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.

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"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.

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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).

Excerpt from:
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.

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"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

SAN DIEGO UC San Diego researchers have used early stage stem cells to restore movement in rats that had suffered severe spinal cord injuries. Researchers say the stem cells in essence rewired the spinal cord.

Previous studies showed stem cells implanted at the site of a spinal cord injury didn't survive for long.

In this study, researchers embedded the stem cells in a gel. That allowed the cells to stick to and completely fill the injury site. Scientists also added a cocktail of growth factors.

UC San Diego's Mark Tuszynski, the study's principal investigator, said the technique generated an enormous amount of new neurons.

"That amount of growth, and the ability of this to improve function after the most severe spinal cord injury, moves this into the realm of potential human translation," Tuszynski explained.

Tuzynski said after the therapy, rats were able to move their hind legs again, but not at full strength.

Paul Lu, assistant research scientist at UC San Diego's Center for Neural Repair, contributed to the study.

Their research is published in the journal Cell.

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UC San Diego Researchers Use Stem Cells To Restore Mobility

The laboratory grew and stored human stem cells, which are capable of becoming any cell in the body, and made them available to scientists nationwide for use in experiments to study diseases such as diabetes and spinal cord injuries. When it is dismantled, several thousand vials of stem cellswill be sent back to the research centers where they originated, and the equipment will be given to other UMass labs.

Susan Windham-Bannister, president of the Massachusetts Life Sciences Center, a quasi-public agency that oversees the $1 billion life sciences initiative, defended the decision to initially fund the stem cell bank. She said there are many examples of technology that in hindsight are unnecessary, but at the time it was conceived, when the investment was made, it was absolutely state of the art. The center, she said, was one of them.

Originally, the bank was seen as a repository for embryonic stem cell lines that were being created but were not eligible for federal funding under Bush-era restrictions. The field has evolved significantly since then, with President Obamas loosening of restrictions on federal funding and the development of new technologies for making stem cells.

Still, stem cell banks are seen as useful by some. The California Institute for Regenerative Medicine, for example, is preparing to invest $10 million in its own stem cell banking initiative, and another $20 million to underwrite the creation of stem cells from patients with specific diseases.

Massachusetts Senate minority leader Bruce Tarr, Republican of Gloucester, said he was concerned that lawmakers had not been told the bank would close.

Given the fact that this is a resource that was created by an act of the Legislature, I would hope anyone seeking to change its status would consult with the Legislature, he said. The notion has always been we have been working hard to make Massachusetts a leader in stem cell research, and I dont know how ceasing the operations of the stem cell bank advances that goal.

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UMass stem cell lab to close

28.06.2012 - (idw) Ruhr-Universitt Bochum

RUB biologists have deliberately transformed stem cells from the spinal cord of mice into immature nerve cells. This was achieved by changing the cellular environment, known as the extracellular matrix, using the substance sodium chlorate. Via sugar side chains, the extracellular matrix determines which cell type a stem cell can generate. Influencing precursor cells pharmacologically so that they transform into a particular type of cell can help in cell replacement therapies in future says Prof. Dr. Stefan Wiese, head of the Molecular Cell Biology work group. Matrix modified Taking the fate of stem cells in hand RUB researchers generate immature nerve cells

RUB biologists have deliberately transformed stem cells from the spinal cord of mice into immature nerve cells. This was achieved by changing the cellular environment, known as the extracellular matrix, using the substance sodium chlorate. Via sugar side chains, the extracellular matrix determines which cell type a stem cell can generate. Influencing precursor cells pharmacologically so that they transform into a particular type of cell can help in cell replacement therapies in future says Prof. Dr. Stefan Wiese, head of the Molecular Cell Biology work group. Therapies, for example, for Parkinsons, multiple sclerosis or amyotrophic lateral sclerosis could then become more efficient. The team describes its findings in Neural Development.

Sulphate determines the fate of stem cells

Sodium chlorate acts on metabolism enzymes in the cell which attach sulphate groups to proteins. If these sulphates are not installed, the cell continues to form proteins for the extracellular matrix, but with modified sugar side chains. These chains in turn send out signals that define the fate of the stem cells. Stem cells can not only develop into nerve cells, but also form astrocytes or oligodendrocytes, which are, for instance, responsible for the mineral balance of the nerve cells or which form their insulation layer. What happens to the stem cells if the sulphate pattern is changed by sodium chlorate was examined by Dr. Michael Karus and his colleagues.

The RUB-laboratories of Prof. Dr. Stefan Wiese, Prof. Dr. Andreas Faissner and Prof. Dr. Irmgard Dietzel-Meyer collaborated for the study. Using antibodies, the researchers showed that cells which they had treated with sodium chlorate developed into nerve cells. They also analysed the flow of sodium ions into the cells. The result: treated cells showed a lower sodium current than mature nerve cells. Sodium chlorate thus favours the development of stem cells into nerve cells, but, at the same time, also inhibits the maturation - a positive side effect, as Wiese explains: If sodium chlorate stops the nerve cells in an early developmental phase, this could enable them to integrate into the nervous system following a transplant better than mature nerve cells would do.

Bibliographic record

M. Karus, S. Samtleben, C. Busse, T. Tsai, I.D. Dietzel, A. Faissner, S. Wiese (2012): Normal sulphation levels regulate spinal cord neural precursor cell proliferation and differentiation, Neural Development, doi:10.1186/1749-8104-7-20

Further information

Prof. Dr. Stefan Wiese, Molecular Cell Biology Work Group, Faculty of Biology and Biotechnology at the Ruhr-Universitt, 44780 Bochum, Germany, Tel. +49/234/32-22041 stefan.wiese@rub.de

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Taking the fate of stem cells in hand: RUB researchers generate immature nerve cells

Local orthopedic physician treated patient who was featured on True Life

BY JENNIFER AMATO

Staff Writer

Dr. Edward Magaziner NORTH BRUNSWICK A young woman named Tamara was suffering from chronic pelvic pain that was affecting her everyday life.

She had undergone two surgeries on her hips, since doctors determined the pain was the result of friction within her hip joints, or impingement that would cause the throbbing pain .

Having even more pain after her second surgery, she visited Dr. Edward Magaziner, medical director of The Center for Spine, Sports, Pain Management and Orthopedic Regenerative Medicine in North Brunswick, to undergo a process called prolotherapy.

Tamara was first filmed for MTVs True Life in 2010 and was featured in the Then and Now follow-up, which debuted on May 17.

In his practice, Magaziner treats spinal, joint, muscle and nerve pain and headaches. Pain management treatments include acupuncture, Botox injections, epidural injections, mesotherapy, trigger-point injections, therapeutic laser and spinal cord simulation. He also performs minimally invasive spinal surgery using endoscopic laserassisted devices.

The more state-of-the-art treatments are bio-regenerative, such as prolotherapy and platelet-rich protein (PRP), said Magaziner. They are effective, he said, because increased blood flow to an injured part of the body is a natural form of healing; concentrating the healing properties of the blood, such as stem cells and growth factors, at the direct site of an injury will enhance healing effects.

The body heals itself naturally when its injured. When there is an injury, theres a biological and chemical signal that is produced at the cellular level where the injury is, to attract platelets and growth factors and stem cells to the area. With a combination of these factors, the body has the ability to heal the injury, said Magaziner, a diplomate of the American Academy of Pain Management.

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North Brunswick doctor appears on MTV show

ROCKVILLE, Md., June 25, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the first patients were dosed in Phase Ib of its ongoing trial to test the safety of NSI-189 in the treatment of major depressive disorder (MDD). NSI-189, the lead compound in Neuralstem's small molecule platform, is a proprietary new chemical entity that stimulates new neuron growth in the hippocampus, a region of the brain believed to be implicated in MDD as well as other diseases and conditions, such as chronic traumatic encephalopathy (CTE), Alzheimer's disease, anxiety, and post-traumatic stress disorder (PTSD). This is the first time the drug will be tested in patients with MDD, as Phase Ia was in healthy volunteers.

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"We are pleased to begin testing the safety of NSI-189 in depression patients," said Karl Johe, PhD, Neuralstem's Board of Directors and Chief Scientific Officer. "We believe it could help patients who suffer from depression via a new mechanism that does not seek to modulate brain chemistry, but rather stimulates new neuron growth in the hippocampus and increases hippocampal volume, thereby potentially addressing the problem at the source."

About NSI-189 Neuralstem's technology enables the creation of neural stem cell lines from many areas of the human CNS, including the hippocampus. The hippocampus is a part of the brain involved in memory and the generation of new neurons. It is also implicated in several major neurological and psychiatric diseases. From its hippocampal neural stem cell lines, Neuralstem has created virtually unlimited amounts of mature human neurons and glia in laboratory dishes. These can be used to mimic the natural brain environment in order to test drug effects.

Neuralstem has been engaged in a drug discovery program with these human hippocampal stem cell lines since 2000. In 2009, Neuralstem was granted U.S. patents on four first-in-class chemical entities that boost the generation of new neurons. NSI-189, the first of these to be in a clinical trial, significantly stimulates the generation of new hippocampal neurons (neurogenesis) in vitro and in animal models.

NSI-189 is the lead compound in Neuralstem's neurogenic small molecule drug platform, which the company plans to develop into orally administered drugs for MDD and other psychiatric and cognitive disorders as diverse as CTE, Alzheimer's disease, anxiety, and PTSD.

NSI-189 has been shown to stimulate neurogenesis of human hippocampus-derived neural stem cells in-vitro and in vivo. In healthy normal adult mice, NSI-189 stimulated neurogenesis in the hippocampus and significantly increased its volume, apparently by increasing its synaptic network after 28 days of daily oral administration. In mouse models of depression, NSI-189 significantly improved behavioral responses associated with depression. In humans, NSI-189 may reverse the human hippocampal atrophy seen in MDD and other disorders and reverse their symptoms. This program has received significant support from both the Defense Advanced Research Projects Agency (DARPA) and the National Institutes of Health (NIH).

About the Trial The NSI-189/MDD trial is a randomized, double-blind, placebo-controlled, multiple-dose escalating trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamic effect of NSI-189 in the treatment of MDD. Phase Ia tested escalating doses of single administration of NSI-189 in healthy patients. Phase Ib is testing the safety of escalating doses of NSI-189 for 28 daily administrations in 24 depressed patients. The Phase Ib portion of the trial is expected to take approximately six months to complete.

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 is 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 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 chronic spinal cord injury.

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First Patients Dosed in Ib Phase of Neuralstem's NSI-189 Trial in Major Depressive Disorder

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Some of the automated sampling equipment in the Rensselaer Stem Cell Research Center in Troy. Some of the automated sampling equipment in the Rensselaer Stem Cell Research Center in Troy. (Mike McMahon / The Record)

By Danielle Sanzone dsanzone@troyrecord.com Twitter.com/DanielleSanzone

State Department of Health Commissioner Nirav Shah, left, and Rensselaer Polytechnic Institute President Dr. Shirley Ann Jackson, right, announce the opening of the Rensselaer Center for Stem Cell Research during a forum at the colleges Troy campus Friday. (Mike McMahon / The Record)

TROY During a Rensselaer Polytechnic Institute forum on Friday, dozens were able to see their first baby picture: a single cell that eventually multiplied, in part due to stem cells, into an organism with trillions of cells.

That, to me, is the most amazing thing in the study of biology, said Glenn Monastersky, director of the Rensselaer Center for Stem Cell Research.

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VIDEO: Stem cell research facility to open at Rensselaer Polytechnic Institute

NEWARK, Calif., June 21, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced initiation of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) referred to as Geographic Atrophy. There are no approved treatments for dry AMD.

The trial is being conducted at the Retina Foundation of the Southwest's (RFSW) Anderson Vision Research Center in Dallas, Texas, one of the leading independent vision research centers in the United States. David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, is the principal investigator of the study.

"Dry AMD is the most common form of macular degeneration, and has a very debilitating effect on quality of life," said Dr. Birch. "Transplanting neural stem cells to protect photoreceptors in patients diagnosed with AMD is an innovative, but logical, approach, well supported by the Company's recently published preclinical data. We are very excited to be conducting this trial at RFSW."

A summary of the Company's preclinical data was featured in the February 2012 issue of the international peer-reviewed European Journal of Neuroscience (available online at http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract). The data demonstrated that 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. Transplantation of HuCNS-SC cells significantly protects photoreceptors from degeneration. 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.

"Unlike others in the field, our clinical strategy is to preserve visual function before it is lost," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Our published preclinical data provides a strong rationale for this approach in dry AMD and we hope to replicate these results in this clinical trial. We are very pleased to be working with Dr. Birch and the Retina Foundation of the Southwest, who have the expertise and referral base to undertake this important study. We anticipate that we will be able to accrue the requisite number of patients for this trial in relatively short order."

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 US 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

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StemCells, Inc. Initiates Phase I/II Clinical Trial in Dry Age-Related Macular Degeneration

By Jason Napodano, CFA

Neuralstem, Inc. (NYSE MKT: CUR ) has developed a technology that allows large-scale expansion of human neural stem cells ("hNSC") from all areas of the developing human brain and spinal cord. The company owns of has exclusive license to 25 patients and 29 patent applications pending worldwide in the field of regenerative medicine and cell therapy. Management is currently focusing the company's efforts on replacing damaged, malfunctioning, or dead neural cells with fully functional ones that may be useful in treating many central nervous system diseases and neurodegenerative disorders.

Neuralstem's lead development program is for Amyotrophic Lateral Sclerosis ("ALS"), also known as Lou Gehrig 's disease, named after the famous New York Yankee first baseman who was diagnosed with the disease in 1939, and passed in 1941 at the age of only 37.

ALS Background

ALS is a rapidly progressive neurodegenerative disease characterized by weakness, muscle atrophy and twitching, spasticity, dysarthria (difficulty speaking), dysphagia (difficulty swallowing), and respiratory compromise. The disease is almost always fatal, typically due to respiratory compromise or pneumonia, in two to four years. Initial symptoms of ALS include weakness and/or stiffness followed by muscle atrophy in the arms and legs. This is followed by slurred speech or difficulty swallowing, and loss of tongue mobility. Approximately a third of ALS patients also experience pseudobulbar affect (uncontrollable emotions). As the disease progresses, worsening dysphagia and respiratory failure leads to death. A small percentage of patients may also experience cognitive affects such as frontotemporal dementia and anxiety.

The vast majority (~95%) of cases are idiopathic, although there is a known hereditary factor that leads to familial ALS associated with a defect on the 21st chromosome that accounts for approximately 1.5% of all cases. There are also suspected environmental causative factors, including exposure to a dietary neurotoxin called BMAA and cyanobacteria, and use of pesticides. However, in all cases, the defining factor of ALS is rapid and progressive death of upper and lower motor neurons in the motor cortex of the brain, brain stem, and spinal cord. Prior to their destruction, motor neurons develop proteinaceous inclusions in their cell bodies and axons. This may be partly due to defects in protein degradation.

Treatment for ALS is limited, and as of today only riluzole, marketed by Sanofi-Aventis as Rilutek, has been found to improve survival to a modest extent (several months). Riluzole preferentially blocks TTX-sensitive sodium channels, which are associated with damaged neurons. This reduces influx of calcium ions and indirectly prevents stimulation of glutamate receptors. Together with direct glutamate receptor blockade, the effect of the neurotransmitter glutamate on motor neurons is greatly reduced. Riluzole does not reverse the damage already done to motor neurons, and people taking it must be monitored for liver damaged (about 10% incidence).

The remaining treatments for ALS are designed to relieve symptoms and improve quality of life. This supportive care includes a multidisciplinary approach that may include medications to reduce fatigue, control spasticity, reduce excess saliva and phlegm, limit sleep disturbances, reduce depression, and limit constipation. As noted above, median survival is two to four years. In the U.S., approximately 30,000 persons are currently living with ALS.

Neuralstem's Approach For ALS

Neuralstem is seeking to treat the symptoms of ALS via transplantation of its hNSCs directly into the gray matter of the patient's spinal cord. In ALS, motor neurons die, leading to paralysis. In preclinical animal work, Neuralstem cells both made synaptic contact with the host motor neurons and expressed neurotrophic growth factors, which are protective of cells.

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Neuralstem Pioneering Efforts In ALS - Analyst Blog