A girl of eight whose rare brain disorder is likely to lead to her death when she is in her teens is taking part in pioneering stem cell research in a bid to save others with same condition.

Lily Harrisss skin cells will first be turned into stem cells and then into brain cells by researchers at University College London as they seek treatments or a cure.

About 100 to 200 cases of BPAN beta-propeller protein-associated neurodegeneration are known worldwide, although this is believed to be an underestimate.

Children often suffer delayed development, sleep problems, epilepsy and lack of speech and their symptoms can be mistaken for other conditions.

Lily, from Luton, was diagnosed when she was five. She has very limited communication skills and uses a wheelchair. She wakes four or five times a night and needs drugs to control seizures.

However, she loves swimming and her father Simon said she has recently began singing on car journeys.

Shes laughed and giggled her way through everything, and shes been through a lot, he said.

Shes a beautiful little girl who can be quite naughty sometimes. Were giving her the best time we can while shes here. We have a beautiful little girl and its just so cruel.

Young people with BPAN develop abnormal muscle tone, symptoms of Parkinsons disease and dementia.

Mr Harriss and his wife Samantha, who work for an airline, know that as Lilys condition progresses she may have difficulty swallowing and require pain management.

Mr Harriss said: Lily can point to things she wants, she uses a little sign language and she can say a few words, like mummy, daddy, hello and goodbye.

Medical research like this for children is just absolutely vital.

We know we wont get a cure for Lily but, as parents, we need to be bigger than that. Other children might benefit through Lily. We are so proud of her.

The UCL study is being funded by 230,000 from childrens charity Action Medical Research and the British Paediatric Neurology Association. Lead researcher Dr Apostolos Papandreou hopes his research will lead to trials of treatments.

He said: The parents Ive met understandably feel devastated at the prospect of their children having a progressive disorder. However, theyre really keen to explore new avenues and participate in research projects.

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Super Foods In Skin Care FOX31 Denver Fruit Stem Cells are advanced high potency juices that take the place of petroleum by-products and fillers for boosted beauty benefits. Apple Juice, a.k.a. Malic Acid is rich in vitamins, potent malic alpha-hydroxy acids, phytonutrients, flavonoids ...

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Ottawa Sun

Jonathan Pitre battles blood, lung infections before second stem cell ... Ottawa Sun Jonathan Pitre is back in a Minneapolis hospital with blood and lung infections complications that will likely delay his second stem-cell transplant. Pitre and ... and more »

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Ottawa Citizen

Jonathan Pitre battles blood, lung infections before second stem cell transplant Ottawa Citizen People with RDEB have a fault in the gene responsible for a specific kind of collagen that connects the outer layer of skin, the epidermis, with those below it. The clinical trial seeks a biochemical correction to that fault. If the transplant works ...

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Adult stem cells collected directly from human fat are more stable than other cells -- such as fibroblasts from the skin -- and have the potential for use in anti-aging treatments, according to researchers from the Perelman School of Medicine at the University of Pennsylvania. They made the discovery after developing a new model to study chronological aging of these cells. They published their findings this month in the journal Stem Cells.

Chronological aging shows the natural life cycle of the cells -- as opposed to cells that have been unnaturally replicated multiple times or otherwise manipulated in a lab. In order to preserve the cells in their natural state, Penn researchers developed a system to collect and store them without manipulating them, making them available for this study. They found stem cells collected directly from human fat -- called adipose-derived stem cells (ASCs) -- can make more proteins than originally thought. This gives them the ability to replicate and maintain their stability, a finding that held true in cells collected from patients of all ages.

"Our study shows these cells are very robust, even when they are collected from older patients," said Ivona Percec, MD, director of Basic Science Research in the Center for Human Appearance and the study's lead author. "It also shows these cells can be potentially used safely in the future, because they require minimal manipulation and maintenance."

Stem cells are currently used in a variety of anti-aging treatments and are commonly collected from a variety of tissues. But Percec's team specifically found ASCs to be more stable than other cells, a finding that can potentially open the door to new therapies for the prevention and treatment of aging-related diseases.

"Unlike other adult human stem cells, the rate at which these ASCs multiply stays consistent with age," Percec said. "That means these cells could be far more stable and helpful as we continue to study natural aging."

ASCs are not currently approved for direct use by the Food and Drug Administration, so more research is needed. Percec said the next step for her team is to study how chromatin is regulated in ASCs. Essentially, they want to know how tightly the DNA is wound around proteins inside these cells and how this affects aging. The more open the chromatin is, the more the traits affected by the genes inside will be expressed. Percec said she hopes to find out how ASCs can maintain an open profile with aging.

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Stem cells collected from fat may have use in anti-aging treatments ... - Science Daily

Human stem cells grown on Kyoto University's "fiber-on-fiber" culturing system(Credit: Kyoto University)

Mighty promising as they are, stem cells certainly aren't easy to come by. Recent scientific advances have however given their production a much-needed boost, with a Nobel-prize winning technology that turns skin cells into embryonic-like stem cells and another that promises salamander-like regenerative abilities being just a couple of examples. The latest breakthrough in the area comes from Japanese researchers who have developed a nanofiber matrix for culturing human stem cells, that they claim improves on current techniques.

The work focuses on human pluripotent stem cells (hPSCs), which have the ability to mature into any type of adult cell, be they those of the eyes, lungs or hair follicles. But that's assuming they can be taken up successfully by the host. Working to improve the odds on this front, scientists have been exploring ways of culturing pluripotent stem cells in a way that mimics the physiological conditions of the human body, allowing them to grow in three dimensions rather than in two dimensions, as they would in a petrie dish.

Among this group is a team from Japan's Kyoto University, which has developed a 3D culturing system it says outperforms the current technologies that can only produce low quantities of low-quality stem cells. The system consists of gelatin nanofibers on a synthetic mesh made from biodegradable polyglycolic acid, resulting in what the researchers describe as a "fiber-on-fiber" (FF) matrix.

The team found that seeding human embryonic stem cells onto this type of matrix saw them adhere well, and enabled an easy exchange of growth factors and supplements. This led to what the researchers describe as robust growth, with more than 95 percent of the cells growing and forming colonies after just four days of culture.

And by designing a special gas-permeable cell culture bag, the team also demonstrated how they could scale up the approach. This is because several of the cell-loaded matrices can be folded up and placed inside the bag, with testing showing that this approach yielded larger again numbers of cells. What's more, the FF matrix could even prove useful in culturing other cell types.

"Our method offers an efficient way to expand hPSCs of high quality within a shorter term," the team writes in its research paper. "Additionally, as nanofiber matrices are advantageous for culturing other adherent cells, including hPSC-derived differentiated cells, FF matrix might be applicable to the large-scale production of differentiated functional cells for various applications."

The research was published in the journal Biomaterials.

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When she was just 11 months old, Billie Sue Wozniaks daughter Juno was diagnosed with type 1 diabetes, an autoimmune disease that affects 1.25 million people and approximately 200,000 children under age 20 in the United States.

The disease had affected several members of Billie Sues family, including her uncle, who passed away at the age of 30.

My first thought was, Her life is going to be short, the 38-year-old from Reno, Nevada recalled. The more that I learned, the more I found that many people with type 1 live longer and the treatment advances are really exciting.

While looking for treatments, Wozniak learned about encapsulation therapy, in which an encapsulated device containing insulin-producing islet cells derived from stem cells is implanted under the skin. The encapsulation device is designed to protect the cells from an autoimmune attack and may help people produce their own insulin.

After learning of the therapy through JDRF, Wozniak saw an ad on Facebook for Store-A-Tooth, a company that offers dental stem cell banking. She decided to move forward with the stem cell banking, just in case the encapsulation device became an option for Juno.

In March 2016, a dentist extracted four of Junos teeth, and sent them to a lab so her stem cells could be cryopreserved. Wozniak plans to bank the stem cells from Junos molars as well.

Its a riskI dont know for sure if it will work out, Wozniak said.

Dental stem cells: a future of possibilities

For years, stem cells from umbilical cord blood and bone marrow have been used to treat blood and bone marrow diseases, blood cancers and metabolic and immune disorders.

Although there is the potential for dental stem cells to be used in the same way, researchers are only beginning to delve into the possibilities.

Dental stem cells are not science fiction, said Dr. Jade Miller, president of the American Academy of Pediatric Dentistry. I think at some point in time, were going to see dental stem cells used by dentistson a daily practice.

Dental stem cells have the potential to produce dental tissue, bone, cartilage and muscle. They may be used to repair cavities, fix a tooth damaged from periodontal disease or bone loss, or even grow a tooth instead of using dental implants.

In fact, stem cells can be used to repair cracks in teeth and cavities, according to a recent mouse study published in the journal Scientific Reports.

Theres also some evidence that dental stem cells can produce nerve tissue, which might eliminate the need for root canals. A recent study out of Tufts University found that a collagen-based biomaterial used to deliver stem cells to the inside of damaged teeth can regenerate dental pulp-like tissues.

Dental stem cells may even be able to treat neurological disorders, spinal cord and traumatic brain injuries.

I believe those are the kinds of applications that will be the first uses of these cells, said Dr. Peter Verlander, Chief Scientific Officer for Store-A-Tooth.

When it comes to treating diseases like type 1 diabetes, dental stem cells also show promise. In fact, a study in the Journal of Dental Research found that dental stem cells were able to form islet-like aggregates that produce insulin.

Unlike umbilical cord blood where theres one chance to collect stem cells, dental stem cells can be collected from several teeth. Also, gathering stem cells from bone marrow requires invasive surgery and risk, and it can be painful and costly.

The stem cells found in baby teeth, known as mesenchymal cells, are similar to those found in other parts of the body, but not identical.

There are differences in these cells, depending on where they come from, Verlander said.

Whats more, mesenchymal stem cells themselves differ from hematopoietic, or blood-forming stem cells. Unlike hematopoietic stem cells, mesenchymal stem cells can expand.

From one tooth, we expect to generate hundreds of billions of cells, Verlander said.

Yet the use of dental stem cells is not without risks. For example, theres evidence that tumors can develop when stem cells are transplanted. Theres also a chance of an immune rejection, but this is less likely if a person uses his own stem cells, Miller said.

The process for banking stem cells from baby teeth is relatively simple. A dentist extracts the childs teeth when one-third of the root remains and the stem cells are still viable. Once the teeth are shipped and received, the cells are extracted, grown and cryopreserved.

Store-A-Tooths fees include a one-time payment of $1,749 and $120 per year for storage, in addition to the dentists fees for extraction.

For families who are interested in banking dental stem cells, they should know that theyre not necessarily a replacement for cord blood banking or bone marrow stem cells.

Theyre not interchangeable, we think of them as complementary, Verlander said.

Although the future is unclear for Junowho was born in 2008her mom is optimistic that shell be able to use the stem cells for herself and if not, someone else.

Ultimately, however, Wozniak hopes that if dental stem cells arent the answer, there will be a biological cure for type 1 diabetes.

I hold out hope that somewhere, someone is going to crack the code, she said.

Julie Revelant is a health journalist and a consultant who provides content marketing and copywriting services for the healthcare industry. She's also a mom of two. Learn more about Julie at revelantwriting.com.

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Scientists discover an unexpected influence on dividing stem cells' fate ScienceBlog.com (blog) When most cells divide, they simply make more of themselves. But stem cells, which are responsible for repairing or making new tissue, have a choice: They can generate more stem cells or differentiate into skin cells, liver cells, or virtually any of ...

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Induced pluripotent stem cells don't increase genetic mutations Science Daily Using skin cells from the same donor, they created genetically identical copies of the cells using both the iPSC and the subcloning techniques. They then sequenced the DNA of the skin cells as well as the iPSCs and the subcloned cells and determined ...

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Science Daily

Scientists discover an unexpected influence on dividing stem cells ... Science Daily When it divides, a stem cell has a choice: produce more stem cells or turn into the specific types of cells that compose skin, muscle, brain, or other tissue. and more »

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In 2016, scientists in Japan revealed the birth of mice from eggs made from a parent's skin cells, and many researchers believe the technique could one day be applied to humans.

The process, called in vitro gametogenesis, allows eggs and sperm to be created in a culture dish in the lab.

Though most scientists agree we're still a long way off from doing it clinically, it's a promising technology that has the potential to replace traditional in vitro fertilization to treat infertility.

If and when this process is successful in humans, the implications would be immense, but scientists are now raising legal and ethical questions that need to be addressed before the technology becomes a reality.

In vitro gametogenesis, or IVG, is similar to IVF -- in vitro fertilization -- in that the joining of egg and sperm takes place in a culture dish.

Trounson believes IVG can provide hope for couples when IVF is not an option.

This procedure can "help men or women who have no gametes -- no sperm or eggs," said Trounson, a renowned stem cell scientist best known for developing human IVF with Carl Wood in 1977.

Another potential benefit with IVG is that there is no need for a woman to receive high doses of fertility drugs to retrieve her eggs, as with traditional IVF.

In addition, same-sex couples would be able to have biological children, and people who lost their gametes through cancer treatments, for instance, would have a chance at having biological children.

In theory, a single woman could also conceive on her own, a concept that Sonia M. Suter, professor of law at George Washington University, calls "solo IVG." She points out that it comes with some risk, as there will be less genetic variety among the babies.

She added that the risk is even greater than with cloning and although you could use genetic diagnosis to find disease in embryos before implantation, it wouldn't fully reduce the risk.

This all contributes to the fact that IVG is much more complicated than one might think, and experts add that the process will be even more complex in humans than in mice.

"It's a much tougher prospect to do this in a human -- much, much tougher. It's like climbing a few stairs versus climbing a mountain," Trounson said.

"Gametogenesis (in a mouse) is much faster. Everything is much faster and less complicated than you have in a human. So you've got to make sure there's very long intervals to get you the right outcome. ... Life, gametogenesis, everything, is much, much briefer than it is in a human."

Most scientists are reluctant to commit to an exact time frame, but it's probably safe to say they're many years away.

Knoepfler used the example of an unapproved and, he says, potentially dangerous three-person baby produced in Mexico in 2016 by a US doctor without FDA approval.

Creating a three-person baby involves a process known as pronuclear transfer, in which an embryo is created using genetic material from three people -- the intended mother and father and an egg donor -- to remove the risk of genetic diseases caused by DNA in a mother's mitochondria. The mitochondria are parts of a cell used to create energy but also carry DNA that is passed on only through the maternal line.

This process recently received approval in the UK, but it remains illegal in many countries, including the US.

"Because it seems rogue biomedical endeavors are on the increase, someone could try IVG without sufficient data or governmental approval in the next five to 10 years," Knoepfler said.

"IVG takes us into uncharted territory, so it's hard to say legal issues that might come up," he said, adding that "even other more extreme technologies, such as cloning, of the reproductive kind are not technically prohibited in the US."

For IVG to be researched further, it will be necessary to perform IVF using the derived gametes and then to study the embryos in ways that would involve their destruction. "At a minimum, federal funding could not be used for such work, but what other laws might come into play is less clear," Knoepler said.

In several countries, the implantation of a fertilized egg is not allowed if it's been maintained longer than 14 days.

Dr. Mahendra Rao, scientific adviser with the New York Stem Cell Foundation, explained that in the US, scientists can legally make sperm and oocytes (immature eggs) from other cells and perform IVF. But they would not be able to perform implantation, even in animals.

He said there needs to be clarity that this rule doesn't apply to "synthetic" embryos scientists are building in culture, where there's no intention of implanting them.

Daley and his co-authors highlight concerns over "embryo farming" and the consequence of parents choosing an embryo with preferred traits.

"IVG could, depending on its ultimate financial cost, greatly increase the number of embryos from which to select, thus exacerbating concerns about parents selecting for their 'ideal' future child," they write.

With a large number of eggs available through IVG, the process might exacerbate concerns about the devaluation of human life, the authors say.

Also worrying is the potential for someone to get hold of your genetic material -- such as sloughed-off skin cells -- without your permission. The authors raise questions about the legal ramifications and how they would be handled in court.

"Should the law consider the source of the skin cells to be a legal parent to the child, or should it distinguish an individual's genetic and legal parentage?" they ask.

As new forms of assisted reproductive technology stretch our ideas about identity, parentage and existing laws and regulations around stem cell research, researchers highlight the need to address these thoughts and have answers in place before making IVG an option.

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Could we one day make babies from only skin cells? - CNN

PARIS and STOCKHOLM, Feb. 9, 2017 /PRNewswire/ -- Laboratoire Fleur de Sants new Champagne Collection uses Extrait de Champagne, fueled by grape seed Phyto-StemCell's Resveratrol, for the ultimate antioxidant protection and photo-aging prevention. By reinforcing the skin's structural matrix (collagen and elastin) and stimulating its natural regeneration process, this powerful antioxidant postpones skin aging and leaves it smooth and even toned. One more reason to love Champagne!

"Antioxidant rich, Champagne extract is used in our products because it's incredibly effective at protecting and nourishing your skin. We believe that beautiful, healthy skin is worth celebrating every day," says Mathias Tonnesson, CEO of Laboratoire Fleur de Sant.

Champagne takes on a whole new meaning in skin care

The most famous sparkling wine in the world isn't just for drinking any more.

Fleur de Sant has captured its essence for the ultimate global anti-aging range of products. Extremely rich in antioxidants (Resveratrol), Champagne is one of the most beneficial ingredients protecting skin from free radicals and stress to which we are exposed every day by breathing in pollution or being unprotected from UV light.

By counteracting these factors, Champagne extract reduces the damaging marks photo-aging leaves on your skin (wrinkles, sagging skin, dark spots). It works by restoring the skin's structural tissue collagen and elastin to make it more resistant to various environmental aggressors. Antioxidants, which Champagne owes to grape seed extract, are of the highest potency, being at least 20 times more powerful than Vitamin C or E. In Fleur de Sant products, the exclusive Extrait de Champagne is further enhanced by grape seed Phyto-StemCell Infusion, which together deliver tremendously strong anti-aging force.

For more information about Fleur de Sant Champagne Collection, visit http://www.fleurdesante.com/products/

What makes phyto-stem cells so special?

Phyto-stem cells counteract the negative effect of the UV light, help maintain skin stem cell's functions and reinforce their capacity to grow, which in turn slows down the skin aging process. On top of this, they accelerate regeneration and the tissue building functions of skin, resulting in restoration of firmness and wrinkle reduction.

About Laboratoire Fleur de Sant

Fleur de Sant was founded in 1980, with the distinction of being the only brand in the world to utilize Swedish and French medicinal flowers in their beneficial formulations. The tradition continues as the brand is experiencing a re-birth with CEO Mathias Tonnesson. His passion to create skin care with "every detail considered" sees the latest clinically proven collections containing antioxidant-rich Champagne extract, plant stem cell-boosted flowers, and airless packaging that makes every formulation more effective. 95% natural and never tested on animals, Fleur de Sant is more than premium skin care it is the result of one man's passion to create products made from love.

Visit: http://www.fleurdesante.com

Contact: Mathias Tonnesson CEO, Laboratoire Fleur de Sant +1 (646) 893-4100Ext: 100 145363@email4pr.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/celebrate-your-skin-with-champagne--phyto-stemcells-300404181.html

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Celebrate Your Skin with Champagne & Phyto-StemCells - PR Newswire (press release)

Stem cells are often in the news. These days its usually about some advance in research. Sometimes the controversy about using embryonic stem cells resurfaces. Despite all the coverage (pro or con) stem cells are not well understood. What are they and why are they important?

In more ways than one, its the potential of stem cells that makes them important. At the moment most of the work with stem cells is still in the laboratory; but thats changing. Within the next few years stem cells, in one form or another, will be at work in medical applications such as repairing a damaged pancreas or a heart. In fact, stem cells will be used to repair or even re-grow tissues all over the body skin, liver, lungs, bone marrow. The production of stem cells, their delivery, and procedures for using them will become the basis of an industry. In the not too distant future stem cells, or the knowledge we gain from working with them, will be used in sophisticated repair of the brain and as part of the development of replacement organs. The potential is enormous.

What are stem cells?

Stem cells are found in most multicellular creatures and come in different varieties; all have an important ability: They can fully reproduce themselves almost indefinitely. For example, in mammals like human beings, blood stem cells (hematopoietic stem cells) are active all our lives in the marrow of bones, where they continually produce the many different kinds of blood cells. Therein is another key property for most stem cells; they can become other kinds of cells. The word for this process is differentiate; blood stem cells can differentiate into red blood cells, white blood cells, blood platelets and so forth. The ability to produce different kinds of cells is why stem cells may be used, for example, to repair or replace damaged heart cells something mature heart cells cannot do on their own.

Stem cell jargon

When you read about stem cells, there are a number of words that jump out jargon, yes, but still descriptive. Stem cells are classified by their potency, that is, what other kinds of cells they can become, or put another way, their ability to differentiate into other cells. There is a rank order from more to less potent:

Totipotent sometimes also called omnipotent stem cells can construct a complete and viable organism. In short, they are the same as a cell created by the fusion of the egg and a sperm (an embryonic cell). Totipotent cells can become any type of cell.

Pluripotent stem cells are derived from totipotent cells and are nearly as versatile. They can become any type of cell, except embryonic.

Multipotent stem cells can become a wide variety of cells, but only those of a close family, for example blood stem cells (hematopoietic cells) can become any of the blood cells, but not other kinds of cells.

Oligopotent stem cells are limited to becoming specific types of cells, such as endoderm, ectoderm, and mesoderm.

Unipotent stem cells can only produce cells of their own type, for example skin cells. They can renew themselves (replicate indefinitely), which distinguishes them from non-stem cells.

To a certain extent the potency of a stem cell relates to its usefulness. In one view of an ideal (lab) world, only totipotent stem cells would be used because they can become any other kind of cell. The real world (lab or otherwise) doesnt work that way. For one thing, stem cells of lesser versatility than totipotent cells are valuable for use in specific applications. Even unipotent stem cells, lowest on the potency poll, are arguably better suited for some targeted uses than more generic stem cells. Most importantly, for many uses, especially for medical purposes, pluripotent stem cells are extremely versatile and less controversial.

Avoiding embryonic stem cells

The true totipotent stem cell is a fertilized egg one embryonic cell. To obtain it means detecting and collecting the cell shortly after fertilization and before it begins to divide. Collecting embryonic stem cells one at a time is very difficult and very expensive. Also, in some parts of the world, using embryonic stem cells is highly controversial, usually on religious grounds. Collecting embryonic stem cells can be considered abortion, since the procedure means the cell(s) will not become an embryo. The label abortion is also applied to collecting embryonic stem cells (by gastrulation) shortly after the first fertilized cell begins to divide. These cells, obviously more numerous, are pluripotent and have been the mainstay of stem cell research.

The history of opposition to the use of embryonic stem cells goes back to the 1990s, when stem cell research was in its own infancy. At that time the only source of viable laboratory stem cells was from in vitro living donors. Most of these were harvested from fertilization clinics. They were so difficult to acquire that only a few stem cell lines (painstakingly cultivated generations of embryonic stem cells) were available. Even those were controversial. The United States banned the taking of embryonic stem cells except for 23 grandfathered lines. (This ban was lifted in 2009.)

The controversy over embryonic stem cells can be avoided primarily in two ways. One way is to use adult stem cells. The word adult is a bit misleading since the cells may be derived from fetuses, newborns, and children, which is why theyre sometimes called somatic stem cells. It means that these stem cells come from relatively mature tissue, cells that are already differentiated to a certain degree. Thats why adult stem cells are almost always classified as multipotent, oligopotent, or unipotent. The other way is to transform adult stem cells into pluripotent stem cells. Many approaches to this transformation are being explored in labs all over the world. Some approaches are derived from fetal/newborn substances such as amniotic fluid and placental or umbilical tissue. Other approaches use mature (differentiated) stem cells, such as those from skin, and genetically modify them until they become pluripotent. Such cells are called induced pluripotent stem cells, often abbreviated as iPSC.

At the moment, it is not possible to say which approaches to stem cell production and application will be the most effective. Even some that seem unlikely (stem cells from skin cells?) may turn out to be the most economical and useful. Still, this is where the payoff for stem cell research lies both in terms of scientific knowledge and in profits for medical applications. Consequently the amount of research work in progress is substantial, and often competitive.

Stem Cell Tourism

Because experimental medical techniques and human desperation can add up to big money, there is a developing market for stem cell applications for a variety of medical disorders. Unfortunately, at least for now, with the exception of blood cell transplants and skin cell treatments, most of these applications are either fraudulent or based on shaky experimental results. In general, most stem cell treatments are at best unethical and often illegal; however, their status around the world is a patchwork quilt of laws and regulations (or their absence). It is a near ideal situation for scam artists to lure desperate people into traveling long distances for stem cell treatment that is illegal in their own country. Hence the name: stem cell tourism.

Tracking the Impact of Stem Cell Research

In relative terms, stem cell research is just getting started. Researchers have been at it since the 1950s; but one of the most important discoveries so far induced pluripotent stem cells dates back to only 2006. This means that stem cells are: a. Not yet well understood and b. Their use in medicine is largely experimental and tentative. Heres a useful listing of what the National Institute of Health (U.S. NIH) considers some of the major open questions about adult stem cells:

How many kinds of adult stem cells exist, and in which tissues do they exist? How do adult stem cells evolve during development and how are they maintained in the adult? Are they leftover embryonic stem cells, or do they arise in some other way? Why do stem cells remain in an undifferentiated state when all the cells around them have differentiated? What are the characteristics of their niche that controls their behavior? Do adult stem cells have the capacity to transdifferentiate, and is it possible to control this process to improve its reliability and efficiency? If the beneficial effect of adult stem cell transplantation is a trophic effect, what are the mechanisms? Is donor cell-recipient cell contact required, secretion of factors by the donor cell, or both? What are the factors that control adult stem cell proliferation and differentiation? What are the factors that stimulate stem cells to relocate to sites of injury or damage, and how can this process be enhanced for better healing? [Source: U.S. National Institute of Health]

SciTechStory Impact Area: Stem Cells

Theres not much debate on the importance of stem cell research. It has already had major impact on our understanding of cell biology, and it will provide more. It is just beginning to have an impact on medicine, with much more to come. In fact, news about stem cell research already occurs once or twice a week (on average) that pace is likely to increase. As a matter of keeping up, its necessary to attempt sorting lab work from practical application, which is to say sorting promise from delivery. Even at that it will be difficult to select which stem cell stories are significant.

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Stem Cells - SciTechStory

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Small sheets of healthy skin are being grown from scratch at a Stanford University lab, proof that gene therapy can help heal a rare disease that causes great human suffering.

The precious skin represents growing hope for patients who suffer from the incurable blistering disease epidermolysis bullosa and acceleration of the once-beleaguered field of gene therapy, which strives to cure disease by inserting missing genes into sick cells.

It is pink and healthy. Its tougher. It doesnt blister, said patient and research volunteer Monique Roeder, 33, of Cedar City, Utah, who has received grafts of corrected skin cells, each about the size of an iPhone 5, to cover wounds on her arms.

More than 10,000 human diseases are caused by a single gene defect, and epidermolysis bullosa is among the most devastating. Patients lack a critical protein that binds the layers of skin together. Without this protein, the skin tears apart, causing severe pain, infection, disfigurement and in many cases, early death from an aggressive form of skin cancer.

The corrected skin is part of a pipeline of potential gene therapies at Stanfords new Center for Definitive and Curative Medicine, announced last week.

The center, a new joint initiative of Stanford Healthcare, Stanford Childrens Health, and the Stanford School of Medicine, is designed to accelerate cellular therapies at the universitys state-of-the-art manufacturing facility on Palo Altos California Avenue. Simultaneously, itisaiming to bring cures to patients faster than before and boost the financial value of Stanfords discoveries before theyre licensed out to biotech companies.

With trials such as these, we are entering a new era in medicine, said Dr. Lloyd B. Minor, dean of the Stanford University School of Medicine.

Gene therapy was dealt a major setback in 1999 when Jesse Gelsinger, an Arizona teenager with a genetic liver disease, had a fatal reaction to the virus that scientists had used to insert a corrective gene.

But current trials are safer, more precise and build on better basic understanding. Stanford is also using gene therapy to target other diseases, such as sickle cell anemia and beta thalassemia,a blood disorder that reduces the production of hemoglobin.

There are several diseases that are miserable and worthy of gene therapy approaches, said associate professor of dermatology Dr. Jean Tang, who co-led the trial with Dr. Peter Marinkovich. But epidermolysis bullosa, she said, is one of the worst of the worst.

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It took nearly 20 years for Stanford researchers to bring this gene therapy to Roeder and her fellow patients.

It is very satisfying to be able to finally give patients something that can help them, said Marinkovich.In some cases, wounds that had not healed for five years were successfully healed with the gene therapy.

Before, he noted, there was only limited amounts of what you can do for them. We can treat their wounds and give them sophisticated Band-Aids. But after you give them all that stuff, you still see the skin falling apart, Marinkovich said. This makes you feel like youre making a difference in the world.

Roeder seemed healthy at birth. But when her family celebrated her arrival by imprinting her tiny feet on a keepsake birth certificate, she blistered. They encouraged her to lead a normal childhood, riding bicycles and gentle horses. Shes happily married. But shes grown cautious, focusing on photography, writing a blog and enjoying her pets.

Scarring has caused her hands and feet digits to become mittened or webbed. Due to pain and risk of injury, she uses a wheelchair rather than walking long distances.

Every movement has to be planned out in my head so I dont upset my skin somehow, she said. Wound care can take three to six hours a day.

She heard about the Stanford research shortly after losing her best friend, who also had epidermolysis bullosa, to skin cancer, a common consequence of the disease. Roeder thought: Why dont you try? She didnt get the chance.

The team of Stanford experts harvested a small sample of skin cells, about the size of a pencil eraser, from her back. They put her cells in warm broth in a petri dish, where they thrived.

To this broth they added a special virus, carrying the missing gene. Once infected, the cells began producing normal collagen.

They coaxed these genetically corrected cells to form sheets of skin. The sheets were then surgically grafted onto a patients chronic or new wounds in six locations. The team reported their initial results in Novembers Journal of the American Medical Association.

Historically, medical treatment has had limited options: excising a sick organ or giving medicine, said Dr. Anthony E. Oro of Stanfords Institute for Stem Cell Biology and Regenerative Medicine. When those two arent possible, theres only symptom relief.

But the deciphering of the human genome, and new tools in gene repair, have changed the therapeutic landscape.

Now that we know the genetic basis of disease, we can use the confluence of stem cell biology, genome editing and tissue engineering to develop therapies, Oro said.

Its not practical to wrap the entire body of a patient with epidermolysis bullosa in vast sheets of new skin, like a mummy, Oro said.

But now that the team has proved that gene therapy works, they can try related approaches, such as using gene-editing tools directly on the patients skin, or applying corrected cells like a spray-on tan.

A cure doesnt take one step, said Tang. It takes many steps towards disease modification, and this is the first big one. Were always looking for something better.

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Stanford team is growing healthy skin for diseased patients - The Mercury News

Human skin can be morphed into genetically modified, cancer-killing brain stem cells, according to a new study. This latest advance has only been tested in mice but eventually, its possible that it could be translated into a personalized treatment for people with a deadly form of brain cancer.

The study builds on an earlier discovery that brain stem cells have a weird affinity for cancers. So researchers, led by Shawn Hingtgen, a professor at University of North Carolina at Chapel Hill, created genetically engineered brain stem cells out of human skin. Then they armed the stem cells with drugs to squirt directly onto the tumors of mice that had been given a human form of brain cancer. The treatment shrank the tumors and extended survival of the mice, according to results recently published in the journal Science Translational Medicine.

The treatment shrank the tumors and extended survival

Usually we think about stem cell therapy in the context of rebuilding or regrowing a broken body part like a spinal cord. But if they could be modified to become cancer-fighting homing missiles, it would give patients with a deadly and incurable brain cancer called glioblastoma a better chance at survival. Glioblastomas typically affect adults, and are highly fatal because they send out a web of cancerous threads. Even when the main mass is removed, those threads remain despite chemotherapy and radiation treatment. This cancer has caused a number of high-profile deaths including Senator Edward (Ted) Kennedy in 2009, and possibly Beau Biden more recently. Approximately 12,000 new cases of glioblastoma are estimated to be diagnosed in 2017.

We really have no drugs, no new treatment options in years to even decades, Hingtgen says. [We] just really want to create new therapy that can stand a chance against this disease.

But theres a problem: brain stem cells arent exactly easy to get. Brain stem cells, more properly known as neural stem cells, hang out in the walls of the brains irrigation canals areas filled with cerebrospinal fluid, called ventricles. They generate the cells of the nervous system, like neurons and glial cells, throughout our lives.

They could be modified to become cancer-fighting homing missiles

A research group at the City of Hope in California conducted a clinical trial to make sure it was safe to treat glioblastoma patients with genetically engineered neural stem cells. But they used a neural stem cell line that theyd obtained from fetal tissue. Since the stem cells werent the patients own, people who were genetically more likely to reject the cells couldnt receive the treatment at all. For the people who could, treatment with the neural stem cells turned out to be relatively safe although at this phase of clinical trials, it hasnt been particularly effective.

More personalized treatments have been held up by the challenge of getting enough stem cells out of the patients own brains, which is virtually impossible, says stem cell scientist Frank Marini at the Wake Forest School of Medicine, who was not involved in this study. You cant really generate a bank of neural stem cells from anybody because you have to go in and resect the brain.

So instead, Hingtgen and his colleagues figured out a way to generate neural stem cells from skin which in the future, could let them make neural stem cells personalized to each patient. For this study, though, Hingtgen and his colleagues extracted the skin cells from chunks of human flesh leftover as surgical waste. That really is the magic piece here, Marini says. Now, all of a sudden we have a neural stem cell that can be used as a tumor-homing vehicle.

That really is the magic piece here.

Using a disarmed virus to infect the cells with a cocktail of new genes, the researchers morphed the skin cells into something in between a skin cell and a neural stem cell. People have turned skin cells back into a more generalized type of stem cell before. But then turning those basic stem cells into stem cells for a certain organ like the brain takes another couple of steps, which takes more time. Thats something that people with glioblastoma dont have.

The breakthrough here is that Hingtgens team figured out how to go straight from skin cells to something resembling a neural stem cell in just four days. The researchers then genetically engineered these induced neural stem cells to arm them with one of two different weapons: One group was equipped with an enzyme that could convert an anti-fungal drug into chemotherapy, right at the cancers location. The other was armed with a protein that binds to the cancer cells and makes them commit suicide in an orderly process called apoptosis.

The researchers tested these engineered neural stem cells in mice that had been injected with human glioblastoma cells, which multiplied out of control to create a human cancer in a mouse body. Both of the weaponized stem cell groups were able to significantly shrink the tumors and keep the mice alive by about an extra 30 days (for scale, mice usually live an average of two years).

Were working as fast as we can.

But injecting the cells directly into the tumor doesnt really reflect how the therapy would be used in humans. Its more likely that a person with glioblastoma would get the bulk of the tumor surgically removed. Then, the idea is that these neural stem cells, generated from the patients own skin, will be inserted into the hole left in the brain. So, the researchers tried this out in mice, and the tumors that regrew after surgery were more than three times smaller in the mice treated with the neural stem cells.

Its a promising start, but it could take a few years still before its in the clinic, Hingtgen says. He and his colleagues started a company called Falcon Therapeutics to drive this new therapy forward. Were working as fast as we can, Hingtgen says. We probably cant help the patients today. Hopefully in a year or two, well be able to help those patients.

One of the things theyll have to figure out first is whether the neural stem cells can travel the much bigger distances in human brains, and whether theyll be able to eliminate every remaining cancer cell. The caveats on this are that, of course, its a mouse study, and whether or not that directly converts to humans is unclear, Marini says. Still, he adds, Theres a very high probability in this case.

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The next weapon against brain cancer may be human skin - The Verge

Glioblastoma is an aggressive form of brain cancer that kills most patients within two years of diagnosis. In tests on mice last year, a team at the University of North Carolina at Chapel Hill showed that adult skin cells could be transformed into stem cells and used to hunt down the tumors. Building on that, they've now found that the process works with human cells, and can be administered quickly enough to beat the ticking time-bombs.

Treatments for glioblastoma include the usual options of surgery, radiation therapy and chemotherapy, but none of them are particularly effective. The tumors are capable of spreading tendrils out into the brain and it can grow back in a matter of months after being removed. As a result, the median survival rate of sufferers is under 18 months, and there's only a 30 percent chance of living more than two years.

"We desperately need something better," says Shawn Hingtgen, the lead researcher on the study.

To find that something better, last year the scientists took fibroblasts a type of skin cell that generates collagen and connective tissue from mice and reprogrammed them into neural stem cells. These stem cells seek out and latch onto cancer cells in the brain, but alone are powerless to fight the tumor. To give them that ability, the scientists engineered them to express a particular cancer-killing protein. The result was mice that lived between 160 and 220 percent longer.

The next step was to test the process with human cells, and in the year since, the team has found that the results are just as promising. The technique differs slightly when scaled up to humans. The patient would be administered with a substance called a prodrug, which by itself does nothing, until it's triggered. The stem cells are engineered to carry a protein that acts as that trigger, activating the prodrug only in a small halo around itself instead of affecting the entire body. That allows the drug to target only a small desired area, ideally reducing the ill side effects that treatments like chemotherapy can induce.

Importantly, the technique can be administered quickly, to give the patients the best chance at survival.

"Speed is essential," says Hingtgen. "It used to take weeks to convert human skin cells to stem cells. But brain cancer patients don't have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient."

The treatment is an important step, but there's still a long way to go.

"We're one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers," says Hingtgen. "This is a big step toward a real treatment and making a real difference."

The research was published in the journal Science Translational Medicine.

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Stem cells beat the clock for brain cancer - New Atlas

Brain cancers can be really tricky to treat. Some, such as glioblastomas, spread roots through the brain tissue, meaning they are often impossible to remove surgically, leading to tragically low survival rates. But researchers are working on a way touse stem cells to track down the cancer, kill it, and then melt it away. By doing this, theyve managed to shrink brain tumors in mice to2 to 5 percent of their original size.

The trick has already been tried before using neural stem cells to hunt down and deliver cancer-killing drugs to tumors in mice. But there is a problem: It's tricky to getneural stem cells from humans. The safest way of doing this would be to take adult cells and then induce them in a two-step process to become neural stem cells. This, however, takes time.

Speed is essential, saysShawn Hingtgen, who led the research published in Science Translational Medicine. It used to take weeks to convert human skin cells to stem cells. But brain cancer patients dont have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient.

The researchers found a way to speed the process up byremoving one of the steps entirely, allowing them to produce the neural stem cells from adult skin cells in just four days. Usually, researchers would need to take the skin cell, induce it to become a generic stem cell, and then push it towards becoming a neural stem cell.

But by treating the skin cells with a cocktail of biochemicals, they were able to get the cells to turn straight into neural stem cells. They then tested these to see if they still had the same properties as original neutral stem cells and home in on tumors both in a petri dish and in animals models. They found they behaved exactly the same.

The final step was to see if they could somehow engineer these newly created cells to deliver drugs that are targeted at the cancer. They therefore got the stem cells to carry a particular protein that activates what is called a prodrug, which the researchers describe as forming a halo of drugs around the stem cell.

Were one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers, says Hingtgen. This is a big step toward a real treatment and making a real difference.

Excerpt from:
Scientists Reprogram Skin Cells To Hunt Down And Shrink Brain Tumors - IFLScience

Mouse and human skin cells can be reprogrammed to hunt down tumors and deliver anticancer therapies.

Imagine cells that can move through your brain, hunting down cancer and destroying it before they themselves disappear without a trace. Scientists have just achieved that in mice, creating personalized tumor-homing cells from adult skin cells that can shrink brain tumors to 2% to 5% of their original size. Althoughthe strategy has yet to be fully tested in people, the new method could one day give doctors a quick way to develop a custom treatment for aggressive cancers like glioblastoma, which kills most human patients in 1215 months. It only took 4 days to create the tumor-homing cells for the mice.

Glioblastomas are nasty: They spread roots and tendrils of cancerous cells through the brain, making them impossible to remove surgically. They, and other cancers, also exude a chemical signal that attracts stem cellsspecialized cells that can produce multiple cell types in the body. Scientists think stem cells might detect tumors as a wound that needs healing and migrate to help fix the damage. But that gives scientists a secret weaponif they can harness stem cells natural ability to home toward tumor cells, the stem cells could be manipulated to deliver cancer-killing drugs precisely where they are needed.

Other research has already exploited this methodusing neural stem cellswhich give rise to neurons and other brain cellsto hunt down brain cancer in mice and deliver tumor-eradicating drugs. But few have tried this in people, in part because getting those neural stem cells is hard, says Shawn Hingtgen, a stem cell biologist at the University of North Carolina inChapel Hill. Right now, there are three main ways. Scientists can either harvest the cells directly from the patient, harvest them from another patient, or they can genetically reprogram adult cells. But harvesting requires invasive surgery, and bestowing stem cell properties on adult cells takes a two-step process that can increase the risk of the final cells becoming cancerous. And using cells from someone other than the cancer patient being treated might trigger an immune response against the foreign cells.

To solve these problems, Hingtgens group wanted to see whetherthey could skip a step in the genetic reprogramming process, which first transforms adult skin cells into standard stem cells and then turns those into neural stem cells. Treating the skin cells with a biochemical cocktail to promote neural stem cell characteristics seemed to do the trick, turning it into a one-step process, he and his colleague report today in Science Translational Medicine.

But the next big question was whether these cells could home in on tumors in lab dishes, and in animals, like neural stem cells. We were really holding our breath, Hingtgen says. The day we saw the cells crawling across the [Petri] dish toward the tumors, we knew we had something special. The tumor-homing cells moved 500 micronsthe same width as five human hairsin 22 hours, and they could burrow into lab-grown glioblastomas. This is a great start, says Frank Marini, a cancer biologist at the Wake Forest Institute forRegenerative Medicine in Winston-Salem, North Carolina,who was not involved with the study. Incredibly quick and relatively efficient.

The team also engineered the cells to deliver common cancer treatments to glioblastomas in mice. Mouse tumors injected directly with the reprogrammed stem cells shrank 20- to 50-fold in 2428 days compared withnontreated mice. In addition, the survival times of treated rodents nearly doubled. In some mice, the scientists removed tumors after they were established, and injected treatment cells into the cavity. Residual tumors, spawned from the remaining cancer cells, were 3.5 times smaller in the treated mice than in untreated mice.

Marini notes that more rigorous testing is needed to demonstrate just how far the tumor-targeting cells can migrate. In a human brain, the cells would need to travel a matter of millimeters or centimeters, up to 20 times farther than the 500 microns tested here, he says. And other researchers question the need to use cells from the patients own skin. An immune response, triggered by foreign neural stem cells, could actually help attack tumors, says Evan Snyder, a stem cell biologist at Sanford Burnham Prebys Medical Discovery Institute in San Diego, California, and one of the early pioneers of the idea of using stem cells to attack tumors.

Hingtgens group is already testing how far their tumor-homing cells can migrate using larger animal models. They are also getting skin cells from glioblastoma patients to make sure the new method works for the people they hope to help, he says. Everything were doing is to get this to the patient as quickly as we can.

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Reprogrammed skin cells shrink brain tumors in mice | Science | AAAS - Science Magazine

Stem cells have tremendous promise to help us understand and treat a range of diseases, injuries and other health-related conditions. Their potential is evident in the use of blood stem cells to treat diseases of the blood, a therapy that has saved the lives of thousands of children with leukemia; and can be seen in the use of stem cells for tissue grafts to treat diseases or injury to the bone, skin and surface of the eye. Important clinical trials involving stem cells are underway for many other conditions and researchers continue to explore new avenues using stem cells in medicine.

There is still a lot to learn about stem cells, however, and their current applications as treatments are sometimes exaggerated by the media and other parties who do not fully understand the science and current limitations, and also by clinics looking to capitalize on the hype by selling treatments to chronically ill or seriously injured patients. The information on this page is intended to help you understand both the potential and the limitations of stem cells at this point in time, and to help you spot some of the misinformation that is widely circulated by clinics offering unproven treatments.

It is important to discuss these Nine Things to Know and any research or information you gather with your primary care physician and other trusted members of your healthcare team in deciding what is right for you.

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Nine Things to Know About Stem Cell Treatments

One of the current challenges for stem cell researchers is to understand how all the skin appendages are regenerated. This could lead to improved treatments for burn patients, or others with severe skin damage.

Researchers are also working to identify new ways to grow skin cells in the lab. Epidermal stem cells are currently cultivated on a layer of cells from rodents, called murine cells. These cell culture conditions have been proved safe, but it would be preferable to avoid using animal products when cultivating cells that will be transplanted into patients. So, researchers are searching for effective cell culture conditions that will not require the use of murine cells.

Scientists are also working to treat genetic diseases affecting the skin. Since skin stem cells can be cultivated in laboratories, researchers can genetically modify the cells, for example by inserting a missing gene. The correctly modified cells can be selected, grown and multiplied in the lab, then transplanted back onto the patient. Epidermolysis Bullosa is one example of a genetic skin disease that might benefit from this approach. Work is underway to test the technique.

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Skin stem cells: where do they live and what can they do ...