Last February, Timothy Ray Brown a.k.a. the Berlin patient celebrated his 10th birthday. Well, sort of. His 10th birthday actually refers to the 10th anniversary marking his recognition as the only person in the world to be cured of HIV, the virus that causes AIDS.

Browns incredible story began in 1995 when he was diagnosed with HIV. For over 10 years, he was able to stave off the disease by taking antiretroviral drugs. But disaster decided to strike again. Aside from HIV, it turned out that he had developed cancer as well, specifically, acute myeloid leukemia.

To fight off the cancer, Browns doctors decided to use chemotherapy and radiation to destroy his immune system, then use donated stem cells via a bone marrow transplant to rebuild it. It was supposed to be a standard treatment, but the doctors tweaked it a bit. The stem cell donor they chose was immune to HIV. Scientifically, this means that the donor had a gene mutation that caused him not to have CCR5 in his cells; CCR5 is the protein that allows HIV to get into a persons blood cells.

Brown received two bone marrow transplants, and the results were nothing short of a miracle he was cured of both HIV and cancer!

That extraordinary feat resulted in a common consensus that it was the transplant that saved Brown from his two lethal diseases. Based on new evidence, however, that conclusion might have to be re-evaluated. It might not have been the transplant that cured him. Rather, his immune systems reaction to the transplant that finally did the trick.

The immune reaction is known as graft-versus-host disease. Essentially, what happens is this: the donors immune cells attack the recipients cells. In Browns case, the donors cells attacked his immune cells (including the HIV contained in the cells). The result was the death of the HIV in his system.

At the IrsiCaixa AIDS Research Institute in Spain, six patients with HIV and cancer who received treatment similar with Browns now appear to be cleared of HIV.

Something to think about, though. Among the six patients, only one received the exact same treatment as Browns the bone marrow donor had the CCR5 gene mutation. Yet all six of them developed graft-versus-host disease.

Unless they stop taking their anti-HIV drugs, it cant be confirmed if they have been completely cleared of HIV. So far, though, all HIV tests on the six of them have been negative for 2 years. And that can certainly add support to the idea that its the graft-versus-host disease that kills HIV, not the transplant.

Still, if this does turn out to be accurate, it might not be such an appealing approach to use because its virtually a deliberate attempt to kill a patients immune cells which can easily turn fatal. Especially for patients who have the means to afford the expensive anti-HIV drugs, exposing ones self to further risk via transplantation is not really a logical option.

Although theres some consolation in the fact that at least there are anti-HIV drugs that can keep the disease at bay as long as you continue taking the drugs, its obviously far from being satisfactory given the fact that not everyone can afford such an expensive lifetime treatment. Which is why so much studying still needs to be done to better understand HIVs behavior and how this nasty virus can be eradicated. Lets hope science eventually leads us to a safer and more affordable cure.

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Attacking A Patient's Immune Cells May Wipe Out HIV - Wall Street Pit

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Hope Ford, WFMY 4:05 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.

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

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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 »

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

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May 5, 2017

Lack of vitamin A in the body has a detrimental effect on the hematopoietic system in the bone marrow. The deficiency causes a loss of important blood stem cells, scientists from the German Cancer Research Center (DKFZ) and the Heidelberg Institute of Stem Cell Research and Experimental Medicine (HI-STEM) now report in the latest issue of the journal Cell. These findings will open up new prospects in cancer therapy.

Many specialized cells, such as in the skin, gut or blood, have a lifespan of only a few days. 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 research team led by Andreas Trumpp, who is a division head at the DKFZ and director of HI-STEM. These cells remain in a kind of dormancy most of the time and only become active in an emergency such as bacterial or viral infections, heavy blood loss, or in the wake of chemotherapy. Once their work is done, the body sends its most potent stem cells back to sleep. The scientists assume that this protects them from dangerous mutations that may lead to leukemia.

The mechanisms that activate these special stem cells or make them go back to sleep after their work is done have remained elusive until now. The scientists have now identified retinoic acid, a vitamin A metabolite, as a crucial factor in this process. If this substance is absent, active stem cells are unable to return to a dormant state and mature into specialized blood cells instead. This means that they are lost as a reservoir. This was shown in studies with specially bred mice whose dormant stem cells are green fluorescent. "If we feed these mice on a vitamin A deficient diet for some time, this leads to a loss of the stem cells," said Nina Cabezas-Wallscheid, who is the first author of the publication. "Thus, we can prove for the first time that vitamin A has a direct impact on blood stem cells."

This finding not only enhances our understanding of the development of blood cells, it also sheds new light on prior studies that demonstrate that vitamin A deficiency impairs the immune system. "This shows how vitally important it is to have a sufficient intake of vitamin A from a balanced diet," Cabezas-Wallscheid emphasized. The body cannot produce its own vitamin A.

The scientists also have hopes for new prospects in cancer treatment. There is evidence that cancer cells, like healthy stem cells, also rest in a state of dormancy. When dormant, their metabolism is almost completely shut downand this makes them resistant to chemotherapy. "Once we understand in detail how vitamin A or retinoic acid, respectively, sends normal and malignant stem cells into dormancy, we can try to turn the tables," explained Trumpp. "If we could make cancer cells temporarily enter an active state, we could thus make them vulnerable to modern therapies."

In addition, in collaboration with colleagues from the European Bioinformatics Institute in Cambridge, the team performed genome-wide analyses of single cells and discovered that the transition from dormant to active stem cells and then on to progenitor cells is a continuous one and follows a different path for each individual cell. So far, scientists had assumed that specific cell types develop step by step in a defined pattern. This finding revolutionizes the previous concept of how cell differentiation in the body takes place.

Explore further: Vitamins and aminoacids regulate stem cell biology

More information: Nina Cabezas-Wallscheid et al, Vitamin A-Retinoic Acid Signaling Regulates Hematopoietic Stem Cell Dormancy, Cell (2017). DOI: 10.1016/j.cell.2017.04.018

Journal reference: Cell

Provided by: German Cancer Research Center

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Vitamin A deficiency is detrimental to blood stem cells - Phys.Org

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April Stevens , WZZM 4:52 PM. EDT May 04, 2017

Nick Keizer during the donation process. He donated stem cells on his birthday, May 2, to a man in Denmark. (Photo: Courtesy of GVSU)

ALLENDALE, MICH. - A Grand Valley State University football player celebrated his birthday doing something utterly selfless -- donating stem cells to man in Denmark.

The Laker football tight end, Nick Keizer, and many of his teammates swabbed their cheeks a Michigan Blood registry drive in March 2016. At the time, Keizer said he never thought he would be a bone marrow match for someone.

"The presentation pulled at my heart and I thought, 'Why not sign up to be a donor?' Yet I also thought the odds of me actually being a match can't be that high," he said.

Being a bone marrow match is quite rare -- about a 1 in 500 chance, according to Caitlin Gallagher, community engagement representation for Michigan Blood and Be the Match.

Michigan Blood was notified in December that Keizer and the Denmark man were potential matches. Keizer was required to undergo more blood work and in February, was deemed a perfect match for a 59-year-old man in Denmark who suffered from a bone marrow disease.

Keizer's non-surgical donation took about four hours, and although he's "not a big needle guy" he went through with it all, "because, that doesn't compare to what the patient is going through."

His stem cells were sent by a volunteer courierwho flew to Denmark on May 2, Keizer's birthday.

Keizer is a Portage native, he graduate from Grand Valley on April 28 with a bachelor's degree in accounting and finance. Keizer is eligible to play one more season of football, and will finish his athletic career in the fall while pursuing a master's degree in business administration.

Makeit easy to keep up to date with more stories like this.Download theWZZM13 app now.

April Stevensis a multi-platform producer atWZZM13. Have a news tip? Emailnews@wzzm13.com, visit ourFacebook pageorTwitter.

2017 WZZM-TV

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From Lubbers Stadium to Denmark: GVSU football player donates stem cells - WZZM

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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 successful stem cell transplant - Royal Central

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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.

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

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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.

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

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Amid uncertain future, state's stem cell agency loses transformational leader - The San Diego Union-Tribune

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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 YearsXconomy all 4 news articles »

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

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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.

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Magenta Nabs More Cash, Licenses Drug To Boost Transplant Pipeline - Xconomy

Bone Marrow Stem Cells Comments Off on Magenta Nabs More Cash, Licenses Drug To Boost Transplant Pipeline – Xconomy | May 2nd, 2017

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 ... - Business Wire (press release)

Bone Marrow Stem Cells Comments Off on Magenta Therapeutics Advances Stem Cell Transplantation Strategy … – Business Wire (press release) | May 2nd, 2017

Home Health News Mouse teeth shown to hold insight into future stem cell tissue regeneration

The use of stem cells throughout the years has been both a decisive topic and one that holds a lot of promise for potential medical therapy. They are essentially undifferentiated biological cells that havent yet been specialized for a specific purpose. The cells of your heart, stomach, and even your brain have all started out as stem cells, and it wasnt until some point during human development that biological processes channeled them to permanently becoming one type of cell. Scientists and researchers around the globe are always in search of the best way to learn about and harvest these valuable cells, and the latest reports suggest the teeth of rodents are an abundant source.

There are considered two main stem cell types in the body: one is from embryonic development when in the womb, and the other are adult stem cells that exist throughout the body. Harvesting embryonic stem cells has been controversial, as it often seen as unethical, but adult stem cellsfound in organs such as the bone marrow, blood vessel, and liver in mammalsis easier to obtain. Stomach linings, for example, require the constant shedding of their cell linings as the acid wears away at them, and having adult stems cells allows for quick replacement of these sloughed off cells.

Weve all seen mice before, and one of their defining characteristics are their front teeth. What most people arent aware of is that their front teeth, or incisors, constantly grow, as they rely on them to be consistently sharp for burrowing and self-defense, and of course, for eating away at your pantry food. As we grow older our teeth start to wear out, and in nature, once you dont have your teeth anymore, you die. As a result, mice and many other animals from elephants to some primates can grow their teeth continuously. Our labs objective is to learn the rules that let mouse incisors grow continuously to help us one day grow teeth in the lab, but also to help us identify general principles that could enable us to understand the processes of tissue renewal much more broadly, said UC San Franciscos Ophir Klein, MD, Ph.D., a professor of orofacial sciences in UCSFs School of Dentistry and of pediatrics in the School of Medicine.

While not all aspects of this process are fully understood just yet, as the exact signals triggering this process have yet to be identified. It, however, marks an advancement of knowledge in the field, and one that bodes well for the future of stem cell therapy. It may prove beneficial for tissue regeneration to treat everything from severe burns to growing entire organs from scratch.

Related:Stem cells from fat may be useful to prevent aging

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Stem cell technique may aid in bone repair

Osteoporosis can be reversed by stem cell therapy, new potential treatment

https://www.ucsf.edu/news/2017/04/406836/mouse-teeth-providing-new-insights-tissue-regeneration http://www.sciencedirect.com/science/article/pii/S1934590917300942 http://www.medicalnewstoday.com/info/stem_cell

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Mouse teeth shown to hold insight into future stem cell tissue regeneration - Bel Marra Health

Bone Marrow Stem Cells Comments Off on Mouse teeth shown to hold insight into future stem cell tissue regeneration – Bel Marra Health | May 1st, 2017

A benefit rummage, bake and vendor sale will be held this Friday and Saturday for Trisha Suits, a Lisbon resident battling leukemia. She is shown with her mother, Alice Loy, and 6-year-old son Landon who proudly displays the jacket Trisha wore while serving as an assistant cross country coach at Lisbon. Despite being virtually blind, the David Anderson High School graduate ran cross country in high school. (Salem News photo by J.D.Creer)

WHAT: Rummage, bake and vendor sale to benefit area resident Trisha Suits who will be undergoing leukemia treatments at the Cleveland Clinic.

WHEN: From 9 a.m.-4 p.m. Friday and Saturday, May 5-6.

WHERE: Guilford Lake Ruritan Hall, state Route 172.

She was born weighing just a pound and eight ounces. But Trisha Suits is hardly a lightweight.

The courageous 30-year-old Lisbon resident, left virtually blind due to her premature birth, has taken on all comers throughout her life. Despite having only 2 percent vision in her right eye and none in her left, she has been a capable mom in helping to raise her son. She is a 2006 David Anderson High School graduate. Remarkably, she ran as a member of the cross-country team, memorizing her routes. Just putting one foot in front of the other, Trisha quipped, saying never fell. She later served as a Blue Devils assistant coach.

Now she is confronting her biggest challenge. She has been diagnosed with a complex form of leukemia and will be undergoing bone marrow and stem cell transplants at the Cleveland Clinic. Due to ongoing treatments, she will be required to remain in the hospital for six weeks. Then she will need to stay at a nearby housing complex for another 100 days.

Trisha was diagnosed in early March, after passing out from severe blood loss. She spent a month in the Cleveland Clinic getting chemotherapy. But due to genetic mutations, she needs bone marrow and stem cell transplants to combat acute myeloid leukemia a type of cancer that starts in the blood-forming cells of marrow.

According to her aunt, Melody Hobbs of Salem, her lengthy stay at the housing complex the Transplant House of Cleveland will cost about $75 per day for just the lodging. A donor has been found. Transplant treatments are expected to begin May 11.

To help offset the costs, a combination rummage, bake and vendor sale for Trisha will be held this Friday and Saturday, May 5-6, from 9 a.m. to 4 p.m. each day at the Guilford Ruritans Hall off of state Route 172.

We just need people to come, Hobbs said. We are trying to raise awareness to get more people out there. All the money raised will pay for Trishas lodging and transportation.

Trishas ordeal is and will continue to be grueling. Admittedly, she gets bitter, angry and frustrated. The uncertainty is overwhelming.

The really hard part of it will be being in Cleveland away from her son and family, said Hobbs.

Indeed, Trishas said her hobby is being the best mom she can be for her son. Six-year-old Landon is a McKinley Elementary School student.

I love my mommy, he offered. Its not just my moms fight, its our fight.

Trisha and her son lives with her parents, Rick Joy and Alice Loy. Her sister, Summer Burkholder, is a co-organizer of this weekends benefit.

The transplants offer a possible cure. Without them, it would be dire.

God only gives me what I can handle, said Trisha who has spent her entire life combating challenges. But I am scared about what this is going to do to my body.

Ongoing updates on Trisha may be found on Facebook. Visit: Trishas Fight with AML. To make an online donation, a link: youcaring.com may be accessed.

jdcreer@salemnews.net

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Benefit planned May 5-6 for area leukemia victim - SalemNews.net

Bone Marrow Stem Cells Comments Off on Benefit planned May 5-6 for area leukemia victim – SalemNews.net | May 1st, 2017

Few things could start out so simple and yet maybe lead you to save a life. But one woman's firsthand experience led precisely down that road, and now she's getting more people on board.

Be the Match is a program offering free kits for people to submit their DNA through mouth swabs to see if they might one day be a match to donate to someone with blood cancers or other blood-based diseases.

And getting that opportunity is about to become even easier for those in the Roaring Fork Valley this coming First Friday (May 5) and then on Dandelion Day (May 13) in Carbondale.

Erica Borum, who works as a civil engineer for the White River National Forest, is setting up a Be the Match booth at these events, where she'll have registry kits with mouth swabs ready to be used and sent off.

This first step is simple. If you're between 18 and 44 you can participate, but this first step doesn't mean that you're automatically going to be donating to someone.

Your mouth swab puts you on a DNA registry. Once your DNA is on the list, that information is available to doctors looking for donors who match with patients in need for stem cells or bone marrow.

According to Be the Match, each year about 14,000 patients are down to one option for a cure: a transplant from someone outside their family.

Borum is pushing this effort in Carbondale after her own experience a little more than a year ago donating to a man with Hodgkin's lymphoma. She wants to give people this opportunity by bringing the kits to them, but she's also on a mission to demystify the process.

"When people are first addressed with it, it's strange and weird and not something that would be of interest," she said. "I'd like to help make it not so foreign."

Borum first heard about this donation process through a friend, who knew of someone needing a bone marrow transplant for leukemia. The patient was down to her last option, but in the end the procedure worked out for her.

Borum got online and started doing her research on the organization Be the Match.

The chances that you'll be selected are actually quite slim. This isn't as simple as finding a person with matching blood type. Because doctors are looking for someone with highly specific blood markers and other characteristics to give the patient the best shot possible for a good transfer, only about one in 430 people end up being suitable match, she said.

There are two types of transfers: a transfer of peripheral blood stem cells or a bone marrow transplant. Ultimately, the patient's doctor choses which route to go, so donors need to be willing to do either.

Donating blood stem cells, which is what Borum did, is a bit like an extended blood draw.

"The first thing to know is my phobia is needles," said Borum. "But knowing that phobias are completely illogical, I went ahead and sent off my swabs. And there wasn't a great chance that I would be a match anyway.

"The prospect of going through a process that's a little uncomfortable for the benefit of saving someone, it's kind of overwhelming," she said. "To be honest, I didn't give it a second consideration, despite the phobia."

She joined the registry in 2013. The mouth swab process took only about 10 minutes, she said.

A little more than two years later, Borum got that call that she was a preliminary match. Did she want to proceed with the process?

Even agreeing at this point isn't the final say in whether you end up donating. First, Borum had her blood drawn and sent off to reinforce that, yes, she was a match. And after that was confirmed she had to get a physical and undergo some pathogen testing, a chest X-ray and some additional testing to make sure she was healthy.

During this process, the identity of the patient is guarded. All Borum was told was that the patient was a 56-year-old man with Hodgkin's lymphoma.

From there it was a repeated process of "test and we'll let you know, test and we'll let you know," she said.

'ONE POKE AT A TIME'

And as each of those came back good, they check to see if you want to proceed. The donor can pull out at any time. But while the donor is going through this process, the patient is going through a parallel preparation of intensive chemotherapy trying to kill as much of the cancer as possible before the transplant.

This is a critical stage for the patient, and if the donor opts out now, it could be life-threatening for the patient, she said.

"I took it one needle poke at a time."

Leading up to the final blood draw, Borum was given several injections of a drug to boost her cell count to help doctors withdraw the stem cells they were looking for.

On the long end, physicians say the blood draw process could take up to six hours. That was their prediction for Borum, who is more petite. During that draw, they run the first samples to a lab to confirm they're getting the right concentration. Borum's body reacted well to the blood cell-boosting drug, and the final process ended up only taking four hours.

To explain her motivation to go through with this procedure, Borum said it's part of her spiritual practice.

"I did it as part of the practice, and recognizing that a lot of things we do are self-referential and self-serving. And I don't think there's a ton of benefit in that, for myself and for others.

"I think the quote from the Dalai Lama goes: If you want others to be happy, have compassion; if you want to be happy, have compassion.

"And it's all a matter of statistics. If there are more people in the registry, it's more likely someone will have a match. If I can benefit one person, it's little effort on my end to try to boost the numbers."

Because of the statistically low chances of finding a match, getting more people on the registry is essential. And the first month or two after the final transplant is a time critical to find out whether their new blood stem cells are working. This doesn't always have a happy ending, said Borum.

But in her case, it did.

BUILDING A BOND

Her transfer was a little more than a year ago. About two months afterward she got word that her cells were successfully making the patient's blood and he had no sign of cancer cells. The patient, who Borum learned lives in New York, sent her a thank you card a few months later, and recently they've exchanged emails.

And in those exchanges, she got her first glimpses into his life.

"I am a husband of 28 years and the father of two boys and was facing a difficult future," he wrote in April of last year. "I am overwhelmed beyond words with this gift you have given me. Please know that I will live the rest of my life with the warmth of your generosity and will do my best to extend it to others in need."

"His email said that he's now been healthy for one year and two months," said Borum. He's also offered to come to Colorado to meet Borum and thank her in person.

His sickness had taken him to a point where walking was a very difficult task, but now he's running again.

The whole process, they say, is about 20 to 30 hours of your time, usually spread over about four to six weeks, according to Be the Match.

"I think that's a small sacrifice to potentially save someone," said Borum.

The patient in this case wasn't available for this article but for pretty much the best reason ever.

Borum said that after being too sick for so long, he was finally leaving for a long-awaited vacation.

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How you can give yourself a chance to save another's life - Glenwood Springs Post Independent

Bone Marrow Stem Cells Comments Off on How you can give yourself a chance to save another’s life – Glenwood Springs Post Independent | April 30th, 2017

Whether your adult mesenchymal stem cells come from bone marrow or from fat probably does not make a difference in terms of clinical results. Although some centers claim that bone marrow derived cells are superior to fat derived cells, there is no evidence to substantiate that. Recent studies show that fat derived cells make bone tissue much better than the bone marrow derived cells. Some studies are showing different outcomes but it is important to realize that these studies are all done in petri dishes and may not relate to living organism. Also, it is important that one is not mislead in some marketing materials by the word bone in bone marrow, possibly implying that since this is an orthopedic source it must be better for treating orthopedic conditions such as cartilage regeneration. In fact, the bone marrow is part of the reticulo-endothelial system (makes blood cells) and just happens to be found in the center of bone. The truth is, both bone marrow derived and stromal (from fat) derived stem cells are both effective for regenerative therapy and both have the potential to differentiate into mature functional cartilage. However, stem cells from fat are 100 to 1000 times more plentiful and this makes same day procedures (allowed in the US) much more effective with fat derived cells. The higher numbers of cells in fat leads to better clinical outcomes. Also, the quality of bone marrow declines with age and it has less numbers of cells and less healthy cells compared to the fat. The diminution in quantity and quality of bone marrow cells related to age and chronic illness is well documented. Last but not least, the ease of removing fat from under the skin using a mini-liposuction under local anesthetic is much less invasive and MUCH LESS painful than undergoing bone marrow aspiration to obtain bone marrow cells.

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Comprehensive-characterization-of-four-different-populations-of-human-mesenchymal-stem-cells-as-regards-their-immune-properties-proliferation-and-differentiation

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Bone Marrow vs. Fat Derived Stem Cells Continued : Stem ...

Bone Marrow Stem Cells Comments Off on Bone Marrow vs. Fat Derived Stem Cells Continued : Stem … | April 29th, 2017

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In my opinion, one of the hardest things to accept is a new type of medical treatment, particularly when it changes the philosophy, parameters and overall results that we are expecting and basically used to receiving. Stem cells are undoubtedly no exception to this rule.

About six weeks ago, Eduardo K (a Cuban doctor with a Masters Degree from the University of Pittsburg in internal medicine and nephrology), brought his wife Maria to our institute, in order to assess the possibility of using stem cells to cure the severe chronic pain in her ankle; a pain so severe that it was basically hindering her ability to walk. Dr. K also expressed his extreme hesitation and concerns about having his wife involved in an invasive ankle surgery at this stage of her adult life.

However, while conducting our usual examining process, reviewing her medical records and MRIs and thoroughly discussing my overall recommendations about a potential stem cell transplant, I quickly realized that Dr. K was not a true believer in Stem Cell therapies, since he thought that there was not much medical evidence of their actual effectiveness and he ultimately also confessed that his wife had basically dragged him to accompany her to this particular appointment.

As always, I respectfully explained the reality that stem cells actually repair the damaged cartilage in a microscopic type fashion and thus, while this repair process would not be clearly reflected immediately on future X-rays, I assured them that the pain she was suffering from will soon subside and possibly even completely disappear. In addition, I expressed that I was extremely confident that she would also regain her mobility skills after the procedure, even if this improvement could not be easily detected via a radiological image.

Since Marias options were somewhat limited, added to the fact that months of traditional physical therapy, injections, medications and previous surgeries had completely failed her, Dr. K finally agreed to grant his wifes wishes to have her stem cell transplant (from her own bone marrow and fat) performed, although he was still very skeptical about the process and was showing little enthusiasm.

This morning, both of them attended our follow up appointment (six weeks after the procedure) and surprisingly, Maria and Dr. K happily confirmed that she felt at least 60 percent better, something that no previous traditional medical treatments had been able to accomplish. It was then that I explained to them that her stem cells had acted much faster than expected (something that possibly taught Dr. K an interesting lesson).

As we began to say our goodbyes, the doctor told me (first in English, then in Spanish) that: in spite of my skepticism about stem cell therapies, I can personally attest that the successful results seen on my wife have been irrefutable, and with a smile on both of their faces, they gratefully thanked my staff and I for this amazing improvement.

As I continued to replay the words expressed by this doctor over and over in my mind, I quickly realized how truly incredulous human beings tend to be, with most of us often needing to fail several times at accomplishing something before being able to realize and accept that we were truly mistaken in the first place!

So if you, a friend or relative would like to receive Stem Cell or PRP treatments, please call us at 305-598-7777. For information visit: http://www.stemcellmia.com (available in both English and Spanish), or watch our amazing video-testimonies on our YouTube Chanel and also please follow us on Facebook and Twitter. If you would like to ask a question directly to Dr. Castellanos, please do so via his direct email: stemdoc305@gmail.com.

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The irrefutable success of stem cell treatments - Miami's Community Newspapers

Bone Marrow Stem Cells Comments Off on The irrefutable success of stem cell treatments – Miami’s Community Newspapers | April 28th, 2017

Ten years ago, the bones currently in your body did not actually exist. Like skin, bone is constantly renewing itself, shedding old tissue and growing it anew from stem cells in the bone marrow. Now, a new technique developed at Caltech can render intact bones transparent, allowing researchers to observe these stem cells within their environment. The method is a breakthrough for testing new drugs to combat diseases like osteoporosis.

The research was done in the laboratory of Viviana Gradinaru (BS '05), assistant professor of biology and biological engineering and a Heritage Medical Research Institute Investigator. It appears in a paper in the April 26 issue of Science Translational Medicine.

In healthy bone, a delicate balance exists between the cells that build bone mass and the cells that break down old bone in a continual remodeling cycle. This process is partially controlled by stem cells in bone marrow, called osteoprogenitors, that develop into osteoblasts or osteocytes, which regulate and maintain the skeleton. To better understand diseases like osteoporosis, which occurs when loss of bone mass leads to a high risk of fractures, it is crucial to study the behavior of stem cells in bone marrow. However, this population is rare and not distributed uniformly throughout the bone.

"Because of the sparsity of the stem cell population in the bone, it is challenging to extrapolate their numbers and positions from just a few slices of bone," says Alon Greenbaum, postdoctoral scholar in biology and biological engineering and co-first author on the paper. "Additionally, slicing into bone causes deterioration and loses the complex and three-dimensional environment of the stem cell inside the bone. So there is a need to see inside intact tissue."

To do this, the team built upon a technique called CLARITY, originally developed for clearing brain tissue during Gradinaru's postgraduate work at Stanford University. CLARITY renders soft tissues, such as brain, transparent by removing opaque molecules called lipids from cells while also providing structural support by an infusion of a clear hydrogel mesh. Gradinaru's group at Caltech later expanded the method to make all of the soft tissue in a mouse's body transparent. The team next set out to develop a way to clear hard tissues, like the bone that makes up our skeleton.

In the work described in the new paper, the team began with bones taken from postmortem transgenic mice. These mice were genetically engineered to have their stem cells fluoresce red so that they could be easily imaged. The team examined the femur and tibia, as well as the bones of the vertebral column; each of the samples was about a few centimeters long. First, the researchers removed calcium from the bones: calcium contributes to opacity, and bone tissue has a much higher amount of calcium than soft tissues. Next, because lipids also provide tissues with structure, the team infused the bone with a hydrogel that locked cellular components like proteins and nucleic acids into place and preserved the architecture of the samples. Finally, a gentle detergent was flowed throughout the bone to wash away the lipids, leaving the bone transparent to the eye. For imaging the cleared bones, the team built a custom light- sheet microscope for fast and high-resolution visualization that would not damage the fluorescent signal. The cleared bones revealed a constellation of red fluorescing stem cells inside.

The group collaborated with researchers at the biotechnology company Amgen to use the method, named Bone CLARITY, to test a new drug developed for treating osteoporosis, which affects millions of Americans per year.

"Our collaborators at Amgen sent us a new therapeutic that increases bone mass," says Ken Chan, graduate student and co-first author of the paper. "However, the effect of these therapeutics on the stem cell population was unclear. We reasoned that they might be increasing the proliferation of stem cells." To test this, the researchers gave one group of mice the treatment and, using Bone CLARITY, compared their vertebral columns with bones from a control group of animals that did not get the drug. "We saw that indeed there was an increase in stem cells with this drug," he says. "Monitoring stem cell responses to these kinds of drugs is crucial because early increases in proliferation are expected while new bone is being built, but long-term proliferation can lead to cancer."

The technique has promising applications for understanding how bones interact with the rest of the body.

"Biologists are beginning to discover that bones are not just structural supports," says Gradinaru, who also serves as the director of the Center for Molecular and Cellular Neuroscience at the Tianqiao and Chrissy Chen Institute for Neuroscience at Caltech. "For example, hormones from bone send the brain signals to regulate appetite, and studying the interface between the skull and the brain is a vital part of neuroscience. It is our hope that Bone CLARITY will help break new ground in understanding the inner workings of these important organs."

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Transparent Bones Enable Researchers to Observe Stem Cells Inside - Laboratory Equipment

Bone Marrow Stem Cells Comments Off on Transparent Bones Enable Researchers to Observe Stem Cells Inside – Laboratory Equipment | April 28th, 2017

Caroline, 11, Elizabeth, 3, Jon Thomas, 13 and James Christopher Allums, 20, do everything as a family. James Christopher and Elizabeth both have a rare medical condition. Their mother, Ellen Allums, said they all go through the process together and support each other with faith and love.(Photo: Courtesy)

Learning that your child has a rare, life-threatening illness is difficult for any family. Everything changes. One family learned that two of their children share the same rare blood disorder.

"That news that we heard was the worst news that we could hear, but it was the best thing that's ever happened to us. It really changed our perspective. It changed our priorities," Chris Allums said.

"We're no longer the same people we were," Ellen Allums said.

Ellen and Chrishave four childrenJames Christopher, 20,Jon Thomas, 13,Caroline,11, andElizabeth, 3.

James Christopher and Elizabeth have Fanconi anemia, a disease that affects the bone marrow's ability to produce blood. Bone marrow or blood stem cell transplants are considered the best treatments, andthey have not yet found a match for either child.

James Christopher was diagnosed 12 years ago and told he had about 18 monthsto live. The family was told he must received a bone marrow transplant.

"We immediately decided that, first of all, we're going to pray and expect a miracle and grow our faith, and next, we're going to try and see if we can find him a bone marrow match and help others along the way, see how many lives that we can affect, that we can save both spiritually and physically," Ellen said.

More than 16,000 people have been added to the worldwide bone marrow registry as a result of drives held on behalf of the Allums. Ellen said they know of at least 41 lives that have been saved because of those efforts, and they're asking more people to commit to donate.

'Looking for a double miracle'

Ellen said a doctor said someone with FA can be like a duck gliding on the water the surface appearance is calm, but people can't see all the effort that goes into staying in motion.

It has a variety of symptoms such as fatigueand can lead to bone marrow or organ failure. Ellen and Chris said FA patients are 500 times more likely to develop some cancers, such as leukemia. James Christopher is subject to constant screenings.

The disease is genetic. According to the National Organization for Rare Disorders, the incidence rate is 1 in 136,000 births. Ellen said her children are two ofsix in Louisiana affected by FA.

Elizabeth's blood counts have been OK, but doctors have said James Christopher has an immediate need for a transplant. DNA needs to be close to an exact match, and many families find a relative who can donate.Elizabeth is a 100 percent match, but she's ineligible because of her FA.

One donor, once found, could help both.A bone marrow transplant won't cure someone with FA, but it can help prolong life.

"Just because you're having to wait doesn't mean the miracle's not going to come. We've been waiting 12 years, but we still have faith that that miracle's coming. Just because it hasn't happened doesn't mean it's not going to. The timing needs to be right," Ellen said. "In our lives, we're looking for an even greater miracle because we're looking for a double miracle, with two children."

FA patients can require blood and platelet transfusions, after which they may become dependent and need additional rounds, which would require a bone marrow transplant quickly.

James Christopher received his first blood transfusion three weeks ago.

"Chris gave. His daddy gave blood to him, and we felt like it was his heavenly father and his earthly father that gave him that blood, and now we're praying and believing that he never has to receive it again," Ellen said.

She said they've dealt with some scary bleeding issues "like Niagara Falls," and James Christopher has almost lost his life a few times. His parents call him a survivor, a warrior. He gets up and stays active daily, even with low blood counts that doctors thinkwould cause fatigue.

"I love to prove doctors wrong. If they give me a boundary, I want to cross it, definitely, when it comes to that," he said. He likes to tell people "keep calm and carry on," like the World War II posters.

Every bump, scratch, scrape and bruise for the siblings is noteworthy, and the whole family works to avoid germs. A simple cough or cold could be devastating, so they're all in tune to notice illness.They're very aware of the importance of handwashing and staying home if ill. Chris said during cold and flu season, they often come in, shower and change clothes before interacting with the others.

Ellen said they respect people who choose not to vaccinate, but all of her children have been vaccinated because measles or chicken pox can kill someone with FA.

All the children home school to help prevent illness. When James Christopher was diagnosed, doctors said it could help him live longer. Chris said all four have excelled fromthe one-on-one time, and they've enjoyed getting to know other families inthe Christian Homeschool Association.

The Allums know their lives are different than those of many other families, but they are running their own race.

"I have to tell you that we have a wonderful life. Sure it's full of hard work, but it's wonderful because of what the Lord has done with it," Chris said.

Read more:Mom says prayer pulled her through transplant|Facing the storm: Mother shares unbelievable story|Big brother to the rescue: Man gives sister half of liver|Man saves 10 in life, death

Joy in the journey

The couple did their homework on hospitals that specialize in the disease and settled on Memorial Sloan Kettering Hospital's cancer center in New York. It had the best survival rates, and they've been going for 12 years.

James Christopher's and Elizabeth's immunity is low, the family cannot travel with the general public. They either have to make the almost 20-hour drive or arrange for a private plane. Ellen said they've had to go there, at times, every three to six months.

The whole family travels to medical appointments.

"Although they don't have the disease, they go through it with them," Ellen said of Jon Thomas and Caroline. She said all of her children have gone to hospitals and played with and prayed for children were facing terminal diagnoses. It's been a blessing to them and a ministry to others.

James Christopher said they try to find fun in the journey. Ellen said they do something fun every time they go to the hospital and embrace John 10:10Jesus came that we might have life and have it abundantly.

James Christopher Allums, 20, holds his sister Elizabeth Allums, 3. The siblings both have a rare medical condition called Fanconi anemia.(Photo: Courtesy)

What happens if there's a match?

"We would be moving to New York for six to eight months for the bone marrow transplant," Ellen said.

Ellen said the a bone marrow recipient with FA will have to go through chemotheraphy for two weeks to kill off the patient's natural bone marrow.

"When the cells are dead, then they receive someone else's bone marrow. It's a liquid, it looks just like an IV, and they lie there and you just pray to God that it's going to take," she said.

After the transplant, the patient is in isolation for 30-40 days. They stay at the transplant hospital for six to eight months and keep a medical mask on for one year. Chris said you hope graph vs. host disease isn't an issue.

Saving lives

She said she used to look at missions that dig wells in other countries and wish they could go save lives, but, after prayer, she realized they are saving people. With the help of family and friends, efforts to add bone marrow donors have helped dozens of people.

"I like to tell people 'You could be the reason someone lives.' ... And I think those words are pretty powerful" Ellen said.

She said the process to donate blood stem cells, which is the most common donation method, involves a needle in each arm for four to six hours.

"It's not even a surgery. It's not like giving a kidney or a lung or a heart, even, but the benefits are that strong. It can truly save a life, but yet all you have to do is like giving blood," Ellen said.

To test for a match, she said, it's even less of a commitment. It takes about five minutes to fill out paperwork and provide a swap from inside the cheek. Anyone 18-55 in good health can register.

The community has come together to help organize a drive for May 1, National Fanconi Anemia Day. A massive drive will take place at more than a dozen locations across northeastern Louisiana, and CenturyLink will be registering employees on-site.Anyone anywhere can order testing kitsonline atdkms.orgorbethematch.org.

A month after testing, people will get a phone call to confirm their position on the registry. Ellen said they pray people will make the commitment.Previous drives for the Allumshave set national records for most registered in one day. Over three days, they tested 5,000 people.

"When people come, we want to educate them on the processin hopes thatwhetherthey are a match in a month or a match in 20 yearsthat they will be committed to beingon that registry to help somebody," Chris said.

They heard of a woman who registered with her family at a previous event andlater developed leukemia. Her sister was found as an instant bone marrow match because theyalreadyhad been tested.

Ellen and Chris said knowing that 41 lives were saved as a result of their family'sefforts makes it all worth it, even though it hasn't been easy.

"But we believe that God is going to heal them both because He told us He would, and we believe that. We hold on to those promises of God. ... and we focus on that. That gives us strength," Ellen said.

Follow Bonnie Bolden on Twitter@Bonnie_Bolden_and on Facebook athttp://on.fb.me/1RtsEEP.

Want to register?

May 1 is National Fanconi Anemia Day, and a more than a dozen locations across northeastern Louisiana will be part of a single registration drive. Times vary and new locations may be added. Check The Friends of James Christopher and Elizabeth Allumson Facebook or visitcaringbridge.organd searchJames Christopher Allums.

Or order a testing kit online at dkms.org or bethematch.org.

Testing sites and times are:

Monroe

West Monroe

Surrounding parishes

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ow you see it, now you dont: Scientists have used a chemical technique to make mouse bones turn transparent. The technique has been used in the past to make brains and kidneyssee-through, but this marks the first time its been used in hard tissues.

The ability to see within a bone couldhave implications for research into bone diseases, by letting researchers get a more accurate picture of bones internal structure.

The technique is called CLARITY, and since 2013, when it was first described, it has been deployed on a wide variety of mammalian tissues and inplants. Caltech neuroscientistViviana Gradinaru, an original developer of the technique, even cleared an entire mouses body in 2014 (except for its bones, which were unaffected, she said).

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The approach works by chemically locking proteins and DNA in place with a hydrogel, after which researchers wash away fats within the tissue. Lipids refract light, so this washing step makes CLARITY-treated tissues transparent.

Flexible 3-D printed scaffolds could mend broken bones

In this case, Gradinaruwanted to look at bone marrow and count the number of stem cells that could ultimately produce new bone cells.

Bone is not a static organ. It iscontinuously changed. The bones we have in our body, we didnt have them 10 years ago, she explained. Acontinuous process of bone cell death and bone cell growth ishappening, spurred by progenitor cells in a bones soft, spongy marrow.

But looking for these cells can bechallenging. There arent that many progenitor cells, soextrapolating the number and distribution based ona small sample isnt ideal. Researchers can slice the bone, but cuttingcan damage the edges. Putting images of the sliced bones back together into a coherent, 3-D picture is very difficult, too.A clear bone avoidsslicing altogether.

Doug Richardson, director of imaging at the Harvard Center for Biological Imaging, said the paper represented a step forward in bone clearing. (Richardson was not involved in this research.)

This technique has the potential to monitor bone health or disease progression over larger volumes with greater accuracy, he said.

Gradinarus team has already demonstrated one possible application. They found that a drug for osteoporosis, currently being developed by Amgen, triggered an increase in the number of stem cells in CLARITY-treated bone.Some Amgen scientists were coauthors of the paper.

Using CLARITY let the team more effectively measure the rate of this increase.This is very important, because you want a controlled increase too much of an increase can lead to tumors, Gradinarusaid.

Other uses could be on the horizon. Being able to make a mouse or rat skull see-through could be useful for Gradinarus fellow neuroscientists who use implants in their research and want to establish the exact position of the impact after experiments are done.

Theres still more work to be done. For instance, finding a way to tagthe samples with antibodies without having to cut a bone in half, as researchers did in this paper would be ideal.Gradinaru also wouldnt mind some speed improvements:In this case, the CLARITY process took nearly a month.

Its not a fast method, by any means, Gradinaru said. However, the result theres no substitute for getting 3-D access to the intact bone marrow.

Kate Sheridan can be reached at kate.sheridan@statnews.com Follow Kate on Twitter @sheridan_kate

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Bone marrow donor forgot he'd registered Jewish Chronicle My phone rang and when I answered they said someone needed my stem cells. They asked me would I still like to donate? I went in the next day for tests and when I was deemed fit and healthy they got me to come back in for the procedure. On Tuesday the ...

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