchers observed the stem cells change to melanocytes and migrate away from their reserve in the hair follicle. With no remaining stem cells, no new pigment cells can be made, and any new hair becomes gray, then white.

When we started to study this, I expected that stress was bad for the body but the detrimental impact of stress that we discovered was beyond what I imagined, Hsu says. After just a few days, all of the melanocyte stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

The authors highlight the need to further study the interactions between the nervous system and stem cells in different tissues and organs. A news release from the NIH said that the knowledge gained in this work will be useful in future investigations into the impact of stress on the body and the development of new interventions.

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Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD – Benzinga

By daniellenierenberg

Patient Analyses and Safety Data Continue to Underscore Positive Impact of KD025 in cGVHD

Pre-NDA Meeting with FDA Planned for March 2020; Topline Results of Primary Analysis to be Announced in Q2 2020

NEW YORK, NY / ACCESSWIRE / February 23, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced expanded results from the previously reported interim analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in chronic graft-versus-host disease (cGVHD). The data were presented today in the oral latebreaker session at the 2020 Transplantation & Cellular Therapy (TCT) Meetings.

As announced in November 2019, KD025 met the primary endpoint of Overall Response Rate (ORR) at the study's planned interim analysis, two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). In the expanded KD025-213 dataset presented today, ORRs were consistent with the previously reported interim analysis across key subgroups, including in patients with four or more organs affected by cGVHD (n=69; 64%), patients who had prior treatment with ibrutinib (n=45; 62%) and patients who had prior treatment with ruxolitinib (n=37; 62%). Three patients achieved a Complete Response. Responses were observed in all affected organ systems, including in organs with fibrotic disease. KD025 has been well tolerated: adverse events were consistent overall with those expected to be observed in cGVHD patients receiving corticosteroids, and no apparent increased risk of infection was observed. Additional secondary endpoints, including duration of response, corticosteroid dose reductions, Failure-Free Survival, Overall Survival and Lee Symptom Scale reductions continue to mature and will be available later in 2020.

"KD025 has been well tolerated and has already demonstrated high response rates in patients with severe and complex cGVHD after a median of five months of follow-up," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member.

"We are extremely pleased with the interim outcomes of this pivotal trial of KD025 in cGVHD, which track closely our findings from our earlier Phase 2 study. KD025 achieved robust response rates across all subgroups of this difficult-to-treat patient population, who had a median of four prior lines of therapy, and 73% of whom had no response to their last line of treatment," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We plan to meet with the FDA for a pre-NDA meeting in March 2020 and to announce topline results from the primary analysis of this trial in Q2 2020."

At the TCT Meetings, Kadmon also presented long-term follow-up data from KD025-208, its ongoing Phase 2 study of KD025 in cGVHD (Abstract #15205). These data were recently presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in December 2019.

About the ROCKstar (KD025-213) Trial

KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg once daily or KD025 200 mg twice daily, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%.

While the ORR endpoint was met at the interim analysis, which was conducted as scheduled two months after completion of enrollment, topline data from the primary analysis of the KD025-213 study, six months after completion of enrollment, will be reported in Q2 2020. Full data from the primary analysis will be submitted for presentation at an upcoming scientific meeting.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.

About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.

Forward Looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe, China, Japan and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel (xx) the potential benefits from any of our product candidates being granted orphan drug or breakthrough designation; (xxi) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxii) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the U.S. Securities and Exchange Commission (the "SEC"), including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Contact Information

Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com

SOURCE: Kadmon Holdings, Inc.

View source version on accesswire.com: https://www.accesswire.com/577466/Kadmon-Announces-Expanded-Results-of-Interim-Analysis-of-Pivotal-Trial-of-KD025-in-cGVHD

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Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD - Benzinga

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