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Archive for the ‘Bone Marrow Stem Cells’ Category

Opinion/Commentary: Seniors put at risk by outdated Medicare policies – The Daily Progress

Almost 30 years ago, the federal government helped make it easier for patients with leukemia and lymphoma to receive lifesaving stem cell transplants. Now, we need the federal governments help again to ensure that Medicare patients with these cancers and other serious blood disorders can access the care they need.

In 1987, Congress approved funding for a national database of patients willing to donate bone marrow or peripheral blood stem cells. That database is now known as the Be The Match Registry, operated by the National Marrow Donor Program/Be The Match. According to the NMDP/Be The Match, patients searching the registry have access to 27 million potential volunteer bone marrow and peripheral blood stem cell donors worldwide, along with more than 680,000 units of cord blood donated by mothers after giving birth.

Having access to such a large registry has made it easier for patients to find a match if they dont have a fully matched sibling donor, which is the case for about 70 percent of patients who receive a stem cell transplant. The registry has helped 80,000 patients receive bone marrow transplants, peripheral blood stem cell transplants, or cord blood transplants from an unrelated donor.

While the federal governments foresight and financial support have helped make adult stem cell and cord blood transplants the only cure available for these diseases possible for thousands of patients, Medicare coverage policies have not kept pace with this breakthrough treatment.

Medicare is more restrictive than private insurance companies in deciding for what indications stem cell transplants and cord blood transplants will be covered. With private insurance companies, we have the opportunity to talk with a medical director about the indication and provide literature to support the decision for a transplant. This opportunity is not available for our Medicare patients.

In most cases, Medicare doesnt decide whether to cover a stem cell or cord blood transplant until after the procedure is completed. This leaves most Medicare patients an impossible choice: Turn down their only chance at a cure or potentially face paying the significant cost of a transplant themselves. Even when Medicare does decide to reimburse for these transplants, according to the NMDP/Be The Match, it covers less than half the cost of the transplant.

Addressing this issue is especially important because seniors make up a large portion of the patients with the cancers and blood diseases that can be cured by a stem cell or cord blood transplant. For example, 24 of the 65 patients who received stem cell or cord blood transplants at University of Virginia Health System in 2016 had Medicare coverage.

So I am asking the Centers for Medicare & Medicaid Services to expand Medicare coverage for stem cell and cord blood transplants, along with paying for the search and procurement costs as they already do for solid organ transplants.

The federal government has helped save the lives of tens of thousands of patients through better access to stem cell and cord blood transplants. I hope now they will act to make sure all Medicare patients who need one of these transplants can receive it.

Tamila L. Kindwall-Keller, DO, MS is the associate clinical director of the Stem Cell Transplant Program at the University of Virginia Health System.

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Opinion/Commentary: Seniors put at risk by outdated Medicare policies - The Daily Progress

‘Reset’ for the immune system – Burlington Times News

By Jessica Williams / Times-News

Shawna Hamlett will be born again June 8.

The 40-year-old Alamance native and single mother was diagnosed with relapsing remitting multiple sclerosis 13 years ago, and has dealt with episodes of numbness, sudden blindness, deafness, difficulty walking, pain and extreme fatigue.

But May 1, Hamlett will travel to Chicagos Northwestern Hospital to undergo a month-and-a-half-long experimental procedure called hematopoietic stemcell transplant, which uses stemcells from the patients own bone marrow, along with chemotherapy, essentially toreset the immune system.

By the time she receives the stemcell transplant June 8, she will have lost her hair and nearly all of the antibodies built up from vaccines that shes received since birth, but will no longer have multiple sclerosis.

This is mind-boggling to me, Hamlett said. My body wont remember that I had MS at all because my stemcells are going to be frozen [for] two weeks, and when they give them back, they dont know they had MS ever. The chemo completely reboots your immune system so much so that the vaccines you got when you were an infant your body no longer has.

She applied for the procedure in November and was, to her surprise, approved for it in late January. It couldnt come a moment too soon, Hamlett said, as the neurologist in Chicago told her during a consultation that shes one legion away from a catastrophic event maybe even paralysis, an even greater concern considering that she has two sons, 13 and 17.

I just wish I had done this years ago, when the doctor first started doing it, because I could have had their whole childhood without having it, and Ive had to struggle at ballgames. Ive been on the sidelines at soccer games with an IV in my hand because Ive had an IV at home and Im not going to miss their games, Hamlett said. But theyre going to grow up, and theyre going to get married and have kids, and I dont want to be not able to be around.

AS WITH ANY MEDICAL procedure, a heavy cost is involved. Along with spending the better part of nearly two months away from her kids, the longest shes ever been away from them, Hamlett will have to pay $25,000to $35,000 after insurance.

Shes enlisted help from community businesses, friends and family to help cover that expense, and has plans for seven fundraisers.

Hamlett says community support is essential for people with MS, and she would advise anyone just diagnosed to seek out others.

Talk to people who are in the same boat as you, she said. Theres a huge community of people on both Instagram and Facebook that suddenly become your family because nobody, we always say, nobody gets it until they get it. People who understand exactly what youre going through are out there, so youre not ever alone.

REGARDLESS OF HOW much money is raised, Hamletts life is about to change for the better. Shes nervous, but eager, and says its going to be a huge relief when shes fully-recovered.

The thing about MS is you never know from day to day how its going to be the next day, which is good and bad because, if its bad, then tomorrow could be a good day, but if its good, then tomorrow could be a bad day, she said. Id like to be on an even keel and just know that after the procedure there wont be any more unknowns.

Hamlet has set up a donation page through HelpHOPELive for donors to contribute to the cost of the procedure and hotel expenses for her time in Chicago. Visit https://helphopelive.org/campaign/12529 or search Shawna Hamlett HelpHopeLive to find out more.

All donations are tax-deductible, held by HelpHOPELive in the South Atlantic Stem Cell Transplant Fund, and administered by HelpHOPELive for transplant-related expenses only.

Reporter Jessica Williams can be reached at jessica.williams@thetimesnews.com or at 336-506-3046. Follow her on Twitter at @jessicawtn.

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'Reset' for the immune system - Burlington Times News

Scientists wage fight against aging bone marrow stem cell niche – Science Daily


Science Daily
Scientists wage fight against aging bone marrow stem cell niche
Science Daily
Red staining reveals the abundant presence of the protein osteopontin (OPN) in bone and endosteum of the marrow cavity, which is important to maintaining a healthy environment for blood-forming hematopoietic stem cells. Shown in blue are cell nuclei.

and more »

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Scientists wage fight against aging bone marrow stem cell niche - Science Daily

Gene therapy shows early promise against sickle cell – Chicago Tribune

Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

RELATED: TRENDING LIFE & STYLE NEWS THIS HOUR

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the U.S., about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant. In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team led by Dr. Marina Cavazzana focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin to replace the abnormal "adult" hemoglobin seen in sickle cell.

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published in March in the New England Journal of Medicine.

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Gene therapy shows early promise against sickle cell - Chicago Tribune

Edmonton police officer appeals for bone marrow donor to save 8-year-old son – CTV News

An Edmonton police officer is counting on the city he serves to help save his sons life.

Brady Mishio, 8, has an aggressive form of leukemia and needs a bone marrow transplant. His dad Terry desperately hopes someone who is a match will hear his plea.

Bradys family received the news of his illness in November after they took him for treatment of a persistent fever and flu.

You become that family that's searching for hope and have all these dreams and things for your children, and then one day, one day it's taken away from you so quickly, said Mishio.

The diagnosis was a form of cancer that starts in blood stem cells called acute myelogenous leukemia (AML). It is the most common leukemia found in adults but is less common in children. Brady began chemotherapy right away.

Four days into chemo he had a reaction to some of the drugs that they were giving him and he quit breathing and had a seizure, said Mishio, a 20-year veteran of the Edmonton Police Service who was once forced off work for a year with a brain injury after being kicked while making an arrest.

Bradys doctors found a drug combination that worked and the boy just finished his third round of treatment. He is in an isolation unit in hospital and the next step is a bone marrow transplant.

It gives Brady a second chance at life, at cleaning out his bone marrow and hopefully eliminating the cancer, said his father, while fighting tears.

Bradys family is not a match and Mishio is reaching out publicly, hoping he can help his child by finding a suitable donor. The test is a simple mouth swab. Donors must be between 17 and 35 and be in relatively good health, says Robyn Henwood, a stem cell territory manager for Canadian Blood Services. Younger people have fewer antibodies in their blood, lowering the risk of rejection.

Once registered, potential donors stay in the agencys registry until they are 60 years old.

Less than 25 per cent of patients find a bone marrow match in their own family, says Henwood.

So every single person we add to our database is giving hope to those who are looking for a match or who are likely going to die if we don't find them one.

In most cases, the process to donate bone marrow is much like giving blood and takes four to six hours to complete.

A clinic is being held Thursday, March 9 from 4 p.m. to 8 p.m. at Holy Cross Ukrainian Catholic Church. Interested donors who cant make that clinic can be tested at the Canadian Blood Services clinic next to University Hospital or register at blood.ca to be mailed a swab kit.

Mishio is amazed by the bravery of his son through this battle.

He's a fighter and there's many days where he'll be rubbing my back and kind of telling me it's going to be OK, and that's when I'm like, 'I got to be strong for him.'

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Edmonton police officer appeals for bone marrow donor to save 8-year-old son - CTV News

Family appeals for bone marrow donor to save father – Easterneye (press release) (subscription)

THE family of a man urgently in need of a bonemarrow transplant has appealed to the Asian community to donate their stem cells in the hope of finding a suitable match to save his life.

Father of two, Yevi Ilangakoon, was diagnosed with myelofibrosis in 2009. It is a rare condition where scar tissue builds up inside the bone marrow, affecting its ability to create healthy blood cells, which affects one person in every 100,000.

The 63-year-old, who is originally from Sri Lanka and now lives in Whitstable, has seen his health deteriorate rapidly and his illness could now progress into leukemia if he is not treated.

His only option is to have a bone-marrow transplant using stem cells. However, specialists have been unable to find a 100 per cent match despite searching worldwide registers.

From the entire register, only four per cent are from a south Asian background.

Yevis son Yovaan told Eastern Eye: Its a lifethreatening disease and has been managed with medication for the past eight years, but the condition has got more and more aggressive, especially over the last few months. If he doesnt have a stem-cell transplant, it will be a few months to a year that he will have to live.

So it is quite crucial that we get as close to 100 per cent match as we can. He gets very, very tired because his hemoglobin levels are low. If he has an injury, it takes ages to heal. We are praying and being positive and trying to raise awareness.

Yovaan highlighted the issue on social media, which attracted the attention of Sri Lankan cricketer Mahela Jayawardena, but the family are still urging members of the public to get on the bone marrow register to find a match for Yevi.

The 29-year-old added: It could be your family member your mum or your dad, you dont know what position you are going to be in in a few years time.

If you are on the register, you have the chance of saving someones life. Its a really easy process.

Signing up online takes two minutes and participants simply need to swab the inside of their cheek with a cotton bud they are sent, and send it back in a pre-paid envelope.

Sarah Rogers of the Anthony Nolan charity said: We urgently need more people from Indian and South Asian backgrounds to register as stem cell donors to make sure that everyone, regardless of background, can receive a second chance at life.

At the moment we find a perfect match for about 60 per cent of northern European patients who need a transplant, but that drops to around 20 per cent for any patient of ethnic minority.

If you are above 30, go to: http://www.dkms.org.uk/en/ register-now. Under 30, register at http://www.anthonynolan.org/apply-join-bone-marrow-register.

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Family appeals for bone marrow donor to save father - Easterneye (press release) (subscription)

Gene Therapy: A Breakthrough for Sickle Cell Anemia? – Twin Falls Times-News

WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Twin Falls Times-News

This simple method can predict a stem cell’s fate – Futurity – Futurity: Research News

Scientists have created an easy way to identify the state and fate of stem cells earlier than previously possible.

Understanding a stem cells fatethe type of cell it will eventually becomeand how far along it is in the process of development can help scientists better manipulate cells for stem cell therapy.

Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases.

The beauty of the method is its simplicity and versatility, says Prabhas V. Moghe, a professor of biomedical engineering and chemical and biochemical engineering at Rutgers and senior author of a study published recently in the journal Scientific Reports. It will usher in the next wave of studies and findings.

Existing methods look at the overall population of cells but arent specific enough to identify individual cells fates. But when implanting stem cells (during a bone marrow transplant following cancer treatment, for example), knowing that each cell will become the desired cell type is essential.

Also, many protein markers used to distinguish cell types dont show up until after the cell has transitioned, which can be too late for some applications.

To identify earlier signals of a stem cells fate, scientists used super-resolution microscopy to analyze epigenetic modifications. Epigenetic modifications change how DNA is wrapped up within the nucleus, allowing different genes to be expressed.

Some modifications signal that a stem cell is transitioning into a particular type of cell, such as a blood, bone or fat cell. Using the new method, the team of scientists was able to determine a cells fate days before other techniques.

Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases, says Rosemarie Hunziker, program director for tissue engineering and regenerative medicine at the National Institute of Biomedical Imaging and Bioengineering. Its a relatively simple way to get a jump on determining the right cells to use.

The approach, called EDICTS (Epi-mark Descriptor Imaging of Cell Transitional States), involves labeling epigenetic modifications and then imaging the cells with super resolution to see the precise location of the marks.

Were able to demarcate and catch changes in these cells that are actually not distinguished by established techniques such as mass spectrometry, Moghe says.

He described the method as fingerprinting the guts of the cell, and the results are quantifiable descriptors of each cells organization (for example, how particular modifications are distributed throughout the nuclei).

The team demonstrated the methods capabilities by measuring two types of epigenetic modifications in the nuclei of human stem cells cultured in a dish. They added chemicals that coaxed some of the cells to become fat cells and others to become bone, while another set served as control.

Within three days, the localization of the modifications varied in cells destined for different fates, two to four days before traditional methods could identify such differences between the cells. The technique had the specificity to look at regional changes within individual cells, while existing techniques can only measure total levels of modifications among the entire population of cells.

The levels are not significantly different, but how theyre organized is different and that seems to correlate with the fact that these cells are actually exhibiting different fates, Moghe says. It allows us to take out a single cell from a population of dissimilar cells, which can help researchers select particular cells for different stem cell applications.

The method is as easy as labeling, staining, and imaging cellstechniques already familiar to many researchers, he says. As the microscopes capable of super resolution imaging become more widely available, scientists can use it to sort and screen different types of cells, understand how a particular drug may disrupt epigenetic signaling, or ensure that stem cells to be implanted wont transform into the wrong cell type.

Collaborators are from Stanford University School of Medicine, Case Western Reserve University, Seoul National University, Princeton University, the University of Akron, the University of Pennsylvania, and MIT.

Source: Teal Burrell for the National Institute of Biomedical Imaging and Bioengineering via Rutgers University

Originally posted here:
This simple method can predict a stem cell's fate - Futurity - Futurity: Research News

Gene therapy ‘cures’ boy of blood disease that affects millions | New … – New Scientist

Many rely on blood transfusions

Joe Amon/The Denver Post via Getty

By Andy Coghlan

A TEENAGE boy with an inherited disease that affects millions worldwide seems to have been cured using gene therapy. The treatment appears to have stopped the painful symptoms of sickle cell disease, demonstrating the potential for gene therapy to treat common genetic diseases.

All the blood tests we performed show that the teenager has been cured of sickle cell disease

The idea of gene therapy using strands of DNA to compensate for a persons malfunctioning genes is almost three decades old. However, the approach has so far mostly been used to treat very rare diseases (see Long road to success). In contrast, sickle cell disease affects 100,000 people in the US alone. If the treatment proves successful in larger trials, it could bring gene therapy into widespread use.

It could be a game changer, says Deborah Gill at the University of Oxford. The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.

People with sickle cell disease make abnormal versions of haemoglobin, the blood protein that carries oxygen around the body. This can be caused by mutations in the gene that makes a subunit of haemoglobin, called beta-globin. The mutations cause haemoglobin to clump together, distorting red blood cells into a sickle-shape that can get stuck in blood vessels around the body.

People with the disorder are given blood transfusions to clear these painful blockages and prevent new ones. Bone marrow transplants can treat the disease, but matching donors can only be found for around 10 per cent of people with the condition.

Now a team in France seems to have developed a treatment that would work for everyone with the disorder. First, the team took bone marrow stem cells from the boy when he was 13, and gave them extra, mutated versions of the gene that codes for beta-globin. These were designed to make beta-globin that would interfere with the boys faulty proteins, stopping them from clumping together.

The researchers then put these stem cells back into the boys body. After around three months, he began producing large quantities of haemoglobin that behaves normally (New England Journal of Medicine, DOI: 10.1056/NEJMoa1609677). The patient is now 15 years old and free of all previous medication, says Marina Cavazzana at the Necker Childrens Hospital in Paris, who led the team. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers.

Cavazzana is confident these benefits will last. All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up. She says her team has treated seven other patients, who are showing promising progress.

We are all very excited by the work, and this success provides support for this and other genetic strategies targeting this horrible disease, says John Tisdale at the US National Heart, Lung, and Blood Institute in Maryland.

David Williams, at Boston Childrens Hospital in Massachusetts, suggests that the boy may still occasionally experience blockages, because his own original genes are still able to produce faulty haemoglobin. Its important to see what happens over time, and how many other patients see similar benefits.

However, should the gene therapy prove to be effective in larger trials, its expense may limit its use to richer nations. We should be realistic in remembering that there are hundreds of thousands of sickle cell patients in less developed countries, and that the therapy is not easily exportable or adaptable to countries with less well-developed health systems, says Stuart Orkin at Harvard Medical School.

Twenty years ago, gene therapy was touted as a cure for everything from cancer to cystic fibrosis. Now it is finally starting to fulfil its promise.

In 2012, Glybera became the first gene therapy to be approved, for people with a rare disorder that makes them unable to process dietary fat. Last year, the first commercial gene therapy that alters a persons DNA was approved for children with a severe immune disorder. Gene therapies for rare forms of blindness are also showing promise.

But these conditions all affect very small numbers of people. Research into sickle cell disease (see main story), beta thalassaemia, haemophilia and cystic fibrosis mean gene therapy may not be too far from becoming mainstream medicine for the most common genetic diseases.

This article appeared in print under the headline Gene therapy breakthrough

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Gene therapy 'cures' boy of blood disease that affects millions | New ... - New Scientist

Gene Therapy: A Breakthrough for Sickle Cell Anemia? – Auburn Citizen

WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Auburn Citizen

Stem cell: Knee arthritis in new $33 million research plan – Capitol Weekly

News

by DAVID JENSEN posted 03.02.2017

The California stem cell agency this week approved nearly $33 million for clinical stage research projects testing treatments for type 1 diabetes, arthritis of the knee, ALS and an immunodeficiency affliction.

The awards were quickly approved with little discussion during a meeting at the Oakland headquarters of the California Institute for Regenerative Medicine or CIRM, as the agency is formally known.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agencys application review summary

The award likely to have an impact on the most people if it is successful is a relatively small, $2.3 million award to the Cellular Biomedicine Group, a Chinese firm with operations in Cupertino, Calif. The stem cell agency by law only finances work in Clifornia. The research would also be supported by $572,993 in co-funding.

The project is aimed at treating osteoarthritis of the knee. More than 51 million people in the United States suffer from arthritis, which is particularly common in the knee.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agencys application review summary. The funding would go to manufacture the product and complete work to secure Food and Drug Administration approval for a phase one safety trial. A treatment for the public would likely be years in the future.

Here are the other winners today of California stem cell cash with links to the summaries of the reviews.

Caladrius Biosciences of New Jersey won $12.2 million for a clinical trial for young people ages 12-17 for newly diagnosed type 1 diabetes. The firm plans to use regulatory T cells from the patients themselves to treat the disease. Caladrius has a California location in Mountain View. (Caladrius press release can be found here.)

St. Judes Research Hospital in Memphis, Tenn., was awarded $11.9 million for a phase one/two trial to treat infants with X-linked severe combined immunodeficiency. The trial would aim at enrolling at least six patients suffering from the catastrophic affliction. The treatment would use the patients own bone marrow stem cells after the cells were specially handled. The agency said in a press release that St. Judes is working with UC San Francisco. (St. Judes press release can be found here.)

The awards were previously approved behind closed doors by the agencys out-of-state reviewers, who do not disclose publicly their economic or professional interests.

Cedars-Sinai Medical Center in Los Angeles was awarded $6.2 million for a phase 1/2A trial to test a treatment for ALS, which has no treatment or cure. The CIRM review summary said a huge unmet need existed. About 20,000 persons in the United States suffer from the affliction.

CIRMs press release did not identify the researchers involved in any of the awards.

The agency is on a push to support more clinical trials, which are the last and most expensive research prior to the possibility of winning federal approval for widespread use of a therapy.

Currently the agency is participating in 27 trials and is planning on adding 37 more in the next 40 months. The agency is expected to run out of funds for new awards in June 2020 and has no source of future financing.

The awards were previously approved behind closed doors by the agencys out-of-state reviewers, who do not disclose publicly their economic or professional interests. The agencys directors rarely overturn a positive decision by the reviewers.

All of the winners have links to two or more members of the 29-member CIRM governing board. Those members are not allowed to vote on applications where they have conflicts of interest.

About 90 percent of the funds awarded by the board since 2005 have gone to institutions that have ties to members of the board, past or present, according to calculations by the California Stem Cell Report. Eds Note: David Jensen is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared. He has published more than 4,000 items on California stem cell matters in the past 11 years.

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Stem cell: Knee arthritis in new $33 million research plan - Capitol Weekly

NBC-5 news anchor Rob Stafford to undergo bone marrow … – Chicago Tribune

NBC-5 news anchor Rob Stafford has been diagnosed with a rare blood disorder and will take a leave of absence from the station for several months as he undergoes a bone marrow transplant and chemotherapy.

Stafford credited his wife, Lisa, for pushing him to get an early diagnosis that doctors say greatly improved his prognosis. He is scheduled to begin treatment Friday at the Mayo Clinic in Rochester, Minn.

"I spent my whole life asking questions, but I asked very few questions about my own health because all I wanted to hear is I'm OK," Stafford said in an interview at his Hinsdale home Tuesday. "You have to ask questions."

Stafford told colleagues at WMAQ-Ch. 5 about his illness in an email Wednesday morning and planned to announce the news publicly Wednesday at the end of the 10 p.m. newscast.

He expects to be away from his desk anchoring the 5, 6 and 10 p.m. weekday news for four to six months while at the Mayo Clinic. Doctors there say Stafford is in the early stages, Stage 2, of the disease, which can lead to heart failure and death if undetected.

Stafford's disorder, called amyloidosis, occurs when an abnormal protein called amyloid is produced in bone marrow that can be deposited in tissues and organs. There are more than 40 types of the disorder that affects the heart, kidneys, liver, spleen, nervous system and digestive tract. Stafford's type known as light chain amyloidosis is rare, with fewer than 2,000 or so cases diagnosed in the U.S. each year, according to Dr. Ronald Go, Stafford's hematologist at the Mayo Clinic. Doctors are optimistic he will go into remission after treatment.

Before his diagnosis in January, Stafford had noticed lower energy levels while biking the Cal-Sag trail and hiking through the Rocky Mountains while on vacation. But the 58-year-old award-winning investigative reporter and father of three adult children dismissed the episodes as signs of getting older, he said.

After a physical detected slightly high cholesterol levels and more than usual amounts of protein in his urine, both indicators that his kidney had been damaged as a result of the blood disease, Stafford's wife insisted that he take the symptoms seriously.

"I believe there's a reason that this happened, and I think there's a calling that is to let people know the importance of early detection," said Lisa Stafford, his wife of 30 years who owns a medical marketing and communications company. She plans to stay with Stafford in temporary housing near the Mayo Clinic as he undergoes treatment, lives in a sterile environment and rebuilds his immune system while recovering from the effects of chemotherapy.

In his email to fellow staffers, Stafford joked that he did not wait for the end of sweeps to schedule treatment, but that Friday was the first opening on the Mayo schedule after Stafford completed all the required medical tests.

"I consider this early diagnosis a gift that left to my own devices I would not have received," Stafford wrote. "I'm going to take full advantage of my good fortune and hit this head on with the most aggressive and proven treatment available."

Stafford joined NBC-5 in 2009 after working as a Chicago-based correspondent for Dateline NBC for 11 years. Prior to that, he was a general assignment reporter at CBS2 Chicago. He has won two national and seven local Emmy awards, and an Edward R. Murrow award for a Dateline investigation into racial profiling.

Stafford said his experience meeting and interviewing people in the news, often "on their worst days," has allowed him to keep his health issues in perspective.

"This thing pales in comparison to 95 percent of the stories I do on a daily basis," said Stafford, who added that his work as a journalist also has helped him to seek out the best support, medical opinions and advice about the disorder.

During treatment, doctors will remove, or "harvest," stem cells from Stafford's own bone marrow and freeze millions of healthy ones. Chemotherapy then will be used to wipe out all of his bone marrow, including the unhealthy cells. As the final part of the treatment, doctors will transplant the healthy stem cells back into Stafford's bone marrow, and they will reproduce themselves, Go said.

"You're rescuing your bone marrow by the stem cells that you stored," Go said.

Although Stafford's parents were both treated for cancers in their 50s, doctors do not know the exact cause of Stafford's illness and do not believe it was is hereditary. Risk factors for amyloidosis include exposure to chemicals, radiation and aging, Go said.

Stafford confided in his NBC-5 co-anchor, Allison Rosati, and meteorologist Brant Miller about his illness shortly after his diagnosis in January. As medical tests forced him to travel, he alerted NBC-5 managers, who encouraged Stafford to do whatever it took to get the medical attention needed, he said.

"Rob is loved by his colleagues in the newsroom. We are encouraged by the news that his illness was detected early, and that he is in the excellent hands of the top doctors at the Mayo Clinic. We all wish Rob the best of luck in the weeks ahead, and we can't wait for his return to the newsroom," station manager and vice president of news Frank Whitaker said in an email.

NBC-5 weekend anchor Dick Johnson will be filling in for Stafford during his absence.

Stafford is expected to lose his hair as a result of the chemotherapy and has experienced weight loss. Doctors have encouraged him to pack on a few pounds in anticipation of further weight loss. He's obliged by indulging on Girl Scout cookies, deep dish pizza, cheeseburgers and milk, he said.

Stafford said he is trying to take emotion out of his upcoming battle, which he is approaching as a fight that he plans to win.

"I am not freaking out because I really am confident I am going to get through this," he said.

Lisa Stafford will be posting updates about her husband's condition on a blog: staffordrecovery.com. Rob Stafford also will offer updates on his Facebook Fan page.

Stafford's announcement came less than 24 hours after Hosea Sanders, veteran news anchor at WLS-Ch. 7, announced on his Facebook page he will undergo surgery for prostate cancer and that he was "very optimistic about the outcome."

Sanders told social media friends Tuesday night that he had been diagnosed several weeks ago and would be taking some time off from the ABC-owned station, for which he anchors the 7 p.m. newscast each weeknight.

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NBC-5 news anchor Rob Stafford to undergo bone marrow ... - Chicago Tribune

Exactly What It’s Like to Donate Bone Marrow and Why More … – Yahoo Health

Abby West first decided to become a bone-marrow donor when she was a young journalist covering a bone-marrow drive. Little did she know that nearly two decades later, shed be called upon to try to help save someones life.

The newspaper I had joined had a reporter who had passed away of sickle cell anemia, and I had become aware of the need for African-Americans to join the registry, West, a senior editor at Yahoo Celebrity, tells Yahoo Beauty about her decision to sign up with Be The Match. I was covering a bone-marrow drive for African-Americans, and it seemed like the right thing to do. West says she simply got her cheek swabbed which yields a sample of cells that doctors use to compare specific protein markers with those of patients who need a bone-marrow transplants and then went about her life.

But in June 2014 17 years later she received an email from Be the Match, a registry of the National Marrow Donation Program, saying that her bone marrow was a potential match for someone in need. She called the organization to learn more. You get a sense of urgency, need, and what is expected of you, she says. To make sure she was a healthy donor, West needed to fill out forms, and once it was determined she met all of the donor criteria, she moved on to a physical examination.

(Photo courtesy Abby West)

West says she underwent seven weeks of blood testing and prep work before she donated bone marrow. Part of the process was making a decision about whether she would do a surgical bone-marrow donation, in which a person is put under general or local anesthesia and liquid marrow is taken from the back of the pelvic bone, or a nonsurgical peripheral blood stem cell (PBSC) harvest, in which blood is drawn from one arm into a machine, where it is spun and the stem cells are extracted. The remaining blood is then returned to the persons body via the other arm. West chose the latter option, and started receiving Neupogen shots, which increased her bodys production of stem cells. It puts your stem cells on overdrive, she explains.

West says the message from Be the Match was always clear: It was her body and she could change her mind at any point. However, she says, they request that if youre going to change your mind, you do so before you begin the shots. Heres why: At this point, the patient she would be donating to was starting chemotherapy in anticipation of receiving her stem cells. You get the sense that someones life is in the balance and theres no turning back only moving forward, she says. You need to understand the commitment to potentially saving someones life.

West says the shots werent painful, but she did experience some flu-like symptoms. You feel a little achy, she says. Your body is becoming laden with stem cells. Its uncomfortable, but not painful. A nurse came to Wests office to give her the shots, but donors can also go to clinics to get them, or do them on their own. They try to make it as seamless and painless as possible, she says, noting that the shots made her feel moretired than usual. I wasnt on my gym grind that week, she says.

The day of the donation, West reported to a blood donation center at 7 a.m., along with a friend to keep her company, and sat in a chair for about eight hours while her blood was drawn, spun, and returned to her body. When you have to go to the bathroom, you just stop, unhook, go the bathroom, and hook back up again, she says. It wasnt a hardship I cant complain about it. It was eight hours of sitting and talking. West says the process was mildly uncomfortable, but she knew that in about 48 hours, someone was going to have a life-saving operation. Afterwards, she felt tired and went to sleep for a little while. I did it on Friday and was back at work on Monday, but I would have been fine to go to work the next day, she says.

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Stem cells are regenerative, and there is no long-term harm to the donor, Muzzafar Qazilbash, MD professor of stem cell transplantation at the University of Texas MD Anderson Cancer Center explains to Yahoo Beauty.

Photo: Courtesy of Abby West

While there is a need for people of all races and ethnicities to donate bone marrow, there a special need for African-Americans to do so, Qazilbash says. African-Americans make up about 10 percent of the U.S. population, and only about 25 percent to 30 percent of people who could potentially benefit from a bone-marrow transplant have a perfectly matched sibling donor available, he says. The rest of the patients have to find matched, unrelated donors from the National Marrow Donor Program. However, the overwhelming majority of approximately 25 million volunteer donors registered with NMDP are people of Western and Northern European origins, and as a result it is very hard to find matching, unrelated matchingdonors for African-American patients, he says. Encouraging more people of African ancestry will increase the possibility of finding unrelated donors for African-American patients, which can be life saving.

Jack Jacoub, MD, medical oncologist and director of thoracic oncology at MemorialCare Cancer Institute at Orange Coast Memorial Medical Center in Fountain Valley, Calif., tells Yahoo Beauty while some ethnicities have very little genetic variability, there is a lot of genetic variability in the African-American population making the probability of having two genetically similar people less likely. Then you bring up the issue of how few African-American donors there are, and there is difficulty finding an adequate donor, he says.

Donor stem cellscan be used to treat life-threatening conditions such as leukemia and lymphoma, as well as sickle-cell anemia, Jacoub says. They can also help people with bone-marrow failure syndromes such as aplastic anemia a condition in which a persons body stops producing blood cells as well as help treat children born with severe immune system deficiencies, Qazilbash says.

West never received information about the person she donated bone marrow to, other than the fact that the patient was a man. She doesnt know how he fared after the donation, but learned that he struggled with graft-versus-host disease, a complication that can occur after a stem-cell or bone-marrow transplant. Its fairly common for people to have it and move forward, but Ive never found out how he ultimately ended up, she says.

West was so moved by her experience that she eventually joined the board of Be the Match, and now urges others tobecome bone-marrow donors. You always think about what you would want someone to do for you or your family member, she says, noting that shes still on the registry and would donate again if there was a need.

Of course, blood and needles are involved, which can scare some people off, but West says its worth it in the end. In order to do something heroic, you have to overcome some discomfort and some trepidations, she said.

To find out more about becoming a bone-marrow donor, please visit BeTheMatch.org.

Read more: Five Oscars Beauty Launches You Need to Know About Janelle Mone $750 Headband Led the Pack of Stunning Oscars Hair Accessories The $15 Product Behind Taraji P. Hensons Amazing Glow at 2017 Oscars

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Exactly What It's Like to Donate Bone Marrow and Why More ... - Yahoo Health

Patient’s own bone marrow used to treat heart failure – Baltimore Sun (blog)

Johns Hopkins Medicine doctors have treated the first person in a key phase of a clinical trial where a high dose of the patient's own bone marrow cells was used to treat heart failure after a heart attack.

The dose was directed precisely at the point of dysfunction in the heart in the hope that it will stimulate the body's natural healing process.

The Hopkins patient was the first to receive what is called the CardiAMP therapy as part of the third phase of a trial taking place at 40 medical centers across the country. Once phase three trials show a treatment works well, doctors can apply for approval with the Food and Drug Administration.

BioCardia, Inc., headquartered in San Carlos, CA., developed the therapy and The Maryland Stem Cell Research Fund provided research money. The fund was created by the Maryland General Assembly in 2006 to promote state-funded stem cell research and cures through grants and loans to public and private entities.

"Funding the clinical trial of this cell therapy, which could be the first cardiac cell therapy approved in the United States, is an important step towards treatments," Dan Gincel, executive director of the Maryland Stem Cell Research Fund, said in a statement. "Through our clinical program, we are advancing cures and improving health care in the State of Maryland."

Heart disease is the number one killer in the United States. About 610,000 people die from heart disease every year and it accounts for one in every four deaths, according to the Centers for Disease Control and Prevention.

The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.

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Patient's own bone marrow used to treat heart failure - Baltimore Sun (blog)

All eyes on Garrett Richards, in hopes stem cells stave off Tommy John surgery – USA TODAY

With MLB spring training underway, there's plenty to talk about. USA TODAY Sports

Garrett Richards is aiming to pitch through a ligament tear via stem cell therapy and other recovery methods.(Photo: Rick Scuteri, USA TODAY Sports)

TEMPE, Ariz. Garrett Richards first thought when he found out about his torn elbow ligament last May was to schedule Tommy John surgery as soon as possible.

It made sense, considering the ligament-replacement procedure has become the standard fix for such injuries. Plus, the Los Angeles Angels ace was familiar with the operating room, having undergone surgery for a ruptured patellar tendon he sustained on Aug. 20, 2014, toward the end of a breakout season.

Richards knew how to handle the seemingly interminable months of rehab, and he wanted to get the clock started on his return.

But a conversation with Angels head physical therapist Bernard Li convinced Richards to consider other alternatives, and in mid-May he tried a relatively novel treatment in which stem cells taken from bone marrow in his pelvis were injected into the damaged area.

Richards did not pitch again the rest of the year except for a stint in the instructional league, but he has been back on the mound throwing bullpen sessions since the first day of the Angels camp and reported no problems.

This weekend, Richards anticipates pitching in a game for the first time since May 1, when his aching elbow forced him from a start after just four innings.

Its nice to know Ill be able to start the season this year and kind of pick up where I left off, Richards said.

A couple of lockers away, fellow starter Andrew Heaney had a different tale to tell.

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The promising left-hander also went down with a torn ulnar collateral ligament early in the season, after making only one start. Their ailments were the two biggest blows to an Angels rotation that was decimated by injuries, dooming the club to a 74-88 record and a fourth-place finish in the AL West.

Heaney also tried stem cell therapy, two weeks before Richards, both under the supervision of team doctor Steve Yoon. Heaneys ligament didnt heal, though, and after experiencing discomfort throwing following his rehab, he had Tommy John surgery July 1. He has been ruled out for the 2017 season.

They tell you its 50-50. It either works or it doesnt, Heaney said of the stem cell procedure. Obviously, me and Garrett are pretty much the proof of that rule.

Even with less-favorable odds than reconstructive surgery, which has an 80% success rate for returning to action and 67% for pitching 10 games or more, stem cell therapy is gaining acceptance as an option for pitchers with partial UCL tears. The recovery time is shorter 3-5 months instead of 12-18 and the treatment less invasive.

There are limitations. Biological approaches based on stem cells or platelet-rich plasma (PRP) wont repair a complete tear of the ligament. The location of the injury and its extent factor into the chances of success. And players whose ligament doesnt recover, then have to undergo surgery, extend their window of time for returning to action.

Even then, the idea of healing without going under the knife is becoming increasingly appealing. New York Yankees ace Masahiro Tanaka treated the small tear in his elbow ligament with PRP and rehabilitation in 2014, sitting out 10 weeks but coming back to pitch in late September.

Hes 26-11 with a 3.26 ERA over the last two seasons, raising the profile of PRP a procedure in which the players own blood is used to promote healing of the injury as a non-surgical alternative.

Now Richards looms as the test case for stem cell treatment to fix partial UCL tears, which make up about 60-70% of these injuries. If the hard-throwing right-hander can return to his old form he was a Cy Young Award candidate before his knee injury in August 2014 other pitchers in his situation are bound to at least consider the route he took.

I hope this opens another path for guys, Richards said. Obviously, if you can prevent being cut on and having surgery, thats the No. 1 priority. I hope guys dont just jump right into Tommy John, that they at least explore this option.

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Ageless veteran Bartolo Colon was the first pitcher widely known to have undergone stem cell therapy as he sought to recover from elbow and shoulder ailments in 2010. At the time, the ethics of the procedure were questioned, especially because the doctor who performed it, South Florida-based Joseph Purita, acknowledged using human growth hormone in previous treatments, though not in Colons.

Since then, the use of stem cells has become more mainstream. They are the focus of Yoons practice.

As more and more people start to use it, youre getting a better sense for what it can and cant do, Yoon said. Baseball definitely has opened up to it quite a bit, and as we see some of the successes like with Garrett, were getting a better understanding that theres a lot of potential here with these types of treatment.

Yoon calls stem cell therapy a super PRP because it combines the curative properties of that treatment with more healing agents, and said it can be used on tendon tears, muscle tears and strains and even to address degenerative joint disease.

However, much remains unknown about the benefits of stem cells. Lyle Cain, an orthopedist who has performed both Tommy John surgeries and stem cell treatments at the Andrews Sports Medicine & Orthopaedic Center in Birmingham, Ala., said most of the research has been anecdotal, not scientific.

We still dont have a good understanding even four or five years into it exactly what the stem cells do, what their method is, Cain said. The theory is theres probably a chemical reaction where it releases chemicals in the cell that help the healing process. The stem cells arent necessarily put in there with the thought theyre going to become ligament, but theres probably a cellular chemical mechanism that helps the healing response.

And as Heaney discovered, theyre not always effective. His tear was located farther down the arm, which reduced his chances of success with stem cell therapy. Richards was a better candidate because his injury, though deemed high grade, was located within the ligament, like a slit on a rubber band.

But because Heaney was looking at likely missing most or all of 2017 even if he had surgery right away, he decided to try stem cells. The timing of the injury plays a major role in whether pitchers contemplate alternatives to surgery, with the more conservative approach often recommended if it happens early in the season.

Heaney said he doesnt regret taking that route, and would have been upset if he had undergone the ligament-replacement operation right away, only to find out he could have returned to action quicker through another means.

Im glad it worked for him, he said of Richards. It would have been really awful if it hadnt worked for either of us. Then wed both look like idiots.

Their peers are paying attention. In a major league pitching community where about a quarter of its members have undergone Tommy John surgery, interest in the effectiveness of alternative cures is high.

The Los Angeles Dodgers Brandon McCarthy was not a candidate because his ligament tore clear off the bone, but said he had heard positive reports about stem cell treatment, not so much about PRP.

The Pittsburgh Pirates Daniel Hudson, a veteran of two Tommy Johns, is encouraged as well.

Its supposed to help repair the tissue. Before, ligaments just wont repair themselves, Hudson said. It might keep a lot of guys from going under the knife.

Thats Cains hope. He regularly treats UCL tears on high school, college and minor-league players with stem cells or PRP, but realizes theres heightened pressure on major leaguers to return to the field.

If more of them can do it without visiting an operating room, it would represent a major advancement for both the players and the industry.

I think overall the biologic treatment of these injuries will certainly progress and it will be somewhat the wave of the future, Cain said. There will be certain ligaments that are damaged enough that we dont have an answer; they have to reconstruct. But I think overall, if you look 15 years down the road, I suspect well be doing a lot more non-surgical treatment than surgical treatment.

Contributing: Gabe Lacques in Bradenton, Fla.

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All eyes on Garrett Richards, in hopes stem cells stave off Tommy John surgery - USA TODAY

Super resolution imaging helps determine a stem cell’s future – Phys.Org

February 27, 2017 Human mesenchymal stem cells were labeled for two epigenetic marks (green and red), and the images were analyzed to forecast the cell developmental fate. Credit: Joseph J. Kim

Scientists at Rutgers and other universities have created a new way to identify the state and fate of stem cells earlier than previously possible.

Understanding a stem cell's fatethe type of cell it will eventually becomeand how far along it is in the process of development can help scientists better manipulate cells for stem cell therapy.

The beauty of the method is its simplicity and versatility, said Prabhas V. Moghe, distinguished professor of biomedical engineering and chemical and biochemical engineering at Rutgers and senior author of a study published recently in the journal Scientific Reports. "It will usher in the next wave of studies and findings," he added.

Existing approaches to assess the states of stem cells look at the overall population of cells but aren't specific enough to identify individual cells' fates. But when implanting stem cells (during a bone marrow transplant following cancer treatment, for example), knowing that each cell will become the desired cell type is essential. Furthermore, many protein markers used to distinguish cell types don't show up until after the cell has transitioned, which can be too late for some applications.

To identify earlier signals of a stem cell's fate, an interdisciplinary team from multiple universities collaborated to use super-resolution microscopy to analyze epigenetic modifications. Epigenetic modifications change how DNA is wrapped up within the nucleus, allowing different genes to be expressed. Some modifications signal that a stem cell is transitioning into a particular type of cell, such as a blood, bone or fat cell. Using the new method, the team of scientists was able to determine a cell's fate days before other techniques.

"Having the ability to visualize a stem cell's future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases," says Rosemarie Hunziker, program director for Tissue Engineering and Regenerative Medicine at the National Institute of Biomedical Imaging and Bioengineering. "It's a relatively simple way to get a jump on determining the right cells to use."

The approach, called EDICTS (Epi-mark Descriptor Imaging of Cell Transitional States), involves labeling epigenetic modifications and then imaging the cells with super resolution to see the precise location of the marks.

"We're able to demarcate and catch changes in these cells that are actually not distinguished by established techniques such as mass spectrometry," Moghe said. He described the method as "fingerprinting the guts of the cell," and the results are quantifiable descriptors of each cell's organization (for example, how particular modifications are distributed throughout the nuclei).

The team demonstrated the method's capabilities by measuring two types of epigenetic modifications in the nuclei of human stem cells cultured in a dish. They added chemicals that coaxed some of the cells to become fat cells and others to become bone, while another set served as control. Within three days, the localization of the modifications varied in cells destined for different fates, two to four days before traditional methods could identify such differences between the cells. The technique had the specificity to look at regional changes within individual cells, while existing techniques can only measure total levels of modifications among the entire population of cells.

"The levels are not significantly different, but how they're organized is different and that seems to correlate with the fact that these cells are actually exhibiting different fates," Moghe said. "It allows us to take out a single cell from a population of dissimilar cells," which can help researchers select particular cells for different stem cell applications.

The method is as easy as labeling, staining and imaging cells - techniques already familiar to many researchers, he said. As the microscopes capable of super resolution imaging become more widely available, scientists can use it to sort and screen different types of cells, understand how a particular drug may disrupt epigenetic signaling, or ensure that stem cells to be implanted won't transform into the wrong cell type.

Explore further: Super-resolution imaging can map critical cell changes several days sooner than current method

More information: Joseph J. Kim et al, Optical High Content Nanoscopy of Epigenetic Marks Decodes Phenotypic Divergence in Stem Cells, Scientific Reports (2017). DOI: 10.1038/srep39406

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Super resolution imaging helps determine a stem cell's future - Phys.Org

Vital need for minority bone barrow donors – Story – KTAL

Shreveport, La. - Jasmine Sewell was diagnosed with bone caner in February of 2014. She and her fiance, Marcus Price, had planned their wedding for September of 2017 but moved up the date after Jasmine was given a prognosis in 2016 of less than a year to live.

Jasmine and Marcus were married February 11, 2017 and many of their guests gave a present to Jasmine by registering as bone marrow donors.

Jasmine has stopped responding to treatments and now needs a bone marrow or stem cell transplant to survive.

Be the Match signs people up to be bone marrow and stem cell donors but there is a shortage of minority donors. The match between the donor and recipient is largely based on genetics an ancestry so the likelihood of a non minority donor being able to help Jasmine is very slim.

The sign up is easy, just a cheek swab, and the donation of stem cells is a process similar to giving blood. Infant recipients can need actual bone marrow which is taken from the bone with a needle but the patient is sedated and most people only feel a bit sore after the procedure.

Jasmine is asking anyone not registered to sign up, and act as if it were their loved ones that needed the transplant.

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Vital need for minority bone barrow donors - Story - KTAL

New antiviral drug cuts cytomegalovirus infection, improves survival in patients undergoing donor stem cell transplant – Science Daily

In a significant advance in improving the safety of donor stem cell transplants, a major clinical trial led by researchers at Dana-Farber Cancer Institute and Brigham and Women's Hospital (BWH) has shown that a novel agent can protect against the most common viral infection that patients face after transplantation.

The results represent a breakthrough in a decade-long effort to identify an effective drug for the prevention of CMV infection in transplant patients that doesn't produce side effects that negate the benefit of the drug itself, the study authors said.

The findings, from an international phase 3 clinical trial of the drug letermovir for preventing cytomegalovirus (CMV) infection in transplant patients, will be presented at the 2017 Bone Marrow Transplant Tandem Meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood and Marrow Transplant Research (CIBMTR) in Orlando, Florida, February 22, 2017.

The study, which involved 565 adult patients at 67 research centers in 20 countries, compared letermovir to placebo in preventing an active CMV infection following transplant with donor stem cells. The patients, who were undergoing transplant as treatment for blood-related cancers or other disorders, all carried a CMV infection from earlier in life that had been wrestled into dormancy by their immune system. Twenty-four weeks after completing up to 14 weeks of treatment, 61 percent of the patients receiving a placebo had developed a CMV infection serious enough to require treatment or had discontinued the trial. By contrast, only 38 percent of those treated with letermovir developed that level of CMV infection or did not complete the trial.

Unlike other drugs able to forestall active CMV infection in stem cell transplant patients, letermovir did so without producing unacceptable toxicities. Most of the side effects associated with letermovir were tolerable, including mild cases of nausea or vomiting, and some swelling, investigators found. Letermovir also conferred a survival benefit: at the 24-week mark, 15 percent of the placebo patients had died, compared to 10 percent of those receiving letermovir.

"For the first time, we seem to have a drug that is a true safe and effective preventive for CMV infection in stem cell transplant patients," said the study's lead author, Francisco Marty, MD, an infectious disease specialist at Dana-Farber and BWH. "Letermovir will allow many patients to avoid infection, usually with no or mild side effects, and seems to provide a survival benefit in the first six months post-transplant."

Transplantation of donor hematopoietic stem cells -- which give rise to all types of blood cells, including white blood cells of the immune system -- is used to treat blood-related cancers such as leukemia, lymphoma, and myeloma, as well as several types of non-cancerous blood disorders. Patients typically receive chemotherapy to wipe out or reduce the bone marrow, where blood cells are formed, followed by an infusion of donor stem cells to rebuild their blood supply and reconstitute their immune system.

While refinements in transplant techniques have sharply improved the safety of the procedure, the reactivation of CMV infection following a transplant has been a longstanding problem.

Infection with CMV, a type of herpes virus, is one of the most common viral infections in the world. In the United States, it's estimated that over 50 percent of people are infected before adulthood. In other parts of the world, infection rates can be significantly higher. The effects of CMV infection can range from no symptoms to a flu-like fever or mononucleosis ("mono") syndrome. Once the immune system has brought the infection under control, the virus persists unobtrusively in the body.

The jolt of a stem cell transplant -- the rapid erasure or diminishment of the immune system produced by pre-transplant chemotherapy, as well as measures to prevent graft-versus-host disease -- can give CMV a chance to reawaken and run amok before the newly reconstituted immune system takes hold. In the early years of bone marrow transplant therapy, 60 to 70 percent of transplant recipients developed CMV infection, Marty recounts. Of those, 20 to 30 percent contracted CMV pneumonia, and of those, 80 percent died of the disease.

In previous clinical trials, several drugs aimed at preventing CMV infection in stem cell transplant patients either were not effective or produced intolerable side effects. In the absence of safe preventive drugs, physicians worked out a "surveillance" approach in which they provide treatment only when patients develop CMV infection, and only for a short period of time. This strategy has largely been a success: patients now have just a 2 or 3 percent chance of getting CMV disease affecting the lungs or other organs. Still, the often harsh side effects of current drugs were reason to continue the search for a useful preventive agent.

Letermovir works by a different mechanism from previously tested agents, which block an enzyme known as DNA polymerase, which viruses use to duplicate their DNA. (Human cells use the same process to replicate their own DNA.) By contrast, letermovir blocks a process by which CMV is "packaged" inside infected cells -- a wrapping that allows it to go on and infect other cells. The fact that this process does not occur in human cells may explain in part why letermovir usually gives rise to only mild side effects, researchers say.

In the trial, patients received letermovir or a placebo beginning an average of nine days after transplant. "The goal was to suppress the virus before it has a chance to become active," Marty remarked. "The results of this trial offer encouragement that letermovir can offer a new strategy for donor stem cell transplant patients in preventing the emergence of CMV infection following transplant."

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New antiviral drug cuts cytomegalovirus infection, improves survival in patients undergoing donor stem cell transplant - Science Daily

Stem Cells Saved His Life. Two Years Later, He Met The Donor – NDTV

Kolkata: Joy knew no bounds for 42-year-old Nilesh Sinha, when he hugged his saviour, 27-year-old Sajat Jain. Mr Sinhacalls it the warmest and most meaningful hug of his life.

Two years ago, Mr Sinha was suffering fromAplastic Anemia, a rare disease in which the bone marrow and the hematopoietic stem cells that reside there are damaged. Only a stem cell transplant could have saved himfrom his deteriorating condition and Mr Jains were a perfect match.

The peripheral blood stem cell or PBSC transplant took place at Kolkatas Tata Memorial Centre in 2015. Last week, the two came face-to-face for the first time at an event organised by Datri, Indias largestadult unrelated blood stem cell donors registry, which aided the transplant between the two.

Mr Sinha said he was ecstatic to find the opportunity to say thanks to the man he owes his life to. Sajat is my childrens new superhero, he said.Mr Jain toocould not believe that his simple act saved someones life.

Mr Sinha told NDTV that he is sure that once people get to know about Mr Jain, they will also come forward to register themselves as stem cell donors and after them, the next generation will also get motivated.

Mr Jain, who runs a healthcare start-up, said that he became a donor while researching for his company and could save Mr Sinhas life just in time, after a donor backed out. He wants more young people to register so that someone in need can be benefited.

I was actually pretty excited. I know I was able to save someones life and not too many people can say that in their lifetime. When I actually saw his face, I remembered his previous condition and was delighted to see how fit he had become, Mr Jain told NDTV.

Kolkata has seen 300 successful stem cell transplants so far. Director of Tata Memorial Centre, Kolkata, Dr Mammen Chandy told NDTV that there is a need for more donors in India. When a donor asks me what is the risk of a donation, I would say, what is the risk of crossing the street outside my hospital and not being hit by a bus? he said.

In 2009, Datri came to the aid of people suffering from life threatening blood disorders like leukaemia, lymphoma, severe aplastic anemia, sickle cell disease, thalassemia among others. It started with 3,000 people pledging to donate stem cells and today there are 93,000 registered donors with them. With this small number of registered donors, however, the possibility of finding a match for an Indian anywhere in the world is very bleak.

Blood stem cells from a donor can give someone a second chance at life and a patient has 25 per cent chance of finding a match within the family. There are three sources of blood-forming cells used in transplants: bone marrow, peripheral blood stem cell or PBSC and umbilical cord blood collected after a baby is born.

The peripheral blood stem cell donation is a painless, non-surgical, outpatient procedure that involves only a needle in the arm vein, similar to platelet donation. However, if a marrow is requested, then it is a surgical procedure.

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Stem Cells Saved His Life. Two Years Later, He Met The Donor - NDTV

Study shows adipose stem cells may be the cell of choice for therapeutic applications – Medical Xpress

February 24, 2017

An international team of researchers, funded by Morris Animal Foundation, has shown that adipose (fat) stem cells might be the preferred stem cell type for use in canine therapeutic applications, including orthopedic diseases and injury.

Researchers at the University of Guelph, University of Western Ontario and Aarhus University, Denmark, ran a battery of tests comparing the physiology characteristics of stem cells derived from adipose tissue versus bone marrow. They found that stem cells from both sources had similar functional properties, including tissue generation and immunomodulating capabilities (ability to adjust immune response), but adipose stem cells grow at a faster rate than bone marrow stem cells. Harvesting adipose stem cells also is less invasive than harvesting bone marrow. The study recently was published in PLoS ONE, an online scientific journal.

In the last decade, the use of stem cell therapy in animals and humans has dramatically increased. In dogs, stem cell therapy is used in the treatment of a variety of orthopedic diseases and injuries. Stem cells are harvested from either fat tissue or bone marrow, purified and grown in culture, then placed back in the patient.

Given the ease of harvesting, adipose tissue has become the site of most stem cell collections in canine patients. But questions persisted regarding the differences between these two sources of stem cells, and which is better suited to therapeutic applications.

"Faster proliferation along with the potential for a less invasive method of their procurement makes them (adipose stem cells) the preferred source for canine mesenchymal stem cells," concluded the research team.

Explore further: Stem cell therapy trial at Sanford first of its kind in US for shoulder injuries

More information: Keith A. Russell et al, Characterization and Immunomodulatory Effects of Canine Adipose Tissue- and Bone Marrow-Derived Mesenchymal Stromal Cells, PLOS ONE (2016). DOI: 10.1371/journal.pone.0167442

Journal reference: PLoS ONE

Provided by: Morris Animal Foundation

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Study shows adipose stem cells may be the cell of choice for therapeutic applications - Medical Xpress

The US is wrong to ban pay for bone-marrow donors – Standard-Examiner

(c) 2017, Bloomberg View.

Two years ago, Doreen Flynn of Lewiston, Maine, won her case against the U.S. government, successfully arguing that bone-marrow donors should be able to receive compensation.

Flynn, a mother of three girls who are afflicted with a rare, hereditary blood disease called Fanconis anemia, has a strong interest in bone-marrow transplantation. At the time of the court ruling, her oldest daughter, Jordan, 14, had already received a transplant, and one of the younger twins, Jorja, was expected to need one in a few years.

Locating a marrow donor is often a needle-in-a-haystack affair. The odds that two random individuals will have the same tissue type are less than 1 in 10,000, and the chances are much lower for blacks. Among the precious few potential donors who are matched, nearly half dont follow through with the actual donation. Too often, patients dont survive the time it takes to hunt for another donor.

Allowing compensation for donations could enlarge the pool of potential donors and increase the likelihood that compatible donors will follow through. So the ruling by a three-judge panel of the U.S. Court of Appeals for the Ninth Circuit was promising news for the 12,000 people with cancer and blood diseases currently looking for a marrow donor. (James F. Childress, an ethicist at the University of Virginia, and I submitted an amicus brief in the case.)

Soon after the verdict, Shaka Mitchell, a lawyer in Nashville, Tennessee, and co-founder of the nonprofit MoreMarrowDonors.org, began collecting funds to underwrite $3,000 donor benefits, which were to be given as scholarships, housing allowances or gifts to charity.

Mitchell also invited a team of economists to evaluate the effects of the ruling on peoples willingness to join a registry and to donate when they are found to be a match. The researchers were to specifically assess whether cash payments would be any more or less persuasive than noncash rewards or charitable donations.

Now comes the bad news. On Oct. 2, the U.S. Department of Health and Human Services proposed a new rule that would overturn the Ninth Circuits decision. The government proposes designating a specific form of bone marrow -- circulating bone-marrow stem cells derived from blood -- as a kind of donation that, under the 1984 National Organ Transplant Act, cannot be compensated. If this rule goes into effect (the public comment period ends today), anyone who pays another person for donating these cells would be subject to as much as five years in prison and a $50,000 fine.

The problem with this rule is that donating bone marrow is not like donating an essential organ. Indeed, the Ninth Circuit based its decision on the fact that modern bone-marrow procurement, a process known as apheresis, is more akin to drawing blood. In the early 1980s, when the transplant act was written, the process was more demanding, involving anesthesia and the use of large, hollow needles to extract marrow from a donors hip. But today, more than two-thirds of marrow donations are done via apheresis. Blood is taken from a donors arm, the bone-marrow stem cells are filtered out, and the blood is then returned to the donor through a needle in the other arm.

The Ninth Circuit panel held that these filtered stem cells are merely components of blood -- no different from blood-derived plasma, platelets and clotting factors, for which donor compensation is allowed.

The strongest opposition to compensation comes from the National Marrow Donor Program, the Minneapolis-based nonprofit that maintains the nations largest donor registry. Michael Boo, the programs chief strategy officer, says of reimbursement, Is that what we want people to be motivated by?

The problem with this logic is that altruism has proven insufficient to motivate enough people to give marrow and, as a result, people die.

HHS is presumably under pressure from the National Marrow Donor Program. The department does not otherwise explain its proposed rule except to claim that compensation runs afoul of the transplant acts intent to ban commodification of human stem cells and to curb opportunities for coercion and exploitation, encourage altruistic donation and decrease the likelihood of disease transmission.

But how could such concerns plausibly apply to marrow stem cells and not to blood plasma? The process of collecting plasma is safe: No serious infection has been transmitted in plasma-derived products in nearly two decades, according to the Plasma Protein Therapeutics Association. Strenuous screening and testing in a robust regulatory environment, coupled with voluntary industry standards and sophisticated manufacturing processes, have created what has been called the safest blood product available today.

Outlawing compensation for stem blood cells but not mature blood cells might even violate the constitutional guarantee of equal protection of the law, according to Jeff Rowes, a lawyer at the Institute for Justice, which represented Flynn.

HHS should withdraw its proposal. Ideally, Congress should thwart future regulatory mischief by amending the National Organ Transplant Act to stipulate that marrow stem cells are not organs.

Each year, 2,000 to 3,000 Americans in need of marrow transplants die waiting for a match. Altruism is a virtue, but clearly it is not a dependable motive for marrow donation.

---

Satel, a psychiatrist and a resident scholar at the American Enterprise Institute, is a co-author of Brainwashed: The Seductive Appeal of Mindless Neuroscience. To contact the editor responsible for this story: Mary Duenwald at mduenwald@bloomberg.net.

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Keywords: SATEL-OP-ED-MARROWDONORS

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The US is wrong to ban pay for bone-marrow donors - Standard-Examiner

New antiviral drug cuts cytomegalovirus infection and improves survival in patients – ScienceBlog.com (blog)

Orlando- In a significant advance in improving the safety of donor stem cell transplants, a major clinical trial led by researchers at Dana-Farber Cancer Institute and Brigham and Womens Hospital (BWH) has shown that a novel agent can protect against the most common viral infection that patients face after transplantation.

The results represent a breakthrough in a decade-long effort to identify an effective drug for the prevention of CMV infection in transplant patients that doesnt produce side effects that negate the benefit of the drug itself, the study authors said.

The findings, from an international phase 3 clinical trial of the drug letermovir for preventing cytomegalovirus (CMV) infection in transplant patients, will be presented at the 2017 Bone Marrow Transplant Tandem Meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood and Marrow Transplant Research (CIBMTR) in Orlando, Florida, February 22, 2017.

The study, which involved 565 adult patients at 67 research centers in 20 countries, compared letermovir to placebo in preventing an active CMV infection following transplant with donor stem cells. The patients, who were undergoing transplant as treatment for blood-related cancers or other disorders, all carried a CMV infection from earlier in life that had been wrestled into dormancy by their immune system. Twenty-four weeks after completing up to 14 weeks of treatment, 61 percent of the patients receiving a placebo had developed a CMV infection serious enough to require treatment or had discontinued the trial. By contrast, only 38 percent of those treated with letermovir developed that level of CMV infection or did not complete the trial.

Unlike other drugs able to forestall active CMV infection in stem cell transplant patients, letermovir did so without producing unacceptable toxicities. Most of the side effects associated with letermovir were tolerable, including mild cases of nausea or vomiting, and some swelling, investigators found. Letermovir also conferred a survival benefit: at the 24-week mark, 15 percent of the placebo patients had died, compared to 10 percent of those receiving letermovir.

For the first time, we seem to have a drug that is a true safe and effective preventive for CMV infection in stem cell transplant patients, said the studys lead author, Francisco Marty, MD, an infectious disease specialist at Dana-Farber and BWH. Letermovir will allow many patients to avoid infection, usually with no or mild side effects, and seems to provide a survival benefit in the first six months post-transplant.

Transplantation of donor hematopoietic stem cells which give rise to all types of blood cells, including white blood cells of the immune system is used to treat blood-related cancers such as leukemia, lymphoma, and myeloma, as well as several types of non-cancerous blood disorders. Patients typically receive chemotherapy to wipe out or reduce the bone marrow, where blood cells are formed, followed by an infusion of donor stem cells to rebuild their blood supply and reconstitute their immune system.

While refinements in transplant techniques have sharply improved the safety of the procedure, the reactivation of CMV infection following a transplant has been a longstanding problem.

Infection with CMV, a type of herpes virus, is one of the most common viral infections in the world. In the United States, its estimated that over 50 percent of people are infected before adulthood. In other parts of the world, infection rates can be significantly higher. The effects of CMV infection can range from no symptoms to a flu-like fever or mononucleosis (mono) syndrome. Once the immune system has brought the infection under control, the virus persists unobtrusively in the body.

The jolt of a stem cell transplant the rapid erasure or diminishment of the immune system produced by pre-transplant chemotherapy, as well as measures to prevent graft-versus-host disease can give CMV a chance to reawaken and run amok before the newly reconstituted immune system takes hold. In the early years of bone marrow transplant therapy, 60 to 70 percent of transplant recipients developed CMV infection, Marty recounts. Of those, 20 to 30 percent contracted CMV pneumonia, and of those, 80 percent died of the disease.

In previous clinical trials, several drugs aimed at preventing CMV infection in stem cell transplant patients either were not effective or produced intolerable side effects. In the absence of safe preventive drugs, physicians worked out a surveillance approach in which they provide treatment only when patients develop CMV infection, and only for a short period of time. This strategy has largely been a success: patients now have just a 2 or 3 percent chance of getting CMV disease affecting the lungs or other organs. Still, the often harsh side effects of current drugs were reason to continue the search for a useful preventive agent.

Letermovir works by a different mechanism from previously tested agents, which block an enzyme known as DNA polymerase, which viruses use to duplicate their DNA. (Human cells use the same process to replicate their own DNA.) By contrast, letermovir blocks a process by which CMV is packaged inside infected cells a wrapping that allows it to go on and infect other cells. The fact that this process does not occur in human cells may explain in part why letermovir usually gives rise to only mild side effects, researchers say.

In the trial, patients received letermovir or a placebo beginning an average of nine days after transplant. The goal was to suppress the virus before it has a chance to become active, Marty remarked. The results of this trial offer encouragement that letermovir can offer a new strategy for donor stem cell transplant patients in preventing the emergence of CMV infection following transplant.

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New antiviral drug cuts cytomegalovirus infection and improves survival in patients - ScienceBlog.com (blog)

Adoring gran who will die without bone marrow transplant desperate to see granddaughter go to school – Mirror.co.uk

As a devoted gran, Sipy Howard looks forward to watching her grandkids grow up.

In two years' time, the 65-year-old hopes to proudly look on as her youngest granddaughter, Sienna, goes off to school .

But devastatingly, she doesn't know if she'll be able to do so - because she urgently needs a bone marrow transplant to survive.

Sipy, a "warm and adoring" gran, was diagnosed with leukaemia on December 15 last year - which also happened to be her birthday.

Although she has been undergoing intense chemotherapy, she is in need of a bone marrow and stem cell transplant to save her life.

Now, her daughters, Emma, 34, Jolene, 32, and Sammy, 26, have launched a desperate online campaign to find a donor for her.

The heart-wrenching campaign, dubbed #SavingSipy , aims to encourage potential donors to sign up to the bone marrow register.

It stresses how simple it is to register as a donor online.

Sipy, married to husband Eli, told Mirror Online she is "moved" by the support she has received from her loved ones - as well as strangers.

"We really need as many people as possible to sign up to the register," said the mum of three, from Kenton, northwest London.

She added: "I want to see my grandchildren growing up and my youngest granddaughter going to school."

Sipy was devastated to be diagnosed with Acute Myeloid Leukemia, an aggressive blood cancer, on her 65th birthday.

Her daughters were also shocked by the diagnosis.

Emma said: "You can never prepare yourself for hearing that a loved one has cancer.

"You are suddenly filled with fear and a desperation you have never quite experienced.

"I felt like I had been hit by a car when my dad broke the news to me on my mum's birthday.

"Keeping yourself emotionally together each day becomes your only goal."

She added that her mum is currently "struggling through" her second, gruelling round of chemotherapy.

"It's the knowing that she needs us more than ever that keeps us going, keeps us fighting," she said.

"We won't give up on her and our goal of finding a match for her."

Sipy, described as "bubbly, always laughing and generally a bit nuts", is a gran to two-year-old Sienna and four-year-old Sofia.

A member of the Jewish community, she has an "open house policy" and "cares for everyone she meets", according to her family.

Her daughters are hoping that the community - specifically the Sephardi community - could help to save her life.

Sammy said: "One of the hardest things about the situation is knowing there may be someone in the world who is a match for my mum, but because they have not registered to be on the bone marrow register, they can't save her.

"This is why we are desperately trying to reach out to everyone we can and get our message across.

"We urgently need people to register now and unfortunately we don't have much time."

Jolene added: "Awareness is key."

The campaign is being supported by the charity DKMS, which aims to help blood cancer patients find matching donors.

For details of how to register as a donor online, you can visit the #SavingSipy Facebook page here or DKMS's website here .

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Adoring gran who will die without bone marrow transplant desperate to see granddaughter go to school - Mirror.co.uk

Steenblock Research Institute Announces New Method of Obtaining … – PRUnderground (press release)

The treatment of chronic diseases and medical conditions using a patients own stem cells is an exciting, dynamic area of medicine that is enjoying unprecedented success thanks to a combination of new discoveries and impressive patient responses. Here in the US, over 600 private clinics are doing stem cell therapies, most using stem cells taken from a patients own bone marrow or fat tissue.

Unfortunately, many ailing people are reluctant or unable to undergo the harvesting of bone marrow or fat tissue. With this in mind, physician David Steenblock, president of the nonprofit Steenblock Research Institute (SRI), asked the scientists at his research institute to come up with a way of getting enough stem cells from a patients blood sample to treat their health issues.

The scientists at SRI went on to develop an FDA compliant method for winding up with as many stem cells in 2 small tubes of blood (drawn from a patient) as are normally obtained from much larger quantities of bone marrow or fat tissue. The blood is exposed to heat, cold, light and electromagnetics which yields 100 million stem cells, an amount which is ten times the minimum of 10 million cells needed to produce a clinical response.

SRIs new method has beenauthorized for use byonly one clinic: Personalized Regenerative Medicine Clinic of San Clemente, California.

Adds Dr. Steenblock, Stem Cell Therapy is experimental and thus is not covered by any insurance plan. This new method is also only available to patients of Personalized Regenerative Medicine Clinic.

Those interested in learning more are invited to call Personalized Regenerative Medicine Clinic (PRMC) at 1-949-367-8870 from 9 am to 4 pm Pacific Time, Monday through Friday. Individuals who enroll as new patients are being offered an initial office visit for free with PRMCs Dr. Donna Hanna.

About David A. Steenblock, D.O., Inc.

Dr. David Steenblock and his Personalized Regenerative Medicine Clinic are both committed to and passionate about advancing medicine. They do this, in part, by creating customized treatment regimens for patients which often includes the use of stem cells and FDA approved stem cell mobilizers and activators. In addition, Dr. Steenblock brings to the table finely honed diagnostic skills as well as a wealth of knowledge, experience, and insight plus the courage to explore, experiment and innovate in a private clinical setting.

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Steenblock Research Institute Announces New Method of Obtaining ... - PRUnderground (press release)

Still Searching For A StemCell/Bone Marrow Donor For Baby Madalayna – windsoriteDOTca News

The search is still on to find a matching stem cell/bone marrow donor for five month old Madalayna Ducharme. The initial search of the Canadian OneMatch Stem Cell and Marrow Network as well as Bone Marrow Donors Worldwide (BMDW) have not yet identified a matching donor for Madalayna.

Madalayna was recently diagnosd with malignant infantile osteopetrosis, a genetic disorder that prevents her bones from working properly and they become too dense. This disorder attacks vision, hearing and is life threatening.

On Friday, February 24th there is another opportunity to help Madalayna find her match. The Katelyn Bedard Bone Marrow Association will host a Get Swabbed! registration event at Holy Names High School 1400 Northwood Street from 10am to 1:30pm.

The general public is invited to join the students and staff at Holy Names High School to keep the groundswell surging in an effort to ultimately find that life saving match for Madalayna.

The age requirement to register as a Stem Cell donor is 17 to 35 years. The registrant must possess a Canadian Government issued health card.

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Still Searching For A StemCell/Bone Marrow Donor For Baby Madalayna - windsoriteDOTca News

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