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.

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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.

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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

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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

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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

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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.

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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.

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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.

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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

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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.

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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.

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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.

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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

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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.

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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

School of Public Health (SPH) student Jake Maxon (Burnsville, MN) became interested in policy while working among microscopes and petri dishes. After getting his neuroscience degree from Brown University, Maxon researched spinal cord injuries, which involved stem cell research.

Policy around stem cells brought a different kind of challenge to our research, and I wanted to know more about how policies are created and implemented, he says.

So Maxon enrolled in SPHs Public Health Administration and Policy program, where hes been able to work with many Twin Cities policy organizations, including as a grant reviewer for the Ryan White HIV/AIDS Program and as a policy intern for Hennepin County.

But he wanted to understand policy on a federal level. So he applied to the White House Internship Program. After a six-month selection and vetting process, he was assigned to work alongside the three-person team in the Office of National AIDS Policy, which works to create an integrated approach to the prevention, care, and treatment of HIV/AIDS.

Weve advanced medical care for HIV/AIDS patients, so many people in our country forget that HIV is still an epidemic and its still a public health crisis, says Maxon. More than 4,300 residents in Hennepin County alone are currently living with HIV/AIDS.

While in Washington, D.C., Maxon continued working on his degree and now, nearly finished, finds himself in a new role as coordinator for Hennepin Countys HIV Positively Hennepin strategybased off of the national strategy he helped implement at the White House.

The vision is to create a county where all residents living with HIV/AIDS have healthy, vibrant lives, where there is equitable access to HIV prevention and care, and where there are no new HIV infections.

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A bold vision - UMN News

Spinal Cord Stem Cells Comments Off on A bold vision – UMN News | August 2nd, 2017

Invivo Therapeutics (NASDAQ:NVIV) (OTCQB:NVIVD) has paused patient enrollment in the approval trial of its bioresorbable spinal implant, Neuro-Spinal Scaffold, after a third patient in the study died. The company states that all three deaths have been determined to be unrelated to the product or the implantation procedure, used in patients rendered paraplegic by a spinal cord injury, but the company's shares tanked 27%.

Unless Invivo can somehow shake off the product's worrisome reputation - to this end, it is talking with the FDA to see if enrollment criteria ought to be changed or the study altered in other ways - it will have to rely on its one remaining product, an injected spinal cord injury therapy based on neural stem cells. But this is still in animal trials, so Invivo really needs its Neuro-Spinal Scaffold to be vindicated.

This is the second disappointment for the company in its attempt to develop this project. Four years ago the FDA refused to let it file on data from a pilot study, setting the approval date back by some years (InVivo Therapeutics suffers from FDA's timidity on biologic grafts, August 29, 2013).

The Neuro-Spinal Scaffold is made of two polymers, polylactic-co-glycolic acid and poly-L-lysine. It is implanted at the site of a spinal cord injury to provide structural support and a matrix through which the patient's neural tissue can regrow, after which the graft breaks down over several weeks.

The Inspire trial is testing its safety and probable benefit for the treatment of complete spinal cord injury at the T2-T12 and L1 positions - from roughly shoulder level to just above the waist. The primary endpoint is improvement of one or more grades on the on the American Spinal Injury Association impairment scale (AIS) at six months after implantation. The study is slated to enrol 20 patients, according to Clinicaltrials.gov, and is intended to support a filing for US approval via the humanitarian device exemption (HDE) route.

The most recent patient to sign up to the Inspire study underwent implantation in late June but died suddenly at a healthcare facility following discharge from the hospital.

Invivo could hardly be blamed for pointing out that some of the patients in the Inspire trial had had positive outcomes. One had improved from a complete injury (grade A on the AIS) to having some restored sensory and motor function (grade C) one month after treatment. Another had regained sensory but not motor function (grade B) at six months.

One patient who had improved from a complete injury (grade A) to having sensory function (B) at two months reverted to complete injury at three months, but was deemed to have regained this motor function at the six-month point. The company says that of the 16 patients currently in follow-up seven have improved on the AIS, four of whom have recovered both sensory and motor function to reach grade C.

Five further patients had not improved at six months, and four had shown no improvement but had not yet reached this point.

With trial enrollment on hiatus Invivo will have to wait to find out whether these results might be sufficient to get the graft an HDE approval. The company now hopes to complete enrollment in the first half of next year, and to file its FDA approval application in the second half of 2018.

Use of the Neuro-Spinal Scaffold in complete and incomplete spinal cord injury, at cervical and thoracic levels, is forecast to bring Invivo revenues of $268m in 2022, according to EvaluateMedTech's consensus. By 2022 the sellside sees it outsold by Invivo's only other product, a biomaterial-based scaffold used to deliver neural stem cells to help reconnect the spinal cord by re-growing nerves.

But the scaffold is the more advanced product, and Invivo will be relying on revenues from this to fund clinical development of the stem cell therapy. The trial delay puts this in jeopardy, as the company's shareholders are well aware.

Editor's Note: This article covers one or more stocks trading at less than $1 per share and/or with less than a $100 million market cap. Please be aware of the risks associated with these stocks.

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Third Trial Death Endangers Invivo - Seeking Alpha

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The hypothalamus, a command center deep in the brain, helps control everything from hunger to sleep.

Roger Harris/Science Source

By Mitch LeslieJul. 26, 2017 , 1:00 PM

If these sweltering summer days prompt you to reach for a cold drink, you can thank your hypothalamus, a region of the brain that helps us regulate body temperature and other internal conditions. Butthe region may failus when we get older. A new study in mice suggests that the hypothalamus promotes aging, hastening physical and mental decline as its stem cells die off.

Its a pretty stunning paper, says Charles Mobbs, a neuroendocrinologist at the Icahn School of Medicine at Mount Sinaiin New York City. The new aging mechanism is totally novel and quite unexpected, adds neuroendocrinologist Marianna Sadagurski of Wayne State University in Detroit, Michigan.

Tucked away deep in the brain, the hypothalamus monitors and maintains our blood concentration, our body temperature, and other physiological variables. Researchers have also suspected that it plays a role in aging. The hypothalamus becomes inflamed as we get older, and 4 years ago a team led by neurodendocrinologist Dongsheng Cai of Albert Einstein College of Medicine in New York City showed that quelling this inflammation delays physical deterioration and boosts life span in mice.

In the new study, the team turned its attention to the hypothalamuss stem cells, which in young animals divide to produce replacements for dead and damaged cells. As mice get older, the scientists found, the number of stem cells in the hypothalamus plunges. By later ages they are basically all gone,Cai says.

To determine whether this loss promotes aging, researchers tried to speed up the process, genetically altering mice so that stem cells in the hypothalamus died when the animals were dosed with an antiviral drug. Knocking off some 70% of the cells shortened the mices lives by about 8%, the team reports today in Nature. The mices memory, coordination, and endurance also suffered. Behaviorally, they were like grumpy grandparents, less social and curious than youthful rodents. For example, when researchers put a new object into their cages, controlmice spent about twice as long exploring it than did their modified counterparts.

Next, the team tried to reverse this deterioration by injecting stem cells into the hypothalami of middle-aged animals. Mice that received the stem cells outlived mice injected with a different type of brain cell by more than 10%, and they retained more of their physical and mental capabilities. In humans, the extra boost could mean a few more years of healthy life, Mobbs notes.

Researchers assume the loss of stem cells causes organs and tissues to wear out gradually because they cant replenish their lost cells. But because injecting stem cells into the mice produced benefits quickly, Cai and his colleagues concluded a faster-acting mechanism was at work.

Their suspicions fell on RNA molecules known as microRNAs, which stem cells manufacture and release. These microRNAs ferry messages to other cells, altering which proteins they produce. The researchers found that stem cells from the hypothalamus pump out huge amounts of microRNAs, packaged in tiny containers called exosomes. They also found that injecting mice with microRNA-rich exosomes isolated from cultures of young hypothalamus stem cells slowed the animals physical and cognitive breakdown almost as much as injections of stem cells.

The big question is how those microRNAs influence function, Mobbs says. The molecules could spur other cells to curb inflammation or stress, Cai says, though he isnt certain how they work. Where the microRNAs exert their effects is also a mystery. Their targets may be other cells in the brain or the spinal cord, but they might also slip into the bloodstream and prod cells elsewhere in the body.

The work suggests that protecting or replacing the hypothalamuss stem cellsor replicating the effects of the microRNAscould slow aging in humans. It might also be possible to suppress the inflammation that provokes the stem cell die-off, Sadagurski says. She says some current drugs, including the diabetes treatment acarbose, curb inflammation in the hypothalamus and may be worth testing.

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The breakdown of this brain region may accelerate aging - Science Magazine

Spinal Cord Stem Cells Comments Off on The breakdown of this brain region may accelerate aging – Science Magazine | July 31st, 2017

Some big scientific breakthroughs are set to take place this year.

And fortunes could be made with a few off-the-radar biotech pioneers.

What I am talking about are the handful of small biotech companies applying regenerative medicine platforms to degenerative diseases and ailments like paralysis.

These degenerative diseases have always been tough to treat, and few therapies are available. Thats not surprising, since weve lacked until recently the ability to create healthy and functioning human cells to replace ones lost in the body to injury and disease.

Now, though, a handful of breakthrough companies are aiming to correct that lack with new pluripotent stem cell technology.

Stem cells have the ability to reproduce and change into functional cells and tissues. And pluripotent stem cells are the most potent. They can turn into any cell type in the human body. Furthermore, they can divide and reproduce without end.

This makes them an ideal starting point for manufacturing cell-based therapies.

One company using this platform to help heal spinal cord injuries and paralysis is already heaving great success in FDA trials with a new cell line.

Their recent FDA Phase 1 trial data shows that patients treated with a new cell line are seeing a significant return of nerve function thanks to these grafts.

And last month, this same company announced nine-month follow-up data for patients that were given a 10 million cell dose to their injury. Even though this is only half of what researchers believe will be a full dose, at least 50% of patients have already shown signs of recovery.

This includes two levels of improvement in motor function, as well as improvements in arm, hand and finger function.

The nine-month data confirms what the company reported at three and six months, too.

Showing us that the new cell therapy has great durability. The introduced cells help heal the injury, and the result is more than temporary.

Under the accumulating evidence, the FDA has also decided to approve an expansion to this trial to include patients with spinal cord injury at the C4 location which is in the middle of the neck.

This is a very significant development.

The C4 spinal location is one of the most common locations for a spinal cord injury, and it often results in paralysis from the neck down.

And now the FDA is allowing the company to expand its treatment window from 1430 days after injury to 2142 days.

All these data bode very nicely for the future.

And not only for the developers of this specific cell therapy, but for other small biotech companies also trying to carve out market share in the pluripotent stem cell therapy space.

According to the National Spinal Cord Injury Statistical Center, there are some 17,000 new spinal cord injuries per year. This represents a large potential market for any company that masters this new technology and provides viable treatments for degenerative injuries.

Even more promising is that this new cell technology may also be used to treat cancer. And another group of tiny biotech companies are leading the way in research for this application, too.

We hope to see the data from these other FDA trials focusing on developing cancer vaccines during the second half of 2017.

If any of these new trial results comes back positive as early indications are showing, then were looking a tsunami sized wave of new opportunities in biotech. And for the handful of small companies and their investors, this should be a game changer.

As it stands currently, most of the companies making the push into regenerative medicine using new stem cell technology remain largely undiscovered.

As the FDA data become more actionable and these treatments move closer to market introduction, there will be some great plays to make.

And when that happens Ill be right here to make sure you have the best chance for the big score with this new technology.

To a bright future,

Ray Blancofor The Daily Reckoning

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Off-the-Radar Biotechs About to Break Out - Daily Reckoning

Spinal Cord Stem Cells Comments Off on Off-the-Radar Biotechs About to Break Out – Daily Reckoning | July 31st, 2017

NEW YORK, July 12, 2017 /PRNewswire/ -- The global stem cell market is expected to reach USD 15.63billion by 2025, growing at a CAGR of 9.2%, according to a new report by Grand View Research, Inc.

Augmentation in research studies that aim at broadening the utility scope of associated products is anticipated to drive the market growth. These research projects have opened the possibility of implementation of several clinical applications of these cells, thereby impacting disease-modifying treatments.

Read the full report: http://www.reportlinker.com/p04998556/Stem-Cells-Market-Analysis-By-Product-Adult-Stem-Cells-hESC-Induced-Pluripotent-Stem-Cells-By-Application-Regenerative-Medicine-Drug-Discovery-By-Technology-By-Therapy-And-Segment-Forecasts.html

Scientists are engaged in discovering novel methods to create human stem cells. This is to address the increasing demand for stem cell production for potential investigation in disease management. This factor is certainly expected to accelerate the development of regenerative medicine, thus driving industrial growth.

Moreover, cellular therapies are recognized as the next major advancements in transforming healthcare. Firms are expanding their cellular therapy portfolio, understanding the future potential of this arena in the treatment of Parkinson's disease, type 1 diabetes, spinal cord injury, Alzheimer's disease, and others.

In March 2016, Scientists at Michigan State University unveiled new kind of cells "induced XEN cells" from a cellular trash pile. This discovery is expected to drive advancements in regenerative medicine. Such discoveries are anticipated to bolster research and sales in this market over the forecast period.

Further key findings from the report suggest: Adult stem cells dominated the market and is expected to maintain its dominance. This can be attributed to the several factors such as lower rejection rates, long term renewal property, and no ethical concerns associated with their usage Application wise regenerative medicine is estimated to hold the substantial share of the revenue Presence of significant number pipeline projects for regenerative medicine is expected to fuel growth in the market In addition, exploding research projects have driven the need of harvesting techniques, thereby propelling progress of acquisition technology Increased R&D activities and huge funds granted by funding bodies to advance cellular research in the U.S. have resulted into the large share of North America Extensive research carried out in Singapore and Japan is anticipated to drive progress with lucrative avenues Advanced Cell Technology Inc, Osiris Therapeutics Inc, Celgene Corporation, BIOTIME, INC., Cynata, and STEMCELL Technologies Inc., are some of the major companies operating in this market A number of companies are engaged in seeking investment from overseas organizations and also developing partnerships with the pharmaceutical organizations

Read the full report: http://www.reportlinker.com/p04998556/Stem-Cells-Market-Analysis-By-Product-Adult-Stem-Cells-hESC-Induced-Pluripotent-Stem-Cells-By-Application-Regenerative-Medicine-Drug-Discovery-By-Technology-By-Therapy-And-Segment-Forecasts.html

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Stem Cells Market Analysis By Product (Adult Stem Cells, hESC ... - PR Newswire (press release)

Spinal Cord Stem Cells Comments Off on Stem Cells Market Analysis By Product (Adult Stem Cells, hESC … – PR Newswire (press release) | July 13th, 2017

According to recent study, advancements in materials from this study could potentially help patients requiring stem cell therapies for spinal cord injuries, stroke, Parkinsons disease, Alzheimers disease, arthritic joints or any other condition requiring tissue regeneration. Earlier research revolved around the role of autoimmunity in terms of a treatment.

Its important in the context of cell therapies for people to cure these diseases or regenerate tissues that are no longer functional, shared Samuel I. Stupp, director of Northwesterns Simpson Querrey Institute for BioNanotechnology and Board of Trustees Professor of Materials Science and Engineering, Chemistry, Medicine and Biomedical Engineering.

Cells in our bodies are constantly being signalled with many types of instructions coming from proteins and other molecules present in the matrices that surround them. For example, these can be cues for cells to express specific genes so they can proliferate or differentiate into several types of cells leading to growth or regeneration of tissues. One of the marvels of this signalling machinery is the built-in capacity in living organisms to make signals stop and restart as needed, or to switch off one signal and activate a different one to orchestrate very complex processes.

Building artificial materials with this type of dynamic capacity for regenerative therapies has been virtually impossible so far. The new work published today reports the development of the first synthetic material that has the capability to trigger reversibly this type of dynamic signalling. The platform could not only lead to materials that manage stem cells for more effective regenerative therapies, but will also allow scientists to explore and discover in the laboratory new ways to control the fate of cells and their functions.

One of the findings is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the operator, trigger their differentiation into neurons and then return the stem cells back to a proliferative state on demand. The paper also reports that spinal cord neural stem cells, initially grouped into structures known as neurospheres, can be driven to spread out and differentiate using a signal.

But when this signal is switched off, the cells spontaneously regroup themselves into colonies. This uncovers strong interactions among these cells that could be important in understanding developmental and regenerative cues. The potential use of the new technology to manipulate cells could help cure a patient with 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 in vitro in the lab and then drive their differentiation into dopamine-producing neurons before transplantation back to the patient. In the new technology, materials are chemically decorated with different strands of DNA, each designed to display a different signal to cells.

People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue. 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, noted Stupp. While this process is currently only done in vitro with the vision of then transplanting cells, Stupp said in the future it might be possible to perform this process in vivo.

The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, via an injection and targeted to a particular spot. Then the soluble molecules would be given to the patient to manipulate proliferation and differentiation of transplanted cells. The study was published in journal Nature Communications.

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Cell therapy may be key to treating Alzheimer's & Parkinson's - Economic Times

Spinal Cord Stem Cells Comments Off on Cell therapy may be key to treating Alzheimer’s & Parkinson’s – Economic Times | July 13th, 2017

"The FDA's decision to allow Asterias to enroll qualified patients with C-4 level injuries is the result of the data supporting the safety of both AST-OPC1 and the procedure to inject the cells, and means that the second most common cervical spinal cord injury population can now be eligible to receive AST-OPC1," said Dr. Edward Wirth, Chief Medical Officer of Asterias. "The overall changes to the study protocol will enhance our ability to enroll qualified patient candidates for our current SCiStar study and we also expect the changes to help enrollment rates in a future, larger clinical study."

The protocol amendment will expand patient eligibility and enable study investigators to administer AST-OPC1 to patients with injuries at one vertebral level higher than the trial's previous C-5 limitation, to the fourth cervical vertebra down, known as C-4, near the middle of the neck. A C-4 cervical level injury, the second most common level of SCI in the SCiStar study's targeted patient population, generally means that the injured person is paralyzed from the neck down and requires round-the-clock care. The lifetime direct costs of care for a patient suffering a high cervical spinal cord injury, such as a C-4 spinal cord injury, can approach $5 million. As suggested by existing research, if these patients can show two motor levels of improvement on at least one side they may regain the ability to perform daily activities such as feeding, dressing and bathing, which increases their quality of life and independence and significantly reduces the overall level of required daily assistance and associated healthcare costs for these patients.

In addition, the amendment to inclusion parameters will also expand the dosing window to 21 to 42 days after a patient's spinal cord injury occurs, providing study investigators more time to screen patients to determine if they are eligible to participate in the SCiStar study. The expansion of the dosing window is supported by recent preclinical research that indicated AST-OPC1 cells can durably engraft at a patient's injury site when administered up to two months after the date of injury.

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 six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells 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 two cohorts of this study. Results to date have continued to support the safety of AST-OPC1, with no serious adverse events related to AST-OPC1 or its administration. 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.

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SOURCE Asterias Biotherapeutics, Inc.

http://www.asteriasbiotherapeutics.com

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Asterias Biotherapeutics Receives FDA Clearance to Enroll C-4 Patients in SCiStar Study - PR Newswire (press release)

Spinal Cord Stem Cells Comments Off on Asterias Biotherapeutics Receives FDA Clearance to Enroll C-4 Patients in SCiStar Study – PR Newswire (press release) | July 11th, 2017

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Stem cell science is an area of medical research that continues to offer great promise. But as this weeks paper in Science Translational Medicine highlights, a growing number of clinics around the globe, including in Australia, are exploiting regulatory gaps to sell so-called stem cell treatments without evidence that what they offer is effective or even safe.

Such unregulated direct-to-consumer advertising typically of cells obtained using liposuction-like methods not only places the health of individuals at risk, but could also undermine the legitimate development of stem cell-based therapies.

Many academic societies and professional medical organisations have raised concerns about these futile and often expensive cell therapies. Despite this, national regulators have typically been slow or ineffective in curtailing them.

As well as tighter regulations here, international regulators such as the World Health Organisation and the International Council on Harmonisation need to move on ensuring patients desperate for cures arent sold treatments with limited efficacy and unknown safety.

Hundreds of stem cell clinics post online claims that they have been able to treat patients suffering from a wide range of conditions. These include osteoarthritis, pain, spinal cord injury, multiple sclerosis, diabetes and infertility. The websites are high on rhetoric of science often using various accreditation, awards and other tokens to imply legitimacy but low on proof that they work.

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Donna McWilliam/APRather than producing independently verified results, these clinics rely on patient testimonials or unsubstantiated claims of improvement. In so doing these shonky clinics understate the risks to patient health associated with these unproven stem cell-based interventions.

Properly administered informed consent is often overlooked or ignored, so patients can be misled about the likelihood of success. In addition to heavy financial burdens imposed on patients and their families, there is often an opportunity cost because the time wasted in receiving futile stem cells diverts patients away from proven medicines.

The many recent reports of adverse outcomes demonstrate the risks of receiving unproven cell therapies are not trivial. In the USA three women were blinded following experimental stem cell treatment for macular degeneration (a degenerative eye disease that can cause blindness). One man was rendered a quadriplegic following a stem cell intervention for stroke. And a woman whose family sought treatment for her dementia died in Australia.

Other notorious cases involving the deaths of patients include the German government shutting down the X-Cell Centre and the Italian government closing the Stamina Foundation it had previously supported.

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REUTERS/Juan Carlos UlateAt present, the only recognised stem cell treatments are those utilising blood stem cells isolated from bone marrow, peripheral blood (the cellular components of blood such as red and white blood cells and platelets) or umbilical cord blood.

Hundreds of thousand of lives have been saved over the last half-century in patients with cancers such as leukaemia, lymphoma and multiple myeloma, as well as rare inherited immune and metabolic disorders.

A few types of cancer and autoimmune diseases may also benefit from blood stem cells in the context of chemotherapy. Different stem cells are also successfully used for corneal and skin grafting.

All other applications remain in the preclinical research phase or are just starting to be evaluated in clinical trials.

Often dismissed by for-profit clinics as red tape hampering progress, the rigour of clinical trials allows for the collection of impartial evidence. Such information is usually required before a new drug or medical device is released into the marketplace. Unfortunately, in the case of for-profit stem cell clinics, their marketing has gazumped the scientific evidence.

Action is required on many fronts. Regulators at both an international and national level need to tackle regulatory loopholes and challenge unfounded marketing claims of businesses selling unproven stem cell interventions.

Researchers need to more clearly communicate their findings and the necessary next steps to responsibly take their science from the laboratory to the clinic. And they should acknowledge that this will take time.

Patients and their loved ones must be encouraged to seek advice from a trained reputable health care professional, someone who knows their medical history. They should think twice if someone is offering a treatment outside standards of practice.

The stakes are too high not to have these difficult conversations. If a stem cell treatment sounds too good to be true, it probably is.

NOW WATCH: America's B-2 stealth bomber is unlike any military aircraft in the world

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Private clinics are peddling untested stem cell treatments it's unethical and dangerous - Yahoo News UK

Spinal Cord Stem Cells Comments Off on Private clinics are peddling untested stem cell treatments it’s unethical and dangerous – Yahoo News UK | July 9th, 2017