• First U.S. stem cells transplanted into spinal cord

For the first time in the United States, stem cells have been directly injected into the spinal cord of a patient, researchers announced Thursday.  Doctors injected stem cells from 8-week-old fetal tissue into the spine of a man in his early 60s who has advanced ALS, or amyotrophic lateral sclerosis. It was part of a clinical trial designed to determine whether it is safe to inject stem cells into the spinal cord and whether the cells themselves are safe.

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ALS is a fatal neurodegenerative disease that causes the deterioration of specific nerve cells in the brain and spinal cord called motor neurons, which control muscle movement. About 30,000 Americans have ALS at any given time, according to the ALS Association.

There is no cure for ALS, which is better known as Lou Gehrig's disease, named after the New York Yankees' first baseman and Hall of Famer who retired from baseball in the 1930s after being diagnosed with the disease.

As the illness progresses, patients lose their ability to walk, talk and breathe. Patients usually die within two to five years of diagnosis, according the ALS Association.

Neuralstem Inc., a Rockville, Maryland-based biotech company, received approval from the U.S. Food and Drug Administration to conduct the clinical trial in September. The company is fully funding the research and provides the stem cells that are being injected into the patients.

Neuralstem announced the start of the clinical trial in a news release Thursday.

Longtime ALS researcher and University of Michigan neurologist Dr. Eva Feldman is overseeing the first human clinical trial of a stem cell treatment in ALS patients.

"We are entering a new era of cell therapeutics for ALS, and in my opinion, it is an new era of hope for patients with ALS," Feldman said.

At least 12 patients are expected to participate in this early research. They are to receive the stem cell transplants at Emory University in Atlanta, Georgia.

"This is the first study to see if the invasive injection into the spinal cord is safe for the patient," said Lucie Bruijn, science director of the ALS Association.

This first patient in the clinical trial received several injections of stem cells into the lumbar region of the spinal cord, the area that controls leg function, because most ALS patients first lose muscle function in their legs, according to Karl Johe, Neuralstem's chairman and chief scientific officer.

Bruijn says there have been a few other occasions outside the United States in which fetal stem cells have been injected into a patient, "but not necessarily using a very [rigorous] trial design." She adds that there were also a couple of small studies in Italy that injected other types of stem cells into a few patients but that this is the first FDA-approved trial in the United States.

"Our biggest hope for stem cells is to significantly slow the progression the disease," Bruijn said.

The ALS Association is not providing funding for this clinical trial, but it has supported the work of Dr. Nick Boulis, the Emory neurosurgeon who developed the surgical technique used to inject the stem cells.

Johe invented the technology that allows the company to manufacture billions of copies of stem cells that are taken from a single source of spinal cord cells: cells that were extracted from fetal tissue, which was donated to the company.

"The cells are human neural stem cells," Johe said, acknowledging that the introduction of stem cells is a very invasive procedure.

"What we are attempting is a novel approach by directly injecting them into the middle of the spinal cord, which to our knowledge has never been done before," Johe said.

Researchers plan to follow this and future patients participating in this trial for a long time to determine the safety of the procedure.

These particular stem cells -- which came from the spinal cord of an 8-week-old fetus -- are neural stem cells, which have the ability to turn into different types of nerve cells. These are not the same stem cells as the controversial human embryonic stem cells, which destroy the embryo when the stem cells are removed.

Johe says that once the safety of this type of transplant is determined, he and his colleagues hope to see whether this is a possible treatment for ALS.

"This is not a cure. We are not replacing those motor neurons [nerve cells which tell muscles to contract]. These stem cells don't generate motor neurons. Instead they protect the still-functioning motor neurons," Johe explained.

Bruijn says that injecting stem cells into the spinal cord -- in the region where the motor neurons are located that affect ALS -- is a breakthrough. But she cautions that this is only the first step in the first part of this clinical trial. It's too early to draw any conclusions about the effectiveness of this treatment, especially since the trial has only just begun.

She notes that everyone involved with the study and other ALS patients have to wait and see what the results of the clinical trial will be.

The FDA granted the first approval for injecting human embryonic stem cells into humans to Menlo Park, California-based Geron Corporation in January 2009. Their trials were expected to start last summer but have yet to begin.

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• Neuralstem Receives Approval to Commence First ALS Stem Cell Trial at Emory ALS Center

Neuralstem, Inc. (NYSE Amex: CUR) today announced that its Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease) with its spinal cord stem cells has been approved by the Institutional Review Board (IRB) at Emory University in Atlanta, GA. The trial, which was approved by the FDA in September, will take place at the Emory ALS Center, under the direction of Dr. Jonathan Glass M.D., Director of the Emory ALS Center, who will serve as the site Principal Investigator (PI).

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The trial will study the safety of Neuralstem's cells and the surgical procedures and devices required for multiple injections of Neuralstem's cells directly into the grey matter of the spinal cord. The Emory ALS Center has posted the relevant trial information for patients on its website at http://www.neurology.emory.edu/ALS/Stem%20Cell.html. ALS affects roughly 30,000 people in the U.S., with about 7,000 new diagnoses per year.

"The commencement of the first trial using our stem cells, and the first ALS stem cell trial in the U. S., represents a significant step in regenerative medicine," said Richard Garr, Neuralstem CEO. "We look forward to working with the Emory ALS Center. We expect to begin treating patients with our stem cells in January. Again, patients who are interested should reach out directly to the Emory ALS Center."

About the Trial

This Phase I trial, which will primarily evaluate safety of the cells and the surgery procedure, will ultimately consist of 18 ALS patients with varying degrees of the disease, who will be treated with spinal injections of Neuralstem's patented human neural stem cells. The FDA has approved the first stage of the trial, which consists of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come about 24 months later.

In addition to Dr. Glass, site PI at Emory, the overall PI for the Neuralstem ALS trial program is Dr. Eva Feldman, M.D., Ph.D., Director of the University of Michigan Health System ALS Clinic and the Program for Neurology Research & Discovery.

About Neuralstem, Inc.
Neuralstem's patented technology enables, for the first time, 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 into mature, physiologically relevant human neurons and glia. The company is targeting major central nervous system diseases including: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington's disease and Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig's disease. Neuralstem plans to initiate a Phase I clinical trial to treat ALS with its stem cells. ALS is a progressive fatal neurodegenerative disease that affects nerve cells in the brain, leading to the degeneration and death of the motor neurons in the spinal cord that control muscle movement. Pre-clinical work has shown Neuralstem's cells to extend the life of rats with ALS (as reported in the journal TRANSPLANTATION, October 16, 2006, in collaboration with Johns Hopkins University researchers), and also reversed paralysis in rats with Ischemic Spastic Paraplegia, (as reported in NEUROSCIENCE, June 29, 2007, in collaboration with researchers at University of California San Diego).

Cautionary Statement Regarding Forward Looking Information
This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward- looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10-K for the year ended December 31, 2008 and the quarterly report on form 10-Q for the period ended September 30, 2009.

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• Emory wins 1st stem cell trial for ALS

Emory University will be the site of the first U.S. clinical trail that focuses on using stem cells to slow the progression of adults with Lou Gehrig's disease.

Rockville, Md.-based Neuralstem Inc. (Amex: CUR) hopes to use neural stem cells from the spinal cord of a fetus to slow the progression of adults with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.  The early-stage trial, which could include up to 18 patients, will test the safety of the injection process and the implanted stem cells.

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“No one’s ever injected cells directly into the gray matter of the spinal cord,” Neuralstem President and CEO Richard Garr said.

ALS is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement. ALS patients typically die within three years of diagnosis. About 30,000 people in the U.S. have the degenerative condition and about 7,000 are newly diagnosed each year. There are more than 500 Georgians with ALS.

Embryonic stem cell research is controversial locally and nationally. President Barack Obama signed an executive order in March lifting restrictions on federal funding for embryonic stem cell research. The Georgia Senate, however, OK’d legislation this year that would have shut down most forms of embryonic stem cell research in the state. However, the proposal, which failed in the Georgia House of Representatives, would not have prevented researchers from using new stem cell lines brought in from out of state or existing stem cell lines.

Unlike Neuralstem’s spinal cord-derived stem cells, most embryonic stem cells are derived from embryos that develop from eggs that have been fertilized in an in vitro fertilization clinic.

The internationally watched Neuralstem trial will put Emory’s ALS program — one of the largest ALS clinics in the country — on the map.

Emory was chosen as the site of the trial because it “has one of the best, if not the best, ALS clinicians and research groups,” Garr said. Emory neurosurgeon Dr. Nicholas Boulis developed the surgical techniques to implant the stem cells in the adult spinal cord.

The high-profile clinical trial will accelerate Emory’s translational research, said Dr. Jonathan Glass, principal investigator for the trial and director of the Emory ALS Center.

“It’s going to make us the center of attention for anybody who wants to do stem cell injections into the spinal cord for other diseases,” Glass said. “They’re going to come to us ... and say, ‘How do you do it?’ ‘What’s the best way to do it?’ and ‘Teach us how to do it.’ ”

The publicity surrounding the trial will also make Atlantans aware of the resources in their own back yard, Glass noted.

“When people get sick, some of them go to the Mayo Clinic,” he said. “The reality is that they have the best thing in town and maybe they need to see that.”

People are born with a specific number of spinal cord neural cells, which typically last a lifetime. In ALS patients, certain neural cells die early. When that happens, the spinal cord isn’t able to send messages to the body’s muscles, which in turn atrophy.

Neuralstem hopes its spinal cord-derived stem cells will protect healthy neural cells and repair those that have ceased communicating with the patient’s muscles. That loss of signal triggers muscle atrophy and eventual paralysis that ALS patients suffer.

“The promise of stem cells has been hanging out there for probably more than a decade,” Glass said. “Nobody has really tried it in a systematic way.”

Stem cells are able to find their way to the injured region and transform into nurturing cells, Glass said. “What I’m hoping for,” he said, “is that ... these [stem] cells will set up shop in this region of injury and provide some kind of nurturing effect that will protect the cells that are still there, and possibly even allow the sick cells to reconnect with the muscles

Neuralstem reported in the online journal Neuroscience that three rats paralyzed by a specific spinal cord injury returned to near-normal ambulatory function six weeks after having stems cells grafted to their spinal cords. Three others showed significant improvement after two months. In all the grafted animals, the majority of the transplanted stem cells survived and became mature neurons, Neuralstem said.

The Phase I human trial will test the safety of the procedure which involves delivering the stem cells to a delicate spot — the spinal cord. “Just looking at the spinal cord can hurt it,” Glass quipped.

There’s a lot more at stake than Neuralstem’s fortunes. “If we mess up,” Glass said, “we could take the whole stem cell therapeutic idea and kind of set it back 10 years.”

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• Neuralstem gets FDA OK to test stem cell drug in humans

Neuralstem Inc (CUR.A: Quote, Profile, Research, Stock Buzz) said U.S. health regulators allowed the stem-cell research company to start an early-stage human trial of its spinal cord stem cells in Lou Gehrig's disease, a fatal neuromuscular condition. Shares of the company soared 63 percent to $3.05 in premarket trade. They closed at $1.87 Friday on the American Stock Exchange.

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The trial, which will test the safety of the cells and the surgery procedure, will ultimately consist of 18 patients suffering from the disease, also known as amyotrophic lateral sclerosis (ALS), the company said.

Neuralstem has only received approval for the first stage of the trial that would consist of 12 patients who will receive stem cell injections in the lumbar area of the spinal cord.

ALS affects roughly 30,000 people in the United States, with about 7,000 new diagnoses per year, the company said in a statement.

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