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Category: Stem Cell Therapy

By ALICIA CHANG, AP Science Writer

LOS ANGELES – Dozens of people who were blinded or otherwise suffered severe eye damage when they were splashed with caustic chemicals had their sight restored with transplants of their stem cells — a stunning success for the burgeoning cell-therapy field, Italian researchers reported Wednesday.

The treatment worked completely in 82 of 107 eyes and partially in 14 others, with benefits lasting up to a decade so far. One man whose eyes were severely damaged more than 60 years ago now has near-normal vision.

“This is a roaring success,” said ophthalmologist Dr. Ivan Schwab of the University of California, Davis, who had no role in the study — the longest and largest of its kind.

Stem cell transplants offer hope to the thousands of people worldwide every year who suffer chemical burns on their corneas from heavy-duty cleansers or other substances at work or at home.

The approach would not help people with damage to the optic nerve or macular degeneration, which involves the retina. Nor would it work in people who are completely blind in both eyes, because doctors need at least some healthy tissue that they can transplant.

In the study, published online by the New England Journal of Medicine, researchers took a small number of stem cells from a patient’s, multiplied them in the lab and placed them into the burned eye, where they were able to grow new corneal tissue to replace what had been damaged. Since the stem cells are from their own bodies, the patients do not need to take.

Adult stem cells have been used for decades to cure blood cancers such as leukemia and diseases like sickle cell anemia. But fixing a problem like damaged eyes is a relatively new use. Researchers have been studying cell therapy for a host of other diseases, including diabetes and heart failure, with limited success.

Adult stem cells, which are found around the body, are different from embryonic stem cells which come from human embryos and have stirred ethical concerns because removing the cells requires destroying the embryos.

Currently, people with eye burns can get an artificial cornea, a procedure that carries such complications as infection and glaucoma, or they can receive a transplant cells from a cadaver, but that requires taking drugs to prevent rejection.

The Italian study involved 106 patients treated between 1998 and 2007. Most had extensive damage in one eye, and some had such limited vision that they could only sense light, count fingers or perceive hand motions. Many had been blind for years and had had unsuccessful operations to restore their vision.

The cells were taken from the limbus, the rim around the cornea, the clear window that covers the eye. In a normal eye, stem cells in the limbus are like factories, churning out new cells to replace dead corneal cells. When an injury kills off the stem cells, scar tissue forms over the cornea, clouding vision and causing blindness.

In the Italian study, the doctors removed scar tissue over the cornea and glued the laboratory-grown stem cells over the injured eye. In cases where both eyes were damaged by burns, cells were taken from an unaffected part of the limbus.

Researchers followed the patients for an average of three years and some as long as a decade. More than three-quarters regained sight after the transplant. An additional 13 percent were considered a partial success. Though their vision improved, they still had some cloudiness in the cornea.

Patients with superficial damage were able to see within one to two months. Those with more extensive injuries took several months longer.

“They were incredibly happy. Some said it was a miracle,” said one of the study leaders, Graziella Pellegrini of the University of Modena’s Center for Medicine in Italy. “It was not a miracle. It was simply a technique.”

The study was partly funded by the Italian government.

Researchers in the United States have been testing a different way to use self-supplied stem cells, but that work is preliminary.

One of the successful transplants in the Italian study involved a man who had severe damage in both eyes as a result of a chemical burn in 1948. Doctors grafted stem cells from a small section of his left eye to both eyes. His vision is now close to normal.

In 2008, there were 2,850 work-related chemical burns to the eyes in the United States, according to the Bureau of Labor Statistics.

Schwab of UC Davis said stem cell transplants would not help those blinded by burns in both eyes because doctors need stem cells to do the procedure.

“I don’t want to give the false hope that this will answer their prayers,” he said.

Dr. Sophie Deng, a cornea expert at the UCLA’s Jules Eye Institute, said the biggest advantage was that the Italian doctors were able to expand the number of stem cells in the lab. This technique is less invasive than taking a large tissue sample from the eye and lowers the chance of an eye injury.

“The key is whether you can find a good stem cell population and expand it,” she said.

Irvine, Calif., March 10, 2010 – A clinical research trial of a new treatment to
restore brain cells damaged by stroke has passed an important safety stage,
according to the UC Irvine neurologist who led the effort.

Dr. Steven C. Cramer said patients showed no ill effects after the sequential
administration of growth factors encouraging the creation of neurons in
stroke-damaged areas of the brain. All new drug treatments must pass this safety
stage before doctors can study their effectiveness in subsequent studies.

Results of the phase IIa trial appear on the Web site of Stroke, a journal of
the American Heart Association.

Within two days of suffering ischemic stroke, patients were put on a nine-day
treatment course, starting with three once-daily injections of beta-hCG, a
hormone that triggers the growth of neural stem cells. They then received three
once-daily injections of erythropoietin, a hormone that directs these neural
stem cells to become neurons.

Cramer, associate professor of neurology at UCI, said this combination of growth
factors had been shown in animal studies to engender neuron creation leading to
the recovery of a range of movement.

In the human safety study, he teamed with physicians from UC Irvine Medical
Center; Hoag Memorial Hospital Presbyterian in Newport Beach, Calif.; and the
University of Calgary in Canada. They administered the treatment to 15 patients.
No safety concerns were noted, and a majority of treated patients had minimal or
no disability after three months.

A phase IIb clinical trial is now under way to compare the stroke therapy with
placebo.

The study is supported by Stem Cell Therapeutics a Canadian biotechnology
company that conceived of an approach using this specific sequence of growth
factors and the National Center for Research Resources.

Dr. Centeno is offering stem cell therapies for orthopedic damage with remarkable results. Is this the future of stem cells in the US?

The FDA has yet to approve stem cell therapies for general use in medicine, but that hasn’t stopped doctors in Colorado from providing them anyway. Chris Centeno and John Schultz have boldly formed Regenerative Sciences Inc. in Broomfield, Colorado. RSI provides its patients with the Regenexx procedure, an adult stem cell transplant that uses your own cells (autologous) to treat joint injuries and bone damage. There’s no surgery needed. A needle extracts bone marrow, RSI isolates the stem cells and cultures them in your own blood, and then these cells are injected into the area where they are needed. They’ve treated 348+ patients with 800+ injections and show no signs of slowing down. According to RSI’s own surveys, 89% of their knee patients showed marked improvement, as did 75% of their hip patients! Within months some patients can walk or run in ways they haven’t been able to in years. We’ve seen these kinds of results from stem cell treatments before, but only in horses and dogs. That’s because human stem cell therapies like this one aren’t approved by the FDA. How can Centeno and Schultz flaunt the lack of federal approval? They claim that Regenexx is solely used as a part of their medical practice, only within the state of Colorado, and as such is no more regulated by the FDA than it would be by the FAA or the Department of Motor Vehicles. For hundreds of patients, he and his team are providing a remarkable hope. They’ve brought lab-cultured medical stem cell therapies to the US. Finally.

Stem cells have been a focal point for hype and hope for years now. Besides healing horses and dogs, they have promising effects on diabetes, corneal blindness, even HIV. It’s pretty clear that they’re also the future of organ transplants. Just the news of a stem cell related development or patent will cause a biotech company’s stocks to soar. The FDA, which regulates all interstate drug sales and related clinical trials is not trying to keep Americans from these “miraculous” cures, it’s simply trying to make sure they are safe first. Apparently, that’s taking too long. Medical tourism agencies are starting to cater to those seeking stem cell treatments. Whether or not they are ready for widespread medical use, stem cell therapies are in high demand, not just in the US but around the world. It’s no longer a question of when we will have access to these treatments, it’s a question of how.

A severely damaged knee healed to a remarkable degree. Must be stem cells. RSI is offering adult stem cell therapies in the US. That’s an important first, but what will happen without FDA approval?
Patient’s interested in the Regenexx procedure face what seems to be a fairly standard experience for autologous stem cell transplants. It takes 20-40 minutes to extract the cells from hip bone marrow with limited anesthesia, and blood is also taken. Over a month RSI’s lab will isolate mesenchymal (multipotent) adult stem cells and multiply them until they have 1 to 10 million. Typically, a patient will receive an injection into the treated area once a month for three months. Positive results are sometimes seen quickly (in 1 to 3 months) but will hopefully develop within 6 to 9 months. Importantly, there’s no down time as a result of the procedure. Patients can leave the clinic and go home after each injection. A round of Regenexx (extraction, cultivation, and 3 injections) costs $7000-$8500. Those who produce exceptional numbers of stem cells can use subsequent injections (even in other parts of the body) for around $3500. Most insurances will not cover the treatment.

The fact that RSI isolates and cultures (multiplies) the cells is a big difference from other clinics that offer stem cell therapies. That process allows the lab to create enough mesenchymal stem cells to really have an effect on the area in which they are injected. Many clinics around the world will take blood, marrow, or tissue and then spin out the stem cells in a centrifuge, injecting them back in on the same day. That style of therapy could possibly be effective, but it is far less likely than with a dose of millions of multipotent stem cells. There are several doctors around the US that will provide such ’single-visit’ stem cell therapies, but RSI is offers the lab cultured mesenchymal therapy in the US. Dr. Centeno has confirmed that he’s the only one, that he knows of, openly using this particular procedure in the US.

In the past, many have been very skeptical of stem cell treatment centers in other countries. First, let’s look at the success RSI is selling. Autologous transplants are offered in the hands, hips, knees, shoulders, back (non-spinal cord injury), ankles, and bone fractures. For each of these procedures you can find many ardent and exceptionally encouraging patient testimonials on their website, or their YouTube channel, along with a flood of supportive media.

Overall, RSI is claiming around 80% patient satisfaction according to its own surveys. That’s incredible, especially when you see some of their patients walking and running again on joints that have experienced years of chronic damage. It also seems Centeno and Schultz have the documented evidence to back up the claims for Regenexx’s success. RSI provides case studies for each of its treatments as well as published scientific research. According to my conversation with Centeno, RSI is currently working on a comprehensive statistical analysis of their more popular treatments so they can publish quantitative results in a peer review journal. In other words, they’ll soon publish the hard numbers – X% of patients feel Y% better Z months after the procedure.

Importantly, RSI seems to be upfront with patients about the limits of their own technique. The website FAQ clearly states that not all results will be like the testimonials, and they even have a dedicated page explaining that stem cell therapies won’t work for everyone. Furthermore, RSI has published the largest study of risks and complications associated with stem cell treatments yet produced in the US (N=227). That paper demonstrates the very low harm associated with stem cell therapies – much lower than the alternative surgery(published in Current Stem Cell Research & Therapy). Centeno claims that if we’re really worried that autologous stem cell therapies are going to hurt someone, this paper pretty much shows they won’t.

The concerns most people have with RSI are not medical, they’re political. Many applaud Centeno and Schultz for supplying the public with the cutting edge technology they demand, but worry about the manner in which it has been accomplished. Skirting FDA approval for a technique through the arguments they use opens the gate to a host of problems. If RSI can provide Regenexx because it is a doctor’s procedure not involved in interstate commerce, does that mean someone else can do the same for another treatment? What are the limits of such procedures? How does a patient know if a doctor’s therapy is safe, or effective, if it hasn’t undergone peer review and government inspection?

Doctors and surgeons are developing new procedures all the time. Surgeons will often create new devices for their own use in surgery, doctors routinely try out new dosing regimes, or therapies on their patients. This is part of the medical profession.

Still, it’s possible that even though RSI is doing what many other doctors routinely do (develop a new therapy for use in their own practice) that the federal government could try to bring them to court. The FDA seems to have taken the stance that all stem cells (whether used autologously or not) are drugs. As such, they would need FDA approval, and would likely only be developed by large pharmaceutical companies.

According to Lee Buckler of Cell Therapy Blog, Centeno’s already received a warning letter from the FDA. Centeno clarified that this is actually an “untitled letter” which has no bearing on regulation. He pointed me to this explanation on untitled letters. RSI has faced concerns from the New York Department of Health, and went so far as to pursue a provisional license, even though they are no where near NY state. Clearly RSI is hoping to avoid bureaucratic problems or at least be very prepared for them if they do arise. Perhaps with enough positive results they can avoid legal battles and even convince insurance companies to cover Regenexx.

Hopefully so. Just look as these results. They’re pretty damn amazing. If you accept the success rates, and the possibilities for long term healing…many people need this.

Centeno says he is working with others to provide the framework through which many more patients could receive mesenchymal stem cell therapies. He’s on the board of the International Cell Medicine Society (ICMS) which is working to track stem cell therapy patients through a registry, as well as certify stem cell clinics for practice. Through conferences and seminars, doctors are trained in IVF to work in fertility clinics. Centeno explained to me that a similar practice could instruct and track physicians interested in providing lab cultured autologous stem cell therapies. In other words, the technique used by RSI could become a regularly seen procedure in specialty clinics across the country. That may mean more patients could have access to stem cells soon.

FDA approval is slow, but it’s coming. Athersys has a patent for a stem cell derived drug, other companies have therapies in clinical trials. Those treatments will be here some day. In the meantime, RSI is filling in the gap. Their work may even catch on as a trend. If largely successful, insurance companies may pay for it and the federal government may end up grandfathering Regenexx in at some point. It could happen. What’s certain is that the public demand for stem cell therapies is real, growing, and seemingly justified. When that sort of pressure for a technology exists nobody can keep it down.

There is no conclusive understanding of what the long term effects of stem cells treatments will be. We do not know if a stem cell treatment will be effective 5-10 years after it is administered, and we know of no large study that has conclusively reviewed patients for cancer, or any long term side effect 5-10 years after a stem cell injection. Part of what ICMS is trying to do (reviewing clinics, tracking patients results over the long term) may yield a better understanding in the future.

[Sources: Regenerative Sciences Inc, ABC 7 News in Denver, Lee Buckler , RSI Blog, Current Stem Cell Research & Therapy]

March 2, 2010 – Houston – A heart patient’s own skin cells soon could be used to
repair damaged cardiac tissue thanks to pioneering stem cell research of the
University of Houston’s newest biomedical scientist, Robert Schwartz.

His new technique for reprogramming human skin cells puts him at the forefront
of a revolution in medicine that could one day lead to treatments for
Alzheimer’s, diabetes, muscular dystrophy and many other diseases.

Schwartz brings his ground-breaking research to UH as the Cullen Distinguished
Professor of Biology and Biochemistry and head of UH’s new Center for Gene
Regulation and Molecular Therapeutics. He also is affiliated with the Texas
Heart Institute at St. Luke’s Episcopal Hospital in the Texas Medical Center,
where he is director of stem cell engineering.

“Professor Schwartz’s work will save lives, and his decision to pursue this
pioneering research at UH is a big leap forward on our way to Tier-One status,”
said John Bear, dean of the UH College of Natural Sciences and Mathematics.
“Together with the many other outstanding scientists we’ve assembled here,
Schwartz will help make this university a major player in medical research.”

Schwartz devised a method for turning ordinary human skin cells into heart
cells. The cells developed are similar to embryonic stem cells and ultimately
can be made into early-stage heart cells derived from a patient’s own skin.
These then could be implanted and grown into fully developed beating heart
cells, reversing the damage caused by previous heart attacks. These new cells
would replace the damaged cardiac tissue that weakens the heart’s ability to
pump, develops into scar tissue and causes arrhythmias. Early clinical trials
using these reprogrammed cells on actual heart patients could begin within one
or two years.

Although Schwartz is not the first scientist to turn adult cells into such stem
cells, his improved method could pave the way for breakthroughs in other
diseases. Schwartz’s method requires fewer steps and yields more stem cells.
Armed with an effective way to make induced stem cells from a patient’s own
skin, scientists can then begin the work of growing all kinds of human cells.

For example, new brain cells could treat Alzheimer’s patients or those with
severe brain trauma, or a diabetic could get new insulin-producing cells in the
pancreas. Generating new kidney, lung or liver tissue is also possible, with
scientists even being able to one day grow an entirely new heart or other organ
from these reprogrammed cells. Additionally, Schwartz and his team are working
on turning induced stem cells into skeletal muscle cells to treat muscular
dystrophy.

“We’re trying to advance science in ways folks never even dreamed about,”
Schwartz said. “The idea of having your own bag of stem cells that you can
carry through life and use for tissue regeneration is at the very cutting edge
of science.”

This latest biomedical hire is a major step in the UH Health Initiative, an
effort aimed at having the university become a world-class center for medical
research. Creating new cross-disciplinary academic and health-related research
opportunities for faculty and students is crucial to this initiative, as are
collaborations with other Texas Medical Center member institutions. One of its
top goals is to increase the amount of sponsored research expenditures awarded
to UH, which is a key factor in attaining Tier-One status.

“Dr. Schwartz will expand UH’s expertise in promising new areas of scientific
discovery to alleviate human disease. By recruiting premier scientists like
Schwartz, UH is fast becoming a major player in the regional biomedical research
community,” said Kathryn Peek, assistant vice president of University Health
Initiatives at UH.

Schwartz has decades of experience at the Texas Medical Center. Before coming
to UH, he was director of the Institute of Biosciences and Technology, a
research component of the Texas A&M Health Science Center. He also was a
longtime tenured professor at Baylor College of Medicine and co-directed the
school’s Center for Cardiovascular Development. The new research center
Schwartz heads at UH will be housed in state-of-the-art laboratory facilities at
the university’s Science and Engineering Research Center.

What attracted him to UH was the commitment of administrators and faculty to
making the university a premier center for biomedical research. His hiring
comes just a year after the arrival of Jan-Åke Gustafsson, a world-renowned
scientist and cancer researcher. They join other leading UH faculty, ranging
from biochemists to computer scientists and mathematicians, who are deeply
involved in cutting-edge medical research.