The skin cells of four adults with schizophrenia provide an unprecedented window into how the disease began before they were born.

Scientists call the findings the first proof of concept for the hypothesis that a common genomic pathway lies at the root of schizophreniaand say the work is a step toward the design of treatments that could be administered to pregnant mothers at high risk for bearing a child with schizophrenia, potentially preventing the disease before it begins.

We show for the first time that there is, indeed, a common, dysregulated gene pathway at work here.

In the last 10 years, genetic investigations into schizophrenia have been plagued by an ever-increasing number of mutations found in patients with the disease, says Michal K. Stachowiak, professor of pathology and anatomical sciences at the University at Buffalo. We show for the first time that there is, indeed, a common, dysregulated gene pathway at work here.

The authors gained insight into the early brain pathology of schizophrenia by using skin cells from four adults with schizophrenia and four adults without the disease. The cells were reprogrammed back into induced pluripotent stem cells and then into neuronal progenitor cells.

By studying induced pluripotent stem cells developed from different patients, we recreated the process that takes place during early brain development in utero, thus obtaining an unprecedented view of how this disease develops, said Stachowiak. This work gives us an unprecedented insight into those processes.

Stachowiak says the research, published in Schizophrenia Research, is a proof of concept for a hypothesis he and colleagues published in 2013 that proposed that a single genomic pathway, called the Integrative Nuclear FGFR 1 Signaling (INFS), is a central intersection point for multiple pathways involving more than 100 genes believed to be involved in schizophrenia.

This research shows that there is a common dysregulated gene program that may be impacting more than 1,000 genes and that the great majority of those genes are targeted by the dysregulated nuclear FGFR1, Stachowiak says.

When even one of the many schizophrenia-linked genes undergoes mutation, by affecting the INFS it throws off the development of the brain as a whole, similar to the way that an entire orchestra can be affected by a musician playing just one wrong note, he says.

The next step in the research is to use these induced pluripotent stem cells to further study how the genome becomes dysregulated, allowing the disease to develop.

We will utilize this strategy to grow cerebral organoidsmini-brains in a senseto determine how this genomic dysregulation affects early brain development and to test potential preventive or corrective treatments.

Other researchers from University at Buffalo and the Icahn School of Medicine at Mt. Sinai are coauthors of the work, which was funded by NYSTEM, the Patrick P. Lee Foundation, the National Science Foundation, and the National Institutes of Health.

Source: University at Buffalo

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Skin cells suggest schizophrenia may start in the womb - Futurity: Research News

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The skin cells of four adults with schizophrenia provide a unique insight into how the disease began before they were born.

Scientists call the findings the first proof of concept for the hypothesis that a common genomic pathway lies at the root of schizophrenia. They add the work is a step toward the design of treatments that could be administered to pregnant mothers at high risk for bearing a child with schizophrenia, potentially preventing the disease before it begins.

Michal K. Stachowiak, professor of pathology and anatomical sciences at the University at Buffalo, says:

In the last 10 years, genetic investigations into schizophrenia have been plagued by an ever-increasing number of mutations found in patients with the disease. We show for the first time that there is, indeed, a common, dysregulated gene pathway at work here.

The authors used skin cells from four adults with schizophrenia and four adults without the disease. The cells were reprogrammed back into induced pluripotent stem cells and then into neuronal progenitor cells.

By studying induced pluripotent stem cells developed from different patients, we recreated the process that takes place during early brain development in utero, thus obtaining an unprecedented view of how this disease develops, said Stachowiak. This work gives us an unprecedented insight into those processes.

Stachowiak says the research is a proof of concept for a hypothesis he and colleagues published in 2013 that proposed that a single genomic pathway, called the Integrative Nuclear FGFR 1 Signaling (INFS), is a central intersection point for multiple pathways involving more than 100 genes believed to be involved in schizophrenia.

This research shows that there is a common dysregulated gene program that may be impacting more than 1,000 genes and that the great majority of those genes are targeted by the dysregulated nuclear FGFR1, Stachowiak says.

When even one of the many schizophrenia-linked genes undergoes mutation, by affecting the INFS it throws off the development of the brain as a whole, similar to the way that an entire orchestra can be affected by a musician playing just one wrong note, he says.

The next step in the research is to use these induced pluripotent stem cells to further study how the genome becomes dysregulated, allowing the disease to develop.

We will utilize this strategy to grow cerebral organoidsmini-brains in a senseto determine how this genomic dysregulation affects early brain development and to test potential preventive or corrective treatments.

The work was funded by NYSTEM, the Patrick P. Lee Foundation, the National Science Foundation, and the National Institutes of Health.

Image: Views of a Foetus in the Womb (c. 1510 1512) by Leonardo da Vinci

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Schizophrenia May Begin In The Womb, Skin Cells Suggest - ReliaWire

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Freezing and rewarming sections of heart tissue successfully raises hopes for doing the same for the entire organ. Photograph: Sebastian Kaulitzki/Alamy

Scientists have succeeded in cryogenically freezing and rewarming sections of heart tissue for the first time, in an advance that could pave the way for organs to be stored for months or years.

If the technique scales up to work for entire organs and scientists predict it will it could save the lives of thousands who die each year waiting for transplants.

The work is being hailed as a major development in the field of cryopreservation as it marks the first time that scientists have been able to rapidly rewarm large tissue samples without them shattering, cracking or turning to a pulp. The US team overcame this challenge by infusing the tissue with magnetic nanoparticles, which could be excited in a magnetic field, generating a rapid and uniform burst of heat.

Kelvin Brockbank, chief executive officer of Tissue Testing Technologies in Charleston, South Carolina and a co-author, said: It is a huge landmark for me. We can actually see the road ahead for clinical use and getting tissues and organs banked and into patients.

Currently, donor organs such as hearts, livers and kidneys must be transplanted within hours because the cells begin to die as soon as the organs are cut off from a blood supply. As a result, 60% of the hearts and lungs donated for transplantation are discarded each year, because these tissues cannot be kept on ice for longer than four hours.

Recent estimates suggest that if only half of unused organs were successfully transplanted, transplant waiting lists could be eliminated within two to three years. The latest paper has been hailed as a significant step towards this goal.

Mehmet Toner, a professor of bioengineering who is working on cryopreservation at Harvard Medical School, said: Its a major breakthrough. Its going to catalyse a lot of people to try this in their laboratories. Im impressed.

Cryopreservation has been around for decades, but while it works well for red blood cells, sperm and eggs, scientists have come up against a barrier for samples with a volume larger than around one millilitre.

Previously, larger samples have been cooled successfully using a technique known as vitrification, in which the tissue is infused with a mixture of antifreeze-like chemicals and an organ preservation solution. When cooled to below -90C (-130F), the fluid becomes a glass-like solid and prevents damaging ice crystals from forming.

The real problem has been the thawing process. Unless the rewarming occurs rapidly and uniformly, cracks will appear in the tissue and tiny ice crystals suddenly expand, destroying cellular structures.

We can freeze tissue and it looks good, but then we warm it and there are major issues, said Toner.

The latest work scales up cryopreservation from one millilitre to about 50ml, and the scientists said they believe the same strategy is likely to work for larger skin transplants, sections of ovarian tissue and entire organs.

John Bischof, professor of mechanical engineering at the University of Minnesota and the senior author of the study, said: We have extremely promising results and we believe that were going to be able to do it but we have not yet done it.

Brockbank and colleagues previously attempted and failed to use microwave warming to generate an even thawing. It failed dreadfully due to the development of hotspots in the tissue, he said.

In the latest paper, published in the journal Science Translational Medicine, the team describe the new nano-warming technique. Pig heart valves and blood vessels were infused with a cryoprotectant solution mixed with iron oxide nanoparticles, coated in silicon to make them biologically inert, and the samples were cooled in liquid nitrogen to -160C (-256F).

For thawing, the sample was placed inside an electromagnetic coil, designed to generate an alternating magnetic field. As the magnetic field is flipped back and forth, the particles jiggle around inside the sample and rapidly and uniformly warm tissue at rates of 100 to 200C per minute, 10 to 100 times faster than previous methods.

In tests of their mechanical and biological properties, the tissues did not show any signs of harm, unlike control samples rewarmed slowly over ice. The researchers were also able to successfully wash away the iron oxide nanoparticles from the sample following the warming although said that further safety testing would be required before the technique could be used in patients.

The team are now testing the technique on rabbit kidneys and human allografts, which are combinations of skin, muscle and blood vessels from donors.

That will be our first trial with human tissues, said Brockbank. If that is successful, we would then progressively move to structures such as the human face for banking and for hands for banking as well as digits.

However, he added that it was difficult to put a timeline on when the developments might have a clinical impact, as this depended on regulatory approval as well as overcoming significant scientific challenges.

The scientists acknowledged that their work may attract interest from the cryonics industry, which promises to freeze the bodies or heads of clients after their death in the hope of bringing them back to life in the future, when medicine has advanced.

There is a certain intellectual connecting of the dots that takes you from the organ to the person... I could see somebody making this argument, said Bischof, but added these ambitions were not science-based as unlike with organs, the person would already be dead when frozen.

Clive Coen, professor of neuroscience at Kings College London, described the technique as ingenious. If the technique can be scaled-up to large organs such as kidneys, the contributions to the field of organ transplantation could be immense, he said. Such painstaking and careful research is to be applauded and must not be confused with wishful thinking about sub-zero storage and subsequent reanimation of a human body, as envisaged by the cryonics industry

Almost 49,000 people in Britain have had to wait for an organ transplant in the past decade and more than 6,000, including 270 children, have died before receiving the transplant they needed, NHS statistics reveal.

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Heart tissue cryogenics breakthrough gives hope for transplant patients - The Guardian

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A B.C. man who is challenging the provinces laws on the preservation of the body after death has signed a groundbreaking cryonic contract. Keegan Macintoshis believed to be the first person to sign a deal with a Canadian provider to keep his body in a state of permanent suspension.

The four-page contract between Keegan Macintosh and the Lifespan Society of B.C. is accepted to be the first run through a Canadian has marked with a neighborhood supplier to keep their body in a condition of lasting suspension.

The agreement is the most recent turn in a strange B.C. Preeminent Court confrontation over the regions Cremation, Interment and Funeral Services Act.

Macintoshs claim says the province is the only place on theplanet to fugitive cryonics.

The issue of cryonics increased overall consideration this month when a British judge allowed the last wishes of a 14-year-old who composed a letter before kicking the bucket of malignancy asking the court to let her mom cryogenically safeguard her body.

The decision made room for the young ladys remaining parts to be taken to an office in the U.S. to begin the conservation procedure at a cost of more than $62,000.

Various Canadians have marked cryonic safeguarding manages U.S. suppliers, however, Lifespan president Carrie Wong says the agreement with Macintosh is accepted to be the first of its kind in Canada.

Mac has altered his unique explanation of claim to mirror the marking of an agreement. Wong said the general public is currently holding up to perceive how the Crown reacts.

Wong said, If theyre really not interested, then anyone in B.C. can go into a cryonics arrangement.

As indicated by the terms of the arrangement, Lifespan will supplant Macintoshs blood with a sort of liquid catalyst to avoid ice gems framing when the body is cooled.

The general public additionally consents to suspend Macintoshs remaining parts at ultra-low temperatures.

Consequently, Macintosh will pay $30 a year.

The agreement gives a progression of capabilities around revival, beginning with the finishing date.

However, Lifespan additionally concurs that when in Lifespans best judgment, it is determined that attempting resuscitation is in the best interests of the cryopreserved member, Lifespan shall attempt to resuscitate (Macintosh).

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Keegan Macintosh-British Columbia Guy Signs First Canadian Cryonic Contract - E Canada Now

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A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursdays New England Journal of Medicine.

About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.

Vexing questions of race and stigma have shadowed the history of its medical treatment, including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.

The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When its defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.

A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but theyre a temporary fix. Gene therapy offers hope of a lasting one.

The boy, now 15, was treated at Necker Childrens Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.

Its not a cure but it doesnt matter, because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French governments research agency.

Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.

Two other gene therapy studies for sickle cell are underway in the U.S. at the University of California, Los Angeles and Cincinnati Childrens Hospital and another is about to start at Harvard and Boston Childrens Hospital using a little different approach.

This work gives considerable promise for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Childrens Hospital doctor who is an inventor on a patent related to gene editing.

The results are quite good in this patient, he said of the French teen. It shows gene therapy is on the right track.

___

Online:

Gene therapy: http://ghr.nlm.nih.gov/primer/therapy/availability

___

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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Gene therapy lets a French teen dodge sickle cell disease - The Seattle Times

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Photo: Michael Cummo / Hearst Connecticut Media

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy CEO and President John Walter inside his office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy CEO and President John Walter inside his office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy Executive Director Margaret Cianci inside her office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy Executive Director Margaret Cianci inside her office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Stamford-based ACGT sees success with support of cancer research

STAMFORD The Alliance for Cancer Gene Therapy recently announced two grants totaling $500,000. But its leaders are already filling the funding pipeline to support future recipients.

Describing itself as the only nonprofit in the country solely dedicated to funding and supporting research into cell and gene therapies for cancer, ACGT has built itself into a fundraising force since its founding in 2001. It has awarded 52 researcher grants totaling almost $27 million. Major events such as Swim Across Americas annual swim between Greenwich and Stamford and a gala held other every other year the next gala is set for April 19 at the Harvard Club in Manhattan are founts for the organizations giving.

We want to keep creating new interest in the organization, new donors and new dollars, ACGT President and CEO John Walter said in an interview Monday. For people who are interested in investing in cancer research, were a good place to turn to because of the success weve had.

Research backed by ACGT focuses on gene and cell mutations, a targeted approach that aims to reduce or eliminate debilitating side effects of traditional treatments.

The recipients of ACGTs latest Young Investigator Awards, the University of Calgarys Marco Gallo and the University of Pittsburghs Greg Delgoffe, each earned a $250,000 grant.

We play a very key role in helping young investigators, said Margaret Cianci, ACGTs executive director. They have wonderful ideas, and we want to help them fund that research and move into the next phase.

ACGT-supported research has produced four companies that are developing major new treatments, according to ACGT officials. They said they hope the first gene-therapy drug will launch in the U.S. later this year. One of the drugs that could soon enter the market is based on Car T cell research by an ACGT fellow, Dr. Carl June, who is an immunotherapy professor at the University of Pennsylvania.

Buoyed by recent advances, ACGT officials said that they want to keep supporting research of emerging treatments.

While you have these excellent immunotherapies, how can we combine them with some of the gene therapy going on? Cianci said. The goal is to be sure that the cancers dont come back.

All of ACGTs donations fund research. The nonprofits board covers administrative expenses for the nonprofits staff of four.

Headquartered on Cummings Point Road in the citys Waterside section, ACGT has made several leadership changes recently. Walter and Barbara Gallagher, national director of philanthropy, both arrived last year. Walter succeeded Barbara Netter, who co-founded ACGT with her late husband, Edward Netter.

Barbara Netter, a Greenwich resident, maintains an important role in the organization as honorary chairman. She said that the organizations work is fulfilling Edward Netters vision.

With the promise of new drugs coming on the market, it should really make a statement about gene therapy, she said. Im very optimistic.

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Stamford-based ACGT sees success with support of cancer research - The Advocate

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Posted Feb. 28, 2017 at 3:39 p.m.

Published: 2017-02-28 15:39:55 Updated: 2017-02-28 15:39:55

By JIM SHAMP, NCBiotech Writer

Raleigh, N.C. The North Carolina Biotechnology Center has announced that Pfizer has committed to providing funding in the amount of $4 million which will enable the Center to establish and administer a multi-year academic fellowship program to help advance North Carolinas fast-growing expertise in gene therapy.

The new program, to be managed by NCBiotech, will support distinguished postdoctoral fellowships in North Carolina university research laboratories providing advanced scientific training in gene therapy-related research.

Absent or faulty proteins linked to genetic mutations cause numerous devastating diseases, making gene therapy an increasingly important treatment strategy.

Pfizers portfolio in North Carolina has grown in recent years. The company already operates a pharmaceutical manufacturing facility in the Lee County community of Sanford, and in August 2016, it acquired leading-edge gene therapy company Bamboo Therapeutics, Inc. in Chapel Hill.

With that acquisition, Pfizer gained the expertise of Bamboos world-renowned co-founder, R. Jude Samulski, Ph.D., director of the Gene Therapy Centerat the University of North Carolina at Chapel Hill. The deal also included an 11,000-square-foot facility for the highly specialized manufacturing of recombinant adeno-associated viral vectors.

Pfizer is one of several biopharmaceutical companies that have added high-profile gene therapy acquisitions, and several partnerships with biotechnology companies and leading academic institutions, to its R&D portfolio. Numerous other North Carolina scientists and companies are also making significant inroads into gene therapy, gene editing and related applications, many with NCBiotech support. For example, Samulski was recruited to UNC in 1993 as part of a $430,000 NCBiotech grant. Additionally, Bamboos former parent company received more than $700,000 in Biotech Center grants and loans.

Gene therapy advances require specific skills in addition to deep scientific knowledge. The fellowship program being established with Pfizers funding aims to boost that talent pipeline, with talent that has already proven to be exceptional in North Carolina. Such funding will enable NCBiotech to provide two-year fellowship support to postdoctoral scientists. The funding will afford the Center the ability to cover salaries, benefits, materials, professional development and travel for such postdoctoral scientists. The Center will encourage competitive applications from scientists interested in establishing research careers in gene therapy and related research activities.

The Biotech Center will also create and manage a related gene therapy Exchange Group. It will join some 25 other exchange groups designed to unite North Carolina-based academic and industry scientists with shared professional interests. The Gene Therapy EG will include these new postdoctoral fellows, their mentors, and others interested in the burgeoning gene therapy sector.

The field of gene therapy research has made tremendous strides in recent years, and we are pleased to be able to further enhance our leadership position in this area through this unique fellowship program, said Mikael Dolsten, M.D., Ph.D., president of worldwide research and development at Pfizer. We believe that gene therapy may hold the promise of bringing true disease modification for patients suffering from devastating diseases, and North Carolina is uniquely positioned to help us take advantage of collaborative opportunities that can develop the specialized talent well need.

Doug Edgeton, president and CEO of the Biotech Center, said he was deeply honored that Pfizer targeted North Carolina, and the Center, for the groundbreaking fellowship program.

Pfizer embraced the opportunity to work with us given weve proven for more than 30 years that we have the expertise and success metrics to maximize impact, said Edgeton. We not only have outstanding research institutions across our state, but we also have a well-respected culture of partnering and collaboration that allows us to be nimble and responsive. This is a wonderful example.

(C) N.C. Biotechnology Center

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An experimental gene therapy that turns a patient's own blood cells into cancer killers worked in a major study, with more than one-third of very sick lymphoma patients showing no sign of disease six months after a single treatment, its maker said Tuesday.

In all, 82 percent of patients had their cancer shrink at least by half at some point in the study.

Its sponsor, California-based Kite Pharma, is racing Novartis AG to become the first to win approval of the treatment, called CAR-T cell therapy, in the U.S. It could become the nation's first approved gene therapy.

A hopeful sign: the number in complete remission at six months 36 percent is barely changed from partial results released after three months, suggesting this one-time treatment might give lasting benefits for those who do respond well.

"This seems extraordinary ... extremely encouraging," said one independent expert, Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center.

The worry has been how long Kite's treatment would last and its side effects, which he said seem manageable in the study. Follow-up beyond six months is still needed to see if the benefit wanes, Herbst said, but added, "this certainly is something I would want to have available."

The therapy is not without risk. Three of the 101 patients in the study died of causes unrelated to worsening of their cancer, and two of those deaths were deemed due to the treatment.

It was developed at the government's National Cancer Institute and then licensed to Kite. The Leukemia and Lymphoma Society helped sponsor the study.

Results were released by the company and have not been published or reviewed by other experts. Full results will be presented at the American Association for Cancer Research conference in April.

The company plans to seek approval from the U.S. Food and Drug Administration by the end of March and in Europe later this year.

The treatment involves filtering a patient's blood to remove key immune system soldiers called T-cells, altering them in the lab to contain a gene that targets cancer, and giving them back intravenously. Doctors call it a "living drug" permanently altered cells that multiply in the body into an army to fight the disease.

Patients in the study had one of three types of non-Hodgkin lymphoma, a blood cancer, and had failed all other treatments. Median survival for such patients has been about six months.

Kite study patients seem to be living longer, but median survival isn't yet known. With nearly nine months of follow-up, more than half are still alive.

Six months after treatment, 41 percent still had a partial response (cancer shrunk at least in half) and 36 percent were in complete remission (no sign of disease).

"The numbers are fantastic," said Dr. Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study and has been a paid adviser to Kite. "These are heavily treated patients who have no other options."

One of his patients, 43-year-old Dimas Padilla of Orlando, was driving when he got a call saying his cancer was worsening, chemotherapy was no longer working, and there was no match to enable a second try at a stem cell transplant.

"I actually needed to park ... I was thinking how am I going to tell this to my mother, my wife, my children," he said. But after CAR-T therapy last August, he saw his tumors "shrink like ice cubes" and is now in complete remission.

"They were able to save my life," Padilla said.

Of the study participants, 13 percent developed a dangerous condition where the immune system overreacts in fighting the cancer, but that rate is lower than in some other tests of CAR-T therapy. The rate fell during the study as doctors got better at detecting and treating it sooner.

Roughly a third of patients developed anemia or other blood-count-related problems, which Locke said were easily treated. And 28 percent had neurological problems such as sleepiness, confusion, tremor or difficulty speaking, but these typically lasted just a few days, Locke said.

"It's a safe treatment, certainly a lot safer than having progressive lymphoma," and comparable to combination chemotherapy in terms of side effects, said the cancer institute's Dr. Steven Rosenberg, who had no role in Kite's study. The first lymphoma patient Rosenberg treated this way, a Florida man, is still in remission seven years later.

There were no cases of swelling and fluid in the brain in this or any other study testing Kite's treatment, company officials said. That contrasts with Juno Therapeutics, which has had a CAR-T study put on hold twice after five patient deaths due to this problem.

Company officials would not say what the treatment might cost, but other types of immune system therapies have been very expensive. It's also being tested for some other types of blood cancer.

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Gene therapy to fight a blood cancer succeeds in major study - Fox News

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Antares Pharma Announces FDA Acceptance of New Drug Application for Quickshot Testosterone P&T Community We continue to believe QST could be an excellent treatment option for men with hypogonadism based upon the positive pharmacokinetic and safety data produced in the two phase three studies now on file with the FDA. In addition to virtually eliminating ... and more »

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Antares Pharma Announces FDA Acceptance of New Drug Application for Quickshot Testosterone - P&T Community

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Testosterone is more than just a sex hormone. Its role goes beyond giving pubescent boys growth spurts, and its effects on the male body are lifelong. But talk of a male menopause, marked by reduced testosterone from middle age, is often met with controversy.

Here are a few things you might not know about men and testosterone:

Testosterone plays a part in maintaining muscle mass, physical energy and mental alertness, as well as libido and sexual stimulation . Since these characteristics are associated with youth, it's no surprise that men produce gradually less testosterone as they age. The rate of decline varies, but levels typically drop by around 20 to 50 per cent between early adulthood when they are at their peak and when a man reaches his 80s .

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The medical term for low testosterone in men is 'male hypogonadism'. It can be caused by problems with the testicles, for example resulting from infection, chemotherapy, certain autoimmune conditions and some tumours.

It can also signal a condition affecting the pituitary gland, which sits at the bottom of the brain stem. If the function of the pituitary gland becomes impaired through a head injury, tumour or using anabolic steroids, for example it has a knock-on effect on certain hormones, which in turn means the testicles don't make as much testosterone.

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Professor Mike Kirby, a GP and visiting professor to the Prostate Centre, says that as they get older, men develop more long-term conditions "such as diabetes, metabolic syndrome and cardiovascular disease and all those things impact on testosterone levels".

Being overweight or obese, also more common in older than in younger men, is also linked with hypogonadism.

So, although it's normal for testosterone levels to fall a certain amount as men get older, the ageing process itself doesn't ordinarily cause testosterone to dip beneath the lower end of normal range. Other health problems are responsible for almost all cases of reduced testosterone in older men, and that, says consultant endocrinologist Dr Richard Quinton, negates the concept of a male menopause. He says:

"For a tiny minority, there is a slightly similar phenomenon to the female menopause, but it's mild and partial rather than complete and absolute."

When a man's testosterone levels are low, he'll often get quite vague symptoms. As well as a change in the sex department loss of libido and erectile dysfunction he might experience difficulty concentrating, insomnia, mood disturbances, weight gain and loss of muscle bulk.

These signs are all-too-easy to ignore, but Dr John Chisholm CBE, a GP and chair of the Men's Health Forum, urges men to get them checked:

"Erectile dysfunction in particular should be looked into because it can be a symptom of serious underlying disease."

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Low testosterone levels can be confirmed by a blood test, and the standard treatment is testosterone replacement by way of a tablet, patch, gel, implant or injection . Evidence suggests that with regular monitoring, testosterone treatment is safe, effective and relatively free of side effects when it's prescribed appropriately .

The problem is that experts disagree about who should be prescribed testosterone. Professor Kirby argues that men with the lowest levels of testosterone level will "almost certainly benefit from treatment regardless of the cause" and that in men whose levels are at the lower end of normal, "it may well be worth addressing the cause first, but some would still benefit from testosterone treatment".

But others, including Dr Quinton, dispute this. He supports treatment only in men who are "genuinely hypogonadal" in other words, those who have consistently low testosterone but aren't obese and have no underlying illness.

"Giving testosterone to men who are either normal, or just have hypogonadism due to chronic ill health including obesity cannot be justified on the basis of available safety and efficacy data."

As well as restoring testosterone levels, testosterone treatment slows the production of certain hormones, switching off sperm production in the testicles. Alternative hormone treatments can work for younger men and those who want to maintain their fertility.

The clear message is that if you're worried about anything, talk to your doctor. The chances are symptoms are nothing to worry about and any problems can be easily rectified

"We'd also recommend they seek help if they're experiencing mental health disturbance. It's better to talk than to avoid issues and conceal symptoms."

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Karaikal, Feb 28 (PTI) 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.

This is published unedited from the PTI feed.

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From left to right: Dr. Fabrice Brunet, The Honorable Michael M Fortier, Mr. Keith Creel, President and CEO of Canadian Pacific, Dr. Gregor Andelfinger, Ms. Maud Cohen, Ms. Janice Pierson, Mr. Richard Lanoue, Mher Mike Stepanian, Samuel Gauthier, Mariama Hawa Barry, Samy Touati, Tyler Lanoue and Olivier Boissonneault. (CNW Group/CHU Sainte-Justine Foundation)

MONTRAL, Feb. 27, 2017 /CNW Telbec/ -An extraordinary $1 million commitment from Canadian Pacific (CP) towards stem cell research will allow the CHU Sainte-Justine to lead the way in developing new treatments to transform the lives of children suffering from complex congenital heart defects. Currently, there is no treatment available to provide a permanent means of repairing the heart. Today, patients and cardiac experts gathered to recognize the major impact of such strong support for research at the CHU Sainte-Justine, as well as the national importance of research in the development of innovative new stem cell technologies.

Thanks to this exceptional gift, CP is making possible the creation of Quebec's first platform for stem cell research and pediatric regenerative medicine. "These funds will allow us to purchase new equipment and recruit an additional researcher, which will significantly accelerate essential research, namely the identification of the mechanisms that form the heart and the types of intervention that can halt the progression of cardiac illnesses in children," stated Dr.Gregor Andelfinger, pediatric cardiologist at the CHU Sainte-Justine and associate research professor in the Department of Pediatrics at the Universit de Montral. "Our aim is to put in place biological factory, capable of producing cardiac tissues from stem cells," he added.

Research remains the best means of understanding, improving the treatment of, and curing congenital heart defects, which are the most commonly occurring birth defects in the world. They affect one in 80 children in Canada every year, many of whom eventually develop fatal heart failure.

"For over a decade, knowledge and understanding about heart defects have grown considerably at the CHU Sainte-Justine, along with the development of new tools for the genetic analysis of families where several family members suffer from a heart defect. Thanks to its team of experts specializing in pediatrics, cardiology, and congenital malformations, the CHU Sainte-Justine is a leader in providing better diagnoses and better targeted therapies to treat congenital heart defects," stated Mr. Fabrice Brunet, CEO of the CHUM-CHU Sainte-Justine.

Ms. Maud Cohen, CEO of the CHU Sainte-Justine Foundation, expressed gratitude for CP's generous support, which provides the hope of regenerating cardiac tissue in babies affected by congenital heart defects. "I am thrilled that the CHU Sainte-Justine is showing such leadership in pediatric regenerative medicine in Quebec, while also increasing our national and international outreach. The CHU Sainte-Justine Foundation is very proud to have the support of CP as a major donor to the Healing More Better campaign. Not only does this remarkable $1 million gift allow for the development of new cures to help save the lives of thousands of children suffering from cardiovascular diseases, but it will also serve as a driver for future funding. This support will enable Dr. Andelfinger's team to quickly undertake activities that show promising early results," she said.

"Since 2014, through our CP Has Heart program, we have been committed to making communities stronger and healthier thanks to research, treatment and prevention. With today's announcement, we have now donated nearly $10 million to this important cause" said Mr. Keith Creel, CP's President and CEO. "When we learned that the CHU Sainte-Justine was seeking to accelerate stem cell research, an extremely promising avenue for the repair of congenital heart defects, we immediately felt that it was an initiative we wanted to support. We firmly believe that a partnership with such a renowned institution as the CHU Sainte-Justine to create the first pediatric research platform in Quebec will significantly improve upon current treatments. This will ensure that the thousands of babies born with heart defects every year will have a chance to grow up with healthy hearts and live healthy lives," Mr. Creel concluded.

For CP, this generous support for stem cell research is a way to pursue its mission to improve heart health throughout North America, and is a natural fit with a cause so close to the company's heart.

The CHU Sainte-Justine Foundation is grateful for CP's invaluable contribution, which will allow the teams at the CHU Sainte-Justine to continue to heal more children, better.

About the CHU Sainte-Justine FoundationThe CHU Sainte-Justine Foundation's mission is to engage the community and support the CHU Sainte-Justine in its pursuit of excellence and its commitment to providing children and mothers with one of the highest levels of healthcare in the world, now and in the future. fondation-sainte-justine.org/en/

About the CHU Sainte-JustineThe Sainte-Justine university hospital centre (CHU Sainte-Justine) is the largest mother-child centre in Canada and the second largest pediatric hospital in North America. A member of the Universit de Montral extended network of excellence in health (RUIS), Sainte-Justine has 5,664 employees, including 1,578 nurses and nursing assistants; 1,117 other healthcare professionals; 502 physicians, dentists and pharmacists; 822 residents and over 200 researchers; 300 volunteers; and 3,400 interns and students in a wide range of disciplines. Sainte-Justine has 484 beds, including 35 at the Centre de radaptation Marie Enfant (CRME), the only exclusively pediatric rehabilitation centre in Quebec. The World Health Organization has recognized CHU Sainte-Justine as a "health promoting hospital." chusj.org

About Canadian PacificCanadian Pacific (TSX:CP)(NYSE: CP) is a transcontinental railway in Canada and the United States with direct links to eight major ports, including Vancouver and Montreal, providing North American customers a competitive rail service with access to key markets in every corner of the globe. CP is growing with its customers, offering a suite of freight transportation services, logistics solutions and supply chain expertise. Visit cpr.ca to see the rail advantages of CP.

About CP Has HeartAt CP, we know that a railroad may serve as the arteries of a nation, but at its heart is community. That's why, through CP Has Heart, we've already committed nearly $10 million to help improve the heart health of men, women and children across North America. And along the way, we're showing heart whenever we can. Find out more on http://www.cpr.ca or @CPhasHeart.

SOURCE CHU Sainte-Justine Foundation

For further information: CHU Sainte-Justine Foundation, Delphine Brodeur, Director, Communication, public relations and donor relations, 514 345-4931, ext. 4356, dbrodeur@fondationSainteJustine.org

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Canadian Pacific makes a $1 million gift to fund stem cell research at the CHU Sainte-Justine - New hope for ... - Canada NewsWire (press release)

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Contributing: Gabe Lacques in Bradenton, Fla.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Vital need for minority bone barrow donors - Story - KTAL

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Transhumanism is one of those technologies that boggles most peoples minds. Do not be mistaken in thinking this has anything to do with being transgender, as transhumanists seek to improve their human capacities beyond what is assumed to be possible. They do so by using top-of-the-line technologies, rather than gadgets or other electronics. Most of these technologies go by unnoticed, which is why we have compiled a brief list below.

Some people may have heard of this technology before. Cryonics is a high-fidelity preservation of the human body after death. The primary reason why anyone would enter a cryogenic sleep is to anticipate a potential future revival. This technology has been widely available for some time, albeit it is rather on the expensive side. Through cryonics, it is feasible to stop cells from decaying. Moreover, the process requires no electricity to do so.

Tampering with the human bodys genes sounds rather risky, but significant advancements have been made in recent years. Gene therapy effectively replaces bad genes with good ones, which allows us to manipulate our genetic code. Scientists have discovered a way to remove genes coding for specific metabolic proteins, ensuring the host remains slim and fit at all times.

Anti-aging therapy is heavily influenced by gene therapy as well and it is believed scientists will eventually reach the longevity escape velocity soon. As a result, humans may become subject to indefinite lifespans. Whether or not that is a positive development, remains to be seen, though.

Introducing cyber enhancements to the human body remains a very risky business to this very day. Implants and other electronics can address a lot of problems our bodies are faced with. Cybernetics are designed in such a way they will be invisible to the casual observer, as they reside beneath the hosts skin. Most current bio modifications are all external, as we have covered in a previous article. Cybernetic systems will improve our everyday experience and even boost the economy as humans will be able to do more work in less time.

While a lot of people are concerned over what the future will bring in terms of robotics, self-replicating robots may be the least of our concerns right now. Replacing manual labor with robots doing the task for us seems like a no-brainer, albeit it will cause some job losses. Self-replicating robots, on the other hand, would be quite beneficial. For example, they can turn uninhabitable areas into living spaces, clean up waste generated by us humans, or even pave the way for human colonization of space.

As creepy as this concept may sound at first, mind uploading or nonbiological intelligence can be quite valuable to our society. Implementing cognitive processing on anything that is not human would be a massive breakthrough. The general public is not too keen of this concept, even though our minds are by far our greatest assets. Synthetic brains are not impossible to achieve by any means, although a lot of research is required before this can become a reality.

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Top 5 Transhumanist Technologies With Major Implications - The Merkle

Recommendation and review posted by Bethany Smith

A high prevalence of hypogonadism in men with Type-2 diabetes mellitus (T2DM) has been reported worldwide.

To evaluate the prevalence of hypogonadism in Indian males with T2DM and assess the primary and secondary hypogonadism along with androgen deficiency.

In this cross-sectional study, 900 men with T2DM were evaluated using androgen deficiency in aging male questionnaire. They were screened for demographic characteristics, gonadal hormone levels, lipid profile, and glycosylated hemoglobin.

The prevalence of hypogonadism in T2DM patients was found to be 20.7% (186 out of 900). Hypogonadism was of testicular origin (primary) in 48/186 (25.8%) patients, of pituitary or hypothalamic origin (secondary) in 14/186 (7.53%), and remaining 124/186 (66.67%) patients were found to have low testosterone with the inappropriate normal level of luteinizing hormone and Follicle-stimulating hormone. 451/900 (50.1%) patients were only symptomatic but had normal testosterone levels. Further 263 patients out 900 were asymptomatic, of which 51/900 (5.7%) patients had low levels of testosterone and 212/900 (23.5%) patients had normal testosterone level without symptoms. There were no deaths or other serious adverse events except mild pyrexia which was not related to the study.

Hypogonadism diagnosis, at times, might not be validated with the help of androgen deficiency questionnaire or symptoms only. Given the large number of patients of T2DM in India, the incidence of hypogonadism is more in diabetic patients as compared to the general population. Hence, implementation of screening programs in diabetic patients is necessary to understand and detect individuals with low serum total testosterone at any early stage and to supplement testosterone accordingly.

Indian journal of endocrinology and metabolism. 0000 Jan [Epub]

Pankaj Kumar Agarwal, Parminder Singh, Subhankar Chowdhury, S K Sharma, Anirban Majumdar, Parag Shah, Rakesh Sahay, S Vageesh Ayyar, Hemant Phatale, Chandar M Batra, Raeesuddin Syed, Pradeep Shetty

Hormone Care and Research Center, Near St. Mary's School, Ghaziabad, Uttar Pradesh, India., Department of Endocrinology, Dayanand Medical College and Hospital, Civil Lines, Ludhiana, Punjab, India., Department of Endocrinology, IPGME&R and SSKM Hospital, Ronald Ross Building, 4th Floor, 244, A J C Bose Road, Kolkata, West Bengal, India., Thyroid and Endocrine Centre, Near 4 No. ESI Hospital, Jaipur, Rajasthan, India., Thyroid and Hormone Clinic, Dhakuria, Kolkata, West Bengal, India., Gujarat Endocrine Centre, 2nd Floor, Silver Brook B, Opposite Doctor House, Near Parimal Crossing, Ahmedabad, Gujarat, India., Department of Endocrinology, Osmania General Hospital, 2nd Floor, Golden Jubilee Block, Afzalgunj, Afzalgunj, Hyderabad, Telangana, India., Department of Endocrinology, St. John's Medical College and Hospital, Bengaluru, Karnataka, India., Samrat Endocrine Institute of Diabetes, Obesity and Thyroid, Aurangabad, Maharashtra, India., Department of Endocrinology, Sarita Vihar, Delhi Mathura Road, New Delhi, India., Global Medical Affairs, MSD Pharmaceuticals Private Limited, 10th Floor, Platina Building, C-59, G-Block, Bandra Kurla Complex, Mumbai, Maharashtra, India.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28217500

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A study to evaluate the prevalence of hypogonadism in Indian males with Type-2 diabetes mellitus. - UroToday

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The CDC says about 38 percent of American adults are considered clinically obese and 71 percent are overweight. Research shows that hormone imbalance can have a huge impact on this back-and-forth weight gain and wellness experts are seeing how balancing the hormones can help with weight loss.

In her hormone therapy clinic, Terri DeNeui says the benefits of hormone replacement therapy go beyond increased energy levels, mood and libido.

"After we would get their hormones balanced and some key nutrients in their thyroid, they would come back the next time and say, wow Ive lost ten to 15 pounds and I didnt even try, whats that about," said DeNeui.

Brandy Prince, a nurse practitioner, had issues such as having no energy, and a pattern of losing and gaining weight over and over.

"I was obese at 208 pounds and I felt terrible, I felt terrible about myself, I got out of bed every morning and everything just hurt," said Brandy Prince.

Using pellets that are inserted under the skin, brandy got testosterone, which helped her build muscle and her thyroid levels were increased. She slept better and she lost weight, eventually more than 50 pounds.

So the weight loss was not something that i expected or anticipated, but it was definitely a wonderful benefit," said prince.

"Hormones and whats going on inside the body at the cellular level where metabolism actually happens has got to be a part of any kind of weight loss regime, otherwise you are just gonna be spinning your wheels," said DeNeui.

Doctors say this type of hormone therapy has minimal risk factors and few side effects, although patients with a history of breast or prostate cancer may need further evaluation and doctors may consider alternate options for those patients.

MEDICAL BREAKTHROUGHS RESEARCH SUMMARY

TOPIC: Hormone Therapy Weight Loss REPORT: MB #4220

BACKGROUND: A person is considered obese when their weight is considered higher than what is healthy for their given height. To determine whether a person is obese or not, a body mass index test, or a BMI, is used. The BMI consist of taking the persons weight in kilograms and dividing it by the square of their height in meters. If the result number is higher than 30.0, then they are in the obese range. According to the CDC, around 36 percent of adults in the United States are obese. Those numbers are higher between 40-59 year olds (40%) and 60 and over year olds (37%), than in young adults between the ages of 20 and 39 (32%).

(Source: https://www.cdc.gov/obesity/data/adult.html)

OBESITY SOLUTIONS: Being obese is a condition that puts you in a higher risk for developing cholesterol problems, high blood pressure, diabetes, heart diseases and strokes. Furthermore, it can lead to a lower life expectancy. The solution to treating obesity is not an easy one since this is a complex problem. In order to lose the weight people have to understand that psychological, behavioral, social, economic and environmental factors contribute to the overall problem of obesity. One person may be obese because of heredity, while another may be because of the food they choose to eat. The solution for each obesity case will depend on the factors that are contributing to the problem.

(Source: http://www.washingtontimes.com/news/2009/aug/16/solutions-dealing-americas-obesity-problem/)

HORMONE THERAPY: Hormones are important in determining how your body functions. The reduction of certain hormones leads to a slower metabolism, increased abdominal fat and less energy for exercising, which results in weight being gained. Hormone therapy is a type of therapy that allows for the hormone imbalance to be restored. As a result, this therapy can help people who suffer from being overweight or obese because of hormonal problems. The therapy consists of using pellets that are inserted under the skin to receive the hormone that is needed for each body. The intake of hormones like testosterone, estrogen and progesterone can help with abdominal fat, thyroid levels, appetite, sugar cravings and insulin resistance.

(Source: http://thebiostation.com/resource-center/hormone-replacement-therapy-rc/gain-insight-on-how-hormone-replacement-therapy-can-help-you-lose-weight/ & Terri DeNeui)

FOR MORE INFORMATION ON THIS REPORT, PLEASE CONTACT: Tara Vreeland 303-929-8363 tvreeland@promoteonpurpose.com

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Wellness experts use hormone balancing to fight obesity - WNDU-TV

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Kelly Ripa made headlines earlier this month after saying on Live with Kelly that her husband, Mark Consuelos, is mean after sex, and while she backpedaled Wednesday by clarifying he is simply disinterested afterward, the damage had already been done. Ripas Feb. 9 admission, whether in jest or not, has been trending on various news sites, including this one, ever since.

Regardless of Ripas intention, her observations about her husbands post-sex behavior beg the question: Do men really get disinterestedafter sex? And if so, why?

Popular sciences explanation for men rolling over after ejaculation is a hormonal response alone, Dr. Raj Dasgupta, a professor of pulmonary and sleep medicine at the University of Southern California, and a spokesperson for the American Academy of Sleep Medicine, told Fox News.

THIS IS YOUR BRAIN ON PORNOGRAPHY

However, experts argue the answer isnt so simple.

In fact, Dasgupta said, the conditions under which couples have sex often have as much to do with whether men or women get sleepy after sex as do their postcoital hormonal differences.

Its a balance of both, Dasgupta said.

Dasgupta explained that most couples have sex at night, potentially in cool, quiet rooms, likely in a comfortable bed in other words, the perfect environment for sleeping. And for those men who may have sleep apnea, the aforementioned conditions can create the perfect environment for dozing off.

If you have a male who has undiagnosed sleep apnea, theyre gonna be more tired than the average person, so it doesnt take a lot to tip them over, Dasgupta said.

According to data from the Cleveland Clinic, that may be a fair assessment. The hospital estimates nearly 22 million Americans suffer from sleep apnea a condition marked by shallow breathing that impacts quality sleep, and can lead to drowsiness and fatigue and men over age 40 are primarily at risk.

Hormones may also play a role in human behavior after sex, though.

9 FOODS THAT CAN HELP BOOST YOUR SEX DRIVE NATURALLY

Much of the research surrounding hormonal interactions following sex involve animals, Dasgupta said, but compared to women, men do have more of the hormone prolactin, which secretes after ejaculation and leads to whats called a relative refractory period. That post-stimulated stage is what animal studies have linked with sleepiness.

Also after ejaculation, the stress hormone cortisol decreases, while oxytocin, the feel-good hormone, increases promoting relaxation. And, if youre in a dark room, the hormone melatonin, which promotes sleep, increases, further encouraging men to fall into slumber, Dasgupta said.

At nighttime, the hormone vasopressin, or ADH (antidiuretic hormone) is also secreted after ejaculation. This is the same hormone that prevents us from getting up at night to use the bathroom, Dasgupta said, and it, too, promotes relaxation.

These are hormones that have been associated with ejaculation at night, said Dasgupta, stressing that much of the research does not draw a causative relationship, just a link. They may be some of the answers to why males are more sleepy at night, but its always gonna be a combo of the social aspects and some of the hormones together.

THIS SWEDISH POLITICIAN THINKS WORKERS SHOULD GET PAID SEX BREAKS

Ian Kerner, a licensed psychotherapist and sex counselor, told Fox News the answer may simply lie in the fundamental differences between men and womens sexual response cycle.

When men become aroused, Kerner said, blood surges to the genitals and eventually leads to ejaculation. Upon ejaculation, that blood flows out and men go into the aforementioned refractory period. Some men, usually those who are young, can go back to having sex immediately, while older men may take days, weeks or month until theyre rearing to go again.

But the main thing is, after a man ejaculates, he returns to the pre-aroused state, Kerner explained, and the neurochemical firework festival of orgasm also produces sort of an effect of relaxation and sleepiness.

After women reach orgasm, on the other hand, it takes longer for their blood flow to leave the genitals, so they do not return as quickly as men to a pre-aroused state, Kerner said.

Instead, he explained, They go into a semi-aroused state, and thats why a lot of women have the capacity to experience multiple orgasms, and remain a little more connected, interested and partner-focused after sex.

Meanwhile during orgasm, pent-up muscular stress in men and women gets released, which may lead men and women to drift into a relaxing state. But its the difference in men and womens aroused states that sometimes causes men to appear sleepier than women, Kerner said.

EXPERTS SAY THIS IS THE BEST WAY TO FLIRT

That research, he pointed out, comes from researchers William Masters and Virginia Johnson, who studied 10,000 hours of male and female orgasms by watching couples in 1950s. They are best known for their research on the human sexual response, on which they published a book of the same name in 1966.

The hit Showtime production "Masters of Sex" is thought to be based on Masters and Johnson's lives.

And yet, despite what's known about how male and female sexual response cycles differ, Kerner said he has counseled couples where the woman will be the one more prone to falling asleep, or either sex taking issue with his or her partner checking email or jumping out of bed after doing the dirty deed.

So I think its gender-based, but its also relationship based, Kerner said.

Excerpt from:
The truth about why some men get sleepy after sex | Fox News - Fox News

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Alex Harris, Miami Herald (TNS) 3:00 p.m. CT Feb. 25, 2017

Diana Guevara, 28, touches up her makeup as she visits the new LGBTQ medical center at the University of Miami Miller School of Medicine on Tuesday, Feb. 7, 2017.(Photo: PATRICK FARRELL, TNS)

MIAMI She asked her mother for her new name.

Mom, when I was a baby, what would you have named me? If we could reverse all this and I was in your stomach again?

She got her answer: Diana Elizabeth Guevara.

It was the final touch on her new, true self. For everything else, the 28-year-old went to Dr. Christopher Salgado at the University of Miami, the lead surgeon at the hospitals new LGBTQ clinic.

Guevara used to identify as a male Miami-Dade police officer, until she injured herself and left the force. With time on her hands to think, Guevara realized what shed known since she was a third-grader: Shes a woman.

I knew I ticked differently, but I didnt know how, she said. Then I figured it out. Oh! Im trans.

Transgender people the T in LGBTQ are those born with sex organs that dont match their gender identities. Some trans people undergo complex (and expensive) surgeries to change their genitalia, facial features, vocal cords. Others choose to live out their true gender identities without altering their bodies.

For Guevara, changing herself physically was something so powerful and so important.

Word of mouth took her to Salgado, who had been performing the tricky surgeries for a few years by the time she saw him in 2012.

Diana Guevara, 28, gets a follow up exam by her Plastic Surgeon Dr. Christopher Salgado during a visit to the new LGBTQ medical center at the University of Miami Miller School of Medicine on Tuesday, Feb. 7, 2017.(Photo: PATRICK FARRELL, TNS)

Even five years ago the surgeries were much less common than they are today, Salgado said during a recent tour of the clinic.

He never saw a single case during his residency in the late 1990s, and when he decided to study the surgery in-depth, his best option was a fellowship in Taiwan.

Much has changed since then.

In 2014, Medicare began covering hormone therapy and sexual reassignment surgery, following the trend set by European insurance companies. With more insurance money available for the costly surgeries, Salgado saw more and more patients. At the same time, media celebrities such as Laverne Cox and Caitlin Jenner raised the profile of trans people in the United States. Equality for and understanding of LGBTQ people became a hot topic.

UM opened its LGBTQ center in January to serve the needs of the growing population. The new clinic brings together specialists in urology, endocrinology and psychiatry, as well as a team of surgeons to accompany the patient into the operating room.

Salgado and other doctors can even perform multiple surgeries on a patient simultaneously, so after a marathon session the patient can emerge with everything done at once.

The university also hired Lauren Foster, a high-profile Miami Beach trans model and activist, as UMs first director of LGBTQ concierge services.

Trans patients live their lives in stealth mode, Foster said, so the clinic focuses on privacy. A lot of patients dont want people to know why theyre here.

Diana Guevara 28, visits the new LGBTQ medical center at the University of Miami Miller School of Medicine on Tuesday, Feb. 7, 2017.(Photo: PATRICK FARRELL, TNS)

She said some wealthier patients, primarily from South America and Europe, have even used the hospitals rooftop helicopter pad as a discreet entrance and exit.

In its fledgling state, the clinic focuses primarily on trans patients, but thats rapidly changing.

Foster said other doctors in the hospital are reaching out and offering to integrate their services, like HIV tests, anal pap smears, or PrEP an HIV prevention treatment.

Dr. Wrood Kassira, who focuses on chest surgery and facial feminization, said the clinic sees about four new trans patients a week.

When she walks her patients through the surgery, she said they dont ask for drastic changes. They just want to be comfortable in their own skin.

They tell me, I dont want to be looked at funny. I dont want people to see me as the other gender, Kassira said.

But the power of gender affirmation surgery, as it is sometimes called, goes beyond that of just altering appearances. Research indicates the operation can drastically improve the mental health of trans people, who studies show are much more likely to attempt suicide than the general population.

Salgado likened the surgery to removing a cancerous tumor. Its life saving, he said.

Before the procedures, Guevara said, her life was emotionally draining. Even a quick trip to the mall left her on edge. But the day after her chest reconstruction, she looked in the mirror and cried tears of joy.

I was changing and being myself, she said. It doesnt seem like it should be such a big step, but it is.

Read or Share this story: http://www.gosanangelo.com/story/life/wellness/2017/02/25/these-transgender-patients-now-have-place-change-their-lives-all-once/98410294/

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These transgender patients now have a place to change their lives all at once - San Angelo Standard Times

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GETTY

A high-level meeting has paved the way for global trials to begin on hundreds of patients.

British scientists have found a way to use stem cells to repair damaged tissue which could help millions living with heart failure, the UKs leading cause of death.

Scarring due to disease or heart attacks affects more than two million people in Britain.

This would be the biggest breakthrough since the first transplants three decades ago

Professor Steve Westaby

Initial trials involving more than 100 patients are being planned for the autumn at two London hospitals.

World renowned cardiac surgeon Professor Steve Westaby, who helped pioneer the revolutionary technique, said it had been thought that repairing heart damage was impossible.

But results from a long-term trial that began in Greece five years ago have shown that this is not the case.

Preliminary data from this trial showed the engineered stem cells, known as Heartcel, can reverse scarring by up to 79 per cent.

The data, presented at the European Society of Cell and Gene Therapy in Florence, showed an average of 40 per cent reduction in heart damage in those on the treatment.

Last month researchers finalised talks with European and US regulators to discuss the timetable for global trials next year involving 500 people.

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6 early signs of a heart attack

Professor Westaby, from the John Radcliffe Hospital, Oxford, said: I am very excited at the prospect of a trial which will hopefully lead to the availability of this stem cell treatment to thousands of patients annually in the UK.

Other scientists have tried in vain to repair damaged heart muscle using stem cells over the past few decades.

This is the first time scarring has been shown to be reversible. It could herald an end to transplants and lead to a treatment for heart failure within three to five years.

GETTY

Professor Westaby said: This would be the biggest breakthrough since the first transplants three decades ago.

Professor Westaby has been working on the technique for more than a decade and is carrying out the study with Professor Kim Fox, head of the National Heart and Lung Institute, at Imperial College London.

The implanted stem cells were created by medical outfit Celixir, co-founded by Nobel laureate Professor Martin Evans, the first scientist to culture mice embryonic stem cells in a laboratory.

Professor Westaby was inspired to work on the breakthrough in 1999 after a four-month-old baby girls heart healed itself after he carried out a major life-saving operation.

Kirsty Collier, from Swindon, was dying of a serious and rare heart defect. In a last ditch effort Professor Westaby cut away a third of her badly damaged heart.

GETTY

GETTY

Surprisingly it began to beat. Fourteen years later a scan has shown that the heart had healed itself.

Now Kirsty, 18, has a normal one. Professor Westaby said: She was essentially dead and was only resurrected by what I regarded at the time as a completely bizarre operation.

The fact there was no sign of heart damage told me there were foetal stem cells in babies hearts that could remove scarring of heart muscle. That never happens in adults.

Its all down to the clues we got from Kirstys operation.

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Heart failure BREAKTHROUGH: Stem cells trial offers hope to millions - Express.co.uk

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February 24, 2017

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

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

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

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

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

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

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

Journal reference: PLoS ONE

Provided by: Morris Animal Foundation

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

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By Jacob Dykes

In Durham, a pioneering technology has been developed which is providing a solution to fundamental issues in tissue engineering and stem cell biology. The development of new innovative technology enables the advancement of the research and discovery process and scientific thinking as a whole. For example, its hard to conceive of a biomedical sphere untouched by the blessing of PCR or DNA sequencing. Technological advancements not only offer solutions to existing obstacles, they open up new avenues of research into previously inconceivable areas.

With the current levels of excitement in the research of stem cell biology, you could be forgiven for envisaging a utopian medical scenario where a process akin to science-fiction allows us to generate complex tissues in a Petri-dish, ready for transplantation into the damaged organism. The scientific community has speculated that the nature of stem cells, in their ability to self-renew and produce cell types of any lineage will eventually provide medical solutions to some of our most vilified tissue diseases.

Transitioning speculation to reality requires time, basic research and technology development. A novel product known as Alvetex has been developed by Reinnervate, a Durham University spin-out company, which enables a new routine approach to study stem cells and their ability to form tissues in the laboratory. The product unlocks the potential of stem cell differentiation by mimicking the natural three-dimensional (3D) microenvironment cells encounter in the body, enabling the formation of 3D tissue-like structures.

Cell behaviour, in general, is guided by the complex 3D microenvironment in which they reside. Dispersal of cell-cell interactions and architectural contacts across the surface of the cell are essential for regulating gene expression, the genetic mechanism by which cells change their character and behaviour. Recreation of this microenvironment in the laboratory is essential to studying physiologically relevant behaviour, and the differentiation process by which cells form new cell types. Alvetex is a micro-engineered 3D polystyrene scaffold into which cells can be impregnated for cultivation. Cells grow within a 200-micron thick membrane of the 3D material bathed in culture medium. The microenvironment enables cells to form 3D contacts with neighbouring cells, recreating the more natural interactions found in real tissues. Overall, this affects the structure and function of the cells, enabling them to behave more like their native counterparts, which in turn improves predictive accuracy when working with advanced cell culture models.

We can take progenitor cells from the skin of donors and produce human skin We can take cell lines from the intestine and reproduce the absorptive lining of the intestine. We can take neural progenitors and recapitulate 3D neural networks.

Stefan Przyborski is a Professor of Cell Technology at Durham University and the founder of Reinnervate. He gave us an insight into his technologys applications;

We can take progenitor cells from the skin of donors and produce a full-thickness stratified human skin model (see image). We can take cell lines from the intestine and reproduce the absorptive lining of the intestine. We can take neural progenitors and recapitulate 3D neural networks to simulate aspects of nervous system function. Each of these models can be used to advance basic research, and extend our understanding of tissue development, and simulate aspects of disease.

Such technology is underpinned by well established fundamental principles such as how cellular structure is related to function, which hails way back to Da Vinci himself. It is well known that if you get the structure and the anatomy correct than the physiology will start to follow.

Alvetex technology has already been used in research that has led the publication of over 60 research papers in the field of tissue engineering and cancer biology. One particular group used the technology to successfully test drugs to prevent glioblastoma dispersal, an innovative application in brain oncology. Another has developed a 3D skin model to better study the development of metastatic melanoma, a persistently incurable invasive tumour of the skin. US scientists have used Alvetex on the International Space Station to study the formation of bone tissue in microgravity conditions.

The technology promises to be a cost-effective and ethical solution to current obstacles in cell culturing methods, producing better quality data relevant to man and reducing the need for animal models. Alvetex technology has offered a generational contribution to the process of tissue engineering research, yet the founder has higher ambitions;

What I would like to see in the next few decades is the increased complexity of the tissues that stem cells can be used to generate. If you consider the structure of an organ, the complexity, arrangement and structural organisation of those cell populations, it is far from where we are today. Advances in technology at the interface between disciplines leads to new innovative ideas to solve problems and open up new opportunities.

The development of stem cell research is an incremental process. We have to remain cautious given the potential of stem cell therapy to cause tumour formation, highlighting the need for more stringent models and controls. However, the clinical transplantation of cultured stem cells in bone and cornea repair demonstrates their enormous potential. Laboratory experiments have also demonstrated the potential of stem cells to produce kidney, pancreatic, liver, cardiac and muscle cells. It is hoped that continued research using more physiologically relevant technologies will increase the complexity of these tissues in the lab, and the diversity of their application.

Innovative technological advances play an important role in the process of biomedical science. Scientists at Durham are instrumental in the development of such new technologies that enable the process of new discoveries.

Photograph: Prof Stefan Przyborski, Durham University

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Durham scientists pioneer innovative stem cell research - Palatinate

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