According to the Christopher and Dana Reeve Foundation, nearly one in 50 people are living with paralysis.

Until now, there wasn't much hope.

But, a new study involving stem cells has doctors and patients excited.

Two years ago, Brenda Guerra's life changed forever.

"They told me that I went into a ditch and was ejected out of the vehicle," says Brenda.

The accident left the 26-year-old paralyzed from the waist down and confined to a wheelchair.

"I don't feel any of my lower body at all," says Brenda.

Brenda has traveled from Kansas to UC San Diego to be the first patient to participate in a ground-breaking safety trial, testing stem cells for paralysis.

"We are directly injecting the stem cells into the spine," says Dr. Joseph Ciacci, a neurosurgeon at UC San Diego.

The stem cells come from fetal spinal cords. The idea is when they're transplanted they will develop into new neurons and bridge the gap created by the injury by replacing severed or lost nerve connections. They did that in animals and doctors are hoping for similar results in humans. The ultimate goal: to help people like Brenda walk again.

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Stem cell procedures for paralysis patients

Damage to neural tissue is typically permanent and causes lasting disability in patients, but a new approach has recently been discovered that holds incredible potential to reconstruct neural tissue at high resolution in three dimensions. Research recently published in the Journal of Neural Engineering demonstrated a method for embedding scaffolding of patterned nanofibers within three-dimensional (3D) hydrogel structures, and it was shown that neurite outgrowth from neurons in the hydrogel followed the nanofiber scaffolding by tracking directly along the nanofibers, particularly when the nanofibers were coated with a type of cell adhesion molecule called laminin. It was also shown that the coated nanofibers significantly enhanced the length of growing neurites, and that the type of hydrogel could significantly affect the extent to which the neurites tracked the nanofibers.

"Neural stem cells hold incredible potential for restoring damaged cells in the nervous system, and 3D reconstruction of neural tissue is essential for replicating the complex anatomical structure and function of the brain and spinal cord," said Dr. McMurtrey, author of the study and director of the research institute that led this work. "So it was thought that the combination of induced neuronal cells with micropatterned biomaterials might enable unique advantages in 3D cultures, and this research showed that not only can neuronal cells be cultured in 3D conformations, but the direction and pattern of neurite outgrowth can be guided and controlled using relatively simple combinations of structural cues and biochemical signaling factors."

The next step will be replicating more complex structures using a patient's own induced stem cells to reconstruct damaged or diseased sites in the nervous system. These 3D reconstructions can then be used to implant into the damaged areas of neural tissue to help reconstruct specific neuroanatomical structures and integrate with the proper neural circuitry in order to restore function. Successful restoration of function would require training of the new neural circuitry over time, but by selecting the proper neurons and forming them into native architecture, implanted neural stem cells would have a much higher chance of providing successful outcomes. The scaffolding and hydrogel materials are biocompatible and biodegradable, and the hydrogels can also help to maintain the microstructure of implanted cells and prevent them from washing away in the cerebrospinal fluid that surrounds the brain and spinal cord.

McMurtrey also noted that by making these site-specific reconstructions of neural tissue, not only can neural architecture be rebuilt, but researchers can also make models for studying disease mechanisms and developmental processes just by using skin cells that are induced into pluripotent stem cells and into neurons from patients with a variety of diseases and conditions. "The 3D constructs enable a realistic replication of the innate cellular environment and also enable study of diseased human neurons without needing to biopsy neurons from affected patients and without needing to make animal models that can fail to replicate the full array of features seen in humans," said McMurtrey.

The ability to engineer neural tissue from stem cells and biomaterials holds great potential for regenerative medicine. The combination of stem cells, functionalized hydrogel architecture, and patterned and functionalized nanofiber scaffolding enables the formation of unique 3D tissue constructs, and these engineered constructs offer important applications in brain and spinal cord tissue that has been damaged by trauma, stroke, or degeneration. In particular, this work may one day help in the restoration of functional neuroanatomical pathways and structures at sites of spinal cord injury, traumatic brain injury, tumor resection, stroke, or neurodegenerative diseases of Parkinson's, Huntington's, Alzheimer's, or amyotrophic lateral sclerosis.

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The work was carried out at the University of Oxford and the Institute of Neural Regeneration & Tissue Engineering, a non-profit charitable research organization.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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3-D neural structure guided with biocompatible nanofiber scaffolds and hydrogels

GERMANTOWN, Md., March 16, 2015 /PRNewswire/ -- Neuralstem, Inc. (NYSE MKT: CUR) (the "Company" or "Neuralstem") today reported its financial results for the fourth quarter and year ended December 31, 2014.

"Neuralstem has progressed into a clinical development stage company focused on the central nervous system (CNS)," said Richard Garr, Neuralstem President and CEO. "During 2014 we added two established industry leaders as Independent Directors, Catherine Angell Sohn, Pharm.D. and Sandford Drexel Smith. Dr. Sohn is the former Senior Vice President of Business Development and Strategic Alliance, GSK Consumer Healthcare, at GlaxoSmithKline. Mr. Smith is the former Executive Vice President of Genzyme Corporation. The Company moved forward two lead clinical assets: our small molecule neurogenic drug candidate NSI-189 and our spinal derived neural stem cell therapeutic candidate NSI-566. We established and/or grew clinical research programs with leading investigators at Emory University, University of California, San Diego (UCSD), University of Michigan and Massachusetts General Hospital. Our investigators published and presented proof of principle data in both lead assets as highlighted below. In 2015, we plan to begin clinical development of our NSI-189 small molecule drug in a second indication for the treatment of cognitive deficit from schizophrenia, and we plan to initiate a Phase II clinical trial for the ongoing development program for the treatment of major depressive disorder (MDD). The cell therapy programs in amyotrophic lateral sclerosis (ALS), chronic spinal cord injury (cSCI) and stroke will also move forward. We expect this to be another important year continuing our development and progress across both platforms."

2014 Clinical Program and Business Highlights

Neurogenic Small Molecule Platform Clinical Development

Cell Therapy Platform Clinical Development

NSI-566 spinal cord-derived stem cell therapy under development for the treatment of ALS

NSI-566 spinal cord-derived cell therapy under development for the treatment of cSCI

NSI-566 spinal cord derived stem cell therapy under development for the treatment of motor deficits in stroke

NSI-532.IGF second generation gene engineered cell therapy

2014 Business Highlights

Continued here:
Neuralstem Reports Fiscal 2014 Fourth Quarter Financial And Year-End Business Results

GERMANTOWN, MD, March 12, 2015 -- Neuralstem, Inc. (NYSE MKT: CUR) announced top line data from the Phase II trial of NSI-566 spinal cord-derived neural stem cells under development for the treatment of amyotrophic lateral sclerosis (ALS). The study met primary safety endpoints. The maximum tolerated dose of 16 million transplanted cells and the surgery was well tolerated.

Secondary efficacy endpoints at nine months post-surgery indicate a 47% response rate to the stem cell treatment, as measured by either near-zero slope of decline or positive slope of ALSFRS score in seven out of 15 patients and by either a near-zero decline, or positive strengthening, of grip strength in seven out of 15 patients. Grip strength is an indicator of direct muscle strength of the lower arm. ALSFRS is a standard clinical test used to evaluate the functional status of ALS patients. The average ALSFRS score for responders at 9 months after treatment was 37. Non-responders scored an average of 14. These scores represent 93%, versus 35%, of the baseline score retained, respectively, by the responders versus non-responders at 9 months, which is a statistically significant difference. As measured by an average slope of decline of ALSFRS, responders' disease progression was -0.007 point per day, while non-responders' disease progression was -0.1 per day, which was again statistically significant. Lung function as measured by Seated Vital Capacity shows that responder patients remained within 94% of their starting scores, versus 71% for non-responder patients. The trial met its primary safety endpoints. Both the surgery and cells were well-tolerated, with one patient experiencing a surgical serious adverse event.

"In this study, cervical intervention was both safe and well-tolerated with up to 8 million cells in 20 bilateral injections," said Karl Johe, PhD, Neuralstem Chief Scientific Officer. "The study also demonstrated biological activity of the cells and stabilization of disease progression in a subset of patients. As in the first trial, there were both responders and non-responders within the same cohort, from patients whose general pre-surgical presentation is fairly similar. However, we believe that through the individual muscle group measurements, we may now be able to differentiate the responders from the non-responders.

"Our therapy involves transplanting NSI-566 cells directly into specific segments of the cord where the cells integrate into the host motor neurons. The cells surround, protect and nurture the patient's remaining motor neurons in those various cord segments. The approximate strength of those remaining motor neuron pools can be measured indirectly through muscle testing of the appropriate areas, such as in the grip strength tests. We believe these types of endpoints, measuring muscle strength, will allow us to effectively predict patients that will respond to treatment, adding a sensitive measure of the therapeutic effects after treatment. Testing this hypothesis will be one of the primary goals of our next trial." The full data is being compiled into a manuscript for publication.

"We believe the top-line data are encouraging," said Eva Feldman MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System, and an unpaid consultant to Neuralstem. "We were able to dose up to 16 million cells in 40 injections, which we believe to be the maximum tolerated dose. As in the first trial, the top-line data show disease stabilization in a subgroup of patients. Perhaps equally as important, we believe the top-line data may support a method of differentiating responders from non-responders, which we believe will support our efforts as we move into the next, larger controlled trial expected to begin this summer."

"The top-line data look very positive and encouraging. If this proportion of patients doing well after treatment can be corroborated in future therapeutic trials, it will be better than any response seen in any previous ALS trials," said site principal investigator, Jonathan D. Glass, MD, Director of the Emory ALS Center. "Elucidating which factors define a patient who may have a therapeutic response to the stem cell treatment will be the next key challenge. We are hopeful that a set of predictive algorithms can be established to help pre-select the responders in our future trials."

"We were very excited to participate as a site in this clinical trial," said Merit Cudkowicz, MD, Chief of Neurology, Massachusetts General Hospital and Co-Chair of the Northeast ALS Consortium (NEALS). "We are hopeful with respect to the top-line results and we need to move swiftly and safely forward to confirm the responder effect and identify people who might benefit from this treatment approach."

The open-label, dose-escalating trial treated 15 ambulatory patients, divided into 5 dosing cohorts, at three centers, Emory University Hospital in Atlanta, Georgia, the ALS Clinic at the University of Michigan Health System, in Ann Arbor, Michigan, and Massachusetts General Hospital in Boston, Massachusetts, and under the direction of principal investigator (PI), Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. Dosing increased from 1 million to 8 million cells in the cervical region of the spinal cord. The final trial cohort also received an additional 8 million cells in the lumbar region of the spinal cord.

The company anticipates commencing a later-stage, multicenter trial of NSI-566 for treatment of ALS in 2015. Neuralstem has received orphan designation by the FDA for NSI-566 in ALS.

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Excerpt from:
Neuralstem announces topline results of Phase II ALS trial

Pioneering treatment has allowed wheelchair-bound patients to run again Patient given high dose of chemotherapy to wipe out faulty immune system Therapy then uses person's own stem cells to fight the devastating disease It may be the first ever treatment tosuccessfullyreverse symptoms of MS

By Fiona Macrae for the Daily Mail

Published: 13:27 EST, 1 March 2015 | Updated: 02:54 EST, 2 March 2015

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Britons left wheelchair-bound by multiple sclerosis can walk, run and even dance again after being given a pioneering stem cell treatment.

Doctors have described the recoveries as miraculous, while patients say they have been given their lives back.

The treatment uses a patients own stem cells the bodys master cells to fight the disease.

Recovery: MS sufferer Holly Drewerybecame wheelchair-bound after the birth of daughter Isla, but thanks tothe stem cell transplant shecan dance, run and chase after Isla in the park

Continued here:
MS stem cell treatment hailed 'miraculous' as patients make dramatic recovery

A new stem-cell treatment that reboots the entire immune system is enabling multiple sclerosis sufferers to walk, run and even dance again, in results branded "miraculous" by doctors.

Patients who have been wheelchair-bound for 10 years have regained the use of their legs in the ground-breaking therapy, while others who were blind can now see again. The treatment is the first to reverse the symptoms of MS, which is incurable, and affects about 100,000 people in Britain.

The two dozen patients who are taking part in the trials at the Royal Hallamshire Hospital, Sheffield, and Kings College Hospital, London, have effectively had their immune systems "rebooted". Although it is unclear what causes MS, some doctors believe it is the immune system itself that attacks the brain and spinal cord, leading to inflammation pain, disability and, in severe cases, death.

In the new treatment, specialists use a high dose of chemotherapy to knock out the immune system before rebuilding it with stem cells taken from the patient's own blood.

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"Since we started treating patients three years ago, some of the results we have seen have been miraculous," Prof Basil Sharrack, a consultant neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, said.

"This is not a word I would use lightly, but we have seen profound neurological improvements."

Holly Drewry, 25, of Sheffield, was wheelchair bound after the birth of her daughter, Isla, two years ago. She claims the new treatment has transformed her life.

"It worked wonders," she said. "I remember being in the hospital ... after three weeks, I called my mum and said: 'I can stand'. We were all crying. I can run a little bit, I can dance. I love dancing, it is silly but I do."

However, specialists warn that patients need to be fit to benefit from the new treatment.

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'Miraculous' stem-cell treatment reverses symptoms of multiple sclerosis

By Ashley Dunkak @AshleyDunkak

CBS DETROIT Murray Howe, the head of the radiology department at ProMedica Toledo Hospital, understands the skepticism of those who question the stem cell treatment his father Gordie, also known as Mr. Hockey, received in December in Tijuana, Mexico.

Gordies health had been slowly declining even before the stroke he suffered in late October, and he was essentially bedridden when Murray and his brother Marty took him to Mexico to participate in a clinical trial. They did not have high hopes he was so far gone, Murray recalled but after each step of the two-part process, Gordie improved rapidly, once again able to walk and talk, repossessed of his wit and humor. Murray and his siblings were floored. So were the therapists who had been working with Gordie after his stroke.

Some physicians have scoffed at the idea of stem cells helping an individual who has had a stroke, but Murray a doctor himself says his fathers recovery after treatment opened his eyes to stem cells as a potential game-changer.

Speaking as a medical professional, its so frustrating when you cant really do anything for a patient, said Howe, the head of the radiology department at Toledo Hospital. You give them kind of a death sentence and you say, Well thats all you get. Theres nothing we can really offer. Its so sad. So now to be able to have on the brink of some huge hope for these patients is really, really exciting. As a medical professional, to me, theres never been anything more exciting in my entire career than this.

Murray does not blame people for being skeptical, and he agrees more research on the capabilities of stem cells is needed to show definitively what they can do. To say Murray is optimistic, however, would be a serious understatement.

Theres quite a few individuals out there who are calling themselves stem cell experts or this or that, kind of saying that theres no data to support that stem cells work on ischemic strokes, but thats really not true at all, Murray said. Theres at least 50 clinical studies that are going on across the world that are demonstrating its safety and working on demonstrating its efficacy, and the preliminary results on the ones that Ive seen are tremendous, so the data is clearly there. I think that people across the world in the next couple years are going to be as blown away as I was with our father when they see the power of stem cells and what they do for patients with not just stroke but with dementia and traumatic brain injuries and spinal cord injuries.

My dads case is by no means the only one, Murray continued. Hes kind of like in the middle. Theres examples of patients that have had a far greater result. Im so thrilled for my dad, but by no means was my dad a fluke or a random event. The studies are ongoing, and I think the point of any of the, I guess, naysayers is that Gordie Howe may be anecdotal and we need more research, and I totally agree with that. In fact, based on what weve seen with my father, I would say that we as a country and as a world should make a concerted effort to put as much time and energy as we can into investigating the power of stem cells because I really think that based on what Im seeing this is going to be a game-changer for medicine and a game-changer for quality of life for so many people that have non-option diseases like stroke or dementia.

Heading to Tijuana for treatment was a last-ditch effort to save Gordie, but it was not one the family undertook on a whim, Murray said.

Im well aware of hucksters and con games and this type of thing, and our family has never been about traveling the world to find the miracle cure, Murray said. Im a very mainstream physician. Ive always relied heavily on data and on long-term studies to prove the safety and efficacy of any treatment. For our father, we just our goal has always just been quality of life and comfort. When my mom was sick with her dementia that was our only priority was just keep her comfortable, keep her healthy, as healthy as possible, and keep her safe, and that was it. We had a number of people contact us saying, You know, we could help your mom with this pill and that pill, and I looked at everything that anybody presented to us, but to me there was nothing that showed any data that would made me want to experiment, if you will, with my mom.

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Gordie Howes Son Says Dads Recovery No Fluke, Excited For Future Of Stem Cell Treatment

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Treatment for the crippling condition is currently limited to basic pain relief or complex joint replacement surgery.

But trials using stem cells have shown astonishing results with tissue almost as good as new after just three months.

Professor Sue Kimber, who led the research, said: This work represents an important step forward in treating cartilage damage using embryonic stem cells to form new tissue.

It may offer a new line of therapy for people with crippling joint pain and we now need this process to be developed for patients.

Osteoarthritis occurs when cartilage at the ends of bones wears away causing severe pain and stiffness.

Researchers say the latest experiments show the procedure could potentially be a safe and effective treatment for more than eight million people who suffer from jointdamage and inflammation.

In the experiments, led by teams at Manchester University and Arthritis Research UK, discarded embryonic stem cells from IVF clinics were transformed into cartilage cells.

These were transplanted into rats with defective joints.

Tests showed the high-quality artificially grown tissue quickly aided the repair of the joint.

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Arthritis cure is on the way: Scientists make new breakthrough using embryonic stem cells

For the last week, an Italian neurosurgeon has been executing a full-blown media offensive, talking up his plan to stitch one persons head to another persons body. If the powers-that-be would just get over their ethical queasiness, Sergio Canavero of the Turin Advanced Neuromodulation Group says he could accomplish the feat by 2017.

But full-body transplants arent so crazy. In fact, it might surprise you that there was a successful operation as far back as 1818. Well, successful if you ignore that the transplantee freaked out and murdered his doctors family. Oh wait. That was Frankenstein. I take it back, full body transplants are totally crazy.

What the hell, going to the moon was crazy too, right? And a maybe-crazy-but-what-the-hell moonshot is exactly how Canavero sees his plan to help patients with severe physical impairments. Why did the US and the Soviet Union just vie for being the first to space? Because it is about measuring dicks. We want to demonstrate as a country, to say: I am the best, he says. Canaveros latest paper glosses over questions of ethics and practicality and tacklesthe trickiest aspect of the head-swapping procedure: The spinal splice.

Canaveros plan focuses on sewingtwo people together by their spinal cords. (Hooking up the rest of the utilitiesblood vessels, airways, blood vesselsis incredibly difficult, but trifling in comparison.) Step one is to sever the cords with a special, ultra-thin blade. Canavero rightly notes most cases of spinal trauma are well, traumatic: Snapping your neck on a skateboard ramp is bound to leave the spinal cord in an untidy condition. Those nerve cells scar, and scarring would impede their regeneration (if cells in the central nervous system could regeneratewell get to that in a sec). A clean wound, on the other hand, heals cleanly. Canavero likens those million sharply severed neurons to spaghetti. Italians adore spaghetti, I love spaghetti, and spaghetti is what is called for here, he says.

The job of fusing those spaghetti-like spinal sections together falls to a substance called polyethylene glycol. This stuff has actually been pretty good at repairing the motor functions in rats with spinal traumathough even the kindest critic will point out that successful rat experiments are a far cry from proving that the stuff will repair human spines. Still, Canavero is raring to go. I have enough animal data, he says. Give me a brain dead organ donor. Say someone is in a traumatic car accident, and doctors say that he cannot be saved. In the time between when the persons family says its OK to pull the plug and the moment the doctors actually do so, Canavero asks for three to four hours. I sever the spinal cord, add polyethylene glycol, and start measuring electrophysiological responses, he says.

After surgery (and during it, one hopes), Canavero will keep the patient in a coma. He estimates it will take about at least two weeks for the first axons to beginlacing themselves together, at which point the patient can be revived. Throughout the coma and for some time after, Canavero will bathe the spinal splice with a mild electrical current. This is not a free Frankenstein joke from the good doctor: Its actually a method thats seen surprisingly promising results healingrealhuman patients with spinal trauma. Canavero is confident that this will keep the muscle cells operational. Combined with physical therapy, Canavero estimates his as-yet-unchosen patient (any volunteers?) will be back on her (new) feet in about a year.

In case this wasnt entirelyclear: Canaveros plan is insane. Like, James Bond villain insane. And its not just because his plan fits together like a Voltron of bad science (which it does). Its kind of a bummer, actually, because his plan couldmaybework, if he was given free rein to cut and sew living peoples heads to dead peoples bodies until he got it right. But besides ethics, theres an unfortunate fact of biology standing in his way: The central nervous system in higher vertebrateslike humansdoes not regenerate. Hes insane. You cant put a head on somebody else! says Binhai Zhang, a neurosurgeon at UC San Diego. The reason why goes down to your DNA. The genes in a mature mammalian central nervous system that control regeneration are repressed, says Michael Beattie, a professor of neurosurgery at UC San Francisco. Theyll stay that way, no matter how much you treat the spinal cord with polyethylene glycol and electrical currents. (Although, hey, who wants to work on un-repressing those genes?)

Nobody knows for sure why the cells in your brain and spine arent wired for regrowth. After all, your peripheral nervous systemthe circuitry for every other part of your bodyconducts electrical impulses in exactly the same way, but its genes can code for self-repair. Beattie says this may have to do the fact the spine and brain contain the circuitry coded for movement, not just for conducting signals. Spinal cells must knit themselves together in super-complex configurations in order to command the motor functions youve learned over a lifetime. Once the connections are made, you dont want the wrong connections getting created, he says.

The only reliable way to induce spinal cell regrowth in higher order vertebrates is with stem cell therapy. Last year scientists showed pluripotent stem cells could regrow damaged spinal cordsbut only in rats. Mark Tuszynski studies stem cells in spinal injury at UC San Diego, and he says even with this advance the research community is years away from attempting suchtreatments on humans. Its not at the stage yet where there can be meaningful advances in clinical trials, he says. Plus stem cells will need help, in the form of drugs that knock down natural regeneration inhibitors that your body creates (because cancer), and still more drugs to keep your body from creating scar tissue around the wound. (Though in fairness, thats the idea behind Canaveros super-thin knife.) All of this research remainsyears away from clinical application.

And this slow, careful tempodo no harm being a hallmark of western medicineis what drives Canaveros bold assertion that he will have a successful head transplant in 24 months. There are all these people who tell you: Who is this guy who can do this in two years? When you go public with something like this, you have to have two balls like this. There are people who are not so strong-balled and will just get crushed by the critics. But I love the critics. This is a feat of theoretical neuroscience and the evidence is there and its going to work. In case you need clarification, his main argument there is Haters gonna hate.

Excerpt from:
Full-Body Transplants Are a Crazy, Wildly Unethical Idea

A new stem cell treatment that reboots the entire immune system is enabling multiple sclerosis sufferers to walk, run and even dance again, in results branded "miraculous" by doctors.

Patients who have been wheelchair-bound for 10 years have regained the use of their legs in the ground-breaking therapy, while others who were blind can now see again. The treatment is the first to reverse the symptoms of MS, which is incurable, and affects about 100,000 people in Britain.

The two dozen patients who are taking part in the trials at the Royal Hallamshire Hospital, Sheffield, and Kings College Hospital, London, have effectively had their immune systems "rebooted". Although it is unclear what causes MS, some doctors believe that it is the immune system itself that attacks the brain and spinal cord, leading to inflammation pain, disability and, in severe cases, death.

In the new treatment, specialists use a high dose of chemotherapy to knock out the immune system before rebuilding it with stem cells taken from the patient's own blood. "Since we started treating patients three years ago, some of the results we have seen have been miraculous," Prof Basil Sharrack, a consultant neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, told The Sunday Times.

"This is not a word I would use lightly, but we have seen profound neurological improvements." Holly Drewry, 25, of Sheffield, was wheelchair bound after the birth of her daughter Isla, now two. But she claims the new treatment has transformed her life.

"It worked wonders," she said. "I remember being in the hospital... after three weeks, I called my mum and said: 'I can stand'. We were all crying. I can run a little bit, I can dance. I love dancing, it is silly but I do. " However, specialists warn that patients need to be fit to benefit from the new treatment. "This is not a treatment that is suitable for everybody because it is very aggressive and patients need to be quite fit to withstand the effects of the chemotherapy," warned Prof Sharrack.

The research was published in the Journal of the American Medical Association.

Continue reading here:
'Miraculous' stem cell treatment may reverse symptoms of multiple sclerosis

Stem-cell advocates pin their hopes on an artificial pancreas to treat diabetes.

Fourteen years ago, during the darkest moments of the stem-cell wars pitting American scientists against the White House of George W. Bush, one group of advocates could be counted on to urge research using cells from human embryos: parents of children with type 1 diabetes. Motivated by scientists who told them these cells would lead to amazing cures, they spent millions on TV ads, lobbying, and countless phone calls to Congress.

Now the first test of a type 1 diabetes treatment using stem cells has finally begun. In October, a San Diego man had two pouches of lab-grown pancreas cells, derived from human embryonic stem cells, inserted into his body through incisions in his back. Two other patients have since received the stand-in pancreas, engineered by a small San Diego company called ViaCyte.

Its a significant step, partly because the ViaCyte study is only the third in the United States of any treatment based on embryonic stem cells. These cells, once removed from early-stage human embryos, can be grown in a lab dish and retain the ability to differentiate into any of the cells and tissue types in the body. One other study, since cancelled, treated several patients with spinal-cord injury (see Geron Shuts Down Pioneering Stem-Cell Program and Stem-Cell Gamble), while tests to transplant lab-grown retina cells into the eyes of people going blind are ongoing (see Stem Cells Seem Safe in Treating Eye Disease).

Type 1 patients must constantly monitor their blood glucose using finger pricks, carefully time when and what they eat, and routinely inject themselves with insulin that the pancreas should make. Insulin, a hormone, triggers the removal of excess glucose from the blood for storage in fat and muscles. In type 1 diabetics, the pancreas doesnt make it because their own immune system has attacked and destroyed the pancreatic islets, the tiny clusters of cells containing the insulin-secreting beta cells.

The routine is especially hard on children, but if they dont manage their glucose properly, they could suffer nerve and kidney damage, blindness, and a shortened life span. Yet despite years of research, there is still just nothing to offer patients, says Robert Henry, a doctor at the University of California, San Diego, whose center is carrying out the surgeries for ViaCyte.

Henry slightly overstates the case, but not by much. There is something called the Edmonton Protocol, a surgical technique first described in the New England Journal of Medicine in 2000. It used islets collected from cadavers; by transplanting them, doctors at the University of Alberta managed to keep all seven of their first patients off insulin for an entire year.

Early hopes for the Edmonton Protocol were quickly tempered, however. Only about half of patients treated have stayed off insulin long-term, and the procedure, which is still regarded as experimental in the U.S., isnt paid for by insurance. It requires recipients to take powerful immune-suppressing drugs for life. Suitable donor pancreases are in extremely short supply.

The early success of the Edmonton Protocol came only two years after the discovery of embryonic stem cells, in 1998. Those pressing for a diabetes cure quickly set a new goal: pair something like the Edmonton Protocol with the technology of lab-grown beta cells, the supplies of which are theoretically infinite.

This biocompatible capsule is designed to protect manufactured pancreas cells.

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A Pancreas in a Capsule

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By Beth Miller

Newswise Restoring function after spinal cord injury, which damages the connections that carry messages from the brain to the body and back, depends on forming new connections between the surviving nerve cells. While there are some delicate surgical techniques that reconnect the nerves, researchers are also looking at ways to restore the connections themselves at a cellular level.

With a five-year, nearly $1.7 million grant from the National Institutes of Health, Shelly Sakiyama-Elbert, PhD, professor of biomedical engineering in the School of Engineering & Applied Science at Washington University in St. Louis, is using novel methods to take a closer look at how these nerve cells grow and make new connections to reroute signals between the brain and the body that could restore function and movement in people with these debilitating injuries.

Sakiyama-Elbert, also associate chair of the Department of Biomedical Engineering, is widely known for her groundbreaking work in tissue engineering techniques. Her research expertly blends biology, chemistry and biomedical engineering to focus on developing biomaterials for drug delivery and cell transplantation to treat peripheral nerve and spinal cord injury.

In the new research, funded by the National Institute of Neurological Disorders and Stroke, she and her lab members want to understand how these nerve cells, or neurons, form connections and rewire after a spinal cord injury, looking closely at which particular cells, or interneurons, are forming these new connections.

There have been a lot of studies where researchers have shown recovery in partial spinal cord injury models, but no one understands at a cellular level which cells are responsible for rewiring or forming the new connections, Sakiyama-Elbert said. If we want to make regeneration more efficient and potentially translatable to humans where it is more challenging, we need to understand whats actually going on at a cellular level.

Once we determine which cells are making connections, we can determine how to transplant more of those cells or try to stimulate tissue-specific stem cells to make those types of neurons and form these types of connections, Sakiyama-Elbert said.

While much is known about motor neurons, less is known about these interneurons in culture or how to direct their connection with other neurons. Sakiyama-Elbert is developing new tools that will allow her to isolate very pure groups of different types of interneurons and then study what encourages them to grow and form new connections.

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NIH Grant Will Help Understanding How Connections Rewire After Spinal Cord Injury

On the weekend, a whos who of hockey legends gathered to pay tribute to Gordie Howe in his hometown of Saskatoon.

In addition to sharing memories about Mr. Hockey, a constant theme of the festivities was his miracle recovery from stroke.

Mr. Howe, 86, suffered two strokes last year and, according to his family, was near death before he travelled to Clinica Santa Clarita in Tijuana, Mexico, in December for experimental stem-cell treatment.

Afterward, Mr. Howe was able to walk again. He regained a lot of weight and he began to resemble his old self. (Most of this is second-hand; Mr. Howe also suffers from dementia and has not or cannot speak of his symptoms or treatment first-hand.)

After his stem-cell treatment, the doctor told us it was kind of an awakening of the body, his son, Marty Howe, told The Canadian Press. They call it the miracle of stem cells and it was nothing less than a miracle.

Mr. Howes Lazarus-like recovery makes for a great tug-at-the-heartstrings narrative for a man whose career has been the embodiment of perseverance and longevity. But if you step back a moment and examine the science, all sorts of alarm bells should go off.

Stem cells, which were discovered in the early 1960s, have the remarkable potential to develop into many different cells, at least in the embryonic stage. They also serve as the bodys internal repair system.

The notion that spinal cords and limbs and heart muscle and brain cells could be regenerated holds a magical appeal.

But, so far, stem-cell therapies have been used effectively to treat only a small number of blood disorders, such as leukemia. (Canada has a public bank that collects stem cells from umbilical-cord blood and a program to match stem-cell donors with needy patients.)

Stem cells also show promise in the treatment of conditions such as spinal-cord injuries, Parkinsons and multiple sclerosis, but those hopes have not yet moved from the realm of science-fiction into clinical medicine.

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The stem-cell miracle is anecdotal

Exciting research . . . Spinal Cord Society research director Dr Jim Faed demonstrates a Terumo sterile tube welder, in use in Dunedin Hospital. PHOTO: BRENDA HARWOOD

A Dunedin-based research team, working on a cure for type 1 diabetes, is reaching out for support.

The Spinal Cord Society of New Zealand research team, based at the University of Otago's Centre for Innovation, has been developing methods for using patients' stem cells to ''turn off'' the auto-immune response that causes type 1 diabetes.

Research director Dr Jim Faed said the work built on the research of a

Chinese-American group, which was able to show a way to cure type 1 diabetes using a patient's own stem cells to reset the body's immune system, helping the return of insulin production.

''That work now needs repeating and improving, to speed up the recovery process,'' Dr Faed said.

Type 1 diabetes destroys the body's insulin-producing cells as an auto-immune response to a trigger, such as an infection, in people with an inherited tendency. These people, who number about 25,000 in New Zealand, ''need some help to flick the switch and turn that auto-immune response off'', he said.

''We feel we have the right strategy for that. What we need now is to buy the equipment to progress from just lab-scale work to producing cells that are safe to use in people [in clinical trials].''

The research was ''on the verge of a real breakthrough'' and could be one of the most exciting scientific advances since antibiotics, he said.

If a cure for type 1 diabetes could be established, it could open the way for researchers to look into other auto-immune diseases, such as rheumatoid arthritis, he said.

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Help us `break through': scientists

Feb. 2, 2015

Su-Chun Zhang (center), a UW professor of neuroscience, talks with staff in his lab as they prepare stem cell cultures in March 2013.Zhangs new research may lay the foundation for treating neurodegenerative diseases like ALS.

Photo: Jeff Miller

This story starts in 1955, upon the death of Albert Einstein, when the pathologist charged with performing the famous scientists autopsy stole his brain.

Fast forward to the 1980s when a University of California, Berkeley scientist was studying parts of the stolen goods involved in complex thinking and discovered that the father of relativity had more of certain types of cells, called astrocytes, than other human brains studied.

Today, another 30 years later, scientists still dont have a solid grasp on everything these cells do in the human nervous system, largely because theyre difficult to study. But Su-Chun Zhang, a professor of neuroscience and neurology at the University of Wisconsin-Madison Waisman Center, and his research team have published a unique model for learning more about the role of human astrocytes in the Journal of Clinical Investigation today.

Su-Chun Zhang

The findings may lay a foundation for the treatment of a number of neurodegenerative diseases, including ALS (amyotrophic lateral sclerosis) and debilitating spinal cord injuries.

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Laying a foundation for treating ALS, spinal cord injury

Peek, a 20-year-old American Black Bear living at the Topeka Zoo, is undergoing treatment, including a CT scan, for a back condition, zoo director Brendan Wiley said Monday.

Peek came out of her den two weeks ago and her keeper noticed the bears hind legs were wobbly. Peek was given pain medication. However, the condition quickly worsened, Wiley said in a news release.

Within the next seven days, Peek continued to lose control of her rear legs.

One of the unique things about this scenario is that Peek hasnt acted like anything is hurting her, said Shanna Simpson, animal care supervisor. It is like the front half of her body can no longer communicate with the back half.

Peeks illness required the use of a CT scan, which used equipment the zoo doesnt have, Wiley said.

Zoo veterinarian Shirley Llizo brought in Larry Snyder and Travis Gratton, of University Bird and Small Animal Hospital, to help harvest fat cells from Peek. This would allow the fat cells to be converted to stem cells to be injected.

Peek was tranquilized Jan. 22 and transported to the zoos hospital so the fat cells could be harvested. After the harvest, Peek was transported to St. Francis Health Center, where she was met by Brent Wilkins, director of imaging services, and his staff.

After confirming Peek would fit in the 72-centimeter CT scanner, Wilkins was able to do the scan, and radiologist James Owen found an area of Peeks spine was experiencing spinal stenosis. This is a narrowing of the spinal column that causes pressure on the spinal cord, according to zoo officials.

Our first priority is human patient safety and access, Wilkins said. We work with the Topeka Zoo to accommodate animals that need CT scans in off hours when one of our CT scanners is available. We made sure the bear was separated from any other patients and performed a high-level decontamination and cleaning of the area, called a terminal cleaning, after the bears visit. Were very happy to help our friends at the zoo in keeping the animals healthy.

After the CT scan, Peek went back to the zoo, where she received the stem cell therapy. Stem cell therapy treatment in bears is new technology, Wiley said.

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Topeka Zoo's bear gets CT scan, stem cell therapy

Peek the American black bear (Courtesy: Topeka Zoo)

Peek the bear is shown undergoing a CT scan at St. Francis Health Center in this photo from the Topeka Zoo.

St. Francis Health Center staff watch as a CT scan is performed on Peek the black bear. (Courtesy: Topeka Zoo)

TOPEKA, Kan. (WIBW) - An American black bear at the Topeka Zoo may be the first bear ever to undergo stem cell treatment for a spinal problem.

Zoo Director Brendan Wiley says Peek, who is 20-years old, started losing control of her hind legs two weeks ago. The condition worsened and zoo staff says pain medication was not having any impact on the situation.

One of the unique things about this scenario is that Peek hasnt acted like anything is hurting her. It is like the front half of her body can no longer communicate with the back half, said Animal Care Supervisor Shanna Simpson.

The zoo worked with St. Francis Health Center to perform a CT scan. Peek was tranquilized and transported to St. Francis' imaging facilities.

"Our first priority is human patient safety and access," says Brent Wilkins, director of Imaging Services at St. Francis Health. "We work with the Topeka Zoo to accommodate animals that need CT scans in off hours when one of our CT scanners is available. We made sure the bear was separated from any other patients and performed a high-level decontamination and cleaning of the area, called a terminal cleaning, after the bears visit."

The scan revealed an area of Peek's spine with spinal stenosis, a narrowing of her spinal column, causing pressure on the spinal cord.

In anticipation of future treatment, Dr. Larry Snyder and Dr. Travis Gratton, veterinarians from Topeka's University Small Animal Hospital were contacted. Before Peek was transported for the CT scan, the two harvested fat cells, which they converted to stem cells to inject back into her. The theory behind the treatment is that the stem cells can stimulate damaged area to repair and heal itself.

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Topeka Zoo Black Bear Undergoes Stem Cell Therapy

TORONTO - Gordie Howe's son says the hockey legend's stroke symptoms have improved since his treatment with stem cells at a Mexican clinic in early December and he wants him to repeat the procedure.

But regenerative medicine experts say there's no scientific evidence such therapies work, and in some cases they can be seriously harmful or even deadly.

The 86-year-old Howe suffered two disabling strokes late last year. In December, the family took him to a Tijuana clinic where he received stem cell injections as part of a clinical trial being run under a licensing agreement with Stemedica Cell Technologies of San Diego, Calif.

The experimental treatment involved injecting neural stem cells into Howe's spinal canal, along with intravenous infusions of mesenchymal stem cells, which are found in bone marrow, fat and umbilical cord blood.

Marty Howe said his father can walk again, his speech is improving and he is regaining some of the weight he lost following the strokes.

"After his stem cell treatment, the doctor told us it was kind of an awakening of the body, and it was all that," he told The Canadian Press while in Calgary for a hockey promotion event Tuesday. "They call it the miracle of stem cells and it was nothing less than a miracle."

However, experts in the field question whether stem cells are responsible for Howe's improvement and caution that most so-called stem cell therapies have not gone through rigorous scientific trials, nor have they been approved as treatments by Health Canada or the U.S. Food and Drug Administration.

Mick Bhatia, director of McMaster University's Stem Cell and Cancer Research Institute, said there are many unknowns in Howe's case, such as how many stem cells were administered, were tests done to see whether they migrated to the targeted area of the body, and did they take up residence where they might have some effect or simply disappear?

"Is this a transient effect, or is it really a perceived or somewhat of a placebo effect and is there something really happening? Scientifically and biologically that is important," Bhatia said Wednesday from Hamilton.

And because Howe received adult stem cells produced from donor cells, he may have needed to take drugs to prevent an immune reaction as well as anti-inflammatory medications, he said.

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Newswise UCLA has received grant funding from the Center for the Advancement of Science in Space (CASIS) to lead a research mission that will send rodents to the International Space Station (ISS). The mission will allow astronauts on the space station and scientists on Earth to test a potential new therapy for accelerating bone growth in humans.

The research will be led by Dr. Chia Soo, a UCLA professor of plastic and reconstructive surgery and orthopaedic surgery, who is member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. Soo is also research director for UCLA Operation Mend, which provides medical care for wounded warriors. The study will test the ability of a bone-forming molecule called NELL-1 to direct stem cells to induce bone formation and prevent bone degeneration.

Other members of the UCLA research team are Dr. Kang Ting, a professor in dentistry who discovered NELL-1 and is leading efforts to translate NELL-1 therapy to humans, Dr. Ben Wu, a professor of bioengineering who modified the NELL-1 molecule to make useful for treating osteoporosis, and Dr. Jin Hee Kwak, an assistant professor of dentistry who will manage daily operations.

Based on results of previous studies supported by the NIH, the UCLA-ISS team will begin ground operations in early 2015. They hope that the study will provide new insights into the prevention of bone loss or osteoporosis as well as the regeneration of massive bone defects that can occur in wounded military personnel. Osteoporosis is a significant public health problem commonly associated with skeletal disuse conditions such as immobilization, stroke, cerebral palsy, muscular dystrophy, spinal cord injury and jaw resorption after tooth loss.

NELL-1 holds tremendous hope, not only for preventing bone loss but one day even restoring healthy bone, Ting said. For patients who are bed-bound and suffering from bone loss, it could be life-changing.

The UCLA team will oversee the ground operations of the mission in tandem with a flight operation coordinated by CASIS and NASA.

A group of 40 rodents will be sent to the International Space Station U.S. National Laboratory onboard the SpaceX Dragon capsule, where they will live for two months in a microgravity environment during the first ever test of NELL-1 in space, said Dr. Julie Robinson, NASAs chief scientist for the International Space Station program at the Johnson Space Center.

CASIS is proud to work alongside UCLA in an effort to promote the station as a viable platform for bone loss inquiry, said Warren Bates, director of portfolio management for CASIS. Through investigations like this, we hope to make profound discoveries and enable the development of therapies to counteract bone loss ailments common in humans.

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Growing Bone in Space: UCLA and CASIS Announce Pioneering Collaborative Study to Test Therapy for Bone Loss on the ...

UCLA has received grant funding from the Center for the Advancement of Science in Space (CASIS) to lead a research mission that will send rodents to the International Space Station (ISS). The mission will allow astronauts on the space station and scientists on Earth to test a potential new therapy for accelerating bone growth in humans.

The research will be led by Dr. Chia Soo, a UCLA professor of plastic and reconstructive surgery and orthopaedic surgery, who is member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. Soo is also research director for UCLA Operation Mend, which provides medical care for wounded warriors. The study will test the ability of a bone-forming molecule called NELL-1 to direct stem cells to induce bone formation and prevent bone degeneration.

Other members of the UCLA research team are Dr. Kang Ting, a professor in dentistry who discovered NELL-1 and is leading efforts to translate NELL-1 therapy to humans, Dr. Ben Wu, a professor of bioengineering who modified the NELL-1 molecule to make useful for treating osteoporosis, and Dr. Jin Hee Kwak, an assistant professor of dentistry who will manage daily operations.

Based on results of previous studies supported by the NIH, the UCLA-ISS team will begin ground operations in early 2015. They hope that the study will provide new insights into the prevention of bone loss or osteoporosis as well as the regeneration of massive bone defects that can occur in wounded military personnel. Osteoporosis is a significant public health problem commonly associated with "skeletal disuse" conditions such as immobilization, stroke, cerebral palsy, muscular dystrophy, spinal cord injury and jaw resorption after tooth loss.

"NELL-1 holds tremendous hope, not only for preventing bone loss but one day even restoring healthy bone," Ting said. "For patients who are bed-bound and suffering from bone loss, it could be life-changing."

The UCLA team will oversee the ground operations of the mission in tandem with a flight operation coordinated by CASIS and NASA.

"A group of 40 rodents will be sent to the International Space Station U.S. National Laboratory onboard the SpaceX Dragon capsule, where they will live for two months in a microgravity environment during the first ever test of NELL-1 in space," said Dr. Julie Robinson, NASA's chief scientist for the International Space Station program at the Johnson Space Center.

"CASIS is proud to work alongside UCLA in an effort to promote the station as a viable platform for bone loss inquiry," said Warren Bates, director of portfolio management for CASIS. "Through investigations like this, we hope to make profound discoveries and enable the development of therapies to counteract bone loss ailments common in humans."

Prolonged space flights induce extreme changes in bone and organ systems that cannot be replicated on Earth.

"Besides testing the limits of NELL-1's robust bone-producing effects, this mission will provide new insights about bone biology and could uncover important clues for curing diseases such as osteoporosis," Wu said.

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Growing bone in space: Study to test therapy for bone loss on the International Space Station