StemCell Therapy

ScienceDaily (Oct. 11, 2012) The generation of functional thyroid tissue from stem cells could allow the treatment of patients, which suffer from thyroid hormone deficiency due to defective function, or abnormal development of the thyroid gland. The team of Sabine Costagliola at the IRIBHM (Universit Libre de Bruxelles) recently developed a protocol that allowed for the first time the efficient generation of functional thyroid tissue from stem cells in mice and published the results of their studies in the scientific journal Nature.

Thyroid hormones are a class of iodide-containing molecules that play a critical role in the regulation of various body function including growth, metabolism and heart function and that are crucial for normal brain development. The thyroid gland, an endocrine organ that has been specialized in trapping iodide, is the only organ where these hormones are produced. It is, however, of note that one out of 3000 human newborns is born with congenital hypothyroidism, a condition characterized by insufficient production of thyroid hormones. In the absence of a medical treatment with thyroid hormones -- initiated during the first days after birth -- the child will be affected by an irreversible mental retardation. Moreover, a life-long hormonal treatment is necessary in order to maintain proper regulation of growth and general metabolism.

By employing a protocol in which two important genes can be transiently induced in undifferentiated stem cells, the researchers at IRIBHM were able to efficiently push the differentiation of stem cells into thyrocytes, the primary cell type responsible for thyroid hormone production in the thyroid gland.

A first exciting finding of these studies was the development of functional thyroid tissue already within the culture dishes. As a next step, the team of Sabine Costagliola transplanted the stem-cell-derived thyrocytes into mice lacking a functional thyroid gland. Four weeks after transplantation, the researchers observed that transplanted mice had re-established normal levels of thyroid hormones in their blood and were rescued from the symptoms associated with thyroid hormone deficiency. These findings have several important implications. First, the cell system employed by the IRIBHM group provides a vital tool to better characterize the molecular processes associated with embryonic thyroid development. Second, the results of the transplantation studies open new avenues for the treatment of thyroid hormone deficiency but also for the replacement of thyroid tissue in patients suffering from thyroid cancer.

The researchers are currently developing a similar protocol based on human stem cells and explore ways to generate functional human thyroid tissue by reprogramming pluripotent stem cells (iPS) derived from skin cells.

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The above story is reprinted from materials provided by Universit Libre de Bruxelles, via AlphaGalileo.

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Generation of functional thyroid tissue from stem cells

ScienceDaily (Oct. 11, 2012) In recent years investigators have discovered that breast tumors are influenced by more than just the cancer cells within them. A variety of noncancerous cells, which in many cases constitute the majority of the tumor mass, form what is known as the "tumor microenvironment." This sea of noncancerous cells and the products they deposit appear to play key roles in tumor pathogenesis.

Among the key accomplices in the tumor microenvironment are mesenchymal stem cells (MSCs), a group of adult progenitor cells which have been shown to help breast cancers maneuver and spread to other parts of the body.

Now, new research sheds further light on how this is happening. Led by investigators at Beth Israel Deaconess Medical Center (BIDMC), the findings demonstrate that the lysyl oxidase (LOX) gene is spurred to production in cancer cells as a result of their contact with MSCs, and once produced, can help ensure the spread of otherwise weakly metastatic cancer cells from primary tumors to the lung and bones. Described on-line in the Proceedings of the National Academy of Sciences (PNAS), this discovery not only provides key insights into the basic biology of tumor formation, but also offers a potential new direction in the pursuit of therapies for the treatment of bone metastasis.

"We don't have a lot of therapies that can target breast cancer once it has metastasized, particularly once cancer cells have lodged in the bone," says senior author Antoine Karnoub, PhD, an investigator in the Department of Pathology at BIDMC and Assistant Professor of Pathology at Harvard Medical School. "When breast cancer cells reach the skeleton, one way in which they cause damage is by breaking down bone tissue, which results in the bone's rich matrix releasing numerous factors. These factors, in turn, feed the cancer cells, setting in motion a vicious cycle that leaves patients susceptible to fractures, pain, and further metastasis."

MSCs are non-hematopoietic progenitor cells predominantly produced in the bone marrow that generate bone, cartilage, fat, and fibrous connective tissue. They additionally support immune cell development and are recruited to inflammatory sites throughout the body to help shut down immune responses and regenerate damaged tissues, as might occur during wound healing. Several years ago, as a postdoctoral researcher at the Whitehead Institute of the Massachusetts Institute of Technology, Karnoub began exploring the idea that MSCs were migrating to tumors after mistaking the cancer sites for inflammatory lesions in need of healing.

"We discovered that once MSCs had reached the tumor sites, they were actually helping in cancer metastasis, causing primary cancer cells to spread to other sites in the body," he explains. In this new paper, Karnoub wanted to find out, in greater molecular detail, how breast cancer cells respond to the influences of MSCs in order to better understand how cancer cells cross-talk with recruited cells in the microenvironment.

His scientific team first embarked on a straightforward experiment. "We took two dishes of cells, cancer cells and MSCs, and mixed them together," explains Karnoub. After three days, they removed the cancer cells and studied them to see how they had changed.

"We found that the lysyl oxidase [LOX] gene was highly upregulated in the cancer cells," he says. "It turns out that when a cancer cell comes in contact with an MSC, it flips on this LOX gene, turning it up by a factor of about 100. So our next question was, 'What happens to the cancer cells when they encounter this boost of LOX that they themselves have produced?'"

The answer, as revealed in subsequent experiments, was that LOX was setting in motion a cell program called epithelial-to-mesenchymal transition (EMT). During EMT, cancer cells that usually clump together undergo a transformation into cells that exhibit decreased adhesion to their neighbors and go their own way. As a result, these cancerous cells are able to migrate, significantly enhancing their ability to metastasize.

"When we put these cells back into mice, they not only formed tumors that metastasized to the lung, but also to the bone," says Karnoub. "This makes you wonder whether the cancer cells in primary tumors have become so acclimated to interacting with bone-derived MSCs that they can now grow more easily in the bone once they leave the tumor."

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Clues to cancer metastasis: Discovery points to potential therapies for bone metastasis

The achievement is the latest success in the relatively new field of regenerative medicine

By Dan Jones and Nature magazine

WE CAN REBUILD HIM: Regenerative successes in mice are adding up. Image: Nature News

Showcasing more than fifty of the most provocative, original, and significant online essays from 2011, The Best Science Writing Online 2012 will change the way...

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From Nature magazine

A series of achievements have stoked excitement about the potential of regenerative medicine, which aims to tackle diseases by replacing or regenerating damaged cells, tissues and organs. A paper in Nature today reports another step towards this goal: the generation of working thyroid cells from stem cells.

Sabine Costagliola, a molecular embryologist at the Free University of Brussels, and her team study the development of the thyroid gland, which regulates how the body uses energy and affects sensitivity to other hormones. Their research shows that thyroid function can be re-established even after the gland has been destroyed at least in mice. If the same technique could be applied to humans, it would help the roughly 1 in 3,000 babies born with deficient thyroid activity, or hypothyroidism, which can result in stunted physical and mental development.

The thyroid is the latest in a growing list of body parts that can now be fixed in mice, with the potential to treat diseases from diabetes to Parkinsons (see 'We can rebuild him'). Progress has been very rapid over the past decade, says Charles ffrench-Constant, director of the MRC Centre for Regenerative Medicine at the University of Edinburgh, UK. In recent years weve seen a number of very important studies in which mouse stem cells have been converted to a desired cell type that has then been shown to be functional in vivo, and to confer benefits in mouse models of human diseases.

Key ingredient Costagliola and her colleagues first genetically engineered embryonic stem cells to express two proteins NKX2-1 and PAX8 that are expressed together only in the thyroid. When these cells were grown in Petri dishes in the presence of thyroid-stimulating hormone, they turned into thyroid cells.

Link:
Hormone-Producing Thyroid Grown from Embryonic Stem Cells

An oligodendrocytethe type of cell that manufactures myelin.

At first, the infants seem to be progressing normally. But it soon turns out they may have vision or hearing problems, and when the time comes to lift their heads, the milestone comes and goes. It often gets worse from there. Children with the rare PelizaeusMerzbacher disease, like others who lack the usual insulating sheaths on their neurons, have trouble controlling their muscles, and often develop serious neurological and motor problems early in life. There is no cure for the genetic disorder. Nor is there a standardized treatment.

PMD, as its called, and related diseases are some of the leading candidates for potential treatment with stem cells. The idea is that if stem cells that produce the missing insulator, the fatty substance called myelin, can be successfully implanted in the brains of patients, perhaps they will pitch in what the patients native cells cannot.

This week saw two incremental but encouraging advances toward such treatments, both published inScience Translational Medicine.In one study, mice without the ability to make myelin were implanted with human neural stem cells that, within weeks, developed into myelin-making cells 60-70% of the time and produced the substance in the brain. In the other study, four young boys with early onset PMD underwent an experimental treatment: the same type of stem cells were implanted into their brains, and, after 9 months of drugs to surpress the childrens immune systems so the cells could take hold, MRI exams, psychological tests, and motor tests are consistent with more myelin having formed.

Since there was no control group in the human study, the scientists have no way of knowing whether the new myelin formation is actually due to the implanted cells (for that, they would need a group of boys who received every step of the treatment except getting the cells, to compare). And there are, of course, only four subjects. But the fact that there have been no major side effectsespecially tumors, which not unheard-of after stem cell treatmentsis in and of itself heartening. It indicates that future studies using these cells can tentatively proceed. Image courtesy of Methoxyroxy / Wikimedia Commons

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Stem Cells Safely Implanted in Brains of Boys with Neurological Disorder | 80beats

Stem cell transfusions may someday replace the need for transplants in patients who suffer from liver failure caused by hepatitis B, according to a new study coming out of Beijing. . The results are published in the October issue of STEM CELLS Translational Medicine. Worldwide more than 500,000 people die each year from this condition.

Durham, NC (PRWEB) October 11, 2012

In China, hepatitis B virus (HBV) infection accounts for the highest proportion of liver failure cases. While liver transplantation is considered the standard treatment, it has several drawbacks including a limited number of donors, long waiting lists, high cost and multiple complications. Our study shows that mesenchymal stem cell (MSCs) transfusions might be a good, safe alternative, said Fu-Sheng Wang, Ph.D., M.D., the studys lead author and director of the Research Center for Biological Therapy (RCBT) in Beijing.

Wang along with RCBT colleague, Drs. Ming Shi and Zheng Zhang of the Research Center for Biological Therapy, The Institute of Translational Hepatology led the group of physician-scientists from the centers and Beijing 302 Hospital who conducted the study.

MSC transfusions had already been shown to improve liver function in patients with end-stage liver diseases. This time, the researchers wanted to gauge the safety and initial efficacy of treating acute-on-chronic liver failure (ACLF) with MSCs. The American Association for the Study of Liver Diseases and the European Association for the Study of the Liver define ACLF as an acute deterioration of pre-existing chronic liver disease usually related to a precipitating event and associated with increased mortality at three months due to multisystem organ failure. The short-term mortality rate for this condition is more than 50 percent.

MSCs have self-renewing abilities and the potential to differentiate into various types of cells. More importantly, they can interact with immune cells and cause the immune system to adjust to the desired level.

Of the 43 patients in this pilot study each of whom had liver failure resulting from chronic HBV infection 24 were treated with MSCs taken from donated umbilical cords and 19 were treated with saline as the control group. All received conventional therapy as well. The liver function, adverse events and survival rates were then evaluated during the 48-week or 72-week follow-up period.

Along with increased survival rates, the patients liver function improved and platelet count increased. No significant side effects were observed throughout the treatment and follow-up period.

While the results are preliminary and this pilot study includes a small number of patients, MSC transfusions appear to be safe and may serve as a novel therapeutic approach for HBV-associated ACLF patients, Dr. Shi said.

The study also highlights several key issues that will need to be considered in the design of future clinical studies, such as the optimal type of stem cells that will be infused, the minimum effective number of the cells and the best route of administration, Dr. Wang added.

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Early Results Show Promise for Stem Cells in Treating Chronic Liver Failure

By Emily Underwood, ScienceNOW

Four young boys with a rare, fatal brain condition have made it through a dangerous ordeal. Scientists have safely transplanted human neural stem cells into their brains. Twelve months after the surgeries, the boys have more myelin a fatty insulating protein that coats nerve fibers and speeds up electric signals between neurons and show improved brain function, a new study in Science Translational Medicine reports. The preliminary trial paves the way for future research into potential stem cell treatments for the disorder, which overlaps with more common diseases such as Parkinsons disease and multiple sclerosis.

This is very exciting, says Douglas Fields, a neuroscientist at the National Institutes of Health in Bethesda, Maryland, who was not involved in the work. From these early studies one sees the promise of cell transplant therapy in overcoming disease and relieving suffering.

Without myelin, electrical impulses traveling along nerve fibers in the brain cant travel from neuron to neuron says Nalin Gupta, lead author of the study and a neurosurgeon at the University of California, San Francisco (UCSF). Signals in the brain become scattered and disorganized, he says, comparing them to a pile of lumber. You wouldnt expect lumber to assemble itself into a house, he notes, yet neurons in a newborn babys brain perform a similar feat with the help of myelin-producing cells called oligodendrocytes. Most infants are born with very little myelin and develop it over time. In children with early-onset Pelizaeus-Merzbacher disease, he says, a genetic mutation prevents oligodendrocytes from producing myelin, causing electrical signals to die out before they reach their destinations. This results in serious developmental setbacks, such as the inability to talk, walk, or breathe independently, and ultimately causes premature death.

Although researchers have long dreamed of implanting human neural stem cells to generate healthy oligodendrocytes and replace myelin, it has taken years of research in animals to develop a stem cell that can do the job, says Stephen Huhn, vice president of Newark, California-based StemCells Inc., the biotechnology company that created the cells used in the study and that funded the research. However, he says, a separate study by researchers at Oregon Health & Science University, Portland, found that the StemCell Inc. cells specialized into oligodendrocytes 60 percent to 70 percent of the time in mice, producing myelin and improved survival rates in myelin-deficient animals. So the team was able to test the cells safety and efficacy in the boys.

Led by Gupta, the researchers drilled four small holes in each childs skull and then used a fine needle to insert millions of stem cells into white matter deep in their frontal lobes. The scientists administered a drug that suppressed the boys immune systems for 9 months to keep them from rejecting the cells and checked their progress with magnetic resonance imaging and a variety of psychological and motor tests. After a year, each of the boys showed brain changes consistent with increased myelination and no serious side effects such as tumors, says David Rowitch, one of the neuroscientists on the UCSF team. In addition, three of the four boys showed modest improvements in their development. For example, the 5-year-old boy the oldest child in the study had begun for the first time to feed himself and walk with minimal assistance.

Although these signs are encouraging, Gupta and Rowitch say, a cure for Pelizaeus-Merzbacher disease is not near. Animal studies strongly support the idea that the stem cells are producing myelin-making oligodendrocytes in the boys, but its possible that the myelination didnt result from the transplant but from a bout of normal growth. Rowitch adds that although such behavioral improvements are unusual for the disease, they could be a fluke. Huhn acknowledges that the study is small and has no control, but hes is still excited. We are for the first time seeing a biological effect of a neural stem cells transplantation into the brain [in humans]. The most important thing, he says, is that the transplants appear safe. This gives the researchers a green light to pursue larger, controlled studies, he says.

It isnt the flashiest thing, but demonstrating that its feasible to transplant these stem cells into childrens brains without negative consequences at least so far is extremely hopeful, says Timothy Kennedy, a neuroscientist at McGill University in Montreal, Canada.

Although hes concerned that myelination seen in mouse models might not scale up to a disease as severe as Pelizaeus-Merzbacher in humans, Ian Duncan, a neuroscientist at the University of Wisconsin, Madison, describes the study as setting a precedent for translating animal research in stem cells to humans. If you could improve quality of life by targeting key areas of the brain with these cells, he says, that would be a huge advance.

Link:
Stem Cells Show Early Promise for Rare Brain Disorder

The Nobel Prize for Physiology or Medicine was announced on Monday. The award this year went to Sir John B. Gurdon and Dr. Shinya Yamanaka. The two men were awarded the Nobel Prize jointly, for their individual work in cloning and stem cell research.

Monday's recognition marked the awarding of the first Nobel Prize for 2012. The rest of the Nobel Prize recipients will be announced throughout the next two weeks.

Here is some of the key information regarding Gurdon and Yamanaka's work and Monday's Nobel Prize announcement.

* Yamanaka and Gurdon did not work together or present shared research, even though they both concentrate their studies on a similar area of research.

* Gurdon is actually being honored for work he did back in 1962. According to a New York Times report, he was the first person to clone an animal, a frog, opening the door to further research into stem cells and cloning.

* Gurdon was able to produce live tadpoles from the adult cells of a frog, by removing the nucleus of a frog's egg and putting the adult cells in its place.

* This "reprogramming" by Gurdon laid the groundwork for Yamanaka's work four decades later. Yamanaka's work, which dates back only six years, to 2006, focused on the mechanisms behind Gurdon's results.

* According to the Los Angeles Times, Yamanaka was sharply criticized at first for his own work, in which he sought to discover how cells are able to reprogram themselves the way that Gurdon's work first suggested that they could.

* Ultimately, Yamanaka was able to isolate just four cells that were needed in order to be able to reprogram other cells back to an embryonic state, allowing them to be manipulated into developing into any particular kind of cell that was needed. These cells have now been dubbed "induced pluripotent stem cells," or iPS cells, according to reports by CNN and other media outlets.

* Scientists are reproducing Yamanaka's technique in their own labs to be able to replicate disease cells, like those of Alzheimer's or Parkinson's, in order to study them and even to test the effects of potential new treatments.

Continued here:
Nobel Prize for Physiology or Medicine Goes to Stem Cell Researchers

David Rowitch, MD, PhD, professor and chief of neonatology, in the NICU.

A Phase I clinical trial led by investigators from the University of California, San Francisco (UCSF) and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.

The study, published in Wednesday's issue of Science Translational Medicine, also demonstrated that the neural stem cells were safe in the patients brains one year post transplant.

The results of the investigation, designed to test safety and preliminary efficacy, are encouraging, said principal investigator David H. Rowitch, MD, PhD, a professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Childrens Hospital and a Howard Hughes Medical Institute Investigator.

Nalin Gupta, MD, PhD

For the first time, we have evidence that transplanted neural stem cells are able to produce new myelin in patients with a severe myelination disease, said Nalin Gupta, MD, PhD, associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital, and co-principal investigator of the PMD clinical trial.

We also saw modestgains in neurological function, and while these cant necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients,the findings represent an important first step that strongly supports further testing of this approach as a means to treat the fundamental pathology in the brain of these patients.

The study, one of the first neural stem cell trials ever conducted in the United States, is emblematic of UCSFs pioneering role in the stem cell field. In 1981, Gail Martin, PhD, professor of anatomy, co-discovered embryonic stem cells in mice. In 2001, Roger Pedersen, PhD, professor emeritus of obstetrics, gynecology and reproductive sciences, derived two of the first human embryonic stem cell lines. On Monday, Shinya Yamanaka, MD, PhD, of the UCSF-affiliated Gladstone Institutes and Kyoto University, received the Nobel Prize in Physiology or Medicine for his discovery that adult cells can be reprogrammed to behave like embryonic stem cells.

In the trial, human neural stem cells developed by Stem Cells, Inc., of Newark, California, were injected directly into the brains of four young children with an early-onset, fatal form of a condition known as Pelizaeus-Merzbacher disease (PMD).

This image illustrates direct injection of human neural stem cells into the brain's white matter, which is composed of bundles of nerve axons. There is lack of myelin, an insulating coating, in the severe pediatric condition Pelizaeus-Merzbacher disease (PMD). Over time, some stem cells become myelinating oligodendrocytes as reported in the papers from Uchida et al. and Gupta et al. Image by Kenneth Probst.

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Transplanted Neural Stem Cells Produced Myelin, UCSF Study Shows

BY LAURA OLENIACZ

loleniacz@heraldsun.com; 919-419-6636

DURHAM Stem cells from umbilical cord blood saved at 14-month-old Jase Howells birth are now being used in research to see if the cells can help his brain heal.

The research is looking into the use of the stem cells to treat brain damage from hydrocephalus, a condition characterized by the buildup of fluid in the skull.

His family traveled from Texas so he could receive an infusion on Tuesday at the Duke Childrens Hospital & Health Center of cord blood that was saved at his birth.

Mommys so proud of you, said LeaAnn Howell, to Jase, as he lay on a hospital bed, surrounded by medical personnel and family.

He periodically lifted his leg up and down to the beat of The Wheels on the Bus and other songs played by music therapist Tray Batson during the procedure.

Like I said, we were going to do anything humanly possible that we can do, Howell said in an interview prior to the procedure. Its a tough thing to fly, but once we (get here), I think the results are worth the wait, I guess.

The research into the use of cord blood stem cells to treat brain injury from hydrocephalus is being led by Dr. Joanne Kurtzberg, chief of the Division of Pediatric Blood and Marrow Transplantation at Duke.

The research is being done under a U.S. Food and Drug Administration Investigational New Drug application, Kurtzberg said.

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Duke med school gets FDA approval for stem cell product

ScienceDaily (Oct. 10, 2012) Physician-scientists at Oregon Health & Science University Doernbecher Children's Hospital have demonstrated for the first time that banked human neural stem cells -- HuCNS-SCs, a proprietary product of StemCells Inc. -- can survive and make functional myelin in mice with severe symptoms of myelin loss. Myelin is the critical fatty insulation, or sheath, surrounding new nerve fibers and is essential for normal brain function.

This is a very important finding in terms of advancing stem cell therapy to patients, the investigators report, because in most cases, patients are not diagnosed with a myelin disease until they begin to show symptoms. The research is published online in the journal Science Translational Medicine.

Myelin disorders are a common, extremely disabling, often fatal type of brain disease found in children and adults. They include cerebral palsy in children born prematurely as well as multiple sclerosis, among others.

Using advanced MRI technology, researchers at OHSU Doernbecher Children's Hospital also recently recognized the importance of healthy brain white matter at all stages of life and showed that a major part of memory decline in aging occurs due to widespread changes in the white matter, which results in damaged myelin and progressive senility (Annals of Neurology, September 2011).

In this breakthrough study, Stephen A. Back, M.D., Ph.D., senior author and clinician-scientist in the Pap Family Pediatric Research Institute at OHSU Doernbecher Children's Hospital, used a transgenic mouse model (Shiverer-immunodeficient) that develops progressive neurological deterioration because it is unable to make a key protein required to make normal myelin. Although this mouse has been widely investigated, prior to this study, true human brain-derived stem cells had not been tested for their potential to make new myelin in animals that were already deteriorating neurologically.

"Typically, newborn mice have been studied by other investigators because stem cells survive very well in the newborn brain. We, in fact, found that the stem cells preferentially matured into myelin-forming cells as opposed to other types of brain cells in both newborn mice and older mice. The brain-derived stem cells appeared to be picking up on cues in the white matter that instructed the cells to become myelin-forming cells," explained Back.

Although Back, in collaboration with investigators at StemCells Inc., had achieved success implanting stem cells in presymptomatic newborn animals, it was unclear whether the cells would survive after transplant into older animals that were already declining in health. Back and his colleagues put these cells to the test by transplanting them in animals that were declining neurologically and found that the stem cells were able to effectively survive and make functional myelin.

The study also is important because the research team was able to confirm by MRI that new myelin had been made by the stem cells within weeks after the transplant. Until now, it was unclear whether stem cell-derived myelin could be detected without major modifications to the stem cells, such as filling them with special dyes or iron particles that can be detected by the MRI.

These studies were particularly challenging, Back explained, because the mice were too sick to survive in the MRI scanner. Fortunately, OHSU is home to a leading national center for ultra-high field MRI scanners that were used to detect the myelin made by normal, unmodified stem cells.

"This is an important advance because it provides proof of principle that MRI can be used to track the transplants as myelin is being made. We actually confirmed that the MRI signal in the white matter was coming from human myelin made by the stem cells," Back said.

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Human neural stem cells study offers new hope for children with fatal brain diseases

Four boys with a rare and often fatal brain disease were implanted with stem cells that began fixing damage that impeded their ability to walk, talk and eat, a trial found.

The findings, published today in the journal Science Translational Medicine, are from the first stage of human tests funded by StemCells Inc. (STEM), a Newark, California-based company.

The children have a genetic disorder called Pelizaeus- Merzbacher, in which the brain cant make myelin, the fatty insulation for nerve cells that helps conduct brain signals. The children all had evidence of myelin growth a year later. The increased abilities shown by three of the boys in the University of California San Francisco study may bode well for other diseases caused by a lack of myelin insulation, including multiple sclerosis and cerebral palsy, the authors wrote.

Those were severely impaired children, said Stephen Back, a professor of pediatrics and neurology at Portlands Oregon Health & Science University, in a telephone interview. The fact that they showed any neurological improvement is very encouraging.

Back did work in mice that preceded todays work in humans, which he wasnt directly involved in. His study, published simultaneously, showed that the animals with no myelin at all grew some after being implanted with human stem cells.

Pelizaeus-Merzbacher disease causes the degeneration of the nervous system, and there is no cure or standard treatment. People with the illness experience a loss of coordination, thinking and motor abilities. Its one of several disorders linked to genes that control myelin production.

The incidence of the disease is 1 in 200,000 to 500,000 people, according to todays study of the boys.

The boys were between the ages of 1 and 6. They were given purified neural stem cells from a fetal brain, which was then grown in culture. The stem cells were inserted into the frontal lobe, using brain imaging as a guide. The boys brains were scanned 24 to 48 hours after surgery to assess safety.

The children were on drugs to suppress their immune systems and prevent their bodies from rejecting the stem cells for nine months. Side effects included rashes, diarrhea and fever. One boy had fluid collect under his scalp, which later vanished on its own. A second subject had some bleeding in the brain after the surgery, which was without clinical consequence, according to the paper.

One of the boys developed the ability to take steps with assistance and began to speak single words. Another started eating solid food on his own. A third began to walk without the assistance of a walker and began eating on his own.

Excerpt from:
Brain Stem-Cell Implants Help Children With Rare Illness

MIAMI--(BUSINESS WIRE)--

More than 500 business executives, entrepreneurs, academics and other stakeholders gathered at the JW Marriott Marquis for BioFloridas 15th Annual Conference, the hallmark industry event for Floridas bioscience industry. This years event was stacked with dynamic speakers and panelists representing the states growing bioscience community. The conference was kicked off with a keynote speech from Floridas 43rd Governor and bioscience champion, Jeb Bush, followed on Tuesday by an engaging keynote address from Bernie Siegel, J.D., founder and executive director of the Genetics Policy Institute and founder and chair of the World Stem Cell Summit.

BioFloridas goal is to support and maintain the momentum that Floridas bioscience industry has gained by coordinating and facilitating informative, interactive and industry-advancing networking events such as this years annual conference, said Russell Allen, president and CEO of BioFlorida.

The BioFlorida conference featured other high-profile speakers including the leaders of two Florida universities: Florida International University President Mark R. Rosenberg and University of Miami President Donna E. Shalala. Frank R. Nero, president and CEO of The Beacon Council, joined Rosenberg and Shalala for a discussion on higher educations role in building a bioscience economy.

Attendees engaged with state and global leaders from top research institutions, including the Sanford-Burnham Medical Research Institute and the University of Florida Sid Martin Biotechnology Incubator; and leading bio-focused companies including Medtronic, Merck & Company Inc., Biotest Pharmaceuticals Corporation and GE Healthcare. The conference discussions included panelists from several different countries including China, Denmark, England and Panama.

Not only did the conference provide a platform for the exchange of innovative ideas, global perspectives and best practices, it provided young companies an opportunity to connect with investors, said Les Croland, conference chair and attorney with Edwards Wildman Palmer, an international law firm that specializes in private equity, venture capital, corporate and finance transactions, among other specialties.

Over the past five years, an increasing number of bio-focused companies have chosen to open facilities in Florida. According to the University of Florida Sid Martin Biotechnology Incubators Florida BioDatabase, 215 biotechnology companies now call Florida home a more than 40 percent increase from five years ago. The state has also seen an increase in venture capital investment among start-up bioscience companies. As outlined in Floridas BioPulse: A Snapshot of the Bioscience Industry, the biomedical venture capital investments in Florida increased during 2011 to nearly $87 million, a 200 percent jump from 2010. BioFlorida is supporting this growth by providing networking and professional development platforms, such as the 15th Annual Conference for the states rising bioscience industry stakeholders.

Dr. Shailesh Chavan senior director of clinical research, medical affairs & drug safety at Biotest Pharmaceuticals, which opened a Florida facility in 2007 said the companys move was fueled by Floridas rising bioscience reputation.

While Biotests corporate headquarters are in Germany, Florida was strategically selected as the location of our state-of-the art manufacturing facility, said Dr. Chavan. Through investment and accelerated growth, Florida has established itself as a state that supports the advancement of bioscience research, innovation and business.

Floridas bioscience industry has a full calendar of events for the remainder of 2012, including the World Stem Cell Summit in West Palm Beach December 3 5. Additional upcoming events hosted by BioFlorida can be found here.

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Florida’s Largest Bioscience Event Exhibits Industry Innovation, Growth

* Vaccine was safe, showed signs of being effective

* Vaccine aims to train immune system to fight cancer

By Julie Steenhuysen

CHICAGO, Oct (KOSDAQ: 039200.KQ - news) 10 (Reuters) - A new type of cervical cancer vaccine made by Inovio Pharmaceuticals (AMEX: INO - news) has shown early promise as a potential treatment for pre-cancerous changes in the cervix, researchers at the company said on Wednesday.

Instead of preventing infections caused by certain strains of the human papillomavirus or HPV, as is the aim of Merck (BSE: MERCK.BO - news) 's Gardasil and GlaxoSmithKline (Other OTC: GLAXF.PK - news) 's Cervarix vaccine, the Inovio vaccine is designed to train the immune system to kill cells that spur cancer growth in women who are already infected.

Cervical cancer is the second most common cancer among women globally, causing 493,000 new cases and 274,000 deaths each year. About 10 to 25 percent of women who develop moderate to severe pre-cancerous lesions in their cervix, known as cervical intraepithelial neoplasia, are able to clear them on their own.

"It was not clear why that happens," said Joseph Kim, chief executive of Inovio Pharmaceuticals, which funded the study.

But many of these women tend to have higher levels of immune system cells known as T cells against two HPV-specific, cancer-causing genes known as E6 and E7 oncogenes.

The company set out to develop a vaccine to train a patient's immune system to make large quantities of these cells which could specifically target and kill these oncogenes.

"That is what this study has shown," Kim said of research published in the journal Science Translational Medicine.

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Vaccine to treat cervical cancer shows early promise

SAN FRANCISCO A Phase I clinical trial led by investigators from the University of California, San Francisco and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.

The study, published in today's (Oct. 10) issue of Science Translational Medicine, also demonstrated that the neural stem cells were safe in the patients' brains one year post transplant.

The results of the investigation, designed to test safety and preliminary efficacy, are encouraging, said principal investigator David H. Rowitch, M.D., Ph.D., a professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Children's Hospital and a Howard Hughes Medical Institute Investigator.

"For the first time, we have evidence that transplanted neural stem cells are able to produce new myelin in patients with a severe myelination disease," said Nalin Gupta, M.D., Ph.D., associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital, and co-principal investigator of the PMD clinical trial.

"We also saw modest gains in neurological function, and while these can't necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients, the findings represent an important first step that strongly supports further testing of this approach as a means to treat the fundamental pathology in the brain of these patients."

In the trial, human neural stem cells developed by StemCells, Inc., of Newark, Calif., were injected directly into the brains of four young children with an early-onset, fatal form of a condition known as Pelizaeus-Merzbacher disease (PMD).

In PMD, an inherited genetic defect prevents brain cells called oligodendrocytes from making myelin, a fatty material that insulates white matter which serves as a conduit for nervous impulses throughout the brain. Without myelin sheathing, white matter tracts short-circuit like bare electrical wires and are unable to correctly propagate nerve signals, resulting in neurological dysfunction and neurodegeneration. Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death between ages 10 and 15. The disease usually occurs in males.

Multiple sclerosis and certain forms of cerebral palsy also involve damage to oligodendrocytes and subsequent demyelination.

Before and after the transplant procedures in the children with PMD, which were conducted between 2010-11, the patients were given standard neurological examinations and developmental assessments, and underwent magnetic resonance imaging (MRI). "MRI is the most stringent non-invasive method we have of assessing myelin formation," said Rowitch.

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Trial: Transplanted neural stem cells produced myelin

Phase I Investigation Demonstrates Signs of Engraftment and Safety at One Year

Newswise A Phase I clinical trial led by investigators from the University of California, San Francisco and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.

The study, published in the Oct. 10, 2012 issue of Science Translational Medicine, also demonstrated that the neural stem cells were safe in the patients brains one year post transplant.

The results of the investigation, designed to test safety and preliminary efficacy, are encouraging, said principal investigator David H. Rowitch, MD, PhD, a professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Childrens Hospital and a Howard Hughes Medical Institute Investigator.

For the first time, we have evidence that transplanted neural stem cells are able to produce new myelin in patients with a severe myelination disease, said Nalin Gupta, MD, PhD, associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital, and co-principal investigator of the PMD clinical trial.

We also saw modestgains in neurological function, and while these cant necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients,the findings represent an important first step that strongly supports further testing of this approach as a means to treat the fundamental pathology in the brain of these patients.

In the trial, human neural stem cells developed by StemCells, Inc., of Newark, California, were injected directly into the brains of four young children with an early-onset, fatal form of a condition known as Pelizaeus-Merzbacher disease (PMD).

In PMD, an inherited genetic defect prevents brain cells called oligodendrocytes from making myelin, a fatty material that insulates white matter which serves as a conduit for nervous impulses throughout the brain. Without myelin sheathing, white matter tracts short-circuit like bare electrical wires and are unable to correctly propagate nerve signals, resulting in neurological dysfunction and neurodegeneration. Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death between ages 10 and 15.The disease usually occurs in males.

Multiple sclerosis and certain forms of cerebral palsy also involve damage to oligodendrocytes and subsequent demyelination.

Before and after the transplant procedures in the children with PMD, which were conducted between 2010-2011, the patients were given standard neurological examinations and developmental assessments, and underwent magnetic resonance imaging (MRI). MRI is the most stringent non-invasive method we have of assessing myelin formation, said Rowitch. The investigators found evidence that the stem cells had successfully engrafted, receiving blood and nutrients from the surrounding tissue and integrating into the brain, a process that Rowitch likened to a plant taking root.

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Study Shows Evidence that Transplanted Neural Stem Cells Produced Myelin

NEWARK, Calif., Oct. 10, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that two papers reporting clinical and preclinical data demonstrating the therapeutic potential of the Company's proprietary HuCNS-SC(R) cells (purified human neural stem cells) for a range of myelination disorders were published in the Oct. 10 edition of Science Translational Medicine, the peer review journal of the American Association for the Advancement of Science (http://stm.sciencemag.org/).

The paper by Gupta, et al. describes the encouraging results of the Company's Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a genetic myelination disorder that afflicts children. In the trial, which was completed in February 2012, four patients were transplanted with the Company's HuCNS-SC cells and all showed preliminary evidence of progressive and durable donor cell-derived myelination. Three of the four patients showed modest gains in their neurological function, which suggests a departure from the natural history of the disease; the fourth patient remained stable. Although clinical benefit cannot be confirmed in a trial without control patients, the small but measureable gains in function at one year may represent signals of a clinical effect to be further investigated in a controlled trial with more patients.

The second of the two papers, by Uchida, et al., summarizes extensive preclinical research which demonstrated that transplantation of the Company's neural stem cells in an animal model of severe myelin deficiency results in new myelin which enhanced the conductivity of nerve impulses. Myelin is the substance that insulates nerve axons, and without sufficient myelination, nerve impulses are not properly transmitted and neurological function is impaired. This preclinical data provided the rationale for the PMD clinical trial and supports the Company's cell therapy approach to other myelination disorders, such as transverse myelitis, certain forms of cerebral palsy, and multiple sclerosis.

"For the first time, we have evidence that transplanted neural stem cells are able to produce new myelin in patients with a severe myelination disease," Nalin Gupta, MD, PhD, associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital, and co-principal investigator of the PMD clinical trial. "We also saw modest gains in neurological function, and while these can't necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients, it is an important first step which provides hope that HuCNS-SC transplantation may be able to address the fundamental pathology in the brain of PMD patients."

Patients with PMD have a defective gene which leads to insufficient myelin in the brain, which leads to a progressive loss of neurological function and death. In the clinical trial, four patients with connatal PMD, the most severe form of the disease, were enrolled and transplanted with HuCNS-SC cells. The patients were followed for twelve months after transplantation, during which time they underwent intensive neurological assessments and magnetic resonance (MR) imaging at regular intervals. The findings from the trial indicate a favorable safety profile for the HuCNS-SC cells and the transplantation procedure. Analysis of the MR imaging data showed changes consistent with increased myelination in the region of the transplantation, and which progressed over time and persisted after the withdrawal of immunosuppression at nine months. The results support the conclusion of durable cell engraftment and donor-derived myelin in the transplanted patients' brains. The development of new myelin signals is unprecedented in patients with connatal PMD. In addition, clinical assessment revealed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable. While clinical benefit cannot be confirmed without a controlled study, these clinical outcomes suggest the HuCNS-SC cells may be having a beneficial effect on the patients.

The second paper, whose lead author is Nobuko Uchida, Vice President of Stem Cell Biology at StemCells, Inc., describes research which shows that when HuCNS-SC cells were transplanted into the shiverer mouse, a common model of severe central nervous system (CNS) dysmyelination, the cells formed new, functional myelin in the mice. Sophisticated analytical techniques were used to confirm that changes measured by MR images were in fact derived from new human myelin generated by the transplanted HuCNS-SC cells. MR imaging is routinely used in the diagnosis and clinical characterization of demyelinating diseases such as multiple sclerosis, and these results supported the use of similar techniques to detect and evaluate the degree of myelination in the Phase I PMD trial. Moreover, the new myelin was shown to be functional as conductivity of nerve impulses in the mice was enhanced.

"Demonstration of functional myelin formation in animals showing disease symptoms is significant and opens up the potential to treat patients with a range of severe myelin disorders," said Stephen A. Back, MD, PhD, professor of pediatrics and neurology at Oregon Health & Science University Doernbecher Children's Hospital, and senior author of the preclinical paper.

Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc., added, "Having these two papers published concurrently illustrates the direct pathway of how we are translating groundbreaking scientific research to the clinical setting. The data in these papers make a powerful statement about the potential of our HuCNS-SC cells to address not only PMD, but a wide spectrum of myelination disorders. We are actively moving forward with our plans to conduct a controlled Phase II clinical study in PMD and evaluating our next steps with respect to other myelination disorders."

Conference Call

StemCells, Inc. will host a live webcast, today, October 10, at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss the data reported in these papers. Interested parties are invited to view the webcast over the Internet via the link at http://www.stemcellsinc.com/News-Events/Events.htm. An archived version of the webcast will be available for replay on the Company's website approximately two hours following the conclusion of the live event and will be available for a period of 30 days.

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StemCells, Inc. Announces Simultaneous Publication of Preclinical and Clinical Results of Its Neural Stem Cells for ...

Public release date: 10-Oct-2012 [ | E-mail | Share ]

Contact: Jennifer O'Brien jennifer.obrien@ucsf.edu 415-502-6397 University of California - San Francisco

A Phase I clinical trial led by investigators from the University of California, San Francisco and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.

The study, published in the Oct. 10, 2012 issue of Science Translational Medicine, also demonstrated that the neural stem cells were safe in the patients' brains one year post transplant.

The results of the investigation, designed to test safety and preliminary efficacy, are encouraging, said principal investigator David H. Rowitch, MD, PhD, a professor of pediatrics and neurological surgery at UCSF, chief of neonatology at UCSF Benioff Children's Hospital and a Howard Hughes Medical Institute Investigator.

"For the first time, we have evidence that transplanted neural stem cells are able to produce new myelin in patients with a severe myelination disease," said Nalin Gupta, MD, PhD, associate professor of neurological surgery and pediatrics and chief of pediatric neurological surgery at UCSF Benioff Children's Hospital, and co-principal investigator of the PMD clinical trial.

"We also saw modest gains in neurological function, and while these can't necessarily be attributed to the intervention because this was an uncontrolled trial with a small number of patients, the findings represent an important first step that strongly supports further testing of this approach as a means to treat the fundamental pathology in the brain of these patients."

In the trial, human neural stem cells developed by StemCells, Inc., of Newark, California, were injected directly into the brains of four young children with an early-onset, fatal form of a condition known as Pelizaeus-Merzbacher disease (PMD).

In PMD, an inherited genetic defect prevents brain cells called oligodendrocytes from making myelin, a fatty material that insulates white matter which serves as a conduit for nervous impulses throughout the brain. Without myelin sheathing, white matter tracts short-circuit like bare electrical wires and are unable to correctly propagate nerve signals, resulting in neurological dysfunction and neurodegeneration. Patients with early-onset PMD cannot walk or talk, often have trouble breathing and undergo progressive neurological deterioration leading to death between ages 10 and 15.The disease usually occurs in males.

Multiple sclerosis and certain forms of cerebral palsy also involve damage to oligodendrocytes and subsequent demyelination.

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UCSF study shows evidence that transplanted neural stem cells produced myelin

LA JOLLA, Calif., Oct. 10, 2012 /PRNewswire/ --New research directions are being explored to find therapies for hard to treat diseases. One exciting new approach is the use of autologous Adult Stem Cells. Multiple Sclerosis (MS) is one of the many notable diseasesadult stem cell therapycould potentially impact. Multiple Sclerosis (MS) is a disorder in which an individual's own immune system attacks the 'myelin sheath'. The myelin sheath serves to protect the nerve cells within the body's central nervous system (CNS). The damage caused by MS may result in many types of symptoms including:

(Photo: http://photos.prnewswire.com/prnh/20121010/LA89802-INFO)

Currently there is no cure for MS, but MS stem cell therapiesattempt to slow the disease's progression and limit symptoms. Since adult stem cells have the ability to differentiate into many different types of cells, such as those required for proper functioning and protection of nerve cells, the use of adult stem cells for MS therapy could be of substantial value. Adult stem cells can be isolated with relative ease from an individual's own 'adipose' (fat) tissue. As a result, adult stem cell therapy is not subject to the ethical or religious issues troubling embryonic methods.

Encouragingly for MS treatment potential, scientific researchers have been studying the properties of adipose-derived stem cells. Their results from canine and equine studies suggest anti-inflammatory and regenerative roles for these stem cells. Also, further research findings suggest these adipose-derived stem cells can have specific immune-regulating properties. Markedly, clinical-based work conducted overseas has indicated that individuals suffering from MS could respond well to adipose-derived stem cell treatment, with a substantially improved quality of life.

The US based company, StemGenex, is pioneering new methods for using adipose derived adult stem cells to help in diseases with limited treatment options like MS. StemGenex has been conducting research with physicians over the last 5 years to advance adult stem cell treatment protocols for alleviating MS symptoms. StemGenex's proprietary protocol includes the use of a double activation process, which increases both the viability and the quantity of stem cells that are received in a single application.

To find out more about stem cell treatments contact StemGenex either by phone at 800.609.7795 or email at Contact@StemGenex.com.

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StemGenex™ on Adult Stem Cell-Based Therapy for Multiple Sclerosis

Nobel laureate Shinya Yamanaka warned patients on Tuesday about unproven "stem cell therapies" offered at clinics and hospitals in a growing number of countries, saying they were highly risky.

The Internet is full of advertisements touting stem cell cures for just about any disease -- from diabetes, multiple sclerosis, arthritis, eye problems, Alzheimer's and Parkinson's to spinal cord injuries -- in countries such as China, Mexico, India, Turkey and Russia.

Yamanaka, who shared the Nobel Prize for Medicine on Monday with John Gurdon of the Gurdon Institute in Cambridge, Britain, called for caution.

"This type of practice is an enormous problem, it is a threat. Many so-called stem cell therapies are being conducted without any data using animals, preclinical safety checks," said Yamanaka of Kyoto University in Japan.

"Patients should understand that if there are no preclinical data in the efficiency and safety of the procedure that he or she is undergoing ... it could be very dangerous," he told Reuters in a telephone interview.

Yamanaka and Gurdon shared the Nobel Prize for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.

"I hope patients and lay people can understand there are two kinds of stem cell therapies. One is what we are trying to establish. It is solely based on scientific data. We have been conducting preclinical work, experiments with animals, like rats and monkeys," Yamanaka said.

"Only when we confirm the safety and effectiveness of stem cell therapies with animals will we initiate clinical trials using a small number of patients."

Yamanaka, who calls the master stem cells he created "induced pluripotent stem cells" (iPS), hopes to see the first clinical trials soon.

"There is much promising research going on," he said.

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Nobel prize winner in medicine warns of rogue 'stem cell therapies'

NEW YORK, Oct. 10, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NBS), an emerging leader in the fast growing cell therapy market, announced today that a new article published by the International Scholarly Research Network provides further evidence that AMR-001, NeoStem's lead product candidate through its Amorcyte subsidiary, appears capable of preserving heart muscle function following a large myocardial infarction. Amorcyte demonstrated in its Phase 1 trial that AMR-001 preserved heart muscle function when a therapeutic dose of cells was administered. No patient experienced a deterioration in heart muscle function who received 10 million cells or more whereas 30 to 40 percent of patients not receiving a therapeutic dose did. The new study shows that cardiac muscle function sparing effects are evident even earlier after treatment than previously shown.

The article titled "Assessment of myocardial contractile function using global and segmental circumferential strain following intracoronary stem cell infusion after myocardial infarction: MRI Feature Tracking Feasibility Study" by Sabha Bhatti, MD, et al. appears in ISRN Radiology Volume 2013, Article ID 371028 and is published online at http://www.isrn.com/journals/radiology/2013/371028. The publication by Dr. Bhatti and colleagues, including Dr. Andrew Pecora, Chief Medical Officer of NeoStem, supports the finding that AMR-001 preserves heart function. Previously, Amorcyte, a NeoStem subsidiary, showed that six months after STEMI AMR-001 improved blood flow to the heart and preserved heart muscle. By using cardiac magnetic resonance imaging, specifically measuring circumferential strain of the left ventricle, the authors show that AMR-001's effects are evident by three months after STEMI.

AMR-001's angiogenic and anti-apoptotic mechanisms of action indicate that preservation of heart muscle function should start within weeks and be evident in fewer than 6 months. This publication, based on blinded analysis of Amorcyte's Phase 1 data, confirms the expected time course for AMR-001's mechanism of action. In the context of previously published results, these effects are durable.

Amorcyte is developing AMR-001, a cell therapy for the treatment of cardiovascular disease, and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving cardiac function and preventing adverse clinical events after a large myocardial infarction.

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy, capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT"), with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, as mentioned above, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cell product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a substantial intellectual property estate, we believe we are well positioned to succeed.

Forward-Looking Statements for NeoStem, Inc.

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property, (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

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NeoStem Announces New Publication That Supports Positive Results of AMR-001 for Treatment of AMI