By 2027 to 2037 scientists will likely be able to create a baby from human skin cells that have been coaxed to grow into eggs and sperm and used to create embryos to implant in a womb.

The process, in vitro gametogenesis, or I.V.G., so far has been used only in mice. But stem cell biologists say it is only a matter of time before it could be used in human reproduction opening up mind-boggling possibilities.

With I.V.G., two men could have a baby that was biologically related to both of them, by using skin cells from one to make an egg that would be fertilized by sperm from the other. Women with fertility problems could have eggs made from their skin cells, rather than go through the lengthy and expensive process of stimulating their ovaries to retrieve their eggs.

IVF (Invitro fertilization) produces 70,000, or almost 2 percent, of the babies born in the United States each year. Worldwide there been more than 6.5 million babies born worldwide through I.V.F. and related technologies.

I.V.G. requires layers of complicated bioengineering. Scientists must first take adult skin cells other cells would work as well or better, but skin cells are the easiest to get and reprogram them to become embryonic stem cells capable of growing into different kinds of cells.

Then, the same kind of signaling factors that occur in nature are used to guide those stem cells to become eggs or sperm.

Last year, researchers in Japan, led by Katsuhiko Hayashi, used I.V.G. to make viable eggs from the skin cells of adult female mice, and produced embryos that were implanted into female mice, who then gave birth to healthy babies.

Nature Reconstitution in vitro of the entire cycle of the mouse female germ line

The female germ line undergoes a unique sequence of differentiation processes that confers totipotency to the egg. The reconstitution of these events in vitro using pluripotent stem cells is a key achievement in reproductive biology and regenerative medicine. Here we report successful reconstitution in vitro of the entire process of oogenesis from mouse pluripotent stem cells. Fully potent mature oocytes were generated in culture from embryonic stem cells and from induced pluripotent stem cells derived from both embryonic fibroblasts and adult tail tip fibroblasts. Moreover, pluripotent stem cell lines were re-derived from the eggs that were generated in vitro, thereby reconstituting the full female germline cycle in a dish. This culture system will provide a platform for elucidating the molecular mechanisms underlying totipotency and the production of oocytes of other mammalian species in culture.

Scientists could make an egg out of skin cells from women who cant produce viable eggsor who have other fertility problems, or who dont want to go through the difficult process of surgical removal of their eggs for IVF. Or men with fertility problems involving their sperm. Two women could make a child that was truly theirs, with eggs from one and sperm made from skin cells of the other. Or two men, vice-versa.

Mouse oocytes created from embryonic stem cells. Credit: Katsuhiko Hayashi, Kyushu Univ

In a couple of decades, Greely predicts, it will be possible to examine and select an embryo not just for a particular genetic disease but also for other traits, ranging from hair color to musical ability to potential temperament.

Greely concedes that Easy PGD will be mostly available in rich countries, but he also thinks it will be widely available in those countries because it will be free. Preventing the birth of people with genes that increase their risk of serious (and expensive) disease will save health care systems so much money that Easy PGD will be convincingly cost-effective.

That will be a powerful incentive to encourage prospective parents to further decouple procreation from sexual intercourse, and make it easy for them to drop off their skin cells at a lab. The lab will then generate a big supply of embryos containing the couples genes, embryos that can be examined for desirable characteristics as well as disease genes. The winner of this elimination contest will, presumably, be selected for implantation.

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Mice embryos from skin cells and by 2037 human embryos from skin cells - Next Big Future

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After 20 years of trying, scientists have transformed mature cells into primordial blood cells that regenerate themselves and the components of blood. The work, described [May 17] in Nature offers hope to people with leukemia and other blood disorders who need bone-marrow transplants but cant find a compatible donor. If the findings translate into the clinic, these patients could receive lab-grown versions of their own healthy cells.

One team, led by stem-cell biologist George Daley of Boston Childrens Hospital in Massachusetts, created human cells that act like blood stem cells, although they are not identical to those found in nature. A second team, led by stem-cell biologist Shahin Rafii of Weill Cornell Medical College in New York City, turned mature cells from mice into fully fledged blood stem cells.

Time will determine which approach succeeds. But the latest advances have buoyed the spirits of researchers who have been frustrated by their inability to generate blood stem cells from iPS cells. A lot of people have become jaded, saying that these cells dont exist in nature and you cant just push them into becoming anything else, [Mick Bhatia, a stem-cell researcher at McMaster University, who was not involved with either study] says.

[Read the Daley study here.]

Read the Rafii study here.]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Lab-grown blood stem cells produced at last

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Breakthrough for bone marrow transplant recipients: Lab-grown blood stem cells produced for first time - Genetic Literacy Project

Bone Marrow Stem Cells Comments Off on Breakthrough for bone marrow transplant recipients: Lab-grown blood stem cells produced for first time – Genetic Literacy Project | May 22nd, 2017

He was tired of the daily pain that made even shaking someone's hand almost unbearable.

Marlette lost his arm in an accident when he was a teenager, but as an active kid, he didn't this slow him down. He continued to play football and golf, running track and even wrestling.

But over time, the strain on his remaining arm and wrist took a toll.

So to relieve his pain, he traveled from Sioux Falls, South Dakota, to Munich, Germany, with the hopes that a special procedure using stem cells could make a difference.

"There's no cartilage," Marlette said of his wrist. "I'm bone-on-bone. It is constantly inflamed and very sore."

As Marlette grew older, even the simplest things, like tucking in his shirt or putting on a jacket, became incredibly painful.

Marlette developed cysts and holes in the bones of his wrist. Doctors prescribed anti-inflammatory medications, but they only managed the pain, doing nothing to actually heal the problem. One day, his doctor, Dr. Bob Van Demark at Sanford Health in South Dakota, where Marlette works in finance, saw a presentation by Dr. Eckhard Alt.

It was about a new treatment using stem cells.

"Following an infection or wound or trauma," Alt said, "there comes a call to the stem cells in the blood vessels, which are silent, and nature activates those cells."

Stem cells are located throughout our bodies, like a reserve army offering regeneration and repair. When we're injured or sick, our stem cells divide and create new cells to replace those that are damaged or killed. Depending on where the cells are in the body, they adapt, becoming specialized as blood cells, muscle cells or brain cells, for example.

Alt was the first person to use adipose tissue, or fat, as a prime source of stem cells, according to Dr. David Pearce, executive vice president for research at Sanford health.

"He observed that the simplest place to get some stem cells is really from the fat," said Pearce. "Most of us could give some fat up, and those stem cells don't have to be programmed in any way, but if you put in the right environment, they will naturally turn into what the cell type around them is."

Fat tissue has a lot of blood vessels, making it a prime source of stem cells, and Alt recognized that stem cells derived from adipose tissue are also particularly good at becoming cartilage and bone.

Bone marrow is another source of stem cells, but these easily turn into blood and immune cells. Stem cells from fat have another fate.

"Fat-derived stem cells have a different lineage they can turn into, that is really cartilage and bone and other sort of connective tissues," said Pearce.

Van Demark traveled to Alt's Munich clinic along with some doctors from Sanford, which is now partnering with Alt on clinical trials in the United States. Marlette's doctor was impressed with what he saw and recommended the treatment to his patient.

Marlette paid his own way to Munich, where he would receive an injection of stem cells from his own fat tissue.

"I had one treatment, and my wrist felt better almost within the next couple weeks," Marlette said. "Through the course of the next seven months, it continued to feel better and better."

One injection was enough for this ongoing improvement.

"We see (from an MRI scan) that those cysts are gone, the bone has restructured, the inflammation is gone, and he formed ... new cartilage," said Alt.

MRIs confirmed what he was feeling: The cartilage had begun to regenerate in his wrist. Because the procedure uses autologous cells, which are cells from the patient's own body, there's little to no chance of rejection by the body's immune system.

Though the procedure worked for Marlette, the use of stem cells as a form of treatment is not without controversy or risk. In the US, they have been mired in controversy because much of the early research and discussion has been centered around embryonic and fetal stem cells.

Marlette traveled to Germany because approved treatments like this are not available in the United States. Clinics have popped up across the country, but they lack oversight from the Food and Drug Administration.

Dr. Robin Smith, founder of the Stem for Life Foundation, first began working in this field 10 years ago. According to Smith, there were 400 clinical trials for stem cells when she first started; now, there are 4,500. She partnered with the Vatican to hold a stem cell conference last year.

"We're moving toward a new era in medicine," said Smith, who was not involved in this research. "(We are) recognizing cells in our body and immune system can be used in some way -- manipulated, redirected or changed at the DNA level -- to impact health and cure disease. It is an exciting time."

Dr. Nick Boulis is a neurosurgeon with Emory University in Atlanta. His team ran the first FDA-approved clinical trials in the US to inject stem cells in the spinal cords of patients with ALS, better known as Lou Gehrig's disease, and he isn't surprised to see procedures like the one at Alt's clinic in Germany have success.

"Joints and bones heal," Boulis said. "The nervous system is very bad at healing. It doesn't surprise me that we're seeing successes in recapitulating cartilage before we're seeing successes in rebuilding the motherboard."

Smith also cautioned patients to do their research, especially about the types of cells being used. "When you have a health problem, and you need a solution, sometimes you don't have three five, seven years to get there," she said, referencing the slow progression of regulations in places like the United States.

"So really ,look for places that have the regulatory approval of the country they're in. Safety has to be number one," she said.

Alt's Munich clinic was approved by the European equivalent of the FDA, the European Medicines Agency. Through the partnership with Sanford, the health group is now launching clinical trials in America, focusing on rotator cuff injuries, a common shoulder injury. This is the first FDA-approved trial of its kind.

Further down the line, Alt hopes to see stem cells used for such issues as heart procedures and treating the pancreas to help diabetics. For him, the growth is limitless.

"I think it will be exponential," he said. "It will be the same thing (we saw) with deciphering the human genome. The knowledge will go up exponentially, and the cost will go exponentially down. For me, the most exciting thing is to see how you can help patients that have been desperate for which there was no other option, no hope, and how well they do."

For Marlette, it has meant a wrist free from pain and a life free from pain medication.

Since the procedure in August, he hasn't taken any of the anti-inflammatory drugs. "I have more range of motion with my wrist, shaking hands didn't hurt anymore," he said. "My wrist seems to continue to improve, and there's less and less pain all the time."

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Patient uses fat stem cells to repair his wrist - CNN

Bone Marrow Stem Cells Comments Off on Patient uses fat stem cells to repair his wrist – CNN | May 22nd, 2017

BENGLURU: Fateh Singh, a six-year-old thalassemia major patient from Amritsar, underwent a bone marrow transplant last May which gave him a new lease of life. A year later, the boy met his saviour, Naval Chaudhary, whose stem cells were used for the procedure. The child was diagnosed with the condition when he was one-and-a-half years old.

On Thursday, the donor and recipient met for the first time. Naval, 28, a professional living in Bengaluru, had registered with DATRI, an unrelated blood stem cell donors registry in 2015. He said: "I was very happy to hear I was a potential match for a patient. But then I was told the donation process had to be done through bone marrow harvesting. Initially, I was a tad hesitant but then I researched the procedure and was counselled by Dr Sunil Bhat, paediatric haemato-oncologist from Mazumdar Shaw Cancer Centre."

"I realized that saving a life is more important than the type of procedure I had to go through. So I decided to go ahead," he added.

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6-year-old thalassemia patient from Punjab meets his stem cell ... - Times of India

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In 2004 California ballot measure Proposition 71 was passed, granting $3 billion ($6 billion including interest) in state funds to support politically controversial embryonic stem cell research in California at a time when the federal government was restricting this research. A public agency was established, the California Institute for Regenerative Medicine, to dole out this money across California universities, medical research institutions and biotech companies. During the election campaign, California voters were assured of breakthroughs and cures for conditions like Parkinsons and spinal cord paralysis through celebrity endorsements featuring actors, Nobel prize winners and other notables. Prop. 71 money is dwindling and there is talk about putting a $5 billion renewal initiative on the ballot. So its reasonable to ask what California taxpayers got out of this deal over the past 13 years. Sadly, CIRM hasnt generated a single approved medical treatment. Through September 2016, CIRM has funded only three stem cell research projects that have reached Phase 3 clinical trials (the final step before FDA marketing approval). One of these trials was terminated and the other two are still recruiting patients and are not expected to report out for several years. During the same time, despite embryonic stem cell research restrictions, the federal National Institutes of Health has funded 50 stem cell research projects in Phase 3 trials. The NIH cost per Phase 3 research trial has been five times lower than the state program. Nearly half of the state funding has gone to research infrastructure rather than to actual research.

There also appears to have been blatant conflicts of interest in CIRM research awards. Around 80 percent of CIRM grants have gone to institutions represented on its board of directors. One out of seven CIRM research dollars has gone to Stanford University. One awardee, StemCells Inc., was co-founded by Irving Weissman, Stanfords stem cell program director. StemCells received at least $40 million from CIRM before going belly up. The CIRM board initially turned down a $20 million funding proposal to StemCells, until Bob Klein, the Northern California real estate investor who drafted Prop. 71 and was the first chairman of CIRMs governing board, was reported to have pressured the board to reverse that decision. CIRMs President Alan Trounson abruptly resigned in October 2013, joined the board of StemCells one week later, and then received $435,000 in cash and stocks from them before the company folded last year.

Does it make sense for California taxpayers to fund biotechnology research? Perhaps. A good case can be made that public investments in basic biotechnology infrastructure can have enormous benefits to Californias economy and job growth while generating significant improvements in human health. But public funding should have broader scope and flexibility to go after all promising new technological advances, not just current scientific fads or political controversies. Public funds should be awarded with rigorous oversight and accountability. There should be a sharp line between basic research, which requires public funding and is unlikely to yield short-term tangible cures, despite what celebrity actors say, and getting new medicines to market. Promising new treatments are already well-funded through private venture capital funds and biotech companies, who are much better at picking winners and losers than California taxpayers.

By not providing adequate oversight over potential conflicts of interest and not holding CIRM funding recipients to the same rigorous standards as NIH grant recipients, CIRMs 13 year record of zero new medicines for $6 billion in taxpayer funds is not an experiment that the voters should regenerate at the ballot box.

Joel W. Hay is a professor of Health Economics and Policy at the University of Southern California.

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Regenerating medical research payouts? - OCRegister

Spinal Cord Stem Cells Comments Off on Regenerating medical research payouts? – OCRegister | May 19th, 2017

On the morning of a game at Youngstown State earlier this season, Illinois-Chicago softball pitcher Karissa Frazier arrived armed for a successful road trip.

Frazier packed enough kits to perform cheek swabs on Youngstown State players who had agreed to add their samples to a worldwide bone-marrow registry. So Frazier hopped on a bus to the ballpark by herself, gave a presentation on the #swab2save campaign in her role as UIC's campus ambassador for Gift of Life, and helped swab the young women she would try to strike out later in the series.

And what a pitch the All-Horizon League hurler made.

One Youngstown State player was inspired enough by Frazier to request 200 swab kits to begin her own drive. Another immediately reached out to Gift of Life the global not-for-profit marrow and blood stem cell donor registry facilitating transplants and became the Ohio campus's representative.

"After Karissa was done swabbing players that day, she came back to our hotel and got ready for the game like normal," UIC coach Lynn Curylo said. "How amazing is that?"

For the UIC softball team, the trip to Eugene, Ore., to play Oregon on Friday in the NCAA tournament, its first appearance in six years, offers an opportunity to provide evidence of progress at the end of Curylo's promising first season. For Frazier, a junior right-hander with a 13-8 record and a 1.53 earned-run average, the journey represents that and more, another chance to spread awareness of a cause as powerful as her fastball.

"This has pushed me in the right direction and opened my eyes to all the things I could do to change people's lives for the better," Frazier said. "I'm hoping to swab all three teams at our NCAA regional. And I'd love to go to the College World Series and swab all the teams there."

Seeing an emotional meeting between a donor and recipient left an indelible impression on Frazier. But a brush with a family friend back home in Temecula, Calif., first lit a fire within the public health major. A friend's decision to become a bone-marrow donor allowed a woman to live an additional six years and see the birth of her first grandchild and the wedding of her daughter.

"I just knew this was something I'd really enjoy doing so one day I could help save someone's life,'' Frazier said.

Back at UIC last August, Frazier interviewed with Gift of Life, which sought college ambassadors to increase potential donors in the 18- to 25-year-old demographic. Frazier's bosses established two goals for her: Swab 500 people overall and 250 males research shows males are three times less likely to sign up than women but twice as likely to be a match. When Frazier left Wednesday for Oregon, she had accumulated more than 700 total swab samples, including nearly 300 from males.

"I used my softball player status to expand getting a broader range of people," Frazier said.

Last fall, Frazier set up a table next to the UIC ticket booth and attended more sporting events than Sparky the mascot. As people passed by, Frazier did her best to demystify the swabbing process.

"I tell people it's easy and if you're willing to take three to five minutes, you could save somebody's life," Frazier said.

Those who say yes start by taking a health survey on their smartphones. Then Frazier gives participants a kit that includes four Q-tips, each to be rubbed on the inside of the corners of a person's mouth. The samples are sealed in the kit, the person's name goes on a label, and the registry grows. It's that simple.

"A lot of people think the process is super scary, but I just explain there's only one in 500 chance of being a match for someone and, if you are a match, then 80 percent of the time you just donate peripheral stem cells via regular blood draw," Frazier said. "And 20 percent of the time, they take bone marrow from your hip. But for the rest of your life, you can say you literally saved someone's life."

Curylo not only encouraged her star pitcher to pursue her passion, even if that meant traveling to Tinley Park on some game days to get swabs from visiting teams, but challenged Frazier to think bigger. It was Curylo's idea to swab every team in the Horizon League, which created the unintended consequence of camaraderie.

"This brought teams in our conference together," said Curylo, the conference coach of the year. "We usually go to games, compete, get on our bus and go home. But after we beat Oakland, we hung out and talked because we were all helping Karissa. She's finding a way to make herself matter off the field as much as she does on it."

She's a college student attacking leukemia and lymphoma as fiercely as she does hitters, a young woman hoping to change the world with the Peace Corps after making it better at UIC.

"What's amazing is Karissa is so completely different as a person than she is as a pitcher," Curylo said. "Pitching, she's poker-faced, no emotion, gets the job done. But away from that, she's one of the sweetest, most giving, best teammates around. She has two sides."

You might say they're a perfect match.

dhaugh@chicagotribune.com

Twitter @DavidHaugh

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NCAA-bound UIC softball pitcher driven to expand bone-marrow donor pool - Chicago Tribune

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Nick Wells, CTVNews.ca Published Wednesday, May 17, 2017 7:04AM EDT Last Updated Wednesday, May 17, 2017 12:28PM EDT

An Ottawa-area boy who suffers from a rare and painful blistering skin disease is recovering in a Minneapolis hospital, after undergoing a second potentially life-changing transplant.

Jonathan Pitre, known as the "Butterfly Boy" because of his delicate, blistering skin, received a second transfusion of his mother Tina Boileaus stem cells in April.

In a Facebook post Tuesday, Boileau said the donor study tests are showing that her son is officially growing her cells.

Pitre was born with a severe form of epidermolysis bullosa (EB), an incurable genetic collagen disorder. The condition causes a never-ending series of raw and painful blisters -- some of which hes had for years.

His mother told CTV News on Wednesday that the positive turn in Pitres long and painful treatment was exactly what we needed.

Boileau said her son has had infections on top of infections and endured much pain over the past year. The second stem cell transplant has been really hard on his body, she said, but there now seems to be light at the end of the tunnel.

Yesterday was just the greatest day. We were speechless. Jonathan hugged me and we were like, We did it, she said in an interview from the hospital.

Boileau said that even some of the nurses were crying when Pitre received the good news.

Its finally now feeling like its all been worth it.

However, she pointed out that if Pitre is unable to grow his own cells, he could be diagnosed with Graft vs. Host disease a condition where the donor's cells take over the host's organs and bodily functions, leading to complications.

We still have a long road ahead of us, but you know what, this is definitely what weve been waiting for, Boileau said.

The $1.5-million transplant procedure Pitre is undergoing is currently only performed as a University of Minnesota clinical trial.

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Encouraging results after Jonathan Pitre's transplant, mother says - CTV News

Skin Stem Cells Comments Off on Encouraging results after Jonathan Pitre’s transplant, mother says – CTV News | May 18th, 2017

Ella1977/DreamstimeIn the not too distant future most human babies will be born using eggs and sperm produced from the skin cells of their parents, claims Stanford University law professor and bioethicist Hank Greely in his book, The End of Sex and the Future of Human Reproduction. Basically, Greely is making informed speculation how in vitro gametogenesis (IVG) will progress over the next few years. And considerable progress has been made.

For example, Japanese researchers have turned skin cells from mice into eggs which they fertilized to produce embryos that were implanted into surrogates that then gave birth to healthy mouse pups. In April, Spanish researchers announced that they had made significant progress toward transforming human skin cells into viable sperm.

Harvard bioethicist Glenn Cohen and his colleagues described how "disruptive reproductive technologies" derived from IVG might evolve in a January article in the journal Science Translational Medicine. They go on to assert that "IVG raises vexing ethical and social policy challenges in need of redress."

First let's consider the biomedical benefits of IVG. One result would be the creation of an unlimited supply of early-stage embryos for research. In the reproductive realm, IVG could produce sperm or eggs for people suffering from various forms of infertilty, e.g., congenital and chemotherapy-induced. In addition, IVG could be used to prevent mitochondrial diseases by creating eggs without those mutations and boost regenerative medicine by creating patient-specific stem cell lines that could be used as transplants to replace diseased tissues and organs.

More speculatively, IVG could be used by same sex couples to produce genetically related children. In addition, since skin cells could be used to produce both sperm and eggs, they might be used to create single-parent children (Women wanting a boy would have to find a donated Y-chromosome.) In addition, there is the possibility that someone lift some cells left behind on a glass or comb by a celebrity and turn them into gametes without their permission. Furthermore, the ability to produce unlimited quantities of gametes and embryos will make it feasible to use genome-editing techniques to correct genetic defects and, perhaps, eventually introduce gene variants that could enhance physical and mental functioning.

Glenn and his colleagues observe that some religious believers object to the creation of embryos outside of human bodies and that doctrinaire anti-market folks oppose the "commodification" of human reproduction. Certainly, opponents are entitled to their opinions, but there is no ethical reason why their beliefs should be permitted to interfere with the biomedical and reproductive choices of those who don't agree with them.

Safety concerns will be paramount before rolling out this technology. With regard to reproduction, one benchmark might be that the likelihood of producing birth defects in babies using IVG is no greater than IVF. As I explained in my Designer Babies and Human Enhancement lecture in Moscow:

Greely believes that in about 40 years half of all American babies will born using what he calls Easy PGD. At that time most people will use gametes produced from their skin cells to create scores of IVF embryos that will each have his or her entire genomes sequenced. Prospective parents will then choose among the embryos based on which combination of genetic traits they would prefer. Presumably they would tend avoid those embryos afflicted with debilitating genetic diseases.

Greely believes that Easy PGD will be extremely cheap, e.g., whole genome testing should fall to around $10 by the beginning of the next decade. Easy PGD would also make it possible for same sex couples to have offspring genetically related to both parents and it might even be possible for a person to have both sperm and eggs created from their skin cells, enabling them to be both mother and father of their child.

Interestingly, biologist Craig Venter, the leader of the group that raced the government to a tie in sequencing the human genome, and now founder of the life extension company Human Longevity, Inc. can sequence a fetal genome and give the mother "a picture of what her future child will look like at 18."

"There is a yuck factor here," said Arthur Caplan, a bioethicist at New York University in The New York Times today. "It strikes many people as intuitively yucky to have three parents, or to make a baby without starting from an egg and sperm. But then again, it used to be that people thought blood transfusions were yucky, or putting pig valves in human hearts." Just so.

Naturally, Glenn and his colleagues call for a vigorous ethical debate and government regulation of the technologies. I would gently suggest that a front page article in the Times means that a vigorous public debate is already taking place.

With regard to government regulation - there may be a role for it to the extent that safety issues cannot be handled by developers of the technology. However, the government should certainly stay far, far away from any eugenic efforts to tell people when and what sort of children they may have. The last time the U.S. government started meddling with the reproductive decisions of Americans, it didn't turn out well.

For more background, see my article, "Is Heaven Populated Chiefly by the Souls of Embryos?"

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Skin Cells Into Babies: Bioethicists Freakout Again - Reason (blog)

Skin Stem Cells Comments Off on Skin Cells Into Babies: Bioethicists Freakout Again – Reason (blog) | May 18th, 2017

Brain balls sound like something straight out of a Tim Burton movie: starting as stem cells harvested from patients, they eventually develop into masses of living neurons, jumbled together in misshapen blobs.

Just like the developing brain, these neurons stretch and grow, reaching out skinny branches that grab onto others to form synapsesjunctions where one neuron talks with the next.

And they do talk: previous attempts at growing these brain organoids found that they spark with electrical activity, much like the webs of neurons inside our heads that lead to thoughts and memories.

Theyre creepy. Theyre fascinating. And they may be neuroscientists best bet at modeling developmental disorders like autism in a dish.

Last week, two studies published in the prestigious journal Nature argued for brain balls as a reductionist model for broken brains. In one study, scientists took skin cells from patients with Timothy syndrome, a devastating neurodevelopmental disorder that often ends with childhood death, and grew them into brain balls to study where and how the developing brain veered off track.

In a separate paper, researchers used cutting-edge technology to profile the inhabitants of brain balls as they matured for eight months in a dish. Heres a creepy teaser: some blobs contained retinal neurons that normally allow us to see. Brain balls with eyes?!

As bizarre as that sounds, the fact that brain balls can develop a variety of neuron types with densely packed synapses is a win. Because theyre made from human cells, brain balls may eventually mimic diseases like schizophrenia, autism, or Alzheimers better than mouse models, revealing what went wrong and offering ample test grounds for potential treatments.

Weve never been able to recapitulate these human-brain developmental events in a dish before, says Dr. Sergiu Pasca at Stanford, who led the Timothy syndrome study. Our method lets us see the entire movie, not just snapshots.

Brain balls, better known by their scientific name cerebral organoids, first came onto the neurodevelopmental scene in 2013.

They often begin their short life as run-of-the-mill skin cells. Scientists first transform them back into stem cells. Then, using a chemical concoction of nutrients and signaling molecules, the stem cells are pushed to spontaneously assemble into little Frankenstein blobs of brain tissue.

But the process isnt just random bursts of division and growth. Rather, the way the brain balls mature roughly echoes how a fetuss cortex develops in the womb: the outer edges curl inward, forming outer and deeper layers.

What really sparked scientists interest was this: almost 90 percent of the neurons within a brain ball had active synapses, often spontaneously shooting electrical pulses to others in their network. While scientists believe brain balls arent capable of thinkingthe high-level cognitive processes constantly churning in our headstheyre definitely doing something.

To begin getting some answers, Dr. Paola Arlotta and team at Harvard followed a number of brain balls for nine months as they gradually maturedroughly the amount of time for human gestation, and much longer than any previous attempts.

Periodically, the researchers harvested more than 80,000 brain balls and ran sophisticated genetic tests to figure out their gene expression profile. Like law enforcement using DNA to match a perpetrators identity, this allowed researchers to profile the inhabitants of the organoids.

It was a cellular bonanza: as expected, excitatory neurons and non-neuronal cells called glia both made an appearance. More surprising were inhibitory neurons that dampen network activity, and cells that normally form the corpus callosum, a highway that connects the brains two hemispheres.

But creepiest by far, every single type of retinal cell also made an appearance. Although they couldnt really see in the normal sense, when bathed under light they did fire off electrical signals.

Just like a developing brain, the older they got the more complex the brain balls became. At eight months old, they contained roughly the same density of synapses as a human fetus cortex.

The cells connect witheach other, forming circuits, and once theyre connected, they can synchronize their activity, potentially mimicking higher-order functions of the human brain, says Arlotta.

Thats great, because it means mini brains could be used to study how different types of neurons connect with each other, and how disrupting the process leads to developmental problems.

Thats the direction the second study took. Rather than letting the mini brains grow wild, Pasca and team at Stanford tweaked the protocol to force them into different identities.

As a fetus brain grows, it gradually separates into an outer layer chock full of excitatory neurons, and an inner sanctum where inhibitory neurons reside. A big part of brain wiring is inhibitory neurons reaching out towards the surface and hooking up with their respective partners.

Starting from skin cells collected from patients with Timothy disease, the scientists used distinct chemical concoctions to form two batches of brain balls, each roughly 1/16 of an inch across and containing one million cells. One batch contained mostly inhibitory neurons, mimicking deeper brain regions, whereas the other modeled the cortex.

The spheroid cells were remarkably similar to those from corresponding regions of the human fetal brain, says Dr. J. Gray Camp and Dr. Barbara Treutlein at the Max Planck Institute in Germany, who were not involved in the studies.

The team then stuck the two types of brain blobs together into the same dish, and as expected, the inhibitory ball started nudging its way into the cortical one, until the two fused together.

As it turns out, the inhibitory neurons from Timothy patients were terrible migrants. Rather than smoothly slithering their way into the mesh of excitatory partners, they stuttered, stopped, but somehow ended up much further than theyre supposed to go, as if making up for their inefficiency.

The problem seemed to be the faulty neurons themselves, rather than defective signals from the environment. When researchers fused a Timothy inhibitory ball with a healthy excitatory one, they still fumbled without heads or tails.

But surprisingly, when treated with a chemical normally used for high blood pressure, the Timothy balls calmed down and migrated normally.

Spheroids are opening up new windows through which we can view the normal development of the fetal human brain, says Pasca. More importantly, it will help us see how this goes awry in individual patients.

While the scientists dont know whether the same drug could help babies with Timothy after theyre bornand their basic brain wiring already establishedPasca hopes that there may be a window of opportunity later on in life to correct the misguided migration.

All said, brain balls are an extremely reductionist model of the human brain. Although its hard to say whether the root of Timothy disease is faulty inhibitory neuron migration, its a great place to start looking for answers.

Pasca is rushing to speed up the process of growing spheroids, hoping to develop a giant depository harvested from many patients to screen for drugs that steers them towards a normal developmental path.

Others are a bit more cautious. These new studies show that brain balls whipped up from the same patient or patients with the same disease can express very different genes, warned Camp and Treutlein. The problem is likely more prominent in neurodevelopmental disorders like autism, in which the cause is a lot more heterogeneous.

But the fact that brain organoids behave like actual brains on several fundamental functionsmaking connections, spontaneously firing, responsive to external cuesis promising, so much so that theyre sparking intense ethical debates. Can they eventually see or think? Do they feel? Will consciousness spontaneously emerge without us detecting it?

For now, the mini brains are simply too tiny for higher-level thinking. Only time will tell what theyll eventually become, and how much information these mini brains can provide, says Camp and Treutlein.

Image Credit:PascaLab

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Bizarre Mini Brains Offer a Fascinating New Look at the Brain - Singularity Hub

Skin Stem Cells Comments Off on Bizarre Mini Brains Offer a Fascinating New Look at the Brain – Singularity Hub | May 16th, 2017

Cardiovascular disease is the number one cause of death worldwide in men, women and children, claiming more than 17 million lives each year. The effects of congestive heart failure and acute myocardial infarction (heart attack) present great challenges for doctors and researchers alike.

In this section:

Heart attacks cause damage to the heart muscle, making it less efficient at pumping blood throughout the circulatory system.

Your heart is constructed of several types of cells. For mending damaged heart tissue, researchers generally focus on three specific heart cell types:

Gladstone Institutes. Close up of a mouse heart stained to reveal the important structural protein that helps heart muscle cells to contract (red). The cell nuclei are labeled in magenta.

Despite major advances in how heart disease is managed, heart disease is progressive. Once heart cells are damaged, they cannot be replaced efficiently, at least not as we understand the heart today.

There is evidence that the heart has some repair capability, but that ability is limited and not yet well understood.

Heart failure is a general term to describe a condition in which the hearts blood-pumping action is weaker than normal. How much weaker varies widely from person to person, but the weakness typically gets worse over time. Blood circulates more slowly, pressure in the heart increases, and the heart is unable to pump enough oxygen and other nutrients to the rest of the body. To compensate, the chambers of the heart may stretch to hold more blood, or the walls of the chambers may thicken and become stiff. Eventually, the kidneys respond to the weaker blood-pumping action by retaining more water and salt, and fluid can build up in the arms, legs, ankles, feet, and even around the lungs. This general clinical picture is called congestive heart failure.

Many conditions can lead to congestive heart failure. Among the most common are:

The American Heart Association defines normal blood pressure for an adult as 120/80 or lower. What do those numbers mean? The top number is the systolic pressure that is, the pressure in your arteries when your heart beats, or contracts. The bottom number measures diastolic pressure, or the pressure in your arteries between beats, when the heart refills with blood.

In the early stages of congestive heart failure, treatment focuses on lifestyle changes (healthy diet, regular exercise, quitting smoking, etc.) and specific medications; the goals are to slow down any progression of the disease, lessen symptoms and improve quality of life.

Medications called beta blockers are often prescribed after a heart attack or to treat high blood pressure. Other medications called ACE inhibitors prevent heart failure from progressing.

For moderate to severe congestive heart failure, surgery may be necessary to repair or replace heart valves or to bypass coronary arteries with grafts. In severe cases, patients may be put on fluid and salt restriction and/or have pacemakers or defibrillators implanted to control heart rhythms.

Acute myocardial infarction, or a heart attack, occurs when the blood vessels that feed the heart are blocked, often by a blood clot that forms on top of the blockage. The blockage is a build-up of plaque that is composed of fat, cholesterol, calcium and other elements found in the blood. Without oxygen and other nutrients from the blood, heart cells die, and large swaths of heart tissue are damaged.

After a heart attack, scar tissue often forms over the damaged part of the heart muscle, and this scar tissue impairs the hearts ability to keep beating normally and pumping blood efficiently. The heart ends up working harder, which weakens the remaining healthy sections of the heart; over time, the patient experiences more heart-related health issues.

Doctors often use a procedure called angioplasty to disrupt the blood clot and widen clogged arteries. Angioplasty involves inserting and inflating a tiny balloon into the affected artery. Sometimes this temporary measure is enough to restore blood flow. However, angioplasty is often combined with the insertion of a small wire mesh tube called a stent, which helps keep the artery open and reduces the chances that it will get blocked again.

Other post-heart attack treatments include the regular use of blood thinners (for example, low-dose aspirin) to prevent new clots from forming and other medications to help control blood pressure and blood cholesterol levels. Lifestyle changes, such as lowering salt and fat intake, exercising regularly, reducing alcohol consumption and quitting smoking are also recommended to reduce the chances of a subsequent heart attack.

Scientists and clinicians have long suspected and recently confirmed that a persons genetic makeup contributes to the likelihood of their having a heart attack. Learn more here

The goals of heart disease research are to understand in greater detail what happens in heart disease and why, and to find ways to prevent damage or to repair or replace damaged heart tissue. Scientists have learned much about how the heart works and the roles different cells play in both normal function and in disease, and they are learning more about how cardiomyocytes and cardiac pacemaker cells operate, including how they communicate with each other and how they behave when damage occurs.

Researchers grow cardiomyocytes in the lab from the following sources:

These cells will beat in unison in a culture dish, the same way they do in a living heart muscle. This is exciting to consider, as researchers explore whether they might someday grow replacement tissue for transplantation into patients. However, it is not yet known whether lab-grown cardiomyocytes will integrate or beat in unison with surrounding cells if they are transplanted into the human body.

Gordon Keller Lab. Heart cells beating in a culture dish.

Scientists also use various types of stem cells to study the hearts natural repair mechanisms and test ways to enhance those repair functions. The evidence we have so far suggest thats the heart may have a limited number of cardiac stem cells that may conduct some repair and replacement functions throughout an individuals life, but we dont know where they live in the heart or how they become activated.

Human cells made from iPS cells are also incredibly useful for creating human models of heart disease to get a better understanding of exactly what goes wrong and for testing different drugs or other treatments. They can also be used to help predict which patients might have toxic cardiac side effects from drugs for other diseases such as cancer.

The key to treating heart disease is finding a way to undo the damage to the heart. Researchers are trying several tactics with stem cells to repair or replace the damaged heart tissue caused by congestive heart failure and heart attacks.

Areas under investigation include:

The Europe-wide BAMI clinical trial (the effect of intracoronary reinfusion of bone marrow-derived mononuclear cells on all-cause mortality in acute myocardial infarction) that began in 2014, is testing the infusion of cells from the participants bone marrow into one of the coronary arteries (one of two major arteries that supply the heart) to spark repair activity. However, it is not yet clear whether these cells will support heart repair function or in what way.

Researchers are also exploring transplantation of cardiomyocytes generated from both iPS cells and cardiac progenitor cells. They need to determine whether these transplanted cells survive and function in the body and whether they help speed up the hearts innate repair mechanisms.

Some of these approaches are still being evaluated in the lab while others are already being tested in clinical trials around the world. However, these trials are in their early stages and the results will not be clear for many years. Indeed, some published data conflict in critical ways, so carefully designed and well-monitored trials are key to working out what is safe and effective.

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Heart disease on Stem Cells - ISSCR

Cardiac Stem Cells Comments Off on Heart disease on Stem Cells – ISSCR | May 15th, 2017

Stem cell transplants may advance ALS treatment by repair of blood-spinal cord barrier Science Daily Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the ... and more »

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A new biomimetic bone tissue may help improve bone marrow transplants.

Engineers at the University of California San Diego have developed a bone-like implant that eliminates the need for donor cells to wipe out the hosts pre-existing cells, by allowing donor cells the space to live and grow.

Weve made an accessory bone that can separately accommodate donor cells. This way, we can keep the host cells and bypass irradiation, bioengineering professor Shyni Varghese, from the UC San Diego Jacobs School of Engineering, said in a statement.

The implants are made of a porous hydrogel matrix that contains calcium phosphate minerals in the outer matrix and donor stem cells that produce blood cells in the inner matrix.

The researchers successfully tested the bone tissues in mice and the donor cells survived for at least six months, while supplying the mice with new blood cells.

The structures matured into bone tissues of the mice that have a working blood vessel network and a bone marrow inside that supplies new blood cells. After a month the implanted marrow contained a mixture of host and donor blood cells, which remained circulating in the bloodstream even after 24 hours.

In the future, our work could contribute to improved therapies for bone marrow disease, Yu-Ru (Vernon) Shih, a research scientist in Vargheses lab and the studys first author said in a statement. That would have useful applications for cell transplantations in the clinic.

The researchers also took stem cells from the implanted marrow and transplanted them into another group of mice with their marrow stem cells eradicated by radiation and drugs. The transplanted cells diffused into the bloodstream of the mice in the second group.

Were working on making this a platform to generate more bone marrow stem cells, Varghese said.

According to Varghese, the implants could only be used in patients with non-malignant bone marrow diseases, where there arent any cancerous cells that need to be eliminated.

The researchers said this discovery indicates that implanted marrow is functional and donor cells can form and survive for long periods of time in the presence of host cells. They also said that the host and donor cells can travel between the implanted marrow and the hosts circulating blood through the blood vessel network formed in the implanted bone tissue.

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Engineered Bone Marrow Improves Transplant Safety - R & D Magazine

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Harvard Gazette

Researchers work to create kidney filtration barrier on a chip ... Harvard Gazette Researchers say their glomerulus-on-a-chip lined by human stem cell-derived kidney cells could help model patient-specific kidney diseases and guide ... and more »

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Researchers work to create kidney filtration barrier on a chip ... - Harvard Gazette

IPS Cell Therapy Comments Off on Researchers work to create kidney filtration barrier on a chip … – Harvard Gazette | May 12th, 2017

Induced pluripotent stem cells have revolutionized stem cell science in the decade since their invention. Theyre yielding clues into the nature of diseases such as cancer and Alzheimers, and are also being tapped for therapy.

But creating these IPS cells is lengthy, complicated and tricky, and the facilities equipped to make them cant accommodate all the scientists whod like to get their hands on them.

A UK-led consortium has removed that bottleneck, by producing 711 lines of ready-to-go IPS cells from healthy individuals. These lines are meant to help scientists understand the normal variations between healthy individuals and those involved in disease, as well as to understand normal human biology and development.

The IPS lines are available for research purposes to academic scientists and industry by contacting the Human Induced Pluripotent Stem Cell Initiative (HipSci), at http://www.hipsci.org and the European Bank for induced Pluripotent Stem Cells at https://www.ebisc.org.

The accomplishment was announced in a study published in Nature. It can be found online at j.mp/711ips.

While many other efforts have generated IPS cells to address rare diseases, this study produces them from healthy volunteers to plumb common genetic variation, Fiona Watt, a lead author on the paper and co-principal investigator of HipSci, from King's College London, said in a statement.

"We were able to show similar characteristics of iPS cells from the same person, and revealed that up to 46 per cent of the differences we saw in iPS cells were due to differences between individuals, Watt said in the statement. These data will allow researchers to put disease variations in context with healthy people."

Andrs Bratt-Leal, director of the Parkinson's Cell Therapy Program at The Scripps Research Institute in La Jolla, agreed.

This kind of study is extremely important because it leads to a deeper understanding of the differences between normal genetic variation and genetic changes that could negatively impact cell behavior, said Bratt-Leal, who was not involved in the study.

This data will help scientists using induced pluripotent stem cells to model diseases as well as scientists developing cell therapies, said Bratt-Leal, who works in the lab of stem cell researcher Jeanne Loring.

Because DNA sequencing has become a routine tool in the lab, enormous amounts of data have been produced, he said. Not only have we have observed a high level of genetic diversity between different people, but also a more subtle variation exists among the cells from an individual person. The next step is a better understanding of how this diversity translates to function and behavior of stem cells and mature cells derived from stem cells.

Loring and Bratt-Leal are studying the use of induced pluripotent stem cells to relieve symptoms of Parkinsons disease. They are in the process of translating the research into a therapy, aided with a grant from the California Institute for Regenerative Medicine.

The work was the product of a large-scale collaboration of scientists from various institutions in the United Kingdom, including the European Molecular Biology Laboratory in Cambridge; Wellcome Trust Sanger Institute in Cambridge; the University of Dundee in Dundee; and the University of Cambridge. Also participating was St Vincent's Institute of Medical Research in Victoria, Australia.

bradley.fikes@sduniontribune.com

(619) 293-1020

UPDATES:

1:00 p.m.: This article was updated with additional details.

This article was originally published at 10:00 a.m.

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Hundreds of new stem cell lines ready to help research - The San Diego Union-Tribune

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Skin cells found at root of balding, gray hair Science Daily The researchers found that a protein called KROX20, more commonly associated with nerve development, in this case turns on in skin cells that become the hair shaft. These hair precursor, or progenitor, cells then produce a protein called stem cell ...

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Brigham and Womens Hospital (BWH) will pay $10 million to resolve allegations that one of their stem cell research laboratories fraudulently obtained grant funding from the National Institutes of Health (NIH), according to a press release.

As per federal regulations and institutional policy requirements, BWH conducted an investigation that identified data integrity concerns in federally funded grant applications submitted by the Anversa lab. After learning of and investigating the allegations of misconduct in the Anversa laboratory, BWH disclosed its concerns to the U.S. Department of Health and Human Services, Office of the Inspector General, and Office of Research Integrity.

BWH independently evaluated the issues relative to the federal false claims requirements, said Lori Schroth, media relations manager at BWH. Following that evaluation, BWH self-disclosed this matter to appropriate government entities and ceased drawing implicated funds.

The settlement resolves the allegations against Dr. Piero Anversa, who ran the laboratory, and Drs. Annarossa Leri and Jan Kajstura. Allegedly, the doctors knew or should have known that their laboratory published and relied upon manipulated and falsified information including microscope images and carbon-14 age data for cells, according to the press release. This information was used in applications for NIH research grant awards concerning the purported ability of stem cells to repair damage to the heart.

The settlement also resolves allegations that the laboratory followed improper protocols, inaccurately characterized cardiac stem cells, and kept recklessly or deliberately misleading records, according to the press release.

Drs. Anversa, Leri, and Kajstura are no longer affiliated with BWH, and the lab has since been closed.

BWH is committed to ensuring that research conducted at the institution is done under the most rigorous scientific standards, and has made significant enhancements to research integrity compliance protocols as a result of this event, said Schroth.

Acting U.S. Attorney William D. Weinreb said in the press release that individuals and institutions that receive research funding from NIH have an obligation to conduct their research honestly and not to alter results to conform with unproven hypotheses.

Medical research fraud not only wastes scarce government resources but also undermines the scientific process and the search for better treatments for serious diseases, Weinreb said, according to the press release. We commend Brigham and Womens for self-disclosing the allegations of fraudulent research at the Anversa laboratory, and for taking steps to prevent future recurrences of such conduct.

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BWH settles research fraud allegations - Mission Hill Gazette

Cardiac Stem Cells Comments Off on BWH settles research fraud allegations – Mission Hill Gazette | May 6th, 2017

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

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

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

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

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

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

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Stem cell technique may aid in bone repair

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

'ONE POKE AT A TIME'

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

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

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

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

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

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

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

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

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

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

But in her case, it did.

BUILDING A BOND

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

'Looking for a double miracle'

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

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

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

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

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

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

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

James Christopher received his first blood transfusion three weeks ago.

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

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

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

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

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

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

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

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

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

Joy in the journey

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

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

The whole family travels to medical appointments.

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

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

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

What happens if there's a match?

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

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

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

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

Saving lives

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

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

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

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

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

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

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

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

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

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

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

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

Want to register?

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

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

Testing sites and times are:

Monroe

West Monroe

Surrounding parishes

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

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