Known as the “Family Cord Blood Banking Act”, new federal legislation in the United States will amend the I.R.S. (Internal Revenue Service) Code to allow couples and individuals to use “tax advantaged dollars” in order to pay for the banking of umbilical cord blood and the adult stem cells contained therein. Tax advantaged financial accounts such as FSAs (flexible spending accounts), HRAs (health reimbursement arrangements) and HSAs (health savings accounts), and variations thereof, will now be applicable to cord blood banking expenses.

The legislation was introduced yesterday in the U.S. House of Representatives by Ron Kind (D-WI), Artur Davis, (D-AL), Wally Herger (R-CA), Mike Thompson (D-CA) and Bill Pascrell Jr. (D-NJ). According to Representative Ron Kind, the chief sponsor of the legislation and a member of the House Ways and Means Subcommittee on Health, “This legislation supports families that choose this potentially life-saving investment by providing tax incentives for these medical expenses.”

A number of private companies have announced that they support the legislation, including the Cord Blood Registry (CBR), which offers collection and preservation services of adult stem cells derived from umbilical cord blood. Currently, families are arbitrarily restricted by tax laws in the use of tax advantaged dollars and in the tax deduction of medical expenses. According to David Zitlow, senior vice president of public affairs and communications at CBR, “Families may pay for over-the-counter cough syrups or heartburn pills using these dollars, but not cord blood banking services. These limitations are unfair and even unwise. Families who opt to deposit into tax advantaged health accounts should have the discretion to spend those dollars as they see fit on qualified medical expenses.”

According to Dr. David Harris, the scientific director at CBR and a stem cell researcher at the University of Arizona, “Research and clinical trials involving cord blood will require more children to have a source of their own cord blood stem cells available for transplant. Consequently, legislation that makes it easier for families to bank cord blood will definitely speed up the time-table for life-saving research and will allow scientists to unlock the vast potential of these amazing cells on a much quicker basis.”

According to Matthew Schissler, CEO and founder of Cord Blood America, an international umbilical cord blood stem cell preservation company, “This would allow individuals and couples to pay for umbilical cord blood banking services through health savings accounts, flexible spending accounts, medical expense tax deduction and health reimbursement arrangements.”

Numerous organizations are involved in raising the awareness of, and lowering the financial barriers to, adult stem cell therapies derived from cord blood. In addition to CBR, other groups who have announced their support of the Family Cord Blood Banking Act include the Coalition for Regenerative Stem Cell Medicine and their member associations which include the Brain Injury Association of America (BIAA), the Association of Nurse Practitioners in Women’s Health, the Parent’s Guide to Cord Blood Foundation, and the National Spinal Cord Injury Association (NSCIA), among others, as well as a growing list of other foundations, companies, university institutions, researchers and disease advocacy groups.

Umbilical cord blood has a history of clinical therapeutic use that predates World War II, and adult stem cells derived from umbilical cord blood have been used in over 14,000 transplants in the treatment of more than 70 different diseases just in the past 20 years alone. While donating to a public cord blood bank is free, private cord blood banking carries associated expenses which may be as high as $2,000 for the first year and $125 for each year thereafter. The advantage, of course, is that the donating family retains the right of exclusive access to their stem cells that are stored in private banks, whereas those who donate to public banks relinquish the right to any future access to their own stem cells. Now, however, the new legislation will lower the cost to families through the new tax incentives, which not only will allow more people to benefit from private cord blood banking but it should also increase the overall supplies of cord blood stem cells.

The harvesting of adult stem cells from umbilical cord blood is a safe and non-invasive procedure which begins with the simple collection of umbilical cord blood at the time of birth. The adult stem cells may then be used throughout the future not only as a therapy for the person who donated the cord blood, but also as a therapy for biological relatives of that person, and for anyone for whom such stem cells may be immunologically compatible.

The Cord Blood Registry (CBR) is the world’s largest stem cell bank. The company is involved exclusively in the collection, processing and storage of adult stem cells derived from umbilical cord blood for future medical use. CBR was the first family cord blood bank to be accredited by the AABB (the American Association of Blood Banks). CBR has been cash-flow positive since 1999 and has thus far stored and processed the umbilical cord blood of more than 260,000 newborns from around the world.

The stem cell company Stempeutics Research has received approval from the Drug Controller General of India (DCGI) to begin the first clinical trial ever to be conducted in India with human stem cells. Specifically, the clinical trial will use mesenchymal stem cells derived from bone marrow in the treatment of two separate conditions, acute myocardial infarction and critical limb ischemia.

According to Nagendra Swamy, the Chief Operating Officer of Manipal Hospital in Bangalore, “It will be a multi-centric placebo-controlled, double blind and allogeneic clinical trial. The aim is to address two diseases: acute myocardial infarction and critical limb ischemia. Since [adult stem cells] derived from a single donor can be manufactured to treat 10,000 patients, we expect the product will provide affordable treatment for all.”

The global market for stem cell therapy is projected to reach $20 billion by the year 2010, and currently the stem cell therapy market in India alone is estimated at $540 million.

In addition to myocardial infarction and critical limb ischemia, which is an advanced form of peripheral artery disease, Stempeutics is also currently developing adult stem cell therapies for the treatment of Parkinson’s disease, spinal cord injury, motor neuron disease, end-stage liver disease, various skin disorders and avascular necrosis.

Aaron Cathcart was told by his doctor that he barely had a year to live, because his heart was so weak that he would not be able to survive surgery. That was over two years ago.

On June 26th of last year, however, Mr. Cathcart received autologous adult stem cell therapy in which his own adult stem cells, derived from his own bone marrow, were administered directly into his heart during open-heart surgery. Prior to receiving the therapy, his heart had an ejection fraction of less than 20%, whereas a normal ejection fraction range is considered to be between 55% and 75%. Three months after receiving the adult stem cell therapy, his ejection fraction had improved to 36%, and by January it had improved even further to 41%. Now, he no longer needs a defibrillator and is almost within the normal range for a healthy ejection fraction.

As Mr. Cathcart now describes, “It used to be that I couldn’t walk a couple hundred feet in the yard without getting pains. I couldn’t go out if the weather dropped below 50 degrees because my heart would strain in the cold.”

Since stem cell therapies are still plodding their way through the multi-year, highly expensive and lethargic FDA clinical trial process that is required for government approval of such therapies in the U.S., most stem cell therapies are not yet available in the U.S. at all, except through FDA-approved clinical trials. In fact, this is exactly how Mr. Cathcart discovered the therapy, since he was fortunate enough to find a clinical trial with adult stem cells that was being conducted not far away from his hometown. The company TCA Cellular Therapy was seeking patients who had suffered heart attacks but had never undergone heart surgery, conditions which applied to Mr. Cathcart and which made him eligible to participate in the trial. The therapy utilized autologous adult stem cells which were taken from each patient’s own bone marrow, expanded in the laboratory and then administered back to each patient according to his or her particular medical condition. Although such therapy usually eliminates the need for surgery, in Mr. Cathcart’s particular case the doctors determined that heart surgery was necessary due to the advanced pathology of his heart, so his own adult stem cells were injected directly into the dead tissue of his heart during bypass surgery.

Embryonic stem cells were never used in the therapy, due to the numerous medical risks and dangers that embryonic stem cells pose, not the least of which is the formation of teratomas (tumors). By sharp contrast, adult stem cells pose no such risks.

As Mr. Cathcart explains, “People hear ‘stem cells’ and they think ‘killing babies’. People are not distinguishing between the two. These were my own stem cells they used. Everyone’s body has them, and if you increase them in concentration, they can repair your body much better than normal.”

The company TCA Cellular Therapy, which is headquartered in Covington, Louisiana, is focused exclusively on the research and development of adult stem cell therapies, not embryonic stem cell therapies. Their latest successes in the treatment of various cardiac conditions hold great promise for the safe, effective and ethical treatment of diseases that afflict a growing number of people each year. In 2005 alone, it was estimated that over 35% of all deaths in the United States were related to cardiovascular disease, and every day approximately 2,400 Americans die from complications of this “silent killer”. Additionally, over 6 million people in the U.S. alone are believed to have blood-flow problems to their legs, which is a contributing factor to cardiovascular disease.

TCA Cellular Therapy owns two patents for proprietary autologous adult stem cell processes and products that are derived from the patient’s own bone marrow, and currently the company is in four separate FDA-approved Phase I and Phase II clinical trials, for the use of autologous bone marrow-derived stem cells in: 1/ the treatment of infarcted myocardium during bypass surgery, 2/ the non-surgical rescue and repair of cardiac muscle following acute myocardial infarction, 3/ the treatment of severe limb ischemia and 4/ the treatment of severe coronary ischemia. Further clinical trials for spinal cord injury and ALS (amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease) are still pending FDA review, and future protocols are currently being designed for the treatment of Parkinson’s disease and idiopathic cardiomyopathy with autologous adult stem cells.

During a conference call for institutional investors held Monday by Summer Street Research Partners, an independent health care research firm, a spinal surgeon and stem cell researcher offered a negative and cautionary view of Geron’s research. Subsequently, TheStreet.com decided not to disclose the doctor’s name due to privacy concerns, “since the call was not intended for public dissemination.” The doctor stated, however, that he has no financial ties to Geron, nor does he own, nor has he shorted, any of Geron’s stock. Nevertheless, the doctor pointed out that the highly publicized upcoming clinical trial for which Geron recently received FDA approval is clinically dubious because the entire rationale for the study is “based on a single experiment in 8 rats.”

In a highly controversial ruling, the FDA granted approval to Geron in January of this year to begin the first clinical trials ever to be conducted in the U.S. with human embryonic stem cells. The clinical trial will administer human embryonic stem cells to patients who have been paralyzed from the waist down with spinal cord injury. Since the primary objective of Phase I clinical trials is to test safety, efficacy will not be tested until years later – assuming, that is, that safety can be proven, although this remains the subject of widespread speculation. Without concrete evidence of safety, the proposed therapy cannot advance to further clinical trials.

In the days immediately following the FDA’s announcement, Geron’s stock soared from $5 a share to more than $48 a share, but such dramatic gains were quickly sold and the stock has steadily fallen ever since then, plummetting to a low of $3.79 on March 5th. According to Adam Feuerstein of TheStreet.com, “Even President Obama’s lifting of the federal research ban on stem cell research Monday failed to provide more than a marginal lift to Geron’s stock price, which closed Tuesday at $4.27”, up a mere 1.4%.

As Mr. Feuerstein further explains, “Wall Street’s health care investors, most notably biotech-focused hedge funds, have been more inclined to steer clear of Geron or short the company’s stock. Geron’s short interest has risen from 8 million shares to 17 million shares over the first two months of the year. Conference calls for Wall Street health care investors with experts critical of Geron’s research, like those held by Summer Street Research on Monday, explain why. The doctor on the Summer Street conference call, a spinal cord injury expert who has also conducted stem cell research, was skeptical about Geron’s study because there is very little animal data to support the theory that a therapy derived from [embryonic] stem cells will benefit patients with severe spinal cord injury.”

Indeed, as the doctor stated, “The fact that Geron’s entire study hinges on this one experiment in eight moderately injured rats is tenuous in terms of efficacy.”

This laboratory experiment upon which Geron’s upcoming clinical trial is based, was conducted by Dr. Hans Keirstead of the University of California at Irvine, using 8 rats in whom spinal cord injury had been deliberately inflicted in order to induce hind-leg paralysis. Rats that were treated with Geron’s human embryonic stem cell product saw a partial return of some function to their paralyzed legs after 7 days, but all of the rats were considered to have “moderate” spinal cord injury. However, when the start of treatment was delayed for more than a week, and also when the experiment was conducted on rats with severe spinal cord injuries, Geron’s therapy had no effect. The human patients who will participate in Geron’s clinical trial will not have moderate spinal cord injury, but instead they will have been chosen for the clinical trial because they suffer from severe spinal cord injury.

According to the doctor who spoke during the conference call, “We don’t know what will happen when these cells are placed into a human, which is the reason immune suppression is required. The risk is that these are not patients you would otherwise want to have on immune suppressants because the severity of their spinal cord injuries, the trauma they’ve suffered, their surgery and wounds make them more susceptible to infection.”

In Geron’s upcoming Phase I clinical trial, human embryonic stem cells will be injected directly into the spinal cords of the human patients. Ordinarily, after undergoing any type of transplantation, immune suppression is required for the remainder of the patient’s life, but the patients in Geron’s clinical trial will be removed from immunosuppressive drugs after 42 days, at which time there is a high risk of immune rejection by the patients’ bodies to the stem cells. In and of itself, this type of immune rejection has the potential to be life-threatening. As Mr. Feuerstein adds, “Another safety concern is the risk that the cells in Geron’s therapy may grow uncontrollably and form tumors on the spinal cord,” to which the doctor from the conference call further adds, “If one patient gets a tumor from the Geron therapy, it will be catastrophic.”

While the CEO of Geron, Tom Okarma, has tried to downplay expectations of efficacy by reminding the public that none of the patients are expected to be instantly cured from the Phase I clinical trial, it is a bit more difficult to dispel concerns about safety.

Despite the fact that Geron’s preclinical data is based upon only one experiment with only 8 rats, Geron’s FDA application nevertheless included a staggering 22,000 pages of data. Although such an unwieldy number might impress those who are uninitiated in “the scientific method”, Mr. Feuerstein points out that such an enormous FDA application “has not stopped some experts from questioning Geron’s science.” One such expert is Dr. Evan Snyder of the California- and Florida-based Burnham Institute for Medical Research, who wrote in an article that was published in the January 30th issue of the journal Science that, “There’s a lot of debate among spinal cord researchers that the preclinical data itself doesn’t justify the clinical trial.” Similarly, in the January 23rd edition of The New York Times, Dr. John Kessler, a neurologist and director of the Stem Cell Institute at Northwestern University, wrote, “We really want the best trial to be done for this first trial, and this might not be it.”

As every investor knows, financial markets are forward-looking, and the price of any particular stock at any particular time is a measure of confidence in that company. In Geron’s case, as with all other biotechs, the financial details are inextricably tied to the scientific details. Precisely for that reason, therefore, at least at this particular moment in history, institutional investors are growing increasingly wary of Geron because of the serious words of caution that are echoed throughout the medical and scientific communities.

An Israeli boy who suffers from a rare, often fatal genetic disease known as ataxia telangiectasia (A-T) has developed a tumor that was directly traced to the fetal stem cell therapy that he received. No known cure exists for A-T, which causes degeneration of the brain regions that control movement and speech, and people with the disease usually do not survive past their teens or twenties. Consequently, when the boy was 9 years old, his family traveled to Moscow so that he could receive an experimental therapy consisting of intracerebellar and intrathecal injections of human neural fetal stem cells – derived from aborted human fetuses – which were then injected into the boy’s brain and spinal cord as a treatment for his A-T. Even though he received two more treatments with the fetal stem cells at the ages of 10 and 12, he still had not shown any improvement by the age of 13 and in fact the severity of his disease had progressed to such an extent that he required a wheelchair. When he began having headaches, tests conducted at the Sheba Medical Center in Tel Aviv revealed a multifocal brain tumor pressing against both his brain stem and his spinal cord, which biopsy revealed to be a glioneural neoplasm. The tumor was surgically removed in 2006 when the boy was 14 years old, at which time cytogenetic and molecular analysis of the tumor revealed it to be of the same tissue as the fetal stem cells, and was therefore caused directly by the fetal stem cell therapy. Among other tests, genetic analysis revealed that some of the cells of the tumor had originated from a female donor and were comprised of 2 normal, healthy copies of the gene in which mutations cause the A-T disease, and which therefore did not match the boy’s own genotype containing abnormal copies of the gene.

Had this tumor not been discovered and surgically removed, it certainly would have been fatal and the boy would have died – not from the A-T disease, but from the therapy which was meant to treat the A-T disease. Indeed, one of the most serious concerns with embryonic and fetal stem cells is that they might constitute “therapies” which are worse than the diseases that they are meant to treat. This particular example with a boy suffering from A-T offers strong evidence to justify exactly such a concern. The boy is being closely monitored for any other tumors which might also possibly develop over time, as a lingering yet direct result of the fetal stem cell therapy that he received.

The formation of tumors has long been one of the main concerns associated with embryonic and fetal stem cells, and the fact that this boy’s tumor was not detected until 4 years after his first treatment raises some new concerns. According to Dr. Marius Wernig of Stanford University, “Stem cell transplantations have a humongous potential. But if people rush out there without really knowing what they’re doing, that really backfires and can bring this whole field to a halt.”

As Josephine Quintavale of the public interest group Comment on Reproductive Ethics adds, “The risks of tumor formation in association with embryonic stem cells are widely acknowledged and one reason why there are very serious concerns about the proposed use of such cells in treating spinal cord injury in the US. It would appear from this report that fetal stem cells are similarly unstable. These are not areas of therapy we should be rushing into, whatever the ethical debates surrounding the use of embryo or fetal tissue per se.”

Dr. Stephen Minger of King’s College London further explains, “This is worrying and we have to be cautious. We need to have long term monitoring and follow-up of the patients who are given stem cells and rigorous regulation of centres providing cell therapy. Although this is just one case, it does show that we need to be careful about the cell population we are using.”

As we have often explained on this website, all stem cells are not created equal, nor do they behave equally, and important distinctions must be made between the different types of stem cells. Generally speaking, all stem cells fall into 2 broad categories: adult stem cells, and everything else. The latter category includes embryonic and fetal stem cells, while stem cells that are derived from umbilical cord and placental blood are categorized as “adult” stem cells. The relatively recently discovered endometrial regenerative cells (ERCs), which have shown to be particularly promising, also fall into the category of “adult” stem cells. It is important to understand that adult stem cells behave very differently from embryonic and fetal stem cells, with one of the major differences being the risk of tumor formation, which has long been known to be inherently problematic in embryonic and fetal stem cells, especially in regard to the formation of teratomas (a specific type of tumor), which is the definitive requirement of pluripotency; by sharp contrast, however, adult stem cells do not cause tumors because adult stem cells are not pluripotent, but are instead multipotent, at best. In the past, the lack of pluripotency in adult stem cells was seen as a disadvantage, although increasingly it is being recognized as a distinct advantage, since it eliminates any danger of tumor formation.

According to a warning issued by Dr. John Gearhart, a stem cell scientist at the University of Pennsylvania, “Patients, please beware. Cells are not drugs. They can misbehave in so many different ways, it just is going to take a good deal of time.”

Although this particular case of tumor formation was originally reported in the PLoS medical journal, a peer-reviewed open access journal published by the Public Library of Science, the story was subsequently republished in all major media outlets around the world. As the authors of the PLoS paper cautiously conclude, “This is the first report of a human brain tumor complicating neural stem cell therapy. The findings here suggest that neuronal stem/progenitor cells may be involved in gliomagenesis and provide the first example of a donor-derived brain tumor. Further work is urgently needed to assess the safety of these therapies.”

Representatives of Neuralstem have announced that the company received official “Notice of Allowance” from the U.S. Patent and Trade Office, for its technology that will immortalize stable neural stem cell lines.

The newly patented process utilizes cMyc-ER, which is a recombinant fusion of two proteins that are normally present in cells, namely, the estrogen receptor (ER) which is a human protein activated by estrogen, and c-Myc, which is a protein that regulates the human cell cycle.

According to Neuralstem president and CEO Richard Garr, “We are pleased to have received the Notice of Allowance on this important technology. The technology behind this patent allows us to grow practically unlimited quantities of neural stem cells from all regions of the brain without regard to the natural mitotic limits of cells from a particular region. Equally important, this technology is a next-generation immortalization process that avoids the harmful effects of traditional immortalization methods, which have invariably resulted in uncontrolled growth. Our technology provides the necessary assurance that the cell lines are fully controlled and remain consistent, trial over trial and year after year. This consistency is ultimately key to the commercialization of any cell products and greatly enhances our ability to deliver cell therapies for very prevalent diseases, such as stroke and traumatic brain injury. It will also enable, for the first time, systematic drug screening against many different kinds of normal human brain cells for new central nervous system drugs, and stem cell-mediated protein delivery for neurologic diseases.”

Neuralstem’s patented technology allows, for the first time, the production of neural stem cells from the human brain and spinal cord in commercial quantities, and for the controlled differentiation of these cells into human neurons and glial cells.

Researchers at Neuralstem are focused on major pathologies of the central nervous system including Huntington’s disease, ischemic spastic paraplegia, traumatic spinal cord injury, and amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease. In December of 2008, Neuralstem filed an IND (investigational new drug) application with the FDA for ALS and has also entered into a collaborative agreement with Albert Ludwigs University in Freiburg, Germany to develop clinical trials for Huntington’s disease.

Neuralstem Inc. announced this morning that it has filed an IND (investigational new drug) application with the U.S. FDA (Food and Drug Administration) to begin the first ever human clinical trials for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, with adult stem cells.

According to Richard Garr, J.D., president and CEO of Neuralstem, “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. We are also proposing secondary endpoints which we hope will be able to measure a slowing down of the degenerative process.”

The treatment will consist of spinal injections of the adult stem cells using the company’s patented and proprietary technology. The clinical trials will take place at Emory University under the direction of Dr. Johnathan Glass, Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, pending offical FDA authorization.

Approximately 30,000 people in the U.S. alone are afflicted with ALS, which is a progressive neurodegenerative disease in which the deterioration of nerve cells and motor neurons progresses from a loss of muscular control to paralysis and ultimately death. Conventional medical modalities have thus far been unsuccessful in halting or reversing the course of the disease, which until now has been considered incurable. Adult stem cell therapies, however, constitute the first type of treatment that is actually capable of offering realistically achievable improvement.

According to Richard Garr, “The filing of this IND is an important event for Neuralstem, 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.”

As described on their website, “Neuralstem, Inc. is a publicly traded biotherapeutics company whose mission is to apply stem cell research and its patented human neural stem cell technology to treat diseases of the central nervous system including ischemic paraplegia, traumatic spinal cord injury, ALS and Parkinson’s disease. Our stem cell research has resulted in patent-protected technology that allows us to produce mature, commercial quantities of neural stem cells with the ability to control the differentiation of the cells into physiologically relevant human neurons and glia.”

Researchers at the Salk Institute in La Jolla, California have announced two ground-breaking accomplishments: one is a demonstration of the fact that amyotrophic lateral sclerosis (ALS) can be improved with specific antioxidants, and the second is the creation of a new model of ALS which is based upon human rather than mouse tissue.

Dr. M. Carol Marchetto of the Salk Institute has created the first human ALS laboratory model ever developed, by using human embryonic stem cells. Previously, laboratory experimentation with ALS has typically been conducted with mouse models, which are only rough approximations of human ALS since the disease is caused by genetic mutations that are unique to the human genome and cannot be identically reproduced in the mouse genome. Dr. Marchetto has circumvented the problems associated with the mouse model by creating the new human model, in which she induced a genetic mutation in SOD1 (superoxide dismutase 1), the gene that instructs the body in how to manufacture the enzyme superoxide dismutase, which, among other properties, defends the body from the oxidative and inflammatory cellular damage caused by free radicals, which have long been suspected of playing a key role in motor neuron death. By studying the cellular environment of the motor neurons in this new human model, the researchers made an important discovery with astrocytes (astroglia), which are the star-shaped glial cells in the brain and spinal cord that play a number of key roles, which include providing nutrients to the nervous tissue, repairing brain tissue and supporting endothelial cells, especially in the blood-brain barrier. In a cellular environment in which ALS is present, the researchers discovered that the astrocytes are constantly bombarding the motor neurons with free radicals. The team of scientists then began testing potential drugs for their antioxidant and anti-inflammatory properties, which could possibly protect the motor neurons from the damage caused by the constant stream of free radicals. Although several pharmaceuticals were found to be prime candidates, the naturally occurring antioxidant apocynin, which is present in many plants, was identified for its ability to prevent neuronal death by blocking both the oxidation and the inflammation of the motor neuron cells. Short of figuring out how to get the astrocytes to stop secreting free radicals, the next best approach is simply to block the damage caused by the free radicals.

Until now there has only one drug approved for the treatment of ALS in the United States, namely, riluzole, which at best can only slow the progession of the disease by a month or two and does nothing to reverse the cellular damage caused by the death of the motor neurons. According to Dr. Fred Gage, professor of genetics and the principal investigator of the study, “There is an urgent need for new models of the disease that have the potential to translate into clinical trials and that could, at a minimum, be used to verify drugs and drug targets.”

Many embryonic stem cell experts, including the pioneering embryologist Dr. James Thomson, have emphasized the point that the development of actual cell-based therapies from embryonic stem cells is a long and complex process, and such therapies are still at least a decade away, if not further. Meanwhile, instead of directly seeking cell-based cures, such embryonic stem cell authorities have advocated an approach that focuses on the use of embryonic stem cells for drug testing and development, and this new human model of ALS is an excellent example of precisely such an approach. Although the use of embryonic stem cells even for this purpose does not pacify the embryonic stem cell opponents, who still find the use of embryonic stem cells for drug testing to be unethical, the new ALS model nevertheless does highlight the sobering scientific reality that therapeutic cell-based cures from embryonic stem cells will not be immediately forthcoming, purely for scientific, not political, reasons. Opponents of embryonic stem cells also point out the fact that adult stem cells could just as easily be used to create new laboratory models of diseases, bioengineered from human rather than mouse tissue, while avoiding entirely the controversial ethics of embryonic stem cells as well as the numerous scientific problems and medical dangers that are inherent in embryonic stem cells, such as their ability to form the specific type of tumor known as a carcinoma, among other problems. Furthermore, laboratory models of diseases created from adult stem cells could be used not only for drug testing but also for the development of actual cell-based therapies that directly use the adult stem cells themselves for the treatment of disease and injury. In fact, such cell-based therapies have already been developed from adult stem cells, and are already in clinical use.

Although a major advantage of Dr. Marchetto’s new ALS model was the fact that it was conducted on human cells, rather than in mice, she and her colleagues are now planning to test apocynin and other chemicals in mouse models of ALS to see whether or not there is any real benefit that can be measured in mouse survival.

Neuralstem has licensed the patent rights for three inventions from the Cleveland Clinic, all of which utilize spinal cord injection technology, and which Neuralstem will use in clinical trials for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease). All three inventions were developed by Dr. Nicholas Boulis, currently at Emory University but formerly of the Cleveland Clinic. Neuralstem plans to file its IND (Investigational New Drug) application with the FDA before the end of this year, with trials scheduled to commence in early 2009.

According to Neuralstem President and CEO, Dr. Richard Garr, “We are very pleased to have licensed-in these important delivery technology rights. As we get ready to move into our expected human trial for ALS, we are focused on the optimal way to deliver our cell therapeutics to enhance the safety and efficacy of the treatments. Transplanting our cells directly into the spinal cord is a challenging but necessary part of trying to treat diseases of the spinal cord, not only in ALS but in other spinal cord injuries and diseases. The technology today will help us meet those goals.”

Specifically, the three inventions consist of devices which are known as a “stabilized platform and microelectrode recording guidance validation”, a “floating cannula for spinal cord therapeutic injection”, and a “spinal platform and methods for delivering a therapeutic agent to a spinal cord target”. According to its website, “Neuralstem, Inc. is a biotherapeutics company utilizing its patented Human Neural Stem Cell technology to create cures for diseases of the CNS (central nervous sysem). The Company’s technology allows for the isolation of CNS stem cells from tissue, the expansion in vitro of each cell up to a billion-billion times (60 doublings), and the controlled differentiation of the cells into mature, physiologically relevant human neurons and glia.”

In addition to ALS, ischemic paraplegia, traumatic spinal cord injury, and Parkinson’s disease are also among Neuralstem’s primary areas of focus.

Mesenchymal stem cells (MSCs) have already proven to offer a viable therapy in the treatment of spinal cord injuries, of which approximately 11,000 new cases occur each year in the United States alone. Worldwide, traumatic accidents involving the spinal cord constitute some of the most debilitating of injuries. Now, the Indian company Stempeutics Research, in collaboration with Manipal Hospital in Bangalore, has conducted a pilot study utilizing newly developed techniques for the administration of MSCs to patients in India, which has one of the highest rates of spinal cord injuries of any country in the world, with approximately 20,000 new cases reported each year.

According to Dr. Sujay Rao, a consulting neurosurgeon at St. Philomena’s Hospital in Bangalore, it is important to deliver the stem cells as close as possible to the physical site of injury. Injecting the MSCs via intra-arterial or intra-spinal routes, with the assisted guidance of CT (computed tomography) imaging, will maximize the efficacy of the stem cells in treating the injury. The MSCs used in the pilot study are derived from adult bone marrow and are able to differentiate into oligodendrocytes, which play a key role in the production of myelin and are an important component in the neurological system.

In the past, permanent paralysis and loss of sensation below the site of injury have usually been the result of spinal cord damage, accompanied also by loss of bladder and bowel control. As a result of this pilot study, however, a number of patients have already shown improvement from the newly developed delivery techniques. One quadriplegic patient has regained upper limb movement as well as bladder control and is now able to stand with support. Similarly, a patient who had been paraplegic is now able to walk with support and has regained bladder as well as bowel control.

A private company dedicated to the research and clinical applications of stem cells, Stempeutics currently has facilities in India and Malaysia.