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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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Stem cells could help treat ALS
A leading neurologist at the University of Michigan will oversee a federally approved clinical trial involving stem cells that researchers hope will hold a key to slowing the progression of Lou Gehrig's disease. New cases of the fatal, neurodegenerative disease known as amyotrophic lateral sclerosis (ALS) are diagnosed in 5,600 Americans annually. Patients with the disease, which ultimately leads to paralysis and the inability to speak or move, have a three- to five-year life expectancy.
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The U-M clinical trial, approved by the U.S. Food and Drug Administration, centers on injecting stem cells into the spines of patients with the disease, with the first step determining safety.
This is not a cure," said Dr. Eva Feldman, the trial's principal investigator, the Russell N. DeJong professor of neurology, and director of the Neuropathy Center at U-M Health System in Ann Arbor. "It's a first step, but a very important step."
One treatment is available for the disease; it extends life by only a few months.
The clinical trial will be conducted with 12 patients at Emory University in Atlanta with fetal-derived neural stem cells patented by Maryland-based Neuralstem Inc. The patients will receive five to 10 stem cell injections in the lumbar area of the spinal cord and be re-examined regularly, with a final data review two years later.
"We will follow these patients for as long as they live," said Richard Garr, president and chief executive of Neuralstem. "We hope that will be a long time."
The trial is based on the research of Feldman, who discovered that injecting stem cells into rats with ALS preserved the large motor neurons and muscle strength that normally die in patients with the disease.
Feldman said the trial also could assess the safety of using stem cells in patients suffering from other neurodegenerative diseases, such as Parkinson's and Alzheimer's.
For now, advocates of patients with ALS were pleased about the announcement, though patients may not see results for years.
"Our hope in the future as the trial expands is that this will be a meaningful approach to treat patients with ALS," said Lucie Bruijn of the ALS Association in California.
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FDA green lights stem-cell clinical trial for Lou Gehrig's disease
The Great Beyond by Monya Baker, September 22, 2009
The Maryland company NeuralStem has the U.S. Food and Drug Administration's permission to test its spinal cord stem cells in twelve patients with amyotrophic lateral sclerosis. The approval comes a month after the FDA placed Geron's planned clinical trial on hold for a second time. NeuralStem's trial had also previously been placed on hold by the FDA in February before receiving the go-ahead in September.
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Though both trials involve placing cells into the spinal cord, NeuralStem’s product is made of cultured neural stem cells derived from a single 8-week fetus; Geron’s product, intended to treat spinal cord injury, is derived from embryonic stem cells that have been differentiated into precursors of neuron-support cells.
“This is certainly the first stem-cell approach for ALS,” says Lucie Bruijn, a scientist at the ALS Association, a patient group that also funds relevant research. Most other approaches for treating ALS are small molecule drugs, she says, and she’s not aware of other cell therapy or other invasive approaches entering human testing in the near future.
ALS has not funded NeuralStem’s work directly, Briujn says, but has advised the company and funded academic scientists who’ve been involved with the company.
NeuralStem’s chief scientific officer Karl Johe says tests of large animal models show that the transplanted cells exert a neuroprotective effect over motor neurons, but it’s not entirely clear how. Earlier this year, Neuralstem and collaborators published results in a rat model of ALS showing that transplanted cells could develop into interneurons that formed synapses with the rats’ motor neurons.
However, Johe emphasized that the upcoming trial will assess safety rather than efficacy. The first few patients selected for the procedure will be those who are no longer able to walk. Because the injected cells protect rather than replace motor neurons, these sicker patients are less likely to benefit from treatment, but they are less able to lose function if something goes wrong. Cells will be injected only on one side of the spinal cord in order to minimize the number of injections into the spinal cord. Only one patient will be injected each month, so that researchers can monitor for effects over a longer period. Eventually, Johe says, the goal is to be able to inject cells in both lower and upper regions of the spinal cord in healthier patients, and see if the injections can keep motor neurons healthy.
The trial is expected to take place at Emory University in Atlanta, Georgia. Though the FDA is allowing the trial to go forward, the university’s patient-safety board will also need to approve the trial before it can proceed. Johe declined to say when that would be but said discussions were well underway.
Other companies using neural cells include ReNeuron, which received permission from UK authorities this January to start clinical trials for stroke. Its cell product is made from genetically modified cultures of neural stem cells, also of fetal origin.
StemCells Inc is conducting trials in Batten’s disease, a neurodegenerative disease that strikes children, and recently received approval for a clinical trial for a similar disease. It also uses neural stem cells from material originally derived from fetuses and has recently published results showing that its cell product delayed some symptoms of the disease by about three weeks.
As with human embryonic stem cells, the patent situation for neural stem cells is contentious. In a pair of dueling press releases this May, NeuralStem and Stem Cells Inc both claimed key intellectual property on these cells.
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FOX Medical Team
