As of now, management is planning to conduct the pivotal program on its own, mostly likely seeking funding through grants with the ALS Association and U.S. National Institutes of Health. However, management is also in discussion with potential pharmaceutical partners on the pivotal program. ALS is a highly attractive area for Big Pharma. Depending on the strength of the phase 1 / 2 data, Neuralstem may be able to strike a commercialization partnership in 2014 to help defer the costs of the planned pivotal trial. We expect that any deal with a larger pharmaceutical company would include a substantial upfront payment that Neuralstem would then use to fund expansion of the development platform into new indications, such as spinal cord injury (IND filed) or stroke.

Market Opportunity

In February 2011, the U.S. FDA granted Neuralstem an Orphan Drug designation for its human spinal cord stem cells (HSSC) for the treatment of ALS. As noted above, there are approximately 30,000 patients in the U.S. living with ALS. We estimate that approximately half of these patients are characterized with an FVC > 60% and may be eligible for treatment with Neuralstems hNSCs. Given the Orphan Drug designation, the limited patient population, and the lack of any meaningful treatment options, we think Neuralstem or its commercialization partner could price this therapy at upwards of $100,000. Therefore, the peak market opportunity for Neuralstem is $1.5 billion.

That being said, drug development in ALS has been a graveyard for pharmaceutical companies. One would assume, based on numerous past clinical failures, that Neuralstems chances in ALS are slim. Small molecules including gabapentin, topiramate, celecoxib, tamoxifen, indinavir, minocycline, and xaliproden, many of which are approved for other indications and have posted annual sales over a billion dollars, have all failed human clinical programs for ALS. Even Vitamin E and Creatine have been tested, to little avail, in ALS. Failed mechanisms of action included calcium channel blockers, glutamate regulators, neuroprotectants, immunosuppressants, GABA receptors, anti-inflammatory agents, and antioxidants.

However, there is one thing in common we see in all of the above failures. They are one molecule targeting one mechanism of action or one pathway. ALS is a high complex and largely uncharacterized disease. Neuralstems approach uses human spinal stem cells that, once injected, can provide multiple mechanisms of action on multiple pathways to affect the disease. Plus, Neuralstems approach is highly targeted, with the cells injected directly into the lumbar or cervical spine. Following grafting, the hypothesis is that the cells rebuild circuitry with the patient motor neurons and protect existing neurons from further degradation. Its clearly a unique approach, and one we believe has a better chance of success than many of the previous failed theories enacted over the past decade.

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CUR - Neuralstem Pioneering Efforts In ALS

ROCKVILLE, Md., June 19, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the first patient to receive stem cell transplantation in both regions of the spinal cord has been treated in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). This is also the 16th patient to be treated in the trial altogether and the first patient returning to the trial for a second treatment. In this treatment, the patient received five injections in the cervical (upper back) region of the spinal cord, in addition to the ten he received previously in the lumbar (lower back) region of the spine, for a total of 15 injections. This is the highest number of injections in the trial so far. Patient 16 is also the first patient in the world to receive stem cell transplants in both the lumbar and cervical regions of the spinal cord in an FDA-approved trial. Two additional previously-treated patients are expected to return to the trial this summer in this cohort, provided they continue to meet the inclusion requirements. The trial is taking place at Emory University Hospital in Atlanta, Georgia.

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

"Transplanting the first of the returning patients represents a major milestone in the trial," said Dr. Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "The ability to safely administer multiple dosings to these patients is a key enabling step in administering the maximum safe dose. Not only are we dosing patients for a second time in this cohort, we are now dosing in both the lumbar and cervical regions of the spinal cord for the first time, where the stem cell therapy could support both walking and breathing."

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 has been underway since January 2010. The trial is designed to enroll up to 18 patients. 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. The current cohort of three will receive injections in both the cervical and lumbar regions of the spinal cord. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously-treated patients to this cohort. The first of these returning patients was just treated. The entire 18-patient 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 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.

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 has received approval from the FDA to conduct 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 CTE (chronic traumatic encephalopathy), Alzheimer's disease, anxiety, and memory disorders.

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Sixteenth Patient Dosed In Neuralstem ALS Stem Cell Trial

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.

Neuralstems lead development program is for Amyotrophic Lateral Sclerosis ("ALS"), also known as Lou Gehrigs 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.

Neuralstems Approach For ALS

Neuralstem is seeking to treat the symptoms of ALS via transplantation of its hNSCs directly into the gray matter of the patients 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

Gov. Dannel P. Malloy Monday announced $9.8 million in grants to 19 stem cell research projects in the state. The Connecticut Stem Cell Research Advisory Committee had selected the recipients at its grant review meeting last Tuesday in Farmington.

"Connecticut's continued support of stem cell research has allowed for exciting and innovative research to take place right here in our state," Malloy said in a statement. "The research projects funded by these grants allow scientists to do revolutionary work that puts Connecticut at the forefront of bioscience industry."

Of the 19 grants, 13 grants totaling $7.25 million were awarded to Yale scientists, five went to University of Connecticut researchers, and one went to a collaboration between Wesleyan and UConn scientists.

The largest grant, $1.8 million, was awarded to D. Eugene Redmond of Yale. Redmond has focused on cellular repair in the nervous system and how it relates to Parkinson's disease.

UConn's Stormy Chamberlain, an assistant professor of genetics and developmental biology at the UConn Health Center, received a $450,000 grant to develop new therapies for Prader-Willi syndrome and Angelman Syndrome, both rare genetic disorders. Children born with Prader-Willi Syndrome have difficulty feeding and develop poor muscle tone, and starting about age 2, they develop an insatiable appetite that lasts for their lifetime. People with Angelman Syndrome suffer speech difficulties, seizures, problems with motor control and balance, and serious intellectual disabilities

Although Chamberlain generally focuses on Angelman Syndrome, the three-year project also will include Prader-Willi because the causes of the two disorders are similar. Angelman Syndrome is caused by the deletion of genes on a certain chromosome on the mother's side, while Prader-Willi Syndrome is caused by the deletion of genes in same chromosome on the father's side.

Chamberlain estimates that she's one of 30 researchers in the U.S. who studies Angelman Syndrome.

"The state funding really helps rare diseases because the foundations that typically fund their research are limited," she said, adding that support often is limited to fundraisers organized by families of those with the conditions.

A stem cell education outreach program, run by Laura Grabel, a professor of biology at Wesleyan, and Ren-He Xu, a professor of genetics at UConn, received $500,000. Grabel said the program, which has been in operation since 2006, holds workshops and retreats for stem cell researchers and educates the general public by sending speakers to schools and various organizations. The program also has representatives speak to high school science teachers about incorporating stem cell science in their curricula.

Although the program was started partly because of the controversy over the use of stem cells, Grabel said "we've seen very little pushback it's been very positive."

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State Awards $9.8 Million For Stem Cell Projects

Twelve stem cell researchers from Yale received $6.75 million from the Connecticut Stem Cell Fund, according to figures supplied by the states Department of Health.

The amount was the largest ever awarded to Yale since the state legislature in 2005 designated $100 million over 10 years to promote stem cell research in Connecticut. Connecticut was the third state to pass legislation authorizing use of funds to study human embryonic stem cells.

Stem cell researchers at Yale very much appreciate Connecticuts vision and determination in supporting this research despite the challenging economy, said Haifan Lin, director of the Yale Stem Cell Center. In return, our work along with research conducted at the University of Connecticut and Wesleyan has made our state a leader in stem cell research and already positively impacted the state economy.

Yale scientists who received major grants and their research goals are:

Eugene Redmond $1.8 million for treatment of Parkinsons disease using neurons derived from stem cells.

Valerie Horsley $750,0000 for generation of skin cells.

Jeffrey Kocsis $750,000 for use of embryonic cells to remyelinate spinal cord tissue.

Yibing Qyang $750,000 for generation of tissue-engineered blood vessels.

Natalia Ivanova $750,000 for the study of how embryonic stem cells control cell fate.

In-Hyun Park $750,000 for regeneration of neurons.

Excerpt from:
Twelve Yale faculty receive grants for work with embryonic stem cells

ROCKVILLE, Md., June 5, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the Emory University Institutional Review Board (IRB) approved the amendment to the ongoing Phase I trial evaluating Neuralstem's spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The amendment permits the return of three previously-treated patients to the trial to receive additional injections of cells. This modification to the protocol was approved earlier by the Food and Drug Administration (FDA). Implementation was contingent upon IRB approval, which has now been secured.

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

"Bringing patients back for a second set of injections should they meet the inclusion requirements at the time of surgery, or giving new patients both lumbar and cervical injections, is a major step forward toward testing the maximum safe dosing of our cell therapy," said Richard Garr, Neuralstem President & CEO. "We have been encouraged by the results of the trial to date, and are eager to commence treating patients with this increased dosage."

About the Study

The ongoing Phase I study is designed to assess the safety of Neuralstem's spinal cord stem cells (HSSC's) and transplantation technique in up to 18 patients with ALS.

The first twelve patients were all transplanted in the lumbar (lower back) region of the spine. Of these, the initial six (Cohort A) were all non-ambulatory with permanent paralysis. The first patient was treated on January 20, 2010. Successive surgeries have followed at the rate of one every one-to-two months. The first three patients (Cohort A1) were each treated with five unilateral HSSC injections in L2-L4 lumbar segments, while the next three patients (Cohort A2) received ten bilateral injections (five on each side) in the same region. The next six patients (Cohort B and C) were all ambulatory. Of these, the first three (Cohort B) received five unilateral injections in the L2-L4 region. The last three patients (Cohort C) in this study group received ten bilateral injections in the same region.

The trial was then approved to progress to cervical transplantations, with two cohorts of three patients (Cohort D and Cohort E). Cohort D has received five injections in the cervical region of the spinal cord. Cohort E will receive a total of fifteen injections, five in the cervical region and ten in the lumbar region.

About Neuralstem

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Neuralstem Updates ALS Stem Cell Trial Progress; Emory University Institutional Review Board Approves Amendment

Scientists at the Swiss Federal Institute of Technology have brought back full movement of the rats paralyzed by spinal cord injuries in a study that might sooner or later be used in people with similar injuries.

Gregoire Courtine and his team at Ecole Polytechnique Federale de Lausanne saw rats with severe paralysis walking and running again after a couple of weeks following a combination of electrical and chemical stimulation of the spinal cord together with robotic support.

"Our rats are not only voluntarily initiating a walking gait, but they are soon sprinting, climbing up stairs and avoiding obstacles," said Courtine, whose results from the five-year study will be published in the journal Science on Friday.

Courtine is quick to point out that it remains unclear if a similar technique could help people with spinal cord damage but he adds the technique does hint at new ways of treating paralysis. Other scientists agree.

"This is ground-breaking research and offers great hope for the future of restoring function to spinal injured patients," said Elizabeth Bradbury, a Medical Research Council senior fellow at King's College London.

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But Bradbury notes that very few human spinal cord injuries are the result of a direct cut through the cord, which is what the rats had. Human injuries are most often the result of bruising or compression and it is unclear if the technique could be translated across to this type of injury.

It is also unclear if this kind of electro-chemical "kick-start" could help a spinal cord that has been damaged for a long time, with complications like scar tissue, holes and where a large number of nerve cells and fibres have died or degenerated.

Nevertheless, Courtine's work does demonstrate a way of encouraging and increasing the innate ability of the spinal cord to repair itself, a quality known as neuroplasticity.

Other attempts to repair spinal cords have focused on stem cell therapy, although Geron, the world's leading embryonic stem cell company, last year closed its pioneering work in the field.

Continued here:
Robot Therapy Helps Paralysed Rats to Walk Again

1 June 2012 Last updated at 12:19 ET

Seven years ago I stood on a bridge over the M40 doing a "piece to camera" for a report about spinal repair. The aim was to come up with a metaphor for how researchers at University College London were trying to overcome spinal cord paralysis.

It went something like this: "Imagine your spinal cord as a motorway, the cars travelling up and down are the nerve fibres carrying messages from your brain to all parts of the body. If this gets damaged the cars can't travel. The messages are blocked, the patient is paralysed.

"Normally there is no way of repairing a severed spinal cord. But the team at UCL took nasal stem cells, and implanted them into the area of damage. These formed a bridge, along which the nerve fibres re-grew and re-connected."

This is the World-Cup of neurorehabilitation. Our rats have become athletes when just weeks before they were completely paralysed.

The research at the Spinal Repair Unit at UCL involved rats, not humans. In my TV report we showed rats unable to climb a metal ladder after one of their front paws had been paralysed to mimic a spinal cord injury. But after an injection of stem cells, the rats were able to move nearly as well as uninjured animals.

The hope then - and now - is that such animal experiments will translate into similar breakthroughs with patients. Seven years on and the team at UCL led by Professor Geoff Raisman are still working on translating this into a proven therapy for patients. He told me "This is difficult and complex work and we want to ensure we get things right."

So it was with a sense of caution that I approached some Swiss research in the latest edition of the journal Science in which paralysed rats were able to walk again after a combination of electrical-chemical stimulation and rehabilitation training.

The research prompted some newspaper reports talking of "new hope" for paralysed patients. The lead researcher, Professor Gregoire Courtine enthused: "This is the World-Cup of neurorehabilitation. Our rats have become athletes when just weeks before they were completely paralysed."

My colleague James Gallagher has reported on the research and you can read his copy here.

Visit link:
'Hope' for the paralysed?

LONDON Scientists in Switzerland have restored full movement to rats paralyzed by spinal cord injuries in a study that spurs hope that the techniques may hold promise for someday treating people with similar injuries.

Gregoire Courtine and his team at Ecole Polytechnique Federale de Lausanne saw rats with severe paralysis walking and running again after a couple of weeks following a combination of electrical and chemical stimulation of the spinal cord together with robotic support.

"Our rats are not only voluntarily initiating a walking gait, but they are soon sprinting, climbing up stairs and avoiding obstacles," said Courtine, whose results from the five-year study will be published in the journal Science on Friday.

Courtine is quick to point out that it remains unclear if a similar technique could help people with spinal cord damage but he adds the technique does hint at new ways of treating paralysis.

Other scientists agree.

"This is ground-breaking research and offers great hope for the future of restoring function to spinal injured patients," said Elizabeth Bradbury, a Medical Research Council senior fellow at King's College London.

But Bradbury notes that very few human spinal cord injuries are the result of a direct cut through the cord, which is what the rats had. Human injuries are most often the result of bruising or compression and it is unclear if the technique could be translated across to this type of injury.

It is also unclear if this kind of electro-chemical "kick-start" could help a spinal cord that has been damaged for a long time, with complications like scar tissue, holes and where a large number of nerve cells and fibres have died or degenerated.

Nevertheless, Courtine's work does demonstrate a way of encouraging and increasing the innate ability of the spinal cord to repair itself, a quality known as neuroplasticity.

Other attempts to repair spinal cords have focused on stem cell therapy, although Geron, the world's leading embryonic stem cell company, last year closed its pioneering work in the field.

Original post:
Paralyzed rats walk again

LONDON Scientists in Switzerland have restored full movement to rats paralyzed by spinal cord injuries in a study that spurs hope that the techniques may hold promise for someday treating people with similar injuries.

Gregoire Courtine and his team at Ecole Polytechnique Federale de Lausanne saw rats with severe paralysis walking and running again after a couple of weeks following a combination of electrical and chemical stimulation of the spinal cord together with robotic support.

"Our rats are not only voluntarily initiating a walking gait, but they are soon sprinting, climbing up stairs and avoiding obstacles," said Courtine, whose results from the five-year study will be published in the journal Science on Friday.

Courtine is quick to point out that it remains unclear if a similar technique could help people with spinal cord damage but he adds the technique does hint at new ways of treating paralysis.

Other scientists agree.

"This is ground-breaking research and offers great hope for the future of restoring function to spinal injured patients," said Elizabeth Bradbury, a Medical Research Council senior fellow at King's College London.

But Bradbury notes that very few human spinal cord injuries are the result of a direct cut through the cord, which is what the rats had. Human injuries are most often the result of bruising or compression and it is unclear if the technique could be translated across to this type of injury.

It is also unclear if this kind of electro-chemical "kick-start" could help a spinal cord that has been damaged for a long time, with complications like scar tissue, holes and where a large number of nerve cells and fibres have died or degenerated.

Nevertheless, Courtine's work does demonstrate a way of encouraging and increasing the innate ability of the spinal cord to repair itself, a quality known as neuroplasticity.

Other attempts to repair spinal cords have focused on stem cell therapy, although Geron, the world's leading embryonic stem cell company, last year closed its pioneering work in the field.

View post:
Paralyzed rats walk again in Swiss study

ARLINGTON HEIGHTS, Ill., May 29, 2012 (GLOBE NEWSWIRE) -- Adipose stem cells (ACSs)--stem cells derived from fat--are a promising source of cells for use in plastic surgery and regenerative medicine, according to a special review in the June issue of Plastic and Reconstructive Surgery(R), the official medical journal of the American Society of Plastic Surgeons (ASPS).

But much more research is needed to establish the safety and effectiveness of any type of ASC therapy in human patients, according to the article by ASPS Member Surgeon Rod Rohrich, MD of University of Texas Southwestern Medical Center, Dallas, and colleagues. Dr. Rohrich is Editor-in-Chief of Plastic and Reconstructive Surgery.

Adipose Stem Cells--Exciting Possibilities, but Proceed with Caution

The authors present an up-to-date review of research on the science and clinical uses of ASCs. Relatively easily derived from human fat, ASCs are "multipotent" cells that can be induced to develop into other kinds of cells--not only fat cells, but also bone, cartilage and muscle cells.

Adipose stem cells promote the development of new blood vessels (angiogenesis) and seem to represent an "immune privileged" set of cells that blocks inflammation. "Clinicians and patients alike have high expectations that ASCs may well be the answer to curing many recalcitrant diseases or to reconstruct anatomical defects," according to Dr. Rohrich and co-authors.

However, even as the number of studies using ASCs increases, there is continued concern about their "true clinical potential." The reviewers write, "For example, there are questions related to isolation and purification of ASCs, their effect on tumor growth, and the enforcement of FDA regulations."

Dr. Rohrich and co-authors performed an in-depth review to identify all known clinical trials of ASCs. So far, most studies have been performed in Europe and Korea; reflecting stringent FDA regulations, only three ASC studies have been performed in the United States to date.

Many Different Uses, But Little Experience So Far

Most ASC clinical trials to date have been performed in plastic surgery--a field with "unique privileged access to adipose tissues." Plastic surgeon-researchers have used ASCs for several types of soft tissue augmentation, such as breast augmentation (including after implant removal) and regeneration of fat in patients with abnormal fat loss (lipodystrophy). Studies exploring the use of ASCs to promote healing of difficult wounds have been reported as well. They have also been used as a method of soft tissue engineering or tissue regeneration, with inconclusive results.

In other specialties, ASCs have been studied for use in treating certain blood and immunologic disorders, heart and vascular problems, and fistulas. Some studies have explored the use of ASCs for generating new bone for use in reconstructive surgery. A few studies have reported promising preliminary results in the treatment of diabetes, multiple sclerosis, and spinal cord injury. No serious adverse events related to ASCs were reported in either group of studies.

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Fat-Derived Stem Cells Show Promise for Regenerative Medicine, Says Review in Plastic and Reconstructive Surgery(R)

San Diego scientists will receive about $18.1 million in the latest round of funding from the California Institute of Regenerative Medicine (CIRM), the agency that's distributing $3 billion in stem cell research money made available through Proposition 71.

Since funding began, San Diego County researchers have been awarded at least $258 million, making the region one of the largest stem cell research clusters in the country.

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Here's a sample of the latest grants:

Mark Tuszynski, UC San Diego, $4.7 million for research on novel stem cell therapies to treat spinal cord injuries.

Peter Schutlz, The Scripps Research Institute, $4.3 million for research to treat multiple sclerosis.

Juan Carlos Izpisua Belmonte, Salk Institute, $2.3 million for research that would help repair damaged blood vessels.

Yang Xu, UC San Diego, $1.8 million for research that would help treat heart failure.

Eric Adler, UC San Diego, $1.7 million for research to help treat Danon disease, which causes major abnormalities in heart and skeletal muscles.

David Schubert, Salk Institute, $1.7 million for research aimed at treating Alzheimer's disease

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SD scientists get $18 million in stem cell funds

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities.

The May 24 grants bring UC San Diego's total to more than $112 million in CIRM funding since the first awards in 2006.

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5 scientists receive stem-cell research grants

May 23, 2012 12:00pm

Replacing dead or dysfunctional nerve cells with new, healthy ones derived from stem cells eases chronic pain in mice, a new study found.

Researchers from the University of California, San Francisco coaxed mouse embryonic stem cells into becoming mature nerve cells that could bridge gaps in the circuitry that triggers neuropathic pain.

One of the major causes of neuropathic pain is the loss of inhibitory control at the level of the spinal cord because of nerve loss or dysfunction, said study author Allan Basbaum, chairman of UCSFs department of anatomy. The idea was to replace or repopulate the spinal cord cells that provide that inhibition.

The same stem cells, destined to become inhibitory neurons that dampen the signals that cause pain, were previously shown to improve symptoms in a mouse model of epilepsy, Basbaum said. The question was whether we could take the exact same cells and put them in the spinal cord.

Before injecting the cells into the spinal cords of mice with neuropathic pain, the researchers labeled them with a fluorescent tracer to track the connections they made.

We were able to show how these cells integrate beautifully, Basbaum said, describingthe waythe transplanted cells looked and behaved like the mouses own.

Not only did the cells set up shop in the spinal cord, sending and receiving signals through a complex network of neurons, they also eased the neuropathic pain.

In four weeks, the animals condition completely disappeared, Basbaum said, adding that transplanted control cells that lacked the inhibitory properties of the stem-cell-derived neurons failed to ease the pain.

The clinical significance is that we think were actually modifying the disease, not just treating the symptoms, Basbaum said, adding that drugs currently used to ease neuropathic pain fail to treat the underlying problem. Instead of taking a drug to suppress the pain, were trying to normalize the circuit that was damaged by the disease or the injury. The cells repopulate, they integrate, and basically they treat the disease.

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Stem Cells Curb Chronic Pain in Mice

NEWARK, Calif., May 17, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM - News) today announced completion of the first planned interim safety review of the Company's Phase I/II spinal cord injury clinical trial, which indicated that the surgery, immunosuppression and the cell transplants have been well-tolerated. The trial, which is designed to evaluate the safety and preliminary efficacy of the Company's proprietary HuCNS-SC(R) cells (purified human neural stem cells), represents the first time that neural stem cells have been transplanted as a potential therapeutic agent for spinal cord injury. A summary of the data will be presented by Armin Curt, M.D., principal investigator for the clinical trial, at the Interdependence 2012 Global SCI Conference, which is being held in Vancouver, British Columbia, from May 15 to 17, 2012.

The interim data is from the first cohort of patients, all of whom suffered a complete spinal cord injury in which there is no neurological function below the level of the injury. All patients enrolled were transplanted with a dose of 20 million cells at the site of injury in the thoracic spinal cord. There were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first four months following transplantation of the cells. Changes in sensitivity to touch were observed in two of the patients. The data from multiple evaluations of the patients during this four month period have been reviewed by an independent Data Safety Monitoring Committee, which has recommended that the study advance to enrollment of patients with incomplete neurological injury. Enrollment is now underway and is open to patients in Europe, the United States and Canada with incomplete spinal cord injury. The trial, which is being conducted at Balgrist University Hospital, Zurich, Switzerland, is the only ongoing clinical trial evaluating neural stem cell transplantation in spinal cord injury.

"We are very encouraged by the interim safety outcomes for the first cohort," said Dr. Curt, who is Professor and Chairman of the Spinal Cord Injury Center at the University of Zurich, and Medical Director of the Paraplegic Center at Balgrist University Hospital. "The patients in the trial are being closely monitored and undergo frequent clinical examinations, radiological assessments by MRI and sophisticated electrophysiology testing of spinal cord function. The comprehensive battery of tests provides important safety data and is very reassuring as we progress to the next stage of the trial."

The Interdependence 2012 Global SCI Conference is intended to bring together international healthcare and research facilities to showcase their work through presentations, workshops and exhibits and to discuss how to advance research, implement new best practices and shape the next generation of spinal cord injury research. Interdependence 2012 is jointly organized by the Rick Hansen Institute, a Canadian not-for-profit organization committed to accelerating the translation of discoveries and best practices into improved treatments for people with spinal cord injuries, and the Rick Hansen Foundation.

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. The Company has dosed the first three patients all of whom have injuries classified as AIS A, in which there is no neurological function below the injury level. The second and third cohorts will be patients classified as AIS B and AIS C, those with less severe injury, in which there is some preservation of sensory or motor function. The injuries are classified according to the American Spinal Injury Association Impairment Scale (AIS). In addition to assessing safety, the trial will assess preliminary efficacy based on defined clinical endpoints, such as changes in sensation, motor and bowel/bladder 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 a separate four-year observational study will be initiated at the conclusion of this trial.

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. 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

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StemCells, Inc. Reports Positive Interim Safety Data From Spinal Cord Injury Trial

Thirty Percent of Patients Show Improved Functioning after Stem Cell Therapy

Philadelphia, Pa. (May 17, 2012) One of the first long-term studies of stem cell treatment for spinal cord injury shows significant functional and other improvements in three out of ten patients, reports a study in the May issue of Neurosurgery, official journal of the Congress of Neurological Surgeons. The journal is published by Lippincott Williams & Wilkins, a part of Wolters Kluwer Health.

The results support the safety of mesenchymal stem cells (MSCs) derived from the patient's own bone marrow, showing "continuous and gradual motor improvement" in at least some patients with disability caused by spinal cord injury. The lead author of the new study was Dr. Sang Ryong Jeon of University of Ulsan College of Medicine, Seoul, South Korea.

Evidence of Improved Function after MSC Treatment for Spinal Cord Injury The researchers performed MSC transplantation in ten patients with permanent motor (movement) deficits or paralysis (paraplegia or quadriplegia) after spinal cord injury. Mesenchymal stem cells are a type of "multipotent" cell that can be cultured from adult bone marrow and induced to develop into many different types of cells.

The cultured MSCs were injected directly into the injured spinal cord and the surrounding (intradural) space. Additional cells were injected after another four and eight weeks. The results were assessed by measuring improvement in the patients' ability to move their arms and hands and to perform key activities of daily living. Imaging scans and tests of muscle activity were performed as well.

During the first six months after MSC transplantation, six of the ten patients showed improvement in motor power of the arms and hands. Of these, three patients had gradual improvement in the ability to perform daily activitiesfor example, preparing meals and typing on a keyboard.

These three patients also showed significant changes on MRI scans of the spinal cord, including evidence of healing around the injured area of the spine. They also had improvement in electrophysiologic studies of muscle electrical activity.

No Long-Term Safety Problems of MSC Transplant None of the ten patients had any permanent complications related to MSC transplantation. This helps to alleviate concerns that MSC injection could lead to later problems like the development of tumors or calcifications.

Previous studies have shown promising results with MSC transplantation in animals and humans with spinal cord injury. Mesenchymal cells have some important potential advantages for stem cell therapy, as they are a relatively easily accessible source of the patient's own cells. The ten patients treated by Dr. Jeon and colleagues represent the first attempt at direct spinal injection of MSCs for the treatment of spinal cord injury in humans.

Following up on a previous study reporting initial improvement in six patients, the new paper describes continued improvementincluding meaningful gains in the ability to perform everyday functional tasksin three patients. Dr. Jeon and colleagues note that all three patients with progressive improvement had some "residual neurological function." They write, "Therefore, MSC treatment is more likely to enhance the remaining neurological function rather than rengeneration." They call for further studies to understand the mechanism of improvement after MSC treatment and to clarify which patients with spinal cord injury are most likely to benefit.

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Stem Cells for Spinal Cord Injury: Some Patients Have Long-Term Improvement

By Jason Napodano, CFA

We are initiating coverage of Neuralstem Inc. (CUR) with an Outperform rating and $3.00 price target. We believe the companys dual-technology platform focused on cell therapy and traditional pharmaceutical products provides investors with the best of both worlds an early-stage investment in a potentially revolutionary pipeline in regenerative medicine and an attractive and under-valued small molecule pipeline sure to attract interest from larger pharmaceutical partners given the novel mechanism of action and broadapplication potential into multiple indications.

Neuralstems cell therapy technology enables the isolation and large-scale expansion of human neural stem cells from all areas of the developing human brain and spinal cord. Neuralstems technology was created to assist the body in producing new cells to replace malfunctioning or dead cells as a way to treat disease and injury. The companys preclinical research shows these cells both help create new circuitry (neurogenesis) and express factors that protect existing cells (neuroprotective). We believe this platform may be useful in treating many diseases and conditions of the central nervous system (CNS) and neurodegenerative disorders. The lead development programs are currently focused on amyotrophic lateral sclerosis (ALS) and spinal cord injuries (SCI).

Preliminary data from the companys phase 1 clinical trial in ALS (abstract / poster) shows encouraging trends in both safety and efficacy. In this trial, the company is injecting human spinal stem cells (HSSC) directly into the gray matter of the spinal cord. This is no easy task. Management licensed a floating injection platform technology from the Cleveland Clinic that allows the injection of the cells into the spinal cord without risk of trauma. Neuralstems Spinal Cord Delivery Platform and Floating Cannula are being utilized in the current phase 1 ALS program, but have the potential for use in additional clinical applications where injection directly into the cord is needed or advantageous, including spinal cord injury. We believe the Floating Cannula technology alone nearly supports the current market value of only $55 million. Now that safety and feasibility have been demonstrated, if Neuralstem were to spin-out this technology into a separate company, we think given the potential to out-license or commercialize with other companies looking to do direct injections into the spine, it would be worth $25 to $50 million in value.

The phase 1 trial is now progressing into the next six patients, all of which will have direct injections into the cervical spine. The previous twelve patients all had injections into the lumbar spine. The FDA wanted to make sure that the safety and adverse event profile was acceptable before progressing into areas of the spine that affect breathing and upper limb function. So far, three patients have been dosed in the cervical spine with no serious complications. We are excited to see the first efficacy data points in approximately six months.

Neuralstem and the FDA are currently in discussions on potentially expanding the phase 1 program, right now capped at 18 patients, into an additional 9 patients bringing the total to 27 that will include reinjecting patients from previous cohorts and increasing the number of cells per injection from 100,000 to 200,000 or 300,000. We think the more data the company can amass from this phase 1 / 2 program, the better chance the FDA will allow movement into a registration program in 2014. The FDA has already granted Neuralstem Orphan Drug designation for ALS. The FDA allowing Neuralstem to move into phase 3 in 2014 is comparable to Fast Track development status. We see the ALS market as wide open. If successful, Neuralstems HSSC for ALS would be a very attractive treatment option for patients with little hope to date.

We have built a sales model for HSSC in ALS that assumes a U.S. filing in 2016 and approval in 2017. Depending on the magnitude of the efficacy, we think Neuralstems HSSC could capture 15% to 20% market share of the vastly underserved 30,000 U.S. ALS population. We think as many as 50% of these patients could be eligible for Neuralstems HSSC treatment. Our model assumes that Neuralstem commercializes HSSC on its own at a price of approximately $100,000 per treatment. With 20% market share, we see the opportunity for Neuralstem at approximately $300 million (30,000 x 50% x 20% x $100,000).

Given the current market capitalization at only $55 million, we think there is little to no value being assigned to the companys novel oral small molecule platform. Neuralstem has developed the ability to screen and test small molecules on living human neurons in vitro. The companys research into hippocampal atrophy as it relates to neurodegenerative diseases such as major depressive disorder (MDD) and Alzheimers disease (AD) has allowed the company to be granted patents on four first-in-class chemical entities.

The first of these oral small molecule compounds to enter human clinical testing is NSI-189. Preclinical data suggests that NSI-189 significantly stimulates the generation of new neurons (neurogenesis) in vitro and in animal models. The data demonstrates clear evidence of increased hippocampal volume in animals with a model of depression. Neuralstem believes NSI-189 has the potential to reverse the hippocampal atrophy associated with major depressive disorder and other related disorders, and to restore fundamental brain physiology. NSI-189 has the potential to address directly the pathology of the disease itself. This is a major paradigm shift from the traditional oral serotonin or norepinephrine molecules currently approved to treat depression and bipolar disorder.

We think if positive, Neuralstem will seek to strike a development and commercialization partnership on NSI-189 in 2013. We believe that management will be seeking enough cash from partnering NSI-189, and additional hippocampal neurogenesis / neuroprotectant molecules can be brought into the clinic. We expect that milestones on the development of NSI-189 will help fund the pivotal registration trials in ALS or SCI in the companys stem cell pipeline. This is a unique opportunity that many of Neuralstems competitors are lacking the ability to self-fund a potentially revolutionary breakthrough in stem cell technology through the advancement of a traditional small molecule platform.

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CUR - Initiating Coverage of Neuralstem, Inc.

By Andrew Beam abeam@troyrecord.com Twitter.com/beam_record

Ryan Gilbert, assistant professor of biomedical engineering at Rensselaer Polytechnic Institute and G.E. Washington, visiting assistant professor of art at The College of Saint Rose, stand under an inflated sculpture of a ganglion knot created as a result of their unique collaboration inside RPIs Center for Biotechnology and Interdisciplinary Studies in Troy Friday. (J.S. Carras/The Record)

TROY A professor and scientist from Rensselaer Polytechnic Institute and a visiting professor of art from the College of Saint Rose were both taken out of their comfort zones to create artistic and educational works based on research being conducted into stem cell technologies and the repair of spinal cord injuries.

The exhibit, titled A Walk Through the Nervous System: Artists View of Nerves and Spinal Cord Injury opened Friday with the hope of making it easier for the community at large to better comprehend not only how nerves work but also how injures affect the spinal cord.

Dr. Ryan Gilbert, an assistant professor in the Department of Biomedical Engineering at RPI, received a $500,000 grant from the National Science Foundation, $10,000 to $20,000 of which is earmarked for community awareness and outreach. The remainder of the grant funds the research Gilbert and his colleagues are conducting in the departments laboratories.

Gilbert said the department is working with biomaterials on both a nano and micro scale, and in the future hopes to implant them into the spinal cord to regenerate it. Currently, Gilbert explained, when someone injures his or her spinal cord, there is not only the potential for paralysis, but also for permanent damage, as no cure exists yet.

To show exactly what materials Gilbert and his colleagues are working with, department head Deepak Vashishth reached out to Washington, a visiting assistant professor of art at the College of Saint Rose, and his colleagues to help create pieces of art to represent them.

Washington said he spoke with Gilbert about the project. After listening to Gilbert explain some of the materials he was working with and realizing the interest he had in what he was doing, Washington himself became more interested in the project.

Its very interesting and sexy work, Washington said.

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Scientist, artist collaborate on exhibit about spinal cord injuries

ROCKVILLE, Md., May 9, 2012 /PRNewswire/ -- Neuralstem, Inc. (CUR) today reported its financial results for the three months ended March 31, 2012 and provided a business and clinical update.

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

"In the first Quarter of 2012, we entered the final cohort of our Phase I clinical trial in ALS at Emory University Hospital. We are encouraged with the recent approval by the FDA to amend the trial protocol to bring back previously transplanted patients for additional dosing. These patients, who have each received ten lumbar injections earlier in the trial, may now receive an additional five cervical injections. These three patients in our ALS Phase I trial will become the first patients to receive neural stem cell injections up the full length of the spinal cord," said Karl Johe, PhD, chairman of the board and chief scientific officer of Neuralstem, Inc. "These patients are currently 15 to 17 months out from their original surgeries, so we are further encouraged by the fact that their disease progression has been slow enough that they can still be considered for these additional cervical injections. These segments of the spinal cord control breathing, and we believe that multiple injections in the cervical region may be the most effective way to help ALS patients. In order to be eligible, these three patients must meet the same inclusion criteria as new patients into the trial both before and at the time of surgery."

Dr. Johe continued, "This year will also see the start and finish of our neuroregenerative small molecule NSI-189 Phase Ib trial to treat major depressive disorder. This is a novel orally active drug that stimulates new neuron growth in the hippocampus which we believe can help patients with major depressive disorder. We are finalizing the preparations for the first of three cohorts of eight patients each that are scheduled to demonstrate the safety of escalating doses of daily administration of NSI-189 during a 28-day cycle. Dr. Maurizio Fava of Harvard University and Massachusetts General helped to design the trial and we thank him for his efforts.

"Internationally, we expect to commence a combined Phase I/II/III clinical trial for chronic motor disorders from stroke at BaYi Brain Hospital in Beijing through our wholly owned subsidiary, Neuralstem China later in the year. We are currently engaged in test runs at our facility in Suzhou, China where we will manufacture the neural stem cells for the trial," concluded Dr. Johe.

Neuralstem's President and CEO Richard Garr added, "This New Year has seen us actively engaged in licensing discussions for our proprietary surgical device, invented by our ALS surgeon, Dr. Nicholas M. Boulis, with both the industry and Academia. We believe it will be the industry standard for such intraspinal procedures.

"We continue to work with our partner Sumitomo's Summit Pharmaceuticals International Corporation with the goal of licensing NSI-189 to a Japanese pharmaceutical company for development of the Japanese market this year," Mr. Garr continued. "We also continue to see strong interest in co-development opportunities for our preclinical library of additional patented novel neuroregenerative compounds. The company is committed to finding the right partner to move these preclinical compounds forward."

Clinical Program and Business Highlights

Cellular Therapy: Phase I Clinical Trial in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) at Emory University Hospital

Corporate News

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Neuralstem Reports First Quarter Financial Results And Provides Business And Clinical Update

NEWARK Stem cells might repair damage in a spinal cord, regenerating tissue currently considered irreparable. Electrical implants are already allowing a quadriplegic to control a robotic hand with their thoughts. And scientists are working on protein therapy that would keep a bad injury from becoming catastrophic .

These were just some of the advances in treating spinal cord injuries that were heralded at a symposium Wednesday at the University of Medicine and Dentistry of New Jersey. It was the second annual meeting of national scientists and researchers coming together in Newark to sharing their expertise.

The presentations show further advances since last years inaugural event, according to the experts.

"Everything Im seeing here is completely different than what I learned as a medical student 25 years ago," said Robert Heary, a UMDNJ neurosurgeon, the co-director of the Reynolds Family Spine Laboratory at the Spine Center of New Jersey, and the organizer of the event.

Stem cell implantation in a dozen spinal-cord patients is underway in Switzerland, according to Aileen Anderson, an associate professor of physical medicine and rehabilitation at the University of California-Irvine who has been involved in the work. She said the human clinical trial involving multipotent cells at the University of Zurich will be going through 2015 at least but stem cells advances have been moving relatively quickly.

"Cholesterol drugs took 30 years to get to market," Anderson said. "Stem cells as potential therapeutics have moved pretty quickly."

Some of the work presented variations upon a microscopic theme.

A UMDNJ team presented work in which an immune-system protein is suppressed to reduce inflammation allowing better recovery after catastrophic injury in mice. Michele Basso, a professor at the Ohio State University College of Medicine, presented work that showed rodents walking was dramatically improved after the another protein was suppressed, and they got exercise.

"We begin to see a gain of function that we wouldnt normally see," said Basso.

Still others focused on the technologies that being used to currently treat patients.

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UMDNJ symposium heralds advances in treating spinal cord injuries