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.

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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

Bone Marrow Stem Cells Comments Off on Gene therapy shows early promise against sickle cell – Chicago Tribune | March 3rd, 2017

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

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

Bone Marrow Stem Cells Comments Off on This simple method can predict a stem cell’s fate – Futurity: Research News | March 3rd, 2017

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)

Bone Marrow Stem Cells Comments Off on Family appeals for bone marrow donor to save father – Easterneye (press release) (subscription) | March 3rd, 2017

Science Times

Suicide Switch for Transplanted Stem Cells The Scientist The team then differentiated the iPSCs into neural stem and progenitor cells, and transplanted them into mice with a spinal cord injury. The mice began to recover some motor function, but as neural tumors and teratomas grew from the transplanted cells ... Stem Cell Assay Market, 2016-2024: By Type, Applications ...QWTJ LIVE One Stop all 17 news articles »

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Suicide Switch for Transplanted Stem Cells - The Scientist

Spinal Cord Stem Cells Comments Off on Suicide Switch for Transplanted Stem Cells – The Scientist | March 2nd, 2017

Notes This is a 7-day-old embryo that scientists kept alive in a laboratory dish. If it developed further, the clusters in green would become cells that shape the body and the red/purple cells would form the placenta.

Ali Brivanlou slides open a glass door at the Rockefeller University in New York to show off his latest experiments probing the mysteries of the human embryo.

"As you can see, all my lab is glass just to make sure there is nothing that happens in some dark rooms that gives people some weird ideas," says Brivanlou, perhaps only half joking.

Brivanlou knows that some of his research makes some people uncomfortable. That's one reason he has agreed to give me a look at what's going on.

His lab and one other discovered how to keep human embryos alive in lab dishes longer than ever before at least 14 days. That has triggered an international debate about a long-standing convention (one that's legally binding in some countries, though not in the U.S.) that prohibits studying human embryos that have developed beyond the two-week stage.

Ali Brivanlou's research team at Rockefeller University in New York was one of two groups internationally that figured out how to keep human embryos alive in lab dishes beyond the 14-day stage of development. Rob Stein/NPR hide caption

And in other experiments, he's using human stem cells to create entities that resemble certain aspects of primitive embryos. Though Brivanlou doesn't think these "embryoids" would be capable of developing into fully formed embryos, their creation has stirred debate about whether embryoids should be subject to the 14-day rule.

Brivanlou says he welcomes these debates. But he hopes society can reach a consensus to permit his work to continue, so he can answer some of humanity's most fundamental questions.

"If I can provide a glimpse of, 'Where did we come from? What happened to us, for us to get here?' I think that, to me, is a strong enough rationale to continue pushing this," he says.

For decades, scientists thought the longest an embryo could survive outside the womb was only about a week. But Brivanlou's lab, and one in Britain, announced last year in the journals Nature and Nature Cell Biology that they had kept human embryos alive for two weeks for the first time.

That enabled the scientists to study living human embryos at a crucial point in their development, a time when they're usually hidden in a woman's womb.

"Women don't even know they are pregnant at that stage. So it has always been a big black box," Brivanlou says.

Gist Croft, a stem cell biologist in Brivanlou's lab, shows me some samples, starting with one that's 12 days old.

"So you can see this with the naked eye," Croft says, pointing to a dish. "In the middle of this well, if you look down, there's a little white speck it looks like a grain of sand or a piece of dust."

Under a microscope, the embryo looks like a fragile ball of overlapping bubbles shimmering in a silvery light with thin hairlike structures extending from all sides.

Croft and Brivanlou explain that those willowy structures are what embryos would normally extend at this stage to search for a place to implant inside the uterus. Scientists used to think embryos could do that only if they were receiving instructions from the mother's body.

"The amazing thing is that it's doing its thing without any information from mom," Brivanlou says. "It just has all the information already in it. That was mind-blowing to me."

The embryos they managed to keep alive in the lab dish beyond seven days of development have also started secreting hormones and organizing themselves to form the cells needed to create all the tissues and organs in the human body.

The two scientists think studying embryos at this and later stages could lead to discoveries that might point to new ways to stop miscarriages, treat infertility and prevent birth defects.

"The only way to understand what goes wrong is to understand what happens normally, or as normally as we can, so we can prevent all of this," Brivanlou says.

The 14-day cutoff

But Brivanlou isn't keeping these embryos alive longer than 14 days because of the rule.

A long-standing rule prohibits scientists from keeping human embryos alive more than two weeks, after which the central nervous system starts to develop. The 14-day rule was developed decades ago to avoid raising too many ethical questions about experimenting on human embryos. It's a law in some countries, and just a guideline in the U.S.

"The decision about pulling the plug was probably the toughest decision I've made in my scientific career," he says. "It was sad for me."

The 14-day rule was developed decades ago to avoid raising too many ethical questions about experimenting on human embryos.

Two weeks is usually the moment when the central nervous system starts to appear in the embryo in a structure known as the "primitive streak."

It's also roughly the stage at which an embryo can no longer split into twins. The idea behind the rule is, that's when an embryo becomes a unique individual.

But the rule was initiated when no one thought it would ever be possible to keep embryos growing in a lab beyond two weeks. Brivanlou thinks it's time to rethink the 14-day rule.

"This is the moment," he says.

Scientists, bioethicists and others are debating the issue in the U.S., Britain and other countries. The rule is law in Britain and other countries and incorporated into widely followed guidelines in the United States.

Insoo Hyun, a bioethicist at Case Western Reserve University, advocates revisiting the rule. It would allow more research to be done on embryos that are destined to be destroyed anyway, he says embryos donated by couples who have finished infertility treatment.

"Given that it has to be destroyed," Hyun says, "some would argue that it's best to get as much information as possible scientifically from it before you destroy it."

But others find it morally repugnant to use human embryos for research at any stage of their development and argue that lifting the 14-day rule would make matters worse.

"Pushing it beyond 14 days only aggravates what is the primary problem, which is using human life in its earliest stages solely for experimental purposes," says Dr. Daniel Sulmasy, a Georgetown University bioethicist.

The idea of extending the 14-day rule even makes some people who support embryo research queasy, especially without first finding another clear stopping point.

Hank Greely, a Stanford University bioethicist, worries that going beyond 14 days could "really draws into question whether we're using humans or things that are well along the path to humans purely as guinea pigs and purely as experimental animals."

Embryo alternative: "Embryoids"

So as that debate continues, Brivanlou and his colleagues are trying to develop another approach. The scientists are attempting to coax human embryonic stem cells to organize themselves into entities that resemble human embryos. They are also using induced pluripotent stem (iPS) cells, which are cells that behave like embryonic stem cells, but can be made from any cell in the body.

Embryoids like this one are created from stem cells and resemble very primitive human embryos. Scientists hope to use them to learn more about basic human biology and development. Courtesy of Rockefeller University hide caption

Embryoids like this one are created from stem cells and resemble very primitive human embryos. Scientists hope to use them to learn more about basic human biology and development.

Brivanlou's lab has already shown that these "embryo-like structures" or "embryoids" can create the three fundamental cell types in the human body.

But the scientists have only been able to go so far using flat lab dishes. So the researchers are now trying to grow these embryonic-like structures in three dimensions by placing stem cells in a gel.

"Essentially, we're trying to, in a way, to re-create a human embryo in a dish starting from stem cells," says Mijo Simunovic, another of Brivanlou's colleagues.

In early experiments, Simunovic says, he has been able to get stem cells to "spontaneously" form a ball with a "cavity in its center." That's significant because that's what early human embryos do in the uterus.

Simunovic says it's unclear how close these structures could become to human embryos entities that have the capability to develop into babies.

"At the moment, we don't know. That's something that's very hot for us right now to try to understand," Simunovic says.

Simunovic argues the scientists are not "ethically limited to studying these cells and studying these structures" by the 14-day rule.

There's a debate about that, however.

"At what point is your model of an embryo basically an embryo?" asks Hyun, especially when the model seems to have "almost like this inner, budding life."

"Are we creating life that, in the right circumstances, if you were to transfer this to the womb it would continue its journey?" he asks.

Dr. George Daley, the dean of the Harvard Medical School and a leading stem cell researcher, says scientists have been preparing for the day when stem-cell research might raise such questions.

"I think what prospects people are concerned about are the kinds of dystopian worlds that were written about by Aldous Huxley in Brave New World," Daley says. "Where human reproduction is done on a highly mechanized scale in a petri dish."

Daley stresses scientists are nowhere near that, and may never get there. But science moves quickly. So Daley says it's important scientists move carefully with close ethical scrutiny.

The latest guidelines issued by the International Society for Stem Cell Research call for intensive ethical review, Daley notes.

Brivanlou acknowledges that some of his experiments have produced early signs of the primitive streak. But that's a very long way from being able to develop a spinal cord, or flesh and bones, let alone a brain. He dismisses the notion that the research on embryoids would ever lead to scientists creating humans in a lab dish.

"They will not get up start walking around. I can assure you that," he says, noting that full human embryonic development is a highly complex process that requires just the right mix of the biology, physics, geometry and other factors.

Nevertheless, Brivanlou says all of his experiments go through many layers of review. And he's convinced the research should continue.

"It would be a travesty," he says, "to decide that, somehow, ignorance is bliss."

Here is the original post:
Human 'Embryoids' And Other Embryo Research Raises Concern ... - NPR

Spinal Cord Stem Cells Comments Off on Human ‘Embryoids’ And Other Embryo Research Raises Concern … – NPR | March 2nd, 2017

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

Bone Marrow Stem Cells Comments Off on Gene Therapy: A Breakthrough for Sickle Cell Anemia? – Auburn Citizen | March 2nd, 2017

Potential Treatment

Gene therapy has been available for quite some time now. Advances in modern medical science, particularly in stem cell research, have made it possible to use DNA to compensate for malfunctioning genes in humans. The therapies haveeven proven effective fortreating rare forms of diseases. Now, a research team in France has shown that gene therapy may be used to cure one of the most common genetic diseases in the world.

The team, led by Marina Cavazzana at the Necker Childrens Hospital in Paris, conducted stem cell treatment on a teenage boy with sickle cell disease. The disease alters theblood through beta-globin mutations, which cause abnormalities in the blood proteinhemoglobin. These abnormalities cause the blood cells (which have an irregular shape, like a sickle, hence their name) to clump together. Patients with sickle cell disease usually need transfusions to clear the blockages their cells cause, and some are able to have bone marrow transplants. About 5 percent of the global population has sickle cell disease,according to the WHO. In the United States alone, the CDC reports that approximately 100,000 people have sickle cell disease.

The patient is now 15 years old and free of all previous medication, Cavazzana saidwhen discussing the outcome of their study. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers. Their research is published in the the New England Journal of Medicine.

The particular treatment given to the teenage boy at Necker Childrens Hospitalbegan when he was 13 years old. The team took bone marrow stem cells from the boy and added mutated versions of the gene that codes for beta-globin before putting these stem cells back into the boys body. The mutated genes were designed to stop hemoglobin from clumping together and blocking blood vessels the hallmark of sickle cell disease.

Two years later, the boys outcomelooks promising.All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up, Cavazzan said. Her team also treated seven other patients who also showed promising progress.

If the method shows success in larger scale clinical trials, it could be a game changer, saidDeborah 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.

Other research involving gene therapy is also showing similar promise. One which has already been approved by the FDA is a potential treatment for blindness. Others look at treating Parkinsons disease or evenprolonging human life. What these studies show is that gene therapyand stem cells may be able togive hope to patients with diseases that have long been considered incurable.

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Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene Therapy - Futurism

Bone Marrow Stem Cells Comments Off on Doctors Claim They’ve Cured a Boy of a Painful Blood Disorder Using Gene Therapy – Futurism | March 2nd, 2017

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

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Sickle cell disease. Image: Flickr

Sickle cell disease is a slow, vicious killer. Most people diagnosed with the red blood cell disorder in the US live to be between 40 and 60. But those years are a lifetime of pain, as abnormal, crescent-shaped hemoglobin stops up blood flow and deprives tissues of oxygen, causing frequent bouts of agony, along with more severe consequences like organ damage. Now, after decades of searching for a cure, researchers are announcing that, in at least one patient, they seem to have found a very promising treatment.

Two years ago, a French teen with sickle cell disease underwent a gene therapy treatment intended to help his red blood cells from sickling. In a paper published Thursday in the New England Journal of Medicine, the researchers revealed that today, half of his red blood cells have normal-shaped hemoglobin. He has not needed a blood transfusion, which many sickle cell patients receive to reduce complications from the disease, since three months after his treatment. He is also off all medicines.

To reiterate, the paper is a case study of just one patient. Bluebird Bio, the Massachusetts biotech company that sponsored the clinical trial, has treated at least six other trials underway in the US and France, but those results have not yet been fully reported. The gene therapy has not worked quite as well in some of those other patients; researchers say they are adjusting the therapy accordingly. It is also possible that the boy may eventually experience some blood flow blockages again in the future.

The results, though early, are encouraging. They represent the promise of new genetics technologies to address a disease that has long been neglected and tinged with racism. Sickle cell disease affects about 100,000 people in the US, most of whom are black. It is an inherited genetic disease caused by a mutation of a single letter in a persons genetic code.

This single-letter mutation makes it a promising candidate for cutting edge technologies, like the gene-editing technique CRISPR-Cas9, and other gene therapies. Recently, a rush of new research has sought to address it. Two other gene therapy studies for sickle cell are underway in the US one at UCLA and another at Cincinnati Childrens Hospital. Yet another is about to start in a collaboration between Harvard and Boston Childrens Hospital. Last fall, researchers all demonstrated the ability to correct the mutation in human cells using CRISPR, though that strategy will yet have to surpass significant scientific and political hurdles before reaching clinical trials.

In the new study, researchers took bone marrow stem cells from the boy and fed them corrected versions of a gene that codes for beta-globin, a protein that helps produce normal hemoglobin. The hope was that those altered stem cells would interfere with the boys faulty proteins and allow his red blood cells to function normally. They continued the transfusions until the transplanted cells began to produce normal-shaped hemoglobin. In the following months, the numbers of those cells continued to increase until in December 2016, they accounted for more than half the red blood cells in his body. In other words, so far so good.

Currently, the only long-term treatment for sickle cell disease is a bone marrow transplant, a high-risk, difficult procedure which many patients are not even eligible for. Pain and other side-effects are treated with blood transfusions for temporary relief. New technologies offer the hope of a solution that could provide long-term relief and allow patients to live some semblance of a normal life.

For decades, gene therapies have been touted as a cure for everything. But so far, successes have been infrequent, and often for very rare diseases. But early success in treating sickle cell disease means that soon, if were lucky, the benefits of this technology may reach hundreds of thousands of people.

[New England Journal of Medicine]

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Will Sickle Cell Be the Next Disease Genetic Engineering Cures? - Gizmodo

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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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By Andy Coghlan

Sarah Harrison and Gaelle Recher, Zernicka-Goetz Lab, University of Cambridge

Artificial mouse embryos grown from stem cells in a dish could help unlock secrets of early development and infertility that have until now evaded us.

Magdalena Zernicka-Goetz at the University of Cambridge and her team made the embryos using embryonic stem cells, the type of cells found in embryos that can mature into any type of tissue in the body.

The trick was to grow these alongside trophoblast stem cells, which normally produce the placenta. By growing these two types of cell separately and then combining them in a special gel matrix, the two mixed and started to develop together.

After around four-and-a-half days, the embryos resembled normal mouse embryos that were about to start differentiating into different body tissues and organs.

They are very similar to natural mouse embryos, says Zernicka-Goetz. We put the two types of stem cells together which has never been done before to allow them to speak to each other. We saw that the cells could self-organise themselves without our help.

This is the first time something resembling an embryo has been made from stem cells, without using an egg in some way. Techniques such as cloning, as done for Dolly the sheep and other animals, bypass the need for sperm, but still require an egg cell.

The artificial embryos are providing new insights into how embryos organise themselves and grow, says Zernicka-Goetz. The team engineered the artificial embryos so the cell types fluoresced in different colours, to reveal their movements and behaviour as the embryos go through crucial changes.

Mammal embryos were already known to start as a symmetrical ball, then elongate, form a central cavity and start developing a type of cell layer called mesoderm, which ultimately goes on to form bone and muscle.

We didnt know before how embryos form this cavity, but weve now found the mechanism for it and the sequential steps by which it forms, says Zernicka-Goetz. Its building up the foundations for the whole body plan.

The work is a great addition to the stem cell field and could be extended to human stem cell populations, says Leonard Zon at Boston Childrens Hospital, Massachusetts. Using the system, the factors that participate in embryo development could be better studied and this could help us understand early events of embryogenesis.

But Robin Lovell-Badge at the Francis Crick Institute in London says that the embryos lack two other types of cell layer required to develop the bodies organs: ectoderm, which forms skin and the central nervous system, and endoderm, which makes our internal organs.

Zernicka-Goetz hopes to see these types of cell layers develop in future experiments by adding stem cells that normally form the yolk sac, a third structure involved in embryonic development, to the mix.

If a similar feat can be achieved using human stem cells, this could tell us much about the earliest stages of our development. Current research is limited by the number of excess embryos that are donated from IVF procedures. But the new technique could produce a limitless supply, making it easier to conduct in-depth research. These artificial embryos may also be easier to tinker with, to see what effect different factors have in early embryogenesis.

Disrupting development in this way may provide new insights into the causes of abnormal embryo development and miscarriage. You would be able to understand the principles that govern each stage of development. These are not normally accessible, because they happen inside the mother, says Zernicka-Goetz.

But it is doubtful that this work could ever lead to fully grown babies in the lab. Lovell-Badge says the artificial embryos are unlikely to develop in vitro much further than shown in the study, as they would soon need the supply of nutrients and oxygen that a placenta normally channels from the mother.

Were not planning to make a mouse in the lab using stem cells, says Zernicka-Goetz. But she is hopeful that adding yolk sac stem cells will allow these artificial embryos to survive long enough to study the beginnings of organs like the heart.

Journal reference: Science, DOI: 10.1126/science.aal1810

Read more: Its time to relax the rules on growing human embryos in the lab

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Artificial embryo grown in a dish from two types of stem cells - New Scientist

Skin Stem Cells Comments Off on Artificial embryo grown in a dish from two types of stem cells – New Scientist | March 2nd, 2017

event

Date: 14 June 2017 - 17 June 2017

Location: Boston Convention and Exhibition Center 415 Summer Street Boston 02210 United States

Website: ISSCR2017.org

Email: [emailprotected]

Telephone: +1 224-592-5700

The International Society for Stem Cell Research (ISSCR) 2017 annual meeting will be held 14-17 June in Boston, Mass., U.S., at the Boston Convention and Exhibition Center. The meeting brings together 4000 stem cell researchers and clinicians from around the world to share the latest developments in stem cell research and regenerative medicine. In a series of lectures, workshops, poster presentations, and a dynamic exhibition floor, researchers focus on recent findings, technological advances, trends, and innovations that are realizing progress in using stem cells in the discovery and validation of novel treatments.

In 2017, the ISSCR is expanding its translational and clinical programming with two half-day, pre-meeting educational sessions geared toward bringing new therapies to the clinic. The Workshop on Clinical Translation (WCT) and the Clinical Advances in Stem Cell Research (CASC) programs are designed for scientists and physicians interested in learning more about the process of developing stem cell-based therapies and advances in stem cell applications in the clinic.

The Presidential Symposium recognizes a decade of progress in iPS cell research and application with a distinguished lineup of speakers including Shinya Yamanaka, discoverer of iPSCs. Additional plenary presentations include distinguished speakers from around the world focusing on organoids and organogenesis, the making of tissues and organs; stem cells and cancer; chromatin and RNA biology; stress, senescence and aging; tissue regeneration and homeostasis; and the frontiers of cell therapy.

Concurrent sessions feature new and innovative developments across the breadth of the field, and incorporate more than 100 abstract-selected speakers. Disease modelling, tissue engineering, stem cell niches, epigenetics, hematopoietic stem cells, and gene modification and gene editing are just a few of the 28 topic areas presented.

Other meeting offerings include career development sessions and networking opportunities. A full listing of the ISSCR 2017 meeting programming can be found at ISSCR2017.org.

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ISSCR 2017 - Drug Target Review - Drug Target Review

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The mystery of what controls the range of developmental clocks in mammals -- from 22 months for an elephant to 12 days for an opossum -- may lie in the strict time-keeping of pluripotent stem cells for each unique species.

Developmental clocks are of high importance to regenerative medicine, since many cell types take long periods to grow to maturity, limiting their usefulness to human therapies. The regenerative biology team at the Morgridge Institute for Research, led by stem cell pioneer and University of Wisconsin-Madison Professor James Thomson, is studying whether stem cell differentiation rates can be accelerated in the lab and made available to patients faster.

In a study published in February online editions of the journal Developmental Biology, Morgridge scientists tested the stringency of the developmental clock in human stem cells during neural differentiation. First, they closely compared the differentiation rates of the cells growing in dishes compared to the known growth rates of human cells in utero. Second, they grew the human stem cells within a mouse host, surrounded by factors -- such as blood, growth hormones and signaling molecules -- endemic to a species that grows much more rapidly than humans.

In both cases -- lab dish and different species -- the cells did not waver from their innate timetable for development, without regard to environmental changes.

"What we found remarkable was this very intrinsic process within cells," says lead author Chris Barry, a Morgridge assistant scientist. "They have self-coding clocks that do not require outside stimulus from the mother or the uterus or even neighboring cells to know their pace of development."

While the study suggests that cellular timing is a stubborn process, the Thomson lab is exploring a variety of follow-up studies on potential factors that could help cells alter their pace, Barry says.

One aspect of the study that's immediately valuable across biology is the realization that how stem cells behave in the dish aligns almost precisely with what happens in nature.

"The promising thing is that we can take species of stem cells, put them in tissue culture, and more confidently believe that events we're seeing are probably happening in the wild as well," Barry says. "That is potentially great news for studying embryology in general, understanding what's going on in the womb, and disease modeling for when things can go wrong."

It also opens up potential avenues in embryology that would have been inconceivable otherwise -- for example, using stem cells to accurately study the embryology of whales and other species with much longer (or shorter) gestation rates than humans.

In order to accurately compare development timing across species with wildly different gestation rates -- nine months compared to three weeks -- the team used an algorithm called dynamic time warping, originally developed for speech pattern recognition. This algorithm will stretch or compress the time frame of one species to match up with similar gene expression patterns in the other. Using this process, they identified more than 3,000 genes that regulate more rapidly in mice and found none that regulate faster in human cells.

The impact of solving the cell timing puzzle could be enormous, Barry says. For example, cells of the central nervous system take months to develop to a functional state, far too long to make them therapeutically practical. If scientists can shorten that timing to weeks, cells could potentially be grown from individual patients that could counteract grave diseases such as Parkinson's, multiple sclerosis, Alzheimer's disease, Huntington's disease and spinal cord injuries.

"If it turns out these clocks are universal across different cell types," says Barry, "you are looking at broad-spectrum impact across the body."

A video highlighting Barry's work can be seen at https://vimeo.com/183526442.

Story Source:

Materials provided by University of Wisconsin-Madison. Original written by Brian Mattmiller. Note: Content may be edited for style and length.

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Stem cells fiercely abide by innate developmental timing, study ... - Science Daily

Spinal Cord Stem Cells Comments Off on Stem cells fiercely abide by innate developmental timing, study … – Science Daily | March 1st, 2017

The mystery of what controls the range of developmental clocks in mammals -- from 22 months for an elephant to 12 days for a opossum -- may lie in the strict time-keeping of pluripotent stem cells for each unique species.

Developmental clocks are of high importance to regenerative medicine, since many cells types take long periods to grow to maturity, limiting their usefulness to human therapies. The regenerative biology team at the Morgridge Institute for Research, led by stem cell pioneer and UW-Madison professor James Thomson, is studying whether stem cell differentiation rates can be accelerated in the lab and made available to patients faster.

In a study published in February online editions of the journal Developmental Biology, Morgridge scientists tested the stringency of the developmental clock in human stem cells during neural differentiation. First, they closely compared the differentiation rates of the cells growing in dishes compared to the known growth rates of human cells in utero. Second, they grew the human stem cells within a mouse host, surrounded by factors -- such as blood, growth hormones and signaling molecules -- endemic to a species that grows much more rapidly than humans.

In both cases -- lab dish or different species -- the cells did not waver from their innate timetable for development, without regard to environmental changes.

"What we found remarkable was this very intrinsic process within cells," said lead author Chris Barry, a Morgridge assistant scientist. "They have self-coding clocks that do not require outside stimulus from the mother or the uterus or even neighboring cells to know their pace of development."

While the study suggests that cellular timing is a stubborn process, the Thomson lab is exploring a variety of follow-up studies on potential factors that could help cells alter their pace, Barry said.

One aspect of the study that's immediately valuable across biology is the realization that how stem cells behave in the dish aligns almost precisely with what happens in nature.

"The promising thing is that we can take species of stem cells, put them in tissue culture, and more confidently believe that events we're seeing are probably happening in the wild as well," Barry said. "That is potentially great news for studying embryology in general, understanding what's going on in the womb and disease modeling for when things can go wrong."

It also opens up potential avenues in embryology that would have been inconceivable otherwise -- for example, using stem cells to accurately study the embryology of whales and other species with much longer (or shorter) gestation rates than humans.

In order to accurately compare development timing across species with wildly different gestation rates -- nine months compared to three weeks -- the team used an algorithm called Dynamic Time Warping, originally developed for speech pattern recognition. This algorithm will stretch or compress the time frame of one species to match up with similar gene expression patterns in the other. Using this process, they identified more than 3,000 genes that regulate more rapidly in mice and found none that regulate faster in human cells.

The impact of solving the cell timing puzzle could be enormous, Barry said. For example, cells of the central nervous system take months to develop to a functional state, far too long to make them therapeutically practical. If scientists can shorten that timing to weeks, cells could potentially be grown from individual patients that could counteract grave diseases such as Parkinson's, Multiple Sclerosis, Alzheimer's, Huntington's disease and spinal cord injuries.

"If it turns out these clocks are universal across different cell types," said Barry, "you are looking at broad-spectrum impact across the body."

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Stem Cells Fiercely Abide by Innate Developmental Timing, Study Shows - Bioscience Technology

Spinal Cord Stem Cells Comments Off on Stem Cells Fiercely Abide by Innate Developmental Timing, Study Shows – Bioscience Technology | March 1st, 2017

SAN CARLOS, Calif., March 1, 2017 /PRNewswire/ --Natera (NTRA), a leader in genetic testing, today announced the upcoming launch of Evercord; a new offering being made commercially available in the second quarter of 2017 that enables expectant parents to collect, store and potentially retrieve their newborn's cord blood and tissue for therapeutic use in transplantation and regenerative medicine applications. Natera aspires to build a different type of national cord blood company, by combining best-in-class cord blood cryopreservation with the company's ability to provide timely information about genetic disease risk; potentially expanding families' stem cell treatment options in the future. Natera will offer Evercord through its leading direct sales channel in the United States.

The Market Potential of Cord Blood Stem Cells Newborn stem cells sourced from umbilical cord blood are known to contain hematopoietic stem cells (HSCs) that can potentially differentiate and regenerate healthy blood and immune systems. Unfortunately, out of the roughly four million births each year in the United States, more than 95% of the cord blood from those births is currently discarded as waste;1 highlighting a significant opportunity both for families who could benefit from cord blood, and for Natera, as it enters the cord blood and tissue banking market. Published data also suggests that one in three people in the United States, or 128million people, could potentially benefit from regenerative medicine applications which, if proven effective, expands the possible therapeutic use of cord blood stem cells.2 More than 300 studies are currently underway, including clinical trials focused on current and new cord blood stem cell therapies in regenerative medicine.3 These trials hold promise for a growing list of conditions, including Alzheimers disease, cerebral palsy, diabetes (Type I/II), spinal cord injury, cartilage and bone repair, and heart defects. More than 30,000 cord blood stem cell treatments have been conducted worldwide and Natera's prenatal tests, including its carrier test, Horizon, currently screen for 35 of the nearly 80 diseases where cord blood stem cell treatment has been administered. Considering the research advances in stem cells and regenerative medicine, it is anticipated that number will, more than likely, continue to grow.

Introducing EvercordThe launch of Evercord is part of a partnership with Bloodworks Northwest, one of the oldest and most reputable public umbilical cord blood banks in the country. Evercord's service will offer expectant families the opportunity to bank their baby's umbilical cord blood and tissue for potential medical use by the child or related family member. Under the terms of the agreement between Natera and Bloodworks, Bloodworks will perform processing and testing services on cord blood samples submitted by Evercord customers and will cryo-preserve the banked cord blood and tissue at its Seattle, Washington-based best-in-class cord blood cryopreservation storage facility. The companies also plan to build a new facility in anticipation of future growth.

The relationship offers several other competitive advantages. Evercord leverages Bloodworks' 20 years of experience processing and banking cord blood; the lab has a strong track record of successfully releasing nearly 1,000 cord blood samples for transplant, more than other leading private cord blood banks.

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Once Evercord is commercially launched, it will expand Natera's existing portfolio of women's reproductive health products, further diversifying Natera's revenue base and adding a traditionally high yield business into its product mix. Evercord will also be well positioned to immediately take advantage of Natera's well-established commercial capabilities, including: patient and healthcare provider digital services through Natera's Patient Portal and Natera Connect, and a specialized salesforce that already calls on the nation's busiest obstetrics and gynecology offices and fertility centers.

"At Natera, we believe adding cord blood and tissue banking to our product offerings is a natural extension of our commitment to family health and beyond," said Matt Rabinowitz, CEO and founder of Natera. "Evercord builds on our excellence as a genetic testing company and our mission to transform the diagnosis and management of genetic diseases. Cord blood stem cells have demonstrated regenerative capabilities that medicine is just beginning to learn how to harness; offering a unique opportunity to potentially generate complete genetic information on a human being at the moment of birth. Many of the genetic diseases that cord blood treats today are also conditions that our tests screen for, which could enable us to offer a far more extensive service offering in the future than those offered by leading 'storage-only' cord blood banks today."

About Bloodworks Northwest's Public Cord Blood ProgramBloodworks Northwest partners with hospitals across the region to recover and store umbilical cord blood from new mothers -- an important source of stem cells for use in cancer treatment, metabolic or immune system disorders, and research. Bloodworks Northwest created the first and only public umbilical cord blood bank in the Northwest of the United States and has program partnerships with 15 hospitals across Washington, Oregon and Hawaii providing nearly 50 units per month to the national cord bloodregistry.

About NateraNatera is a genetic testing company that develops and commercializes non-invasive methods for analyzing DNA. The mission of the company is to transform the diagnosis and management of genetic disease. In pursuit of that mission, Natera operates a CAP-accredited laboratory certified under the Clinical Laboratory Improvement Amendments (CLIA) in San Carlos, CA, and it currently offers a host of proprietary genetic testing services primarily to OB/GYN physicians and fertility centers, as well as to genetic laboratories through its cloud-based Constellation software system.

Product offerings include the Spectrum pre-implantation genetic test for embryo selection during IVF; the Anora miscarriage test to understand the genetic causes of a pregnancy loss; the Horizon carrier test to detect inherited mutations; the Panorama non-invasive prenatal test (NIPT) to identify common chromosomal anomalies in a fetus as early as nine weeks of gestation; and Evercord, a cord blood and tissue banking service offered at birth to expectant parents.

Each test described has been developed and its performance characteristics determined by the CLIA-certified laboratory performing the test.

These tests have not been cleared or approved by the U.S. Food and Drug Administration (FDA). Although FDA does not currently clear or approve laboratory-developed tests in the U.S., certification of the laboratory is required under CLIA to ensure the quality and validity of the tests.

Natera is also applying its unique technologies to develop non-invasive screening and diagnostic tools for earlier detection and improved treatment of cancer. These tests have not been cleared or approved by the U.S. Food and Drug Administration.

Forward-looking statementsThis release contains forward-looking statements. All statements other than statements of historical facts contained in this press release are forward-looking statements. Any forward-looking statements contained in this press release are based upon Natera's historical performance and its current plans, estimates, and expectations, and are not a representation that such plans, estimates, or expectations will be achieved. These forward-looking statements represent Natera's expectations as of the date of this press release.

Subsequent events may cause these expectations to change, and Natera disclaims any obligation to update the forward-looking statements for any reason after the date of this press release. These forward-looking statements are subject to a number of known and unknown risks and uncertainties that may cause actual results to differ materially, including with respect to our efforts to develop and commercialize new product offerings, our ability to successfully increase demand for and grow revenues for our product offerings, whether the results of clinical studies will support the use of our product offerings, whether cord blood or cord tissue will be found to be effective in treating additional conditions, our expectations of the reliability, accuracy and performance of our screening tests, or of the benefits of our screening tests and product offerings to patients, providers and payers.

Additional risks and uncertainties are discussed in greater detail in the sections entitled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" in Natera's Form 10-Q for the quarter ended September 30, 2016. Further information on potential risks that could affect actual results will be included in other filings Natera makes with the SEC from time to time. These documents are available for free on the company's website at http://www.natera.com under the Investor Relations section, and on the SEC's website at http://www.sec.gov.

Contacts:Natera, Inc. Mike Brophy, Chief Financial Officer, 650-249-9091 x1471 mbrophy@natera.com Laura Zobkiw, Corporate and Media Relations, 650-249-9091 x1649 Lzobkiw@natera.com

1https://parentsguidecordblood.org/en/cord-blood-infographic (February 2017). 2 Harris, D., & Rogers, I. (2007). Umbilical Cord Blood: A Unique Source of Pluripotent Stem Cells for Regenerative Medicine.Current Stem Cell Research & Therapy,2(4), 301-309. doi:10.2174/157488807782793790; http://www.mdpi.com/2227-9059/2/1/50/htm 3https://clinicaltrials.gov/ct2/results?term=Umbilical+AND+Stem+Cells (February 2017).

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/natera-inc-announces-launch-of-evercord-cord-blood-and-tissue-banking-service-300415652.html

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Natera, Inc. Announces Launch of Evercord Cord Blood and Tissue Banking Service - Yahoo Finance

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Adult stem cell function declines with age leading to the decline in fitness

The potential therapeutic use of stem cells is a very hot topic these days. Most of the attention has focused on embryonic stem cells and induced Pluripotent Stem cells (iPS cells), which can form every tissue type in the body to regenerate failing organs. The problem is that detailed knowledge is lacking for how to stimulate the embryonic stem cells to form differentiated tissues (e.g. cells that form the heart, pancreas, muscle, and brain). Moreover, because embryonic stem cells are unlimited in their ability to form any type of tissue, the risk of cancer looms large over the therapeutic use of embryonic stem cells. For example, both embryonic and IPS stem cells can form tumors called teratomas when injected into immune-compromised mice. Enter the bodys adult stem cells, which have not generally been associated with cancer and have been used safely as therapeutics in many countries. The problem with adult stem cells is that it is difficult to get enough of them to be effective for most indications or target the harvested adult stem cells to the proper tissue. Moreover, there are scores of different types of adult stem cells in the body, so picking the best type of adult stem cell for a particular therapeutic can be challenging. Thus, adult stem cell therapeutics with all its potential to regenerate damaged organs and tissues is still a work in progress.

But what about the many populations of endogenous adult stem cells that everyone has embedded in every organ system of the body? All the organs and differing tissues of the body appear to have adult stem cells available for regenerating cells in case of injury or disease. It was recently discovered that even brain neurons and heart muscle cells (previously thought to be non-dividing and irreplaceable in adults) have their own reservoirs of adult stem cells for regeneration. Unfortunately, as we age most adult stem cell populations either decline in number and/or lose the ability to differentiate into functional tissue-specific cells. For example, cardiac muscle stem cells exist but old folks have only one half the number of cardiac stem cells found in young people. Thus, adult stem cells become more and more dysfunction with age, which progressively increases organ and tissue dysfunction with age.

There are many examples revealing the role of adult stem cells in aging. First, the outer surface of your skin continuously sloughs off dead cells, so that adult stem cells must continuously replenish the dying skin cells to maintain the skin as an effective protective barrier to the outside world. With age, there are progressively fewer functional skin stem cells, so cell turnover in the skin slows, leading to thinner, dryer skin that loses its elasticity and youthful beauty. Second, hair also thins and goes grey, as functional follicle stem cell decline and the adult stem cells generating hair color also decline. Third, the differing adult stem cells that maintain the tissues composing skeletal muscle, pancreas, heart, bone, liver, kidney, and the immune system lose functional capacity, raising the potential for decline in tissue function or outright failure with age. As a final example, the five senses of sight, hearing, smell, taste, and touch slowly wane with age, as the declining stem cell populations responsible for maintaining these functions are unable to fully replenish the sensory neurons after injury and random cell death.

If your own adult stem cells are a key factor in aging and disease, then one novel way to slow aging and disease is to stimulate your own adult stem cells to maintain their proper numbers and functional capacity to differentiate into the various tissues as needed for repair and regeneration. This makes sense, because in most, if not all, organs of the body, old cells are continually being replaced by new cells coming from the adult stem cell populations. If stem cells are not producing enough new cells, then organs slowly decline in function as you age. Thus, stimulating your own stem cells can be a winning strategy to stave off many of the disorders associated with aging.

In practice, however, stimulating adult stem cell populations in the body is not a simple task. If the proliferation of adult stem cells is over stimulated, then one may get overgrowth of tissues or a potential tumor. Alternatively, one may stimulate the stem cells to proliferate in a balanced and regulated way, but the stem cells lose functionality and cannot differentiate into the desired specialized tissues to replace senescent cells. These twin problems promoting over stimulation or dysfunctional stem cells put real limits on any proposed therapeutic for stimulating stem cells. For example, most current treatments to stimulate immunity or stem cells (nave T cells) rely on complex carbohydrates from mushrooms or microorganisms to provide antigenic material that can stimulate immunity. This will activate the immune system stem cells to make more differentiated non-stem memory T cells directed against the antigenic material, but it does nothing to stimulate more immune stem cells (nave T cells). Indeed, chronic use of such stem cell enhancers may actually lead to stem cell depletion, as more adult stem cells are exhausted from the requirement to respond to the constant presence of the polysaccharide antigen. Indeed, one theory of how the HIV virus causes a defective immune system is that it exhausts the supply of nave T cells by the repeated attacks of the mutating HIV virus.

Stem Cell 100TM is a nutraceutical supplement that improves the function of your existing stem cells rather than over stimulate stem cells to differentiate or divide. By promoting the stability and vitality of adult stem cells they have the capacity to divide when the body signals a need for more stem cells and differentiated cells. When an organ or tissue is damaged, it will send out natural signals that new cells are needed to replace old or damaged cells. Stem Cell 100TM allows the adult stem cells to respond to the damage signal by provided new differentiated cells to replace the old damaged cells and also make more adult stem cells to keep up the stem cell population. Two other compounds in Stem Cell 100TM provide further natural support for stem cells.

(Note that not everyone will experience the same effects, as conditions vary among individuals. The general expectation is that for most health measurements that are in the Normal Range for your age, Stem Cell 100TM will promote readings that you had when some 20 years younger.)

The statements above have not been reviewed by the FDA. Stem Cell 100TM is not meant as a preventive or treatment for any disease.

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Stem Cells and Aging | Life Code

Cardiac Stem Cells Comments Off on Stem Cells and Aging | Life Code | March 1st, 2017

KARAIKAL: Recent advancements in stem cells research have given hope for successfully treating diabetic heart disease (DHD), renowned New Zealand-based researcher in cardiovascular diseases Dr Rajesh Katare said today.

DHD affected the muscular tissues of the heart leading to complications and it had been demonstrated that resident stem cells of myocardium can be stimulated to repair and replace e degenerated cardiac myocytes resulting in a novel therapeutic effect and ultimately cardiac regeneration, he said.

Katare, Director of Cardiovascular Research Division in the University of Otago, New Zealand, was delivering the keynote address at the continuing medical education programme on "Role of Micro-RNAs and stem cells in cardiac regeneration in diabetic heart disease" at the Karaikal campus of premier health institute JIPMER.

Presenting clinical evidences, Katare said stem cell therapy certainly presented a new hope for successfully treating DHD.

Jawaharlal Institute of Post Graduate Medical Education (JIPMER) Director Dr Subash Chandra Parija pointed out that it was the first such programme on the role of stem cells in cardiac regeneration in the whole of the country.

He said as diabetes was highly prevalent in the country, providing treatment for DHD had become a big challenge. Patients suffering from the condition have to undergo lifelong treatment and medications. "In this backdrop, advancements in stem cell therapy assume significance," he said.

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Xconomy Texas

San Antonio StemBioSys, the life sciences company with a system for growing stem cells, has licensed an experimental technology from University of Texas Health San Antonio that may help identify healthy young adult stem cells among large pools of other cells.

Theres plenty of research examining how to possibly use adult stem cells as treatments for medical conditions, ranging from cardiac disease to metabolic disorders, but current uses are rather limited to therapies like bone-marrow transplants for blood disorders, especially in children. Treatments that use patients own stem cells may be safer than using stem cells from someone else because they might reduce the potential for an immune response, according to StemBioSys CEO Bob Hutchens. Thats still theoretical, he says.

Finding large quantities of usable adult stem cells is difficult, though. StemBioSys believes its new technology can potentially identify a few thousand high-quality, young stem cells from a sample of tens of thousands of cells taken from a patient, Hutchens sayspotentially being a key word.

The research is quite earlythe technology has only been studied in animal models and in vitro, and StemBioSys is in the process of applying for federal grants to take the research into animal trials. If StemBioSys new intellectual property can successfully isolate the stem cells, Hutchens says they could grow more of them with StemBioSys core product.

StemBioSys sells a so-called extracellular matrix product made of proteins that provide a hospitable environment for stem cells, helping them divide and produce more stem cells.

Whats intriguing to us is that its a really interesting application of our technology, Hutchens says. You take this combination of identifying this very small population of young healthy cells in elderly people, and use our technology to expand it.

If the company can indeed find the young stem cells of a single patient and replicate them, it would give researchers and physician an accessible pool of the cells that theyd want for potential stem cell transplants and other treatments, Hutchens says.

Terms of the deal werent disclosed. StemBioSys, which was founded based on other University of Texas System research, acquired a portfolio of issued and pending patents. Famed MIT researcher and Xconomist Robert Langer is on the companys board of directors.

Again, theres plenty to prove out with this early stage research, so it will take time before any potential commercialization comes to fruition. Travis Block, the researcher who helpeddevelopthe technology while earning his PhD. last year at the University of Texas Health Science Center at San Antonio, will help shepherd the project along and other regenerative medicine work as StemBioSyss senior scientist.

David Holley is Xconomy's national correspondent based in Austin, TX. You can reach him at dholley@xconomy.com

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StemBioSys Lands Experimental UT Tech That Finds Young Stem Cells - Xconomy

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SAN CARLOS, Calif. & BALTIMORE--(BUSINESS WIRE)--Johns Hopkins Medicine, the Maryland Stem Cell Research Fund (MSCRF) and BioCardia, Inc. (OTC:BCDA) today announced that the first patient has been treated in the pivotal Phase III CardiAMP clinical trial of a cell-based therapy for the treatment of ischemic heart failure that develops after a heart attack. The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, MD, a faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.

The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone marrow cells directly to the point of cardiac dysfunction, potentially stimulating the bodys natural healing mechanism after a heart attack.

The patient experience with CardiAMP therapy begins with a pre-procedural cell potency screening test. If a patient qualifies for therapy, they are scheduled for a bone marrow aspiration. A point of care cell processing platform is then utilized to concentrate the autologous bone marrow cells, which are subsequently delivered in a minimally-invasive procedure directly to the damaged regions in a patients heart.

This cell-based therapy offers great potential for heart failure patients, said Carl Pepine, MD, professor and former chief of cardiovascular medicine at the University of Florida, Gainesville and national co-principal investigator of the CardiAMP trial. We look forward to validating the impact of the therapy on patients quality of life and functional capacity in this important study.

In addition to Dr. Johnston, the CardiAMP research team at Johns Hopkins includes Gary Gerstenblith, MD, Jeffrey Brinker, MD, Ivan Borrello, MD, Judi Willhide, Katherine Laws, Audrey Dudek, Michele Fisher and John Texter, as well as the nurses and technicians of the Johns Hopkins Cardiovascular Interventional Laboratory.

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, said Dan Gincel, PhD., executive director of the MSCRF at TEDCO. Through our clinical program, we are advancing cures and improving healthcare in the State of Maryland.

The CardiAMP Heart Failure Trial is a phase III, multi-center, randomized, double-blinded, sham-controlled study of up to 260 patients at up to 40 centers nationwide, which includes an optional 10-patient roll-in cohort. The primary endpoint for the trial is a significant improvement in Six Minute Walk distance at 12 months post-treatment. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack. The national co-principal investigators are Dr. Pepine and Amish Raval, MD, of the University of Wisconsin.

For information about eligibility or enrollment in the trial, please visit http://www.clinicaltrials.gov or ask your cardiologist.

About BioCardia BioCardia, Inc., headquartered in San Carlos, CA, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the companys biotherapeutic product candidates in clinical development. For more information, visit http://www.BioCardia.com.

About Johns Hopkins Medicine Johns Hopkins Medicine (JHM), headquartered in Baltimore, Maryland, is one of the leading health care systems in the United States. Johns Hopkins Medicine unites physicians and scientists of the Johns Hopkins University School of Medicine with the organizations, health professionals and facilities of The Johns Hopkins Hospital and Health System. For more information, visit http://www.hopkinsmedicine.org.

About Maryland Stem Cell Research Fund The Maryland Stem Cell Research Act of 2006was established by the Governor and the Maryland General Assembly during the 2006 legislative session and created the Maryland Stem Cell Research Fund. This fund is continued through an appropriation in the Governor's annual budget. The purpose of the Fund is to promote state-funded stem cell research and cures through grants and loans to public and private entities in the State. For more information, visit http://www.MSCRF.org.

Forward Looking Statements This press release contains forward-looking statements as that term is defined under the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, among other things, references to the enrollment of our Phase 3 trial, commercialization and efficacy of our products and therapies, the product development timelines of our competitors. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with developing new products or technologies, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardias business and product development plans, competition in the industry in which BioCardia operates and overall market conditions, and whether the combined funds will support BioCardias operations and enable BioCardia to advance its pivotal Phase 3 CardiAMP cell therapy program. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

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Wednesday, March 1, 2017 6:01 AM UTC

PRESS RELEASE

TiGenix to present at Cowen's 37thAnnual Health Care Conference in Boston

Leuven (BELGIUM) - 1st March, 2017, 07:00h CET - TiGenix NV (Euronext Brussels and Nasdaq: TIG), an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic expanded stem cells, today announced that Eduardo Bravo, CEO of TiGenix, will be presenting at the 37th Annual Cowen and Company's Health Care Conference in Boston (USA) at The Boston Marriott Copley Place on Monday, March 6 at 3:20-3:50PM (EST) in Regis, 3rd Floor (breakout at 4:00 PM-04:30PM (EST) at Boston University, 3rd Floor).

The presentation will be webcast live and can be accessed on the day of the event at this link. A replay of the webcast will be available on the Company's website for 30 days following the presentation. To ensure timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast.

The TiGenix management team will be available for one-to-one meetings from Monday, March 6th to Wednesday, March 8th. Please contact Investor Relations at Investor@tigenix.com for a meeting request.

For more information:

Claudia D'Augusta Chief Financial Officer T: +34 91 804 92 64 claudia.daugusta@tigenix.com

About TiGenix

TiGenix NV (Euronext Brussels: TIG) is an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic, or donor-derived, expanded stem cells. Our lead product candidate from the adipose-derived stem cell technology platform is Cx601, which is in registration with the European Medicines Agency for the treatment of complex perianal fistulas in Crohn's disease patients. Our adipose-derived stem cell product candidate Cx611 has completed a Phase I sepsis challenge trial and a Phase I/II trial in rheumatoid arthritis. Effective July 31, 2015, TiGenix acquired Coretherapix, whose lead cellular product candidate, AlloCSC-01, is currently in a Phase II clinical trial in Acute Myocardial Infarction (AMI). In addition, the second product candidate from the cardiac stem cell-based platform acquired from Coretherapix, AlloCSC-02, is being developed in a chronic indication. On July 4, 2016, TiGenix entered into a licensing agreement with Takeda, a large pharmaceutical company active in gastroenterology, under which Takeda acquired the exclusive right to commercialize Cx601 for complex perianal fistulas outside the United States. TiGenix is headquartered in Leuven (Belgium) and has operations in Madrid (Spain).

About Cx601

Cx601 is a suspension of allogeneic expanded adipose-derived stem cells (eASC) locally injected. Cx601 is an investigational agent being developed for the treatment of complex perianal fistulas in Crohn's disease patients with inadequate response to at least one conventional or biologic therapy including antibiotics, immunosuppressants, or anti-TNF agents. Crohn's disease is a chronic inflammatory disease of the intestine and patients can suffer from complex perianal fistulas for which there is currently no effective treatment. In 2009, the European Commission granted Cx601 orphan designation for the treatment of anal fistulas, recognizing the debilitating nature of the disease and the lack of treatment options. Cx601 has met the primary end-point in the Phase III ADMIRE-CD study in Crohn's disease patients with complex perianal fistula, a randomized, double-blind, placebo-controlled trial run in Europe and Israel and designed to comply with the requirements laid down by the EMA. 'Madrid Network' issued a soft loan to help finance this Phase III study, which was funded by the Secretary of State for Research, Development and Innovation (Ministry of Economy and Competitiveness) within the framework of the INNTEGRA plan. The study's primary endpoint was combined remission, defined as clinical assessment at week 24 of closure of all treated external openings draining at baseline despite gentle finger compression, and absence of collections >2cm confirmed by MRI. In the ITT population (n=212), Cx601 achieved statistically significant superiority (p=0.024) on the primary endpoint with 50% combined remission at week 24 compared to 34% in the placebo arm. Efficacy results were robust and consistent across all statistical populations. Treatment emergent adverse events (non-serious and serious) and discontinuations due to adverse events were comparable between Cx601 and placebo arms. The 24-weeks results have been published by The Lancet, one of the most highly regarded and well known medical journals in the world. The Phase III study has completed a follow-up analysis at 52 weeks confirming its sustained efficacy and safety profile. Top line follow-up data showed that in the ITT population Cx601 achieved statistical superiority (p=0.012) with 54% combined remission at week 52 compared to 37% in the placebo arm. The 52-week data also showed a higher rate of sustained closure in those patients treated with Cx601 and in combined remission at week 24 (75.0%) compared to patients in the placebo group (55.9%). Based on the positive 24-weeks Phase III study results, TiGenix has submitted a Marketing Authorization Application to the EMA in early 2016. TiGenix is preparing to develop Cx601 in the U.S. after having reached an agreement with the FDA through a special protocol assessment procedure (SPA) in 2015. On July 4, 2016 TiGenix entered into a licensing agreement with Takeda, a pharmaceutical company leader in gastroenterology, whereby Takeda acquired an exclusive right to commercialize Cx601 for complex perianal fistulas in Crohn's patients outside of the U.S.

Forward-looking information

This press release may contain forward-looking statements and estimates with respect to the anticipated future performance of TiGenix and the market in which it operates. Certain of these statements, forecasts and estimates can be recognised by the use of words such as, without limitation, "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will" and "continue" and similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to predict and may depend upon factors that are beyond the Company's control. Therefore, actual results, the financial condition, performance or achievements of TiGenix, or industry results, may turn out to be materially different from any future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of the publication of this press release. TiGenix disclaims any obligation to update any such forward-looking statement, forecast or estimates to reflect any change in the Company's expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement, forecast or estimate is based, except to the extent required by Belgian law.

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TiGenix to present at Cowen's 37th Annual Health Care Conference in Boston - EconoTimes

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