Theoretically, eternal youth is now within our grasp.

Doctors are close to discovering a real life fountain of youth that could theoretically enable patients to live forever. Advances in stem cell treatments and now, tissue nanotransfection (TNT), which is a new technique, can theoretically provide patients with the benefits of youth for life.

The fountain of youth is within the grasp of science, but so far, only for mice. Human trials come next year.

LOS ANGELES, CA (California Network) -- The quest for eternal life is ancient. It is mentioned in the first and oldest story we have, the Epic of Gilgamesh. In that ancient Sumerian tale, only Utnapishtim, a man who built and ark and survived a great flood in a story that is almost identical to the story of Noah's ark, knows the secret to eternal life, which ultimately proves elusive. In the centuries that followed, people have tried every remedy imaginable to prolong life. They searched for the fabled fountain of youth, and according to some legends, bathed in the blood of virgins and children.

Today, we know none of these endeavors would work because ageing is carried on in the genes. The only way to reverse ageing is to manipulate the genes. And this is precisely what doctors are looking to do in order to produce new cells, and even whole organs.

Researchers now know the primary difference between a young person and an old person is the number of stem cells in their body. Young people have many times more stem cells. This is the basic, underlying reason why young people are so youthful. A young body can repair itself more rapidly and thoroughly than an older one because of the number of stem cells. But if stem cells could be injected into an older body, in quantities similar to those enjoyed by a young person, what would happen then?

Nobody knows for certain because the experiment hasn't been conducted, but the hypothesis is that the older person would become more youthful, healthier, and longer lived.

As stem cells enter the medical mainstream, and may become a standard part of medical treatment in the near future, there is another development that could make stem cells irrelevant. Nanotransfection, abbreviated as TNT, is a new method whereby skin cells can be turned into any other cell in the body using a special microchip and electricity.

The device, called a nanochip, is loaded with genetic material essential to turning cells into other kinds of cells. The electrical current enables the device to inject the genetic material into the skin where it ends up inside the cells. These cells can then travel though the body and take on the properties of healthy cells around damaged tissue, facilitating repair. On other words, a damaged liver or heart can be repaired with this tiny device. The advantage of this method is that stem cells are not required. Your skin cells simply become whether other kind of cells they are told to become by the injected genetic material.

A study affirming the effectiveness of this approach was published in the journal, Nature Nanotechnology. It has been tested on mice and was successful in restoring function to non-functioning limbs. It will be tested on humans within the next year.

Scientists have known they can reprogram cells into other kinds of cells for a long time now, but only recently have they developed the method to do so cheaply and efficiently. The actual procedure requires a chip that is as small as a penny, and takes only a second to work.

If the procedure works on humans, then doctors may have a cheap and efficient way to repair and even replace organs. The discovery is so dramatic is it difficult to believe. More testing is required, but it shows just how far we have come in our ability to edit genes and reprogram cells to grow specific forms of tissue within the body.

In a generation or less, it is reasonable that we will have unlocked the secret to reversing ageing. Of course, this discovery opens a whole host of ethical and philosophical questions, but that's for the ethicists and politicians to work out. For now, science is about to take the first sip from the fountain of youth, and we await the result.

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Pope Francis Prayer Intentions for JULY 2017Lapsed Christians. That our brothers and sisters who have strayed from the faith, through our prayer and witness to the Gospel, may rediscover the merciful closeness of the Lord and the beauty of the Christian life.

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Stem cells: science prepares to take the first sip from the real fountain of youth - Catholic Online

The PASE, or post-implantation amniotic sac embryoid, is a structure grown from human pluripotent stem cells that mimics many of the properties of the amniotic sac that forms soon after an embryo implants in the uterus wall. The structures could be used to study infertility. Credit: University of Michigan

The first few weeks after sperm meets egg still hold many mysteries. Among them: what causes the process to fail, leading to many cases of infertility.

Despite the importance of this critical stage, scientists haven't had a good way to explore what can go wrong, or even what must go right, after the newly formed ball of cells implants in the wall of the human uterus.

But a new achievement using human stem cells may help change that. Tiny lab-grown structures could give researchers a chance to see what they couldn't before, while avoiding ethical issues associated with studying actual embryos.

A team from the University of Michigan reports in Nature Communications that they have coaxed pluripotent human stem cells to grow on a specially engineered surface into structures that resemble an early aspect of human development called the amniotic sac.

The cells spontaneously developed some of the same structural and molecular features seen in a natural amniotic sac, which is an asymmetric, hollow ball-like structure containing cells that will give rise to a part of the placenta as well as the embryo itself. But the structures grown at U-M lack other key components of the early embryo, so they can't develop into a fetus.

It's the first time a team has grown such a structure starting with stem cells, rather than coaxing a donated embryo to grow, as a few other teams have done.

"As many as half of all pregnancies end in the first two weeks after fertilization, often before the woman is even aware she is pregnant. For some couples, there is a chronic inability to get past these critical early developmental steps, but we have not previously had a model that would allow us to explore the reasons why," says co-senior author Deborah Gumucio, Ph.D. "We hope this work will make it possible for many scientists to dig deeper into the pathways involved in normal and abnormal development, so we can understand some of the most fascinating biology on earth." Gumucio is the Engel Collegiate Professor of Cell & Developmental Biology at Michigan Medicine, U-M's academic medical center.

A steady PASE

The researchers have dubbed the new structure a post-implantation amniotic sac embryoid, or PASE. They describe how a PASE develops as a hollow spherical structure with two distinct halves that remain stable even as cells divide.

One half is made of cells that will become amniotic ectoderm, the other half consists of pluripotent epiblast cells that in nature make up the embryonic disc. The hollow center resembles the amniotic cavity - which in normal development eventually gives rise to the fluid-filled sac that protects and cushions the fetus during development.

Gumucio likens a PASE to a mismatched plastic Easter egg or a blue-and-red Pokmon ball - with two clearly divided halves of two kinds of cells that maintain a stable form around a hollow center.

The team also reports details about the genes that became activated during the development of a PASE, and the signals that the cells in a PASE send to one another and to neighboring tissues. They show that a stable two-halved PASE structure relies on a signaling pathway called BMP-SMAD that's known to be critical to embryo development.

Gumucio notes that the PASE structures even exhibit the earliest signs of initiating a "primitive streak", although it did not fully develop. In a human embryo, the streak would start a process called gastrulation. That's the division of new cells into three cell layersendoderm, mesoderm and ectodermthat are essential to give rise to all organs and tissues in the body.

Collaboration provides the spark

The new study follows directly from previous collaborative work between Gumucio's lab and that of the other senior author, U-M mechanical engineering associate professor Jianping Fu, Ph.D.

In the previous work, reported in Nature Materials, the team succeeded in getting balls of stem cells to implant in a special surface engineered in Fu's lab to resemble a simplified uterine wall. They showed that once the cells attached themselves to this substrate, they began to differentiate into hollow cysts composed entirely of amnion - a tough extraembryonic tissue that holds the amniotic fluid.

But further analysis of these cysts by co-first authors of the new paper Yue Shao, Ph.D., a graduate student in Fu's lab, and Ken Taniguchi, a postdoctoral fellow in Gumucio's lab, revealed that a small subset of these cysts were stably asymmetric and looked exactly like early human or monkey amniotic sacs.

The team found that such structures could also grow from induced pluripotent stem cells (iPSCs)cells derived from human skin and grown in the lab under conditions that give them the ability to become any type of cell, similar to how embryonic stem cells behave. This opens the door for future work using skin cells donated by couples experiencing chronic infertility, which could be grown into iPSCs and tested for their ability to form proper amniotic sacs using the methods devised by the team.

Important notes and next steps

Besides working with genetic and infertility specialists to delve deeper into PASE biology as it relates to human infertility, the team is hoping to explore additional characteristics of amnion tissue.

For example, early rupture of the amnion tissue can endanger a fetus or be the cause of a miscarriage. The team also intends to study which aspects of human amnion formation also occur in development of mouse amnion. The mouse embryo model is very attractive as an in vivo model for investigating human genetic diseases.

The team's work is overseen by a panel that monitors all work done with pluripotent stem cells at U-M, and the studies are performed in accordance with laws regarding human stem cell research. The team ends experiments before the balls of cells effectively reach 14 developmental days, the cutoff used as an international limit on embryo researcheven though the work involves tissue that cannot form an embryo. Some of the stem cell lines were derived at U-M's privately funded MStem Cell Laboratory for human embryonic stem cells, and the U-M Pluripotent Stem Cell Core.

Explore further: Team uses stem cells to study earliest stages of amniotic sac formation

More information: Yue Shao et al, A pluripotent stem cell-based model for post-implantation human amniotic sac development, Nature Communications (2017). DOI: 10.1038/s41467-017-00236-w

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Amniotic sac in a dish: Stem cells form structures that may aid of ... - Phys.Org

For patients with severe and end-stage heart failure there are few treatment options left apart from transplants and stem-cell therapy. But a new Israeli study finds that stem-cell therapy may harm heart-disease patients.

The research, led by Prof. Jonathan Leor of Tel Aviv Universitys Sackler Faculty of Medicineand Sheba Medical Center and conducted by TAUs Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissue and contends that this can prove toxic for patients.

We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury, said Leor. Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle.

Tissue or adult stem cells blank cells that can act as a repair kit for the body by replacing damaged tissue encourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, many heart-failure patients have turned to stem-cell therapy as a last resort.

But our findings suggest that stem cells, like any drug, can have adverse effects, said Leor. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient.

The researchers, who published their study in the journal Circulation, also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. Afterward, they focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cells those drawn from the patients themselves in cardiac therapy, Leor said.

We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells, said Leor. Our findings determine the potential negative effects of inflammation on stem-cell function as theyre currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions.

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. We hope our engineered stem cells will be resistant to the negative effects of the immune system, said Leor.

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Stem-cell treatment may harm heart disease patients - ISRAEL21c

Earlier this year, Texas Heart Institute received Alpha Phi Foundations 2017 Heart to Heart Grant. The $100,000 grant will fund research led by Doris Taylor, Ph.D., director of the Regenerative Medicine Research and the Center for Cell and Organ Biotechnology at the Texas Heart Institute, to study cardiac repair in women at the cellular level.

Were just really passionate about these projects that have long-term clinical relevancy, as a women-driven organization and being committed to womens heart health, said Colleen Sirhal, vice chair of the Alpha Phi Foundation.

The study will explore sex differences in blood, bone marrow and stem cells of patients enrolled in cell therapy clinical trials.

While bone marrow cell therapy has been used to treat cardiovascular disease in clinical trials, very few studies have been conducted to assess the sex differences in efficacy and outcomes. By performing a proteomic analysis of the samples and evaluating the proteins that cells produce and secrete, the results could shed light on unanswered questions related to critical sex-specific differences in cardiovascular disease, potentially leading to improved cell therapies.

Its about time that were paying attention to sex differences, Taylor said. Were not just small men. The biology is different.

Heart disease remains the No. 1 cause of death in both men and women in the United States, yet theres a limited understanding in the scientific community as to why it affects men and women differently. For example, women 45 years old and younger have a higher likelihood than men of dying within a year of their initial heart attack.

In addition, women have a higher risk of developing small vessel disease, in which the walls of tiny vessels within the heart muscle become blocked rather than larger arteries, causing heart-related chest pain. Because the major coronary arteries may look normal, women with small vessel disease can have a heart attack go undiagnosed and untreated.

We know heart disease happens differently in men and women, Taylor said. More young women than men die of heart disease. Why is that? Is there something that happens early? If we only look at these women who are older, are we missing something major? By looking at healthy, normal younger women, were going to be able to do comparisons across time, comparisons by disease, and comparisons by sex. I think thats really exciting.

Historically, women and minorities have largely been underrepresented in research and clinical trials, especially pertaining to cardiovascular disease.

Dr. Taylors colleague at the Texas Heart Institute, Stephanie Coulter, M.D., a cardiologist and the director of the Center for Womens Heart and Vascular Health at Texas Heart Institute and a recipient of the 2013 Heart to Heart Grant, is actively recruiting younger women to participate in her research registry.

Since women are typically affected by heart disease a decade or more later than men, age may also have played a role in this underrepresentation, Coulter said. Our Womens Center research is focusing on women age 18 and older to address this very issue.

Coulter added that trials focusing on prevention in women, such as the Womens Health Initiative and Womens Health Study, have, in fact, had clinical impact. However, the percentage of women enrolling in clinical trials continues to be disproportionate to the prevalence of cardiovascular disease in women, but we are seeing improvements thanks to multiple initiatives in the U.S. that continue to address the issue of women in clinical trials.

Its easy for people to assume that if you study men, itll apply to women, but it seems anathema to people to assume that if you study women it might benefit men, Taylor said. At the end of the day, when it comes time to look at the data and ask, How does this treatment work in women? How does this treatment work in men?, oftentimes there arent enough women enrolled in the trials to split that out. Statistically, youd be doing yourself a disservice.

Taylor has spent nearly two decades studying key contributors to cardiac repair at the cellular level, specifically looking at proteins cells produce and secrete based on gender as a new frontier in cell therapy.

Early on in Taylors career, she studied how bone marrow cells behaved based on gender. She extracted cells from male mice and administered them to female mice and vice versa, allowing her to track the Y chromosome. The results showed that only the males treated with female cells improved. This phenomenon raised the question of whether or not the bone marrow cells were the same.

After measuring the bone marrow cells that were present in males and females, Taylor discovered that the cells were inherently different: In the male mice, there were more inflammatory cells, fewer progenitor and stem cells and a different number of immune cells than in the female mice. In addition, when the bone marrow cells were placed in a petri dish, the female cells produced more growth factors responsible for recruiting repair cells after an injury.

Taylor conducted follow-up experiments in which she gave female and male cells to both female and male mice. The results confirmed her hunch: The only cells that were reparative were the female cells.

It made me realize a critical detail for the first time:Every time we take bone marrow from a different person with the intention of delivering it back to them as a therapy, if we look at the cells present in the marrow, theyd be different, Taylor said. Which means, every time were doing an autologous cell therapytrial, in which you take bone marrow and deliver it back to an individual, you are giving each person a completely different or unique drug in that trial.

Through the Heart to Heart grant, the data from Taylors research will allow her to build upon her early research on sex differences and, hopefully, identify a way to optimize cell therapy.

Already cells are as good as some drugs. If we optimize them and choose the right cells for the right patient at the right time, maybe well hit the home run, Taylor said.

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Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair - Texas Medical Center (press release)

Daiichi Sankyo Company has signed an investment contract with Cuorips Inc., an Osaka University spin-off venture to receive an option right concerning the worldwide commercialization of iPS-derived cardiomyocyte (iPS-CM) sheet developed by Cuorips.

The iPS-CM sheet is an allogeneic cell therapy product consisting of cardiomyocyte derived from human iPS cells. Its transplantation is expected to provide improvement of cardiac function and amelioration of heart failure and become a new treatment option for patients with severe heart failure, who have no remedies other than heart transplantation or artificial heart implantation.

Based on the cutting-edge cell therapy research targeting heart diseases, the team at the Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, led by Professor Yoshiki Sawa, has been working on the iPS-CM research and development by participating in the Research Center Network for Realization of Regenerative Medicine, which is run by the Japan Agency for Medical Research and Development (AMED). They are currently preparing for clinical research as well as investigator initiated clinical study.

Cuorips is an Osaka University spin-off venture founded to develop and commercialize iPS-CM sheets based on the research data and technologies developed by the university.

Daiichi Sankyo Group has been conducting research on iPS cell-derived cardiomyocyte and their production, and is currently working on the efficient production process capable for commercial supply.

Daiichi Sankyo and Cuorips are aiming to commercialize iPS-CM sheets as a pioneering treatment for severe heart failure. iPS cells are capable of almost unlimited proliferation and differentiation into any organ, and are expected to be used in the field of cell therapy. There are two types of cell therapy: autologous therapy where the patients own cells are collected, cultured and processed, and allogeneic therapy where a donors cells are collected, cultured and processed.

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Daiichi Sankyo signs Investment contract with Cuorips to commercialize iPS-derived cardiomyocyte sheet - pharmabiz.com

A novel device that reprogrammes skin cells could represent a breakthrough in repairing injured or ageing tissue, researchers say.

The new technique, called tissue nanotransfection, is based on a tiny device that sits on the surface of the skin of a living body. An intense, focused electric field is then applied across the device, allowing it to deliver genes to the skin cells beneath it turning them into different types of cells.

That, according to the researchers, offers an exciting development when it comes to repairing damaged tissue, offering the possibility of turning a patients own tissue into a bioreactor to produce cells to either repair nearby tissues, or for use at another site.

By using our novel nanochip technology, injured or compromised organs can be replaced, said Chandan Sen, from the Ohio State University, who co-led the study. We have shown that skin is a fertile land where we can grow the elements of any organ that is declining.

The ability for scientists to reprogram cells into other cell types is not new: the discovery scooped John Gurdon and Shinya Yamanaka the Nobel Prize in 2012 and is currently under research in myriad fields, including Parkinsons disease.

You can change the fate of cells by incorporating into them some new genes, said Dr Axel Behrens, an expert in stem cell research from the Francis Crick Institute in London, who was not involved in the Ohio research. Basically you can take a skin cell and put some genes into them, and they become another cell, for example a neuron, or a vascular cell, or a stem cell.

But the new approach, says Sen, avoids an intermediary step where cells are turned into what are known as pluripotent stem cells, instead turning skin cells directly into functional cells of different types. It is a single step process in the body, he said.

Furthermore, the new approach does not rely on applying an electric field across a large area of the cell, or the use of viruses to deliver the genes. We are the first to be able to reprogramme [cells] in the body without the use of any viral vector, said Sen.

The new research, published in the journal Nature Nanotechnology, describes how the team developed both the new technique and novel genes, allowing them to reprogramme skin cells on the surface of an animal in situ.

They can put this little device on one piece of skin or onto the other piece of skin and the genes will go there, wherever they put [the device], said Behrens.

The team reveal that they used the technique on mice with legs that had had their arteries cut, preventing blood flow through the limb. The device was then put on the skin of the mice, and an electric field applied to trigger changes in the cells membrane, allowing the genes to enter the cells below. As a result, the team found that they were able to convert skin cells directly into vascular cells -with the effect extending deeper into the limb, in effect building a new network of blood vessels.

Seven days later we saw new vessels and 14 days later we saw [blood flow] through the whole leg, said Sen.

The team were also able to use the device to convert skin cells on mice, into nerve cells which were then injected into the brains of mice who had experienced a stroke, helping them to recover.

With this technology, we can convert skin cells into elements of any organ with just one touch. This process only takes less than a second and is non-invasive, and then youre off, said Sen.

The new technology, said Behrens is an interesting step, not least since it avoids all issues with rejection.

This is a clever use of an existing technique that has potential applications but massive further refinement is needed, he said, pointing out that there are standard surgical techniques to deal with blockages of blood flow in limbs.

Whats more, he said, the new technique is unlikely to be used on areas other than skin, since the need for an electric current and the device near to the tissue means using it on internal organs would require an invasive procedure.

Massive development [would be] needed for this to be used for anything else than skin, he said.

But Sen and colleagues say they are are hoping to develop the technique further, with plans to start clinical trials in humans next year.

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Nanochip could heal injuries or regrow organs with one touch, say researchers - The Guardian

The Potential of CRISPR

The potential of the gene editing toolCRISPRjust seems to keep growing and growing, and the latest experimental use of the technology is creating skin grafts that trigger the release of insulin and help manage diabetes.

Researchers have successfully tested the idea with mice that gained less weight and showed a reversed resistance to insulin because of the grafts (high insulin resistance is a common precursor to type 2 diabetes).

In fact, the team from the University of Chicago says the same approach could eventually be used to treat a variety of metabolic and genetic conditions, not just diabetes its a question of using skin cells to trigger different chemical reactions in the body.

We didnt cure diabetes, but it does provide a potential long-term and safe approach of using skin epidermal stem cells to help people with diabetes and obesity better maintain their glucose levels,says one of the researchers, Xiaoyang Wu.

If youre new to theCRISPR(Clustered Regularly Interspaced Short Palindromic Repeats) phenomenon, its a new and innovative way of editing specific genes in the body, using a biological copy and paste technique: it can doeverything fromcut out HIV virus DNA to slow thegrowth of cancer cells.

For this study, researchers used CRISPR to alter the gene responsible for encoding a hormone calledglucagon-like peptide-1(GLP-1), which triggers the release of insulin and then helps remove excess glucose from the blood.

Type 2 diabetescomes about due to a lack of insulin, also known as insulin resistance.

Using CRISPR, the GLP-1 gene could be tweaked to make its effects last longer than normal. The result was developed into skin grafts that were then applied to mice.

Around 80 percent of the grafts successfully released the edited hormone into the blood, regulating blood glucose levels over four months, as well as reversing insulin resistance and weight gain related to a high-fat diet.

Significantly, its the first time the skin graft approach has worked for mice not specially designed in the lab.

This paper is exciting for us because it is the first time we show engineered skin grafts can survive long term in wild-type mice, and we expect that in the near future this approach can be used as a safe option for the treatment of human patients,says Wu.

Human treatments will take time to develop but the good news is that scientists are today able to grow skin tissue very easily in the lab using stem cells, so that wont be an issue.

If we can make it safe, and patients are happy with the procedure, then the researchers say it could be extended to treat something likehaemophilia, where the body is unable to make blood clots properly.

Any kind of disease where the body is deficient in specific molecules could potentially be targeted by this new technique. And if it works with diabetes, it could be time to say goodbye to needles and insulin injections.

Other scientists who werent directly involved in the research, including Timothy Kieffer from the University of British Columbia in Canada, seem optimistic.

I do predict that gene and cell therapies will ultimately replace repeated injections for the treatment of chronic diseases, Kieffer told Rachel Baxter atNew Scientist.

The findings have been published inCell Stem Cell.

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CRISPR Skin Grafts Could Replace Insulin Shots For Diabetes - Futurism

The new research has been developed by a team led by Dr. Samuel I. Stupp, who is the director of Northwestern Universitys Simpson Querrey Institute for BioNanotechnology. The researcher is also Professor of Materials Science and Engineering, Chemistry, Medicine and Biomedical Engineering.The new technology centers on the way that cells behave in the human body. Our cells are continually being signaled with various instructions, triggered by proteins and other molecules that are located in the matrices that surround them. As an example, such signals can be cues for cells to express specific genes in order for the cells to differentiate into other types of cells. Such a development is important for growth or regeneration of tissues. This sophisticated, biological signaling machinery has the pre-programmed capacity to make signals stop and re-start as needed; or to switch off one signal and activate an alternative signal in order to commence a complex processes. If this could be controlled by medics, then the process of addressing a range of diseases could be achieved. So far, the ability to produce such regenerative therapies has proved impossible.This could be set to change with the development of a synthetic material that can trigger reversibly certain types of signaling. This platform could lead to materials to control stem cells in order to produce regenerative therapies and to control cellular functions. The new technology should help with research into treatments for such diseases as Alzheimers disease, Parkinsons disease, problems with arthritic joints, spinal cord injuries, the effects of stroke, and other conditions requiring tissue regeneration.In trials, the researchers have taken spinal cord neural stem cells (neurospheres) and driven them to differentiate using a signal, helping the scientists to understand developmental and regenerative cues. This cell manipulation technology could help control which cells change and thereby address diseases like Parkinsons, such as converting a patients own skin cells into stem cells. Commenting on the implications of the technology, Dr. Stupp said, in a communication provided to Digital Journal: Its important in the context of cell therapies for people to cure these diseases or regenerate tissues that are no longer functional.The research is an example of the use of digital based bio-nanotechnology. The technology has been published in the journal Nature Communications. The paper Instructing cells with programmable peptide DNA hybrids.

See the article here:
New technology manipulates cells for disease research - Digital Journal

Marrow Drives

The Office of Management and Budget has withdrawn a proposed rule banning compensation for hematopoietic stem cells. In other words, you can get paid when someone harvests stem cells from your bone marrow.

Bone marrow transplantation is used to treat a variety of ailments, including aplastic anemia, sickle cell anemia, bone marrow damage during chemotherapy, and blood cancers such as leukemia, lymphoma, and multiple myeloma. In 1984, Congress passed the National Organ and Transplant Act, which outlawed compensation to the donors of solid organs like kidneys and livers. Oddly, the act also defined renewable bone marrow as a solid organ.

Originally, hematopoietic stem cells were obtained from bone marrow obtained by inserting a needle into donors' hip bones. Researchers later developed a technique in which donors are treated with substance that overstimulates the production of hematopoietic stem cells, which then circulate in their bloodstreams. In a process similar to blood donation, the hematopoietic stem cells are then filtered from the donors' blood. The red blood cells and plasma are returned to the donors.

More Marrow Donors, a California-based nonprofit, wanted to set up a system to encourage hematopoietic stem cell donations with $3,000 awards, in the form of scholarships, housing allowances, or gifts to charity. The Institute for Justice, a libertarian law firm, brought suit on their behalf, and in 2012 a federal appeals court sensibly ruled that the law's ban on compensation for solid organ donations did not apply to stem cells obtained from donors' bloodstreams. The Obama administration reacted by proposing a regulation defining stem cells obtained from blood as the equivalent of a solid organ.

Now the new administration has withdrawn the proposal.

"Banning compensation for donors would have eliminated the best incentive we havemoneyfor persuading strangers to work for each other," Jeff Rowes, a senior attorney with the Institute for Justice, says in a press release. "Predictably, the ban on compensation for blood stem cell donors created chronic shortages and waiting lists. During the past four years, thousands of Americans needlessly died because compensation for bone marrow donors was unavailable."

The system of uncompensated donation is falling far short of meeting patient needs. As the Institute for Justice notes:

At any given time, more than 11,000 Americans are actively searching for a bone marrow donor. According to the New England Journal of Medicine, Caucasian potential donors are available and willing to donate about 51 percent of the time; Hispanic and Asian about 29 percent; and African-American about 23 percent. Caucasian patients can find a matching, available and willing donor about 75 percent of the time; Hispanic about 37 percent; Asian-American about 35 percent; and African-American patients only about 19 percent of the time. This demonstrates the huge gap between the need for compatible donors and the supply.

This is even more true in the case of solid organs from live and brain-dead donors. Right now there are more than 116,000 Americans waiting for a life-saving transplant organ. My colleagues and I at Reason have been arguing for decades in favor of compensating live donors for kidneys and pieces of their livers and the next-of-kin of brain-dead donors for other solid organs. If researchers and entrepreneurs succeed in boosting bone marrow donations by implementing various compensation schemes, perhaps that will prompt Congress to repeal its ill-conceived ban on compensation for organs donated for transplant.

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Trump Administration Withdraws Proposed Obama Ban on Compensation for Bone Marrow - Reason (blog)

Everybody likes playing with origami and making little paper animals, but researchers in the US have taken their hobby to a freaky new level.

Scientists have developed a way of making a kind of bioactive "tissue paper" from real body organs, which is thin and flexible enough to fold into origami animals like the charming crane you see above which was probably once a kidney, liver, or perhaps a heart.

While it definitely sounds a bit (okay, a lot) on the gross side, this organ origami isn't quite as gruesome as it sounds. For starters, the team from Northwestern University aren't sourcing their tissue paper from human organs at least, not that we know of.

Instead, the researchers are picking up unwanted pig and cow offal from a local butcher, and putting those discarded off-cuts to good use because this flexible paper-like material could one day be used to heal wounds, or to help supplement hormone production in cancer patients.

Northwestern University

"This new class of biomaterials has potential for tissue engineering and regenerative medicine as well as drug discovery and therapeutics," says one of the team, materials scientist Ramille Shah.

"It's versatile and surgically friendly."

The team stumbled upon the idea for making organ-based paper after a lucky accident during their research on 3D-printed mice ovaries.

A chance spill of the hydrogel-based gelatin ink used to make the ovaries ended up pooling into a dry sheet in the bench lab, and from one strange innovation, another was born.

"When I tried to pick it up, it felt strong," says one of the researchers, Adam Jakus.

"I knew right then I could make large amounts of bioactive materials from other organs. The light bulb went on in my head. I could do this with other organs."

Turning to pig and cow organs, the researchers extracted structural proteins called the extracellular matrix from animal ovaries, uteruses, kidneys, livers, muscles, and hearts.

These proteins, which help to give organs their form, were dried and then combined with a polymer to process them into their new paper-like structure.

In other words, it's a bit like papier-mch with a touch of H. P. Lovecraft thrown in, but what's important is that the paper retains residual biochemicals from its protein-based origins, holding on to cellular properties from the specific organ it comes from.

During tests in the lab, the team was able to grow functional, hormone-secreting ovarian follicles in culture using tissue paper sourced from a cow ovary.

It might only be a lab test using animal organs, but if the same idea could be replicated with human hormone-producing tissue paper implanted under patients' skin, it could be a big step towards treating cancer patients and hormone deficiency generally.

"This could provide another option to restore normal hormone function to young cancer patients who often lose their hormone function as a result of chemotherapy and radiation," explains one of the researchers, Teresa Woodruff.

What could make the tissue paper so easy to apply for medical purposes is its malleability. It feels and folds much like ordinary paper, and can even be frozen for later use.

"Even when wet, the tissue papers maintain their mechanical properties and can be rolled, folded, cut and sutured to tissue," says Jakus.

In addition to hormone treatment applications, the team says the pliable material could augment tissue when wounds are healing, which might be able to speed up recoveries, or prevent scarring from injuries.

Of course, before we even get close to sticking origami organs inside human patients, the next step will be looking into how the paper works in animal models.

But initial signs look promising. When the team put human bone marrow stem cells on the tissue paper, all the stem cells attached and multiplied.

"That's a good sign that the paper supports human stem cell growth," says Jakus.

"It's an indicator that once we start using tissue paper in animal models it will be biocompatible."

To be clear, there's still a lot more research to be done here before we know how viable organ paper really is, but we'll never know unless we try.

And in the meantime, at least one thing's for sure.

"It is really amazing that meat and animal by-products like a kidney, liver, heart and uterus can be transformed into paper-like biomaterials that can potentially regenerate and restore function to tissues and organs," says Jakus.

"I'll never look at a steak or pork tenderloin the same way again."

The findings are reported in Advanced Functional Materials.

Read the rest here:
Scientists Are Making Actual Origami Out of Body Organ Tissue - ScienceAlert

Stem cell therapy has been claimed to cure cancer, improve chronic conditions such as headaches, and even make your skin look younger. How can that not be a good thing?

Youve probably heard about stem cell research before, but what exactly are stem cells, and how can stem cells injected into the body treat various diseases and conditions?

There has been enormous progress in this field over the last few decades, so let's take a look at how stem cell injections work.

What exactly are stem cells?

Stem cells are the bodys building blocks the reserve cells that the body is made up of. These cells are able to produce multiple different cells, each performing a specific function. Stem cells can be divided into two main categories:

What is stem cell therapy?

Stem cell therapy can be categorised as regenerative medicine. Stem cells used in medical treatments are currently harvested from three sources: umbilical cord blood, bone marrow and blood. These are treatments that restore damaged tissue and regenerate new cells in the case of illness or injury.

While there are other forms of stem cell therapy, these are still in the early stages and regarded as research.

How is stem cell therapy performed?

Adult stem cells are derived from a blood sample and injected back into the patient's blood. The surrounding cells are then activated, stimulating rejuvenation in the area.

Why the controversy?

In 2004 South Africa became the first African nation to open a stem cell bank. This involved embryonic stem cells for cloning research and not the "adult" stem cells used in treatment.

Embryonic stem cells are often viewed as problematic, as they are derived from very young foetuses. It is thus viewed as a form of "abortion" to use embryonic stem cells for treatment. But in most cases of stem cell therapy adult stem cells are used, which causes few ethical problems. Stem cells derived from the umbilical cord are not the same as from the embryo.

What does science say?

Prof Jacqui Greenberg from the University of Cape Town stated that although stem cells can potentially treat various diseases, they should be treated with extreme care.

She has no doubt that in time (in medical science particularly, progress is slow and measured in blocks of 10 years), stem cells will be the solution for many things. "But right now we have to strike a balance of not creating too much hype and raising hope too soon. Stem cells are the future, but the future is not now," Greenberg states.

The reason for this is that stem cells derived from an adult are too volatile at times. Researchers are not clear on how many of these stem cells will actually "survive" and "activate" to treat the condition at hand. Therefore it can't be predicted how many cells will survive and become functional.

There is as yet little proof that stem cells can actually fight disease when injected back into the host.Despite the success of IPS cell technology up to date, there are stillchallenges with regard to the purity of stem cells before their use in therapy.

Availability and cost in South Africa

Stem cell therapy is available at various treatment centres in South Africa. One of the most prominent is the South African Stem Cell Institute in the Free State. Here, various treatments, such as regenerative skin treatments and prolotherapy (regeneration of the joints), are offered.

Therapy starts with an initial consultation. During the second consultation vitals are checked, followed by either the fat harvest procedure under tumescent anaesthesia or bone marrow aspiration under local anaesthesia.

The stem cells are then cryopreserved and injected into the patient as needed. Prices of the treatment vary from R500 (for a once-off treatment in a small area, such as the hand) to R22 500 (a comprehensive process), depending on the condition being treated and length of treatment needed. This excludes the initial consultation fee and after-care.

There are also stem cell banks in South Africa, such as Cryo-Save, where stem cells can be stored at an annual fee (excluding initial consultation, testing and harvesting) and used for treatment.

Do your own research

If you do want to go the stem cell route, make sure that the medical programme being offered is legitimate and that the projected outcome is based on real evidence.

There are a number of private institutions banking on the promise of curing any number of diseases with stem cells from a patient's own blood. The truth, however, is that there is no conclusive proof that the majority of these diseases can be cured with the person's own stem cells annihilating the claim that stem cell therapy is the solution to all diseases.

Excerpt from:
Is stem cell injection the cure-all miracle? - Health24

Brave Ava Stark has gone back to nursery for the first time since undergoing a life-saving bone marrow transplant.

The four-year-old was all smiles when she arrived at Noahs Ark Nursery in Lochgelly, Fife, yesterday morning.

She said she was looking forward to laughing at the nursery teacher and happily ran around the toy-filled garden before starting at 8am.

But her return was cut short after another child was ill and she had to go home due to her lowered immune system.

Mum Marie said that the half an hour she spent at the nursery was a great start and theyre now looking forward to her returning for longer.

The 34-year-old said: Its absolutely amazing that shes managed to get back to nursery for the first time. We honestly didnt think this day would ever happen.

My mum has been teaching her at home and I think shes going to miss her wee side-kick. It really is a big day.

She may only have been able to stay for half hour but thats a great start and were aiming for more next week. She was too excited to sleep last night.

If it wasnt for all those amazing people who heard about Ava and registered to become donors, then we may never have got here.

We just cant thank everyone who supported us enough.

Nursery manager Karen Robertson added: Its absolutely fantastic, we couldnt have wished for a better outcome.

We always said that when she took ill that we couldnt wait until she was well enough to come back and Im just delighted to see her.

Ava underwent her stem cell transplant in December after a Daily Record appeal which saw more than 83,000 people across the UK sign up to try help her.

She was first diagnosed with inherited bone marrow failure in April 2016 and relied on blood and platelet transfusions to keep her alive.

A matching donor was initially found but pulled out weeks before the procedure went ahead prompting her brave mum to launch the worldwide appeal for help.

A second match was then found but they pulled out just 24 hours before the youngster was due to go to hospital leaving her entire family devastated.

The campaign continued and two more matching donors were eventually found meaning she could undergo the operation in December.

She has recently celebrated her 100 day post-transplant milestone and will become the face of a donor recruitment drive by the Anthony Nolan charity.

She was also named one of the Daily Records Little Heroes at an award ceremony in May.

See the original post here:
Bone marrow transplant tot Ava Stark goes back to nursery for first time since live-saving op - Scottish Daily Record

Japanese pharma major Daiichi Sankyo (TYO: 4568) revealed this morning that it has signed an investment contract with Cuorips Inc, an Osaka University spin-off venture to receive an option right concerning the worldwide commercialization of iPS-derived cardiomyocyte (iPS-CM) sheet developed by Cuorips.

The iPS-CM sheet is an allogeneic cell therapy product consisting of cardiomyocyte derived from human iPS cells. Its transplantation is expected to provide improvement of cardiac function and amelioration of heart failure and become a new treatment option for patients with severe heart failure, who have no remedies other than heart transplantation or artificial heart implantation.

Based on the cutting-edge cell therapy research targeting heart diseases, the team at the Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, led by Professor Yoshiki Sawa, has been working on the iPS-CM research and development by participating in the Research Center Network for Realization of Regenerative Medicine, which is run by the Japan Agency for Medical Research and Development (AMED). They are currently preparing for clinical research as well as investigator initiated clinical study.

Cuorips was founded to develop and commercialize iPS-CM sheets based on the research data and technologies developed by the university.

Daiichi Sankyo has been conducting research on iPS cell-derived cardiomyocyte and their production, and is currently working on the efficient production process capable for commercial supply. Daiichi Sankyo and Cuorips are aiming to commercialize iPS-CM sheets as a pioneering treatment for severe heart failure.

Read more from the original source:
Daiichi Sankyo invests in Osaka University spin-off - The Pharma Letter (registration)

Gary Campbell's partner, Leanne Crawford, with helpers Lauryn MacKenna (9) and Kye Crawford (12) at the home baking stall at Sunday's fun day.PICTURE IAIN FERGUSON, THE WRITE IMAGE

Two fundraising events have been held in Lochaber at the weekend to help send a local man halfway round the world for ground-breaking treatment.

Gary Campbell, from Caol, who is just 29, was diagnosed with progressive MS in April.

But family, friends and supporters are pulling out all the stops to raise 45,000 in order to send him to Mexico for stem-cell treatment.

Hematopoietic stem cell transplantation, HSCT, involves the intravenous infusion of stem cells collected from bone marrow or peripheral blood.

On Saturday, youngsters and their parents took park in a teddy bear toddle to raise cash for Mr Campbells cause, while yesterday, a large crowd attended a fun afternoon at An Aird in Fort William.

Leanne Crawford, Garys partner, said: The fun day went really well and there was a good turnout.

There were lots of fund-raising activities including a charity shinty match, bouncy castle, beat the goalie, a nail bar, home baking and raffles with loads of prizes.

Gary used to play shinty and two of his old teams from Caol and Banavie took part. Some people had obviously not played in a wee while and were falling about, but it was great fun.

Unfortunately, Gary wasnt well enough to come along which was a pity as he would have really enjoyed it.

He had a fall in the garden on Saturday night and I couldnt get him up. Fortunately his mum was there and between us we managed to get him back into the wheelchair.

His right leg was shaking constantly it was really stressful for him.

Ms Crawford said she has known Mr Campbell for nine years, and for the past five has noticed different symptoms.

He wasnt very good on his feet and sometimes his legs would give way. It was as though he was drunk when he hadnt been drinking.

Gary actually thought he had a brain tumour and, in a way, it was a relief when he was diagnosed with MS.

Ms Crawford said she is hoping by the time enough cash is raised, HSCT might be available closer to home.

I believe hospitals in Galway and Sheffield are looking into the treatment, but if he has to go to Mexico, Ill get him on the plane even though he is petrified of flying.

I dont know how much has been raised yet from these events, but we collected 663 from a recent baking sale and have 2,500 already on the just giving page. We also have 1,500 in a bank account collected from fund-raising events.

Gary is over the moon with the support he is receiving.

Another Lochaber resident with MS, Frances OConnell, received HSCT in Mexico last year.

Excerpt from:
Fun weekend activities will help send Lochaber man for ground-breaking MS treatment - Press and Journal

Current debate about the future of the Victorian Heart Hospital, which when completed will be Australia's first cardiac hospital,focuses on issues such as cost and contracts. And, in these tight economic times, it is right to ask these questions.

However, Australia's first dedicated specialist heart hospital will be so much more. Thehospital will be in the same league as some of the great cardiac hospitals, such as the Barts Heart Centre in London and the Montreal Heart Institute in Canada.

More Victorians, men and women, die from heart disease than any other cause. People are living longer long enough to have, and survive, heart attacksthat may become heart disease and heart failure further down the line.

In the catchment area that will feed into the Victorian Heart Hospital the population projections for people at risk of heart disease are even worse. Aboutone-quarter (or eight out of 31) of the metropolitan local government areas with above average heart attack rates fall into the catchment area of the new hospital. This is an area whose population needs a facility like this.

But the hospitalwill be so much more than a hospital for patients with cardiovascular disease and events. Much has been said about the dedicated areas for Monash University and Monash Health researchers devoted to cardiac research.

Having the researchers sitting in the midst of the clinicians and patients, and in many cases being situated within the hospital means the problems the scientists address are the ones that are identified by those at the coalface, the clinicians and health professionals.

One of the hospital'score research areas, for example, will be stem cell research. We have recruited some of the best stem cell scientists in the world. They will work with Monash University's Australian Regenerative Medicine Institute and heart hospital clinicians to develop cellular patches that can be created from a patient's own cells to replace the areas of the heart left dead by a heart attack. This damaged tissue, currently cannot be fixed, and often leads to heart failure, so the need for this sort of research is paramount.

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Monash Health has an outstanding international reputation for attracting clinical trials into new heart procedure techniques, with more than 30 trials currently being conducted. As an example, the international medical device makerMedtronicchose Monash Heart cardiologists to conduct the first trial of a new way to replace mitral valves in the hearts of patients whose health would not withstand traditional open-heart surgery. These trial patients have had their life saved by this device.

This is translational research at its best taking new discoveries and therapies and making sure they are safe in patients. These innovations then become, as fast as possible, treatments we can offer all Victorians. It is no surprise that many of Australia's largest medical device manufacturers and innovators are situated around Monash University and benefit from the strong biomedical focus the university offers.

Co-location of the Victorian Heart Hospital at the Monash University campus will strengthen the nexus between industry, biomedical research and clinical care, including clinical trials that will result in Victorians benefiting from the best advances in cardiac care.

The Victorian Heart Hospitalis a way for Victoria to future-proof its citizens against heart disease for the next five decades. It will be where we develop new technologies, devices and treatments that can be used to deal with the patients that come throughour doors.

There will be more non-surgical alternatives and prevention strategies developed and offered. We will provide a health and wellness department that assists patients in dealing with the depression that can follow cardiac surgery, as well as assisting patients in techniques that can help them lower their risk of further cardiac events.

The hospitalwill not only put Victoria on the world map, it will be a groundbreaking commitment to the health of Victorians.

Sarah Newton is deputy dean, external relations, Monash University's faculty of medicine, nursing and health sciences.

See the original post here:
Funding debate aside, this is why we need a new heart hospital - The Sydney Morning Herald

A syrinx is a fluid-filled cavity within the spinal cord (syringomyelia) or brain stem (syringobulbia). Predisposing factors include craniocervical junction abnormalities, previous spinal cord trauma, and spinal cord tumors. Symptoms include flaccid weakness of the hands and arms and deficits in pain and temperature sensation in a capelike distribution over the back and neck; light touch and position and vibration sensation are not affected. Diagnosis is by MRI. Treatment includes correction of the cause and surgical procedures to drain the syrinx or otherwise open CSF flow.

Syrinxes usually result from lesions that partially obstruct CSF flow. At least half of syrinxes occur in patients with congenital abnormalities of the craniocervical junction (eg, herniation of cerebellar tissue into the spinal canal, called Chiari malformation), brain (eg, encephalocele), or spinal cord (eg, myelomeningocele). For unknown reasons, these congenital abnormalities often expand during the teen or young adult years. A syrinx can also develop in patients who have a spinal cord tumor, scarring due to previous spinal trauma, or no known predisposing factors. About 30% of people with a spinal cord tumor eventually develop a syrinx.

Syringomyelia is a paramedian, usually irregular, longitudinal cavity. It commonly begins in the cervical area but may extend downward along the entire length of the spinal cord.

Syringobulbia, which is rare, usually occurs as a slitlike gap within the lower brain stem and may disrupt or compress the lower cranial nerve nuclei or ascending sensory or descending motor pathways.

See the original post:
Syrinx of the Spinal Cord or Brain Stem - Neurologic ...

Dr. Gabe Mirkin

Sam Shepard was a prolific playwright, actor, screenwriter and director who:

acted in more than sixty films and was nominated for an Academy Award for Best Supporting Actor for his portrayal of pilot Chuck Yeager inThe Right Stuff;

wrote more than 55 plays, often focusing on the serious problems that occur in American family life;

won the most Obie Awards (10) for his off-Broadway writing and directing. In 1979 he received a Pulitzer Prize for his play, Buried Child, andNew York Magazinecalled him the greatest American playwright of his generation.

In his late sixties, he developed amyotrophic lateral sclerosis (ALS), the disease that killed baseball great Lou Gehrig at age 37. Shepard died from complications of ALS on July 27, 2017, at age 73.

A Difficult Life

Sam Shepard

He was born on November 5, 1943, in Fort Sheridan, Illinois. His dysfunctional family served as a basis for characters in many of his plays. His father was a United States Army Air Forces bomber pilot during World War II who was also an alcoholic and an abusive husband and father. His loving, supportive mother, a teacher, offset some of the pain and abuse he suffered from his father. In his early years, the family had to move every two years because of army transfers. Later his father left the service and bought an avocado farm in Duarte, Calif. Shepard briefly studied animal husbandry at nearby Mt. San Antonio College, but soon left school to move to New York City, where he worked as a busboy, played in a psychedelic folk band and tried to break into the theater.

At age 35, his acting career took off when he won a role in Terrence MalicksDays of Heaven, with Richard Gere and Brooke Adams. At the same time, he continued to write successful plays and in 1986 (age 43) he was elected to the American Academy of Arts and Letters.

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrigs Disease)

In his last few years, Shepard suffered privately from ALS, but he described his experience in his last book, The One Inside. One of the characters said that he couldnt get up from bed in the morning and felt as though his limbs werent connected to the motor driving his body. They wont take direction wont be dictated to the arms, legs, feet, hands. Nothing moves. Nothing even wants to. The brain isnt sending signals.

ALS is a progressive disease that destroys the nerves that move voluntary muscles. More than 6,000 people in the United States are diagnosed with ALS each year. Nobody knows the cause and there is no cure. The brain is supposed to send messages to nerves in the spinal cord which transmit messages to the nerves that move muscles. When a muscle loses its nerve control, it starts to twitch and can waste away to nothing. Early symptoms of ALS include

muscle weakness

twitching

slurred speech

inability to chew food

tripping or stumbling.

The first sign could be difficulty buttoning a shirt, writing, or turning a key in a lock. The disease usually does not affect a persons ability to think and reason, so affected people are terribly disturbed by their lack of ability to control their voluntary muscles. As the disease progresses, a person loses the ability to speak, eat, walk, and eventually breathe. The most common cause of death is inability to breathe, which typically occurs about 3-5 years after symptoms start. Only about ten percent of affected people live more than ten years after first being diagnosed.

Risk Factors and Diagnosis

The disease usually starts between the ages of 55 and 75, but there are no known specific risk factors. Military veterans appear to be twice as likely as non-veterans to develop ALS. Possible causes could be exposure to occupational or environmental toxins such as lead or pesticides, infections or trauma. Family history does not appear to predict the disease.

There are no specific tests to diagnose ALS. It is usually diagnosed by a history of the symptoms, physical examination and ruling out other causes.

Current Treatments and Research

The U.S. Food and Drug Administration (FDA) has approved riluzole (Rilutek) and edaravone (Radicava) to treat ALS. These drugs offer no hope for a cure, but Riluzole appears to protect nerves by decreasing glutamate, the chemical messenger for nerves that innervate muscles. Intravenous edaravone possibly slows loss of muscle function, but it costs $1,086 per infusion or a yearly cost before government discount of $145,524. Another drug under European review is being developed by French drug maker AB Science SA (ABS.PA). Since there is no cure, all patients should receive physical therapy and speech therapy because inactivity itself causes loss of muscle function.

Since ALS is caused by the death of nerve cells that cause muscles to contract, the most promising line of research is through stem cells. Stem cells are young cells that can become any type of tissue. Treatment in the future may be to program stem cells to become nerve cells that innervate muscles and then inject them into areas where the nerve cells have already died.

Dr. Gabe Mirkin is a Villager. Learn more at http://www.drmirkin.com

Read the rest here:
Sam Shepard and Amyotrophic Lateral Sclerosis - Villages-News

Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntingtons disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.

By creating HD in a dish, the researchers say they have taken a major step forward in efforts to better understand what disables and kills the cells in people with HD, and to test the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

Although the autosomal dominant gene mutation responsible for HD was identified in 1993, there is no cure. No treatments are available even to slow its progression.

The research, published in the journal Cell Stem Cell, is the work of a Huntingtons Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups. The consortium studied several other HD cell lines and control cell lines in order to make sure results were consistent and reproducible in different labs.

The general midlife onset and progressive brain damage of HD are especially cruel, slowly causing jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and eventually death. In some cases (as in the patient who donated the material for the cells made at Johns Hopkins), the disease can strike earlier, even in childhood.

Having these cells will allow us to screen for therapeutics in a way we havent been able to before in Huntingtons disease, says Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the studys lead researchers. For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic.

Ross and his team, as well as other collaborators at Johns Hopkins and Emory University, are already testing small molecules for the ability to block HD iPSC degeneration. These small molecules have the potential to be developed into novel drugs for HD.

The ability to generate from stem cells the same neurons found in Huntingtons disease may also have implications for similar research in other neurodegenerative diseases such as Alzheimers and Parkinsons.

To conduct their experiment, Ross took a skin biopsy from a patient with very early onset HD. When seen by Ross at the HD Center at Hopkins, the patient was just seven years old. She had a very severe form of the disease, which rarely appears in childhood, and of the mutation that causes it. Using cells from a patient with a more rapidly progressing form of the disease gave Ross team the best tools with which to replicate HD in a way that is applicable to patients with all forms of HD.

Her skin cells were grown in culture and then reprogrammed by the lab of Hongjun Song, Ph.D., a professor at Johns Hopkins Institute for Cell Engineering, into induced pluripotent stem cells. A second cell line was generated in an identical fashion in Dr. Rosss lab from someone without HD. Simultaneously, other HD and control iPS cell lines were generated as part of the NINDS funded HD iPS cell consortium.

Scientists at Johns Hopkins and other consortium labs converted those cells into generic neurons and then into medium spiny neurons, a process that took three months. What they found was that the medium spiny neurons deriving from HD cells behaved just as they expected medium spiny neurons from an HD patient would. They showed rapid degeneration when cultured in the lab using basic culture medium without extensive supporting nutrients. By contrast, control cell lines did not show neuronal degeneration.

These HD cells acted just as we were hoping, says Ross, director of the Baltimore Huntington's Disease Center. A lot of people said, Youll never be able to get a model in a dish of a human neurodegenerative disease like this. Now, we have them where we can really study and manipulate them, and try to cure them of this horrible disease. The fact that we are able to do this at all still amazes us.

Specifically, the damage caused by HD is due to a mutation in the huntingtin gene (HTT), which leads to the production of an abnormal and toxic version of the huntingtin protein. Although all of the cells in a person with HD contain the mutation, HD mainly targets the medium spiny neurons in the striatum, part of the brains basal ganglia that coordinates movement, thought and emotion. The ability to work directly with human medium spiny neurons is the best way, researchers believe, to determine why these specific cells are susceptible to cell stress and degeneration and, in turn, to help find a way to halt progression of HD.

Much HD research is conducted in mice. And while mouse models have been helpful in understanding some aspects of the disease, researchers say nothing compares with being able to study actual human neurons affected by HD.

For years, scientists have been excited about the prospect of making breakthroughs in curing disease through the use of stem cells, which have the remarkable potential to develop into many different cell types. In the form of embryonic stem cells, they do so naturally during gestation and early life. In recent years, researchers have been able to produce induced pluripotent stem cells (iPSCs), which are adult cells (like the skin cells used in Rosss experiments) that have been genetically reprogrammed back to the most primitive state. In this state, under the right circumstances, they can then develop into most or all of the 200 cell types in the human body.

The other members of the research consortium include the University of Wisconsin School of Medicine, Massachusetts General Hospital and Harvard Medical School, the University of California, San Francisco, Cardiff University the Universita degli Studi diMilano and the CHDI Foundation.

Primary support for this research came from an American Recovery and Reinvestment Act (ARRA) grant (RC2-NS069422) from the National Institutes of Healths National Institute of Neurological Disorders and Stroke and a grant from the CHDI Foundation, Inc.

Other Johns Hopkins researchers involved in this study include Sergey Akimov, Ph.D.; Nicolas Arbez, Ph.D.; Tarja Juopperi, D.V.M., Ph.D.; Tamara Ratovitski; Jason H. Chiang; Woon Roung Kim; Eka Chighladze, M.S., M.B.A.; Chun Zhong; Georgia Makri; Robert N. Cole; Russell L. Margolis, M.D.; and Guoli Ming, M.D., Ph.D.

Read more here:
Turning Skin Cells Into Brain Cells - 06/28/2012

A proof-of-concept study in mice has demonstrated how skin grafts could deliver gene therapy for obesity and diabetes.

'We think this platform has the potential to lead to safe and durable gene therapy, in mice and we hope, someday, in humans, using selected and modified cells from skin,' said senior author Dr Xiaoyang Wu of the University of Chicago, Illinois.

The technique explores the potential of glucagon-like peptide 1 (GLP1), a hormone which could help to treat conditions like diabetes and obesity. GLP1 reduces appetite and stimulates the release of insulin to lowerblood sugar, butdoes not last long in the blood and is challenging to deliver orally.

The researchers used CRISPR to edit skin stem cellstaken from newborn mice. They inserted a modified version of the GLP1 gene, designed to increase the duration of the hormone, and a genetic'switch' to turn the gene on in the presence of an antibiotic.

They grew the skin stem cells into a skin organoids, and grafted them onto mice. When the mice were fed small amounts of antibiotic, theysuccessfully produced modified GLP1, which lasted for three months, and showed higher levels of insulin and lower levels of glucose.

The researchers also tested feeding the mice a high-fat diet. Compared to controls, the mice with modified GLP1 skin grafts put on less weight.

Dr Wu said the skin graft method could be safer than using engineered viral vectorsto edit genes in patient's own tyissues, as viruses 'may cause a very strong immune reaction and inflammation in vivo.' He added that lab-grown skin grafts have been used clinically for some time to treat burns, and have been proven safe.

Being able to control the gene expression using a drug would also allow doctors to calibrate how much of the enzyme enters a patients bloodstream.

'We think this can provide a long-term safe option for the treatment of many diseases,' Dr Wu said. 'It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as haemophilia. Or it could function as a metabolic sink, removing various toxins.'

Dr Jeffrey Millman of Washington University, St Louis, who was not involved in the study, told The Scientist that more research would be needed to ensure that neither the CRISPR editing nor the stem cell culturing method inadvertently introduce dangerous mutations.

Read more here:
Gene therapy skin grafts for obesity and diabetes - BioNews

Stem cells have been cultured to treat many different of conditions

Lewis Houghton/Science Photo Library

By Andy Coghlan

Last week, two people with type 1 diabetes became the first to receive implants containing cells generated from embryonic stem cells to treat their condition. The hope is that when blood sugar levels rise, the implants will release insulin to restore them to normal.

About 10 per cent of the 422 million people who have diabetes worldwide have type 1 diabetes, which is caused by the bodys immune system mistakenly attacking cells in the pancreas that make insulin. For more than 15 years, researchers have been trying to find a way to use stem cells to replace these, but there have been several hurdles not least, how to get the cells to work in the body.

Viacyte, a company in San Diego, California, is trying a way to get round this. The firms thumbnail-sized implant, called PEC-Direct, contain cells derived from stem cells that can mature inside the body into the specialised islet cells that get destroyed in type 1 diabetes.

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The implant sits just below the skin, in the forearm, for example, and is intended to automatically compensate for the missing islet cells, releasing insulin when blood sugar levels get too high.

If it works, we would call it a functional cure, says Paul Laikind, of Viacyte. Its not truly a cure because we wouldnt address the autoimmune cause of the disease, but we would be replacing the missing cells.

The device has already been safety tested in 19 people with diabetes, using smaller numbers of stem cells. Once implanted, the progenitor cells housed in the device did mature into islet cells, but the trial didnt use enough stem cells to try to treat the condition.

Now Viacyte has implanted PEC-Direct packages containing more cells into two people with type 1 diabetes. A third person will also get the implant in the near future. Once inside the body, pores in the outer fabric of the device allow blood vessels to penetrate inside, nourishing the islet progenitor cells and exposing them to growth factors that push them to mature. Once these cells have matured which should take about three months the hope is that they will be able to monitor sugar levels in the blood, and release insulin as required.

If effective, it could free people with type 1 diabetes from having to closely monitor their blood sugar levels and inject insulin, although they would need to take immunosuppressive drugs to stop their bodies from destroying the new cells.

If successful, this strategy could really change the way we treat type 1 diabetes in the future, says Emily Burns of the charity Diabetes UK. A similar way to treat the condition with pancreas cells from organ donors has been in use for nearly 20 years, successfully freeing recipients from insulin injections, but a shortage of donors limits how many people are able to have this treatment.

This isnt a problem with stem cells. The embryonic stem cells used to make the progenitor cells originally came from a spare early stage embryo donated by a woman who was having IVF. Because embryonic stem cells, and the progenitor cells made from them, can be multiplied in limitless amounts, Laikand says that, if the treatment works, the method would be able to treat everyone who has the condition.

A limitless source of human insulin-producing cells would be a major step forward on the journey to a potential cure for diabetes, says James Shapiro at the University of Alberta, Canada, who has collaborated with Viacyte on this project, and who pioneered the donor pancreas method decades ago. For sure, this will in the end prove to be a durable landmark for progress in diabetes care.

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Excerpt from:
First implants of stem-cell pouches to 'cure' type 1 diabetes - New Scientist