Archive for the ‘Bone Marrow Stem Cells’ Category

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

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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Be a Bone Marrow Hero - University of Utah Health Care

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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Scientists May Have Found A Way To Boost The Body's Ability To Heal Itself - IFLScience

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

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 .

Continued here:
Humans soon able to regrow spines as body given 'new power to heal itself' - Daily Star

Over the past few years, there has been a significant increase in the number of bone marrow registries. Increasing awareness, coupled with a rise in promotional campaigns run by healthcare practitioners, is majorly responsible for the rise in the number of bone marrow registries globally. The trend is especially quite prominent in developed regions.

This press release was orginally distributed by SBWire

Seattle, WA -- (SBWIRE) -- 02/25/2020 -- Bone Marrow Transplant Market report gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies, identify emerging players with the potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. Bone Marrow Transplant market report identifies potential new clients or partners in the target demographic, develop strategic initiatives by understanding the focus areas of leading companies, plan mergers and acquisitions effectively by identifying key players.

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For more information on this press release visit: http://www.sbwire.com/press-releases/bone-marrow-transplant-lower-risk-of-disease-recurrence-and-significant-rise-in-the-number-of-healthy-donors-drive-the-market-growth-1278593.htm

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Bone Marrow Transplant: Lower Risk of Disease Recurrence and Significant Rise in the Number of Healthy Donors Drive the Market Growth - Press Release...

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.

Story continues

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

A new proof-of-concept study has found a combination of two drugs, already approved by the FDA for other uses, may boost the release of stem cells from bone marrow and accelerate the healing of broken bones. Only demonstrated in animals at this stage, the researchers suggest clinical trials could progress rapidly considering the drugs have already been demonstrated as safe in humans.

"The body repairs itself all the time, says corresponding author on the study Sara Rankin. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord.

A great deal of current research is focusing on mesenchymal stem cell (MSC) therapies. MSCs are a type of adult stem cell that can grow into a variety of different cell types including muscle, fat or bone. Many current MSC treatments in development involve extracting a small number from a patient, growing them in laboratory conditions, then injecting them back into the patient.

The new research set out to investigate whether any currently approved drugs can function to mobilize the bodys natural ability in releasing MSCs, with a view on speeding up healing of bone fractures. A study published in the journal npj Regenerative Medicine, describes the testing of two already approved drugs in a rodent spinal injury model.

The two drugs tested were an immunostimulant called Plerixafor, used to stimulate the release of stem cells from bone marrow in cancer patients, and a beta-3 adrenergic agonist developed to help bladder control.

The results suggest the duo of drugs mobilize MSCs into the bloodstream and speed up the process of bone formation and healing by enhancing the binding of calcium to the injury site. Tariq Fellous, first author on the new study, suggests the next step is to investigate whether this drug combination enhances blood MSC levels in human subjects.

We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures, says Fellous. We have the drugs and know they are safe to use in humans - we just need the funding for the human trials.

The researchers say prior studies have identified circulating MSCs increase in volume following injuries such as burns, bone fractures, and even heart attack. The hypothesis is that the release of MSCs is a physiological process aiding general regeneration following injury, and if circulating numbers of MSCs could be pharmacologically enhanced then a variety of types of tissue regeneration could be accelerated.

It is important to note the current study only examined increases in circulating MSCs and the rate of spine injury healing compared to no drug treatment. The current research offers no indication whether the drug duo influences nerve healing or restores movement.

So, more work is certainly necessary to understand how clinically useful these results actually are. However, as the studys co-first author Andia Redpath notes, this re-purposing of existing medicines to boost stem cell activity is an easier, cheaper, and more efficient way to enhance healing compared to other, more complex and time-consuming, stem cell treatments in development.

Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs, says Redpath. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

The new study was published in the journal npj Regenerative Medicine.

Source: Imperial College London

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Experimental study speeds up bone healing with 2 common medications - New Atlas

LEXINGTON, Ky. (WKYT) - Firefighters in Lexington spent their Saturday morning supporting their own with a pancake breakfast raising money for a young woman with a rare disease.

Chris OBryan has worked as a firefighter in Kentucky for 23 years. This past summer his daughter began experiencing stomach pain. After months of testing, doctors diagnosed her PCH, a rare disease caused by a mutation in bone marrow stem cells.

Once other Lexington Firefighters heard about OBryans daughter, they decided to help however they can.

Its seven days that this came together, said firefighter William Tabor.

Tabor initiated the breakfast without ever meeting OBryan and his family before.

Both men say that goes to show the family community within the fire department. Within just more than an hour of beginning the breakfast at 8 Saturday, they raised $5,000.

The money thats raised here is twofold, started OBryan. Some of its for my daughter and some of its for a Louisville firefighter who was tragically killed in Saint Louis.

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Firefighter raise thousands within hours for one of their own - WKYT

FLORENCE, Ala. (AP) Cole Kelley grinned widely and waved Saturday (Feb. 15) as his head kept swiveling from one direction to the other to take in the amazing view surrounding him.

Hundreds of family members, friends, Mars Hill School classmates and other members of the community lined both sides of the school's parking lot to welcome the 8-year-old home.

The Mars Hill student and his parents, John and Caroline Kelley, had been away from home for 15 months while he has battled a rare disease.

That included a 460-day stay at the National Institutes of Health facility in Bethesda, Maryland, which is one of the few facilities with experience treating the disease dada2, which is a deficiency of the adenosine deaminase 2 (ADA2) enzyme.

According to dada2.org, the disease causes "recurrent strokes, severe systemic inflammation, immune deficiency, and damage to many of the body's tissues and organs."

The community organized the homecoming parade, which included an escort from Florence's police and fire departments, as well as people dressed in superhero outfits.

Along with Cole's classmates, Mars Hill High School cheerleaders joined the crowd, as did the school's football players, who showed up in their jerseys. The long reception line spanned the length of the parking lot coming off Cox Creek Parkway.

The procession made two laps through the parking lot amid loud cheers and flowing tears from the congregation before the family, clearly emotional from the outpouring, drove home.

"It's been amazing," Cole's cousin, Sara Beth Searcy, said while wearing a "Best Day Ever" shirt she bought during a Disney World trip Cole and the family took before his long hospital stay.

"This whole community has supported us from the very beginning," she said. "We could not have gotten through this without this entire community wrapping their arms around us."

Cole classmate Rogan Willingham held a sign that read "God answered our prayers."

"He's prayed, I don't know how many times a day, for Cole," his mother, Ginger Willingham, said.

"I'm glad he's back," Rogan said, adding he looks forward to being able to "play with him and all kinds of stuff."

"I was praying for him a lot," he said.

There were numerous other signs with messages, such as "Sweet Home, Alabama," "Super Cole" and "Welcome home, Buddy."

Emily Stutts, a friend of the family who helped organized the welcome, and fellow member of Jackson Heights Church of Christ, said Cole has been sick since he was 2 months old.

He had a bone transplant at the National Institutes.

"He lived off of having blood transfusions because his body did not make red blood cells," Stutts said. "After a while that became risky because his body can't filter out all the iron. So a bone marrow transplant was the only answer."

He was enrolled in the "Be the Match" bone marrow registry and was a match with a young man from Australia, who had volunteered to join the registry.

"He was able to give the bone marrow and they flew it over to Maryland and did the transplant," Stutts said. "It didn't take initially, and Cole has had three stem-cell transplants, all from the one donation from the Australian man.

"The fact that he's coming home is a miracle, because he had some very, very scary times that they didn't think he would survive. He had zero immune until Christmas day. That day his immune response occurred."

She said John and Caroline often talk about the amazing support they have been receiving back home. A community spaghetti supper, T-shirt sales and a Christmas fundraising event are among methods supporters have used to provide financial assistance for the parents, who have not been able to work during their son's hospitalization.

"The people at the hospital there can't believe the support that they've been given from this community," Stutts said. "They told them they see people from all over the world, but they've never seen the outpouring of love like they saw for the Kelleys.

"They deserve it. They're such good people."

Florence company High Cotton Homes provided assistance in a major way, Stutts said.

"They had to have all new heating and air units and vents put in and a water purification system, all new flooring and all new duct work because of his immune system," she said. "High Cotton took that on, got donations and completely remodeled his room and made it so Cole would have his own bathroom to help with germ prevention."

Cole still is susceptible to sickness and will continue to have medical appointments, Stutts said.

"His liver was damaged from the transplant so he's going to go to Vanderbilt for them to check his liver," she said. "There's still things that need to be addressed, but as far as his bone marrow, that is doing OK."

Stutts said Cole's disease has not kept him down.

"He is full of life," she said. "He's a happy kid. Even though he's been sick, if you had seen him, you would have never know it. He's made a huge impact on the people at the National Institutes for Health because no child's been there that long. That became his family. They had a going-away party at one of the doctor's homes."

The emotions of Saturday's homecoming were obvious among the crowd, many of whom hugged one another and cried.

"The one thing I kept hearing from everybody is it was more emotional than they through it would be," said Ronnie Pannell, family minister at Jackson Heights. It's been a big show of love. Mars Hill loves Cole Kelley, there's no doubt about that.

Originally posted here:
Community give child a hero's homecoming - Thehour.com

Alfie Commons and mum Lorna, 40, met Christin Bouvier, 34, for the first time on Wednesday after spending more than two years communicating via anonymous letters due to donation laws.

Alfie, from Toton, Notts, was diagnosed with acute lymphoblastic leukaemia (ALL) at seven months after he had a cold for six weeks.

After chemotherapy failed, Alfie was put on a trial immunotherapy drug which helped him to recover to be eligible for a stem cell transplant.

Unable to find a family match, his family found Christin on a worldwide register operated by blood cancer charity DKMS. She donated her cells in a one-hour op and they were flown to the UK and slowly passed into Alfies body in August 2016.

When the teacher, from Schwerin, Germany, was finally allowed to meet the family at Chiswick Town Hall in west London, the little boy gave a gift of Lacoste Pink perfume.

Mum-of-two Lorna, who works in HR, said: The meet-up was just amazing, it was everything we could have possibly dreamed of there were lots of hugs and tears.

It didnt feel like I was meeting her for the first time because wed been chatting for so long before.

As a family, we owe so much to Christin, words of thanks will never feel enough.

Christin just cried when she heard the recipient was a baby. She said: After I donated my bone marrow and the anaesthetic wore off, I called DKMS.

They told me that Alfie was a small baby and living in the UK but couldnt tell me any more due to the laws. When I found out Alfie was responding to treatment, so many tears of joy ran down my face. I still cant describe that moment.

Its a moment that is always with me. Whenever I feel a bit down, I think back to it as it always brings me so much happiness! She added that the meeting was so amazing. I was very nervous and shaking at first and when we finally met we cried a lot and hugged.

Alfie was shy at first but after a bit of time he became more comfortable and we played with some balloons and had a slice of cake.

After the transplant, Alfie developed a deadly immune condition, but this was controlled by medication. He was given the cancer all-clear in 2017 and has started school.

Lorna added: I just want more people to sign up to become donors theres a match for everyone.

Read more here:
From Germany with love: Alfie, four, meets his stem cell saviour - Express

DEFINITIONLeukemia is a Cancer of blood forming tissues including bone marrow and the lymphatic system.This type of cancer hinders the body's ability to fight infection, leukemia involves the white blood cells.

CAUSES OF LEUKEMIALeukemia can develop due to a problem with blood cell production.

Several factors have been identified which increase the risk of having the cancer:

A family history of leukemiaSmokingGenetic disorder such as Down syndromeExposure to chemicals such as benzeneExposure to high levels of radiationTYPES OF LEUKEMIALeukemia can be Acute or Chronic. In Acute leukemia, Cancer cell multiply quickly while in Chronic leukemia, the disease progresses slowly and early symptoms may be very mild.

Leukemia can also be classified according to types of cells which are myelogenous leukemia and lymphotic leukemia involving myeloid cells and lymphocytes respectively.

SYMPTOMS OF LEUKEMIAExcessive sweating most especially in the nightFatigue and weaknessUnintentional weight lossFever or chillsFrequent infectionsBleeding easily and bruising easilyEnlargement of the liver or spleenLEUKEMIA IN PREGNANCYLeukemia affects approximately 1 in 10000 pregnancies.Women with leukemia have non-specific symptoms and some of them could also be attributed to pregnancy.

The damage in the fetus is correlated with the time of exposition and the fetus is most vulnerable during organnogenesis phase, Although chemotherapy has effect in the fetus, there are reports of cases with successful pregnancy.

It is necessary to study the relation among chemotherapy, the leukemia and the fetus in long term studies where fetus has been exposed to chemotherapy agents and is reported with normal characteristics at birth.

ENZYMES DEFICIENT IN LEUKEMIABlast cells from 100 cases of Acute leukemia were evaluated for the presence of methylthioadenosine phosphorylase (MTAase), an enzyme important in polyamine metabolism.

Ten cases (10%) had undetectable levels of MTAase activity. A relatively high frequency (38%) of MTAase deficiency was seen in all of T-cell origin.

MTAase deficiency occurs in a wide variety of acute leukemia, that the lack of enzyme activity is specific in malignant cells. The absence of MTAase in some leukemia may be therapeutically exploitable.

TREATMENT OF LEUKEMIALeukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer.

The treatment depends on the type and stage of the cancer, the treatment includes the following:

Chemotherapy uses drugs to kill leukemia cells.Radiation therapy uses high energy radiation to damage leukemia cells and inhibit their growth.

Stem cell transplantation replaces diseased bone marrow with healthy bone marrow.

Biological or immune therapy uses treatments that help your immune system recognize and attack cancer cells.

Targeted therapy uses medication advantages of vulnerabilities in cancer cells.

Excerpt from:
Leukemia: Cancer Of The Blood - Modern Ghana

Heart disease is the primary cause of death worldwide, principally because the heart has minimal ability to regenerate muscle tissue. Myocardial infarction (heart attack) caused by coronary artery disease leads to heart muscle loss and replacement with scar tissue, and the heart's pumping ability is permanently reduced. Breakthroughs in stem cell biology in the 1990s and 2000s led to the hypothesis that heart muscle cells (cardiomyocytes) could be regenerated by transplanting stem cells or their derivatives. It has been 18 years since the first clinical trials of stem cell therapy for heart repair were initiated (1), mostly using adult cells. Although cell therapy is feasible and largely safe, randomized, controlled trials in patients show little consistent benefit from any of the treatments with adult-derived cells (2). In the meantime, pluripotent stem cells have produced bona fide heart muscle regeneration in animal studies and are emerging as leading candidates for human heart regeneration.

In retrospect, the lack of efficacy in these adult cell trials might have been predicted. The most common cell type delivered has been bone marrow mononuclear cells, but other transplanted cell types include bone marrow mesenchymal stromal cells and skeletal muscle myoblasts, and a few studies have used putative progenitors isolated from the adult heart itself. Although each of these adult cell types was originally postulated to differentiate directly into cardiomyocytes, none of them actually do. Indeed, with the exception of skeletal muscle myoblasts, none of these cell types survive more than a few days in the injured heart (see the figure). Unfortunately, the studies using bone marrow and adult resident cardiac progenitor cells were based on a large body of fraudulent work (3), which has led to the retraction of >30 publications. This has left clinical investigators wondering whether their trials should continue, given the lack of scientific foundation and the low but measurable risk of bleeding, stroke, and infection.

Additionally, investigators have struggled to explain the beneficial effects of adult cell therapy in preclinical animal models. Because none of these injected cell types survive and engraft in meaningful numbers or directly generate new myocardium, the mechanism has always been somewhat mysterious. Most research has focused on paracrine-mediated activation of endogenous repair mechanisms or preventing additional death of cardiomyocytes. Multiple protein factors, exosomes (small extracellular vesicles), and microRNAs have been proposed as the paracrine effectors, and an acute immunomodulatory effect has recently been suggested to underlie the benefits of adult cell therapy (4). Regardless, if cell engraftment or survival is not required, the durability of the therapy and need for actual cells versus their paracrine effectors is unclear.

Of particular importance to clinical translation is whether cell therapy is additive to optimal medical therapy. This remains unclear because almost all preclinical studies do not use standard medical treatment for myocardial infarction. Given the uncertainties about efficacy and concerns over the veracity of much of the underlying data, whether agencies should continue funding clinical trials using adult cells to treat heart disease should be assessed. Perhaps it is time for proponents of adult cardiac cell therapy to reconsider the approach.

Pluripotent stem cells (PSCs) include embryonic stem cells (ESCs) and their reprogrammed cousins, induced pluripotent stem cells (iPSCs). In contrast to adult cells, PSCs can divide indefinitely and differentiate into virtually every cell type in the human body, including cardiomyocytes. These remarkable attributes also make ESCs and iPSCs more challenging to control. Through painstaking development, cell expansion and differentiation protocols have advanced such that batches of 1 billion to 10 billion pharmaceutical-grade cardiomyocytes, at >90% purity, can be generated.

Preclinical studies indicate that PSC-cardiomyocytes can remuscularize infarcted regions of the heart (see the figure). The new myocardium persists for at least 3 months (the longest time studied), and physiological studies indicate that it beats in synchrony with host myocardium. The new myocardium results in substantial improvement in cardiac function in multiple animal models, including nonhuman primates (5). Although the mechanism of action is still under study, there is evidence that these cells directly support the heart's pumping function, in addition to providing paracrine factors. These findings are in line with the original hope for stem cell therapyto regenerate lost tissue and restore organ function. Additional effects, such as mechanically buttressing the injured heart wall, may also contribute.

Breakthroughs in cancer immunotherapy have led to the adoption of cell therapies using patient-derived (autologous) T cells that are genetically modified to express chimeric antigen receptors (CARs) that recognize cancer cell antigens. CAR T cells are the first U.S. Food and Drug Administration (FDA)approved, gene-modified cellular pharmaceutical (6). The clinical and commercial success of autologous CAR T cell transplant to treat B cell malignancies has opened doors for other complex cell therapies, including PSC derivatives. There is now a regulatory path to the clinic, private-sector funding is attracted to this field, and clinical investigators in other areas are encouraged to embrace this technology. Indeed, the first transplants of human ESC-derived cardiac progenitors, surgically delivered as a patch onto the heart's surface, have been carried out (7). In the coming years, multiple attempts to use PSC-derived cardiomyocytes to repair the human heart are likely.

What might the first human trials look like? These studies will probably employ an allogeneic (non-self), off-the-shelf, cryopreserved cell product. Although the discovery of iPSCs raised hopes for widespread use of autologous stem cell therapies, the current technology and regulatory requirements likely make this approach too costly for something as common as heart disease, although this could change as technology and regulations evolve. Given that it would take at least 6 months to generate a therapeutic dose of iPSC-derived cardiomyocytes, such cells could only be applied to patients whose infarcts are in the chronic phase where scarring (fibrosis) and ventricular remodeling are complete. Preclinical data indicate that chronic infarcts benefit less from cardiomyocyte transplantation than do those with active wound-healing processes.

Adult cells from bone marrow or the adult heart secrete beneficial paracrine factors but do not engraft in the infarcted heart. Pluripotent stem cells give rise to cardiomyocytes that engraft long term in animal models, beat in synchrony with the heart, and secrete beneficial paracrine factors. Long-term cardiomyocyte engraftment partially regenerates injured heart, which is hypothesized to bring clinical benefits.

The need for allogeneic cells raises the question of how to prevent immune rejection, both from innate immune responses in the acute phase of transplantation or from adaptive immune responses that develop more slowly through the detection of non-self antigens presented by major histocompatibility complexes (MHCs). A current strategy is the collection of iPSCs from patients who have homozygous MHC loci, which results in exponentially more MHC matches with the general population. However, studies in macaque monkeys suggest that MHC matching will be insufficient. In a macaque model of brain injury, immunosuppression was required to prevent rejection of MHC-matched iPSC-derived neurons (8). Similarly, MHC matching reduced the immunogenicity of iPSC-derived cardiomyocytes transplanted subcutaneously or into the hearts of rhesus macaques, but immunosuppressive drugs were still required to prevent rejection (9).

Numerous immune gene editing approaches have been proposed to circumvent rejection, including preventing MHC class I and II molecule expression, overexpressing immunomodulatory cell-surface factors, such CD47 and human leukocyte antigen E (HLA-E) and HLA-G (two human MHC molecules that promote maternal-fetal immune tolerance), or engineering cells to produce immunosuppressants such as programmed cell death ligand 1 (PDL1) and cytotoxic T lymphocyteassociated antigen 4 (CTLA4) (10). These approaches singly or in combination seem to reduce adaptive immune responses in vitro and in mouse models. Overexpressing HLA-G or CD47 also blunts the innate natural killer cellmediated response that results from deleting MHC class I genes (11). However, these manipulations are not without theoretical risks. It could be difficult to clear viral infections from an immunostealthy patch of tissue, and possible tumors resulting from engraftment of PSCs might be difficult to clear immunologically.

Ventricular arrhythmias have emerged as the major toxicity of cardiomyocyte cell therapy. Initial studies in small animals showed no arrhythmic complications (probably because their heart rates are too fast), but in large animals with human-like heart rates, arrhythmias were consistently observed (5, 12). Stereotypically, these arrhythmias arise a few days after transplantation, peak within a few weeks, and subside after 4 to 6 weeks. The arrhythmias were well tolerated in macaques (5) but were lethal in a subset of pigs (12). Electrophysiological studies indicate that these arrhythmias originate in graft regions from a source that behaves like an ectopic pacemaker. Understanding the mechanism of these arrhythmias and developing solutions are major areas of research. There is particular interest in the hypothesis that the immaturity of PSC-cardiomyocytes contributes to these arrhythmias, and that their maturation in situ caused arrhythmias to subside.

A successful therapy for heart regeneration also requires understanding the host side of the equation. PSC-derived cardiomyocytes engraft despite transplantation into injured myocardium that is ischemic with poor blood flow. Although vessels eventually grow in from the host tissue, normal perfusion is not restored. Achieving a robust arterial input will be key to restoring function, which may require cotransplanting other cell populations or tissue engineering approaches (13, 14). Most PSC-mediated cardiac cell therapy studies have been performed in the subacute window, equivalent to 2 to 4 weeks after myocardial infarction in humans. At this point, there has been insufficient time for a substantial fibrotic response. Fibrosis has multiple deleterious features, including mechanically stiffening the tissue and creating zones of electrical insulation that can cause arrhythmias. Extending this therapy to other clinical situations, such as chronic heart failure, will require additional approaches that address the preexisting fibrosis. Cell therapy may again provide an answer because CAR T cells targeted to cardiac fibroblasts reduced fibrosis (15).

Developing a human cardiomyocyte therapy for heart regeneration will push the limits of cell manufacturing. Each patient will likely require a dose of 1 billion to 10 billion cells. Given the widespread nature of ischemic heart disease, 105 to 106 patients a year are likely to need treatment, which translates to 1014 to 1016 cardiomyocytes per year. Growing cells at this scale will require introduction of next generation bioreactors, development of lower-cost media, construction of large-scale cryopreservation and banking systems, and establishment of a robust supply chain compatible with clinical-grade manufacturing practices.

Beyond PSC-cardiomyocytes, other promising approaches include reactivating cardiomyocyte division and reprogramming fibroblasts to form new cardiomyocytes. However, these approaches are at an earlier stage of development, and currently, PSC-derived cardiomyocyte therapy is the only approach that results in large and lasting new muscle grafts. The hurdles to this treatment are known, and likely addressable, thus multiple clinical trials are anticipated.

Acknowledgments: C.E.M. and W.R.M. are scientific founders of and equity holders in Sana Biotechnology. C.E.M. is an employee of Sana Biotechnology. W.R.M. is a consultant for Sana Biotechnology. C.E.M. and W.R.M. hold issued and pending patents in the field of stem cell and regenerative biology.

Here is the original post:
Stem cells and the heartthe road ahead - Science Magazine

Stem Cell Therapy Market Insights 2019, is a professional and in-depth study on the current state of the global Stem Cell Therapy industry with a focus on the Global market. The report provides key statistics on the market status of the Stem Cell Therapy manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. Overall, the report provides an in-depth insight of 2019-2025 global Stem Cell Therapy market covering all important parameters.

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The key points of the Stem Cell Therapy Market report:

The report provides a basic overview of the Stem Cell Therapy industry including its definition, applications and manufacturing technology.

The report explores the international and Chinese major industry players in detail. In this part, the report presents the company profile, product specifications, capacity, production value, and 2019-2025 market shares for each company.

Through the statistical analysis, the report depicts the global total market of Stem Cell Therapy industry including capacity, production, production value, cost/profit, supply/demand and Chinese import/export.

The total market is further divided by company, by country, and by application/type for the competitive landscape analysis.

The report then estimates 2019-2025 market development trends of Stem Cell Therapy industry. Analysis of upstream raw materials, downstream demand, and current market dynamics is also carried out.

The report makes some important proposals for a new project of Stem Cell Therapy Industry before evaluating its feasibility.

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There are 3 key segments covered in this report: competitor segment, product type segment, end use/application segment.

For competitor segment, the report includes global key players of Stem Cell Therapy are included:

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: 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.

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.

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.

Customize This Report @ https://www.tmrresearch.com/sample/sample?flag=CR&rep_id=1787&source=atm

Reasons to Purchase this Report:

* Estimates 2019-2025 Stem Cell Therapy market development trends with the recent trends and SWOT analysis

* Market dynamics scenario, along with growth opportunities of the market in the years to come

* Market segmentation analysis including qualitative and quantitative research incorporating the impact of economic and policy aspects

* Regional and country level analysis integrating the demand and supply forces that are influencing the growth of the market.

* Competitive landscape involving the market share of major players, along with the new projects and strategies adopted by players in the past five years

* Comprehensive company profiles covering the product offerings, key financial information, recent developments, SWOT analysis, and strategies employed by the major market players

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Stem Cell Therapy Market: Business Opportunities, Current Trends and Industry Analysis by 2018 2028 - Instant Tech News

Were deep into half-year reporting season and a swarm of medical companies opened their books to investors overnight.

Being biotechs, some dont make money and many of those never will either, if they cant get the drug or device theyre developing to work.

Others are well on the revenue pathway.

This company is developing treatments that spur the bodys immune system to fight cancer. It technically doesnt make money per se, booking a $6m loss, but does earn money from other companies it has licensed drugs to.

In the half year, Immutep earned a milestone payment of $7.4m from GlaxoSmithKline, which dosed its first patient in a phase II clinical trial evaluating a treatment derived from an Immutep antibody in ulcerative colitis.

Immutep has licensed drugs to Novartis, CYTLIMIC, and Chinese company EOC Pharma.

Pharmaceuticals seller Mayne Pharma reported lower numbers across all metrics. Revenue was down 17 per cent, EBITDA dropped by 47 per cent, its loss widened to $17.5m, and even underlying EBITDA and operating cashflow fell too.

CEO Scott Richards said as previously foreshadowed at the AGM the company had faced aggressive competition on its key generic products in the US. Mayne Pharma cut costs by $10m and dumped some generic products.

Richards is hopeful a new oral contraceptive the company has acquired will help it bounce back.

IVF provider Monash also hasnt had a great half, with all key numbers down: revenue dipped to $77m, profit dropped 15 per cent to just under $10m, all forms of EBITDA and EBIT (there are a few ways to spin those numbers) are also lower .

IVF is a hyper competitive market in Australia. Undercut by cheap operators, who were allowed into the market a few years ago, and with strong rivals in the biggest regions of NSW, Queensland and Victoria, there arent many ways to claw back market share or grow without going overseas.

Tasmania and South Australia performed well for Monash in the half, and more women are wanting expensive genetic screening. But even the companys foray into Malaysia delivered bad news as the number of stimulated cycles women undertook fell.

Cynata is trying to cure disease with stem cells. Japanese company Sumitomo tried to buy it for $2-a-share in the half, but they couldnt agree on terms and the talks fizzled.

The company made money in the quarter because FUJIFILM Corporation paid $US3m to exercise a long-awaited licence option for a treatment for graft-versus-host disease (GvHD), a rare condition when donor bone marrow or stem cells attack their new host. However, Cynata made a $2.5m loss and has $5.9m in cash at the end of calendar 2019.

Cynata has three phase two clinical trials expected to start in 2020 for osteoarthritis, critical limb ischemia and GvHD. Its also looking at sepsis, coronary artery disease, and organ transplant rejection.

Another stem cell biotech, Exopharm listed in the prior corresponding half, so its figures are not as simple to compare.

Revenue rose 6,239 per cent to $39,494, although this is entirely from interest on money in the bank, and its loss widened to $3.7m.

The company mainly spent its money on R&D and employees.

The Alzheimers cure researcher has had to dig deep into its data following a spectacular failure of a phase two trial in May.

In the last half, Actinogen found its lead drug Xanamem produced a statistically significant clinical effect on improving cognition in healthy elderly patients at 20mg daily (rather than the lower dose in the phase two trial).

Without a clinical trial underway the loss fell from $7m to $4m, while R&D costs halved.

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Continue reading here:
Biotech: Its not a get-rich-quick scheme judging by these HY reports - Stockhead

02/21/2020 | 12:17am EST

Actinium

Pharmaceuticals,Inc.

Mid-Point Analysis of Pivotal Phase 3 SIERRA Trial Including Materials Presented at 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT)

Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] leads to High Rates of Allogeneic Transplantation and Successful Engraftment in Older Patients with Active, Relapsed or Refractory AML after Failure of Chemotherapy and Targeted Agents: Preliminary Midpoint Results from the Prospective, Randomized Phase 3 SIERRA Trial

SIERRA: Study of Iomab-B in Elderly Relapsed/Refractory AML

Boglarka Gyurkocza, MD, Rajneesh Nath, MD, Patrick J. Stiff, MD, Edward Agura, MD, Mark Litzow, MD, Ben Tomlinson, MD, Hannah K. Choe, MD, Sunil Abhyankar, MD, Stuart Seropian, MD, George L.

Chen, MD, Parameswaran Hari, MBBS, MD, Zaid S. Al-Kadhimi, MD, James M. Foran, MD, Johnnie Orozco, MD, Koen Van Besien, MD, PhD, Mitchell Sabloff, MD, Partow Kebriaei, MD, Camille Abboud, MD, Moshe Yair Levy, MD, Hillard M Lazarus, MD, Sergio A Giralt, MD, Mark S. Berger, MD, Vijay Reddy, MD, PhD and John M. Pagel, MD, PhD

2

Slides adapted from TCT 2020 oral presentation and include additional data from mid-point analysis of 50% of patients from SIERRA trial

Iodine (131I) apamistamab [Iomab-B] CD45 Targeted Conditioning

Leukemic Bone Marrow

Post-Iomab-B Myeloablated Bone Marrow

Iomab-B

SIERRA Phase 3 Trial Design

Study Design (N=150)

Primary End-point:

Durable Complete Response Rate (dCR): CR/CRp lasting 180 days

Secondary End-point:

1-year Overall Survival

SIERRA Key Eligibility Criteria

Two or more cycles of Venetoclax in combination with Azacitidine or Decitabine (newly added)

Analyzing the Inclusion of Targeted Agents in SIERRA

Therapies Prior to Enrollment into the Trial:

85% patients enrolled had failed 2 regimens (induction/re-induction)

33% had failed targeted therapies Therapies After Enrollment: Control Arm

HCT Rate After Iomab-B vs Std of Care Control

100%

(31/31)

Patients of Percentage

18% (7/38) (7/38)

Standard of Care BMT

6

SIERRA Iomab-B Treatment Schedule

Iomab-B Specific

Standard Transplant Procedure

~7 days

~12 days

Therapy Dose

RIC

HCT

Dosimetry

Iomab-B

Iomab-B

FLU

TBI

(~10-20 mCi)

(24 Gy to liver, mean~600mCi)

30 mg/m2/d

200 cGy

-12 to HCT

-4

-3

-2

-1

0

Immunosuppression

Imaging

RIC: Reduced Intensity Conditioning FLU: Fludarabine

TBI: Total Body Irradiation

HCT: Hematopoietic Cell Transplant

Therapy dose individualized and calculated based on upper limit of 24 Gy liver exposure

SIERRA trial: Demographics highlights of first 75 patients (50% Enrollment)

Phase 3 SIERRA Trial Patient Characteristics (N=75)

Randomized to Iomab-B

Randomized to Conventional Care

(N=37)

(N=38)

Age

65 (55-77)

64 (55-76)

median, (range)

Favorable: 0%

Favorable: 5%

Molecular & Cytogenetic Risk1,3

Intermediate: 34%

Intermediate: 29%

Adverse: 66%

Adverse: 66%

% Bone Marrow Blasts at

Randomization

29% (5-88)2

26% (5-97)

median, (range)

Disease Status at Randomization

Primary Induction Failure: 21 (57)

Primary Induction Failure: 18 (47)

First Early Relapse: 6 (16)

First Early Relapse: 6 (16)

N, (%)

Relapse/Refractory: 6 (16)

Relapse/Refractory: 9 (24)

2nd + Relapse: 3 (8)

2nd + Relapse: 5 (13)

# Prior Regimens at

Randomization

3 (1-5)

3 (1-5)

median, (range)

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Actinium Pharmaceuticals : Mid-Point Analysis of Pivotal Phase 3 SIERRA Trial Including Material Presented at 2020 Transplantation & Cellular...

By Lorena Anderson, UC Merced

Professor Joel Spencer and his lab have made a huge breakthrough in stem cell research.

Professor Joel Spencer was a rising star in college soccer and now he is an emerging scientist in the world of biomedical engineering, capturing for the first time an image of a hematopoietic stem cell (HSC) within the bone marrow of a living organism.

Everyone knew black holes existed, but it took until last year to directly capture an image of one due to the complexity of their environment, Spencer said. Its analogous with stem cells in the bone marrow. Until now, our understanding of HSCs has been limited by the inability to directly visualize them in their native environment.

This work brings an advancement that will open doors to understanding how these cells work which may lead to better therapeutics for hematologic disorders including cancer.

Understanding how HSCs interact within their local environments might help researchers understand how cancers use this same environment in the bone marrow to evade treatment.

Spencer studied biological sciences at UC Irvine where he was the captain of the mens Division 1 soccer team. He initially planned to pursue a career in professional soccer until faculty mentors opened doors for research and introduced Spencer to biophotonics the science that deals with the interactions of light with biological matter.

UC faculty were a big part of my research experience; they became mentors and friends, Spencer said. My first foray into research was as a lab tech, and that is where I met people who were doing biomedical imaging, and it just caught my wonder.

An image of a stem cell in its natural niche

Spencer left his native California to earn his Ph.D. in bioengineering at Tufts University in Boston and took a postdoctoral research position in the Wellman Center for Photomedicine at Massachusetts General Hospital and Harvard Medical School. In Boston, he learned about live-animal imaging and his wonder became a passion.

Now his emphasis is on biomedical optics: building new microscopes and new imaging techniques to visualize and study biological molecules, cells and tissue in their natural niches in living, fully intact small animals.

I work at the interface of engineering and biology. My lab is seeking to answer biological questions that were impossible until the advancements in technology we have seen in the past couple decades, he said. You need to be able to peer inside an organ inside a live animal and see whats happening as it happens.

Based on work conducted at UC Merced and in Boston, he and his collaborators including his grad student Negar Tehrani visualized stem cells inside the bone marrow of live, intact mice.

He and his collaborators have a new paper published in the journal Nature detailing the work they conducted to study HSCs in their native environment in the bone marrow.

We can see how the cells behave in their native niches and how they respond to injuries or stresses which seems to be connected to the constant process of bone remodeling, Tehrani said. Researchers have been trying to answer questions that have gone unanswered for lack of technology, and they have turned to engineering to solve those puzzles.

Its important for researchers to understand the mechanics of stem cells because of the cells potential to regenerate and repair damaged tissue.

Spencer, left, and students from his lab

Spencer returned to California three years ago, joining the Department of Bioengineering in the School of Engineering at UC Merced. Hes also an affiliate of the Health Sciences Research Institute and the NSF CREST Center for Cellular and Biomolecular Machines . This is his third paper in Nature, but the first stemming from work conducted in his current lab.

He didnt come to UC Merced just because he loves biology Spencer also joined the campus because of the students.

Now Im back in the UC system Im a homegrown UC student whos now faculty, Spencer said. As a student within the system I was able to participate in myriad opportunities, including mentorships that advanced my career. Now I try to encourage graduate and undergrad students to follow their dreams. I love being able to give them opportunities its something I really want to do for the next generation.

More here:
Breakthrough in Stem Cell Research: First Image of Niche Environment | Newsroom - UC Merced University News

Nes-Ziona, Israel, Feb. 20, 2020 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV), a clinical-stage immunotherapy company, today announced that the company wasselected, for a scientific presentation of two posters: (i) Allocetra-OTS: Early Apoptotic Cells for Immune Homeostasis in Human Stem Cell Transplantation (HSCT) and for the Prevention of Graft Versus Host Disease (GvHD), and (ii) Apoptotic Cells Reprogram Resident Macrophages to Support Chimeric Antigen Receptor (CAR) T Cell Therapy Against Peritoneal Solid Tumor, at the Transplantation & Cellular Therapy Meetings Conference of the ASTCT and CIBMTR (TCT), held on February 19-23, 2020, in Orlando, Florida.

Allocetra-OTS: Early Apoptotic Cells for Immune Homeostasis in Human Stem Cell Transplantation (HSCT) and for the Prevention of Graft Versus Host Disease (GvHD)

Results from preclinical and clinical studiesy suggested that a single infusion of donor early apoptotic cells (Allocetra) as prophylaxis for GvHD in myeloablative HSCT is safe and potentially effective and led to 0% (0/6) of acute high grade II-IV GvHD in the two higher dosages compared to 52% in matched historical control. Enlivex is planning to initiate a Phase 2/3 multi-center, open-label, 2-arm study (ENX-CL-01-002), in Israel and Germany, that will evaluate the efficacy and safety of Allocetra-OTS (140x106cells/kg) with or without anti-thymocyte globulin (ATG) for the prevention of GvHD in subjects undergoing HLA-matched HSCT from an unrelated donor.

Apoptotic Cells Reprogram Resident Macrophages to Support Chimeric Antigen Receptor (CAR) T Cell Therapy Against Peritoneal Solid Tumor

Preclinical studies showed significantly increased duration of survival and overall survival for study subjects who were treated with the combination therapy, as compared to stand-alone solid tumor CAR-T therapy. The results of these preclinical studies showed that the mechanism of action significantly increased the anti-tumor macrophage population surrounding the human solid tumor microenvironment in the subjects who were treated with the combination therapy.

ALLOCETRATMby Enlivex was designed toprovide a novel immunotherapy mechanism of actionthat targets life-threatening clinical indications that are defined as unmet medical needs, includingprevention or treatment of complications associated with bone marrow transplantations (BMT) and/or hematopoietic stem cell transplantations (HSCT); organ dysfunction and acute multiple organ failure associated with sepsis; and enablement of an effective treatment of solid tumors via immune checkpoint rebalancing.

ABOUT ENLIVEXEnlivex is a clinical stage immunotherapy company, developing an allogeneic drug pipeline for immune system rebalancing. Immune system rebalancing is critical for the treatment of life-threatening immune and inflammatory conditions which involve an out of control immune system (e.g. Cytokine Release Syndrome) and for which there are no approved treatments (unmet medical needs), as well as solid tumors immune-checkpoint rebalancing. For more information, visit http://www.enlivex.com.

ABOUT EUROPEAN MOLECULAR BIOLOGY ORGANIZATIONThe TCT | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT Meetings) are the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

Safe Harbor Statement: This press release contains forward-looking statements, which may be identified by words such as expects, plans, projects, will, may, anticipates, believes, should, would, intends, estimates, suggests, has the potential to and other words of similar meaning, including statements regarding expected cash balances, market opportunitiesfor the results of current clinical studies and preclinical experiments, the effectiveness of, and market opportunitiesfor, ALLOCETRATMprograms, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that forward-looking statements involve risks and uncertainties that may affect Enlivexs business and prospects, including the risks that Enlivex may not succeed in generating any revenues or developing any commercial products; that the products in development may fail, may not achieve the expected results or effectiveness and/or may not generate data that would support the approval or marketing of these products for the indications being studied or for other indications; that ongoing studies may not continue to show substantial or any activity; and other risks and uncertainties that may cause results to differ materially from those set forth in the forward-looking statements. The results of clinical trials in humans may produce results that differ significantly from the results of clinical and other trials in animals. The results of early-stage trials may differ significantly from the results of more developed, later-stage trials. The development of any products using the ALLOCETRATMproduct line could also be affected by a number of other factors, including unexpected safety, efficacy or manufacturing issues, additional time requirements for data analyses and decision making, the impact of pharmaceutical industry regulation, the impact of competitive products and pricing and the impact of patents and other proprietary rights held by competitors and other third parties. In addition to the risk factors described above, investors should consider the economic, competitive, governmental, technological and other factors discussed in Enlivexs filings with the Securities and Exchange Commission, including under the heading Risk Factors contained in Enlivexs most recently filed Annual Report on Form 20-F. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements, except as required under applicable law.

ENLIVEX CONTACT: Shachar Shlosberger, CFO Enlivex Therapeutics, Ltd.shachar@enlivex-pharm.com

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Data On Enlivex's Allocetra-OTS Immunotherapy for Peritoneal Solid Tumors and for Prevention of GvHD Selected for Presentation at the Transplantation...

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Latest Research By Business Expansion Plans, Industry Demand Status &...

Conference Call and Webcast @ 8:00 a.m. Eastern Time Today

NEW YORK, Feb. 18, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announces financial results for fiscal year ended December 31, 2019.

2019 was a tremendous year for BrainStorm, with significant progress and achievements across all clinical and operational fronts, stated Chaim Lebovits, President and Chief Executive Officer of BrainStorm. Most importantly, we fully enrolled our pivotal, double blind, placebo-controlled Phase 3 trial of NurOwn for the treatment of ALS. We announced the trial conducted at six major U.S. medical centers of excellence for ALS, was fully enrolled on October 11, 2019, and on October 28, 2019 the Data and Safety Monitoring Board (DSMB), completed the second planned interim safety analysis for the first 106 patients who received repeat dosing of NurOwn in the Phase 3 trial. The DSMB concluded the trial should continue as planned without any clinical protocol changes. He added, In addition, one of the most prestigious peer-reviewed journals, Neurology, published NurOwn Phase 2 Randomized Clinical Trial in ALS: Safety, Clinical and BioMarker Results, bringing news of our investigational therapy to the global scientific community. And, just last week, we were happy to announce that the Company recently held a high level meeting with the U.S. Food and Drug Administration (FDA) to discuss potential NurOwn regulatory pathways for approval in ALS.

Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer of BrainStorm added, 2019 was also a very significant year for those who suffer from progressive Multiple Sclerosis (MS). In February 2019, we announced Cleveland Clinic would serve as our first contracted site for a Phase 2 open-label, multicenter study of repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS (NCT03799718). We enrolled our first patient in March. We contracted with The Stanford University School of Medicine, The Keck School of Medicine of the University of Southern California, and the Mount Sinai Medical Center to further enroll patients. Dr. Kern added, The importance of our research in progressive MS was acknowledged by a $495,000 grant award from the National Multiple Sclerosis Society through its Fast Forward Program, and mid-December, the Data Safety Monitoring Board completed the first, pre-specified interim analysis, of safety outcomes for 9 participants and after careful review of all available clinical trial data, the DSMB unanimously concluded that the study should continue as planned without any protocol modification. As of December 31, 2019 we have enrolled 10 patients in the study (50% enrollment completed).

Fourth Quarter Corporate Highlights:

Financial Results for the Year Ended December 31, 2019 and Recent Updates

For further details on BrainStorms financials, including financial results for the year ended December 31, 2019, refer to the Form 10-K filed with the SEC today.

Conference Call on Tuesday, February 18th @ 8:00 am Eastern Time

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

Story continues

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. Brainstorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. Brainstorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

Safe-Harbor StatementStatements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: 862-397-8160pshah@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Announces Operational Highlights and Financial Results for the Year Ended December 31, 2019 - Yahoo Finance

Medical treatment involving stem cells is an ever-growing, billion-dollar industry, so chances are you have heard about it in the news. Here in the U.S. and around the world, stem cells are being used in various therapies to treat a wide variety of health problems and diseases, including dementia, autism, multiple sclerosis, cerebral palsy, osteoarthritis, cancer, heart disease, Parkinsons disease, and spinal cord injury. Treatments for such health issues may sound promising, but the risk is many of those being sold and advertised arent yet proven to be safe and effective. This is why its so important to educate yourself before jumping into any kind of stem cell treatment.

To gain a better understanding of this new age of medical research, one must first understand what stem cells are and how they work. Stem cells are special human cells that can develop into many different types of cells. They can divide and produce more of the same type of stem cells, or they can turn into different functioning cells. There are no other types of cells in the body that have this natural ability to generate new cell types.

So where do stem cells that are used for research and medical treatments come from? The three main types of stem cells are embryonic (or pluripotent) stem cells, adult stem cells, and induced pluripotent stem cells.

Embryonic stem cells come from unused, in vitro fertilized embryos that are three to five days old. The embryos are only donated for research purposes with the informed consent of the donors. Embryonic stem cells are pluripotent, which means they can turn into any cell type in the body.

Adult stem cells are found in small numbers in developed tissues in different parts of the body, such as bone marrow, skin, and the brain. They are specific to a certain kind of tissue in the body and are limited to maintaining and repairing the tissue in which they are found. For example, liver stem cells can only make new liver tissue; they arent able to make new muscle tissue.

Induced pluripotent stem cells are another form of adult stem cells. These are stem cells that have been manipulated in a laboratory and reprogrammed to work like embryotic (or pluripotent) stem cells. While these altered adult stem cells dont appear to be clinically different from embryonic stem cells, research is still being conducted to determine if the effects they have on humans differ from actual embryonic stem cells.

Stem cells can also be found in amniotic fluid and umbilical cord blood. These stem cells have the ability to change into specialized cells like embryonic stem cells. While more research is being conducted to determine the potential of these types of stem cells, researchers already actively use these through amniocentesis procedures. In this procedure, the stem cells drawn from amniotic fluid samples of pregnant women can be screened for developmental abnormalities in a fetus.

The main difference between embryonic and adult stem cells is how they function. Embryonic stem cells are more versatile. Since they can divide into more stem cells or become any type of cell in the body, they can be used to regenerate or repair a variety of diseased tissue and organs. Adult stem cells only generate the types of cells from where they are taken from in the body.

The ability for stem cells to regenerate under the right conditions in the body or in a laboratory is why researchers and doctors have become so interested in studying them. Stem cell research is helping scientists and doctors to better understand how certain diseases occur, how to possibly generate healthy cells to replace diseased cells, and offer ways to test new drugs.

Clearly, stem cell research is showing great potential for understanding and treating a range of diseases and other health issues, but there is still a lot to learn. While there are some diseases that are showing success using stem cell treatments, many others are yet to be proven in clinical trials and should be considered highly experimental.

In our next article, various stem cell treatments, FDA regulations, and other stem cell hot topics will be explored. It will also focus on what to look for when considering stem cell therapies so people arent misled or misinformed about the benefits and risks.

For more information regarding the basics of stem cells visit these sites:

https://stemcells.nih.gov/info/basics/1.htm

https://www.mayoclinic.org/tests-procedures/bone-marrow-transplant/in-depth/stem-cells/art-20048117

Continued here:
The 411 on Stem Cells: What They Are and Why It's Important to Be Educated - Legal Examiner

DURHAM, N.C., Feb. 18, 2020 (GLOBE NEWSWIRE) -- Chimerix(CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that it will host a live conference call and audio webcast on Tuesday, February 25, 2020 at 8:30 a.m. ET to report financial results for the fourth quarter and full-year ended December 31, 2019, and to provide a business overview.

To access the live conference call, please dial (877) 354-4056 (domestic) or (678) 809-1043 (international) at least five minutes prior to the start time, and refer to conference ID 1397800. A live audio webcast of the call will also be available on the Investors section of the Company's website, http://www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event.

AboutChimerix

Chimerix is a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a potential first-in-class glycosaminoglycan compound derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1, elastase). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggest that DSTAT may also accelerate platelet recovery post-chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is an antiviral drug candidate in development as a medical countermeasure for smallpox. For further information, please visit the Chimerix website, http://www.chimerix.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things, Chimerixs ability to develop BCV as a medical countermeasure for smallpox; Chimerixs ability to submit and/or obtain regulatory approvals for BCV; and Chimerixs ability to enter into a procurement contract for BCV as a medical countermeasure. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that BCV may not obtain regulatory approval from theFDAor such approval may be delayed or conditioned; risks that development activities related to BCV may not be completed on time or at all; Chimerixs reliance on a sole source third-party manufacturer for drug supply; risks that ongoing or future trials may not be successful or replicate previous trial results, or may not be predictive of real-world results or of results in subsequent trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for our drugs; risks that our drugs may be precluded from commercialization by the proprietary rights of third parties; risks related to procurement of brincidofovir for the treatment of smallpox and additional risks set forth in the Company's filings with theSecurities and Exchange Commission. These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements.

CONTACT:Investor Relations: Michelle LaSpaluto919 972-7115ir@chimerix.com

Will OConnorStern Investor Relationswill@sternir.com 212-362-1200

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Chimerix to Report Fourth Quarter and Year End 2019 Financial Results and Provide an Operational Update on February 25, 2020 - Yahoo Finance

NEW YORK, Feb. 18, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announces financial results for fiscal year ended December 31, 2019.

2019 was a tremendous year for BrainStorm, with significant progress and achievements across all clinical and operational fronts, stated Chaim Lebovits, President and Chief Executive Officer of BrainStorm. Most importantly, we fully enrolled our pivotal, double blind, placebo-controlled Phase 3 trial of NurOwn for the treatment of ALS. We announced the trial conducted at six major U.S. medical centers of excellence for ALS, was fully enrolled on October 11, 2019, and on October 28, 2019 the Data and Safety Monitoring Board (DSMB), completed the second planned interim safety analysis for the first 106 patients who received repeat dosing of NurOwn in the Phase 3 trial. The DSMB concluded the trial should continue as planned without any clinical protocol changes. He added, In addition, one of the most prestigious peer-reviewed journals, Neurology, published NurOwn Phase 2 Randomized Clinical Trial in ALS: Safety, Clinical and BioMarker Results, bringing news of our investigational therapy to the global scientific community. And, just last week, we were happy to announce that the Company recently held a high level meeting with the U.S. Food and Drug Administration (FDA) to discuss potential NurOwn regulatory pathways for approval in ALS.

Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer of BrainStorm added, 2019 was also a very significant year for those who suffer from progressive Multiple Sclerosis (MS). In February 2019, we announced Cleveland Clinic would serve as our first contracted site for a Phase 2 open-label, multicenter study of repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS (NCT03799718). We enrolled our first patient in March. We contracted with The Stanford University School of Medicine, The Keck School of Medicine of the University of Southern California, and the Mount Sinai Medical Center to further enroll patients. Dr. Kern added, The importance of our research in progressive MS was acknowledged by a $495,000 grant award from the National Multiple Sclerosis Society through its Fast Forward Program, and mid-December, the Data Safety Monitoring Board completed the first, pre-specified interim analysis, of safety outcomes for 9 participants and after careful review of all available clinical trial data, the DSMB unanimously concluded that the study should continue as planned without any protocol modification. As of December 31, 2019 we have enrolled 10 patients in the study (50% enrollment completed).

Fourth Quarter Corporate Highlights:

Financial Results for the Year Ended December 31, 2019 and Recent Updates

For further details on BrainStorms financials, including financial results for the year ended December 31, 2019, refer to the Form 10-K filed with the SEC today.

Conference Call on Tuesday, February 18th @ 8:00 am Eastern Time

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. Brainstorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. Brainstorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

Safe-Harbor StatementStatements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: 862-397-8160pshah@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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SIOUX CITY (KTIV) - For five years now, the Ruehle family has held a teddy bear drive in memory of Mike Ruehle, who passed from cancer in 2014.

The teddy bears are given to adults and children with a cancer connection, in Mike's memory.

Family and friends knew Mike as "Bear."

The family asks people to consider donating a teddy bear in memory of a loved one, or in honor of healthy family members.

If you'd like to donate, every bear must be new with the tag still attached. The donations will stay in Siouxland.

There are five different locations where you can drop off the bears:

Ruehle's family says the teddy bear drive has donated more than 3,000 stuffed animals to several Siouxland agencies.

But who is the man who inspired the teddy bear drive?

"Very thoughtful person, very giving, he always took the time to listen to other people, just a really compassionate, kindhearted person," said Kerry Ruehle, Mike's Widow.

Mike Ruehle, or as his family called him, Bear, passed away six years ago, after a 12 year battle with cancer.

The family said he was very active in the community and was often coaching numerous sports teams.

They said he had a big heart, and always did what he could to make others feel better, even while he himself had cancer.

"If there were any patients his doctor had, who were having a difficult time with the news or with the adjustment. He would reach out to my dad, and see if my dad would sit down with them and talk about what was going on with them," said Rhett, Mike's son.

Part of the reason the teddy bear drive was started was because of Bear's compassion towards children who were also dealing with cancer.

"He would see a young child going through similar things that he was going through. He would always go out of his way to talk to that little kid and brighten their day a little bit," said Rhett.

Mike had had three different kinds of cancers, and due to the chemotherapy, eventually was diagnosed with MDS, which is a bone marrow failure disorder.

Doctors determined that the best treatment would be a stem cell transplant. The transplant surgery went well, but months later his body began rejecting his brother's stem cells, and within five weeks he had passed away.

"It was a shock because he had been ahead of schedule and everything else. And he had come through things beautifully, so it was quite a shock. But it was god's plan I guess," said Kerry.

But Mike's family wanted a way to keep Mike's memory alive, and that's what also helped start the teddy bear drive.

"My oldest granddaughter is five, she never met her grandpa. But in some ways, she feels as though she did, because of talking about him and she helps me with the bear drive," said Kerry

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Family honors the memory of loved one by collecting teddy bears for families dealing with cancer - KTIV

The latest market report published by Credence Research, Inc.GlobalBone Marrow Transplantation Market Growth, Future Prospects, Competitive Analysis, 2018 2026,

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Market Insights:

Blood cancer is characterized by overproduction of an irregular type of blood cells, resulting in the overproduction of normal cells in the bone marrow preventing normal cells from performing important functions. According to the study references issued by Bristol-Myers Squibb Company by 2040 nearly 1,100,000 people will die from blood cancer, which will account for 7 percent cancer deaths.

Allogeneic bone marrow transplant are reigning the market. The inherent features associated with allogeneic procedures are the graft is free from contaminated tumor cells. Immune graft versus malignancy effect is produced by the immunocompetent cells derived from healthy donor. Low risk of disease recurrence and significant rise in the number of healthy donors drive the allogeneic bone marrow transplantation market growth. Autologous procedure will grow at a rapid pace in the near future due to rise in number of stem cell banks, which store healthy cells from patients and after conditioning treatment are introduced in them to produce healthy blood cells.

Lymphoma is dominating the indication segment for bone marrow transplantation market. There are 2 types Hodgkin lymphoma and Non-Hodgkin lymphoma. The primary factors responsible for its supremacy are significant rise in the patients newly diagnosed with Hodgkin lymphoma and excellent survival rate in patients post bone marrow transplantation procedure. Leukemia is the abnormal high production of white blood cells by the bone marrow. Technological advancement in the bone marrow transplantation technique will reduce the disease recurrence in leukemia patients receiving stem cell therapy.

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North America is presently the leading regional segment for bone marrow transplantation market with a market share of 38%. The major factors responsible for its significant growth are rising prevalence of chronic lymphocytic leukemia and presence of sophisticated healthcare infrastructure. Europe with a market share of 32% is at second position owing to significant rise in the number of myeloma patients and domicile of major players such as PromoCell GmbH, Merck Millipore Corporation and Lonza Group Ltd. Asia Pacific with a market share of 20% will grow at a faster pace in the near future on account of technological advancement in bone marrow transplantation technique and proactive government policies to curb the mortality rate associated with blood cancer.

Biotechnology firms actively engaged in bone marrow transplantation procedures are AllCells LLC., Conversant Bio., Cellular Dynamics International, Gamida Cell Ltd., Hemacare Corporation, Lonza Group Ltd., Merck Millipore Corporation, Mesoblast Ltd., PromoCell GmbH and STEMCELL Technologies.

Key Market Movements:

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Credence Researchis aworldwide market research and counseling firm that serves drivingorganizations, governments, non-legislative associations, and not-for-benefits.We offer our customers some assistance with making enduring enhancements totheir execution and understand their most imperative objectives. Over almost acentury, weve manufactured a firm extraordinarily prepared to this task.

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Bone Marrow Transplantation Market: Lower risk of disease recurrence and significant rise in the number of healthy donors drive the market growth -...