Posts from the PLM site:
Neuralstem, Inc. announced this morning that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to begin a clinical trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
The Company is planning to treat ALS patients through spinal injections of its stem cells via its patented Human Neural Stem Cell technology.
"Like all first human trials, this proposed trial is primarily designed to test the safety and feasibility of both our stem cells and our method of delivering the cells to the spinal cord in ALS patients," said Neuralstem CEO and President, Richard Garr. "We are also proposing secondary endpoints which we hope will be able to measure a slowing down of the degenerative process."
ALS is a progressive 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 the muscles. Loss of the ability to initiate and control muscle movement ends in paralysis and, ultimately, death. ALS affects roughly 30,000 people in the U.S., with about 7,000 new diagnoses per year.
Neuralstem expects to conduct the trial at Emory University with Dr. Johnathan Glass, M.D., Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, as site Principal Investigator (PI). Dr. Eva Feldman, M.D., Ph.D., Head of the A. Alfred Taubman Medical Research Institute and the De Jong Professor of Neurology at the University of Michigan Medical School, will be the overall PI for the ALS trial program. Formal approvals from these institutions to conduct the trial can come only after FDA approval of the trial protocol.
"The filing of this IND is an important event for Neuralstem," said Garr, "but it marks only the beginning of a process which includes working together with the FDA to approve the first human ALS stem cell trial; refining our understanding of how to optimize delivery of our cells into patients; and ultimately delivering a new treatment for patients with this currently incurable disease."
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. Major Central Nervous System diseases targeted by the Company with research programs currently underway include: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington's disease and ALS. The Company filed an IND (Investigational New Drug) application with the FDA for ALS clinical trials in December, 2008, and has entered into a collaborative agreement with Albert-Ludwigs-University, in Freiburg, Germany, to develop clinical trials for Huntington's disease.
In pre-clinical work, the company's cells have extended the life of rats with ALS (Lou Gehrig's disease) as reported the journal TRANSPLANTATION, in collaboration with Johns Hopkins University researchers, and also reversed paralysis in rats with Ischemic Spastic Paraplegia, as reported in NEUROSCIENCE on June 29, 2007, in collaboration with researchers at University of California San Diego.
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An Emory nurse friend tells that that they are in the process of writing the inclusion/exclusion criteria for the trial which could begin anywhere from a few to several months but guessed to be in 2009...
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A sea change in stem cell industry investment is coming, says Richard Garr.
The CEO of Gaithersburg-based Neuralstem happened to be in New York last week, pitching his company to prospective investors, when a member of President-elect Barack Obama's transition team said one of Obama's first priorities as president will be to lift the federal ban on funding embryonic stem cell research imposed by President Bush nearly eight years ago.
When he returned to his biotech this week, Garr said, he had found that "the general feeling in the investment community is that this is going to be a stem cell-friendly administration and a stem cell-friendly Congress." Even in today's difficult investment climate, the stem cell industry is going to see definite benefits, he said.
Human stem cells can be grown into healthy sources of new cells, tissues and organs to help scientists find treatments for diseases such as Alzheimer's, Parkinson's, diabetes and cancer. Some oppose embryonic stem cell research because they believe extracting cells from a viable embryo destroys a human life.
Investors mostly look for signs of progress, politics aside. Garr said he felt "warm interest by investors" at the Acumen BioFin Rodman & Renshaw ninth annual Healthcare Conference in New York because Neuralstem is ready to file an investigative new drug application for the first time and will enter clinical trials with a stem cell drug for amyotrophic lateral sclerosis, or Lou Gehrig's disease, this winter.
"The timing [of the Obama statement] is very good for us," he said.
A change in the federal policy won't directly affect Neuralstem, he said, because his company doesn't receive federal or state money.
But, he said, "It will be a good thing if [Obama] lifts [the funding ban] because it will change the public perception of stem cell companies. ... It will remove the uncertainty of whether or not this is going to be a legal industry. That is huge."
Douglas Doerfler, president and CEO of MaxCyte in Gaithersburg, who testified at a U.S. Senate hearing on the issue in 2005, agreed.
"I believe it will be a huge boon for companies who have been involved in stem cells [and] several are located in Maryland," Doerfler said. "This is a phenomenal area of science, and watching this field closely adds a sense of marvel at the progress being made, especially in regenerative medicine."
Doerfler cautioned, however, that lifting the ban comes late for the U.S. to get "back in the game," as other nations have invested heavily in the promise of stem cells and took competitive advantage while the U.S. was not playing a major role.
Linda Powers, managing director and co-founder of Toucan Capital in Bethesda, Maryland's most active stem cell investment firm, said lifting the ban would be "just the first step" in making the industry more fluid. She hopes, for example, that treatments for heart disease, multiple sclerosis and other serious conditions now being developed in other countries using patients' own bone marrow cells would be developed here without the current requirements of years of clinical trials.
Other restrictions, she said, include regulatory "artificial efficacy" rules on stem cells.
"They are held to much more difficult standards than other medical products," said Powers, a member of the Maryland Stem Cell Research Commission.
Even if the stem cells used are a patient's own, eight to 10 years of clinical trials and hundreds of millions of dollars of expenses are required. "If a patient's own stem cells are used in a therapy, they are regulated as a drug just as stem cells that are not from the patient's own body," Powers said. "That is just plain crazy."
Commercial stem cell research operates in "a very restrictive patient environment as well," which could loosen if federal policies are changed, said Jonathan Auerbach, CEO of GlobalStem, Inc. of Rockville.
Funded partly by Toucan, GlobalStem sells bio-tools for stem cell researchers, such as growth media to feeder cells, characterization tests and assays. Lifting restrictions, Auerbach said, would help because some of the limited embryonic cell lines that are available, called the Bush lines, are old and are "not the best cell lines."
Bowen P. Weisheit Jr., a member of the state stem cell commission and of the state chapter of the Cystic Fibrosis Foundation, said that if Obama lifts the ban, he thinks the effect on current proposals would expand.
This report originally appeared in The Business Gazette.
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Well, I did my part and bought 300 shares of Neuralstem (CUR) stock this morning.
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Researchers Show that a Single Adult Stem Cell Can Self Renew
GEN News Highlights
Stanford University scientists report that they have demonstrated for the first time that a single adult stem cell can repair tissue damage in a live mammal.
The researchers transplanted the skeletal adult muscle stem cells (MusSCs) into special immune-suppressed mice whose muscle satellite cells had been wiped out in a hind limb by irradiation. The stem cells restored lost function to mice with hind limb muscle tissue damage, according to the team.
The adult stem cells used in the study were isolated from a mixed population of satellite cells from the skeletal muscle of mice. Investigators genetically engineered the transplanted MusSC to express Pax7 and luciferase proteins. As a result, every transplanted cell glowed under ultraviolet light and was easy to trace.
The team used luminescent imaging as well as quantitative and kinetic analyses to track each transplanted stem cell as it rapidly proliferated and engrafted its progeny into the irradiated muscle tissue.
The scientists then injured the regenerated tissue, setting off muscle cell growth and repair, and subsequently showed that the MuSC and descendents rescued the second animal’s lost muscle healing function.
After isolating the luciferase-glowing muscle stem cells from the transplanted animal, the scientists cloned the cells in the lab. Like the original MuSC, the cloned copies were intact and capable of self renewal.
In further experiments, the researchers transplanted between 10 and 500 luciferase-tagged MuSC into the leg muscles of mice. These cells also proliferated and engrafted, forming new myofibers and fusing with injured fibers.
Unlike tumor cells, the transplanted stem cells achieved homeostasis, growing to a stable, constant level and ceasing replication. After demonstrating that the transplanted stem cells proliferated and fully restored the animal's lost function, the scientists recovered new stem cells from the transplant with full stem cell potency.
This research was reported at the 48th annual meeting of the American Society for Cell Biology (ASCB) on Sunday, December 14 in San Francisco.
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No, I didn't buy the shares, one of the PLMers did. Maybe something to consider? Also, do any of you know the doctors/researchers mentioned? Just in case? You never know.
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