Archive for the ‘Bone Marrow Stem Cells’ Category

Sensational 8-Year-Old Violinist Living With Painful Disease

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of two.*

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

GoFundMe

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

Every month, Caesar and his family travel to Charlotte for blood transfusions to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.*

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

*This story has been updated to correct information. Lessons for Caesar started at the age of 2 and 300ml of his blood is replaced every month during his blood transfusions.

5 Facts About Sickle Cell Disease (CDC)

More:
Sensational 8-year-old violinist living with painful disease - 13newsnow.com

Technology Networks

Stems Cells Could Help Treat Slipped Discs Technology Networks The study on the sick German shepherds was organized as follows: With the permission of the dog owners, neurologist Frank Steffen and his team removed stem cells from the marrow of the pelvic bone of the affected animals. After the cleaning and ... and more »

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Stems Cells Could Help Treat Slipped Discs - Technology Networks

Iran is expanding investment in stem cell research and its application in various therapies, particularly for hard-to-treat diseases, through the Office of the Vice- Presidency for Science and Technology. As a result of the increase in the number of companies active in the domain of stem cells in the past three years, more than 400 products are processed in the country, indicating a multifold growth compared with the eight-year tenure of previous administration when there were fewer than 50 knowledge-based firms in total.

There are over 40 knowledgebased firms in the field of stem cell and regenerative medicine alone in Iran today, said Amir Ali Hamidiyeh, secretary of the Headquarters for Development of Stem Cell Science and Technology (HDSCST). He made the statement at a press briefing for the second National Festival and International Congress on Stem Cell Sciences and Technologies and Regenerative Medicine to be held July 13- 15 in Tehran, Mehr News Agency reports. According to the conference secretariat, 1,444 people have signed up to attend the event from across the world, including from Iraq, India, Pakistan, Jordan, Russia, Australia, Germany, China, Britain and South Korea. They all are among their countrys respected figures in centers with high academic standing.

The congress is co-sponsored by the Vice-Presidency for Science and Technology and Council on Development of Stem Cell Sciences and Technology. So far, eight stem-cell therapy products for use in hospitals have been produced at the HDSCST laboratories. Manufacturinglicenses have been granted for anadditional number, while others are on thewait list.

Prior to 2014, only 25 knowledgebased companies had applied to operate in this field, of which only one was actively producing quality stem cell products, Hamidiyeh pointed out.

But since then, over 25 workgroups have been formed in cooperation with experts in the specific sciences. Stem cells are cells that have the ability to divide and develop into many different cell types in the body during early life and growth. Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

Future of Medicine in Stem

Cells The future of medicine is interrelated with stem cell therapy and the treatment ofrefractory and incurable diseases is in this field of medicine, according to Dr. Ahmad Vosouq Dizaj, the clinical deputy of Royan Institute. Having access to engineering sciences as well as the combination of biology and medicine can play a crucial role in redressing health problems, he said. Stem cells have the ability to replace damaged cells and treat disease. They can also be used to study diseases and provide a resource for testing new medical treatments. The use of stem cells reduces the risk of viral diseases transmission and incidence of Graft Versus Host Disease (GVHD). The ability to perform organ transplants is among the benefits ofumbilical cord blood transfusion.Using stems cells is also one of thebest ways to treat blood diseases sincethe method has a success rate of 70%worldwide.

Storage of stem cells is a valuable investment. So far, 27 cord blood banks have been launched across the country. There are two types: public and private banks for stem cell storage. The former does not charge a fee for storage. But in the latter, the cost of collection and genetictesting is about $645 and the annualcharge for storage is $33, according toISNA.Iran is a leading country in biomedicalresearch. Researchers and physicians have been successfully performing bone marrow transplants during the past fewyears.Irans stem cell research is centeredat the Royan Institute for ReproductiveBiomedicine, Stem Cell Biology andTechnology, located in northern Tehran.

See original here:
Growth in Stem Cell Research - Financial Tribune

Lab-engineered bone (the outer layer) with functional bone marrow (the inner layer). Image: Varghese Lab at UC San Diego

Thanks to advances in medicine, bone marrow transplants are no longer the last resorts they once were. Every year, thousands of marrow transplants are performed, a common treatment for ailments from bone marrow disease to leukemia. But because they first require a patient undergo radiation to kill off any existing bone marrow stem cells, marrow transplants remain incredibly hard on a patient.

Now, engineers at the University of California San Diego have developed a synthetic bone implant with functional marrow able to produce its own blood cells. So far, researchers revealed in a paper published in the Proceedings of the National Academy of Sciencesthis week, they have successfully tested the engineered bone tissues in mice. But one day, those biomimetic bone tissues could provide new bone marrow for human patients in need of transplants, too.

The implant does away with the need for radiation by giving donor cells their own space in the body to grow. Inside the implant, there is no threat of those cells being overtaken by the bodys native stem cells.

In mice, the researchers implanted the synthetic bone tissues with functional marrow under the skin. After six months, those donor cells were still alive and had begun supplying the mice with new blood cells.

The implants were designed to replicate the long bones in the body, with an outer bone compartment containing calcium phosphate minerals to build bone cells, and an inner area for donor stem cells that produce blood cells.

When implanted, they grew into bone tissues with working blood vessel network and functional bone marrow that supplied the body new blood cells. After 24 weeks, researchers found a mix of host and donor blood cells was still circulating in the bloodstream of the mice.

A treatment based on this technology would only work for patients with non-malignant bone marrow diseases, like aplastic anemia, a condition where the body cant make enough platelets and blood cells. Thats because while the technique can replenish types of cells that are lacking, it cant doing anything to fight off cells that have mutated and are spreading. Cancer patients would still need need to undergo radiation therapy to have their cancerous cells wiped out.

Much more research is needed, of course, before these implants are ready to make their way into human patients. But whats exciting here is that the synthetic bone tissues were not only functional, they allowed donor marrow to grow and survive for many weeks in the presence of host cells, and for the products of that marrow to make their way into the bodys circulatory system. Pretty neat.

See the article here:
This Synthetic Bone Implant Could Replace Painful Marrow Transplants - Gizmodo

Sensational 8-Year-Old Violinist Living With Painful Disease

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

Child Prodigy

We only listen to classical music at home," said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of two.*

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

GoFundMe

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

A Painful Disease

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

A Small Miracle

Every month, Caesar and his family travel to Charlotte for blood transfusions to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.*

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Next Step

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

*This story has been updated to correct information. Lessons for Caesar started at the age of 2 and 300ml of his blood is replaced every month during his blood transfusions.

5 Facts About Sickle Cell Disease (CDC)

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Sensational 8-Year-Old Violinist Living With Painful Disease | KSDK ... - KSDK

Lucknow: In what may come as a relief to over 1 lakh patients of thalassemia in India, a public sector stem cell bank is set to come up at UP's King George's Medical University here. A project of the university's transfusion medicine department, the stem cell bank would roll out stem cell therapy to patients of thalassemia and sickle cell anaemia. The proposal is awaiting clearance from state department of medical education.

Stem cells are omnipotent and can take shape of any cell inside the body. If infused in the pancreas, stem cells will become pancreatic while in the liver, they will become liver cells.

These are found in human bone marrow and can be derived from the umbilical cord which contains blood vessels that connect baby in the womb to the mother to ingest nutrition required for development.

Research on the therapeutic use of stem cells is underway in US, Europe, China, South East Asia besides India. In UP, Sanjay Gandhi Post Graduate Institute of Medical Sciences (SGPGIMS) and KGMU are both trying to explore the potential of stem cells to treat various health problems. SGPGI has, so far, restricted itself to use of allogenic (stem cells derived from bone marrow of a person), while KGMU has used stem cells derived from the umbilical cord.

Head of transfusion medicine department of KGMU, Prof Tulika Chandra said, "Several private sector stem cell banks like Life Cell and Cord Life India are operating in India but they serve only those who have deposited the baby's cord, while our bank will help everyone."

KGMU has sustained access to umbilical cord because of a very developed obstetrics and gynaecology department. The cord is gathered from the placenta in the uterus of pregnant women which nourishes and maintains the baby through the umbilical cord.

Sources in medical education department said the proposal is worth Rs 9 crore including infrastructure cost. "Stem cell bank promises to become financially self-sustaining within 2-3 years of inception," said a directorate officer.

Talking about why children with thalassemia and sickle cell anaemia were chosen, Chandra said, "Global literature shows umbilical cord stem cells can induce extraordinary results on such children. In fact, success rate is around 70-75% and higher score can be achieved if therapy is provided at an earlier age."

See the original post:
First public sector stem cell bank to come up at KGMU - Times of India

Its our goal for this to be a normal NHS procedure, so everyone who has a heart problem [and could benefit from this] will be able to. There are few downsides because theres no rejection as theyre your own stem cells, and every patient who has successfully had this treatment ends up taking less medication.

Jenifer is overjoyed with the progress already made, and knows that Ian would be, too, had he lived to tell his story.

For Ian, the treatment gave him an extra three years of life, but in 2006 he died from heart failure, at the age of 70.

He would be so thrilled, says Jenifer. His concern would be were not doing it quick enough, because for him everything had to be done immediately. But to have achieved this much well, the medical world says weve done it all in a very short space of time.

The couple spent their final years together alternating between their family home in St Johns Wood, north London, and a holiday home in Miami.

They were both each others second spouses, having married in 1980 after a whirlwind romance in Cannes Jenifers first husband had died, while Ian had divorced his wife and did not have children together. But Ian had two children from his first marriage, as well as two young grandchildren who he was able to spend those extra three years with.

Continue reading here:
My husband's heart failure inspired a life-saving stem cell therapy - Telegraph.co.uk

So how did they come together? It was less than 3 years ago that Edwards received the toughest news anyone can receive from a doctor.

"I was then diagnosed with leukemia, a rare form of leukemia," said Edwards.

The treatment for this rare form of blood cancer included multiple rounds of chemotherapy and radiation.

"All in all, it was enough toxins to kill a person if you ask me," said Edwards.

Edwards was also hoping to find help from someone else's blood.

"We started the search through Delete Blood Cancer and found a match," said Edwards.

The goal was to find a donor with a similar genetic makeup who could give Edwards their stem cells.

"We tried to match my brother and sister, but unfortunately there were not. So, we kept the search until we could find a match. It was a little nerve-racking, said Edwards.

That's where Halfkann comes in.

"I got a letter that I can be a stem cell donor, and I must go to the clinic in Cologne," said Halfkann.

Halfkann was already previously registered having signed up after one of her coworkers became ill. Although no successful matches were found back in Germany, in Minnesota, Halfkann was exactly who Merissa was looking for.

"Daniela is the only match in the world," said Edwards.

The news that Halfkann could save a stranger's life in the United States delighted the soft-spoken German.

"I'm so happy. I'm grateful," said Halfkann.

The stem cell procedure was pretty simple. Daniela donated blood. The stem cells were filtered out, then sent to Merissa in Minnesota where they were injected.

"There's a lot of complications after the stem cell transplant that could've gone wrong. Fortunately it didn't, which made Daniela an even more perfect match than she already is," said Edwards.

When Edwards heard about the woman who extended her life, she connected with Halfkann online.

"At first we wrote email, and then we connected on Facebook," said Halfkann.

After just a few notes, it was quickly discovered that the two have more in common than the blood running through their veins.

"We like a lot of the same things. Both have 2 children. Both of our husbands are firefighters," said Edwards.

And Edwards continues to successfully battle cancer.

"Right now I am in remission. That doesn't mean that I'll necessarily be cancer-free, but knock on wood...that's the goal...that the cancer will never come back," said Edwards.

There was only one thing left for Edwards to do; meet the woman and family that saved her life. So just a few weeks ago, the pair met for the very first time at Duluth International Airport.

"She is so nice. She is so lovely. I'm so happy we can be here," said Halfkann.

In the ten days together, they and their families created many memories. Halfkann got a glimpse of the life Edwards is now able to hold on to, and it wasn't long before the pair found more in common.

"We seem to like the same things...fruity tea, crafting, sewing, just similar interests in hobbies. Another common interest, shoes," said Edwards.

Both husbands also enjoyed their time together. At the firehouse, Merissa's husband, Dennis, giving Daniela's husband, Stefan, a tour of some of the American rigs and a ride along during an emergency call.

Back at headquarters, the crew made a home-cooked dinner for Halfkann's family and someone else who helped make all of this happen: Amanda Schamper, a representative of the registry that matched Edwards and Halfkann.

"What we try to do is to raise awareness in all communities that this is a problem out there. People are searching for their donor match and can't find one," Schamper.

Schamper also showed everyone just how easy it is to sign up to be a bone marrow and stem cell donor.

"We do have a statistic that nearly 14,000 patients are told that they needed a transplant each year, and less than half can't get one because they can't find a donor match on the registry, said Schamper.

During the visit, Edward's extended family threw a get-together in honor of Halfkann. Edward's sister-in-law Kris Hansen is just as grateful.

"Just to know that she's here and they've met each other, and that she can save a life...it's incredible. It's nice to be able to see her and her family and her two adorable daughters," said Hansen.

Through the countless hugs at the party, family members repeated one phrase that transcends all languages.

"I guess the biggest thing we have to say is Danka Daniella!" said Hansen.

"Thank you for saving my life. Thank you for letting me be a Mom. Thank you for coming here so I can meet you and meet your beautiful children and your husband," Edwards said to Halfkann.

And with thanks, comes gratitude.

"I'll forever be grateful to you. You will always be a part of my family." said Edwards.

And this bond that will last a lifetime.

"We're forever connected," said Edwards.

"Yes. Forever," said Halfkann.

Edwards says she and her family are making plans to visit the Halfkann's in Germany.

If you're interested in signing up to become a bone marrow or stem cell donor, it's free and only takes a few moments. A link to that website can be found here.

Follow this link:
Duluth Woman Meets, Finds Similarities with Stem Cell Donor - WDIO-TV

Hope Ford, WTSP 5:39 PM. EDT May 08, 2017

Caesar Sant

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

Child Prodigy

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of four.

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

GoFundMe

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

A Painful Disease

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

A Small Miracle

Every month, Caesar and his family travel to Charlotte for blood transfusions. 90 to 95 percent of his blood is replaced every month to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Next Step

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

2017 WFMY-TV

Continued here:
Sensational 8-Year-Old Violinist Living With Painful Disease - 13WMAZ

Hope Ford, WFMY 12:26 PM. EDT May 07, 2017

Caesar Sant

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

Child Prodigy

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of four.

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

A Painful Disease

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

A Small Miracle

Every month, Caesar and his family travel to Charlotte for blood transfusions. 90 to 95 percent of his blood is replaced every month to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Next Step

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

2017 WFMY-TV

Read more here:
Sensational 8-Year-Old Violinist Living With Painful Disease - WFMY News 2

Most of the Gulfs thalassemia sufferers can now be cured of the debilitating blood disease through a safe and effective bone marrow transplant procedure performed in the US, said one of the worlds leading pediatric hematologists, ahead of International Thalassemia Day on May 8.

Thalassemia is a genetic blood disease, common across the wider Middle East and South Asia, in which victims are not able to make enough hemoglobin a necessary component in healthy red blood cells, carrying oxygen to all parts of the body and, thus, suffer from severe anemia and eventual organ failure, and, ultimately, premature death.

The condition is typically treated with life-long, cost-prohibitive supportive care, with most thalassemia sufferers dying before the age of 40. However, the latest advances in bone marrow transplantation significantly reduce both treatment time and cost, giving Gulf thalassemia patients and their families new hope, said a statement.

With thalassemia, we want to treat the underlying disease, not just the symptoms, and this approach requires bone marrow transplantation, said Dr Rabi Hanna, pediatric oncologist at US-based Cleveland Clinic.

Now, finding a matching bone marrow donor is much easier, as we only require a haplo-donor half-match, meaning every patient can find a donor (father, mother or half-sibling), as opposed to only 25 per cent, which has been the case for the last 25 years, added Dr Hanna. Bone marrow transplantation is the process by which a compatible donor, typically a matching sibling, has his or her stem cells transplanted into the thalassemia patients bloodstream via a tube called a central venous catheter. The stem cells travel through the blood into the bone marrow, thus enabling the growth of healthy, oxygen-carrying red blood cells.

The leading US hospital also believes it can work far more effectively with Gulf-based physicians to reduce the standard one-year treatment timeline for transplantation patients, as well as the associated costs and familial inconveniences associated with patient relocation. Some patients may only need to spend as few as three months in the US, it said.

The Dubai Thalassemia Center at the Dubai Health Authority will be one of several healthcare providers in the region to consider the new curative treatment option for its patients.

One such patient, 14-year-old UAE national was seen and treated by Dr Hanna at Cleveland Clinic last year and has benefited from a successful novel reduced intensity Haplo bone marrow transplant in November of 2016.

My life is now completely normal, and I expect to live into old age. I even have high energy levels, enabling me to experience activity for the first time in my life, said the patient.

I no longer require regular blood transfusions, and I can attend school without missing classes and other activities, she said. TradeArabia News Service

Link:
New 'cure' for thalassemia sufferers - Trade Arabia

UPI.com

Vitamin A deficiency is detrimental to blood stem cells Science Daily Therefore, steady replenishment of these cells is indispensable. They arise from so-called "adult" stem cells that divide continuously. In addition, there is a group of very special stem cells in the bone marrow that were first discovered in 2008 by a ... Vitamin A deficiency harms stem cells, study saysUPI.com all 2 news articles »

Read more:
Vitamin A deficiency is detrimental to blood stem cells - Science Daily

Technology Networks

Spheropreservation Method Improves Stem Cell Storage Technology Networks Stem cells are found in various locations of the body such as bone marrow, blood, brain, spinal cord, skin, and corneal limbus. They are responsible for regenerating and repairing damaged tissues and organs in the body. Transplantation of stem cells ... and more »

Read more:
Spheropreservation Method Improves Stem Cell Storage - Technology Networks

The Lehigh football team hosted a bone marrow drive in Lamberton Hallon April 27.

The drive, Be the Match,is a nationwide registry that started at Lehigh in 2009 when Andy Talley, the head football coach at Villanova, reached out to Lehighs head coach Donnie Roberts and asked if he would be interested in contributing to the bone marrow drive.

Roberts said he tries to have more students attend every year and join the cause. Each year, the team strives to get as many students to sign up because the chances of finding a perfect bone marrow match are slim. Since 2009, seven Lehigh students have been perfect matches, four of them football players. Roberts saidthe first student who donated in 2011 ended up having a relationship with the person he donated to.

Yales been (registering) over 400 people every year since theyve been involved, Roberts said. Were not even close to that. But its just the idea that here at Lehigh, if were over 100, I feel good. If we were one, I would feel good, because this is bigger than sports when you have the opportunity to save someones life.

Dan Scassera, 19, left, and Tyler Cavenas, 18, help, from left, Tyler Monaco, 20, Yannick Gbadouwey, 18, and Ben Pingrey, 17, fill out their bone marrow donation forms Thursday, April 27, 2017, in Lamberton Hall. To help donors make a decision, Scassera and Cavenas explain what happens if they are a match. (Roshan Giyanani/B&W Staff)

Participants remain on the registry until their 61st birthday unless they request to be removed from future searches for a match, or they do not meet medical requirements to be eligible. While it is a long-term commitment, Be the Match does its bestto cover all medical and travel costs of donating.

Assistant coach Tyler Ward, 14,said applicants fill out a form, their cheeks are swabbed to retrieve DNA and the samples are sent out to be analyzed.

I think one out of 432 people end up matching with someone, which is why we need so many more people to sign up, Wardsaid.

If applicants are matched with someone, they receive a phone call andgo to a nearby doctor to learn how they can donate. There are two different ways to donate,either through giving blood or bone marrow.

Giving bone marrow is similar to giving blood. Eighty percent ofpeople who donate at all donate blood while 20 percent donate bone marrow. Blood donations are processed through a machine that removes stem cellsand returns blood to the system. Bone marrow donations involve a surgery under anesthesia where marrow is removed from the pelvic bone.Ward saidboth procedures are minor.

At the last station, students are given the option to discreetly remove their consent. This allowed students to decide whether or not they wanted to continue with the donor process. (Roshan Giyanani/B&W Staff)

You dont feel it, youre under anesthesia, and both of them are pretty quick, pretty seamless processes, Ward said.

Roberts said after having the marrow removed, he foundthe pain comparable to lightly bruising a hip. He said whilethe thought of going under anesthesia and having a procedure done is intimidating, it could save someones life.

Julia Wise, 20, helped students with forms at the drive.

I think this is an awesome event, especially on a college campus, because you can recruit so many more people, Wise said. The youngest generation is whats really going to help this cause.

Read more:
Football team hosts 'Be the Match' bone marrow drive - The Brown and White

Californias stem cell agency is on the hunt for a new president and CEO after the surprise announcement this week that C. Randal Mills will be departing the California Institute for Regenerative Medicine. He will leave at the end of June.

Mills, who has headed the agency for three years, will become the next president and CEO of the National Marrow Donor Program. CIRM is replacing him on an interim basis with Maria Millan, M.D., the agencys vice president of therapeutics.

The state agency will soon begin a search for a permanent replacement, said Jonathan Thomas, CIRMs chairman. Millan is a candidate to fill that position, with Mills strong endorsement.

Mills is noted for reorganizing CIRM to provide greater systemic support for translating basic research into clinical science, and to provide quicker and more helpful responses to researchers seeking funding.

His initiative, called CIRM 2.0, was a response to criticism that the agency, funded with $3 billion in California bond money in 2004, has been too slow in getting treatments to patients. ($6 billion will be repaid, including $3 billion in interest)

Agency-supported treatments are now being tested in medical centers throughout the state, including San Diego County. Most prominently, CIRM has established an alpha stem cell clinic at UC San Diego. It is the cell therapy arm of UCSDs Sanford Stem Cell Clinical Center.

Mills said he decided to leave because the National Marrow Donor Program, which he was familiar with, resonated with his own goals of making personal connections with patients.

Before joining CIRM in 2014, Mills was president and CEO of Osiris Therapeutics, developer of a pediatric stem cell drug called Prochymal, used to treat a complication of bone marrow transplants called graft vs. host disease.

If you look at my office, the walls are covered with pictures of the children that we treated who went through bone marrow transplantation, Mills said. Getting to know them, and getting to know their families that had a tremendous effect.

The unexpected announcement drew surprise and concern from stem cell researchers and observers. As admirers of CIRM 2.0, they expressed uncertainty about what direction the agency would take. And with the $3 billion beginning to run out, looking for a new source of funding will be a top concern of Mills successor.

Confidence

But Mills said Wednesday the agency will do well.

If me leaving CIRM is a problem, then I didnt do a good job at CIRM, Mills said. Whether its because Im going to be the head of the National Marrow Donor Program or I get hit by a car, the success of this organization, or any organization thats healthy and functional, should never pivot on one person, Mills said. Ive assembled a team at CIRM that I have absolute, absolute confidence in.

Mills said he would be surprised if Millan didnt turn out to be the agency boards overwhelming choice to be his permanent successor. She assisted in developing the agencys strategic plan and helped it run smoothly, he said.

In 2015, Mills named Millan as senior director of medical affairs and stem cell centers, one of three appointments to CIRMs leadership team. Before joining CIRM, she was vice president and acting chief medical officer at StemCells, Inc. Before that, Millan was director of the Pediatric Liver and Kidney Transplant Program at Stanford University School of Medicine.

Millan said the agencys strategic plan is working, and taking the agency where it needs to go. That plan was developed to guide researchers, doctors and companies over the predictable hurdles they encounter in translating basic research into therapies testable in the clinic and that companies would want to commercialize.

Weve already done the challenging piece of identifying the how how to get to the mission, which is to accelerate these stem cell treatments to those with unmet medical needs, Millan said. Team members are all aligned in accomplishing these goals One cant help but be more energized and motivated to execute on the strategic plan.

About 30 stem cell clinical trials are under way that the agency has funded at one stage or another in research and development.

Jonathan Thomas, the CIRM chairman, said Mills has done what he promised when joining CIRM, and the agency is operating markedly better, in productivity, speed and efficiency.

He has made it, through CIRM 2.0 and beyond, a humming machine that is operating on all cylinders, Thomas said. In doing that, hes worked extensively and highly collaboratively with Maria (Millan) and the rest of the team. That has made CIRM an even better operation than it ever was. So we are in extremely good shape right now to go forward.

Goals accomplished

Jeanne Loring, a CIRM-funded stem cell scientist at The Scripps Research Institute, said Mills made the agency friendlier and more predictable for the scientists it funds.

The first and most dramatic thing he did was to end the process of independent grants, Loring said. Under that process, each grant proposal was considered on its own, with no consideration for success under a previous grant for an earlier stage of the research.

It was always very troubling to people, I think, that they could do very well with CIRM money on an early-stage grant, and that would earn them nothing in a further application to continue the work, Loring said.

As part of CIRM 2.0, Mills emphasized that once projects were accepted for funding, CIRM would become a partner with the scientists to help them accelerate research and development, and ultimately commercialization.

Loring leads a team researching the use of stem cells for Parkinsons therapy. The cells are collected from the patients to be treated, making them a genetic match. They are then genetically reprogrammed to resemble embryonic stem cells, and then matured into the brain cells destroyed in Parkinsons.

Lorings team was awarded $2.4 million in 2016 from CIRM to advance its research. A next-stage grant to translate the research to a clinically ready approach would need about $7 million, Loring said. The work is part of Summit for Stem Cell, a nonprofit alliance of scientists, doctors, patients and Parkinsons disease community supporters.

Veteran stem cell watcher David Jensen praised Mills on his blog, California Stem Cell Report.

"Dr. Mills made substantial contributions to the agency during his tenure, improving both efficiency of the grant making process and transparency of CIRM's operations, Jensen quoted stem cell observer John M. Simpson of Consumer Watchdog as saying.

Simpson added that as CIRM draws down the rest of its $3 billion with no new funding in sight, its not surprising that Mills would accept another job.

Paul Knoepfler, a CIRM-funded stem cell scientist and blogger, wrote Tuesday that Mills had a big positive impact on CIRM and helped it go to the next level.

About the only thing I wasnt a fan of in terms of his leadership was my perception of his negativity toward the FDA and toward FDA oversight of stem cells, and how that manifested at CIRM during his time there, Knoepfler wrote. But good people can strongly disagree on policy.

bradley.fikes@sduniontribune.com

(619) 293-1020

UPDATES:

8:50 a.m.: This article was updated with additional details.

This article was originally published at 2:30 p.m., May 3.

See the article here:
Amid uncertain future, California's stem cell agency loses transformative leader - The San Diego Union-Tribune

Xconomy Boston

Magenta Therapeutics said today it has doubled its money with a $50 million Series B round led by GV, formerly Google Ventures. The Cambridge, MA-based startup spun out of Harvard University last year with nearly $50 million in launch money to develop improved bone marrow transplants.

Magenta has also licensed a drug from Novartis that it says could help boost the number of healthy stem cells that are delivered into a patients body, a key procedure in a transplant.

Used to treat people with cancer and other blood-borne diseases, a bone marrow transplant starts with a procedure to kill a patients diseased blood stem cells, which live in the bone marrow. The diseased cells are then replaced with healthy stem cells, usually from a donor. Though growing safer, its still a risky process, especially for elderly or frail patients. Deaths related to the treatment have dropped below 20 percent in recent years, but Magentas founders as well as researchers at Stanford University are among the groups working to improve the complicated steps.

Magenta is developing three types of drugs, each for a different procedure in the transplant process. It will test them as separate products but try to market them as a suite to transplant clinics, according to management.

The drug Magenta licensed from Novartis is applied to cells from donated umbilical cord blood, which have different properties than cells from blood donated by adults. The drug, which recently completed an early stage study, is meant to stimulate the blood cells to replicate faster outside the body, providing a bigger population to put back into the patient. The more cells, the better the chance that the new healthy cells will engraft, or survive in the patients bone marrow.

Magenta also aims to develop an alternative to chemotherapy or radiation, which a patient receives before a transplant to kill his or her diseased stem cells; and a treatment to coax an adult donors stem cells out of the bone marrow and into the bloodstream, where the cells are easier to harvest for the transplant.

Other investors in the new round are previous backers Third Rock Ventures, Atlas Venture, Partners Innovation Fund, and Access Industries, and new investors including Casdin Capital and BeTheMatch BioTherapies, which is affiliated with the nonprofit international bone marrow registry NMDP/Be The Match.

Magenta said it would work with BeTheMatch BioTherapies on research and development.

Photo Bone Marrow Donation by Andrew Ratto via a Creative Commons 2.0 license.

Alex Lash is Xconomy's National Biotech Editor. He is based in San Francisco.

Follow this link:
Magenta Nabs More Cash, Licenses Drug To Boost Transplant ... - Xconomy

Sacramento Bee

California stem cell agency president steps down as worries mount about its future Sacramento Bee Mills will leave at the end of June to become president of the National Marrow Donor Program in Minneapolis, the world's largest bone marrow donor program. Maria Millan, vice president of therapeutics at the agency, will become its interim president in ... CA Stem Cell Agency Chief Randy Mills to Leave After Three Years ...Xconomy all 4 news articles »

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California stem cell agency president steps down as worries mount about its future - Sacramento Bee

TEL AVIV, Israel, May 3, 2017 /PRNewswire/ --

- Initiation of Phase 3 registrational study expected in second half of this year

- Study to focus on stem cell mobilization for autologous transplantation in multiple myeloma patients

BioLineRx Ltd. (NASDAQ/TASE: BLRX, BLRX.TA), a clinical-stage biopharmaceutical company focused on oncology and immunology, today announced that it has met with the U.S. Food and Drug Administration (FDA) and has gained clarity on the development program and the design of a Phase 3 pivotal study for BL-8040, its robust platform for multiple oncology indications, as a novel stem cell mobilization treatment for autologous bone-marrow transplantation. Following its successful meeting with the FDA, the Company anticipates the initiation of a registrational Phase 3 trial during the second half of 2017. The study will investigate BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF) for mobilization of stem cells from the bone marrow to the peripheral blood, followed by collection and subsequent autologous transplantation in patients with multiple myeloma.

"BL-8040 given as a single injection in a Phase 1/2 study in multiple myeloma patients was previously shown to be highly effective in mobilizing stem cells in combination with G-CSF," said Philip Serlin, Chief Executive Officer of BioLineRx. "Following our recent successful meeting with the FDA, we believe we have a clear development path forward towards registration of BL-8040 as a novel stem cell mobilization treatment for autologous transplantation. We look forward to the initiation of the Phase 3 pivotal study later this year, which, if successful, could pave the way for future commercialization of BL-8040."

"We see clear potential for BL-8040 to benefit multiple myeloma patients undergoing autologous bone marrow transplantation. In parallel, we are continuing to expand the potential of our unique BL-8040 oncology platform, with multiple clinical studies for additional indications that are up and running or expected to commence during 2017. These include several combination studies with immune checkpoint inhibitors, a Phase 2b study in consolidation AML and a Phase 2 study in allogeneic stem-cell mobilization as a monotherapy with topline results expected by the end of 2017," added Mr. Serlin.

About BL-8040

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and stem cell mobilization. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing apoptosis. In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, and T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Stem Cell Mobilization

High-dose chemotherapy followed by stem cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), harvested from the peripheral blood by apheresis, and infused to the patient after chemotherapy. This type of treatment often replaces the use of traditional bone marrow transplantation, because the stem cells are easier to collect and the treatment allows for a quicker recovery time and fewer complications.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on oncology and immunology. The Company in-licenses novel compounds, develops them through pre-clinical and/or clinical stages, and then partners with pharmaceutical companies for advanced clinical development and/or commercialization.

BioLineRx's leading therapeutic candidates are: BL-8040, a cancer therapy platform, which has successfully completed a Phase 2a study for relapsed/refractory acute myeloid leukemia (AML) and is in the midst of a Phase 2b study as an AML consolidation treatment and a Phase 2 study in stem cell mobilization for allogeneic transplantation; and AGI-134, an immunotherapy treatment in development for multiple solid tumors, which is expected to initiate a first-in-man study in the first half of 2018. In addition, BioLineRx has a strategic collaboration with Novartis Pharma AG for the co-development of selected Israeli-sourced novel drug candidates; a collaboration agreement with MSD (known as Merck in the US and Canada), on the basis of which the Company has initiated a Phase 2a study in pancreatic cancer using the combination of BL-8040 and Merck's KEYTRUDA; and a collaboration agreement with Genentech Inc., a member of the Roche Group, to investigate the combination of BL-8040 and Genentech's Atezolizumab in several Phase 1b studies for multiple solid tumor indications and AML.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook, Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 23, 2017. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contacts: PCG Advisory Vivian Cervantes Investor Relations +1-212-554-5482 [emailprotected]

or

Tsipi Haitovsky Public Relations +972-52-989892 [emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx to Initiate Phase ' Study with BL-84 as Novel Stem Cell Mobilization Treatment Following Successful ... - GuruFocus.com

A 26-YEAR-OLD localwoman suffering acute leukemia received a novel stem cell transplant at Shanghai General Hospital today.

Yang Yingjie received the transplant of her fathers steam cell, which is only a half match of hers, and another unit of matched umbilical cord blood to reduce rejection.

The patient is also the 2500thpatient who received umbilical cord blood transfusion from Shanghai Cord Blood Bank, the only licensed umbilical cord blood bank in the city.

Yang was found to have acute leukemia in December last year. She received treatment at Shanghai General Hospital to stabilizeher condition for transplant.

However, she couldnt find a match at stem cell donors bank and had to use her fathersa half match.

We used a unified stem cell transplant therapy in this case. It is also a new breakthrough of leukemia treatmentin recent years,said Dr Wang Chun from Shanghai General Hospital. Each unit of umbilical cord blood is only 50 milliliters, which is not enough for an adult patient. However a unified transplantwith half matched stem cells from adult donor and a unit of fully matched umbilical cord blood can reduce rejection and streamline the transplant success of half-matched stem cells.

Stem cells from umbilical cord blood are less mature than those in adult bone marrow, so they are less prone to rejection by the recipient and more active in developing into different types of cells, experts explained.

Wang said his team has used this therapy to treat somecomplicated leukemia patients and received good results.

There are about 5 million leukemia patients in China and half of them are children. Stem cells have been used in the treatment of many diseases including leukemia, lymphoma and anemia. The most common disease category is leukemia.

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Patient receives novel stem cell transplant - Shanghai Daily (subscription)

Jonathan Pitre received some good news Thursday when blood tests revealed the first sign of white blood cell growth. Tina Boileau / -

After three arduous and eventful weeks in a Minnesota hospital, Jonathan Pitre recorded his first white blood cells Tuesday in the first promising sign since his stem cell transplant.

Pitre, 16, has suffered a series of medical challenges infections, fevers, pain and kidney issues since the April 13 transplant that saw him infused with stem-cell rich blood and marrow drawn from his mother.

But on Tuesday, he finally received some good news when his blood test revealed the first sign of white blood cell growth. The test, which measures white blood cells in a unit of blood, registered 0.1.The normal range is 4.0 to 11.0

We still have to wait for skin biopsies to find out whose cells they are, but its definitely a step in the right direction, said his mother, Tina Boileau.

Last year, after his first stem cell transplant, Pitre and his mother were devastated when biopsies revealed that the white blood cells his body had started to produce were his own. It meant that the transplant had not worked, and that Pitres own stem cells had recolonized his bone marrow.

The family is taking a cautious approach this time, but Tuesdays news was much welcomed after 19 difficult days.

Today is a good day, Boileau tweeted.

A rise in Pitres white blood cell count is the first potential sign that his mothers donated stem cells have started to take root and grow in his bone marrow. But only a biopsy will be able to confirm the cells are being made by Boileaus stem cells, and not his own.

Meanwhile, Pitre will head to surgery again Wednesday to have two central intravenous lines placed in his chest. A third line, a peripherally inserted central catheter (known as a PICC line), will also be installed in case Pitre has to go on dialysis at some point to give his overworked kidneys a break.

During the past three weeks, Pitre has had a central line and a PICC line removed because of infection concerns. It has forced him to use three IVs to support all of his medications.

Boileau said it has been a trying few weeks with one medical problem being fixed only in time for another to appear. The three IVs have also complicated Pitres once-every-second-night bath routine, which is necessary to care for his damaged skin.

Jonathan and I have become very creative with all of his new lines, said Boileau, who is the only one Pitre trusts to remove and re-apply his complex welter of bandages. Its like the most complex puzzle.

Since its not a bath night, they plan to watch the Ottawa Senators play the New York Rangers on Tuesday evening.

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Some good news for Jonathan Pitre: the first white blood cells have appeared - Ottawa Citizen

Stem Cells 101, the Value Proposition

The Key purpose of stem cells is to maintain, heal and regenerate tissues wherever they reside in our body. This is a continuous process that occurs inside the human body throughout its life.

If we did not have stem cells, our lifespan would be about 1 hour, because there would be nothing to replace exhausted cells or damaged tissue. In addition, any time the body is exposed to any sort of toxin, the inflammatory process causes stem cells to swarm the area to repair the damage.

As an example: Say you went to the gym in the morning and did some squats. As a result of that, you would get tiny tears inside the muscle. The stem cells that reside beneath the muscle would come out and repair those little tears.

The reason that, if you continuously go to the gym, you would start to build new muscle, is because those stem cells, hard at work underneath your muscle, are helping to repair and build that new muscle. This would apply to all of the tissues inside your body.

Sure, it is easy to think of stem cell therapy as a magic bullet,but is wise to implement strategies that nourish and thereby help optimize the stem cells we already have in our body.

As noted by Kristin Comella, named # 1 on the Academy of Regenerative Practices list of Top 10 stem cell innovators, has been a stem cell researcher for nearly 20 years: You have to create an appropriate environment for these cells to function in. If you are putting garbage into your body and you are constantly burdening your body with toxins, your stem cells are getting too distracted trying to fight off those toxins.

By creating an appropriate environment, optimizing your diet and reducing exposure to toxins, that will allow the stem cells that were putting in to really home in and focus on the true issue that were trying to treat.

The other thing weve discovered over the years is that [stem cell therapy] is not the type of thing where you take one dose and youre cured forever. Our tissues are constantly getting damaged Youre going to have to repeat-dose and use those stem cells to your advantage.

When you think about a lizard that loses its tail, it takes two years to grow back the tail. Why would we put unrealistic expectations on the stem cells that were trying to apply to repair or replace damaged tissue? This is a very slow process. This is something that will occur over months and may require repeat dosing.

In the past, stem cells were isolated from bone marrow, and were used for bone marrow transplants for cancer patients since the 1930s. But, stem cells come from just about any tissue in the human body, as every tissue contains stem cells.

Human bone marrow has very low amounts of mesenchymal stem cells now believed to be the most important, from a therapeutic perspective.

Mesenchymal stem cells help trigger an immunomodulatory response or a paracrine effect, which means they send signals out to the rest of your body, calling cells to the area to help promote healing.

What researchers have discovered recently is that a more plentiful source of stem cells is actually your fat tissue. Body fat can contain up to 500X more cells than bone marrow, as far as these mesenchymal type stem cells go.

One thing that is also critically important when youre talking about isolating the cells is the number of other cells that are going to be part of that population.

When youre isolating a bone marrow sample, this actually is very high in white blood cells, which are pro-inflammatory.

White blood cells are part of your immune response. When an injury occurs, or a foreign body enters your system, white blood cells will attack. Unfortunately, white blood cells do not discriminate, and can create quite a bit of damage as they clean the area out, Ms. Comella says.

Stem cells, in particular the mesenchymal cells, quiet down the white blood cells and then start the regeneration phase, which leads to new tissue.

Bone marrow tends to be very high in white blood cells and low in the mesenchymal cells. Isolating stem cells from fat tissue is preferred not only because its easier on the patient, but fat also contains a higher population of mesenchymal cells and fewer white blood cells.

The benefit also of isolating [stem cells from] fat is that its a relatively simple procedure. Theres typically no shortage of fat tissue, especially in Americans.

Also, as you age, your bone marrow declines with regards to the number of cells in it, whereas the fat tissue maintains a pretty high number of stem cells, even in older individuals.

We can successfully harvest fat off of just about anyone, regardless of their age or how thin they are. The procedure is done under local [anesthesia], meaning that the patient stays awake. They dont have to go under general anesthesia. We can harvest as few as 15 cubic centimeters of fat, which is a very small amount of fat, and still get a very high number of stem cells, Ms. Comella says.

A stem cell procedure can cost anywhere from $5,000 to $15,000, depending on what is being done, and rarely if ever will insurance cover it.

Still, when compared it to the cost of long-term medications or the out-of-pocket cost of getting a knee replacement, stem cell therapy may still be a less expensive alternative.

Also, a single extraction will typically yield enough stem cells for 20 to 25 future treatments, should one decide to store stem cells for future needs.

I think it is accessible for patients, Ms. Comella says. Its an out-patient procedure. One should plan to be in clinic for about 2 hours; no real limitations afterwards, just no submerging in water, no alcohol, no smoking for a week. But other than that, patients can resume their normal activities and go about their regular daily lives.

Interestingly, Ms. Comella notes that patients who eat a very healthy diet, focusing on Organic and grass fed meat, have body fat that is very hearty and almost sticky, yielding high amounts of very healthy stem cells.

We can grow much better and faster stem cells from that fat than [the fat from] somebody who eats a grain-based diet or is exposed to a lot of toxins in their diet, she says. Their fat tends to be very fluffy, buttery yellow. The cells that come out of that are not necessarily as good a quality. Its just been very interesting. And of note, patients that are cigarette smokers, their fat is actually gray-tinged in color. The stem cells do not grow well at all.

The beauty of stem cell therapy is that it mimics a process that is ongoing in the human body all the time. Our stem cells are continuously promoting healing, and they do not have to be manipulated in any way. The stem cells naturally know how to hone in on areas of inflammation and how to repair damaged tissue.

All we are doing is harnessing the cells from one location where theyre sitting dormant and relocating them to exactly where we want them and we need them to work, Ms.Comella says. Basically, anything inside your body that is inflamed, that is damaged in some way, that is lacking blood supply, the [stem] cells can successfully treat.

That means orthopedics, knee injections, shoulder injections, osteoarthritis, acute injuries, anterior cruciate ligament tears in your back back pain associated with degenerative disc disease or damaged tendons or ligaments, herniated and bulging discs. You can also use it in systemic issues, everything from diabetes, to cardiac, to lungs any tissue organ inside your body thats been damaged.

Autoimmune diseases [can also be treated]. The stem cells are naturally immunosuppressant, meaning they can help quiet down an over reactive immune system and help the immune system function in a more normal way. Neurological diseases, traumatic brain injury, amyotrophic lateral sclerosis, Parkinsons. All of these have to do with tissue thats not functioning properly. The cells can be used to address that.

The list of different diseases that could benefit from this intervention is very impressive.

And one can dramatically improve the benefits of stem cell intervention by combining it with other healthy lifestyle factors that optimize mitochondrial function, such as eating a healthy Real Food diet, exercising, sleeping well, avoiding toxins and detoxifying from toxic influences.

Stem cells can be used as part of an anti-aging program. Ms. Comella has used stem cells on herself for several years, and report feeling better now than she did a decade ago.

The ability to reduce inflammation inside your body is basically making yourself live longer. Inflammation is what kills us all. Its what makes our telomeres shrink. Its what causes us pain and discomfort. Its what makes the tissues start to die. The ability to dose yourself with stem cells and bring down your inflammation, which is most likely caused by any sort of toxin that youve been exposed to breathing air is exposure to toxins this is going to lengthen your lifespan.

I typically will do a dose every 6 to 12 months, regardless of whats going on. If I have anything thats bothering me, if I tweak my knee at the gym, then I absolutely will come in and do an injection in my knee. I want to keep my tissue healthy for as long as possible.

I want to stay strong. I dont want to wait until something is wrong with me. I think that this is the future of medicine. This is what were going to start to see. People will begin to get their regular doses of [their own] stem cells and itll just be common practice.

Keep in mind there is a gradual and progressive decline in the quality and the number of stem cells as we age, so when considering this approach, it would be prudent and advantageous to extract and bank stem cells as early on as possible. There are stem cell banking services available.

Your stem cells are never as young as they are right now. Every minute that you live, your telomeres are shrinking. The ability to lock in the youth of your cells today can be very beneficial for you going forward, and for your health going forward. God forbid something happens. What if you have a heart attack? Youre not going to get clearance to get a mini-lipo aspirate procedure.

If you have your cells waiting in the bank, ready for you, it becomes very easy to pull a dose and do an IV delivery of cells. Its almost criminal that were not doing this for every single one of our cardiac patients. This should be standard practice. We should be having every single patient bank their stem cells at a young age and have them waiting, ready and available. The technology is there. We have it. Im not sure why this technology is not being made available to everyone,says.

I think stem cell therapy is very different than traditional medicine. Stem cell therapy may actually make it so that you dont have to be dependent on pharmaceutical medications. You can actually repair the tissue and thats it. This is a very different way of viewing medicine,Ms. Comella says.

The amniotic products available in the US are not so much stem cell products as they are growth factor products.

According to Ms. Comella, they can be useful in creating an immunomodulatory response, which can help to promote healing, but that differs from the living stem cell procedures that can be done by either isolating cells from body fat or bone marrow. As a general rule, clinical benefits are not achieved when using an amniotic product, primarily because they do not contain living stem cells.

I want to contrast that to what are called embryonic stem cells, Ms.Comella adds. The products obtained from cord blood, from women who are having babies, are not embryonic stem cells. Embryonic stem cells are when you are first bringing the egg and sperm together. Three days after that, you can isolate what is called an inner cell mass. This inner cell mass can be used to then grow cells in culture, or that inner cell mass could eventually lead to the formation of a baby.

Those are embryonic stem cells, and those are pluripotential, meaning that they have the ability to form an entire being, versus adult stem cells or stem cells that are present in amniotic tissue, [which] are multipotential, which only have the ability to form subsets of tissue.

When dealing with different diseases or damaged tissue or inflammation, mostly you want to repair tissue. If somebody has damage in their knee, they do nnot necessarily need embryonic cells because they do not need a baby in their knee. They need new cartilage in their knee.

Stem cell therapy is very different than traditional medicine. Stem cell therapy may actually make it so that we do have to depend on pharmaceutical medications. And we can actually repair the tissue and be done with it. This is a very different way of viewing medicine.

Eat healthy, Be healthy, Live lively

cells, damage, help, medication, medicine, muscle, patients, procedures, repair, stem, therapy, tissues, toxins

Paul A. Ebeling, polymath, excels in diverse fields of knowledge. Pattern Recognition Analyst in Equities, Commodities and Foreign Exchange and author of The Red Roadmasters Technical Report on the US Major Market Indices, a highly regarded, weekly financial market letter, he is also a philosopher, issuing insights on a wide range of subjects to a following of over 250,000 cohorts. An international audience of opinion makers, business leaders, and global organizations recognizes Ebeling as an expert.

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Stem Cells 101, the Value Proposition - Live Trading News

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics, a biotechnology company developing therapies to improve and expand the use of curative stem cell transplantation for more patients, today announced rapid progress in advancing the companys strategic vision, including the completion of a $50 million Series B financing; in-licensing a clinical-stage program from Novartis to support the use of stem cell transplantation in a variety of disease settings; and a strategic partnership with Be The Match BioTherapiesSM, an organization offering solutions for delivering autologous and allogeneic cellular therapies.

The financing announced today is intended to fuel development of innovative product candidates across multiple aspects of transplantation medicine, including more precise preparation of patients, stem cell harvesting and stem cell expansion. The Series B round, which was oversubscribed, was led by GV (formerly Google Ventures), with participation from all existing investors, including Atlas Venture, Third Rock Ventures, Partners Innovation Fund and Access Industries. The financing also included Casdin Capital and other crossover investors, as well as Be The Match BioTherapies, a subsidiary of National Marrow Donor Program(NMDP)/Be The Match, the worlds leading organization focused on saving lives through bone marrow and umbilical cord blood transplantation.

Magenta has quickly established itself as a nexus of innovation in stem cell science, catalyzing interest in this area of medicine with the recognition that improvements will have profound impact on patients, said Jason Gardner, D. Phil., chief executive officer, president and cofounder of Magenta Therapeutics. We aspire to accelerate products that could unleash the potential of transplantation to more patients, including those with autoimmune diseases, genetic blood disorders and cancer. The resounding interest in Magenta from such a high-quality set of investors is a testament to our solid progress since launch, including building a world-class team and a robust pipeline, and generating promising early data.

MGTA-456: Investigational Product Addressing Significant Unmet Need in Stem Cell Transplant

The clinical-stage program in-licensed by Magenta from Novartis, MGTA-456 (formerly HSC835), aims to expand the number of cord blood stem cells used in transplants to achieve superior clinical outcomes compared to standard transplant procedures, and to enable more patients to benefit from a transplant. Under this agreement, Magenta gains rights to use MGTA-456 in selected applications and will develop MGTA-456 in multiple diseases, including immune and blood diseases.

Early results published in Science1 demonstrated the ability of MGTA-456 to significantly increase the number of umbilical cord blood stem cells. Clinical results reported in Cell Stem Cell2 demonstrated that this approach yielded an increased expansion of stem cells.

John E. Wagner, M.D., executive medical director of the Bone Marrow Transplantation Program at the University of Minnesota and the studys lead author, stated: MGTA-456 markedly shortens time to recovery, addressing one of the most significant challenges in stem cell transplantation today. MGTA-456 achieved a remarkable increase in the number of blood-forming stem cells, greater than that observed by all other methods that have been tested to date. This product has the potential to further improve cord blood transplant outcomes.

Be The Match BioTherapies Strategic Partnership Agreement

Magenta and Be The Match BioTherapies also announced today that in addition to the equity investment, the two organizations have initiated a collaboration to support their shared goals of improving transplant medicine. Magenta and Be The Match BioTherapies will explore opportunities to work together across all of Magentas research efforts, from discovery through clinical development. Under this agreement, Magenta may leverage Be The Match BioTherapies capabilities, including its cell therapy delivery platform, industry relationships, clinical trial design and management, and patient outcomes data derived from the NMDP/Be The Match, which operates the largest and most diverse marrow registry in the world. NMDP/Be The Match has a network of more than 486 organizations that support marrow transplant worldwide, including 178 transplant centers in the United States and more than 45 international donor centers and cooperative registries.

We are proud to have made our first equity investment as an organization in Magenta Therapeutics, and we share a vision to improve and advance the use of curative stem cell transplantation for patients with a wide range of diseases, said Amy Ronneberg, president of Be The Match Biotherapies.

About Magenta Therapeutics

Magenta Therapeutics is a biotechnology company harnessing the power of stem cell science to revolutionize stem cell transplantation for patients with immune- and blood-based diseases. By creating a platform focused on critical areas of transplant medicine, Magenta Therapeutics is pioneering an integrated, but modular approach to stem cell therapies to create patient benefits. Founded by internationally recognized leaders in stem cell transplant medicine, Magenta Therapeutics was launched in 2016 by Third Rock Ventures and Atlas Venture and is headquartered in Cambridge, Mass. For more information, please visitwww.magentatx.com.

About Third Rock Ventures

Third Rock Ventures is a leading healthcare venture firm focused on investing and launching companies that make a difference in peoples lives. The Third Rock team has a unique vision for ideating and building transformative healthcare companies. Working closely with our strategic partners and entrepreneurs, Third Rock has an extensive track record for managing the value creation path to deliver exceptional performance. For more information, please visit the firms website atwww.thirdrockventures.com.

About Atlas Venture

Atlas Venture is a leading biotech venture capital firm. With the goal of doing well by doing good, we have been building breakthrough biotech startups since 1993. We work side by side with exceptional scientists and entrepreneurs to translate high impact science into medicines for patients. Our seed-led venture creation strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. For more information, please visitwww.atlasventure.com.

About GV

GV provides venture capital funding to bold new companies. In the fields of life science, healthcare, artificial intelligence, robotics, transportation, cyber security, and agriculture, GV's companies aim to improve lives and change industries. GV's team of world-class engineers, designers, physicians, scientists, marketers, and investors work together to provide these startups exceptional support on the road to success.

Launched as Google Ventures in 2009, GV is the venture capital arm of Alphabet, Inc. GV helps startups interface with Google, providing unique access to the worlds best technology and talent. GV has $2.4 billion under management and is headquartered in Mountain View, California, with offices in San Francisco, Boston, New York, and London. Launched as Google Ventures in 2009, GV is the venture capital arm of Alphabet, Inc. For more information, please visit http://www.gv.com.

About Be The Match BioTherapies

Be The Match BioTherapies partners with organizations pursuing new life-saving treatments in cellular therapy. Built on the foundation established over the last 30 years by theNMDP/Be The Match, the organization has unparalleled experience in personalized patient management with a single point of contact, cell sourcing and collection, cell therapy delivery platform, immunogenetics and bioinformatics, research and regulatory compliance. By leveraging proven capabilities and established relationships, Be The Match BioTherapies can bring customizable solutions to organizations in every stage of cellular therapy developmentfrom discovery through commercialization. Discover how Be The Match BioTherapies can assist your company atBeTheMatchBioTherapies.com.

For more information on todays announcement, see Jason Gardners post in the Life Sci VC blog: https://lifescivc.com/2017/05/building-a-bioteth-a-triple-play/.

1Science.2010 Sep 10;329(5997):1345-8. 2Cell Stem Cell.2016 Jan 7;18(1):144-55.

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Magenta Therapeutics Advances Stem Cell Transplantation Strategy with $50 Million Series B Financing, Licensing of ... - Business Wire (press release)

Anna-Lena Ahlstrm, Royal Court, Sweden

Princess Christina of Sweden, the youngest of King Carl XVI Gustafs four older sisters, has successfully undergone a stem cell transplant.

Swedish newspaper Expressen first reported the news with a confirmation from the Swedish Royal Courts Director of Information and Press Department,Margareta Thorgren. She explained to them, The stem cell operation is completed. Princess Christina is well under the circumstances.

The Princess will remain at home during her recuperation. After such operations, the immune system is considerably weakened, and as a result, doctors commonly advise patients stay isolated while they heal.

It was just last month that the Court made the announcement of the pending transplant, which can be stressful on the body,saying, Princess Christina, Mrs Magnuson has, since October, been treated for blood cancer with regular chemotherapy. The treatment has gone well. But the Princesss blood cancer cannot be cured with this treatment because it occurred in bone marrow stem cells that are resistant to chemotherapy.

In consultation with the family and doctors, the Princess has decided to undergo a stem cell transplant.

She was diagnosed with chronicleukaemia in October of last year. At the time, the Swedish Royal Court said that she was feeling relatively good. It was stated that the73-year-old would scale back her royal duties during her treatmentbut would fulfil her commitments when her health allowed.They also asked that she be able to undergo her chemotherapy in peace.

In 2010, Christina announced that she had undergone treatment for breast cancer including three surgeries and had beaten the disease. After defeating breast cancer, Christina devoted much of her time to bringing attention to cancer issues.

The Princess was born on 3 August 1943 at Haga Palace in Solna, Sweden. She married Tord Magnuson in 1974 at the Royal Chapel in Stockholm Palace. They have three sons: Gustaf, Oscar, and Victor.

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Princess Christina of Sweden undergoes successful stem cell transplant - Royal Central

MONROE, LA - A bone marrow drive for 21-year-old James Christopher Allums and his 3-year-old sister Elizabeth is taking place today, May 1st, at locations throughout Northeast Louisiana.

University of Louisiana Monroe Medical Laboratory Science faculty and students are helping organize the drive. The drive on campus is 9 a.m. to 5 p.m. in the Student Union Building and Quad.

May 1st is National Fanconi Anemia Day. James Christopher and Elizabeth suffer from this disease, which is fatal without a bone marrow or stem cell transplant. They are the children of Chris and Ellen Allums.

Melanie Chapman, assistant professor to the School of Health Professions, said, "This is a wonderful opportunity for ULM Warhawks to fly high by working together and setting aside our busy agendas to give two great kids, and possibly others, the chance to live out their years. I am privileged to be a part of ULM and this community effort."

Bone marrow drive locations:

Times vary and new locations may be added. For information, visit caringbridge.org and search James Christopher Allums .

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Bone Marrow Drive For Allums Siblings At ULM - MyArkLaMiss (press release) (blog)

HACKENSACK, N.J. and PETACH TIKVAH, Israel, May 1, 2017 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced today that the Company will present data from its Phase 2 clinical study of NurOwn in amyotrophic lateral sclerosis (ALS) at the International Society for Cellular Therapy (ISCT) annual conference in London, England, and the World Advanced Therapy and Regenerative Medicine Congress in London, England.

International Society for Cellular Therapy Annual Conference

Date:

Thursday, May 4

Time:

3:30pm - 5:00pm CET

Location:

ExCeL London, London, England

Title:

Safety and Efficacy of Transplantation of NurOwn (Autologous Mesenchymal Stromal Cells Secreting Neurotrophic Factors) in Patients with ALS: a Phase 2 Randomized Double Blind Placebo Controlled Trial

Presenter:

Dr. Yael Gothelf, Chief Regulatory and Scientific Officer, BrainStorm Cell Therapeutics

Date:

Thursday, May 4

Time:

5:00pm - 6.30pm CET

Location:

ExCeL London, London, England

Title:

Poster presentation titled: In vivo modulation of neurotrophic and inflammatory factors in the CSF of ALS patients treated with NurOwn (MSC NTF cells)

Presenter:

Dr. Yael Gothelf, Chief Regulatory and Scientific Officer, BrainStorm Cell Therapeutics

World Advanced Therapies & Regenerative Medicine Congress 2017

Date:

Thursday, May 18

Time:

2:40pm CET

Location:

Business Design Centre, London

Title:

Brainstorm's NurOwn Treatment for Neurodegenerative Diseases

Presenter:

Chaim Lebovits, Chief Executive Officer, BrainStorm Cell Therapeutics

About the International Society for Cellular TherapyEstablished in 1992, the International Society for Cellular Therapy (ISCT) is a global society of clinicians, researchers, regulators, technologists and industry partners with a shared vision to translate cellular therapy into safe and effective therapies to improve patients' lives worldwide. ISCT is the global leader focused on pre-clinical and translational aspects of developing cell-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care. Comprised of over 1300 cell therapy experts across five geographic regions and representation from over 50 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit: http://www.celltherapysociety.org.

About the World Advanced Therapies & Regenerative Medicine Congress12 years ago when the World Stem Cells Congress was launched, the stem cells sector was one of scientific interest. Focusing on the challenges of how to transform these little precursor cells eventually into new tissues and organs. The first stem cells conference was relatively small with only 80 people attending the first event, but we knew we were on the verge of something huge and exciting. In May 2017 the newly named World Advanced Therapies & Regenerative Medicine Congress, will bring together 800+ attendees and explore the rapidly developing world of ATMPs (Advanced Therapy Medicinal Products). From process development to clinical translation this congress will bring you the most exciting case studies and new data. Experts in every area will help you tackle the process and regulatory hurdles of developing these new therapeutic formats all the way through manufacture and into the clinic.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult mesenchymal stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. NurOwn has been administered to approximately 75 patients with ALS in clinical trials conducted in the United States and Israel. In a randomized, double-blind, placebo-controlled clinical trial conducted in the U. S., a clinically meaningful benefit was demonstrated by higher response to NurOwn compared with placebo. For more information, visit the company's website at http://www.brainstorm-cell.com.

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Safe-Harbor StatementStatements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual 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, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://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

Media:Uri Yablonka Brainstorm Cell Therapeutics Inc. Phone: (646) 666-3188 uri@brainstorm-cell.com

Investors:Michael Rice LifeSci Advisors, LLC Phone: 646-597-6979 mrice@lifesciadvisors.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/brainstorm-to-present-at-two-scientific-conferences-in-may-300448600.html

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BrainStorm to Present at Two Scientific Conferences in May - Yahoo Finance