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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

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

Illustration: John Spooner

One of the joys for those who work in the health services area is bringing relief to patients from chronic conditions.

And as the level of desperation rises, some patients will pay over the odds for treatment, pursing unproven options in the hope of some improvement in their condition. And where there is unmet demand, supply soon steps in to fill the gap.

Last year, there was intense global media attention on stem cell treatments following a paralysed patient in Poland who walked after a cell transplant, a project involving Polish and UK researchers.

Stem cells may well offer significant potential promise for patients in a range of treatments. But to date, much of that optimism has run well ahead of the reality.

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Clinical trials to ensure the efficacy and safety of medical treatments is slow and laborious, taking several years, at the very least, to verify the merits of a treatment before then seeking approvals to offer the treatment to patients.

But for those searching for a stem cell treatment in Australia, there is a loophole: a referral from your doctor is often all it takes to get access, even though there is scant proof that the patient benefits.

Clearly, some patients so badly want to believe the treatment is good for them that this will override the necessary caution.

Much of this activity is taking place in private clinics, although sharemarket investors, too, have stem cell groups they can invest in.

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Optimism on stem cells, ahead of reality

LAWTON, Okla._A stem cell surgery procedure, not yet approved by the FDA, could give a local paralyzed veteran the use of his arms again.

Two years ago, retired Senior Airman Ted "TJ" Williams was left as a quadriplegic when his Humvee rolled over in a freak accident while on duty in Montana. He spent several weeks in a coma.

Now, he and his wife have found a surgery that may improve his physical abilities. They're dipping into their funds to pay for the procedure, since it's not covered by insurance, but they've set up a GoFundMe account to raise $7,500 to cover travel expenses out of the country to get the treatment.

Williams is able to move his left wrist and arm more, and has even gained more core control, thanks to therapy. But, he still needs his wife's help for simple tasks like getting dressed and using the restroom.

Williams sits next to his wife in his wheelchair and watches TV. Years ago, he would've been running outside, but one accident changed everything.

"I just remember leaving base and then waking up 2 or 3 weeks later, wondering where am I. I couldn't move anything. It was just shocking seeing my family around my bed. I was just like, Wow. What's going on,'" recalled Williams.

On November 29, 2012, Williams was on duty with his security forces team. He was in the back seat when his Humvee suddenly swerved to miss a herd of deer, rolling several times. He was ejected from the vehicle and was later found 60 feet away.

Williams was rushed to the hospital. When he woke up from the coma, doctors told him he had broken the vertebrae in his neck and lost function from the chest down.

"I was really upset and scared. Me and my wife are young. We haven't had children yet or anything. It scared me not knowing what the future was to hold," said Williams.

He was sent to a VA hospital in San Antonio for in-patient rehab. Once he was finished, he met a physical trainer in who specializes in exercises for those who are suffering from spinal cord and other neurological injuries, which was just what he needed.

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Quadriplegic veteran to receive stem cell treatments

Stem Cell Therapy for Spinal Cord Injuries

SCI or Spinal cord injuries usually occur with a sudden & traumatic injury or blow to the spinal cord that dislocates or fractures the vertebrae. The damage of SCI begins at the point of impact when the displaced disc material,bone fragments, or ligaments either bruise or tear the spinal cord tissue. Most SCI injuries do not sever the spinal cord completely. The SCI is likely to cause minor compressions or fractures of the vertebrae, that crush and destroy the signal carriers called axons. Axons carry electric signals up and down our spinal cords and act as a messenger between our brains and the rest of our bodies. SCI generally cause damage to some,many, or in some cases all the axons.

Some SCI victims can accomplish a complete recovery. Others however,will be left with complete paralysis.SCI are classified in two categories, complete or incomplete. A complete SCI is indicated by the total lack of all sensory and motor functions below the area of injury. An incomplete SCI means that the patient has the ability to convey some messages to &/or from the brain but often in a limited capacity. Most People with incomplete SCI injuries can retain minor sensory and/or motor function below the point of injury. Those who survive SCI will most likely suffer for medical complications like bowel and bladder dysfunction and often have chronic pain. Partial SCI patients also have an increased susceptibility to heart & respiratory problems. Successful recovery usingstem cells to treatspinal cord Injuries depends largely on how well systematic failures and chronic conditions are handled day-to-day.

Cell Therapy for injured Spinal Cordsfocuses on regeneration of the connections between your brain and body that have been broken or severely hampered. Stem cellscan help regain motor functions and regain bowel and bladder dysfunction, regain loss of sensations, help minimize chronic pain,cramps and its associated depression. Conventional treatments for SCI today are focused mainly on providing rehabilitation and the prevention of secondary damage. Recent advancements in spinal cord cell treatments offer hope for thousands of victims around the world who are often left with little or no hope for recovery. Stemcell treatments for spinal cord injury can help support and promote the bodies natural regeneration cycle by stimulating the rapid repair of damaged cells and tissue. The regenerative treatmentgoes well beyond the traditional approach of symptomatic treatments and can help you improve and regain some of the previously lost or impaired physical functions. Cell death normally occurs when our cells are injured. These dead cells are surrounded by both damaged and healthy cells. Stem cells stimulate the healing of these injured cells via the secretion of cytokines and other cells such as NGF or nerve growth factors to trigger the body into self-healing mode.

Thai Medical offers a uniquespinal cordtreatment protocol usinga multi-pronged approach. First, adult stem cells are injected directly into the damaged areas of the spine via the accuracy and precision guidance of a CT-guided intra-spinal injection. The treatment is then supplemented even further with another injection using an LP or lumbar puncture and/or IV drip injections.

CT-guided intra-spinal stem cell treatments are considered the gold standard in the field of Stem Cell treatment for spinal cord injury and spinal injury stem cell research for Stem Cell Treatments in Thailand. The precision of CT guidance allows the neurosurgeon to precisely aim the stem cells inside the healthy spinal cord tissues directly adjacent to the injury and lesions. The CT-guided intra-spinal stem cell treatments avoids the requirements of open spine surgeries of yesteryear. CT-guided intra-spinal stem cell treatments also avoid the risks, pain and subsequent healing time associated with cell treatment for SCI (spinal cord injury.)

The objectives of the enrichedcell treatments for spinal cord injuries is to help repair the injured areas on the cellular level near the point of impacts and lesions. The resulting therapy will generally lead to improved quality of life and improved symptoms primarily in physical function,movements and abilities. The majority of patients who are accepted into the program have show dramatic improvements usually after the first or second treatments and continue to improve and regenerate 6 months to a year after treatment. The results are permanent barring new injuries.

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Stem Cell Treatment for Spinal Cord Injuries SCI Therapy

Washington, DC - infoZine - Three-year outcomes from an ongoing clinical trial suggest that high-dose immunosuppressive therapy followed by transplantation of a person's own blood-forming stem cells may induce sustained remission in some people with relapsing-remitting multiple sclerosis (RRMS). RRMS is the most common form of MS, a progressive autoimmune disease in which the immune system attacks the brain and spinal cord.

Three years after the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant or HDIT/HCT, nearly 80 percent of trial participants had survived without experiencing an increase in disability, a relapse of MS symptoms or new brain lesions. Investigators observed few serious early complications or unexpected side effects, although many participants experienced expected side effects of high-dose immunosuppression, including infections and gastrointestinal problems.

Scientists estimate that MS affects more than 2.3 million people worldwide. Symptoms can vary widely and may include disturbances in speech, vision and movement. Most people with MS are diagnosed with RRMS, which is characterized by periods of relapse or flare up of symptoms followed by periods of recovery or remission. Over years, the disease can worsen and shift to a more progressive form.

In the study, researchers tested the effectiveness of HDIT/HCT in 25 volunteers with RRMS who had relapsed and experienced worsened neurological disability while taking standard medications. Doctors collected blood-forming stem cells from participants and then gave them high-dose chemotherapy to destroy their immune systems. The doctors returned the stem cells to the participants to rebuild and reset their immune systems.

"Notably, participants did not receive any MS drugs after transplant, yet most remained in remission after three years," said Daniel Rotrosen, M.D., director of NIAID's Division of Allergy, Immunology and Transplantation. "In contrast, other studies have shown that the best alternative MS treatments induce much shorter remissions and require long-term use of immunosuppressive drugs that can cause serious side effects."

The study researchers plan to follow participants for a total of five years, recording all side effects associated with the treatment. Final results from this and similar studies promise to help inform the design of larger trials to further evaluate HDIT/HCT in people with MS.

The trial is funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN).

The three-year findings are published in the Dec. 29, 2014, online issue of JAMA Neurology.

Related Link Immune Tolerance Network (ITN)

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Stopping Multiple Sclerosis with Stem Cell Transplants

NIH-funded study yields encouraging early results

Three-year outcomes from an ongoing clinical trial suggest that high-dose immunosuppressive therapy followed by transplantation of a person's own blood-forming stem cells may induce sustained remission in some people with relapsing-remitting multiple sclerosis (RRMS). RRMS is the most common form of MS, a progressive autoimmune disease in which the immune system attacks the brain and spinal cord. The trial is funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN) .

Three years after the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant or HDIT/HCT, nearly 80 percent of trial participants had survived without experiencing an increase in disability, a relapse of MS symptoms or new brain lesions. Investigators observed few serious early complications or unexpected side effects, although many participants experienced expected side effects of high-dose immunosuppression, including infections and gastrointestinal problems. The three-year findings are published in the Dec. 29, 2014, online issue of JAMA Neurology.

These promising results support the need for future studies to further evaluate the benefits and risks of HDIT/HCT and directly compare this treatment strategy to current MS therapies, said NIAID Director Anthony S. Fauci, M.D. If the findings from this study are confirmed, HDIT/HCT may become a potential therapeutic option for people with this often-debilitating disease, particularly those who have not been helped by standard treatments.

Scientists estimate that MS affects more than 2.3 million people worldwide. Symptoms can vary widely and may include disturbances in speech, vision and movement. Most people with MS are diagnosed with RRMS, which is characterized by periods of relapse or flare up of symptoms followed by periods of recovery or remission. Over years, the disease can worsen and shift to a more progressive form.

In the study, researchers tested the effectiveness of HDIT/HCT in 25 volunteers with RRMS who had relapsed and experienced worsened neurological disability while taking standard medications. Doctors collected blood-forming stem cells from participants and then gave them high-dose chemotherapy to destroy their immune systems. The doctors returned the stem cells to the participants to rebuild and reset their immune systems.

Notably, participants did not receive any MS drugs after transplant, yet most remained in remission after three years, said Daniel Rotrosen, M.D., director of NIAIDs Division of Allergy, Immunology and Transplantation. In contrast, other studies have shown that the best alternative MS treatments induce much shorter remissions and require long-term use of immunosuppressive drugs that can cause serious side effects.

The study researchers plan to follow participants for a total of five years, recording all side effects associated with the treatment. Final results from this and similar studies promise to help inform the design of larger trials to further evaluate HDIT/HCT in people with MS.

The work was sponsored by NIAID, NIH, and conducted by the ITN (contract number N01 AI015416) and NIAID-funded statistical and clinical coordinating centers (contract numbers HHSN272200800029C and HHSN272200900057C). The ClinicalTrials.gov identifier for the study High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT-MS) is NCT00288626.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID Web site at http://www.niaid.nih.gov.

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News & Events

Spinal cord injuries are devastating, leaving the person injured facing a life time of challenges, and placing a huge strain on their family and loved ones who help care for them.

The numbers affected are not small. More than a quarter of a million Americans are living with spinal cord injuries and there are more than 11,000 new cases each year.

Its not just a devastating injury, its also an expensive one. According to the National Spinal Cord Injury Statistical Center it can cost more than $775,000 to care for a patient in the first year after injury, and the estimated lifetime costs due to spinal cord injury can be as high as $3 million.

Right now there is no cure, and treatment options are very limited. We have heard for several years now about stem cell research aimed at helping people with spinal cord injuries, but where is that research and how close are we to testing the most promising approaches in people?

Thats going to be the focus of a Google Hangout on Spinal Cord Injury and Stem Cell Research that we are hosting tomorrow, Tuesday, November 18 from noon till 1pm PST.

Well be looking at the latest stem cell-based treatments for spinal cord injury including work being done by Asterias Biotherapeutics, which was recently given approval by the Food and Drug Administration (FDA) to start a clinical trial for spinal cord injury. We are giving Asterias $14.3 million to carry out that trial and you can read more about that work here.

Were fortunate in having three great guests for the Hangout: Jane Lebkowski, Ph.D., the President of research and development at Asterias; Roman Reed, a patient advocate and tireless champion of stem cell research and the founder of the Roman Reed Foundation; and Kevin Whittlesey, Ph.D., a CIRM science officer, who will discuss other CIRM-funded research that aims to better understand spinal cord injury and to bring stem cell-based therapies to clinic trials.

You can find out how to join the Hangout by clicking on the event page link: http://bit.ly/1sh1Dsm

The event is free and interactive, so youll be able to ask questions of our experts. You dont need a Google+ account to watch the Hangout just visit the event page at the specified time. If you do have a G+ account, please RSVP at the event page (link shown above). Also, with the G+ account you can ask questions in the comment box on this event page. Otherwise, you can tweet questions using #AskCIRMSCI or email us at info@cirm.ca.gov.

We look forward to seeing you there!

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Spinal cord injury and stem cell research; find out the ...

Public Abstract:

1.3 million Americans suffer chronically from spinal cord injuries (SCI); each year ~15,000 individuals sustain a new injury. For California, this means nearly 147,000 individuals are living with a SCI which can leave otherwise healthy individuals with severe deficits in movement, sensation, and autonomic function. Recovery after SCI is often limited, even after aggressive emergency treatment with steroids and surgery, followed by rehabilitation. The need to develop new treatments for SCI is pressing. We believe that stem cell therapies could provide significant functional recovery, improve quality of life, and reduce the cost of care for SCI patients. The goal of this Disease Team is to evaluate a novel cell therapy approach to SCI involving transplantation of human neural stem cells. In 2005, the FDA authorized the worlds first clinical testing of human neural stem cell transplantation into the CNS. Since then, our research team has successfully generated clinical grade human neural stem cells for use in three clinical trials, established a favorable safety profile that now approaches five years in some subjects and includes evidence of long-term donor-cell survival. Relevant to this Disease Team, the most recent study began testing human neural stem cells in thoracic spinal cord injury. The initial group of three patients with complete injury has been successfully transplanted. The Disease Team seeks to extend the research into cervical SCI. Neural cell transplantation holds tremendous promise for achieving spinal cord repair. In preliminary experiments, the investigators on this Disease Team showed that transplantation of both murine and human neural stem cells into animal models of SCI restore motor function. The human neural stem cells migrate extensively within the spinal cord from the injection site, promoting new myelin and synapse formation that lead to axonal repair and synaptic integrity. Given these promising proof-of-concept studies, we propose to manufacture clinical-grade human neural stem cells and execute the preclinical studies required to submit an IND application to the FDA that will support the first-in-human neural stem cell transplantation trial for cervical SCI. Our unmatched history of three successful regulatory submissions, extensive experience in manufacturing, preclinical and clinical studies of human neural stem cells for neurologic disorders, combined with an outstanding team of basic and clinical investigators with expertise in SCI, stem cell biology, and familiarity with all the steps of clinical translation, make us an extremely competitive applicant for CIRMs Disease Team awards. This award could ultimately lead to a successful FDA submission that will permit human testing of a new treatment approach for SCI; one that could potentially reverse paralysis and improve the patients quality of life.

Statement of Benefit to California:

Spinal cord injuries affect more than 147,000 Californians; the majority are injuries to the cervical level (neck region) of the spinal cord. SCI exacts a devastating toll not only on patients and families, but also results in a heavy economic impact on the state: the lifetime medical costs for an individual with a SCI can exceed $3.3 million, not including the loss of wages and productivity. In California this translates to roughly $86 billion in healthcare costs. Currently there are no approved therapies for chronic thoracic or cervical SCI. We hope to advance our innovative cell therapy approach to treat patients who suffer cervical SCI. For the past 9 years, the assembled team (encompassing academic experts in pre-clinical SCI models, complications due to SCI, rehabilitation and industry experts in manufacturing and delivery of purified neural stem cells), has developed the appropriate SCI models and assays to elucidate the therapeutic potential of human neural stem cells for SCI repair. Human neural stem cell transplantation holds the promise of creating a new treatment paradigm. These cells restored motor function in spinal cord injured animal models. Our therapeutic approach is based on the hypothesis that transplanted human neural stem cells mature into oligodendrocytes to remyelinate demyelinated axons, and/or form neurons to repair local spinal circuitry. Any therapy that can partially reverse some of the sequelae of SCI could substantially change the quality-of-life for patients by altering their dependence on assisted living, medical care and possibly restoring productive employment. Through CIRM, California has emerged as a worldwide leader in stem cell research and development. If successful, this project would further CIRMs mission and increase Californias prominence while providing SCI therapy to injured Californians. This Team already has an established track record in stem cell clinical translation. The success of this Disease Team application would also facilitate new job creation in highly specialized areas including cell manufacturing making California a unique training ground. In summary, the potential benefit to the state of California brought by a cervical spinal cord Disease Team project would be myriad. First, a novel therapy could improve the quality of life for SCI patients, restore some function, or reverse paralysis, providing an unmet medical need to SCI patients and reducing the high cost of health care. Moreover, this Disease Team would maintain Californias prominence in the stem cell field and in clinical translation of stem cell therapies, and finally, would create new jobs in stem cell technology and manufacturing areas to complement the states prominence in the biotech field.

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The BICEP2 telescope at the South Pole may have spied gravitational waves or dust.

This year may be best remembered for how quickly scientific triumph morphed into disappointment, and even tragedy: breakthroughs in stem-cell research and cosmology were quickly discredited; commercial spaceflight faced major setbacks. Yet landing a probe on a comet, tracing humanitys origins and a concerted push to understand the brain provided reasons to celebrate.

Asian nations soared into space this year. The Indian Space Research Organisation put a mission into orbit around Mars the first agency to do so on its first try. Japan launched the Hayabusa-2 probe, its second robotic voyage to bring back samples from an asteroid. And even as Chinas lunar rover Yutu (or Jade Rabbit) stopped gathering data on the Moons surface, mission controllers took the next step in the countrys lunar exploration programme by sending a test probe around the Moon and back to Earth.

But for commercial spaceflight, it was a bad year. Virgin Galactics proposed tourism vehicle SpaceShipTwo disintegrated during a test flight in California and killed one of its pilots. That came just three days after a launch-pad explosion in Virginia destroyed an uncrewed private rocket intended to take supplies to the International Space Station. The accident wiped out a number of research experiments destined for the station, whose managers are trying to step up its scientific output. Problems on the station also delayed the deployment of a flock of tiny Earth-watching satellites, nicknamed Doves, which are part of the general trend of using miniature CubeSats to collect space data.

On a bigger scale, the European Space Agency successfully launched the first in its long-awaited series of Sentinel Earth-observing satellites.

After a decade-long trip, the European Space Agencys Rosetta spacecraft arrived at comet 67P/ChuryumovGerasimenko in August and settled into orbit. Three months later, Rosetta dropped the Philae probe to 67Ps surface, in the first-ever landing on a comet. Philae relayed science data for 64hours before losing power in its shadowy, rocky landing site.

Meanwhile, a flotilla of Mars spacecraft probes from India, the United States and Europe had an unplanned close brush with comet Siding Spring, which zipped past the red planet in October at a distance of 139,500kilometres about one-third of the distance from Earth to the Moon. NASA rovers continued to trundle along on the Martian surface: Curiosity finally reached the mountain that it has been heading towards since landing in 2012, and Opportunity passed 40kilometres on its odometer, breaking a Soviet lunar rovers distance record for off-Earth driving.

The search for planets beyond the Solar System also got a huge boost. In February, the team behind the now mostly defunct Kepler spacecraft announced that it had confirmed the existence of 715extrasolar planets, the largest-ever single haul. Kepler data also revealed the first known Earth-sized exoplanet in the habitable zone of its star, a step closer to the long-sought Earth twin.

Considering that they have been dead for around 30,000 years, Neanderthals had a hell of a year. Their DNA survives in non-African human genomes, thanks to ancient interbreeding, and two teams this year catalogued humans Neanderthal heritage. Scientists learnt more about the sexual encounters between Homo neanderthalensis and early humans after analysing the two oldest Homo sapiens genomes on record from men who lived in southwest Siberia 45,000years ago and in western Russia more than 36,000years ago, respectively. The DNA revealed hitherto-unknown human groups and more precise dates for when H.sapiens coupled with Neanderthals, which probably occurred in the Middle East between 50,000 and 60,000 years ago. Radiocarbon dating of dozens of archaeological sites in Europe, meanwhile, showed that humans and Neanderthals coexisted there for much longer than was once thought up to several thousand years in some places.

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365 days: 2014 in science

Stem cell therapy is a potential treatment for spinal cord injury and different stem cell types have been grafted into animal models and humans suffering from spinal trauma. Due to inconsistent results, it is still an important and clinically relevant question which stem cell type will prove to be therapeutically effective. Thus far, stem cells of human sources grafted into spinal cord mostly included barely defined heterogeneous mesenchymal stem cell populations derived from bone marrow or umbilical cord blood. Here, we have transplanted a well-defined unrestricted somatic stem cell isolated from human umbilical cord blood into an acute traumatic spinal cord injury of adult immune suppressed rat. Grafting of unrestricted somatic stem cells into the vicinity of a dorsal hemisection injury at thoracic level eight resulted in hepatocyte growth factor-directed migration and accumulation within the lesion area, reduction in lesion size and augmented tissue sparing, enhanced axon regrowth and significant functional locomotor improvement as revealed by three behavioural tasks (open field Basso-Beattie-Bresnahan locomotor score, horizontal ladder walking test and CatWalk gait analysis). To accomplish the beneficial effects, neither neural differentiation nor long-lasting persistence of the grafted human stem cells appears to be required. The secretion of neurite outgrowth-promoting factors in vitro further suggests a paracrine function of unrestricted somatic stem cells in spinal cord injury. Given the highly supportive functional characteristics in spinal cord injury, production in virtually unlimited quantities at GMP grade and lack of ethical concerns, unrestricted somatic stem cells appear to be a highly suitable human stem cell source for clinical application in central nervous system injuries.

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Newswise Stem cells in early embryos have unlimited potential; they can become any type of cell, and researchers hope to one day harness this rejuvenating power to heal disease and injury. To do so, they must, among other things, figure out how to reliably arrest stem cells in a Peter Pan-like state of indefinite youth and potential. Its clear the right environment can help accomplish this, acting as a sort of Neverland for stem cells. Only now are scientists beginning to understand how.

New collaborative research between scientists at Rockefeller University and Memorial Sloan Kettering Cancer Center offers an explanation: Stem cells can rewire their metabolism to enhance an erasure mechanism that helps them avoid committing to a specific fate; in turn, this improves stem cells ability to renew themselves.

Experiments described today (December 10) in Nature link metabolism, chemical reactions that turn food into energy and cellular building materials, with changes to how genes are packaged, and, as a result, read. It turns out that by skewing their metabolism to favor a particular product, stem cells can keep their entire genome accessible and so maintain their ability to differentiate into any adult cell.

All of the principal enzymes charged with modifying DNA as well as DNA-histone protein complexes called chromatin use the products of cellular metabolism to do so. But how specific alterations in metabolic pathways can impact gene expression programs during development and differentiation has remained a mystery, says lead researcher C. David Allis, Joy and Jack Fishman Professor and head of the Laboratory of Chromatin Biology and Epigenetics. This collaborative effort with Craig Thompsons lab at Memorial Sloan Kettering reveals that the nutrients a stem cell uses, and how it uses them, can contribute to a cells fate by changing the chromatin landscape and, as a result, influencing gene expression.

These changes are epigenetic, meaning they do not affect genes themselves, instead they alter how DNA is packaged, making it more or less accessible for expression. In this case, researchers were interested in a specific type of epigenetic change: chemical groups, known as methyl groups, that attach to chromatin. Generally, the addition of these methyl groups compacts and silences regions of the genome. To maintain their ability to give rise to any type of cell in the body, stem cells need all of their genome available, and so they must keep methylation in check.

Some epigenetic marks, such as methyl groups, are themselves products of metabolism metabolites. Whats more some other metabolites participate in the reactions that remove methylations, making genes available for expression. After joining the Allis Lab, postdoc Bryce Carey presented an idea that tied these concepts together: What if in stem cells the changes to chromatin reflect a unique metabolism that helps to drive reactions that help to keep chromatin accessible? This connection would explain how embryonic stem cells are so uniquely poised to activate so much of their genomes, Carey says.

Mouse embryonic stem cells grown in a medium known as 2i are much better at renewing themselves than those grown in the traditional medium containing bovine serum, although researchers dont fully understand why. Carey and co-first author Lydia Finley, a postdoc in Thompsons metabolism-focused lab, compared the metabolism of cells grown in both media.

Carey and Finley first noticed that the 2i cells did not require glutamine, an amino acid most cells need to make the metabolite alpha-ketoglutarate, an important player in a series of metabolic reactions known as the citric acid cycle and a metabolite that had also been previously implicated in regulation of methylations on chromatin. Even without glutamine, however, the 2i cells managed to produce significant amounts of alpha-ketoglutarate.

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Discovery Links Shift in Metabolism to Stem Cell Renewal

When Mount Sinai Hospital researcher Dr. Andras Nagy initiated a huge project to demystify the process by which specialized cells become stem cells, he wasnt expecting to discover a whole new type of stem cell.

Its a big finding, because identifying a new class of stem cells means a 100 per cent increase in possible sources of cells for therapeutic use.

He describes a stem cell as a blank tablet. They hold great potential to treat diseases that result from damaged tissue or loss of cells, such as Alzheimers, spinal cord injuries and blindness.

His latest research, dubbed Project Grandiose because of its sheer scale, has involved employing a team of nearly 50 researchers across four continents to document the process of creating stem cells. These cells called induced pluripotent stem cells, or iPS cells can be used to form any type of cell in the body as an alternative to using the more controversial stem cells derived from embryos.

The findings will be published Thursday in a package of papers in Nature and Nature Communications .

The oldest example of a therapy based on stem calls is bone marrow transplants, which have been performed for more than 40 years.

One of the newest applications of stem cells is treating and preventing the loss of vision using iPS cells. Japan has permitted the use of these cells to regenerate eye tissue this year. A woman in her 70s was the first to receive retinal tissue created from iPS cells to combat a degenerative condition that can lead to blindness.

Nagy characterizes this procedure as an icebreaker, hoping it will lead to further treatment and perhaps even cures in other diseases.

But understanding these cells first is key to safer use.

If we understand this process better and deeper, we will be in a better position to create safer and (more therapeutically useful) cell types in the future, said Nagy.

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Pioneering Toronto scientists latest research to demystify stem cells

A frozen vial of human embryonic stem cells is shown at the University of Michigan Center for Human Embryonic Stem Cell Research Laboratory in Ann Arbor, Mich., on Oct. 22, 2008. THE ASSOCIATED PRESS/Paul Sancya

A Canadian-led international team of researchers has begun solving the mystery of just how a specialized cell taken from a persons skin is reprogrammed into an embryonic-like stem cell, from which virtually any other cell type in the body can be generated.

The research is being touted as a breakthrough in regenerative medicine that will allow scientists to one day harness stem cells to treat or even cure a host of conditions, from blindness and Parkinsons disease to diabetes and spinal cord injuries.

Besides creating the reprogramming roadmap, the scientists also identified a new type of stem cell, called an F-class stem cell due to its fuzzy appearance. Their work is detailed in five papers published Wednesday in the prestigious journals Nature and Nature Communications.

Dr. Andras Nagy, a senior scientist at Mount Sinai Hospital in Toronto, led the team of 50 researchers from Canada, the Netherlands, South Korea and Australia, which spent four years analyzing and cataloguing the day-by-day process that occurs in stem cell reprogramming.

The work builds on the 2006-2007 papers by Shinya Yamanaka, who showed that adult skin cells could be turned into embryonic-like, or pluripotent, stem cells through genetic manipulation, a discovery that garnered the Japanese scientist the Nobel Prize in 2012.

Nagy likened the roughly 21-day process to complete that transformation to a black box, so called because scientists did not know what went on within the cells as they morphed from one cell type into the other.

It was just like a black box, Nagy said Wednesday, following a briefing at the hospital. You start with a skin cell, you arrive at a stem cell but we had no idea what was happening inside the cell.

Nagys team set about cataloguing the changes as they occurred by removing cells from culture dishes at set points during the three-week period, then analyzing such cellular material as DNA and proteins present at that moment.

The result is a database that will be available to scientists around the world, which the team hopes will spur new research to advance the field of stem cell-based regenerative medicine.

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Stem cell breakthrough holds promise for treating blindness, Alzheimers