Ruxolitinib (trade name: Jakavi) has been approved since August 2012 for the treatment of adults with myelofibrosis. In an early benefit assessment pursuant to the Act on the Reform of the Market for Medicinal Products (AMNOG), the German Institute for Quality and Efficiency in Health Care (IQWiG) examined whether this new drug offers an added benefit over the appropriate comparator therapy specified by the Federal Joint Committee (G-BA).

According to the results, there is an indication of considerable added benefit in comparison with "best supportive care" (BSC) because ruxolitinib is better at relieving symptoms. Moreover, a hint of an added benefit with regard to survival can be derived from the dossier. Its extent is non-quantifiable, however.

Bone marrow is replaced by connective tissue

Myelofibrosis is a rare disease of the bone marrow, in which the bone marrow is replaced by connective tissue. As a consequence of this so-called fibrosis, the bone marrow is no longer able to produce enough blood cells. Sometimes the spleen or the liver takes over some of the blood production. Then these organs enlarge and can cause abdominal discomfort and pain. The typical symptoms also include feeling of fullness, night sweats and itching. Some patients with myelofibrosis develop leukemia.

Stem cell transplantation is currently the only option to cure myelofibrosis. The drug ruxolitinib aims to relieve the symptoms of myelofibrosis.

G-BA specifies appropriate comparator therapy

Ruxolitinib is an option for patients with so-called primary or secondary myelofibrosis whose spleen is already enlarged (splenomegaly) or who have other disease-related symptoms.

The G-BA specified "best supportive care" (BSC) as appropriate comparator therapy. BSC means a therapy that provides the patient with the best possible, individually optimized, supportive treatment to alleviate symptoms and improve quality of life. This also includes adequate pain therapy.

Relevant study ongoing until 2015

In its assessment, IQWiG could include one randomized controlled trial (RCT) conducted in 89 centres in Australia, Canada and the United States (COMFORT-I). The 309 patients in total were either treated with ruxolitinib plus BSC or with placebo plus BSC.

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Ruxolitinib for myelofibrosis: Indication of considerable added benefit

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Newswise A new study by Barbara Beltz, the Allene Lummis Russell Professor of Neuroscience at Wellesley College, and Irene Sderhll of Uppsala University, Sweden, published in the August 11 issue of the journal Developmental Cell, demonstrates that the immune system can produce cells with stem cell properties, using crayfish as a model system. These cells can, in turn, create neurons in the adult animal. The flexibility of immune cells in producing neurons in adult animals raises the possibility of the presence of similar types of plasticity in other animals.

We have been suspicious for some time that the neuronal precursor cells (stem cells) in crayfish were coming from the immune system, Beltz wrote. The paper contains multiple lines of evidence that support this conclusion, in addition to the experiments showing that blood cells transferred from a donor to a recipient animal generate neurons.

Beltz, whose research focuses on the production of new neurons in the adult nervous system, uses the crustacean brain as the model system because the generations of precursor cells are spatially segregated from one another. According to Beltz, this separation is crucial because it allowed the researchers to determine that the first generation precursors do not self-renew. For the Developmental Cell study, the cells of one crayfish were labeled and this animals blood was used for transfusions into another crayfish. They found that the donor blood cells could generate neurons in the recipient.

In many adult organisms, including humans, neurons in some parts of the brain are continually replenished. While this process is critical for ongoing health, dysfunctions in the production of new neurons may also contribute to several neurological diseases, including clinical depression and some neurodegenerative disorders.

Beltz notes, of course, that it is difficult to extrapolate from crayfish to human disease. However, because of existing research suggesting that stem cells harvested from bone marrow also can become neural precursors and generate neurons, she says it is tempting to suggest that the mechanism proposed in crayfish may also be applicable in evolutionarily higher organisms, perhaps even in humans.

Prior studies conducted in both humans and mice and published about a decade ago, showed that bone marrow recipients who had received a transplant from the opposite gender had neurons with the genetic signature of the opposite sex. The implication was that cells from the bone marrow generated those neurons. However, it is currently thought that neuronal stem cells in mammals, including humans, are self-renewing and therefore do not need to be replenished. Thus, these findings have not been interpreted as contributing to a natural physiological mechanism.

Every experiment we did confirmed the close relationship between the immune system and adult neurogenesis, Beltz said. Often when one is doing research, experiments can be fussy or give variable results. But for this work, once we started asking the right questions, the experiments worked first time and every time. The consistency and strength of the data are remarkable.

Our findings in crayfish indicate that the immune system is intimately tied to mechanisms of adult neurogenesis, suggesting a much closer relationship between the immune system and nervous system than has been previously appreciated, said Sderhll. If further studies demonstrate a similar relationship between the immune system and brain in mammals, these findings would stimulate a new area of research into immune therapies to target neurological diseases.

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Blood Cells Generate Neurons in Crayfish; Could Have Implications for Treatment of Neurodegenerative Disorders

A new research has suggested that immune cells, known as natural killer cells could help in hunting down and kill cancers that have spread in the body.

The study showed that a protein called MCL-1 was vital for survival of natural killer cells.

Dr. Nick Huntington said that they discovered that MCL-1 was absolutely essential for keeping natural killer cells alive and without natural killer cells, the body was unable to destroy melanoma metastases that had spread throughout the body, and the cancers overwhelmed the lungs.

Huntington said that the natural killer cells led the response that caused rejection of donor stem cells in bone marrow transplantations and they also produced inflammatory signals that could result in toxic shock syndrome, a potentially fatal illness caused by bacterial toxins that causes a whole-body inflammatory reaction.

The study is published in the journal Nature Communications.

(Posted on 15-08-2014)

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'Killer' immune cells destroy body cancer

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Newswise A genetic variation linked to schizophrenia, bipolar disorder and severe depression wreaks havoc on connections among neurons in the developing brain, a team of researchers reports. The study, led by Guo-li Ming, M.D., Ph.D., and Hongjun Song, Ph.D., of the Johns Hopkins University School of Medicine and described online Aug. 17 in the journal Nature, used stem cells generated from people with and without mental illness to observe the effects of a rare and pernicious genetic variation on young brain cells. The results add to evidence that several major mental illnesses have common roots in faulty wiring during early brain development.

This was the next best thing to going back in time to see what happened while a person was in the womb to later cause mental illness, says Ming. We found the most convincing evidence yet that the answer lies in the synapses that connect brain cells to one another.

Previous evidence for the relationship came from autopsies and from studies suggesting that some genetic variants that affect synapses also increase the chance of mental illness. But those studies could not show a direct cause-and-effect relationship, Ming says.

One difficulty in studying the genetics of common mental illnesses is that they are generally caused by environmental factors in combination with multiple gene variants, any one of which usually could not by itself cause disease. A rare exception is the gene known as disrupted in schizophrenia 1 (DISC1), in which some mutations have a strong effect. Two families have been found in which many members with the DISC1 mutations have mental illness.

To find out how a DISC1 variation with a few deleted DNA letters affects the developing brain, the research team collected skin cells from a mother and daughter in one of these families who have neither the variation nor mental illness, as well as the father, who has the variation and severe depression, and another daughter, who carries the variation and has schizophrenia. For comparison, they also collected samples from an unrelated healthy person. Postdoctoral fellow Zhexing Wen, Ph.D., coaxed the skin cells to form five lines of stem cells and to mature into very pure populations of synapse-forming neurons.

After growing the neurons in a dish for six weeks, collaborators at Pennsylvania State University measured their electrical activity and found that neurons with the DISC1 variation had about half the number of synapses as those without the variation. To make sure that the differences were really due to the DISC1 variation and not to other genetic differences, graduate student Ha Nam Nguyen spent two years making targeted genetic changes to three of the stem cell lines.

In one of the cell lines with the variation, he swapped out the DISC1 gene for a healthy version. He also inserted the disease-causing variation into one healthy cell line from a family member, as well as the cell line from the unrelated control. Sure enough, the researchers report, the cells without the variation now grew the normal amount of synapses, while those with the inserted mutation had half as many.

We had our definitive answer to whether this DISC1 variation is responsible for the reduced synapse growth, Ming says.

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Stem Cells Reveal How Illness-Linked Genetic Variation Affects Neurons

ALAMEDA, Calif.--(BUSINESS WIRE)--BioTime, Inc. (NYSE MKT: BTX) today reported financial results for the first quarter ended June 30, 2014 and highlighted recent corporate accomplishments.

We are pleased with our success to date in building toward our goal of developing both near-term commercial applications of our technologies and maintaining our focus on the power of pluripotent stem cells to create innovative human therapeutics, said Dr. Michael D. West, BioTimes Chief Executive Officer. Near-term product development underway includes our subsidiary OncoCyte Corporations three cancer diagnostic products undergoing clinical studies, mobile health product development in our subsidiary LifeMap Solutions, Inc., our Renevia pivotal clinical trial in Europe, steps to prepare for the marketing of our recently FDA-cleared wound healing product Premvia, and growing research product sales by our ESI BIO division.

BioTimes longer-term major therapeutic product opportunities are based on the broad range of cell-based regenerative therapies planned for development from its pluripotent stem cell technology platform. This platform is protected by over 600 patents and patent applications worldwide within the BioTime family of companies. Our subsidiary Asterias Biotherapeutics, Inc. has submitted an amended IND to the FDA for a Phase 1/2a clinical trial of AST-OPC1 for the treatment of cervical spinal cord injury and is currently awaiting clearance from the FDA for that trial. Asterias is also currently undertaking process development of AST-VAC2, a cancer immunotherapy targeting the important antigen called telomerase, for a potential clinical trial in lung cancer. This progress, along with the appointment of Pedro Lichtinger as Asterias CEO and the award of a $14 million grant from the California Institute for Regenerative Medicine, should fuel the development of these first-in-class therapeutic products. Recently, Asterias shares began to trade publicly under the symbol ASTYV, the first of our subsidiaries to have its shares trade publicly. Lastly, we expect that BioTimes subsidiary Cell Cure Neurosciences Ltd. will soon file its IND to begin a clinical trial of OpRegen for the treatment of age-related macular degeneration. Additional important cell-based product development is underway in our disease-focused subsidiaries OrthoCyte Corporation and ReCyte Therapeutics.

As we saw in the first quarter of this year, our expenses have risen compared to recent quarters, but our progress during the second quarter in streamlining our workforce through shared core resources among our subsidiaries should reduce our cash burn rate in the third quarter. We would like to thank those who share our goal of better health in the coming era of regenerative medicine. Their continued support and the diligent efforts of our collaborators at leading academic medical institutions is critical in advancing our products from the lab bench to the clinic, where they are desperately needed.

Second Quarter and Recent Highlighted Corporate Accomplishments

Financial Results

Revenue

For the six months ended June 30, 2014, on a consolidated basis, total revenue was $2.2 million, up $0.3 million or 19% from $1.8 million for the same period one year ago. The increase in revenue is primarily attributable to a $0.4 million increase in grant income primarily from a grant awarded to BioTimes subsidiary Cell Cure Neurosciences Ltd. (Cell Cure Neurosciences) from Israels Office of the Chief Scientist, offset in part by the decline in license fees of $0.1M primarily due to full recognition of the unamortized balance of the Summit license fees received in advance during the fourth quarter of 2013 as a result of the termination of our license agreements with Summit in 2013.

Expenses

Operating expenses for the six months ended June 30, 2014 were $26.0 million, compared to expenses of $18.0 million for the same period of 2013. The increase in operating expenses is primarily attributable to an increase in staffing, and the expansion of research and development efforts, including additional expenses in the Renevia clinical safety trial program, the development of OpRegen by BioTimes subsidiary Cell Cure Neurosciences for the treatment of dry age related macular degeneration, and the increased staffing and operations of Asterias in connection with the Geron stem cell asset acquisition and by LifeMap Solutions. In addition, during the first six months in 2014, operating expenses included $1.5 million of amortization expense of intangible assets recorded in connection with the Geron stem cell asset acquisition in October 2013.

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BioTime Announces Second Quarter 2014 Results and Recent Developments

In a boon to stem cell research and regenerative medicine, scientists at Boston Children's Hospital, the Wyss Institute for Biologically Inspired Engineering at Harvard University and Boston University have created a computer algorithm called CellNet as a "roadmap" for cell and tissue engineering, to ensure that cells engineered in the lab have the same favorable properties as cells in our own bodies. CellNet and its application to stem cell engineering are described in two back-to-back papers in the August 14 issue of the journal Cell.

Scientists around the world are engaged in culturing pluripotent stem cells (capable of forming all the body's tissues) and transforming them into specialized cell types for use in research and regenerative medicine. Available as an Internet resource for any scientist to use, CellNet provides a much needed "quality assurance" measure for this work.

The two papers also clarify uncertainty around which methods are best for stem cell engineering, and should advance the use of cells derived from patient tissues to model disease, test potential drugs and use as treatments. For example, using CellNet, one of the studies unexpectedly found that skin cells can be converted into intestinal cells that were able to reverse colitis in a mouse model.

"To date, there has been no systematic means of assessing the fidelity of cellular engineering -- to determine how closely cells made in a petri dish approximate natural tissues in the body," says George Q. Daley, MD, PhD, Director of the Stem Cell Transplantation Program at Boston Children's and senior investigator on both studies. "CellNet was developed to assess the quality of engineered cells and to identify ways to improve their performance."

Gene Signatures

CellNet applies network biology to discover the complex network of genes that are turned on or off in an engineered cell, known as the cell's Gene Regulatory Network or GRN. It then compares that network to the cell's real-life counterpart in the body, as determined from public genome databases. Through this comparison, researchers can rigorously and reliably assess:

"CellNet will also be a powerful tool to advance synthetic biology -- to engineer cells for specific medical applications," says James Collins, PhD, Core Faculty member at the Wyss Institute and the William F. Warren Distinguished Professor at Boston University, co-senior investigator on one of the studies.

Putting CellNet to the Test

The researchers -- including co-first authors Patrick Cahan, PhD and Samantha Morris, PhD, of Boston Children's, and Hu Li, PhD, of the Mayo Clinic, first used CellNet to assess the quality of eight kinds of cells created in 56 published studies.

In a second study, they applied CellNet's teachings to a recurring question in stem cell biology: Is it feasible to directly convert one specialized cell type to another, bypassing the laborious process of first creating an iPS cell? This study looked at two kinds of directly converted cells: liver cells made from skin cells, and macrophages made from B cells.

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Tissue development 'roadmap' created to guide stem cell medicine

Immunologist Nick Huntington. Photo: Getty Images/Paul Jeffers

The bodys natural killer cells, as their Hollywood-style name suggests, are key to the immune system. They are programmed to hunt out and destroy foreign and diseased cells. But they dont always identify their targets. When this happens, diseases such as cancer can set in.

But a team of researchers at the Walter and Eliza Hall Institute of Medical Research have worked out what the group of highly specialised killer cells need to function at their best. Its a protein called MCL-1.

Immunologist Nick Huntington said the protein was effectively a switch which could turn the killer cells on or off.

The discovery, outlined on Thursday in the journal Nature Communications, opens the way for new drug treatments to tame the spread of a range of diseases, including cancer.

It could also assist patients who undergo donor stem cell or bone marrow transplants - because by manipulating the killer cells switch, foreign bodies such as stem cells could go unchallenged by the bodys immune system.

"Its the only protein which does this in the cell, Dr Huntington said. It needs to be turned on for the cell to survive and when its turned off the cell will die.

While aware of the existence of the MCL-1 protein and its importance at a fundamental level, scientists were previously unaware of its role in natural killer cell function. With colleagues Priyanka Sathe and Rebecca Delconte, Dr Huntington established its role.

That knowledge will prove useful for the development of new drugs to treat cancers.

Potential benefits include reduced side effects from treatment, as the killer cells only target foreign, diseased or cancerous cells, unlike chemotherapy which targets healthy cells as well.

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Scientists discover killer cells' ''on switch''

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Newswise Hematopoietic stem cells (HSCs) give rise to all other blood cell types, but their development and how their fate is determined has long remained a mystery. In a paper published online this week in Nature, researchers at the University of California, San Diego School of Medicine elaborate upon a crucial signaling pathway and the role of key proteins, which may help clear the way to generate HSCs from human pluripotent precursors, similar to advances with other kinds of tissue stem cells.

Principal investigator David Traver, PhD, professor in the Department of Cellular and Molecular Medicine, and colleagues focused on the Notch signaling pathway, a system found in all animals and known to be critical to the generation of HSCs in vertebrates. Notch signaling between emitting and receiving cells is key to establishing HSC fate during development, said Traver. What has not been known is where, when and how Notch signal transduction is mediated.

Traver and colleagues discovered that the Notch signal is transduced into HSC precursor cells from signal emitting cells in the somite embryologic tissues that eventually contribute to development of major body structures, such as skeleton, muscle and connective tissues much earlier in the process than previously anticipated.

More specifically, they found that JAM proteins, best known for helping maintain tight junctions between endothelial cells to prevent vascular leakage, were key mediators of Notch signaling. When the researchers caused loss of function in JAM proteins in a zebrafish model, Notch signaling and HSCs were also lost. When they enforced Notch signaling through other means, HSC development was rescued.

To date, it has not been possible to generate HSCs de novo from human pluripotent precursors, like induced pluripotent stem cells, said Traver. This has been due in part to a lack of understanding of the complete set of factors that the embryo uses to make HSCs in vivo. It has also likely been due to not knowing in what order each required factor is needed.

Our studies demonstrate that Notch signaling is required much earlier than previously thought. In fact, it may be one of the earliest determinants of HSC fate. This finding strongly suggests that in vitro approaches to instruct HSC fate from induced pluripotent stem cells must focus on the Notch pathway at early time-points in the process. Our findings have also shown that JAM proteins serve as a sort of co-receptor for Notch signaling in that they are required to maintain close contact between signal-emitting and signal-receiving cells to permit strong activation of Notch in the precursors of HSCs.

The findings may have far-reaching implications for eventual development of hematopoietic stem cell-based therapies for diseases like leukemia and congenital blood disorders. Currently, it is not possible to create HSCs from differentiation of embryonic stem cells or induced pluripotent stem cells pluripotent cells artificially derived from non-pluripotent cells, such as skin cells that are being used in other therapeutic research efforts.

Co-authors include Isao Kobayashi, Jingjing Kobayashi-Sun, Albert D. Kim and Claire Pouget, UC San Diego Department of Cellular and Molecular Medicine; Naonobu Fujita, UC San Diego Section of Cell and Developmental Biology; and Toshio Suda, Keio University, Japan.

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New Blood: Tracing the Beginnings of Hematopoietic Stem Cells

Aug 13, 2014 Growing stem cells in conditions free of animal material makes them safe for use in humans. Credit: Eraxion/iStock/Thinkstock

Human stem cells produced through genetic reprogramming are beset by safety concerns because current techniques alter the DNA of the stem cells and use material from animals to grow them. Now, A*STAR researchers have developed an efficient approach that produces safe, patient-specific human stem cells.

Human induced pluripotent stem cells have the potential to treat a number of diseases without the ethical issues associated with embryonic stem cells. Pluripotent stem cells can be produced from adult cells by introducing genes that reprogram them. Typically, the stem cells are grown on a layer of mouse cells in solutions (known as media) that contain animal proteinsand therefore, potentially may also carry disease. For such stem cells to be safe for use in humans, they need to be grown in 'xeno-free' conditions, which are devoid of material from other animals.

Andrew Wan and Hong Fang Lu at the A*STAR Institute of Bioengineering and Nanotechnology in Singapore and colleagues set out to develop a new xeno-free system. The researchers carried out the genetic reprogramming of cells on an artificially produced protein substrate rather than mouse cells. They also used media that contained no animal components. The result was more efficient reprogramming than seen with conventional approaches.

"A xeno-free system will eliminate the risk of disease transmission from other species, which is important for regulatory approval," explains Wan. "Yet there have been few studies on cell reprogramming under totally xeno-free conditions."

The researchers went one step further by addressing the problem of cells acquiring alterations to their DNA during reprogramming.

"Incorporation of transgenes into the genome of the cell poses another safety issue, risking unwanted genetic alterations," explains Lu. "In our work, the transgenes were introduced to initiate the reprogramming, but after this they were removed from the cell, leading to transgene-free stem cells."

The researchers demonstrated that after genetic reprogramming and the removal of the added genes, the stem cells could still develop into different cells types. They were even able to induce them to form dopaminergic neurons, the type that degenerates in Parkinson's disease. The conditions in which the stem cells were grown mean that they are suitable for clinical use and can be derived from a patient's own cells, ensuring complete compatibility.

"Regulatory approval for clinical application of stem cells largely depends on the conditions in which the stem cells are derived," says Wan. "We present a workable protocol for the reprogramming of fibroblasts to stem cells that minimizes any potential safety risks."

Explore further: Discovery may make it easier to develop life-saving stem cells

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Animal-free reprogramming of adult cells improves safety

TO SOME people, the term stem cell may seem kind of taboo. I personally would not want something from animals injected into my system. But Im okay with non-invasive treatments, so I was interested to try out a plant-based stem cell facial.

After cleansing and toning, cotton pads moistened with a clear solution were laid on my eyelids to protect them from a three-minute steaming session. This was followed by a special tool called a scrubber that kind of looks like a computer mouse, but helps to remove dead skin cells and unblock pores without using the rather painful pricking tool.

Next, a rejuvenating gel was applied, followed by the plant-derived stem cell formula. A unique cooling machine was used to massage it into the skin for 10 minutes. Using this machine for cold electrophoresis helps the skin absorb serums and vitamins, without having to use injections. This was great for someone like me, who is wary of invasive treatments. The cooling machine feels like having an ice-cold metal ball massaged on the face; very invigorating, indeed.

Just when I thought my skin already got a lot of pampering, the stem cell was followed by a face mask full of natural vitamins. While it penetrated into my skin, I was given an arm and foot massage, which was nice for further relaxation.

With my combination skin, I looked pretty greasy right afterwards. When I woke up the next day, I didnt see a visible difference in my skin, but it was very smooth and supple to the touch. You may not see instant results with a treatment like this, but its a good treatment to maintain radiance, softness and hydration from beneath the surface of the skin.

This type of facial is not recommended for those with oily or acne-prone skin because the added oiliness may exacerbate problems, but it is ideal for those with dry or mature skin, as it is deeply nourishing and moisturizing. After the first treatment or over time, depending on the condition of your skin, stem cell diminishes fine lines, prevents wrinkles, and promotes cell renewal (a process that slows with age) to give that glowing look that signifies healthy, youthful skin.

I tried out the stem cell facial at Lohas skin and slimming center on Paseo Saturnino, Banilad. Its a more upscale experience here with your own room, as opposed to being in one large room with dividers, in case privacy is an issue for you. All of their machines and products are brought in from Korea and their staff, like my therapist Jennylyn, are highly knowledgeable and know just how much pressure to apply during the treatment. The service, facilities and products used add up to a luxurious treatment session that makes one feel very pampered.

Published in the Sun.Star Cebu newspaper on August 15, 2014.

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Trying out a stem cell facial

A cure for a range of blood disorders and immune diseases is in sight, according to scientists who have unravelled the mystery of stem cell generation.

The Australian study, led by researchers at the Australian Regenerative Medicine Institute (ARMI) at Monash University and the Garvan Institute of Medical Research, is published today in Nature. It identifies for the first time mechanisms in the body that trigger hematopoietic stem cell (HSC) production.

Found in the bone marrow and in umbilical cord blood, HSCs are critically important because they can replenish the body's supply of blood cells. Leukemia patients have been successfully treated using HSC transplants, but medical experts believe blood stem cells have the potential to be used more widely.

Lead researcher Professor Peter Currie, from ARMI explained that understanding how HSCs self-renew to replenish blood cells is a "Holy Grail" of stem cell biology.

"HSCs are one of the best therapeutic tools at our disposal because they can make any blood cell in the body. Potentially we could use these cells in many more ways than current transplantation strategies to treat serious blood disorders and diseases, but only if we can figure out how they are generated in the first place. Our study brings this possibility a step closer," he said.

A key stumbling block to using HSCs more widely has been an inability to produce them in the laboratory setting. The reason for this, suggested from previous research, is that a molecular 'switch' may also be necessary for HSC formation, though the mechanism responsible has remained a mystery, until now.

In this latest study, ARMI researchers observed cells in the developing zebra fish -- a tropical freshwater fish known for its regenerative abilities and optically clear embryos -- to gather new information on the signalling process responsible for HSC generation.

Using high-resolution microscopy researchers made a film of how these stem cells form inside the embryo, which captured the process of their formation in dramatic detail.

Professor Currie said when playing back these films they noticed that HSCs require a "buddy" cell type to help them form. These "buddies," known as endotome cells, have stem cell inducing properties,

"Endotome cells act like a comfy sofa for pre HSCs to snuggle into, helping them progress to become fully fledged stem cells. Not only did we identify some of the cells and signals required for HSC formation, we also pinpointed the genes required for endotome formation in the first place," Professor Currie said.

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Cell discovery brings blood disorder cure closer

Australian scientists studying zebrafish have stumbled upon what they say is one of the most significant discoveries in stem cell research.

In research published on Thursday in the journal Nature, the Monash University scientists revealed that they uncovered how one of the most important stem cells in blood and bone marrow, the haematopoietic stem cell (HSC), is formed.

Professor Peter Currie, from Monash University's Australian Regenerative Medicine Institute, said the discovery brought researchers closer to growing HSCs in a lab.

"HSCs are the basis of bone marrow transplantations as a therapy, so when a leukaemia patient receives bone marrow, it's really these HSCs that do the heavy lifting," Professor Currie said.

"So when clinicians do bone marrow transplants, they need to find a matching donor recipients and we know that's a hit-or-miss procedure.

"So for many years people have been trying to make HSCs in the dish, and they've had very little success in doing this."

Professor Currie, who led the study, said the discovery brought scientists much closer to achieving that aim.

"It's the discovery of a completely new cell type that basically is required to give instructions to the HSC to make it become what it needs to become," he said.

"It means we now understand how HSC form in the body better, we can use that information to try to grow these cells in the dish and we hope that will lead to better treatment for people with leukaemia and blood disorders."

Professor Currie said he specialises in muscle stem cell biology and accidentally came across the discovery while studying muscle stem cells in zebrafish.

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Stem cell discovery: Australian scientists make significant find while studying zebrafish

The UC San Diego Health System put out a call Monday for eight spinal cord injury patients to take part in a five-year test of the safety of a new treatment involving neural stem cells.

The researchers are looking for people who suffered an injury to the middle or lower levels of the spine's thoracic vertebrae between one and two years ago. According to UCSD, the injury must be between the seventh and 12th thoracic vertebrae.

"The goal of this study is to evaluate the safety of transplanting neural stem cells into the spine for what one day could be a treatment for spinal cord injuries," said Dr. Joseph Ciacci, the study's principal investigator and a neurosurgeon at UC San Diego Health System. "The study's immediate goal, however, is to determine whether injecting these neural stem cells into the spine of patients with spinal cord injury is safe."

The doctors also want to know how long the transplanted stem cells will last, and whether drugs designed to prevent rejection by the immune system are effective, according to UCSD Health.

The researchers will also look for possible changes in motor and sensory function, bowel and bladder function, and pain levels.

The stem cells were tested in laboratory rats by Ciacci and Dr. Martin Marsala, of the UC San Diego School of Medicine. They detected signs of improved motor function with minimal side effects. The cells have also been tested for safety in human patients with amyotrophic lateral sclerosis commonly known as ALS or Lou Gehrig's Disease.

UCSD cautioned prospective test subjects that since human tests are just beginning, unforeseen risks, complications or unpredictable outcomes are possible.

The clinical trial at UC San Diego Health System is funded by Neuralstem Inc. and was launched and supported by the UC San Diego Sanford Stem Cell Clinical Center. The center was recently created to "advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy," according to UCSD.

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UCSD Looking For Spinal Cord Injury Patients To Test Stem Cell Treatment

HOT SPRINGS | A new proposal to not only save but also enhance the Veterans Affairs hospital in Hot Springs surfaced Monday, and would add not only a medical college but also a medical research component involving the use of stem cells to the facility.

The idea, put forward by an Iowa-based, non-profit corporation, would also be built around treating patients with regenerative therapy, which helps skin grow back.

Bob Krause, president of Veterans National Recover Center, was joined by surgeon Don Swift in Hot Springs to presented the proposal at a press conference Monday morning. Their multi-pronged plan has been submitted for consideration to the VA Black Hills Health Care Systems Environmental Impact Statement.

Our proposal has three main areas, Krause told the small audience that attended the press conference. First, the creation of Battle Mountain College, for the training of doctors in the discipline of osteopathic medicine. Krause noted that by having the additional training, a major first hurdle in the BHHCS proposal to close the Hot Springsan inability to draw doctors to the area would be addressed.

We would also build the Battle Mountain Research Institute, for further research into the regenerative therapies, along with the Battle Mountain Clinic to treat those veterans and others who require this cutting-edge treatment, Krause said.

He added that the proposal stipulated that it is to be considered in its entirety and that if the VA medical center should close, everything is off the table. This proposal is not mutually exclusive of the one presented by Save the VA, he said of the Hot Springs-area group that is fighting to save the hospital from closure by the federal government.

Krause and Swift said that the technology, which was created in Switzerland by the military and is awaiting FDA approval in the United States, utilizes regenerative or restorative cells created from fetal stem cells to jump-start a patients ability to regenerate skin tissue. After the patients own skin begins to grow, the regenerative cells die, Krause said.

He said that submitting the new proposal through the EIS process was important, since the research would need to be conducted on federal property because South Dakota law does not allow stem cell research at this time.

Swift noted that an important part to the regenerative therapy process was access to mineral water to help hydrate the tissue and fight infection. Such water can be found in Hot Springs.

In response to a question, Krause said that he understands that there is a question involving fetal stem cell research. But what is the greater good? he asked. Do we overlook a veteran who has experienced having all of his skin burned away by an [explosion], instead of developing that single cell that could help? Are you going to walk away from that cell?

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New idea for VA would bring an educational focus

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Newswise LOS ANGELES (Aug. 12, 2014) Regenerative medicine experts at the Cedars-Sinai Heart Institute have opened a new clinic to evaluate heart and vascular disease patients for participation in stem cell medical studies.

Led by Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute, and Timothy Henry, MD, director of the Heart Institutes Cardiology Division, the doctors and researchers at the Cedars-Sinai Heart Institute Regenerative Medicine Clinic use a scientific approach to assess the possible benefits of stem cells to repair damaged or diseased cardiovascular tissues. The clinic is believed to be the first at a major U.S. academic medical center dedicated to matching patients with appropriate stem cell clinical trials, whether those research interventions are available at the medical center or at other institutions.

The Heart Institute Regenerative Medicine Clinic offers consultative services for patients with heart and vascular disease who may qualify for investigative stem cell therapy. The goal is to provide research options to patients who remain symptomatic on their current management regimen, or for patients with stable heart disease who are concerned about disease progression.

Over the past decade, medical experts have predicted that in the future, stem cell therapies would transform heart disease treatment and save lives, said Shlomo Melmed, MD, dean of the Cedars-Sinai faculty and the Helene A. and Philip E. Hixon Distinguished Chair in Investigative Medicine. At Cedars-Sinai, we have a track record of successfully directing cardiac stem cell studies as well as transferring innovations from the laboratory to the patient bedside.

In 2009, Marbn and his team completed the worlds first procedure in which a patients own heart tissue was used to grow specialized heart stem cells. The specialized cells were then injected back into the patients heart in an effort to repair and re-grow healthy muscle in a heart that had been injured by a heart attack. Results, published in The Lancet in 2012, showed that one year after receiving the stem cell treatment, heart attack patients demonstrated a significant reduction in the size of the scar left on the heart muscle after a heart attack.

Henry has served as principal investigator of multiple large, multicenter trials in acute coronary syndromes, myocardial infarction and angiogenesis, including several ongoing cardiovascular stem cell trials. He also is principal investigator for one of seven NIH Clinical Cardiovascular Stem Cell Centers.

Our goal is to help make stem cells a regular treatment option for heart disease, Henry said. Right now, many patients with advanced heart disease have limited treatment options. Stem cells offer not only hope but a real chance of a game-changing treatment.

As part of each patients assessment in the Heart Regenerative Medicine Clinic, physicians will evaluate patients interested in participating in stem cell clinical trials at Cedars-Sinai and, for patients willing to travel at other medical institutions across the nation. For patients willing to travel to participate in research, Cedars-Sinai physicians will work closely with investigators at other centers to expedite referrals and seamlessly transfer all relevant medical records.

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Cedars-Sinai Heart Institute Opens First-of-its-Kind Research Stem Cell Clinic for Cardiac Patients

SAN DIEGO The UC San Diego Health System put out a call Monday for eight spinal cord injury patients to take part in a five-year test of the safety of a new treatment involving neural stem cells.

The researchers are looking for people who suffered an injury to the middle or lower levels of the spines thoracic vertebrae between one and two years ago. According to UCSD, the injury must be between the seventh and 12th thoracic vertebrae.

The goal of this study is to evaluate the safety of transplanting neural stem cells into the spine for what one day could be a treatment for spinal cord injuries, said Dr. Joseph Ciacci, the studys principal investigator and a neurosurgeon at UC San Diego Health System. The studys immediate goal, however, is to determine whetherinjecting these neural stem cells into the spine of patients with spinal cord injury is safe.

The doctors also want to know how long the transplanted stem cells will last, and whether drugs designed to prevent rejection by the immune system are effective, according to UCSD Health.

The researchers will also look for possible changes in motor and sensory function, bowel and bladder function, and pain levels.

The stem cells were tested in laboratory rats by Ciacci and Dr. Martin Marsala, of the UC San Diego School of Medicine. They detected signs of improved motor function with minimal side effects. The cells have also been tested for safety in human patients with amyotrophic lateral sclerosis commonly known as ALS or Lou Gehrigs Disease.

UCSD cautioned prospective test subjects that since human tests are just beginning, unforeseen risks, complications or unpredictable outcomes are possible.

The clinical trial at UC San Diego Health System is funded by Neuralstem Inc. and was launched and supported by the UC San Diego Sanford Stem Cell Clinical Center. The center was recently created to advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy, according to UCSD.

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Spinal injury patients needed for stem cell treatment study

SAN DIEGO (CNS) - The UC San Diego Health System put out a call Monday for eight spinal cord injury patients to take part in a five-year test of the safety of a new treatment involving neural stem cells.

The researchers are looking for people who suffered an injury to the middle or lower levels of the spine's thoracic vertebrae between one and two years ago. According to UCSD, the injury must be between the seventh and 12th thoracic vertebrae.

"The goal of this study is to evaluate the safety of transplanting neural stem cells into the spine for what one day could be a treatment for spinal cord injuries," said Dr. Joseph Ciacci, the study's principal investigator and a neurosurgeon at UC San Diego Health System. "The study's immediate goal, however, is to determine whether injecting these neural stem cells into the spine of patients with spinal cord injury is safe."

The doctors also want to know how long the transplanted stem cells will last, and whether drugs designed to prevent rejection by the immune system are effective, according to UCSD Health.

The researchers will also look for possible changes in motor and sensory function, bowel and bladder function, and pain levels.

The stem cells were tested in laboratory rats by Ciacci and Dr. Martin Marsala, of the UC San Diego School of Medicine. They detected signs of improved motor function with minimal side effects. The cells have also been tested for safety in human patients with amyotrophic lateral sclerosis - commonly known as ALS or Lou Gehrig's Disease.

UCSD cautioned prospective test subjects that since human tests are just beginning, unforeseen risks, complications or unpredictable outcomes are possible.

The clinical trial at UC San Diego Health System is funded by Neuralstem Inc. and was launched and supported by the UC San Diego Sanford Stem Cell Clinical Center. The center was recently created to "advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy," according to UCSD.

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UCSD test calls for spinal cord injury patients

Infusing stem cells into the brain may help boost recovery after a stroke, according to a pilot study by Imperial College London.

Scientists believe the cells encourage new blood vessels to grow in damaged areas of the brain.

They found most patients were able to walk and look after themselves independently by the end of the trial, despite having suffered severe strokes.

Larger studies are needed to evaluate whether this could be used more widely.

In this early trial - designed primarily to look at the safety of this approach - researchers harvested stem cells from the bone marrow of five people who had recently had a stroke.

'Independent living'

They isolated particular types of stem cells - known as CD34+. These have the ability to stimulate the growth of new blood vessels.

They were infused directly into damaged sections of the brain, via the major artery that supplies this area.

Scientists monitored the patients for six months, charting their ability to carry out everyday activities independently.

Four of the five patients had suffered particularly severe strokes - resulting in the loss of speech and marked paralysis down one side of the body.

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'Stem Cells Show Promise In Stroke Recovery'

17 hours ago The red swamp crayfish (Procambarus clarkii) is native to the southeastern United States. This species is a popular model organism for studies of the nervous system, and has been used to study fundamental mechanisms involved in the production of new neurons in the adult brain. Credit: Jeanne Benton

Researchers have strived for years to determine how neurons are produced and integrated into the brain throughout adult life. In an intriguing twist, scientists reporting in the August 11 issue of the Cell Press journal Developmental Cell provide evidence that adult-born neurons are derived from a special type of circulating blood cell produced by the immune system. The findingswhich were made in crayfishsuggest that the immune system may contribute to the development of the unknown role of certain brain diseases in the development of brain and other tissues.

In many adult organisms, including humans, neurons in some parts of the brain are continually replenished. While this process is critical for ongoing health, dysfunctions in the production of new neurons may also contribute to several neurological diseases, including clinical depression and some neurodegenerative disorders. Dr. Barbara Beltz of Wellesley College and her colleagues studied crayfish to understand how new neurons are made in adult organisms. When they marked the cells of one crayfish and used this animal as a blood donor for transfusions into another crayfish, the researchers found that the donor blood cells could generate neurons in the recipient.

"These blood cellscalled hemocyteshave functions similar to certain white blood cells in mammals and are produced by the immune system in a blood-forming organ that is functionally analogous to bone marrow," explains Dr. Beltz. "When these cells are released into the circulation, they are attracted to a specialized region in the brain where stem cells divide, and their descendants develop into functional neurons."

The current work demonstrates that the immune system can produce cells with stem cell properties that can give rise to different types of cells, including both hemocytes and nerve cells. "Our findings in crayfish indicate that the immune system is intimately tied to mechanisms of adult neurogenesis, suggesting a much closer relationship between the immune system and nervous system than has been previously appreciated," says co-author Dr. Irene Sderhll, of Uppsala University in Sweden. The flexibility of these immune cells in producing neurons in adult animals raises the intriguing possibility of the presence of similar types of flexibility in other animals. If further studies demonstrated a similar relationship between the immune system and brain in mammals, the findings would stimulate a new area of research into immune therapies to target neurological diseases.

Explore further: New discovery on early immune system development

More information: Developmental Cell, Benton et al.: "Cells from the immune system generate adult-born neurons in crayfish." http://www.cell.com/developmental-cel 1534-5807(14)00405-5

Journal reference: Developmental Cell

Provided by Cell Press

Researchers at Lund University have shed light on how and when the immune system is formed, raising hope of better understanding various diseases in children, such as leukaemia.

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Blood cells are new, unexpected source of neurons in crayfish

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Newswise Researchers at the University of California, San Diego School of Medicine have launched a clinical trial to investigate the safety of neural stem cell transplantation in patients with chronic spinal cord injuries. This Phase I clinical trial is recruiting eight patients for the 5-year study.

The goal of this study is to evaluate the safety of transplanting neural stem cells into the spine for what one day could be a treatment for spinal cord injuries, said Joseph Ciacci, MD, principal investigator and neurosurgeon at UC San Diego Health System. The studys immediate goal, however, is to determine whether injecting these neural stem cells into the spine of patients with spinal cord injury is safe.

Related goals of the clinical trial include evaluating the stem cell grafts survival and the effectiveness of immunosuppression drugs to prevent rejection. The researchers will also look for possible therapeutic benefits such as changes in motor and sensory function, bowel and bladder function, and pain levels.

Patients who are accepted for the study will have spinal cord injury to the T7-T12 level of the spines vertebrae and will have incurred their injury between one and two years ago.

All participants will receive the stem cell injection. The scientists will use a line of human stem cells approved by the U.S. FDA for human trials in patients with chronic traumatic spinal injuries. These cells were previously tested for safety in patients with amyotrophic lateral sclerosis (ALS).

Since stem cell transplantation for spinal cord injury is just beginning clinical tests, unforeseen risks, complications or unpredictable outcomes are possible. Careful clinical testing is essential to ensure that this type of therapy is developed responsibly with appropriate management of the risks that all medical therapies may present.

Pre-clinical studies of these cells by Ciacci and Martin Marsala, MD, at the UC San Diego School of Medicine, showed that these grafted neural stem cells improved motor function in spinal cord injured rats with minimal side effects indicating that human clinical trials are now warranted.

This clinical trial at UC San Diego Health System is funded by Neuralstem, Inc. and was launched and supported by the UC San Diego Sanford Stem Cell Clinical Center. The Center was recently created to advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy.

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Clinical Trial Evaluates Safety of Stem Cell Transplantation in Spine