Sickle cell cure is real, as this Kansas patient proves Kansas City Star Intense pain. Fatigue. Repeated infections, emergency room visits and hospitalizations. Desiree Ramirez endured them often until she became the first adult cured at a Kansas hospital of sickle cell disease. Bone marrow stem cells donated by a ...

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By By Elianna Novitch, The Gazette

May 29, 2017 at 5:00 am | Print View

IOWA CITY More than 20 million people are registered as bone marrow donors in the Be the Match registry, the largest and most diverse donor registry in the world.

But none can help Calder Wills, a 12-year-old Iowa City boy battling stage 4 T-cell lymphoma, or cancer of the blood.

Only one person has been identified as a 100-percent match for Calder, but that person was deemed medically unable to donate bone marrow.

This has left the Wills family with few options.

And so, friends of the family are hosting a donor registry drive on Tuesday to raise awareness about the need for more marrow donors and to perhaps find a match for Calder and others like him.

The event takes place from 3 to 8 p.m. inside the gym at Hoover Elementary School, 2200 E. Court St., Iowa City. Those who attend can join the Be The Match registry. Those who are unable to attend can register online at bethematch.org.

Calder was diagnosed with lymphoma in February 2016. He went into remission within the first 30 days but found out on April 11 the day after his 12th birthday that he had relapsed and would need a bone-marrow transplant. He is one of thousands searching for a match.

He is among the 70 percent of patients who surprisingly dont have a match in their own family, explained Colleen Reardon, manager of the Iowa Marrow Donor Program at the University of Iowa Hospitals & Clinics. We are looking for a tissue type match and each sibling has about a 25 percent chance of being a match.

Calder has three siblings, a twin brother Grayson and sisters Charlotte, 7, and Arden, 5, all of whom were not matches. The next best chance a patient has, statistically, is to find an unrelated donor that is a 100-percent match.

Calders mother Brianna Wills described it as devastating when the family found out that the 58-year-old woman who matched with Calder was deemed medically unable to donate.

That left us with no match, no options, she said. Weve decided to pursue cord blood for his transplant, Wills said. He is going to have a cord blood transplant at the University of Minnesota because a bone marrow match wasnt available and he couldnt wait until one became available.

According to the Be The Match website, cord blood is one of three sources of blood-forming cells used in transplant. The others are bone marrow and peripheral blood stem cells. Cord blood can be used to treat more than 80 diseases, including blood cancers like leukemia and lymphoma. Cord blood comes from a babys umbilical cord.

Wills said that even though Calder is receiving a different type of transplant, she does not want people to not register as a marrow donor.

I dont want that to dissuade people from continuing to do it because he has about a two out of three chance that this transplant will fail because he has T-cell lymphoma that is very aggressive and very hard to treat, Wills said. Realistically, statistically, we are looking at him needing a second transplant down the road and thats when we hope that well find a donor and we can use a bone marrow match then.

Please still do it and not just for Calder, do it for the thousands of people who also dont have a match.

According to Reardon, of every 540 people who register as a donor, only one will be identified as that perfect match for someone and be asked to donate.

Were not realistically hoping to find Calders donor, I mean that would be amazing, but really were hoping to expand the database. Were just hoping that some family in Texas or somewhere else in the world is also doing this and maybe theyll find Calders donor, Wills said. If were all doing it, were going to expand the database for everyones benefit.

Wills recognizes that even though the drive is in Calders honor, it is truly to the benefit of thousands of other people who dont have donors.

There are other ethnic groups that have very little participation and to be a match you need to be matched with donors that have similar ethnic background as you do, Wills said. So African Americans, Hispanics, people that have mixed races, or Asian background wed love to have them come because there are people waiting for donors of all kinds of backgrounds.

What: Bone Marrow Donor Drive

When: 3 to 8 p.m. Tuesday

Where: Hoover Elementary School, 2200 E. Court St., Iowa City

Details: Join the Iowa Marrow Donor Program and Be The Match Registry using a simple cheek swab.

Info: join.bethematch.org/CalderStrong or call the Iowa Marrow Donor Program at (319) 356-3337.

l Comments: (319) 368-8538; elianna.novitch@thegazette.com

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DENVER -- The need is undeniable. The majority of cancer patients in need of a stem cell or bone marrow transplant are not able to get one, in part because they cant find a match.

Doctors hope more people will register to be a donor, and say all it takes to get started is a simple cheek swab.

Paige McCoy, of Parker, did find a match. After she was diagnosed with acute lymphoblastic leukemia at age 28, she needed a stem cell transplant to survive. I honestly thought I was going to die at 28, she said.

But a total stranger absolutely saved her life.

She got to meet her donor for the first time this month at the Gift of Life Gala in New York City. When I saw him I just broke down, because here is basically my hero walking towards me, Paige said.

It was an emotional night. Her donor was a 22 year old student at the University of Tennessee who had registered with a cheek swab at a campus event.

When he agreed to donate, he had to get some shots, then the stem cells were gathered during a type of blood draw. "The blood goes out to the machine. The machine processes the blood, and returns the red blood cells and the rest of the blood products, except for some of the stem cells, back to the donor, said Dr. Michael Maris, the director of research at the Colorado Blood Cancer Institute in partnership with Sarah Cannon Cancer Institute at Presbyterian/St. Lukes.

He says this act, that required no surgery, saved Paiges life.

But Paige knows others werent as lucky. I saw patients that didnt have a donor, and I had a donor and they didnt, and somebody could save their life. Just swab your cheek please. You could really help somebody out, and its so easy, she said.

If you would like to register, you can go to http://www.bethematch.org for cheek swab instructions, or a list of local donation events. Your registration could also help patients needing bone marrow transplants. But the marrow harvesting does require surgery.

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Rahman lends his voice for stem cell donation The Hindu Sign up with me as bone marrow donor, Mr. Rahman says, in a short video made by the Jeevan Stem Cell Foundation, which maintains a registry for those in need to find a stem cell match. The video has been uploaded to Youtube ahead of the World Blood ... and more »

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For the first time, scientists have discovered that a common type of immune cell directly triggers stem cells in the skin that are responsible for hair growth in mice. Without this trigger, hair follicles just don't do their job -even if they have the stem cells necessary to proceed.

As the mechanisms for hair growth in mice are similar in humans, the researchers hope their newly uncovered mechanism could lead to a better understanding of conditions like alopecia, and other types of baldness.

Among the various immune system players we have in the body, there's a subclass of immune cells called regulatory T cells, or Tregs for short.

The vast majority of Tregs live in our lymph nodes, where they help to control inflammation throughout the body. But we also have subsets of Tregs that reside in other body parts, such as muscle or lung tissue.

And studies are starting to show that these 'tissue-resident' Tregs may be performing unique roles specific to the part of body they're in.

Researchers know that both mice and humans have a lot of Tregs in the skin, but so far we know very little about their function there.

Seeing that skin-specific Tregs tend to sit around hair follicles, a team led by researchers from the University of California San Francisco (UCSF) investigated the hypothesis that these immune cells were somehow involved in hair growth.

What they discovered is not just involvement, but a direct trigger - making Tregs a super-important part of the hair growth process.

"Our hair follicles are constantly recycling: when a hair falls out, a portion of the hair follicle has to grow back," senior researcher Michael Rosenblum said in a press statement.

"This has been thought to be an entirely stem cell-dependent process, but it turns out Tregs are essential."

In mammals, hair follicles regenerate in a specific pattern, cycling between growth phases (known as anagen) and rest phases (telogen).

The team tracked the amount of Tregs in the skin of mice during these different phases of hair growth, and found a tight correlation - in the telogen phase these immune cells were much more abundant.

What's more, highly active Tregs were crowding around hair follicles at three times the normal rate, right towards the end of the hair growth rest phase.

Intrigued by this correlation, the scientists took a step further to uncover the biological mechanism involved in the relationship between Tregs and the stem cells that make hair follicles do their job.

To do this, they took genetically modified mice whose Treg cells could be 'knocked out' with a simple intervention.

The researchers clipped the hair on the mice's backs and then applied a depilatory cream for 30 seconds - when you depilate the skin, hair follicles kick into the active hair growth phase.

They monitored the hair regrowth for 14 days, comparing the regrowth between control mice and the ones whose Tregs they had tampered with.

In mice whose Tregs were knocked out in the first three days after depilation, the hair just didn't grow back, leaving them with a bald patch on their backs.

A closer look revealed that Tregs directly trigger the activation of stem cells in the hair follicle through a well-known cell communication mechanism called the Notch signalling pathway, which involves a specific protein called Jag1.

They even found that when they replaced Tregs with microscopic beads covered in Jag1, it triggered the activity in the hair follicles just like Tregs would.

"It's as if the skin stem cells and Tregs have co-evolved, so that the Tregs not only guard the stem cells against inflammation but also take part in their regenerative work," Rosenblum said.

"Now the stem cells rely on the Tregs completely to know when it's time to start regenerating."

It's a really elegant demonstration of a previously unknown mechanism for hair growth in mice, but there's a lot more work to be done before we can tell whether defective skin Tregs could be the culprits behind hair loss in humans.

But there's at least one tantalising clue that the study is onto something here. In genome-wide association studies of alopecia areata, a condition characterised by 'patchy' hair loss, researchers have found mutations on genes that are involved in Treg function.

Next up, the researchers are hoping to expand their results and investigate how Tregs in the skin could be involved in wound healing, and also various hair loss conditions in humans.

"It will be important to determine whether this principle extends to human diseases of epithelial dysfunction and whether Tregs can be exploited to develop new therapies for stem-cell-mediated tissue regenerative disorders," they write in the study.

These new results are also an exciting addition to the growing body of knowledge scientists have about hair growth. Earlier this month, researchers reported the discovery of a protein that causes skin stem cells to develop into hair cells in mice. They are now investigating whether this protein is involved in hair loss in people.

The research has been published in Cell.

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In experiments in mice, UC San Francisco researchers have discovered that regulatory T cells (Tregs; pronounced tee-regs), a type of immune cell generally associated with controlling inflammation, directly trigger stem cells in the skin to promote healthy hair growth. Without these immune cells as partners, the researchers found, the stem cells cannot regenerate hair follicles, leading to baldness.

"Our hair follicles are constantly recycling: when a hair falls out, the whole hair follicle has to grow back, said Michael Rosenblum, MD, PhD, an assistant professor of dermatology at UCSF and senior author on the new paper. This has been thought to be an entirely stem cell-dependent process, but it turns out Tregs are essential. If you knock out this one immune cell type, hair just doesnt grow.

The new study published online May 26 in Cell suggests that defects in Tregs could be responsible for alopecia areata, a common autoimmune disorder that causes hair loss, and could potentially play a role in other forms of baldness, including male pattern baldness, Rosenblum said. Since the same stem cells are responsible for helping heal the skin after injury, the study raises the possibility that Tregs may play a key role in wound repair as well.

Anti-Inflammatory Immune Cells Activate Skin Stem Cells

Normally Tregs act as peacekeepers and diplomats, informing the rest of the immune system of the difference between friend and foe. When Tregs dont function properly, we may develop allergies to harmless substances like peanut protein or cat dander, or suffer from autoimmune disorders in which the immune system turns on the bodys own tissues.

Like other immune cells, most Tregs reside in the bodys lymph nodes, but some live permanently in other tissues, where they seem to have evolved to assist with local metabolic functions as well as playing their normal anti-inflammatory role. In the skin, for example, Rosenblum and colleagues have previously shown that Tregs help establish immune tolerance to healthy skin microbes in newborn mice, and these cells also secrete molecules that help with wound healing into adulthood.

Rosenblum, who is both an immunologist and a dermatologist, wanted to better understand the role of these resident immune cells in skin health. To do this, he and his team developed a technique for temporarily removing Tregs from the skin. But when they shaved patches of hair from these mice to make observations of the affected skin, they made a surprising discovery. We quickly noticed that the shaved patches of hair never grew back, and we thought, Hmm, now thats interesting, Rosenblum said. We realized we had to delve into this further.

In the new research, led by UCSF postdoctoral fellow and first author Niwa Ali, PhD, several lines of evidence suggested that Tregs play a role in triggering hair follicle regeneration.

First, imaging experiments revealed that Tregs have a close relationship with the stem cells that reside within hair follicles and allow them to regenerate: the number of active Tregs clustering around follicle stem cells typically swells by three-fold as follicles enter the growth phase of their regular cycle of rest and regeneration. Also, removing Tregs from the skin blocked hair regrowth only if this was done within the first three days after shaving a patch of skin, when follicle regeneration would normally be activated. Getting rid of Tregs later on, once the regeneration had already begun, had no effect on hair regrowth.

Tregs role in triggering hair growth did not appear related to their normal ability to tamp down tissue inflammation, the researchers found. Instead, they discovered that Tregs trigger stem cell activation directly through a common cell-cell communication system known as the Notch pathway. First, the team demonstrated that Tregs in the skin express unusually high levels of a Notch signaling protein called Jagged 1 (Jag1), compared to Tregs elsewhere in the body. They then showed that removing Tregs from the skin significantly reduced Notch signaling in follicle stem cells, and that replacing Tregs with microscopic beads covered in Jag1 protein restored Notch signaling in the stem cells and successfully activated follicle regeneration.

Its as if the skin stem cells and Tregs have co-evolved, so that the Tregs not only guard the stem cells against inflammation but also take part in their regenerative work, Rosenblum said. Now the stem cells rely on the Tregs completely to know when its time to start regenerating.

Relevance to Autoimmune Hair Loss Rosenblum said the findings may have implications for alopecia areata, an autoimmune disease that interferes with hair follicle regeneration and causes patients to lose hair in patches from their scalp, eyebrows, and faces. Alopecia is among the most common human autoimmune diseases its as common as rheumatoid arthritis, and more common than type 1 diabetes but scientists have little idea what causes it.

After his team first observed hair loss in Treg-deficient mice, Rosenblum learned that the genes associated with alopecia in previous studies are almost all related to Tregs, and treatments that boost Treg function have been shown to be an effective treatment for the disease. Rosenblum speculates that better understanding Tregs critical role in hair growth could lead to improved treatments for hair loss more generally.

The study also adds to a growing sense that immune cells play much broader roles in tissue biology than had previously been appreciated, said Rosenblum, who plans to explore whether Tregs in the skin also play a role in wound healing, since the same follicle stem cells are involved in regenerating skin following injury.

We think of immune cells as coming into a tissue to fight infection, while stem cells are there to regenerate the tissue after its damaged, he said. But what we found here is that stem cells and immune cells have to work together to make regeneration possible.

This article has been republished frommaterialsprovided byUCSF. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference:

Ali, N., Zirak, B., Rodriguez, R. S., Pauli, M. L., Truong, H., Lai, K., . . . Rosenblum, M. D. (2017). Regulatory T Cells in Skin Facilitate Epithelial Stem Cell Differentiation. Cell. doi:10.1016/j.cell.2017.05.002

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CHENNAI: Double Oscar winning Indian composer A R Rahman has made an appeal to youngsters to register themselves as bone marrow donors. The music directors appeal is made on behalf of the Chennai-based Jeevan Stem Cell Foundation to mark the world blood cancer day (May 28, Sunday).

The foundations co-founder and chairman, P Srinivasan said every year over 1.2 lakh Indians are diagnosed with blood cancer and another 10,000 children born with diseases like Thalassemia. They could hope for a 60 to 80% chance of cure, with matching stem cell donors. So, the foundation has created a registry, which is a database of potential stem cell donors, and matching donors are identified when needed.

To encourage more people to register in this database, the foundation with the help of AR Rahman has put out a YouTube video to mark world blood cancer day. Over 90 per cent of us cant find a stem cell match because Indian DNA is different and we dont have a large bone marrow registry.

If you are between 18 and 50, it is your time to save an Indian life, sign up with me as bone marrow donor in Jevan stem cell registry, said Rahman in the video.

Interested individuals can login to http://www.bethecure.in, read who are eligible and register as potential stem cell donors.

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Indian researchers develop 3D bioprinted cartilage The Hindu The bioink has high concentration of bone-marrow derived cartilage stem cells, silk proteins and a few factors. The chemical composition of the bioink supports cell growth and long-term survival of the cells. The cartilage developed in the lab has ...

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Uproxx knows that science, technology, engineering, and math (STEM) disciplines are driving the future of this planet forward. Every day, we see new ideas, fresh innovations, and bold trailblazers in these fields. Follow us this month as we highlight how STEM is shaping the culture of NOW.

Placentas, umbilical cords pretty much anything that comes out of a womans body is awesome in science speak. Stem cells are the master cells of the body, just waiting to help you out when you get sick. Theyre your own personal repair kit, but, like anything, time kind of screws them up. They become damaged or mutated thanks to environmental factors and the aging process and one day, they lose their incredible healing abilities altogether.

The good news is, science has finally tapped into the potential of stem cell research and, in doing so, scientists have found a solution for all that wasted power: babies. Yes, babies are disgusting blobs that poop, eat, and slobber their parents to an early grave, but those little devils also just happen to have a whole army of brand new stem cells still in their original packaging. The key is to get them before they sell out. (Im starting to equate body parts with consumerism and its getting creepy so Ill stop now.)

Placenta blood, placenta tissue, and cord blood are three sources of stem cells doctors are urging new parents to consider saving after the mom gives birth. They provide a range of cool benefits from treating certain forms of cancer to helping people heal from spinal cord injuries and they can be cryogenically frozen to help a body out whenever it needs some extra healing power. And yes, some people do eat them. Google it, there are recipes.

But while the placenta party has been raging for a while now, theres a new method of extracting stem cells that can be done all the way up into a persons teen years, and all it takes is a quick trip to the dentist. Tooth banking has become the latest way people are choosing to cryogenically secure their gene sequence.

In 2013, Songtao Shi, a dentist, was researching regenerative dentistry in a lab when Shi witnessed something extraordinary. He discovered that when you get a cavity, the dentin the inner, hard layer of your tooth that protects the nerve and pulp from exposure builds up. Basically, your tooth tries to protect itself by making more organic matter.

This led Shi to conclude that stem cells did, in fact, exist in teeth. A bit more study found that while stem cells in adult molars were able to create more dentin which is great if you want to re-grow lost teeth instead of paying a fortune for an implant baby teeth, or SHED cells (stem cells from human exfoliated deciduous teeth) contained a whole different set of code.

While cord blood and placenta tissue contain Hematopoietic stem cells which have been used for decades to treat over 80 different diseases, SHED cells contain mesenchymal stem cells which differentiate into nerve cells as well as bone, cartilage, muscle, and fat. Cord blood contains mesenchymal stem cells too, but according to Shis research, SHED cells were able to create something unusual, dentin osteogenic material a material thats not quite dentin, not quite bone but full of possibilities like the ability to reconstruct bone.

Extracting dental stem cells is a complicated and sensitive process. First, the soft tissue has to be extracted, then it has to be disinfected (spoiler alert: your mouth is a cesspool of germs). Scientists then drill through the enamel and dentin to get to the pulp of the tooth where all the stem cells like to hide out. They take the pulp out, digest it with an enzyme, and culture the cells.

Its a lot of work, but the payoff is huge. Even tiny bits of dental pulp can carry hundreds of millions of stem cells.

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26 May 2017

Could tyrosine kinase inhibitors, a standard tool of cancer treatment, help people with amyotrophic lateral sclerosis? Converging evidence suggests that this drug class may slow ALS progression, perhaps through multiple mechanisms. In the May 24 Science Translational Medicine, researchers led by Haruhisa Inoue at Kyoto University, Japan, report that numerous different inhibitors of the tyrosine kinases Src and c-Abl improve the survival of motor neurons from ALS patients. The compounds act by stimulating autophagy, which accelerates the removal of toxic proteins. One of the most potent inhibitors, bosutinib, boosted motor neuron survival by 50 percent and modestly lengthened the lives of ALS model mice, the authorsreport.

In related news, researchers recently reported positive findings from a Phase 3clinical trial of another tyrosine kinase inhibitor, masitinib, at the European Network for the Cure of ALS (ENCALS) annual meeting, held May 18 to 20 in Ljubljana, Slovenia. This inhibitor, which is approved to treat tumors in animals but not people, reportedly doused neuroinflammation in the spinal cord. Patients on the drug maintained motor abilities four months longer than did those on placebo, a statistically significant improvement. AB Science in Paris, the manufacturer, has applied to the European Medicines Agency for approval to use the drug in people, and is planning to start another Phase 3trial this year before applying for approval from the U.S. Food and DrugAdministration.

The data suggest that tyrosine kinase inhibitors might help in other neurodegenerative diseases such as Alzheimers and Parkinsons, which also accumulate toxic proteins and cause neuroinflammation, said Charbel Moussa at Georgetown University, Washington, D.C. He noted that many of these compounds are already FDA-approved for other conditions, and can be used at much lower doses for neurodegenerative disease than for cancer. These drugs represent a promising alternative to antibody and vaccination strategies, he told Alzforum. He was not involved in either of thesestudies.

ALS in a Dish. Stem cells derived from people with familial ALS differentiate into neurons in culture that express motor neuron markers HB9, ChAt, and SMI-32. Nuclei are stained blue. [Courtesy of Science TranslationalMedicine/AAAS.]

The need for new drugs for ALS is immense. In this devastating disease, spinal motor neurons wither, robbing people of motor control and killing them typically within three to five years. Approved treatments are limited to riluzoleand edaravone, which was just approved in the U.S. this month (see May 2017 news). Both modestly slow functional decline, though efficacy data for edavarone remains sparse. Researchers are still seeking betteroptions.

To cast a wider net, Inoue and colleagues screened 1,416 compounds that are either approved for human use or in clinical trials. First author Keiko Imamura generated induced pluripotent stem cells (iPSCs) from a single ALS patient who carried a SOD1 mutation. The authors differentiated these cells into spinal motor neurons and cultured them for seven days, added the compounds, and assessed survival one week later. In this screen, 27 compounds boosted survival more than three standard deviations above that of untreated cells. Half of these compounds targeted the Src/c-Abl signaling pathway. These cytosolic tyrosine kinases participate in numerous cellular processes and are implicated in cancer. To confirm these enzymes mediated the drug effect, the authors knocked down Src and c-Abl with short interfering RNAs, and again saw improved motor neuronsurvival.

Among the hits, the authors selected bosutinib for follow up. This drug is approved to treat chronic myelogenous leukemia, directly inhibits Src and c-Abl, and acts at lower doses than the other compounds in the screen. Bosutinib normalized autophagy in the diseased motor neurons. Compounds that blocked autophagy weakened the protective benefits of bosutinib, suggesting this was its mechanism of action. In keeping with this, other known autophagy boosters, such as rapamycin, also improved motor neuron survival. As might be expected, revving up autophagy cleaned up deposits of misfolded, toxic SOD1. The authors did not detail how inhibition of Src and c-Abl stimulated autophagy, but other work provides clues. Moussa and colleagues have reported that c-Abl inhibition activates the ubiquitin ligase parkin, which then interacts with autophagy proteins such as beclin-1 to stimulate degradation of proteins including A and -synuclein (see Lonskaya et al., 2013; Lonskaya et al., 2014; Wenqiang et al., 2014). A sister compound to bosutinib, nilotinib, is currently in Phase 2 trials for PDand ADthat Moussa and colleagues at Georgetown are running (see Nov 2015 conference news).

Only 2 percent of people with ALS carry SOD1 mutations. What about other forms of the disease? To expand their study, the authors generated motor neurons from three ALS patients with TDP-43 mutations, three with C9ORF72 expansions, and three with sporadic disease. Most people with ALS, regardless of their mutation status, accumulate misfolded TDP-43, and C9ORF72 is the most common familial mutation. In this study, bosutinib lowered levels of misfolded TDP-43 and poly dipeptide repeats formed from the C9ORF72 expansion; it also improved survival in all cell lines save for one from a sporadiccase.

Next, the authors tested bosutinib in the SOD1-G93A mouse model of ALS. These animals become paralyzed at four and die by six months of age. The authors injected a single dose, 5 mg/kg/day, intraperitoneally for six weeks beginning at two months of age. Src and c-Abl activity in the spinal cord was cut in half, indicating target engagement. Treated mice accumulated slightly less misfolded SOD1 and had about three times as many surviving motor neurons in their spinal cords as untreated ones. Nevertheless, treatment delayed disease onset by only 11 days and extended survival by just eightdays.

Why didnt the drug work better in mice, given the promising in vitro data? Nonneuronal cells such as astrocytes contribute to ALS pathology, but Inoues screen did not test for effects of bosutinib on these cells (e.g. Oct 2014 news; Nov 2014 news). In an email to Alzforum, Inoue also suggested that bosutinib could be optimized to better enter the brain and avoid potential off-target effects. Peter Davies at the Feinstein Institute for Medical Research in Manhasset, New York, pointed out that tyrosine kinase inhibitors such as bosutinib are typically not specific for c-Abl. I would like to see pharma make more specific compounds, because then we would learn if the key factor really is c-Abl, rather than another kinase, and there would be fewer off-target effects, Davies wrote to Alzforum. He acknowledged that making specific c-Abl inhibitors is a challenging task, and that companies have tried and abandoned some past efforts for lack ofsuccess.

The findings from bosutinib and nilotinib complement those for masitinib. This veterinary drug seems to act mostly on immune cells. Preclinical studies suggested masitinib inhibits the tyrosine kinases CSF-1R and C-kit in microglia, macrophages, and mast cells, circulating white blood cells that trigger allergic and inflammatory reactions. In animal models, masitinib prevents microgliosis and astrogliosis in the spinal cord, as well as the infiltration of mast cells and macrophages into neuromuscular junctions (see Trias et al., 2016). This provides a rational basis for the protective effects of masitinib in delaying neuromuscular junction denervation. However, more research is needed to understand the detailed mechanism of action of the drug, Luis Barbeito at the Pasteur Institute of Montevideo, Uruguay, wrote to Alzforum. Barbeito presented preclinical data on masitinib atENCALS.

In the Phase 3 trial, 394 patients from nine countries took either 4.5 mg/kg masitinib, 3 mg/kg, or placebo for nearly a year. By prespecified plan, the researchers stratified participants into fast progressors (those who declined more than 1.1 point per month on the revised ALS Functional Rating Scale) and normal progressors. About 85 percent of the participants were normal progressors. Among this group, those taking 4.5 mg/kg masitinib declined 3.4 points less on the ALSFRS-R than the placebo group over the course of the study. This translated to 27 percent less functional decline over this time period, a clinically meaningful difference, according to Jesus Mora at Hospital Carlos III in Madrid, who presented the clinical trial findings at ENCALS. Treated participants maintained greater lung capacity and reported better quality of life than the placebo group. They lasted 20 months before their disease progressed nine points or more on the ALSFRS-R, compared with 16 months for those on placebo. Participants who took the lower 3 mg/kg dose also reported better quality of life, but their trend toward slower functional decline did not reachsignificance.

Other data hinted that the drug was most effective when given at an early stage of disease. When normal and fast progressors were combined, the 4.5 mg/kg dose only slowed decline in those who had had the disease for less than two years. Fast progressors may need earlier treatment, Morasuggested.

The safety profile was acceptable, with no surprises cropping up, the researchers said. The treatment group experienced more serious adverse events than the placebo group. These were scattered across different organ systems and did not fall into any pattern. For oncology use, tyrosine kinase inhibitors are normally given at higher doses, from 6 to 12 mg/kg, with no serious safety issues, the researchers noted.Madolyn BowmanRogers

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"The practice of helping babies by providing stem cells at birth has been around for a long time; it makes sense for the sickest infants," said Anup Katheria, MD, director of the Neonatal Research Institute. "We're focused on producing evidence that shows the benefits. We think this could become the foundation for practice-changing birthing techniques, transforming outcomes for the most critical of newborns nationwide."

As a California-based public/private cord blood banking company and with a strong research focus, StemCyte stands ready to help efficiently and effectively to support the partnership with Sharp HealthCare to educate expecting parents of their options, to ensure the information is delivered accurately and consistently, and to collect the cells in cord blood and process and store them with the highest quality standards in the industry.

"We are excited to work with the Sharp Mary Birch Neonatal Research Institute, and we are looking forward to maximizing the capacity for cord-blood banking. Residents of California and people around the world will benefit from the research and increased availability of umbilical cord blood stem cell transplant units." said Jonas C. Wang, Ph.D., CEO/ Chairman of StemCyte Group.

About StemCyte StemCyte's rich history started with a mission of being dedicated to helping the world's physicians save more lives by providing high quality, safe and effective stem cell transplantation and therapy to all patients in need. Located in the US, India and Taiwan, StemCyte has supplied over 2100 cord blood products for over 40 life-threatening diseases to over 300 leading worldwide transplant centers. StemCyte is actively involved in the development of stem cell therapies. StemCyte was the first to donate umbilical cord blood units (UCB) to Dr. Jaing of Chung Gung Memorial Hospital for his clinical trial to use UCB to treat and cure Beta Thalassemia. More excitingly is the work and accomplishments of Prof Wise Young, MD, PhD. Prof. Young has completed Phase II clinical trials on patients with chronic spinal cord injury with UCB and the results are extremely encouraging. StemCyte is chosen by the US Department of Health and Human Services to help establishing a public National Cord Blood Inventory. Its headquarters are located in Baldwin Park, CA. To learn more visit http://www.StemCyte.com.

For more information call 626.646.2500

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May 26, 2017 14:28 ET | Source: Center for the Advancement of Science in Space

Kennedy Space Center, FL, May 26, 2017 (GLOBE NEWSWIRE) -- The SpaceX Falcon 9 vehicle is slated to launch its 11thcargo resupply mission (CRS-11) to the International Space Station (ISS) no earlier than June 1, 2017 from Kennedy Space Center Launch Complex 39A. Onboard the Falcon 9 launch vehicle is the SpaceX Dragon spacecraft, which will carry more than 40 ISS U.S. National Laboratory sponsored experiments. This mission will showcase the breadth of research possible through the ISS National Laboratory, as experiments range from the life and physical sciences, Earth observation and remote sensing, and a variety of student-led investigations. Below highlights the investigations as part of the SpaceX CRS-11 mission:

ADVANCED COLLOIDS EXPERIMENT-TEMPERATURE CONTROLLED-6 (ACE-T-6)

Matthew Lynch, Procter & Gamble (West Chester, OH)

Implementation Partner: NASA Glenn Research Center and Zin Technologies, Inc.

Colloids are suspensions of microscopic particles in a liquid, and they are found in products ranging from milk to fabric softener. Consumer products often use colloidal gels to distribute specialized ingredients, for instance droplets that soften fabrics, but the gels must serve two opposite purposes: they have to disperse the active ingredient so it can work, yet maintain an even distribution so the product does not spoil. Advanced Colloids Experiment-Temperature-6 (ACE-T-6) studies the microscopic behavior of colloids in gels and creams, providing new insight into fundamental interactions that can improve product shelf life.

EFFICIENCY OF VERMICOMPOSTING IN A CLOSED SYSTEM (NANORACKS-NDC-BMS-VERICOMPOSTING)

Bell Middle School (Golden, CO)

Implementation Partner: NanoRacks

Vermicomposting, or using worms to break down food scraps, is an effective way to reduce waste and obtain a nutrient-rich fertilizer for plants. The NanoRacks-NDC-Bell Middle School-Efficiency of Vermicomposting in a Closed System (NanoRacks-NDC-BMS-Vermicomposting) investigation is a student-designed project that studies whether red wiggler worms, a species of earthworm, are able to produce compost in space. Results are used to study the potential for composting as a form of recycling on future long-duration space missions.

FUNCTIONAL EFFECTS OF SPACEFLIGHT ON CARDIOVASCULAR STEM CELLS (CARDIAC STEM CELLS)

Dr. Mary Kearns-Jonker, Loma Linda University (Loma Linda, CA)

Implementation Partner: BioServe Space Technologies

Functional Effects of Spaceflight on Cardiovascular Stem Cells (Cardiac Stem Cells) investigates how microgravity alters stem cells and the factors that govern stem cell activity, including physical and molecular changes. Spaceflight is known to affect cardiac function and structure, but the biological basis for this is not clearly understood. This investigation helps clarify the role of stem cells in cardiac biology and tissue regeneration. In addition, this research could confirm the hypothesis that microgravity accelerates the aging process.

MULTIPLE USER SYSTEM FOR EARTH SENSING (MUSES)

Paul Galloway, Teledyne Brown Engineering (Huntsville, AL)

Implementation Partner: Teledyne Brown Engineering

Teledyne Brown Engineering developed the Multiple User System for Earth Sensing (MUSES), an Earth imaging platform, as part of the companys new commercial space-based digital imaging business. MUSES hosts earth-viewing instruments (Hosted Payloads), such as high resolution digital cameras, hyperspectral imagers, and provides precision pointing and other accommodations. It hosts up to four instruments at the same time, and offers the ability to

change, upgrade, and robotically service those instruments. It also provides a test bed for technology demonstration and technology maturation by providing long-term access to the space environment on the ISS.

NANORACKS-JAMSS-2LAGRANGE-1

Tomohiro Ichikawa, Lagrange Corp. (Tokyo, Japan)

Implementation Partner: NanoRacks

Spaceflight affects organisms in a wide range of ways, from a reduction in human bone density to changes in plant root growth. NanoRacks-JAMSS-2 Lagrange-1 helps students understand potential spaceflight-related changes by exposing plant seeds to microgravity, and then germinating and growing them on Earth. The plants are compared with specimens grown from seeds that remained on the ground. The investigation also connects students to the space program by sending their photographic likenesses and personal messages into orbit. This connection inspires the next generation of scientists and engineers who will work on international space programs.

NEUTRON CRYSTALLOGRAPHIC STUDIES OF HUMAN ACETYLCHOLINESTERASE FOR THE DESIGN OF ACCERERATED REACTIVATORS (ORNL-PCG)

Dr. Andrey Kovalevsky, Oak Ridge National Laboratory (Oak Ridge, TN)

Implementation Partner: CASIS

The investigative team is trying to improve our understanding of acetylcholinesterase, an enzyme essential for normal communication between nerve cells and between nerve and muscle cells. As a target of deadly neurotoxins produced by animals as venom or by man as nerve agents and pesticides, understanding the structure of acetylcholinesterase is critical to designing better antidotes to poisoning by chemicals that attack the nervous system. The Oak Ridge National Lab team plans to use the microgravity environment of space to grow large crystals of the enzyme that will be imaged back on Earth using a powerful imaging approach called neutron diffraction. Neutron diffraction yields very detailed structural information but requires much larger crystals than traditional x-ray diffraction imaging methods. The investigators hypothesize that structural images of space-grown crystals will bring us closer to more effective and less toxic antidotes for neurotoxins that bind and inhibit acetylcholinesterase.

STUDENT SPACEFLIGHTS EXPERIMENT PROGRAM MISSION 10

Dr. Jeff Goldstein, National Center for Earth and Space Science Education (Washington, D.C.)

Implementation Partner: NanoRacks

The Student Spaceflight Experiments Program (SSEP) provides one of the most exciting educational opportunities available: student-designed experiments to be flown on the International Space Station. The NanoRacks-National Center for Earth and Space Science Education-Odyssey (NanoRacks-NCESSE-Odyssey) investigation contains 24 student experiments, including microgravity studies of plant, algae and bacterial growth; polymers; development of multi-cellular organisms; chemical and physical processes; antibiotic efficacy; and allergic reactions. The program immerses students and teachers in real science, providing first-hand experience conducting scientific experiments and connecting them to the space program.

SYSTEMIC THERAPY OF NELL-1 FOR OSTEOPOROSIS (RODENT RESEARCH-5)

Dr. Chia Soo, University of California at Los Angeles (Los Angeles, CA)

Implementation Partner: NASA Ames Research Center and BioServe Space Technologies

Astronauts living in space for extended durations experience bone density loss, or osteoporosis. Currently, countermeasures include daily exercise designed to prevent bone loss from rapid bone density loss deterioration. However, in space and on Earth, therapies for osteoporosis cannot restore bone that is already lost. The Systemic Therapy of NELL-1 for Osteoporosis (Rodent Research-5) investigation tests a new drug on rodents that can both rebuild bone and block further bone loss, improving health for crew members in orbit and people on Earth. Dr. Soos laboratory has been funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases within the National Institutes of Health. This experiment builds on those previous research investigations.

THE EFFECT OF MICROGRAVITY ON TWO STRAINS OF BIOFUEL PRODUCING ALGAE WITH IMPLICATIONS FOR THE PRODUCTION OF RENEWABLE FUELS IN SPACE-BASED APPLICATIONS

Chatfield High School (Littleton, CO)

Implementation Partner: NanoRacks

Algae can produce both fats and hydrogen, which can each be used as fuel sources on Earth and potentially in space. NanoRacks-National Design Challenge-Chatfield High School-The Effect of Microgravity on Two Strains of Biofuel Producing Algae with Implications for the Production of Renewable Fuels in Space Based Applications (NanoRacks-NDC-CHS-The Green Machine) studies two algae species to determine whether they still produce hydrogen and store fats while growing in microgravity. Results from this student-designed investigation improve efforts to produce a sustainable biofuel in space, as well as remove carbon dioxide from crew quarters.

TOMATOSPHERE-II

Ann Jorss, First the Seed Foundation (Alexandria, VA)

Implementation Partner: CASIS

Tomatosphere is a hands-on student research experience with a standards-based curriculum guide that provides students the opportunity to investigate, create, test, and evaluate a solution for a real world case study. Tomatosphere provides information about how spaceflight affects seed and plant growth and which type of seed is likely to be most suitable for long duration spaceflight. It also exposes students to space research, inspiring the next generation of space explorers. It is particularly valuable in urban school settings where students have little connection to agriculture. In its 15-year existence, the program has reached approximately 3.3 million students.

VALLEY CHRISTIAN HIGH SCHOOL STUDENT EXPERIMENTS

Valley Christian High School (San Jose, CA), in partnership with other high schools throughout the world

Implementation Partner: NanoRacks

Students at Valley Christian High School (VCHS) have a rich history of sending investigations to the ISS through its launch partner, NanoRacks. On SpaceX CRS-11, students from VCHS have partnered with other students from across the world to send 12 total experiments to the ISS National Laboratory. Investigations will range from investigating high quality food nutrients, to the fermentation of microbes, to even an investigation monitoring the growth of a special bacterial strain. The program VCHS has developed with NanoRacks allows students the opportunity to not only conceive a flight project, but learn, understand, and implement the engineering required for a successful experiment in microgravity.

Thus far in 2017, the ISS National Lab has sponsored over 75 separate experiments that have reached the station. This launch manifest adds to an impressive list of experiments from previous missions in 2017 to include; stem cell studies, cell culturing, protein crystal growth, external platform payloads, student experiments, Earth observation and remote sensing. To learn more about those investigations and other station research, visit http://www.spacestationresearch.com.

# # #

About CASIS: The Center for Advancement of Science in Space (CASIS) is the non-profit organization selected to manage the ISS National Laboratory with a focus on enabling a new era of space research to improve life onEarth. In this innovative role, CASIS promotes and brokers a diverse range of research inlife sciences,physical sciences,remote sensing,technology development,andeducation.

Since 2011, the ISS National Lab portfolio has included hundreds of novel research projects spanning multiple scientific disciplines, all with the intention of benefitting life on Earth.. Working together with NASA, CASIS aims to advance the nations leadership in commercial space, pursue groundbreaking science not possible on Earth, and leverage the space station to inspire the next generation.

About the ISS National Laboratory: In 2005, Congress designated the U.S. portion of the International Space Station as the nation's newest national laboratory to maximize its use for improving life on Earth, promoting collaboration among diverse users, and advancing STEM education. This unique laboratory environment is available for use by other U.S. government agencies and by academic and private institutions, providing access to the permanent microgravity setting, vantage point in low Earth orbit, and varied environments of space.

# # #

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Stranger saves life of woman with stem cell transplant FOX31 Denver DENVER -- This is a terrible statistic. Eighty percent of blood cancer patients in need of stem cell or bone marrow transplant are not able to get one, in part because they can't find a match. But you can help change that and save a life by registering ...

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The late actor Telly Savalas said it best: Were all born bald, baby.

And bald CAN be beautiful.

But for many follicly-challenged folks, news out of UC San Francisco this week offers some hope of finally having a bad hair day.

In experiments in mice, researchers there have discovered that regulatory T cells (Tregs; pronounced tee-regs), a type of immune cell associated with controlling inflammation, directly trigger stem cells in the skin to promote healthy hair growth.

Without these immune cells as partners, the researchers found, the stem cells cannot regenerate hair follicles, leading to baldness.

Our hair follicles are constantly recycling: when a hair falls out, the whole hair follicle has to grow back, said Dr. Michael Rosenblum, an assistant professor of dermatology at UCSF and senior author on the new paper.

This has been thought to be an entirely stem cell-dependent process, but it turns out Tregs are essential. If you knock out this one immune cell type, hair just doesnt grow.

In other words: no Tregs, no tresses.

The new study appeared online Friday in Cell, a journal that publishes peer-reviewed articles reporting findings of unusual significance in any area of experimental biology.

For 35 million U.S. men and 21 million women who are experiencing hair loss, according to Statistic Brain Research Institute,the UCSF report would probably qualify as significant.

The study suggests that defects in Tregs could be responsible for alopecia areata, a common autoimmune disorder that causes hair loss, and could potentially play a role in other forms of baldness, including male pattern baldness, Rosenblum said.

And since the same stem cells are responsible for helping heal the skin after injury, the researchers note, the study raises the possibility that Tregs may play a key role in wound repair as well.

Normally, the researchers say, Tregs act as peacekeepers and diplomats, informing the rest of the immune system of the difference between friend and foe. When Tregs dont function properly, people may develop allergies to harmless substances like peanut protein or cat dander, or suffer from autoimmune disorders in which the immune system turns on the bodys own tissues.

Like other immune cells, most Tregs reside in the bodys lymph nodes, but some live permanently in other tissues, where researcher say they seem to have evolved to assist with local metabolic functions as well as playing their normal anti-inflammatory role. In the skin, for example, Rosenblum and colleagues have previously shown that Tregs help establish immune tolerance to healthy skin microbes in newborn mice, and these cells also secrete molecules that help heal wounds into adulthood.

Rosenblum wanted to better understand the role of these resident immune cells in skin health. To do this, he and his team developed a technique for temporarily removing Tregs from the skin. But when they shaved patches of hair from these mice to make observations of the affected skin, they made a surprising discovery.

We quickly noticed that the shaved patches of hair never grew back, and we thought, Hmm, now thats interesting, Rosenblum said. We realized we had to delve into this further.

The researchers including UCSF postdoctoral fellow and first author Niwa Ali believe a betterunderstanding of Tregs critical role in hair growth could lead to improved treatments for hair loss more generally and have implications for alopecia areata, an autoimmune disease that causes patients to lose hair in patches from their scalp, eyebrows, and faces.

For many other baldly confident folks, however, Fridays findings may just warrant a shrug.As they say, No hair, dont care.

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Baldness treatment discovered at UCSF - The Mercury News

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UW Health trial involves injecting... More Headlines

MADISON, Wis. - Doctors at UW Health are involved in a clinical trial using stem cells for the treatment of heart failure.

The CardiAMP therapy involves withdrawing a patients bone marrow. The bone marrow is then processed on-site to separate the stem cells from the plasma. The patients own stem cells are then injected into damaged areas of the heart using a catheter.

It is hopeful that we can improve things. I dont think we can necessarily cure the damage, but I think we can improve things, said Dr. Amish Raval, director of cardiovascular clinical research at UW Health.

The CardiAMP Heart Failure Trial is a phase III study that will eventually enroll up to 260 patients. For the first 10 patients, UW Health is one of three sites nationwide performing the procedure.

I figured it was possibly going to do something good for me, said Dan Caulfield, a Madison man enrolled in the study.

Caulfield, who is 81 years old, has had three heart attacks.

I was 46 years old and had a heart attack. It was called a fatal heart attack in those days, Caulfield said. I had two more heart attacks in 2002, and since then it has been sort of downhill.

Improving the quality of life of individuals with heart failure is a goal of the CardiAMP therapy.

There is about a 50 percent five-year mortality associated with this condition and those five years can be awfully tough on these folks because they have a lot of problems with shortness of breath, weakness and sometimes chest discomfort while walking. So it is not just a matter of quantity of life, it is also a quality of life issue, Raval said.

The procedure involves a very targeted injection of stem cells into the area near where the heart is damaged.

We create a targeted map and based on that targeted map we have a really clear sense of where the damage is. Then it is my task to go in and try to get into the adjacent border areas, Raval said.

In the U.S. there are approximately 6.5 million people living with heart failure. According to the American Heart Association, that number is expected to rise by 46 percent by the year 2030.

This is one of the few pivotal trials in the United States that is really, I think, going to pave the way for future studies, Raval said.

The outcome of the CardiAMP trial will be measured by any change in distance during a six-minute walk 12 months after an initial baseline measurement is taken.

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A new cause of baldness has been accidentally discovered by scientists in the US in a breakthrough that could help develop a way to regrow hair.

The researchers were investigating the role played by anti-inflammatory immune cells called Tregs in skin health generally.

They found a way to temporarily remove the Tregs from the skin of laboratory mice, who had been shaved to allow the effects to be observed.

But the scientists then noticed something unexpected the hairfailed to grow back.

Previously it was thought that stem cells cause hairs to regrow after they fall out, but the team discoveredthat this only happens if Tregs are present.

One of the scientists, Professor Michael Rosenblum, an immunologist and dermatologist at University of California San Francisco, said: Our hair follicles are constantly recycling. When a hair falls out, the whole hair follicle has to grow back.

This has been thought to be an entirely stem cell-dependent process, but it turns out Tregs are essential.

If you knock out this one immune cell type, hair just doesn't grow.

Its as if the skin stem cells and Tregs have co-evolved, so that the Tregs not only guard the stem cells against inflammation but also take part in their regenerative work.

The stem cells rely on the Tregs completely to know when it's time to start regenerating.

The researcher believe that defects in Tregs could be responsible for the immune disease, alopecia areata, which causes hair to fall out in patches and possibly also play a part in other kinds of baldness.

The same stem cells that regrow hair are also involved in healing damage to the skin, so Tregs may also be involved in this process.

Tregs role as previously understood was mainly to regulate the immune system, helping it tell what to attack and what to leave alone.

When they malfunction it can lead to allergies to peanuts and other harmless substances or cause the immune system to attack the body.

Professor Rosenblum and colleagues had previously showed that Tregs help the immune systems of baby mice learn which skin microbes are not harmful and also that they secrete molecules that help heal wounds.

They were investigating these effects further when they noticed that patches of shaved hair on the lab mice were not regrowing.

We thought, Hmm, now thats interesting, Professor Rosenblum said. We realised we had to delve into this further.

Using sophisticated imaging techniques, the researchers were able to show that Tregs gathered around follicle stem cells at the start of the process to regrow a hair.

When Tregs were removed from the skin, this prevented the regrowth of hair but only if this was done within three days of the hair being shaved. After this time, the hair would regrow normally despite the absence of Tregs.

The cause of alopecia is poorly understood, but previous studies have showed genes associated with the condition are mostly related to Tregs. Boosting Treg function has been found to help.

Professor Rosenblum suggested further research into Tregs role could lead to improved treatments for hair loss generally and better understanding of their role in wound healing.

We think of immune cells as coming into a tissue to fight infection, while stem cells are there to regenerate the tissue after it's damaged, he said.

But what we found here is that stem cells and immune cells have to work together to make regeneration possible.

The research was described in the journal Cell.

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New baldness cause accidentally discovered by scientists could lead to hair loss treatment - The Independent

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The new research showed that adult stem cells could help beat heart failure.

A sticking plaster made from adult stem cells could be a significant step towards combatting heart failure, scientists say.

Researchers discovered that stem cells taken from a patients thigh and transplanted onto the heart led to improved heart function after one year.

Heart failure is thought to affect between 500,000 to 900,000 people in the UK. It occurs when the heart becomes too weak to efficiently pump blood around the body.

The authors of the study, published in the Journal of the American Heart Association, said the therapy was potentially a long-term solution to the problem.

They said that, promising results in the safety and functional recovery warrant further clinical follow-up and larger studies, which they hope will confirm the treatments potential.

Professor Metin Avkiran, associate medical director at the British Heart Foundation, hailed the exciting breakthrough.

He said: Heart failure is a cruel and debilitating illness affecting more than half a million people across the UK. Currently, heart failure is incurable, but stem cell-based treatments may offer new hope to people suffering from the disease.

He echoed the call for further research, saying: The study involved only a small number of patients. In order to establish the long-term safety and benefits of the exciting new treatment we would need larger studies.

Heart failure often leaves sufferers struggling for breath and exhausted while carrying out simple everyday tasks, such as eating or getting dressed.

It can be caused by several issues including heart disease, diabetes and high blood pressure, but can also be the result of an unhealthy lifestyle.

Earlier this month, it was revealed that a remarkable new technique allows adult stem cells to be used to treat burn victims.

Taking a sample of skin stem cells and spraying them onto a victims burn caused new layers of skin to form over the burn, potentially healing even severe burns within weeks.

And in January, scientists released findings showing that synthetic cardiac stem cells could be used to treat patients who had suffered a heart attack by repairing the heart muscle damage.

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Stem cell 'plaster' could help heart failure patients - The Christian Institute

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Q: How close are we to curing blood diseases with human stem cells?

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

Advances reported 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.

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, 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 donor's bone marrow and in umbilical cord blood harvested after a baby's 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. So they can provoke an attack if the transplant recipient's body registers the cells as foreign.

This response, called graft-versus-host disease, affects as many as 70 percent of bone-marrow transplant recipients soon after treatment, and 40 percent develop a chronic version of the affliction later. It kills many patients.

Rather than hunt for a donor who's a perfect match, doctors would like to use a patient's own cells to engineer the hematopoietic stem cells.

The patient's 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 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 rejection could be eliminated, physicians might also feel more confident treating blood diseases that are not immediately deadly such as sickle cell disease and immunological disorders with stem cell transplants.

One of two research teams, led by stem cell pioneer Dr. George Q. Daley of Harvard Medical School and the Dana Farber Cancer Institute, started their experiment with human "pluripotent" stem cells primitive cells capable of becoming virtually any type of mature cell.

The scientists then programmed those pluripotent stem cells to become endothelial cells, which line the inside of certain blood vessels.Using suppositions gleaned from experiments with mice, Daley said his team confected a "special sauce" of proteins that sit on a cell's DNA and program its function. When they incubated the endothelial cells in the sauce, they began producing hematopioetic stem cells.

Daley's 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 team, led by researchers from Weill Cornell Medicine's Ansary Stem Cell Institute, achieved a similar result using stem cells from the blood-vessel lining of adult mice.

But Daley cautioned that significant hurdles remain before studies like these will transform the treatment of blood diseases.

"We do know the resulting cells function like blood stem cells, but they still are at some distance, molecularly, from native stem cells," he said.

Melissa Healy, Los Angeles Times

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Andrew Duffy, Postmedia Network May 23, 2017

, Last Updated: 5:01 PM ET

Jonathan Pitre has been on a medical roller-coaster in the week since blood tests revealed that his stem cell transplant has taken root in his bone marrow.

While his white blood cell count has soared its now well within the normal range he has also suffered a series of complications that have severely tested his physical endurance.

It has been a long few days, said his mother, Tina Boileau. Hes been through hell.

Pitre, 16, is battling liver, kidney and gastrointestinal problems.

He has been diagnosed with typhlitis, a serious inflammation in part of his large intestine, that brings with it risk of a bowel perforation. He has undergone a series of x-rays and ultrasounds to check for perforations, all of which have come back negative.

At the same time, Pitre is fighting a liver infection that has caused his fever to spike, and his skin to yellow. His blood pressure has fluctuated, and his kidneys are struggling to process all of the fluids and medications that have been been pumped into his body. He hasnt been allowed to eat or drink for days to protect his damaged gastrointestinal system.

Pitre will undergo surgery Wednesday to have another central line installed so that he can be fed intravenously rather than through his existing g-tube, which sends nutrition directly to his stomach.

All of the complications have made it difficult to deliver enough medication to control Pitres pain levels, his mother said.

Its got to get better, she said.

Boileau is placing her faith in her sons new immune system, which has been rebuilt with the help of her donated stem cells. His white blood cell count is at 6.7 which is amazing, she said. And hopefully, that helps him fight everything hes going through.

A normal white blood cell count ranges from 4.0 to 11.

Pitre found out last Tuesday that the white blood cells in his system were all donor cells, which signalled that his transplant had successfully engrafted in his bone marrow. Bone marrow stem cells produce most of the bodys blood, including the white blood cells that are responsible for fighting bacteria, viruses and other pathogens.

Pitres lead physician, Dr. Jakub Tolar, said last week that the Russell teenager remains extremely fragile and susceptible to all kinds of complications. But Tolar also said the success of the transplant has established the pre-condition for his recovery.

It has now been 40 days since Pitre was infused with stem cells drawn from his mothers hip bone at the University of Minnesota Masonic Childrens Hospital.

In the next three months, doctors will be on the lookout for signs of acute graft-versus-host-disease (GVHD), a complication in which the donors white blood cells turn on the patients tissues and attack them as foreign. Last week, Pitre showed signs of a rash which can sometimes be a telltale sign of the disease, but a skin biopsy showed that the problem was not related to GVHD.

Anyone who receives stem cells from another person is at risk of developing the condition, which can range from mild to life-threatening. It commonly affects the skin, liver or gastrointestinal tract.

Pitre suffers from a severe form of epidermolysis bullosa (EB), a painful and progressive skin disease that has inflicted deep, open wounds on his body.

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'It has been a long few days': Jonathan Pitre on medical roller-coaster - Canoe

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May 24, 2017 by Collene Ferguson Jeff Biernaskies research identifies a factor essential for dermal stem cells to continuously divide during tissue regeneration. Credit: Riley Brandt, University of Calgary

Stem cell researchers at the University of Calgary have found another piece of the puzzle behind what may contribute to hair loss and prevent wounds from healing normally.

Jeff Biernaskie's research, published recently in the scientific journal npj Regenerative Medicine identifies a key signalling protein called platelet-derived growth factor (PDGF). This protein is critical for driving self-renewal and proliferation of dermal stem cells that live in hair follicles and enable their unique ability to continuously regenerate and produce new hair.

"This is the first study to identify the signals that influence hair follicle dermal stem cell function in your skin," says Biernaskie, an associate professor in comparative biology and experimental medicine at the University of Calgary'sFaculty of Veterinary Medicine, and Calgary Firefighters Burn Treatment Society Chair in Skin Regeneration and Wound Healing. Biernaskie is also a member of the Alberta Children's Hospital Research Institute.

"What we show is that in the absence of PDGF signalling hair follicle dermal stem cells are rapidly diminished because of their inability to generate new stem cells and produce sufficient numbers of mature dermal cells within the hair follicle."

Biernaskie and his team of researchers study dermal stem cells located within hair follicles. They are looking to better understand dermal stem cell function and find ways to use these cells to develop novel therapies for improved wound healing after injury, burns, disease or aging.

This study, co-authored byRaquel Gonzalez and Garrett Moffatt,shows that PDGF is key to maintaining a well-functioning stem cell population in skin. And in normal skin, if you don't have enough of it the stem cell pools start to shrink, meaning eventually the hair will no longer grow and wounds will not heal as well.

"It's an important start in terms of how we might modulate these cells towards developing future therapies that could regenerate new dermal tissue or maintain hair growth" says Biernaskie.

Biernaskie's lab is looking at the potential role of stem cells in wound healing and the potential to stimulate these cells to improve skin regeneration, as opposed to forming scars.

Explore further: Using stem cells to grow new hair

More information: Raquel Gonzlez et al. Platelet-derived growth factor signaling modulates adult hair follicle dermal stem cell maintenance and self-renewal, npj Regenerative Medicine (2017). DOI: 10.1038/s41536-017-0013-4

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