What if one day, we could teach our bodies to self-heal like a lizards tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimers or Parkinsons disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institutes Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

One of the things that that attracted me most to the ASU and the Biodesign CMDB was Haos vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology, Stephanopoulos said.

I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice.

Yans research center uses DNA and other basic building blocks to build their nanotechnology structures only at a scale 1,000 times smaller than the width of a human hair.

Theyve already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physics and the creative imaginations of these budding nano-architects.

Learning from nature

The goal of the Center for Molecular Design and Biomimetics is to usenatures design rulesas an inspiration in advancing biomedical, energy and electronics innovation throughself-assembling moleculesto create intelligent materials for better component control and for synthesis intohigher-order systems, said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeleys Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

Weve learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems, Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity, Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to grab the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal off. To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the materials surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinsons disease.

The patients own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the lab and then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue, Stupp said. In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that.

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat, Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos research received a boost with funding from the U.S. Air Forces Young Investigator Research Program (YIP).

The Air Force Office of Scientific ResearchYIP award will facilitate Nicks research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers, Yan said.

Theyll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research teams efforts one step closer to the clinic.

ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications, Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Stephanopoulos recent research was carried out at Stupps Northwesterns Simpson Querrey Institute for BioNanotechnology. The National Institute of Dental and Craniofacial Research of the National Institutes of Health (grant 5R01DE015920) provided funding for biological experiments, and the U.S. Department of Energy, Office of Science, Basic Energy Sciences provided funding for the development of the new materials (grants DE-FG01-00ER45810 and DE-SC0000989 supporting an Energy Frontiers Research Center on Bio-Inspired Energy Science (CBES)).

The paper is titled Instructing cells with programmable peptide DNA hybrids. Samuel I. Stupp is the senior author of the paper, and post-doctoral fellows Ronit Freeman and Nicholas Stephanopoulos are primary authors.

See original here:
Bio-inspired Materials Give Boost to Regenerative Medicine - Bioscience Technology

Spinal Cord Stem Cells Comments Off on Bio-inspired Materials Give Boost to Regenerative Medicine – Bioscience Technology | August 22nd, 2017

Twelve years ago, Robb Martin was an active police officer with Prescott Police Department when a recreational accident left him paralyzed from the chest down.

I was on a four-wheeler in the sand dunes, Martin, 42, said. I was on my way back to camp just putting along when I hit a bump. It threw me off the front, my helmet got stuck in the sand, my legs just kept going and I broke my back right at the chest level.

After getting out of the hospital and going through some rehabilitation to get his arms, shoulders and neck moving normally, he continued to work for the police department in the dispatch center and has been there ever since.

Despite his condition, Martin has remained incredibly active.

The guy is always busy, said Tom Newell, a longtime friend of Martins.

With some help from his friends, he managed to build a workshop on his property and is consistently in there modifying objects to fit his needs or assisting friends and family with various projects.

If hes not helping his wife with her business, hes in his shop welding something, making something or building something to help somebody else out, Newell said.

Since the accident, Martin has looked for ways to improve his mobility. Physical therapy has been helping, allowing him to regain back and stomach muscles in recent years.

I can do pushups and actually support my waist, which is amazing, he said.

His goal, however, is to once again be on his feet.

Just to even stand up and grab something out of a cabinet would be phenomenal, Martin said.

That dream might come true if he can raise the funds to have a recently developed procedure done in Thailand by a company called Unique Access.

The procedure, referred to as epidural stimulation, involves surgically implanting a device along a damaged portion of the nervous system, according to the companys website. The device then applies a continuous electrical current.

It acts kind of like a jumper cable, for lack of a better term, Martin said. It just connects above the affected area and allows the brain to reconnect with the spinal cord under the affected area.

In combination with the implant, several million stem cells are injected into the area to help the regenerative process. These, as well as

an assisted rehabilitation process, take about 40 days to complete.

The procedure has yet to be seriously implemented in the U.S., Martin said, because of how new it is to the medical industry. So far, however, he hasnt heard of any unusual risks associated with the procedure and has spoken with two individuals who successfully went through it.

One guy is walking up to 30 meters unassisted, Martin said. Another guy, the day after surgery, he was standing up by himself in a pool.

Altogether, Martin said its going to cost him $100,000 out of pocket.

Not able to afford that between him and his wife, hes turned to the community for help. Friends and family have already been busy contributing and organizing events.

Just last Saturday, Aug. 12, about $5,000 was raised on his behalf from two fundraising events hosted by his friends Tony and Liko Harwood.

Tony wanted to be involved and couldnt just sit still and not make any money for Rob so here we are, Liko said Saturday during one of the events.

Another $2,000 was raised from a donation bucket placed inside Scouts Gourmet Grub in Prescott.

Quite a bit more was also raised by fundraisers hosted by the Northern Arizona Regional Training Academy (NARTA), the local police academy.

Sitting at about $15,000, Martin is hoping to continue raising money in whatever way he can to reach the full $100,000.

My surgery is approved, theyre just waiting for me to set up a date, Martin said. The funding is really all Im waiting on.

For more information about Martins story and to donate, go to RobbMartin.com.

Read more:
Disabled former police officer raising money for operation in Thailand - The Daily Courier

Spinal Cord Stem Cells Comments Off on Disabled former police officer raising money for operation in Thailand – The Daily Courier | August 19th, 2017

A pregnant British mom hopes she and her unborn baby will be the answer to help prolong her ailing brothers life.

Georgina Russell, of Preston, England, said she was desperate to help her brother, Ashley, when doctors diagnosed him with a slow-growing but deadly brain tumor earlier this year, according to the Daily Mail.

Georgina said she began researching his condition, glioblastoma, online and looking for answers that could save his life. She found one: her pregnancy.

Stem cells produced in the umbilical cord between her and her unborn baby potentially could be used in a treatment to shrink Ashleys tumor, according to the report. Once Georgina gives birth, she said doctors will be able to harvest and store the stem cells until Ashley needs them.

There is no harm to the baby or the mother when doctors harvest stem cells from the umbilical cord unlike embryonic stem cells, which only can be taken by killing a human life in the embryonic stage.

Georgina told the Mail: The blood from the cord is being used in trials across the world. It can do amazing things to help the body repair itself. If we store the stem cells, they can be kept to be used throughout Ashleys treatment when he needs them.

They might be able to inject them into the spinal fluid, to shrink the tumour on the brain, or they may be able to use the tissue grown from them to repair any damage to other parts of his body, if he has to have chemotherapy or radiotherapy.

Ashley Russell, a British military veteran, husband and father, said doctors found the tumor after he began suffering from headaches, dizzy spells and mini-seizures about six months ago. Later, he said he also began having blurred vision. Doctors ran a series of tests before discovering the tumor on his brain.

He said doctors suggested surgery, but the procedure has high risks. They gave him about five years to live, according to the report.

Georgina said she was devastated for her brother and his family, and she began researching ways to help him. In her research online, she said she discovered how stem cells collected from the umbilical cord are helping to treat people with tumors and other diseases.

PRO-LIFE COLLEGE STUDENT? LifeNews is looking for interns interested in writing, social media, or video creation. Contact us today.

Her brother said the idea seemed odd at first, but he is willing to try anything.

I am quite a positive person so although the diagnosis was difficult, I am determined to do whatever I can to keep going, Ashley said. I did think about not being around to see my little girl get married and knew that if there was anything that might help, I would give it a go.

Georgina currently is 33 weeks pregnant with her unborn child, the report states.

Stem cells are so powerful and his new niece or nephew could save his life, she said.

The family set up a JustGiving page to help pay for the storage of the stem cells and Ashleys treatment.

Adult stem cells and those from umbilical cords are proving to be live-saving, while life-destroying embryonic stem cells have not been effective.

David Prentice, vice president and research director for the Charlotte Lozier Institute, explained more about the effectiveness of these life-saving stem cells in 2014:

SUPPORT PRO-LIFE NEWS! Please help LifeNews.com with a donation

Umbilical cord blood stem cells have become an extremely valuable alternative to bone marrow adult stem cell transplants, ever since cord blood stem cells were first used for patients over 25 years ago. The first umbilical cord blood stem cell transplant was performed in October 1988, for a 5-year-old child with Fanconi anemia, a serious condition where the bone marrow fails to make blood cells. That patient is currently alive and healthy, 25 years after the cord blood stem cell transplant.

Prentice said more than 30,000 cord blood stem cell transplants have been done across the world. These stem cells have helped treat people with blood and bone marrow diseases, leukemia and genetic enzyme diseases, he said.

Read more from the original source:
Woman Will Use Stem Cells From Her Baby's Umbilical Cord To Save Her Brother, Who Has a Brain Tumor - LifeNews.com

Spinal Cord Stem Cells Comments Off on Woman Will Use Stem Cells From Her Baby’s Umbilical Cord To Save Her Brother, Who Has a Brain Tumor – LifeNews.com | August 19th, 2017

In a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways. Credit: Northwestern University

What if one day, we could teach our bodies to self-heal like a lizard's tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimer's or Parkinson's disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institute's Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

"One of the things that that attracted me most to the ASU and the Biodesign CMDB was Hao's vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology," Stephanopoulos said.

"I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice."

Yan's research center uses DNA and other basic building blocks to build their nanotechnology structuresonly at a scale 1,000 times smaller than the width of a human hair.

They've already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physicsand the creative imaginations of these budding nano-architects.

Learning from nature

"The goal of the Center for Molecular Design and Biomimetics is to use nature's design rules as an inspiration in advancing biomedical, energy and electronics innovation through self-assembling molecules to create intelligent materials for better component control and for synthesis into higher-order systems," said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeley's Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

"We've learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems," Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos' tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

"DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity," Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new study in Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to "grab" the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal "off." To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the material's surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinson's disease.

The patient's own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the laband then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

"People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue," Stupp said. "In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that."

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

"Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat," Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos' research received a boost with funding from the U.S. Air Force's Young Investigator Research Program (YIP).

"The Air Force Office of Scientific Research YIP award will facilitate Nick's research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers," Yan said.

They'll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research team's efforts one step closer to the clinic.

"ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications," Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Explore further: New technology to manipulate cells could help treat Parkinson's, arthritis, other diseases

More information: Ronit Freeman et al. Instructing cells with programmable peptide DNA hybrids, Nature Communications (2017). DOI: 10.1038/ncomms15982

More:
Bio-inspired materials give boost to regenerative medicine - Medical Xpress

Spinal Cord Stem Cells Comments Off on Bio-inspired materials give boost to regenerative medicine – Medical Xpress | August 19th, 2017

18 Aug 2017

Astrocytes are more than bystanders in neurotransmissionthey take an active role in synaptic activity. However, their functions are hard to study because the cells are difficult to grow in vitro and its hard to coax them to mature from progenitors. Now, researchers from the labs of Sergiu Paca and Ben Barres, both at Stanford University School of Medicine, California, report that astrocytes come of age in spherical balls of human brain cells cultured in a dish for almost two years. As reported in the August 16 Neuron, these astrocytes develop much like those from real brains, undergoing similar transcriptomic, morphologic, and functional changes. Studying the processes involved in this astrocyte maturation will help researchers understand neurodevelopmental disorders such as autism and schizophrenia, researchers say, and might even shed light on problems in adultbrains.

That these 3D cultures can be maintained for such a long time allows us to capture an interesting transition in astrocytes, said Paca. We are starting to appreciate aspects of human brain development to which we would not otherwise haveaccess.

The breakthrough is that they can develop human astrocytes very close to maturity in their 3D culture models, said Doo Yeon Kim, Massachusetts General Hospital, Charlestown, who uses 3D culture models to study pathological process that occur in Alzheimers disease. Some researchers are using 3D cultures to model other neurodegenerative disorders, such as ALS, and still others are planning to use cultured astrocytes for cell therapy. If astrocytes are not mature enough in culture, patterns [we see] may not be the same as in the diseased brain, saidKim.

This developing human astrocyte (red), which comes from a 350-day-old cortical spheroid, is taking shape as a mature cell. [Image courtesy of Sloan et al.Neuron]

A few years back, Pacas group developed a method for differentiating human induced pluripotent stem cells (hiPSCs) into a 3D culture of brain cells. They used special dishes that the cells could not easily attach to, coaxing them to stick to each other instead. Under these conditions the iPSCs balled up into neural spheroids that grew to about 4 mm in diameter. A cocktail of growth factors early on encouraged them to form excitatory pyramidal cells like those in the cortex, and the cells spontaneously organized into layers. These cortical spheroids survived a year or more and spontaneously grew astrocytes in addition to neurons (Paca et al., 2015). Not long after, the Barres lab reported that astrocytes in the adult human brain look different from those isolated from fetuses. They called the latter astrocyte progenitor cells (APCs). Each had their own transcriptional patterns and functions (Jan 2016 news). Together, Barres and Paca wondered if it was possible to see the APCs morph into mature astrocytes in these long-lived corticalspheroids.

To find out, first author Steven Sloan and colleagues examined spheroids generated from iPSCs derived from healthy human fibroblasts. Sloan grew the spheroids for about 20 months. Along the way, he took samples, isolated the astrocytes, and compared them to those isolated from fetal and postnatal humanbrain.

At about 100 days in culture, astrocytes began to sprout spontaneously from within the mostly neuronal milieu of the cortical spheroids. At first, these cells were simple, adorned by few branches and expressing genes akin to those active in APCs. But as the spheroids reached about 250 days, the astrocytes therein looked more mature, having numerous processes. After this point, APC gene expression tapered off and the astrocytes started producing proteins typical of matureastrocytes.

Astrocytes also underwent functional changes as they matured. Early versions divided in fast and furious fashion, much like their counterparts from the fetal tissue. That division slowed as the spheroids aged. Dividing APCs dropped from 35 percent of all astrocytes at day 167 to 3 percent at day 590. Taken from the spheroids at day 150 and cultured in a 2D layer, immature astrocytes also harbored a voracious appetite for added synaptosomes, much like immature astrocytes recently characterized in mice (see image below; Dec 2013 conference news on Chung et al., 2013). However, that hunger waned as astrocytes approached the 590-daymark.

At the older end of the spectrum, mature astrocytes seemed to take on a supportive role, strengthening calcium signaling in nearbyneurons.

Studying the neurons and astrocytes in these cortical spheroids could be useful for addressing certain unanswered questions about human biology, said other researchers. This could be a very strong opportunity to understand what goes wrong in human genetic disorders that affect astrocyte function, said M. Kerry OBanion, University of Rochester Medical Center, New York. Its also possible that such cultures could reveal as yet unknown facets of familial mutations that cause Alzheimers disease, he suggested. However, given that these cultures take a long time to grow and develop, they are unlikely to completely supplant other types of cultures or faster-maturing animal models, hesaid.

Kim agreed, saying, The results are very exciting, but not practical yet for disease modeling." However, Kim hopes that researchers will make progress on accelerating the maturationprocess.

The Barres and Paca labs are trying just that with the spheroid. They will also analyze what they secrete to support neuronal signaling. In addition, they are exploring how to make the astrocytes reactive, as they often are in neurodegenerative diseases, such as Alzheimers. Doing so might reveal how such astrocytes interact withneurons.

An immature astrocyte taken from a 150-day-old spheroid gobbles up added synaptosomes (red). [Neuron, Sloan et al.2017]

To Pacas knowledge, these cortical spheroids are some of the longest human cell cultures ever reported. His group has continued to cultivate these clumps, with the oldest still going strong at day 850. Granted, these systems are missing many cell types: endothelial cells, oligodendrocytes, and microglia to name a few, he said. However, his lab has introduced new ways to add in other cells. Earlier this year, he reported 3D cultures of cortical glutamatergic neurons and GABAergic interneurons that fused together when they were placed side-by-side (Birey et al., 2017).

Clive Svendsen, Cedars-Sinai Medical Center in Los Angeles, California, saw clinical implications for this paper. It shows iPSC derived astrocytes can mature to an adult phenotype, he said. This further supports their use in clinical transplantation, as we are planning to do. His group has begun a Phase 1 clinical trial that implants human fetal astrocytes into the spinal cords of ALS patients.Gwyneth DickeyZakaib

Read the original here:
Brain Spheroids Hatch Mature Astrocytes - Alzforum

Spinal Cord Stem Cells Comments Off on Brain Spheroids Hatch Mature Astrocytes – Alzforum | August 19th, 2017

JENNIE McKEON @JennieMnwfdn

NICEVILLE Jack Massey is ready to go back to school.

Only this time, the University of Florida senior will head back to campus with his mom and a new outlook on life.

Massey suffered a spinal cord injury in a pool accident in March and is paralyzed from the chest down. After months of rehab, he's eager to get back into a familiar routine.

"It's definitely boring," the 21-year-old said at his parents' home in Niceville. "There's not a lot to do. I want to go back to school. I still have my brain. I still have everything I need to be successful."

After the accident March 17, Massey was treated at the University of Florida Shands Hospital and then was transferred to Shepherd Center, a spinal cord and brain injury rehab center in Atlanta. At Shepherd Center he met with a peer mentor, counselors and physical therapists to help him find a new normal.

Jack has remained positive throughout the past six months.

"Jack has been a fighter through all of this," said his mother, Julie. "I think he's done well. I only saw him break down once."

Before the accident, Jack was a well-rounded athlete who playing baseball and basketball and ran. He was a star on the track and field team at Niceville High School, with his 4 X 800 relay winning state his senior year.

He says the biggest challenge now is not being able to do the same things he could before.

"I can't get up and go," he said. "It didn't really start to set in until after I got out of rehab."

Jack has had to find enjoyment in other things, like reading or playing with the dogs. His friends have learned to transfer him from his wheelchair to a car so they can take him to the movies or out to eat. When they recently took a trip to the beach, Julie said five of Jack's friends carried him out to the sand a lesson on how hard it is to navigate the world in a wheelchair.

Jack said he believes technology one day will advance enough that he won't be paralyzed forever. He also volunteered to do stem cell surgery to allow doctors to study the affects of stem cells on his spine for the next 15 years. Instead of wallowing in self pity, he's moving forward. But he'll need help.

"I'm appreciating everything in the now," he said.

Doctors have said Jack has adapted faster than expected, but there are still some everyday essential tasks that are out of his reach. He cannot write or cook. He can shower himself but can't dry himself or transfer himself in and out of his wheelchair. The Massey family hopes to secure a personal care attendant for Jack at school, but until then Julie will be in Gainesville to help him transition. An occupational therapy student from the university will also help Jack on a temporary basis.

Finding proper care for her son has proven to be a learning experience for Julie and her husband, Lance.

"I don't know how people do it," she said. "We have good health care, but then there's hidden costs. There's travel expenses. ... It's kind of humbling. Nobody should have to go to GoFundMe for medical help."

Jack wants to spend his final year as an undergrad as independent as possible. After months of helping him recover, Julie said it will be hard to let her son go. Jack is the oldest of three; his brother Lance is 19 and a student at UF and his sister Alina is 14 and attends Ruckel Middle School.

"It's like letting him go off to kindergarten again," she said.

As for life after college, Jack said he doesn't feel limited in career choices. One of his professors in the geology department encouraged him by saying that there were plenty of opportunities he could pursue in that field. Jack said he may also consider law school. One thing he's learned through this life-altering experience is that there are no limits to what he can achieve.

"I haven't done that much deep thinking. I just go with the flow," he said. "But I learned I have more perseverance. I'm more mentally tough than I thought I was. I'm appreciative for life in general. That's one of the big things."

Read more:
'I still have my brain' - The Northwest Florida Daily News

Spinal Cord Stem Cells Comments Off on ‘I still have my brain’ – The Northwest Florida Daily News | August 16th, 2017

August 15, 2017

What if one day, we could teach our bodies to self-heal like a lizards tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimers or Parkinsons disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institutes Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

One of the things that that attracted me most to the ASU and the Biodesign CMDB was Haos vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology, Stephanopoulos said.

I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice.

Yans research center uses DNA and other basic building blocks to build their nanotechnology structures only at a scale 1,000 times smaller than the width of a human hair.

Theyve already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physics and the creative imaginations of these budding nano-architects.

The goal of the Center for Molecular Design and Biomimetics is to usenatures design rulesas an inspiration in advancing biomedical, energy and electronics innovation throughself-assembling moleculesto create intelligent materials for better component control and for synthesis intohigher-order systems, said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeleys Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

Weve learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems, Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity, Stephanopoulos said.

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to grab the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal off. To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the materials surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinsons disease.

More:
Restoring loss: Bio-inspired materials give boost to regenerative medicine - Arizona State University

Spinal Cord Stem Cells Comments Off on Restoring loss: Bio-inspired materials give boost to regenerative medicine – Arizona State University | August 15th, 2017

Many doctors tell patients and families that recovery does not occur after spinal cord injury. This is not true. Recovery is the rule, not the exception after spinal cord injury.

Segmental recovery. Most patients recover 1-2 segments below the injury site, even after so-called complete spinal cord injuries. For example, a person with a C4/5 injury may have deltoid function on admission and then recover biceps (C5), wrist extensors (C6), and perhaps even triceps (C7) after several months, and the associated dermatomes.

Recovery due to methylprednisolone. The second National Acute Spinal Cord Injury Study (NASCIS 2) showed that patients with complete spinal cord injuries and who did not receive the high-dose steroid methylprednisolone recovered on average 8% of motor function they had lost. If they received methylprednisolone within 8 hours after injury, they recovered on average 21% of what they had lost. In contrast, people with incomplete spinal cord injury recovered on average 59% of motor function and 75% if treated with high dose methylprednisolone.

Recovery of postural reflexes. Most people with cervical or upper thoracic spinal cord injury are initially unable to control their trunk muscles. However, most will recover better trunk control over months or even years after injury.

Walking quads and paras. Most people with incomplete spinal cord injuries, i.e. ASIA C, will recover standing or walking. Walking recovery after complete spinal cord injuries, i.e. ASIA A, are rare but can occur in 5% of the cases. In the 1980s, less than 40% of spinal cord injuries admitted to hospital were incomplete. However, in the 1990s, over 60% of spinal cord injuries are incomplete and thus the incidence of walking quads or walking paras may be higher than most people think.

Both animal and human studies indicate that as little as 10% of spinal cord tracts can support substantial function, including locomotion. People often can walk even though a tumor has damaged 90% of their spinal cord. This is due to the redundancy and plasticity of the spinal cord. Multiple spinal pathways serve similar or overlapping functions. Plasticity refers to the ability of axons to sprout and make new connections. Because transected spinal cords are rare, most people have some spinal axons crossing the injury site. This is the basis of the hope that even slight regeneration of the spinal cord will restore substantial function.

Experimental Therapies for Subacute Spinal Cord Injury

Several experimental therapies are being tested in clinical trial for spinal cord injury during the first days or weeks after injury.

Monosialic ganglioside (GM1, Sygen). In 1991, Fred Geisler and colleagues reported that GM1 injected daily for 6 weeks after injury improve locomotor recovery 37 patients. Fidia Pharmaceutical subsequently tested this therapy in a large multicenter clinical trial in 800 patients, showing that the GM1 accelerated recovery during the first six weeks but did not significantly improve the extent of recovery at 6-12 months after injury. Note that this trial is no longer active. Although the drug is still available in Europe and South America, the company Fidia has been bought by another company. CareCure Forum (GM1) Link

Activated macrophage transplants. In 1998, Michal Schwartz at the Weizmann Institute reported that activated macrophages obtained from blood and transplanted to the spinal cord improve functional recovery in rats. The company Proneuron initiated phase 1 clinical trials to assess feasibility and safety of macrophage transplants in human spinal cord injury. Preliminary reports suggest that the treatment is feasible and safe. All the patients had complete thoracic spinal cord injury and received macrophage transplants within 2 weeks after injury. Three of the 8 patients recovered from ASIA A to ASIA C, more than the expected 5%. A phase 1 clinical trial is continuing at Erasmus Hospital in Brussels, Belgium. A phase 2 trial is being planned in two U.S. centers including Craig Hospital in Denver (CO) and Mt. Sinai in New York City (NY). CareCure Forum (Macrophage) Link

Alternating Current Electrical Stimulation. In 1999, Richard Borgens and colleagues at Purdue University reported that alternating currents applied to dog spinal cords stimulated regeneration and recovery of function in dogs with spinal cord injury. A clinical trial has commenced at Purdue University for people who are within 2 weeks after acute spinal cord injury. CareCure Forum (AC Stim) Link

AIT-082 (Neotrofin). This is a guanosine analog that can be taken orally and reportedly increases neurotrophins or neural growth factors in the brain and spinal cord. Neotherapeutics tested this drug in patients with Alzheimers disease. They started a multicenter clinical trial at Ranchos Los Amigos in Downey (CA), Gaylord Hospital in Wallingford (CT), and Thomas Jefferson Hospital in Philadelphia. The treatment must be started within 2 weeks after spinal cord injury. CareCure Forum (AIT-082) Link

Experimental Therapies for Chronic Spinal Cord Injury

Several therapies are being tested in clinical trials for chronic spinal cord injury, i.e. people whose neurological recovery has stabilized one or more years after injury. Many other treatments are being considered for clinical trial (see article on Advances in Spinal Cord Injury Therapy 25 November 2002).

4-aminopyridine (4-AP). This drug is a small molecule that blocks fast voltage sensitive potassium channel blockers. The drug can be obtained by physician prescription from compounding pharmacies in the United States. In addition, Acorda Therapeutics is carrying out a multicenter phase 3 clinical trial of a sustained release formulation of the drug in people who are more than one and a half years after incomplete spinal cord injury. The drug may improve conduction of demyelinated axons in the spinal cord and preliminary clinical trial results suggest that the drug may reduce spasticity and improve motor or sensory function in as many as a third of people with chronic spinal cord injury. See CareCure Forum (4-AP) Link

Fetal porcine stem cell transplants. Embryonic stem cells have attracted much attention. Several studies of human fetal cell transplants have been carried out in Sweden, Russia, and the United States, showing that transplanted fetal cells will engraft in human spinal cords. However, due in part of the lack of availability of adult human stem cells for transplantation and politics associated with the use of embryonic human stem cells, the first and only stem cell therapy trial for spinal cord injury in the United States used fetal stem cells from pigs. A phase 1 clinical trial at Washington University in St. Louis (MO) and Albany Medical Center in Albany (NY) has transplanted fetal stem obtained from pig fetuses and treated with antibodies to reduce the immune rejection. Sponsored by Diacrin, this trial is aiming to test 10 patients. See CareCure Forum (Diacrin) Link

Olfactory ensheathing glial transplants. Olfactory ensheathing glia (OEG) reside in the olfactory nerve and the olfactory bulb. They are believed to be why the olfactory nerve continuously regenerates in adults. OEG cells are made in the nasal mucosa and migrate up the nerve to the olfactory bulb. Several laboratories have shown that OEG transplants facilitate regeneration of the spinal cord. Three clinical trials have started in Lisbon (Portugal), Brisbane (Australia), and Beijing (China). In Lisbon, they are transplanting nasal mucosa obtained from the patient into the spinal cord. In Brisbane, they are culturing OEG cells from nasal mucosa and transplanting the cells to the spinal cord. In Beijing, they are culturing OEG from human fetal olfactory bulbs and transplanting into the spinal cord. See CareCure Forum Link (Brisbane) and CareCure Forum Link (Beijing)

Summary

Spinal cord injury is devastating, not only for the injured person but for families and friends. While much information is available on Internet, most of the material is scattered and out of date. This article summarizes answers to some of the most frequently asked questions by people who are encountering spinal cord injury for the first time. Spinal cord injury disconnects the brain from the body. This leads not only to loss of sensation and motor control below the injury site but may be associated with abnormal activities of the spinal cord both above and below the injury site, resulting in spasticity, neuropathic pain, and autonomic dysreflexia. Many functions of our body that we take for granted, such as going to the bathroom, sexual function, blood pressure and heart rate, digestion, temperature control and sweating, and other autonomic functions may not only be lost but may be abnormally active. Finally, contrary to popular notions about spinal cord injury, recovery is the rule and not the exception in spinal cord injury. The recovery takes a long time and may be slowed down or blocked by the muscle atrophy and learned non-use. Finally, there is hope. Many therapies have been shown to regenerate and remyelinate the spinal cord. Some of these are now in clinical trials and many more should be in clinical trial soon.

Recovery and TreatmentWise Young, MD, PhD

See the original post:
Does recovery occur after spinal cord injury? | Answers ...

Spinal Cord Stem Cells Comments Off on Does recovery occur after spinal cord injury? | Answers … | August 14th, 2017

FREMONT, Calif., Aug. 14, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced that Cancer Research UK, supported by Asterias technical personnel, has successfully completed manufacture of the first cGMP (current Good Manufacturing Practice) clinical grade lot of AST-VAC2, which meets all specifications for release. This lot will provide clinical trial material for patients enrolling in the first clinical study evaluating AST-VAC2 in non-small cell lung cancer.

The successful production of this first cGMP lot of AST-VAC2 is a major step towards initiating the upcoming study in non-small cell lung cancer, said Mike Mulroy, President and Chief Executive Officer. With its potential as a ready-to-use, off-the-shelf cancer immunotherapy, AST-VAC2 represents an exciting opportunity for Asterias in the rapidly evolving cancer immunotherapy sector.

Investigational therapies intended for human clinical applications must be manufactured in accordance with cGMP standards designed to help assure the safety and potency of drug products. To achieve cGMP standards, a product must be manufactured and released according to rigorous systems designed to ensure appropriate control of manufacturing facilities, equipment, raw materials, processes and testing procedures. Under the companys agreement with Cancer Research UK, Asterias has transferred its innovative laboratory scale AST-VAC2 manufacturing process to Cancer Research UKs Biotherapeutics Development Unit, which has developed and optimized the process for cGMP manufacture and is responsible for producing cGMP AST-VAC2 for use in the upcoming clinical study in non-small cell lung cancer.

About AST-VAC2

AST-VAC2 is an innovative immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells. These non-patient specific (allogeneic) AST-VAC2 cells are engineered to express a modified form of telomerase, a protein widely expressed in tumor cells, but rarely found in normal cells. The modified form of telomerase permits enhanced stimulation of immune responses to the protein. The AST-VAC2 dendritic cells instruct the immune system to generate responses against telomerase which will target tumor cells. AST-VAC2 is based on a specific mode of action that is complementary and potentially synergistic to other immune therapies.

About Non-Small Cell Lung Cancer

Lung cancer (both small cell and non-small cell) is the leading cause of cancer-related death, accounting for about one-quarter of all cancer deaths and more than colorectal, breast, and prostate cancers combined. Non-small cell lung cancer (NSCLC) accounts for about 80% to 85% of lung cancers, according to the American Cancer Society. The three main types of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma. The American Cancer Societys estimates for lung cancer in the United States for 2017 are: about 222,500 new cases of lung cancer, and about 155,870 deaths from lung cancer. Despite the large number of people afflicted by non-small cell lung cancer, patients remain vastly underserved due to a scarcity of effective treatments. According to statistics published by Cancer Research UK, the five year survival rate for lung cancer patients in England and Wales is less than 10%.

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 the first 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.

About Cancer Research UK

Cancer Research UK is the worlds leading cancer charity dedicated to saving lives through research. Cancer Research UKs pioneering work into the prevention, diagnosis and treatment of cancer has helped save millions of lives. Cancer Research UK receives no government funding for its life-saving research. Every step it makes towards beating cancer relies on vital donations from the public. Cancer Research UK supports research into all aspects of cancer through the work of over 4,000 scientists, doctors and nurses.

FORWARD-LOOKING STATEMENTSStatements 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.

See more here:
Asterias Announces Major AST-VAC2 Development Milestone: First ... - GlobeNewswire (press release)

Spinal Cord Stem Cells Comments Off on Asterias Announces Major AST-VAC2 Development Milestone: First … – GlobeNewswire (press release) | August 14th, 2017

BUFFALO, N.Y. People with multiple sclerosis (MS) knowall too well the frustration of hearing that success in treatingthe disease in mice had little or no effect in humans.

Unfortunately, with no large animal models for MS, results thatsuggest promising new treatments in mice often are ineffective inhumans.

Now, University at Buffalo researchers have developed andsuccessfully tested a method for determining how relevant to thehuman disease findings are from mouse models. The researchwas published Aug. 8 in Stem Cell Reports.

This is an important resource for the field as it allowsus to compare human and rodent cells, and provides a point ofreference to understand whether or not gene expression patterns areconserved between species, said Fraser Sim, PhD, seniorauthor and associate professor in the Department of Pharmacologyand Toxicology in the Jacobs School of Medicine and BiomedicalSciences at UB. Co-first authors are Suyog U. Pol PhD, now apostdoctoral fellow, and Jessie J. Polanco, a doctoral candidate,both in the medical school.

MS trial failures

There have been so many failures in clinical trials forMS when promising observations are translated from small animalmodels to the clinic, Sim said. Our primarymotivation was to try to understand, at a molecular level, how thehuman cells responsible for synthesizing myelin differ from theirmuch-better-studied mouse counterparts.

MS and some other neurological diseases occur when there isdamage to myelin the fatty sheath that allows nerve cellsto communicate. So the myelin-producing cells, called humanoligodendrocyte progenitor cells, or OPCs, found in the brain andspinal cord have been a major focus of efforts to better understandMS and develop potential new treatments for it.

Sim explained that undifferentiated OPCs are frequently found inthe brain lesions of MS patients, so boosting the differentiationof these cells could lead to myelination and a reduction ofsymptoms.

From OPCs to oligodendrocytes

One reason why so many clinical trials fail may be because offundamental differences in the types and levels of genes expressedbetween mice and humans. Sim and his colleagues addressed thisquestion by performing gene-expression analysis on differentiatinghuman OPCs.

In this paper, we describe the transcriptional eventsthat underlie how human OPCs develop into oligodendrocytes,said Sim.

To do it, they used a network analysis software tool calledweighted gene coexpression network analysis (WCGNA). The softwareclusters together genes with similar patterns of expression. Italso allows for analysis of both conserved and divergent geneexpression between humans and rodents.

WCGNA looks at the relationships between genes ratherthan absolute differences between conditions in any givenexperiment, Sim said.

He added that the information encoded in levels of geneexpression increasing or decreasing is very reliable andreproducible.

We performed WCGNA in exactly the same manner on cellsisolated from mice, rats and humans, and prepared these cells in asclose to matched conditions as possible, trying to keep things assimilar as possible to facilitate this comparison, saidSim.

It turned out several of the genes the team had identified asrelevant to human disease also are involved in mouse developmentand mouse models of myelin disease.

New myelin-repairing gene

Based on its findings from that analysis, the team had predictedthat GNB4, a protein involved in signal transduction, would beinvolved in the development of OPCs in humans. The researchersfound that over-production of GNB4, a protein involved in thetransduction of extracellular signals, could cause human OPCs torapidly undergo myelination when transplanted into a model forhuman cell therapy in MS.

So this proteins expression in oligodendrocyteprogenitor cells might ultimately become a therapeutic target,potentially promoting oligodendrocyte formation in MSpatients, said Sim.

The approach also identified several other important candidatesthat play key roles in regulating the development of humanoligodendrocytes.

Other co-authors on the paper are Melanie A. OBara,research scientist; Hani J. Shayya, a UB undergraduate and Karen C.Dietz, PhD, research assistant professor, all of the Department ofPharmacology and Toxicology and Richard A. Seidman, amasters candidate in neuroscience.

The research was funded by the National Multiple SclerosisSoociety, the Kalec Multiple Sclerosis Foundation, the SkarlowMemorial Trust and the Empire State Stem Cell Fund (NYSTEM) throughthe New York State Department of Health.

Read more from the original source:
Which research results in mice will help humans with MS? Now there's a way to tell - UB News Center

Spinal Cord Stem Cells Comments Off on Which research results in mice will help humans with MS? Now there’s a way to tell – UB News Center | August 14th, 2017

INCREDIBLE biomedical breakthroughs including growing beating heart tissue and engineering the worlds first muscle flap transplant with its own blood supply have been achieved by Professor Shulamit Levenberg in her lab at Israels Technion since 2007.

Now researchers at three Australian universities will benefit from collaborative projects with the Technions Dean of Biomedical Engineering, strengthened during her visit to Perth and Sydney from July to mid-August, facilitated by Technion Australia.

Levenberg told The AJN that growing 3D tissue structures that have blood vessels, by using stem cells and biodegradable scaffolds, has so much potential to repair damaged organs, treat diabetes and even spinal cord injuries.

To see the whole piece of cardiac tissue we created in the lab spontaneously beating in front of your eyes was really amazing, Levenberg said.

But what really excited us was that we could create a blood vessel network, which is critical in keeping the implant alive and making revascularisation in the body faster.

Tissue engineering has been used to create islet transplants for diabetes patients, to repair severe tissue injuries in mice and even to produce meat in the lab.

That is a new application, but we are very excited about the regenerative medicine direction, and we hope spinal cord regeneration will happen in humans one day, Levenberg said.

The next step is to make the tissues larger and to move from pre-clinical to clinical trials.

Levenberg came on a Raine Fellowship to the University of Western Australias (UWA) Perkins Institute for Biomedical Research, where she lectured, and connected researchers with Technion colleagues.

We are starting several research projects with UWA on using combinations of biomaterials, and weve got funding for spinal cord regeneration research from the Perth-based Shervington Fund, Levenberg said.

Were also discussing further collaborations in spinal cord research with the University of Technology in Sydney, and we now have a collaboration with Professor Tony Weiss at the University of Sydney, where we are using tropo-elastin that he developed to coat scaffolds.

Technion Australia is in the process of planning a fundraising project to support that collaboration.

A passionate mentor to biomedical engineering students and to girls interested in science, Lewenberg said, I think the most important thing is to show young people that to be a scientist is doable and exciting. My advice is to do what excites them, and not be afraid that something may seem too difficult.

SHANE DESIATNIK

More here:
Technion prof inspires Aussie researchers - Australian Jewish News

Spinal Cord Stem Cells Comments Off on Technion prof inspires Aussie researchers – Australian Jewish News | August 13th, 2017

Northern Ireland mum fighting MS: Russian medics are now my last hope

BelfastTelegraph.co.uk

A young Co Down mum is bravely undergoing a gruelling stem cell transplant in Russia in what she believes is her last hope of enjoying some quality of life.

http://www.belfasttelegraph.co.uk/news/northern-ireland/northern-ireland-mum-fighting-ms-russian-medics-are-now-my-last-hope-36023340.html

http://www.belfasttelegraph.co.uk/life/features/article36023337.ece/4289a/AUTOCROP/h342/2017-08-12_lif_33652492_I8.JPG

A young Co Down mum is bravely undergoing a gruelling stem cell transplant in Russia in what she believes is her last hope of enjoying some quality of life.

Lindsay Rice (35) from Warrenpoint has exhausted every treatment on the health service - including chemotherapy normally given to cancer patients - in the hope of treating the chronic condition Rapidly Evolving Severe Relapsing Remitting Multiple Sclerosis.

Paralysis and temporary sight loss are just a few of the many debilitating symptoms which have left the mum-of-two unable to enjoy normal family life.

Desperate to get her help, her family launched an appeal on Facebook and Go Fund Me to raise 50,000 to send her to the National Pirogov Medical Surgical Centre in Moscow where she arrived two weeks ago to start her stem cell transplant.

The treatment alone is expected to cost up to 45,000 and, incredibly, in just 12 weeks the family has raised 32,000 towards a 50,000 target thanks to generous support from friends and the public.

Lindsay, who is married to Liam (36), a financial advisor, has two children, Jamie (17) and Olivia (8).

Liam says: "This is her last hope and she is doing it for her family and her kids and that's what she is focusing on. She just wants to be able to live a normal life and do normal things with the family."

Since starting her treatment on August 1 she has been keeping a daily dairy of her progress through a Facebook page - Lindsay's Last Hope.

While the groundbreaking treatment known as HSCT (Haematopoietic Stem Cell Transplant) is not a cure for MS, Lindsay's hope is that it will halt the progression of the disease and stop the frequent and severe relapses which are destroying her health.

Lindsay will spend a month in the clinic, most of it in isolation, and when she comes home she faces a long recovery period when she will have to remain isolated for up to a year due to the risk of infection.

HSCT aims to 'reset' the immune system to stop it attacking the central nervous system. It uses chemotherapy to remove the harmful immune cells and then rebuild the immune system using a type of stem cell found in the patient's bone marrow.

The haematopoietic stem cells used in the treatment can produce all the different cells in the blood, including immune cells. However, they can't regenerate permanently damaged nerves or other parts of the brain and spinal cord.

Lindsay has successfully had over two million stem cells extracted in a tough procedure which involved having a catheter inserted into her jugular vein. She has also had her head shaved this week in preparation for starting chemotherapy today.

The chemotherapy will wipe out her immune system and she will then have her stem cells transplanted back into her blood by a drip to help regrow a new, stronger immune system.

She will then have to spend 10 days in complete isolation while her new immune system builds.

Also, since arriving in Russia she has been told that her MS is now much worse than she realised and is now at the Secondary Progressive stage.

People with Secondary Progressive MS don't tend to recover completely from a relapse and can expect a general worsening of symptoms, making the treatment even more time-critical.

In a further blow, tests have picked up a potentially dangerous three-centimetre active lesion on her spine which wasn't spotted during MRI's here.

Lindsay faces a tough few weeks in her bid to halt the progression of the disease but as her husband Liam explains, the alternative is the prospect of life in a wheelchair: "Lindsay has come through a lot since her teens.

"She had Jamie quite young at 18 and her condition seemed to really deteriorate after that. She went to a lot of consultants and had many tests but it wasn't until after she had Olivia that she was finally diagnosed in 2011.

"She never knows from day to day how it will affect her. Fatigue is the number one problem and that is crippling. I would come home from work and after dinner she has to go to bed, and even sleep doesn't help it.

"It stops her from doing simple things like taking our daughter to the park or taking the dog for a walk.

"Her motability is not as good as an average person and the other big issue is the relapses.

"They have become very frequent and each relapse is worse in terms of how severe it is. During her last one in February she had to go into hospital and also had to use a walking frame.

"A common misconception is that after each relapse you go back to normal but that's not the case. It leaves its mark and any damage done is permanent. The nature of the relapses could leave her in a wheelchair."

It was after her last relapse and having exhausted all options for treatment on the Health Service that Lindsay decided she wanted to try HSCT.

Her neurologist in Belfast supported her decision and the family applied to the Russian clinic just 12 weeks ago expecting to wait up to two years before admission.

They were surprised to be offered a cancellation on August 1 leaving them facing a race against time to raise 50,000 to cover the cost of treatment and expenses.

Liam says: "We thought we would have at least 12 months and up to two years to get the money together and it has been amazing to see how people have rallied round and what they have done just from the kindness of their hearts, especially strangers.

"We've had quizzes and coffee mornings and online auctions and I recently did the Four Peaks challenge with a group of friends. Lindsay's mum and her best friend are organising a lot of events and we still have some way to go but we are amazed at how much has been raised and donated in such a short time."

Liam flew to Russia with Lindsay on July 31 and stayed with her for five days while she underwent tests to determine that she was suitable for the treatment.

It has already been a punishing two weeks for Lindsay who has come through a batch of invasive procedures including having a catheter inserted in her jugular to extract the stem cells.

Liam says: "It is an intense treatment and Lindsay is so positive and coping brilliantly. She got her hair cut short before she went and decided to have it shaved this week before the chemo starts and it falls out.

"She will have to spend 10 days in complete isolation to allow her immune system to build again and that will be tough.

"She will hopefully be home after 30 days and then when she comes home she will have a long recovery and will have to isolate herself from society for up to a year to keep her safe from infection.

"We will have to deep clean the house and we will all have to wear face masks as she can't risk even getting a cold."

Liam is back at work and trying to keep things as normal as possible at home for the couple's two children, who he said are coping well: "Jamie is 17 and approaching adulthood and understands why she is doing it and is okay, but obviously his mum is away and he has his sixth year exam results coming and he misses her.

"Olivia seems to be fine too. She understands her mum has MS, which stops her doing things with her and she knows this treatment is to help her to be a better mother.

"I've been trying as much as possible to keep her occupied with play dates and sleepovers."

The couple have been impressed by the level of care in the clinic and Lindsay has had the chance to meet and get to know other MS patients from all over the world.

Liam has nothing but admiration for her strength and the positive way she is enduring the extreme procedures she faces.

He adds: "Lindsay is the most determined person you could ever possibly meet. She has had bad days and it can be demoralising for her but she is determined to be as positive as she can be.

"It is not a cure. MS doesn't have a cure but we hope it will stop the progress of the disease. We just hope it will halt it by rebooting her immune system and hopefully stop the severe relapses."

Liam adds: "It is desperately hard and stressful for all of us and we have to put a positive spin, in the grand scheme of things it is just for a month of her life."

Follow Lindsay's journey at Facebook/Lindsay's Last Hope - HSCT in Russia

Fundraising continues as the family has only until the end of the month to reach their target. You can support this young mum in her bid to enjoy a normal quality of life by going to https://www.gofundme.com/lindsay-slasthope

Belfast Telegraph

Read more:
Northern Ireland mum fighting MS: Russian medics are now my last hope - Belfast Telegraph

Spinal Cord Stem Cells Comments Off on Northern Ireland mum fighting MS: Russian medics are now my last hope – Belfast Telegraph | August 12th, 2017

It is also the story of her family, friends, and community and their spiritual and financial commitment in support of her fight.

Inherently it is also about the unenviable position millions of Americans find themselves in, isolated between insurance companies and the medical establishment of this country, forced to seek medical care beyond our borders.

"Over a two-year period, I started having these terrible dizzy spells, losing my balance, and when I would bend my head forward, I would go numb all over. I was losing my vision and couldn't hear out of my right ear. I was 25," recalled Somerset's Barb Rivard.

Rivard grew up in Glenwood City. She has three grown children and six grand children. She tended bar for 19 years, graduated from WITC in 1999, and worked as a scheduler in the physical therapy department at Westfields Hospital & Clinic in New Richmond for 14 years. She reluctantly gave up her position at the hospital three years ago because of her disease.

Ask around and you will find she has a reputation for being independent, some might say stubborn, and she wants to keep it that way.

"I don't want people feeling sorry for me. I've been called bullheaded. For me it's tough, I don't like to ask for help. I was a single mother with my boys for a lot of years," said Rivard.

When she first started experiencing symptoms, her family doctor sent her to a neurologist who concluded she had an issue causing spine inflammation and he sent her on her way.

When her symptoms persisted, nearly costing her her job tending bar, she returned to the neurologist for more testing. At the time, Rivard's mother was dying from brain cancer, leading her to wonder if she might also have brain cancer. Meanwhile, her family physician speculated it might be a brain tumor.

"The tests came back and the neurologist told me I had multiple sclerosis (MS). He said, 'You're a young healthy woman and it will never bother you again.' He sent me on my way, again. At that point, I couldn't see out of my left eye, but I thought, 'Okay good, at least we know something.' I knew absolutely nothing about MS," said Rivard. It was 1990.

What is MS?

MS presents in people in four different ways according to International Advisory Committee on Clinical Trials of MS: clinically isolated syndrome (CIS), relapsing-remitting MS (RRMS), primary progressive MS (PPMS), and secondary progressive MS (SPMS).

Rivard was diagnosed with RRMS.

MS is a chronic disease that attacks the central nervous system, (brain, spinal cord and optic nerves). Symptoms can include loss of vision, pain, fatigue, muscle spasms, impaired coordination, and numbness in the limbs. In severe cases, the patient can become paralyzed or blind.

Often the severity and progression of the disease is determined by an MRI to identify lesions within the central nervous system.

Treatment

The approved course of treatment in the U.S. is any one of a number of powerful drugs known as disease modifying-therapies (DMT), taken either by injection, intravenously or orally, designed to decrease the frequency of relapses and delay the progression of the disease. Attacks or relapses are frequently treated with high doses of steroids for immediate relief. There is currently no known cure for MS.

Upset that the neurologist had failed to communicate with him regarding Rivard's condition, her family physician sent her to a second neurologist. Over the next four years, a succession of neurologists, approved by her health insurance, treated her with a prescription of DMTs. The injections can be painful and expensive.

"They kept putting me on these once-a-month injections that were extremely high priced. They ran anywhere from $2,000 to $4,000 a month and that was 25 years ago. Some of the drugs I took every day, some were every other day, and one of them was this once-a-week self-injection. They made me super, super sick. I had this big needle I had to stick in the top of my leg. It was horrible. I had big welts everywhere," said Rivard.

All in all, Rivard tried the various drug regimens for 15 years. She continued to suffer relapses accompanied by sickness brought on by the drugs themselves only compounding her frustration with her doctors.

"At one point, one of my neurologists told me I didn't know what I was talking about when it came to my own body. I wasn't so pleasant when I told him I didn't need his services any more," recalled Rivard.

Five years ago, she hooked up with Dr. Rita Richardson, a neurologist who visits at Westfields Hospital & Clinic in New Richmond.

"Dr. Richardson and I just really get along. I absolutely love her. She's one of those doctors who will sit there and listen. She actually cares. Finally, after 20 years," said Rivard.

A new approach

Three years ago, Rivard implemented a new approach to her disease, no more DMTs. She began working with a nutritionist.

"We don't eat out of a box anymore. We eat healthier and we know what we are eating," said Rivard.

She and her husband eat beef they raise themselves and vegetables and fruit from their own garden. In addition to a new diet, Rivard tries to maintain a regular physical fitness routine swimming five times a week, riding her bike and attempting a little yoga at home. She feels better both physically and financially having eliminated expensive drugs from her budget. However the MS continued to relapse causing debilitating episodes and regular spasticity particularly in her legs.

"My whole body goes wild. I can't walk. I either sleep all the time or I don't sleep. I go to the bathroom. I might as well just sit in the bathroom. Usually I feel weak, very weak. My husband can sense it coming on more than I can. I live with it, but he witnesses it. Most of the time, he'll say, 'I think I need to take you in.' After the first dose of steroids, I usually feel better," said Rivard describing a relapse.

Four years ago, Rivard had a Baclofen pump inserted to control the spasticity in her legs. Baclofen is a muscle relaxant and antispastic commonly used to address spasticity in MS patients. She resigned her position at the hospital.

"I knew I couldn't do this anymore, so I told them I was resigning so as not to leave them hanging. I miss my job, but I still have my care team. When I go in for my treatments, everybody still comes up and hugs and kisses me," said Rivard.

Clinica RUIZ

After years of feeling experimented on and left out of the equation when it came to managing her own care, Rivard began reading extensively about MS to educate herself about her disease. That is when she learned of hematopoietic stem cell transplants (HSCT) and Clinica RUIZ in Puebla City, Mexico.

HSCT treatment for MS essentially rewrites a diseased person's immune system. A person's stem cells are harvested; their immune system is wiped out taking with it any memory of the disease. Their stem cells are reintroduced to a disease free environment where they repair and reconfigure neural damage done by the disease. Ideally it halts any progression of the disease and returns function to varying degrees depending on the individual without the use of any DMTs.

Rivard initially applied to the only HSCT program in the U.S. being conducted at Chicago's Northwestern Memorial Hospital. The program has been in place since 2015 but is highly selective and expensive, $125,000 per patient. She was rejected for the program due to her age and her MRI revealed no active lesions. However, through the Chicago program's website, Rivard connected to an online community where alternatives to the program in Chicago were explored. That is where she met Bill, who lives in Roberts, and Nancy, who lives in Amery, fellow MS sufferers who had both undergone HSCT at Clinica RUIZ in Mexico.

"I had the information sitting here for about two months. Finally I talked to Dr. Richardson about it and she said, 'Go for it.' The clinic has an application process online. It took me a half hour to fill out. I applied on Sunday and was accepted the next day," said Rivard.

Clinica RUIZ is operated by Dr. Guillermo Ruiz Argelles in Puebla City, Mexico. Ruiz Arguelles has conducted more than 700 stem cell procedures since 1996. According to his web site, hsctmexico.com, a simplification of the grafting process (collection of the stem cells) refined over the years, has resulted in a substantial decrease in cost to the patient. It has also enabled most of the procedures to be conducted on an outpatient basis. Ruiz Arguelles and his staff have produced numerous academic articles and received numerous awards and recognition including election as a Distinguished Mayo Alumnus and Master of the American College of Physicians.

Rivard relapsed three times between January and May this year. The prospect of hope and promise of a high percentage of success (better than 80 percent of patients experience some degree of success halting the disease's progression and better yet, reversing their disability) was all encouraging to her, still it was Mexico and so far it was just words.

Seeing actual, physical results in the persons of Bill and Nancy and being able to ask them questions about their personal experiences convinced Rivard this opportunity was the real deal.

Nancy was diagnosed with MS one year ago. She returned from her HSCT treatment at Clinica RUIZ on March 27, 2017. She is 44 years old. She also shares Rivard's neurologist, Dr. Richardson.

"I saw these two pictures posted by a woman. One of her the day she was leaving the hospital in Chicago after she received treatment four years ago. And the other was that day, after she had finished a ten-mile run. I asked myself, 'Why am I waiting? I can't run.' Even if this treatment did nothing but stop it, I was happy to try it. I had started to use a cane, which was mentally difficult for me. It's not my goal to need a neurologist to aggressively treat me. My goal is that I beat this. I just need somebody to help me if I need it. Dr. Richardson has been really accepting that I had this radical treatment. This treatment has helped me way more than I had ever hoped. This morning I posted a video of me doing hopscotch. I saw immediate improvement during treatment. I ditched my cane two weeks into treatment. Now I'm working out at the gym. I'm getting my balance back and learning how to jump rope," described Nancy.

In May, Barb paid $54,500 to Clinica RUIZ in advance of her treatment. Her health insurance will not pay a dime toward her treatment. As of mid June, numerous fundraisers organized by friends and family including a live auction, meat raffle and spaghetti feed have raised more than $41,000 to steadily chip away at the financial obligation. In exchange for a rusting antique grain truck engulfed in weeds in her backyard, Rivard will receive three round-trip airline tickets to Mexico City courtesy of her brother-in-law.

"He's had his eye on that old truck for years. That's a win, win for me," said Rivard with a big smile.

The next step

She will leave for Mexico Saturday, Aug. 12. From the airport in Mexico City, it is a two-hour bus ride to the clinic in Puebla City. On Monday, Aug. 14, Rivard will be assigned to one of four groups of five patients and undergo a full day of testing. The clinic has the capacity to treat 20 patients at a time.

Over the course of the next three weeks, Rivard will undergo potent chemotherapy to kill off any infections and eliminate memory cells in her immune system. She will then receive a series of injections to stimulate the growth of her stem cells. Stem cells from her own body will then be harvested using a process called aphaeresis. Following the harvest, she will receive a second round of chemo preceding the reintroduction of her previously harvested stem cells back into her body. Once the stem cells have been transplanted she will enter a neutropenic period during which her body is very susceptible to infection. She will eat a specific diet to help her body recover and have very little contact with the outside world. During that period the stem cells are beginning to grow in an environment cleansed of the previous disease beginning to repair and reconfigure any neural damage done by the disease. Before she leaves Mexico, she will begin receiving a course of Rituxan injections intended to hold her immune system at bay killing off cells, which would otherwise attack the new stem cells impeding the recovery process. Those injections continue every other month for nine months. Staff at Clinica RUIZ will be in contact with her hematologist, Dr. McCormack, before she leaves to begin monitoring her recovery. To aid in Clinica RUIZ's research, Rivard will continue to update her progress every three months using an online report form. Provided everything goes as planned, Barb will return home Sept. 9.

The range of recovery stories is amazing. People restricted to wheelchairs are walking. Rivard's friend Nancy went back to work, sans cane, two weeks after she returned home. Her friend, Bill, is continuing to improve a year and a half removed from his trip to Clinica RUIZ. Studies indicate patients can continue to improve for two or more years after treatment.

"Nothing else is helping me. I've been reading about this for a long time. The biggest drawback is, it doesn't work. I have to do it. I'm excited to go."

Follow her journey on Facebook at: http://www.facebook.com/bean.langness.

Follow this link:
The power of hope - Richmond-News

Spinal Cord Stem Cells Comments Off on The power of hope – Richmond-News | August 12th, 2017

Umbilical cord blood contains haematopoietic (blood) stem cells. These cells are able to make the different types of cell in the blood - red blood cells, white blood cells and platelets. Haematopoietic stem cells, purified from bone marrow or blood, have long been used in stem cell treatments for leukaemia, blood and bone marrow disorders, cancer (when chemotherapy is used) and immune deficiencies.

Since 1989, umbilical cord blood has been used successfully to treat children with leukaemia, anaemias and other blood diseases. Researchers are now looking at ways of increasing the number of haematopoietic stem cells that can be obtained from cord blood, so that they can be used to treat adults routinely too.

Beyond these blood-related disorders, the therapeutic potential of umbilical cord blood stem cells is unclear. No therapies for non-blood-related diseases have yet been developed using HSCs from either cord blood or adult bone marrow. There have been several reports suggesting that umbilical cord blood contains other types of stem cells that are able to produce cells from other tissues, such as nerve cells. Some other reports claim that umbilical cord blood contains embryonic stem cell-like cells. However, these findings are highly controversial among scientists and are not widely accepted.

Continue reading here:
Umbilical Cord Stem Cells - Current Uses & Future Challenges

Spinal Cord Stem Cells Comments Off on Umbilical Cord Stem Cells – Current Uses & Future Challenges | August 11th, 2017

The human body is an amazing thing. For each one of us, it's the most intimate object we know. And yet most of us don't know enough about it: its features, functions, quirks, and mysteries. Our series The Body explores human anatomy, part by part. Think of it as a mini digital encyclopedia with a dose of wow.

If you say someone's getting on your nerves, you could just cut to the chase and say they're getting on your sciatic nervethis nerve is plenty big enough for both minor and major irritations. It's the largest nerve in the body, running a lengthy route from each side of your lower spine, deep into your buttock, wrapping around to the back of the thigh and into the foot. Mental Floss spoke to Loren Fishman, medical director of Manhattan Physical Medicine and Rehabilitation in NYC andassociate clinical professor at Columbia Medical School. Here are 13 things we learned about this important part of the nervous system.

No wonder this nerve hurts when it gets irritatedat its biggest point, it's one heck of a large nerve, says Fishman.

The sciatic nerve is more accurately five nerves that come together on the right and left sides of the lower spine. Technically, the fourth and fifth lumbar nerves and the first three nerves in the sacral spine come together and merge into the unified sciatic.

"The sciatic nerve gives feeling and strength to the muscles and skin of the calf and foot, supplies sensation from the joints, bones, and just about everything else below the knee," says Fishman.

The nerve connects the spinal cord with the outside of the thigh, the hamstring muscles in the back of the thigh, and the muscles in your lower leg and feet. This is why sciatic nerve impingement often results in muscle weakness, numbness and/or tingling in the leg, ankle, foot, and toes.

After severe spinal cord injury, the nerve itself is often just fine, but the connection between it and the brain has been severed, Fishman says. Until now, there's been no way to fix such injuries, but "recent work with stem cells has begun to restore the connection in dogs and other animals."

A variety of lower back problems can lead to pain that radiates along the sciatic nerve. Most commonly, sciatica pain is caused when a herniated disc at the L5 (lower lumbar back) irritates the S1 (sacrum) nerve root in the lower spine. The exiting nerve roots are highly sensitive, and the bits of the disc that herniate contain inflammatory proteins such as interleukin and tumor necrosis factor that can also aggravate the nerve.

In a small number of people, a condition called cauda equina syndrome (so named because the nerve bundle at the base of the spinal cord resembles a horse's tail) can masquerade as sciaticabut it also usually causes weakness that extends to bowel or bladder incontinence and sometimes weakness or loss of sensation in the legs that gets progressively worse. In this case, immediate medical attention should be sought, and recovery may not be as quick as with common sciatica.

When the ancient Greek and Roman physicians were treating the pain we now commonly know as sciatica, they believed it stemmed from "diseases of the hip joint," according to a 2007 study in Spinal Cord. It wasn't until 1764, write the authors, "that leg pain of 'nervous' origin was distinguished from pain of 'arthritic' origin."

Among the many treatments Hippocrates and his ilk came up with for this painful condition were: "Fumigations, fasting, and subsequently, laxatives, and ingestion of boiled milk of the female ass." In his Treatise of the Predictions, Hippocrates noted that elderly patients with "cramps and colds at the loin and the legs" would experience their pain for up to a year, whereas young people could be free of pain in about 40 days.

The modern name for the disease, according to Fishman, comes from 15th-century Florence. "They called sciatica ischiatica, since they thought it came from tuberculosis that worked its way down to the ischial tuberosity (the sit-bones)," Fishman says. These medieval doctors had the cause wrong, but the name stuck.

Different researchers in different countries began to make sciatic breakthroughs when doing autopsies on corpses with fractured or herniated discs, where they noticed compression on the sciatic nerve.

A 1991 cross sectional study of 2946 women and 2727 men published in Spine found that neither gender nor body mass made any difference in the likelihood of developing sciatica. Body height did, however, in males between the ages of 50 and 64, with taller men being more likely to have the condition. Other studies have found a similar link [PDF]. Over 5'8"? Your risk is higher.

Sciatica has a surprisingly common negative impact on daily life. "Low back pain and sciatica are the second biggest reason for lost days of workjust behind the common cold," says Fishman.The condition is most commonly found in people over 50 andrarely seen in anyone under 20 years oldand then it most often has a genetic cause.

View original post here:
Attention Knitters: Oklahoma Needs 5000 Baby Hats - Mental Floss

Spinal Cord Stem Cells Comments Off on Attention Knitters: Oklahoma Needs 5000 Baby Hats – Mental Floss | August 11th, 2017

Share this story with your network

FREMONT, Calif., Aug. 08, 2017 (GLOBE NEWSWIRE) Asterias Biotherapeutics, Inc. (NYSE MKT:AST) today announced that Lucas Lindner of Eden, Wisconsin, a quadriplegic patient who has regained functional use of his fingers, hands and lower arms after receiving the companys investigational stem cell therapy for complete cervical spinal cord injury, AST-OPC1, will throw out the ceremonial first pitch of a Major League Baseball game in Milwaukee on Sunday, August 13th.

Lucas has been an inspiration to our employees at Asterias who have worked so hard to bring AST-OPC1 to where it is now being administered to patients in a clinical trial, as well as to thousands of others who have seen his story on the internet or on television, said Mike Mulroy, President and CEO of Asterias. We are excited about the progress he has made since receiving 10 million cells of AST-OPC1 and look forward to cheering him on as he takes the field before the game.

Lucas suffered a severe spinal cord injury when his car swerved off the road into a tree to avoid hitting a deer in May 2016. He was flown to the hospital and received immediate surgery to stabilize his spine. He was left without the ability to move his limbs below the neck and upper arms.

In June 2016, Lucas received 10 million cells of AST-OPC1 in Asterias ongoing SCiStar Phase 1/2a clinical study by Shekar N. Kurpad, MD, PhD, Sanford J. Larson Professor and Chairman, Department of Neurosurgery at the Medical College of Wisconsin and Director of the Froedtert & Medical College of Wisconsin Spinal Cord Injury Program. Lucas has since regained the ability to move triceps, hands and fingers.

As of his latest follow-up visit (12 months following administration of AST-OPC1), Lucas has achieved two motor levels of improvement on one side of his body. As suggested by existing research, patients with severe spinal cord injuries that show two motor levels of improvement on at least one side may regain the ability to perform daily activities such as feeding, dressing and bathing, which significantly reduces the overall level of daily assistance needed for the patient and associated healthcare costs.

Throwing out the first pitch at a Major League game is not something I could have imagined a year ago, said Lucas. I want to show everyone that there is hope that spinal cord injury patients can regain function. I am looking forward to going back to school, pursuing my dream of working in the IT field and living independently someday.

When I first met Lucas about a year ago, he had some use of his arms and little to no use of his hands or fingers, said Dr. Kurpad. The fact that he is throwing out the first pitch at a Major League Baseball game is amazing. It illustrates the strides medical science is starting to make in helping paralyzed patients regain useful function. Im very encouraged by the early results we are seeing with AST-OPC1 and am grateful for the improvement Lucas has made.

Asterias has now completed enrollment and dosing in four of the five planned SCiStar study cohorts and enrolled twenty-two patients in the SCiStar study. Twenty-seven patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial and results-to-date continue to support the safety of AST-OPC1. In June 2017, Asterias reported 9 month data from the AIS-A 10 million cell cohort that showed improvements in arm, hand and finger function observed at 3-months and 6-months following administration of AST-OPC1 were confirmed and in some patients further increased at 9-months. The company intends to complete enrollment of the entire SCiStar study later this year, with multiple safety and efficacy readouts anticipated during the remainder of 2017 and 2018.

To view a video on Lucas story, click on the following link:https://youtu.be/1DerDpM_FO4.

Broadcast quality b-roll footage is available for news media use by request by contactingmark@reachthenextlevel.com.

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 subacute, C-4 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 have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 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 eight 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, Santa Clara Valley Medical Center in San Jose jointly with Stanford University, Thomas Jefferson University Hospital, in partnership with Magee Rehabilitation Hospital, in Philadelphia, and UC San Diego Health in San Diego.

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 originally isolated in 1998, 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. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials. Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The companys 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 companys 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 atwww.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.

Follow this link:
Former Quadriplegic Enrolled in Asterias' SCiStar Study to Throw Ceremonial First Pitch at Major League Baseball Game - OrthoSpineNews

Spinal Cord Stem Cells Comments Off on Former Quadriplegic Enrolled in Asterias’ SCiStar Study to Throw Ceremonial First Pitch at Major League Baseball Game – OrthoSpineNews | August 11th, 2017

The new research has been developed by a team led by Dr. Samuel I. Stupp, who is the director of Northwestern Universitys Simpson Querrey Institute for BioNanotechnology. The researcher is also Professor of Materials Science and Engineering, Chemistry, Medicine and Biomedical Engineering.The new technology centers on the way that cells behave in the human body. Our cells are continually being signaled with various instructions, triggered by proteins and other molecules that are located in the matrices that surround them. As an example, such signals can be cues for cells to express specific genes in order for the cells to differentiate into other types of cells. Such a development is important for growth or regeneration of tissues. This sophisticated, biological signaling machinery has the pre-programmed capacity to make signals stop and re-start as needed; or to switch off one signal and activate an alternative signal in order to commence a complex processes. If this could be controlled by medics, then the process of addressing a range of diseases could be achieved. So far, the ability to produce such regenerative therapies has proved impossible.This could be set to change with the development of a synthetic material that can trigger reversibly certain types of signaling. This platform could lead to materials to control stem cells in order to produce regenerative therapies and to control cellular functions. The new technology should help with research into treatments for such diseases as Alzheimers disease, Parkinsons disease, problems with arthritic joints, spinal cord injuries, the effects of stroke, and other conditions requiring tissue regeneration.In trials, the researchers have taken spinal cord neural stem cells (neurospheres) and driven them to differentiate using a signal, helping the scientists to understand developmental and regenerative cues. This cell manipulation technology could help control which cells change and thereby address diseases like Parkinsons, such as converting a patients own skin cells into stem cells. Commenting on the implications of the technology, Dr. Stupp said, in a communication provided to Digital Journal: Its important in the context of cell therapies for people to cure these diseases or regenerate tissues that are no longer functional.The research is an example of the use of digital based bio-nanotechnology. The technology has been published in the journal Nature Communications. The paper Instructing cells with programmable peptide DNA hybrids.

See the article here:
New technology manipulates cells for disease research - Digital Journal

Spinal Cord Stem Cells Comments Off on New technology manipulates cells for disease research – Digital Journal | August 8th, 2017

A syrinx is a fluid-filled cavity within the spinal cord (syringomyelia) or brain stem (syringobulbia). Predisposing factors include craniocervical junction abnormalities, previous spinal cord trauma, and spinal cord tumors. Symptoms include flaccid weakness of the hands and arms and deficits in pain and temperature sensation in a capelike distribution over the back and neck; light touch and position and vibration sensation are not affected. Diagnosis is by MRI. Treatment includes correction of the cause and surgical procedures to drain the syrinx or otherwise open CSF flow.

Syrinxes usually result from lesions that partially obstruct CSF flow. At least half of syrinxes occur in patients with congenital abnormalities of the craniocervical junction (eg, herniation of cerebellar tissue into the spinal canal, called Chiari malformation), brain (eg, encephalocele), or spinal cord (eg, myelomeningocele). For unknown reasons, these congenital abnormalities often expand during the teen or young adult years. A syrinx can also develop in patients who have a spinal cord tumor, scarring due to previous spinal trauma, or no known predisposing factors. About 30% of people with a spinal cord tumor eventually develop a syrinx.

Syringomyelia is a paramedian, usually irregular, longitudinal cavity. It commonly begins in the cervical area but may extend downward along the entire length of the spinal cord.

Syringobulbia, which is rare, usually occurs as a slitlike gap within the lower brain stem and may disrupt or compress the lower cranial nerve nuclei or ascending sensory or descending motor pathways.

See the original post:
Syrinx of the Spinal Cord or Brain Stem - Neurologic ...

Spinal Cord Stem Cells Comments Off on Syrinx of the Spinal Cord or Brain Stem – Neurologic … | August 7th, 2017

Dr. Gabe Mirkin

Sam Shepard was a prolific playwright, actor, screenwriter and director who:

acted in more than sixty films and was nominated for an Academy Award for Best Supporting Actor for his portrayal of pilot Chuck Yeager inThe Right Stuff;

wrote more than 55 plays, often focusing on the serious problems that occur in American family life;

won the most Obie Awards (10) for his off-Broadway writing and directing. In 1979 he received a Pulitzer Prize for his play, Buried Child, andNew York Magazinecalled him the greatest American playwright of his generation.

In his late sixties, he developed amyotrophic lateral sclerosis (ALS), the disease that killed baseball great Lou Gehrig at age 37. Shepard died from complications of ALS on July 27, 2017, at age 73.

A Difficult Life

Sam Shepard

He was born on November 5, 1943, in Fort Sheridan, Illinois. His dysfunctional family served as a basis for characters in many of his plays. His father was a United States Army Air Forces bomber pilot during World War II who was also an alcoholic and an abusive husband and father. His loving, supportive mother, a teacher, offset some of the pain and abuse he suffered from his father. In his early years, the family had to move every two years because of army transfers. Later his father left the service and bought an avocado farm in Duarte, Calif. Shepard briefly studied animal husbandry at nearby Mt. San Antonio College, but soon left school to move to New York City, where he worked as a busboy, played in a psychedelic folk band and tried to break into the theater.

At age 35, his acting career took off when he won a role in Terrence MalicksDays of Heaven, with Richard Gere and Brooke Adams. At the same time, he continued to write successful plays and in 1986 (age 43) he was elected to the American Academy of Arts and Letters.

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrigs Disease)

In his last few years, Shepard suffered privately from ALS, but he described his experience in his last book, The One Inside. One of the characters said that he couldnt get up from bed in the morning and felt as though his limbs werent connected to the motor driving his body. They wont take direction wont be dictated to the arms, legs, feet, hands. Nothing moves. Nothing even wants to. The brain isnt sending signals.

ALS is a progressive disease that destroys the nerves that move voluntary muscles. More than 6,000 people in the United States are diagnosed with ALS each year. Nobody knows the cause and there is no cure. The brain is supposed to send messages to nerves in the spinal cord which transmit messages to the nerves that move muscles. When a muscle loses its nerve control, it starts to twitch and can waste away to nothing. Early symptoms of ALS include

muscle weakness

twitching

slurred speech

inability to chew food

tripping or stumbling.

The first sign could be difficulty buttoning a shirt, writing, or turning a key in a lock. The disease usually does not affect a persons ability to think and reason, so affected people are terribly disturbed by their lack of ability to control their voluntary muscles. As the disease progresses, a person loses the ability to speak, eat, walk, and eventually breathe. The most common cause of death is inability to breathe, which typically occurs about 3-5 years after symptoms start. Only about ten percent of affected people live more than ten years after first being diagnosed.

Risk Factors and Diagnosis

The disease usually starts between the ages of 55 and 75, but there are no known specific risk factors. Military veterans appear to be twice as likely as non-veterans to develop ALS. Possible causes could be exposure to occupational or environmental toxins such as lead or pesticides, infections or trauma. Family history does not appear to predict the disease.

There are no specific tests to diagnose ALS. It is usually diagnosed by a history of the symptoms, physical examination and ruling out other causes.

Current Treatments and Research

The U.S. Food and Drug Administration (FDA) has approved riluzole (Rilutek) and edaravone (Radicava) to treat ALS. These drugs offer no hope for a cure, but Riluzole appears to protect nerves by decreasing glutamate, the chemical messenger for nerves that innervate muscles. Intravenous edaravone possibly slows loss of muscle function, but it costs $1,086 per infusion or a yearly cost before government discount of $145,524. Another drug under European review is being developed by French drug maker AB Science SA (ABS.PA). Since there is no cure, all patients should receive physical therapy and speech therapy because inactivity itself causes loss of muscle function.

Since ALS is caused by the death of nerve cells that cause muscles to contract, the most promising line of research is through stem cells. Stem cells are young cells that can become any type of tissue. Treatment in the future may be to program stem cells to become nerve cells that innervate muscles and then inject them into areas where the nerve cells have already died.

Dr. Gabe Mirkin is a Villager. Learn more at http://www.drmirkin.com

Read the rest here:
Sam Shepard and Amyotrophic Lateral Sclerosis - Villages-News

Spinal Cord Stem Cells Comments Off on Sam Shepard and Amyotrophic Lateral Sclerosis – Villages-News | August 7th, 2017

FREMONT, Calif., Aug. 03, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced that two additional clinical sites have opened to enroll subjects for the companys ongoing SCiStar Phase 1/2a clinical study of AST-OPC1 in complete cervical spinal cord injury (SCI). The additional clinical sites include: Thomas Jefferson University Hospital, in partnership with Magee Rehabilitation Hospital, in Philadelphia, PA; and UC San Diego Health in San Diego, CA. Asterias now has eight clinical sites throughout the country enrolling patients in the study.

We are excited about the clinical site openings at Thomas Jefferson University Hospital and UC San Diego Health, stated Dr. Edward Wirth III, Chief Medical Officer of Asterias Biotherapeutics. These sites provide additional geographical reach and previous experience with spinal cord injury trials to our SCiStar study. We have recently reported completion of enrollment in four out of five cohorts in our SCiStar study so we hope these institutions will also participate in a future, larger study of AST-OPC1.

Each of the two additional clinical sites is recognized in the treatment of SCI:

The two additional clinical sites join existing clinical sites for the SCiStar study at 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 completed enrollment and dosing in four of the five planned SCiStar study cohorts and enrolled twenty patients in the SCiStar study. Twenty-five patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial and results-to-date continue to support the safety of AST-OPC1. In June 2017, Asterias reported 9 month data from the AIS-A 10 million cell cohort that showed improvements in arm, hand and finger function observed at 3-months and 6-months following administration of AST-OPC1 were confirmed and in some patients further increased at 9-months. The company intends to complete enrollment of the entire SCiStar study later this year, with multiple safety and efficacy readouts anticipated during the remainder of 2017 and 2018.

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 subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) 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 have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which has provided $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 originally isolated in 1998, 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 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. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials. Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

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.

Read more:
Asterias Biotherapeutics Opens Two Additional Clinical Sites for ... - GlobeNewswire (press release)

Spinal Cord Stem Cells Comments Off on Asterias Biotherapeutics Opens Two Additional Clinical Sites for … – GlobeNewswire (press release) | August 4th, 2017