A team of researchers at the Gladstone Institutes uncovered a new strategy to treat heart failure, a leading contributor to mortality and healthcare costs in the United States. Despite widespread use of currently-approved drugs, approximately 40% of patients with heart failure die within 5 years of their initial diagnosis.

"The current standard of care is clearly not sufficient, which highlights the urgent need for new therapeutic approaches," said Saptarsi Haldar, MD, an associate investigator at Gladstone and senior author of a new study featured on the cover of the scientific journal Science Translational Medicine. "In our previous work, we found that a drug-like small molecule called JQ1 can prevent the development of heart failure in mouse models when administered at the very onset of the disease. However, as the majority of patients requiring treatment already have longstanding cardiac dysfunction, we needed to determine if our strategy could also treat established heart failure."

As part of an emerging treatment strategy, drugs derived from JQ1 are currently under study in early-phase human cancer trials. These drugs act by inhibiting a protein called BRD4, a member of a family of proteins called BET bromodomains, which directly influences heart failure. With this study, the scientists found that JQ1 can effectively treat severe, pre-established heart failure in both small animal and human cell models by blocking inflammation and fibrosis (scarring of the heart tissue).

"It has long been known that inflammation and fibrosis are key conspirators in the development of heart failure, but targeting these processes with drugs has remained a significant challenge," added Haldar, who is also a practicing cardiologist and an associate professor in the Department of Medicine at the University of California, San Francisco. "By inhibiting the function of the protein BRD4, an approach that simultaneously blocks both of these processes, we are using a new and different strategy altogether to tackle the problem."

Currently available drugs used for heart failure work at the surface of heart cells. In contrast, Haldar's approach goes to the root of the problem and blocks destructive processes in the cell's command center, or nucleus.

"We treated mouse models of heart failure with JQ1, similarly to how patients would be treated in a clinic," said Qiming Duan, MD, PhD, postdoctoral scholar in Haldar's lab and co-first author of the study. "We showed that this approach effectively treats pre-established heart failure that occurs both after a massive heart attack or in response to persistent high blood pressure (mechanical overload), suggesting it could be used to treat a wide array of patients."

Using Gladstone's unique expertise, the scientists then used induced pluripotent stem cells (iPSCs), generated from adult human skin cells, to create a type of beating heart cell known as cardiomyocytes.

"After testing the drug in mice, we wanted to check whether JQ1 would have the same effect in humans," explained co-first author Sarah McMahon, a UCSF graduate student in Haldar's lab. "We tested the drug on human cardiomyocytes, as they are cells that not only beat, but can also trigger the processes of inflammation and fibrosis, which in turn make heart failure progressively worse. Similar to our animal studies, we found that JQ1 was also effective in human heart cells, reaffirming the clinical relevance of our results."

The study also showed that, in contrast to several cancer drugs that have been documented to cause cardiac toxicity, BRD4 inhibitors may be a class of anti-cancer therapeutics that has protective effects in the human heart.

"Our study demonstrates a new therapeutic approach to successfully target inflammation and fibrosis, representing a major advance in the field," concluded Haldar. "We also believe our current work has important near-term translational impact in human heart failure. Given that drugs derived from JQ1 are already being tested in cancer clinical trials, their safety and efficacy in humans are already being defined. This key information could accelerate the development of a new heart failure drug and make it available to patients more quickly."

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Materials provided by Gladstone Institutes. Note: Content may be edited for style and length.

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Cancer-cardiac connection illuminates promising new drug for heart failure - Science Daily

New research has nudged scientists closer to one of regenerative medicines holy grails: the ability to create customized human stem cells capable of forming blood that would be safe for patients.

Advances reported Wednesday in the journal Nature could not only give scientists a window on what goes wrong in such blood cancers as leukemia, lymphoma and myeloma. They could also improve the treatment of those cancers, which affect some 1.2 million Americans.

The stem cells that give rise to our blood are a mysterious wellspring of life. In principle, just one of these primitive cells can create much of a human beings immune system, not to mention the complex slurry of cells that courses through a persons arteries, veins and organs.

While the use of blood-making stem cells in medicine has been common since the 1950s, it remains pretty crude. After patients with blood cancers have undergone powerful radiation and chemotherapy treatments to kill their cancer cells, they often need a bone-marrow transplant to rebuild their white blood cells, which are destroyed by that treatment.

The blood-making stem cells that reside in a donors bone marrow and in umbilical cord blood that is sometimes harvested after a babys birth are called hematopoietic, and they can be life-saving. But even these stem cells can bear the distinctive immune system signatures of the person from whom they were harvested. As a result, they can provoke an attack if the transplant recipients body registers the cells as foreign.

This response, called graft-versus-host disease, affects as many as 70% of bone-marrow transplant recipients in the months following the treatment, and 40% develop a chronic version of the affliction later. It can overwhelm the benefit of a stem cell transplant. And it kills many patients.

Rather than hunt for a donor whos a perfect match for a patient in need of a transplant a process that can be lengthy, ethically fraught and ultimately unsuccessful doctors would like to use a patients own cells to engineer the hematopoietic stem cells.

The patients mature cells would be reprogrammed to their most primitive form: stem cells capable of becoming virtually any kind of human cell. Then factors in their environment would coax them to become the specific type of stem cells capable of giving rise to blood.

Once reintroduced into the patient, the cells would take up residence without prompting rejection and set up a lifelong factory of healthy new blood cells.

If the risk of deadly rejection episodes could be eliminated, physicians might also feel more confident treating blood diseases that are painful and difficult but not immediately deadly diseases such as sickle cell disease and immunological disorders with stem cell transplants.

The two studies published Wednesday demonstrate that scientists may soon be capable of pulling off the sequence of operations necessary for such treatments to move ahead.

One of two research teams, led by stem-cell pioneer Dr. George Q. Daley of Harvard Medical School and the Dana Farber Cancer Institute in Boston, started their experiment with human pluripotent stem cells primitive cells capable of becoming virtually any type of mature cell in the body. Some of them were embryonic stem cells and others were induced pluripotent stem cells, or iPS cells, which are made by converting mature cells back to a flexible state.

The scientists then programmed those pluripotent stem cells to become endothelial cells, which line the inside of certain blood vessels. Past research had established that those cells are where blood-making stem cells are born.

Here, the process needed a nudge. Using suppositions gleaned from experiments with mice, Daley said his team confected a special sauce of proteins that sit on a cells DNA and program its function. When they incubated the endothelial cells in the sauce, they began producing hematopioetic stem cells in their earliest form.

Daleys team then transferred the resulting blood-making stem cells into the bone marrow of mice to see if they would take. In two out of five mice who got the most promising cell types, they did. Not only did the stem cells establish themselves, they continued to renew themselves while giving rise to a wide range of blood cells.

A second research team, led by researchers from Weill Cornell Medicines Ansary Stem Cell Institute in New York, achieved a similar result using stem cells from the blood-vessel lining of adult mice. After programming those cells to revert to a more primitive form, the scientists also incubated those stem cells in a concoction of specialized proteins.

When the team, led by Raphael Lis and Dr. Shahin Rafii, transferred the resulting stem cells back into the tissue lining the blood vessels of the mice from which they came, that graft also took. For at least 40 weeks after the incubated stem cells were returned to their mouse owners, the stem cells continued to regenerate themselves and give rise to many blood-cell types without provoking immune reactions.

In addition to making a workhorse treatment for blood cancers safer, the new advances may afford scientists a unique window on the mechanisms by which blood diseases take hold and progress, said Lee Greenberger, chief scientific officer for the Leukemia and Lymphoma Society.

From a research point of view you could now actually begin to model diseases, said Greenberger. If you were to take the cell thats defective and make it revert to a stem cell, you could effectively reproduce the disease and watch its progression from the earliest stages.

That, in turn, would make it easier to narrow the search for drugs that could disrupt that disease process early. And it would speed the process of discovering which genes are implicated in causing diseases. With gene-editing techniques such as CRISPR-Cas9, those offending genes could one day be snipped out of hematopoietic stem cells, then be returned to their owners to generate new lines of disease-free blood cells.

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Scientists get closer to making personalized blood cells by using patients' own stem cells - Los Angeles Times

Jonathan Pitre in a 2015 TSN profile.

This time, it worked.

Suffering from a severe form of epidermolysis bullosa (EB), an incurable genetic condition which causes the skin to blister and create painful wounds, Pitre, who turns 17 next month, was given the moniker Butterfly Boy due to his delicate skin.

EB can be fatal, with many people who have severe EB dying from skin cancer in their twenties. Pitre underwent his second stem cell transplant procedure at the University of Minnesota Masonic Childrens Hospital, a pioneer in treating EB though stem cell transplants.

Paediatric hematoligist-oncologist with the University of Minnesota Jakub Tolar calls EB the worst disease youve never heard of, as it affects only one in 20,000 people. Research by Tolar and his colleagues led to the discovery that bone marrow transplantation, a procedure typically used to treat blood cancers in the bone marrow such as leukemia, could benefit those with EB.

This had never been done before, says Tolar, who directs the U of Ms Stem Cell Institute, in a press release. I didnt know it at the time we started this research 10 years ago, but it opened a totally new field in transplantation biology.

Stem cell transplants involve a persons blood-forming stem cells (immature cells that can become various types of specialized cells in the body, in this case, becoming different types of blood cells) from the bone marrow and replacing them with healthy stem cells.

For Pitre, his earlier bone marrow transplant last October proved unsuccessful as doctors learned that his own stem cells had recolonized his bone marrow. This time around, the results look more promising. Pitres mother, Tina Boileau, who was the donor, is now full of joy and relief, according to an Ottawa Citizen report, which states that newly created white blood cells in Pitres system contain a pair of X chromosomes, indicating that they came from Boileaus donated cells.

This is the best news ever, the best Mothers Day gift, said Boileau. Jon is full of me. He doesnt have any T-cells that are his.

Its been over 30 years since bone marrow cells were first used to treat cancer, but recent advances have shown the potential application of stem cell transplantation for a variety of diseases and conditions, from brain and spinal cord injury to neurodegenerative diseases like Alzheimers to HIV/AIDS. Researchers at Cardiff University in Wales, for example, have just announced commencement of stem cell transplants for patients with Huntingtons disease.

The Ontario government has just announced $32 million in new funding to help shorten the long wait times for stem cell transplants in the province, meaning that 150 more patients a year will be able to receive transplant therapy. As reported in the Hamilton Spectator, $10 million of the new funds will be going to the Juravinski Hospital and Cancer Centre in Hamilton for a dedicated unit with 15 inpatient and five outpatient beds.

Below: TSN Original: The Butterfly Child

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Ontario teen Jonathan Pitre's second attempt at stem cell transplant is a success - Cantech Letter

Scientists have healed severe bone fractures in pigs by blasting tiny bubbles with ultrasound in the animals bones. The technique encourages the pigs bodies to regenerate themselves, and could one day be used to help humans especially the elderly heal dangerous bone injuries.

Broken bones are common: you wrap an arm or wrist in a cast and the bone eventually heals on its own. But sometimes, people have nonunion fractures, meaning bones fail to produce new bone tissue and dont heal properly. There are about 100,000 cases of this in the United States every year. One solution is bone grafts, or bone transplants using donated marrow, but this procedure is invasive and there is a risk that the body will reject the marrow. Another solution is to use viruses to deliver bone morphogenetic proteins (BMPs) that encourage the bodys own stem cells to create more bone marrow. But using a virus can have negative side effects like inflammation.

In a study published today in Science Translational Medicine, scientists healed a 0.4-inch fracture in pigs in eight weeks without invasive surgery. Going from something invasive to something like this that potentially could be an outpatient procedure has been the holy grail in orthopedics, says Edward Schwarz, director of the University of Rochesters Center for Musculoskeletal Research, who was not involved with the study. He adds that, though these nonunion fractures arent the most common health problem, theyre a serious one. People are shocked when I tell them that the life expectancy with a nonunion fracture is shorter than with pancreatic cancer, he says. Were like horses. If we cant get up and walk again, then were done.

In the study, the researchers first caused a 0.4-inch fracture in the shins of 18 minipigs. Then, they inserted a biodegradable scaffolds into the broken shins, says co-author Gadi Pelled, a professor of surgery at Cedars-Sinai Medical Center. The scaffold helped support bone stem cells in the area. The scientists let the stem cells migrate and populate over the scaffold for two weeks but that wast enough. The stem cells had to be triggered to actually heal the injury. So the scientists injected microbubbles mixed with bone morphogenetic proteins. Immediately after the injection, they applied ultrasound, which stimulated the BMPs to enter into the stem cells and activate them.

The stem cells then turned into bone cells and healed the fracture after eight weeks. This method doesnt have the side effects associated with using viruses, and the fact that it uses the bodys own stem cells means theres no risk of rejection, says co-author Zulma Gazit, also at Cedars-Sinai. This ultrasound and microbubbles combo has already been approved by the Food and Drug Administration and is often used in radiology, so the new technique could be readily approved for use in humans.

Next, says Pelled, the team is studying whether the same technology can also work with tissues like ligaments; they gathering more comprehensive information. Before we move forward into humans, we need to determine that this technology is safe, says Pelled. Theyre hopeful that a clinical trial is on the way.

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Blasting tiny bubbles at broken pig bones makes them heal on their own - The Verge

Press Release: New Stem Cell Collection Center Opens in Boston The Scientist We support biomedical researchers globally by offering human hematopoietic stem cells and blood derived cell products from bone marrow, cord blood, peripheral blood and mobilized peripheral blood. StemExpress guarantees every sample delivers only ...

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Press Release: New Stem Cell Collection Center Opens in Boston - The Scientist

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

A team of scientists has successfully repaired the broken bones of lab animals without invasive surgery, by using microbubbles and ultrasound to stimulate the growth of stem cells.

In a study published in the journal Science Translational Medicine on Wednesday, Maxim Bez and a team of Cedars Sinai-led scientists were able to facilitate the natural growth of stem cells to create more bone marrow in broken bones that cannot heal on their own, known as nonunion fractures.

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While certain bone injuries only require a few weeks in a cast to heal, more severe injuries can cause large gaps between the edges of a fracture that cannot be healed without invasive surgery or bone grafting.

There are currently two methods for bone grafting, either using autografts that transfer bone marrow from a different part of the patients own body, or allografts that use donated bone marrow from another patient. Artificial transplants are often rejected by the body, making another bone graft necessary.

There are more than 2 million bone grafting procedures performed around the world each year, with roughly 100,000 cases in the US alone.

Nonunion bone fractures can cause great damage to the body, leaving patients crippled or with other severe complications.

In a first of its kind study, Bez and his team developed an alternative that uses microbubbles and ultrasound to facilitate the bodys natural stem-cell growth.

"This study is the first to demonstrate that ultrasound-mediated gene delivery to an animal's own stem cells can effectively be used to treat nonhealing bone fractures," said Gadi Pelled, assistant professor of surgery at Cedars-Sinai and co-author of the study, according to Medical XPress. "It addresses a major orthopedic unmet need and offers new possibilities for clinical translation."

In the study, Bez and his team first created severe bone fractures the tibiae bones of large pigs. Then, they inserted a biodegradable collagen scaffold in the fracture, which supported stem cell growth. Two weeks later, after the stem cells grew around the scaffold, the scientists injected microbubbles containing growth-promoting genes. Finally, they used an ultrasound pulse, which causes the stems cells to become bone cells, healing the fracture.

The technique was able to completely heal nonunion fractures in eight weeks. Bez and his team found their method healed bones to the point that they were just as strong as those treated with bone grafts.

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The technique is minimally invasive and does not have the side effects associated with bone grafts. If the method is found to be safe for humans, it would provide patients with an alternative to replace bone grafting.

Bez and his team say that their method could potentially be used in tissue engineering applications in the future.

"We are just at the beginning of a revolution in orthopedics," said Dan Gazit, co-director of the Skeletal Regeneration and Stem Cell Therapy Program in the Department of Surgery and the Cedars-Sinai Board of Governors Regenerative Medicine Institute and co-author of the study, according to Medical XPress. "We're combining an engineering approach with a biological approach to advance regenerative engineering, which we believe is the future of medicine."

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'Future of medicine': Broken pig bones healed using tiny bubbles & ultrasound - RT

New York Times

Babies From Skin Cells? Prospect Is Unsettling to Some Experts ... New York Times Researchers say that scientists may soon be able to create a baby from human skin cells that have been coaxed to grow into eggs and sperm. and more »

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Babies From Skin Cells? Prospect Is Unsettling to Some Experts ... - New York Times

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

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)

International Stem Cell Corporation Announces Operating Results for the Quarter ended March 31, 2017 P&T Community ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin ... and more »

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International Stem Cell Corporation Announces Operating Results for the Quarter ended March 31, 2017 - P&T Community

Inquirer.net

Approaching a decades-old goal: Making blood stem cells from patients' own cells Science Daily ... lab using pluripotent stem cells, which can make virtually every cell type in the body. The advance opens new avenues for research into the root causes of blood diseases and to creating immune-matched blood cells for treatment purposes, derived ... 'Milestone' in quest to make blood cells studiesInquirer.net all 21 news articles »

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Approaching a decades-old goal: Making blood stem cells from patients' own cells - Science Daily

Patients dealing with blood and immune disorders, especially those in the most advanced stages, often have no choice but to undergo bone marrow transplants. Ironically, even if the treatment can be life-saving, it would only work when the bone marrow cells of the recipients are completely eliminated using drugs and radiation. And this could cause serious negative side effects such as organ damage, cataracts, infertility, new cancers, and even death.

Thanks to the work of engineers at the University of California San Diego (UCSD), that kind of bone marrow transplant may soon be rendered obsolete. Rather than using a live bone marrow from a compatible donor or from the patients themselves, a synthetic bone implant will instead be used and such will not require the use of drugs that can cause all those harmful side effects.

Bone marrow is the flexible tissue inside the bones that is responsible for producing red blood cells from stem cells. If, for some reason, the bone marrow fails to do its job, the result can either be severe anemia or an impaired immune system. Whichever of these conditions arise, the most effective treatment is typically a bone marrow transplant.

To reduce the undesirable side effects caused by traditional bone marrow transplants, the UCSD team of bioengineers led by Shyni Varghese have developed a synthetic bone implant with a practical marrow that can produce its own blood cells. The implant is divided into two sections, both of which are engineered from a hydrogel matrix. The exterior layer containing calcium phosphate minerals functions as a bone, while the interior layer contains donor stem cells for bone marrow growth. The exterior layer works together with the hosts cells to assist in bone building, merging the implant with the natural structure of the body.

According to the team, they have tested their engineered implant in mice, and the tests proved successful. Specifically, they implanted the synthetic bone under the skin of mice, some of which had functional bone marrow, and some of which had defective bone marrow due to radiation.

Within a four-week observation period, the implant developed bone-like structures that didnt only have blood vessels, but also marrow that actually produced red blood cells. And after six months, the synthetic implants and the bloodstream of the mice showed a mix of blood cells from both the donor and the host. This shows that the implants can function as natural bones, with the blood cells produced by the synthetic implant naturally circulating within the hosts bloodstream without being rejected.

As promising as those results are, however, there is no guarantee that the technique will be as effective in humans. Further study will be required before it can be accepted and approved by the FDA.

Theres also the matter of the treatment only being effective on patients with non-malignant bone marrow disorders. The implant cannot do anything to stop or prevent cancerous mutation from spreading, which means when it comes to cancer patients, undergoing radiation therapy will still be required to kill off their cancer cells, before a bone marrow transplant can work.

Nevertheless, this is still considered a step forward and an exciting development, particularly for individuals suffering from blood disorders. Not only will the treatment ease their pain and distress because theyll be free of their disease; it will also keep them from suffering negative side effects.

The research was recently published in the journal PNAS.

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New Discovery Could Soon Replace The Painful Bone Marrow Transplant - Wall Street Pit

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Posted By: Dave Bernstein May 15, 2017

UPDATE: Commissioner Dennis Bolz talks about how much he missed PUD

Chelan County PUD Commissioner Dennis Bolz was able to attend his first board meeting in person this morning after a lengthy absence while undergoing stem cell transplant therapy.

Commissioner Bolz talked with NewsRadio 560 KPQs Dave Bernstein moments before his first board meeting at 10am Tuesday..

Commissioner Bolz has beenin Seattle since January receiving chemotherapy for myelodysplastic syndrome or MDS. The cancer affects the formation of red blood and white cells in the bone marrow.

Bolz thanked the PUD Board , staff and the community for their support and encouragement during his absence and talked about how much he missed the important work at the PUD and being in the Wenatchee Valley.

Commissioner Bolz was presented with a 10 year service pin hemissed receiving in January whilein Seattle undergoing treatment

Read more:
PUD Commissioner Returns after Stem Cell Transplant - KPQ

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

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

Neuralstem Cell Therapy:

Different regions of the brain and spinal cord house different, specialized cells. Neuralstem's technology enables the isolation and expansion of human neural stem cells from each of these regions of the developing central nervous system (CNS) in virtually unlimited numbers from a single donated tissue.

The goal of cell therapy is to replace and/or repair dead or diseased cells. Unlike other stem cell technologies, Neuralstem is growing regionally specific cells that are already suited to the task prescribed to them once transplanted into the CNS. In spinal cord indications, for instance, the company will be using human NSI-566 spinal cord stem cells only. Additionally, once inside the body, Neuralstem cells also do not become any cell other than that to which they are fated.

There are two primary ways that these cells can provide therapeutic effects. Create: The transplanted cells may help create new circuitry Express: The transplanted cells may express factors that protect existing cells

We believe that Neuralstem's cells do both.

In preclinical work conducted at major research centers across the U.S., Neuralstem cells integrated and made synaptic contact with the host. The cells also expressed one or more growth factors. These are special chemicals that the CNS uses to operate and thrive. Many of these growth factors are protective of cells. View published papers here: 1, 2, 3.

Neuralstems transplanted cells survive in patients and integrate into the host tissue, creating new circuitry and expressing growth factors. This dual function is important. In spinal cord injury, for instance, the company hopes to create circuitry that will help signals from the brain get to where they need to go. In many indications, the goal is to slow down or halt the degeneration of cells caused by disease, or by injury, by expressing neuroprotective growth factors into the system.

A vital component to the Neuralstem cell therapy platform is the delivery of the cells directly into the gray matter of the spinal cord, where they can protect and integrate with the patient's spinal cord neurons.

Neuralstem's proprietary Spinal Cord Delivery Platform and Floating Cannula were designed specifically by Neuralstem's ALS trial neurosurgeon, Nicholas M. Boulis, MD, for the world's first intraspinal delivery of stem cells. The safety of the device was first reported in data presented at the American Association of Neurologists' 2011 Annual Meeting, and its safety has since been repeatedly validated in the companys completed two ALS clinical studies, in a total of thirty patients, which met primary safety endpoints. In addition to ALS, NSI-566 is also in a Phase I trial in chronic spinal cord injury at UC San Diego School of Medicine. You can view this breakthrough medical device in surgery here.

The Spinal Cord Delivery Platform and Floating Cannula will be utilized to deliver Neuralstem cells in the spinal cord safely and effectively for myriad diseases and injuries. Expected to be the standard in the industry and research community for intraspinal procedures, Neuralstem is licensing the breakthrough cell therapy device to industry and academia.

Delivery of neural stem cells into the brain will be accomplished using well-established stereotactic injection procedures. NSI-566 is in clinical development to treat ischemic stroke utilizing one-time treatments of these intracerebral injections to safely transplant cells near the stroke lesions of ischemic stroke patients.

Follow this link:
Repairing and Replacing Damaged Cells - Neuralstem

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.

Link:
Heart disease on Stem Cells - ISSCR

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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Stem cell transplants may advance ALS treatment by repair of blood-spinal cord barrier - Science Daily

On Tuesday, Carson Tahoe Cancer Center opened a new blood and bone marrow transplant care clinic with support from the Huntsman Cancer Institute (HCI) at the University of Utah. Under the collaboration, a Bone Marrow Transplant (BMT) physician and nurse from HCI will travel to Carson City once a month to treat patients both before and after they receive a transplant.

Blood and marrow transplants are performed in patients with cancers of the blood and lymphatic systems, including leukemia, lymphoma and multiple myeloma. The transplants replace bone marrow that has been damaged or destroyed with a supply of healthy blood stem cells, which in turn travel to the bone marrow and promote growth of new marrow.

Currently, patients in the Northern Nevada area who need a transplant must travel outside the area for treatment. Through this model, patients will still receive their transplant at HCI in Salt Lake City. But they will now be able to receive care at the Carson Tahoe clinic for planning and follow-up appointments, which typically occur every month for a year following transplant.

"This clinic is going to enable patients to receive more of their pre-and post-BMT care closer to home," said Daniel Couriel, MD, Professor of Medicine at the University of Utah and Director of HCI's BMT program. "We hope to maximize the time patients can spend in their own homes with their loved ones as they recover."

The BMT clinic will be open the third Monday of every month. Patients can access the clinic by referral.

"Because of the added bench strength we receive from HCI, we are better equipped to provide outstanding bone marrow transplant care, close to home," said Ed Epperson, CEO of CTH.

CTH formally affiliated with University of Utah Health in 2013 and with Huntsman Cancer Institute in 2015 in an effort to improve accessibility to specialty care for Northern Nevada residents. The relationship between the health care systems provides resources that allow Carson Tahoe Health to meet the ever-changing health care needs of the community.

"Both organizations share a commitment to providing the highest quality cancer care to patients, no matter where they live," said John Sweetenham, MD, Executive Medical Director at Huntsman Cancer Institute and Professor of Medicine at the University of Utah. "BMT treatment is a very unique type of care, and we look forward to working with Carson Tahoe to bring this service to the community."

To find out more about the clinic, residents can call Carson Tahoe Cancer Center at 775-445-7500.

Originally posted here:
Carson Tahoe Health opens blood and bone marrow transplant care clinic - Nevada Appeal

An umbilical cord after birth yields about three to five ounces of cell-rich cord blood. That's not a lot, but enough of it can help treat more than 80 or so diseases. A North Texas oncologist says education's key to boosting limited supply.

The KERA Interview with Dr. Sharif

Dr. Suhail Sharif is a surgical oncologist with Texas Health Fort Worth.

Interview Highlights:

Whats special about cord blood: Cord blood has immature blood cells, and you can use these stem cells to, basically, harvest into these patients that have problems with their own blood; for example, because of leukemia or lymphoma or other types of diseases that affect their own blood lines. These can grow into the red blood cells if [they're] deficient or the white blood cells if [they're] deficient or even platelets, for that matter.

Cord blood cells vs. bone marrow cells: Cord blood stem cells are actually stored in a blood bank that you can use on patients that need it. But bone marrow, you actually have to go through a process of harvesting the bone marrow. Its a very painful procedure for whoever is donating the bone marrow. And then they have to go through an extensive and rigorous testing, not only for infectious causes, but also to see if they match with the patient. And then they have to harvest, and they basically have to transplant it. Now, that whole process can take a few months. If you just have cord blood stem cells, these have already been stored and are readily available. And if you have a match with the donor and the recipient, you can use them right away.

Cord blood is limited in supply: If you think about the blood that is in the placenta and the cord, its in the range of three to five ounces. Thats about like half a cup. Thats the reason why you have to gather it from a lot of patients. At this point, there are, I believe, close to 175,000 matched cord blood available."

But its not enough: If you think about what percentage of deliveries actually translate into donating cord blood, its very miniscule. Thats why were educating the parents about the benefits of cord blood so they can donate to a public blood bank so that we can use it in treating patients with deadly cancers and so forth in our community.

For more information:

Excerpt from:
Cord Blood: A Small Amount Does A Lot Of Good - KERA News