Tuesday, July 28, 2009

Stem-Cell Breakthrough

It's a chilling thought. In the coming year, 130,000 people worldwide will suffer spinal-cord injuries?in a car crash, perhaps, or a fall. More than 90 percent of them will endure at least partial paralysis. There is no cure. But after a decade of hype and controversy over research on embryonic stem cells?cells that could, among other things, potentially repair injured spinal cords?the world's first clinical trial is about to begin. As early as this month, the first of 10 newly injured Americans, paralyzed from the waist down, will become participants in a study to assess the safety of a conservative, low-dose treatment. If all goes well, researchers will have taken a promising step toward a goal that once would have been considered a miracle?to help the lame walk.

The trial signals a new energy permeating the field of stem-cell research. More than 3,000 scientists recently met in Barcelona for the annual conference of the International Society for Stem Cell Research, compared with just 600 researchers five years ago. Money from major pharmaceutical companies is following the advances. Former U.S. vice president Al Gore, now a partner in the venture-capital firm Kleiner Perkins Caufield & Byers, has thrown his weight behind the research. In April, the firm joined with Highland Capital Partners to invest $20 million in iZumi Bio (now iPierian), a startup firm working on stem-cell therapies.
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Despite the considerable hype surrounding stem cells in recent years, the possibilities now appear to be broader than most people realize. In addition to helping replace damaged cells in patients with diseases like diabetes or Parkinson's, stem cells have the potential to change how we develop drugs and unravel the biology of disease. They may even be used one day to create replacement organs. "There's been a massive injection of optimism into the field," says stem-cell biologist Alan Trounson, president of the California Institute for Regenerative Medicine. "It's remarkable how fast it's progressing."

Much of the excitement comes from the development of a new type of stem cells, called "induced pluripotent" stem cells, or iPS. Shinya Yamanaka first concocted the cells in his Kyoto University lab by inserting four genes into fully formed adult skin cells. They began to behave like embryonic stem cells, capable of forming unlimited copies of any of the body's 220 cell types. Because iPS cells can be derived from a patient's own adult cells, they do not carry the risk of rejection by the immune system. Equally important, because iPS cells are not derived from embryos, they skirt a major ethical and religious problem.

The first iPS cells, however, will not be used as replacement tissue for spinal cords and other organs. Because iPS cells have subtle (and potentially dangerous) differences from true embryonic stem cells, many doctors are leery of putting them directly into patients until more research is done. But the cells could be immensely important in helping scientists understand and treat genetically based diseases.

By the time a full-blown disease has emerged, says Harvard stem-cell biologist Konrad Hochedlinger, it's like an airplane that has crashed. You can examine the wreckage for clues, but what you really want is the plane's black boxes?the flight-data and cockpit voice recorders that tell you exactly how electrical systems failed, hardware malfunctioned, and pilots made crucial errors. That's what doctors think iPS cells could provide. By coaxing some iPS cells into becoming the cell types affected in Huntington's disease, type 1 diabetes, or ALS (Lou Gehrig's disease), scientists will be able to watch in the lab as the disease unfolds. They'll be able to understand how the disease starts, which could lead to new ways of blocking it.

Embryonic stem cells are still regarded as the gold standard. That's why there is intense interest in the U.S. spinal-cord-injury trial. Sponsored by Geron Corp. in California, the trial will recruit patients within one to two weeks of their injuries, before scar tissue has formed. Doctors will inject a derivative of stem cells, called progenitor cells, that manufacture myelin, the substance that coats the long, spindly projections on nerve cells, much the same way that insulation coats electrical wires. Damage to cells that make and maintain the myelin sheath, as happens in spinal-cord injuries, prevents nerves from conveying messages from the brain. Although it's not clear yet whether the treatment is effective or safe, the restoration of even partial function would be a huge advance.

Geron's CEO, Dr. Thomas Okarma, thinks that spinal injury is a logical place to begin. Because patients will be completely paralyzed from the waist down, any improvement will be the result of the therapy, not chance. And the spinal cord is an "immune-privileged site," meaning that the attack cells of the immune system cannot get in and destroy the embryo-derived cells. "If the therapy is safe and effective, the potential impact will extend way beyond spinal-cord injury," says Okarma. "It will mark the start of a new era in medical therapeutics."

Other companies aren't waiting for the results. The U.S. pharmaceutical giant Pfizer is pursuing two other embryonic-stem-cell-based therapies, which it hopes to have in clinical trials by 2011. In April the company partnered with University College London to pursue a therapy for macular degeneration, the principal cause of blindness in the elderly. The disease leads to the gradual destruction of the macula, the sensitive central portion of the retina. But Peter Coffey, professor of cellular therapy and visual sciences at UCL, is using embryonic cells to make the same type of support cells that lie just behind the retina, providing it with nutrients. The goal is to implant a disc-shaped layer of the cells behind the retina. Immune rejection should not be a problem, since the eye is also immune-privileged.

Pfizer's other collaboration, with Novocell in California, aims to devise a treatment for some of the 100 million patients worldwide with insulin-dependent diabetes. Novocell is using embryonic stem cells to help regenerate all five of the pancreas's cell types. But there's a hitch. Unlike the eye or the spinal cord, the pancreas has no immune protection. For this, Novocell has devised a clever solution. It encases the stem-cell-derived progenitor cells in a capsule that can be implanted in the body. The pore size of the fabric is large enough to allow oxygen, glucose, and insulin to pass through but small enough to keep out big immune cells. "If problems should develop, the surgeon can easily remove the capsule," says Liz Bui, director of intellectual property for Novocell.

Some researchers aren't interested in just replacing impaired cells. They're using adult stem cells?which exist within organs to help with minor repairs?to grow entire replacement organs and tissues. Dr. Anthony Atala, director of the Institute for Regenerative Medicine at Wake Forest University in North Carolina, has made human bladders in this way. He starts by taking a small bladder biopsy from the patient and extracting his or her stem cells. After allowing the cells to multiply in the lab for about a month, he spreads them onto a collagen scaffold fashioned in the shape of a bladder. He then incubates the would-be organ in a bioreactor that provides the same temperature, oxygen level, growth factors, and nutrients that would be found in the body. In two weeks, he has a small but functional organ, ready for a patient.

In the early 2000s, Atala completed the procedure on seven children with spina bifida, who never developed fully functional bladders. He has now followed these patients for eight years to make sure there are no drastic failures or side effects. And he has moved on to other possible replacement parts. "We're working on 22 tissues and organs, including kidneys, heart valves, and cartilage," he says.

Because any new therapy is inherently risky, researchers are careful about creating false hopes that cures are just around the corner. Therapies that succeed in the idealized world of the lab can fail in real life or take decades to put into practice. As doctors and regulators begin to consider treating patients, they still have basic questions. Will the cells survive for long in the body? Will they integrate to form functioning tissue? Will the benefits outweigh risks that may become apparent only decades from now? Scientists are daring to hope, though, that after a decade of hype, real progress is imminent. Millions of patients worldwide could one day be the beneficiaries.

By Anne Underwood | NEWSWEEK

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Sunday, December 14, 2008

Fat Cells May Restore Spinal Cord Function Post Injury

Mature fat cells helped mice recover from spinal cord injuries, according to a promising new study. They could become a source for cell replacement therapy to treat central nervous system disorders in humans.

Yuki Ohta of the St. Mariana University School of Medicine, Kawasaki, Japan, who led the study, said fat or adipose-derived stem cells have been shown to differentiate into neuronal cells in a test tube setting.

Now, for the first time fat cells have been shown to successfully differentiate into neuronal cells in in-vivo (animal models) tests. The fat cells are grown under culture conditions that result in their becoming de-differentiated fat (DFAT) cells, according to a St Mariana release.

"These cells, called DFAT cells, are plentiful and can be easily obtained from adipose tissue without discomfort and represent autologous (same patient) tissue," said Ohta.

Tests in animal models confirmed that the injected cells survived without the aid of immunosuppression drugs and that the DFAT-grafted animals showed significantly better motor function than controls, said Ohta and colleagues.

"We concluded that DFAT-derived neurotrophic factors contributed to promotion of functional recovery after spinal cord injury (SCI)," said Ohta.

"Transplanting DFAT cells into SCI rats significantly promoted the recovery of their hind limb function."

"These studies demonstrate the ability to obtain stem cells from a patient?s own fat that can help repair injury to the spinal cord," said Paul R. Sanberg, University of South Florida Health, and joint editor-in-chief of Cell Transplantation, which published the report.

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Friday, November 21, 2008

Nose Cells May Heal Spinal Cord Injuries

People paralysed by spinal cord injuries could soon be "repaired" using cells from their own noses, say Otago University researchers.


The Health Ministry's ethics committee has just approved an application by the Spinal Cord Society to open the way for a clinical trial involving 12 patients, which could start next year.

The society's president, Noela Vallis, said there was no shortage of volunteers ready to take part.

"Some have already gone overseas out of a sense of frustration that they can't access it [the experimental treatment] here," Mrs Vallis said.

About 5000 Kiwis are in wheelchairs as a result of accidents - the highest rate of any country in the developed world.

Research director Jim Faed, who heads the the Spinal Cord Society's lab at Otago University, has spent five years developing laboratory methods for growing cells potentially useful for spinal cord injury repair.

His team is focusing on two promising cell types: one is a kind of adult stem cell produced by a patient's own bone marrow.

However, researchers are likely to begin trials using olfactory (scent receptor) cells from the patient's nose, injecting them into damaged spinal cord.

"The olfactory tissue in the nose is unique because it is the only place in the body where there is constant replacement of nerve cells throughout life," Dr Faed said.

"There is growing medical opinion that these cells can help overcome the blocks that prevent nerve cells regenerating after damage to the spinal cord."

The nasal tissue acts like "nurse cells", providing growth factor hormone to nerve cells, enabling them to make "meaningful connections".

Internationally, several research groups have done animal trials using the cells, but there has been only one human trial - in Portugal in 2006. The Otago group is in contact with Portuguese neuropathologist Carlos Lima, who pioneered that trial.

Dr Faed said some participants experienced side-effects, but they were "few and manageable" and none had been fatal.

Positive benefits for patients included return of some muscle function and sensation in parts of the body which previously had no feeling.

Dr Faed said the Dunedin lab hoped to get full approval for the trial before Christmas, and would then begin recruiting patients. The first 12 could start treatment next year.

Mrs Vallis - who founded the society after her late husband was paralysed in an accident - said the group aimed to raise $1 million to fund the trial, in addition to the $300,000 it finds every year to run the lab. "We should be at the forefront of developing this medical treatment, given the number of our citizens in wheelchairs."

Feilding man Iain Scott, a quadriplegic since dislocating his neck while playing rugby 19 years ago, said the possibility of the treatment was "huge" and gave hope to people with spinal cord injuries. "If nothing happens, at least you had a go ... you don't want to die wondering."

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Sunday, July 20, 2008

Stem Cells Identified for Spinal-Cord Repair

A researcher at MIT?s Picower Institute for Learning and Memory has pinpointed stem cells within the spinal cord that, if persuaded to differentiate into more healing cells and fewer scarring cells following an injury, may lead to a new, non-surgical treatment for debilitating spinal-cord injuries.

The work, reported in the July issue of the journal PLoS (Public Library of Science) Biology, is by Konstantinos Meletis, a postdoctoral fellow at the Picower Institute, and colleagues at the Karolinska Institute in Sweden. Their results could lead to drugs that might restore some degree of mobility to the 30,000 people worldwide afflicted each year with spinal-cord injuries.

In a developing embryo, stem cells differentiate into all the specialized tissues of the body. In adults, stem cells act as a repair system, replenishing specialized cells, but also maintaining the normal turnover of regenerative organs such as blood, skin or intestinal tissues.

The tiny number of stem cells in the adult spinal cord proliferate slowly or rarely, and fail to promote regeneration on their own. But recent experiments show that these same cells, grown in the lab and returned to the injury site, can restore some function in paralyzed rodents and primates.

The researchers at MIT and the Karolinska Institute found that neural stem cells in the adult spinal cord are limited to a layer of cube- or column-shaped, cilia-covered cells called ependymal cells. These cells make up the thin membrane lining the inner-brain ventricles and the connecting central column of the spinal cord.

?We have been able to genetically mark this neural stem cell population and then follow their behavior,? Meletis said. ?We find that these cells proliferate upon spinal cord injury, migrate toward the injury site and differentiate over several months.?

The study uncovers the molecular mechanism underlying the tantalizing results of the rodent and primate and goes one step further: By identifying for the first time where this subpopulation of cells is found, they pave a path toward manipulating them with drugs to boost their inborn ability to repair damaged nerve cells.

?The ependymal cells? ability to turn into several different cell types upon injury makes them very interesting from an intervention aspect: Imagine if we could regulate the behavior of this stem cell population to repair damaged nerve cells,? Meletis said.

Upon injury, ependymal cells proliferate and migrate to the injured area, producing a mass of scar-forming cells, plus fewer cells called oligodendrocytes. The oligodendrocytes restore the myelin, or coating, on nerve cells? long, slender, electrical impulse-carrying projections called axons. Myelin is like the layer of plastic insulation on an electrical wire; without it, nerve cells don?t function properly.

?The limited functional recovery typically associated with central nervous system injuries is in part due to the failure of severed axons to regrow and reconnect with their target cells in the peripheral nervous system that extends to our arms, hands, legs and feet,? Meletis said. ?The function of axons that remain intact after injury in humans is often compromised without insulating sheaths of myelin.?

If scientists could genetically manipulate ependymal cells to produce more myelin and less scar tissue after a spinal cord injury, they could potentially avoid or reverse many of the debilitating effects of this type of injury, the researchers said.

Provided by MIT

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Monday, May 05, 2008

Advances Offer Hope for Spinal Cord Injury Patients

Cell transplantation, physical therapy show promise in restoring function

There is no cure for a spinal cord injury, but much headway has been made in clinical research that could lead to one. Other therapies have helped to restore some function in spinal cord injured patients. A look at some efforts?

The latest in cell transplantation

Cell-based therapies hold the potential for replacing cells and restoring function lost to disease or injury. Among those being developed to help treat spinal cord injuries:

Stem cells are building blocks capable of turning into various cells and tissues found in the body. Embryonic stem cells, in particular, are able to transform into any tissue, given the right biochemical instructions, and could be used to replace spinal cord cells lost during injury. However, human embryonic stem cell research is politically controversial, because culling the cells destroys embryos. Still in the research phase, stem cells have helped paralyzed rodents move again in several ways, including helping to regrow destroyed nerve cells in the spinal cord and successfully restoring myelin, a nerve fiber insulation that helps maintain the electrical conduction required to move.

Olfactory tissue, which covers about one-inch of the upper nasal cavity, contains many cells with regenerative potential, including olfactory ensheathing cells (OECs). Several experiments have found that OECs promote nerve regeneration and may restore function when implanted into an injured spinal cord. These OECs produce insulating myelin sheaths around the damaged nerve cells, encouraging regrowth. While research continues, some scientists, including Portugal's Dr. Carlos Lima, have transplanted olfactory tissue into patients with spinal cord injuries. Preliminary results were published in 2006 in the Journal of Spinal Cord Medicine.

Schwann cells: Another type of "ensheathing" cell, Schwann cells may also help stimulate nerve regeneration of an injured spinal cord. According to Dr. Wise Young, founding director of the W.M. Keck Center for Collaborative Neuroscience at Rutgers University, many laboratories have shown that Schwann cells alone will improve function after spinal cord injury in animals and even more so when they are combined with other therapies, such as OECs.

New advances in physical rehabilitation

Functional electrical stimulation: When connections between the brain and spinal cord are diminished by trauma, the ability to control movement can be eroded or lost. Functional electrical stimulation, or FES, systems can act as a substitute for those lost signals. FES systems apply a small electrical current that stimulates muscle contractions via electrodes that are either taped to the skin or surgically embedded. The contractions help maintain muscle mass, initiate movement in hands or legs or even stimulate the bladder or diaphragm. Dr. John McDonald of Baltimore's Kennedy Krieger Institute uses special exercise bicycles hooked up to FES systems to help paralyzed patients pedal, believing the repetitive activity helps restore function and also may stimulate regrowth of the damaged neural connections. McDonald also used FES in working with the late actor Christopher Reeve.

McDonald says: "We're focused on incremental improvements. What we ... say is this: No one can tell you whether you can walk or not walk. All I can say is doing an activity-based program in today's world is your best chance at meeting the cure halfway.''

Treadmill training uses repetitive motion to try to teach the legs how to walk again. A paralyzed person is suspended in a harness above a treadmill, reducing weight the legs have to bear. As the treadmill starts, therapists move the person's legs in a walking pattern. The theory driving the work is that paralysis causes "learned nonuse" of muscles, but the injured nervous system may be capable of recovery when certain conditions are optimized, including the patterned neural activity that accompanies treadmill walking. (Source: The Christopher and Dana ReeveFoundation Paralysis Resource Center.)

Activity-based, exercise or aggressive physical rehabilitation: Based on the same activity-triggering premise, several centers across the nation are using aggressive exercise, or activity-based therapy, to help restore function in some spinal cord injured patients. Results vary, depending on the patient's level of injury and how much time has passed since the injury.

But researchers like Young, of Rutgers, voice encouragement: "Many of the people who are currently not walking, if trained properly, would be able to walk. What is really necessary is more evidence-based medicine to indicate that these things really work and then to show to the insurance companies that this is an effective therapy so that they will cover it. Hundreds of thousands of peoples' lives would be affected.''

Centers include: Project Walk in Carlsbad, Calif.; BeyondTherapy, at the Shepherd Center in Atlanta; The Center for SCIRecovery at the Rehabilitation Institute of Michigan.

Pharmaceutical

Methylprednisolone, a steroid drug, became a standard treatment for acute spinal cord injury in 1990 when a large-scale clinical trial showed significantly better recovery in patients who were given the drug within the first eight hours after their injury. It appears to reduce the damage to nerve cells and decrease inflammation near the injury site by suppressing activities of immune cells. (Source: National Institute of Neurological Disorders and Stroke.)

Other drug-related research now under way includes: Studies to determine whether Riluzole, now used to treat Lou Gehrig's disease, may protect nerve cells and promote motor recovery when administered after spinal cord injury; and a trial involving the drug Cethrin, which has been found in animal studies to lessen post-traumatic neural cell death.

Gene therapy

Gene therapy carries the potential to provide the injured spinal cord with the specific gene products, or proteins, that it needs to promote functional recovery. Gene therapy is not a current treatment for spinal cord injuries but is being studied with animal models of spinal cord injury. The concept is to transfer into the spinal cord a gene encoding a therapeutic protein, such as a growth factor or an axon guidance molecule, or to transplant cells modified to incorporate the gene. When the gene is expressed, the cell makes the desired protein. (Source: "Spinal Cord Injury: Progress, Promise and Priorities,"' a publication of The Institute of Medicine, an arm of The National Academies.)

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Monday, April 07, 2008

Experimental Russian Stem Cell Treatments Credited for Woman's Progress

Experimental Russian stem cell treatments for spinal injury credited for woman's progress


Notice: The following excerpts are taken from the Grand Rapids Press. A link the the entire article is listed below, and is well worth the time to read.
When Kadi DeHaan took her first steps in December, two years after a car accident forced her into a wheelchair, she did it in typical Kadi style: low-key, nonchalant and with a confident grin.


Apparently, she knew all along she would walk away from her pink and black wheelchair and her customized leg braces, despite a spinal cord injury at chest level and a grim prognosis that she would never walk again.

It happened after two years of intensive therapy and six trips to Russia, where her stem cells were harvested and then injected into her spinal cord to restore nerves.

Kadi's progress is "very much a unique and wonderful thing," said physical therapist Sandy Burns, director of the Center for Spinal Cord Injury Recovery in Rockford, a clinic affiliated with the Detroit Medical Center.

No one can say for sure if nearly two years of experimental treatments or hours upon hours of physical therapy -- a trio of three-hour sessions every week -- led Kadi to where she is today.

Probably both, said Burns, whose clients sometimes head to Russia or Portugal or China for treatments that aren't approved in the U.S. and generally aren't covered by insurance.

The physical therapy is a very important component, "but it's definitely Russia," that put Kadi back on her own two feet, Kadi's mom, Bonnie, insisted. "There are just too many coincidences. Kadi knows that what she's got she got from Russia."

After fundraising dollars ran out more than a year ago, Kadi's parents took out a loan to pay for the trips to Russia. The three-year protocol recommended by Moscow doctors will cost in excess of $150,000.

At the time, Kadi had just a bit of feeling in her feet and could walk only with lots of help from custom-built leg braces and a walker.

Since then, she's given up the braces and is "tons stronger" and "a lot more independent," she said. She's a full-time student at Davenport University who quaffs Mountain Dew and confesses to sending text messages during class.

"I've seen a lot of changes. I've seen motor return, sensory return, everything," Kadi said.

She's so convinced of the gains made at the NeuroVita Clinic that she's planning her seventh trip there in August. Quite a change of attitude after she declared the first trip "the worst three weeks of my life."

Burns, who is quick to say her clinic does not endorse any of the alternative treatments, acknowledged that the stem cell injections do seem to make a difference, at least for Kadi.

"Folks that have gone there have, I think, consistently reported that they are noticing changes. They are feeling more," Burns said.

She tempers her optimism with the reality of what she sees every day: some of her clients will never accomplish half as much as Kadi has. Progress often depends upon the severity of the spinal injury, not just the region of the spine that was damaged.

That's why Burns doesn't make predictions about what her clients will eventually accomplish. But of course, she hopes Kadi continues to make great strides.




The Neurovita Clinic


Where: Moscow, Russia
What: Treats spinal cord injuries, degenerative disorders and some cancers with patient's own stem cells, which are harvested, grown and re-injected. Clinic moved away from use of embryonic stem cells because of compatibility issues.
Insurance: Because treatment is experimental and not performed here, U.S. insurance policies don't cover it.
Website: neurovita.ru/eng_index.html

The NeuroVita clinic was founded by neurologist Andrey S. Bryukhovetskiy in 2002. It's located on the campus of the Russian State Medical University and can accommodate 35 patients.

The clinic dabbled in embryonic stem cell treatments but now uses only autologous material -- that which is obtained from the patient -- because there are no problems with compatibility, not to mention politics and religion, according to the Web site.

About 11 of every 100 patients with spinal cord injuries walk again after the stem cell treatments, Bryukhovetskiy told them.

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Friday, September 07, 2007

Skin Stem Cells Used to Mend Spines of Rats

Toronto research shows injured subjects walking better after injections

A Toronto-led team of researchers has found a way to use stem cells derived from skin to treat spinal cord injuries in rats.

The finding lends promise to the idea that stem cells could one day be used to heal spinal cord injuries in humans, helping thousands to walk again.

Injured rats injected with skin-derived stem cells regained mobility and had better walking co-ordination, according to the study published yesterday in the Journal of Neuroscience. The skin-derived stem cells, injected directly into the injured rats' spinal cords, were able to survive in their new location and set off a flurry of activity, helping to heal the cavity in the cord.

Freda Miller, a senior scientist at The Hospital for Sick Children and lead author of the study, said skin-derived stem cells have some advantages over other stem cell types. Scientists who use skin to generate stem cells do not need to use embryos, for example, and skin-derived stem cells can potentially be harvested from patients themselves, she said.

"You can imagine a scenario for people with spinal cord injuries, that maybe, just maybe, we could take a piece of their skin, grow the cells up and transplant them (the patient) with their own cells," she said. "You wouldn't have to give them immunosuppressive drugs. That's a tremendous clinical advantage if it comes true."

Miller and her colleagues from The Hospital for Sick Children and the University of British Columbia have been exploring the possibilities of using skin to derive stem cells since 2001.

Over the course of their research, the team found that skin-derived stem cells share characteristics with embryonic neural stem cells, which generate the nervous system. They also showed skin-derived stem cells can produce Schwann cells, a cell type that creates a good growth environment to repair injured central nervous system axons ? the long nerve cell fibres that conduct electrical impulses between nerves ? and that these Schwann cells put down myelin along the injured spinal cord. Like the insulation around an electrical cord, myelin wraps around nerves, creating a sheath that helps quickly conduct nerve impulses.

Miller said the next step was to see whether transplanting the Schwann cells directly into spinal cords would help treat injured rats.

To test their hypothesis, Miller and her team generated stem cells from the skin of rats and mice and forced them to differentiate into Schwann cells, which were then transplanted into the rats. After 12 weeks, the rats were able to walk better, with more co-ordination.

Miller said the cells thrived within the injured spinal cord. Before treatment, the injured rats had a cavity in their spinal cord, a result of their injury. But after treatment, Miller said the Schwann cells had created a bridge that spanned the cavity, and helped nerves grow through the bridge.

The next step is to see whether stem cells derived from human skin can produce similar results.

"We are highly encouraged," said Miller.

Story by: Megan Ogilvie

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Friday, August 24, 2007

Stem-cell therapy: Cure or hoax in China?

'Some get miracles'; others are skeptical

The website for Beike Biotechnology bursts with stories that can only be categorized as medical miracles: a paraplegic can move his legs again; a man with muscular dystrophy can carry a cup of water, a stroke victim can speak.

These tales of ailments treated come from all over the world - England, Hungary, Russia, Canada - and back the healing claims of a controversial Chinese treatment that purports to cure the incurable.

"I saw miracles every day I was there," says Leslie Wells, who flew to China in April, 11 years after a swimming pool accident rendered her arms and legs limp. "It can be a crapshoot. Some people get miracles, some people get nothing."

Doctors at Beike - based in Shenzhen, China - are treating a host of nerve disorders with stem-cell therapy, a procedure still under early clinical trials in much of the Western world. In just two years, doctors at Beike have injected stem cells from umbilical cords into the spines of nearly 1,000 patients from outside the country. Roughly 30 of those patients came from Canada, according to a Beike spokeswoman.

Approval for such treatments in Canada is years away, and the medical community here stands firmly opposed to people seeking them in China, citing possible health risks. In a research paper published in Neurorehabilitation and Neural Repair last year, several doctors in Canada and the United States followed up with patients of Hongyun Huang, who has been offering stem-cell treatments in China for several years. Few of the patients had improved since returning from China.

"If it sounds too good to be true, it's too good to be true," says Michael Rudnicki, Canada Research chair in molecular genetics at the University of Ottawa.

But increasing numbers of Canadians are sidestepping domestic regulations and venturing to China in hopes of a cure.

Ms. Wells, of Milton, Ont., first heard about Beike in a news story she read about two Ontario women who had suffered spinal-cord injuries in a car accident and then received the stem-cell treatments in China with some success.

As she flipped through the beaming testimonials on the company's website, it crossed her mind that the whole thing might be a scam. She just wanted a cure to nerve pain so crippling that "no painkiller known to man would help."

Her spine specialists warned her against it.

Eventually, she decided. "I was like, all right, what do I have to lose? Just a little money."

Ms. Wells paid $23,000 for the procedure and travelled to Nanshan Hospital in China, where she received six injections teeming with stem cells into her spinal fluid. Beike says the stem cells repair damaged nerves.

After her second injection, the pain that had made jobs and school seem impossible, was nearly gone.

"On a scale of one to 10, it went from like a nine down to a two. I haven't taken a single painkiller since."

She's not alone in her praise of the injections. With his speech and balance failing, George Arruda, an Ancaster, Ont., landscaper with ataxia, flew to Nanshan for four spine injections and two IV drips.

Ataxia is a progressive disorder that prematurely kills the nerve cells responsible for balance and co-ordination, and is one of the long list of neural conditions that Beike will treat. That list also includes epilepsy, ALS, cerebral palsy, spinal-cord injury and strokes,

Mr. Arruda knew it was an uncertain therapy, but his wife had recently given birth to a daughter. "I just wanted to be a healthy strong dad for her."

Before the trip, he could get around only with the aid of a walker. One night, about midway through his treatment, he was surprised to find himself walking to the bathroom unassisted.

"Immediately, I was about 20 per cent better," he says.

Since returning to Canada in February, he's had a relapse of symptoms. But he says that was probable considering the degenerative nature of ataxia. He's now looking at other stem-cell treatments.

Western medical experts chalk up the positive testimonials to the placebo effect. "We can give people a sugar pill and tell them it will get rid of all their pain and they'll insist that it works," Dr. Rudnicki says, "so I'm highly doubtful of testimonials. If I just spent $30,000 on a procedure, I would want to say it worked too."

Researchers at the University of Alberta are in the midst of studying the proliferating number of companies offering stem-cell cures. So far, they've discovered more than 30 based all over the world.

"The term stem cell has so much currency around the world right now," says Tim Caulfield, Canada Research chair in health law at the University of Alberta and member of a Canadian network of stem-cell researchers. "Even though the scientific community is deeply skeptical, people just associate the term with hope. It's a perfect area for quackery."

Eventually, Dr. Caulfield expects that researchers can use the University of Alberta study to make policy recommendations. "If there is fraud, we want to find it. The people going in for this are often tremendously sick and desperate. We want to ensure they are not being exploited."

Beike is open to the scrutiny. Patients are encouraged to post pictures and blog entries online documenting their time in China. Most depict a pristine hospital with cheery medical staff.

"Most of the doctors who work for us have been trained in Europe or the U.S.," says Kirshner Ross-Vaden, lead medical consultant with Beike's North American operations. "These are people who are leading the entire medical field. We have the nicest hospitals in China. The North American medical establishment is simply behind the times."

Beike says that 86 per cent of their clients show some measure of improvement.

Researchers in Canada say that while clinical trials have begun to look at the possibilities of stem-cell treatments, the therapies won't be available to the public for years - if they actually work.

Until that day comes, researchers here continue to advise against a stem-cell trip to China.

"They are ... putting patients at risk," Dr. Rudnicki says.

By: PATRICK WHITE

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Thursday, June 28, 2007

Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury

Patients Experienced Increased Mobility After Treatment; Preliminary Results Involving 25 Patients Presented at International Society for Cellular Therapy?s Annual Meeting in Sydney

PrimeCell? Therapeutics LLC announced that it provided research support and pre-clinical studies for a clinical trial to assess the safety, feasibility and efficacy of implanting autologous bone marrow stem cells into spinal cord injury (SCI) patients.

Dr. Luis Geffner presented a preliminary report at the 13th Annual Meeting of the International Society for Cellular Therapy, held here June 24-27. From May 2006 to January 2007, 25 patients with SCI were treated at Luis Vernaza Hospital in Guayaquil, Ecuador. They were treated with autologous bone marrow stem cells ? meaning the cells were extracted from the patients' own bone marrow.

Fifteen patients (60 percent) could stand up, ten patients (40 percent) could walk on the parallels with braces, seven (28 percent) could walk without braces, and four (16 percent) could walk with crutches. Patients demonstrated improvements in sensitivity, motility, bladder sensation, even controlling sphincters, erection and ejaculation. No adverse event was observed.

"These preliminary results, while encouraging, must be interpreted cautiously and prudently, and we must continue work examining the benefits of surgically implanted autologous bone marrow stem cells to patients with spinal cord injuries," said Geffner, director of the stem cell program at the Junta de Beneficencia de Guayaquil. He emphasized that this work was done with the help and support of the Junta de Beneficencia, Benemerita Sociedad de Lucha Contra el Cancer (SOLCA), and with the research support and pre-clinical studies performed by stem cell biologist and senior author Francisco Silva and his team at PrimeCell Therapeutics, based in Irvine, Calif.

The study included in vitro (laboratory tests), pre-clinical (animal) and clinical (human) data.

"There is evidence demonstrating significant improvement in the quality of life of patients receiving the treatment, including spinal cord regeneration and additional clinical improvements following these stem cell transplants,? said Silva, vice president of research and development for PrimeCell Therapeutics. ?More research is needed, of course, but this is very encouraging. Our ultimate goal, as always, is not just research ? but research that will lead to timely viable therapies."

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Thursday, May 10, 2007

Stem Cells Closer to Trials

Despite the limitations on federal funding for embryonic stem cell research, two companies recently said they are close to entering clinical trials with the versatile cells.

Geron plans to file an investigational new drug application with the Food and Drug Administration by the end of the year for using cells derived from embryonic stem cells for treating spinal injuries.

Advanced Cell Technology, which previously said it planned to file an IND this year for using stem cell-derived therapies for treating macular degeneration, announced this week it has developed a technique to generate a type of progenitor cell that could move into the clinic in 2008 for treating a variety of ills.

Robert Lanza, Advanced Cell's vice president of medical and scientific affairs, told United Press International that the cells -- called hemangioblasts that his group derived from human embryonic stem cells -- have proven their ability to repair vascular damage in the eyes and limbs of animals. This indicates the cells could prove beneficial for treating heart attacks, reversing vascular damage that now requires limbs to be amputated, and other conditions.

"We're planning to file with the FDA next year to use them in patients," Lanza said.

Advanced Cell's technique is described in the online issue of Nature Methods. Although it's still in the early days, he said the hemangioblasts also could be used to create immune tolerance so the body does not reject the cells as foreign.

"This would allow us to transplant any type of replacement cell or organ generated from a specific stem cell line without rejection," Lanza said. "It would make therapeutic cloning unnecessary and obviate the need for millions of human eggs."

Lanza said animal studies his firm currently has in progress indicate the hemangioblasts could help repair lung damage and generate enough red blood cells for transfusion.

Other potential indications include treating strokes, microvascular complications of diabetes and atherosclerosis.

Advanced Cell, whose California facility could be a benefactor of the $3 billion stem cell program in that state, also may reap the rewards on the other coast where its Worcester, Mass.-based facility is located. Massachusetts Gov. Deval Patrick Tuesday announced his proposal to make $1.25 billion available for funding stem cell and other research in the state over 10 years.

Under the terms of the proposal, the majority of the funding would come from the state, while $250 million would come from private businesses.

UPI could not reach Geron CEO Thomas Okarma by press time Wednesday, but the company has said it anticipate filing an IND for GRNOPC1 for treating spinal-cord injuries around the December timeframe.

GRNOPC1, which consists of oligodendroglial progenitor cells derived from human embryonic stem cells, has been shown to stimulate the regeneration of damaged neurons in pre-clinical studies.

Lazard analyst Joel Sendek, who rates the stock a "hold," notes Geron's products, since they are cellular-based therapies, carry substantially more risk than conventional drugs or protein therapies.

Despite that uncertainty, the company's GRNOPC1 may have an advantage over stem cell-based therapies aimed at other indications.

"We believe the bar for signs of efficacy is low, given that (spinal-cord injury) patients have no other options for restoration of function," Sendek stated in a research report.

However, the FDA is concerned about the potential for stem cell-derived therapies to cause tumors in humans, so Geron will have to overcome that barrier with the agency, Sendek said.

He anticipates the company will file the IND for GRNOPC1 in the fourth quarter and start a phase 1/2 program in the first half of 2008.

The phase 1/2a trial, which Sendek anticipates will take two years to complete, will initially involve 75 patients with spinal-cord injuries. GRNOPC1 cells will be injected into the spinal-cord lesion and the patients will also be given an immunosuppressant drug to prevent rejection of the cells.

Mark Monane, an analyst with Needham, thinks the IND filing for GRNOPC1 and advancement of its other pipeline candidates will be significant events for Geron, but added they probably won't add much value to the stock.

"Given the current technology value of $288 million, we believe that the market has already priced in the expected pipeline progression," Monane stated in a research report. "Going forward, we believe that the stock will perform in line with the overall market until (generation of) further clinical efficacy data from Geron's multiple product candidates."

The company's other candidates include GRN163L for chronic lymphocytic leukemia. A potential catalyst for the stock is Geron's slated presentation of early phase 1/2 data for GRN163L at the Pan Pacific Lymphoma Conference in June.

By STEVE MITCHELL
UPI Senior Medical Correspondent

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