Spinal cord | Define Spinal cord at Dictionary.com
By LizaAVILA
Contemporary Examples
Infections can strike joints, airways, the lungs, the brain and the tissues lining the spinal cord, or the bloodstream.
Other spinal cord regeneration efforts involve using stem cells to regrow damaged or lost neurons.
As a result, Smith lost his right arm underneath the elbow and parts of his leg, hip and spinal cord.
Generally, a human will only be infected if they eat the nerve tissuebrains or spinal cordof an infected animal.
The bulleta live fire bulletexited through his back but not before severing his spinal cord.
British Dictionary definitions for spinal cord Expand
the thick cord of nerve tissue within the spinal canal, which in man gives rise to 31 pairs of spinal nerves, and together with the brain forms the central nervous system
spinal cord in Medicine Expand
spinal cord n. The thick, whitish cord of nerve tissue that extends from the medulla oblongata down through the spinal column and from which the spinal nerves branch off to various parts of the body. Also called spinal marrow.
spinal cord in Science Expand
The long, cordlike part of the central nervous system that is enclosed within the vertebral column (spine) and descends from the base of the brain, with which it is continuous. The spinal cord branches to form the nerves that convey motor and sensory impulses to and from the tissues of the body.
spinal cord in Culture Expand
The thick column of nerve tissue that extends from the base of the brain about two thirds of the way down the backbone. As part of the central nervous system, the spinal cord carries impulses back and forth between the brain and other parts of the body through a network of nerves that extend out from it like branches.
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Spinal cord | Define Spinal cord at Dictionary.com
Familial ALS Linked to Both Neuron and Astrocyte Pathology – Alzforum
By LizaAVILA
17 Jun 2017
Animal models and engineered cells have contributed their fair share of insight into the pathogenesis of amyotrophic lateral sclerosis (ALS), but researchers do not often have the opportunity to peer into afflicted human neurons. In a study published May 30 in Cell Reports, researchers led by Rickie Patani and Sonia Gandhi at University College London offer a new model for human disease by using motoneurons and astrocytes derived from amyotrophic lateral sclerosis (ALS) patient stemcells.
The researchers generated spinal motoneurons and astrocytes from induced pluripotent stem cells (iPSCs) of ALS patients carrying mutations in the valosin-containing protein (VCP) gene. VCP mutations account for 2 percent of familial ALS cases and have been linked to other disorders as well, including hereditary inclusion body myopathy with Paget disease of bone and frontotemporal dementia (IBMPFD), jointly termed multisystemproteinopathy.
Previously, researchers reported that VCP mutations promoted the formation of cytoplasmic aggregates of TDP-43,one of the hallmarks of motoneuron disease, and that these aggregates were at least partially responsible for the VCP-induced neurodegeneration seen in a fly model (Neumann et al., 2007; Ritson et al., 2010).
Few studies have tested what VCP mutations do to human neurons, however. Using an iPSC-derived neuronal model, Virginia Kimonis at the University of California, Irvine, found that the VCP R155H mutation boosted cells levels of TDP-43 and several proteins involved in protein disposal (Dec et al., 2014).
In search of a more complete view of how VCPs effects unfold over time, first co-authors Claire Hall, Zhi Yao, Minee Choi, and Giulia Tyzach used iPSC-derived populations of spinal cord motoneurons and astrocytes to track several cellular functions. The cells originated from two patients (four clones) with VCP mutations and three healthycontrols.
TDP-43 (red) stays in the nucleus (blue) in control motoneurons (left) but leaks into the cytoplasm in three-day old VCP-mutant motoneurons (right). [Cell Reports, Hall et al.,2017.]
More VCP-mutant motoneurons than controls died; they also made fewer synapses with their neighbors, and had less intense and fewer coordinated bursts of firing than controls. Monitoring the whereabouts of TDP-43, the authors found no difference between mutant and normal cells when the cells were at an early stage of differentiation, as neural precursor cells. But by the third day after becoming motoneuron-like, the mutant cells were leaking TDP-43 from their nuclei into the cytoplasm (see image above). At the same time, they had increased levels of markers of endoplasmic reticulum (ER) stress, became less able than controls to survive an ER stress assay, and began producing reactive oxygen species (ROS) at a higherrate.
By day 17, the mutant motoneurons had sickly ER tubules. Some were swollen, others cozied up to mitochondria, both signs of continued ER stress. The neurons also had low mitochondrial membrane potential and low glutathione levels, indicating mitochondrial dysfunction and oxidative stress,respectively.
Putting these findings together, Patani and Gandhi suggested that cyotosolic TDP-43 generates ER stress, which next triggers increased tethering of the ER to mitochondria. This could depolarize mitochondria, which could impair mitochondrial function and ultimately lead to high levels of free radical production and oxidative stress.The findings support a role for TDP-43 wreaking havoc in the cytoplasm, saidPatani.
The mechanism that links TDP-43 and ER stress remains uncertain, said Kimonis. Patani and Gandhi acknowledge the link is probably complex, noting that, in addition to cyotplasmic TDP-43 aggregates inducing ER stress, ER stress itself seems to drive TDP-43 leakage (Walker et al., 2013).
The researchers then probed how mutant VCP might affect astrocytes. Studies of astrocytes in other ALS models have yielded varying results depending on the mutation. Superoxide dismutase 1 (SOD1) mutations, for example, can turn astrocytes into motoneuron killers (see Oct 2011 news),but it is unclear if they endanger the survival of the astrocytes themselves. Conversely, mutations in TDP-43 seem to sicken astrocytes without making them toxic to neighboring neurons (Serio et al., 2013).
On the left, healthy astrocytes (red) protect VCP-mutant motoneurons (yellow) from death (green). VCP-mutant astrocytes are much less protective (right). [Courtesy of ZhiYao.]
Interestingly, Patani and Gandhi found that VCP mutations had both cell-autonomous and non-cell-autonomous effects. They increased astrocytes risk of death and rendered them less able to support the survival of both control and VCP-mutant motoneurons (see image at right). The authors also found that the cell-autonomous effects on astrocytes differed from those in motoneurons. VCP-mutant astrocytes seemed more resistant when challenged with an ER stressor and had only a transient drop in mitochondrial membrane potential and a transient increase in ROS, with no change in glutathionelevels.
The study is a validation of key prior findings using iPS-derived neurons and glia from patients, which is a nice advance, wrote Paul Taylor at St. Jude Childrens Research Hospital in Memphis, Tennessee. The characterization of the role of glia is also interesting and potentially important.
Looking ahead, Patani and Gandhi want to generate isogenic controls, that is, cells derived from the same patients but with repaired VCP mutations. They also want to create iPSC-derived upper motoneurons, and to better understand muscle pathology, Kimonis hopes to extend her investigation of iPSC-derived muscle cells (Llewellyn et al., 2017).
Other model systems will be of value, too. iPSC-derived models are not equivalent to the decades-old cells inhabiting an adult nervous system. A study of dopaminergic neurons, for example, revealed that gene expression and DNA methylation patterns differed between iPSC-derived neurons and their in vivo counterparts (Roessler et al., 2014).
In the long term, Patani hopes that shedding light on the sequence of events that marks motoneuron degeneration in human cells will enable researchers to more effectively search for ALS therapies.MarinaChicurel
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Familial ALS Linked to Both Neuron and Astrocyte Pathology - Alzforum
Eva Feldman steps down as Taubman Institute director – The Detroit News
By JoanneRUSSELL25
Eva Feldman, M.D., Ph.D.(Photo: Detroit News file photo)
World-renowned researcher Dr. Eva Feldman is stepping down as director of the University of Michigans A. Alfred Taubman Medical Research Institute, an organization that supports research for cures for debilitating and deadly diseases.
Feldman was the founding director of the institute launched by philanthropist and businessman A. Alfred Taubman, who died in 2015 at age 91. Feldman spent the last 10 years at the helm as the institute developed new drugs, surgeries and therapies for diseases such as adult and childhood cancer, diabetes, cardiovascular disease and, Feldmans research interest, amyotrophic lateral sclerosis.
When Alfred and I began the institute 10 years ago together, we had a vision of creating an institute where clinician scientists would take their most novel discoveries from their laboratory and translate that into patient clinical trials, said Feldman, adding that the team of four has grown to over 200. Weve really been able to realize that vision.
Feldman, 65, said Thursday that she raised the institute from its infancy.
Its now time for the next person to take it through its adolescence, because its still growing, she said. So it just seemed the perfect time to pass the baton to someone to have the next 10 best years of their life the way I just had the 10 best years of my life.
The institute will select the next director soon, Feldman said.
A Russell N. DeJong Professor of Neurology, Feldman will continue to run the ALS clinic at UM and her own laboratory, Program for Neurology Research & Discovery. Her team of 30 scientists have spent years working on human clinical trials that may lead to a treatment for ALS, a neurological disease also known as Lou Gehrigs disease. Most ALS patients lose the ability to walk and talk, and death may occur within three to five years of diagnosis.
In 2010, Feldman started the first human clinical trial approved by the U.S. Food and Drug Administration to use stem cells to treat ALS. The FDA recently approved Feldman to move onto the final, nationwide phase of the trial that involves injecting stem cells into the spinal cord of ALS patients. If approved, the drug could be marketed to patients.
Besides focusing on the trial, Feldman said shell have more time to do a deep dive into why people with diabetes get neurological complications.
She also was elected to the National Academy of Medicine and plans to use her position to help others understand the value of philanthropy.
Philanthropy can be a game changer Al Taubman showed us that and one of my goals is to help other clinician scientists throughout the country understand the process, she said.
Feldman said shes much closer to a cure for ALS, but were not there yet. For now, she wants to press the pause button on the disease.
When the patient comes to me, I want to be able to offer them a therapy so they will stay right where they are the first day I see them, she said. I dont think that stem cells will necessarily make people go back to where they are before they got ill, but if we can stop the disease in its tracks, my patients will be happy and I will be happy.
Feldman was named a 2011 Detroit News Michiganian of the Year. Shes served as the president of the American Neurological Association, the third woman to hold the position in 130 years, and was named one of Americas Top Doctors in 2016.
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Eva Feldman steps down as Taubman Institute director - The Detroit News
Banks targets embryonic stem cell research funding – Fort Wayne Journal Gazette
By JoanneRUSSELL25
Rep. Jim Banks, R-3rd, introduced legislation Thursday that would prevent the use of federal funds for stem cell research involving human embryos.
Banks' bill would direct the U.S. Department of Health and Human Services to give priority to medical research with the greatest potential for near-term clinical benefit in human patients and that does not use stem cells from destroyed, discarded or created embryos.
Scientists say embryonic stem cells show potential for transforming into other cells that might repair tissue damaged by disease or injury. Human embryonic stem cells used in research come from donated, unused fertilized eggs developed for in vitro fertilization procedures.
Adult blood stem cells are used to treat leukemia, and adult neural stem cells have been tested for brain disorders and spinal cord injuries.
This bipartisan bill prioritizes stem cell research that has a real impact on patients suffering right now while ensuring that research is conducted ethically without destroying human embryos, Banks, a freshman lawmaker from Columbia City, said in a statement.
Rep. Dan Lipinski, D-Ill., co-sponsored Banks' bill, which is called the Patients First Act of 2017.
The Dickey-Wicker Amendment of 1996 prohibited HHS from funding research using created or destroyed human embryos. But a federal court ruled in 2011 that Dickey-Wicker was ambiguous and did not ban research using stem cells from in vitro fertilization.
The Alliance for Regenerative Medicine, a coalition of medical companies, research institutions and patient advocacy groups that support embryonic stem cell research, had little to say Thursday about Banks' legislation.
As an organization representing the broader global regenerative medicine sector, our position is that we are in favor of government funds supporting the best science in an effort to speed safe and efficacious products to patients in need, Lyndsey Scull, senior communications director for ARM, said in an email.
Scull said ARM would monitor Banks' bill in the legislative process.
Banks' proposal states it would promote the derivation of pluripotent stem cell lines without the creation of human embryos for research purposes and without the destruction or discarding of, or risk of injury to, a human embryo.
The National Institutes of Health defines pluripotent stem cells as those that can give rise to any type of cell in the body except those needed to support and develop a fetus in the womb. They come from embryos and fetal tissue, although induced pluripotent stem cells are genetically reprogrammed cells taken from adult tissues.
In May, Banks led a letter signed by 40 other Republican House members that asked President Donald Trump to replace Dr. Francis Collins as the director of the NIH because of Collins' support for human embryonic stem cell research. Trump announced last week that he is retaining Collins, a geneticist nominated for NIH chief by President Barack Obama and confirmed by unanimous consent by the Senate in 2009.
The NIH is an HHS agency.
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Banks targets embryonic stem cell research funding - Fort Wayne Journal Gazette
Stem Cell Therapy Offers Hope for Multiple Sclerosis Remission – Healthline
By JoanneRUSSELL25
By combining an experimental stem cell treatment with a nanoparticle delivery system, researchers may eventually stop MS and other autoimmune diseases.
An innovative stem cell therapy could change how we treat multiple sclerosis (MS), but are we any closer to a cure?
The work of Dr. Su Metcalfe, founder and chief scientific officer of the biotech company LIFNano, appears to be breathing new life into that hope.
Metcalfe and her team developed a way to fight MS by using the bodys own natural mechanisms but it hasnt been tested in humans yet.
MS is an inflammatory and neurodegenerative autoimmune disease that can result in an array of neurological symptoms including fatigue, muscle spasms, speech problems, and numbness. It is caused by the immune system attacking myelin, the insulating coating that runs along the outside of nerve cells. The result is damage to the brain and central nervous system.
The disease currently affects roughly 2.5 million people worldwide. About 200 new cases are diagnosed each week in the United States.
LIFNano uses a new treatment based on LIF a stem cell protein that forms naturally in the body to signal and regulate the immune systems response to myelin.
LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy, Metcalfe recently told Cambridge News.
In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged, she said.
Metcalfe has spent years studying LIF, but only recently realized its potential for treatment likening it to an on/off switch for the immune system.
However, once she discovered its potential, there were almost immediate problems in its application. One of the earliest was how quickly LIF breaks down once it is administered into the body.
If you try just to inject it into a patient, it dissipates or disappears in about 20 minutes, Olivier Jarry, CEO of LIFNano, told Healthline.
That makes it unusable in a clinic. You would have to have some kind of pump and inject it continually.
A breakthrough came for Metcalfe when she took findings from her studies of LIF and applied them to nanotechnology. The treatment she is now developing relies on nanospheres derived from a well-established medical polymer known as PLGA, which is already used in materials like stitches. And because it is biodegradable, it can be left to dissolve inside the body.
Storing LIF inside these PLGA nanospheres before administering them into the bloodstream allows for a sustained dose over the course of several days.
The process differs significantly from the current drugs used to treat MS. These treatments most often fall under the category of drugs known as immunosuppressors, which inhibit the bodys overall immune system response.
LIF is theoretically much more precise than immunosuppressors, and should keep the immune system functioning against harmful infections and disease.
Were not using any drugs, said Metcalfe. Were simply switching on the bodys own systems of self-tolerance and repair. There arent any side effects because all were doing is tipping the balance. Autoimmunity happens when that balance has gone awry slightly, and we simply reset that.
The team cautions that LIF therapy is still several years away.
While some outlets have run wild with Metcalfes research, announcing that a cure for MS is right around the corner, those headlines are speculative.
Some MS advocacy groups have even made public statements calling coverage of her work premature and irresponsible.
Jarry told Healthline that LIFNano is expecting to enter FDA phase I trials in 2020. This would be the first time that it is used in human subjects. But even if the treatment proves to be safe and effective, the soonest it could be on the market is 2023, he estimated.
The main focus of LIF therapy is now on MS. But it has potential for treating other autoimmune diseases including psoriasis and lupus.
We are optimistic in the sense that we may provide a long-term remission for patients with MS, said Jarry.
Is it a cure? Wed love at some point to use the term cure, but we are very cautious.
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Stem Cell Therapy Offers Hope for Multiple Sclerosis Remission - Healthline
Rabbi has warning after concluding reviving dead ‘clearly possible’ – WND.com
By Sykes24Tracey
El Grecos Resurrection
A rabbi has issued a warning after concluding that raising the dead is clearly possible.
Its not for man to do.
The comments come from Rabbi Moshe Avraham Halperin and were reported by Breaking Israel News.
If they are able to revive a person from total brain death, it will be considered techiyat hamaytim [resurrection], he told BIN. Torah laws puts limits on man, forbidding him from some areas which are strictly divine. Reviving the dead is one of them.
Further, another rabbi said such experiments never will succeed.
The Rambam states that we must believe that the resurrection of the dead will happen when it is Gods will for it to take place and at no other time, Rabbi Yosef Berger, of King Davids Tom on Mount Zion in Jerusalem, told the news agency.
Transhumanism: Recreating humanity reveals the secret ways scientists are using technologyto pursue immortality, omniscience and ultimate power. Now available in the WND Superstore.
He was citing the 13 Principles of Faith set down by Rabbi Moses ben Maimon, whose 12th century work established him as a Torah authority and gave him the acronym Rambam.
He stated, I believe by complete faith that there will be a resurrection of the dead at the time that will be pleasing before the Creator.
That, Berger suggested, means, Not only does this effort by scientists go against this principle of faith, but we know that true resurrection can only happen by Divine will. Resurrection of the dead is described in depth, and it is proof of Gods rule over the physical world. But it is also stated that before the Messiah, there is no return from the grave.
The comments come on the heels of plans by a biomedical company, Bioquark, a startup based in Pennsylvania, to experiment with stem cells in an attempt to revive brain-dead patients.
BIN reported the company said it would launch experiments before the end of the year on such patients.
The trial will involve a multi-pronged approach, involving injecting stem cells and peptides into the spinal cords, electrical nerve stimulation, and laser therapy. The researchers hope this will grow new neurons and spur them to connect to each other, bringing the brain back to life, BIN reported.
The story pointed out that Bioquark reportedly tried such an experiment in India in 2016 but it was not with the approval of the nations drug regulators.
Amar Jesani, editor of the Indian Journal of Medical Ethics in Mumbai, cautioned that even partial success would traumatize families that had come to terms with a situation they believed irreversible. In point of fact, no families permitted their loved ones to be part of the experiment, BIN reported.
Halperin said, This is like using genetics to create a new form of life. There are realms that are strictly divine. Resurrection of the dead is clearly possible. It is definitely going to happen after the Messiah, but it restricted to God.
It was reported about a year ago that Bioquark and another company were embarking on the Reanima project, using a new drug formula involving stem cells.
Their plan was to use neurons, proteins, peptides and more that would create a microenvironment in which the stems cells can mature.
The report said, Inspired by organisms like salamanders that can regrow severed or damaged tails, Bioquark researchers have been developing regenerative treatments for a host of uses, from cancer to spinal cord injuries.
WND had reported when the company was given the green light on the visionary project.
The company said then it was capable of creating dynamics in mature tissues that are normally only seen during human fetal development, as well as during limb and organ regeneration in organisms like amphibians.
Christian author and filmmaker Tom Horn, at that time, said scientists are redefining what it means to be human, with the goal of transcending humanity.
Right here in North Carolina at your university, they have what is called a transgenic lab, which means they have mice that have human genetic material, for testing to see if the human parts in that animal are responding, he told TV host Sid Roth in a recent interview.
Using a gene-editing technique, one university lab cured cancer in a group of rats, but the unintended consequences were that the rats started aging very quickly and died at half-life, and nobody knows why that happened, Horn said. There is a danger in playing God because youre not God and you dont know.
Transhumanism: Recreating humanity reveals the secret ways scientists are using technologyto pursue immortality, omniscience and ultimate power. Now available in the WND Superstore.
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Rabbi has warning after concluding reviving dead 'clearly possible' - WND.com
Eva Feldman: Stepping down, not stopping – Crain’s Detroit Business
By Sykes24Tracey
Eva Feldman is stepping down as director of the A. Alfred Taubman Medical Research Institute at the University of Michigan, but at the age of 65, she has no intention of retiring.
She was the founding director of the institute created with tens of millions of dollars from mall mogul A. Alfred Taubman to fund research into intractable diseases like Alzheimer's, ALS and diabetes and ran it for 10 years. It is time, she says, for her next, but not necessarily last, 10-year plan.
"I birthed a baby and it's grown to be 10. It's a tweener, now, and ready for someone else to take it to adolescence," she told Crain's, following a meeting she had with one of the finalists of a national search to replace her.
Feldman said she expects a replacement to be named by the end of June.
"We started with four people, and now we have more than 200 investigators," she said. "We're strongly established, very robust. We have the best scientist-clinicians at the university. It's time for the next director to take over. There are other things I want to do, now. This takes up a lot of time, and I want to spend more time on my own research."
Feldman will continue to run the ALS Clinic at UM. Each Tuesday, she and a small staff diagnose and treat patients with amytrophic lateral sclerosis, sometimes known as Lou Gehrig's disease. The Russell N. DeJong Professor of Neurology at the U-M Medical School, she will also continue to run her own 30-scientist laboratory, the Program for Neurology Research & Discovery.
She said her new 10-year plan has three major goals.
This would be the last trial before the procedure is approved.
As soon as Feldman was done enumerating those goals, she enumerated two more.
She wants to help get more women into research positions at major universities. "More women than men graduate from the medical school at Michigan, but there are just a blip of women who are full professors," she said.
Last Wednesday, she and the Taubman Institute hosted a symposium called "Strategies to Empower Women to Achieve Academic Success," with keynote speakers on gender equality in academic medicine and a panel discussion on how to use negotiation and networking skills for career advancement.
And she wants to raise the $5 million it will take to fund the large-animal studies she needs to do before she can launch FDA human trials using embryonic stem cells to treat Alzheimer's. She said stem-cell trials with mice with dementia have been promising.
Researchers injected two groups of Alzheimer's mice, one group with a saline solution to serve as a control, the other with stem cells. Both of those groups and a group of healthy mice were then put through three tests of cognition, including one that required finding a platform hidden in a pool of water.
Previously, both sets of Alzheimer's mice flunked the cognition tests. After the stem-cell injections, the Alzheimer's mice injected with cells performed the tests as well as healthy mice. The Alzheimer's mice injected with saline solution flunked the tests.
"I am persistent. I will get that funded," she said.
Feldman took a sabbatical this spring to go to Australia, where she helped the Royal Children's Hospital in Melbourne set up a neuropathy-screening program for children with diabetes, which dovetails with one of her clinical trials treating neuropathy in children. And in September she heads to Chennai, India, to set up a similar program at a hospital there.
Neuropathy is a painful condition resulting from the nerve damage that diabetes can cause.
Feldman has published more than 350 peer-reviewed articles, 60 book chapters and three books and has had continuous funding from the National Institutes of Health for more than 20 years, including 12 current grants totaling about $5.5 million.
She is past president of the Peripheral Nerve Society and served as president of the American Neurological Association from 2011 to 2013, the group's third woman president in 130 years. Last June, she was named among the 100 most influential women in Michigan by Crain's.
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Eva Feldman: Stepping down, not stopping - Crain's Detroit Business
Patients volunteer for pioneering treatment by Cambridge scientist on verge of curing multiple sclerosis – Cambridge News
By Sykes24Tracey
Scores of people living with multiple sclerosis have put themselves forward for clinical trials as part of a Cambridge scientists bid to develop a treatment to cure the disease.
MS, an auto-immune condition which affects 2.3 million people around the world, attacks cells in the brain and the spinal cord, causing an array of physical and mental side effects including blindness and muscle weakness.
At the moment theres no cure, but Cambridge scientist Su Metcalfe and her company, LIFNano, hope to change that.
On Sunday the News told how Su has married one of the bodys cleverest functions with some cutting-edge technology. The natural side of the equation is provided by a stem cell particle called a LIF.
Su said: I discovered a small binary switch, controlled by a LIF, which regulates inside the immune cell itself. LIF is able to control the cell to ensure it doesnt attack your own body but then releases the attack when needed.
That LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy. In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged.
So I thought, this is fantastic. We can treat auto-immune disease, and weve got something to treat MS, which attacks both the brain and the spinal cord. So you have a double whammy that can stop and reverse the auto-immunity, and also repair the damage caused in the brain.
Sus company LIFNano has already attracted two major funding awards, from drug firm Merck and the Governments Innovate UK agency.
The company hopes to attract more investment, with the aim of starting clinical trials in 2020.
Off the back of the article regarding Sus work, people commented in their droves offering to trial Dr Metcalfes treatment, which is being touted as a possible cure.
Many also shared their experiences of living with MS.
James Stokes, 52 from Reading, was just one of many to comment on our story.
He told the News : I was diagnosed with MS on my 18th birthday of all days, but I wasnt told then that it was MS.
I had a big attack and was put in hospital; I lost my sight and couldnt speak or walk.
Even then I still wasnt officially told it was MS until a few years later.
About 10 years ago, things started to go downhill and I found myself basically housebound; I can still walk but not a great distance.
I use a walking stick, and have to use a wheelchair if Im going long distances.
MS has robbed me of my life, but when I saw this article there was a glimmer of hope.
Id love to know more about it [the process] and would be willing to take part in clinical trials.
If they could reverse my MS that would mean everything. At the moment, I feel as if Ive been given a life sentence but with no parole.
If a cure for MS is found, I could get a new lease of life and a lot more hope.
Many others also commented on the story regarding Sus work.
Debbie Brown wrote: This gives me some hope that things will improve in my lifetime. Diagnosed with RRMS in 2000. Very interested in registering for a clinical trial.
Amy Keighley said: My father was diagnosed with MS in 1996. He has been on several medications and is currently being treated by Dr Tornatori at Georgetown University Medical Center.
He fights every day and lives life to the fullest. He and my mother have been together since 1950. They were 15 years old. They are both retired from operating a jewellery store together for 23 years.
They try to travel every month and my dad's spirits remain high. I would like to ask that my dad be considered for the clinical trial.
Asterias Biotherapeutics to Present New 9-Month Efficacy Data from AST-OPC1 SCiStar Study at the International … – PR Newswire (press release)
By Sykes24Tracey
Workshop Details
Title: Clinical Development of hESC-derived Oligodendrocyte Progenitors for the Treatment of Spinal Cord injury Date: Tuesday, June 13, 2017 Time: 10:45 - 11:10 AM Room: 205ABC
Poster Presentation Details
Title: Safety and Efficacy of Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury Poster Session III-ODD Poster Board F-1225 Date: Friday, June 16, 2017 Time: 6:00 - 7:00 PM
About the SCiStar Trial
The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.
Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.
Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).
About AST-OPC1
AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.
In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.
FORWARD-LOOKING STATEMENTS
Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/asterias-biotherapeutics-to-present-new-9-month-efficacy-data-from-ast-opc1-scistar-study-at-the-international-society-for-stem-cell-research-isscr-2017-annual-meeting-300470804.html
SOURCE Asterias Biotherapeutics, Inc.
http://www.asteriasbiotherapeutics.com
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Asterias Biotherapeutics to Present New 9-Month Efficacy Data from AST-OPC1 SCiStar Study at the International ... - PR Newswire (press release)
Origin of age-related muscle loss discovered – Bel Marra Health
By raymumme
Home General Health Origin of age-related muscle loss discovered
When were young, we feel like we can accomplish anything. We can go anywhere and do anything because we dont feel encumbered by our physical limitations. However, when we age, we start to lose this feeling of empowerment. Our once-impressive physical physique fades away, leaving us weak and defenseless.
However, new research into the reasons why we lose muscle cells as we age may lead to the development of new drugs that can slow the process of muscle decline.
It was previously thought that the driving factor behind age-related muscle decline was the loss of motor neuronsnerve cells in the spinal cord controlling muscle. Starting in our 30s, we begin to notice that activities that once came easily are now difficult.
When we reach our 70s and 80s, we become weak and frail, having to depend on others to lend us their strength on a daily basis. No amount of diet and exercise can prevent this inevitable result.
The pursuit of preserving our youth has been a goal for many researchers and scientists. Understand how and why the body ages over time is something worth pursuing in the hopes of improving lives.
Research using mouse models has found that muscle stem cells play a very important role in lifelong maintenance of muscle. This challenges the current, widely accepted theory of motor neurons.
Muscle stem cell pools were seen to reside in muscle tissue that responds to exercise and injury. These stem cells are directly involved in repair and growth of muscles. It is the dying off of these stem cells that is theorized to be the driving factor for muscle loss.
Mice that were genetically altered to prevent muscle stem cell loss maintained healthier muscles in old age.
Subsequently, no evidence was found linking motor neurons to age-related muscle loss.
I think weve shown a formal demonstration that even for aging sedentary individuals, your stem cells are doing something. They do play a role in the normal maintenance of your muscle throughout life, said study author Joe Chakkalakal, Ph.D., assistant professor of Orthopaedics in the Center for Musculoskeletal Research at the University of Rochester Medical Center.
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Origin of age-related muscle loss discovered - Bel Marra Health
Stem cell-based spinal cord therapy expanded to more patients – The San Diego Union-Tribune
By Sykes24Tracey
An experimental therapy to repair spinal cord injury with stem cell-derived tissue is progressing smoothly, according to a leader of that trial who spoke at a conference on stem cell therapy.
The Phase 1 safety trial is proceeding with no complications, said Dr. Joseph Ciacci, a University of California San Diego neurosurgeon. The trial is being conducted at the universitys Sanford Stem Cell Clinical Center. The conference was held last week at the Sanford Consortium for Regenerative Medicine in La Jolla.
With safety looking good, the green light has been given to treat more patients, Ciacci said. However, to produce effectiveness, more cells will need to be transplanted.
Four patients have been treated with neural stem cells, injected into the spinal cord. They had experienced complete loss of motor and sensor function below the injury. They had been injured between 1 and 2 years previously.
Moreover, the cells show signs of integrating with the surrounding tissue in animal studies, Ciacci said. If the preliminary evidence holds up, Ciacci and colleagues plan to submit a paper detailing the results.
Curing paralysis from spinal cord injury was a big selling point for those who successfully advocated Proposition 71, which authorized selling $6 billion in state bonds to establish and fund the California Institute for Regenerative Medicine, or CIRM. The institute got $3 billion, the remaining half is going for interest over the life of the bonds.
While CIRM has been under pressure to show results, doctors are taking great care to establish safety first in the spinal cord treatment, because of potential risks in the procedure.
We are now enrolling and recruiting for the second cohort, which is for chronic cervical spinal cord injuries, Ciacci said. They are medically classified as C5-C7 ASIA A Complete.
Chronic injuries need to have taken place more than 1 year before treatment. For this study, the injury must also be under two years old. The trial is being conducted at UCSD with Ciacci serving as the principal investigator.
For more information on the Phase I Chronic SCI study, contact Ciaccis research group at (619) 471-3698, nksidhu@ucsd.edu.
In addition, the researchers have been approved to start another spinal cord injury trial with a different set of cells. These oligodendrocyte progenitor cells, derived from embryonic stem cells, can turn into several different types of neural cells.
The trial, sponsored by Asterias, treats newly injured patients, between 14 and 30 days after injury.
For more information on the Asterias trial, contact the UCSD Alpha Stem Cell Clinic at 858-534-5932 alphastemcellclinic@ucsd.edu or visit http://www.scistar-study.com and j.mp/ucsdast.
Asterias acquired the technology from Geron, which had undertaken the work with a CIRM grant. Geron later canceled the work and refunded the money to CIRM. Asterias got funding from CIRM to continue the work.
The Asterias trial will use the same technique as used with the Chronic SCI trial, a technique which can improve safety, Ciacci said. The cells will be injected in a series of progressively larger amounts that may give evidence of the dose relates to effectiveness, although safety remains the main concern.
This cell line is cryopreserved, its sent to us as a single dose the day of surgery, Ciacci said. Were going to study different doses 2 million, 10 million, 20 million cells per injection. Its going to be a direct injection, just like what weve done before.
As in previous treatments, patients will also receive immune suppression to prevent rejection of the cells. Likewise, they will be monitored for many years after treatment.
Another trial coming to UCSD will test for efficacy in ALS, Ciacci said.
Ciacci said hes looking for qualified patients for these trials, and urged those in the audience to help find them.
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bradley.fikes@sduniontribune.com
(619) 293-1020
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Stem cell-based spinal cord therapy expanded to more patients - The San Diego Union-Tribune
Bioquark aims to bring brain-dead people back to life next year – Metro – Metro
By NEVAGiles23
The Sun | Bioquark aims to bring brain-dead people back to life next year - Metro Metro It sounds like the plot of a horror movie - but a new trial aims to regenerate the brains of brain-dead people, by injecting them with stem cells. A U.S.... Controversial Study Wants To Resurrect Brain-Dead People ... US Firm Tries 'Reawakening The Dead' With Stem Cells [VIDEO] Company working on a way to reverse death |
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Bioquark aims to bring brain-dead people back to life next year - Metro - Metro
Meet the Cambridge scientist on verge of curing Multiple Sclerosis – Cambridge News
By LizaAVILA
Dr Su Metcalfe is sitting quietly reading through some documents in the lobby of the Judge Business School when I arrive for our interview. It would be easy to walk right past her and not know you were in the presence of a woman who could be on the verge of curing multiple sclerosis.
MS, an auto-immune condition which affects 2.3 million people around the world, attacks cells in the brain and the spinal cord, causing an array of physical and mental side effects including blindness and muscle weakness. At the moment theres no cure, but Su and her company, LIFNano, hope to change that.
Some people get progressive MS, so go straight to the severe form of the disease, but the majority have a relapsing or remitting version, she says.
It can start from the age of 30, and theres no cure, so all you can do is suppress the immune response, but the drugs that do that have side effects, and you cant repair the brain. The cost of those drugs is very high, and in the UK there are a lot of people who dont get treated at all.
But now a solution could be in sight thanks to Su, who has married one of the bodys cleverest functions with some cutting-edge technology. The natural side of the equation is provided by a stem cell particle called a LIF.
Su was working at the universitys department of surgery when she made her big breakthrough: I was looking to see what controls the immune response and stops it auto-attacking us, she explains.
I discovered a small binary switch, controlled by a LIF, which regulates inside the immune cell itself. LIF is able to control the cell to ensure it doesnt attack your own body but then releases the attack when needed.
That LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy. In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged.
So I thought, this is fantastic. We can treat auto-immune disease, and weve got something to treat MS, which attacks both the brain and the spinal cord. So you have a double whammy that can stop and reverse the auto-immunity, and also repair the damage caused in the brain.
Presumably Su, who has been in Cambridge since she was an undergraduate but retains a soft accent from her native Yorkshire, was dancing a jig of delight around her lab at this point, but she soon hit a snag; the LIF could only survive outside the cell for 20 minutes before being broken down by the body, meaning there was not enough time to deploy it in a therapy. And this is where the technology, in the form of nano-particles, comes in.
They are made from the same material as soluble stitches, so theyre compatible with the body and they slowly dissolve, says Su.
We load the cargo of the LIF into those particles, which become the delivery device that slowly dissolve and deliver the LIF over five days. The nano-particle itself is a protective environment, and the enzymes that break it down cant access it. You can also decorate the surface of the particles with antibodies, so it becomes a homing device that can target specific parts of the brain, for example. So you get the right dose, in the right place, and at the right time.
The particles themselves were developed at Yale University, which is listed as co-inventor with Su on the IP. But LIFNano has the worldwide licence to deploy them, and Su believes we are on the verge of a step-change in medicine.
She says: Nano-medicine is a new era, and big pharma has already entered this space to deliver drugs while trying to avoid the side effects. The quantum leap is to actually go into biologics and tap into the natural pathways of the body.
Were not using any drugs, were simply switching on the bodys own systems of self-tolerance and repair. There arent any side effects because all were doing is tipping the balance. Auto-immunity happens when that balance has gone awry slightly, and we simply reset that. Once youve done that, it becomes self-sustaining and you dont have to keep giving therapy, because the body has its balance back.
LIFNano has already attracted two major funding awards, from drug firm Merck and the Governments Innovate UK agency. Su herself is something of a novice when it comes to business, but has recruited cannily in the form of chairman Florian Kemmerich and ceo Oliver Jarry, both experienced operators in the pharma sector. With the support of the Judge, the company hopes to attract more investment, with the aim of starting clinical trials in 2020.
The 2020 date is ambitious, but with the funding weve got and the funding were hoping to raise, it should be possible, says Su.
Weve got everything we need in place to make the nano-particles in a clinically compliant manner, its just a case of flicking the switch when we have the money. Were looking at VCs and big pharma, because they have a strong interest in this area. Were doing all our pre-clinical work concurrently while bringing in the major funds the company needs to go forward in its own right.
Immune cells have been a big part of Sus career, and as we talk, her passion for her subject is obvious. I wanted to understand something that was so simple on one level but also so complex, she says.
The immune cell is the only single cell in the body that is its own unity, so it functions alone. Its probably one the most powerful cells in the body because it can kill you, and if you havent got it you die because you havent got it.
And MS may just be the start for LIFNano.
MS is our key driver at the moment, but its going to be leading through to other major auto-immune disease areas, Su adds.
Psoriasis is high up on our list, and diabetes is another. Downstream there are all the dementias, because a LIF is a major health factor for the brain. So if we can get it into the brain we can start protecting against dementia.
Now that would be something.
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Meet the Cambridge scientist on verge of curing Multiple Sclerosis - Cambridge News
Stem Cell Research Facts – Adult Stem Cell Success
By Dr. Matthew Watson
"I Will Walk Again" The Laura Dominguez Story If there was ever a woman on a mission, its Laura Dominguez. Doctors once told her shed never walk again. And while shes not ready to run a marathon, shes already proving them wrong, with the best yet to come.
An oil spill on a San Antonio freeway is blamed for the car crash that sent Laura and her brother directly into a retaining wall one summer afternoon in 2001. Laura was just 16 years old at the time and the crash left her completely paralyzed from the neck down. Surgeons say she suffered whats known as a C6 vertebrae fracture that severely damaged her spinal cord.
I refused to accept their prognosis that I never would walk again and began searching for other options, says Laura. After stays in several hospitals for nearly a year, Laura and her mother relocated to San Diego, CA so that she could undergo extensive physical therapy. While in California, they met a family whose daughter was suffering from a similar spinal cord injury. They were also looking for other alternatives to deal with spinal cord injuries.
After extensive research and consultations with medical experts in the field of spinal cord injuries, they decided to explore a groundbreaking new surgical procedure using adult stem cells pioneered by Dr. Carlos Lima of Portugal.
The surgery involved the removal of tissue from the olfactory sinus area at the back of the nose--and transplanting it into the spinal cord at the injury site. Both procedures, the harvesting of the tissue and the transplant, were done at the same time. Laura was the tenth person in the world and the second American to have this procedure done and was featured in a special report by PBS called Miracle Cell. (Link to Miracle Cell (PBS) Episode)
Following the surgery she returned to California where she continued with the physical therapy regimen, then eventually returned home to San Antonio. Upon her return home, an MRI revealed her spinal cord was beginning to heal. Approximately 70% of the lesion now looked like normal spinal cord tissue. More importantly to Laura, she began to regain feeling in parts of her upper body and within six months of the surgery regained feeling down to her abdomen.
Improvements in sensory feelings have continued until the present time. She can feel down to her hips, and has regained feeling and some movement in her legs. Lauras upper body has gained more strength and balance and one of the most evident improvements has been her ability to stand and remain standing, using a walker, and with minimal assistance. When she stands she can contract her quadriceps and hamstring muscles.
Every week it seems Im able to do something new, something different that I hadnt done the week before, says Laura.
Now Lauras story is poised to take a new, potentially groundbreaking turn. In the Fall of 2009, she traveled again to Portugal where adult stem cells were extracted from her nose for culturing. As this story is written, she is preparing to fly back to Portugal where scar tissue at her injury site will be removed and her own adult stem cells injected in the area of her original wound.
The Laura Dominguez story is not complete. The next chapter may or may not yield the results she seeksbut no one can deny the determination and courage of Laura. For her part, she has one goal in mind: I will walk again.
We shall update this site and keep you informed on her progress.
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Stem Cell Research Facts - Adult Stem Cell Success
Scientists Hope to Use Stem Cells to Reverse Death in … – Futurism – Futurism
By raymumme
In BriefBioquark is about to begin a trial that will attempt to bringbrain-dead patients back to life using stem cells. However, thetrial is raising numerous scientific and ethical questions forother experts in the field. Back From The Dead
Researchers seem to be setting their sights on increasinglylofty goals when it comes to the human body from the worlds first human head transplant, to fighting aging, and now reversing death altogether. Yes, you read that right. A company called Bioquarkhopes to bring people who have been declared clinically brain-dead back to life. The Philadelphia-based biotech company is expected to start on the project later this year.
This trial was originally intended to go forward in 2016 in India, but regulators shut it down. Assuming this plan will be substantially similar, it will enroll 20 patients who will undergo various treatments. The stem cell injection will come first, with the stem cells isolated from that patients own blood or fat. Next, the protein blend gets injected directly into the spinal cord, which is intended to foster growth of new neurons. The laser therapy and nerve stimulation follow for 15 days, with the aim of prompting the neurons to make connections. Meanwhile, the researchers will monitor both behavior and EEGs for any signs of the treatment causing any changes.
While there is some basis in science for each step in the process, the entire regimen is under major scrutiny. The electrical stimulation of the median nerve has been tested, but most evidence exists in the form of case studies. Dr. Ed Cooper has described dozens of these cases, and indicates that the technique can have some limited success in some patients in comas. However, comas and brain death are very different, and Bioquarks process raises more questions for most researchers than it answers.
One issue researchers are raising about this study is informed consent. How can participants in the trial consent, and how should researchers complete their trial paperwork given that the participants are legally dead and how can brain death be conclusively confirmed, anyway? What would happen if any brain activity did return, and what would the patients mental state be? Could anything beyond extreme brain damage even be possible?
As reported by Stat News, In 2016, neurologist Dr. Ariane Lewis and bioethicist Arthur Caplan wrote in Critical Care that the trial is dubious, has no scientific foundation, and suffers from an at best, ethically questionable, and at worst, outright unethical nature. According to Stat News, despite his earlier work with electrical stimulation of the median nerve, Dr. Cooper also doubts Bioquarks method, and feels there is no way this technique could work on someone who is brain-dead. The technique, he said, relies on there being a functional brain stem one of the structures that most motor neurons go through before connecting with the cortex proper. If theres no functional brain stem, then it cant work.
Pediatric surgeon Charles Cox, who is not involved in Bioquarks work, agrees with Cooper, commenting to Stat News on Bioquarks full protocol, its not the absolute craziest thing Ive ever heard, but I think the probability of that working is next to zero. I think [someone reviving] would technically be a miracle.
Pastor remains optimistic about Bioquarks protocol. I give us a pretty good chance, he said. I just think its a matter of putting it all together and getting the right people and the right minds on it.
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Scientists Hope to Use Stem Cells to Reverse Death in ... - Futurism - Futurism
Exciting Times For Spinal Cord Injury Clinical Trials …
By LizaAVILA
These are indeed exciting times for spinal cord injury (SCI) clinical trials. There are trials ongoing around the world targeting different repair strategies. In this article we want to take the opportunity to explain some of the high profile clinical trials ongoing in the United States utilizing cells as a therapeutic intervention.
Miami Project Schwann Cells
As many of our readers know, The Miami Projects 1st Phase I clinical trial testing Schwann cells began in November 2012 and we are happy to announce that the final participant was transplanted in August 2015. Schwann cells come from your own body and they are a type of cell found throughout the entire peripheral nervous system (PNS). The PNS includes all nerves going out to muscles as well as sensory nerves coming from the muscles back to the spinal cord. Schwann cells are a type of support cell in the PNS and some important points about Schwann cells are that they 1) insulate (myelinate) individual nerve fibers (axons), which is necessary for sending appropriate electrical signals throughout the nervous system, 2) are not stem cells, they are adult cells and can only be Schwann cells, and 3) can be obtained from each persons own body thereby eliminating the need for immunosuppression medicine.
This trial is specifically targeting people with new SCI, less than 30 days after injury, having sustained a trauma-induced lesion between thoracic levels T3-T11 and whom were neurologically complete. This is a dose escalation treatment trial, meaning that we will test 3 different doses: 5 million, 10 million, and 15 million Schwann cells. There were a total of 39 people screened for eligibility, 9 were enrolled, and 6 participants were transplanted. The first two participants received the 5 million cell dose, the second two received the 10 million cell dose, and the final two received the 15 million cell dose. Thus far, there have been no treatment-related adverse effects in any of the transplanted subjects, which is excellent news. Remember, safety is the determinate of success for this phase I trial. We are not releasing any other information about the participants or results because the trial is still ongoing and we cannot compromise the data. After the final participant is 12 months post-transplant we will prepare the results for publication in a peer-reviewed scientific journal.
Our 2nd Phase I clinical trial began in February 2015 for chronic SCI and will also be primarily focused on safety, but in addition it will involve a preliminary evaluation of the efficacy of combining Schwann cells with exercise and rehabilitation. For humans with chronic SCI, we hypothesize that axons might show improved function if myelin repair is induced with the implantation of autologous Schwann cells. In addition, spinal cord cavitation may be reduced and neural sprouting and plasticity may be enhanced via neurotrophic effects. In this trial, participants will receive three months of fitness conditioning and locomotor rehabilitation prior to transplantation in order to validate the stability of their neurological baseline as well as to enhance their fitness level thereby reducing any deconditioning effects. They will also receive fitness conditioning and rehabilitation for six months post-transplantation to maintain health and promote neuronal activity and potential neuroplasticity. We believe that this combination of cell therapy with intense rehabilitation prior to and following cell transplantation will enhance our chances of seeing improved recovery in the chronic setting https://clinicaltrials.gov/ct2/show/NCT02354625 .
StemCells Inc
Drs. Allan Levi and Kim Anderson, along with several other University of Miami faculty members, are also participating in a clinical trial testing a different cell therapy neural stem cells. That trial, referred to as the Pathway Study, is sponsored by a company called StemCells, Inc.
The Pathway Study is testing the safety and potential benefit of a very specific stem cell type known as a neural stem cell; these are not Schwann cells. The neural stem cells being used in the Pathway Study were derived from fetal brain tissue and have the ability to self-renew and become the main types of mature cells found both in the brain and spinal cord. These cells do not come from your own body, therefore anyone who receives them into their body has to be on immunosuppression medicine. Studies of SCI in animals have shown that these human neural stem cells can survive and lead to recovery of function through remyelination and possibly neuronal cell replacement.
Prior to the Pathway Study, the company conducted a Phase I/II safety & preliminary efficacy clinical trial in humans with thoracic SCI. Twelve participants were transplanted within 3 to 12 months of injury. The results they have disclosed at scientific meetings indicate that neural stem cell transplantation appears to be safe; several participants have regained some sensation.
The Pathway Study is a larger Phase II efficacy clinical trial designed to determine if neural stem cells can help people with cervical SCI recover spinal cord function and gain strength and sensation. They will enroll up to 52 participants. Individuals may be able to join the study if they are 18 to 60 years old, have a cervical SCI that is classified as ASIA Impairment Scale grade A, B, or C, are less than two years post-injury, and are generally in good health. Individuals that are eligible for the study will participate for approximately 12 months. There are several sites around the country that are enrolling https://clinicaltrials.gov/ct2/show/NCT02163876 .
Asterias Biotherapeutics
Many of you have probably heard of the Geron clinical trial that was prematurely halted a few years ago for financial reasons. In 2013, a new company called Asterias Biotherapeutics took over the rights for everything related to the prior trial. The first trial was a Phase I safety trial using a human embryonic stem cell line pre-differentiated into oligodendrocyte progenitor cells. The oligodendrocyte progenitor cells are targeting reduction of the size of the injury cavity as well as remyelination of demyelinated axons to restore conduction. These cells also cannot be obtained from your own body, hence require immunosuppression medicine as well when administered to anyone. In that trial, 5 individuals with complete thoracic injury received the cells within 14 days after their injury. The results they have disclosed at scientific meetings indicate that the cell transplantation appears to be safe and that four of the five participants appear to have a smaller cavity when evaluated by MRI.
In 2015, they began a Phase I/IIa dose escalation trial, the SCI-Star study. This trial is enrolling individuals with cervical injury between levels C5-C7 whom are neurologically complete. The cells have to be injected between 14 to 30 days post-injury; up to 13 participants will receive the cells. There are at least 3 centers enrolling https://clinicaltrials.gov/ct2/show/NCT02302157 .
Neuralstem
The final cell therapy of high profile is being conducted by a company called Neuralstem. This is a Phase I safety trial using human fetal spinal cord neural precursor cells. These stem cells are targeting growth factor replacement and possibly neuronal cell replacement. Again, because these cells do not come from ones own body, they require immunosuppression medicine. The company previously completed a Phase I safety trial using the same cells in individuals with Lou Gehrigs disease. They transplanted 18 participants in mid- to late stages of the disease and demonstrated safety. The company then obtained approval for the SCI Phase I trial. A total of 4 participants with complete thoracic injury, between one and two years post-injury, will be transplanted. The study procedures are all performed in California https://clinicaltrials.gov/ct2/show/NCT01772810 .
To find out more information about the trials being conducted at The Miami Project, contact The Miami Project Education Department at 305-243-7108 or MPinfo@med.miami.edu . More information about all of our clinical trials and studies is available at http://www.themiamiproject.org/trials .
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Exciting Times For Spinal Cord Injury Clinical Trials ...
Looking at the role of the protein TDP-43 in ALS – Brandeis University
By Dr. Matthew Watson
A postdoctoral fellow will examine the protein's effects in human cells.
By Lawrence GoodmanJune 1, 2017
In ALS, also known as Lou Gehrigs disease, the bodys motor neurons degenerate and eventually die. As a result, muscles waste away, leading to an inability tospeak, move and, eventually, breathe. Patients typically die within five years of symptom onset.
One possible target for a drug treatment for ALS is the protein TDP-43. Mutations in the gene encoding TDP-43 cause some cases of inherited ALS and almost all sufferers of sporadic ALS to develop clumps of TDP-43 protein intheir neurons.
In recent years, postdoctoral fellow Mugdha Deshpande has been working withassociate professors of biology Avital Rodal and Suzanne Paradis to uncover how the TDP-43 protein damages neurons in model organisms such as the fruit fly Drosophila melanogaster. Now, they want to take the next step and see whether the same effects occur in human cells.
Deshpande is the Blazeman Postdoctoral Fellow for ALS Research, a position funded by the Rhode Island-based Blazeman Foundation for ALS. Based on her discoveries of how TDP-43 affects neurons in model organisms, she recently received a Brandeis Provost Research award to further her research on TDP-43 in human cells.
Deshpandes research focuses on motor neurons, whose nuclei are located in thespinal cord and whose nerve fibers, or axons, stretch throughout the body. In flies, defective TDP-43 has been shown to cause damage in the area where axonsconnect to muscles.
To test whether the same defects occur in humans, Deshpande will utilize a line of induced pluripotent stem cells isolated from an ALS patients skin cells and developed at the University of Massachusetts Medical School. In collaboration with the Human Neuron Core at Boston Children's Hospital, she will transform the stem cells into neurons.
Deshpande plans to study the defects that arise when human neurons develop whileharboring a genetic mutation in the TDP-43 gene. We need to gain an understanding of whats going on, she says. Without that, we are not going to get a therapy for ALS.
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Looking at the role of the protein TDP-43 in ALS - Brandeis University
Resurrected: A controversial trial to bring the dead back to life plans a restart – STAT
By Sykes24Tracey
F
or any given medical problem, it seems, theres a research team trying to use stem cells to find a solution. In clinical trials to treat everything from diabetes to macular degeneration to ALS, researchers are injecting the cells in efforts to curepatients.
But in one study expectedto launch later this year, scientists hope to use stem cells in a new, highly controversial way to reverse death.
The idea ofthe trial, run by Philadelphia-based Bioquark, isto inject stem cells into the spinal cords of people who have been declared clinically brain-dead. The subjects will also receive an injected protein blend, electrical nerve stimulation, and laser therapy directed at the brain.
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The ultimate goal: to grow new neurons and spur them to connect to each other, and thereby bring the brain back to life.
Its our contention that theres no single magic bullet for this, so to start with a single magic bullet makes no sense. Hence why we have to take a different approach, said Ira Pastor, CEO of Bioquark.
A dogged quest to fix broken spinal cords pays off with new hope for the paralyzed
But the scientific literature scarce as it is seems to show that even several magic bullets are unlikely to accomplish what Bioquark hopes itwill.
This isnt the first start for the trial. The study launched in Rudrapur, India, in April 2016 but it never enrolled any patients. Regulators shut the study down in November2016 because, according to Science, IndiasDrug Controller General hadnt cleared it.
Now, Pastor said, the company is in the final stages of finding a new location to host trials. The company willannounce a trial in Latin America in coming months, Pastor told STAT.
If that trial mirrors the protocol for the halted Indian one, itll aim to enroll 20 patients wholl receive a barrage of treatments. First theres the injection of stem cells isolatedfrom the individuals own fat or blood. Second, theres a peptide formula injected into the spinal cord, purported to help nurture new neurons growth. (The company has tested the same concoction, called BQ-A, in animalmodels of melanoma, traumatic brain injuries, and skin wrinkling.) Third, theres a regimen of nerve stimulation and laser therapyover 15 days to spur the neurons to form connections. Researcherswilllook to behavior and EEG for signs that the treatment is working.
But the process is fraught with questions. How do researchers complete trial paperwork when the person participating is, legally, dead? (In the United States, state laws most often define death as the irreversible loss of heart and lung or brain function.) If the person did regain brain activity, what kind of functional abilities would he or she have? Are families getting their hopes up for an incredibly long-shot cure?
Answers to most of those questions are still far off. Of course, many folks are asking the what comes next? question, Pastor acknowledged. While full recovery in such patients is indeed a long term vision of ours, and a possibility that we foresee with continued work along this path, it is not the core focus or primary endpoint of this first protocol.
No real template exists to know whether this approach might work and its gotten some prominent backlash. Neurologist Dr. Ariane Lewis and bioethicist Arthur Caplan wrote in a 2016 editorial that the trial borders on quackery, has no scientific foundation, and gave families a cruel, false hope for recovery. (Exploratory research programs of this nature are not false hope. They are a glimmer of hope, Pastor responded.)
The company hasnt tested the full, four-pronged treatment, even in animal models. Studies have evaluated the treatments singly for other conditions stroke, coma but brain death is a quite different proposition.
Stem cell injections to the brain or spinal cord have shown some positive results for children with brain injuries; trials using similar procedures to treat cerebral palsy and ALS have also been completed. One small, uncontrolled studyof 21 stroke patients found that they recoveredmore mobility after they received an injection of donor stem cells into their brains.
On transcranial laserdevices, the evidence is mixed. The approach has been shown to stimulate neuron growth in some animal studies. However, a high-profile Phase 3 study of one such device in humans was halted in 2014 after it showed no effect on 600 patients physical capabilities as they recovered from a stroke. Othertrialsto revive people from comasusing laser therapy are underway.
The literature around electrical stimulation of the median nerve whichbranches from the spinal cord downthe arm and to the fingers primarily consists of case studies.Dr. EdCooper wrote some of those papers, one of which described dozens of patients treated in his home state of North Carolina, including 12 who had a Glasgow Coma Score of 4 an extremely low score on the scale. With time (and with the nerve stimulation), four of those 12people made a good recovery, the paper described; others were left with minor or major disabilities after their coma.
Mini-me brains-in-a-dish mimic disease, raise hope for eventual therapies
But Cooper, an orthopedic surgeon by training who worked with neurosurgeons on the paper, said unequivocally that there is no way this technique could work on someone who is brain-dead. The technique, he said, relies on there being a functional brain stem one of the structuresthat mostmotor neurons go through before connecting with the cortex proper. If theres no functional brain stem, then it cant work.
Pastor agreed but heclaimed the technique would work because there are a small nestofcells that still function in patients who are brain-dead.
Complicating such trials, there is noclear-cut confirmatory test for brain death meaning a recovery in the trial might not be entirely due to the treatment. Some poisons and drugs, for instance, can make people look brain-dead.Bioquark plans to rely on local physicians in the trials host country to make the declaration. Were not doing the confirmatory work ourselves, Pastor said, but each participant would have undergone a battery of tests considered appropriate by local authorities.
But asurvey of 38 papers published over 13 years found that, if the American Academy of Neurology guidelines for brain death had been met, no brain-dead people have ever regained brain function.
Of Bioquarks full protocol, its not the absolute craziest thing Ive ever heard, but I think the probability of that working is next to zero, said Dr. Charles Cox, a pediatric surgeon who has doneresearch with mesenchymal stem cells the type used in the trial at the University of Texas Health Science Center at Houston. Cox is not involved in Bioquarks work.
Some studies have found that cells from a part of thebrain called the subventricular zone can grow in culture even after a person is declared dead, Cox said. However, its unlikely that the trials intended outcome to havea stem cell treatment result in new neurons or connections would actually happen. Neurons would likely struggle tosurvive, because blood flow to the brain isalmost always lostin people whohave been declared brain-dead, Cox said.
But Pastor thinksBioquarks protocol will work. I give us a pretty good chance, he said. I just think its a matter of putting it all together and getting the right people and the right minds on it.
Cox is less optimistic. I think [someone reviving] would technically be a miracle, he said. I think the pope would technically call that a miracle.
Kate Sheridan can be reached at kate.sheridan@statnews.com Follow Kate on Twitter @sheridan_kate
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Resurrected: A controversial trial to bring the dead back to life plans a restart - STAT
World Multiple Sclerosis Day 2017: Cellular Therapy helps induce long-term remission of Multiple Sclerosis – TheHealthSite
By JoanneRUSSELL25
Genetically and immune-mediated disease, Multiple Sclerosis symptoms and progression are unpredictable at the time of diagnosis.
Multiple Sclerosis is an inflammatory disease that affects the brain and spinal cord of an individual. It occurs due to the combination of genetic susceptibility and can also occur due to low vitamin levels, virus, and environmental factors. The Multiple Sclerosis Foundation estimates that more than 400,000 people in the United States and about 2.5 million people around the world have Multiple Sclerosis. No large epidemiological studies have been reported from India but calculations based on hospital data in the 1970s suggested an approximate prevalence rate of only 0.17 to 1.33 per 100,000 in different parts of India. Increased awareness and the rise in the number of neurologists and availability of MRI has led the current estimates to about 7 to 10 per 100,000. As there are many Indians who still do not have access to adequate medical facilities especially in the rural sector, there can be a rise in the figures mentioned too. As per hospital-based studies within India, an increase in the incidence of Multiple Sclerosis from 1.58% to 2.54% has been noted in the last decade.
This immune-mediated disease affects the protective covering (myelin sheath) around the nerves which result in neurological defects. With the help cellular therapy, utilising the various properties of stem cells, Multiple Sclerosis can be treated. In autologous cell-based therapy, stem cells from the patients own body are transplanted, which resets the immune system. A patient suffering from Multiple Sclerosis is often treated with immune-suppressive drugs and monoclonal antibodies. But, these agents are associated with side effects with long-term use and are not entirely effective in managing symptoms. Autologous stem cells are neuroprotective and also have other paracrine properties by which patients of Multiple Sclerosis can benefit. The immunomodulatory properties of the stem cells help reduce damage in the central nervous system of patients with Multiple Sclerosis. It also helps in regeneration of the injured nerves, said Dr Pradeep Mahajan, Regenerative Medicine researcher at StemRx Bioscience Solutions Pvt. Ltd.
The time taken to heal varies from patient to patient and can go from 2 months to 1 year. There are various ways to administer the stem cells back into the body, the route depending on the condition and requirement of the patient. In neurological conditions, the appropriate route of administration would be the one which facilitates cell delivery into the brain.
Published: May 30, 2017 4:09 pm | Updated:May 30, 2017 4:10 pm
Disclaimer: TheHealthSite.com does not guarantee any specific results as a result of the procedures mentioned here and the results may vary from person to person. The topics in these pages including text, graphics, videos and other material contained on this website are for informational purposes only and not to be substituted for professional medical advice.
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World Multiple Sclerosis Day 2017: Cellular Therapy helps induce long-term remission of Multiple Sclerosis - TheHealthSite
Neurosurgeon Dr. Jay Jagannathan discussed the future of spine surgery, motion-sparing techniques and minimally … – PR Newswire (press release)
By Sykes24Tracey
TROY, Mich., May 30, 2017 /PRNewswire/ --Jay Jagannathan, M.D., known as one of the United States' top neurosurgeons, was featured on WJR AM-760 radio show Anything is Possible! hosted by Jack Krasula on May 27, 2017. During the one-hour show, Dr. Jagannathan discussed the importance of patient-centric care in spine surgery. "It is important that patients know the full spectrum of surgical and non-surgical options," he said, adding that "a full understanding of their options puts patients in a position to make the best decision for themselves."
When asked by Jack Krasula show about the future of spine surgery, Dr. Jagannathan specifically pointed to motion-sparing techniques. Motion-sparing techniques aim to preserve motion in the spine, and are increasingly relevant given the recent FDA approval of 2-level cervical artificial disks. "The idea of preserving motion will permit many patients who were not previously candidates for spine surgery to have procedures that can help with pain while still maintaining normal spinal motion and hopefully reducing the future need for re-operation," he said. Dr. Jagannathan also discussed the importance of stem cells, which are undifferentiated, primitive cells that have the capability of maturing into specific tissue types. According to Dr. Jagannathan, "stem cells not only have the ability to possibly enhance spinal fusion outcomes, but also to serve as a vector to induce healing following spinal cord injury or stroke." Dr. Jagannathan also pointed to the advances in imaging modalities and technology, which has allowed surgeons to provide minimally invasive treatment for pathology which previously were untreatable. "What MIS has taught us is that using image-guided targeting while decreasing tissue manipulation can greatly reduce post-operative pain, hospital stays and post-operative drug use."
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Neurosurgeon Dr. Jay Jagannathan discussed the future of spine surgery, motion-sparing techniques and minimally ... - PR Newswire (press release)