Stem Cells and Spinal Cord Injury:

Spinal cord injuries are described at various levels of "incomplete", which can vary from having no effect on the patient to a "complete" injury which means a total loss of function.

Treatment of spinal cord injuries starts with restraining the spine and controlling inflammation to prevent further damage. The actual treatment can vary widely depending on the location and extent of the injury. In many cases, spinal cord injuries require substantial physical therapy and rehabilitation, especially if the patient's injury interferes with activities of daily life.

After a spinal cord injury, many of the nerve fibers at the injury site lose their insulating layer of myelin. As a result, the fibers are no longer able to properly transmit signals between the brain and the spinal cord contributing to paralysis. Unfortunately, the spinal cord lacks the ability to restore these lost myelin-forming cells after trauma.

Tissue engineering in the spinal cord involves the implantation of scaffold material to guide cell placement and foster cell development. These scaffolds can also be used to deliver stem cells at the site of injury and maximize their regenerative potential.

When the spinal cord is damagedeither accidentally (car accidents, falls) or as the result of a disease (multiple sclerosis, infections, tumors, severe forms of spinal bifida, etc.)it can result in the loss of sensation and mobility and even in complete paralysis.

Spinal Cord Injury and Stem Cell Treatment

Adult stem cell transplants for spinal cord injury repair: current state in preclinical research.

Hernndeza J, Torres-Espna A, Navarro X.

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Spinal Cord Injury Stem Cell Treatment - ASCI - Stem Cell Rejuvenation

The Promise of Stem Cells

Studying stem cells will help us understand how they transform into the dazzling array of specialized cells that make us what we are. Some of the most serious medical conditions, such as cancer and birth defects, are due to problems that occur somewhere in this process. A better understanding of normal cell development will allow us to understand and perhaps correct the errors that cause these medical conditions.

Another potential application of stem cells is making cells and tissues for medical therapies. Today, donated organs and tissues are often used to replace those that are diseased or destroyed. Unfortunately, the number of people needing a transplant far exceeds the number of organs available for transplantation. Pluripotent stem cells offer the possibility of a renewable source of replacement cells and tissues to treat a myriad of diseases, conditions, and disabilities including Parkinson's disease, amyotrophic lateral sclerosis, spinal cord injury, burns, heart disease, diabetes, and arthritis.

Scientists have been able to do experiments with human embryonic stem cells (hESC) since 1998, when a group led by Dr. James Thomson at the University of Wisconsin developed a technique to isolate and grow the cells. Although hESCs are thought to offer potential cures and therapies for many devastating diseases, research using them is still in its basic stages. hESCs are thought to offer potential cures and therapies for many devastating diseases, and we are now seeing the first clinical trials using cells derived from hESCs.

The NIH funded its first basic research study on hESCs in 2002. Since that time, biotechnology companies have built upon those basic foundations to begin developing stem cell-based human therapies. There are currently two active clinical trials using cells derived from human embryonic stem cells, both being conducted by a biotechnology company called ACT. The company has laboratories in Marlborough, Massachusetts and corporate offices in Santa Monica, California. ACT has begun enrolling patients for Phase I (safety and tolerability) clinical trials of two hESC-derived stem cell products:

In January, 2012, the investigators published a preliminary report on the first two patients treated with hESC-derived cells: http://www.ncbi.nlm.nih.gov/pubmed/22281388. A third patient was treated on April 20, 2012.

Late in 2007, scientists reported that they had been able to reprogram adult human skin cells to behave like hESCs. This type of stem cells is known as induced pluripotent stem cells, or iPSCs. Since these first reports, researchers have rapidly improved the techniques to generate iPSCs, creating a powerful new way to "de-differentiate" cells whose developmental fates were thought to be determined. In July 2013, Japans health minister approved the first clinical trial using cells derived from iPSCs. Masayo Takahashiin Kobe, Japan will use the cells to attempt to treat a form of blindness - age-related macular degeneration.

Bone marrow contains blood-forming stem cells (hematopoietic stem cells) that have been used for decades to treat blood cancers and other blood disorders. Umbilical cord blood is another source of hematopoietic stem cells that is being used in treatment. You can see a list of diseases that may currently be treated with hematopoietic stem cells at the website of the National Marrow Donor Program. You may also search for clinical trials testing "bone marrow stem cells" or "umbilical cord blood" on the ClinicalTrials.gov website.

A biotechnology company called Neuralstem (corporate headquarters in Rockville, Maryland) is conducting a clinical trial testing the use of human spinal cord stem cells to treat Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrigs Disease. The company obtained FDA approval to conduct a Phase I trial (safety and tolerability study) and began enrolling patients in January 2010. Twelve participants have received lumbar transplants, and in March 2012, the second participant received an injection in the cervial region. Details about this trial are listed on the ClinicalTrials.gov website.

Osiris Therapeutics (Columbia, Maryland) is conducting three different Phase 2 clinical trials with a product from adult mesenchymal cells (called Prochymal). The three trials are for:

See the article here:
Stem Cells and Diseases [Stem Cell Information]

CIRM funds a variety of research projects focused on finding a treatment for people with spinal cord injury. These projects range from basic work understanding how nerve cells are damaged in these injuries to projects trying move therapies into clinical trials.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research: Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials: CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

About 250,000 people in the U.S. live with spinal cord injuries. Half of those are quadriplegic, with the paralysis impacting all four limbs to some extent. For those individuals the lifetime cost of managing their condition is estimated to be between $2 million and $3 million.

Spinal cord injury became the first condition targeted in a human clinical trial using cells made from embryonic stem cells. That trial, begun by Geron in 2010 and based on the findings of a team CIRM currently funds, was later cancelled by Geron for financial reasons. By the time of the cancellation five patients around the country had been enrolled in the study, including two at Stanford, who entered the trial during a period when CIRM funded Geron. Those patients continue to be followed to learn as much as possible about this approach.

Californias stem cell agency retains many grants for research to move potential spinal cord injury therapies forward (the full list is below). Much of this work focuses on trying to determine which type of nerve cell is the best one to transplant, and deciding which type of stem cell is the best starting point for making those cells. Other research is trying to see if these transplanted cells become part of the existing nerve system, helping create new pathways that can transmit nerve signals to muscles. The researchers are also looking at ways to try and improve the ability of these transplanted cells to become part of the nerve system.

One obstacle that some teams are trying to overcome is the tendency of the scar at the site of injury to block the growth of these transplanted cells. One group is trying to overcome that by combining stem cells with synthetic scaffolds that can be placed at the site of injury, to help the cells bridge the scar and restore signals. In animal models this combination has resulted in an increase in mobility compared to stem cell grafts alone.

Progress and Promise toward a stem cell-based therapy for spinal cord injury

Total:

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Spinal Cord Injury Fact Sheet | California's Stem Cell Agency

ROCKVILLE, Md., Dec. 10, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that Jonathon Glass, MD, Director of the Emory ALS Center, presented new data from the Phase I trial of Neuralstem's human spinal cord stem cells, NSI-566, in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) at the International Symposium on ALS/MND in Chicago, sponsored by the Motor Neurone Disease Association. In a Thursday presentation, "RESULTS OF PHASE 1 TRIAL OF SPINAL CORD TRANSPLANTATION OF NEURAL PROGENITOR CELLS IN ALS (THE NEURALSTEM, INC. TRIAL)," Dr. Glass revealed that researchers were able to establish the long-term survival of Neuralstem's transplanted spinal cord stem cells in autopsied patients, through a technology called DNA fingerprinting. Dr. Glass, who is the principal site investigator of the trial at Emory, also announced that the study team has received a grant from the National Institutes of Health (NIH) to cover a majority of the cost of an upcoming Phase II trial.

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"We are quite pleased by our ability to perform all of the surgeries planned for the Phase I trial without evidence of significant surgical or medical complications, including those patients who received both lumbar and cervical transplantations," said Dr. Glass. "We can also report that we found evidence of cell survival in all of the patients who came to autopsy, including our first patient who died 30 months after transplantation.

This is very positive news, supporting our plan to accelerate this study by increasing the dose of stem cells delivered to the cervical spinal cord in the hopes of delaying respiratory failure and prolonging life. The next phase of the study has been partially funded by a generous grant from the National Institutes of Health, and we will begin once the FDA approves our new protocol," Dr. Glass concluded.

"This is a major finding," said Karl Johe, Ph.D., Neuralstem Chairman of the Board and Chief Scientific Officer. "There is currently no way to confirm the survival of the cells in patients while they are alive. Levels of functional recovery, or a slowdown in the progression of the disease in various patients, have given us reason to believe the cells have survived. Now, cell survival has been demonstrated by definitive evidence.

Among the six patients autopsied (five died of ALS disease progression and one, of unrelated heart failure), the survival period, from stem cell transplantation to death, ranged from 196 921 days. Five of these patients had discontinued all immune suppression medications for 57 638 days prior to death, but showed the stem cell DNA content in the range of 0.67% - 5.4% of total DNA in some spots of cord treated with the stem cells. There was no correlation of DNA content to survival period without immune suppression medication. These data, therefore, suggest that long-term immuno suppression of patients is not required for long-term survival of our cells, which points towards the feasibility of needing only transient immune suppression in future ALS trials," Dr. Johe concluded.

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem has recently treated the last patient in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in spinal cord injury.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company is in a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD).Additional indications could include chronic traumatic encephalopathy (CTE), Alzheimer's disease, and post-traumatic stress disorder (PTSD).

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Neuralstem Investigator Presents New ALS NSI-566 Data at International Symposium on ALS/MND

BANGKOK, Oct 18 - A Siriraj Hospital medical team today announced its successful isolation of stem cells from amniotic fluid which they said will contribute to treatments of many chronic diseases.

Dr Udom Kachintorn, dean of the Siriraj medical school, said further research will be conducted on the use of stem cells to treat various illnesses including Alzheimers, osteoarthritis, diabetes and spinal cord pain. These chronic ailments are related to deteriorating stem cells in a human body.

A preliminary lab test has been done with animals but it will take some time before the medical team starts testing on human beings, he said.

Dr Udom said stem cells normally spread all over a person's body but they are abundant and pure in an infant's umbilical cord and placenta.

Dr Tassanee Permthai, chief of the stem cell research project, said stem cells from the placenta of a four-month-old baby can be transplanted in a patient like fully-developed stem cells.

Once transplanted in a patients body, the stem cells evolve into cells in different parts of the body, she explained, adding that the transplantation of stem cells can also prevent tumours.

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Pattaya Mail

ScienceDaily (Oct. 15, 2012) New animal studies provide additional support for investigating stem cell treatments for Parkinson's disease, head trauma, and dangerous heart problems that accompany spinal cord injury, according to research findings released today.

The work, presented at Neuroscience 2012, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health, shows scientists making progress toward using stem cell therapies to repair neurological damage.

The studies focused on using stem cells to produce neurons -- essential, message-carrying cells in the brain and spinal cord. The loss of neurons and the connections they make for controlling critical bodily functions are the chief hallmarks of brain and spinal cord injuries and of neurodegenerative afflictions such as Parkinson's disease and ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease.

Today's new findings show that:

Other recent findings discussed show that:

"As the fields of developmental and regenerative neuroscience mature, important progress is being made to begin to translate the promise of stem cell therapy into meaningful treatments for a range of well-defined neurological problems," said press conference moderator Jeffrey Macklis, MD, of Harvard University and the Harvard Stem Cell Institute, an expert on development and regeneration of the mammalian central nervous system. "Solid, rigorous, and well-defined pre-clinical work in animals can set the stage toward human clinical trials and effective future therapies."

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The above story is reprinted from materials provided by Society for Neuroscience (SfN), via AlphaGalileo.

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Realizing the potential of stem cell therapy: Studies report progress in developing treatments for diseases and injuries

ROCKVILLE, Md., Oct. 15, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that data on Neuralstem's NSI-566 spinal cord-derived neural stem cell line in a rat model of ischemic stroke was presented in a poster, "Histopathological Assessment of Adult Ischemic Rat Brains after 4 Weeks of Intracerebral Transplantation of NSI-566RSC Cell Line," at The Society for Neurosciences Annual Meeting (http://www.sfn.org/AM2012/). This study was conducted independently in the laboratory of Dr. Cesar Borlongan, who is the director at the Center of Excellence for Aging and Brain Repair at the University of South Florida College of Medicine. Post-mortem histology was conducted in collaboration with Neuralstem. Rats that suffered ischemic stroke by middle cerebral artery occlusion, were transplanted 7 days post-stroke with increasing doses of NSI-566 into the stroke area. The animals were followed for safety and behavioral response for 56 days post-transplantation. Researchers reported Saturday that there was significant improvement in both motor and neurological tests in the stem cell-treated rats. There were significant dose-dependent differences in the behavioral improvement across treatment groups at post-transplantation periods, with the highest dose showing the most significant improvement in both motor and neurological tests. Similarly, there were significant differences in the behavioral performance among treatment groups at post-transplantation periods, with the most significant improvement in both motor and neurological tests seen at day 56 post-transplantation.

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"This study was designed to evaluate the potential therapeutic value of intracerbral dosing of human neural stem cells (NSI-566, supplied by Neuralstem) in an animal model of adult ischemic stroke," said Cesar V. Borlongan, Ph.D., University of South Florida College of Medicine, and the lead study author. "The results are very clear. The recovery of motor and neurological tests demonstrated by high-dose transplanted stroke animals was significantly better throughout the 56-day study period compared to vehicle-infused stroke animals, or low-dosed animals. In addition, there was stable improvement in the high-dose animals, and they showed a trend of better improvement over time."

A separate poster, "Survival and Differentiation of Human Neural Stem Cells (NSI-566RSC) After Grafting into Ischemia-Injured Porcine Brain," was also presented on Saturday. This study was independently carried out by Dr. Martin Marsala and his colleagues. Dr. Marsala is a professor and the head of the Neuroregeneration Laboratory at University of California San Diego and also a member of the Sanford Consortium for Regenerative Medicine. In this study, the same stem cells were transplanted into the brains of pigs that received an ischemic stroke on one side of the brain. 8-9 weeks after the ischemic event, which models chronic stroke in humans, feasibility and safety of escalating cell doses and injections were assessed. Body temperature, behavior, muscle tone and coordination, sensory function, food consumption, defecation, and micturition were monitored at least twice daily for the first 7 days, and once weekly thereafter, until termination. Up to 12 million cells in 25 cell injection deposits via 5 cannula penetrations were shown to be safe, which closely mimics the intended clinical route and method of delivery in future human clinical trials. At 6 weeks post-transplantation, there were no complications from the cell transplantation method or the cells. All animals recovered and showed progressive improvement with no distinction. All treated animals showed effective engraftment and neuronal maturation with extensive axonal projections. These data support the application of NSI-566RSC cell line to be transplanted into a chronic stage of previously ischemia-injured brain for treatment of motor deficits resulting from stroke.

"Our study was designed to evaluate the potential value of Neuralstem's cells in a chronic model of ischemic stroke and in a species that allowed for the use of human scale transplantation tools and dosing," said Martin Marsala, MD, at the University of California at San Diego Medical School, and the lead study author of the porcine study. "We have demonstrated clearly that both the route of administration and the cells are safe and well tolerated and that the cells survived and differentiated into mature neurons in the host brain tissue."

"We have demonstrated safety and efficacy of NSI-566RSC in a subacute model of ischemic stroke in rats and feasibility and safety in a chronic model of ischemic stroke in mini-pigs," said Karl Johe, PhD, Chairman of Neuralstem's Board of Directors and Chief Scientific Officer. "Together, these two studies demonstrate strong proof of principle data that our NSI-566 cells are ready to go into humans to treat paralysis in stroke patients."

Neuralstem has recently completed a Phase I trial testing the safety of NSI-566 in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and has been approved to initiate a human clinical trial in ischemic stroke in China, through its subsidiary, Suzhou Neuralstem.

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem has recently treated the last patient in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in spinal cord injury.

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Significant Recovery Of Motor And Neurological Functions In Ischemic Stroke Rats With Neuralstem NSI-566 Cells

NEWARK, Calif., Oct. 12, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced the issuance of U.S. Patent Number 8,283,164 titled "Liver engrafting cells, assays, and uses thereof." The patent broadly covers purified populations of human liver cells, including the Company's human liver engrafting cells (hLEC). hLEC cells were first isolated by Company researchers in the late 1990s, and Company scientists have repeatedly demonstrated the cells' engraftment and robust bioactivity in vivo and that they are expandable. While the Company's hLEC cells are purified from donated adult livers not suitable for transplant, the newly issued '164 patent importantly claims cells independent of tissue source. Therefore, the '164 patent has potential relevance to those deriving liver cells from iPS or ESC platforms. The term of the '164 patent extends into 2022.

"This new patent extends our IP protection around hLEC cells and should be of interest to those searching for an expandable human liver cell," said Martin McGlynn, President and Chief Executive Officer of StemCells, Inc. "Because the liver is such a key organ, finding an expandable, reliable and well-characterized liver cell population is an important step forward in both medical research and drug development. For example, liver disease afflicts some 25 million Americans and transplantation of an expandable liver cell could potentially address many of the shortcomings of whole liver transplantation. Moreover, the right liver cells could make profound contributions to drug screening and toxicity testing."

In October 2011, StemCells formed a wholly-owned subsidiary to focus on both the therapeutic and research tool applications of its hLEC technologies and to serve as an investment vehicle for those interested in a "pure play" liver cell company. The '164 patent is one of several patents issued to the Company on a worldwide basis claiming expandable liver cells, including U.S. Patent Nos. 7,811,818 and 7,211,404, Japan Patent No. 4445876, Australian Patent No. 2002315392, and European Patent No. 1406998. Patent prosecution in the family is ongoing on a worldwide basis, including China application 02816528.4.

About StemCells, Inc.

StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. The Company recently reported results from a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children. The trial results showed preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The Company is also conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and a Phase I/II clinical trial in dry age-related macular degeneration in the United States. In addition, the Company is pursuing preclinical studies of its HuCNS-SC cells in Alzheimer's disease. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand. Further information about StemCells is available at http://www.stemcellsinc.com.

The StemCells, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7014

Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the Securities Act of 1933, as amended, and the Securities Exchange Act of 1934, as amended, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the prospect of enforcing the Company's intellectual property against infringers, the potential breadth and length of patent protection in the United States or in any other geography; and the likelihood that any of the Company's intellectual property will be found to be valid and enforceable. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including the Company's ability to obtain the increased capital resources needed to continue its current operations and to conduct the research, preclinical development and clinical trials necessary for regulatory approvals and for continued patent prosecution efforts; uncertainty regarding the validity and enforceability of the Company's existing patents; and other factors that are described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2011, and in its subsequent reports on Form 10-Q and Form 8-K.

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StemCells, Inc. Awarded Broad U.S. Patent Covering Expandable Liver Cells

SAN FRANCISCO (KGO) -- Bay Area stem cell researchers are reporting early, encouraging results from two clinical trials. The first, involves patients, paralyzed with spinal cord injuries and a treatment that could offer new hope for their future.

Nearly 20 years after the football injury that left him paralyzed, Roman Reed still holds onto the hope that he will someday walk again.

"One hundred percent, without a doubt. I've been wrong about the date, but not the fact I will walk again," said Reed.

Reed now runs a foundation to promote stem cell research and has been closely watching a clinical trial being conducted by Bay Area based Stem Cells Inc. Its goal is to use stem cell therapy to restore motor function in patients with spinal cord injuries.

"We're on the road on to being able to cure paralysis, it's so important, and stem cells are the way to do it," said Reed.

Stephen Huhn, M.D., Ph.D., from Stem Cells Inc., says the test procedure began a two hour surgery to clear a path to the spinal cord. Researchers then injected the cells directly into the damaged area.

"So the first three patients in the trial were designed to enroll patients who had the worst of the worst injuries. In other words, complete loss of sensory function and complete loss of motor function below the level of injury," said Huhn.

The phase one trials are all about establishing safety, but six months out, the researchers began measuring some intriguing improvements in two of those three patients. Both reported feeling in areas below the areas of their injuries.

The company cautions that the data is very preliminary, but they say researchers were able to measure the improved sensory response using several testing methods, including electrical stimulation, and response to heat -- which are considered more accurate than the patient's own self-reporting.

"You can't fake that. When we saw that data, that's when we became very excited," said Martin McGlynn, the CEO of Stems Cells Inc.

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Bay Area stem cell researchers see encouraging results

Public release date: 11-Oct-2012 [ | E-mail | Share ]

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (October 11, 2012) For the second day, The New York Stem Cell Foundation (NYSCF) Seventh Annual Translational Stem Cell Research Conference hosts the world's most preeminent stem cell scientists to present their findings on how advances in stem cell science lead to better treatments and cures for disease and injury. The conference is held at The Rockefeller University in Manhattan on October 10-11.

Today, in disease-specific sessions, scientists will share their latest finds in moving stem cell research to treatments in the following areas: cancer and blood disease; diabetes and autoimmunity; heart and muscles; neurodegeneration and spinal cord injury.

In Cancer and Blood Disease, Elaine Fuchs, PhD, The Rockefeller University, will share findings on identification of skin cancer stem cells, which have implications in understanding other cancers as well as stem cells. Joanne Kurtzberg, MD, Duke University, will discuss her work developing therapies for disease with autologous cord blood transplants. Ravi Majeti, PhD, Stanford University, will describe his recent insights into acute myeloid leukemia and how stem cell technologies can lead to new cancer treatments.

Dieter Egli, PhD, The New York Stem Cell Foundation (NYSCF), will open the session on Diabetes and Autoimmunity by detailing his group's development of stem cell-derived models of pancreatic beta cells for the study of diabetes. Yuval Dor, PhD, Hebrew University, Israel, will discuss experiments with pancreatic beta cells with the goal to understand the regenerative potential of these cells. Matthias von Herrath, MD, Novo Nordisk, will delve into another aspect of Type 1 diabetes, the problem of autoimmunity. He will close the session by sharing insights into the need for an immune modulated therapy to diabetes.

Before the afternoon sessions, Shahin Rafii, MD, Weill Medical College of Cornell University will deliver the first of two keynote addresses of the conference. He will describe his recent successes in deriving vascular cells from amniotic cells.

In the afternoon session on Heart and Muscle Diseases, Amy Wagers, PhD, Harvard University, will focus on advances in treatments and explain how studies into the mechanisms of tissue stem cell renewal may have relevant therapeutic implications. Gordon Keller, PhD, McEwen Centre for Regenerative Medicine, Canada, will describe modeling cardiac cell development from human pluripotent cells for use in toxicology and electrophysiology studies. Helen Blau, PhD, Stanford University, will describe her research to improve stem cell culture in the direction of stem cell fate and for drug screens.

In Neurodegeneration and Spinal Cord Injury, Paola Arlotta, PhD, Harvard University and a NYSCF-Robertson Stem Cell Investigator, will address the application of stem cells to understanding and possibly treating these debilitating diseases and conditions, and will describe investigations to direct reprogramming of neurons into different neuronal lineages. Lorenz Studer, MD, Memorial Sloan-Kettering Cancer Center, will discuss the potential stem cell technology holds in the treatment of Parkinson's disease. Despite past failures in the replacement of lost dopamine neurons, Dr. Studer will describe his novel protocols for the generation of these neurons for eventual use in clinical trials.

Rudolf Jaenisch, MD, The Whitehead Institute, will deliver the second keynote address of the day. Building on Shinya Yamanaka's paradigm-changing work in induced pluripotent stem (iPS) cell reprogramming, Dr. Jaenisch will discuss new methods to counter the generally low successful output of these cells. He will also summarize how targeted genome editing may help unleash the potential of iPS cells and embryonic stem cells for both the study of and therapy for disease.

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Scientists discuss stem cell discoveries at New York Stem Cell Foundation Conference

Kyoto University Professor Shinya Yamanaka talks with Japan's Prime Minister Yoshihiko Nada by a mobile phone during a news conference in Kyoto, western Japan, in this photo taken by Kyodo October 8, 2012.

By Tan Ee Lyn Reuters Wednesday, Oct 10, 2012

HONG KONG - 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.

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Nobel laureate Yamanaka warns of rogue "stemcell therapies"

The techniques pioneered by the winners of this years Nobel Prize in medicine, John B. Gurdon and Shinya Yamanaka, have already allowed scientists to generate stem cells and clone animals.

But it is the potential these discoveries hold that truly boggles the mind. If stem cells the primitive cells that develop into tissue like skin, blood, nerves, muscle and bone can be harnessed, the belief is they can be used as a repair kit for the body.

In theory, a few skin cells could be harvested to rebuild a spinal cord damaged by trauma, to replace brain cells destroyed by dementia, to rebuild heart muscle damaged by a heart attack or to grow a new limb ravaged by diabetes. It is the stuff of science fiction, so close we can taste it.

But these dreams of miracle cures must be tempered with a strong dose of realism.

Despite billions of dollars in investment in research, from government agencies and biotech companies, there is little evidence that stem cell therapies work.

Yes, some hearing has been restored in gerbils and there have been modest improvements in paralyzed lab rats using stem cell treatments, but these are baby steps. In humans, the gains have been far more modest.

We can treat some forms of cancer, like leukemia and multiple myeloma, with stem cell transplants. But this is simply a refinement of an earlier technique, bone marrow transplant. And to perform such a transplant, the immune system must, for all intents and purposes, be destroyed a punishing regime with a significant mortality rate.

It is a far cry from the notion of an injection of magic stem cells that allow people to walk again or restore their memories.

The International Society for Stem Cell Research says that while there are hundreds of conditions that can purportedly be treated with stem cells, the treatments that have actually been shown to be beneficial are extremely limited. Aside from the cancer treatments mentioned above, some bone, skin and corneal conditions have been treated by grafting stem cells, growing them in the lab and transplanting them.

But in all these cases, the stem cells are tissue-specific, meaning the cells are carrying out a function they were designed to do. This is very different from the notion that undifferentiated stem cells can be used to treat a broad range of conditions.(And we wont delve into potential problems, such as rejection and the concern that stem cells could grow out of control and cause cancerous tumours.)

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Stem cell therapy a miracle cure? Not quite yet

ROCKVILLE, Md., Oct. 9, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE Amex: CUR) announced that Eva Feldman, MD, PhD, principal investigator of the Phase I trial to test Neuralstem's NSI-566 spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease), updated data on the trial at the American Neurological Association annual meeting in Boston, MA, yesterday. (http://www.aneuroa.org/i4a/pages/index.cfm?pageid=3311). Dr. Feldman, who is President of the American Neurological Association, presented interim results on all 18 procedures in 15 patients, including the last three patients from earlier cohorts who received second procedures. The trial will conclude six months after the last patient was treated, which was in August.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

"This has been a very successful trial so far," said Dr. Feldman, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. "With the transplantation of these neural stem cells, we are exploring a paradigm shift in the treatment of ALS. We have demonstrated that intraspinal transplantation is feasible and well-tolerated. Although this phase of the trial was not powered to demonstrate efficacy, we appear to have interrupted the progression of the disease in one subgroup of patients. We are anxious to move to future trial phases to examine therapeutic efficacy." Dr. Feldman is an unpaid consultant to Neuralstem.

"The purpose of this trial was to assess the safety of both the intraspinal transplantation procedure, the first in the world, and of the cells themselves, " said Karl Johe, PhD, Chairman of the Board and Chief Scientific Officer of Neuralstem, Inc. "All assessments show both to be safe. Additionally, we believe we are seeing evidence of a treatment effect in some patients over a sustained period of time. We need now to move forward to more advanced, larger trials to increase the dosage and more effectively look at possible efficacy."

About the Trial

The Phase I trial to assess the safety of Neuralstem's NSI-566 spinal cord neural stem cells and intraspinal transplantation method in ALS patients commenced in January 2010, and consisted of 18 treatments in 15 patients. The trial was designed to follow a risk escalation paradigm. The first 12 patients were each transplanted in the lumbar (lower back) region of the spine, beginning with non-ambulatory and advancing to ambulatory cohorts.

The trial then advanced to transplantation in the cervical (upper back) region of the spine. The first cohort of three was treated in the cervical region only. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously-treated patients to this cohort. Consequently, the last cohort of three patients received injections in the cervical region in addition to the lumbar injections they had received earlier. All injections delivered 100,000 cells, for a dosing range of up to 1.5 million cells. The last patient was treated in August, 2012. The entire trial concludes six months after the final surgery.

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem has recently treated the last patient in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in spinal cord injury.

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Dr. Eva Feldman, Principal Investigator, Updates Interim Data On Completed Neuralstem ALS Phase I Trial

STOCKHOLM: Scientists from Britain and Japan shared the Nobel Prize in Medicine on Monday for the discovery that adult cells can be reprogrammer back into stem cells which can turn into any kind of tissue and may one day repair damaged organs. John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos. They share the $1.2 million prize equally. These groundbreaking discoveries have completely changed our view of the development and specialization of cells, the Nobel Assembly at Stockholms Karolinska Institute said in a statement. The big hope for stem cells is that they can be used to replace damaged tissues in everything from spinal cord injuries to Parkinsons disease. All of the tissue in the body starts as stem cells, before developing into mature skin, blood, nerves, muscle and bone. Scientists once thought it was impossible to turn adult tissue back into stem cells, which meant that new stem cells could only be created by harvesting embryos. But Yamanaka and Gurdon showed that development can be reversed, turning adult cells back into cells that behave like embryos. With induced pluripotency stem cells, or iPS cells, ordinary skin or blood cells from adults are transformed back into stem cells which doctors hope will be able to repair damaged organs without being rejected by the immune system. There are concerns, however, that iPS cells could grow out of control and develop into tumors. The eventual aim is to provide replacement cells of all kinds, Gurdons Institute explains on its website. We would like to be able to find a way of obtaining spare heart or brain cells from skin or blood cells. The important point is that the replacement cells need to be from the same individual, to avoid problems of rejection and hence of the need for immunosuppression. Gurdon discovered in 1962 that the specialization of cells could be reversed. In what the prize committee called a classic experiment, he replaced the immature cell nucleus in an egg cell of a frog with the nucleus from a mature intestinal cell. This modified egg cell developed into a normal tadpole, proving that the mature cell still had all the information needed to develop all cells in the frog. More than 40 years later, in 2006, Yamanaka discovered how intact mature cells in mice could be reprogrammer to become stem cells by adding just a few genes. Thanks to these two scientists, we know now that development is not strictly a one-way street, said Thomas Perlmann, Nobel Committee member and professor of Molecular Development Biology at the Karolinska Institute. There is lot of promise and excitement, and difficult disorders such as neurodegenerative disorders, like perhaps Alzheimers and, more likely, Parkinsons disease, are very interesting targets.

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Japan, UK scientists win Nobel for stem cell breakthroughs

Yamanaka and Gurdon

Two sets of experiments, performed 40 years apart, have been recognized with today's Nobel Prize in Physiology or Medicine. Cambridge University's John Gurdon won for showing that adult cells contain all the genetic information necessary to create every tissue in the body. That work set the stage for Shinya Yamanaka, who demonstrated that a relatively simple process could convert adult cells into embryonic stem cells. That development is already opening new avenues of research, and it holds the promise of new ways to repair tissues damaged by injury or disease.

As an embryo develops from a single fertilized egg, its cells become increasingly specialized. Although the initial cells can form any tissue in the body, groups of them adopt specific fates. A cell might first commit to being a neuron, after which it may be further limited to the roles required in the spinal cord, before finally specializing in the activities needed to control muscles. What doesn't seem to happen, however, is for the cell to switch developmental tracksdeveloping as, for example, a liver cell.

The apparent permanence of these fate decisions left most researchers thinking that they were in fact permanentthat the genomes of the cells undergo irreversible changes. At least in the case of immune cells, that seemed to be true: as part of generating the ability to recognize a diverse array of threats, B and T cells delete large stretches of their DNA and irreversibly commit themselves to recognizing a single threat.

But it's not true of all cells. John Gurdon performed key experiments back in the 1960s that showed how most cells maintain their general capacity to develop in any direction, although it took decades for the significance of his work to be fully appreciated. Using the eggs of a frog, Gurdon carefully removed the nucleus, which contains its genome. He then transferred in the nucleus of a specialized cell from an adult frog. If the general perception turned out to be correct, the DNA from that cell should have been permanently committed to its fate (in this case, intestine). Instead, Gurdon was able to get the hybrid cell to develop into a tadpole and, eventually, a healthy adult.

These results clearly demonstrated that adult cells contain all the genomic ingredients to make every cell in an organism. But it took time to develop the technology that took advantage of the fact. A key step in that development was honored by the Nobel Committee in 2007: the development of embryonic stem cells derived from mice. These cells, derived from early embryos, could divide indefinitely in culture without adopting any particular fate, but given the right chemical nudges, could form any type of adult cell. If injected into an early embryo, they would go on to contribute to every tissueincluding the germ cells, which allowed these cells to go from a culture dish to future generations of mice.

This work led to the controversial development of human embryonic stem cells. But it also allowed people to ask what makes an embryonic stem cell distinct. Over time, scientists created a list of a few dozen genes that were consistently active in stem cells of various types. Some of these would undoubtedly be a consequence of the cells' stem-cell-ness. But others would be responsible for putting the cells there in the first place.

Shinya Yamanaka, an MD who says he got into research because he wasn't any good at surgery, decided to find out which of this list of genes was likely to be in control. Starting with about 20 known regulatory genes on the list, he inserted groups of them into adult cells, seeing which sets could turn them into a stem cell. By process of elimination, he gradually whittled that list down to just four genes. Inserting them into an adult cell would force it to get rid of any specializations and go on to adopt a stem cell fate. Once that was done, the cells could then be induced to form any type of adult cell in culture, or be injected into an embryo and contribute to an adult.

Stem cell work in general has raised the prospect that we could repair injured or damaged tissue with newly generated cells that are just as specialized as the ones they are replacing. But Yamanaka's work has turned that prospect into a vision of on-demand tissues, generated with a simple lab procedure, and a perfect genetic match for their recipient. The cells produced with the procedure he pioneered don't seem to be an exact match for cells derived from embryos, but it appears that they may be close enough that the difference doesn't matter.

It might be hard to imagine that research could take 40 years to come to fruition. But it's widely accepted that Gurdon's work fostered a change in perspective that was necessary for people to even start thinking about the studies that eventually led to stem cell manipulations. A year ago, I spoke to Martin Evans, who was a co-winner of the 2007 prize for stem cells, and he was already describing a long line of developments that led from Gurton through his own work and that of others, and that eventually culminated in Yamanaka's experiments. Two years ago, Gurdon and Yamanaka were honored with a Lasker Prize, which often precedes Nobel status.

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A not-so-simple twist of fate: Nobel awarded for stem cell reprogramming

10/5/2012 6:38 AM ET (RTTNews) - Amyotrophic Lateral Sclerosis is a progressive neurodegenerative disease that attacks nerve cells in the brain and spinal cord, leading to complete paralysis, and eventually, death. Also known as Lou Gehrig's disease, Amyotrophic Lateral Sclerosis, or ALS, is said to affect as many as 30,000 Americans, with 5,600 new cases being diagnosed each year.

Currently, there are two FDA-approved drugs to treat ALS namely, Sanofi-Aventis' (SNY:Quote) Riluzole, which prolongs life by 2-3 months, and Avanir Pharmaceuticals Inc.'s (AVNR:Quote) Nuedexta, which treats emotional instability that accompanies this disease.

Developing a neural stem cell therapy for ALS is Rockville, Maryland-based biotechnology company Neuralstem Inc. (CUR:Quote).

For readers who are new to Neuralstem, here's a brief overview of the company's pipeline and the upcoming events to watch out for...

The company is testing its cell product - NSI-566 human spinal cord stem cells, via transplantation technique, in the treatment of ALS symptoms. The phase I NSI-566 study was completed as recently as August of this year. This groundbreaking trial, the first to be approved by the FDA to test neural stem cells in patients with ALS, began in January 2010.

The trial was designed to enroll up to 18 patients, the last of which was treated in August of this year. The entire trial concludes six months after the final surgery.

The interim data on the NSI-566 ALS trial will be updated on October 8, 2012, according to the company.

NSI-566 will also be evaluated in treating motor deficits due to ischemic stroke. The company has received approval to commence a combined phase I/II ischemic stroke trial with NSI-566 in China, and it is expected to begin early next year.

The trial is designed to test up to 118 patients who have suffered an ischemic stroke with chronic residual motor disorder with NSI-566 cell line, 4-24 months post-stroke. The duration of the combined trial, including patient monitoring and data collection, is approximately two years.

Ischemic strokes, the most common type of stroke, occur as a result of an obstruction within a blood vessel supplying blood to the brain. After a stroke, many patients suffer from paralysis in arms and legs, which can be permanent.

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Walkthrough With Neuralstem

NEWARK, Calif., Oct. 4, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the first patient in its Phase I/II clinical trial in dry age-related macular degeneration (AMD) has been enrolled and transplanted. The trial is designed to evaluate the safety and preliminary efficacy of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) as a treatment for dry AMD, and the patient was transplanted with the cells yesterday at the Retina Foundation of the Southwest (RFSW) in Dallas, Texas, one of the leading independent vision research centers in the United States. AMD afflicts approximately 30 million people worldwide and is the leading cause of vision loss and blindness in people over 55 years of age.

"This trial signifies an exciting extension of our on-going clinical research with neural stem cells from disorders of the brain and spinal cord to now include the eye," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Studies in the relevant animal model demonstrate that the Company's neural stem cells preserve vision in animals that would otherwise go blind and support the therapeutic potential of the cells to halt retinal degeneration. Unlike others in the field, we are looking to intervene early in the course of the disease with the goal of preserving visual function before it is lost."

David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory and principal investigator of the study, added, "We are excited to be working with StemCells on this ground breaking clinical trial. There currently are no effective treatments for dry AMD, which is the most common form of the disease, and there is a clear need to explore novel therapeutic approaches."

In February 2012, the Company published preclinical data that demonstrated HuCNS-SC cells protect host photoreceptors and preserve vision in the Royal College of Surgeons (RCS) rat, a well-established animal model of retinal disease which has been used extensively to evaluate potential cell therapies. Moreover, the number of cone photoreceptors, which are responsible for central vision, remained constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed in the study. In humans, degeneration of the cone photoreceptors accounts for the unique pattern of vision loss in dry AMD. The data was published in the international peer-reviewed European Journal of Neuroscience.

About Age-Related Macular Degeneration

Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s, and progresses painlessly, gradually destroying central vision. According to the RFSW website, there are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the United States with more than seven million at risk of developing AMD.

About the Trial

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial should contact the site at (214) 363-3911.

About HuCNS-SC Cells

StemCells' proprietary product candidate, HuCNS-SC cells, is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system (CNS) with no sign of tumor formation or adverse effects. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation. StemCells believes that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS, and to date has demonstrated human safety data from completed and ongoing clinical studies.

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StemCells, Inc. Announces First Transplant of Neural Stem Cells Into Patient in Clinical Trial for Dry Age-Related ...

ROCKVILLE, Md., Oct.4, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE Amex: CUR) announced that Eva Feldman, MD, PhD, principal investigator of the Phase Itrial to test Neuralstem's human spinal cord stem cells, NSI-566, in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease), will update trial data at the American Neurological Association annual meeting on Monday, October 8th (http://www.aneuroa.org/i4a/pages/index.cfm?pageid=3311). Dr. Feldman's poster presentation, "Completion and Outcomes of Phase I Intraspinal Stem Cell Transplantation Trial for ALS," will be up from 11:30-6:30. Dr. Feldman will be discussing the data between 5:30-6:30.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

Dr. Feldman is the President of the American Neurological Association, as well as Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. Dr. Feldman is an unpaid consultant to Neuralstem.

About the Trial

The Phase I trial to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in ALS patients commenced in January 2010, and consisted of 18 treatments in 15 patients. The trial was designed to follow a risk escalation paradigm. The first 12 patients were each transplanted in the lumbar (lower back) region of the spine, beginning with non-ambulatory and advancing to ambulatory cohorts.

The trial then advanced to transplantation in the cervical (upper back) region of the spine. The first cohort of three was treated in the cervical region only. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously-treated patients to this cohort. Consequently, the last cohort of three patients received injections in the cervical region in addition to the lumbar injections they had received earlier. All injections delivered 100,000 cells, for a dosing range of up to 1.5 million cells. The last patient was treated in August, 2012. The entire trial concludes six months after the final surgery.

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem has recently completed an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic spastic paraplegia and chronic stroke. The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in spinal cord injury.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company is in a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD).Additional indications could include chronic traumatic encephalopathy (CTE), Alzheimer's disease, and post-traumatic stress disorder (PTSD).

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Dr. Eva Feldman, Principal Investigator, To Update Interim Data On Neuralstem ALS Trial

NEWARK, Calif., Sept. 27, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the first patient with an incomplete spinal cord injury has been enrolled in the Company's Phase I/II clinical trial in chronic spinal cord injury and transplanted with the Company's proprietary HuCNS-SC(R) neural stem cells. The patient, a Canadian man who suffered a thoracic spinal cord injury from a sports-related accident, was administered the cells yesterday at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation. This is the first patient in the second cohort of the trial, which will be comprised of four patients who retain some sensory function below the level of trauma and are therefore considered to have an incomplete injury.

"This is an important milestone for StemCells and the spinal cord injury community as it is the first time anyone has ever transplanted neural stem cells into a patient with an incomplete injury," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Given the encouraging interim data from the most severely injured patient cohort that we reported earlier this month, testing patients with less severe injury should afford us an even better opportunity to continue to test safety and to detect and assess clinical changes. Unlike the patients in the first cohort, patients with incomplete injuries have retained a degree of spinal cord function that might be even further augmented by transplantation with neural stem cells."

Earlier this month, the Company reported that interim six-month data from the first patient cohort in the Phase I/II clinical trial continued to demonstrate a favorable safety profile, and showed considerable gains in sensory function in two of the three patients compared to pre-transplant baselines. Patients in the first cohort all suffered a complete injury to their spinal cord, leaving them with no neurological function below the level of injury. Following transplantation with HuCNS-SC cells, there were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first six months after transplantation. Changes in sensitivity to touch, heat and electrical stimuli were observed in well-defined and consistent areas below the level of injury in two of the patients, while the third patient remained stable. Importantly, the changes in sensory function were confirmed objectively by measures of electrical impulse transmission across the site of injury, each of which correlated with the clinical examination.

About the Spinal Cord Injury Clinical Trial

The Phase I/II clinical trial of StemCells, Inc.'s HuCNS-SC(R) purified human adult neural stem cells is designed to assess both safety and preliminary efficacy. Twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level are planned for enrollment, and their injuries must have occurred within three to twelve months prior to transplantation of the cells. In addition to assessing safety, the trial will assess preliminary efficacy based on defined clinical endpoints, such as changes in sensation, motor function and bowel/bladder function. The Company has dosed the first patient cohort, all of whom have injuries classified as AIS A according to the American Spinal Injury Association Impairment Scale (AIS). In AIS A injuries, there is no neurological function below the injury level. The second cohort will be patients classified as AIS B, in which there is some preservation of sensory or motor function below the injury level. The third cohort will be patients classified as AIS C, in which there is some preservation of both sensory and motor function.

All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord and will be temporarily immunosuppressed. Patients will be evaluated regularly in the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any recovery of neurological function below the injury site. The Company intends to follow the effects of this therapy long-term, and each of the patients will be invited to enroll into a separate four year observational study after completing the Phase I/II study.

The trial is being conducted at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation, and is open for enrollment to patients in Europe, Canada and the United States. Enrollment for the second cohort is now underway. If you believe you may qualify and are interested in participating in the study, please contact the study nurse either by phone at +41 44 386 39 01 or by email at stemcells.pz@balgrist.ch.

Additional information about the Company's spinal cord injury program can be found on the StemCells, Inc. website at http://www.stemcellsinc.com/Therapeutic-Programs/Clinical-Trials.htm and at http://www.stemcellsinc.com/Therapeutic-Programs/Spinal-Cord-Injury.htm, including video interviews with Company executives and independent collaborators.

About Balgrist University Hospital

Balgrist University Hospital, University of Zurich is recognized worldwide as a highly specialized center of excellence providing examination, treatment and rehabilitation opportunities to patients with serious musculoskeletal conditions. The clinic owes its leading international reputation to its unique combination of specialized medical services. The hospital's carefully-balanced, interdisciplinary network brings together under one roof medical specialties including orthopedics, paraplegiology, radiology, anesthesiology, rheumatology, and physical medicine. More information about Balgrist University Hospital is available at http://www.balgrist.ch.

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StemCells, Inc. Achieves Spinal Cord Injury Milestone With First Neural Stem Cell Transplant Into Patient With Sensory ...

ROCKVILLE, Md., Sept. 27, 2012 /PRNewswire/ -- Neuralstem, Inc. (NYSE MKT: CUR) announced it has been approved to commence a clinical trial to treat motor deficits due to ischemic stroke with its spinal cord stem cells (NSI-566) at BaYi Brain Hospital, in Beijing, China, through its subsidiary, Neuralstem China (Suzhou Neuralstem Biopharmaceutical Company, Ltd.). The trial approval includes a combined phase I/II design and will test direct injections into the brain of NSI-566, the same cell product tested by Neuralstem in a recently-completed Phase IALS trial in the United States. The trial is expected to begin early next year. Ischemic strokes, the most common type of stroke, occur as a result of an obstruction within a blood vessel supplying blood to the brain. Post-stroke motor deficits include paralysis in arms and legs and can be permanent.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

"China is rapidly moving to become a prominent player in the field of stem cell transplantation and regenerative medicine," said Karl Johe PhD, Chairman of Neuralstem's Board of Directors and Chief Scientific Officer. "We are honored and excited to begin this process at BaYi Brain Hospital in Beijing, China. BaYi is one of the premier neurological hospitals in China, and we can assure all of our stake holders that the quality of the medical care and treatment our patients receive, as well as the entire protocol, will be the equal of any trial we do anywhere in the world, including our FDA-approved trial in the U.S."

"BaYi prides itself on its world class research capabilities," said Professor Xu RuXiang, President of the BaYi Hospital and principal investigator of the trial. "Chronic motor disorder from stroke is a serious public health issue for China, where we, as a population, now suffer over 2 million ischemic strokes per year.We are ready to be the world's first site for Neuralstem's cell therapy stroke trial and excited about its potential."

About the Trial

The trial is designed to enroll up to 118 patients who have suffered an ischemic stroke with chronic residual motor disorder with Neuralstem's NSI-566 cell line, 4-24 months post-stroke. The stem cell treatment involves a one-time treatment of intracerebral injections of Neuralstem's neural stem cells into the stroke area using well-accepted stereotactic injection procedures. The trial will be conducted in 2 parts. The first part of the study, Phase I, will be open-label and enroll up to 18 patients who will be assigned to 3 cohorts. Each of these will receive ascending doses of NSI-566 to define the maximal safe dose. The maximal safe dose defined in the first phase will be used to evaluate efficacy in the second part of the study, a Phase II/Proof-of-Concept study. This Phase will be a multi-site, randomized, controlled, single-blind study and enroll up to 100 randomized subjects. 50% of the subjects will receive a one-time treatment with the cells and physical therapy and the other 50% will receive only the physical therapy with no surgery. Outcome measures during the follow-up period in Phase II will be conducted in single-blinded manner. The combined study, including patient monitoring and data collection, is expected to take approximately two years.

About Neuralstem China

Founded in 2010, Neuralstem China (Suzhou Sun-Now Biopharmaceutical Co. Ltd. is a wholly-owned subsidiary of Neuralstem, Inc., headquartered in Suzhou, roughly one hundred miles west of Shanghai. Neuralstem China has constructed a clinical-grade manufacturing facility), and holds the license required by the Chinese government for conducting business in China. Neuralstem's mission in China is to bring its world-leading neural stem cell technology to China to address the unmet medical needs of tens of millions of patients suffering from diseases of, and injuries to, the central nervous system (CNS).

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Neuralstem Approved To Commence Ischemic Stroke Trial In China